FDA grants orphan drug designation to CNP-106 for gMG
Experimental treatment aimed at reprogramming immune system in disorder
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CNP-106, an experimental treatment that Cour Pharmaceuticals is developing to reprogram the immune system in generalized myasthenia gravis (gMG).
“Receiving orphan drug designation for CNP-106 is an important development and regulatory milestone for our myasthenia gravis program,” Dannielle Appelhans, Cour’s president and CEO, said in a company press release announcing the FDA’s decision. The designation includes a series of benefits, such as a seven-year market exclusivity period if CNP-106 is approved.
The company is still enrolling up to 54 adults with gMG into its ongoing Phase 1b/2a clinical trial (NCT06106672), which is testing how safe CNP-106 is and how well it works compared with a placebo at retraining the immune system to tolerate proteins that are a healthy part of the body.
With the first patient dosed nearly a year ago, and “as enrollment in our clinical trial for CNP-106 in gMG continues to advance, we are looking forward to sharing data readouts from our differentiated tolerance approach,” Appelhans said. There are a dozen trial locations currently open across the U.S.
CNP-106 carries 7 pieces of key protein
Myasthenia gravis is caused by self-reactive antibodies that target proteins — most often the acetylcholine receptor (AChR) — essential for proper nerve-to-muscle communication. The generalized form of the disease is marked by widespread muscle weakness.
Some treatments can help ease symptoms, but many suppress the immune system without addressing the underlying cause of the disease.
“CNP-106 represents a novel tolerance-based approach aimed at addressing the underlying immune response that [causes] anti-AChR antibody positive gMG,” Appelhans said.
CNP-106 is a very small, biodegradable particle that carries seven pieces of the AChR. It is designed to instruct the immune system to recognize them as a healthy part of the body rather than as a threat, thereby reducing the production of anti-AChR antibodies. This should restore nerve-to-muscle communication, easing disease symptoms.
CNP-106 represents a novel tolerance-based approach aimed at addressing the underlying immune response that [causes] anti-AChR antibody positive gMG.
The goal of the ongoing clinical trial is to provide proof of concept evidence that CNP-106 can induce immune tolerance to the AChR in a relatively small number of patients, which should inform the design of future later-stage clinical trials. To be eligible, patients must test positive for anti-AChR antibodies.
They will be randomly assigned to receive two intravenous, or into-the-vein, infusions of CNP-106, or a placebo, spaced one week apart. The first group of patients must be MG Foundation of America (MGFA) Class III or IV, meaning they must have moderate or severe generalized muscle weakness.
A second group of patients — who will receive a higher dose of CNP-106 if the lower dose is deemed safe, or a placebo — may also include those in MGFA Class II. These are patients with mild weakness in muscles other than those in the eyes who may also experience ocular muscle weakness, as in Class III or IV.
Researchers will monitor the frequency of side effects, including serious ones, over about six months. Secondary measures include tracking changes in the numbers of immune T-cells that specifically recognize the AChR.
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