On February 2, 2021 Immunomic Therapeutics, Inc. (ITI), a privately held, Maryland-based biotechnology company, reported that the company will present at the BIO CEO & Investor Digital Conference, February 16-18, 2021 (Press release, Immunomic Therapeutics, FEB 2, 2021, View Source [SID1234574510]). Chief Executive Officer at ITI, Dr. Bill Hearl, will present a talk titled, "ITI-1000-A Novel Immunotherapy for GBM." Dr. Hearl will discuss ITI’s investigational UNiversal Intracellular Targeted Expression (UNITE) platform and its application in immuno-oncology, specifically glioblastoma multiforme (GBM). ITI’s technology platform has the potential to utilize the body’s natural biochemistry to develop a broad immune response and is currently being employed in a Phase II clinical trial as a cancer immunotherapy.

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Presentation details are as follows:

Who: William Hearl, Ph.D., Founder and CEO of Immunomic Therapeutics

What: ITI-1000–A Novel Immunotherapy for GBM

Where: Immunomic Therapeutics, Inc. – BIO CEO & Investor Digital Conference | BIO

About UNITE

ITI’s investigational UNITE platform, or UNiversal Intracellular Targeted Expression, works by fusing target antigens with the Lysosomal Associated Membrane Protein, an endogenous protein in humans, for immune processing. In this way, ITI’s vaccines (DNA or RNA) have the potential to utilize the body’s natural biochemistry to develop a broad immune response including antibody production, cytokine release and critical immunological memory. This approach could put UNITE technology at the crossroads of immunotherapies in a number of illnesses, including cancer, allergy and infectious diseases. UNITE is currently being employed in Phase II clinical trials as a cancer immunotherapy. ITI is also collaborating with academic centers and biotechnology companies to study the use of UNITE in cancer types of high mortality, including cases where there are limited treatment options like glioblastoma and acute myeloid leukemia. ITI believes that these early clinical studies may provide a proof of concept for UNITE therapy in cancer, and if successful, set the stage for future studies, including combinations in these tumor types and others. Preclinical data is currently being developed to explore whether LAMP nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and be used to create immune responses to tumor types that otherwise do not provoke an immune response.

On February 2, 2021 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and Sarah Cannon Research Institute (Sarah Cannon reported that the first patient has been dosed in the first-in-human phase 1 study evaluating DS-6000, a CDH6 directed antibody drug conjugate (ADC), in patients with advanced renal cell carcinoma or ovarian cancer with disease progression following standard treatment (Press release, Daiichi Sankyo, FEB 2, 2021, https://www.businesswire.com/news/home/20210202005385/en/Daiichi-Sankyo-Initiates-Clinical-Development-of-Sixth-DXd-ADC-DS-6000-with-Sarah-Cannon-Research-Institute [SID1234574508]).

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Despite recent advances in targeted treatment, five-year survival rates for both renal cell carcinoma and ovarian cancer remain low and new therapeutic strategies are needed for tumors that continue to progress on currently available medicines.1,2 CDH6 is a cadherin family protein overexpressed in several cancers, particularly renal cell and ovarian.3 CDH6 overexpression is associated with tumor growth and proliferation and has been correlated with poor prognosis in renal cell carcinoma.4 No CDH6 directed cancer therapies are currently approved.

"With DS-6000, we have applied our innovative DXd ADC technology to a promising molecular target, CDH6, and it has potential to serve as a new treatment modality for patients with renal cell or ovarian cancer," said Arnaud Lesegretain, Vice President Oncology R&D and Head, Alpha Portfolio, Daiichi Sankyo. "We are pleased to continue our successful collaboration with Sarah Cannon, working together on advancing the development of another novel ADC with first-in-class potential."

DS-6000 is the sixth DXd ADC from the oncology pipeline of Daiichi Sankyo to enter clinical development and the third being developed in collaboration with Sarah Cannon Research Institute.

"We look forward to developing DS-6000 as a potential treatment option for people facing renal cell carcinoma or ovarian cancer," says Erika Hamilton, MD, Director, Breast Cancer and Gynecologic Cancer Research Program, Sarah Cannon Research Institute at Tennessee Oncology. "In partnership with Daiichi Sankyo, we will further evaluate whether DS-6000 may serve as a new and effective therapy for patients who have progressed on standard treatments."

About the Study
The two-part, multicenter, open-label, first-in-human phase 1 trial will evaluate the safety and efficacy of DS-6000 in adult patients with advanced renal cell carcinoma or ovarian cancer resistant or refractory to standard of care therapy.

The first part of the study (dose escalation) will assess the safety and tolerability of increasing doses of DS-6000 to determine the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE) in approximately 46 patients with advanced renal cell carcinoma or ovarian tumors. The second part of the study (dose expansion) will further evaluate the safety and efficacy of DS-6000 at the RDE in two cohorts including approximately 30 patients with advanced renal cell carcinoma in cohort 1 and 25 patients with advanced ovarian cancer in cohort 2.

The study will evaluate safety endpoints including dose-limiting toxicities and adverse events and efficacy endpoints including overall response rate, duration of response, disease control rate, clinical benefit rate, time to response and progression-free survival. Pharmacokinetic and exploratory biomarker endpoints will also be assessed.

A total of approximately 102 patients are expected to be enrolled in this study at multiple sites in the U.S. For more information, please visit Clinicaltrials.gov.

About Renal Cell Carcinoma and Ovarian Cancer
Renal cell carcinoma accounts for 90 percent of all kidney cancer.5 There were approximately 431,000 new cases of kidney cancer and over 179,000 deaths reported worldwide in 2020.6 Ovarian cancer is one of the three most common gynecological malignancies.7 There were approximately 313,000 new cases of ovarian cancer and over 207,000 deaths reported worldwide in 2020.8 The five-year survival rate is 13 percent and 30 percent, respectively, for patients diagnosed with metastatic renal cell carcinoma and ovarian cancer.9,10

About DS-6000
DS-6000 is a potential first-in-class CDH6 directed antibody drug conjugate (ADC). Designed using Daiichi Sankyo’s proprietary DXd ADC technology, DS-6000 is the sixth ADC in the Daiichi Sankyo oncology pipeline to enter clinical development.

DS-6000 is comprised of a humanized anti-CDH6 IgG1 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a tetrapeptide-based cleavable linker. Preclinical research shows that DS-6000 binds to CDH6 on the surface of cancer cells, and it is proposed that DS-6000 is then brought inside the cell where lysosomal enzymes break down the linker and release the DXd payload to destroy the cell. In preclinical studies, DS-6000 inhibited tumor growth and induced tumor regression in CDH6 expressing renal cell and ovarian tumors.

DS-6000 is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.

About Daiichi Sankyo Cancer Enterprise
The mission of Daiichi Sankyo Cancer Enterprise is to leverage our world-class, innovative science and push beyond traditional thinking to create meaningful treatments for patients with cancer. We are dedicated to transforming science into value for patients, and this sense of obligation informs everything we do. Anchored by our DXd antibody drug conjugate (ADC) technology, our powerful research engines include biologics, medicinal chemistry, modality and other research laboratories in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in Berkeley, CA. For more information, please visit: www.DSCancerEnterprise.com.

On February 2, 2021 Exicure, Inc. (Nasdaq: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) constructs, reported that David Giljohann, CEO, will present at the 2021 BIO CEO & Investor Digital Conference, February 16-18, 2021 (Press release, Exicure, FEB 2, 2021, View Source [SID1234574507]).

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The presentation will be available to registered conference attendees for on-demand viewing beginning February 15, 2021 at 9:00AM ET via the virtual conference link.

Registered conference attendees may schedule a 1×1 meeting with Exicure via the conference scheduling link.

Replays of the presentation will be available on Exicure’s website for 30 days following the presentation.

On February 2, 2021 Genomic Testing Cooperative, LCA (GTC) reported that they are establishing a program offering comprehensive molecular profiling (DNA+RNA) testing to patients with cancer who are affected by cancer disparity and unable to pay due to lack of insurance or lack of coverage of this type of testing (Press release, Genomic Testing Cooperative, FEB 2, 2021, View Source [SID1234574506]). Ethnic and racial minorities, impoverished people, sexual and gender minorities (LGBTQ) are typically affected more negatively with cancer. One of the reasons for this disparity is poor access to precision medicine and exclusion from clinical trials or studies evaluating the potential differences in the biology of their cancer.

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GTC molecular profiling will provide the treating physicians and patients with proper diagnosis and classification of the tumor, help in determining prognosis, selecting therapy and in developing a strategy for treatment that is specific for the patient. The molecular profiling report provides information regarding potential clinical trials that will help the patients evaluate their options to participate and be treated in these clinical trials. Participation in this program will increase access of underserved patients and reduce disparity within community-based cancer care. In addition, the data generated from this program will be de-identified and made available to appropriate academic and scientific groups for the purpose of developing more personalized cancer treatment for minority groups of patients.

GTC is committed to donating 5% of its annual testing volume to this program. GTC is also establishing a donation fund allowing others to support this program and to increase the number of patients benefiting from this program. Individual donors and organizations can contribute to this program with 100% of the raised funds being used to pay for the actual cost of testing.

Patients must be nominated for this program by their physicians. Patients with solid tumors or hematologic neoplasms are eligible for testing. Hematologists/Oncologists can download a simple nomination form from the GTC website, fill in the required information and fax or e-mail to GTC. Patients can mention this program to their hematologists/oncologists and request nomination for this program.

Dr. Maher Albitar, GTC Chief Executive Officer and Chief Medical Officer, stated "GTC is committed to making cancer molecular profiling available to all patients with cancer. We all know that patients seen in academic centers are different from real-world patients. Minority patients are not adequately represented in the process for developing innovative medicine nor in the implementation of state-of-the-art medicine. As a diagnostic company, we are doing our part by defining the precise molecular abnormalities that can be targeted but having access to the expensive targeted therapy is a different struggle. We are hoping that pharmaceutical companies will join our effort and do their part in providing the appropriate drugs to these patients and will develop a mechanism to recruit them in their clinical trials."

A recent study reported that one-third of disparities in survival between white and black patients with stage IV colorectal cancer is a product of treatment gaps (HemOnctoday, January 21/2021).

On February 2, 2021 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that a preclinical study in animals demonstrated a potentially significant therapeutic benefit of Annamycin against metastatic osteosarcoma (Press release, Moleculin, FEB 2, 2021, View Source [SID1234574505]). This appears to be the result of the high cytotoxic potential of Annamycin previously demonstrated in vitro against sarcoma cells in combination with its high uptake by the lungs where the tumors in this study are localized. Computerized tomography (CT) scans demonstrated that animals treated with Annamycin exhibited significant suppression of tumor growth and not a single death was observed in the treated animals, whereas significant tumor burden contributed to the rapid death of 90% of untreated animals. While the study continues, as of day 130, the survival rate for animals treated with Annamycin was 100%, compared with only 10% for untreated animals.

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Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

Osteosarcoma is among a class of tumors that initiate in the bone of patients, with bone-related sarcomas representing the second most common form of sarcoma after soft tissue sarcoma. While many bone sarcomas can be addressed through surgical removal, it is estimated that as many as 40% of bone sarcomas will eventually metastasize to the lungs, where treatment can become more problematic. Researchers have more recently referred to the lungs and certain other vital organs as "sanctuary sites" for cancer where tumors can develop out of reach from conventional chemotherapies.

Once metastasized to the lungs, if tumors cannot be surgically removed, the primary chemotherapy regimen is the anthracycline doxorubicin (also known as Adriamycin). While 10% to 30% of patients with sarcoma lung metastases may initially respond to doxorubicin, most will relapse leaving the majority of these patients without an alternative chemotherapy. Moleculin recently announced findings from its sponsored research showing that doxorubicin has a limited ability to accumulate in the lungs of animals, which may help explain its limited efficacy in this sanctuary site. Treatment options are further limited because of the inherent cardiotoxicity of currently approved anthracyclines, including doxorubicin, which limits the amount of anthracycline that can be given to patients.

Annamycin is a "next generation" anthracycline that has recently been shown in animal models to accumulate in the lungs at up to 34 times the level of doxorubicin, which may account for the 100% survival rate attained in this most recent osteosarcoma lung metastases study. Importantly, Annamycin has also demonstrated a lack of cardiotoxicity in recently conducted human clinical trials of Annamycin for the treatment of acute myeloid leukemia, so the use of Annamycin may not face the same dose limitations imposed on doxorubicin.

Moleculin recently announced that the FDA has allowed the Company’s request for investigational new drug (IND) status in order to study Annamycin for the treatment of soft tissue sarcoma metastasized to the lungs. In addition, the FDA granted Orphan Drug Designation for Annamycin for the treatment of soft tissue sarcomas.

"Our ongoing sponsored research continues to expand the potential uses for Annamycin," commented Walter Klemp, Chairman and CEO of Moleculin. "We expect that one, and potentially two, clinical trials in sarcoma lung metastases should be up and running this year."