Cohort

EQS-News: Heidelberg Pharma announces progress into Cohort 6 with its proprietary ATAC candidate HDP-101 in Phase I/IIa multiple myeloma study

Retrieved on: 
Wednesday, April 10, 2024
Multiple myeloma, Bone marrow, Cohort, Infection, Incidence, BCMA, Patient, Plasma cell, Mortality, B cell, Spacecraft, AACR, Annual report, Antibody, The Annual Report, Safety, Pharmacokinetics, HPHA, FDA, ATAC, Food, Pharmaceutical industry

Multiple myeloma is a type of blood cancer that develops from plasma cells in the bone marrow and can affect more than one part of the body.

Key Points: 
  • Multiple myeloma is a type of blood cancer that develops from plasma cells in the bone marrow and can affect more than one part of the body.
  • Plasma cells are a type of blood cell that makes antibodies to fight infection, created by bone marrow.
  • Heidelberg Pharma’s Phase I/IIa clinical study is an ongoing, non-randomised, open label study which is actively enrolling patients with relapsed or refractory multiple myeloma or other plasma cell disorders expressing BCMA.
  • Prof. Dr. Andreas Pahl, Chief Executive Officer at Heidelberg Pharma, said: “Our proprietary ATAC candidate HDP-101 is showing exciting potential for treating multiple myeloma.

Inozyme Pharma Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency (PXE) and ENPP1 Deficiency

Retrieved on: 
Monday, April 8, 2024
Patient, Cerebrovascular disease, Stroke, Accelerated approval (FDA), European Medicines Agency, Doctor of Philosophy, Osteomalacia, PROMIS, Therapy, Conditional, Natural history, CTX, Pros, ADAS, Risk, DSM-IV codes, Cohort, Aes, Alkaline phosphatase, Rickets, EMA, PXE, Safety, Epilepsy, Clinical trial, Food, Hospital, Disability, BSAP, Cardiovascular disease, Disease, PD, Paresis, Consultant, Hypophosphatemia, Fatigue, Survivor, Sclera, FDA, Observational study, Pyrophosphate, Choroid, Incidence, Retina, Biomarker, Life, Event, Medical imaging

BOSTON, April 08, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive topline safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE, pseudoxanthoma elasticum) and ENPP1 Deficiency.

Key Points: 
  • “We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
  • The patients were assigned to three dose cohorts of INZ-701: 0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=4).
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.

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)].

Ocugen Provides Business Update with Certain Financials for the Year Ending 2023

Retrieved on: 
Tuesday, April 2, 2024
Stargardt disease, Disease, Safety, Webcast, Inflammation, Patient, CGMP, Data, Inhalation, Cohort, RP, Life, Bank statement, Investment, Clinical trial, NIAID, Injection, AMD, Vaccine, Nextgen, Geographic atrophy, RHO, FDA, Lipid metabolism, Government, Oxidative stress, Pharmaceutical industry

Completed Cohort 1 dosing for OCU410 and OCU410ST gene therapy clinical studies for geographic atrophy (GA) and Stargardt disease, respectively

Key Points: 
  • Completed Cohort 1 dosing for OCU410 and OCU410ST gene therapy clinical studies for geographic atrophy (GA) and Stargardt disease, respectively
    MALVERN, Pa., April 02, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today provided a general business update with certain financials for the year ending 2023.
  • During the fourth quarter of 2023, the Company announced its mucosal vaccine candidate, OCU500, was chosen for the multi-billion-dollar NIAID Project NextGen initiative.
  • The first patient was dosed in the Phase 1/2 trials to assess the safety and efficacy of OCU410ST for Stargardt disease in November 2023.
  • Ocugen’s cash, cash equivalents, and investments totaled $39.5 million as of December 31, 2023, compared to $90.9 million as of December 31, 2022.

Kiniksa Pharmaceuticals Announces Development Indication for Abiprubart

Retrieved on: 
Tuesday, April 2, 2024
Janus, Week, CD154, Rheumatoid arthritis, Jaki, Patient, Cohort, KNSA, Joint, Clinical trial, C-reactive protein, CD40, Least squares, Pharmacokinetics, Safety, FDA, Rheumatoid factor, Pharmaceutical industry

HAMILTON, Bermuda, April 02, 2024 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (Kiniksa), a commercial-stage biopharmaceutical company with a pipeline of immune-modulating assets designed to target a spectrum of cardiovascular and autoimmune diseases, today announced plans to initiate a Phase 2b trial with abiprubart in Sjogren’s Disease. Additionally, the company announced data from Cohort 4 of the Phase 2 clinical trial of abiprubart in rheumatoid arthritis. Abiprubart is an investigational humanized anti-CD40 monoclonal antibody designed to inhibit CD40-CD154 (CD40 ligand) interaction.

Key Points: 
  • HAMILTON, Bermuda, April 02, 2024 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (Kiniksa), a commercial-stage biopharmaceutical company with a pipeline of immune-modulating assets designed to target a spectrum of cardiovascular and autoimmune diseases, today announced plans to initiate a Phase 2b trial with abiprubart in Sjogren’s Disease.
  • Additionally, the company announced data from Cohort 4 of the Phase 2 clinical trial of abiprubart in rheumatoid arthritis.
  • Abiprubart is an investigational humanized anti-CD40 monoclonal antibody designed to inhibit CD40-CD154 (CD40 ligand) interaction.
  • “We believe abiprubart has the potential to provide meaningful benefit to patients suffering from Sjogren’s Disease, a debilitating disorder with no current FDA-approved therapies.

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)].

Perspective Therapeutics Provides Recent Business Highlights and Reports Fiscal Year 2023 Financial Results

Retrieved on: 
Thursday, March 28, 2024
Stony Brook University, University, Adenocarcinoma, PRRT, Patient, Mayo Clinic, Sale, Bristol Myers Squibb, Society, IND, European Journal, Therapy, SPECT, Melanoma, Research, MC1R, Fortis Healthcare, SNMMI, Cohort, Trial of the century, Radiopharmaceutical, Cancer, Nivolumab, Safety, CATX, PSMA, Food, LNTH, BMY, IIT, Prostate cancer, Nuclear medicine, EANM, NETS, Institute of technology, Diagnosis, Glomus tumor, PET, Prostate, Small business, Bangladesh Technical Education Board, Molecular imaging, Pharmaceutical industry

SEATTLE, March 28, 2024 (GLOBE NEWSWIRE) -- Perspective, Therapeutics, Inc. (NYSE AMERICAN: CATX), a radiopharmaceutical company that is pioneering advanced treatment applications for cancers throughout the body, today provided a business update and announced fiscal year 2023 financial results for the period ended December 31, 2023.

Key Points: 
  • SEATTLE, March 28, 2024 (GLOBE NEWSWIRE) -- Perspective, Therapeutics, Inc. (NYSE AMERICAN: CATX), a radiopharmaceutical company that is pioneering advanced treatment applications for cancers throughout the body, today provided a business update and announced fiscal year 2023 financial results for the period ended December 31, 2023.
  • “We made tremendous progress during 2023 in building a fully integrated radiopharmaceuticals company dedicated to advancing potentially best- or first-in-class alpha-particle therapies,” said Thijs Spoor, Perspective Therapeutics' CEO.
  • Preliminary results from Cohorts 1 and 2 of the Phase 1/2a trial are expected in the third quarter 2024.
  • In January 2024, Perspective Therapeutics entered into strategic agreements with Lantheus Holdings, Inc. and its affiliates (“Lantheus”) (NASDAQ: LNTH).

4DMT Announces Update on Regulatory Interactions and Development Path for 4D-710 for Treatment of Cystic Fibrosis

Retrieved on: 
Thursday, March 28, 2024
CDMO, Conference, TDN, Patient, Lung, Cohort, RNA, EMA, Technology transfer, Cystic fibrosis, European Medicines Agency, GMP, Clinical trial, ECFS, Safety, CFTR, AMD, Food, FDA, VG, Pharmaceutical industry

Phase 3 clinical endpoints include changes after 4D-710 treatment in ppFEV1, quality-of-life (Cystic Fibrosis Questionnaire Revised Respiratory Domain, CFQ-R-RD) and frequency of pulmonary exacerbations.

Key Points: 
  • Phase 3 clinical endpoints include changes after 4D-710 treatment in ppFEV1, quality-of-life (Cystic Fibrosis Questionnaire Revised Respiratory Domain, CFQ-R-RD) and frequency of pulmonary exacerbations.
  • The Company anticipates initiation of technology transfer to a commercial contract development and manufacturing organization (CDMO) in H1 2025.
  • Our goal is to initiate Phase 3 development in H2 2025 with 4D-710 suspension GMP process clinical trial material.
  • We look forward to sharing interim clinical data from the AEROW Phase 1/2 clinical trial at the ECFS conference in June 2024.”

Omega Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Strategic Update

Retrieved on: 
Thursday, March 28, 2024
HCC, Research, Epigenomics, Toxicity, Obesity, Cell death, Growth, Burns, Gene expression, Novo Nordisk, NSCLC, AASLD, Appetite, OMEGA, DCR, Patient, Lung, Tissue, Cohort, CXCL9, CXCL10, Omega, G&A, Cancer, Messenger, Lung cancer, Human, Life expectancy, Liver regeneration, DLT, Fibrosis, Thermogenesis, Therapy, MYC, Liver, Pharmacokinetics, Hepatitis, CXCL11, LNP, Pharmaceutical industry

Research and development (R&D) expenses for the fourth quarter of 2023 were $15.5 million, compared to $26.0 million for the fourth quarter of 2022.

Key Points: 
  • Research and development (R&D) expenses for the fourth quarter of 2023 were $15.5 million, compared to $26.0 million for the fourth quarter of 2022.
  • General and administrative (G&A) expenses for the fourth quarter of 2023 were $6.2 million, compared to $5.7 million for the fourth quarter of 2022.
  • Net loss for the fourth quarter of 2023 was $20.2 million, compared to $30.8 million for the fourth quarter of 2022.
  • The decrease in net loss for 2023 compared to 2022 was primarily due to decreases in R&D expenses.

Zafin™ Unveils Two Powerful Capabilities: Dynamic Cohorts and Signals

Retrieved on: 
Wednesday, March 27, 2024
Senior, Cohort, Software as a service, Growth, Analytics, Retail

Dynamic Cohorts helps address these challenges by transforming the way bank employees group customers, offering a more agile approach to traditional segmentation.

Key Points: 
  • Dynamic Cohorts helps address these challenges by transforming the way bank employees group customers, offering a more agile approach to traditional segmentation.
  • With the ability to quickly create cohorts that segment customers based on demographics and current banking behaviors, Dynamic Cohorts empowers banks with accelerated customer retargeting capabilities.
  • To complement Dynamic Cohorts, Zafin also introduces Signals , an advanced alerting capability that enables banks to deliver timely updates tailored to customers' evolving needs.
  • Dynamic Cohorts and Signals are available today as part of the integrated Zafin platform.