Whilst XLRP is obviously a different form of Retinitis Pigmentosa than the one that PYC’s VP-001 is aiming to treat, I see this as a very significant development for PYC.
I referred to this Lumeos Phase 3 trial in a post on this thread early last week (28 April) and noted that it had already been seven months since trial completion. The trial failure explains the delay.
As mentioned in that post, Meira GTx sold bota-vec to Janssen in December 2023 (during Phase 3) in a deal worth up to US$415m (A$650m).
The reason I mentioned the Lumeos trial was because Rohan Hockings had made a comparative reference to the MeiraGTx/Janssen AAV gene therapy treatment (bota-vec) in the Phase 1/2 VP-001 results presentation released that same day.
Currently, there is just one FDA approved gene therapy for an inherited retinal disease and that is Luxturna, acquired by Roche when it bought Spark Therapeutics for US$4.3 bn (A$6.7bn) in 2019. Luxturna is indicated for individuals with an inherited retinal dystrophy due to two mutations in the gene RPE65.
In March, Roche announced that that it had completely written off the value of Spark. Luxturna, Spark’s sole commercial product, has had disappointing sales. Sales last year were just US$20 m, down 59% YOY.
A comparative reference was also made to Luxturna in the PYC presentation released last Friday (Slide 29).
Gene therapies would appear to be losing their shine as the favoured modality for treatment of inherited retinal disease. Could eyes now turn to PYC’s RNA approach?
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(20min delay)