Soleno Soars on Positive Prader-Willi Syndrome Data
14 Jun 2022
First in Class
California’s Soleno Therapeutics, a clinical-stage biopharmaceutical company, announced promising results of its diazoxide choline extended-release (DCCR) tablets in patients with Prader-Willi syndrome (PWS).
Revealed during an oral presentation at the 2022 Endocrine Society Annual Meeting & Expo (ENDO), the data showed that one year of DCCR treatment led to significant improvements in patients’ lean body mass (p<0.0001) and the ratio of lean body mass to fat mass (p=0.0005). These positive changes were accompanied by a significant drop in fasting levels of insulin (p=0.0004) and leptin (p<0.0001).
DCCR likewise induced significant improvements in insulin sensitivity (p=0.0033), partly due to the patients’ lower fat mass and better leptin resistance. Recipients also saw a significant increase in the cardioprotective marker adiponectin (p<0.0001). Of note, focusing the analysis on patients with obesity did not weaken DCCR beneficial effects.
“These important results show that the benefits of DCCR also extend to positive changes in objective metabolic parameters, in addition to prior data demonstrating improvements in hyperphagia and behavioral symptoms of PWS,” Dr. Eric Felner of Emory University School of Medicine said in a statement. “These results further emphasize the potential for DCCR to be a meaningful treatment that improves multiple key symptoms of PWS, which has significant implications for patients and families struggling to manage this devastating disease.”
The data presented at ENDO came from two trials: C601, a Phase III, randomized, double-blind, placebo-controlled study and C602, its ongoing open-label extension. A total of 82 PWS patients participated in the trials, of whom 40 had obesity. Patients were treated with DCCR tablets, which contain the crystalline salt of diazoxide and are administered once a day. Felner, who presented the latest results at ENDO, is a principal investigator of the C601/C602 trials.
DCCR also demonstrated an acceptable safety pro a separate poster presentation at ENDO, delivered by Dr. Michael Woloschak, vice president of clinical development at Soleno. Common adverse events, such as peripheral edema, hyperglycemia and hypertrichosis, were consistent with previous experiences involving DCCR.
PWS is a rare genetic disorder that affects around one in every 15,000 live births in the United States. Commonly, patients with PWS suffer from its hallmark symptom, hyperphagia, or being insatiably hungry. Hyperphagia causes patients to overeat, which, in turn, leads to uncontrollable weight gain. Often, PWS complications arise due to obesity.
Currently, there is no approved treatment for the appetite and metabolic aspects of PWS, a great unmet need that Soleno hopes to fulfill. Perhaps due to the disease’s complex nature, Soleno is entering a relatively unoccupied PWS space with its DCCR tablets.
In March, Tonix Pharmaceuticals’ investigational drug TNX-2900 was granted the orphan-drug designation by the United States Food and Drug Administration for the treatment of PWS. Unlike Soleno’s DCCR tablets, TNX-2900 comes in the form of a nasal spray to deliver a patent-protected formulation that can increase the specificity of the user’s oxytocin receptors.
A month earlier, clinical-stage biopharma company Aardvark Therapeutics announced that after encouraging results from a Phase I first-in-human trial, it was initiating enrollment for a Phase II study to test its investigational drug ARD-101 in PWS. A first-in-class small-molecule drug, ARD-101 is designed to target the extraoral bitter taste receptors and was shown to be effective in promoting weight loss in preclinical studies.
“With a majority of adults in the U.S. being either overweight or obese and no approved treatments for PWS, clearly there is a need to develop new effective treatments,” Dr. Tien Lee, M.D., chief executive officer of Aardvark, said in a statement. “ARD-101 represents such a candidate therapy that has demonstrated safety and tolerability in studies to date and offers the convenience of oral administration.”
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