WHIM syndrome

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 Fourth-Quarter and Full-Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, March 21, 2024
Security (finance), WHIM, WHIM syndrome, Conference call, Conference, RPD, New Drug Application, Ageing, Orphan, Wart, NDA, Priority review, Research and development, Immune system, File, ID, FDA, X4, PDUFA, Pharmaceutical industry

BOSTON, March 21, 2024 (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 fourth quarter and full year ended December 31, 2023, and highlighted key 2023 events and expected upcoming milestones.

Key Points: 
  • “Following an incredibly productive 2023, we are expecting a transformative year in 2024,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • 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 .
  • Following the completion of the call, a webcast replay of the conference call will be available on the company website.

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 Second-Quarter 2023 Financial Results, Provides Corporate Updates, and Reports Emerging Data from Chronic Neutropenia Clinical Program

Retrieved on: 
Thursday, August 10, 2023
Addition, Christophe, Maintenance, ANC, Priority review, Therapy, SG, European Hematology Association, Line, Conference call, Federation of Clinical Immunology Societies, Neutropenia, PIPE, Disclosure, Clinical trial, CIS, Research, Cash, Doctor of Philosophy, ID, Research and development, State Administrative Expenses, Russell, MD, Physician, Patient, WHIM syndrome, Patent, Immune system, Annual Meetings of the International Monetary Fund and the World Bank Group, EHA, Growth, Wart, G-CSF, R, Rare, Restricted, Conference, Safety, Pharmaceutical industry, Cryptocurrency, Bank, Infection, Hypogammaglobulinemia

Additional data from this ongoing Phase 2 trial are expected to be shared in the fourth quarter of 2023.

Key Points: 
  • Additional data from this ongoing Phase 2 trial are expected to be shared in the fourth quarter of 2023.
  • X4 is planning to initiate a Phase 3 clinical trial evaluating mavorixafor in certain chronic neutropenic disorders in the first half of 2024.
  • 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 and slide presentation can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .

X4 Pharmaceuticals Announces Industry Veteran Dr. Christophe Arbet-Engels to Join as Chief Medical Officer

Retrieved on: 
Tuesday, August 8, 2023
University, Interim, Assistance Publique–Hôpitaux de Paris, Drug development, Roche, Therapy, WHIM, Harvard University, Salk Institute for Biological Studies, Biogen, Hospital, MBA, Research, CMO, Doctor of Philosophy, Merck & Co., Rutgers University, Pierre and Marie Curie University, X4, Boehringer Ingelheim, MD, Patient, WHIM syndrome, Immune system, Growth, Rare, Pharmaceutical industry, Ligand Pharmaceuticals

“We are thrilled to welcome Christophe to our leadership team at this exciting time at X4,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.

Key Points: 
  • “We are thrilled to welcome Christophe to our leadership team at this exciting time at X4,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • We would also like to thank Dr. Murray Stewart, who has been serving as our Interim Chief Medical Officer, for his exceptional contributions and his leadership.
  • The company has made great progress and I look forward to adding to its continued forward momentum.
  • He received his MD and PhD in internal medicine/endocrinology-metabolism from the University of Paris, France, and completed his MBA at Rutgers University.

X4 Pharmaceuticals Closes $115 Million Loan Facility with Hercules Capital

Retrieved on: 
Thursday, August 3, 2023
Partnership, Therapy, Senior, WHIM, WHIM syndrome, Immune system, Rare, NYSE, Pharmaceutical industry, Bank

BOSTON, Aug. 03, 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 announced the closing of a $115 million loan facility with Hercules Capital, Inc. (NYSE: HTGC) (“Hercules”).

Key Points: 
  • BOSTON, Aug. 03, 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 announced the closing of a $115 million loan facility with Hercules Capital, Inc. (NYSE: HTGC) (“Hercules”).
  • “This expanded loan facility creates significant financial flexibility for X4 as we continue preparations for the potential commercial launch of mavorixafor in individuals with WHIM syndrome and continue to advance mavorixafor for certain chronic neutropenic disorders,” said Adam Mostafa, Chief Financial Officer of X4 Pharmaceuticals.
  • The term loan facility provides for up to $115 million of term loans in the aggregate, available to be funded in multiple tranches.
  • The facility refinances $32.5 million in outstanding principal indebtedness and extends the initial interest-only period and maturity of existing and future borrowings.

X4 Pharmaceuticals Announces Positive Phase 3 Results Showing Mavorixafor Reduced the Rate, Severity, and Duration of Infections vs. Placebo in Participants Diagnosed with WHIM Syndrome

Retrieved on: 
Tuesday, May 16, 2023
Infection, WHIM syndrome, Arm, Therapy, European Hematology Association, WHIM, Federation of Clinical Immunology Societies, CIS, CN, Patient, Immune system, EHA, Rare, FDA, Caregiver, Pharmaceutical industry

Following the announcement of positive top-line data from the 4WHIM trial in November 2022, today’s event focuses on the impact of mavorixafor on the rate, severity, and duration of infections in trial participants.

Key Points: 
  • Following the announcement of positive top-line data from the 4WHIM trial in November 2022, today’s event focuses on the impact of mavorixafor on the rate, severity, and duration of infections in trial participants.
  • “Today, we’re announcing that mavorixafor treatment demonstrated statistically significant and clinically meaningful improvements across a number of key infection metrics versus placebo in our pivotal, Phase 3 trial in those diagnosed with WHIM Syndrome,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • Approximately 90% of participants in the trial continued on to receive mavorixafor treatment in the ongoing open-label extension study.
  • The X4 live-event webcast presentation and the accompanying slides are now accessible on the investor relations section of the X4 website at www.x4pharma.com .

X4 Pharmaceuticals Announces $65 Million Private Placement Priced At-the-Market

Retrieved on: 
Tuesday, May 16, 2023
Therapy, Stifel, OrbiMed, WHIM, PIPE, Exchange, Sale, Adage Capital Management, NEA, WHIM syndrome, Immune system, Webcast, Security (finance), Rare, New Enterprise Associates, Safety, Pharmaceutical industry

X4 anticipates that gross proceeds from the PIPE will be approximately $65 million, before deducting fees to the placement agents and other estimated offering expenses payable by the Company.

Key Points: 
  • X4 anticipates that gross proceeds from the PIPE will be approximately $65 million, before deducting fees to the placement agents and other estimated offering expenses payable by the Company.
  • The closing of the financing is expected to occur on or about May 18, 2023, subject to customary closing conditions.
  • The webcast can also be accessed on the investor relations section of X4’s website at www.x4pharma.com.
  • X4 expects to use these funds for continued clinical development and commercial readiness of its lead candidate, mavorixafor, and for working capital, and general corporate purposes.