Spark Therapeutics Reports Second Quarter 2015 Results
"During the past quarter we've made great strides in advancing our clinical programs and we are reaffirming our guidance to report top-line results from our pivotal Phase 3 trial of SPK-RPE65 before the end of the year," said
Highlights from the period include:
- On track to report top-line data from our Phase 3 study before the end of the year.
Received orphan product designation in both
the United Statesand the European Unionfor the treatment of patients with a diagnosis of retinitis pigmentosa due to RPE65 mutations.
- Completed enrollment in the first dose cohort of our Phase 1/2 trial of SPK-CHM.
- Continue to demonstrate that SPK-CHM is safe, with no product-related serious adverse events noted to date.
- With Pfizer, initiated a Phase 1/2 clinical trial of SPK-9001, the lead vector in our SPK-FIX hemophilia B collaboration following clearance of the IND.
In June, presented preclinical data on SPK-9001 at the 2015
International Society on Thrombosisand Haemostasis Congress.
- Inherited Retinal Dystrophies: Initiated the first preclinical studies evaluating the delivery of Spark vectors to the suprachoroidal space by using Clearside Biomedical's microinjector technology.
- Hematologic Disorders: Nominated the lead product candidate in our hemophilia A program and are on schedule to initiate IND-enabling studies later this year.
Neurodegenerative Diseases: Selected lead indication and, based on encouraging preclinical proof-of-concept studies, intend to initiate IND-enabling studies in 2015.
- Continue to make progress building our organization across the board, with our headcount now at over 80 employees.
Strong Cash Position:
Closed the second quarter of 2015 with
$223.0 millionin cash and equivalents.
- Closed the second quarter of 2015 with
Three Months Ended
In the three months ended June 30, 2015, we recognized
Our research and development expenses for the three months ended
General and administrative expenses for the three months ended June 30, 2015 were
Our net loss applicable to common stockholders for the three months ended June 30, 2015 was
Six Months Ended
In the six months ended June 30, 2015, we recognized
Our research and development expenses for the six months ended
General and administrative expenses for the six months ended June 30, 2015 were
Our net loss applicable to common stockholders for the six months ended June 30, 2015 was
As of June 30, 2015, Spark had cash and cash equivalents of
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 orphan diseases where no, or only palliative, therapies exist. Spark's most advanced product candidate, SPK-RPE65, which has received both breakthrough therapy and orphan product designation, is in a fully enrolled pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark is leveraging the experience and technology utilized in the development of SPK-RPE65 to address a broad spectrum of blinding conditions, starting with the development of SPK-CHM for the potential treatment of choroideremia, currently in a Phase 1/2 clinical trial. Spark also is establishing a pipeline of gene therapy candidates to treat hematologic disorders and neurodegenerative diseases, including through a global collaboration with
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 financial condition and results of operations, the sufficiency of its cash, cash equivalents and marketable securities, as well as the advancement of the Company's product candidates and clinical studies and anticipated milestones for 2015. 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 results of our Phase 3 clinical trial for SPK-RPE65 may be insufficient to support further development of that product candidate; (ii) 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 LCA; (iii) the improvements in functional vision demonstrated by SPK- RPE65 in our Phase 1 clinical trials may not be sustained; (iv) our product candidate, SPK-CHM, will not demonstrate sufficient clinical data to warrant continued development; (v) that our collaboration with Pfizer will not be successful; and (vi) any one or more of our product candidates in preclincial or clinical development will not successfully be developed and commercialized. For a discussion of other risk 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 and Quarterly Reports on Form 10-Q filed with the
|Spark Therapeutics, Inc.|
|Cash and cash equivalents||$74,566,963||$223,047,436|
|Prepaid expenses and deferred financing costs||2,551,912||1,111,171|
|Total current assets||77,363,268||225,091,669|
|Property and equipment, net||12,674,372||13,490,170|
|Liabilities and Stockholders' Equity|
|Accrued expenses and other||3,163,154||3,234,730|
|Current portion of deferred rent||—||565,458|
|Current portion of deferred revenue||10,014,377||5,578,558|
|Total current liabilities||15,854,228||13,394,178|
|Long-term deferred rent||8,618,489||8,469,083|
|Long-term deferred revenue||10,767,414||11,640,137|
|Preferred stock, $0.001 par value. Authorized, 5,000,000 shares; no shares issued or outstanding||—||—|
|Series A convertible preferred stock, $0.001 par value. Authorized, 5,000,000 shares; issued and outstanding, 5,000,000 shares at December 31, 2014||10,000,000||—|
|Series B convertible preferred stock, $0.001 par value. Authorized, 45,186,334 shares; issued and outstanding, 45,186,334 shares at December 31, 2014||72,437,203||—|
|Common stock, $0.001 par value. Authorized, 150,000,000 shares; issued and outstanding, 6,290,317 and 24,543,923 shares at December 31, 2014 and June 30, 2015, respectively.||6,290||24,544|
|Additional paid-in capital||54,364,833||311,135,063|
|Total stockholders' equity||55,205,720||205,486,652|
|Total liabilities and stockholders' equity||$90,445,851||$238,990,050|
|Spark Therapeutics, Inc.|
|Statements of Operations|
|Three months ended June 30,||Six months ended June 30,|
|Research and development||2,129,573||9,343,972||5,517,306||17,678,080|
|General and administrative||2,038,909||6,333,123||3,055,032||10,018,003|
|Total operating expenses||4,168,482||15,677,095||8,572,338||27,696,083|
|Loss from operations||(4,168,482)||(14,388,466)||(8,552,338)||(24,132,987)|
|Preferred stock dividends||—||—||—||(634,794)|
|Net loss applicable to common stockholders||$(4,168,384)||$(14,336,842)||$(8,552,240)||$(24,705,143)|
|Basic and diluted net loss per common share||$(0.79)||$(0.60)||$(1.65)||$(1.17)|
|Weighted average basic and diluted common shares outstanding||5,246,089||24,080,420||5,172,231||21,031,708|
CONTACT: Investor Relations
Spark Therapeutics, Inc. Stephen W. WebsterChief Financial Officer (888) 772-7560 or Media Contact Ten Bridge Communications Dan Quinn(781) 475-7974