A gene-editing treatment from Intellia Therapeutics led to elevated liver enzymes in one patient with polyneuropathy, prodding NTLA stock to topple Thursday.
The biotech company is testing its treatment in patients with transthyretin amyloidosis, a condition in which abnormal protein builds up on the organs. In this study, patients experience that build up on their nerves, leading to systemic problems.
Intellia says one patient who received a higher dose of its drug experienced increased liver enzymes. The patient didn't have any symptoms and the investigator determined the event wasn't serious. The incident led Intellia to back off that heightened dose in a study of patients with the same disorder who experience built-up protein on their heart, known as cardiomyopathy.
NTLA stock tumbled 8.7% to close at 65.43 on the stock market today.
NTLA Stock: Similar Responses To Doses
RBC Capital Markets analyst Luca Issi said the sell-off was overdone.
Intellia noted the doses led to similar reductions in the culprit protein in polyneuropathy patients. The low dose led to an average 86% reduction, while the high dose resulted in a 93% decline. At the maximum, the two doses led to a respective 97% and 98% reduction.
"Importantly, backing off the dose should not drive a detrimental impact on (effectiveness) as the company announced today that the two doses were indistinguishable in the new (cardiomyopathy) data that we have yet to see," Issi said.
He kept his outperform rating and 150 price target on NTLA stock.
Alnylam Update Promising
Despite Issi's upbeat take-away, NTLA stock gave back its nearly 12% gain from Wednesday. Bullish news from Alnylam Pharmaceuticals helped shares pop. Alnylam stock rocketed more than 49% on Wednesday.
Alnylam said its drug helped cardiomyopathy patients walk farther over six minutes — a marker of their heart fitness. Recipients of its drug also reported better quality of life on a questionnaire.
Alnylam is taking a different approach. Whereas Intellia is using gene editing, Alnylam is testing a drug that turns off the gene responsible for making the aberrant protein.
"We are buyers into the weakness as we think (polyneuropathy) data in hand materially de-risks the platform," Issi said of NTLA stock.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
