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]

Stonegate Capital Partners Updates Coverage on OS Therapies Inc. (OSTX) 3Q25November 21, 2025 4:15 PM EST | Source: Reportable, Inc.Dallas, Texas--(Newsfile Corp. - November 21, 2025) - OS Therapies Inc. (NYSE American: OSTX): Stonegate Capital Partners updates their coverage on OS Therapies Inc. (NYSE American: OSTX). OS Therapies advanced key clinical, regulatory, and commercial milestones in 3Q25 as it moves closer to bringing OST-HER2 to patients with recurrent, fully resected, pulmonary m ...

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.

BridgeBio Pharma (NasdaqGS:BBIO) 2025 Conference November 18, 2025 11:00 AM ET Company ParticipantsMatt Outten - Chief Commercial OfficerThomas Trimarchi - CFOConference Call ParticipantsAndrew Tsai - Senior Biotech AnalystAndrew TsaiWelcome to Day Two of our London Healthcare Conference. I'm Andrew Tsai, Senior Biotech Analyst at Jefferies, and it's my pleasure to have the BridgeBio team with me. To my direct right, Thomas Trimarchi, CFO, and to his right, Matt Outten, Chief Operating Officer.Matt OuttenCo ...

Overview of SKID Treatment - The report focuses on a clinical trial for Severe Combined Immunodeficiency (SKID), also known as "bubble boy disease," and its successful treatment [2][3] - Untreated SKID has a life expectancy of approximately 2 years [4] - A clinical trial at UCLA, led by Dr Donald Con, offered gene therapy and bone marrow transplant as potential treatments [5][6] Treatment and Success Rate - The experimental treatment involves extracting stem cells from the patient's bone marrow, adding a normal ADA gene, and reintroducing the cells after chemotherapy to rebuild the immune system [7] - The clinical trial achieved a 95% success rate, with 59 out of 62 patients experiencing completely restored immune function over a 5-year period [8] - Post-treatment, children are able to live normal lives, attending school and engaging in typical childhood activities without unusual infections [8] Impact and Future Availability - UCLA has licensed the gene therapy to a biotech company, aiming for FDA approval within the next 2 to 3 years [15] - Babies are now screened for SKID before leaving the hospital, providing parents with early awareness [13]

Core Insights - Moleculin Biotech, Inc. is progressing with its pivotal Phase 2B/3 "MIRACLE" study of Annamycin for treating adult patients with relapsed or refractory acute myeloid leukemia (AML), with 60% of the target enrollment completed as of November 4, 2025 [1][2][4] - The company expects to complete treatment for the first 45 subjects by Q1 2026, with initial unblinded data anticipated shortly thereafter [1][4][5] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for AML treatment, and it is designed to avoid common cardiotoxicity associated with existing anthracyclines [7][9] Enrollment and Study Progress - The MIRACLE study is a global, multi-center, randomized, double-blind, placebo-controlled trial, combining data from its Phase 2B and Phase 3 portions to measure primary efficacy endpoints [3][10] - The first unblinding will involve 30 subjects treated with Annamycin in combination with HiDAC and 15 subjects treated with HiDAC plus placebo [3] - Recruitment is ongoing, with subjects enrolled from five countries, although some European sites are experiencing bed shortages affecting enrollment [5][2] Future Expectations - The company anticipates a second unblinding in the first half of 2026, following the initial unblinding of data [4] - The accelerated timeline for recruitment is attributed to positive feedback from potential investigators [4] - Annamycin is positioned as a potentially safer and more effective treatment option for AML, addressing a significant gap in current therapies [2][4]

Financial Data and Key Metrics Changes - Theravance Biopharma achieved non-GAAP break-even in Q3 2025, reflecting strong execution and financial discipline [2][18] - The company ended the quarter with approximately $333 million in cash and no debt, indicating a solid financial position [4][18] - Collaboration revenue increased to $20 million, up 19% year-over-year, driven by UPELRI's strong performance [18] Business Line Data and Key Metrics Changes - UPELRI net sales increased 15% year-over-year to $71.4 million, supported by strong demand growth and favorable net pricing [6][18] - UPELRI's profit margins reached record levels, with hospital volume increasing 29% year-over-year [7][9] - The company is on track to achieve a $25 million milestone from Viatris based on UPELRI's year-to-date sales [3][6] Market Data and Key Metrics Changes - UPELRI's share in the long-acting nebulized hospital market reached approximately 21%, a new high since launch [7] - Strong growth trends for Trelegy are expected to lead to a $100 million milestone in 2026 [4][17] Company Strategy and Development Direction - Theravance is focused on advancing the pivotal phase III Cypress trial of ampraloxetine, with top-line results expected in early 2026 [3][15] - The company aims to position ampraloxetine as the first precision therapy for symptomatic neurogenic orthostatic hypotension in patients with multiple system atrophy [4][15] - A new disease education campaign for healthcare professionals was launched to raise awareness of neurogenic orthostatic hypotension associated with MSA [5] Management Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution of the Cypress study and the potential of ampraloxetine to be a significant value inflection point for the company [3][20] - The company is optimistic about achieving near-term milestones totaling $75 million in Q4 2025 [4][19] - Management highlighted the importance of the upcoming Cypress data readout as a transformational catalyst for the company [20] Other Important Information - The company plans to host a KOL event for investors on December 8 to discuss the unmet medical need for patients with MSA and the potential of ampraloxetine [4][16] - The operational execution of the Cypress study has been aligned with FDA requirements, ensuring adequate powering for the trial [39] Q&A Session Summary Question: How does the company plan to balance returning capital to shareholders with the potential launch of ampraloxetine? - Management emphasized the importance of financial strength and the need to evaluate capital return timing based on the execution of the Cypress study [22][23] Question: Are there any pipeline assets being considered for future development? - Management stated that the current focus is on UPELRI and ampraloxetine, with potential evaluation of other options post-ampraloxetine success [25][27] Question: How does the recently published manuscript inform expectations for the Cypress data? - Management indicated that the publication is important for understanding the minimally clinically important difference needed for the Cypress study [30][33] Question: Can the company share enrollment numbers for the randomized withdrawal portion of the Cypress study? - Management confirmed confidence in enrollment numbers and operational execution, stating that the study is adequately powered [39][40]

Core Insights - AnaptysBio Inc. announced data from the Phase 2 trial of rosnilimab for moderate-to-severe ulcerative colitis, indicating that the drug did not meet its primary or key secondary endpoints [2][5]. Trial Results - Rosnilimab was found to be safe and well-tolerated, but it performed no better than placebo at Week 12, with clinical remission rates of 7% for patients receiving 400mg Q4W and 800mg Q2W, compared to 5% and 4% for endoscopic remission [2][5]. - The placebo rates in the trial were within expected historical ranges [2]. - Preliminary data suggested an increase in remission rates between Week 12 and Week 24, but these rates did not meet the six-month target product profile [5]. Financial Implications - The company will discontinue the ulcerative colitis trial, resulting in at least $10 million in savings [3]. Future Plans - AnaptysBio remains optimistic about the potential of rosnilimab in rheumatoid arthritis (RA) and plans to provide an update in H1 2026, including funding strategies that do not dilute royalties [4]. - The company is also conducting a Phase 1b study for ANB033 for celiac disease and plans to announce another Phase 1b study in a different inflammatory disease in 2026 [7]. Market Reaction - Following the announcement, AnaptysBio shares fell by 13.63%, trading at $34.24 [8]. Strategic Moves - AnaptysBio reiterated its intention to separate biopharma assets from substantial royalty assets in 2026, including expected royalties of over $390 million per year from Jemperli at GSK's peak sales guidance of over $2.7 billion [8].

Core Insights - Tvardi Therapeutics, Inc. experienced a significant stock decline of over 80% following disappointing preliminary data from its Phase 2 REVERT clinical trial for TTI-101 in idiopathic pulmonary fibrosis [5]. Company Overview - Tvardi Therapeutics, Inc. is currently under investigation by Faruqi & Faruqi, LLP for potential claims related to the significant losses suffered by investors [4]. - The firm has a history of recovering hundreds of millions of dollars for investors since its establishment in 1995 [4]. Clinical Trial Details - 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 lung function as measured by Forced Vital Capacity (FVC) [5]. - The baseline characteristics of patients were similar across treatment arms, except for a lower percent predicted FVC in the placebo group compared to the TTI-101 group [5].

Core Insights - Phathom Pharmaceuticals has initiated the Phase 2 pHalcon-EoE-201 clinical trial for VOQUEZNA (vonoprazan) as a treatment for eosinophilic esophagitis (EoE) in adults, marking a significant step in addressing unmet medical needs in gastrointestinal diseases [1][2][4] Company Overview - Phathom Pharmaceuticals is focused on developing and commercializing novel treatments for gastrointestinal diseases, having in-licensed the rights to vonoprazan for the U.S., Europe, and Canada [30] - VOQUEZNA is currently approved in the U.S. for multiple indications, including the treatment of Erosive Esophagitis and as part of combination therapies for H. pylori infection [29][30] Clinical Trial Details - The pHalcon-EoE-201 study is a two-part, randomized, double-blind, placebo-controlled trial involving approximately 80 adults with endoscopic-confirmed EoE and dysphagia, with the first part lasting 12 weeks [2][4] - Topline primary and secondary results from the trial are expected to be available in 2027 [3] Treatment Context - Eosinophilic esophagitis is a chronic immune condition characterized by eosinophil accumulation in the esophagus, leading to inflammation and swallowing difficulties [5] - Current treatment options for EoE are limited, with only two approved therapies available, highlighting the potential significance of VOQUEZNA's acid suppression profile as a non-steroidal treatment option [2][5]