Pharming announces first patient enrolled in Phase III clinical trial of leniolisib for the treatment of APDS in Japan
Single-arm Phase III study in
LEIDEN,
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
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
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
In May 2023, leniolisib was granted orphan drug designation (ODD) by the Ministry of Health, Labour and Welfare of
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
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
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
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,
For more information, visit www.pharming.com and find us on LinkedIn.
Forward-Looking Statements
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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,
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,
Victoria Foster Mitchell/Alex Shaw/Amy Byrne
T: +44 203 727 1000
LifeSpring Life Sciences Communication,
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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