What's Happening?
Harmony Biosciences has decided to pause its Phase II study of the cannabidiol candidate ZYN002 following a disappointing outcome in a Phase III trial for fragile X syndrome. The gel, which delivers synthetic cannabidiol through the skin, was intended
to address diseases with dysregulated endocannabinoid signaling. However, the Phase III RECONNECT study did not meet its primary efficacy endpoint, which was improvement in social avoidance, due to a higher than expected placebo response rate. As a result, Harmony is conducting a full review of the RECONNECT study and has suspended the ZYN002 study in 22q11.2 deletion syndrome, also known as DiGeorge syndrome. The company has not specified when it will release its analysis or resume the study.
Why It's Important?
The suspension of the ZYN002 study highlights the challenges faced in developing effective treatments for genetic disorders like fragile X syndrome and 22q11.2 deletion syndrome. These conditions affect a significant number of individuals in the U.S., with 22q affecting approximately 1 in 4,000 people. The failure to meet the primary endpoint in the RECONNECT study underscores the complexities of clinical trials, particularly in conditions with variable symptoms and responses. This development may impact stakeholders, including patients and healthcare providers, who are seeking new therapeutic options. Additionally, it reflects the broader challenges in the pharmaceutical industry of balancing innovation with the realities of clinical trial outcomes.
What's Next?
Harmony Biosciences is expected to complete its review of the RECONNECT study to understand the factors contributing to the trial's failure. The company may adjust its research and development strategies based on these findings. Meanwhile, Harmony's financial performance remains strong, with Q3 earnings showing a 29% increase year-on-year, driven by sales of Wakix, a drug for narcolepsy. Analysts have noted the potential for continued strong performance of Wakix, despite some anticipated challenges in sales growth. The outcome of the ZYN002 review could influence future research directions and investment decisions within the company.
