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# Ultragenyx Cuts About 20% Of Staff To 1,000 After Angelman Trial Miss, Targets 2027 Profit
- URL: https://www.bullished.co/ultragenyx-cuts-about-20-of-staff-to-1-000-after-angelman-trial-miss-targets-2027-profit-aaa2f8c1/
- Published: 2026-11-03T23:26:01.000Z
- Updated: 2026-11-03T23:26:01.000Z
- Description: The drugmaker will end its Wilson disease program, hold 2027 R&D below $350 million and says two voucher sales remove any need to raise cash.
- Author: Matteo Parrino
- Tags: Earnings, RARE, Industry: Healthcare

[Ultragenyx Pharmaceutical](https://www.bullished.co/tag/rare/), which develops treatments for rare genetic diseases, will cut its headcount by approximately 20%, to roughly 1,000 employees, after a late-stage Angelman syndrome trial failed, executives said on the company's third-quarter earnings call. Chief Financial Officer Howard Horn said the company still expects to reach full-year profit in 2027 and now has the cash to get there without additional fundraising.

## Angelman miss forces the cuts

Chief Executive Emil Kakkis said the Phase 3 data for GTX102, the Angelman syndrome therapy, were disappointing and "surprised us despite compelling Phase 2 data," which "now has necessitated significant reductions across the company." He said the aim is to bring the cost structure in line with revenue.

Asked by an analyst from Morgan Stanley what drove the miss, Kakkis said there was too much variation within the control group to distinguish changes, across multiple endpoints rather than any single one. Some Phase 2 patients, he said, showed transformative benefit over three to five years, but the one-year randomized trial showed no distinctive separation.

Horn said the plan winds down all GTX102 studies, trims the real estate footprint and right-sizes general and administrative costs.

Separately, Kakkis said the company evaluated UX701, its Wilson disease gene therapy, and found it did not meet the criteria to advance to Phase 3\. A majority of patients did not reach a sufficient biochemical response and come off standard care, he said, and given the need to manage expenses, Ultragenyx will end the program. Kakkis said he still believes gene therapy is a good strategy for Wilson disease and that the company may return to it someday.

## The 2027 spending ceiling

Horn guided 2027 research and development spending below $350 million, a reduction of more than 50% from 2025 levels, with most of it supporting commercial products. He said selling, general and administrative expenses should come in below $375 million, an increase of up to 7% from 2025.

Horn said 2027 spending will go to commercial efforts, finishing the Phase 3 study of DTX301 for OTC deficiency, deciding the path for UX143 in osteogenesis imperfecta, and measured work on early programs.

For 2026, the company reaffirmed revenue of $730 million to $760 million, excluding the two new launches, and expects combined R&D and SG&A to be flat to down low single digits from 2025\. That includes about $25 million of severance and other restructuring charges expected in the fourth quarter.

## Voucher sales fund the path

Ultragenyx sold two priority review vouchers in October. The first deal closed last week and the second is expected to close before the end of the month, Horn said. Net proceeds of about $350 million, after payments to licensors and fees, will count as other income in 2026\. The 2027 profit target does not include voucher income, he said, and the company expects positive operating cash flow that year.

The cuts come as the company launches two gene therapies approved in August and September, for glycogen storage disease type 1a and Sanfilippo syndrome type A, bringing it to six products. Chief Commercial Officer Eric Harris said 35 qualified treatment centers were active at the end of October, with the company on track to exceed its target of 40 by year end. Kakkis said revenue is growing about 20% a year.

Ultragenyx plans to give 2027 revenue guidance early in the new year, and Kakkis said an update on Sanfilippo start forms will come in January.

Kakkis framed the restructuring as a trade: a narrower pipeline in exchange for being, in his words, "in control of our future."

Learn

- [How To Read An Earnings Call Transcript: Start With Guidance, Then Find What's Missing →](https://www.bullished.co/learn/how-to-read-an-earnings-call/)  
How to read an earnings call transcript: check guidance first, look for dropped metrics, compare growth with last quarter, and watch hedged wording.

### More on Ultragenyx Pharmaceutical

- [Ultragenyx To Cut About 20% Of Staff, Wind Down GTX-102 Studies And Drop UX701](https://www.bullished.co/ultragenyx-to-cut-about-20-of-staff-wind-down-gtx-102-studies-and-drop-ux701-3ae355a5/)
- [Madison Avenue Group Reports 6.4% Passive Stake In Ultragenyx, 6,307,798 Shares](https://www.bullished.co/madison-avenue-group-reports-6-4-passive-stake-in-ultragenyx-6-307-798-shares-4d76ed87/)
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[All Ultragenyx Pharmaceutical (RARE) coverage →](https://www.bullished.co/tag/rare/)