Ovid Therapeutics Announces Phase 3 NEPTUNE Clinical Trial of OV101 for the Treatment of Angelman Syndrome Did Not Meet Prima...
December 01 2020 - 4:52PM
Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical
company committed to developing medicines that transform the lives
of people with rare neurological diseases, today announced topline
results from the Company’s Phase 3 NEPTUNE clinical trial of OV101
(gaboxadol) for the treatment of Angelman syndrome.
NEPTUNE is a randomized, double-blind, placebo-controlled, Phase
3 study that enrolled and treated 97 patients diagnosed with
Angelman syndrome, 4-12 years of age, and 7 patients diagnosed with
Angelman syndrome ages 2-3 years for safety and pharmacokinetic
evaluation only. The study was designed to assess the effects of
treatment with OV101 (oral, once-daily dosing) versus placebo over
12 weeks. The sole primary endpoint was change in overall score on
the Clinical Global Impression-Improvement-Angelman syndrome
(CGI-I-AS) scale. Secondary endpoints included sleep,
communication, motor function, socialization, daily living skills
and behavior domains.
The primary endpoint of the NEPTUNE study was not achieved.
Patients given OV101 showed a 0.7 point improvement in CGI-I-AS
over baseline while placebo also showed a 0.8 point improvement in
CGI-I-AS (p=NS). Secondary endpoints continue to be evaluated,
although initial results show no difference between OV101 and
placebo.
OV101 was well-tolerated, with no significant safety issues
observed. Ovid plans to complete a full analysis of the results of
the NEPTUNE study and discuss these results with the U.S. Food and
Drug Administration (FDA) to determine next steps, if any, for the
program. The Company will continue to offer study drug to patients
enrolled in the open-label extension trial (ELARA) pending further
analysis of the NEPTUNE study. The Company expects to report data
from the ELARA study in the first quarter of 2021.
“We are deeply disappointed with the outcome of the NEPTUNE
trial which did not achieve its primary endpoint,” said Jeremy
Levin, DPhil, MB, BChir, Chairman and Chief Executive Officer
of Ovid Therapeutics. “Other than the ongoing ELARA study, we
plan to pause our OV101 program in Angelman syndrome pending a full
understanding of this outcome and discussions with regulators and
investigators.”
Dr. Levin added: “For now, the focus of our future development
efforts will concentrate on the development of our other late-stage
asset, OV935, in two rare epilepsies: Dravet and Lennox-Gastaut
syndromes. With our partner Takeda, we plan to initiate pivotal
trials in these conditions in the first half of 2021.”
“NEPTUNE is our first study focused on the pediatric and
adolescent population in Angelman syndrome, and we will fully
assess all the data from this trial to understand this outcome and
determine next steps, if any, for OV101 in this and other
conditions, including Fragile X syndrome,” said Amit Rakhit M.D.,
President and Chief Medical Officer. “We are sincerely grateful for
the commitment and dedication of patients, families, investigators
and employees to this program, and in particular, to those who
participated in the NEPTUNE trial.”
About OV101
(gaboxadol)OV101 (gaboxadol) is a delta
(δ)-selective GABAA receptor agonist. These receptors are
thought to have a central role in tonic inhibition, a key
physiological process of the brain believed to be a core
pathophysiology underlying certain neurodevelopmental
disorders.
About Angelman Syndrome
Angelman syndrome is a rare genetic condition that is characterized
by a variety of signs and symptoms. Characteristic features of this
condition include delayed development, intellectual disability,
severe speech impairment, problems with movement and balance,
seizures, sleep disorders and anxiety.
About Ovid TherapeuticsOvid Therapeutics
Inc. is a New York-based biopharmaceutical company using
its BoldMedicine® approach to develop medicines that transform
the lives of patients with rare neurological disorders. Ovid has a
broad pipeline of potential first-in-class medicines in
development. The Company is developing OV935 (soticlestat) in
collaboration with Takeda Pharmaceutical Company Limited for the
potential treatment of rare developmental and epileptic
encephalopathies (DEEs). OVID is evaluating OV101 (gaboxadol) for
the treatment of Angelman syndrome and Fragile X syndrome. For more
information on Ovid, please visit www.ovidrx.com.
Forward-Looking StatementsThis press release
includes certain disclosures that contain “forward-looking
statements,” including, among other things, statements regarding
uncertainties regarding the impact of the NEPTUNE trial results on
the clinical development of OV101 in Angelman syndrome, likelihood
that data will support future development, the association of data
with treatment outcomes, the design, progress, timing, scope and
results of the Company’s clinical trials, the anticipated timing of
disclosure of results of clinical trials and the likelihood of
obtaining regulatory approval of Ovid’s product candidates. You can
identify forward-looking statements because they contain words such
as “will,” “appears,” “believes” and “expects.” Forward-looking
statements are based on Ovid’s current expectations and
assumptions. Because forward-looking statements relate to the
future, they are subject to inherent uncertainties, risks and
changes in circumstances that may differ materially from those
contemplated by the forward-looking statements, which are neither
statements of historical fact nor guarantees or assurances of
future performance. Important factors that could cause actual
results to differ materially from those in the forward-looking
statements include uncertainties in the development and regulatory
approval processes, and the fact that initial data from clinical
trials may not be indicative, and are not guarantees, of the final
results of the clinical trials and are subject to the risk that one
or more of the clinical outcomes may materially change as patient
enrollment continues and/or more patient data become available.
Additional risks that could cause actual results to differ
materially from those in the forward-looking statements are set
forth in Ovid’s filings with the Securities and Exchange Commission
under the caption “Risk Factors.” Such risks may be amplified by
the COVID-19 pandemic and its potential impact on Ovid’s business
and the global economy. Ovid assumes no obligation to update any
forward-looking statements contained herein to reflect any change
in expectations, even as new information becomes available.
Contacts
Investors and Media:Ovid Therapeutics
Inc.Investor Relations & Public Relationsirpr@ovidrx.com
OR
Investors:Argot PartnersMaeve Conneighton/Dawn
Schottlandt212-600-1902ovid@argotpartners.com
Media:Dan
Budwick1ABdan@1abmedia.com
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