Ultragenyx Reports Second Quarter 2024 Financial Results and Corporate Update
Ultragenyx reported second quarter 2024 financial results with total revenue of $147 million, up 36% YoY. Key revenue drivers were Crysvita ($114 million, +37%) and Dojolvi ($19 million, +17%). The company raised its 2024 revenue guidance to $530-$550 million from $500-$530 million.
Ultragenyx also disclosed significant clinical advancements including positive data from Phase 1/2 study in Angelman syndrome, Phase 2/3 study in osteogenesis imperfecta, and Phase 3 study in GSDIa. Phase 3 studies for Angelman syndrome and UX111 for Sanfilippo syndrome type A are on track to start by end of 2024.
The company reported a net loss of $132 million, down from $160 million YoY. Operating expenses were $263 million, including $39 million in non-cash stock-based compensation. Cash reserves stood at $874 million as of June 30, 2024.
Ultragenyx ha riportato i risultati finanziari del secondo trimestre del 2024 con un fatturato totale di 147 milioni di dollari, in aumento del 36% rispetto all'anno precedente. I principali motori di fatturato sono stati Crysvita (114 milioni di dollari, +37%) e Dojolvi (19 milioni di dollari, +17%). L'azienda ha rivisto al rialzo le stime di fatturato per il 2024 a 530-550 milioni di dollari, rispetto ai 500-530 milioni di dollari precedentemente annunciati.
Ultragenyx ha anche rivelato avanzamenti clinici significativi, inclusi dati positivi dallo studio di Fase 1/2 nella sindrome di Angelman, dallo studio di Fase 2/3 nell'osteogenesi imperfetta, e dallo studio di Fase 3 nella GSDIa. Gli studi di Fase 3 per la sindrome di Angelman e UX111 per la sindrome di Sanfilippo di tipo A sono previsti per iniziare entro la fine del 2024.
L'azienda ha riportato una perdita netta di 132 milioni di dollari, in calo rispetto ai 160 milioni di dollari dell'anno precedente. Le spese operative sono ammontate a 263 milioni di dollari, inclusi 39 milioni di dollari per compensi in azioni non monetari. Le riserve di liquidità ammontavano a 874 milioni di dollari al 30 giugno 2024.
Ultragenyx presentó los resultados financieros del segundo trimestre de 2024 con ingresos totales de 147 millones de dólares, un aumento del 36% interanual. Los principales impulsores de ingresos fueron Crysvita (114 millones de dólares, +37%) y Dojolvi (19 millones de dólares, +17%). La empresa elevó su guía de ingresos para 2024 a 530-550 millones de dólares, desde 500-530 millones de dólares.
Ultragenyx también reveló avances clínicos importantes, incluidos datos positivos de un estudio de Fase 1/2 en síndrome de Angelman, un estudio de Fase 2/3 en osteogénesis imperfecta y un estudio de Fase 3 en GSDIa. Se espera que los estudios de Fase 3 para el síndrome de Angelman y UX111 para el síndrome de Sanfilippo tipo A comiencen para finales de 2024.
La compañía reportó una Pérdida neta de 132 millones de dólares, en comparación con los 160 millones de dólares interanuales. Los gastos operativos fueron de 263 millones de dólares, incluidos 39 millones de dólares en compensación en acciones no monetarias. Las reservas de efectivo eran de 874 millones de dólares al 30 de junio de 2024.
Ultragenyx는 2024년 2분기 재무 결과를 발표했으며, 총 수익은 1억 4,700만 달러로 전년 대비 36% 증가했습니다. 주요 수익 원인은 Crysvita (1억 1,400만 달러, +37%)와 Dojolvi (1,900만 달러, +17%)입니다. 회사는 2024년 수익 전망을 5억 3천만-5억 5천만 달러로 상향 조정했으며, 이전 전망은 5억-5억 3천만 달러였습니다.
Ultragenyx는 중요한 임상 발전도 밝혔으며, 안젤맨 증후군에 대한 1/2상 연구, 골형성이상증에 대한 2/3상 연구, 그리고 GSDIa에 대한 3상 연구에서 긍정적인 데이터를 포함하고 있습니다. 안젤맨 증후군 및 산필리포 증후군 A형에 대한 UX111의 3상 연구는 2024년 말까지 시작될 예정입니다.
회사는 1억 3,200만 달러의 순손실을 보고했으며, 이는 전년 대비 1억 6,000만 달러에서 감소한 것입니다. 운영 비용은 2억 6,300만 달러이며, 이에는 비현금 주식 보상으로 3,900만 달러가 포함됩니다. 2024년 6월 30일 기준 현금 보유액은 8억 7,400만 달러입니다.
Ultragenyx a publié les résultats financiers du deuxième trimestre 2024, avec un chiffre d'affaires total de 147 millions de dollars, en hausse de 36 % par rapport à l'année précédente. Les principaux moteurs de revenus étaient Crysvita (114 millions de dollars, +37 %) et Dojolvi (19 millions de dollars, +17 %). La société a relevé ses prévisions de chiffre d'affaires pour 2024 à 530-550 millions de dollars, contre 500-530 millions de dollars précédemment.
Ultragenyx a également divulgué des avancées cliniques significatives, y compris des données positives d'une étude de Phase 1/2 sur le syndrome d'Angelman, d'une étude de Phase 2/3 sur l'ostéogenèse imparfaite et d'une étude de Phase 3 sur la GSDIa. Les études de Phase 3 pour le syndrome d'Angelman et UX111 pour le syndrome de Sanfilippo de type A devraient débuter d'ici la fin de 2024.
La société a annoncé une perte nette de 132 millions de dollars, en baisse par rapport à 160 millions de dollars l'année précédente. Les dépenses d'exploitation se sont élevées à 263 millions de dollars, dont 39 millions de dollars de rémunération en actions non monétaires. Les réserves de liquidités s'élevaient à 874 millions de dollars au 30 juin 2024.
Ultragenyx berichtete über die finanziellen Ergebnisse des zweiten Quartals 2024 mit einem Gesamtumsatz von 147 Millionen Dollar, was einem Anstieg von 36% im Vergleich zum Vorjahr entspricht. Die Hauptumsatztreiber waren Crysvita (114 Millionen Dollar, +37%) und Dojolvi (19 Millionen Dollar, +17%). Das Unternehmen hat die Umsatzprognose für 2024 auf 530-550 Millionen Dollar erhöht, von zuvor 500-530 Millionen Dollar.
Ultragenyx gab auch bedeutende klinische Fortschritte bekannt, darunter positive Daten aus der Phase 1/2-Studie zur Angelman-Syndrom, der Phase 2/3-Studie zur Osteogenesis imperfecta und der Phase 3-Studie zur GSDIa. Die Phase-3-Studien für das Angelman-Syndrom und UX111 für das Sanfilippo-Syndrom Typ A sollen bis Ende 2024 beginnen.
Das Unternehmen berichtete von einem Nettiverlust von 132 Millionen Dollar, ein Rückgang im Vergleich zu 160 Millionen Dollar im Vorjahr. Die Betriebskosten beliefen sich auf 263 Millionen Dollar, einschließlich 39 Millionen Dollar an nicht zahlungswirksamen aktienbasierten Vergütungen. Die Barmittel betrugen zum 30. Juni 2024 874 Millionen Dollar.
- Total revenue increased by 36% YoY to $147 million in Q2 2024.
- Raised 2024 revenue guidance to $530-$550 million.
- Crysvita revenue grew by 37% YoY to $114 million.
- Dojolvi revenue grew by 17% YoY to $19 million.
- Positive clinical data from Phase 1/2 studies in Angelman syndrome and Phase 2/3 studies in osteogenesis imperfecta.
- Net loss reduced to $132 million from $160 million YoY.
- Net loss of $132 million in Q2 2024.
- Total operating expenses increased to $263 million.
Insights
Ultragenyx's Q2 2024 results demonstrate strong financial performance, with total revenue reaching
However, it's important to note that despite the revenue growth, Ultragenyx is still operating at a loss. The company reported a net loss of
The company's cash position remains strong at
Ultragenyx's pipeline progress is impressive, with several key developments across multiple rare disease programs. The UX143 (setrusumab) for Osteogenesis Imperfecta showed particularly promising results, with a
The company's gene therapy programs are also advancing well. The GTX-102 for Angelman syndrome is moving to Phase 3, with FDA alignment on the study design. This is a significant milestone, as Angelman syndrome currently has no approved treatments. The positive developments with UX111 for Sanfilippo syndrome type A, including the FDA's agreement on using CSF heparan sulfate as a surrogate endpoint, could potentially accelerate the path to market.
The positive results from the Phase 3 study of DTX401 for Glycogen Storage Disease Type Ia are particularly noteworthy. The statistically significant reduction in daily cornstarch intake while maintaining glucose control could significantly improve patients' quality of life if approved. These advancements across multiple rare disease indications position Ultragenyx well in the orphan drug market, although investors should be aware that the path to approval and commercialization in rare diseases can be complex and time-consuming.
Second quarter total revenue of
Increased 2024 expected total revenue guidance to
NOVATO, Calif., Aug. 01, 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 reported its financial results for the quarter ended June 30, 2024.
“Our strong financial performance in the second quarter was driven by growing revenue across our commercial therapies from increasing global demand, leading us to raise our total revenue guidance for this year,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “In the quarter, we also reported positive data from our Phase 1/2 study in Angelman syndrome, our Phase 2/3 study in osteogenesis imperfecta, and our Phase 3 study in GSDIa. We are in an excellent position to achieve additional key milestones in the second half of the year including initiating our Phase 3 Angelman study and filing for accelerated approval for UX111 in Sanfilippo syndrome type A.”
Second Quarter 2024 Selected Financial Data Tables and Financial Results
Revenues (dollars in thousands), (unaudited) | |||||||||||||||
Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||||||
Crysvita | |||||||||||||||
Product sales | $ | 40,449 | $ | 16,884 | $ | 76,690 | $ | 38,118 | |||||||
Revenue in Profit-Share Territory | 67,045 | 61,314 | 107,447 | 111,220 | |||||||||||
Royalty revenue in European Territory | 6,176 | 4,816 | 12,118 | 9,698 | |||||||||||
Total Crysvita Revenue | 113,670 | 83,014 | 196,255 | 159,036 | |||||||||||
Dojolvi | 19,355 | 16,491 | 35,717 | 30,794 | |||||||||||
Mepsevii | 6,145 | 8,439 | 12,756 | 16,919 | |||||||||||
Evkeeza | 7,856 | 365 | 11,131 | 577 | |||||||||||
Daiichi Sankyo | — | — | — | 1,479 | |||||||||||
Total revenues | $ | 147,026 | $ | 108,309 | $ | 255,859 | $ | 208,805 | |||||||
Total Revenues
Ultragenyx reported
Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||||||||||
Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||||||
Total revenues | $ | 147,026 | $ | 108,309 | $ | 255,859 | $ | 208,805 | |||||||
Operating expenses: | |||||||||||||||
Cost of sales | 21,280 | 9,914 | 38,813 | 22,171 | |||||||||||
Research and development | 161,503 | 164,949 | 339,990 | 330,647 | |||||||||||
Selling, general and administrative | 80,604 | 81,403 | 158,764 | 158,049 | |||||||||||
Total operating expenses | 263,387 | 256,266 | 537,567 | 510,867 | |||||||||||
Net loss | $ | (131,598 | ) | $ | (159,828 | ) | $ | (302,282 | ) | $ | (323,800 | ) | |||
Net loss per share, basic and diluted | $ | (1.52 | ) | $ | (2.25 | ) | $ | (3.54 | ) | $ | (4.58 | ) | |||
Operating Expenses
Total operating expenses for the second quarter of 2024 were
Net Loss
For the second quarter of 2024, Ultragenyx reported net loss of
Net Cash Used in Operations and Cash Balance
For the three months ended June 30, 2024, net cash used in operations was
2024 Full Year Financial Guidance
- Total revenue guidance increased to be in the range of
$530 million to$550 million (previously$500 million to$530 million ) - Crysvita revenue expected to be towards the upper end of the range of
$375 million to$400 million . This includes all regions where Ultragenyx will recognize revenue: product sales in Latin America and Turkey, royalties in Europe, which have been ongoing, and royalties in North America, which began in April 2023. - Dojolvi revenue in the range of
$75 million to$80 million - Net Cash Used in Operations less than
$400 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): 14-month data resulted in a large, sustained
Positive 14-month results from the Phase 2 portion of the ongoing Phase 2/3 Orbit study demonstrated that, as of the May 24, 2024 data cut-off date, treatment with setrusumab continued to significantly reduce incidence of fractures in patients with OI. Treatment with setrusumab also resulted in ongoing and meaningful improvements in lumbar spine bone mineral density (BMD) at month 12 without evidence of plateau.
The median annualized rate of radiologically confirmed fractures across all 24 patients in the 2 years prior to treatment was 0.72. Following a mean treatment duration period of 16 months, the median annualized fracture rate was reduced
As of the data cut-off, there were no treatment-related serious adverse events observed in the study and there were no reported hypersensitivity reactions related to setrusumab.
More detailed 14-month data will be presented at a future scientific meeting.
GTX-102 antisense oligonucleotide for Angelman syndrome: Successful End-of-Phase 2 (EOP2) meeting with Food and Drug Administration (FDA); on track to initiate Phase 3 by the end of the year
In July 2024, Ultragenyx completed a successful EOP2 meeting with the FDA supporting the pivotal Phase 3 Aspire study design, which will be a global, randomized, double-blind, sham-controlled trial and will include a 48-week primary efficacy analysis period enrolling approximately 120 patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The primary endpoint will be improvement in cognition assessed by Bayley-4 cognitive raw score. The key secondary endpoint will be the Multi-domain Responder Index (MDRI) across all five domains of cognition, receptive communication, behavior, gross motor function, and sleep. Individual secondary endpoints were also discussed and aligned on with the FDA for the domains of communication, behavior, motor function and sleep. Additionally, the company plans to initiate Aurora, an open-label clinical study, to evaluate the safety and efficacy of GTX-102 for the treatment of patients with other Angelman syndrome genotypes and in other age groups.
The company has also participated in a PRIME meeting with the European Medicines Agency, receiving acceptance of the overall Phase 3 study design, dosing and evaluations and has met with Japan’s Pharmaceuticals and Medical Devices Agency to inform and discuss its Phase 3 study design.
The company expects the pivotal Phase 3 Aspire study to start by the end of 2024 and the Aurora study to start in 2025.
UX701 AAV gene therapy for Wilson disease: Last patient in Cohort 3 dosed; expect interim Stage 1 data in the second half of 2024
All patients in the three dose-escalation cohorts of Stage 1 have been dosed. During Stage 1, the safety and efficacy of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2, which is the pivotal, randomized, placebo-controlled stage of the study. Data from Stage 1 are expected in the second half of 2024, which will be followed by dose selection and initiation of Stage 2.
UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval
In June 2024, Ultragenyx announced a successful meeting with the FDA during which the company reached agreement with the FDA that CSF HS is a reasonable surrogate endpoint that could support submission of a biologics license application, or BLA, seeking accelerated approval for UX111. As discussed with the FDA, the BLA filing will be based on the available data including from the ongoing pivotal Transpher A study evaluating the safety and efficacy of UX111 in children with MPS IIIA. The details of a BLA will be finalized with the FDA in a pre-BLA meeting that is expected to happen in the second half of 2024, with the intent to file the application late this year or early next year.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Positive top-line results from Phase 3 Study resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control
In May 2024, Ultragenyx announced positive topline results from the Phase 3 GlucoGene study for the treatment of patients aged eight years and older. The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48. The mean percent reduction was
Full 48 Week data from the Phase 3 study will be presented at a scientific conference later this year. These results will be discussed with regulatory authorities to support a marketing application in 2025.
DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients; expect enrollment to be completed in the second half of 2024
Ultragenyx is randomizing and dosing patients in the ongoing Phase 3 study. The pivotal, 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels. Enrollment is currently expected to be completed in the second half of 2024.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Thursday, August 1, 2024, at 2 p.m. PT/5 p.m. ET to discuss the second quarter 2024 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for one year.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercial supply of Crysvita, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, the transition back to Kyowa Kirin of our exclusive rights to promote Crysvita in the United States and Canada and unexpected costs, delays, difficulties or adverse impact to revenue related to such transition, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.
For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 3, 2024, and its subsequent periodic reports filed with the SEC.
In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/).
Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com
Ultragenyx Pharmaceutical Inc. | |||||||||||||||
Selected Statement of Operations Financial Data | |||||||||||||||
(in thousands, except share and per share amounts) | |||||||||||||||
(unaudited) | |||||||||||||||
Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||||||
Statement of Operations Data: | |||||||||||||||
Revenues: | |||||||||||||||
Product sales | $ | 73,805 | $ | 42,179 | $ | 136,294 | $ | 86,408 | |||||||
Royalty revenue | 73,221 | 46,331 | 119,565 | 51,213 | |||||||||||
Collaboration and license | — | 19,799 | — | 71,184 | |||||||||||
Total revenues | 147,026 | 108,309 | 255,859 | 208,805 | |||||||||||
Operating expenses: | |||||||||||||||
Cost of sales | 21,280 | 9,914 | 38,813 | 22,171 | |||||||||||
Research and development | 161,503 | 164,949 | 339,990 | 330,647 | |||||||||||
Selling, general and administrative | 80,604 | 81,403 | 158,764 | 158,049 | |||||||||||
Total operating expenses | 263,387 | 256,266 | 537,567 | 510,867 | |||||||||||
Loss from operations | (116,361 | ) | (147,957 | ) | (281,708 | ) | (302,062 | ) | |||||||
Change in fair value of equity investments | (3,991 | ) | 261 | (245 | ) | (73 | ) | ||||||||
Non-cash interest expense on liabilities for sales of future royalties | (15,960 | ) | (15,375 | ) | (31,807 | ) | (31,011 | ) | |||||||
Other income, net | 5,572 | 3,975 | 12,791 | 10,573 | |||||||||||
Loss before income taxes | (130,740 | ) | (159,096 | ) | (300,969 | ) | (322,573 | ) | |||||||
Provision for income taxes | (858 | ) | (732 | ) | (1,313 | ) | (1,227 | ) | |||||||
Net loss | $ | (131,598 | ) | $ | (159,828 | ) | $ | (302,282 | ) | $ | (323,800 | ) | |||
Net loss per share, basic and diluted | $ | (1.52 | ) | $ | (2.25 | ) | $ | (3.54 | ) | $ | (4.58 | ) | |||
Shares used in computing net loss per share, basic and diluted | 86,580,516 | 70,897,991 | 85,433,443 | 70,639,015 | |||||||||||
Ultragenyx Pharmaceutical Inc. Selected Activity included in Operating Expenses (in thousands) (unaudited) | |||||||||||||||
Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||||||
Non-cash stock-based compensation | $ | 39,363 | $ | 34,653 | $ | 76,297 | $ | 66,592 | |||||||
UX143 clinical milestone | — | $ | 9,000 | — | $ | 9,000 |
Ultragenyx Pharmaceutical Inc. | ||||||||
Selected Balance Sheet Financial Data | ||||||||
(in thousands) | ||||||||
(unaudited) | ||||||||
June 30, | December 31, | |||||||
2024 | 2023 | |||||||
Balance Sheet Data: | ||||||||
Cash, cash equivalents, and marketable debt securities | $ | 874,490 | $ | 777,110 | ||||
Working capital | 691,774 | 451,747 | ||||||
Total assets | 1,618,437 | 1,491,013 | ||||||
Total stockholders' equity | 432,418 | 275,414 |
FAQ
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