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Tenaya Therapeutics Reveals Critical Risk Factors in Largest Pediatric MYBPC3-Associated HCM Study

Rare DiseaseOrphan Drug
  • Tenaya Therapeutics presented interim data from MyClimb, the largest natural history study of pediatric MYBPC3-associated hypertrophic cardiomyopathy with 213 participants under 18 years old.
  • The study revealed that 93% of participants had nonobstructive HCM with no approved treatments, and genetic inheritance patterns significantly predict disease severity and outcomes.
  • Left Ventricular Mass Index emerged as a significant predictor of risk, with every 10-unit increase associated with 10% higher hazard of serious events.
  • Homozygous patients showed the most severe outcomes with nearly all requiring heart transplant or dying before age one, highlighting urgent need for disease-modifying therapies.

Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy

NCT05112237Recruiting
Tenaya Therapeutics
Posted 11/1/2021

Neurotech's NTI164 Cannabinoid Therapy Shows Promise in Pediatric Neurological Disorders with Positive Phase II/III Data

Orphan Drug
  • Neurotech International's NTI164, a broad-spectrum oral cannabinoid therapy, met all primary and key secondary endpoints in its Phase II/III Harmony Study for Autism Spectrum Disorder, showing statistically significant improvements in anxiety, depression, and mood.
  • The company's first-in-human pharmacokinetic study confirmed NTI164 is rapidly absorbed with sustained plasma concentrations and excellent tolerability, supporting its suitability for long-term pediatric use.
  • NTI164 received orphan drug designation from both the FDA and European Commission for Rett Syndrome, with the Australian TGA determining eligibility for provisional designation to enable earlier patient access.
  • Neurotech appointed Dr. Anthony Filippis as CEO and is pursuing dual regulatory pathways in Australia and the US, with plans for an Investigational New Drug application to the FDA scheduled for 2026.

Sanofi's Wayrilz Becomes First BTK Inhibitor Approved for Immune Thrombocytopenia Treatment

Drug ApprovalOrphan DrugRare Disease
  • The FDA approved Wayrilz (rilzabrutinib) as the first BTK inhibitor for adults with persistent or chronic immune thrombocytopenia who have insufficient response to prior treatments.
  • The approval was based on the LUNA 3 phase 3 study showing 23% of Wayrilz patients achieved durable platelet response at week 25 compared to 0% in the placebo group.
  • Wayrilz targets the root causes of ITP through multi-immune modulation, offering a novel mechanism of action for the more than 25,000 US adults who could benefit from advanced treatment.
  • The drug demonstrated faster time to platelet response (36 days vs. not reached with placebo) and improved quality of life measures across nine health-related domains.

Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)

NCT04562766Active, Not RecruitingPhase 3
Principia Biopharma, a Sanofi Company
Posted 12/14/2020

Moleculin's Annamycin Shows Promising Survival Data in Relapsed AML Phase 1B/2 Trial

Orphan Drug
  • Moleculin Biotech reported completion of its Phase 1B/2 trial (MB-106) evaluating Annamycin combined with cytarabine for acute myeloid leukemia, showing median overall survival of 15 months for patients achieving complete remission.
  • The trial demonstrated significantly improved survival outcomes compared to industry expectations, with median overall survival of 9 months for the intent-to-treat population versus the typical 4-6 months for relapsed AML patients.
  • Among second-line patients, the complete remission rate reached 50%, with half of those achieving remission proceeding to curative bone marrow transplant, while no cardiotoxicity was observed across all enrolled subjects.
  • The company continues advancing its pivotal Phase 3 MIRACLE trial, with plans to unblind safety and efficacy data for the first 45 patients by year-end 2025.

Study of Liposomal Annamycin in Combination with Cytarabine for the Treatment of Subjects with Acute Myeloid Leukemia (AML)

NCT05319587Active, Not RecruitingPhase 1
Moleculin Biotech, Inc.
Posted 9/29/2022

L-Annamycin for Injection in Combination With Cytarabine Injection as Second Line Therapy for Remission Induction in Adult Subjects With Refractory/Relapsed AML

NCT06788756RecruitingPhase 2
Moleculin Biotech, Inc.
Posted 3/12/2025

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

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

NCT03750552CompletedPhase 3
Theravance Biopharma
Posted 1/24/2019

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

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.

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