Libra Therapeutics, a San Diego startup focused on applying autophagy to counter the loss of protein function in neurodegenerative diseases, said today it has formally launched by raising $29 million in Series A financing.
Libra’s small molecule therapeutics are designed to restore cellular balance disrupted by the production and decreased clearance of neurotoxic proteins in neurodegenerative diseases that include amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, and frontotemporal dementia (FTD).
“ALS is our primary disease indication,” Isaac Veinbergs, Ph.D., President and CEO of Libra Therapeutics, told Clinical OMICs. “We’ve selected ALS for two reasons: One, the high unmet need and the tremendous burden on patients and caregivers and families. And two, from a clinical development plan, we have the ability to look at direct biomarkers—decreased production of dipeptide repeats.”
For both ALS and FTD, the most frequent genetic cause is the expansion of the C9orf72 gene, which has been shown in studies to regulate intracellular trafficking and autophagy in neuronal cells. These repeat expansions, according to Libra, have also been identified as potential contributors in other neurodegenerative diseases through the gain of toxicity caused by the production of the dipeptide repeats or RNA repeats.
Libra said its treatments are intended to increase autophagy to counter the loss of C9orf72 function, through a therapeutic platform designed to discover and develop novel small molecule drugs that can both increase autophagy to more rapidly clear toxic proteins and reduce the production of neurotoxic proteins.
Libra’s lead program is focused on increasing autophagy to more rapidly clear toxic proteins, while its two other programs are designed to pursue different paths toward attenuating the production of those proteins.
“We have three programs in our pipeline with distinct molecular approaches, and each is intended to go into ALS as a primary indication,” Veinbergs added. “These mechanisms are central to other neurodegenerative diseases, so we have the potential to explore these treatment approaches in other indications, such as Alzheimer’s, Parkinson’s, and Huntington’s.”
The company’s science was initially developed by Axxam, an Italian discovery research organization that provides Libra Therapeutics with novel and proprietary chemical matter, exclusive assays, and drug discovery expertise. Axxam identified interesting novel targets for neurodegenerative diseases and set up high-throughput screens.
Through the companies’ collaboration, Axxam said, Libra plans to further optimize the molecules it identifies as having treatment potential through assays on genetically validated targets
Libra said it will use proceeds from the financing not only to advance its pipeline of novel small molecule drug candidates for the treatment of neurodegenerative diseases, but to build its team.
Veinbergs joined Libra from ACADIA Pharmaceuticals, where he previously served as VP, Corporate Development. Earlier, he held positions at Sanofi and its Sanofi Genzyme subsidiary, where he advanced to Head of Business Development and Licensing, Multiple Sclerosis & Neurosciences.
“Over the next 6-12 months, Libra will be looking to hire key leadership, including CSO, head of chemistry, and head of R&D,” Veinbergs said. “Through Libra’s service providers, we have access to chemists and biologists, so we have a great opportunity to access their expertise and drive discovery while we are building out the team.”
Boehringer Ingelheim Venture Fund (BIVF), Epidarex Capital, and Santé co-led the Series A financing, with participation by Yonjin Venture, Dolby Family Ventures, and Sixty Degree Capital.
Libra Therapeutics takes a unique approach to tackling neurodegenerative diseases with two distinct but complementary approaches that target key pathways that drive neurodegeneration,” added Martín Heidecker, Ph.D., Managing Director, BIVF USA. “By modulating scientifically and genetically validated targets to decrease neurotoxic proteins, there’s a clearly defined development path with translational and clinical biomarkers.”