Ultragenyx Updates on 2026 Clinical and Regulatory Catalysts

Written by Emily J. Thompson, Senior Investment Analyst

Updated: Jan 12 2026

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Ultragenyx provided a corporate update. 2026 Clinical and Regulatory Catalysts: DTX401 - Biologics License Application rolling submission completed in December 2025; UX111 - Resubmission of the BLA is on track for early in 2026; GTX-102 - ata from the fully enrolled, pivotal, Phase 3 Aspire study in patients with a genetically confirmed diagnosis of UBE3A deletion is expected in the second half of 2026; UX701 - Enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data are expected in the first half of 2026

RARE

$24.62+Infinity%1D

Analyst Views on RARE

Wall Street analysts forecast RARE stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for RARE is 61.65 USD with a low forecast of 35.00 USD and a high forecast of 120.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.

18 Analyst Rating

17 Buy

1 Hold

0 Sell

Strong Buy

Current: 24.310

Low

35.00

Averages

61.65

High

120.00

Current: 24.310

Low

35.00

Averages

61.65

High

120.00

Morgan Stanley

Maxwell Skor

Overweight

downgrade

$55 -> $50

2026-01-08

Reason

Morgan Stanley

Maxwell Skor

Price Target

$55 -> $50

AI Analysis

2026-01-08

downgrade

Overweight

Reason

Morgan Stanley analyst Maxwell Skor lowered the firm's price target on Ultragenyx to $50 from $55 and keeps an Overweight rating on the shares. The firm sees a continuation of U.S. small-to-mid cap biotech's outperformance in 2026 as commercial names transition from "capital consumers to producers" and Large Cap Biopharma faces a looming patent cliff, the analyst tells investors in an outlook note for the group.

Guggenheim

Buy

downgrade

$64 -> $52

2026-01-05

Reason

Guggenheim

Price Target

$64 -> $52

2026-01-05

downgrade

Buy

Reason

Guggenheim lowered the firm's price target on Ultragenyx to $52 from $64 and keeps a Buy rating on the shares after the company announced the failure of the ORBIT and COSMIC studies evaluating setrusumab in osteogenesis imperfecta.

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H.C. Wainwright

Buy

downgrade

$80 -> $60

2026-01-05

Reason

H.C. Wainwright

Price Target

$80 -> $60

2026-01-05

downgrade

Buy

Reason

H.C. Wainwright lowered the firm's price target on Ultragenyx to $60 from $80 and keeps a Buy rating on the shares after the two setrusumab pivotal trials in osteogenesis imperfecta failed to meet their primary endpoint. The firm views the setrusumab failure as "merely a bump in the road" and tells investors to consider accumulating Ultragenyx shares at current levels. Ultragenyx remains "a diversified, commercial-stage company with multiple revenue streams from marketed products as well as a rich pipeline of clinical-stage assets," contends H.C. Wainwright.

BofA

Buy

downgrade

$72 -> $58

2026-01-02

Reason

BofA

Price Target

$72 -> $58

2026-01-02

downgrade

Buy

Reason

BofA lowered the firm's price target on Ultragenyx to $58 from $72 and keeps a Buy rating on the shares after the company announced the Phase 3 Orbit and Cosmic studies evaluating setrusumab in osteogenesis imperfecta failed to demonstrate a statistically significant benefit for the primary endpoint. Following the news, the firm removed OI value from its model and adjusted its operating expense estimates for the company's plans to significantly reduce expenses.

About RARE

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. Its four approved product candidates include Crysvita (burosumab) for the treatment of X-linked hypophosphatemia (XLH), and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome, Dojolvi (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its clinical product candidates include DTX401, DTX301, UX701, UX143, UX111, and GTX-102. UX143 for the treatment of Osteogenesis Imperfecta.

About the author

Emily J. Thompson

Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.