RARE Daily

ProQR Winds Down Clinical Trials to Focus Exclusively on Axiomer RNA-editing Technology

August 11, 2022

ProQR Therapeutics, following the European Medicines Agency recommendation that the company conduct an additional phase 3 study of sepofarsen, its experimental RNA therapy designed to restore vision in people with Leber congenital amaurosis, said it will now focus exclusively on its Axiomer RNA-editing technology platform.

Photo: Daniel de Boer, CEO of ProQR Therapeutics

The company said to continue advancement of its portfolio of ophthalmic product candidates, including sepofarsen for LCA10 and ultevursen (QR-421a) for USH2A-mediated Usher syndrome and retinitis pigmentosa, the company will seek to identify a strategic partner to take the ophthalmology portfolio forward.

To preserve operating capital, and until a partner is found that can fund the clinical programs moving forward, the current ongoing trials of sepofarsen and ultevursen – including Illuminate, Insight, and Brighten for sepofarsen, along with Sirius and Helia for ultevursen – will be wound down. For people currently participating in these trials, ProQR will offer continued access to currently available sepofarsen or ultevursen.

In April, following the pivotal late-stage trail failure of its lead candidate sepofarsen as a treatment for the severe inherited rare eye disease CEP290-mediated Leber congenital amaurosis 10, or LCA10, the company announced it would pare its workforce by 30 percent, about 50 employees including Chief Scientific Officer Naveed Shams, and streamline its pipeline to extend its cash runway and deliver on its commitment to advance RNA therapies for diseases with high unmet need.

Based on the wind down of sepofarsen and ultevursen clinical trials and associated workforce reduction, ProQR will realize cost savings in headcount, program, and related support activities, which are expected to extend ProQR’s cash runway into 2026. The cash runway into 2026 excludes any revenue generated from the company’s existing partnership and any potential new partnering deals.

Axiomer is ProQR’s next-generation RNA technology, which could potentially yield a new class of medicines for genetic diseases. Axiomer “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer EONs are designed to recruit an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G).

The company said it continues to execute on its global licensing and research partnership with Eli Lilly, focused on the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system, and may selectively enter additional partnerships designed to advance and capture the full potential value of the platform.

“The feedback we received from the EMA is helpful in designing an additional registration trial for sepofarsen based on the learnings from the Illuminate trial and we will seek a strategic partner for the further development of our ophthalmology programs, including sepofarsen and ultevursen,” said Daniel de Boer, CEO of ProQR Therapeutics.

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.