Organovo Announces Positive Phase 2 Results for FXR314 in Metabolic Dysfunction-Associated Steatohepatitis (MASH) Showing Both Reduction in Liver Fat Content and Strong Safety and Tolerability Compared to Placebo
- Statistically significant reduction in liver fat content from baseline with FXR314 compared to placebo.
- LS mean percent reduction at end of treatment of 22.8% with 3 mg and 17.5% with 6 mg doses of FXR314 compared to 6.1% in the placebo group.
- Proportion of subjects with >30% MRI-PDFF reduction was 29.2% for 3 mg and 32.2% for 6 mg FXR314, compared to 9.5% with placebo.
- Improvements in hepatocellular damage and liver function observed with no evidence of worsening of liver fibrosis.
- FXR314 demonstrated excellent tolerability with minimal treatment-emergent adverse events.
- No evidence of common adverse events like pruritus or LDL-C level changes with FXR314.
- Data supportive of further clinical development of FXR314 in MASH.
- None.
Insights
The Phase 2 clinical trial results for FXR314, a FXR agonist developed by Organovo, show significant promise in the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The detailed statistical data, which highlights both efficacy in liver fat reduction and a strong safety profile, is particularly noteworthy for stakeholders in the biotechnology and pharmaceutical sectors. The trial's outcome not only indicates the potential of FXR314 as a treatment option but also places Organovo in a favorable position within the industry, potentially impacting its market valuation.
From a medical research perspective, the reduction in liver fat content and absence of worsening liver fibrosis are key indicators of the drug's therapeutic potential. Moreover, the low incidence of pruritus and negligible drug-related treatment discontinuation rates suggest a better tolerability profile compared to other drugs in the same class. These results might influence the strategic decisions of competitors, leading to shifts in research focus and investment in the area of MASH treatment.
Analyzing the financial implications of the Phase 2 results, the positive outcome positions Organovo to potentially secure additional funding, partnerships, or even become an attractive target for acquisition by larger pharmaceutical companies. The success of FXR314 in the trial stages may lead to an uptick in investor confidence, reflected in the company's stock performance. However, investors should consider the drug's path to market, including the duration and cost of forthcoming Phase 3 trials, regulatory hurdles and the competitive landscape. In the long term, the market potential for MASH treatments, which is currently underserved, could translate into significant revenue streams for Organovo, should FXR314 receive FDA approval.
There is a growing interest in therapies for MASH due to an increasing global prevalence of metabolic disorders. The positive trial results for FXR314 bode well for Organovo, as proven safety and efficacy can position the drug favorably against existing treatments. This development could reshape market dynamics, with potential shifts in market share and pricing power once the drug is launched. The triumph over pruritus, a common side effect in this drug class, could also be a key differentiating factor for FXR314, influencing physician prescribing habits and patient adherence to therapy. Market researchers will closely monitor the anticipated Phase 3 trials and the drug's progress towards commercialization, as these will have significant implications for market forecasts and business strategies within the sector.
SAN DIEGO, April 15, 2024 (GLOBE NEWSWIRE) -- Organovo Holdings, Inc. (Nasdaq:ONVO), a clinical stage biotechnology company focused on developing novel treatment approaches based on demonstration of clinical promise in three-dimensional (3D) human tissues, today released the complete details of its 16-week, randomized, placebo-controlled, multi-center Phase 2 study of the non-steroidal, non-bile acid FXR agonist FXR314 for the treatment of metabolic function-associated steatohepatitis (MASH). Study results demonstrated statistically significant reduction in liver fat content from baseline in patients receiving FXR314 compared to placebo.
Study subjects receiving FXR314 achieved statistically significant reduction in liver fat content from baseline, with LS mean percent reduction at end of treatment of
| FXR314 3 mg | FXR314 6 mg | Placebo | ||||
| Liver fat reduction (LS mean reduction from baseline, SE) | 22.8 + p=0.0010 | 17.5 + p=0.0267 | 6.1 + | |||
| Subjects with > | p=0.0023 | p=0.0020 | ||||
| Pruritus | ||||||
| Pruritus-related treatment discontinuation | ||||||
FXR314 was also found to be safe and well tolerated. Treatment-emergent adverse events were mostly mild to moderate in severity, with incidence comparable between FXR314 3 mg, 6 mg, and placebo. Drug-related treatment discontinuation was minimal and similar across groups. There was also no evidence with FXR314 of adverse events considered common in the FXR class, including measures of pruritus (3 mg
“The key findings of this study are that once daily oral FXR314 demonstrated statistically significant liver fat reduction and excellent tolerability. Whereas other FXR agonists have had challenges in providing clear benefit without significant pruritus or other adverse events, or have had lack of efficacy potentially related to lack of sustained exposure, we are pleased to note FXR314’s data demonstrate it clearly rises above previous problems seen with the class,” stated Keith Murphy, Organovo’s Executive Chairman. “Given these exciting findings, we believe the data are supportive of further clinical development of FXR314 in MASH.”
The clinical trial evaluated the safety, tolerability, and pharmacological activity of FXR314, as measured by reductions in liver fat content with magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF), changes in liver enzymes, low-density lipoprotein cholesterol (LDL-C) levels, and incidence of pruritus. The treatment population were MASH patients diagnosed via biopsy, magnetic resonance elastography (MRE), or transient elastography (TE FibroScan), and who had liver fat content ≥
About Organovo
Organovo is a clinical stage biotechnology company that is developing drugs that are demonstrated to be effective in three-dimensional (3D) human tissues as candidates for drug development. The company’s lead molecule, FXR314, is on the path for Phase 2 investigation in inflammatory bowel disease and has applications in metabolic liver disease and oncology. The company has proprietary technology used to build 3D human tissues that mimic key aspects of native human tissue composition, architecture, function, and disease. For more information visit Organovo's website at www.organovo.com.
Forward Looking Statements
Any statements contained in this press release that do not describe historical facts constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. Any forward-looking statements contained herein are based on current expectations but are subject to a number of risks and uncertainties. These risks and uncertainties and other factors are identified and described in more detail in the Company’s filings with the SEC, including its Annual Report on Form 10-K filed with the SEC on July 14, 2023, as such risk factors are updated in its most recently filed Quarterly Report on Form 10-Q filed with the SEC on November 9, 2023. You should not place undue reliance on these forward-looking statements, which speak only as of the date that they were made. These cautionary statements should be considered with any written or oral forward-looking statements that the Company may issue in the future. Except as required by applicable law, including the securities laws of the United States, the Company does not intend to update any of the forward-looking statements to conform these statements to reflect actual results, later events, or circumstances or to reflect the occurrence of unanticipated events.
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Source: Organovo, Inc.
FAQ
What are the key findings of Organovo's Phase 2 study for FXR314 in MASH?
How was the study population selected for the Phase 2 trial of FXR314?
What were the results of liver fat reduction with FXR314 in the Phase 2 study?
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