Industry Council

Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing medicines. As the global leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second anti-FcRn therapy, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. Further information about Alexion can be found at: www.alexion.com.

About Genentech

Mechanism of Action

SA237 is a humanized anti-human IL-6R neutralizing monoclonal antibody that was designed by application of recycling antibody technology to the approved anti-IL6 receptor antibody, tocilizumab, which is currently marketed as a treatment for rheumatoid arthritis (RA), systemic juvenile idiopathic arthritis, polyarticular juvenile idiopathic arthritis and Castleman’s disease. The recycling antibody technology enabled SA237 to bind to IL-6 receptor multiple times and be slowly cleared from plasma, which is expected to contribute to improvement of patient’s convenience by once monthly dosing frequency. The longer plasma half-life of SA237 compared with tocilizumab was confirmed based on the result of non-clinical study and Phase 1 study in healthy volunteers.

Study Details

This research is being conducted to evaluate the efficacy, safety, pharmacodynamic, pharmacokinetic and immunogenic profiles of a humanized anti-human IL-6R neutralizing monoclonal antibody (SA237) in patients with Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD).

This research is being conducted in the US and Canada.

Ninety (90) patients are going to participate in this research.

Eligible participants

Inclusion Criteria:

1. NMO or NMOSD

2. Age 18 to 74 years, inclusive at the time of informed consent.

Exclusion Criteria:

1. Pregnancy or lactation.

2. Evidence of other demyelinating disease or PML.

3. Known active infection (excluding fungal infections of nail beds or caries dentium) within 4 weeks prior to baseline.

Contact information

If you are interested in participating, please contact:

Contact: Clinical trials information clinical-trials@chugai-pharm.co.jp

Horizon

Mechanism of Action of MEDI-551

MEDI-551 is a humanized monoclonal antibody that acts against the specific antigen CD19 attached to the surface of B lymphocytes cells. These CD 19 B cells, are responsible for the production of autoantibodies against the AQP4 channel protein in the nervous system, which are playing a major role in causing the nerve tissue injury seen in NMO. Human studies have shown that when MEDI-551 attaches to the surface of B cells in the blood it starts a process that eliminates these cells. Reducing the number of these B cells may reduce the ability of the immune system to produce the antibodies that attack the healthy nerve tissues and may delay or prevent an NMO/NMOSD attack.

Study Details

The main objective of this study is to determine if MEDI-551 can significantly delay the time it takes for a new NMO/NMOSD attack to occur.

This is a multinational randomized, double-masked, placebo-controlled study with an open label period extension. “Double-masked” means that patient and the study staff (for example the doctor/nurse) giving the study drug does not know who will be receiving MEDI-551 or placebo. Placebo controlled means that some patients will receive MEDI-551 and some will receive placebo, an inactive substance designed to look like MEDI-551. “Open label” means a period in the study where there is no placebo arm and all patients receive MEDI-551.

Eligible NMO patients will be randomized in a 3:1 ratio to received either IV MEDI-551 or placebo. This random selection is made by a computer and will give a 25% (1 in 4) chance of getting placebo and a 75% (3 in 4) chance of getting MEDI-551. After being enrolled in the study, patients will be followed for 28 weeks; this period is called the placebo-controlled treatment period. Patients will have the option to enroll into the open label extension, if a confirmed attack occurred during the 28 weeks. Subjects who complete the 28 weeks without experiencing an attack will also be given the option to enroll in the open-label extension. During the 28weeks of the study, investigators are allowed to treat NMO attacks with standard rescue medications. MEDI-551 or placebo will be administer intravenously (IV) on day 1 and day 15 and then every 6 months.

Eligibility

(For the complete list of the inclusion/exclusion criteria please visit https://clinicaltrials.gov/ct2/show/NCT02200770?term=NMO&rank=8)

Inclusion Criteria:

Men and women 18 years or older with diagnosis of NMO/NMOSD

Confirmation of NMO/NMOSD status:

AQP4-IgG sero-positive and sero-negative NMO/NMOSD with at least one attack requiring rescue therapy in the last year or two attacks requiring rescue therapy in the last 2 years

EDSS <= 7.5

Exclusion Criteria:

Lactating and pregnant females.

Known history of a severe allergic reaction or anaphylaxis following any biologic therapy.

Known active severe bacterial, fungal or viral, or other infection or any major episode of infection requiring hospitalization.

Emeritus Members

Industry Council Contributions Support:

The Guthy-Jackson Charitable Foundation invites all members of industry to join our industry council.

Clinical Trials Education Electronic Media
(e.g. podcasts, webinars)

A series of educational Webinars with a target audience of NMO stakeholders, including patients, caregivers, researchers, physicians, and other advocates. These resources offer information for stakeholders

Annual Roundtable

The largest international three-day conference that convenes researchers, clinicians, industry representatives, advocates, KOLs and other stakeholders from 22+ countries around the world.

Regional and Annual Patient Days

Regional NMO Advocacy days join patients, caregivers, families and friends who come together for the purpose of NMO education, information, and support. The GJCF Annual Patient Day is the largest international one day conference that convenes NMO advocates, clinicians, researchers, and industry from around the world.

Stakeholder Meetings

Summits, conferences, symposia and other venues for idea and information sharing.

Media Messaging

Outreach through social, digital, educational, and advocacy media to heighten awareness of NMO/SD and clinical trials.