ProQR Therapeutics hopes further analysis of the failure of a clinical trial of its lead RNA therapy will save the experimental eye drug, but European regulators have told the biotech that another clinical trial is needed. Instead of doing this research, ProQR plans to discontinue ophthalmology research and instead focus on other applications of its RNA editing technology.
Further development of ProQR’s two most advanced ophthalmic drug candidates, sepofarsen and ultevursen, will depend on another company. ProQR, based in Leiden, the Netherlands, said on Thursday it was looking for a strategic partner for all of its ophthalmic assets. ProQR estimates it will save enough cash to support the company through 2026 by ending clinical trial work on its two most advanced RNA candidates.
Sepofarsen has performed pivotal testing in Leber’s congenital amaurosis10, an inherited eye disease characterized by degeneration or dysfunction of retinal photoreceptors. RNA therapy injected into the eye aims to repair the genetic mutation at the root of the disease.
Earlier this year, ProQR reports that sepofarsen fails phase 2/3 study, the results published in the treatment group did not differ from the control group given sham surgery.But the company sees a glimmer of light data analysis After the study is completed. When comparing the treated eyes with untreated eyes in the same patients, improved vision was observed at month 12, ProQR said. This effect was not observed in those who received the sham injection.
The results of the additional analysis were not enough to convince the European Medicines Agency, but ProQR chief executive Daneil de Boer said in a prepared statement that the agency’s regulatory feedback helped design additional clinical trials that could support a marketing authorization application. This information will be useful to any company looking to acquire or partner to purchase sepofarsen or other ProQR ophthalmic assets. The ultevursen program is enrolling patients in pivotal Phase 2/3 studies in Usher syndrome, a rare genetic disorder that causes vision and hearing loss, and retinitis pigmentosa, another degenerative retinal disease. RNA therapy saw encouraging results in phase 1/2 testing last year.
Going forward, ProQR will focus on its RNA editing technology platform, Axiomer. Initial therapeutic areas will include the liver and central nervous system (CNS). Both areas are consistent with the company’s approach to supplying oligonucleotides, which are short strands of synthetic RNA, ProQR said. The liver and central nervous system are also areas of focus for an Axiomer-based research partnership with Eli Lilly. The deal paid ProQR $50 million up front and could bring in up to $1.25 billion in additional revenue, depending on how research progresses. ProQR said it may forge more partnerships with the platform.
As ProQR continues its strategic shift, the company expects to announce new pipeline targets by the end of this year or early next, according to one company Investor introduction. The company also hopes to provide an update on its partnership with Eli Lilly.In its Report In its financial results for the second quarter of 2022 last week, ProQR said it had 156.4 million euros in cash and cash equivalents as of June 30.
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