Pharming Group reports second quarter and first half 2024 financial results and provides business update

Second quarter 2024 total revenues increased by 35% to US$74.1 million, compared to the second quarter 2023, driven by strong RUCONEST® and Joenja® revenue growth

RUCONEST® second quarter revenue increased by 23% to US$63.0 million, compared to the second quarter 2023

Joenja® (leniolisib) second quarter revenue increased by 16% to US$11.1 million, compared to the first quarter 2024

First half total revenues increased by 33% to US$129.7 million, compared to the first half 2023

On track for 2024 total revenue guidance of US$280 million - US$295 million (14 - 20% growth)

Overall cash and marketable securities declined to US$161.8 million at the end of the second quarter 2024 from US$203.5 million at the end of the first quarter 2024, primarily due to convertible bond refinancing

Pharming to host a conference call today at 13:30 CEST (7:30 am EDT)

Leiden, the Netherlands, August 1, 2024: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM/Nasdaq: PHAR) presents its preliminary (unaudited) financial report for the second quarter and first half year ended June 30, 2024.

Chief Executive Officer, Sijmen de Vries, commented:

“Pharming has delivered another strong quarter of revenue growth, increasing quarterly revenues by 35% to US$74.1 million. We are firmly on track to meet our 2024 total revenue guidance of US$280 million - US$295 million. We also narrowed our operating loss on a like for like basis compared to the second quarter of the prior year.

RUCONEST® performed particularly strongly, with second quarter revenue increasing by 23% compared to 2Q 2023, and we have seen continued strength in underlying demand as well as a growing number of new patient enrollments.

A year after the U.S. launch of Joenja®, the number of new patients on therapy is increasing quarter-on-quarter, with an increase of eight patients this quarter compared to two in the first quarter, and we are seeing high adherence rates for patients on paid therapy. Our patient finding and family testing efforts are continuing to help get new patients diagnosed, and we have seen positive early results on Variant of Uncertain Significance (VUS) reclassifications which suggest a significant increase in the identified APDS patient population in the US. We continue to work with regulatory authorities worldwide to make Joenja® (leniolisib) available to as many patients as possible, and expect a decision on the Marketing Authorisation Application from the UK MHRA in the fourth quarter. While we were disappointed by the delay to the European marketing authorisation, we are continuing to work closely with the EMA and CHMP, and are pleased that the CHMP determined the clinical benefit of leniolisib to be positive.

As we work to expand the leniolisib market opportunity to other primary immunodeficiency (PID) disorders, we will initiate the Phase II proof-of-concept clinical trial in PIDs with immune dysregulation linked to PI3Kẟ signaling in the third quarter. We are also continuing to prepare a clinical development plan for a third PID indication.

We expect this positive momentum to continue for the rest of the year, and I would like to thank all our employees for their significant achievements in the first half.”

Second quarter and first half highlights

Commercialized assets

RUCONEST® marketed for the treatment of acute HAE attacks

RUCONEST® continued to perform well in the second quarter of 2024, with revenue of US$63.0 million, a 23% increase compared to the second quarter of 2023. Revenue for the first half of 2024 was US$109.0 million, a 16% increase compared to the same period in 2023.

The U.S. market contributed 96% of second quarter revenues, while the EU and Rest of World contributed 4%.

In the U.S. market, we saw continued strength in the second quarter in underlying in-market demand for RUCONEST®, including over 100 new patient enrollments. We achieved strong overall performance in the second quarter in other leading key revenue indicators including new physicians prescribing RUCONEST® and the total number of patients on therapy. Increasing enrollments helped to drive a sharp increase in unique patient shipments in the second quarter.

Joenja® (leniolisib) marketed in the U.S. - the first and only approved disease modifying treatment for APDS

Joenja® revenues increased to US$11.1 million in the second quarter of 2024, a 16% increase compared to the first quarter of 2024 and a 192% increase compared to the second quarter of 2023. This increase was mostly driven by higher volume from the continued increase in patients on paid therapy in the U.S., higher adherence rates for patients on paid therapy, and revenues from EU and Rest of World. Revenue for the first half of 2024 was US$20.7 million, compared to US$3.8 million for the same period in 2023.

As of June 30, 2024, we have 91 patients on paid therapy in the U.S. and an additional two patients enrolled and pending authorization, representing an increase of eight patients on paid therapy during the second quarter as compared to an increase of two patients during the first quarter.

EU and Rest of World revenues are from product provided on a named patient basis. Pharming has named patient and other funded early access programs in certain countries where leniolisib is not commercially available: physicians can request leniolisib on behalf of individual patients living with APDS who meet the eligibility criteria and receive local health authority approval.

APDS patient finding

As of June 30, 2024, Pharming had identified over 870 diagnosed APDS patients of all ages in global markets, including over 230 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®. Over 780 of these globally identified patients are in target markets for Pharming in the U.S., Europe, the U.K., Japan, Asia Pacific, Middle East, Latin America and Canada, with estimated total prevalence of approximately 2,400 APDS patients.

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.

Pharming’s Variant of Uncertain Significance (VUS) resolution efforts are ongoing, including validation studies with various laboratories to confirm which VUSs can be classified as APDS. As results become available, patients with validated variants could be diagnosed with APDS and, therefore, potentially be eligible for Joenja® treatment. Completion of the in vitro high throughput screening study is expected during the fourth quarter of 2024.

In the U.S. market, the number of diagnosed APDS patients increased by 10 during the second quarter 2024.

APDS patient finding - VUS resolution

Pharming has identified approximately 1,200 patients in the U.S. with a VUS in the PIK3CD or PIK3R1 genes and is performing 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 pathogenic / likely pathogenic variants classified as APDS.

A literature review, which includes more than 1.5 million patients, showed that 20% of reclassified VUSs are upgraded to likely pathogenic / pathogenic. Pharming also recently completed a pilot study with 25 patients with a VUS who then underwent further testing of the PI3K-AkT-mTOR pathway, and results were consistent, with VUS reclassifications to APDS for five of these patients.

Together, these data suggest that there could be a significant increase in the number of APDS patients in the US once those patients with a VUS are reclassified.

Pharming is expanding the pilot study to allow additional patients with a VUS to be tested, and the data generated are being shared with central databases (ClinVar) and genetic testing labs so that the same variants found in the future may be classified correctly without need for further testing.

Additionally, Pharming is working with researchers to be able to evaluate large numbers of VUSs without the need for additional patient testing. VUS resolution via high throughput screening methods is a common approach and is accepted as strong evidence by various expert organizations including the American College of Medical Genetics (ACMG) and ClinGen (a National Institutes of Health-funded resource). An initial evaluation of this approach has recently been published and confirmed the ability to differentiate benign vs. pathogenic variants, and identified new pathogenic variants. Full results from this study are on track to be available by the end of 2024.

Joenja® (leniolisib) strategic highlights - regulatory, clinical and commercial strategy updates

Pharming made continued progress in the second quarter of 2024 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets. In addition, Pharming progressed ongoing clinical trials to support regulatory filings for approval in Japan and pediatric label expansion.

Pharming’s strategy is to expand the commercial availability of leniolisib for APDS patients to key markets in Europe, U.K., Japan, Asia Pacific, Middle East, Latin America and Canada. Pharming intends to market leniolisib directly in most of these markets following regulatory approval.

In total, there are currently 150 patients in a leniolisib Expanded Access Program (compassionate use), an ongoing clinical study, or a named patient program.

European Economic Area (EEA)

As announced on May 30, 2024, the European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP) issued an updated List of Outstanding Issues (LoOI) regarding the leniolisib Marketing Authorisation Application (MAA). The LoOI affirmed the positive clinical benefit and safety of leniolisib, in agreement with the assessment by the Ad Hoc Expert Group (AEG), and included one remaining chemistry, manufacturing and controls (CMC) request. The CMC request relates to the definition of regulatory starting materials used in the manufacturing process for leniolisib. The CHMP requested that this work be completed pre-approval and granted Pharming an extension to January 2026 to submit a response. Pharming plans to complete the manufacturing activities requested by the CHMP and submit a response prior to this deadline.

On March 12, 2024, Pharming submitted an MAA for leniolisib with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), through the International Recognition Procedure (IRP) on the basis of the U.S. FDA approval. The MAA for leniolisib was validated on April 17, 2024. Pharming received the MHRA Day 70 Request for Further Information on July 3, 2024. There were no major objections. Upon Pharming’s satisfactory response to MHRA requests, it is expected that the MHRA will issue its decision in the fourth quarter of 2024.

Additional markets - Canada

Pharming filed a regulatory submission in Canada for leniolisib for APDS in the third quarter of 2023. Pharming recently submitted a response to a Health Canada Notice of Deficiency in July 2024. Pharming is awaiting feedback on next steps but no longer anticipates regulatory approval in 2024.

Leniolisib for additional indications (PI3Kδ platform) - Primary immunodeficiencies (PIDs) beyond APDS

Pharming is planning a Phase II, proof of concept, clinical trial in targeted PID genetic disorders with immune dysregulation linked to PI3Kẟ signaling in lymphocytes, with similar clinical phenotypes to APDS. These PID disorders include ALPS-FAS, CTLA4 haploinsufficiency and PTEN deficiency. The epidemiology of these targeted PID genetic disorders suggests a prevalence of approximately five patients per million.

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 the targeted PID population. The trial has been designed to inform a subsequent Phase III program. Pharming is in the final stages of preparation for the start of the trial, which it expects to initiate shortly.

Pharming has also prioritized development of leniolisib for an additional PID indication. Pharming will provide further updates and details on our plans after obtaining regulatory feedback on the proposed clinical development plan.

Financial highlights

For the second quarter of 2024, revenues increased by US$19.2 million, or 35%, to US$74.1 million, compared to US$54.9 million in the second quarter of 2023. RUCONEST® revenues amounted to US$63.0 million, a 23% increase compared to the second quarter of 2023. The 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$11.1 million in the second quarter of 2024, a 16% increase compared to the first quarter of 2024 and a 192% increase compared to the second quarter of 2023. This increase in Joenja® revenues was primarily driven by an increase in volume.

Gross profit increased by US$16.9 million or 34% to US$66.1 million (2Q 2023: US$49.2 million), mainly due to the increase in revenues.

Other income decreased to US$0.9 million compared to US$21.9 million in the second quarter of 2023. Other income in the second quarter of 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$21.1 million.

The operating loss amounted to US$3.1 million compared to an operating profit of US$5.3 million in the second quarter of 2023. In addition to the support in gross profit and other income mentioned above, this change was mainly due to an expected increase in operating expenses from US$65.8 million in the second quarter of 2023 to US$70.1 million in the second quarter of this year. The second quarter 2023 operating expenses included milestone payments for Joenja® amounting to US$10.5 million. The increase in operating expenses was caused by 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 leniolisib franchise and increased payroll expenses due to business growth. Excluding the proceeds from the PRV sale and the milestone payment expenses for Joenja® in the second quarter of 2023, the operating loss decreased from US$5.3 million to US$3.1 million in the second quarter of the current year.

The net finance result amounted to a gain of US$3.4 million compared to a loss of US$1.8 million in the second quarter of 2023. This was primarily driven by a fair value gain of US$5.1 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. For more information on the matter, reference is made to Note 18. Convertible bonds of the Notes to the condensed consolidated interim financial statements of this press release. This fair value gain is tax exempt, resulting in a favorable income tax credit.

The Company had a net loss of US$1.2 million, compared to a net profit of US$1.3 million in the second quarter of 2023. In addition to the support in other income from the PRV sale and the milestone payments for Joenja® in the second quarter of 2023, the change was mainly due to an increase in operating expenses, offset by an increase in revenues and the fair value gain upon the reclassification of the convertible bond-related derivative to equity.

Cash and cash equivalents, including restricted cash and marketable securities, decreased from US$203.5 million at the end of first quarter of 2024 to US$161.8 million at the end of the second quarter of 2024. This decrease was primarily driven by the repurchase of the outstanding convertible bonds amounting to US$134.9 million, offset by net proceeds of US$104.8 million for newly issued convertible bonds. In addition, the decrease was accompanied by a US$12.4 million increase in receivables in the second quarter of 2024, resulting from higher revenues.

First half year 2024

Total revenues increased 33% during the first half of 2024 to US$129.7 million, versus US$97.4 million during the first half of 2023. For the first half of 2024, total RUCONEST® revenues were 16% higher at US$109.0 million, compared to revenues of US$93.6 million for the first half of 2023. Joenja® revenues amounted to US$20.7 million in the first half of 2024, a 44% increase compared to the second half of 2023. This increase in Joenja® revenues was primarily driven by an increase in volume.

Gross profit increased by US$25.7 million or 29% to US$113.3 million (1H 2023: US$87.6 million), mainly due to the increase in revenues.

Further details on revenue and gross profit segmentation is provided in Note 7. Segment information in the Notes to the condensed consolidated interim financial statements of this press release.

Other income decreased to US$1.3 million compared to US$22.5 million in the first half of 2023. Other income in the first half of 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$21.1 million.

The operating loss amounted to US$19.4 million compared to an operating loss of US$8.4 million in the first half of 2023. In addition to the support in gross profit and other income mentioned above, this change was mainly due to an expected increase in operating expenses from US$118.5 million in the first half of 2023 to US$134.0 million in the first half of this year. The second quarter 2023 operating expenses included milestone payments for Joenja® amounting to US$10.5 million. The increase in operating expenses was caused by 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 leniolisib franchise and increased payroll expenses due to business growth. Excluding the proceeds from the PRV sale and the milestone payment expenses for Joenja® in the first half of 2023, the operating loss increased from US$19.0 million to US$19.4 million in the first half of the current year.

The net finance result amounted to a gain of US$3.6 million compared to a loss of US$4.5 million in the first half of 2023. This was primarily driven by a fair value gain of US$5.1 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. For more information on the matter, reference is made to Note 18. Convertible bonds of the Notes to the condensed consolidated interim financial statements of this press release. In addition, EUR/USD exchange rate developments led to a foreign currency gain of US$0.2 million compared to a loss of US$2.3 million in the first half of 2023.

The Company had a net loss of US$13.7 million, compared to a net loss of US$10.9 million in the first half of 2023. In addition to the support in other income from the PRV and the milestone payments for Joenja® in the first half of 2023, the change was mainly due to an increase in revenues, 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.

Cash and cash equivalents, including restricted cash and marketable securities, decreased from US$215.0 million at the end of 2023 to US$161.8 million at the end of the first half of 2024. This decrease was primarily driven by the repurchase of the outstanding convertible bonds amounting to US$134.9 million, offset by net proceeds of US$104.8 million for newly issued convertible bonds, and negative cash flows from operations amounting to US$20.9 million.

On 5 October 2023, Orchard Therapeutics Plc. (Orchard) announced it had entered into a definitive agreement with Japanese company Kyowa Kirin Co. LTD for the acquisition of Orchard. During the first half of 2024, Pharming received US$2.0 million in cash for its shares held in Orchard. Pharming has terminated the research collaboration & licensing agreement with Orchard Therapeutics and discontinued the OTL-105 program.

For 2024, the Company anticipates:

Total revenues between US$280 million and US$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 and Australia.

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

The conference call presentation is available on the Pharming.com website from 07:30 CEST today.

The conference call will begin at 13:30 CEST/07:30 EDT on Thursday, August 1. 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.

Conference call dial-in details:

Additional information on how to register for the conference call/webcast can be found on the

Pharming.com website.

Financial Calendar 2024

3Q 2024 financial results                        October 24

For further public information, contact:

Pharming Group N.V., Leiden, the Netherlands

Michael Levitan, VP Investor Relations & Corporate Communications

T: +1 (908) 705 1696

E: investor@pharming.com

FTI Consulting, London, UK

Victoria Foster Mitchell/Alex Shaw

LifeSpring Life Sciences Communication, Amsterdam, the Netherlands

T: +31 6 53 81 64 27

E: pharming@lifespring.nl