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Clinical Trial News

UCLA Scientists Achieve First-in-Human Reprogramming of Stem Cells to Generate Renewable Cancer-Fighting T Cells

CAR-T
  • UCLA researchers successfully demonstrated the first-in-human reprogramming of blood-forming stem cells to continuously produce cancer-targeting T cells in a groundbreaking clinical trial.
  • The approach creates an "internal factory" that generates tumor-targeting immune cells over time, potentially offering longer-lasting protection against cancer recurrence.
  • One patient with aggressive sarcoma showed tumor regression and maintained detectable levels of engineered immune cells for months following treatment.
  • The therapy targets NY-ESO-1, a cancer-testis antigen found in approximately 80% of synovial sarcomas but rarely in healthy adult tissues.

Genetically Engineered PBMC and PBSC Expressing NY-ESO-1 TCR After a Myeloablative Conditioning Regimen to Treat Patients With Advanced Cancer

NCT03240861TerminatedPhase 1
Jonsson Comprehensive Cancer Center
Posted 7/26/2017

Immunophotonics Completes Phase 1b/2a Trial of IP-001 Immunotherapy for Advanced Solid Tumors

  • Immunophotonics has completed treatment of the last patient in its 41-patient INJECTABL-1 Phase 1b/2a trial evaluating IP-001 for advanced solid tumors across three cancer types.
  • The multicenter trial tested IP-001, a proprietary glycan polymer designed to transform tumor ablation into personalized systemic cancer immunotherapies by retaining tumor antigens and activating immune responses.
  • IP-001 is administered by injection into the ablation zone following tumor destruction, aiming to enable systemic tumor surveillance and reduce recurrence after standard ablation therapy.
  • The company reports early positive signals and has expanded clinical collaborations to further assess the novel therapy's efficacy in addressing unmet medical needs.

Serina Therapeutics Advances Once-Weekly Injectable VMAT2 Inhibitor for Tardive Dyskinesia Treatment

  • Serina Therapeutics announced the advancement of SER-270 (POZ-VMAT2i), a novel once-weekly subcutaneous VMAT2 inhibitor designed to address medication adherence challenges in tardive dyskinesia patients.
  • The drug candidate targets underserved patient populations including those non-compliant with daily oral medications, institutionalized patients, and individuals with swallowing difficulties.
  • The U.S. tardive dyskinesia market exceeded $3.7 billion in 2024 and is projected to reach $5.4 billion by 2030, despite fewer than 30% of patients being diagnosed.
  • Serina plans to explore POZ-VMAT2i development for Huntington's disease chorea as a secondary indication, leveraging advantages for patients with swallowing difficulties.

FDA Approves Leqvio as First-Line Monotherapy for Cholesterol Management

Drug Approval
  • The FDA has approved an updated label for Novartis' Leqvio (inclisiran) allowing its use as monotherapy alongside diet and exercise to reduce LDL cholesterol in adults with hypercholesterolemia.
  • The approval removes the previous requirement for Leqvio to be used in combination with statin therapy, positioning it as a first-line treatment option.
  • Up to 80% of atherosclerotic cardiovascular disease patients in the US fail to reach guideline-recommended LDL cholesterol targets below 70 mg/dL.
  • Leqvio's twice-yearly dosing schedule administered by healthcare providers is designed to improve patient adherence and long-term cholesterol management.

A Randomized Study to Evaluate the Effect of an "Inclisiran First" Implementation Strategy Compared to Usual Care in Patients With Atherosclerotic Cardiovascular Disease and Elevated LDL-C Despite Receiving Maximally Tolerated Statin Therapy (VICTORION-INITIATE)

NCT04929249CompletedPhase 3
Novartis Pharmaceuticals
Posted 6/25/2021

Efficacy and Safety of Inclisiran as Monotherapy in Patients With Primary Hypercholesterolemia Not Receiving Lipid-lowering Therapy.

NCT05763875CompletedPhase 3
Novartis Pharmaceuticals
Posted 3/15/2023

Vir Biotechnology's Dual-Masked T-Cell Engagers Show Promise in Solid Tumors and mCRPC

  • Vir Biotechnology's VIR-5818 and VIR-5500, dual-masked T-cell engagers, demonstrate encouraging preliminary safety and efficacy in Phase 1 trials.
  • VIR-5818 targets HER2-expressing solid tumors, showing dose-dependent tumor shrinkage in heavily pretreated patients, including those with colorectal cancer.
  • VIR-5500 targets PSMA in metastatic castration-resistant prostate cancer (mCRPC), with dose escalation ongoing and early clinical response signals observed.
  • The PRO-XTEN masking technology aims to selectively activate TCEs in the tumor microenvironment, potentially mitigating toxicity to healthy cells.

To Access the Safety and Effects of Intravenous Administration of AMX-818 Alone and in Combination With Pembrolizumab in Adult Participants With Locally Advanced or Metastatic HER2-Expressing Cancers

NCT05356741RecruitingPhase 1
Vir Biotechnology, Inc.
Posted 4/13/2022

Safety and Preliminary Efficacy of VIR-5525 and VIR-5525 + Pembrolizumab in Participants With Locally Advanced or Metastatic Solid Tumors

NCT06960395RecruitingPhase 1
Vir Biotechnology, Inc.
Posted 7/22/2025

BioCryst Names Charlie Gayer as New CEO Following Jon Stonehouse's Retirement Announcement

Leadership ChangeRare Disease
  • BioCryst Pharmaceuticals announced that CEO Jon Stonehouse will retire on December 31, 2025, with current Chief Commercial Officer Charlie Gayer set to succeed him as president in August and CEO in January 2026.
  • Gayer led the successful commercial launch of ORLADEYO (berotralstat), the first oral prophylactic therapy for hereditary angioedema, driving exceptional revenue growth with expected peak sales of $1 billion.
  • The leadership transition comes as BioCryst has matured into a profitable company generating increasing cash resources through ORLADEYO revenue while advancing a promising pipeline of rare disease therapies.
  • Gayer brings over 10 years of experience at BioCryst and extensive rare disease commercial expertise from previous roles at Talecris Biotherapeutics, Grifols, and GlaxoSmithKline.

Frazier Healthcare Partners Closes $1.3 Billion Fund for Early-Stage Biotech Investments

Oncology
  • Frazier Healthcare Partners has successfully closed a $1.3 billion venture capital fund specifically targeting early-stage biotechnology companies and startup creation.
  • The firm was one of the most active biotech investors in 2024, participating in 17 deals and leading nearly one-third of those transactions.
  • Recent successful exits include Scorpion Therapeutics' $2.5 billion drug sale to Eli Lilly and major acquisitions like Novartis' $3.5 billion purchase of Chinook Therapeutics.
  • The new fund launch comes amid declining biotech venture funding, which dropped from $7 billion to $4.8 billion in Q2 2025 according to HSBC Innovation Banking.

Altasciences Partners with VoxCell BioInnovation to Integrate 3D Bioprinting Technology into Preclinical Drug Development

  • Altasciences and VoxCell BioInnovation have formed a strategic collaboration to enhance preclinical research by integrating 3D bioprinted tissue platforms into early-phase drug development services.
  • The partnership combines Altasciences' comprehensive drug development capabilities with VoxCell's cutting-edge 3D tissue technology to create more predictive and human-relevant preclinical testing environments.
  • VoxCell's proprietary bioprinted tissues closely mimic human tumors and biological structures, offering advanced platforms for evaluating drug efficacy, safety, and toxicity before animal or human studies.
  • The collaboration will initially focus on drug screening for ocular indications, with plans to expand into additional therapeutic areas and applications.

Protopia AI Secures $1.25M Air Force Contract to Advance Privacy-Preserving AI Technology for Secure Code Development

AI
  • Protopia AI received a $1.25 million Direct-to-Phase II SBIR contract from the U.S. Air Force to demonstrate its Stained Glass Transform technology for secure AI-powered software development.
  • The technology enables Air Force teams to use coding large language models without exposing proprietary codebases in plain text, addressing critical security concerns in AI adoption.
  • This partnership with the Air Force Sustainment Center Software Directorate represents a significant milestone for privacy-preserving AI applications in mission-critical environments.
  • The contract highlights the growing need for dual-use technologies that enable secure generative AI adoption across both military and commercial applications.

University of Colorado Launches $29 Million EFFECT Trial to Evaluate Early Fibrinogen Replacement in Trauma Care

  • The University of Colorado Anschutz Medical Campus received a $29 million grant from Octapharma to lead the EFFECT Trial, evaluating early fibrinogen replacement in trauma patients with life-threatening bleeding.
  • The multicenter randomized trial will enroll 800 participants across 12 top-tier trauma centers, representing the first U.S. clinical trial to rigorously evaluate fibrinogen concentrate as a frontline trauma intervention.
  • The study aims to address hemorrhage, the primary cause of preventable death after traumatic injury, by targeting fibrinogen depletion which occurs first among coagulation factors.
  • Results could transform trauma resuscitation protocols for both civilian and military care, potentially influencing global trauma treatment guidelines.

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