Crispr Treatments Show Early Promise

"Intellia Therapeutics Inc. reported encouraging early-stage study results for its Crispr gene-editing treatments, the latest sign that the pathbreaking technology could result in commercially available drugs in the coming years.

Intellia said Friday that one of its treatments, code-named NTLA-2002, significantly reduced levels of a protein that causes periodic attacks of swelling in six patients with a rare genetic disease called hereditary angioedema, or HAE.

In a separate study building on previously released trial data, Intellia's treatment NTLA-2001 reduced a disease-causing protein by more than 90% in 12 people with transthyretin-mediated amyloidosis cardiomyopathy, or ATTR-CM, a genetic disease that can lead to heart failure.

Despite the positive results, questions remain about whether therapies based on Crispr will work safely and effectively, analysts said.

Intellia's latest studies involved a small number of patients, and were disclosed in news releases and haven't been published in a peer-reviewed journal. The NTLA-2002 study results were presented at the Bradykinin Symposium in Berlin, a medical meeting focused on angioedema.

The data came from small, so-called Phase 1 studies conducted in New Zealand and the U.K. that didn't include control groups. Results from such early studies can be unreliable predictors of a drug's safety and effectiveness once the compound is tested in larger numbers of patients.

Intellia shares fell 4.2% in trading on Friday, partly due to safety concerns because liver enzyme levels temporarily rose in some people in the studies.

The findings, nevertheless, add to preliminary but promising evidence of the potential for drugs based on the gene-editing technology. Last year, Intellia said that NTLA-2001 reduced the disease-causing protein involved in ATTR patients.

"We've shown that the first time wasn't an aberration," Intellia Chief Executive John Leonard said.

In the HAE study, three patients were given a 25-milligram treatment dose and had their protein levels reduced by 65% on average over at least 16 weeks. Before the study, the patients typically experienced 1.1 to 7.2 swelling attacks a month; the attacks were reduced by 91% on average over 16 weeks, Intellia said.

The other three HAE patients received a 75-milligram dose. Intellia, based in Cambridge, Mass., said the study subjects had even greater average protein reductions of 92%, but it couldn't report their swelling attack rates because the subjects hadn't completed the observation period yet.

People in the HAE study were allowed to keep taking previously prescribed drugs to prevent swelling attacks, which is likely to have biased the results in favor of Intellia's treatment, BMO Capital Markets analyst Kostas Biliouris said in a note to clients.

Intellia said some patients in the two studies reported on Friday had temporary, low-grade elevations of enzymes measured in lab tests for signs of liver damage. The patients didn't experience any symptoms, and their enzyme levels later returned to normal, Intellia said.

Still, the liver enzyme signals seen in the small studies "hint at increased risks as trials get larger," Citi's David Lebowitz said in a client note." [1]

 

 We have Crispr specialists in Lithuania, but we have no practical results. We need to seriously think about why.

 

1. U.S. News: Crispr Treatments Show Early Promise
Walker, Joseph. 
Wall Street Journal, Eastern edition; New York, N.Y. [New York, N.Y]. 17 Sep 2022: A.6.