GlaxoSmithKline and Synta announce elesclomol granted US orphan drug designation by the FDA

GlaxoSmithKline (GSK) and Synta Pharmaceuticals Corp. (NASDAQ: SNTA) today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to elesclomol (formerly STA-4783) for the treatment of patients with metastatic melanoma. Elesclomol is being developed under a global collaboration agreement between Synta and GSK. Elesclomol is an investigational drug that is not approved for any indication in any market at this time.


Orphan drug status is designed to encourage biotechnology and pharmaceutical companies to develop drugs for rare diseases which affect fewer than 200,000 people in the United States. In November 2006 elesclomol received Fast Track designation from the FDA for development in metastatic melanoma.


“We are pleased that the FDA granted elesclomol orphan drug status for the treatment of metastatic melanoma,” said Eric Jacobson, M.D., Senior Vice President and Chief Medical Officer, Synta Pharmaceuticals. “With the incidence of melanoma increasing more rapidly than any other cancer during the past ten years, there is a significant need for innovative therapies such as elesclomol.”


“Orphan drug status is an acknowledgment of the significant need to develop new therapies for patients with metastatic melanoma, a disease for which there are few treatment options,” said Paolo Paoletti, Senior Vice President of the OncologyMedicineDevelopmentCenterat GSK. “Through the development of products like elesclomol, GSK Oncology is reaffirming its commitment to address clinical needs in cancer treatment and improve the lives of patients.”


About elesclomol (formerly STA-4783)

Elesclomol is a novel, injectable, investigational drug candidate that is believed to kill cancer cells by elevating oxidative stress levels beyond a breaking point, triggering programmed cell death. This mechanism of action, called oxidative stress induction, represents a novel way of selectively killing cancer cells.


A pivotal Phase 3 clinical trial of elesclomol in combination with paclitaxel in metastatic melanoma (the SYMMETRYSM trial) was initiated in October 2007 and Phase 2 trials in other indications, and in combination with other agents, are planned. Information about the SYMMETRY trial can be found at www.clinicaltrials.gov.


In a double-blind, randomised, controlled Phase 2b clinical trial in 81 patients with metastatic melanoma, elesclomol in combination with paclitaxel met the primary endpoint, doubling the median time patients survived without their disease progressing, compared to paclitaxel alone (p = 0.035). The most common adverse events in the elesclomol plus paclitaxel group included fatigue, alopecia, constipation, nausea, hypoaesthesia, arthralgia, insomnia, diarrhoea, and anaemia.


About US orphan drug status

The Orphan Drug Act (ODA) provides economic incentives to encourage biotechnology and pharmaceutical companies to develop drugs for rare diseases which affect fewer than 200,000 people in the United States. Orphan drug designation entitles GSK and Synta to seven years of market exclusivity for elesclomol for the treatment of patients with metastatic melanoma. Additional incentives for orphan drug development include tax credits related to development expenses, reduction in FDA user fees and FDA assistance in clinical trial design.


About metastatic melanoma

The incidence of melanoma has increased more rapidly than any other cancer during the past ten years. According to the American Cancer Society, melanoma accounts for approximately five percent of all skin cancers but causes about 75% of all skin cancer-related deaths. An estimated 60,000 people will be diagnosed and nearly 8,200 people will die from melanoma this year in the USalone. If diagnosed and surgically removed while localised in the outermost skin layer, melanoma is potentially curable; however, for patients with metastatic disease, the prognosis is poor. Treatments are limited and the expected survival for patients with metastatic melanoma is only six to nine months.


About GlaxoSmithKline

GlaxoSmithKline – one of the world's leading research-based pharmaceutical and healthcare companies – is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For company information, visit GlaxoSmithKline at www.gsk.com.


GSK Oncology is dedicated to producing innovations in cancer that will make profound differences in the lives of patients. Through GSK’s “bench to bedside” approach, we are transforming the way treatments are discovered and developed, resulting in one of the most robust pipelines in the oncology sector. Our worldwide research in oncology includes collaborations with more than 160 cancer centers. GSK is developing a new generation of patient-focused cancer treatments in prevention, supportive care, chemotherapy and targeted therapies.




About Synta Pharmaceuticals

Synta Pharmaceuticals Corp. is a biopharmaceutical company focused on discovering, developing, and commercialising small molecule drugs to extend and enhance the lives of patients with severe medical conditions, including cancer and chronic inflammatory diseases. Synta has a unique chemical compound library, an integrated discovery engine, and a diverse pipeline of clinical- and preclinical-stage drug candidates with distinct mechanisms of action and novel chemical structures. AllSynta drug candidates were invented by Synta scientists using our compound library and discovery capabilities. Synta has a partnership with GlaxoSmithKline for the joint development and commercialisation of elesclomol. For more information, please visit www.syntapharma.com.