Pharming Group Q1 Earnings Call Highlights

Key Points

• RUCONEST revenue fell (down ~15% Y/Y) largely due to specialty pharmacy inventory drawdown and the company’s exit from certain non‑U.S. markets, but management expects inventory normalization in H2 and highlights continued demand with ~50 new patient enrollments and 23 new prescribers.
• Joenja is a key growth driver—revenues rose 34% Y/Y (about $14.1M) with U.S. paid patients up 25% Y/Y—and Pharming has resubmitted a pediatric sNDA for ages 4–11 with an FDA decision expected within six months and a second lower‑dose submission planned for the summer.
• Pharming reiterated 2026 revenue guidance of $405–$425 million despite Q1 revenue of EUR72.4M (down 8%), reported positive operating cash flow (~EUR2M) and EUR171.8M in cash/marketable securities, while keeping 2026 operating expense guidance and adding R&D investment ahead of two Phase II leniolisib readouts later this year.

Pharming Group NASDAQ: PHAR executives said first-quarter 2026 results reflected an expected decline in RUCONEST revenue tied largely to specialty pharmacy inventory movements and the company’s planned exit from certain non-U.S. markets, while Joenja continued to post strong growth and the company advanced regulatory and clinical milestones across its pipeline.

RUCONEST revenue declined as inventory dynamics played out

CEO Fabrice Chouraqui said quarterly revenue fell primarily due to RUCONEST, a decline he said was “largely expected due to inventory drawdown at specialty pharmacy,” which the company previously discussed on its fourth-quarter 2025 call. Chouraqui also pointed to the “commercial exit from non-U.S. markets” as a contributor to the year-over-year decline, a decision the company announced last year “as part of our renewed financial discipline since the commercialization of RUCONEST in this market was not financially sustainable.”

Chief Commercial Officer Leverne Marsh said RUCONEST revenue was down 15% year-over-year, driven by three factors: inventory dynamics that reduced quarterly revenue by 8%, the ex-U.S. exit contributing about 3%, and what she described as a measured impact from competition in the U.S. hereditary angioedema (HAE) market.

Despite the headline decline, Marsh emphasized continued demand indicators underneath revenue. She said the company added about 50 new patient enrollments in the quarter and brought on 23 new prescribers. Marsh framed these trends as evidence that clinicians continue to use RUCONEST for a “high attack, high severity” subset of patients.

On competitive dynamics, Marsh said that nine months after the launch of a new oral competitor in the U.S., the “overwhelming majority” of RUCONEST patients have remained on therapy. She added that among those who explored alternatives, “many high-burden patients are returning to RUCONEST” when response to new treatments is not adequate.

During Q&A, Marsh explained why the company expects additional RUCONEST pressure in the second quarter, citing the lag needed to see trialing and switching behavior through “three to four reorder cycles.” CFO Kenneth Lynard added that the company expects inventory patterns to normalize, saying Pharming anticipates an inventory build in the second half of the year “to basically reflect the demand.”

Chouraqui clarified that the roughly 50 new patient enrollments discussed in the quarter represent patients “in the pipes” who have received a prescription but are not yet necessarily on therapy, noting there is typically a delay between enrollment and treatment start.

Joenja revenue rose 34% as U.S. patient counts increased

Pharming highlighted Joenja as an early-stage growth driver. Chouraqui said Joenja revenues grew 34% year-over-year with “strong momentum both in the U.S.” and in international markets. Marsh reported Joenja revenue of $14.1 million globally in the quarter and said that by quarter-end the company had 127 patients on paid therapy in the United States, a 25% increase over the first quarter of 2025.

Marsh said Pharming added seven net new U.S. patients on paid therapy in the quarter, an acceleration compared with the prior two quarters. She also cited an 85% U.S. fill rate, which she attributed to reimbursement support and patient services.

Beyond patients already on therapy, Marsh said the company has identified 187 U.S. APDS patients older than 12 who are eligible, plus 57 eligible patients in the four-to-11 age group, calling pediatrics “the next frontier for growth in the U.S.”

On international progress, Marsh said Pharming is seeing strong uptake in the U.K. and growth in government-supported access programs elsewhere. Looking ahead, she said the company is positioning for launches in Europe and Japan later this year. In response to an analyst question, Marsh said the first European launch is expected in Germany “toward the second quarter,” and she said Pharming anticipates commercial, paid patients in the second quarter. She also said the company expects to launch in Japan in August.

Pediatric Joenja resubmission underway after FDA CRL

Management provided an update on its efforts to expand Joenja’s U.S. label into pediatrics (ages four to 11) following a complete response letter (CRL) received in January. The company said it held a Type A meeting with the FDA at the end of March, which included two APDS expert physicians. Executives described the interaction as constructive and said the FDA recognized the unmet need and challenges of recruiting young children into trials for an ultra-rare disease.

Chief Medical Officer Anurag Relan said Pharming and the FDA aligned on a two-step approach. The company resubmitted the sNDA in April for the 40 mg and 50 mg doses, which Relan said cover “a meaningful proportion” of children ages four to 11. He said an FDA decision is expected within six months or sooner, and the company plans to issue a press release upon FDA acceptance.

Relan said a second sNDA for the lowest-weight patients is planned for the summer, also expected to follow a six-month review timeline. Responding to questions about dosing, Relan said the Type A discussion did not change Pharming’s overall dosing strategy and that the planned low-dose submission does not require an additional clinical trial.

Marsh said roughly half of the identified four-to-11 population would be eligible for the higher-dose resubmission, with the other half expected to fall into the lower-dose group.

Pipeline updates: phase II readouts and expanded access signals

Pharming also discussed lifecycle expansion opportunities for leniolisib (Joenja) beyond APDS, focusing on primary immunodeficiencies (PIDs) with immune dysregulation, including common variable immune deficiency (CVID) with immune dysregulation.

Relan said Pharming has two phase II, proof-of-concept studies that are now fully enrolled:

• A multicenter CVID study enrolling 20 patients
• A genetic PID study conducted at the NIH enrolling 12 patients

Relan said both are single-arm, open-label dose range-finding studies, and readouts are expected later this year. He added that one study is a month shorter in duration, so results could become available slightly earlier for one program than the other.

At the Clinical Immunology Society meeting, Relan said Pharming and collaborators are presenting seven abstracts, including data from an expanded access program in which six CVID or CVID-like patients with immune dysregulation received leniolisib for a median of 1.4 years. Relan said clinicians reported improvement and “no patient showing progression” across manifestations including cytopenias, splenomegaly, lymphadenopathy, liver disease, and lung disease. He also cited immune profile changes, including reduced transitional and CD21 low B cells, which he said are consistent with PI3K delta pathway modulation seen in APDS.

Relan cautioned the dataset was clinician-reported and not from a prospective clinical study, but he called the consistency of improvement across endpoints an encouraging early signal ahead of formal study readouts in the second half of the year.

Chouraqui also pointed to napazimone (formerly KL1333) for primary mitochondrial disease as a major pipeline program, saying the registrational study is expected to complete enrollment this year with a readout next year.

Financial results, cash flow, and guidance reiterated

CFO Kenneth Lynard said first-quarter revenue totaled EUR 72.4 million, down 8% year-over-year. He reiterated RUCONEST revenue declined 15% and attributed most of the change to the expected U.S. inventory normalization (an 8% headwind, consistent with the 7% to 9% headwind discussed on the prior call) and the ex-U.S. exit (3%). He also noted that the first quarter is typically the lowest seasonal quarter for RUCONEST due to ordering patterns.

Joenja revenue increased 34% year-over-year, and Lynard said revenue was “modestly affected by inventory timing,” adding that excluding this effect growth would have been $1 million to $2 million higher.

Lynard said operating expenses fell 9% year-over-year, and on an adjusted basis—excluding non-recurring acquisition costs tied to Abliva in the prior year—expenses were flat, which he said reflected the company’s ability to increase pipeline investment without increasing overall costs. Pharming generated EUR 2 million in positive operating cash flow in the quarter. Total cash and marketable securities fell EUR 9.3 million to EUR 171.8 million, driven primarily by a EUR 12.3 million payment related to early termination of a facility lease.

Management reaffirmed 2026 revenue guidance of $405 million to $425 million, which the company said implies 8% to 13% growth versus 2025. Lynard said the outlook assumes low single-digit RUCONEST growth at the midpoint, with pressure expected in the second quarter and growth anticipated in the second half. He said guidance now includes expected U.S. pediatric label revenues later in the year, which previously had been excluded. Lynard added that hitting the upper end of the range would be most influenced by faster-than-expected pediatric approval and launch timing.

Pharming reiterated an operating expense outlook of $330 million to $335 million for 2026, including $60 million in incremental R&D investment to advance the pipeline and “up to $30 million additional for the development of napazimone.” Lynard also referenced a previously announced 20% structural headcount reduction in G&A, which he said delivers a $9 million benefit, while marketing and sales spending remains stable. Gross margin is expected to be about 90%.

In closing remarks, Chouraqui said the company believes it is making “important progress across the business” while maintaining financial discipline, and he pointed to upcoming catalysts including the two phase II leniolisib readouts later this year and completion of enrollment for the napazimone registrational study.

About Pharming Group NASDAQ: PHAR

Pharming Group N.V. is a clinical-stage biopharmaceutical company headquartered in Leiden, the Netherlands, with a primary focus on developing and commercializing innovative protein replacement therapies for patients living with rare diseases. The company employs a proprietary transgenic technology platform designed to produce recombinant human proteins in the milk of transgenic animals, enabling scalable and cost-efficient manufacturing of complex therapeutic proteins.

The company's lead product, RUCONEST (recombinant human C1 esterase inhibitor), is approved for the treatment of acute hereditary angioedema (HAE) attacks in multiple markets, including the United States and Europe.

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