Regeneron’s MED MED for muscle disease achieves test objectives; FDA deposit scheduled for next year

A Regeneron pharmaceutical drug which reduces the levels of a protein driven by the disease has achieved the objectives of a pivot test in serious myasthenia generalized by rare disease. On the basis of these results, Regeneron plans to request the approval of the FDA for therapy, potentially introducing a new mechanism of action in the field of increasingly competitive treatments for this neuromuscular disorder.

The medication Regeneron, Cemdisiran, adopts a new approach to the treatment of serious myasthenia, a disease that causes difficulties in swallowing as well as muscle weakness. Autoimmune disorder develops as the body attacks key proteins for communication between nerves and muscles. Abnormal antibodies produced by the body activate the complement system, part of the immune system. Cemdisiran is a small interferring RNA medication (SIRNA) designed to reduce the circulation levels of the C5 protein of the supplement system.

In the results of the upper line of the study of the phase 3 controlled by a 24 -week placebo, Regeneron said Tuesday that Cemdisiran in monotherapy had led to an average inhibition of 74% of the additional activity. The test also assessed the combination of Cemdisiran with the approved inhibitor of Regeneron Pozlimab, the name of the Veopoz brand. The combination even carried out an inhibition of the supplement system, Regeneron signaling the pairing of drugs led to an inhibition of almost 99% of the activity of the supplement.

Nevertheless, the main objective of the test is to measure the change of score according to a scale which measures the activities of daily operation. On this measure, Cemdisiran monotherapy has shown digitally better scores, which indicates better improvement in symptoms and better treatment effect. Regeneron leaders said the results indicate that it may not be necessary to completely block the activity of the supplement system.

“The efficiency potential of the best category with a less than complete complement blockage with Cemdisiran monotherapy can also provide a more favorable security profile,” said Regeneron president and scientific director George Yancopoulos in a prepared statement. “These exciting results highlight the potential transformer of our SIRNA pipeline and genetic drugs to provide paradigm therapies for patients.”

Regeneron said that the detailed results of the phase 3 study will be presented at a next medical meeting. Pending discussions with the FDA, the company provides for American regulatory submission in the first quarter of next year.

Myasthenia Gravis has welcomed several new drugs in recent years. The Argenx Vyvgart, approved in 2021, is a fragment of antibodies designed to block the neonatal FC receptor (FCRN), leading to the degradation of autoanticorps driven by the disease by the integrated cell elimination system. The UCB has two myasthenia gravis drugs, the FCRN Rystiggo blocking antibodies and the C5 Zilbrysq inhibitor, both approved in 2023. Myasthenia Gravis is also one of the indications for the Astrazeneca C5 blockbuster C5 Inhibiters Soliris and Ultomiris. The most recent drug Myasthenia Gravis is Imavy by Johnson & Johnson, an FCRN blocking antibody granted FDA approval in May.

In a note sent to investors, Leerink Partners’ analyst David Risisher said that although differences in clinical studies make inter-procedure comparisons difficult, the results of the effectiveness of Regeneron’s medication are not below drugs that block FCRN. But he added that the results of the Regeneron medication were roughly comparable to the inhibitors of the Astrazeneca C5, and Cemdisiran could stand out here.

C5 inhibitors are delivered with requirements that make them binding compared to other Myasthenia Gravis therapies. Astrazeneca drugs are only approved for patients who test positive for a particular type of disease driver of disease, anti-acetylcholine antibodies, said risinger. Inhibition of the supplement system increases the risk of potentially fatal meningococcal infections. This risk is reported in a black box warning which advises vaccination against meningococcal at least two weeks before taking a C5 inhibitor. In addition, C5 inhibitors are only available through a risk assessment program and mitigation strategy (REMS) which manages the risks of these therapies.

Cemdisiran comes from the Alnylam Pharmaceuticals RNA interference specialist. Regeneron conceded global rights to develop the Sirna medication by itself and in combination with C5 blocking antibodies. Alnylam has the right to receive payments of regulatory milestones and royalties thanks to the sales of an approved product.

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