Pharming announces first patient dosed in pediatric clinical trial
for children aged 1 to 6 years for leniolisib
The multinational Phase III study is
evaluating a new pediatric granulated formulation of leniolisib in
children aged 1 to 6 years with APDS, a rare primary
immunodeficiency
Leiden, The Netherlands, November 21,
2023: Pharming Group N.V. (“Pharming” or “the Company”)
(EURONEXT Amsterdam: PHARM)/(Nasdaq: PHAR) announces that the first
patient has been dosed in its Phase III pediatric clinical trial
evaluating a new pediatric formulation of the investigational drug
leniolisib, an oral, selective phosphoinositide 3-kinase delta
(PI3Kδ) inhibitor, in children ages one to six years, with
activated phosphoinositide 3-kinase delta syndrome (APDS).
At sites in the U.S., Japan, and the EU, the
single-arm, open-label, multinational clinical trial will evaluate
the safety, tolerability, and efficacy of leniolisib in 15
children, one to six years of age, who have a confirmed APDS
diagnosis. These patients will receive a specific, pediatric
granulated formulation of leniolisib. The study’s primary efficacy
endpoints and secondary endpoints mirror those used to evaluate the
clinical outcomes in previous leniolisib Phase II/III APDS trials
for patients aged 12 and older.
Dr. Eveline Wu, MD, MSCR, Division
Chief, Paediatric Rheumatology & Associate Professor of
Paediatric Rheumatology and Allergy/Immunology at The University of
North Carolina School of Medicine,
commented:
“As a physician who has seen how significantly
APDS can affect children’s lives, I’m excited that a targeted
treatment may soon become available for even younger patients.
While standard supportive therapies can ease some of the
progressive, immune-related symptoms that characterize APDS,
leniolisib is designed to help prevent those symptoms from arising.
This could improve the standard of care for these children with the
hope of a more full and enjoyable participation in the typical
activities of childhood.”
Anurag Relan, MD, MPH, Chief Medical
Officer of Pharming, commented:
“With the first patient dosed in this pediatric
trial of leniolisib, using a new, granulated formulation, we are
continuing to demonstrate Pharming’s ongoing commitment to
providing new treatment options for younger pediatric patients with
APDS. We look forward to generating the necessary supporting data
to facilitate additional regulatory filings, thereby ensuring that
patients from as broad an age range as possible can potentially
benefit from this disease-modifying treatment.”
This is the second pediatric clinical trial
launched by Pharming this year for the study of leniolisib in
pediatric patients with APDS. The first pediatric study, announced
in February 2023, is ongoing and will evaluate 15 children, four to
11 years of age, receiving leniolisib’s tablet formulation as an
investigational treatment for 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.
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 leniolisib
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. 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
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.
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 2022 Annual Report and the Annual
Report on Form 20-F for the year ended December 31, 2022, 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
- 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. 2019;May 21.
- Condliffe AM,
Chandra A. Front Immunol. 2018;9:338.
- RAO VK, et al
Blood. 2023 Mar 2;141(9):971-983.
For further public information,
contact:
Pharming Group, Leiden, The NetherlandsMichael
Levitan, VP Investor Relations & Corporate CommunicationsT: +1
(908) 705 1696
Heather Robertson, Investor Relations &
Corporate Communications ManagerT: +31 71 524 7400 E:
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 PREthan MetelenisE:
Ethan.Metelenis@precisionvh.comT: +1 (917) 882 9038
EU PRClaire DobbsE: claire.dobbs@solarishealth.com
T: +44 7864 640093
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