PLYMOUTH MEETING, Pa., Dec. 3, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced that Harmony's management team will participate in a fireside chat at the upcoming Oppenheimer Movers in Rare Disease Summit in New York, NY on Thursday, December 12, 2024, at 9:00 a.m. ET.A webcast of the fireside chat will be available on the investor page of Harmony's website at https://ir.harmonybiosciences.com/.About Harmony BiosciencesHarmony Biosciences is a pharmaceutical company de ...

NOVATO, Calif., Nov. 26, 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. Citi’s 2024 Global Healthcare Conference (Miami, FL) Tuesday, December 3, 2024, Emil Kakkis, M.D., Ph.D., CEO and President and Howard Horn, Chief Financial Officer, will participate in a fireside cha ...

MITIGATE Phase 3 Study Results Reinforce Promise of UPLIZNA® as the First Potential Treatment tor IgG4-RDPhase 4 AGILE Data Support Shortening KRYSTEXXA® Infusion TimeTHOUSAND OAKS, Calif., Nov. 14, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced the presentation of new data across its rare disease portfolio and pipeline at the annual American College of Rheumatology (ACR) Convergence 2024 conference in Washington, D.C., Nov. 14-19, 2024. New data showcase reduction in disease activity by UPLIZNA® ...

Thank you for reading my research on Seeking Alpha. If you want to learn even more about my method and how I discover these investment opportunities, please check out my subscription marketplace service, Compounding Healthcare , and sign up for a free trial.It has been years since I have considered restarting a position in Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE ) after having a string of big wins in gene therapy tickers following Biogen Inc.'s ( BIIB )He is the leader of the investing group Compoundin ...

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]

分组1 - Ultragenyx reported a quarterly loss of $1.40 per share, better than the Zacks Consensus Estimate of a loss of $1.45, and an improvement from a loss of $2.23 per share a year ago, resulting in an earnings surprise of 3.45% [1] - The company achieved revenues of $139.49 million for the quarter ended September 2024, exceeding the Zacks Consensus Estimate by 4.04%, and showing a significant increase from $98.05 million in the same quarter last year [2] - Over the last four quarters, Ultragenyx has surpassed consensus EPS estimates three times and topped consensus revenue estimates three times as well [2] 分组2 - The stock has gained approximately 6.8% since the beginning of the year, while the S&P 500 has increased by 19.8%, indicating underperformance relative to the broader market [3] - The company's earnings outlook is crucial for investors, as it includes current consensus earnings expectations for upcoming quarters and any recent changes to these expectations [4] - The current consensus EPS estimate for the upcoming quarter is -$1.25 on revenues of $147.36 million, and for the current fiscal year, it is -$6.29 on revenues of $537.3 million [7] 分组3 - The Zacks Industry Rank places the Medical - Biomedical and Genetics sector in the top 38% of over 250 Zacks industries, suggesting a favorable outlook for stocks within this industry [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors or through tools like the Zacks Rank [5][6]

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]