ProQR Therapeutics N.V. (NASDAQ:PRQR) on Thursday reported positive early-stage clinical data for its investigational RNA editing therapy AX-0810, while simultaneously announcing a $50 million underwritten offering aimed at supporting development of its pipeline and broader corporate operations.
AX-0810 Demonstrates Strong Early Activity In Phase 1 Trial
The clinical-stage biotechnology company said findings from its ongoing Phase 1 multiple ascending dose study showed evidence of target engagement for AX-0810, its first investigational Axiomer RNA editing oligonucleotide, in healthy volunteers.
AX-0810 is designed to selectively modulate NTCP, a transporter involved in bile acid uptake into the liver.
The therapy aims to reduce toxic bile acid buildup associated with cholestatic liver diseases, including biliary atresia.
The ongoing study enrolled 33 healthy volunteers, including 24 participants receiving AX-0810 and nine receiving a placebo across three dose cohorts. Results released Wednesday covered 22 participants treated in the 3 mg/kg and 6 mg/kg cohorts.
According to ProQR, AX-0810 produced dose-dependent increases in serum bile acids of up to eightfold, surpassing the company’s predefined twofold threshold for meaningful NTCP modulation.
Additional biomarker changes, including increased circulating TUDCA levels and higher urinary excretion of conjugated bile acids, further supported target engagement.
Safety Profile Supports Continued Development
The company said AX-0810 demonstrated a favorable safety and tolerability profile in the evaluated cohorts. No serious adverse events or cases of pruritus were reported, while pharmacokinetic data indicated an estimated half-life of approximately eight weeks.
ProQR added that hormone levels remained unchanged, suggesting the therapy selectively edits the NTCP bile acid binding pocket while preserving other protein functions.
The company expects to present complete Phase 1 findings at a medical or scientific conference later this year.
Separately, ProQR highlighted progress with AX-0811, a next-generation NTCP-targeting RNA editing candidate developed using its AI-enabled discovery platform.
The company plans to submit a Clinical Trial Application for AX-0811 in mid-2026.
ProQR Raises Capital To Advance Pipeline
Alongside the clinical update, ProQR priced its underwritten registered direct offering of 27.6 million shares at $1.81 per share, generating gross proceeds of approximately $50 million.
In a separately negotiated transaction, existing shareholder and strategic partner Eli Lilly and Co (NYSE:LLY) agreed to purchase approximately 5.1 million shares for about $9.2 million to maintain its pro rata ownership stake.
ProQR said proceeds from the offering, the concurrent private placement, and existing cash resources will primarily fund research and clinical development activities, working capital, capital expenditures, and other general corporate purposes.
PRQR Price Action: ProQR Therapeutics shares were down 12.71% at $1.58 at the time of publication on Thursday, according to Benzinga Pro data.
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