First generalized MG patient dosed in immune-modifying therapy trial

CNP-106 is designed to prevent wrong immune attack against AChRs

Katherine Poinsatte, PhD avatar

by Katherine Poinsatte, PhD |

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The first patient has been dosed in a proof-of-concept clinical trial evaluating CNP-106, an immune-modifying nanoparticle that’s designed to reprogram the immune system in people with generalized myasthenia gravis (gMG).

The Phase 1b/2a trial NCT06106672), sponsored by CNP-106’s developer Cour Pharmaceuticals, will assess the safety, tolerability, efficacy, and pharmacological properties of multiple ascending doses of the therapy in adults with gMG.

“CNP-106 has the potential to be a first-in-class treatment that offers people living with gMG a disease-modifying therapy without burdensome immune system suppression,” Dannielle Appelhans, president and CEO of Cour, said in a company press release.

Myasthenia gravis (MG) is an autoimmune disease caused by self-reactive antibodies that target and attack proteins that are essential for proper communication between nerve and muscle cells, most commonly acetylcholine receptors (AChRs). The generalized form of the disease is marked by widespread muscle weakness and fatigue.

There is no cure for MG, but some treatments can help ease its symptoms. Many medications used to manage MG reduce the activity of the immune system, but don’t tackle the disease’s root cause.

“Currently approved treatments for gMG work via broad immunosuppression and do not address the underlying autoimmunity of the disease,” Appelhans said.

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Clinical testing set for possible first-in-class generalized MG therapy

How does CNP-106 work in generalized MG?

CNP-106, on the other hand, is designed to prevent the erroneous immune attack launched against AChRs by reprogramming the immune system to better distinguish between self and non-self proteins and promote immune tolerance. When the immune system is tolerant of its own proteins, it doesn’t usually attack the body’s own cells, tissues, and organs.

The therapy works by loading parts of the AChR into tiny particles, called nanoparticles, along with other co-stimulating factors that instruct the immune system to recognize them as “self-proteins,” thereby preventing the autoimmune attack that drives MG.

“In preclinical studies to date, CNP-106 has demonstrated a unique ability to induce tolerance to [AChR], leading to improved muscle function in an experimental model of autoimmune myasthenia gravis,” Appelhans said.

CNP-106 is being tested in a Phase 1b/2a clinical trial that’s expected to enroll 54 adults with gMG who test positive for self-reactive antibodies against AChR.

After up to 42 days of screening, the participants will receive intravenous (into-the-vein) infusions of CNP-106 or a matched placebo. The trial is predicted to last 222 days, or about seven months. Its main goal is to assess the frequency of adverse events and serious adverse events that occur through the 180th day of the study. Changes in the levels of immune cells that respond to self-reactive antibodies that target AChR will also be assessed at multiple points as secondary goals.

Earlier this year, Cour raised $105 million in Series A financing to support the development and clinical testing of CNP-106 and CNP-103, a potential type 1 diabetes therapy candidate.