- Agios Pharmaceuticals will pay Alnylam Pharmaceuticals $17.5 million to gain the rights to an experimental blood disease therapy and help further a corporate makeover begun in 2020.
- The agreement includes royalties for Alnylam if the treatment reaches the market, as well as $130 million in potential payments for meeting certain development and commercial milestones, Agios said Thursday. Agios is taking over total responsibility for the program, although Alnylam will continue to help with manufacturing through Phase 1 research.
- So far, the experimental therapy has only been tested in animals. Agios CEO Brian Goff says his company aims to conduct research this year that will allow it to seek Food and Drug Administration permission to begin human testing.
Agios’s story has been one of evolution over the last four years. Built as an oncology and rare disease company, Agios was led for 10 years by former Genentech and Millennium Pharmaceuticals executive David Schenkein, who oversaw the approval of two cancer medicines before stepping down in 2019.
The new CEO, Celgene veteran Jackie Fouse, steered the company in a different direction by shedding cancer assets and turning Agios’ focus to uncommon blood disorders. In December 2020, Agios announced it would sell an approved leukemia medicine and three experimental cancer therapies to Servier for $1.8 billion. Agios had previously licensed another approved cancer drug to Celgene.
In February 2022, Fouse scored her first major win after the strategic shift, with the approval of Pyrukynd for a rare form of anemia. The company then decided in May 2022 to move away from exploratory research, cutting about 50 jobs to focus on more advanced drug candidates. Three months later, Fouse stepped down as CEO and Goff took over the head job.
The deal with Alnylam gives Agios another preclinical asset to round out its pipeline, which includes a drug called AG-946 in early human testing. The company is also working to expand the use of Pyrukynd, which brought in just $6.7 million in revenue in the second quarter.
The therapy licensed from Alnylam is designed to treat polycythemia vera, a potentially deadly condition that causes an overproduction of red blood cells and affects about 100,000 people in the U.S. Unlike other Agios drugs, the treatment uses Alnylam’s RNA interference technology. The technology has drawn attention from several large drugmakers over the years, including most recently Roche, which signed a $310 million deal with Alnylam in July.