TIDMCLIN
RNS Number : 7304S
Clinigen Group plc
13 July 2020
13 July 2020
Clinigen granted Orphan Drug Designation by the FDA for
aldesleukin
in the treatment of Amyotrophic Lateral Sclerosis (ALS)
Clinigen Group plc (AIM: CLIN, 'Clinigen' or the 'Group'), the
global pharmaceutical and services company, announces that the US
Food and Drug Administration (FDA) Office of Orphan Products
Development (OOPD) has granted Orphan Drug Designation for
aldesleukin in the treatment of Amyotrophic Lateral Sclerosis
(ALS). ALS is a severe, neurodegenerative disease which affects
motor neurons leading to progressive muscle weakness, paralysis and
ultimately death within a median time of 2-4 years from disease
onset.
An Orphan Drug Designation in the US recognizes the potential
therapeutic role of aldesleukin in this devastating disease and
could provide a number of benefits for Clinigen should it obtain a
marketing approval for this indication. These benefits include
seven years marketing exclusivity within the United States upon
launch, along with tax credits for clinical development costs and
fee waivers.
Studies in ALS patients have shown that decreased levels of
regulatory T-cells (Tregs), were associated with increased disease
severity and predictive of disease progression and survival,
suggesting that Tregs may be an attractive therapeutic target.
Aldesleukin, when administered at doses significantly lower than
those used in oncologic indications, has been shown to enhance Treg
function in inflammatory and auto-immune conditions.
Clinigen is supplying aldesleukin being used in the ongoing
MIROCALS study evaluating its clinical potential within ALS and is
investigating the optimal pathway to generating the data required
to support an application for a marketing authorisation.
Shaun Chilton, Group Chief Executive Officer, Clinigen,
said:
"The Orphan Drug Designation issued by the FDA recognizes the
potential of aldesleukin as a possible valuable new treatment for
patients with ALS where there is a significant level of unmet need
within the disease area. Clinigen is also exploring the use of
aldesleukin in several other therapeutic areas where its modulatory
effects on Tregs may have a beneficial clinical effect."
- Ends -
Contact details
Clinigen Group plc Tel: +44 (0) 1283 495010
Shaun Chilton, Group Chief Executive Officer
Nick Keher, Group Chief Financial Officer
Matt Parrish, Head of Investor Relations
J.P.Morgan Cazenove - Nominated Adviser Tel: +44 (0)20 7742 4000
& Joint Broker
James Mitford / Hemant Kapoor
RBC Capital Markets - Joint Broker Tel: +44 (0) 20 7653 4000
Marcus Jackson / Elliot Thomas
Instinctif Partners Tel: +44 (0) 20 7457 2020
Melanie Toyne-Sewell / Phillip Marriage Email: clinigen@instinctif.com
Notes to Editors
About US Food and Drug Administration (FDA) Office of Orphan
Products Development (OOPD)
The FDA Office of Orphan Products Development (OOPD) mission is
to advance the evaluation and development of products (drugs,
biologics, devices, or medical foods) that demonstrate promise for
the diagnosis and/or treatment of rare diseases or conditions. In
fulfilling that task, OOPD evaluates scientific and clinical data
submissions from sponsors to identify and designate products as
promising for rare disease and to further advance scientific
development of such promising medical products. The office also
works on rare disease issues with the medical and research
communities, professional organizations, academia, governmental
agencies, industry, and rare disease patient groups.
About Aldesleukin
It is a recombinant interleukin 2 (IL-2), a native cytokine
whose role in the immune system has been extensively studied.
Aldesleukin is currently marketed under the brand name Proleukin.
At conventional doses it is used in the treatment of kidney cancer
that has spread to another part of the body (metastatic kidney
cancer). It is also currently being studied as part of adoptive
cellular therapy. At significantly lower doses than those used in
oncology aldesleukin has demonstrated immunomodulatory effects in
certain auto-immune and inflammatory disease states by boosting the
levels of a particular subset of white blood cells called
T-regulatory lymphocytes (Tregs).
About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS) is a life threatening and
chronically debilitating disease. It is considered to be a
multifactorial disease characterised by certain mechanisms
initiating motor neuron injury, requiring non-neuronal cells for
rapid disease progression and motor neuron cell death.
Motor neurons are nerve cells that extend from the brain to the
spinal cord and to muscles throughout the body. These motor neurons
initiate and provide vital communication links between the brain
and the voluntary muscles.
In ALS, both the upper motor neurons and the lower motor neurons
degenerate or die and stop sending messages to the muscles. Unable
to function, the muscles gradually weaken, start to twitch (called
fasciculations), and waste away (atrophy). Eventually, the brain
loses its ability to initiate and control voluntary movements.
The disease is progressive, meaning the symptoms get worse over
time. Currently, there is no cure for ALS and no effective
treatment to halt, or reverse, the progression of the disease.
Most people with ALS die from respiratory failure, usually
within 2 to 4 years from when the symptoms first appear. However,
about 10 percent of people with ALS survive for 10 or more
years.
About MIROCALS
MIROCALS (Modifying Immune Responses and Outcomes in ALS) is a
randomised placebo-controlled trial of low dose aldesleukin in
people with ALS. The study is designed to assess the effectiveness
of aldesleukin in improving survival and the ability of people with
ALS to perform day to day activities. The study is scheduled to end
in July 2021 with results in late 2021.
About Clinigen Group
Clinigen Group plc (AIM: CLIN) is a global pharmaceutical and
services company with a unique combination of businesses focused on
providing ethical access to medicines. Its mission is to deliver
the right medicine to the right patient at the right time through
three areas of global medicine supply; clinical trial, unlicensed
and licensed medicines. The Group has sites in North America,
Europe, Africa and the Asia Pacific region.
Clinigen now has over 1,100 employees across five continents in
14 countries, with supply and distribution hubs and operational
centres of excellence in key long-term growth regions. The Group
works with 22 of the top 25 pharmaceutical companies; interacting
with over 15,000 registered users across over 100 countries,
shipping approximately 6.4 million units in the year.
For more information on Clinigen, please visit
www.clinigengroup.com
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END
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