Pharming N : Presentatie - mei 2024

Market RUCONEST®

for acute HAE attacks in key

markets - U.S. focus

Positive cash flow from RUCONEST® revenue funds Joenja® (leniolisib) launches & pipeline development

FY23 revenue US$227.1M

1Q24 revenue US$46.0M (+8%)

Increase in patients and prescribers driving growth

Patients reliant on RUCONEST® despite increased therapy options

Global approvals and commercialization of Joenja® (leniolisib) for APDS

Successful commercialization of Joenja® (leniolisib) - first and only FDA approved treatment for APDS - U.S. launch April 2023

Revenue FY23 US$18.2M

1Q24 US$9.6M (+21% vs. 4Q23) Strong focus on patient finding

Israel approval (April 2024)

Regulatory reviews ongoing in EUR, U.K., CAN, AUS Pediatric and Japan clinical trials

Ongoing pipeline development

and management of rare

disease assets

Advance internal projects and rare disease in-licensing and acquisition strategy

Leniolisib development for PIDs with immune dysregulation beyond APDS - preparing Ph2

BD focus on clinical programs in immunology, hematology, respiratory and gastroenterology

OTL- 105 discontinued

Leniolisib for Primary Immunodeficiencies (PIDs)

• Phase II POC trial in PIDs with immune dysregulation linked to PI3Kẟ signaling
• Symptoms similar to APDS

Complement System1-6

There are 3 known cascades that can lead to an HAE attack

Contact Activation System1,7-10

Fibrinolysis System8,14

C1r/C1s

MASP-1

Consumption

of

C2 C4

C2a C4a

HAE diagnosis based on

• C1-INHlevel
• C1-INHfunction
• C4 level

Trigger(s)12

• Trauma
• Stress
• Infection
• Unknown

Kallikrein-

independent

pathway

MASP-1

upregulates

B2-R

Icatibant inhibits BK by binding

to B2 receptors

FXIIa

K cleaves HMWK, releasing BK

BK

B2-RB1-R

1. Kallikrein

Vascular Leakage (Edema)

Adapted from a clinical cascade developed in partnership with Dr. Allen Kaplan. This is a current scientific understanding of the cascades. Clinical implications are unknown.

7

The only recombinant treatment that targets the root cause of HAE by replacing missing or dysfunctional C1-INH

Well-tolerated and effective treatment option for acute hereditary angioedema (HAE) - including breakthrough attacks

Strong U.S. in-market demand - New enrollments up 25% in FY23 Almost 70 enrollments in 1Q24

Revenue:

FY23 US$227.1M (+10%) 1Q24 US$46.0M (+8%)

Second most prescribed product for acute attacks

97%: needed just 1 dose of RUCONEST®1

93%: acute attacks stopped with RUCONEST® for at least 3 days2

Performing well in leading U.S. revenue indicators: active patients, vials shipped, physicians prescribing (744, +15 vs. 2023)

Continued growth in 2024, strong positioning vs. acute orals in late-stage development

Joenja® (leniolisib) is a prescription medicine that is used to treat activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older

In a randomized placebo-controlled trial of patients

with APDS

• Joenja® met both primary end points with significant efficacy results
• Demonstrated significant improvement in other secondary and exploratory parameters

There were no drug-related serious adverse events or study withdrawals in Joenja® trials

Joenja® reported additional findings from an ongoing long-term open- label extension study interim analysis: reductions/discontinuations in IRT and reduction in infection rates

Extension study interim analysis demonstrated safety consistent with the randomized, controlled trial. We continue to collect observational long-term data on lymphadenopathy, naive B cells and IgM