Regulatory Pathways - where we stand with the FDA

Bio-techs notoriously require patience from investors. That said it is understandable many MSB investors have lost patience with management, somewhat ignorant to the regulatory difficulties and hurdles they have faced with apparently long unexplained delays to applications following Phase III results and intiial regulatory applications made as long ago as mid-2020.

Whilst it is understandable many investors have closed out losses or lost patience, it would appear they are doing so when MSB is on the verge of regulatory success. Where we stand:

C-19 ARDS:

MSB was waiting for 60 and 90-day results and analysis, but as of 19 July following the 90-day results presentations, is "shortly" due to meet with the FDA. The adoption of 60-day mortality benchmarks in C-19, the changing standard of care, pre-determined population samples, etc have been discussed to death on Hot Copper. Suffice to say the company and us as investors are finally approaching an FDA meeting far more informed on the subject.

"Recently published guidance to industry by the U.S. Food and Drug Administration (FDA) has recommended demonstration of mortality benefit for at least 60 days in critically ill patients. Mesoblast will be meeting shortly with the FDA to discuss the durable mortality reduction seen in patients under 65 years old who received remestemcel-L in this randomized controlled trial, and the regulatory pathway for remestemcel-L in this patient population." (19 July)

CHF:

Possiblly the most disappointing progression given it has been 7 months since the Phase III trial result (perhaps pushed given partnership aspirations/negotiations). MSB is still ‘intending to meet with the FDA to discuss potential next steps in the regulatory pathway'. Whilst the company is no doubt in possession of far more supportive information for its meeting with the FDA, we as investors remain somewhat uninformed to the strategy and still guessing at a timeline.

"Based on the observed reduction in mortality and morbidity in this [DREAM-HF] trial, Mesoblast intends to meet with the FDA to discuss potential nest steps on the regulatory pathway"(3 June)

CLBP:

Some 4 months after the Phase III results, a Type C meeting with the FDA formally requested as of June. Perhaps the quiet dark horse amongst the group.

“Mesoblast has filed a request and expects to hold a Type C meeting with the US Food & Drug Administration (FDA) during the current quarter to discuss the pathway to US regulatory approval for rexlemestrocel-L following the recently completed 404 patient Phase 3 trial in patients with chronic inflammatory back pain due to degenerative disc disease.” (1 July)

GvHD:

Since an essentially failed Type A meeting with the FDA on 17 November, finally as of early June, the company has begun direct communications with the CBER on “outstanding chemistry, manufacturing and controls (CMC) items, including potency assay validation.”

We all know the ODAC history, etc, but clearly progress has been made with the FDA:

“Mesoblast continues to be in discussion with the United States Food & Drug Administration (FDA) through a well-established regulatory process that may include a resubmission with a six-month review with the aim of achieving approval of remestemcel-L in the treatment of steroid-refractory acute graft versus host disease (SR-aGVHD) in children.”

”As part of this process, Mesoblast recently met with the FDA’s Center for Biologics Evaluation and Research (CBER). Following CBER’s recommendation after this meeting, Mesoblast as a next step will discuss with CBER’s review team at the Office of Tissue and Advanced Therapies (OTAT) our approach to address certain outstanding chemistry, manufacturing and controls (CMC) items, including potency assay validation.” (3 June)

Most investors will of course be well aware of the above, but hopefully this will serve as a reminder of how close we are to some critical regulatory steps after some 7 months of waiting in some part. It is perhaps something we can all simply append to for simple, easy reference for others and leave debate to other forums.