HRMY

• Commercial Performance of WAKIX

  • WAKIX achieved record quarterly net revenue, bouncing back from seasonal access headwinds that impacted Q1 2026
  • The brand has a 40% compounded annual growth rate over the past 5 years, with over 80% of U.S. patient lives covered by payers, and holds a unique position as the only non-scheduled treatment for narcolepsy
  • A 20% expansion of the commercial field team (the largest in brand history) was fully deployed in Q2, alongside a new prescribing portal and updated reimbursement support that has improved patient access speed and success rates
  • Total addressable narcolepsy population is ~170,000, with diagnosis rates under 50% and current brand penetration of ~20%, leaving significant room for continued growth
  • Two life cycle extension programs are in development: pitolisant GR (reduced GI side effects, no titration requirement) and pitolisant HD (higher optimized dose, potential differentiated labeling for unmet symptom needs)

• Pipeline Progress

  • Positive top-line data was released from the Phase I single ascending dose (SAD) study for BP-205, the company's potential best-in-class orexin-2 receptor agonist
  • BP-205 SAD data showed: rapid absorption (Tmax 30-75 minutes), 25-hour mean half-life supporting once-daily dosing, dose-proportional exposure, no age/gender PK differences, and a favorable safety profile with no severe/serious adverse events
  • Multiple ascending dose (MAD) study data is expected in Q4 2026; a Phase Ib study in sleep-deprived healthy volunteers will initiate in Q3 2026, with top-line data expected in early 2027; Phase II trials in multiple CNS indications will initiate mid-2027
  • The pitolisant GR NDA was submitted and accepted for review in Q2 2026, with a PDUFA date of April 1, 2027
  • Two Phase III trials for pitolisant HD (ONSTRIDE 1 in narcolepsy, ONSTRIDE 2 in idiopathic hypersomnia) are ongoing, with top-line data expected in 2027 and a PDUFA target of 2028; the program targets unmet needs: fatigue in narcolepsy and sleep inertia in idiopathic hypersomnia, with no currently approved treatments for these symptoms
  • The Phase III TEMPO study for Prader-Willi syndrome is actively recruiting, with top-line data now expected mid-2027 (delayed due to low patient prevalence of eligible participants); the study will complete the requirement for WAKIX pediatric exclusivity
  • Two global Phase III trials for EPX-100 (clemizole hydrochloride) for Dravet syndrome and Lennox-Gastaut syndrome are ongoing, with top-line data expected mid-2027 and PDUFA in 2028

• Business Development and Intellectual Property

  • The company ended Q2 with $962.5 million in cash/cash equivalents/investments and $155 million in debt, giving it capacity to pursue strategic transactions
  • BD efforts are focused on late-stage (Phase III/registered/marketed) CNS assets in Sleep/Wake, epilepsy, and rare orphan CNS, with expected revenue contribution between 2028 and 2032; multiple smaller transactions are prioritized over one large transformative deal, with M&A and licensing both under consideration
  • The company's multilayered IP estate protects WAKIX exclusivity through March 2030, including 6 months of pediatric exclusivity that remains on track to be obtained; next-generation pitolisant formulations have patent protection extending into the 2040s
  • 6 of 7 ANDA filers have settled litigation; two active proceedings remain against the lone remaining filer, with closing arguments scheduled for October 2026; management is confident it will prevail in both cases

• Financial Performance

  • Q2 2026 net income was $75.4 million ($1.28 per diluted share), up from $39.8 million ($0.68 per diluted share) year-over-year
  • Total operating expenses were $108.8 million, down from $114.2 million year-over-year, primarily due to the absence of a $15 million 2025 upfront fee, partially offset by increased R&D and commercial investments

• Full year 2026 net revenue guidance for WAKIX is maintained at $1 billion to $1.04 billion, with the company on track to exceed $1 billion in annual net revenue
• Top-line BP-205 MAD study data is expected to be released in Q4 2026, with Phase Ib study data expected in early 2027
• BP-205 Phase II trials in multiple CNS indications are on track to initiate in mid-2027
• Pitolisant GR PDUFA is set for April 1, 2027
• Top-line data for pitolisant HD Phase III trials, the Prader-Willi syndrome Phase III study, and the EPX-100 Phase III studies are all expected in mid-2027, with PDUFA for pitolisant HD and EPX-100 targeted for 2028
• WAKIX pediatric exclusivity remains on track to be obtained, supporting exclusivity through March 2030

Harmony Biosciences has only one commercially available product segment: WAKIX (pitolisant) for narcolepsy. In Q2 2026, WAKIX delivered record net revenue of $261.3 million, representing 100% of the company's total net revenue for the quarter. This reflects a 30% year-over-year increase and a 21% sequential increase from Q1 2026. Cost of product sold for WAKIX was 24.2% of net revenue in the quarter, up from 19% year-over-year due to new royalties from the Novitium license agreement signed in Q1 2026. Average patient count on WAKIX reached 8,950, an increase of 450 patients from Q1 2026, the second highest quarterly patient increase in the brand's history.

• Actual clinical trial results and future product performance may differ materially from current expectations, due to inherent risks in clinical development and regulatory approval processes
• Ongoing ANDA litigation with the remaining generic filer could result in an unfavorable ruling that would allow generic entry before March 2030, impacting WAKIX revenue
• BP-205's clinical profile and benefit-risk balance may not ultimately prove to be best-in-class compared to competing orexin-2 agonists from other developers that are further along in development
• Patient recruitment for rare disease trials (such as the Prader-Willi syndrome study) can be slower than expected, leading to additional delays in data readout and regulatory submission
• The company may not be able to identify or complete attractive strategic business development transactions on favorable terms, or transactions may not deliver the expected long-term value
• The broader market for new CNS indications such as ADHD or MS fatigue is more promotion-intensive than the company's historic rare disease focus, requiring additional investment and scaling that may not yield expected returns

Q: How do BP-205's short Tmax, 25-hour half-life, and high potency translate to a differentiated clinical profile compared to competitors that require twice-daily dosing, and how does this enable use in broader CNS indications? / A: A short 30-75 minute Tmax enables rapid efficacy onset, which is particularly beneficial for hypersomnolence disorders like idiopathic hypersomnia with sleep inertia, as well as broader indications like ADHD. The 25-hour half-life, which matches the range of other successful once-daily neuropsychiatric drugs including WAKIX, supports convenient once-daily dosing and sustains wakefulness through the afternoon and early evening. BP-205's status as the most potent orexin-2 agonist in clinical development allows use of lower doses across a broad range of CNS indications, including those without orexin deficiency, while its high 600-fold selectivity over orexin-1 receptors provides a large safety margin that minimizes off-target adverse events. Target engagement markers like transient insomnia and polyuria were observed in the 15-day multiple ascending dose study, but no events were serious or sustained, with full MAD data to be disclosed in Q4.

Q: BP-205 uses a novel chemical scaffold. What advantages does this provide over other orexin-2 agonist scaffolds, and how should we think about development timelines relative to competitors like Takeda? / A: Unlike the common pyrrolidine sulfonamide bicyclic scaffolds used by other candidates, BP-205's novel structure avoids molecular features linked to historical issues of hepatotoxicity and cardiac toxicity observed in earlier orexin-2 agonist programs. While Harmony is not expected to be first to market for hypersomnolence indications, management plans to accelerate development of multiple Phase II trials in broader CNS indications starting in mid-2027, with the potential to be first or second to market in these larger indications. Management notes that first-to-market assets are not always best-in-class, and BP-205's emerging differentiated profile positions it well to capture market share across indications.

Q: How is Harmony prioritizing rare versus broad large-market indications for BP-205, and do you have the commercial infrastructure to enter larger non-orphan markets? What is the BP-205 IP expiry timeline? / A: Harmony will pursue multiple indications in parallel, building on its existing commercial foundation in the rare hypersomnolence space while accelerating development of broader CNS indications. Multiple Phase II trials starting mid-2027 will generate data to inform future Phase III and commercialization decisions. Management notes that the company has substantial in-house experience with large-market commercialization from the team's prior experience, and its existing $1 billion annual revenue base provides the scale and capacity to expand commercial infrastructure for larger markets. BP-205's core composition patent expires in 2043, with potential for additional patent term extensions.

Q: How would you define a best-in-class safety profile for BP-205, and what directional context can you provide on the incidence of insomnia and polyuria observed in the MAD study? / A: A best-in-class safety profile requires minimizing both off-target adverse events linked to poor compound design and avoiding severe/sustained on-target adverse events. To date, BP-205 has shown no off-target cardiovascular, hepatic, or visual adverse events (issues that have impacted other candidates) and has very high selectivity that minimizes off-target interactions. Full data on adverse event incidence, dose response, and resolution is still being analyzed and will be disclosed comprehensively in Q4 2026. To date, all observed insomnia and polyuria events have been transient, not severe or sustained, which is consistent with the favorable emerging profile management expects.