Pharming Group N.V.

First quarter 2024 financial results and business update

May 8, 2024

NASDAQ: PHAR | EURONEXT Amsterdam: PHARM

SPEAKERS

Sijmen de Vries, MD

Stephen Toor

Anurag Relan, MD

Jeroen Wakkerman

Chief Executive Officer

Chief Commercial Officer

Chief Medical Officer

Chief Financial Officer

Forward-looking statements

This presentation 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, commercial, competitive and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2023 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2023, 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 presentation 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 presentation and are based on information available to Pharming as of the date of this presentation. 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.

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CEO

Sijmen de Vries, MD

Chief Executive Officer

Introduction

Building a leading global rare disease biopharma company

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

2024 Total Revenue Guidance - $280 - $295M (14 - 20% growth)

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Driven by Joenja®

Joenja® (leniolisib) franchise - multi-year growth potential

Joenja® U.S. (APDS)

Leniolisib (APDS)

•

Marketed (12+)

•

Patients on early

•

access/ named

Significant portion

•

patient programs

of identified

Global expansion /

patients on paid

regulatory reviews

therapy

•

Ongoing patient

•

Pediatric studies

finding and VUS

resolution efforts

Prevalence:

~1.5 / million

~2,000 patients

Leniolisib for Primary Immunodeficiencies (PIDs)

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

~5 / million

6

CCO

Stephen Toor

Chief Commercial Officer

Commercial update

RUCONEST® (rhC1INH): trusted treatment cornerstone for HAE

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

References: 1. RUCONEST®. Prescribing information. Pharming Healthcare Inc; 2020. 2. Bernstein JA, et al. Ann Allergy Asthma Immunol. 2017;118(4):452-453. 3. Data on file. Pharming Healthcare Inc; 2019

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The most common adverse reactions (incidence ≥2%) were headache, nausea and diarrhea. The most serious adverse reaction reported in clinical trials was anaphylaxis.

Joenja® U.S. launch: strong commercial execution

Strong commercial execution 12 months into U.S. launch

Continue to enroll and add patients on paid therapy in 1Q24

83 patients on paid therapy at end 1Q24, with 5 additional enrollments pending authorization >50 diagnosed patients (12+) not yet enrolled and >50 pediatric

1Q24 revenue US$9.6M (+21% vs. 4Q23) Includes US$1.1M Europe and RoW revenue

~500 APDS patients in the U.S.* with >220 diagnosed at end 1Q24

+15 diagnosed patients in 1Q24, including patients diagnosed via VUS resolution

Significant focus on genetic family testing

Variant of uncertain significance (VUS) validation studies to complete in 4Q24 focused on >1100 patients identified in the U.S. with VUSs

* Prevalence estimated at 1.5 patients per million population, based on available literature

As of December 31, 2023, Pharming has identified >840 diagnosed APDS patients in global markets

>730 of these patients are in key global launch markets in the U.S., Europe, the U.K., Japan, Asia Pacific,

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Middle East, and Canada with total prevalence of ~2000 APDS patients

Joenja® (leniolisib) franchise - multi-year growth potential

Joenja® U.S. (APDS)

Leniolisib (APDS)

•

Marketed (12+)

•

Global expansion /

• 83 patients on paid

regulatory reviews

•

therapy / 5 pending

Pediatric studies

•

Found >220 of

•

Found >840

~500 patients

patients globally

•

>50 diagnosed

•

138 patients on

patients (12+) not

therapy (access

yet enrolled and

programs and

>50 pediatric

clinical studies)

  • Ongoing patient finding and VUS resolution efforts

Leniolisib for Primary Immunodeficiencies (PIDs)

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

Joenja® U.S. and Europe / RoW access program revenues support 2024 guidance

U.S. Pricing: 30-day supply $47,220, Annual cost (WAC) $566,640

Global expansion focused on Europe, U.K., Japan, Asia Pacific, Middle East, and Canada

Prevalence:

~1.5 / million

~2,000 patients

~5 / million

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Disclaimer

Pharming Group NV published this content on 08 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 08 May 2024 05:26:06 UTC.