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Xenon Pharmaceuticals Halts Psychiatry Trials Due to Psychosis Events, Submits Epilepsy Drug for FDA Review

Xenon Pharmaceuticals Halts Psychiatry Trials Due to Psychosis Events, Submits Epilepsy Drug for FDA Review

Xenon Pharmaceuticals Halts Psychiatry Trials Due to Psychosis Events, Submits Epilepsy Drug for FDA Review
ReutersReuters
Experts urge FDA to set clear safety and access standards for psychedelic drugs (September 14)

Experts urge FDA to set clear safety and access standards for psychedelic drugs (September 14)

(Corrects quote attribution in paragraph 8 of September 14 story from Lynn Watkins, a U.S.
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Sling Therapeutics Secures $123 Million for Thyroid Eye Disease Drug Trial

Sling Therapeutics Secures $123 Million for Thyroid Eye Disease Drug Trial

Sling Therapeutics Secures $123 Million for Thyroid Eye Disease Drug Trial
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MaaT Pharma Shifts Focus to U.S. Clinical Trial for MaaT013 Following Negative European Regulatory Opinion

MaaT Pharma Shifts Focus to U.S. Clinical Trial for MaaT013 Following Negative European Regulatory Opinion

MaaT Pharma, a clinical-stage biotechnology company, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) maintained its negative opinion regarding the Conditional Marketing Authorization (CMA) application for MaaT013 (Xervyteg®). This decision, following a re-examination, concerns the treatment of acute Graft-versus-Host Disease (aGvHD) in adult patients with gastrointestinal involvement refractory to prior lines of therapy. The CHMP's view is that the available clinical data, based on a single-arm trial, does not sufficiently characterize the benefit-risk profile of MaaT013. Consequently, MaaT Pharma will now prioritize advancing its U.S. development plan for MaaT013, focusing on a randomized controlled Phase 3 trial named PHOENIX. Feedback from a Type C interaction with the Food and Drug Administration (FDA) supports the advancement of PHOENIX as a registrational Phase 3 trial, with potential first patient enrollment in the first half of 2027, c...
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Stratified Medicine Advances with AI and Genomic Technologies for Personalized Treatment

Stratified Medicine Advances with AI and Genomic Technologies for Personalized Treatment

Stratified medicine, also known as precision medicine, is evolving rapidly through the integration of artificial intelligence (AI) and genomic technologies. This approach focuses on customizing medical treatment for individual patients by utilizing biomarkers to identify specific subgroups within a diagnosis. The advancements allow for more targeted and effective therapies. AI and genomic technologies are enhancing the analysis of complex multimodal data, which includes imaging, physiological signals, and electronic health records. This comprehensive data analysis supports earlier diagnosis, more refined phenotyping, and improved risk stratification across various medical conditions. High-throughput pluripotent stem cell models combined with AI modeling are accelerating the development of precision medicine, particularly in fields such as oncology, neurodevelopmental and neurodegenerative disorders, and metabolic diseases. This shift moves away from a one-size-fits-all medical approach towards treatments t...
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Psychiatry Lags in Biomarker-Guided Treatment, ACNP Paper Proposes Roadmap for Precision Medicine

Psychiatry Lags in Biomarker-Guided Treatment, ACNP Paper Proposes Roadmap for Precision Medicine

A new position paper from the American College of Neuropsychopharmacology (ACNP), co-led by UCLA Health, highlights a significant gap in psychiatric treatment compared to other medical fields like cancer and Alzheimer's disease. Unlike these areas, psychiatric clinical trials predominantly rely on symptom checklists rather than objective biological signs (biomarkers) to enroll patients. This approach makes it difficult to identify specific patient subgroups and hinders the development of targeted treatments. The paper, published in NPP – Digital Psychiatry and Neuroscience, proposes a roadmap to integrate biomarkers into psychiatry, aiming to personalize treatment and improve outcomes. The authors, including university researchers, pharmaceutical industry representatives, and scientists from the National Institute of Mental Health and the FDA's Office of Neuroscience, suggest using measurable signals from blood, brain activity, or wearable devices to identify patient subgroups more precisely and design mor...
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Eli Lilly Receives FDA Approval for Combination Breast Cancer Therapy

Eli Lilly Receives FDA Approval for Combination Breast Cancer Therapy

Eli Lilly's oral selective estrogen receptor degrader (SERD) imlunestrant (Inluriyo) has secured a second FDA approval, this time for use in combination with the CDK4/6 inhibitor abemaciclib (Verzenio), another drug from Lilly. This combination therapy is approved for ER-positive, HER2-negative breast cancer patients with ESR1 mutations whose disease has progressed after at least one line of endocrine therapy. The approval follows results from the phase 3 EMBER-3 trial, which demonstrated that the combination therapy doubled the median progression-free survival (PFS) to 11 months, compared to five and a half months for Inluriyo alone. According to Jacob Van Naarden, executive vice president and president of Lilly Oncology, this full approval expands the capabilities of Inluriyo in combination with Verzenio, offering a regimen with confirmed benefits that aligns with the clinical practice of changing therapy upon clinical progression. The all-oral combination targets two distinct drivers of tumor growth: on...
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Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics, Inc., a clinical-stage biotechnology company, has announced the enrollment of its 100th participant in the Phase 2/3 ALSTARS Trial (NCT07161999) for COYA 302. This investigational drug, a combination of proprietary low-dose IL-2 and CTLA-4 Ig, is being evaluated for the treatment of amyotrophic lateral sclerosis (ALS). The company anticipates completing the full enrollment of 120 participants in the coming weeks, with topline data expected in early Q2 2027. COYA 302 has received FDA Fast Track designation for ALS treatment. The ALSTARS Trial is a randomized, multi-center, double-blind, placebo-controlled study designed to assess the efficacy and safety of COYA 302. Participants are randomized to receive one of two dosing regimens or a placebo over a 24-week double-blind treatment phase, followed by a 24-week blinded active-treatment extension phase. The primary endpoint focuses on changes in disease severity as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised...
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Armata Pharmaceuticals Receives FDA Breakthrough Therapy Designation for AP-SA02 to Treat Complicated Bacteremia

Armata Pharmaceuticals Receives FDA Breakthrough Therapy Designation for AP-SA02 to Treat Complicated Bacteremia

Armata Pharmaceuticals, Inc., a biotechnology company focused on bacteriophage therapeutics, has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for its product AP-SA02. This intravenously administered multi-phage product is under development to treat complicated bacteremia caused by methicillin-sensitive or methicillin-resistant Staphylococcus aureus. The designation acknowledges the critical need for new treatments for S. aureus bacteremia, a condition with persistently poor patient outcomes. AP-SA02 has also received Fast Track and Qualified Infectious Disease Product (QIDP) designations. The Breakthrough Therapy designation is supported by results from the Phase 1b/2a diSArm study, which showed that when AP-SA02 was combined with the best available antibiotic therapy, it led to higher clinical cure rates compared to a placebo. Specifically, 100% of patients treated with AP-SA02 maintained a clinical response without relapse at day 28 post-treatment, in contr...
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Cell and Gene Therapy Industry Faces Scale-Up Challenges, Lifecycle Approach Proposed

Cell and Gene Therapy Industry Faces Scale-Up Challenges, Lifecycle Approach Proposed

Cell and Gene Therapy Industry Faces Scale-Up Challenges, Lifecycle Approach Proposed
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Thryv Therapeutics Publishes Evidence Supporting THRV-1268 for Heart Failure Treatment

Thryv Therapeutics Publishes Evidence Supporting THRV-1268 for Heart Failure Treatment

Thryv Therapeutics Inc. has announced the publication of a new study in JACC: Heart Failure, presenting human genetic, preclinical, and clinical evidence that supports the use of its lead SGK1 inhibitor, THRV-1268, for treating heart failure with reduced ejection fraction (HFrEF). The study highlights SGK1 as a genetically validated cardiometabolic therapeutic target. Key findings indicate that THRV-1268 maintained cardiac function and outperformed empagliflozin monotherapy in an animal model of heart failure. Additionally, Phase 1 studies demonstrated a favorable safety profile and statistically significant QTcF shortening in individuals with obesity, consistent with cardiac target engagement. These findings provide a strong rationale for advancing THRV-1268 into Phase 2a clinical development through the ASPIRE-HF study, which is expected to begin enrollment in Q4 2026.
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Circle Pharma Secures $92.5 Million Series E Financing to Advance Cancer Drug into Clinical Trials

Circle Pharma Secures $92.5 Million Series E Financing to Advance Cancer Drug into Clinical Trials

Circle Pharma Secures $92.5 Million Series E Financing to Advance Cancer Drug into Clinical Trials