Amgen, Sanofi back a round for real-world evidence platform tech; Daiichi Sankyo's pexidartinib secures speedy FDA review
→ Aetion has garnered $27 million from a new venture round that includes some of the biggest names in healthcare. The company plans to use the cash to build up its platform of real-world evidence data, a burgeoning field where patient outcomes can inform drug makers and marketers what works, when. The round was backed by Amgen Ventures, Sanofi, UCB, McKesson Ventures, Horizon Health Services and others.
→ Despite the hiccup of two cases of serious liver toxicity in the late-stage trial of Daiichi Sankyo‘s “breakthrough” drug pexidartinib in treating rare cases of tenosynovial giant cell tumor (TGCT) among patients who did not qualify for surgical resection, trial data were deemed compelling enough for the oral treatment to qualify for the FDA’s priority review. There are no currently approved systemic therapies for TGCT, the Japanese drugmaker said, noting that the FDA is now expected to announce its decision in six months (PDUFA: August 3) instead of the standard 10 months.
→ Moleculin Biotech has secured the FDA’s orphan drug status for its early-stage experimental drug, WP1066, to treat glioblastoma — an aggressive form of brain cancer that killed Senators John McCain and Ted Kennedy. The drug, forms part of a new class of drugs called “Immune/Transduction Modulators” that in preclinical testing have shown to stimulate a natural immune response to tumors and directly attack tumor cells, according to Moleculin. The company’s shares $MBRX jumped about 7.4% in early trading.