Zinger Key Points
- iECURE's ECUR-506 showed promising safety and efficacy in a Phase 1/2 study for neonatal-onset OTC deficiency, with no major safety issues.
- BMO Capital Markets upgraded Precision BioSciences to Outperform with a $34 target, citing ARCUS validation and HBV program potential.
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On Thursday, Precision BioSciences Inc.’s DTIL partner iECURE reported clinical efficacy and safety data in the first patient dosed with ECUR-506 in the Phase 1/2 OTC-HOPE study.
ECUR-506 is iECURE’s in vivo gene insertion program designed to treat neonatal-onset Ornithine Transcarbamylase (OTC) deficiency. It utilizes a PCSK9-specific ARCUS nuclease, licensed from Precision, that enables the insertion of a functional copy of the OTC gene.
Ornithine transcarbamylase deficiency is a rare genetic condition that causes ammonia to build up in the blood. The condition is more common in boys than girls and tends to be more severe when symptoms emerge shortly after birth.
Treatment with ECUR-506 was generally well tolerated in first infant dosed with no significant clinical safety concerns apart from asymptomatic transaminitis at four weeks.
BMO Capital Markets upgraded Precision BioSciences, “Acknowledging the N=1, iECURE data appear very promising, providing early clinical validation for ARCUS.”
Analyst Kostas Biliouris writes that early ECUR-506 efficacy provides initial clinical derisking of in vivo ARCUS efficacy/editing platform.
In November, Precision BioSciences shared preclinical data for the PBGENE-HBV program that reinforced the safety profile and potential to deliver a functional cure for chronic hepatitis B, supporting advancement into first-in-human clinical study.
The company is conducting ELIMINATE-B Phase 1 trial. ELIMINATE-B is open and currently screening and accruing patients, and Precision expects to report data from the study as it matures throughout 2025.
BMO Capital analyst writes that the HBV readout in 2025 could drive a 100-200%+ stock upside.
On Thursday, BMO upgraded from Market Perform to Outperform with a price target of $34 based on ECUR-506 initial clinical derisking of in vivo ARCUS editing and a proprietary gene editing platform with a differentiated mechanism and potentially competitive.
Price Action: DTIL stock is up 25.8% at $5.66 at last check Friday.
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