E-DRUG: DNDi: Innovative Partnership Advances Novel Drug Candidate for Sleeping Sickness
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[Question from Moderator: What will be put in place to ensure that the product will be genuinely affordable to those who need it?]
http://www.dndi.org/press-releases/892-oxaborolesbio.html
Innovative Partnership Advances Novel Drug Candidate to Combat Sleeping
Sickness
PLoS Journal article highlights initial research success of a boron-based
compound, ready to enter into clinical development, resulting from
collaboration between two U.S. biotechs and DNDi
WASHINGTON/GENEVA (28 JUNE 2011)The Drugs for Neglected Diseases initiative
(DNDi), Anacor Pharmaceuticals, and SCYNEXIS Inc. today announced the
successful completion of pre-clinical studies for the first new oral drug
candidate discovered specifically to combat human African trypanosomiasis
(HAT), also known as sleeping sickness. An article released today in the
open-access journal PLoS Neglected Tropical Diseases, reveals the initial
successful results of pre-clinical studies of the new compound, which will
soon advance to Phase I human clinical trials.
Two-thirds of all reported sleeping sickness cases are found in the
Democratic Republic of the Congo (DRC), where healthcare is often
inaccessible to large parts of the population due to violent conflict, great
distances patients must travel to health facilities, and extreme poverty.
Dr. Miaka Mia Bilenge, special advisor to the National Control Program for
Human African Trypanosomiasis in the DRC, explained that if the new drug
candidate proves to be successful, it could become a major weapon to control
sleeping sickness.
People in the DRC and many countries in Africa have hoped for a safe,
easy-to-use, and effective treatment against sleeping sickness for
generations, Dr. Miaka Mia Bilenge said. We very much look forward to the
start of human trials, and we hope that at long last this will give us a
tool for sustainable elimination of this dreaded disease.
In pre-clinical studies, the new drug candidate, SCYX-7158, also registered
as AN5568, demonstrated safety and the ability to cross the blood-brain
barrier making it efficacious against stage 1 and stage 2 of the disease. In
addition, its oral formulation, short duration of therapy and excellent
pre-clinical safety profile imply that SCYX-7158 (AN5568) could change the
way sleeping sickness is treated, reduce its incidence in humans, and
contribute to elimination of the disease.
The successful development of this new compound was the result of a unique
collaboration between Anacor Pharmaceuticals, a biopharmaceutical company in
Palo Alto, California, SCYNEXIS, a drug discovery and development company
based in Research Triangle Park, North Carolina, and DNDi, a not-for-profit
research and development (R&D) organization that develops new drugs to
address the needs of the worlds most neglected populations.
This innovative collaboration, expounding upon the expertise and engagement
of two biotech companies and a not-for-profit R&D organization, has brought
excellent results thus far, and this in an incredibly short timeframe, said
Dr. Bernard Pécoul, Executive Director of DNDi. The message is clear that
it is possible to stimulate patient needs-driven R&D for neglected tropical
diseases, even if there is no commercial market, added Pécoul.
SCYX-7158 (AN5568) is a product of Anacors novel boron chemistry which has
produced a number of compounds with efficacy against a range of fungal,
inflammatory and bacterial diseases. Realizing this technology could also
be used for neglected diseases, Anacor, with the help of the Sandler Center
for Drug Discovery of the University of California, San Francisco, screened
its library of boron-based compounds for activity against the sleeping
sickness parasites and identified an attractive lead series. In order to
ensure further development of these compounds, Anacor approached DNDi, which
was actively seeking compounds for its lead optimization program. DNDi,
Anacor, SCYNEXIS, and a consortium including Pace University and the Swiss
Tropical and Public Health Institute then worked on the series of molecules
in pre-clinical studies that led to the development of the compound for
which DNDi is filing a dossier for first in man (FIM) studies to enter Phase
I clinical trials in Europe.
Due to its safety and efficacy against both stages of the disease, and its
ability to be administered orally, this compound could be a breakthrough in
the treatment of sleeping sickness and could significantly improve the lives
of those who suffer from this insidious disease, said David Perry, CEO of
Anacor Pharmaceuticals. We are committed to applying our boron chemistry
platform to the discovery and development of new treatments for neglected
diseases, and its only with the support of organizations like DNDi and
their donors that we are able to fulfill that commitment.
SCYNEXIS CEO, Dr. Yves Ribeill, said the program is part of SCYNEXIS policy
of contributing to neglected disease projects. Success so far was in large
part due to the work of several organizations working closely together. If
we are going to find effective medicines against diseases that exact a
terrible toll in developing countries, we need to find new approaches in the
discovery process. This program is an example, said Ribeill.
Thanks to continuous support from public and private donors, DNDi secured
USD$14.8 million in support of this lead optimization and pre-clinical
program. Most of the funding was provided by the Bill & Melinda Gates
Foundation. In addition, specific funding was obtained from the Ministry of
Foreign and European Affairs (MAEE), France, and core funding from the
Spanish Agency of International Cooperation for Development (AECID), Spain
and Doctors Without Borders/Médecins Sans Frontières (MSF). Additional
funding is being sought for the clinical phases of this program.
..
About sleeping sickness
Sleeping sickness, which threatens millions in 36 countries in sub-Saharan
Africa, is fatal if left untreated. The disease is caused by parasites
transmitted by the bite of a tsetse fly and is often asymptomatic for years
until the infection reaches stage 2 where it crosses into the central
nervous system and brain. Without effective treatment, sleeping sickness is
fatal. Currently available treatments are limited to drugs developed decades
ago that are either highly toxic, difficult to administer in
resource-limited settings, or are only effective in one stage of the
disease. In addition, prior to being treated, the stage of the disease must
be determined using a diagnostic spinal tap to extract cerebrospinal fluid
from the patient.
About Anacor Pharmaceuticals
Anacor is a biopharmaceutical company focused on discovering, developing and
commercializing novel small-molecule therapeutics derived from its boron
chemistry platform. Anacor has five compounds in clinical development, all
of which were internally discovered, including its three lead programs:
AN2690, a topical antifungal for the treatment of onychomycosis; AN2728, a
topical anti-inflammatory PDE-4 inhibitor for the treatment of psoriasis and
atopic dermatitis; and GSK 2251052, or GSK 052 (formerly referred to as
AN3365), a systemic antibiotic for the treatment of infections caused by
Gram-negative bacteria, which has been licensed to GlaxoSmithKline under the
companies' research and development agreement. In addition, Anacor is
developing AN2718 as a topical antifungal product candidate for the
treatment of onychomycosis and skin fungal infections, and AN2898 as a
topical anti-inflammatory product candidate for the treatment of psoriasis
and atopic dermatitis.
Contact:
DeDe Sheel, Director, Investor Relations and Corporate Communications