Spark Therapeutics, a gene therapy developer founded last year to commercialize technologies initially developed at Children’s Hospital of Philadelphia (CHOP), today said it plans to raise up to $86.25 million through an initial public offering.
CHOP owns more than half (53.44%) of the company’s stock, nearly 43.9 million shares. Spark’s board of directors is chaired by CHOP CEO Steven Altschuler, M.D., who “may be deemed to have voting and investment power over the shares held by CHOP” although he disclaims ownership over such shares, the company stated in a Form S-1 Registration Statement filed yesterday with the U.S. Securities and Exchange Commission.
According to the Form S-1, Spark said both the number of shares to be offered, and the price range for the offering, had yet to be decided. The company added that it will trade its shares under the symbol “ONCE” on The NASDAQ Global Market.
Spark is a developer of one-time treatments for genetic diseases, with an initial focus on treating rare diseases. The company’s most advanced product candidate, SPK-RPE65, is in a Phase III trial assessing its ability to treat inherited retinal dystrophies caused by non-sex-linked, or autosomal recessive, mutations in the RPE65 gene.
The trial is fully enrolled, Spark disclosed, with data is expected in the second half of 2015 on SPK-RPE65, which has received both breakthrough therapy and orphan product designations from the FDA. If the trial is successful, Spark plans to submit a biologics license application (BLA) to the FDA in 2016.
“SPK-RPE65 has the potential to be the first gene therapy approved in the United States for the treatment of a genetic disease and the first approved pharmacologic treatment for any IRD,” the company stated.
SPK-RPE65 is engineered via a vector derived from an adeno-associated virus (AAV). Encoding DNA from the viral genes is removed and replaced with the therapeutic gene sequence for the RPE65 protein, delivered via injection to the retina. Production of RPE65 protein in the retina helps convert light into an electrical signal needed for vision.
“Following a single injection of SPK-RPE65 in one eye, the children from our initial Phase 1 trial no longer depended on visual aids to carry out classroom activities and were able to walk and play more like normally sighted kids,” Spark stated. “Furthermore, inclusive of the subjects in our ongoing Phase 3 clinical trial, we have not observed any drug-related serious adverse events to date.”
The company said it expects to launch a clinical trial in the first half of 2015 for its second product candidate, SPK-CHM, targeting choroideremia, the company said in its filing. Spark’s pipeline also includes product candidates in development for treating additional blinding conditions, blood disorders, and neurodegenerative diseases.
Spark’s activities also include a collaboration with Pfizer announced earlier this month to develop and commercialize SPK-FIX, a gene therapy for treating hemophilia B.
According to the S-1, Spark lost $57.3 million from its inception March 13, 2013, through Dec. 31, 2013, and lost $15.3 million during the first nine months of 2014.
J.P. Morgan Securities and Credit Suisse Securities (USA) will act as lead book-running managers for the IPO, with Cowen and Company acting as lead manager and Sanford C. Bernstein & Co., as co-manager.