Roth Capital Partners launched coverage of Intellia Therapeutics (NASDAQ:NTLA) with a “neutral” rating and $16 price target, saying the stock is fully valued ahead of first-in-human studies. The stock closed at $14.36, down 70 cents, on June 10.
Intellia is a gene-editing company focused on developing CRISPR/Cas9-based therapeutics. Its lead program is for in-vivo editing to find a potentially curative treatment option for transthyretin amyloidosis (ATTR).
Analyst Tony Butler writes that ATTR is a progressive condition characterized by a build-up of abnormal amyloids in tissues, but clinical improvements can be achieved from rather low transthyretin (TTR) editing rates of 40%. In addition, in the liver, where TTR is produced, transduction is rather efficient.
He said Intellia has presented data, which showed a 78% TTR edit rate, and a 96% reduction in TTR. From a safety perspective, clearance of the LNP delivery system and the CRISPR/Cas9 components has been shown.
The IND for the ATTR clinical candidate, NTLA-2001, which is being developed in partnership with Regeneron, is expected to be submitted during 2020 and the appropriate IND enabling toxicity studies are on track. “The submission timeline was delayed (originally expected during 2019), which did create some overhang on the stock as other gene-editing companies entered the clinic,” he added.
Intellia also has several early-stage programs, including an in-vivo program with Regeneron for Hemophilia B; an ex-vivo AML program using gene-edited TCRs; and on-going collaboration with Novartis to develop ex-vivo technologies.
