PR98214

LEIDEN, Netherlands, Oct. 11, 2022 /PRNewswire=KYODO JBN/ --

-- Application is based on randomized, controlled and long-term extension data

for leniolisib as a treatment for APDS, a rare primary immunodeficiency

-- This submission follows the grant of accelerated assessment allowing an

expedited review for leniolisib from a standard 210 days to 150 days

Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam:

PHARM/Nasdaq: PHAR) announces that it has submitted a Marketing Authorisation

Application (MAA) to the European Medicines Agency (EMA) for leniolisib, an

oral, selective phosphoinositide 3-kinase delta (PI3K delta) inhibitor, as a

treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare

primary immunodeficiency, in adults and adolescents 12 years or older.

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On August 1, 2022, Pharming announced the leniolisib MAA was granted

accelerated assessment by EMA's Committee for Medicinal Products for Human Use

(CHMP). The accelerated assessment reduces the review timeframe from 210 days

to 150 days. Upon request, the EMA will grant an accelerated assessment of an

MAA if they decide the product is of major interest for public health, and in

particular, from the viewpoint of therapeutic innovation. Marketing

authorisation for leniolisib in the EEA is anticipated in H1 2023.

Anurag Relan, Chief Medical Officer of Pharming, commented:

"This MAA submission, under an accelerated regulatory pathway, is an important

step towards approval of our second product in the EEA and highlights

Pharming's ongoing commitment to advancing leniolisib as a treatment for

patients with APDS. There is a significant unmet need for therapies to improve

outcomes for these patients, which, if left untreated, can result in permanent

lung damage and lymphoma. Leniolisib has the potential to be the first approved

treatment for this rare and orphan-designated disease, and we look forward to

continuing our work with key stakeholders to bring this new product to

patients."

The MAA is supported by positive data from a Phase II/III study of leniolisib,

announced on February 2, 2022, which met its co-primary endpoints of reduction

in lymph node size and correction of immunodeficiency in the target population.

Furthermore, safety data from the study showed that leniolisib was well

tolerated by participants. Also submitted as part of the MAA were data from a

long-term, open-label extension clinical trial in patients with APDS treated

with leniolisib.

About Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately 1 to 2

people per million. It is caused by variants in either of two genes, PIK3CD or

PIK3R1, that regulate maturation of white blood cells. Variants of these genes

lead to hyperactivity of the PI3K delta (phosphoinositide 3-kinase delta)

pathway.(1,2) Balanced signaling in the PI3K delta pathway is essential for

physiological immune function. When this pathway is hyperactive, immune cells

fail to mature and function properly, leading to immunodeficiency and

dysregulation.(1,3) APDS is characterized by 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, people with APDS are frequently misdiagnosed and

suffer a median 7-year diagnostic delay.(6) As APDS is a progressive disease,

this delay may lead to an accumulation of damage over time, including permanent

lung damage and lymphoma.(4-7) The only way to definitively diagnose this

condition is through genetic testing.

About Leniolisib

Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa

catalytic subunit of class IA PI3K with immunomodulating and potentially

anti-neoplastic activities. Leniolisib inhibits the production of

phosphatidylinositol-3-4-5-trisphosphate (PIP3). PIP3 serves as an important

cellular messenger activating AKT (via PDK1) and regulates a multitude of cell

functions such as proliferation, differentiation, cytokine production, cell

survival, angiogenesis, and metabolism. Unlike PI3K alpha and PI3K beta, which

are ubiquitously expressed, PI3K delta and PI3K gamma are expressed primarily

in cells of hematopoietic origin. The central role of PI3K delta in regulating

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 PI3K delta is a valid and

potentially effective therapeutic target for several immune diseases. To date,

leniolisib has been well tolerated during both the Phase 1 first-in-human trial

in healthy subjects and the Phase II/III registration-enabling study.

About Pharming Group N.V.

Pharming Group N.V. (Euronext Amsterdam: PHARM) (NASDAQ: PHAR (

https://www.prnewswire.com/news-releases/pharming-announces-us-fda-acceptance-for-priority-review-of-its-new-drug-application-for-leniolisib-301634867.html#financial-modal

)) 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 medicines, including small molecules, biologics, and

gene therapies that are in early to late-stage development. Pharming is

headquartered in Leiden, Netherlands, and has employees around the globe who

serve patients in over 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 (

https://www.linkedin.com/company/36477/admin/ )

Forward-Looking Statements

This press release contains forward-looking statements, including with respect

to timing and progress of Pharming's preclinical studies and clinical trials of

its product candidates, Pharming's clinical and commercial prospects,

Pharming's ability to overcome the challenges posed by the COVID-19 pandemic to

the conduct of its business, 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 and

technical developments. In light of these risks and uncertainties, and other

risks and uncertainties that are described in Pharming's 2021 Annual Report and

the Annual Report on Form 20-F for the year ended December 31, 2021 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. 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.

Inside Information

This press release relates to 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 further public information, contact:

Pharming Group, Leiden, The Netherlands

Heather Robertson, Investor Relations & Corporate Communications Manager

T: +31 71 524 7400

E: investor@pharming.com

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: pharming@lifespring.nl

US PR:

Ethan Metelenis

T: +1 (917) 882 9038

E: Ethan.Metelenis@precisionvh.com

EU PR:

Dan Caley

T: +44 (0) 787 546 8942

E: Dan.caley@aprilsix.com

SOURCE Pharming Group N.V.