- Topline results from the Phase 1 study showed a favorable
safety and tolerability profile with no drug-related severe or
serious adverse events.
- Topline data from the multiple ascending dose cohorts of 40,
80 and 120 mg/kg demonstrated the average level ("Cavg")
of functional alpha-1 antitrypsin ("AAT") achieved by INBRX-101 was
40.4 micromolar ("µM") over the 21-day dosing interval following
the third 80 mg/kg dose.
- Functional AAT levels collected from 65 healthy individuals
with the MM genotype revealed a 5th/95th percentile range of 23 to
57 µM and a median of 38 µM.
SAN
DIEGO, May 16, 2022 /PRNewswire/ -- Inhibrx Inc.
(Nasdaq: INBX), a biotechnology company with four clinical programs
in development and an emerging pre-clinical pipeline, today
announced topline results from a Phase 1 clinical trial evaluating
the safety, pharmacokinetics ("PK") and pharmacodynamics ("PD") of
INBRX-101, an optimized recombinant human AAT-Fc fusion protein, in
patients with alpha-1 antitrypsin deficiency ("AATD").
Data from this multi-country Phase 1 study are from 31 patients
with AATD: 26 with the ZZ genotype, 3 with the SZ genotype and 2
with the MZ genotype of the SERPINA1 gene, the underlying cause of
AATD. Treatment was well tolerated with no severe or serious
adverse events related to the study drug. Drug-related adverse
events were predominantly mild and those few that were moderate in
severity were all transient and reversible, with minimal or no
symptomatic care. No safety-related or PK/PD-related signs of
neutralizing anti-drug antibodies were observed.
Dose-related increases in maximal and total INBRX-101 exposure
occurred across the entirety of the single and multiple ascending
dose ranges.
Data from the multiple ascending dose cohorts of INBRX-101 at
40, 80 and 120 mg/kg IV every three weeks showed the expected
accumulation of functional AAT levels. Based on PK modeling,
accumulation is expected to continue following subsequent doses and
reach a steady-state after a total of approximately five to six
consecutive doses, administered every three weeks.
The current standard of care, plasma-derived AAT, dosed once
weekly at 60 mg/kg, achieves a Cavg of functional AAT of
17.8 µM over the weekly dosing interval as calculated from
steady-state area under the curve ("AUC") values reported
in Stocks et al. BMC Clinical Pharmacology 2010, 10:13.
INBRX-101 achieved a mean Cavg of functional AAT of 40.4
µM over the 21-day dosing interval following the third 80 mg/kg
dose.
To date, bronchoalveolar lavage fluid samples have been
processed from two 80 mg/kg multiple ascending dose cohort
individuals and confirm the presence of INBRX-101 in the lung
fluid.
Additionally, functional AAT levels were measured in plasma
samples from 65 normal MM genotype individuals. This analysis
revealed the 5th and 95th percentiles of
functional AAT levels in the normal MM genotype individuals were 23
and 57 µM, respectively, with a median of 38 µM.
"We believe this data demonstrates the potential of INBRX-101 to
change the paradigm of treatment of AAT deficiency by maintaining
patients in the normal range of functional AAT while reducing
infusions from 52 annually to as few as 12 annually. We look
forward to working with regulators, clinicians and patients to
expedite this therapy to AAT deficient patients as rapidly as
possible," said Mark Lappe, CEO of
Inhibrx.
The Company will host a live webcast presentation today,
May 16th, at 1:30 p.m. PT to further discuss the results.
About the Conference
Call
Investors may join via the web:
https://app.webinar.net/60dmpLaBwqx or may listen to the call
by dialing (1-888-220-8451). Please refer to Inhibrx, Inc. or
confirmation code 2516861 when calling in. Following the webcast,
the presentation may be accessed through a link on the investors
section of Inhibrx's website at
https://inhibrx.investorroom.com/events-and-presentations. The
webcast will be available for 60 days following the event.
Following the presentation, Inhibrx will update its corporate
presentation within the "Investors" section of its website at
www.inhibrx.com.
About INBRX-101 and AATD
INBRX-101 is a precisely engineered recombinant human AAT-Fc
fusion protein designed to safely achieve and maintain levels of
AAT found in healthy individuals with the potential for
once-monthly dosing.
AATD is an inherited orphan disease affecting an estimated
100,000 patients in the United
States. AATD is characterized by deficient levels of the AAT
protein, which causes loss of lung tissue and function and
decreased life expectancy. Plasma-derived AAT is the current
standard of care and does not maintain patients in the normal AAT
range, requires frequent and inconvenient once-weekly IV dosing,
and relies on plasma collection practices that might not be
sustainable.
About Inhibrx, Inc.
Inhibrx is a clinical-stage biotechnology company focused on
developing a broad pipeline of novel biologic therapeutic
candidates in oncology and orphan diseases. Inhibrx utilizes
diverse methods of protein engineering to address the specific
requirements of complex target and disease biology, including its
proprietary sdAb platform. Inhibrx has collaborations with 2seventy
bio (formerly bluebird bio), Bristol-Myers Squibb and Chiesi. For
more information, please visit www.inhibrx.com.
Forward-Looking
Statements
Inhibrx cautions you that statements contained in this press
release regarding matters that are not historical facts are
forward-looking statements. These statements are based on Inhibrx's
current beliefs and expectations. These forward-looking statements
include, but are not limited to, statements regarding: Inhibrx's
and its investigators' judgments and beliefs regarding the observed
safety and efficacy to date of its therapeutic candidate,
INBRX-101, discussions with and beliefs regarding future action by
the U.S. Food and Drug Administration, statements and beliefs
regarding the current standard of care for AAT and the
sustainability of current plasma collection practices and the
potential for INBRX-101 to change the standard of care, future
clinical development, application and dosage of INBRX-101 and the
presumption that topline data will be representative of final data
and that PK modeling is an accurate predictor of PK levels on a
broader basis. Actual results may differ from those set forth in
this press release due to the risks and uncertainties inherent in
Inhibrx's business, including, without limitation, risks and
uncertainties regarding: the initiation, timing, progress and
results of its preclinical studies and clinical trials, and its
research and development programs; its ability to advance
therapeutic candidates into, and successfully complete, clinical
trials; its interpretation of initial, interim or preliminary data
from its clinical trials, including interpretations regarding
disease control and disease response; the timing or likelihood of
regulatory filings and approvals; the successful commercialization
of its therapeutic candidates, if approved; the pricing, coverage
and reimbursement of its therapeutic candidates, if approved; its
ability to utilize its technology platform to generate and advance
additional therapeutic candidates; the implementation of its
business model and strategic plans for its business and therapeutic
candidates; its ability to successfully manufacture therapeutic
candidates for clinical trials and commercial use, if approved; its
ability to contract with third-party suppliers and manufacturers
and their ability to perform adequately; the scope of protection it
is able to establish and maintain for intellectual property rights
covering its therapeutic candidates; its ability to enter into
strategic partnerships and the potential benefits of these
partnerships; its estimates regarding expenses, capital
requirements and needs for additional financing and financial
performance; its expectations regarding the impact of the COVID-19
pandemic on its business; and other risks described in Inhibrx's
filings with the U.S. Securities and Exchange Commission (the
"SEC"), including under the heading "Risk Factors" in Inhibrx's
Annual Report on Form 10-K for the year ended December 31,
2021, as filed with the SEC on February 28,
2022, as well as its Quarterly Reports on Form 10-Q, and
supplemented from time to time by its Current Reports on Form 8-K.
You are cautioned not to place undue reliance on these
forward-looking statements, which speak only as of the date hereof,
and Inhibrx undertakes no obligation to update these statements to
reflect events that occur or circumstances that exist after the
date hereof. All forward-looking statements are qualified in their
entirety by this cautionary statement, which is made under the safe
harbor provisions of the Private Securities Litigation Reform Act
of 1995. This press release contains estimates and other
statistical data made by independent parties and by Inhibrx. This
data involves a number of assumptions and limitations, and you are
cautioned not to give undue weight to such estimates.
Investor and Media Contact:
Kelly Deck, CFO
kelly@inhibrx.com
858-795-4260
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