Orchard Therapeutics plc

Total revenue in Q2 2023 was $7.3 million, including $6.6 million of Libmeldy sales and $0.7 million of collaboration revenue. Cost of product sales was $2.2 million, and gross margin was approximately 70% for the quarter. Total operating expenses declined 22% versus Q2 2022, with both R&D and SG&A down more than 20% year over year. Cash and investments were approximately $155 million at quarter-end, with runway into mid-2025. Management said it expects to hear from the FDA on BLA acceptance in Q3 and is requesting priority review, which could support a potential U.S. approval in the first half of 2024. For the rest of 2023, the company’s goals are to keep growing Libmeldy sales year over year and continue reducing operating expenses, with annual burn expected to trend down.

Bobby Gaspar framed the quarter as a broad execution update across regulatory, commercial, clinical, manufacturing, and early research, saying the company is “one significant step closer” to U.S. approval for OTL-200. He highlighted the breadth of the HSC gene therapy platform and said recent data presentations reinforce Orchard’s leadership in neurometabolic and CNS disorders. His tone was confident and upbeat, emphasizing that the company is becoming “well funded” and positioned to be a “breakout leader” in HSC gene therapy.

Frank Thomas emphasized stronger Libmeldy revenue, operating discipline, and financing support. He said the second closing of the strategic financing brought in $34 million, sold at $8 per ADS, which he described as a 64% premium to the share price on the date of issuance, and said first- and second-closing proceeds extend cash runway to mid-2025. He also pointed to $7.3 million in Q2 revenue, $2.2 million in cost of product sales, about 70% gross margin, and a 22% decline in operating expenses year over year.

Analysts asked about the likelihood of an FDA AdCom for OTL-200, and Bobby Gaspar said it is possible because this is the first submission for an MLD therapy, adding that Orchard would welcome it to show the unmet need and lack of alternatives. Questions also focused on manufacturing inspection readiness; management said AGC Biologics has EU commercial manufacturing experience but not an FDA inspection yet, and Orchard has run mock inspections to prepare. On the U.S. launch, Braden Parker said commercialization should be similar to Europe in patient population and center count, but reimbursement should happen much sooner, while patient identification may still be uneven until newborn screening expands. For the MPS-1 pivotal study, management said the FDA study design is already final and the remaining work is operational site setup, with opening planned by year-end.