DGAP-News: Centogene NV / Key word(s): Agreement
CENTOGENE and Alnylam Pharmaceuticals Launch a New Clinical Program Aimed at
Revolutionizing the Diagnosis of Hereditary Transthyretin-Related
Amyloidosis ('ATTRv')
2020-12-08 / 12:30
The issuer is solely responsible for the content of this announcement.
*CENTOGENE and Alnylam Pharmaceuticals Launch a New Clinical Program Aimed
at Revolutionizing the Diagnosis of Hereditary Transthyretin-Related
Amyloidosis ("ATTRv")*
*CAMBRIDGE, Mass. and ROSTOCK, Germany, and BERLIN and MUNICH, Germany,
December 08, 2020* - Centogene N.V. (Nasdaq: CNTG), a commercial-stage
company focused on rare diseases that transforms real-world clinical and
genetic data into actionable information for patients, physicians and
pharmaceutical companies, and Alnylam Pharmaceuticals, Inc. ("*Alnylam*"),
the leading RNAi therapeutics company, announced today the launch of a new
joint clinical screening program: the genetic screening of the at-risk
population for hereditary *T*ransthy*R*etin-related *AM*yloidosis and
longitudinal *moni*toring of *TTR*positive subjects (the "TRAMmoni_TTR_
Study").
The new TRAMmoni_TTR_ Study is a follow-up to the preceding epidemiological
analysis for hereditary *T*ransthy*R*etin-related *AM*yloidosis (the "*TRAM*
*Study*" (NCT03237494)). Since 2017, 5,000 participants from Germany,
Austria and Switzerland with polyneuropathy and/or cardiomyopathy of no
obvious etiology have been screened for ATTRv as part of the TRAM Study.
More than 1% of participants were eventually diagnosed with ATTRv,
clinically characterized and regionally mapped.
The follow-up TRAMmoni_TTR _Study will include both symptomatic and
asymptomatic _TTR_ positive participants, who will be invited to take part
in the longitudinal phase to monitor their clinical status. Using
CENTOGENE's metabolomics profiling platform, the Company has discovered and
now characterizes novel ATTRv biomarkers. Such molecules are crucial to
accelerate ATTRv diagnostics and treatment personalization. The monitoring
of these biomarkers in _TTR_ positive participants will support the
validation process.
Prof. Peter Bauer, Chief Genomic Officer of CENTOGENE, said, "This is the
first study where both symptomatic and asymptomatic _TTR _positive
participants will be monitored for two years. This will allow us to validate
our ATTRv biomarkers and later utilize them for treatment
individualization."
Dr. Volha Skrahina, Director of Clinical Studies at CENTOGENE, added,
"Within the initialTRAM Study, we were able to deliver truly valuable
insights. More than 50 participants suffering from the known and treatable
disease received the diagnosis. We will now proceed with the screening in
TRAMmoni_TTR_ in order to accelerate the diagnosis for those suffering and
awaiting answers. This is crucial due to the progressive nature of the
disease."
"We are excited about expanding our collaboration with CENTOGENE in its
epidemiology and biomarker work through the initiation of a new clinical
program (TRAMmoni_TTR_) focused on Hereditary Transthyretin-Related
Amyloidosis," said Dr. Bernhard Kaumanns, VP Medical Affairs CEMEA (Canada,
Europe, Middle East & Africa) at Alnylam. "This program will help to better
understand diagnostic pathways and identify possible biomarkers to
accelerate the diagnosis of this devastating disease. Equally important is
the patient follow-up that this program provides; long term evidence-based
data will be generated to improve the understanding of this disease under
conditions of daily clinical practice."
*About Hereditary Transthyretin-Related Amyloidosis ("ATTRv")*
ATTRv is an autosomal dominant condition caused by a pathogenic variant in
the _TTR_ gene (Plante-Bordeneuve _et al._ 2011). The _TTR _gene is coding
for transthyretin, formerly known as prealbumin. Transthyretin ("*Ttr*") is
found primarily in the serum (secreted by the liver) and the cerebrospinal
fluid (secreted by the choroid plexus), and functions as a carrier for the
hormone thyroxine (T4) and retinol-binding protein (bound to retinol or
vitamin A). The destabilization of the Ttr protein and the formation of
misfolded Ttr proteins results in the transthyretin amyloidosis diseases.
There are more than 150 _TTR _pathogenic variants described; the disease
phenotype is _TTR_ variant dependent (Ando _et al_. 2013, Rowczenio _et al_.
2014, Rowczenio _et al_. 2015).
On average, the diagnosis is delayed by 4-5 years, especially in non-endemic
areas. Also, there is potential for misdiagnosis due to ATTRv's clinical
heterogeneity (Adams _et al_., 2016). The common misdiagnosis of ATTRv with
polyneuropathy includes chronic inflammatory demyelinating
polyradiculoneuropathy, idiopathic axonal polyneuropathy, lumbar spinal
stenosis and, more rarely, diabetic neuropathy and AL amyloidosis (Koike _et
al_., 2011; Adams _et al_., 2019).
In order to facilitate an early diagnosis, treatment choice and
individualization, ATTRv biomarkers are critical.
*About CENTOGENE *
CENTOGENE engages in diagnosis and research around rare diseases
transforming real-world clinical and genetic data into actionable
information for patients, physicians and pharmaceutical companies. Our goal
is to bring rationality to treatment decisions and to accelerate the
development of new orphan drugs by using our extensive rare disease
knowledge, including epidemiological and clinical data, as well as
innovative biomarkers. CENTOGENE has developed a global proprietary rare
disease platform based on our real-world data repository with over 3.6
billion weighted data points from approximately 570,000 patients
representing over 120 different countries as of August 31, 2020.
The Company's platform includes epidemiologic, phenotypic and genetic data
that reflects a global population, and also a biobank of these patients'
blood samples. CENTOGENE believes this represents the only platform that
comprehensively analyzes multi-level data to improve the understanding of
rare hereditary diseases, which can aid in the identification of patients
and improve our pharmaceutical partners' ability to bring orphan drugs to
the market. As of August 31, 2020, the Company collaborated with over 40
pharmaceutical partners covering over 45 different rare diseases.
*About Alnylam Pharmaceuticals*
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference
("*RNAi*") into a whole new class of innovative medicines with the potential
to transform the lives of people afflicted with rare genetic,
cardio-metabolic, hepatic infectious, and central nervous system/ocular
diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent
a powerful, clinically validated approach for the treatment of a wide range
of severe and debilitating diseases. Founded in 2002, Alnylam is delivering
on a bold vision to turn scientific possibility into reality, with a robust
RNAi therapeutics platform. Alnylam's commercial RNAi therapeutic products
are ONPATTRO(R) (patisiran), approved in the U.S., EU, Canada, Japan, Brazil
and Switzerland, and GIVLAARI(R) (givosiran), approved in the U.S., EU,
Brazil and Canada, and OXLUMO(TM) (lumasiran) approved in the U.S. and the
EU. Alnylam has a deep pipeline of investigational medicines, including six
product candidates that are in late-stage development. Alnylam is
headquartered in Cambridge, MA.
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declines, any obligation to update any such statements or to publicly (MORE TO FOLLOW) Dow Jones Newswires
December 08, 2020 06:30 ET (11:30 GMT)
