Pharming Group reports first quarter 2025 financial results and provides business update

   -- First quarter 2025 total revenues increased by 42% to US$79.1 million, 
      compared to the first quarter 2024 
 
   -- RUCONEST$(R)$ revenue increased by 49% to US$68.6 million, compared to the 
      first quarter 2024, reflecting continued momentum and the product's 
      unique position in the on-demand HAE market 
 
   -- Joenja(R) (leniolisib) volume increase by 18% and revenue increased by 9% 
      to US$10.5 million, compared to the first quarter of 2024, with 
      accelerating patient uptake compared to the prior few quarters 
 
   -- Operating profit, adjusted to exclude the impact of non-recurring Abliva 
      acquisition-related expenses, amounted to US$0.8 million compared to a 
      US$16.3 million loss in the first quarter 2024 
 
   -- Launched Joenja(R) (leniolisib) in England and Wales in April and 
      preparing to file for U.S. FDA approval for pediatrics in the third 
      quarter 2025 
 
   -- Completed the acquisition of Abliva AB during the quarter and, in April, 
      promptly started the second wave of recruitment for the pivotal FALCON 
      clinical trial in mitochondrial DNA-driven primary mitochondrial diseases 
 
   -- 2025 total revenue guidance raised to US$325 - US$340 million, up from 
      prior US$315 - US$335 million 
 
   -- Announces that Jeroen Wakkerman, Chief Financial Officer, will leave 
      Pharming at the end of the month, to pursue other opportunities 
 
   -- Pharming to host a conference call today at 13:30 CEST (7:30 am EDT) 

Leiden, the Netherlands, May 8, 2025: Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM / Nasdaq: PHAR) presents its preliminary unaudited financial report for the three months ended March 31, 2025.

Chief Executive Officer, Fabrice Chouraqui commented:

"We ended 2024 with great momentum, and 2025 has started on an even stronger note. First quarter RUCONEST(R) revenue increased by 49% vs. 2024 and we are seeing an acceleration in the number of Joenja(R) patients on commercial therapy ahead of the growth expected in the second half from VUS patients reclassified as APDS patients. Our strong performance and solid fundamentals give us the confidence to raise our full-year revenue guidance. In addition, we are optimizing capital allocation to drive sustainable growth by targeting a 15% or $10 million annual reduction in G&A expenses.

Our efforts to make Joenja(R) available to additional APDS patients are bearing fruit. We launched Joenja(R) in England and Wales in late April following a positive reimbursement decision from NICE and we received our fourth country regulatory approval in Australia. We are preparing to file for U.S. FDA approval for pediatrics in the third quarter and potentially launch in the first quarter of 2026.

We are making strong progress advancing high value programs in our pipeline. Patient enrollment is on track in both proof of concept trials studying leniolisib in larger PID indications. In addition, approximately a month after completing the Abliva acquisition, we successfully started the second wave of patient recruitment in the pivotal FALCON clinical trial for KL1333 in primary mitochondrial diseases, achieving a key milestone in our integration plan.

I would like to thank Jeroen Wakkerman for his contributions to the growth of Pharming and the development of the Finance team over the past four years. We have initiated a search for a new CFO to lead our financial strategy going forward.

Overall, this strong quarter is another illustration of our execution capabilities and future growth prospects. We look forward to providing future updates on our significant upcoming near- and long-term catalysts."

First quarter 2025 highlights

Commercialized products

RUCONEST(R) marketed for the treatment of acute HAE attacks

RUCONEST(R) demonstrated significant strength in the first quarter of 2025, with revenues of US$68.6 million, a 49% increase compared to the first quarter of 2024.

The U.S. market contributed 97% of first quarter revenues, while the EU and Rest of World contributed 3%.

The strong performance was mainly driven by the continued increase in prescribers and patients on therapy in the U.S. We achieved over 90 new patient enrollments in the U.S. in the first quarter, demonstrating continued strength in underlying in-market demand for RUCONEST(R). Unit sales volume in the U.S. increased by 37% due to increased demand and reduced customer inventory destocking compared to the first quarter of 2024.

Joenja(R) (leniolisib) marketed for the treatment of APDS

Joenja(R) revenues increased to US$10.5 million in the first quarter of 2025, a 9% increase compared to the fourth quarter of 2024. Unit sales volume increased by 18% due to the continued increase in patients on paid therapy. Quarter over quarter revenue growth was below unit sales volume growth, reflecting higher gross-to-net adjustments compared to the prior year. Gross-to-net adjustment in the current quarter were in line with expectations.

The U.S. market contributed 90% of first quarter revenues, while the EU and Rest of World contributed 10%.

As of March 31, 2025, we had 102 patients on paid therapy in the U.S., representing a 23% increase from the 83 patients at the end of the first quarter of 2024 and an increase of six patients during the quarter. We are making continued progress finding, enrolling and transitioning eligible patients to paid therapy and the increase in patients added during the quarter was the largest since the second quarter of 2024. The acceleration in the growth of patients on commercial therapy in the U.S. was achieved independent of the expected positive impact from Variant of Uncertain Significance, or VUS, patient reclassifications later this year.

We launched Joenja(R) in the U.K. in April. On April 23, 2025 the National Institute for Health and Care Excellence $(NICE)$ issued positive final guidance recommending Joenja(R) (leniolisib) for reimbursement and use within the National Health Service $(NHS)$ in England and Wales for the treatment of APDS in adult and pediatric patients 12 years of age and older. Leniolisib is now available for use and funded in England through the Innovative Medicines Fund, ensuring immediate patient access. In Wales, leniolisib is expected to be funded starting in July through the NHS in specialist centers.

APDS patient finding

We have now identified approximately 250 APDS patients in the U.S., including over 160 patients 12 years of age or older who are eligible for treatment with Joenja(R), demonstrating progress finding additional patients. As of December 31, 2024, we had identified over 880 diagnosed APDS patients worldwide.

APDS patient finding - VUS reclassification

There are currently over 1,300 patients in the U.S. with a Variant of Uncertain Significance, or VUS, in the PIK3CD or PIK3R1 genes. As previously communicated, an in vitro high throughput screening study was completed in the fourth quarter of 2024, identifying many novel variants leading to PI3K hyperactivity. We expect the results of this study to be published shortly, and clinical genetics laboratories in the U.S. are undertaking efforts to reclassify variants they deem to be disease-causing and thus issue amended genetic testing reports with an APDS diagnosis for many of the VUS patients. We anticipate that these initiatives will lead to the identification of new patients with APDS eligible for therapy with Joenja(R), adding an additional growth lever during the second half of 2025.

Joenja(R) (leniolisib) development

In total, there are currently 204 patients in a leniolisib Expanded Access Program (compassionate use), an ongoing clinical study, or a named patient program, of whom 187 are APDS patients.

Leniolisib for APDS

Pediatric clinical development

On May 2, 2025, positive clinical results from the multinational Phase III clinical trial evaluating leniolisib tablets in children 4 to 11 years of age with APDS were presented at the 2025 Annual Meeting of the Clinical Immunology Society (CIS) in Philadelphia, PA. The data are consistent with the improvements seen in the previously reported randomized controlled trial in adolescent and adult APDS patients. Based on U.S. FDA feedback, we plan to submit a regulatory filing for pediatric label expansion in the U.S. in the third quarter of 2025.

In April 2025, the Phase III pediatric clinical trial evaluating a new pediatric formulation of leniolisib in children 1 to 6 years of age completed enrollment with 16 patients.

Japan

We are on track to submit a regulatory filing with Japan's Pharmaceuticals and Medical Devices Agency (PMDA) in mid-2025. An approval decision would be expected nine months later based on priority review of the application due to orphan drug designation $(ODD)$ by the Ministry of Health, Labour and Welfare of Japan $(MHLW.SI)$ for the treatment of APDS.

European Economic Area $(EEA)$

In regard to the ongoing review of the leniolisib Marketing Authorisation Application $(MAA)$ for the treatment of adult and pediatric patients 12 years of age and older, we are on track to complete the manufacturing activities requested by the European Medicines Agency's (EMA) Committee for Human Medicinal Products $(CHMP)$ and submit a response by the January 2026 deadline.

Additional markets

Joenja(R) was approved in Israel in 2024 and in Australia in March 2025, with reimbursement discussions/negotiations ongoing with government payors in both countries.

Regulatory reviews are ongoing for APDS patients 12 years of age and older in Canada and Saudi Arabia, with decisions expected in 2026, and South Korea, where we filed a regulatory submission in March 2025.

Leniolisib for additional primary immunodeficiencies (PIDs)

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