PTC Therapeutics forged a financing deal valued at up to $1 billion with Blackstone that will allow it to deliver one new therapy every two or three years, the New Jersey-based biotech reported.
Looking to expand its pipeline, PTC Therapeutics forged a financing deal valued at up to $1 billion with Blackstone that will allow it to deliver one new therapy every two or three years, the New Jersey-based biotech reported Thursday.
Blackstone granted PTC an initial $350 million that will kick-start the company’s plans, with an additional $650 million waiting in reserve.
During a Thursday call with investors and media, Chief Executive Officer Stuart W. Peltz, Ph.D. said the financing will support the company’s mission to build enough programs in its pipeline at a steady state and “bring transformative medicines to patients globally.”
PTC intends to build its pipeline through a combination of potential external partnerships and deals as well as internal development.
“Blackstone gives us a great opportunity to accelerate, to take molecules that we have and have additional indications in them, as well as to look, which we already do, for assets that are later stage…or commercializable,” Peltz said. He indicated the investment would enable near-term commercialization efforts and the opportunity to add to the company’s later-stage pipeline.
Although the PTC leadership team did not provide details regarding the expanded pipeline, the company has identified some assets in its pipeline it hopes to explore for potential use in additional indications,” Emily Hill, chief financial officer, noted during the call. The initial $350 million round will allow for that, Hill said.
The financing was announced weeks after PTC temporarily halted the U.S. portion of its Phase II Huntington’s disease trial following a request from the FDA for additional data. The requested data is particularly related to the dosing and duration proposed in the 12-month study.
The trial is assessing PTC’s experimental drug candidate PTC518, which has been designed to selectively lower huntingtin mRNA and proteins.
Peltz said the PIVOT-HD study remains active and is currently enrolling in many European countries and Australia. Data from the 12-week portion of the trial is expected in the first half of 2023.
Beyond the Huntington’s program, Peltz said PTC remains on target for three important data results over the next nine months:
- Fourth Quarter 2022: A readout from the Phase III APHENITY trial assessing sepiapterin (PTC923) in Phenylketonuria is expected.
- Q1 2023: A readout from the MIT-E Phase II/III trial assessing vatiquinone for mitochondrial disease-associated seizures is anticipated.
- Q2 2023: Data from the MOVE-FA Phase III study evaluating vatiquinone in Friedreich’s ataxia is expected.
The company continues its quest to secure potential approval for its Duchenne Muscular Dystrophy drug, Translarna (ataluren), Peltz noted. The drug has been approved in Europe but rejected multiple times in the U.S.
With topline data from the Study 041 program showing positive benefits for patients with nonsense mutation DMD, a subset of the disease that impacts between 10% and 15% of patients, PTC is trying again for potential approval. However, the road may still be a difficult one, Peltz said.
Translarna demonstrated consistent treatment effect across multiple clinical endpoints in previous trials, the company reported. Pooled analysis from three placebo-controlled trials confirms significant treatment benefit, according to PTC.
The FDA provided initial feedback on the Study 041 data that suggests it “does not provide substantial evidence of effectiveness.”
Peltz said the company will follow up with the regulatory agency in order to understand whether or not the evidence from the intent-to-treat population in the trial, along with confirmatory evidence from other studies, could support approval.
There are “clear examples of this approach of using trial results along with confirmatory evidence to support an NDA filing, as we’ve seen recently for rare neurological diseases with Amylyx and Reata,” Peltz said.
He referenced the recent approval for Amylyx’s ALS drug and the potential approval of Reata’s Friedreich ataxia treatment.
Matthew Klein, chief operating officer, said PTC is “in the early innings” of what could become a series of back-and-forth discussions with the FDA.
PTC is certain the regulatory agency sees the data from the Translarna studies, as well as the potential “to satisfy what has been used for other sponsors and other rare disorders to meet the criteria and allow then for the (New Drug Application) submission,” he said.
