Regulatory News:
MaaT Pharma (EURONEXT: MAAT – the “Company”), a French
clinical-stage biotech and a pioneer in the development of
Microbiome Ecosystem TherapiesTM (MET) dedicated to improving
survival outcomes for patients with cancer, provides a
corporate update highlighting the progress, adjustments to clinical
programs and key development milestones expected in 2023.
“MaaT Pharma has delivered important clinical milestones during
2022 including the expansion of its development pipeline with the
launch of two clinical trials for its lead drug candidate, MaaT013
in hemato-oncology (Phase 3) and in immuno-oncology (Phase 2a). The
year 2022 also marks an important milestone for the global
microbiome industry with the regulatory approvals of the first
microbiome-based drugs by the FDA in the U.S. and by the TGA in
Australia,” stated Hervé Affagard, CEO and co-founder of
MaaT Pharma. “Despite difficult market conditions, we continue
to advance our pioneering science and technology and we remain
focused on delivering new therapeutic solutions to the benefit of
patients with severe conditions as well as building value for our
shareholders. 2023 will be a year with important inflection points
and will require us to act decisively to reach our objectives. We
thank our investors for their support throughout this year, and
their confidence in our ability to achieve the next level of
corporate development.”
Clinical development
Hemato-oncology
MaaT013, a full-ecosystem, standardized, pooled-donor native
Microbiome Ecosystem Therapy for acute, hospital use (enema
administration)
MaaT013 for the treatment of acute Graft-versus-Host Disease
(aGvHD): MaaT013 has received an Orphan Drug Designation by
both the U.S. Food and Drug Administration (FDA) and the European
Medical Agency (EMA).
- MaaT013 is currently being evaluated in Europe in an ongoing
international multicenter open-label, single arm, pivotal Phase 3
trial (ARES) in 75 patients with steroid-resistant gastrointestinal
aGvHD. The Company has received full regulatory approvals in the
following countries, which is more than initially envisioned:
Austria, Belgium, France, Germany, Italy, and Spain. A safety and
efficacy data review will be conducted by an independent data
safety and monitoring board (DSMB) after enrollment of half of the
patients in the study. The review is expected in the first half of
2023 and the Company will communicate the DSMB’s recommendations
following the review.
- The initiation of clinical trials following the Investigational
New Drug (IND) application for MaaT013 in the United States (U.S.)
will depend on the outcome of ongoing exchanges with the FDA in
response to the August 2022 continued clinical hold letter. The
most recent communication received by the Company from the FDA
stated that the complete response provided by the Company in
response to the letter received in August 2022, was still under
review. Feedback could be expected in the early part of 2023 and
the Company will update investors accordingly. The Company
understands that the FDA’s Center for Biologics Evaluation and
Research (CBER) that currently hosts all microbiome-based drug
candidates, continues to prioritize their work to advance the
nation’s response to the COVID-19 public health emergency, which is
creating additional delays. Despite this, the Company remains
focused on achieving regulatory approval for MaaT013 in the U.S.
and providing a therapeutic solution for patients in the
country.
- MaaT013 has already been successfully evaluated in a Phase 2
clinical trial in patients with steroid-resistant grade III-IV
gastro-intestinal (GI) aGvHD (HERACLES) as well as in an ongoing
compassionate use program (EAP) in France in patients with Grade
II-IV GI-aGvHD having failed previous therapies, with promising
results presented at the 2022 American Society of Hematology (ASH)
conference in an oral presentation and in a poster at the 2022
edition of the International Human Microbiome Consortium (IHMC).
The compelling data demonstrates that the pooled microbiota product
is well tolerated and shows a good safety profile in a fragile
patient population. Efficacy of MaaT013 correlates with engraftment
of the product’s microbial species in the gut and clinical response
to MaaT013 translates to increased overall survival in patients
with aGvHD.
MaaT033, a donor-derived, standardized, high-richness,
high-diversity Microbiome Ecosystem Therapy for oral
administration, currently being developed as an adjunctive and
maintenance therapy to improve overall survival in patients
receiving HSCT1.
MaaT033 for the improved survival of patients with
hematological malignancies receiving allo-HSCT
- Based on promising results following the Phase 1b trial
presented at the 2022 edition of the ASH annual meeting, and
learnings from this study, along with an in-depth review of the
Protocol Assistance with the EMA, and discussions with potential
partners showing an interest in the license and commercialization
of MaaT033 in this therapeutic area, the Company has optimized and
adjusted the protocol for the Phase 2b trial of MaaT033 (PHOEBUS).
In this context and given the strategic importance of the trial,
the Company has decided to take over the sponsorship of the
clinical trial in Europe. At the time of the IPO, it was
anticipated that AP-HP2 would sponsor the trial in France as an
Investigator-sponsored trial. The study is expected to start in Q2
2023 and would now be conducted in Europe in accordance with EMA
recommendations. The number of patients enrolled in the study would
expand from 341 to 387 and the number of sites would be increased
from 20 to 56, enabling the recruitment period to be reduced from
36 to 24 months.
- While the primary endpoint of the study is to evaluate the
efficacy of MaaT033 in improving overall survival at 12 months,
secondary endpoints would include the evaluation of safety and
tolerability before and after allo-HSCT, and the evaluation of the
engraftment of beneficial microbial species from MaaT033 and the
activity in preventing allo-HSCT complications (infections,
GvHD).
- Additional readouts have been included throughout the 24-month
recruitment period, such as interim safety analysis with stopping
rules3 when the trial has enrolled and randomized, respectively, 60
and 120 patients.
- The expected timelines including the overall survival endpoint
(data to be expected in H1 2026) announced by MaaT Pharma during
the IPO should be confirmed based on the adjustments mentioned
above.
- In parallel to starting the trial in Europe, the Company plans
to discuss this program with the FDA upon conclusion of ongoing
dialogues with the Agency regarding its pooling technology.
Immuno-oncology – Clinical and nonclinical programs
MaaT013 for the improvement of patients’ response to Immune
Checkpoint Inhibitors (ICI) –proof of concept Phase 2a clinical
trial sponsored by AP-HP
- The proof-of-concept double blind randomized Phase 2a clinical
trial, PICASSO (n=60), initiated in April 2022, evaluating
MaaT013’s impact on the efficacy of ICI treatments (ipilimumab +
nivolumab) in patients with metastatic melanoma, remains on track
as planned.
- This trial is an Investigator-sponsored trial by AP-HP, where
MaaT Pharma supplies drug candidates and performs the microbiome
profiling of patients using its proprietary gutPrint®
platform.
- Key study endpoints after 23 weeks of treatment include
MaaT013’s safety profile and best-overall response rate vs placebo
as an add-on treatment to ipilimumab + nivolumab.
- The Company is expected to receive biological biomarker data in
H1 2023 after half of the patients have been enrolled and achieved
their evaluation 9 weeks after randomization.
- Results are expected in H2 2024, as planned.
MaaT03x, a new generation high richness, co-cultured,
designed Microbiome Ecosystem TherapyTM in oral formulation for the
increase in the response rate to ICI in patients with solid tumors
- currently in preclinical testing.
- As MaaT03x is expected to be used in large markets, MaaT Pharma
has been focused on increasing the level of readiness and decided
to enter a Phase 1b trial in Europe and the U.S. in the first half
of 2024, as opposed to the second half of 2023 as initially
announced. This is a result of changes and adjustments following
announcements of first regulatory approvals for microbiome-based
products and the Company’s work for the development of its native
products (MaaT013 & MaaT033) in Europe. MaaT Pharma is
consolidating in vivo/in vitro data, continuing product development
characterization and conducting regulatory readiness of MaaT03x.
Preclinical data are expected to be shared at scientific
conferences in 2023.
- MaaT Pharma has initiated early interactions with the EMA in
2022 including a meeting with the EMA's Innovation Task Force
(ITF). Further interactions in Europe are expected to take place in
2023. The Company is also planning early interactions with the FDA
in H1 2023 to present its MaaT03x platform and has already filed a
meeting request.
- The Company will be able to strengthen its intellectual
property (IP) portfolio in France and internationally thanks to the
co-cultured new generation products. Currently, the Company holds
the IP for 14 patent families.
Therapeutic pipeline extension
Recent studies have highlighted the importance of the
microbiota-gut-brain axis and the impact of the gut microbiome in
brain diseases. Interestingly, patients with neurodegenerative
disorders display both central nervous system (CNS) and
gastrointestinal symptoms4. In parallel, scientific research has
started to shed light on the role of the gut microbiome, linking
abnormalities to diseases such as Amyotrophic lateral sclerosis
(ALS5). The link between gut microbiota and ALS first emerged from
preclinical evidence and then from clinical observations indicating
a disease-modifying role for the gut microbiome. To date, there is
no effective treatment for ALS, a disease that leads to death
within an average of 3-5 years after diagnosis6. ALS affects nerve
cells in the brain and the spinal cord causing loss of muscle
control. Since its inception, MaaT Pharma has been committed to
restoring a microbial symbiosis in life-threatening diseases with
high unmet clinical needs. Growing evidence suggests that ALS
patients show increased inflammation in the gut with changes in the
composition of gut microbes and low levels of beneficial
bacteria.
MaaT Pharma has decided to extend its scientific research to the
management of ALS, which could pave the way for the treatment of
several neurodegenerative diseases. The Company is leveraging the
strong safety profile of its native MET products (MaaT033/MaaT013)
and their inherent product characteristics of promoting immune
modulation/anti-inflammatory properties and acting as a homeostasis
hub. The number of ALS cases is expected to increase substantially
in the developing world over the next 25 years and could reach a
total of 60,000 patients in U.S. and Europe by 20407. Every year,
5,000 new patients are diagnosed in the U.S. and in Europe and the
incidence of ALS ranges from 3 to 4 per 100,000 person-years7.
Leveraging 12 months of feasibility assessment in MaaT Pharma’s
Discovery team, the Company expects to initiate in H1 2023 a Phase
1b pilot study in ALS evaluating MaaT033 to slow down disease
progression. The study, developed with experts from the ALS network
(FILSLAN and ACT4ALS-MND) and strongly supported by the French
patient association (Tous en Selles contre la SLA), will enroll up
to 15 patients presenting initial motor deficit i.e., at least 6
months up to 24 months at the time of the screening. This pipeline
expansion to a new indication demonstrates the strong potential of
MaaT033 to be used in an acute or chronic setting as a standalone,
adjunctive and maintenance therapy. Data readout from the pilot
study is expected for the first half of 2024.
If this initial trial is successful, the Company could extend
further to other chronic diseases/ immuno-inflammatory diseases as
MaaT Pharma collects data and strengthens in-depth understanding of
the mechanism of action. Funding for the trial is already secured
and the program has been designed to minimize the risk and not
impact the Company’s lead programs in oncology. The key study
endpoints are assessment of safety and tolerability of multiple
doses of MaaT033, the assessment of gut microbiota composition
evolution and the identification of biomarkers sensitive to
treatment before considering a larger randomized controlled
efficacy study.
cGMP manufacturing facilities
MaaT Pharma is currently building its future manufacturing
facilities, in partnership with Skyepharma. The 1600m2 facility
will be able to support the needs of clinical and commercial
production of its native MET (MaaT013 & MaaT033), R&D and
clinical batches of cultured products, MaaT03x, up to 2034.
Funding is secured and the completion of the facility is
expected in mid-2023. Under the terms of the partnership agreement,
MaaT Pharma will retain the know-how of its bio-manufacturing
processes and agility (full command end-to-end manufacturing
processes), while leveraging Skyepharma’s experience in running a
cGMP facility. Having this facility is a strong competitive
advantage for a late-stage company preparing for commercial phase,
which MaaT Pharma believes will facilitate potential commercial
partnerships. The site will be the first in France, and the largest
specialized manufacturing facility for full ecosystem microbiome
therapies in Europe to date. The facility will also increase
potential synergies within the microbiome industry and contribute
to structuring the production of microbiome-based drugs. It will
provide a fully integrated manufacturing and development platform
that will allow quick and efficient product development, scale-up
and GMP processes.
Financing plans & update on cash runway
Following an in-depth review of its ongoing programs, in
particular the deferral of the clinical study of MaaT03x from 2023
to 2024 and savings on discretionary spending, the Company has
optimized and prioritized its operations, extending its cash runway
to end of Q4 2023, as compared to end of Q3 2023 as previously
announced. The Company is also evaluating options that will finance
operations and bring its late-stage products close to commercial
launch. The focus is on both dilutive and non-dilutive financing,
including business deals. The Company is confident in the
continuing and longstanding support of its historical
investors.
The Company has updated its corporate presentation, which can be
download here: https://www.maatpharma.com/investors/
About MaaT Pharma
MaaT Pharma, a clinical stage biotechnology company, has
established a complete approach to restoring patient-microbiome
symbiosis in oncology. Committed to treating cancer and
graft-versus-host disease (GvHD), a serious complication of
allogeneic stem cell transplantation, MaaT Pharma has launched, in
March 2022, an open-label, single arm Phase 3 clinical trial in
patients with acute GvHD, following the achievement of its proof of
concept in a Phase 2 trial. Its powerful discovery and analysis
platform, gutPrint®, supports the development and expansion of its
pipeline by determining novel disease targets, evaluating drug
candidates, and identifying biomarkers for microbiome-related
conditions.
The company’s Microbiome Ecosystem Therapies are produced
through a standardized cGMP manufacturing and quality control
process to safely deliver the full diversity of the microbiome, in
liquid and oral formulations. MaaT Pharma benefits from the
commitment of world-leading scientists and established
relationships with regulators to support the integration of the use
of microbiome therapies in clinical practice.
MaaT Pharma is listed on Euronext Paris (ticker: MAAT).
Forward-looking Statements
All statements other than statements of historical fact included
in this press release about future events are subject to (i) change
without notice and (ii) factors beyond the Company’s control. These
statements may include, without limitation, any statements preceded
by, followed by or including words such as “target,” “believe,”
“expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,”
“project,” “will,” “can have,” “likely,” “should,” “would,” “could”
and other words and terms of similar meaning or the negative
thereof. Forward-looking statements are subject to inherent risks
and uncertainties beyond the Company’s control that could cause the
Company’s actual results or performance to be materially different
from the expected results or performance expressed or implied by
such forward-looking statements.
1 HSCT= Hematopoietic stem-cell transplantation 2
AP-HP = Assistance Publique - Hôpitaux de Paris 3 A stopping
rule is a set of safety criteria primarily used to determine when
the study needs to be paused or halted. 4 Gebrayel et al, J
Transl Med, 2022, Singh et al, J Clin Invest. 2021 5 Rowin
et al., 2017; Nicholson et al, 2021; Blacher et al, 2019, Mazzini
et al, 2020 6
https://tousensellescontrelasla.fr/la-sla-cest-quoi/ 7
Longinetti.et.al 2019, Arthur, K., Calvo, A., Price, T. et al., US
Centers for disease control and prevention - National ALS Registry
2016
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version on businesswire.com: https://www.businesswire.com/news/home/20230124005830/en/
MaaT Pharma – Investor Relations Hervé AFFAGARD
Co-Founder and CEO Siân CROUZET, COO/ CFO +33 4 28 29 14 00
invest@maat-pharma.com
MaaT Pharma – Media Relations Pauline RICHAUD Senior PR
& Corporate Communications Manager +33 6 14 06 45 92
media@maat-pharma.com
Trophic Communications – Corporate and Medical
Communications Gretchen SCHWEITZER or Charlotte SPITZ +49 171
351 27 33 maat@trophic.eu
Maat Pharma (EU:MAAT)
過去 株価チャート
から 11 2023 まで 12 2023
Maat Pharma (EU:MAAT)
過去 株価チャート
から 12 2022 まで 12 2023
