Novo Nordisk to Test Treatment for ATTR Amyloidosis in Phase 2 Trial

Trial will evaluate NNC6019 in patients whose hearts are affected

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by Steve Bryson, PhD |

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Hands, pills, and a stethoscope frame a graph showing clinical trial results.

Novo Nordisk is moving forward on a global Phase 2 clinical trial evaluating its experimental treatment NNC6019 in people with hereditary transthyretin amyloidosis (ATTR) affecting the heart.

The company’s ATTR amyloidosis research program, recently acquired from Prothena, also includes NNC6019 as a treatment for familial amyloid polyneuropathy (FAP) — a type of hereditary ATTR amyloidosis that mainly affects the network of nerves, called the peripheral nervous system, that extend from the brain and spinal cord.

The Phase 2 study of NNC6019 expects to enroll up to 99 adults, ages 18–85, with heart disease caused by ATTR amyloidosis.

Recruiting is underway at some sites in the U.S., with additional study locations expected to open in other states and in Canada in North America, and in Europe and Asia. Altogether, the trial will be conducted at 27 locations.

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Novo Nordisk now testing ATTR amyloidosis treatment

Due to the advancement of the program in the Phase 2 study, Prothena has earned a $40 million milestone payment from Novo Nordisk, according to a press release. Under the terms of the partnership, Prothena is eligible to receive up to $1.2 billion based on several clinical development and sales milestones, including $100 million earned so far.

Hereditary ATTR amyloidosis is caused by mutations in the TTR gene, which lead to the production of a faulty version of a protein called transthyretin (TTR). Defective TTR proteins tend to form toxic deposits in various tissues and organs in the body.

FAP is a form of the disease in which these toxic protein aggregates, or clumps, mainly accumulate in peripheral nerves, leading to symptoms of nerve damage (neuropathy). Often, abnormal TTR deposits also build up in the heart, damaging muscle tissue and leading to a condition known as cardiomyopathy.

NNC6019 (formerly known as PRX004) is an antibody-based treatment designed to selectively target and clear the disease-causing faulty forms of TTR, without affecting the normal protein.

In preclinical studies, NNC6019 demonstrated the ability to block the formation of toxic deposits and promote their clearance.

Prior to Novo Nordisk’s acquisition, Prothena completed a Phase 1 trial (NCT03336580) that evaluated the investigational therapy in 21 people with hereditary ATTR amyloidosis, including FAP patients and individuals with other tissue involvement. NNC6019 was found to reduce nerve damage progression and improve heart function.

The new Phase 2 study (NCT05442047) is enrolling adults who have heart disease due to TTR amyloidosis. Eligible participants will be randomly assigned to receive one of two doses of NNC6019, or a placebo, both by into-the-vein infusion in addition to standard care. The infusions will be gvien once every four weeks for 52 weeks (one year).

The trial’s primary goal is to assess changes after one year, compared with a placebo, in the distance walked in six minutes test, and in the blood levels of NT-proBNP, a marker of heart strain. Secondary goals include heart muscle assessments, heart function and neuropathy questionnaires, and treatment-emergent adverse events, or side effects of the therapy.