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– First use of the Ciitizen platform as a source of real-world data in the regulatory repository –
SAN FRANCISCO and BOSTON, September 20, 2022 /PRNewswire/ — Guests ( New York stock market : NVTA) and Praxis Precision Medicines, Inc. (NASDAQ: PRAX), today announced that real-world clinical information from Invitee’s Ciitizen platform was used as natural history data to support the submission of the application. Praxis’ investigational new drug (IND) for PRAX-222 for the treatment of pediatric patients with early-onset SCN2A developmental and epileptic encephalopathy (DEE). Practice announcement earlier this month, the US Food and Drug Administration (FDA) cleared the IND application for the starting dose cohort for the PRAX-222 EMBRAVE clinical study.
For many well-established diseases, natural history studies and other rich datasets are available to support regulatory interactions and IND applications. For many rare diseases, including some serious genetic pediatric epilepsies such as SCN2A-DEE, natural history studies are not yet available to document the high disease burden and high unmet medical need. Additionally, the usual method of collecting this data by having patients seen in many geographically dispersed sites is expensive, time-consuming, and not well suited for rare diseases. Invitee’s Ciitizen platform enables rapid and comprehensive collection and analysis of medical history data, which helps to understand patient population and disease severity, can be used as natural history data for regulatory submissions and can inform protocol design and inclusion and exclusion criteria for clinical studies. studies.
“The comprehensive real-world clinical evidence generated by Invitee’s Ciitizen platform was a critical component of the PRAX-222 IND application, integrating a significant amount of natural history data in a highly efficient manner to help bring PRAX closer together. -222 of SCN2A- DEE patients,” said Steven Petrou, Ph.D., co-founder and scientific director of Praxis. “In order to make real progress and offer hope to patients living with SCN2A-DEE and their caregivers, it requires an engaged ecosystem and a community ready to consider innovative approaches to drug development. look forward to our continued partnership with Invitae for PRAX-222 and 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 anonymized and shared with their consent, and represents the richest aggregation of real clinical evidence for SCN2A-DEE patients. The data generated by Invitee’s Ciitizen platform is comprehensive, leveraging HIPAA right of access to bring together complete medical records, longitudinally, from all patient care sites. This approach addresses many of the limitations of other data sources, such as anonymized electronic medical records from providers and claims data, that have been 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, 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 dataset underlying this natural history study provides a unique model for data collection and sharing in which patients, patient advocacy groups, and other researchers have all access to collected data. Patients have full access to records for their own use and can also stay involved and informed about the research throughout the study, underscoring the benefits of this unique patient-centered research model. “This is, to my knowledge, the first example of a patient medical records platform being used as the primary source of natural history data for a successful IND filing with the FDA,” said Alexandra Berk, director of medical affairs at Invitae. “This is an important development in terms of the FDA’s openness to including new data sources as part of the regulatory process.”
“We are excited to partner with Praxis and see how the high-quality data from our Ciitizen platform can advance the needle for patients living with this rare genetic epilepsy,” said Robert Nussbaum, MD, Chief Medical Officer of Invitee. “Our goal is to advance the understanding of SCN2A-DEE and other diseases and use this data to bring new therapies to market faster. rare diseases, essential data for regulatory processes and the development of 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 Invitee’s Ciitizen platform or to request access to consented real-world data for research purposes, email [email protected]
About PRAX-222
PRAX-222 is an ASO designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-onset SCN2A-DEE to treat seizures and other symptoms in patients with SCN2A mutations at gain of function. In vitro studies of PRAX-222 have demonstrated a reduction in both SCN2A gene expression and protein levels. In vivo, PRAX-222 demonstrated a significant, dose-dependent reduction in seizures, improved behavioral and locomotor activity, and increased survival in SCN2A mouse models, with the potential to be the first modifying treatment of the disease. 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 in a collaboration with Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) and RogCon, Inc. To learn more about SCN2A-DEE and the EMBRAVE study, please visit https:/ /scn2a.com/.
About Invite
About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic knowledge into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis applies insights from genetic epilepsies to rare and more prevalent neurological disorders, using our understanding of shared biological targets and circuits in the brain. Praxis has established a broad pipeline with multiple programs, including product candidates in movement disorders, epilepsy and psychiatric disorders, with four clinical-stage product candidates. For more information, please visitwww.praxismedicines.com and follow us onLinkedIn andTwitter.
Invitee 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 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 from those reported and should not be relied upon as an indication of future performance. These risks and uncertainties include, but are not limited to: the company’s loss history; the company’s ability to be competitive; the company’s inability to effectively manage growth; the company’s need to evolve its infrastructure in anticipation of demand for its testing and to increase demand for its testing; the company’s ability to use rapidly changing genetic data to interpret test results accurately and consistently; security breaches, data loss and other disruptions; the laws and regulations applicable to the company’s activity; and other risks disclosed in the company’s filings with the Securities and Exchange Commission, including the risks disclosed 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: the 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 relating to Praxis’ programs and operations, as described in its Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 and other documents filed with the Securities and Exchange Commission. Although Praxis’ forward-looking statements reflect the good faith judgment of its management, such statements are based solely on information and factors currently known to Praxis. Accordingly, 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.
Guest contact:
Amy Hadsock
[email protected]
628-213-3283
Praxis Precision Medicines Contact:
Alex Kane
[email protected]
617-300-8481
SOURCE Invite Corporation
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