Vanqua Bio Will Use $85M Funding to Advance New Tech Platform

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by Steve Bryson, PhD |

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Vanqua Bio has raised $85 million in funding to accelerate the development of its small molecule technology platform for treating neurodegenerative disorders such as Parkinson’s disease.

Led by Omega Funds, the Series B financing will advance the research of Dimitri Krainc, MD, PhD, of Northwestern University, an expert on molecular pathways underlying neurodegenerative diseases.

Researchers are hoping to develop next-generation small molecule therapies for Parkinson’s and other diseases, including Gaucher, Alzheimer’s, and amyotrophic lateral sclerosis (ALS).

“Vanqua Bio is helping usher in a new era of hope for people living with neurodegenerative disorders,” Jim Sullivan, PhD, co-founder and CEO of Vanqua Bio, said in a press release.

“We are a patient-founded company with a technology platform based on seminal research conducted by Dimitri Krainc” — chair of the neurology department at Northwestern’s Feinberg School of Medicine — “that is allowing us to identify a new generation of therapeutics with transformative potential,” Sullivan said.

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“Our mission — to develop effective therapies that slow or stop the progression of [Parkinson’s], [Alzheimer’s], ALS, and Gaucher disease — is a very personal one, and we are excited to have the support of world-class investors,” he added.

The company’s lead program is focused on developing small molecules that activate glucocerebrosidase, known as GCase, an enzyme that regulates fat and fat-like (lipid) molecules in cells.

Low GCase activity impairs the function of lysosomes, a cell’s recycling center, which leads to the buildup and aggregation (clumping) of toxic forms of alpha-synuclein protein, known as Lewy bodies. The accumulation of toxic alpha-synuclein is a hallmark of Parkinson’s.

Mutations in the gene — called GBA1 — that carries instruction for GCase cause Gaucher disease, an inherited metabolic disorder that affects the spleen, liver, nerves, and bones. GBA1 mutations also are associated with a form of Parkinson’s, called GBA-Parkinson’s disease, and a subset of Lewy body dementia (GBA–LBD) cases.

“Given the strong genetic validation for GCase as a target for GBA Parkinson’s and Gaucher patients, the high unmet need in these populations, and the expertise of the Vanqua Bio team in developing small molecule drugs, we are very enthusiastic about Vanqua’s strategy,” said Sara Nayeem, MD, of Avoro Ventures, one of the new investors.

The company will leverage proprietary research tools and in vitro (in the lab) disease modeling based on patient-derived nerve cells to translate genetic findings into effective therapies. Vanqua Bio expects to begin human testing of GCase activators within the next two years, it said, initially focusing on GBA-Parkinson’s and Gaucher.

“Of the eight million Parkinson’s patients around the world, up to 800,000 have GBA-PD. These individuals urgently need targeted therapies that can improve their outcomes,” said Jonathan Silverstein, a member of Vanqua’s board of directors and founder of the Silverstein Foundation for Parkinson’s with GBA.

“Vanqua Bio’s unique approach to discovering novel GCase activators holds great promise in enabling new targeted therapies for GBA-PD patients,” Silverstein said.

In addition to the GCase activator research, Vanqua Bio will focus on targeting an overactive innate immune system, which can stimulate the progression of several neurological diseases. The company said it plans to initially concentrate on ALS and Alzheimer’s, developing small molecule therapeutics as well as antisense oligonucleotides designed to modulate abnormal protein production.

The investor group included Series A investor OrbiMed, along with new investors Omega Funds, Avoro Ventures, Surveyor Capital, Eli Lilly, Casdin Capital, Pontifax, Logos Capital, and Osage University Partners.

Nayeem, of Avoro, and Bernard Davitian, a partner at Omega, are joining Vanqua Bio’s board, which includes Sullivan and Silverstein, as well as Mona Ashiya, PhD, of OrbiMed, and Stephen Squinto, PhD, also of OrbiMed.

“We are delighted to have successfully led this financing for Vanqua Bio, and to partner with Jim and his team and the company’s strong syndicate of dedicated, long-term investors,” Davitian said.

“Omega believes Vanqua Bio’s accomplished management team is well positioned to deliver on the immense potential of the company’s precision medicine approach to neurodegeneration,” he said.