On November 23, 2025 J & D Pharmaceuticals LLC reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s investigational therapy for the treatment of Hepatocellular Carcinoma (HCC) a rare and life-threatening disorder that is estimated to occur in approximately 73,000 individuals in the United States in 2025.

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HCC is a devastating disorder that has a very poor prognosis. With FDA approved medications the amount of time extended in patient with HCC is only 2.5 months. There is a need for a safer and more effective medication for HCC.

"Receiving Orphan Drug Designation is yet another significant milestone for J & D Pharmaceuticals," said Lenard Lichtenberger, PhD, Chief Scientific of J & D Pharmaceuticals LLC. "This designation adds to the two previous orphan drug designations we received and continues to underscore the urgent need for innovative therapies for HCC patients and strengthens our commitment to developing solutions that we expect to transform the lives of those affected by this debilitating condition not just by adding years to their lives but life to those years."

The FDA’s Orphan Drug Designation program provides incentives to encourage the development of treatments for rare diseases, including tax credits for qualified clinical testing, exemption from certain FDA fees, and the potential for seven years of market exclusivity upon regulatory approval.

J & D Pharmaceuticals plans to advance its HC program into clinical development and will continue working closely with the FDA and the HCC community to accelerate progress toward delivering a novel treatment option to patients in need.

(Press release, J & D Pharmaceuticals, NOV 23, 2025, View Source [SID1234660994])

On November 23, 2025 Harbour BioMed ("HBM" or the "Company"; HKEX: 02142), a global biopharmaceutical company committed to the discovery and development of novel antibody therapeutics for immunology and oncology, reported an update and advancement of its global strategic collaboration with AstraZeneca, originally established in March 2025. The collaboration aims to discover and develop next-generation biotherapeutics, including antibody-drug conjugates (ADCs) and T cell engagers, leveraging the knowledge of both companies.

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Under the terms of the agreement, AstraZeneca will continue to nominate discovery programs to Harbour BioMed each year over the next four years, reflecting the continued progress of the partnership, and will retain the option to license these programs for further development. Harbour BioMed will be eligible to receive option and option exercise fees, development and commercial milestone payments, plus tiered royalties on future net sales on such licensed programs. The economic terms are consistent with the financial framework established in March 2025.

Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed, said: "We are pleased to advance our collaboration with AstraZeneca to develop next-generation biotherapeutics in oncology. Harbour BioMed has collaborated with AstraZeneca on multiple programs since 2022, and over time, the two parties have established a trusted and solid partnership. With our strong capabilities enabled by our proprietary antibody platforms, we are well positioned to support AstraZeneca in developing innovative biotherapeutics that can address significant unmet medical needs and improve patient outcomes globally."

(Press release, Harbour BioMed, NOV 23, 2025, View Source [SID1234660873])

On November 21, 2025 Moleculin presented its corporate presentation.

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(Presentation, Moleculin, NOV 21, 2025, View Source [SID1234660901])

On November 21, 2025 Investigators from Hackensack Meridian John Theurer Cancer Center (JTCC)—a leading research partner of the NCI-designated Lombardi Comprehensive Cancer Center at Georgetown University, and number one Cancer Center in New Jersey— reported it will present 65 studies at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, taking place December 6–9, 2025, in Orlando.

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This represents one of JTCC’s largest and most diverse scientific contributions to ASH (Free ASH Whitepaper) to date, highlighting innovations in cell therapy, targeted agents, AI-driven diagnostics, stem cell transplantation, and real-world evidence across virtually every hematologic disease area.

"John Theurer Cancer Center continues to help shape the future of blood cancer care," said André Goy, MD, chair, physician-in-chief and vice president of oncology at Hackensack Meridian Health. "Our teams are redefining transplantation, advancing CAR-T science, and co-leading trials testing next-generation targeted therapies and immunotherapies. The depth and breadth of our ASH (Free ASH Whitepaper) presentations underscore our mission: to bring transformational science rapidly to the clinic for patients with blood cancers and other serious blood disorders."

Highlights of JTCC Research to be presented at ASH (Free ASH Whitepaper) 2025 can be found here.

Leukemia

High event-free (EFS) and overall survival (OS) after non-total body irradiation (TBI) conditioning and allogeneic hematopoietic cell transplantation (HCT) in next-generation-sequencing minimal residual disease (NGS-MRD) negative B-acute lymphoblastic leukemia (B-ALL): Results from the EndRAD trial (PTCTC ONC1701) (ABSTRACT 25-12959)

A phase 2 dose confirmation trial of oral ASTX030, a combination of oral azacitidine with cedazuridine among patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia (ABSTRACT 25-7509)

Ziftomenib in combination with venetoclax and azacitidine in relapsed/refractory NPM1-m or KMT2A-r acute myeloid leukemia: Updated phase 1a/b safety and clinical activity results from KOMET-007 (ABSTRACT 25-3910)

MRD-guided therapy of sonrotoclax (BGB-11417) + obinutuzumab (O) in patients with treatment-naive CLL: Initial results from an ongoing phase 1/1b study, BGB-11417-101 (ABSTRACT 25-7489)

Real world outcomes of bispecific T-cell engagers in plasma cell leukemia (ABSTRACT 25-4651)

Etoposide can be safely removed from induction chemotherapy without impacting survival for pediatric acute myeloid leukemia – a report from the Children’s oncology group study AAML1831 (ABSTRACT 25-12270)

Updated response and safety analyses from a Phase 1 study of ivosidenib combined with intensive chemotherapy in patients with newly diagnosed (ND) Acute Myeloid Leukemia with isocitrate dehydrogenase (IDH)1 mutation (ABSTRACT 25-421)

AI-derived prediction of response and relapse to venetoclax plus hypomethylating agent based therapy in Acute Myeloid Leukemia (ABSTRACT 25-14588)

TSC-101 eliminates recipient hematopoietic cells and demonstrates potential for improved relapse-free survival in patients with AML, ALL, or MDS undergoing allogeneic HCT: Updated results from the Phase 1 (ALLOHA) trial (ABSTRACT 25-12098)

Trials in progress: Design of a registrational Phase 2 trial (ALLOHA) using an external control arm for TSC-101 for prevention of relapse post allogeneic HCT in patients with ALL, AML, or MDS (ABSTRACT 25-13827)

Developing artificial intelligence-based transcriptomic signature for selecting patients with HOXA-MEIS1 pathway abnormalities for the treatment with menin inhibitors (ABSTRACT 25-4126)

Evaluation of ventoclax initiation prophylaxis and monitoring outcomes at each dose level and time point in patients with chronic lymphocytic leukemia: A real-world experience (ABSTRACT 25-2129)

Developing transcriptomic signature for IDH1 and IDH2 acute leukemia and the demonstration of high prevalence of these signatures in mutation-negative leukemia (ABSTRACT 25-4127)

Impact of DUSP22 and TP63 rearrangements in patients with ALK-negative ALCL treated with frontline BV-CH(E)P (ABSTRACT 25-1798)

Harnessing repressive LEF1/β-catenin complexes to overcome drug resistance in chronic lymphocytic leukemia (ABSTRACT 25-14485)

Reducing Acute Myeloid Leukemia resistance to CAR T cell therapy by epigenetic activation of the tumor inflammasome-pyroptosis signaling (ABSTRACT 25-13458)
Lymphoma

Epcoritamab + R-mini-CHOP results in 2-year remissions and high MRD negativity rates in elderly patients with newly diagnosed DLBCL: Results from the EPCORE NHL-2 trial (ABSTRACT 25-3828)

Epcoritamab with rituximab + lenalidomide (R2) and epcoritamab maintenance deliver deep and durable remissions in previously untreated (1L) follicular lymphoma (FL): 3-year outcomes from epcore NHL-2 arms 6 and 7 (ABSTRACT 25-2787)

Liquid-biopsy mutation landscape and its concordance with skin biopsies in cutaneous T-cell lymphoma (ABSTRACT 25-2858)

ZUMA-25 preliminary analysis: A Phase 2 study of brexucabtagene autoleucel (brexu-cel) in patients (Pts) with relapsed/refractory (R/R) Burkitt lymphoma (BL), substudy C (ABSTRACT 25-2841)

Final results of a phase 1 trial with soquelitinib (SQL), a selective interleukin-2-inducible T cell kinase (ITK) inhibitor for treatment of relapsed/refractory (R/R) T cell lymphomas (TCL) (ABSTRACT 25-2574)

Acalabrutinib plus venetoclax and rituximab in patients with treatment-naive (TN) mantle cell lymphoma (MCL): Results from the Phase 2 TrAVeRse study (ABSTRACT 25-7289)

Two-year update of ZUMA-2 Cohort 3: Brexucabtagene autoleucel (Brexu-cel) in patients (pts) with relapsed/refractory mantle cell lymphoma (R/R MCL) who had not received prior Bruton tyrosine kinase inhibitor (BTKi) therapy (ABSTRACT 25-2240)

Phase 2 bellwave-003 cohort f: Updated clinical outcomes of nemtabrutinib in participants with relapsed or refractory marginal zone lymphoma (ABSTRACT 25-2322)

A real-world analysis of safety and outcomes with first line nivolumab in combination with doxorubicin, vinblastine, and dacarbazine (NAVD) in patients with classic Hodgkin lymphoma (cHL) – a multicenter cohort study (ABSTRACT 25-2368)

Safety and efficacy of brexucabtagene autoleucel in elderly patients with relapsed or refractory Mantle Cell Lymphoma: A retrospective, multicenter, international study (ABSTRACT 25-2034)

Developing artificial intelligence-based transcriptomic signature for the diagnosis of dark zone lymphoma in patients without MYC gene rearrangement (ABSTRACT 25-7855)

A multicenter real-world analysis of combined chemotherapy followed by consolidative radiation versus chemotherapy alone in the management of early-stage Hodgkin lymphoma – the HODGKIN25 study (ABSTRACT 25-1471)

Nivolumab with doxorubicin, vinblastine, and dacarbazine (NAVD) in older adults with classic Hodgkin lymphoma: Do S1826 results hold up in the real world? (ABSTRACT 25-7840)

First-line salvage therapies in relapsed/refractory large B-cell lymphoma after second- or third-line CD19-directed CAR T-cell therapy (ABSTRACT 25-3402)

Multicenter, randomized Phase II study of epcoritamab for patients with large B-cell lymphomas achieving a partial response after CD19-directed CAR T-cell therapy: Trial in progress (ABSTRACT 25-15528)

Zanubrutinib + obinutuzumab + sonrotoclax in patients with treatment-naive chronic lymphocytic leukemia/small lymphocytic lymphoma (TN CLL/SLL): Initial results from an ongoing phase 1/1b study, BGB-11417-101 (ABSTRACT 25-4113)

Sustained remissions beyond 4 years with epcoritamab monotherapy: Long term follow-up results from the pivotal EPCORE NHL-1 trial in patients with relapsed or refractory large B-cell lymphoma (ABSTRACT 25-7543)
Multiple Myeloma

Real-world outcomes with elranatamab in multiple myeloma: A multi-center analysis from the United States multiple myeloma immunotherapy consortium (ABSTRACT 25-2557)

Phase 1 study of ktx-1001, a first-in-class oral MMSET/NSD2 inhibitor, demonstrates clinical activity in relapsed/refractory multiple myeloma (ABSTRACT 25-2077)

Phase 2 registrational study of anitocabtagene autoleucel for the treatment of patients with relapsed and/or refractory multiple myeloma: Updated results from iMMagine–1 (ABSTRACT 25-4541)

Alterations in the gut microbiome and the association of butyrate producers with progression-free survival in multiple myeloma patients undergoing autologous stem cell transplantation (ABSTRACT 25-14794)

Talquetamab outcomes in relapsed/refractory myeloma with extramedullary and paraskeletal soft tissue plasmacytomas (ABSTRACT 25-7804)

Safety and efficacy of talquetamab in patients with relapsed and refractory multiple myeloma (RRMM) with and without renal impairment (ABSTRACT 25-7724)

Enhancing the safety of ciltacabtagene autoleucel in relapsed multiple myeloma (MM): Identification of potentially modifiable risk-factors associated with delayed neurotoxicity and non-relapse mortality (ABSTRACT 25-2357)

An open-label, multi-center Phase 2 study to assess the safety and efficacy of burixafor (GPC-100) and propranolol with G-CSF for the mobilization of hematopoietic progenitor cells in patients with multiple myeloma (ABSTRACT 25-14982)

Prospective real-world evaluation of SKY92 for risk stratification in multiple myeloma: Comparison with updated ims/IMWG criteria in the prommis study (ABSTRACT 25-8818)

Pomalidomide salvage in T-cell engager monotherapy failures: Real-world experience with talquetamab or elranatamab with pomalidomide combinations in heavily pretreated multiple myeloma (ABSTRACT 25-10539)

Prolonged elranatamab treatment interruption in patients with relapsed or refractory multiple myeloma (RRMM) is feasible: A retrospective analysis from MagnetisMM-3 (ABSTRACT 25-8338)

Talquetamab, a GPRC5D×CD3 bispecific antibody, in combination with pomalidomide in patients with Relapsed/Refractory multiple myeloma: Updated safety and efficacy results from the Phase 1b monumental-2 study (ABSTRACT 25-11949)

Prospective study of fluoroquinolone resistance colonization in patients undergoing autologous hematopoietic stem cell transplantation in the treatment of multiple myeloma (ABSTRACT 25-13625)

Identifying high-risk profiles and adverse prognoses in relapsed/refractory multiple myeloma treated with bispecific antibodies: A real-world analysis of 943 treatment initiations (ABSTRACT 25-8860)

Intratumoral cellular immunotherapy with autologous hyperactivated M1 SIRPα -low macrophages in non-Hodgkin lymphoma: Clinical results from a first-in-human Phase 1 study (ABSTRACT 25-8309)

Real-world disease burden and treatment patterns among triple-class–exposed patients with relapsed/refractory multiple myeloma and extramedullary disease in the US: A retrospective analysis using Flatiron Health electronic medical records (ABSTRACT 25-9053)

Shared immune features correspond to high-risk multiple myeloma across multiple human subtypes and murine models (ABSTRACT 25-13910)

Real-world efficacy and safety of teclistamab in relapsed or refractory multiple myeloma: Results from 87 patients treated by the polish myeloma group (ABSTRACT 25-11305)
Myeloproliferative Neoplasms

Preliminary data from the Phase I/II study of nuvisertib, an oral investigational selective PIM1 inhibitor, in combination with momelotinib showed clinical responses in patients with relapsed/refractory myelofibrosis (ABSTRACT 25-3882)

Safety and efficacy results from A phase 1b study of R289, a dual irak 1/4 inhibitor, in patients with Relapsed/Refractory (R/R) lower risk myelodysplastic syndrome (LR-MDS) (ABSTRACT 25-13480)

Nuvisertib, an oral investigational selective PIM1 kinase inhibitor, showed clinical responses strongly correlating with cytokine modulation in patients with relapsed/refractory myelofibrosis in the ongoing global phase I/II study (ABSTRACT 25-2614)

Bone marrow microenvironment overlap between vexas and myelodysplastic syndrome demonstrated by targeted transcriptomic and artificial intelligence (ABSTRACT 25-7376)
Noncancerous Blood Disorders

Reduced intensity haploidentical bone marrow transplantation in children with severe sickle cell disease (SCD): BMT CTN 1507 (ABSTRACT 25-11982)

End-of-study results from the ICON3 pines trial, a phase 3, randomized trial of eltrombopag vs. standard first-line treatment for newly diagnosed immune thrombocytopenia in children (ABSTRACT 25-4324)

The real-world safety and efficacy of BCMA-directed chimeric antigen receptor T-cell therapy in systemic AL amyloidosis (ABSTRACT 25-2732)

Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study (ABSTRACT 25-2531)

Children and adolescents with sickle cell disease demonstrate improved health-related quality of life and low decisional regret after hematopoietic cell transplantation: A sickle cell transplant advocacy and research alliance (STAR) study (ABSTRACT 25-12128)

Increased age-adjusted mortality rates from hemophagocytic lymphohistiocytosis (HLH), 2010-2023 (ABSTRACT 25-7365)

Rapid decrease in age-adjusted mortality rates associated with ITP following eltrombopag and romiplostim approvals, but not in TMA following eculizumab approval, 1999-2023 (ABSTRACT 25-9103)
Technology

B- and T-cell clonality using peripheral blood cell-free RNA (cfRNA) in liquid biopsy (ABSTRACT 25-7865)

Not so exclusive: Co-mutations in JAK2, MPL and CALR define distinct hematologic and clonal signatures (ABSTRACT 25-10661)

Temporal control of CAR expression enables thymic generation of autoreactive T cells targeting tumor-associated antigens (ABSTRACT 25-8390)

(Press release, Hackensack University Medical Center, NOV 21, 2025, View Source [SID1234660874])

On November 21, 2025 Parabilis Medicines, a clinical-stage biopharmaceutical company committed to creating extraordinary medicines for people living with cancer, reported preliminary clinical data demonstrating the therapeutic potential of its lead investigational Helicon peptide, FOG-001 – the first and only direct inhibitor of the "undruggable" β-catenin:TCF interaction – in adamantinomatous craniopharyngioma (ACP).

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The ACP data, presented today as a mini-oral at the 30th Society for Neuro-Oncology (SNO) Annual Meeting and first shared at last month’s AACR (Free AACR Whitepaper)-NCI-EORTC 2025 "Triple" Meeting, represent the second low-complexity Wnt/β-catenin-driven tumor subgroup – following desmoid tumors – to show tumor reductions in all patients treated to date with FOG-001 monotherapy.

ACP is a rare brain tumor that is often associated with severe endocrine, visual, and neurological complications, where unmet patient needs are high. Due to the location of tumors, surgery and radiation, the mainstays of treatment, can be challenging and carry a high risk of complications, and no approved systemic therapies exist. Nearly all ACP tumors are driven by CTNNB1 mutations and abnormal activation of the Wnt/β-catenin-driven pathway, creating a strong mechanistic rationale for direct inhibition of the β-catenin:TCF interaction, the key node in the Wnt pathway.

"ACPs are devastating tumors associated with high morbidity, and patients have long faced limited treatment options due to the challenges in systemically addressing the underlying disease biology," said Mathai Mammen, M.D., Ph.D., Chairman, CEO and President of Parabilis Medicines. "Our preliminary clinical data demonstrate that FOG-001 can directly inhibit the β-catenin:TCF interaction, which has long been considered ‘undruggable,’ and offer early evidence of meaningful clinical benefit. This has the potential to be a promising advancement for patients confronting this difficult diagnosis for which targeted, effective treatment options are not currently available. More broadly, we continue to see the potential of FOG-001 in treating multiple tumor types, with desmoid and ACP representing just the beginning of what is possible."

In the company’s Phase 1/2 trial, as of the data cutoff date of August 11, 2025, three patients with ACP with visual field impairment had been treated with FOG-001 at doses of 144 mg/m2 (n=1) and 360/m2 mg (n=2), and all patients (n=3) showed tumor reduction with well-managed safety and tolerability. Two patients achieved a partial response with 56.0% and 48.0% reduction in tumor size, and one patient had stable disease with a 19.2% decrease in tumor size. No treatment-related serious adverse events, dose reductions, or treatment discontinuations have been reported.

At the Triple Meeting, Parabilis also reported single-patient activity in three additional low-complexity Wnt/β-catenin-driven tumors, including ameloblastoma, salivary gland cancer, and solid pseudopapillary neoplasm, primarily driven by dysregulated Wnt/β-catenin signaling, as well as preclinical and clinical data supporting further evaluation of rational combinations in more complex tumor types – including microsatellite stable colorectal cancer.

FOG-001 was recently granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors. The Phase 1/2 clinical trial remains ongoing, enrolling patients across a range of Wnt/β-catenin-driven cancers.

About FOG-001
FOG-001 is an investigational first-in-class competitive inhibitor of β-catenin interactions with the T-cell factor (TCF) family of transcription factors and is currently in clinical development. By directly targeting the β-catenin:TCF protein-protein interaction, FOG-001 is intended to block the Wnt signaling pathway irrespective of the various APC and β-catenin mutations that typically drive disease.

FOG-001 combines key features that distinguish it from previously reported Wnt/β-catenin pathway modulators: FOG-001 acts inside the cell where it binds directly to the key oncogenic driver β-catenin; and FOG-001 blocks the Wnt pathway at the key downstream node, disrupting the interaction between β-catenin and the TCF transcription factors, thereby abrogating the signal transmission by which Wnt pathway mutations are believed to drive oncogenesis.

FOG-001 is currently being evaluated in a first-in-human Phase 1/2 clinical trial in patients with locally advanced or metastatic solid tumors.

About the Phase 1/2 trial of FOG-001
FOG-001 is being evaluated in a first-in-human Phase 1/2 multicenter, open-label study (NCT05919264) assessing its safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity. The trial includes dose-escalation and dose-expansion phases and is testing FOG-001 both as a monotherapy and in combination with other anticancer agents in patients with advanced or metastatic solid tumors likely or known to harbor a Wnt pathway–activating mutation (WPAM).

(Press release, Parabilis Medicines, NOV 21, 2025, View Source [SID1234660871])