With $29M in Series A, Boehringer-backed Libra looks to tackle neurodegeneration through cellular cleaning
Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?
That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.
“All these diseases have the commonality of the accumulation of toxic proteins that Libra intends to address,” Veinbergs told Endpoints News. “On one side preventing the production of these proteins, and on the other side increasing the clearance of these proteins, thereby enhancing the homeostasis of neurons and prolonging their viability and function.”
Wednesday’s round was co-led by the Boehringer Ingelheim Venture Fund (BIVF), Epidarex Capital and Santé. Other firms chipping in included Yonjin Venture, Dolby Family Ventures, and Sixty Degree Capital.
Libra’s science comes from Axxam S.p.A, an Italian research organization providing the new biotech with proprietary chemical matter. The central idea involves regulating cellular autophagy — that natural cleaning process — to combat the buildup of toxic proteins typically seen in neurodegenerative diseases.
In the lead program, Libra is focusing on the loss of function in the C9orf72 gene, which is the most frequent genetic cause of ALS and FTD. Though the entire mechanism of the gene is not fully understood, studies have suggested it is involved in intracellular trafficking and autophagy in neuronal cells, Veinbergs said. By boosting autophagy capabilities, Libra aims to counteract the lost activity and expunge the accumulated proteins.
“It’s taking out the trash from inside the cells in order to maintain a balanced and clean inside-the-cell status,” Veinbergs said. “Thereby the cells can function correctly, they don’t have a backup of these proteins that then are detrimental to the function of these neurons.”
Most cases of ALS are not hereditary, Veinbergs added, with about 10 to 20 percent being familial. About a third of such cases are associated with the expansion of C9orf72. Despite comprising a relatively small portion of all ALS cases, it’s the largest risk factor in familial ALS, and since so much about the disease is still unknown, Veinbergs believes it’s a good starting point.
Ultimately, Veinbergs wants this program to improve the quality of life for ALS patients rather than focus simply on delaying disease progression. Where Libra hopes to differentiate itself from other ALS treatments is in this regard, with Veinbergs saying currently approved therapies only intervene toward the end of the progression.
“What we would love to be able to accomplish is to try to slow down the progression of the disease to have more impact in the long run,” Veinbergs said. “Whether that translates into less neuronal loss in earlier or mid-to-late disease progression, that leads to, say, patients not going into a wheelchair, not needing a respirator, having better motoric controls in the earlier stages, that to me would be increasing in the quality of life.”
Libra’s two other preclinical programs, further along the pipeline, are directed less at the autophagy of cells and more at the production of the neurotoxic proteins themselves. The company is still building out its chemical matter there, though, and still working in animal models.
It’s also too early to give a timeline for when the lead program could hit the clinic, Veinbergs said. With the new funds, Libra is seeking to get there with the lead programs and get close with another. But everything will be dependent on the progress Libra makes within the next six to 12 months.
“We realize that this is an extremely challenging area, neurodegeneration as a whole,” Veinbergs said. “Having very good starting points from the chemistry perspective, from the capabilities perspective, I think gives us an edge on trying to progress these programs to where people have not had a lot of success.”
