PTC Therapeutics reported Q4 and full-year 2022 results Tuesday ahead of a pivotal 2023 featuring three late-stage readouts in registration-directed trials.
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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.
In an interview with BioSpace, Peltz said PTC is hopeful of seeing the same regulation of huntingtin protein reduction in patients that it saw in a Phase I trial of healthy volunteers.
Over the next year, the company will be looking at biomarkers such as HTT protein in the CSF and neurofilaments.
“If those go down, and then the MRI goes along with that, we’ll have a set of data that could actually show that the drug is effective,” Peltz said. If that happens, he said, “there could be the potential for accelerated approval.”
This would then require a longer-term study to show full improvement, he added.
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.
Peltz said PTC is increasing its internal R&D efforts with the target of getting a product approved every two-to-three years. To accomplish this, “We think you need somewhere between 30 to 40 programs ongoing,” he said.
Based on Tuesday’s report, the company appears well on its way.
