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Clinical Trial News

XEOMIN Receives First-Ever Approval in Japan for Chronic Sialorrhea Treatment

Drug ApprovalRare Disease
  • Teijin Pharma has received approval from Japan's Ministry of Health, Labor and Welfare for XEOMIN (incobotulinumtoxinA) to treat chronic sialorrhea, marking the first drug approved for this indication in Japan.
  • The approval was based on Phase III clinical trials conducted by Merz Therapeutics in Germany and Poland, as well as trials conducted by Teijin in Japan.
  • XEOMIN works by inhibiting acetylcholine release from cholinergic nerve endings, effectively suppressing saliva secretion and reducing water and electrolyte secretion from salivary glands.
  • Chronic sialorrhea significantly impacts patients' quality of life, causing social isolation and difficulties with speaking and eating, while placing heavy burdens on families and caregivers.

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Episodic Migraine

NCT07018700RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Chronic Migraine

NCT07018713RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

FDA Approves Enhanced Labeling for Endari Based on Post-Marketing Pharmacokinetic Data

Drug ApprovalRare Disease
  • Emmaus Life Sciences received FDA approval for enhanced labeling of Endari (L-glutamine oral powder) based on additional post-marketing pharmacokinetic study data.
  • The updated label provides more comprehensive prescribing information, including confirmation of body weight-based dosing, no unwanted accumulation with twice-daily dosing, and flexible administration with or without food.
  • Endari remains approved to reduce acute complications of sickle cell disease in adult and pediatric patients five years and older, addressing a condition affecting approximately 100,000 people in the United States.
  • The label enhancement represents a significant improvement in clinical guidance for healthcare providers treating sickle cell disease patients with this prescription-grade L-glutamine therapy.

IMCD Acquires Trichem to Strengthen Pharmaceutical Supply Chain in India's Generic Drug Market

  • IMCD N.V. has acquired 100% of Trichem Healthcare Private Limited and its subsidiaries to expand its pharmaceutical distribution capabilities in India and the Middle East.
  • Trichem, founded in 1998 and based in Mumbai, generates approximately INR 1.7 billion (EUR 18 million) annually through supplying active pharmaceutical ingredients and pharmaceutical intermediates.
  • The acquisition positions IMCD to capitalize on India's role as a major supplier of generic drugs globally while adding regulatory expertise for highly regulated pharmaceutical markets.

ZyVersa Therapeutics Initiates Phase 2a Trial for First-in-Class Diabetic Kidney Disease Treatment VAR 200

  • ZyVersa Therapeutics has activated its first clinical site in San Antonio, Texas, to begin patient recruitment for a Phase 2a trial of VAR 200, a potential first-in-class treatment targeting renal lipotoxicity in diabetic kidney disease.
  • VAR 200 addresses an unmet medical need by targeting excess cholesterol and lipid accumulation in kidney glomeruli, a neglected pathogenic pathway with no currently available therapies.
  • The 16-week open-label study will evaluate VAR 200's efficacy and safety in patients with type 2 diabetes and diabetic kidney disease, with preliminary data expected in H2-2025.
  • Preclinical studies demonstrated VAR 200's ability to reduce cholesterol and lipid levels, protect against renal injury and fibrosis, and improve proteinuria across multiple kidney disease models.

C7 Neurotomy Combined with Speech Therapy Shows Superior Results for Chronic Post-Stroke Aphasia

  • A clinical trial published in The BMJ demonstrates that combining C7 neurotomy neck surgery with intensive speech therapy produces significantly greater improvements in communication abilities for chronic post-stroke aphasia patients compared to speech therapy alone.
  • The intervention group showed an average 11.16-point increase in Boston Naming Test scores at one month versus 2.72 points in the control group, with improvements remaining stable at six months.
  • Patients receiving the combined treatment also experienced significant improvements in quality of daily life and reduced post-stroke depression, with no serious adverse events reported at six months.
  • The study involved 50 Chinese patients aged 40-65 with chronic aphasia and right arm spasticity following left-brain stroke, though researchers acknowledge limitations in generalizability due to the specific patient population.

ProFound Therapeutics Partners with Novartis in $775M Cardiovascular Drug Discovery Deal

Drug Discovery
  • ProFound Therapeutics announced a four-year strategic collaboration with Novartis to discover novel cardiovascular therapeutics using its ProFoundry Platform to mine the expanded human proteome.
  • The partnership includes $25 million in upfront and near-term milestone payments, with potential downstream milestones worth $750 million per selected target plus tiered royalties.
  • The collaboration combines ProFound's capabilities in identifying novel proteins from the expanded proteome with Novartis' cardiovascular drug development expertise to address unmet medical needs.
  • The ProFoundry Platform uses advanced computational tools and biological data to uncover previously unknown proteins as potential drug targets from the expanded human proteome.

Phase 3 Trial Demonstrates Selumetinib Efficacy in Adults with Neurofibromatosis Type 1

Rare Disease
  • The KOMET phase 3 trial published in The Lancet shows selumetinib significantly reduces tumor size in one in five adults with neurofibromatosis type 1 plexiform neurofibromas.
  • This represents the first effective pharmacological treatment for adult NF1 patients, who previously relied solely on complex surgical interventions with limited success.
  • The randomized, double-blind study included 145 adult participants worldwide and demonstrated rapid pain reduction alongside decreased need for analgesics.
  • The innovative trial design allowed placebo participants to access selumetinib under specific conditions, enabling comprehensive efficacy assessment across different treatment timelines.

Ovid Therapeutics Sells Ganaxolone Royalty Rights to Immedica Pharma for $7 Million

Rare Disease
  • Ovid Therapeutics has entered into a definitive agreement with Immedica Pharma AB to sell its future royalty rights for ganaxolone sales outside China for $7 million in cash.
  • The transaction provides non-dilutive funding to support Ovid's ongoing operations, while Immedica strengthens its focus on ganaxolone by acquiring additional intellectual property rights.
  • Ganaxolone is an approved medicine in the EU, Great Britain, US, and China for treating epileptic seizures associated with CDKL5 deficiency disorder in patients aged 2-17 years.
  • Immedica will also assume financial responsibility for all costs related to the licensed intellectual property portfolio and plans to expand the license to include additional indications.

Former Pfizer CSO Mikael Dolsten Joins Two Biotech Boards, Advancing Gene Editing and Mast Cell Therapeutics

Gene EditingRare Disease
  • Dr. Mikael Dolsten, former Pfizer Chief Scientific Officer, has joined the boards of Arbor Biotechnologies and MC Sciences as both companies advance novel therapeutic platforms.
  • Dolsten's 16-year tenure at Pfizer included advancing over 150 drug candidates into clinical studies and leading regulatory approval of 36 medicines and vaccines.
  • Arbor Biotechnologies is progressing its lead gene editing program ABO-101 for primary hyperoxaluria type 1 into clinical trials.
  • MC Sciences is developing first-in-class mast cell-targeting therapeutics for diseases including chronic urticaria, systemic mastocytosis, and asthma.

Real-World Study Reveals Only 8% of Patients Continue GLP-1 Obesity Treatment After Three Years

Real World Evidence
  • Prime Therapeutics' three-year analysis of 5,780 commercially insured adults found only 8.1% remained on GLP-1 obesity medications throughout the study period.
  • High-potency obesity-approved drugs like semaglutide (Wegovy) showed better persistence at 14.3% compared to daily liraglutide at 2.5% over three years.
  • One-year persistence rates improved dramatically from 33.2% in 2021 to 62.7% in 2024, largely attributed to resolved drug shortages and better clinical management.
  • The study highlights the critical need for comprehensive care management programs and behavioral interventions to support long-term obesity treatment success.

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