Ocugen to Discontinue Phase 3 oGVHD Trial
June 01 2020 - 9:20AM
Ocugen, Inc. (NASDAQ: OCGN), a clinical-stage company focused on
discovering, developing and commercializing transformative
therapies to treat the whole eye, today announced the decision to
discontinue the Phase 3 trial of OCU300 for ocular Graft vs. Host
Disease (oGVHD). The decision to stop the trial is based on results
of a pre-planned interim sample size analysis conducted by an
independent Data Monitoring Committee, which indicated the trial
was unlikely to meet its co-primary endpoints upon completion. The
study was not stopped based on safety concerns. We will
analyze the full data when it is available.
“This is disappointing news, especially for those who suffer
from oGVHD. Our hope was to provide the first treatment for
this complex, orphan disease. We are grateful to the
patients, their families and the investigators who participated in
our clinical trial,” said Shankar Musunuri, Chairman, CEO and
Co-Founder of Ocugen. “We are committed to advancing our
transformative therapies and will shift our resources to focus on
our breakthrough modifier gene therapy platform and novel biologic
programs for patients suffering from blindness diseases. We
remain on track to enter the clinic by next year with our first
gene therapy product candidate, OCU400, which targets inherited
retinal diseases. Our additional pipeline products, OCU410
and OCU200, which are focused on major retinal diseases, are
targeted to enter the clinic by 2022. As a result of
discontinuing this study and proceeds from our ATM program we
believe that we have sufficient cash to fund our current operations
into the fourth quarter of 2020.
About OCU400
Ocugen is developing a modifier gene therapy platform to
generate therapies designed to fulfill unmet medical needs in the
area of retinal diseases, including inherited retinal diseases
(“IRDs”). This modifier gene therapy platform is based on nuclear
hormone receptors (“NHRs”), which have the potential to restore
homeostasis, the basic biological processes in the retina and
potentially rescue photoreceptors from degeneration. This
technology was licensed from the Harvard Medical School. Unlike
single-gene replacement therapies, which only target one gene at a
time, we believe OCU400, through its use of NHRs, represents a
novel approach in that it may address multiple retinal diseases
with one product. Consisting of a functional copy of the nuclear
hormone receptor gene NR2E3, OCU400 is delivered to target cells in
the retina using an adeno-associated viral (AAV) vector. OCU400 has
received two Orphan Drug Designations from the Food and Drug
Administration, one for the treatment of NR2E3 mutation-associated
retinal diseases and the other for the treatment of CEP290
mutation-associated retinal diseases. OCU400 has the potential to
eliminate the need for developing more than 150 individual products
and provide one treatment option for all Retinitis Pigmentosa (RP)
patients.
About Ocugen, Inc.
Ocugen, Inc. is a clinical-stage biopharmaceutical company
focused on discovering, developing and commercializing
transformative therapies to treat the whole eye. Our breakthrough
modifier gene therapy platform has the potential to treat multiple
retinal diseases with one drug – “one to many” and our novel
biologic product candidate aims to offer better therapy to patients
with underserved diseases such as wet age-related macular
degeneration, diabetic macular edema and diabetic retinopathy. For
more information, please visit www.ocugen.com.
Cautionary Note on Forward-Looking
Statements
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995, which are subject to risks and uncertainties. We may, in some
cases, use terms such as “predicts,” “believes,” “potential,”
“proposed,” “continue,” “estimates,” “anticipates,” “expects,”
“plans,” “intends,” “may,” “could,” “might,” “will,” “should” or
other words that convey uncertainty of future events or outcomes to
identify these forward-looking statements. Such statements are
subject to numerous important factors, risks and uncertainties that
may cause actual events or results to differ materially from our
current expectations. These and other risks and uncertainties are
more fully described in our periodic filings with the Securities
and Exchange Commission (the “SEC”), including the risk factors
described in the section entitled “Risk Factors” in the quarterly
and annual reports that we file with the SEC. Any forward-looking
statements that we make in this press release speak only as of the
date of this press release. Except as required by law, we assume no
obligation to update forward-looking statements contained in this
press release whether as a result of new information, future events
or otherwise, after the date of this press release.
Corporate Contact:
Ocugen, Inc.
Kelly Beck
kelly.beck@ocugen.com
+1 484-328-4698
Media Contact:
LaVoieHealthScience
Emmie Twombly
etwombly@lavoiehealthscience.com
+1 857-389-6042
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