Abcuro raises $200m to take muscle disease drug to market

US biotech Abcuro has reeled in $200 million in third-round financing that should allow it to complete its pivotal trial of lead drug ulviprubart and file it for approval for inclusion body myositis (IBM).

IBM is an autoimmune disease affecting the muscles that cause muscle inflammation and weakness similar to that seen with muscular dystrophy, affecting an estimated 20,000 people in the US, according to the Muscular Dystrophy Association. It usually develops after age 50 and is more likely to affect men than women.

The new financing, led by NEA and joined by new investor Foresite Capital, as well as Abcuro's existing stable of investors that includes Sanofi, should support the company right through preparations for commercial launch, assuming its phase 2/3 MUSCLE trial delivers positive results.

The Series C "validates our vision for the potential that ulviprubart may have as a novel treatment for progressive and devastating diseases mediated by highly cytotoxic T-cells," commented Abcuro's chief executive, Alex Martin.

There are no FDA-approved treatments for IBM, which doesn't typically respond to corticosteroid or immunosuppressant therapy that can benefit patients with other autoimmune diseases. At the moment, patients are generally managed using physical therapy, occupational therapy, speech therapy, and exercise.

Ulviprubart – also known as ABC08 – is an antibody targeting killer cell lectin-like receptor G1 (KLRG1) and has the potential to become the first drug in that class to reach the market, according to Abcuro, which is hoping to have data from MUSCLE in the first half of next year.

The trial completed its enrolment target of around 230 patients in the middle of last year and is comparing ulviprubart given by subcutaneous injection at two dose levels to placebo, with the main outcome measures safety over 20 weeks and the average change in IBM Functional Rating Scales (IBMFRS) after 76 weeks of follow-up.

Along with the trial costs, some of the new financing is earmarked for "expansion of manufacturing capabilities and other pre-commercial activities this year," said Martin. Abcuro's Series B, completed in August 2023, raised $155 million.

"IBM is a tremendously debilitating disease, which drastically and irreversibly reduces quality of life," commented Paula Eichenbrenner, executive director of The Myositis Association.

"Exercise and physical therapy can preserve muscle and stave off disease progression, but only to a degree," she added. "The lack of disease-modifying treatment options places more burden on IBM families as they must help their loved ones to manage anxiety about the inevitable muscle loss they will endure and feelings of being trapped with this rare, catastrophic disease."

Ulviprubart is also being developed for other indications, notably T-cell large granular lymphocytic leukaemia (T-LGLL), which causes anaemia and neutropenia and can leave patients dependent on blood transfusions, and various lymphomas associated with T and NK cells.