FDA grants orphan drug status to Plus Therapeutics’ cancer treatment

Published 06/03/2025, 13:40
FDA grants orphan drug status to Plus Therapeutics’ cancer treatment

HOUSTON - Plus Therapeutics, Inc. (NASDAQ:PSTV), a clinical-stage pharmaceutical company with a market capitalization of $2.1 million, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational treatment, Rhenium (186Re) Obisbemeda, for leptomeningeal metastases (LM) in lung cancer patients. According to InvestingPro data, the company maintains more cash than debt on its balance sheet, though analysts do not expect profitability this year.

This designation is a critical step for the company, aiming to address the unmet medical needs of LM patients, a rare and typically terminal cancer complication where the disease spreads to the cerebrospinal fluid and membranes around the brain and spinal cord. Lung cancer patients with LM currently have limited treatment options, with the condition often leading to death within weeks to several months if left untreated.

The FDA’s ODD status provides benefits to drug developers, including potential market exclusivity for seven years, tax credits for clinical trials, and exemptions from certain regulatory fees. This support is particularly significant given the $4.3 million Prescription Drug User Fee Act charge in 2025.

Plus Therapeutics has recently completed a Phase 1 single-dose trial for Rhenium (186Re) Obisbemeda, establishing the recommended Phase 2 dose. The company is progressing with a Phase 2 single-dose expansion trial and a Phase 1 multiple-dose trial. Discussions with the FDA are ongoing to determine the strategy for pivotal trials. InvestingPro analysis reveals the company is quickly burning through cash, with a current ratio of 0.44, indicating potential challenges in meeting short-term obligations.

Rhenium (186Re) Obisbemeda is a novel injectable radiotherapy designed to deliver targeted high-dose radiation to central nervous system tumors. It leverages the radioisotope Rhenium-186, which is considered ideal for CNS applications due to its short half-life and suitable radiation properties for both treatment and imaging.

The development of Rhenium (186Re) Obisbemeda has been supported by significant funding, including a three-year $17.6 million grant from the Cancer Prevention & Research Institute of Texas for the ReSPECT-LM clinical trials. Plus Therapeutics is focused on enhancing clinical outcomes for difficult-to-treat CNS cancers and is headquartered in key cancer clinical development hubs in Texas.

This news is based on a press release statement from Plus Therapeutics, Inc. and does not include personal opinions or recommendations.

In other recent news, Plus Therapeutics, Inc. has made significant advancements in its clinical trials and corporate developments. The company has identified a recommended Phase 2 dose for its ReSPECT-LM Phase 1 trial, which focuses on treating leptomeningeal metastases, a severe cancer complication. This dose determination follows the observation of no dose-limiting toxicities and a complete response in one patient. Plus Therapeutics is now moving ahead with Phase 2 and Phase 1 trials using this dose, with plans to complete enrollment within the year.

Additionally, the company has secured approximately $5.7 million in funding through a combination of private placement financing and a grant advance from the Cancer Prevention and Research Institute of Texas. These funds are allocated for the continued development of Rhenium (186Re) Obisbemeda and the CNSide LM diagnostic test. In a strategic move, Plus Therapeutics appointed Dr. Michael Rosol as Chief Development Officer to oversee clinical and biomarker development activities. Dr. Rosol’s extensive background in oncology and radiotherapeutic drug development is expected to support the company’s transition to pivotal trials. The company is actively working with strategic partners to advance its pipeline and improve treatment outcomes for central nervous system cancers.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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