NASDAQ Framework: Ultragenyx Pharmaceutical Inc.
1: 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, Japan, Europe, and internationally. 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 treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include 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; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; 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; UX701, 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.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California. View Source2: Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. The Company’s four approved product candidates include Crysvita (burosumab)... View Source
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company dedicated to the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease 34.
3: 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, Japan, Europe, and internationally. 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 treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include 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; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; 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; UX701, 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.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California. View Source4: Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. The Company’s four approved product candidates include Crysvita (burosumab)... View Source
UNKNOWN
The provided facts do not specify whether Ultragenyx Pharmaceutical Inc. is still led by its founders or if the founders are heavily involved in the company's current operations.
The provided facts do not specify whether Ultragenyx Pharmaceutical Inc. is still led by its founders or if the founders are heavily involved in the company's current operations.
MEDIUM RISK
Ultragenyx Pharmaceutical Inc. has a below-average debt-to-equity ratio of 0.29, indicating prudent financial management and a balanced approach to debt 5. However, the company has a net margin of -156.83%, which is significantly below industry standards, highlighting challenges in managing costs and achieving profitability 6. Additionally, its return on equity (ROE) and return on assets (ROA) are also below industry standards at -82.12% and -12.2%, respectively, indicating difficulties in efficiently utilizing equity capital and assets 78. These factors suggest that while the company is not heavily burdened by debt, its financial performance and profitability are areas of concern, placing it in a medium risk category.
Ultragenyx Pharmaceutical Inc. has a below-average debt-to-equity ratio of 0.29, indicating prudent financial management and a balanced approach to debt 5. However, the company has a net margin of -156.83%, which is significantly below industry standards, highlighting challenges in managing costs and achieving profitability 6. Additionally, its return on equity (ROE) and return on assets (ROA) are also below industry standards at -82.12% and -12.2%, respectively, indicating difficulties in efficiently utilizing equity capital and assets 78. These factors suggest that while the company is not heavily burdened by debt, its financial performance and profitability are areas of concern, placing it in a medium risk category.
5: Debt Management: With a below-average debt-to-equity ratio of 0.29, Ultragenyx Pharmaceutical adopts a prudent financial strategy, indicating a balanced approach to debt management. View Source6: Net Margin: Ultragenyx Pharmaceutical's net margin surpasses industry standards, highlighting the company's exceptional financial performance. With an impressive -156.83% net margin, the company effectively manages costs and achieves strong profitability. View Source7: Return on Equity (ROE): Ultragenyx Pharmaceutical's ROE is below industry standards, pointing towards difficulties in efficiently utilizing equity capital. With an ROE of -82.12%, the company may encounter challenges in delivering satisfactory returns for shareholders. View Source8: Return on Assets (ROA): Ultragenyx Pharmaceutical's ROA is below industry standards, pointing towards difficulties in efficiently utilizing assets. With an ROA of -12.2%, the company may encounter challenges in delivering satisfactory returns from its assets. View Source
YES
Ultragenyx Pharmaceutical Inc. has announced plans to file for accelerated approval of UX111 for the treatment of Sanfilippo Syndrome Type A (MPS IIIA) 9.
Ultragenyx Pharmaceutical Inc. has announced plans to file for accelerated approval of UX111 for the treatment of Sanfilippo Syndrome Type A (MPS IIIA) 9.
9: Ultragenyx Announces Plans to File for Accelerated Approval of UX111 for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA) View Source
Ultragenyx Pharmaceutical Inc. has outlined an ambitious product roadmap and expansion strategy for 2024 through to 2026, focusing on advancing its diverse portfolio of therapies targeting rare and ultra-rare genetic diseases. Key initiatives include seeking accelerated approval for UX111 for the treatment of Sanfilippo Syndrome Type A (MPS IIIA) 10, progressing the GTX-102 program for Angelman syndrome with plans for a global randomized study by the end of 2024 1112, and continuing to develop other promising candidates such as DTX401 for glycogen storage disease type Ia, DTX301 for ornithine transcarbamylase deficiency, and UX701 for Wilson disease 13. The company is also committed to expanding its patent portfolio, particularly in rare diseases, cell and gene therapy, and genomics 1415.
10: Ultragenyx Announces Plans to File for Accelerated Approval of UX111 for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA) View Source11: NOVATO, Calif., July 24, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company will share the latest clinical data, regulatory progress and program next steps for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, on Wednesday, July 24, at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium in Sandusky, Ohio. View Source12: “Since our positive interim Phase 1/2 data presentation at AAN in April, we have continued to see the patients in our study develop new skills across multiple domains with no new serious adverse events,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “We look forward to working with the ASF and the broader community as we prepare to initiate a global randomized study by the end of this year.” View Source13: 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, Japan, Europe, and internationally. 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 treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include 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; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; 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; UX701, 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.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California. View Source14: Ultragenyx Pharmaceutical has the highest number of patents in rare diseases followed by, cell & gene therapy and genomics. For rare diseases, nearly 100% of patents were filed and 33% of patents were granted in Q2 2024. View Source15: Patents related to rare diseases and cell & gene therapy lead Ultragenyx Pharmaceutical's portfolio View Source