LEIDEN, Netherlands, April 11,
2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming")
(Euronext Amsterdam: PHARM) (Nasdaq: PHAR) announces the first
commercial shipments of Joenja® (leniolisib) to patients in
the United States. Joenja®, an
oral, selective PI3Kδ inhibitor, is the first and only treatment
approved in the U.S. for activated phosphoinositide 3-kinase delta
(PI3Kδ) syndrome (APDS), a rare and progressive primary
immunodeficiency, in adult and pediatric patients 12 years of age
and older.
Under the terms of Pharming's 2019 exclusive license agreement
with Novartis for leniolisib, the corresponding first commercial
sale of Joenja® triggers a $10
million milestone payment by Pharming to Novartis.
Stephen Toor,
Chief Commercial Officer of
Pharming, commented:
"We are pleased to announce that
the first Joenja® shipments to
patients, with payor
reimbursement, were delivered
approximately two weeks following
FDA approval, achieving an important milestone for
patients suffering with APDS. We look forward to making
Joenja® widely available across the U.S. as the first
and only approved treatment for patients with
APDS."
About Activated Phosphoinositide 3-Kinase δ Syndrome
(APDS)
APDS is a rare primary immunodeficiency that was first
characterized in 2013. APDS is caused by variants in either one of
two identified genes known as PIK3CD or PIK3R1, which are vital to
the development and function of immune cells in the body. Variants
of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide
3-kinase delta) pathway, which causes immune cells to fail to
mature and function properly, leading to immunodeficiency and
dysregulation.1,2,3 APDS is characterized by a variety
of symptoms, including 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, it has been
reported that 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 A definitive diagnosis can be made through
genetic testing. APDS affects approximately 1 to 2 people per
million worldwide.
About Joenja® (leniolisib)
Joenja® (leniolisib) is an oral small molecule phosphoinositide
3-kinase delta (PI3Kẟ) inhibitor approved in the US as the first
and only targeted treatment of activated phosphoinositide 3-kinase
delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12
years of age and older. Joenja® inhibits the production of
phosphatidylinositol-3-4-5-trisphosphate, which serves as an
important cellular messenger and regulates a multitude of cell
functions such as proliferation, differentiation, cytokine
production, cell survival, angiogenesis, and metabolism. Results
from a randomized, placebo-controlled Phase II/III clinical trial
demonstrated clinical efficacy of Joenja® in the co-primary
endpoints; demonstrating statistically significant impact on immune
dysregulation and normalization of immunophenotype within these
patients, and interim open label extension data has supported the
safety and tolerability of long-term Joenja®
administration.8 Leniolisib is currently under
regulatory review by the European Medicines Agency, with plans to
pursue further regulatory approvals in the UK, Canada, Australia and Japan. Leniolisib is also being evaluated in a
Phase III clinical trial in children aged 4 to 11 with APDS, with a
further trial planned in children aged 1 to 6 years with
APDS. For information about Joenja®, visit:
Joenja.com
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 and find us on
LinkedIn.
Forward-looking Statements
This press release may contain forward-looking statements.
Forward-looking statements are statements of future expectations
that are based on management's current expectations and assumptions
and involve known and unknown risks and uncertainties that could
cause actual results, performance, or events to differ materially
from those expressed or implied in these statements. These
forward-looking statements are identified by their use of terms and
phrases such as "aim", "ambition", ''anticipate'', ''believe'',
''could'', ''estimate'', ''expect'', ''goals'', ''intend'',
''may'', "milestones", ''objectives'', ''outlook'', ''plan'',
''probably'', ''project'', ''risks'', "schedule", ''seek'',
''should'', ''target'', ''will'' and similar terms and phrases.
Examples of forward-looking statements may include statements with
respect to timing and progress of Pharming's preclinical studies
and clinical trials of its product candidates, Pharming's clinical
and commercial prospects, 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. All
forward-looking statements contained in this press release are
expressly qualified in their entirety by the cautionary statements
contained or referred to in this section. Readers should not place
undue reliance on forward-looking statements. 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. Pharming does not undertake any obligation to publicly
update or revise any.
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(1):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.
8. RAO VK, et al Blood. 2023 Mar
2;141(9):971-983.
For further public information, contact:
Pharming Group, Leiden, The
Netherlands
Michael Levitan, VP Investor Relations & Corporate
Communications
T: +1 (908) 705 1696
Heather Robertson, Investor
Relations & Corporate Communications Manager
E: investor@pharming.com
FTI Consulting, London,
UK
Victoria Foster
Mitchell/Alex
Shaw/Amy Byrne
T: +44 203 727 1000
LifeSpring Life Sciences Communication, Amsterdam, The Netherlands
Leon Melens
T: +31 6 53 81 64 27
E: pharming@lifespring.nl
US PR
Ethan Metelenis
E: Ethan.Metelenis@precisionvh.com
T: +1 (917) 882 9038
EU PR
Claire Dobbs
E: claire.dobbs@solarishealth.com
T: +44 7864 640093
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