Ultragenyx Pharmaceutical(RARE) - 2024 Q3 - Quarterly Report

Product Portfolio - Ultragenyx Pharmaceutical Inc. has a diverse portfolio of approved therapies and clinical product candidates targeting serious rare and ultrarare genetic diseases[65] - Crysvita is the only approved treatment for X-Linked Hypophosphatemia (XLH), with approximately 48,000 patients in the developed world, including 36,000 adults and 12,000 children[68] - Mepsevii is approved for Mucopolysaccharidosis VII (MPS VII), affecting an estimated 200 patients in the developed world[69] - Dojolvi is approved for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), with approximately 8,000 to 14,000 patients in the developed world[70] - Evkeeza is approved for Homozygous Familial Hypercholesterolemia (HoFH), with an estimated 3,000 to 5,000 patients in the developed world outside the U.S.[71] - UX143 (setrusumab) received Breakthrough Therapy Designation from the FDA based on positive Phase 2 results showing a decrease in fracture rate in Osteogenesis Imperfecta patients[79] - GTX-102 for Angelman Syndrome is set to begin a pivotal Phase 3 study by the end of 2024, enrolling approximately 120 patients[80] - UX111 for MPS IIIA is expected to file a Biologics License Application (BLA) around the end of 2024 following a successful pre-BLA meeting with the FDA[81] - DTX401 for Glycogen Storage Disease Type Ia (GSDIa) has an estimated 6,000 patients in the developed world[75] - UX701 for Wilson Disease affects more than 50,000 patients in the developed world and has received Orphan Drug Designation in both the U.S. and EU[77] Financial Performance - Total revenues for the three months ended September 30, 2024, were $139.5 million, a 42$98.1 million in the same period in 2023[85] - The company incurred a net loss of $133.5 million for the three months ended September 30, 2024, compared to a net loss of$159.6 million for the same period in 2023[85] - Crysvita product sales increased by 85% to $35.6 million for the three months ended September 30, 2024, from$19.2 million in the same period in 2023[87] - Total product sales for the nine months ended September 30, 2024, were $213.5 million, an increase of 66$128.8 million in the same period in 2023[89] - Cost of sales for the three months ended September 30, 2024, was $21.0 million, a 91$11.0 million in the same period in 2023[90] - Total gene therapy programs revenue reached $52.7 million, a 21$43.7 million in 2023[92] - UX111 program revenue surged by 202% to $18.1 million from$6.0 million in 2023[92] - Total biologic and nucleic acid programs revenue increased by 41% to $39.3 million from$27.9 million in 2023[92] - Interest income for the three months ended September 30, 2024, was $10.7 million, an increase of 82$5.9 million in 2023[97] - Change in fair value of equity investments recorded a net increase of $0.7 million for the three months ended September 30, 2024, compared to a loss of$1.4 million in 2023[98] - Other income increased by $3.8 million for the three months ended September 30, 2024, compared to a loss of$0.7 million in 2023[100] Expenses and Cash Flow - Research and development expenses increased significantly, reflecting ongoing clinical studies and development activities[91] - Total research and development expenses increased by $12.9 million (8$22.2 million (5%) for the nine months ended September 30, 2024, compared to the same periods in 2023[94] - Selling, general and administrative expenses increased by $5.4 million (7$6.1 million (3%) for the nine months ended September 30, 2024, compared to the same periods in 2023[96] - Non-cash interest expense on liabilities for sales of future royalties decreased by $1.9 million (11$1.5 million from $0.5 million in 2023[92] - Cash used in operating activities for the nine months ended September 30, 2024 was$334.7 million, reflecting a net loss of $435.8 million, compared to$390.9 million in the same period of 2023[105] - Cash used in investing activities for the nine months ended September 30, 2024 was $120.5 million, primarily related to marketable debt securities[106] Financing Activities - Cash provided by financing activities for the nine months ended September 30, 2024 was$396.1 million, primarily from the issuance of common stock and pre-funded warrants[107] - The company completed a public offering in October 2023, raising $326.5 million net of underwriting discounts and commissions, and another offering in June 2024, raising$381.0 million[102][103] Future Outlook - The company plans to submit a marketing application for DTX401 in mid-2025 following positive Phase 3 study results[82] - Enrollment for the Phase 3 study of DTX301 is expected to be completed by the end of 2024, with a target of 50 patients aged 12 years and older[83] - The company anticipates continuing annual losses for the next several years as it develops and seeks regulatory approvals for its product candidates[107][108] Assets and Obligations - The company had $824.7 million in available cash, cash equivalents, and marketable debt securities as of September 30, 2024[85] - As of September 30, 2024, the company had$824.7 million in available cash, cash equivalents, and marketable debt securities, an increase from $777.1 million as of December 31, 2023[102][109] - Future minimum lease payments under non-cancellable leases as of September 30, 2024, were approximately$42.5 million[108] - The company has obligations of approximately $35.6 million related to manufacturing and service contracts as of September 30, 2024[108] - A hypothetical 100 basis point change in interest rates would not have had a material impact on the fair market value of the company's cash equivalents and marketable debt securities as of September 30, 2024[109] Taxation - The provision for income taxes decreased by 53% for the three months ended September 30, 2024, compared to the same period in 2023[101]