12:17 PM EDT, 08/26/2024 (MT Newswires) -- PTC Therapeutics ( PTCT ) is positioned for potential upside with several key catalysts on the horizon, UBS said in a note Monday.

"With multiple [New Drug Application] filings this year & potential commercial launches next year (PKU, FA, AADC) along with upcoming clinical catalysts [HD data and ALS Phase 2 results in Q4), we see upside potential for shares," UBS said.

The Committee for Medicinal Products for Human Use of the European Medical Agency has issued a negative opinion on the renewal of the conditional marketing authorization of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy in June.

PTC is seeking marketing authorization from the European Medicines Agency for sepiapterin as a treatment for phenylketonuria, or PKU, a rare metabolic disease that affects the brain.

The company said last month the application for European marketing authorization for sepiapterin is under review while marketing authorization applications for Japan and Brazil are anticipated to be filed later this year.

UBS said that while Translarna's recent removal from the European market has overshadowed PTC Therapeutics' ( PTCT ) pipeline, it sees an "upside" in the potential approval/launch of PKU treatment next year.

The brokerage said it estimates unadjusted peak sales of $1.3 billion for metabolic disorder treatment sepiapterin due to its oral administration advantage and broader eligible age range.

The US Food and Drug Administration in May granted priority review for its biologics license application for gene therapy Upstaza, a one-time gene replacement therapy for aromatic L-amino acid decarboxylase, or AADC, deficiency in patients 18 months and older.

Upcoming decisions on AADC and FA, as well as potential regulatory feedback on HD, further enhance PTC Therapeutics' ( PTCT ) outlook, UBS said.

UBS said it resumed PTC Therapeutics ( PTCT ) coverage with a buy rating and a $47 price target.

Shares of the company rose more than 1% in recent trading.

Price: 34.58, Change: +0.43, Percent Change: +1.26