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Biotech / Medical : Biotech Valuation

CRSP 56.98

+1.1%

3:59 PM EST

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To: A.J. Mullen who wrote (52048)	4/25/2023 2:50:15 PM
From: Art Bechhoefer	Read Replies (1) of 52153

I listened to the IONS presentation on eplontersen. The 35 and 66 week data were impressive. Ionis expects the drug will be approved shortly after its FDA submission in December and is already in the process of manufacturing and commercializing it. Among comments at the conference call, they pointed out that there were no side effects and that the drug is designed for self injection once a month. Most of the data were already known, at least in general terms, before the conference call. I have no idea why the stock has remained depressed. By the way, the April 21 issue of Science (AAAS weekly journal) mentions another Ionis drug, not by name, but by association with research on spinal muscular atrophy. When the Ionis drug was combined with gene editing treatment on mice, the improvement was better than from gene editing alone. This result is similar to what Ionis and Biogen are seeing as they do clinical trials on Spinraza, administered to patients previously treated with gene editing, which by itself did not stop SMA progression. Art

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