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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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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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Insilico Medicine Utilizes AI to Discover Potential Anti-Aging Drug Rentosertib

Insilico Medicine Utilizes AI to Discover Potential Anti-Aging Drug Rentosertib

Insilico Medicine, an AI-driven drug discovery company, has announced that its drug candidate, rentosertib, initially developed for idiopathic pulmonary fibrosis (IPF), shows potential in slowing the aging process. The drug's molecular structure was assisted by artificial intelligence. New data from a clinical trial, published in Nature Biotechnology, indicates that rentosertib reduces biological markers of age as measured by six different 'aging clocks'—AI technology designed to predict morbidity and mortality. While the clinical trial involved only 42 patients with chronic lung disease, the findings suggest a broader application beyond its initial purpose. This development highlights the growing role of AI in accelerating drug discovery and potentially identifying novel anti-aging treatments, a field that has historically seen many failures in developing new drugs.
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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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Elvinix Licenses Peptide Therapy from Zealand Pharma to Restore Insulin Production in Type 1 Diabetes

Elvinix Licenses Peptide Therapy from Zealand Pharma to Restore Insulin Production in Type 1 Diabetes

Elvinix Inc., a biopharmaceutical company based in Dover, Delaware, has secured an exclusive license agreement with Zealand Pharma A/S to develop and commercialize Zealand’s GLP-1/gastrin dual-agonist peptide portfolio. This agreement grants Elvinix the rights to advance a novel peptide therapy aimed at regenerating and protecting insulin-producing beta cells in patients with type 1 diabetes (T1D). The therapy is designed to be disease-modifying, with the potential to restore insulin secretion, thereby reducing the risk of hypoglycemia and diabetic complications, and lessening the burden of insulin therapy. Elvinix was co-founded by Dr. Alex Rabinovitch, Dr. Jay Skyler, and Dr. Alexander Fleming, experts in diabetes and metabolic medicine. The company is currently in preclinical development and plans to move its lead candidate toward clinical studies, leveraging an extensive package of preclinical data obtained through the licensing agreement.
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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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Preclinical Study Advances CRISPR-Cas9 Gene Editing for Wiskott-Aldrich Syndrome Towards Clinical Use

Preclinical Study Advances CRISPR-Cas9 Gene Editing for Wiskott-Aldrich Syndrome Towards Clinical Use

A comprehensive preclinical study has been published detailing the in-depth characterization of stem cell potency and genotoxicity for clinical-scale ex vivo CRISPR-Cas9 gene editing. This research, using Wiskott-Aldrich Syndrome as a model, established an integrated strategy for editing human hematopoietic stem cells (HSCs) and assessing genotoxicity. The study's findings have been instrumental in securing Orphan Drug Designation from both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). This designation is a critical step in accelerating the development and review of therapies for rare diseases. The research focuses on the practical aspects of moving gene editing from laboratory settings to clinical application, emphasizing the need to demonstrate not only the effectiveness of the edit but also the potency, genotoxicity, and consistency of the process at a clinical scale. The project was driven by Asma Naseem, with significant contributions from collaborators including...
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Arrowhead Pharmaceuticals Announces Positive Interim Clinical Data for Dual-Functional RNAi Therapeutic ARO-DIMER-PA for Mixed Hyperlipidemia

Arrowhead Pharmaceuticals Announces Positive Interim Clinical Data for Dual-Functional RNAi Therapeutic ARO-DIMER-PA for Mixed Hyperlipidemia

Arrowhead Pharmaceuticals Announces Positive Interim Clinical Data for Dual-Functional RNAi Therapeutic ARO-DIMER-PA for Mixed Hyperlipidemia
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Diasome Pharmaceuticals Strengthens Leadership Team Ahead of Phase 3 Development for HDV-Insulin

Diasome Pharmaceuticals Strengthens Leadership Team Ahead of Phase 3 Development for HDV-Insulin

Diasome Pharmaceuticals Strengthens Leadership Team Ahead of Phase 3 Development for HDV-Insulin
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Evecxia Therapeutics Announces Positive Phase 1b Clinical Results for OCD and Depression Drug Candidate EVX-101

Evecxia Therapeutics Announces Positive Phase 1b Clinical Results for OCD and Depression Drug Candidate EVX-101

Evecxia Therapeutics, a clinical-stage neuroscience company, has announced positive results from its Phase 1b PK/PD target engagement trial for EVX-101, a drug candidate aimed at treating obsessive-compulsive disorder (OCD) and depression. The trial demonstrated that the active compound, 5-HTP, successfully entered the brain, elevated serotonin synthesis biomarkers, and increased serotonin activity beyond the effects of SSRI/SNRI therapy. EVX-101 is an oral, gastro-retentive, sustained-release tablet formulation of 5-HTP and low-dose carbidopa, designed to amplify serotonin synthesis. The company plans to initiate Phase 2 development for EVX-101 in OCD patients in the first half of 2027. This drug is intended as an adjunctive therapy for patients who do not adequately respond to first-line serotonin reuptake inhibitor antidepressants.
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FDA Expands Joenja Approval to Children Aged 4 to 11 Years with APDS

FDA Expands Joenja Approval to Children Aged 4 to 11 Years with APDS

The U.S. Food and Drug Administration (FDA) has expanded the approval of Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) to include children aged 4 to 11 years who weigh at least 27kg. This new approval builds upon the existing authorization for patients aged 12 years and older. APDS is a rare primary immunodeficiency caused by genetic variants in the PIK3CD or PIK3R1 genes, which are crucial for immune cell function. These variants lead to the overactivation of the PI3Kδ pathway, resulting in immunodeficiency and dysregulation. Joenja is an oral, small molecule PI3Kδ inhibitor designed to target this pathway. The FDA's decision was supported by data from a single-arm, phase 3 study involving 8 patients in the 4 to 11 age group, which showed consistent pharmacokinetic and safety profiles compared to older patients. The approval also introduces new 40mg and 50mg tablet strengths to accommodate pediatric dosing based on body weight.
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Enveric Biosciences Secures U.S. Patent for Non-Hallucinogenic Mescaline Derivatives for PTSD Treatment

Enveric Biosciences Secures U.S. Patent for Non-Hallucinogenic Mescaline Derivatives for PTSD Treatment

Enveric Biosciences Secures U.S. Patent for Non-Hallucinogenic Mescaline Derivatives for PTSD Treatment