|[July 03, 2014]
Akashi Therapeutics Receives Collective Funding of $1.5 Million to Support HT-100 Clinical Development
CAMBRIDGE, Mass. --(Business Wire)--
Therapeutics, Inc. announced today that the Muscular Dystrophy
Association (MDA (News - Alert)) has made an investment through the MDA Venture
Philanthropy program to help fund clinical development of HT-100
(delayed-release halofuginone), an orally available, small molecule drug
candidate intended to reduce fibrosis and inflammation and promote
healthy muscle regeneration in boys with DMD.
The investment from MDA is part of collective funding totaling $1.5
million that Akashi has received in its most recent fundraising round
from MDA and 15 other non-profit organizations to support clinical
development of HT-100. The current funding is in addition to more than
$1 million in non-profit support from an earlier fundraising round, for
a cumulative total of more than $2.5 million raised by Akashi from 25
patient advocacy organizations.
This approach to attracting cooperative funding reflects Akashi's
distinct business model, based on the company's founding by two leading
patient organizations, Charley's
Fund and the Nash
Avery Foundation. The organizations that support Akashi's clinical
development efforts are listed on the company's website at www.akashirx.com/financial-supporters.
"MDA is pleased to provide this funding to evaluate the clinical
potential of HT-100 for the treatment of DMD," said MDA Executive Vice
President and Chief Medical and Scientific Officer Valerie A. Cwik, M.D.
"Significant need remains for safe and effective treatment options for
DMD and we look forward to working together with Akashi Therapeutics to
explore the potential of HT-100 to help individuals living with this
progressive muscle disease."
HT-100 is currently being tested in patients with DMD in a phase 1b/2a
multi-center clinical program to evaluate the safety and tolerability
and assess trends in a range of exploratory biomarkers and efficacy
"We are delighted that these disease foundations, including MDA, are
joining forces with our mission at Akashi to realize the potential of
HT-100 and seek new treatment options for muscular dystrophy," said Marc
B. Blaustein, CEO of Akashi Therapeutics. "We are continuing to
treat patients in the phase 1b/2a multi-center clinical program and
anticipate reporting additional progress in the months to come."
(delayed-release halofuginone) is an orally available, small molecule
drug candidate designed to reduce fibrosis and inflammation and promote
healthy muscle fiber regeneration in DMD patients. HT-100 has been
granted orphan designation for DMD in both the U.S. and E.U., and fast
track designation in the U.S.
About Duchenne muscular dystrophy (DMD)
Affecting approximately 1 in 3,600 boys worldwide, DMD
is the most common of the muscular dystrophies and the most lethal
genetic disorder of childhood. It is caused by a genetic mutation that
renders boys unable to make functional dystrophin, a protein critical
for normal muscle function. Young men with DMD show progressive signs of
physical impairment as early as age three, lose the ability to walk in
their teens, and die of cardiac or respiratory failure in their late
twenties or early thirties.
About Akashi Therapeutics
Therapeutics is a clinical stage biopharmaceutical company whose
mission is to develop treatments for Duchenne
muscular dystrophy and other rare pediatric diseases. Akashi was
founded by leading patient organizations and biotechnology industry
veterans and is managed by a seasoned team of drug development experts
to impact a central problem in rare diseases: rapid therapy development.
Akashi is developing a pipeline
of therapies with the goal of transforming Duchenne from a 100%
fatal, aggressive muscle-wasting disease to a chronic, manageable
condition. For more information, please visit www.akashirx.com.
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