Harmony Biosciences Holdings, Inc. Q2 FY2025 earnings call

• Harmony is a profitable, self-funding biotech with an innovative late-stage pipeline. WAKIX had double-digit revenue growth, with $200.5M in Q2 net revenue, extending a 4-year profitability streak.
• Pipeline focus includes sleep/wake, neurobehavioral (ZYN002 Phase III RECONNECT study in Fragile X completed enrollment, on track for top-line data this quarter), and rare epilepsies. ZYN002 is a synthetic cannabidiol for Fragile X syndrome.
• Next-gen pitolisant formulations: pitolisant HD and GR are in development. Pitolisant HD Phase III trials in narcolepsy and idiopathic hypersomnia set to start in Q4 2025.
• Collaboration with Circ Biosciences for cell therapy in refractory epilepsy and treatment-resistant narcolepsy, aligning with the pipeline.

In the second quarter, Harmony Biosciences reported net revenue of $200.5 million, representing a 16% year-over-year increase. WAKIX, the key product, contributed significantly, with a 16% Y/Y growth and an addition of 400 average patients during the quarter. WAKIX remains the first and only non-scheduled treatment for narcolepsy, driving this revenue performance.

• Reiterated revenue guidance of $820 million to $860 million for 2025. Confident in WAKIX achieving $1 billion plus in annual revenue in narcolepsy alone.
• Expect continued Q/Q net revenue growth. Anticipate $29M in milestones for ZYN002 Phase III trial in Fragile X, $15M in Q3, and $10M for positive top-line data from the trial. Also expect $4M in orexin-2 program milestones.

Forward-looking statements are subject to risks and uncertainties. Actual results may differ materially from expectations. Consult risk factors in SEC filings for additional details.

Q: Can you provide details on the RECONNECT study's top-line data timing and confidence?

A: On track for Q3, confident in replicating positive findings from the Phase II/III CONNECT study in patients with complete methylation.

Q: What about the data disclosure and filing strategy for Fragile X syndrome?

A: Standard top-line data readout including demographics, safety, and efficacy. Potential priority review by FDA, aiming for approval by end of next year.

Q: What are the growth levers for WAKIX?

A: Focus on average referrals per day, conversion to dispensing events, and patient retention through strong service to HCPs, office staff, and patients.

Q: Details on placebo response and adherence in the RECONNECT study?

A: Rigorous inclusion criteria, high adherence seen in CONNECT study. Study is well-powered to detect statistically significant differences, with confidence in the study design to mitigate placebo response.