Viridian Therapeutics announces positive topline results from veligrotug (VRDN-001) phase 3 THRIVE clinical trial in patients with active thyroid eye disease
- Viridian Therapeutics reported positive topline data from the THRIVE phase 3 trial of veligrotug (VRDN-001) in patients with active thyroid eye disease (TED).
- The trial met primary and all secondary endpoints at 15 weeks, showing significant improvements in proptosis, diplopia, and clinical activity score (CAS).
- Veligrotug demonstrated a rapid onset of action, with 53% of patients achieving a proptosis response after just one infusion.
- The safety profile was favorable, with most adverse events being mild and a low rate of discontinuations (4%).
- Viridian plans to submit a Biologics License Application (BLA) for veligrotug in the second half of 2025.
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Stoke Therapeutics presents zorevunersen data showing substantial reductions in seizures and improvements in cognition and behavior in Dravet syndrome
- Stoke Therapeutics presented data at the 15th European Epilepsy Congress showing zorevunersen (STK-001) significantly reduces seizures and improves cognition and behavior in Dravet syndrome.
- Phase 1/2a ADMIRAL study data showed improvements in cognition and behavior during the first year of treatment, with further increases as treatment continued.
- Two-year natural history study indicated high seizure rates and plateaued neurodevelopment despite standard-of-care antiseizure medications.
- Plans for a Phase 3 registrational study are underway, focusing on dose regimen and clinical endpoints.
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T-Neuro Pharma's trailblazing Alzheimer's research receives acclaim in PNAS commentary
- T-Neuro Pharma's Alzheimer's research highlighted in a PNAS commentary.
- Research challenges the amyloid cascade hypothesis, suggesting T cell changes may initiate Alzheimer's pathology.
- Potential paradigm shift in Alzheimer's research, focusing on T cell changes rather than amyloid deposits.
- New research directions could lead to novel therapeutic approaches for Alzheimer's disease.
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Incendia Therapeutics enrolls first patient in Phase 1c clinical trial of PRTH-101, a novel DDR1 inhibitor
- Incendia Therapeutics has enrolled the first patient in the Phase 1c study of PRTH-101 for advanced or metastatic solid tumors.
- The study will evaluate the safety, tolerability, and anti-tumor activity of PRTH-101 as a monotherapy and in combination with pembrolizumab.
- PRTH-101 targets DDR1, a collagen binding protein, to disrupt tumor-associated collagen alignment and permit immune cell access.
- The trial aims to enroll up to 270 patients in the US and will inform the design of the Phase 2/3 program.
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Delta-Fly Pharma initiates phase I/II combo-study of DFP-10917 with Venetoclax in AML patients
- Delta-Fly Pharma has initiated a Phase I/II clinical trial of DFP-10917 in combination with Venetoclax (VEN) for AML patients.
- The trial is approved by the US FDA and the first patient was enrolled at the University of Virginia Hospital.
- The study aims to enroll up to 39 patients, focusing on endpoints such as complete remission (CR) rate and progression-free survival (PFS).
- Successful completion of this study may lead to cooperation with a mega pharmaceutical company for a New Drug Application (NDA).
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Basking Biosciences doses first patients in phase 2 clinical trial of reversible thrombolytic BB-031 for acute ischemic stroke
- Basking Biosciences has initiated patient enrollment in the RAISE Phase 2 clinical trial for BB-031.
- The trial will evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of BB-031 in 156 patients.
- RAISE is a multicenter, double-blind, placebo-controlled, randomized single ascending dose study.
- BB-031 targets von Willebrand Factor (vWF) and aims to offer a safer, more effective thrombolytic option for acute ischemic stroke patients.
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Inflammasome Therapeutics completes enrollment in study for first oral dual inflammasome inhibitor for ophthalmic and neuroinflammatory diseases
- Inflammasome Therapeutics has completed enrollment for a Phase I PK/Safety study of its drug K9.
- K9 targets neuroinflammatory and degenerative diseases like Alzheimer’s, MS, ALS, Parkinson’s, and autoimmune diseases such as lupus.
- The drug penetrates the brain and retina, blocking two specific inflammasomes, showing superior results compared to single inflammasome inhibitors.
- The successful completion of this study will pave the way for further clinical trials expected to commence later this year.
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Nxera Pharma’s partner Centessa announces positive interim phase 1 clinical data with its novel orexin receptor 2 (OX2R) agonist, ORX750, in acutely sleep-deprived healthy volunteers
- ORX750 showed significant improvements in mean sleep latency in acutely sleep-deprived healthy volunteers.
- The drug demonstrated a favorable safety and tolerability profile with no frequently reported adverse events.
- Centessa plans to advance ORX750 into Phase 2 studies in Q4 2024 for narcolepsy type 1, narcolepsy type 2, and idiopathic hypersomnia.
- The 2.5 mg dose restored normative wakefulness with a mean sleep latency of 32 minutes in the Maintenance of Wakefulness Test (MWT).
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Kallyope announces license agreement with Novo Nordisk
- Kallyope has licensed a novel ligand to Novo Nordisk for further development, including potential obesity treatment.
- Novo Nordisk will handle preclinical and clinical development, manufacturing, and commercialization.
- Kallyope received an upfront fee and may receive additional milestone payments and royalties.
- The collaboration leveraged Kallyope's Klarity platform to identify potential peptide-based therapies.
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Reveal Genomics announces positive top-line results for HER2DX in CLEOPATRA phase III trial
- Reveal Genomics released positive results for HER2DX, a genomic test for HER2+ breast cancer, from the CLEOPATRA phase III trial.
- The HER2DX ERBB2 mRNA score showed a strong association with progression-free survival (PFS) and overall survival (OS) in 214 patients treated with THP.
- The CLEOPATRA trial compared docetaxel-trastuzumab with and without pertuzumab, leading to the FDA approval of pertuzumab in 2012.
- Detailed results will be presented at an upcoming medical congress and submitted for publication.
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