On November 19, 2020 Novartis announced today that new research data from a broad range of hematology medicines and investigational therapies will be presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, taking place virtually December 5-8 (Press release, Novartis, NOV 19, 2020, View Source [SID1234571414]). More than 65 abstracts from Novartis-sponsored and investigator-initiated trials that include results for asciminib (ABL001), Kymriah (tisagenlecleucel), Jakavi (ruxolitinib)*, sabatolimab (MBG453) and Adakveo (crizanlizumab) underscore the Novartis vision to deliver transformative innovation to address unmet medical needs.

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"Our research and development strategy focuses on developing transformative treatments with the aspiration of dramatically improving quality of life and addressing the underlying disease process," said Susanne Schaffert, PhD, President, Novartis Oncology. "The ASH (Free ASH Whitepaper) presentations demonstrate how we are pursuing these goals in hematology with research that focuses on developing advanced therapeutic approaches across an array of blood cancers and difficult-to-treat hematologic diseases."

Key highlights of data accepted by ASH (Free ASH Whitepaper):

Novel, investigational STAMP inhibitor asciminib (ABL001) evaluated for safety and efficacy for TKI-resistant and intolerant CML patients:

Efficacy and Safety Results From ASCEMBL, a Multicenter, Open-label, Phase 3 Study of Asciminib vs Bosutinib (BOS) in Patients (Pts) with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Previously Treated with ≥2 Tyrosine Kinase Inhibitors (TKIs) [Abstract #LBA-4; oral presentation; Tuesday, December 8, 8:15 AM PST]
Asciminib, a First-in-Class STAMP Inhibitor, Provides Durable Molecular Response in Patients (Pts) With Chronic Myeloid Leukemia (CML) Harboring the T315I Mutation: Primary Efficacy and Safety Results From a Phase 1 Trial [Abstract #650; oral presentation: Monday, December 7, 12:15 PM PST]
Structural and Biochemical Studies Confirming the Mechanism of Action of Asciminib, an Agent Specifically Targeting the ABL Myristoyl Pocket (STAMP) [Abstract #3961; online publication]
New data for the first anti-TIM-3 antibody in hematology, sabatolimab (MBG453):

Efficacy and Safety of Sabatolimab (MBG453) in Combination With Hypomethylating Agents (HMAs) in Patients With Acute Myeloid Leukemia (AML) and High-risk Myelodysplastic Syndrome (HR-MDS): Updated Results From a Phase 1b Study [Abstract #657; oral presentation: Monday, December 7, 12:30 PM PST]
Sabatolimab (MBG453) Dose Selection and Dose-Response Analysis in Myelodysplastic Syndrome (MDS)/Acute Myeloid Leukemia (AML): Population Pharmacokinetics (PK) Modeling and Evaluation of Clinical Efficacy/Safety by Dose [Abstract #2192; poster presentation: Sunday, December 6, 7:00 AM PST]
Kymriah (tisagenlecleucel) results from the first analysis of the Phase II ELARA trial in r/r follicular lymphoma and a clinical update of 40-month median follow-up from the pivotal JULIET trial in r/r diffuse large B-cell lymphoma (DLBCL):

Efficacy and Safety of Tisagenlecleucel in Adult Patients With Relapsed/Refractory Follicular Lymphoma: Interim Analysis of the Phase 2 ELARA Trial [Abstract #1149; poster presentation: Saturday, December 5, 7:00 AM PST]
Myc Expression and Tumor-Infiltrating T Cells Are Associated With Response in Patients (Pts) With Relapsed/Refractory Diffuse Large B-Cell Lymphoma (r/r DLBCL) Treated With Tisagenlecleucel in the JULIET Trial [Abstract #1194; poster presentation: Saturday, December 5, 7:00 AM PST]
Adakveo (crizanlizumab) results from the SOLACE trial and a post-hoc analysis of the SUSTAIN trial for vaso-occlusive pain crises in sickle cell disease, and extended results from the Sickle Cell World Assessment Survey (SWAY):

Pharmacokinetics/Pharmacodynamics, Safety and Efficacy of Crizanlizumab in Patients With Sickle Cell Disease and a History of Vaso-Occlusive Crises: Results From the Phase II, Multicenter, Open-Label SOLACE-Adults Study [Abstract #1715; poster presentation: Sunday, December 6, 7:00 AM PST]
The Effect of Crizanlizumab on the Number of Days Requiring Opioid Use for Management of Pain Associated With Vaso-Occlusive Crises in Patients With Sickle Cell Disease: Results From the SUSTAIN Trial [Abstract #796; poster presentation: Saturday, December 5, 7:00 AM PST]
Global Treatment Satisfaction Levels and Treatment Patterns From the International Sickle Cell World Assessment Survey (SWAY): Hydroxyurea (HU) Versus No HU [Abstract #17; oral presentation: Saturday, December 5, 8:30 AM PST]
Primary findings from the REACH3 trial for steroid-refractory chronic graft-vs-host disease (GvHD) and additional findings from REACH2 for steroid-refractory acute GvHD treated with Jakavi (ruxolitinib)*:

Ruxolitinib vs Best Available Therapy in Patients With Steroid-Refractory/Steroid-Dependent Chronic Graft-vs-Host Disease: Primary Findings From the Phase 3, Randomized REACH3 Study [Abstract #77; oral presentation: Saturday, December 5, 8:00 AM PST]
Biomarker Analysis in Patients With Steroid-Refractory Acute Graft vs Host Disease Treated With Ruxolitinib or Best Available Therapy in the Randomized, Phase 3 REACH2 Study [Abstract #1519; poster presentation: Saturday, December 5, 7:00 AM PST]
Safety Analysis of Ruxolitinib (RUX) vs Best Available Therapy in Patients With Steroid-Refractory Acute Graft-vs-Host Disease in the Randomized Phase 3 REACH2 Study [Abstract #2440; poster presentation: Sunday, December 6, 7:00 AM PST]
Early pipeline results for ianalumab (VAY736) in chronic lymphocytic leukemia:

Phase Ib Study of Ianalumab (VAY736) and Ibrutinib in Patients With Chronic Lymphocytic Leukemia (CLL) on Ibrutinib Therapy [Abstract #1309; poster presentation: Saturday, December 5, 7:00 AM PST]
Product Information
Approved indications for products vary by country and not all indications are available in every country. Safety and efficacy profiles have not been established for investigational compounds or are outside the approved indications for marketed products. Because of the uncertainty of clinical trials, there is no guarantee that compounds will become commercially available or receive additional indications if already marketed.

On November 19, 2020 Varian (NYSE: VAR) and the Cincinnati Children’s/UC Health Proton Therapy Center reported the start of the first clinical trial of FLASH therapy as part of the recently opened FAST-01 study (FeAsibility Study of FLASH Radiotherapy for the Treatment of Symptomatic Bone Metastases) (Press release, Varian Medical Systems, NOV 19, 2020, View Source [SID1234571413]). The clinical trial involves the investigational use of Varian’s ProBeam particle accelerator modified to enable radiation therapy delivery at ultra-high dose rates (dose delivered in less than 1 second) and is being conducted at the Cincinnati Children’s/UC Health Proton Therapy Center with John C. Breneman M.D., Medical Director of the center, serving as principal investigator.

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The first clinical trial patient was treated this week. The FAST-01 study is expected to enroll up to 10 patients with bone metastases to evaluate clinical workflow feasibility, treatment-related side effects, and efficacy of treatment as assessed by measuring pain relief of trial participants. The clinical trial, informed by years of preclinical work, was designed by experts at Varian and multiple centers in the FlashForwardTM Consortium, including Cincinnati’s Children’s/UC Health Proton Therapy Center and the New York Proton Center.

Varian FLASH Cincinnati

Varian FLASH Cincinnati
"Treating the first patient in this FLASH clinical trial is a milestone that many thought was still years ahead of us," said Kolleen Kennedy, Chief Growth Officer and President of Proton Therapy Solutions at Varian. "There was overwhelming support from Dr. Breneman and his team for this clinical trial, which was designed in collaboration with the FlashForward Consortium and with significant contributions from the New York Proton Center. These efforts help Varian safely advance potential therapy options towards our vision of a world without fear of cancer."

Breneman noted that, because this is the first in human trial of FLASH radiotherapy, it will build a foundation for extending this therapy to other types of cancer treatments.

"Trials using FLASH radiotherapy for lung cancer and other malignancies are currently being developed," said Breneman, a UC Health radiation oncologist and a professor emeritus at the University of Cincinnati College of Medicine. "Using FLASH treatment for these cancers could deliver higher cancer-killing doses without causing inordinate side effects, which would be a real advance."

"FLASH therapy has the potential to be practice changing and dramatically improve the experience of cancer care for a new generation of patients. The launch of the first FLASH clinical trial, a project that has come to fruition after years of intensive study, is an important milestone in the progress of radiation therapy," said FlashForward Consortium member Dr. Charles B. Simone, II, FACRO, Chief Medical Officer at New York Proton Center. "We are optimistic that the results of the FAST clinical development program will transform the way the industry approaches treatment. The New York Proton Center is proud to be a partner in this future-focused study."

John Perentesis, MD, Director of the Division of Oncology & Cancer Programs at Cincinnati Children’s, said FLASH is potentially a transformational advance for cancer treatment for many patients."If the side effects of radiation on the normal tissues surrounding a tumor can be significantly reduced, the dose of radiation to treat a cancer can be greatly increased," Perentesis said. "This would raise hope to cure malignancies that respond to radiation but aren’t completely cured at current doses, including pediatric brain tumors like DIPG/pontine glioma and medulloblastoma, sarcomas, and neuroblastoma."

On November 19, 2020 OBI Pharma, Inc. (TPEx: 4174), a leader in Glycosphingolipid Immuno-Oncology therapeutics targeting the Globo Series antigens (Globo H and SSEA-4), reported that data from the OBI-833 Phase 1 clinical study targeting the Globo H antigen in lung cancer will be presented at the European Society of Clinical Oncology Asia (ESMO Asia) 2020 Virtual Scientific Program from November 20-22, 2020 (Press release, OBI Pharma, NOV 19, 2020, View Source [SID1234571406]).

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These results will be presented by the lead investigators of OBI Pharma’s novel anti-Globo H therapeutic cancer vaccine, OBI-833.

"Based upon our anti-Globo H targeted approaches in cancers of high unmet needs, OBI Pharma is proud to have presentations on the progress of our trial presented at ESMO (Free ESMO Whitepaper)-Asia 2020 for our novel therapeutic cancer vaccine, OBI-833." Ming-Tain Lai, PhD, Chief Scientific Officer at OBI Pharma stated, "In the trial, OBI-833 demonstrated a favorable safety profile and generated detectable anti-Globo H IgM/IgG responses. In addition, OBI-833 can elicit a beneficial immune response in NSCLC patients and had rendered some TKI-treated patients a durable stable disease status. We look forward to providing updates of our study, which we believe could offer potential therapeutic benefits to patients suffering from lung cancer."

1. Presentation number: 397P / Poster: ID 680

Title: A phase I cohort expansion trial of OBI-833 in non-small cell lung cancer patients

Presenter: Ching-Liang Ho MD, et.al. Department of Internal Medicine, Tri-Service General Hospital, Taipei, Taiwan.

Session Title: E-poster Display session – Thoracic tumours, metastatic

Session Date and Time: Friday, November 20, 2020. 9:00 – 20:00 Eastern Time

View Source

2. Presentation number: 71MO / Oral: ID 798

Title: OBI-833 was safe and immunogenic, without treatment-related SAEs, in a Phase 1 dose-escalation trial

Presenter: Her-Shyong Shiah, MD*., et.al. Department of Hematology and Oncology, Taipei Medical University Hospital, Taipei, Taiwan

*current affiliation: Department of Hematology and Oncology, Taipei Tzu Chi Hospital, Taipei, Taiwan

Session Title: Mini-oral session – Developmental and Precision Medicine

Session Date and Time: Friday, November 20, 2020. 19:52 – 19:57 Eastern Time

View Source

The above poster presentations will be available online at www.obipharma.com on November 23, 2020

On November 19, 2020 Achilles Therapeutics ("Achilles"), a clinical-stage biopharmaceutical company developing personalised T cell therapies targeting clonal neoantigens, a novel class of tumour target, reported it has raised £52.7 million in an oversubscribed Series C financing (Press release, Achilles Therapeutics, NOV 19, 2020, View Source [SID1234571405]). OrbiMed, Boxer Capital of Tavistock Group, and other prominent US-based, healthcare-focused institutional investors join existing investors including RA Capital, Syncona, Forbion, Invus, Perceptive Advisors and Redmile Group.

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Proceeds from this financing will be used to accelerate the Company’s R&D activities and further build the clinical network to support the Company’s ongoing Phase I/IIa trials, the THETIS trial in patients with recurrent or metastatic malignant melanoma and the CHIRON trial in patients with advanced non-small cell lung cancer. Both clinical trials use the Company’s innovative personalised T cell therapy approach targeting clonal neoantigens and are currently enrolling patients with interim data expected from both studies in the first half of 2021. In addition, the financing will enable the continued build out of Achilles’ manufacturing capabilities and broaden its growing pipeline of solid tumour pre-clinical product candidates.

"Achilles has made tremendous progress since its founding in 2016, and with this financing round we are further strengthening our outstanding syndicate with more leading US healthcare investors. I am delighted with this strong support for our innovative platform, team, and ambitious development strategy," said Dr. Iraj Ali, CEO of Achilles Therapeutics. "As we progress our two lead programmes in non-small cell lung cancer and melanoma through clinical trials, we believe that our personalised T cell therapy approach has the potential to transform how certain cancers are treated, bringing much needed novel cancer therapies to patients."

On November 19, 2020 AstraZeneca reported that it will present new research aimed at addressing key unmet needs facing patients with blood cancers at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, held virtually from 5 to 8 December 2020 (Press release, AstraZeneca, NOV 19, 2020, View Source [SID1234571404]).

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The Company will present 27 abstracts spanning five medicines and potential new medicines and eight different haematology conditions that demonstrate the Company’s commitment to advancing haematology research and treatments for patients living with haematologic malignancies.

Key data presentations include:

A pooled analysis of data from four trials – ELEVATE TN, ASCEND, ACE-CL-001 and 15-H-0016 – expanding on the cardiovascular safety profile of Calquence monotherapy treatment for patients with chronic lymphocytic leukaemia (CLL)
Extended follow-up data from the pivotal Phase II ACE-LY-004 trial that support long-term treatment with Calquence in adult patients with relapsed or refractory mantle cell lymphoma (MCL)
Data on Calquence in combination with venetoclax and either obinutuzumab or rituximab in patients with CLL, showing a safety profile consistent with previous trials with high complete responses and undetectable minimal residual disease rates after a median follow-up of 26.9 months, with minimal to no drug-drug interactions in the Phase Ib ACE-CL-003 trial
First-in-human data from the potential new medicine B-cell maturation antigen (BCMA)-targeted antibody drug conjugate, MEDI2228, presenting data on safety and efficacy at all dose levels in relapsed or refractory multiple myeloma
Data showing pre-clinical evidence of overcoming resistance in relapsed or refractory MCL from the dual BCL2/XL inhibitor, AZD4320, which blocks the anti-apoptotic effect of BCL2 and BCLXL
Phase I data from the anti-inducible co-stimulator anti-ICOS monoclonal antibody, MEDI-570, demonstrating promising early clinical activity in poor-risk refractory and heavily pre-treated patients with angioimmunoblastic T-cell lymphoma
Multiple studies on roxadustat, the first in a new class of medicines evaluating its clinical effectiveness and safety profile in both the dialysis dependent and non-dialysis dependent anaemia of CKD patient populations
Data on roxadustat assessing efficacy in anaemia secondary to lower-risk myelodysplastic syndrome (MDS) regardless of baseline factors. In approximately one in three patients MDS leads to acute myeloid leukaemia.1
Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: "Our data at ASH (Free ASH Whitepaper) this year continue to support Calquence as a well-tolerated treatment with impressive efficacy and safety across multiple blood cancers, reinforcing physicians’ confidence in treating patients with Calquence over the long term. Data at the meeting will also explore Calquence combinations with commonly used therapies, showing potential across a variety of regimens in chronic lymphocytic leukaemia to best suit the unique needs of each patient."

José Baselga, Executive Vice President, Oncology R&D, said: "As we rapidly expand our presence in haematology, we are focused on identifying novel targets and mechanisms of action that can address the most urgent unmet needs across various haematological malignancies. Our early portfolio at this year’s ASH (Free ASH Whitepaper) clearly demonstrates our commitment to following the science in combating treatment-resistant and rare blood cancers."

Key AstraZeneca presentations during the 62nd ASH (Free ASH Whitepaper) Annual Meeting and Exposition

Key AstraZeneca presentations during the 62nd ASH (Free ASH Whitepaper) Annual Meeting and Exposition
Calquence

Calquence (acalabrutinib) is a next-generation, selective inhibitor of Bruton’s tyrosine kinase (BTK). Calquence binds covalently to BTK, thereby inhibiting its activity.2,3 In B-cells, BTK signalling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis, and adhesion.2

As part of an extensive clinical development programme, AstraZeneca and Acerta Pharma are currently evaluating Calquence in more than 20 company-sponsored clinical trials. Calquence is being developed for the treatment of multiple B-cell blood cancers including CLL, MCL, diffuse large B-cell lymphoma, Waldenström macroglobulinaemia, follicular lymphoma, and other haematologic malignancies.

Roxadustat

Roxadustat is a first-in-class oral small molecule hypoxia-inducible-factor prolyl hydroxylase inhibitor (HIF-PHI) that promotes erythropoiesis through increased endogenous production of erythropoietin; improved iron absorption, transport and mobilisation; and downregulation of hepcidin, which helps to overcome the negative impact of inflammation on haemoglobin synthesis and red blood cell production. Roxadustat is approved in China for the treatment of anaemia in adult patients with CKD, both on dialysis and not on dialysis. In Japan, roxadustat is approved for the treatment of anaemia in CKD patients on dialysis, and a supplemental New Drug Application (NDA) for the treatment of anaemia in CKD patients not on dialysis is under regulatory review. The roxadustat NDA for the treatment of anaemia in CKD in both NDD and DD is under review by the US Food and Drug Administration with a decision expected in Q4 2020. The Marketing Authorisation Application for roxadustat for the treatment of anaemia in CKD in both NDD and DD was filed by Astellas and accepted by the European Medicines Agency for review on 21 May 2020. Roxadustat is also in clinical development for anaemia associated with MDS and for chemotherapy-induced anaemia.

AstraZeneca and FibroGen Inc. (FibroGen) are collaborating on the development and commercialisation of roxadustat for the potential treatment of anaemia in the US, China and other markets in the Americas and in Australia/New Zealand, as well as Southeast Asia. Astellas and FibroGen are collaborating on the development and commercialisation of roxadustat for the potential treatment of anaemia in territories including Japan, Europe, the Commonwealth of Independent States, the Middle East and South Africa.

AstraZeneca in haematology

Leveraging its strength in oncology, AstraZeneca has established haematology as one of four key oncology disease areas of focus. The Company’s haematology franchise includes two medicines approved by the US Food and Drug Administration and a robust global development programme for a broad portfolio of potential blood cancer treatments. Acerta Pharma serves as AstraZeneca’s haematology research and development arm. AstraZeneca partners with like-minded science-led companies to advance the discovery and development of therapies to address unmet need.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With seven new medicines launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers.

By harnessing the power of six scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response, Antibody Drug Conjugates, Epigenetics, and Cell Therapies – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.