Investigational New Drug

REPEAT -- Quoin Pharmaceuticals Announces Additional Positive Clinical Data from Open-Label, Single-Arm Clinical Trial in Netherton Syndrome

Retrieved on: 
Tuesday, October 24, 2023
Attention deficit hyperactivity disorder predominantly inattentive, IND, Itch, Skin, Quality of life, Syndrome, Safety, Patient, Investigational New Drug, Pharmaceutical industry, Netherton syndrome, Netherton

ASHBURN, Va., Oct. 24, 2023 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a specialty pharmaceutical company focused on rare and orphan diseases, announces additional positive clinical data from its ongoing open-label study evaluating the safety and efficacy of QRX003 as a potential treatment for Netherton Syndrome (NS).

Key Points: 
  • All subjects in the study are continuing to receive off-label systemic therapy for the duration of the trial.
  • Quoin CEO, Dr. Michael Myers, said, “While acknowledging that this is still early stage data, we are very pleased to announce today additional positive results from our ongoing open-label study in Netherton Syndrome.
  • Furthermore, the absence of any safety concerns from the study to date is a positive indicator for the ongoing clinical development of the product.
  • For more information about Quoin’s clinical trials in Netherton Syndrome, please visit: https://www.nethertonsyndromeclinicaltrials.com/

Quoin Pharmaceuticals Announces Additional Positive Clinical Data from Open-Label, Single-Arm Clinical Trial in Netherton Syndrome

Retrieved on: 
Tuesday, October 24, 2023
Attention deficit hyperactivity disorder predominantly inattentive, IND, Itch, Skin, Quality of life, Syndrome, Safety, Patient, Investigational New Drug, Pharmaceutical industry, Netherton syndrome, Netherton

ASHBURN, Va., Oct. 24, 2023 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a specialty pharmaceutical company focused on rare and orphan diseases, announces additional positive clinical data from its ongoing open-label study evaluating the safety and efficacy of QRX003 as a potential treatment for Netherton Syndrome (NS).

Key Points: 
  • ASHBURN, Va., Oct. 24, 2023 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a specialty pharmaceutical company focused on rare and orphan diseases, announces additional positive clinical data from its ongoing open-label study evaluating the safety and efficacy of QRX003 as a potential treatment for Netherton Syndrome (NS).
  • All subjects in the study are continuing to receive off-label systemic therapy for the duration of the trial.
  • Quoin CEO, Dr. Michael Myers, said, “While acknowledging that this is still early stage data, we are very pleased to announce today additional positive results from our ongoing open-label study in Netherton Syndrome.
  • For more information about Quoin’s clinical trials in Netherton Syndrome, please visit: https://www.nethertonsyndromeclinicaltrials.com/

Endogena Therapeutics Secures FDA Green Light for Breakthrough AMD Treatment

Retrieved on: 
Tuesday, October 24, 2023
Vance DeGeneres, FDA, IND, Food, Hope, AMD, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Doctor of Philosophy, MBA, Geographic atrophy, Cell, Retinitis pigmentosa, Iris (anatomy), EA, RPE, GA, Regenerative medicine, Therapy, Patient, SAN, Investigational New Drug, Pharmaceutical industry, Management

The U.S. Food and Drug Administration (FDA) has granted clearance for Endogena's Investigational New Drug (IND) application, opening the door to a groundbreaking clinical investigation of EA 2351.

Key Points: 
  • The U.S. Food and Drug Administration (FDA) has granted clearance for Endogena's Investigational New Drug (IND) application, opening the door to a groundbreaking clinical investigation of EA 2351.
  • This innovative compound offers hope to patients battling geographic atrophy (GA), an advanced form of age-related macular degeneration (AMD).
  • Endogena Therapeutics remains dedicated to its mission of unlocking the body's regenerative potential and making a meaningful impact on patients' lives.
  • This latest achievement reinforces the company's commitment to advancing innovative therapies for conditions that urgently require novel treatment options.

SAB Biotherapeutics Receives Australian Approval to Commence Phase 1 Clinical Trial of SAB-142, a Potential Disease-Modifying Treatment for Type 1 Diabetes

Retrieved on: 
Thursday, October 19, 2023
FDA, Trial of the century, IND, Food, MD, MBA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmacokinetics, ATG, Clinical trial, U.S. FDA, Immunotherapy, Higgs phase, Safety, Patient, SAB, Investigational New Drug, Pharmaceutical industry, T1D

SIOUX FALLS, S.D., Oct. 19, 2023 (GLOBE NEWSWIRE) -- SAB Biotherapeutics, Inc. (Nasdaq: SABS), a clinical-stage biopharmaceutical company with a novel immunotherapy platform that is developing fully-human anti-thymocyte immunoglobulin (hIgG) for delaying the onset or progression of type 1 diabetes (T1D), today announced that it completed the approval process to commence a first-in-human Phase 1 clinical trial investigating SAB-142 in Australia.

Key Points: 
  • This first-in-human trial is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the SAB-142.
  • Approval by the HREC is confirmation that SAB has successfully completed all pre-clinical safety and efficacy testing required to commence a Phase 1 clinical trial.
  • More information about the Phase 1 clinical trial with SAB-142 (ACTRN:12623001089628) can be found here .
  • “We’d like to thank our clinical trial partners and express gratitude to everyone involved for their shared dedication to advancing innovative therapeutic options for those living with T1D.”

Vaxcyte Announces FDA Clearance of Investigational New Drug Application for VAX-31 for the Prevention of Invasive Pneumococcal Disease in Adults

Retrieved on: 
Thursday, October 19, 2023
PCV, FDA, Trial of the century, Genetically modified bacteria, IND, Ageing, Food, IPD, Disease, Streptococcus pneumoniae, Regulation of tobacco by the U.S. Food and Drug Administration, Meningitis, Safety, SAN, Investigational New Drug, Vaccine, Pharmaceutical industry

SAN CARLOS, Calif., Oct. 19, 2023 (GLOBE NEWSWIRE) -- Vaxcyte, Inc. (Nasdaq: PCVX), a vaccine innovation company engineering high-fidelity vaccines to protect humankind from the consequences of bacterial diseases, announced today that the U.S. Food and Drug Administration (FDA) has cleared the Company’s adult Investigational New Drug (IND) application for VAX-31, a 31-valent pneumococcal conjugate vaccine (PCV) candidate designed to prevent invasive pneumococcal disease (IPD). Vaxcyte expects to initiate the VAX-31 Phase 1/2 clinical study in adults in the fourth quarter of this year and announce topline safety, tolerability and immunogenicity results in the second half of 2024.

Key Points: 
  • -- VAX-31 is a 31-Valent Pneumococcal Conjugate Vaccine Designed to Provide Coverage for Approximately 95% of Disease Currently Circulating in the U.S.
  • Adult Population --
    SAN CARLOS, Calif., Oct. 19, 2023 (GLOBE NEWSWIRE) -- Vaxcyte, Inc. (Nasdaq: PCVX), a vaccine innovation company engineering high-fidelity vaccines to protect humankind from the consequences of bacterial diseases, announced today that the U.S. Food and Drug Administration (FDA) has cleared the Company’s adult Investigational New Drug (IND) application for VAX-31, a 31-valent pneumococcal conjugate vaccine (PCV) candidate designed to prevent invasive pneumococcal disease (IPD).
  • Vaxcyte expects to initiate the VAX-31 Phase 1/2 clinical study in adults in the fourth quarter of this year and announce topline safety, tolerability and immunogenicity results in the second half of 2024.
  • This is important given IPD contributes to high-case fatality rates, antibacterial resistance and meningitis, all of which are particularly concerning in the older adult population.”

Actimed Therapeutics Appoints Martin Walton as Chief Financial Officer

Retrieved on: 
Thursday, October 19, 2023
IND, AIM, Venture capital, Commercial bank, Cachexia, Toronto-Dominion Bank, Acquisition, Martin, NHS, Executive, Private equity, Association of British HealthTech Industries, LSE, CFO, Vulnerability and Risk Committee, Investment, Initial public offering, Liverpool Life Sciences UTC, Investigational New Drug, Wholesaling, Pharmaceutical industry, ActiMates

Actimed Therapeutics Ltd (“Actimed”), a UK based clinical stage specialty pharmaceutical company focused on bringing innovation to the treatment of cancer cachexia and other muscle wasting disorders, today announces the appointment of Martin Walton as Chief Financial Officer.

Key Points: 
  • Actimed Therapeutics Ltd (“Actimed”), a UK based clinical stage specialty pharmaceutical company focused on bringing innovation to the treatment of cancer cachexia and other muscle wasting disorders, today announces the appointment of Martin Walton as Chief Financial Officer.
  • Since 2009, Martin has been an active Venture Capital and Private Equity investor, portfolio manager, and advisor in life sciences working with several of the largest investors in the sector.
  • Robin Bhattacherjee, Actimed CEO, commented: “I am delighted to welcome Martin to Actimed, who brings with him a set of highly complementary skills and extensive industry experience.
  • Martin Walton, Actimed CFO commented: “I am excited to be joining Actimed at this important point in the company’s journey to develop the first globally approved product for cancer cachexia.

Appili Therapeutics’ Biodefense Vaccine Candidate ATI-1701 is Awarded Additional Funding from the U.S. Air Force Academy

Retrieved on: 
Wednesday, October 25, 2023
Health, Infectious Diseases, Research, Pharmaceutical, Science, Biotechnology, IND, Bioterrorism, Investigational New Drug, Infection, Francisella tularensis, Drug development, Pharming, DTRA, Defense Threat Reduction Agency, Partnership, TSX, Tularemia, Pharmaceutical industry, USAFA

ATI-1701 is the Company’s potential first-in-class vaccine candidate for the prevention of infection with Francisella tularensis, the causative agent of tularemia and a top-priority biothreat.

Key Points: 
  • ATI-1701 is the Company’s potential first-in-class vaccine candidate for the prevention of infection with Francisella tularensis, the causative agent of tularemia and a top-priority biothreat.
  • With this additional 6.6 million award, Appili’s ATI-1701 program has been awarded the total US$14 million in USAFA funding commitments announced on May 8, 2023.
  • In May 2023, Appili was awarded the first stage of funding commitment from USAFA of US$7.3 million.
  • Appili has also entered into an agreement with its lender whereby if needed, Appili has obtained temporary waivers of a minimum cash balance requirement.

KaliVir Immunotherapeutics Announces FDA Clearance of Investigational New Drug (IND) for Systemic Oncolytic Virus ASP1012 for Phase I Clinical Trials for Locally Advanced or Metastatic Solid Tumors

Retrieved on: 
Tuesday, October 24, 2023
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Trial of the century, IND, MBA, Drug development, Senior, Partnership, Cancer, Potyvirus, Patient, Investigational New Drug, Pharmaceutical industry, Astellas Pharma

KaliVir Immunotherapeutics, Inc. , a biotech company developing cutting-edge, multi-mechanistic oncolytic viral immunotherapy programs, today announced clearance of an Investigational New Drug (IND) application by the United States Food and Drug Administration (FDA) to initiate a Phase 1 clinical study of ASP1012 in participants with locally advanced or metastatic solid tumors.

Key Points: 
  • KaliVir Immunotherapeutics, Inc. , a biotech company developing cutting-edge, multi-mechanistic oncolytic viral immunotherapy programs, today announced clearance of an Investigational New Drug (IND) application by the United States Food and Drug Administration (FDA) to initiate a Phase 1 clinical study of ASP1012 in participants with locally advanced or metastatic solid tumors.
  • Discovered and developed by Kalivir and licensed to Astellas Pharma Inc. in December 2020, ASP1012 (formerly named VET2-L2) is a systemic oncolytic vaccinia virus therapy in which the virus is delivered intravenously and expresses Leptin-IL2 fusion protein as a therapeutic payload.
  • The trial is expected to begin in Q1 2024.
  • “ASP1012 was the first candidate built from our unique Vaccinia Enhanced Template (VET™) platform and we are thrilled to reach this important milestone,” said Steve Thorne, Ph.D, Chief Scientific Officer and founder of Kalivir.

First Patient Dosed in Beckley Psytech’s International Phase IIb Study of BPL-003, a Novel Synthetic Intranasal Formulation of 5-MeO-DMT, for Treatment Resistant Depression (TRD)

Retrieved on: 
Tuesday, October 24, 2023
Alternative Medicine, Mental Health, Health, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, FDA, United Kingdom, MADRS, IND, Food, Treatment, Investigational New Drug, Maintenance, Drug development, Atypical depression, Clinical trial, Depression, Safety, Patient, DSM-IV codes, TRD, Beckley Foundation, Pharmaceutical industry, Radiant exposure

It follows Beckley Psytech’s receipt of the U.S. Food and Drug Administration’s (FDA) first ever Investigational New Drug (IND) approval for a Phase IIb study of a short-acting psychedelic compound in February this year.

Key Points: 
  • It follows Beckley Psytech’s receipt of the U.S. Food and Drug Administration’s (FDA) first ever Investigational New Drug (IND) approval for a Phase IIb study of a short-acting psychedelic compound in February this year.
  • The study will be quadruply masked, with the patient, investigator, therapist and outcomes assessor all masked to the dose allocation of the subject in order to reduce expectancy bias.
  • The doses for this Phase IIb study were selected after Beckley Psytech’s preclinical and Phase I studies showed BPL-003 to be well-tolerated with a predictable dose-linear pharmacokinetic profile.
  • Beckley Psytech has partnered with Fluence to provide training for the standardised psychological support that will be offered to patients in the trial.

ReAlta Life Sciences Receives FDA Clearance for Phase 2 Trial of RLS-0071 in Patients with Acute Exacerbations of Chronic Obstructive Pulmonary Disease

Retrieved on: 
Tuesday, October 24, 2023
Research, FDA, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, Other Science, Science, Patient, IND, Food, Biomarker, Hypoxia (medical), Investigational New Drug, MD, HIE, Rodels-Realta railway station, Doctor of Philosophy, Inflammation, Quality of life, System, Immune system, Risk, Genetically modified bacteria, Safety, Hospital, AE, Food and Drug Administration, Chronic obstructive pulmonary disease, Death, COPD, Neutrophil extracellular traps, Pharmaceutical industry

RLS-0071 is the Company’s lead dual-action complement and innate inflammatory inhibitor peptide in development for AE-COPD, acute graft-versus-host disease (aGvHD), hypoxic-ischemic encephalopathy (HIE), and other rare and acute inflammatory diseases.

Key Points: 
  • RLS-0071 is the Company’s lead dual-action complement and innate inflammatory inhibitor peptide in development for AE-COPD, acute graft-versus-host disease (aGvHD), hypoxic-ischemic encephalopathy (HIE), and other rare and acute inflammatory diseases.
  • “Patients with chronic obstructive pulmonary disease (COPD) face the persistent threat of hospitalization, mechanical ventilation and death from an acute exacerbation, an acute worsening of symptoms triggered by a viral or bacterial infection, or environmental irritant.
  • “This FDA clearance marks an important new milestone for ReAlta as we explore the potential of RLS-0071 across multiple therapeutic areas."
  • The Phase 2 clinical trial will be a randomized, double-blind, placebo-controlled trial in hospitalized patients with AE-COPD.