Biohaven’s bipolar disorder candidate has failed to improve mania symptoms in a phase 2/3 trial.
The registrational study assessed BHV-7000, a drug designed to selectively activate Kv7.2/Kv7.3, an ion channel involved in neuronal signaling and regulation. An estimated 250 participants with bipolar I disorder were enrolled, with participants randomized to receive 75 mg of the investigational drug or comparator once a day.
Top-line results from the three-week trial reveal no statistically significant difference between comparator and treatment, as measured by change in Young Mania Rating Scale total score, according to the company’s quarterly earnings release shared March 3. The scale consists of 11 items used to evaluate symptoms of mania.
Specific data were not shared. Biohaven said it is conducting additional analyses and expects to present the full data set at an upcoming scientific meeting, according to the release.
BHV-7000 was well tolerated, with no serious treatment-emergent adverse events reported. Most adverse events were mostly mild and resolved spontaneously, the company said.
The investigational treatment has "shown an impressive tolerability profile," William Blair analysts wrote in a March 4 note, citing the lack of CNS adverse events, such as dizziness, that are often tied to other antiseizure and psychiatric meds.
Biohaven designed BHV-7000 using its Kv7 platform and is considering the candidate as a potential treatment for pain disorders, according to the company’s website.
The asset is currently being evaluated in three other phase 2/3 studies across major depressive disorder (MDD), focal epilepsy and generalized epilepsy. The MDD findings are slated for release in the second half of this year, while focal epilepsy results are expected in the first half of next year.
The upcoming MDD data will be "key for determining the overall competitive profile of the drug in an evolving Kv7-potentiator space," the William Blair analysts wrote.
In a separate release, also posted March 3, Biohaven detailed early-stage clinical findings for its subcutaneous small molecule asset dubbed BHV-1300. The ongoing phase 1 study found that 1000 mg of BHV-1300 per week reduced total IgG by up to 84% for patients with Graves’ disease.
The reduction slightly exceeds those seen in subcutaneously administered FcRN-targeted therapies, such as 600 mg weekly of Immunovant's IMVT-1402, which showed a mean reduction of 74% with weekly dosing in a phase 1 trial of healthy adults.
Biohaven's four-week study found the asset to be safe and well tolerated, according to the company. A phase 2 trial will be launched in the same indication in mid-2025, with additional follow-on studies in other autoimmune diseases also expected.
The protein degrader is designed to selectively target IgG1, 2 and 4 while sparing IgG3.
“Biohaven's unique extracellular degrader technology leverages the body's natural hepatic clearance mechanism to remove targeted antibodies contributing to disease and promises to usher in a new era of tunable, selective and self-administered immune therapy,” Chief Translational Officer Tova Gardin, M.D., said in the release.
Biohaven’s stock dropped 14% Monday, sinking from $36.95 at market open to $32 by the end of the day.
Editor's note: This story was updated at 9:30 a.m. ET on March 4 to include analysis from William Blair.