X4 Pharmaceuticals Provides Business and Clinical Development Update
- Expects to commence the global Phase 3 pivotal trial of mavorixafor
for the treatment of patients with Warts, Hypogammaglobulinemia,
Infections and Myelokathexis (WHIM) syndrome in second quarter 2019
having finalized the protocol based on
FDA guidance Receives World Health Organization (WHO) approval for mavorixafor as recommended International Non-proprietary Name (INN) for X4P-001
“This has been a transformative period for X4 with our listing on
Nasdaq, finalization of our Phase 3 clinical protocol in WHIM syndrome
and the recent approval from the WHO for the use of mavorixafor as our
lead candidate name for X4P-001,” said
Business Update
In
In
X4 also recently received approval from the
Clinical Development Update
X4 has finalized the clinical trial protocol based on guidance from the
X4 continues to conduct the Phase 2 open label extension study following
the completion of the dose titration portion of the Phase 2 trial for
the treatment of patients with WHIM syndrome in
In
X4 is on track to commence a Phase 1 clinical trial of mavorixafor for
the treatment of patients with severe congenital neutropenia (SCN) in
X4 also plans to commence a multi-national Phase 1/2 clinical trial of mavorixafor in combination with ibrutinib for the treatment of patients with Waldenström macroglobulinemia in 2019. The study population will focus on patients with WHIM-like mutations in CXCR4 who are known to respond poorly to standard of care.
X4 has completed enrollment in the open label Phase 2a portion of its ongoing Phase 1/2 clinical trial of mavorixafor in combination with axitinib in clear cell renal cell carcinoma (ccRCC) patients. The Company plans to unveil progression-free survival (PFS) data as part of an anticipated abstract to be submitted for presentation at a major medical conference in the second half of 2019.
About
Forward-Looking Statements
This press release contains
forward-looking statements that involve substantial risks and
uncertainties. All statements, other than statement of historical facts,
included in this press release regarding our strategy, future
operations, and plans are forward-looking statements. Examples of such
statements include, but are not limited to, statements relating to plans
for, or progress, scope, cost, duration or results or timing for the
initiation, completion or availability of results of development of
mavorixafor (X4P-001) or any of our other product candidates or
programs, including regarding the Phase 3 clinical trial of mavorixafor
for the treatment of patients with WHIM syndrome, the target
indication(s) for development, the size, design, population, location,
conduct, objective, duration or endpoints of any clinical trial, or the
timing for initiation or completion of or reporting of results from any
clinical trial, the potential benefits of mavorixafor, or any other
product candidate or program or the commercial opportunity in any target
indication; or the potential benefits of orphan drug designation. Actual
results or events could differ materially from the plans, intentions,
expectations and projections disclosed in the forward-looking
statements. Various important factors could cause actual results or
events to differ materially from the forward-looking statements that X4
makes, including, but not limited to, the risk that trials and studies
may be delayed and may not have satisfactory outcomes, potential adverse
effects arising from the testing or use of mavorixafor or other product
candidates, the risk that costs required to develop mavorixafor or other
product candidates or to expand our operations will be higher than
anticipated and other risks described in the “Risk Factors” section of
the Registration Statement on Form S-4 filed by
View source version on businesswire.com: https://www.businesswire.com/news/home/20190401005255/en/
Source:
Investors:
Stephanie Carrington
Westwicke, an ICR
company
646-277-1282
Stephanie.Carrington@icrinc.com
Media:
Darcie Robinson
Westwicke, an ICR company
203-919-7905
Darcie.Robinson@icrinc.com