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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.

India-UK Free Trade Agreement Raises Concerns Over Access to Affordable Medicines

  • Intellectual property experts warn that the India-UK Free Trade Agreement may weaken compulsory licensing mechanisms, a critical tool for ensuring access to affordable life-saving medicines during public health emergencies.
  • The FTA's emphasis on voluntary licensing over compulsory licensing could shift control of medicine access to market forces rather than government intervention, potentially limiting India's ability to act swiftly in health crises.
  • Critics argue the agreement represents a move toward TRIPS-plus provisions that go beyond World Trade Organization minimum standards, while the government maintains the deal preserves India's existing legal framework and public health priorities.
  • The FTA allows disclosure of commercial patent working once every three years instead of annually, which experts say could make it harder to prove unmet demand and justify compulsory licensing decisions.

FDA Complete Response Letters Highlight Growing CMC Challenges in Immunotherapy Development

Drug Approval
  • Recent FDA complete response letters for Replimune's RP1 and Capricor's CAP-1002 underscore increasing regulatory emphasis on analytical validation, tech transfer execution, and manufacturing controls in immunotherapy development.
  • Despite following FDA guidance throughout development, both companies received unexpected rejections, with 74% of CRLs issued from 2020 to 2024 citing quality or manufacturing deficiencies.
  • Industry experts warn that analytical methods and tech transfer plans that pass early scrutiny often collapse under commercial-scale expectations, requiring CMC rigor to be treated as front-line regulatory strategy.
  • The FDA's heightened scrutiny reflects evolving standards for biologics and advanced therapies, demanding robust manufacturing control strategies and comprehensive CMC data to ensure consistency, safety, and efficacy.

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03406780CompletedPhase 2
Capricor Inc.
Posted 4/4/2018

Study of RP1 Monotherapy and RP1 in Combination With Nivolumab

NCT03767348RecruitingPhase 2
Replimune Inc.
Posted 9/20/2017

Lyell Immunopharma Secures $100 Million Private Placement to Advance CAR T-Cell Therapy Development

CAR-T
  • Lyell Immunopharma announced a private placement agreement for up to $100 million in gross proceeds to advance its next-generation CAR T-cell therapy programs.
  • The funding includes an initial $50 million closing at $13.32 per share, with potential for an additional $50 million upon achieving clinical milestones related to the LYL314 PiNACLE pivotal trial.
  • The proceeds will fund two pivotal-stage clinical trials of LYL314 and extend the company's cash runway into mid-2027, supporting a planned Biologics License Application submission for large B-cell lymphoma treatment.
  • LYL314 is a dual-targeting CD19/CD20 CAR T-cell therapy designed to improve complete response rates and duration compared to approved CD19-targeted therapies.

Bristol Myers Squibb Appoints AstraZeneca's Cristian Massacesi as New Chief Medical Officer

Oncology
  • Bristol Myers Squibb has appointed Cristian Massacesi, M.D., former AstraZeneca Chief Medical Officer, as Executive Vice President, Chief Medical Officer, and Head of Development, effective August 1, 2025.
  • Massacesi replaces Samit Hirawat, who led Bristol Myers' development efforts for six years and oversaw the launch of blockbuster drugs including Reblozyl, which generated nearly $1.8 billion in revenue in 2024.
  • The leadership change comes as Bristol Myers faces significant challenges from generic competition for Revlimid and looming patent expirations for key drugs Opdivo and Eliquis.
  • Massacesi brings over 20 years of biopharmaceutical experience and previously led AstraZeneca's global organization of more than 3,000 employees, advancing over 150 clinical studies and securing multiple regulatory approvals.

European Regulators Recommend Approval of Vorasidenib for IDH-Mutant Grade 2 Glioma

Drug ApprovalTargeted TherapyOncology
  • The European Medicines Agency's CHMP has issued a positive opinion recommending approval of vorasidenib (Voranigo) for treating grade 2 IDH-mutant glioma in patients aged 12 years and older following surgical resection.
  • The recommendation is based on the phase 3 INDIGO trial showing vorasidenib significantly improved progression-free survival to 27.7 months versus 11.1 months with placebo.
  • If approved by the European Commission, vorasidenib would become the first targeted therapy for grade 2 IDH-mutant diffuse glioma in the European Union.
  • The drug is already approved in the United States, Canada, Australia, Israel, UAE, Saudi Arabia, and Switzerland for this indication.

Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)

NCT04164901Active, Not RecruitingPhase 3
Institut de Recherches Internationales Servier
Posted 1/5/2020

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