SpringWorks Therapeutics announces FDA approval of GOMEKLI for NF1-PN
- GOMEKLI (mirdametinib) is approved by the FDA for treating NF1-PN in patients aged 2 and older.
- Approval is based on Phase 2b ReNeu trial results showing significant tumor volume reduction and a manageable safety profile.
- SpringWorks received a rare pediatric disease priority review voucher from the FDA.
- GOMEKLI is expected to be available in the U.S. within two weeks, with an EMA decision anticipated in 2025.
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Abcuro secures $200 million to advance therapy for inclusion body myositis
- Abcuro has closed a $200 million Series C financing round led by New Enterprise Associates.
- Funds will support the Phase 2/3 MUSCLE trial of ulviprubart, targeting inclusion body myositis (IBM).
- Ulviprubart is a monoclonal antibody designed to deplete cytotoxic T cells, sparing other T cell types.
- Assuming positive trial results, Abcuro plans to file a BLA and prepare for commercial launch.
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Cognito Therapeutics announces Spectris treatment demonstrates myelin and synaptic biomarker changes in Alzheimer’s disease
- Cognito Therapeutics published a study showing Spectris' impact on myelin and synaptic biomarkers in Alzheimer's patients.
- The FLICKER clinical trial involved 10 participants with amyloid-positive MCI using Spectris daily for 8 weeks.
- Significant changes in 110 proteins linked to Alzheimer's pathways were observed, suggesting a therapeutic effect.
- The study supports Spectris' potential to modulate core mechanisms driving Alzheimer's pathology.
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Lineage initiates clinical study of OPC1 for spinal cord injury
- Lineage Cell Therapeutics has launched the DOSED clinical study to evaluate the safety and utility of the MI PSD System for delivering OPC1 in spinal cord injury patients.
- The study will include both subacute and chronic spinal cord injury patients, marking the first time OPC1 is administered to chronic patients.
- OPC1 is an allogeneic stem cell-derived transplant designed to replace or support dysfunctional cells in the spinal cord, potentially improving mobility and quality of life.
- The study will be conducted at UC San Diego Health, with long-term safety monitoring ongoing from previous trials showing no unexpected serious adverse events.
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Algernon NeuroScience appoints Dr. Sándor Nardai as principal investigator for phase 2a DMT stroke study
- Algernon NeuroScience has appointed Dr. Sándor Nardai as the Principal Investigator for its upcoming Phase 2a DMT study.
- The study will be a randomized, double-blind, placebo-controlled trial involving 40 stroke patients in Europe.
- Dr. Nardai is a leading stroke expert and has previously conducted significant research on DMT's effects on stroke recovery.
- Patient enrollment for the study is expected to begin in Q3 of 2025.
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