NIJMEGEN, Netherlands,
April 26, 2018 /PRNewswire/ --
Khondrion, a leading clinical-stage pharmaceutical company
focusing on small molecule therapeutics for mitochondrial diseases,
today announced the publication of a scientific article reporting
on the development and unique mode of action of KH176, a
clinical-stage drug candidate for the treatment of mitochondrial
diseases.
About the study
The work was carried out by the Khondrion R&D team in
collaboration with Inoviem Scientific and the Radboud University
Medical Center. The results were published in Scientific
Reports
(http://www.nature.com/articles/s41598-018-24900-3).
KH176 is an orally bio-available small molecule in development
by Khondrion for the treatment of mitochondrial (-related)
diseases. The present study reports on the early development and
selection of KH176 as Khondrion's lead candidate and further
explains its mode of action in primary cells obtained from
mitochondrial patients. "Elevated Reactive Oxygen Species (ROS)
levels and a high sensitivity to redox-stress are two frequent
cellular consequences in mitochondrial disease. Interestingly, we
could demonstrate in several patient-derived cells that KH176 is an
attractive candidate to correct both pathological phenotypes,
illustrating its unique potential for this group of patients" says
Dr. Julien Beyrath, Chief Operating Officer at Khondrion and first
author of the study. KH176 displays a dual mode of action,
targeting both ROS and Redox disturbances. "Using cutting-edge
target deconvolution technology, we could identify peroxiredoxin
enzyme as the primary and novel target of KH176" added Dr. Beyrath.
Research is ongoing to further unravel the exact molecular
mechanism of interaction of KH176 with the peroxiredoxin enzyme,
which will help the Khondrion R&D team to further improve their
drug candidates. "The results presented highlight the potential of
KH176 as a single molecule to be effective in a large group of
heterogeneous mitochondrial diseases, but also other diseases with
underlying ROS and Redox perturbations" adds Prof. dr. Jan Smeitink, CEO at Khondrion and last author
of the study.
About KH176
KH176 was already shown to rescue specific pathological
phenotypes in a relevant mouse model of Leigh disease, one of the
most severe mitochondrial diseases (De Haas, Scientific Reports,
2017 Sep 15;7(1):11733). Khondrion also reported that KH176
demonstrated a favorable pharmacokinetic profile and an acceptable
safety profile in randomized, placebo-controlled, double blind
Phase I clinical trials, performed in healthy male volunteers
(Koene et al, Orphanet Journal of Rare Diseases, 2017 Oct
16;12(1):163).
KH176 efficacy and safety have been evaluated in the KHENERGY
study, a Phase II exploratory trial with oral KH176 in the
m.3243A>G multisystem mitochondrial MELAS and MIDD syndromes and
mixed phenotypes. Based on the outcome of the KHENERGY study (see
earlier press release) the company is preparing for a pivotal
program to confirm the potential benefits of KH176 in patients with
mitochondrial disease.
About Khondrion
Khondrion is a leading clinical-stage pharmaceutical company
focusing on developing small molecule therapeutics for
mitochondrial (-related) diseases. The potential of several
lead compounds to serve as new treatment modalities for
mitochondrial disease is currently being explored.
Khondrion's KH176 has been granted Orphan Drug Designation
(ODD) for Leigh disease and MELAS syndrome in Europe and for all inherited mitochondrial
respiratory chain disorders in the USA. Khondrion has established collaborations
with patient organizations, patient advocacy groups, university
expert centers and research groups around the world. The company is
supported by the Dutch Foundations Energy4All, Join4Energy,
Road4Energy, Ride4Kids, Tim Foundation, Zeldzame Ziekten Fonds, and
National and European Governments. Khondrion has a strong
intellectual property position protecting its emerging product
portfolio via granted and multiple, broad patent applications. For
more information, please visit http://www.khondrion.com
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements. All
statements, other than statements of historical facts, contained in
this press release, including statements regarding our strategy,
future operations, future financial position, future revenues,
projected costs, prospects, plans and objectives of management, are
forward-looking statements. The words "anticipate," "believe,"
"estimate," "expect," "intend," "may," "plan," "predict,"
"project," "target," "potential," "will," "would," "could,"
"should," "continue," and similar expressions are intended to
identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Any
forward-looking statements represent our views only as of today and
should not be relied upon as representing our views as of any
subsequent date. If underlying assumptions prove inaccurate or
known or unknown risks or uncertainties materialize, actual results
could vary materially from the expectations and projections of
Khondrion. Risks and uncertainties include, but are not
limited to: challenges and uncertainties inherent in product
development, including the uncertainties of clinical success and
the timeline for the availability of KH176. While we may elect to
update these forward-looking statements at some point in the
future, we specifically disclaim any obligation to do so, even if
our views change.