Dive Brief:
- Wave Life Sciences got a significant vote of confidence in its technology after GSK agreed to a four-year deal to develop genetic medicines together.
- Under an agreement announced Tuesday, GSK will pay $120 million upfront and make a $50 million equity investment, extending Wave’s cash runway into 2025. The deal also includes hundreds of millions of dollars in potential milestone payments for Wave and an option to expand and extend the collaboration later.
- In return, GSK will gain access to Wave’s drug development platform and work with the company on eight preclinical programs it chooses to advance as well as three advanced by Wave. The British drugmaker will also get exclusive rights to Wave’s preclinical treatment for a rare disease called alpha-1 antitrypsin deficiency that affects the liver and lungs.
Dive Insight:
The partnership centers on oligonucleotides, short strands of DNA or RNA that can be used in a variety of ways to fix genetic issues that cause disease. GSK already has two oligonucleotide therapies in clinical trials, one entering Phase 3 for hepatitis B and another moving to Phase 2 for non alcoholic steatohepatitis, or NASH.
Wave’s technology and preclinical candidate seem to fit well with GSK’s focus on lung and liver diseases. The British company is a leader in the respiratory market, with blockbuster drugs such as Trelegy.
Wave’s alpha-1 antitrypsin deficiency program uses a technology called AIMer, designed to redirect natural systems in the body to correct mutations in an RNA transcript without making permanent changes to the genome.
The deal ranks among the strongest in RNA editing and underscores the value of the approach, Cantor Fitzgerald analyst Jennifer Kim wrote in a note to clients.
Wave shares shot up Tuesday after the news and continued their climb Wednesday, jumping 55% to above $6 apiece in morning trading. The stock has been battered in recent years after the company was forced to shelve potential therapies for Duchenne muscular dystrophy and Huntington’s disease. Wave went public in 2015, selling shares for $16 a piece; the stock soared above $50 in 2018 before the research setbacks.
Alpha-1 antitrypsin deficiency has also drawn the interest of Vertex Pharmaceuticals, which secured regulatory clearance in October to begin a clinical trial of its own candidate for the disease.