- Pharming announces that the European Medicines Agency (EMA) is validating its application for marketing authorization under accelerated evaluation for leniolisib

Marketing authorization in the European Economic Area scheduled for the first half of 2023

LEIDEN, The Netherlands, Oct. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the company") (EURONEXT Amsterdam: PHARM / Nasdaq: PHAR) announces today that its Marketing Authorization Application (MAA) for leniolisib has been validated for scientific evaluation under an accelerated evaluation by the liaison of the European Medicines Agency (EMA). The application, filed in early October 2022, is for the investigational drug, leniolisib, an oral selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS). ), a rare primary immunodeficiency, in adolescents and adults older than 12 years.

In August 2022, Pharming announced that the EMA CHMP granted an accelerated review to the MAA of leniolisib. Expedited review reduces the review time from 210 days to 150 days. Upon request, the EMA will grant an accelerated evaluation of an MAA if it decides that the product is of great interest for public health and, in particular, from the point of view of therapeutic innovation. The marketing authorization for leniolisib in the European Economic Area is scheduled for the first half of 2023.

The MAA is backed by positive data from a phase II/III study of leniolisib, announced Feb. 2, 2022, which met its coprimary evaluation criteria of reduction in lymph node size and increase in the percentage of virgin B cells in patients with APDS. In addition, the study safety data showed that participants tolerated leniolisib well. Also presented as part of the MAA were data from a long-term open-label extension clinical trial in patients with APDS treated with leniolisib.

Anurag Relan, MD, MPH, Pharming's chief medical officer, commented:

"EMA's validation for review of our MAA under an accelerated evaluation pathway highlights Pharming's continued commitment to promote leniolisib as a targeted treatment for adults and adolescents 12 years or older with APDS. We anticipate leniolisib will cover an unmet need." for patients with APDS, who currently depend on supportive therapies to treat their main symptoms.This review constitutes a key milestone in Pharming’s effort to offer healthcare providers and their patients global access to leniolisib.We look forward to collaborating with the EMA as necessary throughout the normative process."

About Activated Phosphoinositide 3-kinase δ Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately 1 to 2 people per million. APDS is caused by variants in either of two genes, PIK3CD or PIK3R1, that regulate white blood cell maturation. Variants of these genes lead to hyperactivity of the PI3Kδ (phosphoinositide 3-kinase delta) pathway.1,2 Balanced signaling in the PI3Kδ pathway is essential for physiological immune function. When this pathway is overactive, immune cells fail to mature and function properly, leading to immunodeficiency and dysregulation.1,3 APDS is characterized by severe and recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.4,5 Because these symptoms may be associated with a variety of conditions, including other primary immunodeficiencies, people with APDS are frequently misdiagnosed and suffer a median diagnostic delay of 7 years.6 Since APDS is a progressive disease, this delay can take to an accumulation of damage over time, including damage and lymphoma.4-7 The only way to definitively diagnose this condition is through genetic testing.

Acerca de leniolisib

Leniolisib is a small molecule inhibitor of the delta isoform of the class IA 110 kDa catalytic subunit PI3K. PI3Kδ is predominantly expressed in hematopoietic cells and is essential for the normal function of the immune system through the conversion of phosphatidylinositol-4-5-triphosphate (PIP2) to phosphatidylinositol-3-4-5-triphosphate (PIP3). Leniolisib inhibits the production of PIP3, and PIP3 serves as an important cellular messenger that activates AKT (via PDK1) and regulates a multitude of cellular functions including proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism. Unlike PI3Kα and PI3Kβ, which are ubiquitously expressed, PI3Kẟ and PI3Kγ are expressed primarily in cells of hematopoietic origin. The central role of PI3Kẟ in the regulation of numerous cellular functions of the adaptive immune system (B cells and, to a lesser extent, T cells), as well as the innate immune system (neutrophils, mast cells, and macrophages), strongly indicates that PI3Kẟ is a target. valid and potentially effective therapy for immune-mediated diseases such as APDS. To date, leniolisib has been well tolerated during the Phase 1 first-in-human trial in healthy subjects and the Phase II/III registration enablement study in patients with APDS.

Acerca de 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 drugs, including small molecules, biologics and gene therapies that are in early or late stages of development. Pharming is headquartered in Leiden, the Netherlands, and has employees around the world 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.

Forward-looking statements

This press release may contain forward-looking statements. Forward-looking statements are forward-looking statements 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. statements. These forward-looking statements are identified by the use of terms and phrases such as "target", "ambition", "anticipate", "believe", "could", "estimate", "expect", "goals", " intend'', ''may'', "milestones", ''targets'', ''outlook'', ''plan'', ''probably'', ''project'', ''risks'', "schedule," "seek," "should," "target," "will," and similar terms and phrases. Examples of forward-looking statements may include statements regarding the timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and business 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 clinical trials and their ramifications for the cost thereof yes; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties described in Pharming's 2021 Annual Report and Annual Report on Form 20-F for the year ended December 31, 2021 filed with the Securities and Exchange Commission and US Securities, 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 by them. 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. All 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 undertakes no obligation to publicly update or revise any forward-looking statement as a result of new information, future events or other information.

internal information

This press release concerns the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.

References

1. Lucas CL, et al. Nat Immunol. 2014;15:88-97.2. 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.

For more public information, contact:

Pharming Group, Leiden, The NetherlandsHeather Robertson, Director of Investor Relations and Corporate Communications T: 31 71 524 7400E: investor@pharming.com

FTI Consulting, Londres, Reino UnidoVictoria Foster Mitchell/Alex Shaw/Amy ByrneT: 44 203 727 1000

LifeSpring Life Sciences Communication, Ámsterdam, Países BajosLeon MelensT: 31 6 53 81 64 27E: pharming@lifespring.nl

RR PP from United States:Ethan MetelenisE: Ethan.Metelenis@precisionvh.comT: 1 (917) 882 9038

RR P.P. EU: Dan Caley E: Dan.caley@aprilsix.com T: 44 (0) 787 546 8942

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