While Harmony management has not disclosed future plans for ZYN002, Jefferies analysts expect the asset to be shelved.
Harmony Biosciences’ cannabidiol candidate ZYN002 missed its primary efficacy endpoint in a late-stage study of fragile X syndrome, sparking questions about the asset’s future.
In a brief news release on Wednesday, Harmony said the drug failed to significantly improve social avoidance in patients in the Phase III RECONNECT trial. The biotech pointed to a “higher than expected placebo response rate” to explain the miss, though it did not provide specific data in its press announcement. Harmony did not disclose its plans for ZYN002 moving forward.
Harmony management, however, did tell Jefferies that the company will suspend the initiation of another Phase III study for ZYN002 while it conducts an assessment of RECONNECT’s findings, according to a Wednesday note from the analyst group.
“In our view, the most likely outcome is that the asset will be shelved,” Jefferies wrote.
At market close on Wednesday, Harmony was trading at $26.76, down 16.5% from its closing price the previous day.
Formulated as a gel, ZYN002 is a synthetic cannabidiol that does not contain THC and is designed with enhanced permeation through the skin and into the circulatory system. Harmony came to own the drug when it bought Zynerba Pharmaceuticals in August 2023 for $60 million upfront and the promise of $140 million in potential milestones.
The FDA has previously granted the therapy orphan drug designation for fragile X syndrome, a rare, genetic disorder afflicting 1 in around 4,000 to 7,000 males and 1 in 8,000 to 11,000 females. The disease is caused by deficient levels of a protein called FMRP, which in turn dysregulates the endocannabinoid system, leading to neurobehavioral symptoms such as irritability, social avoidance and intellectual disabilities.
Aside from fragile X syndrome, ZYN002 is also being tested for 22q11.2 deletion syndrome, a genetic deficiency that can lead to severe developmental delays.
In February, Harmony was hit with a refusal to file letter from the FDA, which denied to even review the biotech’s application for its oral drug Wakix, being proposed for idiopathic hypersomnia. A refusal to file letter is issued to companies that file incomplete submissions.
