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Clinical Trial News

Aurion Biotech Launches Vyznova (neltependocel) in Japan for Bullous Keratopathy

• Aurion Biotech has commercially launched Vyznova (neltependocel) in Japan, the first approved allogeneic cell therapy for corneal endothelial disease. • Vyznova offers a new treatment option for bullous keratopathy, addressing the critical shortage of donor corneas for traditional transplants. • Clinical trials in the U.S. and Canada are underway, with the FDA granting Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations to Aurion Biotech. • Vyznova's development stems from over 25 years of research pioneered by Professor Shigeru Kinoshita at Kyoto Prefecture University of Medicine.

A Phase 1/2 Study of AURN001 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction (ABA-1)

NCT06041256Active, Not RecruitingPhase 1
Aurion Biotech
Posted 10/18/2023

KSQ Therapeutics' CRISPR-Engineered TIL Therapy, KSQ-004EX, Receives FDA IND Clearance for Advanced Solid Tumors

• KSQ Therapeutics received FDA clearance for its IND application for KSQ-004EX, a CRISPR/Cas9-engineered tumor-infiltrating lymphocyte (TIL) therapy. • KSQ-004EX targets advanced solid tumors by inactivating SOCS1 and Regnase-1 genes in TILs, enhancing anti-tumor potency and persistence. • A Phase 1/2 open-label, dose-escalation study will assess the safety, tolerability, and anti-tumor activity of KSQ-004EX in select advanced solid tumors. • Preclinical data suggests KSQ-004EX demonstrates enhanced anti-tumor function in solid tumor models refractory to PD-1 inhibition.

Pakistan Gears Up for CPEC Phase II with Focus on Economic Growth

  • Pakistan's Finance Minister announced the imminent commencement of the second phase of the China-Pakistan Economic Corridor (CPEC).
  • CPEC Phase II will prioritize economic growth, industrialization, and agricultural development across Pakistan.
  • The initiative aims to enhance Pakistan's infrastructure and connectivity, fostering trade and investment opportunities.
  • The commencement of CPEC Phase II signifies strengthened economic ties between Pakistan and China.

UNITY Biotechnology to Highlight Foselutoclax (UBX1325) Data at Ophthalmology Day

• UNITY Biotechnology will host an Ophthalmology Day on October 15, 2024, focusing on its ASPIRE study. • The ASPIRE study evaluates foselutoclax (UBX1325) against aflibercept for diabetic macular edema (DME). • Twenty-four-week safety and efficacy data from the ASPIRE study are expected in Q1 2025. • Key opinion leaders will discuss UBX1325's potential in addressing unmet needs in DME.

Univo IRB Expands Services with Canadian Research Ethics Board Review

  • Univo IRB has launched Canadian Research Ethics Board (REB) review services to support clinical trials across North America, ensuring high ethical and regulatory standards.
  • The new service aims to streamline the REB approval process for researchers and sponsors, facilitating more efficient clinical trials in compliance with Canadian guidelines.
  • Univo IRB's Canadian REB includes experts in ethics, law, and science, guided by the Tri-Council Policy Statement for ethical research involving humans.
  • This expansion reaffirms Univo IRB's commitment to advancing research responsibly, unifying clinical research with patient protection, education, and advocacy.

Silexion's LODER Shows Promise in Non-Resectable Pancreatic Cancer

  • Silexion Therapeutics' LODER demonstrated a 56% objective response rate in patients with non-resectable pancreatic cancer bearing KRAS G12D/V mutations.
  • The objective response rate increased to 67% in patients whose tumors became resectable following treatment with LODER.
  • Patients treated with LODER plus chemotherapy showed a 9.3-month improvement in overall survival compared to chemotherapy alone.
  • Silexion is advancing SIL-204, a next-generation therapy targeting a broader range of KRAS mutations with enhanced stability.

Zevra Therapeutics' KP1077 Shows Promise in Phase 2 Trial for Idiopathic Hypersomnia

  • Zevra Therapeutics' KP1077 (serdexmethylphenidate) demonstrated clinically meaningful improvements in key symptoms of idiopathic hypersomnia (IH) in a Phase 2 trial.
  • The Phase 2 trial was a placebo-controlled, double-blind, randomized withdrawal study evaluating the safety and efficacy of KP1077 in IH patients.
  • Top-line data from the trial will inform the design of a Phase 3 study, marking a significant step in developing a potential new treatment for IH.
  • KP1077 has been granted Orphan Drug Designation by both the FDA and the European Commission for the treatment of idiopathic hypersomnia.

Placebo-Controlled, Double-Blind, Study to Determine the Safety and Efficacy of SDX in Patients With IH

NCT05668754CompletedPhase 2
Zevra Therapeutics
Posted 12/28/2022

Wave Life Sciences' WVE-N531 Shows Promise in Duchenne Muscular Dystrophy Trial

  • Wave Life Sciences announced positive interim data from its FORWARD-53 trial of WVE-N531 in Duchenne Muscular Dystrophy (DMD) patients amenable to exon 53 skipping.
  • The trial showed a mean muscle content-adjusted dystrophin expression of 9.0% and improved muscle health biomarkers with bi-weekly 10 mg/kg dosing.
  • WVE-N531 demonstrated a favorable safety profile and a 61-day tissue half-life, supporting potential monthly dosing.
  • Wave Life Sciences plans to discuss accelerated approval pathways with regulators, with complete 48-week data expected in Q1 2025.

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT04281485Active, Not RecruitingPhase 3
Pfizer
Posted 11/5/2020

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03406780CompletedPhase 2
Capricor Inc.
Posted 4/4/2018

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT05126758Active, Not RecruitingPhase 3
Capricor Inc.
Posted 6/22/2022

Open-label Study of WVE-N531 in Patients with Duchenne Muscular Dystrophy (FORWARD-53)

NCT04906460Active, Not RecruitingPhase 1
Wave Life Sciences Ltd.
Posted 9/28/2021

The Effect of RNS60 on ALS Biomarkers

NCT03456882CompletedPhase 2
Mario Negri Institute for Pharmacological Research
Posted 5/30/2017

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372Active, Not RecruitingPhase 2
Pfizer
Posted 8/8/2022

UCB and Biogen's Dapirolizumab Pegol Shows Promise in Phase 3 Lupus Trial

  • UCB and Biogen's dapirolizumab pegol met the primary endpoint in a Phase 3 trial for moderate-to-severe systemic lupus erythematosus (SLE).
  • The drug demonstrated significant clinical improvement in disease activity compared to placebo when added to standard of care.
  • A second Phase 3 trial, PHOENYCS FLY, is planned to further evaluate the efficacy and safety of dapirolizumab pegol in SLE patients.
  • Dapirolizumab pegol targets the CD40L pathway, offering a novel approach to reduce B cell activation and autoantibody production in SLE.

A Study to Evaluate the Efficacy and Safety of Dapirolizumab Pegol in Study Participants With Moderately to Severely Active Systemic Lupus Erythematosus

NCT04294667CompletedPhase 3
UCB Biopharma SRL
Posted 8/12/2020

RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus

NCT06121297RecruitingPhase 1
Cabaletta Bio
Posted 2/16/2024

United Therapeutics to Present Tyvaso DPI Data at CHEST 2024

  • United Therapeutics will present data from the BREEZE open-label extension study of Tyvaso DPI, detailing long-term outcomes in pulmonary arterial hypertension patients.
  • The company is sponsoring a symposium on Tyvaso DPI, focusing on clinical pearls and drug-device characteristics, at the CHEST 2024 Annual Meeting.
  • Additional presentations will cover patient-reported outcomes, healthcare resource utilization, and the impact of inhaled treprostinil in pulmonary hypertension.
  • United Therapeutics also sponsors the Women in Chest Medicine Annual Luncheon and the Advanced Practice Providers (APPs) in Chest Medicine Forum.

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