I think that there is a very real chance that GvHD is the most immediate catalyst to get the share price jump started, and it would not shock me if we found out after the fact that management had shifted focus and funds to accelerating the GvHD enrollment, even prioritized over enrolling mono. I'm assuming that they have enough mono patients enrolled at this point to support the safety data requirements for the combo BLA, based on their most recent expected timelines for submission. At the very least, they should be nearing the needed number.
Consider:
- GvHD is a very serious, very unmet medical need. Just the type of thing that the FDA has shown an affinity for approving quickly.
- The effectiveness shown in the animal data was shocking.
- The FDA just agreed to a major protocol change after 10 patients, and is planning another IDMC after 10 more patients are treated for 30 days (reduced from 60...).
I've thought about this quite a bit, and my only logical read of the actions taken on the GvHD trial are that the results are bearing out significant efficacy, and the FDA is amenable to speeding things along. If - and only if - the data on the next 10 open label patients is compelling, I expect that the FDA will move to get this indication on the market nearly immediately via conditional approval. I see no other rationale for the wholesale change in protocol and the stated intent to review the data after 10 more patients on a reduced timeline. I'd love to hear what others, particularly those with experience in clinical trials and drug approvals, think about this possibility.
Assuming that my hypothesis is anywhere near valid, I expect management to throw as many resources as possible at getting those next 10 enrolled and treated. It would be a far more impactful use of funds in the near term than focusing on mono enrollment. Call me crazy and/or optimistic (I've been called worse), but it wouldn't shock me if we're halfway there or better at this point. The PR was three weeks ago, they have five sites open, the data appears promising given the protocol change, and management has the incentive to funnel resources there in the short term.
I've been saying it for a long time privately, but I think GvHD ends up being the surprise game changer here. The real money is in mono... but GvHD is the first domino to fall.
