Core Viewpoint - Galectin Therapeutics reported promising results for its lead drug belapectin in treating MASH-associated liver cirrhosis and portal hypertension, highlighting significant clinical efficacy and potential as a new treatment option [2][3][4]. Financial Highlights - As of March 31, 2025, the company had $7.4 million in cash and cash equivalents and $11 million available under credit lines [12]. - Research and development expenses for Q1 2025 were $6.5 million, down from $8.1 million in Q1 2024, primarily due to timing of expenditures related to the NAVIGATE clinical trial [12]. - General and administrative expenses decreased to $1.4 million in Q1 2025 from $1.6 million in Q1 2024 [12]. - The net loss applicable to common stockholders for Q1 2025 was $9.6 million, or ($0.15) per share, compared to a net loss of $11.5 million, or ($0.19) per share in Q1 2024 [12][20]. Clinical Trial Results - The NAVIGATE trial demonstrated that the 2 mg/kg dose of belapectin significantly reduced the incidence of new varices compared to placebo, validating earlier Phase 2 findings [2][3]. - Liver stiffness measurements (LSM) showed statistically significant reductions in the belapectin 2 mg/kg treatment arm at Weeks 26, 52, and 78 [11][20]. - The analysis indicated that 64% more patients in the placebo group experienced an absolute increase in LSM of >10 kPa compared to the 2 mg/kg group, reinforcing the treatment's efficacy [8][11]. Belapectin Program Highlights - Belapectin targets galectin-3, a protein involved in the pathogenesis of MASH and fibrosis, and has received Fast Track Designation from the FDA [5][16]. - The NAVIGATE trial is a global, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of belapectin for preventing esophageal varices in MASH cirrhosis [7][20]. Company Overview - Galectin Therapeutics focuses on developing therapies for chronic liver disease and cancer, with belapectin as its lead drug targeting MASH-related fibrosis [16].

撰文丨王聪 编辑丨王多鱼 排版丨水成文 代谢功能障碍相关脂肪性肝病 （MASLD） 及其进展形式—— 代谢功能障碍相关脂肪性肝炎 （MASH） ，已成为日益严重的公共卫生负担，且治疗选择有限。近期的研究表明，基于 成纤维细胞生长因子-21 （FGF21） 的类似物，能够显著改善 MASH，但其作用机制尚不明确。 在这项最新研究中，研究团队证明了 成纤维细胞生长因子-21 （FGF21） 对逆转 代谢功能障碍相关脂肪 性肝炎 （MASH） 的有益代谢作用，是通过不同的机制来独立降低肝脏甘油三酯和胆固醇水平来实现的。 具体而言，FGF21 信号直接作用于中枢神经系统 （CNS） 中的谷氨酸能神经元 （ glutamatergic neurons ） ，可刺激肝脏甘油三酯的减少以及纤维化的逆转，而 FGF21 信号直接作用于肝细胞，则足以 降低肝脏中的胆固醇水平。 从机制上来说，研究团队证明了 FGF21 在中枢神经系统 （CNS） 中发挥作用，增加肝脏交感神经活动， 从而抑制肝脏从头脂肪生成 （ de novo lipogenesis ） 。 2025 年 5 月 13 日， 爱荷华大学的研究人员在 Cell 子 ...

Core Insights - Madrigal Pharmaceuticals announced positive two-year results from the Phase 3 MAESTRO-NAFLD-1 trial of Rezdiffra, showing significant improvements in liver health metrics among patients with compensated MASH cirrhosis [1][2][3] Group 1: Study Results - The study involved 122 patients, with 113 completing two years of treatment, demonstrating a mean reduction of 6.7 kPa in liver stiffness, which is statistically significant [5][8] - 65% of patients with clinically significant portal hypertension (CSPH) at baseline moved to lower risk categories by year two, indicating a positive shift in patient outcomes [4][8] - Among patients with probable CSPH at baseline, 57% transitioned to the no/low CSPH category, further supporting the efficacy of Rezdiffra [4] Group 2: Safety and Tolerability - Rezdiffra was well-tolerated, with a low discontinuation rate due to adverse events; the most common side effects included diarrhea, COVID-19, and nausea [6][8] - Safety data were consistent with previous studies, reinforcing the drug's favorable safety profile [6] Group 3: Mechanism and Future Trials - Rezdiffra acts as a THR-β agonist, which is mechanistically supported to improve outcomes in patients with compensated MASH cirrhosis [7] - A larger placebo-controlled study is anticipated to confirm the benefits of Rezdiffra in this high-risk population, with ongoing trials expected to provide further insights [2][14] Group 4: Market Context - MASH is projected to become the leading cause of liver transplantation in the U.S., highlighting the urgent need for effective treatments [10][11] - An estimated 1.5 million patients have been diagnosed with MASH in the U.S., with Madrigal targeting approximately 315,000 patients with moderate to advanced fibrosis [12]

Results support potential benefit of efruxifermin (EFX) to elicit fibrosis improvement in patients with compensated cirrhosis (F4 fibrosis) due to MASH 96-week data from SYMMETRY trial presented at EASL Congress 2025 SOUTH SAN FRANCISCO, Calif., May 09, 2025 (GLOBE NEWSWIRE) -- Akero Therapeutics, Inc. (Nasdaq: AKRO), a clinical-stage company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, today announced publication of results from the ...

Core Insights - Akero Therapeutics presented promising results from the Phase 2b SYMMETRY trial for efruxifermin (EFX), indicating its potential to improve fibrosis in compensated cirrhosis caused by metabolic dysfunction-associated steatohepatitis (MASH) [1][2] Company Overview - Akero Therapeutics is a clinical-stage company focused on developing treatments for serious metabolic diseases with high unmet medical needs, particularly MASH [12] - Efruxifermin (EFX) is Akero's lead product candidate, currently undergoing evaluation in three ongoing Phase 3 studies [11][12] Clinical Trial Results - The SYMMETRY trial involved 182 patients with biopsy-confirmed compensated cirrhosis (F4) due to MASH, with 181 patients receiving EFX or placebo for 96 weeks [10] - At Week 96, 39% of patients treated with EFX 50mg showed fibrosis improvement compared to 15% in the placebo group (p=0.009) [2][3] - In the intent-to-treat (ITT) analysis, 29% of patients in the EFX 50mg group experienced fibrosis improvement versus 11% for placebo (p=0.031) [2][3] Efficacy and Safety - EFX demonstrated a significant increase in treatment effect over time, with a placebo-adjusted treatment effect doubling from 10% at Week 36 to 24% at Week 96 [5] - Non-invasive measures of liver fibrosis and injury, such as ELF test scores and liver stiffness by Fibroscan, showed continued improvement for the EFX 50mg group over 96 weeks [6] - The safety profile of EFX was consistent with previous trials, with mild to moderate gastrointestinal adverse events being the most common [8] Future Directions - Akero plans to continue evaluating EFX across all stages of MASH in the Phase 3 SYNCHRONY program [2] - The company aims to address the complex, multi-system disease state of MASH, including improvements in cardiovascular risk factors linked to the condition [11]

Core Insights - Altimmune, Inc. presented new analyses at the EASL Congress™ highlighting the predictive capability of the MASH Resolution Index (MASHResInd) for MASH resolution in patients treated with pemvidutide, a dual receptor agonist [1][2] Group 1: MASH Resolution Index (MASHResInd) - MASHResInd is a composite score that integrates multiple non-invasive tests, including MRI-PDFF, ALT, and AST levels, to predict MASH resolution on biopsy [2] - In a trial involving pemvidutide for MASLD, MASHResInd indicated a high probability of MASH resolution with treatment [2] Group 2: Pemvidutide Treatment Results - After 24 weeks of treatment, MASHResInd responses were observed in 69.2%, 92.3%, and 90.9% of subjects receiving 1.2 mg, 1.8 mg, and 2.4 mg of pemvidutide, respectively, compared to 22.2% in the placebo group [3][4] - The results suggest that pemvidutide may lead to significant histologic improvements in MASH, with high response rates at the higher doses [3][4] Group 3: Future Developments - The upcoming IMPACT Phase 2b Trial readout is anticipated to be reported this quarter, with confidence in achieving statistical significance based on the trial's results [4] - Pemvidutide has received Fast Track designation from the U.S. FDA for the treatment of MASH and is also being studied for obesity, alcohol use disorder, and alcohol-related liver disease [8][9]

Core Viewpoint - Sagimet Biosciences Inc. is advancing its clinical-stage biopharmaceutical development, particularly focusing on denifanstat for treating metabolic dysfunction associated steatohepatitis (MASH), with promising results from recent clinical trials and plans for further studies [2][5]. Clinical Development - The Phase 2b FASCINATE-2 trial of denifanstat in MASH patients showed successful results, especially in F3 stage patients [2]. - Denifanstat demonstrated similar pharmacokinetic characteristics and tolerability in a Phase 1 trial for patients with and without hepatic impairment [2]. - A Phase 1 clinical trial to evaluate the combination of denifanstat and resmetirom is anticipated to start in the second half of 2025, with results expected in the first half of 2026 [3][12]. Preclinical Data - Preclinical data presented at EASL 2024 indicated that the combination of a FASN inhibitor (TVB-3664) and resmetirom significantly improved liver disease markers, achieving an 80% improvement in NAS compared to 33% and 25% improvements from monotherapies [3][6]. Financial Results - For the quarter ended March 31, 2025, Sagimet reported a net loss of $18.2 million, compared to a net loss of $6.6 million for the same period in 2024 [8][16]. - Research and development expenses increased to $15.3 million from $5.3 million year-over-year, while general and administrative expenses rose to $4.5 million from $3.5 million [12][16]. - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $144.6 million [12][17]. Corporate Updates - The company successfully completed end-of-Phase 2 interactions with the FDA in October 2024, paving the way for Phase 3 trials in MASH [5]. - Leadership changes include George Kemble transitioning to non-executive Chair of the Board and the appointment of Beth Seidenberg as Lead Independent Director [5]. Industry Context - MASH is a severe liver disease affecting over 115 million people globally, with limited treatment options available [10]. - The renaming of NAFLD to MASLD and NASH to MASH aims to reduce stigma and improve diagnosis [10].

Core Insights - 89bio, Inc. is advancing its clinical-stage biopharmaceutical development, focusing on therapies for liver and cardiometabolic diseases, particularly targeting metabolic dysfunction-associated steatohepatitis (MASH) and severe hypertriglyceridemia (SHTG) [11] Clinical Trials - The Phase 3 ENLIGHTEN-Fibrosis and ENLIGHTEN-Cirrhosis trials are designed to support accelerated approval for treating patients with MASH, with topline data expected in 1H 2027 and 2028, respectively [1][5] - The ENTRUST trial for SHTG has been fully enrolled, with topline data anticipated in 1Q 2026 [1][5] - Pegozafermin has been recognized in a meta-analysis as one of the most effective agents for fibrosis improvement and MASH resolution, highlighting its potential in the market [2] Financial Position - As of March 31, 2025, the company reported cash, cash equivalents, and marketable securities totaling approximately $638.8 million, an increase from $439.9 million at the end of 2024 [7][15] - The company completed a follow-on equity offering in 1Q 2025, generating gross proceeds of $287.5 million [4] - A $150 million credit facility with K2 HealthVentures has been established, with $35 million drawn down [4] Expenses and Losses - Research and development expenses for Q1 2025 were $64.4 million, up from $47.4 million in Q1 2024, primarily due to increased clinical development costs [8] - General and administrative expenses rose to $11.5 million in Q1 2025 from $9.8 million in Q1 2024, driven by higher personnel-related costs [9] - The net loss for Q1 2025 was reported at $71.3 million, compared to a net loss of $51.7 million in Q1 2024, attributed to higher R&D and G&A expenses [10]

First-quarter 2025 Rezdiffra™ (resmetirom) net sales of $137.3 million As of March 31, 2025, more than 17,000 patients on Rezdiffra Two-year compensated MASH cirrhosis (F4c) data from MAESTRO-NAFLD-1 trial selected as oral late-breaker at EASL Congress (May 7-10)Appointed David Soergel, M.D., to Chief Medical Officer; Rebecca Taub, M.D., named Senior Scientific and Medical Advisor Appointed Jacqualyn Fouse, Ph.D., to Board of DirectorsReports cash, cash equivalents, restricted cash and marketable securities ...

Core Viewpoint - Viking Therapeutics has experienced a significant decline in stock price in 2025, down 35% year to date, despite a strong performance in 2024 due to promising clinical progress [1][2][3] Group 1: Company Performance - The company's leading candidate, VK2735, an investigational GLP-1 weight loss therapy, showed excellent phase 2 results and is expected to advance to phase 3 studies soon [5] - Viking's market cap is approximately $3 billion, and it is considered one of the more promising bets in the weight loss therapy sector, despite competition from larger companies like Eli Lilly and Novo Nordisk [8][6] - The company is also developing VK2809 for metabolic dysfunction-associated steatohepatitis (MASH), which has also passed phase 2 studies, and VK0214 for X-linked adrenoleukodystrophy, which has completed phase 1 studies [9][10] Group 2: Financial Position - Viking Therapeutics ended the first quarter with $852 million in cash and equivalents, down from $903 million at the end of 2024, indicating a solid financial position to support ongoing studies [11] - The company is expected to access additional funding easily due to its clinical progress, which should mitigate any immediate funding concerns [11] Group 3: Market Context - The weight loss market is experiencing rapid growth, with sales of existing therapies increasing significantly, and analysts predict continued growth in this sector at least until the early 2030s [7] - Despite the influx of investigational weight loss therapies, most have shown modest results, making Viking's promising data stand out in the competitive landscape [8]