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Fly News Breaks for October 30, 2019
PTCT
Oct 30, 2019 | 07:11 EDT
Citi analyst Joel Beatty lowered his price target for PTC Therapeutics to $55 from $62 saying the company's Q3 earnings report is an incremental negative for the stock. The familial dysautonomia splicing program was discontinued, Friedreich's ataxia gene therapy investigational new drug application was delayed from the end of 2019 to mid-2020, 2019 opex guidance was increased by $20M, and Tegsedi market size estimate appears to have dropped to 5,000 from 6,000, Beatty tells investors in a research note. The analyst, however, keeps a Buy rating on the shares.
News For PTCT From the Last 2 Days
PTCT
Mar 28, 2024 | 08:34 EDT
PTC Therapeutics submitted the sepiapterin MAA to the European Medicines Agency, or EMA. The MAA submission is for the treatment of pediatric and adult patients with Phenylketonuria, including the full spectrum of disease subtypes. Phenylketonuria, or PKU, is a rare, inherited metabolic disease, which affects the brain. The sepiapterin MAA includes the results of the phase 3 APHENITY trial in which sepiapterin had a statistically significant and clinically meaningful reduction in blood phenylalanine levels in pediatric and adult PKU patients. PTC expects to submit the sepiapterin NDA to the FDA no later than the third quarter of 2024. Submissions in 2024 are planned in a number of additional key countries where PTC has existing rare disease commercial infrastructure including Brazil and Japan.