EMA accelerated assessment allows a
shorter review period for leniolisib from a standard 210 days to
150 days
Pharming is on track to submit its Marketing Authorisation
Application for leniolisib in H2 2022
LEIDEN, the Netherlands, Aug. 1, 2022
/PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the
Company") (EURONEXT Amsterdam: PHARM) (Nasdaq: PHAR) announces that
the European Medicines Agency's (EMA) Committee for Medicinal
Products for Human Use (CHMP) has granted an accelerated
assessment for the Marketing Authorisation Application (MAA) for
leniolisib. Leniolisib has been studied for the treatment of
activated PI3K delta syndrome (APDS), a rare primary
immunodeficiency, in adults and adolescents age 12 or older in the
European Economic Area (EEA). Pharming is on track and plans to
submit its MAA for leniolisib to the EMA in October 2022.
Accelerated assessment reduces the timeframe for the CHMP
to review an MAA from 210 days to 150 days. The EMA will
grant, upon request, accelerated assessment of an MAA if they
decide the product is of major interest for public health and
therapeutic innovation.
The clinical development for leniolisib includes positive data
from a Phase II/III study of the product, which met both its
co-primary endpoints in the target patient population of evaluated
reduction in lymph node size and correction of immunodeficiency.
The primary efficacy results demonstrated clinical efficacy of
leniolisib over placebo with a statistically significant reduction
from baseline in the log10 transformed sum of product of diameters
(SPD) in the index lymphadenopathy lesions (p=0.0012) and
normalization of immune dysfunction, as evidenced by increased
proportion of naïve B cells from baseline (p<0.0001). The
shrinking of lymphadenopathy lesions and increased proportion of
naïve B cells are important in patients as they indicate a
reduction in APDS disease markers.
In the study, leniolisib was generally well-tolerated, with the
majority of reported adverse events in both treatment groups
classified as mild. 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 the placebo group. None of the SAEs were
suspected to be related to study treatment.
Anurag Relan, Chief Medical
Officer of Pharming, commented:
"The acceptance of an accelerated regulatory review for
leniolisib underlines the high unmet need for patients with APDS,
with the product potentially being the first approved treatment for
this rare disease. This is an important milestone for the APDS
community and for Pharming and is built on the successful Phase
II/III data, which we first reported in February 2022. We remain focused on progressing
leniolisib through the regulatory review process, with our MAA on
track for submission in October of this year, as we seek to make
this important new product available to immunologists,
hematologists, and their patients in Europe."
About Activated Phosphoinositide 3-Kinase
δ 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 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 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 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 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.
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, et al. 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.
2020;59(3):323-333.
7. Condliffe AM, Chandra A. Front Immunol. 2018;9:338.
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