Antisense Therapeutics’ FDA ruling could be worth ‘hundreds of millions’
Health & Biotech
Health & Biotech
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Antisense Therapeutics (ASX:ANP) shares are up more than 20 per cent in morning trade after the Melbourne-based biotech announced it had squeaked through an expiring US law and been deemed eligible for a regulatory voucher worth hundreds of millions of dollars.
Antisense said on Wednesday that the Food and Drug Administration had granted its request to designate its drug candidate ATL1102 as a drug for a rare pediatric disease – in this case, Duchenne muscular dystrophy (DMD), a rare and fatal muscle-wasting disease in boys.
This means that if ATL1102 is approved, Antisense would likely receive a priority review voucher – a “ticket” of sorts that can be used to cut the FDA approval process by four to six months, shortening the time it takes to bring a drug to market.
Created to incentivise industry interest in creating drugs for rare pediatric diseases, the vouchers can be sold on a secondary market for hundreds of millions of dollars.
Since 2009, when the first voucher was awarded, they have sold for between $US68 million and $US350 million ($95m to $490m), according to Antisense.
Kazia Therapeutics (ASX:KZA) earlier this month received a rare paediatric disease designation for its lead drug paxalisib in relation to a rare and aggressive childhood brain cancer, while Neuren Pharmaceuticals (ASX:NEU) was granted a rare pediatric disease designation in July for its possible treatment for Rett syndrome, a rare neurological disorder in girls.
Given that Antisense has a market capitalisation of just $40 million, receiving a voucher worth up to $490 million would obviously be huge for the company. (Kazia and Neuren aren’t worth too much more – $48 million and $130 million).
Antisense just beat the clock in getting the rare pediatric disease designation, as it was created by a law known as the Creating Hope Act that expires on Wednesday.
Under the sunset provisions, Antisense and other companies working on what the FDA has designated as rare pediatric diseases still could receive the vouchers if they receive final FDA approval for their drugs by September 30, 2022.
Legislation to extend the Creating Hope Act through 2026 is pending in Congress.
“We are very encouraged by the granting of the rare pediatric disease designation to ATL1102 by the FDA, which recognises a great need for new and improved therapies for boys with DMD,” Antisense chief executive Mark Diamond said. “We look forward to future interactions with FDA as we refine our strategy for development of ATL1102 in DMD in the US.”
As previously reported, ATL1102 is used to treat a key aspect of the disease process, muscle inflammation. The company is preparing for a Phase 2b clinical trial of the drug following a successful Phase 2 one.