Sensorion nabs rare pediatric designation for gene therapy; Avalo sells off remaining drug rights for $5M

2022-11-07

基因疗法优先审批

A French biotech is moving forward on its hearing loss gene therapy, and the FDA is giving the candidate a regulatory boost. Sensorion put out word Monday morning that the US regulatory agency granted Rare Pediatric Disease Designation to its lead gene therapy candidate, called OTOF-GT. It is being tested to treat otoferlin gene-mediated hearing loss, a hearing loss caused by otoferlin deficiency that affects up to 20,000 people in the US and Europe, per the biotech. The FDA’s designation, given for potential therapies for diseases that primarily affect children and fewer than 200,000 people in the US, allows a company to get a priority review voucher after a drug or biologic for a rare pediatric disease gets approved. The company added that it is planning to file to start clinical trials within the first half of next year. The company had failed a Phase II study earlier this year with hearing loss drug SENS-401. Avalo Therapeutics is squeezing some more cash out of previously sold candidates. The biotech announced that it is selling off its rights to future payments from candidates it previously out-licensed. to ES Therapeutics, the buyer, is paying a total of $5 million.The sale is expected to conclude by the end of the month. Per the agreement, Avalo is selling to ES all rights to milestone payments from Janssen Pharmaceuticals and others related to previous license agreements. Avalo CEO Garry Neil — who joined the biotech earlier this year — said in a statement that the sale strengthens the biotech’s balance sheet and supports its development of lead candidate AVTX-002 for non-eosinophilic asthma. An ongoing Phase II trial is expected to read out in the first half of 2023. Garry Neil replaced former CEO Mike Cola back in February, the same time as former CFO Schond Greenway left the company to “pursue other interests.” Board chairman Steven Boyd mentioned at the time that “we believe that Garry and Chris will be the right team to execute an optimal strategy focused on prioritizing our most promising programs.”

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