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Tofersen Shows Promise in Phase 1/2 Trial for Inherited Form of ALS

5 years ago

Biogen's experimental drug tofersen demonstrated safety and successfully lowered levels of disease-causing SOD1 protein in patients with a rare inherited form of ALS in a phase 1/2 clinical trial.
The antisense oligonucleotide treatment reduced SOD1 protein concentrations by up to 33% in the high-dose group, with some indications it may slow disease progression, prompting advancement to phase 3 trials.
While targeting only approximately 2% of ALS patients with SOD1 mutations, researchers believe this approach could establish a platform for treating other forms of ALS and neurodegenerative conditions through protein regulation.

7 years ago

The FDA has approved emapalumab-lzsg (Gamifant) as the first treatment specifically indicated for primary hemophagocytic lymphohistiocytosis (HLH), an ultra-rare and life-threatening immune disorder.
In a pivotal phase 2/3 study of 27 patients with refractory disease, 63% demonstrated overall response and 70% proceeded to hematopoietic stem cell transplant.
The monoclonal antibody targets interferon gamma, representing the first significant improvement in primary HLH induction therapy in 24 years.
Primary HLH typically affects children within the first year of life and has a median survival of less than two months without treatment.

NCT01818492CompletedPhase 2

Swedish Orphan Biovitrum

Posted 7/1/2013