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SELLAS Announces Positive Overall Survival in Cohort 3 from the Ongoing Phase 2 Trial of SLS009 in r/r AML

On April 8, 2025 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported Cohort 3 data from the ongoing Phase 2 trial of SLS009 (tambiciclib), a highly selective CDK9 inhibitor, in relapsed/refractory acute myeloid leukemia (r/r AML) (Press release, Sellas Life Sciences, APR 8, 2025, View Source [SID1234651837]).

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"The remarkable results from Cohort 3 of the ongoing Phase 2 trial reinforce the potential of SLS009 to transform outcomes for these heavily pretreated AML patients," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "Not only have we observed unprecedented survival benefits, but the high response rate underscores the therapy’s efficacy profile. The data reveal that relapsed or refractory to venetoclax-based regimens patients receiving 30 mg BIW achieved a mOS of 8.8 months, far surpassing the historical benchmark of 2.5 months. Additionally, the therapy demonstrated a 67% ORR in patients with AML-MRC and 46% in all evaluable patients, significantly exceeding the targeted 20% ORR. With responses seen across different genetic mutations, this approach could be transformational for many underserved patients. We are continuing to explore SLS009’s potential in expansion cohorts to further validate its potential to address critical unmet medical needs."

Patients Characteristics:

14 relapsed and refractory AML patients who previously failed venetoclax-based therapies were enrolled and treated with SLS009 and venetoclax/azacitidine in Cohort 3.
10 out of 14 patients (71%) had AML MRC (acute myeloid leukemia with myelodysplasia-related cytogenetics).
4 of those patients had myelomonocytic phenotype (M4 per FAB classification). The myelomonocytic phenotype has been shown to exhibit inferior responses to venetoclax-based therapies. It is thought to depend more on the MCL1 anti-apoptotic protein than on the BCL2 anti-apoptotic protein.
In terms of specific mutations, 6 patients had ASXL1, 5 had RUNX1 and 3 had TP53.
The median age was 71 (range 35-89 years).
The median number of prior failed therapy was 1 (range 1-6).
13/14 patients were evaluable for efficacy.
All patients had adverse risk cytogenetics per ELN 2022.
Key Results from Cohort 3:

The median overall survival (mOS) for all patients in Cohort 3 was 8.8 months, while the mOS in AML MRC patients reached 8.9 months.
The overall response rate (ORR) in all evaluable patients was 46% in all Cohort 3 patients and 67% in AML-MRC patients, far exceeding the targeted ORR of 20%.
In the subgroup of patients with myelomonocytic AML, 75% of patients responded (3 out of 4).
Among patients with mutation ASXL1, 4/6 (67%) responded; among those with RUNX1 3/5 (60%) responded, and among those with TP53 1/3 (33%) responded. In addition, there were 3 patients with adverse karyotypes and 1 responded.
SLS009 was well-tolerated with no new safety signals observed to date as the regimen remains safe in additional patients enrolled to date.
Phase 2 trial continues in expansion cohorts 4 and 5, in patients with AML-myelodysplasia-related changes (AML-MRC) with ASXL1 mutation (cohort 4) and mutations and cytogenic changes other than ASXL1 (cohort 5).
The Phase 2 clinical trial of SLS009 is an open-label, single-arm, multi-center study designed to evaluate the safety, tolerability, and efficacy of SLS009 in combination with venetoclax and azacitidine at two dose levels, 45 and 60 mg. In the 60 mg dose cohort patients were treated at either a 60 mg dose once per week or a 30 mg dose two times per week. The trial was expanded to include two additional cohorts, one with ASXL1 mutated AML patients and one with patients with myelodysplasia-related molecular abnormalities other than ASXL1. The target response rate at the optimal dose level is 20% with a target median survival of at least 3 months. In addition, the study aims to identify biomarkers for the target patient population and enrichment for further trials. For more information on the study, visit clinicaltrial.gov identifier NCT04588922.

Nurix Therapeutics Reports First Quarter 2025 Financial Results and Provides a Corporate Update

On April 8, 2025 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines, reported financial results for the fiscal quarter ended February 28, 2025, and provided a corporate update (Press release, Nurix Therapeutics, APR 8, 2025, View Source [SID1234651836]).

"Nurix had a strong first quarter marked by important collaboration and regulatory achievements and key additions to our leadership team and Board," said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. "Nurix remains on track to initiate pivotal trials of bexobrutideg, our oral, brain-penetrant, BTK degrader for the treatment of patients with chronic lymphocytic leukemia in 2025. In addition, Nurix continues to make significant progress with our pipeline of degrader-based drugs for the treatment of autoimmune disease and inflammation. Most recently we announced that Sanofi exercised its option to exclusively license an undisclosed Nurix program targeting a previously undruggable transcription factor that is a central regulator of the inflammation response and is distinct from the previously disclosed STAT6 degrader program."

Recent Business Highlights

•Bexobrutideg is the new nonproprietary name for NX-5948: In March 2025, in collaboration with the national naming authority, the United States Adopted Name (USAN) Council, Nurix’s lead Bruton’s tyrosine kinase (BTK) degrader, NX-5948, was assigned the nonproprietary name "bexobrutideg." The U.S. and international drug naming convention is designed to select a single name of worldwide acceptability for each active substance that is intended to be marketed as a pharmaceutical. Most notable with bexobrutideg is the designation of a new suffix, "deg," which references bexobrutideg’s novel degradation mode of action. Targeted protein degraders are characterized by their bifunctional nature, binding to both a target protein and a ligase to drive ubiquitination and catalytic degradation of the target through the proteasome. The new deg suffix is an important recognition that the mechanism of action, pharmacokinetics and pharmacodynamics of targeted protein degraders are fundamentally different than inhibitors, which all use the "ib" suffix. The central stem of the name, "bruti," references the target, Bruton’s tyrosine kinase (as used in ibrutinib, zanubrutinib and acalabrutinib), and the prefix "bexo" is the unique identifier of a specific agent in the class and is often used for ease of reference to the agent.

•Bexobrutideg received U.S. FDA Orphan Drug designation for Waldenström macroglobulinemia: In March 2025, bexobrutideg was granted U.S. Food and Drug Administration (FDA) Orphan Drug Designation for the treatment of Waldenström macroglobulinemia (WM). The FDA’s Orphan Drug Designation program provides orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.
•Announced the appointment of Roy Baynes to Nurix’s board of directors: In March 2025, Nurix announced the appointment of Roy D. Baynes, MB.Bch., M.Med., Ph.D., to its board of directors. Dr. Baynes currently serves as executive vice president and chief medical officer of Eikon Therapeutics and has had a distinguished career in hematology and oncology and over 22 years of clinical leadership experience in pharmaceutical and biotech companies. Dr. Baynes previously served as chief medical officer and head of global clinical development at Merck, where he supervised the entire clinical portfolio at Merck Research Laboratories. Earlier in his career, Dr. Baynes served as Senior Vice President of Oncology, Inflammation and Respiratory Therapeutics at Gilead Sciences, Inc., as Vice President of Global Development and head of the hematology/oncology development team at Amgen, Inc., and as Professor of Medicine at University of Kansas Medical Center and Wayne State University in Detroit, where he held the Charles Martin endowed chair of Cancer Research.

•Announced the appointment of John Northcott as chief commercial officer: In January 2025, Nurix announced the appointment of John Northcott as chief commercial officer. Mr. Northcott joins the executive team as Nurix prepares to launch its pivotal clinical program for NX-5948 in chronic lymphocytic leukemia (CLL) and potentially other B-cell malignancies. Mr. Northcott has extensive U.S. and global commercial leadership experience including the successful commercialization of the first marketed BTK inhibitor ibrutinib, and in a wide range of other therapeutic areas.
Upcoming Program Highlights*
Bexobrutideg (NX-5948): Bexobrutideg is an investigational, orally bioavailable, brain-penetrant, small molecule degrader of BTK. Nurix currently is conducting a Phase 1b clinical trial of bexobrutideg in adults with relapsed or refractory B-cell malignancies. In 2025, Nurix plans to commence a suite of clinical trials designed to support global registration of bexobrutideg for the treatment of patients with CLL. In addition, Nurix anticipates moving into autoimmune and inflammatory diseases and expects to open a new Phase 1b cohort for patients with CLL and associated autoimmune hemolytic anemia and is exploring the filing of a non-malignant hematology IND for autoimmune cytopenias in 2025. Future clinical updates in patients with both CLL and non-Hodgkin’s lymphoma are anticipated in 2025. Additional information on the NX-5948 clinical trial can be accessed at www.clinicaltrials.gov (NCT05131022).

NX-2127: NX-2127 is an orally bioavailable degrader of BTK and the cereblon neosubstrates IKZF1 (Ikaros) and IKZF3 (Aiolos) for the treatment of relapsed or refractory B-cell malignancies. Nurix currently is conducting a Phase 1a/b clinical trial of NX-2127, which includes Phase 1b expansion cohorts focused on patients with diffuse large B-cell lymphoma and mantle cell lymphoma. Following a decision in March 2024 in which the FDA lifted a manufacturing-related, partial clinical hold on the NX-2127 clinical trial, Nurix reinitiated enrollment in a dose escalation study within the current Phase 1a/1b trial using its new chirally controlled drug product. Future clinical updates are anticipated in 2025. Additional information on the NX-2127 clinical trial can be accessed at www.clinicaltrials.gov (NCT04830137).
NX-1607: NX-1607 is an orally bioavailable inhibitor of the E3 ligase Casitas B-lineage lymphoma proto-oncogene B (CBL-B) for immuno-oncology indications, including a range of solid tumor types and lymphoma. Nurix currently is evaluating NX-1607 in an ongoing Phase 1 trial in monotherapy and in a combination cohort utilizing paclitaxel in adults in a range of oncology indications. This study includes a thorough investigation of both dose and schedule in Phase 1a. Future clinical updates are anticipated in 2025. Additional information on the NX-1607 clinical trial can be accessed at www.clinicaltrials.gov (NCT05107674).

GS-6791 (previously NX-0479): GS-6791 is a potent, selective, oral degrader of IRAK4. Degradation of IRAK4 by GS-6791 has potential applications in the treatment of rheumatoid arthritis and other inflammatory diseases. Nurix’s partner, Gilead, is responsible for conducting IND-enabling studies and advancing this program to clinical development, which Nurix anticipates in 2025.

STAT6 degrader: In April 2024, Nurix announced an extension of the ongoing research program with Sanofi for STAT6 (signal transducer and activator of transcription 6), a key drug target in type 2 inflammation, with the goal of nominating a development candidate in the first year of the extended term.
Continued pipeline advancement of strategic collaborations with Gilead, Sanofi and Pfizer: Nurix expects to continue to achieve substantial research collaboration milestones throughout the terms of its collaborations with Gilead, Sanofi and Pfizer.
*Expected timing of events throughout this press release is based on calendar year quarters.

Fiscal First Quarter 2025 Financial Results

Revenue for the three months ended February 28, 2025, was $18.5 million, compared with $16.6 million for the three months ended February 29, 2024. The increase was primarily due to increased revenue from the collaboration with Sanofi resulting from achievement of research milestones and a higher percentage of completion of performance obligations in the current period related to the collaboration with Pfizer. During the three months ended February 28, 2025, Nurix achieved $7.0 million of research milestones under its collaboration with Sanofi. Subsequent to February 28, 2025, Nurix achieved and received a $15.0 million license extension payment under its collaboration with Sanofi.

Research and development expenses for three months ended February 28, 2025, were $69.7 compared with $50.0 million for three months ended February 29, 2024. The increase was primarily related to clinical, contract manufacturing and consulting costs as Nurix continued to accelerate the enrollment of patients in the ongoing trial of bexobrutideg and prepare for the initiation of pivotal trials.

General and administrative expenses for the three months ended February 28, 2025, were $11.7 million, compared with $11.8 million for the three months ended February 29, 2024. The decrease was primarily due to a decrease in professional service costs and local taxes, offset by an increase in consulting costs.
Net loss for the three months ended February 28, 2025, was $56.4 million, or ($0.67) per share, compared with $41.5 million, or ($0.76) per share, for the three months ended February 29, 2024.
Cash, cash equivalents and marketable securities was $549.7 million as of February 28, 2025, compared to $609.6 million as of November 30, 2024. Cash, cash equivalents and marketable securities as of February 28, 2025, does not include $7.0 million of milestones earned in the three months ended February 28, 2025, and a $15.0 million license extension payment received post fiscal quarter end.

Manhattan BioSolutions Secures Expanded Rights to Novel Legumain-Cleavable Topoisomerase 1 Inhibitor (TOP1i) ADC Platform After Successful Multi-Target Validation

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.

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.

April 8, 2025: MaaT Pharma Announces Positive Safety Interim Analysis from DSMB for Phase 2b Trial Evaluating MaaT033 for Patients Receiving Allo-HSCT

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).

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).

Indivumed celebrates milestone of its patient-centric R&D platform: Numerous novel cancer targets discovered for first-in-class therapeutics

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.

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.