Exploring the therapeutic potential of 3D intestinal organoid cell models for adenomatous polyposis syndromes

On September 2, 2020 Cellesce reported that partnership with the Inherited Tumour Syndromes Research Group at Cardiff University, has won support for further organoid research from the Accelerate programme through the programme’s Clinical Innovation Accelerator (CIA) (Press release, Cellesce, SEP 2, 2020, View Source [SID1234564271]). Accelerate is a pioneering collaboration between Welsh universities and the Life Sciences Hub Wales. Working with industry, it helps translate innovative ideas into new technology, products, and services for the health and care sector. The Accelerate programme is supported by the Welsh Government and the European Regional Development Fund. This research will focus on establishing an organoid based model for the pre-clinical testing of drugs for Familial Adenomatous Polyposis (FAP) and MUTYH-associated polyposis (MAP) patients.

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FAP and MAP are genetic syndromes which predispose patients to a near-100% lifetime risk of colorectal cancer. This project aims to establish a pre-clinical organoid model of the syndromes, in vitro, to target the drivers of the disease. Cellesce will be working with Laura Thomas and her team, who are based in the Cardiff University Cancer Genetics building at the University Hospital of Wales, Heath Park. The aim is to derive organoids from tissue donated by patients with FAP and MAP and grow them at scale. These intestinal organoids will contain all the different cell types found in the original organ and recreate the spatial organisation of the original tissue. They will be used as pre-clinical models of intestinal tumour initiation in order to identify the genetic mechanisms of the disease, to test potential drugs for the treatment of FAP and MAP and for developing therapeutic strategies for the prevention of inherited and sporadic intestinal cancers. These organoid models will complement Cellesce’s existing range ofcolorectal cancer organoids, providing models at both early and late stages of intestinal cancer development made commercially available under license terms already agreed.

With support from CIA, the academic team, led by Professor Julian Sampson, will bring in their expertise of biology/medicine, genomics and bioinformatics, and experience in governance and accreditation under the Human Tissue Act (HTA). This will be complemented by Cellesce’s scientific knowledge, experience in organoid culture, bioengineering technology and expertise in the expansion of organoids. The project will also benefit from the project team’s links with the NHS including access to donated patient tissue through the Wales Cancer Bank and support from geneticists, gastroenterologists and surgeons.

"The Accelerate support won with Cellesce enables the Cardiff University Inherited Tumour Syndrome Research Group to build upon its track record of genetic discovery research and translation of its findings to pre-clinical and clinical trials of novel targeted treatment," said Professor Julian Sampson. "This presents a fantastic opportunity to improve care for patients with inherited tumour syndromes and to further develop innovation in precision medicine in Wales." The project started in October 2019 and is expected to run until March 2021.

Patient-derived breast cancer organoids to transform drug discovery screening assays

On September 2, 2020 Cellesce reported that this award supports a collaborative project that aims to produce organoids from breast cancer patient biopsies, and grow those organoids at scale to enable the discovery of new breast cancer therapies (Press release, Cellesce, SEP 2, 2020, View Source [SID1234564270]). This project will build on the expertise of the team that has already successfully established lines of colorectal cancer organoids.

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One of the major hurdles of discovering new and effective cancer treatments is the availability of good representative "model" systems which can be used to test large numbers of candidate therapeutic compounds, since cells in a petri dish frequently don’t behave as they would in the body. By growing cells in 3D, it is possible to recreate much more of the complexity that is seen in patients’ tumours and therefore, get a better idea of how effective new therapies will be. The team is using donated tumour tissue, taken from patients in hospital, to grow organoids. By growing tumours as organoids from many different patients, we aim to derive organoid models from all the major breast cancer sub-types, covering the whole range of tumour types seen in humans.

This project will allow us to extend the range of cancer tissues that we can offer at scale, and in turn enable more pharmaceutical companies to embrace organoid technology. We believe this will provide our customers with a more robust, relevant and cost-effective model for screening new drug compounds.

The project has also opened up the opportunity to work with tissues taken from Patient-derived xenograft (PDX) models of cancer, where the tissue or cells from a patient’s tumour are implanted and propagated in immunodeficient or humanised mice.

The project started in July 2018 and is due to complete in late 2020. Carried out in collaboration with Cardiff University, it will lead to Cellesce being able to license and make a number of breast cancer organoid lines commercially available under license terms already agreed.

IGM Biosciences to Present at Four Upcoming Investor Conferences

On September 2, 2020 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported that Fred Schwarzer, Chief Executive Officer, will present at four upcoming investor conferences (Press release, IGM Biosciences, SEP 2, 2020, View Source [SID1234564269]):

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Baird 2020 Global Healthcare Conference on Wednesday, September 9 at 3:10 p.m. ET.
H.C. Wainwright 22nd Annual Global Investment Conference on Wednesday, September 16 at 1:00 p.m. ET.
2020 Cantor Global Virtual Healthcare Conference on Thursday, September 17 at 4:40 p.m. ET.
Morgan Stanley 18th Annual Global Healthcare Conference on Friday, September 18 at 11:45 a.m. ET.
A live webcast of the events will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcasts will be archived on the Company’s website for 90 days following the event.

Bicycle Therapeutics Announces First Patient Dosed in Phase IIa Trial of Bicycle® Toxin Conjugate BT1718

On September 2, 2020 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported that the first patient has been dosed in the Phase IIa expansion portion of the Phase I/IIa trial sponsored by Cancer Research UK, the world’s leading cancer charity dedicated to saving lives through research (Press release, Bicycle Therapeutics, SEP 2, 2020, View Source [SID1234564268]). The trial is evaluating BT1718 in patients with MT1-MMP-positive squamous non-small cell lung cancer (NSCLC) and a basket of other MT1-MMP-positive solid tumors.

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"With the Phase IIa expansion, we look forward to building on the encouraging data generated through the Phase I dose escalation, in which BT1718 appeared tolerable with manageable adverse events and preliminary signs of anti-tumor activity were observed," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "BT1718 is a Bicycle Toxin Conjugate, or BTC, that targets the antigen MT1-MMP, which was observed in our translational research to be highly expressed on the membrane of tumors of a squamous cell origin. Squamous NSCLC represents a key patient segment where there is a high unmet need for new treatment options, given the limitations of current therapies. We believe BT1718 represents a potential first-in-class therapy that could have significant potential in the treatment of squamous NSCLC, as well as more broadly across other MT1-MMP-expressing cancers with high unmet need."

BT1718 is a BTC targeting MT1-MMP and is comprised of a mono-hindered disulfide cleavable linker and a cytotoxic DM1 payload. The Phase IIa expansion will evaluate BT1718 administered once weekly at the recommended Phase II dose (RP2D) of 20 mg/m2 in an MT1-MMP-positive squamous NSCLC cohort, as well as in an MT1-MMP-positive solid tumor "basket" cohort. For the first time in the Phase I/IIa trial, patients will be preselected based on levels of antigen expression in target lesions, as determined by the Company’s proprietary clinical-grade MT1-MMP immunohistochemistry (IHC) assay. The primary objectives of the Phase IIa expansion portion are to evaluate the safety and tolerability profiles of BT1718. One of the secondary objectives is to assess anti-tumor response, as measured by RECIST criteria. The trial is sponsored by Cancer Research UK’s Centre for Drug Development and will be conducted at sites in the UK.

Dr. Nigel Blackburn, Cancer Research UK’s Director of Drug Development, said: "We’re delighted to be sponsoring this Phase IIa trial of BT1718. Looking to the future, we believe BT1718 has the potential to create an entirely new treatment option for people with non-small cell lung cancer and other solid cancers whose therapeutic need is unaddressed by current medicines."

Syndax Announces Participation at Three Upcoming Investor Conferences

On September 2, 2020 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that members of its management team will participate in three upcoming virtual investor conferences. The details for the three conferences are (Press release, Syndax, SEP 2, 2020, View Source [SID1234564267]):

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Citi 15th Annual BioPharma Virtual Conference on Wednesday, September 9, 2020. Panel at 11:40 a.m. ET.
Baird 2020 Virtual Global Healthcare Conference on Thursday, September 10, 2020. Fireside chat at 4:20 p.m. ET.
Morgan Stanley 18th Annual Global Virtual Healthcare Conference on Monday, September 14, 2020. Fireside chat at 2:15 p.m. ET.
A live webcast of the events can be accessed from the Investor section of the Company’s website at www.syndax.com, where a replay of the event will also be available for a limited time.