Telangiectasia, Hereditary Hemorrhagic

Diagonal is advancing its lead program to treat the underlying cause of hereditary hemorrhagic telangiectasia (HHT)WATERTOWN, Mass., Oct. 08, 2024 (GLOBE NEWSWIRE) -- Diagonal Therapeutics, a biotechnology company pioneering a new approach to discovering and developing agonist antibodies, today announced they will participate in multiple presentations at Cure HHT’s upcoming 15th HHT International Scientific Conference, taking place in Mandelieu-la-Napoule, France, October 15-19th, 2024. HHT is a severely debilitating bleeding disorder caused by mutations in ALK1 or Endoglin genes. These mutations lead to severe nosebleeds and gastrointestinal bleeds that lead to transfusion-dependent anemia. People with HHT are also at risk for life-threatening arteriovenous malformation (AVM) ruptures in critical organs such as the liver, lungs, and brain. The disease affects roughly 150,000 people in the United States and European Union. “We’re grateful to Cure HHT for inviting us to speak at their 15th annual meeting, which focuses on advancements in HHT research and emerging therapeutic options for patients. The need for better treatment options is evident, and we are pleased to be able to provide an update on our lead program," said Alex Lugovskoy, Ph.D., Chief Executive Officer of Diagonal. “While still early, our ALK1 agonist antibody, designed specifically for people with HHT, has demonstrated the ability to prevent and reverse the formation of AVMs in two HHT-relevant in vivo models and restored normal signaling in patient-derived cells. We hope these data can translate to significant patient benefit in the clinic." Details on the presentations are as follows: Poster: Bispecific antibodies targeting the ENG-ALK1-BMPRII axis as a novel approach for the treatment of HHT Presenter: JC Hus, Ph.D., Senior Director, Informatics, Diagonal Therapeutics Invited Talk: Therapeutic Potential of ALK1 Activating Drugs in HHT Models Presenter: Philippe Marambaud, Ph.D., Professor, Institute of Molecular Medicine, Feinstein Institutes for Medical Research and Diagonal Therapeutics scientific collaborator Invited Talk: Advancing a New Approach to Tackle the Root Cause of HHT Presenter: Eric Duhaime, SVP, Finance and Corporate Development, Diagonal Therapeutics About Hereditary Hemorrhagic Telangiectasia (HHT) HHT is a rare disease that affects more than 150,000 people in the US and EU, and for which there are currently no approved therapies. In HHT, mutations in the TGF-β receptor superfamily complex create two types of abnormal blood vessels – telangiectasias and arteriovenous malformations (AVMs) – that are fragile and susceptible to rupture and bleeding. These bleeding events lead to chronic anemia, necessitating frequent iron infusions or red blood cell transfusions. AVMs, if left untreated, may result in lung and brain hemorrhage, stroke, heart failure, and death. Diagonal’s agonist antibody candidates reactivate dormant signaling, addressing the root cause of the disease. About Diagonal TherapeuticsDiagonal Therapeutics is a biotech company pioneering a new approach to discovering and developing agonist antibodies. The Company's DIAGONAL platform combines proprietary computational and experimental techniques to overcome historical challenges associated with agonist antibody drug discovery. Diagonal's emerging pipeline – discovered using the DIAGONAL platform – has the potential to deliver life-changing therapies to patients by tackling the underlying cause of disease. For more information, please visit www.diagonaltx.com.Media Contact:Cory Trombleemedia@diagonaltx.com

MONDAY, Sept. 23, 2024 -- For patients with hereditary hemorrhagic telangiectasia (HHT), pomalidomide yields a significant reduction in epistaxis severity, according to a study published in the Sept. 19 issue of the New England Journal of Medicine . Hanny Al-Samkari, M.D., from Massachusetts General Hospital in Boston, and colleagues conducted a randomized, placebo-controlled trial to assess the safety and efficacy of pomalidomide for HHT. Patients were randomly assigned to receive 24 weeks of pomalidomide at a dose of 4 mg daily or matching placebo (95 and 49 patients, respectively). After a planned interim analysis met a prespecified threshold for efficacy, the trial was closed to enrollment in June 2023. The researchers found that the baseline mean Epistaxis Severity Score was 5.0 ± 1.5, consistent with moderate-to-severe epistaxis. The mean difference between the pomalidomide and placebo groups in the change from baseline in the Epistaxis Severity Score was −0.94 points at 24 weeks. The mean between-group difference in the change in the HHT-specific quality-of-life score was −1.4 points. The pomalidomide group more often had adverse events, including neutropenia, constipation, and rash. "The benefits of pomalidomide were most apparent over the course of the second 12 weeks of the trial, persisted during the four weeks after the end of the treatment period, and were not dependent on HHT genotype or baseline epistaxis severity," the authors write. Bristol Meyers Squibb donated the pomalidomide and matching placebo.

▎药明康德内容团队编辑 小分子疗法获FDA优先审评资格 Soleno Therapeutics今天宣布，美国FDA已接受为其在研疗法DCCR缓释片递交的新药申请（NDA），用于治疗4岁及以上经基因检测确认的Prader-Willi综合征（PWS）患者的暴食症。FDA同时授予这一申请优先审评资格，预计在今年年底之前完成审评。DCCR是一款二氮嗪胆碱（diazoxide choline）缓释片剂，已经获得美国FDA授予的突破性疗法认定和快速通道资格，并在美国和欧盟获得孤儿药资格。 DCCR是一种含有二氮嗪胆碱的创新专有缓释剂型，每日口服一次。DCCR开发项目的数据支持来自五项在健康志愿者中完成的1期临床研究和3项完成的2期临床研究，其中一项是在PWS患者中进行的。在PWS的3期临床开发项目中，DCCR在解决PWS的标志性症状暴食症以及其他几种症状（如攻击性/破坏性行为、脂肪质量和其他代谢参数）方面显示出可喜的效果。 别构AKT抑制剂概念验证临床试验获积极结果 Vaderis Therapeutics公司今日宣布，在研别构AKT抑制剂VAD044在治疗遗传性出血性毛细血管扩张症（Hereditary Haemorrhagic Telangiectasia，HHT）患者的随机双盲、安慰剂对照概念验证（POC）临床试验中取得了积极结果。 HHT是世界上第二常见的遗传性出血性疾病，常常导致严重的疾病负担、降低预期寿命和生活质量。尽管如此，目前全球范围内尚无获批的HHT治疗方法。Vaderis正在开发VAD044，这是一种口服、每日一次的别构AKT抑制剂。 在这项双盲对照试验中，75名来自美国和欧洲的患者被随机分配接受安慰剂、30 mg或40 mg的VAD044治疗，为期12周。安全性是主要终点，VAD044在脱靶不良事件（AEs）的发生频率和严重程度上与安慰剂相似。与AKT通路抑制相关的不良事件大多为轻度、短暂并可在研究期间解决。 几乎所有HHT患者都遭受不可预测、频繁且使人衰弱的鼻出血，这被认为是衡量整体HHT疾病活动和疾病负担的关键指标。在本研究中，VAD044在HHT的次要和探索性疗效终点上表现出剂量依赖性应答，包括关键的鼻出血终点。在12周治疗期结束时，接受40 mg剂量的患者在鼻出血频率、持续时间和无鼻出血天数方面均有临床意义的改善。 为癌症患者发现靶向治疗选择，FDA批准Illumina癌症生物标志物检测 Illumina公司今天宣布，其体外诊断（IVD）产品TruSight Oncology（TSO）Comprehensive检测获得美国FDA的批准，并获得了首批两项伴随诊断（CDx）适应症的批准。该检测可分析超过500个基因，以对患者的实体瘤进行全面分析，从而提高识别免疫肿瘤学生物标志物或临床可操作性生物标志物的可能性，这些标志物可以用于指导靶向治疗的选择或帮助临床试验患者入组。 TSO Comprehensive已获FDA批准作为伴随诊断工具，识别携带神经营养性酪氨酸受体激酶（NTRK）基因融合的成人和儿童实体瘤患者，这些患者可能从拜耳公司的Vitrakvi（larotrectinib）治疗中受益。该检测还获批用于识别RET融合阳性的局部晚期或转移性非小细胞肺癌（NSCLC）成人患者，这些患者可能从礼来公司的Retevmo（selpercatinib）治疗中受益。 新闻稿指出，大多数CDx测试通常仅针对一种类型的癌症，而TSO Comprehensive获得批准用于NTRK伴随诊断的所有实体瘤适应症，这有助于最大化每位患者活检中找到可针对信息的可能性。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Soleno Therapeutics Announces U.S. FDA Acceptance for Filing and Priority Review of NDA for DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome. Retrieved August 27, 2024, from https://www.globenewswire.com/news-release/2024/08/27/2936152/0/en/Soleno-Therapeutics-Announces-U-S-FDA-Acceptance-for-Filing-and-Priority-Review-of-NDA-for-DCCR-Diazoxide-Choline-Extended-Release-Tablets-in-Prader-Willi-Syndrome.html [2] Vaderis Announces Positive Clinical Proof-of-Concept Trial in HHT. Retrieved August 27, 2024, from https://www.prnewswire.com/news-releases/vaderis-announces-positive-clinical-proof-of-concept-trial-in-hht-302230320.html [3] FDA approves Illumina cancer biomarker test with two companion diagnostics to rapidly match patients to targeted therapies. Retrieved August 27, 2024, fromhttps://www.prnewswire.com/news-releases/fda-approves-illumina-cancer-biomarker-test-with-two-companion-diagnostics-to-rapidly-match-patients-to-targeted-therapies-302230979.html 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新