These projects include the pediatric Rett trial and Alzheimer’s trial still expected to read out in H2, still planning to meet with the FDA to get going on a Phase 3 Parkinson’s trial, still planning the Parkinson’s imaging trial, and still having $153 million in cash, enough for 4 years.
Truth be told, not much has changed, except certain long awaited readouts are that much closer now. And this lack of anything significant to get investors excited about — along with the recent market swoon — has led to the drop off in the shares.
It's hard to believe that AVXL was over the mid-$20’s on two runs in the past year — and now we’re back near $8. It's good that we sold half on the way up. Some may want to reload somewhat or nibble here ... though it takes some nerve the way this market has been acting. But it’s time to look 6-9 months out past the current malaise to when some important results finally get reported.
Let me point out one more time that as good as phase 2 results looked, many drugs fail anyway in a more rigorous Phase 3. And while I am optimistic based on everything we know … you just never do know. So you don’t want to bet the ranch here, but we do have several shots on goal.
Based on the conference call, the company is showing increasing priority (now #3 behind Alzheimer's disease and Rett) and excitement for moving ahead on Fragile X due to strong/convincing data from animal models for Fragile X including biomarkers and ADAM score (it will be presenting on this at a conference in July) and also because Fragile X is a huge unmet need that is 7 times the size of the Rett Syndrome population. The company is working on designing a potentially pivotal study that it hopes to propose to the FDA in H2.
Also — and this is new — there is an Investor Day planned for June 21st at which the company will take a deep dive into its pipeline, which is growing. Nibble for the longer term … if you dare. I can’t guarantee the shares won’t go lower for no good reason, but this remains an enticing price with the goal line growing ever closer.