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 leniolisibLeniolisib 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 NetherlandsMichael
Levitan, VP Investor Relations & Corporate CommunicationsT: +1
(908) 705 1696E: investor@pharming.com
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 RenfroeT: +1 (636) 352-7883E:
Christina.Renfroe@precisionaq.com
- Pharming announces topline data pediatric clinical trial
leniolisib_EN_11DEC24
Grafico Azioni Pharming Group NV (EU:PHARM)
Storico
Da Dic 2024 a Gen 2025
Grafico Azioni Pharming Group NV (EU:PHARM)
Storico
Da Gen 2024 a Gen 2025
