Pharming Group N.V. PHAR
Pharming Group N.V. (Nasdaq: PHAR) is an immunology/inflammation drug developer. Its lead program, Joenja (leniolisib), is in Phase 3 for Activated PI3K delta syndrome (APDS). Its next catalyst on record is leniolisib: Pharming expects to report Phase II topline results for leniolisib in CVID patients in November 2026. (November 2026).
Upcoming catalysts
- November 2026leniolisib — Pharming expects to report Phase II topline results for leniolisib in CVID patients in November 2026.
Pharming expects to report Phase II results for leniolisib in CVID patients with immune dysregulation, with or without an identified genetic cause, in November 2026.
Next further out: Joenja (leniolisib) — Joenja expected to become available to pediatric patients covered by the expanded U.K. indication in February 2027. (February 2027). 4 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| RUCONEST (recombinant C1 esterase inhibitor) | Acute hereditary angioedema attacks | protein/peptide | Approved |
| Joenja (leniolisib) | APDS | small molecule | Approved |
| KL1333 | mtDNA-driven primary mitochondrial disease (FALCON pivotal trial) | small molecule | Phase 3 |
| Leniolisib | CVID with immune dysregulation (Phase II) | small molecule | Phase 2 |
| Leniolisib | Genetically defined PIDs with immune dysregulation (CTLA4, SOCS1, NFKB1, FAS) (Phase II) | small molecule | Phase 2 |
Recent results and decisions
- Nov 6, 2026approval U.K. MHRA approves extension of Joenja marketing authorization to include children aged 4 to 11 years weighing at least 27 kg with APDS.
The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved an extension of the marketing authorization of Joenja®, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to include children aged 4 to 11 years weighing at least 27kg with activated PI3K delta syndr
Filing - Oct 14, 2026positive ESID presentation of Phase II data (n=13): mean spleen volume reduction 26.4% (n=11), lymph node index lesion reduction 33.7%, DLCO +8.0%; generally well tolerated.
Spleen volume: Mean reduction from baseline of 26.4% (n=11), with reductions observed in 10 of 11 evaluable participants.
Filing - Oct 14, 2026positive Pharming presented positive topline data from Phase II trial of leniolisib in genetically identifiable PIDs with immune dysregulation at ESID meeting.
On October 14, 2026, we presented positive topline data from our Phase II trial of leniolisib in genetically identifiable PIDs with immune dysregulation at the 22nd Biennial Meeting of the European Society for Immunodeficiencies (ESID).
Filing
8 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 immunology/inflammation companies
- Sanofi SNY
- Argenx SE ARGX
- Roivant Sciences Ltd. ROIV
- Immunovant, Inc. IMVT
- Celldex Therapeutics, Inc. CLDX
- Biohaven Ltd. BHVN
- BioCryst Pharmaceuticals, Inc. BCRX
- Zenas BioPharma ZBIO
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.