On November 28, 2023 Clarity Pharmaceuticals (ASX: CU6) ("Clarity", "the Company"), a clinical stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, reported the successful completion of the first stage of cohort 3 of the Phase I/IIa theranostic trial, SECuRE, evaluating 64Cu/67Cu-SAR-bisPSMA in patients with mCRPC where 3 participants have been treated at the highest dose level of 12GBq of 67Cu-SAR-bisPSMA (Press release, Clarity Pharmaceuticals, NOV 29, 2023, View Source [SID1234638003]). No adverse events were reported in relation to 64Cu-SAR-bisPSMA. Only 1 adverse event was reported and related to the 12GBq cycle of 67Cu-SAR-bisPSMA in 1 of the 3 participants, which was a grade 1 decrease in neutrophil count, and the patient has fully recovered. No ongoing adverse events and no DLTs have been reported and the SRC has recommended the trial progresses with the 3 additional participants as planned in cohort 3.

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The SECuRE trial (NCT04868604)1 is a Phase I/IIa theranostic trial for identification and treatment of Prostate-Specific Membrane Antigen (PSMA) expressing mCRPC using 64Cu/67Cu-SAR-bisPSMA. 64Cu-SAR-bisPSMA is used to visualise PSMA expressing lesions and select candidates for subsequent 67Cu-SAR-bisPSMA therapy. The trial is a multi-centre, single arm, dose escalation trial with a cohort expansion involving up to 44 patients in the US. The aim of the trial is to determine the safety and efficacy of 67Cu-SAR-bisPSMA for the treatment of prostate cancer.

Cohort 3 of the trial has a 3+3 study design with the intent to gather and analyse data from the first 3 participants before progressing with an additional 3 participants. The initial data is very encouraging with no DLTs observed at the highest dose of 67Cu-SAR-bisPSMA (12GBq) and the SRC, responsible for assessing safety of participants and overseeing the general progress of the trial, has assessed the data and recommended the trial continues.

Cohort 3 is the last to assess single doses of 67Cu-SAR-bisPSMA and will be followed by a multi-dose cohort, pending safety evaluation. The initial 3 participants in cohort 3 were heavily pre-treated prior to entering the trial, having received multiple lines of therapy including other investigational products, radioligand therapy and chemotherapy. They continue to be monitored by their physicians for safety and treatment response as per the trial protocol. All 3 participants in cohort 3 remain on the trial following their recent administration of 12GBq of 67Cu-SAR-bisPSMA, with 2 demonstrating a PSA reduction within weeks of dosing, one of which is greater than 90% reduction and the second approximately 40% reduction to date.

Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "We are excited by the PSA declines seen in almost all patients to date in cohorts 2 and 3 from just a single cycle. This result is also seen in patients that have been heavily pre-treated and have failed many other therapies that are either commercial or investigational. Moreover, the safety profile is very favorable and there have been no DLTs reported to date.

"SAR-bisPSMA was designed to be a best-in-class PSMA product as, unlike all other PSMA products in the market, it has dual targeting. The product was optimised to address the challenges of low uptake and retention in lesions that the first generation of PSMA products suffer from, and in both pre-clinical and clinical development to date we have observed two to three times the uptake of SAR-bisPSMA in lesions, followed by retention in lesions out to at least 96 hours. So far, the higher uptake and retention of product, coupled with the advantageous properties of copper-67, has shown quite impressive responses measured by PSA reductions in patients from single cycles of product to date.

"A number of patients from the SECuRE trial have either received, or will soon receive, additional doses of 67Cu-SAR-bisPSMA under the US Food and Drug Administration’s (FDA) Expanded Access Program (EAP). This demonstrates the initial clinical benefit observed in such patients after the administration of a single cycle of the product during the trial.

"As we have seen PSA reductions in the majority of patients after a single cycle of 67Cu-SAR-bisPSMA across all dose levels, which is known to be an independent prognostic indicator of improved overall survival following radio-ligand therapy2,3, we hope to achieve long-term and durable responses once we progress to the multi-dose cohorts of the trial. Furthermore, with commercial quantities of the 67Cu radioisotope now being routinely produced domestically in the US, we see a clear path to commercialisation as we can resolve the supply and manufacturing issues which have plagued the commercial launch of first-generation products. We look forward to sharing more data along with any further updates from patients who may receive single or multiple cycles of 67Cu-SAR-bisPSMA in our programs and bringing this product to the greater prostate cancer patient population," said Dr Taylor.

About SAR-bisPSMA
SAR-bisPSMA derives its name from the word "bis", which reflects a novel approach of connecting two PSMA-targeting agents to Clarity’s proprietary sarcophagine (SAR) technology that securely holds copper isotopes inside a cage-like structure, called a chelator. Unlike other commercially available chelators, the SAR technology prevents copper leakage into the body. SAR-bisPSMA is a TCT that can be used with isotopes of copper-64 (Cu-64 or 64Cu) for imaging and copper-67 (Cu-67 or 67Cu) for therapy.

64Cu-SAR-bisPSMA and 67Cu-SAR-bisPSMA are unregistered products. Individual results may not represent the overall safety and efficacy of the products. The data outlined in this announcement has not been assessed by health authorities such as the US Food and Drug Administration (FDA). A clinical development program is currently underway to assess the efficacy and safety of these products. There is no guarantee that these products will become commercially available.

About Prostate Cancer
Prostate cancer is the second most common cancer diagnosed in men globally and the fifth leading cause of cancer death worldwide4. The American Cancer Institute estimates in 2023 there will be 288,300 new cases of prostate cancer in the US and around 34,700 deaths from the disease5.

On November 28, 2023 Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, reported program updates for pan-RAF inhibitor naporafenib and central nervous system (CNS)-penetrant EGFR inhibitor ERAS-801, as well as a strategic program prioritization that extends its projected cash runway from H2 2025 to H1 2026 (Press release, Erasca, NOV 28, 2023, View Source [SID1234639351]).

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"We are excited about the recent progress across key clinical programs, including the regulatory alignment with U.S. and European health authorities for our global naporafenib registrational trial which is on track to initiate in the first half of 2024, the establishment of the maximum tolerated dose (MTD) for ERAS-801 in patients with glioblastoma (GBM), and the promising preliminary activity of ERAS-007 plus encorafenib and cetuximab (EC) in EC-naïve patients with BRAF mutant colorectal cancer (CRC) presented at ASCO (Free ASCO Whitepaper)," said Jonathan E. Lim, M.D., Erasca’s chairman, CEO, and co-founder. "To further maximize resources, we have strategically refined our pipeline to focus on programs with the greatest therapeutic potential for patients with high unmet needs using a data-driven approach. As such, we are deprioritizing our ERAS-601 clinical trial (FLAGSHP-1) and our preclinical ERAS-5 ULK inhibitor and ERAS-10 protein degrader programs. Importantly, this pipeline prioritization extends our cash runway into the first half of 2026 and through key milestones, including important clinical trial readouts for naporafenib, ERAS-801, and ERAS-007."

Recent Program Updates

Naporafenib Plus Trametinib for Patients with NRAS mutant (NRASm) Melanoma in Pivotal SEACRAFT-2 Trial
End of Phase 2 meetings with U.S. Food and Drug Administration (FDA) and European health authorities confirm SEACRAFT-2 Phase 3 trial design and provide clarity on registrational pathway:

Enrollment of patients with high unmet medical need who progressed on, or are intolerant to, standard of care immune checkpoint inhibitor therapy
Comparator arm is physicians’ choice of cytotoxic chemotherapy or trametinib
Dual primary endpoint evaluation of progression free survival (PFS) and overall survival (OS), with PFS acceptable for potential initial approval
The Phase 3 trial consists of two stages: a randomized, controlled, dose optimization stage, for which we expect to have a data readout in 2025, and a randomized, controlled stage to support regulatory approval
Phase 3 SEACRAFT-2 trial initiation remains on track for H1 2024
ERAS-801 for Patients with Recurrent GBM in Phase 1 THUNDERBBOLT-1 Trial
Thirty-three patients with recurrent glioblastoma (rGBM) were enrolled in seven dose escalation cohorts. ERAS-801 monotherapy has been granted Orphan Drug and Fast Track Designations (ODD and FTD) by the FDA. Interest in THUNDERBBOLT-1 from trial investigators continues to be robust.

MTD identified as 240 mg once a day (QD) and MTD-1 identified as 160 mg QD for ERAS-801
ERAS-801 was safe and tolerable at the MTD and MTD-1 dose levels. The adverse event profile was consistent with the mechanism of action and observations in non-clinical studies
ERAS-801 exhibited well behaved pharmacokinetic (PK) characteristics: ERAS-801 showed rapid absorption and had dose-dependent increases in PK exposure. Steady-state PK exposures at doses of 160 mg QD and above exceeded the concentration at which 90% tumor growth inhibition was achieved in the GBM patient-derived orthotopic xenograft (PDOX) mouse model.
Expansion cohorts actively enrolling to collect additional safety, tolerability, and preliminary efficacy data to support dose optimization and selection
Phase 1 dose escalation data to be presented at a scientific meeting in H1 2024; expansion cohort data are anticipated in H2 2024
Shannon R. Morris, M.D., Ph.D., Erasca’s chief medical officer, added, "ERAS-801 is an orally bioavailable CNS-penetrant EGFR inhibitor specifically designed to target the unique EGFR alterations observed in GBM, initially as a monotherapy treatment. Identification of the MTD signals the transition of ERAS-801 to the next stage of development with a focus on characterizing the clinical activity of the molecule in patients with EGFR-amplified rGBM, who may achieve maximum benefit from treatment. Notably, this population represents nearly half of all patients with GBM and 85% of all patients with EGFR-altered GBM."

Prioritized Clinical Programs and Key Upcoming Milestones
Naporafenib – Pan-RAF Inhibitor

Dosing of the first patient in pivotal Phase 3 SEACRAFT-2 trial in patients with NRASm melanoma expected H1 2024
Initial signal-seeking Phase 1b efficacy data in relevant tumor types from patients with RAS Q61X solid tumors in ongoing SEACRAFT-1 trial expected between Q2-Q4 2024
ERAS-801 – CNS-penetrant EGFR Inhibitor

Phase 1 monotherapy dose escalation and expansion data in rGBM expected in 2024
ERAS-007 – ERK1/2 Inhibitor

Initial dose expansion data from Phase 1b/2 HERKULES-3 trial further evaluating encouraging early efficacy data with ERAS-007 in combination with EC in EC-naïve patients with BRAF mutant CRC expected between H2 2023 and H1 2024
Deprioritized Opportunities
Select trials or programs were deprioritized, despite their potential differentiation, to focus the company’s resources on the most promising programs, which is expected to extend the company’s cash runway from H2 2025 to H1 2026:

FLAGSHP-1 – Phase 1b combination trial of ERAS-601 SHP2 inhibitor with cetuximab was deprioritized. Though ERAS-601 achieved confirmed responses as a monotherapy and in combination with cetuximab, preliminary data do not justify further development of this combination in FLAGSHP-1 indications
ERAS-5 – Preclinical ULK inhibitor
ERAS-10 – Preclinical protein degrader

On November 28, 2023 Sermonix Pharmaceuticals Inc., a privately held biopharmaceutical company developing innovative therapeutics to specifically treat metastatic breast cancers harboring ESR1 mutations, reported that it had five abstracts accepted as poster presentations at the 2023 San Antonio Breast Cancer Symposium (SABCS), which will be held Dec. 5-9 at the Henry B. Gonzalez Convention Center in San Antonio (Press release, Sermonix Pharmaceuticals, NOV 28, 2023, View Source [SID1234638047]).

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Three posters delve into clinical data tied to Sermonix’s Evaluation of Lasofoxifene in ESR1 Mutations (ELAINE) studies. One assesses baseline genomic alterations and the activity of lasofoxifene, Sermonix’s investigational novel endocrine therapy, and Eli Lilly and Company’s CDK4/6 inhibitor abemaciclib during the ELAINE-2 study. A second poster addresses pharmacokinetics of lasofoxifene as a monotherapy and in combination with abemaciclib. The third poster is a trial-in-progress update on ELAINE-3, which is studying the efficacy and safety of lasofoxifene in combination with abemaciclib in treating locally advanced or ER+/HER2- mBC with an ESR1 mutation.

The other two posters address the results of Sermonix’s third ESR1 and Quality of Life Survey (EQUALS 3), which was developed to help inform practicing oncologists about patients’ understanding of ESR1 mutations and the quality of life for metastatic breast cancer (mBC) patients. One poster addresses patient-provider communication challenges with respect to side effects of mBC treatments, and the second discusses how minimizing treatment toxicity and side effects impacts quality of life for ER+/HER2- mBC patients.

"Sermonix looks forward to updating our peers at SABCS 2023 about our growing understanding of lasofoxifene as a monotherapy and combination therapy, our initiated ELAINE-3 study, and our continuing work to learn more about how metastatic breast cancer impacts patient quality of life," said Dr. David Portman, Sermonix founder and chief executive officer. "We are committed to investigating lasofoxifene’s efficacy in combating metastatic breast cancer, as well as observing its impacts on vaginal and sexual health, all issues that affect patients greatly."

Poster sessions details are as follows:

Poster Title: Baseline genomic alterations and the activity of lasofoxifene (LAS) plus abemaciclib (Abema) in patients with ER+/HER2- metastatic breast cancer (mBC): the ELAINE-2 study
Poster ID: P02-14-09
Date: Wednesday, Dec. 6, 2023
Time: 5-7 p.m. CT
Poster Title: Pharmacokinetics (PK) of lasofoxifene (LAS) monotherapy and combined with abemaciclib (Abema)
Poster ID: P03-18-05
Date: Thursday, Dec. 7, 2023
Time: 12-2 p.m. CT
Poster Title: Trial in progress: Open-label, randomized, multicenter, phase 3, ELAINE-3 study of the efficacy and safety of lasofoxifene plus abemaciclib for treating locally advanced or ER+/HER2- metastatic breast cancer with an ESR1 mutation
Poster ID: P01-18-12
Date: Wednesday, Dec. 6, 2023
Time: 12-2 p.m. CT
Poster Title: Patient (pt)-provider communication challenges about side effects/ from metastatic breast cancer (mBC) treatments]
Poster ID: P03-12-05
Date: Thursday, Dec. 7, 2023
Time: 12-2 p.m. CT
Poster Title: Minimization of treatment toxicity/side effects and their impact on quality of life (QoL) in patients (pts) with ER+/HER2- metastatic breast cancer (mBC)
Poster ID: P03-12-06
Date: Friday, Dec. 8, 2023
Time: 12-2 p.m. CT
To learn more about Sermonix Pharmaceuticals and lasofoxifene, visit View Source For more information about the ELAINE studies, visit View Source

About Lasofoxifene
Lasofoxifene is an investigational novel endocrine therapy in clinical development which has demonstrated robust target engagement as an ESR1 antagonist in the breast, particularly in the presence of ESR1 mutations. Lasofoxifene has demonstrated anti-tumor activity as monotherapy and in combination with a CDK4/6 inhibitor in Phase 2 studies and has unique tissue selectivity distinguishing it from other current and investigational endocrine therapies, with beneficial effects seen on vagina and bone in previous clinical studies. Lasofoxifene, which Sermonix licensed globally from Ligand Pharmaceuticals Inc. (NASDAQ:LGND), has been studied in previous comprehensive Phase 1-3 non-oncology clinical trials in more than 15,000 postmenopausal women worldwide. Lasofoxifene’s bioavailability and activity in mutations of the estrogen receptor could potentially hold promise for patients who have acquired endocrine resistance due to ESR1 mutations, a common finding in the metastatic setting and an area of high unmet medical need. Lasofoxifene’s novel activity in ESR1 mutations was discovered at Duke University and Sermonix has exclusive rights to develop and commercialize the product in this area. Lasofoxifene, a novel targeted and tissue selective oral endocrine therapy could, if approved, play a critical role in the precision medicine treatment of advanced ER+ breast cancer.

On November 28, 2023 Ribometrix, a biotechnology company developing small molecule therapeutics that modulate RNA biology, reported that it will present preclinical data showing anti-tumor benefit of eIF4E inhibition in Estrogen Receptor-positive (ER+) breast cancer at the annual San Antonio Breast Cancer Symposium, held December 5-9, 2023, in San Antonio, TX (Press release, Ribometrix, NOV 28, 2023, View Source [SID1234638045]).

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Ribometrix is evaluating its portfolio of inhibitors targeting eIF4E in preclinical studies in cancer, including melanoma and breast cancer. eIF4E is both a potent oncogene and a regulator of resistance to therapies targeting common oncogenic drivers, and Ribometrix’s data support the potential efficacy of its eIF4E inhibitors alone or in combination with standard-of-care in treatment naïve and treatment-resistant settings.

Presentation Details
Abstract Title: Pharmacological eIF4E inhibition suppresses anti-tumor activity in ER+ breast cancer
Abstract: PO5-27-09
Presenter: Matthew Friedersdorf, Ph.D., Associate Director, Translational Medicine at Ribometrix
Session: Poster Session 5
Date:  Friday, December 8, 2023
Time:  12:00pm – 2:00pm CT

At the time of presentation, Ribometrix’s poster will be made available on the "Publications" page of the "News" section of its website.

About eIF4E
Eukaryotic translation initiation factor 4E (eIF4E), the main regulatory component of cap-dependent mRNA translation, selectively promotes pro-oncogenic protein synthesis in response to activation of multiple tumor signaling pathways. Interestingly, many pro-oncogenic signaling pathways require eIF4E activity to promote tumor growth. Clinically, eIF4E activity is elevated in many tumor indications and it is typically associated with poor prognosis. Thus, targeting eIF4E has the potential to enhance anti-cancer activity when given in combination with standard-of-care. Additionally, eIF4E is also central to many resistance mechanisms, therefore eIF4E inhibition has the potential to overcome drug resistance and re-sensitize tumors to anti-cancer therapies. Ribometrix is developing eIF4E inhibitors as a promising therapeutic strategy to inhibit oncogene expression to enhance efficacy in combination and overcome resistance to targeted anti-cancer therapies.

On November 28, 2023 AstraZeneca reported that it will present new clinical and real-world data in multiple haematological conditions at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego, CA, 9 to 12 December 2023 (Press release, AstraZeneca, NOV 28, 2023, View Source [SID1234638029]).

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A total of 63 abstracts will feature 14 approved and potential new medicines across the Company’s portfolio and pipeline including from Alexion, AstraZeneca’s Rare Disease group, in chronic lymphocytic leukaemia (CLL) and several types of lymphoma, paroxysmal nocturnal haemoglobinuria (PNH), atypical haemolytic uraemic syndrome (aHUS) and amyloid light-chain (AL) amyloidosis.

Anas Younes, Senior Vice President, Haematology R&D, AstraZeneca, said: "Our data at ASH (Free ASH Whitepaper) will exemplify how we are advancing a range of innovative modalities including antibody drug conjugates, next-generation immunotherapies and T-cell engagers in haematology. Updated clinical data for AZD0486, our CD19/CD3 T-cell engager, reinforce our belief in this approach as a potential new treatment for lymphoma, and new Calquence data continue to demonstrate long-term efficacy and safety in chronic lymphocytic leukaemia with further follow up."

Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, Alexion, said: "Alexion has transformed the treatment landscape and redefined care for the paroxysmal nocturnal haemoglobinuria patient community over the past two decades. At the ASH (Free ASH Whitepaper) Annual Meeting, new results from our pivotal ALPHA trial will demonstrate the promise of Factor D inhibition to advance care for the small subset of patients with paroxysmal nocturnal haemoglobinuria who experience clinically significant extravascular haemolysis. We are proud to further our leadership in rare disease by sharing data from our robust haematology pipeline, reflecting our commitment to innovation and improving outcomes for the patients and families we serve."

Calquence continues to demonstrate long-term benefits in CLL

Six-year follow-up data from the pivotal ELEVATE-TN Phase III trial will further support the continued efficacy, safety and tolerability of Calquence for long-term use in patients with treatment-naïve CLL.1

Data from a Phase II trial will show the safety and efficacy of Calquence and rituximab followed by chemotherapy and autologous stem cell transplantation in fit patients with treatment-naïve mantle cell lymphoma (MCL).2

An analysis of five prospective Calquence trials, including three randomised, controlled Phase III trials and two non-randomised trials, will show acceptable safety outcomes based on rates of nonfatal and fatal ventricular arrhythmias and sudden death in patients with CLL.3

Novel early assets show potential to improve outcomes for blood cancer patients

Data from our early portfolio will demonstrate how the Company is advancing multiple modalities across several scientific platforms, including Immuno-Oncology, Immune-Engagers, Antibody Drug Conjugates (ADCs) and Epigenetics as part of its strategy to attack cancer from multiple angles.

Updated Phase I data for AstraZeneca’s CD19/CD3 T-cell engager, AZD0486, will further demonstrate the acceptable safety profile and high response rate of this treatment in relapsed/refractory (R/R) B-cell non-Hodgkin lymphoma (NHL).4 We will also present the first clinical data on sabestomig, a PD-1/TIM-3 targeting bispecific antibody, in R/R Hodgkin lymphoma, showing encouraging early signals of activity.5

The first preclinical data for AZD9829, a novel CD123-targeting ADC, using AstraZeneca’s proprietary linker technology to deliver a topoisomerase I inhibitor warhead, will demonstrate promising anti-tumour activity in acute myeloid leukaemia.6 In addition, preclinical data will demonstrate anti-tumour activity of AstraZeneca’s novel PRMT5 inhibitor in MTAP silenced Hodgkin lymphoma models.7

Showcasing advances to bolster our leadership in PNH with new data on Factor D inhibition and impact of C5 inhibition in long-term disease control

New results from the 24-week and long-term extension period from the pivotal ALPHA Phase III trial will reinforce the potential for danicopan add-on therapy to address clinically significant extravascular haemolysis (EVH) in the small subset of PNH patients who experience this condition while treated with C5 inhibitor therapy, allowing them to maintain control of intravascular haemolysis (IVH) through standard-of-care treatment with Ultomiris (ravulizumab) or Soliris (eculizumab).8

Further, patient-reported outcomes from the ALPHA trial will suggest danicopan as an add-on to Ultomiris or Soliris improved quality of life compared to C5 inhibitor therapy alone in patients with PNH who experience clinically significant EVH.9

Additionally, Alexion will present an analysis of six-year outcomes from the Phase III clinical trial evaluating the safety and efficacy of Ultomiris in patients with PNH who did not have previous treatment with a C5 inhibitor.10 The analysis compared survival against untreated patients in the International PNH Registry, the largest global real-world database of patients with PNH. Results will suggest Ultomiris improved survival and maintained effective long-term control of IVH, the most significant contributor to morbidity and premature mortality in PNH.10

Improving diagnosis and management of life-threatening rare diseases, including amyloidosis

24-month results of a Phase II trial will demonstrate the safety and tolerability of CAEL-101 in combination with cyclophosphamide-bortezomib-dexamethasone with or without daratumumab for the treatment of AL amyloidosis.11

Real-world analyses across AL amyloidosis, aHUS and haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) will also be presented, advancing the scientific understanding of these rare, haematological conditions.12-16

Key presentations during the 65th ASH (Free ASH Whitepaper) Annual Meeting and Exposition

Lead author

Abstract title

Presentation details

Calquence (acalabrutinib)
Sharman, JP

Acalabrutinib ± Obinutuzumab vs Obinutuzumab + Chlorambucil in Treatment-naive Chronic Lymphocytic Leukemia: 6-year Follow-up of ELEVATE-TN

Abstract # 636

Oral Session: 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Frontline Treatment with Targeted Agents in Patients with Chronic Lymphocytic Leukemia

10 December 2023

17:45 PST

Location: Seaport Ballroom ABCD (Manchester Grand Hyatt San Diego)

Westin, J

Smart Stop: Lenalidomide, Tafasitamab, Rituximab and Acalabrutinib Alone and with Combination Chemotherapy for the Treatment of Newly Diagnosed Diffuse Large B-cell Lymphoma

Abstract # 856

Oral Session: 626. Aggressive Lymphomas: Prospective Therapeutic Trials: Initial treatment strategies in Aggressive B-Cell Lymphomas

11 December 2023

15:30 PST

Location: Seaport Ballroom ABCD (Manchester Grand Hyatt San Diego)

Hawkes, EA

A Window Study of Acalabrutinib and Rituximab, Followed by Chemotherapy and Autograft (ASCT) in Fit Patients with Treatment-naïve Mantle Cell Lymphoma (MCL): First Report of the Investigator-initiated Australasian Leukaemia and Lymphoma Group NHL33 ‘WAMM’ Trial

Abstract # 735

Oral Session: 623. Mantle Cell, Follicular and Other Indolent B-Cell Lymphomas: Clinical and Epidemiological: Prospective Clinical Trials in Mantle Cell Lymphoma Incorporating Novel Agents

11 December 2023

11:00 PST

Location: Grand Hall B (Manchester Grand Hyatt San Diego)

Hawkes, EA

TrAVeRse: A Phase 2, Open-Label, Randomized Study of Acalabrutinib in Combination with Venetoclax and Rituximab in Patients with Treatment- naïve Mantle Cell Lymphoma

Abstract # 3054

Poster Session: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphomas: Clinical and Epidemiological: Poster II

10 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)

Sharman, J

Analysis of Ventricular Arrhythmias and Sudden Death with Acalabrutinib From 5 Prospective Clinical Trials

Abstract # 4643

Poster Session: 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster III

11 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)

Ferrajoli, A

Cumulative Review of Hypertension in Patients with Chronic Lymphocytic Leukemia (CLL) and Other Hematologic Malignancies Treated with Acalabrutinib: Data from Clinical Trials

Abstract # 1917

Poster Session: 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Poster I

9 December 2023

17:30 – 19:30 PST

Location: Halls G-H (San Diego Convention Center)

Sun, C

Extended Follow-Up and Resistance Mutations in CLL Patients Treated with Acalabrutinib

Abstract # 1891

Poster Session: 641. Chronic Lymphocytic Leukemias: Basic and Translational: Poster I

9 December 2023

17:30 – 19:30 PST

Location: Halls G-H (San Diego Convention Center)

Jain, P

Acalabrutinib with Rituximab as First-line Therapy for Older Patients with Mantle Cell Lymphoma – A Phase II Clinical Trial

Abstract # 3036

Poster Session: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphomas: Clinical and Epidemiological: Poster II

10 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)

Perini, G

ACRUE: A Phase 2, Open-label Study of Acalabrutinib in Combination with Rituximab in Elderly and/or Frail Patients with Treatment-naïve Diffuse Large B-Cell Lymphoma

e-Publication

Online Only

AZD0486
Gaballa, S

Double Step-Up Dosing (2SUD) Regimen Mitigates Severe ICANS and CRS While Maintaining a High Efficacy in Subjects with Relapsed/Refractory (R/R) B-cell Non-Hodgkin Lymphoma (NHL) Treated with AZD0486, a Novel CD19xCD3 T-cell Engager (TCE): Updated Safety and Efficacy Data from the Ongoing First-in-Human (FIH) Phase I Trial

Abstract # 1662

Poster Session: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphomas: Clinical and Epidemiological: Poster I

9 December 2023

17:30 – 19:30 PST

Location: Halls G-H (San Diego Convention Center)

AZD9829

Dutta, D

First Disclosure of AZD9829, a TOP1i-ADC Targeting CD123: Promising Preclinical Activity in AML Models with Minimal Effect on Healthy Progenitors

e-Publication

Online Only

AZD7789

Mei, M

Safety and Preliminary Efficacy of Sabestomig (AZD7789), an Anti-PD-1 and Anti-TIM-3 Bispecific Antibody, in Patients with Relapsed or Refractory Classical Hodgkin Lymphoma Previously Treated with Anti-PD-(L)1 Therapy

Abstract # 4433

Poster Session: 624. Hodgkin Lymphomas and T/NK cell Lymphomas: Clinical and Epidemiological: Poster III

11 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)

PRMT5 inhibitor

Urosevic, J

Epigenetic Silencing of MTAP in Hodgkin’s Lymphoma Renders it Sensitive to a 2nd Generation PRMT5 Inhibitor

Abstract # 4185

Poster Session: 605. Molecular Pharmacology and Drug Resistance: Lymphoid Neoplasms: Poster III

11 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)

Danicopan

Kulasekararaj, A

Danicopan as Add-On Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis: Phase 3 Long-term Data

Abstract # 576

Oral Session: 508. Bone Marrow Failure: Acquired: Unraveling the Future of PNH Therapy from Clinical Trials

10 December 2023

17:45 PST

Location: Room 7 (San Diego Convention Center)

Piatek, C

Patient-reported Outcomes: Danicopan as Add-On Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis

Abstract # 1346

Poster Session: 508. Bone Marrow Failure: Acquired: Poster I

9 December 2023

17:30 – 19:30 PST

Location: Halls G-H (San Diego Convention Center)

Ultomiris (ravulizumab)

Kulasekararaj, A

Ravulizumab Provides Durable Control of Intravascular Hemolysis and Improves Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Follow-Up of Study 301 and Comparisons with Patients of the International PNH Registry

Abstract # 2714

Poster Session: 508. Bone Marrow Failure: Acquired: Poster II

10 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)

Röth, A

Ravulizumab Effectiveness in the Real-world: Evidence from the International PNH Registry

Abstract # 2722

Poster Session: 508. Bone Marrow Failure: Acquired: Poster II

10 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)

Piatek, C

Efficacy and Safety of Subcutaneous Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Prior Intravenous Eculizumab: 2-Year Follow-Up

Abstract # 2713

Poster Session: 508. Bone Marrow Failure: Acquired: Poster II

10 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)

CAEL-101

Valent, J

Safety and Tolerability of CAEL-101, an Anti-Amyloid Monoclonal Antibody, Combined with Anti-Plasma Cell Dyscrasia Therapy in Patients with Light-Chain Amyloidosis: 24-Month Results of a Phase 2 Study

Abstract # 540

Oral Session: 654. MGUS, Amyloidosis and Other Non-Myeloma Plasma Cell Dyscrasias: Clinical and Epidemiological: From Light Chain to Fibril–Novel Diagnostics to Treatments for Amyloidosis

10 December 2023

13:15 PST

Location: Seaport Ballroom EFGH (Manchester Grand Hyatt San Diego)

Costello, M

CAEL-101 Enhances the Clearance of Light Chain Fibrils and Intermediate Aggregates by Phagocytosis

Abstract # 3307

Poster Session: 651. Multiple Myeloma and Plasma Cell Dyscrasias: Basic and Translational: Poster II

10 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)

AL Amyloidosis

Lyons, G

Treatment Patterns and Outcomes for Patients with Light Chain (AL) Amyloidosis: Analysis of a Large US Claims Database

e-Publication

Online Only

Thompson, J

Real-world Treatment Patterns Following Update to National Comprehensive Cancer Network Guidelines for Light-Chain Amyloidosis: Results from a US Administrative Claims Database

e-Publication

Online Only

Laires, P

Prevalence, Incidence, and Characterization of Light Chain Amyloidosis in the USA: A Real-world Analysis Utilizing Electronic Health Records (EHR)

e-Publication

Online Only

aHUS

Wang, Y

Patient Characteristics and Diagnostic Journey of Thrombotic Microangiopathy Associated with a Trigger: A Real-world, Retrospective, Multi-national Study

e-Publication

Online Only

HSCT-TMA

Wang, Y

Real-world Analysis of the Underdiagnosis, Clinical Outcomes and Associated Burden of Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (HSCT-TMA) in the United States of America

Abstract # 491

Oral Session: 904. Outcomes Research – Non-Malignant Conditions: What to Know: Management Costs and Outcomes in Various Non-Malignant Disorders

10 December 2023

10:30 PST

Location: Pacific Ballroom Salons 15-17 (Marriott Marquis San Diego Marina)

PNH

Wagner-Ballon, O

Neutrophil PNH Type II Cells Are Associated with Thrombosis and Bone Marrow Failure Without Hemolysis: Evidence from Analysis of the 5-year French Nation-Wide Multicenter Observational Study

Abstract # 4083

Poster Session: 508. Bone Marrow Failure: Acquired: Poster III

11 December 2023

18:00 – 20:00 PST

Location: Halls G-H (San Diego Convention Center)