Published: Feb 22, 2023
By Tristan Manalac
PTC Therapeutics reported Q4 and full-year 2022 results Tuesday ahead of a pivotal 2023 featuring three late-stage readouts in registration-directed trials.
In an investor call, Stuart Peltz, Ph.D., founder and CEO, said the company is anticipating results from the Phase III APHENITY trial evaluating sepiapterin in phenylketonuria (PKU) in May 2023. APHENITY is comparing the candidate’s efficacy against placebo in 178 PKU patients. The study was initially scheduled to return results by the end of Q4, 2022.
PTC gained access to sepiapterin in 2020 when it bought Censa Pharmaceuticals in a cash-and-stock deal valued at $51 million upfront.
The New Jersey biotech also expects key data this spring from vatiquinone, an investigational small molecule inhibitor of the 15-lipoxygenase enzyme, which is central to inflammatory response and oxidative stress pathways in neurological diseases.
PTC is testing vatiquinone in a Phase II/III trial for mitochondrial disease-associated seizures, for which results are expected in the second quarter of 2023.
The candidate is also being trialed for Friedreich’s ataxia in the Phase III study MOVE-FA. Findings from this study are also anticipated in the second quarter of this year.
The company also expects to complete enrollment in the Phase II PIVOT-HD trial of PCT518 in Huntington’s disease. Data from the first 12 weeks of PIVOT-HD are also expected to roll in during the second quarter of 2023, Peltz said.
Shooting for 30% Growth in ‘23
On Tuesday, Peltz set the company’s goals for the coming year, saying that PTC expects to “continue to accelerate” its growth in 2023 and is gunning for total revenue between $940 million and $1.0 billion, “which would represent a more-than 30% year-over-year growth.”
In 2022, the New Jersey biotech recorded $740 million in revenue. At constant exchange rates, this represents a 37% year-over-year increase.
Much of PTC’s strong performance was driven by its Duchenne muscular dystrophy (DMD) franchise. Translarna (ataluren), an oral DMD therapeutic approved in the European Union, earned the company $288.6 million.
PTC’s U.S.-approved DMD drug is Emflaza (deflazacort), an oral corticosteroid greenlit by the FDA in 2017. Emflaza recorded $218.3 million in net product revenues in 2022.
Outside of its DMD business, PTC continues to earn royalties from spinal muscular atrophy drug Evrysdi (risdiplam), which it developed in collaboration with Roche. Evrysdi’s royalty revenue for 2022 was $113.5 million.