Quite a lot of action this month, which may pale in comparison to what I think is going to happen in May.
Much to the horror of one of the resident, growing doubt non-holding psychopaths, Mesoblast have again confirmed in the current quartlerly " Remestemcel-L is being developed for inflammatory diseases in children and adults including .... acute respiratory distress syndrome."
The FDA internal meeting provides management and review teams with an opportunity to discuss the review status, key findings, and any issues, update timelines, and make a determination about the need for formal risk evaluation and mitigation strategies (REMS) and any additional post marketing requirement (PMR) and post marketing commitment (PMC) studies.
Generally, within 2 weeks of the mid-cycle meeting, the RPM and other appropriate members of the review team will call the Sponsor to provide an update on the review status. This is known as the Mid-Cycle Communication. One of the most significant events besides the full review of the quality sections (i.e., chemistry, manufacturing, and controls [CMC]) of the marketing application is the Pre-approval Inspection (PAI). If a PAI is conducted, FDA will send a team of experts to the manufacturing facility to assure that the facility is capable of manufacturing the drug and that data submitted with the marketing application are accurate and complete.
Mid May 23 The mid cycle review phone call should have been completed around now, and MSB should be aware of the direction that the BLA is proceeding in based on the feedback from the FDA. In particular the commitment for a post marketing study including adults against SOC - which the company previously agreed to will likely come up again. How much ( if any ) of this meeting will be made public is anyone's guess. The company may not wish to disclose anything to avoid lawsuits until the final decision is made, even if there are no major issue identified, and you can understand why in this case. On the other hand shareholders would welcome confirmation that there are no major issues.
The Singapore plant inspection ( if conducted 60 days after formal notification ) , will also be completed mid May with any issues identified being passed to the sponsor for rectification. We may get news about this also.
June/July 23 If the product is not approved by this stage, then likley there may be a Late-Cycle Meeting between the FDA and Sponsor to discuss any major deficiencies, the need for a REMS or other risk management actions, major labeling issues, additional post marketing studies, and other updates/issues as appropriate.
If approval is anticipated, labeling discussions are held between FDA and the Sponsor in order to reach agreement on the contents and wording of the prescribing information (i.e., product label, package insert). This is a critical step because it determines the labeled use for the product and impacts how the product may be marketed.
August 2 - Planned PDUFA payment
August - in the version where Ryoncil gets approved. 40 million USD in loan drawdown is unlocked, probably leaving a bank balance of around $100 million USD + CLBP trial is likely initiated if not already underway. Partnership deals ( for CHF and CLBP ) that were on hold awaiting the outcome of the FDA decision on Ryoncil will continue towards completion. Ryoncil sales start.
August - in the version where Ryoncil doesn't get approved. This is a little tricker to predict, because the response will be proportionate to exactly what the issue is, and if any of the issues are a continuation of previous problems ( not resolveable without a new trial ), or new plant related problems ( process - fixable ).
Ryoncil - non trial related issue. I think in this case the items will be remediated, which will blow out the approval timelines another year~ish.
Trial related issues . I think the company now know enough thanks to the CRL, and potency assay work, and 4 year survival data to just go ahead and run the ( originally planned as a phase 4 ) adult trial against SOC and blow everyone away with the results - gaining access to the entire SR - GvHD market in the process and not just children. This is no quick task, and in all likelyhood would result in CLBP being approved prior to GvHD approval........
Best case we get approved with a phase 4 expansion into adults, worst case CLBP looks like it may be the first approved product. Seeing as this is the main even for share holders, that is not all doom and gloom either.
On a side note, After the expected mass exodus of shorts in the CR, they have commenced two days of price manipulative war, pilling back in, selling around $4 million in stock over just 2 days to keep the share price down !
That is - about 50% of every trade yesterday, and the day before was a short selling a borrowed share. 0.76% Short trades 1.49% Total traded volume over 2 days.
Why they would go short at 88 cents...? Missed the CR exit maybe and trying to manufacture a lower exit while they still can?
MSB Price at posting:
86.3¢ Sentiment: Buy Disclosure: Held
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