Voyager Therapeutics Inc. is getting $30 million up front in a potential $630 million gene therapy deal with Pfizer Inc., the company’s first such agreement since a strategic refocusing effort earlier this year and a much-needed endorsement of a next-generation AAV capsid platform that has shown promising though early stage data.
With new therapeutics under FDA review for migraine, Alzheimer’s disease and seizures, and several rare disease gene therapies in development, stocks included in BioWorld’s Neurological Diseases Index are tracking 6.09% ahead for the year.
Delivering antibodies in the form of their DNA could enable their therapeutic use under several circumstances where traditional antibodies fall short. One of those is resource-poor settings where the current cost of antibodies makes them a nonstarter. Perhaps the largest opportunity to expand antibody use in such settings is for HIV, where broadly neutralizing antibodies have the potential to be the next best thing to a vaccine or a cure – if they can be made to last, for cheap.
Monoclonal antibodies are a triumph of modern medicine. They are also too expensive to be a standard therapy in all but the wealthiest countries. “Having 10% or 15% of your population on antibodies is not sustainable even in wealthy countries,” Rachel Liberatore told BioWorld. Liberatore is director of research and development at Renbio Inc., which is testing the intramuscular delivery of antibody-encoding DNA to prevent and treat infections, including SARS-CoV-2 and HIV.
Shares of Voyager Therapeutics Inc. and its partner, Neurocrine Biosciences Inc., fell in early trading Dec. 23 after Voyager announced an FDA clinical hold on the phase II Restore-1 trial of VY-AADC, an adeno-associated virus-based gene therapy Neurocrine is developing as NBIb-1817 for the treatment of Parkinson's disease. The trial had been paused since at least November as its data safety monitoring board reviewed MRI abnormalities in some study participants.
Bold up-fronts and even bigger milestones defined ambitious neurology deals Abbvie Inc. struck with Voyager Therapeutics Inc. in 2018 and 2019. With vectorized antibodies, they planned to target multiple indications tied to excess aggregations of tau and tragic synucleinopathies. Considerable progress was made, said Omar Khwaja, Voyager's chief medical officer. But despite millions of dollars invested in the programs, Abbvie has now decided to quit the venture, leaving Voyager to either go it alone or find a new partner in its work on the challenging indications.
