SALT LAKE CITY, June 30, 2025 (GLOBE NEWSWIRE) -- Clene, Inc. (Nasdaq: CLNN) and its subsidiary, Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company dedicated to advancing therapies for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), today provided a regulatory update following a Type C meeting with the U.S. Food and Drug Administration (FDA), and announced two additional meetings scheduled with the FDA in the 3rd quarter of 2025. In th ...

Core Insights - Neurocrine Biosciences announced significant reductions in cognitive and motor-related burdens in adults with Huntington's disease chorea treated with INGREZZA (valbenazine) capsules, based on new patient-reported post-hoc data from the KINECT-HD study [1][4] Group 1: Study Findings - The KINECT-HD study is the first clinical trial to show measurable changes in cognitive and motor disease burden with a vesicular monoamine transporter 2 inhibitor in Huntington's disease patients [1][2] - In the 12-week Phase 3 KINECT-HD clinical trial, INGREZZA showed a significant reduction in chorea severity, with a placebo-adjusted mean reduction in the Total Maximal Chorea (TMC) score of 3.2 units (p<0.0001) [11] - Patient-reported analyses indicated greater reductions in cognitive and motor-related disease burden for INGREZZA compared to placebo across various domains, including memory loss and mobility limitations [3][4] Group 2: Cognitive and Motor-Related Improvements - Statistically significant improvements were observed in cognition-related items, such as memory loss (-0.9 for INGREZZA vs -0.2 for placebo) and decision-making abilities (-1.0 for INGREZZA vs -0.3 for placebo) [3] - For motor-related items, INGREZZA demonstrated larger reductions in scores compared to placebo, including abnormal movements (-1.5 for INGREZZA vs -0.8 for placebo) and impaired coordination (-1.0 for INGREZZA vs -0.4 for placebo) [3][5] Group 3: Background on Huntington's Disease - Huntington's disease is a hereditary progressive neurodegenerative disorder affecting approximately 41,000 adults in the U.S., characterized by motor, cognitive, and psychiatric symptoms [7] - Chorea, a common symptom of Huntington's disease, involves irregular and unpredictable movements that can interfere with motor coordination and daily activities [7] Group 4: About INGREZZA - INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for treating chorea associated with Huntington's disease and tardive dyskinesia [14][15] - The drug is designed for once-daily administration without the need for titration, making it convenient for patients [16]

Corporate Presentation May 2025 1 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Any statement describing Acumen's goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Words such as "believes," "expects," "anticipates," "could," "would," "seeks," "aims," "plans," "potential," "will" and similar expres ...

Core Insights - Ocular Therapeutix has launched a new corporate branding and website, marking a significant transformation towards becoming a retina-focused company [1][8] - The new branding reflects the company's innovative vision and commitment to redefining the retina experience, particularly in the treatment of retinal diseases [3][6] Company Overview - Ocular Therapeutix, Inc. is a biopharmaceutical company dedicated to redefining the retina experience, with a focus on innovative treatments for retinal diseases [9] - The investigational therapy AXPAXLI™ (OTX-TKI) is an axitinib hydrogel designed for intravitreal injection, aimed at improving treatment durability and long-term outcomes for patients with wet age-related macular degeneration (wet AMD) [7][9] Market Context - Wet AMD affects approximately 1.8 million people in the U.S., and current treatment options often require up to 12 injections per year, leading to patient burden and potential treatment discontinuation [6] - The company aims to disrupt existing treatment paradigms for retinal diseases, addressing the high burden associated with current pulsatile treatments [6][3] Product Pipeline - Ocular's pipeline includes AXPAXLI™, currently in Phase 3 clinical trials for wet AMD, and DEXTENZA, an FDA-approved corticosteroid for ocular inflammation and pain [10] - The investigational product OTX-TIC is in Phase 2 clinical trials for open-angle glaucoma or ocular hypertension, leveraging the company's proprietary ELUTYX™ technology [10]

June 2025 NasdaqGS: ZVRA 1 A Rare Approach to Therapeutics Cautionary Note Regarding Forward-Looking Statements Presentation may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements relate to future events or our future financial performance. We generally identify forward-looking statements by terminology such as "may," "will," "would," "should," "expects," "plans," "anticipates," "could," "intends," "target," "projects," " ...

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]

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 ...

June 24, 2025 7:00 AM EDT | Source: InMed Pharmaceuticals Vancouver, British Columbia--(Newsfile Corp. - June 24, 2025) - InMed Pharmaceuticals Inc. (NASDAQ: INM) ("InMed" or the "Company"), a pharmaceutical company focused on developing a pipeline of proprietary small molecule drug candidates for diseases with high unmet medical needs, today announced new preclinical data demonstrating that INM-901 significantly reduces inflammation in ex vivo models of neuroinflammation, further supporting its potential a ...

Core Insights - Cumberland Pharmaceuticals Inc. presented positive results from the Phase 2 FIGHT DMD trial for ifetroban, a novel oral therapy targeting Duchenne muscular dystrophy (DMD) heart disease, at the PPMD conference, indicating its potential to protect the heart and reduce cardiac damage in DMD patients [1][2][8] Group 1: Trial Results - The 12-month Phase 2 FIGHT DMD trial demonstrated a significant 5.4% improvement in left ventricular ejection fraction (LVEF) in patients treated with high-dose ifetroban compared to a control group [4] - High-dose ifetroban treatment was associated with reduced blood levels of cardiac damage markers (NT-proBNP and cardiac troponin I), while these markers increased in placebo-treated patients, suggesting ifetroban's potential to prevent ongoing cardiac injury [5] - All patients who completed the study opted to continue with the open-label extension, indicating confidence in the treatment [7] Group 2: Pharmacokinetics and Tolerability - The study revealed that DMD patients receiving higher doses of ifetroban achieved similar plasma levels to typical adults without evidence of drug accumulation, supporting the 300 mg daily dosing used in the high-dose group [6] - Ifetroban was well-tolerated with an acceptable pharmacokinetic profile in patients with DMD, despite the higher dosing requirements [6] Group 3: Market Potential and Regulatory Pathway - Ifetroban is positioned to address the leading cause of death in DMD patients, with no approved treatment specifically targeting DMD heart disease, highlighting a critical unmet medical need [9][10] - The company plans to analyze long-term treatment results and engage in discussions with the FDA regarding the regulatory pathway forward based on the trial's encouraging results [10]

ArriVent BioPharma (AVBP) Update / Briefing June 23, 2025 08:00 AM ET Speaker0 Good morning and welcome to the Forte Biosciences Investor Webcast. At this time, all attendees are in a listen only mode. A Q and A session will follow the formal presentations. As a reminder, this call is being recorded and a replay will be made available on the Forte Biosciences Biosciences website following the conclusion of the event. I'd now like to turn the call over to Doctor. Paul Wagner, Chief Executive Officer of Forte ...