Pharming reports positive Phase II leniolisib results
Pharming reports positive Phase II leniolisib data in rare immune disorders and plans Phase II CVID topline readout in Q4 2026.
Rhea-AI Filing Summary
Pharming Group N.V. (PHAR) reports positive topline results from a single-arm, open-label Phase II trial of its oral PI3Kδ inhibitor leniolisib in 13 patients with genetically defined primary immunodeficiencies (PIDs) with immune dysregulation linked to PI3Kδ signaling. A late-breaking abstract of these data has been accepted for presentation at the ESID 2026 meeting in October in the Netherlands.
The study showed a favorable safety and tolerability profile, with infections as the most common adverse events and no new safety signals identified. Clinical outcomes included improvements in lymphoproliferative disease, notably a mean 26.4% reduction in spleen volume and decreases in index lesion size, indicating reduced lymphoproliferation.
Of the 13 enrolled patients, nine also had a diagnosis of common variable immunodeficiency (CVID). Pharming states that these results support continued development of leniolisib in PIDs beyond APDS and plans to present additional data at ESID 2026. Separately, the company expects to report topline Phase II results of leniolisib in CVID patients with immune dysregulation in the fourth quarter of 2026, which it says will inform plans for a potential registrational study in the broader CVID population.
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Key Figures
Phase II PID patients: 13 subjects
Mean spleen volume reduction: 26.4%
Patients with CVID diagnosis: 9 patients
+4 more
7 metrics
Phase II PID patients
13 subjects
Single-arm, open-label, intra-patient dose-escalation Phase II leniolisib study in genetically defined PIDs
Mean spleen volume reduction
26.4%
Improvement in lymphoproliferative disease observed in the Phase II leniolisib PID trial
Patients with CVID diagnosis
9 patients
Of the 13 patients enrolled in the PIDs study, nine also had common variable immunodeficiency (CVID)
ESID 2026 meeting dates
October 14–17, 2026
Dates of the 22nd Biennial ESID Meeting where the leniolisib abstract will be presented
Expected CVID Phase II topline timing
Q4 2026
Planned timing for topline results from Pharming’s Phase II leniolisib trial in CVID with immune dysregulation
APDS age approval (U.S., EU and others)
12 years and older
Leniolisib approved for APDS in adult and pediatric patients 12 years and older in multiple territories
APDS pediatric approval (U.S., 4–11 years)
At least 27 kg
Leniolisib approved in the U.S. for children 4 to 11 years of age who weigh at least 27 kg
Key Terms
primary immunodeficiencies (PIDs), common variable immunodeficiency (CVID), activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), open-label extension, +2 more
6 terms
primary immunodeficiencies (PIDs) medical
"Phase II trial with leniolisib in genetically identifiable primary immunodeficiencies (PIDs) with immune dysregulation"
common variable immunodeficiency (CVID) medical
"Phase II results for leniolisib in CVID patients with immune dysregulation"
Common variable immunodeficiency (CVID) is a chronic immune disorder in which the body makes too few protective antibodies, leaving people prone to repeated infections and other immune problems; it typically requires ongoing medical care such as immunoglobulin replacement and monitoring. Investors should care because CVID creates a long-term market for diagnostics, therapies and supportive care—like a building that needs permanent security upgrades—so advances, approvals, or changes in treatment cost or reimbursement can meaningfully affect companies in diagnostics, biologics and specialty care.
activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) medical
"approved as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)"
A rare, inherited immune disorder caused by genetic changes that leave a key immune “on” switch — the PI3Kδ enzyme — stuck in a high-activity state, which weakens infection control and can cause swollen lymph nodes, autoimmune problems and higher cancer risk. For investors, APDS matters because it defines a clearly identifiable patient group with an urgent unmet need and a specific biological target, shaping clinical trial design, regulatory pathways and the commercial opportunity for therapies aimed at correcting that overactive pathway.
open-label extension medical
"open label extension data has supported the safety and tolerability of long-term leniolisib administration"
An open-label extension is a continuation of a clinical trial where all participants and researchers know which treatment is being given, often after an initial blinded phase. It allows further study of a drug's long-term safety and effectiveness. For investors, it can indicate ongoing interest and confidence in a product's potential, influencing perceptions of its future value.
inborn errors of immunity medical
"study of leniolisib in patients with inborn errors of immunity linked to dysregulated PI3K pathway signaling"
phosphoinositide 3-kinase delta (PI3Kδ) inhibitor medical
"Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3Kδ) inhibitor"
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What did Pharming Group N.V. (PHAR) announce about its Phase II leniolisib trial?
Pharming announced positive topline data from a Phase II trial of leniolisib in 13 patients with genetically defined primary immunodeficiencies (PIDs) with immune dysregulation linked to PI3Kδ signaling, showing favorable safety, tolerability, and clinical improvements in immune dysregulation measures.
What efficacy results were reported for leniolisib in Pharming (PHAR)'s PID study?
Pharming reported clinical improvements in lymphoproliferative disease, including a mean 26.4% spleen volume reduction and reductions in the size of index lesions in the 13-patient Phase II study of leniolisib in primary immunodeficiencies with immune dysregulation.
How well was leniolisib tolerated in Pharming (PHAR)'s Phase II PID trial?
Leniolisib was described as generally well-tolerated, with a favorable safety and tolerability profile. Infections were the most common adverse events, and no new safety signals were identified compared with the known safety profile of leniolisib.
What are Pharming (PHAR)'s plans for leniolisib in CVID?
Pharming states it expects to report topline Phase II results for leniolisib in CVID patients with immune dysregulation in the fourth quarter of 2026. The company says these data will help inform plans for a potential registrational study in the broader CVID population.
Where and when will Pharming (PHAR) present the new leniolisib Phase II data?
The positive Phase II topline data have been accepted as a late-breaking abstract at the 22nd Biennial ESID Meeting, which will take place on October 14–17, 2026 in the Netherlands. Additional data from the trial will be presented there.
For which indication is leniolisib currently approved, according to Pharming (PHAR)?
Leniolisib is approved as the first and only targeted treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in specified age groups in the U.S., U.K., Australia, Israel, the EU, Canada, South Korea, and Japan; it is under regulatory review in several other countries.
AI-generated analysis. How Rhea-AI works. Not financial advice.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 6-K
REPORT OF FOREIGN PRIVATE ISSUER
PURSUANT TO RULE 13a-16 OR 15d-16
UNDER THE SECURITIES EXCHANGE ACT OF 1934
For the Month of September 2026
Commission File Number: 001-39822
Pharming Group N.V.
(Exact Name of Registrant as Specified in Its Charter)
Darwinweg 24
2333 CR Leiden
The Netherlands
(Address of principal executive offices)
Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.
Form 20-F ☒ Form 40-F ☐
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ☐
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ☐
Furnished as Exhibit 99.1 to this Report on Form 6-K is a press release of Pharming Group N.V., or the Company, dated September 22, 2026.
EXHIBIT INDEX
Exhibit No. | Description | |||||||
99.1 | Pharming announces positive Phase II topline data for leniolisib in PIDs with immune dysregulation accepted as late-breaking abstract at ESID 2026 | |||||||
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
Pharming Group N.V. | |||||||||||
By: | /s/ Fabrice Chouraqui | ||||||||||
Name: | Fabrice Chouraqui | ||||||||||
Title: | CEO | ||||||||||
Date: September 22, 2026
Pharming announces positive Phase II topline data for leniolisib in PIDs with immune dysregulation accepted as late-breaking abstract at ESID 2026
•Leniolisib was generally well-tolerated, with clinical improvements across multiple measures of immune dysregulation, including reductions in lymphoproliferation
•Separately, Pharming expects to report topline results from a Phase II trial of leniolisib in CVID in Q4 2026
Leiden, the Netherlands, September 22, 2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that a late-breaking abstract highlighting positive topline data from its Phase II trial with leniolisib in genetically identifiable primary immunodeficiencies (PIDs) with immune dysregulation linked to PI3Kd signaling has been accepted at the 22nd Biennial Meeting of the European Society for Immunodeficiencies (ESID), which will take place October 14-17, in the Netherlands.
This trial is a single-arm, open-label, intra-patient dose-escalation Phase II study of leniolisib evaluating safety and tolerability, pharmacokinetic, pharmacodynamic and efficacy measures in 13 subjects with genetically defined PIDs. The abstract will highlight leniolisib’s favorable safety and tolerability profile, as well as clinical improvements across measures of immune dysregulation. Clinical results included improvements in lymphoproliferative disease, with a mean 26.4% spleen volume reduction (SVR) and reductions in the size of index lesions. The safety observations were consistent with the known safety profile of leniolisib, with infections as the most common events observed in study subjects, and no new safety signals identified. Additional data will be presented at ESID 2026.
Of the 13 patients enrolled in the study, nine also had a diagnosis of common variable immunodeficiency (CVID). Pharming expects to report Phase II results for leniolisib in CVID patients with immune dysregulation, with or without an identified genetic cause, in the fourth quarter of 2026.
“These results mark an important step in assessing leniolisib’s potential to address immune dysregulation in PIDs beyond APDS, potentially benefiting a substantially larger patient population,” said Anurag Relan, Chief Medical Officer of Pharming. “Given PI3Kδ’s central role in immune dysregulation mechanisms, these results are encouraging and support ongoing development of leniolisib in PID patients with APDS-like manifestations. We look forward to sharing additional results from this trial at ESID, along with topline results from our separate Phase II trial in CVID, expected in the fourth quarter, which will inform our plans for a potential registrational study in the broader CVID population.”
Abstract details:
Title: Single-arm, open-label, Phase 2 study of leniolisib in patients with inborn errors of immunity linked to dysregulated PI3K pathway signaling: Topline safety and efficacy outcomes
Author: Gulbu Uzel, MD
The presentation will be available for viewing at ESID 2026 for the full duration of conference.
About leniolisib
Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor approved 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 in the U.S., U.K., Australia, Israel, the EU, Canada, and South Korea; in children 4 to 11 years of age who weigh at least 27 kg in the U.S., and for patients 4 years of age and older in Japan.
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 III clinical trial demonstrated statistically significant improvement in the coprimary endpoints, reflecting a favorable impact on the immune dysregulation and deficiency seen in these patients, and open label extension data has supported the safety and tolerability of long-term leniolisib administration.1,2
Leniolisib is currently under regulatory review for the treatment of APDS in several other countries. Leniolisib is also being evaluated in two Phase II clinical trials in primary immunodeficiencies (PIDs) with immune dysregulation. The safety and efficacy of leniolisib has not been established for PIDs with immune dysregulation beyond APDS.
About Pharming
Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global biotechnology company that develops and commercializes innovative medicines for people living with rare immune and genetic diseases.
We combine specialized scientific, medical, regulatory and commercial expertise to advance a focused portfolio of approved medicines and development programs that address significant unmet medical needs. Guided by insights from patients and the wider rare disease community, we are dedicated to delivering innovative therapies for some of the most challenging rare diseases.
Pharming is headquartered in Leiden, the Netherlands, with operations in the United States and Europe.
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 2025 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2025, 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 forward-looking statement as a result of new information, future events or other information.
References
1.Rao VK, et al. Blood. 2023;141(9):971-983.
2.Rao VK, et al. J Allergy Clin Immunol 2024;153:265-74.
For further public information, contact:
Pharming
Michael Levitan, VP Investor Relations & Capital Markets
T: +1 (908) 705 1696
E: investor@pharming.com
Saskia Mehring, Head of Corporate Communications
T: +31 6 28 32 60 41
E: media.relations@pharming.com
Media Relations
Julia Deutsch (Lyra Strategic Advisory on behalf of Pharming)
E: JDeutsch@lyraadvisory.com
Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming)
T: +31 6 53 81 64 27