Lead candidate is in a Phase II AML trial and targets FLT3.
Ambit Biosciences completed a Series D-2 round of equity financing, raising $30 million in new capital. The company is developing small molecule kinase inhibitors for the treatment of cancer and other conditions. The funding allows the firm to withdraw plans for an initial public offering, according to Alan Lewis, president and CEO.
“This recent capital raise will allow us to continue the clinical development of our lead product candidate, AC220, which is currently in a Phase II pivotal trial in acute myeloid leukemia (AML),” says Lewis. “This financing also provides for the continued development of our other two clinical assets, AC430 and AC480, both of which are in Phase I trials.”
AC220 is a potent, highly selective, orally bioavailable FMS-like tyrosine kinase-3 (FLT3) inhibitor. The molecule is currently under evaluation in a Phase II trial as monotherapy for adult and elderly patients with relapsed/refractory AML that have an internal tandem duplication (ITD) mutation in the FLT3 gene.
ITD mutations in the FLT3 gene occur in 25–30% of AML patients, according to Ambit. FLT3 ITD mutations confer poor prognosis, with early relapse and lower survival following treatment with existing therapies including chemotherapy and hematopoietic stem cell transplant.
The drug is being developed in collaboration with Astellas Pharma, which paid $40 million up front for rights to all indications in December 2009. AC220 is in preclinical studies against melanoma and gastrointestinal stromal tumor.
Besides this compound, the companies will also work together on R&D of additional FLT3 inhibitors. Precommercialization milestone fees under the worldwide development and commercialization deal could reach $350 million. The firms will share equally in the responsibilities and expenses of developing all products in the U.S. and Europe. Astellas has sole responsibility to fund development in all other territories.
Astellas will handle commercialization of all products, including funding. In the U.S. Ambit will also have the option to co-promote AC220 and other products. If exercised, Astellas and Ambit will share equally in profits and losses generated from U.S. sales.
Ambit’s pipeline has two more clinical-stage small molecules. AC480, a pan-Her inhibitor, is in early- and mid-stage trials as a treatment for various cancers including non-small-cell lung cancer, breast cancer, glioblastoma multiforme, and brain metastases. AC430, an oral JAK2 inhibitor, is in Phase I trials in autoimmune diseases and lymphomas. Ambit’s pipeline of preclinical candidates includes CEP-32496, a BRAF inhibitor licensed to Cephalon.
