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.
相关临床试验
536
25 进行中
药物批准
19
批准总数
监管机构
1
监管机构数
成立时间
1978
进行中（未招募）
20
3.7%
Approved For Marketing
1
0.2%
已完成
374
69.8%
Enrolling By Invitation
3
0.6%
No Longer Available
2
0.4%
尚未招募
2
0.4%
招募中
21
3.9%
终止
90
16.8%
撤回
23
4.3%
- City Therapeutics filed an S-1 on September 24, 2026 to list on the Nasdaq Global Market under the ticker CTY, with Goldman Sachs, Jefferies, Stifel and Oppenheimer as lead underwriters. - Proceeds are earmarked for CITY-FXI through Phase 1 and a planned Phase 2 total knee arthroplasty study, plus CITY-RBP4 and CITY-TFR2 development. - CITY-FXI is a GalNAc-conjugated siRNA targeting Factor XI that showed dose-dependent FXI knockdown in healthy volunteers, aiming to cut bleeding risk versus current anticoagulants. - The company reported $13.6 million in collaboration revenue for 2025, up from $2.8 million in 2024, and a net loss of $58.5 million.
- Eli Lilly will pay InnoCare Pharma up to $100 million in upfront and near-term payments under a strategic research collaboration and license agreement. - InnoCare is eligible for up to approximately $3.25 billion in development and commercial milestone payments plus single-digit tiered royalties on annual net product sales. - InnoCare will use its proprietary drug discovery platform to discover and advance compounds against up to five undisclosed targets addressing unmet medical needs. - The partners have not disclosed the specific targets or therapeutic areas, and InnoCare's pipeline spans cancer and autoimmune diseases with three approved drugs.
- 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.
- China's National Medical Products Administration has approved a self-administered subcutaneous formulation of LEQEMBI for early Alzheimer's disease, developed by Biogen and partner Eisai. - The new option allows once-weekly at-home dosing via an autoinjector instead of clinic-based infusions, for patients with early-stage Alzheimer's who qualify under the label. - China is only the second country worldwide to clear the at-home subcutaneous version of LEQEMBI, according to Simply Wall St.
- 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.
- BioArctic and Eisai said Leqembi Iqlik, a subcutaneous autoinjector form of lecanemab, is now available in the US for initiation therapy after FDA approval on 13 July. - The starting dose is 500 mg given as two 250 mg injections of about 15 seconds each, replacing the biweekly intravenous infusions previously required in a clinic. - It is the first anti-amyloid treatment cleared for at-home dosing at both treatment initiation and maintenance, with MRI monitoring still required to watch for ARIA.
- 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.
