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OHR Pharma Offers Positive Interim Top-Line Clinical Data from Phase II Study of Squalamine Eye Drops in Patients with WetAMD

Ohr Pharmaceutical, Inc.
(Nasdaq: OHRP), an ophthalmology research and development company, today
announced positive top-line interim results for its double-masked,
placebo-controlled Phase II clinical trial of Squalamine eye drops in patients
with wet age-related macular degeneration (wet AMD). The data demonstrated a
positive benefit in visual function across multiple clinically relevant
endpoints, including a mean change in visual acuity at the end of study visit
for the interim analysis group of +10.4 letters with Squalamine eye drops plus
Lucentis^® PRN versus +6.3 letters in the placebo eye drops plus Lucentis PRN
arm, a 65 percent additional relative benefit (p=0.18). The visual acuity
improvements were seen as early as four weeks and the relative difference in
visual acuity between the two treatment arms continued to increase throughout
the study.

All patients in the study received an initial Lucentis injection followed by
Lucentis as needed (PRN) based on clinical response. The two treatment arms
were Squalamine eye drops administered twice daily plus Lucentis PRN
("Squalamine" arm or group) versus standard-of-care treatment: placebo eye
drops administered twice daily plus Lucentis PRN ("placebo" arm or group).

This planned interim analysis was conducted on the first 62 patients (29
treated in the Squalamine arm, 33 treated in the placebo arm), who completed
the entire nine months of the treatment protocol (representing approximately
50 percent of the targeted study population). The Squalamine-treated group
demonstrated improved best-corrected visual acuity (BCVA) gains relative to
the placebo group at all timepoints evaluated from four to 38 weeks. In the
interim analysis group, 48.3 percent of Squalamine-treated patients showed
BCVA gains of ≥ 15 letters (≥ 3 lines) on a standard ETDRS eye-chart, compared
with 21.2 percent in the placebo arm at the end of the study (p=0.025). In
addition, patients receiving Squalamine drops were more than twice as likely
to gain ≥ 4 and ≥ 5 lines of vision compared with patients in the placebo eye
drop arm (≥ 4 lines p=0.022, ≥ 5 lines p=0.059). Importantly, the visual
acuity gains for the placebo eye drop arm were consistent with those observed
in previous clinical studies using Lucentis monotherapy treatment. Squalamine
eye drops were well tolerated and had a comparable safety profile to placebo
eye drops.

"The beneficial effects of Squalamine on visual acuity that we've seen thus
far, through its inhibition of multiple angiogenic growth factors and
pathways, and in particular, the improvement in gains of three or more lines
in vision compared with the placebo group, are truly remarkable," said Dr.
Jason Slakter, Chief Medical Officer of Ohr and retina specialist at
Vitreous-Retina-Macula Consultants of NY. "Visual acuity is the most
clinically relevant endpoint for back-of-the-eye disorders. For wet-AMD
patients, such enhanced gains of visual acuity over standard-of-care anti-VEGF
treatments, and the restoration of vision lost to this devastating disease of
the elderly using a convenient eye drop therapy is a very important clinical
outcome."

In the interim analysis, there were no significant differences in the
frequency of Lucentis PRN injections, which was the primary endpoint of the
study. The mean number of Lucentis injections was 6.2 for the Squalamine arm
and 6.4 for the placebo arm, which included the baseline injection and any
injections required up to and including the final study visit for the interim
analysis group.

"The interim results seen in this trial are encouraging," said Dr. Jeffrey S.
Heier, Director of Vitreoretinal Service at Ophthalmic Consultants of Boston,
member of Ohr's scientific advisory board, and study investigator. "The
potential to treat patients with a non-invasive therapeutic option to provide
additional visual acuity benefit over the current standard-of-care, and do it
with a less than monthly injection frequency, would be a significant advance
in the treatment of retinal neovascular disease and beneficial for our
patients. We look forward to the results of the full data set and Phase III
trials."

"We are very excited by these promising interim results from this wet-AMD
trial," said Dr. Irach B. Taraporewala, President and Chief Executive Officer
of Ohr. "The data further validate not only the clinical utility of
non-invasive topical eye drop therapies for macular and retinal disorders, but
also the soundness of our company's drug development science, and our
proprietary formulation technologies that enable topical dosing to achieve
positive therapeutic effects in back-of-the-eye disorders. These data give us
a clear path for future registration studies, and we plan to discuss Phase III
registration study design and the path forward with the regulatory authorities
in the coming months."

The company plans to present the full data from this interim analysis at an
ophthalmology conference in the second half of this year, with final clinical
trial data expected in the first calendar quarter of 2015.

Study Design

The ongoing clinical trial (Study OHR-002) is a randomized, double-masked,
placebo-controlled Phase II study to evaluate the efficacy and safety of
Squalamine eye drops used in combination with Lucentis PRN for the treatment
of the advanced, exudative form of age-related macular degeneration (AMD),
also known as "wet AMD." The trial has enrolled 142 newly diagnosed, treatment
naïve patients, of which the first 62 had completed the treatment period at
the time of the interim analysis. The inclusion criteria allowed for patients
with visual acuity levels similar to previous Lucentis trials, varying lesion
sizes up to 12 disc areas in size, and any lesion composition, including
classic and occult only types of wet AMD. The trial also included diabetics
with no concomitant diabetic retinopathy.

Patients received an initial intravitreal injection of Lucentis at study
entry, and then underwent a 1:1 randomization to receive a daily dose of
either Squalamine eye drops or placebo eye drops administered twice daily for
nine months. Patients had monthly follow-up clinic visits, where they were
evaluated and retreated as needed with Lucentis if pre-specified clinical
criteria were met. The primary endpoint was the mean number of Lucentis
injections and secondary endpoints included visual acuity as well as
diagnostic imaging outcomes. The planned interim analysis was performed when
more than 50 percent of the study population finished their final study visit.

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