EudraCT

MEDIA RELEASE (PDF) Kinarus Receives Positive Recommendation from DSMB to Continue Phase 2 Trial in COVID-19

Retrieved on: 
Tuesday, April 12, 2022
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Phase 2 trial assesses Kinarus' oral therapy, KIN001, to reduce mortality and the need for respiratory support in hospitalized COVID-19 patients

Key Points: 
  • Phase 2 trial assesses Kinarus' oral therapy, KIN001, to reduce mortality and the need for respiratory support in hospitalized COVID-19 patients
    Basel, Switzerland, April 12, 2022.
  • Kinarus AG ('Kinarus'), a Swiss clinical-stage biopharmaceutical company announced today that the independent Data and Safety Monitoring Board ('DSMB') has completed its interim safety review of the Kinarus Phase 2 KINETIC study.
  • DSMB recommends that the study continue as designed with Kinarus' oral therapy, KIN001, for treating hospitalized patients with severe COVID-19.
  • The unblinded interim safety review by the DSMB was conducted after the enrollment and completion of the first 40 patients.

Aeterna Zentaris Provides Update on Ongoing Pivotal Phase 3 DETECT-Trial for the Diagnosis of Childhood-Onset Growth Hormone Deficiency

Retrieved on: 
Monday, March 21, 2022
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TORONTO, ONTARIO, March 21, 2022 (GLOBE NEWSWIRE) -- Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the “Company”), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products, today provided an update for its ongoing pivotal Phase 3 safety and efficacy study AEZS-130-P02 (the “DETECT-trial") evaluating macimorelin for the diagnosis of childhood-onset growth hormone deficiency (CGHD).

Key Points: 
  • The DETECT-trial is a multicenter, open-label trial to investigate the efficacy and safety of a single oral dose of 1.0 mg/kg macimorelin acetate as growth hormone stimulation test (GHST) in pediatric patients with suspected growth hormone deficiency (GHD).
  • Children and adolescents from two to less than 18 years of age with suspected growth hormone deficiency are to be included.
  • The macimorelin GHST will be performed twice for repeatability data and two standard GHSTs will be used as controls: arginine (i.v.)
  • The Company's lead product, macimorelin (Macrilen), is the first and only U.S. FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD).

Actuate Therapeutics Announces First Patient Enrolled in Phase 2 Clinical Trial of Elraglusib Plus Retifanlimab as Investigational First Line Therapy for Patients With Advanced Pancreatic Cancer

Retrieved on: 
Tuesday, March 1, 2022
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Elraglusib is Actuates proprietary small molecule glycogen synthase kinase-3 beta (GSK-3) inhibitor which is being developed for adults and children with advanced refractory cancers.

Key Points: 
  • Elraglusib is Actuates proprietary small molecule glycogen synthase kinase-3 beta (GSK-3) inhibitor which is being developed for adults and children with advanced refractory cancers.
  • We are very excited to launch this first study of elraglusib combined with retifanlimab as a potentially synergistic immune-modulatory approach for patients with advanced pancreatic cancer, said Anwaar Saeed, MD, study principal investigator.
  • Actuate also recently announced the initiation of a randomized study of elraglusib plus FOLFIRINOX alone or with Losartan also for patients with advanced pancreatic cancer in the first-line setting (NCT05077800).
  • Actuate is a clinical stage pharmaceutical company focused on the development and commercialization of novel therapeutics for cancers and inflammatory diseases.

Actuate Therapeutics Announces Initiation of a Multicenter Randomized Trial of Elraglusib Plus FOLFIRINOX As First Line Therapy for Advanced Pancreatic Cancer

Retrieved on: 
Monday, February 7, 2022
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Elraglusib is Actuates proprietary small molecule glycogen synthase kinase-3 beta (GSK-3) inhibitor which is being developed for adults and children with advanced refractory cancers.

Key Points: 
  • Elraglusib is Actuates proprietary small molecule glycogen synthase kinase-3 beta (GSK-3) inhibitor which is being developed for adults and children with advanced refractory cancers.
  • Novel approaches for patients with advanced pancreatic cancer are urgently required, said Dr Weekes.
  • The pre-clinical and clinical data being generated with elraglusib in a spectrum of cancers, including pancreatic cancer, is extremely encouraging and we are delighted to have initiated this study with elraglusib.
  • Actuate is a clinical stage pharmaceutical company focused on the development and commercialization of novel therapeutics for cancers and inflammatory diseases.

uniQure Announces Dosing of First Patients in European Open-Label Clinical Trial of AMT-130 Gene Therapy in Huntington’s Disease

Retrieved on: 
Monday, February 7, 2022
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and AMSTERDAM, Feb. 07, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the dosing of the first two patients in its European open-label Phase Ib/II clinical trial of AMT-130, a potential one-time gene-therapy approach for the treatment of Huntingtons disease.

Key Points: 
  • and AMSTERDAM, Feb. 07, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the dosing of the first two patients in its European open-label Phase Ib/II clinical trial of AMT-130, a potential one-time gene-therapy approach for the treatment of Huntingtons disease.
  • The clinical trial is taking place at several sites in Poland, the United Kingdom and Germany.
  • Since 1995, our center at the Institute of Psychiatry and Neurology (IPiN) has offered genetic testing and clinical care to Polish patients with Huntingtons disease, said Dr. Grzegorz Witkowski M.D.
  • uniQure is delivering on the promise of gene therapy single treatments with potentially curative results.

ORYZON Announces Approval for Serbian Arm of PORTICO, Vafidemstat’s Phase IIb Trial in Borderline Personality Disorder

Retrieved on: 
Tuesday, January 4, 2022
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: 2020-003469-20, ClinicalTrials.gov Identifier NCT04932291) is a multicenter, double-blind, randomized, placebo-controlled, Phase IIb trial to evaluate the efficacy and safety of vafidemstat in adult BPD patients.

Key Points: 
  • : 2020-003469-20, ClinicalTrials.gov Identifier NCT04932291) is a multicenter, double-blind, randomized, placebo-controlled, Phase IIb trial to evaluate the efficacy and safety of vafidemstat in adult BPD patients.
  • The trial will be conducted in 15-20 sites in Europe and US and aims to include about 160 patients distributed between two arms.
  • The PORTICOs protocol pre-specifies an interim analysis after the first 90 participants have completed the trial.
  • Currently, vafidemstat is in two Phase IIb trials in borderline personality disorder (PORTICO) and in schizophrenia patients (EVOLUTION).

Rallybio Reports Positive Data in Its Clinical Program for the Prevention of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

Retrieved on: 
Wednesday, December 1, 2021
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RLYB211, a plasma-derived polyclonal anti-HPA-1a antibody, is being evaluated for the prevention of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT), a potentially life-threatening rare disease that can cause uncontrolled bleeding in fetuses and newborns.

Key Points: 
  • RLYB211, a plasma-derived polyclonal anti-HPA-1a antibody, is being evaluated for the prevention of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT), a potentially life-threatening rare disease that can cause uncontrolled bleeding in fetuses and newborns.
  • We are both pleased with the RLYB211 findings and excited to move into the next phase of our FNAIT program by advancing RLYB212.
  • These data support Rallybios first-in-human clinical trial of its lead candidate RLYB212, a novel human monoclonal anti-HPA-1a antibody which has the same mechanism of action as RLYB211.
  • Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) is a potentially life-threatening rare disease that can cause uncontrolled bleeding in fetuses and newborns.

BioNTech Receives FDA Fast Track Designation for its FixVac Candidate BNT111 in Advanced Melanoma

Retrieved on: 
Friday, November 19, 2021
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MAINZ, GERMANY, November 19, 2021 BioNTech SE (Nasdaq: BNTX, BioNTech or the Company) today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for BNT111, an investigational cancer immunotherapy for the potential treatment of advanced melanoma.

Key Points: 
  • MAINZ, GERMANY, November 19, 2021 BioNTech SE (Nasdaq: BNTX, BioNTech or the Company) today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for BNT111, an investigational cancer immunotherapy for the potential treatment of advanced melanoma.
  • The Fast Track Designation underlines the potential of our FixVac platform to address current treatment challenges of pre-treated and immune checkpoint blocker experienced melanoma with limited standard of care therapy options left.
  • With the Fast Track status and support by the FDA, we aim to expedite the further development of the BNT111 program to provide a new therapeutic option for patients with life-threatening, hard-to-treat melanoma.
  • With the Fast Track Designation, the development of BNT111 can benefit from more frequent engagement with the FDA, which will support the collection of appropriate data needed to accelerate BNT111s development.

ORYZON Enrolls First Patient in EVOLUTION, a Phase IIb Clinical Trial With Vafidemstat in Schizophrenia

Retrieved on: 
Wednesday, November 17, 2021
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: 2021-000350-26) is a multicenter, double-blind, randomized, placebo-controlled, 24-week Phase IIb trial to evaluate the efficacy and safety of vafidemstat in adult schizophrenia patients under stable antipsychotic therapy.

Key Points: 
  • : 2021-000350-26) is a multicenter, double-blind, randomized, placebo-controlled, 24-week Phase IIb trial to evaluate the efficacy and safety of vafidemstat in adult schizophrenia patients under stable antipsychotic therapy.
  • The trial has two primary independent objectives: to assess the effect of vafidemstat on negative symptoms of schizophrenia, and to assess improvement on cognitive impairment associated with schizophrenia (CIAS).
  • Oryzons Chief Medical Officer for CNS, Dr. Michael Ropacki, said: We are excited for the first patient in (FPI) in EVOLUTION, our Phase IIb trial focused on the negative symptoms and cognitive impairments associated with schizophrenia.
  • Currently, vafidemstat is in two Phase IIb trials in borderline personality disorder (PORTICO) and in schizophrenia patients (EVOLUTION).

Axalbion Announces First Patients Treated in Phase 2 Clinical Trial in Chronic Cough with AX-8, a Novel TRPM8 Agonist

Retrieved on: 
Wednesday, October 20, 2021
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AX-8 has a novel mechanism of action targeting the TRPM8 ion channel, which may help normalize upper airway sensitivity in chronic cough patients, distinguishing it from other strategies in development.

Key Points: 
  • AX-8 has a novel mechanism of action targeting the TRPM8 ion channel, which may help normalize upper airway sensitivity in chronic cough patients, distinguishing it from other strategies in development.
  • With its safety profile and ease of administration, AX-8 has the potential to be a first-in-class drug to treat chronic cough.
  • The Phase 2 trial is a randomized, double-blind, placebo-controlled, crossover study evaluating the efficacy and safety of AX-8 in approximately 50 patients with refractory or unexplained chronic cough.
  • Axalbion is a privately held, clinical-stage biotechnology company developing novel medicines for the millions of patients who suffer from chronic respiratory disorders such as chronic cough.