会议主要讨论的核心内容 4D-150在广泛湿AMD患者群体中的安全性和有效性 - 4D-150在广泛湿AMD患者群体中继续保持安全性和耐受性 [6][9][33] - 4D-150在高剂量组(3E10 vg)中展现出强大的临床活性,包括显著降低患者的抗VEGF注射次数、改善视力和解剖学指标 [12][37][38][39] - 4D-150在低剂量组(1E10 vg)也显示出生物活性,但疗效不及高剂量组 [95] 4D-150的长期疗效和持久性 - 3名1.5-2年前接受4D-150治疗的重度患者至今仍保持无需补充注射的状态,视力和解剖学指标也保持稳定 [42] - 4D-150在广泛湿AMD患者群体中也展现出持久的临床活性,77%的高剂量组患者在24周内无需补充注射 [37][39] 4D-150的安全性概况 - 4D-150总体安全性良好,139名患者中未出现任何4D-150相关的严重不良事件 [33][43] - 在高剂量组(3E10 vg),30名患者中无一例出现前房或玻璃体内显著炎症反应 [33][34][35] - 所有患者均顺利完成局部激素预防性用药方案,无需继续使用激素 [33] 4D-150的III期临床试验计划 - 公司计划进行两项III期非劣效性试验,以视力作为主要疗效指标,评估4D-150 3E10 vg剂量与阿柏西普每8周2mg的对照组 [44][45] - 每项试验计划入组约450名患者,与本次人群扩展队列的特征和给药方案一致 [44] - 公司已获得FDA的RMAT认定和EMA的PRIME认定,有利于加快产品开发 [45][46] 问答环节重要的提问和回答 关于患者特征与注射免疫状态的关系 - 目前尚未发现任何基线特征与注射免疫状态的获得存在相关性,公司将继续研究这一问题 [50] 关于CST变化情况 - 高剂量组77%注射免疫患者的CST保持稳定,无波动,这可能转化为视力获益 [51][52] - 部分需要补充注射的患者,CST会逐渐升高,但仍优于标准治疗 [83][84] 关于低剂量组(1E10 vg)的疗效数据 - 低剂量组的疗效整体劣于高剂量组,但仍显示出一定的生物活性 [95] 关于第4周补充注射的影响 - 错过第4周补充注射的5名患者中,有4名(80%)最终实现长期无需注射 [103] - 第4周补充注射可能有助于覆盖基因表达的上升期,但并非所有患者都需要 [103]

4D Molecular Therapeutics, Inc. (NASDAQ:FDMT) Special Call July 17, 2024 6:30 AM ET Company Participants Uneek Mehra - Chief Financial and Business Officer David Kirn - Chief Executive Officer and Co-Founder Robert Kim - Chief Medical Officer Arshad Khanani - Lead Investigator of PRISM Clinical Trial, Sierra Eye Associates Conference Call Participants Salveen Richter - Goldman Sachs Tazeen Ahmad - BofA Securities Jonathan Miller - Evercore ISI Gena Wang - Barclays Clara Dong - Jefferies Operator With that, ...

Julian Pei Head of Investor Relations and Corporate Finance Investor.Relations@4DMT.com Robust reduction in anti-VEGF injection treatment burden through Week 24 achieved in 30 patients at planned Phase 3 dose (3E10 vg/eye) with 89% reduction in annualized injection rate; 93% of patients received 0 or 1 injection and 77% were injection-free Improvement in mean best corrected visual acuity (BCVA) from baseline through Week 24 achieved at 3E10 vg/eye dose (+4.2 letters); dose response in favor of 3E10 vg/eye d ...

Investors are bullish on the stock due to the recent pipeline progress. Last month, the FDA cleared the company's investigational new drug application or 4D-175, an R100 vector-based intravitreal genetic medicine, for the treatment of patients with geographic atrophy. Earlier, the company presented positive data on its lead candidate, 4D-150, in wet age-related macular degeneration. While earnings and revenue growth expectations are important in evaluating the potential strength in a stock, empirical resear ...

About 4D-175 for Geographic Atrophy Katherine Smith Inizio Evoke Comms Katherine.Smith@inizioevoke.com "GA is a leading cause of irreversible vision loss for over 5 million people globally and while current bolus complement inhibitor treatments reduce the rate of growth in GA lesions, they require burdensome monthly or bimonthly intravitreal injections and do not demonstrate functional vision benefit," said David Kirn, M.D., Cofounder and Chief Executive Officer of 4DMT. "4D-175 has the potential for durabl ...

EMERYVILLE, Calif., June 18, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ:FDMT, 4DMT or the Company))), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced that the Company will present the initial interim 24-week landmark analysis from the Population Extension cohort of the PRISM Phase 2 Clinical Trial evaluating 4D-150 in a broad wet AMD patient population at the ASRS Annual Scientific ...

Injection-free subgroup results demonstrated that a single intravitreal dose of 4D-150 without any supplemental anti-VEGF injections resulted in stable mean visual acuity that was equal to or higher than in the standard bimonthly aflibercept control group at all six timepoints through Week 24 Single intravitreal 3E10 vg/eye dose resulted in sustained reduction and stabilization of mean central subfield thickness (CST) compared to aflibercept at all timepoints EMERYVILLE, Calif., June 08, 2024 (GLOBE NEWSWIR ...

Clinically meaningful improvements in ppFEV1 at 12 months observed in 2 of 3 participants with mild to moderate baseline lung function impairment (ppFEV1 50-80%) and >6 months follow up Aerosolized 4D-710 was well tolerated at doses up to 1E15 vg (n=6) Dose-dependent and widespread 4D-710-mediated CFTR transgene RNA and protein expression observed in all lung biopsies from all participants evaluated to date Pre-existing AAV immunity cross-reactive with A101 did not affect transgene expression, biological ac ...

Clinically meaningful improvements in ppFEV1 at 12 months observed in 2 of 3 participants with mild to moderate baseline lung function impairment (ppFEV1 50-80%) and >6 months follow up Aerosolized 4D-710 was well tolerated at doses up to 1E15 vg (n=6) Dose-dependent and widespread 4D-710-mediated CFTR transgene RNA and protein expression observed in all lung biopsies from all participants evaluated to date Pre-existing AAV immunity cross-reactive with A101 did not affect transgene expression, biological ac ...

Interim clinical data will be presented by Jennifer L. Taylor-Cousar, M.D., Principal Investigator of the AEROW clinical trial at the 47th European Cystic Fibrosis Conference on Thursday, June 6, 2024 at 5:00 p.m. BST Company to host webcast on Thursday, June 6, 2024 at 8:00 a.m. ET EMERYVILLE, Calif., May 30, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines ...