Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of 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.
We completed an IPO onto NASDAQ in June of this year. Prior to last year’s BIO Investor Forum in San Francisco, we were still in the very early stages of contemplating an IPO onto the U.S. markets. Our decision to list in the U.S. markets was not an easy one. Given a European biotech company had not accomplished its primary listing on NASDAQ in almost a decade, it was imperative that we gain exposure with U.S.-based investors prior to embarking on the road to an IPO.
As much of the investment in the biotech industry is concentrated in the U.S., we felt that listing on NASDAQ would provide us with the necessary access to capital to be able to further develop our DMD portfolio of 6 unique compounds. BIO’s legislative efforts leading to the passage of the JOBS Act in 2012 have been very supportive for IPO’s.
We evaluated several conferences that would provide Prosensa with the opportunity to meet with a number of high quality investors. Upon receiving an invitation to present and to participate on a rare disease panel, we found the BIO Investor Forum to be an ideal venue to share our company’s unique story. The visibility and ability to meet with a number of a high quality investors in a 2 day period was an ideal foray into our interactions with U.S. investors . This conference provided us with further confidence about conducting a possible IPO on NASDAQ, and less than a year later, we are proud to be part of this new class of biotech IPOs, many of whom leveraged the BIO Investor Forum as a starting point to in their outreach to Wall Street.
Furthermore, our participation on the rare disease session was extremely timely with the newly passed Food and Drug Administration Safety and Innovation Act (FDASIA), which eventually enabled GSK, as our licensee, to receive breakthrough therapy designation on our lead investigational compound for Duchenne muscular dystrophy (DMD), drisapersen. According to a chart recently made available by the FDA, in the period between October 1, 2012 and August 23, 2013, CDER has received a total of 82 requests for Breakthrough Therapy Designation, 25 of which were granted and 32 were denied, with another 25 pending. We are honored that drisapersen has been granted this new designation by the FDA, highlighting the need for new therapies for rare genetic diseases to make it to patients as quickly as possible.
The BIO Investor Forum is one of the best venues for an emerging company to share its story in a neutral forum. Not only were some of our VC investors attending the meeting to source their next opportunity, but we also met with esteemed public investors that would eventually become some of our first shareholders as a publicly traded biotech company.
Hans Schikan has served as Prosensa’s Chief Executive Officer since January 2009. Mr. Schikan has more than 25 years of senior managerial experience in the pharmaceutical and biotechnology industries.