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A drug being co-developed by Roche to treat spinal muscular atrophy (SMA) helped improve development scores in babies with the genetic disease, a study released on Monday showed, as the race heats up for therapies destined to be among the drug industry’s most expensive.
PTC Therapeutics, which struck a licensing deal with Roche in 2011 for its SMA program, said more than 90 percent of babies with severe Type 1 SMA given the RG7916 drug achieved a greater than four-point increase in a test to measure their neuromuscular progress six months after treatment began.
PTC shares, which are listed on the Nasdaq, rose as much as 30 percent.
The companies hope their medicine, also known as risdiplam, will be approved to take on rival drug Spinraza from Biogen, which sells for $750,000 for the first year of therapy and about $375,000 annually after that.
Novartis is also quickly advancing in the SMA field with its $8.7 billion acquisition this year of U.S.-based Avexis that is working on a gene therapy for the disorder.
Analysts from Barclays project Novartis’s one-time treatment could run to $1.25 million per patient.
“We are delighted that up to 6.5-fold increase of protein production has translated into clinical impact for these babies,” PTC Chief Executive Stuart Peltz said in a statement about the RG7916 study, adding no babies required a tracheostomy or permanent ventilation since the study began, and no baby lost his or her ability to swallow.
SMA is an often-deadly genetic neuromuscular disorder caused by a missing or defective gene that normally produces a protein needed for development of motor neurons in the spinal cord. This leads to muscle wasting. Many babies with the severest form of the disorder die, while others never stand or walk.
Barclays analyst Emmanuel Papadakis expects that if Roche and PTC’s data holds up on RG7916 with regulators, it could become a fierce competitor to Spinraza, now the only licensed SMA therapy.
Cowen analysts also said on Monday they view PTC’s update as very encouraging for approval — and for competitiveness versus Spinraza, which is administered into the spine about four times a year. Roche’s and PTC’s drug is taken orally.
Novartis Chief Executive Vas Narasimhan also has high hopes for his own newly acquired SMA treatment, saying in April after the Swiss company bought Avexis that its gene therapy has “multi-billion dollar peak sales potential”.