Release Date: October 29, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Harmony Biosciences Holdings Inc (HRMY, Financial) reported strong revenue growth for WAKIX, with net sales reaching $186 million in Q3 2024.
- The company surpassed $2 billion in cumulative net revenue for WAKIX in less than five years on the market.
- Harmony Biosciences Holdings Inc (HRMY) has a robust late-stage pipeline with plans to launch one or more new products or indications each year over the next five years.
- The company is advancing its pitolisant next-gen programs, with pitolisant GR and HD formulations expected to expand the patient base and address unmet needs.
- Harmony Biosciences Holdings Inc (HRMY) maintains a strong financial position with approximately $505 million in cash, cash equivalents, and investments as of September 30, 2024.
Negative Points
- Operating expenses increased by 29% compared to the same quarter in 2023, driven by investments in the expanding late-stage pipeline.
- The company faces competition in the rare epilepsy space, with other investigational products being developed.
- There is uncertainty regarding the regulatory approval process for new indications and products, such as the IH sNDA submission.
- The timeline for some pipeline products, like EPX-100, extends to 2026 for topline data, indicating a long wait for potential market entry.
- The company is navigating a complex payer landscape, especially with the anticipated entry of more generic oxybate products in the market.
Q & A Highlights
Q: Can you explain the strategy for managing the coexistence of WAKIX, Pitolisant GR, and HD when they are all on the market?
A: Jeffrey Dierks, Chief Commercial Officer, explained that WAKIX will remain the foundational business with a billion-dollar-plus opportunity. Pitolisant GR, expected in 2026, will expand the patient base by activating previous WAKIX patients who discontinued due to side effects or lack of benefit, and attracting new patients. Pitolisant HD, anticipated in 2028, is expected to convert most WAKIX patients due to its enhanced efficacy and unique fatigue indication.
Q: Could you provide more details on the orexin-2 receptor agonist program and when we might see additional data?
A: Kumar Budur, Chief Scientific Officer, mentioned that the orexin-2 receptor agonist has shown high potency and selectivity in preclinical studies. The company plans to present additional data at scientific meetings next year, working with their partner Bioprojet.
Q: What differentiates EPX-100 from other treatments in the developmental epileptic encephalopathies (DEE) space?
A: Kumar Budur highlighted that EPX-100 has a well-established safety profile, having been on the market for 20 years without significant safety signals. It offers a simple BID dosing regimen, which is beneficial for DEE patients. The drug is further along in clinical development compared to competitors, with ongoing Phase 3 trials for Dravet syndrome and Lennox-Gastaut syndrome.
Q: How is the payer landscape expected to evolve in 2025, especially with more generics entering the market?
A: Jeffrey Dierks stated that the payer landscape for 2025 is expected to remain similar to the current situation, with WAKIX maintaining a favorable position due to its unique non-scheduled status. No new generic oxybates are anticipated in 2025, and WAKIX is not required to be stepped through oxybates, ensuring continued patient access.
Q: Can you clarify the dosing strategy for Pitolisant HD and its potential benefits?
A: Jeffrey Dayno clarified that Pitolisant HD aims to dose up to two times the maximum labeled dose of WAKIX, with safety margins established up to five times the current dose. The optimized formulation is expected to provide higher exposure and reduced inter-dose variability, enhancing clinical impact.
Q: Why is the RECONNECT study including non-complete methylation patients, and what are the implications for FDA approval?
A: Kumar Budur explained that while the primary endpoint focuses on complete methylation patients, including non-complete methylation patients allows for potential broader label discussions with the FDA if supportive data is observed.
Q: How are you planning to measure fatigue in the Pitolisant HD study for narcolepsy, and how prevalent is fatigue in this population?
A: Kumar Budur noted that fatigue affects about 60-70% of narcolepsy patients. The company is developing a specific instrument to measure fatigue in narcolepsy patients, which will be discussed with regulatory agencies to ensure its appropriateness.
Q: What is the current percentage of patients on both oxybate and WAKIX, and how might this change with new treatments?
A: Jeffrey Dierks stated that the percentage of patients on both treatments has remained in the low double digits. The reimbursement landscape has been stable, and while new treatments may enter the market, rare orphan categories like narcolepsy tend to be less managed due to the limited patient population.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
