Study shows protein inhibitor as potential treatment approach for common mutations found in non-Hodgkin lymphomas

On January 8, 2020 A study at The University of Texas MD Anderson Cancer Center reported a potential new approach to treating two of the most common subtypes of lymphoma through manipulation of molecular programs controlled by the cAMP-response element binding protein (CREBBP) (Press release, MD Anderson, JAN 8, 2020, View Source [SID1234553265]). Mutations of CREBBP are frequently found in follicular lymphoma and diffuse large B-cell lymphomas (DLBCL), and allow malignant cells to hide from the immune system.

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Study results were published in the Jan. 8 online issue of Cancer Discovery. Co-lead investigators, Michael Green, Ph.D., assistant professor of Lymphoma & Myeloma at MD Anderson and Ari Melnick, M.D., of Weill Cornell Medical School, reported on how inhibition of a protein called histone deacetylase 3 (HDAC3) restores immune programs lost as a result of CREBBP mutations, paving the way for potential immunotherapy approaches for common forms of non-Hodgkin lymphoma.

CREBBP is the second most frequently mutated chromatin-modifying gene in both follicular lymphoma and DLBCL. It encodes a protein that alters the activity of genes by modifying the histone proteins around which DNA is wrapped.

"CREBBP mutations are highly recurrent in B-cell lymphomas and either inactivate its histone acetyltransferase (HAT) domain or truncate the protein," said Green." We showed that these two classes of mutations yield different degrees of disruption of the epigenome, with HAT mutations being more severe and associated with inferior clinical outcome."

Through CRISPR/Cas9 gene editing of cell lines and using mouse models, the research team also showed that HDAC3 selective inhibitors reverse aberrant epigenetic programming caused by CREBBP resulting in growth inhibition of lymphoma cells and restoration of immune surveillance.

"Our study characterized the molecular consequences of CREBBP mutations and identified key cellular pathways silenced as a result of unopposed HDAC3 activity," said Green. "We demonstrated how inhibition of HDAC3 restores these pathways, suppressing growth and most critically enabling T cells to recognize and kill lymphoma cells."

HDAC3 inhibitors appear to affect expression of major histocompatibility molecular class II (MHC class II), molecules, which are antigen presentation proteins crucial for initiating adaptive immune responses.

"The frequency of MHC class II loss in DLBCL exceeds the frequency of CREBBP mutations in this disease through unknown mechanisms," said Green. "The ability of HDAC3 inhibition to induce MHC class II expression may have potentially broad implications for immunotherapy. We believe that inhibition of HDAC3 represents a novel mechanism-based immune-epigenetic therapy for CREBBP- mutant lymphomas."

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The study was funded by the National Institutes of Health (R01 CA201380, R01 CA055349, U54 OD020335 01, P50 CA192937, P30 CA016672, and P30 CA008748); the Chemotherapy Foundation; the Star Cancer Consortium; the Jaime Erin Follicular Lymphoma Research Consortium; the Schweitzer Family Fund; and the Futcher Foundation. The study was also supported with funding from the B-Cell Lymphoma Moon Shot, part of MD Anderson’s Moon Shots Program, a collaborative effort to accelerate the development of scientific discoveries into clinical advances that save patients’ lives. Green previously served on the Scientific Advisory Board of KDAc Therapeutics, and he owns stock equity in the company.

MD Anderson study participants included Saber Tadros, Ph.D.; Neeraj Jain, Ph.D.; Haopeng Yang, Ph.D.; Man Chun John Ma, Ph.D.; Sreejoyee Ghosh, Ph.D.; Loretta Nastoupil, M.D.; and Sattva Neelapu, M.D., all of the Department of Lymphoma & Myeloma; and Cassian Yee, M.D., of the Department of Melanoma Medical Oncology.

UroGen to Present at 38th Annual J.P. Morgan Healthcare Conference

On January 8, 2020 UroGen Pharma Ltd. (Nasdaq: URGN), a biopharmaceutical company dedicated to building novel solutions that treat specialty cancers and urologic diseases because patients deserve better options, reported that Liz Barrett, President and Chief Executive Officer, will present at the 38th Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2020 at 1:30 PM Pacific Time (Press release, UroGen Pharma, JAN 8, 2020, View Source [SID1234552865]). The event will be held in San Francisco at the Westin St. Francis.

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A live audio webcast of the event will be available on the Investors section of UroGen’s website, www.urogen.com. A replay of the webcast will be available on the website for approximately two weeks.

ArcherDX to Present at 38th Annual J.P. Morgan Healthcare Conference

On January 8, 2020 ArcherDX, Inc., a molecular diagnostics company dedicated to developing breakthrough solutions that advance the application of personalized genomic medicine, reported that Jason Myers, Ph.D., Chief Executive Officer, will present an overview of the company and its products at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco, California (Press release, ArcherDX, JAN 8, 2020, View Source [SID1234552881]).

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Event:

38th Annual J.P. Morgan Healthcare Conference

Presenter:

Jason Myers, Ph.D., Chief Executive Officer, ArcherDX

Date:

Tuesday, January 14, 2020

Time:

9:00 a.m. Pacific Time

Dr. Myers will outline ArcherDX’s plans to introduce IVD test kits for pan-solid tumor companion diagnostics (STRATAFIDE) and personalized cancer monitoring, which, once approved, will enable individualized genomic cancer treatment to be practiced at any level of the health care system, from academic centers to community practices. He will also provide an update on the growing demand for on-site and local genomic testing for both research and clinical applications.

Vertex to Present at the J.P. Morgan Healthcare Conference on January 13

On January 8, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that management will present at the J.P. Morgan Healthcare Conference on Monday, January 13, 2020 at 2:30 p.m. ET (11:30 a.m. PT) (Press release, Vertex Pharmaceuticals, JAN 8, 2020, View Source [SID1234552934]).

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The audio portion of management’s remarks can be accessed live through Vertex’s website, www.vrtx.com, in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company’s website.

Almirall enters into an option agreement to acquire Bioniz Therapeutics and to establish a broad research agreement to further expand its innovative pipeline in medical dermatology

On January 8, 2020 Almirall, S.A. (ALM) has reported the execution of an option agreement to acquire Bioniz Therapeutics, Inc. a clinical stage biopharmaceutical company based in Irvine, California, which develops first-in-class peptide treatments that selectively inhibit multiple cytokines to treat immuno-inflammatory diseases and T-cell malignancies (Press release, Almirall, JAN 8, 2020, View Source;articleId=4224566&id=4224567 [SID1234553266]). If Almirall exercises the option, it will also enter into a broader research agreement with Bioniz NewCo, using its multiple cytokine inhibitor platform with the objective to deliver at least 3 IND-approved candidates.

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Through this collaboration, Almirall executes its strategy to develop and expand its R&D pipeline with new treatment modalities with the objective to address highly underserved diseases within oncodermatology and immunodermatology.

Under the terms of the agreement, Almirall will strengthen its medical dermatology pipeline. If Almirall opts-in, Almirall will have access to a clinical stage asset in development for several indications and will start a research collaboration to expand the early stage pipeline:

BNZ-1, a novel multicytokine inhibitor in development for: i) refractory Cutaneous T-cell Lymphoma (CTCL), an orphan disease with high unmet medical need and ii) alopecia areata, an autoimmune hair disorder with no approved treatments.
Research collaboration using Bioniz multiple cytokine inhibitor platform technology with the objective to deliver at least 3 IND-approved candidates.
During the option collaboration period, Bioniz will complete the current ongoing phase 1/2 clinical trial in CTCL and the parties will collaborate to define the future CTCL development activities. After opting-in, Bioniz will spin off other assets than BNZ-1 as well as the proprietary platform technology to a NewCo. and Almirall will continue with the development of BNZ-1.

Under the terms of the agreement, Almirall will make an initial payment of $15 MM to Bioniz in exchange for an option to acquire all Bioniz outstanding shares. Following the availability of phase 1/2 results in CTCL, certain human biomarkers laboratory data and the official FDA End of Phase 2 meeting minutes, Almirall will have 60 days to exercise its option. If Almirall elects to exercise its option, the company will pay an option exercise fee of $47 MM in different instalments in the following years. Almirall will make additional payments upon the achievement of certain development, regulatory and commercial milestones.

Bhushan Hardas, MD, MBA, Almirall’s Chief Scientific Officer, commented that "this agreement is a big step forward for us in our objective of becoming a leader in Onco-dermatology and Immuno-dermatology. With Bioniz Therapeutics Inc. combined with our long experience and understanding in the area of dermatology, we will be able to identify new avenues with the potential to develop the right therapies for patients with high unmet needs".

Nazli Azimi, Founder and Chief Executive Officer of Bioniz Therapeutics, Inc. stated "we are delighted to enter into this agreement with Almirall, a global leader in medical dermatology who shares our vision of building an Immuno-dermatology franchise for BNZ-01. This partnership gives prominence to our leadership position in the discovery and development of first-in-class multi-cytokine inhibitor peptides to treat immuno-inflammatory diseases and cancer."

About BNZ-01

Bioniz’s lead development candidate, BNZ-01, is a PEGylated peptide that functions as a selective inhibitor of cytokines IL-2, IL-9, and IL-15. Bioniz Therapeutics is developing BNZ-01 for the treatment of CTCL and autoimmune diseases (Alopecia Areata). BNZ-01 has been evaluated in Ph1 Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) and is currently being evaluated in a Phase 1/2a study in CTCL patients and ready to start a Phase 2 PoC study in Alopecia Areata.