| Market Capitalization (Millions $) |
- |
| Shares
Outstanding (Millions) |
35 |
| Employees |
- |
| Revenues (TTM) (Millions $) |
36 |
| Net Income (TTM) (Millions $) |
-627 |
| Cash Flow (TTM) (Millions $) |
124 |
| Capital Exp. (TTM) (Millions $) |
2 |
Business Description
A biopharmaceutical company that is dedicated to discovering novel therapies for a range of rare neurodegenerative diseases, including lysosomal storage disorders, protein aggregation, and mitochondrial dysfunction.
Orphazyme A is a Danish company that was founded in 2009, and it is headquartered in Copenhagen. The company has a strong focus on developing treatments for rare diseases, which are often overlooked by larger pharmaceutical companies. Its mission is to improve the quality of life for patients suffering from these conditions by providing effective and innovative therapies.
The company's main focus is on the development of arimoclomol, a candidate drug that has shown promising results in the treatment of several rare neurological disorders. Arimoclomol is a small molecule that acts by increasing the production of heat shock proteins, which are responsible for helping the body repair damaged proteins and prevent protein misfolding, a process that is implicated in many neurodegenerative diseases.
Orphazyme A has conducted several clinical trials to test the safety and efficacy of arimoclomol in patients with different rare diseases. One of the most promising trials was conducted in patients with Niemann-Pick disease type C (NPC), a rare genetic disorder characterized by a deficiency in the NPC protein, which leads to the accumulation of cholesterol in the brain and other tissues.
In this trial, arimoclomol demonstrated significant improvements in several important disease parameters, including motor function, speech, and swallowing ability. The drug was also found to be safe and well-tolerated in these patients.
Orphazyme A has several ongoing clinical trials exploring the use of arimoclomol in other rare diseases, including amyotrophic lateral sclerosis (ALS) and sporadic inclusion body myositis (sIBM). The company is also exploring other small molecules that target different aspects of protein aggregation and misfolding.
In addition to its focus on drug development, Orphazyme A is also committed to patient advocacy and awareness-raising. The company works closely with patient organizations and advocates to ensure that the needs of rare disease patients are addressed and that their voices are heard.
Orphazyme A is a leading player in the field of rare diseases and has made significant progress in developing innovative therapies for these conditions. Its dedication to patient advocacy and awareness-raising is an important part of its mission to improve the lives of those suffering from rare diseases.
Company Address: Ole Maaløes Vej 3 Copenhagen N 0
Company Phone Number: 28 98 90 55 Stock Exchange / Ticker: ORPH
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