--Encouraging progression-free survival data in overall population; continued follow-up needed to allow data to mature further, especially in low ctDNA subgroup where events accrue more slowly --21% relative risk reduction of progression or death with GRANITE vs. control in all treated population (HR=0.79 [95% CI, 0.42-1.50])-- -- 38% relative risk reduction of progression or death with GRANITE vs. control in low ctDNA subgroup (HR=0.62 [95% CI, 0.23-1.70]) -- -- Strength of neoantigen specific

Istaroxime treatment significantly improved systolic blood pressure as well as cardiac output and renal function without increasing heart rate or clinically significant arrythmias NYHA heart failure classification was improved up to 72 hours with a serious adverse event profile generally similar to placebo Sept. 30, 2024 -- Windtree Therapeutics, Inc. (“Windtree” or the “Company”) (NasdaqCM: WINT), a biotechnology company focused on advancing early and late-stage innovative therapies for criti

-- Achieves primary endpoint showing a linear relationship between adherence to PoNS Therapy® in Phase 2 and improvement in DGI scores from Phase 1 to end of treatment at week 14 -- -- Confirms the therapeutic benefits of PoNS Therapy for gait deficit improvement in people with MS -- Sept. 30, 2024 -- Helius Medical Technologies, Inc. (Nasdaq:HSDT) (“Helius” or the “Company”), a neurotech company focused on delivering a novel therapeutic neuromodulation approach for balance and gait deficits, t

2024年9月30日—北海康成制药有限公司（以下简称“北海康成”，股票代码“1228.HK”）， 是一家在中国领先并专注罕见疾病领域的全球化的生物制药公司，致力于创新疗法的研究、开发和商业化。公司今日宣布，治疗戈谢病1类新药注射用CAN103（维拉苷酶β）获得国家药监局药品审批中心（CDE）优先审评资格。 CAN103是北海康成与药明生物（2269.HK）在罕见病领域的首个合作项目，是中国首个针对戈谢病开发的酶替代疗法（ERT）。CAN103旨在长期治疗I型和III型戈谢病的成人和青少年患者。据弗若斯特沙利文统计，2020年中国约有3,000名戈谢病患者。 我们很高兴CDE授予CAN103优先审查的资格，这是对中国戈谢病患者迫切的医疗需求和我们临床试验结果的认可。CAN103具有成为变革性疗法的潜力，并对罕见病生态圈做出巨大贡献。在首个重组酶替代疗法（ERT）获批30多年后，中国和全球许多患者仍然无法获得现有的治疗方案，他们的需求一直未得到满足。我们在全力以赴加速CAN103的审批流程，使其成为首个在中国本土研发和生产的酶替代疗法。 CAN103（维拉苷酶β）是正在开发的用于治疗I

28-week data show ESK-001 was generally well tolerated and most patients treated with the top dose of 40 mg twice daily achieved PASI 75 Three additional data presentations further support ESK-001’s potential to offer a highly differentiated and best-in-class treatment profile for people with moderate-to-severe plaque psoriasis Full 52-week Phase 2 OLE dataset expected 1H 2025; Phase 3 clinical program ongoing Sept. 27, 2024 -- Alumis Inc. (Nasdaq: ALMS), a clinical stage biopharmaceutical comp

Updated Phase 1 data presented at ESH-iCMLf 26th Annual John Goldman Conference Reported cumulative MMR rate of 44% (8/18) by 24 weeks, with stable or deepening responses between weeks 12 and 24, which continues to compare favorably to precedent Phase 1 trials of approved BCR::ABL1 TKIs ELVN-001 remains well-tolerated with no dose reductions reported with 39 patients enrolled and a median treatment duration of 20 weeks at cutoff Sept. 28, 2024 -- Enliven Therapeutics, Inc. (Enliven or the Compa

Landmark CARTITUDE-4 study results show CARVYKTI® reduced the risk of death by 45 percent after three-year follow-up Late-breaking data featured in an oral presentation at the 21st International Myeloma Society Annual Meeting Sept. 27, 2024 -- Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, today announced late-breaking three-year follow-up data from the Phase 3 CARTITUDE-4 study that shows a single infusion of CARVYKTI® (ciltacabtagene autoleucel) s

New patent expected to provide market exclusivity into 2036 Zembrace® SymTouch® (sumatriptan succinate injection) 10mg is indicated for the acute treatment of migraine in adults Sept. 27, 2024 -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that the United States Patent and Trademark Office issued U.S. Patent No. 12,097,183 to the Company on

近日，四川百利天恒药业股份有限公司（以下简称“百利天恒”）宣布其合作伙伴百时美施贵宝（以下简称“BMS”，纽交所代码：BMY）收到美国食品药品监督管理局（以下简称“FDA”）的通知，合作的BL-B01D1 联合用药项目用于治疗晚期实体瘤的 1/2a 期临床试验申请已获得 FDA 许可。这一里程碑标志着百利天恒与BMS合作的BL-B01D1项目正在加速全球临床开发，并逐步推进至国际市场。 BL-B01D1是全球首创且唯一进入临床阶段的靶向EGFR×HER3双抗ADC。自2023年12月百利天恒与BMS签署独家许可协议以来，双方紧密合作，积极推动该产品在全球范围内的临床试验和商业化进程。此次FDA许可的获得，将进一步推动BL-B01D1在多个实体瘤适应症中的国际化临床研究。 目前，BL-B01D1正在中国和美国进行超过20项用于多种肿瘤类型的临床试验。其中包括评估BL-B01D1单药用于多种实体瘤的III期试验，以及与PD-(L)1疗法或TKI疗法联合用药用于一线实体瘤治疗的II期临床研究，覆盖肺癌、乳腺癌、头颈癌、食管癌、尿路上皮癌等多种高发癌症类型。此次新获得的FDA许可联合用药研

Enliven Therapeutics在欧洲血液学会国际慢性粒细胞白血病基金会（ESH-iCMLf）第 26 届年度 John Goldman 会议上宣布了第 1 阶段临床试验的最新积极数据，该试验评估了 ELVN-001 对已失败或患者对已知可用于治疗其 CML 的现有疗法（NCT05304377）不耐受或不适合治疗的慢性粒细胞白血病 (CML) 患者的作用。 CML一种影响白细胞的癌症，通常由于费城染色体的染色体异常引起。由于疾病发展缓慢，可能在数年内无明显症状，但可以通过血液检查诊断。治疗通常包括使用靶向药物如酪氨酸激酶抑制剂。 ELVN-001 是一种强效、高选择性、可能成为同类最佳的小分子激酶抑制剂，旨在专门针对 BCR-ABL 基因融合，即 CML 患者的致癌驱动因素。 ELVN-001 的 1 期临床试验是一项剂量递增和扩展试验，旨在评估安全性和耐受性，并确定进一步临床评估 ELVN-001 在复发、难治或不耐受 TKI 的伴有和不伴有 T315I 突变的 CML 患者中的推荐剂量。该试验的主要终点是安全性。次要终点包括药代动力学、中央定量反转录聚合酶链反应测定的M