PTC Therapeutics, Inc. (NASDAQ: PTCT) stock surged 27.51% on June 18th, 2018 but again crashed over June 19th, 2018 (as of 1:02 PM GMT-4; Source: Google finance)
The company said its experimental drug for spinal muscular atrophy has helped the vast majority of babies who got the treatment. Over the weekend, PTC Therapeutics had presented promising research at the CureSMA researcher conference in Dallas.
Over a stretch of 182 days, 90 percent of babies with Type 1 SMA that received the therapy showed improvement in their conditions, including the ability to control their heads, roll or sit. The data were presented at the SMA Researcher Meeting in Dallas, along with footage showing the babies rolling, sitting and controlling their head movements. PTC Therapeutics’ experimental drug poses a serious threat to Spinraza, the first drug that was approved to treat SMA. Biogen Inc., that sells a treatment for spinal muscular atrophy called Spinraza, saw its shares fell by 5.9 percent. PTC is co-developing the drug with Roche. Novartis also is a pursuing an SMA therapy in the wake of its acquisition of Avexis. Biogen has two new drugs in the pipeline, including an MS drug it is developing in partnership with Alkermes that could help Biogen maintain its leadership in MS treatment. But Toung noted the new drugs “are at least two years away reaching the market. Moreover, Biogen has two new drugs in the pipeline, including an MS drug it is developing in partnership with Alkermes that could help Biogen maintain its leadership in MS treatment. But Toung noted the new drugs “are at least two years away reaching the market.
Additionally, PTCT has traded significantly higher after the company have recently published a positive update from its risdiplam trial in type 1 SMA babies. Risdiplam’s efficacy data is compared favorably with that of Spinraza, and the drug also boasts superior convenience and strong tolerability. Risdiplam results shown at CureSMA give the increased confidence that the drug is achieving a clinical benefit and while Roche is responsible for commercialization. The company believe that, if successful, risdiplam could conservatively net about $150 million in peak royalties to PTC as well as an additional $410 million in remaining milestones note. The prospects for risdiplam compound additional opportunities for Translarna’s label and geographic market expansion.