Denali Therapeutics has started dosing of subjects in a Phase I/II clinical trial of DNL593 to treat frontotemporal dementia-granulin (FTD-GRN).

Developed by the company’s Protein Transport Vehicle (PTV) technology, DNL593 is an investigational, brain-penetrant recombinant progranulin (PGRN) replacement therapy.

Denali entered a strategic collaboration with Takeda to jointly develop and commercialise DNL593, which is intended to slow or prevent FTD-GRN progression by increasing intracellular and extracellular functional PGRN levels.

The randomised, multicentre, placebo-controlled, double-blind Phase I/II study is designed to evaluate DNL593’s pharmacokinetics, pharmacodynamics, tolerability, and safety in healthy subjects and FTD-GRN patients.

Denali Therapeutics chief medical officer Carole Ho said: “Denali has a diverse portfolio of therapeutic candidates to address neurodegenerative and lysosomal diseases, and DNL593 is our second Transport Vehicle (TV) technology-enabled programme to enter clinical development.

“Initiation of this Phase I/II trial is an important development milestone for the programme, and we look forward to continued collaboration with Takeda and the FTD community in our unified purpose to develop DNL593 as a treatment option for people and families living with FTD-GRN.”

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The trial is a three-part (Parts A-C) study, and subject dosing has commenced in the single ascending dose Part A in healthy participants.

The multiple dose, 25-week Part B study will be conducted in FTD-GRN patients, and the 18-month open-label Part C study is the extension of FTD-GRN patients who participated in Part B.

FTD-GRN is characterised by a deficiency of progranulin (PGRN) in the brain. In 2019, Denali commenced patients’ dosing in a Phase Ib clinical trial of DNL151 to treat Parkinson’s disease.