Alrighty
I just finished watching the webcast, and here are the points that I take away.
GVHDGvHD is currently in the process of regulatory approval. Company believe the FDA wanted an extra potency Assay during manufacturing. Mesoblast have developed one. Silviu said they are ready now to submit the new data to the review team as soon as they can get a meeting. If the OTAT Review Team agree on this extra potency assay then Mesoblast will re-submit for approval with AGVHD, with a 6 month approval decision timeline ( After the re-submission ).Discussions are expected over the next few weeks
..... so maybe 10 weeks
NovartisRelationship with Novartis is expected to close in the short term. Currently Mesoblast are waiting for Novartis to complete review of the secondary data. Notably Mesoblast revealed that it now also has 90 day data - but did not indicate anything contained in it, just that they now had it. Maybe this was what Novartis was waiting for. The 90 day end point was only for mortality and it is the last near term end point before jumping to 6 and 12 month end points of hospital stay, number of re-admissions, 12 month total of ICU stays. and pulmonary symptoms at 6 and 12 months. All biomarker data stopped at 30 days. Lessons learned in Covid ARDS will help inform the all cause ARDS trial design
Covid ARDS under and over 65 year oldThe first piece of info I was not aware of, the dosing used in the COVID trial was entirely based off the GvHD dosing - which was only tested in under 65's. This is the reason why the under and over 65 groups were created, as they had no existing data showing either efficacy , or what an adequate dose would have been for an over 65. This is great news really, as I am sure people here are thinking they just cherry picked the data, when in fact there was a legitimate reason for the pre-specified grouping of results. As it turns out, another massive goal kicked by MSB management.
They are meeting with the FDA to discuss what the next steps should be, but are planning a confirmatory phase 3 in under 65's with dexamethazone, and a second group which will be over 65's on either longer dose or on a higher dose, or maybe three + arms with both and SOC.
No mention on what the 90 day data indicates, but Silviu did say they only just received it, and the results in the under 65 ( Which did meet the primary end point if considered without the over 65's ), were as they expected
Heart FailureCurrently in discussions with potential un-named strategic partner. Mentioned they are currently exploring biomarker data to support the MOA, to create an intermin dataset that will predict mortality reduction based on biomarkers
...... The stars are aligning
Back PainPlanning a global confirmatory phase 3 trial with Grunenthal, to satisfy both FDA and European regulator requirements. On top of this will discuss a second potential pathway for treatment of opioid receiving patients after trial demonstrated 40% opioid use reduction over 24 months.
BIOGEN APPROVAL COMMENTS Si's takeaway from the entire Biogen Saga was that the FDA demonstrated flexibility when dealing with a novel therapy - treating a disease with no alternative treatment. He then mentioned that he thought in particular the Heart Failure treatment fell right into this group - as no other therapy even came close to achieving what MSB achieved with a single dose in the class 2 patients. They sound like they are gearing up for an approval discussion with FDA at the next meeting.
All in all, lots of old information covered, but with a bit more explanation around the edges. Well done Si.
I'm holding this until it takes me to the moon !
(20min delay)