Biogen picks up Ionis SOD1 treatment option

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lgelb

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This is important not just for advancing SOD1 tx, but establishing another case where a smaller firm (where a lot of ALS work is being done) has successfully handed off a therapy to a larger firm. This helps incentivize ALS research.

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[Adapted from Seeking Alpha] Having seen positive early-stage data, Biogen has exercised its option to develop and commercialize Ionis Pharmaceuticals' IONIS-SOD1 rx (BIIB067) for the treatment of SOD1 mutations, representing ~2% of total ALS cases.

The action triggers a $35M milestone payment to Ionis, who is eligible for up to $55M in milestones and low-to-mid-teen royalties on net sales.
 

lgelb

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More good news around this compound. At AAN this week, results of a 12-week study were/will be announced. Here are some snippets. As noted in the report, limitations are the 12-week time period and only 50 patients (at varying doses). So we cannot jump for joy just yet, but it's promising.

"Participants who received the 100 mg dose experienced an average 1.1-point decline [on the ALSFRS] compared with an average 5.3-point decline among those in the placebo group. The 100 mg treatment group also performed better on slow vital capacity and muscle strength measures.

The differences were even more pronounced between the 100 mg and placebo groups in participants with SOD1 mutations known to be rapidly progressive, the investigators note.

The majority of adverse events were mild or moderate in severity and included headache, pain due to the procedure, and post-lumbar puncture syndrome."
 
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