Novocure is planning to file for FDA approval of its tumor-treating electric field device using results from a phase 2 pilot trial. Management wants to bring the device to market in mesothelioma via the rarely used humanitarian device exemption the FDA established to encourage rare disease research.
Optune delivers alternate electric fields to disrupt cell division. The approach is built on the fact that tubulin and septin—proteins involved in cell division—are highly polar and therefore feel the force of certain electric fields. When the proteins are exposed to fields that alternate 100,000 to 300,000 times a second, they are unable to put themselves in the positions needed to enable cell division, leading to cancer cell death.
That idea has turned Novocure into a company with a $2 billion market cap and ambitions to target a wide range of cancers. The mesothelioma program is toward the back of Novocure’s clinical pipeline but the company thinks the rarity of the disease will enable it to leapfrog the rest of the pack.
Novocure’s plan is enabled by the FDA’s humanitarian device exemption, which lowers the evidence bar for products aimed at diseases that affect 8,000 or fewer U.S. patients a year. Around 3,000 new cases of mesothelioma are diagnosed each year. The upshot is Novocure may be able to bring the device to market by providing the FDA with reasonable assurance it is safe and by showing probable efficacy.
Having seen data from an 82-patient phase 2 that combined Optune with a chemotherapy regimen based on Eli Lilly's Alimta, Novocure thinks it can clear that bar.
The company has yet to share a look at the data, saying only that they are better than interim results from 2016 and show “clinically meaningful” improvements in overall and progression-free survival. The interim data linked Optune to a median PFS of 7.3 months and a one-year survival rate of 80%. Novocure compared the results favorably to its chosen historical controls, which reported figures of 5.7 months and 50%, respectively, in patients who received chemotherapy alone.
The trial was yet to reach median OS by the interim review cutoff. But given the Alimta phase 3 trial Novocure took the one-year survival rate from achieved an OS of 12.1 months, Optune will need to keep patients alive for more than a year to support efficacy claims.
Shares in Novocure rose 8% following the news.