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Clinical Trial News

Real-World Evidence Validates Amivantamab-Lazertinib Combination in Advanced NSCLC Treatment

Real World Evidence
  • Real-world implementation of amivantamab plus lazertinib combination therapy has demonstrated outcomes consistent with clinical trial results, with practitioners reporting remarkable responses across various clinical settings.
  • The regimen has shown effectiveness in challenging scenarios such as patients with leptomeningeal disease, where responses have been observed lasting over 10 months.
  • Standardized prophylactic protocols including dexamethasone premedication and graduated infusion rates have significantly reduced the severity and frequency of infusion-related reactions.
  • Key success factors include comprehensive patient education, proactive adverse effect management, and demonstration of treatment efficacy to maintain patient confidence and adherence.

Modified NeoVax Cancer Vaccine Shows Enhanced Immune Response in Melanoma Patients

  • Dana-Farber researchers developed NeoVaxMI, an improved version of their personalized cancer vaccine that demonstrated enhanced immune responses in all nine melanoma patients tested.
  • The modified vaccine combines personalized neoantigens with new immune-boosting compounds and is administered alongside nivolumab and ipilimumab immunotherapies.
  • Phase 1 trial results published in Cell show the vaccine induced cytotoxic CD8+ T cell responses in six of nine patients and confirmed vaccine-induced T cells infiltrated tumors.
  • The enhanced formulation and delivery approach represents a significant advancement in personalized cancer vaccine development with improved immunogenicity compared to previous trials.

IL-2 Inhibitor Pipeline Expands with 20+ Companies Developing 22+ Therapeutic Candidates

  • DelveInsight's 2025 pipeline report reveals over 20 pharmaceutical companies are actively developing 22+ interleukin-2 inhibitor drugs across various clinical stages.
  • The FDA granted fast track designation to rezpegaldesleukin for moderate to severe atopic dermatitis in February 2025, while nemvaleukin development was discontinued following failed Phase III trial results.
  • Leading pipeline candidates include Soquelitinib in Phase III for relapsed/refractory PTCL and multiple Phase II programs targeting autoimmune diseases and cancer indications.
  • The growing prevalence of autoimmune diseases and organ transplant procedures is driving increased demand for IL-2 inhibitors due to their role in preventing immune rejection.

Sernova and Eledon Partner to Advance Type 1 Diabetes Bio-hybrid Organ Therapy with Novel Immunosuppression

Monoclonal Antibody
  • Sernova Biotherapeutics and Eledon Pharmaceuticals announced a collaboration to evaluate tegoprubart, an anti-CD40L antibody, in Sernova's Phase 1/2 Cell Pouch Bio-hybrid Organ clinical trial for type 1 diabetes.
  • Tegoprubart will replace tacrolimus in Cohort C of the trial, potentially offering superior islet cell protection with reduced toxicity compared to standard immunosuppression.
  • Previous study results showed three patients achieved insulin independence with tegoprubart, demonstrating islet engraftment three to five times higher than tacrolimus-based treatments.
  • The partnership combines Sernova's retrievable Cell Pouch technology with Evotec's stem cell-derived islet clusters and Eledon's novel immunosuppressive approach to advance a potential functional cure for type 1 diabetes.

Eolas Therapeutics Assumes Full Development Rights to First-in-Class Orexin-1 Receptor Antagonist AZD4041 for Substance Use Disorders

  • Eolas Therapeutics has secured full development rights to AZD4041 from AstraZeneca, a highly selective orexin-1 receptor antagonist targeting opioid use disorder and other substance use disorders.
  • AZD4041 represents a potential first-in-class, non-opioid therapeutic that targets neural circuits regulating craving, reward, and stress rather than traditional drug-signaling pathways.
  • The compound has completed Phase I studies with promising preclinical data and will advance to Phase II trials with support from a NIDA UG3 cooperative agreement.
  • This therapeutic approach addresses brain circuitry of addiction directly, offering a novel treatment strategy for opioid, stimulant, and alcohol use disorders.

Niagen Bioscience Secures Exclusive License for Nicotinamide Riboside as Parkinson's Disease Therapy

  • Niagen Bioscience has obtained worldwide exclusive commercial rights to develop nicotinamide riboside (Niagen) as a potential Parkinson's disease therapy through a licensing agreement with Haukeland University Hospital in Norway.
  • The agreement is supported by data from the NOPARK trial, the largest clinical investigation of NAD+ augmentation in early Parkinson's disease, involving 400 patients across 12 Norwegian sites who received either 500mg nicotinamide riboside or placebo for 52 weeks.
  • This licensing deal positions Niagen Bioscience as the only company with rights to seek regulatory approval for pharmaceutical nicotinamide riboside therapy in Parkinson's disease patients.
  • The company has established a wholly-owned subsidiary to transition from supplement science to regulated drug development for neurodegenerative diseases with high unmet medical needs.

University of Cincinnati Joins International Phase 1 Trial Testing CAR-T Cell Therapy for Multiple Sclerosis

CAR-T
  • The University of Cincinnati Gardner Neuroscience Institute has become one of the first U.S. sites participating in an international Phase 1 clinical trial testing CAR-T cell therapy for multiple sclerosis patients.
  • The innovative treatment involves genetically modifying patients' own T cells to recognize and eliminate malfunctioning immune cells that drive MS, potentially reaching areas current therapies cannot access effectively.
  • Unlike current MS therapies that mainly target inflammatory cells in the bloodstream, engineered CAR-T cells are designed to find and eliminate harmful immune cells throughout the body, including in the brain and other tissues.
  • The trial includes both relapsing and progressive MS patient cohorts, with researchers having already successfully screened their first patient in the progressive MS group.

Jazz Pharmaceuticals Names Renee Gala as CEO in Strategic Leadership Transition

Drug Approval
  • Jazz Pharmaceuticals' Board of Directors unanimously selected Renee Gala as President and CEO, effective August 11, 2025, succeeding co-founder Bruce Cozadd who will remain as Board Chairperson.
  • Gala's leadership has driven Jazz's transformation with nearly 90% revenue growth and diversified portfolio expansion, positioning the company for sustainable growth with a robust oncology and neuroscience pipeline.
  • The leadership transition comes as Jazz pursues ambitious growth targets including a $5 billion revenue goal by 2025, with key regulatory milestones and clinical readouts expected in the coming months.

FDA Grants Breakthrough Therapy Designation to TSND-201 for PTSD Treatment

  • The FDA has granted Breakthrough Therapy designation to TSND-201 (methylone) for PTSD treatment, potentially expediting its development and review process.
  • Phase 2 trial results showed TSND-201 achieved rapid and durable PTSD symptom improvements, with significant placebo-adjusted CAPS-5 improvements by day 10.
  • The designation addresses an urgent unmet need, as only two FDA-approved PTSD medications exist with modest efficacy for 13 million affected Americans.
  • Transcend Therapeutics plans to initiate Phase 3 trials for this rapid-acting neuroplastogen that works on monoamine transporters without hallucinogenic effects.

Genmab Advances Two Phase 3 and Phase 2 Trials Testing Acasunlimab Combinations in Lung Cancer and Melanoma

Oncology
  • Genmab is conducting a Phase 3 trial evaluating acasunlimab combined with pembrolizumab versus docetaxel in PD-L1 positive metastatic non-small cell lung cancer patients who progressed after prior PD-1/PD-L1 inhibitor and platinum-containing chemotherapy.
  • The company is simultaneously running a Phase 2 study testing acasunlimab as monotherapy and in combination with pembrolizumab for advanced melanoma patients who progressed after checkpoint inhibitor treatment.
  • Both trials represent significant opportunities to establish new treatment options for challenging cancer types with limited therapeutic alternatives, potentially impacting Genmab's market position and stock performance.

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