# Ultragenyx Sets 2027 Expense Targets as It Winds Down GTX-102

By John Kim

Canonical URL: https://www.tokenpost.com/news/business/48733
Published: 2026-11-04T19:07:10.000Z
Updated: 2026-11-04T19:07:10.000Z
Section: Business

> The company plans to reduce its workforce by about 20% and reaffirmed its forecast for full-year GAAP profitability in 2027.

Ultragenyx Pharmaceutical Inc. set lower expense targets for 2027 while reaffirming its forecast for full-year GAAP profitability, tying the plan to a roughly 20% workforce reduction and the wind-down of GTX-102 studies.

The company announced Tuesday, Nov. 3, that it expects full-year research and development expenses below $350 million and selling, general and administrative expenses below $375 million in 2027. The R&D target is at least 50% below 2025 levels. Ultragenyx develops and commercializes treatments for rare and ultra-rare genetic diseases.

The reductions include winding down all ongoing studies of GTX-102, an investigational treatment for Angelman syndrome also known as apazunersen. The Phase 3 Aspire trial missed its primary Bayley-4 cognitive measure and its key secondary measure on the Multidomain Responder Index. Ultragenyx announced the results Sept. 2.

The company also plans to terminate UX701, a Wilson disease program that did not meet its criteria to advance to Phase 3, and reduce real estate and other expenses. Its R&D priorities include the Phase 3 DTX301 data, a decision on UX143’s future and limited spending on early-stage programs. DTX301 data are expected in the first half of 2027.

Ultragenyx expects to cut its workforce by about 20%, leaving roughly 1,000 employees. The company expects the reduction to be substantially complete by the end of 2026. It anticipates about $25 million in severance and other non-recurring restructuring charges in the fourth quarter of 2026.

For the three months ended Sept. 30, Ultragenyx reported revenue of $173 million and a net loss of $118 million. Cash, cash equivalents and marketable securities totaled $401 million at quarter-end. The expense plan accompanies a shift toward commercial operations following 2026 FDA approvals for GENGLYCOS, for glycogen storage disease type Ia, and FAYUVI, for Sanfilippo syndrome type A.

President and CEO Emil D. Kakkis said, “we expect meaningful profitability in 2027 and going forward.”