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JCR Pharmaceuticals Completes Enrollment in Global Phase III Trial for Blood-Brain Barrier-Penetrating Hunter Syndrome Therapy

Rare Disease
  • JCR Pharmaceuticals has achieved full enrollment in its global Phase III clinical trial for JR-141 (pabinafusp alfa), a novel enzyme replacement therapy designed to treat the neurological symptoms of Hunter syndrome.
  • The therapy utilizes JCR's proprietary J-Brain Cargo technology to deliver iduronate-2-sulfatase across the blood-brain barrier, addressing an unmet medical need for central nervous system symptoms in Hunter syndrome patients.
  • JR-141 is already approved in Japan and represents the first-ever enzyme replacement therapy worldwide that can penetrate the blood-brain barrier to treat lysosomal storage disorders.
  • The Phase III trial is ongoing across the United States, Latin America, and Europe, targeting a rare disease that affects an estimated 2,000-3,000 individuals worldwide.

Neurocrine Biosciences Advances CAH Treatment Portfolio with Long-Acting Injectable Therapy

Rare DiseaseOrphan Drug
  • Neurocrine Biosciences initiated Phase 1 trials for NBIP-01435, a long-acting injectable CRF1 receptor antagonist for congenital adrenal hyperplasia (CAH), complementing its recently approved oral therapy CRENESSITY.
  • CRENESSITY demonstrated significant clinical benefits in Phase 3 trials, achieving 27% glucocorticoid dose reduction in adults and 18% in pediatric patients while maintaining hormonal control.
  • The dual-mechanism approach positions Neurocrine to capture the majority of the CAH market, projected to exceed $1.5 billion by 2030, with regulatory protections including seven years of market exclusivity.
  • Q1 2025 sales of CRENESSITY reached $14.5 million with 413 patient starts, while analysts project $500 million in annual sales by 2027.

First Oral Drug MA-5 Shows Promise for Rare Barth Syndrome in Preclinical Studies

Rare Disease
  • Researchers at Tohoku University discovered that oral drug MA-5 can improve both heart and muscle problems in Barth syndrome, a rare genetic disorder affecting 1 in 300,000 births worldwide.
  • MA-5 boosted cellular energy production by up to 50% and protected cells from oxidative stress-induced death by enhancing interactions between mitochondrial proteins mitofilin and ATP synthase.
  • The drug dramatically improved climbing ability and normalized elevated heart rates in Drosophila models, while correcting abnormal mitochondrial structures in human muscle cells derived from patient iPS cells.
  • Phase I clinical trials in Japan have been completed successfully, with Phase II trials being prepared, making MA-5 the first oral medication for Barth syndrome to progress to clinical trial stage.

Soligenix Completes Manufacturing Transfer of Synthetic Hypericin for Rare Skin Cancer Treatment

Orphan DrugRare Disease
  • Soligenix successfully transferred manufacturing of synthetic hypericin from Europe to the U.S. through partnership with Sterling Pharma Solutions, establishing scalable cGMP production for clinical trials.
  • The synthetic hypericin serves as the active ingredient in HyBryte™ and SGX302, topical photodynamic therapies being developed for cutaneous T-cell lymphoma and psoriasis treatment.
  • HyBryte™ demonstrated significant efficacy in Phase 3 trials with 16% of patients achieving 50% lesion reduction versus 4% with placebo, leading to FDA orphan drug and fast track designations.
  • A second confirmatory Phase 3 trial (FLASH2) is planned to enroll approximately 80 patients with early-stage CTCL, following EMA agreement on key design components.

IntraBio Completes Phase III Recruitment for Ataxia-Telangiectasia Treatment in Record Time

Rare DiseaseOrphan Drug
  • IntraBio's Phase III pivotal trial for N-Acetyl-L-Leucine (IB1001) in Ataxia-Telangiectasia completed recruitment in under two months, over-enrolling by 167%.
  • The randomized, placebo-controlled, double-blind crossover study enrolled patients across ten multinational sites in six countries.
  • Data readout is expected in Q1 2026, potentially bringing the first approved treatment for A-T patients closer to reality.
  • The rapid enrollment demonstrates significant unmet medical need and community enthusiasm for this investigational neurological therapy.

A study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia

2024-517706-29-00Active, Not RecruitingPhase 3
Intrabio Limited
Posted 3/18/2025

Apellis Secures $300 Million in Non-Dilutive Financing Through Sobi Royalty Agreement for Rare Kidney Disease Therapy

Rare Disease
  • Apellis Pharmaceuticals secured $275 million upfront and up to $25 million in milestone payments from Sobi through a capped royalty purchase agreement for Aspaveli's ex-U.S. commercialization rights.
  • The transaction involves Sobi acquiring 90% of Apellis' future ex-U.S. royalties for systemic pegcetacoplan, while Apellis retains full U.S. commercialization rights for the therapy.
  • Aspaveli is currently under regulatory review in both the EU and U.S. for treating C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis, rare kidney diseases affecting up to 13,000 patients globally.
  • The non-dilutive financing strengthens Apellis' balance sheet as the company approaches sustainable profitability and expands its rare disease portfolio.

Healx Partners with SCI Ventures in $2M AI-Driven Initiative to Develop Spinal Cord Injury Treatments

AIDrug DiscoveryRare Disease
  • Healx has partnered with SCI Ventures in a $2 million collaboration to apply AI-powered drug discovery to spinal cord injury, which affects over 20 million people globally with limited treatment options.
  • The partnership combines Healx's machine learning expertise with SCI Ventures' neuroregeneration domain knowledge and unique dataset access to target chronic spinal cord injury treatments.
  • The collaboration aims to dramatically shorten the path from discovery to clinical application by prioritizing AI-driven target discovery and drug repurposing strategies.
  • Success in spinal cord injury could open doors to AI-powered therapies for other neurological conditions including traumatic brain injury, stroke, ALS, multiple sclerosis, Parkinson's, and Alzheimer's disease.

Neurocrine Biosciences Initiates Phase 1 Trial for Long-Acting CAH Treatment NBIP-01435

Rare Disease
  • Neurocrine Biosciences has launched a Phase 1 first-in-human clinical study evaluating NBIP-01435, a long-acting corticotropin-releasing factor type 1 receptor antagonist for congenital adrenal hyperplasia treatment.
  • NBIP-01435 represents the first investigational peptide from Neurocrine's biologics pipeline to advance to clinical trials, administered as a subcutaneous injection.
  • The compound builds on Neurocrine's CAH expertise following their December 2024 FDA approval of crinecerfont, the first new CAH treatment in 70 years.
  • CRF1 antagonism has demonstrated potential to improve androgen control while enabling lower, more physiological glucocorticoid dosing regimens for CAH patients.

BridgeBio Secures $300 Million Royalty Financing to Support ATTR-CM Drug Launch

Drug ApprovalRare Disease
  • BridgeBio Pharma secured $300 million in upfront financing by selling 60% of European royalties for BEYONTTRA to HealthCare Royalty and Blue Owl Capital.
  • The transaction monetizes royalties on the first $500 million of annual BEYONTTRA net sales in Europe with a 1.45x cap on total investor payments.
  • Acoramidis demonstrated rapid clinical benefits in Phase 3 trials, showing 42% reduction in cardiovascular events and 50% reduction in cardiovascular hospitalizations.
  • The drug is approved as Attruby in the US and BEYONTTRA in Europe for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

ESTEVE Acquires Caprelsa Rights from Sanofi to Expand Rare Cancer Treatment Portfolio

Rare DiseaseOncology
  • ESTEVE has entered into an agreement with Sanofi to acquire Caprelsa® (vandetanib) rights in more than 50 countries for treating aggressive and symptomatic medullary thyroid cancer in adults and children above 5 years of age.
  • Caprelsa is a protein tyrosine kinase inhibitor that reduces blood supply to cancer cells and slows tumor growth, offering treatment options beyond surgery for this rare neuroendocrine tumor.
  • This acquisition represents another strategic step in ESTEVE's transformation into a highly specialized pharmaceutical company focused on rare diseases, following recent acquisitions in endocrinology and pediatric oncology.
  • The transaction is subject to customary regulatory clearances and strengthens ESTEVE's portfolio of treatments addressing significant unmet medical needs in specialized therapeutic areas.

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