RELEASE: Pharming Announces Release of Phase 3 Study Data of Leniolisib in APDS Patients in ASH "Blood" (1)

(Information sent by the signatory company).

RELEASE: Pharming Announces Release of Phase 3 Study Data of Leniolisib in APDS Patients in ASH "Blood" (1)

(Information sent by the signatory company)

Leniolisib was well tolerated and significant improvement over placebo was notable in co-primary endpoints, reflecting a favorable impact on patients' immune dysregulation and impairment

Peer-reviewed publication enhances international understanding of APDS, a rare and newly characterized immunodeficiency

LEIDEN, The Netherlands, Dec. 7, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM) (Nasdaq: PHAR) today announces positive results from a Phase 3 clinical trial of investigational drug Leniolisib, an oral, selective phosphoinositol 3-kinase inhibitor delta (PI3Kδ) in adult and adolescent patients with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare immunodeficiency, have been published in Blood,1 the peer-reviewed international medical journal. by the American Society of Hematology (ASH) peer review. Data from this study were previously announced on February 2, 2022.

The article, titled "Phase 3 Randomized Placebo-Controlled Trial of the PI3Kδ Inhibitor Leniolisib for Activated PI3Kδ Syndrome," described the results of the randomized, triple-blind, placebo-controlled, multinational clinical trial that included 31 patients. with APDS 12 years or older. Patients were randomized in a 2:1 ratio to receive Leniolisib 70 mg or placebo twice daily for 12 weeks. Improvement over placebo was significant in the co-primary endpoints evaluating lymph node reduction and naïve B cell increase, reflecting the impact on immune dysregulation and correction of immunodeficiency in these patients. patients, respectively. The adjusted mean change (95% CI) between Leniolisib and placebo in lymph node size was -0.7 Dec. (-0.38, -0.12; P=0.0006; N=26 ) - and in the percentage of naïve B cells was 37.30 (24.06, 50.54; P=0.0002; N=13). Leniolisib was well tolerated, and fewer patients receiving Leniolisib reported treatment-related adverse events (mostly grade 1 or 2) compared to those receiving placebo (23.8% vs. 30.0%).

Vicki Modell, co-founder of the Jeffrey Modell Foundation, an international nonprofit organization dedicated to helping individuals and families affected by primary immunodeficiency disorders, commented:

"Pharming continues to offer significant support to the immunodeficiency community. The Jeffrey Modell Foundation is dedicated to early diagnosis and finding meaningful treatments for primary immunodeficiency, and we are keenly aware of the challenges faced by individuals with APDS. The publication of the study results in this patient population in such a distinguished and widely read journal furthers these goals."

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

"As we continue to seek a better understanding of APDS as a newly characterized rare disease, we remain committed to sharing our findings with researchers and clinicians around the world. With this commitment in mind, we are pleased that the results of this clinical trial of Phase III of Leniolisib have been published in the flagship journal of the American Society of Hematology.

The APDS patient population and their families have lived with unmet needs and without specific therapies, and the publication of this study is an integral step to improve the patient journey in this community. We are proud to share these results that demonstrated that Leniolisib is a well-tolerated and targeted therapy for APDS. We thank all study participants and investigators for their efforts and the essential role they played in the development of Leniolisib."

About Activated Phosphoinositol 3-Kinase δ Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately 1 - 2 people per million. APDS is caused by variants in one of two genes, PIK3CD or PIK3R1, that regulate white blood cell maturation. Variants in these genes lead to hyperactivity of the PI3Kδ - (phosphoinositol 3-kinase delta) pathway.2,3 Balanced signaling in the PI3Kδ pathway is essential for physiological immune function. When this pathway is overactive, immune cells do not mature and function properly, leading to immunodeficiency and dysregulation.2,4 APDS is characterized by severe and recurrent sinopulmonary infections, lymphoproliferation, autoimmunity, and enteropathy.5,6 Because these Symptoms can be associated with a variety of diseases, like other primary immunodeficiencies, people with APDS are frequently misdiagnosed and suffer a median delay to diagnosis of 7 years. 7 Because APDS is a progressive disease, this delay can lead to an accumulation of damage over time, including permanent lung damage and lymphoma.5-8 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 PI3K 110 kDa catalytic subunit. PI3Kδ is expressed predominantly in hematopoietic cells and is essential for normal function of the immune system through the conversion of phosphatidylinositol-4-5-trisphosphate (PIP2) to phosphatidylinositol-3-4-5-trisphosphate (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 such as cell proliferation, differentiation, cytokine production, angiogenesis, metabolism, and cell survival. Unlike PI3Kα and PI3Kβ, which are ubiquitously expressed, PI3Kẟ and PI3Kγ are mainly expressed 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), clearly indicates that PI3Kẟ is a target Valid and potentially effective therapeutics for immune diseases such as APDS. To date, Leniolisib has been adequately tolerated during the Phase 1 first-in-human trial in healthy subjects and the Phase II/III registration clearance 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 markets and develops an innovative range of precision medicines and protein replacement therapies, including small molecules, biologics and gene therapies that are in early to late development. Pharming is headquartered in Leiden, The Netherlands, with employees around the world serving patients in more than 30 markets in North America, Europe, the Middle East, Africa and Asia-Pacific.

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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 assumptions and expectations and involve known and unknown uncertainties and risks that could cause actual results, performance or events to differ materially from those expressed or implicit in these statements. These forward-looking statements are identified by the use of terms and phrases such as "target", "ambition", "anticipates", "believes", "may", "estimates", "expects", "goals", "intends", " can", "milestones", "goals", "projection", "plan", "probably", "project", "risks", "program", "seeks", "should", "purpose", "will" , and similar terms and phrases. Some examples of forward-looking statements may include statements regarding the schedule and progress of preclinical studies and clinical trials of Pharming's product candidates, Pharming's clinical and business prospects, and Pharming's expectations regarding its monetary and projected working capital requirements, the statements of which 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 cost ramifications thereof, and clinical, scientific, regulatory and technical developments. In view 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. 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 an undue level of reliance on any 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 publication. Pharming assumes no obligation to publicly update or correct any of them.


For more public information, contact:

Pharming Group, Leiden, The NetherlandsMichael Levitan, Vice President of Investor Relations and Corporate Communications,Heather Robertson, Manager of Investor Relations and Corporate Communications. Email:

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

Life Science Communication LifeSpring, Amsterdam, The Netherlands.Leon MelensPhone: 31 6 53 81 64 27Email:

RR pp. in the US: Ethan Metelenis. Email: Phone: 1 (917) 882 9038

RR pp. in the EU: Dan Caley. Email: Phone: 44 (0) 787 546 8942


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