Accelerated Approval for ME-401

I'm certainly paying attention to COVID-19 (and still working through my own anxieties about it), but I'm not sure I have a whole lot more to say about it than I've already said. Thinking and reading about kind of puts me in a holding pattern. I'm focusing on the present, trying to take things day by day.

But, at the same time, I need to look ahead. Hope is all about looking ahead, isn't it? Recognizing that things aren't necessarily great right now, and wishing that the future will be better?

With that in mind, it's time to get back to some Follicular Lymphoma news.

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The FDA has designated ME-401 for Fast Track consideration. I've seen a few notices for this online, but not really any details beyond what you'll see here.

The application is for patients with Relapsed or Refractory Follicular Lymphoma (their last treatment stopped working, or didn't work at all) who have received at least 2 prior systemic therapies (chemo, immunotherapy, etc.)

Fast Track designation by the FDA means the treatment is "serious" (likely to result in death if not treated), and the treatments "meets an unmet need" (it is shown to either be more effective or safer than what is available).  Fast Tracking might mean more frequent communication between the FDA and the company making the treatment, so that approval (if it comes) can come sooner.

There's no guarantee, of course, that approval will come at all.

ME-401 is a PI3K, or Phosphoinositide 3-Kinase, inhibitor. As you know from reading, inhibitors work by inhibiting, or stopping, processes that go on in a cell that make it cancerous. Inhibiting Phosphoinositide 3-Kinase means the treatment will stop one of the PI3K enzymes that tell cells to grow. When cells grow without knowing when to stop, they become cancer cells. PI3K inhibitors tell them to stop growing.

ME-401 is, in particular, a PI3K Delta inhibitor. There are four different PI3K enyzmes (alpha, beta, gamma, and delta), and a few different inhibitors that try to target one or more of those different enzymes. They are kind of a common treatment in the Follicular Lymphoma pipeline these days, with some familiar names -- Idelalisib, Duvelisib, and Copanlisib.

The Fast Track designation seems to be based on the results of a phase 1b trial, announced last fall. It's a small trial, with 55 Follicular Lymphoma patients (as of last fall, anyway). But the results were very promising, with 78% of patients in the trial responding. That seems more effective than other PI3K inhibitors.

It will be interesting to see hoe the FDA responds. A phase 1b trial is pretty small, so they may ask for more updated results (it has moved on to a phase II trial now, with 120 participants). I haven't seen a breakdown of how many of the responses were Complete and how many were Partial, which makes me think there were a lot more Partial Responses than Complete Responses. So maybe this ends up being one of those treatments that is taken every day for a year or two to help hold off the need for additional, more aggressive treatment? Certainly patients who would be interested in that.

The good news is, there is another treatment being considered for us. Not the best news, but good news. We'll wait for additional trial results, and more from the FDA, before we start getting especially positive about it.

But we can be hopeful.