Therapy

Search documents
CareDx (CDNA) 2025 Conference Transcript
2025-06-05 15:30
CareDx (CDNA) 2025 Conference June 05, 2025 10:30 AM ET Speaker0 Good morning. I'm John Hanna, the president and CEO at CareDx. Thank you all for joining us today. This is our safe harbor statement. We may make forward looking statements during the presentation. Please refer to the safe harbor on our website. At CareDx we've built an experienced leadership team in the molecular diagnostics market to drive profitable growth. We have six fifty employees globally, 180 driving our commercial growth in the marke ...
FDA Grants Platform Technology Tag to Sarepta's Gene Therapy Vector
ZACKS· 2025-06-05 15:06
Key Takeaways FDA grants platform tech designation to SRPT's rAAVrh74 viral vector used in SRP-9003 The tag lets Sarepta reuse clinical and manufacturing data across other rAAVrh74-based therapies. SRP-9003 is in a phase III trial for LGMD2E/R4; data expected later this month.Sarepta Therapeutics (SRPT) announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, used in the investigational gene therapy SRP-9003. This therapy is being evaluated in a late-stage study for ...
Regenxbio (RGNX) Earnings Call Presentation
2025-06-05 14:15
RGX-202: AFFINITY DUCHENNE® New Phase I/II Interim Functional Data June 2025 Forward-looking statements This presentation includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," " ...
SELLAS Life Sciences Expands Scientific Advisory Board with Addition of Two World-Renowned Hematology and Oncology Experts
Globenewswire· 2025-06-05 12:45
New appointments of Drs. Philip C. Amrein and Alex Kentsis Validate Lead Galinpepimut-S and SLS009 Programs as Company Approaches Key Inflection Points This YearNEW YORK, June 05, 2025 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced the appointment of two distinguished oncology leaders to its Scientific Advisor ...
Cocrystal Pharma's Novel Norovirus Antiviral to be Presented at Department of Defense Medical Conference
GlobeNewswire News Room· 2025-06-05 12:00
Core Viewpoint - Cocrystal Pharma, Inc. is presenting its broad-spectrum protease inhibitor CDI-988 at the 2025 Military Health System Research Symposium, highlighting its potential as a prophylaxis and treatment for norovirus, which currently lacks approved antiviral agents or vaccines [1][2]. Company Overview - Cocrystal Pharma, Inc. is a clinical-stage biotechnology company focused on discovering and developing novel antiviral therapeutics targeting the replication processes of various viruses, including noroviruses, coronaviruses, and hepatitis C viruses [6]. - The company utilizes unique structure-based technologies and expertise in enzymology and medicinal chemistry to create first- and best-in-class antiviral drugs [6]. Product Development - CDI-988 is designed as a broad-spectrum inhibitor targeting a highly conserved region in the active site of 3CL viral proteases, representing a first-in-class oral treatment for noroviruses and coronaviruses [3]. - The company has completed a Phase 1 study evaluating the safety, tolerability, and pharmacokinetics of CDI-988 in healthy adults, which included a food effect cohort [3]. Technology Platform - Cocrystal's proprietary structure-based platform technology provides a three-dimensional structure of inhibitor complexes at near-atomic resolution, facilitating the identification of novel binding sites and rapid structural information processing [4]. - The goal of this technology is to develop antiviral therapies that are safe, well-tolerated, and effective against all viral subtypes, with a high barrier to viral resistance [4]. Industry Context - The Military Health System Research Symposium is an annual event that gathers military medical care providers, researchers, and industry representatives to discuss healthcare initiatives related to military infectious diseases and operational medicine [5].
NewAmsterdam Pharma to Host R&D Day on June 11, 2025
GlobeNewswire News Room· 2025-06-05 12:00
Company Overview - NewAmsterdam Pharma Company N.V. is a late-stage clinical biopharmaceutical company focused on developing oral, non-statin medicines for patients at risk of cardiovascular disease (CVD) with elevated low-density lipoprotein cholesterol (LDL-C) [1][5] - The company aims to address the unmet need for safe, well-tolerated, and convenient LDL-lowering therapies, particularly for patients who do not respond adequately to existing treatments [5] R&D Day Announcement - NewAmsterdam will host an R&D Day event for analysts and investors on June 11, 2025, at 9:00 a.m. ET in New York City [1] - The event will feature key members of the management team, including the CEO and Chief Scientific Officer [2] Obicetrapib Development - Obicetrapib is a novel, oral, low-dose CETP inhibitor being developed by NewAmsterdam to improve LDL-lowering treatment outcomes [4] - The company has conducted multiple Phase 2 and Phase 3 trials, demonstrating statistically significant LDL-lowering effects with a side effect profile similar to placebo [4] - The Phase 3 PREVAIL CVOT trial, which commenced in March 2022, has enrolled over 9,500 patients to assess obicetrapib's potential in reducing major adverse cardiovascular events (MACE) [4] Commercialization Rights - The commercialization rights for obicetrapib in Europe have been exclusively granted to the Menarini Group, a leading international pharmaceutical and diagnostics company [4]
REGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA FROM PHASE I/II AFFINITY DUCHENNE® TRIAL OF RGX-202
Prnewswire· 2025-06-05 11:05
Core Insights - REGENXBIO Inc. announced positive interim data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, showing consistent functional benefits and safety [1][2][3] Functional Data - RGX-202 participants at dose level 2 (2x10^14 GC/kg) showed significant functional improvements at both 9 and 12 months, exceeding natural history controls on key measures such as the North Star Ambulatory Assessment (NSAA) [4][5] - At 9 months, participants improved an average of 4 points from baseline on NSAA, and at 12 months, the improvement was 4.5 points from baseline, with a 6.8-point increase compared to natural history [5][6] Biomarker Data - Biomarker data indicated robust microdystrophin expression, with one participant aged 2 showing an expression level of 118.6% compared to control [8][10] - The primary endpoint for the pivotal phase is the proportion of participants with microdystrophin expression ≥10% at Week 12 [8] Safety and Tolerability - RGX-202 demonstrated a favorable safety profile with no serious adverse events reported, and common drug-related adverse events included nausea, vomiting, and fatigue [11][12] - The proactive immune modulation regimen and high product purity levels contributed to the positive safety outcomes [11] Pivotal Trial and Future Plans - REGENXBIO is enrolling participants for the pivotal portion of the AFFINITY DUCHENNE trial, aiming to support a Biologics License Application (BLA) submission under accelerated approval by mid-2026 [14][15] - The company plans to share top-line data in the first half of 2026, including biomarker, functional, and safety data [15] About RGX-202 - RGX-202 is designed to improve function and outcomes in Duchenne muscular dystrophy, utilizing a differentiated microdystrophin construct that encodes key regions of dystrophin [17][18] - The therapy aims to support targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV® AAV8 vector [18] About Duchenne Muscular Dystrophy - Duchenne muscular dystrophy is a severe, progressive muscle disease affecting 1 in 3,500 to 5,000 boys born each year, caused by mutations in the dystrophin gene [19] About REGENXBIO Inc. - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline including RGX-202 for Duchenne and other investigational therapies for rare diseases [20]
Ocugen, Inc. Announces Signing of Binding Term Sheet for the License of OCU400 Modifier Gene Therapy for Retinitis Pigmentosa in Korea
Globenewswire· 2025-06-05 11:02
Core Viewpoint - Ocugen, Inc. has signed a binding term sheet to negotiate a licensing agreement for exclusive rights to its gene therapy OCU400 in Korea, aimed at treating retinitis pigmentosa (RP) [1][4]. Group 1: Licensing Agreement Details - The licensing agreement will provide Ocugen with upfront fees and near-term development milestones totaling up to $11 million [2][8]. - Ocugen will receive sales milestones of $1 million for every $15 million in net sales in Korea, along with a royalty of 25% on net sales generated by its partner [2][8]. - The company will manufacture the commercial supply of OCU400 under a supply agreement [2]. Group 2: Market Opportunity - There are approximately 15,000 individuals in South Korea affected by RP, presenting a significant market opportunity for the partner to lead in gene therapy [3]. - The regional licensing strategy aligns with Ocugen's goal to partner with established companies to maximize patient reach while generating returns for shareholders [4]. Group 3: Development Timeline - Ocugen is advancing OCU400 through Phase 3 clinical development, with a target for filing a Biologics License Application by mid-2026 [5].
Innovent's IBI363 (PD-1/IL-2α-bias Bispecific Antibody Fusion Protein) Receives Second NMPA Breakthrough Therapy Designation for Immuno-resistant Squamous Non-Small Cell Lung Cancer
Prnewswire· 2025-06-05 00:23
Core Insights - Innovent Biologics has received a second Breakthrough Therapy Designation (BTD) from China's NMPA for IBI363, a bispecific antibody fusion protein targeting squamous non-small cell lung cancer (sqNSCLC) [1][3] - IBI363 has also received Fast Track Designations (FTDs) from the U.S. FDA for sqNSCLC and melanoma, highlighting its potential in treating immunotherapy-resistant cancers [1][8] - The drug aims to address unmet clinical needs in patients who have progressed after anti-PD-(L)1 immunotherapy and platinum-based chemotherapy [1][4] Company Overview - Innovent Biologics is a leading biopharmaceutical company founded in 2011, focusing on developing high-quality medicines for various diseases, including oncology and autoimmune disorders [9] - The company has launched 15 products and has multiple assets in various stages of clinical trials, including 3 new drug applications under regulatory review [9] - Innovent collaborates with over 30 global healthcare companies, enhancing its research and development capabilities [9] Clinical Development - IBI363 has shown promising results in Phase 1 clinical studies, demonstrating manageable safety and encouraging efficacy in immunotherapy-resistant patients [2][6] - The drug's dual mechanism of action combines PD-1 blockade and IL-2-driven T-cell expansion, potentially reshaping the tumor microenvironment [3][5] - Innovent is accelerating global development for IBI363, with ongoing clinical studies in China, the U.S., and Australia targeting various tumor types [7]
Innovent and HUTCHMED Jointly Announce NDA Acceptance in China for Sintilimab Combination with Fruquintinib for the Treatment of Advanced Renal Cell Carcinoma
Prnewswire· 2025-06-05 00:00
SAN FRANCISCO and SUZHOU, China, June 4, 2025 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, cardiovascular and metabolic, autoimmune, ophthalmology and other major diseases, and HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM:HCM; HKEX:13), today jointly announce that the New Drug Application (NDA) for the combination of sintilimab and fruquint ...