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PTCT

PTC Therapeutics

$40.95

0.66 (1.64%)

13:03
10/07/18
10/07
13:03
10/07/18
13:03

PTC Therapeutics announces initial data from patient registry

PTC Therapeutics announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna, underscoring the long-term clinical benefit of Translarna when used in routine clinical practice in delaying irreversible muscle loss in children with Duchenne caused by a nonsense mutation, when compared with published natural history. The data show that children and adolescents receiving Translarna in the real-world setting are continuing to walk years longer than untreated children, and are staying more physically able. A time-to-event analysis for loss of ambulation has shown that patients on Translarna had a median age of loss of ambulation of 16.5 years of age - up to 5 years later than seen with natural disease progression in untreated children. The data were presented as a late breaker at the 23rd International Annual Congress of the World Muscle Society in Argentina. Patients who received Translarna in routine clinical practice also experienced a slower decline in their physical function compared with the placebo arm of Phase 3 Study 020, as measured by a series of timed function tests. Safety outcomes for patients in the STRIDE Registry were consistent with the known safety profile of Translarna. The analysis was based on data captured from 216 patients, the majority of whom had not been previously enrolled in an ataluren clinical trial, across 11 European countries and Israel. Patients had a mean age of 9.8 years at first assessment and had been diagnosed with Duchenne caused by a nonsense mutation at approximately five years of age. Almost 90% had previously or were still receiving corticosteroids.

PTCT PTC Therapeutics
$40.95

0.66 (1.64%)

10/03/18
JPMS
10/03/18
NO CHANGE
JPMS
PTC Therapeutics 'unfounded' weakness a buying opportunity, says JPMorgan
10/03/18
PIPR
10/03/18
NO CHANGE
PIPR
Overweight
PTC, Roche data add uncertainty to Biogen drug outlook, says Piper Jaffray
The risdiplam spinal muscular atrophy data released this morning by PTC Therapeutic (PTCT) and Roche (RHHBY) stack up fairly well to Biogen's (BIIB) Spinraza clinical data, Piper Jaffray analyst Christopher Raymond tells investors in a research note. Even taking into account all of the usual caveats regarding cross-trial comparisons, risdiplam is shaping up well compared to Spinraza, the analyst contends. He sees the new data as adding uncertainty to the Spinraza trajectory. Nonetheless, Raymond keeps an Overweight rating on shares of Biogen. The analyst made no changes to his Biogen model, but he admits risdiplam, if the data holds up, may impact Spinraza's ultimate market potential. Biogen in midday trading is up 2% to $351.41.
10/03/18
BOFA
10/03/18
UPGRADE
BOFA
Neutral
PTC Therapeutics upgraded to Neutral from Underperform at BofA/Merrill
10/03/18
BARD
10/03/18
NO CHANGE
Target $348
BARD
Neutral
Risdiplam data incrementally negative for Biogen, says Baird
Biogen analyst Brian Skorney views this morning's risdiplam data for use in treating spinal muscular atrophy patients as incrementally negative for Biogen (BIIB). The data from PTC Therapeutics (PTCT) and PTC Therapeutics (RHHBY) are compelling so far, and suggest that risdiplam could impact the market share of Biogen's Spinraza, if it comes to market, Skorney tells investors in a research note. He highlights risdiplam's convenience as an oral small molecule and says the Phase II data "continues to impress." The analyst keeps a Neutral rating on Biogen with a $348 price target. The stock in afternoon trading is up $6.90, or 2%, to $352.31.

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