Recent Corporate Highlights
- Initial Public Offering Prosensa announced the pricing of its IPO at $13 on June 27, 2013 and the stock began trading the following day on the NASDAQ Global Market under the ticker symbol "RNA". The IPO of 6.9 million of the company's ordinary shares closed on July 3, and raised gross proceeds of $89.7 million.
- Continued Progress with Drisapersen Development
- On April 11, 2013 GSK presented results for the DMD114117 (DEMAND II) clinical trial. The primary endpoint in this double-blind, placebo-controlled, exploratory study was met and a statistically and clinically meaningful difference of 35 meters in the six-minute walk test between the continuous dosing regimen of 6mg/kg per week and placebo was achieved at 24 weeks. On July 31, 2013, summary results from this study were posted on GSK's Clinical Study Register.
- On June 27, 2013, GSK announced that the U.S. Food and Drug Administration, or FDA, had provided notification that drisapersen has been granted Breakthrough Therapy Designation for the treatment of DMD. Breakthrough Therapy Designation is an FDA program intended to facilitate and expedite development and review of new drugs to address unmet medical need in the treatment of serious or life-threatening conditions.
- FP7 Grant Awarded for Development of PRO045 On August 1, 2013, Prosensa announced that it is part of a high quality, pan-European consortium which has been awarded a prestigious Framework Programme 7 (FP7) research grant of EUR 6 million from the European Commission to support the ongoing clinical study of Prosensa's third novel DMD development candidate, PRO045. The project is expected to run for three years.
- Supervisory Board Changes David Mott, General Partner of New Enterprise Associates, was appointed Chairman of the Supervisory Board upon completion of the IPO. Mr. Mott joined the Supervisory Board in 2012 and brings over 25 years of experience in the biotech industry to Prosensa.
- Cash Position and Cash Consumption Our cash and cash equivalents as of June 30, 2013 were €30.8 million, compared to €40.7 million as of December 31, 2012. Our cash consumption, excluding cash flows from financing in the first half of 2013 was €10.5 million. The IPO proceeds, received on July 3, 2013, after the deduction of underwriting discounts, amount to €64.0 million ($83.4 million).
- Revenue & Deferred Revenue Revenue was €2.0 million for the second quarter 2013 and €4.4 million for the six months ended June 30, 2013, compared to €1.2 million and €3.2 million for the comparable periods in 2012 due to increased license income of €0.5 million and collaboration revenue of €0.3 million for the second quarter 2013 and increased license income of €0.5 million and collaboration revenue of €0.7 million for the six months ended June 30, 2013. All of our license revenue and collaboration revenue were generated under the GSK Agreement.
- R&D Expense Research and development expense was €4.5 million in the second quarter 2013 and €8.6 million for the six months ended June 30, 2013, compared to €2.7 million and €4.5 million for the comparable periods in 2012. Research and development expense mainly increased for the second quarter 2013 as well as for the six months ended June 30, 2013 due to the commencement of the phase I/II study of PRO045, the preparations for the initiation of the phase I/II study of PRO053 and higher intellectual property expenses.
- G&A Expense General and administrative expense was €2.1 million in the second quarter 2013 and €3.9 million for the six months ended June 30, 2013, compared to €0.9 million and €2.0 million for the comparable periods in 2012. The increase is primarily due to expenses related to the IPO for an amount of €0.8 million recorded in the second quarter 2013 and €1.5 million for the six months ended June 30, 2013.
- Net Loss Net loss for the second quarter 2013 was €4.7 million in the second quarter 2013 and €8.2 million for the six months ended June 30, 2013, compared to €2.4 million and €5.1 million for the comparable periods in 2012.
Near-Term News Flow
- Drisapersen In the fourth quarter 2013, GSK aims to make available and present dystrophin results from the DMD114117 (DEMAND II) study as well as results from the DMD114044 (DEMAND III) and DMD114876 (DEMAND V) studies at forthcoming scientific meetings.
- PRO053 In the third quarter 2013, Prosensa anticipates the first patient to be dosed with PRO053 in the phase I/II safety and dose finding study which is currently in the recruitment screening phase.
- PRO044 In the fourth quarter 2013, results from the phase I/II study will be presented at forthcoming scientific meetings.
Conference Call / Webcast InformationProsensa will host a conference call on Wednesday, August 28, 2013 at 8:00am ET, 2:00pm CET to discuss second quarter 2013 financial results. In order to participate in the conference call, please dial 1-877-249-9037 (US domestic) and refer to conference ID 6727193. International dial-in numbers and an audio webcast can be accessed under "Events & Presentations" through the Investors & Media section of the Prosensa corporate website www.prosensa.com . About Prosensa Holding N.V. Prosensa (Nasdaq:RNA) is an innovative biotechnology company engaged in the discovery and development of ribonucleic acid-modulating, or RNA-modulating, therapeutics for the treatment of genetic disorders. Our primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntington's disease. Our clinical portfolio of RNA-based product candidates is focused on the treatment of Duchenne muscular dystrophy, or DMD. Each of our DMD compounds has been granted orphan drug status in the United States and the European Union. Our first product candidate, drisapersen, can address a variety of mutations in the dystrophin gene, such as a deletion of exon 50 or exons 48 to 50.