OPKO Pharmaceuticals LLC, a subsidiary of
OPKO Health,
Inc. (NASDAQ:OPK) announces that the Company has received
orphan drug designation from the U.S. Food and Drug Administration
(FDA) Office of Orphan Products Development for OPKO’s
oligonucleotide-based AntagoNAT (CUR-1916) for the treatment of
Dravet Syndrome. Currently, there is no approved treatment
for Dravet Syndrome in the U.S. On March 7, 2017, OPKO Health
received orphan drug designation for CUR-1916 for the treatment of
Dravet Syndrome from the European Commission.
Orphan drug designation provides certain marketing exclusivity,
tax credits for research and a waiver of the New Drug Application
user fee.
AntagoNAT, anti-Natural Antisense Transcripts, is an OPKO
platform technology in which single strand oligonucleotide
molecules are designed to interfere with regulatory gene expression
in order to enhance production of endogenous functional
proteins. The AntagoNAT technology, part of CURNA
Pharmaceuticals, acquired by OPKO in 2011, was further developed in
OPKO’s Miami research laboratories under the direction of Jane
Hsiao, Ph.D., OPKO’s Vice Chairman and Chief Technical Officer.
OPKO has studied over 250 genes and confirmed involvement of
natural antisense transcripts (NAT) in their regulatory pathways.
Of those, 89 genes were demonstrated to be subject to significant
upregulation of mRNA in in vitro screening, and seven AntagoNAT
oligonucleotides have been validated in vivo to date. OPKO plans to
initiate a clinical trial of CUR-1916 for treatment of Dravet
Syndrome this year.
Oligonucleotides are synthetic chemical compounds consisting of
mixtures of modified DNAs and RNAs. Only five oligonucleotide
compounds are approved by FDA for various indications and others
have been reported to be in late phase clinical development. The
majority of the compounds work by down regulating, or depressing
transcription (anti-sense) or by correcting gene defects. CUR-1916
works by upregulating a defective gene to increase the production
of functional protein.
About FDA Orphan Drug Designation
Under the Orphan Drug Act (ODA) the FDA grants Orphan Drug
status to drugs, vaccines, and diagnostic agents intended to treat
a disease affecting less than 200,000 American citizens. Under the
ODA, orphan drug sponsors qualify for seven-year FDA administered
market Orphan Drug Exclusivity, tax credits of up to 50% of R&D
costs, R&D grants, waived FDA fees, protocol assistance and may
get clinical trial tax incentives.
What is Dravet Syndrome?
Dravet Syndrome, also called severe myoclonic epilepsy of
infancy (SMEI), is a severe form of epilepsy that affects children
and adults. It is caused by defects in the SCN1A genes (voltage
gated sodium channel) required for the proper function of brain
cells.
In Dravet Syndrome, seizures begin in the first year of life,
and are most often associated with elevated body temperature
(febrile convulsions). Later, other types of seizures occur,
including status epilepticus (seizures lasting at least 5 minutes
and requiring emergency medical care). From age 2, the child’s
development begins to decline or reverse, and results in impaired
mental and motor skills, leading to long term disability. Dravet
Syndrome also qualifies as a Rare Pediatric Disease under Section
529 of Food, Drug, and Cosmetic Act (the FD&C Act).
Dravet Syndrome is debilitating and the death rate is reported
to be 10-15%.
About OPKO Health
OPKO Health is a diversified healthcare company that seeks to
establish industry leading positions in large, rapidly growing
markets. Our diagnostics business includes BioReference
Laboratories, the nation’s third largest clinical laboratory with a
core genetic testing business and a 400 person sales and marketing
team to drive growth and leverage new products, including the
4Kscore® prostate cancer test and the Claros® 1 in office
immunoassay platform. Our pharmaceutical business features
RAYALDEE, an FDA approved treatment for SHPT in stage 3-4 CKD
patients with vitamin D insufficiency (launched in November 2016),
VARUBI™ for chemotherapy induced nausea and vomiting (oral
formulation launched by partner TESARO and IV formulation pending
FDA approval), TT401, a once or twice weekly oxyntomodulin for type
2 diabetes and obesity, in phase 2 clinical trials,
among the new class of GLP-1 glucagon receptor dual agonists,
and TT701, a selective androgen receptor modulator for
benign prostatic hyperplasia (phase 2). Our biologics
business includes hGH-CTP, a once weekly human growth hormone in
phase 3 and partnered with Pfizer; and a long-acting Factor VIIa
drug for hemophilia in phase 2a. More information
available at www.opko.com
Cautionary Statement Regarding Forward-Looking
Statements
This press release contains "forward-looking statements," as
that term is defined under the Private Securities Litigation Reform
Act of 1995 (PSLRA), which statements may be identified by words
such as "expects," "plans," "projects," "will," "may,"
"anticipates," "believes," "should," "intends," "estimates," and
other words of similar meaning, including statements regarding
expectations about CUR-1916 and its effectiveness in treating
Dravet syndrome, whether the drug will receive orphan designation
in the U.S., whether we will commence clinical trials for CUR-1916
this year or at all, whether the data from any of our trials
will support approval, validation and/or reimbursement for our
products, as well as other non-historical statements about
our expectations, beliefs or intentions regarding our business,
technologies and products, financial condition, strategies or
prospects. Many factors could cause our actual activities or
results to differ materially from the activities and results
anticipated in forward-looking statements. These factors include
those described in our Annual Reports on Form 10-K filed and to be
filed with the Securities and Exchange Commission and in our other
filings with the Securities and Exchange Commission, as well as the
risks inherent in funding, developing and obtaining regulatory
approvals of new, commercially-viable and competitive products and
treatments. The forward-looking statements contained in this press
release speak only as of the date the statements were made, and we
do not undertake any obligation to update forward-looking
statements. We intend that all forward-looking statements be
subject to the safe-harbor provisions of the PSLRA.
Company OPKO Health, Inc. David Malina,
305-575-4100 Investor Relations or Media Rooney
Partners Terry Rooney, 212-223-0689trooney@rooneyco.comorMarion
Janic,
212-223-4017mjanic@rooneyco.comorInvestorsLHAAnne
Marie Fields, 212-838-3777afields@lhai.comorBruce Voss,
310-691-7100bvoss@lhai.com
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