PharmaTher
Files for FDA Orphan Drug Designation for Ketamine to Treat Lou
Gehrig's Disease
TORONTO,
May 25,
2021
--
InvestorsHub NewsWire -- PharmaTher Holdings Ltd. (the "Company" or
"PharmaTher") (OTCQB: PHRRF) (CSE: PHRM), a clinical-stage
psychedelics biotech
company, is pleased to announce that it has filed an application
with the U.S. Food and Drug Administration ("FDA") to receive
Orphan Drug Designation ("ODD") for ketamine to treat amyotrophic
lateral sclerosis ("ALS"), also known as Lou Gehrig's
disease.
ALS is a
progressive neuromuscular disease with a life expectancy of only
two to six years after diagnosis. Currently, there is no known cure
for ALS. ALS affects approximately 50,000 people in the U.S. and
Europe, with over 5,000 new cases diagnosed annually. As ALS
advances, upper and lower motor neurons die, causing the brain to
lose its ability to control muscle movement. ALS patients
experience progressive loss of voluntary muscle action as an effect
of the disease, resulting in the inability to speak, eat, move and,
eventually, breathe. The FDA approved only three pharmaceuticals
for the treatment of ALS: riluzole, edaravone, and Nuedexta
(dextromethorphan HBr and quinidine sulfate). These drugs are
effective against disease mechanisms of ALS but fail to have
measurable effects on attenuating disease progression or improve
survival. Therefore, there is an imperative need for new
pharmacological therapies that can stop or slow the muscle decline
associated with ALS progression and extend the life expectancy of
the ALS patient.
Peak
sales for a new drug to treat ALS has the potential to achieve over
USD $1 billion.
The
rationale for ketamine as a potential treatment for ALS
Ketamine
has the potential to effectively increase the life expectancy of
those with ALS at any stage and slow the progressive loss of muscle
associated with poor outcomes of the disease.
The
University of Kansas Medical Center researchers and inventors of
the potential use of ketamine to treat ALS, Dr. Richard J. Barohn,
M.D., John A. Stanford, Ph.D., and Dr. Matthew Macaluso, D.O., have
made the promising discovery that ketamine can be administered as
an effective treatment for ALS. Unpublished and patent-pending
preclinical research has shown that the administration of ketamine
preserves muscle function in advancing ALS and increases life
expectancy when given in the early stages of muscle
decline.
Ketamine
works by blocking the action of the ionotropic glutamate receptor,
the NMDA receptor. Unlike other inhibitors of NMDA receptor
function, such as riluzole, ketamine dampens NMDA receptor-related
glutamate excitotoxicity indirectly. Further, ketamine can lower
D-serine concentrations intracellularly and also partially
activates dopamine receptors. Collectively, these mechanisms of
ketamine contribute in part to the drug's neuroprotective effects,
which may extend to the motor neurons targeted in ALS.
PharmaTher previously announced that it has entered into an
exclusive license agreement with The University of Kansas for the
development and commercialization of the intellectual property of
ketamine in the treatment of ALS.
Fabio
Chianelli, Chief Executive Officer of PharmaTher, said, "Ketamine
has tremendous potential for not only mental illness and pain
disorders but also for neurodegenerative diseases such as
ALS.
Our FDA
orphan drug application for ketamine to treat this life-threatening
disease complements our patent portfolio that aims to protect the
proposed method of use and dosage claims of ketamine for
ALS.
Although
patent protection or market exclusivity could last beyond 2040, our
intention to seek orphan drug designation is to leverage the
expedited regulatory and clinical development incentives offered by
the FDA for repurposed drugs in treating rare
diseases.
We are
working with our clinical and regulatory advisors in preparing our
clinical development plan and study protocol while leveraging our
recent investigational new drug application approved by the FDA for
Parkinson's disease to proceed with a potential phase 2 clinical
study in ALS patients this year."
The
Orphan Drug Act grants special status to a drug or biological
product to treat a rare disease or condition upon request of a
sponsor. This status is referred to as orphan designation (or
sometimes "orphan status"). The FDA grants orphan status to
products that treat rare diseases, providing incentives to sponsors
developing drugs or biologics. The FDA defines rare diseases as
those affecting fewer than 200,000 people in the United States at
any given time. The incidence of ALS is well within the limit of
200,000 for orphan indication status. Orphan drug designation would
qualify ketamine for certain benefits and incentives, including
seven years of marketing exclusivity if regulatory approval is
ultimately received for the designated indication, potential tax
credits for certain clinical drug testing costs, activities,
eligibility for orphan drug grants, and the waiver of the FDA New
Drug Application filing fee of approximately $2.4
million.
About
PharmaTher Holdings Ltd.
PharmaTher Holdings
Ltd. (OTCQB: PHRRF) (CSE: PHRM) is a clinical-stage
psychedelics biotech
company focused on the research, development and commercialization
of novel uses, formulations and delivery methods of psychedelics to
treat mental illness, neurological and pain
disorders.
PharmaTher is
currently conducting
an
FDA approved phase 2 clinical study with ketamine to treat
Parkinson's disease and is developing a novel microneedle
patch for the intradermal
delivery of psychedelics.
Learn more at:
PharmaTher.com
and follow us on
Twitter
and
LinkedIn.
For more
information about
PharmaTher, please
contact:
Fabio
Chianelli
Chief
Executive Officer
PharmaTher
Holdings
Ltd.
Tel:
1-888-846-3171
Email:
info@pharmather.com
Website:
www.pharmather.com
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