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Soraprazan - a new regenerative therapy for Stargardt's disease

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A possible cure for a potentially blinding disease

The results of a phase II study could lead to the first treatment for Stargardt disease, an eye condition that can cause blindness.

Health

Although relatively rare, Stargardt disease affects about 1 in every 10 000 people. The rare genetic eye disease occurs when fatty material called lipofuscin builds up on a part of the retina that is critical for sight. The disease typically first manifests during childhood, with vision becoming progressively worse with age. At some point, most people with Stargardt will become legally blind. While there are methods to mitigate Stargardt symptoms, there is still no cure. However, there is hope – thanks in part to work being done by companies like Katairo, a pharmaceutical company dedicated to developing a drug to treat Stargardt disease. With the support of EU funding for project Soraprazan incorporating a consortium of medical research organisations, the company has developed a promising proof of concept drug called Remofuscin. “The EU funding has been instrumental in advancing our drug and conducting a clinical phase II study in Stargardt patients,” says Katairo CEO Wolfgang Klein. As Klein explains, a phase II study focusses on assessing an investigational drug candidate’s safety and efficacy. “Our tests, which were the longest continuous application of our drug candidate in humans to date, showed good tolerability,” he explains. “This finding allowed us to extend the duration of the treatment, and we hope to get even more telling results on the drug’s effectiveness soon.”

Overcoming challenges

Achieving this finding was anything but easy. For example, the project’s use of non-CE marked equipment to measure its primary end point raised several regulatory eyebrows. “Our study took place in four different European countries, each of which has its own regulatory environment,” says Klein. “Different regulatory authorities raised different questions, so it took a varying amount of work to satisfy everyone’s requirements.” As with any clinical study, recruiting qualified patients was also a challenge, but Katairo expected this and was able to take steps to minimise delays. What they didn’t expect was to have their study take place during a global pandemic. “Recruiting had to be stopped and visits had to be done via video,” notes Klein. “But, thanks to a dedicated team and excellent collaboration across the consortium, we were able to adjust, and the study was conducted as planned.”

Results expected soon

The extended study is expected to conclude at the end of 2022, with initial results available by early 2023. “Everything now depends on the efficacy data,” adds Klein. “If we see that the drug helps patients, then it may soon be the first treatment for Stargardt disease.” According to Klein, a positive outcome could also open the door to using the drug to treat other eye diseases such as dry age-related macular degeneration (AMD). Even if the data does not suggest efficacy, Klein stresses that all is not lost. “Sometimes we can learn as much from what doesn’t work as from what does,” he concludes. “Such information could open the door to new routes that ultimately lead to a cure for this terrible, blinding disease.”

Keywords

Soraprazan, disease, Stargardt disease, eye condition, eye disease, blindness, Katairo, Remofuscin, clinical study

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