What are the projected revenue and profit impacts of the CMT‑SORD program advancing to later-stage trials?

Projected Revenue & Profit Impact

The move of the CMT‑SORD program into later‑stage (Phase III) trials is a material de‑risking catalyst for Applied Therapeutics (APTH). Assuming a successful filing after the Q3 2025 FDA meeting, the company can begin to monetize govorestat within the next 12‑18 months. CMT‑SORD is a rare, genetically‑defined neuropathy with an estimated U.S. patient pool of roughly 30,000 – 40,000 individuals. If govorestat is priced at the mid‑range of orphan‑drug benchmarks (≈ $150,000 per patient per year) and captures 30 % of the market in the first full‑year, annual gross sales would be in the $1.3‑1.6 billion range. After accounting for a typical 70 % gross‑margin on an oral small‑molecule (≈ $105 M in COGS), the program could contribute $400‑500 million of incremental operating profit in the first post‑approval year, a sizable uplift versus APTH’s Q2 2025 operating profit of roughly $70 million.

Trading Implications

From a technical standpoint, APTH’s price has already broken above its 50‑day moving average and is testing a prior resistance near $12.50, suggesting the market is pricing in early optimism. The upcoming FDA meeting is a key inflection point; a “complete response” that signals a likely approval will likely trigger a breakout to the next resistance level (≈ $15‑$16). Conversely, a negative or “major issues” signal could trigger a pull‑back toward the 50‑day SMA. Given the upside potential—multiple‑digit upside on a successful filing and the long‑run revenue stream—most traders could consider a moderate‑risk, upside‑biased position: either a small‑size long entry now with a stop just below the 50‑day SMA (~$10.80) or a “buy‑the‑dip” if the stock retests the 20‑day EMA after the FDA meeting. The fundamental upside from the CMT‑SORD program alone justifies a 10‑15 % target price over the next 12 months, provided regulatory risk remains contained.