DA-1726

75%患者的癌细胞完全消失，基因疗法3期临床结果积极Ferring Pharmaceuticals日前公布了在日本开展的一项3期临床试验的初步结果，该试验评估了基因疗法nadofaragene firadenovec在高危卡介苗（BCG）无应答的非肌层浸润性膀胱癌（NMIBC）伴原位癌（CIS）患者中的疗效和安全性。Nadofaragene firadenovec是该患者群体中首个获美国FDA批准的膀胱内给药非复制型基因疗法，商品名为Adstiladrin。数据显示，接受每季度一次治疗的患者，在3个月时的完全缓解率（CR）达75%（15/20）。此外，妙佑医疗国际（Mayo Clinic）最近公布的真实世界数据显示，接受nadofaragene firadenovec治疗的患者CR高达79%，证明了nadofaragene firadenovec在真实临床环境中同样具有持续一致的疗效和安全性。Nadofaragene firadenovec是一种基于非复制型腺病毒载体的基因疗法，每3个月经导管灌注，将携带编码干扰素α-2b的转基因的腺病毒载体递送至膀胱。通过转染膀胱内皮细胞，导致膀胱产生大量的干扰素α-2b蛋白。这种基因疗法可以将患者自身膀胱壁细胞转化为制造干扰素的微型工厂，增强机体抵抗癌症的天然防御能力。治疗致命脑瘤，CAR-T疗法获FDA突破性疗法认定BrainChild Bio公司今日宣布，在研自体B7‑H3靶向CAR-T细胞疗法BCB‑276已获美国FDA授予突破性疗法认定（BTD），用于治疗难治性儿童弥漫性内生性桥脑胶质瘤（DIPG）。这一FDA认定基于1期临床试验BrainChild‑03中观察到BCB‑276治疗脑瘤患者的总生存获益，该研究由BrainChild Bio的学术合作伙伴西雅图儿童医院开展，于近期发表于Nature Medicine。对于所有接受治疗的患者（n=21），从首次CAR-T细胞输注起的中位总生存期为10.7个月；从确诊起的中位总生存期为19.8个月。其中有3例患者从确诊至今仍存活，随访时间分别为44个月、45个月和52个月。BrainChild Bio正准备启动关键性2期临床试验，以支持向FDA提交BCB‑276的生物制品许可申请（BLA），用于治疗儿童及青少年DIPG患者。双重机制减重疗法公布最新临床试验结果MetaVia公司今日公布了在研疗法DA‑1726在1期临床试验多剂量递增（MAD）部分的额外顶线结果。DA‑1726是一种创新胃泌酸调节素（OXM）类似物，兼具胰高血糖素样肽‑1受体（GLP‑1R）和胰高血糖素受体（GCGR）激动活性。新闻稿指出，它具有成为“best-in-class”减重药物的潜力。在该研究的MAD部分，在8 mg至32 mg剂量范围内，受试者体重出现明显的剂量依赖性下降趋势，意味着更高剂量和更长期用药可能获得更佳疗效。此外，身体质量指数（BMI）在治疗组与安慰剂（PBO）组之间的差异更为显著，进一步支持了DA‑1726对体重相关指标的剂量依赖效应。▲1期临床试验MAD部分患者的BMI数据（图片来源：参考资料[3]）参考资料：[1] Ferring Announces Initial Data from Phase 3 Trial in Japanese Patients Demonstrating 75% Complete Response Rate at 3 Months with (nadofaragene firadenovec) in BCG-unresponsive NMIBC Patients. Retrieved April 22, 2025, from https://www.businesswire.com/news/home/20250421675994/en/Ferring-Announces-Initial-Data-from-Phase-3-Trial-in-Japanese-Patients-Demonstrating-75-Complete-Response-Rate-at-3-Months-with-nadofaragene-firadenovec-in-BCG-unresponsive-NMIBC-Patients[2] FDA Grants Breakthrough Therapy Designation for BrainChild Bio’s B7-H3 CAR T-Cell Therapy for Incurable Pediatric Brain Tumors. Retrieved April 22, 2025, from https://www.businesswire.com/news/home/20250422418430/en/FDA-Grants-Breakthrough-Therapy-Designation-for-BrainChild-Bios-B7-H3-CAR-T-Cell-Therapy-for-Incurable-Pediatric-Brain-Tumors[3] MetaVia Reports Additional Positive Top-Line Results From the MAD Part 2 of Its Phase 1 Study of DA-1726, a Novel 3:1 Ratio GLP-1 and Glucagon Dual Receptor Agonist to Treat Obesity, Further Demonstrating Its Best-In-Class Potential. Retrieved April 22, 2025, from https://www.prnewswire.com/news-releases/metavia-reports-additional-positive-top-line-results-from-the-mad-part-2-of-its-phase-1-study-of-da-1726-a-novel-31-ratio-glp-1-and-glucagon-dual-receptor-agonist-to-treat-obesity-further-demonstrating-its-best-in-class-potenti-302433906.html免责声明：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：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

Top-Line Data From MAD Part 2 of the Phase 1 Trial of DA-1726 Expected in April of 2025 Announced Positive Top-Line 16-Week Results from the Phase 2a Trial of DA-1241 for the Treatment of MASH, in December, Demonstrating Direct Hepatic Action in Addition to Its Glucose Lowering Effect $16.0 Million in Cash at End of Fourth Quarter Expected to Fund the Company Into the Third Quarter of 2025 CAMBRIDGE, Mass., March 20, 2025 /PRNewswire/ -- MetaVia Inc. (Nasdaq: MTVA), a clinical-stage biotechnology company focused on transforming cardiometabolic diseases, today announced financial results for the year ended December 31, 2024 and provided a corporate strategic update. "Throughout 2024, we made exceptional progress advancing the clinical development of our two, next generation cardiometabolic assets. We eagerly anticipate reporting top-line results from the expanded cohorts of the multiple ascending dose (MAD) Part 2 of our Phase 1 clinical trial of DA-1726, a novel, dual oxyntomodulin (OXM) analog agonist that functions as a glucagon-like peptide-1 receptor (GLP1R) and glucagon receptor (GCGR), for the treatment of obesity," stated Hyung Heon Kim, President and Chief Executive Officer of MetaVia. "In light of the strong safety profile from the planned cohorts of the SAD Part 1 and MAD Part 2 of the study, we are adding additional cohorts to further explore the maximum tolerated dose, which will allow us to realize the full potential of DA-1726. It is important to note that many patients using current GLP-1 agonists discontinue treatment due to tolerability issues, with 20% to 30% stopping within the first two months and up to 70% discontinuing within a year. Based on the preclinical data gathered so far and DA-1726's balanced activation of GLP1R and glucagon receptors, which enhances energy expenditure, we remain confident that DA-1726 has the potential to become a best-in-class obesity drug, offering superior tolerability compared to currently marketed GLP-1 agonists and those in late-stage clinical trials. The top-line data from the MAD Part 2 study, expected shortly, will give us an early read on clinical efficacy. We remain committed to the rapid clinical development of DA-1726 and continue to plan for an early proof-of-concept study to assess the efficacy and safety of DA-1726, which is expected to commence during the fourth quarter of 2025." Mr. Kim continued, "In December, we reported positive top-line 16-week results from the two-part Phase 2a clinical trial of DA-1241, a novel G-Protein-Coupled Receptor 119 (GPR119) agonist, in patients with presumed metabolic dysfunction-associated steatohepatitis (MASH). DA-1241 met the primary endpoint, achieving a reduction in alanine transaminase (ALT) levels through direct hepatic effects, along with key secondary endpoints, including significantly lower hemoglobin A1C (HbA1C) levels compared to placebo. These are very positive results especially given the small study size. Importantly, DA-1241 demonstrated excellent tolerability, with mostly mild adverse events and no drug-related serious adverse events in the treatment groups. Based on these findings, we remain confident that DA-1241's novel mechanism of action, which targets the inflammation associated with MASH, has the potential to result in a safe and effective treatment for this disease. We continue to conduct preclinical studies with a focus on exploring combination therapies for DA-1241, which has the potential to offer further benefits across the full spectrum of MASH. We look forward to reporting the complete data set, which we intend to present at a major medical meeting. Additionally, we anticipate scheduling an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) in the first half of 2025." Fourth Quarter 2024 and Subsequent Highlights January 2025: Held an Advisory Committee meeting at the 9th Annual MASH-TAG 2025 Conference, to discuss the positive top-line 16-week results from the two-part Phase 2a clinical trial of DA-1241. December 2024: Announced positive top-line 16-week results from the two-part Phase 2a clinical trial in patients with presumed MASH. In this trial, DA-1241 (100mg) demonstrated a statistically significant reduction in ALT levels at weeks 4 and 8, with a near statistically significant reduction at week 16. Statistically significant results were also achieved in multiple secondary endpoints including reductions in controlled attenuation parameter (CAP) and HbA1c. DA-1241 demonstrated similar trends in other liver enzymes including aminotransferase (AST) and gamma-glutamyl transferase (GGT). November 2024: Announced a strategic realignment with a name change from "NeuroBo Pharmaceuticals, Inc." to "MetaVia Inc.", reflecting the Company's focus on cardiometabolic diseases. In parallel, the Company's common stock began trading on the Nasdaq Stock Market under the new ticker symbol, "MTVA." November 2024: Announced completion of the last patient visit in the Company's two-part, Phase 2a clinical trial evaluating the efficacy and safety of DA-1241 for the treatment of patients with presumed MASH. Anticipated Clinical Milestones DA-1726 in Obesity: Top-line data from the additional cohorts in the MAD Part 2 is expected in April of 2025. The planned Phase 1 Part 3 will evaluate early proof of concept, with the first patient expected to be enrolled during the fourth quarter of 2025, followed by an interim data readout in or around mid-2026 and top-line results are expected in the second half of 2026. DA-1241 in MASH: The full data set from the two-part Phase 2a clinical trial of DA-1241 in MASH are expected to be presented at a major medical meeting in 2025. The Company expects to have an end-of-Phase 2 meeting with the FDA in the first half of 2025. Full Year 2024 Financial and Operating Results Research and Development (R&D) Expenses were approximately $21.6 million for the year ended December 31, 2024, as compared to approximately $9.2 million for the year ended December 31, 2023. The increase of approximately $12.4 million was primarily attributable to (i) $9.3 million in higher clinical trial expenditures, (ii) $2.5 million in higher expenditures for investigational drug manufacturing, non-clinical and preclinical costs related to expenses incurred under the Shared Services Agreement with Dong-A, and (iii) $1.2 million in higher employee compensation and benefits. These increases were partially offset by (i) $0.2 million in lower consulting expenditures and (ii) $0.4 million in lower other R&D costs. General and Administrative Expenses were approximately $7.3 million for the year ended December 31, 2024, compared to approximately $6.7 million for the year ended December 31, 2023. The increase of approximately $0.5 million was primarily due to $1.0 million in higher employee compensation and benefits, partially offset by (i) $0.4 million in lower consulting expenditures, and (ii) $0.1 million in legal and professional fees. Total Operating Expenses were approximately $28.8 million for the year ended December 31, 2024, compared to approximately $15.9 million for the year ended December 31, 2023. The approximately $12.9 million increase was attributable to higher R&D and G&A expenses. Other Income was approximately $1.2 million for the year ended December 31, 2024, compared to approximately $3.4 million for the year ended December 31, 2023. The decrease of approximately $2.2 million was primarily attributable to a $2.7 million lower gain related to the change in fair value of warrant liabilities due to warrant exercises in 2023 and the impact of the common stock's declining stock price during the last few years, partially offset by $0.5 million in higher interest income primarily due to higher average invested amounts in 2024. Net Loss for the year ended December 31, 2024, was $27.6 million, or $3.56 per basic and diluted share, based on 7,757,128 weighted average shares of common stock outstanding, compared with a net loss of $12.5 million, or $2.46 per basic and diluted share, based on 5,071,101 weighted average shares of common stock outstanding for the year ended December 31, 2023. Cash was $16.0 million as of December 31, 2024, compared with $22.4 million as of December 31, 2023. The company expects its cash position will be adequate to fund operations into the third quarter of 2025. About MetaVia MetaVia Inc. is a clinical-stage biotechnology company focused on transforming cardiometabolic diseases. The company is currently developing DA-1726 for the treatment of obesity, and is developing DA-1241 for the treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH). DA-1726 is a novel oxyntomodulin (OXM) analogue that functions as a glucagon-like peptide-1 receptor (GLP1R) and glucagon receptor (GCGR) dual agonist. OXM is a naturally-occurring gut hormone that activates GLP1R and GCGR, thereby decreasing food intake while increasing energy expenditure, thus potentially resulting in superior body weight loss compared to selective GLP1R agonists. DA-1241 is a novel G-protein-coupled receptor 119 (GPR119) agonist that promotes the release of key gut peptides GLP-1, GIP, and PYY. In pre-clinical studies, DA-1241 demonstrated a positive effect on liver inflammation, lipid metabolism, weight loss, and glucose metabolism, reducing hepatic steatosis, hepatic inflammation, and liver fibrosis, while also improving glucose control. In a Phase 2a clinical study, DA-1241 demonstrated direct hepatic action in addition to its glucose lowering effects. For more information, please visit . Forward Looking Statements Certain statements in this press release may be considered forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "believes", "expects", "anticipates", "may", "will", "should", "seeks", "approximately", "potential", "intends", "projects", "plans", "estimates" or the negative of these words or other comparable terminology (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this press release, including, without limitation, those risks associated with MetaVia's ability to execute on its commercial strategy; our expectations regarding the sufficiency of our existing cash on hand to fund our operations; the timeline for regulatory submissions; the ability to obtain regulatory approval through the development steps of MetaVia's current and future product candidates; the ability to realize the benefits of the license agreement with Dong-A ST Co. Ltd., including the impact on future financial and operating results of MetaVia; the cooperation of MetaVia's contract manufacturers, clinical study partners and others involved in the development of MetaVia's current and future product candidates; potential negative interactions between MetaVia's product candidates and any other products with which they are combined for treatment; MetaVia's ability to initiate and complete clinical trials on a timely basis; MetaVia's ability to recruit subjects for its clinical trials; whether MetaVia receives results from MetaVia's clinical trials that are consistent with the results of pre-clinical and previous clinical trials; impact of costs related to the license agreement, known and unknown, including costs of any litigation or regulatory actions relating to the license agreement; the effects of changes in applicable laws or regulations; the effects of changes to MetaVia's stock price on the terms of the license agreement and any future fundraising; and other risks and uncertainties described in MetaVia's filings with the Securities and Exchange Commission, including MetaVia's most recent Annual Report on Form 10-K. Forward-looking statements speak only as of the date when made. MetaVia does not assume any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Contacts: MetaVia Marshall H. Woodworth Chief Financial Officer +1-857-299-1033 [email protected] Rx Communications Group Michael Miller +1-917-633-6086 [email protected] - Tables to Follow - SOURCE MetaVia Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED