New York, May 22, 2026, 08:03 EDT
- RARE finished Thursday at $23.71, up 1.45%. Shares last traded at $24.06 ahead of the Nasdaq open in extended hours.
- Cantor Fitzgerald lifted its price target on the stock to $96, up from $84, and kept its Overweight rating.
- U.S. stock markets will run regular hours Friday. Markets are closed Monday for Memorial Day.
Ultragenyx Pharmaceutical Inc. was up in premarket trade Friday after Cantor Fitzgerald raised its price target on the company and flagged its late-stage Angelman syndrome program. Shares finished Thursday at $23.71, gaining 1.45%, and were at $24.06 at 7:50 a.m. Eastern in the extended session.
Cantor lifted its price target on Ultragenyx to $96 from $84 and stayed at Overweight, the bank’s way of saying it sees the stock topping the peer group. The firm also raised its success odds for GTX-102, Ultragenyx’s Angelman syndrome therapy candidate, bumping the probability to 60% from 25%.
Rare moves into a key stretch over the next few months. Ultragenyx said Phase 3 data from the Aspire trial of GTX-102 are still slated for the second half of 2026, and its two BLAs — applications for U.S. approval of biologic drugs — are still in front of the FDA.
Ultragenyx CEO Emil D. Kakkis called 2026 “an important year,” saying the company is getting ready for “two potential gene therapy approvals and launches.” Kakkis also said updated GTX-102 data “support the potential of the program,” with Phase 3 results coming up. Last10K
Cantor’s Kristen Kluska boosted her price target, pointing to how the GTX-102 Phase 3 trial could hit its main statistical endpoint, according to a note picked up by The Fly on TipRanks. The note said this study could let both non-hierarchical endpoints count — meaning either of the two trial measures could lead to success, with no need for one outcome to trigger the next.
Ultragenyx has company in Angelman syndrome. Ionis Pharmaceuticals is running a Phase 3 REVEAL trial testing ION582. The FDA awarded ION582 breakthrough therapy status in 2025, which is supposed to quicken review for drugs with early signs of benefit in severe disease. At the time, Ionis said there were no approved disease-modifying therapies for Angelman—nothing approved that goes beyond symptoms and actually changes the disease process.
The market had a positive tone, but gains stayed small. The Nasdaq Composite added 0.09% to close at 26,293.10 Thursday. The S&P 500 was up 0.2%. U.S. stock markets are open as usual Friday before a holiday break for Memorial Day on Monday.
Ultragenyx’s first-quarter revenue dropped to $136 million from $139 million a year ago. The company posted a net loss of $185 million. As of March 31, Ultragenyx reported $534 million in cash, equivalents and marketable securities. The company said it expects to stay unprofitable for now as it keeps working on development and regulatory approvals for its drug candidates.
The bullish argument is fragile. Poor GTX-102 data, an FDA review that drags, issues in manufacturing, or if cash burn runs high, investors are left with a biotech still losing money and valued mostly on what it might get approved down the road.
Markets were not open yet. If stocks start higher, it would suggest the Cantor call is bringing buyers off the sidelines. If shares slip, investors may still be waiting to see clinical data before moving, rather than just respond to a new model price.
