Pendrea’s approach to cancer therapy is highly innovative and quite different from the technologies used by other companies. Our leading product development programs, Ceraxa and PINT, both have dual mechanisms of action that combine to make them highly effective. Our 3rd anti-cancer technology, RNA Therapeutics, is a versatile and highly efficient means of stimulating the immune system to attack cancer cells. It can also be used to deliver RNA coding for therapeutic proteins.
Ceramides are class of natural compounds that are central mediators of the body’s natural defense mechanism against neoplasia and cancer progression. Pendrea’s lead product, Ceraxa, is a proprietary, fully synthetic formulation of ceramide embedded in the outer membrane of a nanoliposome. Ceraxa, which is the only known method to deliver ceramide pharmacologically, delivers the active form of ceramide (C-6) intracellularly and without any significant side effects. Moreover, Ceraxa works in combination with numerous chemotherapeutic products to improve and/or restore efficacy of standard of care chemotherapeutics. Ceraxa is also a unique and
powerful immuno-oncology agent that complements already approved immuno-oncology technologies such as checkpoint inhibitors and Car-T to improve their efficacy. Ceraxa has completed a Phase 1 clinical study and is ready to begin Phase 2 testing.
Results include tumor regression and an 85% increase in survival in a humanized, fibrogenic model
of hepatocellular carcinoma (HCC or Liver Cancer) as a monotherapy. They also include 67% disease
free survival when used in combination with Vinblastine in AML, as well as dramatic improvement of
efficacy with other drugs in pancreatic and prostate as well as other cancers. They also include
improved efficacy in combination with Immuno-Oncology agents.
Photo Immuno Nano Therapy (PINT)
PINT repurposes a well-known human imaging agent, Indocyanine Green, for therapeutic use by
encapsulating it in highly biocompatible calcium-phosphate nanoparticles (NanoJackets or NJs).
PINT operates by a dual mechanism of action: (1) intracellular delivery of ICG encapsulated in
NanoJackets (NJs) and irradiated by harmless Near Infrared (NIR) light results in direct killing of
cancer cells by extremely high efficiency Photo Dynamic Therapy (PDT). (2) PINT generates a
sphingosine kinase that reduces inflammation in the tumor microenvironment triggering T-cell
attack against the targeted tumor.
Results include 29% disease free survival in leukemia (CLL), complete cessation of Breast cancer
growth with one dose, and active targeting of leukemic stem cells and pancreatic cells via
bioconjugation of target specific ligands to the NJ surface.
Pendrea’s NanoJackets offer a versatile and highly efficient means of delivering small interfering
RNA (siRNA), micro RNA (miRNA) and messenger RNA (mRNA) to the interior of the cell.
NanoJackets protect the active agent while in the bloodstream and release the RNA payload upon
pH change inside the cell. NanoJackets can be actively targeted to specific tumor types via
bioconjugation of different ligands on their surface. The RNA encapsulated in the NJs can code for
therapeutic and prophylactic vaccines as well as therapeutic proteins.
- siRNA-NJs -- include 98% knockdown of target proteins, and 80% tumor regression in breast
- miRNA-NJs -- an actively targeted micro RNA formulation achieved 4 times higher
concentration of the expressed protein in the lung than a chemically modified micro RNA
- mRNA-NJs -- high levels of protection of mRNA from enzymatic degradation and confirmed
mRNA functionality via expression after mRNA release from NJs in cells.