Pharming Reports Positive Interim Data From Study Of Leniolisib In Rare Primary Immunodeficiency

(RTTNews) - Pharming Group N.V. (PHAR) announced data, including new evidence, from an interim analysis of its open-label extension study evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor, to treat adult and adolescent patients with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.

The ongoing extension study included 37 patients with APDS aged 12 years or older who, at the time of data cutoff for the interim analysis, had received 70 mg of the selective PI3Kd inhibitor leniolisib twice a day for up to six years and three months, with a median duration on study therapy of 102 weeks.

The results demonstrated the long-term tolerability of leniolisib, with a median duration on study therapy of just over 2 years (102 weeks) and 5 subjects being treated for 5 years or more. Individuals with APDS frequently suffer from recurrent infections and lifelong Immunoglobulin Replacement Therapy (IRT) is required to reduce this burden.

Notably, leniolisib treatment demonstrated a significant reduction in the annualized infection rate, while 37% of study patients on IRT were able to either reduce or altogether stop their IRT regimens.

According to the company, Six patients became IRT-independent, with four of those patients having been IRT-independent for 1 to 2.5 years at the data cutoff. As of the data cut-off for the interim analysis, among three patients who had a history of lymphoma prior to the trial, none had a recurrence or new lymphoma while participating in the study.

The company noted that the interim analysis demonstrated leniolisib to be well tolerated and indicated the durability of the efficacy results seen in the Phase II/III randomized, controlled trial.

The U.S. Food and Drug Administration is conducting a priority review of Pharming's New Drug Application for leniolisib as a treatment for adolescents and adults with APDS and has an assigned Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023.

In addition, Pharming's Marketing Authorisation Application (MAA) for leniolisib in the same patient population has been validated for evaluation under an accelerated assessment by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP). Marketing authorization for leniolisib in the European Union is anticipated in the first-half of 2023.

APDS is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. APDS is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3Kd (phosphoinositide 3-kinase delta) pathway.

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