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Clinical Trial News

Embolization Inc. Receives FDA Clearance for Novel Nonmetal Vascular Coil Device

  • Embolization Inc.'s nitinol-enhanced device (NED) has received FDA 510(k) clearance for arterial and venous embolization in peripheral vasculature.
  • The device utilizes shape-memory biocompatible polymers to achieve better vascular occlusion while minimizing MRI and CT imaging artifacts compared to traditional metal devices.
  • Animal studies demonstrated reduced imaging shadowing, easier repeat embolizations, faster and more reliable embolization, and improved ease of use through a 1-mm diameter implantation tool.
  • The Boulder-based company is now moving toward commercialization and market introduction of the innovative embolization technology.

Cellarity Initiates Phase 1 Trial of CLY-124, First-in-Class Oral Therapy for Sickle Cell Disease

AIRare Disease
  • Cellarity has dosed the first patient in a Phase 1 clinical trial of CLY-124, a novel oral medicine that increases fetal hemoglobin through a globin-switching mechanism to treat sickle cell disease.
  • The drug was discovered using AI-powered transcriptomic analysis and demonstrated the ability to increase fetal hemoglobin above 20% in preclinical studies without cytotoxicity.
  • CLY-124 represents a potential breakthrough for sickle cell disease treatment, offering a once-daily oral alternative to current therapies that carry dose-limiting toxicity.
  • The Phase 1 global trial will evaluate safety, tolerability, and pharmacokinetics in healthy volunteers before advancing to sickle cell disease patients.

Altimmune Shares Plunge 60% Despite Positive MASH Trial Results as Fibrosis Endpoint Falls Short

  • Altimmune's stock crashed over 60% after its Phase 2b IMPACT trial of pemvidutide for MASH showed mixed results, with strong MASH resolution but insufficient fibrosis improvement.
  • The drug achieved statistically significant MASH resolution in 59.1% of patients at 1.2 mg dose versus 19.1% on placebo, along with meaningful weight loss of up to 6.2%.
  • Despite meeting the primary endpoint, investors were disappointed by the lack of statistical significance in fibrosis reduction, a critical benchmark for MASH treatment success.
  • Altimmune plans to meet with the FDA for an End-of-Phase 2 meeting by late 2025 and is considering a Phase 3 trial, while analysts maintain a "Strong Buy" rating.

Provectus' PV-10 Immunotherapy Shows Promise in Head and Neck Cancer Through Novel Cell Death Mechanism

  • Moffitt Cancer Center researchers published preclinical findings showing PV-10 triggers immunogenic cell death in head and neck squamous cell carcinoma models via endoplasmic reticulum stress and apoptosis.
  • The study published in Molecular Cancer Therapeutics reveals novel mechanistic insights that could support future clinical development of PV-10 for locally recurrent head and neck cancer.
  • Provectus plans to advance its rose bengal sodium-based immunotherapy platform with a Phase 1 trial in penile squamous cell carcinoma potentially later this year at Moffitt Cancer Center.

Ontario Expands Public Coverage of XTANDI for High-Risk Prostate Cancer Patients

Oncology
  • Ontario's Drug Benefit Program now covers XTANDI (enzalutamide) for non-metastatic castration-sensitive prostate cancer patients with high-risk biochemical recurrence, making it the first androgen receptor pathway inhibitor reimbursed for this indication in the province.
  • The decision follows Health Canada's expedited approval of XTANDI for this indication in January 2024 through Project Orbis, providing faster access to promising cancer treatments.
  • XTANDI is now publicly reimbursed in Ontario for all of its approved prostate cancer indications, addressing a critical need as about 9 out of 10 men with high-risk biochemical recurrence develop metastatic disease.
  • The expanded access removes barriers for physicians to provide comprehensive care to prostate cancer patients, particularly important given that prostate cancer accounts for 76 new diagnoses daily among Canadian men.

GenSight Biologics Successfully Transfers LUMEVOQ Manufacturing to Catalent for LHON Gene Therapy

Rare Disease
  • GenSight Biologics has successfully transferred the upstream manufacturing process for LUMEVOQ, its gene therapy for Leber Hereditary Optic Neuropathy (LHON), to manufacturing partner Catalent Inc.
  • The partnership is expected to improve yield and upgrade analytical methods ahead of clinical use and regulatory submissions for the rare mitochondrial disease treatment.
  • Catalent manufactured a drug product batch released as safe for human use in November 2024, which will supply France's named patient early access program and dose-ranging study.
  • The collaboration positions GenSight for its planned global Phase III RECOVER trial beginning in 2026 and upcoming regulatory submissions to FDA and EMA.

Blood Test Shows Promise for Early Detection of Heart Transplant Rejection, Could Replace 80% of Invasive Biopsies

  • Researchers have developed a donor-derived cell-free DNA blood test that outperformed traditional tissue biopsies in detecting acute heart transplant rejection in a study of 171 patients.
  • The test can detect rejection as early as 28 days after transplantation and at least three months before current biopsy methods, potentially eliminating up to 80% of invasive procedures.
  • The blood test showed particular promise for detecting antibody-mediated rejection, one of the deadliest forms of rejection that is difficult to diagnose with current methods.
  • The noninvasive test could provide significant financial savings and safer monitoring during viral outbreaks like COVID-19, while helping address health disparities in transplant outcomes.

China Approves First Once-Daily Nebulized LAMA for COPD, Triggering $7.5M Milestone for Theravance

Drug Approval
  • China's NMPA has approved YUPELRI (revefenacin) as the first once-daily nebulized long-acting muscarinic antagonist for maintenance treatment of chronic obstructive pulmonary disease.
  • The regulatory approval triggers a $7.5 million milestone payment from Viatris to Theravance Biopharma, with additional sales milestones up to $37.5 million and tiered royalties of 14-20% on net sales.
  • Viatris assumes full responsibility for YUPELRI's launch and commercialization in China, while Theravance incurs no commercial costs in the region.
  • The approval strengthens Theravance's financial position, which includes $131 million in cash and potential near-term milestone payments from multiple revenue streams.

Phase 3 Efficacy and Durability of Ampreloxetine for the Treatment of Symptomatic nOH in Participants With Multiple System Atrophy

NCT05696717Active, Not RecruitingPhase 3
Theravance Biopharma
Posted 6/27/2023

Lisata's Certepetide Shows Promising Results in Phase 2b Pancreatic Cancer Trial

  • Lisata Therapeutics' certepetide demonstrated significant improvements in progression-free survival and response rates when combined with standard chemotherapy in metastatic pancreatic cancer patients.
  • The ASCEND Phase 2b trial's Cohort B showed 60.8% six-month progression-free survival for certepetide-treated patients versus 25% for placebo, with median progression-free survival of 7.5 months versus 4.7 months.
  • The dual-dose regimen achieved a 45.2% objective response rate compared to 19% for placebo, supporting continued development of this novel tumor-penetrating therapy.

The ASCEND Study: Gemcitabine and Nab-Paclitaxel With LSTA1 (Certepetide) or Placebo in Patients With Untreated Metastatic Pancreatic Ductal Adenocarcinoma

NCT05042128Active, Not RecruitingPhase 2
Australasian Gastro-Intestinal Trials Group
Posted 4/13/2022

Oxford Scientists Develop Single-Shot Malaria Vaccine Using Programmable Microcapsule Technology

  • University of Oxford researchers have developed a programmable microcapsule delivery system that enables complete malaria vaccination in a single injection, eliminating the need for booster visits.
  • The chip-based microfluidics technology uses biodegradable capsules that release booster doses weeks or months after initial injection, matching multi-dose regimen efficacy in preclinical mouse trials.
  • The scalable manufacturing approach is compatible with existing pharmaceutical production methods and could transform immunization access in communities with limited healthcare infrastructure.
  • The innovation addresses three key challenges in delayed vaccine delivery: programmability, injectability, and scalability, with potential applications beyond malaria to other multi-dose vaccines.

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