Large Granular Lymphocytic Leukemia

NEWTON, MA, USA I February 12, 2025 I Abcuro, Inc., a clinical-stage biotechnology company developing therapies for the treatment of autoimmune diseases and cancer through precise modulation of cytotoxic T cells, today announced the closing of a $200 million Series C financing led by New Enterprise Associates (NEA) with Foresite Capital joining the round and participation of existing investors including RA Capital Management, Bain Capital Life Sciences, Redmile Group, Samsara BioCapital, Sanofi Ventures, Pontifax, Mass General Brigham Ventures, New Leaf Ventures, funds managed by abrdn Inc., funds and accounts managed by BlackRock, Eurofarma Ventures, and Soleus Capital. Proceeds from the Series C financing will be used to complete the registrational Phase 2/3 MUSCLE clinical trial evaluating ulviprubart (ABC008), a first-in-class monoclonal antibody targeting killer cell lectin like receptor G1 (KLRG1), for the treatment of inclusion body myositis (IBM). Assuming positive results from the MUSCLE clinical trial, Abcuro plans to file a BLA and will use a portion of the proceeds to support commercial launch preparation. “Continued support from all of our investors in this latest financing round 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, including Inclusion Body Myositis” said Alex Martin, Chief Executive Officer of Abcuro. “We are in a strong position to execute on our clinical development plan, including completing our ongoing, registrational Phase 2/3 MUSCLE clinical trial of ulviprubart in IBM, and expect to report initial data in the first half of 2026. We will also look to fund the expansion of manufacturing capabilities and other pre-commercial activities this year.” “Abcuro represents an exciting opportunity with its lead candidate, ulviprubart, a potential first-in-class therapy that could make a big impact to the treatment paradigm of IBM, an indication with a significant unmet clinical need,” said Michele Park, PhD, Partner at NEA. “Ulviprubart targets a unique mechanism that can selectively deplete cytotoxic T cells, backed by encouraging clinical and preclinical data that have been presented to date.” About Ulviprubart Ulviprubart (ABC008) is a first-in-class anti-KLRG1 antibody product candidate capable of selectively depleting highly cytotoxic T cells, while sparing naïve, regulatory and central memory T cells. Ulviprubart is designed to treat diseases mediated by highly cytotoxic T cells, including the autoimmune muscle disease inclusion body myositis (IBM) and T cell large granular lymphocytic leukemia (T-LGLL). The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have each granted orphan drug designation to ulviprubart for the treatment of IBM. About Inclusion Body Myositis (IBM) IBM is an autoimmune disease in which highly cytotoxic T cells chronically attack muscle tissue leading to progressive weakness and limb muscle atrophy. Patients progressively lose muscle function, including loss of grip, dexterity and mobility. There are currently no available disease-modifying treatment options for IBM. Based on published epidemiology literature, it is estimated that there are more than 50,000 people with IBM across the US and Europe. About Abcuro Abcuro is a clinical stage biotechnology company developing first-in-class immunotherapies for the treatment of autoimmune diseases and cancer through precise modulation of highly cytotoxic T cells. The company’s lead program is ulviprubart (ABC008) and is currently in clinical trials for inclusion body myositis (IBM) and T cell large granular lymphocytic leukemia. For more information, visit us on LinkedIn and at abcuro.com . SOURCE: Abcuro

Abcuro is earmarking a portion of new series C funds to prepare for a potential commercial launch of ulviprubart.\n Sanofi-backed Abcuro has secured $200 million in series C funds to power its anti-KLRG1 antibody through a phase 2/3 muscle disease trial ahead of a planned push to regulators.Abcuro is targeting KLRG1 to drive the depletion of cytotoxic T cells in autoimmune diseases. While other players in the space are targeting CD25, CD2, CD52 and ICOS with the same goal in mind, Abcuro has been betting on KLRG1 because expression is restricted to highly cytotoxic late-differentiated effector memory and effector T cells.The idea is that its anti-KLRG1 antibody ulviprubart, also known as ABC008, will deplete cells that drive autoimmune disease without affecting regulatory T cells and central memory T cells.Ulviprubart is currently in a phase 2/3 trial for inclusion body myositis (IBM), a muscle wasting disease that affects about 50,000 people across the U.S. and Europe. Abcuro has a lot riding on the study, with plans to file an approval application with the FDA if the study is a success when it reads out in the first half of 2026.To that end, the biotech is also earmarking a portion of the series C funds to prepare for a potential commercial launch of ulviprubart. Some more of the cash will be spent on expanding the company’s manufacturing capabilities.  The financing round was led by New Enterprise Associates, with new investor Foresite Capital joining a list of previous backers like Sanofi Ventures, RA Capital, Bain Capital Life Sciences, Redmile Group, Samsara BioCapital, Pontifax, Mass General Brigham Ventures, New Leaf Ventures and funds and accounts managed by BlackRock, to name a few.“Continued support from all of our investors in this latest financing round 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, including inclusion body myositis,” Abcuro CEO Alex Martin said in a Feb. 12 release.IBM looks like a good proving ground for ulviprubart’s potential. The disease is driven by T-cell attacks on muscle fibers that lead to loss of hand function and ability to walk. In 2019, Steven Greenberg, M.D., a neurologist at Brigham and Women’s Hospital and Abcuro founder, co-authored a paper that pointed to a link between KLRG1-expressing T cells and IBM.It led Mass General Brigham Ventures to team up with Sanofi Ventures to oversee a series A-1 round back in 2021 to build on Greenberg’s research. The biotech followed this up with a $155 million series B in 2023.As well as the later-stage study in IBM, the company is also overseeing a phase 1/2 clinical trial of ulviprubart in T-cell large granular lymphocytic leukemia.

After quickly enrolling its pivotal trial and recruiting more patients with a rare muscle disease than anticipated, Abcuro went to its board to explore a $60 million to $80 million fundraise. That initial ask in May turned into a $200 million Series C, CEO Alex Martin told Endpoints News , and the fresh funding is expected to help the company prepare to commercialize its monoclonal antibody, dubbed ulviprubart, or ABC008. The Newton, MA-based biotech announced the financing on Thursday morning. New Enterprise Associates led the round, while Foresite Capital also joined the investor syndicate alongside existing backers Bain Capital Life Sciences and RA Capital — and at least 10 other investors, including Sanofi’s venture arm. Abcuro expects to have data from a Phase 2/3 trial in inclusion body myositis (IBM) in the first half of 2026 and then file for approval in the US and Europe, Martin said. The company enrolled 272 patients against an original target of 231 participants for the 76-week trial. There are no FDA-approved medicines for the rare autoimmune disease mainly found in those 50 years and older. It leads to progressive muscle weakness and can thwart the ability to walk, swallow and perform other daily functions. The biotech’s drug targets killer cell lectin-like receptor G1, or KLRG1. By hampering cytotoxic T cells, and sparing regulatory and memory T cells, Abcuro thinks it can tamp down inflammation and help muscles “regenerate and decrease the chronic pathogenesis of the disease,” chief medical officer Jeff Wilkins told Endpoints in 2023 when the company announced a $155 million Series B . Wilkins noted that other treatments, including methotrexate and rituximab, have failed in IBM. Novartis tested an antibody as a treatment for IBM, but the investigational medicine was repurposed by another company for obesity after it failed in IBM. Eli Lilly now owns the asset, named bimagrumab. Orphazyme, which is now owned by Zevra, also tried arimoclomol as a treatment for IBM. The drug was approved last year for a different progressive disease, called Niemann-Pick disease type C. “We noticed that they had screen failed about 12% of patients” in the arimoclomol trial, Martin said. “We ended up screen failing almost twice the rate of that because we wanted to make sure we had a patient population we thought would be best suitable for this trial.” Abcuro has received offers for both acquisitions and partnerships from mid- and large-sized companies, according to Martin. Instead, the company is looking for a chief commercial officer and “beginning to look at the broader picture at the commercial team,” Martin said. It currently employs about 40 people. “Right now, we’re very comfortable with the plan that we have to take this drug through registration,” he said. Abcuro also plans to take early-stage clinical data in patients with T cell large granular lymphocytic leukemia to the FDA in the “coming quarter or two” to see about a Phase 2/3 program for ulviprubart, according to Martin. It’s also considering moving into other indications like multiple sclerosis or Sjögren’s disease, the CEO said. “We’re going to learn a lot from the IBM trial,” Martin said. It’ll help inform whether Abcuro wants to “stick in the neurology area or we want to venture further afield. There’s implications in type 1 diabetes, lupus and other autoimmune diseases, so we’ll see what we see in the IBM trial and then make a decision.”