Pharming Group Initiates Phase II Study of Leniolisib for CVID

Pharming Group Launches Phase II Clinical Trial of Leniolisib

Multi-center clinical trial includes various locations worldwide.

Exploration of leniolisib for primary immunodeficiencies expands treatment prospects.

Common variable immunodeficiency (CVID) affects many, with a notable global prevalence.

Leiden, the Netherlands: Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is excited to announce that the first patient has been dosed in a Phase II clinical trial to evaluate the effectiveness of leniolisib in patients suffering from common variable immunodeficiency (CVID) coupled with immune dysregulation.

This Phase II trial is a single-arm, open-label study that aims to determine the efficacy and safety of leniolisib by treating approximately 20 patients aged 12 and older diagnosed with CVID. Participants must show signs of lymphoproliferation and at least one other manifestation of immune dysregulation, such as interstitial lung disease, autoimmune cytopenias, or enteropathy. The trial's objectives focus on assessing safety, tolerability, pharmacokinetics, pharmacodynamics, and examining the clinical efficacy of leniolisib. Findings from this study will provide valuable insights that will assist in planning future Phase III trials. Dr. Jocelyn Farmer, M.D./Ph.D., directs the Clinical Immunodeficiency Program at Beth Israel Lahey Health and leads this Phase II study, incorporating clinical sites in the U.S., U.K., and EU.

Insights from Dr. Jocelyn Farmer
Dr. Jocelyn Farmer shared her perspectives on the disease burden endured by CVID patients, noting, "In my experience overseeing a large group of CVID patients, the complications arising from this condition, particularly the autoimmune disorders and end-organ involvement, present significant challenges. The lack of effective therapies means these patients can experience worsening symptoms, increasing mortality risk. With leniolisib's mechanism as a multi-faceted lymphocyte regulator, it holds considerable promise for this population. I am thrilled that we have commenced treatment with leniolisib in this Phase II trial, which could provide crucial support for a group with a significant unmet medical need."

CVID is recognized as the largest category within symptomatic primary immunodeficiency (PID) patients, where around half suffer from various complications due to immune dysregulation. The mortality rate for CVID patients dealing with immune dysregulation is significantly higher compared to those with infections alone, presenting an unmet medical need.

Available data suggests that the global prevalence of CVID patients with immune dysregulation stands at approximately 39 per million.

Comments from Anurag Relan, CMO of Pharming
Anurag Relan highlighted the importance of this trial, stating, "By initiating this second Phase II clinical trial beyond the APDS indication, we are greatly expanding our focus on primary immunodeficiencies. Unlike our initial trials which were centered on genetic criteria, CVID patients are diagnosed based on clinical findings. The unmet clinical needs are substantial, and we’re encouraged by the potential of leniolisib to address the treatment void for CVID patients with immune dysregulation. We eagerly anticipate the enrollment of more participants in the upcoming periods."

Leniolisib, marketed as Joenja® in the U.S., is the first and only treatment targeting activated phosphoinositide 3-kinase delta (PI3K?) syndrome (APDS) in both adult and pediatric patients aged 12 and older. This small molecular inhibitor plays a crucial role in regulating various cell functions, including cell growth, cytokine production, and immune response. Results from preceding Phase III trials have showcased statistically significant improvements in the disease's manifestation, supporting the safety of long-term leniolisib use.

About Pharming Group N.V.
Headquartered in Leiden, the Netherlands, Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) dedicates itself to enhancing the lives of patients afflicted with rare and critical diseases. The company innovates in developing therapies, prioritizing protein replacement treatments and precision medicines in various global markets, including North America, Europe, and Asia-Pacific.

For further information, please reach out to:
Pharming Group, Leiden, the Netherlands
Michael Levitan, VP Investor Relations & Corporate Communications
T: +1 (908) 705 1696
E: investor@pharming.com

Frequently Asked Questions

What is the purpose of the Phase II trial for leniolisib?

The trial aims to evaluate the safety, tolerability, and clinical efficacy of leniolisib in patients with CVID and immune dysregulation.

Who is leading the clinical trial?

The clinical trial is led by Dr. Jocelyn Farmer, who specializes in clinical immunodeficiency.

What population is targeted in this trial?

Patients aged 12 years and older diagnosed with CVID showing signs of immune dysregulation will be enrolled.

Why is leniolisib significant for CVID patients?

Leniolisib addresses the unmet medical needs of CVID patients by potentially offering an effective treatment option.

What is Pharming Group’s mission?

Pharming Group aims to improve lives for patients with rare and life-threatening conditions through innovative therapies.

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