First patient dosed in the pediatric cohort of the Phase 1/2 trial of OPGx-LCA5; initial data on the cohort anticipated by Q3 2025 New 12-month data on the first three adult OPGx-LCA5 patients to be presented at a major medical conference in Q2 2025 FDA meeting scheduled in March 2025 to discuss Phase 3 trial design and registrational endpoints for OPGx-LCA5 Feb. 18, 2025 -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the tr

Trial decision follows unanticipated events of elevated unconjugated bilirubin levels Company advancing multiple next-generation PTH1R agonists with distinct and unrelated chemical structures relative to SEP-786 Feb. 18, 2025 -- Septerna, Inc. (Nasdaq: SEPN), a clinical-stage biotechnology company pioneering a new era of GPCR drug discovery, today announced its decision to discontinue the Phase 1 single- and multiple-ascending dose (SAD/MAD) clinical trial of SEP-786 in healthy volunteers. SEP-

Data reinforce unique clinical advantages of sotagliflozin Published study is aligned with research presented in December 2024 at the American Society of Hematology showing clinical benefits of sotagliflozin compared to empagliflozin Feb. 18, 2025 -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced The Lancet Diabetes & Endocrinology has published a research paper analyzing the ability of sotagliflozin, a dual SGLT1 and SGLT2 inhibitor, to reduce the risks of life-threatening cardio

Feb. 18, 2025 -- TG Therapeutics, Inc. (NASDAQ: TGTX), today announced the schedule of presentations highlighting data from the ULTIMATE I & II Phase 3 trials and the ENHANCE Phase 3b trial evaluating BRIUMVI® (ublituximab-xiiy) in patients with relapsing forms of multiple sclerosis (RMS) to be presented at the upcoming Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) annual forum, being held February 27 – March 1, 2025, in West Palm Beach, Florida. Abstracts are n

Feb. 18, 2025 -- Bionic Sight, Inc., a company developing a novel gene therapy for retinal degenerative diseases, announced today that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational product BS01 for the treatment of retinitis pigmentosa (RP) patients with advanced stage vision loss. RMAT designation is part of the 21st Century Cures Act, which aims to accelerate the development of regenerative medicine therapies, including gene therapy, for se

Feb. 18, 2025 -- Alvotech (NASDAQ: ALVO), a global biotech company specializing in the development and manufacture of biosimilar medicines for patients worldwide, and Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), today announced that the U.S. Food and Drug Administration (FDA) has accepted for review a Biologics License Application (BLA) for AVT06, Alvotech’s proposed biosimilar to Eylea® (aflibercept), a biologic used to treat eye disorder

Dupixent sBLA accepted for FDA priority review for the targeted treatment of bullous pemphigoid If approved, Dupixent would be the first and only targeted medicine to treat BP in the US; FDA decision expected by June 20, 2025 Priority review granted based on positive pivotal results demonstrating significant improvements in sustained disease remission with Dupixent compared to placebo BP is a chronic, debilitating and relapsing skin disease with underlying type 2 inflammation characterized by in

吉利德科学（Gilead Sciences）公司今天宣布，美国FDA已接受该公司为lenacapavir递交的新药申请（NDA），用于HIV感染的暴露前预防（PrEP）。FDA同时授予这一申请优先审评资格，预计在今年6月19日之前完成审评。新闻稿指出，如果获得批准，lenacapavir将成为首款只需一年两次注射的HIV预防选择。 这一新药申请基于吉利德公司开展的3期临床试验PURPOSE 1和PURPOSE 2的积极数据。在PURPOSE 1中，数据显示，接受每半年一次lenacapavir注射的女性参与者无任何人出现HIV感染，感染风险降低100%，与背景HIV发病率相比表现出显著优势。在PURPOSE 2中，lenacapavir治疗组中99.9%的参与者未感染HIV，与背景HIV感染率相比，感染风险降低96%。 在两项试验中，每半年一次注射的lenacapavir与每日口服Truvada相比，在预防HIV感染方面表现出显著优势，同时总体耐受性良好，无重大或新增安全问题。 全球每年新增约130万艾滋病毒感染病例。近年来研究发现，在未受到感染时服用抗病毒药物，可以降低艾滋病毒

Sarcopenia, a condition characterized by age - related muscle loss and strength decline, is a growing health concern. This article examines the key drugs being developed for sarcopenia, their mechanisms, and the potential they hold for improving the quality of life of patients affected by this condition.Sarcopenia was first defined by Rosenberg in 1989 as “muscle wasting”. In 2018, the European Working Group on Sarcopenia in Older People defined it as a progressive degenerative condition as

Chimerix公司宣布，美国FDA已受理该公司为在研疗法dordaviprone递交的新药申请（NDA），用于治疗复发性H3 K27M突变弥漫性胶质瘤患者。FDA同时授予该申请优先审评资格，预计在2025年8月18日之前完成审评。Dordaviprone是一款潜在“first-in-class”小分子药物，能够选择性靶向线粒体蛋白酶ClpP和多巴胺受体D2。新闻稿指出，dordaviprone有望成为首款针对这一患者群体的获批疗法。 H3 K27M突变弥漫性胶质瘤是一种高度侵袭性的致命脑瘤，由于肿瘤多发于脑室、脑干和脊髓等部位，难于通过手术进行切除。目前有效的治疗方法局限于放疗，尚无针对这一疾病的获批疗法。患者在确诊后中位总生存期只有一年。 Dordaviprone的新药申请得到2期临床试验主要分析结果的支持，此前发布的数据显示，在使用RANO 2.0（一种神经肿瘤学缓解评估标准）进行评估时，dordaviprone在H3 K27M突变弥漫性胶质瘤患者中达到28%的客观缓解率，中位缓解持续时间为10.4个月。 Dordaviprone可通过两种信号通路产生抗癌作用，作为多巴胺受