OUR PIPELINE AND PROGRAMS

UNMATCHED BREAKTHROUGH

COUR’s CNP-101 for celiac disease, partnered with Takeda, is the first therapy to induce antigen-specific immune tolerance in any autoimmune disease in humans.

VALIDATED PLATFORM WITH BROAD APPLICATIONS

COUR’s Nanoparticle Platform can be employed to induce disease-specific tolerance for the treatment of a myriad of autoimmune and allergic diseases by changing the antigen or allergen that is encapsulated within the nanoparticle.

COUR is advancing its pipeline of first-in-class therapies for immune-mediated disease.

IMMUNE REPROGRAMMING

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CELIAC DISEASE (CNP-101)

Celiac disease is a genetic autoimmune disorder that affects approximately 1% of the global population. In celiac disease, ingestion of gluten leads to damage of the villi in the small intestine causing malabsorption of nutrients. This can lead to long-term health conditions such as nutritional and vitamin deficiencies often precipitating early onset of osteoporosis or osteopenia. Furthermore, it can lead to more malignant conditions such as Non-Hodgkin lymphoma and small intestinal adenocarcinoma.

There is no approved drug for the treatment of celiac disease, or medication to prevent the immune attack or repair the damage to the villi resulting from exposure to gluten. Current treatment is limited to maintaining a gluten-free diet. Failure to comply with a gluten-free diet will typically lead to relapse as the underlying pathology of celiac disease remains unaddressed.

COUR has developed CNP-101, a biodegradable nanoparticle encapsulating gliadin proteins – the major component of dietary gluten. CNP-101 is the first agent to induce antigen-specific immune tolerance in any autoimmune disease.

In a Phase 2, double-blind, placebo-controlled clinical trial, patients with celiac disease were treated with CNP-101 and, starting one week later, were fed wheat gluten for 14 days. While patients receiving placebo developed severe immune responses to gliadin and damaging inflammation in their small intestine, patients treated CNP-101 showed significantly less inflammation. In addition, CNP-101 showed a trend toward protecting the intestines from gluten-related injury when compared to untreated patients.

Based on these Phase 2 clinical results, Takeda acquired the license to CNP-101 in October 2019 and will lead further clinical development.

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PEANUT ALLERGY (CNP-201)

Food and Environmental allergies are Immunoglobulin E-mediated (IgE) responses to food and environmental allergens. Symptoms can range from allergic rhinitis to life-threatening anaphylaxis. Typical treatment of allergies involves avoidance of the allergen or management of the symptoms with medications such as anti-histamines. Furthermore, current immunotherapy treatments provide limited protection showing only limited protection against extremely small amounts of the allergen.

Eight types of food account for about 90% of all food-related allergic reactions. These foods are: Peanuts, Eggs, Milk, Tree Nuts, Fish, Shellfish, and Soy. Environmental allergens can be plant, animal, or insect based. Common environmental allergens include pollen, cats, dogs, and dust mites. COUR’s lead program in allergy is directed against the growing threat of peanut allergies in adults and children.

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MULTIPLE SCLEROSIS (CNP-202)

Multiple Sclerosis (MS) is a debilitating autoimmune disease affecting an estimated one million people in the U.S. MS is characterized by the dysregulated immune response towards myelin proteins of the central nervous system, which causes the destruction of neurons, disrupts the flow of information within the central nervous system, and ultimately results in disease symptoms such as loss of movement, impaired vision, and fatigue. Existing therapies for MS rely on aggressive and escalating immune-suppressant regimens that cause severe side effects and compromise quality of life.

COUR’s CNP-202 is a first-in-class therapeutic approach for MS, designed to reprogram the immune system to rebuild tolerance to myelin proteins, and provide long-term therapeutic benefit by preventing immune-mediated destruction of neurons and allowing the regeneration of damaged neurons (remyelination).

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NEUROMYELITIS OPTICA (CNP-Aquaporin 4)

Neuromyelitis Optica (NMO) is a rare autoimmune disease that stems from immune reactivity to self-antigens, primarily Aquaporin-4, found on cells in the eye and the central nervous system. NMO is characterized by symptoms such as blindness, weakness or paralysis, loss of sensation, and spinal cord damage.

Existing therapies in use in the clinic or under development for the treatment of NMO are aimed at broadly suppressing the immune system. While these therapies relieve symptoms, they do not address the underlying causes driving disease and result in severe side-effects.

COUR’s CNP-Aquaporin-4 is designed to address the fundamental mechanisms that drive NMO disease symptoms by reprogramming the immune system to tolerate the self-antigens associated with NMO. Our breakthrough technology will provide long-term therapeutic benefit without causing immune suppressive side-effects.

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NEUTRALIZING ANTIBODIES (CNP-AAVc)

The immune system recognizes biologic and gene therapies as foreign, and responds by producing neutralizing antibodies against them. Neutralizing antibodies swiftly bind and inhibit biologics and gene therapies, which renders them therapeutically ineffective. Neutralizing antibodies are a major clinical challenge as repeated dosing of biologics and gene therapies, essential for continued therapeutic efficacy, becomes impossible.

So far, clinicians have relied on co-administration of immune suppressant regimens with biologics and gene therapies to overcome neutralizing antibodies; however, these immune suppressive regimens have limited usefulness and come with the high cost of severe side-effects and poor quality of life. CNP-AAVc is designed to reverse this unwanted immunogenic response to enable both repeat and long-term dosing of biologic, protein replacement and gene therapies.