The FDA stopped Voyager gene therapy to treat Huntington's disease

October 13, Voyager Therapeutics shares fell more than 11 percent in pre-market trading after the company announced that its experimental gene therapy for Huntington's disease had been halted before it went to trial.Later on Tuesday, Cambridge, Massachusetts-based Voyager announced that the FDA had issued a clinical stay on its application for a new drug (IND) for the company's gene therapy VY-HTT01 for huntington's disease. Voyager submitted an IND application for clinical studies of the drug program to the FDA in September. The FDA said it was shelving the project mainly because it was waiting for some chemical, manufacturing and control (CMC) issues to be resolved.In a recent announcement, Voyager said it expects to receive some feedback from the FDA on CMC issues over the next 30 days and plans to address them in order to initiate clinical studies.In addition to Voyager, other biotech companies are actively developing treatments for the disease. Last month, Vaccinex released data for a promising Phase 2 clinical study, but the study did not reach a common primary endpoint. In June, uniQure launched a clinical study of huntington's disease gene therapy AMT-130, which uses an irrational viral vector to deliver DNA to target cells. On the 13th of this month, UniQure announced that two more patients had received infusions from AAV gene therapy. Voyager's progress in this area will be greatly delayed.VY-HTTO1 is Voyager's most advanced gene therapy candidate. Prior to that, Voyager and Sanofi Genzyme had been working together on the early development of VY-HTT01. In fact, since 2015, Voyager has worked with Sanofi on gene therapy programs for a variety of central nervous system diseases, including Parkinson's disease, Friedreich's syndrome, and Huntington's disease. The former is responsible for research and development activities and implements projects through proof-of-concept. At the time, Sanofi was entitled to commercial rights to the projects, while Voyager held commercial rights to several research and development assets in the United States and shared U.S. profits from the Huntington's disease candidate VY-HTTO1 with Sanofi. To that end, Voyager also received an advance of $100 million and was committed to an estimated $745 million in development and sales milestone payments.In October 2017, however, Sanofi waived its right to enter a 2/3 clinical study of Parkinson's disease. In June 2019, Sanofi relinquished its options for Friedreich's Common Disorders and Huntington's disease program, modified its partnership, and eventually acquired two gene therapies for non-central nervous system adaptation using adeno-related viral shell technology, and all rights to VY-HTT01 were ultimately owned by Voyager.But before reorganizing the partnership with Sanofi, Voyager had found new partners for some of the projects in its product line. In January 2019, Neurocrine paid $165 million in advance for the use of Four Gene Therapy programs at Voyager, including Forkinson and Freemasonry Disorders. Once the data from the Second Phase of Parkinson's Research, Voyager can choose to share profits and costs with Neurocrine, or give up global copyrights in exchange for milestones and royalties.In addition to VY-HTTO1, the company has a research gene therapy VY-AADC (NBIb-1817) related to neuroendocrine biosciences. Last month, Voyager and Neurocrine announced three years of validity and safety data for patients with Parkinson's disease receiving VY-AADC treatment. Studies have shown that patients treated with one-time doses of the research gene therapy VY-AADC continued to improve their motor function, including longer exercise "continuous" periods without disturbing movement disorders, as well as a reduction in the third part of the unified Parkinson's Disease Assessment Scale (UPDRS) for Parkinson's patients and a decrease in medication.Source: 1.Voyager Official website 2. Voyager Takes a Hit After FDA Places Hold on Gene Therapy for Huntington's Disease 3.Voyager's Huntington gene therapy face FDA hold shans to manufacturing systems 4.Source:Supplied With Update vY-HTT01 program
(Sina Pharmaceutical News)