What happened
Shares of Denali Therapeutics (DNLI 4.38%) had sunk 11.2% as of 12:02 p.m. EDT on Monday. The decline came after the company presented interim results on Sunday from a phase 1/2 study evaluating experimental enzyme replacement therapy DNL310 in treating the rare neurodegenerative disorder Hunter syndrome.
So what
You'd probably expect that Denali's phase 1/2 interim results would be bad considering how the biotech stock fell. But the company touted the interim data as "positive." The reality is that there was both good and bad news with the latest results.
Image source: Getty Images.
The good news was that DNL310 was able to quickly reduce and normalize heparan sulfate in cerebrospinal fluid (CSF). This suggests that the blood-brain barrier crossing that Denali uses worked well. The company also reported results from two standardized assessment scales that indicated clinical improvement in patients who received DNL310.
The bad news was that levels of neurofilament light (Nf-L), which is a potential biomarker of neuronal damage, increased. Denali had hoped the Nf-L levels would decrease. However, the interim results were only from five patients after six months of treatment with DNL310. It's possible that is too early to see a significant impact on Nf-L.
Now what
Denali now plans to accelerate its efforts to begin a pivotal phase 2/3 clinical study evaluating DNL310. This study could kick off in the first half of 2022. The company also intends to begin enrollment in a third cohort in the phase 1/2 study "to further investigate clinical endpoints."
