Harmony Biosciences Q1 Earnings Call Highlights

Key Points

• WAKIX drove Q1 net product revenue of $215.4 million, up 17% year‑over‑year, with roughly 8,500–8,600 patients on therapy and full‑year net revenue guidance reiterated at $1.0B–$1.04B.
• Harmony has strengthened its pitolisant IP (including a Novitium license with patents extending to 2042), settled six of seven ANDA challengers, and says it expects to maintain exclusivity into 2030 while the remaining AET litigation and related suits proceed.
• Multiple pipeline catalysts are nearing readouts: Pitolisant GR NDA is on track for Q2 2026 (PDUFA Q1 2027), Pitolisant HD phase III top‑line data in 2027 (targeting 2028 PDUFA), BP‑205 SAD PK data due mid‑2026, and EPX‑100 phase III epilepsy data expected H2 2027.

Harmony Biosciences NASDAQ: HRMY reported first-quarter 2026 net product revenue of $215.4 million, a 17% increase from $184.7 million in the prior-year quarter, as the company highlighted continued demand for its narcolepsy treatment WAKIX while outlining a four-part strategy focused on franchise protection, lifecycle expansion, pipeline advancement, and business development.

“We are a profitable, self-funding biotech company that continues to operate from a position of strength,” President and CEO Dr. Jeffrey Dayno said, adding that the company is “now turning our focus on four key pillars of value creation for shareholders.”

WAKIX growth and full-year guidance reiterated

Management said first-quarter performance reflected sustained underlying demand for WAKIX, tempered by typical first-quarter market access friction. Dr. Dayno said Harmony averaged roughly 8,500 patients on WAKIX during the quarter and exited Q1 with about 8,600 average patients on therapy. He also reiterated the company’s full-year net revenue guidance of $1.0 billion to $1.04 billion.

Chief Commercial Officer Adam Zaeske said the first quarter is typically slower for the industry due to seasonal dynamics, and this year included “slightly higher market access headwinds than previous years with a higher level of plan changes, plan switching by consumers, and higher premium increases which can delay patient starts.” Still, he said demand remained strong, noting that March new prescription levels were higher than all but two months in 2025. Zaeske said the company expects the balance of 2026 to follow a familiar seasonal pattern consistent with prior years.

Zaeske also pointed to WAKIX’s positioning as “the only non-scheduled treatment option for narcolepsy patients,” and said healthcare providers view it as a “familiar go-to option” due to efficacy, safety and tolerability, low drug-drug interactions, and broad payer coverage. In response to a question about discontinuations, Zaeske said persistency has “remained very steady for many years,” and he did not cite any meaningful change in discontinuation dynamics.

On commercial execution, Zaeske said Harmony completed a field team expansion that increased its presence by about 20% across field sales, remote sales, and field reimbursement, with all positions in place as of April 1. He added that Harmony plans in the second quarter to launch an online portal intended to simplify prescribing, alongside “significant changes” to reimbursement support aimed at helping patients access therapy more quickly and with improved success rates.

IP strategy and litigation updates

Dayno said Harmony has “solidified our IP position around the pitolisant franchise through a multi-layered approach,” and described the pitolisant intellectual property estate as covering formulations, methods of use, and next-generation applications. He said the company’s strategy supports WAKIX exclusivity into 2030, including six months of pediatric exclusivity, and could extend protection into the 2040s through additional issued patents and pending applications.

In January, Harmony acquired an exclusive license from Novitium for intellectual property covering an amorphous form of pitolisant hydrochloride, including issued patents extending to 2042. Dayno said Harmony has now settled with six of the seven abbreviated new drug application (ANDA) filers, maintaining loss of exclusivity (LOE) until March 2030, inclusive of pediatric exclusivity.

The remaining ANDA filer, AET, went to trial in February. Dayno said the ANDA litigation is ongoing and in post-trial briefing, with that portion expected to be completed toward the end of May. During the February trial, according to management, AET and Sandoz stated their product contains an amorphous form of pitolisant hydrochloride rather than a crystalline form covered by Harmony’s ’197 polymorph patent. Dayno said Harmony and Novitium recently filed a separate patent infringement lawsuit against AET Pharma US and Sandoz alleging infringement of the ’920 patent covering the amorphous form.

Chief Operating Officer Peter Anastasiou said the newer lawsuit was filed after evidence presented during the ANDA trial, and “in no way reflects any other belief” than the company’s view that it will prevail in the ANDA case. He said Harmony filed the additional suit “to make sure that we’re asserting our full rights,” and characterized the strategy as multi-layered.

On timing, Anastasiou said the 30-month stay ends in February 2027 and the company expects a ruling on the ANDA case around that timeframe, followed by potential appeals. He added that the second case “could take 24 to 30 months in and of itself, plus an appeals process,” and said the company believes “in almost every scenario” it maintains exclusivity into 2030 inclusive of pediatric exclusivity. Dayno said the company could not comment on whether other parties might pursue an at-risk launch, while Anastasiou said Harmony would use available legal remedies to defend its intellectual property.

Lifecycle management: Pitolisant GR, Pitolisant HD, and amorphous formulation work

Management provided updates on multiple pitolisant lifecycle programs. Dayno said Pitolisant GR remains on track for an NDA submission in the second quarter of 2026, with a target PDUFA date in Q1 2027. Chief Medical and Scientific Officer Dr. Kumar Budur said Pitolisant GR is designed with enteric coating intended to reduce potential gastrointestinal side effects and demonstrated bioequivalence to WAKIX in a pivotal bioequivalence study. Budur added that Pitolisant GR could allow patients to start at a therapeutic dose range of 17.8 mg without titration.

For Pitolisant HD, Budur said two phase III registrational trials are ongoing: ONSTRIDE 1 in narcolepsy and ONSTRIDE 2 in idiopathic hypersomnia (IH). He said the company anticipates top-line data in 2027 and is targeting a 2028 PDUFA date. Budur said the programs are pursuing differentiated labels focused on fatigue in narcolepsy and sleep inertia in IH, which he described as symptoms with no currently approved treatments. He also said both Pitolisant GR and Pitolisant HD have utility patents filed until 2044.

On the newly licensed amorphous form of pitolisant, Budur said its physicochemical properties “could potentially lend itself to a different mode of oral delivery,” which may result in a pharmacokinetic profile better suited for broader CNS indications. He said Harmony is optimizing the formulation with the goal of initiating a phase I PK study.

Budur also said top-line data from the phase III TEMPO study in Prader-Willi syndrome is anticipated in the second half of 2026, calling it a key requirement tied to pediatric exclusivity that would provide an additional six months of regulatory exclusivity at the back end of the longest WAKIX patent.

Pipeline focus: BP-205 and EPX-100

Harmony highlighted BP-205, an orexin-2 receptor agonist, as a central pipeline program. Dayno described it as a “highly potent and selective” candidate and said the company expects a top-line readout from its phase I clinical PK single ascending dose (SAD) study in mid-2026. Budur said BP-205 has shown preclinical potency, selectivity, safety, and efficacy, and potential for once-a-day dosing. He called it “the most potent orexin-2 receptor agonist in clinical development” and said it demonstrated wake-promoting effect at the lowest dose tested in transgenic mouse models for narcolepsy.

Budur said the company continues dosing healthy volunteers in a phase I SAD/MAD PK study in Europe, remains on track for SAD PK data in mid-2026, and expects a U.S. IND submission in mid-2026. He said Harmony also plans to commence a sleep-deprived healthy volunteer study in the second half of 2026. Addressing why Harmony is conducting sleep-deprived healthy volunteer work rather than moving directly into patient proof-of-concept studies, Budur said the approach helps establish a dose range and will be conducted in parallel with other phase I work, adding that it should not add time to the overall development timeline.

Budur said Harmony is also conducting preclinical experiments exploring broader neuropsychiatric indications including mood, ADHD, cognition, and fatigue. In response to a question about combination potential, Budur said there is a scientific rationale to potentially combine pitolisant with an orexin agonist to achieve synergistic effects, citing known neurocircuitry between orexinergic and histaminergic neurons.

In epilepsy, Budur discussed EPX-100, which is in two global phase III registrational programs: the ARGUS study in Dravet syndrome and the LIGHTHOUSE study in Lennox-Gastaut syndrome. He said the company presented “on-course safety and effectiveness” data at the American Academy of Neurology meeting from an open-label extension (OLE) in Dravet syndrome showing what he called clinically meaningful seizure reductions and a favorable safety and tolerability profile. Budur said that among patients with at least six months of exposure to EPX-100, the company observed a median 50% reduction in seizures as measured by CMS-28 and that 50% of patients had a 50% reduction in seizures.

Budur said EPX-100 was used as adjunctive therapy in a population typically taking four to six anti-seizure medications, and in the study patients averaged about four anti-seizure medicines. He added that EPX-100 “doesn’t require any special monitoring,” citing no need for echocardiograms and no liver function test monitoring based on the data observed. He said diarrhea occurred in about 2% of patients and was the adverse event of note. Budur said to accelerate recruitment, sites are actively enrolling patients in India and China in addition to North America and Europe. He said Harmony anticipates top-line data in the second half of 2027 and is targeting a 2028 PDUFA date.

Financial results: higher operating expenses tied to licensing and investment

New CFO Glenn Reicin reported first-quarter operating expenses of $133.6 million, up from $96.5 million a year earlier, reflecting increased R&D investment and commercialization spending. Reicin said operating expenses included $32 million in upfront licensing fees related to the amorphous license agreements; excluding those costs, he said operating expenses rose about 5%.

Reicin reported GAAP net income of $32.5 million, or $0.55 per share, compared with $45.6 million, or $0.78 per share, in the prior-year quarter. He attributed part of the year-over-year change to the in-license costs, which he said were $0.45 per share on a fully taxed basis.

Cost of goods sold was 20.7% of net sales, compared with 17.3% in the year-ago quarter. Reicin said the increase was “almost entirely driven by new royalties related to the Novitium license agreement.” He added that gross margins will also reflect step-up royalties related to Bioprojet. On expenses, he said the company expects R&D costs to ramp as Harmony advances and accelerates trials, while he did not anticipate substantial increases in other run-rate expenses.

Harmony ended the quarter with $878.5 million in cash and cash equivalents and $160 million in debt. Reicin said cash flow in the quarter was muted by licensing fees, a large reduction in accrued expenses, and a modest reduction of debt, but said cash flow generation “will re-accelerate in the coming quarters.”

Dayno said business development is a renewed priority, with the company targeting opportunities with revenue potential in the 2028 to 2032 timeframe and prioritizing phase III, registrational, or on-market assets. Anastasiou said Harmony is considering M&A, licensing, and collaborations, while aiming to stay close to core competencies in sleep-wake, rare/orphan CNS disorders, epilepsy, and CNS adjacencies. Reicin said Harmony is focused on substantial returns on invested capital and “wouldn’t be just doing a deal for the purpose of growth.”

About Harmony Biosciences NASDAQ: HRMY

Harmony Biosciences Holdings, Inc is a commercial‐stage biopharmaceutical company focused on developing and delivering therapies for people with rare neurological and endocrine diseases. Founded in 2017 and headquartered in Plymouth Meeting, Pennsylvania, Harmony Biosciences went public in 2020 and trades on the Nasdaq under the ticker HRMY. The company's mission centers on identifying and advancing medicines that address critical unmet needs in patient populations underserved by existing treatments.

The company's flagship product is WAKIX (pitolisant), the first and only histamine H3 receptor antagonist/inverse agonist approved by the U.S.

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