Retrotope has ongoing trials for RT001 in three of the four most common orphan neurodegenerative diseases. Credit: Shutterstock

Retrotope is aiming for a $100m round in the first quarter of 2022 to fund late-stage development of an extensive neurodegenerative disease platform.

California-based Retrotope’s lead asset, RT001, is a synthetic linoleic acid that fights cellular degeneration and oxidative stress caused by lipid peroxidation, chief medical officer Peter Milner explained.

Currently, Retrotope has ongoing trials for RT001 in three of the four most common orphan neurodegenerative diseases— amyotrophic lateral sclerosis (ALS), progressive supranuclear palsy (PSP) and Friedreich’s ataxia (FA). The ALS and PSP trials are Phase II, while the FA trial is a Phase II/III potentially IND-enabling study, company president Anil Kumar said.

However, unlike most companies at this late development stage, Retrotope currently has no large institutional investors. Since its founding in 2006, about half of the $100m Retrotope has raised came from a single wealthy family in Silicon Valley, Kumar said.

Retrotope looks to broaden investor base

Though the family has indicated a willingness to continue providing funds, Retrotope will look to bring in additional investors willing to stick with the company as it likely goes public in the coming years, Kumar said.

Retrotope expects data by the end of 2021 for the FA and ALS trial, as well as data from a fourth ongoing trial in the rare disease infantile neuroaxonal dystrophy. As a result, the start of 2022 should mark an inflection point in Retrotope’s valuation, priming it for a fundraise, Kumar said.

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Elsewhere in its pipeline, Retrotope is preparing to bring its second asset RT011 – a synthetic docosahexaenoic acid – into clinic in early 2022 for the treatment of geographic atrophy, Kumar said. And depending on forthcoming decisions about Retrotope’s future pipeline plans – namely whether the company will pursue Alzheimer’s disease – the upcoming round could aim for as much as $150m, Kumar explained.

A previous Series D round was targeted at $40-50m, though it could increase to $60m should additional investors come in prior to additional data releases and the next round, Kumar noted.