American multinational biotechnology company based in Cambridge, Massachusetts, specializing in neurological disease treatments; primary products include Tecfidera, AVONEX, and Tysabri for multiple sclerosis, spinal muscular atrophy, and Alzheimer's disease.
Clinical Trials
535
25 active
Approvals
19
Total approvals
Agencies
1
Regulatory bodies
Founded
1978
Active, not recruiting
20
3.7%
Approved For Marketing
1
0.2%
Completed
374
69.9%
Enrolling By Invitation
3
0.6%
No Longer Available
2
0.4%
Not yet recruiting
2
0.4%
Recruiting
20
3.7%
Terminated
90
16.8%
Withdrawn
23
4.3%
- Eisai and Biogen announced Japan's approval of LEQEMBI Pen, a subcutaneous autoinjector formulation of lecanemab, as a new route of administration for early Alzheimer's disease. - The approval makes Japan the third country globally to clear the subcutaneous formulation, following the United States in July 2026 and China in September 2026. - Two pens totaling 500 mg are given once weekly, with each injection taking about 15 seconds and demonstrating exposure similar to biweekly intravenous dosing. - Systemic injection-related reactions occurred less frequently with subcutaneous administration at 1.4% compared with intravenous administration, with ARIA monitoring by MRI unchanged.
- Novartis discontinued development of VHB937 (lifonebart) after the Phase 2 ASTRALS trial in early-stage ALS missed both primary and secondary endpoints. - The 251-patient trial tested the TREM2-stabilizing monoclonal antibody or placebo for 40 weeks, with no numerical data released by the company. - The setback adds to a difficult pipeline stretch for Novartis, following Phase 3 misses for pelacarsen and del-desiran and halted rap-cel studies. - Detailed ASTRALS findings are scheduled for presentation at the 37th International Symposium on ALS/MND in Amsterdam on December 9-11, 2026.
- Axoltis' 82-patient Phase 2 SEALS trial of intravenous NX210c in ALS missed its primary endpoint, with no significant change in neurofilament light chain or Qalb at week six. - Post-hoc analyses showed monthly ALSFRS-R decline of 0.77 points at 5 mg/kg and 1.06 at 10 mg/kg versus 1.57 for placebo at week 10. - The 51% reduction in functional decline with the lower dose reached statistical significance, but the lack of dose-response and small size limit interpretation.
- Lipocine has initiated BLOOM, a second Phase 3 study of oral brexanolone LPCN 1154 in severe postpartum depression, with first participant dosing expected in early Q4 2026. - The prior 90-patient Phase 3 trial missed its primary HAM-D17 endpoint at hour 60, but post hoc analyses flagged one high-enrolling site where about 40% of treated participants had no detectable drug. - BLOOM will enroll approximately 120 participants using the same 48-hour dosing regimen and HAM-D17 endpoint, adding third-party site selection, centralized rating and prespecified sensitivity analyses. - Lipocine reported $23.3 million in unrestricted cash as of June 30, 2026, sufficient to run BLOOM at roughly $1 million monthly burn but not to reach approval or commercialization.
- The FDA approved Isembyld (apitegromab-mstn) for SMA patients aged two and older already receiving an SMN2-targeted therapy, marking Scholar Rock's first product approval. - Isembyld is the first and only muscle-targeted SMA therapy, blocking myostatin activation to increase muscle mass and strength rather than targeting motor neurons. - In the Phase 3 SAPPHIRE trial, Isembyld produced a statistically significant 2.2-point HFMSE motor function improvement over placebo after 52 weeks of treatment. - The approval follows a prior complete response letter tied to manufacturing compliance issues, and Scholar Rock plans European and Japanese filings plus development in FSHD and obesity-related muscle loss.
- Alteogen signed an exclusive option and license agreement with Novartis for its hyaluronidase ALT-B4, with maximum payments reaching $3.223 billion if all options and milestones are achieved. - Novartis secured multiple options to develop and commercialize subcutaneous formulations of biopharmaceuticals using ALT-B4, choosing it over Halozyme's Enhanze platform. - ALT-B4's patent protection extends into the early 2040s, while Halozyme's core composition-of-matter patents expire between 2027 and 2029, reinforcing Alteogen's long-term exclusivity position.
- Teitur Trophics reported positive Phase I results for TT-P34, a first-in-class neuroprotective peptide tested in 55 healthy volunteers and 12 patients with early-stage Parkinson's disease. - Once-weekly subcutaneous dosing was well tolerated at all levels tested with no dose-limiting findings, and pharmacokinetics confirmed dose-dependent exposure in the brain and central nervous system. - Early biomarker signals were consistent with lysosomal pathway engagement, supporting a planned Phase II trial in Parkinson's disease patients in 2027. - Teitur is now raising a Series B round to fund the Phase II study, which will explore TT-P34's therapeutic benefit and disease-modifying potential.
- Encoded Therapeutics closed a $275 million Series F financing co-led by GV and an undisclosed healthcare fund, with participation from ARCH Venture Partners, SoftBank Vision Fund 2 and others. - Proceeds will fund a pivotal trial of ETX101 in infants and young children with SCN1A-positive Dravet syndrome plus an expansion study up to age 18. - Interim Phase 1/2 POLARIS data presented at the European Epilepsy Congress showed up to 79% reductions in monthly seizures at the third dose level after one year. - Funding also supports internal GMP manufacturing scale-up and advancement of ETX301 toward a 2027 IND submission for post-amputation neuroma pain.
- Biogen has extended its felzartamab program with an open-label long-term extension study in kidney transplant patients who developed antibody-mediated rejection or microvascular inflammation in the TRANSCEND or TRANSPIRE trials. - Felzartamab is administered as a slow intravenous infusion every eight weeks and is designed to calm immune attacks on transplanted kidneys, with safety as the study's primary goal. - Biogen executives describe antibody-mediated rejection as the foundational opportunity for felzartamab, noting there are no approved therapies and roughly 11,000 U.S. patients experience secondary kidney transplant rejection. - The company also plans to evaluate felzartamab in IgA nephropathy and membranous nephropathy, supported by nephrology infrastructure gained through its Apellis acquisition.
- Biogen reported Q2 2026 adjusted earnings of $3.60 per share on revenue of $2.74 billion, surpassing analyst estimates of $2.95 and $2.46 billion respectively. - The company raised its full-year 2026 revenue guidance to mid-single-digit growth from a prior forecast of mid-single-digit decline, citing rare disease portfolio strength. - Global sales of Alzheimer's drug Leqembi rose 15% year-over-year to approximately $184 million, with a new subcutaneous formulation expected to boost uptake. - Biogen cut its 2026 adjusted EPS guidance to $12–$13 from $14.25–$15.25, reflecting a $3.85 per share impact from the $5.6 billion Apellis Pharmaceuticals acquisition.
