Post by JHam on Jul 21, 2014 5:01:50 GMT
Many of you from the Google board know that I have been following/trading this company for a few years now. There are some very major events possible in the near term. Here is a rundown I gave at the GB:
I am starting to get that FOLD itch again. Here is a bit of a refresher of where we are with this company and why I think now is the time for me to get back in:
- They had very positive results for their Phase III trial (study 011) released in April
- Patients at both 12 months and 24 months continued to show clinical significance while on Migalastat
- Almost 90% of those patients elected to stay on Migalastat voluntarily post trial
- Subjects who switched from placebo to Migalastat after month 6 demonstrated a statistically significant reduction in kidney interstitial capillary GL-3 at month 12 (p=0.013)
So we know that Migalastat is working in patients with Fabry disease. Here is why I am looking to buy again now:
- Second Phase III trial (012 study) top line data is expected to be released in Q3 2014. I expect these results to follow the trend of the other Phase III study (011)
- FOLD has said they anticipate filing an NDA by the end of 2014/early 2015. As soon as they have the complete data set analyzed and ready to submit to the FDA. If you'll remember, FOLD initially wanted to submit an NDA for "conditional approval" much earlier, but the FDA told them they would not get approved without the full data. So FOLD scrapped the conditional approval idea an decided get the full data set and then file for full NDA approval instead. Now they have the 24/12 month data collected on the 011 study and will soon have the full 18 month data on the 012 study, so that filing date is approaching
- FOLD is going it alone. Crowley is even quoted as saying the following on the last call: "When you think about that, we can be in a position in the second half of this year to have hopefully a successful Phase 3 Monotherapy program and a program that is unpartnered. It is an Amicus program with very modest royalties in most geographies. That is our program that we could drive forward. We absolutely could commercialize the program. There is a significant amount of potential partner interests that we are considering. Although, we said to those potential partners and to the broader public here that our bar for partnering would be very high."
- Fabry disease represents a $1B, largely untapped market
- Current market cap of FOLD is $186.59M
- $71.6M cash on hand at the end of Q1, but based on the quarterly burn rate it is probably closer to $60-55M now. Still in very good shape with cash
- Major institutional investment. Glaxo holds 18% of the shares. Other major healthcare oriented institutions/funds including Perceptive. Perceptive, who has a billion dollar fund and was rated as the 2013 4th best performing mid-sized hedge fund in the world, bought a huge amount of FOLD at the very end of May, putting it in their top 20 of largest holdings.
In summary, I think investors are really anxious to see this last top line data set. If it follows the positive trend then I believe that will seal the deal for NDA approval.
I am starting to get that FOLD itch again. Here is a bit of a refresher of where we are with this company and why I think now is the time for me to get back in:
- They had very positive results for their Phase III trial (study 011) released in April
- Patients at both 12 months and 24 months continued to show clinical significance while on Migalastat
- Almost 90% of those patients elected to stay on Migalastat voluntarily post trial
- Subjects who switched from placebo to Migalastat after month 6 demonstrated a statistically significant reduction in kidney interstitial capillary GL-3 at month 12 (p=0.013)
So we know that Migalastat is working in patients with Fabry disease. Here is why I am looking to buy again now:
- Second Phase III trial (012 study) top line data is expected to be released in Q3 2014. I expect these results to follow the trend of the other Phase III study (011)
- FOLD has said they anticipate filing an NDA by the end of 2014/early 2015. As soon as they have the complete data set analyzed and ready to submit to the FDA. If you'll remember, FOLD initially wanted to submit an NDA for "conditional approval" much earlier, but the FDA told them they would not get approved without the full data. So FOLD scrapped the conditional approval idea an decided get the full data set and then file for full NDA approval instead. Now they have the 24/12 month data collected on the 011 study and will soon have the full 18 month data on the 012 study, so that filing date is approaching
- FOLD is going it alone. Crowley is even quoted as saying the following on the last call: "When you think about that, we can be in a position in the second half of this year to have hopefully a successful Phase 3 Monotherapy program and a program that is unpartnered. It is an Amicus program with very modest royalties in most geographies. That is our program that we could drive forward. We absolutely could commercialize the program. There is a significant amount of potential partner interests that we are considering. Although, we said to those potential partners and to the broader public here that our bar for partnering would be very high."
- Fabry disease represents a $1B, largely untapped market
- Current market cap of FOLD is $186.59M
- $71.6M cash on hand at the end of Q1, but based on the quarterly burn rate it is probably closer to $60-55M now. Still in very good shape with cash
- Major institutional investment. Glaxo holds 18% of the shares. Other major healthcare oriented institutions/funds including Perceptive. Perceptive, who has a billion dollar fund and was rated as the 2013 4th best performing mid-sized hedge fund in the world, bought a huge amount of FOLD at the very end of May, putting it in their top 20 of largest holdings.
In summary, I think investors are really anxious to see this last top line data set. If it follows the positive trend then I believe that will seal the deal for NDA approval.
It is higher than I was hoping for, but I still think that it has a lot of room to grow with positive final phase III data and any good news on the NDA filing. It's a small position, but I may continue to accumulate. Earnings should be out any day and if they beat estimates it could cause a nice little pop.
