XBiotech Pauses Rheumatology program

Rhea-AI Summary

XBiotech (NASDAQ: XBIT) announced the suspension of its rheumatology clinical program following irregularities in its Phase II study of Natrunix for Rheumatoid Arthritis. The study, which enrolled approximately 230 subjects with moderate to severe rheumatoid arthritis, failed to meet its primary endpoint of ACR20 response rate after 12 weeks. The trial evaluated Natrunix (200mg or 400mg weekly) in combination with methotrexate (MTX) against placebo with MTX.

Significant irregularities were discovered at the highest enrolling clinical sites, including multiple enrollments of the same subjects, raising concerns about data interpretation. The company has put additional planned studies in arthritis and ankylosing spondylitis on hold while evaluating these findings.

Positive

• None.

Negative

• Phase II Rheumatoid Arthritis study failed to meet primary endpoint
• Clinical program in rheumatology halted due to study irregularities
• Multiple planned studies now on hold, including ankylosing spondylitis trials
• Data integrity issues discovered with multiple enrollments of same subjects

Insights

Clinical Research Analyst

The Phase II clinical trial failure for XBiotech's Natrunix represents a significant setback in their rheumatology program. The study's integrity has been severely compromised by the discovery of subjects being enrolled multiple times at major trial sites, raising serious concerns about data quality and reliability. This type of irregularity is particularly problematic as it undermines the statistical power and validity of the trial results.

The trial design was robust, involving 230 subjects across three treatment arms testing two doses (200mg and 400mg) of Natrunix with methotrexate against placebo. However, the failure to meet the primary endpoint of ACR20 response rate at 12 weeks, combined with data irregularities, creates a complex situation that could delay the development timeline by 12-18 months while investigations are conducted and potential protocol modifications are implemented.

For investors, this means the company's entire rheumatology pipeline, including planned studies in ankylosing spondylitis, is now in jeopardy. The need to pause and reassess will likely result in increased development costs and a significant delay in potential market entry, impacting the company's valuation and near-term prospects.

Biotech Investment Analyst

From a market perspective, this development is particularly concerning for XBiotech, given their <money>$185.6M</money> market cap. The rheumatology program was a key value driver and this setback could trigger significant market reaction. The irregularities in patient enrollment at major trial sites raise red flags about operational oversight and could lead to increased scrutiny from regulators.

The immediate implications include: <ul><li>Potential write-off of substantial R&D investments</li><li>Delayed revenue potential from the rheumatology pipeline</li><li>Increased costs for potential repeat trials</li><li>Possible need for additional capital raising</li></ul>

This news will likely pressure the stock price as investors reassess the company's pipeline potential and operational capabilities. The timing of the announcement, just before year-end, suggests management is taking a conservative approach to disclosure, but also means the company enters 2024 with significant uncertainty around a major development program.

Findings from recently completed Rheumatoid Arthritis Study raise questions

AUSTIN, Texas, Dec. 23, 2024 (GLOBE NEWSWIRE) -- XBiotech (NASDAQ: XBIT) announced today data that it is halting its clinical program in rheumatological disease while it seeks to understand the outcome from its recently completed Phase II, Double-Blind, Placebo-Controlled, Randomized study in Rheumatoid Arthritis. The Phase II study failed to meet its primary endpoint amid substantial irregularities that make unequivocal interpretation of the findings difficult. XBiotech was planning launch of additional studies in arthritis as well as other areas of rheumatology, including ankylosing spondylitis, which are now on hold while recent findings are evaluated.

About 230 subjects with moderate to severe rheumatoid arthritis were enrolled into the Phase II arthritis study, which examined the Company’s candidate drug, Natrunix, in combination with methotrexate (MTX). The study’s primary endpoint was the American College of Rheumatology (ACR) 20 response rate after 12 weeks of treatment. Various other rheumatological assessments were also performed, including NRS-pain scores, arthritic joint counts, quality of life assessments, and safety. The study population was randomized into three groups in a 1:1:1 ratio: 200 mg or 400mg Natrunix weekly in combination with MTX; or placebo weekly with MTX.

Irregularities in the Phase II study involved the highest enrolling clinical sites, including numerous subjects being enrolled multiple times. Although the study did not meet efficacy endpoints, discrepancies found during data analysis suggest caution in interpreting results. Findings of the study are still being analysed in order to better understand the implications of the findings and determine how these results can be used to guide potential further rheumatology studies for Natrunix.

About XBiotech
XBiotech is pioneering the discovery and development of therapeutics based on its True Human™ antibody technology. The Company has several candidate products including Natrunix, which are cloned from individual donors who possess natural immunity against certain diseases. Located just minutes from downtown Austin, the XBiotech campus headquarters includes GMP manufacturing facilities, research and testing laboratories, infectious disease research facilities, quality control and clinical operations. For more information, visit www.xbiotech.com.

Cautionary Note on Forward-Looking Statements and Study Results
This press release contains forward-looking statements, including declarations regarding management's beliefs and expectations that involve substantial risks and uncertainties. Forward-looking statements are subject to inherent risks and uncertainties in predicting future results and conditions that could cause the actual results to differ materially from those projected in these forward-looking statements. These risks and uncertainties are subject to the disclosures set forth in the "Risk Factors" section of certain of our SEC filings. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Contact

Wenyi Wei
wwei@xbiotech.com
Tel. 737-207-4600 

FAQ

Why did XBiotech (XBIT) halt its rheumatology program in December 2024?

XBiotech halted its rheumatology program due to failed primary endpoints in its Phase II Rheumatoid Arthritis study and substantial irregularities in data collection, including multiple enrollments of the same subjects at high-enrolling sites.

What were the main issues in XBiotech's (XBIT) Phase II Rheumatoid Arthritis trial?

The main issues were failure to meet the ACR20 response rate primary endpoint after 12 weeks and data irregularities at highest enrolling sites, where numerous subjects were enrolled multiple times.

What dosages of Natrunix were tested in XBiotech's (XBIT) Phase II trial?

The trial tested two Natrunix dosages: 200mg and 400mg weekly, both in combination with methotrexate (MTX), compared against placebo with MTX in a 1:1:1 ratio.

Which planned XBiotech (XBIT) studies are now on hold due to the Phase II results?

Additional planned studies in arthritis and ankylosing spondylitis are now on hold while XBiotech evaluates the findings from the Phase II Rheumatoid Arthritis study.