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]

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]

Financial Data and Key Metrics - Total revenue for Q2 2024 was $147 million, driven by strong performance across the commercial portfolio [12][13] - Crysvita contributed $114 million, with $67 million from North America, $40 million from Latin America and Turkey, and $6 million from Europe [13] - Dojolvi revenue was $19 million, Evkeeza revenue was $8 million, and Mepsevii revenue was $6 million in Q2 2024 [13] - Operating expenses were $263 million, including R&D expenses of $162 million, SG&A expenses of $81 million, and cost of sales of $21 million [13] - Net loss for Q2 2024 was $132 million or $1.52 per share [14] - Cash, cash equivalents, and marketable securities totaled $874 million as of June 30, 2024 [14] Business Line Performance - Crysvita demand in the US remained strong, with 60% of stock forms from adult patients and over 40 new prescribers in Q2 2024 [8] - In Latin America, Crysvita added approximately 60 new patients, totaling over 620 patients on reimbursed therapy since launch [9] - Dojolvi added approximately 30 start forms and 30 patients on reimbursed therapy in the US, with a split of 65% pediatric and 35% adult patients [10] - Evkeeza added approximately 60 new patients in the EMEA region in Q2 2024, with a successful launch in Japan [11] Market Performance - Latin America, particularly Brazil, continues to drive the majority of Crysvita revenue, with increasing contributions from Argentina and Mexico [9] - In Europe and the MENA region, Dojolvi revenue is driven by named patient sales requests, with approximately 215 patients treated across 12 countries [10] - Japan's Evkeeza launch gained meaningful traction following regulatory and pricing approvals, with 35 stock forms processed in Q2 2024 [11] Strategic Direction and Industry Competition - The company is advancing multiple late-stage programs, including UX-111 for Sanfilippo syndrome and GTX-102 for Angelman syndrome, with positive Phase III results for DTX401 and UX143 [5][6] - The company is on track to submit two BLAs (Biologics License Applications) by the end of 2024 and 2025, respectively [25] - The company is confident in its competitive position, particularly with GTX-102 for Angelman syndrome, citing superior long-term data and potency compared to competitors [46][53] Management Commentary on Operating Environment and Future Outlook - Management raised total revenue guidance for 2024 to $530 million - $550 million, reflecting strong performance across all products [14][15] - The company expects to file two BLAs, provide Phase III data for UX143, and advance the GTX-102 Phase III study over the next 12-18 months [25] - Management highlighted the importance of patient urgency in driving commercial success for gene therapies, particularly for GSDIa [58][59] Other Important Information - The company completed a successful end of Phase II meeting with the FDA for GTX-102, aligning on Phase III study design and key endpoints [6][19] - The company is evaluating life cycle management strategies for GTX-102, including the potential use of lumbar catheter-type devices to improve patient experience [33] - The company is monitoring the potential expiration of the rare pediatric disease voucher program, which could impact future rare disease drug development [74][75] Q&A Session Summary Question: Competitive landscape for Angelman syndrome and trial design for GTX-102 [26] - The company is confident in GTX-102's competitive position, citing superior long-term data and potency compared to competitors [46] - The Phase III trial design includes a reduction in loading doses from four to three, with cognition raw scores as the primary endpoint and expressive communication as a secondary endpoint [27] Question: Wilson disease data update and next steps [36] - The delay in the Wilson disease data update was due to longer-than-expected patient enrollment and the need for at least six months of data from the last patient [36] - The company is focused on biochemical markers, including copper levels, to assess the efficacy of the gene therapy [28] Question: Setrusumab's impact on pain and fracture rates in Osteogenesis Imperfecta (OI) patients [38][40] - Setrusumab has shown significant improvements in bone mineral density and fracture rates, with patients reporting reduced pain and increased activity levels [38][39] - The company expects that increased activity levels will further enhance bone strength, despite the potential for higher fracture risk [41] Question: GTX-102's Phase III trial design and FDA requirements [51] - The FDA agreed to a continuous variable analysis for the Bayley-4 endpoint, with no set threshold for success [52] - The company aims to replicate the Phase II results, which showed significant improvements in multiple domains, including cognition, communication, and behavior [53] Question: Setrusumab's Phase III enrollment and patient types [69][72] - The Phase III Orbit study enrolled more severe OI patients (Type 3 and 4) compared to the Phase II study, with a focus on capturing data across different age groups and disease types [70][72] - The study is well-powered to detect significant fracture reductions, with a target enrollment of 150 patients [72] Question: Rare pediatric disease voucher program and its impact on drug development [74] - The company has benefited from the rare pediatric disease voucher program, generating significant cash from the sale of two vouchers [75] - The company supports the reauthorization of the program, which has been instrumental in advancing rare disease drug development [76] Question: Potential competition from Amgen's romosozumab in OI [78] - The company believes its data for setrusumab is superior to Amgen's Phase II data for romosozumab, citing higher bone mineral density improvements and fracture reductions [79] - The company is confident in its competitive position, given the optimized dosing and superior efficacy of setrusumab [80]

Ultragenyx Pharmaceutical (RARE) incurred a second-quarter 2024 loss of $1.52 per share, narrower than the Zacks Consensus Estimate of a loss of $1.64. The company had reported a loss of $2.25 per share in the yearago quarter. Ultragenyx's total revenues amounted to $147 million in the reported quarter, up 36% year over year. The top line also beat the Zacks Consensus Estimate of $123 million on the back of higher product revenues. The company markets four drugs, namely Crysvita, Mepsevii, Dojolvi and Evkee ...

Ultragenyx (RARE) reported $147.03 million in revenue for the quarter ended June 2024, representing a yearover-year increase of 35.8%. EPS of -$1.52 for the same period compares to -$2.25 a year ago. The reported revenue compares to the Zacks Consensus Estimate of $123.42 million, representing a surprise of +19.13%. The company delivered an EPS surprise of +7.32%, with the consensus EPS estimate being -$1.64. While investors scrutinize revenue and earnings changes year-over-year and how they compare with Wa ...