Conclusion
PLX's Fabry program has finally arrived at a finish line of sorts, with the completion of BALANCE, PLX's only randomized, controlled (with an active comparator control vs. open-label without a control) phase 3 Fabry trial.
It all goes well from now on, the company plans to resubmit their application to the FDA in the second half of 2022.
Needless to say, the company and the investors share a great hope for PRX-102's market potential (up to >$1B in potential milestone payments). See below.
According to this market report, the global Fabry drug market in 2021 was ~$2.1B.
If PRX-102 is approved, it would be the fourth Fabry treatment in the EU after Sanofi and Shire's ERT and Amicus' Galaford (an oral treatment) and the third in the US (Shire's ERT is not approved in the US).
In other words, PRX-102 would not be the first-in-class Fabry drug, and in terms of renal efficacy, it is certainly not the best-in-class Fabry drug (only a non-inferior ERT [to Fabrazyme] for adults, where the need is not as urgent as for young children diagnosed of Fabry Disease).
Furthermore, as stated recently, I believe that results from BRIGHT will not support the approval of a 4-week regime of PRX-102, which would have been an advantage over the 2-week regime of currently available ERTs.
Having said all this, I do understand that the short-term price action of small/micro biotech stocks are driven by many factors, e.g. speculative FOMO, penny stock/momentum trading, wider market volatility, besides the trial data or profitability of the company.
In my opinion, for long-term investors, the most significant risk is that PRX-102's prospect may not be sufficiently strong to ensure a real (sustained) turn-around in this stock, as:
Regulatory approval, in the EU and/or in the US, is not a sure thing;
If approved, it is a late comer without superiority in renal efficacy to compete in the established Fabry drug market;
Studies/approval for pediatric use is very far away, if ever.
