Harmony Biosciences(US:HRMY)2025-11-04 13:42Drug Approvals and Clinical Trials - Pitolisant was approved by the FDA for the treatment of EDS in adult patients with narcolepsy in August 2019, with commercial launch initiated in November 2019[123]. - The FDA granted Orphan Drug designation to pitolisant for the treatment of PWS in February 2024, with the Phase 3 TEMPO study initiated in Q1 2024[125]. - The Phase 2 trial for pitolisant in adult patients with DM1 showed clinically meaningful improvements in EDS and fatigue, with topline results announced in Q4 2023[125]. - The pivotal bioequivalence study for Pitolisant GR is anticipated to have topline data readout in Q4 2025, with a PDUFA date expected in Q1 2027[129]. - The company received positive pre-clinical data for BP1.15205, an orexin-2 receptor agonist, with first-in-human studies expected to begin in Q4 2025[130]. - A research collaboration agreement with CiRC Biosciences was established in June 2025 to develop candidates for refractory epilepsies and treatment-resistant narcolepsy[131]. - The FDA did not approve the sNDA for expanding the WAKIX label for pediatric patients with cataplexy in June 2024, but a Type A meeting was held to discuss a resubmission path[127]. - The company is committed to obtaining pediatric exclusivity for WAKIX, supported by recent FDA approvals and ongoing studies[128]. - The RECONNECT study for ZYN002 in Fragile X Syndrome did not meet its primary endpoint due to a higher than expected placebo response rate, leading to a review of the full data set[134]. - EPX-100 is currently in two Phase 3 registrational clinical trials, the ARGUS Study for Dravet Syndrome and the LIGHTHOUSE Study for Lennox-Gastaut Syndrome[135]. Financial Performance - Net product revenue for the three months ended September 30, 2025, was $239.5 million, a 28.7% increase compared to the same period in 2024, driven by a 26.8% increase in units shipped[156]. - For the nine months ended September 30, 2025, net product revenue reached $624.7 million, reflecting a 21.7% increase year-over-year, primarily due to an 18.0% increase in units shipped[156]. - The cost of product sales for the three months ended September 30, 2025, was $59.7 million, up from $42.8 million in the same period in 2024[155]. - Research and development expenses for the three months ended September 30, 2025, were $55.0 million, significantly higher than $25.4 million in the same period in 2024, reflecting increased clinical program efforts[155]. - Interest expense decreased by $0.7 million, or 16.7%, for the three months ended September 30, 2025, and decreased by $2.2 million, or 16.4%, for the nine months ended September 30, 2025, compared to the same periods in 2024[164]. - Interest income increased by $0.8 million, or 16.2%, for the three months ended September 30, 2025, and increased by $2.0 million, or 14.3%, for the nine months ended September 30, 2025, compared to the same periods in 2024[165]. - The company achieved a net income of $136.2 million for the nine months ended September 30, 2025, compared to $96.0 million in 2024, marking a 42% increase[183][184]. Expenses and Cost Management - The company expects sales and marketing expenses to rise in the near- and mid-term to support WAKIX's commercialization and potential new indications[147]. - General and administrative expenses are anticipated to increase due to hiring additional personnel and compliance costs associated with being a public company[150]. - Cost of product sales increased by $16.9 million, or 39.4%, for the three months ended September 30, 2025, and increased by $27.4 million, or 26.7%, for the nine months ended September 30, 2025, compared to the same periods in 2024[157]. - Research and development expenses increased by $29.6 million, or 116.5%, for the three months ended September 30, 2025, and increased by $28.5 million, or 25.6%, for the nine months ended September 30, 2025, compared to the same periods in 2024[159]. - Sales and marketing expenses increased by $2.0 million, or 7.2%, for the three months ended September 30, 2025, and increased by $7.0 million, or 8.4%, for the nine months ended September 30, 2025, compared to the same periods in 2024[162]. - General and administrative expenses increased by $1.2 million, or 4.3%, for the three months ended September 30, 2025, and increased by $13.5 million, or 16.6%, for the nine months ended September 30, 2025, compared to the same periods in 2024[163]. Cash Management and Investments - As of September 30, 2025, the company had cash, cash equivalents, and investments of $778.4 million, outstanding debt of $170.0 million, and retained earnings of $138.4 million[168]. - For the nine months ended September 30, 2025, cash provided by operating activities was $222.0 million, up from $144.3 million in the same period of 2024, representing a 54% increase[182]. - Net cash used in investing activities for the nine months ended September 30, 2025, was $22.0 million, a decrease from $60.3 million in 2024, indicating improved cash management[185][186]. - The company reported net cash used in financing activities of $6.0 million for the nine months ended September 30, 2025, compared to $8.3 million in 2024, reflecting a reduction in financing costs[187][188]. - As of September 30, 2025, the company had $685.2 million in investments, with an immediate 10% change in market interest rates not expected to materially impact the investment portfolio[194]. - The company had $170.0 million in borrowings outstanding as of September 30, 2025, with interest rates based on a margin ranging from 2.50% to 4.00% depending on the leverage ratio[195]. Acquisitions and Agreements - In October 2023, the company acquired Zynerba Pharmaceuticals, gaining global rights to develop and commercialize ZYN002, a synthetic cannabidiol gel for transdermal delivery[134]. - In April 2024, the company acquired Epygenix Therapeutics, obtaining an exclusive license for EPX-100, targeting Dravet Syndrome and Lennox-Gastaut Syndrome, with a total addressable market of approximately 5,000 and 35,000 patients respectively[135]. - The company acquired HBS-102, a MCHR1 antagonist, with encouraging results from preclinical studies reported in Q3 2024[133]. - The company entered into a License and Commercialization Agreement with Bioprojet in July 2022, obtaining exclusive rights to develop new products based on pitolisant in the U.S. and Latin America[129]. - The company entered into a sublicense agreement with Bioprojet in April 2024, paying an upfront license fee of $25.5 million and potentially up to $367.5 million upon achieving development and sales milestones[177]. - The company entered into a research collaboration with CiRC Biosciences, Inc., paying an upfront fee of $15.0 million and potential milestone payments of $2.0 million for each of the two candidates[179][180]. - A clinical milestone for ZYN002 was achieved in September 2025, triggering a $15.0 million payment to contingent value rights holders, expected to be paid in Q4 2025[181].