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January 2015 eNewsletter



Scientific First for Rare Diseases:

Small Biotech (Vtesse, Inc.) Gets Rights to Rare Disease Drug

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: CLICK HERE

Ara Parseghian Medical Research Foundation – APMRF
Dana’s Angels Research Trust – DART
Support Of Accelerated Research for NPC Disease – SOAR
National Niemann-Pick Disease Foundation – NNPDF
Vtesse, Inc.  –
Vtesse – January 7th, 2015 Press Release

The NNPDF Central Offices will continue to assist with the dissemination of information pertaining to the news and updates relating to the ongoing efforts associated with this clinical trial.  Should you have any questions regarding this post, please feel free to contact the NNPDF Central Offices at: or the foundation web site at: .  In addition, for a complete historical timeline on the Cyclodextrin Clinical trial, please refer to the NNPDF’s Cyclodextrin web page for more details.

Clinical Trial News and Updates

Moving into 2015, the foundation is slated to support three clinical trial therapies for Niemann-Pick type C and one for Niemann-Pick Disease Type B (or Acid Sphingomyelinase Deficiency (ASMD), for a total of four clinical trials! We’ve also been fortunate to fund NNPDF Fellowships for both NPC and ASMD in 2014 thanks to your continued contributions!

Below are links to the latest clinical trial news, updates & history available:

Cyclodextrin Trial For Niemann-Pick Disease Type C ~ Pediatrics
Dateline: 01/07/2015

Clinical Trial Updates for NPC Adults HDAC Inhibitor ~ Vorinostat 
Dateline: 09/12/2014
Update 2015 ~ Continuing to Recruit

Niemann-Pick Disease Type B Adult & Pediatrics ERT Clinical Trial Update 
Dateline: 12/09/2014

Clinical Trial Update for Niemann-Pick C
Dateline: 01/15/2015

Visit the Fellowships jointly sponsored by the NNPDF and CCNNPDF as well as Research Grants jointly sponsored by the NNPDF and CCNNPDF pages for additional information on NNPDF funded research.

The Infiniti Coaches Charity Challenge

NPD Community Worldwide Unite!
Vote Daily ~ Vote Matt Painter!

We have an unparalleled opportunity to raise $100,000 towards NPC research, but it will only happen if YOU take action and encourage others to do the same. It’s easy! Vote once a day online between now and March 15th to push the Trent and Julie Smith Family Foundation “BReaK Thru Fund” (three of their children have been diagnosed with Niemann-Pick Disease) for NPC research to the finish line and the top prize of $100,000. 

Click Here to Vote for Matt Painter!

Matt Painter, Purdue University Coach and the Smith Family BReaK Thru Fund are working together with the NPC community to vote Matt Painter into the final round! With your help, NPC research could garner $100,000, but only if you vote daily! We CAN do this if everyone gets behind this project.

The first round of voting opens on January 5th, 2015 and continues through January 25th, 2015 when 24 Coaches out of the initial 48 will advance to round two of four total.  Vote daily for Matt Painter to ensure he makes it to the final round!

Share this with your families, share this with your friends and encourage them to do the same. Alone we are rare, but together we are strong!

2015 NNPDF Annual Board Meeting & NNPDF / CCNPDF Board Retreat

The 2015 Annual Board Meeting of the National Niemann-Pick Disease Foundation (NNPDF) will be held on Thursday, March 12th thru Sunday, March 15th, 2015, in Chicago Illinois. The NNPDF Board of Directors will be joined by the Board Members of our sister-chapter, the Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) for a board development and retreat meeting. All family members of the NNPDF are welcome to attend and encouraged to join us as we recap the year past and plan for the months and years ahead!  If you are interested in attending the annual meeting and taking part ~ please contact the NNPDF Central Offices for more details. 920-563-0930 ~

9 8.16

PO Box 49, Fort Atkinson, WI 53538-0049
Phone: 920-563-0930  |  Toll Free: 877-287-3672 |


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