Ophthotech Announces Results from Pivotal Phase 3 Trials of Fovista® in Wet Age-Related Macular Degeneration

• No benefit observed upon addition of Fovista^®
  to monthly Lucentis^® regimen for the
  treatment of wet age-related macular degeneration –
• Conference Call and Webcast Today, December 12, 2016 at 8:30 a.m.
  ET –

NEW YORK–(BUSINESS WIRE)–Ophthotech Corporation (Nasdaq:OPHT) today announced that the
pre-specified primary endpoint of mean change in visual acuity at 12
months was not achieved in its two pivotal Phase 3 clinical trials
investigating the superiority of Fovista^® (pegpleranib)
anti-PDGF therapy in combination with Lucentis^® (ranibizumab)
anti-VEGF therapy compared to Lucentis^® monotherapy for the
treatment of wet age-related macular degeneration (AMD). The addition
of Fovista^® to a monthly Lucentis^® regimen did
not result in benefit as measured by the mean change in visual acuity at
the 12 month time point.

“We are very disappointed in the results from these trials, particularly
for patients afflicted with wet AMD,” commented David R. Guyer, M.D.,
Chief Executive Officer of Ophthotech. “We are thankful to the patients
and clinical investigators and their staff for participating in the
trials. We will continue to analyze the data from these two studies to
better understand the trial results.”

The two clinical trials (OPH1002 and OPH1003) were international,
multicenter, randomized, double-masked, controlled Phase 3 studies
evaluating the safety and efficacy of 1.5 mg of Fovista^®
administered in combination with Lucentis^® (Fovista^®
combination therapy) compared to Lucentis^® monotherapy. In
each of these trials, patients were randomized to one of two
approximately equal sized treatment groups. The two Phase 3 trials
enrolled an aggregate of 1,248 patients with wet AMD. The results from
the databases of the two trials were unmasked and analyzed concurrently.

The combined analysis from the two trials (OPH1002 and OPH1003) showed
that patients receiving Fovista^® combination therapy gained a
mean of 10.24 letters of vision on the Early Treatment of Diabetic
Retinopathy Study (ETDRS) standardized chart at 12 months, compared to a
mean gain of 10.01 ETDRS letters for patients receiving Lucentis^®
monotherapy, a difference of 0.23 ETDRS letters. In OPH1002, consisting
of 619 treated patients, subjects receiving Fovista^®
combination therapy gained a mean of 10.74 letters of vision on the
ETDRS standardized chart at 12 months, compared to a mean gain of 9.82
ETDRS letters in patients receiving Lucentis^® monotherapy, a
resulting difference of 0.92 ETDRS letters (p=0.44). In OPH1003,
consisting of 626 treated patients, subjects receiving Fovista^®
combination therapy gained a mean of 9.91 letters of vision on the ETDRS
standardized chart at 12 months, compared to a mean gain of 10.36 ETDRS
letters in patients receiving Lucentis^® monotherapy, a
resulting difference of -0.44 ETDRS letters (p=0.71). None of these
results of the pre-specified primary efficacy analysis were
statistically significant.

In the pooled analysis of pre-specified secondary endpoints from both
trials, 24.2% of patients receiving Fovista^® combination
therapy gained 20 or more ETDRS letters from baseline at month 12,
compared to 22.1% of patients receiving Lucentis^®
monotherapy. In OPH1002, 25.9% of patients receiving Fovista^®
combination therapy gained 20 or more ETDRS letters from baseline at
month 12, compared to 20.0% of patients receiving Lucentis^®
monotherapy. In OPH1003, 22.5% of patients receiving Fovista^®
combination therapy gained 20 or more ETDRS letters from baseline at
month 12, compared to 24.1% of patients receiving Lucentis^®
monotherapy.

In the pooled analysis, 12.1% of patients receiving Fovista^®
combination therapy lost 5 or more ETDRS letters from baseline at month
12, compared to 11.2% of patients receiving Lucentis^®
monotherapy. In OPH1002, 12.0% of patients receiving Fovista^®
combination therapy lost 5 or more ETDRS letters at month 12, compared
to 12.3% of patients receiving Lucentis^® monotherapy. In
OPH1003, 12.2% of patients receiving Fovista^® combination
therapy lost 5 or more ETDRS letters at month 12, compared to 10.2% of
patients receiving Lucentis^® monotherapy.

In addition, in the pooled analysis, 13.5% of patients receiving Fovista^®
combination therapy achieved visual acuity of 20/25 or better at month
12, compared to 13.9% of patients receiving Lucentis^®
monotherapy. In OPH1002, 13.6% of patients receiving Fovista^®
combination therapy achieved visual acuity of 20/25 or better, compared
to 13.2% of patients receiving Lucentis^® monotherapy. In
OPH1003, 13.5% of patients receiving Fovista^® combination
therapy achieved visual acuity of 20/25 or better, compared to 14.6% of
patients receiving Lucentis^® monotherapy.

Based on a preliminary analysis of the safety data from these two
trials, Fovista^® combination therapy and Lucentis^®
monotherapy were generally well tolerated after one year of treatment.
The ocular adverse events more frequently reported in the Fovista^®
combination therapy group compared to the Lucentis^®
monotherapy group were mainly related to the injection procedure. The
incidence of reported serious systemic adverse events was generally
similar in both treatment groups as was the incidence of myocardial
infarction or cerebrovascular accident.

Conference Call Information
Ophthotech’s
management team will host a live conference call and webcast today at
8:30 a.m. Eastern Time to discuss the data. To participate in the
conference call, dial 877-675-4749 (USA Toll Free) or 719-325-4781
(International Toll), passcode 4199836. A live, listen-only audio
webcast of the conference call can be accessed on the Investor Relations
section of the Ophthotech website, www.ophthotech.com.
A replay will be available approximately two hours following the live
call, and will be accessible for two weeks. The replay number is
888-203-1112 (USA Toll Free), passcode 4199836. The audio webcast can be
accessed at: www.ophthotech.com.

About Ophthotech Corporation
Ophthotech is a
biopharmaceutical company specializing in the development of novel
therapeutics to treat back of the eye diseases, with a focus on
developing innovative therapies for age-related macular degeneration
(AMD). Ophthotech’s most advanced product candidate, Fovista^®
anti-PDGF therapy, is in Phase 3 clinical trials for use in combination
with anti-VEGF therapy that represents the current standard of care for
the treatment of wet AMD. Ophthotech’s second product candidate, Zimura^®,
an inhibitor of complement factor C5, is being developed for the
treatment of geographic atrophy, a form of dry AMD, and in combination
with anti-VEGF therapy in wet AMD patients. For more information, please
visit www.ophthotech.com.

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 timing, progress and results of the Fovista^®
Phase 3 clinical program. 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 conduct of clinical
trials, availability of data from clinical trials and expectations for
regulatory approvals or other actions 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.

OPHT-G

Contacts

Investors
Ophthotech Corporation
Kathy Galante,
212-845-8231
Vice President, Investor Relations and Corporate
Communications
kathy.galante@ophthotech.com
or
Media
SmithSolve
LLC on behalf of Ophthotech Corporation
Alex Van Rees,
973-442-1555 ext. 111
alex.vanrees@smithsolve.com