- Full year 2024 total revenues increased by 21% to
US$297.2 million and exceeded our guidance, driven by record
RUCONEST® revenue and strong Joenja® (leniolisib)
growth
- Fourth quarter 2024 total revenues increased by 14% to
US$92.7 million, compared to the fourth quarter 2023
- RUCONEST® full year revenue increased by 11% to
US$252.2 million and fourth quarter revenue increased by 9% to
US$79.6 million, compared to the fourth quarter 2023
- Joenja® revenue increased by 147% to US$45.0 million in
the first full year post-launch and fourth quarter revenue
increased by 66% to US$13.1 million, compared to the fourth quarter
2023
- Fourth quarter operating profit increased to US$6.7
million from US$1.1 million in the fourth quarter
2023
- Generated operating profit and positive net cash flows
from operations for the second quarter in a row
- Two ongoing Phase II clinical trials of leniolisib for
additional primary immunodeficiencies (PIDs) with immune
dysregulation, including common variable immunodeficiency
(CVID)
- Completed the acquisition of Abliva AB, adding KL1333
for mitochondrial DNA-driven primary mitochondrial diseases to
Pharming’s late-stage clinical pipeline, in line with our vision to
become a leading global rare disease company
- Fabrice Chouraqui appointed as Chief Executive Officer
and Executive Director at EGM held on March 4, 2025
- 2025 total revenue guidance of US$315 million – US$335
million
- Pharming to host a conference call today at 13:30 CET
(8:30 am EDT)
Leiden, the Netherlands, March 13,
2025: Pharming Group N.V. (“Pharming” or “the Company”)
(Euronext Amsterdam: PHARM / Nasdaq: PHAR) presents its preliminary
(unaudited) financial report for the three months and full year
ended December 31, 2024.
Chief Executive Officer, Fabrice
Chouraqui commented: “I am delighted to have been
appointed as CEO of Pharming and am deeply excited for the
opportunities that lie ahead. The strength of the results presented
today, with record RUCONEST® revenue and strong Joenja® growth, is
testament to Pharming’s momentum.
We ended 2024 on a strong note, growing total
revenues by 21% to US$297.2 million and exceeding our revenue
guidance range of US$280-$295 million. This achievement underscores
the continued importance of RUCONEST® in the HAE treatment
landscape as well as the strong commercial performance of Joenja®
in just its first full year of sales.
We enter 2025 with a number of regulatory
reviews for leniolisib ongoing as we prepare for launches in key
markets and for pediatrics. We also advanced our efforts to expand
the addressable patient population for leniolisib, with two Phase
II trials in additional PIDs now underway. The genetically
identifiable PIDs indication and the CVID indication first
announced today represent significantly larger market opportunities
than APDS, with blockbuster revenue potential.
We are also strengthening our clinical pipeline
with the acquisition of Abliva, which has now been completed. This
adds a potential first-in-disease treatment for primary
mitochondrial diseases, KL1333, which has the opportunity to
further transform Pharming’s growth trajectory as it too has
blockbuster potential. The acquisition aligns with our vision to
become a leading global rare disease company and is well aligned
with our operational capabilities. We are now moving to start the
second wave of patient recruitment for the pivotal FALCON clinical
trial as soon as possible.
Pharming’s 2024 financial performance was also
noteworthy, with the company generating an operating profit and
positive net cash flows from operations in the last two quarters of
the year. This performance highlights the financial strength of our
core commercial business.
Looking to 2025, we will continue to invest
diligently to grow our portfolio in the U.S. and Joenja® in key
countries, to reach more patients with APDS, and to progress the
significant opportunities in our R&D pipeline for PIDs and
primary mitochondrial diseases.
I firmly believe that Pharming is
well-positioned to build on this positive momentum and embrace a
new cycle of growth, delivering strong long-term value creation for
all stakeholders and continuing our mission to serve the unserved
rare disease patients. I look forward to working with the Pharming
team to make this a reality.”
Fourth quarter and full year 2024
highlights
Commercialized productsRUCONEST®
marketed for the treatment of acute HAE attacks
RUCONEST® demonstrated significant strength in
the fourth quarter of 2024, with record revenues of US$79.6
million, a 9% increase compared to the fourth quarter of 2023.
RUCONEST® revenues for the full year 2024 were a record US$252.2
million, an 11% increase compared to 2023, significantly above our
expectation for mid single digit percentage growth.
The U.S. market contributed 98% of 2024
revenues, while the EU and Rest of World contributed 2%.
The strong RUCONEST® revenue growth in 2024 can
be attributed to strong performance in new physicians prescribing
RUCONEST®, new patient enrollments, and the total number of
patients, which together resulted in over 6% unit sales growth vs.
2023. We achieved over 100 new patient enrollments in the fourth
quarter and total enrollments in 2024 were up 24% vs. 2023. We also
increased the RUCONEST® physician prescriber base by 11% during the
year, in many cases adding previously unknown HAE prescribers.
Joenja® (leniolisib) marketed in the U.S. - the first
and only approved disease modifying treatment for APDS
Joenja® revenues increased to US$13.1 million in
the fourth quarter of 2024, a 66% increase compared to the fourth
quarter of 2023. This increase was mostly driven by higher volume
from the increase in patients on paid therapy in the U.S. compared
to the fourth quarter of 2023, and a significant increase in
revenues from EU and Rest of World which are from product provided
on a named patient basis. Revenue for 2024, the first full year of
sales following launch in April 2023, was US$45.0 million, compared
to US$18.2 million in 2023.
The U.S. market contributed 90% of 2024
revenues, while the EU and Rest of World contributed 10%.
As of December 31, 2024, we had 96 patients on
paid therapy in the U.S. and an additional five patients enrolled
and pending authorization, representing an increase of active
patients during the fourth quarter and continued progress enrolling
and transitioning eligible patients to paid therapy.
APDS patient finding
As of December 31, 2024, Pharming had identified
over 880 diagnosed APDS patients in global markets, including over
240 patients in the U.S. Of the identified patients in the U.S.,
over 150 patients are 12 years of age or older and eligible for
treatment with Joenja®.
Pharming continues to advance several
initiatives to diagnose additional APDS patients, including a
sponsored genetic testing program in the U.S. and Canada,
partnerships with several genetic testing companies who undertake
their own testing efforts, and family testing programs.
APDS patient finding - VUS resolution
Pharming is aware of approximately 1,200
patients in the U.S. with a Variant of Uncertain Significance, or
VUS, in the PIK3CD or PIK3R1 genes and is supporting validation
studies with various laboratories to confirm which of these
variants are pathogenic for APDS. Patients with disease-associated
variants would receive a molecular diagnosis of APDS and,
therefore, potentially be eligible for Joenja® treatment. Based on
data from Pharming’s navigateAPDS sponsored genetic testing
program, PIK3CD and PIK3R1 VUSs are found at four times the
frequency of those mutations currently classified as pathogenic /
likely pathogenic for APDS. Furthermore, a completed literature
review and pilot study resulted in 20% of VUS patients being
reclassified to APDS, suggesting that there could be a significant
increase in the number of APDS patients in the US once those
patients with a VUS are reclassified.
As previously communicated, Pharming is
supporting independent research to evaluate large numbers of VUSs
without the need for additional patient testing. VUS resolution via
high throughput screening methods is an established approach that
is accepted as strong functional evidence for variant
classification by various expert organizations including the
American College of Medical Genetics (ACMG) and ClinGen (a National
Institutes of Health-funded resource). One in vitro high throughput
screening study was completed in the fourth quarter of 2024,
identifying many novel variants leading to PI3Kδ hyperactivity.
Pharming is now supporting clinical genetics laboratories across
the US to be able to use their independent variant interpretation
to reclassify variants and thus issue amended genetic testing
reports for any variants these laboratories deem to be
disease-causing. We anticipate that these endeavors will lead to
the identification of new patients with APDS.
Joenja® (leniolisib)
developmentLeniolisib for APDS
During 2024, we received regulatory approvals
for leniolisib for patients 12 years of age or older in the U.K.
and Israel, and advanced additional regulatory filings, to bring
leniolisib to APDS patients outside of the U.S. We also made strong
progress in our clinical trials to support APDS marketing approval
for leniolisib in Japan and pediatric label expansion.
In total, there are currently 188 patients in a
leniolisib Expanded Access Program (compassionate use), an ongoing
clinical study, or a named patient program.
Pediatric clinical development
On December 11, 2024, we announced positive top
line results for the multinational Phase III clinical trial for
children 4 to 11 years of age, which has been evaluating leniolisib
tablets in 21 children with APDS. The data are consistent with the
improvements seen in the previously reported randomized controlled
trial in adolescent and adult APDS patients. Global regulatory
filings are planned to begin with a U.S. submission in the second
half of 2025.
Japan clinical trial
We completed an interim analysis, after 12-weeks
of treatment, for the Phase III clinical trial in Japan evaluating
leniolisib for the treatment of APDS in adult and pediatric
patients 12 years of age and older. The study’s safety and efficacy
findings were in line with data from the randomized controlled
trial used to support approvals in adolescent and adult APDS
patients in the U.S. and other countries, and support a regulatory
filing with Japan’s Pharmaceuticals and Medical Devices Agency
(PMDA) which is planned for mid-2025. An approval decision would be
expected in nine months based on priority review of the application
due to orphan drug designation (ODD) by the Ministry of Health,
Labour and Welfare of Japan (MHLW) for the treatment of APDS.
European Economic Area (EEA)
We are on track to complete the manufacturing
activities requested by the European Medicines Agency’s (EMA)
Committee for Human Medicinal Products (CHMP) in regard to the
ongoing review of the Marketing Authorisation Application (MAA) for
leniolisib for the treatment of adult and pediatric patients 12
years of age and older and submit a response prior to the January
2026 deadline.
United Kingdom
On September 25, 2024, the U.K. Medicines and
Healthcare products Regulatory Agency (MHRA) granted marketing
authorization for Joenja® (leniolisib) for the treatment of APDS in
adult and pediatric patients 12 years of age and older. Leniolisib
is currently under evaluation by the National Institute for Health
and Care Excellence (NICE) regarding reimbursement within the
National Health Service (NHS) in England and Wales.
Additional markets
In Australia, we filed a regulatory submission
for patients 12 years of age and older in the third quarter of 2023
and, after positive feedback from the Australian Advisory Committee
on Medicines, expect a final decision by mid-March 2025.
We filed a regulatory submission in Canada for
APDS patients 12 years of age and older in the third quarter of
2023. We have had ongoing interactions with Health Canada that
recently granted an extension to February 2026 to respond to a
request for additional CMC data, in line with the EMA extension. We
plan to respond in early 2026 and expect a regulatory decision in
2026.
Leniolisib for additional primary immunodeficiencies
(PIDs)
We made strong progress during 2024 on our
leniolisib indication expansion efforts. We are developing
leniolisib for additional primary immunodeficiencies, or PIDs,
which affect significantly more patients than APDS. These include
(i) genetically identifiable PIDs with immune dysregulation linked
to altered PI3Kδ signaling and (ii) common variable
immunodeficiency, or CVID, with immune dysregulation identified
independently of genetics.
We started the Phase II clinical trial
evaluating leniolisib for PIDs with immune dysregulation linked to
altered PI3Kẟ signaling in October 2024, and started a Phase II
clinical trial for CVID with immune dysregulation in February
2025.
Primary immunodeficiencies (PIDs) with immune
dysregulation linked to altered PI3Kδ signaling
On October 10, 2024, Pharming announced the
start of a Phase II, proof of concept, clinical trial evaluating
leniolisib in PIDs with immune dysregulation linked to PI3Kẟ
signaling in lymphocytes, with similar clinical phenotypes and
unmet medical needs to APDS. The first patient was dosed in the
study on October 29, 2024 and enrollment is progressing. The
clinical trial includes PID patients with ALPS-FAS, CTLA4
haploinsufficiency, NFKB1 haploinsufficiency and PTEN deficiency,
among others. Epidemiology suggests a prevalence of approximately
seven and a half patients per million in this targeted PID
population, compared to one to two patients per million for
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 are to
assess safety and tolerability, pharmacokinetics, pharmacodynamics,
and explore clinical efficacy of leniolisib in the targeted PID
population.
In February 2025, the FDA granted Fast Track
designation for leniolisib for the treatment of PIDs linked to
PI3Kẟ signaling. Fast Track is an FDA process designed to
facilitate the development and expedite the review of drugs to
treat serious conditions and fill an unmet medical need.
Common variable immunodeficiency (CVID) with immune
dysregulation
CVID with immune dysregulation represents a much
larger group of PID patients, which may be identified independently
of genetics, and with a targeted population prevalence of
approximately 39 patients per million. The majority of CVID
patients with immune dysregulation exhibit a spectrum of
noninfectious autoimmune and end-organ lympho-infiltrative disease
manifestations with similarities to APDS.
We engaged with the FDA and EMA on the CVID
indication, and received positive feedback regarding the large
unmet medical need and the rationale for evaluating leniolisib in
CVID patients with immune dysregulation. We have initiated a Phase
II study for leniolisib in CVID patients with immune dysregulation,
with the first patient expected to be dosed in late March 2025.
Acquisition of Abliva AB
In December 2024, we announced the proposed
acquisition of Abliva AB, via a public cash offer to the
shareholders to acquire all issued and outstanding shares of
Abliva, for approximately US$66.1 million. Abliva’s lead product
KL1333 is currently in a pivotal clinical trial in mitochondrial
DNA-driven primary mitochondrial diseases and has the potential to
significantly enhance our future growth trajectory. We announced
that we did not require external funding to fund the acquisition
and development costs for KL1333.
KL1333 for mitochondrial DNA-driven primary
mitochondrial diseases
KL1333 is in a pivotal clinical study (FALCON)
in adult patients with genetically confirmed primary mitochondrial
disease (PMD) with mitochondrial DNA (mtDNA) mutations who
experience consistent, debilitating fatigue and muscle weakness
(myopathy), and reduced life expectancy. Over 30,000 patients
diagnosed with mtDNA mitochondrial disease would be potentially
addressable by KL1333 in the U.S., EU4 (France, Germany, Italy,
Spain) and the UK, offering blockbuster potential for this product
in the U.S. alone.
KL1333 has shown positive clinical effects in a
proof-of-concept Phase 1b study, and a pre-planned interim analysis
of the ongoing pivotal FALCON trial demonstrated promising
differences over placebo in both alternate primary efficacy
endpoints passing futility. KL1333 has received Fast Track
designation in the U.S. and Orphan Drug Designation for the
treatment of PMD in the U.S. and EU. We are now moving to start the
second wave of patient recruitment for the pivotal FALCON clinical
trial as soon as possible. We anticipate the trial to read-out in
2027 with potential FDA approval by end of 2028.
Organizational updates
Ms. Inés Bernal was appointed Chief People
Officer, or CPO, as of December 1, 2024, to lead the development,
execution and monitoring of Pharming's people and culture
strategy.On October 24, 2024, we announced that Mr. Sijmen de
Vries, Executive Director and Chief Executive Officer, had informed
the Board of Directors that he would not be available for
reappointment at our next AGM.
Subsequent events
On January 21, 2025, we announced that the Board
of Directors had nominated biopharmaceutical leader Mr. Fabrice
Chouraqui as Pharming’s new Chief Executive Officer and Executive
Director, succeeding Mr. Sijmen de Vries.
Mr. Chouraqui was appointed for a term of four
years at the Extraordinary General Meeting of Shareholders (EGM)
that took place on March 4, 2025.
Upon the appointment of Mr. Chouraqui, Mr.
Sijmen de Vries resigned from the Board of Directors. To ensure a
smooth hand-over of tasks and responsibilities, Mr. de Vries will
remain a strategic advisor to the new CEO until December 31,
2025.
On February 20, 2025, we announced ownership of
shares and voting rights in Abliva AB exceeding 90% and thereby
initiated the necessary activities to delist the Company from the
Nasdaq Stockholm exchange. Following delisting, we expect to be
able to start the second wave of patient recruitment for the
ongoing pivotal FALCON clinical trial for KL1333 for the treatment
of mtDNA-driven primary mitochondrial diseases. Pharming has
initiated a compulsory acquisition procedure in respect of the
remaining shares in Abliva under the Swedish Companies Act. On
March 3, 2025, Nasdaq Stockholm approved Abliva’s application for
delisting and the last day of trading will be March 17, 2025. With
these events, the acquisition of Abliva is now completed.
The acquisition of Abliva will be accounted for
as a business combination. Substantially all of the value of the
acquisition is concentrated in a single asset, KL1333. Following
delisting as expected in March 2025, the acquisition would be
reflected in our financial statements beginning with the first
quarter 2025. At this point we expect the US$66.1 million
acquisition price to be allocated to the fair value of the acquired
identifiable assets and liabilities, with any excess to be recorded
as goodwill. We do not expect any P&L impact at the acquisition
date, besides recognition of acquisition costs incurred in
2025.Financial Summary
Consolidated Statement of Income |
4Q 2024 |
4Q 2023 |
2024 |
2023 |
Amounts in US$m except per share data |
|
|
|
|
Total Revenues |
92.7 |
81.2 |
297.2 |
245.3 |
Cost of sales |
(12.2) |
(7.1) |
(35.4) |
(25.2) |
Gross profit |
80.5 |
74.1 |
261.8 |
220.1 |
Other income |
0.1 |
0.6 |
2.2 |
23.3 |
Research and development |
(22.3) |
(11.6) |
(83.2) |
(68.9) |
General and administrative |
(24.6) |
(24.1) |
(70.6) |
(55.9) |
Marketing and sales |
(27.0) |
(37.9) |
(118.8) |
(124.0) |
Other Operating Costs |
(73.9) |
(73.6) |
(272.6) |
(248.8) |
Operating profit (loss) |
6.7 |
1.1 |
(8.6) |
(5.4) |
Fair value gain (loss) on revaluation |
(0.2) |
(0.9) |
5.0 |
(0.9) |
Other finance income |
3.1 |
1.6 |
6.8 |
3.7 |
Other finance expenses |
(2.5) |
(4.5) |
(9.9) |
(9.1) |
Share of net profits in associates using the equity method |
(0.5) |
0.7 |
(1.8) |
(0.3) |
Profit (loss) before tax |
6.6 |
(2.0) |
(8.5) |
(12.0) |
Income tax credit (expense) |
(2.9) |
(1.1) |
(2.5) |
1.5 |
Profit (loss) for the period |
3.7 |
(3.1) |
(11.0) |
(10.5) |
Share Information |
|
|
|
|
Basic earnings per share (US$) |
0.005 |
(0.004) |
(0.016) |
(0.016) |
Diluted earnings per share (US$) |
0.005 |
(0.004) |
(0.016) |
(0.016) |
Segment information - Revenues |
4Q 2024 |
4Q 2023 |
2024 |
2023 |
Amounts in US$m |
|
|
|
|
Revenue - RUCONEST® (US) |
78.2 |
71.9 |
246.6 |
221.2 |
Revenue - RUCONEST® (EU and RoW) |
1.4 |
1.4 |
5.6 |
5.9 |
Total Revenues - RUCONEST® |
79.6 |
73.3 |
252.2 |
227.1 |
Revenue - Joenja® (US) |
11.8 |
7.6 |
40.5 |
17.9 |
Revenue - Joenja® (EU and RoW) |
1.3 |
0.3 |
4.5 |
0.3 |
Total Revenues - Joenja® |
13.1 |
7.9 |
45.0 |
18.2 |
|
|
|
|
|
Total Revenues - US |
90.0 |
79.5 |
287.1 |
239.1 |
Total Revenues - EU and RoW |
2.7 |
1.7 |
10.1 |
6.2 |
|
|
|
|
|
Total Revenues |
92.7 |
81.2 |
297.2 |
245.3 |
Consolidated Balance Sheet |
December 31, 2024 |
December 31, 2023 |
Amounts in US$m |
|
|
Cash and cash equivalents, restricted cash and marketable
securities |
169.4 |
215.0 |
Current assets |
278.7 |
316.3 |
Total assets |
400.8 |
462.9 |
Current liabilities |
73.8 |
78.0 |
Equity |
221.9 |
218.8 |
Financial highlights
Fourth quarter 2024
Revenues in the fourth quarter of 2024 increased
by 14% to US$92.7 million from US$81.2 million in the fourth
quarter of 2023 (US$74.8 million in the third quarter of 2024).
RUCONEST® revenues amounted to US$79.6 million, a 9% increase
compared to the fourth quarter of 2023. A volume increase in the
U.S., and a U.S. price increase in line with CPI, were the primary
factors behind this increase in RUCONEST® revenues. Joenja®
revenues amounted to US$13.1 million in the fourth quarter of 2024,
a 66% increase compared to the fourth quarter of 2023. This
increase was primarily driven by an increase in volume.
Gross profit increased by US$6.4 million or 9%
to US$80.5 million (4Q 2023: US$74.1 million), mainly due to the
increase in revenues. This was partially offset by increased cost
of sales, in part due to a one-off inventory impairment due to a
power outage during the production process (US$ 1.1 million).
The operating profit amounted to US$6.7 million
compared to an operating profit of US$1.1 million in the fourth
quarter of 2023. This increase was primarily due to the increase in
gross profit mentioned above, offset by the increase in operating
expenses from US$73.6 million in the fourth quarter of 2023 to
US$73.9 million. Fourth quarter 2024 operating expenses increased
by US$9.2 million from US$64.7 million in the third quarter of
2024. Part of this 2024 increase was caused by one-off expenses,
totaling US$6.2 million, including the full impairment of US$5.1
million (4Q 2023: US$4.7 million) on the leased DSP facility at
Pivot Park in Oss, the Netherlands. Also contributing to the
fourth-quarter increase were legal and advisory fees associated
with the acquisition of Abliva AB, totaling US$1.1 million for the
fourth quarter of 2024.
The net finance result amounted to a gain of
US$0.4 million compared to a loss of US$3.8 million in the fourth
quarter of 2023. This was primarily driven by favorable EUR/USD
exchange rate developments, resulting in a foreign currency gain of
US$2.6 million compared to a loss of US$2.9 million in the fourth
quarter of 2023. Interest expense increased by US$1.5 million in
the fourth quarter of 2024 compared to the previous year, following
the convertible bond issuance in the second quarter of 2024.
In the fourth quarter of 2024, a net profit of
US$3.7 million was realized, in contrast to a net loss of US$3.1
million in the fourth quarter of 2023. This improvement was
primarily driven by the rise in gross profit supported by favorable
EUR/USD exchange rate developments, partially offset by the
increase in operating expenses.
Cash and cash equivalents, including restricted
cash and marketable securities, decreased from US$173.3 million at
the end of third quarter of 2024 to US$169.4 million at the end of
the fourth quarter of 2024. This decrease was primarily driven by
EUR/USD exchange rate developments (vast majority of cash and
marketable securities position is EUR denominated), offset by
positive cash flows from operations of US$9.3 million (4Q 2023:
US$11.6 million), which includes a deduction of US$1.7 million in
paid taxes (4Q 2023: US$0.7 million).
Full year 2024
In 2024, Pharming revenues increased by 21% to
US$297.2 million. However, the operating loss declined to a loss of
US$8.6 million, compared to a loss of US$5.4 million in 2023.
Similarly, the net loss declined to US$11.0 million, compared to a
net loss of US$10.5 million in 2023.
This section will further elaborate on
Pharming's financial performance in 2024.
Revenues and Gross Profit
Total revenues for 2024 grew by 21%, reaching
US$297.2 million, compared to US$245.3 million in 2023. Total
RUCONEST® revenues were 11% higher at US$252.2 million, compared to
revenues of US$227.1 million for 2023. Joenja® revenues amounted to
US$45.0 million in 2024, a 147% increase compared to 2023 (first
sales commenced at the start of the second quarter of 2023). This
increase was primarily driven by an increase in volume.
Cost of sales increased by 40% from US$25.2
million in 2023 to US$35.4 million in 2024. Cost of inventories
recognized as expenses in 2024 amounted to US$25.6 million compared
to US$21.4 million in 2023. In addition to the higher unit sales
volume, the rise was primarily attributed to rising production
costs for RUCONEST®. The remainder of the increase in cost of sales
in 2024 mainly stem from one-off impairment charges on inventory of
US$4.8 million (2023: US$1.7 million) and royalty payments to
Novartis on Joenja® sales of US$4.9 million (2023: US$2.1
million).
Gross profit increased by US$41.7 million, or
19%, to US$261.8 million for the year 2024. The primary driver for
this increase was higher sales volumes of RUCONEST® and
Joenja®.
Other income
Other income decreased to US$2.2 million
compared to US$23.3 million in 2023. Other income in 2023 was
supported by the sale of the Rare Pediatric Disease Priority Review
Voucher (PRV) to Novartis for a pre-agreed, one-time payment of
US$21.3 million.
Operating Profit (loss) and Other Operating
Costs
For 2024, the operating loss increased to a loss
of US$8.6 million compared to a loss of US$5.4 million for the
prior year. This change was mainly due to the decrease in other
income and the expected increase in operating expenses from
US$248.8 million in 2023 to US$272.6 million, offset by the above
mentioned increase in gross profit in 2024. The increase in
operating expenses in 2024 was primarily related to a combination
of continuing investments in Joenja® in the U.S., launch
preparation for leniolisib outside of the U.S., increasing R&D
investments to expand the addressable patient population for
leniolisib, and increased payroll expenses due to business growth.
Operating expenses in 2024 include one-off expenses, totaling
US$6.2 million, including expenses related to the full impairment
of the DSP facility at Pivot Park in Oss, the Netherlands,
amounting to US$5.1 million (2023: US$4.7 million). Also
contributing to the increase were legal and advisory fees
associated with the acquisition of Abliva AB, totaling US$1.1
million in the fourth quarter of 2024.
Operating Profit (loss) excluding one-time
events
The 2023 operating expenses included milestone
payments for Joenja® of US$10.5 million in the second quarter and
other income included one-time proceeds from the PRV sale of
US$21.3 million. When compared on a like-for-like basis, excluding
these one-time events in 2023, the operating loss decreased from
US$16.2 million in 2023 to US$8.6 million in the current year.
Finance income and expenses
The net finance result amounted to a gain of
US$1.9 million compared to a loss of US$6.3 million in 2023. This
was primarily driven by a fair value gain of US$7.0 million upon
the reclassification of the convertible bond-related derivative to
equity. This fair value gain was a result of the decrease in value
of the option component classified as a derivative from issuance
until the physical settlement date of the newly issued convertible
bond. Further positive results stem from favorable EUR/USD exchange
rate developments, which led to a foreign currency gain of US$2.0
million compared to a loss of US$3.0 million in 2023. In addition,
interest income from investments in marketable securities, which
commenced in the second quarter of 2023, increased by US$1.2
million. These positive results were partially offset by the
negative fair value adjustments in the BioConnection preference
share of US$2.1 million (2023: US$0.9 million negative), US$2.8
million higher interest expenses and fees of US$1.2 million related
to the 2024 issued convertible bond.
Income tax credit (expense)Income tax credit
(expense) shifted from a US$1.5 million credit for the year ending
December 31, 2023, to a US$2.5 million expense for the year ending
December 31, 2024. This tax expense mainly results from the profits
of Pharming in the US being taxed against a US Federal and State
combined tax rate of 27.96%, while the losses in the Netherlands
only partly result in an offsetting tax credit, as the share-based
compensation expenses and losses in associates are generally
non-deductible based on Dutch tax law.Net loss for the
year
The Company had a net loss of US$11.0 million in
2024, compared to a net loss of US$10.5 million in 2023. In
addition to the support in other income from the PRV and the
milestone payments for Joenja® in 2023, the change was mainly due
to an increase in gross profit, favorable EUR/USD exchange rate
developments and the fair value gain upon the reclassification of
the convertible bond-related derivative to equity, offset by an
increase in operating expenses, higher tax expenses and higher
interest expenses and fees on the 2024 issued convertible
bonds.
Intangible assets
In 2024, intangible assets decreased by US$10.2
million, from US$71.3 million in 2023 to US$61.0 million in 2024.
This decrease primarily resulted from regular amortization
(amounting to US$6.3 million) and negative foreign currency effects
(equivalent to US$4.0 million).
The amortization relates to regular amortization
of software, the RUCONEST® licenses (US and EU) and the Joenja®
license. The RUCONEST® license has a remaining amortization period
of 13 years for the US and 7 years for the EU. The Joenja® license
has a remaining amortization period of 12 years.
Property, plant and equipment
The value of property, plant and equipment
decreased from US$9.7 million in 2023 to US$7.8 million in 2024.
This decline was primarily driven by regular depreciation (US$2.3
million) and negative foreign currency effects (US$0.5 million),
partially offset by capital expenditures (US$0.8 million).
Right-of-use assets
The right-of-use assets decreased from US$23.8
million in 2023 to US$16.1 million in 2024. This decline was
primarily driven by regular depreciation (amounting to US$3.9
million), negative foreign currency effects (equivalent to US$0.9
million) and the full impairment of the DSP facility at Pivot Park
in Oss, the Netherlands (totaling US$5.1 million).
The decrease in the right-to-use assets is
partially offset by additions of cars (US$2.4 million) and building
remeasurements (US$0.3 million). The 2024 building remeasurements
were related to adjustments in the existing right-of-use assets to
account for inflation-related higher lease payments.
Investments
Investments decreased by US$6.2 million to
US$4.2 million as of December 31, 2024. This decline was primarily
driven by the disposal of the equity investment in Orchard of
US$2.1 million (following take-over), Pharming’s share in the net
loss of BioConnection of US$1.1 million and US$0.6 million
impairment to equity value (accounted for using the equity method)
and a fair value decrease of US$2.1 million in the preference share
in BioConnection, carried at fair value through the statement of
profit and loss (FVTPL).
Inventories
Inventories decreased from US$56.8 million as of
December 31, 2023 to US$55.7 million as of December 31, 2024.
Cash and cash equivalents and marketable
securities
Cash and cash equivalents alone decreased by
US$6.8 million to US$54.9 million as of December 31, 2024. The cash
and cash equivalents position is managed in combination with the
marketable securities position.
The combined total of cash and cash equivalents,
together with restricted cash and marketable securities decreased
from US$215.0 million at year-end 2023 to US$169.4 million at
year-end 2024. This decrease was primarily driven by paid taxes of
US$15.6 million and the repurchase of the outstanding convertible
bonds amounting to US$134.9 million, offset by net proceeds of
US$104.5 million for newly issued convertible bonds. Following
negative operating cash flow in the first half of 2024, the third
and fourth quarter operating cash flows were positive, also when
adjusted for share based compensation.
Shareholders’ equity
Shareholders’ equity increased by US$1.6 million
from US$218.8 million for the year ended December 31, 2023 to
US$220.4 million for the year ended December 31, 2024. This
increase was primarily driven by transactions recognized directly
in equity relating to share based compensation and exercised
options (totaling US$13.9 million) and the recognition of the value
conversion rights of US$12.2 million related to the issued
convertible bond in 2024. These increases were offset by the net
loss of US$12.5 million and the other comprehensive loss of US$11.9
million. The other comprehensive loss was primarily driven by
currency translation differences.
Convertible bond
The convertible bond position has decreased by
US$56.0 million to US$82.4 million at year-end 2024, moving from
US$138.4 million as of December 31, 2023. This decrease was mainly
driven by the repurchase of the outstanding convertible bonds
amounting to US$134.9 million, offset by the initial recognition of
the newly issued convertible bonds for US$81.8 million. The
difference between the initial recognition of the newly issued
convertible bonds and the respective net proceeds of US$104.5
million relates to the initial value of the conversion option
component. Following a fair value gain of US$7.0 million until the
physical settlement date, the value of the conversion option
component was reclassified to equity. Subsequently, the value of
this equity component is not remeasured and amounts to US$12.2
million, net of income tax effects, at December 31, 2024.
Lease liabilities
Lease liabilities decreased by US$3.2 million,
moving from US$33.1 million as of December 31, 2023 to US$29.9
million as of December 31, 2024. This decrease was primarily driven
by monthly or quarterly lease payments of US$5.1 million and for
the most part offset by new leases (amounting to US$2.4
million).
Outlook/Summary
For 2025, the Company anticipates:
- Total revenues between US$315
million and US$335 million (6% to 13% growth), with quarterly
fluctuations expected.
- Total operating expenses not to
exceed the prior year pre-Abliva impact, and a preliminary estimate
of US$30 million in Abliva-related operating expenses, including
research and development and non-recurring transaction and
integration expenses.
- Significant progress finding
additional APDS patients in the U.S., supported by VUS validation
efforts and subsequently converting patients to paid Joenja®
(leniolisib) therapy.
- Increasing ex-U.S. revenues for
leniolisib - driven by funded access programs and commercial
availability in the U.K.
- Progress towards additional
regulatory approvals for leniolisib for APDS patients 12 years of
age or older, and submitting regulatory filings in Japan and for
pediatric label expansion in key global markets.
- Advancing the two ongoing Phase II
clinical trials in PIDs with immune dysregulation to significantly
expand the long-term commercial potential of leniolisib.
- Advancing the ongoing pivotal
FALCON clinical study for KL1333 in mitochondrial DNA-driven
primary mitochondrial diseases.
- 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 2025
is provided.
Additional informationPresentation
The conference call presentation is available on
the Pharming.com website from 07:30 CET today.
Conference Call
The conference call will begin at 13:30
CET/08:30 am EDT on Thursday, March 13. A transcript will be made
available on the Pharming.com website in the days following the
call.
Please note, the Company will only take
questions from dial-in attendees.
Webcast Link: https://edge.media-server.com/mmc/p/7qvoovac
Conference call dial-in details:
https://register.vevent.com/register/BI7d716758d12d4ad588b70fdbca665b6a
Additional information on how to register for
the conference call/webcast can be found on thePharming.com
website.
Financial Calendar 2025
Annual Report and 20-F
2024 April
31Q 2025 financial results
May
8Annual General Meeting of
Shareholders June
112Q/1H 2025 financial
results July
313Q 2025 financial
results November
6
For further public information, contact:
Pharming Group N.V., Leiden, the
NetherlandsMichael Levitan, VP Investor Relations & Corporate
Communications T: +1 (908) 705 1696E: investor@pharming.com
FTI Consulting, London, UKSimon Conway/Alex
Shaw/Amy ByrneT: +44 203 727 1000
LifeSpring Life Sciences Communication,
Amsterdam, the NetherlandsLeon MelensT: +31 6 53 81 64 27E:
pharming@lifespring.nl
About Pharming Group N.V.
Pharming Group N.V. (EURONEXT Amsterdam:
PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated
to transforming the lives of patients with rare, debilitating, and
life-threatening diseases. Pharming is commercializing and
developing an innovative portfolio of protein replacement therapies
and precision medicines, including small molecules and biologics.
Pharming is headquartered in Leiden, the Netherlands, and has
employees around the globe who serve patients in over 30 markets in
North America, Europe, the Middle East, Africa, and
Asia-Pacific.
For more information, visit www.pharming.com and
find us on LinkedIn.
Risk profile
We continue to closely monitor and manage the
key risks and opportunities, and will respond appropriately to any
emerging risk. We will issue a full overview of our risk profile in
our Annual report 2024 to be published on April 3, 2025.
Related party transactions
There are no material changes in the nature,
scope, and (relative) scale in this reporting period compared to
last year.
Auditor’s involvement
The Condensed Consolidated Interim Financial
Statements have not been audited by the Company’s statutory
auditor.
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
Pharming's preclinical studies and clinical trials of its product
candidates, Pharming's clinical and commercial prospects, and
Pharming's expectations regarding its projected working capital
requirements and cash resources, which statements are subject to a
number of risks, uncertainties and assumptions, including, but not
limited to the scope, progress and expansion of Pharming's clinical
trials and ramifications for the cost thereof; and clinical,
scientific, regulatory, commercial, competitive and technical
developments. In light of these risks and uncertainties, and other
risks and uncertainties that are described in Pharming's 2023
Annual Report and the Annual Report on Form 20-F for the year ended
December 31, 2023, filed with the U.S. Securities and Exchange
Commission, the events and circumstances discussed in such
forward-looking statements may not occur, and Pharming's actual
results could differ materially and adversely from those
anticipated or implied thereby. All forward-looking statements
contained in this press release are expressly qualified in their
entirety by the cautionary statements contained or referred to in
this section. Readers should not place undue reliance on
forward-looking statements. Any forward-looking statements speak
only as of the date of this press release and are based on
information available to Pharming as of the date of this release.
Pharming does not undertake any obligation to publicly update or
revise any forward-looking statement as a result of new
information, future events or other information.
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.
Pharming Group N.V.
Condensed Consolidated Financial
Statements in U.S. Dollars (unaudited)
For the period ended December 31, 2024
- 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 INTERIM STATEMENT OF
INCOME |
For the
period ended December 31 |
|
Amounts in US$ ‘000 |
2024 |
2023 |
Revenues |
297,200 |
245,316 |
Costs of sales |
(35,399) |
(25,212) |
Gross profit |
261,801 |
220,104 |
Other income |
2,177 |
23,349 |
Research and development |
(83,161) |
(68,914) |
General and administrative |
(70,619) |
(55,877) |
Marketing and sales |
(118,819) |
(124,049) |
Other Operating Costs |
(272,599) |
(248,840) |
Operating profit (loss) |
(8,621) |
(5,387) |
Fair value gain (loss) on revaluation |
4,990 |
(930) |
Other finance income |
6,820 |
3,663 |
Other finance expenses |
(9,944) |
(9,069) |
Finance result, net |
1,866 |
(6,336) |
Share of net profits (loss) in associates using the equity
method |
(1,758) |
(289) |
Profit (loss) before tax |
(8,513) |
(12,012) |
Income tax credit (expense) |
(2,514) |
1,464 |
Profit (loss) for the period |
(11,027) |
(10,548) |
Basic earnings per share (US$) |
(0.016) |
(0.016) |
Diluted earnings per share (US$) |
(0.016) |
(0.016) |
CONDENSED CONSOLIDATED INTERIM STATEMENT OF COMPREHENSIVE
INCOME |
For the
period ended December 31 |
|
Amounts in US$ ‘000 |
2024 |
2023 |
Profit (loss) for the period |
(11,027) |
(10,548) |
Currency translation differences |
(11,992) |
5,936 |
Items that may be subsequently reclassified to profit or
loss |
(11,992) |
5,936 |
Fair value remeasurement investments |
79 |
1,167 |
Items that shall not be subsequently reclassified to profit
or loss |
79 |
1,167 |
Other comprehensive income (loss), net of tax |
(11,913) |
7,103 |
Total comprehensive income (loss) for the
period |
(22,940) |
(3,445) |
CONDENSED
CONSOLIDATED INTERIM BALANCE SHEET |
|
|
|
|
|
Amounts in US$ ‘000 |
December 31, 2024 |
December 31, 2023 |
Non-current assets |
|
|
Intangible assets |
61,039 |
71,267 |
Property, plant and equipment |
7,752 |
9,689 |
Right-of-use assets |
16,382 |
23,777 |
Long-term prepayments |
90 |
92 |
Deferred tax assets |
31,090 |
29,761 |
Investment accounted for using the equity method |
466 |
2,285 |
Investments in equity instruments designated as at FVTOCI |
— |
2,020 |
Investment in debt instruments designated as at FVTPL |
3,767 |
6,093 |
Restricted cash |
1,505 |
1,528 |
Total non-current assets |
122,091 |
146,512 |
Current assets |
|
|
Inventories |
55,724 |
56,760 |
Trade and other receivables |
55,079 |
46,158 |
Marketable securities |
112,949 |
151,683 |
Cash and cash equivalents |
54,944 |
61,741 |
Total current assets |
278,696 |
316,342 |
Total assets |
400,787 |
462,854 |
Equity |
|
|
Share capital |
7,769 |
7,669 |
Share premium |
488,990 |
478,431 |
Other reserves |
(222) |
(2,057) |
Accumulated deficit |
(274,675) |
(265,262) |
Shareholders’ equity |
221,862 |
218,781 |
Non-current liabilities |
|
|
Convertible bonds |
78,154 |
136,598 |
Lease liabilities |
26,968 |
29,507 |
Total non-current liabilities |
105,122 |
166,105 |
Current liabilities |
|
|
Convertible bonds |
4,245 |
1,824 |
Trade and other payables |
66,611 |
72,528 |
Lease liabilities |
2,947 |
3,616 |
Total current liabilities |
73,803 |
77,968 |
Total equity and liabilities |
400,787 |
462,854 |
CONDENSED CONSOLIDATED INTERIM STATEMENT OF CHANGES IN
EQUITY |
For the
period ended December 31 |
Attributable to owners of the parent |
|
|
|
|
|
|
Amounts in US$ ‘000 |
Share capital |
Share premium |
Other reserves |
Accumulated deficit |
Total equity |
Balance at January 1, 2023 |
7,509 |
462,297 |
(8,737) |
(256,431) |
204,638 |
Profit (loss) for the period |
— |
— |
— |
(10,548) |
(10,548) |
Reserves |
— |
— |
— |
— |
— |
Other comprehensive income (loss) for the period |
— |
— |
7,103 |
— |
7,103 |
Total comprehensive income (loss) for the
period |
— |
— |
7,103 |
(10,548) |
(3,445) |
Other reserves |
— |
— |
(423) |
423 |
— |
Income tax benefit from excess tax deductions related to
share-based payments |
— |
— |
— |
204 |
204 |
Share-based compensation |
— |
— |
— |
9,251 |
9,251 |
Options exercised / LTIP shares issued |
160 |
16,134 |
— |
(8,161) |
8,133 |
Value of conversion rights of convertible bonds |
— |
— |
— |
— |
— |
Total transactions with owners, recognized directly in
equity |
160 |
16,134 |
(423) |
1,717 |
17,588 |
Balance at December 31, 2023 |
7,669 |
478,431 |
(2,057) |
(265,262) |
218,781 |
|
|
|
|
|
|
Balance at January 1, 2024 |
7,669 |
478,431 |
(2,057) |
(265,262) |
218,781 |
Profit (loss) for the period |
— |
— |
— |
(11,027) |
(11,027) |
Reserves |
— |
— |
1,555 |
(1,555) |
— |
Other comprehensive income (loss) for the period |
— |
— |
(11,913) |
— |
(11,913) |
Total comprehensive income (loss) for the
period |
— |
— |
(10,358) |
(12,582) |
(22,940) |
Other reserves |
— |
— |
(31) |
31 |
— |
Income tax benefit from excess tax deductions related to
share-based payments |
— |
— |
— |
(66) |
(66) |
Share-based compensation |
— |
— |
— |
11,248 |
11,248 |
Options exercised / LTIP shares issued |
100 |
10,559 |
— |
(8,044) |
2,615 |
Value of conversion rights of convertible bonds |
— |
— |
12,224 |
— |
12,224 |
Total transactions with owners, recognized directly in
equity |
100 |
10,559 |
12,193 |
3,169 |
26,021 |
Balance at December 31, 2024 |
7,769 |
488,990 |
(222) |
(274,675) |
221,862 |
CONDENSED CONSOLIDATED INTERIM STATEMENT OF CASH
FLOWS |
For
the period ended December 31 |
|
Amounts in $’000 |
2024 |
2023 |
Profit (loss) before tax |
(8,513) |
(12,012) |
Adjustments to reconcile net profit (loss) to net cash used
in operating activities: |
|
|
Depreciation, amortization, impairment of non-current assets |
16,070 |
15,925 |
Equity settled share based payments |
11,248 |
9,251 |
Fair value loss (gain) on revaluation |
(4,990) |
930 |
Gain on disposal from PRV sale |
— |
(21,279) |
Disposal of leases |
22 |
— |
Other finance income |
(6,820) |
(3,663) |
Other finance expenses |
9,887 |
9,069 |
Share of net profits in associates using the equity method |
1,758 |
289 |
Other |
— |
(1,079) |
Operating cash flows before changes in working
capital |
18,662 |
(2,569) |
Changes in working capital: |
|
|
Inventories |
(503) |
(14,434) |
Trade and other receivables |
(6,783) |
(18,539) |
Payables and other current liabilities |
(2,769) |
16,228 |
Restricted cash |
(17) |
(216) |
Total changes in working capital |
(10,072) |
(16,961) |
|
|
|
Interest received |
5,201 |
2,883 |
Income taxes received (paid) |
(15,584) |
(655) |
Net cash flows generated from (used in) operating
activities |
(1,793) |
(17,302) |
|
|
|
Capital expenditure for property, plant and equipment |
(790) |
(1,437) |
Proceeds on PRV sale |
— |
21,279 |
Investment intangible assets |
(6) |
(27) |
Disposal of investment designated as at FVOCI |
2,098 |
— |
Purchases of marketable securities |
(284,314) |
(382,014) |
Proceeds from sale of marketable securities |
314,630 |
232,811 |
Net cash flows generated from (used in) investing
activities |
31,618 |
(129,388) |
|
|
|
Payment of lease liabilities |
(4,008) |
(4,038) |
Interests on lease liabilities |
(1,141) |
(1,088) |
Net proceeds of issued convertible bonds |
104,539 |
— |
Repurchase of convertible bonds |
(134,924) |
— |
Interests on convertible bonds |
(4,457) |
(4,046) |
Settlement of share based compensation awards |
5,579 |
8,133 |
Net cash flows generated from (used in) financing
activities |
(34,412) |
(1,039) |
|
|
|
Increase (decrease) of cash |
(4,587) |
(147,729) |
Exchange rate effects |
(2,210) |
2,128 |
Cash and cash equivalents at the beginning of the period |
61,741 |
207,342 |
|
|
|
Total cash and cash equivalents at December
31 |
54,944 |
61,741 |
---ENDS---
- Pharming reports 4Q_FY 2024 financial results_EN_13MAR25
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