Ophthotech Reports Fourth Quarter and Full Year 2018 Financial and Operating Results
- Conference Call and Webcast Today,
“2018 was a significant year for transforming the Company by executing
multiple business development transactions that resulted in the addition
of four gene therapy research and development programs targeting orphan
inherited retinal diseases and an age-related therapeutic development
program to our retina portfolio,” stated
Gene Therapy Program Highlights of 2018
In 2018, the Company initiated multiple innovative gene therapy collaborations to discover and develop next-generation therapies for the treatment of inherited retinal diseases.
June 2018, the Company entered into an exclusive global license agreement with the University of Florida Research Foundation (UFRF), Incorporatedand the Trustees of the University of Pennsylvania( Penn) for rights to develop and commercialize a novel adeno-associated virus (AAV) gene therapy product candidate for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), an orphan monogenic retinal disease. The novel AAV gene therapy construct is designed to knock down toxic rhodopsin and deliver a transgene for healthy rhodopsin via a single AAV vector in a mutation independent manner. The Company expects to initiate a Phase 1/2 clinical trial in RHO-adRP in 2020.
In August, proof-of-concept study results of this product
candidate in a naturally occurring canine disease model were
published in the prestigious journal Proceedings of the
National Academy of Sciences of the USA. This publication entitled: “Mutation-independent Rhodopsin Gene Therapy by Knockdown and Replacement with a Single AAV vector” was published by scientists at Pennand University of Florida.
- In August, proof-of-concept study results of this product candidate in a naturally occurring canine disease model were published in the prestigious journal Proceedings of the
October 2018, the Company entered into its second series of gene therapy agreements with Pennand UFRF, including an exclusive option agreement for rights to negotiate to acquire an exclusive global license to develop and commercialize novel AAV gene therapy product candidates for the treatment of diseases impacted by the bestrophin, or BEST1, gene. The Company expects to initiate a Phase 1/2 clinical trial in 2021.
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 Center. The research seeks to utilize a minigene strategy to create novel AAV gene therapy product candidates for Leber Congenital Amaurosis type 10 due to CEP290 mutations (the most common type of LCA), and autosomal recessive Stargardt disease due to ABCA4 mutations, both of which are orphan inherited retinal diseases, and to evaluate different AAV gene delivery methods for application in the eye.
Therapeutic Program Highlights of 2018
Complement Factor C5 Inhibitor Program: Zimura®
October 2018, the Company completed patient enrollment for its ongoing randomized, double-masked, sham controlled, multi-center Phase 2b clinical trial of Zimura for the treatment of geographic atrophy secondary to dry AMD. The Company expects initial top-line data for this trial to be available in the fourth quarter of 2019.
- Patient enrollment in the 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) is completed and initial top-line data is expected to be available in the second half of 2020.
Corporate Highlights of 2018
October 2018, Ophthotechacquired Inception 4, Inc., a privately held company backed by Versant Ventures. Ophthotechgained worldwide development and commercialization rights to Inception 4’s small molecule inhibitors of HtrA1 (high temperature requirement A serine peptidase 1 protein). In addition, Versant Venturesagreed to help Ophthotechidentify opportunities to expand the pipeline. Ophthotechobtained approximately $6.1 millionin cash through its acquisition of Inception 4 and issued approximately 5.2 million shares to the shareholders of Inception 4. After giving effect to the transaction, funds affiliated with Versant Venturesown approximately 12.5% of the outstanding shares of Ophthotech’s common stock.
During 2018, the Company expanded its Board of Directors by adding
leading industry experts:
Adrienne L. Graves, Ph.D., former Chief Executive Officer of Santen, Inc., and Jane Pritchett Henderson, Chief Financial Officer of Turnstone Biologics and former Chief Financial Officer and Senior Vice President of Corporate Development at Voyager Therapeutics. Effective January 1, 2019, Calvin W. Roberts, M.D., Senior Vice President and Chief Medical Officer, Eye Care at Bausch Health Companiesand Clinical Professor of Ophthalmology at Weill Medical College of Cornell Universitywas elected to Ophthotech’s Board of Directors.
2019 Operational Update
2018 Financial Highlights
- Gain on Extinguishment of Royalty Purchase Liability: The
Company recognized a gain on extinguishment of its royalty purchase
$125 millionfor the quarter and year ended December 31, 2018, due to the December 2018termination of the Company’s royalty purchase and sale agreement with Novo Holdings A/S. The termination of the agreement relieved the Company of any obligation to pay Novo Holdings A/S future product royalties on sales of certain platelet derived growth factor (PDGF) antagonists and did not impact the Company’s cash balance.
- Revenues: The Company did not have any collaboration revenue
for the quarters ended
December 31, 2018and 2017 or during the year ended December 31, 2018, compared to $210 millionfor the year ended December 31, 2017. Collaboration revenue decreased for 2018 as compared to 2017 as the Company had recognized all deferred revenue associated with the completion of the Company’s licensing and commercialization agreement with Novartis Pharma AGduring the third quarter of 2017 and had no collaboration revenue in 2018.
- R&D Expenses: Research and development expenses were
$16.1 millionfor the quarter ended December 31, 2018, compared to $7.9 millionfor the same period in 2017. Research and development expenses increased primarily due to $6.9 millionin charges associated with the Company’s October 2018acquisition of Inception 4 and its HtrA1 inhibitor program and increases in costs associated with the Company’s ongoing Zimura and gene therapy development programs. For the year ended December 31, 2018, research and development expenses were $41.7 millioncompared to $66.3 millionfor 2017. 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 partially offset by charges associated with the Company’s October 2018acquisition of Inception 4 and its HtrA1 inhibitor program and increases in costs associated with the Company’s ongoing Zimura and gene therapy development programs.
- G&A Expenses: General and administrative expenses were
$5.7 millionfor the quarter ended December 31, 2018, compared to $6.9 millionfor the same period in 2017. For the year ended December 31, 2018, general and administrative expenses were $23.6 millioncompared to $35.7 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 included facilities lease termination expenses incurred during the first quarter of 2017.
- Net Income: The Company reported net income for the quarter
December 31, 2018of $104.1 million, or $2.62per diluted share, compared to net loss of $9.5 million, or ($0.26)per diluted share, for the same period in 2017. For the year ended December 31, 2018, the Company reported net income of $63.1 million, or $1.70per diluted share, compared to net income of $114.2 million, or $3.17per diluted share, for the same period in 2017.
Conference Call/Web Cast Information
Any statements in this press release about Ophthotech’s future
expectations, plans and prospects constitute forward-looking statements
for purposes of the safe harbor provisions under the Private Securities
Litigation Reform Act of 1995. Forward-looking statements include any
statements about Ophthotech’s strategy, future operations and future
expectations and plans and prospects for
|Selected Financial Data (unaudited)|
|(in thousands, except per share data)|
|Three months ended December 31,||Year ended December 31,|
|Statements of Operations Data:|
|Research and development||16,128||7,946||41,737||66,289|
|General and administrative||5,667||6,913||23,612||35,683|
|Total operating expenses||21,795||14,859||65,349||101,972|
|Income (loss) from operations||(21,795||)||(14,859||)||(65,349||)||108,005|
|Gain on extinguishment of Royalty Purchase Liability||125,000||-||125,000||-|
|Other income (expense)||1||-||(16||)||(34||)|
|Income (loss) before income tax benefit||103,883||(14,450||)||62,024||109,493|
|Income tax benefit||(231||)||(4,908||)||(1,063||)||(4,712||)|
|Net income (loss)||$||104,114||$||(9,542||)||$||63,087||$||114,205|
|Net income (loss) per common share:|
|Weighted average common shares outstanding:|
|December 31, 2018||December 31, 2017|
|Balance Sheets Data:|
|Cash and cash equivalents||$||131,201||$||166,972|
|Royalty purchase liability||-||125,000|
|Additional paid-in capital||545,585||522,759|
|Total stockholders' equity||$||123,959||$||38,041|
Vice President, Investor Relations and Corporate Communications
Alex Van Rees, 973-442-1555 ext. 111
SmithSolve LLC on behalf of Ophthotech Corporation