Pharming announces positive topline data in pediatric clinical
trial of leniolisib
Multinational Phase III study is
evaluating leniolisib tablets in children aged 4 to 11 years with
APDS, a rare primary immunodeficiency
Data consistent with the improvements
seen in the previously reported randomized controlled trial in
adolescent and adult APDS patients
Global regulatory filings planned to
begin in 2025
Leiden, the Netherlands, December 11,
2024: Pharming Group N.V. (“Pharming” or “the Company”)
(EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces positive topline
results of data from 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 U.S. 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 data from a clinical trial for younger pediatric
patients with APDS, who have a significant unmet need for a disease
modifying treatment. Two hallmarks of APDS, lymphoproliferation and
abnormal immunophenotype, showed improvement from baseline to 12
weeks in this single arm study. More than a quarter of known APDS
patients are below the age of 12, so having a potential treatment
option for these patients who suffer from a progressive, serious
condition could be very important. We look forward to initiating
regulatory filings for these younger pediatric patients in
2025.”
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 APDS trials for patients aged 12 and older.
All 21 patients enrolled completed the 12-week
treatment period. Lymphoproliferation improved as measured by a
mean reduction in index lesion size and immunophenotype correction
was demonstrated by an increase in the percent of naïve B cells.
The improvement in lymphoproliferation and immunophenotype
correction were seen across the four dose levels being investigated
and were consistent with the improvements previously reported in
adolescent and adult patients. All treatment emergent adverse
events were reported to be mild to moderate in nature. There were
no drug related serious adverse events, and all patients completed
the 12-week treatment period.
Manish Butte, MD, PhD, E. Richard
Stiehm Endowed Chair and Professor and Division Chief, Department
of Pediatrics, Division of Immunology, Allergy and Rheumatology,
and Department of Microbiology, Immunology and Molecular Genetics
UCLA, commented:
“The 12-week data from the first clinical study evaluating
leniolisib in pediatric APDS patients is encouraging. These results
highlight the potential for leniolisib to help pediatric patients
living with APDS and their families. The pediatric APDS community
is in need of more treatment options, and we look forward to
leniolisib being one of those options.”
Full results will be presented at an upcoming
medical conference and published in a peer-reviewed journal.
Eligible patients enrolled in this study are
continuing to receive leniolisib for an additional year through an
open-label extension trial to further evaluate the safety,
tolerability, and efficacy in these patients. In addition, a
separate Phase III clinical trial including children aged 1 to 6
years with APDS is ongoing to evaluate a new pediatric formulation
of leniolisib in this younger population.
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 dysregulation1,2,3 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 leniolisib
Leniolisib is an oral small molecule phosphoinositide 3-kinase
delta (PI3Kẟ) inhibitor approved in the U.S. and several other
countries as the first and only targeted treatment indicated for
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 III clinical trial
demonstrated statistically significant improvement in the coprimary
endpoints, reflecting a favorable impact on the immune
dysregulation and deficiency seen in 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 and Australia for APDS, with plans to pursue further
regulatory approvals in Japan and South Korea. Leniolisib is also
being evaluated in two Phase III clinical trials in children with
APDS and in a Phase II clinical trial in primary immunodeficiencies
(PIDs) with immune dysregulation linked to altered PI3Kẟ signaling
in lymphocytes. The safety and efficacy of leniolisib has not been
established for PIDs with immune dysregulation beyond 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 and biologics. Pharming is headquartered
in Leiden, the 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 forward-looking statement as a result of new
information, future events or other information.
References
- Lucas CL, et al. Nat Immunol.
2014;15(1):88-97.
- Elkaim E, et al. J Allergy Clin
Immunol. 2016;138(1):210-218.
- Nunes-Santos C, Uzel G, Rosenzweig SD.
J Allergy Clin Immunol. 2019;143(5):1676-1687.
- Coulter TI, et al. J Allergy Clin
Immunol. 2017;139(2):597-606.
- Maccari ME, et al. Front Immunol.
2018;9:543.
- Jamee M, et al. Clin Rev Allergy
Immunol. 2020 Dec;59(3):323-333.
- Condliffe AM, Chandra A. Front
Immunol. 2018;9:338.
- Rao VK, et al Blood. 2023 Mar
2;141(9):971-983.
- Rao VK, et al. J Allergy Clin Immunol
2024;153:265-74.
For further public information,
contact:
Pharming Group, Leiden, the Netherlands
Michael Levitan, VP Investor Relations & Corporate
Communications
T: +1 (908) 705 1696
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
Christina Renfroe
T: +1 (636) 352-7883
E: Christina.Renfroe@precisionaq.com
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