CHICAGO and FORT WORTH, Texas - Actuate Therapeutics, Inc. (NASDAQ: ACTU), a biopharmaceutical company focused on developing treatments for cancers, announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to its drug, elraglusib, for the treatment of Ewing sarcoma.
Ewing sarcoma is a highly metastatic form of bone cancer primarily affecting children and adolescents, with 25% of patients having metastatic disease at diagnosis. This designation by the FDA is a significant milestone for elraglusib, which is currently being evaluated in a Phase 1/2 trial for safety and efficacy in pediatric patients with relapsed or refractory malignancies, including Ewing sarcoma.
Daniel Schmitt, President & CEO of Actuate, expressed optimism about the early clinical data from the trial, which has shown promising anti-tumor activity. Two patients have achieved ongoing durable Complete Responses among the first six treated. The trial, identified as Actuate-1902 (NCT 04239092), has enrolled eight patients so far.
The rare pediatric disease designation is granted to drugs that treat serious or life-threatening diseases affecting fewer than 200,000 people in the U.S., primarily those under 18 years of age. Should the New Drug Application for elraglusib be approved, Actuate could receive a Priority Review Voucher (PRV), which may be used by the company or sold to another entity.
Elraglusib is a novel inhibitor of glycogen synthase kinase-3 beta (GSK-3β), a molecule involved in promoting tumor growth and resistance to conventional cancer drugs. It also aims to mediate anti-tumor immunity by inhibiting nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and regulating immune checkpoints and cell function.
This press release statement contains forward-looking statements regarding Actuate's clinical trials and development plans. The company cautions that the development process is lengthy and outcomes are uncertain, with no guarantee of successful results or timely regulatory approval.
Actuate's ongoing efforts in the development of elraglusib underscore the urgent need for new treatments for Ewing sarcoma, aiming to provide therapeutic options where current treatments fall short.
In other recent news, Actuate Therapeutics has gained significant ground in its clinical trials and internal operations. The U.S. Food and Drug Administration granted Orphan Drug Designation to the company's investigational drug, elraglusib, for the treatment of soft tissue sarcoma. This marks a crucial milestone for elraglusib, a novel inhibitor of glycogen synthase kinase-3 beta, which is instrumental in the growth and drug resistance of tumors.
Actuate has also reported promising 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 aims to enroll up to 12 Ewing Sarcoma patients in this trial, pushing the development path for elraglusib towards commercial registration.
In terms of corporate changes, Actuate 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. The decision was made by Actuate's Audit Committee for the fiscal year ending December 31, 2024. The company 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 developments offer an insight into Actuate's recent progress in both its clinical trials and financial operations.
InvestingPro Insights
Actuate Therapeutics' recent FDA designation for elraglusib marks a significant milestone in the company's journey to address rare pediatric cancers. While this development is promising, it's crucial to consider the financial landscape of the company as it progresses through clinical trials.
According to InvestingPro data, Actuate Therapeutics has a market capitalization of $175.2 million, reflecting its current valuation in the biotech sector. The company's stock has shown strong momentum recently, with a 22.04% price return over the past month. This surge could be attributed to positive investor sentiment following the FDA's rare pediatric disease designation for elraglusib.
However, it's important to note that Actuate is currently not profitable, as indicated by its negative P/E ratio of -6.34 for the last twelve months as of Q2 2024. This is not uncommon for biotech companies in the development stage, as they often incur significant research and development expenses before generating revenue from drug sales.
InvestingPro Tips highlight that Actuate operates with a moderate level of debt, which could provide some financial flexibility as it advances its clinical programs. The company's ability to manage its financial resources will be crucial as it progresses through the costly phases of drug development and potential commercialization.
For investors interested in a deeper analysis, InvestingPro offers 5 additional tips that could provide valuable insights into Actuate Therapeutics' financial health and market position. These tips can help in assessing the company's potential as it works towards bringing innovative treatments like elraglusib to market.
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