Single-arm Phase III study in Japan evaluating leniolisib in patients aged
12 years and older with APDS, a rare primary
immunodeficiency
LEIDEN, The Netherlands,
Aug. 9, 2023 /PRNewswire/ -- Pharming
Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam:
PHARM/Nasdaq: PHAR) announces that the first patient has been
enrolled in its Phase III clinical trial in Japan evaluating leniolisib for the treatment
of activated phosphoinositide 3-kinase delta syndrome (APDS) in
adult and pediatric patients 12 years of age and older.
Pharming's single-arm, open-label clinical trial will evaluate
the safety, tolerability, and efficacy of leniolisib in
three patients 12 years of age and older who have a confirmed
APDS diagnosis. Each patient will receive weight-based dosing up to
70mg of leniolisib twice daily for 12 weeks. The study's primary
efficacy endpoints and secondary endpoints mirror those used to
evaluate the clinical outcomes in each of the leniolisib APDS
trials.
Pharming plans to include data from the trial in a future
registration application for the approval of leniolisib to be filed
with Japan's Pharmaceuticals and
Medical Devices Agency (PMDA). Eligible patients enrolled in the
trial will continue to receive the investigational drug for at
least one year through an open-label extension trial.
Hirokazu Kanegane, Professor of the
Department of Child Health and Development, Tokyo Medical and
Dental University, commented:
"The
initiation of this clinical study is a positive step for the APDS
community in Japan. With patients
currently reliant on supportive treatments, the prospect of a
disease-modifying treatment for this rare primary immunodeficiency
could represent an exciting new treatment option for patients,
their families, caregivers and their doctors in Japan."
Anurag Relan, MD, MPH,
Chief Medical Officer of Pharming,
commented:
"Building on the
success of our multinational Phase II/III study of leniolisib in
patients with APDS 12 years of age and older, I am pleased to
confirm the initiation of our Phase III trial supporting the same
population in Japan. By conducting
this study, Pharming's goal is to introduce an oral treatment
option that has the potential to alter the course of disease for
patients with APDS, a rare and progressive disease, in Japan. Following the FDA's recent approval of
Joenja® in the U.S., we are working with regulatory
authorities to expand access to this targeted treatment for
patients across the globe through additional market
authorizations."
In May 2023, leniolisib was
granted orphan drug designation (ODD) by the Ministry of Health,
Labour and Welfare of Japan (MHLW)
for the treatment of APDS. There is currently no approved
therapy in Japan for this complex
and progressive disease.
The MHLW's ODD system promotes the research and development of
investigational drugs designed to treat diseases associated with
significant unmet medical need and which affect fewer than 50,000
patients across Japan.
Investigational drugs granted ODD in Japan benefit from additional guidance and
subsidies for research and development activities, consultation for
clinical development, and priority review of marketing
authorization applications.
Leniolisib received regulatory approval from the United States
Food and Drug Administration (FDA) for the treatment of APDS in
patients 12 years of age or older in March
2023 and was commercially launched under the brand name
Joenja® in the U.S. in April
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 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 Joenja® (leniolisib)
Joenja® (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. Joenja® 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 Joenja® 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 Joenja®
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. For
information about Joenja®, visit: Joenja.com
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
1. 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.
For further public information, contact:
Pharming Group, Leiden, The
Netherlands
Michael Levitan, VP Investor Relations
& Corporate Communications
T: +1 (908) 705 1696
Heather Robertson, Investor
Relations & Corporate Communications Manager
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
Ethan Metelenis
E: Ethan.Metelenis@precisionvh.com
T: +1 (917) 882 9038
EU PR
Claire Dobbs
E: claire.dobbs@solarishealth.com
T: +44 7864 640093
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