Miércoles 22 de Noviembre 2017

Takeda and Ovid Therapeutics Announce Innovative Clinical Development and Commercialization Collaboration for TAK-935 in Rare Pediatric Epilepsies

Collaboration Between Big Pharma and Small Biotech Underscores
Potential for New Approaches to Partnering

Ovid and Takeda Will Share Equally in Building on the Discovery from
the Laboratories of Takeda, Bringing Together Capabilities of Both
Companies in Development, Regulatory and Commercialization Activities

OSAKA, Japan & NEW YORK–(BUSINESS WIRE)–Takeda Pharmaceutical Company Limited (TSE: 4502) and Ovid Therapeutics
Inc., a privately-held biopharmaceutical company committed to developing
medicines that transform the lives of people with rare neurological
diseases, today announced the formation of a global collaboration
focused on the clinical development and commercialization of Takeda’s
investigational new drug TAK-935, a novel, potent and highly selective
CH24H inhibitor, in rare pediatric epilepsies. TAK-935 has successfully
completed Phase 1 clinical development under Takeda’s leadership and
will be moving into Phase 1b/2a clinical studies in rare epileptic
encephalopathies where patients continue to suffer from significant
unmet medical needs.

Innovative Structure and Terms of Collaboration

Under the terms of the agreement, Takeda received equity in Ovid and may
be eligible to receive certain milestone payments based on the
advancement of TAK-935. The companies will share in the development and
commercialization costs on a 50/50 basis and, if successful, the
companies will share in the profits on a 50/50 basis. Takeda will lead
commercialization in Japan, and has the option to lead in Asia and other
selected geographies. Ovid will lead clinical development activities and
commercialization of TAK-935 in the United States, Europe, Canada and
Israel. All activities of the collaboration regarding TAK-935 will be
guided by the Takeda/Ovid “One Team” concept, an integrated and
interdisciplinary team from both companies devoted to the successful
advancement of TAK-935 across rare epilepsy syndromes. If mutually
agreed, additional orphan central nervous system indications may also be
pursued. Additional financial details were not disclosed.

“Ovid’s agility, exclusive focus on developing therapies for rare
neurological diseases and specialized capabilities in central nervous
system drug development are highly differentiated and well suited to
this important program,” said Emiliangelo Ratti, head of the central
nervous system therapeutic area for Takeda Pharmaceuticals. “Takeda is
driven by the urgent need to provide novel medicines for people with
psychiatric, neurological and rare central nervous system disorders for
whom there are no treatments available. This agreement is a prime
example of our commitment to partnering select development programs with
prominent companies that will enable us to remain at the leading edge of
innovation.”

Clinical Development Strategy

The companies expect to initiate a Phase 1b/2a study in 2017 in patients
with rare epileptic encephalopathies including Dravet syndrome,
Lennox-Gastaut syndrome and Tuberous Sclerosis Complex. These rare
epilepsies often present in infancy and cause significant morbidities
for patients and their families throughout their lives. Despite the
availability of medicines for epilepsy, there are few treatment options
for these specific disorders, creating a significant medical need for
the development of novel therapies.

“Working together with Takeda we believe we can build on the strengths
and interests of both companies. This is a creative alliance between a
biotechnology and pharmaceutical company where not only do we both share
the passion and commitment to develop meaningful medicines that may
improve the lives of patients worldwide but also we are able to unlock
value in both companies’ pipelines and talent,” said Jeremy Levin,
DPhil, MB BChir, chairman and chief executive officer of Ovid
Therapeutics. “This alliance advances our strategy to become a leader in
the rare neurological disorders field. Building on our work with OV101
in Angelman and Fragile X syndromes, the collaboration in rare
epilepsies extends our ability to help patient communities who face
neurological conditions with limited to no therapeutic options.”

About TAK-935

TAK-935, which is being studied in rare pediatric epilepsies, is a
potent, highly-selective, first-in-class inhibitor of the enzyme
cholesterol 24-hydroxylase (CH24H). CH24H is predominantly expressed in
the brain, where it plays a central role in cholesterol homeostasis.
CH24H converts cholesterol to 24-S-hydroxycholesterol (24HC) which then
exits the brain into the blood plasma circulation.i 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.ii
Recent literature indicates CH24H is involved in over-activation of the
glutamatergic pathway through modulation of the NMDA channel,iii
implying its potential role in CNS diseases such as epilepsy. To our
knowledge, TAK-935 is the only molecule with this mechanism of action in
clinical development.

TAK-935 has been tested in preclinical models to provide data to support
the advancement of the drug into human clinical studies in patients
suffering from rare epilepsy syndromes. A novel proprietary PET ligand,
developed by Takeda and Molecular Neuroimaging, LLC (MNI), has been used
to determine target occupancy of TAK-935 in the brain.iv In
addition, TAK-935’s effect in the brain has been measured from the
change in the plasma concentration of 24HC.

TAK-935 has completed four Phase 1 clinical studiesv,vi,vii,viii
which have assessed tolerability and target engagement at doses which
are believed to be therapeutically relevant.

About Takeda Pharmaceutical Company

Takeda Pharmaceutical Company Limited is a global, research and
development-driven pharmaceutical company committed to bringing better
health and a brighter future to patients by translating science into
life-changing medicines. Takeda focuses its R&D efforts on oncology,
gastroenterology and central nervous system therapeutic areas plus
vaccines. Takeda conducts R&D both internally and with partners to stay
at the leading edge of innovation. New innovative products, especially
in oncology and gastroenterology, as well as Takeda’s presence in
Emerging Markets, are currently fueling the growth of Takeda. More than
30,000 Takeda employees are committed to improving quality of life for
patients, working with Takeda’s partners in health care in more than 70
countries. For more information, visit http://www.takeda.com/news.

About Ovid Therapeutics

Ovid Therapeutics Inc. is a privately-held, New York-based,
biopharmaceutical company using its BoldMedicineTM approach
to develop therapies that transform the lives of patients with rare
neurological diseases. Ovid’s lead product candidate, OV101, is
currently in development for the treatment of symptoms of Angelman
syndrome and Fragile X syndrome.

For more information, visit http://www.ovidrx.com/.

Takeda’s Forward-Looking Statements

This press release contains “forward-looking statements.”
Forward-looking statements include all statements other than statements
of historical fact, including plans, strategies and expectations for the
future, statements regarding the expected timing of filings and
approvals relating to the transaction, the expected timing of the
completion of the transaction, the ability to complete the transaction
or to satisfy the various closing conditions, future revenues and
profitability from or growth or any assumptions underlying any of the
foregoing. Statements made in the future tense, and words such as
“anticipate,” “expect,” “project,” “continue,” “believe,” “plan,”
“estimate,” “pro forma,” “intend,” “potential,” “target,” “forecast,”
“guidance,” “outlook,” “seek,” “assume,” “will,” “may,” “should,” and
similar expressions are intended to qualify as forward-looking
statements. Forward-looking statements are based on estimates and
assumptions made by management that are believed to be reasonable,
though they are inherently uncertain and difficult to predict. Investors
and security holders are cautioned not to place undue reliance on these
forward-looking statements.

Forward-looking statements involve risks and uncertainties that could
cause actual results or experience to differ materially from that
expressed or implied by the forward-looking statements. Some of these
risks and uncertainties include, but are not limited to: required
regulatory approvals for the transaction may not be obtained in a timely
manner, if at all; the conditions to closing of the transaction may not
be satisfied; competitive pressures and developments; applicable laws
and regulations; the success or failure of product development programs;
actions of regulatory authorities and the timing thereof; changes in
exchange rates; and claims or concerns regarding the safety or efficacy
of marketed products or product candidates in development.

The forward-looking statements contained in this press release speak
only as of the date of this press release, and neither Ovid nor Takeda
undertake any obligation to revise or update any forward-looking
statements to reflect new information, future events or circumstances
after the date of the forward-looking statement. If one or more of these
statements is updated or corrected, investors and others should not
conclude that additional updates or corrections will be made.

Ovid’s Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements. Forward-looking
statements contained in this press release include, without limitation,
statements regarding the potential use of TAK-935 to treat epilepsy and
various central nervous system indications, the scope and timing of the
clinical development of TAK-935 and Ovid’s potential payment of
milestone payments. Words such as “may,” “believe,” “will,” “expect” and
similar expressions (as well as other words or expressions referencing
future events, conditions or circumstances) are intended to identify
forward-looking statements. These forward-looking statements are not
guarantees of future performance and involve a number of unknown risks,
assumptions, uncertainties and factors that are beyond Ovid’s control.
All forward-looking statements are based on Ovid’s expectations and
assumptions as of the date of this press release. Actual results may
differ materially from these forward-looking statements. Except as
required by law, Ovid expressly disclaims any responsibility to update
any forward-looking statement contained herein, whether as a result of
new information, future events or otherwise.

i Russell DW, Halford RW, Ramirez DMO, Shah R, Kotti T.
Cholesterol 24-Hydroxylase: An Enzyme of Cholesterol Turnover in the
Brain. Annu Rev Biochem. 2009; 78: 1017–1040.

ii Mehta A, Prabhakar M, Kumar P, Deshmukh R, Sharma PL.
Excitotoxicity: bridge to various triggers in neurodegenerative
disorders. Eur J Pharmacol 2013;698(1-3):6-18.

iii Paul SM, Doherty JJ, Robichaud AJ, Belfort GM, Chow BY,
Hammond RS, et al. The major brain cholesterol metabolite
24(S)-hydroxycholesterol is a potent allosteric modulator of
N-methyl-D-aspartate receptors. J Neurosci 2013;33(44):17290-300.

iv https://www.clinicaltrials.gov/ct2/show/NCT02497235?term=TAK-935&rank=1

v https://www.clinicaltrials.gov/ct2/show/NCT02497235?term=TAK-935&rank=1

vi https://www.clinicaltrials.gov/ct2/show/NCT02906813?term=TAK-935&rank=2

vii https://www.clinicaltrials.gov/ct2/show/NCT02201056?term=TAK-935&rank=3

viii https://www.clinicaltrials.gov/ct2/show/NCT02539134?term=TAK-935&rank=4

Contacts

Ovid Contacts:
Investors:
Burns McClellan
Steve
Klass
Sklass@burnsmc.com
+1-212-213-0006
or
Media:
Pure
Communications, Inc.
Katie Engleman
katie@purecommunicationsinc.com
+1-910-509-3977
or
Takeda
Contacts:

Tsuyoshi Tada – Japan
tsuyoshi.tada@takeda.com
+81-3-3278-2417
or
Julia
Ellwanger – USA
julia.ellwanger@takeda.com
+1-224-554-7681