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FDA Advisory Panel Rejects GSK's Blenrep Combination Therapies for Multiple Myeloma

Drug Approval
  • FDA advisers voted 5-3 against approving GSK's Blenrep in combination with bortezomib and dexamethasone for relapsed or refractory multiple myeloma patients.
  • The panel voted 7-1 against Blenrep combined with pomalidomide and dexamethasone, citing unfavorable benefit-risk profile for the proposed patient population.
  • FDA briefing documents expressed concerns about Blenrep's unclear benefit-risk profile, safety, tolerability, and appropriate dosages.
  • Multiple myeloma is the third most common blood cancer and is generally treatable but not curable.

FDA Authorizes Juul E-Cigarettes for Continued Market Sale After Multiyear Review

Drug Approval
  • The FDA has authorized Juul Labs to continue selling its tobacco and menthol-flavored e-cigarettes after determining they are "appropriate for the protection of public health."
  • The decision follows a multiyear regulatory review that began in 2022 when the FDA initially ordered Juul to remove its products from the market.
  • Juul provided evidence showing high rates of adult smokers switch from traditional cigarettes to its products, reducing exposure to deadly carcinogens.
  • The authorization makes Juul one of only two U.S. companies permitted to sell menthol-flavored vaping products to adult consumers.

FDA-Approved Rectal Cancer Drug Metric Fails to Predict Long-Term Survival, Tulane Study Finds

Drug ApprovalOncology
  • A Tulane University meta-analysis of 25 clinical trials involving nearly 12,000 rectal cancer patients found no statistical relationship between pathologic complete response (pCR) and overall survival.
  • The study challenges the FDA's 2012 decision to allow pharmaceutical companies to use tumor-free status post-therapy as a surrogate for overall survival in drug approvals.
  • Researchers warn that cancer drugs may be fast-tracked for approval without demonstrating meaningful long-term improvements over existing treatments.
  • The findings suggest that relying solely on pCR as an endpoint may lead to increased costs for drug companies investing in therapies that cannot guarantee improved survival rates.

Rectal Cancer And Pre-operative Induction Therapy Followed by Dedicated Operation. The RAPIDO Trial

NCT01558921Active, Not RecruitingPhase 3
University Medical Center Groningen
Posted 6/21/2011

Ambulatory Pharmacists Emerge as Key Players in Value-Based Care Models to Address Rising Drug Costs

  • Ambulatory clinical pharmacists serve three critical roles in value-based care: managing chronic diseases, preventing hospitalizations through population health outreach, and providing financial expertise on medication costs.
  • These pharmacists help keep high-risk patients with conditions like diabetes, heart failure, and hypertension out of hospitals by managing complex medication regimens and ensuring adherence through technology-enabled outreach programs.
  • With new medications costing over $5,000 per dose, pharmacists' expertise in drug pricing is becoming essential for health systems to negotiate contracts and avoid financial losses from high-cost therapies.

Citizens' Petition Seeks FDA Accelerated Approval for NurOwn Stem Cell Therapy in ALS Based on Survival Data

Drug ApprovalReal World Evidence
  • A 309-page Citizens' Petition has been filed with the FDA seeking accelerated approval of NurOwn, a mesenchymal stem cell therapy for ALS, based on unprecedented survival and respiratory data from trial participants.
  • The petition presents compelling real-world evidence including 100% five-year survival in the Expanded Access Program versus 20% natural history (p-value ~0.000335), and median 7-year tracheostomy-free survival compared to 30 months in natural history.
  • Neurologist Dr. Danielle Geraldi-Samara documented clinical improvements in multiple NurOwn recipients, including patients who regained motor function and maintained respiratory independence years beyond typical ALS progression timelines.
  • The petition argues that the FDA's focus on primary endpoints overlooked the totality of evidence, including patient experiences and expert clinical observations that demonstrate NurOwn's efficacy in slowing disease progression.

Renovaro's Engineered Dendritic Cell Therapy Achieves 81.7% Tumor Reduction in Pancreatic Cancer Models

  • Renovaro Biosciences published peer-reviewed preclinical data in Vaccines journal showing their genetically engineered dendritic cell therapy reduced pancreatic tumor burden by up to 81.7% in humanized mouse models.
  • The therapy uses CD34+ stem cells from healthy donors to create "off-the-shelf" dendritic cells engineered to express CD93, CD40L, and CXCL13 proteins that enhance immune system activation.
  • Treated animals demonstrated significant increases in CD4+ helper T cells and cytotoxic CD8+ T cells in blood, bone marrow, and tumor microenvironments.
  • The allogeneic approach addresses manufacturing scalability challenges that have limited previous dendritic cell therapies like Provenge, potentially improving cost structure and market accessibility.

One Biosciences Secures €15 Million Series A to Advance AI-Powered Single-Cell Cancer Diagnostics Platform

AI
  • One Biosciences raised €15 million in Series A financing led by Redmile Group and Blast to advance its OneMap platform for single-cell cancer diagnostics.
  • The AI-powered platform generates detailed functional profiles of patient tumors to guide treatment decisions and improve patient selection for clinical trials.
  • Funding will accelerate clinical development of OneMap and support strategic partnerships with pharmaceutical and biotech companies.
  • The company's total funding now exceeds €20 million, positioning OneMap as a clinical tool for precision oncology applications.

Keros Therapeutics Initiates Phase 3 RENEW Trial for Elritercept in Myelodysplastic Syndromes, Triggers $10M Takeda Milestone

  • Keros Therapeutics has dosed the first patient in the Phase 3 RENEW clinical trial evaluating elritercept for transfusion-dependent anemia in myelodysplastic syndromes.
  • The milestone triggers a $10 million payment from Takeda under their global licensing agreement, which includes potential payments exceeding $1.1 billion.
  • The randomized, double-blind, placebo-controlled trial aims to evaluate elritercept's efficacy in reducing red blood cell transfusions in MDS patients.
  • Elritercept is an engineered ligand trap targeting TGF-β signaling pathways to treat cytopenias including anemia and thrombocytopenia.

A study of elritercept to treat transfusion-dependent anemia due to very low, low, or intermediate risk myelodysplastic syndromes

2024-516009-22-00RecruitingPhase 3
Takeda
Posted 5/6/2025

Seaport Therapeutics Initiates Phase 2b Trial of Novel Oral Antidepressant GlyphAllo

  • Seaport Therapeutics has dosed the first patient in its phase 2b BUOY-1 trial of GlyphAllo, a novel oral prodrug of allopregnanolone for major depressive disorder with or without anxious distress.
  • The global study will enroll up to 360 participants randomized to receive GlyphAllo or placebo once daily over six weeks, with the primary endpoint being change in HAM-D-17 depression scores.
  • Phase 1 data showed GlyphAllo achieved nine times greater allopregnanolone exposure than oral dosing and matched efficacious IV levels, while phase 2a results demonstrated significant stress hormone reduction.
  • If successful, GlyphAllo could become a first-in-class treatment for the approximately 280 million people globally affected by major depression, with nearly 60% also experiencing anxious distress.

MOMA Therapeutics Initiates First-in-Human Trial of Werner Helicase Inhibitor MOMA-341 for MSI-H Solid Tumors

Precision Medicine
  • MOMA Therapeutics has dosed the first patient in a Phase 1 clinical trial of MOMA-341, a novel oral Werner helicase inhibitor targeting advanced or metastatic solid tumors with high microsatellite instability.
  • The drug represents a potentially best-in-class therapeutic with a novel chemical scaffold discovered through MOMA's proprietary KNOMATIC platform, designed to exploit Werner helicase dependency in MSI-H/dMMR tumor cells.
  • The global multi-center trial will evaluate MOMA-341 as monotherapy and in combination with chemotherapy or immunotherapy, with initial monotherapy data expected in mid-2026.
  • This marks MOMA's second clinical entry within a year, following their Pol θ helicase inhibitor MOMA-313, demonstrating the company's rapid pipeline advancement in precision oncology.

Study of Orally Administered MOMA-341 in Participants With Advanced or Metastatic Solid Tumors

NCT06974110RecruitingPhase 1
MOMA Therapeutics
Posted 7/16/2025

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