Is Ultragenyx Stock a Buy After GTX-102 Flop? Analysts Point to Commercial Pipeline

Live updates

1. 11d ago

   Ultragenyx Stock Plummets After GTX-102 Fails Phase 3 Angelman Trial

   Ultragenyx shares crashed 44% to a record low after its GTX-102 drug failed a late-stage phase 3 trial for Angelman syndrome. The failure of the antisense oligonucleotide (ASO) triggered a market sell-off despite some investor optimism surrounding a separate Genglycos win. In response to the clinical setback, the company is planning significant expense reductions. Some analysts suggest the stock may still be a buy by focusing on the company's remaining commercial pipeline.

   Why it matters

   Angelman syndrome is a rare disease. This trial failure removes a high-potential candidate from the company's development path. The resulting stock crash reflects the high risk associated with late-stage biotech trials.

   What is confirmed

   • Ultragenyx failed a phase 3 trial for a drug treating Angelman syndrome.
   • The failure of the GTX-102 drug triggered a stock sell-off.

   Still unconfirmed

   • Ultragenyx stocks were trading up 3.3 percent following promising clinical trial results.

   What to watch next

   • Details on the specific expense reduction measures
   • Analyst reports on the valuation of the remaining commercial pipeline
   Sources used for this update (6)

   1. Fierce Biotech — Ultragenyx fails phase 3 Angelman test, plots ‘significant expense reductions’
   2. STAT — Ultragenyx drug to treat Angelman syndrome, a rare disease, fails late-stage trial
   3. Barron's — Is Ultragenyx Stock a Buy After GTX-102 Flop? Analysts Point to Commercial Pipeline
   4. FirstWord Pharma — Ultragenyx's ASO fails in Angelman syndrome, triggering sell-off
   5. Investor's Business Daily — 'Fall From Grace' — Biotech Crashes 44% To A Record Low
   6. www.timothysykes.com — Ultragenyx Stock Crashes After GTX-102 Failure Despite Genglycos Win
   confidence 90%