Severe congenital neutropenia

X4 Pharmaceuticals Announces New Positive Phase 1b Data Supporting Mavorixafor’s Broad Potential in Chronic Neutropenia (CN)

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星期二, 九月 27, 2022
Lists of diseases, Neutropenia, Therapy, WHIM syndrome, Safety, Infection, ET, Trial of the century, Q&A, ANC, Risk, Neutrophil, WM, Immunodeficiency, Blood, Population, Private Securities Litigation Reform Act, Partnership, Forward-looking statement, U.S. Securities and Exchange Commission, Patient, Degenerative disease, Clinical trial, Cancer, B-cell lymphoma, Biology, Quality of life, CN, Bone marrow, Cell, Medication, Birth, G-CSF, Research, Security (finance), GLOBE, SEC, CXCR4, WHIM, Immune system, Severe congenital neutropenia, Cyclic neutropenia, Dentistry, Pharmaceutical industry

In addition, we are encouraged by the results of three exploratory sub-analyses we completed assessing the potential of mavorixafor to treat chronic neutropenia as monotherapy or concurrently with G-CSF.

Key Points: 
  • In addition, we are encouraged by the results of three exploratory sub-analyses we completed assessing the potential of mavorixafor to treat chronic neutropenia as monotherapy or concurrently with G-CSF.
  • We believe these data support the further study of mavorixafors potential to enable patients to reduce or even discontinue G-CSF treatment.
  • We anticipate this amended trial to begin generating additional clinical data in the first half of 2023.
  • X4 will host an investor webinar to present and discuss the new data today from 8:00 - 9:15 am ET.

Emendo Biotherapeutics’ next generation CRISPR gene editing technologies achieve breakthrough results with allele-specific approach for ELANE-related Severe Congenital Neutropenia as presented at ASGCT Annual Meeting

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星期二, 五月 17, 2022
Biotechnology, Health, Genetics, Science, Research, Senior, Chief technology officer, Patient, Gene editing, Society, OMNI, Research, Gene, CD34, CRISPR, Program, Technology, Neutrophil, Severe congenital neutropenia, Birth defect, Cell, Research and development, CEO, Pharmaceutical industry

Significantly, the lack of off-target achieved by Emendos engineered and optimized OMNI nuclease also eliminated any translocations.

Key Points: 
  • Significantly, the lack of off-target achieved by Emendos engineered and optimized OMNI nuclease also eliminated any translocations.
  • ELANE-based Severe Congenital Neutropenia, Emendos lead indication, is a devastating disease affecting pediatric patients that until now has been incurable, said David Baram, Ph.D., President & CEO of Emendo Biotherapeutics.
  • And of course, we look forward to the discussion generated by our discoveries around Type II CRISPR nuclease classifications that promise to be ground-breaking in the field.
  • Senior members of the Emendo Biotherapeutics R&D team including Chief Technology Officer Lior Izhar, Ph.D. and Executive Vice-President Research & Development Rafi Emmanuel, Ph.D. presented Emendos research on-site at the conference.

X4 Pharmaceuticals Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Corporate Update

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星期四, 三月 17, 2022
Mutation, PID, State Administrative Expenses, Conference, Risk, Pids, Immunoglobulin M, G&A, EDT, Lymphocyte, U.S. Securities and Exchange Commission, Severe congenital neutropenia, WHIM syndrome, SEC, R rating, Private Securities Litigation Reform Act, AACR, COVID-19, Medication, Wart, Forward-looking statement, Completeness, Clinical trial, Neutrophil, Research, Cancer, Immune system, Research and development, Genetic testing, Society, Oral, CIS, Conference call, Monocyte, Therapy, ASH, Security (finance), Prevalence, Clear cell renal cell carcinoma, Achievement, Disease, GLOBE, ID, Immunodeficiency, SCN, Safety, Adult, Axitinib, R, Biology, Pharmaceutical industry, Vaccine, Lithium, Infection, Hypogammaglobulinemia, CXCR4, Patient

BOSTON, March 17, 2022 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel CXCR4-targeted small molecule therapeutics to benefit people with diseases of the immune system, today reported financial results for the fourth quarter and twelve months ended December 31, 2021, and provided a corporate update.

Key Points: 
  • Top-line data from the 4WHIM trial are expected in the fourth quarter of 2022.
  • Cash, Cash Equivalents & Restricted Cash: X4 had $83.1 million in cash, cash equivalents, and restricted cash as of December 31, 2021.
  • The company expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022.
  • X4 will host a conference call and webcast today at 8:30 am EDT to discuss financial results and business highlights.

X4 Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

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星期五, 十月 1, 2021
WHIM, Financial compensation, Employment, CXCR4, Cancer, Patient, Medication, Biology, Immunodeficiency, Severe congenital neutropenia, Research, Safety, WHIM syndrome, GLOBE, Pharmaceutical industry

BOSTON, Oct. 01, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today announced that, on September 30, 2021, the Compensation Committee of X4s Board of Directors issued inducement awards to new employees under the X4 Pharmaceuticals, Inc. 2019 Inducement Equity Incentive Plan (the 2019 Inducement Plan).

Key Points: 
  • BOSTON, Oct. 01, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today announced that, on September 30, 2021, the Compensation Committee of X4s Board of Directors issued inducement awards to new employees under the X4 Pharmaceuticals, Inc. 2019 Inducement Equity Incentive Plan (the 2019 Inducement Plan).
  • The 2019 Inducement Plan is used exclusively for the grant of equity awards to individuals who were not previously an employee of X4.
  • The inducement awards consist of options to purchase an aggregate of 95,200 shares of X4s common stock and restricted stock units covering 6,000 shares of X4s common stock.
  • These stock awards were granted as an inducement material to the new employees entering into employment with X4 in accordance with Nasdaq Listing Rule 5635(c)(4).

X4 Pharmaceuticals to Participate in Upcoming Investor Conferences

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星期三, 九月 15, 2021
Biology, CXCR4, Medication, Immunodeficiency, Immune system, Conference, WHIM, Patient, WHIM syndrome, Safety, GLOBE, Research, Cancer, Severe congenital neutropenia, Pharmaceutical industry

Live webcasts of the Oppenheimer Fall Healthcare Life Sciences & MedTech Summit and Cantor Global Healthcare Conference events will be available on the investors section of the X4 Pharmaceuticals website at www.x4pharma.com .

Key Points: 
  • Live webcasts of the Oppenheimer Fall Healthcare Life Sciences & MedTech Summit and Cantor Global Healthcare Conference events will be available on the investors section of the X4 Pharmaceuticals website at www.x4pharma.com .
  • After the live webcasts, the events will remain archived on the X4 Pharmaceuticals website for approximately 90 days.
  • The companys lead candidate, mavorixafor, is a first-in-class, small molecule antagonist of chemokine receptor CXCR4 being developed as a once-daily oral therapy.
  • X4 believes that inhibition of the CXCR4 receptor creates the potential for mavorixafor to provide therapeutic benefit across a wide variety of diseases, including primary immunodeficiencies and certain types of cancer.

X4 Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

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星期三, 九月 1, 2021
Employment, Severe congenital neutropenia, CXCR4, Cancer, Financial compensation, WHIM syndrome, GLOBE, Research, Immunodeficiency, WHIM, Biology, Medication, Safety, Patient, Pharmaceutical industry

BOSTON, Sept. 01, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today announced that, on August 31, 2021, the Compensation Committee of X4s Board of Directors issued an inducement award to new employees under the X4 Pharmaceuticals, Inc. 2019 Inducement Equity Incentive Plan (the 2019 Inducement Plan).

Key Points: 
  • BOSTON, Sept. 01, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today announced that, on August 31, 2021, the Compensation Committee of X4s Board of Directors issued an inducement award to new employees under the X4 Pharmaceuticals, Inc. 2019 Inducement Equity Incentive Plan (the 2019 Inducement Plan).
  • The 2019 Inducement Plan is used exclusively for the grant of equity awards to individuals who were not previously an employee of X4.
  • The inducement awards consist of options to purchase an aggregate of 20,350 shares of X4s common stock and were granted as an inducement material to the new employees entering into employment with X4 in accordance with Nasdaq Listing Rule 5635(c)(4).
  • The options are subject to the terms and conditions of the 2019 Inducement Plan and the terms and conditions of an award agreement covering the grant.

X4 Pharmaceuticals to Participate in Upcoming Virtual Investor Conferences

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星期三, 九月 1, 2021
Severe congenital neutropenia, CXCR4, Cancer, WHIM syndrome, GLOBE, Immune system, Research, Immunodeficiency, WHIM, Biology, Medication, Safety, Patient, Citigroup, Conference, Pharmaceutical industry

A live webcast of the H.C. Wainwright 23rd Annual Global Investment Conference fireside chat will be available on the investor section of the X4 Pharmaceuticals website at www.x4pharma.com .

Key Points: 
  • A live webcast of the H.C. Wainwright 23rd Annual Global Investment Conference fireside chat will be available on the investor section of the X4 Pharmaceuticals website at www.x4pharma.com .
  • The event will remain archived on the X4 Pharmaceuticals website for approximately 90 days.
  • The companys lead candidate, mavorixafor, is a first-in-class, small molecule antagonist of chemokine receptor CXCR4 being developed as a once-daily oral therapy.
  • X4 believes that inhibition of the CXCR4 receptor creates the potential for mavorixafor to provide therapeutic benefit across a wide variety of diseases, including primary immunodeficiencies and certain types of cancer.

X4 Pharmaceuticals to Participate at Canaccord Genuity’s Virtual 41st Annual Growth Conference

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星期三, 八月 4, 2021
Chemokine receptors, Branches of biology, CXCR4, Canaccord Genuity, CXC chemokine receptors, Genuity, HIV, Chemokine

BOSTON, Aug. 04, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases of the immune system resulting from dysfunction of the CXCR4 pathway, today announced that management will participate in a fireside chat at Canaccord Genuitys 41st Annual Growth Conference on Thursday, August 12, 2021.

Key Points: 
  • BOSTON, Aug. 04, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases of the immune system resulting from dysfunction of the CXCR4 pathway, today announced that management will participate in a fireside chat at Canaccord Genuitys 41st Annual Growth Conference on Thursday, August 12, 2021.
  • A live webcast of the fireside chat from the Canaccord Genuity 41st Annual Growth Conference will be available on the investors section of the X4 Pharmaceuticals website at www.x4pharma.com .
  • After the live webcast, the event will remain archived on the X4 Pharmaceuticals website for approximately 90 days.
  • The companys lead candidate, mavorixafor, is a first-in-class, small molecule antagonist of chemokine receptor CXCR4 being developed as a once-daily oral therapy.

X4 Pharmaceuticals Initiates Phase 1b Clinical Trial of Mavorixafor in Combination with Ibrutinib for the Treatment of Waldenström’s Macroglobulinemia (WM)

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星期一, 十二月 30, 2019
Biotechnology, Health, Pharmaceutical, Clinical trials, Oncology, Medical specialties, Organ systems, Medicine, Blood disorders, Autosomal dominant disorders, Tyrosine kinase inhibitors, Lymphoma, Waldenström's macroglobulinemia, Ibrutinib, Severe congenital neutropenia, CXCR4, Clear cell renal cell carcinoma, Waldenström’s Macroglobulinemia (WM), Mavorixafor, X4 Pharmaceuticals

This trial is being conducted as part of a collaboration with The Leukemia & Lymphoma Society (LLS) to accelerate the development of mavorixafor for the treatment of WM.

Key Points: 
  • This trial is being conducted as part of a collaboration with The Leukemia & Lymphoma Society (LLS) to accelerate the development of mavorixafor for the treatment of WM.
  • Mavorixafor is also being developed by X4 to treat Severe Congenital Neutropenia (SCN), Waldenstrms macroglobulinemia (WM), and clear cell renal cell carcinoma (ccRCC).
  • X4 is further investigating mavorixafor in a Phase 1b clinical trial for the treatment of Severe Congenital Neutropenia (SCN), and with ibrutinib for the treatment of Waldenstrms macroglobulinemia (WM).
  • The WHIM-like CXCR4(S338X) somatic mutation activates AKT and ERK, and promotes resistance to ibrutinib and other agents used in the treatment of Waldenstroms Macroglobulinemia.