The Thesis Is Working (Better Than We Expected)
Revenue Doubled. The Portfolio Grew. And Management Just Guided to $200M.
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Five months ago, we profiled a microcap rare disease company hitting commercial inflection at 18x forward earnings.
The pitch was simple: a growing orphan drug portfolio, margins headed toward 75%, five near-term FDA catalysts, and a stock priced like nothing was about to happen.
Here’s what’s happened since:
The company’s biggest pipeline candidate received FDA approval and launched commercially within two weeks. The label came in cleaner and broader than expected - expanding the addressable market well beyond the original forecast.
Full-year 2025 revenue more than doubled versus 2024.
Q4 revenue grew 83% year-over-year.
Adjusted gross margins recovered to 73%, confirming the prior quarter’s compression was temporary - exactly as we flagged.
Adjusted EBITDA margin hit 29%, up from 18% a year ago.
The company turned GAAP profitable.
The portfolio expanded from 8 to 10 commercial products through a disciplined acquisition funded entirely with cash - no dilution, no debt.
2026 guidance calls for revenue exceeding $110 million with EBITDA margins above 30%.
Management just set new long-term targets: $200 million revenue run rate by end of 2027, 50% EBITDA margins by 2028, and $500 million in revenue by 2030.
The stock is up 35% from our write-up.
But the fundamental story has arguably improved by more than the stock has moved.
Our original base case implied roughly 42% upside. Our bull case outlined nearly 200% upside over 2-3 years. With the catalysts now materializing, the updated setup may offer even more.
Let’s walk through what’s happened, what’s changed, and where the risk/reward sits today…