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Ionis Reports Positive Phase 3 Results for Olezarsen in Familial Chylomicronemia Syndrome

Rare DiseaseOrphan Drug
  • Ionis Pharmaceuticals' olezarsen met primary and key secondary endpoints in Phase 3 BALANCE trial for familial chylomicronemia syndrome (FCS), demonstrating significant triglyceride reduction.
  • The antisense therapy showed a favorable safety profile with no major adverse events reported, potentially offering a new treatment option for this rare genetic disorder.
  • Ionis plans to submit regulatory applications in the coming months, positioning olezarsen to potentially become the first approved therapy specifically targeting FCS.

iOnctura Secures €80 Million Series B to Advance PI3Kδ Cancer Therapy Through Phase II Trials

Orphan Drug
  • iOnctura raised €80 million in Series B financing led by Syncona to advance its pipeline of oral cancer treatments targeting neglected and hard-to-treat cancers.
  • Lead asset roginolisib, the first allosteric modulator of PI3Kδ, demonstrated promising efficacy in uveal melanoma with 70% overall survival rate and minimal toxicity in Phase I trials.
  • The funding will accelerate Phase II trials for roginolisib in uveal melanoma and expand development into non-small cell lung cancer and primary myelofibrosis starting late 2024.
  • Second asset cambritaxestat represents the only autotaxin inhibitor in clinical development for highly fibrotic tumors like metastatic pancreatic cancer.

A Study to Assess a PI3Kδ Inhibitor (IOA-244) in Patients with Metastatic Cancers

NCT04328844Active, Not RecruitingPhase 1
iOnctura
Posted 2/25/2020

Dose Escalation Phase 1 Study of IOA-289 in combination with gemcitabine/nab-paclitaxel

2024-515674-27-00Not Yet RecruitingPhase 1
iOnctura SA

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.

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