Ultragenyx Pharmaceutical (RARE) incurred a third-quarter 2024 loss of $1.40 per share, narrower than the Zacks Consensus Estimate of a loss of $1.45. The company had reported a loss of $2.23 per share in the year-ago quarter.      Ultragenyx’s total revenues amounted to $139.5 million in the reported quarter, up 42% year over year. The top line surpassed the Zacks Consensus Estimate of $134 million on the back of higher product revenues.The company markets four drugs, namely Crysvita, Mepsevii, Dojolvi and ...

Ultragenyx (RARE) reported $139.49 million in revenue for the quarter ended September 2024, representing a year-over-year increase of 42.3%. EPS of -$1.40 for the same period compares to -$2.23 a year ago.The reported revenue represents a surprise of +4.04% over the Zacks Consensus Estimate of $134.08 million. With the consensus EPS estimate being -$1.45, the EPS surprise was +3.45%.While investors closely watch year-over-year changes in headline numbers -- revenue and earnings -- and how they compare to Wa ...

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% increase from $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% from $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% increase from $11.0 million in the same period in 2023[90] - Total gene therapy programs revenue reached $52.7 million, a 21% increase from $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% compared to $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%) for the three months and $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%) for the three months and $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%) for the three months ended September 30, 2024, compared to 2023[99] - CMC costs for gene therapy programs rose by 177% to $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]

Ultragenyx (RARE) came out with a quarterly loss of $1.40 per share versus the Zacks Consensus Estimate of a loss of $1.45. This compares to loss of $2.23 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 3.45%. A quarter ago, it was expected that this biotechnology company would post a loss of $1.64 per share when it actually produced a loss of $1.52, delivering a surprise of 7.32%.Over the last four quarters, the company has s ...

Third quarter 2024 total revenue grew 42% versus prior year to $139 million, including Crysvita® revenue of $98 million and Dojolvi® revenue of $21 million Reaffirmed 2024 expected total revenue guidance of $530 million to $550 million Breakthrough Designation granted for setrusumab (UX143) in osteogenesis imperfecta DTX401 Phase 3 follow-up data demonstrated higher and faster 62% mean reduction in cornstarch in crossover patients with glycogen storage disease type Ia (GSDIa) NOVATO, Calif., Nov. 05, 2024 ( ...

Core Insights - Ultragenyx Pharmaceutical Inc. reported positive results from the Phase 1/2/3 Cyprus2+ study of its UX701 gene therapy for Wilson disease, showing meaningful clinical activity and improvements in copper metabolism [1][2][3] Group 1: Study Results - In Stage 1, 15 patients were enrolled in three dosing cohorts, with six patients completely tapering off standard-of-care treatment, and a seventh patient beginning to taper [2][4] - Non-ceruloplasmin bound copper (NCC) levels stabilized to normal in patients who tapered off standard-of-care, indicating improved copper metabolism [2] - UX701 was well tolerated with no unexpected treatment-related adverse events reported as of the data cut-off [2] Group 2: Future Plans - The company plans to enroll an additional cohort at a moderately increased dose and with an optimized immunomodulation regimen to enhance the therapy's efficacy [1][3] - A fourth dosing cohort will be added, and all patients in Stage 1 will be evaluated over 52 weeks [4] Group 3: Study Design - The study is designed in three stages, with Stage 1 focusing on safety and efficacy across multiple dose levels of UX701 [4][5] - In Stage 2, a new cohort will be randomized to receive either the selected dose of UX701 or a placebo, with primary efficacy endpoints including changes in 24-hour urinary copper concentration [5] Group 4: About Wilson Disease - Wilson disease is a rare genetic disorder caused by mutations in the ATP7B gene, leading to copper accumulation in the body and various health issues [6] - The disease affects over 50,000 people in commercially accessible geographies, highlighting a significant unmet medical need [6] Group 5: About UX701 - UX701 is an investigational AAV9 gene therapy designed to deliver stable expression of the ATP7B copper transporter following a single intravenous infusion [7] - It has received Orphan Drug Designation in the U.S. and EU, as well as Fast Track Designation in the U.S. [7] Group 6: About Ultragenyx Pharmaceutical Inc. - Ultragenyx is focused on developing novel therapies for rare and ultrarare genetic diseases, with a diverse portfolio aimed at addressing high unmet medical needs [8] - The company emphasizes time- and cost-efficient drug development to deliver safe and effective therapies urgently [9]

NOVATO, Calif., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it will present seven abstracts related to its ongoing late-stage program evaluating setrusumab (UX143) and osteogenesis imperfecta (OI), including a late-breaker oral presentation of the 14- month data from the Phase 2/3 Orbit study, at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting. The meeting is being held September 27-30, 2024, in Toronto, Canada. "Presen ...

Morgan Stanley 22nd Annual Global Healthcare Conference on September 4 Cantor Global Healthcare Conference on September 17 Bank of America Global Healthcare Conference on September 18 NOVATO, Calif., Aug. 29, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced participation in three upcoming investor conferences. Morgan Stanley 2 ...

Andrzej Rostek Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops innovative therapies for rare and ultra-rare genetic diseases. Its commercial portfolio includes Crysvita, Mepsevii, Dojolvi, and Evkeeza, which address unmet medical needs in conditions like X-linked hypophosphatemia [XLH], Mucopolysaccharidosis VII [MPS VII], long-chain fatty acid oxidation disorders [LC-FAOD], and homozygous familial hypercholesterolemia [HoFH]. Additionally, RARE's pipeline targets severe diseases such as osteogenesis ...

Core Insights - The completion of the human genome sequencing in 2003 has significantly advanced the understanding of genetic medicine, presenting new investment opportunities in genomics stocks as modern therapies increasingly rely on patient genetics [1][3] - Genomics companies differ from traditional healthcare stocks as they often profit from supporting the broader medical industry rather than solely relying on drug sales [2] - Investing in genomics stocks can provide slower yet more stable growth, helping to dilute portfolio risk when appropriately allocated [3] Company Summaries - **Sarepta Therapeutics (SRPT)**: The company has positioned itself well in the biotech sector, focusing on gene editing and RNA technologies. Its stock has increased by 24% since January, driven by its narrow patient population and potential to treat genetic muscular dystrophies, which creates a competitive advantage [4][5]. A notable project involves using RNA molecules to produce proteins in target cells, offering symptom relief for genetic diseases [6] - **Bio-Techne (TECH)**: This company supports the genomics industry by providing workflow services and products for gene therapies and genetic research. Its focus on creating replicable processes is crucial for achieving broader acceptance in the medical field. As genomics research grows, Bio-Techne stands to benefit from increased sales of its products and services, making it a candidate for long-term investment [7][8] - **Ultragenyx Pharmaceutical (RARE)**: Known for developing treatments for ultra-rare genetic diseases, Ultragenyx has successfully gained FDA approval for its monoclonal antibodies and enzyme replacements. The company's stock has risen by 40% over the past year, driven by strong earnings per share and revenue growth. Monitoring the commercialization of its genetic treatments could be key for investors looking to capitalize on potential profits [9][10][11]