SAN
DIEGO, Oct. 4, 2022 /PRNewswire/ -- Inhibrx, Inc.
(Nasdaq: INBX), a clinical-stage biopharmaceutical company
dedicated to the development of therapeutics for oncology and rare
diseases, announced today that, based on discussions with the U.S.
Food and Drug Administration (FDA), there is potential to pursue an
accelerated approval in the U.S. for INBRX-101, an optimized
recombinant human AAT-Fc fusion protein, in patients with emphysema
due to alpha-1 antitrypsin deficiency (AATD) using functional
alpha-1 antitrypsin (AAT) serum levels as the surrogate endpoint.
Inhibrx also announced the detection of INBRX-101 in the
bronchoalveolar lavage fluid (BALF) samples from all AATD patients
tested in the Phase 1 study.
Inhibrx plans to initiate in the first quarter of 2023 a
potential registration-enabling clinical trial using functional AAT
as a surrogate endpoint with the intent to submit for regulatory
approval under the FDA's Accelerated Approval Program. The FDA
expressed support to collaborate and work with Inhibrx to address
the regulatory challenges associated with AATD drug development.
The FDA emphasized the importance of being able to demonstrate
INBRX-101's ability to maintain a trough level within the normal
range of AAT in healthy individuals. Based on data from the
completed Phase 1 study of INBRX-101, the dosing of INBRX-101 every
three or four weeks in patients with AATD is predicted to maintain
patients above the lower threshold of the normal range and achieve
an average level of functional AAT that approximates that of
healthy non-deficient (MM genotype) adults.
The FDA also requested additional data on the correlation
between functional AAT levels and the clinical benefit in AATD to
further support serum AAT levels as a surrogate endpoint that is
reasonably likely to predict clinical benefit, the prerequisite for
accelerated approval. Inhibrx intends to collaborate with experts
in the field and the Alpha-1 Foundation to illustrate, based on
existing registry, health records and published data, that
increased functional AAT levels are reasonably likely to predict
clinical benefit in AATD patients.
The accelerated approval pathway can be used for a drug intended
for a serious or life-threatening illness that provides a
meaningful therapeutic advantage over existing treatments.
Accelerated approval can be based on the drug's effect on a
surrogate endpoint that is reasonably likely to predict a clinical
benefit to patients, with a required post-approval trial(s) to
verify that the drug provides the expected clinical benefit.
Independent of FDA discussions, Inhibrx also completed analysis
and reported BALF data demonstrating the post-dose presence of
INBRX-101 in every patient and in all three lobes of the lung
collected from each of these patients. This data showed emerging
evidence of a dose dependent increase of INBRX-101 lung exposure.
These BALF samples were from eleven AATD patients in the 80mg/kg
and 120mg/kg multiple ascending dose cohorts of the Phase 1 study
and were analyzed using a proprietary and validated mass
spectrometry assay developed by Inhibrx to detect INBRX-101
specifically.
Additionally, on October 3, 2022,
the Company amended the milestone terms of the last remaining
tranche under its loan and security agreement with Oxford Finance
LLC to provide for the funding of $30.0
million upon the announcement of the regulatory path for
INBRX-101 rather than upon the initiation of a potential
registration-enabling clinical trial of INBRX-101. The Company has
thirty days from this announcement to initiate this draw.
The Company will host a live webcast presentation today at
5:30 a.m. PT to further discuss the
regulatory path and to provide an update on the clinical
pipeline.
About the Conference
Call
Investors may join via the web:
https://app.webinar.net/8GArp0rQd3z or may listen to the call
by dialing (1-877-870-4263). Please refer to Inhibrx, Inc. or
confirmation code 10171898 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
alpha-1 antitrypsin, or AAT, found in healthy individuals with the
potential for a less frequent dosing interval compared to the
weekly infusion interval of the currently available plasma-derived
AAT therapies.
Alpha-1 antitrypsin deficiency, or 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. Augmentation
therapy with plasma-derived AAT is the current standard of care but
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.
Data from the Phase 1 multiple ascending dose study of INBRX-101
at 40, 80 and 120 mg/kg IV every three weeks, showed the expected
accumulation of functional AAT levels and the ability to achieve
fully normal functional AAT levels in severely deficient AATD
patients. Based on PK modeling, accumulation is expected to
continue following subsequent doses and reach steady state after a
total of approximately five to six consecutive doses, administered
every three or four weeks.
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.
In March 2022, the FDA granted
orphan-drug designation for INBRX-101 for the treatment of
AATD.
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 FDA, including any potential accelerated regulatory pathway,
evaluations and observations of FDA discussions, statements and
beliefs regarding the current standard of care for AAT and the
sustainability of current plasma collection practices, future
clinical development, application and dosage of INBRX-101 and any
presumption of or implied presumption of positive results from
pre-clinical studies or Phase 1 clinical trials or later clinical
trials. 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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