Ultragenyx voucher sale would bring about $168 million, a few months' burn

Ultragenyx sells medicines for rare genetic diseases. Its biggest seller is Crysvita, which partner Kyowa Kirin sells in some big markets, including the United States. [R01][R01] Second quarter 2026 releasea biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseasesUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R03][R03] Second quarter 2026 releaseCrysvita revenue in the second quarter of 2026 was $156 millionUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R13][R13] Second quarter 2026 release, risksdependence on Kyowa Kirin for the commercialization of Crysvita in certain major marketsUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm Revenue in the second quarter was $214 million, a company record. That was up 28% from a year earlier, and the company expects $730 million to $760 million for 2026. [R02][R02] Second quarter 2026 releaseTotal 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 millionUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R14][R14] Second quarter 2026 releaseIn the second quarter we generated the highest quarterly revenue in the history of the companyUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R09][R09] Second quarter 2026 release, revenue tableTotal revenues $214 $167Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm It still loses money. It lost $92 million in the second quarter and $575 million in 2025. [R05][R05] Second quarter 2026 releaseNet loss for the second quarter 2026 was $92 millionUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [T04][T04] Form 10-K 2025, statement of operationsNet loss $ (575)Ultragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm

In August, regulators gave GENGLYCOS an early approval, called accelerated approval. More study is still required. [G01][G01] Form 8-K, August 19, 2026granted accelerated approval for GENGLYCOSUltragenyx Form 8-K on the accelerated approval of GENGLYCOS, filed August 19, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526357185/rare-20260819.htm [G02][G02] Form 8-K, August 19, 2026Ultragenyx has agreed to provide two years of safety and efficacy clinical data from open-label commercial treatment of 50 patients and 20 control patientsUltragenyx Form 8-K on the accelerated approval of GENGLYCOS, filed August 19, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526357185/rare-20260819.htm It treats a rare disorder called glycogen storage disease type Ia. The approval earned Ultragenyx a voucher. [G03][G03] Form 8-K, August 19, 2026also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type IaUltragenyx Form 8-K on the accelerated approval of GENGLYCOS, filed August 19, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526357185/rare-20260819.htm [K04][K04] Form 8-K, Item 1.01The Company was awarded the PRV by the U.S. Food and Drug Administration ("FDA") on August 19, 2026 upon approval of GENGLYCOSUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm On September 2, its late-stage trial of apazunersen in Angelman syndrome missed its main goal. Reuters reported that the shares fell 45% in after-hours trading. [A01][A01] Form 8-K, September 2, 2026The study did not achieve the primary endpointUltragenyx Form 8-K on the Phase 3 Aspire results in Angelman syndrome, filed September 2, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526380165/rare-20260902.htm [A04][A04] Reuters, September 2, 2026sending its shares down 45% in aftermarket tradingReuters · Read October 7, 2026Open source ↗https://www.reuters.com/business/healthcare-pharmaceuticals/ultragenyxs-rare-disease-drug-fails-late-stage-trial-shares-plunge-2026-09-02/ On September 17, FAYUVI won full approval. It is a gene therapy for Sanfilippo syndrome type A, and it earned the company a second voucher. [V01][V01] Form 8-K, September 17, 2026granted standard full approval of FAYUVIUltragenyx Form 8-K on the approval of FAYUVI, filed September 17, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm [V04][V04] Form 8-K, September 17, 2026also known as UX111, for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A)Ultragenyx Form 8-K on the approval of FAYUVI, filed September 17, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm [V05][V05] Form 8-K, September 17, 2026the second gene therapy approval for UltragenyxUltragenyx Form 8-K on the approval of FAYUVI, filed September 17, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm [V02][V02] Form 8-K, September 17, 2026The Company received a Priority Review Voucher upon this approval.Ultragenyx Form 8-K on the approval of FAYUVI, filed September 17, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm

A priority review voucher is a reward for getting a drug approved for a rare childhood disease. The owner can use it to speed up the review of a different drug, or sell it. [X04][X04] Ultragenyx release, About the programa sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a PRV that can be redeemed to receive a priority review of a subsequent marketing application for a different productGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html [X05][X05] Ultragenyx release, About the programThe sponsor receives the PRV upon approval of the rare pediatric disease product application, and it can be sold or transferredGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html Three other voucher sales this year went for $205 million, $180 million and $195 million. The sellers were Cyprium, a unit of Fortress Biotech, and Rocket and Denali. [W01][W01] Fortress Biotech release, March 30, 2026Priority Review Voucher (“PRV”) for gross proceeds of $205 millionGlobeNewswire (Fortress Biotech) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/03/30/3265013/0/en/fortress-biotech-s-subsidiary-cyprium-therapeutics-closes-sale-of-rare-pediatric-disease-priority-review-voucher-for-205-million.html [W02][W02] Rocket Pharmaceuticals release, April 28, 2026Rocket has entered into a definitive agreement to sell its PRV for $180 millionBusiness Wire (Rocket Pharmaceuticals) · Read October 7, 2026Open source ↗https://www.businesswire.com/news/home/20260428133676/en/Rocket-Pharmaceuticals-Announces-%24180-Million-Sale-of-Priority-Review-Voucher [W03][W03] Denali Therapeutics release, June 18, 2026to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $195 millionGlobeNewswire (Denali Therapeutics) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/06/18/3314101/0/en/denali-therapeutics-enters-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-195-million.html

On October 6, Ultragenyx agreed to sell its GENGLYCOS voucher. The buyer will pay $210 million in cash when the deal closes. [K09][K09] Form 8-K, Item 1.01On October 6, 2026, Ultragenyx Pharmaceutical Inc.Ultragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [K01][K01] Form 8-K, Item 1.01the Company agreed to sell a Rare Pediatric Disease Priority Review VoucherUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [K04][K04] Form 8-K, Item 1.01The Company was awarded the PRV by the U.S. Food and Drug Administration ("FDA") on August 19, 2026 upon approval of GENGLYCOSUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [K02][K02] Form 8-K, Item 1.01the buyer agreed to pay the Company $210 million, payable in cash, upon the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm The NIH is the government's medical research agency. Under a 2018 patent license, it gets 20% of the price after closing. That is about $42 million, which leaves Ultragenyx about $168 million. [T05][T05] Form 10-K 2025, NIH licenseWe have a non-exclusive license from the National Institutes of Health, or NIH, to an issued U.S. patent expiring in 2034Ultragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm [K08][K08] Form 8-K, Item 1.01Pursuant to the terms of a patent license agreement with the U.S. Department of Health (the"NIH") dated December 10, 2018Ultragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [K03][K03] Form 8-K, Item 1.0120% of the gross proceeds from the sale of the PRV will be payable by the Company to the NIH after the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm That figure is before fees to Jefferies, its adviser on the sale, and before any taxes. [X03][X03] Ultragenyx release, October 7, 2026Jefferies LLC is serving as exclusive financial advisorGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html

The buyer gets the right to a faster review of a different drug, one of its own. Ultragenyx gets cash without selling new shares. Its finance chief called it non-dilutive capital. [X04][X04] Ultragenyx release, About the programa sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a PRV that can be redeemed to receive a priority review of a subsequent marketing application for a different productGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html [X01][X01] Ultragenyx release, October 7, 2026Monetizing this PRV provides significant non-dilutive capital to advance our efforts to bring forward first-ever therapies for rare and ultra-rare diseases, and supports our path to profitabilityGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html The company says the money will help it develop new rare-disease drugs and supports its path to profit. It added that cash from an earlier voucher sale helped pay for GENGLYCOS itself. [X01][X01] Ultragenyx release, October 7, 2026Monetizing this PRV provides significant non-dilutive capital to advance our efforts to bring forward first-ever therapies for rare and ultra-rare diseases, and supports our path to profitabilityGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html [X02][X02] Ultragenyx release, October 7, 2026GENGLYCOS itself benefited from capital generated from a previous PRV saleGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html

The deal carries standard promises and protections, with some limits. The full agreement will be filed with the 2026 annual report. [K06][K06] Form 8-K, Item 1.01contains customary representations, warranties, covenants, and indemnification provisions subject to certain limitationsUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [K07][K07] Form 8-K, Item 1.01a copy of which will be filed with the Company's Annual Report on Form 10-K for the year ended December 31, 2026Ultragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm Closing depends on standard conditions, including the end of a waiting period under federal antitrust law. Ultragenyx still has its second voucher, from the FAYUVI approval. [K05][K05] Form 8-K, Item 1.01The transaction remains subject to customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976Ultragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [V02][V02] Form 8-K, September 17, 2026The Company received a Priority Review Voucher upon this approval.Ultragenyx Form 8-K on the approval of FAYUVI, filed September 17, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm

Ultragenyx had $436 million in cash and securities on June 30. It had $737 million at the end of 2025. [R08][R08] Second quarter 2026 release, balance sheet tableCash, cash equivalents, and marketable securities $436 $737Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R04][R04] Second quarter 2026 releaseCash, 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.Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm In the first half, its operations used $294 million of cash. That is about $147 million a quarter. The pace slowed to $97 million in the second quarter, after about $197 million in the first. [Q03][Q03] Form 10-Q, LiquidityCash used in operating activities for the six months ended June 30, 2026 was $294 millionUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [R04][R04] Second quarter 2026 releaseCash, 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.Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm First-half cash use included one-time items: 2025 bonus payouts and costs from stopping UX143 manufacturing. At the second-quarter pace, the voucher cash lasts about five months. At the first-half pace, it lasts about 3.4 months. [Q22][Q22] Form 10-Q, Liquiditydecreases in accrued manufacturing due to payment or settlement of accrued costs related to stoppage of manufacturing for UX143 and the payout of 2025 annual bonusesUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [Q03][Q03] Form 10-Q, LiquidityCash used in operating activities for the six months ended June 30, 2026 was $294 millionUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [K02][K02] Form 8-K, Item 1.01the buyer agreed to pay the Company $210 million, payable in cash, upon the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [K03][K03] Form 8-K, Item 1.0120% of the gross proceeds from the sale of the PRV will be payable by the Company to the NIH after the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm

Part of the loss uses no cash. In the second quarter, it included $34 million of stock pay and $22 million of non-cash interest on royalty deals. [R12][R12] Second quarter 2026 releaseTotal operating expenses for the second quarter 2026 were $289 million, including $34 million of non-cash stock-based compensationUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R20][R20] Second quarter 2026 release, results tableNon-cash interest expense on liabilities for sales of future royalties (22)Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm Some items will not repeat. The first half carried $30 million of restructuring costs. The company also said revenue growth came partly from when shipments went out. [R21][R21] Second quarter 2026 release, operating expenses tableRestructuring expense - - 30 -Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [Q23][Q23] Form 10-Q, Results of OperationsThe increase in revenue was largely driven by an increase in demand for our approved products, as well as timing of shipmentsUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm In 2025, $112 million of Crysvita royalty revenue was non-cash, because those royalties go to the buyers of the royalty deals. The voucher gain will be a one-time item too. [T10][T10] Form 10-K 2025, cash flow statementNon-cash royalty revenue (112) (101) (69)Ultragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm [Q24][Q24] Form 10-Q, Note 2for certain royalty payments from net sales of Crysvita in applicable territories that were sold to RPI and OMERS, the Company records the non-cash royalty revenueUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [K02][K02] Form 8-K, Item 1.01the buyer agreed to pay the Company $210 million, payable in cash, upon the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm

Bills due soon include $13 million of lease payments within a year. [Q21][Q21] Form 10-Q, Contractual ObligationsFuture minimum lease payments under non-cancellable leases as of June 30, 2026, were approximately $36 million, of which $13 million is due within one yearUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm FAYUVI began as UX111, which Ultragenyx licensed from Abeona. Ultragenyx owes Abeona royalties of up to 10% of sales and milestone payments of up to $30 million that depend on approval. [V04][V04] Form 8-K, September 17, 2026also known as UX111, for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A)Ultragenyx Form 8-K on the approval of FAYUVI, filed September 17, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm [T07][T07] Form 10-K 2025, Note 8, AbeonaIn May 2022, the Company announced an exclusive License Agreement for the AAV gene therapy for UX111 with AbeonaUltragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm [T06][T06] Form 10-K 2025, Note 8, Abeonathe Company is obligated to pay tiered royalties of up to 10% on net sales and commercial milestone payments of up to $30.0 million contingent upon regulatory approval of the productUltragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm FAYUVI was approved on September 17, so those payments may now be coming due, but the filings do not say when. Nationwide Children's Hospital is also owed up to $1 million in milestones and low single-digit royalties. [V01][V01] Form 8-K, September 17, 2026granted standard full approval of FAYUVIUltragenyx Form 8-K on the approval of FAYUVI, filed September 17, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm [T06][T06] Form 10-K 2025, Note 8, Abeonathe Company is obligated to pay tiered royalties of up to 10% on net sales and commercial milestone payments of up to $30.0 million contingent upon regulatory approval of the productUltragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm [T08][T08] Form 10-K 2025, Note 8, Abeonathe Company is obligated to pay up to $1 million contingent upon achievement of development and regulatory milestones as well as royalties in the low single-digits of net salesUltragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm

In past years, Ultragenyx sold slices of its future Crysvita royalties for upfront cash. The buyers were Royalty Pharma and OMERS, an Ontario pension plan. [Q16][Q16] Form 10-Q, Note 2The Company sold the right to receive certain royalty payments from net sales of Crysvita in certain territories to RPI Finance Trust, or RPI, an affiliate of Royalty Pharma, and to OCM LS23 Holdings LP, an investment vehicle for Ontario Municipal Employees Retirement System, or OMERSUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm On the June 30 balance sheet, those deals are about $1.2 billion of liabilities, out of $1.55 billion in total. Each deal ends once its cap is reached. [Q04][Q04] Form 10-Q, balance sheetLiabilities for sales of future royalties 76 69Ultragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [Q05][Q05] Form 10-Q, balance sheetLiabilities for sales of future royalties 1,126 1,147Ultragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [Q12][Q12] Form 10-Q, balance sheetTotal liabilities 1,548 1,605Ultragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [Q06][Q06] Form 10-Q, Note 8will automatically terminate and the payments or royalties will cease once the aggregate payments as specified in the agreement, or the CAP, are reachedUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm From January 2028, OMERS also gets an extra 25% of the royalties from the United States and Canada. Under that 2025 deal, OMERS will keep taking 30% of those Crysvita sales after the 2022 deal's $725 million cap is reached. [Q07][Q07] Form 10-Q, Note 7In November 2025, the Company sold to OMERS its right to an additional 25% of the future royalty payments, beginning in January 2028Ultragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [Q25][Q25] Form 10-Q, Note 8OMERS will continue to receive 30% of Crysvita net sales in the U.S. and Canada following the achievement of $725 million in aggregate payments under the 2022 OMERS AgreementUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm

Ultragenyx can buy back the 2025 stake for $540 million until November 2027. That is more than the $436 million in cash on June 30, so the buyback looks out of reach. [Q26][Q26] Form 10-Q, Note 8OMERS granted the Company an option to repurchase the entire purchased interest, that expires in November 2027, for 1.35 times the purchase price, or $540 millionUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [R04][R04] Second quarter 2026 releaseCash, 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.Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm Shareholders' equity was negative $291 million on June 30. The company loses money before interest, so debt-to-earnings ratios do not apply. [R11][R11] Second quarter 2026 release, balance sheet tableTotal stockholders' deficit (291)Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [Q20][Q20] Form 10-Q, statement of operationsLoss from operations (75)Ultragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm

There were 98.6 million shares on July 31. Counting pre-funded warrants, the loss per share used 101.9 million. [Q01][Q01] Form 10-Q, cover pageAs of July 31, 2026, the registrant had 98,588,873 shares of common stock issued and outstandingUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [R10][R10] Second quarter 2026 release, share countShares used in computing net loss per share, basic and diluted 101.9Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [Q17][Q17] Form 10-Q, Note 10weighted-average number of shares of common stock outstanding, pre-funded warrants, and treasury stockUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm Options and stock awards cover up to 18.2 million more shares. About 8.9 million of those were options with an average exercise price of $60.87 at the end of 2025, about four times the October 5 price. So most near-term dilution would come from about 6.6 million restricted and performance stock units. [Q08][Q08] Form 10-Q, Note 10Options to purchase common stock, restricted stock units, and performance stock units 18.2Ultragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [T11][T11] Form 10-K 2025, Note 11, optionsOutstanding - December 31, 2025 8.9 60.87Ultragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm [P02][P02] IEX closing priceRARE closed at $14.52 on October 5, 2026 (IEX)IEX closing prices for RARE and XBI, September 15 to October 5, 2026Open source ↗https://iextrading.com/trading/market-data/ [T12][T12] Form 10-K 2025, Note 11, RSUsRSUs cancelled (0.7) 49.78 Unvested - December 31, 2025 5.9Ultragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm [T13][T13] Form 10-K 2025, Note 11, PSUsPSUs cancelled (0.1) 49.95 Unvested - December 31, 2025 0.7Ultragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm Ultragenyx can sell up to $350 million of stock into the market over time. It has raised $80 million that way, which leaves roughly $270 million, and it sold none in the first half of 2026. [Q10][Q10] Form 10-Q, Liquiditythe Company may offer and sell shares of the Company's common stock having an aggregate offering proceeds up to $350 millionUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [Q09][Q09] Form 10-Q, LiquidityTo date, we have sold 2.2 million shares under the ATM for net proceeds of $80 millionUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [Q18][Q18] Form 10-Q, LiquidityNo shares were sold under the ATM during the three and six months ended June 30, 2026Ultragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm Its quarterly report says it may need more money, but that its cash should last at least 12 months. The chief accounting officer sold 1,283 shares at $13.57 on September 15, about $17,000 worth, under a pre-set trading plan. That is too small to signal much. [Q13][Q13] Form 10-Q, LiquidityWe may require additional capital to fund our operationsUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [Q02][Q02] Form 10-Q, LiquidityWe believe that our existing capital resources will be sufficient to fund our projected operating requirements for at least the next 12 monthsUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [I01][I01] Form 4, Theodore Huizenga, filed September 15, 2026Common Stock 09/15/2026 S 1,283 D $13.57Form 4 for Theodore Huizenga, chief accounting officer, filed September 15, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526392233/xslF345X06/ownership.xml [I02][I02] Form 4, Theodore HuizengaX Check this box to indicate that a transaction was made pursuant to a contract, instruction or written plan for the purchase or sale of equity securities of the issuer that is intended to satisfy the affirmative defense conditions of Rule 10b5-1(c)Form 4 for Theodore Huizenga, chief accounting officer, filed September 15, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526392233/xslF345X06/ownership.xml

The company's goal is a profit in 2027, and it repeated that goal after the trial failed. It expects research and selling costs in 2027 to fall at least 15% from 2025, when they were about $1.1 billion. [R07][R07] Second quarter 2026 releaseremain on path to profitability in 2027Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [A03][A03] Ultragenyx release, September 2, 2026This strong commercial foundation will support our pipeline, while continuing toward profitability in 2027.GlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/09/02/3355481/20739/en/ultragenyx-announces-phase-3-aspire-results-in-angelman-syndrome.html [R06][R06] Second quarter 2026 releasecombined R&D and SG&A expenses in 2027 are expected to decrease by at least 15%Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [T02][T02] Form 10-K 2025, statement of operationsResearch and development 750 698 648Ultragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm [T03][T03] Form 10-K 2025, statement of operationsSelling, general and administrative 349 321 310Ultragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm It cut about 10% of its workforce in February, before the trial result. On September 2 it said it would find big new cost cuts. These are goals, not results. [Q19][Q19] Form 10-Q, Funding RequirementsIn February 2026, we implemented a strategic restructuring plan designed to reduce operating expenses and focus resources on our highest value drivers, including a reduction in workforce of approximately 10%Ultragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [A02][A02] Form 8-K, September 2, 2026The Company will evaluate the apazunersen program in light of this outcome and make a decision on its disposition. The Company will also assess its planned operations to define and implement significant expense reductions, while supporting its growing commercial business.Ultragenyx Form 8-K on the Phase 3 Aspire results in Angelman syndrome, filed September 2, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526380165/rare-20260902.htm

The case for: the price beats the three other voucher sales this year, and no new shares are sold. The cash is about 12% of the company's market value of about $1.4 billion at the October 5 close. [K02][K02] Form 8-K, Item 1.01the buyer agreed to pay the Company $210 million, payable in cash, upon the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [W01][W01] Fortress Biotech release, March 30, 2026Priority Review Voucher (“PRV”) for gross proceeds of $205 millionGlobeNewswire (Fortress Biotech) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/03/30/3265013/0/en/fortress-biotech-s-subsidiary-cyprium-therapeutics-closes-sale-of-rare-pediatric-disease-priority-review-voucher-for-205-million.html [W02][W02] Rocket Pharmaceuticals release, April 28, 2026Rocket has entered into a definitive agreement to sell its PRV for $180 millionBusiness Wire (Rocket Pharmaceuticals) · Read October 7, 2026Open source ↗https://www.businesswire.com/news/home/20260428133676/en/Rocket-Pharmaceuticals-Announces-%24180-Million-Sale-of-Priority-Review-Voucher [W03][W03] Denali Therapeutics release, June 18, 2026to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $195 millionGlobeNewswire (Denali Therapeutics) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/06/18/3314101/0/en/denali-therapeutics-enters-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-195-million.html [X01][X01] Ultragenyx release, October 7, 2026Monetizing this PRV provides significant non-dilutive capital to advance our efforts to bring forward first-ever therapies for rare and ultra-rare diseases, and supports our path to profitabilityGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html [Q01][Q01] Form 10-Q, cover pageAs of July 31, 2026, the registrant had 98,588,873 shares of common stock issued and outstandingUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [P02][P02] IEX closing priceRARE closed at $14.52 on October 5, 2026 (IEX)IEX closing prices for RARE and XBI, September 15 to October 5, 2026Open source ↗https://iextrading.com/trading/market-data/ Revenue is growing, and in August the company said it was ready to launch two gene therapies. FAYUVI was set to ship to treatment centers within 30 to 60 days of approval. [R09][R09] Second quarter 2026 release, revenue tableTotal revenues $214 $167Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R15][R15] Second quarter 2026 releaseWe are ready to launch two gene therapy productsUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [V03][V03] Ultragenyx release, September 17, 2026commercial product is expected to be available to ship to Qualified Treatment Centers within 30-60 daysGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/09/17/3364357/20739/en/ultragenyx-announces-approval-of-fayuvi-gene-therapy-the-first-ever-fda-approved-treatment-for-sanfilippo-syndrome-type-a-mps-iiia.html

The case against, strongest first: this is one-time cash, not steady revenue. At the first-half pace it pays for only about 3.4 months of operations. [Q03][Q03] Form 10-Q, LiquidityCash used in operating activities for the six months ended June 30, 2026 was $294 millionUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [K02][K02] Form 8-K, Item 1.01the buyer agreed to pay the Company $210 million, payable in cash, upon the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [K03][K03] Form 8-K, Item 1.0120% of the gross proceeds from the sale of the PRV will be payable by the Company to the NIH after the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm The company had called the Angelman trial a key event for the second half of 2026. Now it must decide what to do with apazunersen. [R16][R16] Second quarter 2026 releaseCatalysts in second half of 2026 include two PDUFA dates and pivotal data readout from GTX-102 Phase 3 Aspire study for Angelman syndromeUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [A02][A02] Form 8-K, September 2, 2026The Company will evaluate the apazunersen program in light of this outcome and make a decision on its disposition. The Company will also assess its planned operations to define and implement significant expense reductions, while supporting its growing commercial business.Ultragenyx Form 8-K on the Phase 3 Aspire results in Angelman syndrome, filed September 2, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526380165/rare-20260902.htm A sale of the second voucher in 2027 could bring a one-time gain that helps the 2027 profit goal without the business itself turning a profit. The company has not said whether its goal counts voucher gains. [V02][V02] Form 8-K, September 17, 2026The Company received a Priority Review Voucher upon this approval.Ultragenyx Form 8-K on the approval of FAYUVI, filed September 17, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm [R07][R07] Second quarter 2026 releaseremain on path to profitability in 2027Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm Part of the Crysvita royalties is already sold, and OMERS takes more from 2028. GENGLYCOS still needs two years of data from 50 treated and 20 control patients. And the antitrust review could delay the cash. [Q07][Q07] Form 10-Q, Note 7In November 2025, the Company sold to OMERS its right to an additional 25% of the future royalty payments, beginning in January 2028Ultragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [G02][G02] Form 8-K, August 19, 2026Ultragenyx has agreed to provide two years of safety and efficacy clinical data from open-label commercial treatment of 50 patients and 20 control patientsUltragenyx Form 8-K on the accelerated approval of GENGLYCOS, filed August 19, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526357185/rare-20260819.htm [K05][K05] Form 8-K, Item 1.01The transaction remains subject to customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976Ultragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm

Dojolvi, another of its drugs, has mixed protection. A settlement keeps one generic maker, Esjay, from selling a copy until January 1, 2033, subject to customary exceptions. [D01][D01] Form 8-K, August 11, 2026the Company will grant Esjay a non-exclusive, royalty-free license to market its generic version of DOJOLVI beginning January 1, 2033, which would be extended to July 1, 2033 if pediatric exclusivity is granted for DOJOLVI, subject to certain conditions and exceptions customary for agreements of this typeUltragenyx Form 8-K on the Dojolvi patent settlement, filed August 11, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526343525/rare-20260807.htm But suits against other generic makers are still pending. The court-ordered delay on approving their copies ends December 30, 2027. [D02][D02] Form 8-K, August 11, 2026Similar patent litigation brought by the Company against other ANDA filers related to DOJOLVI remains pendingUltragenyx Form 8-K on the Dojolvi patent settlement, filed August 11, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526343525/rare-20260807.htm [T14][T14] Form 10-K 2025, Legal Proceedingsstay extends to December 30, 2027Ultragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm

The stock closed at $14.52 on October 5, up about 11% from $13.06 on September 15. A biotech stock fund, XBI, rose about 1% over the same days. [P01][P01] IEX closing priceRARE closed at $13.06 on September 15, 2026 (IEX)IEX closing prices for RARE and XBI, September 15 to October 5, 2026Open source ↗https://iextrading.com/trading/market-data/ [P02][P02] IEX closing priceRARE closed at $14.52 on October 5, 2026 (IEX)IEX closing prices for RARE and XBI, September 15 to October 5, 2026Open source ↗https://iextrading.com/trading/market-data/ [P03][P03] IEX closing priceXBI closed at $154.18 on September 15, 2026 (IEX)IEX closing prices for RARE and XBI, September 15 to October 5, 2026Open source ↗https://iextrading.com/trading/market-data/ [P04][P04] IEX closing priceXBI closed at $156.2 on October 5, 2026 (IEX)IEX closing prices for RARE and XBI, September 15 to October 5, 2026Open source ↗https://iextrading.com/trading/market-data/ The voucher news came out on October 7, after the last close in our price data. [X06][X06] Ultragenyx release, datelineNOVATO, Calif., Oct. 07, 2026GlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html

The loss for the full year is the number to watch. In the first half of 2026, Ultragenyx lost $277 million. At the second-quarter loss of $92 million a quarter, the year would come to about $461 million without the voucher. [R18][R18] Second quarter 2026 release, results tableNet loss $ (92) $ (115) $ (277) $ (266)Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R05][R05] Second quarter 2026 releaseNet loss for the second quarter 2026 was $92 millionUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm A gain of up to about $168 million, if the sale closes in 2026, would bring it to about $293 million. So our call depends on the sale closing this year and being booked as a gain. [R18][R18] Second quarter 2026 release, results tableNet loss $ (92) $ (115) $ (277) $ (266)Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R05][R05] Second quarter 2026 releaseNet loss for the second quarter 2026 was $92 millionUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [K02][K02] Form 8-K, Item 1.01the buyer agreed to pay the Company $210 million, payable in cash, upon the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [K03][K03] Form 8-K, Item 1.0120% of the gross proceeds from the sale of the PRV will be payable by the Company to the NIH after the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm The call would be wrong if the antitrust review pushes the closing into 2027, or if charges from the cost cuts or from ending apazunersen are large. Last year, third-quarter results came on November 4, and the full year will be in the 2026 annual report. [K05][K05] Form 8-K, Item 1.01The transaction remains subject to customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976Ultragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [A02][A02] Form 8-K, September 2, 2026The Company will evaluate the apazunersen program in light of this outcome and make a decision on its disposition. The Company will also assess its planned operations to define and implement significant expense reductions, while supporting its growing commercial business.Ultragenyx Form 8-K on the Phase 3 Aspire results in Angelman syndrome, filed September 2, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526380165/rare-20260902.htm [W04][W04] Ultragenyx release, third quarter 2025November 04, 2025 16:05 ETGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2025/11/04/3180883/20739/en/ultragenyx-reports-third-quarter-2025-financial-results-and-corporate-update.html

Dates to watch include the end of the antitrust wait and FAYUVI's first shipments. Data from a Wilson disease gene therapy trial is due in the fourth quarter of 2026. [K05][K05] Form 8-K, Item 1.01The transaction remains subject to customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976Ultragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [V03][V03] Ultragenyx release, September 17, 2026commercial product is expected to be available to ship to Qualified Treatment Centers within 30-60 daysGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/09/17/3364357/20739/en/ultragenyx-announces-approval-of-fayuvi-gene-therapy-the-first-ever-fda-approved-treatment-for-sanfilippo-syndrome-type-a-mps-iiia.html [R19][R19] Second quarter 2026 releaseUX701 (rivunatpagene miziparvovec) AAV9 gene therapy for the treatment of Wilson diseaseUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm [R17][R17] Second quarter 2026 releaseData from this stage are expected in the fourth quarter of 2026Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm The case for would weaken if the deal fails to close or cash burn climbs back toward the first quarter's level. Third-quarter results will show the burn rate after the trial failed. [K05][K05] Form 8-K, Item 1.01The transaction remains subject to customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976Ultragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm [Q03][Q03] Form 10-Q, LiquidityCash used in operating activities for the six months ended June 30, 2026 was $294 millionUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm

Who bought the voucher. The October 7 filing calls it only the buyer. [K02][K02] Form 8-K, Item 1.01the buyer agreed to pay the Company $210 million, payable in cash, upon the closing of the transactionUltragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm

When the sale will close. The filing names the antitrust waiting period as a condition but gives no date. [K05][K05] Form 8-K, Item 1.01The transaction remains subject to customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976Ultragenyx Form 8-K on the sale of a rare pediatric disease priority review voucher, filed October 7, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526416251/rare-20261006.htm

How much Ultragenyx will pay in adviser fees and taxes, and so exactly how much cash it keeps. [X03][X03] Ultragenyx release, October 7, 2026Jefferies LLC is serving as exclusive financial advisorGlobeNewswire (Ultragenyx) · Read October 7, 2026Open source ↗https://www.globenewswire.com/news-release/2026/10/07/3376457/20739/en/ultragenyx-enters-into-agreement-to-sell-rare-pediatric-disease-priority-review-voucher-for-210-million.html

Whether the sale will be booked as revenue or as a gain, and in which quarter.

When Ultragenyx will pay Abeona the milestones tied to FAYUVI's approval, and how much of the up to $30 million is due. [T06][T06] Form 10-K 2025, Note 8, Abeonathe Company is obligated to pay tiered royalties of up to 10% on net sales and commercial milestone payments of up to $30.0 million contingent upon regulatory approval of the productUltragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm

Whether anyone shares in a sale of the FAYUVI voucher. The annual report gives voucher-sale shares to GeneTx and Mereo, partners on other drugs, but does not mention vouchers in its description of the FAYUVI licenses. [Q14][Q14] Form 10-Q, Note 7If the Company receives and resells an FDA priority review voucher, or PRV, in connection with a new drug application approval, GeneTx unitholders are entitled to receive a portion of proceedsUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm [T09][T09] Form 10-K 2025, Note 8, MereoIf the Company receives and resells an FDA priority review voucher, or PRV, in connection with a new drug application approval, Mereo is entitled to receive a portion of proceedsUltragenyx annual report on Form 10-K for 2025, filed February 18, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526057329/rare-20251231.htm

Whether the 2027 profit goal counts gains from selling vouchers, such as a 2027 sale of the FAYUVI voucher. [R07][R07] Second quarter 2026 releaseremain on path to profitability in 2027Ultragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm

Whether Ultragenyx will sell stock through its at-the-market program after the voucher cash arrives. [Q10][Q10] Form 10-Q, Liquiditythe Company may offer and sell shares of the Company's common stock having an aggregate offering proceeds up to $350 millionUltragenyx quarterly report on Form 10-Q for the quarter ended June 30, 2026, filed August 5, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526333233/rare-20260630.htm

Whether, when and for how much Ultragenyx will sell its second voucher, from FAYUVI. [V02][V02] Form 8-K, September 17, 2026The Company received a Priority Review Voucher upon this approval.Ultragenyx Form 8-K on the approval of FAYUVI, filed September 17, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm

How large the cost cuts promised on September 2 will be, and when they start. [A02][A02] Form 8-K, September 2, 2026The Company will evaluate the apazunersen program in light of this outcome and make a decision on its disposition. The Company will also assess its planned operations to define and implement significant expense reductions, while supporting its growing commercial business.Ultragenyx Form 8-K on the Phase 3 Aspire results in Angelman syndrome, filed September 2, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526380165/rare-20260902.htm

What Ultragenyx will do with apazunersen, and whether ending it brings new charges. [A02][A02] Form 8-K, September 2, 2026The Company will evaluate the apazunersen program in light of this outcome and make a decision on its disposition. The Company will also assess its planned operations to define and implement significant expense reductions, while supporting its growing commercial business.Ultragenyx Form 8-K on the Phase 3 Aspire results in Angelman syndrome, filed September 2, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526380165/rare-20260902.htm

How much cash operations used in the third quarter, after the trial failed.

The prices and early sales of GENGLYCOS and FAYUVI. [R15][R15] Second quarter 2026 releaseWe are ready to launch two gene therapy productsUltragenyx second quarter 2026 results release, Exhibit 99.1 to Form 8-K, August 4, 2026Open exact filing on SEC EDGAR ↗https://www.sec.gov/Archives/edgar/data/1515673/000119312526332802/rare-ex99_1.htm

The stock's high and low over the past year. Our price data starts in mid-September.