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(Sharecast News) - A treatment for a progressive eye disease that is being developed by a Sycona-backed company has delivered positive topline trial results, the life sciences investor confirmed on Monday, sending shares in the FTSE 250 firm sharply higher.
The gene therapy laru-zova is being developed by Beacon Therapeutics, a Syncona portfolio company, for patients with XLRP, an inherited retinal disease that leads to progressive vision loss. There are currently more than 20,000 people in the US and Europe with the disease, for which there are no approved treatment options.
Syncona confirmed that a phase II/III trial had met its primary endpoint of demonstrating statistically significant low luminesce visual acuity (LLVA) in patients. A LLVA is an eye test that measures how well someone can read an eye chart when a dimming filter is placed in front of the eye.
Syncona said the full clinical dataset will be presented at the American Academy of Ophthalmology's annual meeting next month, but confirmed Beacon now planned to engage with global regulatory authorities. A rolling biologics licence application submission is expected to get underway for later this year.
Chris Hollywood, chief executive of Syncona Investment Management Ltd (SIML), said: "Seeing laru-zova generate positive pivotal data is a powerful validation of the potential we saw in this asset and of the work of everyone who has helped bring it to this point. The SIML team's view is that significant value can be accessed in life science investment at the late stage, and today's data marks a key value inflection point." Syncona's 38.4% holding in Beacon was valued at £183.4m as of 30 June.
Lance Baldo, Beacon chief executive, said: "These results represent a landmark moment for the thousands of patients worldwide living with XLRP who currently have no treatment option and no way to slow the loss of their sight."
As at 0845 BST, shares in Syncona had jumped nearly 8% at 120p.
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