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Medidata Rave Omics Collaboration Uncovers Novel Insights for Rare Disease Research

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Discovery of biomarker evidence in Castleman Disease exemplifies the
value of personalized medicine in rare diseases

  • The Castleman Disease Collaborative Network presented new research
    supported by Medidata at 60th Annual Meeting of the American Society
    of Hematology
  • Application of Medidata Rave Omics reveals new patient subgroups
    that can accelerate diagnosis and treatment
  • Medidata data analysis in collaboration with the Castleman Disease
    Collaborative Network advances personalized medicine for
    life-threatening disorder

The Castleman Disease Collaborative Network (CDCN) discovered new
patient subgroups, based on previously unknown proteomic signatures,
with Medidata's Rave Omics, a machine learning-based solution. These
discoveries provide novel insights into treatment response and potential
new drug targets, highlighting the value of precision medicine.

Medidata (NASDAQ:MDSO) and the CDCN presented
these insights at the 60th Annual Meeting of the American Society of
Hematology (ASH).

Idiopathic Multicentric Castleman Disease (iMCD) is a rare, difficult to
diagnose, life-threatening disorder.1 The CDCN advances
research programs to develop better diagnostic methods, identify
patients that will respond to approved therapy and find new drug targets
to develop new therapies.

"iMCD stumped my doctors, and they didn't think I would survive. My
mission today is to bring new treatment options and hope to Castleman
Disease patients and other poorly understood rare diseases," said Dr.
David Fajgenbaum, co-founder and executive director of the Castleman
Disease Collaborative Network. "Rare diseases often lack sufficient
sample sizes and necessary resources to make critical discoveries, which
has limited the development of new treatment options for patients. This
collaborative study combined patient samples from around the world and
the Rave Omics tool to overcome these challenges and help to better
understand this disease. We are now working together to use this data to
personalize treatment for Castleman disease."

Medidata Rave Omics enabled the discovery of novel biomarkers for
Castleman disease. With unparalleled industry expertise, Medidata data
scientists collaborated with the Castleman Disease Collaborative Network
to make the following insights:

  • Six new patient subsets reflecting either distinct subtypes or
    proteomic disease states
  • Evidence of proteomic predictors of anti-interleukin-6 treatment
    response
  • Etiological insights into the poorly understood rare disease and
    toward new potential drug targets

"Medidata's analytics empower researchers to make new discoveries for
all patients, including those with rare diseases," said Glen de Vries,
co-founder and president, Medidata. "We're proud to help make
personalized medicine and the development of targeted treatments
possible with Rave Omics."

Medidata Rave Omics streamlines omic data capture, linking and analysis
inside the clinical study process. To learn more about Medidata, visit mdsol.com.

1 Fajgenbaum D, Ruth J, Kelleher D, Rubenstein A. Lancet
Haematology. 2016;3:150-152

About Medidata

Medidata is leading the digital transformation of life sciences, with
the world's most used platform for clinical development, commercial, and
real-world data. Powered by artificial intelligence and delivered by the
#1 ranked industry experts, the Intelligent Platform for Life Sciences
helps pharmaceutical, biotech, medical device companies, and academic
researchers accelerate value, minimize risk and optimize outcomes.
Medidata serves more than 1,000 customers and partners worldwide and
empowers more than 100,000 certified users every day to create hope for
millions of patients. Discover the future of life sciences: www.mdsol.com

About Castleman Disease Collaborative Network (CDCN)

CDCN is a global initiative dedicated to accelerating research and
treatment for Castleman disease (CD) to improve survival for all
patients with CD. The CDCN's innovative approach first involved building
a global community of over 400 physicians and researchers, assembling a
scientific advisory board of 28 experts from eight countries, and
supporting and engaging patients in research prioritization. Then, the
CDCN crowdsourced among the global community to identify gaps in medical
knowledge and determine high priority research projects. In parallel,
the CDCN connects and supports thousands of CD patients around the
world. Now, the CDCN recruits top researchers to conduct studies, and
works with patients, loved ones, and the public to raise funding to
enable these studies. More information is available at: www.CDCN.org

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