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 (CVID): Phase II topline results (November 2026).
Upcoming catalysts
- November 2026Leniolisib (CVID) — Phase II topline results
We look forward to reporting topline results from our separate Phase II trial in CVID, expected in November, which will inform our plans for a potential registrational study in the broader CVID population.
Next further out: KL1333 — FALCON pivotal trial readout (H2 2027). 1 catalyst 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
- 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 - Sep 25, 2026accepted FDA accepted the lower-dose pediatric sNDA for Joenja with Priority Review and a PDUFA date of January 30, 2027.
The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2027.
Filing - Sep 22, 2026positive Positive topline Phase II single-arm data (n=13): mean 26.4% spleen volume reduction; well tolerated; accepted as ESID late-breaker.
Clinical results included improvements in lymphoproliferative disease, with a mean 26.4% spleen volume reduction (SVR) and reductions in the size of index lesions.
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
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.