PTC Therapeutics, Inc. (NASDAQ:PTCT) stock fell 0.43% (As on August 27, 11:53:02 AM UTC-4, Source: Google Finance) after UBS upgraded the company to Buy from Neutral with a $47 price target after resuming coverage of the name. With multiple new drug application filings this year and potential commercial launches next year along with upcoming clinical catalysts, there is upside potential for the shares, the analyst tells investors.

Analyst Eliana Merle said the potential rise is due to an underappreciation of the company’s pipeline. The biotech has two candidates, sepiapterin and vatiquinone, for, respectively, phenylketonuria and Friedreich ataxia. Applications for sepiapterin have been submitted to the U.S. FDA and the European Medicines Agency, while an application for vatiquinone to the FDA is expected by the end of the year. “We think the focus on Translarna headwinds (surprising removal from the market in Europe) has left many of the company’s other programs underappreciated,” Merle wrote.

On the other hand, the company has submitted an NDA to the FDA for sepiapterin for the treatment of PKU in July 2024. Further regulatory submissions are planned for Japan and Brazil later in 2024. PTC resubmitted the NDA for Translarna for the treatment of nmDMD in July 2024. PTC achieved all objectives in the interim readout of the Phase 2 PIVOT-HD study of PTC518 in Huntington’s disease patients, which were disclosed in the second quarter. PTC plans to submit an NDA for vatiquinone for the treatment of Friedreich ataxia in late 2024. PTC expects to share topline data for the CardinALS trial of utreloxastat for the treatment of ALS in the fourth quarter of 2024. The BLA for PTC’s gene therapy for AADC deficiency was accepted by FDA with priority review; the target regulatory action date is November 13, 2024.

Meanwhile, PTC anticipates total revenues for full year 2024 to be between $700 million and $750 million. PTC anticipates GAAP R&D and SG&A expenses for full year 2024 to be between $740 million and $835 million, including expected R&D expense milestone payments of up to $65 million. PTC anticipates up to $90 million of payments for full year 2024 upon achievement of potential regulatory success-based milestones from previous acquisitions, of which up to $65 million will be recorded as R&D operating expenses. As of June 30, 2024, $35 million in milestones have been achieved, of which $15 million has been recorded as R&D operating expenses and $20 million as contingent consideration payable.