Aton Pharma, Inc., a privately-held biopharmaceutical company, announced that it had presented results from the oral phase 1 clinical trial on Saha, its lead product candidate, at the AACR/NCI/EORTC international conference on "Molecular Targets and Cancer Therapeutics" in Boston, Massachusetts.
The company also announced that it has entered into a clinical trials agreement with the Cancer Therapy Evaluation Program (CTEP) of the National Cancer Institute (NCI) to collaborate on the clinical development of Saha.
Nicholas G Bacopoulos, PhD, president and CEO of Aton said, "Aton is making important contributions to the clinical exploration of histone deacetylase inhibitors in cancer patients. We are excited about the encouraging results from our Phase I trial, as well as the growing interest in Saha shown by CTEP, the organizers of this important conference, and the oncology community in general. The results reported by our academic collaborators support our continued efforts to bring SAHA to cancer patients as expeditiously as we can."
Aton is conducting its comprehensive Phase 1 trial in patients with refractory solid tumours, lymphomas, and leukemias in collaboration with W Kevin Kelly, DO and other leading investigators at Memorial Sloan-Kettering Cancer Center (MSKCC). The primary objective of the study is to define a safe daily oral dosing regimen for patients with either solid tumours or hematological malignancies. Secondary objectives include evaluation of the pharmacokinetic profile of oral Saha, determination of the oral bioavailability of Saha in a fasting and non-fasting state, assessment of the biological effects of Saha on normal and malignant tissue, and documentation of anti-tumour effects of the treatment.
Study investigators reported a reduction in measurable disease among some patients with solid tumours as well as hematologic malignancies (including one complete remission). Side effects consisted mainly of the constitutional symptoms of diarrhea, anorexia, dehydration and fatigue. However, the effects subsided after the patients stopped taking the drug.
"We continue to be encouraged by the strong evidence of anti-tumour activity in both hematologic malignancy and solid tumour patients. The collaboration with CTEP, in addition to our ongoing phase 2 program, will allow us to further evaluate the safety and efficacy of Saha" said Carolyn Paradise, MD, senior vice president and CMO.
The agreement with CTEP supports development of SAHA in several clinical trials both as a single agent and in combination with other therapies. CTEP is soliciting for a single-agent pediatric Phase 1 study of Saha and single-agent Phase 2 studies. Program priorities for the initial round of phase 2 studies include non-Hodgkin's lymphoma, renal cell carcinoma, bladder cancer, breast cancer, lung cancer, mesothelioma, glioma, and thyroid cancer. CTEP is also soliciting for Phase 1 studies of Saha in combination with 5-fluorouracil (5-FU) and other possible agents which may include anthracycline, platinum, taxane, imatinib mesylate, retinoids, trastuzumab, gemcitabine, hypomethylating agents, radiation therapy, and agents that block the function of Bcl-2. Priority will be given to proposals with correlative studies directed at gaining insight into the molecular mechanisms involved in the induction of apoptosis in tumours by Saha.
Victoria Richon, PhD, co-founder of Aton Pharma, Inc and executive director of biology, will also give an invited presentation at the AACR/NCI/EORTC International Conference entitled "Development of Histone Deacetylase Inhibitors", as part of a plenary session tomorrow on "Emerging Targets in the Clinic".