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Clinical Trial News

Genetic Leap Receives FDA IND Clearance for First-in-Class Oral IL-2 Modulator GL-IL2-138

  • Genetic Leap announced FDA clearance of its IND application for GL-IL2-138, a small-molecule modulator that binds endogenous IL-2 mRNA to drive controlled production of natural IL-2 protein by immune cells.
  • The oral drug offers superior potency and durability compared to recombinant IL-2 protein, with potential applications across oncology, immunology, and neurodegeneration diseases.
  • GL-IL2-138 will now enter a randomized, double-blind, placebo-controlled phase 1 clinical trial to evaluate its tolerability, pharmacokinetics and pharmacodynamics in humans.

CUE-101 Combination Therapy Achieves 50% Response Rate in HPV+ Head and Neck Cancer Trial

  • Cue Biopharma's CUE-101 combined with pembrolizumab demonstrated a 50% overall response rate in patients with recurrent/metastatic HPV+ head and neck squamous cell carcinoma, significantly outperforming historical pembrolizumab monotherapy data.
  • The combination therapy showed remarkable survival benefits with 12-month overall survival of 88% and median overall survival of 32 months, compared to 57% and 12.3 months respectively with pembrolizumab alone.
  • An additional complete response was observed in a patient with multiple tumor sites, including lung metastases that cleared before the target lesion achieved complete response.
  • The therapy maintained efficacy across different PD-L1 expression levels, achieving 50% response rates even in patients with low combined positive scores (1-19).

A Study of Pembrolizumab (MK-3475) for First Line Treatment of Recurrent or Metastatic Squamous Cell Cancer of the Head and Neck (MK-3475-048/KEYNOTE-048)

NCT02358031CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 3/19/2015

A Phase 1 Study in Patients with HPV16+ Recurrent/ Metastatic Head and Neck Squamous Cell Carcinoma

NCT03978689Active, Not RecruitingPhase 1
Cue Biopharma
Posted 7/30/2019

Johnson & Johnson Raises 2025 Outlook as Oncology Portfolio Drives 24% Growth

CAR-TOncology
  • Johnson & Johnson reported strong Q2 2025 results with total sales of $23.7 billion, driven by 24% growth in oncology and 15.1% growth in neuroscience divisions.
  • The company raised its full-year 2025 sales guidance from $91-91.8 billion to $93.2-93.6 billion and set an ambitious target to grow oncology sales to $50 billion by 2030.
  • Multiple myeloma drug Darzalex led oncology growth with $3.54 billion in sales, while CAR-T therapy Carvykti generated $439 million and new bispecific antibodies contributed to portfolio expansion.
  • Despite strong performance in growth areas, Stelara sales declined 42.7% to $1.65 billion due to biosimilar competition, creating an 1,170 basis point impact on overall growth.

FDA Reviewers Question Efficacy of Otsuka's Brexpiprazole-Zoloft Combination for PTSD Treatment

Drug Approval
  • FDA staff reviewers have raised significant efficacy concerns over Otsuka Pharma's brexpiprazole combination therapy for PTSD, citing inconsistent trial results and modest treatment effects that may lack clinical meaningfulness.
  • The combination drug failed to demonstrate substantial evidence of effectiveness when added to sertraline, with one late-stage study showing no statistically significant differences between combination therapy and sertraline monotherapy.
  • An FDA advisory panel meeting scheduled for Friday will provide recommendations on the approval of brexpiprazole combined with Viatris' Zoloft for treating adults with PTSD.
  • The FDA has delayed its original February 8 decision deadline and has not set a new action date for the drug application.

Endeavor BioMedicines Receives Dual Orphan Drug Designation for Taladegib in Idiopathic Pulmonary Fibrosis

Rare Disease
  • Both the FDA and European Commission have granted Orphan Drug Designation to taladegib (ENV-101) for treating idiopathic pulmonary fibrosis, highlighting significant unmet medical need.
  • The investigational Hedgehog pathway inhibitor aims to reverse disease progression by stopping abnormal myofibroblast accumulation that causes lung fibrosis.
  • Endeavor is currently enrolling patients in the Phase 2b WHISTLE-PF trial, with enrollment expected to complete in 2026.
  • IPF affects more than 150,000 adults in the United States with limited treatment options and an average life expectancy of three to five years after diagnosis.

A Phase 2, Multi-Center, Randomized, Double-Blind, Controlled Trial Evaluating the Safety and Efficacy of ENV-101 in Patients with Lung Fibrosis (WHISTLE-PF Trial)

2024-511754-41-00RecruitingPhase 2
Endeavor Biomedicines Inc.
Posted 11/15/2024

LogiCare3PL Secures Exclusive Distribution Partnership for ChiRhoStim Diagnostic Product

Orphan DrugRare Disease
  • LogiCare3PL, a division of BioCare Inc., has entered into an exclusive third-party logistics partnership with ChiRhoClin Inc. for the distribution of ChiRhoStim (Human Secretin for Injection), effective July 14, 2025.
  • The partnership leverages LogiCare3PL's specialized expertise in orphan drug logistics and operates through two cGMP-compliant, NABP-accredited distribution centers in Arizona and Mississippi.
  • ChiRhoStim serves as a critical diagnostic product for pancreatic and gastrointestinal conditions, supporting ChiRhoClin's mission to provide timely access to diagnostic treatments for rare diseases.

Stargardt Disease Pipeline Shows Promising Progress with Multiple Gene Therapies Advancing to Late-Stage Trials

Rare DiseaseFDA Approval
  • Ocugen's OCU410ST gene therapy has received FDA approval to launch a Phase 2/3 pivotal confirmatory trial for treating all forms of Stargardt disease, with the potential to serve as the foundation for a biologics license application.
  • SpliceBio has administered the first dose in its Phase 1/2 ASTRA clinical trial evaluating SB-007, a dual AAV vector-based gene therapy for Stargardt disease, marking another significant milestone in gene therapy development.
  • Alkeus Pharmaceuticals reported positive interim results from its TEASE-3 study, showing that early-stage Stargardt disease patients receiving oral gildeuretinol acetate exhibited no disease progression and maintained stable visual acuity over multiple years.
  • The Stargardt disease pipeline now includes over 20 companies developing more than 20 treatment therapies, with emerging therapies like MCO-010, Tinlarebant, and others expected to significantly impact the market.

FDA Accepts Biosimilar Application for BAT2506, Proposed Golimumab Alternative

Monoclonal AntibodyDrug Approval
  • The FDA has accepted Bio-Thera Solutions' Biologics License Application for BAT2506, a proposed biosimilar to Simponi (golimumab), with a target review completion date of May 16, 2026.
  • The application seeks approval for all current Simponi indications including rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, and ulcerative colitis, with interchangeability designation requested.
  • BAT2506 demonstrated similar efficacy, safety, and immunogenicity to reference golimumab in comprehensive Phase I and Phase III clinical studies.
  • Accord BioPharma will serve as the marketing authorization holder and commercialize the biosimilar in the United States if approved.

SandboxAQ and iOncologi Partner to Develop AI-Driven mRNA Vaccine for Glioblastoma

  • SandboxAQ and iOncologi announced a strategic collaboration to develop a novel high-fidelity mRNA vaccine for glioblastoma, the most aggressive malignant brain tumor in adults.
  • The partnership combines SandboxAQ's AQBioSim platform using Large Quantitative Models with iOncologi's immunotherapy expertise to overcome treatment barriers in brain cancer.
  • The joint program aims to deliver a lead therapeutic candidate into clinical trials within 18 months, targeting a disease with limited treatment options and survival rates under two years.
  • Glioblastoma accounts for approximately 15% of all primary brain tumors, with about 300,000 new cases and over 200,000 deaths worldwide annually according to the World Health Organization.

Tempus AI Receives FDA 510(k) Clearance for ECG-Low EF Software to Detect Heart Failure Risk

AI
  • Tempus AI received FDA 510(k) clearance for its Tempus ECG-Low EF software, which uses artificial intelligence to identify patients with low left ventricular ejection fraction from standard ECG recordings.
  • The software analyzes 12-lead ECG recordings to detect signs associated with LVEF ≤40% in patients aged 40 and older at risk of heart failure, including those with diabetes, hypertension, and other cardiovascular conditions.
  • This represents Tempus's second FDA-cleared ECG-AI device following Tempus ECG-AF, expanding the company's cardiovascular diagnostic capabilities for early detection of serious cardiac conditions.
  • The technology is designed as a screening tool that requires clinical interpretation alongside other diagnostic information and is not intended as a standalone diagnostic device.

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