MedPath

Clinical Trial News

Novel Royalty-Based Investment Fund Proposes New Model for ALS Drug Development

  • Researchers from MIT, Massachusetts General Hospital, and Boston University propose a novel "Fund of Adaptive Royalties" (FAR) that finances half the cost of adaptive platform trials in exchange for future drug royalties.
  • The innovative financing model generated an expected return of 28% with a 22% probability of total loss in simulations based on HEALEY ALS Platform Trial data.
  • Adaptive platform trials allow multiple ALS drug candidates to be tested simultaneously under a single protocol, offering lower costs and shorter durations compared to traditional clinical trials.
  • The approach aims to address the funding bottleneck that has slowed ALS therapy development by attracting risk-tolerant investors like hedge funds and philanthropists.

Regener-Eyes Receives FDA Closure of Voluntary Recall, Returns to Full Operations with Enhanced Safety Features

FDA Approval
  • The U.S. Food and Drug Administration has officially closed Regener-Eyes' voluntary product recall, marking full regulatory resolution and return to normal operations for the dry eye treatment company.
  • The company proactively initiated the recall out of abundance of caution and introduced an enhanced bottle design featuring advanced backflow prevention technology to protect against microbial contamination.
  • Regener-Eyes is now in full production and distribution of its upgraded bottle design while maintaining confidence in the product's continued safety and efficacy for treating dry eyes.

Invivyd Forms SPEAR Study Group to Investigate Monoclonal Antibody Therapy for Long COVID

Monoclonal Antibody
  • Invivyd has established the SPEAR Study Group with leading researchers to investigate monoclonal antibody therapy for Long COVID and Post-Vaccination Syndrome, following anecdotal reports of symptom relief with PEMGARDA.
  • The study group will conduct rigorous clinical trials to evaluate the safety and efficacy of anti-SARS-CoV-2 spike protein monoclonal antibodies in patients with persistent viral reservoirs or circulating spike protein.
  • Long COVID affects approximately 5% or more of Americans with no approved treatments currently available, representing a significant unmet medical need.
  • The initiative aims to generate data for potential registrational studies and regulatory pathways, with Invivyd planning FDA discussions on rapid approval pathways for COVID-19 monoclonal antibodies.

Headlands Research Expands to Caribbean with CMRCenter Acquisition, Enhancing Hispanic Patient Access to Clinical Trials

  • Headlands Research acquired CMRCenter (CMRC) in San Juan, Puerto Rico, expanding its clinical trial network to 22 sites across the United States, Canada, and Puerto Rico.
  • The acquisition provides pharmaceutical sponsors streamlined access to one of the largest Hispanic patient populations under FDA oversight, with CMRC maintaining a 90% retention rate in trials.
  • CMRC brings expertise in CNS disorders, vaccines, and specialty conditions, with a bilingual team and database of over 10,000 engaged participants.
  • The deal combines CMRC's local knowledge of Puerto Rico's medical and regulatory landscape with Headlands' centralized infrastructure to accelerate clinical research.

Argon AI Secures $5.5M to Transform Pharmaceutical Data Management with AI-Native Workspace

AI
  • Argon AI raised $5.5 million in seed funding led by Crosslink Capital and Wireframe Ventures to develop an AI-native workspace specifically designed for life sciences companies.
  • The platform addresses critical inefficiencies in pharmaceutical data management, where internal data doubles every two years and teams process tens of thousands of documents annually in unwieldy formats.
  • Argon's AI-enabled workspace integrates internal company data with proprietary clinical and commercial databases, automating tasks like market research, competitive tracking, and clinical trial benchmarking.
  • The company is already working with two top-15 global pharmaceutical companies and deployed at premier consulting firms, with potential to save millions in misdirected R&D spending.

Delhi High Court Orders CDSCO to Review Safety of GLP-1 Diabetes Drugs Used for Weight Loss

Drug Approval
  • The Delhi High Court has directed India's Central Drugs Standard Control Organisation (CDSCO) to decide within three months on a petition challenging the licensing of GLP-1 receptor agonist drugs for weight loss treatment.
  • The petition raises safety concerns about semaglutide, tirzepatide, and liraglutide being approved for weight management despite limited India-specific clinical trial data and inadequate safety evaluation.
  • These drugs were originally developed for Type 2 diabetes treatment but have been repurposed for obesity management, with the petitioner citing risks including pancreatitis, gastrointestinal damage, and potential cancers.
  • The court emphasized that CDSCO must consult experts and relevant stakeholders before making its decision on the regulatory concerns raised in the petition.

BioVersys and Shionogi Form Strategic Partnership to Develop Novel NTM Treatments Worth Up to CHF 484 Million

  • BioVersys and Shionogi have entered a research collaboration and exclusive license option agreement to develop novel ansamycin leads from BioVersys' BV500 program targeting non-tuberculous mycobacteria infections.
  • The partnership includes CHF 5.0 million in upfront and research payments, with potential regulatory and sales milestones reaching CHF 479 million plus tiered royalties on global sales.
  • The BV500 program addresses a critical unmet medical need, as NTM pulmonary disease affects approximately 250,000 people annually with limited effective treatment options.
  • BioVersys' lead candidates demonstrate broad-spectrum anti-NTM activity without cross-resistance to existing therapeutic classes, potentially offering superior treatment outcomes for patients with these challenging infections.

EPO and UPC Reach Opposite Conclusions on Amgen's PCSK9 Antibody Patent Validity

Monoclonal Antibody
  • The European Patent Office (EPO) Opposition Division upheld Amgen's PCSK9 antibody patent EP 3666797, finding it inventive, while the Unified Patent Court (UPC) Central Division revoked the same patent for lacking inventive step.
  • Both tribunals agreed on the starting point for assessment and that skilled persons would be motivated to develop PCSK9-blocking antibodies, but diverged on whether there was a "reasonable expectation of success" for therapeutic efficacy.
  • The EPO emphasized uncertainties about therapeutic effectiveness based on prior art, while the UPC concluded that antibody development challenges were routine and did not prevent obviousness.
  • This case highlights potential systematic differences between EPO and UPC approaches to inventive step assessment, particularly relevant for biotechnology patents where "obvious to try" versus "reasonable expectation of success" standards may vary.

PPP2R1A Mutations Predict Improved Survival After Cancer Immunotherapy in Ovarian Clear Cell Carcinoma

  • Patients with ovarian clear cell carcinoma harboring PPP2R1A mutations demonstrated significantly improved overall survival of 66.9 months compared to 9.2 months in patients without these mutations following immunotherapy treatment.
  • The study analyzed 34 patients with platinum-resistant ovarian clear cell carcinoma treated with combination immune checkpoint inhibitors durvalumab and tremelimumab, identifying PPP2R1A mutations in 32.4% of patients.
  • Laboratory studies confirmed that targeting PPP2R1A enhanced immunotherapy responses both in vitro and in vivo, suggesting a causal relationship and potential therapeutic target.
  • Validation across multiple cancer types including endometrial cancer and over 9,000 patients confirmed the predictive value of PPP2R1A mutations for immunotherapy outcomes.

A Study of Nivolumab by Itself or Nivolumab Combined With Ipilimumab in Patients With Advanced or Metastatic Solid Tumors

NCT01928394CompletedPhase 1
Bristol-Myers Squibb
Posted 10/24/2013

Durvalumab and Tremelimumab in Treating Participants With Recurrent or Refractory Ovarian, Primary Peritoneal, or Fallopian Tube Cancer

NCT03026062Active, Not RecruitingPhase 2
M.D. Anderson Cancer Center
Posted 5/18/2017

Safety and Efficacy of Targeting PP2A in Ovarian Clear Cell Carcinoma Using Dostarlimab and LB-100

NCT06065462RecruitingPhase 1
M.D. Anderson Cancer Center
Posted 11/10/2023

Fidelio Capital Acquires Majority Stake in Alphalyse to Expand LC-MS Host Cell Protein Testing Globally

  • Fidelio Capital has acquired a majority stake in Danish CRO Alphalyse, which specializes in GMP-validated LC-MS assays for host cell protein impurity testing in biologic drugs.
  • The partnership aims to expand Alphalyse's capacity and international footprint as demand grows for precise HCP impurity analysis in the expanding biologics pipeline.
  • LC-MS technology is emerging as the preferred method over legacy ELISA technology, supported by new U.S. Pharmacopeia General Chapter 1132.1 and industry discussions.
  • Alphalyse's GMP-validated assays support customers from discovery to commercial release, providing quantitative HCP data that de-risks regulatory submissions and shortens development timelines.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.