AAA Gets $57M for Pivotal Cancer Drug Trial, Acquisition

BioWorld International Correspondent

Advanced Accelerator Applications SA (AAA) closed a €40 million (US$57.4 million) funding round that will enable the company to move its lead drug candidate Lutathera (lutate; ¹⁷⁷Lutetium DOTA⁰-Tyr³-Octreotate) into pivotal trials in cancer later this year and to complete the acquisition of an as yet undisclosed firm that is developing a drug for ovarian cancer.

AAA, based in Saint Genis Pouilly, France, has raised €74 million since its inception and is backed by more than 100 investors, including private investors, private companies and institutional shareholders. Milan, Italy-based pharmaceutical firm Domp Group participated in the latest round and now holds a 7.7 percent stake in AAA. The company now has a post-money valuation of more than €200 million, founder and CEO Stefano Buono told BioWorld International.

AAA, a nuclear medicine specialist, has a pretty unusual background for a European life sciences firm. It was spun out from CERN, the Geneva-based European Organization for Nuclear Research, in 2002 with a patent for producing radioactive elements using a particle accelerator. Buono worked for 10 years with Nobel prizewinner Carlo Rubbia, long one of Europe's most prominent scientists.

The company has built a profitable business around the production of radiolabeled tracers and diagnostics. It posted revenues of €27.4 million last year and a net profit of €1.7 million. It gained ownership of Lutathera in 2010 via a stock-based acquisition of St. Louis-based Biosynthema Inc., and it is planning to move the drug, a radiolabeled analogue of the peptide hormone somatostatin, into a Phase III trial in patients with metastatic gastro-entero-pancreatic neuroendocrine tumors (GEP-NETs) before year-end.

Lutathera works by targeting somatostatin receptors, which are overexpressed in many neuroendocrine tumors. It has already completed an investigator-sponsored study at the Erasmus Medical Center, Rotterdam, the Netherlands, where more than 600 patients have received the therapy.

A paper published in the May 1, 2008, issue of the Journal of Clinical Oncology, titled "Treatment With the Radiolabeled Somatostatin Analog [¹⁷⁷Lu-DOTA⁰,-Tyr³] Octreotate: Toxicity, Efficacy, and Survival," reported that the therapy resulted in median overall survival of 46 months from the start of therapy or 128 months from diagnosis. Compared with historical controls, the authors claimed an overall survival benefit of 40 to 72 months from diagnosis.

Lutathera, which has orphan drug status in its lead indication, has potential in any tumor characterized by overexpression of somatostatin receptors, Buono said.

"Currently it's also used for thyroid tumors that are not responsive to iodine," he said. It could also be used in certain breast cancers, Buono added. "It's a long way for a small company. That's why we really need to concentrate on getting [our first] market authorization before we start thinking about other indications," he said.

The company is aiming for an approval by 2016, and it plans to produce and commercialize Lutathera itself, as the number of centers equipped to practice nuclear medicine is limited, Buono said. "Also, it's a very well-known procedure; it's already being done in academia," he added.

The company is seeking parallel scientific advice from the FDA and the European Medicines agency in advance of the upcoming trial, which will recruit around 300 patients through 40 centers throughout the U.S. and Europe.

Published: July 27, 2011