Vertex Announces Inaxaplin (VX-147) Granted Breakthrough Therapy Designation by U.S. FDA and Priority Medicines (PRIME) Designation by the EMA
June 08 2022 - 8:00AM
Business Wire
- Vertex granted nine Breakthrough Therapy
Designations and three PRIME designations across its pipeline
programs to date –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced the U.S. Food and Drug Administration (FDA) has granted
inaxaplin (VX-147) Breakthrough Therapy Designation for
APOL1-mediated focal segmental glomerulosclerosis (FSGS) and the
European Medicines Agency (EMA) has granted inaxaplin Priority
Medicines (PRIME) designation for APOL1-mediated chronic kidney
disease (AMKD). Inaxaplin is the first investigational therapy
aimed at treating the underlying cause of AMKD.
The FDA's Breakthrough Therapy Designation is intended to
expedite development and review of medicines that aim to address a
serious condition with preliminary clinical evidence indicating
that the drug may demonstrate substantial improvement over existing
treatments on one or more clinically significant endpoints. The
Breakthrough Therapy Designation was granted based on the Phase 2
clinical study of inaxaplin in patients with APOL1-mediated FSGS, a
form of AMKD.
The EMA’s PRIME designation is a regulatory mechanism that
provides early and proactive support to developers of promising
medicines, to optimize the generation of robust data and enable
accelerated assessment so these medicines can potentially reach
patients faster. The goal of PRIME is to help patients benefit as
early as possible from innovative new therapies that have the
potential to significantly address an unmet medical need. PRIME
designation was granted based on clinical proof-of-concept data
from Vertex’s Phase 2 study of inaxaplin in APOL1-mediated FSGS.
Inaxaplin is only the second nephrology product to be granted PRIME
designation.
Vertex now holds three of the approximately 70 non-oncological
PRIME designations granted to date, including its two PRIME
designations for exagamglogene autotemcel (exa-cel), formerly known
as CTX001, one for transfusion-dependent beta thalassemia and one
for sickle cell disease. In the US, this is the ninth breakthrough
therapy designation granted to Vertex across its portfolio
programs.
About the Inaxaplin (VX-147) Pivotal Program A
randomized, double-blind, placebo-controlled Phase 2/3 adaptive
study is ongoing and will first evaluate two doses of inaxaplin for
12 weeks to select a dose for Phase 3 and subsequently evaluate the
efficacy and safety of the single, selected dose in the Phase 3
portion of the study.
Patients aged 18 to 60 years, with two APOL1 mutations, urine
protein to creatinine ratio (UPCR) ≥0.7 g/g to <10 g/g,
estimated glomerular filtration rate (eGFR) ≥25 to <75
mL/min/1.73m2 and on stable doses of standard of care medications
are eligible to enroll. Approximately 66 patients are planned to be
enrolled in the Phase 2 dose-ranging portion of the study, and
approximately 400 additional patients are planned to be enrolled in
the Phase 3 portion of the study.
The primary efficacy endpoint for the final analysis is eGFR
slope in patients receiving the inaxaplin selected dose compared to
placebo. The secondary efficacy endpoint is time to composite
clinical outcome, which will also be assessed at the final analysis
and is defined as a sustained decline of ≥30% from baseline in
eGFR, the onset of end-stage kidney disease (i.e., maintenance
dialysis for ≥28 days, kidney transplantation, or a sustained eGFR
of <15 mL/min/1.73 m2), or death. The final study analysis will
occur when subjects have at least two years of eGFR data and when
approximately 187 composite clinical outcomes have occurred.
The study is also designed to have a pre-planned interim
analysis at Week 48 evaluating eGFR slope, supported by a percent
change from baseline in UPCR in the inaxaplin arm versus placebo.
If positive, the interim analysis may serve as the basis for Vertex
to seek accelerated approval of inaxaplin in the U.S. for patients
with AMKD.
About APOL1-Mediated Kidney Disease APOL1-mediated kidney
disease is a form of chronic kidney disease caused by mutations in
the APOL1 gene. Approximately 100,000 people in the U.S. and Europe
have two APOL1 genetic mutations and proteinuric kidney disease.
People who inherit two mutations in the APOL1 gene have a course of
disease that is far more aggressive than in the absence of APOL1
genetic mutations. Inherited APOL1 genetic mutations cause kidney
disease through a toxic gain of function, which leads to podocyte
injury. This injury disrupts filtration, resulting in proteinuria
and rapidly progressive kidney disease. Progressive kidney disease
can result in dialysis, kidney transplant or death.
About Vertex Vertex is a global biotechnology company
that invests in scientific innovation to create transformative
medicines for people with serious diseases. The company has
multiple approved medicines that treat the underlying cause of
cystic fibrosis (CF) — a rare, life-threatening genetic disease —
and has several ongoing clinical and research programs in CF.
Beyond CF, Vertex has a robust pipeline of investigational small
molecule, cell and genetic therapies in other serious diseases
where it has deep insight into causal human biology, including
sickle cell disease, beta thalassemia, APOL1-mediated kidney
disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and
Duchenne muscular dystrophy.
Founded in 1989 in Cambridge, Mass., Vertex's global
headquarters is now located in Boston's Innovation District and its
international headquarters is in London. Additionally, the company
has research and development sites and commercial offices in North
America, Europe, Australia and Latin America. Vertex is
consistently recognized as one of the industry's top places to
work, including 12 consecutive years on Science magazine's Top
Employers list and one of the 2021 Seramount (formerly Working
Mother Media) 100 Best Companies. For company updates and to learn
more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter,
LinkedIn, YouTube and Instagram.
Special Note Regarding Forward-Looking Statements This
press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, as amended,
including, without limitation, statements regarding the expedited
development of inaxaplin resulting from the FDA’s Breakthrough
Therapy Designation and the EMA’s PRIME designation, the potential
benefits of inaxaplin, the anticipated timelines and dosing
associated with ongoing and future clinical trials, study design,
including expectations on patient enrollment, expectations
regarding efficacy endpoints, and plans for interim evaluation, and
plans for submission for regulatory approval in the U.S. While
Vertex believes the forward-looking statements contained in this
press release are accurate, these forward-looking statements
represent the company's beliefs only as of the date of this press
release and there are a number of risks and uncertainties that
could cause actual events or results to differ materially from
those expressed or implied by such forward-looking statements.
Those risks and uncertainties include, among other things, that
data from a limited number of patients may not be indicative of
final clinical trial results, that the trial may not be completed
in the expected timeframe, or at all, that data from the company's
development programs may not support registration or further
development of its compounds due to safety, efficacy, or other
reasons, and other risks listed under the heading “Risk Factors” in
Vertex's most recent annual report filed with the Securities and
Exchange Commission (SEC) and available through the company's
website at www.vrtx.com and on the SEC’s website at www.sec.gov.
You should not place undue reliance on these statements. Vertex
disclaims any obligation to update the information contained in
this press release as new information becomes available.
(VRTX-GEN)
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Vertex Pharmaceuticals Incorporated Investors:
Michael Partridge, +1 617-341-6108 or Miroslava Minkova, +1
617-341-6135
Media: mediainfo@vrtx.com or U.S.: +1 617-341-6992 or
Heather Nichols: +1 617-839-3607 or International: +44 20 3204
5275
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