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Clinical Trial News

Diagonal Therapeutics' DIAG723 Shows Disease-Modifying Potential for Hereditary Hemorrhagic Telangiectasia in Preclinical Studies

Rare Disease
  • Diagonal Therapeutics will present preclinical data at EHA 2025 Congress demonstrating their clustering antibody approach can restore ALK1-mediated cellular signaling and prevent arteriovenous malformations in HHT models.
  • The company's bispecific antibody DIAG723 improved survival from 25% to 100% in mouse models and prevented HHT-associated anemia and heart enlargement.
  • DIAG723 has received orphan drug designation from both the US FDA and EMA for treating HHT, a rare bleeding disorder affecting over 150,000 people with no approved therapies.

FDA Approves Expanded Pediatric Trial for Moleculin's Annamycin in Acute Myeloid Leukemia

Orphan Drug
  • FDA has approved Moleculin's pediatric study plan for Annamycin in combination with cytarabine as second-line therapy for children with relapsed/refractory acute myeloid leukemia.
  • The regulatory agency recommended including patients as young as 6 months old, younger than Moleculin's original proposal of 2 years, expanding potential treatment access.
  • Annamycin aims to eliminate cardiotoxicity associated with anthracyclines, which are used to treat approximately 60% of children with cancer.
  • The pediatric trial is planned to begin in the second half of 2027, while the ongoing Phase 3 MIRACLE trial in adults continues with initial data expected in late 2025.

L-Annamycin for Injection in Combination With Cytarabine Injection as Second Line Therapy for Remission Induction in Adult Subjects With Refractory/Relapsed AML

NCT06788756RecruitingPhase 2
Moleculin Biotech, Inc.
Posted 3/12/2025

FDA Approves First Integrated Sacral Neuromodulation System for Urinary Urge Incontinence

FDA Approval
  • The FDA has approved Neuspera Medical's integrated sacral neuromodulation (iSNM) system, the first battery-free device for treating urinary urge incontinence (UUI).
  • The pivotal SANS-UUI trial demonstrated that 84.2% of 128 patients achieved at least 50% reduction in urgent leaks, with 42% of responders becoming completely dry.
  • The innovative system uses a miniaturized implanted neurostimulator activated by an external disc worn for 2 hours daily, eliminating complications from implanted batteries.
  • The approval addresses a significant unmet need for approximately 1 in 5 women in the U.S. affected by overactive bladder conditions.

Neuspera's Implantable Sacral Nerve Stimulation System in Patients With Symptoms of Urinary Urgency Incontinence (UUI)

NCT04232696Active, Not RecruitingNot Applicable
Neuspera Medical, Inc.
Posted 12/13/2019

Health Canada Approves Expanded Dosing and Pediatric Indication for FIRDAPSE in Lambert-Eaton Myasthenic Syndrome

Drug ApprovalRare Disease
  • Health Canada has approved Kye Pharmaceuticals' supplemental new drug submission for FIRDAPSE (amifampridine), allowing some patients with Lambert-Eaton myasthenic syndrome to benefit from an increased maximum daily dose of up to 100mg.
  • The expanded approval includes pediatric dosing guidance for patients 6 years of age and older, providing pediatric neurologists with a proven treatment option for children diagnosed with LEMS.
  • FIRDAPSE is a potassium channel blocker that works by increasing acetylcholine release at the neuromuscular junction, helping improve muscle function in patients with this rare autoimmune disorder.
  • The label update offers healthcare providers and patients greater flexibility in treatment regimens for managing LEMS, a condition characterized by muscle weakness and fatigue.

MD Anderson Study Identifies Three B-Cell Lymphoma Subgroups with Distinct CD19 CAR-T Response Patterns

CAR-TPrecision Medicine
  • Researchers at MD Anderson Cancer Center analyzed 232 patients with large B-cell lymphoma to identify three distinct tumor microenvironment subgroups with different responses to CD19 CAR-T therapy.
  • The study profiled over 1.8 million cells and validated findings using ZUMA-7 Phase III trial data, revealing that the lymph node subgroup benefits most from CAR-T therapy while the T cell exhausted group shows no significant benefit.
  • The findings provide a precision medicine framework to guide treatment selection and identify patients who may need alternative therapeutic approaches beyond CAR-T cell therapy.
  • The research opens opportunities for targeted therapies in development to address the needs of patients in subgroups with poorer CAR-T outcomes.

ReGelTec Initiates U.S. Pivotal Trial for HYDRAFIL System in Chronic Low Back Pain Treatment

FDA Approval
  • ReGelTec has enrolled the first patient in its HYDRAFIL-D FDA investigational device exemption clinical trial, marking a significant milestone for the percutaneous hydrogel implant system designed to treat chronic low back pain caused by degenerative disc disease.
  • The 225-patient, multicenter, randomized controlled trial follows promising European clinical data showing more than 80% improvement in disability scores and greater than 70% reduction in pain scores sustained for two years in 75 patients.
  • The HYDRAFIL System addresses a critical treatment gap for the more than 10 million Americans with degenerative disc disease, offering a minimally invasive outpatient alternative to major spine surgery performed under local anesthesia.

Biogen Launches Phase 3 Pediatric Trial of Omaveloxolone for Friedreich Ataxia

Rare DiseaseOrphan Drug
  • Biogen has initiated the BRAVE study, a global Phase 3 clinical trial evaluating omaveloxolone in children aged 2 to 16 years with Friedreich ataxia.
  • The study will enroll approximately 255 pediatric participants and use the Upright Stability Score as the primary endpoint to measure disease progression.
  • This represents the first potential treatment option for pediatric Friedreich ataxia patients, addressing a critical unmet medical need in this vulnerable population.
  • Omaveloxolone is currently approved as SKYCLARYS for adults and adolescents aged 16 and older in over 40 countries worldwide.

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

NCT06623890Recruiting
Biogen
Posted 12/12/2024

A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

NCT06953583RecruitingPhase 3
Biogen
Posted 6/9/2025

Scholar Rock's Apitegromab Preserves Lean Muscle Mass During Weight Loss with Tirzepatide in Phase 2 Trial

Monoclonal Antibody
  • Scholar Rock's Phase 2 EMBRAZE trial demonstrated that apitegromab preserved 54.9% more lean mass compared to tirzepatide alone, adding 4.2 pounds of lean muscle during weight loss treatment.
  • Patients receiving the combination therapy achieved higher quality weight loss with 85% fat mass reduction versus 70% fat mass reduction with tirzepatide alone over 24 weeks.
  • The myostatin inhibitor apitegromab was well tolerated with no serious adverse events related to treatment, addressing concerns about muscle loss associated with GLP-1 obesity medications.
  • The positive results validate Scholar Rock's platform for developing selective myostatin inhibitors while the company remains focused on apitegromab's potential approval for spinal muscular atrophy.

Actio Biosciences Raises $66M Series B to Advance Rare Disease Programs Targeting Ion Channel Disorders

Rare DiseaseOrphan DrugPrecision Medicine
  • Actio Biosciences secured $66 million in Series B funding co-led by Regeneron Ventures and Deerfield Management to advance two lead programs targeting rare genetic diseases.
  • The company's most advanced program, ABS-0871, is currently in Phase 1 trials for Charcot-Marie-Tooth disease type 2C, with potential expansion to overactive bladder treatment.
  • ABS-1230, targeting KCNT1-related epilepsy affecting approximately 2,500 individuals in the U.S., received FDA rare pediatric and orphan drug designations and is expected to enter Phase 1 trials in late 2025.
  • The company's "inclusive precision medicine" approach aims to develop treatments that address multiple mutations within the same genetic pathway, potentially benefiting broader patient populations.

Aligos Therapeutics Strengthens CMC Leadership with Industry Veteran Ahead of Phase 2 Trial

  • Aligos Therapeutics has appointed Kieron Wesson, PhD as Vice President, Head of Chemistry Manufacturing Controls (CMC), bringing over 20 years of pharmaceutical development experience to the company.
  • The appointment comes at a strategic time as Aligos prepares to begin its Phase 2 study of ALG-000184 in mid-2025 for liver and viral diseases.
  • Dr. Wesson previously led CMC operations at Kezar Life Sciences and AN2 Therapeutics, and supported the approval and commercial launches of Kerydin® and Eucrisa® at Anacor Pharmaceuticals.
  • The hire reinforces Aligos' commitment to advancing its pipeline of therapeutics targeting chronic hepatitis B virus infection, metabolic dysfunction-associated steatohepatitis (MASH), and coronaviruses.

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