Neuvivo’s NP001 shows promise in extending survival for ALS patients
- Neuvivo's NP001 immunotherapy demonstrated significant benefits in ALS patients, slowing lung function decline and extending survival.
- A new analysis showed NP001-treated patients had a median survival of 58 months compared to 36 months for the control group.
- The study highlighted a 50% reduction in vital capacity loss, suggesting improved respiratory function contributed to survival benefits.
- NP001 has been granted Orphan Drug and Fast Track Designations by the FDA, indicating its potential as a novel ALS treatment.
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Tiziana Life Sciences announces positive results in treating spinal cord injury with nasal anti-CD3
- Tiziana Life Sciences reported promising preclinical results using nasal anti-CD3 monoclonal antibody for spinal cord injury (SCI).
- The treatment showed significant improvements in motor functions by modulating microglial inflammation.
- Nasal anti-CD3 is part of Tiziana's broader strategy to expand its technology from multiple sclerosis to SCI.
- The company is advancing its intranasal foralumab, currently in a Phase 2a trial for multiple sclerosis.
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Amneal receives U.S. FDA approval for memantine/donepezil and everolimus, and tentative approval for rifaximin
- The FDA approved memantine/donepezil extended-release capsules for moderate to severe Alzheimer's dementia, with 180-day exclusivity.
- Everolimus tablets for oral suspension received approval for treating TSC-associated SEGA in patients aged one year or older.
- Tentative approval was granted for rifaximin tablets for IBS-D, pending litigation resolution.
- U.S. annual sales for these products are significant, with rifaximin sales reaching $2.6 billion.
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āshibio doses first patient in ANDECAL study for FOP treatment
- āshibio has dosed the first participant in the ANDECAL study, a Phase 2/3 trial for andecaliximab in fibrodysplasia ossificans progressiva (FOP).
- The trial evaluates safety, efficacy, pharmacokinetics, and pharmacodynamics of andecaliximab, a humanized antibody targeting MMP-9 enzyme.
- The study is divided into two parts, with Part 1 currently underway in the U.S., involving two cohorts of FOP patients.
- The FDA has granted andecaliximab a Rare Pediatric Disease Designation for FOP treatment.
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Oryzon announces first patient dosed in phase I study of iadademstat in myelodysplastic syndrome
- Oryzon Genomics has initiated a Phase I trial of iadademstat, a selective LSD1 inhibitor, in combination with azacitidine for myelodysplastic syndrome (MDS).
- The study, led by the Medical College of Wisconsin, aims to evaluate the safety, tolerability, and recommended Phase II dose of the combination therapy.
- MDS is a hematological malignancy with limited treatment options, and the trial seeks to address the unmet need for new therapies.
- The trial is part of Oryzon's broader strategy to explore iadademstat's potential in various hematologic and solid cancers.
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Larimar Therapeutics begins dosing adolescents in study for Friedreich’s ataxia
- Larimar Therapeutics has started dosing adolescents aged 12-17 in a pediatric pharmacokinetic (PK) run-in study for Friedreich’s ataxia.
- Participants receive a weight-based dose equivalent to the 50 mg adult dose and are randomized 2:1 to receive either nomlabofusp or placebo.
- Adolescents completing the PK run-in study can screen for the ongoing open label extension (OLE) study.
- A cohort for children aged 2-11 is planned for the first half of 2025, with long-term data expected mid-2025.
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