Praxis Precision Medicines Partners With Ciitizen to Improve Patient-Guided Drug Development

Rhea-AI Summary

Praxis Precision Medicines has announced a collaboration with Ciitizen to improve drug development for SCN2A developmental and epileptic encephalopathy (SCN2A-DEE). The partnership aims to leverage real-world data through the Ciitizen platform for enhanced clinical trial designs and biomarker identification. Both PRAX-222 and PRAX-562 have received rare pediatric disease and orphan drug designations from the FDA. PRAX-562 is currently in a Phase 1 clinical trial, while PRAX-222 is in IND-enabling studies. This collaboration underscores Praxis' commitment to patient-centered approaches.

Positive

• Collaboration with Ciitizen to enhance real-world data analysis for drug development.
• PRAX-222 and PRAX-562 have received rare pediatric disease designations and orphan drug status from the FDA.
• Potential for accelerated drug development process and improved clinical trial designs.

Negative

• None.

Innovative collaboration to be implemented across multiple indications starting with SCN2A-DEE

CAMBRIDGE, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal imbalance, today announced a collaboration with Ciitizen, a Palo Alto based healthcare technology company that helps patients get full control of their medical records so they can find better treatment options, participate in research and find clinical trials. Praxis will utilize the Ciitizen platform to provide deeper insights across disease populations through the analysis of real-world data, initially to inform the development of PRAX-222 and PRAX-562 in SCN2A developmental and epileptic encephalopathy (SCN2A-DEE).

“As a patient-guided company, this partnership with Ciitizen is well aligned with Praxis’ core principles,” said Marcio Souza, president and chief executive officer of Praxis. “By integrating the Ciitizen platform, we expect to improve and accelerate the drug development process through more informed clinical trial designs and endpoints, potentially getting new therapies to patients more efficiently. We also see its potential to inform key biomarkers and add to our understanding of the genetics underlying specific disorders through genotype-phenotype analysis.”

“Our partnership with Praxis is designed to help guide the development of innovative therapies and deliver a holistic view of critical health data to patients, caregivers and their networks of doctors and providers,” said Anil Sethi, chief executive officer of Ciitizen. “Enabling fully informed treatment decisions can benefit patients, and we can continue to improve care through additional insights into disease signs, symptoms and progression over time.”

Patients in the U.S. with SCN2A-related disorders can sign up to participate in this collaboration at https://www.ciitizen.com/scn2a/. Within 30 days of signing up to the Ciitizen platform, a comprehensive view of the patient’s medical records, pharmacy records, genomic data and imaging data will be made available for the caregiver and patient to use at their discretion. There is no cost to sign up and no onsite clinical visits are required. Patients will retain exclusive ownership of all the data. With patient consent, aggregated data will be provided to Praxis.

For additional information about SCN2A-related disorders, please visit https://scn2a.com/.

PRAX-222 and PRAX-562 have been granted rare pediatric disease designation and PRAX-222 has been granted orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of SCN2A-DEE. PRAX-562 is currently being evaluated in a Phase 1 clinical trial in adult healthy volunteers and PRAX-222 is currently being evaluated in IND-enabling studies.

About SCN2A-DEE
SCN2A-DEE is a rare developmental and epileptic encephalopathy caused by a variant in the SCN2A gene. The SCN2A gene is critical in the formation of sodium channel proteins in the brain, which control the flow of sodium ions into neurons. This movement of sodium ions is a major component of generating electrical signals called action potentials, the way in which the cells communicate. Patients suffer from recurrent, typically drug-resistant seizures which start as early as the first day of life. The seizures can be of multiple different types, up to dozens per day, with poor response to current treatment options. Patients with SCN2A-DEE may have moderate to severe cognitive and developmental impairments, other neurological disorders such as dystonia and chorea, and disturbances in other organ systems including gastrointestinal function.  

About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance. Praxis is applying insights into the genetic mutations that drive excitation-inhibition imbalance in diseases to select biological targets for severe pediatric epilepsies and more broadly for prevalent psychiatric diseases and neurologic disorders. Praxis has established a broad portfolio, including five disclosed programs across multiple central nervous system disorders including depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates.

About Ciitizen
Ciitizen is a leading consumer health technology company whose mission is to help patients get full control of their medical records so they can find better treatment options, participate in research and find clinical trials. As a patient-directed care platform, Ciitizen believes patients should have full control of directing the use of their data, which serves as one of the richest sources of real world evidence, unlocking new options for patients and the community.  Please see https://www.ciitizen.com/research to learn more.

FAQ

What is the significance of the collaboration between Praxis and Ciitizen?

The collaboration aims to leverage real-world data to enhance drug development processes, particularly for SCN2A-DEE therapies.

What designations have PRAX-222 and PRAX-562 received?

Both PRAX-222 and PRAX-562 have been granted rare pediatric disease designation and orphan drug designation by the FDA.

What stage is PRAX-562 currently in?

PRAX-562 is currently undergoing a Phase 1 clinical trial in adult healthy volunteers.

How will the Ciitizen platform be utilized in this collaboration?

The Ciitizen platform will provide insights into disease populations and aid in informed clinical trial designs and endpoints.

What is SCN2A-DEE?

SCN2A-DEE is a rare neurological condition caused by a variant in the SCN2A gene, leading to severe, drug-resistant seizures and developmental impairments.