AbbVie and Voyager Therapeutics said today they will launch a potentially $1.5 billion-plus gene therapy collaboration—the companies’ second billion-dollar-plus alliance in as many years, with the new partnership focused on treating Parkinson’s disease and other diseases characterized by abnormal accumulation of misfolded alpha-synuclein protein.
The companies said they plan to develop “one or more” treatments that apply Voyager’s vectorized antibody platform and approach to delivering antibodies across the blood-brain barrier.
Voyager uses adeno-associated virus (AAV) capsids designed for a one-time intravenous delivery of genes that encode antibodies that target and bind to the alpha-synuclein protein. Voyager says its approach could deliver higher levels of therapeutic antibodies in the brain compared with current systemic administration of antibodies.
“Our scientific platform allows us to develop unique AAV gene therapies that are designed to knock down disease-causing gene expression, increase the expression of missing proteins, or enable the expression of therapeutic antibodies through vectorization,” Voyager president and CEO Andre Turenne said in a statement. “We are excited to expand our efforts towards pathological species of alpha-synuclein given its role in the progression of disease, and AbbVie is the ideal partner to advance this new target and therapeutic modality.”
Parkinson’s is a key disease area of focus for Voyager. Just last month, Neurocrine Biosciences agreed to develop and commercialize Voyager’s gene therapy programs for Parkinson’s disease and Friedreich’s ataxia, plus two other programs to be determined, through a collaboration that could generate more than $1.865 billion for Voyager.
Among gene therapy candidates covered by that collaboration are Voyager’s lead candidate, VY-AADC for Parkinson’s. Neurocrine has agreed to fund the Phase II/III pivotal program for VY-AADC, which has advanced to the Phase II RESTORE-1 trial (NCT03562494). RESTORE-1 dosed its first patient in December 2018. The following month, after a Type B meeting with the FDA, Voyager agreed to increase RESTORE-1’s target number of patients to between 75 and 100, and conduct a staggered-parallel Phase 3 trial (RESTORE-2) of similar size and design to RESTORE-1.
Earlier $1.1B tau partnership
The new collaboration comes a year and two days after AbbVie and Voyager launched a potentially $1.1-billion-plus partnership focused on developing and commercializing gene therapies for tau-related neurodegenerative diseases that include Alzheimer’s disease, frontotemporal dementia, and progressive supranuclear palsy.
That partnership is in its lead candidate selection phase, Voyager states on its website.
“The expansion of AbbVie’s partnership with Voyager represents the potential we see in the ability of its vectorized antibody platform to surpass the blood-brain barrier and more effectively deliver biologic therapies,” said Jim Summers, PhD, vice president, discovery neuroscience research, AbbVie. “We are hopeful that Voyager’s technology will enable further development of transformative treatments for patients with neurodegenerative diseases.”
In the new partnership, AbbVie agreed to pay Voyager $65 million upfront and up to $245 million in preclinical and Phase I development option payments. AbbVie may exercise one or more exclusive development options to select up to a total of four research compounds and their corresponding product candidates to proceed to development, Voyager stated in a regulatory filing.
AbbVie also agreed to pay Voyager up to an additional $728 million in payments tied to achieving development and regulatory milestones for each alpha-synuclein vectorized antibody compound developed, and up to $500 million in commercial milestone payments.
In addition, Voyager is eligible to receive tiered royalties from AbbVie on the global commercial net sales of each alpha-synuclein vectorized antibody developed.
Investors responded to Voyager’s expanded partnership with AbbVie with a buying surge that sent Voyager’s share price rising about 18% from yesterday’s closing price of $10.68, to $12.58 in early market trading this morning as of 10:01 a.m.
