Spark Therapeutics, Inc., (ONCE, NASDAQ), Philadelphia, PA, a retinal gene therapy company, made an impressive IPO debut on the NASDAQ, Friday, January 30th. The company gained a substantial 117% jump in price, from an original offer of $23 per share to close out their first day of trading at $50.65. The company raised $161M from selling all offer stock at the $23/share price valuing the company at approximately $1.5 billion by the end of the day.
Spark is focused on the treatment of orphan diseases with a lead therapy in Phase 3 clinical trials for the treatment of RPE65-mediated inherited retinal degeneration. The company anticipates reporting final results from the clinical study in the second half of 2015. Spark’s lead therapy, an AAV delivered RPE65 gene, currently referenced as “SPK-RPE65”, could be ready for submission of a biological license application (BLA) at the FDA in early 2016 with a potential product launch between Q2 and Q4 of the same year. If successful, the product could be the first gene therapy product approved in the USA. A follow-on product, “SPK-CHM” aims to provide a treatment for choroideremia, an X-linked inherited retinal degeneration thought to afflict approximately 12,500 males in the US and five of the major European markets. A dose-escalating Phase 1/2 clinical trial of SPK-CHM, enrolling up to 10 patients, is expected to initiate dosing before the end of Q1FY2015. Spark is additionally in a global collaboration agreement with Pfizer for the development and commercialisation of a gene therapy treatment of haemophilia B. Prior to the company’s IPO, Spark and Pfizer signed an agreement in which Spark received a $20M upfront payment with a potential $260M to follow, subject to various milestones including a royalty on product sales.
Spark will aim to use the proceeds of their IPO to complete the clinical development of SPK-RPE65 and launch a product on the market. In addition, the company’s core strategy is to establish a global franchise of gene therapies for other retinal degenerative disorders in order to ensure it becomes the partner of choice for new and emerging gene technologies in the ophthalmic space. Spark also has designs, via its Pfizer collaboration, on the haemophilia B market and on several as yet to be disclosed neurodegenerative programmes. Originally formed as AAVenue Therapeutics LLC in March 2013, Spark has made enormous advances since launch, not least of due to the strategic acquisition of rights to significant IP and know-how from the Children’s Hospital of Philadelphia, the University of Iowa and the University of Pennsylvania.