Ophthotech Reports Second Quarter 2018 Financial and Operating Results
(Conference Call and Webcast Today,
“During the first half of the year, we continued implementing our
strategy to broaden and advance our ophthalmic portfolio as we enter the
emerging field of gene therapy by securing collaborations with three
leading academic institutions, and continued advancing our therapeutic
portfolio with Zimura®,” stated
First Half 2018: Key Highlights
Zimura® Complement Factor C5 Inhibitor Program
April 2018, the Company completed patient recruitment in its randomized, dose-ranging, open-label, uncontrolled, multi-center Phase 2a clinical trial of Zimura (avacincaptad pegol) in combination with the anti-vascular endothelial growth factor (anti-VEGF) agent Lucentis® (ranibizumab) in patients with wet age-related macular degeneration (AMD) who have not been previously treated with anti-VEGF therapies. This trial is designed to assess the safety of Zimura combination therapy at different dosages and to detect a potential efficacy signal. Data will be evaluated at month six and initial top-line data is expected to be available by the end of 2018.
- Patient recruitment for the Company’s ongoing randomized, double-masked, sham controlled, multi-center Phase 2b clinical trial of Zimura for the treatment of geographic atrophy secondary to dry AMD is on track. The Company expects to complete recruitment in the third quarter of this year with initial top-line data expected to be available during the second half of 2019.
January 2018, the Company started enrolling patients in a Phase 2b randomized, double-masked, sham-controlled, multi-center clinical trial assessing the efficacy and safety of Zimura in patients with autosomal recessive Stargardt disease (STGD1). Initial top-line data is expected to be available in 2020.
Gene Therapy Programs
The Company has initiated an innovative gene therapy program focused
on applying novel gene therapy technology to discover and develop new
therapies for ocular diseases.
June 2018, the Company entered into an exclusive global license agreement with the University of Florida Research Foundation, Incorporatedand the Trustees of the University of Pennsylvania( Penn) for rights to develop and commercialize a novel adeno-associated virus gene therapy product candidate for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), an orphan monogenic disease. The construct for the RHO-adRP product candidate combines a transgene expressing a highly efficient novel short hairpin RNA (shRNA) designed to target and knock-down endogenous rhodopsin (RHO) in a mutation-independent manner with a human RHO replacement transgene made resistant to RNA interference, in a single adeno-associated viral (AAV 2/5) vector. Ophthotechand Pennhave also entered into a master sponsored research agreement, facilitated by the Penn Center for Innovation, pursuant to which Ophthotechand Penn plan to conduct natural history studies in RHO-adRP patients and additional preclinical studies. In parallel with the sponsored research, Ophthotechplans to commence IND-enabling activities. Based on current timelines and subject to regulatory review, Ophthotechexpects to initiate a Phase 1/2 clinical trial in RHO-adRP in 2020.
February 2018, the Company entered into a series of sponsored research agreements with the University of Massachusetts Medical School(UMMS) and its Horae Gene Therapy Centerto utilize their next generation “minigene” therapy approach for the potential treatment of orphan degenerative retinal diseases such as Leber Congenital Amaurosis (LCA) type 10 due to CEP290 mutations (the most common type of LCA), and autosomal recessive Stargardt disease (STGD1) due to ABCA4 mutations. Further, the Company and UMMS are also evaluating novel gene delivery methods to target retinal diseases. UMMS has granted Ophthotechan option to obtain an exclusive license to any patent or patent applications that result from this research.
2018 Operational Update
This estimate does not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or associated development that the Company may pursue.
2018 Financial Highlights
- Revenues: The Company did not have any collaboration revenue
for the quarter and six months ended
June 30, 2018, compared to $1.7 millionand $3.3 millionfor the same periods in 2017. Collaboration revenue decreased due to the completion of the Company’s deliverables under its previous licensing and commercialization agreement with Novartis Pharma AGand the recognition of all associated deferred revenue during the third quarter of 2017.
- R&D Expenses: Research and development expenses were
$8.5 millionfor the quarter ended June 30, 2018, compared to $15.7 millionfor the same period in 2017. For the six months ended June 30, 2018, research and development expenses were $16.2 millioncompared to $47.6 millionfor 2017. As the Company pursues its ongoing and planned Zimura and gene therapy development programs, research and development expenses decreased primarily due to decreases in expenses related to the discontinuation of the Company’s FovistaPhase 3 clinical program and decreases in costs associated with the Company’s 2017 reduction in personnel program.
- G&A Expenses: General and administrative expenses were
$6.3 millionfor the quarter ended June 30, 2018, compared to $8.6 millionfor the same period in 2017. For the six months ended June 30, 2018, general and administrative expenses were $12.0 millioncompared to $21.7 millionfor 2017. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure and decreases in costs associated with its 2017 reduction in personnel program, which includes facilities lease termination expenses incurred during the first quarter of 2017.
- Net Loss: The Company reported a net loss for the quarter ended
June 30, 2018of $13.2 million, or ($0.37)per diluted share, compared to a net loss of $22.2 million, or ($0.62)per diluted share, for the same period in 2017. For the six months ended June 30, 2018, the Company reported a net loss of $26.3 million, or ($0.73)per diluted share, compared to a net loss of $65.3 million, or ($1.82)per diluted share, for the same period in 2017.
Conference Call/Web Cast Information
Any statements in this press release about Ophthotech’s future
expectations, plans and prospects constitute forward-looking statements
for purposes of the safe harbor provisions under the Private Securities
Litigation Reform Act of 1995. Forward-looking statements include any
statements about Ophthotech’s strategy, future operations and future
expectations and plans and prospects for
|Selected Financial Data (unaudited)|
|(in thousands, except per share data)|
|Three Months Ended June 30,||Six Months Ended June 30,|
|Statements of Operations Data:|
|Research and development||8,516||15,657||16,202||47,636|
|General and administrative||6,332||8,552||11,977||21,711|
|Total operating expenses||14,848||24,209||28,179||69,347|
|Loss from operations||(14,848)||(22,548)||(28,179)||(66,024)|
|Loss before income tax provision (benefit)||(14,246)||(22,205)||(27,120)||(65,324)|
|Income tax provision (benefit)||(1,037)||(1)||(838)||2|
|Net loss per common share:|
|Basic and diluted||$||(0.37)||$||(0.62)||$||(0.73)||$||(1.82)|
|Weighted average common shares outstanding:|
|Basic and diluted||36,188||35,858||36,171||35,831|
June 30, 2018
|December 31, 2017|
|Balance Sheets Data:|
|Cash, cash equivalents, and marketable securities||$||145,991||$||166,972|
|Royalty purchase liability||125,000||125,000|
|Additional paid-in capital||528,530||522,759|
|Total stockholders' equity||$||17,530||$||38,041|
Kathy Galante, 212-845-8231
Vice President, Investor Relations and Corporate Communications
SmithSolve LLC on behalf of Ophthotech Corporation
Alex Van Rees, 973-442-1555 ext. 111