ProQR Therapeutics Announces FDA Orphan Drug Designation for QR-1123 in Autosomal Dominant Retinitis Pigmentosa

On November 25, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that biotechnology company ProQR Therapeutics (ProQR) announced that its therapeutic candidate QR-1123 for autosomal dominant retinitis pigmentosa (ADRP) had received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).

Orphan Drug Designation provides preferential treatment for drugs targeting rare diseases that are currently in the development stage. Upon receiving FDA Orphan Drug Designation, a drug becomes eligible for benefits such as tax credits for clinical development costs and waivers of New Drug Application (NDA) fees. Furthermore, the sponsoring company is granted seven years of market exclusivity following the drug’s commercial approval.

Autosomal dominant retinitis pigmentosa (ADRP) is a severe, rare genetic disorder. Patients typically present with symptoms such as night blindness during childhood, and there is a high probability of progressing to blindness by middle age. Studies have shown that ADRP may be caused by the P23H mutation in the rhodopsin (RHO) gene, leading to misfolding of the RHO protein. The misfolded protein exerts toxicity on photoreceptor cells and simultaneously impairs the function of the wild-type allele. Over time, this results in the gradual death of visual cells, causing progressive vision loss. Currently, there are no approved therapies available for P23H-associated ADRP.

QR-1123 is an RNA oligonucleotide therapeutic discovered by Ionis Pharmaceuticals using its proprietary antisense technology, primarily indicated for the treatment of autosomal dominant retinitis pigmentosa (ADRP) caused by the P23H mutation. The drug specifically binds to the mRNA transcribed from the mutant gene to inhibit the formation of toxic mutant RHO protein. QR-1123 is administered primarily via intravitreal injection. It received Investigational New Drug (IND) clearance in August 2019 and was granted Fast Track designation by the FDA.

ProQR conducted Phase I/II clinical trials of QR-1123. The Aurora trial, also known as the PQ-1123-001 study, was the first-in-human clinical study of QR-1123. Conducted at expert sites in North America, the trial enrolled 35 adult patients with autosomal dominant retinitis pigmentosa (ADRP) caused by the P23H mutation. It employed single ascending dose and multiple dose regimens to characterize the drug’s pharmacokinetic profile and to evaluate its safety, tolerability, and efficacy.

ProQR, founded in 2012 and headquartered in Leiden, the Netherlands, is a biotechnology and pharmaceutical company. Established by experts in the field of genetic disorders, the company is dedicated to pioneering scientific research. ProQR leverages its proprietary RNA repair platform technology to develop RNA-based transformative therapies, providing new treatment options for patients with severe hereditary rare diseases.

Ionis Pharmaceuticals (NASDAQ: IONS), founded in 1989 and headquartered in California, USA, is a leading biotechnology company. A pioneer in the field of RNA-targeted therapeutics, the company has developed a portfolio of innovative drugs based on its highly efficient and broadly applicable drug discovery platform, providing higher-value treatments for patients with spinal muscular atrophy (SMA), motor neuron diseases, and other rare disorders.

ProQR CEO Daniel de Boer stated, “We are delighted that QR-1123, our therapeutic candidate for ADRP, has received orphan drug designation, signaling new treatment options for patients with this form of progressive blindness. Our goal is to actively advance a portfolio of development programs to deliver targeted therapies for inherited retinal diseases such as ADRP.”

(Compiled by Xu Xiaoxue)