Libra Therapeutics Launches With Pipeline of 3 ALS Treatment Candidates

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by David Melamed, PhD |

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Libra Therapeutics has officially announced its launch with a platform dedicated to developing new treatments for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

The company also has secured $29 million in Series A funding. The funds will be used to expand Libra’s team and advance the development of three small molecule drug candidates that are part of the company’s pipeline, which has been built on research originally carried out by Axxam.

“My career has been dedicated to CNS drug discovery. I have seen firsthand the devastation diseases like ALS, Alzheimer’s, and Parkinson’s disease have on patients and their families,” Isaac Veinbergs, PhD, president and CEO of Libra Therapeutics, said in a press release.

“We are delighted to build on Axxam’s pioneering foundational research to accelerate the development of novel therapies with the potential to slow or prevent disease progression to deliver meaningful improvements in patient outcomes across a wide range of neurodegenerative diseases,” Veinbergs said.

Libra plans to take advantage of its unique platform of small molecule candidates to counteract the effects of mutations in the C9orf72 gene, the most common genetic cause of ALS and FTD.

The C9orf72 gene provides instructions to make a protein that has been shown to play a role in the transport and degradation of molecules, specifically within brain cells.

Mutations in this gene are thought to lead to the production of toxic molecules, and at the same time lower cells’ ability to process waste products, ultimately leading to the accumulation of toxic compounds that can cause neurodegenerative symptoms.

To tackle these issues, Libra is leveraging its therapeutic platform to discover and develop new small molecule drug candidates that can increase autophagy — the process by which a cell is able to clear waste products — and at the same time lower the production of toxic molecules inside nerve cells.

“Libra Therapeutics takes a unique approach to tackling neurodegenerative diseases with two distinct but complementary approaches that target key pathways that drive neurodegeneration,” said Martín Heidecker, PhD, managing director of the Boehringer Ingelheim Venture Fund (BIVF), who co-led Libra’s financing round.

“By modulating scientifically and genetically validated targets to decrease neurotoxic proteins, there’s a clearly defined development path with translational and clinical biomarkers,” Heidecker said.

In addition to BIVF, Libra’s Series A funding round was led by Epidarex Capital, and Santé, with additional contributions from Yonjin Venture, Dolby Family Ventures, and Sixty Degree Capital.

“We are delighted to have played a key role in the creation and funding of Libra Therapeutics as well as co-leading the financing,” said Henning Steinhagen, PhD, a venture partner at Epidarex Capital.

“The science behind the company is outstanding and has enabled the recruitment of a highly experienced executive and an exceptional international investor syndicate. By bringing together these key components, Libra Therapeutics has a highly differentiated platform to generate novel therapies that aim to improve the lives of patients with neurodegenerative diseases,” Steinhagen added.