US-based Dicerna Pharmaceuticals has dosed the first patient in its Phase Ib/II clinical trial of DCR-MYC, an investigational Dicer substrate short interfering RNA (DsiRNA) therapeutic to treat advanced hepatocellular carcinoma (HCC).
The new Phase Ib/II trial expands the development programme for DCR-MYC, which is also being evaluated in a Phase I trial in patients with solid tumours and hematological malignancies.
DCR-MYC targets the driver oncogene MYC, which is essential to the growth of many tumour types, including HCC.
Dicerna chief medical officer Dr Pankaj Bhargava said: "MYC is a highly relevant target in many cancers, including HCC, but remains challenging to drugs with conventional modalities.
"The potential of inhibiting the MYC pathway with DsiRNA therapeutics is very exciting and could be of tremendous therapeutic value for patients."
A total of 72 patients with locally advanced unresectable or metastatic HCC will be enrolled in the multi-centre, open-label trial at sites in the US, South Korea and Singapore.
The Phase Ib portion of the trial is designed to evaluate the safety, tolerability, maximum tolerated dose and recommended Phase II dosing of DCR-MYC.
The trial’s Phase II portion will evaluate preliminary antineoplastic activity of DCR-MYC in addition to safety and tolerability.
Harvard Medical School associate professor of medicine, Massachusetts General Hospital Cancer Center director of liver cancer research and an investigator in the DCR-MYC Phase Ib/II trial Andrew Zhu said: "The incidence of HCC is on the rise in the US and Europe, primarily due to the prevalence of chronic hepatitis C infection.
"Patients with advanced HCC have limited treatment options, and there are no approved therapies for those who are intolerant of, or progress after treatment with sorafenib."
Patients who do not respond to or do not tolerate standard-of-care sorafenib therapy, or patients who lack access to sorafenib or for whom no suitable therapy is available will only be enrolled in this trial.
During the trial, DCR-MYC will be administered by two-hour intravenous infusion (IV) once a week for two weeks, followed by a week of rest, with the potential to evaluate dosing every other week.
Image: Intermediate magnification micrograph of hepatocellular carcinoma the most common form of primary liver cancer. Photo: courtesy of Nephron.