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Clinical Trial News

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.

AbbVie's Atogepant Demonstrates Superior Tolerability and Efficacy Over Topiramate in Phase 3 Migraine Prevention Trial

  • AbbVie's Phase 3 TEMPLE study shows atogepant (QULIPTA/AQUIPTA) achieved significantly fewer treatment discontinuations due to adverse events compared to topiramate for migraine prevention.
  • The study met all primary and secondary endpoints, with 64.1% of atogepant patients achieving ≥50% reduction in monthly migraine days versus 39.3% on topiramate.
  • Results support CGRP pathway inhibitors as first-line preventive treatment options for migraine, addressing significant unmet medical needs in this debilitating neurological condition.

Comparative Study of Oral Atogepant Versus Oral Topiramate to Assess Adverse Events in Adult Participants With Migraine

NCT05748483Active, Not RecruitingPhase 3
AbbVie
Posted 10/7/2023

A Study of Eptinezumab in Participants With Migraine and Medication Overuse Headache

NCT05452239CompletedPhase 4
H. Lundbeck A/S
Posted 7/1/2022

Draig Therapeutics Emerges from Stealth with $140M to Advance Novel Neuropsychiatric Therapies

  • Draig Therapeutics launched with $140 million in Series A funding to develop next-generation treatments for neuropsychiatric disorders, marking the largest commercial investment in Welsh research to date.
  • The company's lead candidate DT-101, an AMPA receptor positive allosteric modulator for major depressive disorder, will enter Phase 2 trials in 2025 following successful Phase 1 results in over 60 subjects.
  • Founded by Cardiff University professors targeting glutamate and GABA neurotransmitter pathways, Draig aims to address significant unmet medical needs in neuropsychiatric conditions with improved safety profiles.
  • The funding round was led by Access Biotechnology with participation from major investors including Canaan Partners, SR One, and Sanofi Ventures, supporting advancement of multiple pipeline candidates.

SK Biopharmaceuticals Partners with PhnyX Lab to Deploy AI-Powered Drug Development Platform

AI
  • SK Biopharmaceuticals signed a strategic agreement with AI startup PhnyX Lab to establish an AI-powered drug development framework using the Cheiron generative AI platform.
  • The collaboration will automate key processes including literature searches, data analysis, and regulatory documentation for clinical trial initiation to reduce development time and costs.
  • The partnership represents what SK Biopharmaceuticals calls a "full-scale AI transformation" of its drug development workflow, building on its existing Huble AI platform.
  • Cheiron integrates with official regulatory databases from the FDA and Korea's Ministry of Food and Drug Safety to deliver high accuracy in pharmaceutical operations.

Senate Republicans Drop Orphan Drug Exemptions from Trump Tax Bill, Dealing Blow to Rare Disease Drug Development

Rare Disease
  • Senate Republicans excluded the Orphan Cures Act from their version of Trump's "One Big Beautiful Bill Act," removing provisions that would have exempted multi-indication orphan drugs from Medicare price negotiations.
  • The bipartisan Orphan Cures Act aimed to address concerns that current Inflation Reduction Act provisions discourage rare disease drug development, with data showing a 48% drop in drugs receiving second orphan designations post-IRA.
  • Patient advocacy groups celebrated the Senate's decision, arguing the act was an industry attempt to evade necessary drug price negotiations and maintain lower costs for Medicare beneficiaries.
  • The Senate's departure from the House version indicates further legislative revisions are likely before Republicans' July 4 target deadline for final passage.

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