NLRP3

▎药明康德内容团队编辑 创新PKC抑制剂获临床概念验证，有望挺进3期临床 IDEAYA Biosciences今日宣布，在研疗法darovasertib在治疗葡萄膜黑色素瘤（UM）的2期临床试验中获得积极数据。Darovasertib是一种强效的选择性蛋白激酶C（PKC）抑制剂，正在开发用于治疗原发性和转移性UM。目前，darovasertib正在四项正在进行的临床试验中接受评估，其中两项与辉瑞公司合作进行。Darovasertib与crizotinib联合治疗转移性葡萄膜黑色素瘤已获得FDA的快速通道资格。 IDE196-009是IDEAYA Biosciences发起的2期临床试验，评估darovasertib作为新辅助治疗用于UM患者，以及在眼球摘除术或放射治疗之后作为辅助治疗的效果。此外，还有一项由研究者发起的试验（IST）也在评估darovasertib作为UM的新辅助治疗的效果。 最新临床数据显示，接受新辅助治疗后，约59%患者的眼部肿瘤按直径乘积计算缩小超过20%，约49%患者的眼部肿瘤按直径乘积计算缩小超过30%，患者眼球保留率达到61%。综合来看，临床疗效数据为darovasertib用于新辅助治疗葡萄膜黑色素瘤提供了进一步临床概念验证。 IDEAYA Biosciences同时宣布已经与美国FDA就3期临床试验的设计达成共识，在确定临床试验最终方案后将启动3期临床试验。 赛诺菲布局NLRP3抑制剂 Ventyx Biosciences今日宣布，赛诺菲已同意对该公司进行2700万美元的战略投资。作为这项股权投资的一部分，Ventyx同意授予赛诺菲在VTX3232特定项目开发权益上的优先谈判权。 VTX3232是一种口服、选择性、可进入中枢神经系统（CNS）的NLRP3抑制剂，具有治疗多种神经炎症和神经退行性疾病的潜在疗效，包括帕金森病、心脏代谢疾病、阿尔茨海默病和多发性硬化等。今年第一季度，Ventyx公布了VTX3232在健康成人志愿者中的1期临床试验结果，新闻稿表示，这些数据支持VTX3232作为治疗神经炎症疾病的潜在“best-in-class”CNS渗透性NLRP3抑制剂的潜力。目前，VTX3232的开发项目包括针对早期帕金森病患者的2a期试验，以及针对肥胖且具有额外心脏代谢风险因素受试者的2期试验。两项试验的数据预计将在2025年公布。 开发创新多价疫苗，新锐完成1亿美元B轮融资 Vicebio今日宣布完成1亿美元的B轮融资，获得的资金将用于推动其双价疫苗VXB-241的1期临床试验，该疫苗靶向呼吸道合胞病毒（RSV）和人偏肺病毒（hMPV）。这两种病毒在老年患者和免疫系统较弱的人群中造成了重大疾病负担。这笔资金还将支持Vicebio多价疫苗管线的加速开发，包括VXB-251，这是一种靶向RSV、hMPV和副流感病毒3型（PIV3）的三价疫苗。 Vicebio致力于开发针对呼吸道病毒的下一代疫苗，采用其专有的分子钳技术（molecular clamp）。该技术由昆士兰大学发现，能够独特地稳定病毒糖蛋白处于高度免疫原性的融合前构象，这对于引发强有力的保护性免疫反应至关重要。该创新方法不仅能生产高度有效的疫苗，而且易于制造，并将以预充式注射器的形式提供使用。分子钳技术适用于多种病毒，包括RSV、hMPV、副流感病毒、流感病毒和冠状病毒。 ▲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] Ventyx Biosciences Announces $27 Million Strategic Investment from Sanofi. Retrieved September 23, 2024, from https://ir.ventyxbio.com/news-releases/news-release-details/ventyx-biosciences-announces-27-million-strategic-investment [2] Is Sanofi making a move into obesity? French pharma invests $27M into Ventyx. Retrieved September 23, 2024, from https://endpts.com/is-sanofi-making-a-move-into-obesity-french-pharma-invests-27m-into-ventyx/ [3] IDEAYA Biosciences Announces Positive Interim Phase 2 Data for Darovasertib and Successful FDA Type C Meeting on Registrational Trial Design for Regulatory Approval in Neoadjuvant Uveal Melanoma. Retrieved September 23, 2024, from https://prnmedia.prnewswire.com/news-releases/ideaya-biosciences-announces-positive-interim-phase-2-data-for-darovasertib-and-successful-fda-type-c-meeting-on-registrational-trial-design-for-regulatory-approval-in-neoadjuvant-uveal-melanoma-302254630.html [4] Vicebio Announces $100 Million Series B Financing and Initiation of Phase 1 Clinical Study of RSV/hMPV Bivalent Vaccine. Retrieved September 23, 2024, from https://vicebio.com/vicebio-100million-financing-clinicalstudy/ 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

LEHI, Utah, Sept. 23, 2024 /PRNewswire/ -- Halia Therapeutics, Inc. (Halia), a clinical-stage biopharmaceutical company advancing novel therapies for inflammation-related diseases, today announced promising preclinical results from their investigational compound HT-6184, combined with the GLP-1 agonist semaglutide. The study revealed significant weight loss in a diet-induced obesity (DIO) mouse model, demonstrating a synergistic effect that enhances weight reduction and preserves lean muscle mass, addressing two critical components of obesity management. Muscle tissue burns more calories at rest compared to fat tissue. Therefore, maintaining or increasing lean muscle mass can help boost the metabolic rate, assisting in more effective and sustained weight loss. In the 28-day DIO study, mice treated with the HT-6184 and semaglutide combination experienced a notable increase in weight loss. Combining HT-6184 with a subtherapeutic dose of semaglutide (1 nmol/kg) resulted in 50% greater weight loss than semaglutide alone over the study period. When HT-6184 was combined with a higher dose of semaglutide (2 nmol/kg), the combination achieved 25% greater weight loss than semaglutide alone, breaking through the typical weight loss plateau observed with semaglutide during the 28 days. Additionally, the combination therapy preserved lean mass, with a 10% improvement in lean mass preservation compared to the semaglutide-only group by the end of the study. "These preclinical findings are truly exciting, showcasing HT-6184's potential as a game-changing therapy for obesity," said Dr. David Bearss, Chief Executive Officer at Halia Therapeutics. "Combining HT-6184 with semaglutide enhances weight loss and preserves lean muscle mass—a crucial factor for overall metabolic health. These results bring us closer to a treatment that addresses the complex biology of obesity more holistically." Key Highlights of HT-6184: Synergistic Weight Loss: HT-6184, combined with semaglutide, resulted in 50% greater weight loss with subtherapeutic doses and 25% greater weight loss with higher doses than semaglutide alone. Lean Mass Preservation: Unlike current treatments that lead to muscle loss, the HT-6184 combination helped preserve lean body mass, with a 10% greater preservation than semaglutide alone. Anti-inflammatory Mechanism: HT-6184 targets inflammation, a key driver of obesity-related metabolic dysfunction. Potential in Type 2 Diabetes: Preliminary studies indicate that HT-6184 may also offer therapeutic benefits in Type 2 Diabetes by reducing inflammation and improving insulin sensitivity. Halia will present these findings at the upcoming 22nd Annual Discovery on Target Conference in Boston, MA, on September 30, 2024, at 12:25 p.m. ET. Dr. David Bearss will deliver a presentation titled "Allosteric Modulation of NEK7 to Inhibit the NLRP3 Inflammasome," highlighting HT-6184's mechanism of action, which targets the interaction between NEK7 and NLRP3 to disrupt inflammatory pathways. Dr. Bearss will discuss the potential of these preclinical findings to translate into human trials and the market outlook for obesity therapeutics. Dr. Bearss will also participate in the panel discussion: 'Novel Kinase Modulators' which will take place on September 30, at 1 p.m. ET. Halia plans to advance HT-6184 into a Phase 2 clinical trial for obesity later this year. This trial will evaluate the safety, tolerability, and efficacy of HT-6184 in combination with semaglutide for weight loss in adults who are obese or overweight with a weight-related comorbid condition. About Halia Therapeutics, Inc. Halia Therapeutics is discovering and developing a pipeline of novel therapeutics to improve patients' lives with chronic inflammatory disorders and neurodegenerative diseases, with its initial programs targeting NEK7 and LRRK2. Halia's lead candidate, HT-6184, a novel NEK7/NLRP3 inhibitor, has completed a Phase I study ( NCT05447546) evaluating the safety and tolerability of HT-6184 when administered as single or multiple oral doses at escalating dose levels in healthy volunteer subjects and has also completed a Phase II trial evaluating the efficacy of HT-6184 on post-procedure diagnostic biomarkers of inflammation and pain ( NCT06241742). Halia is currently undergoing a Phase II trial investigating the efficacy of HT-6184 for the treatment of lower-risk myelodysplastic syndromes (LR-MDS). The company's headquarters are in Lehi, Utah. For more information, visit or follow us on LinkedIn and Twitter (X). Company Contact Halia Therapeutics [email protected] +1 (385) 355-4315 Media Contact Ignacio Guerrero-Ros, Ph.D. Russo Partners, LLC +1 (646) 942-5604 [email protected] SOURCE Halia Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Sanofi to receive an exclusive right of first negotiation for Ventyx’s CNS-penetrant NLRP3 inhibitor VTX3232SAN DIEGO, Sept. 23, 2024 (GLOBE NEWSWIRE) -- Ventyx Biosciences, Inc. (Nasdaq: VTYX) (“Ventyx”), a clinical-stage biopharmaceutical company focused on advancing novel oral therapies that address a broad range of inflammatory diseases with significant unmet medical need, today announced that Sanofi has agreed to make a $27 million strategic investment in the Company at an as-converted price of $3.8243 per share of common stock, pursuant to which Sanofi will purchase 70,601 of the Company’s Series A non-voting convertible preferred stock, each share of which is initially convertible into 100 shares of common stock. The closing of the transaction is expected to occur on September 23, 2024. “We are very pleased with this strategic investment from Sanofi,” said Raju Mohan, Chief Executive Officer. “Sanofi is a global leader in immunological and inflammatory diseases, and we believe this transaction is a recognition that VTX3232 could potentially offer a disease-modifying approach for the treatment of a number of neuroinflammatory conditions with high unmet medical need. We look forward to strengthening our relationship with Sanofi as the VTX3232 clinical programs progress, with data from the Phase 2a trial in patients with early Parkinson’s disease and data from the Phase 2 trial in subjects with obesity and additional cardiometabolic risk factors, both expected in 2025.” In connection with the equity investment, the Company has agreed to grant Sanofi an exclusive right of first negotiation with respect to certain VTX3232 program rights. The proceeds from the investment are expected to further strengthen the Company’s cash position, and current cash, cash equivalents and marketable securities, inclusive of the proceeds from the investment, are expected to be sufficient to fund planned operations into at least the second half of 2026. About VTX3232 VTX3232 is an oral, selective, CNS-penetrant NLRP3 inhibitor with potential therapeutic utility for a range of neuroinflammatory and neurodegenerative conditions, including Parkinson’s disease, cardiometabolic disease, Alzheimer’s disease, and multiple sclerosis, among others. In the first quarter of this year, Ventyx announced results from a Phase 1 trial of VTX3232 in adult healthy volunteers where steady-state exposures achieved with once-daily doses of VTX3232 exceeded the interleukin-1β (IL-1β) IC90 in both plasma and cerebrospinal fluid over 24 hours. We believe these data support the potential for VTX3232 to emerge as a best-in-class CNS-penetrant NLRP3 inhibitor for the treatment of neuroinflammatory diseases. Current development plans for VTX3232 include a Phase 2a trial in patients with early Parkinson’s disease and a Phase 2 trial in subjects with obesity and additional cardiometabolic risk factors. Data from both trials are expected in 2025. About Ventyx Biosciences Ventyx is a clinical-stage biopharmaceutical company focused on developing innovative oral medicines for patients living with autoimmune and inflammatory disorders. We believe our ability to efficiently discover and develop differentiated drug candidates will allow us to address important unmet medical needs with novel oral therapies that can shift inflammation and immunology markets from injectable to oral drugs. Our current pipeline includes internally discovered clinical programs targeting NLRP3, S1P1R and TYK2, positioning us to become a leader in the development of oral immunology therapies for peripheral and neuroinflammatory diseases. Ventyx is headquartered in San Diego, California. For more information about Ventyx, please visit www.ventyxbio.com. Forward-Looking Statements Ventyx cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on Ventyx’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the potential for VTX3232 to offer a disease-modifying approach for the treatment of neuroinflammatory conditions with high unmet medical need; the impact of Sanofi’s strategic investment and Ventyx’s relationship with Sanofi; clinical progress regarding VTX3232, including the timing of data from the Phase 2a trial in patients with early Parkinson’s disease and data from the Phase 2 trial in subjects with obesity and additional cardiometabolic risk factors; the expected timeframe for funding Ventyx’s operating plan with current cash, cash equivalents and marketable securities, inclusive of the proceeds from Sanofi’s investment; the potential therapeutic utility of VTX3232 for a range of neuroinflammatory and neurodegenerative conditions, including Parkinson’s disease, cardiometabolic disease, Alzheimer’s disease, and multiple sclerosis, among others; the potential of VTX3232 to emerge as a best-in-class CNS-penetrant NLRP3 inhibitor for the treatment of neuroinflammatory diseases; Ventyx’s ability to address important unmet medical needs with novel oral therapies that can shift inflammation and immunology markets from injectable to oral drugs; and Ventyx’s positioning to become a leader in the development of oral immunology therapies for peripheral and neuroinflammatory diseases. The inclusion of forward-looking statements should not be regarded as a representation by Ventyx that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Ventyx’s business, including, without limitation: Ventyx’s relationship with Sanofi may not result in the anticipated positive outcomes; potential delays in the enrollment and completion of clinical trials; Ventyx’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; disruptions in the supply chain, including raw materials needed for manufacturing and animals used in research, delays in site activations and enrollment of clinical trials; the results of preclinical studies and clinical trials; early clinical trials not necessarily being predictive of future results; the ongoing contributions to scientific literature as pertains to the relationships between NLRP3, neuroinflammation and Parkinson’s disease; unexpected adverse side effects or inadequate efficacy of Ventyx’s product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; Ventyx’s ability to obtain and maintain intellectual property protection for its product candidates; the use of capital resources by Ventyx sooner than expected; and other risks described in Ventyx’s prior press releases and Ventyx’s filings with the Securities and Exchange Commission (SEC), including in Part II, Item 1A (Risk Factors) of Ventyx’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, filed on August 8, 2024, and Ventyx’s subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Ventyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Relations ContactPatti BankManaging DirectorICR Westwicke(415) 513-1284IR@ventyxbio.com