PYC has said that its latest animal studies have demonstrated efficacy at a dose that is substantially lower than competitive approaches for ASO administration in the vitreous without the benefit of PYC’s CPP delivery technology. Lower dose reduces the likelihood of adverse events. It then referenced the work of ASO therapy biotechs, Wave Life Sciences and ProQR.
The US biotech, Wave Life Sciences, only revealed its plans to develop therapies for the treatment of various rare, genetic eye diseases last October.
The company intends to use its proprietary stereopure oligonucleotide platform to develop twice-yearly, intravitreally injected, disease-modifying therapies to address a number of rare, genetically-defined eye diseases. Initially the company is targeting therapeutics in four inherited retinal conditions that result in progressive vision loss - retinitis pigmentosa, Stargardt disease, Usher syndrome type 2A and Leber congenital amaurosis. It said it expected to announce its first development candidate in H2 this year.
Wave has published data that show that a single intravitreal stereopure oligonucleotide injection in the eye of non-human primates (450 microgram dose) led to more than 95% knockdown of a target RNA in the retina for at least four months.
But Wave has suffered a few recent setbacks in another of its ASO development programs. In April, results from an early-stage clinical study were badly received by investors. In a Phase 1 trial of its Duchenne muscular dystrophy candidate, suvodirsen, adverse events worsened at dose levels of 7mg/kg and 10mg/kg.
At the same time the company unveiled details of a planned phase II/III trial. Investors were concerned that the chosen doses of 3mg/kg and 4.5mg/kg might be too low to show effectiveness, especially as Sarepta’s marginally effective drisapersen was dosed at 6mg/kg in clinical trials.
Dutch biotech ProQR, is also working on ASO therapies for the rare genetic eye diseases of retinitis pigmentosa, Stargardt disease, Usher syndrome and Leber’s congenital amaurosis.
Its retinitis pigmentosa candidate, QR-1123, was licensed last November from Ionis Pharmaceuticals and ProQR plans to begin a phase 1/2 clinical trial some time this year following submission of an IND to the FDA.
Last September, ProQR announced encouraging results from a Phase 1/2 study of its QR-110 ASO candidate therapy (sepofarsen) for Leber’s congenital amaurosis (LCA) type 10, which were compared favourably with Spark Therapeutic’s Luxturna. A clinically meaningful response was seen by ProQR in six of 10 patients at the interim stage, on visual acuity scores and on a mobility test. In April this year, ProQR moved sepofarsen into a planned two-year pivotal Phase 2/3 trial.
Early last year, Spark Therapeutics received FDA approval for Luxturna, its once-only treatment for individuals with inherited retinal disease caused by mutations in both copies of the RPE65 gene. Luxturna, an AAV2 vector, was the first in vivo gene therapy approved by the FDA.
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