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Oryzon Receives EMA Approval for First LSD1 Inhibitor Trial in Sickle Cell Disease

Orphan Drug
  • Oryzon Genomics received European Medicines Agency approval to initiate the RESTORE Phase Ib trial of iadademstat in sickle cell disease, marking the first clinical investigation of an LSD1 inhibitor in this indication.
  • The study will enroll 40 adult patients across multiple Spanish sites to evaluate safety, tolerability, and the drug's ability to induce fetal hemoglobin production.
  • Preclinical data showed iadademstat significantly increased fetal hemoglobin levels in baboons after a single dose, targeting a clinically meaningful endpoint recognized by the FDA.
  • The sickle cell disease treatment market is projected to grow from approximately $3 billion in 2025 to around $8 billion by 2032, highlighting significant commercial potential.

Theravance Biopharma Completes Phase 3 Enrollment for Ampreloxetine in Rare Neurological Disorder

Orphan DrugRare Disease
  • Theravance Biopharma has completed enrollment in the pivotal Phase 3 CYPRESS study of ampreloxetine for neurogenic orthostatic hypotension in multiple system atrophy patients.
  • The investigational once-daily norepinephrine reuptake inhibitor could become the first therapy to provide durable benefit for 40,000 U.S. patients with this devastating condition.
  • Topline results are expected in Q1 2026, with plans for expedited NDA submission and priority FDA review if data prove supportive.
  • Previous Study 0170 showed a 72% reduction in treatment failure odds for MSA patients, demonstrating compelling symptom improvement without worsening supine hypertension.

Phase 3 Efficacy and Durability of Ampreloxetine for the Treatment of Symptomatic NOH in Participants with Multiple System Atrophy

NCT05696717RecruitingPhase 3
Theravance Biopharma
Posted 6/27/2023

Phase 3 Clinical Effect Durability of TD-9855 for Treating Symptomatic nOH in Subjects With Primary Autonomic Failure

NCT03829657TerminatedPhase 3
Theravance Biopharma
Posted 2/22/2019

Clinical Effect of Ampreloxetine (TD-9855) for Treating Symptomatic nOH in Subjects With Primary Autonomic Failure

NCT03750552CompletedPhase 3
Theravance Biopharma
Posted 1/24/2019

Akiram's Targeted Radiotherapy AKIR001 Advances to Next Phase After Completing First Patient Cohort Without Safety Concerns

Targeted TherapyOncologyOrphan Drug
  • Swedish biotech Akiram Therapeutics completed the first patient cohort of its Phase I trial for AKIR001, a targeted radiopharmaceutical combining an anti-CD44v6 antibody with lutetium-177 for aggressive solid tumors.
  • No dose-limiting toxicities or safety concerns were observed in the initial cohort, allowing the trial to proceed to the next stage as planned at Karolinska University Hospital.
  • The study targets patients with difficult-to-treat cancers including anaplastic thyroid, head and neck, gynecological, and non-small cell lung cancers.
  • AKIR001 delivers radiation directly to tumor cells while minimizing damage to surrounding healthy tissue through its selective targeting of the CD44v6 cancer marker.

[177Lu]Lu-AKIR001 First-in-human Study

NCT06639191Not Yet RecruitingEarly Phase 1
Karolinska University Hospital
Posted 11/1/2024

Leads Biolabs Advances First-in-China GPRC5D T-Cell Engager LBL-034 to Phase II Trial

Orphan Drug
  • Nanjing Leads Biolabs has successfully dosed the first patient in the Phase II trial of LBL-034, a GPRC5D/CD3 bispecific T-cell engager for relapsed/refractory multiple myeloma.
  • The therapy demonstrated CAR-T-like efficacy with robust objective response rates at higher doses and showed promising results in difficult-to-treat extramedullary plasmacytomas during Phase I trials.
  • LBL-034 is positioned to become the first domestic T-cell engager therapy targeting GPRC5D in China and is the second most clinically advanced globally.
  • The multi-center Phase II trial involves over 20 hospitals across China and aims to evaluate efficacy and safety in various relapsed/refractory plasma cell neoplasms.

Neuren Pharmaceuticals Launches First-Ever Phase 3 Trial for Phelan-McDermid Syndrome

Orphan DrugRare Disease
  • Neuren Pharmaceuticals has initiated the first investigational site in the United States for its Phase 3 clinical trial of NNZ-2591 for Phelan-McDermid syndrome, marking the first-ever Phase 3 trial for this severe neurodevelopmental disorder.
  • The randomized, double-blind, placebo-controlled trial will assess 13 weeks of treatment in approximately 160 children aged 3 to 12 with PMS, with all participants eligible for a 12-month open-label extension.
  • Phelan-McDermid syndrome affects an estimated 1 in 8,000 to 1 in 15,000 people and currently has no approved treatments available.
  • The trial program is fully funded by Neuren's existing cash reserves, following FDA alignment on the Phase 3 trial design including primary efficacy endpoints.

VASTHERA Receives FDA Clearance for Phase 1 Trial of Novel PAH Drug VTB-10

Orphan Drug
  • VASTHERA received FDA IND clearance on August 6, 2025, to initiate Phase 1 clinical trials for VTB-10, a first-in-class drug candidate targeting pulmonary arterial hypertension.
  • VTB-10 is developed using VASTHERA's proprietary Redoxizyme™ platform and functions as a small molecule enzyme that replicates peroxiredoxin function to address enzyme deficiency in PAH lesions.
  • Preclinical studies demonstrated VTB-10's ability to reverse abnormal vascular remodeling and restore functional endothelium, differentiating it from existing PAH therapies.
  • The drug candidate previously received FDA Orphan Drug Designation in November 2024 and is supported by funding from the Korea Drug Development Fund.

Quoin Pharmaceuticals Appoints Sally Lawlor as CFO to Navigate QRX003 Commercialization

Orphan DrugRare Disease
  • Quoin Pharmaceuticals has appointed Sally Lawlor as Chief Financial Officer, bringing over 20 years of pharmaceutical finance experience to guide the company through commercialization preparations.
  • The appointment comes as Quoin advances QRX003 through two pivotal clinical studies for Netherton Syndrome, with full enrollment expected in early to mid-Q1 2026.
  • QRX003 has received regulatory advantages including Orphan Drug Designation from the EMA and Rare Pediatric Disease Designation from the FDA.
  • The company reported $7.8 million in cash as of June 30, 2025, providing operational runway into Q1 2026 as it prepares for potential commercialization.

Ascelia Pharma Nears Orviglance NDA Submission for Orphan Liver Imaging Agent

Orphan Drug
  • Ascelia Pharma is finalizing its New Drug Application (NDA) for Orviglance, a first-in-class oral MRI contrast agent for liver imaging in kidney-impaired patients.
  • The pivotal Phase 3 SPARKLE trial demonstrated statistically significant improvements in lesion visualization compared to unenhanced MRI across three independent readers (p < 0.001).
  • Orviglance addresses a $300-450 million annual U.S. market opportunity with no direct competitors, targeting 100,000 liver MRI procedures in renal-impaired patients.
  • The drug has received Orphan Drug Designation from the FDA, providing seven years of market exclusivity and expedited review pathways upon approval.

Vedanta Biosciences' VE202 Fails Phase 2 Trial for Ulcerative Colitis, Company Refocuses on C. difficile Program

Orphan Drug
  • Vedanta Biosciences announced that VE202 did not meet the primary endpoint of endoscopic response in the Phase 2 COLLECTiVE202 study for mild-to-moderate ulcerative colitis treatment.
  • The microbiome-based therapy was well tolerated with no treatment-related serious adverse events, but showed no statistical difference from placebo in response rates.
  • The company is redirecting resources to its lead program VE303, which demonstrated over 30% reduction in C. difficile recurrence risk and is currently in Phase 3 trials.
  • VE303 has received FDA Fast Track and Orphan Drug designations and could become the first approved live biotherapeutic product for any indication.

VE202 in Patients With Mild-to-Moderate Ulcerative Colitis

NCT05370885Active, Not RecruitingPhase 2
Vedanta Biosciences, Inc.
Posted 5/8/2023

MMJ BioPharma Files Emergency Motion to DEA as Trump Signals Openness to Medical Cannabis

Orphan Drug
  • MMJ BioPharma Cultivation filed an emergency motion to DEA Administrator Terry Cole seeking immediate approval of its seven-year-delayed application to manufacture pharmaceutical-grade cannabis for FDA clinical trials.
  • The company holds FDA Orphan Drug Designations and has filed Investigational New Drug applications for cannabis-based treatments targeting Huntington's disease and Multiple Sclerosis.
  • President Trump acknowledged the medical benefits of marijuana and announced a reclassification decision could come within the next few weeks.
  • MMJ's GMP-compliant approach aligns with federal law and operates separately from the recreational cannabis industry, addressing the balance between public safety and legitimate medical need.

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