Pharming Group N.V.
PHAR evidence brief
What we can confirm
What it means
What to watch
Prepared research question
What matters most for PHAR—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · PHAR
Pharming Group N.V. is a global biopharmaceutical company focused on developing and commercializing therapies for rare diseases, with two marketed products (RUCONEST® for HAE and Joenja® for APDS) and a pipeline including leniolisib and napazimone (KL1333) for mitochondrial disease.
Key risk: Pipeline development risk
BioSniper Score
Based on 2 of 6 signals
Limited data coverage
Market Cap
$828.1M
Cash Runway
—
Lead Asset
—
Next Catalyst
—
Financial data as of Dec 31, 2025.
Pharming Group N.V. is a global biopharmaceutical company focused on developing and commercializing therapies for rare diseases, with two marketed products (RUCONEST® for HAE and Joenja® for APDS) and a pipeline including leniolisib and napazimone (KL1333) for mitochondrial disease.
- Total revenue of $376.1M and net income of $2.5M for FY 2025 [sec, as of 2025-12-31].
- Cash, restricted cash, and marketable securities of $159.5M as of mid-2025 [sec, filed 2026-07-30].
- 2026 revenue guidance of $405M–$425M (8%–13% growth) and operating expenses of $330M–$335M [sec, filed 2026-03-12].
- Acquired Abliva AB in 2025, adding napazimone (KL1333) for mitochondrial disease, now in pivotal FALCON study [sec, filed 2026-04-02].
- Joenja® (leniolisib) for APDS is approved in the U.S. and expanding ex-U.S., with a pediatric label expansion underway [sec, filed 2026-04-02].
What works
- Two approved commercial products (RUCONEST® and Joenja®) generating growing revenue, with 2026 guidance of $405M–$425M [sec, filed 2026-03-12].
- Pipeline includes leniolisib in Phase II for primary immunodeficiencies and napazimone (KL1333) in a pivotal study for mitochondrial disease, targeting large underserved populations [sec, filed 2026-02-03].
- Improved net loss trend: net loss of $5.2M in Q1 2026 vs. $14.9M in Q1 2025 [sec, filed 2026-05-07].
- Strong cash position of $159.5M as of mid-2025 supports ongoing operations and pipeline advancement [sec, filed 2026-07-30].
What to weigh
- Net loss of $5.2M in Q1 2026, though improved year-over-year, indicates ongoing unprofitability [sec, filed 2026-05-07].
- Cash used in operations of $7.7M in H1 2025, a reversal from $12.0M generated in H1 2024 [sec, filed 2026-07-30].
- RUCONEST® faces a more competitive HAE market, requiring reinforcement of its value proposition [sec, filed 2026-04-02].
- Pipeline programs are in early-to-mid-stage development, with no guarantee of regulatory approval or commercial success [sec, filed 2026-04-02].
From the filings
Quoted directly from source documents.
2026 revenue guidance of $405M–$425M
“Total revenues between US$405 million and US$425 million (8% to 13% growth)”
SEC filings · March 12, 2026
Net loss of $5.2M in Q1 2026
“In the first quarter of 2026, a net loss of US$5.2 million was realized”
SEC filings · May 7, 2026
Acquisition of Abliva AB added napazimone (KL1333) for mitochondrial disease
“This acquisition expanded our presence into mitochondrial disease, added a pivotal-stage program with significant long-term value potential”
SEC filings · April 2, 2026
Cash and equivalents of $159.5M as of mid-2025
“Cash and cash equivalents, restricted cash and marketable securities decreased by US$21.6 million to US$159.5 million”
SEC filings · July 30, 2026
Risks & what to watch (4)›
Key risks
- Pipeline development risk — Leniolisib and napazimone are in early-to-mid-stage trials; failure could impair growth prospects [sec, filed 2026-04-02].
- Competitive pressure on RUCONEST® — RUCONEST® faces a more competitive HAE market, requiring reinforcement of its value proposition [sec, filed 2026-04-02].
- Operating cash flow volatility — Cash used in operations of $7.7M in H1 2025 vs. $12.0M generated in H1 2024 [sec, filed 2026-07-30].
- Regulatory and commercial execution — Expanding Joenja® ex-U.S. and achieving pediatric label approval depend on regulatory success [sec, filed 2026-04-02].
What to watch
- Enrollment completion and readout of the FALCON pivotal study for napazimone (KL1333) in mitochondrial disease, expected late 2027 [sec, filed 2026-03-12].
- Progress of leniolisib Phase II trials for primary immunodeficiencies, including CVID [sec, filed 2026-01-08].
- Ex-U.S. approvals and commercial launches for Joenja® in APDS, including pediatric label expansion [sec, filed 2026-04-02].
- Quarterly revenue and operating expense trends relative to 2026 guidance of $405M–$425M revenue and $330M–$335M opex [sec, filed 2026-03-12].
Sources: SEC filings · Generated August 14, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 2 of 6 signals · derived from public data
No clinical pipeline tracked — this score reflects financial & catalyst signals only.
Profitable (positive operating cash flow)
D/E 0.80
No tracked pipeline phase
No tracked pipeline candidates
No upcoming catalysts tracked
No insider transactions in the last 180 days
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
8
Total value held
$568K
| Holder | Shares | Value |
|---|---|---|
| morgan stanley | 11,489 | $191K |
| smartharvest portfolios, llc | 10,797 | $180K |
| citadel advisors llc | 10,189 | $170K |
| eversource wealth advisors, llc | 1,997 | $17K |
| bnp paribas financial markets | 400 | $7K |
| osaic holdings, inc. | 128 | $2K |
| stone house investment management, llc | 79 | $1K |
| cornerstone financial management llc | 32 | $532 |
Short Interest
as of May 22, 2026- % of float
- 0.0%
- Days to cover
- 0.7
- Shares short
- 8,217
- Daily short volume (Jul 17, 2026)
- 3.6%
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
PHAR short interestFrequently asked questions
What is the AI investment thesis for Pharming Group N.V.?
Pharming Group N.V. is a global biopharmaceutical company focused on developing and commercializing therapies for rare diseases, with two marketed products (RUCONEST® for HAE and Joenja® for APDS) and a pipeline including leniolisib and napazimone (KL1333) for mitochondrial disease.
Who is the CEO of Pharming Group N.V.?
Mr. Fabrice Chouraqui M.B.A., Ph.D., Pharm.D. is the chief executive officer of Pharming Group N.V. (PHAR).
Where is Pharming Group N.V. headquartered?
Pharming Group N.V. (PHAR) is headquartered in Leiden, Netherlands.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.