Seeking Alpha: Amicus Fairly Rewarded For Substantial Progress

Amicus Therapeutics (NASDAQ:FOLD) is shaping up as a positive example of what happens when patience is matched with good underlying science. There have definitely been some large potholes along the way, including accusations that the company was playing fast and loose with post-hoc analysis, but the underlying idea that Fabry disease is a heterogenous disease and that Galafold (migalastat) can help some, but not all, of those patients as a monotherapy seems to have ultimately won the day.

Although these shares don't look particularly cheap today, there are still value-creating opportunities in front of the company. Actual approval should de-risk the valuation and final pricing of the drug could be a value-driver as well. Amicus's pipeline behind Galafold monotherapy isn't particularly mature, but the opportunities to leverage Galafold into combination therapies and advance additional rare disease treatments into the clinic are potential value drivers down the road.

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Amicus Fairly Rewarded For Substantial Progress 

Seeking Alpha: Amicus Therapeutics Has Driven Value Through The Clinic

In prior articles on Amicus Therapeutics (NASDAQ:FOLD) I spoke of the significant value creation potential of successful clinical trials. If Amicus could show investors that its lead drug migalastat was both safe and effective as a treatment for Fabry disease, the market would reward the company with a substantially higher valuation.

That has happened. Data from the '012 study and additional extension data from the '011 study have established that migalastat offers comparable efficacy to enzyme replacement therapy (or ERT) and meaningful benefits to cardiac and renal function. While the path to FDA approval is still a little murky, investors should have more information relatively soon and I believe the odds now favor approval and commercial success - at least in a subset of patients with amenable mutations. Migalastat's future as a part of combo therapy is still uncertain, but offers further upside, as do clinical programs in Pompe's disease and MPS-1.

Amicus Therapeutics has risen more than 160% over the past year, but still looks undervalued on the basis of its market potential in Fabry disease. With a more convenient administration (it's an oral medication) and a potential safety benefit, there could be still more upside from pricing and/or market share. Value creation through de-risking the Pompe and MPS-1 programs is certainly still possible (positive data will support higher odds of regulatory/commercial success), but those events are further off.

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Amicus Therapeutics Has Driven Value Through The Clinic

Seeking Alpha: Amicus Therapeutics Still A "Show Me" Story

When investors are keen on a sub-sector within biotech, as they have been relatively recently for immuno-oncology, RNAi, and liver disease, companies and their stocks often get the benefit of the doubt, with gaudy sales forecasts and approval odds well in excess of historical norms. On the flip side, and in a case like Amicus Therapeutics (FOLD), once investors have largely written off a company it can be very hard to regain their interest and confidence.

To be very clear, I believe Amicus still has a difficult road ahead of it. The data on lead compound migalastat are not clean and sufficient evidence of efficacy to drive approval (and/or market adoption) is no guarantee. Likewise, the company's 3-in-3 strategy to get three rare disease enzyme replacement therapies (or ERTs) into the clinic over the next three years is ambitious but high-risk. These shares do still appear to be undervalued, but I can frankly understand why many investors may conclude that there are better reward-to-risk opportunities elsewhere in biotech.

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Amicus Therapeutics Still A "Show Me" Story

Seeking Alpha: Amicus Takes A Certain Step Back For An Uncertain Step Forward

I'll happily grant that the best path between two points isn't always a straight line, but with all of the ups and downs (mostly downs) from Amicus (FOLD) over the past year, I can understand if investors no longer want to wait around to see if this recent strategic retrenchment proves a sound move for the long-term.

With lead drug migalastat unlikely to make it to market as a monotherapy, little data on combo therapy in Fabry's, and a one year delay in the Pompe program, not to mention Glaxo (GSK) bowing out of the migalastat program, bears have a lot to chew on. I'm somewhat more bullish on the acquisition of Callidus, though, and I think the market may be overlooking signs that migalastat could still be viable as part of a combo therapy. Amicus shares could still be worth as much as $4 today, but investors should note that this is a very high-risk/high-uncertainty opportunity.

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Amicus Takes A Certain Step Back For An Uncertain Step Forward

Seeking Alpha: Don't Fold On Amicus Therapeutics Just Yet

It's hard enough to pick winners in biotech, but when companies try to explain away bad trial data with "don't look at that, look at *this*" post hoc analysis, all manner of alarms and sirens should go off in investors' heads. And yet, for every rule there is an exception, and I think Amicus Therapeutics (FOLD) may just be that rare exception. While I realize that hope and belief are more suited to theology than biotechnology, I do believe that Amicus's lead drug is effective and safe, and I hope that a 12-month follow-up of its pivotal study will be able to demonstrate that sufficiently for the FDA to grant approval.

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Don't Fold On Amicus Therapeutics Just Yet