Former FDA vaccines deputy Krause heads down under as Mesoblast tries again on nearly entire pipeline
Philip Krause will take his 11 years of insight from the upper echelons of the FDA — especially during pandemic vaccine nods — and apply it to an Australian biotech that wants a second chance at securing approval in the US, particularly as it looks to bounce back from a nixed Novartis deal.
The former second-in-command for vaccines leadership at the FDA departed under murky circumstances last fall when he and Marion Gruber, director of the regulator’s Office of Vaccines Research & Review, reportedly were miffed by the federal administration’s sidestepping of the FDA on matters of Covid-19 booster shots.
Now, Krause will provide regulatory lessons to Mesoblast as a board member. The move comes at a critical juncture for the Aussie drug developer as it looks to win over the FDA for its cell therapy to treat children with steroid-refractory acute graft versus host disease (SR-aGVHD). The FDA rejected the drug, remestemcel-L, on the grounds that a single-arm, open-label study was not enough in an October 2020 decision.
The agency wanted a randomized trial, but Mesoblast applied after a single-arm study showed statistically significant benefit on the primary goal against a historical control rate in a phase III study. Regulators convened an advisory committee to discuss the application, and the study design was difficult for some members to endorse since parents and pediatricians would be reluctant to place children in a placebo arm.
Since then, the biotech has said it’s been in a “well-established process with FDA’s Center for Biologics Evaluation and Research.” The biotech is teeing up for a resubmission of the therapy.
“Mesoblast has now generated substantial new data that it believes establish the relevance of the proposed potency assay measuring remestemcel-L’s in vitro anti-inflammatory and immunomodulatory activity to the in vivo clinical effect of the product in the Phase 3 trial in children with SR-aGVHD, including survival and biomarkers of in vivo activity,” the company said last month in its latest quarterly report.
As a newly minted board member, Krause did not mince words on the central role he thinks he can play in Mesoblast’s path to market.
“I believe I can make a substantial contribution at this very important time in the company’s transition towards commercialization,” the 30-year FDA veteran said in a statement.
Krause, who was involved in vaccines deliberations during the height of the pandemic, joins Mesoblast as it also looks to rebound from a failed $1.2 billion deal with Novartis in which the biotech tested the aGVHD drug in ventilator-dependent Covid-19 patients with acute respiratory distress syndrome.
The November 2020 deal flunked within weeks as the data safety monitoring board recommended they no longer enroll new patients and wrap the trial early because it was unlikely to meet a 30-day mortality reduction goal. Twelve months later, Novartis ditched its collaborator.
But, as it appears to go at Mesoblast, there’s a second try at everything. Last month, the biotech chalked up the continuing need for new Covid-19 therapies due to new variants and still high numbers of patients on ventilators.
After talks with the FDA, Mesoblast will conduct another trial in this patient population — people with moderate or severe ARDS due to the pandemic disease — and that could pave the way for an emergency use nod, a route that Krause has familiarity with around the Covid-19 jabs.
Also on the docket is a new Phase III trial in the US for rexlemestrocel-L in patients with chronic low back pain. That study may support approval submissions in the US and EU, Mesoblast said last month. Don’t fret, Mesoblast also wants to give the cell therapy another shot in chronic heart failure after a Phase III flop in December 2020.
For Krause, this isn’t the first biotech he’s linked arms with since leaving FDA HQ. He became a strategic advisor at Adjuvance Technologies in January, a little-known Lincoln, NE biotech working on whooping cough and shingles vaccines.