@vinn It is so exciting. Mesoblast has a global exclusivity arrangement with Lonza with respect to all allogenic production of Mesenchymal cells. I understand Mesoblast may have already developed a proprietary media for harvesting the cells which may exceed the performance parameters of even Lonza’s latest cell processing methodology which you have highlighted . People will soon come to realise, that Rexlemostrocel’s precursor cells (MPCs) used in our CHF trial should be far superior to MSC’s, ( Ryoncil) which were developed by Osiris...i think Silviu likes to refer to them as first and second generation therapies. Once everyone accepts the activity of our cells most of the questions will revert to potential manufacturing bottlenecks ...and why we cant make enough !!! I suspect the FDA will be well and truely hung drawn and quartered if they don’t find creative solutions to a speedy review of Lonza’s new 3D manufacturing protocols to assist with the availability of the new treatment options. Incidentally, the patented fucosylation homing technologies licensed by Sackstein to Mesoblast ( a protein attached to the cell receptors which allows superior cloaking increasing half life and potency) combined with the latest 3D bioreactor techniques have the potential to bring the COGS down to the 20% level...but it will require investing in several new manufacturing suites over the next several months to build production up to nearer the 100,000 treatment level. Provided we are successful with our application on the first review cycle (which is highly likely, as 93% of BLA applications to CDER/FDA succeeded on their first review attempt in 2019 ) I believe we will be able to attract a bidding war with the global pharmaceutical companies for our IP. Furthermore all 100% of applications in 2019 were approved by their given PDUFA date. I should caution, however, that there is always a chance that the pandemic may cause minor paperwork delays, which makes relying totally on historical performance statistics more questionable.
I still believe we will comfortably show “overwhelming efficacy” at the 45% Interim analysis..unless the DSMB are intent on waiting until 180 patients (60%) to build up a more robust data set which i think (assuming good results) would be unethical Furthermore, I do not agree with Bell Potter’s view that the next interim analysis will arrive in late October...i think enrolment was particularly robust in July and we should expect news early October at the latest. This view also seems to resonate with comments made by the Prof in the recent article in the Australian that new results our “weeks away” and his webcast assertion that our trial should be able to prove efficacy with “a fraction “ of the total trial size..
Covid ARDS appears to suit our mechanism of action perfectly....even more perfectly than influenza or other causes of ARDS. It is effectively a disease of the blood vessels which creates damage to the endothelial cells....as this latest piece of breakthrough research reveals (see second page of article)
‘Kishimoto drew a distinction, however: COVID-19 patients can develop severe respiratory failure, suggesting a distinct immune reaction compared to patients with bacterial sepsis . SARS-CoV-2 directly infects and activates endothelialcells rather than macrophages, as occurs in sepsis”
That is not say we will not have a therapeutic effect with sepsis just that they are different ...We have already proven, as referenced by @Tunnah in a recent post, that we have potent therapeutic benefit in treating influenza A (H7N9) with ARDS , where a peer reviewed study showed a mortality rate of only 17.6% with MSCs versus 54.5% in the control.
Within 2 to 6 weeks I believe that Mesoblast should become a household name for all the right reasons. What a risk reward opportunity!
Check out how far ahead we are to our competition in this review of all trials
(20min delay)