Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50

Huntington’s Trial Completes Enrollment

By Drug Discovery Trends Editor | June 21, 2012

Raptor Pharmaceutical Corp. announced that enrollment is complete in its Phase 2/3 clinical trial of cysteamine bitartrate delayed-release capsules (RP103) for the potential treatment of Huntington’s Disease. The 96-patient, Phase 2/3 clinical trial is being conducted under a collaboration agreement with The Centre Hospitalier Universitaire d’Angers (“CHU d’Angers”), with 8 active clinical sites throughout France.

The clinical trial is an 18-month, double-blind, placebo-controlled study to be followed by an open-label extension study with all patients taking RP103 for up to an additional 18 months. The primary end point of the clinical trial is based upon the Unified Huntington’s Disease Rating Scale (UHDRS). Blood levels of brain-derived neurotrophic factor (BDNF) are being measured as a secondary endpoint. Under the collaboration agreement with CHU d’Angers, Raptor supplies RP103 and placebo capsules for the clinical trial and open-label extension study in exchange for regulatory and commercial rights to the clinical trial results. Clinical expenses of the study are covered by a grant from the French government (PHRC 2004-03bis CYST-HD). Interim results of this study following 18 months of treatment are expected to be announced in the first half of calendar 2014.

Christophe Verny, M.D., of CHU d’Angers, said, “Having completed enrollment of patients, we are excited to analyze the data and examine the potential of delayed release cysteamine as a neuroprotectant for treating Huntington’s Disease. We hope this clinical trial will indicate whether RP103 could potentially lead to a new treatment for this serious, debilitating disease.”

“The completion of enrollment in the Phase 2/3 clinical trial is an exciting milestone for this program and we look forward to next announcing interim results of the blinded phase of this clinical trial in the first half of calendar 2014,” said Dr. Patrice P. Rioux, Raptor’s Chief Medical Officer. “Preclinical data published by researchers from the Curie Institute in France suggested that cysteamine increased brain and blood levels of BDNF, a growth factor known to be deficient in Huntington’s Disease patients. We look forward to examining the results of this clinical trial to see if RP103 could slow the progression of Huntington’s Disease. At present, the standard of care for Huntington’s Disease is limited to therapies that can help to minimize some of the symptoms, but do not slow disease progression by targeting the underlying causes of the disease.”

Raptor holds exclusive worldwide licenses to intellectual property related to cysteamine for the potential treatment of Huntington’s Disease from the University of California, San Diego and from the Weizmann Institute of Science in Israel and Japan’s Niigata University.

In 2008, Raptor received FDA orphan drug designation for cysteamine for the potential treatment of Huntington’s Disease.

Date: June 18, 2012
Source: Raptor Pharmaceutical Corp.


Filed Under: Drug Discovery

 

Related Articles Read More >

Takeda Pharmaceutical in the Drug Discovery & Development Pharma 50
Takeda’s Takhzyro fares well in pediatric hereditary angioedema study
Novartis in the Pharma 50
Novartis to cut up to 8,000 positions
Gilead Sciences in the Drug Discovery & Development Pharma 50
Gilead resubmits application to FDA for twice-yearly HIV drug lenacapavir
George Floyd mural
How the pandemic and George Floyd made clinical trial diversity a priority

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.

Need Drug Discovery news in a minute?

We Deliver!
Drug Discovery & Development Enewsletters get you caught up on all the mission critical news you need. Sign up today.
Enews Signup
Drug Discovery and Development
  • MASSDEVICE
  • DEVICETALKS
  • Medical Design & Outsourcing
  • MEDICAL TUBING + EXTRUSION
  • MEDTECH 100
  • Medical Design Sourcing
  • Subscribe to our Free E-Newsletter
  • Contact Us
  • About Us
  • Advertise With Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2022 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50