CTI BioPharma Announces Topline Data from Final Analysis of the PRE-VENT Clinical Trial Evaluating Pacritinib as a Treatment for Severe COVID-19

Rhea-AI Summary

CTI BioPharma Corp. announced topline results from the PRE-VENT trial evaluating pacritinib in hospitalized patients with severe COVID-19. Despite the study's well-tolerated profile, no significant improvement in the primary endpoint of progression to invasive mechanical ventilation or death (26.3% vs. 24.8%, P=0.98) was found. As a result, the company will not pursue further development of pacritinib for this indication. CTI BioPharma will instead focus on pacritinib's launch for myelofibrosis, with a PDUFA date of November 30, 2021.

Positive

• Pacritinib was well tolerated, with lower rates of grade 3+ events and serious adverse events (29.2% vs. 40.6%).
• The most common adverse events were comparable between treatment and placebo groups, indicating safety.

Negative

• No significant clinical benefit of pacritinib in the treatment of severe COVID-19 was demonstrated.
• The decision to discontinue development for COVID-19 reduces potential market opportunity.

SEATTLE, Oct. 1, 2021 /PRNewswire/ -- CTI BioPharma Corp. (Nasdaq: CTIC) today announced topline results from the PRE-VENT trial (NCT04404361) of pacritinib in hospitalized patients with severe COVID-19. PRE-VENT, a randomized, double-blind, placebo-controlled multicenter study, compared pacritinib/standard of care versus placebo/standard of care in hospitalized patients with severe COVID-19, including patients with and without cancer. The primary endpoint of the trial was the proportion of patients who progressed to invasive mechanical ventilation and/or extracorporeal membrane oxygenation or died by Day 28. The study was recently amended to a Phase 2 design due to the availability of COVID-19 vaccines and an evolving COVID-19 environment.

The final PRE-VENT analysis was conducted following the randomization of 200 patients. In patients with severe COVID-19 treated with pacritinib/standard of care, compared to placebo/standard of care, a statistically significant improvement in the primary endpoint of progression to invasive mechanical ventilation and/or extracorporeal membrane oxygenation or death by Day 28 was not demonstrated (26.3% vs. 24.8% [OR: 1.01 (95% CI: 0.51-1.99)], P=0.98).

Pacritinib was generally well-tolerated by patients with severe COVID-19, with lower rates of grade 3+ events and serious adverse events being reported in patients treated with pacritinib/standard of care compared to placebo/standard of care (29.2% vs. 40.6% and 20.8% vs. 32.7%, respectively). The most common adverse events comparing pacritinib/standard of care to placebo/standard of care were increased alanine aminotransferase (15.6% vs. 16.8%), anemia (12.5% vs. 17.8%), bradycardia (15.6% vs. 10.9%), constipation (12.5% vs. 12.9%), hypokalemia (12.5% vs. 12.9%), and hyperkalemia (9.4% vs. 10.9%). 

"Last year, we launched the PRE-VENT study of pacritinib in patients with severe COVID-19 in response to the pandemic and an unprecedented medical need to improve outcomes for these patients," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. "Although pacritinib was well tolerated on study, it did not demonstrate a clinical benefit in treating the sickest patients with COVID-19. We would like to thank all the patients, families, physicians, and medical teams who participated in this study during such a difficult time. Based on the results of this study, we have decided not to pursue further development of pacritinib for the treatment of severe COVID-19. We will continue to focus our efforts on launch preparedness activities for pacritinib in myelofibrosis, which is currently under Priority Review with a PDUFA date of November 30, 2021."

The Company expects to publish a full report on the study in a medical journal.

About Pacritinib
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, IRAK1, and CSF1R, but not JAK1. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia, and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT.

About CTI BioPharma Corp.
We are a biopharmaceutical company focused on the acquisition, development and commercialization of novel targeted therapies for blood-related cancers that offer a unique benefit to patients and their healthcare providers. We concentrate our efforts on treatments that target blood-related cancers where there is an unmet medical need. In particular, we are focused on developing and commercializing pacritinib, our product candidate currently in active late-stage development. We are headquartered in Seattle, Washington.

Forward-Looking Statements
Statements included in this press release that are not historical in nature are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based on current assumptions that involve risks, uncertainties and other factors that may cause the actual results, events or developments to be materially different from those expressed or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: our ability to conduct and complete clinical trials in our currently anticipated timeframes; our ability to successfully demonstrate the safety and efficacy of pacritinib; our expectations regarding the completion and outcome of our PACIFICA Phase 3 trial; the risk that pacritinib may fail in its development through our PACIFICA trials; risks and uncertainties related to the COVID-19 pandemic as it relates to our operations and ongoing clinical trials; and those risks more fully discussed in the section entitled "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020 and subsequent quarterly reports on Form 10-Q. These forward-looking statements speak only as of the date hereof and we assume no obligation to update these forward-looking statements, and readers are cautioned not to place undue reliance on such forward-looking statements.

"CTI BioPharma" and the CTI BioPharma logo are registered trademarks or trademarks of CTI BioPharma Corp. in various jurisdictions. All other trademarks belong to their respective owner.

CTI BioPharma Investor Contacts:
Argot Partners
+212-600-1902
cti@argotpartners.com

 

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SOURCE CTI BioPharma Corp.

FAQ

What were the results of the PRE-VENT trial for CTIC's pacritinib?

The PRE-VENT trial showed no significant improvement in the primary endpoint, indicating pacritinib did not benefit patients with severe COVID-19.

What is the PDUFA date for pacritinib in myelofibrosis?

The PDUFA date for pacritinib regarding myelofibrosis is November 30, 2021.

Why has CTI BioPharma discontinued pacritinib for COVID-19 treatment?

CTI BioPharma decided to discontinue pacritinib for COVID-19 due to the lack of demonstrated clinical benefit in the trial.

What is the focus of CTI BioPharma after the PRE-VENT trial results?

CTI BioPharma will now focus on the launch activities for pacritinib in myelofibrosis.

How well-tolerated was pacritinib in the PRE-VENT trial?

Pacritinib was generally well-tolerated, with lower rates of serious adverse events compared to placebo.