Burosumab-TWZA

Second quarter total revenue of $214 million, Crysvita® revenue of $156 million and Dojolvi® revenue of $27 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path to profitability in 2027 Catalysts in second half of 2026 include two PDUFA dates and pivotal data readout from GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif., Aug. 04, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended June 30, 2026 and reaffirmed its financial guidance for 2026. “In the second quarter we generated the highest quarterly revenue in the history of the company, supporting our full-year revenue guidance, and keeping us on track toward profitability in 2027,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “As we look to the second half of the year, we are entering a transformative period with multiple important catalysts. We are ready to launch two gene therapy products and are preparing for a pivotal Phase 3 GTX-102 data readout in Angelman syndrome. This puts us in position to broaden our patient impact with sustained growth in the years to come.” Second Quarter 2026 Revenue Highlights and 2026 Revenue Guidance Total revenue in the second quarter of 2026 was $214 million. The company reaffirms its full year 2026 total revenue guidance of $730 million to $760 million, which excludes revenue from potential new product launches. Crysvita revenue in the second quarter of 2026 was $156 million, consistent with expected seasonality in the U.S. and Canada and ordering patterns in Latin America. The company reaffirms its full year 2026 Crysvita revenue guidance of $500 million to $520 million. Dojolvi revenue in the second quarter 2026 was $27 million. The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million. Evkeeza® revenue in the second quarter 2026 was $21 million, driven by increased demand from new country launches and early access. Mepsevii® revenue in the second quarter 2026 was $10 million. Milestones and Upcoming Catalysts Dojolvi for the treatment of LC-FAOD: In May 2026, Dojolvi was listed on the National Health Insurance (NHI) drug price list and was launched in Japan following the receipt of manufacturing and marketing approval under the Conditional Approval System for Pharmaceuticals on March 23, 2026. DTX401 (pariglasgene brecaparvovec) AAV8 gene therapy for the treatment of glycogen storage disease type Ia (GSDIa): In February 2026, the U.S. Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) seeking approval of DTX401 as a treatment for GSDIa and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026. UX111 (rebisufligene etisparvovec) AAV9 gene therapy for the treatment of Sanfilippo syndrome type A (MPS IIIA): In April 2026, the FDA accepted for review the resubmitted BLA seeking accelerated approval for UX111 as a treatment for MPS IIIA and assigned a PDUFA action date of September 19, 2026. GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): The Phase 3 Aspire study, in patients with a full maternal UBE3A gene deletion, enrolled 129 patients, randomized 1:1 to GTX-102 or sham. Data from this study are expected in the September or October timeframe. Enrollment in the open-label Phase 2/3 Aurora study, evaluating GTX-102 in other genotypes and ages, began in October 2025 and is expected to complete in the second half of 2026. UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for the treatment of Wilson disease: Enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data from this stage are expected in the fourth quarter of 2026. UX016 novel prodrug for sialic acid used as a substrate replacement therapy for the treatment of GNE myopathy: The FDA cleared the Investigational New Drug (IND) application for UX016 and an externally funded Phase 1/2 study is expected to begin in the second half of 2026. DTX301 (avalotcagene ontaparvovec) AAV8 gene therapy for the treatment of Ornithine Transcarbamylase, or OTC, deficiency: The Phase 3 Enh3ance study continues with patients in both treatment and cross-over groups progressing through 64 weeks of follow-up. Data from the second primary endpoint, which evaluates reduction in treatment burden, including use of ammonia scavengers and dietary management, are expected in the first half of 2027. Summary of Second Quarter 2026 Financial Results Selected Financial Data (dollars in millions, except per share amounts), (unaudited) Operating Expenses Total operating expenses for the second quarter 2026 were $289 million, including $34 million of non-cash stock-based compensation. The company reaffirms its full year 2026 and 2027 guidance for combined R&D and SG&A operating expenses: compared to 2025, combined R&D and SG&A expenses in 2026 are expected to be flat to down low-single digits, and combined R&D and SG&A expenses in 2027 are expected to decrease by at least 15%. Net Loss Net loss for the second quarter 2026 was $92 million, or $0.90 per share basic and diluted, compared with a net loss for the second quarter 2025 of $115 million, or $1.17 per share basic and diluted. Cash Balance and Net Cash Used in Operations Cash, cash equivalents, and marketable securities were $436 million as of June 30, 2026. For the three months ended June 30, 2026, net cash used in operations was $97 million. Conference Call and Webcast Information Ultragenyx will host a conference call today, Tuesday, August 4, 2026, at 2 p.m. PT/5 p.m. ET to discuss the second quarter 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 three months. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare 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 regarding Ultragenyx’s expectations and projections concerning its future operating results and financial performance, including its 2026 revenue guidance for total revenue, Crysvita and Dojolvi, its anticipated R&D and SG&A expenses in 2026 and 2027, anticipated benefits and savings from its strategic restructuring plan, and the timing and sustainability of profitability; the timing, progress, results and plans for its clinical programs and studies, including the anticipated timing and outcome of the Phase 3 Aspire study of GTX-102, enrollment in the Aurora study, data from the UX701 and DTX301 studies, and initiation of the UX016 study; the FDA’s review of the BLAs for DTX401 and UX111, including the anticipated PDUFA action dates, the potential approval of either product candidate, whether the FDA may require additional information, studies or manufacturing changes, and the timing and outcome of regulatory inspections; Ultragenyx’s manufacturing and commercial readiness and the timing and success of any potential launches of DTX401 and UX111, if approved; and the potential patient impact, commercial opportunity and growth associated with Ultragenyx’s products and product candidates, 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 the company’s clinical development programs, commercial success of its products and product candidates, continued 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 commercialization of Crysvita in certain major markets, including the U.S. and Canada, and for our commercial supply of Crysvita in those markets, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, our ability to successfully manage the expansion of our company, delays or unexpected costs and other adverse effects related to the strategic restructuring plan, competition from other therapies or products, regulatory scrutiny of the company’s products and product candidates, the company’s limited experience as a company in operating its own manufacturing facility, market acceptance of our products, uncertainty related to insurance coverage and reimbursement, 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 candidate. 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 6, 2026, 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-/). Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com

iStock, ismagilov With the second quarter earnings period underway, BioSpace examines five biotechs with meaningful catalysts that analysts and investors are watching. Biotechs are heading into the second quarter earnings period ready to pressure their Big Pharma peers with new drug launches. More than ever, smaller biotechs are choosing to go at it alone to market their drugs, rather than partner up or sell. This has created a dynamic environment among these upstart companies, with plenty of eyes watching to see if they succeed. From Agios Pharmaceuticals to Ultragenyx, BioSpace takes a look at five biotechs with major catalysts to report on over the next few weeks. ezstandalone.cmd.push(function () { ezstandalone.showAds(129); }); Earnings Q2 earnings arrive as pharma picks up steam, buoyed by dealmaking, policy tailwinds Biopharma is entering its second-quarter earnings season riding high on a wave of massive deals and venture capital flow, plus a clearing of regulatory and policy overhangs. What can industry watchers expect to hear on the upcoming investor calls? July 15, 2026 · 6 min read · Tristan Manalac Read more Agios Pharmaceuticals Q2 stock performance: 35% Earnings date: July 30 After reporting the failure of a Phase 2 study for tebapivat in sickle cell disease, Agios’ management will have to show confidence in the biotech’s remaining pipeline when reporting second quarter earnings. Last week, Agios announced that the next-generation PK activator would be discontinued after failing to differentiate from the company’s own mitapivat or Novo Nordisk’s etavopivat. Analysts hadn’t given tebapivat a lot of credit heading into the readout, but the candidate served as a “narrative enricher for longer-term franchise durability,” according to Truist Securities. The failure of the candidate puts more “concentrated risk” on mitapivat, just as the drug class gets more competition from Novo Nordisk. Mitapivat is already approved as Pyrukynd for pyruvate kinase (PK) deficiency and Aqvesme for alpha- or beta-thalassemia. In the second quarter presentation, Truist will be watching for details on the thalassemia launch and the drug’s potential in sickle cell disease with tebapivat out of the running. The drug is under FDA review via the accelerated pathway for the indication with a decision date set for November 1. Arrowhead Pharmaceuticals FY3Q stock performance: 567% Earnings date: August 4 Arrowhead Pharmaceuticals has already begun its fiscal fourth quarter of 2026 on strong footing, with the announcement last week that two Phase 3 studies of plozasiran in severe hypertriglyceridemia (sHTG) were successful . The results sent the company’s shares up 20% that day to $91.48, while rival Ionis Pharmaceuticals dipped. Analysts had been anticipating the readout in the leadup to fiscal third quarter earnings. Beyond the outstanding results of those sHTG trials, analysts are watching for a second half readout for Arrowhead’s obesity programs , ARO-INHBE and ARO-ALK7. ARO-INHBE has already shown a doubling of the weight loss achieved by Eli Lilly’s tirzepatide in an interim readout from a small group of patients in two Phase 1/2a trials. “ARWR is ahead of peers across both the INHBE+ALK7 programs, w/ multiple paths forward depending on data,” Jefferies wrote in an earnings preview. Arrowhead is also expecting data for the Alzheimer’s disease therapy ARO-MAPT in the third quarter, which could help validate the company’s blood-brain barrier delivery tech. On the commercial side, Arrowhead celebrated its first ever approval late last year, when plozasiran was approved to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). As of the company’s last earnings call, the drug was “trending better” than Ionis’ rival product, according to Jefferies. The firm is expecting sales of about $6 million for the quarter. C-suite Caught In Sarepta ‘Downdraft,’ Arrowhead Becomes RNAi Knight in Shining Armor Sarepta’s troubles had nothing to do with Arrowhead’s assets, and yet both companies have seen their stock prices decline this past month. BioSpace caught up with Arrowhead’s Chris Anzalone to talk about the biotech’s role as an RNAi pipeline savior. August 12, 2025 · 7 min read · Annalee Armstrong Read more BridgeBio Q2 stock performance: 125% Earnings date: TBD BridgeBio is on track to deliver a strong second quarter report, as Attruby continues to steal market share in the crowded transthyretin amyloid cardiomyopathy (ATTR-CM) space, according to Jefferies. The drug is expected to make sales of $215 million for the quarter thanks to increases in front-line uptake, the firm predicted in a July 19 note to clients. This growth is expected to continue quarter-over-quarter going forward, which will in turn build investor confidence in BridgeBio’s next three planned launches in rare disease, Jefferies said. The company is working toward FDA approvals for BBP-418 in limb girdle muscular dystrophy type 2, infigratinib in alternating hemiplegia of childhood and encaleret in autosomal dominant hypocalcemia type 1. Infigratinib was approved in 2021 for cholangiocarcinoma and is marketed as Truseltiq. Each of these could mean $1 billion opportunities for BridgeBio, Jefferies noted, while Attruby is on track to deliver $3–$4 billion in peak sales in ATTR-CM. The drug faces competition from Pfizer’s Vyndaqel and Vyndamax, Alnylam’s Amvuttra and Ionis/AstraZeneca’s eplotersen in the space, but fears that BridgeBio will lose significant market share are subsiding as the market becomes more mature, according to Jefferies. Ionis Pharmaceuticals Q2 performance: 190% Earnings date: July 29 Ionis is well ahead in the marketplace with Tryngolza, which now has two approved indications in familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG) but is still facing immense pressure from Arrowhead. Analysts are expecting Ionis’ overall revenue to decline by more than half when the company reports earnings this week, as the price of the drug declines significantly to match the entry into a more common sHTG indication. Guggenheim analysts have revenue estimates above consensus at $209 million. This would mark a year-over-year decline of about 54% from last year, when revenue was $452 million . The slightly optimistic take as compared to consensus stems from higher than expected estimates for R&D collaboration revenue, since Recordati paid a $30 million upfront fee for a partnership with Ionis at the end of June. With the price reduction, Guggenheim has lowered its sales expectations to $2.9 billion as compared to $3.2 billion prior. But the analysts are optimistic that the dip for Tryngolza, which is now approved for FCS and sHTG, will be short lived as the sHTG label highlighted the therapy’s ability to reduce the risk of acute pancreatitis. Recent data also showed its ability to normalize hepatic fat. Moving forward, analysts will be focused on three upcoming Phase 3 readouts, including the Lp(a) HORIZON trial of pelacarsen, which is testing the drug for reducing lipoprotein(a). Rare diseases Ionis Paves Its Own Path as Initial Tryngolza Launch Defies Expectations Long an R&D company that partnered off assets, RNAi biotech Ionis Pharmaceuticals shifted in 2025 to bring two medicines to market alone. Analysts are already impressed—and there’s more to come in 2026. February 18, 2026 · 3 min read · Annalee Armstrong Read more Ultragenyx 2Q performance: -1.33% Earnings date: TBD Ultragenyx has been through the wringer this year, witha high pro for a bone disease drug and an FDA rejection for a rare disease gene therapy. Heading into the third quarter, analysts are keenly watching for any details on a Phase 3 readout for apazunersen in Angelman syndrome, which could help turn the ship around. Jefferies anticipates peak worldwide sales of about $1.87 billion for the drug in 2036 if ultimately approved. Ultragenyx executives have had to make some tough decisions following the events of 2025, including implementing an expense reduction plan and layoffs . They also sold off two priority review vouchers that were awarded with the recent approvals of rare disease therapies Mepsevii and Crysvita. The biotech is awaiting two key regulatory decisions. Pariglasgene brecaparvovec, an AAV8 gene therapy, is under review for glycogen storage disease type 1a with an FDA action date of August 23. And rebisufligene etisparvovec, an AAV9 gene therapy, was accepted for priority review by the FDA with a decision date of September 19. Jefferies believes that both launches could be successful because there is unmet need in both indications. As for sales, Jefferies predicts Crysvita to come in between $500 and $520 million while Dojolvi could take home $100–110 million.