LEIDEN, the Netherlands, Nov. 24, 2014 – ProQR Therapeutics N.V. (Nasdaq:PRQR), an innovative biopharmaceutical company engaged in the discovery and development of RNA-based therapeutics for the treatment of severe genetic disorders, today announced results for the third quarter of 2014.
“ProQR has made significant accomplishments, both financially and operationally, in 2014,” said Daniel de Boer, Chief Executive Officer of ProQR. “With the successful completion of our recent initial public offering, we have strengthened our balance sheet, which will allow us to advance the clinical development of QR-010 for the treatment of patients with cystic fibrosis and continue to build our platform and pipeline of proprietary programs.”
Net cash generated by financing activities during the three and nine months ended September 30, 2014 was €80.2 million and €119.7 million, respectively. This includes the net proceeds from our IPO in the third quarter of 2014 and the net proceeds from the private placement in the second quarter, as well as proceeds from borrowings, representing receipts from our Innovation credit grant. As of September 30, 2014, we held cash and cash equivalents of €115.3 million. Net cash used in operating activities during the three and nine months ended September 30, 2014 was €4.3 million and €9.5 million, respectively.
Research and development costs were €2.5 million for the three months ended September 30, 2014, compared to €0.7 million for the same period in 2013. Research and development costs for the nine months ended September 30, 2014 were €7.0 million compared to €1.3 million for the same period in 2013. The increase reflects the expansion of research and development activities to support the further development of our lead product candidate, QR-010, for the treatment of cystic fibrosis, as well as the development of our other pipeline product candidates, including QR-110 for the treatment of Leber’s Congenital Amaurosis, the leading genetic cause of childhood blindness.
General and administrative costs were €2.4 million for the three months ended September 30, 2014, compared to €0.2 million for the same period in 2013. General and administrative costs for the nine months ended September 30, 2014 were €4.5 million compared to €0.4 million for the same period in 2013. The increase reflects the preparatory costs associated with the Company’s IPO on the Nasdaq Global market, a grant to ProQR’s protection foundation, and increased costs reflecting the Company’s growth.
Net loss for the three months ended September 30, 2014 was €3.4 million or €0.43 per share, compared to €0.9 million or €0.14 per share for the same period in 2013. Net loss for the nine months ended September 30, 2014 was €10.1 million or €1.45 per share, compared to €1.6 million or €0.30 per share for the same period in 2013. For further financial information for the period ending September 30, 2014, please refer to the financial statements appearing at the end of this release.
• Entered into an agreement with Cystic Fibrosis Foundation Therapeutics, Inc. (“CFFT”), a subsidiary of the Cystic Fibrosis Foundation, in August 2014, pursuant to which CFFT agreed to provide us with up to $3 million to support the clinical development of QR-010. The Cystic Fibrosis Foundation is a professional patient advocacy organization that supports select CF drug development programs by providing financial support and access to its Therapeutic Development Network, a network of CF key opinion leaders, as well as to an extensive clinical trial network infrastructure.
• Successfully completed our initial public offering on NASDAQ in September 2014, placing 8,625,000 ordinary shares at a public offering price of $13.00 per share, which includes the exercise in full by the underwriters of their option to purchase 1,125,000 additional ordinary shares, resulting in aggregate net proceeds, after underwriting discounts and commissions and other estimated offering expenses, of approximately $102 million (€79.3 million).
• Appointed Alison Lawton as a member of our Supervisory Board of Directors in September 2014. Ms. Lawton brings significant operational, international, regulatory and senior management experience within the pharmaceutical and biotechnology industries. She currently serves as a board member of Cubist Pharmaceuticals, Inc. (Nasdaq:CBST) and Verastem, Inc. (Nasdaq:VSTM) and previously served as Chief Operating Officer of OvaScience, Inc. (Nasdaq:OVAS).
• Appointed Smital Shah as our Chief Financial Officer in October 2014. Ms. Shah has a track record of management and leadership experience in the biopharmaceutical industry as well as in investment banking, with a particular focus on financial strategy and capital markets.
• Entered into an agreement with PARI Pharma GmbH in October 2014, pursuant to which the Company is granted exclusive access to the use of the PARI e-flow nebulizer for the administration of nucleotide-based drugs in ∆F508 mutation in cystic fibrosis, with the exclusive option to expand this exclusivity to the use in other CF mutations. Pursuant to the terms of the agreement, we have made an upfront payment, hourly fees for development work and are obligated to make sales-based royalty payments after market authorization.
ProQR is an innovative biopharmaceutical company engaged in the discovery and development of RNA-based therapeutics for the treatment of severe genetic disorders, with an initial focus on Cystic Fibrosis. Utilizing our unique, proprietary RNA repair technologies, we believe we will be able to treat genetic disorders in which a single protein is defective due to certain types of genetic mutation. We believe that this is a unique approach that offers advantages compared with small molecule, gene therapy and other therapeutic strategies.
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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 the initiation of clinical trials of QR-010 and statements regarding our ongoing and planned discovery and development of product candidates. 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 described under the heading “Risk Factors” in ProQR’s prospectus filed with the Securities and Exchange Commission dated September 19, 2014. 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.
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Source: ProQR Therapeutics N.V.