On December 9, 2024 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs), reported updated data from the investigator-initiated Phase 2 clinical trial evaluating ZYNLONTA (loncastuximab tesirine-lpyl) in combination with rituximab to treat relapsed or refractory (r/r) follicular lymphoma (FL) were published in the December issue of The Lancet Haematology, following an oral presentation of the data at the recent 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, ADC Therapeutics, DEC 9, 2024, View Source [SID1234648941]).

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"We are excited by the publication of these results in The Lancet Haematology demonstrating ZYNLONTA’s robust clinical activity in follicular lymphoma, particularly in patients classified as high-risk POD24 and those with high tumor burden where there remains significant unmet need," said Mohamed Zaki, MD, PhD, Chief Medical Officer of ADC Therapeutics. "In addition, encouraging data from another investigator-initiated trial of ZYNLONTA as a single agent to treat marginal zone lymphoma were also presented at ASH (Free ASH Whitepaper). Collectively, we believe these data underscore ZYNLONTA’s promise for patients with indolent B-cell lymphomas and add to a growing body of evidence showing the potential of ZYNLONTA beyond diffuse large B-cell lymphoma."

ZYNLONTA in combination with rituximab to treat r/r follicular lymphoma (FL)
The investigator-initiated trial conducted at the Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine evaluated the combination in patients with r/r FL treated with ≥1 line of systemic therapy presenting high-disease burden as defined by GELF criteria or POD24 at enrollment. The primary endpoint of the study is complete response rate (CR) by week 12 PET/CT based on Lugano 2014 criteria. The trial enrolled 39 patients, all of which were evaluated for safety and 35 of which were evaluated for efficacy.

Patients were a median age of 68 years (range 47 to 89) and the majority received one previous line of therapy (n=26; 67%). R-CHOP was the most common first-line therapy (n=22; 56%) followed by bendamustine with rituximab (n=10; 26%), single-agent rituximab (n=6; 15%) and fludarabine, mitoxantrone and dexamethasone (n=1; 3%).

Highlights from the results published in The Lancet Haematology included:

Best overall response rate (ORR) of 97.4% (n=38) and CR rate of 76.9% (n=30)
After a median follow-up of 15.6 months, the median progression-free survival (PFS) was not reached, and the 12-month PFS was 94.6%
The most common treatment-emergent adverse events (TEAEs) were hyperglycemia (n=17; 43.6%) followed by increased alkaline phosphatase (n=16; 41%) and neutropenia, fatigue and increased aspartate aminotransferase and alanine aminotransferase (n=15; 38.5%)
The most common grade ≥3 TEAE were lymphopenia (n=8; 20.5%) followed by neutropenia (n=5; 12.9%)
No Grade 5 TEAEs occurred.
The publication titled, "Loncastuximab tesirine with rituximab in patients with relapsed or refractory follicular lymphoma: a single centre, single arm Phase 2 trial," is now available online and will be published in the December issue of The Lancet Haematology. The results were also presented during a session on indolent B-cell lymphomas at ASH (Free ASH Whitepaper) by Juan Pablo Alderuccio, MD, lead investigator and Associate Professor of Medicine and Hematologist at Sylvester. More details on the trial can be found at View Source (identifier: NCT04998669).

ZYNLONTA as a single agent to treat r/r marginal zone lymphoma (MZL)
Data from an open-label, multi-institutional investigator-initiated trial evaluating the safety and efficacy of ZYNLONTA in 23 adult r/r MZL patients, previously treated with ≥1 line of systemic therapy, were also shared as a poster presentation at ASH (Free ASH Whitepaper) by lead investigator, Izidore Lossos, MD, Professor of Medicine and Chief of the Lymphoma Section of the Division of Hematology at the Sylvester Comprehensive Cancer Center, University of Miami. The median age in this study was 65 years (range 45-82). The median number of previous treatments was 2 (range 1 to 4).

As of October 15, 2024, 23 patients were evaluable for response. Highlights from the results presented include:

ORR of 91% (n=21); 70% CR (n=16). ZYNLONTA led to CR in 7 of 11 patients (64%) with POD24 assessed for response and one patient who progressed after CAR-T.
All but 1 CR are currently maintained with the longest follow-up of 27 months from the start of treatment (median duration of CR is 11.5 months).
All of the 23 enrolled patients experienced expected adverse events (AE), most commonly grade 1 or 2. Grade 3 and 4 AEs were observed in 15 and 1 (neutropenia) patients, respectively. Local edema was observed in 10 (43.4%) patients. Three patients needed dose reduction and one patient discontinued treatment after cycle 4 because of cholestatic hepatitis. The patient clinically fully recovered with normalization in liver function test abnormalities.
More details on this ongoing Phase 2 clinical trial can be found at View Source (identifier: NCT05296070).

About ZYNLONTA (loncastuximab tesirine-lpyl)
ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval and in the European Union under conditional approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Please see full prescribing information including important safety information about ZYNLONTA at www.ZYNLONTA.com.

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.

On December 9, 2024 Cantex Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions, reported that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to Cantex’s azeliragon for the treatment of brain metastasis from breast cancer (Press release, Cantex, DEC 9, 2024, View Source [SID1234648940]). This new azeliragon designation adds to azeliragon’s two other Orphan Drug Designations for the treatment of pancreatic cancer and glioblastoma, received in mid-2024 and early 2023, respectively.

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Cantex’s azeliragon is a well-tolerated compound, administered orally once-a-day, that inhibits the receptor for advanced glycation end products (known as RAGE). The binding of RAGE on cancer cells to S100 proteins and other ligands has been linked to resistance to radiation, disease progression, and development of metastasis in breast cancer.

The development of brain metastasis is a life-threatening complication of breast cancer. Although critically important life-extending advances in the treatment of brain metastasis from some forms of breast cancer have recently been made, brain metastasis from triple-negative breast cancer, an aggressive subtype of breast cancer, remains a therapeutic challenge greatly in need of improved treatments.

"Receiving FDA Orphan Drug Designation for azeliragon for the treatment of brain metastasis from breast cancer highlights a continued need for new treatment options for these patients," commented Stephen G. Marcus, M.D., Chief Executive Officer of Cantex. "This designation reflects our continued commitment to developing new azeliragon treatment options for patients with life-threatening cancer."

In addition to azeliragon’s already issued composition of matter and other patents, FDA Orphan Drug Designations provide Cantex with seven years of azeliragon marketing exclusivity from the time of product launch for the orphan indication, and several other important benefits, including assistance in the drug development process, tax credits for clinical costs, and exemptions from certain FDA fees.

About Azeliragon
Azeliragon is an orally administered capsule, taken once daily, that inhibits interactions of the receptor for advanced glycation end products (known as RAGE) with certain ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Azeliragon was discovered by and originally under development for Alzheimer’s disease by vTv Therapeutics Inc. (NASDAQ: VTVT) from which Cantex licensed worldwide rights to azeliragon. Clinical safety data from those trials, involving more than 2000 individuals dosed for periods up to 18 months, indicate that azeliragon is very well tolerated.

Cantex has ongoing clinical trials in brain metastasis, glioblastoma, breast cancer, pancreatic cancer, and hospitalized patients with pneumonia. These trials are based on azeliragon’s robust preclinical data as well as its extensive clinical safety information from randomized placebo-controlled clinical trials.

On December 9, 2024 Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a leader in macrophage-focused therapeutics, reported a strategic reprioritization of its pipeline, cessation of development of CT-0525, and a reduction in the workforce by 34% (Press release, Carisma Therapeutics, DEC 9, 2024, View Source [SID1234648939]). These measures will enable Carisma to focus its resources on advancing its in vivo macrophage engineering platform for the development of fibrosis, oncology and autoimmune disease therapies. This decision aligns Carisma’s efforts with next-generation, high-potential programs addressing significant unmet patient needs while enhancing operational efficiency.

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"Following a comprehensive review of our portfolio, we have made the strategic decision to prioritize advancing our in vivo macrophage engineering platform," said Steven Kelly, President and Chief Executive Officer of Carisma. "The compelling data generated by both the Moderna-partnered in vivo CAR-M oncology programs as well as our internal liver fibrosis program underscore the potential to revolutionize treatment paradigms with an innovative and patient-centric approach."

"These strategic initiatives, re-directing our investments to the in vivo macrophage engineering platform, discontinuing development of our anti-HER2 program and reducing our workforce, aim to streamline our operations and reduce operating expenses over time," Kelly continued. "While these decisions are very challenging, they are made in the best interest of our shareholders. We remain deeply grateful for the significant contributions of the employees departing Carisma."

Reprioritization Plan, Pipeline Updates, and Upcoming Milestones:

As part of this reprioritization of our pipeline, Carisma will discontinue development of the anti-human epidermal growth factor receptor 2 ("anti-HER2") program, and redirect the Company’s focus to developing off-the-shelf products using its in vivo macrophage engineering platform:

Fibrosis

Carisma’s initial fibrosis program is focused on addressing liver fibrosis, a significant global health challenge. Preclinical data presented at the American Association for the Study of Liver Diseases (AASLD) Liver Meeting 2024 demonstrated the potential of Carisma’s engineered macrophages to reduce inflammation, resolve fibrosis, and promote liver regeneration.
The Company plans to nominate a development candidate for its liver fibrosis program in the first quarter of 2025, reflecting its expertise in macrophage biology and fibrotic diseases.
Oncology

In collaboration with Moderna, Inc. (Nasdaq: MRNA), Carisma is advancing multiple programs utilizing an in vivo chimeric antigen receptor macrophage and monocyte ("CAR-M") plus mRNA/LNP approach. The lead program is an anti-glypican 3 (GPC3) in vivo CAR-M therapy that has demonstrated the potential for this scalable, patient-friendly approach to transform solid tumor therapy. In addition to this program, Moderna has nominated four undisclosed oncology research targets under the collaboration and has the right to designate up to ten oncology targets as development targets.
Autoimmune

In collaboration with Moderna, Carisma has two in vivo CAR-M research programs for the treatment of autoimmune diseases associated with two distinct targets where there is significant unmet medical need.
Carisma retains all rights in autoimmune disease beyond these two nominated targets.
Discontinuation of anti-HER2 Development

The Company’s decision was based on an assessment of the competitive landscape in anti-HER2 treatments, including the impact of recently approved anti-HER2 therapies on HER2 antigen loss/downregulation, and the effects on the future development strategy of any anti-HER2 treatment.
The Company has completed patient enrollment of the Phase 1 clinical trial of CT-0525 and will not enroll patients in the previously planned Cohort 3 of the study.
Based on the data available to date from the anti-HER2 program, CAR-M cell therapy has been shown to be safe, well-tolerated, and feasible to manufacture, and it holds the potential to become a meaningful treatment option for patients.
Corporate Updates

As part of the strategic restructuring, Carisma will reduce its workforce by 34%. The Company expects the reduction in workforce to be substantially complete and to pay the majority of related reduction in workforce amounts by the end of the first quarter of 2025. The Company is committed to supporting affected employees through this transition.
As part of the workforce reductions, our Chief Financial Officer, Richard Morris, our General Counsel, Eric Siegel, and our Senior Vice President, Human Resources, Terry Shields, will leave the Company effective December 31, 2024. Carisma expresses gratitude for their contributions.
The Company expects to incur approximately $2.7 million in connection with the reduction in the workforce, which primarily represents one-time employee termination benefits directly associated with the workforce reduction.

On December 9, 2024 Sumitomo Pharma America, Inc. (SMPA) reported new clinical data supporting further development of nuvisertib, an investigational small molecule being researched for the treatment of relapsed/refractory myelofibrosis (MF), and enzomenib, an investigational oral small molecule being researched for relapsed/refractory acute leukemia, at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (Press release, Sumitomo Pharmaceuticals, DEC 9, 2024, View Source [SID1234648938]).

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Preliminary data presented from the ongoing Phase 1/2 study of nuvisertib monotherapy (N=74) in patients with relapsed/refractory MF show nuvisertib was well tolerated with no dose-limiting toxicities (DLTs). Preliminary data in evaluable patients showed clinical activity including spleen volume reduction (SVR25 of 22.2%), symptom reduction (TSS50 of 44.4%), and bone marrow fibrosis (47.8% patients) and hemoglobin (25% patients) and platelet count (27.6% patients) improvement. Additional data presented show nuvisertib treatment led to significant cytokine modulation (e.g. EN-RAGE, IL-18, MIP-1β, and adiponectin) over time correlating with spleen and symptom responses. The global study has been expanded to evaluate nuvisertib add-on to ruxolitinib, the first approved Janus-associated kinase (JAK) inhibitor, and in combination with momelotinib, a recently approved JAK inhibitor for MF patients with anemia, to assess safety and potential clinical activity.

New preliminary clinical and translational data from the enzomenib Phase 1/2 study were also presented at the conference by Dr. Joshua Zeidner from the University of North Carolina. The safety population included 84 total patients with acute leukemia, most of whom (94%, 79/84) had acute myeloid leukemia (AML). The study population was diverse, with 47.6% non-white (40/84), and also heavily pre-treated, with a median of 3 prior regimens.

In these patients, enzomenib was administered twice daily in continuous 28-day study cycles at dose levels from 40 mg BID up to 300 mg BID. Enzomenib was well tolerated with low overall rates of drug-related adverse events and no dose limiting toxicity (DLT) reported. Differentiation syndrome was reported in 10.7% of patients but did not result in patient deaths nor did it require permanent discontinuations of enzomenib.

Also presented were clinical activity results from the dose optimization cohorts at the dose levels of 200 mg BID and 300 mg BID. The data included results from all patients with KMT2A rearrangement (KMT2Ar) or NPM1 mutation (NPM1m) who had received at least one dose of enzomenib and who had not received a menin inhibitor previously.

In 23 patients with KMT2Ar, the Objective Response Rate (ORR) using ELN 2017 criteria was 65.2% (15/23) and the proportion to achieve CR+CRh was 30.4% (7/23). In the subset of patients with KMT2Ar to receive 300 mg BID (n = 15), the ORR was 73.3% (11/15) and CR+CR was 40% (6/15). In the 17 total patients with NPM1 mutation (NPM1m) who received 200 mg BID or 300 mg BID, the ORR was 58.8% (10/17) and the proportion to achieve CR+CRh was 47.1% (8/17). The 400 mg BID dose optimization cohort is ongoing, with 21 patients enrolled as of the clinical cut-off and data planned for presentation at a future conference.

These encouraging clinical activity results combined with an excellent safety profile suggest that enzomenib may play an important role in the treatment of patients with relapsed/refractory acute leukemia with KMT2A rearrangement or NPM1 mutation.

"These data signal growing momentum in our oncological pipeline and an important milestone in our development of new treatments for relapsed/refractory MF and acute leukemia," said Tsutomu Nakagawa, Ph.D., President and Chief Executive Officer of SMPA. "With these promising results in hand, we remain committed to advancing nuvisertib and enzomenib as we believe our investigational therapies may have the potential to improve the lives of patients living with these critical cancers who do not respond to currently available therapeutics."

MF is a rare type of blood cancer that is characterized by the buildup of fibrous tissue in the bone marrow, which can affect the production of blood cells. This buildup is caused by a dysregulation in the JAK signaling pathway. MF is a serious and rare disease with 0.7 new cases per 100,000 people worldwide each year.4

Leukemia is a type of cancer that forms in blood-forming tissue, characterized by the uncontrolled growth of blood cells, usually white blood cells, in the bone marrow. Acute leukemia, a form of leukemia, requires immediate treatment as blood cells multiply rapidly leading to a sudden onset of symptoms.1 Approximately 30% of AML patients have NPM1 mutations2 and 5-10% of AML patients have KMT2A (MLL) rearrangements.3

"Patients and families living with incurable cancers of MF and AML are in need of new treatment options – and we are encouraged by the promising clinical activity for both nuvisertib and enzomenib," said Jatin Shah, M.D., Chief Medical Officer, Oncology, SMPA. "This additional preliminary data reinforces the strong potential for PIM1 inhibition in myelofibrosis, and for menin inhibitors in acute leukemia."

About enzomenib (DSP-5336)
Enzomenib (DSP-5336) is an investigational small molecule inhibitor of the menin and mixed-lineage leukemia (MLL) protein interaction. Menin is a scaffold nuclear protein which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.5,6 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A (MLL) rearrangements or NPM1 mutations.5,7 Enzomenib reduced the expression of the leukemia-associated genes HOXA9 and MEIS1, and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with MLL rearrangements and NPM1 mutation.8,9 The safety and efficacy of enzomenib is currently being clinically evaluated in a Phase 1/2 dose escalation/dose expansion study in patients with relapsed or refractory acute leukemia (NCT04988555). The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022. The FDA granted Fast Track Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in June 2024. The PMDA granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in September 2024.

About nuvisertib (TP-3654)
Nuvisertib (TP-3654) is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.10,11 Nuvisertib was observed to inhibit proliferation and induce apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2V617F mutation.10 Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization, and also reduced spleen size and bone marrow fibrosis in JAK2V617F and MPLW515L murine models of myelofibrosis.11 The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate and high-risk myelofibrosis (NCT04176198). The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for nuvisertib for the indication of myelofibrosis in May 2022. The PMDA granted Orphan Drug Designation for nuvisertib for the indication of myelofibrosis in November 2024.

On December 9, 2024 TransThera Sciences Nanjing, Inc. (the "TransThera") reported the poster presentation at the 2024 European Society For Medical Oncology (ESMO) (Free ESMO Whitepaper) Asia Congress to discuss the clinical study of tinengotinib in combination with atezolizumab (Tecentriq) in biliary tract carcinoma (BTC) (Press release, TransThera Biosciences, DEC 9, 2024, View Source [SID1234648937]).

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Title: Tinengotinib (TT-00420) in Combination with Atezolizumab in Chinese Patients with Biliary Tract Carcinoma (BTC): Efficacy and Safety Results from a Phase Ib/II Study

Poster number: 140P

Immune-checkpoint inhibitor (ICI) combined with chemotherapy have been approved as first-line therapy for BTC. However, no standard of care has been established after first-line treatment and the survival of the patients is short, there is a huge unmet clinical need. Tinengotinib is a novel multi-kinase inhibitor. It may play a role of synergistic effect when combined with ICI. The efficacy and safety data of tinengotinib in combination with atezolizumab in Chinese BTC patients has been explored in a phase Ib/II study.

Results:

As of September 26, 2024, a total of 31 heavily pretreated advanced BTC patients were enrolled and treated with tinengotinib plus atezolizumab. 71.0% were priorly treated with at least one immunotherapy.

Promising efficacy:

A total of 20 intrahepatic cholangiocarcinoma patients were efficacy evaluable. The objective response rate (ORR) and disease control rate (DCR) were 25.0% and 80.0%, the median progression free survival (mPFS) reached 8.77 months and the 12-month overall survival (OS) rate was 70.1%.
A total of 28 cholangiocarcinoma patients were efficacy evaluable. The ORR and DCR were 25.0% and 75.0%, the mPFS reached 5.72 months and the 12-month OS rate was 64.8%.
All 31 BTC patients were efficacy evaluable. The ORR and DCR were 22.6% and 74.2%, the mPFS reached 4.11 months and the 12-month OS rate was 61.8%.
Similar efficacy was observed regardless of prior ICI(s) therapy.
Good safety and tolerability:

No DLT was observed in the dose escalation phase.
The combination therapy was generally well tolerated in heavily pre-treated BTC patients.
These encouraging results suggest the combination therapy may have synergistic effect in treating some solid tumor such as BTC, and support the further exploration to evaluate the safety and efficacy of tinengotinib plus ICI in BTC patients who were previously treated with or without ICI therapy.

About Tinengotinib

Tinengotinib is an internally discovered, global phase III multi-kinase inhibitor that exerts antitumor effects by targeting FGFRs and VEGFRs, mitotic kinases Aurora A/B and Janus kinases (JAK). Ongoing clinical trials in the US and China have revealed the potential of tinengotinib to be efficacious in various solid tumors. It was granted the Orphan Drug Designation (ODD) and Fast Track Designation (FTD) by the FDA for the treatment of CCA, the Breakthrough Therapy Designation (BTD) by NMPA in China, the Orphan Drug Designation (ODD) for the treatment of biliary tract cancer by EMA.