End of Cooperation Again! Biogen and Karyopharm Halt ALS Drug Development

A few days ago, Biogen announced that it would terminate its amyotrophic lateral sclerosis asset purchase agreement with Karyopharm Therapeutics, and the two companies will stop cooperating to develop KPT-350 (BIIB100). Currently, the therapy is undergoing Phase 1 testing, primarily for the treatment of amyotrophic lateral sclerosis (ALS).

As early as four years ago, Biogen paid Karyopharm up to $10 million upfront, and at the same time obtained the rights to KPT-350, an oral selective nuclear export inhibitor, and related assets, Biogen also Expressed its support for Karyopharm's further development of amyotrophic lateral sclerosis therapeutics. If, as part of the collaboration, the therapy reaches a milestone endpoint, Karyopharm will be eligible to receive up to $270 million in additional revenue.

However, cancer treatment developer Karyopharm wrote in a recent filing with the U.S. Securities and Exchange Commission (SEC) that the partnership deal signed by the two companies on January 24, 2018 will no longer be valid, and Karyopharm has no future rights Receive any royalties or milestone payments associated with this partnership. The filing did not detail why the companies chose to end their partnership.

As of now, Biogen has not commented on the cancelled deal with Karyopharm. Amyotrophic lateral sclerosis has a market value of up to $3 billion, there is still a huge unmet need, and there is currently no cure or treatment for the disease. The FDA has previously approved some therapies for this indication, such as edaravone and riluzole, but these drugs have only modest ability to slow disease progression.

Coincidentally, Biogen has also encountered setbacks in the development of other amyotrophic lateral sclerosis treatments. In March of this year, Biogen and its partner Ionis Pharmaceuticals decided to discontinue research and development after a phase BIIB0781 study in people with a specific type of amyotrophic lateral sclerosis related to the C9orf72 gene failed to meet clinical endpoints. (For details, please click "The R&D of New ALS Drugs Frustrated: Bojian/Ionis Terminates Phase I Trials, and FDA Questions the Listing of Amylyx's Drugs")

Biogen is currently collaborating with Ionis on a Phase III study of tofersen, an investigational antisense drug targeting superoxide dismutase 1 (SOD1) in patients with amyotrophic lateral sclerosis. Although the drug had previously failed to meet its primary endpoint, the latest data from the two companies' Phase III VALOR trial, which lasted 12 months earlier this month, showed the drug's potential to slow declines in muscle stress, clinical and respiratory function. . In addition, early survival data also suggest that if the drug is given early for treatment, it may reduce the risk of permanent ventilation or death.