Invitae’s Real-World Ciitizen Data Utilized in Praxis Precision Medicines’ PRAX-222 IND Filing
Praxis Precision Medicines, Inc.
First use of Ciitizen platform as source of real-world data in regulatory filing
SAN FRANCISCO and BOSTON, Sept. 20, 2022 (GLOBE NEWSWIRE) --Invitae(NYSE: NVTA) andPraxis 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). Praxisannouncedearlier 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 diseases. 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 represent 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 the 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 include 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.”
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Invitae Corporation, a medical genetics company, integrates genetic information into mainstream medicine to improve healthcare of people in the United States, Canada, and internationally. The company offers genetic tests in various clinical areas, including hereditary cancer, cardiology, neurology, pediatrics, oncology, metabolic conditions, and rare diseases; digital health solutions; and health data services. It serves patients, healthcare providers, biopharma companies, and other partners. The company was formerly known as Locus Development, Inc. and changed its name to Invitae Corporation in 2012. Invitae Corporation was incorporated in 2010 and is headquartered in San Francisco, California.