Santhera announces positive topline results from LIONHEART study with vamorolone
- Santhera Pharmaceuticals announced positive results from the LIONHEART study, confirming vamorolone’s unique action as a mineralocorticoid receptor antagonist.
- The study involved 30 healthy adult male subjects and demonstrated a significant increase in the urinary sodium/potassium ratio in the vamorolone arm compared to placebo.
- These findings further differentiate vamorolone’s pharmacological profile, setting it apart from other corticosteroids.
- Further analysis of the data is ongoing and will be presented at medical conferences.
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CLINUVEL files Canadian new drug submission for SCENESSE in EPP
- CLINUVEL has submitted a New Drug Submission (NDS) to Health Canada for SCENESSE (afamelanotide) to prevent phototoxicity in adult EPP patients.
- If approved, SCENESSE would be the first treatment available for EPP patients in Canada.
- The submission includes data from FDA approval and long-term follow-up studies.
- Two Canadian Specialty Centers are already trained to treat EPP patients with SCENESSE, with more centers identified for future treatment access.
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KalVista submits additional marketing authorization applications for sebetralstat
- KalVista Pharmaceuticals has submitted Marketing Authorization Applications (MAAs) for sebetralstat in the UK, Switzerland, Australia, and Singapore.
- The submissions are part of the Access Consortium framework, which aims to streamline regulatory collaboration and review processes.
- Sebetralstat is an investigational oral plasma kallikrein inhibitor for on-demand treatment of hereditary angioedema (HAE) in patients aged 12 and older.
- The MAAs are supported by data from the KONFIDENT phase 3 trial and the KONFIDENT-S open label extension trial, showing significant efficacy and safety.
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Precision BioSciences submits first clinical trial applications for PBGENE-HBV
- Precision BioSciences has submitted Clinical Trial Applications (CTA) to initiate a Phase 1 study for PBGENE-HBV.
- PBGENE-HBV aims to potentially cure chronic hepatitis B by eliminating cccDNA and inactivating integrated HBV DNA in hepatocytes.
- The regulatory submissions are supported by robust non-human primate safety studies and efficacy in preclinical models.
- The company plans to initiate the Phase 1 study soon and expects to report data in 2025.
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Adaptive Research begins enrolling patients in AriBio’s Phase 3 POLARIS-AD clinical trial
- Adaptive Research has started patient enrollment for AriBio’s Phase 3 POLARIS-AD trial.
- The trial is a double-blind, randomized, placebo-controlled study evaluating AR1001 in early Alzheimer's disease.
- Two community practices within the Adaptive Research network are participating in the trial.
- AriBio aims to enroll 1150 participants globally to assess the efficacy and safety of AR1001 over 52 weeks.
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FDA grants rare pediatric disease designation to MDL-101 for congenital muscular dystrophy type 1a
- Modalis Therapeutics announced that the FDA has granted Rare Pediatric Disease (RPD) designation to MDL-101.
- MDL-101 is being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD).
- RPD designation is given to treatments for serious diseases affecting children under 18 and fewer than 200,000 people in the U.S.
- Modalis has also submitted an Orphan Drug application for MDL-101, which is under separate review by the FDA.
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BPGbio receives FDA rare pediatric disease designation for primary coenzyme Q10 deficiency treatment
- BPGbio's BPM31510IV has been granted FDA Rare Pediatric Disease designation for primary CoQ10 deficiency.
- Primary CoQ10 deficiency is an ultra-rare mitochondrial disorder affecting fewer than 1 in 100,000 people.
- The designation makes BPGbio eligible for a priority review voucher upon BPM31510's approval.
- BPGbio is preparing to initiate a trial targeting multiple CoQ10 deficiency mutations.
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Astria Therapeutics receives FDA orphan drug designation for navenibart for the treatment of hereditary angioedema
- Navenibart (STAR-0215) has been granted Orphan Drug Designation by the FDA for treating hereditary angioedema (HAE).
- Navenibart is a monoclonal antibody inhibitor of plasma kallikrein, designed for long-acting attack prevention.
- Initial results from the Phase 1b/2 ALPHA-STAR clinical trial show a 90-96% reduction in monthly attack rates.
- Astria plans to share additional results in Q4 and initiate a Phase 3 trial in Q1 2025.
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Enhertu granted priority review in the U.S. for patients with HER2 low or HER2 ultralow metastatic breast cancer
- Daiichi Sankyo and AstraZeneca's sBLA for Enhertu has been accepted and granted Priority Review by the FDA.
- The application is for treating adult patients with unresectable or metastatic HER2 low or HER2 ultralow breast cancer who have received at least one line of endocrine therapy.
- The Priority Review is based on data from the DESTINY-Breast06 phase 3 trial, which showed Enhertu reduced the risk of disease progression or death by 37% compared to chemotherapy.
- The FDA action date for the regulatory decision is February 1, 2025.
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InCarda Therapeutics announces results from the RESTORE-1 Phase 3 trial and plans for continued development
- RESTORE-1 Phase 3 trial showed statistically significant cardioversion of PAF with inhaled flecainide despite early termination.
- The trial was stopped prematurely due to lower-than-expected efficacy and plasma flecainide levels, not safety concerns.
- Transition to a novel drug-delivery platform by Aerogen Ltd. aims to improve the efficiency and reliability of flecainide delivery.
- Preliminary Phase 1 results with the new platform show promising pharmacokinetics and safety, supporting further development.
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