Neuren Pharmaceuticals shrugs off short attack as key trial...

Neuren Pharmaceuticals shrugs off short attack as key trial results to be announcedCAMERON ENGLAND2 hours agoThe Australian Business Network0 CommentsNeuren is about to release new clinical trial data.Sales of Neuren Pharmaceuticals’ flagship Rett syndrome treatment Daybue have held up, flying in the face of a short attack launched earlier this year, as the company prepares to release key clinical data flowing on another drug’s use in treating Angelman syndrome.US outfit Culper Research released a report in February alleging that Daybue was shaping up as a “total flop”, in a bid to profit from shorting the stock of US-based Acadia Pharmaceuticals, which has the exclusive licence to market the drug.The Culper report said there were “horror stories” about adverse effects circulating through the Rett syndrome community and suggested that revenues from the drug would drop sharply.Neuren’s market update on Wednesday appears to put that theory to bed, with the company saying Acadia’s net sales for the second quarter of this calendar year were $US84.6m, up from $US75.9m in the first quarter, earning Neuren royalties of $13m.“The rate of new patient starts was 12 per cent higher than the previous quarter and the rate of discontinuations was 46 per cent lower than in the previous quarter,’’ Neuren said.“Penetration continues to increase, with approximately 30 per cent of the 5000 diagnosed patients having initiated therapy. “In market research, physicians surveyed stated that over the next 24 months they expect to expand prescribing to more than 70 per cent of their eligible patients.’’Neuren said while the momentum in the second quarter was encouraging, the rate of patient additions was slower than expected, which had caused Acadia to downgrade its net sales guidance for the calendar year to $US340m-$US370m, from $US370m-$US420m.“Assuming the updated guidance range is met and an exchange rate of 0.65, Neuren would earn royalties of $55m-61m (was $61m-70m), plus $77m from the first sales milestone payment of $US50 million due for the first calendar year in which net sales exceed $US250m,’’ Neuren said.Neuren said Acadia presented new real-world Daybue data in June, which showed caregivers for 67.7 per cent to 82.2 per cent of enrolled participants reported improvements at months one to six in at least one Rett syndrome symptoms category. “The most consistently reported improvements over six months were non-verbal communication, alertness and social interaction/connectedness,’’ Neuren said.The company also said there was the potential for Daybue to be approved for use in Canada around the end of this calendar year, with a marketing application to be filed in Europe early next year and discussions are under way in Japan.Neuren also placed its shares into a trading halt as it prepares to release top line results of its Phase 2 clinical trial in the use of compound NNZ-2591 in treating Angelman syndrome.The compound has already demonstrated positive top line results in a Phase 2 trial in patients with Pitt Hopkins Syndrome and Phelan McDermid syndrome.Neuren shares last changed hands at $17.09.CAMERON ENGLANDBUSINESS EDITOR