ProQR Therapeutics N.V. announced positive target engagement data from its ongoing Phase 1 clinical study evaluating AX-0810 in healthy volunteers, while simultaneously securing $50 million through an underwritten offering to fund development. The data demonstrated dose-dependent target engagement on all key biomarkers in the evaluable 3 mg/kg and 6 mg/kg cohorts, with total bile acids in serum increasing up to 8-fold. This exceeded the 2-fold threshold identified by the company as a meaningful indicator of NTCP modulation, signaling potential efficacy for the investigational Axiomer RNA editing oligonucleotide.

AX-0810 is a GalNAc-conjugated, subcutaneously administered Axiomer RNA editing oligonucleotide (EON) designed to selectively modulate NTCP, a transporter responsible for bile acid uptake from the bloodstream into the liver. The study enrolled 33 healthy volunteers, including 24 participants receiving AX-0810 and nine receiving placebo across the 3 mg/kg, 6 mg/kg, and 9 mg/kg cohorts. Results released covered 22 participants treated in the 3 mg/kg and 6 mg/kg cohorts. The company observed a favorable safety and tolerability profile to date, with no serious adverse events or pruritus reported. Pharmacokinetic findings support sustained target engagement, including a half-life of eight weeks. Hormone levels remained unchanged, suggesting the therapy selectively edits the NTCP bile acid binding pocket while preserving other protein functions.

Key Clinical Findings

The biomarker findings support the advancement of the next-generation candidate AX-0811 and future clinical studies in biliary atresia. AX-0811 demonstrated robust cholestasis reduction in a preclinical cholestasis animal model and is projected to support at least 4-fold higher editing at lower dose levels, with a projected half-life greater than three months. ProQR expects to submit a Clinical Trial Application (CTA) in mid-2026 for AX-0811 and anticipates initial human clinical data in healthy volunteers by year-end 2026.

Capital Raise and Strategic Investment

To support its pipeline and corporate operations, ProQR priced an 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 agreed to purchase approximately 5.1 million shares for about $9.2 million to maintain its pro rata ownership stake. 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.

Upcoming Milestones

• AX-0810 data from the 9 mg/kg cohort and 12-week follow-up expected by year-end 2026.
• AX-0811 CTA filing in mid-2026, with initial data in healthy volunteers expected by year-end 2026.
• Investigator-initiated trial in pediatric biliary atresia in China, with initial data targeted for the first half of 2027.
• Potentially registration-enabling Phase 2 program expected to start in mid-2027, with first interim analysis data expected by mid-2028.

ProQR Therapeutics N.V. has secured approximately $59.2 million in gross proceeds through a registered direct offering and a concurrent private placement to fund research and clinical development. The company priced an underwritten registered direct offering of 27,624,310 ordinary shares at $1.81 per share, yielding about $50.0 million before expenses. Separately, Eli Lilly and Company agreed to purchase 5,100,780 ordinary shares at the same price for approximately $9.2 million to maintain its pro rata beneficial ownership stake.

Transaction Details

The registered direct offering and the concurrent private placement are distinct transactions. The sale to Lilly is not registered as part of the Offering and is not subject to underwriting discounts or commissions. The closing of the Offering is not contingent on the private placement, though both are expected to finalize on or about June 26, 2026, subject to customary conditions.

Regulatory and Use of Proceeds

The shares in the concurrent private placement are issued pursuant to Section 4(a)(2) of the Securities Act of 1933 and have not been registered under the Securities Act or applicable state securities laws. ProQR intends to use the net proceeds from both offerings, alongside existing cash, primarily to fund research and clinical development of its pipeline candidates, as well as for working capital and general corporate purposes.

BofA Securities, Evercore ISI, and Cantor are acting as joint lead bookrunning managers, while Oppenheimer & Co. serves as a bookrunning manager. A shelf registration statement on Form F-3 was filed with the SEC on September 30, 2024, and declared effective on October 10, 2024.