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Orphazyme


This page is dedicated to news and information about Orphazyme’s therapies for use in Niemann-Pick Disease Type C (NPC). Information is released as it becomes available.

GLOSSARY OF TERMS


About Orphazyme:

Orphazyme ApS is a Danish biopharmaceutical company, that develops paradigm-changing medicines for the treatment of genetic diseases. The lead program is in development as a treatment for lysosomal storage diseases. This family of genetic disorders consists of more than 45 diseases and includes Niemann-Pick Disease Type C. Lysosomal storage diseases often affect children, most of whom are currently untreatable. For more information, please visit Orphazyme.com.


ORPHAZYME
AIDNPC CLINICAL PROGRAMME UPDATE #14

Now Available Online

CLICK BELOW FOR UPDATES IN SEVERAL LANGUAGES

orphazyme-updates-button

02/27/2017 jjb

ORPHAZYME
AIDNPC CLINICAL PROGRAMME UPDATE #13

Now Available Online

CLICK BELOW FOR UPDATES IN SEVERAL LANGUAGES

orphazyme-updates-button

01/31/2017 jjb

ORPHAZYME
AIDNPC CLINICAL PROGRAMME UPDATE #12

Now Available Online

CLICK BELOW FOR UPDATES IN SEVERAL LANGUAGES

orphazyme-updates-button

01/02/2017 jjb

Christmas Newsletter From Orphazyme
.

ORPHAZYME CHRISTMAS NEWSLETTER

12/27/2016 jjb

ORPHAZYME
AIDNPC CLINICAL PROGRAMME UPDATES

Now Available Online

CLICK BELOW FOR UPDATES IN SEVERAL LANGUAGES

orphazyme-updates-button

 11/28/2016 jjb

AIDNPC CLINICAL PROGRAMME UPDATE:

Orphazyme Expands NPC Study to Three Sites in the USA
.

FULL PRESS RELEASE

10/31/2016 jjb

AIDNPC CLINICAL PROGRAMME UPDATE:

Orphazyme announces update to its Niemann-Pick
Disease Type C Clinical Programme — AIDNPC

Dateline: October 27, 2016
.

NPC COMMUNITY UPDATES FROM ORPHAZYME

10/28/2016 jjb

Press Release from Orphazyme

Dateline:  September 22, 2016
.

FULL PRESS RELEASE     FULL ARTICLE     SUPPLEMENTAL MATERIALS

09/27/2016 jjb

AIDNPC CLINICAL PROGRAMME UPDATE:

Orphazyme announces update to its Niemann-Pick
Disease Type C Clinical Programme — AIDNPC

Dateline: August 26, 2016

NPC Community Updates from Orphazyme

08/27/2016 jjb

AIDNPC CLINICAL PROGRAMME UPDATE:

Orphazyme announces update to its Niemann-Pick
Disease Type C Clinical Programme — AIDNPC

Dateline: July 29, 2016

Click links below for NPC Community Updates from Orphazyme

July 28, 2016     June 29, 2016          April 28, 2016
 March 31, 2016      January 14, 2016          November 25, 2015

07/29/2016 cdk

AIDNPC CLINICAL PROGRAMME UPDATE:

Orphazyme announces update to its Niemann-Pick
Disease Type C Clinical Programme — AIDNPC

Dateline: June 29th, 2016

Click links below for NPC Community Updates from Orphazyme

June 29, 2016          April 28, 2016          March 31, 2016
January 14, 2016          November 25, 2015

07/01/2016 jjb

The NNPDF Central Office has received
the following press release from Orphazyme
with regards to FDA Grants Fast Track Designation
for the Phase III Clinical Investigation of Arimoclomol
as a treatment of Niemann-Pick Disease Type C

Dateline: June 27th, 2016
FULL PRESS RELEASE

06/27/2016 jjb

Orphazyme Begins Dosing of Patients in
Arimoclomol “AIDNPC” Clinical Trial Programme

Copenhagen, Denmark, 15 June 2016. Orphazyme announced today that the first two patients have been dosed with Arimoclomol in the Phase III ‘AIDNPC’ Niemann-Pick disease type C clinical trial programme.
.

CLICK HERE FOR COMPLETE ANNOUNCEMENT

06/17/2016 jjb

The NNPDF Central Office has received the
following press release 
from Orphazyme with regards
to its arimoclomol study for treatment of

Niemann-Pick Disease Type C

Dateline: June 6th, 2016
Click here for the full press release.

06/06/2016 jjb

Clinical Update:
Orphazyme announces update to its
Niemann-Pick
Disease Type C Clinical Programme — AIDNPC

Dateline: April 28th, 2016

Click below for NPC Community Updates from Orphazyme

June 29, 2016

April 28, 2016          March 31, 2016

January 14, 2016          November 25, 2015

May 6, 2016 ~ jjb

Clinical Update:
Orphazyme announces update to its
Niemann-Pick
Disease Type C Clinical Programme — AIDNPC

Dateline: March 31st, 2016

001 OBSERVATIONAL STUDY
Orphazyme’s clinical program AIDNPC that investigates the orally available small molecule arimoclomal in Niemann-Pick disease type C is continuing with enrollment in Europe in the 001 Observational Study.

Families should follow enrollment criteria as provided on the clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit clinicaltrials.gov and refer to study reference number: NCT02435030

The title of the trial is listed as: “A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C.”

002 INTERVENTIONAL STUDY
The 002 Interventional Study is progressing well. Families should follow enrollment criteria as provided on the clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit clinicaltrials.gov and refer to study reference number: NCT02612129

The title of the trial is listed as: “Arimoclomol Prospective Study in Patients Diagnosed With Niemann-Pick Disease Type C”

Patients are encouraged to contact their physicians regarding this information and their physicians will be able to further contact the trial transparency team at Orphazyme provided in the clinicaltrials.gov clinical trial information page.

April 21, 2016 ~ jjb

Clinical Update:
Orphazyme announces update to its
Niemann-Pick
Disease Type C Clinical Programme — AIDNPC

Dateline: September 10th, 2015

Orphazyme’s clinical program AIDNPC that investigates the orally available small molecule arimoclomal in Niemann-Pick disease type C is initiating in Europe, with recruitment commencing in September.  If proven effective, the program will be moved as quickly as possible to registration to become available for all patients suffering from Niemann-Pick disease type C.

Click here to read the full Press Release from Orphazyme

Families should follow enrollment criteria as provided on the clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit clinicaltrials.gov and refer to study reference number: NCT02435030

The title of the trial is listed as: “A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C.”

Patients are encouraged to contact their physicians regarding this information and their physicians will be able to further contact the trial transparency team at Orphazyme provided in the clinicaltrials.gov clinical trial information page.

Sept 16th, 2015 ~ blg

Orphazyme Announcement:
Orphazyme Clinical Trial Development Update on Arimoclomol in NPC
Dateline: 05/11/2015

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation has been advised by Orphazyme that an “Observational” clinical study for NP-C, tied to their clinical development program of arimoclomol in NPC, has been registered and made available at clinicaltrials.gov.  The link to the information pertaining to just this trial is at: https://clinicaltrials.gov/ct2/show/NCT02435030?term=02435030&rank=1.

This information was updated as of April 30, 2015.

At this time, no site in the United States has been announced and currently the study is not open for participant recruitment.  Orphazyme has indicated that they are targeting a clinical trial to start date in the United States toward the end of first quarter 2016.  As we receive further announcements and updates, it will be posted to our NPD family community!

You can read either the full text view or the tabular view depending upon your preference.  The text allows you to see the inclusion and exclusion information along with the primary outcome measures they will be looking at.

Sincerely,

Nadine M. Hill
Executive Director
National Niemann-Pick Disease Foundation


Orphazyme Announcement:
Orphazyme Kicks off Clinical Program of Arimoclomol in Niemann-Pick Disease Type C
Dateline: 04/15/2015

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation is pleased to share with you the following announcement received from Orphazyme ApS of an upcoming clinical trial for Niemann-Pick Disease Type C.  Orphazyme ApS (Copenhagen, Denmark) develops new therapies for the treatment of rare and genetic diseases.

Below is the official announcement as provided to the NNPDF, which is being shared globally today by Orphazyme.

Orphazyme Kicks off Clinical Program of Arimoclomol in Niemann-Pick Disease Type C

Orphazyme has interacted with key opinion leaders and experts from around the world to design a clinical programme that appropriately assesses the therapeutic response of Arimoclomol in Niemann-Pick disease type C.  Arimoclomol is a small-molecule inducer of heat shock proteins. It is an orally available experimental drug that readily crosses the blood-brain barrier.

Arimoclomol’s safety profile has been established through extensive pre-clinical studies and multiple Phase I clinical trials. After the meaningful feedback from EU and US regulators, the Orphan Drug Designations (EMA and FDA), and successful fundraising, Orphazyme has entered into the phase of clinical trial applications.

We are pleased to inform that submissions to various countries have been undertaken, and that the French competent authority (ANSM) has already approved the initiation of the “Observational” study of the clinical programme of Arimoclomol in Niemann-Pick disease type C.  Submission to the relevant authorities is part of this undertaking. Upon the respective approvals being obtained from the corresponding regulatory agencies and ethics committees, enrolment of patients will take place.

The programme is composed of two studies: a) an “Observational” study will start a staggered enrolment in the second quarter of 2015; b) after the 6-month follow-up, all the patients that complete the “Observational” study will be offered to enter into the “Interventional” study, a Phase II/III clinical trial with Arimoclomol. This will be a multi-centre clinical programme involving clinical centres throughout Europe. The clinical development programme also includes a clinical trial in the USA.

Carlos R. Camozzi
Chief Medical Officer of Orphazyme
E-mail: crc@orphazyme.com

As more information becomes available we will continue to update this page, as well as, our social media sites.

April 15th, 2015 ~ blg

FDA Grants Orphan Drug Designation for Arimoclomol
Dateline: 01/27/2015

Dear NNPDF Families and Friends,

Orphazyme today announced that the Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted an orphan drug designation for Orphazyme’s Arimoclomol as a new treatment for Niemann-Pick disease type C.

The US orphan drug designation complements the corresponding EMA orphan drug designation that was granted for the program in 2014.

“The recent successful completion of a €20m Series B financing round, together with the FDA and EMA orphan drug designations, establish a solid foundation for initiating our clinical studies in NP‑C with the purpose of providing arimoclomol as a safe and effective therapeutic option to patients suffering from this devastating disease as well as relief to their families,” says Dr. Carlos R. Camozzi, Chief Medical Officer of Orphazyme.

Orphazyme plans to start a clinical trial in NP-C in 2015.

See the FDA log of its decision here and the EMA log here.

For further information:
Carlos Camozzi, CMO at Orphazyme ApS
E-mail: crc@orphazyme.com

Click here to view the full press release

“The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet.”

Jan 27th, 2015 ~ blg

Dear NNPDF Families and Friends,

RE:  Orphazyme ~ “Orphazyme ApS (www.orphazyme.com) was founded in June 2009 with the aim of developing heat shock protein based therapy for the treatment of diseases caused by defects in the function and/or metabolism of proteins.”

The NNPDF is making press release announcements from Orphazyme, pertaining to their advances towards a clinical trial for Niemann-Pick Disease type C, available to the wider NPC Community.

Listed below are the links back to the Orphazyme APS (LLC) website to read the press releases.

Jan 15th, 2015 ~ blg

Orphazyme ApS (LLC)*
Clinical Trial Updates for Niemann-Pick Type C Disease
September 8th, 2014

Update from: Anders M. Hinsby
Orphazyme Chief Executive Officer

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation is pleased to share with you the following announcement received from Orphazyme ApS of an upcoming clinical trial for Niemann-Pick Disease Type C.  Orphazyme ApS (Copenhagen, Denmark) develops new therapies for the treatment of rare and genetic diseases.

Below is the official announcement as provided to the NNPDF, which is being shared globally today by Orphazyme.  As more information becomes available we will continue to update this page as well as our social media sites.

Orphazyme moves towards clinical development in Niemann-Pick type C

Orphazyme has identified and evaluated two candidate products through preclinical development for Niemann-Pick type C. One is the recombinant human protein HSP70 and the other is an orally available small molecule, Arimoclomol that works as an inducer of heat shock proteins, including HSP70**.

Both product candidates have produced encouraging biochemical and disease-modifying effects in studies in the Niemann-Pick type C mouse model performed in collaboration with Prof. Frances Platt (University of Oxford). Both compounds have successfully completed toxicity studies and are now enabled for clinical trials. In the third quarter of 2014 Orphazyme will select the product candidate for a clinical Niemann-Pick type C trial. We expect to start the multi-centre clinical trial in patients in the first quarter of 2015. Final inclusion criteria and participating clinical sites are still to be confirmed.

It is a priority for Orphazyme to ensure the clinical and regulatory feasibility of our program in continuous dialogue with patient organizations and national regulatory agencies including EMA and the FDA.

Orphazyme’s Chief Scientific Officer and Founder, Dr. Thomas Kirkegaard Jensen, and Chief Medical Officer Dr. Carlos R. Camozzi, MD, will present the preclinical results and supply further information about the planned clinical trial at the Niemann-Pick Disease Group (NPDG) UK meeting, 26-28. September 2014.

We look forward to sharing the exciting data and plans with the patient community.

Kindest regards,
Anders M. Hinsby, Orphazyme Chief Executive Officer

*Orphazyme ApS is founded on the pioneering research from the laboratory of Professor Marja Jäättela, and with the aim of converting human chaperone proteins into effective therapies for degenerative diseases. Orphazyme’s novel technology also has the potential to address the neurodegenerative aspects of these devastating diseases.

Orphazyme’s core program is developing heat shock protein based therapies for the treatment of diseases caused by defects in the function and/or metabolism of proteins. The company focuses on severe and often fatal diseases with a very high unmet need.

What is HSP70?**
Heat shock proteins (HSP) are a class of functionally related proteins involved in the folding and unfolding of other proteins and are named according to their molecular weight. Proteins with similar structure exist in virtually all living organisms.

Production of high levels of HSPs can be triggered by exposure to different kinds of environmental stress conditions, such as infection, inflammation or exercise. They also occur under non-stressful conditions, acting as “monitors” for the cell’s proteins, by carrying old proteins to the cell’s “recycling bin” (proteasome) and helping newly synthesised proteins to fold properly. These activities are part of a cell’s own repair system, called the “cellular stress response” or the “heat-shock response”.

HSP70 chaperones are required for key cellular processes. A chaperone is a protein that assists folding/unfolding in molecular biology. The HSP70s are an important part of the cell’s machinery for protein folding, and help to protect cells from stress. HSP70 also aids in trans-membrane transport of proteins, by stabilizing them in a partially folded state.

Please refer to their website for more information: http://www.orphazyme.com/

Sept 8th, 2013 ~ blg

Orphazyme Update – 10/31/2013
Orph001 (rhHSP70) Project Progress

Dear Families and Friends,

The NNPDF Central Office received an update pertaining to the current research and clinical trial work as it relates to Orphazyme and their proposed clinical trial titled: Orph001 (rhHSP70).

Dear Reader,

We would like to update you about the progress of the Orph001 clinical development programme.

With the aim of reaching the highest achievable quality in standards and procedures certain amendments have been introduced into the Orph001 development programme to ensure full dose definition, and high alignment to regulatory recommendations and requirements in Europe and US.
Please, let us give you a short review of our development program and the steps ahead.

Orphazyme History and Lead R&D Programme HSP70

Orphazyme ApS was founded in June 2009 with the aim of developing heat shock protein based therapy for the treatment of diseases caused by defects in the function and/or metabolism of proteins. The cornerstone in the company is the research of its scientific founders, Professor Marja Jäättela and Thomas Kirkegaard Jensen, on the cytoprotective properties of human heat shock proteins (HSPs), a class of functionally related proteins involved in the folding and metabolism of other proteins.

Orphazyme leading lysosomal storage disease (LSDs) programme, Orph001, a recombinant version of human HSP70 (rhHSP70), is being developed for the treatment of Niemann-Pick Disease type C.

rhHSP70 (OR0005) has shown to be effective in correcting conditions of lysosomal functional deficiency and concurrent cellular stress. The initial studies were published in Nature by Orphazyme’s scientific founders (Kirkegaard et al. Nature 2010, 463:549-53).

Non-clinical development

Orphazyme has elucidated OR0005 (rhHSP70) therapeutic potential by demonstrating in vitro a remarkable reversion of the cellular pathology in primary cells from NP-C patients as well as other LSDs, including all the major sphingolipidoses. The beneficial effect of OR0005 on primary cells was observed after a single exposure to rhHSP70.

Subsequently, in vivo studies in murine models of NP-C, performed at Oxford University, have provided pharmacological proof of concept that the treatment with OR0005 may modify the progressive clinical manifestations of the Niemann-Pick Disease type C. In addition to the marked disease modification, biochemical analyses of CNS lipid storage and analyses of the CNS penetration of HSP70 show that it enters into the brain and it reduces the pathological lipid storage burden in the selected neurodegenerative models. Meaningful improvements were observed in biological and pathological variables including the symptoms arising from compromised CNS functionality. A manuscript is being prepared for submission in Q4 2013.

Cynomolgus monkey (Macaca fascicularis) presents 100% interspecies (humans and non-human primates) homology of HSP70. The cynomolgus monkey is therefore considered to be the most suitable non-rodent species for the required toxicity studies. Results are expected before the initiation of the clinical development programme.

Clinical Development

NP-C is an extremely rare disease. The estimated prevalence in the EU is 0.1 cases in 10,000. Clinical symptoms of NP-C and natural disease progression show a broad inter-individual variability. This reality represents a significant challenge for clinical development design. In order to obtain an efficacy signal in the clinical development setting, the participating patient population must be as homogeneous as possible regarding metabolic and immunological parameters, and clinical and biological disease status.

The clinical programme is as follows:

  • A PK, safety and dose escalation Phase I study is under preparation to be started in 2014.
  • The initiation of enrolment of NP-C patients for the “observational study” has now been set up by second half of 2014,
  • The Phase II interventional Clinical trial in the already selected population will follow. This study will evaluate safety, efficacy and quality of life within 52 weeks follow-up upon biological, clinical and imaging markers. The objective of the study is to define the clinical benefit of OR0005 in the NP-C patients.
    Regulatory Steps

Close and fruitful interactions with health authorities (national and EMA/FDA) are going on to review the progress in research and development outcome. Scientific advice meetings were already held from 2011 to 2013 with the Danish Agency (DMKA), Swedish Medicinal Products Agency (MPA), United Kingdom Medicines an Healthcare products Regulatory Agency (MHRA) and EMA (July 2013). The EMA’s scientific advice enhanced company’s focus in key areas like the selection of representative biomarkers based on expertise, knowledge and understanding of newer definitions and publications.

Plans for protocol optimization

Orphazyme concentrates efforts to increase the standards in protocol optimization and clinical operations procedures. We are confident that these improvements will bring even higher certainty in regulatory steps for the benefit of NP-C patients.

We will keep you informed about the latest development and timelines as more information is made available.

Click here for a summary of the information available from Orphazyme originally provided & developed by the NPD UK Group ~ Sept-Oct 2013.

Nov 5th, 2013 ~ blg