Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
| Business Activity | PASS |
| Debt Ratio | N/A |
| Cash Ratio | N/A |
| Receivables Ratio | N/A |
| Revenue Ratio (0.00%) | PASS |
| Overall | PASS |
$3.1B
-28.02
0.386
19
$670.0M
$1.2B
1.47
8.25
0.1%
1,371
| Shares | Cost | Profit to Target | R:R |
|---|---|---|---|
| To Consensus ($52.05, 19 analysts) | |||
| 10 | $233.90 | +$286.60 (123%) | 8.2R |
| 50 | $1,169.50 | +$1,433.00 (123%) | 8.2R |
| 100 | $2,339.00 | +$2,866.00 (123%) | 8.2R |
- Elevated short interest (8.25 days to cover)
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| Ticker | Correlation |
|---|---|
| HQH | 0.358 |
| DNLI | 0.358 |
| ARKG | 0.353 |
| CRNX | 0.337 |
| IDYA | 0.320 |
| Ticker | Correlation |
|---|---|
| LEJUY | -0.193 |
| AMAN.AE | -0.172 |
| 5069.KL | -0.160 |
| 1183.SR | -0.158 |
| BAES.L | -0.153 |
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