Regenerative Medicine and Cell Therapies Virtual Event, page-48

No curve balls. We've always known that an additional phase III trial is needed for both heart failure and back pain indications. It's stated in the FDA guidance that 'two adequate and well-controlled clinical investigations remain the standard approach to generating substantial evidence of effectiveness in many disease settings'.

For large scale trials like this, there are no ethical considerations stopping the requirement of two trials. The FDA guidance is non-binding but I doubt SI will try and cut corners. This will increase the chances of approval. The only avenue to submit with one trial is if the data from the first trial is irrefutable - this is also from the guidance 'when a large multicenter trial has demonstrated a clinically meaningful and statistically very persuasive effect on mortality, irreversible morbidity, or prevention of a disease with potentially serious outcome, a second trial would be impracticable or unethical.' I think there is an off-chance that this may be the case for the heart failure trial considering the results from the Phase II but I wouldn't put my money on it.

Patience is a virtue in biotech investing... considering the requirement for additional large scale trials and the timeline for drug applications, I would put a 3-5 year time frame for the commercialisation/approval of both heart failure and back pain indications. Keep in mind that the additional trials conducted will be in different jurisdictions, paving the way for regulatory approval in those areas too (Europe/Latin America etc).