Myelokathexis

Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, WHIM, WHIM syndrome, Myelokathexis, Pneumonia, Bone marrow, CXCR4, Risk, Patient, Committee, Knowledge, Hypogammaglobulinemia, Antibody, Regulation, Viral, EMA, IRIS, Wart, Otitis, Protein, Lymphocyte, European, Infection, COMP, Immune system, European Commission, Neutrophil, Blood, Safety, Consultant, Marketing, Movement, Pharmaceutical industry

Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

Key Points: 


Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive

X4 Pharmaceuticals Reports Third-Quarter 2023 Financial Results and Provides Corporate Updates

Retrieved on: 
Thursday, November 9, 2023
Priority review, FDA, Ageing, PDUFA, Addition, Rare, Physician, Christophe, SG, MD, Conference, Organization, Doctor of Philosophy, PRV, Primary immunodeficiency, R, Society, Neutropenia, ASH, Wart, WHIM, Disease, ID, WHIM syndrome, Immune system, Research and development, Line, File, Security (finance), New Drug Application, NDA, Safety, Conference call, Patient, Diagnosis, State Administrative Expenses, Pharmaceutical industry, Rare-earth element, Hypogammaglobulinemia, Infection, Myelokathexis

BOSTON, Nov. 09, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the third quarter ended September 30, 2023 and highlighted key recent and upcoming expected milestones.

Key Points: 
  • ET
    BOSTON, Nov. 09, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the third quarter ended September 30, 2023 and highlighted key recent and upcoming expected milestones.
  • The FDA has notified X4 that it does not currently plan to hold an advisory committee meeting to review the filing.
  • Cash, Cash Equivalents, Restricted Cash, and Short-Term Marketable Securities: X4 had $142.7 million in cash, cash equivalents, restricted cash, and marketable securities as of September 30, 2023.
  • The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .

X4 Pharmaceuticals Announces FDA Acceptance with Priority Review of U.S. NDA for Mavorixafor in WHIM Syndrome

Retrieved on: 
Tuesday, October 31, 2023
Priority review, FDA, Ageing, PDUFA, Orphan, Rare, RPD, CXCR4, PRV, Primary immunodeficiency, Wart, WHIM, WHIM syndrome, Immune system, New Drug Application, NDA, Pharmaceutical industry, Hypogammaglobulinemia, Infection, Myelokathexis

BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that the United States Food and Drug Administration (FDA) has accepted for filing the company’s New Drug Application (NDA) for once-daily, oral mavorixafor to treat individuals aged 12 and older with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, primary immunodeficiency. The FDA granted Priority Review of the mavorixafor NDA, establishing a goal of six months review from the date of acceptance and assigning a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024.

Key Points: 
  • The FDA granted Priority Review of the mavorixafor NDA, establishing a goal of six months review from the date of acceptance and assigning a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024.
  • “The FDA’s acceptance of our mavorixafor NDA with priority review represents yet another significant step forward towards a potential treatment for those with WHIM syndrome, a rare disease for which there are currently no approved therapies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • Upon FDA approval of a product with RPD designation, the sponsor can receive a Priority Review Voucher that can be used to obtain priority review for a subsequent application or sold to another drug sponsor.
  • The NDA is supported by the results of the global, pivotal, 4WHIM Phase 3 clinical trial of once-daily, oral mavorixafor in individuals with WHIM syndrome.

X4 Pharmaceuticals Announces Submission of New Drug Application (NDA) to U.S. FDA for Mavorixafor in WHIM Syndrome

Retrieved on: 
Tuesday, September 5, 2023
European Hematology Association, File, WHIM, Federation of Clinical Immunology Societies, NDA, CIS, WHIM syndrome, Immune system, EHA, Primary immunodeficiency, Wart, Ageing, Rare, FDA, Pharmaceutical industry, Infection, Myelokathexis, Hypogammaglobulinemia

“The submission of our first NDA is a significant milestone in X4's journey to transform the care of those living with rare immunodeficiencies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.

Key Points: 
  • “The submission of our first NDA is a significant milestone in X4's journey to transform the care of those living with rare immunodeficiencies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • “We’re excited that this submission moves us one step closer to introducing what could be the first approved product in the U.S. for those with WHIM syndrome.
  • X4 has requested priority review for the application which, if granted, would provide a target FDA review period of six months from the application acceptance for filing date.
  • The NDA submission is supported by the results of the global, pivotal, 4WHIM Phase 3 clinical trial of once-daily, oral mavorixafor in individuals with WHIM syndrome.

X4 Pharmaceuticals Reports First-Quarter 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, May 4, 2023
CIS, Doctor of Philosophy, Associate, Icahn School of Medicine at Mount Sinai, X4, MD, WHIM syndrome, Patient, Translational research, Cell, Research and development, R, University, University of Massachusetts, Therapy, WHIM, Host, Wart, Immunology, Hospital, Rare, Pediatric Blood & Cancer, Safety, Abstract, Conference call, Rheumatology, Mount Sinai, ID, Immune system, Duke University School of Medicine, Federation of Clinical Immunology Societies, Annual Review of Cancer Biology, Restricted, ANC, SG, Clinical trial, Harvard Medical School, State Administrative Expenses, Annual general meeting, Upcoming, Pharmaceutical industry, Medical imaging, Dentistry, Management, Myelokathexis, Pediatrics, Conference, Hypogammaglobulinemia, Infection, Medicine

BOSTON, May 04, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a leader in the discovery and development of novel small-molecule therapeutics to benefit people with rare diseases of the immune system, today reported financial results for the first quarter ended March 31, 2023 and highlighted key upcoming expected milestones.

Key Points: 
  • “This is an exciting time at X4 as we look forward to delivering on several expected major milestones throughout the rest of 2023,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • Cash, Cash Equivalents & Restricted Cash: X4 had $94.4 million in cash, cash equivalents, and restricted cash as of March 31, 2023.
  • X4 will host a conference call and webcast today at 8:30 am ET to discuss these financial results and business highlights.
  • The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .

X4 Pharmaceuticals’ Presentations at ASH 2022 Further Highlight Opportunity for Mavorixafor in the Treatment of Chronic Neutropenia

Retrieved on: 
Monday, December 12, 2022
Security (finance), Fatigue, WHIM, Primary immunodeficiency, ASH, Disease, Neutrophil, Neutropenia, Pediatrics, Medication, Research, Blood, COVID-19, Diagnosis, Clinical trial, G-CSF, Trial of the century, Prevalence, FDA, Safety, Bone marrow, Birth, Society, HCPS, Therapy, Wart, Assistant, Quality of life, Hospital, Medicine, University, WHIM syndrome, Immune system, ANC, Patient, Knowledge Organization (journal), Cell, CXCR4, Degenerative disease, Cyclic neutropenia, Calendar, Risk, Pharmaceutical industry, Health insurance, Myelokathexis, Infection, Hypogammaglobulinemia

BOSTON, Dec. 12, 2022 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a leader in the discovery and development of novel small-molecule therapeutics to benefit people with diseases of the immune system, today provided a summary of its chronic neutropenia-related presentations at this year’s annual meeting of the American Society of Hematology (ASH), taking place December 10-13, 2022 in New Orleans.

Key Points: 
  • Data analyses continue to show that a single dose of oral mavorixafor effected meaningful increases in ANC across all participants, regardless of disease etiology or use of G-CSF.
  • The Phase 1b clinical trial has now been amended and a Phase 2 trial ( NCT04154488 ) is being initiated by X4 to assess the durability, safety, and tolerability of the chronic use of once-daily, oral mavorixafor in a larger chronic neutropenia patient population.
  • Participants were dosed with a single dose of 400 mg oral mavorixafor to assess the magnitude of treatment response.
  • X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law.

X4 Pharmaceuticals to Host Investor Webinar Highlighting New Mavorixafor Phase 1b Data in Chronic Neutropenia

Retrieved on: 
Wednesday, August 31, 2022
G-CSF, B-cell lymphoma, Partnership, Security (finance), Safety, Cancer, CXCR4, Trial of the century, Lists of diseases, Research, Neutropenia, Medication, Immunodeficiency, WHIM, WHIM syndrome, SEC, U.S. Securities and Exchange Commission, Cyclic neutropenia, GLOBE, WM, Biology, Immune system, Therapy, Risk, Clinical trial, Private Securities Litigation Reform Act, Q&A, Forward-looking statement, Patient, Pharmaceutical industry, Infection, Hypogammaglobulinemia, Myelokathexis

Members of X4 Pharmaceuticals management team will present clinical data on 25 patients from this fully enrolled trial and discuss mavorixafors potential to treat broader chronic neutropenia populations beyond its lead indication in Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) syndrome.

Key Points: 
  • Members of X4 Pharmaceuticals management team will present clinical data on 25 patients from this fully enrolled trial and discuss mavorixafors potential to treat broader chronic neutropenia populations beyond its lead indication in Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) syndrome.
  • The clinical trial ( NCT04154488 ) is a proof-of-concept Phase 1b open-label, multicenter study designed to assess the safety and tolerability of daily, oral mavorixafor with or without G-CSF, in participants with chronic neutropenic disorders including severe congenital, idiopathic, or cyclic neutropenia.
  • Participants were dosed with a single dose of oral mavorixafor to assess the magnitude of treatment response.
  • X4 Pharmaceuticals is a late-stage clinical biopharmaceutical company leading the discovery and development of novel therapies for people with diseases of the immune system.

X4 Pharmaceuticals Completes Enrollment in Phase 3 Mavorixafor Trial in Patients with WHIM Syndrome

Retrieved on: 
Monday, October 4, 2021
Degenerative disease, Neutropenia, Clinical trial, WHIM, Patient, NDA, Bone marrow, Therapy, Infection, SCN, Security (finance), Private Securities Litigation Reform Act, CXCR4, U.S. Securities and Exchange Commission, Cancer, Immunodeficiency, Orphan, Physician, Population, Medication, Adult, Caregiver, Immune system, Biology, WHIM syndrome, Autosomal dominant polycystic kidney disease, Ageing, Growth, Wart, Lymphocyte, Monocyte, Risk, Primary immunodeficiency, HPV, COVID-19, Trial of the century, Research, Cell, Safety, Forward-looking statement, Myelokathexis, Neutrophil, Quality of life, Time, Hypogammaglobulinemia, SEC, GLOBE, Pharmaceutical industry

BOSTON, Oct. 04, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel CXCR4-targeted small molecule therapeutics to benefit patients with diseases of the immune system, announced today that it has completed enrollment in the ongoing pivotal Phase 3 clinical trial of its lead candidate, mavorixafor, in the treatment of patients with genetically confirmed WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, a primary immunodeficiency caused by gain-of-function mutations in the CXCR4 gene. Thirty-one adult and pediatric patients have enrolled in the 4WHIM trial, which compares a once-daily, oral dose of mavorixafor to placebo across primary and secondary endpoints that include clinically relevant counts of neutrophils and lymphocytes, the frequency and severity of both infections and warts, as well as certain quality of life measurements. The trial was originally designed to enroll 18-28 patients.

Key Points: 
  • This major milestone achievement for X4 is a hopeful step forward for the thousands of WHIM patients with no disease-modifying treatment options, said Paula Ragan, Ph.D., President and Chief Executive Officer of X4.
  • The 4WHIM Phase 3 clinical trial ( NCT03995108 ) is a global, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of mavorixafor in genetically confirmed WHIM patients.
  • Originally designed to enroll 18-28 patients, the trial has enrolled 31 patients aged 12 and older who are receiving either 400 mg mavorixafor or placebo orally once daily for 52 weeks; all patients then become eligible to receive treatment with mavorixafor in an open-label trial extension.
  • X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law.

X4 Pharmaceuticals Announces Fast Track Designation Granted by the FDA to Mavorixafor for the Treatment of WHIM Syndrome

Retrieved on: 
Thursday, October 8, 2020
Autosomal dominant disorders, Rare diseases, Health, Chemokine receptors, Clinical medicine, Medical specialties, WHIM syndrome, CXCR4, Myelokathexis, Fast track, Breakthrough therapy, Hypogammaglobulinemia

Food and Drug Administration(FDA) has granted Fast Track Designation to its lead asset, mavorixafor, for the treatment of adult patients with WHIM (Warts,Hypogammaglobulinemia,Infections, andMyelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene.

Key Points: 
  • Food and Drug Administration(FDA) has granted Fast Track Designation to its lead asset, mavorixafor, for the treatment of adult patients with WHIM (Warts,Hypogammaglobulinemia,Infections, andMyelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene.
  • Mavorixafor is a potential first-in-class, once-daily, oral, small molecule antagonist of chemokine receptor CXCR4, currently being investigated in a global pivotal Phase 3 clinical trial, 4WHIM , for the treatment of WHIM syndrome.
  • The Fast Track Designation of mavorixafor for the treatment of WHIM syndrome further recognizes WHIM as a serious condition with a clear unmet need for an effective and potentially disease-modifying therapy, said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • In addition to Fast Track Designation, mavorixafor was previously granted Breakthrough Therapy Designation by the FDA, as well as Orphan Drug status by theFDA and theEuropean Commission for the treatment of WHIM syndrome.

X4 Pharmaceuticals Showcases Strategic Focus on WHIM Syndrome During 2020 Analyst Day Webinar

Retrieved on: 
Tuesday, April 7, 2020
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, FDA, Clinical trials, Medicine, Autosomal dominant disorders, Rare diseases, Congenital disorders, Articles, Health, Myelokathexis, Whim, Whim, Immunodeficiency, X4, Disease

The Analyst Day discussions provided details and background on WHIM (Warts, Hypogamma-globulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited immunodeficiency disease, and included a patient experience via video and an update on recently completed X4 market research on the prevalence of WHIM in the U.S.

Key Points: 
  • The Analyst Day discussions provided details and background on WHIM (Warts, Hypogamma-globulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited immunodeficiency disease, and included a patient experience via video and an update on recently completed X4 market research on the prevalence of WHIM in the U.S.
  • The event included a Q&A session with Dr. Dale and X4 senior management.
  • This robust algorithm then searched the database, identifying between 800 and 2,400 additional potential but unconfirmed and undiagnosed WHIM patients.
  • X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law.