Martin Shkreli, known as Pharma Bro, said Ultragenyx Pharmaceutical (NASDAQ:RARE) is undervalued and a potential takeover target for rare-disease peers like BioMarin Pharmaceuticals (NASDAQ:BMRN) after a trial setback.
He said "$RARE is very cheap" and urged BioMarin and other rare-disease companies to explore buying the company, adding that "everyone and their brother with 3 months of med school/shkreli training knew this trial would fail."
Ultragenyx’s Cost-Cutting Measures
- Ultragenyx is planning significant expense reductions after its Phase 3 Aspire study for the Angelman syndrome therapy, apazunersen, failed to meet primary endpoints.
- The trial’s failure has prompted the company to review the program’s future and implement cost-cutting measures.
Analyst Downgrades for Ultragenyx
- In a Thursday note, analysts downgraded Ultragenyx’s stock, with Baird’s Joel Beatty cutting the price target from $40 to $16.53.
- Evercore ISI Group’s Liisa Bayko also downgraded the stock and reduced the target price to $16.53, reflecting the impact of the trial results.
- These downgrades were part of a broader analyst review, which also included changes for Moderna, Inc.

Technical Analysis
RARE’s RSI(14) is at 18.99, indicating an oversold condition. The stock trades 47.39% below its 50-day SMA of $27.99 and 43.54% below its 200-day SMA of $26.08. The 50-day SMA remains above the 200-day SMA.
RARE Stock Price Activity: Ultragenyx Pharmaceutical shares were down 43.95% at $14.87 on Thursday, according to Benzinga Pro data.
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