FDA Clears New Drug Application For Pluristem's PLX-R18; What's Next?

Pluristem Therapeutics Inc. PSTI, which develops placenta-based cell therapy products, announced Monday that the FDA cleared an investigational new drug application for PLX-R18 cell therapy to treat acute radiation syndrome.

Pluristem shares were rallying 7.26 percent Monday afternoon to $1.33. 

An IND App Primer 

An IND application is the first step in FDA's drug review process. The company developing the new molecule — the sponsor — submits the application after determining through preclinical testing, typically animal testing, that it is safe to be moved into clinical trials and human testing. 

An IND application comprises:

• Animal study data and toxicity data.
•  Manufacturing information.
• Clinical protocols for studies to be conducted.
• Data from any prior human research.
• Information about the investigator.

The sponsor must wait 30 calendar days before initiating any clinical trials, which also gives the FDA time to review the IND application for safety. The FDA responds with either the approval to begin clinical trials or a clinical hold to delay or stop the investigation.

A clinical hold is announced if the safety of the molecule is in question, investigators are unqualified or the application does not include enough information about the trial's risks.

A clinical hold is rare, according to the FDA, as the agency provides comments for improving the quality of the trial. Once the trials meet FDA standards, the sponsor is given the go-ahead.

When a Phase 1 trial is initiated, the molecule is tested for safety and dosage with 20-100 healthy volunteers or people with the disease or condition.

The Moleucle And Indication

ARS is a condition where the victims are exposed to high-dose radiation due to a nuclear attack or accident. PLX-R18 has already been studied by the Department of Defense to support armed forces; the molecule is being tested for treatment prior to or within the first 24 hours of radiation exposure. 

The molecule will be provided up to 96 hours after radiation exposure, with the dosage being 4 million cells per kilogram, according to the IND protocol.

"This FDA clearance is one of the most significant milestones in the development of PLX-R18 to date and should provide Pluristem with significant support in advancing its off-the-shelf cell therapy into a pivotal trial," the company said.

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