On April 30, 2021 Shaperon, a new drug development bio company, reported that it would sign a business agreement (MOU) with Dong-A ST for the joint development of new bio drugs (Press release, Shaperon, APR 30, 2021, View Source;idx=45&pNo=3&code=press_en [SID1234629321]).

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This joint development agreement was made with the goal of jointly developing treatments for cancer and inflammatory diseases using nanobody-based biopharmaceuticals. Nanobodies are antibodies that are 1/10 the size of existing antibodies, and are attracting attention as a key technology for next-generation immuno-oncology drugs because of their excellent stability and water solubility, high production yield, and high homology with human antibodies, making it easy to develop therapeutics and diagnostic platforms.

The two companies will develop new bio drugs by combining Chaperone’s nanobody technology and Dong-A ST’s new drug development platform. Chaperone builds a nanobody library based on its own technology, develops an antibody suitable for the selected target, verifies the effect at the cellular level, and then proceeds with the effect verification in an animal model in collaboration with Dong-A ST. When efficacy verification is completed in an animal model, Dong-A ST will be in charge of building a cell line for the nanobody antibody. In addition, the two companies plan to jointly discover anti-cancer targets throughout the preclinical process and evaluate the efficacy of candidate antibodies.

Lee Myung-se, CEO of Chaperone, said, "Through this MOU, we expect to see results such as the development of new biologics to treat cancer and chronic inflammation and the creation of corporate value through technology transfer." We will do our best to succeed in commercialization," he said.

Chaperon is the only domestic company that has nanobody development platform technology. In the trend of new drug development, where the structure of protein-based drugs such as bispecific antibodies or antibody drug conjugates is becoming more complex, the side effects and low production efficiency of existing antibodies can be overcome, and various routes of administration such as oral or inhalation are available. It is high tech. Based on the nanobody platform, Chaperon is developing various pipelines such as immune checkpoint inhibitory bispecific antibodies and coronavirus neutralizing antibodies.

On April 30, 2021 Microbiotica, a leading player in microbiome-based therapeutics and biomarkers, reported that it has been highly commended in the "Life Science Company of the Year" category at the 4th Cambridge Independent Science and Technology Awards (Press release, Microbiotica, APR 30, 2021, View Source [SID1234583882]).

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The Company was recognised for its transformational technology and significant progress over the past year. Recent achievements include a key new strategic collaboration with Cancer Research UK and Cambridge University Hospitals, progressing lead programmes in ulcerative colitis and immuno-oncology towards the clinic, and moving into purpose-configured facilities at Chesterford Research Park.

Mike Romanos, CEO of Microbiotica, said: "We are proud to be recognised for the milestones we have achieved this past year. Despite challenging conditions, we have progressed our ongoing programmes, expanded and strengthened our team, signed a key strategic collaboration, and moved to our new facility in Chesterford Research Park, so we can now house all our scientists in the same building for the first time. I would like to congratulate all the winners and highly commended entries and thank the entire Microbiotica team for their continued efforts."

The Cambridge Independent Science and Technology Awards recognise outstanding life science and biotechnology companies in Cambridge. The ceremony took place virtually in a bespoke interactive environment, and was attended by leaders of businesses, organisations and research institutes across the Cambridge region on April 15, 2021.

On April 30, 2021 The Janssen Pharmaceutical Companies of Johnson & Johnson reported that they have submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of cilta-cel, an investigational B cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of patients with relapsed and/or refractory multiple myeloma (Press release, Janssen Pharmaceuticals, APR 30, 2021, View Source [SID1234578899]).

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The application is supported by positive results from the ongoing Phase 1b/2 CARTITUDE-1 study, investigating the safety and efficacy of cilta-cel.1,2 The latest results were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2020 Annual Meeting. Clinical development is ongoing with patients enrolled globally in various studies, including sites in Europe, the United States of America, China and Japan.1,2

"Despite advances in the treatment of multiple myeloma, there remains a high unmet need, especially for patients whose disease continues to progress," said Peter Lebowitz, M.D., PhD., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. "Through our collaboration with Legend Biotech, we continue to expedite the development of cilta-cel with a focus and priority on the patients who may benefit from this novel immunotherapy in the future."

CAR‑T therapy is a highly personalised treatment platform where a patient’s own T-cells are re-programmed to recognise and attack cancer cells.3 In early 2021, the EMA granted accelerated assessment for cilta-cel.4 Accelerated assessment is granted when a medicinal product is expected to be of major public health interest and a therapeutic innovation, and can significantly reduce the review timelines to evaluate an MAA.5

"Janssen has been advancing the science of oncology for more than 30 years, and we see great opportunity in the area of cell therapy and through our innovative platforms," says Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, Johnson & Johnson. "We are continuing to harness our deep scientific expertise in multiple myeloma as we look to advance therapeutic options, deepen clinical responses, and drive towards improved patient outcomes."

"Today’s submission to the EMA epitomises how we strive to make a meaningful impact in the multiple myeloma landscape through advancing innovative treatments for patients," says Saskia De Haes, Vice President, EMEA Regulatory Affairs, Janssen R&D BE. "We look forward to working in partnership with health authorities, as part of the accelerated assessment process, to support these patients by ensuring timely access to the latest therapeutic options."

A Biologics License Application seeking approval of cilta-cel for the treatment of relapsed and/or refractory multiple myeloma is currently under review by the United States Food and Drug Administration.6

# ENDS #

About CARTITUDE-1

CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label, multicentre study evaluating the safety and efficacy of JNJ-68284528 (JNJ-4528) in adults with relapsed or refractory multiple myeloma who have received at least three prior lines of therapy or are double refractory to a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD); have received a PI, IMiD and an anti-CD38 antibody.2 The primary objective of the Phase 1b portion of the study is to characterise the safety and confirm the dose of JNJ-68284528 (JNJ-4528), which was informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2).2 The primary objective for the Phase 2 portion of the study is to evaluate the efficacy of JNJ-4528 (primary endpoint: overall response rate as defined by the International Myeloma Working Group response criteria).2

About Ciltacabtagene Autoleucel (cilta-cel)

Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy for the treatment of patients with multiple myeloma. Cilta-cel is a differentiated CAR-T therapy with two BCMA-targeting single domain antibodies.1 CAR-T cells are an innovative approach to targeting cancer cells by harnessing the power of a patient’s own immune system. 7 BCMA is a protein that is highly expressed on myeloma cells.8

In December 2017, Janssen Biotech, Inc. (Janssen) entered into an exclusive worldwide license and collaboration agreement with Legend Biotech to develop and commercialise cilta-cel.9 In May 2018, Janssen initiated a Phase 1b/2 CARTITUDE-1 trial (NCT03548207) to evaluate the efficacy and safety of cilta-cel in adults with relapsed and/or refractory multiple myeloma, informed by the LEGEND-2 study results.2,10

In 2019, cilta-cel was granted PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA).11 PRIME offers enhanced interaction and early dialogue with developers of promising medicines, to optimise drug development plans and speed up evaluation of cutting-edge, scientific advances that target a high unmet medical need.12 In 2020, the European Commission granted orphan designation for cilta-cel.13

About Multiple Myeloma

Multiple myeloma (MM) is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells.14 In Europe, 50,918 people were diagnosed with MM in 2020, and more than 32,400 patients died.15 Around 50 percent of newly diagnosed patients do not reach five-year survival,16 and approximately 10 percent of patients with multiple myeloma will die within one year of diagnosis.17

Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure.18 Refractory MM is when a patient’s disease progresses within 60 days of their last therapy.19 Relapsed cancer is when the disease has returned after a period of initial, partial or complete remission.20 While some patients with MM have no symptoms at all, others are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.21 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and require new therapies for continued disease control.22

On April 30, 2021, ImmunityBio, Inc. (the "Company") reported that it entered into an Open Market Sale Agreement (the "Sale Agreement") with Jefferies LLC (the "Sales Agent") under which it may offer and sell up to $500,000,000 of shares of its common stock, par value $0.0001 per share (the "Shares"), from time to time through the Sales Agent, acting as the Company’s sales agent (Filing, 8-K, NantKwest, APR 30, 2021, View Source [SID1234578961]). The sales and issuances of the Shares under the Sale Agreement will be made pursuant to the Company’s effective shelf registration statement on Form S-3 (File No.333-255699) (the "Registration Statement"), that was automatically effective upon filing with the U.S. Securities and Exchange Commission (the "SEC") on April 30, 2021. The offering is described in the Company’s Prospectus, as supplemented by a Prospectus Supplement dated April 30, 2021, as filed with the SEC on April 30, 2021 (together, the "Prospectus").

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Pursuant to the Sale Agreement, sales, if any, of the Shares, will be made under the Registration Statement and an applicable prospectus supplement, by any method permitted by law, including without limitation (i) by means of ordinary brokers’ transactions (whether or not solicited), (ii) to or through a market maker, (iii) directly on or through any national securities exchange or facility thereof, a trading facility of a national securities association, an alternative trading system, or any other market venue, (iv) in the over-the-counter market, (v) in privately negotiated transactions with the Company’s consent, (vi) block transactions or (vii) through a combination of any such methods. The Sales Agent is not required to sell any specific amount of securities, but will act as our sales agent using commercially reasonable efforts to sell the Shares from time to time (the "Offering"), consistent with its normal trading and sales practices, applicable state and federal laws, rules and regulations and the rules of The Nasdaq Global Select Market, based upon instructions from the Company (including any price, time or size limits or other customary parameters or conditions the Company may impose). The Company has agreed to pay the Sales Agent a commission of up to 3.0% of the aggregate gross proceeds from each sale of Shares pursuant to the Sale Agreement and to provide the Sales Agent with customary indemnification and contribution rights, including for liabilities under the Securities Act of 1933, as amended. The Sales Agent’s obligations to sell the Shares under the Sale Agreement are subject to satisfaction of certain conditions, including customary closing conditions.

The Company is not obligated to sell any Shares under the Sale Agreement and may at any time suspend solicitation and offers under the Sale Agreement. The Sale Agreement may be terminated by the Company at any time by giving written notice to the Sales Agent for any reason or by the Sales Agent at any time by giving written notice to the Company for any reason or immediately under certain circumstances, and shall automatically terminate upon the issuance and sale of all of the Shares.

The foregoing description of the Sale Agreement is not complete and is qualified in its entirety by reference to the full text of the Sale Agreement, a copy of which is filed herewith as Exhibit 10.1 to this Current Report on Form 8-K and is incorporated herein by reference.

This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy the securities discussed herein, nor shall there be any offer, solicitation, or sale of the securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

Wilson Sonsini Goodrich & Rosati, Professional Corporation, counsel to the Company, has issued a legal opinion relating to the validity of the Shares being offered pursuant to the Sale Agreement. A copy of such legal opinion, including the consent included therein, is filed as Exhibit 5.1 to this Current Report on Form 8-K and is incorporated herein by reference.

On April 30, 2021 Treadwell Therapeutics, a clinical-stage biotechnology company developing novel, cross-modality medicines for unmet needs in cancer, reported the initiation of patient dosing in TWT-202, its Phase 1b/2 study to evaluate its CFI-400945, an oral, first-in-class inhibitor of Polo-like kinase 4 (PLK4) in patients with Leukemia as a monotherapy or in combination with hypomethylating agents (Press release, Tio Bioventures, APR 30, 2021, View Source [SID1234578817]). Dosing of the first patient in the trial commenced April 16th at the University of Texas MD Anderson Cancer Center, Houston, TX.

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"Oral PLK4 inhibition opens a completely new approach to target therapeutic vulnerability in high risk MDS and AML and is very amenable to combination with other effective therapeutic agents," said Principal Investigator, Dr. Gautam Borthukar, MD, Professor, Department of Leukemia, Division of Cancer Medicine, MD Anderson Cancer Center.

"We are excited about the initiation of TWT-202 with our highly potent PLK4 inhibitor," said Dr. Michael Tusche, Treadwell co-CEO. "Previous clinical studies have shown that CFI-400945 can lead to single agent complete remissions in high-risk refractory AML patients. We look forward to continuing the development of this agent with the goal of delivering first in class medicines to patients in need."

The Phase 1b/2 clinical trial of CFI-400945, is an open-label, multi-center, dose optimization study designed to assess the safety, tolerability, pharmacokinetic and pharmacodynamic profiles of CFI-400945 as a single agent or in combination with azacytidine or decitabine in patients with acute myeloid leukemia, myelodysplastic syndrome or chronic myelomonocytic leukemia. The trial will enrol approximately 72 patients at up to 20 sites in North America and Asia. It will involve 3 arms including monotherapy and combination dose optimization and expansion, as well as a food effect study.

About CFI-400945

CFI-400945 is a highly potent and selective inhibitor of the serine/threonine kinase PLK4, a cell cycle kinase known to be the master upstream regulatory of centriole duplication and is critical for the maintenance of genomic integrity. PLK4 is overexpressed in a variety of solid tumors and elevated expression is associated with poor clinical outcomes. Depletion of PLK4 expression in cancer cells by RNA interference leads to mitotic defects and cell death. PLK4 was identified as a drug target based on functional screening to identify vulnerabilities of genomically unstable cancers. Anti-tumor activity of CFI-400945 has been shown in mice bearing human cancer xenografts, including robust tumor growth inhibition and durable tumor regression in primary tumor xenografts from breast cancer. CFI-400945 is a potent, selective, orally administered, first-in-class inhibitor of the serine/threonine kinase, polo-like kinase 4 (PLK4). CFI-400945 is currently in multiple investigator-initiated studies in solid and liquid malignancies,