Global Briefs: Biogen, Regeneron & FDA 

A roundup of news from Biogen, Regeneron and the US Food and Drug Administration. Highlights below.  

* Biogen Terminates Alzheimer’s Drug Aduhelm; Reprioritizes Strategy 
* Regeneron Launches New Cell-Therapy Unit 
* FDA Issues Final Guidance To Facilitate Generic-Drug Reviews   

Biogen Terminates Alzheimer’s Drug Aduhelm; Reprioritizes Strategy 
Biogen has announced plans to reprioritize its resources in Alzheimer’s disease (AD), a strategic therapeutic area expected to drive near and long-term growth. The company will continue to advance Leqembi (lecanemab-irmb), an anti-amyloid beta treatment developed with Eisai, which gained traditional approval by the Food and Drug Administration in July (July 2023). It will accelerate development of potential new treatment modalities, including its ASO-targeting tau (BIIB080) and an oral small-molecule inhibitor of tau aggregation (BIIB113). The company will discontinue the development and commercialization of Aduhelm (aducanumab-avwa) 100 mg/mL injection for intravenous use and will terminate a post-marketing confirmatory clinical study for the drug. The company says that the decision is not related to any safety or efficacy concerns. A large portion of the resources released resulting from termination of the Aduhelm program will be redeployed in Biogen’s AD franchise. 

Aduhelm, once touted as a potential blockbuster drug, was approved under the FDA’s accelerated approval pathway in June 2021. The accelerated approval pathway allows for earlier approval of drugs that treat serious conditions and that fill an unmet medical need but requires post-approval confirmatory trials. The US Centers for Medicare and Medicaid Services  issued a national policy for coverage of Aduhelm and any future monoclonal antibodies directed against amyloid for treating Alzheimer’s disease that are approved under FDA’s accelerated approval pathway only to patients taking part in approved clinical trials, which includes post-approval confirmatory trials. If the post-approval trials confirm clinical benefit, then the drug may receive traditional approval. The narrow Medicare reimbursement policy for Aduhelm and other factors contributed to weak sales.   

In January 2023, Biogen began a strategic review of its research and development efforts, including seeking potential partners or external financing for Aduhelm as part of a focus on prioritizing the company’s portfolio. During this process, Biogen considered the time and investment required for the post-marketing confirmatory study and the likely advancements in the field by the time of potential FDA traditional approval of the drug. Despite an extensive process, the company said it did not identify potential strategic partners or external financing. 

Biogen has recorded a one-time charge of approximately $60 million related to close-out costs for the program in the fourth quarter of 2023. Biogen licensed aducanumab from Neurimmune, a Swiss-based bio/pharmaceutical company and has terminated that license. The rights to aducanumab will revert to Neurimmune. 

Source: Biogen 

Regeneron Launches New Cell-Therapy Unit 
Regeneron Pharmaceuticals has announced the formation of Regeneron Cell Medicines, a newly formed research & development (R&D) unit to advance cell therapies and combination approaches in oncology and immunology.  

Regeneron is launching the new unit following an agreement with 2seventy bio, a Cambridge, Massachusetts-based bio/pharmaceutical company, to acquire full development and commercialization rights to 2seventy bio’s pipeline of investigational immune cell therapies, along with its discovery and clinical manufacturing capabilities. The pending acquisition does not include Abecma (idecabtagene vicleucel), a CAR T therapy approved for treating multiple myeloma, for which 2seventy bio is partnered with Bristol-Myers Squibb.  

In addition, Regeneron has agreed to sublease a portion of the office, lab and manufacturing space of 2seventy bio’s in Cambridge, Mass. and sublease the company’s entire facility in Seattle, Washington. As part of this agreement, approximately 160 2seventy bio employees will transition to Regeneron, including chief scientific officer, Philip Gregory and chief medical officer, Steve Bernstein. 

In 2018, Regeneron and bluebird bio (which spun out 2seventy bio in 2021) entered an agreement to discover cell therapies for treating cancer. Under the original agreement, Regeneron had the right to opt-in to a co-development/co-commercialization arrangement for collaboration targets. Under the new agreement, Regeneron will acquire full development and commercialization rights of 2seventy bio’s preclinical and clinical stage pipeline and will assume ongoing program, infrastructure and personnel costs related to these programs. There will be an upfront payment of $5 million and a single milestone payment from Regeneron to 2seventy bio for the first major market approval of the first approved product. Regeneron will pay 2seventy bio a low single-digit percent royalty on revenues generated by the products.  

The transaction is expected to close in the first half of 2024, subject to certain closing conditions.  

Source: Regeneron 

FDA Issues Final Guidance To Facilitate Generic-Drug Reviews   
The US Food and Drug Administration (FDA) has issued two final guidances for the generic drug application and review process.  

The new guidance, ANDA Submissions—Amendments and Requests for Final  Approval to Tentatively Approved ANDAs, is intended to assist applicants in preparing and submitting amendments to tentatively approved abbreviated new drug applications (ANDAs), including requests for final  approval. The guidance provides recommendations on the timing and content of amendments to tentatively approved ANDAs to facilitate submission in a timely fashion to enable final approval  on the earliest date on which the ANDA may lawfully be approved based on patent and/or exclusivity protections.  

The second guidance, Revising ANDA Labeling Following Revision of the RLD Labeling,  is intended to assist applicants and holders of ANDAs in updating their labeling following revisions to the approved labeling of a reference listed drug. The guidance provides recommendations on identifying RLD labeling updates and submitting ANDA amendments or supplements to update generic drug labeling.  The guidance finalizes the draft guidance of the same title issued on January 27, 2022.The final guidance provides clear expectations for when updates to labeling are required, the process for updating labeling, and the types of submissions for labeling updates.  

Source: The US Food and Drug Administration (ANDA submissions) and The US Food and Drug Administration (ANDA labeling)