Pharming announces completion of enrollment in pediatric clinical trial of leniolisib
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Insights
The completion of patient enrollment in Pharming Group's Phase III clinical trial for leniolisib is a significant milestone for the company, as it marks a step closer to potentially expanding the drug's indication to a younger pediatric population. The trial's successful execution could pave the way for a new treatment option for children with APDS, a demographic that currently has limited therapeutic choices.
From a medical research perspective, the focus on the primary efficacy endpoints—reduction in index lymph node size and an increase in naïve B cells—reflects a targeted approach to address the pathological manifestations of APDS. These endpoints are important for demonstrating the drug's direct impact on the disease's biomarkers and its potential to improve patients' immune function.
Furthermore, the inclusion of health-related quality of life measures as secondary endpoints is commendable. It shows a holistic understanding of the disease's impact on the patient's daily life, which is vital for evaluating the broader benefits of leniolisib beyond clinical metrics.
As a Pediatric Healthcare Specialist, the emphasis on early intervention with leniolisib in children aged 4 to 11 is noteworthy. Intervening at a younger age could indeed prevent the progression of immune-related complications associated with APDS, which can be debilitating over a lifetime.
It's also important to recognize the unique challenges faced in pediatric trials, such as ensuring the safety and tolerability of treatments in a developing immune system. The open-label design of the trial will provide valuable data on leniolisib's safety profile in this young population, which is essential for regulatory approval and eventual clinical use.
The study's international scope, with sites in the United States, Europe and Japan, enhances the diversity of the patient population and may improve the generalizability of the findings. This is particularly significant for rare diseases like APDS, where patient populations are often limited and geographically dispersed.
An analysis of the business implications of Pharming Group's announcement reveals potential for market expansion and revenue growth. The pediatric approval of leniolisib, if granted, would allow Pharming to tap into a new segment of the APDS market, possibly increasing the drug's profitability.
The strategic planning of regulatory filings starting in 2025 suggests a calculated approach to market entry, which could provide Pharming with a competitive edge in the rare disease pharmaceutical space. Moreover, the existing approval for patients 12 years and older could facilitate a smoother regulatory process for the pediatric indication due to the established safety and efficacy profile of leniolisib in an adjacent age group.
Investors and stakeholders should monitor the outcomes of this Phase III trial closely, as positive results could lead to a significant uptick in the company's valuation, while any setbacks could have the opposite effect. It's also worth noting the potential for leniolisib to become a standard of care in APDS treatment, which would have long-term implications for the company's market share and industry standing.
This multinational Phase III study is evaluating leniolisib tablets in children aged 4 to 11 years with APDS, a rare primary immunodeficiency
Leiden, the Netherlands, April 8, 2024: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces the completion of patient enrollment in 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 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. 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 clinical trial for younger pediatric patients with APDS, who have a significant unmet need for a disease modifying treatment. I am pleased with the progress made in our Phase III pediatric clinical program evaluating leniolisib in children with APDS. By intervening at a younger age, we may help prevent patients from developing immune-related complications that are likely to progress throughout their lives.”
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 Phase II/III APDS trials for patients aged 12 and older.
The first patient was dosed in November 2023 in a separate Phase III clinical trial that includes children aged 1 to 6 years with APDS to evaluate a new pediatric formulation of leniolisib. Eligible patients enrolled in either of the pediatric trials will continue to receive leniolisib for a year after the initial 12-week treatment period through an open-label extension trial.
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.8,9 Leniolisib is currently under regulatory review in the European Economic Area, Canada, Australia and Israel and a regulatory submission has been made in the U.K., with plans to pursue regulatory approval in Japan. Leniolisib is also being evaluated in two Phase III clinical trials in children 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, 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.
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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.
9. Rao VK, et al. J Allergy Clin Immunol 2024;153:265-74.R.
For further public information, contact:
Pharming Group, Leiden, The Netherlands
Michael Levitan, VP Investor Relations & Corporate Communications
T: +1 (908) 705 1696
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
Christina Renfroe
E: Christina.Renfroe@precisionvh.com
T: +1 (636) 352-7883
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