Biogen higher after tofersen study shows potential for ALS treatment

On Wednesday night, Biogen (BIIB) announced interim results of a phase 1/2 study of tofersen, an antisense oligonucleotide, or ASO, being studied for the potential treatment of amyotrophic lateral sclerosis, or ALS, in adults with a confirmed superoxide dismutase 1 or SOD1, mutation. The data will be presented at the American Academy of Neurology Annual Meeting in Philadelphia. “The interim results of this study, which achieved proof-of-biology and proof-of-concept, support the initiation of a phase 3 clinical trial to confirm the efficacy and safety of tofersen in SOD1-ALS patients and further demonstrate the potential of ASOs to target the genetic driver of disease,” said Michael Ehlers, M.D., Ph.D., executive vice president, research and development at Biogen. “We are committed to bringing a potential breakthrough therapy to patients with ALS and we are expediting our efforts with the aim of addressing this urgent unmet need.” PROGRESSIVE DISEASE: ALS, also called Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. People with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death. The average life span after diagnosis is two to five years. Approximately 10% of all ALS cases are genetic and about a fifth of those are caused by SOD1 gene mutations, says the American Academy of Neurology or AAN.”The treatment that we researched in this study, an antisense oligonucleotide called tofersen or BIIB067, works by targeting and reducing protein created by the mutated gene,” said study author Timothy M. Miller, MD, PhD, of Washington University School of Medicine in St. Louis and a member of the American Academy of Neurology. “That mutated protein is toxic and leads to ALS by damaging the nerve cells that control movement. Our research aimed to decrease the production of that protein.” OWNING BIOGEN MAKES SENSE: According to Piper Jaffray analyst Christopher Raymond, owning Biogen shares at these levels “makes a lot of sense.” With valuation and sentiment near all-time lows, the company needs only a few things to go right in order for the stock to start working again, says the analyst. He believes a number of near term catalysts, namely a settlement with Mylan (MYL) over Tecfidera and positive pipeline updates, could “meaningfully change the narrative” on Biogen shares. Raymond kept an Overweight rating on the name with a $280 price target in a note to investors out earlier on Thursday. According to BioSpace, Sanofi (SNY) makes a drug to treat ALS by the name of Rilutek. It was the first drug approved by the FDA to treat ALS, but “it’s no cure for ALS,” though it can slow progression of the disease.

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