Hoppa pb met de details van de fase 3 studie. Vind de inhoud altijd erg lastig als leek, maar de toon is erg positief. Benieuwd of er nog een leuke stijging van het aandeel plaats vind. Hier het persbericht:
Pharming Announces Positive Data from Phase II/III Leniolisib Trial Presented at Clinical Immunology Society 2022 Annual Meeting
Leiden, The Netherlands, April 1, 2022
Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces new data from the pivotal Phase II/III trial of leniolisib for the treatment of activated phosphoinositide 3-kinase delta (PI3Kd) syndrome (APDS), a primary immunodeficiency. Principal Investigator V. Koneti Rao, M.D., a staff physician in the Primary Immune Deficiency Clinic at the National Institutes of Health in Bethesda, Maryland, shared the findings in a presentation at the Clinical Immunology Society (CIS) 2022 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, met its co-primary endpoints, which evaluated reduction in lymph node size and correction of immunodeficiency. The shrinking of lymphadenopathy lesions and increased proportion of naïve B cells are important in this population, as they indicate a reduction in APDS disease markers. Presented for the first time at CIS, the co-primary endpoints at day 85 after baseline demonstrated:
In the index lymphadenopathy lesions, a statistically significant adjusted mean change in the log10 transformed sum of product of diameters (SPD) of -0.30 among patients who received leniolisib compared with -0.06 among patients who received placebo (95% CI: -0.37, -0.11; p=0.0012).
From a baseline level of ?48%, an increase of 34.76% in the proportion of naïve B cells in patients who received leniolisib versus a -5.37% decrease in patients who received placebo (95% CI: 28.51, 51.75; p?0.0001).
The study drug was well-tolerated. There were no adverse events that led 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 study treatment.
Charlotte Cunningham-Rundles, M.D., Ph.D., David S. Gottesman Professor of Immunology at the Mount Sinai School of Medicine in New York, said:
“It is great news that leniolisib has achieved such positive results in this Phase III study in APDS. It is extremely encouraging to see that this medication is capable of targeting the cause of this difficult disease, both improving care and reducing patients’ symptoms. Progress toward a treatment that is tailor-made for our patients with APDS is a milestone we have long awaited.”
Pharming plans to begin submitting global regulatory filings for leniolisib in the second quarter of 2022 and, subject to approval, launching the treatment in the U.S. in the first quarter of 2023 and starting a series of European launches in the second half of 2023.
Anurag Relan, Chief Medical Officer of Pharming, commented:
“Pharming is delighted that leniolisib achieved significance in both co-primary endpoints and was well tolerated in these APDS patients, as the product’s approval would address an unmet need for those with this rare disease, who currently rely on supportive therapies such as antibiotics and immunoglobulin replacement therapy. In addition to working closely with regulatory authorities across the globe to make leniolisib available to immunologists, hematologists, and their patients, we will continue to develop this treatment through our open-label extension study and two additional studies that will enroll children under the age of 12, as well as potentially extending the geographic reach of the product.”
About Activated Phosphoinositide 3-Kinase d 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 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.1,2 Balanced signaling in the PI3Kd pathway is essential for physiological immune function. When this pathway is hyperactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.1,3 APDS is characterized by severe, recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms can be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and suffer a median 7-year diagnostic delay.6 As APDS is a progressive disease, this delay may lead to an accumulation of damage over time, including permanent lung damage and lymphoma.4-7 The only way to definitively diagnose this condition is through genetic testing.
About leniolisib
Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially anti-neoplastic activities. Leniolisib inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an important cellular messenger specifically activating AKT (via PDK1) and regulates a multitude of cell functions such as proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3Ka and PI3Kß, which are ubiquitously expressed, PI3K? and PI3K? are expressed primarily in cells of hematopoietic origin. The central role of PI3K? in regulating numerous cellular functions of the adaptive immune system (B-cells and, to a lesser extent, T cells) as well as the innate immune system (neutrophils, mast cells, and macrophages) strongly indicates that PI3K? is a valid and potentially effective therapeutic target for several immune diseases.
To date, leniolisib has been well tolerated during both the Phase 1 first-in-human trial in healthy subjects and the Phase II/III registration-enabling study.