On August 12, 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), and Mission Bio, the pioneer in targeted single-cell DNA analysis and precision genomics, reported that they have formed a collaboration to utilize the Mission Bio TapestriⓇ Platform for targeted single-cell DNA analysis to study Onconova’s novel cancer therapy, rigosertib, through clinical trials (Press release, Onconova, AUG 12, 2019, View Source [SID1234538597]).

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With the growing complexity of clinical trials, precision biomarkers are needed to reduce the time and costs associated with the drug development cycle. Broad-based sequencing technologies lack the sensitivity to identify the earlier initial single cell events that contain the driver mutations that initiate the oncologic disease. With the Mission Bio Tapestri Platform, researchers can detect rare cancer subclones and co-occurring cancer mutations at the single-cell level, offering a precise way to measure therapy response and disease progression. Supporting the pharma and biopharma industries through clinical trials and commercialization continues to be a focus for Mission Bio.

Ras proteins control cell proliferation, and mutation of this protein can lead to cancer in affected individuals. Ras is mutated in over 30 percent of patients with cancer, making it one of the most sought-after targets. Onconova is developing rigosertib, a first-in-class, small molecule Ras mimetic, to target this mutation. Rigosertib blocks the activation of Ras effector proteins, thus modulating the Ras pathway. Onconova’s goal is to fully enroll INSPIRE, its phase 3 clinical trial studying rigosertib in higher-risk MDS patients who fail the current standard of care, by year-end.

"Through single-cell genomics, we can identify mutations with far better resolution than that of traditional sequencing methods. This allows a view into each patient’s disease at a level never before achieved," explained Darrin Crisitello, CCO of Mission Bio. "The Tapestri Platform can identify subclones that help monitor a patient’s response to research drugs in clinical trials, supporting the advancement of rigosertib to the clinic."

"Rigosertib has the potential to be the first new higher-risk MDS treatment in more than 15 years, for a condition affecting an estimated 59,000 patients with low and higher-risk MDS in the United States alone," said Dr. Steve Fruchtman, CEO of Onconova. "In adding the Tapestri Platform to our research and development program, we are including the opportunity to study single cell clones in MDS and determine the sequence of genetic events and the influence of rigosertib on these events along with clinical outcomes. These studies have the potential to make a meaningful difference in the lives of patients in need."

On August 12, 2019 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) partner CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and accelerating the launch of innovative therapeutics and pharmaceutical products in China, the U.S., and throughout the world, reported the official product launch of melphalan hydrochloride for injection (EVOMELA) in China which is the first commercial product launch for CASI. EVOMELA uses Ligand’s Captisol technology in its formulation (Press release, Ligand, AUG 12, 2019, View Source [SID1234538596]).

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Melphalan hydrochloride for injection (EVOMELA) received market approval by the China National Medical Products Administration (NMPA) for use as high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplant in patients with multiple myeloma, and as a palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate. It is the only approved melphalan product available in China.

About Multiple Myeloma

Multiple myeloma is a malignant hematological disorder that is characterized by abnormal proliferation of clonal plasma cells in the bone marrow and the secretion of monoclonal immunoglobulins that are detectable in the serum or urine. CASI Pharmaceuticals estimates that multiple myeloma accounts for 10-13% of hematological malignancies1,2 and in Western countries, the estimated incidence is 5.6 cases per 100,000 persons2. The estimated incidence of multiple myeloma in China is ~2.0 cases per 100,000 persons3, for an estimated annual incidence of approximately 27,8003. The estimated number of patients in China with multiple myeloma who are candidates for hematopoietic progenitor (stem) cell transplantation is estimated to be approximately 16,900/year. The current number of patients with multiple myeloma who undergo hematopoietic progenitor (stem) cell transplantation in China is estimated to be approximately 800/year. Autologous stem cell transplantation (ASCT) has been demonstrated to improve complete response rates and prolong median overall survival in patients with multiple myeloma1,3 and is considered standard of care for transplant-eligible patients. The preferred conditioning regimen for ASCT is melphalan1.

About Captisol

Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella, University Distinguished Professor at the University of Kansas’ Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled several FDA-approved products, including Amgen’s KYPROLIS, Baxter International’s NEXTERONE, Acrotech Biopharma L.L.C.’s EVOMELA (US marketer), Melinta Therapeutics’ BAXDELA and Sage Therapeutics’ ZULRESSO. There are many Captisol-enabled products currently in various stages of development.

On August 12, 2019 Heat Biologics, Inc. (NASDAQ:HTBX), a clinical-stage biopharmaceutical company specialized in the development of therapeutics designed to activate a patient’s immune system against cancer, reported that the U.S. Food & Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application to initiate a Phase 1 clinical trial of HS-130, in combination with HS-110, for patients with advanced solid tumors refractory to standard of care (Press release, Heat Biologics, AUG 12, 2019, View Source [SID1234538595]).

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HS-130 is Heat’s allogeneic ("off-the-shelf") cell line engineered to express the extracellular domain of OX40 ligand fusion protein (OX40L-Fc), a key costimulator of T cells, with the potential to augment antigen-specific CD8+ T cell response. HS-130 was manufactured by utilizing the Company’s proprietary process of reprogramming a live, genetically modified cell line. Improved efficacy and safety were demonstrated in multiple preclinical scenarios using OX40L-Fc via cell-based delivery compared to systemic delivery of an OX40 agonist antibody in combination with HS-110.

"HS-130 represents a major advance in the broad utility and versatility of our T-Cell Activation Platform (TCAP)," said Jeff Wolf, Founder & CEO of Heat. "We are leveraging the scientific, clinical and manufacturing expertise that we refined in the development of our HS-110 program in our effort to advance multiple cell-based cancer therapeutics for the activation of patients’ immune system. We look forward to providing further updates on both the upcoming trial and clinical enrollment, which we expect to commence in the fourth quarter of 2019."

"HS-130 is the first cell-based approach that utilizes OX40 co-stimulation," said Jeff Hutchins, Ph.D., Heat’s Chief Scientific and Operating Officer. "In our first-in-human dose escalation study, we are evaluating the potential synergy of combining HS-130 with HS-110, our NSCLC cell line that secretes up to 90 "neoantigens" combined with gp96, the body’s most powerful natural immune adjuvant. Once the optimal dose and ratio of OX40L-Fc and GP96-Fc are confirmed clinically, our future development plan includes engineering a single cell line that secretes both agents."

About T-Cell Activation Platform (TCAP)

Heat Biologics’ proprietary TCAP is an allogenic ("off-the-shelf"), cell-based system. This platform enables the combination of multiple T cell stimulators and/ or activators within a single cell system. Currently HS-110 and HS-130 are in clinical development with engineered gp96-Fc and OX40L-Fc, respectively.

About HS-110

HS-110 is the company’s lead product candidate utilizing TCAP. This product candidate is designed by engineering gp96-Fc to deliver 78 cancer antigens to stimulate the patients’ immune system and activate a robust cytotoxic T cell response. HS-110 is currently being evaluated in a Phase 2 clinical trial for advanced non-small cell lung cancer, in combination with Bristol-Myers Squibb’s nivolumab (Opdivo) or with Merck’s pembrolizumab (Keytruda).

About HS-130

HS-130 is a new cell line developed using TCAP that is expected to be entering clinical trials in Q4/19. HS-130 is designed to secrete OX40L-Fc, a potent inducer of antigen-specific CD8+ T cell proliferation. The first-in-human study aims to evaluate the safety and dose-response of HS-130 in combination with HS-110 in patients with advanced solid tumors.

On August 12, 2019 DiaMedica Therapeutics Inc. (Nasdaq: DMAC) reported that Rick Pauls, President and CEO, will be presenting at the 2019 Intellisight Conference, Wednesday, August 14th at 9:00 am Central Time at University of St. Thomas in Minneapolis, Minnesota (Press release, DiaMedica, AUG 12, 2019, View Source [SID1234538594]).

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About Intellisight

Intellisight is a 2-day conference sponsored by the University of St. Thomas – School of Law for institutional investors to meet with leadership teams from numerous companies, and for company leadership to meet with influential investors representing more than 200 buy-side firms.

On August 12, 2019 Boehringer Ingelheim and The University of Texas MD Anderson Cancer Center reported a new multi-year partnership to conduct collaborative research to rapidly advance therapies for various types of cancers, including gastrointestinal and lung cancers (Press release, Boehringer Ingelheim, AUG 12, 2019, View Source [SID1234538593]). The establishment of a joint Virtual Research and Development Center will enable effective data sharing and analysis between the organizations.

The partnership is built on a flexible framework, allowing for projects to enter at different stages (research, development and/or clinical stage) over several years. It further combines the unique patient-driven drug-development capabilities of MD Anderson’s Therapeutics Discovery division (link is external) with the innovative pipeline of novel medicines from Boehringer Ingelheim.

MD Anderson’s Therapeutics Discovery division is a multidisciplinary team of clinicians and researchers focused on advancing the next generation of cancer therapies. As part of the division, the TRACTION (link is external) (Translational Research to Advance Therapeutics and Innovation in Oncology) platform conducts cutting-edge translational research to better understand how new medicines work and which patients will see most benefit.

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Dr Victoria Zazulina

"We could not have chosen a better partner with all its research, translational and clinical expertise in lung and gastrointestinal cancers. Together, we hope to transform the treatment landscape for these diseases by tackling their root causes and drivers, that have so far remained elusive, exploring new and smart ways of killing cancer cells," said Dr Victoria Zazulina, Corporate Vice President and Global Head of Oncology, Medicine, at Boehringer Ingelheim. "Our innovative oncology pipeline coupled with strong partnerships like this will contribute to unravelling the complexities of these diseases and bringing innovative solutions to people with various types of cancers."

The Virtual Research and Development Center will focus on the development of potential new treatments including:
KRAS inhibition concepts, as mutations in the KRAS gene are common in various cancers, specifically in certain types of lung and gastrointestinal cancers.
a TRAILR2 agonistic antibody, with the potential to selectively induce cancer cell death (apoptosis).
"Within MD Anderson, we are committed to a singular goal of ending cancer," said Tim Heffernan, Ph.D., executive director of TRACTION at MD Anderson. "We look forward to working with Boehringer Ingelheim to advance their innovative pipeline of cancer medicines. Our Therapeutics Discovery team is well-poised to conduct impactful translational research, and this partnership will allow us to more rapidly advance much-needed new therapies to patients."

More than 4.1 million people die from gastrointestinal and lung cancers every year worldwide1, indicating an urgent need for new treatment approaches. Gastrointestinal cancers represent a heterogeneous complex array of diseases, and include oesophageal (throat), gastric (stomach), liver, pancreatic, and colorectal cancers. In 2018, lung cancer caused more than 1.7 million deaths1. There are two main types of lung cancer: small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC).

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This press release is issued from our Corporate Headquarters in Ingelheim, Germany and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved products may vary from country to country, and a country-specific press release on this topic may have been issued in the countries where we do business.