Pharming Group N.V., a biopharmaceutical company specializing in rare diseases, recently released their Q1 2024 earnings transcript. While the headline numbers were positive, a deeper dive into the details of their Joenja launch reveals a potentially explosive trend that appears to have slipped under the radar of most analysts.

The company reported $9.6 million in Joenja sales for the quarter, a respectable 21% increase over Q4 2023. The growth, according to management, was driven by the continued identification and conversion of APDS patients to paid therapy. This explanation, while accurate, might be obscuring a far more intriguing dynamic.

Pharming disclosed that 83 APDS patients were receiving paid therapy at the end of Q1. This represents a net increase of just two patients from the 81 reported at the end of Q4 2023. The company did note that 15 newly diagnosed patients were in the process of being converted to paid therapy. However, even accounting for these patients, the simple math suggests a significant increase in revenue per patient.

Breaking Down the Numbers

This translates to an 18.8% increase in revenue per patient quarter-over-quarter. What could be driving such a substantial jump?

One possible explanation is a shift in patient mix towards those with more severe APDS. More severe patients might require higher doses of Joenja, leading to increased revenue per patient. However, management's commentary on the severity of newly diagnosed patients doesn't necessarily support this hypothesis.

A second, and perhaps more compelling, explanation lies in the burgeoning Named Patient Program (NPP) for Joenja outside the US. Pharming reported $1.1 million in sales from NPPs during the quarter, a significant jump from the $0.3 million recorded in Q4 2023. While management declined to provide specific patient numbers or pricing details, they did indicate that NPP prices were generally in line with US prices.

This suggests a possible scenario where the NPP is driving a hidden surge in Joenja sales, contributing to the substantial increase in revenue per patient. Could it be that analysts, focused on the US launch, are overlooking the rapid uptake of Joenja through NPPs globally?

The Potential of the Named Patient Program

If this hypothesis holds true, it has significant implications for Pharming's future growth trajectory. A successful and robust NPP could generate significant revenue streams even before formal regulatory approvals and reimbursement processes are in place in those international markets. Furthermore, it would validate the global demand for Joenja and bode well for its eventual commercial launch in these territories.

Revenue Growth: RUCONEST vs. Joenja

The following chart shows the quarterly revenue growth of Pharming's two main products, RUCONEST and Joenja. Data is from Q1 2023 to Q1 2024.

While only time will tell if this sales surge is a sustainable trend, the potential for Joenja to outperform expectations is evident. Analysts and investors would be wise to delve deeper into the NPP dynamics and not simply rely on the headline patient numbers to assess the true commercial potential of this innovative drug.

"Fun Fact: Pharming's RUCONEST, derived from the milk of transgenic rabbits, was a groundbreaking innovation in protein replacement therapy for HAE. This unique approach to drug development highlights the company's commitment to pushing the boundaries of science in their pursuit of treatments for rare diseases."