“I’ll remind you, if you do wish to ask a question, press zero/one on your telephone keypad,” said the operator, breaking an awkwardly long and silent pause during Oncopeptides’ press conference on Monday.
But no questions were forthcoming after the company’s CEO explained Oncopeptides had rescinded its letter to the FDA three months ago that effectively pulled its multiple myeloma treatment Pepaxto—which had an accelerated approval from the agency—off the market.
There were no questions for CEO Jakob Lindberg because he had just said the “regulatory framework” prevented his providing a scientific rationale for the company's new decision.
And it was quite an about-face. In October, when the Sweden-based company withdrew the drug, it was because the drug's confirmatory study had shown Pepaxto may increase the risk of death. Friday, the company announced that it had reversed that decision—and its hopes for a full FDA approval are still alive.
“We fully acknowledge that the action that was announced Friday night might seem confusing to some,” Lindberg said Monday. “We have gathered a lot of new information as part of our European application process, so this decision was not made lightly. It’s not an attempt to game the system. We are simply following the science.”
Oncopeptides' application for Pepaxto approval in Europe remains on schedule for a decision in the second quarter of this year, Lindberg said.
The move to sideline the drug in the U.S. came six days before an FDA advisory committee was set to discuss whether a confirmatory trial had shown the drug increased the risk of death. Oncopeptides explained at the time that its decision came after interactions with the FDA.
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The company said then that it would shut down its business units in the United States and Europe and return to its R&D-focused roots. Three weeks later, the company announced another change as former CEO Lindberg was reinstalled by its board to replace Marty Duvall.
On Monday, Lindberg acknowledged the mixed messages the company was sending.
“As a public company and as a pharmaceutical company, we are part of two regulatory frameworks in parallel. As a public entity we are (subject to) regulations which mean that material information needs to be provided to the market immediately,” Lindberg said. “On the other hand, we are also part of the regulatory framework dictating the rules for companies … and leads to the fact that we cannot in detail disclose scientific information that led to this decision today.”
Chief medical officer Klaas Bakker added that questions raised by the European Medicines Agency during its review of Pepaxto had led the company to a new conclusion on the drug.
“Sometimes you encounter things that you were not even looking for but that is material for the interpretation of the drug,” Bakker said. “It was only very recently that we arrived at this conclusion, that the scientific interpretation of the study is different with this new analysis.”
Bakker added that it would be “very speculative” to discuss a new timeline for its application with the FDA.
Five months after the FDA endorsed Pepaxto under its accelerated program, it alerted patients and doctors to the drug’s potential safety issue and suspended patient enrollment in the agency-required confirmatory trial. In the phase 3 OCEAN study, 10% more patients treated with Pepaxto and the steroid dexamethasone died compared with those who were given Bristol Myers Squibb’s Pomalyst and dexamethasone.
The snafu was seen as a blow to the FDA’s accelerated approval program, which is designed to get novel drugs to patients faster. The program came under heavy scrutiny with the controversial approval of Biogen’s Alzheimer’s disease treatment Aduhelm.
On Monday, Lindberg acknowledged the unusual circumstances of the company’s action and the difficult position it presents for investors.
“This is not done in spite or for any bad reasons,” he said. “We are just following the rules here.”