Updated from 11:30 a.m. EDT
After the pain, comes the lesson.
(MATX) shareholders are smarting Friday after U.S. drug reviewers all but slammed the door on any chances that the company's experimental cancer treatment, IntraDose, would be approved. But shareholders should limp away wiser from the experience if they learn one important lesson: The U.S. Food and Drug Administration is not a rubber stamp agency.
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Simply put, the FDA doesn't put much weight behind what Matrix, or any other biotech firm, concludes about its own drug test results, even when those results appear to be strongly supported by evidence. Instead, the agency's reviewers take great pleasure in doing their own analysis, and the conclusions they reach are often starkly different.
This was abundantly clear after reading the negative FDA staff review of IntraDose, posted to the agency's Web site Friday morning in advance of an independent advisory panel meeting Monday to discuss and vote whether to approve IntraDose. Trading in Matrix was halted Friday and Monday pending the panel vote.
"The reviewing medical officer recommends against marketing approval for IntraDose ... based on the finding of extremely limited efficacy and moderate to severe toxicity reproducibly demonstrated in two clinical trials specifically designed to evaluate safety and efficacy of IntraDose," the FDA review documents stated.
This conclusion couldn't be more different from those reported by Matrix. IntraDose is a tumor-shrinking gel that is injected directly into tumors of patients dying from head and neck cancer. The treatment does not cure these patients, but is designed to shrink the tumors enough to make the end of their lives more comfortable. And according to the company's test results, the treatment worked like a charm -- shrinking tumors in 34% of its patients in one of two trials. The supporting evidence of this tremendous efficacy result looked clean, leading many biotech mavens to predict IntraDose's approval.
Not so fast, said FDA regulators. They took apart Matrix's application, examined individual patient records from this trial and came to a very different conclusion: IntraDose was able to shrink tumors in only between 19% and 27% of patients, pending further discussion of tumor measurement. The FDA also found serious errors in the dosing levels for patients receiving IntraDose, which could have skewed results even more in the company's favor, according to the FDA review comments.
Since IntraDose is not a cancer cure, Matrix was asked to prove that its terminally ill patients benefited from the treatment. This subjective measure of "patient benefit" was always Matrix's Achilles heel because the company's stated results were borderline positive at best.
But again, FDA reviewers shredded the company for both the design of the patient benefit test and its results. Matrix was criticized for designing a test that didn't eliminate biased findings from clouding results. Once those results were re-examined, the FDA found that Matrix fell far from proving that IntraDose really helped patients in any meaningful way.
Furthermore, it seems clear from the FDA staff review that this "patient benefit" analysis was more important than tumor shrinkage in determining whether to approve IntraDose. Matrix officials led shareholders and analysts to believe the opposite. In effect, the FDA is telling Matrix that IntraDose does shrink tumors, but so what? It doesn't benefit patients at all.
So, there you have it. Matrix joins a long list of biotech companies with what looks like a strong drug application, but is actually quite weak once FDA staffers finish their dissection.
But let's just add another crazy element to the mix -- Friday's negative FDA staff recommendation for IntraDose is just that -- a recommendation. Officials from Matrix, as well as its outside investigators, will be able to argue and present their case for IntraDose approval on Monday to the Oncologic Drugs Advisory Committee of the FDA. The panel's recommendation is then passed on to the FDA, which makes the final decision on the treatment's fate. The FDA usually follows the recommendation of the panel.
So, there's still hope for Matrix, as bleak as things may seem. Last month,
received the same kind of sandblasting from the FDA staff for its experimental pulmonary arterial hypertension drug, Remodulin. But the FDA advisory panel convened to discuss the drug's prospects turned around and voted to recommend its approval. The final fate of Remodulin now rests with the FDA.
Trying to predict the outcome of an FDA drug review is a crapshoot at best. Friday, Matrix and its supporters threw snake eyes, but they get a second run at the table on Monday.