Pfizer, NEA-backed rare disease startup bumps up Series A to $42M

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The first spinoff from Cydan, an orphan disease incubator created by Pfizer Ventures and New Enterprise Associates, is headed full steam ahead. Known as Vtesse, the startup has nabbed an additional $17 million to bump up its Series A financing to total $42 million.

The additional financing is complete an ongoing pivotal Phase IIb/III clinical trial for the company’s only candidate, VTS-270 to treat Niemann-Pick type C1 disease. New sites in France, Spain and Turkey for the global trial have just started enrolling patients in the sham-controlled trial that’s slated for about 51 subjects.

"This additional capital enables Vtesse to broaden development and expand trial access to patients globally," said NEA General Partner David Mott in a statement.  "The additional funding also allows Vtesse to invest in further product and technology improvements and to develop the commercial strategy to ensure product availability."

All the existing investors, including Alexandria Venture Investments, Bay City Capital, Lundbeckfond Ventures, NEA and Pfizer Venture Investments, participated in the extension of the financing.

Vtesse launched in January 2015, focused at the time on studying VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) as part of a Cooperative Research and Development Agreement with the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Center for Advancing Translational Sciences, each part of the National Institutes of Health.

Then in January 2016, the Gaithersburg, MD-based startup got a breakthrough therapy designation from the FDA for VTS-270 to treat NPC.

The ongoing Phase IIb/III prospective, randomized trial involves intrathecal administration of VTS-270 every two weeks. It already completed a dose-finding portion or the trial that selected a 900 mg dosage. In Phase I/II testing of 14 NPC patients, NPC Neurological Severity Score (NSS) was reduced by about 60% as compared to a matched natural history control group.

NPC is a genetic disease caused by a defect in cell lipid transportation that leads to excessive accumulation I the brain, liver and spleen. VTS-270 specifically targets cholesterol storage to bypass the NPC1/NPC2 pathway to enable normal cholesterol transportation in the lysosomes of cells. It’s expected to work to restore the normal cholesterol metabolism and regulation at the cellular level.

"The early development of VTS-270 has benefited from a close collaboration with parents, patient support groups, the National Institutes of Health (NIH), and our academic collaborators," said Vtesse President and CEO Ben Machielse. "We thank them for their ongoing efforts as we broaden the footprint of our pivotal clinical trial."

- here is the release

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