Mallinckrodt Pharmaceuticals
01/20/2021 jjb
Mallinckrodt Update to the NPC Community on Adrabetadex As previously mentioned during Mallinckrodt’s webinar on July 16th 2020, Dr. Steve Romano, Mallinckrodt’s Executive Vice President and Chief Scientific Officer, has prepared the following video for the NPC community which provides an update on Adrabetadex. CLICK HERE for video update.
09/03/2020 jjb
Mallinckrodt Message to the NPC Community: Update on Adrabetadex clinical studies Please take a moment to read this important announcement from Mallinckrodt Pharmaceuticals in regard to the clinical trial of Adrabetadex for NPC-1. Dr. Elizabeth Berry-Kravis and MNK are hosting a webinar this evening, Thursday, July 16 at 8 PM EST to answer questions from the NPC community. The link is https://notredame.zoom.us/j/93668717914?pwd=QzlLNThRdkJ3b0VUMnVLWUREVHd0UT09 and the password is 050409. CLICK HERE for complete announcement.
07/16/2020 jjb
Mallinckrodt Announces Agreement in Principle for Global Opioid Settlement Mallinckrodt Pharmaceuticals has provided the following update: Mallinckrodt has reached a broad based agreement in principle on the terms of a global settlement that would resolve all opioid-related claims against Mallinckrodt, Specialty Generics, and their other subsidiaries. The terms of the agreement include placing their Specialty Generics business in Chapter 11, however the Specialty Brands business is not included in this process. Mallinckrodt is continuing to operate as normal and is committed to improving patient lives with innovative therapies and supporting their advocacy partners. CLICK HERE for complete announcement. CLICK HERE for additional information.
02/27/2020 jjb
Mallinckrodt Message to NPC Community: Update on VTS-270 CLICK HERE for message.
10/29/2019 jjb
Mallinckrodt VTS301 Study Resumes in France CLICK HERE FOR Complete Annoucement
10/14/2019 jjb
Mallinckrodt VTS301 Study Resumes in the UK CLICK HERE FOR Complete Annoucement
10/07/2019 jjb
Mallinckrodt Update on Phase 2b/3 Study Suspension in UK and France CLICK HERE FOR UPDATE
09/13/2019 jjb
Mallinckrodt Clinical Trial update Letter to NNPDF Regarding VTS301 Phase 2b/3 Study Suspension in UK and France
08/02/2019 jjb
Mallinckrodt responses to questions from NPC community about suspension of VTS-270 301 in UK and France08/09/2019 jjb
Mallinckrodt VTS-270-303 Phase 2 Update CLICK HERE FOR Complete Annoucement
05/09/2019 jjb
NNPDF Industry Update Webinar Series featuring Mallinckrodt CLICK HERE FOR RECORDING
03/26/2019 jjb
Mallinckrodt Investor Call VTS-270 Update CLICK HERE FOR Complete Annoucement
02/26/2019 jjb
Mallinckrodt VTS-270 Phase 2b.3 Q & A Response Following Mallinckrodt’s announcement regarding early phase data from their VTS-270 study on November 6th, a comprehensive list of questions posed by the global NPC community, was submitted to the company. You will find Mallinckrodt’s response to these questions accompanied by a video presentation from Mallinckrodt’s Chief Scientific Officer, Steve Romano at the following links. Mallinckrodt VTS-270 Q & A Response Video Message from Mallinckrodt to NPC Community Please contact the NNPDF for further information by email at nnpdf@nnpdf.org or telephone (920) 563-0930.
11/30/2018 jjb
Mallinckrodt VTS-270 Phase 2b.3 Data Announcement CLICK HERE FOR Complete Annoucement
11/06/2018 jjb
Mallinckrodt Committed to Niemann-PickDisease Type C1 Patients, Therapy Click here for complete announcement
06/01/2018 jjb
Mallinckrodt Completes Acquisition of Sucampo Pharmaceuticals, Inc. Click here for complete announcement
2/13/2018 cdk
UPDATE FROM THE NNPDF BOARD Notification of Intended Pharmaceutical Acquisition The Board would like to make everyone aware of the planned acquisition of Sucampo Pharmaceuticals by Mallinckrodt Pharmaceuticals, which was announced earlier this week. We also look forward to learning about this acquisition and the plans for working with our community. As information becomes available, we will share it with you. As a reminder to families, we will continue to share information in the format below:
- The office posts information received about trials and expanded access therapies from companies on the NNPDF website under the appropriate pharmaceutical company..
- When information is received, it is posted to the website and a summary post is made on the NNPDF Facebook Page and/or NNPDF Awareness Group, with a link back to our website where you can find more detailed information. .
- All updates from pharmaceutical companies are included in the next monthly e-newsletter with links back to the original posting on the website. .
- All those interested in receiving this important and educational information should subscribe to the NNPDF Blog via the website.
12/29/2017 jjb
Vtesse May 2017 Newsletters are Now Available Online Click below for different languages
06/19/2017 jjb
Sucampo Acquires Vtesse Inc. Acquisition provides Sucampo with VTS-270, which is in a pivotal study for the treatment of Niemann-Pick disease Type C1 (NPC-1) Click here for full press release Vtesse ANNOUNCEMENT LETTER IN VARIOUS LANGUAGES SUCAMPO-Vtesse FACT SHEET
04/04/2017 jjb
Vtesse February/March Newsletters are Now Available Online Click below for different languages
03/17/2017 jjb
Vtesse Completes Enrollment of Phase 2b/3 Pivotal Clinical Trial of VTS-270 in Niemann-Pick Type C1 Disease Click here for complete announcement
03/15/2017 jjb
Vtesse January Newsletters are Now Available Online Click below for different languages
02/16/2017 jjb
Vtesse JANUARY 2017 Newsletter Update on the Clinical Trial of VTS-270: The NPC Study Vtesse JANUARY 2017 Newsletter
02/13/2017 jjb
Vtesse Press Release Vtesse Announces Dosing of First Patient in Australia in Phase 2b/3 Clinical Trial of VTS-270 in Niemann-Pick Type C1 Disease Click here for full release
01/10/2017 jjb
Vtesse December Newsletters are Now Available Online Click below for different languages
01/06/2017 jjb
Vtesse Announces Appointment of Jason Meyenburg as Chief Commercial Officer This new position has been created to help provide access to VTS-270 for children with Niemann-Pick Type C1 Disease upon approval by regulatory authorities. Click below for the complete announcement. Complete Annoucement
01/05/2017 jjb
Vtesse DECEMBER 2016 Newsletter Update on the Clinical Trial of VTS-270: The NPC Study Vtesse DECEMBER 2016 Newsletter
01/05/2017 jjb
Vtesse Press Release Vtesse Receives Rare Pediatric Disease Designation by the FDA for VTS-270 for the Treatment of Niemann-Pick Type C1 Disease Click here for full press release
11/29/2016 jjb
Vtesse October Newsletters are Now Available Online Click below for different languages
11/15/2016 jjb
Vtesse Press Release Medicines and Healthcare Products Regulatory Agency (“MHRA”) Designates VTS-270 as a Promising Innovative Medicine Updated with PIM Designation Explanation Click here for full press release CLICK HERE FOR PIM DESIGNATION EXPLANATION
11/10/2016 jjb
Vtesse Press Release Medicines and Healthcare Products Regulatory Agency (“MHRA”) Designates VTS-270 as a Promising Innovative Medicine Click here for full release
11/02/2016 jjb
Vtesse Press Release Vtesse Announces Dosing of First Patient in Germany for Phase 2b/3 Clinical Trial of VTS-270 in Niemann-Pick Type C1 Disease Click here for full release
10/05/2016 jjb
Vtesse September 2016 Frequently Asked Questions Click below for FAQs in different languages
09/27/2016 jjb
Vtesse August Newsletters are Now Available Online Click below for different languages
09/07/2016 jjb
Vtesse August 2016 Newsletter Update on the Clinical Trial of VTS-270: The NPC Study Click here for Newsletter
09/01/2016 jjb
Vtesse Town Hall Webinar If you are a parent or caregiver of a person with NPC or a physician who sees NPC patients, please join us for Vtesse, Inc.’s September 1, 2016 Town Hall Webinar. Representatives from Vtesse, along with co-principal investigators Dr. Elizabeth Berry-Kravis of Rush University Medical Center and Dr. Forbes (Denny) Porter of the National Institutes of Health, will:
- provide information on the latest 18-month data from the Phase 1 trial at NIH,
- explain what the “blood brain barrier” is and why it is important in the treatment of NPC,
- outline who to contact for more information about the NPC study in the US, and
- be available to answer any questions you may have.
NPC Conversation with Vtesse CEO CLICK HERE FOR LINK 08/26/2016 jjb
Vtesse July 2016 Newsletter Update on the Clinical Trial of VTS-270: The NPC Study Click here for Newsletter 07/29/2016 jjb
Vtesse Press Release Vtesse Secures Additional Funding to Support Further Product Development and Expand the Ongoing Clinical trial of VTS-270 Click here for full release 07/25/2016 jjb
Vtesse Town Hall Webinar If you are a parent or caregiver of a person with NPC or a physician who sees NPC patients, please join us for Vtesse, Inc.’s 6th July 2016 Town Hall Webinar. Representatives from Vtesse, along with clinical investigator Suresh Vijay, MD, of Birmingham Children’s Hospital, and principal investigator Paul Gissen, PhD, MRCPCH, of Great Ormond Street Hospital, will:
- provide information on the latest 18 month data from the Phase 1 trial at NIH,
- explain what the “blood/brain barrier” is and why it is important to the treatment of NPC,
- outline who to contact for more information about the NPC study in the UK, and
- be available to answer any questions you may have.
Click below for “A Day In the Life” videos in several languages.
Vtesse June Newsletters are Now Available Online Click below for different languages
Vtesse has released an update on the Phase 2b/3 Clinical Trial of VTS-270 in Niemann-Pick Type C1 Disease with Dose Selection for Evaluation in Second and Final Portion of Trial and Expansion into Europe. Click here for more details May 23rd, 2016 ~ jjb
Vtesse, Inc. is sponsoring a webinar for physicians that will provide an update on the clinical trial of VTS-270 on May 20, 2016. Click here for more details May 18th, 2016 ~ jjb
Vtesse has released an update on the clinical trial of VTS-270:The NPC Study Besides an update on the Vtesse Clincial Trial of VTS-270 you will also find the VTS-270 Clinical Trial Patient Experience video link, 18 month data from NIH Phase 1 clinical trial and an update on the device for administering VTS-270. Click here for the full newsletter May 6th, 2016 ~ jjb
Vtesse has released a video to help caregivers and patients decide if the VTS-270 Clinical Trial is right for them. You can view it at thenpcstudy.com. There is also additional information that you can access about the NPC Study. April 27th, 2016 ~ jjb
Vtesse, Inc. Announces Phase 1/2 Clinical Data Showing Slowing of Disease Progression from VTS-270 Treatment for Niemann-Pick Type C1 Disease Dateline: 03/04/2016 The National Niemann-Pick disease Foundation (NNPDF) is pleased to share with our NPC Community Members a press release from Vtesse, Inc. regarding data from the Phase 1/2 clinical trial that was presented at the 2016 World Symposium on Lysosomal Storage Disease in San Diego, California. GAITHERSBURG, Md., March 4, 2016 /PRNewswire/ — Vtesse, Inc. announced today data from a Phase 1/2 clinical trial demonstrating a reduction in the rate of Niemann-Pick Type C1 (NPC1) Disease progression from Vtesse’s lead drug candidate VTS-270. The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Center for Advancing Translational Sciences, two components of the National Institutes of Health, collaborated closely with patients, patient advocacy groups and academia to initiate the drug development phase for VTS-270. Vtesse is leading the late-stage formal drug development process. The data were presented at the 2016 World Symposium on Lysosomal Storage Disease in San Diego, California. Click here to read the full Press Release dated 4 March 2016. [March 4th, 2016 ~ nmh]
Vtesse, Inc. Announces FDA’s Granting of Breakthrough Therapy Designation for VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Niemann-Pick Type C1 Disease Dateline: 01/06/2016 The National Niemann-Pick disease Foundation (NNPDF) is pleased to share with our NPC Community Members that the Food and Drug Administration (FDA) has granted VTS-270 (2-hydroxypropyl-β-cyclodextrin) aBreakthrough Therapy Designation. The FDA Breakthrough Therapy designation is designed to expedite the development and review of drugs within the FDA regulatory process. This designation is granted by the FDA when the preliminary clinical data indicates that the drug may demonstrate substantial improvement on clinically significant endpoint(s). The Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review, and will allow for a close collaboration between Vtesse and the FDA on the VTS-270 (2-hydroxypropyl-β-cyclodextrin) development program. Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial. For additional information regarding VTS-270 (2-hydroxypropyl-β-cyclodextrin) clinical trial,visit Vtesse’s website. Click here to Read the Press Release dated Jan 6th, 2015 In addition to the above press release, the NNPDF Central Office recently obtained the Webinar recording and slides associated with the Vtesse “Town Hall” Webinar hosted for the NPC patient community on December 14th, 2015. [Jan 6th, 2016 ~ blg]
NPC Vtesse Clinical Trial & Central Office Updates Dateline: Dec 21st, 2015 Dear NNPDF Family & Friends, The National Niemann-Pick disease Foundation (NNPDF) is pleased to be able to share with our NPD community families updates associated with the following:
- Vtesse VTS-270 NPD Type C Clinical Trial UPDATES
- NNPDF Equipment Exchange Program and NEW posted items
ALERT: New Recruiting Sites for VTS-270 (2-hydroxypropyl-β-cyclodextrin) for NPC Dateline: 12/09/2015 ALERT: United States NPD Type C Families & Friends The NNPDF central office noted today that additional investigational sites for Vtesse’s VTS-270 (2-hydroxypropyl-β-cyclodextrin) Phase 2b/3 clinical trial for NPC has been updated and is now actively recruiting patients at the approved clinical trial centers in Orange, CA & Allentown, PA. Families should follow enrollment criteria as provided on thewww.clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit www.clinicaltrials.gov and refer to study reference number: NCT02534844 NIH Phase 1 Clinical Trial Historical Timeline [Dec 9th, 2015 ~ blg]
Register for Webinar on VTS-270 (2-hydroxypropyl-β-cyclodextrin) for NPC Dateline: 12/07/2015 9:30am Pacific, 10:30am Mountain, 11:30am Central, 12:30pm Eastern Click here to view the Slideshow via PDF Dear NNPDF Niemann-Pick Type C (NPC) Family Members, The National Niemann-Pick disease Foundation (NNPDF) is pleased to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse, Inc. with regards to the VTS-270 (2-hydroxypropyl-β-cyclodextrin) clinical trial for Niemann-Pick disease, Type C (NPC). Please join Vtesse on December 14, 2015 where Dr. Paul Gissen and Dr. Elizabeth Berry-Kravis will be providing an update on the clinical trial and will be available to address your questions. Vtesse is committed to keeping the community fully informed and soliciting feedback as they move through the clinical trial process. As a participant, you will be able to ask any questions you may have and gain an understanding of:
- Clinical trial status update (including site updates)
- Protocol updates
- Frequently asked questions
- Q&A
NPD Current Clinical Trial Recruiting Update ~ e-Blast Dear National Niemann-Pick disease Foundation (NNPDF) Family Membership, The NNPDF Central Office is working diligently to ensure that our entire NPD membership community is aware of the various clinical trial opportunities available for those seeking innovative therapeutic options for Niemann-Pick disease. With this in mind, the foundation is providing an update and links to updates which we have received from the three pharmaceutical companies currently moving forward with clinical trial opportunities for NPD patients. To view the e-news Blast with the updated information dated November 11th, 2015, click here [Nov 11th, 2015 ~ blg]
Vtesse, Inc. Expands Scientific Advisory Board and Appoints new VP of Clinical Operations to Support Late-Stage Clinical Study of Lead Drug Candidate VTS-270 Dateline: 10/22/2015 Dear NNPDF Type C Families and Friends, The NNPDF Central Office is pleased to share with our NPC Community a Press Release from Vtesse regarding a new addition of Elizabeth Berry-Kravis, M.D., Ph.D. to the Scientific Advisory Board for Vtesse and Michael Massaro as the new Vice President of Clinical Operations. These new additions to Vtesse will enhance the team that is working to advance the clinical study of VTS-270 for Niemann-Pick disease Type C. Click here to view the full Press Release Vtesse dated October 22, 2015 [Oct 23rd, 2015 ~ blg]
VTS-270 Cyclo Clinical Trial Recruitment Update Dateline: 10/07/2015 Dear NNPDF Type C Families and Friends, The NNPDF Central Office is please to share with our NPC Community that a second Vtesse Cyclodextrin Clinical Trial Site has received authorization to recruits patients for the Phase 2b/3 trials. The 2nd authorized site location is at: NIH in Bethesda, Maryland. (The 1st site location was at Rush in Chicago, Illinois.) For more information regarding the clinical trial recruitment please go to: https://www.clinicaltrials.gov/ct2/show/study/NCT02534844?term=Vtesse&rank=1#contacts We will continue to keep our NPC patient community membership updated as to the availability of new clinical sites as they are approved. United States, Maryland National Institute of Child Health and Human Development at NIH – RECRUITING Bethesda, Maryland, United States, 20878 Contact: Forbes D. Porter, M.D., Ph.D. 301-435-4432 fdporter@mail.nih.gov Contact: Nicole Farhat, NP 301-597-1765 nicole.farhat@nih.gov Vtesse’s Phase 2b/3 prospective, randomized, double-blind, sham-controlled trial of VTS-270 will be conducted in patients affected by NPC disease. The trial will take place in up to 20 centers in the United States and Europe. It is a three-part, efficacy and safety trial of VTS-270, administered by the lumbar intrathecal route (IT) every two weeks, with a planned enrollment of approximately 51 patients (in Parts A and B). In Part A of the study, researchers will evaluate three different dose levels of VTS-270 versus sham-control to determine the dose level for Parts B and C. All trial participants will be eligible to receive treatment with VTS-270 in Part C, the open-label extension, until the time of regulatory decisions. For more information regarding the clinical trial recruitment please go tohttps://www.clinicaltrials.gov/ct2/show/study/NCT02534844?term=Vtesse&rank=1#contacts [Oct 7th, 2015 ~ blg]
Bio Report Podcast Featuring Ben Machielse from Vtesse, Inc. Dateline: 10/06/2015 Dear NNPDF Type C Families and Friends, Last week, Ben Machielse, President and CEO at Vtesse, had an opportunity to sit down with Daniel Levine for a segment featured on the Bio Report Podcast. The Bio Report Podcast focuses on the intersection of biotechnology with business, science, and policy. Ben was able to focus on the progress made in their clinical development program associated with the Phase II/III for VTS-270 a modified form of cyclodextrin. We are sharing the link for you to listen to the audio recording of the pod cast below: Rare Disease Drug Developer Shows Speed of Business Model [Oct 6th, 2015 ~ blg]
Vtesse, Inc. Initiates Phase 2b/3 Clinical Trial of VTS-270 for Treatment of Niemann-Pick Type C1 (NPC) Disease Dateline: 09/28/2015 Click here to read the letter recently mailed out to families from the NNPDF regarding recent Vtesse Clinical Trial Updates ~ Dated: September 28th, 2015 The NNPDF Central Offices are pleased to share with our NPC community that Vtesse, Inc. today announced that the first three patients have been screened for inclusion in its pivotal Phase 2b/3 clinical trial with VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC). This clinical trial follows a Phase 1 study conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). NPC is a progressive, irreversible, chronically debilitating – and ultimately lethal – genetic disease. It is caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen. Vtesse has worked extensively with regulators in the United States and Europe with the goal of conducting its pivotal study under one global protocol that will evaluate safety and efficacy of VTS-270 to support approval of the drug by regulatory agencies in both regions. Ben Machielse, Drs., President and Chief Executive Officer of Vtesse, Inc., said, “Starting the pivotal clinical trial for VTS-270 within nine months of the company’s launch underscores our dedication to serve the NPC community. A key ingredient that enabled us to start the trial so quickly is the complete package of high-quality pre-clinical data that clearly outlines the potential benefits of VTS-270, such as prolonged survival and preservation of neurons, as well as encouraging clinical data from the Phase 1 trial. The pivotal trial will allow us to further evaluate safety and efficacy of VTS-270 in the pivotal trial to build a foundational dataset for the NPC community.” He added, “While initiation of the first site at Rush University Medical Center and the screening of three patients is a key milestone, we intend to open several sites in quick succession in the United States and the European Union to ensure availability of sites in close proximity to the patients who are eligible to enroll in the trial.” Vtesse’s Phase 2b/3 prospective, randomized, double-blind, sham-controlled trial of VTS-270 will be conducted in patients affected by NPC disease. The trial will take place in up to 20 centers in the United States and Europe. It is a three-part, efficacy and safety trial of VTS-270, administered by the lumbar intrathecal route (IT) every two weeks, with a planned enrollment of approximately 51 patients (in Parts A and B). In Part A of the study, researchers will evaluate three different dose levels of VTS-270 versus sham-control to determine the dose level for Parts B and C. All trial participants will be eligible to receive treatment with VTS-270 in Part C, the open-label extension, until the time of regulatory decisions. Preliminary analyses of the Phase 1 trial, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardized measure) in children treated with VTS-270 as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients. The VTS-270 pivotal study addresses a pressing need to discover new treatments for the disease, according to Forbes D. Porter, M.D., Ph.D., Co-Principal Investigator of the Phase 2b/3 study, Senior Investigator and Program Head in the intramural research program of the Developmental Endocrinology and Genetics Program (PDEGEN), NICHD. He added that the trial has the potential to provide important information about the disease as well as how patients may tolerate VTS-270 and its potential effects on the symptoms of NPC. “The recent analysis of the preliminary Phase 1 clinical data for VTS-270 has provided encouraging support suggesting this treatment for NPC may be able to alter the course of this fatal disease,” said Elizabeth Berry-Kravis, M.D., Ph.D., Co-Principal Investigator of the Phase 2b/3 study, and Professor of Pediatrics, Neurological Sciences, Biochemistry, Rush University Medical Center. “The beginning of patient screening for the pivotal Phase 2b/3 study for VTS-270 marks another important step in the journey toward targeted treatment of NPC, as clinical trials are the only means to determine the effectiveness of VTS-270. This pivotal trial will allow us to gather the data required to advance the regulatory process and move toward U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) approval for VTS-270.” More information on this Phase 2b/3 study can be found at Vtesse’s clinical trial website:www.theNPCstudy.com. Vtesse launched the website this summer as a resource to patients, parents, caregivers and physicians. For more information on enrollment criteria, please see:www.theNPCstudy.com/ – !trial-enrollment/c1ine. About VTS-270 Vtesse’s lead compound, VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for NPC. It is a well-characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at the National Institutes of Health, as well as under individual compassionate-use Investigational New Drug applications (iINDs) and in other academic labs. Most recently, Vtesse announced positive clinical results from the on-going Phase 1 study that demonstrated VTS-270 stabilizes the overall measures of NPC while improving certain disease measures. NPC is a genetic disease affecting an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or under-diagnosed. Affected patients are usually identified in early childhood with ataxia, exhibit progressive impairment of motor and intellectual function, and often die before adulthood. About Vtesse Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved. Vtesse is working collaboratively with the NIH and other leading academic centers to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other lysosomal storage diseases (LSDs). Vtesse is led by a highly experienced management team that has been involved in the development of more than 20 approved drugs and vaccines. Its experienced consortium of investors, led by New Enterprise Associates, has committed initial funding that is expected to bring this compound through pivotal clinical trial. Vtesse is based in Gaithersburg, Maryland and is the first spin-out company from Cydan Development, Inc. For more information, visit www.vtessepharma.com. Corporate Contact: Ravi Venkataramani, Ph.D. ravi@vtessepharma.com [Sept 28th, 2015 ~ cdk]
Vtesse “Pivotal Clinical Trial for VTS-270” Document Vtesse has provided the NNPDF with a document entitled “Pivotal Clinical Trial for VTS-270”. This document provides the community with information regarding the Phase 2/3 clinical trial which will begin recruiting patients in the United States shortly. Other sites will begin recruitment on a rolling-basis. Special note should be taken of slide 4 & 5 and slide 15. Slide 4 & 5 discuss the difference in dosing in the Central Nervous System versus Systemic Dosing. In slide 15 you can play an audio simulation of the hearing loss that was experienced by a Phase I patient. The dark blue boxes are the ones you can click on and listen to. Within the Vtesse power point below there is an audio section included in slide 15 and in order to hear it you must download it to your computer and use Adobe Acrobat Reader to view it. Viewing the document inside an internet browser will disable the audio within the presentation. To continue on without the audio simulation, simply click on the link. Instructions To Download So You Can Hear the Audio Simulation: Right click on the link and choose “Save as”, then save to your desktop to make the document easy to locate. Open it on your desktop within Adobe Acrobat Reader. Vtesse Powerpoint (PDF) [Sep 25th, 2015 ~ blg]
Vtesse, Inc. Announces Preliminary Data from Ongoing Phase 1 Study of VTS-270 for Treatment of Niemann-Pick disease Type C Click here to read the letter recently mailed out to families from the NNPDF regarding recent Vtesse Clinical Trial Updates ~ Dated: August 17th, 2015 The NNPDF Central Offices are pleased to share with our NPC community that Vtesse, Inc. announced preliminary results today from an open-label Phase 1 clinical trial with VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for treatment of Niemann-Pick disease Type C (NPC) conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). Preliminary analyses, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardized measure) in children treated with VTS-270 in the Phase 1 trial as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients. The analyses also show that children treated with VTS-270 demonstrated improvement on several disease domains. NPC is a progressive, irreversible, chronically debilitating – and ultimately lethal – genetic disease. It is caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen. Researchers at NIH’s National Center for Advancing Translational Sciences (NCATS) and NICHD, in close collaboration with Vtesse, patients and patient advocacy groups, developed VTS-270 as part of a project focused on finding treatments for NPC. VTS-270 has been shown to significantly reduce disease progression in naturally occurring animal models and is currently being tested in this Phase 1 clinical trial. Both the ongoing Phase 1 study and the natural history study use a standardized measurement of disease progression, the NPC score, which relies on a specific rating scale that scores the disease along major domains (or traits). Researchers have matched participants in the Phase 1 study to individuals from the natural history data set according to baseline age and disease severity. The rate of disease progression of this matched cohort was compared to the rate of disease progression of children treated with VTS-270 in the Phase 1 study. In the Phase 1 trial, among the 12 children treated with direct administration of VTS-270 into the cerebrospinal fluid via an intrathecal administration for more than six months and up to 12 months, the overall NPC score showed a slowing down in the rate of decline. In these patients, when the scores for impact on hearing were removed, the NPC score showed disease stabilization and halting of progression. Domains of cognition and speech have shown improvement of the disease state for participants in the study; while ambulation, fine motor skills, cognition, swallowing, and memory demonstrate slowing down of decline. Measurements of eye movement and hearing appeared to have worsened among participants in the Phase 1 trial. “This initial analysis of the data look encouraging and this therapy may make a meaningful difference for children with NPC,” said Ben Machielse, Drs., President and Chief Executive Officer of Vtesse, Inc. “Based on discussions with regulators, we anticipate that a single pivotal trial would form the basis for approval of VTS-270 in the United States and Europe. The pivotal trial design will be randomized and controlled, and our planned implementation of the trial underscores the essence of our commitment to the NPC patients and their caregivers. We intend to execute flawlessly and with the greatest care for this sensitive group of patients who are in dire need of new treatment options.” In the Phase 1 trial to date, VTS-270 has been well tolerated other than observed worsening in the eye movement and hearing of some trial participants. Clinicians have administered more than 250 intrathecal administrations of the drug to study participants thus far with minimal administration-related side effects. Vtesse plans to submit more complete Phase 1 clinical trial results for presentation at a scientific meeting later this year. More details about the study can be found athttps://www.clinicaltrials.gov/ct2/show/NCT01747135. About VTS-270 Vtesse’s lead compound, VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for NPC. It is a well-characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at NIH, as well as under individual compassionate use investigational new drug applications (iINDs) and in other academic labs. NPC is a genetic disease affecting an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or under-diagnosed. Affected patients are usually identified in early childhood with ataxia, exhibit progressive impairment of motor and intellectual function, and often die before adulthood. Vtesse is working expediently with NIH’s National Center for Advancing Translational Sciences (NCATS) and NICHD, regulatory authorities, patient/parent organizations, physicians and other key stakeholders to soon start a Phase II/III clinical trial to assess the efficacy of the compound for the treatment of NPC. Based upon productive discussions with U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA), Vtesse will anticipates that this trial will begin enrolling patients in September 2015. About Vtesse Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved. Vtesse is working collaboratively with the NIH and other leading academic centers to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other LSDs. Vtesse is led by a highly experienced management team that has been involved in the development of more than 20 approved drugs and vaccines. Its experienced consortium of investors, led by New Enterprise Associates, has committed initial funding that is expected to bring this compound through pivotal clinical trials. Vtesse is based in Gaithersburg, Maryland and is the first spin-out company from Cydan Development, Inc. For more information, visit www.vtessepharma.com. Corporate Contact: Ravi Venkataramani, Ph.D. ravi@vtessepharma.com Click here to read the letter recently mailed out to families from the NNPDF regarding recent Vtesse Clinical Trial Updates ~ Dated: August 17th, 2015 [Aug 6th, 2015 ~ blg]
Hello NNPDF Families and Friends,
We are delighted to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse’s leadership and clinical team. As mentioned in the initial announcement, Vtesse is committed to keeping the community fully informed and soliciting feedback as we move toward the clinical trial process.
Two webinars will be held on July 14, 2015. As a participant, you will be able to ask any questions you may have and gain an understanding of:
- Recent developments at FDA and EMA
- Phase 1 data review
- Final Protocol including the inclusion and exclusion criteria
- Introduction of the Clinical Trial Website
- Update on the Fight NPC Patient Map
RE: Vtesse NPC Community Town Hall Webinar ~ July 14th, 2015 Vtesse invites you to hear about their progress, ask questions and provide feedback Dear NNPDF Niemann-Pick Type C (NPC) Family Members, The NNPDF is delighted to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse’s company leadership and clinical team. As mentioned in their initial announcement, dated January 7th, 2015, Vtesse is committed to keeping the community fully informed and soliciting feedback as they move toward the clinical trial process for VTS-270 (cyclodextrin) for Niemann-Pick disease, Type C (NPC). This is your opportunity to be part of that discussion! Two webinars will be held on July 14th, 2015. As a participant, you will be able to ask any questions you may have and gain an understanding of:
- Recent developments at Food and Drug Administration (FDA) and European Medicines Agency (EMA)
- Phase 1 data review
- Protocol: inclusion and exclusion criteria
- Introduction of the Clinical Trial Website
- Update on the Fight NPC Patient Map
Vtesse Webinar Summary & SoundCloud Podcast with CEO Ben Machielese Dear NNPDF Families and Friends, The NNPDF Central Office is pleased to provide our Niemann-Pick type C family membership community with a summary from the April 16th Vtesse sponsored webinar for those of you who could not be in attendance. Please find the PDF document via this link: Click here to view the PDF summary of the Vtesse Webinar The Vtesse webinar addressed the progress and updates tied to plans for the VTS-270 (cyclodextrin) Phase II & III clinical trials for Niemann-Pick disease, Type C (NPC). To see an agenda of the topics discussed during the call ~ please follow this link: April 16th Vtesse sponsored webinar In addition, we are including a podcast with Ben Machielse, CEO of Vtesse, Inc as he discusses:
- What is Niemann-Pick disease type C.
- The role that NPC parents & patients have played in getting the research moving forward.
- How NIH & NCATS paved the way for the Cyclodextrin clinical trial.
- Vtesse, Inc, Cydan and how they became involved with Cyclodextrin.
Register for Webinar on Cyclodextrin for NPC Dateline: April 16th, 2015 Vtesse invites you to hear about their progress, ask questions and provide feedback Dear NNPDF Niemann-Pick Type C (NPC) Family Members, Many of you have probably been looking forward with great anticipation to the progress updates on Vtesse, Inc.’s plans for VTS-270 (cyclodextrin) for Niemann-Pick disease, Type C (NPC), announced in January 2015. The National Niemann-Pick disease Foundation (NNPDF) is pleased to be able to share with you an opportunity to attend a town hall webinar hosted by the company’s leadership at Vtesse. As mentioned in their initial announcement, Vtesse is committed to keeping the community fully informed and soliciting feedback as they move forward with clinical development. The company has informed us it is still in the process of planning the pivotal clinical trial, and as such, it will not have all the answers to your questions. However, this webinar offers an exciting opportunity to be part of the discussion with Vtesse to help shape certain aspects of the company’s plan. We hope that the audience will consist of patients, their families and clinicians. A 90-minute webinar will be held in English on April 16, 2015 at 6:30 PM CT geared toward a US and Canadian audience and one geared toward the EU Audience at 6:30 PM GMT. As a participant, you can expect to:
- Meet the company’s leadership
- Hear the company’s view on the advantages of conducting a clinical trial
- Get a family perspective on participation in Phase I of the Cyclodextrin trial at NIH
- Hear the company’s preliminary thoughts on the clinical trials
- Provide feedback to the company on certain aspects of its clinical trial plans
- Ask any questions that you may have
~Vtesse, Inc. Press Release~ Dateline: 03/25/2015 Vtesse, Inc. Forms Scientific Advisory Board Dear NNPDF Families and Friends, We are pleased to share with the foundation’s Niemann-Pick disease type C (NPC) families and community a press release regarding the recently incorporated, Vtesse, a rare disease company spun-off from Cydan Development, Inc. Vtesse announced today the formation of its Scientific Advisory Board (SAB), to include Niemann-Pick disease experts Drs. Paul Gissen, Marc Patterson and Forbes D. Porter (who both are currently are on the NNPDF’s International SAB), along with biopharmaceutical industry leader Dr. Cristina Csimma, to join Vtesse in their quest to develop new treatments for people facing Niemann-Pick disease Type C and rare diseases. The press release reads as follows: Gaithersburg, MD, March 25, 2015 – Vtesse, Inc., a rare disease company focused on developing drugs for Niemann-Pick disease Type C (NPC) and other severe diseases with great unmet need, announced today the formation of its Scientific Advisory Board (SAB). Comprising experts from a range of fields including pediatrics, translational medicine, biomarker research and genetics, the SAB will advise on clinical and regulatory strategy as well as product development for the company’s lead candidate, VTS-270. “Our SAB is comprised of leading international scientific and medical experts in NPC and in many disciplines that are important for the successful development of drugs for those facing rare diseases. These prominent researchers share Vtesse’s mission to develop new treatments to significantly improve the quality of life for people suffering from NPC and other lysosomal storage diseases (LSDs),” said Ben Machielse, Drs, President and Chief Executive Officer of Vtesse, Inc. “We also share a common, strong belief that VTS-270, our lead clinical candidate, has the potential to be an important drug based on the trove of research completed and effort made to date from parents, scientists, and clinicians.” Members of the Vtesse SAB include:
- Cristina Csimma, PharmD, MHP, Co-Chair, an established leader across the biopharmaceutical industry, venture capital and academic settings, and former founding chief executive officer of Cydan Development, Inc.
- Forbes D. Porter, MD, PhD, Co-Chair, a Senior Investigator and Program Head in the intramural research program of the Developmental Endocrinology and Genetics Program (PDEGEN) of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), National Institutes of Health (NIH), Principal Investigator for Phase I Clinical Trial of VTS-270
- Paul Gissen, MBChB, PhD, Professor, University College of London; Honorary Consultant in Paediatric Metabolic Diseases, Great Ormond Street Hospital; Wellcome Trust Senior Research Fellow in Clinical Sciences, University College London Institute of Child Health
- Marc C. Patterson, MD, FRACP, Chair, Division of Child and Adolescent Neurology; Professor of Neurology, Pediatrics and Medical Genetics, Mayo Clinic Children’s Center, Rochester, MN
~Cyclodextrin Update~ 01/07/2015 Update on NPC Cyclodextrin Trial Leading Life Science Syndicate Commits $25 Million to Series A Funding to Launch Vtesse, Inc., the First Rare Disease Company Spun Out of Cydan Development, Inc. Additional Resources:
- National Institutes of Health (NIH) News: NIH Teams with Industry to Develop Treatments for Niemann-Pick Type C
- Wall Street Journal (WSJ) Article Small Biotech Gets Rights to Rare Disease Drug (paid subscription required to read)
FOR IMMEDIATE RELEASE Leading Life Science Syndicate Commits $25 Million to Series A Funding to Launch Vtesse, Inc., the First Rare Disease Company Spun Out of Cydan Development, Inc. Gaithersburg, MD, and Cambridge, MA, January 7, 2015 – Vtesse, Inc., a rare disease company focused on developing drugs for Niemann-Pick disease Type C (NPC) and other severe diseases with great unmet need, announced today that it has raised $25 million in Series A funding. Vtesse is the first spin-off company for Cydan Development, Inc., an orphan-drug accelerator that shares with Vtesse the same syndicate of leading life sciences investors that are committed to funding additional rare disease companies. New Enterprise Associates (NEA) led the Vtesse financing with participation from Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments. Vtesse also announced that it has established a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Center for Advancing Translational Sciences (NCATS), each a component of the National Institutes of Health (NIH). Vtesse and NCATS have also entered into a licensing agreement for the current rights held by NIH for the worldwide use of cyclodextrin, delta-tocopherol, and derivatives of tocopherol, alone or in combination, for the treatment of lysosomal storage diseases (LSDs), including NPC. Regulatory orphan designations for the U.S. and EU will be also be transferred to Vtesse. Vtesse will use the proceeds from its Series A financing to conduct a clinical program for VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for NPC, and to discover and pre-clinically evaluate additional novel drugs for NPC and other LSDs. NPC is a genetic disease associated with mutations in NPC1 and NPC2 genes. NPC affects an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or underdiagnosed. Affected patients are usually identified in early childhood with ataxia and exhibit progressive impairment of motor and intellectual function, and often die before adulthood. “We’ve launched Vtesse to rapidly advance the clinical development of VTS-270, which we hope to make widely available to the many young patients suffering from the debilitating effects of NPC,” said Ben Machielse, Drs., President and Chief Executive Officer of Vtesse, Inc. “We expect to listen to and learn from the physicians, independent researchers, parents and patients who have worked tirelessly for many years to find a treatment for this devastating disorder. We are grateful for the work they and the NIH have conducted thus far, and we are committed to developing a broadly available treatment for NPC.” “The launch of Vtesse is an important milestone for Cydan as this new company holds great promise for patients and is an excellent example of collaboration among scientific, patient and investment stakeholders,” said Chris Adams, Ph.D., Founder and Chief Executive Officer at Cydan and an independent member of the Vtesse Board of Directors. “Our goal at Cydan Development is to advance innovative treatments for patients with rare diseases by identifying promising assets, accelerating their pre-clinical and clinical development, selecting the right management team, and, ultimately, creating more companies like Vtesse.” Vtesse and NIH to Collaborate on VTS-270 Clinical Study Under the CRADA, Vtesse, NICHD and NCATS intend to collaborate to launch a second VTS-270 clinical study for the treatment of NPC as well as to develop other novel drugs (delta-tocopherol and combinations) for NPC and other LSDs. Under the terms of the agreement, NICHD, which is the current sponsor of a Phase I clinical trial of VTS-270 in patients with NPC, Type 1, will transfer its investigational new drug (IND) application to Vtesse along with all background data on the program. Vtesse will be responsible for all further development of VTS-270 with the intent to seek marketing approval for VTS-270 from U.S. and European regulatory agencies. “This is an excellent example of how launching a project to study the underlying biology of one disease can lead to advances that hold promise for an entire group of diseases — the NCATS goal of finding what is common among diseases and the translational science process,” said NCATS Director Christopher P. Austin, M.D. “I am grateful to all of the NPC patients, their families and patient support groups who have been equal partners in our efforts to find therapeutic solutions to these devastating disorders.” Experienced Life Science Industry Team to Lead Vtesse Biopharmaceutical industry veteran Ben Machielse, Drs., will serve as Vtesse President and Chief Executive Officer. Before founding Vtesse, Drs. Machielse led drug development of Omthera Pharmaceutical Inc.’s recently approved product, EPANOVA®, and was involved in the sale of the specialty drug maker to AstraZeneca. Previously, he held senior executive positions at Centocor, Xoma and MedImmune/AstraZeneca. Vtesse’s management team also includes Allan Darling, Ph.D., Vice President, Technical Operations; Sarah Frech, D.V.M., M.P.H., Vice President, Clinical Research; and Ravi Venkataramani, Ph.D., Chief Business Officer. Each of these individuals has extensive industry experience at companies such as MedImmune/AstraZeneca, Invitrogen/BioReliance, Baxter Healthcare and Genocea Biosciences, and all have been involved in the successful clinical development and commercialization of drugs. The Board of Directors for Vtesse, Inc. includes David Mott, NEA, Board Chair; Ben Machielse, Drs., Vtesse, Inc.; Sara Nayeem, M.D., NEA; Barbara Dalton, Ph.D., Pfizer Venture Investments; Mette Kirstine Agger, Lundbeckfond Ventures; and Carl Goldfisher, M.D., Bay City Capital; as well as veteran rare disease industry experts, Cristina Csimma, Pharm.D., and Chris Adams, Ph.D., both of whom will serve as independent directors. “Vtesse combines an experienced management team with a strong financial and scientific foundation and a commitment to the NPC community,” said Mr. Mott. “With this vision and support, we expect Vtesse to move VTS 270 forward quickly and with the utmost attention to medical excellence.” About VTS-270 Vtesse’s lead compound, VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for Niemann-Pick disease Type C (NPC). It is a well characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at NCATS, NICHD and the NIH Clinical Center, as well as under individual compassionate use investigational new drug applications (iINDs) and in other academic labs. Vtesse aims to work expediently with NCATS and NICHD, regulatory authorities, patient/parent organizations, physicians and other key stakeholders to start a Phase II/III clinical trial to assess the efficacy of the compound for the treatment of NPC. Pending the outcomes of discussions with the U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA), Vtesse will provide an update on anticipated timing for such a trial. About Vtesse Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved. The first spin-out company from Cydan Development, Inc., an orphan-drug accelerator that identifies and de-risks programs with therapeutic and commercial potential, Vtesse is working collaboratively with the NIH to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other LSDs. Vtesse is led by a highly experienced management team that has been involved in the development of more than 20 approved drugs and vaccines. Its experienced consortium of investors, led by New Enterprise Associates, has committed initial funding that is expected to bring this compound through pivotal clinical trials. Vtesse is based in Gaithersburg, Maryland. For more information, visit www.vtessepharma.com. About Cydan Development, Inc. Cydan is an orphan-drug accelerator that identifies and de-risks orphan drug products with significant therapeutic and commercial potential, with the goal of starting multiple companies to develop such therapies. Cydan was launched in 2013 by a management team with extensive drug discovery, clinical development, and business development experience. Cydan is financed by leading life sciences investors NEA, Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments. The accelerator is based in Cambridge, Massachusetts. For more information, visit www.cydanco.comor contact Cydan at info@cydanco.com. Corporate Contact: Ravi Venkataramani, Ph.D. rvenkataramani@vtessepharma.com Vtesse Media Contact: Jamie Lacey-Moreira PressComm PR, LLC 410-299-3310 jamielacey@presscommpr.com Cydan Media Contact: Dave Connolly LaVoieHealthScience 617-374-8800, ext. 108 dconnolly@lavoiehealthscience.com
Click here for the PDF Press Release [Jan 7th, 2015 ~ blg]
Patient and Trial Information
MEDICAL AFFAIRS:
email: medinfo@mnk.com
phone: 1-800-778-7898
Latest News
Mallinckrodt Update to the NPC Community
Mallinckrodt Pharmaceuticals has shared an important update regarding the adrabetadex (VTS-270) clinical development program with us for the NPC community. CLICK HERE for complete update.
01/20/2021 jjb
Mallinckrodt Update to the NPC Community on Adrabetadex
As previously mentioned during Mallinckrodt’s webinar on July 16th 2020, Dr. Steve Romano, Mallinckrodt’s Executive Vice President and Chief Scientific Officer, has prepared the following video for the NPC community which provides an update on Adrabetadex. CLICK HERE for video update.
09/03/2020 jjb
Mallinckrodt Message to the NPC Community: Update on Adrabetadex clinical studies
Please take a moment to read this important announcement from Mallinckrodt Pharmaceuticals in regard to the clinical trial of Adrabetadex for NPC-1. Dr. Elizabeth Berry-Kravis and MNK are hosting a webinar this evening, Thursday, July 16 at 8 PM EST to answer questions from the NPC community. The link is https://notredame.zoom.us/j/93668717914?pwd=QzlLNThRdkJ3b0VUMnVLWUREVHd0UT09 and the password is 050409.
CLICK HERE for complete announcement.
07/16/2020 jjb
Mallinckrodt Announces Agreement in Principle for Global Opioid Settlement
Mallinckrodt Pharmaceuticals has provided the following update:
Mallinckrodt has reached a broad based agreement in principle on the terms of a global settlement that would resolve all opioid-related claims against Mallinckrodt, Specialty Generics, and their other subsidiaries. The terms of the agreement include placing their Specialty Generics business in Chapter 11, however the Specialty Brands business is not included in this process. Mallinckrodt is continuing to operate as normal and is committed to improving patient lives with innovative therapies and supporting their advocacy partners.
CLICK HERE for complete announcement.
CLICK HERE for additional information.
02/27/2020 jjb
Mallinckrodt Message to NPC Community: Update on VTS-270
CLICK HERE for message.
10/29/2019 jjb
Mallinckrodt VTS301 Study Resumes in France
CLICK HERE FOR Complete Annoucement
10/14/2019 jjb
Mallinckrodt VTS301 Study Resumes in the UK
CLICK HERE FOR Complete Annoucement
10/07/2019 jjb
Mallinckrodt Update on Phase 2b/3 Study Suspension in UK and France
09/13/2019 jjb
Mallinckrodt Clinical Trial update
Letter to NNPDF Regarding VTS301 Phase 2b/3 Study Suspension in UK and France
08/02/2019 jjb
.
08/09/2019 jjb
Mallinckrodt VTS-270-303 Phase 2 Update
CLICK HERE FOR Complete Annoucement
05/09/2019 jjb
NNPDF Industry Update Webinar Series featuring Mallinckrodt
03/26/2019 jjb
Mallinckrodt Investor Call VTS-270 Update
CLICK HERE FOR Complete Annoucement
02/26/2019 jjb
Mallinckrodt VTS-270 Phase 2b.3 Q & A Response
Following Mallinckrodt’s announcement regarding early phase data from their VTS-270 study on November 6th, a comprehensive list of questions posed by the global NPC community, was submitted to the company. You will find Mallinckrodt’s response to these questions accompanied by a video presentation from Mallinckrodt’s Chief Scientific Officer, Steve Romano at the following links.
Mallinckrodt VTS-270 Q & A Response
Video Message from Mallinckrodt to NPC Community
Please contact the NNPDF for further information by email at nnpdf@nnpdf.org or telephone (920) 563-0930.
11/30/2018 jjb
Mallinckrodt VTS-270 Phase 2b.3 Data Announcement
CLICK HERE FOR Complete Annoucement
11/06/2018 jjb
Mallinckrodt Committed to Niemann-PickDisease Type C1 Patients, Therapy
Click here for complete announcement
06/01/2018 jjb
Mallinckrodt Completes Acquisition of Sucampo Pharmaceuticals, Inc.
Click here for complete announcement
2/13/2018 cdk
UPDATE FROM THE NNPDF BOARD Notification of Intended Pharmaceutical Acquisition
The Board would like to make everyone aware of the planned acquisition of Sucampo Pharmaceuticals by Mallinckrodt Pharmaceuticals, which was announced earlier this week. We also look forward to learning about this acquisition and the plans for working with our community. As information becomes available, we will share it with you.
As a reminder to families, we will continue to share information in the format below:
- The office posts information received about trials and expanded access therapies from companies on the NNPDF website under the appropriate pharmaceutical company..
- When information is received, it is posted to the website and a summary post is made on the NNPDF Facebook Page and/or NNPDF Awareness Group, with a link back to our website where you can find more detailed information. .
- All updates from pharmaceutical companies are included in the next monthly e-newsletter with links back to the original posting on the website. .
- All those interested in receiving this important and educational information should subscribe to the NNPDF Blog via the website.
Press releases regarding the acquisition of Sucampo Pharmaceuticals by Mallinckrodt Pharmaceuticals have been issued by both companies and are on their respective websites.
12/29/2017 jjb
Vtesse May 2017 Newsletters are Now Available Online
Click below for different languages
06/19/2017 jjb
Sucampo Aquires Vtesse Inc. Acquisition provides Sucampo with VTS-270,
which is in a pivotal study for the treatment of
Niemann-Pick disease Type C1 (NPC-1)
Click here for full press release
Vtesse ANNOUNCEMENT LETTER IN VARIOUS LANGUAGES
04/04/2017 jjb
Vtesse February/March Newsletters are Now Available Online
Click below for different languages
03/17/2017 jjb
Vtesse Completes Enrollment of Phase 2b/3
Pivotal Clinical Trial of VTS-270 in
Niemann-Pick Type C1 Disease
Click here for complete announcement
03/15/2017 jjb
Vtesse January Newsletters are Now Available Online
Click below for different languages
02/16/2017 jjb
Vtesse JANUARY 2017 Newsletter
Update on the Clinical Trial of VTS-270:
The NPC Study
Vtesse JANUARY 2017 Newsletter
02/13/2017 jjb
Vtesse Press Release
Vtesse Announces Dosing of First Patient in Australia in
Phase 2b/3 Clinical Trial of VTS-270 in Niemann-Pick Type C1 Disease
01/10/2017 jjb
Vtesse December Newsletters are Now Available Online
Click below for different languages
01/06/2017 jjb
Vtesse Announces Appointment of
Jason Meyenburg as Chief Commercial Officer
This new position has been created to help provide access to VTS-270 for children with Niemann-Pick Type C1 Disease upon approval by regulatory authorities. Click below for the complete announcement.
01/05/2017 jjb
Vtesse DECEMBER 2016 Newsletter
Update on the Clinical Trial of VTS-270:
The NPC Study
Vtesse DECEMBER 2016 Newsletter
01/05/2017 jjb
Vtesse Press Release
Vtesse Receives Rare Pediatric Disease Designation by the FDA
for VTS-270 for the Treatment of Niemann-Pick Type C1 Disease
Click here for full press release
11/29/2016 jjb
Vtesse October Newsletters are Now Available Online
Click below for different languages
11/15/2016 jjb
Vtesse Press Release
Medicines and Healthcare Products
Regulatory Agency (“MHRA”) Designates VTS-270
as a Promising Innovative Medicine
Updated with PIM Designation Explanation
Click here for full press release
CLICK HERE FOR PIM DESIGNATION EXPLANATION
11/10/2016 jjb
Vtesse Press Release
Medicines and Healthcare Products
Regulatory Agency (“MHRA”) Designates VTS-270
as a Promising Innovative Medicine
11/02/2016 jjb
Vtesse Press Release
Vtesse Announces Dosing of First Patient in
Germany for Phase 2b/3 Clinical Trial of VTS-270
in Niemann-Pick Type C1 Disease
10/05/2016 jjb
Vtesse September 2016 Frequently Asked Questions
Click below for FAQs in different languages
09/27/2016 jjb
Vtesse August Newsletters are Now Available Online
Click below for different languages
09/07/2016 jjb
Vtesse August 2016 Newsletter
Update on the Clinical Trial of VTS-270:
The NPC Study
Click here for Newsletter
09/01/2016 jjb
Vtesse Town Hall Webinar
If you are a parent or caregiver of a person with NPC or a physician who sees NPC patients, please join us for Vtesse, Inc.’s September 1, 2016 Town Hall Webinar.
Representatives from Vtesse, along with co-principal investigators Dr. Elizabeth Berry-Kravis of Rush University Medical Center and Dr. Forbes (Denny) Porter of the National Institutes of Health, will:
- provide information on the latest 18-month data from the Phase 1 trial at NIH,
- explain what the “blood brain barrier” is and why it is important in the treatment of NPC,
- outline who to contact for more information about the NPC study in the US, and
- be available to answer any questions you may have.
Details and Registration
Date: September 1, 2016
Time: 8:00 p.m. ET/7:00 p.m. CT/6:00 p.m. MT/5:00 p.m. PT
To register, click here.
If you would like to access a playback of this Town Hall Webinar, please email me atCarrie@vtessepharma.com. You may also use my email to submit questions in advance.
Thank you!
Carrie Burke
Senior Director, Patient Advocacy, Vtesse, Inc.
NPC Conversation with Vtesse CEO
08/26/2016 jjb
Vtesse July 2016 Newsletter
Update on the Clinical Trial of VTS-270:
The NPC Study
07/29/2016 jjb
Vtesse Press Release
Vtesse Secures Additional Funding to Support Further Product Development
and Expand the Ongoing Clinical trial of VTS-270
Click here for full release
07/25/2016 jjb
Vtesse Town Hall Webinar
If you are a parent or caregiver of a person with NPC or a physician who sees NPC patients, please join us for Vtesse, Inc.’s 6th July 2016 Town Hall Webinar.
Representatives from Vtesse, along with clinical investigator Suresh Vijay, MD, of Birmingham Children’s Hospital, and principal investigator Paul Gissen, PhD, MRCPCH, of Great Ormond Street Hospital, will:
- provide information on the latest 18 month data from the Phase 1 trial at NIH,
- explain what the “blood/brain barrier” is and why it is important to the treatment of NPC,
- outline who to contact for more information about the NPC study in the UK, and
- be available to answer any questions you may have.
Details and Registration
Date: Wednesday, July 6, 2016
Time: 3:00 p.m. EST/20:00 p.m. BST
To register, CLICK HERE. If you would like to access a playback of this Town Hall Webinar, please email me at Carrie@vtessepharma.com. You may also use my email to submit questions in advance.
Thank you!
Carrie Burke
Senior Director, Patient Advocacy, Vtesse, Inc.
Click below for “A Day In the Life” videos in several languages.
Vtesse June Newsletters are Now Available Online
Click below for different languages
Vtesse has released an update on the Phase 2b/3 Clinical Trial of VTS-270 in Niemann-Pick Type C1 Disease with Dose Selection for Evaluation in Second and Final Portion of Trial and Expansion into Europe.
May 23rd, 2016 ~ jjb
Vtesse, Inc. is sponsoring a webinar for physicians that will provide an update on the clinical trial of VTS-270 on May 20, 2016.
May 18th, 2016 ~ jjb
Vtesse has released an update on the clinical trial of VTS-270:The NPC Study
Besides an update on the Vtesse Clincial Trial of VTS-270 you will also find the VTS-270 Clinical Trial Patient Experience video link, 18 month data from NIH Phase 1 clinical trial and an update on the device for administering VTS-270.
Click here for the full newsletter
May 6th, 2016 ~ jjb
Vtesse has released a video to help caregivers and patients decide if the VTS-270 Clinical Trial is right for them.
You can view it atthenpcstudy.com.
There is also additional information that you can access about the NPC Study.
April 27th, 2016 ~ jjb
Vtesse, Inc. Announces Phase 1/2 Clinical Data Showing Slowing of Disease Progression from VTS-270 Treatment for Niemann-Pick Type C1 Disease
Dateline: 03/04/2016
The National Niemann-Pick disease Foundation (NNPDF) is pleased to share with our NPC Community Members a press release from Vtesse, Inc. regarding data from the Phase 1/2 clinical trial that was presented at the 2016 World Symposium on Lysosomal Storage Disease in San Diego, California.
GAITHERSBURG, Md., March 4, 2016 /PRNewswire/ — Vtesse, Inc. announced today data from a Phase 1/2 clinical trial demonstrating a reduction in the rate of Niemann-Pick Type C1 (NPC1) Disease progression from Vtesse’s lead drug candidate VTS-270. The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Center for Advancing Translational Sciences, two components of the National Institutes of Health, collaborated closely with patients, patient advocacy groups and academia to initiate the drug development phase for VTS-270. Vtesse is leading the late-stage formal drug development process.
The data were presented at the 2016 World Symposium on Lysosomal Storage Disease in San Diego, California.
Click here to read the full Press Release dated 4 March 2016.
[March 4th, 2016 ~ nmh]
Vtesse, Inc. Announces FDA’s Granting of Breakthrough Therapy Designation for VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Niemann-Pick Type C1 Disease
Dateline: 01/06/2016
The National Niemann-Pick disease Foundation (NNPDF) is pleased to share with our NPC Community Members that the Food and Drug Administration (FDA) has granted VTS-270 (2-hydroxypropyl-β-cyclodextrin) aBreakthrough Therapy Designation. The FDA Breakthrough Therapy designation is designed to expedite the development and review of drugs within the FDA regulatory process.
This designation is granted by the FDA when the preliminary clinical data indicates that the drug may demonstrate substantial improvement on clinically significant endpoint(s). The Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review, and will allow for a close collaboration between Vtesse and the FDA on the VTS-270 (2-hydroxypropyl-β-cyclodextrin) development program.
Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial. For additional information regarding VTS-270 (2-hydroxypropyl-β-cyclodextrin) clinical trial,visit Vtesse’s website.
Click here to Read the Press Release dated Jan 6th, 2015
In addition to the above press release, the NNPDF Central Office recently obtained the Webinar recording and slides associated with the Vtesse “Town Hall” Webinar hosted for the NPC patient community on December 14th, 2015.
[Jan 6th, 2016 ~ blg]
NPC Vtesse Clinical Trial & Central Office Updates
Dateline: Dec 21st, 2015
Dear NNPDF Family & Friends,
The National Niemann-Pick disease Foundation (NNPDF) is pleased to be able to share with our NPD community families updates associated with the following:
- Vtesse VTS-270 NPD Type C Clinical Trial UPDATES
- NNPDF Equipment Exchange Program and NEW posted items
Click here to view the NNPDF e-Blast
[Dec 21st, 2015 ~ blg]
ALERT: New Recruiting Sites for VTS-270 (2-hydroxypropyl-β-cyclodextrin) for NPC
Dateline: 12/09/2015
ALERT: United States NPD Type C Families & Friends
The NNPDF central office noted today that additional investigational sites for Vtesse’s VTS-270 (2-hydroxypropyl-β-cyclodextrin) Phase 2b/3 clinical trial for NPC has been updated and is now actively recruiting patients at the approved clinical trial centers in Orange, CA & Allentown, PA.
Families should follow enrollment criteria as provided on thewww.clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit www.clinicaltrials.gov and refer to study reference number: NCT02534844
NIH Phase 1 Clinical Trial Historical Timeline
[Dec 9th, 2015 ~ blg]
Register for Webinar on VTS-270 (2-hydroxypropyl-β-cyclodextrin) for NPC
Dateline: 12/07/2015
9:30am Pacific, 10:30am Mountain, 11:30am Central, 12:30pm Eastern
Click here to view the Slideshow via PDF
Dear NNPDF Niemann-Pick Type C (NPC) Family Members,
The National Niemann-Pick disease Foundation (NNPDF) is pleased to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse, Inc. with regards to the VTS-270 (2-hydroxypropyl-β-cyclodextrin) clinical trial for Niemann-Pick disease, Type C (NPC).
Please join Vtesse on December 14, 2015 where Dr. Paul Gissen and Dr. Elizabeth Berry-Kravis will be providing an update on the clinical trial and will be available to address your questions. Vtesse is committed to keeping the community fully informed and soliciting feedback as they move through the clinical trial process.
As a participant, you will be able to ask any questions you may have and gain an understanding of:
- Clinical trial status update (including site updates)
- Protocol updates
- Frequently asked questions
- Q&A
You will be able to ask questions during the live webinar, but also please feel free to send any questions prior to the webinar so that we can make sure those are answered within the content of the meeting. You can send any correspondence to Carrie Burke, Senior Director Advocacy at carrie@vtessepharma.com.
After registering, you will receive a confirmation email containing information about joining the webinar.
It is hoped you will take advantage of this opportunity to continue a two-way dialogue, receive information and provide input to the company on the clinical trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) as it moves forward.
Sincerely,
Nadine M. Hill
Executive Director
National Niemann-Pick disease Foundation
[Dec 7th, 2015 ~ blg]
NPD Current Clinical Trial Recruiting Update ~ e-Blast
Dear National Niemann-Pick disease Foundation (NNPDF) Family Membership,
The NNPDF Central Office is working diligently to ensure that our entire NPD membership community is aware of the various clinical trial opportunities available for those seeking innovative therapeutic options for Niemann-Pick disease. With this in mind, the foundation is providing an update and links to updates which we have received from the three pharmaceutical companies currently moving forward with clinical trial opportunities for NPD patients.
To view the e-news Blast with the updated information dated November 11th, 2015, click here
[Nov 11th, 2015 ~ blg]
Vtesse, Inc. Expands Scientific Advisory Board and Appoints new VP of Clinical Operations to Support Late-Stage Clinical Study of Lead Drug Candidate VTS-270
Dateline: 10/22/2015
Dear NNPDF Type C Families and Friends,
The NNPDF Central Office is pleased to share with our NPC Community a Press Release from Vtesse regarding a new addition of Elizabeth Berry-Kravis, M.D., Ph.D. to the Scientific Advisory Board for Vtesse and Michael Massaro as the new Vice President of Clinical Operations.
These new additions to Vtesse will enhance the team that is working to advance the clinical study of VTS-270 for Niemann-Pick disease Type C.
Click here to view the full Press Release Vtesse dated October 22, 2015
[Oct 23rd, 2015 ~ blg]
VTS-270 Cyclo Clinical Trial Recruitment Update
Dateline: 10/07/2015
Dear NNPDF Type C Families and Friends,
The NNPDF Central Office is please to share with our NPC Community that a second Vtesse Cyclodextrin Clinical Trial Site has received authorization to recruits patients for the Phase 2b/3 trials. The 2nd authorized site location is at: NIH in Bethesda, Maryland. (The 1st site location was at Rush in Chicago, Illinois.) For more information regarding the clinical trial recruitment please go to: https://www.clinicaltrials.gov/ct2/show/study/NCT02534844?term=Vtesse&rank=1#contacts
We will continue to keep our NPC patient community membership updated as to the availability of new clinical sites as they are approved.
United States, Maryland
National Institute of Child Health and Human Development at NIH – RECRUITING
Bethesda, Maryland, United States, 20878
Contact: Forbes D. Porter, M.D., Ph.D. 301-435-4432 fdporter@mail.nih.gov
Contact: Nicole Farhat, NP 301-597-1765 nicole.farhat@nih.gov
Vtesse’s Phase 2b/3 prospective, randomized, double-blind, sham-controlled trial of VTS-270 will be conducted in patients affected by NPC disease. The trial will take place in up to 20 centers in the United States and Europe. It is a three-part, efficacy and safety trial of VTS-270, administered by the lumbar intrathecal route (IT) every two weeks, with a planned enrollment of approximately 51 patients (in Parts A and B). In Part A of the study, researchers will evaluate three different dose levels of VTS-270 versus sham-control to determine the dose level for Parts B and C. All trial participants will be eligible to receive treatment with VTS-270 in Part C, the open-label extension, until the time of regulatory decisions.
For more information regarding the clinical trial recruitment please go tohttps://www.clinicaltrials.gov/ct2/show/study/NCT02534844?term=Vtesse&rank=1#contacts
[Oct 7th, 2015 ~ blg]
Bio Report Podcast Featuring Ben Machielse from Vtesse, Inc.
Dateline: 10/06/2015
Dear NNPDF Type C Families and Friends,
Last week, Ben Machielse, President and CEO at Vtesse, had an opportunity to sit down with Daniel Levine for a segment featured on the Bio Report Podcast. The Bio Report Podcast focuses on the intersection of biotechnology with business, science, and policy.
Ben was able to focus on the progress made in their clinical development program associated with the Phase II/III for VTS-270 a modified form of cyclodextrin. We are sharing the link for you to listen to the audio recording of the pod cast below:
Rare Disease Drug Developer Shows Speed of Business Model
[Oct 6th, 2015 ~ blg]
Vtesse, Inc. Initiates Phase 2b/3 Clinical Trial of VTS-270 for Treatment of
Niemann-Pick Type C1 (NPC) Disease
Dateline: 09/28/2015
The NNPDF Central Offices are pleased to share with our NPC community that Vtesse, Inc. today announced that the first three patients have been screened for inclusion in its pivotal Phase 2b/3 clinical trial with VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC). This clinical trial follows a Phase 1 study conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
NPC is a progressive, irreversible, chronically debilitating – and ultimately lethal – genetic disease. It is caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen. Vtesse has worked extensively with regulators in the United States and Europe with the goal of conducting its pivotal study under one global protocol that will evaluate safety and efficacy of VTS-270 to support approval of the drug by regulatory agencies in both regions.
Ben Machielse, Drs., President and Chief Executive Officer of Vtesse, Inc., said, “Starting the pivotal clinical trial for VTS-270 within nine months of the company’s launch underscores our dedication to serve the NPC community. A key ingredient that enabled us to start the trial so quickly is the complete package of high-quality pre-clinical data that clearly outlines the potential benefits of VTS-270, such as prolonged survival and preservation of neurons, as well as encouraging clinical data from the Phase 1 trial. The pivotal trial will allow us to further evaluate safety and efficacy of VTS-270 in the pivotal trial to build a foundational dataset for the NPC community.”
He added, “While initiation of the first site at Rush University Medical Center and the screening of three patients is a key milestone, we intend to open several sites in quick succession in the United States and the European Union to ensure availability of sites in close proximity to the patients who are eligible to enroll in the trial.”
Vtesse’s Phase 2b/3 prospective, randomized, double-blind, sham-controlled trial of VTS-270 will be conducted in patients affected by NPC disease. The trial will take place in up to 20 centers in the United States and Europe. It is a three-part, efficacy and safety trial of VTS-270, administered by the lumbar intrathecal route (IT) every two weeks, with a planned enrollment of approximately 51 patients (in Parts A and B). In Part A of the study, researchers will evaluate three different dose levels of VTS-270 versus sham-control to determine the dose level for Parts B and C. All trial participants will be eligible to receive treatment with VTS-270 in Part C, the open-label extension, until the time of regulatory decisions.
Preliminary analyses of the Phase 1 trial, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardized measure) in children treated with VTS-270 as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients.
The VTS-270 pivotal study addresses a pressing need to discover new treatments for the disease, according to Forbes D. Porter, M.D., Ph.D., Co-Principal Investigator of the Phase 2b/3 study, Senior Investigator and Program Head in the intramural research program of the Developmental Endocrinology and Genetics Program (PDEGEN), NICHD. He added that the trial has the potential to provide important information about the disease as well as how patients may tolerate VTS-270 and its potential effects on the symptoms of NPC.
“The recent analysis of the preliminary Phase 1 clinical data for VTS-270 has provided encouraging support suggesting this treatment for NPC may be able to alter the course of this fatal disease,” said Elizabeth Berry-Kravis, M.D., Ph.D., Co-Principal Investigator of the Phase 2b/3 study, and Professor of Pediatrics, Neurological Sciences, Biochemistry, Rush University Medical Center. “The beginning of patient screening for the pivotal Phase 2b/3 study for VTS-270 marks another important step in the journey toward targeted treatment of NPC, as clinical trials are the only means to determine the effectiveness of VTS-270. This pivotal trial will allow us to gather the data required to advance the regulatory process and move toward U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) approval for VTS-270.”
More information on this Phase 2b/3 study can be found at Vtesse’s clinical trial website:www.theNPCstudy.com. Vtesse launched the website this summer as a resource to patients, parents, caregivers and physicians. For more information on enrollment criteria, please see:www.theNPCstudy.com/ – !trial-enrollment/c1ine.
About VTS-270
Vtesse’s lead compound, VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for NPC. It is a well-characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at the National Institutes of Health, as well as under individual compassionate-use Investigational New Drug applications (iINDs) and in other academic labs. Most recently, Vtesse announced positive clinical results from the on-going Phase 1 study that demonstrated VTS-270 stabilizes the overall measures of NPC while improving certain disease measures. NPC is a genetic disease affecting an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or under-diagnosed. Affected patients are usually identified in early childhood with ataxia, exhibit progressive impairment of motor and intellectual function, and often die before adulthood.
About Vtesse
Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved. Vtesse is working collaboratively with the NIH and other leading academic centers to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other lysosomal storage diseases (LSDs). Vtesse is led by a highly experienced management team that has been involved in the development of more than 20 approved drugs and vaccines. Its experienced consortium of investors, led by New Enterprise Associates, has committed initial funding that is expected to bring this compound through pivotal clinical trial. Vtesse is based in Gaithersburg, Maryland and is the first spin-out company from Cydan Development, Inc. For more information, visit www.vtessepharma.com.
Corporate Contact:
Ravi Venkataramani, Ph.D.
ravi@vtessepharma.com
[Sept 28th, 2015 ~ cdk]
Vtesse “Pivotal Clinical Trial for VTS-270” Document
Vtesse has provided the NNPDF with a document entitled “Pivotal Clinical Trial for VTS-270”. This document provides the community with information regarding the Phase 2/3 clinical trial which will begin recruiting patients in the United States shortly. Other sites will begin recruitment on a rolling-basis.
Special note should be taken of slide 4 & 5 and slide 15. Slide 4 & 5 discuss the difference in dosing in the Central Nervous System versus Systemic Dosing. In slide 15 you can play an audio simulation of the hearing loss that was experienced by a Phase I patient. The dark blue boxes are the ones you can click on and listen to.
Within the Vtesse power point below there is an audio section included in slide 15 and in order to hear it you must download it to your computer and use Adobe Acrobat Reader to view it. Viewing the document inside an internet browser will disable the audio within the presentation. To continue on without the audio simulation, simply click on the link.
Instructions To Download So You Can Hear the Audio Simulation: Right click on the link and choose “Save as”, then save to your desktop to make the document easy to locate. Open it on your desktop within Adobe Acrobat Reader.
[Sep 25th, 2015 ~ blg]
Vtesse, Inc. Announces Preliminary Data from Ongoing Phase 1 Study of VTS-270 for Treatment of Niemann-Pick disease Type C
Click here to read the letter recently mailed out to families from the NNPDF regarding recent Vtesse Clinical Trial Updates ~ Dated: August 17th, 2015
The NNPDF Central Offices are pleased to share with our NPC community that Vtesse, Inc. announced preliminary results today from an open-label Phase 1 clinical trial with VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for treatment of Niemann-Pick disease Type C (NPC) conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
Preliminary analyses, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardized measure) in children treated with VTS-270 in the Phase 1 trial as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients. The analyses also show that children treated with VTS-270 demonstrated improvement on several disease domains.
NPC is a progressive, irreversible, chronically debilitating – and ultimately lethal – genetic disease. It is caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen. Researchers at NIH’s National Center for Advancing Translational Sciences (NCATS) and NICHD, in close collaboration with Vtesse, patients and patient advocacy groups, developed VTS-270 as part of a project focused on finding treatments for NPC. VTS-270 has been shown to significantly reduce disease progression in naturally occurring animal models and is currently being tested in this Phase 1 clinical trial. Both the ongoing Phase 1 study and the natural history study use a standardized measurement of disease progression, the NPC score, which relies on a specific rating scale that scores the disease along major domains (or traits).
Researchers have matched participants in the Phase 1 study to individuals from the natural history data set according to baseline age and disease severity. The rate of disease progression of this matched cohort was compared to the rate of disease progression of children treated with VTS-270 in the Phase 1 study.
In the Phase 1 trial, among the 12 children treated with direct administration of VTS-270 into the cerebrospinal fluid via an intrathecal administration for more than six months and up to 12 months, the overall NPC score showed a slowing down in the rate of decline. In these patients, when the scores for impact on hearing were removed, the NPC score showed disease stabilization and halting of progression. Domains of cognition and speech have shown improvement of the disease state for participants in the study; while ambulation, fine motor skills, cognition, swallowing, and memory demonstrate slowing down of decline. Measurements of eye movement and hearing appeared to have worsened among participants in the Phase 1 trial.
“This initial analysis of the data look encouraging and this therapy may make a meaningful difference for children with NPC,” said Ben Machielse, Drs., President and Chief Executive Officer of Vtesse, Inc. “Based on discussions with regulators, we anticipate that a single pivotal trial would form the basis for approval of VTS-270 in the United States and Europe. The pivotal trial design will be randomized and controlled, and our planned implementation of the trial underscores the essence of our commitment to the NPC patients and their caregivers. We intend to execute flawlessly and with the greatest care for this sensitive group of patients who are in dire need of new treatment options.”
In the Phase 1 trial to date, VTS-270 has been well tolerated other than observed worsening in the eye movement and hearing of some trial participants. Clinicians have administered more than 250 intrathecal administrations of the drug to study participants thus far with minimal administration-related side effects. Vtesse plans to submit more complete Phase 1 clinical trial results for presentation at a scientific meeting later this year. More details about the study can be found athttps://www.clinicaltrials.gov/ct2/show/NCT01747135.
About VTS-270
Vtesse’s lead compound, VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for NPC. It is a well-characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at NIH, as well as under individual compassionate use investigational new drug applications (iINDs) and in other academic labs. NPC is a genetic disease affecting an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or under-diagnosed. Affected patients are usually identified in early childhood with ataxia, exhibit progressive impairment of motor and intellectual function, and often die before adulthood. Vtesse is working expediently with NIH’s National Center for Advancing Translational Sciences (NCATS) and NICHD, regulatory authorities, patient/parent organizations, physicians and other key stakeholders to soon start a Phase II/III clinical trial to assess the efficacy of the compound for the treatment of NPC. Based upon productive discussions with U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA), Vtesse will anticipates that this trial will begin enrolling patients in September 2015.
About Vtesse
Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved. Vtesse is working collaboratively with the NIH and other leading academic centers to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other LSDs. Vtesse is led by a highly experienced management team that has been involved in the development of more than 20 approved drugs and vaccines. Its experienced consortium of investors, led by New Enterprise Associates, has committed initial funding that is expected to bring this compound through pivotal clinical trials. Vtesse is based in Gaithersburg, Maryland and is the first spin-out company from Cydan Development, Inc. For more information, visit www.vtessepharma.com.
Corporate Contact:
Ravi Venkataramani, Ph.D.
ravi@vtessepharma.com
[Aug 6th, 2015 ~ blg]
Hello NNPDF Families and Friends,
We are delighted to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse’s leadership and clinical team. As mentioned in the initial announcement, Vtesse is committed to keeping the community fully informed and soliciting feedback as we move toward the clinical trial process.
Two webinars will be held on July 14, 2015. As a participant, you will be able to ask any questions you may have and gain an understanding of:
- Recent developments at FDA and EMA
- Phase 1 data review
- Final Protocol including the inclusion and exclusion criteria
- Introduction of the Clinical Trial Website
- Update on the Fight NPC Patient Map
For those who aren’t able to join the call ~ please be advised that Vtesse WILL be recording the sessions and making them available at a later date.
Vtesse is also welcoming any questions you may have prior to the webinar. You may send any correspondence to Carrie Burke at: carrie@vtessepharma.com.
Kind regards,
Nadine Hill
NNPDF Executive Director
Click here for the Powerpoint slides
[Jul 10th, 2015 ~ blg]
RE: Vtesse NPC Community Town Hall Webinar ~ July 14th, 2015
Vtesse invites you to hear about their progress, ask questions and provide feedback
Dear NNPDF Niemann-Pick Type C (NPC) Family Members,
The NNPDF is delighted to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse’s company leadership and clinical team. As mentioned in their initial announcement, dated January 7th, 2015, Vtesse is committed to keeping the community fully informed and soliciting feedback as they move toward the clinical trial process for VTS-270 (cyclodextrin) for Niemann-Pick disease, Type C (NPC).
This is your opportunity to be part of that discussion!
Two webinars will be held on July 14th, 2015. As a participant, you will be able to ask any questions you may have and gain an understanding of:
- Recent developments at Food and Drug Administration (FDA) and European Medicines Agency (EMA)
- Phase 1 data review
- Protocol: inclusion and exclusion criteria
- Introduction of the Clinical Trial Website
- Update on the Fight NPC Patient Map
Please note: There is no cost to participate in this webinar and Vtesse welcomes any questions which you may have prior to the webinar. This webinar is for NPC patients worldwide and is currently set-up to accommodate families and participants from 18 countries. You are encouraged to pre-register and submit your questions to Vtesse via the “GoToWebinar” registration site listed below:
The NNPDF looks forward to participating in this webinar on behalf of all our community membership and we hope you will take advantage of this opportunity as well. Please feel free to reach out to the NNPDF Office Staff at nnpdf@nnpdf.org if you have any questions.
We WILL PERSEVERE in our Quest for a Cure!
What is a Webinar?
A webinar is sometimes also referred to as a “webcast”, “online event” or “web seminar”
Webinars typically have audio and visual components and enable the sharing of PowerPoint presentations, videos, web pages or other multimedia content with audiences that can be located anywhere. The visual component of a webinar is shared through a web conferencing tool or Internet browser. The audio portion of a webinar is usually broadcast through the audience’s computers (through speakers and media players) or through the telephone.
[Jun 30th, 2015 ~ blg]
Vtesse Webinar Summary & SoundCloud Podcast with CEO Ben Machielese
Dear NNPDF Families and Friends,
The NNPDF Central Office is pleased to provide our Niemann-Pick type C family membership community with a summary from the April 16th Vtesse sponsored webinar for those of you who could not be in attendance. Please find the PDF document via this link:
Click here to view the PDF summary of the Vtesse Webinar
The Vtesse webinar addressed the progress and updates tied to plans for the VTS-270 (cyclodextrin) Phase II & III clinical trials for Niemann-Pick disease, Type C (NPC). To see an agenda of the topics discussed during the call ~ please follow this link: April 16th Vtesse sponsored webinar
In addition, we are including a podcast with Ben Machielse, CEO of Vtesse, Inc as he discusses:
- What is Niemann-Pick disease type C.
- The role that NPC parents & patients have played in getting the research moving forward.
- How NIH & NCATS paved the way for the Cyclodextrin clinical trial.
- Vtesse, Inc, Cydan and how they became involved with Cyclodextrin.
[May 5th, 2015 ~ blg]
Register for Webinar on Cyclodextrin for NPC
Dateline: April 16th, 2015
Vtesse invites you to hear about their progress, ask questions and provide feedback
Dear NNPDF Niemann-Pick Type C (NPC) Family Members,
Many of you have probably been looking forward with great anticipation to the progress updates on Vtesse, Inc.’s plans for VTS-270 (cyclodextrin) for Niemann-Pick disease, Type C (NPC), announced in January 2015.
The National Niemann-Pick disease Foundation (NNPDF) is pleased to be able to share with you an opportunity to attend a town hall webinar hosted by the company’s leadership at Vtesse. As mentioned in their initial announcement, Vtesse is committed to keeping the community fully informed and soliciting feedback as they move forward with clinical development. The company has informed us it is still in the process of planning the pivotal clinical trial, and as such, it will not have all the answers to your questions. However, this webinar offers an exciting opportunity to be part of the discussion with Vtesse to help shape certain aspects of the company’s plan. We hope that the audience will consist of patients, their families and clinicians.
A 90-minute webinar will be held in English on April 16, 2015 at 6:30 PM CT geared toward a US and Canadian audience and one geared toward the EU Audience at 6:30 PM GMT. As a participant, you can expect to:
- Meet the company’s leadership
- Hear the company’s view on the advantages of conducting a clinical trial
- Get a family perspective on participation in Phase I of the Cyclodextrin trial at NIH
- Hear the company’s preliminary thoughts on the clinical trials
- Provide feedback to the company on certain aspects of its clinical trial plans
- Ask any questions that you may have
It is hoped you will take advantage of this opportunity to begin a two-way dialogue and receive information and provide input to the company on the exciting possibilities that lie ahead. Please feel free to reach out to me at nnpdf@nnpdf.org if you have any questions.
For our family membership residing in Europe, Vtesse is hosting a call April 16, 2015 at 6:30 PM BST; 7:30 PM CET aimed at the UK and European Audience. Although the Webinars are aimed at audiences from the USA, Canada and Europe, participants are welcome from all around the world.
Sincerely,
Nadine M. Hill
Executive Director
National Niemann-Pick disease Foundation
[Apr 7th, 2015 ~ blg]
~Vtesse, Inc. Press Release~
Dateline: 03/25/2015
Vtesse, Inc. Forms Scientific Advisory Board
Dear NNPDF Families and Friends,
We are pleased to share with the foundation’s Niemann-Pick disease type C (NPC) families and community a press release regarding the recently incorporated, Vtesse, a rare disease company spun-off from Cydan Development, Inc.
Vtesse announced today the formation of its Scientific Advisory Board (SAB), to include Niemann-Pick disease experts Drs. Paul Gissen, Marc Patterson and Forbes D. Porter (who both are currently are on the NNPDF’s International SAB), along with biopharmaceutical industry leader Dr. Cristina Csimma, to join Vtesse in their quest to develop new treatments for people facing Niemann-Pick disease Type C and rare diseases.
The press release reads as follows:
Gaithersburg, MD, March 25, 2015 – Vtesse, Inc., a rare disease company focused on developing drugs for Niemann-Pick disease Type C (NPC) and other severe diseases with great unmet need, announced today the formation of its Scientific Advisory Board (SAB). Comprising experts from a range of fields including pediatrics, translational medicine, biomarker research and genetics, the SAB will advise on clinical and regulatory strategy as well as product development for the company’s lead candidate, VTS-270.
“Our SAB is comprised of leading international scientific and medical experts in NPC and in many disciplines that are important for the successful development of drugs for those facing rare diseases. These prominent researchers share Vtesse’s mission to develop new treatments to significantly improve the quality of life for people suffering from NPC and other lysosomal storage diseases (LSDs),” said Ben Machielse, Drs, President and Chief Executive Officer of Vtesse, Inc. “We also share a common, strong belief that VTS-270, our lead clinical candidate, has the potential to be an important drug based on the trove of research completed and effort made to date from parents, scientists, and clinicians.”
Members of the Vtesse SAB include:
- Cristina Csimma, PharmD, MHP, Co-Chair, an established leader across the biopharmaceutical industry, venture capital and academic settings, and former founding chief executive officer of Cydan Development, Inc.
- Forbes D. Porter, MD, PhD, Co-Chair, a Senior Investigator and Program Head in the intramural research program of the Developmental Endocrinology and Genetics Program (PDEGEN) of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), National Institutes of Health (NIH), Principal Investigator for Phase I Clinical Trial of VTS-270
- Paul Gissen, MBChB, PhD, Professor, University College of London; Honorary Consultant in Paediatric Metabolic Diseases, Great Ormond Street Hospital; Wellcome Trust Senior Research Fellow in Clinical Sciences, University College London Institute of Child Health
- Marc C. Patterson, MD, FRACP, Chair, Division of Child and Adolescent Neurology; Professor of Neurology, Pediatrics and Medical Genetics, Mayo Clinic Children’s Center, Rochester, MN
“It’s an honor for me to serve as co-chair of the Vtesse SAB and to work alongside key thought leaders to support the goal of developing new drugs for children facing NPC and for those with other rare diseases,” said Dr. Csimma. “The development of VTS-270 is an important step in the understanding of NPC and enables further progress towards defeating this devastating disease.”
About VTS-270
Vtesse’s lead compound, VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for NPC. It is a well-characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at NIH, as well as under individual compassionate use investigational new drug applications (iINDs) and in other academic labs. Vtesse aims to work expediently with NIH’s National Center for Advancing Translational Sciences (NCATS) and NICHD, regulatory authorities, patient/parent organizations, physicians and other key stakeholders to start a Phase II/III clinical trial to assess the efficacy of the compound for the treatment of NPC. Pending the outcomes of discussions with the U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA), Vtesse will provide an update on anticipated timing for such a trial.
About Vtesse
Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved. The first spin-out company from Cydan Development, Inc., an orphan-drug accelerator that identifies and de-risks programs with therapeutic and commercial potential, Vtesse is working collaboratively with the NIH to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other LSDs. Vtesse is led by a highly experienced management team that has been involved in the development of more than 20 approved drugs and vaccines. Its experienced consortium of investors, led by New Enterprise Associates, has committed initial funding that is expected to bring this compound through pivotal clinical trials. Vtesse is based in Gaithersburg, Maryland. For more information, visit www.vtessepharma.com.
Corporate Contact:
Ravi Venkataramani, Ph.D.
rvenkataramani@vtessepharma.com
Click here to read the official press release in PDF.
[March 25th, 2015 ~ blg]
~Cyclodextrin Update~ 01/07/2015
Update on NPC Cyclodextrin Trial
Leading Life Science Syndicate Commits $25 Million to Series A Funding to Launch Vtesse, Inc., the First Rare Disease Company Spun Out of Cydan Development, Inc.
Additional Resources:
- National Institutes of Health (NIH) News:
NIH Teams with Industry to Develop Treatments for Niemann-Pick Type C - Wall Street Journal (WSJ) Article
Small Biotech Gets Rights to Rare Disease Drug (paid subscription required to read)
Dear NNPDF Families and Friends,
We are pleased to share with the foundations Niemann-Pick disease type C (NPC) families and community a recent development pertaining to the Cyclodextrin clinical trial. The recently incorporated, Vtesse, a rare disease company spun-off from Cydan Development, Inc., which is focused on developing drugs for Niemann-Pick disease Type C (NPC) and other severe diseases with great unmet need, will begin collaborating with the National Institutes of Health on furthering development of Cyclodextrin (VTS-0270) for Niemann-Pick type C.
Vtesse also announced that it has established a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Center for Advancing Translational Sciences (NCATS), each a component of the National Institutes of Health (NIH). Vtesse and NCATS have also entered into a licensing agreement for the current rights held by NIH for the worldwide use of cyclodextrin, delta-tocopherol, and derivatives of tocopherol, alone or in combination, for the treatment of lysosomal storage diseases (LSDs), including NPC. Regulatory orphan designations for the U.S. and EU will be also be transferred to Vtesse.
Vtesse will use the proceeds from the $25 Million raised through its Series A financing to conduct a clinical program for VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for NPC, and to discover and pre-clinically evaluate additional novel drugs for NPC and other lysosomal storage diseases.
For more information on phase 2 & 3 of the Cyclodextrin clinical trials & the transition from NIH to Vtesse, Inc, please review the full press release here:
FOR IMMEDIATE RELEASE
Leading Life Science Syndicate Commits $25 Million to Series A Funding to Launch Vtesse, Inc., the First Rare Disease Company Spun Out of Cydan Development, Inc.
Gaithersburg, MD, and Cambridge, MA, January 7, 2015 – Vtesse, Inc., a rare disease company focused on developing drugs for Niemann-Pick disease Type C (NPC) and other severe diseases with great unmet need, announced today that it has raised $25 million in Series A funding. Vtesse is the first spin-off company for Cydan Development, Inc., an orphan-drug accelerator that shares with Vtesse the same syndicate of leading life sciences investors that are committed to funding additional rare disease companies. New Enterprise Associates (NEA) led the Vtesse financing with participation from Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments.
Vtesse also announced that it has established a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Center for Advancing Translational Sciences (NCATS), each a component of the National Institutes of Health (NIH). Vtesse and NCATS have also entered into a licensing agreement for the current rights held by NIH for the worldwide use of cyclodextrin, delta-tocopherol, and derivatives of tocopherol, alone or in combination, for the treatment of lysosomal storage diseases (LSDs), including NPC. Regulatory orphan designations for the U.S. and EU will be also be transferred to Vtesse.
Vtesse will use the proceeds from its Series A financing to conduct a clinical program for VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for NPC, and to discover and pre-clinically evaluate additional novel drugs for NPC and other LSDs. NPC is a genetic disease associated with mutations in NPC1 and NPC2 genes. NPC affects an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or underdiagnosed. Affected patients are usually identified in early childhood with ataxia and exhibit progressive impairment of motor and intellectual function, and often die before adulthood.
“We’ve launched Vtesse to rapidly advance the clinical development of VTS-270, which we hope to make widely available to the many young patients suffering from the debilitating effects of NPC,” said Ben Machielse, Drs., President and Chief Executive Officer of Vtesse, Inc. “We expect to listen to and learn from the physicians, independent researchers, parents and patients who have worked tirelessly for many years to find a treatment for this devastating disorder. We are grateful for the work they and the NIH have conducted thus far, and we are committed to developing a broadly available treatment for NPC.”
“The launch of Vtesse is an important milestone for Cydan as this new company holds great promise for patients and is an excellent example of collaboration among scientific, patient and investment stakeholders,” said Chris Adams, Ph.D., Founder and Chief Executive Officer at Cydan and an independent member of the Vtesse Board of Directors. “Our goal at Cydan Development is to advance innovative treatments for patients with rare diseases by identifying promising assets, accelerating their pre-clinical and clinical development, selecting the right management team, and, ultimately, creating more companies like Vtesse.”
Vtesse and NIH to Collaborate on VTS-270 Clinical Study
Under the CRADA, Vtesse, NICHD and NCATS intend to collaborate to launch a second VTS-270 clinical study for the treatment of NPC as well as to develop other novel drugs (delta-tocopherol and combinations) for NPC and other LSDs. Under the terms of the agreement, NICHD, which is the current sponsor of a Phase I clinical trial of VTS-270 in patients with NPC, Type 1, will transfer its investigational new drug (IND) application to Vtesse along with all background data on the program. Vtesse will be responsible for all further development of VTS-270 with the intent to seek marketing approval for VTS-270 from U.S. and European regulatory agencies.
“This is an excellent example of how launching a project to study the underlying biology of one disease can lead to advances that hold promise for an entire group of diseases — the NCATS goal of finding what is common among diseases and the translational science process,” said NCATS Director Christopher P. Austin, M.D. “I am grateful to all of the NPC patients, their families and patient support groups who have been equal partners in our efforts to find therapeutic solutions to these devastating disorders.”
Experienced Life Science Industry Team to Lead Vtesse
Biopharmaceutical industry veteran Ben Machielse, Drs., will serve as Vtesse President and Chief Executive Officer. Before founding Vtesse, Drs. Machielse led drug development of Omthera Pharmaceutical Inc.’s recently approved product, EPANOVA®, and was involved in the sale of the specialty drug maker to AstraZeneca. Previously, he held senior executive positions at Centocor, Xoma and MedImmune/AstraZeneca. Vtesse’s management team also includes Allan Darling, Ph.D., Vice President, Technical Operations; Sarah Frech, D.V.M., M.P.H., Vice President, Clinical Research; and Ravi Venkataramani, Ph.D., Chief Business Officer. Each of these individuals has extensive industry experience at companies such as MedImmune/AstraZeneca, Invitrogen/BioReliance, Baxter Healthcare and Genocea Biosciences, and all have been involved in the successful clinical development and commercialization of drugs. The Board of Directors for Vtesse, Inc. includes David Mott, NEA, Board Chair; Ben Machielse, Drs., Vtesse, Inc.; Sara Nayeem, M.D., NEA; Barbara Dalton, Ph.D., Pfizer Venture Investments; Mette Kirstine Agger, Lundbeckfond Ventures; and Carl Goldfisher, M.D., Bay City Capital; as well as veteran rare disease industry experts, Cristina Csimma, Pharm.D., and Chris Adams, Ph.D., both of whom will serve as independent directors.
“Vtesse combines an experienced management team with a strong financial and scientific foundation and a commitment to the NPC community,” said Mr. Mott. “With this vision and support, we expect Vtesse to move VTS 270 forward quickly and with the utmost attention to medical excellence.”
About VTS-270
Vtesse’s lead compound, VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for Niemann-Pick disease Type C (NPC). It is a well characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at NCATS, NICHD and the NIH Clinical Center, as well as under individual compassionate use investigational new drug applications (iINDs) and in other academic labs. Vtesse aims to work expediently with NCATS and NICHD, regulatory authorities, patient/parent organizations, physicians and other key stakeholders to start a Phase II/III clinical trial to assess the efficacy of the compound for the treatment of NPC. Pending the outcomes of discussions with the U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA), Vtesse will provide an update on anticipated timing for such a trial.
About Vtesse
Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved. The first spin-out company from Cydan Development, Inc., an orphan-drug accelerator that identifies and de-risks programs with therapeutic and commercial potential, Vtesse is working collaboratively with the NIH to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other LSDs. Vtesse is led by a highly experienced management team that has been involved in the development of more than 20 approved drugs and vaccines. Its experienced consortium of investors, led by New Enterprise Associates, has committed initial funding that is expected to bring this compound through pivotal clinical trials. Vtesse is based in Gaithersburg, Maryland. For more information, visit www.vtessepharma.com.
About Cydan Development, Inc.
Cydan is an orphan-drug accelerator that identifies and de-risks orphan drug products with significant therapeutic and commercial potential, with the goal of starting multiple companies to develop such therapies. Cydan was launched in 2013 by a management team with extensive drug discovery, clinical development, and business development experience. Cydan is financed by leading life sciences investors NEA, Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments. The accelerator is based in Cambridge, Massachusetts. For more information, visit www.cydanco.comor contact Cydan at info@cydanco.com.
Corporate Contact:
Ravi Venkataramani, Ph.D.
rvenkataramani@vtessepharma.comVtesse Media Contact:
Jamie Lacey-Moreira
PressComm PR, LLC
410-299-3310
jamielacey@presscommpr.comCydan Media Contact:
Dave Connolly
LaVoieHealthScience
617-374-8800, ext. 108
dconnolly@lavoiehealthscience.com
Click here for the PDF Press Release
[Jan 7th, 2015 ~ blg]