Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical
company committed to developing medicines that transform the lives
of people with rare neurological diseases, today reported financial
results for the fourth quarter and full year ended December 31,
2019 and provided an overview of the Company’s recent progress and
key clinical readouts anticipated in 2020.
“We are entering a transformational period for Ovid,” said
Jeremy Levin, DPhil, MB, BChir, Chairman and Chief Executive
Officer of Ovid Therapeutics. “2020 is a year where the team at
Ovid looks to deliver on the hard work of the last several years.
In anticipation of these important clinical readouts, during the
fourth quarter of 2019 we raised approximately $56.0 million,
providing us with sufficient capital to achieve all of these
clinical data points. If we are successful, we have the potential
to change the practice of medicine in one area and drive exciting
new changes in a second.”
Amit Rakhit, M.D. MBA, President and Chief Medical Officer,
said, “We have made significant clinical progress across the
pipeline during 2019, which leads us to an eventful 2020 with
multiple clinical data readouts expected this year. This includes
initial data from the ongoing open-label Phase 2 ARCADE study in
CDKL5 deficiency disorder and Dup15q syndrome by the end of the
first quarter of 2020. In addition, early in the second quarter of
2020, we will have data from our signal-finding Phase 2 ROCKET
trial in Fragile X syndrome along with SKYROCKET, our
non-interventional trial in Fragile X syndrome. Also, we completed
enrollment significantly ahead of schedule in our
placebo-controlled Phase 2 ELEKTRA trial in children with Dravet
syndrome and Lennox-Gastaut syndrome, and as a result, we now
expect data to be available in the third quarter of 2020. We
are also pleased to report that all patients who have completed the
ELEKTRA and ARCADE trials have rolled over into our ENDYMION
open-label extension study.”
Dr. Rakhit continued, “We are also very excited to see the
results of our pivotal Phase 3 NEPTUNE trial in Angelman syndrome.
Patient interest in our trial has been strong, and we are on track
for clinical data mid-year. There are currently no approved
therapies for Angelman syndrome. Achieving a clinically significant
improvement in the NEPTUNE trial will indicate that OV101 has the
potential to provide these patients and their families with a
much-needed treatment, one for which the community has been waiting
since the condition was first described over 50 years ago. If
successful, this may serve as a basis for an oral once-a-day
treatment option for people with Angelman syndrome.”
OV101 (gaboxadol) for Angelman Syndrome and Fragile X
Syndrome
- Ovid continues to enroll patients in the pivotal Phase 3
NEPTUNE trial in Angelman syndrome. NEPTUNE is a Phase 3,
randomized, double-blind, placebo-controlled pivotal trial to
evaluate the efficacy and safety of gaboxadol in children ages 4 to
12 with a confirmed diagnosis of Angelman syndrome. The primary
endpoint, CGI-I-AS, is an objective clinician-assessed measure of
the totality of a patient’s change in a number of key affected
areas over the duration of the clinical trial. NEPTUNE, if
positive, will be part of a broad data set intended to support
registrational filings for gaboxadol in the U.S. and the rest of
the world. We expect to release topline data from NEPTUNE mid-year
2020.
- Ovid expects results from the Phase 2 ROCKET trial and the
SKYROCKET trial in Fragile X syndrome early in the second quarter
of 2020. There are no approved treatments for Fragile X
syndrome.
- The ROCKET trial is a signal-finding, randomized, double-blind,
parallel-group trial to evaluate the safety, tolerability and
efficacy of gaboxadol in males ages 13 to 22 with a confirmed
diagnosis of Fragile X syndrome. The primary objective of the study
is safety and tolerability of gaboxadol over 12 weeks of treatment
in three different active dose arms. The secondary objective is to
evaluate changes in behavior after 12 weeks of treatment using the
ABC-C scale adapted for Fragile X syndrome (ABC-FXS). If positive,
the data from ROCKET, along with data from SKYROCKET, will inform
next steps in the clinical path for gaboxadol in Fragile X
syndrome.
- SKYROCKET is a non-drug study of males ages 5 to 30 with a
confirmed diagnosis of Fragile X to assess the suitability of
scales for the measurement of behavior, sleep and functioning. This
trial is designed to provide additional data on the key endpoints
that are being explored in ROCKET, gain deeper understanding of
placebo effects in this disorder and provide comparative data on
the benefit offered by the standard of care.
OV935/TAK935 (soticlestat) for Rare Developmental and Epileptic
Encephalopathies (DEE)
- The ARCADE trial is enrolling patients with CDKL5 deficiency
disorder (CDD) and Dup15q syndrome, both of which have no approved
treatments. Initial data from this trial are expected before the
end of the first quarter 2020.
- ARCADE is a multicenter, open-label pilot study that will
evaluate the treatment of soticlestat in patients ages 2 to 55 with
refractory motor seizures associated with CDD or Dup15q syndrome.
This study consists of a four- to six-week screening period to
establish baseline seizure frequency followed by a 20-week
treatment period. The primary endpoint is the percent change in
motor seizure frequency in patients treated with soticlestat by
disorder. This initial data set may inform us on the development
path forward. Full results from the Phase 2 ARCADE trial are
expected in early 2021.
- ARCADE open-label long-term cohort: Once patients complete the
ARCADE trial, they are offered the chance to immediately enroll in
the open-label ENDYMION trial. To date, all patients who have
finished the ARCADE trial have opted to enroll in the ENDYMION
trial. Along with the initial ARCADE data in first quarter of 2020,
Ovid plans to report longer-term data from the ARCADE patients who
have enrolled in the ENDYMION trial.
- The ELEKTRA trial has completed enrollment in patients with
Dravet syndrome and Lennox-Gastaut syndrome (LGS). Topline results
are expected in the third quarter of 2020.
- ELEKTRA is an international, multicenter, randomized,
double-blind, placebo-controlled study that will evaluate the
treatment of soticlestat in pediatric patients ages 2 to 17 with
seizures associated with Dravet syndrome (convulsive seizures) or
LGS (drop seizures). The study is fully enrolled and was enrolled
substantially ahead of schedule. The study consists of a four- to
six-week screening period to establish baseline seizure frequency
followed by a 20-week treatment period. The primary endpoint is the
percent change from baseline in seizure frequency in patients
treated with soticlestat compared to placebo.
- ELEKTRA open-label long-term cohort: Once patients complete the
ELEKTRA trial, they are offered the chance to immediately enroll in
the open-label ENDYMION trial. All patients who have finished the
ELEKTRA trial have opted to enroll in the ENDYMION trial. Ovid
plans to report longer-term data from ENDYMION in conjunction with
the results of the Phase 2 ELEKTRA trial expected in the third
quarter of 2020.
- ENDYMION open-label long-term trial for all patients who
previously participated in a trial of soticlestat: The primary
objective of ENDYMION is to assess the long-term safety and
tolerability of soticlestat over four years of treatment in
patients with rare epilepsies and secondarily, to evaluate the
effect of soticlestat on seizure frequency over time. Ovid
anticipates that the longer-term ENDYMION data to be reported with
the results from ELEKTRA will also include patients from Ovid’s
Phase 1b/2a adult clinical trial in DEE and patients from the
ARCADE trial.
Summary of Anticipated Clinical Data Readouts
|
|
|
|
|
Product Candidate |
Trial |
Condition or Disease |
Phase of Clinical Trial |
Expected Timing of Data Release |
Soticlestat |
ARCADE (Initial Data) |
CDD or Dup15q syndrome |
Phase 2 |
End of 1Q 2020 |
Soticlestat |
ENDYMION – ARCADE Cohort |
CDD or Dup15q syndrome |
Open-label Extension |
End of 1Q 2020 (concurrent with ARCADE) |
Gaboxadol |
ROCKET |
Fragile X |
Phase 2 |
Early 2Q 2020 |
Gaboxadol |
SKYROCKET |
Fragile X |
Non-treatment, observational |
Early 2Q 2020 |
Gaboxadol |
NEPTUNE |
Angelman syndrome |
Phase 3 |
Mid-2020 |
Soticlestat |
ELEKTRA |
Dravet syndrome or LGS |
Phase 2 |
3Q of 2020 |
Soticlestat |
ENDYMION – All Patients |
CDD, Dup15q syndrome, Dravet syndrome, LGS, other DEEs |
Open-label Extension |
3Q of 2020 (concurrent with ELEKTRA) |
Soticlestat |
ARCADE (Full Data) |
CDD or Dup15q syndrome |
Phase 2 |
Early 2021 |
Fourth Quarter 2019 Corporate Update
- Raised approximately $56.0 million of net proceeds
from the October public offering and from the sales of common
stock through Ovid’s At-The-Market (ATM) program, strengthening the
balance sheet as we enter a year of important clinical
milestones.
- Strengthened the leadership team with the appointment of Jason
Tardio as Chief Commercial Officer as the Company prepares for its
next stage of growth.
Fourth Quarter and Year Ended December 31, 2019
Financial Results
- As of December 31, 2019, cash, cash equivalents and short-term
investments totaled $76.7 million, including net proceeds of $33.7
million from a public equity offering in October 2019, including
the exercise of the underwriters’ option to purchase additional
shares, and after deducting the underwriting discounts and
commissions and other offering expenses. This cash balance
also increased due to $22.3 million in net proceeds generated from
the sale of common stock under Ovid’s ATM facility during the
fourth quarter of 2019.
- Research and development expenses were $12.1 million and $42.2
million for the fourth quarter and year ended December 31, 2019,
respectively, as compared to $8.6 million and $33.8 million for the
same periods in 2018. The increase for the year ended December 31,
2019, was primarily due to an increase in development
activities related to our ongoing development programs.
- General and administrative expenses were $5.2 million and $19.3
million for the fourth quarter and year ended December 31, 2019,
respectively, as compared to $4.5 million and $19.1 million for the
same periods in 2018. The difference was primarily due to an
increase in legal and professional fees, offset by decreases in
general office expenses, payroll and payroll-related
expenses.
- The Company reported a net loss of $17.0 million, or basic and
diluted net loss per share attributable to common stockholders of
$0.35, for the fourth quarter of 2019, as compared to a net loss of
$12.9 million, or basic and diluted net loss per share attributable
to common stockholders of $0.52, for the same period in
2018. The Company reported a net loss of $60.5 million,
or basic and diluted net loss per share attributable to common
stockholders of $1.54, compared to a net loss of $52.0 million,
or basic and diluted net loss per share attributable to common
stockholders of $2.11, for the year ended December 31, 2018.
About OV101 (gaboxadol)Gaboxadol is believed to
be the only delta (δ)-selective GABAA receptor agonist in
development and the first investigational drug to specifically
target the disruption of tonic inhibition, a central physiological
process of the brain that is thought to be the underlying cause of
certain neurodevelopmental disorders. Gaboxadol has been
demonstrated in laboratory studies and animal models to selectively
activate the δ-subunit of GABAA receptors, which are found in the
extrasynaptic space (outside of the synapse), and thereby impact
neuronal activity through modulation of tonic inhibition.
Ovid is developing gaboxadol for the treatment of Angelman
syndrome and Fragile X syndrome to potentially restore tonic
inhibition and thereby address several core symptoms of these
disorders. In both these syndromes, the underlying pathophysiology
includes disruption of tonic inhibition modulated through the
δ-subunit of GABAA receptors. In preclinical studies, it was
observed that gaboxadol improved symptoms of Angelman syndrome and
Fragile X syndrome. This compound has also previously been tested
in more than 4,000 patients (more than 1,000 patient-years of
exposure) and was observed to have favorable safety and
bioavailability profiles. Ovid is conducting a pivotal Phase 3
clinical trial in Angelman syndrome (NEPTUNE) as well as a Phase 2
signal-finding clinical trial in Fragile X syndrome (ROCKET).
The FDA has granted Orphan Drug and Fast Track designations for
gaboxadol for both the treatment of Angelman syndrome and Fragile X
syndrome. The European Commission (EC) has granted orphan drug
designation to gaboxadol for the treatment of Angelman syndrome.
The U.S. Patent and Trademark Office has granted Ovid patents
directed to methods of treating Angelman syndrome and Fragile X
syndrome using gaboxadol. The issued patents expire in 2035 without
regulatory extensions.
About OV935/TAK935 (soticlestat)Soticlestat is
a potent, highly-selective, first-in-class inhibitor of the enzyme
cholesterol 24-hydroxylase (CH24H), with the potential to reduce
seizure susceptibility and improve seizure control. CH24H is
predominantly expressed in the brain, where it converts cholesterol
into 24S-hydroxycholesterol (24HC) to adjust the homeostatic
balance of brain cholesterol. 24HC is a positive allosteric
modulator of the NMDA receptor and modulates glutamatergic
signaling associated with epilepsy. Glutamate is one of the main
neurotransmitters in the brain and has been shown to play a role in
the initiation and spread of seizure activity. Recent literature
indicates that CH24H is involved in over-activation of the
glutamatergic pathway through modulation of the NMDA channel and
that increased expression of CH24H can disrupt the reuptake of
glutamate by astrocytes, resulting in epileptogenesis and
neurotoxicity. Inhibition of CH24H by soticlestat reduces the
neuronal levels of 24HC and may improve excitatory/inhibitory
balance of NMDA channel activity. To Ovid’s knowledge, soticlestat
is the only molecule with this mechanism of action in clinical
development as an anti-epileptic drug (AED).
Ovid and Takeda are conducting a comprehensive Phase 2 clinical
development program with soticlestat in people with Developmental
and Epileptic Encephalopathies (DEE), a heterogeneous group of rare
highly-refractory epilepsy syndromes that encompasses Dravet
syndrome, Lennox-Gastaut syndrome and others. The FDA has granted
orphan drug designation to soticlestat for the treatment of both
Dravet syndrome and Lennox-Gastaut syndrome.
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. The company’s
most advanced investigational medicine, OV101 (gaboxadol), is
currently in clinical development for the treatment of Angelman
syndrome and Fragile X syndrome. Ovid is also developing OV935
(soticlestat) in collaboration with Takeda Pharmaceutical
Company Limited for the potential treatment of rare developmental
and epileptic encephalopathies (DEE). For more information on Ovid,
please visit http://www.ovidrx.com/.
Forward-Looking Statements This press release
includes certain disclosures that contain “forward-looking
statements,” including, without limitation, statements regarding:
advancing and commercializing Ovid’s product candidates, progress,
timing, scope and the potential therapeutic benefits based on
results of clinical trials for Ovid’s product candidates; and the
anticipated reporting schedule of clinical data regarding Ovid’s
product candidates. You can identify forward-looking statements
because they contain words such as “will,” “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”. Ovid
assumes no obligation to update any forward-looking statements
contained herein to reflect any change in expectations, even as new
information becomes available.
Condensed Consolidated Statements of
Operations (Unaudited)
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
|
2019 |
|
|
|
2018 |
|
|
|
2019 |
|
|
|
2018 |
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development |
$ |
12,105,209 |
|
|
$ |
8,621,585 |
|
|
$ |
42,157,641 |
|
|
$ |
33,790,031 |
|
General and
administrative |
|
5,162,720 |
|
|
|
4,504,711 |
|
|
|
19,251,826 |
|
|
|
19,141,652 |
|
Total operating expenses |
|
17,267,929 |
|
|
|
13,126,296 |
|
|
|
61,409,467 |
|
|
|
52,931,683 |
|
Loss from operations |
|
(17,267,929 |
) |
|
|
(13,126,296 |
) |
|
|
(61,409,467 |
) |
|
|
(52,931,683 |
) |
Interest income |
|
298,720 |
|
|
|
226,364 |
|
|
|
948,224 |
|
|
|
952,073 |
|
Net loss and comprehensive
loss |
$ |
(16,969,209 |
) |
|
$ |
(12,899,932 |
) |
|
$ |
(60,461,243 |
) |
|
$ |
(51,979,610 |
) |
Net loss attributable to
common stockholders |
$ |
(16,969,209 |
) |
|
$ |
(12,899,932 |
) |
|
$ |
(60,461,243 |
) |
|
$ |
(51,979,610 |
) |
Net loss per share
attributable to common stockholders, basic and diluted |
$ |
(0.35 |
) |
|
$ |
(0.52 |
) |
|
$ |
(1.54 |
) |
|
$ |
(2.11 |
) |
Weighted-average common shares outstanding basic and
diluted |
|
49,142,504 |
|
|
|
24,635,038 |
|
|
|
39,217,223 |
|
|
|
24,631,011 |
|
Selected Condensed Balance Sheet Data
(Unaudited)
|
December 31, |
December 31, |
|
2019 |
2018 |
|
|
|
Cash, cash equivalents and short-term investments |
$ |
76,739,113 |
$ |
41,500,652 |
Working capital1 |
$ |
69,279,584 |
$ |
35,423,690 |
Total assets |
$ |
80,843,731 |
$ |
47,649,602 |
Total stockholders'
equity |
$ |
70,023,561 |
$ |
38,805,145 |
1Working capital defined as current assets less
current liabilities
Contacts
Investors and Media:Ovid Therapeutics
Inc.Investor Relations & Public Relationsirpr@ovidrx.com
Or
Investors: Steve KlassBurns McClellan,
Inc.sklass@burnsmc.com (212) 213-0006
Media: Katie Engleman 1AB katie@1abmedia.com
(919) 333-7722
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