Regeneron’s immune disorder therapy improves daily functions in trial

(Corrects paragraph 4 to remove reference to placebo and scale)

By Siddhi Mahatole

(Reuters) -Regeneron Pharmaceuticals said on Tuesday its experimental therapy met the late-stage main goal of helping patients with a rare immune disorder significantly improve their daily functions such as talking and eating.

The therapy, cemdisiran, was being tested in adults with generalized myasthenia gravis, a condition where the body attacks itself, weakening the skeletal muscles, especially those controlling the eyes, mouth, throat and limbs.

Cemdisiran, an under-the-skin injection, inhibits the activity of the C5 protein, which triggers the body’s immune response.

Patients on cemdisiran showed a 74% reduction in C5 levels in the 24-week trial.

The reduction was nearly 99% when combined with Regeneron’s drug, Veopoz, the company said.

The drug pozelimab, branded as Veopoz, has been approved to treat another immune disorder, Chaple, in which patients have a mutated CD55 gene that interferes with the body’s mechanism to destroy microbes.

Cemdisiran alone showed slightly better results on measures specific to the condition, compared to the combination, including improvements in daily activities, Regeneron said.

The company plans to submit a marketing application in early 2026 with the U.S. Food and Drug Administration for the therapy.

Shares of Tarrytown, New York-based drugmaker were up nearly 2% at $583.34 in a largely flat broader market at midday trading.

TD Cowen analyst Tyler Van Buren said the data and the therapy’s potential for at-home dosing make it more attractive and convenient for patients, compared to the weekly intravenous treatments that require a physician to administer them.

The treatment showed better symptom control than existing C5 inhibitors, he said, estimating a $2 billion-plus market opportunity.

Currently approved treatments for the condition include Johnson & Johnson’s Imaavy and AstraZeneca’s Soliris that typically require a physician’s supervision. Argenx SE’s Vyvgart Hytrulo is approved as an under-the-skin self-administered injection once every four weeks.

The therapy could potentially be self-injected just once every three months, said Andres Sirulnik, Regeneron’s clinical development head for hematology.

The company is also testing cemdisiran and pozelimab in separate late-stage trials for immune disorders such as rare blood disorder paroxysmal nocturnal hemoglobinuria and vision loss from a condition called geographic atrophy.

(Reporting by Siddhi Mahatole in Bengaluru; Editing by Shilpi Majumdar and Sriraj Kalluvila)