Month: September 2025

uniQure Announces Positive Topline Results from Pivotal Phase I/II Study of AMT-130 in Patients with Huntington’s Disease

uniQure in the NEWS On September 24, 2025, uniQure N.V. (QURE) - a gene therapy company advancing transformative therapies for patients with severe medical needs, announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease. The study met its prespecified primary . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Important: Two Press Releases in the Past 2 Days from Grail & Intellia Therapeutics

Important News from Grail Inc. and Intellia Therapeutics  Grail Inc Press Realease GRAL to Present New Galleri® Data from More Than 32,000 Participants Across the PATHFINDER 2, SYMPLIFY and REFLECTION Studies at ESMO Congress 2025 and EDCC First PATHFINDER 2 Results Accepted as a Late-Breaking Presentation at the European Society for Medical Oncology (ESMO) Congress 2025; Results to be Submitted to FDA as Part of …
VectorY Therapeutics and Shape Therapeutics, both private firms Announce Option and License Agreement to Advance Vectorized Antibodies for the treament of Neurodegenerative Diseases Using an AAV5-Derived CNS Capsid 

VectorY Therapeutics and Shape Therapeutics, both private firms Announce Option and License Agreement to Advance Vectorized Antibodies for the treament of Neurodegenerative Diseases Using an AAV5-Derived CNS Capsid 

VectorY Therapeutics in the NEWS  September 18, 2025 – VectorY Therapeutics, a biotechnology company developing innovative vectorized antibody therapies for the treatment of neurodegenerative diseases, and Shape Therapeutics Inc., a biotechnology company harnessing AI and RNA technologies to engineer next-generation genetic medicines, announced Today that they have entered into an option and license agreement. The Agreement The agreement grants VectorY an exclusive option to evaluate …

In Data Presented at the European Association for the Study of Diabetes and NEUROdiabetis Meetings, Lexicon Pharmaceuticals Product Pilavapadin Provided Meaningful Pain Reduction in Adults with Diabetic Peripheral Neuropathic Pain.   

Lexicon Pharmaceuticals in the NEWS Yesterday, September 17, 2025, Lexicon Pharmaceuticals (LXRX) announced the presentation of clinical data from the company’s Phase 2b PROGRESS study for its investigational, AAK1-inhibitor, pilavapadin, for the treatment of diabetic peripheral neuropathic pain (DPNP) at two medical meetings: “New Promise for Painful Diabetic Neuropathy: Results from recently finished clinical trials of Pilavapadin, an oral, non-opioid . . . This content …

Eli Lilly’s Oral GLP-1, Orforglipron, is Superior to Oral Semaglutide in Head-to-Head Trial

Eli Lilly Eli Lilly and Company (LLY) today September 17, announced positive topline results from ACHIEVE-3, Phase 3 clinical trial evaluating the safety and efficacy of orforglipron compared to oral semaglutide, administered according to approved label instructions in 1,698 adults with type 2 diabetes inadequately controlled with metformin. The 52-week trial compared orforglipron (12 mg and 36 mg) to oral . . . This content is …

Jazz Pharmaceuticals to Treat H3 K27M-Mutant Diffuse Midline Glioma

Jazz Pharmaceuticals On September 9, 2025, Jazz Pharmaceuticals plc (JAZZ) announced that Modeyso™ (dordaviprone) is recommended by the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) as a category 2A single-agent treatment option for pediatric and adult patients with recurrent or progressive diffuse high-grade glioma harboring an H3 K27M mutation. Modeyso was granted . . . This content is for paid subscribers. Please …

The FDA is Taking Concrete Steps to Reduce Opioid Misuse, Addiction and Overdose Strengthening Guidance on Their Appropriate Use

Xenon Pharmaceuticals A Company  Treating Chronic Pain Xenon Pharmaceuticals (XENE) - a neuroscience-focused biopharmaceutical company dedicated to drug discovery, development, and commercialization of life-changing therapeutics for patients in need, announced equity inducement grants to seven new non-officer employees consisting of an aggregate of 55,300 share options. Xenon’s lead molecule, azetukalner, azetukalner -  a novel, potent, selective Kv7 potassium channel opener in Phase . . . …

Alnylam Pharmaceuticals Announces Proposed Offering of $500 Million Convertible Senior Notes. The Stock’s Price is Soaring.  

Alnylam Pharmaceuticals Press Release Alnylam Pharmaceuticals, Inc. (ALNY), the leading RNAi therapeutics company, announced today that it has commenced a private offering of $500 million aggregate principal amount of convertible senior notes due 2028 (the “notes”) to persons reasonably believed to be qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the “Securities Act”). In connection with this offering …

Regeneron Pharmaceuticals Libtayo® Plus Chemotherapy Results at Five Years Reinforce Significant and Durable Improvements in Survival Outcomes for Advanced Non-Small Cell Lung Cancer

Regeneron Pharmaceuticals Announcement Late-breaking data from exploratory analyses at WCLC show Libtayo plus chemotherapy demonstrates a more than double five-year overall survival rate of 19.4%, compared to 8.8% with chemotherapy alone In a five-year analysis, consistent efficacy was observed across histologies with especially notable benefit in the squamous patient population where median overall survival was 22.3 months. Regeneron Pharmaceuticals, Inc. (REGN . . . This content …

Alkermes: Detailed Positive Results from Vibrance-1 Phase 2 Study of Alixorexton in Patients with Narcolepsy Type 1 at World Sleep Congress 2025

Alkermes in the NEWS Today, Alkermes Inc., (ALKS) announced detailed positive results from the Vibrance-1 phase 2 study evaluating alixorexton in patients with narcolepsy type 1 (NT1). Alixorexton, (formerly ALKS 2680), is a novel, investigational, oral, selective orexin 2 receptor (OX2R) agonist in phase 2 development as a once-daily treatment for NT1, narcolepsy type 2 . . . This content is for paid subscribers. Please …

Ionis Pharmaceuticals Product Olezarsen Significantly Reduces Triglycerides and Acute Pancreatitis Events in Landmark Pivotal Studies for People with Severe Hypertriglyceridemia

Ionis Pharmaceuticals in the NEWS Today, Sep. 2, 2025, Ionis Pharmaceuticals (IONS) announced positive topline results from the pivotal Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG). In the studies, olezarsen demonstrated a highly statistically significant placebo-adjusted mean reduction in fasting triglycerides of up to 72% and a highly statistically significant reduction in acute . . . This content …