ProQR Therapeutics sets goals for 2023

The company is developing a platform of RNA medicines to correct genetic defects in patients with inherited retinal diseases.

ProQR Therapeutics, a company focused on treating rare diseases, announced plans to advance its pipeline of RNA medicines to treat inherited retinal diseases to include two fully approved commercial products in Europe and the U.S., three late-stage clinical therapies and seven early-stage therapies by 2023.

The company, founded in 2012, is developing a platform of RNA medicines to correct genetic defects in the patient’s RNA without having to modify DNA, ProQR CEO Daniel A. de Boer told Ocular Surgery News.

“This allows us to target a wide variety of diseases. Our therapies go to both the central and peripheral parts of the retina. ... This allows us to treat such diseases as retinitis pigmentosa or Usher syndrome,” he said.

The therapies in the ProQR pipeline utilize an RNA oligonucleotide technology platform to repair genetic defects, targeting significant parts of the mutations that are known causes of inherited retinal diseases.

Using this new platform, de Boer noted there are more than 100 potential opportunities for targeted drug development.

“We can help a tremendous number of patients who currently have no treatment options available for their diseases and no hope because nobody else is really making an attempt to help their disease,” he said.

By 2021, the company hopes to submit a new drug application to the FDA for sepofarsen, a treatment for Leber’s congenital amaurosis 10. It is designed to treat the disease by restoring normal mRNA, which then results in normal CEP290 protein production to stop or reverse the effects of the disease.

The therapy is currently in two clinical trials, with early results indicating positive outcomes, de Boer said.

After one dose of the drug, patients went from “not being able to see anything to being able to read letters on a letter chart and being able to play sports,” de Boer said.

The drug is currently in a phase 2/3 study for which the company expects to have topline data by 2020. If the results are as expected, de Boer said the company hopes to submit the product for approval by 2021.

All therapies in the ProQR ophthalmology pipeline are delivered intravitreally, allowing them to get a “very broad distribution” and exposure to the entire retina.

“This is a major advantage. This technology allows patients to have a routine, noninvasive procedure that has a very broad impact on the disease. It treats not only the central vision, but also the peripheral vision,” he said.

The next product in the pipeline is QR-421a, a therapy for Usher syndrome caused by mutations in exon 13 of the USH2A gene. It is in the phase 1/2 STELLAR proof-of-concept clinical trial and could be registered as a new product by 2023, de Boer said.

“In the next 18 months, we’ll have five programs in clinical trials. ... This year we’ll have three ongoing clinical trials for sepofarsen, QR-421a and QR-1123, a treatment for autosomal dominant retinitis pigmentosa caused by the P23H mutation,” he said.

In the first half of 2020, the company plans to begin clinical trials for QR-411a for the PE40 mutation causing Usher syndrome and QR-504 for Fuchs’ endothelial corneal dystrophy. – by Robert Linnehan

Disclosure: De Boer reports he is the CEO of ProQR.

ProQR Therapeutics, a company focused on treating rare diseases, announced plans to advance its pipeline of RNA medicines to treat inherited retinal diseases to include two fully approved commercial products in Europe and the U.S., three late-stage clinical therapies and seven early-stage therapies by 2023.

The company, founded in 2012, is developing a platform of RNA medicines to correct genetic defects in the patient’s RNA without having to modify DNA, ProQR CEO Daniel A. de Boer told Ocular Surgery News.

“This allows us to target a wide variety of diseases. Our therapies go to both the central and peripheral parts of the retina. ... This allows us to treat such diseases as retinitis pigmentosa or Usher syndrome,” he said.

The therapies in the ProQR pipeline utilize an RNA oligonucleotide technology platform to repair genetic defects, targeting significant parts of the mutations that are known causes of inherited retinal diseases.

Using this new platform, de Boer noted there are more than 100 potential opportunities for targeted drug development.

“We can help a tremendous number of patients who currently have no treatment options available for their diseases and no hope because nobody else is really making an attempt to help their disease,” he said.

By 2021, the company hopes to submit a new drug application to the FDA for sepofarsen, a treatment for Leber’s congenital amaurosis 10. It is designed to treat the disease by restoring normal mRNA, which then results in normal CEP290 protein production to stop or reverse the effects of the disease.

The therapy is currently in two clinical trials, with early results indicating positive outcomes, de Boer said.

After one dose of the drug, patients went from “not being able to see anything to being able to read letters on a letter chart and being able to play sports,” de Boer said.

The drug is currently in a phase 2/3 study for which the company expects to have topline data by 2020. If the results are as expected, de Boer said the company hopes to submit the product for approval by 2021.

All therapies in the ProQR ophthalmology pipeline are delivered intravitreally, allowing them to get a “very broad distribution” and exposure to the entire retina.

“This is a major advantage. This technology allows patients to have a routine, noninvasive procedure that has a very broad impact on the disease. It treats not only the central vision, but also the peripheral vision,” he said.

The next product in the pipeline is QR-421a, a therapy for Usher syndrome caused by mutations in exon 13 of the USH2A gene. It is in the phase 1/2 STELLAR proof-of-concept clinical trial and could be registered as a new product by 2023, de Boer said.

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“In the next 18 months, we’ll have five programs in clinical trials. ... This year we’ll have three ongoing clinical trials for sepofarsen, QR-421a and QR-1123, a treatment for autosomal dominant retinitis pigmentosa caused by the P23H mutation,” he said.

In the first half of 2020, the company plans to begin clinical trials for QR-411a for the PE40 mutation causing Usher syndrome and QR-504 for Fuchs’ endothelial corneal dystrophy. – by Robert Linnehan

Disclosure: De Boer reports he is the CEO of ProQR.