Pharming announces completion of enrollment in pediatric clinical
trial of leniolisib
This multinational Phase III study is
evaluating leniolisib tablets in children aged 4 to 11 years with
APDS, a rare primary immunodeficiency
Leiden, the Netherlands, April 8,
2024: Pharming Group N.V. (“Pharming” or “the Company”)
(EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces the completion
of patient enrollment in its Phase III clinical trial (NCT05438407)
evaluating the investigational drug leniolisib, an oral, selective
phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children aged
4 to 11 years with activated phosphoinositide 3-kinase delta
syndrome (APDS).
Leniolisib, marketed under the brand name
Joenja® in the U.S., received approval from the US Food and Drug
Administration (FDA) for the treatment of APDS in adult and
pediatric patients 12 years of age and older in March 2023.
Pharming plans to include data from this 4–11-year-old trial in
regulatory filings worldwide for the approval of leniolisib for
pediatric patients with APDS, beginning in 2025.
Anurag Relan, MD, MPH, Chief Medical
Officer of Pharming, commented:
“This is the first clinical trial for younger
pediatric patients with APDS, who have a significant unmet need for
a disease modifying treatment. I am pleased with the progress made
in our Phase III pediatric clinical program evaluating leniolisib
in children with APDS. By intervening at a younger age, we may help
prevent patients from developing immune-related complications that
are likely to progress throughout their lives.”
The study enrolled 21 children with APDS ages 4
to 11 years at sites in the United States, Europe, and Japan. The
single-arm, open-label clinical trial is evaluating the safety,
tolerability, and efficacy of leniolisib. The study’s primary
efficacy endpoints are a reduction in index lymph node size and an
increased proportion of naïve B cells out of total B cells from
baseline at 12 weeks. Secondary endpoints include an assessment of
the ability of leniolisib to modify health-related quality of life
based on measures of physical, social, emotional, and school
functioning using a validated patient questionnaire. These
endpoints mirror those used to evaluate the clinical outcomes in
previous leniolisib Phase II/III APDS trials for patients aged 12
and older.
The first patient was dosed in November 2023 in
a separate Phase III clinical trial that includes children aged 1
to 6 years with APDS to evaluate a new pediatric formulation of
leniolisib. Eligible patients enrolled in either of the
pediatric trials will continue to receive leniolisib for a year
after the initial 12-week treatment period through an open-label
extension trial.
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 leniolisibLeniolisib 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. Leniolisib 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 leniolisib in the coprimary
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 leniolisib administration.8,9
Leniolisib is currently under regulatory review in the European
Economic Area, Canada, Australia and Israel and a regulatory
submission has been made in the U.K., with plans to pursue
regulatory approval in Japan. Leniolisib is also being evaluated in
two Phase III clinical trials in children 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 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, commercial,
competitive and technical developments. In light of these risks and
uncertainties, and other risks and uncertainties that are described
in Pharming's 2023 Annual Report and the Annual Report on Form 20-F
for the year ended December 31, 2023, 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 InformationThis 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.
References1. 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):971-983.9. Rao VK, et al. J Allergy Clin Immunol
2024;153:265-74.R.
For further public information,
contact:Pharming Group, Leiden, The NetherlandsMichael
Levitan, VP Investor Relations & Corporate CommunicationsT: +1
(908) 705 1696
FTI Consulting, London, UKVictoria Foster
Mitchell/Alex Shaw/Amy ByrneT: +44 203 727 1000
LifeSpring Life Sciences Communication, Amsterdam,
The NetherlandsLeon MelensT: +31 6 53 81 64 27E:
pharming@lifespring.nl
US PRChristina RenfroeE:
Christina.Renfroe@precisionvh.com T: +1 (636) 352-7883
Pharming Group NV (EU:PHARM)
Historical Stock Chart
From Oct 2024 to Nov 2024
Pharming Group NV (EU:PHARM)
Historical Stock Chart
From Nov 2023 to Nov 2024
