Market Overview

Ophthotech Reports Second Quarter 2018 Financial and Operating Results


(Conference Call and Webcast Today, August 1, 2018, at 8:00 a.m. ET)

Ophthotech Corporation (NASDAQ:OPHT) today announced financial and
operating results for the second quarter ended June 30, 2018 and
provided a business update.

"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 Glenn P. Sblendorio, Chief
Executive Officer and President of Ophthotech. "Looking ahead to the
remainder of 2018, we expect to report data for our Phase 2a clinical
trial for Zimura combination therapy with anti-VEGF in wet-age related
macular degeneration (AMD), complete recruitment for our Phase 2b
clinical trial for Zimura monotherapy in geographic atrophy secondary to
dry AMD and potentially enter into new opportunities to further expand
our portfolio in both therapeutics and gene therapies for retinal

First Half 2018: Key Highlights

Zimura® Complement Factor C5 Inhibitor

  • In 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.
  • In 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.
    • In June 2018, the Company entered into an exclusive global license
      agreement with the University of Florida Research Foundation,
      Incorporated and 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. Ophthotech and Penn have also entered into
      a master sponsored research agreement, facilitated by the Penn
      Center for Innovation, pursuant to which Ophthotech and Penn plan
      to conduct natural history studies in RHO-adRP patients and
      additional preclinical studies. In parallel with the sponsored
      research, Ophthotech plans to commence IND-enabling activities.
      Based on current timelines and subject to regulatory review,
      Ophthotech expects to initiate a Phase 1/2 clinical trial in
      RHO-adRP in 2020.
    • In 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 to 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 Ophthotech an option
      to obtain an exclusive license to any patent or patent
      applications that result from this research.

2018 Operational Update

As of June 30, 2018, the Company had $146 million in cash and cash
equivalents. The Company estimates that its year end 2018 cash and cash
equivalents will range between $112 million and $117 million based on
its current 2018 business plan and planned capital expenditures. This
estimate includes continuation of the Company's development programs for
Zimura® and RHO-adRP gene therapy product candidate and the
continuation of the Company's collaborative gene therapy research
programs as currently planned.

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
    million and $3.3 million for 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 AG and the recognition of all associated deferred
    revenue during the third quarter of 2017.
  • R&D Expenses: Research and development expenses were $8.5
    million for the quarter ended June 30, 2018, compared to $15.7 million
    for the same period in 2017. For the six months ended June 30, 2018,
    research and development expenses were $16.2 million compared to $47.6
    million for 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 Fovista Phase 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
    million for the quarter ended June 30, 2018, compared to $8.6 million
    for the same period in 2017. For the six months ended June 30, 2018,
    general and administrative expenses were $12.0 million compared to
    $21.7 million for 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, 2018 of $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

Ophthotech will host a conference call/webcast to discuss the Company's
financial and operating results and provide a business update. The call
is scheduled for August 1, 2018 at 8:00 a.m. Eastern Time. To
participate in this conference call, dial 800-458-4121 (USA) or
323-794-2597 (International), passcode 3698278. A live, listen-only
audio webcast of the conference call can be accessed on the Investor
Relations section of the Ophthotech website at:
A replay will be available approximately two hours following the live
call for two weeks. The replay number is 888-203-1112 (USA Toll Free),
passcode 3698278.

About Ophthotech Corporation

Ophthotech is a science-driven biopharmaceutical company specializing in
the development of novel therapies to treat ophthalmic diseases, with a
focus on age-related and orphan retinal diseases. For more information,
please visit

Forward-looking Statements

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 Ophthotech, and any other
statements containing the words "anticipate," "believe," "estimate,"
"expect," "intend", "goal," "may", "might," "plan," "predict,"
"project," "target," "potential," "will," "would," "could," "should,"
"continue," and similar expressions. In this press release, Ophthotech's
forward looking statements include statements about the implementation
of its strategic plan, Ophthotech's projected use of cash and cash
balances, the timing, progress and results of clinical trials and other
research and development activities, the potential utility of its
product candidates and the potential for its business development
strategy, including its collaborative gene therapy research programs and
any potential in-license or acquisition opportunities. Such
forward-looking statements involve substantial risks and uncertainties
that could cause Ophthotech's clinical development programs, future
results, performance or achievements to differ significantly from those
expressed or implied by the forward-looking statements. Such risks and
uncertainties include, among others, those related to the initiation and
the conduct and design of research programs and clinical trials,
availability of data from these programs, expectations for regulatory
matters, need for additional financing and negotiation and consummation
of in-license and/or acquisition transactions and other factors
discussed in the "Risk Factors" section contained in the quarterly and
annual reports that Ophthotech files with the Securities and Exchange
Commission. Any forward-looking statements represent Ophthotech's views
only as of the date of this press release. Ophthotech anticipates that
subsequent events and developments will cause its views to change. While
Ophthotech may elect to update these forward-looking statements at some
point in the future, Ophthotech specifically disclaims any obligation to
do so except as required by law.


Ophthotech Corporation
Selected Financial Data (unaudited)
(in thousands, except per share data)
Three Months Ended June 30, Six Months Ended June 30,
  2018   2017   2018   2017
Statements of Operations Data:
Collaboration revenue $ - $ 1,661 $ - $ 3,323
Operating expenses:
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)
Interest income 602 344 1,075 722
Other expense   -   (1)   (16)   (22)
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 $ (13,209) $ (22,204) $ (26,282) $ (65,326)
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
(in thousands)
Balance Sheets Data:
Cash, cash equivalents, and marketable securities $ 145,991 $ 166,972
Total assets 151,661 175,576
Royalty purchase liability 125,000 125,000
Total liabilities 134,131 137,535
Additional paid-in capital 528,530 522,759
Accumulated deficit (511,036) (484,754)
Total stockholders' equity $ 17,530 $ 38,041

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