PR97279

LEIDEN, the Netherlands, August 1, 2022 /PRNewswire=KYODO JBN/ --

EMA accelerated assessment allows a shorter review period for leniolisib from a

standard 210 days to 150 days

Pharming is on track to submit its Marketing Authorisation Application for

leniolisib in H2 2022

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

(Nasdaq: PHAR) announces that the European Medicines Agency's (EMA) Committee

for Medicinal Products for Human Use (CHMP) has granted an accelerated

assessment for the Marketing Authorisation Application (MAA) for leniolisib.

Leniolisib has been studied for the treatment of activated PI3K delta syndrome

(APDS), a rare primary immunodeficiency, in adults and adolescents age 12 or

older in the European Economic Area (EEA). Pharming is on track and plans to

submit its MAA for leniolisib to the EMA in October 2022.

Accelerated assessment reduces the timeframe for the CHMP to review an MAA from

210 days to 150 days. The EMA will grant, upon request, accelerated assessment

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

therapeutic innovation.

The clinical development for leniolisib includes positive data from a Phase

II/III study of the product, which met both its co-primary endpoints in the

target patient population of evaluated reduction in lymph node size and

correction of immunodeficiency. The primary efficacy results demonstrated

clinical efficacy of leniolisib over placebo with a statistically significant

reduction from baseline in the log10 transformed sum of product of diameters

(SPD) in the index lymphadenopathy lesions (p=0.0012) and normalization of

immune dysfunction, as evidenced by increased proportion of naïve B cells from

baseline (p<0.0001). The shrinking of lymphadenopathy lesions and increased

proportion of naïve B cells are important in patients as they indicate a

reduction in APDS disease markers.

In the study, leniolisib was generally well-tolerated, with the majority of

reported adverse events in both treatment groups classified as mild. There were

no adverse events that led to discontinuation of study treatment, there were no

deaths, and the incidence of serious adverse events (SAEs) was lower in the

leniolisib group than the placebo group. None of the SAEs were suspected to be

related to study treatment.

Anurag Relan, Chief Medical Officer of Pharming, commented:

"The acceptance of an accelerated regulatory review for leniolisib underlines

the high unmet need for patients with APDS, with the product potentially being

the first approved treatment for this rare disease. This is an important

milestone for the APDS community and for Pharming and is built on the

successful Phase II/III data, which we first reported in February 2022. We

remain focused on progressing leniolisib through the regulatory review process,

with our MAA on track for submission in October of this year, as we seek to

make this important new product available to immunologists, hematologists, and

their patients in Europe."

About Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)

APDS is a rare primary immunodeficiency that affects approximately one to two

people per million. Also known as PASLI, 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 specifically 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) 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.

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, et al. 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. 2020;59(3):323-333.

7.  Condliffe AM, Chandra A. Front Immunol. 2018;9:338.

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SOURCE: Pharming Group N.V.