AC Immune wins $500K to advance TDP-43 diagnostic programs

Grants were awarded by Michael J. Fox, Target ALS foundations

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by Mary Chapman |

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AC Immune has been awarded more than $500,000 in nonprofit grants by the Michael J. Fox Foundation (MJFF) and the Target ALS Foundation to support its programs to detect abnormal forms of the TDP-43 protein via imaging scans and fluid samples.

“It is an honor to have the support of MJFF and Target ALS, two leading international organizations that recognize the pressing need for diagnostics to detect pathological TDP-43,” said Andrea Pfeifer, PhD, AC Immune CEO, in a company press release.

TDP-43 accumulates and forms toxic protein clumps in nerve cells in about 97% of amyotrophic lateral sclerosis (ALS) patients, which is associated with nerve cell dysfunction. This buildup also occurs in about half of the patients with the neurodegenerative disease frontotemporal degeneration (FTD).

While this makes it a promising disease biomarker for ALS and FTD, there are no technologies that accurately detect abnormal forms of this protein in tissues of the central nervous system (the brain and spinal cord) or in biofluids such as the blood or spinal fluid.

The two grants are meant to help AC Immune advance its efforts to develop these technologies.

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MJFF is supporting AC Immune’s TDP-43 positron emission tomography (PET) tracer program to produce the first imaging agent capable of detecting and monitoring the progression of neurodegenerative diseases associated with TDP-43 defects.

The Target ALS grant, meanwhile, will support AC Immune’s collaboration with world-class institutions to develop innovative biofluid tests to detect TDP-43-associated disease.

“We firmly believe that a sensitive and accurate diagnostic will represent a breakthrough for the field and will accelerate clinical development of therapeutic candidates against this novel target,” Pfeifer said. “Given the heterogeneity and irreversible nature of neurodegeneration, our precision medicine approach represents the most promising strategy to identify the right patients and treat them earlier.”

The company’s TDP-43-PET tracers have been able to target TDP-43 as intended and with high selectivity in brain tissue. AC Immune expects to announce a clinical trial candidate this year.

“Brain imaging agents uncovering aggregated pathological protein hold great promise to enable earlier and more accurate diagnosis of [neurodegenerative diseases], and we are pleased to be expanding our relationship with AC Immune to supports its TDP-43 tracer program,” said Jamie Eberling, PhD, MJFF senior vice president of research resources. “AC Immune and its collaborators recently demonstrated their expertise in developing cutting-edge PET imaging agents by providing the first images of alpha synuclein. With this new grant, we hope to make similar progress in the development of a TDP-43-PET tracer.”

The Target ALS grant will accelerate the efforts of a consortium comprised of AC Immune, Kansas City University, the Barrow Neurological Institute, and the International Center for Engineering and Biotechnology to develop an antibody-based assay to detect disease-related species of TDP-43 in fluid samples.

“We are delighted to support the collaborative consortium in which AC Immune is participating,” said Manish Raisinghani, PhD, Target ALS CEO. “The development of a TDP-43 specific biofluid-based diagnostic test has the potential to more rapidly enable confirmed early diagnosis.”