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Clinical Trial News

Annexon's Vonaprument Selected for EMA's Product Development Coordinator Pilot Program for Dry AMD Treatment

  • The European Medicines Agency has selected Annexon's vonaprument to participate in its new Product Development Coordinator pilot program, providing enhanced regulatory support for the potential first-in-class dry AMD treatment.
  • Vonaprument is the only investigational therapy in geographic atrophy to demonstrate significant vision preservation on best corrected visual acuity and low luminance visual acuity endpoints in clinical trials.
  • The Phase 3 ARCHER II trial has completed enrollment with over 630 patients, with topline data expected in the second half of 2026 for this potential breakthrough therapy.
  • Geographic atrophy affects approximately one million people in the United States and eight million globally, with no currently approved therapies shown to significantly prevent vision loss.

FDA Approves First Liquid Embolic System for Hypervascular Tumor Treatment

Drug Approval
  • The FDA has granted premarket approval to the Embrace Hydrogel Embolic System, marking the first liquid embolic specifically approved for hypervascular tumor embolization based on randomized controlled trial data.
  • The PEG hydrogel system demonstrated 88.6% technical success and 99% freedom from major adverse events at 30 days in a pivotal study of 150 patients across 22 institutions.
  • The system addresses critical limitations of traditional embolic agents by enabling deep penetration into vessels as small as 10 microns while avoiding catheter entrapment and imaging artifacts.
  • Hypervascular tumors in the liver, kidney, and bone present significant surgical challenges due to bleeding risk, making this new embolization option particularly valuable for non-surgical candidates.

Instylla HES Hypervascular Tumor Pivotal Study

NCT04523350CompletedNot Applicable
Instylla, Inc.
Posted 1/4/2021

Sequence LifeScience Receives IRB Approval for Novel Matched Controls Trial in Diabetic Foot Ulcers

  • Sequence LifeScience and SerenaGroups Inc. received IRB approval for a multicenter clinical trial evaluating placental membrane allografts in treating diabetic foot ulcers.
  • The innovative matched controls design uses historical data to reduce control group sizes and accelerate promising therapies to patients.
  • The study will enroll over 300 participants across 30 U.S. sites with primary endpoint of complete wound healing at 12 weeks.
  • Patient recruitment begins July 2025 with results expected in third quarter 2026, addressing suboptimal healing rates in diabetic foot ulcers.

SELLAS Life Sciences Receives Positive IDMC Recommendation to Continue Phase 3 REGAL Trial of Galinpepimut-S in AML

  • The Independent Data Monitoring Committee completed a pre-specified analysis of SELLAS' Phase 3 REGAL trial and recommended continuing the study without modification, finding no safety concerns.
  • The trial enrolled 126 AML patients who achieved complete remission following second-line salvage therapy, with final analysis expected by year-end upon occurrence of 80 events.
  • Galinpepimut-S targets the WT1 protein and represents a potential therapeutic advance for acute myeloid leukemia patients in complete remission after salvage therapy.
  • The positive IDMC recommendation validates the risk-benefit profile of GPS and supports continued evaluation under the current study protocol.

Galinpepimut-S Versus Investigator's Choice of Best Available Therapy for Maintenance in AML CR2/CRp2

NCT04229979Active, Not RecruitingPhase 3
Sellas Life Sciences Group
Posted 2/8/2021

Quince Therapeutics Partners with Option Care Health to Streamline eDSP Commercialization for Rare Disease Treatment

Rare DiseaseOrphan Drug
  • Quince Therapeutics has formed a strategic partnership with Option Care Health to support the commercial development of encapsulated dexamethasone sodium phosphate (eDSP) for Ataxia-Telangiectasia treatment.
  • The collaboration leverages Option Care Health's network of 90+ specialty pharmacies and 180+ ambulatory infusion suites to provide nationwide access to eDSP administration.
  • eDSP is currently in Phase 3 NEAT trial with 105 patients enrolled and topline results expected in Q1 2026, potentially becoming the first approved therapy for A-T.
  • The partnership positions Quince to scale into additional rare disease markets, including Duchenne muscular dystrophy, while reducing operational complexity and costs.

Strand Therapeutics Raises $153M Series B to Advance Programmable mRNA Cancer Therapies

  • Strand Therapeutics closed a $153 million Series B funding round led by Kinnevik, with participation from major pharmaceutical companies including Eli Lilly, Amgen, and Regeneron Ventures.
  • The company's lead candidate STX-001, a self-replicating mRNA therapy encoding IL-12, showed promising Phase I results including multiple RECIST responses and complete responses in treatment-resistant solid tumor patients.
  • Strand's programmable mRNA platform uses genetic circuits to control location, timing, and degree of therapeutic expression, with STX-003 representing the world's first systemically administrable mRNA therapy with tumor targeting.
  • The funding brings Strand's total raised capital to over $250 million and will support advancement of their pipeline targeting cancer and other chronic conditions.

Phase 1/2 Study of Intratumoral Injection of STX-001 in Advanced Solid Tumors as Monotherapy or in Combination With Pembrolizumab

NCT06249048RecruitingPhase 1
Strand Therapeutics Inc.
Posted 5/3/2024

Cardiol Therapeutics Stock Plunges 20% as CBD-Based CardiolRx Misses Primary Endpoints in Myocarditis Trial

  • Cardiol Therapeutics' cannabidiol-based therapy CardiolRx failed to meet co-primary endpoints in the Phase II ARCHER trial for myocarditis, with extracellular volume reduction missing statistical significance (p=0.0538).
  • The company's stock dropped 20% from $1.35 to $1.07 following the trial results, despite CEO David Elsley expressing satisfaction with the outcomes.
  • While CardiolRx showed some cardiac improvements including significant left ventricular mass reduction, it failed to improve global longitudinal strain scores compared to placebo.
  • The company continues advancing its Phase III MAVERIC trial for recurrent pericarditis and plans to integrate ARCHER findings into broader clinical development strategy.

Impact of CardiolRx on Myocardial Recovery in Patients With Acute Myocarditis

NCT05180240CompletedPhase 2
Cardiol Therapeutics Inc.
Posted 6/22/2022

CardiolRx in Recurrent Pericarditis Following IL-1 Blocker Cessation

NCT06708299RecruitingPhase 3
Cardiol Therapeutics Inc.
Posted 4/7/2025

FDA-Approved Asthma Drug Zileuton Shows Promise in Preventing Food Allergy Reactions

  • Northwestern Medicine researchers discovered that Zileuton, an FDA-approved asthma drug, nearly eliminated life-threatening allergic reactions in mice by blocking a previously unknown pathway involving the DPEP1 gene.
  • The treatment reversed susceptibility from 95% to 95% protection, with mice showing almost no symptoms of anaphylaxis after receiving Zileuton before peanut extract exposure.
  • The discovery identifies cysteinyl leukotrienes in the gut as key regulators of food allergy reactions, offering a new therapeutic approach for the 33 million Americans affected by food allergies.
  • An early-stage clinical trial launched in July 2025 will test whether this pathway-blocking approach is as effective in humans as demonstrated in the preclinical studies.

Annovis Bio Secures Comprehensive Patent Protection for Crystal Buntanetap Through 2046

  • Annovis Bio has successfully transferred all patents from the original semi-crystalline form of buntanetap to cover the new crystalline form, securing comprehensive intellectual property protection through 2046.
  • The company now maintains 13 patent families filed internationally, covering both forms of buntanetap including composition of matter, mechanism of action, multiple indications, and drug combinations.
  • The crystalline form demonstrates improved solid-state stability with bioequivalent pharmacokinetic properties compared to the original form, as presented at AAIC 2025.
  • FDA has approved the use of crystal buntanetap for the ongoing pivotal Phase 3 clinical trial in early Alzheimer's disease, with over 75 sites secured across the US.

A Double-blind Dual Study Assessing Safety and Efficacy of Buntanetap in Participants With Early AD

NCT06709014RecruitingPhase 3
Annovis Bio Inc.
Posted 2/4/2025

Tegoprazan Demonstrates Superior Long-Term GERD Management in Phase 3 TRIUMpH Study

  • Tegoprazan achieved statistically superior maintenance of healing compared to lansoprazole across all grades of erosive esophagitis, including the most severe cases, in a 24-week maintenance phase study.
  • Both 100mg and 50mg doses of the novel P-CAB demonstrated non-inferiority and superiority to lansoprazole 15mg for sustained healing, with favorable safety profiles and normal gastrin levels.
  • Sebela Pharmaceuticals plans to file New Drug Applications with the FDA for both erosive esophagitis and non-erosive reflux disease indications in Q4 2025.
  • The TRIUMpH program enrolled over 2,000 patients across two large Phase 3 studies, representing a demographically diverse US population with GERD.

A Study to Evaluate the Efficacy and Safety of BLI5100 in Patients With Non-Erosive Reflux Disease

NCT05587322CompletedPhase 3
Braintree Laboratories
Posted 9/6/2022

A Study to Evaluate the Efficacy and Safety of BLI5100 in Patients with Erosive Esophagitis

NCT05587309Active, Not RecruitingPhase 3
Braintree Laboratories
Posted 10/30/2022

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