ProQR Therapeutics reported encouraging safety and pharmacokinetic data from the first cohort of its AX‑0810 Phase 1 trial, the first human test of its Axiomer RNA‑editing platform. The trial, conducted in the Netherlands, enrolled 24 participants receiving AX‑0810 and 9 receiving placebo across three dose cohorts, with the data presented coming from the 3 mg/kg cohort.
No serious adverse events or clinically meaningful laboratory abnormalities were observed in the 3 mg/kg cohort, and pharmacokinetic parameters matched pre‑clinical expectations, supporting continued dosing as planned. This milestone validates the platform’s safety profile in healthy volunteers and sets the stage for the target‑engagement readout scheduled for the first half of 2026.
ProQR also highlighted progress on its broader pipeline. The company has selected development candidates for AX‑2402, targeting MECP2 for Rett syndrome, and AX‑2911, targeting the PNPLA3 I148M mutation for metabolic‑associated steatohepatitis. Pre‑clinical data for AX‑2402 showed statistically significant functional improvements in a mouse model, while AX‑2911 demonstrated over 80 % reduction in hepatic fat content in a humanized mouse model. First‑in‑human trials for AX‑2402 are expected in the first half of 2027.
The collaboration with Eli Lilly generated $4.5 million in milestone payments in 2025, providing non‑dilutive capital that extends ProQR’s cash runway into mid‑2027. The company’s nine‑month net loss for the period ended September 30 2025 was €33.3 million, up from €18.5 million in the same period a year earlier, underscoring the need for continued investment in its pipeline.
Chief Medical Officer Cristina Lopez Lopez emphasized that the initial human data confirm the platform’s safety and pharmacokinetic profile, while Chief Executive Officer Daniel de Boer noted that the company’s pipeline breadth and partnership with Lilly reinforce confidence in the Axiomer technology’s potential to transform treatment for rare and metabolic diseases.
Investors reacted cautiously, with market sentiment tempered by concerns over ProQR’s financial trajectory and valuation, despite the positive clinical data. The company’s next milestone—target‑engagement data—will be critical in demonstrating the platform’s ability to edit RNA in vivo and could unlock broader therapeutic opportunities and attract additional partnerships or financing.
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