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Clinical Trial News

Health Canada Approves Clearside Biomedical's XIPERE for Uveitic Macular Edema Treatment

Drug Approval
  • Health Canada has granted approval for XIPERE (triamcinolone acetonide injectable suspension) for suprachoroidal use in treating uveitic macular edema, expanding the drug's global reach.
  • XIPERE is now approved in the United States, Canada, Australia, and Singapore, with regulatory review ongoing in China, demonstrating continued international validation.
  • The approval utilizes Clearside's proprietary suprachoroidal space injection platform featuring the SCS Microinjector, which provides targeted drug delivery to the back of the eye.
  • Bausch + Lomb holds exclusive commercialization rights for XIPERE in the US and Canada, while Arctic Vision has licensing rights across Greater China and other Asian markets.

Omeros Raises $22 Million in Direct Offering to Fund Narsoplimab Development Amid Regulatory Review

  • Omeros Corporation completed a $22 million registered direct offering with Polar Asset Management Partners, issuing 5.37 million shares at $4.10 per share, representing a 14% premium to the July 24 closing price.
  • The biotech company is using the funds to support R&D activities, including its pivotal narsoplimab program currently under FDA review for hematopoietic stem cell transplant-associated thrombotic microangiopathy.
  • The offering results in 8.7% dilution to existing shareholders as Omeros navigates a challenging financial position with $415 million in debt and negative EBITDA of $164 million.
  • FDA has extended the PDUFA date for narsoplimab's biologics license application to December 26, 2025, while the European Medicines Agency validated the Marketing Authorization Application with a decision expected mid-2026.

UVA Launches First-of-Its-Kind Psilocybin Trial for Prolonged Grief Disorder

  • The University of Virginia has initiated a groundbreaking clinical trial testing a single 25-milligram dose of psilocybin to treat prolonged grief disorder, a clinically recognized condition affecting approximately 10% of mourning individuals.
  • The study, funded by an $895,866 grant from the Tiny Blue Dot Foundation, will enroll 20 participants and represents the first research of its kind at UVA.
  • Researchers hypothesize that psilocybin's ability to target serotonin receptors and induce heightened connectedness may help patients reframe their relationship with grief when traditional treatments like antidepressants and cognitive behavioral therapy prove insufficient.

Agenus Advances BOT/BAL Combination Therapy with Phase 3 BATTMAN Trial Launch for Metastatic Colorectal Cancer

  • Agenus announced the launch of the BATTMAN Phase 3 trial evaluating botensilimab (BOT) and balstilimab (BAL) combination therapy in metastatic microsatellite stable colorectal cancer patients who have exhausted treatment options.
  • Clinical data showed BOT demonstrated a 42% two-year survival rate and 21-month median overall survival in late-stage MSS colorectal cancer patients, significantly outperforming traditional chemotherapy's 10-14 month survival.
  • The combination therapy showed promising results across multiple cancer types, with neoadjuvant trials reporting 70% pathological complete response in MSI-H tumors and 20% in MSS tumors.
  • Company executives called for regulatory reform to accelerate patient access to promising therapies, citing the urgent need for FDA approval processes to match the pace of scientific advancement.

Recognify's Inidascamine Misses Primary Endpoint in Phase 2b Trial for Schizophrenia-Associated Cognitive Impairment

  • Recognify Life Sciences' Phase 2b trial of inidascamine for cognitive impairment associated with schizophrenia failed to meet its primary endpoint of improvement on the MCCB neurocognitive composite score at Week 6.
  • The study enrolled 242 patients across the United States and Europe and demonstrated numerical improvements across multiple cognitive measures compared to placebo, though without statistical significance.
  • Inidascamine maintained a favorable safety profile with no evidence of sedation, weight gain, or extrapyramidal symptoms commonly associated with schizophrenia treatments.
  • The company plans to analyze secondary endpoints and subgroup data to identify potential responder populations and inform future development strategies for this unmet medical need.

A Study to Evaluate RL-007 in the Treatment of Cognitive Impairment Associated With Schizophrenia (CIAS)

NCT05686239CompletedPhase 2
Recognify Life Sciences
Posted 12/8/2022

EMA Recommends Approval of Aqneursa for Niemann-Pick Type C Disease Treatment

Drug ApprovalRare DiseaseOrphan Drug
  • The European Medicines Agency's Committee for Medicinal Products for Human Use has issued a positive opinion recommending approval of Aqneursa (levacetylleucine) for treating neurological manifestations of Niemann-Pick type C disease in adults and children.
  • The recommendation is based on Phase III trial data showing statistically significant improvement in neurological symptoms within 12 weeks, with patients demonstrating better SARA scores compared to placebo.
  • Aqneursa represents a potential new frontline treatment option for NPC patients in Europe, following its FDA approval in September 2024.
  • The drug targets underlying neurological dysfunction processes and can be used in combination with miglustat or as monotherapy when miglustat is not tolerated.

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288RecruitingPhase 3
IntraBio Inc
Posted 6/30/2022

Insmed Opens Cambridge R&D Facility Focused on Synthetic Rescue Platform for Rare Diseases

  • Princess Anne officially opened Insmed's new 17,000 square foot research facility in Cambridge, UK, dedicated to synthetic rescue technology for rare diseases.
  • The facility employs 80 scientists using CRISPR and human genetic data mining to identify modifier genes that could restore cellular health in serious diseases.
  • Insmed's synthetic rescue platform aims to find therapeutic targets outside disrupted biological pathways that can potentially correct underlying gene defects.
  • The Cambridge team has already achieved significant breakthroughs in neurodegenerative disease research, with over 7,000 rare diseases representing substantial unmet medical need.

Comprehensive Pipeline Analysis Reveals 200+ Investigational Drugs for Metastatic Hormone Refractory Prostate Cancer

OncologyTargeted Therapy
  • A new comprehensive report analyzes over 200 pipeline drugs from 180+ companies targeting metastatic hormone refractory prostate cancer, highlighting the extensive research efforts in this challenging therapeutic area.
  • Key late-stage candidates include PNT2002 by Lantheus targeting PSMA in Phase III trials, and Opevesostat by Merck, an oral CYP11A1 inhibitor also in Phase III development.
  • The pipeline spans multiple development stages from discovery to Phase III, with promising mid-stage candidates like XL092 by Exelixis exploring receptor tyrosine kinase inhibition in Phase II trials.
  • Current treatment approaches involve next-generation androgen receptor inhibitors and chemotherapy agents, but significant unmet needs remain for patients experiencing disease progression despite anti-androgen treatments.

Leveragen and Propeller Bio Form Strategic Partnership to Advance Next-Generation Antibody Discovery Platform

CAR-T
  • Leveragen, a Boston-based biotech company, has announced a strategic collaboration with newly launched Propeller Bio to advance antibody and protein-based therapeutics development.
  • The partnership provides Propeller Bio access to Leveragen's proprietary Singularity Sapiens Mouse platform, which produces fully human single-domain antibodies with strong developability and diverse epitope coverage.
  • Propeller Bio is led by David Shen, former CEO of Proteologix, which was acquired by Johnson & Johnson for $850 million upfront in 2024.
  • The Singularity Sapiens Mouse platform supports multiple therapeutic formats including bispecifics, antibody-drug conjugates, CAR-T therapies, and mRNA-encoded antibodies.

GLP-1 Drugs Show Promise for Asthma Control in Obese Patients Beyond Weight Loss Benefits

Real World Evidence
  • A UK study of over 60,000 patients found that GLP-1 receptor agonists significantly improved asthma control scores in obese individuals with asthma compared to matched controls.
  • The improvements occurred despite modest weight loss of only 0.9 kg over one year, suggesting anti-inflammatory mechanisms beyond weight reduction may contribute to respiratory benefits.
  • Researchers propose GLP-1 drugs could offer an alternative to steroid treatments for obese asthma patients who often show resistance to standard inhaled corticosteroids.
  • The findings add to growing evidence of GLP-1 drugs' therapeutic potential beyond diabetes and weight management, though further research is needed to establish direct respiratory treatment applications.

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