- Third quarter 2023 revenues increased 23% to
US$66.7 million , compared to the third quarter 2022, driven by RUCONEST® revenue and theU.S. commercial launch of Joenja® - Nine month year to date 2023 revenues increased 9% to
US$164.1 million, compared to nine month year to date 2022 - RUCONEST® revenues increased 11% in the third quarter 2023 to
US$60.2 million , compared to the third quarter 2022, and increased 18% compared to the second quarter 2023 - RUCONEST® nine month year to date 2023 revenues increased by 2% YoY; on track for low single digit annual revenue growth
- Continued strength in
U.S. Joenja® (leniolisib) launch during the third quarter 2023; 63 patients on paid therapy andUS$6.5 million revenue - Overall cash and marketable securities of
US$199.2 million at the end of the third quarter 2023, compared toUS$194.1 million at the end of the second quarter 2023
Leiden,
“Pharming delivered a strong third quarter, increasing quarterly revenues to
To ensure we continue to find new Joenja® patients and grow revenues,
Looking towards 2024, we continue to progress in our efforts to make Joenja® (leniolisib) available to APDS patients in key global launch markets. Discussions with the EMA are ongoing and we continue to expect a CHMP opinion in the fourth quarter of 2023. In addition, the regulatory submissions filed for leniolisib in
The third quarter was an example of Pharming’s strong commercialization acumen as we succeeded in not only finding new Joenja® patients and quickly put them on therapy, but also in our ability to deliver growth for RUCONEST® in the competitive HAE landscape. None of this could have been achieved without our employees and partners who work tirelessly to bring our global HAE and APDS patients the medicines they need.”
Third quarter highlights
Commercialized assets
RUCONEST® marketed for the treatment of acute HAE attacks
RUCONEST® continued to perform well in the third quarter of 2023, with revenues of
The U.S. market contributed 97% of revenues, while the EU and Rest of World contributed 3% in the third quarter.
In the
Joenja® (leniolisib) marketed in the
The
As of
Access and reimbursement discussions have been proceeding as previously disclosed. We have seen high approval rates and fast timelines to covered therapy. Pharming’s market access teams have made significant progress working with government and private payors to provide the resources needed by the payors to formulate their policies to ensure access and reimbursement.
APDS patient finding
Based on available literature,
First, we continue to raise awareness of APDS and share new data on leniolisib at medical and scientific conferences worldwide.
Next, we continue to emphasize the importance of genetic testing when APDS is suspected. In addition to our sponsored genetic testing program in the
Finally, as APDS is an inherited genetic disease, we know that APDS patients may have affected family members. We have initiated a number of programs collaborating with clinicians and patients to aid in reducing the barriers and allowing the appropriate testing in families with APDS.
APDS patient finding - Variant of Uncertain Significance (VUS) resolution
APDS is diagnosed based on clinical symptoms, assessment of immune cell function, and genetic testing. For a patient to receive a definitive APDS diagnosis, a genetic test revealing a disease-causing (pathogenic or likely pathogenic) variant in either the PIK3CD or PIK3R1 genes is required.
Patients with clinical symptoms compatible with APDS frequently receive inconclusive genetic variant test results, i.e. previously unseen variants in the PIK3CD or PIK3R1 genes. It is important to determine if these Variants of Uncertain Significance (VUS) cause APDS.
Joenja® (leniolisib) strategic highlights - regulatory and clinical updates
Leniolisib for APDS
EEA and
As part of the review process of the Marketing Authorisation Application (MAA) for leniolisib for patients 12 years of age and older,
As announced on
In the
In August,
The single-arm, open-label clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in three patients, 12 years of age and older, who have a confirmed APDS diagnosis. Each patient will receive weight-based dosing up to 70mg of leniolisib twice daily for 12 weeks.
The study’s primary efficacy endpoints and secondary endpoints mirror those used to evaluate the clinical outcomes of the earlier leniolisib APDS trials.
Additional markets -
Pediatric clinical development
The majority of patients for our Phase III pediatric clinical trial with leniolisib for the treatment of APDS in patients 4 to 11 years of age have been enrolled. The single-arm, open-label, multinational clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children at sites in the
The second pediatric clinical trial for APDS patients 1 to 6 years of age commenced in the third quarter of 2023. The trial is currently recruiting and we anticipate the first patient will be enrolled in the fourth quarter. The single-arm, open-label, multinational clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children at sites in
Both studies are being conducted as part of Pharming’s Pediatric Investigational Plan for leniolisib as a treatment for APDS in children.
Leniolisib for additional indications (PI3Kδ platform)
As announced in our Joenja® approval conference call on
Pre-Clinical Pipeline
OTL-105
Work is continuing on the preclinical proof of concept studies. We anticipate providing further updates as OTL-105 progresses towards an Investigational New Drug (IND) filing.
Organizational highlights
At the Extraordinary General Meeting of Shareholders held on
On
Financial Summary
Amounts in US$m except per share data | 3Q 2023 | 3Q 2022 | 9M 2023 | 9M 2022 |
Income Statement | ||||
Revenue - RUCONEST® | 60.2 | 54.2 | 153.8 | 151.0 |
Revenue - Joenja® | 6.5 | 0.0 | 10.3 | 0.0 |
Total Revenues | 66.7 | 54.2 | 164.1 | 151.0 |
Cost of sales | (8.3) | (2.3) | (18.1) | (11.3) |
Gross profit | 58.4 | 51.9 | 146.0 | 139.7 |
Other income | 0.3 | 0.6 | 22.8 | 15.6 |
Research and development | (20.8) | (12.3) | (57.3) | (41.6) |
General and administrative | (10.9) | (12.0) | (31.9) | (28.5) |
Marketing and sales | (25.1) | (20.4) | (86.1) | (56.8) |
Operating profit (loss) | 1.9 | 7.8 | (6.5) | 28.4 |
Other finance income | 1.2 | 2.8 | 2.1 | 9.3 |
Other finance expenses | 0.7 | (1.2) | (4.6) | (4.0) |
Share of net profits in associates using the equity method | (0.5) | (0.1) | (1.0) | (0.6) |
Profit (loss) before tax | 3.3 | 9.3 | (10.0) | 33.1 |
Income tax credit (expense) | 0.2 | (0.2) | 2.6 | (4.8) |
Profit (loss) for the period | 3.5 | 9.1 | (7.4) | 28.3 |
Share Information | ||||
Basic earnings per share (US$) | 0.005 | 0.014 | (0.011) | 0.043 |
Diluted earnings per share (US$) | 0.005 | 0.013 | (0.011) | 0.040 |
Amounts in US$m | ||
Balance Sheet | ||
Cash and cash equivalents, restricted cash and marketable securities | 199.2 | 208.7 |
Current assets | 291.7 | 277.5 |
Total assets | 436.5 | 425.8 |
Current liabilities | 68.1 | 59.7 |
Equity | 209.9 | 204.6 |
Financial highlights
Third quarter 2023
Revenues in the third quarter of 2023 increased to
Gross profit in the third quarter increased by
Operating expenses increased by
In the third quarter of 2023, an operating profit of
Nine months 2023
Revenues for the first nine months of 2023 were
Gross profit for the first nine months of 2023 increased by
Operating loss for the first nine months of 2023 amounted to
Net result for the first nine months of 2023 was negative
Cash and cash equivalents, together with restricted cash and marketable securities, decreased from
Outlook
For the financial year of 2023:
- On track for low single digit growth in annual revenues from RUCONEST®.
- We anticipate the CHMP to issue their opinion for leniolisib in 4Q 2023. Subject to a positive opinion, Marketing Authorisation in
Europe is expected ~2 months later, followed by commercial launches in individual EU countries. - We intend to submit an ECDRP filing for leniolisib with the
U.K. MHRA shortly after a positive CHMP opinion, with approval expected several months later. Pharming will continue to allocate resources to accelerate future growth. Investments in launch preparations, commercialization and focused clinical developments for leniolisib including to support pediatric and key market approvals, as well as for the development of leniolisib in additional indications. These investments will continue to impact profit throughout 2023. Our current cash on hand, including the continued cash flows from RUCONEST® and Joenja® sales, are expected to be sufficient to fund these investments.- Further details on our plans to develop leniolisib in additional indications to be provided in 4Q 2023.
- Investments and continued focus on in-licensing or acquisitions of mid to late-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 2023 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/599pzbd7
Conference call dial-in details:
https://register.vevent.com/register/BIb15561b07c01418199d56096c5c3c8f9
Additional information on how to register for the conference call/webcast can be found on the Pharming.com website.
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
Auditor’s involvement
The Condensed Consolidated Interim Financial Statements have not been audited by the Company’s statutory auditor.
Risk profile
The risks outlined in the 2022 Annual Report continued to apply in the first nine months of 2023 and are expected to apply for the rest of the financial year. We continue to closely monitor the key risks and opportunities, and will respond appropriately to any emerging risk.
Related party transactions
There are no material changes in the nature, scope, and (relative) scale in this reporting period
compared to last year.
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
This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.
Condensed Consolidated Interim Financial Statements in US Dollars (unaudited)
For the period ended
- Condensed consolidated interim statement of profit and loss
- Condensed consolidated interim statement of comprehensive income
- Condensed consolidated interim balance sheet
- Condensed consolidated interim statement of changes in equity
- Condensed consolidated interim statement of cash flow
CONDENSED CONSOLIDATED INTERIM STATEMENT OF PROFIT AND LOSS | ||
For the 9-month period ended 30 September | ||
Amounts in US$ ‘000 | 9M 2023 | 9M 2022 |
Revenues | 164,099 | 151,001 |
Costs of sales | (18,094) | (11,288) |
Gross profit | 146,005 | 139,712 |
Other income | 22,811 | 15,602 |
Research and development | (57,287) | (41,639) |
General and administrative | (31,849) | (28,446) |
Marketing and sales | (86,136) | (56,819) |
Other Operating Costs | (175,272) | (126,904) |
Operating profit (loss) | (6,456) | 28,410 |
Other finance income | 2,050 | 9,297 |
Other finance expenses | (4,621) | (3,978) |
Finance cost net | (2,571) | 5,319 |
Share of net profits in associates using the equity method | (954) | (660) |
Profit (loss) before tax | (9,981) | 33,069 |
Income tax credit (expense) | 2,556 | (4,765) |
Profit (loss) for the period | (7,425) | 28,304 |
Basic earnings per share (US$) | (0.011) | 0.043 |
Fully-diluted earnings per share (US$) | (0.011) | 0.040 |
CONDENSED CONSOLIDATED INTERIM STATEMENT OF COMPREHENSIVE INCOME | ||
For the 9-month period ended 30 September | ||
Amounts in US$ ‘000 | 9M 2023 | 9M 2022 |
Profit (loss) for the period | (7,425) | 28,304 |
Currency translation differences | (2,079) | (26,313) |
Items that may be subsequently reclassified to profit or loss | (2,079) | (26,313) |
Fair value remeasurement investments | 419 | (573) |
Items that shall not be subsequently reclassified to profit or loss | 419 | (573) |
Other comprehensive income (loss), net of tax | (1,660) | (26,886) |
Total comprehensive income (loss) for the period | (9,085) | 1,418 |
CONDENSED CONSOLIDATED INTERIM BALANCE SHEET | ||
As at | ||
Amounts in US$ ‘000 | ||
Non-current assets | ||
Intangible assets | 69,849 | 75,121 |
Property, plant and equipment | 9,648 | 10,392 |
Right-of-use assets | 27,834 | 28,753 |
Long-term prepayments | 88 | 228 |
Deferred tax assets | 26,608 | 22,973 |
Investments accounted for using the equity method | 1,541 | 2,501 |
Investment in equity instruments designated as at FVTOCI | 949 | 403 |
Investment in debt instruments designated as at FVTPL | 6,749 | 6,827 |
Restricted cash | 1,464 | 1,099 |
Total non-current assets | 144,730 | 148,297 |
Current assets | ||
Inventories | 53,439 | 42,326 |
Trade and other receivables | 40,521 | 27,619 |
Restricted cash | 212 | 213 |
Marketable securities | 142,912 | — |
Cash and cash equivalents | 54,653 | 207,342 |
Total current assets | 291,737 | 277,500 |
Total assets | 436,467 | 425,797 |
Equity | ||
Share capital | 7,650 | 7,509 |
Share premium | 475,983 | 462,297 |
Legal reserves | (10,915) | (8,737) |
Accumulated deficit | (262,776) | (256,431) |
Shareholders’ equity | 209,942 | 204,638 |
Non-current liabilities | ||
Convertible bonds | 129,733 | 131,618 |
Lease liabilities | 28,734 | 29,843 |
Total non-current liabilities | 158,467 | 161,461 |
Current liabilities | ||
Convertible bonds | 1,748 | 1,768 |
Trade and other payables | 62,540 | 54,465 |
Lease liabilities | 3,770 | 3,465 |
Total current liabilities | 68,058 | 59,698 |
Total equity and liabilities | 436,467 | 425,797 |
CONDENSED CONSOLIDATED INTERIM STATEMENT CHANGES IN EQUITY |
For the 9-month period ended |
Amounts in $ ‘000 | Share capital | Share premium | Other reserves | Accumulated deficit | Total equity |
Balance at | 7,429 | 455,254 | 3,400 | (273,167) | 192,916 |
Profit (loss) for the period | — | — | — | 28,304 | 28,304 |
Other comprehensive income (loss) for the period | — | — | (27,546) | 660 | (26,886) |
Total comprehensive income (loss) for the period | — | — | (27,546) | 28,964 | 1,418 |
Legal reserves | — | — | — | — | — |
Income Tax expense from excess tax deductions related to Share-based payments | — | — | — | 273 | 273 |
Share-based compensation | — | — | — | 4,522 | 4,522 |
Options exercised /LTIP shares issued | 53 | 4,196 | — | (3,124) | 1,125 |
Total transactions with owners, recognized directly in equity | 53 | 4,196 | — | 1,671 | 5,920 |
Balance at | 7,482 | 459,450 | (24,146) | (242,532) | 200,254 |
Balance at | 7,509 | 462,297 | (8,737) | (256,431) | 204,638 |
Profit (loss) for the period | — | — | — | (7,425) | (7,425) |
Other comprehensive income (loss) for the period | — | — | (1,660) | — | (1,660) |
Total comprehensive income (loss) for the period | — | — | (1,660) | (7,425) | (9,085) |
Legal reserves | — | — | (518) | 518 | — |
Income Tax expense from excess tax deductions related to Share-based payments | — | — | — | 574 | 574 |
Share-based compensation | — | — | — | 5,935 | 5,935 |
Options exercised / LTIP shares Issued | 141 | 13,686 | — | (5,947) | 7,880 |
Total transactions with owners, recognized directly in equity | 141 | 13,686 | (518) | 1,080 | 14,389 |
Balance at | 7,650 | 475,983 | (10,915) | (262,776) | 209,942 |
CONDENSED CONSOLIDATED INTERIM STATEMENT OF CASH FLOWS | |||
For the 9-month period ended 30 September | |||
Amounts in $’000 | 9M 2023 | 9M 2022 | |
Profit before tax | (9,981) | 33,069 | |
Adjustments to reconcile net profit (loss) to net cash used in operating activities: | |||
Depreciation, amortization, impairment | 8,370 | 6,216 | |
Equity settled share based payments | 5,935 | 4,522 | |
Gain on disposal of investment in associate | — | (12,382) | |
Gain on disposal from PRV sale | (21,080) | — | |
Other finance income | (2,050) | (9,296) | |
Other finance expense | 4,621 | 3,978 | |
Share of net profits in associates using the equity method | 954 | 660 | |
Other | (1,130) | — | |
Operating cash flows before changes in working capital | (14,361) | 26,767 | |
Changes in working capital: | |||
Inventories | (11,113) | (6,196) | |
Trade and other receivables | (12,902) | 1,155 | |
Payables and other current liabilities | 8,075 | 272 | |
Restricted Cash | 363 | 169 | |
Total changes in working capital | (15,577) | (4,600) | |
Interest received (paid) | 1,059 | 31 | |
Income taxes paid (received) | — | (4,975) | |
Net cash flows generated from (used in) operating activities | (28,879) | 17,223 | |
Capital expenditure for property, plant and equipment | (1,133) | (1,071) | |
Proceeds on PRV sale | 21,080 | — | |
Investment intangible assets | 23 | (591) | |
Investment in associate | — | 7,384 | |
Purchases of marketable securities | (231,901) | — | |
Proceeds from sale of marketable securities | 86,451 | — | |
Net cash flows used in investing activities | (125,480) | 5,722 | |
Payment of lease liabilities | (3,847) | (2,385) | |
Interests on loans and leases | (4,052) | (3,999) | |
Settlement of share based compensation awards | 7,880 | 1,124 | |
Net cash flows generated from (used in) financing activities | (19) | (5,260) | |
Increase (decrease) of cash | (154,378) | 17,685 | |
Exchange rate effects | 1,689 | (20,906) | |
Cash and cash equivalents at the start of the period | 207,342 | 191,924 | |
Total cash and cash equivalents at the end of the period | 54,653 | 188,703 |
Source:
2023 GlobeNewswire, Inc., source