High placebo responses common in tests of neuropsychiatric drugs make any clinical trial in major depressive disorder risky. Intra-Cellular Therapies now has data showing its drug met key goals of a pivotal clinical trial, beating a placebo in this indication. The results build the case for expanding the product’s label, which could push its sales into blockbuster territory.
According to the preliminary results reported Tuesday, the drug, lumateperone, led to statistically significant and clinically meaningful reductions in scores measured on a widely used rating scale for assessing depression episodes, which was the main clinical trial goal. The drug also met a key secondary goal demonstrating statistically significant and clinically meaningful reduction in score according to a different scale in which a clinician assesses illness severity and response to treatment.
Shares of Intra-Cellular topped $81 in midday trading Tuesday, up more than 25% from Monday’s closing price.
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Lumateperone is a small molecule that binds to receptors in the brain to modulate levels of brain chemicals that affect mood. The once-daily pill was approved in 2019 for the treatment of schizophrenia in adults. Two years later, the drug’s label was expanded to include the treatment of bipolar depression. For both indications, lumateperone is marketed under the brand name Caplyta.
The Phase 3 test in major depressive disorder, or MDD, studied lumateperone as an adjunct to antidepressant therapy. A total of 485 study participants were randomly assigned to receive the study drug or a placebo for six weeks. Intra-Cellular said statistically significant efficacy was observed as early as week 1, which is important because most depression drugs can take weeks to start showing an effect.
In addition to meeting the main and secondary goals at week 6, the drug showed a safety and tolerability profile consistent with its use in schizophrenia and bipolar depression. Dry mouth, fatigue, and tremors were the most common adverse events reported in the major depressive disorder study. Intra-Cellular said these side effects were mostly mild to moderate and resolved in a short period of time.
Caplyta is Intra-Cellular’s only FDA-approved drug. For 2023, the company reported $462.2 million in product revenue, an 85.5% increase from 2022 sales. Leerink Partners had already projected the drug would grow to become a blockbuster product, achieving peak sales of $1 billion to $1.5 billion.
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In a research note sent to investors Tuesday, Leerink analyst Marc Goodman said that given the data supporting Caplyta in its prior approved indications, the firm expected the drug would work in MDD. He noted that achieving statistical significance at week 1 makes it possible for Intra-Cellular to include “rapid onset” in an expanded label, though the drug still must succeed in one more Phase 3 study before the company can apply for an expanded FDA approval. A second Phase 3 test is expected to read out later this quarter. A third study is still enrolling patients, and data are not expected until next year. If the second study yields negative results, Goodman expects Intra-Cellular will seek expanded approval based on the drug’s totality of data, combining the successful MDD trial with the results from tests in prior indications. This strategy has some risk but it may also be enough for regulators given the strength of the MDD data reported Tuesday, Goodman said.
“This dataset supports our view that Caplyta will eventually be approved for adjunctive MDD and be a key player in the space with its tolerability profile and robust efficacy across a broad label, and we currently model an additional $1 billion of peak sales in MDD by 2033 (which we think is conservative and certainly has the potential to be higher),” Goodman wrote.
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