Recent advances in understanding venous malformation disease pathogenesis highlight the PI3K/AKT/mTOR pathway as a key driver of disease proliferation, spurring real-world off-label use of systemic rapamycin (sirolimus) Publication includes a systematic review of 26 studies evaluating the use of rapamycin for the treatment of venous malformations Study authors highlight unmet clinical need for venous malformations and lack of FDA-approved pharmacologic options while advocating for an FDA-approved targeted t ...

NEW HAVEN, Conn., Sept. 17, 2025 (GLOBE NEWSWIRE) -- Invivyd, Inc. (Nasdaq: IVVD) today announced the appointment of Kristie Kuhl as Chief Communications Officer. Ms. Kuhl will lead the Company’s communications and patient advocacy efforts as Invivyd advances its mission of providing monoclonal antibodies for the prevention and treatment of viral disease. Ms. Kuhl brings deep expertise in healthcare and pharmaceutical communications, and she has been recognized by industry media for her influence in health ...

Featured presentations showcase the positive Phase 1 healthy volunteer trial results supporting KT-621’s oral, dupilumab-like profile KT-621 BroADen Phase 1b trial in moderate to severe atopic dermatitis (AD) patients on track to report data in 4Q25 KT-621 Phase 2b trials in AD and asthma on track to initiate in 4Q25 and 1Q26, respectively WATERTOWN, Mass., Sept. 17, 2025 (GLOBE NEWSWIRE) -- Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing a new class of oral sm ...

Data support advancement of GS-6791 into clinical testing in patients with inflammatory diseases; first-in-human Phase 1 trial in healthy volunteers is ongoing Data are being presented at the European Academy of Dermatology and Venereology (EADV) Congress SAN FRANCISCO, Sept. 17, 2025 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX) today announced the presentation of preclinical data from GS-6791/NX-0479, a novel IRAK4 protein degrader discovered as part of the company’s ongoing research collabo ...

Denifanstat met all primary and secondary endpoints versus placebo Denifanstat was generally well tolerated Sagimet initiated first-in-human Phase 1 clinical trial of a second FASN inhibitor, TVB-3567, for development in acne SAN MATEO, Calif., Sept. 17, 2025 (GLOBE NEWSWIRE) -- Sagimet Biosciences Inc. (Nasdaq: SGMT), a clinical-stage biopharmaceutical company developing novel therapeutics targeting dysfunctional metabolic and fibrotic pathways, today reported that data from a Phase 3 clinical trial for th ...

Six poster presentations showcase clinical and real-world data in major depressive disorder, Alzheimer’s disease agitation, narcolepsy, and obstructive sleep apneaNEW YORK, Sept. 17, 2025 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) disorders, today announced six presentations across its broad portfolio of innovative neuroscience products and product candidates at Psych Congress 2025, being held S ...

Once-daily oral brepocitinib 30 mg demonstrated clinically meaningful and statistically significant improvement compared to placebo on the primary endpoint and all nine key secondary endpoints, including measurements of skin disease, muscle disease, steroid-sparing effect, and rapidity of onsetOn the primary endpoint, brepocitinib 30 mg achieved a week 52 mean Total Improvement Score (TIS) of 46.5 compared to 31.2 for placebo (p=0.0006​), even with nearly twice as many patients coming off background steroid ...

Pilavapadin 10 mg resulted in a two-point reduction from baseline in average daily pain scores (ADPS) by week 11 and was well tolerated in PROGRESS Phase 2b studyTHE WOODLANDS, Texas, Sept. 17, 2025 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced the presentation of clinical data from the company’s Phase 2b PROGRESS study for its investigational, AAK1-inhibitor, pilavapadin, for the treatment of diabetic peripheral neuropathic pain (DPNP) at two medical meetings: “New Promis ...

核心观点 - Arcutis Biotherapeutics在2025年欧洲皮肤病与性病学会(EADV)年会上公布ZORYVE(roflumilast)针对三种常见皮肤病的多项临床数据 证明其在脂溢性皮炎、特应性皮炎和斑块状银屑病治疗中具有显著疗效和广泛适用性 覆盖不同种族、肤色类型及年龄群体 [1][9][10] 脂溢性皮炎数据(STRATUM试验) - ZORYVE泡沫剂0.3%在8周时总体人群的IGA治疗成功率达79.5% 显著优于对照组的58.0% [5] - 各亚组疗效一致：白种人(80.3% vs 57.2%)、黑种人/非裔美国人(71.5% vs 59.4%)、其他种族(82.0% vs 62.5%)；西班牙裔/拉丁裔(77.5% vs 70.8%)和非西班牙裔/拉丁裔(80.0% vs 55.1%)；FST I-III型皮肤(80.7% vs 55.1%)和IV-VI型皮肤(76.3% vs 66.4%) [5] - 瘙痒改善显著：总体WI-NRS评分显示ZORYVE组疗效优于对照组 各种族亚组改善率分别为白种人(60.1% vs 41.5%)、黑种人/非裔美国人(59.7% vs 48.9%)、其他种族(83.0% vs 23.1%) [5] - 色素异常改善：基线存在色素减退的参与者中57.1%白种人和87.5%黑种人/非裔美国人实现部分或完全缓解；色素沉着过度者中75%白种人和60%黑种人/非裔美国人获得改善 [5] 特应性皮炎数据(INTEGUMENT-PED试验) - ZORYVE乳膏0.05%在2-5岁儿童中显示4周后患者报告结局(PROs)显著改善 包括生活质量提升和家庭负面影响降低 [4][6] - 在SCORAD总分及组件评分(瘙痒、睡眠损失、干燥强度)以及POEM测量的疾病严重程度和影响方面均观察到改善 [6] - 耐受性良好 用药部位无或仅有最小刺激反应 包括首次用药后 [6] 银屑病数据(DERMIS-1/2和ARRECTOR试验) - ZORYVE乳膏0.3%和泡沫剂0.3%在8周时均能改善面部和/或生殖器部位斑块状银屑病的体征和症状 [7] - 面部和/或生殖器受累亚组的改善程度与总体人群相当 乳膏和泡沫制剂疗效一致 [7] - 两种剂型均表现出良好耐受性 [7] 产品地位与认可 - ZORYVE是三大炎症性皮肤病(特应性皮炎、脂溢性皮炎和斑块状银屑病)处方量第一的品牌外用疗法 [10] - 2024年ZORYVE乳膏0.15%获Glamour美容健康奖"湿疹产品奖" [11] - 2025年ZORYVE乳膏0.3%和泡沫剂0.3%获国家银屑病基金会认可章 为首个获此荣誉的FDA批准产品 [11] - ZORYVE获Allure"2025最佳美容突破奖" 为首个同时获批特应性皮炎、斑块状银屑病和脂溢性皮炎的FDA批准药物 [11] 公司背景 - Arcutis Biotherapeutics为商业阶段生物制药公司 专注于免疫皮肤病学领域 [17] - 拥有针对三大炎症性皮肤病的先进靶向外用药物组合 [17] - 通过独特皮肤病学开发平台构建针对多种炎症性皮肤病的高潜力管线 [17]

核心观点 - 赛诺菲旗下brivekimig在治疗中重度化脓性汗腺炎的2a期研究中取得积极结果 显示在主要终点和关键次要终点上均优于安慰剂组 药物耐受性良好且无严重不良事件[1][2][4] 药物临床数据 - 主要终点HiSCR50应答率：brivekimig组达67%（n=48） 安慰剂组为37%（n=23） 估计差异29%（90%可信区间10%-47%） 优效概率99.28%[8] - 次要终点HiSCR75应答率：brivekimig组54% 安慰剂组22% 估计差异29%（90% CI 11%-48% p=0.0171）[8] - 次要终点HiSCR90应答率：brivekimig组31% 安慰剂组9% 估计差异20%（90% CI 5%-34% p=0.0576）[8] - 引流隧道数量平均变化：brivekimig组较基线减少56.0% 安慰剂组增加10.9% 估计差异-67.0%（90% CI -105.2%至-28.8% p=0.005）[8] 药物机制与研发定位 - brivekimib是一种双靶点纳米抗体 同时抑制肿瘤坏死因子（TNF）和OX40配体 针对免疫调节通路[7][9] - 目前正针对多种免疫介导性疾病及炎症性疾病进行开发[7][9] - 代表公司在复杂慢性皮肤疾病炎症机制治疗领域的研发策略[4][7] 研究设计 - HS-OBTAIN研究为随机、双盲、安慰剂对照的2a期概念验证研究（临床试验标识号NCT05849922）[4][10] - 研究对象为生物制剂初治的中重度化脓性汗腺炎成人患者 按2:1随机分组[4][11] - 给药方案：每两周皮下注射150mg brivekimig或安慰剂 持续16周[11] - 主要分析采用贝叶斯逻辑回归模型 并根据Hurley分期进行调整[11] 疾病背景 - 化脓性汗腺炎是一种慢性致残性炎症性皮肤疾病 特征为疼痛性皮肤结节、脓肿和引流隧道[3] - 欧盟约有19.6万成人患者受该疾病影响[3] - 当前治疗选择有限 存在未满足临床需求[4] 公司战略 - 公司致力于通过深度理解免疫系统开发创新药物 管线聚焦免疫介导性疾病领域[12] - 强调通过双靶点抑制策略应对慢性炎症疾病的复杂性和异质性[4][7]