-- FDA alignment on accelerated and full approval pathways for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD) ---- Jeffrey M. Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE trial investigator, will present topline del-brax data from dose escalation cohorts in oral presentation ---- Stephen Tapscott, M.D., Ph.D., Professor of Human Biology and Clinical Research, Fred Hutchinson Cancer Center, will highlight results on the charac ...

Avidity Biosciences (RNA) Update / Briefing June 09, 2025 08:00 AM ET Speaker0 Good morning, and thank you so much for joining us today. I'm Annette Lang, Chief Business Officer at Avidity Biosciences. Today, we are excited to share several updates relating to our VELBAX program for people living with FSHD. Before we get started, I would like to share that this presentation contains forward looking statements as defined under applicable law. Forward looking statements involve risks and uncertainties, both k ...

This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and growth and other data about our industry. This data involves a number of assumptions and limitations, and the reader is cautioned not to give undue weight to such estimates. In addition, projections, assumptions, and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and ri ...

-- Unprecedented data from FORTITUDE™ dose escalation cohorts for del-brax treated participants, compared to placebo, demonstrate improvement in function, strength and PROs as well as rapid and significant reduction in biomarkers ---- Data support planned accelerated approval BLA submission in H2 2026 ---- Data being presented at the 32nd Annual FSHD Society International Research Congress (IRC); Investor and analyst webcast event today, Monday, June 9 at 8:00 a.m. ET --SAN DIEGO, June 9, 2025 /PRNewswire/ ...

纪要涉及的公司 Avidity Biosciences 纪要提到的核心观点和论据 1. **与FDA的互动** - 核心观点：公司与FDA特定部门合作多年，未受FDA人员变动影响，该部门专业且能按时完成工作，但行业需关注FDA变化并积极发声[5][6] - 论据：公司在FDA的一个部门内，合作的人员未变，该部门在三个项目上一直表现出色，按时完成任务 2. **知识产权与生产** - 核心观点：公司IP位于美国，在多个药物和技术方面有广泛专利，生产策略是分地区供应，与经验丰富的CDMO合作，不在中国生产[8][9][10] - 论据：公司在美国和其他国家生产药物，目标是美国药供美国、欧洲药供欧洲、亚洲通过亚洲枢纽供应 3. **公司概况与未来规划** - 核心观点：公司愿景是革新RNA领域，三个项目处于后期开发阶段，今年底计划提交首个BLA，多项研究按计划推进[11][12][13] - 论据：Del Zotia计划年底提交BLA，HARVEST研究预计年中完成患者招募，FSHD项目正与FDA讨论加速批准途径和全球三期研究 4. **FSHD项目** - 核心观点：公司是FSHD治疗领域领先者，选择2mg/kg每六周给药方案基于疗效和安全性，与FDA讨论进展积极，有望获得加速批准[13][18][24][40] - 论据：此前公布2mg/kg队列数据显示对DUX4调节基因有超50%的降低，循环生物标志物变化超25%，伴随肌酸激酶变化和功能改善趋势；两个剂量的循环生物标志物和肌酸激酶表现相似，选择低剂量可确保siRNA持续抑制DUX4；与FDA讨论详细且有成果，研究设计有灵活性 5. **DM1项目** - 核心观点：DM1项目进展顺利，有望明年获得全球批准，研究设计从疗效、安全和监管方面降低风险，支持报销，目标是尽快让药物惠及更多患者[44][45][51][55] - 论据：一期研究显示早期功能改善且持续到一年，安全性良好；与全球监管机构就研究设计达成一致；HARVEST研究规模大、设计严谨，可减少安慰剂效应和组间不平衡 其他重要但是可能被忽略的内容 1. **FSHD疾病的未满足需求**：FSHD是严重疾病，患者生活受极大影响，有很高的未满足医疗需求，患者最看重疾病稳定性，希望药物能长期控制病情[33][35][37] 2. **研究设计考虑报销因素**：公司在设计三期研究时会考虑报销问题，确保研究终点既符合监管要求，又便于支付方快速报销，如HARVEST研究和FSHD的全球三期研究[52]

Avidity Biosciences, Inc. (RNA) came out with a quarterly loss of $0.90 per share versus the Zacks Consensus Estimate of a loss of $0.88. This compares to loss of $0.79 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -2.27%. A quarter ago, it was expected that this company would post a loss of $0.84 per share when it actually produced a loss of $0.80, delivering a surprise of 4.76%.Over the last four quarters, the company has ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q _____________________________________________________ x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number 001-39321 _____________________________________________________ Avidity Biosciences, Inc. (Exact name of registrant as specified in its charter) _____________________________________________________ (State or other jurisdiction of incorporation ...

Exhibit 99.1 Avidity Biosciences Reports First Quarter 2025 Financial Results and Recent Highlights Company Highlights On track to deliver key regulatory updates for del-brax and topline data from dose escalation cohorts in the FORTITUDE trial in the second quarter TM Positive topline del-zota data further supports first BLA submission at year end 2025 – continues to highlight reproducibility and consistency across three late-stage clinical trials for DMD44, DM1 and FSHD Executing on global commercial infra ...

文章核心观点 - 阿维迪蒂生物科学公司股价因投资者对其罕见病产品线的积极态度而飙升，但预计季度亏损且营收下降，需关注后续走势；米鲁姆制药公司股价上涨，盈利预期有变化 [1][2][3] 阿维迪蒂生物科学公司（Avidity Biosciences） - 上一交易日股价飙升11.3%，收于29.77美元，成交量可观，过去四周股价下跌6.4% [1] - 股价突然上涨归因于投资者对公司杰出的罕见病产品线持积极态度，公司正在对三种不同肌肉疾病适应症的三个研究候选药物进行不同的中后期临床研究 [2] - 即将公布的报告预计季度每股亏损0.88美元，同比变化-11.4%，营收预计为295万美元，较去年同期下降16.7% [3] - 过去30天该季度的共识每股收益预期保持不变，股票目前的Zacks排名为3（持有），属于Zacks医疗 - 生物医学和遗传学行业 [4] 米鲁姆制药公司（Mirum Pharmaceuticals） - 上一交易日收盘上涨2.2%，收于45美元，过去一个月回报率为-2.6% [4] - 即将公布的报告的共识每股收益预期在过去一个月变化了-3.2%，至-0.37美元，与去年同期每股收益相比变化为+31.5%，目前Zacks排名为3（持有） [5]

On track to be first globally approved drug for FSHD SAN DIEGO, March 31, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced the completion of enrollment in the biomarker cohort in the Phase 1/2 FORTITUDE™ clinical trial of delpacibart braxlosiran (del-brax) in people living with facioscapulohumeral muscular dystrophy (FSHD). A total of 51 partici ...