ASCEND comprises two dosing regimens of certepetide evaluated in two separate study arms enrolled sequentially Positive signal in progression-free survival and objective response rate observed in certepetide-treated group compared to placebo-treated group Cohort B data corroborate Cohort A data indicating certepetide has a treatment effect and an attractive safety profile Full study data from both cohorts expected later this year BARCELONA, Spain, June 26, 2025 (GLOBE NEWSWIRE) -- The Australasian Gastro-In ...

MALVERN, Pa., June 26, 2025 (GLOBE NEWSWIRE) -- Annovis Bio, Inc. (NYSE: ANVS) ("Annovis" or the “Company”), a late-stage clinical drug platform company pioneering transformative therapies for neurodegenerative diseases such as Alzheimer's disease (AD) and Parkinson's disease (PD), today announced its senior management will attend the Alzheimer’s Association International Conference (AAIC), taking place July 27–31, 2025, in Toronto, Canada. The Company will present four scientific posters highlighting advan ...

Attractive safety profile demonstrated across all dosing cohorts and routes of administration (IV, SC, and IM); all reported adverse events (AEs) deemed unrelated or classified as mild to moderate and largely related to injection site and infusion reactions with no serious or severe adverse events observedFollowing a single dose, serum concentrations of VYD2311 remained high at six months with an observed half-life of the IM dose route having the longest duration at 76.0 (CI: 68.5 – 90.7) days Comprehensive ...

Core Insights - Viatris Inc. announced positive top-line results from the VEGA-3 Phase 3 trial for MR-141, demonstrating significant improvement in near visual acuity for patients with presbyopia without compromising distance vision [1][2] - The trial involved 545 patients and showed that MR-141 significantly outperformed placebo in achieving primary and secondary endpoints [5][6] - The company plans to submit an application to the U.S. FDA in the second half of 2025 [1][8] Company Overview - Viatris is a global healthcare company that combines generics and brand medications to address healthcare needs, supplying high-quality medicines to approximately 1 billion patients annually [7] - The company is headquartered in the U.S. with global centers in Pittsburgh, Shanghai, and Hyderabad [7] Industry Context - Presbyopia affects nearly 128 million people in the U.S., approximately 90% of adults over 45, and is projected to impact 2.1 billion people globally by 2030 [6] - The condition leads to significant economic losses, estimated between $11 billion and $25 billion due to uncorrected cases, highlighting the need for effective near-vision correction solutions [6]

Core Insights - Edgewise Therapeutics has reported positive results from its sevasemten program for Becker and Duchenne muscular dystrophies, demonstrating sustained disease stabilization and a clear path to potential registration as the first therapy for Becker [1][3]. Group 1: Becker Muscular Dystrophy - The MESA trial data showed that 99% of eligible participants (n=85) are enrolled, with significant improvements in North Star Ambulatory Assessment (NSAA) scores, indicating sustained disease stabilization over 18 months [2]. - Participants from the CANYON trial who transitioned to MESA exhibited an average NSAA score improvement of 0.8 points from baseline, while those on placebo showed a 0.2 point improvement after starting sevasemten [2]. - The FDA has provided a clear path for sevasemten registration, emphasizing the importance of NSAA as a clinically meaningful endpoint for traditional approval [3]. Group 2: Duchenne Muscular Dystrophy - Encouraging topline data from Phase 2 trials (LYNX and FOX) indicate that sevasemten is well-tolerated and shows potential for reducing functional decline at a target dose of 10 mg [4][6]. - The LYNX trial focuses on four- to nine-year-old participants, while the FOX trial involves six- to fourteen-year-old participants previously treated with gene therapy, both assessing safety and biomarkers of muscle damage [5][6]. - The company plans to meet with the FDA in Q4 2025 to discuss Phase 3 design, aiming to initiate the pivotal study in 2026 [7]. Group 3: Company Overview - Edgewise Therapeutics is a biopharmaceutical company focused on developing novel therapeutics for muscular dystrophies and cardiac conditions, with sevasemten being a first-in-class fast skeletal myosin inhibitor [9][12]. - The company has achieved significant regulatory milestones, including FDA Orphan Drug Designation and Fast Track designations for both Becker and Duchenne [9]. - Edgewise is dedicated to changing the lives of patients affected by serious muscle diseases, with a strong emphasis on innovative therapeutic solutions [12].

Core Insights - Vor Bio has secured exclusive global rights (excluding Greater China) to develop and commercialize telitacicept, a dual-target recombinant fusion protein for autoimmune diseases [1][6] - RemeGen has received an initial payment of $125 million, which includes a $45 million upfront payment and $80 million in warrants, along with potential milestones exceeding $4 billion and tiered royalties [1][5] - Jean-Paul Kress, MD, has been appointed as the new CEO and Chairman of Vor Bio, bringing extensive experience in clinical development and commercialization [3][4] Company Developments - Vor Bio is focused on advancing telitacicept through Phase 3 clinical development to address serious autoantibody-driven conditions globally [7] - RemeGen is conducting a global Phase 3 clinical trial for telitacicept, with initial results expected in the first half of 2027 [2][6] - The strategic out-licensing of telitacicept's ex-China rights is aimed at maximizing its clinical and commercial potential on a global scale [5] Product Information - Telitacicept targets key immune pathways by inhibiting BlyS (BAFF) and APRIL, which are critical for B cell survival, thereby reducing autoreactive B cells and autoantibody production [2][5] - In a Phase 3 trial in China for generalized myasthenia gravis, telitacicept showed a 4.8-point improvement in the MG-ADL scale compared to placebo at 24 weeks [5]

Summary of Outlook Therapeutics Conference Call Company Overview - **Company**: Outlook Therapeutics - **Ticker**: OTLK (trades on Nasdaq) [4] Recent Achievements - **Product**: Lightnova - **Regulatory Approvals**: Lightnova approved in the EU and UK; received positive recommendation from NICE for use in the UK [5][6] - **Launch**: Launched simultaneously in Germany and the UK in May 2025; first patients dosed in both countries [6] Market Landscape - **Industry**: Treatment of wet Age-related Macular Degeneration (AMD) - **Market Size**: The European market for wet AMD treatments is valued at approximately $3.6 billion annually, with 8 million anti-VEGF injections per year [9] - **Injection Distribution**: Germany accounts for 1.7 million injections, while the UK accounts for 1.3 million [10] Competitive Positioning - **Market Share**: Off-label bevacizumab holds a 33% market share in Europe, with Germany at 50% and the UK at 5% [11][12] - **Cost-Effectiveness**: Lightnova positioned as a cost-effective first-line treatment option for wet AMD [9][12] Pricing Strategy - **UK Pricing**: List price set at £470, the lowest among approved products; patient access scheme in place for competitive net pricing [16][18] - **Germany Pricing**: List price set at €751.61; negotiations for a post-launch price expected in early 2026 [20][19] Commercial Launch Strategy - **Market Access**: Focus on subnational access critical for success; rebate agreements in place covering 50% of the population in Germany, expected to increase to 90% by year-end [26] - **Team Experience**: Strong teams with extensive ophthalmology experience in both Germany and the UK [24] Physician Engagement - **Receptiveness**: Positive feedback from ophthalmologists regarding Lightnova; significant interest in an ophthalmic standard formulation of bevacizumab [13][14] - **Educational Efforts**: Focus on educating physicians about the ophthalmic formulation and clinical data supporting Lightnova [35][36] Future Expansion Plans - **Market Expansion**: Approved in 31 countries; focus on launching in Italy, Spain, France, Ireland, and the Nordics [44][47] - **Potential Partnerships**: Currently focused on maximizing Lightnova's potential independently, but open to partnerships if beneficial [48] FDA Approval Update - **BLA Submission**: BLA submitted to the FDA in February 2025; PDUFA goal date set for August 27, 2025 [50][51] - **Expectations**: Confident in the data provided to the FDA supporting approval for wet AMD treatment [75][76] Financial Outlook - **Liquidity**: Recent capital raised through a CMPO; sufficient funds to support operations through the PDUFA decision [58] - **Profit Margins**: Anticipated healthy profit margins, with a focus on minimizing dilution during financing [63] Conclusion - **Key Milestones**: Upcoming quarterly results expected in August, along with the FDA decision; preparations for further European launches ongoing [84][85]

Basel, June 25, 2025 – Novartis today announced that it has successfully completed its acquisition of Regulus Therapeutics Inc. (“Regulus”). With the completion of the acquisition, shares of common stock, par value $0.001 per share (the “Shares”), of Regulus, have ceased trading on the Nasdaq Stock Market LLC and Regulus is now an indirect wholly owned subsidiary of Novartis. “We are pleased to complete this transaction and take the next step in advancing clinical development for a potential first-in-class ...

Core Insights - Silo Pharma has entered into a non-binding letter of intent to form a 50:50 joint venture with Hoth Therapeutics to develop a treatment for obesity and metabolic diseases based on technology licensed from the U.S. Department of Veterans Affairs [1][3] - The joint venture aims to leverage a novel therapeutic platform centered on glial cell line-derived neurotrophic factor (GDNF), which has shown anti-obesity effects in preclinical models [2][4] - The obesity market is valued at approximately $16 billion, with the potential for significant impact on veterans and civilians suffering from metabolic disorders [3][4] Unmet Need - Over 40% of U.S. adults are affected by obesity, which is a leading risk factor for various health issues, including diabetes and cardiovascular disease [4] - Veterans face unique challenges related to obesity, including chronic inflammation and PTSD-related metabolic disruptions, highlighting the need for effective treatments [4] Strategic Alignment - The partnership will utilize the exclusive U.S. license from the Department of Veterans Affairs, ensuring comprehensive coverage for all fields of use [7] - The joint venture structure allows for equal equity and governance participation between Silo Pharma and Hoth Therapeutics [7] - The collaboration combines the clinical infrastructure of the VA, Hoth's regulatory expertise, and Silo's translational capabilities [8]

Core Insights - Soleno Therapeutics presented new data on VYKAT XR (diazoxide choline) extended-release tablets at the 2025 International Prader-Willi Syndrome Conference, highlighting its potential benefits for individuals with Prader-Willi Syndrome (PWS) [1][2] Group 1: Efficacy of VYKAT XR - VYKAT XR significantly reduces hyperphagia symptoms in PWS patients managed with strict food controls, showing statistically significant reductions in HQ-CT Total scores (p ≤ 0.0001) [5] - Participants in highly food-restricted environments benefited from VYKAT XR treatment similarly to those in less restricted environments, indicating broad applicability of the treatment [5] Group 2: Safety and Efficacy in Co-Morbid Conditions - 60% of study participants had evidence of pre-diabetes or diabetes (PD/DM) at baseline, with efficacy outcomes at Week 156 showing similar results for both PD/DM and normoglycemic groups (p<0.0001) [7] - Although a higher proportion of hyperglycemia-related adverse events were reported in the PD/DM group (42.7% vs 24.0%), these events were generally manageable, and treatment discontinuation rates were low (4.0% in PD/DM vs 6.0% in normoglycemic) [8][9] Group 3: Background on Prader-Willi Syndrome - Prader-Willi Syndrome is a rare genetic neurodevelopmental disorder affecting approximately 1 in every 15,000 live births, characterized by hyperphagia, which can lead to severe health complications [10][11] - Hyperphagia in PWS can result in significant mortality and long-term co-morbidities such as diabetes, obesity, and cardiovascular disease [12] Group 4: Product Information - VYKAT XR was approved by the FDA on March 26, 2025, and is indicated for the treatment of hyperphagia in adults and pediatric patients aged 4 years and older with PWS [13][18]