Matinas BioPharma (NYSE American: MTNB), a clinical-stage
biopharmaceutical company focused on delivering groundbreaking
therapies using its lipid nanocrystal (LNC) platform delivery
technology, announces alignment with the U.S. Food and Drug
Administration (FDA) on the design of a single Phase 3 registration
trial of MAT2203 in patients with invasive aspergillosis who have
limited treatment options (the “ORALTO” trial).
The ORALTO trial design is built upon the
successful Phase 2 EnACT trial that confirmed MAT2203’s efficacy
and safety as a step-down and all-oral therapy in HIV patients with
cryptococcal meningitis, as well as the Company’s ongoing
Compassionate/Expanded Use Access Program in individuals suffering
from severe invasive fungal infections with no other treatment
options.
“We are pleased to achieve alignment with the
FDA on the design of a single Phase 3 trial to support the
registration of MAT2203 for the treatment of invasive aspergillosis
in patients with limited treatment options,” commented Dr. Theresa
Matkovits, Chief Development Officer at Matinas. “We greatly
appreciate the opportunity to work collaboratively with the FDA,
which was essential for reaching consensus on all critical elements
of the registrational path for MAT2203. Importantly, we view ORALTO
as a registration trial with a high probability of clinical success
given the ongoing positive patient clinical experience in our
Compassionate/Expanded Use Access Program, which mirrors the
patients to be enrolled in our upcoming Phase 3 registration
trial.”
“Reaching this agreement with the FDA is a major
milestone for Matinas and a testament to the dedication and
expertise of our collective team,” added Jerome D. Jabbour, Chief
Executive Officer of Matinas. “We are energized by the support and
guidance from the FDA, and believe this alignment provides the
requisite certainty to advance our partnership discussions for this
important product. We are pursuing every opportunity to secure a
partner and to advance MAT2203 into this Phase 3 registration trial
as quickly as possible.”
About ORALTO
ORALTO is a Phase 3, randomized, multicenter,
open-label, adjudicator-blinded study to evaluate the efficacy and
safety of MAT2203 as an oral step-down treatment following
treatment with AmBisome (liposomal IV-amphotericin B) compared with
the standard of care in patients with invasive aspergillosis who
have limited treatment options. The primary efficacy endpoint is
all-cause mortality at study day 42. Key secondary objectives
include:
(a) demonstration of
superiority of oral-step down treatment with MAT2203 compared with
AmBisome for treatment-related toxicities leading to changes in
treatment (i.e., dose adjustment/discontinuations or changes to
treatment regimens); (b) long-term
survival benefit of MAT2203 using all-cause mortality at study day
84;(c) evaluation of the impact of MAT2203 on
healthcare resource utilization and quality of life
impact.Enrollment is expected to include approximately 216 adults
with recently diagnosed probable or proven invasive aspergillosis
who are being treated with AmBisome due to their inability to
receive an IV mold-active azole and with limited alternative
treatment options. Following up to two days of initial treatment
with AmBisome, eligible study participants will be entered into the
study and randomized in a 2:1 ratio to receive either oral MAT2203
or continued AmBisome treatment followed by standard of care.
All study participants will receive up to 12
weeks of treatment starting from the first day of treatment with
AmBisome. It is anticipated that all study participants will be
hospitalized during the initial AmBisome treatment period. After
step-down to oral MAT2203, study participants may be discharged
from the hospital to continue treatment on an outpatient basis, as
clinically appropriate.
An independent Data Review Committee, who will
be blinded to treatment, will adjudicate primary and secondary
endpoints, including clinical, radiological, and mycological
responses. Once approximately 75% of participants are
enrolled, an independent Data Safety Monitoring Board will review
the overall pooled all-cause mortality rate in a blinded fashion to
ensure that the sample size assumptions are reasonable and that the
study is adequately powered. Should the pooled event differ
substantially from expected levels, a sample size adjustment can be
made to the trial.
ORALTO will be a global trial conducted at
approximately 65 investigator sites in the U.S., Europe, South
America, Middle East, and Asia Pacific. Enrollment is expected to
commence in the second half of 2024 and is expected to require
approximately 24 months.
About Invasive Aspergillosis in Patients
with Limited Treatment Options
Invasive aspergillosis (IA) is a serious and
life-threatening invasive fungal infection that occurs primarily in
severely immunocompromised patients with hematological malignancies
and in transplant recipients. IA has been increasing globally due
to advancements in the medical management of these patients and has
recently been recognized as a global public health concern. In
2022, the World Health Organization released their Fungal Priority
Pathogen List that designated Aspergillus fumigatus, the most
common cause of IA, to be in the Critical Priority group
(i.e., the highest perceived public health threat).
Aspergillus is also included in the FDA qualified designation list
of pathogens that pose a serious and life-threatening risk.
The one-year mortality for patients with IA has
been reported to be 41% in solid organ transplant recipients and up
to 75% in stem cell transplant recipients. Although outcomes have
improved since the development of mold-active azoles, the use of
these agents is often complicated by treatment-limiting toxicities,
drug-drug interactions, and the recent emergence of drug
resistance.
The Infectious Diseases Society of America
(IDSA) Treatment Guidelines recommend that patients with IA receive
treatment with a mold-active azole for a minimum of 6 to 12 weeks,
largely dependent on the degree and duration of immunosuppression,
site of disease, and evidence of disease. Although the mold-active
azoles are generally effective, their use requires a considerable
degree of expertise to manage toxicities and drug-drug interactions
which often limit duration of treatment.
IA caused by Aspergillus species that are
resistant to mold-active azoles has been increasing globally due to
the chronic use of azoles and the widespread use of azole
fungicides in agriculture. This emerging resistance is particularly
concerning because IA due to azole-resistant Aspergillus is a
life-threatening disease with a high mortality rate.
Patients with hematological malignancies and
transplant recipients often receive antifungal prophylaxis with a
mold-active azole to prevent infection during high-risk periods.
Recently, cases of breakthrough IA have been reported in patients
receiving antifungal prophylaxis. The reason for these apparent
failures of prophylaxis may be non-compliance, poor absorption,
drug-drug interactions, or infection with a drug-resistant
Aspergillus species.
The IDSA Treatment Guidelines recommend an
intravenously administered amphotericin B, such as AmBisome, as an
alternative for patients with IA who are unable to receive
treatment with a mold-active azole. However, IV amphotericin B can
cause nephrotoxicity and electrolyte abnormalities which usually
requires hospitalization for close monitoring and electrolyte
supplementation. Other complications of IV amphotericin B include
acute infusion reactions with dyspnea, hypoxia, chest and back
pain, IV-site phlebitis, anemia, and hepatotoxicity. As such,
treatment with IV amphotericin B for more than a few weeks is
generally not safe or practical. There are no oral antifungals
available that will enable these patients to complete the
recommended 6 to 12 weeks of treatment. Accordingly, there is a
critical unmet medical need for effective and well-tolerated oral
antifungal agents to treat these patients with IA.
MAT2203 is not yet licensed or approved anywhere
globally.
About MAT2203Matinas BioPharma
is developing MAT2203 as a potential oral broad-spectrum treatment
for invasive deadly fungal infections. Although amphotericin B is a
fungicidal agent, it is currently only available through an
intravenous route of administration, which is known to be
associated with several significant safety issues such as renal
toxicity and anemia due to very high circulating levels of
amphotericin B. MAT2203 has the potential to overcome the
significant limitations of the currently available amphotericin B
products due to its targeted oral delivery, combining comparable
fungicidal activity with targeted delivery resulting in a lower
risk of toxicity and potentially creating the ideal antifungal
agent for the treatment of invasive fungal infections. MAT2203 was
successfully evaluated in the completed Phase 2 EnACT study in
cryptococcal meningitis, meeting its primary endpoint and achieving
robust survival.
About Matinas BioPharmaMatinas
BioPharma is a biopharmaceutical company focused on delivering
groundbreaking therapies using its lipid nanocrystal (LNC) platform
delivery technology.
In addition to MAT2203, preclinical and clinical
data have demonstrated that this novel technology can potentially
provide solutions to many challenges of achieving safe and
effective intracellular delivery of both small molecules and
larger, more complex molecular cargos including small
oligonucleotides such as ASOs and siRNA. The combination of its
unique mechanism of action and flexibility with routes of
administration (including oral) positions Matinas’ LNC technology
to potentially become a preferred next-generation orally available
intracellular drug delivery platform. For more information, please
visit www.matinasbiopharma.com.
Forward-looking StatementsThis
release contains "forward-looking statements" within the meaning of
the Private Securities Litigation Reform Act of 1995, including
those relating to our business activities, our strategy and plans,
the future development of its product candidates, including
MAT2203, the Company’s ability to identify and pursue development,
licensing and partnership opportunities for its products, including
MAT2203, or platform delivery technology on favorable terms, if at
all, and the ability to obtain required regulatory approval and
other statements that are predictive in nature, that depend upon or
refer to future events or conditions. All statements other than
statements of historical fact are statements that could be
forward-looking statements. Forward-looking statements include
words such as "expects," "anticipates," "intends," "plans,"
"could," "believes," "estimates" and similar expressions. These
statements involve known and unknown risks, uncertainties and other
factors which may cause actual results to be materially different
from any future results expressed or implied by the forward-looking
statements. Forward-looking statements are subject to a number of
risks and uncertainties, including, but not limited to, our ability
to continue as a going concern, our ability to obtain additional
capital to meet our liquidity needs on acceptable terms, or at all,
including the additional capital which will be necessary to
complete the clinical trials of our product candidates; our ability
to successfully complete research and further development and
commercialization of our product candidates; the uncertainties
inherent in clinical testing; the timing, cost and uncertainty of
obtaining regulatory approvals; our ability to protect the
Company’s intellectual property; the loss of any executive officers
or key personnel or consultants; competition; changes in the
regulatory landscape or the imposition of regulations that affect
the Company’s products; and the other factors listed under "Risk
Factors" in our filings with the SEC, including Forms 10-K, 10-Q
and 8-K. Investors are cautioned not to place undue reliance on
such forward-looking statements, which speak only as of the date of
this release. Except as may be required by law, the Company does
not undertake any obligation to release publicly any revisions to
such forward-looking statements to reflect events or circumstances
after the date hereof or to reflect the occurrence of unanticipated
events. Matinas BioPharma’s product candidates are all in a
development stage and are not available for sale or use.
Investor ContactLHA Investor
RelationsJody Cain Jcain@lhai.com310-691-7100
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