Presentation to highlight data from the ongoing long-term
open-label extension study of leniolisib, a PI3Kδ inhibitor under
investigation for APDS, a rare primary immunodeficiency
LEIDEN, Netherlands,
Nov. 23, 2022 /PRNewswire/ --
Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT
Amsterdam: PHARM) (Nasdaq: PHAR) announces today that new clinical
data for leniolisib, an oral, selective phosphoinositide
3-kinase delta (PI3Kδ) inhibitor, an investigational treatment for
activated phosphoinositide 3-kinase delta syndrome (APDS), a rare
primary immunodeficiency, will be presented by V. Koneti Rao, MD, FRCPA, staff physician in the
Primary Immune Deficiency Clinic at the National Institute of
Health in Bethesda, Maryland, at
the 64th American Society of Hematology (ASH) Annual
Meeting and Exposition, in New Orleans,
Louisiana taking place from Saturday,
December 10 through Tuesday, December 13, 2022.
Information regarding Pharming's data presentation can be found
below, and on the ASH conference website:
https://www.hematology.org/meetings/annual-meeting.
Presentation title: Interim Analysis of Safety and
Hematological Parameters of an Ongoing Long-Term Open-Label
Extension Study of Investigational PI3Kδ Inhibitor Leniolisib for
Patients with Activated PI3Kδ Delta Syndrome (APDS) through
December 2021
Presentation type: oral
Session name: 203. Lymphocytes and Acquired or
Congenital Immunodeficiency Disorders: Delineating Immunity from
Mice to Humans
Abstract number: 608
Session date and time: Monday, December 12, 2022
from 10:30AM – 12:30PM CST
Presentation date and time: Monday, December 12,
2022 at 10:45AM CST
Location: Room: Ernest N. Morial Convention Center,
278-282
About Activated Phosphoinositide 3-Kinase
δ Syndrome (APDS)
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
PI3Kδ (phosphoinositide 3-kinase delta) pathway.1,2
Balanced signaling in the PI3Kδ 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. PI3Kδ is expressed predominately in
hematopoietic cells and is essential to normal immune system
function through conversion of
phosphatidylinositol-4-5-trisphosphate (PIP2) to
phosphatidylinositol-3-4-5-trisphosphate (PIP3). Leniolisib
inhibits the production of PIP3 and PIP3 serves as an important
cellular messenger activating AKT (via PDK1) and regulates a
multitude of cell functions such as proliferation, differentiation,
cytokine production, cell survival, angiogenesis, and metabolism.
Unlike PI3Kα and PI3Kβ, which are ubiquitously expressed, PI3Kẟ and
PI3Kγ are expressed primarily in cells of hematopoietic origin. The
central role of PI3Kẟ 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 PI3Kẟ is a valid
and potentially effective therapeutic target for immune diseases
such as APDS. 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 in patients with APDS.
About Pharming Group N.V.
Pharming Group N.V.
(EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global
biopharmaceutical company dedicated to transforming the lives of
patients with rare, debilitating, and life-threatening diseases.
Pharming is commercializing and developing an innovative portfolio
of protein replacement therapies and precision medicines, including
small molecules, biologics, and gene therapies that are in early to
late-stage development. Pharming is headquartered in Leiden,
Netherlands, and has employees
around the globe who serve patients in over 30 markets in
North America, Europe, the Middle
East, Africa, and
Asia-Pacific.
For more information, visit www.pharming.com.
Forward-Looking Statements
This press release contains forward-looking statements,
including with respect to timing and progress of Pharming's
preclinical studies and clinical trials of its product candidates,
Pharming's clinical and commercial prospects, Pharming's ability to
overcome the challenges posed by the COVID-19 pandemic to the
conduct of its business, and Pharming's expectations regarding its
projected working capital requirements and cash resources, which
statements are subject to a number of risks, uncertainties and
assumptions, including, but not limited to the scope, progress and
expansion of Pharming's clinical trials and ramifications for the
cost thereof; and clinical, scientific, regulatory and technical
developments. In light of these risks and uncertainties, and other
risks and uncertainties that are described in Pharming's 2021
Annual Report and the Annual Report on Form 20-F for the year ended
December 31, 2021 filed with the U.S.
Securities and Exchange Commission, the events and circumstances
discussed in such forward-looking statements may not occur, and
Pharming's actual results could differ materially and adversely
from those anticipated or implied thereby. Any forward-looking
statements speak only as of the date of this press release and are
based on information available to Pharming as of the date of this
release.
Inside Information
This press release relates to the disclosure of information
that qualifies, or may have qualified, as inside information within
the meaning of Article 7(1) of the EU Market Abuse
Regulation.
References
1. Lucas CL, et al. Nat Immunol. 2014; 15:88-97.
2. Elkaim E, et al. J Allergy Clin Immunol. 2016;
138(1):210-218.
3. Nunes-Santos C, Uzel G, Rosenzweig SD. J Allergy Clin Immunol.
2019; 143(5):1676-1687.
4. Coulter TI, et al. J Allergy Clin Immunol. 2017;
139(2):597-606.
5. Maccari ME, et al. Front Immunol. 2018; 9:543.
6. Jamee M, et al. Clin Rev Allergy Immunol. 2019; May 21.
7. Condliffe AM, Chandra A. Front Immunol. 2018; 9:338.
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