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Invitae’s Real-World Ciitizen Data Utilized in Praxis Precision Medicines’ PRAX-222 IND Filing
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:
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"First use of Ciitizen platform as source of real-world data in regulatory filing\nSAN FRANCISCO and BOSTON, Sept. 20, 2022 (GLOBE NEWSWIRE) -- 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 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 ...