UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 6-K
Report of Foreign Private Issuer
Pursuant to Rule 13a-16 or 15d-16 of
the Securities Exchange Act of 1934
June 1, 2016
PROQR THERAPEUTICS N.V.
Darwinweg 24
2333 CR Leiden
The Netherlands
Tel: +31 88 166 7000
(Address, Including ZIP Code, and Telephone Number,
Including Area Code, of Registrant’s Principal Executive Offices)
Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.
Form 20-F x Form 40-F ¨
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ¨
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ¨
On June 1, 2016, ProQR Therapeutics N.V. issued two press releases titled, “ProQR’s Drug Candidate QR-110 for Leber’s Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation” and “ProQR Announces Presentations at the European CF Conference and the JMP Life Science Conference.” Copies of these press releases are attached hereto as Exhibit 99.1 and Exhibit 99.2 and are incorporated herein by reference.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| PROQR THERAPEUTICS N.V. | ||||||
| Date: June 1, 2016 | By: | /s/ Smital Shah | ||||
| Smital Shah | ||||||
| Chief Financial Officer | ||||||
INDEX TO EXHIBITS
| Number |
Description | |
| 99.1 | Press Release of ProQR Therapuetics N.V. dated June 1, 2016, titled “ProQR’s Drug Candidate QR-110 for Leber’s Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation.” | |
| 99.2 | Press Release of ProQR Therapeutics N.V. dated June 1, 2016, titled “ProQR Announces Presentations at the European CF Conference and the JMP Life Science Conference.” | |
Exhibit 99.1
| ProQR Therapeutics N.V. Press Release June 1, 2016 |
|
FINAL – FOR RELEASE
ProQR’s Drug Candidate QR-110 for Leber’s Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation
LEIDEN, the Netherlands, June 1, 2016 — ProQR Therapeutics N.V. (Nasdaq: PRQR) today announced that the company’s investigational drug QR-110 has received orphan drug designation (ODD) from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of Leber’s congenital amaurosis Type 10 (LCA10), the most common cause of genetic blindness in childhood.
ODD in the U.S. and the European Union confers a special status for investigational drugs that are being developed for rare diseases. We expect that development of QR-110 will benefit from tax credits for certain research and a waiver of the NDA application user fee, as well as market exclusivity for seven years in the US for the product and its indication, and ten years in the European Union after market approval.
“LCA10 is a severe genetic eye disease that causes children to go blind in the first few years of life and there is currently no approved therapy available for these patients. We are working on a novel and elegant approach to make a meaningful impact on the lives of patients suffering from LCA10 due to the p.Cys998X mutation”, said Daniel de Boer, Chief Executive Officer of ProQR. “Obtaining orphan drug designations from EMA and FDA for QR-110 highlights the high unmet need in for this disease and are important milestones for our program.”
About LCA10
Leber’s congenital amaurosis is the most common cause of blindness in children and consists of a group of diseases of which LCA Type 10 (LCA10) is one of the more severe forms. LCA10 leads to progressive loss of vision causing most patients to lose their sight in the first few years of life. To date, there are no treatments approved or products in clinical development that treat the underlying cause of the disease. LCA10 is an autosomal recessive disease caused by mutations in the CEP290 gene. The p.Cys998X mutation, the leading cause of LCA10, is a point mutation in the CEP290 gene resulting in an activation of a cryptic splice site. Although prevalence rates vary, we believe approximately 2,000 people in the Western world have LCA10because of this mutation. The mutated gene and the resulting defective protein lead to dysfunction of the light perceiving cells in the retina (rods and cones), eventually causing blindness.
About QR-110
QR-110 is a first-in-class oligonucleotide, designed to address the underlying cause of LCA10 due to the p.Cys998X mutation. QR-110 is designed to restore wild-type, or normal CEP290 mRNA by binding the pre-mRNA and thereby restoring normal splicing of the pre-mRNA leading to the expression of wild-type CEP 290 protein. QR-110 is designed to be administered through intravitreal injections in the eye.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe orphan diseases such as cystic fibrosis and Leber’s congenital amaurosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Since 2012.
ProQR Therapeutics N.V. | Darwinweg 24, 2333 CR Leiden, The Netherlands | +31 88 166 7000 | info@proqr.com | www.proqr.com
FORWARD-LOOKING STATEMENTS
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. 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. These forward-looking statements include, but are not limited to, statements regarding QR-110, including its clinical development and therapeutic potential, and the orphan drug designations for QR-110 for the treatment of LCA10. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.
ProQR Therapeutics N.V.:
Sariette Witte
Head of Communications
T: +1 213 261 8891
ir@proqr.com
Exhibit 99.2
| ProQR Therapeutics N.V. Press Release June 1, 2016 |
|
FINAL – FOR RELEASE
ProQR Announces Presentations at the European CF Conference and the JMP Life Science Conference
LEIDEN, the Netherlands, June 1, 2016 — ProQR Therapeutics N.V. (NASDAQ: PRQR) today announced that the company will present pre-clinical data for QR-010 at the 39th European Cystic Fibrosis Conference (ECFS) held on June 8 – 11, 2016 in Basel, Switzerland and that Smital Shah, Chief Financial Officer, will present during the JMP Securities Life Science Conference held on June 21 - 22, 2016 in New York, NY.
39th European Cystic Fibrosis Conference (ECFC)
QR-010, the company’s lead molecule, is currently being studied in two active clinical trials in patients with cystic fibrosis associated with the DF508 mutation in the CFTR gene. Pre-clinical data on the delivery of QR-010 will be presented in an oral presentation entitled: ‘QR-010 penetrates the CF-like mucus barrier in vitro and in vivo’. The presentation is part of workshop 18. ‘CFTR: Functional tests for therapeutic interventions’ on June 10, 2016 from 3pm - 4:30pm CET.
2016 JMP Securities Life Science Conference
On June 22, 2016 at 4pm ET Smital Shah, Chief Financial Officer, will take part in an analyst led fireside chat during the conference.
The live and archived webcast of the fireside chat will be accessible from the Investor Relations section of ProQR’s website (www.proqr.com) under Events and Presentations. The archived webcast will be available for 30 days following the presentation date.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe orphan diseases such as cystic fibrosis and Leber’s congenital amaurosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Since 2012.
About QR-010
QR-010 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by repairing the mRNA defect encoded by the DF508 mutation in the CFTR gene of CF patients. The DF508 mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA and guide the insertion of the three missing nucleotides, thus repairing the mRNA and subsequently producing wild-type, or normal CFTR protein. QR-010 is designed to be self-administered through a small, handheld aerosol delivery device, or nebulizer, in the form of a mist inhaled into the lungs. We believe this method could allow maximum exposure of QR-010 to the primary target organ, the lung, as well as significant exposure to other affected organs through systemic absorption into the blood. QR-010 has been granted orphan drug designation in the United States and the European Union. The QR-010 project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.
ProQR Therapeutics N.V. | Darwinweg 24, 2333 CR Leiden, The Netherlands | +31 88 166 7000 | info@proqr.com | www.proqr.com
FORWARD-LOOKING STATEMENTS
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. 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. These forward-looking statements include, but are not limited to, statements about QR-010 and its clinical development and therapeutic potential, and the ECFS and JMP conferences. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.
ProQR Therapeutics N.V.:
Sariette Witte
Head of Communications
T: +1 213 261 8891
ir@proqr.com