Marty Schildhouse, communications consultant to BioHeart, Inc (BHRT-$2.45), a biotech developing autologous (patient - derived) cell therapies for heart failure, responds to questions the 10Q Detective asked about the pivotal MARVEL trial:
- Please comment on why MyoCell [based on pending MARVEL data in 2009] might be viewed positively by FDA—given stem cell trials to-date—failed to show clinically significant improvement in ventricular contractility?
As you have noted, previous stem cell clinical trials were designed to show mechanical improvement as their primary endpoints. Our confidence in the MARVEL Trial's potential to be viewed positively by the FDA stems from the fact that we have designed this trial differently than our previous trials, or those of other stem cell trials.
The trial design focuses on measurements the FDA has expressed interest in, as well as used as the basis for approvals of complimentary therapies. As such, we are confident that we have a trial that meets the FDA's expectation in terms of primary and secondary endpoints. The primary endpoints in the MARVEL Trial are based on qualitative measurements, aligning to the FDA's belief that these measurements are more reflective of, and appropriate for, this patient population. This is consistent with FDA's review/approval of bi-ventricular assist devices targeting this same population.
Mechanical endpoints, such as LVEF, will be measured as secondary endpoints in this trial. As the trial is powered to meet the more demanding statistical needs of a qualitative measurement, the MARVEL Trial is also appropriately powered for the secondary endpoints. Additionally, along with qualitative and mechanical endpoints, MARVEL will have secondary endpoints based on gathering pharmacoeconomic and health economic data. MARVEL is the largest, and most clinically rigorous trial of its kind.
- Does BioHeart plan on a private placement to raise the $17 million needed to complete the MARVEL trial—or are third-party agreements a possibility?
We are currently pursuing a secondary financing transaction and hope to report on our progress in our Q-2 communication. The company continues to be open to potential other sources of financing and will seek those sources based on the best terms and long-term benefit to the organization.
The 10Q gives a thumbs up to the transparency proffered by Schildhouse and BioHeart.
Editor David J. Phillips does not hold a financial interest in any stocks mentioned in this article. The 10Q Detective has a Full Disclosure Policy.