Summary: Harmony Biosciences reported strong second-quarter results and advancements in its pitolisant high-dose (HD) development program, aiming for a PDUFA date in 2028. The company highlighted revenue growth driven by Wakix and continued progress across its sleep/wake disorder portfolio, including new product launches and ongoing clinical trials. Harmony is also focused on developing a diverse range of treatments for rare central nervous system conditions.
Key Takeaways:
- Pitolisant High-Dose Development: Harmony Biosciences is advancing its pitolisant-HD program with a targeted PDUFA date in 2028, which aims to extend the pitolisant franchise and offer a differentiated product profile.
- Strong Financial Performance: The company reported a 29% increase in net revenue for the second quarter of 2024, driven by strong sales of Wakix, and projects full-year revenue between $700 million and $720 million.
- Pipeline and Product Expansion: Harmony is progressing its late-stage pipeline, with new drug applications and clinical trials for various sleep/wake and rare central nervous system disorders, aiming for at least one new product or indication launch annually over the next five years.
Harmony Biosciences Holdings reported second-quarter results, announcing that its next-generation pitolisant-HD development program is advancing and on track toward an expected PDUFA date in 2028.
“We made substantial progress and continue to advance our pitolisant high-dose development program, targeting a PDUFA (Prescription Drug User Fee Act) date in 2028, giving us the opportunity to extend the pitolisant franchise beyond 2040,” says Jeffrey M. Dayno, MD, president and chief executive officer of Harmony, in a release.
Dayno adds in a release, “In addition, we are executing on our late-stage pipeline across three orphan/rare [central nervous system] franchises, which we expect to deliver at least one new product or indication launch every year over the next five years, with multi-billion-dollar revenue potential extending beyond 2040. We also delivered another strong quarter of revenue growth for Wakix, confirming our confidence in Wakix being a billion-dollar plus market opportunity in narcolepsy alone while gaining approval and launching Wakix in pediatric narcolepsy.”
Key Franchise Highlights
Key franchise highlights in Harmony’s sleep/wake portfolio for the quarter include:
- Wakix net revenue of $172.8 million in the second quarter of 2024, representing 29% growth over the same period in 2023.
- The average number of patients on Wakix increased by approximately 250 patients sequentially to approximately 6,550 for the quarter.
- Wakix patent upheld again. Harmony announced that the United States Patent and Trademark Office issued a final denial of the petition for reexamination, which was filed by a short seller. This denial is non-appealable.
- Received FDA approval for Wakix for the treatment of excessive daytime sleepiness in pediatric patients 6 years and older with narcolepsy on June 21 and executed commercial launch July 1.
- On track to submit supplemental new drug application for pitolisant in idiopathic hypersomnia in the fourth quarter of 2024.
- Pitolisant Gastro-Resistant (GR): On track to initiate a dosing optimization study in the fourth quarter of 2024 and a pivotal bioequivalence study in the first quarter of 2025. A PDUFA date is expected in 2026. A provisional patent was filed with the potential for patent protection out to 2044.
- Pitolisant HD: Pilot PK data showed meaningful differentiation with at least a ~ 20% increase in relative bioavailability and a decrease in the variability of the PK profile compared to an equivalent Wakix dose of 35.6 mg, the highest labeled dose. Formulation optimization work continues, and Harmony plans to study up to two times the current highest labeled dose of Wakix, where the company expects to demonstrate a further increase in relative bioavailability and decrease in variability in the PK profile. An optimized PK profile, along with a higher dose, GR coating, and targeting unique symptoms (eg, fatigue in narcolepsy, in addition to excessive daytime sleepiness and cataplexy) is expected to provide a differentiated product profile and label compared to Wakix. PDUFA date expected in 2028. Provisional patent filed with the potential for patent protection out to 2044.
- Patient enrollment is ongoing in phase 3 TEMPO study in patients with Prader-Willi syndrome.
- TPM-1116, a highly potent and selective oral orexin-2 receptor agonist that will be evaluated for the treatment of narcolepsy and other sleep-wake disorders. Investigational New Drug (IND)-enabling studies are ongoing, and Harmony expects to file an IND in mid-2025 and initiate first-in-human studies in the second half of 2025.
- Completed pre-clinical proof-of-concept study for HBS-102 in Prader-Willi syndrome with encouraging initial results. The final study report and results are expected to be shared later this year.
Financial Results
Net product revenues for the quarter ended June 30 were $172.8 million, compared to $134.2 million for the same period in 2023.
The 29% 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 health care providers that Harmony calls on (about 5,000 of whom do not participate in an oxybate REMS program).
Harmony expects full-year 2024 net product revenue of $700 million to $720 million.
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