– NIH investigators present collaborative interim
data of MAT2203 study at The American Society for Microbiology’s
ASM Microbe 2017 Conference –
Matinas BioPharma Holdings, Inc. (NYSE MKT:MTNB), a clinical-stage
biopharmaceutical company focused on developing innovative
anti-infectives for orphan indications, today announced that
investigators from the National Institutes of Health (“NIH”)
presented interim data from two patients enrolled in the
collaborative Phase 2a clinical study of Matinas’ lead
anti-infective product candidate MAT2203 for the treatment of
chronic refractory mucocutaneous candidiasis (“CMC”) infection, at
The American Society for Microbiology’s ASM Microbe 2017 Conference
being held June 1–5 in New Orleans, LA. Two out of the two patients
with long-standing azole resistant mucocutaneous candidiasis met
the primary endpoint of the Phase 2a study, achieving ≥ 50%
clinical response with treatment of MAT2203. MAT2203 was well
tolerated with majority of adverse events observed being mild in
severity and unrelated to study drug.
Matinas management will host a conference call
and live webcast for investors, analysts and other interested
parties to review the interim data on Monday, June 5, 2017 at 8:30
a.m. ET (details below).
MAT2203 is the Company’s orally-administered,
encochleated formulation of the broad spectrum fungicidal
medication amphotericin B. Matinas BioPharma’s proprietary
lipid-crystal nano-particle formulation of amphotericin B has a
novel mechanism of absorption and distribution to infected tissues
and has the potential to transform the way this potent fungicidal
agent is administered and used in clinical practice.
The abstract entitled, “Oral Encochleated
Amphotericin B (CAMB) in the Treatment of Chronic Azole Resistant
Mucocutaneous Candidiasis,” was presented today in poster session
focused on new antifungal agents, by Alexandra Freeman, M.D., of
the National Institute of Allergy and Infectious Diseases (NIAID)
Laboratory of Clinical Infectious Diseases, Principal Investigator
of the Phase 2a study sponsored by Matinas BioPharma. To access the
poster, click here.
“We are incredibly pleased with the interim
safety and efficacy results of this Phase 2a study of MAT2203.
While we understand the results are representative of just two
patients, these patients are difficult to treat because of their
severe underlying immunocompromising condition. With the
statistical success hurdle that was prospectively set at a 20%
patient-response probability, seeing a clinical response in two out
of two patients brings us very close to the 3 out of 16 clinical
responders required for the study to meet its primary endpoint, “
said Roelof Rongen, Chief Executive Officer.
“We believe that with these interim results, we
have made a significant step toward establishing proof-of-concept
for treating fungal infections in immunocompromised patients, and
importantly have begun to demonstrate in a clinical setting the
depth and breadth of our cochleate technology to deliver
amphotericin B orally as a chronic treatment. We are
encouraged by these initial results and believe they have the
potential to be predictive of the completed study outcome, and look
forward to continuing the study to further understand the potential
of MAT2203. We are extremely grateful to the patients for their
participation and to the NIH for conducting this study,” commented
Raphael J. Mannino, Ph.D., Matinas BioPharma’s Chief Scientific
Officer.
The interim data presented showed that the first
two patients in this study, both with Job’s Syndrome and
long-standing azole resistant mucocutaneous candidiasis for >20
years, achieved ≥ 50% clinical response after 14 days of treatment
at an efficacious orally administered dosage of MAT2203, thus
meeting the primary endpoint. Job’s Syndrome, also known as
Autosomal dominant Hyper IgE Syndrome (AD-HIES), is a hereditary
condition rendering the patients severely immunocompromised and
exposes them to chronic infections, including CMC, often involving
the oral, esophageal and vaginal mucosas and nails. Both patients
suffered from chronic azole resistant oral CMC (or oral thrush) as
their primary infection and had an inadequate response to current
oral antifungal therapy. Clinical efficacy criteria were met at
400mg and 200mg of MAT2203 oral suspension twice daily in patient
01 and patient 02, respectively, with improvement upon exam in
clinical symptoms and semi-quantitative fungal cultures of the oral
thrush condition. The clinical severity score for oral thrush
(composed of oral pain, burning, dysphagia, odynophagia, and
presence of plaques) decreased by 57% for patient 01 and by 85% for
patient 02, with corresponding reduction in fungal culture counts.
Both patients reported meaningful quality-of-life
improvements.
MAT2203 was generally well tolerated and there
were no signs of nephrotoxicity, hypokalemia or hepatoxicity
(measured by ALT and AST). Indicators of kidney and liver toxicity
remained within normal limits throughout a 6-8 week treatment
period.
As expected, oral thrush promptly returned after
stopping treatment with MAT2203. Therefore, Matinas’ preliminary
clinical data indicate that MAT2203 is promising as an oral
systemically-absorbed broad-based antifungal without the toxicity
of parenteral amphotericin B.
Both of the patients have elected to enroll in
the open-label extension study.
“These results are very encouraging. I have
patients like these individuals in my practice and they are very
difficult to treat because they are immunocompromised and often
resistant to azoles. There is a need for new drugs that can
overcome resistance and be administered safely for extended
periods. Seeing the data from the first two patients, I am
optimistic that MAT2203 can be administered safely for long-periods
and can treat resistant mucosal disease. Moving forward, I
would like to see MAT2203 studied for the treatment and prevention
of invasive fungal disease,” commented Peter G. Pappas, MD, FACP,
William E. Dismukes Professor of Medicine in the Division of
Infectious Diseases in the Department of Medicine at the University
of Alabama at Birmingham and the Principal Investigator for the
Mycoses Study Group Education and Research Consortium.
The Phase 2a study is being conducted at the
National Institutes of Health Clinical Center in Bethesda, MD,
under the direction of Dr. Freeman. The ongoing open-label,
dose-titration study is designed to assess the efficacy, safety,
tolerability and pharmacokinetics of MAT2203 in predominantly
hereditary immunodeficient patients with a recurrent or chronic
mucocutaneous candidiasis infection (esophageal, oropharyngeal,
vaginal) who are refractory or intolerant to standard
non-intravenous therapies. The study will enroll up to 16 patients,
and study endpoint in the statistical analysis plan is defined as a
response in three or more patients. The study includes 14-day
dosing and evaluation periods. Depending on clinical response
during each treatment period, investigators will have the ability
to continue the effective dose for 28 total days or increase the
dose of MAT2203 up to two times and extend treatment to a maximum
of 54 days. In March 2017, the Company announced that the
Institutional Review Board of the NIAID, NIH granted approval for a
6-month open-label safety extension of the Phase 2a study.
The U.S. Food and Drug Administration (FDA) has
designated MAT2203 as a Qualified Infectious Disease Product (QIDP)
with Fast Track status for the treatment of invasive candidiasis,
aspergillus, and prophylaxis (prevention) of invasive fungal
infections in patients of immunosuppressive therapy. MAT2203 is
also being explored for treatment of additional infections
including cryptococcal meningoencephalitis, and is being developed
to be eligible for Orphan Drug designations in various
indications.
Conference Call and Webcast Information
Matinas will host a conference call and live
webcast for investors, analysts and other interested parties on
Monday, June 5, 2017 at 8:30 am ET to provide an update and
overview for the clinical development of MAT2203. Joining Matinas
management on the call will be Dr. Edmund C. Tramont, MD, National
Institute of Health, Allergy and Infectious Diseases, Associate
Director for Special Projects, Former Director, Division of AIDS
and Co-Investigator of the study.
The conference call and live webcast will be
accompanied by presentation slides. To participate in the call,
please dial (877) 407-5976 (domestic) or (412) 902-0031
(international). The live webcast and accompanying slides will be
accessible on the Events page of the Investors section of Matinas’
website, www.matinasbiopharma.com, and will be archived for 60
days.
About Mucocutaneous Candidiasis
Mucocutaneous candidiasis is a group of
syndromes resulting in infections of the skin, nails and mucous
membranes. These infections are caused by opportunistic candida
yeast, the most common cause of fungal infections worldwide. There
are more than 20 species of candida that can cause infection in
humans, the most common of which is candida albicans. A variety of
disorders including endocrine dysfunctions, hereditary
immune-system disorders, alopecia, vitiligo, malabsorption
syndromes, neoplasms and other infections may also occur in
patients with chronic reoccurring mucocutaneous candidiasis and
autoimmune disorders. Current anti-fungal treatment management
options are limited and relapse is common following discontinuation
of certain therapies. In addition, the increasing resistance of
certain strains to standard antifungal treatments is a growing
concern.
About MAT2203
MAT2203 is an orally-administered, encochleated
formulation of amphotericin B (a broad spectrum fungicidal agent).
Little to no clinical resistance has been reported to date with
amphotericin B as compared to the rapidly emerging drug resistance
seen in other antifungal therapies. Currently, IV-only administered
amphotericin B is the only broad spectrum fungicidal available but
its IV-delivery results in significant treatment-limiting side
effects, including nephrotoxicity. The ability to provide
amphotericin B orally using our proprietary and novel oral
formulation may offer a new and promising alternative for patients
and doctors. Currently, there are two Phase 2 studies underway with
MAT2203. The first is an open-label Phase 2a
NIH/NIAID-sponsored clinical study with MAT2203 in
immunocompromised patients with refractory mucocutaneous
candidiasis. The second is a Phase 2 study of MAT2203 in patients
with vulvovaginal candidiasis (VVC). Data from both studies is
expected to be announced in June of 2017. The FDA has
designated MAT2203 as a Qualified Infectious Disease Product (QIDP)
for the treatment of invasive candidiasis and the treatment of
aspergillosis, as well as for the prevention of invasive fungal
infections due to immunosuppressive therapy. MAT2203 is also being
explored for treatment of additional anti-fungal indications and
may have the potential for Orphan Drug Designation in certain of
these indications.
About Matinas BioPharma
Matinas BioPharma is a clinical-stage
biopharmaceutical company focused on developing innovative
anti-infectives for orphan indications. The Company's proprietary,
disruptive technology utilizes lipid-crystal nano-particle
cochleates to nano-encapsulate existing drugs, making them safer,
more tolerable, less toxic and orally bioavailable.
The Company's lead anti-infective product
candidates, MAT2203 and MAT2501, position Matinas BioPharma to
become a leader in the safe and effective delivery of
anti-infective therapies utilizing its proprietary lipid-crystal
nano-particle cochleate formulation technology. For more
information, please visit www.matinasbiopharma.com and connect with
the Company on Twitter, LinkedIn, Facebook, and Google+.
Forward Looking Statements:
This release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995,
including those relating to the Company's strategic focus and the
future development of its product candidates, including MAT2203 and
MAT2501, the anticipated timing of regulatory submissions, the
anticipated timing of clinical studies, the Company’s ability to
identify and pursue development and partnership opportunities for
its products 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 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 maintain and derive benefit from the Qualified
Infectious Disease Product (QIDP), Orphan and/or Fast Track
designations for MAT2203 and MAT2501, which does not change the
standards for regulatory approval or guarantee regulatory approval
on an expedited basis, or at all; 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 Contact
Jenene Thomas
Jenene Thomas Communications, LLC
Phone: +1 (908) 938-1475
Email: jenene@jenenethomascommunications.com