DSMB

Galectin Therapeutics Reports the Positive Outcome of the Fifth Data and Safety Monitoring Board Meeting for NAVIGATE Phase 2b/3 Study of Belapectin in Patients with Cirrhotic Portal Hypertension Caused by Metabolic Dysfunction-Associated SteatoHepatitis

Retrieved on: 
Tuesday, April 9, 2024
Data monitoring committee, Cirrhosis, Metabolism, Disease, Risk, Patient, Hope, MASH, Final Analysis, Therapy, GALT, Safety, DSMB, Interim, Plasma protein binding, Esophageal varices, Liver, Pharmaceutical industry

The objective of this fifth DSMB meeting was to further review the emerging tolerance and safety profiles of belapectin.

Key Points: 
  • The objective of this fifth DSMB meeting was to further review the emerging tolerance and safety profiles of belapectin.
  • Based on its deliberation, which included an unblinded review of the data collected thus far, the DSMB concluded that NAVIGATE can continue as designed, without modifications.
  • The Phase 2b/3 NAVIGATE study is a global, seamless, adaptive, randomized, placebo-controlled, double-blind trial.
  • Enrolled patients have been randomized to receive every two weeks a blinded infusion of belapectin at 2 mg/kg/LBM or 4 mg/kg/LBM or placebo.

Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410—a Modifier Gene Therapy—in Phase 1/2 ArMaDa Study for Geographic Atrophy

Retrieved on: 
Friday, April 5, 2024
Retina, Disease, Inflammation, Patient, Data, Cohort, Șaeș, Life, Clinical trial, Armada, MPH, Injection, Safety, DSMB, FACS, Vaccine, Lipid metabolism, Geographic atrophy, Oxidative stress, Pharmaceutical industry

Three subjects with geographic atrophy (GA) were dosed in the Phase 1/2 clinical trial to date.

Key Points: 
  • Three subjects with geographic atrophy (GA) were dosed in the Phase 1/2 clinical trial to date.
  • An additional three subjects will be dosed with the medium dose (Cohort 2) and three patients with the high dose (Cohort 3) of OCU410 in the dose-escalation phase.
  • “The DSMB has recommended moving forward to medium dose for dosing subjects with GA,” said Dr. Peter Chang, MD, FACS, DSMB Chair for the OCU410 clinical trial.
  • Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL)].

Verve Therapeutics Announces Updates on its PCSK9 Program

Retrieved on: 
Tuesday, April 2, 2024
LDLR, Cardiovascular disease, Gene editing, PCSK9, ALT, Patient, RNA, Health care, Health Canada, IND, N-Acetylgalactosamine, MHRA, Asialoglycoprotein receptor, Clinical trial, CTAS, Therapy, Bleeding, Safety, Moyamoya disease, DSMB, FDA, Liver, ASCVD, LNP, Medsafe, Hypercholesterolemia, Food, Pharmaceutical industry

BOSTON, April 02, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today announced updates from the Heart-1 Phase 1b clinical trial of VERVE-101 and clearance of its Clinical Trial Applications (CTAs) by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and Health Canada for VERVE-102, with the Heart-2 Phase 1b clinical trial expected to initiate in the second quarter of this year.

Key Points: 
  • Six participants have been dosed at 0.45 mg/kg of VERVE-101, with a total of 13 participants dosed in the study.
  • Verve is conducting an investigation into the laboratory abnormalities and based on those results, expects to work with regulatory authorities to define a path forward for VERVE-101.
  • Verve is now prioritizing the development of VERVE-102 and the initiation of the Heart-2 clinical trial.
  • VERVE-102 uses the same base editor and guide RNA for PCSK9 but a different lipid nanoparticle (LNP) delivery system than VERVE-101.

Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410ST—a Modifier Gene Therapy—in GARDian Study for Stargardt Disease

Retrieved on: 
Monday, April 1, 2024
Stargardt disease, Gardian, Patient, Cohort, Șaeș, Clinical trial, MPH, Safety, DSMB, FACS, Vaccine, FDA, Pharmaceutical industry

Three patients with Stargardt disease were dosed in the Phase 1/2 clinical trial to date.

Key Points: 
  • Three patients with Stargardt disease were dosed in the Phase 1/2 clinical trial to date.
  • An additional three patients will be dosed with the medium dose (Cohort 2) and three patients with the high dose (Cohort 3) of OCU410ST in the dose-escalation phase.
  • “The DSMB has recommended moving forward to medium dose for dosing subjects with Stargardt disease,” said Dr. Peter Chang, MD, FACS, DSMB Chair for the OCU410ST clinical trial.
  • Phase 1 is a multicenter, open-label, dose ranging study consisting of three dose levels [low dose (3.75×1010 vg/mL), medium dose (7.5×1010 vg/mL), and high dose (2.25×1011 vg/mL)].

Galectin Therapeutics Reports 2023 Financial Results and Provides Business Update

Retrieved on: 
Friday, March 29, 2024
Research, Data monitoring committee, Cirrhosis, Disease, Metabolism, AASLD, Safety, Patient, Food and Drug Administration, Liver, Mortality, Liver transplantation, Hope, Hepatology, Fibrosis, Clinical trial, Annual report, Therapy, GALT, Pharmacokinetics, Data, DSMB, NASH, Food, Esophageal varices

NORCROSS, Ga., March 29, 2024 (GLOBE NEWSWIRE) -- Galectin Therapeutics, Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, today reported financial results and provided a business update for the year ended December 31, 2023.

Key Points: 
  • NORCROSS, Ga., March 29, 2024 (GLOBE NEWSWIRE) -- Galectin Therapeutics, Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, today reported financial results and provided a business update for the year ended December 31, 2023.
  • Joel Lewis, Chief Executive Officer and President of Galectin Therapeutics, said “We have been focused on advancing our Metabolic Dysfunction-Associated Steatohepatitis (MASH, formerly known as NASH) cirrhosis program.
  • Dr. Jamil’s extensive experience in advanced liver disease, specifically for late-stage cirrhotic patients, is a tremendous asset to our program.
  • These results are included in the Company's Annual Report on Form 10-K, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov .

Atea Pharmaceuticals Completes Patient Enrollment in Global Phase 3 SUNRISE-3 Trial Evaluating Oral Antiviral Bemnifosbuvir for COVID-19 in High-Risk Patients

Retrieved on: 
Wednesday, March 27, 2024
Infection, Risk, COVID-19, Patient, Fast Track, Viral, Data monitoring committee, Death, Doctor of Philosophy, Safety, DSMB, Immunodeficiency, Atea, Food, Hospital, Pharmaceutical industry

BOSTON, March 27, 2024 (GLOBE NEWSWIRE) -- Atea Pharmaceuticals, Inc. (Nasdaq: AVIR) (Atea), a clinical-stage biopharmaceutical company engaged in the discovery and development of oral antiviral therapeutics for serious viral diseases, today announced that the company has completed enrollment of the global Phase 3 SUNRISE-3 trial evaluating bemnifosbuvir, an oral nucleotide polymerase inhibitor, or placebo for the treatment of COVID-19. Over 2,200 patients were randomized into the supportive care monotherapy cohort and less than 80 patients were randomized into the combination cohort. The primary endpoint of the trial is all-cause hospitalization or death through Day 29 post-treatment in the bemnifosbuvir supportive care monotherapy cohort of high-risk patients. Secondary endpoints include other measurements of patient outcomes through Day 60 post-treatment.

Key Points: 
  • Over 2,200 patients were randomized into the supportive care monotherapy cohort and less than 80 patients were randomized into the combination cohort.
  • The primary endpoint of the trial is all-cause hospitalization or death through Day 29 post-treatment in the bemnifosbuvir supportive care monotherapy cohort of high-risk patients.
  • Secondary endpoints include other measurements of patient outcomes through Day 60 post-treatment.
  • “COVID-19 continues to be a threat, leaving the most vulnerable at risk for severe outcomes from infection.

Panbela Provides Business Update and Reports Q4 and FY 2024 Financial Results

Retrieved on: 
Tuesday, March 26, 2024
Research, Topotecan, PBLA, Neuroblastoma, Patient, Data monitoring committee, Acquisition, Celecoxib, Doctor of Philosophy, AACR, MSN, CPP, Pancreatic cancer, Therapy, DSMB, Poster, DFMO, Cyclophosphamide, Pharmaceutical industry

MINNEAPOLIS, March 26, 2024 (GLOBE NEWSWIRE) -- Panbela Therapeutics, Inc. (NASDAQ:PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, today provides a business update and reports financial results for the quarter and full year ended December 31, 2023.

Key Points: 
  • MINNEAPOLIS, March 26, 2024 (GLOBE NEWSWIRE) -- Panbela Therapeutics, Inc. (NASDAQ:PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, today provides a business update and reports financial results for the quarter and full year ended December 31, 2023.
  • “Looking ahead, Panbela remains unwavering in its commitment to patients and in its pursuit of maximizing value for stockholders.
  • Notes payable, plus accrued interest, on the balance sheet, the result of the acquisition of CPP, totaled approximately $5.4 million.
  • Gross proceeds from the raise, which closed on January 31, 2024, were approximately $9 million.

AACR 2024: PDC*line Pharma Presents Interim Clinical Results From Last Cohort of Patients in Phase I/II Trial with PDC*lung01 Cancer Vaccine

Retrieved on: 
Monday, April 8, 2024
Stem Cells, Biotechnology, Health, Pharmaceutical, Biometrics, Oncology, Other Science, Research, Genetics, Science, Clinical Trials, KU Leuven, Mutation, Database, Survival, Best, DCR, Patient, Data, Aes, Șaeș, Progression-free survival, AACR, Kaplan–Meier estimator, Clinical trial, Cancer, Injection, ESMO, Safety, Symantec Endpoint Protection, DSMB, Poster, PFS, Vaccine, PDC

PDC*lung01 is the company’s off-the shelf therapeutic cancer vaccine candidate for Non-Small Cell Lung Cancer (NSCLC).

Key Points: 
  • PDC*lung01 is the company’s off-the shelf therapeutic cancer vaccine candidate for Non-Small Cell Lung Cancer (NSCLC).
  • The phase I/II trial (PDC-LUNG-101) aimed to assess the safety, tolerability, immunogenicity and preliminary clinical activity of PDC*lung01 in NSCLC patients, alone or in combination with anti-PD-1 treatment.
  • PDC*Line is reporting preliminary efficacy results for 19 evaluable patients in the B2 cohort that reached the 9-month PFS mark.
  • The final analysis of the clinical trial including the 45 patients from the B2 cohort will be conducted in Q3, 2024.

Cassava Sciences Announces Completion of an Interim Safety Review of Oral Simufilam On-going Phase 3 Trials

Retrieved on: 
Monday, March 25, 2024
Conclusion, Cassava Sciences, MRI, Patient, Data, News, ARIA, Safety, DSMB, Dementia, Cassava, Pharmaceutical industry

An Independent Data and Safety Monitoring Board (DSMB) Recently Evaluated the Interim Patient Safety Database for Oral Simufilam in On-going Phase 3 Trials.

Key Points: 
  • An Independent Data and Safety Monitoring Board (DSMB) Recently Evaluated the Interim Patient Safety Database for Oral Simufilam in On-going Phase 3 Trials.
  • AUSTIN, Texas, March 25, 2024 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (Nasdaq: SAVA), a biotechnology company focused on Alzheimer’s disease, today announced the completion of another interim safety review of simufilam in on-going Phase 3 clinical trials in patients with Alzheimer’s disease.
  • A routine, scheduled meeting of a Data and Safety Monitoring Board (DSMB) recommended that both of Cassava Sciences’ on-going Phase 3 studies continue as planned, without modification.
  • “This interim safety review is yet another milestone for the clinical development of simufilam,” said Remi Barbier, President & CEO.

Sequana Medical announces three month follow-up data from MOJAVE non-randomized cohort confirming dramatic improvement in diuretic response and virtual elimination of loop diuretics following DSR® therapy

Retrieved on: 
Monday, March 25, 2024
Cardiorenal syndrome, Congestion, Myocardial infarction, DSR, Brussels Stock Exchange, Liver disease, Partnership, RED, Patient, 2.0, Blood urea nitrogen, Cancer, Fluid, Heart, Heart failure, Furosemide, EGFR, Safety, DSMB, Overload, Hyponatremia, Hypertension, Pharmaceutical industry

These final data support DSR’s mechanism of action as breaking the vicious cycle of cardiorenal syndrome.

Key Points: 
  • These final data support DSR’s mechanism of action as breaking the vicious cycle of cardiorenal syndrome.
  • Unlike loop diuretics which, on average, have a negative impact on renal function and diuretic response, DSR has again demonstrated its potential to restore renal health and control of volume status.
  • Dramatic improvement in diuretic response and stable kidney function: During the four-week DSR treatment period, all three patients maintained euvolemia without the need of loop diuretics.
  • In January 2024, the independent DSMB approved the start of the randomized MOJAVE cohort following review of the safety data reported from the non-randomized cohort.