4D Molecular Therapeutics Announces First Patient Dosed in Phase 1/2 Trial of 4D-125 for the Treatment of XLRP
4D Molecular Therapeutics (4DMT) announced that the first patient has been dosed in the phase 1/2 clinical trial of 4D-125 for X-linked retinitis pigmentosa (XLRP). 4D-125 is an AAV gene therapy with an optimized and proprietary vector designed to deliver a functional copy of the RPGR gene to photoreceptors in the retina. This vector enables both administration by intravitreal injection, a safe and routine clinical route, plus the potential to treat broader and earlier-stage patient populations as compared to subretinal approaches. 4D-125 is a 4DMT-declared ophthalmology therapeutic candidate under 4DMT’s collaboration with Roche, where Roche has an option to take an exclusive world-wide license prior to initiation of a pivotal trial.
“4DMT’s Therapeutic Vector Evolution platform harnesses the power of directed evolution to develop precision-guided AAV gene medicines. The result is products with characteristics optimized for specific diseases, including enhanced transduction, optimized delivery route, improved tolerability, lower doses, and antibody-resistance,” David Kirn, MD, co-founder, chairman and chief executive officer of 4DMT, said in a company news release. “Dosing the first patient in the phase 1/2 clinical trial of 4D-125 marks the second of three Therapeutic Vector Evolution pipeline candidates expected to enter the clinic in 2020. I am grateful for the XLRP patient community, their families and caregivers, and the clinical trial physicians and staff, who share in our vision of a next-generation gene therapy for XLRP patients.”
“Presently there is no effective treatment for people suffering from this devastating disease, which leads to vision loss and ultimately blindness,” Dr. Marc Mathias, Assistant Professor, Ophthalmology, University of Colorado School of Medicine and a principal investigator on 4DMT’s phase 1/2 clinical trial of 4D-125, said in the news release. “4DMT’s gene therapy approach for XLRP aims to address the underlying cause of XLRP through the therapeutic delivery of the RPGR gene into patients’ retinal cells to produce functional protein. Furthermore, 4D-125 is promising for patients relative to other gene therapy approaches because it is delivered by intravitreal injection, a routine clinical route of administration.”
The phase 1/2 open-label, dose-exploration and dose-expansion study is expected to enroll approximately 18 male patients with X-linked retinitis pigmentosa. The study is designed to assess the preliminary safety, tolerability and biological activity of a single intravitreal injection of 4D-125. In addition, the clinical trial will evaluate the effect of 4D-125 on the visual function and retinal degeneration.