Get all your news in one place.
100’s of premium titles.
One app.
Start reading
Benzinga
Benzinga
Business
Vandana Singh

Korro Bio Early Trial Results Fall Short; Stock Hits New Low

Medical,Development,Laboratory:,Caucasian,Female,Scientist,Looking,Under,Microscope,,Analyzes

Korro Bio, Inc. (NASDAQ:KRRO) stock is sinking on Thursday with a session volume of 6.68 million compared to the average volume of 1.42 million, as per data from Benzinga Pro.

The genetic medicines company provided a program update for its Phase 1/2a REWRITE clinical trial of KRRO-110 in alpha-1 antitrypsin deficiency (AATD).

AAT deficiency is a genetic disorder where the body doesn't produce enough of the AAT protein, which protects the lungs and liver from damage.

Also Read: Sanofi Drug Shows Superior Efficacy In Genetic Disease Compared To Current Standard Therapy

The company announced that KRRO-110 generated functional M-AAT protein in AATD patients. While a single administration of KRRO-110 achieved functional protein production, it did not achieve the protein levels projected based on preclinical data.

Across five AATD patients in the two single ascending dose cohorts evaluable with turbidimetry, the greatest peak total AAT protein was approximately 10 µM, and the greatest increase of total AAT protein from baseline was approximately 3 µM.

The total AAT protein levels following single-dose administration did not reach the protective threshold of 11 µM.

No dose-limiting toxicities or treatment-emergent serious adverse events observed.

Pipeline Restructure

"To focus our resources on generating clinical data and advancing additional GalNAc-conjugated programs targeting the liver, we are implementing a strategic restructuring that reduces our workforce by approximately a third while extending our cash runway into the second half of 2027," said Ram Aiyar, CEO and President of Korro Bio.

Korro has nominated KRRO-121 as its next development candidate for hyperammonemia, including patients with urea cycle disorders (UCD) and hepatic encephalopathy (HE).

Regulatory filing to enable commencement of first-in-human trial for KRRO-121 is anticipated in the second half of 2026.

Korro amended its research collaboration and license agreement with Novo Nordisk A/S (NYSE:NVO). This amendment establishes a 12-month pause to reassess the rationale for the current target under the first research program.

Korro reduced its workforce by approximately 34%.

Cash, cash equivalents, and marketable securities were $102.5 million, extending the cash runway into the second half of 2027 by implementing a strategic restructuring.

Analyst Take

William Blair downgraded Korro shares from Outperform to Market Perform.

Analyst Myles Minter said, "On the one hand, the presence of M-AAT in AATD patients is yet another validation of viable ADAR-mediated editing in humans; on the other hand, the KRRO-110 clinical profile looks non-competitive at the moment."

Analyst Minter said, "We are still believers in ADAR editing and are intrigued by Korro's expansion into urea cycle disorders with KRRO-121, but with Novo placing its collaboration on pause and Korro moving back to a preclinical-stage company, we are moving to the sidelines for now."

Price Action: KRRO stock is down 80% at $6.27 at the last check on Thursday.

Read Next:

Photo via Shutterstock

Sign up to read this article
Read news from 100’s of titles, curated specifically for you.
Already a member? Sign in here
Related Stories
Top stories on inkl right now
One subscription that gives you access to news from hundreds of sites
Already a member? Sign in here
Our Picks
Fourteen days free
Download the app
One app. One membership.
100+ trusted global sources.