The Leukemia & Lymphoma Society Therapy Acceleration Program® (LLS TAP) Announces Five New Investments Supporting Next Generation Cell Therapies and a Novel Immune Checkpoint Inhibitor

On April 22, 2021 The Leukemia & Lymphoma Society Therapy Acceleration Program (LLS TAP) reported five new investments aimed at speeding the development of new and improved immunotherapies for the treatment of blood cancers (Press release, The Leukemia & Lymphoma Society, APR 22, 2021, View Source;lymphoma-society-therapy-acceleration-program-lls-tap-announces-five-new-investments-supporting-next-generation-cell-therapies-and-a-novel-immune-checkpoint-inhibitor-301274791.html [SID1234578384]. LLS has dedicated more than $100 million over the past several decades, through both grants and TAP investments, to advancing pioneering approaches that harness cellular immunotherapies to fight blood cancers .

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"We are very proud of our role in bringing groundbreaking immunotherapies to patients, including chimeric antigen receptor (CAR) T-cell therapy, which harnesses the power of T cells to kill tumor cells," said Louis DeGennaro, Ph.D., LLS President and Chief Executive Officer. "Our TAP venture philanthropy initiative is investing in the next generation of immunotherapies to find cancer treatments that are even more effective, safer, longer-lasting, easier to use, and active against more types of cancer."

LLS TAP is a strategic venture philanthropy funding initiative to accelerate high-risk, innovative blood cancer therapeutics and change the standard of care in leukemia, lymphoma, and multiple myeloma. LLS TAP makes individual investments of up to approximately $10 million and offers insights to funded companies from its extensive knowledge of blood cancer indications, the treatment landscape, and the expertise of LLS staff. LLS TAP due diligence and investment credibility can help companies raise needed funds to drive clinical-focused research forward. Today’s investments include three companies that have novel immunotherapies in early human clinical trials and two companies with promising programs in preclinical development for blood cancers:

Caribou Biosciences, Inc, co-founded by CRISPR pioneer and Nobel Prize winner Jennifer Doudna, Ph.D., uses next-generation CRISPR genome editing technology, referred to as chRDNA, to develop "off-the-shelf" CAR T-cell therapies for hard-to-treat blood cancers. Off-the-shelf CAR T-cell therapies use healthy donor T cells instead of reengineering a patient’s own cells. This approach could make treatment less costly and immediately available to patients with rapidly progressing disease.

The company’s lead therapeutic candidate, CB-010, which targets the CD19 protein, is in a phase 1 clinical trial (ANTLER) for treatment of patients with relapsed or refractory non-Hodgkin lymphoma. The company is working on additional off-the-shelf CAR T-cell therapies targeting BCMA and CD371 proteins, which play key roles in multiple myeloma and acute myeloid leukemia, respectively. Caribou is also working on adding a CAR to induced pluripotent stem cells to differentiate them into "natural killer" or NK cells to create a new type of immune cell therapy called CAR-NK, which holds promise for the treatment of solid tumors and metastases in addition to blood cancers.

NexImmune, a company that benefitted from an initial TAP investment nearly four years ago and now received a second investment as part of the company’s initial public offering, is taking a different approach. The company’s Artificial Immune Modulation, or AIM platform, does not rely on genetic manipulation of T cells. Instead, it uses nanoparticles—specific tiny molecules—to directly activate the body’s T cells to fight specific types of cancer. The company has two novel products in phase 1 clinical trials; NEXI-001 to treat advanced acute myeloid leukemia or myelodysplastic syndrome, and NEXI-002 for the treatment of advanced multiple myeloma.

Immune-Onc Therapeutics is developing a novel type of myeloid immune checkpoint inhibitor. Their lead candidate, called IO-202, a first-in-class antibody targeting the immune inhibitory receptor Leukocyte Immunoglobulin-Like Receptor subfamily B (LILRB) member 4 (LILRB4, also knowns ILT3), has entered a phase 1 clinical trial for the treatment of advanced acute myeloid leukemia and chronic myelomonocytic leukemia. The company also plans to evaluate this compound in solid tumors soon. The company’s work builds on early research by Chengcheng (Alec) Zhang, Ph.D. at The University of Texas Southwestern Medical Center that was also funded by LLS grants. Checkpoint inhibitors work by ‘releasing the brake’ from the body’s own immune cells so that they can effectively attack cancer cells. In addition to their lead drug candidate, Immune-Onc has active preclinical programs targeting other members of the LILRB family including IO-108, a novel antagonist antibody targeting LILRB2 (also known as ILT4) which is currently in the IND-enabling stage, IO-106, a first-in-class anti-LAIR1 antibody, and other undisclosed programs for solid tumors and blood cancers.

CARISMA Therapeutics is a spin out company from the University of Pennsylvania (Penn), founded by Saar Gill, M.D., Ph.D. and Michael Klichinsky, PharmD, Ph.D., SVP of Research. Early work at Penn was supported in part by LLS grants. CARISMA is developing CAR-engineered macrophages, white blood cells that exert broad effects on the immune system, to infiltrate solid tumors, eat away at cancer and activate the adaptive immune system. Based on preclinical studies, the company’s highly differentiated CAR-macrophage (CAR-M) platform may have the potential to overcome challenges encountered by other cell therapies such as trafficking limitations to the tumor site, immunosuppressive tumor microenvironments and the heterogeneous expression of tumor-associated antigens. CARISMA is actively enrolling patients for its phase 1 clinical trial for CT-0508, a HER2-targeted CAR-M. CARISMA is also working closely with LLS TAP to develop one or more CAR-M therapies for blood cancers.

Abintus Bio is developing cutting-edge in vivo CAR therapies that allow for powerful CAR T cells to be generated directly in a patient’s body, eliminating the need for time-consuming and costly collection, engineering and re-infusion of patient T cells. The innovative Abintus approach aims to reprogram T cells inside the body to attack and eliminate tumors. This technology is currently in preclinical testing and could, if successful, improve patient access with an off-the-shelf profile while advancing patient outcomes.
"Funding from leaders like LLS TAP helps fuel the progress of our clinical development program, bringing next-generation treatments that much closer to patients," said Rachel Haurwitz, Ph.D., President and Chief Executive Officer of Caribou Biosciences. "We’re particularly excited to be working with LLS TAP because it gives us access to their deep knowledge of blood cancer and their network of patients and drug development experts."

"The LLS track record in accelerating lifesaving blood cancer treatments is unparalleled," said Lee Greenberger, Ph.D., LLS Chief Scientific Officer. "The continuity of our funding from preclinical through clinical research demonstrates our commitment to supporting innovative therapies as they move from the laboratory into practice."

"Since 2017, three TAP-supported therapies have been approved in the U.S.," added Lore Gruenbaum, Ph.D., VP of TAP. "TAP partnership provides companies with deep blood cancer expertise and increases their credibility with the investment community. We look forward to our ongoing partnerships with these five companies as we work together to provide new hope for patients."

Chugai Announces 2021 1st Quarter Results

On April 22, 2021 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported its financial results for the first quarter of fiscal year 2021 (Press release, Chugai, APR 22, 2021, View Source [SID1234578335]).

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"In the first quarter of 2021, despite growth in royalties and other operating income, both revenues and profits declined due to a decline in domestic and overseas sales. Still, there is no change to the full-year forecast for growth in revenues and profits as sales of mainstay products Tecentriq and Kadcyla are progressing significantly higher than expected at the beginning of the fiscal year, and we are also anticipating increases in exports to Roche and in royalty and profit-sharing income from the overseas growth of in-house products such as Hemlibra. In terms of R&D, we have received regulatory approval for our new anti-cancer drug Polivy and the liquid biopsy test FoundationOne Liquid CDx Cancer Genomic Profile, and we have begun a domestic Phase I clinical trial for the antibody cocktail casirivimab and imdevimab for COVID-19. We will continue striving to deliver innovation to patients as quickly as possible," said Dr. Osamu Okuda, Chugai’s President and CEO.

[First quarter results for 2021]

Chugai reported declines in revenues and operating profit for the first quarter (Core-basis) by approximately 6% and 12% year-on-year, respectively.

Revenues decreased as a whole due to a decrease in domestic and overseas sales, despite an increase in royalties and other operating income. Domestic sales declined by approximately 7% overall. Despite the sales increase in Oncology field driven by the double-digit growths of Tecentriq and Kadcyla, sales in the Primary field decreased by 20%, significantly impacted by the NHI drug price revision in April 2020 as well as generic competition. Overseas sales decreased by less than 20%, due to a decrease in exports of Actemra and other products to Roche. On the other hand, royalties and other operating income increased by double digits mainly due to an increase in royalty and profit-sharing income of Hemlibra, despite a decrease in other operating income from one-time income.

Cost to sales ratio remained at the same level as the same period last year. Operating expenses increased by approximately 9% as research and development expenses increased approximately by 15% due to steady progress in development projects while marketing and distribution expenses and general and administration expenses were almost flat year-on-year. As a result, operating profit declined by double digits.

The Company also made good progress in research and development. Chugai obtained regulatory approval in March 2021 for Polivy in relapsed or refractory diffuse large B-cell lymphoma, and FoundationOne Liquid CDx Cancer Genomic Profile as the first blood-based comprehensive genomic profiling test for solid tumors in Japan. A Phase III clinical trial evaluating Tecentriq in early-stage lung cancer achieved its primary endpoint, making a steady progress toward the regulatory application for line extension in 2021.

Regarding the clinical development of Actemra for COVID-19 pneumonia, Chugai announced in March 2021 that the Phase III REMDACTA study in combination with remdesivir did not meet its primary endpoint. Chugai is continuing further evaluation of overall risk-benefit profile based on the results of J-COVACTA, REMDACTA, COVACTA, EMPACTA trials and other studies of Actemra for COVID-19 pneumonia. In addition, a Phase I clinical trial in Japan was initiated in March 2021 for the investigational antibody cocktail casirivimab and imdevimab for COVID-19 in-licensed from Roche.

Ligand to Report First Quarter Financial Results on May 3, 2021

On April 22, 2021 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported that it will report financial results for the three months ended March 31, 2021 after the close of the U.S. financial markets on Monday, May 3, 2021 and will hold a conference call that same day beginning at 4:30 p.m. Eastern time (Press release, Ligand, APR 22, 2021, View Source [SID1234578353]). Speakers on the call will include Ligand’s CEO John Higgins, President and COO Matt Foehr and Executive Vice President and CFO Matt Korenberg.

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Blueprint Medicines to Report First Quarter 2021 Financial Results on Thursday, April 29, 2021

On April 22, 2021 Blueprint Medicines Corporation (NASDAQ: BPMC), reported that it will host a live conference call and webcast at 8:30 a.m. ET on Thursday, April 29, 2021 to report its first quarter 2021 financial results and provide a corporate update (Press release, Blueprint Medicines, APR 22, 2021, View Source [SID1234578369]).

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To access the live conference call, please dial (855) 728-4793 (domestic) or (503) 343-6666 (international), and refer to conference ID 9292306. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

AVECTAS Leads €7.23 million Consortium Awarded Part Funding From Disruptive Technologies Innovation Fund

On April 22, 2021 Avectas reported that it is leading a consortium which will invest €7.23 million, including €4.4 million awarded under the Irish Government’s Disruptive Technology Innovation Fund (DTIF), to develop a high-throughput scale of its proprietary cell engineering platform Solupore (Press release, Avectas, APR 22, 2021, View Source [SID1234578385]). With consortium partners Bluebridge Technologies and NIBRT, the project expands Avectas’ development towards commercialising an advanced large-scale, digitalised cell engineering platform optimised to manufacture ‘off-the-shelf’ cell-based therapies for cancer treatment.

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The Disruptive Technologies Innovation Fund (DTIF) is a €500 million fund established under Project Ireland 2040 and run by the Department of Enterprise Trade and Employment with support from Enterprise Ireland.

Headquartered in Ireland, with locations in Toronto, Canada and Cambridge, USA, Avectas collaborates with leading cell therapy businesses and research institutes to address their cell engineering challenges using its patented Solupore technology. Cell therapy is a rapidly evolving therapeutic approach for cancer. Immune cells are taken from donors, engineered to make them more effective and then administered to patients. This award will allow Avectas to expand its Solupore platform to manufacture next-generation allogeneic products that can be produced at scale for large numbers of patients.

Bluebridge Technologies will develop a digitalisation dimension for the platform through the project. They will design, build and test software components to underpin the manufacturing technology, highlighting the convergence of cell therapy manufacturing and digitalisation. NIBRT will test and validate the platform in the context of an end-to-end manufacturing process and will facilitate technology adoption by situating the Solupore platform at their world-class facilities. All parties will work synergistically to disrupt the manufacture and delivery of cell-based therapeutics to patients.

Avectas has previously won significant award support from the European Union (under the Horizon 2020 programme). The company is engaged in work programmes with several therapeutic companies and leading academic laboratories, including The Simon Laboratory at UC Davis, California, the Centre for Commercialization of Regenerative Medicine (CCRM) in Toronto and the new NK Cell Centre of Excellence at Karolinska Institute, Sweden.

Speaking today, Dr. Michael Maguire, CEO of Avectas, remarked, "cell-based therapies offer the extraordinary potential for the treatment of cancers, and we believe that our Solupore Platform will disrupt the current cell therapy manufacturing process and help make Ireland a world centre for this developing area". Prof. Niall Barron commented, ‘NIBRT is delighted to be involved in this exciting DTIF-funded project which will accelerate the development of transformative technology for manufacturing revolutionary new therapies which go beyond being just treatments and in many cases actually cure patients. This project is a perfect example of the innovation and ambition that exists in Ireland to be at the forefront of this exciting new field".

Garret Coady, CEO of Bluebridge Technologies, added, "BlueBridge Technologies is delighted to be part of this project, which will showcase the power and promise of digital technologies that have the potential, not only to enable the delivery of novel therapies but to do so viably, at scale."

Dr. Maguire thanked the Department of Enterprise, Trade and Employment for their support: "This is expensive but hugely valuable work which could not exist without the support of the Department and the DTIF. We are grateful for that support."