The Multiple Myeloma Research Consortium(MMRC), a 509(a)3 non-profit organization that integrates leading academic institutions to accelerate drug development in multiple myeloma, has started clinical trial evaluating ganetespib, a second generation Hsp90 inhibitor being developed by Synta Pharmaceuticals, as a single agent and in combination with the proteasome inhibitor bortezomib (Velcade) for the treatment of relapsed multiple myeloma.

The trial, made possible by the funding up to $1 million by the Multiple Myeloma Research Foundation (MMRF), will be conducted through the MMRC. The MMRC is a consortium of sixteen world-renowned academic institutions and community centres whose mission is to accelerate the development of novel and combination treatments for patients with multiple myeloma by promoting and facilitating collaborative research between industry and academia.

Funding for this trial is made possible by the donor-supported MMRF Clinical Fund, which enables the MMRF to invest in the development of industry-owned compounds that have stalled in multiple myeloma development due to financial or market constraints, but have potential for treating this disease.

“Ganetespib has already demonstrated clear signals of single agent activity in several tumour types and a favourable safety profile. We believe that patients with multiple myeloma may also benefit from treatment with an Hsp90 inhibitor such as ganetespib,” said Sagar Lonial, M.D., Winship Cancer Institute of Emory University and principal investigator on the phase 1 trial. “An earlier phase 1b clinical trial of the first generation Hsp90 inhibitors 17AAG and 17DMAG in combination with bortezomib in multiple myeloma demonstrated signs of activity in patients who had experienced a median of four prior therapies. Based on these results, we believe that ganetespib, a second generation, small molecule inhibitor of Hsp90 may provide benefit to patients with multiple myeloma.”

“Our continued investment in drug development, whether through our annual Biotech Investment Awards or through our new Clinical Fund projects with biopharmas like Synta, signify the MMRF’s continued commitment to share in the risk of drug development to ensure promising treatments are brought to patients as quickly as possible,” said Kathy Giusti, Founder and CEO of the MMRF and MMRC, and a multiple myeloma patient.

“Working with the MMRF and the MMRC provides both the resources and the access to top-tier investigators and clinical trial sites that can be of tremendous help in accelerating the potential of novel therapies, such as ganetespib, to benefit patients with multiple myeloma,” said Safi Bahcall, Ph.D., president and CEO, Synta Pharmaceuticals. “We are excited to begin this partnership.”

A recent study showed that the MMRC enrolls patients 10% faster when compared to their baseline enrollment timeline, with 67% of trials meeting their pre-study enrollment commitment 34%, or 4.5 months faster than their baseline enrollment timeline.

Ganetespib is the most advanced of the next-generation, synthetic Hsp90 inhibitors with more than 500 patients treated to date; 20 trials completed, ongoing, or initiating; and the Phase 2b/3 GALAXY Trial in second-line non-small cell lung cancer (NSCLC) active in the US and Europe.

Ganetespib has shown anti-tumour activity in heavily pretreated patients with lung cancer, breast cancer, and other tumour types and has been well tolerated with no evidence of severe liver or common ocular toxicities seen with other Hsp90 inhibitors. The most common adverse event seen to date has been diarrhoea, which has been manageable with standard supportive care.

Hsp90 is a molecular chaperone required for the proper folding and activation of many cancer-promoting proteins, and is recognized as a key facilitator of cancer cell growth and survival. Many of the “client proteins” of Hsp90 – such as ALK, AKT, BCR-ABL, BRAF, KIT, MET, EGFR, FLT3, HER2, PDGFRA, VEGFR are the targets of clinically validated cancer drugs. In preclinical studies, inhibiting Hsp90 causes the degradation of multiple client proteins and leads to cancer cell death.

Multiple Myeloma Research Foundation (MMRF) was established in 1998 as a 501(c)3 non-profit organization by twin sisters Karen Andrews and Kathy Giusti, soon after Kathy's diagnosis with multiple myeloma. The mission of the MMRF is to relentlessly pursue innovative means that accelerate the development of next-generation multiple myeloma treatments to extend the lives of patients and lead to a cure. As the world's number-one private funder of multiple myeloma research, the MMRF has raised over $190 million since its inception to fund nearly 120 laboratories worldwide, including 70 new compounds and approaches in clinical trials and pre-clinical studies and has facilitated more than 30 clinical trials through its affiliate organization, the Multiple Myeloma Research Consortium (MMRC). As exceptional stewards of its donors' investments, the MMRF has been consistently recognized for its sound fiscal management.

Synta Pharmaceuticals Corp. is a biopharmaceutical company focused on discovering, developing, and commercializing small molecule drugs to extend and enhance the lives of patients with severe medical conditions, including cancer and chronic inflammatory diseases.