Harmony Biosciences has paused a mid-stage trial of ZYN002 in 22q11.2 deletion syndrome after the THC-free cannabinoid drug failed to significantly improve social avoidance in a late-stage study in fragile X Syndrome.
Harmony Biosciences paused a Phase II study of its cannabidiol candidate ZYN002 after a disappointing Phase III readout in fragile X syndrome.
ZYN002 is a synthetic cannabidiol formulated as a gel, delivering the drug substance through the skin. That, in turn, helps address diseases marked by dysregulated endocannabinoid signaling, such as fragile X syndrome.
Harmony, however, announced in September that the Phase III RECONNECT study in this indication had failed to meet its primary efficacy endpoint of improvement in social avoidance, blaming the miss on a “higher than expected placebo response rate.”
In its third-quarter business report on Tuesday, the Pennsylvania biotech said it is currently conducting a “full review” of RECONNECT and in the meantime will suspend the ZYN002 study in 22q11.2 deletion syndrome, also called DiGeorge syndrome or 22q. The company did not indicate when it plans to share its analysis of RECONNECT or lift the pause on 22q.
Affecting around 1 patient per 4,000 people in the U.S., 22q is a genetic disease caused by the random deletion of a small portion of chromosome 22. Symptoms of 22q are varied and can affect different organ systems, including the heart, immune system and brain. Some manifestations may be mild but can also be severe, such as developmental delays, vision and hearing loss and congenital heart disease. In some cases, 22q can be life-threatening.
Aside from the ZYN002 update, Harmony on Tuesday also reported Q3 earnings of $239.5 million, a 29% year-on-year increase. Wakix, indicated for excessive daytime sleepiness in narcolepsy, accounted for the entirety of Harmony’s sales, comfortably beating the $220 million analyst consensus.
In a note to investors on Tuesday, Jefferies analysts called Wakix’s Q3 sales “strong,” driven by “a record number of new patient adds.” Harmony reported that it had started approximately 500 patients on Wakix in Q3, resulting in a total of around 8,100 patients taking the drug. This also exceeds the consensus expectations of around 8,000 patients, according to Jefferies.
Jefferies pointed out, however, that despite raising its full-year outlook, Harmony’s Wakix guidance suggests the biotech is anticipating a 4% quarter-on-quarter decline in sales of the drug. Some challenges facing continued Wakix uptake include uncertainties in wholesaler orders, the analysts added, nevertheless noting that they expect the drug to sustain its strong performance.
“The updated guide looks overly conservative to us,” Jefferies said. “We expect Wakix to beat the high-end of the FY25 range.”
