Dive Brief:
- Neurocrine Biosciences is pulling out of a partnership to develop Voyager Therapeutics’ Parkinson’s disease gene therapy, several weeks after the Food and Drug Administration placed a clinical hold on a Phase 2 study testing the treatment.
- Neurocrine’s decision was based on a “portfolio review and prioritization” of assets, Voyager said in a statement Tuesday. Neurocrine confirmed the move in a filing with the Securities and Exchange Commission, without commenting further.
- Termination of the companies’ collaboration in Parkinson’s, which takes effect Aug. 2, won’t affect work on three other programs between the two. Voyager said it will continue to support Neurocrine, which is the sponsor of the trial on hold, as it responds to requests from the FDA about data.
Dive Insight:
Neurocrine’s move isn’t entirely surprising after safety concerns prompted the FDA clinical hold, but it is a disappointment, wrote Brian Skorney, an analyst at Baird, in a note to investors. “The collaboration termination does not bode well for the future of the program,” he wrote.
Voyager said it’s still evaluating the financial impact of Neurocrine’s decision and subsequent steps for its lead program, known as VY-AADC. The company’s shares tumbled 15% in early trading before paring back some of those losses. The stock has lost roughly 70% of its value since a recent peak in the summer of 2019.
Voyager’s gene therapy has a long history, and development has taken several twists and turns. The Massachusetts-based biotech entered a licensing agreement with Sanofi in 2015, only to see the French drugmaker decide against picking up ex-U.S. rights to the therapy in 2017. Neurocrine then jumped in as a partner in 2019, promising development funding and milestone payments.
The RESTORE-1 Phase 2 trial testing VY-AADC was already facing challenges from the COVID-19 pandemic when an independent committee overseeing the trial asked for a pause to evaluate data. Then late last year, the FDA placed a clinical hold on the program because of abnormalities seen in MRI scans of some participants.
More broadly, the FDA appears to be giving close scrutiny to clinical data and manufacturing processes for gene therapies as the field rapidly expands, and Neurocrine and Voyager aren’t the only ones to hit setbacks in Parkinson’s. Last year, Axovant delayed enrollment of its Parkinson’s treatment until 2022 because the development of a new manufacturing process was taking longer than expected.
Voyager’s gene therapy is administered into the brain via an operation guided by MRI scans. Treatment is designed to help the body convert levodopa, an active ingredient in standard Parkinson’s treatment, into the natural brain substance dopamine, which is needed to help prevent the tremors, stiffness and slow movements associated with the disease.
Voyager was also exploring another approach, which it dubbed “vectorized antibodies,” and had enlisted AbbVie as a development partner. In August of last year, however, AbbVie terminated its collaboration agreement and handed full rights back to Voyager.
