Flagship-backed AI startup Valo shelves eye disease drug

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Valo Health chief executive Brian Alexander, who is also CEO-Partner of Flagship Pioneering

Valo Health chief executive Brian Alexander, who is also CEO-Partner of Flagship Pioneering

Valo Health has said it will no longer develop a drug candidate for diabetic retinopathy on its own after a phase 2 readout – but will seek a partner to take the programme forward.

The start-up – founded by investment group Flagship Pioneering – said that OPL-0401 did not meet either the primary or secondary endpoints of the SPECTRA trial, but added that the drug was well-tolerated and some of the data points to potential for preventing disease progression in diabetic retinopathy, a leading cause of blindness.

It's a disappointing outcome for Valo Health, as OPL-0401 – a drug in the Rho kinase (ROCK1/2) inhibitor class – is its lead and only clinical-stage drug candidate. However, the company was quick to point out that it did not emerge from its Opal computational drug discovery platform, which uses real-world patient data to discover and develop new therapies.

Opal is claimed to predict some characteristics missing in other AI-based drug discovery systems, such as pharmacokinetics, ADME (absorption, distribution, metabolism, and excretion), toxicity, and the functional effects of compounds, and has already attracted an R&D alliance with Novo Nordisk, which paid $60 million upfront to kick off programmes in cardiometabolic diseases in a deal worth up to $2.7 billion.

Now, Valo Health will focus its efforts on using Opal to "discover new therapeutic targets in real-world data, validate those targets in human-centric models, and develop new medicines with [its] AI-enabled closed loop small molecule design," said chief executive Brian Alexander, who is also CEO-Partner of Flagship Pioneering.

Alexander joined Valo Health and Flagship Pioneering last month, replacing interim CEO Graeme Bell, and previously held roles including CEO of genomic profiling specialist Foundation Medicine, a subsidiary of Roche.

He remains optimistic that there could be a role to play in diabetic retinopathy for OPL-0401, a small-molecule, orally active candidate that was originally licensed from Sanofi in 2021 and has been tested in around 300 patients to date.

"There is a significant unmet medical need for patients suffering from diabetic retinopathy, a leading cause of blindness in the US, and current treatment options consist mainly of invasive intravitreal injections and laser treatment targeted to patients with more severe or advanced disease," said Alexander.

He added that the totality of the data in non-proliferative diabetic retinopathy suggests clinical activity at "certain doses" and supports further clinical evaluation of the drug in this indication.