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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.

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.

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

F2G's Olorofim Shows 44% Response Rate in Phase 2b Trial for Invasive Fungal Infections

Orphan Drug
  • F2G announced positive Phase 2b data for olorofim showing a 44% complete or partial response rate at day 42 in 100 patients with invasive fungal infections who had limited treatment options.
  • The novel antifungal demonstrated low mortality rates of 15% at day 42 and 20% at day 84, with generally good tolerability except for drug-induced liver injury in 8% of patients.
  • Olorofim is the only antifungal with FDA Breakthrough Therapy Designation and works through a unique mechanism targeting dihydroorotate dehydrogenase in the pyrimidine synthesis pathway.
  • The drug showed activity against multiple resistant fungi including Aspergillus species, Lomentospora prolificans, and Coccidioides species, addressing significant unmet medical needs in immunocompromised patients.

Evaluate F901318 (Olorofim) Treatment of Invasive Fungal Infections in Participants Lacking Treatment Options

NCT03583164CompletedPhase 2
F2G Biotech GmbH
Posted 6/6/2018

Olorofim Aspergillus Infection Study

NCT05101187RecruitingPhase 3
F2G Biotech GmbH
Posted 3/31/2022

PharmaTher Secures U.S. Patent for Advanced Ketamine Production Technology

Orphan Drug
  • PharmaTher Holdings received U.S. Patent No. 11,286,230 for "Ketamine Flow Synthesis," a continuous-flow process technology that improves ketamine and analog production efficiency.
  • The patented technology addresses traditional batch production limitations by offering better yield, reproducibility, and purity while requiring a smaller manufacturing footprint.
  • The company plans to leverage this technology to support upcoming pivotal clinical studies for Parkinson's disease and ALS, with an FDA filing expected in Q4-2022.
  • PharmaTher's expanding intellectual property portfolio includes novel ketamine applications for neurological disorders and innovative delivery systems like microneedle patches.

First Patient Dosed in Phase 2b Trial of RT234, a Novel Inhaled Therapy for Pulmonary Arterial Hypertension

Orphan Drug
  • Respira Therapeutics has dosed the first patient in its Phase 2b VIPAH-PRN trial evaluating RT234, an innovative dry powder inhaled formulation of vardenafil for on-demand symptom relief in PAH patients.
  • RT234 represents a first-in-class approach for PAH, designed for "as needed" use to acutely improve exercise capacity and reduce breathlessness, unlike current maintenance therapies that don't address episodic symptoms.
  • The multicenter dose-escalation study will assess RT234's ability to provide rapid symptom relief for patients with NYHA Functional Class II-III PAH symptoms, potentially addressing a significant unmet need in disease management.

FDA Approves First Treatment for Primary Hemophagocytic Lymphohistiocytosis, Marking 24-Year Breakthrough

Monoclonal AntibodyDrug ApprovalRare DiseaseOrphan Drug
  • The FDA has approved emapalumab-lzsg (Gamifant) as the first treatment specifically indicated for primary hemophagocytic lymphohistiocytosis (HLH), an ultra-rare and life-threatening immune disorder.
  • In a pivotal phase 2/3 study of 27 patients with refractory disease, 63% demonstrated overall response and 70% proceeded to hematopoietic stem cell transplant.
  • The monoclonal antibody targets interferon gamma, representing the first significant improvement in primary HLH induction therapy in 24 years.
  • Primary HLH typically affects children within the first year of life and has a median survival of less than two months without treatment.

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

NCT01818492CompletedPhase 2
Swedish Orphan Biovitrum
Posted 7/1/2013

Life Molecular Imaging Secures FDA Fast Track Designation for [18F]florbetaben in Cardiac Amyloidosis Diagnosis

Orphan Drug
  • Life Molecular Imaging received FDA Fast Track Designation for [18F]florbetaben to diagnose cardiac amyloid light-chain (AL) and amyloid transthyretin-related (ATTR) amyloidosis.
  • The PET imaging agent is already approved as Neuraceq® for detecting brain amyloid plaques and has demonstrated capability to identify amyloid deposits in the heart.
  • A Phase 3 trial (NCT05184088) is currently underway to validate [18F]florbetaben's efficacy in cardiac amyloidosis diagnosis.
  • The designation aims to streamline the complex diagnostic process for cardiac AL amyloidosis, potentially providing earlier access to therapy and improved patient monitoring.

Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

NCT05184088RecruitingPhase 3
Life Molecular Imaging GmbH
Posted 1/13/2023

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