Precision BioSciences Shares Surge 60% After Breakthrough Clinical Results for OTC Deficiency Treatment

January 10, 2025 – Precision BioSciences, Inc. (NASDAQ: DTIL) saw a remarkable 60% surge in its stock price following the announcement of a complete clinical response in the first infant dosed in a Phase 1/2 clinical trial aimed at treating Ornithine Transcarbamylase (OTC) deficiency, a rare genetic disorder.

Positive Clinical Trial Results

The trial, conducted in collaboration with iECURE, showcased promising results in the OTC-HOPE study, where the infant received treatment with ECUR-506, a gene-editing therapeutic leveraging Precision BioSciences’ ARCUS® gene editing platform.

The key highlights of the study include:

• The infant showed no significant clinical safety concerns, with only asymptomatic transaminitis observed, which was managed and resolved with immunosuppressive therapy.
• Twelve weeks after the treatment, the infant was able to discontinue ammonia scavenger medication and significantly increase protein intake without elevating ammonia levels, a major marker of the disorder.
• Six months post-treatment, the positive response remained intact, indicating a complete clinical response.

These results underscore the potential of ARCUS gene editing technology to perform sophisticated gene edits in vivo, with applications for gene insertion, excision, and elimination.

Ongoing Clinical Developments

The OTC-HOPE study continues to enroll patients across multiple countries and is expected to be completed in 2025. Full program data is anticipated to be available by the first half of 2026.

In addition to this breakthrough, Precision BioSciences is advancing its ELIMINATE-B trial for PBGENE-HBV, targeting chronic hepatitis B. Phase 1 data for this trial is expected to be released in 2025, further expanding the company’s gene-editing portfolio.

Analyst Upgrades and Future Outlook

The promising data has led to a price target upgrade from BMO Capital analyst Kostas Biliouris, who upgraded Precision BioSciences from Market Perform to Outperform with a new price target of $34.00. Biliouris noted the early clinical validation of the ARCUS platform and estimated a 65% probability for Precision’s HBV program to achieve acceptable safety and strong efficacy, which could lead to significant stock gains, similar to the performance of other companies in the gene-editing space.

The analyst further highlighted the favorable risk/reward profile for Precision BioSciences, given its valuation at about one-third of its net cash position, which extends its financial runway well into the second half of 2026.