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Gene Silencing Treatments Licensed for Heart Disease

Heart check

(Gerd Altmann, Pixabay)

30 September 2016. Biopharmaceutical company Amgen is acquiring the rights to a technology that stops the actions of inherited genes associated with a leading type of heart disease. The deal is expected to provide Arrowhead Pharmaceuticals Inc., a biotechnology company in Pasadena, California, with as much as $674 million.

Arrowhead’s technology is based on ribonucleic acid or RNA interference (RNAi), a natural process to silence the expression of genes causing disease. RNA is genetic material related to DNA that the body uses to transmit genetic information to cells and synthesize proteins. RNA interference targets specific genes, making it a potentially powerful therapeutic technique, while minimizing damage to other genes, thus limiting side effects.

The company delivers RNAi molecules in nanoscale — 5 to 20 nanometer — packets called dynamic polyconjugates made of biocompatible polymers, combined with signaling molecules that seek out specific cell targets in the body. The dynamic polyconjugate packet binds to the cells, releasing the RNAi molecules to turn on or off the targeted genes.

The target in this case is lipoprotein(a), a protein that dissolves or combines with other lipids or natural oils in the blood stream. People with elevated lipoprotein(a) levels, an inherited condition, face the prospect of the material accumulating in and narrowing arteries, resulting in a form of atherosclerosis, leading to heart attacks and strokes. Lipoprotein(a) Foundation says 1 in 5 people have this inherited disorder.

Arrowhead’s experimental drug code-named ARC-LPA applies its RNA interference techniques to block the genetic process producing a component of lipoprotein(a) known as apolipoprotein A. The company says ARC-LPA is its first drug candidate formulated for injections under the skin, currently in preclinical testing.

The agreement gives Amgen, in Thousand Oaks, California, a worldwide exclusive license to ARC-LPA, where Amgen will be responsible for clinical trials and commercialization. Under the deal, Amgen is paying Arrowhead an initial licensing fee of $35 million, and taking an equity stake in Arrowhead valued at $21.5 million. In addition, Arrowhead will be eligible for future option, development, regulatory, and sales milestone payments totaling as much as $617 million, as well as royalties on sales of products developed under the deal.

In a separate agreement, Amgen is receiving an option for a worldwide exclusive license to a second RNA interference therapy developed by Arrowhead that addresses cardiovascular disease. Neither the precise target nor financial details of that deal were disclosed.

Arrowhead’s lead products are designed to address liver disorders, including hepatitis B and alpha-1 antitrypsin deficiency, now in early- or intermediate-stage clinical trials. Alpha-1 antitrypsin deficiency is caused by a mutation in the gene that makes the protein alpha-1 antitrypsin, produced in the liver and protecting the body from an enzyme called neutrophil elastase. Without the protection of alpha-1 antitrypsin, neutrophil elastase destroys air sacs in the lungs, resulting in a condition similar to chronic obstructive pulmonary disease or COPD.

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