Recently, a lot of speculation has surrounded one small cap that has a phase 3 complement to enzyme replacement therapy for fabry disease. Amicus Therapeutics (NASDAQ:FOLD), a Cranbury, New Jersey based biotech company gained 800% from Q1 2014 to Q3 2015. The share price gain gave them a multi-billion dollar MKT Cap and expectations of close to half a billion dollars in annual revenues from global sales for their one late-stage drug, Migalastat. Their phase 3 small molecule monotherapy is on the fringe of EU approval and is aiming for FDA approval after the board required further studies with fabrazyme.
Protalix BioTherapeutics (NYSEMKT:PLX) : This Israeli based penny stock trades on the AMEX and just terminated a deal with Pfizer in which global rights to Elelyso were returned to PLX. The taliglucerase alfa injection for type 1 Gaucher's disease is their only approved therapy. The company also has a series of recombinant therapeutic proteins in its pipeline for major indications such as fabry disease, cystic fibrosis (inhaled), and Gaucher (oral).
Disclosure: I/we have no positions in any stocks mentioned, but may initiate a short position in FOLD, PLX over the next 72 hours.