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Following positive top-line results from pivotal Phase 3 trial of SPK-RPE65 for genetic blinding conditions, company significantly expands and advances gene therapy pipeline
Begins 2016 with a cash position over
“This past year was one of significant growth, progress and accomplishment for Spark that has set the stage for us to dramatically expand and advance our pipeline and to submit our first Biologics Licensing Application (BLA) in 2016,” said
- Reported Positive Top-line Results from Pivotal Phase 3 Trial of SPK-RPE65 - As reported in October, the pivotal Phase 3 trial met its primary endpoint, the bilateral mobility test (MT) change score (p = .001), and the first two secondary endpoints: full-field light sensitivity threshold testing (FST) (p < .001) and first eye MT change score (p =.001). In the trial, SPK-RPE65 demonstrated a substantial restoration of functional vision, with approximately two-thirds of the subjects in the intervention group achieving the maximum improvement measurable on the mobility test, and with subjects in the intervention group improving approximately 100-fold in light sensitivity as measured by FST.
- SPK-RPE65 Continues to Demonstrate Long-lasting Benefit - At the
Retina Society48th Annual Scientific Meeting in Parisin November, principal investigator Dr. Stephen R. Russellpresented three-year MT and FST follow-up data for a cohort of subjects from an earlier Phase 1 trial. This cohort of subjects (n = 8), that would have met the Phase 3 eligibility criteria and that received the same dose of SPK-RPE65 that was administered in the Phase 3 trial, is now out at least three years, with measurements ongoing.
- Preparing for BLA Submission in 2016 - Spark is preparing to submit a BLA with the
U. S. Food and Drug Administration( FDA) in the second half of 2016. Spark is planning a rolling submission, with the non-clinical module submitted first, followed by the clinical module and then the CMC module. If approved, SPK-RPE65 would be the first gene therapy approved for a genetic disease in the U.S. As such, the company is actively pursuing pre-launch activities, including the launching of a genetic testing initiative in 2016. Further, Spark received positive written advice from the European Medicines Agency(EMA) in December, and anticipates filing a marketing authorization application (MAA) with EMA in early 2017.
- SPK-CHM Phase 1/2 Clinical Trial Dosing Complete - Spark initiated clinical testing of its second ophthalmic gene therapy product candidate, SPK-CHM for choroideremia, in early 2015 and recently completed enrollment of the second dose cohort in the dose-escalating trial. Early results from this trial are anticipated in the second half of 2016.
- Two Additional Preclinical IRD Programs Acquired - With the recent acquisition of former collaboration partner
Genable Technologies Ltd.and the in-licensing of technology targeting Leber hereditary optic neuropathy (LHON), Spark added two additional IRD preclinical programs. Genable’s RhoNova is in preclinical development for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP) and SPK- LHON is in preclinical development for Leber hereditary optic neuropathy (LHON). Both of these programs will leverage Spark’s technology and experience with SPK-RPE65 and SPK-CHM.
- SPK-FIX Program Milestone Earned - In 2015, Pfizer and Spark initiated human clinical testing of the lead SPK-FIX candidate in their hemophilia B collaboration in a dose-escalating Phase 1/2 clinical trial. In
December 2015, Spark earned a $15 millionmilestone payment under the collaboration. Initial efficacy data from this trial are anticipated in mid-2016.
Program for Hemophilia A Advancingto IND - At the American Society of Hematology(ASH) meeting in December, Spark reported that in preclinical models, the lead product candidate for hemophilia A demonstrated production of the highest levels of expression of unmodified human FVIII in the literature to date at doses that have been safely delivered to humans in hemophilia B gene therapy trials.
- First Program Targeting Neurodegenerative Diseases Named - In late 2015, Spark unveiled that its lead gene therapy program addressing neurodegenerative diseases is targeting TPP1 deficiency, a form of Batten disease also known as CLN2. In a naturally occurring, well-established preclinical model of TPP1 deficiency, administration of SPK-TPP1 to the ependymal cells of the brain ventricular system resulted in delayed onset of clinical symptoms and disease progression, protection from cognitive decline and extension of lifespan relative to untreated controls. IND-enabling studies were initiated in 2015.
Expanded Human Capitaland Strong Financial Position - During 2015 we continued to grow our team across all disciplines, with the number of employees having more than doubled to over 100. The balance sheet remains strong, with over $293 millionin cash at December 31, 2015, which does not reflect the receipt of the $15 millionmilestone payment from Pfizer that was earned in December 2015and received in January 2016.
In the year ended
Our research and development expenses for the year ended
General and administrative expenses for the year ended
Our net loss applicable to common stockholders for the year ended
Spark is a gene therapy leader seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Spark’s initial focus is on treating rare diseases where no, or only palliative, therapies exist. Spark’s most advanced product candidate, SPK-RPE65 (voretigene neparvovec), which has received both breakthrough therapy and orphan product designation, recently reported positive top-line results from a pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark’s validated gene therapy platform is being applied to a range of clinical and preclinical programs addressing serious genetic diseases, including inherited retinal dystrophies, hematologic disorders and neurodegenerative diseases. Spark builds on two decades of research, development and manufacturing at The Children’s Hospital of
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 lead product candidate, SPK-RPE65. 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 from our Phase 3 clinical trial of SPK-RPE65 may not support a label for the treatment of RPE65-mediated IRDs other than Leber congenital amaurosis (LCA); (ii) the improvements in functional vision demonstrated by SPK-RPE65 in our clinical trials may not be sustained over extended periods of time; (iii) we could experience delays in submitting our regulatory filings, including our Biologics Licensing Application with
Chief Financial Officer
(855) SPARKTX (1-855-772-7589)
Ten Bridge Communications Dan Quinn
|Spark Therapeutics, Inc.|
|Cash and cash equivalents||$||74,566,963||$||293,530,590|
|Prepaid expenses and other current assets||2,551,912||1,132,626|
|Total current assets||77,363,268||311,607,784|
|Property and equipment, net||12,674,372||16,999,445|
|Liabilities and Stockholders’ Equity|
|Current portion of deferred rent||—||715,959|
|Current portion of deferred revenue||10,014,377||5,182,835|
|Total current liabilities||15,854,228||22,115,651|
|Long-term deferred rent||8,618,489||8,084,509|
|Long-term deferred revenue||10,767,414||9,034,559|
|Series A convertible preferred stock, $0.001 par value. Authorized, issued and outstanding, 5,000,000 shares at December 31, 2014; no shares authorized, issued or outstanding as of December 31, 2015||10,000,000||—|
|Series B convertible preferred stock, $0.001 par value. Authorized, issued and outstanding, 45,186,334 shares at December 31, 2014; no shares authorized, issued or outstanding, at December 31, 2015||72,437,203||—|
|Common stock, $0.001 par value. Authorized, 150,000,000 shares; issued and outstanding, 6,290,317 shares at December 31, 2014; 27,082,493 shares issued and 27,073,287 outstanding at December 31, 2015||6,290||27,083|
|Additional paid-in capital||54,364,833||419,791,732|
|Treasury stock, at cost 9,206 shares at December 31, 2015||—||(552,636||)|
|Total stockholders’ equity||55,205,720||290,537,795|
|Total liabilities and stockholders’ equity||$||90,445,851||$||329,772,514|
|Spark Therapeutics, Inc.|
|Statements of Operations|
|December 31,||For the Year Ended December 31,|
|Research and development||4,897,152||16,351,005||46,029,314|
|Acquired in-process research and development||50,000,000||750,000||—|
|General and administrative||2,380,645||7,863,256||23,352,171|
|Total operating expenses||57,277,797||24,964,261||69,381,485|
|Loss from operations||(57,277,797||)||(24,330,329||)||(47,317,811||)|
|Preferred stock dividends||—||(707,342||)||(634,794||)|
|Net loss applicable to common stockholders||$||(57,277,797||)||$||(25,032,151||)||$||(47,760,572||)|
|Basic and diluted net loss per common share||$||(8.44||)||$||(4.64||)||$||(2.10||)|
|Weighted average basic and diluted common shares outstanding||6,788,396||5,397,599||22,710,105|