Release Date: August 01, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Blueprint Medicines Corp (BPMC, Financial) reported a strong quarter with AYVAKIT achieving $114.1 million in net product revenue, reflecting a 185% year-over-year growth.
- The company raised its AYVAKIT product revenue guidance to $435 million to $450 million for 2024, indicating strong confidence in continued growth.
- Blueprint Medicines Corp (BPMC) has seen strong and steady growth in patients on AYVAKIT, driven by new patient starts and low discontinuation rates.
- The company has initiated a healthy volunteer study for its wild-type KIT inhibitor, Blue 808, which has the potential to impact a range of mast cell diseases.
- Blueprint Medicines Corp (BPMC) has a solid financial position with $868.5 million in cash on hand, providing financial flexibility to invest in innovation and growth.
Negative Points
- The company anticipates some seasonal dynamics that may impact the timing of patient starts, which could affect quarterly revenue growth.
- There is a potential revenue impact from German pricing negotiations, which are expected to be finalized at the end of the year.
- The company faces challenges in expanding the breadth and depth of the AYVAKIT prescriber base, particularly in community settings.
- Blueprint Medicines Corp (BPMC) has not yet fully captured the international market for AYVAKIT, with launches in additional countries expected in 2025.
- The company acknowledges the inherent challenges in setting guidance early in the launch into a brand-new market, which may lead to variability in revenue projections.
Q & A Highlights
Q: With regard to the ISM launch, now that you are a full year post-approval, how are you thinking about quarterly dynamics? Could you discuss any impact from the Part D redesign and how this might evolve? Also, for your wild-type KIT, should we expect healthy volunteer data this year or early next year?
A: We are pleased with yet another strong quarter in our launch, which has strengthened our conviction in the peak potential of AYVAKIT. We are learning to expect some seasonal dynamics, as patients with chronic rare diseases may be hesitant to try new treatments around vacations and holidays. Regarding the Part D redesign, we believe the proportion of free goods has stabilized at just under 20% and will remain relatively stable for the rest of the year. For the wild-type KIT, we expect the SAD/MAD data to be available early in 2025, which will be an important milestone for us.
Q: How should we think about the right eligibility proportion for AYVAKIT? What is your initial read on the type of patient starting therapy today at legacy practices with good AYVAKIT experience?
A: We see a trend towards providers broadening the lens on who is an appropriate patient for AYVAKIT, moving from the most symptomatic patients to those with one or two predominant symptoms significantly impacting their quality of life. This trend is reflected in our claims data and the degree of symptom burden, ER visits, and other measures of disease severity.
Q: Over the last few quarters, AYVAKIT has been growing significantly. Your guidance seems to assume a slowdown. Is there a dynamic beyond seasonal impacts driving this? Was there some sort of stocking impact in Q2?
A: There was no stocking impact. Our guidance reflects a range of variables, including patients on therapy, new patient starts, low discontinuation rates, seasonal impacts, and the proportion of free goods. We expect continued strong year-over-year growth, and our updated guidance represents more than doubling our revenue over last year.
Q: Can you comment on whether there have been any net pricing increases quarter over quarter? If so, could you break down revenue growth by price versus volumes?
A: There have been no net pricing increases quarter over quarter. The revenue growth is driven by volume rather than price increases.
Q: Can you contextualize the trends you are seeing for prescribers at academic centers versus community centers?
A: We have seen broad adoption across all specialties and settings, including academic and community hematology oncology, as well as allergists. The trend towards deepening is likely occurring more within centers of excellence, but we expect continued breadth and depth across all specialties and settings over time.
Q: How do you view business development with the growing cash position and ongoing acceleration of the AYVAKIT launch? Do you have an appetite for bolt-on deals to bring in earlier or late-stage clinical assets?
A: We consistently think about business development as a strategic lever to optimize our portfolio. Our focus remains on advancing our mast cell disorder franchise, including the continued execution of the AYVAKIT launch and advancing our clinical development programs. We are open to strategic opportunities that align with our priorities.
Q: Do you anticipate any challenges with enrolling patients for the HARBOR study with AYVAKIT on the market for the same indication? Will there be any patients enrolled who may be refractory to AYVAKIT?
A: We do not expect major challenges in recruiting patients for the HARBOR study. The study will open in the U.S. and internationally, allowing patients from other countries to access the trial. We are confident in our ability to execute the study.
Q: What are the real-world discontinuation rates of AYVAKIT? How much of the clinical development for wild-type KIT is baked into your financial guidance?
A: Real-world discontinuation rates are very low, and patients are staying on therapy in a highly compliant way. The development plan for wild-type KIT is factored into our guidance, and we prioritize allocating capital to high-value R&D opportunities that will drive long-term growth.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
