THE DAILY BIOPHARMACEUTICAL NEWS SOURCE
JUNE 6, 2014
BIOTECH’S MOST RESPECTED NEWS SOURCE FOR MORE THAN 20 YEARS
VOLUME 25 , NO. 109
NewCo News: Start-up Ocugen eyes new opthalmology opportunities
By Michael Fitzhugh, Staff Writer
Entrepreneur Shankar Musunuri’s newest venture, Ocugen Inc., is
jumping into the race to develop a treatment for the rare disease
retinitis pigmentosa (RP) and another therapy for wet age-related
macular degeneration (AMD), with two preclinical biologics
exclusively licensed from the University of Colorado.
Musunuri, co-founder and former CEO of Nuron Biotech Inc., is
now president and CEO of Iogenetics Inc. He co-founded Aurora,
Colo.-based Ocugen late last year. As chairman of the fledgling
company, he’s putting development of OCU100, the company’s
lead candidate, into motion and recruiting an executive team to
lead the work. Meanwhile, with FDA orphan status for the drug
already in hand, Musunuri told BioWorld Today that OCU100 could
enter phase I testing for RP in late 2015, assuming the company can
If successful, OCU100 could provide the first approved drug for
some of the 100,000 people in the U.S. with the rare inherited eye
disease. People with RP experience a gradual decline in their vision
as the photoreceptor cells in the retina die.
Most people with the condition are legally blind by age 40. While
medical need in the area is high, so is development interest, with
33 active phase I, II and III trials, according to the Cortellis Clinical
Trials Intelligence database.
Ocugen’s other co-founder and board member, Uday Kompella,
invented both drugs. A former classmate of Musunuri’s, the CU
professor took the academic path, focusing on drug delivery and
opthalmology, while Musunuri honed his business chops at Pfizer
Inc. and Wyeth.
The two kept in touch and, when Kompella reached out to share his
inventions with Musunuri, they decided to found Ocugen to develop
After what Musunuri said was an easy and efficient negotiation with
CU, in March Ocugen finished licensing assets for the recombinant
lens epithelium-derived growth factor 1-326, now called OCU100,
and an anti-angiogenic tumstatin fusion protein, now OCU200.
A variety of mutations, including P23H mutation in
rhodopsin, an important protein in the retina, have been
linked to the development of RP, Kompella said. “P23H
rhodopsin is known to form large clusters or aggregates
within retinal cells, leading to cellular stress and ultimately
cell death,” he said.
OCU100 can “rescue” retinal cells from protein aggregation
and the stresses that aggregation poses.
Though Musunuri declined to say how much Ocugen would
need to advance the drug, Sucampo Pharmaceuticals Inc.
last year secured up to $22 million, which it said would
cover the majority of development costs for its unrelated
RP drug. That phase III program, testing the alreadyapproved eye drug unoprostone Rescula (isopropyl) for
the new indication, is expected to yield top-line results in
Ocugen’s second candidate, OCU200, has shown early
promise in treating wet AMD, a disease that leads to
blindness for the majority of the nearly 600,000 cases
diagnosed globally each year.
Musunuri provided some of the seed funding for Ocugen
himself, he said.
As he begins the journey to finance the company further, he’ll
likely be helped by growing investor interest in advanced
opthalmology therapies. Optogenetics is taking off as
researchers and investors take a closer look at techniques to
deliver genes or chemicals to activate neurons in response
Genable Technologies Ltd., for instance, recently tapped
Philadelphia-based Spark Therapeutics LLC to provide
manufacturing services and clinical development assistance
on its preclinical lead program, GT038 for rhodopsin-linked
autosomal dominant RP. (See BioWorld Today, Sept. 30,
2013, and March 26, 2014.) //
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