Pharming Group NV · EURONEXT:PHARM
Pharming Group NV — FY2021 Earnings Presentation
37 slides from Pharming Group NV's earnings presentation, Mar 17, 2022
Slide 1: Pharming Group N.V. Full Year 2021 Financial Results
Pharming Group N.V. Full Year 2021 Financial Results Analyst Call March 17, 2022 NASDAQ: PHAR | Euronext Amsterdam: PHARM
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 Anurag Relan Sijmen de Vries Jeroen Wakkerman Chief Medical Officer Chief Executive Officer Chief Financial Officer Q&A
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 within Long-term identification and Grow our global fully integrated our rare/ultra-rare in-house expertise to development of solutions for patients commercial infrastructure grow our business w
Slide 6: Strategy in action: Progress during 2021
Strategy in action: Progress during 2021 Commercial: RUCONEST® Late-stage pipeline Earlier-stage pipeline Positive top-line data in the pivotal In-licensing of OTL-105, a potentially Expanding reach of RUCONEST® with Phase II/III study of leniolisib for the curative candidate for HAE from commercial
Slide 7: HAE & RUCONEST
HAE & RUCONEST® Ongoing strong sales performance supporting future investment in long-term growth
Slide 8: 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 9: 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 Dec ‘21. Based on partial payer data, that we estimate represents ~25% of the market
Slide 10: APDS & leniolisib
APDS & leniolisib Expanding our commercial portfolio and leveraging our existing infrastructure to drive growth
Slide 11: Significant unmet need in APDS
Significant unmet need in APDS (activated PI3Kδ syndrome) Burden of APDS1-4 Current treatment options for APDS6 Estimated >1,350 patients (500 US, 675 EU, 190 Japan) live with APDS Supportive/non-specific therapies (e.g. antibiotics, Often used together steroids) • More than 350 patients already ide
Slide 12: Uncovering APDS: US targeted patient identification strategy
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 sophisticated & targeted di
Slide 13: Leniolisib in development for the treatment of APDS
Leniolisib in development for the treatment of APDS US PDUFA target date APDS : Activated PI3K Delta Syndrome Q1/2023 leniolisib1,2 Effective oral selective PI3Kδ inhibitor Precision biomarker response demonstrates impact on root cause Potential to mitigate progression of disease & reduce treatment
Slide 14: 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 15: Leniolisib APDS Part 1 data
Leniolisib APDS Part 1 data Safety & Efficacy1 Leniolisib was well tolerated at all oral doses (10 mg, 30 mg, 70 mg), but most effective at 70 mg in patients >45 kg No significant side effects or study drug discontinuations 12 weeks of leniolisib resulted in less disease activity and greater patient
Slide 16: 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 Slide courtesy of Dr V. Koneti Rao. Leniolisib is an investigational new drug tha
Slide 17: Leniolisib APDS Part 2: Efficacy data
Leniolisib APDS Part 2: Efficacy data Primary efficacy results demonstrated clinical efficacy of leniolisib over placebo Reduction from baseline in log10 transformed sum of product diameter in index lymphadenopathy lesions (p=0.0012) Normalization of immune dysfunction, as evidenced by increased pro
Slide 18: 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 Initiation of Anticipated Pivotal study Anticipated EMA Anticipated US Japanese clinical commercial EU results
Slide 19: HAE & OTL-105
HAE & OTL-105 Grow and extend our HAE franchise
Slide 20: OTL-105: developing a best-in-class HAE gene therapy
OTL-105: developing a best-in-class HAE gene therapy Collaboration with Orchard Therapeutics to develop and commercialize an ex vivo autologous hematopoietic stem cell (HSC) gene therapy for HAE OTL-105 inserts one or more functional copies of the SERPING1 gene into patients own HSCs ex vivo which a
Slide 21: Financial highlights 2021: Revenue (1/6)
Financial highlights from 2021: Building a sustainable business (1/6) REVENUE Revenues for the full year of 2021 were US$198.9 million a 6% decrease from US$212.2 million in 2020. RUCONEST® Revenue (US$ Million) 212,6 198,9 187,5 151,9 146,1 138,7 97,8 93,2 87,6 54,5 43,6 35,2 Q1 2019 H1 2019 Q3 201
Slide 22: Financial highlights 2021: Gross profit (2/6)
Financial highlights from 2021: Building a sustainable business (2/6) Revenues from US sales in 2021 were US$193.4 million a 5% decrease compared to 2020 (US$202.7 million). • Ongoing recovery in sales following the impact of COVID-19 on the US healthcare economy in Q1 REVENUE 2021, as previously no
Slide 23: Financial highlights 2021: Operating profit & cost (3/6)
Financial highlights from 2021: Building a sustainable business (3/6) Operating profit of US$36.9 million in 2021, before US$23.3 million of one-off costs, relating to investment in the pipeline of US$13.1 million to in-license OTL-105 from Orchard Therapeutics and impairment of tangible and intangi
Slide 24: Financial highlights 2021: Cash & cash equivalents (4/6)
Financial highlights from 2021: Building a sustainable business (4/6) Cash and cash equivalents, together with restricted cash, decreased from US$206.7 million at the end of 2020 to US$193.0 million at the end 2021, • Mainly due to positive cash flows from operating activities of US$37.8 million inc
Slide 25: Financial highlights 2021: Profit before tax 2020–2021 (5/6)
Financial highlights from 2021: Profit before tax 2020 – 2021 (5/6) (US$ Million) 44.1 -10.9 -13.1 4.1 23.1 -10.2 4.3 -14.0 34.1 -7.5 -7.8 Profit Underlying OTL-105 Impairment Increase Increased Increased FX effect Orbimed loan Other Profit before tax business investment tangible and in R&D SG&A M&S
Slide 26: 2021: Cashflow January 2021 – December 2021 (6/6)
2021: Cashflow 1 January 2021 – 31 December 2021 (6/6) (US$ Million) -4.9 42.8 -21.3 -2.9 -25.0 -1.9 205.2 191.9 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 equivalen
Slide 27: Outlook
Outlook A return to 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 comm
Slide 28: Q&A
Q&A Anurag Relan Sijmen de Vries Jeroen Wakkerman Chief Medical Officer Chief Executive Officer Chief Financial Officer
Slide 29: 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 30: Statement of profit and loss
Statement of profit and loss % Pharming Amounts in US$ ‘000 2021 2020 Revenues 198,871 212,174 Costs of sales (21,142) (23,539) Gross profit 177,729 188,635 Other income 2,620 1,829 Research and development (70,369) (38,519) General and administrative (36,974) (24,085) Marketing and sales (59,445) (
Slide 31: Balance sheet – assets
Balance sheet — assets % Pharming Non-current assets Intangible assets 83.834 94.083 Property, plant and equipment 13.222 12.226 Right-of-use assets 19.943 9.427 Long-term prepayments 194 0 Deferred tax assets 21.216 31.877 Investment accounted for using the equity method 7.201 7.118 Investments in
Slide 32: Balance sheet – liabilities
Balance sheet — liabilities % Pharming Equity Share capital 7.282 7.165 Share premium 453.190 445.066 Legal reserves 2.172 19.859 Accumulated deficit (269.727) (288.655) Shareholders’ equity 192.917 183.435 Non-current liabilities Convertible bonds 139.007 149.727 Lease liabilities 18.456 8.230 Othe
Slide 33: Cash flow (1/2)
Cash flow (1/2) % Pharming Amounts in $’000 Profit before tax 23.079 44.094 Non-cash adjustments: Depreciation, amortization, impairment of non-current assets 19.610 8.314 Equity settled share based payments 9.056 6.537 Fair value gain (loss) loss on revaluation of derivatives (114) (69) Other finan
Slide 34: Cash flow (2/2)
Cash flow (2/2) % Pharming Capital expenditure for property, plant and equipment (10.739) (4.657) Investment intangible assets (3.447) (9.060) Investment associate 0 (329) Investment in equity instruments designated as at FVTOCI (4.589) 0 Acquisition of license (2.530) (1.583) Net cash flows used in
Slide 35: PI3KCD Patients (N=6): Clinical Demographics
PI3KCD Patients (N=6): Clinical Demographics Age at enrollment Splenomegaly Patient ID Cytopenia Pulmonary Problems History of lymphoma (years) Lymph-adenopathy 1 17.3 Yes No Bronchiectasis, asthma No Thrombocytopenia, Yes, 2 24.3 Yes No Neutropenia Large B-cell Lymphoma 3 17.3 Yes No Asthma, recurr
Slide 36: The HSC gene therapy approach
The HSC gene therapy approach 5. The gene-corrected 1. Cells are collected cells are infused into from the patient the patient 2. Blood stem cells are selected and purified 4. The patient is conditioned to receive treatment 3. A working copy of the gene is inserted into the cells using a viral vecto
Slide 37: HSC gene therapy has led to multiple approved and effective products
HSC gene therapy has led to multiple approved and effective products Modality HSC Gene Therapy AAV- GT Gene Editing - Multiple products - No liver-directed AAV is - No approved products approved and pipeline approved with impressive data - Selectivity for specific Proven Approach - HSC GT and CAR-T