Slide 1: Pharming Group N.V. Q1 2022 Financial Results Analyst Call
Pharming Group N.V. Q1 2022 Financial Results Analyst Call May 12, 2022 NASDAQ: PHAR | Euronext Amsterdam: PHARM 1
Slide 2: Forward looking statements
Forward looking statements This presentation may contain forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future
Slide 3: Speakers
Speakers Sijmen de Vries Jeroen Wakkerman Anurag Relan Chief Executive Officer Chief Financial Officer Chief Medical Officer 3
Slide 4: Execution delivering future growth: Investment summary
Execution delivering future growth: Investment summary A well-funded business supported by commercial sales and a growing pipeline for the treatment of rare and ultra diseases with unmet medical needs Lead product, RUCONEST® (rhC1INH), launched in over 40 countries with sales of over US$198.9 millio
Slide 5: Three-pillar objectives to build a fully integrated sustainable business
Three-pillar objectives to build a fully integrated sustainable business Near-term expansion of portfolio Long-term identification and Grow our global fully integrated leveraging our in-house expertise in rare development of solutions for patients commercial infrastructure disease/unmet need to grow
Slide 6: HAE & RUCONEST®
HAE & RUCONEST® Ongoing strong sales performance supporting future investment in long-term growth
Slide 7: RUCONEST® positioning in the treatment of HAE
RUCONEST® positioning in the treatment of HAE HAE is caused by a deficiency of C1-INH, resulting in attacks of Convenience market severe swelling (angioedema) in various parts of the body (Prophylactic therapies) Patients use medication for treatment and prevention (prophylaxis) of attacks RUCONEST®
Slide 8: Ongoing demand for acute therapy following stabilization of prophylactic market
Ongoing demand for acute therapy following stabilization of prophylactic market Source: Payer sourced Claims through Mar ‘22. 8
Slide 9: APDS & leniolisib
APDS & leniolisib Expanding our commercial portfolio and leveraging our existing infrastructure to drive growth
Slide 10: PI3Kδ hyperactivity leads to APDS symptoms
PI3Kδ hyperactivity leads to APDS symptoms Common Symptoms of APDS4,5 Excess of immature or senescent cells Deficit of functional cells Severe, Recurrent, Hyperactive Persistent Infections: • Sinopulmonary PI3Kδ activity • Herpesvirus (especially EBV and CMV) Lymphoproliferation: • Lymphadenopathy B
Slide 11: Pivotal trial design
Pivotal trial design1,2 Part 1 Part 2 – Randomized, triple-blinded (patient, Dose-finding Placebo-controlled caregiver, investigator), placebo- controlled, fixed-dose study Randomized period 12 weeks Co-primary efficacy endpoints 12 weeks (lymphadenopathy and N=6 N=31 immunophenotype normalization)
Slide 12: Patient demographics – safety analysis
Patient demographics – safety analysis Leniolisib (n=21) Placebo (n=10) Total (N=31) Age Median (range), years 20.0 (12-54) 19.5 (15-48) 20.0 (12-54) < 18 years, n (%) 8 (38.1) 4 (40.0) 12 (38.7) Sex: Male/female, % 52.4/47.6 40.0/60.0 48.4/51.6 Weight: Median (range), kg 67.1 (46.9-100.6) 68.9 (50.
Slide 13: Leniolisib reduced lymphadenopathy
Leniolisib reduced lymphadenopathy Change from baseline at day 85 in log10 transformed SPD* in index lesions (n=26†) 0.1 0.05 Leniolisib Placebo 0 n=18 n=8 Adjusted Mean Change (log10 SPD) -0.05 -0.1 -0.06 -0.15 -0.2 -0.25 -0.3 -0.35 -0.30 -0.4 ** p=0.0012 Data were analyzed using ANCOVA model with
Slide 14: Additional analysis: SPD of index lesions by patient in PD data
Additional analysis: SPD of index lesions by patient in PD data Change from baseline at day 85 in SPD of index lesions (n=28) 60 53.83 40.53 40 20 10.27 Change from Baseline (%) 0 -4.72 -6.53 -8.89 -20 -14.37 -17.45 -24.97 -30.38 -30.90 -31.86 -33.70-34.07 -40 -35.09 -36.27 -37.84 -40.41 -43.12 -45.
Slide 15: Supportive analysis: naïve B cells
Supportive analysis: naïve B cells 90 Absolute percentage of cells over time† Change from baseline at day 85 in naïve B 80 cells (n=21*) 40 70 *** 35 27.87 p=0.0005 60 30 Adjusted Mean Change (%) 50 Naïve B Cells (%) 25 20 40 15 30 10 0.92 5 20 n=13 n=8 0 Leniolisib Placebo 10 -5 -10 0 Baseline Day
Slide 16: Secondary and exploratory analyses: leniolisib reduced spleen and liver size
Secondary and exploratory analyses: leniolisib reduced spleen and liver size Spleen Liver Change from baseline at day 85 in bi- Change from baseline at day 85 in bi- dimensional spleen size (n=27) dimensional liver size (n=28) 1000 ** 410.48 4000 Adjusted Mean Change (mm2) p=0.0079 * p=0.0416 1547.5
Slide 17: Leniolisib over three months was well tolerated
Leniolisib over three months was well tolerated Leniolisib (n=21) Placebo (n=10) Total (N=31) nE, nS (%)* nE, nS (%) nE, nS (%) AEs, Patients with AEs 92, 18 (85.7) 46, 9 (90.0) 138, 27 (87.1) Grade 1 AEs 65, 15 (71.4) 27, 8 (80.0) 92, 23 (74.2) Grade 2 AEs 19, 9 (42.9) 13, 5 (50.0) 32, 14 (45.2) Gr
Slide 18: Long term leniolisib results (N=6)
Long term leniolisib results (N=6) Patients have stopped (*) or decreased (§) immunoglobulin supplementation as a reflection of the normalization of their B cell function. Dashed lines indicate patient not on treatment 18 Slide courtesy of Dr V. Koneti Rao. Leniolisib is an investigational new drug
Slide 19: Launch preparations: Uncovering “APDS”
Launch preparations: Uncovering “APDS” US targeted patient identification strategy 1. 2. 3. BUILD APDS OUTREACH & ANALYTICS & AI NETWORK EDUCATION Genetic Testing The US has created a KOL network Patient identification using ‘Free of charge’ genetic testing, & referral pathway of prescribers sophist
Slide 20: Next steps: upcoming milestones
Next steps: upcoming milestones* Anticipated FDA Initiation of Anticipated US Anticipated EU filing pediatric studies regulatory approval regulatory approval H1 2022 H2 2022 H1 2023 H2 2023 Anticipated Anticipated EMA Initiation of Anticipated US commercial EU and MHRA filings Japanese clinical tria
Slide 21: Financial highlights from Q1 2022: Revenue (1/5)
Financial highlights from Q1 2022: Building a sustainable business (1/5) REVENUE Total revenues for Q1 2022 increased by 7% to US$46.6 million compared to US$43.6 million in Q1 2021 RUCONEST® Revenues (US$ Million) 212.6 198.9 151.9 146.1 97.8 93.2 54.5 43.6 46.6 Q1 2020 H1 2020 Q3 2020 FY 2020 Q1 2
Slide 22: Financial highlights from Q1 2022: Revenue from sales (2/5)
Financial highlights from Q1 2022: Building a sustainable business (2/5) US revenues increased by 7% to US$45.3 million compared to Q1 2021 REVENUE FROM SALES • Increase in the number of patients treated partly offset tighter inventory management at larger specialty pharmacies. Gross profit increase
Slide 23: Financial highlights from Q1 2022: Operating profit (3/5)
Financial highlights from Q1 2022: Building a sustainable business (3/5) Operating profit decreased to US$2.8 million (Q1 2021: US$6.3m), mainly due to an expected OPERATING increase in operating expenses from US$32.7 million in Q1 2021 to US$39.8 million in Q1 2022 PROFIT • A combination of launch
Slide 24: Profit before tax Q1 2021 – Q1 2022 (4/5)
Financial highlights from Q1 2022: Profit before tax Q1 2021 – Q1 2022 (4/5) (US$ Million) Profit Underlying Increased Increased Increased Financial Other Profit before tax business R&D G&A M&S result before tax Q1 2021 gross profit expenditure expenditure expenditure Q1 2022 -7.8 24
Slide 25: Q1 2022: Cashflow (5/5)
Q1 2022: Cashflow 1 January 2022 – 31 March 2022 (5/5) (US$ Million) Cash and cash Operating cash Total changes in Net cash Net cash flows Exchange rate Cash and cash equivalents on flow before working capital flows used in generated from effects equivalents on 1 January 2022 changes in investing (u
Slide 26: Outlook
Outlook Single digit growth in Group revenues from RUCONEST® sales, driven by the US and expanded EU operations, subject to the progression of the COVID-19 pandemic. Quarterly fluctuations in revenues are expected The submission of leniolisib regulatory filings to FDA and EMA, with commercial launch
Slide 27: Q&A
Q&A Sijmen de Vries Jeroen Wakkerman Anurag Relan Chief Executive Officer Chief Financial Officer Chief Medical Officer 27
Slide 28: www.pharming.com investor@pharming.com
www.pharming.com investor@pharming.com Euronext Amsterdam: PHARM NASDAQ: PHAR Bloomberg: PHAR.AS This presentation and a recording of this call will be made available on the company’s website.
Slide 29: Statement of profit and loss
Statement of profit and loss % Pharming Amounts in USS ‘000 YTD YTD 2021 Revenues 46,617 43,564 Costs of sales (4,877) (4,843) Gross profit 41,740 38,721 Other income 873 259 Research and development (14,863) (10,700) General and administrative (7,728) (7,161) Marketing and sales (17,197) (14,836) O
Slide 30: Balance sheet – assets
Balance sheet — assets % Pharming Amounts in US$ ‘000 March 31, 2022 December 31, 2021 Non-current assets Intangible assets 81,321 83,834 Property, plant and equipment 12,465 13,222 Right-of-use assets 19,432 19,943 Long-term prepayments 238 194 Deferred tax assets 20,194 21,216 Investment accounted
Slide 31: Balance sheet – liabilities
Balance sheet — liabilities % Pharming Equity Share capital 7,468 7,429 Share premium 457,961 455,254 Legal reserves (910) 3,400 Accumulated deficit (270,498) (273,167) Shareholders’ equity 194,021 192,916 Non-current liabilities Convertible bonds 135,564 139,007 Lease liabilities 17,900 18,456 Othe
Slide 32: Cash flow (1/2)
Cash flow (1/2) % Pharming Amounts in US$’000 YTD YTD 2021 Profit before tax 4,233 12,792 Non-cash adjustments: Depreciation, amortization, impairment of non-current assets 2,190 2,063 Equity settled share based payments 1,070 1,909 Fair value gain (loss) loss on revaluation of derivatives - (30) Ot
Slide 33: Cash flow (2/2)
Cash flow (2/2) % Pharming Capital expenditure for property, plant and equipment (208) (1,956) Investment intangible assets (167) (460) Investment associate — 398 Investment in equity instruments designated as at FVTOCI - - Acquisition of license - (547) Net cash flows used in investing activities (