ProQR plans to independently advance its pipeline of RNA medicines to establish a multi-product, platform company in IRD. By 2023, the company expects its pipeline to have at least two commercial products, and at least three late-stage and seven early-stage programs in development. In parallel, the company will continue to expand its RNA platform capabilities in other therapeutic areas, and plans to selectively partner programs in non-core therapeutic areas and non-rare diseases.
“The ‘ProQR Vision 2023’ strategy presents a clear path for ProQR to become a fully-integrated company that is independently developing and commercializing innovative RNA medicines for patients who suffer from IRDs. Given the high unmet need across many of the 300 IRDs known today, we believe this integrated approach will allow us to more efficiently turn our scientific innovation into multiple potentially life-changing medicines for patients,” said
“ProQR Vision 2023” strategy and key goals
The “ProQR Vision 2023” strategy provides a clear path forward to develop the company’s platform of RNA medicines for IRD patients in need. The programs in ProQR’s pipeline utilize the RNA oligonucleotide technology platform that repairs the genetic defect in the RNA to address the underlying cause of genetic diseases. The product candidates in ProQR’s pipeline target diseases with a well-understood genetic cause where rational drug design can be applied to yield RNA molecules with therapeutic potential. One notable differentiator for ProQR’s therapeutic candidates for IRDs is that they are designed for intravitreal delivery. This design is intended to promote rapid delivery of the molecules to the target cells across the entire retina, which we believe is an advantage in the development of medicines for retinal disease.
The key deliverables for “ProQR Vision 2023” include:
- Sepofarsen (formerly QR-110) for Leber's congenital amaurosis 10 (LCA10): Complete pivotal program around year-end 2020 for submission of a New Drug Application (NDA) in the U.S. and a Marketing Authorization Application (MAA) in
- QR-421a for Usher syndrome Exon 13: Start the Phase 1/2 STELLAR proof-of-concept clinical trial, with interim data in mid-2019 and initiate an adaptive multiple-dose trial with projected readout in 2021
- QR-1123 for P23H autosomal dominant retinitis pigmentosa (adRP): Initiate a Phase 1/2 proof-of-concept clinical trial in 2019, with data expected in 2020
- QR-411a for Usher syndrome PE40: Conduct Investigational New Drug (IND)-enabling work in 2019 to start a proof-of-concept clinical trial in 2020
- QR-504 for Fuchs endothelial corneal dystrophy (FECD): Complete IND-enabling activities in 2019 to start a proof-of-concept clinical trial in 2020
- Accelerate discovery: Expand efforts to generate additional programs for inherited retinal diseases amenable to RNA oligo treatment with the goal of establishing at least seven new programs for development
- Selectively explore and expand platform: Expand platform to develop medicines in new therapeutic areas and create opportunities to build businesses in other therapeutic areas
Key 2019 business goals
In pursuit of the “ProQR 2023 Vision” strategy, ProQR will focus on critical success factors in 2019:
- Clinical trial execution: Expand our clinical trial operational infrastructure to support enrollment of more than four clinical programs, including the execution of a pivotal Phase 2/3 trial in LCA10, while also preparing the next wave of ophthalmology drugs through IND-enabling activities and advancing to the clinic
- Start building commercial capabilities: Build a commercial infrastructure in preparation for a potential commercial launch of sepofarsen in 2021 and QR-421a in
North Americaand Europein 2023, if these candidates are approved
- Expand pipeline for inherited blindness: Scale up discovery and lead development efforts to expand the IRD pipeline into other forms of genetic blindness
- Validate and expand Axiomer®: Further validate and expand ProQR's proprietary Axiomer® RNA editing platform through first development candidate selection and potential additional alliances
- Selectively partner non-core programs: Explore alliances based on the technology platform and pipeline programs outside of IRDs
- Financial discipline: Maintain strong financial discipline by operating the business in a capital efficient way
“Our ‘ProQR Vision 2023’ strategy presents a long-term plan that allows us to focus on our most promising assets and leverage this progress to expand our business in order to create the most value for patients and other stakeholders, including investors,” said
ProQR R&D day details
ProQR is holding an R&D day today in New York. The event will be webcast live and is accessible from the Investor Relations section of ProQR’s website (www.proqr.com) under Events and Presentations. An archived recording of the event will be available via webcast for 90 days following the presentation date.
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such statements include those relating to our ProQR Vision 2023 strategy and our key 2019 goals, the development and therapeutic potential of our product candidates, including sepofarsen, QR-1123, QR-421a, QR-411a, QR-504, the potential of our Axiomer® editing platform, our plans and timing of initiating and obtaining results from our ongoing and planned clinical trials, our plans for building commercial infrastructure to support the launch of our product candidates, if approved, our plans and timing of submitting applications for and receiving marketing approval of our product candidates, our expectations for our platform and discovery of new product candidates, and our plans for strategic collaborations and alliances for our programs. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the
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Source: ProQR Therapeutics N.V.