Pharming Group N.V.
Corporate Presentation
May 2024
NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
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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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) | 3 |
Driven by Joenja® | |
Pipeline - multiple commercial stage rare disease products
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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 | 5 |
RUCONEST®
C1-INH targets the root cause of HAE
Complement System1-6
There are 3 known cascades that can lead to an HAE attack
Contact Activation System1,7-10
Fibrinolysis System8,14
Classical | Lectin |
Pathway | Pathway |
Immune Complexes | |
FXIIf | |
MBL | |
C1 |
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
C1-INH therapy regulates | |
FXII | multiple pathways11 |
FXIIa
Feedback Loop | ||
K activates FXII → FXIIa | ||
HMWK-PK | HMWK-K | Lanadelumab and |
berotralstat inhibit K12,13 |
K cleaves HMWK, releasing BK
BK
B2-RB1-R
Plasminogen | |
uPA/tPA | |
Plasmin | |
Tranexamic Acid inhibits | |
plasmin9 | |
ABBREVIATIONS | |
BK | Bradykinin |
C1-INH | C1 inhibitor |
C# | Complement component |
FXII | Coagulation factor XII |
FXIIa | Activated FXII |
FXIIf | Fragment of FXIIa |
HAE | Hereditary angioedema |
HMWK | High-molecular-weight kininogen |
- 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.
MASP | MBL-associated serine protease |
MBL | Mannose-binding lectin |
PK | Prekallikrein |
tPA | Tissue plasminogen activator |
uPA | Urokinase plasminogen activator |
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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 | 8 |
The most common adverse reactions (incidence ≥2%) were headache, nausea and diarrhea. The most serious adverse reaction reported in clinical trials was anaphylaxis. |
Joenja® (leniolisib)
U.S. launch of Joenja®: a much-needed treatment for APDS patients and another achievement for Pharming
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
Please see Important Safety Information and full Prescribing Information available at joenja.com | 10 |
Rao VK, et al. Blood. 2023;141(9):971-983 |
Rao VK, et al. Poster presented at: 64th Annual American Society of Hematology Annual Meeting; December 10-13, 2022; New Orleans, LA.
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Pharming Group NV published this content on 15 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 15 May 2024 13:19:02 UTC.