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Krystal Biotech Initiates Phase 3 Trial of KB803 Gene Therapy for Corneal Complications in Dystrophic Epidermolysis Bullosa

Rare Disease
  • Krystal Biotech has dosed the first patient in its Phase 3 IOLITE trial evaluating KB803 eye drop gene therapy for treating corneal abrasions in dystrophic epidermolysis bullosa patients.
  • The crossover study will enroll approximately 16 subjects and aims to demonstrate KB803's efficacy in reducing corneal abrasion symptoms compared to placebo over 24 weeks.
  • Previous compassionate use data showed dramatic results, with one patient achieving full corneal healing at three months and visual acuity improvement from hand motion to 20/25 by eight months.
  • Over 25% of DEB patients suffer from ocular complications, with no current corrective therapies available beyond supportive wound care and surgical interventions.

A Study Assessing KB803 Compared to Matching Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa

NCT07016750RecruitingPhase 3
Krystal Biotech, Inc.
Posted 6/20/2025

A Study Assessing KB801 for the Treatment of Stage 2 or 3 Neurotrophic Keratitis

NCT06999733RecruitingPhase 1
Krystal Biotech, Inc.
Posted 5/1/2025

Sterotherapeutics Doses First Patient in Phase 2 Trial of ST-002 for Cushing's Syndrome

Orphan DrugRare Disease
  • Sterotherapeutics has successfully dosed the first patient in its Phase 2 clinical trial evaluating ST-002 for Cushing's Syndrome treatment.
  • The multicenter European study aims to assess safety, efficacy, and tolerability of ST-002 in patients with this rare endocrine condition caused by chronic excess cortisol exposure.
  • ST-002 has received FDA Orphan Drug Designation and addresses a significant unmet medical need, as no universally effective treatment currently exists for Cushing's Syndrome.
  • The trial will evaluate complications including diabetes and MASLD using magnetic resonance imaging and targeted measurements across multiple European sites.

Lundbeck's Lu AG13909 Receives Orphan Drug Designation for Congenital Adrenal Hyperplasia Treatment

Monoclonal AntibodyRare Disease
  • Lundbeck received orphan drug designation from both the FDA and EMA for Lu AG13909, a novel humanized monoclonal antibody targeting ACTH for congenital adrenal hyperplasia treatment.
  • The first-in-class anti-ACTH antibody represents an innovative therapeutic approach for CAH, a rare genetic disorder affecting approximately 1 in 14,000-18,000 live births worldwide.
  • An expanded Phase I/II clinical trial is currently enrolling adults with classic CAH across North America and seven European countries, with monthly intravenous dosing.
  • Animal studies demonstrated significant and durable reductions in corticosterone/cortisol and aldosterone levels with no adverse effects after six months of treatment.

CRISPR Base Editing Shows Promise for Treating Fatal Pediatric Vascular Disease MSMDS

Gene EditingRare Disease
  • Researchers from Mass General Brigham and UT Southwestern have developed customized CRISPR-Cas9 base editing therapies that successfully treat multisystemic smooth muscle dysfunction syndrome (MSMDS) in preclinical models.
  • The bespoke gene-editing approach extended survival four-fold in mouse models and prevented disease symptoms when administered early, targeting the ACTA2 gene mutation that causes this lethal pediatric condition.
  • Both research teams have engaged with the FDA for clinical trial preparation, with Mass General Brigham securing rare disease designations to accelerate development toward human testing.
  • The breakthrough represents the first CRISPR-based therapeutic specifically designed to target vascular smooth muscle cells, potentially opening new treatment avenues for other genetic vascular diseases.

Cure Rare Disease Advances LGMDR9 Gene Therapy Following Positive FDA Pre-IND Meeting

Rare Disease
  • Cure Rare Disease completed a successful pre-IND meeting with the FDA for CRD-003, a gene therapy targeting Limb-Girdle Muscular Dystrophy type 2I/R9 using a novel AAVMYO2 capsid.
  • The FDA endorsed the company's clinical trial design and biomarker strategy while agreeing that a 4-month murine toxicology study could support first-in-human trials.
  • The agency suggested CRD-003 may be eligible for accelerated approval based on Phase I/II results, marking a significant regulatory milestone for this rare neuromuscular disease treatment.

Arcturus Therapeutics to Present Phase 2 Data for ARCT-810 mRNA Therapy in Rare Genetic Disorder

Rare DiseaseOrphan Drug
  • Arcturus Therapeutics will host a virtual presentation on June 30, 2025, featuring Phase 2 interim data for ARCT-810, an investigational mRNA therapeutic for ornithine transcarbamylase deficiency.
  • The presentation will review safety and pharmacodynamics data from multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency, the most common urea cycle disorder.
  • Leading experts Dr. Marshall Summar and Dr. Johannes Häberle will participate in the presentation, bringing decades of expertise in urea cycle disorders and metabolic medicine.
  • ARCT-810 has received multiple regulatory designations including Orphan Drug status and Fast Track designation from the FDA for treating this rare genetic condition affecting approximately 10,000 people in Europe and the U.S.

Cumberland Pharmaceuticals Reports Positive Phase 2 Results for Ifetroban in Duchenne Muscular Dystrophy Heart Disease

Rare DiseaseOrphan Drug
  • Cumberland Pharmaceuticals' Phase 2 FIGHT DMD trial demonstrated that high-dose ifetroban treatment resulted in a significant 5.4% improvement in left ventricular ejection fraction compared to control groups.
  • The study showed reduced blood levels of cardiac damage markers (NT-proBNP and cardiac troponin I) in patients receiving ifetroban, while these markers increased in placebo-treated patients.
  • Ifetroban represents a potential breakthrough for DMD heart disease, which is the leading cause of death in DMD patients and currently has no approved targeted treatments.
  • All patients who completed the 12-month study opted to continue with the open-label extension, demonstrating confidence in the treatment approach.

Oral Ifetroban in Subjects with Duchenne Muscular Dystrophy

NCT03340675Active, Not RecruitingPhase 2
Cumberland Pharmaceuticals
Posted 10/19/2020

MBX Biosciences Strengthens Leadership with Endocrinology Expert to Advance Hypoparathyroidism Treatment

Rare Disease
  • MBX Biosciences appointed Andreas Moraitis, M.D., as Senior Vice President of Clinical Development to lead advancement of canvuparatide for hypoparathyroidism treatment.
  • Dr. Moraitis brings over a decade of experience from Corcept Therapeutics, where he led clinical programs in rare endocrine diseases and supported NDA filing for relacorilant.
  • The appointment strengthens MBX's clinical capabilities as canvuparatide progresses through Phase 2 development for chronic hypoparathyroidism, addressing significant unmet medical needs.
  • MBX's pipeline includes multiple candidates targeting endocrine and metabolic disorders, with canvuparatide as the lead product candidate and additional programs in obesity and post-bariatric hypoglycemia.

Novadip Receives FDA RMAT Designation for NVD003 Regenerative Therapy in Rare Pediatric Bone Disease

Orphan DrugRare Disease
  • Novadip Biosciences received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for NVD003, an autologous stem cell therapy for congenital pseudarthosis of the tibia (CPT).
  • Clinical data showed 88% of patients achieved fracture healing with NVD003, including children whose prior surgeries had failed.
  • The company plans to launch a pivotal phase 3 trial this month with recruitment in the US and Europe, targeting market entry by 2027.
  • NVD003 represents potential peak sales of $1.4 billion for large bone defects in pediatric and adult patients.

NVD-003 in the Treatment of Congenital Pseudarthrosis of the Tibia

NCT05693558Active, Not RecruitingPhase 1
Novadip Biosciences
Posted 11/24/2022

CLINUVEL CEO Returns to Drive US Expansion with Vitiligo Treatment Approval Strategy

Drug ApprovalRare DiseaseLeadership Change
  • CEO Dr. Philippe Wolgen has returned to lead CLINUVEL Pharmaceuticals following his full recovery, positioning the company for its next growth phase in North America.
  • The company is pursuing approval of SCENESSE® for vitiligo treatment in the United States and Canada, which could represent a significant shift in CLINUVEL's business model.
  • CLINUVEL is accelerating US market presence through targeted M&A activities, with 104 of 120 EPP treatment centers currently active and ready for potential vitiligo patient treatment.
  • Analysts maintain a Buy recommendation with AUD 22.00 target price, representing 126% upside potential from current share price levels.

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