Release Date: August 05, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Biomarin Pharmaceutical Inc (BMRN, Financial) reported a 20% year-over-year growth in total revenues, reaching a record $712 million for Q2 2024.
- VOXZOGO showed strong demand and patient uptake, with nearly 900 children beginning treatment in the first half of the year, marking the highest in its history.
- The company announced a 78% growth in non-GAAP earnings per share, demonstrating significant profitability improvements.
- Biomarin Pharmaceutical Inc (BMRN) received approval for Brineura, expanding access to children under the age of three years with CLN-2 disease.
- The company has made substantial progress in its R&D pipeline, including advancing development programs for multiple new indications such as idiopathic short stature, Turner syndrome, and Noonan syndrome.
Negative Points
- Despite progress, the company still faces challenges in achieving full market penetration for VOXZOGO, particularly in the U.S. and other key markets.
- ROCTAVIAN's profitability is not expected until the end of 2025, and the company has had to reduce its annual direct expenses for the product to approximately $60 million.
- There are ongoing challenges in securing reimbursement and providing access to ROCTAVIAN in markets like Germany and Italy.
- The company discontinued the development of BMN 293, a gene therapy for hypertrophic cardiomyopathy, due to commercial landscape and resource allocation concerns.
- Biomarin Pharmaceutical Inc (BMRN) faces competitive pressures in the achondroplasia market, with potential new entrants and alternative treatments on the horizon.
Q & A Highlights
Q: Can you talk a bit more about the changes aside from eliminating a bunch of territories or changing the strategy, our investment in the regions where you're going to stay U.S., Germany, and Italy?
A: Our approach allows us to focus geographically on the U.S., Germany, and Italy, and also to focus our efforts within those geographies. We are stopping our lifecycle development activities to focus on the main indication and reducing our manufacturing expenses associated with ROCTAVIAN. This allows us to operate within a $60 million envelope in 2025 and underpins our confidence in achieving profitability for ROCTAVIAN.
Q: Could you speak to the metrics that are giving you confidence in ROCTAVIAN's breakeven situation by 2025?
A: We are encouraged by the progress in the U.S., with three patients treated in the quarter and a fourfold increase in operationally ready sites. In Italy, we have seen rapid progress since receiving pricing and reimbursement in January. In Germany, we have finalized an agreement with one of the seven insurers, covering approximately 25% of patient lives. These metrics, along with a revised $60 million cost envelope, give us confidence in achieving profitability.
Q: How are you thinking about the evolution of the competitive landscape in achondroplasia? Would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia?
A: We feel very confident about our competitive profile in achondroplasia and subsequent indications. We are focusing on treating the youngest infants for optimal outcomes. Regarding combination with growth hormone, we believe the CMP franchise can achieve superior efficacy and convenience without needing a combination approach.
Q: How are you thinking about the evolution of the competitive landscape in achondroplasia? Would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia?
A: We feel very confident about our competitive profile in achondroplasia and subsequent indications. We are focusing on treating the youngest infants for optimal outcomes. Regarding combination with growth hormone, we believe the CMP franchise can achieve superior efficacy and convenience without needing a combination approach.
Q: How are you thinking about the evolution of the competitive landscape in achondroplasia? Would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia?
A: We feel very confident about our competitive profile in achondroplasia and subsequent indications. We are focusing on treating the youngest infants for optimal outcomes. Regarding combination with growth hormone, we believe the CMP franchise can achieve superior efficacy and convenience without needing a combination approach.
Q: How are you thinking about the evolution of the competitive landscape in achondroplasia? Would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia?
A: We feel very confident about our competitive profile in achondroplasia and subsequent indications. We are focusing on treating the youngest infants for optimal outcomes. Regarding combination with growth hormone, we believe the CMP franchise can achieve superior efficacy and convenience without needing a combination approach.
Q: How are you thinking about the evolution of the competitive landscape in achondroplasia? Would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia?
A: We feel very confident about our competitive profile in achondroplasia and subsequent indications. We are focusing on treating the youngest infants for optimal outcomes. Regarding combination with growth hormone, we believe the CMP franchise can achieve superior efficacy and convenience without needing a combination approach.
Q: How are you thinking about the evolution of the competitive landscape in achondroplasia? Would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia?
A: We feel very confident about our competitive profile in achondroplasia and subsequent indications. We are focusing on treating the youngest infants for optimal outcomes. Regarding combination with growth hormone, we believe the CMP franchise can achieve superior efficacy and convenience without needing a combination approach.
Q: How are you thinking about the evolution of the competitive landscape in achondroplasia? Would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia?
A: We feel very confident about our competitive profile in achondroplasia and subsequent indications. We are focusing on treating the youngest infants for optimal outcomes. Regarding combination with growth hormone, we believe the CMP franchise can achieve superior efficacy and convenience without needing a combination approach.
Q: How are you thinking about the evolution of the competitive landscape in achondroplasia? Would you ever consider evaluating VOXZOGO in combination with growth hormone in achondroplasia?
A: We feel very confident about our competitive profile in achondroplasia and subsequent indications. We are focusing on treating the youngest infants for optimal outcomes. Regarding combination with growth hormone, we believe the CMP franchise can achieve superior efficacy and convenience without needing a combination approach.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
