Mesoblast crashed 50pc after FDA response. Picture Getty
Health & Biotech
Regenerative medicine company Mesoblast (ASX:MSB) was hammered down over 54% this morning after receiving a response on its Biologics License Application (BLA) from the US FDA.
This BLA was a resubmission for lead drug remestemcel-L, a potential treatment for children with steroid-refractory acute graft versus host disease (SR-aGVHD).
In its complete response, the FDA said it requires more data to support marketing approval.
The FDA said that to obtain this data, Mesoblast must conduct a targeted, controlled study in adult patients, specifically those in the highest-risk category with the greatest mortality.
While this represents a big setback for Mesoblast, the company said the adult study is in line with its overall commercial strategy in the first place since adults comprise 80% of the SR-aGVHD market.
Meanwhile, the FDA’s inspection of Mesoblast’s manufacturing process resulted in no observed concerns.
The agency also raised no safety issues across more than 1300 patients who have received remestemcel-L to date, and acknowledged improvements to its potency assay.
“We remain steadfast in making remestemcel-L available to both children and adults suffering from this devastating disease, and have received substantial clarity in how to bring this much-needed product to these patients,” said Mesoblast CEO, Silviu Itescu.
Mesoblast now intends to enrol adult patients at highest mortality risk with SR-aGVHD where existing therapy has not improved outcomes and 90-day survival remains as low as 20-30%.
The company has already been working with leading investigators at various US centers of excellence to establish the adult follow-on study protocol, potentially utilising established clinical trials networks.
Mesoblast says it will seek alignment with FDA on the trial design for the adult study at a Type A meeting within 45 days.
AdAlta (ASX:1AD) rose after announcing the first participants in its Phase I extension study of AD-214 have successfully received their first dose.
The study will investigate healthy volunteers and patients with interstitial lung disease or chronic kidney disease, and will use higher doses than in the previous Phase 1 study.
AdAlta will initially be enrolling up to eight healthy volunteers – six participants to receive AD-214, and two participants placebo.
Clinical data from this extension study is crucial as it will better inform the safety profile and target dosing schedule of AD-214 for future Phase 2 studies.
The company says it’s on track to obtain interim results in 2023, and full results in Q1 2024.
Meanwhile, large capped medical devices company, ResMed (ASX:RMD), fell more than 10% after a big drop in margins.
Resmed reported a revenue increase of 21% to $4.2 billion for the full year FY23, which was up 21% on pcp.
However its margins contracted by 80bps to 55.8%, while non-GAAP gross margin contracted 120 bps to 56.5%.
ResMed provided no guidance for FY24.
Health & Biotech
Health & Biotech
Health & Biotech
