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Clinical Trial News

Retension Pharmaceuticals Secures $15M Series B to Advance Novel Hypertension Drug RTN-001 into Phase 2b Trial

  • Retension Pharmaceuticals completed a $15 million Series B financing round to advance RTN-001, a novel PDE5 inhibitor designed for uncontrolled and resistant hypertension treatment.
  • The company received a new U.S. patent extending RTN-001 protection through at least 2044, covering preferred crystalline polymorph forms of the drug candidate.
  • A Phase 2b trial will commence in Q4 2025, enrolling approximately 280 patients to evaluate a once-daily modified-release formulation designed for sustained 24-hour blood pressure control.
  • RTN-001 has demonstrated clinically significant blood pressure reductions in 243 human subjects across previous Phase 2 studies, targeting patients on up to four other antihypertensive medications.

Relay Therapeutics Advances RLY-2608 to Phase 3 Trial with Promising Breast Cancer Data

Oncology
  • Relay Therapeutics initiated the Phase 3 ReDiscover-2 trial for RLY-2608, a pan-mutant selective PI3Kα inhibitor targeting breast cancer patients with high unmet medical needs.
  • Updated ASCO 2025 data showed RLY-2608 achieved a median progression-free survival of 10.3 months and 39% objective response rate in PI3Kα-mutated, HR+/HER2- metastatic breast cancer patients.
  • The company reduced R&D expenses by 30.5% year-over-year while maintaining $656.8 million in cash reserves, projected to fund operations into 2029.

BioMendics Launches TAMES-02 Trial for First-in-Class EB Simplex Therapy TolaSure

Rare DiseaseOrphan Drug
  • BioMendics has initiated the TAMES-02 clinical trial, a multicenter, randomized, double-blind, placebo-controlled study evaluating TolaSure, a first-in-class topical therapy for patients with generalized intermediate to severe Epidermolysis Bullosa Simplex.
  • The trial builds on promising TAMES-01 results and represents a potential breakthrough for the 30,000-50,000 patients worldwide affected by this rare inherited blistering disorder, which currently lacks disease-modifying treatment options.
  • TolaSure works by targeting mutant keratin proteins responsible for skin fragility, offering a novel mechanism to reinforce fragile keratinocytes and directly address the root cause of blister formation.
  • The study is conducted in partnership with Stanford University and Northwestern University, enrolling patients aged 4 and older with coverage for travel costs to ensure broad access across the United States and internationally.

Cassava Sciences Appoints Joseph Hulihan as Chief Medical Officer to Lead Simufilam Development for TSC-Related Epilepsy

  • Cassava Sciences has appointed Dr. Joseph Hulihan as Chief Medical Officer, bringing over 25 years of experience in neurological disorder therapeutics development.
  • Hulihan will focus on advancing simufilam, an investigational oral small molecule targeting filamin A protein for treating Tuberous Sclerosis Complex-related epilepsy.
  • The first clinical study for simufilam in TSC-related epilepsy is expected to begin in the first half of 2026, following positive preclinical results.
  • Hulihan previously served as CMO at Marinus Pharmaceuticals, where he developed ganaxolone for seizure disorders including TSC-related epilepsy.

Arcellx Reports Strong CAR-T Clinical Data Despite 72% Revenue Drop in Q2 2025

CAR-TFDA Approval
  • Arcellx's lead CAR-T therapy anito-cel achieved a 97% overall response rate and 93.3% minimal residual disease negativity in 117 multiple myeloma patients from the Phase 2 iMMagine-1 study.
  • The company's Q2 2025 collaboration revenue dropped 72% to $7.6 million, missing analyst expectations by 43.8% due to completion of clinical trial manufacturing activities.
  • Net loss more than doubled to $52.8 million as the company increased commercial readiness spending ahead of a planned 2026 product launch.
  • FDA authorized an investigational new drug application for ACLX-004, expanding Arcellx's pipeline into acute myeloid leukemia treatment.

Ignite Proteomics Partners with Vanderbilt to Advance Breast Cancer Immunotherapy Biomarker Discovery

  • Ignite Proteomics and Vanderbilt University Medical Center have launched a one-year research collaboration to characterize protein-signaling pathways that help breast tumor cells evade immune system detection.
  • The partnership will utilize Ignite's reverse-phase protein array platform to analyze up to 80 tumor specimens, profiling priority targets including MHC-I/II, PD-L1, AKT/mTOR, and EGFR/HER2 signaling pathways.
  • The collaboration aims to identify actionable biomarkers that could refine immunotherapy strategies, addressing the current limitation where objective response rates to immune checkpoint blockade remain below 40 percent.
  • Initial data packages are expected within two months of sample receipt, with Ignite receiving first rights to negotiate exclusive licensing for any jointly developed intellectual property.

FibroGen Receives FDA Approval for Phase 3 Trial of Roxadustat in Lower-Risk MDS Patients with High Transfusion Burden

FDA Approval
  • FibroGen has reached agreement with the FDA on key design elements for a pivotal Phase 3 trial of roxadustat in lower-risk myelodysplastic syndrome patients with high transfusion burden.
  • The oral HIF-PH inhibitor demonstrated promising efficacy in a post-hoc analysis, with 36% of high transfusion burden patients achieving transfusion independence compared to 7% on placebo.
  • The randomized, placebo-controlled trial will enroll approximately 200 patients and evaluate 8-week or 16-week transfusion independence as the primary endpoint.
  • FibroGen plans to submit the Phase 3 protocol to the FDA in the fourth quarter of 2025, addressing a significant unmet medical need in this patient population.

Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden

NCT03263091TerminatedPhase 3
FibroGen
Posted 1/29/2018

Iovance Reports Strong Q2 2025 Results with $60M Revenue as Amtagvi Adoption Accelerates

  • Iovance Biotherapeutics achieved $60.0 million in total product revenue for Q2 2025, representing a 93% year-over-year increase driven by strong Amtagvi adoption.
  • More than 100 patients were treated with Amtagvi in Q2 2025, with real-world data showing a 48.8% objective response rate in commercial use.
  • The company implemented strategic restructuring to extend cash runway into Q4 2026 while maintaining full pipeline development programs.
  • Health Canada approval for Amtagvi is expected imminently, marking the first ex-U.S. regulatory milestone for the groundbreaking TIL therapy.

Daxor Receives FDA Clearance for Next-Generation Blood Volume Analyzer with 3X Faster Analysis

  • Daxor Corporation received FDA 510(k) clearance for its next-generation rapid, compact, hand-held Blood Volume Analyzer (BVA) that delivers results three times faster than its predecessor.
  • The device addresses volume derangement affecting millions of patients annually, including over seven million heart failure patients and 7.4 million critical care admissions requiring precise fluid management.
  • Peer-reviewed studies demonstrate BVA-guided care reduced heart failure one-year mortality by 86% and ICU mortality by 66% in septic/ARDS patients.
  • The lightweight 7-pound device enables broader clinical use across cardiology, nephrology, critical care, and emergency medicine settings where resources are limited.

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.

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