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Aardvark Therapeutics Expands Phase 3 HERO Trial for Prader-Willi Syndrome Treatment to Include Younger Patients

Rare DiseaseOrphan Drug
  • Aardvark Therapeutics received FDA alignment on a protocol amendment to expand its Phase 3 HERO trial of ARD-101 for Prader-Willi Syndrome, lowering the minimum age eligibility from 13 to 10 years old.
  • The expansion allows the company to reach a larger segment of the PWS patient population, with historical data suggesting younger patients are more likely to benefit from early intervention.
  • ARD-101 is a gut-restricted small molecule that activates bitter taste receptors to stimulate release of satiety hormones including GLP-1 and CCK, targeting the insatiable hunger characteristic of PWS.
  • The company expects topline data readout from this potentially pivotal trial in the third quarter of 2026.

FDA Authorizes Expanded Access Program for First-in-Class Tourette Syndrome Drug Ecopipam

Orphan Drug
  • The FDA has authorized Emalex Biosciences' Expanded Access Program for ecopipam, an investigational first-in-class treatment for Tourette syndrome that selectively blocks dopamine-1 receptors.
  • The program allows physicians to request access for eligible patients who have failed current FDA-approved treatments or experienced tolerability issues with existing therapies.
  • Emalex plans to submit a New Drug Application to the FDA in late 2025 for this novel therapeutic approach that differs from current treatments targeting dopamine-2 receptors.
  • Clinical studies in pediatric patients showed ecopipam was generally well tolerated, with the most common adverse events being headache, insomnia, fatigue, and anxiety.

Expanded Access Program for Treatment With Ecopipam for Tourette's Disorder

NCT07093541Unknown Status
Emalex Biosciences Inc.

FDA Issues Complete Response Letter for Xspray Pharma's Dasynoc, Citing Manufacturing Issues

Drug ApprovalOrphan Drug
  • The FDA issued a complete response letter to Xspray Pharma's new drug application for Dasynoc, a lower-dose bioequivalent formulation of dasatinib for chronic myeloid leukemia and acute lymphoblastic leukemia treatment.
  • The regulatory setback stems from Good Manufacturing Practice issues at Xspray's contract manufacturer, though no observations were directed at the specific production line used for Dasynoc.
  • Dasynoc has demonstrated bioequivalence to dasatinib at a 30% lower dose and maintains compatibility with proton pump inhibitors, addressing a significant drug interaction concern in CML treatment.
  • The company plans to work with the manufacturer and FDA to implement corrective actions, with a meeting scheduled for December to expedite the resubmission process.

Cellectar Biosciences Raises $5.8 Million Through Warrant Exercise to Advance Cancer Drug Pipeline

Orphan DrugTargeted TherapyOncology
  • Cellectar Biosciences secured approximately $5.8 million in gross proceeds through institutional investor warrant exercises, with Ladenburg Thalmann serving as exclusive placement agent.
  • The funding will support the company's Phase 1b clinical study of CLR 121125 in triple-negative breast cancer and preparation for European regulatory filing.
  • The company's proprietary Phospholipid Drug Conjugate platform targets next-generation cancer treatments with improved efficacy and safety profiles.
  • Cellectar's lead asset iopofosine I 131 has received six Orphan Drug, four Rare Pediatric Drug, and two Fast Track designations from the FDA.

RIBOMIC's Umedaptanib Pegol Shows Promising Growth Rate Improvements in Phase 2 Achondroplasia Trial

Orphan DrugRare Disease
  • RIBOMIC's Phase 2 trial of umedaptanib pegol demonstrated significant height growth improvements in children with achondroplasia, with some patients achieving increases of up to 5.0 cm/year.
  • The biweekly high-dose regimen (0.6 mg/kg) showed comparable efficacy to the approved daily treatment Voxzogo, with four subjects exceeding Voxzogo's average growth rate of 1.7 cm/year.
  • The company plans to initiate Phase 3 trials in Q1 2026 with higher doses and younger patients, targeting regulatory approval by fiscal year 2028 under orphan drug designation.

Dyne Therapeutics Reports Sustained One-Year Functional Improvements with Zeleciment Basivarsen in Myotonic Dystrophy Type 1

Orphan Drug
  • Dyne Therapeutics announced one-year data from the Phase 1/2 ACHIEVE trial showing sustained functional improvements with zeleciment basivarsen at 6.8 mg/kg in six adults with myotonic dystrophy type 1.
  • The investigational therapy demonstrated meaningful improvements across multiple measures including muscle strength, myotonia, upper and lower limb function, and patient-reported outcomes.
  • New data revealed improvements in manual dexterity via the 9-Hole Peg Test and comprehensive muscle strength gains across all tested muscle groups in both upper and lower extremities.
  • Both patients and physicians reported meaningful improvements in overall disease burden, with the therapy showing a favorable safety profile across 56 enrolled patients.

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

NCT05481879RecruitingPhase 1
Dyne Therapeutics
Posted 9/5/2022

Neuraptive Therapeutics Initiates Phase 3 Trial for NTX-001 in Traumatic Peripheral Nerve Injuries

Orphan Drug
  • Neuraptive Therapeutics has enrolled the first patient in a Phase 3 clinical trial evaluating NTX-001 as an adjunct to standard care for upper extremity nerve injuries requiring surgical repair.
  • The multicenter, randomized, controlled study will enroll approximately 110 patients across 24 US clinical sites to compare NTX-001 plus standard care versus standard care alone.
  • NTX-001 has received FDA Fast Track Designation and Orphan Drug Designation, with the trial supported by Department of Defense funding through the Armed Forces Institute of Regenerative Medicine.
  • The therapy aims to address significant unmet medical needs in peripheral nerve injury treatment and accelerate functional recovery for trauma patients.

FDA Grants Breakthrough Therapy Designation to Nacuity's NPI-001 for Retinitis Pigmentosa Treatment

Orphan Drug
  • The U.S. FDA has granted Breakthrough Therapy Designation to NPI-001 (N-acetylcysteine amide) tablets, developed by Nacuity Pharmaceuticals for treating retinitis pigmentosa, a serious inherited blinding disease.
  • NPI-001 targets oxidative stress by boosting glutathione levels to prevent retinal cell damage, representing a novel therapeutic approach for a condition affecting approximately 100,000 Americans.
  • The designation recognizes early clinical evidence suggesting NPI-001 may deliver substantial treatment effects for patients with retinitis pigmentosa, for which no FDA-approved standard treatments currently exist.
  • This regulatory milestone adds to NPI-001's existing Fast Track and Orphan Drug Designations, providing enhanced FDA guidance and seven years of regulatory exclusivity upon approval.

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome

NCT04355689Active, Not RecruitingPhase 1
Nacuity Pharmaceuticals, Inc.
Posted 9/3/2020

Lundbeck Advances Innovative Phase 3 Trial Design for Amlenetug in Multiple System Atrophy

Monoclonal AntibodyOrphan DrugRare Disease
  • Lundbeck will present details of its Phase 3 MASCOT trial for amlenetug, a first-in-class monoclonal antibody targeting α-synuclein aggregation in Multiple System Atrophy patients.
  • The innovative trial design employs Bayesian progression modeling methods to address unique challenges in rare disease drug development with limited patient populations.
  • Multiple System Atrophy is a rapidly progressing neurodegenerative disease with no approved treatments, affecting patients who typically live 6-9 years after symptom onset.
  • Amlenetug has received Orphan Drug Designation from FDA and EMA, plus SAKIGAKE designation in Japan, highlighting its potential therapeutic significance.

A Trial of Lu AF82422 in Participants With Multiple System Atrophy (MSA)

NCT06706622RecruitingPhase 3
H. Lundbeck A/S
Posted 12/3/2024

A Study of Lu AF82422 in Participants With Multiple System Atrophy

NCT05104476Active, Not RecruitingPhase 2
H. Lundbeck A/S
Posted 11/16/2021

AliveGen's ALG-801 Demonstrates Superior Efficacy Over Standard PAH Therapies in Preclinical Studies

Orphan Drug
  • AliveGen's investigational therapy ALG-801 outperformed current standard-of-care agents including Sildenafil and Sotatercept in preclinical studies using a gold standard pulmonary arterial hypertension animal model.
  • The therapy demonstrated superior prevention of pulmonary hypertension during disease development and significantly reversed established PAH symptoms including elevated pulmonary arterial pressure and right ventricular hypertrophy.
  • ALG-801 has completed Phase 1 trials and received FDA Orphan Drug Designation, positioning it as a promising candidate for Phase 2 clinical development in a disease where median transplant-free survival remains only six years.

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