相关临床试验
31
9 进行中
药物批准
32
批准总数
监管机构
6
监管机构数
成立时间
N/A
进行中（未招募）
7
22.6%
已完成
12
38.7%
尚未招募
2
6.5%
招募中
7
22.6%
终止
1
3.2%
Unknown
2
6.5%
- THX Pharma, Biocodex, and the Beyond Batten Disease Foundation jointly serve as Platinum Sponsors of the 2026 BDSRA Annual Family Conference, marking International Batten Disease Awareness Day on June 9. - The partners will deliver an update on Batten-1, an oral miglustat-based investigational therapy, and the planned initiation of an international pivotal Phase 3 study later in 2026. - Phase 1/2 results demonstrated a favorable safety profile, slowed motor symptom progression, reduced neurofilament light chain levels, and real-world data suggesting clinical benefit on visual acuity. - Biocodex is funding the Phase 3 study under a global licensing agreement, with the ambition of making Batten-1 available to patients by 2029 pending regulatory submissions.
- MRM Health's lead Live Biotherapeutic Product MH002 has been granted Fast Track designation by the FDA for treating mild-to-moderate ulcerative colitis, recognizing its potential to address significant unmet medical needs. - The designation was supported by Phase 2a trial results showing excellent safety and encouraging efficacy signals over eight weeks, with no adverse reactions observed and evidence of mucosal healing and clinical remission. - MH002 is composed of six well-characterized commensal bacterial strains designed to restore microbiome balance and represents the most advanced Live Biotherapeutic Product targeting IBD-specific mechanisms. - The company plans to advance MH002 into a Phase 2b study (STARFISH-UC) enrolling approximately 204 patients, with results expected in Q4 2027.
- THX Pharma and Biocodex have signed a strategic licensing agreement worth up to €173 million to develop treatments for three rare neurological diseases: Batten disease, Gaucher disease, and Niemann-Pick disease type C. - Biocodex acquired exclusive global rights to Batten-1, a drug candidate preparing for Phase 3 trials in 2026 for juvenile Batten disease, which currently has no approved treatments. - The deal includes a €12 million upfront payment, up to €161 million in milestone payments, and tiered double-digit royalties, with THX Pharma leading clinical development while Biocodex handles commercialization. - TX01, based on an adapted oral formulation of an already approved compound, received regional licensing for the US and Canada to treat Gaucher disease and Niemann-Pick disease type C.
- MRM Health secured FDA IND clearance to initiate the STARFISH-UC Phase 2b trial of MH002, a rationally-designed live microbial consortium for mild-to-moderate ulcerative colitis treatment. - The randomized, placebo-controlled study will enroll approximately 204 patients inadequately controlled by standard care, featuring a 12-week induction phase followed by a 40-week extension. - Previous Phase 2a trials demonstrated excellent safety and encouraging efficacy signals, including mucosal healing, anti-inflammatory effects, and clinical remission induction. - MH002 represents the most advanced Live Biotherapeutic Product based on disease-specific bacterial consortia, engineered using MRM Health's proprietary CORAL platform.
- MRM Health NV raised €55 million in Series B funding led by Biocodex to advance its microbiome-based therapeutic pipeline, including Phase 2b trials for lead program MH002 in ulcerative colitis. - The company's lead product MH002 represents the most advanced rationally-designed live microbial consortium therapy for inflammatory bowel diseases, showing positive Phase 2a safety and efficacy data. - Strategic partnership with Biocodex will provide additional non-dilutive funding and manufacturing capabilities, while the company advances two additional programs to IND approval targeting inflammation and immune-oncology. - The funding validates MRM Health's proprietary CORAL® platform technology for developing scalable, standardized microbiome therapeutics for chronic inflammatory diseases with high unmet medical need.
• A new report offers an overview of the clinical trial landscape for Dravet Syndrome, also known as Severe Myoclonic Epilepsy of Infancy, providing key data and analysis. • The review includes data on trial numbers, average enrollment, and top countries involved, segmented by region, phase, status, endpoints, and sponsor type. • Key companies like Jazz Pharmaceuticals, UCB, Takeda, and others are profiled, with details on their ongoing trials and prominent drugs in development for Dravet Syndrome. • The report identifies trends in clinical trial enrollment over the past five years and highlights recent news related to Dravet Syndrome research and development.
