OPKO Health, Inc. (NASDAQ:OPK) through its
subsidiaries, Eirgen Pharma Limited, Ireland and OPKO
Pharmaceuticals LLC, has received notification from the European
Commission designating OPKO’s oligonucleotide-based AntagoNAT
(CUR-1916) an orphan medicinal product under Regulation (EC) No
141/2000 for the treatment of Dravet Syndrome. An Orphan Drug
Application is under review by the U.S. FDA. There is currently no
approved treatment for Dravet Syndrome in the U.S.
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 was part of CURNA
Pharmaceuticals, acquired by OPKO in 2011, and then further
developed in OPKO’s Miami research laboratories under the direction
of Dr. 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 7 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.
It is worth noting that several oligonucleotide compounds have
been reported to be in late phase clinical development and one has
been approved by the FDA in 2016. They work by down regulating
transcription (antisense) or by correcting gene defects.
About EMA Orphan Designation
The European Medicines Agency (EMA) grants Orphan Designation to
medicines intended to treat, prevent or diagnose life threatening
and debilitating diseases, with a prevalence no greater than five
in 10,000 in the EU, and for which no satisfactory method of
treatment, prevention or diagnosis exists, and the proposed
medicine offers significant medical benefit to those with the
condition. Following Orphan Designation, sponsors can access a
number of incentives including market exclusivity for a ten-year
period following approval, protocol assistance, and potential fee
reductions.
On November 24, 2016, OPKO requested Scientific Advice for the
clinical development strategy of CUR-1916, and Committee for the
Medicinal Products for Human Use (CHMP) has since met with OPKO and
provided its advice for the CUR-1916 clinical development program.
This is an important milestone for initiating a clinical trial of
CUR-1916 designed to assess drug safety and significant medical
benefits to patients with Dravet Syndrome.
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 is debilitating and the death rate is reported
to be 10-15%.
About OPKO Health, Inc.
OPKO Health is a diversified healthcare company that seeks to
establish industry-leading positions in large, rapidly growing
markets. Our diagnostics business includes Bio-Reference
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 which is a clinically advanced drug
candidate among the new class of GLP-1 glucagon receptor dual
agonists (phase 2), and TT701, an androgen receptor modulator for
androgen deficiency indications (phase 2). Our biologics business
includes hGH-CTP, a once weekly human growth hormone injection (in
phase 3 and partnered with Pfizer), and a long-acting Factor VIIa
drug for hemophilia (in phase 2a). We also have production and
distribution assets worldwide, multiple strategic investments and
an active business development strategy. 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. Tara Mackay,
305-575-4100 Investor Relations or Media Rooney
Partners Terry Rooney, 212-223-0689 trooney@rooneyco.comorMarion
Janic, 212-223-4017mjanic@rooneyco.com
or Investors LHA Anne Marie Fields,
212-838-3777 afields@lhai.com or Bruce Voss, 310-691-7100
bvoss@lhai.com
Opko Health (NASDAQ:OPK)
Historical Stock Chart
From Mar 2024 to Apr 2024
Opko Health (NASDAQ:OPK)
Historical Stock Chart
From Apr 2023 to Apr 2024