Clinical Trials

Date: 2017-06-26

Type of information: Presentation of results at a congress

phase: preclinical

Announcement: presentation of results at the International Society of Thrombosis and Haemostasis (ISTH) Congress

Company: Alnylam Therapeutics (USA - MA)

Product: ALN-F12

Action mechanism:

RNAi. ALN-F12 is an investigational RNAi therapeutic targeting F12 for the treatment of hereditary angioedema (HAE). The F12 gene encodes coagulation factor XII ( FXII) but it also plays a role in stimulating inflammation. When its is activated, it also interacts with plasma prekallikrein. This interaction leads to the release of a bradykinin that promotes inflammation by increasing the permeability of blood vessel walls. Certain mutations in the F12 gene result in the production of factor XII with increased activity. As a result, more bradykinin is generated and blood vessel walls become more leaky, which leads to episodes of swelling in people with hereditary angioedema type III.
Pre-clinical data showed that administration of ALN-F12 resulted in dose-dependent reduction of vascular permeability in two different mouse models of bradykinin-driven vascular leakage. In non-human primates, a single subcutaneous dose of ALN-F12 at 3 mg/kg resulted in potent and durable knockdown of serum FXII of greater than 85 percent, with knockdown of over 50 percent sustained out to three months following administration.

Disease: hereditary angioedema, prevention of thrombosis

Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases

Country:

Trial details:

Latest news:

• On June 26, 2017, Alnylam Pharmaceuticals announced that the company will be presenting nonclinical data on ALN-F12, a development candidate targeting Factor XII (ALN-F12) for the treatment of hereditary angioedema and the prevention of thrombosis at the International Society of Thrombosis and Haemostasis (ISTH) Congress, being held from July 8 – 13, 2017 in Berlin, Germany.