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Philikos Initiates Phase 1/2 Trial of T-Guard for Diffuse Cutaneous Systemic Sclerosis

Monoclonal AntibodyOrphan Drug
  • Philikos has enrolled the first patient in a Phase 1/2 trial evaluating T-Guard for diffuse cutaneous systemic sclerosis (dcSSc), a severe autoimmune disorder with limited treatment options.
  • The open-label study will assess safety and preliminary efficacy in 12 early-stage dcSSc patients whose disease remains refractory despite prior immunosuppressive therapy.
  • T-Guard, administered as four infusions over one week, aims to provide a safer alternative to hematopoietic stem cell transplantation by selectively depleting disease-associated T cells and NK cells.

FDA Greenlights Promontory Therapeutics' Phase 3 Trial Design for PT-112 in Metastatic Prostate Cancer

OncologyOrphan Drug
  • Promontory Therapeutics has successfully completed an End of Phase 2 meeting with the FDA, reaching agreement on key aspects of a registrational Phase 3 trial for PT-112 in metastatic castration-resistant prostate cancer.
  • The FDA approved the proposed dosing regimen, patient population, study comparator, and endpoints, with an interim analysis provision that could allow for drug approval before study completion.
  • Preliminary clinical outcomes from the Phase 2 trial of PT-112 will be presented at the upcoming ASCO 2025 Annual Meeting on June 2nd, following recent presentation of immune response biomarker data at AACR 2025.

PT-112 in Subjects With Thymoma and Thymic Carcinoma

NCT05104736RecruitingPhase 2
National Cancer Institute (NCI)
Posted 4/6/2022

A Study Evaluating the Safety, Pharmacokinetics, and Clinical Effects of Intravenously Administered PT-112 Injection in Subjects With Advanced Solid Tumors and Subsequent Dose Expansion Cohorts

NCT02266745Active, Not RecruitingPhase 2
Promontory Therapeutics Inc.
Posted 7/1/2014

Be Biopharma Initiates First-in-Human Trial of CRISPR-Based Gene Therapy for Haemophilia B

Orphan DrugRare Disease
  • Be Biopharma has dosed the first patient in the BeCoMe-9 Phase I/II trial of BE-101, marking the first clinical application of B Cell Medicines technology for haemophilia B treatment.
  • BE-101 uses CRISPR/Cas9 gene editing to insert the human Factor IX gene into patients' own B cells, designed to continuously secrete Factor IX without requiring preconditioning or immunosuppression.
  • The two-part trial will enroll up to 24 subjects with moderately severe to severe haemophilia B, with participants monitored for 52 weeks to evaluate safety and clinical activity.
  • The therapy has received FDA fast-track and orphan drug designations, positioning it as a potentially transformative treatment option for this rare bleeding disorder.

BeCoMe-9: a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B

NCT06611436RecruitingPhase 1
Be Biopharma
Posted 12/4/2024

Juvena Therapeutics Initiates First-in-Human Trial of JUV-161 for Muscle Regeneration in Myotonic Dystrophy

Orphan Drug
  • Juvena Therapeutics has begun enrolling participants in the first human clinical trial of JUV-161, a novel fusion protein designed to enhance muscle regeneration for treating Myotonic Dystrophy Type 1 and sarcopenia.
  • JUV-161, described as "insulin for muscle," works by restoring AKT signaling pathways that regulate muscle growth and metabolism, offering a unique approach compared to existing RNA-targeting or gene therapy strategies.
  • The therapy was discovered using Juvena's proprietary JuvNET platform, which combines AI and stem cell secretome biology to identify therapeutic proteins with regenerative potential.

EMA Designates Allopurinol as First Orphan Drug for Marfan Syndrome

Orphan DrugRare Disease
  • The European Medicines Agency has designated allopurinol as the first orphan drug for Marfan syndrome, a rare connective tissue disease affecting approximately 7 in 100,000 people in the European Union.
  • Researchers from the University of Barcelona, IDIBAPS, and CIBERER have demonstrated allopurinol's potential to halt and prevent aortic aneurysms in animal models, with international clinical trials in patients planned for the future.
  • This repurposing of allopurinol, currently used for gout treatment, represents a significant advancement for Marfan syndrome patients who currently have no curative options beyond limited palliative treatments and high-risk surgical interventions.

AKANTIOR® Receives UK Marketing Authorization as First Approved Treatment for Acanthamoeba Keratitis

Orphan DrugDrug ApprovalRare Disease
  • SIFI's AKANTIOR® (polihexanide 0.08%) has received both Marketing Authorization and Promising Innovative Medicine designation from the UK's MHRA, marking it as the first approved treatment for Acanthamoeba keratitis.
  • The approval confirms AKANTIOR's Orphan Drug Designation and New Active Substance status, recognizing its efficacy against an ultra-rare corneal infection that can lead to blindness if untreated.
  • Following its European approval in August 2024, this UK authorization represents a significant advancement for patients with this devastating eye infection, with SIFI planning to file for NICE reimbursement by June 2025.

China Approves First Domestically Developed Enzyme Replacement Therapy for Gaucher Disease

Orphan DrugDrug ApprovalRare Disease
  • CANbridge Pharmaceuticals has received NMPA approval for velaglucerase-beta (Gaurunning), China's first domestically developed enzyme replacement therapy for Type I and III Gaucher disease in patients aged 12 and above.
  • The pivotal clinical trial demonstrated statistically significant reductions in spleen volume at both 60 U/kg (P<0.0001) and 30 U/kg (P<0.001) doses, meeting its primary efficacy endpoint.
  • Developed in collaboration with WuXi Biologics, Gaurunning represents a breakthrough in rare disease treatment in China, potentially improving accessibility and affordability for the estimated 3,000 Chinese Gaucher disease patients.

NICE Extends Access to Brineura for CLN2 Batten Disease While Seeking Long-term Solution

Rare DiseaseOrphan Drug
  • NICE has secured continued access to cerliponase alfa (Brineura) for current patients and those starting treatment before December 2025, despite not recommending it for routine NHS use due to cost concerns.
  • The enzyme replacement therapy, costing over £500,000 per patient annually, has demonstrated effectiveness in slowing CLN2 progression in the short term, but lacks sufficient long-term efficacy data.
  • NICE, NHS England, and manufacturer BioMarin continue negotiations to reach a sustainable pricing agreement that could extend access to all future patients with this rare, life-limiting condition.

Avadel Pharmaceuticals Appoints Susan Rodriguez as Chief Operating Officer to Drive LUMRYZ Commercial Expansion

Rare DiseaseOrphan Drug
  • Avadel Pharmaceuticals has appointed Susan Rodriguez as Chief Operating Officer to lead commercial strategy and operations for LUMRYZ, the first once-at-bedtime oxybate treatment for narcolepsy.
  • Rodriguez brings over 30 years of life sciences experience, including leadership roles at Ardelyx, Tolmar Pharmaceuticals, and Abbott, with expertise in rare disease commercialization.
  • The appointment comes as Avadel scales to meet increasing patient demand for LUMRYZ and works toward potential label expansion to idiopathic hypersomnia.
  • LUMRYZ has received FDA approval for both adult and pediatric narcolepsy patients and holds 7 years of Orphan Drug Exclusivity due to clinical superiority over existing treatments.

Nanoscope Therapeutics Showcases MCO-010 Optogenetic Therapy Data at ASGCT 2025

Orphan Drug
  • Nanoscope Therapeutics presented multiple research presentations at ASGCT 2025 highlighting the safety and efficacy of MCO-010 optogenetic therapy for retinal degenerative diseases.
  • The company reported positive therapeutic benefits in Stargardt disease patients similar to durable vision gains previously observed in retinitis pigmentosa patients.
  • MCO-010 represents the first mutation-agnostic therapy shown to restore vision in advanced RP patients via a one-time intravitreal injection.
  • The company plans to initiate BLA submission for MCO-010 to treat RP in 2025 following positive Phase 2b RESTORE trial results.

Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease

NCT05417126CompletedPhase 2
Nanoscope Therapeutics Inc.
Posted 7/5/2022

Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

NCT04945772CompletedPhase 2
Nanoscope Therapeutics Inc.
Posted 7/13/2021

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