Proof of concept clinical trial will
evaluate leniolisib in PIDs with immune dysregulation linked to
altered PI3Kẟ signaling in lymphocytes
PIDs to include ALPS-FAS, CTLA4
haploinsufficiency, NFKB1 haploinsufficiency and PTEN deficiency,
with prevalence approximately five times that of
APDS
Clinical trial being conducted at the
National Institutes of Health (NIH)
Leiden, the Netherlands, October 10,
2024: Pharming Group N.V. (“Pharming” or “the Company”)
(EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces the start of a
Phase II, proof of concept, clinical trial evaluating leniolisib in
primary immunodeficiencies (PIDs) with immune dysregulation linked
to altered PI3Kẟ signaling in lymphocytes.
The clinical trial is open for enrollment and
will include PID patients with ALPS-FAS, CTLA4 haploinsufficiency,
NFKB1 haploinsufficiency and PTEN deficiency, among others. These
PID patients exhibit altered PI3Kẟ signaling in lymphocytes and
likewise display similar clinical phenotypes to activated
phosphoinositide 3-kinase delta syndrome (APDS) patients.
Epidemiology suggests a prevalence of approximately seven patients
per million in this targeted PID population, compared to one to two
patients per million for APDS.
The Phase II clinical trial is a single arm,
open-label, dose range-finding study to be conducted in
approximately 12 patients. The objectives for the trial will be to
assess safety and tolerability, pharmacokinetics, pharmacodynamics,
and explore clinical efficacy of leniolisib in the targeted PID
population. The trial has been designed to inform a subsequent
Phase III program. The Phase II clinical trial is being conducted
at the National Institute of Allergy and Infectious Diseases
(NIAID) – part of the National Institutes of Health (NIH) – with
lead investigator Gulbu Uzel, M.D., Senior Research Physician, and
co-investigator V. Koneti Rao, M.D., FRCPA, Senior Research
Physician, Primary Immune Deficiency Clinic (ALPS Clinic).
Anurag Relan, MD, MPH, Chief Medical
Officer of Pharming, commented:“The initiation of this
study is an important milestone for Pharming as it represents the
second primary immunodeficiency (PID) clinical program for
leniolisib. Based on our experience in APDS, and the significant
role of PI3Kd in regulating lymphocytes, leniolisib has the
potential to address the underlying immune dysregulation and
deficiency in a number of rare PID disorders with significant unmet
medical needs, including ALPS-FAS, CTLA4 haploinsufficiency, NFKB1
haploinsufficiency and PTEN deficiency. We are excited to be
leading this important scientific effort and to sharing the results
of the study with the medical community.”
The first patient is expected to be enrolled in
the study in the coming weeks.
This is the first clinical trial initiated by
Pharming to study leniolisib in PIDs with immune dysregulation
beyond APDS. The unique genetic drivers in ALPS-FAS, CTLA4
haploinsufficiency, NFKB1 haploinsufficiency and PTEN patients lead
to enhanced PI3Kd signaling and clinical phenotypes of immune
dysregulation shared with APDS. Specifically, PTEN patients with
immunodeficiency are frequently described as ‘APDS-like’1, patients
with ALPS-FAS display predominantly lymphoproliferative clinical
manifestations with frequent cytopenic episodes2, and CTLA4
haploinsufficiency3 as well as NFKB1 haploinsufficiency4 patients
demonstrate lymphoproliferative, cytopenic, and/or organ-specific
autoimmune/inflammatory complications of immune dysregulation.
Leniolisib is marketed in the U.S. and approved
in several other countries, for the treatment of APDS in adult and
pediatric patients 12 years of age and older.
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 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 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.5,6 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
- Tsujita Y, et al. J Allergy Clin
Immunol 2016;138:1672-80.
- Bride K & Teachey D. F1000Res.
2017;6:1928.
- Kuehn HS, et al. Science
2014;345:1623-27.
- Lorenzini T, et al. J Allergy Clin
Immunol 2020;146:901-11.
- 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 start Phase II trial of leniolisib for
PIDs_EN_10OCT24
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