An Endocrine News roundup of the week’s pharmaceutical news, breakthroughs, and general information. *
Olfactive Biosolutions Granted Patent to Repurpose Food Molecules for Treatment of Hypertension
On October 15, Olfactive Biosolutions, the leader in adapting food molecules to treat chronic diseases, was recently granted U.S. patent 12,102,611, Compositions And Methods For Treating Hypertension By Modulating Endocrine Activity, for its breakthrough food ingredient formulation that reduces high blood pressure.
Protenx™ Blood Pressure can reduce blood pressure without the side effects and drawbacks of current pharmaceutical solutions, using food molecules from the FDA-approved GRAS (Generally Recognized As Safe) list.
The Protenx™ formulation will be available as a supplement sold direct to consumers in Q2 of next year. Also, food and beverage companies will add Protenx Blood Pressure to their consumer products, bringing the potential of achieving various benefits consistent with current prescription-only pharmaceuticals, without side effects and without requiring any changes in consumer behavior or routines.
Hypertension is one of the most widespread chronic diseases in the U.S. and is estimated by the Centers for Disease Control and Prevention (CDC) to cost the United States more than $131 billion a year in healthcare costs. The CDC also estimates that at least 47% of U.S. adults (approximately 121.5 million people) have high blood pressure, which significantly increases the risk of developing numerous life-threatening diseases and disorders.
Although pharmaceuticals and therapies prescribed for high blood pressure have generally been effective in treating essential hypertension, there are several disadvantages associated with them, including adverse reactions when prescribed with other medications. Additionally, some subjects have hypertension that is broadly resistant to pharmaceuticals, commonly referred to as resistant hypertension. Resistant hypertension often requires more aggressive treatment measures and can be very difficult to treat with pharmaceutically active agents alone. Protenx Blood Pressure offers an effective alternative for these patients.
First Patient Enrolled in ReCET Study
On October 15, Endogenex, Inc., announced the the enrollment of the first patient in the ReCET Clinical Study. This multicenter, prospective, randomized, double-blinded, sham-controlled trial is designed to assess the safety and effectiveness of the ReCET™ System in adult patients whose type 2 diabetes is inadequately controlled with non-insulin, glucose-lowering medications.
Endogenex is a clinical-stage medical device company focused on improving outcomes for patients with type 2 diabetes.
“This milestone is a major step forward in our mission to transform type 2 diabetes treatment by addressing the underlying causes impacting millions of patients living with type 2 diabetes,” stated Stacey Pugh, CEO of Endogenex. “We are thrilled to have completed the first enrollment and look forward to continued collaboration with our ReCET Clinical Study sites in advancing study enrollment.”
The first patient was successfully enrolled at Orlando Health, Orlando, FL, under the leadership of Andre Teixeira, Medical Director of the Orlando Health Weight Loss and Bariatric Surgery Institute. “We are excited to be part of this landmark study,” Teixeira said. “Clinical evidence continues to evolve, supporting the central role of the duodenum in regulating glucose metabolism. Therapeutic interventions targeting the upper gastrointestinal tract are demonstrating improved outcomes for patients with type 2 diabetes. The ability to reset pathologic signaling in the duodenum using the ReCET Technology, delivered through an endoscopic procedure, holds great promise for transforming care for type 2 diabetes patients.”
“We are proud to have reached this important milestone in the ReCET pivotal study,” stated Richard Pratley, medical director of the Advent Health Diabetes Institute in Orlando, Fla., and co-principal investigator for the ReCET Study. “The ReCET Study offers new hope for advancing treatment options and the potential to provide patients a better opportunity to manage their diabetes more effectively.”
The ReCET Study is a pivotal trial approved by the U.S. Food and Drug Administration (FDA) under the Investigational Device Exemption (IDE). The study aims to enroll up to 350 patients at approximately 40 sites in the United States and Australia.
About the ReCET™ Procedure
ReCET is a novel, endoscopic outpatient procedure that targets the cellular pathology of the duodenum. This pathology may contribute to the development and progression of type 2 diabetes.
The ReCET System aims to initiate the body’s natural regenerative process by applying highly controlled, non-thermal pulsed electric fields to the mucosa and sub-mucosa duodenal tissue. This approach may help restore proper cellular signaling from the duodenum and improve metabolic function, including better control of blood glucose levels.
The ReCET System has been evaluated in feasibility clinical studies, such as REGENT-1 US, REGENT-1 Australia, and EMINENT in the Netherlands. These studies assessed the safety and efficacy of the treatment in adults with type 2 diabetes whose blood glucose levels were inadequately controlled despite using insulin and non-insulin medications. Preliminary outcomes from these studies have been presented at medical conferences globally.
Positive Topline Results from Phase 2 Trial to Treat Wolfram Syndrome
On October 18, Amylyx Pharmaceuticals, Inc. announced positive topline data from the Phase 2 open-label HELIOS clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in 12 adults living with Wolfram syndrome.
Wolfram syndrome is a rare, progressive, monogenic disease impacting approximately 3,000 people in the U.S. HELIOS showed improvement in pancreatic function, as measured by C-peptide response after 24 weeks of treatment with AMX0035, the study’s primary efficacy endpoint, in contrast to the expected decrease in pancreatic function with disease progression. Similar overall improvements or stabilization were observed across all secondary endpoints, including hemoglobin A1c (HbA1c), time in target glucose range assessed by continuous glucose monitoring, and visual acuity. Patient- and physician-reported global impressions of change showed disease stability or improvement in all participants, meeting prespecified responder criteria.
In addition, longer-term data for all participants who completed Week 36 (n=10) and Week 48 (n=6) assessments showed sustained improvement over time. Data from HELIOS are being presented today at the International Society for Pediatric and Adolescent Diabetes (ISPAD) 50th Annual Congress and during a webcast held by the company.
“The topline results of HELIOS indicate that AMX0035 has the potential to favorably change the trajectory of Wolfram syndrome, a progressive disease with no approved treatment options. These results build on the interim data presented in April of this year and show an improvement on multiple measures of pancreatic beta cell function, glycemic control, and vision,” said Fumihiko Urano, MD, PhD, principal investigator of the Phase 2 HELIOS clinical trial in Wolfram syndrome and the Samuel E. Schechter Professor of Medicine in the Division of Endocrinology, Metabolism & Lipid Research at Washington University School of Medicine in St. Louis. “In addition, the participants who reached their Week 36 or Week 48 assessments demonstrated sustained improvement over baseline in C-peptide and HbA1c, which are objective laboratory measures of pancreatic function and glycemic control. These data are encouraging since Wolfram syndrome is a progressive disease.”
The analysis performed includes Week 24 data for all 12 participants and data for all participants who completed their Week 36 (n=10) and Week 48 (n=6) assessments as of the data cutoff. The primary efficacy endpoint of the trial measures change from baseline in C-peptide, an established, objective laboratory measure of pancreatic beta cell function and a surrogate marker of glycemic control, assessed using a mixed meal tolerance test (MMTT) at Week 24. Secondary and exploratory outcomes include the assessment of other diabetic measures and other domains affected by the disease.
“Wolfram syndrome is a progressive disease that is expected to consistently worsen over time, despite best supportive care, because of the underlying endoplasmic reticulum stress and mitochondrial dysfunction that occurs due to mutations in the WSF1 gene.” – Camille L. Bedrosian, MD, chief medical officer, Amylyx
HELIOS showed improvements in its primary endpoint of C-peptide response with a change from baseline to Week 24 at 120 minutes of +3.8 minutes*ng/mL (min*ng/mL) [standard error (SE): 19.3] in the Intent to Treat group (N=12) and +20.2 min*ng/mL [SE: 11.2] in the Per Protocol group (N=11). In addition, as outlined in the table below, participants receiving AMX0035 had improved glycemic control, as measured by markers of glucose metabolism; improved visual acuity in some participants, as measured by the Snellen chart; and improvement or stabilization of the disease, as measured by the Clinician Reported Global Impression of Change (CGIC) and Patient Reported Global Impression of Change (PGIC).
The safety profile of AMX0035 in HELIOS was consistent with prior safety data. AMX0035 was generally well-tolerated. All adverse events (AEs) were mild or moderate, and there were no serious AEs related to AMX0035 treatment. The most common AE was diarrhea.
“These outcomes indicate that treatment with AMX0035 may result in meaningful improvements across multiple measures of disease progression,” said Endocrine Society member Camille L. Bedrosian, MD, chief medical officer of Amylyx. “Wolfram syndrome is a progressive disease that is expected to consistently worsen over time, despite best supportive care, because of the underlying endoplasmic reticulum stress and mitochondrial dysfunction that occurs due to mutations in the WSF1 gene. AMX0035 is believed to target both of these critical pathways. In addition, we are encouraged by the sustained improvement observed in all participants who completed Week 36 or Week 48 assessments, and we thank the Wolfram syndrome community for their continued collaboration and support in researching the potential of AMX0035. We continue to engage with stakeholders and plan to meet with the FDA to inform a Phase 3 program.”
The FDA and the European Commission granted Orphan Drug Designation to AMX0035 for the treatment of Wolfram syndrome in November 2020 and August 2024, respectively.
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