OUR LEAD CANDIDATE
PT-112
PT-112’s mechanism of action leads to immunogenic cancer cell death (ICD) and an anti-cancer immune response, validated in experimental models and in Phase 2 patient immunoprofiling.
These effects are also delivered to bone sites of disease, due to PT-112’s bio-distribution.
Because PT-112 affects cancer cells selectively, this can be accomplished safely without common immune-related adverse events (irAEs)
Addressing unmet patient needs.
THE CHALLENGE
A minority of patients respond to I-O therapy.
And of those patients, only a small subset experience the promise of long-term benefit.
THE SOLUTION
PT-112 promotes an anti-cancer immune response.
PT-112 inhibits ribosomal biogenesis (RiBi) and selectively kills cancer cells. PT-112’s best-in-class ICD effects stimulate dendritic cells to recruit and activate T-cells to the tumor microenvironment (TME), creating the potential for effective and durable responses. Results from our published non-clinical studies validate this approach.
THE CHALLENGE
Many patients are unable to tolerate anti-cancer therapies due to toxicity.
This is not merely a problem of traditional chemotherapy or radiation, which can readily kill healthy cells. New modalities can also be limited by safety issues. This has been a matter of concern within immunotherapy, with the risk of systemic immune-related adverse events (irAEs).
THE SOLUTION
PT-112 is a safe small molecule.
Across multiple clinical trials to date, PT-112 — as a single agent and in combination — has been reasonably well tolerated in very heavily pre-treated patients. Phase 2 dose optimization efforts fostered good safety and tolerance, and led to a recommended Phase 3 dose (RP3D). By causing the cancer cell to initiate immune signaling, anti-cancer immune effects are engendered without irAEs common to immune checkpoint inhibitors, T cell engagers or cell therapies.
THE CHALLENGE
There is a need for therapies that effectively treat cancers affecting the bone.
Tumors that originate in or metastasize to the bone are difficult to treat and may cause severe pain and morbidity. This compromises quality of life and presents a drug development challenge. As an example, so-called skeletal related events (SREs) in advanced metastatic prostate cancer have been associated with a >2-fold increase in the risk of death.
THE SOLUTION
PT-112’s osteotropism allows for targeting disease in the bone.
PT-112’s pyrophosphate component has affinity for bone (osteotropism). This provides a rationale for addressing cancers affecting the bone at that site of disease. These include metastatic prostate, lung or breast cancer, and hematological malignancies such as multiple myeloma.
THE CHALLENGE
Late-stage cancer patients often have limited therapeutic options.
New approaches are needed for those patients who have exhausted available therapies, and may face multi-treatment resistance and heterogeneous disease.
THE SOLUTION
PT-112 has a novel mechanism of action with demonstrated activity in late-stage patients with heterogeneous disease.
PT-112’s inhibition of ribosome biogenesis, with its downstream anti-cancer immune effects, offers a different approach to treating such patients who have become resistant or refractory to other agents.
