Core Viewpoint - Adial Pharmaceuticals has executed agreements with Cambrex and Thermo Fisher to support the production of its lead investigational drug AD04, aimed at treating Alcohol Use Disorder (AUD) in the U.S. [1][2][3] Company Overview - Adial Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing therapies for addiction and related disorders, with AD04 being a genetically targeted serotonin-3 receptor antagonist [4]. - The company recently conducted the ONWARD™ pivotal Phase 3 clinical trial for AD04, which showed promising results in reducing drinking among heavy drinking patients without significant safety concerns [4]. Agreements and Collaborations - The collaboration with Thermo Fisher as the Contract Development and Manufacturing Organization (CDMO) and Cambrex as the drug substance supplier has commenced, focusing on the completion of demonstration batches necessary for clinical trials [2][3]. - The agreements encompass all manufacturing phases for clinical supplies and the Chemistry, Manufacturing, and Controls (CMC) documentation required for the NDA submission to the FDA [2]. Strategic Importance - The selection of CDMOs with proven track records in both drug substance development and manufacturing was crucial for the agreements [2]. - The partnerships are expected to be vital for meeting timelines for the upcoming Phase 3 clinical trials and addressing commercial demands for AD04, especially considering recent tariff implications [3]. Future Potential - AD04 is not only targeted for AUD but is also believed to have potential applications in treating other addictive disorders such as Opioid Use Disorder, gambling, and obesity [4].

Nurix Therapeutics (NRIX) Update / Briefing June 12, 2025 08:00 AM ET Speaker0 Welcome to the New York's Therapeutics twenty twenty five investor call. At this time, all participants are in a listen only mode. Later, we will conduct a question and answer session. I would now like to turn the conference over to Newark's President and CEO, Arthur Sands. Arthur, you may begin. Speaker1 Thanks, Paul. Welcome to our call and our presentation today entitled meeting the needs of patients with CLL and Waldenstrom's ...

Phase 2 trial results continue to show clinically meaningful efficacy and durable responses, including 36.5-month median overall survival after four years of follow-up, with a manageable safety profileFindings presented today at ASCO 2025 and concurrently published in The Lancet OncologyFor U.S. media and investors only DUBLIN, June 2, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced long-term data, including the first report of median overall survival (OS) from the Phase 2 trial ...

Core Insights - Taiho Oncology, Inc. and Cullinan Therapeutics, Inc. announced positive results from the REZILIENT1 trial for zipalertinib in treating non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations [1][4] - The trial results will be presented at the 2025 ASCO Annual Meeting [1] Company Overview - Taiho Oncology specializes in developing and commercializing orally administered anti-cancer agents, focusing on solid tumors and hematological malignancies [9] - Cullinan Therapeutics is dedicated to creating new standards of care in cancer and autoimmune diseases, with a diversified portfolio of clinical-stage assets [11] Clinical Trial Details - REZILIENT1 is a Phase 1/2 global study evaluating the efficacy and safety of zipalertinib in patients with advanced or metastatic NSCLC harboring EGFR exon 20 insertion mutations [5] - The primary endpoints of the trial were overall response rate (ORR) and duration of response (DOR) [5] Efficacy Results - Zipalertinib demonstrated a confirmed ORR of 35.2% overall, with a median DOR of 8.8 months and progression-free survival of 9.4 months [8] - In patients who received prior platinum-based chemotherapy, the ORR was 40% with a median DOR of 8.8 months [8] - Subgroup analyses showed a confirmed ORR of 30% and median DOR of 14.7 months in patients who had prior amivantamab [8] Safety Profile - The safety profile of zipalertinib was manageable and consistent with previously reported data [8]

Core Insights - Ascentage Pharma announced new clinical data for its drug candidates, lisaftoclax and alrizomadlin, which will be presented at the 2025 ASCO Annual Meeting, indicating their potential in treating various cancers [1][2] Group 1: Lisaftoclax - Lisaftoclax is a Bcl-2 inhibitor being evaluated in a Phase 1b/2 study in combination with azacitidine for treatment-naïve or prior venetoclax-exposed myeloid malignancies [2][5] - The study enrolled nearly 100 patients, showing that the combination was well tolerated with preliminary efficacy; the overall response rate (ORR) for treatment-naïve myelodysplastic syndromes (MDS) was 64% [6][8] - In patients with venetoclax-refractory acute myeloid leukemia (AML), the ORR was 17%, while for high-risk MDS, it was 50% [6][8] Group 2: Alrizomadlin - Alrizomadlin is an MDM2-p53 inhibitor showing initial clinical activity in advanced adenoid cystic carcinoma (ACC) and other solid tumors [9][10] - The monotherapy arm demonstrated an ORR of 22.2% in ACC patients, with a disease control rate (DCR) of 100% in MPNST patients [12] - In the combination arm with toripalimab, the ORR was 20% in biliary-tract cancer patients, and 16.7% in liposarcoma patients, with prolonged progression-free survival observed in some MPNST patients [13]

Summary of ALX Oncology Holdings (ALXO) Conference Call Company Overview - **Company**: ALX Oncology Holdings (ALXO) - **Focus**: Development of ALX2004, a differentiated antibody-drug conjugate (ADC) targeting EGFR Key Points and Arguments Industry and Product Development - **ADC Focus**: ALX2004 is designed to maximize therapeutic window and overcome historical toxicity challenges associated with EGFR-targeted ADCs [5][6][10] - **Clinical Pipeline**: The company is advancing its ADC in combination with anti-cancer antibodies, specifically targeting breast and colorectal cancers [4][5] - **IND Clearance**: ALX2004 has received Investigational New Drug (IND) clearance, with plans to launch a Phase I trial in mid-2025 [5][7] Scientific Rationale - **Payload and Linker Design**: ALX2004 utilizes a proprietary topoisomerase one inhibitor payload, designed to minimize off-target toxicity while maximizing tumor cell killing [17][20] - **Immunogenic Cell Death**: The payload triggers immunogenic cell death, potentially enhancing long-term tumor control through adaptive immune response [18][28] - **Optimized Antibody**: The antibody component is designed to block EGFR signaling and bind to a unique epitope, potentially overcoming resistance to existing therapies [30][32] Clinical Development Plans - **Phase I Study**: The study will focus on four tumor types: head and neck squamous cell carcinoma, colorectal cancer, non-small cell lung cancer, and esophageal squamous cell carcinoma, targeting patients with relapsed or refractory disease [40][41] - **Safety and Efficacy Goals**: Initial safety data is expected in the first half of 2026, with an emphasis on establishing a recommended dosing schema [42][43] Competitive Landscape - **Market Positioning**: ALX Oncology aims to be a leader in the ADC space targeting EGFR, which currently lacks an approved drug [43][85] - **Comparison with Competitors**: The company is aware of other ADCs in development, such as MRG003, but believes its optimized design gives it a competitive edge [85] Additional Important Insights - **Preclinical Data**: Robust preclinical data supports the efficacy and safety profile of ALX2004, with encouraging results in various tumor models [14][34] - **Toxicity Management**: The design aims to avoid common toxicities seen in previous EGFR-targeted ADCs, such as skin toxicity and interstitial lung disease [14][72] - **Funding and Capital Allocation**: The company has sufficient cash to fund its ongoing studies into 2024, with plans to explore additional capital options as needed [51][52] This summary encapsulates the critical aspects of ALX Oncology's conference call, highlighting the company's strategic focus on developing ALX2004 and its potential impact on the oncology market.

Targeted Therapy Delivered Corporate Presentation | May 8, 2025 Nasdaq: LSTA www.lisata.com © Lisata Therapeutics, Inc. 2025. All rights reserved. Disclosure Forward-looking statements advisory This presentation contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this communication regarding strategy, futur ...

CARLSBAD, Calif., May 01, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced favorable data from a Phase 1 single- and multiple-ascending dose (SAD/MAD) trial of DT-168 in healthy volunteers, which will be presented on May 2, 2025 at Eyecelerator @ Park City 2025, an event backed by the American Academy of Ophthalmology highlighting industry advancements and innovative new ...

Financial Data and Key Metrics Changes - Scholar Rock reported an outstanding year in 2024, with significant progress towards regulatory milestones for apitegromab, including the submission of the BLA in the U.S. in January and the upcoming MAA submission in the EU in March [6][36]. - The company anticipates a transformative year in 2025, with plans for a potential launch of apitegromab in the U.S. in Q4 2025 and in the EU in 2026 [37]. Business Line Data and Key Metrics Changes - The SAPPHIRE study demonstrated that apitegromab plus standard-of-care resulted in a 1.8% improvement in the Hammersmith Functional Motor scale compared to placebo, with 30% of patients achieving a 3-point or greater improvement [9][10]. - The cardiometabolic program is on track to share top-line data from the EMBRAZE study in Q2, with the IND filing for SRK-439 planned for Q3 [8][35]. Market Data and Key Metrics Changes - Approximately two-thirds of the 10,000 individuals living with SMA in the U.S. and 35,000 globally have received SMA-targeted therapy, indicating a significant market opportunity for apitegromab as a muscle-targeted therapy [14][15]. - The current market for GLP-1 receptor agonists is projected to generate over $100 billion in sales, highlighting the potential impact of Scholar Rock's cardiometabolic programs [22]. Company Strategy and Development Direction - Scholar Rock aims to establish a neuromuscular franchise starting with SMA, with plans to expand treatment to other neuromuscular disorders [37]. - The company is focused on building a strong commercial team and infrastructure to support the launch of apitegromab, including outreach to payers and scaling the customer-facing team [19][20]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of apitegromab to transform the standard of care for SMA, emphasizing the importance of addressing progressive muscle weakness [12][15]. - The company is optimistic about the EMBRAZE study and the potential for SRK-439 to enhance weight management therapies by preserving lean muscle mass [35][36]. Other Important Information - Scholar Rock has initiated a disease education campaign called "Life Takes Muscle" to raise awareness about the impacts of muscle weakness in SMA [16][17]. - The company is preparing to offer home infusion services at launch to enhance patient support [19]. Q&A Session Summary Question: Discussion on SAPPHIRE data presentation - Management highlighted the importance of presenting additional data points at the MDA conference, focusing on the consistency and impact across age groups and endpoints [42][43]. Question: Insights on obesity and lean mass preservation - Management clarified that a 20% to 40% improvement in lean mass is considered clinically meaningful, and they are looking for trends in the EMBRAZE study to inform the development of SRK-439 [41][47]. Question: Exploration of additional neuromuscular indications - Management is actively engaging experts to explore adjacent neuromuscular indications and is assessing the unmet need and technical success probability for potential studies [60][63]. Question: Implications of FDA guidance on obesity trials - Management noted that the FDA's guidance reinforces the importance of assessing lean mass loss and that they will continue to follow this closely while developing SRK-439 [69][71]. Question: Comparison of SRK-439 and apitegromab - Management expressed confidence in the profile of SRK-439, indicating it may have greater affinity for the target and could potentially offer advantages in dosing and efficacy [80][82]. Question: Initial payer discussions and pricing strategy - Payers have shown receptiveness to Scholar Rock's innovation, and management expects access to be reflective of the value apitegromab brings to SMA treatment [85][86]. Pricing details are not yet disclosed but are expected to align with other SMA treatments [89].

Incyte (INCY) Update / Briefing September 14, 2024 01:00 PM ET Speaker0 Greetings. Welcome to Incyte Investor Event at ESMO twenty twenty four. At this time, all participants are in a listen only mode. As a reminder, this conference is being recorded. I would now like to turn the conference over to Pablo Carreoni, Head of Research and Development. Thank you. You may begin. Speaker1 Thank you. Thank you everyone for coming. Thank you everyone who's joining us online for the webcast. We're very excited about ...