Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical ($RARE)
Based: Novato, CA
IPO price: $21
Close Sept. 30: $56.60
Change: Up 170%
Raised: $122 million

Scoop: Among 2014's early high fliers, only Ultragenyx was able to sustain its Wall Street performance throughout the year, avoiding the fate of fellow winter boom beneficiaries Dicerna ($DRNA) and uniQure ($QURE). The 2013 Fierce 15 honoree moved 5.8 million shares and above its expected range, and its share price has soared ever since. The biotech is putting its IPO haul toward its four midstage rare disease programs: KRN23, designed to treat the rare disease X-linked hypophosphatemia; triheptanoin, which Ultragenyx is developing for patients with long-chain fatty acid oxidation disorders and those with glucose transporter type 1 deficiency syndrome; and SA-ER, which targets the muscle-destroying hereditary inclusion body myopathy. The company, led by BioMarin ($BMRN) veteran Emil Kakkis, is pursuing a broad strategy of in-licensing rare disease candidates to maximize their value.

For more:
Special Report: 2013 Fierce 15 - Ultragenyx
Rare-disease specialist Ultragenyx fires off a fast $86M IPO
Startup Ultragenyx snags PhII program for growing rare-disease pipeline

Ultragenyx Pharmaceutical
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