Ultragenyx Pharmaceutical Inc. Under Investigation as Stock Plummets 87.64% Following Phase 3 Study Results

Written by Emily J. Thompson, Senior Investment Analyst

RARE+1.28%

Source: PRnewswire

Updated: 5d ago

0mins

Source: PRnewswire

Stock Plunge: Ultragenyx's stock price fell by $2.02, or 87.64%, to close at $0.28 per share on December 29, 2025, following the announcement of two Phase 3 study results that failed to meet primary endpoints, severely impacting investor confidence.
Study Results Analysis: While both studies achieved strong statistical significance in improving bone mineral density, they did not demonstrate statistical significance in reducing clinical fracture rates compared to placebo or bisphosphonates, indicating limitations in treatment efficacy.
Legal Investigation Initiated: Pomerantz LLP is investigating whether Ultragenyx and its executives engaged in securities fraud or other unlawful business practices, potentially exposing the company to legal liabilities and further unsettling investors.
Company Background: Pomerantz LLP is a prominent securities class action law firm with over 85 years of experience, focusing on advocating for victims of securities fraud and corporate misconduct, highlighting the seriousness of Ultragenyx's potential legal risks.

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.