Biogen's Autoimmune Disease Candidate Gets FDA's Breakthrough Status
- Class: Humanized IgG1 mAb
- Target: BDCA2
- Indication: Cutaneous Lupus (CLE)
- Status: FDA Breakthrough
- Phase III: AMETHYST (2027 Readout)
- Stock (6mo): +36.7%
Biogen BIIB announced that the FDA has granted the Breakthrough Therapy designation to its investigational, first-in-class, humanized IgG1 mAb targeting BDCA2, litifilimab (BIIB059), to treat patients with cutaneous lupus erythematosus (CLE).
Mechanism and FDA Designation
Litifilimab (BIIB059) is a humanized anti-BDCA2 IgG1 monoclonal antibody administered subcutaneously. Litifilimab can inhibit the production of type I interferons, cytokines and chemokines. It can be used in the research of diseases such as systemic lupus erythematosus.
The FDA’s Breakthrough Therapy designation is a process that speeds up the development and review of drugs for serious or life-threatening conditions. This designation is granted when early clinical evidence suggests the drug may significantly improve over existing treatments on one or more important clinical measures. Drugs with this designation receive more intensive guidance and organizational support from senior FDA managers.
Clinical Evidence: Phase II LILAC Study
The FDA’s decision was supported by the full body of evidence for Biogen’s litifilimab, including results from the phase II LILAC study. The study demonstrated that litifilimab significantly reduced skin disease activity in patients with CLE compared to placebo.
Current standard-of-care CLE treatments, such as topical steroids, antimalarials and immunosuppressants, primarily manage symptoms but do not slow or modify disease progression. In the past six months, BIIB stock has gained 36.7% compared with the industry’s 23% growth.
Disease Context: Cutaneous Lupus Erythematosus (CLE)
CLE is a chronic autoimmune skin disease that can occur with or without systemic lupus, causing rashes, pain, itching, photosensitivity, and progressive skin damage that may result in permanent scarring, hair loss, and discoloration.
Lupus affects mostly women, often beginning between the ages of 15 and 40, and disproportionately impacts several ethno-racial groups. There is currently no cure.
Phase III AMETHYST and Future Outlook
Presently, Biogen is evaluating the efficacy and safety of litifilimab in the phase III AMETHYST study for CLE, with a data readout expected in 2027. The company is simultaneously evaluating the candidate in two separate late-stage studies for the potential treatment of systemic lupus erythematosus (SLE).
Biogen’s Other Immunology Pipeline Programs
Biogen’s immunology pipeline comprises two other late-stage candidates being developed across various indications:
- Dapirolizumab pegol: An anti-CD40L antibody currently undergoing phase III development for active SLE.
- Felzartamab: An anti-CD38 antibody undergoing phase III evaluation for antibody-mediated rejection, immunoglobulin A nephropathy, and primary membranous nephropathy. It is also in an early-stage study for lupus nephritis.
Last year, Biogen inked a research deal with Dayra Therapeutics to strengthen its immunology pipeline by adding a new class of oral therapies that could deliver biologic-like benefits by tapping Dayra’s macrocyclic peptide platform.
