Avidity Biosciences, Inc.’s breakthroughs in RNA therapeutics are bringing hope for patients with rare skeletal and muscular diseases and bolstering its own bottom line.
Avidity last week announced a global licensing and research collaboration with Bristol Myers Squibb (BMS) with potential cumulative payments of up to $2.3 billion. The deal is an expansion of a 2021 research collaboration with BMS subsidiary MyoKardia.
The BMS collaboration will focus on the discovery, development and commercialization of new cardiovascular targets using Avidity’s Antibody Oligonucleotide Conjugates (AOC) platform, which solves “one of the biggest challenges” in the RNA therapeutic space: “how to get your RNA to the right cell and tissue type,” said Avidity President and CEO Sarah Boyce. “What we’ve been able to do with our AOC platform is we use our antibody as, basically, a delivery vehicle to get our RNA to the cell we want to get to.”
Unlike the mRNA COVID vaccine, which spurred the body to make a protein the cell, Avidity’s AOC platform delivers RNAs is to do something called “silencing,” Boyce said. “We’re looking for the body to stop producing proteins that it shouldn’t be producing.”
Proof of Concept
Last year, Avidity made a breakthrough with the platform, showing for the first time ever in the RNA space that RNA can be delivered directly to muscle cells in patients with a rare genetic disease and see signs of early functional change.
“Essentially, we proved the concept – showed we can deliver RNA to where it needs to be, hit our target and get change,” Boyce said.
Avidity and BMS are now setting out to prove that AOCs can deliver RNA therapeutics top the heart.
“This is opening up a brand-new space for RNA therapies,” Boyce said. “We’ve had a small collaboration with BMS for about a year on a single genetic target where we’ve been able to work with them. The teams have got to know each other, they understand our science, they understand what our capabilities are and how potentially transformative our technology is.”
Francisco Ramírez-Valle, senior vice president and head of the Immunology & Cardiovascular Thematic Research Center at Bristol Myers Squibb, said BMS leverages technologies like the AOC platform to identify “meaningful targets and develop new medicines.”
“This collaboration with Avidity represents an important part of our continued investment in innovative therapeutic approaches that have the potential to provide transformative outcomes to patients living with serious cardiovascular conditions,” he said.
Under the terms of the collaboration agreement, Avidity will receive $100 million upfront – a $60 million cash payment as well as the purchase of approximately $40 million of Avidity (NASDAQ: RNA) common stock at a purchase price of $7.88 per share.
Avidity shares were trading at around $6 prior to the deal announcement on Nov. 28, spiking up more than 30% to a high of $8.35 on Nov. 29 before settling to just above $7.80.
In addition to the upfront cash and stock payment, Avidity is also eligible to receive up to approximately $1.35 billion in research and development milestone payments, up to approximately $825 million in commercial milestone payments, and tiered royalties up to low double-digits on net sales. BMS will also fund all future clinical development, regulatory and commercialization activities coming from the collaboration.
Outside of the targets in the BMS collaboration, Avidity continues to advance its internal research and development of its pipeline of drugs in rare muscles diseases – three of which are in clinic.
Boyce said the promise of precision RNA therapies makes “this field incredibly exciting, and we’re at the forefront of it,” adding that the AOC platform technology “was developed entirely in-house in our labs in San Diego, so this is true, top-to-bottom San Diego innovation.”
CEO: Sarah Boyce
Headquarters: Torrey Pines
Business: RNA therapeutics
Stock: RNA (NASDAQ)
Revenue: $9.2 (FY2022)
Notable: Avidity’s AOC platform is the first to successfully deliver RNA therapies to muscle cells.