Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec). Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted in Elevidys’ prescribing information.
While data on functional endpoints are still to come, Avidity Biosciences Inc. executives said the firm is moving ahead with plans for a BLA filing by the end of 2025 for del-zota, an antibody-oligonucleotide conjugate, in Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44), based on positive top-line data that analysts say bode well for Avidity’s other late-stage programs targeting rare neuromuscular diseases.
Bristol Myers Squibb Co.’s decision this week to snag Bluebird Bio Inc. spinout 2seventy Bio Inc. for $102 million net – just weeks after investors bid $30 million for Bluebird itself – seemed to place a final blow on what was once a promising gene therapy company. The space in general has struggled to make business sense out of the one-time therapies that often involve complicated manufacturing and exorbitant prices, despite the life-changing value that gene therapies bring to patients. But despite some recent setbacks, biopharmas continue to plow forward with promising research in the field.
The Vivani Medical Inc. combination appears to be over less than three years since its inception after the company reported plans to spin off Cortigent, the division that develops brain implants. Vivani said it plans to create two focused companies dedicated to driving current and future value in their respective therapeutic areas.
Positive 16-week extension data from Cervomed Inc.’s phase IIb in dementia with Lewy bodies followed a failure from December. New results from the Rewind-LB trial testing neflamapimod, a brain-penetrant, orally administered small molecule that inhibits the intracellular enzyme p38MAP kinase alpha, have encouraged the company to pursue a phase III study.
The BioWorld Neurological Diseases Index closed 2024 down 20.27%, extending its decline from -13.4% at the end of November. After falling to -12.2% in April, the index briefly rebounded, narrowing losses to under 4% by June, before experiencing a sharp downturn in the second half of the year.
Trimtech Therapeutics closed a £25 million (US$31 million) oversubscribed seed funding round to advance its targeted protein degradation treatments for neurodegenerative and inflammatory diseases.
Persica Pharmaceuticals Ltd. has delivered positive 12-month follow-up data from its phase Ib study of PP-353, an injectable antibiotic for treating chronic lower back pain, and is now looking for a partner and further financing to take the product into phase III development.
The march toward better drugs in Parkinson’s disease suffered a setback as Irlab Therapeutics AB rolled out top-line results from the phase IIb study with pirepemat called React-PD.
In an advance that could significantly lighten the load for caretakers in the “sandwich generation” and reduce loneliness in elderly patients, Aspargo Labs Inc. developed a metered delivery device that optimizes absorption of pharmaceuticals and reminds users to take their medications.
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