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Zydus Therapeutics Reports Positive Phase 3 Results for Saroglitazar in Primary Biliary Cholangitis

Rare Disease
  • Zydus Therapeutics announced positive results from the EPICS-III Phase 2b/3 trial of Saroglitazar in Primary Biliary Cholangitis patients who had inadequate response to standard therapy.
  • The trial met its primary endpoint with a statistically significant 48.5% treatment difference in biochemical response compared to placebo (P<0.001).
  • Saroglitazar represents the first PPAR alpha/gamma agonist to demonstrate positive Phase 3 data in PBC patients, with US regulatory filing planned for Q1 2026.
  • The drug was generally well tolerated with adverse events balanced between treatment and placebo groups in this rare autoimmune liver disease.

Saroglitazar Magnesium for Treatment of Primary Biliary Cholangitis

NCT05133336CompletedPhase 2
Zydus Therapeutics Inc.
Posted 4/1/2022

Entera Bio to Present Phase 2 Osteoporosis Data and Next-Generation Oral Peptide Programs at September 2025 Conferences

Rare Disease
  • Entera Bio will present Phase 2 data for EB613, the first once-daily oral anabolic tablet for postmenopausal osteoporosis, showing rapid BMD increases at both cortical and cancellous bone sites.
  • The company plans a global Phase 3 registration study for EB613 following FDA alignment in July 2025, targeting the significant unmet need in osteoporosis treatment.
  • Data on next-generation EB613 utilizing proprietary N-Tab technology and a first-in-class oral GLP-2 analog for short bowel syndrome will also be presented at ASBMR and ESPEN conferences.

Personalized ALS Drug Shows Breakthrough Results, Slowing Disease Progression by More Than Half

Rare DiseasePrecision Medicine
  • A custom-built antisense oligonucleotide drug targeting the CHCHD10 gene mutation has slowed ALS progression by more than half in Dr. Rakesh Parekh, marking the first treatment for this specific genetic variant.
  • The personalized therapy, developed by the n-Lorem Foundation, cost $1.2 million and took three years to create but can now be adapted for other patients with the same mutation.
  • This breakthrough represents a new era of "N-of-1" medicine for ALS, with gene-targeting therapies showing potential to transform treatment for the 15-20% of patients with known genetic mutations.

Hong Kong Approves Four New Drugs Under Accelerated "1+" Mechanism, Including IgA Nephropathy Treatment

Drug ApprovalRare DiseaseOncology
  • Hong Kong has approved four new drugs under its accelerated "1+" approval mechanism, including a treatment for primary immunoglobulin A nephropathy and therapies for thyroid eye disease, cervical cancer, and severe hemophilia.
  • Since implementation in November 2023, the "1+" mechanism has approved 15 new drugs total, with seven already listed in the Hospital Authority Drug Formulary.
  • The mechanism allows new drugs to gain Hong Kong approval with support from just one reference regulatory authority instead of two, significantly streamlining the approval process.
  • Hong Kong plans to establish the Centre for Medical Products Regulation by end of 2025 and implement "primary evaluation" for drug registration beginning next year.

Vanda Pharmaceuticals Receives FDA Orphan Drug Designation for Novel JAK2-Selective Antisense Therapy VGT-1849B in Polycythemia Vera

Rare Disease
  • The FDA has granted Orphan Drug Designation to VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor developed by Vanda Pharmaceuticals for treating polycythemia vera.
  • VGT-1849B utilizes novel OliPass Peptide Nucleic Acid (OPNA) chemistry to selectively target JAK2 mRNA, potentially offering improved safety compared to existing JAK inhibitors that affect multiple kinases.
  • The antisense oligonucleotide specifically addresses the JAK2 V617F mutation found in over 95% of polycythemia vera patients, with the disease affecting 44-57 per 100,000 people in the United States.
  • Unlike current JAK inhibitors such as Jakafi, Inrebic, Ojjaara, and Vonjo, VGT-1849B aims to avoid off-target kinase effects that can lead to increased toxicity and adverse side effects.

Rafael Holdings Reports 78% Success Rate for NPC1 Treatment in Phase 3 Pediatric Sub-Study

Rare DiseaseOrphan Drug
  • Rafael Holdings announced preliminary data from its Phase 3 TransportNPC sub-study showing that 7 of 9 patients under 3 years old with NPC1 demonstrated stabilization or improvement after 48 weeks of treatment with Trappsol Cyclo.
  • The investigational drug hydroxypropyl-beta-cyclodextrin showed a favorable safety profile with the majority of adverse events reported as mild to moderate, and no serious adverse events were considered related to the study drug.
  • The data was presented at the 15th International Congress of Inborn Errors of Metabolism, representing the first clinical evidence for treating NPC1 in this youngest patient population over a 48-week period.

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

NCT04860960Active, Not RecruitingPhase 3
Cyclo Therapeutics, Inc.
Posted 7/20/2021

Expanded Access With Trappsol(R) Cyclo (TM) for an Individual Patient With Late Onset Alzheimer's Disease

NCT03624842No Longer Available
Cyclo Therapeutics, Inc.

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

NCT03893071Unknown StatusPhase 1
Cyclo Therapeutics, Inc.
Posted 5/23/2019

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

NCT02939547CompletedPhase 1
Cyclo Therapeutics, Inc.
Posted 10/11/2017

A 6-Month Study to Evaluate the Safety & Potential Efficacy of Trappsol Cyclo in Patients With Early Alzheimer's Disease

NCT05607615RecruitingPhase 2
Cyclo Therapeutics, Inc.
Posted 9/23/2022

Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients

NCT02912793CompletedPhase 1
Cyclo Therapeutics, Inc.
Posted 3/20/2017

Cleveland Clinic Deploys AI Platform to Accelerate Clinical Trial Recruitment Across Multiple Specialties

AIRare Disease
  • Cleveland Clinic has launched Dyania Health's Synapsis AI platform across its clinical research enterprise following successful pilot programs in cardiology, oncology, and neurology.
  • The AI platform identified melanoma trial patients in 2.5 minutes with 96% accuracy, dramatically outperforming specialized nurses who required over 400 minutes for similar accuracy.
  • For a rare heart disease trial, the system screened 1.2 million records and identified 30 eligible participants in one week, compared to 14 patients found through routine methods over 90 days.
  • The collaboration addresses critical recruitment challenges where 80% of clinical trials miss enrollment timelines and 50% of trial sites fail to recruit any participants.

Amylyx Discontinues AMX0035 Development for Progressive Supranuclear Palsy After Phase 2b Trial Fails to Meet Primary Endpoints

Rare Disease
  • Amylyx Pharmaceuticals has discontinued the ORION program of AMX0035 for progressive supranuclear palsy after the drug failed to show differences compared to placebo on primary or secondary outcomes at Week 24.
  • The Phase 2b trial, which targeted 110 patients, assessed disease progression as the primary endpoint, with AMX0035 also failing to outperform placebo in motor aspects of daily living activities.
  • Despite the setback, Amylyx will continue developing AMX0035 for Wolfram syndrome and maintains focus on its Phase 3 LUCIDITY trial of avexitide for post-bariatric hypoglycemia, with enrollment completion expected in 2025.
  • The company expects its cash runway to extend through the end of 2026 and will also progress AMX0114 development in ALS with early cohort data expected in 2025.

Avexitide for Treatment of Post-Bariatric Hypoglycemia

NCT06747468RecruitingPhase 3
Amylyx Pharmaceuticals Inc.
Posted 4/29/2025

AMX0035 and Progressive Supranuclear Palsy

NCT06122662Active, Not RecruitingPhase 2
Amylyx Pharmaceuticals Inc.
Posted 12/21/2023

Comprehensive Clinical Trials Review Reveals Global Lennox-Gastaut Syndrome Research Landscape

Rare Disease
  • A new comprehensive report analyzes the global clinical trials landscape for Lennox-Gastaut Syndrome, providing critical data on trial numbers, enrollment figures, and regional distribution across G7 and E7 nations.
  • The analysis covers diverse aspects of clinical trials categorized by region, trial phase, status, sponsorship type, and endpoint analysis, highlighting prominent drugs currently under investigation.
  • Major pharmaceutical companies including Jazz Pharmaceuticals, Takeda, UCB, GSK, and Eisai are actively conducting trials in this rare epilepsy syndrome space.
  • The report aims to facilitate strategic business planning and investment decisions while identifying optimal locations for conducting efficient clinical trials in this challenging therapeutic area.

EMA Grants First-Ever Orphan Drug Designation for Radiation Maculopathy Treatment

Rare DiseaseOncology
  • The European Medicines Agency has granted orphan drug designation to Roca Therapeutics' RCT002, marking the first formal recognition of radiation maculopathy as a distinct medical indication.
  • RCT002 is a first-in-class eye drop therapy designed to address resistant neovascularization, inflammation, fibrosis, and oxidative stress in patients experiencing vision loss after radiotherapy.
  • The designation opens pathways for accelerated regulatory support and targeted therapeutic development for a condition that currently has no approved treatment options.
  • Roca Therapeutics plans to initiate first-in-human clinical trials for RCT002 in 2026 while currently fundraising to support the program.

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