Tamuzimod

10月22日，Ventyx Biosciences宣布了其对患有肥胖和心血管危险因素的患者进行每日一次口服VTX3232的 2 期研究的积极结果。 越来越多的证据表明，直接靶向 NLRP3 炎症小体可以显著减少潜在的炎症，而潜在炎症是动脉粥样硬化、心律失常、心力衰竭和相关心脏代谢紊乱的关键驱动因素。 这项 175 名参与者的研究检查了 VTX3232 与安慰剂，单独或与司美格鲁肽联合使用，评估安全性和耐受性作为主要终点，对炎症的影响（通过高敏 C 反应蛋白或 hsCRP 测量）作为次要终点。 临床试验设计 在改良分析集 （“MAS”） 中接受VTX3232单一疗法治疗的参与者2与基线相比，第 12 周时 hsCRP 降低了 78%，而安慰剂组增加了 3% （p<0.0001）。在完整分析集 （“FAS”） 中接受 VTX3232 治疗的参与者3与安慰剂组相比，第 12 周时 hsCRP 降低了 64%，而安慰剂组增加了 3% （p<0.0001）。接受VTX3232单一疗法治疗的受试者也显示出 IL-6 （p<0.0001） 降低至与心血管风险降低相关的水平 （IL-6 ≤1.65ng/L）1. 该研究还评估了VTX3232对与心血管风险相关的其他炎症生物标志物、肝脂肪变性和炎症以及包括血脂水平、血糖控制和体重减轻在内的代谢参数的影响。值得注意的是，VTX3232治疗导致脂蛋白（a）（“Lp（a）”）、纤维蛋白原和红细胞沉降率（“ESR”）的统计学显着降低。还观察到与脂肪变性无关的肝脏炎症显着减少（通过 cT1 校正的 MRI 测量）。 VTX3232单药治疗没有导致体重减轻，VTX3232与司美格鲁肽联合治疗也没有显示出比单独使用司美格鲁肽的增量体重减轻。 VTX3232在 2 期研究中安全且耐受性良好，不良事件发生率与安慰剂相当。 “我们对这项研究的结果非常满意，在给药的第一周内，hsCRP 降低了 ~80%，并且在具有可测量药物水平的参与者的整个 12 周给药期间保持不变。VTX3232还使近 70% 的研究参与者恢复到低于 2mg/L 的目标 hsCRP 水平，这是确定残余炎症风险的关键阈值。我们在这项研究中看到的对 IL-6、hsCRP、Lp（a） 和其他异常全身炎症标志物的影响，使我们相信VTX3232为新一代口服抗炎疗法带来了希望，这些疗法与降脂正交，可以进一步降低心血管事件的风险，“首席执行官 Raju Mohan 博士说。 “压倒性的证据表明，炎症标志物 C 反应蛋白或 CRP 的升高可以强烈预测未来的心血管事件。NLRP3 抑制剂等新型药物可以强效降低 CRP，为治疗动脉粥样硬化和预防其并发症的抗炎疗法提供了新的选择，从而增加了降低胆固醇疗法的既定益处，“麻省总医院布莱根心脏与血管研究所心血管专家、国际动脉粥样硬化学会前任主席 Peter Libby 医学博士说。 “在这项研究中，VTX3232强烈抑制了 NLRP3 通路，导致炎症级联反应显着减少，并表现出令人鼓舞的安全性，”首席医疗官 Mark Forman 医学博士说。“在与司美格鲁肽联合治疗组中，我们发现与单独使用司美格鲁肽相比，hsCRP、IL-6、Lp（a）和肝脏炎症显着减少，这表明该联合疗法可以作为适当患者GLP-1治疗的有力辅助疗法。这些结果支持进一步的发展和VTX3232解决炎症引起的高疾病负担的潜力。 关于Ventyx Biosciences Ventyx Biosciences 是一家临床阶段的生物制药公司，为自身免疫性疾病、炎症性和神经退行性疾病患者开发创新的口服疗法。 除了这些顶线结果外，VTX3232最近还完成了一项针对早期帕金森病患者的 2 期生物标志物研究。NLRP3 抑制剂产品组合还包括 VTX2735，这是一种外周限制性 NLRP3 抑制剂，目前正在进行针对复发性心包炎患者的 2 期研究。我们的炎症性肠病产品组合包括两种 2 期化合物：他木兹莫德 （VTX002），一种 S1P1R 调节剂，以及塞那替尼 （VTX958），一种 TYK2 抑制剂。 关于NLRP3靶点： 除Ventyx Biosciences还有少数几家公司布局NLRP3靶点，并已进入临床阶段，如成都先导的合作伙伴BioAge。 临床试验进展 BGE-102 启动 I 期临床研究：成都先导的合作伙伴 BioAge Labs, Inc. 启动了 NLRP3 抑制剂 BGE-102 的 I 期临床研究。BGE-102 是一种具有新颖结构、高活性及良好脑渗透性的口服小分子 NLRP3 抑制剂，开发用于治疗肥胖症。 IFM-2427/DFV890 开展多项临床研究：IFM Tre 开发的 NLRP3 拮抗剂 IFM-2427/DFV890 在首次人体剂量爬坡试验中耐受良好。2021 年启动了在家族性冷自炎综合征（FCAS）与膝骨关节炎的 II 期研究，2023/2024 年开展了髓系疾病、冠心病且高 hsCRP 人群、伴 CHIP 人群的 Ib/IIa 期研究。 关注公众号，获取更多资讯。 我是专注医药投资、商业健康险和药企团财险的邹军辉 18602014940  欢迎交流、询价。 真诚、专业、领先、服务

Positive topline Phase 2a biomarker data position VTX3232 as a potential disease-modifying therapy for patients with Parkinson’s disease Data from the Phase 2 study of VTX2735 in patients with recurrent pericarditis expected in Q4 2025 Phase 2 study of VTX3232 in participants with obesity and cardiometabolic risk factors is fully-enrolled and on track to yield topline data in early Q4 2025 Cash, cash equivalents and marketable securities balance of $209.0M as of June 30, 2025 expected to fund planned operations into at least H2 2026 SAN DIEGO, Aug. 07, 2025 (GLOBE NEWSWIRE) -- Ventyx Biosciences, Inc. (Nasdaq: VTYX) (“Ventyx”, “Company”), a clinical-stage biopharmaceutical company focused on developing innovative oral therapies for patients with autoimmune, inflammatory, and neurodegenerative diseases, today reported second quarter financial results and highlighted recent pipeline and business progress. “We continue to enroll patients in our Phase 2 trial of VTX2735 in recurrent pericarditis (RP), where we believe upregulation of NLRP3 and Caspase-1 inflammasome are central to the pathogenesis of this disease. VTX2735, an oral agent with potent inhibition of NLRP3 and Caspase-1, has the potential to displace biologic therapies used today in patients with severe RP, as well as penetrate RP market segments underserved by current biologic therapies” said Raju Mohan, PhD, President and Chief Executive Officer. “The other ongoing Phase 2 study of VTX3232 in obese participants with cardiometabolic risk factors is fully enrolled and we are on track to release topline results in early Q4 2025. Finally, recapping positive data from our Phase 2a trial of VTX3232 in patients with early-stage Parkinson’s disease, the study accomplished its primary objectives by demonstrating safety and tolerability, achieving high drug exposure in the cerebral spinal fluid (CSF) and potent suppression of NLRP3-related biomarkers in CSF and plasma. As previously indicated, we continue with Phase 2 planning in Parkinson’s disease with input from academic and industry experts.” Pipeline Updates and Anticipated Milestones NLRP3 Inhibitor Portfolio: Ventyx’s portfolio of potential best-in-class oral NLRP3 inhibitors in clinical development include VTX2735, a peripherally restricted NLRP3 inhibitor, and VTX3232, a central nervous system (CNS)-penetrant NLRP3 inhibitor. VTX3232 in Parkinson’s Disease (Positive topline Phase 2a data, June 2025): The Phase 2a trial of VTX3232 in patients with early stage, idiopathic Parkinson’s disease met its goal of establishing the safety and tolerability of VTX3232 in this patient population with no drug-related treatment-emergent adverse events observed. The compound demonstrated high drug exposures with steady state concentrations in CSF and plasma exceeding the IC90 for NLRP3 inhibition by ≥3-fold for 24-hours post-dose with a once-daily formulation. VTX3232 also showed clear evidence of target engagement in plasma and CSF with robust reductions in downstream biomarkers of NLRP3 inhibition, such as IL-1β, IL-6 and high-sensitivity C-reactive protein (hsCRP). In addition, VTX3232 treatment was associated with improved motor and non-motor symptoms of Parkinson’s disease, as measured by the MDS-UPDRS. Given the Phase 2a study met Ventyx’s internal criteria for continued clinical development, the Company initiated planning discussions for a placebo-controlled, Phase 2 trial in Parkinson’s disease and potentially in additional neurodegenerative disorders such as Alzheimer’s disease. Ventyx has been invited to present the Phase 2a data for VTX3232 at two Parkinson’s disease meetings this fall. An abstract detailing the Phase 2a VTX3232 study results has been selected for an Oral Platform Presentation at the International Congress of Parkinson’s Disease and Movement Disorders from October 5th – 9th. In addition, Ventyx will present the Phase 2a VTX3232 results at the Michael J. Fox Foundation’s Parkinson’s Disease Therapeutics Conference, being held on October 16th. VTX2735 in Recurrent Pericarditis (Q4 2025 data): The ongoing Phase 2, multicenter, 30-patient, open-label trial is evaluating VTX2735, dosed twice daily, in patients with recurrent pericarditis over a 6-week primary treatment period, followed by a 7-week extension period. Key endpoints include safety, change in the numerical rating scale (NRS) pain score, and change in hsCRP. Ventyx expects to release topline results from this study in Q4 2025. With the possibility of treating patients experiencing an active flare and preventing future recurrences with an oral agent, VTX2735 has the potential to streamline treatment for patients with recurrent pericarditis. VTX3232 in Cardiometabolic Diseases (Q4 2025 data): The Phase 2, multicenter, double-blind, placebo-controlled trial of VTX3232 in participants with obesity and cardiometabolic risk factors is fully-enrolled with subjects randomized to one of four groups for a 12-week primary treatment period: monotherapy placebo, monotherapy VTX3232, combination semaglutide + placebo, or combination semaglutide + VTX3232. Key endpoints include safety and change in hsCRP. The trial will also assess a panel of exploratory endpoints, including biomarkers of inflammation and cardiometabolic disease, as well as imaging to assess body composition and liver fat. Data from the Phase 2 trial are expected to guide future development of the Company’s NLRP3 inhibitors in cardiometabolic diseases. Inflammatory Bowel Disease (IBD) Portfolio: Tamuzimod (S1P1R Modulator, ulcerative colitis): Phase 2 induction data published in The Lancet (January 2025, doi:10.1016/S2468-1253(24)00386-8) showed that patients treated with tamuzimod experienced robust clinical and endoscopic remission rates compared to placebo. We believe combination treatment is emerging as a compelling concept in IBD to break through the modest clinical remission rates seen with monotherapies today. Tamuzimod’s efficacy and safety profile could position it as the backbone of future combination regimens with another oral or biologic agent. The Company is exploring partnership opportunities for tamuzimod in ulcerative colitis. VTX958 (TYK2 Inhibitor, Crohn’s disease): Phase 2 data suggest that VTX958 may have disease-modifying benefits in Crohn’s disease. Presentation of results in Crohn’s disease (20th Congress of the European Crohn’s and Colitis Organisation (ECCO), February 2025, Journal of Crohn's and Colitis, doi.org/10.1093/ecco-jcc/jjae190.1175 demonstrated a robust, dose-dependent endoscopic response at Week 12 for VTX958 compared to placebo, with a greater magnitude of reduction in two key biomarkers of inflammation, CRP and fecal calprotectin. Ventyx is exploring multiple options for continued development of VTX958 in Crohn's disease, including partnership opportunities. Second Quarter Financial Results Cash Position: Cash, cash equivalents and marketable securities were $209.0 million as of June 30, 2025. We believe our current cash, cash equivalents and marketable securities are sufficient to fund our planned operations into at least H2 2026. Research and Development (R&D) expenses: R&D expenses were $22.3 million for the second quarter of 2025, compared to $27.8 million for the second quarter of 2024. General and Administrative (G&A) expenses: G&A expenses were $7.1 million for the second quarter of 2025, compared to $7.9 million for the second quarter of 2024. Net loss: Net loss was $27.0 million for the second quarter of 2025, compared to $32.0 million for the second quarter of 2024. About Ventyx Biosciences Ventyx Biosciences is a clinical-stage biopharmaceutical company developing innovative oral therapies for patients with autoimmune, inflammatory, and neurodegenerative diseases. Our expertise in medicinal chemistry, structural biology, and immunology enables the discovery of differentiated oral small molecule therapeutics for conditions with high unmet medical need, and our extensive experience in clinical development allows the rapid progression of these drug candidates through clinical trials. Our portfolio of NLRP3 inhibitors includes VTX2735, a peripherally restricted NLRP3 inhibitor in Phase 2 development for recurrent pericarditis, and VTX3232, a CNS-penetrant NLRP3 inhibitor in Phase 2 development for neurodegenerative and cardiometabolic diseases. Our inflammatory bowel disease portfolio includes two Phase 2 compounds: tamuzimod (VTX002), an S1P1R modulator, and VTX958, a TYK2 inhibitor. For more information on Ventyx, please visit our website at https://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 of each of Ventyx’s product candidates, including the potential of VTX2735 and VTX3232, to emerge as best-in-class NLRP3 inhibitors and produce safe, effective or disease modifying results for the treatment of systemic inflammatory conditions or cardiometabolic or neurodegenerative diseases, the potential of VTX3232 as a disease-modifying therapy for Parkinson’s disease and additional neurodegenerative disorders such as Alzheimer’s disease, the anticipated timing of the enrollment of subjects and the estimated total subjects to enroll in the Phase 2 trials for VTX2735 and VTX3232; the timing of reporting data from either Phase 2 trial in Q4 2025; the potential for VTX2735 to penetrate the market for recurrent pericarditis, whether for severe cases or otherwise; management’s belief that upregulation of NLRP3 and Caspase-1 inflammasome are central to the pathogenesis of RP; management’s plans with respect to additional clinical trials of VTX3232 in patients with Parkinson’s, Alzheimer’s or another neurodegenerative disease as well as any cardiometabolic diseases, including the timing to commence, or funding of, such trials; management’s plans with respect to the commitment of internal resources toward further analysis, or development, including future studies, partnerships or other source of non-dilutive financing for tamuzimod in ulcerative colitis and VTX958 in Crohn’s disease, and related statements regarding strategic positioning; the potential for VTX3232 and VTX2735 in multiple cardiometabolic, systemic or neurological diseases and the continued development related thereto; the utility, safety or efficacy of a combination regimen including tamuzimod; the publication or presentation of clinical trial results at the International Congress of Parkinson’s Disease and Movement Disorders and the MJFF Parkinson’s Disease Therapeutics Conference; and the expected timeframe for funding Ventyx’s operating plan with current cash, cash equivalents and marketable securities. 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: potential delays in the commencement, 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; interim results not necessarily being predictive of final results; the potential of one or more outcomes to materially change as a trial continues and more patient data become available and following more comprehensive audit and verification procedures; regulatory developments in the United States and foreign countries; economic uncertainty in global markets caused by, among other things, geopolitical conditions, tariffs, military conflicts, and inflation volatility; 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, 2025, filed on or about the date hereof, 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 Contact: Joyce Allaire Managing Director LifeSci Advisors IR@ventyxbio.com