Company Research Report: Ultragenyx Pharmaceutical Inc.

Company Overview

• Name: Ultragenyx Pharmaceutical Inc.

• Mission of the Company: To advance innovative medicines for rare and ultrarare diseases that have never been treated before, delivering transformative therapies across multiple indications. Ultragenyx focuses on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a meaningful way for rare disease communities.

• Founded by: Emil D. Kakkis, M.D., Ph.D.

• Key People:

• Emil D. Kakkis, M.D., Ph.D. - Founder, President, and Chief Executive Officer

• Howard Horn - Chief Financial Officer and Executive Vice President, Corporate Strategy

• Eric Crombez, M.D. - Chief Medical Officer and Executive Vice President

• Erik Harris - Chief Commercial Officer and Executive Vice President

• Dennis Huang - Chief Technical Operations Officer and Executive Vice President, Gene Therapy Research and Development

• Thomas Kassberg - Chief Business Officer and Executive Vice President

• Ernie Meyer - Chief Human Resources Officer and Executive Vice President

• Karah Parschauer - Chief Legal Officer and Corporate Affairs, EVP

• John Pinion - Chief Quality Operations Officer and Executive Vice President, Translational Sciences

• Headquarters: No information is available

• Number of Employees: No information is available

• Revenue: No information is available

• What is the Company Known For: Ultragenyx is known for delivering transformative therapies for rare and ultrarare diseases and having one of the most robust and diverse clinical pipelines in the rare disease sector.

Products

Approved Products

• CRYSVITA® (burosumab-twza)

• Indication: X-linked hypophosphatemia (XLH), Tumor-induced osteomalacia (TIO)

• Description: Anti-FGF23 monoclonal antibody

• MEPSEVII® (vestronidase alfa-vjbk)

• Indication: Mucopolysaccharidosis type VII (MPS VII)

• Description: Enzyme replacement therapy

• EVKEEZA® (evinacumab-dgnb)

• Indication: Homozygous familial hypercholesterolemia (HoFH)

• Description: Anti-ANGPTL3 monoclonal antibody

• DOJOLVI® (triheptanoin)

• Indication: Long-chain fatty acid oxidation disorders (LC-FAOD)

• Description: Substrate replacement

Products in Pipeline

• UX143 (setrusumab)

• Indication: Osteogenesis imperfecta (OI)

• Description: Anti-sclerostin monoclonal antibody

• Gene Therapy Products:

• UX111 (rebisufligene etisparvovec) for MPS IIIA

• DTX401 (pariglasgene brecaparvovec) for Glycogen storage disease type Ia (GSDIa)

• DTX301 (avalotcagene ontaparvovec) for OTC deficiency

• UX701 (rivunatpagene miziparvovec) for Wilson disease

• UX055 for CDKL5 deficiency disorder (CDD)

• UX810 for Duchenne muscular dystrophy (DMD)

• ASO/mRNA Products:

• GTX-102 for Angelman syndrome

Recent Developments

• October 29, 2024: Ultragenyx announced plans to host a conference call for third-quarter 2024 financial results and corporate update.

• October 18, 2024: Ultragenyx reported an inducement grant under Nasdaq Listing Rule 5635(c)(4).

• October 07, 2024: Ultragenyx received Breakthrough Therapy Designation for Setrusumab (UX143) in treating Osteogenesis Imperfecta.

New Partnerships

• Acquisition: In 2022, Ultragenyx acquired GeneTx Biotherapeutics to develop an investigational drug to treat Angelman syndrome.

Development Approach

• Trial Design: Utilizing innovative techniques such as adaptive trial designs and seamless Phase 1/2/3 trials to accelerate the drug development process.

Conclusion

Ultragenyx Pharmaceutical Inc. is a pioneering company in the biopharmaceutical industry devoted to developing treatments for rare and ultrarare diseases. Through a blend of innovative therapeutic approaches and a commitment to patient-focused healthcare, Ultragenyx continuously expands its footprint in the rare disease community globally.

Note: The above report includes only the information extracted from the provided data. "No information is available" indicates unavailable data from the sources given.