Pharming Group (PHARM) 25th Annual Needham Virtual Healthcare Conference summary

Strategic transformation, vision, and growth priorities

• Transitioned from a single-asset to a two-commercial-asset company, both showing double-digit growth, supported by a high-value pipeline with two programs each having revenue potential above $1 billion.

• Aims to become a leading global rare disease company with a diverse portfolio and strong presence in major markets, leveraging efficient clinical development and commercial infrastructure.

• Focuses on high-value pipeline assets and sustained commercial growth, particularly in rare diseases.

• Enhanced capital allocation and disciplined cost management are driving value creation and profitability.

• Leadership changes and a focus on operational efficiency aim to support scalability and future pipeline expansion.

Commercial and financial performance

• Achieved 27% revenue growth in 2025 to $376M, driven equally by RUCONEST and Joenja, with a shift to operating profit and positive cash flow.

• 2026 revenue guidance is $405–$425 million (8–13% growth), with operating expenses of $330–$335 million, including $60M incremental R&D investments and $9M in G&A cost reductions.

• Financial discipline emphasized, with available cash and future cash flows expected to cover pipeline and pre-launch costs.

• RUCONEST remains a cornerstone treatment for severe HAE patients, maintaining strong cash generation and mid-single-digit growth expected in 2026.

• $9M in structural G&A cost reductions and a 20% reduction in G&A headcount implemented.

Product and pipeline updates

• RUCONEST, a recombinant C1 inhibitor, serves a specific subsegment of severe HAE patients, showing superior efficacy in indirect comparisons to newer therapies, with 97% of acute attacks resolved with one dose.

• Joenja (leniolisib) is approved for APDS in the US and Japan, with accelerating uptake, expansion into pediatric and international markets, and strong international launches.

• Two phase II trials for Joenja in higher prevalent PIDs and CVID are expected to read out in the second half of 2026, potentially expanding its addressable market.

• Napazimone (KL1333), acquired via Abliva, is in a pivotal phase II trial for primary mitochondrial disease, with positive interim analysis and full readout expected by end of 2027.

• Expanded Access Program for Joenja in other PIDs shows promising early results, with improvements in biomarkers and patient-reported outcomes.