On March 24, 2021 SunRock Biopharma reported the consortium formed by SunRock Biopharma and the Vall d’Hebron Institute of Oncology (VHIO) has been awarded the 2021 Public-Private Collaboration Projects call from the State Research Agency (Press release, SunRock Biopharma, MAR 24, 2021, View Source [SID1234647346]). Both entities are jointly participating in the TRADBI project, which seeks new BISPECIFIC ANTIBODIES TARGETING HER3 FOR THE TREATMENT OF AGGRESSIVE TUMORS, funded with 1.7 million euros from the Recovery, Transformation, and Resilience Plan.

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Cancer is one of the leading causes of morbidity and mortality worldwide, with over 19 million new cases and 10 million deaths worldwide in 2020. Despite advances in survival due to the introduction of immunotherapy treatments, there are still limitations in patients who do not respond or develop resistance to these therapies, making it necessary to develop new approaches to overcome these deficiencies.

In this regard, the TRADBI project will use a completely novel perspective to accelerate the arrival of a new, more effective and safe biological drug for the treatment of solid tumors such as breast cancer, colorectal cancer, or head and neck cancer. It is a next-generation antibody-drug-conjugate (ADC) that will be developed from SunRock Biopharma’s SRB19 antibody, a molecule targeting HER3 and EGFR, which has already demonstrated its high efficacy in preclinical models. This combination increases the antitumor efficacy of the treatment and reduces both the adverse effects of chemotherapy and the appearance of associated resistances.

This consortium brings together SunRock’s capacity for the development of innovative therapeutic antibodies with the experience of VHIO’s Tumor Biomarkers Group in identifying predictive tumor biomarkers in patients. The consortium also counts on the clinical leadership of Dr. Javier Cortés (IBCC) as a reference in the treatment of breast cancer, who will complete the transition of this new compound (SRB19-ADC) from its earliest stages of development to the arrival at clinical phases. The results of the TRADBI project will be of great significance for the upcoming arrival of this new therapy focused on the treatment of tumors with unmet clinical needs.

On March 24, 2021 The Korea Research Institute of Bioscience and Biotechnology reported on the 24th that it had transferred technology to the biotech Ingenium Therapeutics for the differentiation and mass proliferation of highly active NK cells (natural killer cells) from hematopoietic stem cells and the treatment of leukemia and lung cancer using the same (Press release, Ingenium Therapeutics, MAR 24, 2021, View Source [SID1234643518]).

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Ingenium Therapeutics (CEO: Jin-ok Ko) is a biotech company established in 2020 to develop immune cell therapy technology.

Through this technology transfer (exclusive license) contract, Korea Life Insurance receives a total of KRW 9.5 billion in fixed technology fees from Ingeniom and an additional technology fee of KRW 145 billion based on future sales performance. After technology transfer, we plan to conduct joint research with the goal of obtaining commercial clinical trial approval within this year.

NK cells are immune cells that make up approximately 10% of human blood immune cells. It has cancer cell killing ability (cytotoxicity) that kills cancer cells at the forefront without any other stimulation. The Korea Life Research Institute explained that the NK cell-based anti-cancer immune cell therapy technology transferred this time is expected to cause fewer side effects compared to T cell-based immune cell therapy because it does not proliferate on its own after injection into the human body but attacks cancer cells and gradually disappears. .

Dr. Inpyo Choi’s team at the Life Research Center’s Immunotherapy Research Center developed a technology to separate and differentiate NK cells from hematopoietic stem cells and to mass proliferate highly active NK cells. The explanation is that the efficacy and safety of NK cells were confirmed in non-clinical tests.

Afterwards, in a clinical trial conducted jointly by Dr. Choi’s team and Professor Lee Kyu-hyung’s team at Asan Medical Center, NK cells were administered to 41 patients with incurable leukemia, and the results showed that cancer progression was suppressed and survival rate was increased, and several papers were published. According to the researcher’s clinical results published in Blood Marrow Transplant in 2014 as a representative paper, administering NK cells to patients with incurable leukemia slowed the progression of the disease and increased the survival rate compared to the non-administered group (doi: 10.1016/j.bbmt.2014.01.031).

Director Kim Jang-seong of the Korea Life Research Institute said, "The Korea Life Research Institute is currently actively conducting research on CAR-NK cell therapy and iNK (induced NK) cell therapy using dedifferentiation technology, creating continuous research results in the field of gene cell therapy." "Based on this, we expect to contribute to the development of safe and highly effective anti-cancer immunotherapy agents for the future aging era."

Meanwhile, the technology is being developed through the Immunotherapy (Customized i-Medicine, CiM) convergence research group project of the National Science and Technology Research Council (NST) under the Ministry of Science and ICT, the national research and development project of the Ministry of Science and ICT and the Ministry of Health and Welfare, and the unique project of the Life Research Institute. This is a research achievement achieved through long-term, stable support.

On March 24, 2021 Ascentage Pharma, a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has recommended the novel candidate drug olverembatinib (HQP1351) of Guangzhou Healthquest Pharma Co., Ltd, a wholly-owned subsidiary of Ascentage Pharma, for a Breakthrough Therapy Designation (BTD) for the treatment of patients with chronic-phase chronic myeloid leukemia (CP CML) resistant and/or intolerant to first- and second-generation tyrosine kinase inhibitors (TKIs) (Press release, Ascentage Pharma, MAR 24, 2021, View Source [SID1234633502]). This BTD recommendation marks another major development for olverembatinib following the Priority Review granted by the CDE in October 2020. The indication of BTD recommendation is an expansion from that of the Priority Review designation.

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According to the NMPA’s Provisions for Drug Registration (SAMR Order No. 27) and Working Procedures for Review of Breakthrough Therapeutics (No. 82 of 2020) implemented on July 1, 2020, the breakthrough therapy review policy is designed to promote the research and creation of drugs with apparent clinical advantages, which are intended for the prevention or treatment of serious life-threatening diseases or diseases which severely impact the qualify of life for which there is no existing treatment or where sufficient evidence indicates advantages of the novel drug over currently available treatment options. Drugs that have been granted the BTD are prioritized by the CDE in communications and exchange, and in receiving guidance to promote the drug development progress. Furthermore, BTD designated drugs will be eligible to the Priority Review status that will accelerate the review process at the stage of application for commercialization. In conclusion, this measure will effectively accelerate the development and review of drugs presenting significant clinical value or addressing urgent clinical needs.

CML is a hematologic malignancy of the white blood cells. Following the commercialization of BCR-ABL TKIs, the treatment of CML has been revamped. However, despite clinical benefits offered by the first-generation BCR-ABL inhibitor imatinib (GLEEVEC), and several second-generation TKIs, acquired resistance to TKIs remains a major challenge in the treatment of CML. BCR-ABL tyrosine kinase mutations represent a key mechanism of acquired drug resistance; T315I, which is the most common drug-resistant mutation, occurs in about 25% of patients with drug-resistant CML. Patients with T315I-mutant CML are resistant to both first- and second-generation BCR-ABL inhibitors, presenting an urgent unmet medical need for an effective new generation treatment.

Olverembatinib is a novel, orally active, potent third-generation BCR-ABL inhibitor designed to effectively target BCR-ABL mutants, including T315I, and the first China-developed third-generation BCR-ABL inhibitor targeting drug-resistant CML. In July 2019, olverembatinib was cleared by the US Food and Drug Administration (FDA) to enter a Phase Ib clinical study. In May 2020, olverembatinib was granted an Orphan Drug Designation and a Fast Track Designation by the FDA. In October 2020, olverembatinib was granted Priority Review by the CDE in China for the treatment of adult patients resistant to TKI and with T315I-mutant chronic phase CML or accelerated phase CML. In December 2020, clinical trial results of olverembatinib were selected for oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting for the third consecutive year. These data further demonstrated the favorable safety and promising efficacy profiles of olverembatinib.

"Acquired drug resistance to TKIs remains an urgent unmet medical need and a major challenge in the treatment of CML," commented Professor Xiaojun Huang, Director of the Institute of Hematology, Peking University, and the principal investigator of olverembatinib in China. "Currently, no third-generation TKI has been approved in China, and no TKIs approved overseas is expected to enter China anytime soon. Multiple studies of olverembatinib, a China-developed BCR-ABL TKI, have consistently demonstrated promising efficacy and favorable safety in patients with drug-resistant CML, signifying significant potential clinical benefit to the patient population. We hope this BDT will further accelerate the clinical development and the review process for olverembatinib, and ultimately allow patients to soon benefit from this novel therapeutic."

"Olverembatinib is our first candidate drug entering the NDA-stage, and it is expected to become the first approved third-generation BCR-ABL inhibitor in China," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "This BTD for olverembatinib for the treatment of patients with CP CML intolerant or resistant to first- and second-generation TKIs represents the strong recognition of olverembatinib’s therapeutic potential in addressing the unmet medical needs in the treatment of CML. Being granted this BTD will strengthen our communication with the CDE, and more importantly, pave the way for a Priority Review designation to the future NDA for this indication, thus accelerate the clinical development and the approval of olverembatinib in China, and help us to make olverembatinib available to patients as early as possible."

On March 24, 2021 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the fourth quarter and year ended December 31, 2020 (Press release, Sana Biotechnology, MAR 24, 2021, View Source [SID1234584003]).

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"Engineering cells, whether done in vivo or ex vivo, has the potential to transform outcomes for patients across many diseases. We are excited about our significant progress in 2020 in turning this vision into a reality – continuing to build our scientific team, expanding our technology with key acquisitions and licenses, and generating important data across multiple platforms and programs," said Steve Harr, Sana’s President and Chief Executive Officer. "With the completion of our initial public offering in February, we have additional capital to execute our long-term vision of engineering cells to treat serious diseases such as cancer, various genetic disorders, type 1 diabetes, heart disease, and central nervous system diseases. We look forward to providing updates at multiple scientific conferences throughout the year and driving our multiple programs toward the clinic."

Recent Corporate Highlights

Hired key talent to our senior leadership team, including Ed Rebar, Ph.D., Chief Technology Officer, Terry Fry, M.D., Head of T Cell Therapeutics, and Ke Liu, M.D., Ph.D., Head of Regulatory Affairs & Strategy.
Entered into an exclusive license agreement with Washington University for certain intellectual property rights related to methods of generating, compositions of, and use of cells of endodermal lineage and beta cells.
Acquired Oscine Corp., a privately-held early stage biotechnology company developing glial progenitor cells focused on brain disorders to complement our broader ex vivo cell engineering platform.
Expanded our Board of Directors with the addition of Joshua Bilenker, M.D., former CEO of Loxo Oncology, Alise Reicin, M.D., CEO of Tectonic Therapeutic, and Michelle Seitz, CFA, Chairman and CEO of Russell Investments.
Entered into a non-exclusive license and development agreement with FUJIFILM Cellular Dynamics, Inc. (FCDI) for access to FCDI induced pluripotent stem cells (iPSCs).
Further strengthened our balance sheet with net proceeds of $626.6 million from the sale of 27 million shares of common stock in our initial public offering, bringing pro forma cash to over $1 billion as of February 28, 2021.
Fourth Quarter and 2020 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of December 31, 2020 were $412.0 million compared to $139.0 million as of December 31, 2019, an increase of $273.0 million. During the year ended December 31, 2020, Sana sold 27.2 million shares of its Series B convertible preferred stock at $16.00 per share for gross proceeds of $435.5 million.
Research and Development Expenses: Research and development expenses for the quarter and year ended December 31, 2020, inclusive of non-cash expenses, were $104.1 million and $257.9 million, respectively, compared to $39.3 million and $119.4 million for the same periods in 2019. The increases of $64.8 million and $138.5 million were primarily due to personnel-related expenses related to increased headcount to expand Sana’s research and development capabilities, costs for laboratory supplies and preclinical studies, and facility costs. The increase was also due to non-cash expenses for the increase in the estimated fair value of the success payment liabilities of $31.0 million and $70.2 million for the quarter and year ended December 31, 2020, respectively, and the increase in the estimated fair value of the contingent consideration of $33.8 million and $34.9 million for the quarter and year ended December 31, 2020, respectively. The increases in 2020 were offset by higher costs incurred in 2019 for the acquisition of technology. Research and development expense included stock-based compensation of $2.3 million and $4.9 million for the quarter and year ended December 31, 2020, respectively, and $0.4 million and $1.2 million for the same periods in 2019.
General and Administrative Expenses: General and administrative expenses for the quarter and year ended December 31, 2020, inclusive of non-cash expenses, were $9.2 million and $28.3 million, respectively, compared with $5.8 million and $21.8 million for the same periods in 2019. The increases of $3.4 million and $6.5 million for the quarter and year ended December 31, 2020, respectively, were primarily due to increased personnel-related expenses attributable to an increase in headcount to build our infrastructure, facility costs, and consulting fees.
Net Loss: Net loss for the quarter and year ended December 31, 2020 was $113.2 million, or $7.40 per share, and $285.3 million, or $21.92 per share, respectively. This compares to $43.0 million, or $4.94 per share, and $130.8 million, or $26.68 per share for the same periods in 2019.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the quarter and year ended December 31, 2020 was $37.8 million and $125.0 million, respectively, compared to $27.9 million and $76.2 million for the same periods in 2019. Non-GAAP operating cash burn is the decrease in cash, cash equivalents, and marketable securities excluding cash inflows from financing activities, cash outflows from business development activities, and the purchase of property and equipment.
Non-GAAP Research and Development Expenses: Non-GAAP research and development expenses for the quarter and year ended December 31, 2020 were $36.5 million and $123.0 million, respectively, and $28.3 million and $72.1 million for the same periods in 2019. Non-GAAP research and development expense excludes one-time costs to acquire technology and non-cash expenses related to the change in the estimated fair value of its contingent consideration and success payments.
Non-GAAP Net Loss: Non-GAAP net loss for the quarter and year ended December 31, 2020 was $45.5 million, or $2.98 per share, and $150.4 million, or $11.56 per share, respectively. This compares to $32.1 million, or $3.68 per share, and $83.5 million, or $17.04 per share, respectively, for the same periods in 2019. Non-GAAP net loss excludes one-time costs to acquire technology and non-cash expenses related to the change in the estimated fair value of its contingent consideration and success payments.
A discussion of non-GAAP measures, including a reconciliation of GAAP and non-GAAP measures, is presented below under "Non-GAAP Financial Measures."

On May 24, 2021 Hutchison China MediTech Limited ("HUTCHMED") (Nasdaq/AIM: HCM) reported that it has reached an agreement with GL Mountrose Investment Two Limited, a company controlled and managed by GL Capital Group ("GL Capital"), to sell its entire indirect interest in Hutchison Whampoa Guangzhou Baiyunshan Chinese Medicine Company Limited ("HBYS"), a non-core and non-consolidated over-the-counter ("OTC") drug joint venture business (Press release, Hutchison China MediTech, MAR 24, 2021, View Source [SID1234583629]).

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"HUTCHMED’s focus is the discovery and development of novel therapies in oncology and immunology. Over the past 20 years, we have invested in establishing one of the leading innovation-driven, global biopharmaceutical companies based in China," said Simon To, Chairman of HUTCHMED. "The sale of our shares in HBYS, and exit from the OTC drug arena, will allow us to focus our organization and resources on our primary aim of accelerating investment in our Oncology/Immunology assets in China and beyond."

Jeffrey Li, Founder and Chief Executive Officer of GL Capital, said, "As a long-term shareholder and supporter of HUTCHMED, GL is pleased to acquire its share in one of the best-known OTC businesses in China. This transaction is in-line with GL’s strategy of building a leadership position in the OTC drug area to serve patients’ needs for self-medication and cost containment of their overall healthcare budget."

The aggregate amount to be received by HUTCHMED of approximately $169 million in cash represents about 22 times HBYS’ adjusted net profit attributable to HUTCHMED equity holders of $7.7 million in 20201. Of the proceeds, approximately $127 million is related to its shareholding in HBYS with the approximately $42 million balance related to distributions of the previously announced land compensation and prior year undistributed profits.

A deposit of $15.9 million is payable by GL Capital immediately following signing of the agreement which will be credited against the proceeds due on closing of the transaction. The transaction is subject to regulatory approval in China and is expected to close in mid-2021.