Conforma Therapeutics Corp., which has focused on oncology products targeting heat-shock protein 90 since forming in 2001, late last month added a synthetic anthracycline compound to its pipeline for the treatment of small-cell lung cancer.
The company licensed Amrubicin HCL from Osaka, Japan-based Sumitomo Pharmaceuticals Co. Ltd., which won Japanese marketing approval for the product, sold as Calsed, in both small-cell lung cancer and non-small-cell lung cancer in late 2002. Conforma gains exclusive rights to develop and market the product in North America and Europe.
"For us, this is really an opportunity to expand our portfolio into oncology, beyond our founding technology of Hsp90," said Chris LeMasters, vice president of corporate development for Conforma, "and to take advantage of this clinical development capability that we've built."
Specific terms were not disclosed, but LeMasters said Conforma's early investment will center on its overall development plan for Amrubicin, rather than the up-front and milestone payments involved in typical licensing deals. Sumitomo will be responsible for manufacturing Amrubicin, so the back-end of the deal will be focused primarily on a supply price arrangement, instead of royalties.
"That communicated to us the continued collaborative nature" of the deal, he said, adding that it preserves the company's existing resources to begin developing Amrubicin.
Conforma plans to begin Phase II trials in small-cell lung cancer early next year, after filing an investigational new drug application. The IND will include safety and efficacy data compiled by Sumitomo, which received marketing approval after completing a 33-patient Phase II study.
"In Japan, cancer products are often approved with Phase II trials," LeMasters said, adding that the Japanese regulatory agency also looks for extensive post-marketing data. "That bodes well for us because we'll be able to work collaboratively to take advantage of each other's data."
Amrubicin is a third-generation synthetic anthracycline that has demonstrated antitumor activity and has been administered to about 2,000 patients without causing the cardiotoxicity commonly seen in the anthracycline class of compounds, he told BioWorld Today.
Sumitomo also is investigating the drug in non-Hodgkin's lymphoma, and LeMasters said Conforma might consider additional indications for the drug beyond small-cell lung cancer.
Amrubicin will join CNF1010 as the company's most advanced products. The first candidate developed using Conforma's Hsp90 technology, CNF1010, is expected to enter Phase II trials late this year or early next year. Hsp90 is described as a family of molecular chaperones that control protein shape and affect key signaling molecules involved in the growth and survival of tumor cells. The company's first synthetic Hsp90 inhibitor, CNF2024, is set to enter clinical trials soon.
"Our chemists are really in the sweet spot right now with this target," LeMasters said of Hsp90, "so we'll continue to keep our eye in that area."
That target also has potential in applications outside of oncology, such as autoimmune, inflammatory and neurodegenerative diseases.
In early May, Conforma completed the final tranche of its Series C financing, which raised a total of $41.5 million. The last tranche itself netted the company about $11 million. (See BioWorld Today, May 3, 2005.)
"That was not completely coincidental," LeMasters said. "We shared our vision with [Amrubicin] for investors, and their enthusiasm, coupled with their support of our Hsp90 program, helped us in closing that extension."
Although he acknowledged that the Amrubicin deal is "a little unusual," given that Conforma was just as concerned about the product's development plan as it was the product's mechanism of action, he said the approach might work best for small biotech firms that can't afford hefty in-licensing fees.
"I think it will be the future of biotech to come up with a vision that, perhaps, no else has seen," he said.