Argenica Details FDA’s Data Demands to Lift Clinical Hold on ARG-007
Argenica Therapeutics has received detailed FDA feedback outlining specific data requirements to lift the clinical hold on its ARG-007 stroke trial in the US, setting a clear path forward for its neuroprotective drug.
- FDA requests additional safety data from ongoing Phase 2 trial
- Three new in vitro studies mandated, including cardiac and genotoxicity assays
- Argenica to update investigational brochure with more Phase 1 data
- Studies to be conducted with clinical research organisations (CROs)
- Company expects to leverage September Phase 2 safety results in FDA response
FDA Clarifies Clinical Hold Requirements
Argenica Therapeutics has taken a significant step in addressing the US Food and Drug Administration’s clinical hold on its investigational new drug (IND) application for ARG-007, a promising neuroprotective peptide aimed at reducing brain damage after acute ischaemic stroke. The company has now received the FDA’s full clinical hold letter, which details the additional data and studies required to lift the hold and allow clinical trials to proceed in the United States.
Targeted Data Requests Focus on Safety Assurance
The FDA’s primary concern centers on ensuring that the proposed dosing regimen for ARG-007 can be safely administered to humans. To address this, Argenica will incorporate safety data from its ongoing Phase 2 acute ischaemic stroke trial, with results expected in September, into its submission. This data will be pivotal in demonstrating the drug’s safety profile at the intended clinical dose.
In addition, the FDA has requested three specific in vitro studies to fill identified data gaps. These include an assay on Tenecteplase (TNK), a clot-busting drug recently approved in the US, to confirm ARG-007’s interaction; a hERG assay to evaluate potential effects on cardiac potassium channels, which are critical for heart rhythm; and a mammalian cell gene mutation assay to assess genotoxicity risks. Notably, previous tests showed no genotoxicity or cardiac concerns, but these additional studies will provide further regulatory reassurance.
Efficient Path Forward with CRO Partnerships
Argenica plans to conduct these assays through reputable clinical research organisations (CROs), leveraging their expertise to expedite completion. The company’s Chief Scientific Officer has prior experience with TNK assays, which should streamline the process. Furthermore, Argenica will promptly update the investigational brochure to include more detailed Phase 1 data, a straightforward step requested by the FDA.
Strategic Implications for Argenica’s Development Timeline
While the clinical hold represents a regulatory hurdle, the clarity provided by the FDA’s letter allows Argenica to focus on targeted, manageable studies rather than broad additional trials. The company’s leadership expressed confidence that these standard assays are efficient to perform and that the upcoming Phase 2 safety data will strengthen their case. This measured approach could help minimise delays and costs, preserving momentum for ARG-007’s development.
As Argenica works closely with the FDA to ensure the adequacy of its response, investors and observers will be watching closely for the timing of study completions and the FDA’s subsequent decisions. The outcome will be critical not only for Argenica’s stroke program but also for its broader ambitions in neuroprotection and brain injury therapeutics.
Bottom Line?
Argenica’s focused response to FDA feedback sets the stage for a potential breakthrough in its US clinical development journey.
Questions in the middle?
- When will the FDA review and decide after Argenica submits the additional data?
- Could further FDA requests or delays emerge beyond the current clinical hold?
- How might the Phase 2 safety data influence investor confidence and trial timelines?
