Novartis ($NVS) said its in-development skin cancer drug aced a Phase II study, completely eradicating the disease in some patients, and now the company plans to loop in regulators, possibly signaling an early FDA application for the treatment.
The drug, LDE225, is an oral therapy for basal cell carcinoma, which Novartis said accounts for more than 80% of non-melanoma cancers. In a mid-stage study, the treatment met its primary efficacy endpoint of objective response rate at 6 months, Novartis said, including complete response, in which patients are left with no signs of cancer, and partial response, wherein subjects chart clinically significant tumor shrinkage. In keeping with industry norms, Novartis is withholding the study's full data with plans to present them at a later scientific meeting.
Those top-line results show a lot of promise for a patient population with limited treatment options, and Novartis oncology president Alessandro Riva said in a statement that the company is pleased with LDE225's potential and "look(s) forward to sharing these data with regulatory authorities worldwide."
It's unclear whether that means Novartis plans to seek FDA approval based on Phase II results alone, and a spokesman for the notoriously opaque drugmaker said only that "these data could serve as the basis for future regulatory filings." That said, LDE225 does appear in the 2014 column of Novartis' list of planned filings.
The drug, which also has applications in brain cancer and solid tumors, is among 10 pipeline treatments and new indications Novartis' ambitious oncology division expects to launch by 2017. That list includes LDK378 for non-small cell lung cancer, a breakthrough-designated therapy for which Novartis hopes to win approval off of Phase II data alone.
LDE225 works by inhibiting a receptor called smoothened, which regulates the hedgehog signaling pathway, thereby blocking tumor growth.
- read the statement