Ultragenyx Pharmaceutical Inc. (RARE) SEC Filings & Earnings Calls

Generic entry risk for DOJOLVI in 2033, potential revenue erosion.

“Navinta will market generic DOJOLVI starting Jan 1, 2033 (or Jul 1, 2033 with pediatric exclusivity).”

Transaction subject to HSR antitrust waiting period; no other material risks disclosed.

“The 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 1976.” · word for word

20% of gross proceeds payable to NIH reduces net cash inflow

“20% of the gross proceeds from the sale of the PRV will be payable by the Company to the NIH” · word for word

Transaction subject to HSR antitrust waiting period

“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” · word for word

Pipeline setback with apazunersen failure; potential write-off of R&D investment and loss of future revenue opportunity.

“Phase 3 Aspire study did not achieve primary or key secondary endpoints”

Significant expense reductions may impact other pipeline programs and employee morale.

“Implement significant expense reductions” · word for word

Post-marketing study requirements and long-term safety data needed.

“Ultragenyx has agreed to provide two years of safety and efficacy clinical data from open-label commercial treatment of 50 patients and 20 control patients through enhancement of its existing GSDIa Disease Monitoring Program.” · word for word

Patent litigation partially resolved, but other ANDA filers still pose risk.

“Similar patent litigation brought by the Company against other ANDA filers related to DOJOLVI remains pending.” · word for word

Generic entry allowed in 2033, which could impact revenue if DOJOLVI is still significant.

“license to market its generic version of DOJOLVI beginning January 1, 2033.” · word for word

Operating losses and cash burn

“Cash used in operating activities for the six months ended June 30, 2026 was $294 million and primarily reflected a net loss of $277 million” · word for word

Patent litigation risk for Dojolvi

“In September 2024, we filed a patent infringement suit ... against Navinta ... seeking FDA approval to market a generic version of Dojolvi” · word for word

Dependence on single-source suppliers

“The failure to supply by any of our single-source suppliers could adversely affect our business.” · word for word

Net loss and cash burn; reliance on pipeline success for future growth.

“Net loss of $92M; net cash used in operations $97M in Q2.”

Revenue concentration in Crysvita

“Crysvita product sales and royalty revenue combined represent $93 million of $136 million total revenue (68%).”

Dependence on single-source suppliers

“The failure to supply by any of our single-source suppliers could adversely affect our business.” · word for word

Litigation risk from securities class action

“In February 2026, Steven Bailey filed a putative class action on behalf of certain of our stockholders against the Company, our CEO and our Chief Medical Officer in the United States District Court for the Northern District of California.” · word for word

Manufacturing litigation with Catalent

“Our suit seeks monetary damages from Catalent in excess of $100 million.” · word for word

Operating loss history

“Since inception, we have been engaged in substantial research and development and capital investments, and we have operated at an operating loss each year and expect to continue doing so in the near term.” · word for word

Net loss widening and high cash burn rate; path to profitability dependent on successful product launches and cost reductions.

“Net loss $185M vs $151M prior year; net cash used in operations $197M.”

Regulatory and clinical execution risk: two BLAs under review and Phase 3 data expected; any delays or negative outcomes could impact stock.

“Two BLAs under review; Phase 3 data expected in H2 2026.”

Regulatory approval is not guaranteed; FDA may require additional data.

“the FDA may not accept or agree that the submitted data are sufficient to support accelerated or full approval” · word for word

Clinical development risk; early-stage program with no efficacy data yet

“Phase 1/2 study expected to begin in H2 2026; safety and efficacy not yet established.” · word for word

Clinical trial failure risk

“we reported disappointing results for our Phase 3 Orbit and Cosmic studies for UX143 for the treatment of osteogenesis imperfecta when the studies failed to meet their primary endpoints.” · word for word

Regulatory delay risk

“we received a Complete Response Letter (CRL) for UX111 for the treatment of Sanfilippo syndrome type A, with the CRL citing specific chemistry, manufacturing controls related to our facilities and processes, which significantly delayed potential FDA approval of UX111.” · word for word

Equity dilution risk

“If the Third A&R Plan is approved, our overhang will be approximately 21% as of March 9, 2026.” · word for word

Stock option underwater risk

“many of the stock options attributing to our overhang are underwater...The 9,497,800 outstanding stock options currently underwater account for 8% of our overhang if the Third A&R Plan is approved.” · word for word

Say-on-pay support risk

“We received 68% support for our say-on-pay proposal at our 2025 Annual Meeting.” · word for word

Clinical trial risk; one SAE of acute hepatitis resolved with steroids; study continuing to second primary endpoint.

“One serious adverse event of acute hepatitis was assessed as treatment-related and resolved with steroids.”

Regulatory and clinical development risks remain; approval not guaranteed

“Forward-looking statements highlight risks of clinical development, regulatory approval, and manufacturing”

Clinical trial failure pressure

“Adverse results or delays in preclinical or clinical studies could cause our stock price to be highly volatile.” · word for word

Regulatory compliance cost

“Any delay in filing a regulatory submission for any of our product candidates and any adverse development or perceived adverse development with respect to the applicable regulatory agency’s review of such regulatory submission.” · word for word

Litigation liability pressure

“We have been defending a lawsuit filed in the U.S. District Court for the District of Maryland by the Estate of Henrietta Lacks alleging unjust enrichment arising from our receipt and use of HeLa cells.” · word for word

Dollar strength pressure

“A weakening of foreign currencies relative to the U.S. dollar may negatively affect our revenue and operating income as expressed in U.S. dollars.” · word for word

Restructuring with workforce reduction and manufacturing agreement terminations indicates financial pressure and cost-cutting measures.

“10% workforce reduction and ~$50M in restructuring charges.”

Regulatory risk: BLA resubmission under FDA review; PDUFA date expected Q3 2026; no guarantee of approval

“The Company anticipates up to a six-month review period from the date of resubmission per FDA regulations, with a PDUFA date expected in the third quarter of 2026.” · word for word

Regulatory risk; FDA may not approve or may require additional data.

“BLA resubmission; previous CRL in July 2025.”

Regulatory and clinical development risks for pipeline candidates

“Forward-looking statements highlight risks of clinical drug development, regulatory approvals, and reliance on third parties.”

Dependence on Kyowa Kirin for Crysvita commercialization and supply

“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.” · word for word

Regulatory approval risk for DTX401

“uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals” · word for word

Pipeline risk: setrusumab program uncertain after Phase 3 miss; expense reductions may impact other programs.

“Neither study achieved statistical significance against the primary endpoints” · word for word

Patent litigation risk for Dojolvi

“On September 26, 2024, we filed a patent infringement suit under the Hatch-Waxman Act against Navinta LLC, Aurobindo Pharma Limited, and Esjay Pharma LLC... seeking FDA approval to market a generic version of Dojolvi.” · word for word

Manufacturing litigation with Catalent

“On October 9, 2024, we filed a suit against Catalent... alleging that Catalent fraudulently mispresented its manufacturing capabilities and serially breached the terms of its manufacturing agreement with us. Our suit seeks monetary damages from Catalent in excess of $100 million.” · word for word

Cash burn and need for additional capital

“We may require additional capital to fund our operations, to complete our ongoing and planned clinical studies, to commercialize our products... and such funding may not be available to us on acceptable terms or at all.” · word for word

Dependence on Crysvita royalty stream; future royalty payments capped and shared with OMERS

“OMERS receives 25% of future Crysvita royalties from 2028, capped at 1.55x purchase price”

Clinical trial risk; data from Aspire study expected H2 2026.

“Data from the Aspire study are expected in the second half of 2026.” · word for word