Soleno Looking to Expand Potential Therapies That Work Like DCCR

Marisa Wexler MS avatar

by Marisa Wexler MS |

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Soleno Therapeutics has partnered with Vanderbilt University to develop therapies that work as ATP-dependent potassium channel activators for rare diseases, similar to the diazoxide choline controlled release (DCCR) tablets the company thinks might treat Prader-Willi syndrome (PWS).

ATP-dependent potassium (or KATP) channels are proteins present in multiple body tissues. As their name suggests, these proteins regulate the movement of potassium in and out of cells, and they use ATP — adenosine triphosphate, the “energy currency” of cells — to do so.

Activating these channels may be beneficial in treating various rare conditions. According to Soleno, one example is hyperinsulinemic hypoglycemia, or low blood sugar caused by abnormally high levels of the hormone insulin.

“Based on the role KATP channels play in controlling and regulating cellular functions, we believe therapeutics targeting this channel could have potential in treating multiple rare diseases,” Craig Lindsley, PhD, a professor at Vanderbilt and director of the its Warren Center for Neuroscience Drug Discovery (WCNDD), said in a press release.

Lindsley is leading the collaboration, along with Jerod Denton, PhD, also a Vanderbilt professor and director of Ion Channel Pharmacology at the WCNDD.

The initial goal of the collaboration is to develop tools using cells in dishes (in vitro) or animal models (in vivo) to spot and characterize KATP channel activators from new classes of chemistry. Soleno is expecting to advance candidates identified in this collaboration into future clinical testing.

DCCR, the company’s investigational therapy for PWS, is a KATP channel activator administered as a once-daily tablet.

Recent data from a Soleno-sponsored Phase 3 clinical trial, called DESTINY (NCT03440814), indicated that treatment with DCCR could ease hyperphagia (excessive hunger) and behavioral symptoms such as aggressiveness and anxiety. Top-line results of this trial, however, are believe to have substantially affected by the COVID-19 pandemic.

“We look forward to working closely with Soleno, whose ongoing development of DCCR for PWS has yielded encouraging clinical results to date, to further our research in this important and widely applicable area,” Lindsley said.

“Having discovered the only new KATP channel activator chemistries in the last 20 years, the highly experienced team of Drs. Lindsley and Denton are ideally suited to work with us to identify and characterize novel drug candidates that could serve as the underpinnings for our next generation of products,” said Anish Bhatnagar, MD, Soleno’s CEO.

“We believe that combining their expertise with our experience around the therapeutic potential of KATP channel activators based on our Phase 3 program of DCCR in PWS will be an effective and efficient path to new therapeutic products,” Bhatnagar added.