On April 8, 2025 Manhattan BioSolutions, Inc. ("Manhattan Bio" or "MABS"), an emerging biotechnology company developing new classes of precision biologics, reported a significant milestone with the successful validation of its proprietary legumain (LEG)-cleavable linker-payload platform across multiple oncology programs and subsequent securing of expanded rights to this breakthrough antibody-drug conjugate (ADC) technology (Press release, Manhattan BioSolutions, APR 8, 2025, View Source [SID1234651835]). Manhattan Bio’s innovative platform directly addresses the most critical challenges facing current-generation ADCs, particularly premature payload release that leads to off-target toxicity and narrow therapeutic windows. The technology leverages the lysosomal protease legumain, which is frequently overexpressed in various tumor types. The LEG-cleavable platform has now been successfully validated across three distinct oncology programs, first with conventional auristatin payloads and subsequently with novel topoisomerase I inhibitor (TOP1i) payloads derived from exatecan, positioning the company at the forefront of next-generation ADC development.

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In rigorous preclinical studies, the company’s LEG-TOP1i ADCs demonstrated complete tumor regression with strong durability in multiple difficult-to-treat solid tumor xenograft models. These compelling efficacy results were accompanied by favorable pharmacokinetic properties. The platform’s enhanced stability in circulation, with reduced premature payload release, represents a potentially significant advancement over certain existing ADC technologies that often suffer from systemic toxicity due to payload release in plasma. Upon completion of this comprehensive validation program, Manhattan Bio finalized an agreement that considerably expands its rights to the technology. This strategic transaction enables the company to broadly apply its LEG-cleavable platform across an extensive range of therapeutic targets and cancer indications, substantially enhancing its ability to build a diverse pipeline of next-generation ADCs. The expanded rights significantly strengthen the company’s commercial position and open new avenues for high-value strategic partnerships.

"By solving the fundamental stability and selectivity challenges that have limited current ADCs, we’ve created a versatile platform with potential applications across numerous high-value oncology targets," said Dr. Borys Shor, Chief Executive Officer of Manhattan Bio. "This agreement dramatically increases our ability to build internal pipeline assets and establish strategic collaborations that could accelerate bringing important new medicines to patients."

Manhattan Bio is rapidly applying its proprietary technology to high-value oncology targets using both conventional single-payload ADC approaches and innovative dual-payload designs. These next-generation configurations leverage the enhanced stability and tumor-selective activation properties of the company’s LEG-cleavable platform, with proprietary LEG-TOP1i linker-payload technology at their core.

On April 8, 2025 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 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 (Press release, MaaT Pharma, APR 8, 2025, View Source [SID1234651834]). The study compares the efficacy and safety of MaaT033 (experimental arm) to placebo in patients undergoing an Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT).

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Early after allo-HSCT, patients are highly vulnerable and face a significant risk of non-relapse mortality. Therefore, the study protocol includes a specific safety analysis that would trigger a stopping rule in case a pre-defined mortality excess in the experimental arm would be identified after 30 patients have been randomized to receive MaaT033 (approximately 60 enrolled in the study) and monitored for 90 days after allo-HSCT. This analysis is distinct from the ongoing safety assessments conducted every six months, whose positive outcomes have last been communicated on January 21, 2025. 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.

"We are pleased to report that MaaT033’s safety profile continues to be positive. The confirmed absence of a pre-specified mortality excess in patients receiving MaaT033 is of critical relevance", said Gianfranco Pittari, MD, PhD, Chief Medical Officer of MaaT Pharma. "These patients would enormously benefit from innovative therapies enhancing hematopoietic stem cell transplantation outcomes while avoiding toxic effects."

Patient enrollment is ongoing in France, Germany, Belgium, Spain, Netherlands and the United Kingdom. The Phoebus trial is an international, multi-center, randomized, double-blinded study comparing MaaT033 (a standardized, oral, freeze-dried, multi-donor microbiotherapy) to placebo in patients receiving an allo-HSCT. The trial is expected to enroll 387 patients and is set to be conducted in up to 60 clinical investigational sites (NCT05762211).

Building on the demonstrated safety and efficacy profile of MaaT013, the capsule formulation MaaT033, a high-value asset and the second candidate from MaaT Pharma’s native ecosystem platform, is designed to reach a larger patient population through its oral administration. By enabling outpatient use, MaaT033 also supports optimized patient care.

Next steps:

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.

About MaaT033

MaaT033, a donor-derived, high-richness, high-diversity oral Microbiome Ecosystem TherapyTM containing anti-inflammatory ButycoreTM species, is currently being developed as an adjunctive therapy to improve overall survival in patients receiving HSCT and other cellular therapies. It aims to ensure optimal microbiota function and to address a larger patient population in a chronic setting. MaaT033 has been granted Orphan Drug Designation by the European Medicines Agency (EMA).

On April 8, 2025 Indivumed reported major milestones achieved, cementing its position as a leading oncology R&D company. Its unique, patient-centric approach to precision oncology, including data-driven AI to decipher cell biology in thousands of highly standardized cancer tissue samples, has successfully passed its proof-of-concept phase (Press release, Indivumed Therapeutics, APR 8, 2025, View Source [SID1234651833]). Numerous therapeutically novel targets and the first prototype compounds are presented, promising to significantly advance drug development in oncology. Indivumed has expanded its leadership team with Prof. Dr. Anton Wellstein as Chief Scientific Officer (CSO) and Dr. Matthias Evers as Chief Business Officer (CBO) to drive the continued growth of the company.

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Unique Approach and Unmatched Data Quality
Indivumed’s unique patient-centric R&D approach to cancer research is driven by a standardized collection process with a cold ischemia time of under 10 minutes, ensuring the integrity of tissue samples and preserving therapeutic target expression. The resulting database, IndivuType, is built on over 20 years of global clinical collaboration. It combines comprehensive multi-omics including proteomics and phospho-proteomics, and longitudinal clinical data, providing unparalleled molecular insights that are critical for oncology drug discovery.

Building on a Legacy of Excellence
IndivuType is the foundation of Indivumed’s data-driven target identification and validation process. By utilizing advanced data analytics and AI algorithms Indivumed creates a holistic view of deep and multi-modal molecular data to discover therapeutically novel targets. In addition, in-house generated patient-derived 2D and 3D cellular models, closely mimicking original tumor biology, enable effective validation of targets and reduce risk in downstream drug development.
Drug discovery activities and collaborations encompass virtual and fragment-based ligand screening, hit and lead optimization, and preclinical development, aiming to advance promising candidates toward IND. In-depth knowledge of the molecular and clinical characteristics of the targeted patient population further enables the company to support tailor-made clinical trial and patient stratification strategies at an early stage.

Deep insights leading to therapeutically novel targets
The current R&D focus lies on colorectal cancer, a disease with a high unmet medical need. Indivumed’s pipeline includes a range of targets in different stages of validation and drug discovery. Discussions with potential pharmaceutical partners are ongoing to develop hit/lead molecules towards IND and into valuable precision therapeutics.

On April 8, 2025 Kura Oncology, Inc. (Nasdaq: KURA, "Kura"), and Kyowa Kirin Co., Ltd. (TSE: 4151, "Kyowa Kirin"), reported Kura submitted a New Drug Application (NDA) for ziftomenib, a highly selective, once-daily, oral, investigational menin inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM 1) mutation to the U.S. Food and Drug Administration (FDA) on March 31, 2025 (Press release, Kura Oncology, APR 8, 2025, View Source [SID1234651832]).

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Ziftomenib has received Breakthrough Therapy, Fast Track, and Orphan Drug Designations. The FDA has a 60-day filing review period to determine whether the NDA is complete and accepted for review; Kura expects to receive notification from the FDA on this preliminary evaluation in the second quarter of 2025. Priority Review was requested, which, if granted, would provide a target FDA review period of six months after NDA acceptance.

"This NDA submission brings us one step closer to our goal of advancing ziftomenib to market as a new therapeutic option for adult patients with R/R NPM1-m AML, a devastating disease for which there are currently no FDA-approved targeted therapy options," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We look forward to working closely with the FDA throughout the review process and are optimistic about the potential of ziftomenib to impact patients with NPM1-mutant AML. We extend our gratitude to the team at Kura, our dedicated investigators, study site teams, and most importantly, to the patients who participated in our clinical trials, and their families and caregivers, who all helped make this possible. We appreciate the support and cooperation we enjoy with our partner Kyowa Kirin, and we look forward with confidence to the continued progress of this program and our collaboration."

About NPM1-Mutant AML

AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells, or platelets. Despite the many available treatments for AML, prognosis for patients remains poor and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1 mutations are among the most common, representing approximately 30% of AML cases. While patients with NPM1-m AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the R/R setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A, and IDH1/2, with prognosis heavily influenced by the presence of such co-occurring mutations. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5.3 months in 3rd line, and 3.5 months following the 4th line1. There are currently no FDA-approved therapies targeting NPM1-m AML.

About Ziftomenib

Ziftomenib is a potent and selective, oral, investigational menin inhibitor currently in development for the treatment of genetically defined AML patients with high unmet need. In April 2024, ziftomenib received Breakthrough Therapy Designation (BTD) from the FDA for the treatment of adult patients with R/R AML with a NPM1 mutation based on data from Kura’s KOMET-001 clinical trial. Additional information about clinical trials for ziftomenib can be found at www.kuraoncology.com/clinical-trials/#ziftomenib.

On April 8, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, reported that it has executed financing to provide further working capital and support its ongoing business operations (Press release, ImmunityBio, APR 8, 2025, View Source [SID1234651831]). The Company entered into a securities purchase agreement for a registered direct offering with a single institutional investor, providing for the issuance of common stock of ImmunityBio as well as warrants for the purchase of additional shares of common stock of ImmunityBio that is expected to result in gross proceeds at closing of approximately $75 million before deducting any offering-related expenses, subject to customary closing conditions. If fully exercised, the warrants could result in additional gross proceeds of up to approximately $90 million.

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The securities to be sold by the Company are offered under its automatic shelf registration statement on Form S-3 (Registration No. 333-278770). A final prospectus supplement, which contains additional information relating to the offering, will be filed with the SEC and will be available on the SEC’s website at www.sec.gov.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.