Amyotrophic lateral sclerosis (ALS) has no cure and few available treatments but the past decade has revealed new knowledge about the pathology of the disease. Libra Therapeutics is turning that research into potential therapies, and it now has $29 million to advance its work.
While the exact causes of many neurological disorders are unknown, Libra President and CEO Isaac Veinbergs says one common thread is toxic proteins. These proteins may have proper roles and functions, but mutations or misfolding can lead to an unhealthy protein buildup that is toxic to the body.
The name Libra, which translates from Latin as “balance” or “scales,” is a reference to the company’s goal of restoring balance to neurons, Veinbergs says. The San Diego-based biotech is developing drugs that get rid of protein aggregation by either reducing the production of toxic proteins or clearing away the protein that has already built up. The second approach taps into autophagy, a built-in cellular process for getting rid of cellular waste.
“It’s like taking out the trash,” says Veinbergs, a former executive of Acadia Pharmaceuticals (NASDAQ: ACAD) and Sanofi (NYSE: SNY). “It clears out the toxic proteins that build up in the neuron, thereby allowing the neuron to function properly.”
In ALS, the toxic protein buildup is caused by a mutation to C9ordf72, a gene that research has shown is involved in autophagy. The mutation leads to repeating sequences of DNA code, known as expansions, Veinbergs says. A Libra drug would increase autophagy to overcome the loss of the function of C9orf72. Veinbergs declined to specify what Libra’s drug would do or how it would work, other than to say it would increase the activity of the autophagy pathway, which in turn increases a cell’s ability to clear away toxic proteins.
Two other programs aim to decrease levels of toxic proteins by preventing the repeats produced by the mutated C9orf72 gene. Veinbergs says the approaches are not specific to that gene and could have applications to other neurological disorders, such as Huntington’s disease and frontotemporal dementia.
Libra’s fresh capital, a Series A round of financing, was co-led by Boehringer Ingelheim Venture Fund (BIVF), Epidarex Capital, and Santé. Other participants included Yonjin Venture, Dolby Family Ventures, and Sixty Degree Capital. Libra is based on research from Axxam, an Italy-based contract research organization. In addition to its contract services, the firm also starts research programs and then places them with pharmaceutical companies or finds financing to spin them out as independent entities.
Henning Steinhagen, a Germany-based venture partner at Epidarex, said via email that his firm’s strategy is to identify promising early-stage science, mainly in the US and the UK, and then work hands-on with founders to create new companies that have “exciting potential” for patients and investors. He says Epidarex and BIVF spent several months assessing and following Axxam’s neuroscience drug research, which included discussions about the science, the business plan, the budget, and how to shape the company. According to Veinbergs, Libra, the first US-based Axxam spinout, formed at the end of last year and began hunting for capital during the annual J.P. Morgan Healthcare Conference.
Libra is not the only company trying to treat ALS or FTD by harnessing autophagy or reducing the production of toxic proteins. Early this year, Cambridge, MA-based EnClear Therapies unveiled $10 million in Series A financing to support the development of a medical device that filters from cerebral spinal fluid the toxic proteins caused by C9orf72 mutations. In May, QurAlis, also based in Cambridge, raised $42 million to support research that includes an antisense oligonucleotide drug intended to block an enzyme that inhibits autophagy. Yet another Cambridge startup, Arkuda Therapeutics, emerged late last year with $44 million in financing and a small molecule FTD drug candidate intended to boost the function of the lysosome, the cellular component that carries out autophagy.
Veinbergs says that unlike some of the experimental neuro drugs in development, Libra’s medicines will be small molecules, which have a better chance of crossing the blood-brain barrier than large molecule drugs. Small molecules can also be made into pills, which are easier for patients to take than large molecule drugs that must be injected into the spinal cord or the brain.
The research for all three Libra programs is preclinical. The company’s plan is to first develop those programs for ALS before testing them in other disorders such as Alzheimer’s, Parkinson’s, and Huntington’s. Veinbergs says ALS is the lead target due to the high unmet medical need and the rapid progression of the disorder. That fast progression means the company can run a quicker, more focused clinical trial that won’t burn as much cash as a longer one, such as an Alzheimer’s disease study. Veinbergs added that it’s too early to say when the company’s experimental therapies could reach human testing.
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