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PHAR 资讯
PHAR 事件
Pharming Cuts FY26 Operating Expense Outlook to $310M-$315M
Consensus $383.35M. Cuts FY26 operating expense view to $310M-$315M from $315M-$320M. The company said, "At ESID 2026, Pharming presented positive Phase II data for leniolisib in patients with genetically defined primary immunodeficiencies (PIDs) with immune dysregulation linked to PI3Kd signaling; Pharming expects topline Phase II results from its separate trial in CVID patients with immune dysregulation, with or without an identified genetic cause, in November 2026. We are working towards a single registrational Phase III trial in the broader CVID indication, incorporating patient populations from both studies. The estimated prevalence rate for CVID with immune dysregulation is approximately 39 patients per million, compared with approximately 1.5 patients per million for APDS, representing substantial growth potential for Joenja. For Napazimone for mtDNA-driven mitochondrial disease, we expect approximately 145 of the planned 180 subjects to be enrolled in the pivotal FALCON clinical trial by the end of 2026. We now expect enrollment to be completed in the first half of 2027. Topline results from the pivotal FALCON study are expected approximately one year after the completion of enrollment."
Company Reports 21% Year-on-Year Revenue Growth in Q3
The company said, "This represents growth of approximately 21% year-on-year, reflecting strong performance from RUCONEST and Joenja. Third quarter revenues comprise approximately US$93.8 million from RUCONEST, a 14% increase compared to the third quarter of 2025 and a 30% increase compared to the second quarter of 2026, and US$23.8 million from Joenja(R), a 58% increase compared to the third quarter of 2025. The RUCONEST revenue performance reflects stabilization and continued recovery of demand in the US, early seasonal increase in channel inventory at a single U.S. specialty pharmacy customer following significant drawdowns earlier in the year, and the completion of the planned withdrawal from non-U.S. markets."
Pharming Board and CEO Chouraqui Agree on Immediate Resignation
Pharming announced that the Board of Directors and Fabrice Chouraqui have mutually agreed that Chouraqui will step down as CEO and Executive Director, with immediate effect. To ensure continuity of leadership, the Board of Directors has appointed Leverne Marsh, Chief Commercial Officer, and Kenneth Lynard, Chief Financial Officer, as Interim Co-CEOs, effective immediately. Marsh will lead the patient-focused operations while Lynard will lead the global corporate functions. The Board has started a formal search for a permanent successor. Following discussions about the execution of Pharming's strategic priorities for the next phase of the company, the Board of Directors and Chouraqui have concluded that their views on how to advance the company's strategy are not sufficiently aligned. In good consultation, the parties have therefore agreed on a leadership transition.
Pharming's Joenja Low-Dose Application Accepted by FDA
Pharming announced that the U.S. Food and Drug Administration has accepted its supplemental New Drug Application seeking approval for lower doses of Joenja, an oral, selective phosphoinositide 3-kinase delta inhibitor, for children aged 4 years and older who weigh between 13 kg and 27 kg with activated phosphoinositide 3-kinase delta syndrome, a rare primary immunodeficiency. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act target action date of January 30, 2027. The sNDA is supported by positive data from an open-label, multinational, single-arm Phase III study in children aged 4 to 11 years, which showed improvements over 12 weeks in two clinically relevant hallmarks of the condition, reduced lymphadenopathy and increased naive B cells, together indicating correction of the underlying immune defect. The submission also includes additional scientific and clinical pharmacology assessments supporting the proposed dosing in lower-weight pediatric patients. The FDA grants Priority Review to applications for medicines that, if approved, would offer significant improvements in effectiveness or safety of the treatment, prevention, or diagnosis of serious conditions. The FDA approved Joenja for adults and pediatric patients aged 12 years and older with APDS in March 2023 and expanded the approval in September 2026 to include children aged 4 to 11 years weighing at least 27 kg.
Pharming Showcases Positive Phase II Trial Data for Leniolisib at European Immunodeficiencies Meeting
Pharming announced that a late-breaking abstract highlighting positive topline data from its Phase II trial with leniolisib in genetically identifiable primary immunodeficiencies with immune dysregulation linked to PI3Kd signaling has been accepted at the 22nd Biennial Meeting of the European Society for Immunodeficiencies, which will take place October 14-17, in the Netherlands. This trial is a single-arm, open-label, intra-patient dose-escalation Phase II study of leniolisib evaluating safety and tolerability, pharmacokinetic, pharmacodynamic and efficacy measures in 13 subjects with genetically defined PIDs. The abstract will highlight leniolisib's favorable safety and tolerability profile, as well as clinical improvements across measures of immune dysregulation. Clinical results included improvements in lymphoproliferative disease, with a mean 26.4% spleen volume reduction and reductions in the size of index lesions. The safety observations were consistent with the known safety profile of leniolisib, with infections as the most common events observed in study subjects, and no new safety signals identified. Additional data will be presented at ESID 2026. Of the 13 patients enrolled in the study, nine also had a diagnosis of common variable immunodeficiency. Pharming expects to report Phase II results for leniolisib in CVID patients with immune dysregulation, with or without an identified genetic cause, in the fourth quarter of 2026.
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