* Ohr seeks to follow commercial success of Regeneron, Roche
* Eye drop could complement competitors' injection therapies
* Millions of Americans affected by wet age-related macular
* Interim analysis of mid-stage trial data expected in 2014
By Esha Dey
Dec 3 Patients who are being treated for a
leading cause of blindness among the elderly might be able to
scale back on eye injections if trials confirm the potential of
an eye drop being developed by a small New York-based company.
Ohr Pharmaceutical Inc's Squalamine drug - an eye
drop, as opposed to the injection therapies sold by Regeneron
Pharmaceuticals Inc and Roche Holdings -
doesn't necessarily need to outsell its formidable competitors.
Simply proving that the drop can be used as a complementary
treatment to Regeneron's Eylea or Roche's Lucentis could be
enough to ensure commercial success for Ohr in treating wet
age-related macular degeneration (AMD).
The main challenge will be to demonstrate Squalamine's
ability to travel to the back of the eye and stay there long
enough to be absorbed. A mid-stage clinical trial is under way.
Ohr Pharmaceutical, backed by Baltimore and New York
investors, was formed in August 2009. Within a month, it bought
the rights to Squalamine for $200,000 from a trust set up to
monetize the assets of now-defunct drug developer Genaera Corp.
The company's stock has doubled in the last year but, with a
market capitalization of about $160 million, Ohr is a minnow
alongside its competitors in the lucrative market to treat AMD.
Regeneron's Eylea drug, approved in November 2011, has
transformed a relatively obscure biotech firm to a company worth
nearly $30 billion today. In 2012, its first full year in the
market, Eylea generated $838 million in sales.
Roche's drug, Lucentis, which is used to treat AMD and two
other eye diseases, had sales of $1.5 billion in the United
States last year.
AMD affects 1.8 million Americans aged 40 and above,
according to the Centers for Disease Control and Prevention.
With millions more at "substantial risk" of developing the
condition, the number of patients could reach nearly 3 million
in 2020, it says.
AMD is a chronic condition that requires visually impaired
patients to make monthly visits to the clinic.
This is where Squalamine comes in. The eye drop is being
tested to determine whether it can reduce the frequency of eye
injections needed after an initial dose of Lucentis.
Neil Bressler, chief of the retina division at the Wilmer
Eye Institute at Johns Hopkins Medicine in Baltimore, said
patients using Eylea or Lucentis required an average of seven
injections during their first year of treatment and five or six
in the second year.
"Even if you reduce those 12 or 13 injections to nine or 10,
and if it is just a drop, then the safety and the ease is very,
very good," said Bressler, an independent observer who has no
affiliation with Ohr.
Regeneron and Roche declined to comment for this article.
Interim analysis of data from the mid-stage trial is
expected in the second quarter of 2014.
Jonathan Aschoff, analyst at New York-based investment bank
Brean Capital LLC, said he believed the data could be
The only analyst listed by Thomson Reuters as covering Ohr,
Aschoff has a target of $21 on the stock - more than
two-and-a-half times its closing price of $7.98 on the Nasdaq on
He estimated peak Squalamine sales in excess of $1 billion a
year by 2020, assuming an annual price of $4,000 for the eye
drop as a complementary treatment to - not a replacement for -
While extremely effective, Lucentis and Eylea - biotech
drugs, as opposed to Ohr's chemical-based pharmaceutical drug -
are relatively expensive; Lucentis costs about $2,000 per
injection and Eylea about $1,850.
If Squalamine is proven to be effective, its lower price
would make it an attractive proposition for patients, insurers
and austere governments.
"Complete replacement (for injections) is completely
unnecessary for Ohr's success," said Aschoff. "Just look at how
well Eylea is doing with reduction of injection frequency as its
Like Eylea and Lucentis, Squalamine targets a specific
protein, VEGF, or vascular endothelial growth factor, in order
to block the formation and growth of new blood vessels in the
eye. It also targets other factors behind blood vessel growth.
The biggest questions to date over its effectiveness center
on the drug's ability to travel behind the eye. Unlike everyday
cleansing eye drops, which work on the front of the eye,
Squalamine requires absorption at the back.
Genaera, Squalamine's former owner, tested the drug
intravenously in successful early trials. In eye-drop form, it
has been tested on rabbits, also with successful results.
Ohr Chief Executive Irach Taraporewala said the drug's
chemical composition would "allow the molecule, once it got to
the back of the eye, to stay there long enough to potentially
allow for once- or twice-a-day delivery."
The next set of trial data will be key for the investors
behind Ohr, including 7.1 percent-owner AIGH Investment
Partners, a Baltimore-based investment firm founded by Ohr
director Orin Hirschman.
If the results are good, Aschoff said it might eventually
make sense for Ohr, which is also developing a treatment for
cancer cachexia, a severe wasting disorder, to sell itself or
Squalamine to a bigger competitor.
"(The acquirer) could buy it to sit on it, and not develop
it, to save their injectable franchise," he said. "Or they could
buy it to develop it - and see whichever one brings in the most