Pharming Group N.V. Form 6-K Summary
Filing date: March 24, 2023. Reporting period: March 2023. The filing reports the U.S. FDA approval of Joenja® (leniolisib), Pharming’s second commercial product, for activated phosphoinositide 3-kinase delta syndrome (APDS).
Business Context
Pharming is a Leiden, Netherlands-based global biopharmaceutical company focused on rare diseases. Joenja is an oral, selective PI3Kδ inhibitor approved for adults and pediatric patients aged 12 years and older with APDS, a rare progressive primary immunodeficiency estimated to affect approximately 1 to 2 people per million.
Key Financial and Operating Metrics
| Metric | Reported information |
|---|---|
| Revenue, profit and margins | The filing text does not provide current-period or comparative revenue, profit, earnings-per-share or margin figures. |
| Cash flow and liquidity | The filing text does not provide current cash, operating cash flow, liquidity or working-capital balances. It references potential risks concerning cash deposited with SVB US and SVB UK. |
| Debt | The filing text does not provide debt or leverage figures. |
| Clinical evidence | A Phase II/III randomized trial included 31 patients aged 12 years and older. A long-term open-label extension included 38 patients, with median Joenja exposure of two years. |
| Clinical efficacy | At 70 mg twice daily over 12 weeks, Joenja achieved statistically significant co-primary endpoint results: adjusted mean difference in lymph node size of -0.25, with 95% CI of -0.38 to -0.12 and P=0.0006; and adjusted mean difference in naïve B-cell percentage of 37.30, with 95% CI of 24.06 to 50.54 and P=0.0002. |
| Common adverse reactions | Headache, sinusitis and atopic dermatitis, each reported at an incidence above 10% in clinical trials. |
Material Developments Versus the Prior Comparable Period
- The FDA approved Joenja as the first and only U.S.-approved treatment specifically indicated for APDS.
- Pharming expected U.S. product availability and shipment within one to two weeks of approval.
- The approval represents the addition of Pharming’s second commercial product and expands the company’s rare-disease portfolio beyond its existing products.
- The European Medicines Agency’s review of the marketing authorization application remained ongoing, with a CHMP opinion expected in the second half of 2023.
Outlook, Commitments and Risks
- Pharming plans to pursue additional regulatory approvals in the United Kingdom, Canada, Australia and Japan.
- Leniolisib is being evaluated in a Phase III trial for children aged 4 to 11, with a further trial planned for children aged 1 to 6.
- Under the 2019 exclusive license agreement with Novartis, Pharming must make approval and first-commercial-sale milestone payments totaling $10.5 million to Novartis and another party.
- Additional sales-based milestone payments to Novartis may total up to $190 million. Pharming also agreed to pay tiered royalties ranging from low double-digit to high-teen double-digit percentages of net leniolisib sales.
- The FDA granted Pharming a priority review voucher for the rare pediatric disease approval. Novartis has the right to purchase the voucher for a small minority share of its value.
- Safety considerations include potential fetal harm, reduced effectiveness of live attenuated vaccines, non-recommended use in moderate to severe hepatic impairment, and no recommended dosage for patients weighing less than 45 kg.
- Seven patients, or 33% of Joenja-treated patients in the cited trial, developed an absolute neutrophil count between 500 and 1,500 cells per microliter. No patient developed an ANC below 500, and no infection associated with neutropenia was reported.
- Forward-looking statements are subject to clinical, regulatory, commercial, technical and financing risks. The filing also references uncertainty regarding the recovery of cash deposited with SVB US and SVB UK.
Important Facts for Investors to Verify
- Actual timing and commercial performance of the U.S. Joenja launch, including patient identification, reimbursement, pricing and net sales.
- The amount and timing of milestone, royalty and other payments under the Novartis license agreement.
- The outcome and timing of the EMA review and any subsequent international regulatory submissions.
- Results, enrollment and safety findings from pediatric leniolisib trials.
- Pharming’s cash balances, liquidity, exposure to SVB-related deposits and ability to fund commercialization and clinical development.
- Long-term safety, durability of efficacy and post-approval regulatory requirements for Joenja.
- The conference-call date, which is inconsistent within the release: one section states March 24, while the detailed dial-in information states March 27, 2023.