– First use of Ciitizen platform as source of
real-world data in regulatory filing –
SAN
FRANCISCO and BOSTON, Sept. 20,
2022 /PRNewswire/ -- Invitae (NYSE: NVTA) and
Praxis Precision Medicines, Inc. (NASDAQ: PRAX), today announced
that real-world clinical insights from Invitae's Ciitizen platform
were utilized as natural history data to support the submission of
Praxis' Investigational New Drug (IND) application for PRAX-222 for
the treatment of pediatric patients with early-onset SCN2A
developmental and epileptic encephalopathy (DEE). Praxis announced
earlier this month that the U.S. Food and Drug Administration (FDA)
cleared the IND application for the initial dose cohort for the
PRAX-222 EMBRAVE clinical study.
For many well-established diseases, natural history studies and
other rich data sets are available to support regulatory
interactions and IND applications. For many rare diseases,
including certain severe genetic pediatric epilepsies such as
SCN2A-DEE, natural history studies are not yet available to
document the high disease burden and significant unmet medical
need. In addition, the usual method of collecting these data by
having patients seen at many geographically dispersed sites is cost
intensive, time consuming and not well suited to rare disorders.
Invitae's Ciitizen platform enables the rapid and comprehensive
collection and analysis of medical history data, which supports
understanding of the patient population and disease severity, may
be used as natural history data for regulatory submissions, and can
inform protocol design and inclusion and exclusion criteria for
clinical studies.
"The comprehensive real-world clinical evidence generated
through Invitae's Ciitizen platform was a critical component to the
PRAX-222 IND application, integrating a significant amount of
natural history data in a highly efficient manner to help bring
PRAX-222 one step closer to SCN2A-DEE patients," said Steven Petrou, Ph.D., co-founder and chief
scientific officer of Praxis. "In order to make real progress and
offer hope to patients living with SCN2A-DEE and their caregivers,
it requires a committed ecosystem and a community willing to
consider innovative approaches to drug development. We look forward
to our continued partnership with Invitae for PRAX-222 and for
other precision medicines targeting rare genetic epilepsies with
high unmet need."
The data, collected on behalf of SCN2A-DEE patients or their
parents/guardians, is de-identified and shared with their consent,
and represents the richest aggregation of real-world clinical
evidence for SCN2A-DEE patients. The data generated by Invitae's
Ciitizen platform is comprehensive, leveraging the HIPAA right of
access to gather full medical records, longitudinally, from all of
the patients' sites of care. This approach addresses many of the
limitations of other data sources, such as de-identified provider
electronic medical record and claims data, that were raised by the
FDA in recent draft guidance documents on the use of real-world
data in regulatory submissions. This novel data collection and
extraction model also addresses many of the logistical, financial
and methodological limitations of site-based natural history
studies, by rapidly enrolling a diverse and representative sample
of patients directly without the need to engage with sites for
recruitment and data collection. Additionally, the real-world data
set underlying this natural history study provides a unique data
collection and sharing model wherein the patients, patient advocacy
groups and other researchers all have access to the data collected.
Patients have complete access to the records for their own use and
are also able to remain involved and informed about the research
throughout the study, highlighting the benefits of this unique
patient-centered research model. "This is, to my knowledge, the
first instance of a patient-mediated medical records platform being
utilized as the primary natural history data source for a
successful IND filing with the FDA," stated Alexandra Berk, medical affairs director at
Invitae. "It's an important development in terms of the FDA's
openness to including novel data sources as part of the regulatory
process."
"We are thrilled to partner with Praxis and see how our Ciitizen
platform's high quality data can move the needle forward for
patients living with this rare, genetic epilepsy," said
Robert Nussbaum, M.D., chief medical
officer of Invitae. "Our goal is to advance the understanding of
SCN2A-DEE and other diseases and utilize these data to bring new
therapies to market faster. Ciitizen is a compelling platform for
rapidly gathering both genotypic and phenotypic data with patient
consent and engagement for natural history studies for rare
diseases, data that are essential to regulatory processes and for
developing new therapies."
Watch a recorded webinar with speakers from Praxis and Invitae
describing this natural history data in more detail here. To learn
more about Invitae's Ciitizen platform or to request access to the
consented real-world data for research, email
partners@invitae.com.
About
PRAX-222
PRAX-222 is an ASO designed to selectively decrease SCN2A gene
expression, directly targeting the underlying cause of
early-seizure-onset SCN2A-DEE to treat seizures and other symptoms
in patients with gain-of-function SCN2A mutations. In-vitro studies
of PRAX-222 have demonstrated reduction in both SCN2A gene
expression and protein levels. In-vivo, PRAX-222 has demonstrated
significant, dose-dependent reduction in seizures, improvement in
behavioral and locomotor activity and increased survival in SCN2A
mouse models, with potential to be the first disease-modifying
treatment. PRAX-222 has received Orphan Drug Designation (ODD) and
Rare Pediatric Disease Designation (RPD) from the FDA, and ODD from
the European Medicines Agency (EMA) for the treatment of SCN2A-DEE.
The PRAX-222 program is ongoing under a collaboration with Ionis
Pharmaceutics, Inc. (NASDAQ: IONS) and RogCon, Inc. To learn more
about SCN2A-DEE and the EMBRAVE study, please visit
https://scn2a.com/.
About Invitae
Invitae Corporation (NYSE: NVTA) is a leading medical genetics
company, whose mission is to bring comprehensive genetic
information into mainstream medicine to improve healthcare for
billions of people. Invitae's goal is to aggregate the world's
genetic tests into a single service with higher quality, faster
turnaround time and lower prices. For more information, visit the
company's website at invitae.com.
About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical
company translating genetic insights into the development of
therapies for CNS disorders characterized by neuronal
excitation-inhibition imbalance. Praxis is applying insights from
genetic epilepsies to both rare and more prevalent neurological
disorders, using our understanding of shared biological targets and
circuits in the brain. Praxis has established a broad portfolio
with multiple programs, including product candidates across
movement disorders, epilepsy and psychiatric disorders, with four
clinical-stage product candidates. For more information, please
visit www.praxismedicines.com and follow us
on LinkedIn and Twitter.
Invitae Forward-Looking
Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, including statements relating to the benefits of the data
generated through the company's Ciitizen platform. Forward-looking
statements are subject to risks and uncertainties that could cause
actual results to differ materially, and reported results should
not be considered as an indication of future performance. These
risks and uncertainties include, but are not limited to: the
company's history of losses; the company's ability to compete; the
company's failure to manage growth effectively; the company's need
to scale its infrastructure in advance of demand for its tests and
to increase demand for its tests; the company's ability to use
rapidly changing genetic data to interpret test results accurately
and consistently; security breaches, loss of data and other
disruptions; laws and regulations applicable to the company's
business; and the other risks set forth in the company's filings
with the Securities and Exchange Commission, including the risks
set forth in the company's Quarterly Report on Form 10-Q for the
quarter ended June 30, 2022. These
forward-looking statements speak only as of the date hereof, and
Invitae Corporation disclaims any obligation to update these
forward-looking statements.
Praxis Precision Medicines
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of 1995
and other federal securities laws, including express or implied
statements regarding Praxis' future expectations, plans and
prospects, including, without limitation, statements regarding the
development of our product candidates, including the design of our
clinical trials and the treatment potential of our product
candidates. The express or implied forward-looking statements
included in this press release are only predictions and are subject
to a number of risks, uncertainties and assumptions, including,
without limitation: uncertainties inherent in clinical trials; the
expected timing of submissions for regulatory approval or review by
governmental authorities; regulatory approvals to conduct trials;
and other risks concerning Praxis' programs and operations as
described in its Quarterly Report on Form 10-Q for the quarter
ended June 30, 2022 and other filings
made with the Securities and Exchange Commission. Although Praxis'
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on information and
factors currently known by Praxis. As a result, you are cautioned
not to rely on these forward-looking statements. Any
forward-looking statement made in this press release speaks only as
of the date on which it is made. Praxis undertakes no obligation to
publicly update or revise any forward-looking statement, whether as
a result of new information, future developments or
otherwise.
Invitae Contact:
Amy Hadsock
pr@invitae.com
628-213-3283
Praxis Precision Medicines Contact:
Alex Kane
investors@praxismedicines.com
617-300-8481
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SOURCE Invitae Corporation