CHICAGO and FORT WORTH, Texas - Actuate Therapeutics, Inc. (NASDAQ: ACTU), a clinical-stage biopharmaceutical company, has announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to elraglusib, the company's investigational drug for the treatment of soft tissue sarcoma (STS). The designation is a significant milestone for elraglusib, which is a novel inhibitor of glycogen synthase kinase-3 beta (GSK-3β), a molecule involved in the growth and drug resistance of tumors.
The American Cancer Society projects that around 13,590 new cases of STS will be diagnosed in the United States in 2024, with an estimated 5,200 deaths expected from the disease. STS represents a group of over 70 different histological subtypes of tumors, which complicates treatment. While surgery is effective for localized cancer, metastatic STS has a dire prognosis with median overall survival of less than 6-12 months.
According to Dr. Steven D. Reich, Senior VP of Clinical Development and Acting Chief Medical Officer at Actuate, the current first-line systemic treatment for metastatic STS is doxorubicin, a drug approved nearly half a century ago with limited antitumor activity. Elraglusib has shown promising results in preclinical studies, inducing significant STS cell apoptosis and enhancing the effects of chemotherapy, providing a strong basis for its clinical evaluation.
The ODD status conferred by the FDA will aid Actuate in the development of elraglusib by offering benefits such as development assistance, tax credits, FDA fee exemptions, and seven years of marketing exclusivity post-approval.
Elraglusib is designed to inhibit molecular pathways in cancer, including DDR pathways, and mediate anti-tumor immunity by inhibiting NF-kB. It also regulates multiple immune checkpoints and immune cell functions. Actuate's focus is on the development of therapies for high-impact, difficult-to-treat cancers.
The information presented in this article is based on a press release statement from Actuate Therapeutics.
In other recent news, Actuate Therapeutics has reported promising preliminary results from its Phase 1/2 trial of elraglusib for patients with relapsed/refractory Ewing Sarcoma, with two ongoing complete responses and a disease control rate of approximately 62%. The company plans to enroll up to 12 total Ewing Sarcoma patients in this trial, aiming to accelerate the development path for elraglusib towards commercial registration. Elraglusib, a novel glycogen synthase kinase-3 beta inhibitor, is being tested in combination with chemotherapy agents.
In other changes, Actuate Therapeutics has announced a significant shift in its independent registered public accounting firm. KMJ Corbin & Company LLP has resigned and will be replaced by Crowe LLP effective immediately. This decision was made by Actuate's Audit Committee for the fiscal year ending December 31, 2024.
Despite the change, Actuate Therapeutics has confirmed that there were no disagreements with KMJ on any matter of accounting principles or practices, financial statement disclosure, or auditing scope or procedure. These are the recent developments in the company's financial operations and clinical trials.
InvestingPro Insights
As Actuate Therapeutics, Inc. (NASDAQ: ACTU) secures Orphan Drug Designation for elraglusib, investors and stakeholders are closely monitoring the company's financial health and stock performance. According to InvestingPro data, Actuate's market capitalization stands at approximately $156.72 million. Despite the potential of elraglusib, the company's financial metrics indicate some challenges, with a negative price-to-earnings (P/E) ratio of -0.49 for the last twelve months as of Q1 2024, which further declined to -5.73 when adjusted. This suggests that the company has not been profitable over the past year.
Additionally, the stock has experienced a decline in its price total return over various time frames, with a -4.76% return over the past month, three months, six months, and year-to-date as of the end of 2024. This could be reflective of the risks associated with clinical-stage biopharmaceutical investments, where significant capital is often required with no guarantee of successful product development or regulatory approval.
InvestingPro Tips for Actuate Therapeutics highlight that the stock is currently in overbought territory based on the Relative Strength Index (RSI), which may concern some investors about the sustainability of recent price movements. Furthermore, the company suffers from weak gross profit margins and its short-term obligations exceed its liquid assets, indicating potential liquidity risks. It's also noteworthy that Actuate operates with a moderate level of debt and does not pay a dividend to shareholders, which could influence investment decisions for those seeking regular income or less leveraged companies.
For investors looking for a deeper dive into Actuate Therapeutics' financials, there are additional InvestingPro Tips available at https://www.investing.com/pro/ACTU, which provide a comprehensive analysis of the company's performance and potential investment risks and opportunities.
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