-- Plenary Presentation at the 15th Inter­na­tional Myeloma Workshop --

Toronto, ON and San Diego, CA (Press Release) – Triphase Accelerator Corpo­ra­tion, a private drug de­vel­op­ment com­pany dedicated to ad­vanc­ing novel com­­pounds through Phase 2 proof-of-concept, today an­nounced pre­lim­i­nary results of a Phase 1 dose-escalation study of marizomib in com­bi­na­tion with poma­lido­mide (POM) and low dose dex­a­meth­a­sone (Lo-DEX) in patients with re­lapsed and refractory multiple myeloma. The marizomib/POM/Lo-DEX com­bi­na­tion was able to de­crease myeloma proteins by at least 50 per­cent to achieve an over­all response rate of 62 per­cent in a patient pop­u­la­tion that was refractory to multiple prior ther­a­pies. Marizomib, a novel and highly potent pan-proteasome inhibitor, POM and DEX were generally well tolerated, with no dose-limiting toxicities observed at the four dose levels tested. The study was one of only six studies highlighted during a special plenary session at the Inter­na­tional Myeloma Society’s 15th Inter­na­tional Myeloma Workshop in Rome, Italy.

“Nearly all patients with re­lapsed and refractory multiple myeloma will eventually relapse on cur­rently avail­able ther­a­pies, underscoring the importance of de­vel­op­ing new treat­ment options,” said Professor Andrew Spencer, lead author, Head of the Malignant Hematology & Stem Cell Transplantation Service at The Alfred Hospital and Professor of Hematology at Monash University in Melbourne, Australia. “Marizomib does not appear to add toxicity to the estab­lish­ed POM/DEX safety profile, and the com­bi­na­tion of drugs dem­onstrated encouraging activity in some of the sickest patients with multiple myeloma. I believe that this com­bi­na­tion of drugs holds great promise for the treat­ment of multiple myeloma.”

“We are encouraged by the results of this Phase 1 study and are work­ing diligently with our clin­i­cal sites and Celgene Corpo­ra­tion to accelerate the clin­i­cal devel­op­ment of marizomib,” said Mohit Trikha, Ph.D., Chief Scientific Officer and Head of R&D at Triphase Accelerator. “Now that the dose-escalation stage of the study has been com­pleted, we are enrolling patients in a larger dose-expansion stage to select the dosing regi­men for larger-scale clin­i­cal studies.”

Study Design and Key Results

The multi­center, open-label, dose-escalation study eval­u­ated the com­bi­na­tion of in­tra­venous marizomib plus oral POM and Lo-DEX in patients with re­lapsed and refractory multiple myeloma. All patients had received at least two prior myeloma ther­a­pies, which must have in­cluded the approved myeloma drugs, lena­lido­mide and bor­tez­o­mib, and the patients must have been refractory to their last myeloma ther­apy. The study was conducted at the Dana Farber Cancer Institute in Boston, the University of Maryland Medical Center in Baltimore, the Karmanos Cancer Center in Detroit, and the Peter MacCallum Cancer Centre and the Alfred Hospital in Australia. The Multiple Myeloma Research Consortium also sup­ported this research.

Results for the 13 patients evaluable for response in­cluded 8 (62 per­cent) with a partial response, 2 (15 per­cent) with a minimal response, and 3 (23 per­cent) with stable disease. All of the patients had a de­crease in their myeloma protein by the end of the first treat­ment cycle. The most common Grade 3 (severe) adverse events related to at least one of the study treat­ments were neu­tro­penia, anemia and thrombo­cytopenia.

About Marizomib

Marizomib is a novel and highly potent pro­te­a­some inhibitor that irreversibly binds and inhibits all three pro­te­a­some subunits, which translates into longer duration of effect and poten­tially im­proved clin­i­cal activity. Triphase Accelerator is devel­op­ing marizomib in both in­tra­venous and oral formulations as a poten­tial best-in-class pro­te­a­some inhibitor for hema­to­logic ma­lig­nan­cies and solid tumors. The IV formulation has been eval­u­ated in more than 270 patients in six clin­i­cal studies in patients with solid and hema­to­logic ma­lig­nancies, either as a single agent or in com­bi­na­tion with dex­a­meth­a­sone, a histone deacetylase inhibitor, or an immuno­modu­la­tory drug.

Triphase Accelerator is cur­rently eval­u­ating marizomib in a Phase 1 proof-of-concept clin­i­cal study in com­bi­na­tion with bevacizumab (Avastin^®) in patients with Grade IV malignant glioma (glioblastoma). Triphase Accelerator is also eval­u­ating an oral formulation in pre­clin­i­cal studies. The Company has received Orphan Drug desig­na­tion for marizomib in multiple myeloma in the United States and the European Union.

About Triphase Accelerator

Triphase Accelerator is a private drug devel­op­ment com­pany with a pri­mary focus on on­col­ogy and with operations in Toronto and San Diego. Triphase Accelerator is dedicated to ad­vanc­ing novel com­­pounds through Phase 2 proof-of-concept clin­i­cal studies using a unique, science-based, high-quality model that is faster and more cost-effective than traditional pharma­ceu­tical and bio­tech industry drug devel­op­ment ap­proaches. Triphase Accelerator was spun out of the Ontario Institute for Cancer Research (OICR), with sup­port from the Fight Against Cancer Innovation Trust (FACIT), MaRS Innovation and MaRS. It has a stra­te­gic rela­tion­ship with Celgene for on­col­ogy-focused drug devel­op­ment oppor­tu­ni­ties. For more in­for­ma­tion, visit www.triphaseco.com or Linkedin.

Source: Triphase Accelerator.