AstraZeneca, Merck win red carpet treatment at the FDA as a 3-time loser seeks speedy OK for rare tumors
AstraZeneca’s R&D group doesn’t take failure lying down.
Hit with pivotal setbacks for their MEK 1/2 inhibitor selumetinib in several cancer studies, they regrouped and included it in their $8.4 billion alliance with Merck as the therapy was steered into a Phase II for rare cases of neurofibromatosis type 1, or NF1.
That data gave the pharma giant a breakthrough drug designation in the spring, and now they are on track for a Q2 2020 PDUFA after the FDA added a priority review to the gift package today.
By inhibiting the MEK enzyme in the RAS/MAPK pathway investigators have demonstrated that the drug can control errant signaling that controls cell growth — at least relevant to NF1.
Back in 2018, when Ned Sharpless was running the National Cancer Institute — and where he’s headed back after a stint as acting director of the FDA — he held up the research work on selumetinib to a House committee as one of the most important projects in their cancer moonshots program, citing the before-and-after pictures from parents with kids suffering from inoperable tumors sparked by the disease.
According to a report from the Children’s Tumor Foundation, which invested in the preclinical work, investigators tracked a 70% response rate, with tumors shrinking at least 20%.
Five years ago, as AstraZeneca CEO Pascal Soriot pledged to make AstraZeneca into a roaring success with $45 billion in annual sales while fending off a takeover attempt by Pfizer, selumetinib loomed large among the experimental drugs he tagged for a blockbuster future. Then the failures started to mount.
AstraZeneca was forced to concede 3 years ago that the drug flopped in KRAS-positive non-small cell lung cancer. Selumetinib whiffed on progression-free survival and failed to score on overall survival as well. And AstraZeneca had to walk away from the first Phase III for selumetinib in 2015 after the drug — in-licensed from Array — failed for an eye cancer called uveal melanoma. Most recently the drug failed a Phase III study for differentiated thyroid cancer in the summer of 2018.
A win here would be sweet for the resurgent oncology team at AstraZeneca, which has fueled AstraZeneca’s long-awaited comeback over the past year with big successes for Lynparza, Tagrisso and to a more limited extent for the PD-L1 Imfinzi.