On September 19, 2024 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported its half year financial results for the six-month period ended June 30, 2024, and provided a business overview (Press release, MaaT Pharma, SEP 19, 2024, View Source [SID1234646746]).

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"Building on the positive data for MaaT013 in April 2024 presented at the EBMT Congress, and the success of our recent fundraising, we have dedicated the first half of 2024 to pursuing recruitment and preparing for the topline results of our ongoing Phase 3 clinical trial for MaaT013. This trial is designed to address the urgent unmet medical need of patients with acute graft-versus-host disease not responding to current treatments. Currently, patients requiring third-line treatment options face an 85% mortality rate within one year. Furthermore, by extending our cash runway by an additional quarter, we are well-positioned to deliver on our short-term milestones," stated Siân Crouzet, Chief Financial Officer of MaaT Pharma.

Pipeline Highlights

MET-N platform

MaaT013

In hemato-oncology:
In March 2024, the Company announced the launch of a retrospective multicenter trial called CHRONOS in Europe. Its primary objective is to provide the Company efficacy data for 3rd-line therapies for aGvHD patients not receiving MaaT013 or any microbiome intervention. This retrospective study does not impact cash projections as funding is already secured.
In April 2024, at the 50th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), the Company presented promising extended survival data from the Early Access Program in Europe, involving 140 patients with steroid-refractory (SR) or steroid-dependent (SD) acute graft-versus-host disease with gastrointestinal involvement (GI-aGvHD) treated with MaaT013. Data highlighted a high response rate (Complete Response and Very Good Partial Response) to MaaT013, demonstrating a clear reduction in disease burden and improved Overall Survival (OS) at 18 months compared to published data.
In immuno-oncology:
In March 2024, the Company informed on the completion of patient recruitment for the Phase 2a randomized clinical trial (NCT04988841) (PICASSO) sponsored by AP-HP and in collaboration with INRAE and Institut Gustave Roussy, evaluating MaaT013 in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy) and nivolumab (Opdivo), in metastatic melanoma patients. A total of 70 patients have been enrolled since April 2022. The Company provided its MaaT013 drug candidate and placebo and will contribute to the microbiome profiling of patients using its proprietary gutPrint AI research engine. As previously announced, data readout is expected in Q4 2024/Q1 2025.
MaaT033

In hemato-oncology:
In May 2024, the Company announced its participation in the IMMUNOLIFE RHU program, a consortium including academic partners, such as Institut Gustave Roussy (IGR), a world-renowned center in the field of cancer treatment, and biotech companies. MaaT033, an oral, pooled fecal microbiotherapy, developed by MaaT Pharma 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.
As a post-period event, in July 2024, the Company announced first DSMB positive review of the Phase 2b trial PHOEBUS and recommended continuation of the trial without modification. The DSMB concluded that the safety profile was acceptable and the treatment well-tolerated. The trial is an international, multi-center, randomized, double-blind, testing MaaT033, in patient receiving HSCT, an oral freeze-dried formulation against placebo, set to be conducted in up to 56 clinical investigation sites and is expected to enroll 387 patients (NCT05762211).
Recruitment for PHOEBUS trial is ongoing in France, Germany, Spain, and Belgium, with the trial already approved in the Netherlands and the United Kingdom. Upcoming milestones include a second safety assessment by the DSMB expected in early Q1 2025. The interim analysis following the recruitment of 60 patients, is expected in H1 2025, instead of H2 2024. This slight delay is due to the strategic option taken by the Company’s management in early 2024 to prioritize resources for the Phase 3 ARES trial and open new trial sites in countries outside France and Germany in a more sedate manner than originally planned.
In neurodegenerative diseases:
In February 2024, the Company announced a positive review by the DSMB on the Phase 1 clinical trial (IASO) evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS) for the first 8 patients. The DSMB recommended that the trial proceed without modification.
In May 2024, the Company announced the completion of patient recruitment for IASO.

MET-C platform

MaaT034

In combination with immune checkpoint inhibitors in solid tumors
In April 2024, the Company presentednew in vitro data characterizing the metabolites produced by MaaT034 and their impact on immune modulation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024 in San Diego, California. MaaT034, the first product from MaaT Pharma’s MET-C platform, is a ground-breaking full ecosystem synthetic microbiota product being developed for patients with solid tumors to improve responses to immunotherapy in combination with an ICI treatment, which represents a potentially large market. In today’s challenging economic environment, the Company has prioritized resources to focus on MaaT013, specifically preparing marketing authorization activities in Europe and the upcoming Phase 3 topline results in Europe. This approach aimed at optimizing both short-term validation and clinical validation, has resulted in a deferral of activities related to MaaT034. Thus, clinical activities for MaaT034 are now expected to begin in 2026 and not 2025, as previously announced.

Corporate updates

In March 2024, the Company announced the appointment of Jonathan Chriqui, PharmD, as Chief Business Officer and member of the executive management team. Jonathan Chriqui will be responsible for MaaT Pharma’s business development and partnering strategies.
In May 2024, the Company announced the successful completion of its offering of 18.2 million euros. The net proceeds from the Primary Offering were €17.3 million.
In May 2024, the Company announced the production of MaaT013 batches for clinical supply in the US, while advancing the readiness phase to initiate clinical activities. This includes ongoing discussions with prominent US clinicians in stem cell transplantation.
In June 2024, the Company announced the appointment of Gianfranco Pittari, M.D., Ph.D., as Chief Medical Officer. Dr. Pittari brings over 15 years of international experience in clinical research and drug development in hematology, oncology, and immunology. The Company also welcomed Carole Ifi, as head of Regulatory Affairs. Former Senior director, global regulatory lead at UCB, Carole Ifi brings over 25 years of expertise in regulatory affairs and will be instrumental in leading MaaT013 submission.

On September 19, 2024 Kiromic BioPharma, Inc. (OTCQB: KRBP) ("Kiromic" or the "Company") reported that the Deltacel-01 Safety Monitoring Committee (SMC) has unanimously voted in favor of proceeding with the expansion phase of the Deltacel-01 clinical trial (Press release, Kiromic, SEP 19, 2024, View Source [SID1234646745]). This trial is evaluating Deltacel (KB-GDT-01), the Company’s allogeneic, off-the-shelf, Gamma Delta T-cell (GDT) therapy, in patients with stage 4 metastatic non-small cell lung cancer (NSCLC) who have failed to respond to standard therapies.

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This recommendation by the SMC follows a favorable review of safety data from the first two cohorts, including recent results from the 40-day follow-up visit of the sixth patient in Deltacel-01, which demonstrated a positive safety and tolerability profile with no dose-limiting toxicities (DLTs) observed.

As part of the expansion phase of Deltacel-01, Kiromic will enroll approximately nine patients. Screening for new participants is expected to commence later this month. The expansion phase, or Part 2 of Deltacel-01, will further assess the effectiveness of Deltacel treatment.

"We are delighted to receive unanimous SMC approval to move forward with the expansion phase of the Deltacel-01 trial," said Pietro Bersani, Chief Executive Officer of Kiromic BioPharma. "As we enter this next phase with more activated clinical sites, we expect a solid cadence of patient enrollment. We are optimistic about the potential to further evaluate Deltacel’s impact on patient outcomes and address critical unmet needs in solid tumors."

About Deltacel-01

In Kiromic’s open-label Phase 1 clinical trial, titled "Phase 1 Trial Evaluating the Safety and Tolerability of Gamma Delta T Cell Infusions in Combination With Low Dose Radiotherapy in Subjects With Stage 4 Metastatic Non-Small Cell Lung Cancer" (NCT06069570), patients with stage 4 NSCLC will receive two intravenous infusions of Deltacel with four courses of low-dose, localized radiation over a 10-day period. The primary objective of the Deltacel-01 trial is to evaluate safety, while secondary measurements include objective response, progression-free survival, overall survival, time to progression, time to treatment response and disease control rates.

About Deltacel

Deltacel (KB-GDT-01) is an investigational gamma delta T-cell (GDT) therapy currently in the Deltacel-01 Phase 1 trial for the treatment of stage 4 metastatic NSCLC. An allogeneic product consisting of unmodified, donor-derived gamma delta T cells, Deltacel is the leading candidate in Kiromic’s GDT platform. Deltacel is designed to exploit the natural potency of GDT cells to target solid cancers, with an initial clinical focus on NSCLC, which represents about 80% to 85% of all lung cancer cases. Data from two preclinical studies demonstrated Deltacel’s favorable safety and efficacy profile when it was combined with low-dose radiation.

On September 19, 2024 Kiromic BioPharma, Inc. (OTCQB: KRBP) ("Kiromic" or the "Company") reported that the University of Arizona Cancer Center (UACC) has been activated as the fifth clinical trial site in the Deltacel-01 Phase 1 trial evaluating Deltacel (KB-GDT-01), Kiromic’s allogeneic, off-the-shelf, Gamma Delta T-cell (GDT) therapy, in patients with stage 4 metastatic non-small cell lung cancer (NSCLC) who have failed to respond to standard therapies (Press release, Kiromic, SEP 19, 2024, View Source [SID1234646744]).

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The UACC is one of the 57 NCI-Designated Comprehensive Cancer Centers in the U.S. Dr. Ricklie Ann Julian, Assistant Professor of Medicine at UACC, will serve as Principal Investigator at the site.

"We are delighted to partner with UACC, expanding our Deltacel-01 clinical trial to a fifth site. We believe we are well-positioned to enroll the next cohort of patients, and we are optimistic that we will continue to register encouraging results in the expansion phase of the study. The UACC has earned a reputation as a premier research institution actively engaged in clinical trials and delivering the highest-quality care to those with cancer. We look forward to working with their team," stated Pietro Bersani, CEO of Kiromic BioPharma.

The site initiation visit at UACC has been completed and patient enrollment is expected to begin in the coming weeks.

On September 19, 2024 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM") reported positive preliminary data from the Phase 1b/2 study ("DURIPANC") evaluating the combination of AIM’s Ampligen (rintatolimod) and AstraZeneca’s anti-PD-L1 immune checkpoint inhibitor Imfinzi (durvalumab) in the treatment of late-stage pancreatic cancer (Press release, AIM ImmunoTech, SEP 19, 2024, View Source [SID1234646742]).

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DURIPANC is an investigator-initiated, exploratory, open-label, single-center study. AIM previously announced that investigators at Erasmus Medical Center ("Erasmus MC") in the Netherlands had completed the safety evaluation of subjects enrolled in the first dose level of the dose escalation design, finding the combination therapy to be generally well-tolerated with no severe adverse events or dose-limiting toxicities. That first cohort has now reached the pre-determined 6-month stability assessment timepoint and AIM is pleased to announce that two of the three subjects remain stable. The subjects will continue to be treated and receive formal assessment of progression every three months. The standard for calculating median progression-free survival ("PFS") requires that 50% or more of the subjects have seen disease progression. Because 67% of the patients in the cohort evaluated at 6 months have remained stable, AIM cannot yet report on PFS.

Two of the three subjects in the higher-dose second cohort of subjects also have stable disease, although they have not yet reached the 6-month stability assessment timepoint. Investigators continue to treat and monitor these subjects.

Prof. Casper H.J. van Eijck, MD, PhD, Pancreato-biliary Surgeon at Erasmus MC and Coordinating Investigator for the DURIPANC study, commented: "First of all, we have observed improvements in quality of life and we saw no toxicity at all – with ‘quality of life’ recognized as an indicator of stable disease. As a comparison, approximately 80% of patients at Erasmus with similar disease, but who did not receive the treatment, showed disease progression after only three months. While this new data is extremely preliminary, it is also highly encouraging. To have multiple patients with metastatic pancreatic cancer who still have stable disease after six or seven months of maintenance therapy is remarkable – as is having stable disease for 15 or more months after starting FOLFIRINOX. Based on the changes we have seen in immune infiltration into metastatic lesions, I am optimistic that there is a chance that these patients could still have partial or complete responses to this therapy."

"Ampligen continues to demonstrate its synergistic potential as a combination therapy with checkpoint inhibitors and these data – following the positive data from the Dutch-government approved Early Access Program – underscore Ampligen’s potential for the treatment of late-stage pancreatic cancer, where there remains a significant and lethal unmet need," commented AIM Chief Executive Officer Thomas K. Equels.

Read more at ClinicalTrials.gov NCT05927142 – "Combining anti-PD-L1 immune checkpoint inhibitor durvalumab with TLR-3 agonist rintatolimod in patients with metastatic pancreatic ductal adenocarcinoma for therapy effect"

Learn more about the clinical collaboration between AIM, AstraZeneca and Erasmus MC.

On September 19, 2024 Achilles Therapeutics plc (NASDAQ: ACHL) reported the discontinuation of its TIL-based cNeT program and closure of the Phase I/IIa CHIRON and THETIS clinical trials (Press release, Achilles Therapeutics, SEP 19, 2024, View Source [SID1234646741]). The Company will refocus its strategy to explore further engagement with third parties who are developing alternative modalities to target clonal neoantigens for the treatment of cancers, such as neoantigen vaccines, ADCs, and TCR-T therapies. Concurrently, the Company has engaged BofA Securities as a financial advisor in the process of exploring and reviewing value-maximizing strategies.

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"Our data continue to illustrate the importance of clonal neoantigens as targets and show some clinical activity, however our studies in lung cancer and melanoma have not met our goals for commercial viability. We are grateful for the support and commitment of our patients, investigators, employees and shareholders throughout this journey," said Dr Iraj Ali, Chief Executive Officer of Achilles Therapeutics. "We are actively exploring new opportunities to leverage our substantial assets and cutting-edge technology platforms. Our goal remains to drive the development of effective treatments for patients and create long-term value for our shareholders."

In connection with the strategic update, the Company is implementing an employee consultation process in line with UK legislation proposing a workforce reduction and undertaking other cost-cutting measures. The Company recognizes the significant contributions of its talented team and is committed to supporting all employees throughout this transition period. Achilles intends to retain all employees essential for supporting value-realization as part of its strategic review.

As of June 30, 2024, the Company had $95.1 million in cash and cash equivalents.

The full clinical data generated from the Phase I/IIa CHIRON trial in patients with advanced non-small cell lung cancer (NSCLC) and the Phase I/IIa THETIS trial in patients with recurrent or metastatic melanoma will be presented in an upcoming forum.

The process of exploring strategic alternatives may include, but is not limited to, an acquisition, merger, reverse merger, business combination, asset sale, licensing, or other transactions. There can be no assurance that the exploration of strategic alternatives will result in any agreements or transactions, or as to the timing of any such agreements or transactions. Achilles Therapeutics does not intend to discuss or disclose further developments regarding the exploration of strategic alternatives unless and until its Board of Directors has approved a definitive action or otherwise determined that further disclosure is appropriate or required by law.