TM-1803/P-PantSAc shows potential in reducing severe outcomes of the
disease; study published in Scientific Reports
LEIDEN, The Netherlands(BUSINESS WIRE)
TM3 Therapeutics, a biopharmaceutical company focused on developing
metabolite replacement therapies for rare, genetic neurological
diseases, today announced the publication of novel preclinical data on
its lead compound, TM-1803 (referred in the publication as P-PantSAc),
in mice and in other models of Pantothenate Kinase-Associated
Neurodegeneration (PKAN). The lead candidate was shown to have
serum-stability, low toxicity, and efficacy in mitigating the phenotypes
associated with the disease. The paper titled,
Acetyl-4-phosphopantetheine is stable in serum and prevents phenotypes
induced by pantothenate kinase deficiency can be accessed in the online
edition of the journal, Scientific Reports published yesterday,
September 12, 2017.
TM3 Therapeutics was established in 2017 to further the research and
development started through the European Union-funded TIRCON consortium,
and we are excited to move our program forward and toward the clinic.
Our compound has already been given Orphan Drug Designation by the
European Medicines Agency, said Enej Kuscer, PhD, Chief Executive
Officer at TM3 Therapeutics. We strongly believe in the potential of
our drug candidate as an effective treatment option for patients with
Ody C.M. Sibon, PhD, Professor in the Department of Cell Biology,
University Medical Center Groningen, and corresponding author of the
paper added: Our study validates the ability of TM-1803 to serve as a
replacement metabolite in the pathway for coenzyme A synthesis and can
thus effectively bypass the deficiency caused by impairment of
Susan J. Hayflick, MD, Chair of the Department of Molecular & Medical
Genetics, Oregon Health & Science University, who originally discovered
the PANK2 gene and showed that mutations cause PKAN, commented:
This exciting data and TM3 Therapeutics commitment to developing this
program offer hope for a promising drug candidate to help PKAN patients,
who have no effective treatment options.
PKAN is an extremely rare hereditary disorder caused by mutations in the PANK2
gene and accounts for 50-70% of cases of Neurodegeneration with Brain
Iron Accumulation (NBIA), a genetically heterogeneous group of
disorders. The protein, PANK2, is one of four functional human PANK
enzymes that catalyze a key step in the synthesis of coenzyme A (CoA)
from vitamin B5 (pantothenate). CoA is an essential cellular metabolite
that is vital for hundreds of biological processes including cell growth
and cell death. Patients whose cells lack a functional pantothenate
kinase 2 protein can suffer from either classic PKAN, manifested by
early onset, rapid loss of function and death by early adulthood, or
atypical PKAN, characterized by Parkinson disease-like symptoms in later
childhood and adult life.
Valeria Tiranti, PhD, Associate Professor, Unit of Molecular
Neurogenetics, IRCCS Foundation Neurological Institute Carlo Besta,
Milano stated: We are especially encouraged that these results
reinforce the potential effectiveness of TM-1803 for PKAN by not only
preventing CoA depletion but completely reverting a very severe
phenotype induced in a mouse model of CoA deprivation. These results
have been made possible also thanks to AISNAF, the Italian advocacy
association made up of families and patients, that provided the funds
necessary to support the work of my collaborator Ivano Di Meo, first
author of the paper.
TM3 Therapeutics was established to further the research program started
through the European FP7 consortium TIRCON, which included a network of
leading clinicians, academic researchers and industrial contributors
with the purpose of finding new treatments for PKAN. TM3 Therapeutics
lead compound, TM-1803 is the continuation of the initial research
conducted in the scope of TIRCON by ACIES BIO d.o.o., a Slovenian
biotechnology firm and the University Medical Center Groningen.
About TM3 Therapeutics B.V.
TM3 Therapeutics is focused on novel treatments for neurodegenerative
and metabolic diseases by developing unique metabolite replacement
therapies. Our goal is to improve survival outcomes and quality of life
of patients with rare, genetic neurological diseases that have limited
or no treatment options. Our clinical development program specifically
targets the coenzyme A biosynthetic pathway, whose malfunction leads to
impaired quality of life, progressive loss of function and significantly
reduced life expectancy. Our vision is to work together with key opinion
leaders, researchers and clinicians to expedite the development of new
therapeutics to treat diseases with significant unmet medical need.
Based in Leiden, The Netherlands, the company was founded in 2017.
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TM3 Therapeutics B.V.
Enej Kuscer, Ph.D., CEO
+386 40 414 818
MacDougall Biomedical Communications
Tel: +49 (0) 89 2424 3494
+49 173 364 1607
Source: TM3 Therapeutics