Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — H.C. Wainwright 24th Annual Investment Conference (September 2022)
18 slides from Pharming Group Nv's conference presentation, Sep 13, 2022
Slide 1: H.C Wainwright 24th Annual Investment Conference
Pharming Group N.V. H.C Wainwright 24th Annual Investment Conference September 13, 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: Company overview
Company overview One commercialized asset: RUCONEST® (conestat alfa) for the treatment of acute hereditary angioedema (HAE) Founded in 1988 in Leiden, the Netherlands. Pharming Group 1988 N.V. has a rich history within the Science Park. Global headquarters: Leiden, the Netherlands US headquarters: W
Slide 4: Continuing to build a sustainable business in rare diseases
Continuing to build a sustainable business in rare diseases Building a sustainable business by Anticipated approval and Ongoing pipeline development and focusing on RUCONEST® sales commercialization of leniolisib management of rare disease assets Positive cash flow from Successful commercialization
Slide 5: Pipeline at a glance
Pipeline at a glance % Pharming’ Commericalized asset - rhC1INH In-licensed Programs Additional Platform Program
Slide 6: Clinical Features of APDS
Clinical Features of APDS Overt Non-malignant Lymphoproliferation Malignant Lymphadenopathy – peripheral, Hepatosplenomegaly Nodular lymphoid hyperplasia of the airway and GI mucosa Lymphoproliferation mediastinal, mesenteric Lymphoma – diffuse large B cell, Hodgkin’s, marginal zone B cell, MALToma,
Slide 7: Leniolisib reduced lymphadenopathy
Leniolisib reduced lymphadenopathy Primary Outcome Analysis* Individual Index Lesion Sizes Change from baseline in index lesions Safety analysis set 0.1 SPD of Index Lymph Node Lesions CFB at D85 in log10-Transformed 0.0 n=8 n=18 -0.1 -0.02 -0.2 -0.27 -0.3 *** *** p=0.0006 -0.4 Leniolisib Placebo n=
Slide 8: Leniolisib Increased the Percentage of Naïve B Cells Out of Total B Cells
Leniolisib Increased the Percentage of Naïve B Cells Out of Total B Cells Primary Outcome Analysis* Mean Percentage of Naïve B Cells Over Time Change from baseline in naïve B cells Safety analysis set p=0.0002 50 80 *** 66.52 Total CD19+ B Cells (%) 65.80 61.81 40 CFB at D85 in Naïve B Cells/Total B
Slide 9: Leniolisib over three months was well tolerated
Leniolisib over three months was well tolerated 100 Patients with AEs Percentage of Patients, % 90.0 85.7 80.0 Grade 1 80 71.4 Grade 2 60 Grade 3 50.0 42.9 Grade 4 40 Study drug- 30.0 30.0 23.8 20.0 related AEs 20 14.3 9.5 9.5 10.0 SAEs 0 Leniolisib (n=21) Placebo (n=10) Number of AEs 92 65 19 3 3 6
Slide 10: Long term leniolisib results (N=6)
Long term leniolisib results (N=6) Leniolisib is an investigational new drug that has not been approved for any use. 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 11: Strategic highlights: leniolisib progress
Strategic highlights: leniolisib progress We remain on track for the commercial approval of leniolisib in the US, UK and the EU USA UK / EU Pharming receives positive EMA decision on pediatric investigation plan (PIP) Filing of New Drug Authorization with the FDA for leniolisib in Europe JAN 6 JUL 2
Slide 12: Upcoming milestones for leniolisib
Upcoming milestones for leniolisib* JULY 29: Filing of New Drug Initiation of Anticipated Anticipated Authorization with pediatric US regulatory EU regulatory the FDA studies approval approval H2 2022 H1 2023 H2 2023 Anticipated Anticipated Anticipated EMA and US commercial commercial MHRA filings l
Slide 13: Commercialized asset at a glance: RUCONEST (conestat alfa)
Commercialized asset at a glance: RUCONEST® (conestat alfa) RUCONEST® sales of US$198.9 million in FY 2021 RUCONEST® sales growth supported by an increase in physicians prescribing and number of patients Safe and reliable acute treatment option for hereditary angioedema (HAE) Market representation (
Slide 14: Financial Highlights H1 2022
Financial Highlights H1 2022
Slide 15: Financial highlights
Financial highlights Total revenues up 4% compared to H1 2021 Net profit increased by 33% compared to H1 2021 Positive cash flows, offset by exchange rate effect Stake held by Pharming in BioConnection reduced, received a one-off US$7.5 million cash payment and recognized gain of US$12.8 million. 15
Slide 16: Financial highlights: H1 2022
Financial highlights: H1 2022 TOTAL TOTAL Increased by 4% REVENUES US$93.2 million REVENUES to US$96.8 million (US$ Million) H1 2021 H1 2022 96.8 GROSS GROSS 93.2 Increased by 5% PROFIT US$83.8 million PROFIT to US$87.9 million H1 2021 H1 2022 OPERATING OPERATING 43.6 46.6 Increased by 20% PROFIT US
Slide 17: Outlook for the remainder of 2022
Outlook for the remainder of 2022 Single digit growth in Group revenues from RUCONEST® sales, quarterly fluctuations are expected. On track for leniolisib regulatory filings to EMA and UK MHRA in H2 2022. Commercial approval of leniolisib from FDA in Q1 2023, with an anticipated launch and commercia
Slide 18: This presentation will be made available on the company's website
This presentation will be made available on the company’s website. www.pharming.com | investor@pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM Bloomberg: PHAR.AS
Pharming Group — H.C. Wainwright 24th Annual Investment Conference (September 2022) on 6ix