Pharming Group

PHAR

時価総額 $6.62億

First clinical data for leniolisib in a larger group of PID participants beyond APDS, supporting the potential of targeted PI3Kδ inhibition to address immune dysregulation Leniolisib was well-tolerated, with benefits observed across multiple measures of immune dysregulation, including improvement in lymphoproliferation and positive trends in lung and liver disease markers All participants who completed the study elected to continue receiving treatment in a long-term extension study Leiden, the Netherlands, October 14, 2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today presented positive topline data from its Phase II trial of leniolisib in genetically identifiable primary immunodeficiencies (PIDs) with immune dysregulation linked to altered PI3Kδ signaling. The data, accepted as a late-breaking abstract, were presented as a poster at the 22nd Biennial Meeting of the European Society for Immunodeficiencies (ESID), taking place October 14-17, 2026, in Maastricht, the Netherlands.

RUCONEST is Pharming Group's main revenue source today. This asset has already shown a sequential sales rebound that makes me more optimistic. However, RUCONEST still has oral competition that could make stabilizing that revenue bleed more challenging. Still, my bull case assumes RUCONEST's issues are manageable. If so, that could buy time for Joenja to grow and improve the business mix.

Leiden, the Netherlands, September 29, 2026 : Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the Board of Directors and Fabrice Chouraqui have mutually agreed that Mr. Chouraqui will step down as Chief Executive Officer and Executive Director, with immediate effect.

If approved, J oenja would be available to eligible patients aged 4 years and older with APDS who weigh 13 kg or more Decision follows recent approval of Joenja for children aged 4 to 11 years with APDS weighing at least 27 kg PDUFA target action date of January 30, 2027 Leiden, the Netherlands, September 25,  2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) seeking approval for lower doses of Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, for children aged 4 years and older who weigh between 13 kg and 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2027.

Leniolisib was generally well-tolerated, with clinical improvements across multiple measures of immune dysregulation, including reductions in lymphoproliferation Separately, Pharming expects to report topline results from a Phase II trial of leniolisib in CVID in Q4 2026 Leiden, the Netherlands, September 22, 2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that a late-breaking abstract highlighting positive topline data from its Phase II trial with leniolisib in genetically identifiable primary immunodeficiencies (PIDs) with immune dysregulation linked to PI3Kd signaling has been accepted at the 22nd Biennial Meeting of the European Society for Immunodeficiencies (ESID), 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.

FDA approval expands Joenja indication to include children with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) ages 4 to 11 who weigh at least 27kg Newly approved Joenja doses are expected to be available in October Leiden, the Netherlands, September 11, 2026: Pharming (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global biotechnology company focused on rare immune and genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for 40 mg and 50 mg twice-daily dosing of Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. With this approval, Joenja becomes the first FDA-approved treatment for children aged 4 to 11 years with APDS in the U.S. The newly approved Joenja doses are expected to be available to eligible pediatric patients in the U.S. in October through Pharming's established specialty distribution network and patient-support infrastructure.

Pharming Group to participate in investor conferences Sep. 14-15. H.C. Wainwright Growth Conference and Morgan Stanley Healthcare Conference in New York.

Leiden, the Netherlands, August 13, 2026: Pharming Group (Euronext Amsterdam: PHARM/Nasdaq: PHAR), today announced the commercial launch of Joenja (leniolisib) in Japan for adult and pediatric patients with activated PI3K delta syndrome (APDS) aged 4 years and older, following its inclusion on the Japan National Health Insurance (NHI) drug price list. With this launch, Japan is now the first market where Joenja is commercially available for children aged 4 to 11.

Pharming Group told investors to expect up to $425 million in 2026 revenue. The Company now expects as little as $375 million -- and the stock lost 22% in a single session.

LOS ANGELES--(BUSINESS WIRE)--Glancy Prongay Wolke & Rotter LLP, a leading national shareholder rights law firm, today announced that it has commenced an investigation on behalf of Pharming Group N.V. (“Pharming” or the “Company”) (NASDAQ: PHAR) investors concerning the Company's possible violations of the federal securities laws. IF YOU ARE AN INVESTOR WHO LOST MONEY ON PHARMING GROUP N.V. (PHAR), CLICK HERE TO INQUIRE ABOUT POTENTIALLY PURSUING CLAIMS TO RECOVER YOUR LOSS. What Happened?.