Ultragenyx Pharmaceutical agrees to US$203M priority review voucher sale
Cash is payable at closing, subject to antitrust conditions; two announced voucher agreements total US$413 million.
Theo Marchetti
In the United States, a rare pediatric disease priority review voucher sale announced Wednesday carries a US$203 million price under a conditional agreement.
Ultragenyx Pharmaceutical Inc. NASDAQRARE said in its Oct. 14 release that the deal would provide US$203 million in funding without issuing shares. The California biotechnology company develops and commercializes treatments for rare genetic diseases.
The agreement was signed Oct. 8, according to its Form 8-K filed Wednesday. The buyer agreed to pay cash at closing, subject to customary conditions including expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.
Shares were down 0.23 per cent to US$14.89 on Nasdaq at 3 p.m. EDT on Wednesday NASDAQRARE. The filing supplies no closing date, net-proceeds estimate or revised cash-runway endpoint.
Two Agreements Total US$413 Million
Ultragenyx received the voucher when the FDA approved FAYUVI, also known as UX111, on Sept. 17, according to the filing. The treatment is for pediatric patients with mucopolysaccharidosis type IIIA, also called Sanfilippo syndrome Type A.
The company had already announced a separate US$210 million voucher sale agreement on Oct. 7. That voucher followed approval of GENGLYCOS, also known as DTX401, for glycogen storage disease type Ia.
The two announced prices total US$413 million before any transaction costs. The Oct. 7 release also described a conditional sale, so that combined amount represents agreed consideration rather than confirmed cash receipts.
"The sale of this PRV delivers meaningful non-dilutive funding that strengthens our ability to provide innovative treatments for rare and ultra-rare diseases while supporting our objective of achieving profitability," chief financial officer Howard Horn said in the Oct. 14 release.
Ultragenyx reported US$436 million in cash, cash equivalents and marketable securities at June 30. Net cash used in operations was US$97 million during the quarter ended June 30.
The US$203 million agreed price exceeds twice that quarterly operating cash use. The June balance predates both October agreements and does not establish the company's cash position when either sale closes.
The Aug. 4 financial update retained the company's objective of profitability in 2027. The Oct. 14 documents describe funding to support that objective, but do not quantify additional months of runway or disclose net proceeds.
The Voucher Provides Priority Review
The FDA programme allows a qualifying rare pediatric disease product sponsor to sell or transfer its voucher. Its holder can redeem it for priority review of a later marketing application for a different product, according to the Oct. 14 release.
The FDA's priority review explanation sets a six-month action goal, compared with 10 months for standard review. Priority review leaves the scientific and medical approval standards unchanged.
Closing remains the next transaction step, with no deadline given in Wednesday's documents. Ultragenyx says it will file the full purchase agreement with its annual report for the year ending Dec. 31, 2026.
