FDA clears Plus Therapeutics’ pediatric brain cancer treatment trial

Published 25/06/2025, 12:34
FDA clears Plus Therapeutics’ pediatric brain cancer treatment trial

HOUSTON - The U.S. Food and Drug Administration has cleared Plus Therapeutics’ (NASDAQ:PSTV) Investigational New Drug application for REYOBIQ to treat pediatric patients with high-grade glioma and ependymoma, the company announced Wednesday. The micro-cap biotech, currently valued at $9.54 million, has seen its stock trade at $0.19, with InvestingPro data showing significant volatility in recent months.

The Phase 1/2a clinical trial, named ReSPECT-PBC, will be funded by a $3.0 million grant from the U.S. Department of Defense. The study will enroll children aged 6 to 21 years, with consideration for patients up to 25 years on a case-by-case basis. According to InvestingPro analysis, Plus Therapeutics maintains a healthy balance sheet with more cash than debt, though the company is currently burning through cash reserves.

The trial will follow a two-part design. Phase 1a/b will enroll approximately 24 patients to establish the maximum tolerated dose, while Phase 2a will include about 32 patients to assess efficacy.

REYOBIQ (Rhenium Re186 Obisbemeda) is designed to deliver beta radiation directly to brain tumors while minimizing damage to surrounding healthy tissue. The treatment uses convection enhanced delivery to bypass the blood-brain barrier.

"Surgery and external beam radiation have been the mainstays of treatment for pediatric high-grade glioma and, sadly, outcomes have not improved for many decades," said Dr. Ashley S. Plant, Principal Investigator for the trial and attending physician at Ann & Robert H. Lurie Children’s Hospital of Chicago.

Pediatric high-grade gliomas, including ependymoma, are rare but aggressive brain tumors with limited treatment options. Standard treatments often fail to prevent recurrence, with five-year survival rates as low as 22% for high-grade glioma.

The pediatric trial builds on Plus Therapeutics’ adult recurrent glioblastoma trial (ReSPECT-GBM), which according to a press release statement showed favorable safety and clinical response. While analysts expect revenue challenges in the near term, InvestingPro subscribers can access 14 additional key insights and a comprehensive Pro Research Report that provides detailed analysis of the company’s financial health and growth prospects.

In other recent news, Plus Therapeutics has restructured its $15 million equity financing to minimize potential dilution, canceling warrants that could have led to the issuance of approximately 1.51 billion shares. This restructuring includes the cancellation of about 25 million shares of common stock or pre-funded warrants, with the company planning to use 90% of proceeds from future capital raised to repay certain shareholders. Additionally, Ascendiant Capital has raised its price target for Plus Therapeutics to $20.50, maintaining a Buy rating due to confidence in the company’s drug development progress and anticipated milestones. In clinical developments, Plus Therapeutics presented new data on its lead drug, REYOBIQ™, at a recent conference, showing promising safety and clinical benefits for CNS cancers, with a Clinical Benefit Rate of 76% in the Phase 1 ReSPECT-LM trial. Furthermore, the company announced the appointment of Kyle Guse to its Board of Directors, bringing over 30 years of financial and legal expertise. The company also reported having $10 million in cash with no debt, having raised approximately $18 million in the first quarter, which analysts believe will sustain operations into 2026. Plus Therapeutics continues to focus on advancing its pipeline of radiotherapeutics, with leading programs targeting leptomeningeal metastases and recurrent glioblastoma.

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