Protalix (PLX) is an Israeli company that is working on incredibly rare diseases that you might only see on House. Essentially, I had to go back to my second year in medical school to remember half of these things, and I admit, had to use a little bit of wikipedia for it.
Taliglucerase alfa (on the market)
Let’s talk about Gaucher’s disease. Those who are in premed, pay attention. It’s a lysosomal storage disorder in which a patient is born with an enzyme not working (glucocerebrosidase). This enzyme’s normal function is to break down glucocerebroside, but since it doesn’t work, you have an accumulation of glucocerebroside and messes with your liver and spleen and sometimes your brain. So Protalix created a synthetic version of the enzyme and the FDA approved it.
There are now officially FOUR enzyme replacements on the market. So this creates an issue with market share. Not to mention, there’s also another drug called Miglustat that actually REDUCES the buildup of glucocerebroside to begin with, so there’s another market share issue. If taliglucerase alfa is the janitor that empties your wastebasket, Miglustat is the guy that comes by and takes it from you before it even makes it to the wastebasket.
Fabry’s disease, also another lysosomal storage disease. This one, works similarly to Gaucher, but a different enzyme (alpha galactosidase) and causes an accumulation of globotriaosylceramide that messes with a lot of organs (heart, kidney, skin, GI, etc). The current treatment is similar, an enzyme replacement therapy called Fabrazyme (by Genzyme) as well as another one called Replagal (approved in EU, but not in US). Genzyme charges something like $300k a year for Fabrazyme, and actually had some issues back in the day due to contamination, which led to shortage, and people petitioning to break the patent due to people needing the drug, but that didn’t go through that well.
Overall, that, also should work well, because it’s very similar to Fabrazyme with one or two amino acids changed.
Ulcerative colitis is a inflammatory bowel disorder, which I’m sure you’ve heard of, and have seen many commercials of a woman riding her bike or something or another. We use a LOT of meds for this, and there are surgery options as well as probiotics, fecal transplantation, etc. etc. etc. The drug we currently use are anti-TNF (like humira), so the drug they’re working on is pretty similar to it, so I don’t see a downside there.
Another possible winner for CF. We currently use something called Pulmozyme, and this is very similar to it, so it SHOULD work.
In conclusion, Protalix has taken a popular drug on the market, changed it just a tad, and is trying to steal market share. A bearish opinion would be that the company has so many in the pipeline, that their resources may be a bit strained. Developing all these drugs can be a huge cash drain, and if in the case that one of the analogs fails, it could take a bigger hit.
TL;DR – the company isn’t discovering breakthrough medicine, just taking something on the market and changing it slightly to take market share. This doesn’t mean it’s guaranteed to work. That, combined with the rarity of the diseases that they are working on, the market is a very small place. Proceed with caution.
Disclosure: I do not have a position in this company.