Synthekine granted U.S. FDA fast track designation for CD19 CAR-T and orthogonal IL-2 investigational therapy
- Synthekine's SYNCAR-001 + STK-009 therapy has received Fast Track designation from the FDA for treating severe, refractory systemic lupus erythematosus (SLE) without lymphodepletion.
- SYNCAR-001 is an autologous CD19-targeting CAR-T cell therapy, while STK-009 is an engineered IL-2 cytokine.
- The therapy is currently being tested in a Phase 1 study for CD19+ hematologic malignancies and has an IND application cleared for a Phase 1 study in non-renal SLE and lupus nephritis (LN).
- The multi-center, dose escalation clinical trial will assess the safety and clinical activity of SYNCAR-001 + STK-009 in patients with non-renal SLE and LN.
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Vaxcyte announces pricing of $1.3 billion public offering
- Vaxcyte is selling 10,194,175 shares of common stock and pre-funded warrants to purchase 2,427,184 shares.
- Common stock is priced at $103.00 per share, and pre-funded warrants at $102.999 per warrant.
- The offering is expected to generate approximately $1.3 billion in gross proceeds.
- The offering is set to close on September 6, 2024, subject to customary closing conditions.
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Adaptin Bio announces FDA clearance of IND application for APTN-101 in glioblastoma
- Adaptin Bio has received FDA clearance for its IND application for APTN-101 in glioblastoma (GBM).
- APTN-101 is a BRiTE therapeutic targeting EGFRvIII, a protein linked to aggressive brain tumors.
- The Phase 1 clinical trial will evaluate the safety and efficacy of APTN-101 in patients with WHO Grade IV Malignant Glioma.
- Preclinical studies showed a 7-fold increase in brain distribution and significant potential in eradicating malignant glioma tumors.
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ALX Oncology announces first patients dosed with evorpacept and Sarclisa in phase 1/2 UMBRELLA study with Sanofi
- ALX Oncology and Sanofi have dosed the first patients in the UMBRELLA phase 1/2 clinical study.
- The study evaluates evorpacept in combination with Sarclisa and dexamethasone in patients with relapsed or refractory multiple myeloma (RRMM).
- Part 1 of the study focuses on determining the optimal dose of evorpacept, while Part 2 assesses the efficacy and safety of the combination.
- Sanofi will conduct the trial, and ALX Oncology will supply evorpacept.
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ADVANZ PHARMA secures temporary suspension of the European Commission decision on the Ocaliva conditional marketing authorisation in Europe
- The General Court of the European Union has temporarily suspended the European Commission's decision to revoke the conditional marketing authorisation (CMA) for Ocaliva (obeticholic acid).
- The CMA for Ocaliva remains valid in Europe, including EU Member States and the European Economic Area countries, until further notice.
- Ocaliva will continue to be available for new and existing patients with primary biliary cholangitis (PBC) across Europe.
- The suspension ensures immediate continuity of supply until the next decision by the General Court.
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The UK’s Medicines & Healthcare Products Regulatory Agency grants CEL-SCI a pediatric waiver for Multikine
- CEL-SCI received a pediatric waiver from the UK's MHRA for Multikine in treating head and neck cancer.
- The waiver means CEL-SCI won't need to evaluate Multikine in a pediatric population for UK marketing clearance.
- Multikine has shown a 73% survival rate in its target patient population compared to 45% without it.
- CEL-SCI is preparing for a confirmatory FDA Registration Study in the U.S. and other regions.
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IGC Pharma advances IGC-AD1 toward clinical trials as a potential anti-amyloid plaque treatment for Alzheimer's disease
- IGC Pharma is advancing its proprietary formulation, IGC-AD1, toward clinical trials for Alzheimer's disease.
- IGC-AD1 targets amyloid plaques through two mechanisms: inhibiting amyloid protein production and preventing its aggregation.
- Preliminary data suggests IGC-AD1 may reduce plaque aggregation by approximately 20% and decrease its production by up to 40%.
- The initiation of disease-modifying investigational trials is expected in early 2025.
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Pharvaris presents deucrictibant long-term extension data for HAE treatment
- Long-term prophylaxis data shows a 93% reduction in HAE attacks over one year.
- On-demand treatment data indicates a median onset of symptom relief in 1.1 hours.
- 85.8% of HAE attacks resolved completely within 24 hours with deucrictibant.
- Deucrictibant was well-tolerated with no new safety signals observed.
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Crestone announces positive data from phase 2 clinical trial of CRS3123 for C. difficile infections
- Crestone reported positive topline results from the Phase 2 trial of CRS3123 in patients with Clostridioides difficile infection (CDI).
- Clinical cure rates at day 12 were 97% for CRS3123 and 93% for vancomycin, with no clinical failures.
- CDI recurrence rates at day 40 were significantly lower for CRS3123 (4%) compared to vancomycin (23%).
- The National Institute of Allergy and Infectious Diseases (NIAID) has provided $4.5 million in new funding for further studies.
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Verona Pharma to present additional analyses of phase 3 ENHANCE studies in COPD at ERS International Congress 2024
- Verona Pharma will present one oral presentation and three posters on Ohtuvayre (ensifentrine) at the ERS International Congress 2024.
- Ohtuvayre is a first-in-class, selective dual inhibitor of PDE3 and PDE4, combining bronchodilator and non-steroidal anti-inflammatory effects.
- The analyses will cover efficacy and safety in subgroups, including patients with and without chronic bronchitis, and European patients.
- A pooled analysis of patient-reported outcomes, including the effect on reducing cough and sputum, will also be presented.
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