PTC Therapeutics’ results in SMA has Biogen nervous

Biogen currently has the only product on the market approved to treat spinal muscular atrophy (SMA), in Spinraza, and that treatment is pretty central to the biotech’s portfolio, bringing in $364 million in revenue in the first quarter.

So, news that a competitor had posted strong results at Phase 1, with a more convenient method of delivery came as a blow to the company – enough to send shares down over 5%.

The rival is PTC Therapeutics and its drug candidate risdiplam (RG7916). It released interim Phase 1 results, which showed that 90% of the 21 babies on the trial achieved a greater than 4-point increase in the CHOP-INTEND score.

The treatment itself is dosed orally, meaning that if it can carry these results forward through to later trial stages then it will pose a significant threat to Spinraza – this treatment has to be delivered directly into the spinal cord.

SMA is a genetic disorder which results in a loss of motor neurons and progressive muscle wasting that can lead to an early death.

"We are delighted that up to 6.5-fold increase of protein production has translated into clinical impact for these babies in the FIREFISH study," said Stuart W. Peltz, Chief Executive Officer of PTC Therapeutics. "The survival data and CHOP-INTEND scores are very promising, since babies with Type 1 SMA typically do not experience functional motor milestone improvement based on natural history. We look forward to sharing updates for the programs as the data further develop at upcoming medical meetings."

The main aim of the Phase 1 trial was not to test for efficacy but to determine safe dosing levels; however, the opportunity to see strong results came as a huge boon to the company, with its share price rocketing up by 27%.

It’s not the only company working in the area, other than Biogen there is also Novartis, which used its considerable financial firepower to acquire AveXis for $8.7 Billion. AveXis is working towards launching its treatment at some point next year.