On December 8, 2025 Amphista Therapeutics ("the Company" or "Amphista"), a leader in the discovery of next generation targeted protein degradation (TPD) medicines, reported that full details of its new preclinical data with its lead Targeted Glue AMX-883, an orally bioavailable, potent and selective degrader of BRD9 were presented on 6 December during the 67th American Society of Haematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, Florida.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The compelling findings support the potential of AMX-883 as a first-line treatment option in the earlier disease setting of acute myeloid leukaemia (AML), one of the most aggressive blood cancers where 5-year survival rates remain at just 33% and where resistance to standard of care treatments like venetoclax remains a major clinical challenge. Based on these data, the Company will advance AMX-883 as a karyotype-independent, pro-differentiation agent into the clinic for AML in H2 2026.

The key data presented at ASH (Free ASH Whitepaper) showed that:

AMX-883 demonstrates potent degradation as a monotherapy and synergistic benefit in combination with venetoclax

AMX-883 (30mg/kg twice daily and 100mg/kg once daily) significantly reduced cancer growth by achieving picomolar potency degradation of BRD9 across a panel of AML cell lines and significantly reduced leukemic burden in bone marrow and blood in vivo, compared to venetoclax alone.
Synergistic efficacy was observed when each dose of AMX-883 was combined with venetoclax (75mg/kg once daily), leading to significant blocking of tumour growth at therapeutically relevant concentrations.
AMX-883 prevents resistance to venetoclax when dosed in combination

The combination of AMX-883 with venetoclax in a venetoclax resistant cell line, prevented the emergence of resistance, and cells had comparable sensitivity to venetoclax from their first exposure.
AMX-883 actively degrades BRD9 in venetoclax resistant cells, prevents upregulation of key resistance markers, including MCL-1 and BCL-2, and increases levels of cell death markers.
Martin Pass, Chief Development Officer at Amphista Therapeutics said, "The important preclinical data presented for the first time show that our Targeted Glue AMX-883, a selective degrader of BRD9, extends the durability and efficacy of combination therapy with venetoclax and prevents AML cancer cells from becoming resistant to venetoclax. We are very pleased to receive positive feedback from experts in the field on this compelling profile, which gives us strong reason to believe that AMX-883 could be a viable treatment option for patients with AML. We look forward to commencing the first clinical trial for this devastating blood cancer in H2 2026."

To deliver the first clinical study of AMX-883, Amphista has built its development capabilities with three key strategic leadership appointments, including Dr. Lisa Butler, Senior Vice President of Clinical Operations who was previously Head of Study Leadership, early Oncology Clinical at AstraZeneca.

The abstract presented at ASH (Free ASH Whitepaper) is available on the congress website.

(Press release, Amphista Therapeutics, DEC 8, 2025, View Source [SID1234661249])

On December 8, 2025 Akari Therapeutics presented its corporate presentation (Presentation, Akari Therapeutics, DEC 8, 2025, View Source [SID1234661247]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On December 8, 2025 Abcuro, Inc., a late-stage clinical biotechnology company developing potentially first-in-class immunotherapies designed to benefit people living with debilitating and progressive rare autoimmune diseases and for other indications where certain cytotoxic T cells are pathogenic, reported interim data from the ongoing Phase 1/2 clinical trial evaluating ulviprubart (ABC008) in patients with T cell large granular lymphocytic leukemia (T-LGLL) with anemia and/or neutropenia, in an oral presentation at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 6-9, 2025 in Orlando, Florida.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Ulviprubart is a potentially first-in-class, potent, monoclonal antibody targeting KLRG1, a novel mechanism of action to drive selective depletion of highly differentiated T cells and modify disease progression. Ulviprubart was designed to target KLRG1-expressing T cells while sparing B and regulatory T cells that are required to maintain normal immune system homeostasis.

"The data presented at ASH (Free ASH Whitepaper) continue to support the potential of ulviprubart to selectively target and deplete highly differentiated T cells that drive debilitating diseases like T-LGLL. At the interim analysis, ulviprubart had an acceptable safety profile and was generally well tolerated across ascending doses, further supporting ulviprubart’s potential in treating patients with T-LGLL," said H. Jeffrey Wilkins, MD, Chief Medical Officer of Abcuro.

T-LGLL is a hematological cancer driven by pathogenic expansion of immune cells that are frequently KLRG1+, resulting in neutropenia and anemia. Neutropenia can lead to frequent infections, a major cause of premature death in patients with T-LGLL, while anemia results in transfusion dependence in approximately one-third of patients.

Key Highlights from Oral Presentation:

The Phase 1/2 clinical trial (NCT05532722) is an open label, ascending dose study of ulviprubart patients with T-LGLL. The primary objective is to evaluate safety and tolerability. Secondary objectives include evaluating initial efficacy and pharmacokinetic/pharmacodynamic (PK/PD) profile of ulviprubart.

As of the data cut-off date of November 15, 2025:

21 patients were enrolled and evaluable for safety. 95% (n=20) of patients had neutropenia and 57% (n=12) of patients had anemia at baseline.
62% (n=13) of patients achieved >12 weeks of Q4W dosing on ulviprubart.
Among evaluable patients, seven experienced sustained depletions of >50% of CD8+ CD57+ KLRG1+ T cells at two or more consecutive visits. Three patients experienced sustained depletions of >90% of both CD8+ CD57+ KLRG1+ T cells and the CD8+ CD57+ parent population.
Among evaluable patients, all had neutropenia and nine patients had anemia at baseline.
With treatment, seven patients had a neutropenia response, defined as an absolute neutrophil count (ANC) increase of ≥50% from baseline for ≥4 weeks or ANC levels ≥1000 cells/µL for ≥4 weeks.
With treatment, two patients had an anemia response, defined as hemoglobin increased ≥1 g/dL for ≥4 weeks and not attributable to transfusion or growth factor.

About Ulviprubart
Ulviprubart (ABC008) is potentially a first-in-class, potent, monoclonal antibody targeting KLRG1, a novel proposed mechanism of action to drive selective depletion of highly differentiated T cells and modify disease progression. KLRG1 is a cell surface receptor predominantly expressed on highly differentiated T cells, while moderately or minimally expressed on other immune cells. Ulviprubart was designed to selectively deplete KLRG1-expressing T cells while sparing B and regulatory T cells that are required to maintain normal immune system homeostasis.

(Press release, Abcuro, DEC 8, 2025, View Source [SID1234661246])

On December 7, 2025 ALX Oncology Holdings Inc., ("ALX Oncology" or the "Company") (Nasdaq: ALXO), a clinical-stage biotechnology company advancing a pipeline of novel therapies designed to treat cancer and extend patients’ lives, reported that positive data from a Phase 2 investigator-sponsored trial (IST) of evorpacept, in combination with standard-of-care rituximab and lenalidomide, for patients with indolent B-cell non-Hodgkin lymphoma (iNHL) is being presented Sunday, December 7 during a poster presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2025 in Orlando, Florida.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are excited to see the results of this Phase 2 study in frontline indolent non-Hodgkin lymphoma patients, where the addition of evorpacept added a meaningful benefit over the historical data for the standard-of-care regimen of rituximab and lenalidomide," said Jason Lettmann, Chief Executive Officer at ALX Oncology. "Dr. Strati pursued this frontline study after seeing promising activity using the same combination in the relapsed refractory setting, which was previously published. Today, we are pleased to see this efficacy replicated in the frontline setting. Additionally, as non-Hodgkin lymphoma is a disease marked by high CD47 expression, this data further supports the relevance of blocking CD47 in the presence of an anti-cancer antibody, rituximab in this case, to allow for the destruction of cancer cells. We look forward to longer term follow up and the evaluation of MRD from this study."

The clinical trial conducted by Dr. Paolo Strati, the trial’s lead investigator and Associate Professor of Lymphoma-Myeloma at The University of Texas MD Anderson Cancer Center, along with his colleagues enrolled a total of 24 patients with previously untreated iNHL, 14 patients with follicular lymphoma and 10 patients with marginal zone lymphoma. The primary objective of best CR rate above 80% was met with evorpacept added to R2 in the context of a historical R2 CR rate of 50%. The investigators found the addition of evorpacept to R2 to be a well-tolerated frontline non-chemotherapy regimen for patients with iNHL, resulting in a high CR rate. 92% of patients achieved a complete response, and 8% achieved a partial response (PR), with the overall response rate (ORR) being 100%. One year progression free survival (PFS) rate was 91%, and one year overall survival (OS) rate was 100%.

Details of the poster to be presented at ASH (Free ASH Whitepaper) 2025 are as follows:
Title: A phase II investigator-initiated frontline trial of evorpacept (ALX148), lenalidomide and rituximab for high tumor burden indolent B-cell non-Hodgkin lymphoma
Presenter: Paolo Strati, M.D., Associate Professor of Lymphoma-Myeloma, The University of Texas MD Anderson Cancer Center
Presentation ID: 3571
Date/Time: December 7, 2025; 8:00 a.m. – 8:00 p.m. EST (poster session viewing); 6:00 p.m. – 8:00 p.m. EST (presentation)
Session Type: Poster Session
Part of Session: 623. Mantle Cell, Follicular, Waldenstrom’s, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster II
Location/Room: OCCC – West Halls B3-B4

(Press release, ALX Oncology, DEC 7, 2025, View Source [SID1234661248])

On December 7, 2025 Incyte (Nasdaq:INCY) reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to INCA033989, a first-in-class mutant calreticulin (mutCALR)-targeted monoclonal antibody, for the treatment of patients with essential thrombocythemia (ET) harboring a Type 1 CALR mutation who are resistant or intolerant to at least one cytoreductive therapy.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

ET is a chronic myeloproliferative neoplasm (MPN) characterized by persistently elevated platelet counts due to abnormal blood cell production in the bone marrow. CALR mutations are the second most common oncogenic driver mutation and are observed in 25% of patients with ET. A 52-bp deletion, also known as a Type 1 mutation, occurs in 55% of patients with a CALR mutation, and is associated with the highest risk of transformation to myelofibrosis (MF) among all ET patients.

"Incyte has long been committed to improving outcomes for patients with MPNs, and this Breakthrough Therapy designation underscores the potential of INCA033989 to be a novel therapy that could significantly transform the treatment of ET patients, who today have limited treatment options," said Pablo J. Cagnoni, M.D., President and Head of Research and Development, Incyte. "The designation allows us to expedite the development pathway for INCA033989 in patients with Type 1 mutations. Looking ahead, we plan to initiate a Phase 3 program evaluating INCA033989 in ET patients with all types of CALR mutations in mid-2026, following alignment with regulators in the first half of next year."

The FDA Breakthrough Therapy Designation was supported by the early Phase 1 data evaluating INCA033989 in ET patients with a Type 1 CALR mutation available at the time of submission.

The preliminary Phase 1 data were presented earlier this year at the 2025 European Hematology Association (EHA) (Free EHA Whitepaper) Congress. In the study, INCA033989 was well-tolerated and demonstrated rapid and durable normalization of platelet counts across evaluated doses, with greater responses seen at higher doses across both mutation types. Updated results from the Phase 1 dose escalation and expansion trial are planned for presentation at the 2025 ASH (Free ASH Whitepaper) Annual Meeting in Orlando (Session 634; Publication #1024; December 8, 4:30-6:00 p.m. ET).

Incyte plans to develop INCA033989 for patients with Type 1 and non-Type 1 CALR mutations and, following discussions with regulatory agencies, plans to initiate a registrational program evaluating patients with ET with a Type 1 or non-Type 1 CALR mutation who are resistant or intolerant to at least one cytoreductive therapy in the first half of next year.

About Essential Thrombocythemia
Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) characterized by persistently elevated platelet counts due to abnormal blood cell production in the bone marrow. People living with ET are at increased risk for blood clots and bleeding and a proportion of patients may progress over time to myelofibrosis or acute leukemia.

About Mutations in Calreticulin (mutCALR)
Calreticulin (CALR) is a protein involved in the regulation of cellular calcium levels and normal protein folding. Somatic, or non-inherited, DNA mutations in the CALR gene (mutCALR) can result in abnormal protein function and lead to the development of myeloproliferative neoplasms (MPNs),1 a closely related group of clonal blood cancers in which the bone marrow functions abnormally, overproducing blood cells.2,3 In Essential thrombocythemia (ET) and myelofibrosis (MF), CALR mutations occur in ~25-35% of patients.1,2

Incyte is at the forefront of developing novel therapies for patients with mutCALR ET or MF that target only malignant cells, sparing normal cells, including INCA033989, a first-in-class, mutCALR-specific therapy.

(Press release, Incyte, DEC 7, 2025, View Source [SID1234661225])