ProQR Therapeutics Reports Q3 2025 Loss of €33.3 Million, Cash Runway to Mid‑2027, AX‑0810 Phase 1 Launch

PRQR
November 06, 2025

ProQR Therapeutics reported a nine‑month net loss of €33.3 million for the period ended September 30 2025, a widening from the €18.5 million loss recorded for the same period last year. Research and development expenses climbed to €34.8 million, up from €25.7 million, while general and administrative costs rose to €11.2 million versus €9.7 million. Cash and cash equivalents stood at €106.9 million, down from €149.4 million at the end of 2024, and the company used €39.4 million of cash in operating activities, an increase from €27.0 million in the prior year. A milestone payment of $2.0 million (≈ €1.8 million) from its collaboration with Eli Lilly was recorded during the quarter.

The company’s earnings per share of –$0.12 fell short of the consensus estimate of –$0.10, a miss of $0.02 or roughly 20 %. Revenue for the nine months was $3.4 million, missing the consensus estimate by about 60 %. The miss reflects the fact that ProQR remains a pre‑revenue company; the modest revenue generated is largely from milestone payments and early‑stage clinical activity, while the bulk of the loss is driven by the company’s aggressive investment in its RNA‑editing platform and the initiation of the AX‑0810 Phase 1 study.

Cash burn of €39.4 million per quarter translates to a runway of roughly 2.5 years, extending into mid‑2027. The decline in cash from €149.4 million to €106.9 million is largely attributable to the increased R&D spend and the absence of significant product sales. The milestone payment from Lilly partially offsets the burn, but the company’s runway remains contingent on future milestone receipts and the successful progression of its clinical pipeline.

The launch of the AX‑0810 Phase 1 study in healthy volunteers marks a critical milestone for ProQR’s flagship program targeting NTCP for cholestatic diseases. The study’s initiation follows a Clinical Trial Application (CTA) authorization, and the company expects initial safety and pharmacokinetic data by year‑end 2025, with target‑engagement data in the first half of 2026. The milestone payment from Lilly underscores the partnership’s value and provides a potential source of additional funding if subsequent milestones are achieved.

CEO Daniel A. de Boer emphasized that the company’s “strong cash position provides runway into mid‑2027, giving management time to achieve key clinical milestones and potentially secure additional milestone payments from its Lilly partnership.” He added that the AX‑0810 program “represents the first clinical evaluation of an Axiomer editing oligonucleotide and positions us to deliver initial safety and PK data before year‑end, followed by target engagement data in the first half of 2026.” These comments highlight the company’s focus on de‑risking its pipeline while maintaining financial flexibility.

Overall, ProQR’s Q3 2025 results illustrate the typical trade‑off for an early‑stage biotech: a widening loss driven by heavy R&D investment, a modest cash burn that still supports a multi‑year runway, and a strategic partnership that offers both milestone revenue and a path to future product development. The company’s next milestones—clinical data from AX‑0810 and potential additional Lilly payments—will be key indicators of its trajectory toward a first‑in‑class RNA‑editing therapy.

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