On September 23, 2019 I-Mab Biopharma ("I-Mab"), a China and U.S.-based clinical stage biopharmaceutical company exclusively focusing on the discovery and development of potential first-in-class and best-in-class biologics in immuno-oncology and autoimmune diseases, reported that it has entered into a clinical research collaboration agreement with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ., USA, to evaluate the combination of I-Mab’s TJC4 and MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in patients with multiple cancer types (Press release, I-Mab Biopharma, SEP 23, 2019, View Source [SID1234539726]).

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Under this collaboration agreement, MSD will supply KEYTRUDA (pembrolizumab) to I-Mab for use in clinical studies in combination with TJC4, a fully human anti-CD47 monoclonal antibody. Both parties will collaborate on a Phase 1 clinical trial, based on an agreed and finalized protocol, to evaluate the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of TJC4 and KEYTRUDA in patients with multiple cancer types.

"TJC4 is a highly differentiated anti-CD47 antibody currently under clinical development. This collaboration will provide an excellent opportunity to further explore therapeutic potential of combining TJC4 with KEYTRUDA across different tumor types." said Dr. Jingwu Zang, Founder and Chairman of I-Mab.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

About TJC4

CD47 represents one of the most promising immuno-oncology targets. The current investigational drugs targeting the CD47-SIRPa pathway have undesirable properties of binding to human red blood cells (RBC) or platelets. Such unwanted binding properties have been shown in pre-clinical and clinical settings to cause hematologic side-effects, e.g., anemia and thrombocytopenia, and the so-called "antigen sink effect" that affects pharmacokinetics of the investigational drugs. TJC4 is a highly differentiated monoclonal antibody made, by design, to recognize a unique epitope on CD47 and avoid strong binding to red blood cells. As such, TJC4 did not cause anemia in non-human primate studies，while it was shown to maintain anti-tumor activities. TJC4 is currently under clinical development in the U.S. and has been granted an IND approval by the National Medical Products Administration (NMPA) to start clinical trials in China.

On September 23, 2019 Celsius Therapeutics, a company focused on bringing personalized medicine to patients with cancer, autoimmunity and other complex diseases, reported that it has been named by FierceBiotech as one of 2019’s Fierce 15 biotechnology companies, designating it as one of the most promising private biotechnology companies in the industry (Press release, Celsius Therapeutics, SEP 23, 2019, View Source [SID1234539743]).

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"It is an incredible honor and we are humbled to be named a ‘Fierce 15’ company, which is a testament to both our promising single cell genomics-driven drug discovery approach as well as the hard work of our entire team," said Tariq Kassum, M.D., chief executive officer of Celsius. "At Celsius, the combination of patient tissue samples, massive datasets, and sophisticated machine learning algorithms allows us, for the first time, to understand the specific cells driving disease. With this new level of clarity, our approach has fierce potential to deliver a powerful new class of precision medicines in autoimmune disease and cancer."

"This year has seen unrivalled scientific talent in the early-stage life sciences world and it has been a pleasure to for us at FierceBiotech to speak to all 15 winners and hear their passion, progress and panache," said Ben Adams, Senior Editor of FierceBiotech. "Each company brought something different, exciting and potentially life-changing for myriad patients around the world across a host of diseases and disorders, using cutting-edge science, top-notch teams and a drive to genuinely make the world a better place, despite the risks and challenges that, as ever in biotech, lay ahead."

The Fierce 15 celebrates the spirit of being "fierce" – championing innovation and creativity, even in the face of intense competition. Every year FierceBiotech evaluates hundreds of private companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of its technology, partnerships, venture backers and a competitive market position. This is FierceBiotech’s 17th annual Fierce 15 selection.

On September 23, 2019 Genprex recently reported that independent researchers found that company’s TUSC2 (tumor suppressor candidate 2) prevented tumor growth in triple-negative breast cancer (TNBC) (Press release, Genprex, SEP 23, 2019, View Source [SID1234551277]). These independent researchers have no affiliation with Genprex.

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Genprex takes a deeper dive into the data of the study to provide analysis and a better understanding of what the study found and what it means for TUSC2.

The study, "MicroRNA-138 is a Prognostic Biomarker for Triple-Negative Breast Cancer and Promotes Tumorigenesis via TUSC2 repression," takes a look at one particular subtype of breast cancer, triple-negative breast cancer (TNBC).

Triple Negative Breast Cancer

According to the National Breast Cancer Foundation, TNBC occurs in about 10-20 percent of diagnosed breast cancers, and is more likely to affect younger patients (under the age of 50 years old), African Americans, Hispanics and those with a BRCA1 gene mutation.1,5

A diagnosis for TNCB means that the patient does not have any of the three most common types of receptors that enable breast cancer growth, including estrogen, progesterone and the HER2 gene, thus the name for triple-negative breast cancer.1 TNBC progresses rapidly and is more likely to metastasize to the brain and viscera relative to HER2+ cancers.5

Treating TNBC with today’s emerging targeted therapies and immunotherapies are typically ineffective because these receptors are not present. This makes TNBC difficult to treat, and as an aggressive cancer, it is increasingly a feared diagnosis. With few treatment options, most TNBC patients must undergo a lumpectomy, a mastectomy, radiation, chemotherapy or a combination of these treatments.2TNBC patients are significantly less likely to survive than other breast cancer patients following the first metastatic event.5

The Role of MicroRNA-138

MicroRNA, or miRNA, help to downregulate gene expression in a variety of manners.3Thousands of miRNAs have been identified, and miRNAs possess the capacity to target between tens and hundreds of genes simultaneously. They perform a key role in tumorigenesis as important modulators in cellular pathways by regulating target gene expression through translation repression or mRNA degradation.4

Increased miR-138 expression is highly specific to TNBC, and the Nature report notes that miR-138 correlates with poor prognosis in patients, and it is functionally relevant to cancer progression.

The independent researchers explored the role of miR-138 in TNCB and found that it is a specific molecular signature of TNBC and a prognostic biomarker for breast cancer pathogenesis. Researchers also found miR-138 to be a pro-survival oncomiR for TNBC, meaning that overexpression of the gene leads to cancerous tumor growth.6Lastly, when miR-138 is depleted, it leads to apoptotic cell death in vitroand prevents tumorigenesis in vivo. In finding a way to deplete miR-138, this could lead to cancer cell death.

The Role of TUSC2

The independent researchers also found that TUSC2, which is the active agent in Genprex’s lead drug candidate, was a direct target downregulated by miR-138. TUSC2 expression is elevated and TUSC2 protein levels are enhanced following miR-138 knockdown.In TNBC mouse model studies using TUSC2, tumors were barely detectable under miR-138 knockdown conditions, and the implantation of TUSC2 overexpressing cells yielded tumors that are significantly smaller than those in the control group. TUSC2 expression was found to substantially mimic miR-138 knockdown and prevent tumor growth, revealing a potential new treatment for TNBC.

On September 23, 2019 Flagship Pioneering, a unique life science innovation enterprise, reported that it unveiled Omega Therapeutics – a genomics medicine company focused on selectively directing the human genome to treat and cure disease by precisely controlling genomic expression without changing nucleic acid sequences (Press release, Omega Therapeutics, SEP 23, 2019, View Source [SID1234561269]). Mahesh Karande has been appointed as the company’s President, CEO and member of the Board of Directors. Mr. Karande was the former President and CEO of Macrolide Pharmaceuticals and a long-time Novartis AG executive where he held positions of increasing responsibility running large businesses in the U.S. and internationally. Flagship Pioneering developed and launched Omega Therapeutics through its innovation foundry, Flagship Labs.

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Since its founding in 2017, under the leadership of David Berry, M.D., Ph.D., General Partner of Flagship Pioneering, Omega Therapeutics has created a proprietary platform that precisely tunes Insulated Genomic Domains (IGDs), the fundamental regulator, or GenomStats, of the human genome, which are 3D closed-loop structures of DNA that control genomic activity, to unlock the native power of the genome to cure diseases. This enables the unique, rapid development of specific therapeutics that treat disease and help patients, without altering nucleic acid sequences, with implications across a variety of indications. Engineered genomic modulators, called Omega Controllers, precisely tune genomic activity to desired therapeutic levels to address the biological root of disease. Our therapeutics are designed to be more specific and durable and, as such, potentially more effective therapeutic options for patients.

"By mapping the topology of the human genome and using computational biology, we have been able to identify the correct genomic target for a therapeutic influence. Omega’s proprietary platform technology allows us to develop therapeutics for disease control, and potentially impact the way we treat and manage a broad range of illnesses," said Dr. Berry. "In addition to our own capabilities for therapeutic development, our technology also presents the opportunity to partner and optimize other targeted in vivo and ex vivo therapeutics to enhance their ability to treat disease and further help a wide range of patient populations with significant unmet needs."

Many health conditions, particularly complex diseases, are the result of malfunctions in the regulation of gene expression. Omega leverages the founding Flagship team’s pioneering innovations that extended the breakthrough work on epigenetics by Richard Young, Ph.D., Professor of Biology, Massachusetts Institute of Technology, and Member of the Whitehead Institute, and Rudolf Jaenisch, M.D., Professor of Biology, Massachusetts Institute of Technology and Founding Member of the Whitehead Institute, who first described how genomic activity is controlled by 3D closed-loop structures of DNA, now called Insulated Genomic Domains (IGDs), that contain one or more genes and their regulatory elements.

These insulated, or closed-loop, structures ensure that interaction between the regulatory elements and gene(s) leading to gene expression is contained within the IGD. Diseases often manifest due to alterations in gene expression or changes in IGD structure. As such, the IGD is the fundamental regulator, or GenomStat, of the human genome. Omega is pioneering the discovery and development of precision Omega Controllers to control the GenomStat. By precisely tuning the GenomStat, Omega Controllers regulate single- or multi-gene expressions to desired levels to treat or cure disease. Scientific strategy and research and development will be led by Chief Science Officer, Thomas McCauley, Ph.D., former Shire executive and more recently CSO of Macrolide Pharmaceuticals and CSO of Translate Bio (formerly RaNA Therapeutics).

"Flagship is focused on discovering value and innovating well beyond the boundaries of current research in order to build first-of-their-kind companies. Omega’s proprietary product-platform is taking an entirely new approach to tackling important, unsolved diseases," said Noubar Afeyan, Ph.D., Chief Executive Officer of Flagship Pioneering and Chair of Omega’s Board of Directors. "The executive team, board of directors and scientific advisors have created an unparalleled company that fits the extraordinary scope of the therapeutic potential of Omega Controllers, a new class of medicines."

"The depth and breadth of our platform gives us the ability to create transformative genomic medicines that deliver specificity of targeting, tunability, and durability of single and multi-gene expression, all without altering nucleic acid sequences. Our platform enables us to develop proprietary Omega Controllers, a new class of therapeutics targeting the control of a wide range of diseases in unprecedented ways," said Mr. Karande. "With a team of world-class scientists, founders and advisors, we are already executing several ambitious early-stage discovery programs in the near-term, as we strengthen our platform to address the vast opportunity of treating diseases resulting from genomic malfunction or dysregulation."

Omega Therapeutics is targeting the biological root of disease and designing novel therapeutics and optimizing current therapies to potentially address a wide range of disease categories, including rare genetic diseases, immunology, inflammation, metabolic diseases and oncology.

The Omega Therapeutics Board of Directors:

– David Berry, M.D., Ph.D., General Partner, Flagship Pioneering, Co-founder, Omega Therapeutics

– Richard Young, Ph.D., Professor of Biology, Massachusetts Institute of Technology, Member, Whitehead Institute

– Paul-Peter Tak, M.D., Ph.D., Chief Executive Officer, Kintai Therapeutics, Venture Partner, Flagship Pioneering, former Senior Vice President and Global Head of R&D for Immuno-inflammation, Oncology and Infectious Disease, as well as Chief Immunology Officer, GlaxoSmithKline (GSK)

– Mary Szela, Chief Executive Officer, TriSalus Life Sciences, former Chief Executive Officer, Novelion Therapeutics, and Chief Executive Officer, Melinta Therapeutics

– Mahesh Karande, President and Chief Executive Officer, Omega Therapeutics

– Noubar Afeyan Ph.D., Co-founder and Chairman of the Board, Omega Therapeutics, CEO Flagship Pioneering

ABOUT MAHESH KARANDE, PRESIDENT AND CHIEF EXECUTIVE OFFICER

Mahesh Karande is deeply experienced in running biopharma businesses across discovery, preclinical development, clinical development, commercialization and product life-cycle management stages. He has strong leadership and operational experience combined with a global work history spanning the United States, Europe, Asia and Africa. Most recently, Mr. Karande, as President and CEO, took Macrolide Pharmaceuticals, from discovery into early development. Earlier, Mr. Karande spent several years at Novartis, leading various U.S. and international business units. Mr. Karande left Novartis to join Intarcia Therapeutics as VP and General Manager to lead the launch of their first product; a role he left to take the helm at Macrolide.

On September 23, 2019 Onconova Therapeutics, Inc. (Nasdaq: ONTX), a Phase 3 stage biopharmaceutical company discovering and developing novel products to treat cancer, with a focus on Myelodysplastic Syndromes (MDS), reported that it has entered into securities purchase agreements with certain institutional and accredited investors to purchase an aggregate of 2,136,193 shares of its common stock, at a purchase price of $1.60 per share in a registered direct offering for aggregate gross proceeds of approximately $3.4 million (Press release, Onconova, SEP 23, 2019, View Source [SID1234539694]). The offering is expected to close on or about September 25, 2019, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

Onconova also has agreed that for each share of common stock purchased in the offering by an investor, a corresponding number of warrants of Onconova that were issued to such investor in February 2018 and/or April 2018, as applicable, will be amended to have a reduced exercise price of $1.60 per share and the term of exercise will be extended to December 31, 2022.

The shares of common stock are being offered by the Company pursuant to a "shelf" registration statement on Form S-3 (File No. 333-221684) previously declared effective by the Securities and Exchange Commission (the "SEC") on December 28, 2017. The shares of common stock may be offered only by means of a prospectus supplement that forms a part of the effective registration statement. A prospectus supplement and the accompanying prospectus relating to the offering will be filed with the SEC. Electronic copies of the prospectus supplement and the accompanying prospectus may be obtained, when available, from H.C. Wainwright & Co., LLC, 430 Park Avenue, 3rd Floor, New York, NY 10022, by calling (646) 975-6996 or by emailing [email protected] or at the SEC’s website at View Source

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these 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.