Hyperinsulinism

Rezolute Reports Validation of the Potential Use of RZ358 for Treatment of Non-Islet Cell Tumor Hypoglycemia (NICTH)

Retrieved on: 
Wednesday, March 6, 2024

Tumor hyperinsulinism (HI) may be caused by a variety of different tumor types, resulting in islet cell tumor hypoglycemia (ICTH) and NICTH.

Key Points: 
  • Tumor hyperinsulinism (HI) may be caused by a variety of different tumor types, resulting in islet cell tumor hypoglycemia (ICTH) and NICTH.
  • The Company previously reported on the successful use of RZ358 under its Expanded Access Program (EAP) to treat patients with insulin-producing pancreatic islet cell tumors (ICTs), or insulinomas, causing severe and uncontrolled hypoglycemia.
  • The therapeutic potential of RZ358 in this setting was anticipated given that ICTH is mediated by insulin and that RZ358 is known to work at the insulin receptor to decrease excess insulin binding and activity.
  • The inclusion of NICTH patients in a potential addressable market for RZ358 in tumor HI would more than double the population.

Rezolute Reports Second Quarter Fiscal 2024 Results and Provides Business Update

Retrieved on: 
Tuesday, February 13, 2024

REDWOOD CITY, Calif., Feb. 13, 2024 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, today announced its financial results for the second quarter of fiscal 2024 ended December 31, 2023, and provided an update on recent business developments and outlook.

Key Points: 
  • REDWOOD CITY, Calif., Feb. 13, 2024 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, today announced its financial results for the second quarter of fiscal 2024 ended December 31, 2023, and provided an update on recent business developments and outlook.
  • Rezolute initiated sunRIZE, a global, pivotal, Phase 3 clinical study in participants with cHI, in Europe and other geographies outside of the U.S.
  • Innovation and Licensing Application Passport (ILAP) designation awarded to RZ358 for the treatment of cHI by the U.K.
  • Topline results expected in the second quarter of 2024.

Rezolute Reports First Quarter Fiscal 2024 Results

Retrieved on: 
Monday, November 13, 2023

REDWOOD CITY, Calif., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, today announced its financial results for the first quarter of fiscal 2024 ended September 30, 2023.

Key Points: 
  • REDWOOD CITY, Calif., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, today announced its financial results for the first quarter of fiscal 2024 ended September 30, 2023.
  • “We are on track to commence our Phase 3 study for RZ358 to treat congenital hyperinsulinism prior to year-end and are delighted to have recently obtained PRIME eligibility from the European Medicines Agency for this indication,” said Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute.
  • “We also anticipate completing enrollment this quarter for our ongoing Phase 2 study of RZ402 for the treatment of diabetic macular edema and plan to provide an update on the study prior to year end.”
    Plan to initiate sunRIZE, a pivotal Phase 3 clinical study in patients with cHI, in Europe and other geographies outside the US in the fourth quarter 2023
    Continuing the administration of RZ358 in the US with FDA approval under a compassionate use program to treat patients with tumor associated hyperinsulinism, including for a patient with refractory hypoglycemia due to metastatic insulinoma who has remained on RZ358 for nearly a year
    Multi-center, randomized, double-masked, placebo-controlled, parallel-arm study ongoing to evaluate the safety, efficacy, and pharmacokinetics of RZ402 administered as a monotherapy over a 12-week treatment period in participants with DME who are naïve to or have received limited anti-VEGF injections
    Cash, cash equivalents and investments in marketable debt securities totaled $106.9 million as of September 30, 2023, compared to $118.4 million as of June 30, 2023
    Research and development expenses were $12.2 million for the first quarter of fiscal 2024, compared to $7.7 million for the same period in fiscal 2023, with the increase primarily attributable to increased expenditures in clinical trial activities, manufacturing costs and higher personnel-related expenses which included employee compensation and stock-based compensation
    General and administrative expenses were $3.7 million for the first quarter of fiscal 2024, compared to $2.5 million for the same period in fiscal 2023, with the increase primarily attributable to higher personnel-related expenses, including employee compensation and stock-based compensation
    Net loss was $14.5 million for the first quarter of fiscal 2024, compared to $9.8 million for the same period in fiscal 2023

Rezolute Announces Further Evidence of RZ358’s Efficacy in Tumor-Mediated Hyperinsulinism

Retrieved on: 
Wednesday, October 11, 2023

The Company previously reported on the successful use of RZ358 for a patient with refractory hypoglycemia due to metastatic insulinoma, which has since been published in a recent edition of the New England Journal of Medicine (389;8).

Key Points: 
  • The Company previously reported on the successful use of RZ358 for a patient with refractory hypoglycemia due to metastatic insulinoma, which has since been published in a recent edition of the New England Journal of Medicine (389;8).
  • Notably, the individual with metastatic insulinoma has remained on RZ358 for nearly a year.
  • “We believe that this provides further validation of our long-held view of hyperinsulinism as a group of related conditions, and RZ358 as a potential universal treatment for any of the various causes of hyperinsulinism, whether congenital, induced by gastric bypass, or mediated by tumors.
  • We are in the process of evaluating next steps to develop RZ358 in these additional indications.”

Crinetics’ Once-Daily Oral Paltusotine Achieved the Primary and All Secondary Endpoints in the Phase 3 PATHFNDR-1 Study Evaluating Treatment of Patients with Acromegaly

Retrieved on: 
Sunday, September 10, 2023

The frequency of participants with at least one treatment emergent adverse event (TEAE) was comparable in the paltusotine (PAL) treatment arm vs placebo (PBO) arm (80% vs. 100% respectively).

Key Points: 
  • The frequency of participants with at least one treatment emergent adverse event (TEAE) was comparable in the paltusotine (PAL) treatment arm vs placebo (PBO) arm (80% vs. 100% respectively).
  • The frequency of adverse events considered related to acromegaly was notably lower in paltusotine treated participants compared to placebo treated participants (30% vs. 86% respectively).
  • “We designed paltusotine to be the preferred therapeutic option for people living with acromegaly.
  • The Company is also conducting an open-label Phase 2 study to evaluate paltusotine in patients with carcinoid syndrome and intends to report preliminary results later this year.

Twist Bioscience Publishes Preclinical Data in Diabetes Validating GLP-1R Antagonist Antibody as Potential Treatment for Congenital Hyperinsulinism

Retrieved on: 
Monday, June 26, 2023

It is caused by excessive pancreatic beta cell insulin secretion, resulting in hypoglycemia that can cause brain damage or death without treatment.

Key Points: 
  • It is caused by excessive pancreatic beta cell insulin secretion, resulting in hypoglycemia that can cause brain damage or death without treatment.
  • Twist researchers had previously identified a highly potent and specific GLP-1R antagonist antibody, TB-001-003, from synthetic antibody libraries designed to target G protein-coupled receptors.
  • Subsequently, this lead was optimized further for greater activity against GLP-1R using the Twist Antibody Optimization (TAO) library platform.
  • One antagonist, TB-222-023, was identified and shown to be more potent than exendin-(9-39), a clinical stage GLP-1R antagonist peptide.

Children's Hospital of Philadelphia's Congenital Hyperinsulinism Center Celebrates 600 Surgeries

Retrieved on: 
Wednesday, December 21, 2022

PHILADELPHIA, Dec. 21, 2022 /PRNewswire/ -- The Congenital Hyperinsulinism (HI) Center at Children's Hospital of Philadelphia (CHOP), the nation's largest and most active center of its kind, is celebrating the completion of 600 pancreatectomies, a surgical procedure that involves removing all or part of the pancreas. The procedure is used to treat some forms of congenital HI, a life-threatening genetic disorder in which the insulin cells of the pancreas, called beta cells, secrete too much insulin, leading to low blood sugar (hypoglycemia).

Key Points: 
  • PHILADELPHIA, Dec. 21, 2022 /PRNewswire/ -- The Congenital Hyperinsulinism (HI) Center at Children's Hospital of Philadelphia (CHOP), the nation's largest and most active center of its kind, is celebrating the completion of 600 pancreatectomies, a surgical procedure that involves removing all or part of the pancreas.
  • Established in 1998, CHOP's HI Center treats children with congenital HI from all over the world.
  • The multidisciplinary team in CHOP's HI Center works together to provide specialized, patient-centered care for children with congenital HI and their families.
  • About Children's Hospital of Philadelphia: A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital.

In Partnership with Global Genes, Congenital Hyperinsulinism International Hosts Inaugural Family Meet-Up

Retrieved on: 
Thursday, October 20, 2022

Global Genes , a leading patient advocacy organization, and Congenital Hyperinsulinism International (CHI), a foremost nonprofit dedicated to improving the lives of children and adults living with congenital hyperinsulinism (HI), today announced the inaugural CHI Family Meet-Up .

Key Points: 
  • Global Genes , a leading patient advocacy organization, and Congenital Hyperinsulinism International (CHI), a foremost nonprofit dedicated to improving the lives of children and adults living with congenital hyperinsulinism (HI), today announced the inaugural CHI Family Meet-Up .
  • By bringing families and friends in the HI community together we provide the opportunity to learn and connect with those affected by this rare disorder, said Julie Raskin, Executive Director of Congenital Hyperinsulinism International.
  • Global Genes is a 501(c)(3) leading international non-profit organization that is establishing a globally connected community committed to overcoming the challenges of rare disease.
  • Congenital Hyperinsulinism International (CHI) is a nonprofit 501(c)3 corporation dedicated to improving the lives of babies, children, and adults affected by congenital hyperinsulinism (HI).

Innovation Passport Granted for CRN04777 for the Treatment of Congenital Hyperinsulinism

Retrieved on: 
Thursday, October 6, 2022

(Nasdaq: CRNX) today announced today that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted CRN04777 an Innovation Passport for the treatment of congenital hyperinsulinism (HI), which enables Crinetics to access the Innovative Licensing and Access Pathway (ILAP).

Key Points: 
  • (Nasdaq: CRNX) today announced today that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted CRN04777 an Innovation Passport for the treatment of congenital hyperinsulinism (HI), which enables Crinetics to access the Innovative Licensing and Access Pathway (ILAP).
  • We believe the Innovation Passport provides important external validation for the CRN04777 program and shows that the MHRA recognizes the struggle congenital HI patients and caregivers face each day.
  • Oral administration of CRN04777 has been shown to potently inhibit insulin secretion and normalize glucose levels in preclinical models of hyperinsulinism.
  • The U.S. Food and Drug Administration granted rare pediatric disease designation for CRN04777 for the treatment of congenital hyperinsulinism.

Crinetics Pharmaceuticals Reports Positive Top-line Results Including Strong Adrenal Suppression from CRN04894 Phase 1 Study Multiple-Ascending Dose Cohorts

Retrieved on: 
Wednesday, May 25, 2022

SAN DIEGO, May 25, 2022 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, today announced positive results from the multiple-ascending dose (MAD) portion of a first-in-human Phase 1 clinical study of CRN04894, the company's first-in-class, investigational, oral, nonpeptide adrenocorticotropic hormone (ACTH) antagonist that is being developed for the treatment of Cushing’s disease, congenital adrenal hyperplasia (CAH) and other conditions of excess ACTH. Following administration of CRN04894, results showed serum cortisol below normal levels and a marked reduction in 24-hour urine free cortisol excretion in the presence of sustained, disease-like ACTH concentrations.

Key Points: 
  • Safety and pharmacokinetic data were consistent with expectations from the single-ascending dose cohorts in the Phase 1 study.
  • Crinetics plans to present additional details of safety, efficacy, and biomarker results from the CRN04894 Phase 1 study at an endocrinology-focused medical meeting in 2022.
  • Crinetics will hold a conference call and live audio webcast today, May 25, 2022, at 8:00 a.m. Eastern Time to discuss results from the MAD cohorts of the Phase 1 study of CRN04894.
  • Participants were divided into multiple cohorts in the single ascending dose (n=39) and multiple ascending dose (n=49) portions of the study.