Pharming Announces Positive Phase II/III Leniolisib Clinical Trial Data Presented at the 2022 Society for Clinical Immunology Annual Meeting

announces new data from the pivotal phase II clinical trial of Leniolisib /III for the treatment of active phosphoinositide 3-kinase delta (PI3K?) syndrome (APDS), a primary immunodeficiency. Principal Investigator V. Koneti Rao , MD, staff physician at the Primary Immune Deficiency Clinic at the National Institute of Health in Bethesda, Maryland , shared the results in a presentation at the 2022 Society for Clinical Immunology (CIS) Annual Meeting. As announced on February 2, 2022, the multinational, triple-blind, placebo-controlled, randomized, phase III portion of the clinical trial conducted by Novartis has reached its co-primary endpoints, which served to assess the reduction in ganglion size. lymphatic system and correction of immunodeficiency. Shrinking lymphadenopathy lesions and increasing proportion of mature B cells are important in this group as they indicated a reduction in APDS disease markers. First presented at CIS, co-primary outcomes at day 85 after baseline demonstrated: In the lymphadenopathy lesion index, there was a statistically significant mean change in the transformed log10 sum of the product of diameters (SPD) of -0.30 among patients receiving Leniolisib compared to -0.06 among patients receiving placebo ( CI 95%: -0.37, -0.11; p=0.0012). From baseline of ?48%, there was a 34.76% increase in the proportion of independent B cells in patients receiving Leniolisib compared to a -5.37% reduction in patients receiving placebo (CI 95%: 28.51, 51.75; P:0.0001). Study drug was well tolerated. There were no adverse events leading to discontinuation of study treatment, there were no deaths, and the incidence of serious adverse events (SAEs) was lower in the Leniolisib group than in the placebo group. None of the SAEs were suspected to be related to the study treatment. Charlotte Cunningham-Rundles, MD, Ph.D., David S. Gottesman , professor of immunology at Mount Sinai School of Medicine in New York , said: "It is great news that Leniolisib has achieved such positive results in this Phase III trial of APDS. It is extremely encouraging to see that this drug is able to target the root cause of this difficult disease, both improving treatment and reducing patients' symptoms. Progress toward tailor-made treatment for our APDS patients is a long-awaited milestone." Pharming plans to begin filing global regulatory filings for Leniolisibe in the second quarter of 2022 and, once approved, will launch the treatment in the US in the first quarter of 2023 and begin a series of European launches in the second half of 2023. Anurag Relan , medical director at Pharming, commented: "Pharming is very pleased that Leniolisib achieved significance in both co-primary outcomes and was well tolerated in these patients with APDS, as the product approval will address an unmet need for patients with this rare disease, which currently rely on supportive therapies such as antibiotics and immunoglobulin replacement therapy. In addition to working closely with regulatory authorities around the world to make Leniolisib available to immunologists, hematologists, and their patients, we will continue to develop this treatment through our open-label extension clinical trial and two additional studies enrolling children under 12 years of age, and we will also potentially extend the geographic reach of the product." About Active Phosphoinositide 3-Kinase Syndrome (APDS) APDS is a rare primary immunodeficiency that affects approximately one to two people per million. Also known as PASLI, it is caused by variants of either of the two genes, PIK3CD or PIK3R1, which regulate the maturation of white blood cells. Variants of these genes lead to hyperactivity of the PI3K? (Phosphoinositide 3-kinase delta) pathway. 1.2 Balanced signaling in the PI3K? pathway is essential for immune function. When this pathway is overactive, immune cells do not mature or function properly, resulting in immunodeficiency and dysregulation. 1.3 APDS is characterized by severe and recurrent sinopulmonary infections, lymphoproliferation, autoimmunity and enteropathy. 4.5Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are often misdiagnosed and experience a median diagnosis delay of 7 years. 6 As APDS is a progressive disease, this delay can lead to aggregation of damage over time, including permanent lung damage and lymphoma. 4-7 The only way to definitively diagnose this disease is through genetic testing. About Leniolisib Leniolisib is a small molecule inhibitor of the 110 kDa catalytic subunit delta isoform of class IA PI3K with immunomodulatory and potentially antineoplastic activities. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 acts as an important cellular messenger that specifically activates AKT (via PDK1) and regulates a multitude of cellular functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis and metabolism. Unlike PI3K? and PI3K? which are expressed in a general way, PI3K? and PI3K? are expressed mainly in cells of hematopoietic origin. The central role of PI3K? in the regulation of numerous cellular functions of the adaptive immune system (B cells and, to a lesser extent, To date, Leniolisib has been well tolerated during the first Phase I study in healthy subjects and the Phase II/III enrollment enablement study.

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