- First quarter 2024 revenues increased by 31% to
US$55 .6 million, compared to the first quarter 2023, driven by theU.S. commercial launch of Joenja® and revenue growth of RUCONEST® - RUCONEST® revenues increased by 8% to
US$46 .0 million, compared to the first quarter 2023 - Joenja® (leniolisib) revenue of
US$9 .6 million, a 21% increase compared to the fourth quarter of 2023 - On track for 2024 total revenue guidance of
US$280 million -US$295 million (14 - 20% growth) - Overall cash and marketable securities of
US$203 .5 million at the end of the quarter Pharming to host a conference call today at13:30 CEST (7:30 am EDT )
Leiden,
Chief Executive Officer,
“Pharming delivered a strong first quarter, increasing quarterly revenues by 31% year-over-year to
We now have 83 patients on Joenja® paid therapy in the
We continue to be very active with regulatory agencies worldwide in our efforts to make Joenja® (leniolisib) available to patients in as many markets as possible, and continue to prepare for the commercialization of leniolisib in additional key global markets this year. Just last week Joenja® received regulatory approval in
With the recent completion of enrollment in the leniolisib clinical trial for children aged 4 to 11, we are also progressing towards regulatory filings worldwide for pediatric approval, beginning in 2025.
In addition, we are working to expand the leniolisib market opportunity through clinical development for larger primary immunodeficiency (PID) disorders. Our team has made significant progress towards commencing a Phase II proof of concept clinical trial in PIDs with immune dysregulation linked to PI3Kẟ signaling. We are now also preparing a clinical development plan for an additional PID indication, and look forward to providing further updates after obtaining regulatory feedback.
Finally, following the end of the quarter, we completed a successful refinancing by issuing €100 million in new convertible bonds due in 2029 to repurchase the majority of our €125 million convertible bonds due in 2025. These transactions strengthen our financial position while enhancing flexibility for the continued execution of our business strategy over the next several years.”
First quarter highlights
Commercialized assets
RUCONEST® marketed for the treatment of acute HAE attacks
RUCONEST® continued to perform well in the first quarter of 2024, with revenues of
The U.S. market contributed 97% of first quarter revenues, while the EU and Rest of World contributed 3%.
In the U.S. market, we saw continued strength in underlying in-market demand for RUCONEST®, including almost 70 new patient enrollments in the first quarter. We achieved strong overall performance in leading key revenue indicators including new physicians prescribing RUCONEST®, new patient enrollments, and the total number of patients.
Joenja® (leniolisib) marketed in the
Joenja® revenues increased to
As of
EU and Rest of World revenues are from product provided on a named patient basis.
APDS patient finding
As of
In the U.S. market, the number of diagnosed APDS patients increased by 15 during the first quarter 2024, including a few patients diagnosed via VUS resolution, bringing the number of identified patients in the
Leniolisib highlights - regulatory, clinical and commercial strategy updates
Leniolisib for APDS
Pharming’s strategy is to expand the commercial availability of leniolisib for APDS patients to key markets in
In total, there are currently 138 patients on leniolisib therapy as part of an Expanded Access Program (compassionate use), an ongoing clinical study, or a named patient program.
European Economic Area (EEA)
On
Additional markets -
Pediatric clinical development
In 2023,
In
Leniolisib for additional indications (PI3Kδ platform) - Primary immunodeficiencies (PIDs) beyond APDS
The Phase II clinical trial is a single arm, open-label, dose range-finding study to be conducted in approximately 12 patients. The objectives for the trial will be to assess safety and tolerability, pharmacokinetics, pharmacodynamics, and explore clinical efficacy of leniolisib in this new PID population. The trial has been designed to inform a subsequent Phase III program.
OTL-105
Consistent with Pharming’s current strategy as well as prioritization of clinical development expansion of leniolisib into additional PID indications,
Subsequent event - convertible bond refinancing
On
Financial summary
Amounts in US$m except per share data | 1Q 2024 | 1Q 2023 |
Consolidated Statement of Income | ||
Revenue - RUCONEST® | 46.0 | 42.5 |
Revenue - Joenja® | 9.6 | 0.0 |
Total Revenues | 55.6 | 42.5 |
Cost of sales | (8.4) | (4.0) |
Gross profit | 47.2 | 38.5 |
Other income | 0.3 | 0.6 |
Research and development | (18.5) | (15.6) |
General and administrative | (15.1) | (10.1) |
Marketing and sales | (30.2) | (27.1) |
Operating profit (loss) | (16.3) | (13.7) |
Other finance income | 1.8 | 0.1 |
Other finance expenses | (1.6) | (2.8) |
Share of net profits in associates using the equity method | (0.5) | (0.3) |
Profit (loss) before tax | (16.6) | (16.7) |
Income tax credit (expense) | 4.2 | 4.5 |
Profit (loss) for the period | (12.4) | (12.2) |
Share Information | ||
Basic earnings per share (US$) | (0.019) | (0.019) |
Diluted earnings per share (US$) | (0.019) | (0.019) |
Amounts in US$m | ||
Consolidated Balance Sheet | ||
Cash and cash equivalents, restricted cash and marketable securities | 203.5 | 215.0 |
Current assets | 296.6 | 316.3 |
Total assets | 441.7 | 462.9 |
Current liabilities (excluding convertible bonds) | 72.4 | 76.1 |
Equity | 205.9 | 218.8 |
Financial highlights
Total revenues for the first quarter of 2024 increased by 31% to
Amounts in US$ millions | 1Q 2024 | 1Q 2023 | ||||
RUCONEST® | Joenja® | Total | RUCONEST® | Joenja® | Total | |
Revenues | ||||||
US | 44.8 | 8.5 | 53.3 | 40.9 | — | 40.9 |
1.2 | 1.1 | 2.3 | 1.6 | — | 1.6 | |
Total revenues | 46.0 | 9.6 | 55.6 | 42.5 | — | 42.5 |
Gross profit increased by 23% to
The operating loss amounted to
The Company had a net loss of
Negative cash flows from operations amounted to
On
Outlook/Summary
For 2024, the Company anticipates:
- Total revenues between
US$280 million andUS$295 million (14% to 20% growth), with quarterly fluctuations expected. - Continued progress finding additional APDS patients in the
U.S. , supported by family testing and VUS validation efforts, and subsequently converting patients to paid Joenja® (leniolisib) therapy. - Increasing ex-
U.S. revenues leniolisib - from commercial availability or through our Named Patient Program and other funded early access programs in key global markets. - Completion of leniolisib clinical trials to support regulatory filings for approval in
Japan and pediatric label expansion in key global markets. - Progress towards regulatory approvals for leniolisib in the EEA, the
U.K. ,Canada andAustralia . - Initiate and advance a Phase II clinical trial for leniolisib in PIDs with immune dysregulation linked to PI3Kδ signaling to significantly expand the long-term commercial potential of leniolisib.
- Continued operating cost investments to accelerate future revenue growth. Our current cash on hand and the continued cash flow from product revenues are expected to be sufficient to fund these investments. No material cash burn is expected prior to the impact of potential acquisition or in-licensing transactions.
- Continued focus on potential acquisitions and in-licensing of clinical stage opportunities in rare diseases. Financing, if required, would come via a combination of our strong balance sheet and access to capital markets.
No further specific financial guidance for 2024 is provided.
Additional information
Presentation
The conference call presentation is available on the Pharming.com website from
Conference Call
The conference call will begin at
Please note, the Company will only take questions from dial-in attendees.
Webcast Link:
https://edge.media-server.com/mmc/p/ov2ruv3b
Conference call dial-in details:
https://register.vevent.com/register/BI6f55067b569a46d7a67be9f94edefc95
Additional information on how to register for the conference call/webcast can be found on the
Pharming.com website.
Financial Calendar 2024
Annual General Meeting of Shareholders
2Q/1H 2024 financial results
3Q 2024 financial resultsOctober 24
For further public information, contact:
T: +1 (908) 705 1696
E: investor@pharming.com
FTI Consulting,
T: +44 203 727 1000
LifeSpring Life Sciences Communication,
T: +31 6 53 81 64 27
E: pharming@lifespring.nl
About
For more information, visit www.pharming.com and find us on LinkedIn.
Forward-looking Statements
This press release 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
Inside Information
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Condensed Consolidated Interim Financial Statements in US Dollars (unaudited)
For the period ended
- Condensed consolidated statement of income
- Condensed consolidated statement of comprehensive income
- Condensed consolidated balance sheet
- Condensed consolidated statement of changes in equity
- Condensed consolidated statement of cash flow
CONDENSED CONSOLIDATED STATEMENT OF INCOME | ||
For the period ended 31 March | ||
Amounts in US$ ‘000 | 1Q 2024 | 1Q 2023 |
Revenues | 55,586 | 42,541 |
Costs of sales | (8,386) | (4,075) |
Gross profit | 47,200 | 38,466 |
Other income | 345 | 579 |
Research and development | (18,521) | (15,620) |
General and administrative | (15,087) | (9,981) |
Marketing and sales | (30,249) | (27,107) |
Other Operating Costs | (63,857) | (52,708) |
Operating profit (loss) | (16,312) | (13,663) |
Other finance income | 1,779 | 123 |
Other finance expenses | (1,556) | (2,795) |
Finance result, net | 223 | (2,672) |
Share of net profits (loss) in associates using the equity method | (535) | (339) |
Profit (loss) before tax | (16,624) | (16,674) |
Income tax credit (expense) | 4,176 | 4,466 |
Profit (loss) for the period | (12,448) | (12,208) |
Basic earnings per share (US$) | (0.019) | (0.019) |
Diluted earnings per share (US$) | (0.019) | (0.019) |
CONDENSED CONSOLIDATED STATEMENT OF COMPREHENSIVE INCOME | ||
For the period ended 31 March | ||
Amounts in US$ ‘000 | 1Q 2024 | 1Q 2023 |
Profit (loss) for the period | (12,448) | (12,208) |
Currency translation differences | (3,734) | 3,813 |
Items that may be subsequently reclassified to profit or loss | (3,734) | 3,813 |
Fair value remeasurement investments | 51 | 127 |
Items that shall not be subsequently reclassified to profit or loss | 51 | 127 |
Other comprehensive income (loss), net of tax | (3,683) | 3,940 |
Total comprehensive income (loss) for the period | (16,131) | (8,268) |
CONDENSED CONSOLIDATED BALANCE SHEET | ||
Amounts in US$ ‘000 | ||
Non-current assets | ||
Intangible assets | 68,299 | 71,267 |
Property, plant and equipment | 9,013 | 9,689 |
Right-of-use assets | 22,849 | 23,777 |
Long-term prepayments | 90 | 92 |
Deferred tax assets | 35,686 | 29,761 |
Investment accounted for using the equity method | 1,707 | 2,285 |
Investments in equity instruments designated as at FVTOCI | — | 2,020 |
Investment in debt instruments designated as at FVTPL | 5,974 | 6,093 |
Restricted cash | 1,500 | 1,528 |
Total non-current assets | 145,118 | 146,512 |
Current assets | ||
Inventories | 55,883 | 56,760 |
Trade and other receivables | 38,697 | 46,158 |
Marketable securities | 150,078 | 151,683 |
Cash and cash equivalents | 51,892 | 61,741 |
Total current assets | 296,550 | 316,342 |
Total assets | 441,668 | 462,854 |
Equity | ||
Share capital | 7,681 | 7,669 |
Share premium | 479,657 | 478,431 |
Other reserves | (4,001) | (2,057) |
Accumulated deficit | (277,392) | (265,262) |
Shareholders’ equity | 205,945 | 218,781 |
Non-current liabilities | ||
Convertible bonds | — | 136,598 |
Lease liabilities | 28,438 | 29,507 |
Total non-current liabilities | 28,438 | 166,105 |
Current liabilities | ||
Convertible bonds | 134,889 | 1,824 |
Trade and other payables | 68,516 | 72,528 |
Lease liabilities | 3,880 | 3,616 |
Total current liabilities | 207,285 | 77,968 |
Total equity and liabilities | 441,668 | 462,854 |
CONDENSED CONSOLIDATED STATEMENT OF CHANGES IN EQUITY | |||||
For the period ended 31 March | |||||
Attributable to owners of the parent | |||||
Amounts in US$ ‘000 | Share capital | Share premium | Other reserves | Accumulated deficit | Total equity |
Balance at | 7,509 | 462,297 | (8,737) | (256,431) | 204,638 |
Profit (loss) for the period | — | — | — | (12,208) | (12,208) |
Reserves | — | — | — | — | — |
Other comprehensive income (loss) for the period | — | — | 3,831 | 109 | 3,940 |
Total comprehensive income (loss) for the period | — | — | 3,831 | (12,099) | (8,268) |
Other reserves | — | — | — | — | — |
Income tax benefit from excess tax deductions related to share-based payments | — | — | — | 720 | 720 |
Share-based compensation | — | — | — | 1,558 | 1,558 |
Options exercised / LTIP shares issued | 9 | 925 | (239) | 695 | |
Total transactions with owners, recognized directly in equity | 9 | 925 | — | 2,039 | 2,973 |
Balance at | 7,518 | 463,222 | (4,906) | (266,491) | 199,343 |
Balance at | 7,669 | 478,431 | (2,057) | (265,262) | 218,781 |
Profit (loss) for the period | — | — | — | (12,448) | (12,448) |
Reserves | — | — | 1,770 | (1,770) | — |
Other comprehensive income (loss) for the period | — | — | (3,683) | — | (3,683) |
Total comprehensive income (loss) for the period | — | — | (1,913) | (14,218) | (16,131) |
Other reserves | — | — | (31) | 31 | — |
Income tax benefit from excess tax deductions related to share-based payments | — | — | — | (16) | (16) |
Share-based compensation | — | — | — | 2,427 | 2,427 |
Options exercised / LTIP shares issued | 12 | 1,226 | — | (354) | 884 |
Total transactions with owners, recognized directly in equity | 12 | 1,226 | (31) | 2,088 | 3,295 |
Balance at | 7,681 | 479,657 | (4,001) | (277,392) | 205,945 |
CONDENSED CONSOLIDATED STATEMENT OF CASH FLOWS | ||
For the period ended 31 March | ||
Amounts in $’000 | 1Q 2024 | 1Q 2023 |
Profit (loss) before tax | (16,624) | (16,674) |
Adjustments to reconcile net profit (loss) to net cash used in operating activities: | ||
Depreciation, amortization, impairment of non-current assets | 5,921 | 2,306 |
Equity settled share based payments | 2,427 | 1,558 |
Other finance income | (1,779) | (123) |
Other finance expenses | 1,556 | 2,795 |
Share of net profits in associates using the equity method | 535 | 339 |
Other | 783 | (455) |
Operating cash flows before changes in working capital | (7,181) | (10,254) |
Changes in working capital: | ||
Inventories | 877 | (5,801) |
Trade and other receivables | 7,461 | (5,313) |
Payables and other current liabilities | (9,414) | (1,211) |
Restricted cash | 28 | 117 |
Total changes in working capital | (1,048) | (12,208) |
Interest received | 582 | 117 |
Income taxes received (paid) | — | (440) |
Net cash flows generated from (used in) operating activities | (7,647) | (22,785) |
Capital expenditure for property, plant and equipment | (80) | (215) |
Disposal of investment designated as at FVOCI | 1,971 | — |
Purchases of marketable securities | (94,778) | — |
Proceeds from sale of marketable securities | 93,551 | — |
Net cash flows generated from (used in) investing activities | 664 | (215) |
Payment of lease liabilities | (1,324) | (1,312) |
Interests on convertible bonds | (2,031) | (2,013) |
Settlement of share based compensation awards | 884 | 695 |
Net cash flows generated from (used in) financing activities | (2,471) | (2,630) |
Increase (decrease) of cash | (9,454) | (25,630) |
Exchange rate effects | (395) | 3,068 |
Cash and cash equivalents at | 61,741 | 207,342 |
Total cash and cash equivalents at | 51,892 | 184,780 |
Source:
2024 GlobeNewswire, Inc., source