As a problem solver and partner, Akcea Therapeutics Inc. (NASDAQ:AKCA) has worked with several healthcare providers over the years. Together they have been advancing transformative treatments for serious and rare diseases. The target has been RNA-targeted medicines. Through innovation, the company has been able to meet not only the needs of patients who desperately need therapy but also their physicians and caregivers.
The majority-owned affiliate of Ionis Pharmaceuticals, Inc. has been carrying out a Phase 2 clinical trial of vupanorsen (AKCEA-ANGPTL3-LRx); an investigational antisense therapy. From the data presented in an online Late-Breaking Clinical Trial Session at the ESC 2020, the treatment met the primary endpoint. Significant reductions in triglyceride (TG) levels were observed compared to placebo as well as multiple secondary endpoints. These were clear indicators of favorable safety and tolerability profile for vupanorsen a potential treatment for patients with certain cardiovascular diseases.
Results from the Phase 2 Study May Help Guide Disease Management Strategies
With the current situation in the world due to the COVID 19 pandemic, there are all reasons to be ahead of the normal disease management strategies; for immediate or future application. According to a professor of medicine, Department of Medicine, University of Montreal, Daniel Gaudet, the Phase 2 study of the investigational therapy is timely.
Its impressive and meaningful data could be a guide to clinical research and disease management strategies. From the 105 patients dosed with either 40 mg or 80 mg of vupanorsen every four weeks for six months, there were no adverse events other than injection site reactions. This was after the patients had explored different doses and dose regimens vs. placebo.
Akcea and Ionis Close a Worldwide Exclusive Licensing Agreement with Pfizer Inc
The two companies have been working together to bring to the market AKCEA-ANGPTL3-LRx. The Chief Scientific Officer of Pfizer, Mikael Dolsten, says that each of them has extensive levels of experience in cardiovascular and metabolic diseases. Hence, they are suited to accelerate the clinical development of antisense therapy. The agreement is worth $250 million as an upfront license fee and will be settled by two companies.