Rare Diseases, Startups, Venture

$49M round; experienced team; A-list VCs — Inozyme launches with rare disease strategy and a yen for bigger things to come

Axel Bolte

It was Axel Bolte’s work as a venture partner for HBM that originally took him to the campus at Yale, with a chance to start meeting the scientists there. And that introduction eventually led him to Demetrios Braddock, who had been doing lab work on calcification that he’s devoutly hoping could have an impact on a long line of diseases.

“Ultimately I always wanted to be directly involved as an entrepreneur and operational in companies,” says Bolte, “which is what I did before in large organizations.”

It was Braddock’s work that made that happen. And today Bolte is officially opening the shop at Inozyme, the latest in a long lineup of startups to set out to make their mark in Cambridge, MA.

The real research work at Inozyme got started early this year, Bolte tells me. That was after he spent much of 2016 raising cash, which led to today’s announced $49 million A round with some VCs he knows well. Longitude Capital took the lead for a round that includes New Enterprise Associates, Novo Ventures and Sanofi Ventures.

Demetrios Braddock

They’re backing a very familiar strategy that starts with rare diseases, a classic strategy that allows for pivotal data on small patient groups.

Their lead enzyme replacement therapy is for the treatment of Generalized Arterial Calcification of Infancy — GACI, which hits newborns — and Autosomal Recessive Hypophosphatemic Rickets Type 2.  GACI is triggered by a genetic mutation that causes low levels of pyrophosphate and calcification of the arteries and heart. But after getting established in rare diseases, Bolte sees a bigger future ahead in major diseases like chronic kidney disease.

Right now the company has 8 staffers, right in line for where it’s at at this stage. It includes some highly experienced biotechies with resumes that lean heavily on rare diseases:

  • COO Henric Bjarke: former vice president and therapeutic area head for the metabolic business unit at Alexion, where he was responsible for asfotase-alfa for the treatment hypophosphatasia and other development programs.
  • Chief technical operations officer Steven Jungles, a vet from Ultragenyx and BioMarin.
  • Chief medical officer Eric Yuen, with stints at Merck, Johnson & Johnson, Ultragenyx and BioClin.
  • Regulatory chief Ruhi Ahmed from Ultragenyx and BioMarin.
  • Finance VP Stephen Basso out of Alexion.

Now Bolte plans to double that number as the company heads to the clinic.

“We have some experience in CMC and these kinds of things,” says the CEO. “We expect an IND in 2019 and then clinical trials should start at the end of ’19, with initial trial results in 2020.”


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