Ambit^®, a data and technology-enabled biopharma services company, today announced that leadership will debut Ambit's data-driven methodology for projecting prevalence and diagnosis rates for difficult-to-quantify DEEs at the 51^st Annual Child Neurology Society Meeting on Oct. 13, 2022. Ambit's analytics team has developed this methodology in collaboration with Praxis Precision Medicines, Inc. (NASDAQ: PRAX) and the UCSF Weill Institute for Neurosciences and Benioff Children's Hospitals.

For many rare DEEs, reliable prevalence estimates are not available. With a robust and growing pipeline of precision medicine therapies in rare epilepsy and neurodevelopmental disorders, estimating the prevalence of these conditions with a data-driven methodology can be highly valuable to drug developers, medical device companies and other healthcare decision-makers.

"Ambit's expertise in a data-driven approach to prevalence projections for rare neurological conditions has allowed Praxis to characterize DEE populations with a high degree of confidence," said Marcio Souza, president and chief executive officer of Praxis. "As we seek to bring first-in-class, potentially disease-modifying therapies to DEE patients in need, our collaboration with Ambit has been invaluable in increasing our understanding of these patient groups."

Despite the availability of sponsored genetic testing and increased awareness of the benefit of genetic testing, over 50% of patients with rare DEEs remain undiagnosed. Sizing these patient populations and diagnosis rates underscores the importance of earlier genetic testing for unexplained seizure disorders and rare DEEs. "Even today, the path to accurate diagnosis and appropriate care for rare genetic epilepsies can be long and full of challenges," said Adam Numis, M.D., of the UCSF Benioff Children's Hospital. "Ambit's methodology underscores the need for more treatment options to improve outcomes and quality of life for the rare DEE population."

Ambit chief executive officer, Robert Sederman, will present this methodology on Oct. 13 as part of the Platform II Presentation Session (PL2-1) at the 2022 Annual Child Neurology Society Meeting. Presentations will run from 7-9 a.m. ET in the Junior Ballroom CD at the Duke Energy Center. Ambit will be presenting from 7-7:15 a.m. The associated abstract will be published in a supplement to the October issue of Annals of Neurology.

"We are excited to partner with Praxis and UCSF to showcase Ambit's DEE projection methodology at this year's meeting and, ultimately, see the impact of this collaboration on greater access to accurate diagnosis and treatment for patients with rare DEEs in the future," said Sederman.
 

About Ambit Inc.

Ambit Inc.^® is a data and technology-enabled biopharma and healthcare services company with a focus on rare and specialty disease. Ambit's patient identification services drive patient activation and earlier diagnosis for patients that may be suffering from rare diseases. Ambit's analytics and professional services support rare and specialty biopharma across the clinical development and commercialization of potentially life-changing therapies. To learn more about Ambit's DEE Projection approach and other capabilities, please reach out to inquiries@ambitinc.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.