HUTCHMED announces positive results from SAVANNAH Phase II trial for lung cancer treatment
- TAGRISSO plus ORPATHYS showed high response rates in EGFR-mutated NSCLC with MET overexpression/amplification.
- The trial focused on patients who developed resistance to TAGRISSO, a common issue in EGFR-targeted therapies.
- The combination therapy received Fast Track designation from the US FDA in 2023.
- The global SAFFRON Phase III trial will further assess this combination against standard chemotherapy.
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Akeso reports positive results from phase 3 study of cadonilimab in cervical cancer
- Akeso Biopharma announced positive results from the Phase 3 COMPASSION-16 study of cadonilimab in cervical cancer.
- The study evaluated cadonilimab with or without chemotherapy and bevacizumab against a placebo regimen.
- Cadonilimab significantly improved progression-free survival (PFS) and overall survival (OS) in the study.
- The study results were presented at IGCS 2024 and published in The Lancet.
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Bioversys completes phase 2 trial for BV100 in VABP
- BioVersys completed the last patient visit in the Phase 2 trial of BV100 for ventilator-associated bacterial pneumonia (VABP).
- Preliminary results indicate BV100 is safe, well-tolerated, and shows strong efficacy against CRAB infections.
- The trial met its primary endpoint, with lower mortality rates compared to the control group.
- A global Phase 3 trial is planned for the second half of 2025, with a Phase 1 study in China starting in 1H 2025.
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OKYO Pharma starts phase 2 trial for neuropathic corneal pain
- OKYO Pharma has initiated a Phase 2 trial for OK-101 to treat neuropathic corneal pain (NCP), a rare disorder with no FDA-approved treatments.
- The trial is a 12-week, randomized, placebo-controlled study involving 48 patients, focusing on pain relief measured by the Visual Analog Scale.
- OK-101 previously showed significant pain relief in a Phase 2 trial for dry eye disease, indicating potential for NCP treatment.
- The trial is led by Dr. Pedram Hamrah at Tufts Medical Center, with patient recruitment underway.
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Wave Life Sciences achieves first-ever therapeutic RNA editing in humans
- Wave Life Sciences announced positive proof-of-mechanism data from the RestorAATion-2 trial of WVE-006 for alpha-1 antitrypsin deficiency (AATD).
- A single subcutaneous dose of WVE-006 resulted in mean plasma total AAT levels of ~11 micromolar, with wild-type M-AAT representing over 60% of total AAT.
- The trial demonstrated durable editing with M-AAT protein observed through 57 days, and the treatment was well-tolerated with no serious adverse events.
- Wave plans to share multidose data from the RestorAATion-2 trial in 2025, with development and commercialization responsibilities transferring to GSK post-trial.
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Valneva and LimmaTech receive FDA fast track for shigella vaccine
- Valneva and LimmaTech's tetravalent Shigella vaccine candidate, S4V, has been granted FDA Fast Track designation.
- The Fast Track status aims to expedite the development and review of the vaccine, addressing a significant unmet medical need.
- Following positive Phase 1/2 results, upcoming studies include a Phase 2 Controlled Human Infection Model in the U.S. and a pediatric study in LMICs.
- Valneva will handle further development and commercialization, with plans to leverage CHIM studies for initial adult approval.
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Innovent announces phase 2 clinical study of picankibart in Chinese patients with ulcerative colitis met primary endpoint
- Innovent's phase 2 study of picankibart in ulcerative colitis met its primary endpoint during the 12-week induction period.
- The study involved 150 patients randomized to receive placebo, picankibart 200 mg, or picankibart 600 mg.
- Clinical remission was significantly higher in the picankibart groups compared to placebo, with favorable safety profiles.
- The maintenance period is ongoing, with further data to be published in future academic forums.
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European commission grants orphan medicinal product designation for navenibart
- Astria Therapeutics' navenibart receives Orphan Medicinal Product Designation from the European Commission for hereditary angioedema (HAE).
- Navenibart is a monoclonal antibody inhibitor of plasma kallikrein, showing a 90-96% reduction in monthly attack rates in Phase 1b/2 ALPHA-STAR trial.
- The designation provides regulatory and financial incentives for developing treatments for rare conditions in the EU.
- A Phase 3 trial for navenibart is expected to commence in the first quarter of 2025.
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Alzamend Neuro reveals full data from phase IIA trial for AL001 in Alzheimer's dementia
- Alzamend Neuro has completed a phase IIA trial for AL001, determining a maximum tolerated dose (MTD) for Alzheimer's dementia.
- The MTD delivers lithium at a dose of 240 mg, three times daily, potentially eliminating the need for traditional therapeutic drug monitoring.
- AL001 is designed to overcome the toxicities of conventional lithium salts, offering a safer alternative for fragile populations.
- Alzamend plans to initiate five phase II trials to evaluate increased lithium levels in the brain compared to marketed lithium salts.
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Adicet Bio receives FDA clearance for IND amendment to evaluate ADI-001 in autoimmune diseases
- Adicet Bio's IND amendment for ADI-001 in idiopathic inflammatory myopathy (IIM) and stiff person syndrome (SPS) has been cleared by the FDA.
- The Phase 1 trial will now include IIM and SPS, with patient enrollment starting in Q1 2025.
- The trial will have four arms, with IIM and SPS patients in the fourth arm, receiving a single dose of ADI-001.
- Primary objectives are safety and tolerability, with secondary objectives including cellular kinetics and disease activity scores.
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Theriva Biologics receives orphan medicinal product designation for VCN-01
- The European Commission granted orphan medicinal product designation to VCN-01 for retinoblastoma treatment.
- VCN-01 is an oncolytic adenovirus designed to selectively replicate within tumor cells and degrade tumor stroma.
- The designation provides benefits like 10 years of market exclusivity and reduced regulatory fees.
- Theriva plans to refine its clinical strategy for VCN-01 as an adjunct to chemotherapy in pediatric retinoblastoma.
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Cullinan Therapeutics receives FDA clearance for CLN-978 in lupus trial
- Cullinan Therapeutics' IND application for CLN-978 has been cleared by the FDA.
- The Phase 1 trial will assess CLN-978 in patients with moderate to severe systemic lupus erythematosus (SLE).
- The trial includes a dose escalation phase to determine the target dose and a dose expansion phase to explore multiple schedules.
- Primary objective is to evaluate safety, with secondary objectives including pharmacokinetics and clinical activity.
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ARTHEx Biotech announces promising preclinical research for myotonic dystrophy type 1
- ARTHEx Biotech published research in Science Advances on a dual mechanism of anti-miRs for DM1 treatment.
- The study focused on AntimiRs targeting miR-23b and miR-218, which restore MBNL1 levels and correct cellular defects.
- AntimiR-23b treatment showed significant reduction in toxic DMPK mRNA and reversed 68% of dysregulated genes.
- The research highlights potential therapeutic avenues for DM1, a disease with no current approved treatments.
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Neurogastrx announces positive proof-of-concept data for NG101
- Neurogastrx reported positive results from a study of NG101, a dopamine D2 receptor antagonist, reducing nausea and vomiting from GLP-1 agonist semaglutide.
- The study was a randomized, double-blind, placebo-controlled proof-of-concept trial with 90 participants aged 18-55.
- NG101 reduced nausea incidence by 40% and vomiting frequency by 56%, with no new safety concerns or serious adverse events.
- Plans are underway to discuss with the FDA for a larger Phase 2/3 study in 2025.
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Update on Novavax's COVID-19-influenza combination and stand-alone influenza phase 3 trial
- The FDA has placed a clinical hold on Novavax's IND application for its COVID-19-Influenza Combination and stand-alone influenza vaccine candidates.
- The hold is due to a serious adverse event of motor neuropathy reported in a Phase 2 trial participant.
- The trial was completed in July 2023, and the adverse event was reported in September 2024.
- Novavax is working with the FDA to resolve the hold and aims to start the Phase 3 trial soon.
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