Summary: Harmony Biosciences has reported cumulative revenues of over $2 billion for Wakix since its launch for adult narcolepsy in 2019. At its recent Investor Day, the company highlighted new data showing pitolisant’s effectiveness in treating idiopathic hypersomnia (IH), with plans to submit a supplemental new drug application (sNDA) for this expanded indication by the end of Q4. Harmony also presented updates on its evolving CNS pipeline, including future developments for high-dose and gastro-resistant formulations of pitolisant.
Key Takeaways:
- Revenue Milestone for Wakix: Since its 2019 launch, Wakix has reached over $2 billion in cumulative revenue, underscoring its broad adoption in narcolepsy treatment.
- Expansion into IH Treatment: New data supports Wakix’s potential for treating idiopathic hypersomnia, with Harmony planning to submit an sNDA for this indication in Q4 2024.
- Pipeline Growth: Harmony is advancing multiple pitolisant formulations, including high-dose and gastro-resistant versions, to address additional needs within CNS and sleep-related disorders.
Harmony Biosciences Holdings Inc reported a record $186 million in net revenue for the quarter ending on Sept 30, 2024, surpassing $2 billion in cumulative net revenues since the launch of Wakix in adult narcolepsy in November of 2019.
In addition, the company recently held an Investor Day on Oct 1, outlining its progress toward becoming a self-sustaining biotech company with a growing late-stage pipeline.
“Going into Q4, Harmony has great momentum. During our Investor Day, we shared new data in support of our confidence and excitement about the company’s growth trajectory as we advance our robust, catalyst-rich, late-stage pipeline and expand into additional rare [central nervous system] therapeutic areas. We are building on our success in sleep/wake with a strategy focused on continuous innovation, patient impact, and long-term value creation for our shareholders,” says Jeffrey M. Dayno, MD, president and chief executive officer of Harmony Biosciences, in a news release.
Key Sleep/Wake Portfolio Highlights:
Wakix:
- Net Sales for the quarter were $186 million. With these quarterly sales, Wakix surpassed $2 billion in cumulative net revenue in less than five years on the market.
- The average number of patients on Wakix increased by approximately 250 patients sequentially to approximately 6,800 for the quarter ended Sept 30, 2024.
Pitolisant in idiopathic hypersomnia:
- New data from the long-term extension study demonstrate robust and sustained efficacy of pitolisant in patients with idiopathic hypersomnia.
- Mean improvement in Epworth Sleepiness Scale (ESS) was ~9 points from baseline out beyond one year, with the majority of patients in the normal range as measured by the ESS.
- Sustained efficacy was also observed on the Idiopathic Hypersomnia Severity Scale and Sleep Inertia Questionnaire beyond one year.
- Data supports astrong benefit/risk proposition, and Harmony is on track to submit a supplemental new drug application for pitolisant in idiopathic hypersomnia in Q4 2024.
Pitolisant-HD (high dose) program:
- Pitolisant-HD is an enhanced formulation of pitolisant designed with the following attributes:
- A higher dose with an optimized pharmacokinetic profile to drive greater efficacy in excessive daytime sleepiness and cataplexy
- Targeting a unique indication for fatigue in narcolepsy
- A gastro-resistant coating with no need for a titration dose
- Preliminary safety data up to five times the current highest labeled dose of Wakix are consistent with the established safety profile of Wakix and establish safety margins for the pitolisant-HD development program.
- Pitolisant-HD is on track for Prescription Drug User Fee Act (PDUFA) in 2028 with the goal to extend the pitolisant franchise to mid-2040s.
- Provisional patents have been filed until 2044 with the opportunity to grow the pitolisant franchise by pursuing additional indications.
Pitolisant-GR (gastro-resistant) program:
- Pitolisant-GR is a gastro-resistant formulation of pitolisant designed to minimize GI tolerability issues in patients with narcolepsy; approximately 90% of patients with narcolepsy experience GI symptoms partly related to the underlying disease mechanism.
- Harmony is on track to initiate a pivotal bioequivalence study and dosing optimization study (to remove the titration dose) in Q1 2025.
- PDUFA is on track for 2026 with intellectual property to the mid-2040s.
Orexin-2 agonist program:
- BP1.15205 (formerly TPM-1116) potential to be best-in-class orexin-2 receptor agonist.
- Based on a novel chemical scaffold.
- Demonstrated greater potency compared to all publicly disclosed data on orexin-2 agonists; allows for dosing flexibility to target all central disorders of hypersomnolence.
- The potency was consistent across species along with an excellent selectivity of greater than 600x which translates to over 140-fold margin over orexin-1 receptors at the anticipated maximum human dose.
- In addition, BP1.15205 demonstrated over 1000-fold selectively over 150 other targets of interest.
- Preclinical PK data consistent with once-a-day dosing.
Third Quarter 2024 Financial Results:
Net product revenues for the quarter ended Sept 30, 2024, were $186 million, compared to $160.3 million for the same period in 2023. The 16% growth versus the same period in 2023 is primarily attributed to strong commercial sales of Wakix driven by continued organic demand tapping into a large market opportunity (approximately 80,000 patients diagnosed with narcolepsy in the US) and the broad clinical utility of Wakix across the approximately 9,000 healthcare professionals that Harmony calls on (about 5,000 of whom do not participate in an oxybate Risk Evaluation and Mitigation Strategy program).
Photo caption: Wakix
File Photo/Harmony Biosciences
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