Financial Data and Key Metrics Changes - The company's first quarter revenue was $17.3 million, a 117% increase compared to $8 million in the first quarter of 2024 [37] - Product sales reached $14 million, up 76% from $8 million in the same quarter last year, driven by increased sales of Alkindi Sprinkle and the addition of Incralyx [38] - Adjusted gross profit was $12 million, or 69.5% of total revenue, compared to $5.2 million or 65.6% in the prior year [39] - Adjusted EBITDA for the first quarter was $3.7 million, compared to $500,000 in the prior year [43] - The total net loss was $1.6 million, compared to a net loss of $800,000 in the prior year [43] Business Line Data and Key Metrics Changes - The acquisition and relaunch of Incralyx and Dalzan contributed to strong growth in existing products [6] - Alkindi Sprinkle has shown consistent growth for 17 consecutive quarters, with an increase in new patient referrals in 2025 [16] - The company expects to reach approximately 80,000,000 annual revenue run rate by the end of 2025 [38] Market Data and Key Metrics Changes - The company is focusing on pediatric endocrinology, leveraging its existing sales force and commercial infrastructure to raise awareness of severe primary IGF-1 deficiency [8] - The market for ET400 is estimated to have around 5,000 adrenal insufficiency patients under nine years of age, presenting a compelling opportunity [18] Company Strategy and Development Direction - The company aims to become a leading player in the ultra-rare disease market, with a focus on expanding its product portfolio through acquisitions and licensing [31] - The strategy includes harmonizing U.S. and EU labels for Incralyx to expand treatment opportunities significantly [13] - The company is also developing ET700, an extended-release version of Galzin, to address dosing challenges and improve patient outcomes [24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory of existing products and the potential for new product approvals in the near future [6] - The company is optimistic about the upcoming launch of ET400, expecting rapid uptake and significant revenue impact in subsequent quarters [60] - Management noted that the tariff situation is not a major concern, as most products are produced in the U.S. and the impact of European purchases is minimal [34][35] Other Important Information - The company has made significant investments in infrastructure to support growth, including a dedicated metabolic sales team and commercial activities for product relaunches [41] - The company reported a cash balance of $17.4 million and generated $2.1 million in operating cash flow during the quarter [43] Q&A Session Summary Question: Update on Incralyx label expansion meeting with FDA - The meeting request has been submitted, and the company expects to have it in the coming weeks, likely in July [45] Question: Recent interactions with FDA regarding ET400 - The company is on track for approval within two weeks, with the final step being labeling discussions [47] Question: Weekly Incralyx prescription trends - The company has significantly increased the number of patients and expects to meet or exceed revenue goals this year [56] Question: Expected timing for commercial revenue from ET400 - The launch is expected around the approval date, with revenue anticipated in Q3 [59] Question: Feedback on Galzin relaunch - The relaunch has been well-received, with a strong uptake and zero copay for patients, improving access significantly [70]

Financial Data and Key Metrics Changes - The company's first quarter revenue was $17.3 million, a 117% increase compared to $8 million in the first quarter of 2024 [38] - Product sales reached $14 million, up 76% from $8 million in the same quarter last year, driven by increased sales of Alkindi Sprinkle and the addition of Incralyx [39] - Adjusted gross profit was $12 million, or 69.5% of total revenue, compared to $5.2 million or 65.6% in the prior year [40] - Adjusted EBITDA for Q1 2025 was $3.7 million, compared to $500,000 in Q1 2024 [43] - The net loss for the quarter was $1.6 million, compared to a net loss of $800,000 in the prior year [44] Business Line Data and Key Metrics Changes - Alkindi Sprinkle has shown strong growth for 17 consecutive quarters, with an increase in new patient referrals in 2025 [15] - Incralyx has seen a rapid increase in active patients, reaching over 90, with a goal of 100 by the end of the year [8] - Galzin, a treatment for Wilson's disease, has been relaunched with a $0 copay program, improving access for patients [22] Market Data and Key Metrics Changes - The company is focusing on pediatric endocrinology, leveraging its existing sales force and commercial infrastructure to drive growth [6] - The market for ET400, an upcoming product, is estimated to have around 5,000 adrenal insufficiency patients under nine years of age, presenting a significant opportunity [17] Company Strategy and Development Direction - The company aims to expand its product portfolio through acquisitions and licensing, focusing on ultra-rare diseases [32] - The strategy includes harmonizing U.S. and EU labels for Incralyx to expand treatment opportunities significantly [12] - The company is preparing for the launch of ET400, with a PDUFA date approaching, and expects to generate substantial revenue from this product [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory, expecting to exit 2025 with an annual revenue run rate of approximately $80 million [39] - The company is optimistic about the upcoming product launches and the potential for significant revenue growth from its pipeline [33] - Management noted that the tariff situation would have minimal impact on the company, as most products are produced in the U.S. [34] Other Important Information - The company has made significant investments in infrastructure to support growth, including a dedicated metabolic sales team [42] - The company expects to maintain adjusted gross margins of approximately 70% for the full year 2025 and exceed 75% by 2028 [40] Q&A Session Summary Question: Update on Incralyx label expansion meeting with FDA - The meeting request has been submitted, and the company expects to have it in the coming weeks, likely in July [46] Question: Recent interactions with FDA regarding ET400 - The company has received feedback on labeling discussions and believes it is on track for approval within two weeks [48] Question: Weekly Incralyx prescription trends - The company has significantly increased the number of patients and expects to provide further updates on prescription trends in the coming months [58] Question: Expected timing for commercial revenue from ET400 - The company anticipates a launch around the approval date, likely in Q3, with a gradual revenue uptake thereafter [61] Question: Feedback on Galzin relaunch - The relaunch has been well-received, with a zero copay program and improved access for patients, leading to steady patient additions [72]