On April 29, 2019 Beam Therapeutics, a biotechnology company developing precision genetic medicines through base editing, reported the presentation of preclinical data from the company’s base editing platform at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 22nd Annual Meeting being held April 29 – May 2, 2019 in Washington, D.C (Press release, Beam Therapeutics, APR 29, 2019, View Source [SID1234535443]).

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In the experiment, the base editor BE4 demonstrated high efficiency multiplex base editing of three cell surface targets in primary human T cells (TRAC, B2M, and PD-1), knocking out expression of each gene in 95%, 95% and 88% of cells, respectively, in a single electroporation. Editing each of these genes may be useful in the creation of CAR-T cell therapies with improved therapeutic properties. Each of the genes was silenced by a single targeted base change (C to T) without the creation of double strand breaks. As a result, the BE4-treated cells also did not have any measurable translocations (large-scale genomic rearrangements), whereas cells receiving the same three edits with a nuclease did show detectable genomic rearrangements.

"Beam is actively applying base editing across a wide range of serious genetic diseases using both ex vivo and in vivo delivery approaches, and we are pleased to begin sharing some of the research progress in our therapeutic programs," said John Evans, chief executive officer of Beam. "The data presented today underscore one of the exciting emerging applications of base editing technology, which is to enable multiplex editing of CAR-T cells without genomic rearrangements. For advanced cellular therapies requiring a large number of simultaneous edits, base editing represents an important new technology that could open up new options for patients with cancer and other immune-mediated diseases."

Presentation Details:
Title: (#140) Base editors generate allogeneic CAR-T cells with no detectable genomic rearrangements and reduced genotoxicity
Session: CAR T Cell Therapies for Cancer
Date/time: Monday April 29, 2019, 4:00-4:15 p.m.
Room: IBR West

On April 29, 2020 Oncoceutics, Inc. reported that the United States Patent and Trademark Office (USPTO) has issued patent #10,266,533 entitled "7-BENZYL-4-(2-METHYLBENZYL)-2,4,6,7,8,9-HEXAHYDROIMIDAZO [1,2-A]PYRIDO[3,4-E]PYRIMIDIN-5(1H)-ONE, ANALOGS THEREOF, AND SALTS THEREOF AND METHODS FOR THEIR USE IN THERAPY" with an expiration date of January 29, 2036 (Press release, Oncoceutics, APR 29, 2019, View Source [SID1234558357]). This patent covers the composition of matter for ONC213, its di-salt formulation, and its use in the treatment of cancer. In addition to the claims related to ONC213, the patent also contains claims for millions of structurally-related imipridones.

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ONC213 is the fourth molecule in the company’s pipeline of "imipridone" family of anti-cancer small molecules that target G protein-coupled receptors, following ONC201, ONC206 and ONC212. As with other members of the imipridone class, ONC213 has very attractive chemical and biological properties including oral bioavailability, chemical stability and a large therapeutic index.

"We are delighted by the decision of the US Patent Office to grant Composition of Matter to the novel molecule ONC213," said Martin Stogniew, Ph.D., Chief Development Officer of Oncoceutics. "This patent paves the way for future generations of imipridones to enter the clinic and eventually benefit the lives of patients."

ONC213 has demonstrated anti-cancer activity and safety in various preclinical oncology models across hematological malignancies and solid tumors tested in the lab of Principal investigator Dr. Yubin Ge, Associate Professor of Oncology at the Karmanos Cancer Institute and Wayne State University School of Medicine. Results presented at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated that ONC213 targets leukemic stem cells in patient-derived xenograft mouse models, is well tolerated and combines synergistically with Bcl-2 inhibitor Venetoclax. Dr. Ge recently received a grant from the Kids Without Cancer and the Children’s Hospital of Michigan Foundation to further determine the mechanism of action of ONC213 and enable biomarker selection for clinical studies.

"Our team at the Karmanos Cancer Institute is excited by the potential of ONC213 as a new type of cancer therapy," said Dr. Yubin Ge. "We look forward to continuing development of this novel agent as it makes its way towards the clinic."

On April 29, 2019 MorphoSys AG (FSE: MOR; Prime Standard Segment, MDAX & TecDAX; NASDAQ: MOR) and I-Mab Biopharma (I-Mab), a China-based clinical stage biopharmaceutical company exclusively focused on the development of innovative biologics in immuno-oncology and autoimmune diseases, reported that the first patient has been dosed in a phase 3 randomized and multi-center clinical study in Taiwan to evaluate MorphoSys’s investigational human CD38 antibody MOR202/TJ202 in combination with lenalidomide in patients with relapsed or refractory multiple myeloma (Press release, MorphoSys, APR 29, 2019, View Source [SID1234556333]). I-Mab has exclusive rights for development and commercialization of MOR202/TJ202 in China, Taiwan, Hong Kong and Macao.

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"The initiation of our first phase 3 trial represents another important milestone in advancing MOR202/TJ202 towards registration with the hope of providing more therapeutic options for multiple myeloma patients globally. With planned enrollment of 291 patients, this will be a broad trial of this second most common blood cancer worldwide," said Dr. Joan Shen, M.D., Head of R&D at I-Mab. "In parallel with our pivotal phase 2 trial of MOR202/TJ202 in combination with dexamethasone, the phase 3 study will further assess the efficacy of MOR202/TJ202 as a potential second line treatment in multiple myeloma."

Under I-Mab’s fast-to-market development strategy, the phase 3 study, if successful, could lead to a biologics license application (BLA) in Greater China. The randomized, open-label, parallel-controlled, multicenter study will be conducted in mainland China and Taiwan to evaluate the efficacy and safety of the combination of MOR202/TJ202 plus lenalidomide (LEN) and dexamethasone (DEX) versus the combination of LEN and DEX in patients with relapsed or refractory multiple myeloma who received at least one prior line of treatment. The primary endpoint is to evaluate the progression-free survival (PFS) comparing the efficacy of MOR202/TJ202 plus LEN/DEX versus LEN/DEX.

The dosing of the first patient triggers a milestone payment of USD 3 million to MorphoSys.

"We are delighted that our partner I-Mab has started a phase 3 trial of MOR202/TJ202 in combination with lenalidomide in Asia in addition to the ongoing phase 2 trial of MOR202 in combination with dexamethasone. We see a high medical need for the treatment of patients with multiple myeloma in the Chinese region and look forward to supporting I-Mab in developing this investigational compound for these patients," said Dr. Malte Peters, Chief Development Officer of MorphoSys AG.

With MorphoSys’s support through a licensing agreement in November 2017, I-Mab is currently leading the clinical development of MOR202/TJ202 in Greater China, including mainland China, Hong Kong, Macao and Taiwan. In addition to Taiwan, I-Mab has filed an investigational new drug (IND) application to China’s National Medical Products Administration in August 2018. Previously on March 20, 2019, MorphoSys and I-Mab announced the first patient dosing of MOR202/TJ202 in a phase 2 multi-center clinical study in Taiwan in patients with relapsed or refractory multiple myeloma.

About MOR202/TJ202
MOR202/TJ202 is an investigational human monoclonal antibody derived from MorphoSys’s HuCAL antibody technology. The antibody is directed against CD38 on the surface of multiple myeloma cells, which has been characterized as one of the most strongly and uniformly expressed antigens on the surface of malignant plasma cells. According to its suggested mode of action, the antibody recruits cells of the body’s immune system to kill the tumor through antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). The antibody does not involve complement dependent cytotoxicity, or CDC, an additional immune mechanism involved in tumor cell killing. Scientific research suggest that an anti-CD38 antibody may have therapeutic potential also in other cancers as well as autoimmune diseases. Based on an exclusive regional licensing agreement signed in late 2017, I-Mab owns the exclusive rights for development and commercialization of MOR202/TJ202 in China, Taiwan, Hong Kong and Macao.

On April 29, 2019 EirGenix, Inc. reported that it has entered into a license agreement with global generic and biosimilar drug manufacturer Sandoz AG, granting an exclusive license to Sandoz for right of commercialization of EirGenix ‘s breast cancer biosimilar drug, EG12014 (Trastuzumab Biosimilar to Roche / Genentech’s Herceptin) globally with the exception of Taiwan and mainland China (Press release, EirGenix, APR 29, 2019, View Source [SID1234535444]). According to the terms of the agreement, EirGenix will receive an upfront payment, milestone payments, and is entitled to receive profit share payments for sales in the territory. The signing of this agreement is one of the most significant achievements for EirGenix’s product development business since the company’s establishment, and represents an exciting moment for Taiwan’s biotech industry.

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Sandoz is a Novartis division, and a global leader in generic pharmaceuticals and biosimilars and a pioneer in the emerging field of prescription digital therapeutics. Sandoz has a long history and extensive experience in the development and commercialization of biosimilar and cancer drugs in markets such as Europe and the United States. The collaboration between EirGenix and Sandoz will leverage the combined strength of EirGenix ‘s R&D of biosimilar drugs, with Sandoz’s substantial experience in global drug sales and advantages in market access. This collaboration will be conducive to EirGenix ‘s market development and expansion in the pharmaceutical market and will be extremely positive for EirGenix ‘s financial and business development as the collaboration will enhance the visibility and competitiveness of its products in the global market, which would thereby improve the company’s overall operating scale and profitability. With the successful market entry of EG12014, HER2-positive breast cancer patients will benefit from more treatment options.

EG12014 (Trastuzumab biosimilar) has entered a global Phase 3 clinical trial (Study No.: EGC002) which has been to date approved to conduct such trial by ten regulatory authorities including the U.S. FDA, Taiwan TFDA, as well as the authorities in Russia, Belarus, Ukraine, South Africa, Georgia, South Korea, India and Chile. A total of 800 breast cancer patients will be enrolled for this Phase 3 clinical trial, and the primary endpoint analysis is expected to be completed in the second half of 2020 to support the product registration. According to Roche’s 2018 annual report, Herceptin’s global sales amounted to 6.982 billion Swiss Francs. Herceptin tops the list in drug spending by Taiwan’s National Health Insurance, with an annual expenditure of nearly NT$3 billion.

EirGenix, Inc. has utilized reverse engineering technology in developing its four biosimilar products. In addition to the two antibody biosimilars for the treatment of HER2-positive breast cancers, there are two other anti-angiogenesis biosimilar drugs in development. EirGenix recently opened its commercial mass production facility in Zhubei, Taiwan, and aims to complete multiple batches of commercial-scale productions in 2019. In terms of CDMO business, it already reached break-even point in the first-half of 2016 and the revenue grew more than 35 fold since 2013 and is expected to steadily drive its revenue growth in the coming year. It is without a doubt that through its diligent pursuit of rapid business innovation, EirGenix has become one of the fastest growing companies in Taiwan’s biotech/pharmaceutical industry.

On April 29, 2019 Immunomedics, Inc., (NASDAQ: IMMU) ("Immunomedics" or the "Company"), a leading biopharmaceutical company in the area of antibody-drug conjugates (ADC) and Everest Medicines II Limited ("Everest Medicines"), a C-Bridge Capital-backed biopharmaceutical company, reported an exclusive license agreement to develop, register, and commercialize sacituzumab govitecan in Greater China, South Korea and certain Southeast Asian countries (Territory) (Press release, Immunomedics, APR 29, 2019, View Source [SID1234535687]).

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"We are pleased to be partnering with Everest Medicines to commercialize sacituzumab govitecan in the Asian market outside Japan and further develop the product in solid tumor indications of high unmet need in the territory," said Usama Malik, Chief Financial Officer and Chief Business Officer of Immunomedics. "This agreement sets a new benchmark for a single-asset licensing deal for regional China and exemplifies our commitment to expand the geographic footprint and clinical use of sacituzumab govitecan for the benefit of cancer patients worldwide."

"Sacituzumab govitecan is an innovative and unique ADC with a highly favorable clinical benefit rate across solid tumors and provides us the opportunity to address hard to treat cancers in key underserved regions across Asia. We look forward to partnering with Immunomedics’ clinical and regulatory teams to accelerate the development of sacituzumab govitecan across multiple indications in Greater China where there is great need for innovative cancer therapeutics of major impact," said Eric Rowinsky, M.D., U.S. Chief Medical Officer for Oncology, and Yang Shi, M.D., China Chief Medical Officer for Oncology of Everest Medicines.

Immunomedics will receive an upfront payment of $65 million and an additional $60 million based on U.S. FDA approval of sacituzumab govitecan in metastatic triple-negative breast cancer. Everest will develop and commercialize the product in various global and local indications across the Territory. The Company is eligible to receive development and sales milestone payments of up to $710 million, as well as escalating tiered royalties that begin in the mid-teens based on net sales within the Territory.

Pursuant to the agreement, Everest Medicines will be responsible for all costs associated with the clinical development and commercialization of sacituzumab govitecan in the Territory, while a Joint Steering Committee will be established between the companies to oversee the overall strategy and priorities.

"This transformative deal reinforces Everest Medicines’ position as the preeminent biopharmaceutical company to develop and commercialize globally innovative products in Greater China and other emerging Asia Pacific markets," said Ian Woo, President and Chief Financial Officer of Everest Medicines.

"Everest Medicines’ highly experienced team has a proven track record of delivering industry leading execution and we look forward to leveraging our deep knowledge and cumulative insights to address diseases with unmet need that are highly prevalent in these underserved regions," said Sean Cao, Interim CEO of Everest Medicines.

"Today’s announcement culminates a thoughtful and deliberate process, that expedites regional expansion of our lead product candidate in the world’s fastest growing pharmaceutical market and significantly enhances shareholder value. This partnership allows the Company to continue on its path to becoming a global biopharmaceutical company, and importantly maintaining the strategic optionality in core established markets worldwide," said Behzad Aghazadeh, Chairman of the Board of Directors of Immunomedics.

About Sacituzumab Govitecan

Sacituzumab govitecan, Immunomedics’ most advanced product candidate, is a novel, first-in-class antibody-drug conjugate targeting the Trop-2 receptor expressed by many solid cancers and delivering the moderately-toxic drug, SN-38, directly to the tumor. Sacituzumab govitecan has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for the treatment of patients with metastatic triple-negative breast cancer who have received two prior therapies for metastatic disease.