Updated cash guidance provides runway into at least mid-2026 MDNA11 Phase 1/2 clinical trial on track for data readouts in second half of the year as a single agent and in combination with KEYTRUDA® at medical conferences and a planned KOL event MDNA11 continues to exhibit compelling deep and durable anti-tumor activity in difficult-to-treat solid tumors with best-in-class potential relative to competing IL-2 programs Results presented at the 2025 AACR showed response rates in the 30-50% range in various tu ...

Châtillon, France, June 25, 2025 DBV Technologies Announces First Subject Screened in COMFORT Toddlers Supplemental Safety Study in Peanut Allergic Toddlers 1 – 3 Years Old First subject screened at the Respiratory Medicine Research Institute of Michigan with Dr. Jeffrey Leflein acting as Principal InvestigatorAdditional sites, including Allergy and Asthma Center of Minnesota and Hamilton Allergy and Immunology Clinic of Ontario, Canada have been activated and are scheduling screenings DBV Technologies (Eu ...

Châtillon, France, June 25, 2025 DBV Technologies Announces First Subject Screened in COMFORT Toddlers Supplemental Safety Study in Peanut Allergic Toddlers 1 – 3 Years Old First subject screened at the Respiratory Medicine Research Institute of Michigan with Dr. Jeffrey Leflein acting as Principal InvestigatorAdditional sites, including Allergy and Asthma Center of Minnesota and Hamilton Allergy and Immunology Clinic of Ontario, Canada have been activated and are scheduling screenings DBV Technologies (Eu ...

Summary of Lyell Immunopharma (LYEL) Conference Call - June 25, 2025 Company Overview - **Company**: Lyell Immunopharma - **Focus**: Development of next-generation cell therapies for cancer, specifically targeting hematologic malignancies and solid tumors with CAR T cell therapies [4][5] Core Points and Arguments Product Development - **Lead Program**: Autologous CAR T cell therapy targeting CD19 and CD20, aimed at achieving more complete responses and longer remission periods [4][5] - **Clinical Trials**: Currently in pivotal clinical trials for relapsed/refractory large B cell lymphoma, with a focus on outpatient administration due to a favorable safety profile [6][19] Clinical Data - **Third-Line Setting**: - Presented data from 51 patients showing an 88% overall response rate and a 72% complete response rate, significantly higher than the approximately 50% seen with existing CD19 CAR therapies [15][16] - 71% of patients who achieved complete response remained in remission at six months, compared to about 40% for CD19 CARs [16][43] - **Second-Line Setting**: - First data presented showed a 91% overall response rate and a 70% complete response rate in patients with primary refractory disease [18][19] - The patient population included older patients with no upper age limit, broadening the inclusion criteria compared to competitors [18][65] Competitive Landscape - **Comparison with Competitors**: - Lyell's dual-targeting approach (CD19 and CD20) is positioned as a significant advancement over existing CD19-only therapies, which have limitations in patient response [7][26] - Notable competitors include Yescarta (Axi-cel) and Breyanzi (Liso-cel), both of which have shown similar response rates but with higher rates of neurotoxicity [21][23][31] - Lyell's safety profile shows lower rates of cytokine release syndrome (CRS) and neurotoxicity compared to competitors [31][32] Regulatory and Market Strategy - **Regulatory Designation**: Received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, indicating strong early data and facilitating more frequent interactions with the agency [50][56] - **Pivotal Trial Design**: A seamless phase one/two design for the pivotal trial, with plans to accelerate patient recruitment by adding more centers [51][52] - **Market Opportunity**: Targeting a significant patient population in both the second and third-line settings, with an estimated 30,000 new DLBCL cases annually in the U.S. [60] Additional Important Insights - **Patient Demographics**: The focus on older patients and those with primary refractory disease highlights the unmet need in the market and the potential for Lyell's therapies to address this gap [18][62] - **Intellectual Property**: Lyell holds a strong intellectual property position, with patents originating from UCLA, which may provide a competitive edge in the market [36][37] - **Future Outlook**: The company is optimistic about the potential for national priority review vouchers, which could expedite the approval process for their therapies [56][57] This summary encapsulates the key points discussed during the conference call, highlighting Lyell Immunopharma's strategic positioning, clinical advancements, and competitive landscape in the CAR T cell therapy market.

Key Takeaways CRSP is the first to market a CRISPR/Cas9 therapy, with Casgevy approved for SCD and TDT in multiple regions CRSP's pipeline spans ex vivo and in vivo therapies, including early success with CTX310. NTLA focuses on late-stage in vivo programs but faces setbacks, including a liver safety concern with nex-z.CRISPR Therapeutics (CRSP) and Intellia Therapeutics (NTLA) are leading developers of therapies that utilize the Nobel Prize-winning CRISPR/Cas9 gene editing technology. While CRSP is the f ...

Creating transformative gene-based medicines for serious diseases Corporate Overview Q2 2025 CRISPR THERAPEUTICS – CONFIDENTIAL © 2025 CRISPR Therapeutics | 11 Forward-Looking Statements Statements contained in this presentation and other related materials regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ material ...

Scope of Agreement with Cambrex to Provide Ondansetron HCL Drug Substance Under FDA Approved Drug Master File Scope Of Agreement With Thermo Fisher to Provide Manufacturing Services Including Demonstration, Clinical, Registration And Validation Batches of AD04 for US Clinical Trials And NDA Submission GLEN ALLEN, Va., June 25, 2025 (GLOBE NEWSWIRE) -- Adial Pharmaceuticals, Inc. (NASDAQ: ADIL) ("Adial" or the "Company"), a clinical-stage biopharmaceutical company focused on developing therapies for the trea ...

Corvus co-founded Angel Pharma to develop its pipeline in greater China. Angel Pharma licensed the rights from Corvus to develop, manufacture and commercialize soquelitinib in greater China and is responsible for all expenses related to its development in China. "Atopic dermatitis affects patients worldwide, including a significant number in China where treatment with biologics and other systemic therapies is growing in use," said Richard A. Miller, M.D., co-founder, president and chief executive officer of ...

- End-of-Phase 2 Meeting with FDA for Mild-to-Moderate Alzheimer’s Disease Scheduled - - Applications for IND and Breakthrough Status Filed for Dementia with Lewy Bodies - PURCHASE, N.Y., June 25, 2025 (GLOBE NEWSWIRE) -- Cognition Therapeutics, Inc., (the Company or Cognition) (NASDAQ: CGTX), a clinical stage company developing drugs that treat neurodegenerative disorders, announced that the company will conduct an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) on July 9, 2025. Dur ...

Core Insights - Biogen Inc. announced topline results from the Phase 1 study of salanersen, an antisense oligonucleotide for spinal muscular atrophy (SMA), showing potential for high efficacy and once yearly dosing [1][5] - The Phase 1 study demonstrated substantial slowing of neurodegeneration and clinically meaningful improvements in motor function in children previously treated with gene therapy [2][5] - Biogen is engaging with global health authorities to advance salanersen into registrational studies based on encouraging Phase 1 data [4][5] Study Details - The Phase 1 study included two parts: a randomized placebo-controlled segment in healthy adults and an open-label segment in pediatric SMA participants who had previously received ZOLGENSMA [2][4] - Interim results from the open-label segment (n=24) indicated that both 40 mg and 80 mg doses of salanersen were well-tolerated, with a mean reduction in neurofilament light chain (NfL) of 70% at 6 months [2][4] - Exploratory data showed that half of the participants (4 out of 8) achieved new WHO motor milestones after receiving salanersen [3][4] Safety Profile - The safety profile of salanersen was generally well-tolerated, with most adverse events being mild to moderate, including pyrexia and upper respiratory tract infections [4][5] - The study's findings suggest that salanersen could address critical unmet needs in SMA treatment, building on Biogen's extensive experience in the field [3][5] Industry Context - SMA is a rare genetic neuromuscular disease affecting approximately 1 in 10,000 live births, characterized by progressive muscle atrophy and weakness [7][8] - SPINRAZA, another treatment for SMA, has been approved in over 71 countries and has treated more than 14,000 individuals worldwide, establishing a foundation of care in SMA [9][10]