Spark Therapeutics Announces Presentation of Preclinical Data in Pompe Disease and CLN2 Disease at 15th Annual WORLDSymposium™

PHILADELPHIA, Jan. 31, 2019 (GLOBE NEWSWIRE) — Spark Therapeutics (NASDAQ:ONCE), a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, announced today the presentation of preclinical data for SPK-3006 for Pompe disease and SPK-1001 for CLN2 disease, a form of Batten disease, on Wednesday, Feb. 6, 2019, at the 15th Annual WORLDSymposium™ in Orlando, FL.

  • Giuseppe Ronzitti, Ph.D., team leader at Genethon and researcher at the French National Institute of Health and Medical Research (INSERM), will present preclinical data for SPK-3006, an investigational liver-directed adeno-associated viral (AAV) gene therapy for Pompe disease, at 8:45am ET. During his presentation on the “Safety and efficacy evaluation of investigational liver gene transfer for secretable GAA in the treatment of Pompe disease,” Dr. Ronzitti will share previously disclosed data from investigational new drug (IND)-enabling studies that support an IND filing by Spark Therapeutics expected 2Q 2019.
  • David Anderson, Ph.D., CNS research lead at Spark Therapeutics, will present a poster, entitled “Safety and tolerability evaluation in non-human primates of ependymal transduction with an AAV2-CAG-hTpp1 vector for the treatment of late infantile neuronal ceroid lipofuscinosis,” on preclinical data for SPK-1001, an investigational AAV gene therapy for CLN2 disease, a form of Batten disease, scheduled between 4:30-6:30pm ET.

About SPK-3006 for Pompe disease
SPK-3006 is an investigational liver-directed AAV gene therapy for Pompe disease. SPK-3006 has been engineered to produce a modified enzyme that is secreted from the liver, which may sustain GAA plasma levels and lower immunogenicity to GAA to potentially provide greater uptake in muscle tissue. The transgene was in-licensed in 2017 from Genethon, a non-profit research and development organization dedicated to the development of gene therapies for orphan genetic diseases from research to clinical validation. Spark Therapeutics retains global commercialization rights to SPK-3006.

About SPK-1001 for CLN2 disease, a form of Batten disease
SPK-1001 is an investigational central nervous system (CNS)-directed AAV gene therapy that has demonstrated compelling preclinical proof-of-concept in a naturally occurring model of TPP1 deficiency, a form of Batten disease. Batten disease is a fatal neurological disorder involving mutations of the TPP1 gene that begins in early childhood. Spark Therapeutics has received orphan drug designation from the U.S. Food and Drug Administration for SPK-1001 for the treatment of CNL2 disease [neuronal ceroid lipofuscinosis (NCL)] caused by TPP1 deficiency. Spark Therapeutics retains global rights to SPK-1001.

About Spark Therapeutics
At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We have successfully applied our technology in the first gene therapy approved in both the U.S. and EU for a genetic disease, and currently have four programs in clinical trials, including product candidates that have shown promising early results in patients with hemophilia. At Spark, we see the path to a world where no life is limited by genetic disease. For more information, visit, and follow us on Twitter and LinkedIn.

Spark Therapeutics Cautionary note on forward-looking statements
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the company’s SPK-3006 and SPK-1001 programs. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘predict,’’ ‘‘will,’’ ‘‘would,’’ ‘‘could,’’ ‘‘should,’’ ‘‘continue’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that: (i) the data for SPK-3006 IND-enabling studies may not be sustained; (ii) we may not submit an IND application or Clinical Trial Application or initiate a U.S. and EU Phase 1/2 clinical trial in adult patients when expected or at all; and (iii) the data from our safety and tolerability evaluation in non-human primates for SPK-1001 may not be sustained. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in our Annual Report on Form 10-K, our Quarterly Reports on Form 10-Q and other filings we make with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of the release, and Spark undertakes no duty to update this information unless required by law.

Investor Contact:    Media Contact:
Ryan Asay  Monique da Silva 
(215) 239-6424  (215) 282-7470
Spark Therapeutics, Inc.
Consolidated balance sheets (unaudited)
(in thousands, except share and per share data)
  December 31,
  December 31,
Current assets:      
Cash and cash equivalents $ 96,748     $ 95,247  
Marketable securities 423,419     358,359  
Trade and other receivables 7,906     47,385  
Inventory     25,637  
Prepaid expenses and other current assets 5,093     40,512  
     Total current assets 533,166     567,140  
Restricted cash     53,000  
Marketable securities 20,035     94,678  
Property and equipment, net 61,713     95,998  
Goodwill 1,254     1,198  
Other assets 628     2,338  
     Total assets $ 616,796     $ 814,352  
Liabilities and Stockholders’ Equity      
Current liabilities:      
Accounts payable $ 14,183     $ 19,492  
Accrued expenses 24,697     34,790  
Current portion of long-term debt 312     4,822  
Current portion of deferred rent 969     1,365  
Current portion of deferred revenue 11,969      
Current other liabilities 1,557     1,885  
     Total current liabilities 53,687     62,354  
Long-term debt 912     46,090  
Long-term deferred rent 8,318     10,885  
Long-term deferred revenue     160,000  
Other liabilities 40,255     38,510  
     Total liabilities 103,172     317,839  
Stockholders’ equity:      
Preferred stock, $0.001 par value. Authorized, 5,000,000 shares; no shares issued or outstanding      
Common stock, $0.001 par value. Authorized, 150,000,000 shares; 37,131,626 shares issued and 37,111,404 shares outstanding as of December 31, 2017; 37,764,213 shares issued and 37,686,301 shares outstanding as of December 31, 2018 37     38  
Additional paid-in capital 1,026,590     1,091,873  
Accumulated other comprehensive loss (5,914 )   (1,050 )
Treasury stock, at cost, 20,222 shares as of December 31, 2017 and 77,912 shares as of December 31, 2018 (1,226 )   (4,661 )
Accumulated deficit (505,863 )   (589,687 )
     Total stockholders’ equity 513,624     496,513  
     Total liabilities and stockholders’ equity $ 616,796     $ 814,352