On April 29, 2021, Acceleron Pharma reported that Bristol Myers Squibb ("BMS") has announced net sales of REBLOZYL (luspatercept-aamt) to be approximately $112 million for the quarter ended March 31, 2021. As previously disclosed, under the collaboration agreement between Acceleron Pharma Inc. (the "Company") and BMS for REBLOZYL, the Company is eligible to receive tiered royalty payments from BMS on net sales of REBLOZYL in the low-to-mid 20% range. The Company expects to report royalty revenue of approximately $22.4 million from net sales of REBLOZYL in the quarter ended March 31, 2021.

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This preliminary unaudited revenue estimate is the responsibility of management and is subject to the completion of the Company’s customary quarter-end financial closing procedures, including management’s review and finalization, as well as review procedures by the Company’s independent registered public accounting firm, which have not yet been completed. During the course of the Company’s review process, items may be identified that would require it to make adjustments, which could result in material changes to the Company’s preliminary unaudited estimated financial results. Consequently, this revenue estimate should not be viewed as a substitute for the Company’s earnings release and the Quarterly Report on Form 10-Q for the quarter ended March 31, 2021.

The information contained in this Item is being furnished and shall not be deemed "filed" for any purpose, and shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, regardless of any general incorporation language in any such filing.

On April 28, 2021 Zetagen Therapeutics, Inc. a private, clinical-stage, biopharmaceutical company dedicated to driving breakthrough innovation in the treatment of metastatic bone cancers and osteologic interventions reported that the European Patent Office issued Patent. No. 3148517 to the Company (Press release, Zetagen Therapeutics, APR 28, 2021, View Source [SID1234643692]). The patent, entitled Composition and Methods to Promote Bone Formation, covers the use of methods for stimulating bone growth using its small molecule, ZetaMet (Zeta-BC-003). ZetaMet (Zeta-BC-003) is a synthetic, small-molecule, inductive biologic, delivered via a drug-eluting implant on an osteopromotive carrier.

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The European patent follows the previously issued Australian Patent. No. 2015267006, U.S. Patent. No. 10208306 and South African PCT. 201700029 covering the use of a method for stimulating bone growth using a small molecule and U.S. Patent Nos. 102656437 covering the use of methods to promote controlled bone creation and destruction as a means to repair large bone segmental defects.

"I am pleased with both our Clinical and IP Global progress to bring our novel technologies to the worldwide market, so patients suffering from painful metastatic lesions have access to our products", said Joe C. Loy, CEO Zetagen Therapeutics, Inc.

The new patent is part of an expanding and comprehensive portfolio of patents, patent applications and other intellectual property covering the composition, synthesis, manufacturing, formulations and uses for the treatment of a variety of metastatic bone lesions. Zetagen exclusively licensed its platform technology from the State University of New York in 2016.

On April 28, 2021 Shanghai BDgene Technology Co., Ltd. ("BDgene"), a leading global gene therapy development company with original delivery technology, reported the company has successfully completed a round A financing of 60 million yuan (Press release, BDgene Therapeutics, APR 28, 2021, View Source [SID1234641044]). This financing was led by Tsinghua Holdings Capital , Ennovation Ventures, Longmen Capital, ETP Ventures, Innovation Angel Fund and other institutions followed in the investment. Probe Capital served as the exclusive financial advisor. The funds will be used for preclinical and clinical development of multiple products, laboratory and team building etc. BDgene had previously received a strategic investment from Serve Accurate Faithful Evaluation in June 2019 , and a pre-A round of investment from Keytone Ventures in March 2020.

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According to the data, BDgene was established in 2018 and is an innovative company focusing on the development of in vivo gene editing and in vitro transgene therapy therapies. Since its establishment, the company has attracted much attention from investment institutions and has received investment from many institutions. Why is it sought after by capital? What is the core technology? What are the competitive advantages of leading products?

Gene therapy has become the third revolutionary therapy, but delivery technology is still the most difficult point

For difficult to treat genetic, infectious, and degenerative diseases, gene therapy has the characteristic of "can be cured once". In recent years, it has become the third revolutionary therapy after chemical drugs and antibody drugs.

From the first appearance of the third-generation gene editing technology CRISPR in 2012, to the first in-vivo clinical trials of CRISPR in March 2020, to October 2020 after CRISPR won the Nobel Prize in Chemistry and other major events, the gene editing technology gradually matured , But delivery technology has always been the biggest obstacle in the field of gene editing therapy.

In January 2020, Nobel Prize winner Jennifer Doudna wrote an article in "Nature" magazine that "Delivery remains perhaps the biggest bottleneck to somatic-cell genome editing".

In the past, gene therapy delivery technologies that have been clinically verified by humans in the world mainly include three platforms: Adeno-associated virus, lentivirus vector, and lipid nanoparticles. These platforms have certain drawbacks for gene editing delivery. The ideal gene editing delivery tool needs to have both instantaneous and high-efficiency characteristics to ensure the safety and effectiveness of the treatment.

Create China’s original delivery technology to help gene therapy achieve a leap from "1 to 100"

Dr. Cai Yujia, the founder of BDgene, studied under the professors of Karolinska Institutet in Sweden, the Secretary-General of the Nobel Committee, Professor Thomas Perlmann, and Professor Jacob G. Mikkelsen, Aarhus University, Denmark, and he received a doctorate degree in gene therapy. He has mastered the core technology of gene therapy vectors and has rich experience in the production design and transformation of gene therapy virus vectors. After returning to China, he served as a researcher at the Institute of Systems Biomedicine, Shanghai Jiaotong University. In recent years, BDgene has successfully developed VLP-mRNA, a gene therapy delivery platform that has been clinically validated by humans, to deliver CRISPR/Cas9 mRNA to achieve safe and controllable gene editing therapy in vivo.

BDgene successfully completed the world’s second clinical study of in vivo CRISPR gene editing therapy in early November 2020 (in March 2020, Editas’ Zhang Feng team completed the world’s first case), which is also the world’s first CRISPR antiviral therapy Clinical research. This delivery technology delivers Cas9 mRNA through the lentiviral shell, which combines the high efficiency of viral vectors in infecting cells (in vitro infection rate of 99.8%, in vivo infection rate> 50%) and transient mRNA expression (degraded within 72 hours). Therapeutic effects have been achieved on animal models of different diseases, with technical advantages such as transient expression in vivo, no long-term safety risks associated with AAV expression of CRISPR, no obvious off-target, and low immunogenicity. Currently, with the permission of the Ethics Committee of the Eye, Ear, Nose and Throat Hospital Affiliated to Fudan University, the team of Professor Hong Jiaxu is presiding over the initial safe and controllable clinical application research based on this technology. In the future, this technology will be extended to the treatment of other viral infections, eye diseases and neurological diseases.

Mr. Zhang Yang, Chairman of Tsinghua Holdings Capital said, "The gene therapy industry has the characteristics of huge market space and extremely high technical threshold. In the past, gene editing technology has gradually broken through, but delivery technology is still the biggest bottleneck. In the past 10 years, Dr. Cai Yujia has been devoted to the development of gene therapy Delivery technology, It has successively successfully developed multiple original delivery technology platforms such as virion delivery CRISPR/Cas9 mRNA technology and next-generation lentivirus delivery technology. BDgene’s original viral keratitis treatment product has perfect preclinical animal experiment data. It is currently conducting non-registered human clinical trials in top domestic eye hospitals. BDgene also deployed gene editing technology to treat hereditary, infectious and degenerative diseases, viral vectors to treat blood diseases, and mRNA vaccines. "

Speaking of this Series A financing being exclusively led by Tsinghua Holdings Capital, Mr. Zhang Yang said, "Thank you very much for the trust of BDgene founder Professor Cai Yujia and old shareholders. In the future, he will support the development of the company in the long-term and jointly promote BDgene’s original delivery technology and The product helps gene therapy to achieve a leap from "1 to 100", so that more patients who are refractory to and incurable can really benefit in the future."

On April 28, 2021, Akeso, Inc. reported that, the Center for Drug Evaluation ("CDE") of the National Medical Products Administration (NMPA) of the People’s Republic of China (the "PRC") agreed to start the evaluation of the first-in-class novel drug Cadonilimab (PD-1/CTLA-4 bi-specific antibody, research and development code: AK104), which is an immuno-oncology therapy independently developed by the Company, plus platinum-based chemotherapy in combination with/without bevacizumab for a randomized, double-blind, placebo-controlled phase III clinical trial for first-line treatment of persistent, recurrent or metastatic cervical cancer (Press release, Akeso Biopharma, APR 28, 2021, View Source [SID1234633507]). This is the first phase III clinical trial of dual immunotherapy for first-line treatment of cervical cancer in the PRC.

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Recently, the Company successfully held a seminar of this global phase III clinical trial attended by experts and scholars of world-renowned gynecological oncology research institutions from China, the United States, Europe and Australia. The seminar reviewed the current status of cervical cancer treatment in the world, summarized the results of early stage clinical trials of Cadonilimab on several tumor indications, and discussed in detail the framework proposal of the global phase III clinical trial of Cadonilimab.

President & CEO of Akesobio, Michelle Xia, PhD, said, "Cadonilimab has demonstrated exceptional clinical efficacy in early stage clinical trials on cervical cancer. Based on this, the Company will further carry out the global phase III clinical trial for the first-line treatment of cervical cancer, which shows the excellent clinical operation efficiency of the company and will be beneficial to more patients suffering from cervical cancer around the world. The Company believes that Cadonilimab will become the first PD-1/CTLA-4 bi-specific antibody novel drug approved for market launch in the world."

Because of its satisfactory clinical data, Cadonilimab obtained fast track designation from the Food and Drug Administration of the United States (FDA) for treating patients with recurrent or metastatic cervical cancer after standard therapies in August 2020. Cadonilimab was also approved by the CDE for the inclusion in the "Breakthrough Therapy Designation" list for treating patients with recurrent or metastatic cervical cancer after standard therapies in October 2020. Patient enrollment of the registrational phase II clinical trial of Cadonilimab for the treatment of recurrent or metastatic cervical cancer was completed in January 2021 in the PRC.

INFORMATION ABOUT CADONILIMAB (PD-1/CTLA-4 BI-SPECIFIC ANTIBODY)

Cadonilimab (AK104) is a novel, potential next-generation, first-in-class bi-specific PD-1/ CTLA-4 immuno-oncology backbone drug independently developed by the Company, and its major indications include liver cancer, cervical cancer, lung cancer, gastric cancer, esophageal squamous cell cancer and nasopharyngeal carcinoma. The preliminary research data of cervical cancer, gastric cancer and other tumors shows that, as compared with the combination therapy of PD-1 and CTLA-4, Cadonilimab has much lower toxicity and demonstrated promising safety profile and efficacy.Our AK104 project has been incorporated in the Major New Drug Innovation Program under the 13th Five-year Plan for Major Technology Project (The 13th Five-Year Plan for Major Technology Project) issued by National Health Commission and Ministry of Science and Technology in 2017 and has been enlisted in the 2017 Pearl River Talent Program of Guangdong Province — Introduction of Innovation and Entrepreneurship Team Support Program (2017 Guangdong Province "Pearl River Talent Program" Introduction of Innovation and Entrepreneurship Team Support Project). It was also jointly rated by China Medical Biotechnology Association and Chinese Medicinal Biotechnology as one of the 2017 Top Ten Medicinal Biotechnology Advancements in China (2017 Top Ten Medicinal Biotechnology Advancements in China).It was also jointly rated by China Medical Biotechnology Association and Chinese Medicinal Biotechnology as one of the 2017 Top Ten Medicinal Biotechnology Advancements in China (2017 Top Ten Medicinal Biotechnology Advancements in China).It was also jointly rated by China Medical Biotechnology Association and Chinese Medicinal Biotechnology as one of the 2017 Top Ten Medicinal Biotechnology Advancements in China (2017 Top Ten Medicinal Biotechnology Advancements in China).

On April 28, 2021 Pfizer Inc. (NYSE: PFE) reported that it has acquired Amplyx Pharmaceuticals, Inc., a privately-held company dedicated to the development of therapies for debilitating and life-threatening diseases that affect people with compromised immune systems (Press release, Pfizer, APR 28, 2021, View Source [SID1234597887]). Amplyx’s lead compound, Fosmanogepix (APX001), is a novel investigational asset under development for the treatment of invasive fungal infections.

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More than 1.5 million cases of invasive fungal infections occur worldwide each year, with mortality rates as high as 30-80% across infection typesi. Fosmanogepix has a novel mechanism of action with the potential to target fungal strains resistant to standard of care therapy. As there are only three classes of antifungal medications currently available, antifungal resistance can severely limit treatment options; a potential new therapeutic class may therefore be of importance for both physicians and patientsii. There has been no novel therapeutic class of antifungal therapies approved by the U.S. Food and Drug Administration (FDA) in nearly 20 years.

"The COVID-19 pandemic has been a stark reminder of the devastating impact of infectious diseases, highlighting the continuous need for new anti-infective therapies to treat both emerging and difficult to treat bacterial, viral and fungal infections," said Angela Lukin, Global President, Pfizer Hospital. "We are deeply committed to helping patients suffering from infectious diseases, continuously seeking opportunities to build our portfolio of anti-infective therapies. We’ve already invested in assets that, if approved, could help address drug-resistant bacterial infections and critical viral infections; with this acquisition, we look forward to progressing the development of a novel anti-fungal as well."

Fosmanogepix is currently in Phase 2 clinical trials evaluating the safety and efficacy of both intravenous (IV) and oral formulations for the treatment of patients with life-threatening invasive fungal infections caused by molds, yeasts and rare molds (e.g., Aspergillus spp, Candida spp including Candida auris, Fusarium spp. and Scedosporium spp). Fosmanogepix has demonstrated broad-spectrum activity in-vitro and has shown wide distribution to various tissues including the brain, lung, kidney and eye. With both IV and oral formulations in development, Fosmanogepix may allow for the transition from IV to oral, thus potentially enabling, for the benefit of patients, the continuation of treatment outside the hospital.

In addition to Fosmanogepix, with this acquisition, Pfizer has secured ownership of Amplyx’s early-stage pipeline that includes potential antiviral (MAU868) and antifungal (APX2039) therapies.

Globally, infectious diseases are responsible for more than 8.4 million deaths annually*iii, accounting for two of the World Health Organization’s top ten causes of death worldwideiv. Infections are caused by different types of pathogens, including bacteria, viruses, fungi and parasites, and can be acquired in the community or in a hospital or healthcare setting.

The acquisition of Amplyx follows an initial equity investment by Pfizer in December 2019 as part of Amplyx’s Series C financing. At that time, Pfizer joined a world class group of biotechnology investors that included 3×5 Partners, Adage Capital Management, Arix Bioscience, BioMed Ventures, Lundbeckfonden Ventures, New Enterprise Associates, Pappas Capital, RiverVest Venture Partners and Sofinnova Investments.

Financial terms of this acquisition were not disclosed.

DLA Piper LLP (US) served as Pfizer Inc.’s legal advisor for the transaction, while Cooley LLP served as Amplyx’s legal advisor and Evercore as its financial advisor.