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Pharming Group provides an update on the ongoing regulatory review of leniolisib for the treatment of APDS in the European Union

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Pharming Group NVPharming Group NV

Pharming Group NV

Leiden, the Netherlands, May 30, 2024: : Pharming Group NV (“Pharming” or the “Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) today announces an update on the ongoing review of its Marketing Authorization Application (MAA) for leniolisib for the treatment of adult and pediatric patients 12 years of age life and elderly with activated phosphoinositide delta 3-kinase syndrome (APDS) approved by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

Following the CHMP meeting on May 27-30, Pharming received an updated list of outstanding issues (LoOI) from the CHMP. The LoOI confirmed the positive clinical benefits and safety of leniolisib, as assessed by the Ad Hoc Expert Group (AEG), and included one remaining chemical, manufacturing and control (CMC) application.

The CMC proposal concerns the definition of regulatory starting materials used in the manufacturing process of leniolisib. As Pharming is committed to meeting all specific CHMP requirements, additional data and quality controls were provided and Pharming proposed to implement the CMC request upon approval. The CHMP requested that this work be completed before approving the medicine and granted Pharming an extension until January 2026 to submit its response. Pharming has already started the manufacturing activities required by the CHMP and plans to complete them before this deadline.

Sijmen de Vries, MD, CEO of Pharming, commented:
“While we are understandably disappointed by the delay in granting the European license, we are pleased that the CHMP found the clinical benefits of leniolisib to be positive. The efficacy and safety demonstrated in clinical trials and current experience from over 300 patient-years of treatment confirm that leniolisib addresses unmet medical needs. We will continue to work closely with the EMA and CHMP to obtain approval for leniolisib in Europe for people with APDS. In the meantime, all of our clinical development and early access programs will continue.”

The MAA for leniolisib was based on the results of an international, randomized, triple-blind, placebo-controlled Phase II/III clinical trial that achieved both co-primary endpoints. The study assessed the effectiveness and safety of 31 patients diagnosed with APDS aged 12 years and older. The application also submitted data from a long-term, open-label, extension clinical trial in which 37 patients received leniolisib for an average of three years.

Leniolisib is currently commercially available in the United States. The U.S. Food and Drug Administration (FDA) approved leniolisib in March 2023 based on an assessment that leniolisib meets clinical and manufacturing standards.

Pharming maintains its previously provided total revenue guidance for 2024.

About activated phosphoinositide 3-kinase δ syndrome (APDS)
APDS is a rare primary immunodeficiency disorder that was first characterized in 2013. APDS is caused by variants of one of two identified genes, known as PIK3CD Or PIK3R1, which are necessary for the development and functioning of immune cells in the body. Variants in these genes lead to overactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway, which causes immune cells to fail to mature and function properly, leading to immune deficiency 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 often misdiagnosed, resulting in an average delay in diagnosis of 7 years.6 Because APDS is a progressive disease, this delay can lead to accumulation of damage over time, including permanent lung damage and lymphoma.4-7 The final diagnosis can be made based on genetic testing. APDS affects approximately 1 to 2 people in a million worldwide.

About leniolisib
Leniolisib is an oral small molecule inhibitor of phosphoinositide delta 3-kinase (PI3Kδ) approved in the US and Israel as the first and only targeted drug for the treatment of activated phosphoinositide delta 3-kinase (PI3Kδ) syndrome (APDS) in adults and children aged 12 years and older . Leniolisib inhibits the production of phosphatidylinositol 3-4-5-triphosphate, which serves as an important cell messenger and regulates many cellular 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 on similar primary endpoints; demonstrated a statistically significant effect on immune dysregulation and normalization of the immunophenotype in these patients, and interim open-label extension data confirmed the safety and tolerability of long-term administration of leniolisib.8.9 Leniolisib is currently under regulatory review in the European Economic Area, the United Kingdom, Canada and Australia, with plans to obtain further regulatory approvals in Japan and South Korea. Leniolisib is also being evaluated in two Phase 3 clinical trials in children with APDS.

Information about Pharming Group NV
Pharming Group NV (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company committed to transforming the lives of patients suffering from rare, devastating and life-threatening diseases. Pharming deals with the commercialization and development of an innovative portfolio of protein replacement therapies and precision medicines, including small molecule and biological drugs. Pharming is headquartered in Leiden, the Netherlands, with global employees serving patients in more than 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 about future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by the use of terms and phrases such as “goal”, “ambition”, “anticipate”, “believe”, “could”, “estimate”, “expect”, “targets”, “intend” , “may”, “milestones”, “goals”, “outlook”, “plan”, “probably”, “project”, “risk”, “schedule”, “search”, “should”, “target”, “will” and similar terms and expressions. Examples of forward-looking statements may include statements regarding the timing and progress of Pharming’s preclinical and clinical development of product candidates, Pharming’s clinical and commercial prospects, and Pharming’s expectations regarding anticipated working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, without limitation, the scope, progress and development of Pharming’s clinical trials and the cost implications thereof; and clinical, scientific, regulatory, commercial, competitive and technical developments. In light of these risks and uncertainties and the other risks and uncertainties described in Pharming’s 2023 Annual Report and 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 may differ materially and adversely from those anticipated or implied. All forward-looking statements contained in this press release are expressly qualified in their entirety by the cautionary statements contained or referenced 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 press release. Pharming has no obligation to publicly update or correct any content.

Inside information
This press release concerns the disclosure of information that qualifies or may qualify as confidential information within the meaning of Art. 7 section 1 of the EU Market Abuse Regulation.

Bibliography

  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 Immunol for allergies. Dec 2020;59(3):323-333.

  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.

For further public information please contact:
Pharming Group, Leiden, The Netherlands
Michael Levitan, vice president of investor relations and corporate communications
T: +1 (908) 705 1696
E: [email protected]

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: [email protected]

USA PR
Christina Renfroe
E: [email protected]
T: +1 (636) 352-7883