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Clinical Trial News

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

AIIMS-Led Study Demonstrates Equal Efficacy of Three Two-Drug Hypertension Combinations in South Asian Patients

  • The TOPSPIN trial, led by AIIMS Delhi, found that three commonly used two-drug combination pills for hypertension showed equal effectiveness in reducing blood pressure among South Asian patients, with all combinations achieving approximately 14/8 mmHg reduction in 24-hour ambulatory measurements.
  • Nearly 70% of the 1,981 participants achieved blood pressure control below 140/90 mmHg after 6 months of treatment, representing a significant improvement over current national control rates in India.
  • All three combinations (amlodipine-perindopril, amlodipine-indapamide, and perindopril-indapamide) demonstrated similar safety profiles with less than 3% of participants discontinuing therapy due to side effects.
  • The study provides the first robust evidence for optimal dual combination therapy in South Asian populations, who represent a quarter of the world's population and face an enormous hypertension burden with poor control rates.

FDA Approves Doptelet for Pediatric Immune Thrombocytopenia with New Sprinkle Formulation

Drug Approval
  • The FDA has approved Doptelet (avatrombopag) for treating thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia who have had insufficient response to prior therapy.
  • The approval includes a new pediatric-friendly sprinkle formulation for children ages one to less than six years, offering flexible oral administration with no food restrictions.
  • In the pivotal AVA-PED-301 phase 3 study, 27.8% of patients receiving Doptelet achieved durable platelet response compared to 0% in the placebo group, with 81.5% achieving alternative platelet response endpoints.
  • The oral thrombopoietin receptor agonist was generally well-tolerated, with the most common side effects being viral infections, nasopharyngitis, cough, fever, and sore throat.

Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

NCT04516967Active, Not RecruitingPhase 3
Sobi, Inc.
Posted 3/5/2021

Kintor Pharma's KX-826 Hair Loss Treatment Achieves Primary Endpoint in Phase II Trial

  • Kintor Pharma's KX-826 tincture 1.0% achieved its primary endpoint in a Phase II clinical trial for male androgenetic alopecia treatment.
  • Both 0.5% and 1.0% twice-daily dosing groups showed statistically significant hair growth improvements compared to placebo after 24 weeks.
  • The treatment demonstrated satisfactory safety profile with no drug-related sexual dysfunction adverse events reported.
  • An independent Data Monitoring Committee recommended proceeding to Phase III without modifications to study design or sample size.

Plant Virus Nanoparticles Show Promise as Novel Cancer Immunotherapy in Preclinical Studies

  • Researchers at UC San Diego have demonstrated that cowpea mosaic virus (CPMV) nanoparticles derived from black-eyed pea plants can effectively stimulate immune responses against cancer in preclinical models.
  • The treatment showed remarkable success in improving survival rates and suppressing metastatic tumor growth across colon, ovarian, melanoma, and breast cancer models when administered systemically.
  • CPMV uniquely activates type I, II, and III interferons and toll-like receptor 7 (TLR7), creating both immediate and long-lasting anti-tumor immune responses.
  • The plant virus-based therapy offers a cost-effective alternative to traditional immunotherapies, as it can be cultivated using molecular farming with basic agricultural resources.

European Regulators Recommend Expanding Novo Nordisk's Alhemo for Hemophilia A and B Without Inhibitors

Monoclonal Antibody
  • The European Medicines Agency's Committee for Medicinal Products for Human Use issued a positive opinion recommending label expansion of Alhemo (concizumab) to treat severe hemophilia A and moderate/severe hemophilia B without inhibitors.
  • Phase 3 explorer8 trial results showed Alhemo prophylaxis reduced bleeding episodes by 86% in hemophilia A patients and 79% in hemophilia B patients compared to no prophylaxis treatment.
  • The label expansion would make Alhemo available to approximately 12,000 EU patients with hemophilia A and B without inhibitors, representing 79.2% of the market segment.
  • Patient-reported outcomes demonstrated 70.9% of participants preferred Alhemo over their previous hemophilia treatment, with improvements in quality of life and reduced treatment burden.

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors

NCT04082429Active, Not RecruitingPhase 3
Novo Nordisk A/S
Posted 11/13/2019

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors

NCT04083781Active, Not RecruitingPhase 3
Novo Nordisk A/S
Posted 10/21/2019

MilliporeSigma and Washington University Strengthen 90-Year Partnership to Accelerate Life Sciences Innovation

  • MilliporeSigma and Washington University in St. Louis have signed a memorandum of understanding to expand their nearly century-long collaboration, focusing on joint research initiatives and technology development.
  • The partnership aims to build a robust research and development talent pipeline in St. Louis, where MilliporeSigma employs approximately 2,300 people across production and R&D facilities.
  • The collaboration will provide university researchers with access to MilliporeSigma's portfolio of over 300,000 products and technical expertise to accelerate the transition from laboratory discoveries to clinical applications.
  • MilliporeSigma has invested more than $250 million in its St. Louis operations over the past five years and previously contributed nearly $14 million worth of laboratory equipment to WashU scientists.

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