Company Announcements

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."

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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