Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) (“Eagle” or the
“Company”) and Enalare Therapeutics Inc. (“Enalare”) today
announced that the U.S. Food and Drug Administration (“FDA”) has
granted Orphan Drug Designation (“ODD”) to ENA-001 for the
treatment of Apnea of Prematurity (“AoP”), a new chemical entity
with a novel mechanism of action as a respiratory stimulant. AoP is
a development disorder attributed to immaturity of the pulmonary
system characterized by either cessation of breathing for more than
20 seconds or cessation of breathing that lasts less than 20
seconds but is accompanied by either bradycardia or hypoxemia. The
condition affects approximately 25% of all preterm infants.1
ENA-001 is designed to work peripherally by inhibiting Big
Potassium (BK) ion channels in the carotid bodies, which are
located in the neck. By inhibiting these channels, ENA-001 utilizes
the body’s own ventilation control system to stimulate breathing
and it does so across multiple causes (etiologies) of respiratory
depression.
“Eagle has a great deal of confidence in ENA-001, and the
granting of Orphan Drug Designation by the FDA validates our belief
in its potential to help this very vulnerable population of
premature infants. More broadly, we believe that this compound can
play an important role in addressing a significant unmet medical
need, notably for patients with post-operative respiratory
depression and in combatting community drug overdose. With its
compelling clinical and health economic value propositions, ENA-001
is an excellent long-term opportunity for Eagle and would fit well
within our hospital/critical care portfolio,” stated Scott Tarriff,
President and Chief Executive Officer of Eagle Pharmaceuticals.
“We are extremely gratified to receive Orphan Drug Designation
for ENA-001 in the treatment of neonates experiencing Apnea of
Prematurity. This is an important milestone that builds on the Rare
Pediatric Designation granted by the FDA for ENA-001 last December.
Currently, pharmacologic treatment for these neonates is typically
limited to caffeine or other methylxanthines. We are eager to
advance this program and bring improved options for the preterm
infants facing this challenging respiratory condition,” said Herm
Cukier, President and CEO of Enalare Therapeutics.
In August 2022, Eagle made an equity investment of $12.5 million
in Enalare, with a commitment to invest another $12.5 million six
months later and make two potential follow-on equity investments of
$15 million each contingent upon (i) the commencement of the
ENA-001 Phase 2 clinical trial, and (ii) the ENA-001 Phase 2
clinical trial reaching 50% enrollment. Eagle also has the option
to acquire the remaining Enalare shares for an aggregate purchase
price ranging from $100-$175 million plus royalty rights ranging
from 9%-12% on all future global net sales of any Enalare product,
paid to the ex-Eagle holders of Enalare shares at the time of
acquisition.
Orphan Drug Designation is granted to drugs or biological
products for the treatment of rare diseases or conditions that
impact fewer than 200,000 people in the United States. Incentives
that come with the designation include eligibility for federal
grants, research and development tax credits, waiver of filing
fees, and the potential for a seven-year marketing exclusivity
period. The designation does not alter the standard regulatory
requirements and process for obtaining marketing approval.
ENA-001 is also being developed in an Intramuscular (“IM”)
formulation in partnership with the Biomedical Advanced Research
and Development Authority (“BARDA”), part of the Administration for
Strategic Preparedness and Response in the U.S. Department of
Health and Human Services (contract number 75A50122C00072). The
funding is provided by BARDA to support the advanced research and
development of medical countermeasures (MCM) for chemical,
biological, radiological and nuclear (CBRN) agents, pandemic
influenza, and emerging infectious diseases that threaten the U.S.
civilian population.
The development of ENA-001 is also supported by the National
Institute on Drug Abuse (“NIDA”) of the National Institutes of
Health (“NIH”) under award number R44DA057133. The content of this
document is the responsibility of its authors and does not
necessarily represent the official views of the National Institutes
of Health.
About ENA-001
Enalare’s lead compound, ENA-001, is a one-of-a-kind new
chemical entity (NCE) designed as an agnostic respiratory
stimulant. The compound has a novel mechanism of action that
affects ventilation via the peripheral chemoreceptor pathways in
the carotid body. It utilizes the body’s own ventilation control
system to beneficially influence breathing and has been shown to be
effective and well tolerated in five human studies to date. With
its novel mechanism of action and based on findings to date, it
could potentially improve the lives of those impacted by several
life-threatening conditions, including community drug overdose,
post-operative respiratory depression, and apnea of prematurity.
ENA-001 is an investigational compound and is not approved for use
by the FDA.
About Apnea of Prematurity
Apnea of Prematurity is a development disorder attributed to
immaturity of the pulmonary system characterized by either
cessation of breathing for more than twenty seconds or cessation of
breathing which lasts less than twenty seconds but is accompanied
by either bradycardia or hypoxemia. Apnea of Prematurity affects
approximately twenty-five percent of all preterm infants and is
inversely correlated with gestational age and birth weight,
including nearly 100 percent in neonates with a gestational age of
less than 29 weeks or birth weight less than 1,000 grams. Apnea of
prematurity exposes these delicate neonates to repeated episodes of
hypoxemia which is shown to increase risk of mortality in the NICU
as well as short and long-term impaired neurological
development.
About Enalare Therapeutics Inc.
Enalare Therapeutics Inc. is a clinical-stage biopharmaceutical
company dedicated to developing novel therapies for patients
suffering from life-threatening acute respiratory and critical care
conditions, including community drug overdose, post-operative
respiratory depression, and apnea of prematurity. Enalare maintains
global rights to its novel compounds and intends to start
additional clinical trials with ENA-001 for several indications in
the near term.
About Eagle Pharmaceuticals, Inc.
Eagle is a fully integrated pharmaceutical company with research
and development, clinical, manufacturing and commercial expertise.
Eagle is committed to developing innovative medicines that result
in meaningful improvements in patients’ lives. Eagle’s
commercialized products include vasopressin, PEMFEXY®, RYANODEX®,
BENDEKA®, BELRAPZO®, TREAKISYM® (Japan), and BYFAVO® and BARHEMSYS®
through its wholly owned subsidiary Acacia Pharma Inc. Eagle’s
oncology and CNS/metabolic critical care pipeline includes product
candidates with the potential to address underserved therapeutic
areas across multiple disease states. Additional information is
available on Eagle’s website at www.eagleus.com.
Forward-Looking StatementsThis press release
contains “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995, as amended, and
other securities law. Forward-looking statements are statements
that are not historical facts. Words and phrases such as
“anticipated,” “forward,” “will,” “would,” “may,” “remain,”
“potential,” “prepare,” “expected,” “believe,” “plan,” “near
future,” “belief,” “guidance,” and similar expressions are intended
to identify forward-looking statements. These statements include,
but are not limited to, statements with respect to the development
of, potential benefits of and potential FDA submission for ENA-001,
including a potential IM formulation that could potentially enable
more rapid deployment in emergency situations and the potential to
develop an innovative and rapid treatment for respiratory
depression in a variety of settings; expectations with respect to
the BARDA award providing funding to Enalare to accelerate the
development of ENA-001; the achievement of milestones and
deliverables; the potential further investment by Eagle in Enalare
and Eagle’s development programs, products and pipeline; the
potential use of ENA-001 to help preterm infants with respiratory
conditions; the ability of ENA-001 and other products and product
candidates to address unmet clinical needs, including for patients
with post-operative respiratory depression and in combatting
community drug overdose; the potential market opportunity for
products or product candidates, including ENA-001; and the
availability of ODD merits such as potential exclusivity periods
and certain research and development tax incentives. All of such
statements are subject to certain risks and uncertainties, many of
which are difficult to predict and generally beyond the Company’s
or Enalare’s control, that could cause actual results to differ
materially from those expressed in, or implied or projected by, the
forward-looking information and statements. Such risks and
uncertainties include, but are not limited to: the impacts of the
ongoing COVID-19 pandemic, including interruptions or other adverse
effects on clinical trials and delays in regulatory review or
further disruption or delay of any pending or future litigation;
delay in or failure to obtain regulatory approval of the Company's
or Enalare’s product candidates and successful compliance with FDA,
European Medicines Agency and other governmental regulations
applicable to product approvals; the outcome of litigation
involving any of its products or that may have an impact on any of
its products; the strength and enforceability of the Company’s
intellectual property rights or the rights of third parties; the
risks inherent in drug development and in conducting clinical
trials; the ability of Enalare to achieve milestones and
deliverables under the BARDA agreement and otherwise accelerate and
achieve successful results in the development of ENA-001; and those
risks and uncertainties identified in the “Risk Factors” sections
of the Company's Annual Report on Form 10-K for the year ended
December 31, 2021, filed with the Securities and Exchange
Commission (the “SEC”) on March 8, 2022, the Company’s Quarterly
Report on Form 10-Q for the quarter ended March 31, 2022, filed
with the SEC on May 9, 2022, the Company’s Quarterly Report on Form
10-Q for the quarter ended June 30, 2022, filed with the SEC on
August 9, 2022 and its other subsequent filings with the SEC.
Readers are cautioned not to place undue reliance on these
forward-looking statements. All forward-looking statements
contained in this press release speak only as of the date on which
they were made. Except to the extent required by law, the Company
undertakes no obligation to update such statements to reflect
events that occur or circumstances that exist after the date on
which they were made.
Investor Relations for Eagle Pharmaceuticals,
Inc.:
Lisa M. WilsonIn-Site Communications, Inc. T: 212-452-2793 E:
lwilson@insitecony.com
1 Martin RJ, Abu-Shaweesh JM, Baird TM (2004) Apnoea of
prematurity. Paediatr Respir Rev, 5 Suppl A, S377-382.
Eagle Pharmaceuticals (NASDAQ:EGRX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Eagle Pharmaceuticals (NASDAQ:EGRX)
Historical Stock Chart
From Apr 2023 to Apr 2024