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Belite Bio Announces China NMPA Agrees to New Drug Application with Priority Review based on Interim Analysis Results for the Treatment of Stargardt Disease with Tinlarebant

Belite Bio Announces China NMPA Agrees to New Drug Application with Priority Review based on Interim Analysis Results for the Treatment of Stargardt Disease with Tinlarebant

✨ Onyx Summary Belite Bio said China’s NMPA has accepted, with priority review, its NDA for Tinlarebant in Stargardt disease based on interim Phase 3 DRAGON results showing statistical significance on the primary endpoint. If approved, Tinlarebant would become the first therapy for Stargardt; Belite plans to report final topline

SpyGlass Pharma’s Novel BIM-IOL System Demonstrated a Statistically Significant Reduction in Mean IOP at 24 Months

SpyGlass Pharma’s Novel BIM-IOL System Demonstrated a Statistically Significant Reduction in Mean IOP at 24 Months

✨ Onyx Summary SpyGlass Pharma reported 24-month interim results from its first-in-human trial of the Bimatoprost Drug Pad-IOL System (BIM-IOL System), showing a 42% mean reduction in intraocular pressure and elimination of all topical medications in patients with open-angle glaucoma or ocular hypertension. The device demonstrated durable efficacy, safety, and improved

Alzheon Announces Peer-Reviewed Scientific Publication of Results from Pivotal APOLLOE4 Phase 3 Trial of Oral Valiltramiprosate/ALZ-801 in APOE4/4 Homozygous Individuals with Early Alzheimer’s Disease

Alzheon Announces Peer-Reviewed Scientific Publication of Results from Pivotal APOLLOE4 Phase 3 Trial of Oral Valiltramiprosate/ALZ-801 in APOE4/4 Homozygous Individuals with Early Alzheimer’s Disease

✨ Onyx Summary Alzheon announced the peer-reviewed publication of results from its pivotal Phase 3 APOLLOE4 trial evaluating oral valiltramiprosate (ALZ-801) in APOE4/4 homozygous individuals with early Alzheimer’s disease, showing cognitive and functional benefits in patients at the mild cognitive impairment stage, along with neuroprotective effects on brain atrophy

Piramal Pharma Solutions and NewAmsterdam Pharma Invest in Dedicated Suite to Enhance Oral Solid Dosage Production Capabilities at Piramal’s Sellersville, Pennsylvania Site

Piramal Pharma Solutions and NewAmsterdam Pharma Invest in Dedicated Suite to Enhance Oral Solid Dosage Production Capabilities at Piramal’s Sellersville, Pennsylvania Site

✨ Onyx Summary Piramal Pharma Solutions and NewAmsterdam Pharma announced a multi-million-dollar investment to establish a dedicated oral solid dosage suite at Piramal’s Sellersville, Pennsylvania site, enhancing production capacity for NewAmsterdam’s fixed-dose combination of obicetrapib and ezetimibe. The initiative expands Piramal’s integrated CDMO network, supports future commercial demand

Lexicon Pharmaceuticals Presents Additional Pilavapadin Data in Diabetic Peripheral Neuropathic Pain at 19th Annual Pain Therapeutics Summit

Lexicon Pharmaceuticals Presents Additional Pilavapadin Data in Diabetic Peripheral Neuropathic Pain at 19th Annual Pain Therapeutics Summit

✨ Onyx Summary Lexicon Pharmaceuticals presented new Phase 2 analyses for pilavapadin (LX9211) at the 19th Annual Pain Therapeutics Summit, confirming the 10 mg once-daily dose as the optimal candidate for Phase 3 development in diabetic peripheral neuropathic pain (DPNP). The findings—demonstrating validated biological activity, clinically meaningful pain reduction, and

Affinia Therapeutics to Participate in Upcoming Investor Conferences

Affinia Therapeutics to Participate in Upcoming Investor Conferences

✨ Onyx Summary Affinia Therapeutics CEO Rick Modi will participate in a panel on in vivo gene therapies for genetic cardiac diseases at Chardan’s 9th Annual Genetic Medicines Conference on Oct. 21 and hold one-on-one investor meetings at the Jefferies Global Healthcare Conference in London on Nov. 17–20. The

Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission of KRESLADI™ for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission of KRESLADI™ for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)

✨ Onyx Summary Rocket Pharmaceuticals announced that the U.S. FDA has accepted its resubmitted BLA for KRESLADI™ (marnetegragene autotemcel; marne-cel), an investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I), setting a PDUFA date of March 28, 2026. The filing is supported by Phase 1/2 data showing 100% survival

Verily, UCHealth, University of Colorado Anschutz, and RefinedScience launch strategic collaboration for AI-powered research and care transformation

Verily, UCHealth, University of Colorado Anschutz, and RefinedScience launch strategic collaboration for AI-powered research and care transformation

✨ Onyx Summary Verily has entered a multi-year strategic collaboration with UCHealth, the University of Colorado Anschutz, and RefinedScience to advance precision medicine through AI-driven data integration and analytics on Verily’s healthcare technology platform. The partnership will create AI-ready biomedical data pipelines, develop novel models across major therapeutic areas, and

Geron Corporation Announces Executive Leadership Transitions and Appointments

Geron Corporation Announces Executive Leadership Transitions and Appointments

✨ Onyx Summary Geron Corporation announced multiple leadership changes to align with its commercial growth strategy, including the appointment of Ahmed ElNawawi as EVP and Chief Commercial Officer, alongside new senior hires in technical operations, investor relations, and portfolio management. These appointments strengthen Geron’s leadership capabilities as it drives commercialization

Seer Highlights How Proteomics Complements Genomics to Advance Precision Medicine at American Society of Human Genetics (ASHG) 2025

Seer Highlights How Proteomics Complements Genomics to Advance Precision Medicine at American Society of Human Genetics (ASHG) 2025

✨ Onyx Summary Seer, Inc. announced its participation at the 2025 American Society of Human Genetics Annual Meeting, where it will showcase how its Proteograph® Product Suite enables integration of proteomics and genomics to advance precision medicine. The company will host a featured CoLab session and support multiple scientific presentations demonstrating

Rocket Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Rocket Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

✨ Onyx Summary Rocket Pharmaceuticals granted inducement equity awards to newly appointed Chief Medical Officer Dr. Syed Rizvi, consisting of stock options and restricted stock units totaling approximately 729,765 shares. The awards, approved under Nasdaq’s inducement grant rule, align long-term incentives with Rocket’s strategic growth as it advances

Aspect Biosystems to Present at 2025 Cell & Gene Meeting on the Mesa

Aspect Biosystems to Present at 2025 Cell & Gene Meeting on the Mesa

✨ Onyx Summary Aspect Biosystems Chief Scientific Officer Sam Wadsworth will present at the 2025 Cell & Gene Meeting on the Mesa on October 7 in Phoenix, highlighting the company’s advancements in regenerative medicine. The annual conference, organized by the Alliance for Regenerative Medicine, gathers global leaders in cell and