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Can-Fite Receives IRB Approval for Phase IIa Pancreatic Cancer Trial of Namodenoson

Orphan DrugOncology
  • Can-Fite BioPharma received Institutional Review Board approval from Rabin Medical Center to initiate a Phase IIa open-label study evaluating Namodenoson in advanced pancreatic adenocarcinoma patients.
  • The multicenter trial will enroll approximately 20 patients with disease progression on first-line therapy, administering oral Namodenoson 25 mg twice daily in 28-day cycles.
  • Namodenoson demonstrated efficacy in preclinical pancreatic cancer models through deregulation of Wnt/β-catenin, NF-κB, and RAS signaling pathways.
  • The A3 adenosine receptor-targeting drug has shown promising results in liver cancer, with one patient achieving complete response lasting over 7 years.

Namodenoson Treatment of Advanced Pancreatic Cancer

NCT06387342RecruitingPhase 2
Can-Fite BioPharma
Posted 11/10/2024

Intellia's CRISPR Gene Therapy Shows Potential as Functional Cure for Hereditary Angioedema in Long-Term Study

Rare DiseaseGene EditingOrphan Drug
  • Intellia Therapeutics' NTLA-2002, an in vivo CRISPR-based gene editing therapy, demonstrated a 98% mean reduction in monthly hereditary angioedema attack rates across all patients, with follow-up extending beyond two years.
  • Eight of ten patients remained completely attack-free following the initial 16-week observation period, with the longest attack-free duration reaching over 26 months and continuing.
  • The single-dose treatment showed a favorable safety profile across all dose levels, with no serious adverse events reported, positioning NTLA-2002 as a potential functional cure for this rare genetic disease.

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830Active, Not RecruitingPhase 1
Intellia Therapeutics
Posted 12/10/2021

RemeGen's Telitacicept Receives Priority Review for Myasthenia Gravis Treatment Following Successful Phase III Trial

Orphan Drug
  • RemeGen's Telitacicept received priority review from China's NMPA for treating generalized myasthenia gravis after achieving its primary endpoint in a Phase III clinical trial.
  • The dual-target fusion protein demonstrated favorable efficacy and safety profiles with continuous reduction of clinical symptoms in gMG patients compared to placebo.
  • Myasthenia gravis affects approximately 1.146 million individuals worldwide, with around 217,000 in China, and currently has no definitive treatment available.
  • Telitacicept has already received breakthrough therapy designation from China's NMPA and orphan drug plus fast track designations from the US FDA.

Ocugen's OCU400 Becomes First Gene Therapy to Enter Phase 3 for Broad Retinitis Pigmentosa Treatment

Orphan Drug
  • Ocugen received FDA clearance to initiate a Phase 3 clinical trial for OCU400, marking the first gene therapy to enter Phase 3 with a broad retinitis pigmentosa indication.
  • The trial will enroll 150 participants across two arms, including 75 patients with RHO gene mutations and 75 gene-agnostic patients, addressing a significant unmet medical need.
  • OCU400 represents a novel modifier gene therapy approach that could potentially treat multiple forms of retinitis pigmentosa, compared to existing therapies limited to single gene mutations.
  • The company targets 2026 for BLA approval, with Phase 1/2 data suggesting more than 50% of RHO patients could meet responder criteria in the Phase 3 study.

Stoke Therapeutics and Biogen Initiate Phase 3 EMPEROR Trial for Zorevunersen in Dravet Syndrome

Orphan DrugRare Disease
  • Stoke Therapeutics and Biogen have dosed the first patient in the global Phase 3 EMPEROR study evaluating zorevunersen, an investigational antisense oligonucleotide for Dravet syndrome treatment.
  • The 52-week randomized, sham-controlled trial will enroll patients aged 2-18 with confirmed SCN1A gene variants, measuring seizure frequency reduction and cognitive improvements as primary endpoints.
  • Zorevunersen represents a potential first-in-class disease-modifying treatment targeting the underlying genetic cause of Dravet syndrome, which affects up to 38,000 people globally.
  • The drug has received FDA Breakthrough Therapy Designation and orphan drug status, with previous Phase 1/2 studies showing substantial seizure reductions and cognitive improvements.

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

NCT06872125RecruitingPhase 3
Stoke Therapeutics, Inc
Posted 6/4/2025

Chemomab Secures Global Patent Protection for CM-101 Antibody Targeting Primary Sclerosing Cholangitis

Monoclonal AntibodyOrphan Drug
  • Chemomab Therapeutics has secured new patents in Brazil, Israel, and Europe for CM-101, its first-in-class monoclonal antibody targeting CCL24 for fibro-inflammatory diseases.
  • The patents provide protection for CM-101's composition of matter and use in liver diseases including primary sclerosing cholangitis (PSC) until 2038, with potential five-year extensions.
  • CM-101 is currently in a Phase 2 SPRING trial for PSC treatment with completed patient enrollment and topline data expected midyear 2024.
  • PSC represents a significant unmet medical need as a potentially lethal condition with no FDA-approved therapies, often requiring liver transplantation.

Revive Therapeutics Advances Bucillamine Development for Long COVID Treatment Through Manufacturing Partnership

Orphan Drug
  • Revive Therapeutics has partnered with Attwill Medical Solutions to develop a novel lyophilized formulation of Bucillamine, with enhanced solubility showing more than double improvement over standard formulations.
  • The company is preparing a Phase 2/3 clinical protocol for long COVID treatment, leveraging data from a previous Phase 3 trial that showed no hospitalizations in the high-dose Bucillamine group.
  • Long COVID affects 7.5% of U.S. adults with an estimated economic burden of $3.7 trillion, representing a significant unmet medical need for effective treatments.
  • Bucillamine's thiol-based mechanism decreases SARS-CoV-2 spike protein binding and inhibits viral cell entry, providing scientific rationale for its potential in treating COVID-related conditions.

Novartis Secures $1.3 Billion Deal with Chong Kun Dang for HDAC6 Inhibitor Targeting CMT Disease

Orphan DrugRare Disease
  • Novartis has signed a $1.3 billion licensing agreement with Korean biotech Chong Kun Dang Pharmaceutical for CKD-510, an HDAC6 inhibitor with FDA orphan drug designation for Charcot-Marie-Tooth disease.
  • The deal includes an $80 million upfront payment plus $1.2 billion in potential milestone payments, with Novartis gaining exclusive global rights to develop and commercialize the drug outside Korea.
  • This acquisition follows Novartis' recent procurement of DTX-1252, another CMT1A therapeutic candidate, signaling the company's strategic expansion into rare neurological disease treatments.

Novartis' Ianalumab Achieves Primary Endpoint in Phase III ITP Trial, Offering Potential for Extended Treatment-Free Periods

Monoclonal AntibodyOrphan DrugRare Disease
  • Novartis announced positive Phase III results for ianalumab in immune thrombocytopenia, with the drug significantly prolonging time to treatment failure compared to placebo when combined with eltrombopag.
  • Patients receiving ianalumab experienced significantly higher rates of sustained platelet count improvements at six months, the key secondary endpoint of the VAYHIT2 study.
  • The monoclonal antibody demonstrated a consistent safety profile with no new safety signals, and could potentially offer long-term disease control through just four once-monthly doses.
  • Regulatory submissions are planned for 2027 alongside results from the ongoing first-line ITP trial, with ianalumab having received Orphan Drug Designation from both FDA and EMA.

Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)

NCT05653349Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 3/6/2023

A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis

NCT06470048RecruitingPhase 2
Novartis Pharmaceuticals
Posted 10/9/2024

Two-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome

NCT05350072Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 7/28/2022

A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia

NCT05648968RecruitingPhase 3
Novartis Pharmaceuticals
Posted 12/30/2022

A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids

NCT05653219Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 2/2/2023

Safety, Efficacy and Tolerability of Ianalumab Versus Placebo, Combination With SoC Therapy, in Participants With Active Lupus Nephritis

NCT05126277RecruitingPhase 3
Novartis Pharmaceuticals
Posted 7/14/2022

Phase 3 Study to Evaluate Two Regimens of Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 1)

NCT05639114RecruitingPhase 3
Novartis Pharmaceuticals
Posted 3/2/2023

Three-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome

NCT05349214Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 8/4/2022

IntraBio Completes Over-Enrollment for Pivotal Niemann-Pick Disease Type C Trial, Data Expected Q2 2023

Rare DiseaseOrphan Drug
  • IntraBio successfully completed recruitment for its Phase III pivotal trial of IB1001 (N-Acetyl-L-Leucine) for Niemann-Pick disease type C, enrolling 130% of target patients across 13 international sites.
  • The company over-enrolled by 125% due to high community interest and unmet medical need, substantially increasing the statistical power of the randomized, placebo-controlled, double-blind crossover study.
  • Data readout is anticipated before the end of Q2 2023, with the trial designed to support accelerated approval pathways with both FDA and EMA for chronic NPC symptom treatment.
  • Upon approval, IntraBio would be eligible for an FDA priority review voucher, which have sold for over $100 million each in the past two years.

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288RecruitingPhase 3
IntraBio Inc
Posted 6/30/2022

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