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 ...

Ultragenyx (RARE) came out with a quarterly loss of $1.52 per share versus the Zacks Consensus Estimate of a loss of $1.64. This compares to loss of $2.25 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of 7.32%. A quarter ago, it was expected that this biotechnology company would post a loss of $1.72 per share when it actually produced a loss of $2.03, delivering a surprise of -18.02%. Over the last four quarters, the company h ...

Zacks Consensus Estimate The stock might move higher if these key numbers top expectations in the upcoming earnings report. On the other hand, if they miss, the stock may move lower. This biotechnology company is expected to post quarterly loss of $1.64 per share in its upcoming report, which represents a year-over-year change of +27.1%. Estimate Revisions Trend Investors should keep in mind that the direction of estimate revisions by each of the covering analysts may not always get reflected in the aggrega ...

Ultragenyx Pharmaceutical (RARE) announced that it has reached alignment with the FDA regarding the phase III study design and endpoints for GTX-102, an antisense oligonucleotide for Angelman syndrome, following the completion of a successful end-of-phase II meeting with the regulatory body. The company expects to initiate the same by the end of 2024. Per RARE, the pivotal phase III sham-controlled study will evaluate the safety and primary efficacy of GTX102 in approximately 120 Angelman syndrome patients, ...

NOVATO, Calif., June 13, 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 the pricing of its underwritten public offering of 7,435,898 shares of its common stock at a price to the public of $39.00 per share. In addition, in lieu of issuing common stock to certain investors, the company is offering pre-funded warrants to purcha ...

and, when available, its final prospectus supplement, and the documents incorporated by reference therein, including its Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 21, 2024, and its subsequent periodic reports filed with the Securities and Exchange Commission. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitati ...

For Ultragenyx, the consensus EPS estimate for the quarter has remained unchanged over the last 30 days. And a stock's price usually doesn't keep moving higher in the absence of any trend in earnings estimate revisions. So, make sure to keep an eye on RARE going forward to see if this recent jump can turn into more strength down the road. The stock rallied after the company, along with its partner Mereo BioPharma, announced new positive 14- month data from the phase II portion of the phase II/III Orbit stud ...

A registration statement relating to these securities has been filed with the Securities and Exchange Commission and became automatically effective on February 21, 2024. This offering is being made solely by means of a prospectus supplement and accompanying prospectus. When available, copies of the preliminary prospectus supplement and the accompanying prospectus related to the offering may be obtained from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, Ne ...