ESCONDIDO, Calif.--(BUSINESS WIRE)--Enthorin Therapeutics, LLC, a biotechnology company focused on circuit-modulating treatments for neurological and neurodevelopmental disorders, announced that its licensing partner for MRM-3379 (formerly ENT-3379), Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM), in December, reported the initiation of the BLOOM Phase 2 clinical study evaluating MRM-3379 in Fragile X syndrome (FXS). MRM-3379 is an orally available, highly brain-penetrant, selective phosphodiestera. ...

5 January 2026 China Pharma and Biotech China Pharma and Biotech 2026 Outlook: Off to the races Rebecca Liang, Ph.D. +852 2123 2656 rebecca.liang@bernsteinsg.com Ellie Li +852 2123 2621 ellie.li@bernsteinsg.com "The stock market is a device for transferring money from the impatient to the patient." Positive outlook at sector level: valuation-wise we're back in a more rational range than July - Aug, with most stocks dialing back 20-30%. The major healthcare and biotech indices are below the price levels of 2 ...

RAHWAY, N.J.--(BUSINESS WIRE)---- $MRK #MRK--Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the initiation of KANDLELIT-007, a Phase 3 clinical trial evaluating calderasib (MK-1084), an investigational oral selective KRAS G12C inhibitor, in combination with KEYTRUDA QLEXâ"¢ (pembrolizumab and berahyaluronidase alfa-pmph) for the first-line treatment of patients with KRAS G12C-mutant, advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC). This randomi ...

Core Viewpoint - Tvardi Therapeutics, Inc. experienced a significant decline in stock value, dropping over 80% following disappointing preliminary data from its Phase 2 REVERT clinical trial for TTI-101 in idiopathic pulmonary fibrosis [4] Group 1: Company Overview - Tvardi Therapeutics, Inc. is publicly traded on NASDAQ under the ticker TVRD [2] - The company is currently under investigation for potential claims related to its stock performance [2] Group 2: Clinical Trial Results - The Phase 2 REVERT clinical trial aimed to evaluate the safety, pharmacokinetics, and exploratory outcomes related to lung function of TTI-101 [4] - Preliminary data indicated that the trial did not meet its goals, with changes in Forced Vital Capacity (FVC) being a key measure [4] - Patients' baseline characteristics were similar across treatment arms, except for a lower percent predicted FVC in the placebo group compared to the TTI-101 group [4]

Core Insights - MediciNova, Inc. has provided an update on its Phase 2b/3 clinical trial of MN-166 (ibudilast) for Amyotrophic Lateral Sclerosis (ALS), known as the COMBAT-ALS study, with results presented at the 36th International Symposium on ALS/MND [1][2] Company Overview - MediciNova, Inc. is a clinical-stage biopharmaceutical company focused on developing novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases, with a late-stage pipeline including MN-166 (ibudilast) and MN-001 (tipelukast) [4][6] Clinical Trial Update - The COMBAT-ALS trial has successfully randomized a total of 234 participants, completing enrollment in September 2025 [5] - The baseline characteristics of participants include a mean age of 60.6 years, with 36.8% female and 63.2% male, and a racial distribution predominantly Caucasian (90.2%) [5] - The mean ALSFRS-R score at screening was 40.6, indicating the severity of the disease among participants [5] Drug Development and Designation - MN-166 (ibudilast) has received Orphan Drug Designation and Fast Track Designation from the FDA, as well as Orphan Designation from the European Commission for ALS treatment [3][2] - The drug is also in late-stage development for other neurodegenerative diseases and conditions, including progressive multiple sclerosis and glioblastoma [3][4] Future Expectations - Top-line data from the COMBAT-ALS study is anticipated by the end of 2026, with the company expressing hope that MN-166 will provide a significant therapeutic advance for ALS patients [2]

Group 1 - Faruqi & Faruqi, LLP is investigating potential claims against Tvardi Therapeutics, Inc. following significant losses suffered by investors in Tvardi stock or options [2][5] - The law firm has a history of recovering hundreds of millions of dollars for investors since its establishment in 1995 [4] - Moonlake Immunotherapeutics experienced a drastic decline in share price, dropping over 80% due to disappointing preliminary data from a Phase 2 clinical trial of TTI-101 [5] Group 2 - The Phase 2 REVERT clinical trial aimed to assess safety, pharmacokinetics, and exploratory outcomes related to lung function in patients with idiopathic pulmonary fibrosis [5] - Preliminary data indicated that the study did not meet its goals, with baseline characteristics being similar across treatment arms, except for a lower percent predicted Forced Vital Capacity (FVC) in placebo-treated patients compared to TTI-101-treated arms [5]

Core Viewpoint - BiomX Inc. is currently under FDA evaluation for its nebulizer device used in the Phase 2b trial of BX004 for cystic fibrosis, with a clinical hold in place due to data review concerns regarding the device [1][2][3]. Group 1: FDA Evaluation and Clinical Hold - The FDA has placed a clinical hold on the Phase 2b study while reviewing data submitted by BiomX regarding the third-party nebulizer used for BX004 delivery [2][4]. - No concerns were raised about the BX004 drug candidate itself in the clinical hold notification, and patient enrollment and dosing outside the U.S. continue as per protocol [3][4]. Group 2: Company Actions and Future Expectations - BiomX is collaborating with the nebulizer manufacturer to address the FDA's follow-up information requests, which are considered readily addressable [4]. - The company anticipates that U.S. enrollment will resume once the FDA's concerns are resolved, and topline results are now expected in the second quarter of 2026 following protocol updates [5][6]. Group 3: Financial Position and Stock Performance - As of September 30, 2025, BiomX reported a cash balance of $8.1 million, which is projected to fund operations into the first quarter of 2026 [7]. - BiomX shares experienced a decline of 7.18%, trading at $5.36, close to its 52-week low of $4.65 [7].

Core Viewpoint - Tvardi Therapeutics, Inc. experienced a significant decline in stock value, dropping over 80% following disappointing preliminary data from its Phase 2 REVERT clinical trial for TTI-101 in idiopathic pulmonary fibrosis [5] Group 1: Company Overview - Tvardi Therapeutics, Inc. is listed on NASDAQ under the ticker TVRD [2] - The company is currently under investigation by Faruqi & Faruqi, LLP for potential claims related to the significant losses suffered by investors [2][4] Group 2: Clinical Trial Results - The Phase 2 REVERT clinical trial aimed to evaluate the safety, pharmacokinetics, and exploratory outcomes related to lung function of TTI-101 [5] - Preliminary data indicated that the trial did not meet its goals, with safety data and exploratory efficacy results showing no significant improvement in Forced Vital Capacity (FVC) among treated patients compared to placebo [5] - The baseline characteristics of patients were similar across treatment arms, except for a lower percent predicted FVC in the placebo group [5]

Core Insights - OS Therapies Inc. (OSTX) is advancing towards bringing OST-HER2 to patients with recurrent, fully resected, pulmonary metastatic osteosarcoma, with significant clinical and regulatory milestones achieved in 3Q25 [1][3] - The company reported a 2-year overall survival (OS) rate of 75% for OST-HER2 compared to 40% in historical controls, with 100% 2-year survival among patients who were event-free at 12 months [1][5] - A planned spinoff of OS Animal Health (OSAH) into a standalone public company is expected in 1H26, allowing OSTX shareholders to receive direct equity participation [1][5] Regulatory Developments - The regulatory path is on track with a UK pre-MAA submission in December and FDA Type C filings targeted for January 2026, supported by the Phase 2b trial results [5] - The company is preparing for harmonized U.S. and ex-U.S. filings, with submissions to the UK MHRA and FDA expected around year-end [1][5] Financial Overview - As of the end of the quarter, OS Therapies had approximately $1.9 million in cash and reported a net loss of $6.9 million [5] - Post-quarter, the company secured $7.8 million in warrant proceeds and has an At-The-Market (ATM) facility in place, extending its financial runway into late 2026 [5]

Summary of Syndax Pharmaceuticals Conference Call Company Overview - **Company**: Syndax Pharmaceuticals (NasdaqGS:SNDX) - **Focus**: Oncology, specifically hematology with two approved products: Revuforj and Niktimvo [3][4] Key Products and Market Opportunities Revuforj - **Indications**: Approved for acute AML (KMT2A and NPM1) and ALL, targeting 40-50% of patients in relapsed refractory settings [3][4] - **Market Potential**: Estimated $2 billion opportunity in the relapsed refractory setting, with expansion into frontline therapy [4][9] - **Launch Performance**: Strong initial uptake with 70% of patients treated in second or third line; approximately one-third of patients are going to transplant [8][9] - **Patient Outcomes**: 50% overall response rate, with a duration of response of 23 months for responders [20][21] - **Future Growth**: Anticipated increase in patients returning for maintenance therapy post-transplant, potentially reaching 70-80% [24][25] Niktimvo - **Indications**: Approved for chronic GVHD in the third-line setting, with ongoing expansion into earlier lines [4][37] - **Market Potential**: Initial $2 billion opportunity, with potential growth to $5 billion as it moves into frontline settings [37][38] - **Launch Performance**: High patient persistency rates of 80-90% since launch [10][11] - **ASH Presentations**: Over 10 presentations planned, focusing on long-term therapy and real-world data [39][41] Clinical Trials and Research - **ASH Conference**: 23 abstracts submitted, with significant data expected for both Revuforj and Niktimvo [5][6] - **Ongoing Trials**: - **Revuforj**: Evolve trial comparing Venaza (Venetoclax and Azacitidine) with Revuforj in newly diagnosed patients [28][29] - **Niktimvo**: Phase 2 trial in IPF, targeting a large patient population with a new mechanism of action [44][45] Financial Outlook - **Profitability Guidance**: Expected to achieve profitability in the next few years, supported by stable expenses and robust revenue growth from both products [46] Additional Insights - **Physician Feedback**: Positive experiences reported with Revuforj, with no significant changes in prescribing behavior despite label updates [16][17] - **Patient Management**: Emphasis on the importance of monitoring and managing patient treatment duration, with expectations of increased therapy duration as maintenance becomes more common [27][28] This summary encapsulates the key points discussed during the conference call, highlighting the strategic direction and market potential for Syndax Pharmaceuticals' products.