ProQR Restructures, Cuts Workforce and Select Rare Disease Programs

Rare Daily Staff

ProQR Therapeutics has joined a growing list of companies paring their workforce and programs to stay afloat during a bear market on Wall Street and after a trial failure, which sent shares down 67 percent in February.

After 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, in February, the RNA therapeutics-focused biotech says it will 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.

Although ProQR’s antisense therapeutic sepofarsen failed to beat placebo on any efficacy measures in the Illuminate study, the company says it has taken another look at the topline data and sees “an encouraging efficacy signal when comparing the active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints and are consistent with feedback received from the investigators,” said Aniz Girach, chief medical officer of ProQR. “While we were disappointed by the outcome of the primary analysis, we believe that these post-hoc analyses and the observation that approximately a third of the patients benefited across multiple concordant endpoints in this trial, in combination with the high unmet need in LCA10, warrants a discussion with the regulators.”

The company plans to meet with U.S. and EU regulatory authorities in the third quarter of this year to figure out next steps, and will continue the 2-year study, an extension study, and a pediatric trial. But it said it has no plans to invest in any new trials of sepofarsen or prepare for commercialization at this time.

Among its portfolio prioritization and restructuring initiatives with the aim to reduce expenses, ProQR said it will continue developing the ultevursen (QR-421a) program for USH2A-mediated Usher syndrome and retinitis pigmentosa on a single phase 2/3 Sirius trial with the potential addition of an interim/futility analysis in 2023.

The company will suspend development of QR-1123 for autosomal dominant retinitis pigmentosa and QR-504a for Fuchs endothelial corneal dystrophy and all other internal R&D activities.

Suspend all other IRD-related research activities; and

Reduce the workforce by approximately 30%, which will include the departure of our Chief Scientific Officer Naveed Shams, MD, PhD, expected to be effective in Q2.

ProQR said it will accelerate the development of its Axiomer RNA editing platform and pipeline activities and expand into areas beyond the eye, including initially liver and CNS, which have strong alignment with ProQR’s oligonucleotide delivery approaches.  In parallel, the company will continue to execute on its partnership with Lilly and selectively enter into additional partnerships designed to advance and capture the full potential value of the platform.

“We are focusing our strategy on accelerating our Axiomer RNA-base editing platform technology, and a select pipeline of RNA therapies for inherited retinal diseases as we remain committed to developing RNA therapies for patients with high unmet need,” said Daniel de Boer, founder and CEO of ProQR Therapeutics. “ProQR has a strong cash position with runway into 2025, and we look forward to continued progress with the business, including providing an update following our discussions in Q3 with the EMA and FDA, and sharing details of our development plans for Axiomer in the second half of 2022.”

Photo: Daniel de Boer, founder and CEO of ProQR Therapeutics