MaaT033 / MaaT Pharma - LARVOL DELTA

A Multicentre, Randomized, Double-Blinded, Phase 2b Study Evaluating the Efficacy and Safety of MaaT033, an Oral, Pooled Microbiome Ecosystem Therapy in Patients Undergoing Allogenic Hematopoietic Cell Transplantation to Improve Overall Survival: The Phoebus Trial (ASH 2023) - P2 | "Exclusion criteria comprise non-myeloablative conditioning regimen, conventional myeloablative conditioning regimen, in-vitro T-cell depletion, alloHCT with cord blood cells, alloHCT from an unrelated donor with ≥ 3/10 HLA-mismatches, use of alemtuzumab, vedolizumab or abatacept for GvHD prophylaxis, history of chronic digestive disease...Exploratory endpoints will describe the impact of MaaT013 on immune recovery, the nutritional status of the patients, and resource utilization...The study is approved in France and Germany; expansion to other countries is planned for 2024. Study start is expected in September 2023."

Clinical • P2b data • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Oncology • Transplantation

MaaT Pharma Launches a Capital Increase of Approximately €9 Million (Businesswire) - "Preparation of the commercialization of Xervyteg and related regulatory activities in Europe during the second half of 2026, in collaboration with our commercial partner, contingent upon Xervyteg approval (potential marketing authorization (MA) could be delivered around mid-2026). Advancement of MaaT033 clinical development in Europe, currently being evaluated in an ongoing Phase 2b trial, designed to be pivotal, aimed at improving survival in patients undergoing allogeneic hematopoietic stem cell transplantation."

Financing • Acute Graft versus Host Disease • Transplantation

MaaT Pharma Announces Positive Second Safety Interim Analysis from DSMB for Phase 2b PHOEBUS Trial Evaluating MaaT033 for Patients Receiving Allo-HSCT (Businesswire) - "The DSMB reviewed unblinded safety data on 120 enrolled patients (including 60 patients randomized to receive MaaT033) and monitored patients for 90 days after allo-HSCT as per the trial protocol...Following its review, the DSMB recommended that the study continue as planned, having identified no safety concerns and no excessive mortality related to MaaT033 as of today...All assessments to date have confirmed a favorable safety profile for MaaT033, and recommended continuation of the trial without modifications....Next steps include the routine DSMB review for ongoing safety for MaaT033, conducted every six months, with the next analysis expected for the first quarter of 2026."

DSMB • P2b data • Transplantation

…MaaT033 – Exploratory trials using the MET-N platform (donor derived conducted as Investigator-Sponsored Trials (ISTs) to inform further developments (MaaT Pharma Press Release) - "This investigator-sponsored, randomized, multicenter Phase 2 trial will evaluate MaaT033 in patients with advanced non-small cell lung cancer (NSCLC), with MaaT Pharma supplying the investigational product. The trial is expected by the sponsor to start in H2 2025."

Trial status • Non Small Cell Lung Cancer

Allogenic Hematopoietic Stem Cell Transplant (allo-HSCT) - MaaT033 (Businesswire) - "The last patient enrollment in the trial is anticipated for mid-2026 while the 1-year OS results are expected in H2 2027."

Enrollment status • P2b data • Transplantation

MAAT033 TO ENSURE OPTIMAL GUT MICROBIOTA TO IMPROVE SURVIVAL IN PATIENTS? RECEIVING ALLOGENEIC HCT: PHOEBUS TRIAL (EHA 2025) - "Exclusion criteria comprise non-myeloablative and conventional myeloablative conditioning regimen in vitro T-cell depletion alloHCT with cord blood cells use of alemtuzumab vedolizumab or abatacept for GvHD prophylaxis and history of chronic digestive disease.Study recruitment is currently ongoing in France Germany Spain Belgium The Netherlands and the UK. The PHOEBUS trial explores for the first time the potential of a full microbiota ecosystem therapy to enhance survival in alloHCT recipients. Recruitment is ongoing and preliminary safety assessments are favorable."

Clinical • Gut Microbiota • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Oncology

IASO: Safety and Gut Microbiota Analysis of an Oral Microbiotherapy in Patients With Amyotrophic Lateral Sclerosis (clinicaltrials.gov) - P1 | N=15 | Completed | Sponsor: MaaT Pharma | Active, not recruiting ➔ Completed | Trial completion date: Dec 2024 ➔ Jul 2024

Trial completion • Trial completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

IMMUNOLIFE2: Oral Pooled Fecal Microbiotherapy (MaaT033) Concomitant to Cemiplimab Versus Best Investigator's Choice in Patients With Resistance to Treatment Due to Antibiotics Uptake With Advanced Non-small Cell Lung Cancer (clinicaltrials.gov) - P2 | N=162 | Not yet recruiting | Sponsor: Gustave Roussy, Cancer Campus, Grand Paris

New P2 trial • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor

MaaT Pharma To Present Updated Data for MaaT013 in Early Access Program at the European Hematology Association (EHA) Annual Congress (Businesswire) - "Oral presentation will highlight updated data in Early Access Program (EAP) for 173 patients with acute Graft-vs-Host Disease (aGvHD) treated with MaaT013. Poster presentation selected for ongoing Phase 2b trial of MaaT033 in patients receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT)"

Clinical data • Clinical protocol • Acute Graft versus Host Disease

MaaT Pharma Announces Promising Final Data Readout for Phase 1b Evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS) (Businesswire) - P1 | N=15 | NCT05889572 | Sponsor: MaaT Pharma | "An external Scientific Advisory Committee took place at the end of March 2025 to review the full data. Below are the key takeaways from the Committee’s review: MaaT033 demonstrated a favorable safety and tolerability profile, supported by biomarker and microbiome analyses. A rapid and sustained engraftment of bacterial species from MaaT033 was observed, mostly occurring within the first month and maintained during the 1-month follow-up period. A slower rate of disease progression (based on ALSFRS-R slopes) was noted to be interpreted with caution given the short follow up, limited sample size and single-arm Phase 1b design."

P1 data • Amyotrophic Lateral Sclerosis

The Capital Increase will enable the Company to pursue its developments plan and confirm its potential for being a key player in hemato-oncology. (Businesswire) - "...the Company is targeting to submit its Market Authorization Application for MaaT013 in aGvHD with the European agency in June 2025, with a potential approval expected in H2 2026; the Company is also preparing for a commercial launch in Europe, based on compelling Phase 3 data released in January 2026...in the US, the Company is preparing for the launch of a dedicated US Phase 3 trial in aGvHD...in view of a late 2026 commercial launch, if approved...the Company will continue the ongoing allo-HSCT Phase 2b study 'PHOEBUS' (1yr-OS read out expected in late 2027)."

EMA approval • EMA filing • Launch Europe • Launch US • P2b data • Acute Graft versus Host Disease • Transplantation

MaaT Pharma Announces Positive Safety Interim Analysis from DSMB for Phase 2b Trial Evaluating MaaT033 for Patients Receiving Allo-HSCT (Businesswire) - P2b | N=387 | PHOEBUS (NCT05762211) | Sponsor: MaaT Pharma | "MAAT...announced the positive outcome of a key DSMB safety interim analysis for the Phase 2b trial PHOEBUS, the world’s largest randomized controlled trial evaluating microbiome therapy in oncology to date....As a result of their unblinded analysis, the DSMB recommended the trial to proceed as planned, showing no excessive mortality related to MaaT033 as of today. This additional positive outcome further reinforces MaaT033’s safety profile and supports MaaT033’s integration in the allo-HSCT setting without significant risks of severe adverse events....The next DSMB unblinded interim analysis with mortality monitoring is scheduled for Q3 2025 at the 120-patient mark. The routine DSMB review for ongoing safety, conducted every six months, is also expected for Q3 2025."

DSMB • P2b data • Transplantation

Gut microbiota restoration with oral pooled fecal microbiotherapy after intensive chemotherapy: the phase Ib CIMON trial. (PubMed, Blood Adv) - P1 | "The purpose of this dose-ranging phase I study (CIMON) was to evaluate the first-in-man use of MaaT033, a pooled, allogeneic, lyophilized, and standardized fecal microbiotherapeutic product, formulated as a delayed-release capsule for oral administration...Moreover, inflammatory markers (C-reactive protein, interleukin-6) decrease with treatment, while short-chain fatty acids increase over time. A randomized, placebo-controlled phase IIb trial, in recipients of alloHCT patients in ongoing."

Journal • P1 data • Transplantation • CRP • IL6

MaaT Pharma Announces Positive Second DSMB Review of Ongoing Phase 2b Clinical Trial Evaluating MaaT033 for Patients Receiving Allo-HSCT (Businesswire) - "MaaT Pharma...announced that the DSMB completed its second safety assessment of the Phase 2b trial PHOEBUS, the largest randomized controlled trial assessing microbiome therapy in oncology to date, and recommended continuation of the trial without modification. The routine DSMB, which convenes every six months, is composed of 5 independent experts, reviewed safety data on 59 patients (cutoff date as of October 31st, 2024) and concluded that MaaT033 is well tolerated and has an acceptable safety profile....Upcoming milestones include the routine DSMB review for ongoing safety, conducted every six months, as well as a DSMB assessing mortality imbalance between arms, respectively at 60 patients (expected in Q1 2025) and at 120 patients (expected in Q3 2025), after 90 days of follow-up post Allo-HSCT."

DSMB • Transplantation

Oral Pooled Fecal Microbiotherapy to Prevent Allogeneic Hematopoietic Cell Transplantation Complications (PHOEBUS Trial) (clinicaltrials.gov) - P2 | N=387 | Recruiting | Sponsor: MaaT Pharma | Phase classification: P2b ➔ P2

Phase classification • Hematological Malignancies • Transplantation

MaaT Pharma Announces Positive Phase 1b Results, Meeting Primary Endpoint in the Evaluation of MaaT033 in Amyotrophic Lateral Sclerosis (ALS) (Businesswire) - P1 | N=15 | IASO (NCT05889572) | Sponsor: MaaT Pharma | "MaaT Pharma...announced that the exploratory single-arm, open-label Phase 1b clinical trial named IASO (NCT05889572) evaluating MaaT033 in ALS has met its primary endpoint assessing the safety and tolerability of MaaT033 with multiple doses. The independent Data Safety and Monitoring Board (DSMB) concluded that MaaT033 showed good safety and tolerability in ALS patients when dosed for two months. Preliminary microbiome analysis confirms the successful engraftment of MaaT033, the Company’s oral capsule, outlining further the safety and tolerability outcome...Additional study endpoints are expected to be analysed in the coming months. Based on the current evidence from the Phase 1b IASO study, the DSMB supports proceeding to Phase 2. MaaT Pharma plans to determine the next steps based on a comprehensive analysis of the study’s overall data, expected in early 2025."

DSMB • P1 data • Amyotrophic Lateral Sclerosis • CNS Disorders

Baseline and safety data from IASO, a pioneering open-label phase 1b pilot trial assessing the safety, tolerability and gut microbiota Analysis of an Oral microbiotherapy in amyotrophic lateral sclerosis (ALS-MND 2024) - P1 | "A total of 18 patients meeting the Revised El Escorial criteria for possible, probable, laboratory-supported probable or definite ALS were screened for eligibility, of whom 15 qualified to initiate the bowel preparation (Baseline) and 14 initiated MaaT033 treatment in 2 ALS expert centers in France. The main baseline demographic and disease characteristics are: 64% male, 36% female, median age (min; max) of 59 (34; 74) years old, 93% limb onset. An initial Data Safety Monitoring Board found that MaaT033 was generally well tolerated with a good safety profile based on the clinical data from the first 8 patients who received at least one dose of MaaT033."

Clinical • Gut Microbiota • P1 data • Amyotrophic Lateral Sclerosis • CNS Disorders • Inflammation

MaaT Pharma Publishes its Half Year 2024 Results and Provides a Business Update (Businesswire) - "MaaT033:...MaaT033...will be tested as a concomitant treatment to cemiplimab (Regeneron), an anti-PD1 therapy, to assess the potential increase in response rate in patients having received antibiotics. This randomized multicenter Phase 2 clinical trial will include advanced non-small cell lung cancer (NSCLC) patients. The related costs for MaaT Pharma are limited to clinical product supply in line with previous cash projections. The trial is expected to start in H1 2025."

New P2 trial • Non Small Cell Lung Cancer

MaaT Pharma To Present and Participate in Investor and Medical Conferences in September (Businesswire) - "MaaT Pharma...announced that it will present two posters at the Society of Hematologic Oncology Annual Meeting taking place September 4-7, 2024, in Houston, Texas, USA."

P2 data • P3 data • Acute Graft versus Host Disease • Graft versus Host Disease • Immunology • Transplantation

A Multicentre, Randomized, Double-Blinded, Phase 2b Study Evaluating The Efficacy And Safety Of Maat033, an Oral, Pooled Microbiome Ecosystem Therapy In Patients Undergoing Allogenic Hematopoietic Cell Transplantation to Improve Overall Survival: the PHOEBUS trial (SOHO 2024) - P2b | "Exclusion criteria comprise nonmyeloablative conditioning regimen; conventional myeloablative conditioning regimen; in-vitro T-cell depletion; alloHCT with cord blood cells; alloHCT from an unrelated donor with ≥3/10 HLA mismatches; use of alemtuzumab, vedolizumab, or abatacept for GvHD prophylaxis; history of chronic digestive disease. Patients will be followed monthly up to 6 months after alloHCT and then every 3 months up to 12 months. Recruitment is ongoing in Europe."

Clinical • P2b data • Hematological Malignancies • Oncology

MaaT Pharma Announces First DSMB Positive Review of Ongoing Phase 2 Clinical Trial Evaluating MaaT033 for Patients Receiving Allo-HSCT (Businesswire) - "The Independent Data Safety and Monitoring Board (DSMB) has recommended that the trial proceed as planned without modifications. MaaT033 has shown to have an acceptable safety profile and was well tolerated in patients treated for blood cancers and receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT)....The DSMB, composed of 5 independent experts, reviewed safety data on the first 20 patients (cutoff date as of April 30th, 2024) and concluded that safety was acceptable and well tolerated."

DSMB • Hematological Malignancies • Oncology

IASO: Safety and Gut Microbiota Analysis of an Oral Microbiotherapy in Patients With Amyotrophic Lateral Sclerosis (clinicaltrials.gov) - P1 | N=15 | Active, not recruiting | Sponsor: MaaT Pharma | Recruiting ➔ Active, not recruiting

Enrollment closed • Amyotrophic Lateral Sclerosis • CNS Disorders

MaaT Pharma Announces Positive Review from the DSMB on the Ongoing Phase 1 Clinical Trial Evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS) (Businesswire) - "MaaT Pharma...announced that the DSMB reviewed safety data in the first 8 patients with Amyotrophic Lateral Sclerosis (ALS) treated with MaaT033 in the IASO clinical trial. The DSMB recommended that the trial continue without modifications....Data readout is now expected in early H2 2024....MaaT033 has shown a good safety profile and was generally well tolerated in the first 8 patients with ALS treated with MaaT033 used in a chronic setting."

DSMB • P1 data • Amyotrophic Lateral Sclerosis

A MULTICENTRE, RANDOMIZED, DOUBLE-BLINDED, PHASE 2B TRIAL EVALUATING MAAT033, AN ORAL, POOLED MICROBIOME ECOSYSTEM THERAPY IN ALLOHCT PATIENTS TO IMPROVE OVERALL SURVIVAL : PHOEBUS TRIAL (EBMT 2024) - P2b | "Exclusion criteria comprise non-myeloablative conditioning regimen, conventional myeloablative conditioning regimen, in-vitro T-cell depletion, alloHCT with cord blood cells, alloHCT from an unrelated donor with ≥ 3/10 HLA-mismatches, use of alemtuzumab, vedolizumab or abatacept for GvHD prophylaxis, history of chronic digestive disease...Secondary endpoints will include evaluation of the safety of MaaT033, GvHD-free survival, cumulative incidence of acute GvHD and chronic GvHD, non-relapse mortality, relapse-free survival, GvHD-free, relapse-free survival, the proportion of patients with severe infections, gut microbiota composition, and quality of life.Exploratory endpoints will describe the impact of MaaT013 on immune recovery, the nutritional status of the patients, and resource utilization.Patients will receive MaaT033 or placebo (3 capsules per day) for 1 week before the start of the conditioning regimen... The study has already been approved in France and..."

Clinical • P2b data • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Oncology • Transplantation

MaaT Pharma Presents Positive Results with Lead Product MaaT013 and Provides Insights on Ongoing Phase 2b Trial with MaaT033 at ASH 2023 (Businesswire) - P=NA | N=111 | ATLAS (NCT04768907) | "MaaT Pharma...presented positive results from the Early Access Program (EAP) in Europe involving 111 patients with steroid-refractory (SR) or steroid-dependent (SD) gastrointestinal acute graft-versus-host Disease (GI-aGvHD) treated with MaaT013, at the 2023 American Society of Hematology (ASH) Annual Meeting....GI-ORR of 53% at day 28, with Complete Response observed in more than two thirds of responders (35%); Overall Response Rate (ORR) considering all organs was 50% with 31% Complete Response (CR). OS was 56% at 6 months and 47% at 12 months with a median follow up of 355 days....MaaT Pharma also presented its ongoing Phase 2b trial design for MaaT033, its second candidate, at the 2023 ASH Annual Meeting. Developed as an adjunctive therapy to enhance OS in HSCT (hematopoietic stem cell transplantation) and cellular therapy recipients."

Clinical data • Clinical protocol • Acute Graft versus Host Disease • Transplantation