Financial Data and Key Metrics Changes - The company reported approximately $570 million in cash, providing a runway into 2028 based on the current operating plan [7] - The overall response rate in the RESILIENT-one study was 35%, with a 40% response rate in patients who progressed after chemotherapy [17][18] - The median duration of response was just under nine months across all groups [18] Business Line Data and Key Metrics Changes - The company is advancing multiple clinical-stage programs, including CLN978 for autoimmune diseases and CLN619 for non-small cell lung cancer [6] - Zipilertinib, the oral EGFR tyrosine kinase inhibitor, has shown promising results in patients with EGFR exon 20 mutations, with breakthrough therapy designation by the FDA [11][12] Market Data and Key Metrics Changes - EGFR mutated non-small cell lung cancer accounts for 16% of all non-small cell lung cancer, with exon 20 mutations representing approximately 12%, translating to an annual incidence of about 3,000 to 5,000 patients in the U.S. [67] - The company noted that patients with exon 20 mutations tend to have a poorer prognosis, highlighting the unmet need for effective therapies [68] Company Strategy and Development Direction - The company plans to pursue regulatory interactions for a potential U.S. NDA filing in the second half of the year [22][23] - There is an expansive development program for zipilertinib across multiple patient segments, including relapsed refractory disease and frontline settings [69][70] - The company retains 50% of the rights for zipilertinib in the U.S. and has a co-development and co-commercialization arrangement with partners at Tahoe [77][78] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the clinical profile of zipilertinib, noting its favorable safety profile compared to existing therapies [75] - The company anticipates significant uptake of zipilertinib once it becomes available, particularly as an oral option for patients [60][62] Other Important Information - The RESILIENT-two study will provide data on patients with active brain metastases and those with uncommon EGFR mutations in the second half of the year [79] - The RESILIENT-three study is ongoing, comparing zipilertinib plus chemotherapy to standard care [72] Q&A Session Summary Question: What is the efficacy of zipilertinib in patients with brain metastases? - The drug has shown encouraging preliminary data in patients with brain metastases, with good disease control observed [84][86] Question: What is the breakdown of patients in real-world settings? - Most patients are heavily pretreated, often having received chemotherapy and amivantamab before seeking other options [88][90] Question: Is there any data on the role of MET amplification in exon 20? - The response to this question was not directly addressed in the provided content, indicating a need for further research [94]

Core Insights - Cartesian Therapeutics has enrolled the first participant in its Phase 3 AURORA trial for Descartes-08, a cell therapy for myasthenia gravis (MG) [1][3] - Descartes-08 is an autologous engineered CAR-T therapy targeting B-cell maturation antigen (BCMA), designed for outpatient administration without preconditioning chemotherapy [2][5] - The Phase 3 trial aims to compare Descartes-08 to a placebo in approximately 100 participants, focusing on improvements in MG Activities of Daily Living (MG-ADL) scores [3][4] Company Overview - Cartesian Therapeutics is a clinical-stage biotechnology company focused on cell therapy for autoimmune diseases, with Descartes-08 as its lead asset in Phase 3 development for generalized MG [5] - The company is also developing Descartes-15, a next-generation CAR-T therapy currently in Phase 1 trials for multiple myeloma [5] Clinical Trial Details - The Phase 3 AURORA trial will randomize participants 1:1 to receive either Descartes-08 or placebo, with the primary endpoint being a three-point improvement in MG-ADL scores at Month 4 [3][4] - Previous Phase 2b trial results showed a significant average reduction of 4.8 points in MG-ADL scores at Month 12, with even greater reductions in patients without prior biologic therapy [4]

Core Insights - Cabaletta Bio, Inc. is a clinical-stage biotechnology company focused on developing curative targeted cell therapies for autoimmune diseases [3] - The company will participate in a fireside chat at the Jefferies Global Healthcare Conference on June 4, 2025 [1] - The lead strategy, CARTA, is centered on the investigational therapy rese-cel, which is currently in the RESET clinical development program [3] Company Overview - Cabaletta Bio is dedicated to advancing engineered T cell therapies that may provide deep and durable, potentially curative treatments for a variety of autoimmune diseases [3] - The company is headquartered in Philadelphia, PA, and has a comprehensive platform that includes two complementary strategies for therapy development [3] - Rese-cel is a fully human CD19-CAR T cell therapy that contains 4-1BB and is being evaluated across multiple therapeutic areas, including rheumatology, neurology, and dermatology [3]

Core Insights - Generation Bio Co. is focused on developing innovative therapeutics for T cell-driven autoimmune diseases [3] - The company will present at the Jefferies Global Healthcare Conference on June 4, 2025 [1] - A live webcast of the presentation will be available on the company's investor website [2] Company Overview - Generation Bio is developing redosable therapeutics that aim to reprogram T cells in vivo to combat autoimmune diseases [3] - The company utilizes cell-targeted lipid nanoparticles (ctLNP) for selective delivery of small interfering RNA (siRNA) to T cells [3] - This approach targets historically undruggable genes associated with autoimmune diseases, potentially unlocking new treatment avenues [3]

Zenas BioPharma Enabling patients with autoimmune diseases to reimagine life Corporate Presentation May 2025 1 Disclaimer FORWARD-LOOKING STATEMENTS: This presentation contains "forward-looking statements" which involve risks, uncertainties and contingencies, many of which are beyond the control of the Company, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. All statements other than statements of historical facts cont ...

Completed reverse merger and commenced trading on Nasdaq as JBIO Closed financing, raising approximately $300 million to date, providing cash runway through 2027 Lead candidate JADE101 expected to enter the clinic in second half of 2025, with interim biomarker-rich data expected in first half of 2026 SAN FRANCISCO and VANCOUVER, British Columbia, May 14, 2025 (GLOBE NEWSWIRE) -- Jade Biosciences, Inc. (“Jade”) (Nasdaq: JBIO), a biotechnology company focused on developing best-in-class therapies for autoimmu ...

Nektar Therapeutics (NKTR) Q1 2025 Earnings Call May 08, 2025 05:00 PM ET Company Participants Corinne Franklin - Investor RelationsHoward Robin - President and Chief Executive OfficerJonathan Zalevsky - Chief Research & Development OfficerSandra Gardiner - Chief Financial OfficerJulian Harrison - Managing DirectorMayank Mamtani - Senior Managing DirectorYu He - VP - Equity Research Conference Call Participants Yasmeen Rahimi - Sr. Research AnalystJay Olson - Managing Director & Senior Analyst - Biotechnolo ...

Corvus Pharmaceuticals (CRVS) Q1 2025 Earnings Call May 08, 2025 04:30 PM ET Company Participants Zack Kubow - Managing DirectorLeiv Lea - Chief Financial OfficerRichard Miller - Co-Founder, President, CEO & Chairman of the BoardGraig Suvannavejh - Managing DirectorLi Watsek - DirectorSean Lee - VP - Equity Research Conference Call Participants Aydin Huseynov - Equity Research AnalystJeff Jones - Managing Director and Senior AnalystRoger Song - Senior Equity Research Analyst Operator Good afternoon, everyon ...

NEW YORK, May 2, 2025 /PRNewswire/ -- Immunic, Inc. (Nasdaq: IMUX), a biotechnology company developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune diseases, today announced participation in the following scientific and industry conferences in May: May 3-6: Digestive Disease Week (DDW). Two abstracts discussing Immunic's phase 1/1b clinical trial of IMU-856, an orally available and systemically acting small molecule modulator that targets SIRT6 ...

Core Insights - Immunic, Inc. announced positive results from its phase 2 CALLIPER trial for vidofludimus calcium (IMU-838) in patients with progressive multiple sclerosis (PMS), showing a 20% reduction in the relative risk of 24-week confirmed disability worsening (24wCDW) events compared to placebo [1][2][8] Clinical Efficacy - In the overall PMS patient population (n=467), vidofludimus calcium reduced the relative risk of 24wCDW events by 20% compared to placebo, with a 30% reduction observed in the primary progressive multiple sclerosis (PPMS) subgroup (n=152) [2][8] - The drug also demonstrated a 15% reduction in the non-active secondary progressive multiple sclerosis (naSPMS) subgroup (n=268) [2] - A consistent reduction in disability worsening was noted in patients without gadolinium-enhancing lesions at baseline, with a 29% reduction in 24wCDW events compared to placebo [4][8] MRI Endpoints - Vidofludimus calcium reduced the annualized rate of thalamic brain volume loss by 20% compared to placebo, indicating a significant neuroprotective effect [5][16] - The total volume of new or enlarging T2 lesions showed a mean percent change of -0.22% for vidofludimus calcium versus +2.97% for placebo at month 24, highlighting the drug's efficacy in reducing lesion formation [6] Safety and Tolerability - The CALLIPER trial confirmed a favorable safety and tolerability profile for vidofludimus calcium, with treatment-emergent adverse events occurring in 69.4% of treated patients compared to 68.5% in the placebo group [9] - Serious adverse events were rare, observed in 8.1% of vidofludimus calcium-treated patients versus 6.5% in the placebo group, with no new safety signals identified [9] Future Directions - The company plans to advance vidofludimus calcium to a phase 3 registration study based on the positive results from the CALLIPER trial, particularly focusing on its potential as a neuroprotective treatment option for PMS [8][11] - Ongoing analysis of the full CALLIPER data set will be presented at upcoming scientific meetings, with the phase 3 clinical trial program for relapsing multiple sclerosis expected to be completed in 2026 [11][17]