PR98524

LEIDEN, Netherlands, Oct. 28, 2022 /PRNewswire=KYODO JBN/ --

-- Marketing authorisation in the European Economic Area anticipated in H1 2023

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

Nasdaq: PHAR) announces today that its Marketing Authorisation Application

(MAA) for leniolisib has been validated for scientific evaluation under an

accelerated assessment by the European Medicines Agency's (EMA) Committee for

Medicinal Products for Human Use (https://www.ema.europa.eu/en/glossary/committee-medicinal-products-human-use )

(CHMP ( https://www.ema.europa.eu/en/glossary/chmp )). The application,

submitted earlier in October 2022, is for the investigational drug, 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 adolescents and adults 12 years or older.

Logo - https://mma.prnewswire.com/media/1454235/Pharming_Group_NV_Logo.jpg

In August 2022, Pharming announced the leniolisib MAA was granted accelerated

assessment by EMA’s CHMP. The accelerated assessment reduces the review

timeframe from 210 days to 150 days. Upon request, 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 European

Economic Area is anticipated in H1 2023.

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 increase in percentage of naïve B cells in patients with

APDS. 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.

Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:

"EMA's validation for review of our MAA under an accelerated assessment pathway

highlights Pharming’s ongoing commitment to advance leniolisib as a targeted

treatment for adults and adolescents 12 years of age or older with APDS. We

anticipate that leniolisib will fill an unmet need for patients with APDS, who

currently rely on supportive therapies to treat their primary symptoms. This

review constitutes a key milestone in Pharming's effort to give healthcare

providers and their patients global access to leniolisib. We look forward to

collaborating with EMA as needed throughout the regulatory process."

About Activated Phosphoinositide 3-Kinase Delta 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 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. PI3K delta is expressed predominately in

hematopoietic cells and is essential to normal immune system function through

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

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 immune diseases such as APDS. 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 in

patients with APDS.

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 may contain forward-looking statements. Forward-looking

statements are statements of future expectations 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. These

forward-looking statements are identified by their use of terms and phrases

such as "aim", "ambition", "anticipate", "believe", "could", "estimate",

"expect", "goals", "intend", "may", "milestones", "objectives", "outlook",

"plan", "probably", "project", "risks", "schedule", "seek", "should", "target",

"will" and similar terms and phrases.  Examples of forward-looking statements

may include statements with respect to timing and progress of Pharming's

preclinical studies and clinical trials of its product candidates, Pharming's

clinical and commercial 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'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. 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. 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. Pharming does not

undertake any obligation to publicly update or revise any forward-looking

statement as a result of new information, future events or other information.

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

E: Ethan.Metelenis@precisionvh.com

T: +1 (917) 882 9038

EU PR:

Dan Caley

E: Dan.caley@aprilsix.com

T: +44 (0) 787 546 8942

SOURCE Pharming Group N.V.