Pharmacokinetics

Allakos Provides Business Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Thursday, March 14, 2024
Research, Atopic dermatitis, PD, Inflammation, Patient, SAN, American Academy, Allergy, Investment, Antibody, Pharmacokinetics, SAD, AAAAI, Annual general meeting, Pharmaceutical industry

Research and development expenses were $53.8 million in the fourth quarter of 2023 compared to $35.4 million in the fourth quarter of 2022, an increase of $18.4 million.

Key Points: 
  • Research and development expenses were $53.8 million in the fourth quarter of 2023 compared to $35.4 million in the fourth quarter of 2022, an increase of $18.4 million.
  • Allakos reported a net loss of $62.6 million in the fourth quarter of 2023 compared to $43.0 million in the fourth quarter of 2022.
  • Net loss per basic and diluted share was $0.71 for the fourth quarter of 2023 compared to $0.50 in the fourth quarter of 2022.
  • Allakos ended the fourth quarter of 2023 with $170.8 million in cash, cash equivalents and investments resulting in a net decrease in cash, cash equivalents and investments of $23.1 million during the fourth quarter of 2023.

Pharming Group reports fourth quarter and full year 2023 financial results

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Thursday, March 14, 2024
Patient, Health care, Epidemiology, Executive Committee, Laboratory, Microsoft Access, Lymphocyte, Clinical trial, European Commission, APDS, MHLW, AEG, Welfare, Ageing, Committee, EMA, Trial of the century, PID, CTLA4, Safety, Pharmacokinetics, Ministry, CHMP, ODD, U.S. FDA, Prevalence, PTEN, Disease, HAE, Immunodeficiency, VUS, Literature, PIK3R1, PMDA, FDA, DSM-IV codes, Diagnosis, Civil service commission, IRP, Haploinsufficiency, Genetic testing, MAA, Autoimmunity, World Games, Iberian languages, Marketing, MHRA, Euronext Amsterdam, PIK3CD, Physician, Pharmaceutical industry

The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.

Key Points: 
  • The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.
  • Revenues increased to US$7.9 million in the fourth quarter of 2023, driven by the continued increase in patients on paid therapy, and revenues were US$18.2 million for 2023.
  • Pharming made continued progress in the fourth quarter of 2023 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets.
  • Pharming filed regulatory submissions in Canada and Australia in the third quarter of 2023, and Israel in the second quarter.

Spero Therapeutics Announces Fourth Quarter and Full Year 2023 Operating Results and Provides a Business Update

Retrieved on: 
Wednesday, March 13, 2024
Gram-negative bacteria, Department of Defence, Urinary tract infection, GSK, Patient, Human services, Quality of life, IND, Tebipenem, Clinical trial, Therapy, Infection, Research, Policy, Treasurer, Carbapenem-resistant enterobacteriaceae, Safety, Pharmacokinetics, Federal, Food, AP, Drug discovery, Disease, Kidney, SPA, Acinetobacter baumannii, MDR, Pyelonephritis, Annual report, FDA, Army, HBR, Government, Unitary state, NTM, Assistant, Fungal infection, Pseudomonas aeruginosa, Hope, National Institutes of Health, ID, Mycobacterium avium complex, Bangladesh Technical Education Board, Administration, Pharmaceutical industry

“2023 was a productive year for Spero and we have been focused on execution across our programs,” said Sath Shukla, President, and Chief Executive Officer of Spero.

Key Points: 
  • “2023 was a productive year for Spero and we have been focused on execution across our programs,” said Sath Shukla, President, and Chief Executive Officer of Spero.
  • Top-line data from the Phase 2a proof-of-concept clinical trial in treatment-naive and treatment-experienced non-refractory NTM-PD patients is expected in 2H 2024.
  • Spero commenced enrollment in PIVOT-PO, a global, randomized, double-blind, Phase 3 clinical trial of tebipenem HBr in patients with cUTI, including AP in December 2023.
  • Total revenue for the fourth quarter of 2023 was $73.5 million, compared with total revenue of $47.4 million for the fourth quarter of 2022.

Orchestra BioMed Demonstrates Strength of Cardiovascular Pipeline with Virtue® SAB and AVIM Therapy Presentations at CRT 2024 Annual Meeting

Retrieved on: 
Wednesday, March 13, 2024
Blood pressure, CRT, Investigational device exemption, HOPE, Patient, Inflammation, Stroke, Gold, Restenosis, ISR, Medtronic, Beat (music), Trial of the century, Virtue, Sirolimus, Stent, Safety, Pharmacokinetics, SAB, Hypertension, Molecular mass, Food, Medical device

A second presentation featured recently reported clinical data on atrioventricular interval modulation (“AVIM”) therapy (also known as BackBeat CNT), an investigational therapy delivered via standard dual-chamber pacemakers designed to immediately, substantially and persistently lower blood pressure.

Key Points: 
  • A second presentation featured recently reported clinical data on atrioventricular interval modulation (“AVIM”) therapy (also known as BackBeat CNT), an investigational therapy delivered via standard dual-chamber pacemakers designed to immediately, substantially and persistently lower blood pressure.
  • The AVIM presentation also outlined the design of the currently enrolling BACKBEAT global pivotal study evaluating AVIM therapy in hypertensive pacemaker patients in collaboration with Medtronic.
  • Specifically, the presented PK data showed that:
    Molecular weight of Sostenocel remained unchanged prior to elimination, showing no evidence of in-vivo degradation.
  • AVIM therapy demonstrated:
    Consistent favorable hemodynamic effects using both conduction system, as well as traditional right ventricular lead placements.

Crinetics Announces Positive Topline Results from Phase 2 Trial of Paltusotine for the Treatment of Carcinoid Syndrome

Retrieved on: 
Tuesday, March 12, 2024
Conference, Acromegaly, Patient, SAN, Carcinoid syndrome, Conference call, Safety, Pharmacokinetics, Therapy, FDA, Pharmaceutical industry

SAN DIEGO, March 12, 2024 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors, today announced positive topline results from its open-label Phase 2 carcinoid syndrome study of paltusotine, an oral, once-daily investigational compound being developed for the treatment of acromegaly and carcinoid syndrome.

Key Points: 
  • “We are very pleased that these results from our Phase 2 study of paltusotine in carcinoid syndrome confirm our decision to move expeditiously toward Phase 3,” said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics.
  • “These results highlight the potential of paltusotine to deliver significant benefits to patients living with the debilitating symptoms of carcinoid syndrome.
  • Twenty-six of the 30 participants who completed the randomized treatment phase enrolled in the long-term extension phase of the study.
  • “There is a critical need for better treatment options for patients with neuroendocrine tumors who experience carcinoid syndrome.

Inozyme Pharma Reports Full Year 2023 Financial Results and Provides Business Highlights

Retrieved on: 
Tuesday, March 12, 2024
Administration, Plasma, Kidney disease, ECTS, Massachusetts General Hospital, Pyrophosphate, PD, ASN, Partnership, Pharmacokinetics, Calciphylaxis, G&A, PROPEL, Doctor of Philosophy, Chemistry, Investment, PXE, Society, Pathology, Therapy, Safety, Research and development, ESKD, ENPP1, MMSc, Natural history, Pharmaceutical industry

BOSTON, March 12, 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 reported financial results for the full year ended December 31, 2023, and provided recent business highlights.

Key Points: 
  • “We believe we are well-positioned to continue to advance INZ-701 through several anticipated value-creating milestones in the year ahead,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
  • Calciphylaxis represents a devastating condition characterized by pathologic mineralization and intimal proliferation.
  • Patients will receive 1.8 mg/kg of INZ-701 once weekly, coinciding with their hemodialysis treatment, for a total of 30 days.
  • The study’s primary endpoint will assess safety and change from baseline plasma PPi concentration, with secondary endpoints including PK and PD parameters.

Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients

Retrieved on: 
Tuesday, March 12, 2024
Pharmacokinetics, Niemann–Pick disease, Hospital, NPC, GM2, Patient, RAINBOW, Lead, Safety, Pharmaceutical industry

Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW ( Phase 2 RAINBOW study NCT05758922 ).

Key Points: 
  • Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW ( Phase 2 RAINBOW study NCT05758922 ).
  • The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients from the age of 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC).
  • The aim of the RAINBOW study is to determine the safety, pharmacodynamics, and pharmacokinetics of two different doses of nizubaglustat in patients, in order to identify the target dosage for Azafaros’ planned Phase 3 pivotal studies.
  • Azafaros is grateful to the patients and their families who made the decision to participate in this study.”

NiKang Therapeutics Presents the Discovery and Unique Mechanism of Action of a Selective CDK2 Inhibitor NKT3447 at AACR Annual Meeting 2024

Retrieved on: 
Friday, April 5, 2024
Oncology, Health, Other Science, Clinical Trials, Research, Science, Biotechnology, CDK1, Phosphorylation, CDK2, CDK, Patient, Cell cycle, AACR, Therapy, Cancer, Safety, Pharmacokinetics, Cyclin E, Pharmaceutical industry

NKT3447 is an orally bioavailable small molecule CDK2 inhibitor that exhibits high selectivity against CDK1 and other CDKs, prolonged pharmacodynamic effects, and a distinct mechanism of action.

Key Points: 
  • NKT3447 is an orally bioavailable small molecule CDK2 inhibitor that exhibits high selectivity against CDK1 and other CDKs, prolonged pharmacodynamic effects, and a distinct mechanism of action.
  • It downregulates cyclin E and suppresses activating phosphorylation of CDK2 on Thr160.
  • “We are pleased to share the discovery of NKT3447, a highly selective CDK2 inhibitor with unique properties, at the AACR Annual Meeting”, said Zhenhai Gao, Ph.D., co-founder, president, and CEO of NiKang.
  • Discovery of a selective slow-off CDK2 inhibitor NKT3447 with distinct features of suppressing CDK2, downregulating cyclin E, and achieving prolonged pathway inhibition

Merck Initiates Phase 3 Clinical Trial of MK-1084, an Investigational Oral KRAS G12C Inhibitor, in Combination with KEYTRUDA® (pembrolizumab) for First-Line Treatment of Certain Patients With Metastatic Non-Small Cell Lung Cancer

Retrieved on: 
Thursday, April 4, 2024
Oncology, Health, FDA, General Health, Clinical Trials, Pharmaceutical, Biotechnology, MSD, Congress, Mutation, NSCLC, Progression-free survival, Patient, Neoplasm, Merck & Co., Cancer, Lung cancer, TPS, MRK, ESMO, Safety, Pharmacokinetics, PD-L1, Astex Pharmaceuticals, Pharmaceutical industry

“KRAS is among the most prevalent mutations in cancer and KRAS G12C is the most common KRAS mutation in patients with non-small cell lung cancer,” said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories.

Key Points: 
  • “KRAS is among the most prevalent mutations in cancer and KRAS G12C is the most common KRAS mutation in patients with non-small cell lung cancer,” said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories.
  • The primary endpoints of the study are progression-free survival and overall survival, and key secondary endpoints include objective response rate and duration of response.
  • Preliminary safety and efficacy data from this trial were previously presented at the European Society for Medical Oncology (ESMO) Congress in 2023.
  • MK-1084 is being developed through a collaboration with Taiho Pharmaceutical Co. Ltd and Astex Pharmaceuticals (UK), a wholly owned subsidiary of Otsuka Pharmaceutical Co., Ltd.

iECURE Announces FDA Clearance of Investigational New Drug Application for ECUR-506 to Initiate OTC-HOPE Trial for Treatment of Neonatal Onset Ornithine Transcarbamylase Deficiency in the U.S.

Retrieved on: 
Thursday, April 4, 2024
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Gene editing, Therapeutic Goods Administration, University, Patient, Gene, MD, Quality of life, IND, GTP, Liver, MHRA, OTC, Pediatrics, Safety, Pharmacokinetics, TGA, Food, Medication, Pharmaceutical industry

ECUR-506 will be evaluated in the OTC-HOPE study in newborn males with genetically confirmed neonatal onset OTC deficiency.

Key Points: 
  • ECUR-506 will be evaluated in the OTC-HOPE study in newborn males with genetically confirmed neonatal onset OTC deficiency.
  • The OTC-HOPE study was previously cleared to begin in the United Kingdom by the Medicines & Healthcare Products Regulatory Agency (MHRA) and Australia by the Therapeutic Goods Administration (TGA).
  • “There is a significant need for clinical research and treatment options for newborns with severe, neonatal onset OTC deficiency,” said Gabriel M. Cohn, MD, Chief Medical Officer of iECURE.
  • “We hope that treatment with this investigational therapeutic will show promise for clinically meaningful improvements for infants with neonatal onset OTC deficiency and their families.”