Stock Watch: Advanced Therapies Deliver Fourth-Quarter Flop

BioMarin Pharmaceutical Inc.’s fourth-quarter and full-year 2023 financial results announcement prompted a modest outperformance by its stock compared with the NASDAQ Biotech Index’s (NBI’s) 1% increase the day after BioMarin’s post-market announcement. Analysts and investors were focused on the majority of BioMarin’s commercial operations and sales of its advanced gene therapy product – Roctavian (valoctocogene roxaparvovec) for hemophilia A – seemed largely forgotten despite growing by nearly 240% on the third quarter of 2023. This was because the percentage of BioMarin’s fourth-quarter total sales contributed by Roctavian was so low that it looked more like a highly significant p-value.

Following Roctavian’s approval at the end of June 2023, BioMarin’s fourth-quarter commentary on it was limited to the “build-out of global access channels.” BioMarin’s investors are probably getting used to Roctavian disappointments as full-year 2023 sales guidance had been lowered from between $50m and $150m before BioMarin’s third-quarter 2023 results, to less than $10m after it. Then, after full-year 2023 Roctavian sales of $4m missed analysts’ consensus estimates by a third, unspecified Roctavian sales were included in BioMarin’s full-year 2024 financial guidance. This likely helped its sales expectations for 2024 to miss analysts’ estimates by nearly 3%. Roctavian’s commercial challenges are not unique and the worrying issue of how gene therapies are monetized seems to be resonating through big pharma and smaller biotech companies alike