On June 11, 2020 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for DKN-01 for the treatment of gastric and gastroesophageal junction cancer (Press release, Leap Therapeutics, JUN 11, 2020, View Source [SID1234561000]). DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein, a modulator of Wnt/Beta-catenin signaling.

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"Orphan Drug Designation for DKN-01 in gastric and gastroesophageal junction cancer is another significant milestone in our DKN-01 development program and underscores the need for new treatment options for these indications," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "We believe DKN-01 has the potential to be an important new therapy for this patient population that remains an area of high unmet medical need."

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation provides to Leap certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

DKN-01 is currently being evaluated in Phase 1/2 and Phase 2 clinical trials for gastroesophageal, gynecologic, hepatobiliary, and prostate cancers. Site initiation activities are now underway for the Company’s combination study of DKN-01 plus tislelizumab, BeiGene, Ltd.’s anti-PD-1 antibody, in patients with gastric or gastroesophageal junction cancer with dosing of the first patients expected in the third quarter of 2020.

About DKN-01

DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein, a modulator of Wnt/Beta-catenin signaling, a signaling pathway frequently implicated in tumorigenesis and suppressing the immune system. DKK1 has an important role in tumor cell signaling and in mediating an immuno-suppressive tumor microenvironment through enhancing the activity of myeloid-derived suppressor cells and downregulating NK ligands on tumor cells.

On June 11, 2020 Accellix, the Cell Therapy QC company, reported a Series D financing round of $9.5 million led by bioMérieux, a world leader in the field of in vitro diagnostics for over 55 years (Press release, Accellix, JUN 11, 2020, View Source [SID1234561016]). All existing major investors participated in this raise and have also agreed to convert their pre-existing convertible note in the amount of $8.5 million, bringing the company’s total funding in this round to $18 million. This latest round of funding will be used to further expand the market reach of the Accellix system for Cell Therapy QC, enabling its customers greater access to the expertise, scale, resources, and technology needed to effectively assess the quality of life saving immunotherapies.

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"Companies developing cellular therapies in the area of immuno-oncology and other therapeutic areas understand the need to replace costly and complicated flow cytometry based procedures with an easy to use sample-to-answer system. Being recognized by bioMérieux for our achievements to-date and having access to their vast array of resources and expertise, will help us serve our existing customers while attracting new ones," said Nir Nimrodi, Accellix Chairman and CEO.

"The Accellix platform eliminates extensive training and complex operational procedures by automating inherent manual complex flow cytometry processes. It provides accurate and immediate flow cytometry results at the point of need, removing the requirement of sending samples out to a central lab. We have been searching for such a platform that will complement our Industry-leading Microbial Quality Control offering and it is our intention to work with Accellix on enabling them to advance their market reach and service and support capabilities," stated Michael Reynier, bioMérieux Vice President, Healthcare Industries Business Area. "Testing a precious cell therapy product for sterility and for quality, swiftly, accurately and reproducibly, will improve patients’ access to the lifesaving cellular therapies that are currently being developed."

On June 11, 2020 ChemoCentryx, Inc. (Nasdaq:CCXI) reported that it has commenced an underwritten public offering of its common stock (Press release, ChemoCentryx, JUN 11, 2020, View Source [SID1234561001]). In connection with this offering, ChemoCentryx plans to grant the underwriters a 30-day option to purchase up to an additional 15 percent of the number of shares sold. SVB Leerink and Piper Sandler are acting as joint bookrunning managers for the offering. All shares of the common stock to be sold in the offering will be offered by ChemoCentryx. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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The offering will be made by ChemoCentryx pursuant to a registration statement on Form S-3 previously filed with the U.S. Securities and Exchange Commission (SEC), which became effective upon filing on June 10, 2020. A preliminary prospectus supplement related to the offering and the accompanying prospectus have been filed with the SEC and are available on the SEC’s website located at View Source These documents may also be obtained from: SVB Leerink LLC, Attn: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6218, or by email at [email protected]; or Piper Sandler & Co., Attn: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924, or by email at [email protected].

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 other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state or other jurisdiction.

On June 11, 2020 AVM Biotechnology reported that it has been awarded a National Cancer Institute Small Business Innovation Research (SBIR) Grant from the National Institutes of Health (NIH) to study the use of their lead molecule AVM0703 as a preconditioning agent to allow safe and efficient delivery of therapeutic immune cells for cancer treatment (Press release, AVM Biotechnology, JUN 11, 2020, View Source [SID1234561017]). This novel solution could offer clinical advantages to any cell-based immunotherapy, improving access to potentially life-saving therapies by all cancer patients, including those too frail to receive chemotherapy.

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Cellular immunotherapy has the potential to become a crucial solution for cancer. However, toxic chemotherapy is currently required as preconditioning treatment to impair graft rejection and maintain therapeutic cells in the bloodstream where they can target cancer cells. Chemotherapy is hardly tolerated by frail cancer patients, and it fuels the side effects of immunotherapy such as toxic cytokine releases (CRS) and neuroedemas. AVM0703, which could be easily administered to increase efficient delivery of adoptive cellular immunotherapy, induces safe lymphodepletion in only 24 hours, sparing platelets, stem cells and red blood cells in animal models. AVM0703 can safely deplete monocytes, known to be a key inducer of CRS. CRS toxicities occur as frequently as 90% with half of them determined as severe. Severe CRS complications can be life threatening if not treated in a timely manner. Unlike chemotherapy, AVM0703 could safely deplete monocytes reducing the risk of CRS and making cellular immunotherapy accessible to high-risk individuals.

This Phase I grant will be used to validate the efficacy and safety of preconditioning by AVM0703 in an established tumor mouse models of multiple myeloma (MM). The proposal was regarded as very significant in addressing a clinical need of better ways for improving efficacy and reducing toxicity of cellular therapies. Moreover, preconditioning using AVM0703 was seen as well supported by a good rationale and strong preliminary data. AVM0703 mode of action could offer an exemplary preconditioning regimen.

Named one of the top 10 best Biotech and Pharma companies to keep your eye on in 2019 by Mirror Review Online Magazine, AVM Biotechnology was founded in 2008 by Dr. Theresa Deisher, Ph.D. With over 30 years of successful pharmaceutical research experience and holding over 47 patents, Dr. Deisher leads a team of scientists dedicated to changing what a diagnosis of cancer, autoimmunity, or chronic infectious disease means to patients and their loved ones. AVM received FDA IND approval in April 2020 to test AVM0703 for treatment of relapsed/refractory lymphoid malignancies. AVM’s passion is to deliver drugs that work rapidly and that are safe, effective, and affordable, to treat serious worldwide illness like cancer, autoimmunity, and life-altering infectious disease. They develop products that improve outcomes without additional suffering because they believe that side effects from treatments of cancers or infections should never be worse than the diseases themselves.

AVM is the only company to receive homologous use designation for a patient’s own bone marrow used for an indication outside of blood disorders. Additionally, in 2019 AVM Biotechnology was awarded an SBIR grant for targeted lympho-ablation as an alternative to cure Type I Diabetes by the National Institute of Diabetes and Digestive and Kidney Disease (NIDDK).

This award was granted by the National Institutes of Health under Award Number R43CA246896. The content of this press release is solely the responsibility of the author and does not necessarily represent the official views of the NIH.

On June 11, 2020 Invitae reported that New recommendations from a large, multidisciplinary consensus conference published this week in the Journal of Clinical Oncology suggest expanding use of genetic testing to guide treatment for men with prostate cancer, including the use of panel testing and testing patients with early stage disease (Press release, Invitae, JUN 11, 2020, View Source [SID1234561384]). Taken together with research recently presented by Invitae (NYSE: NVTA), a leading genetics company, the publications underscore the utility of increased access to genetic testing for men with prostate cancer across all stages of disease.

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

Invitae was among the non-voting sponsors of the conference, which gathered more than 100 experts across a number of specialties with the goal of developing recommendations for how clinicians can use genetic testing to help patients benefit from precision medicine approaches to prostate cancer.

"Inherited prostate cancer is starting to get the attention it deserves, but we have a long way to go to catch up to the research and testing that has been done in other cancers, such as breast cancer," said Sarah Nielsen, M.S., L.C.G.C. a medical affairs liaison now at Invitae who previously participated in the conference. "This framework provides a very thoughtful approach to implementing genetic testing for prostate cancer treatment, screening and family testing. Importantly, the framework recognizes that changes in a number of different genes can increase prostate cancer risk and therefore encourages greater use of panel testing for men with metastatic disease. With new precision therapies linked to specific genetic changes, increased genetic testing can help identify patients who could benefit from these approaches."

Among the consensus conference recommendations:

Larger panels are useful for patients with metastatic disease

Large germline panels and somatic testing were recommended for patients with metastatic prostate cancer. Of the approximately 12-17% of men with metastatic prostate cancer who harbor germline variants, the majority are found in DNA damage repair (DDR) genes such as BRCA1, BRCA2, ATM, CHEK2, PALB2, and the DNA mismatch repair (MMR) genes. Large panels provided information across these and other genes of significance, information which is increasingly informing options for PARP inhibitors, immune checkpoint inhibitors, platinum chemotherapy, and clinical trials.

Genetic information can support early diagnosis and inform disease surveillance

Germline test results are increasingly important for early detection, as men with BRCA2 variants exhibit higher rates of prostate cancer, often with a younger age at diagnosis and more clinically significant disease. Among patients with early-stage disease, emerging data suggest that men with germline BRCA2 mutations and possibly ATM mutations have higher rates of upgrading of prostate biopsies while on active surveillance, suggesting the utility of genetic information in shaping surveillance strategies after diagnosis.

Importance of using genetic information requires novel strategies to increase access to counseling resources

The guidelines recommend broad access to genetic counseling support but shortages of genetic counselors and wait times for traditional genetic counseling workflows will require development of alternate models for timely and responsible delivery of genetic testing for men and their families. The consensus framework provides suggestions for clinicians on how to counsel and provide alternatives to traditional in-person appointments for patients across a number of issues related to testing, including using pretest education materials and the use of telehealth genetic counseling sessions.

"This framework provides an important step in helping clinicians incorporate genetic testing into care for a wide range of prostate cancer patients," said Robert Nussbaum, M.D., chief medical officer of Invitae. "Research has shown that narrow testing criteria will miss men with clinically relevant variants that could inform their care. Providing a framework for more clinicians to expand their use of genetic testing will increase the number of patients who benefit from precision medicine approaches."

Research underscores frequency of clinically important variants that may be missed by narrow testing criteria

In addition, a study presented recently at the American College of Medical Genetics and Genomics online annual meeting that further underscored the frequency of actionable variants expanded testing can help uncover.

The study of 2,252 men who participated in Invitae’s Detect Prostate Cancer program found an overall positive rate of 13% with no statistical differences in rates among stages of disease. Only half of patients with an actionable variant reported a family history suggestive of increased risk. Nearly three-quarters (71%) of positive patients were eligible for management guidelines and/or potentially eligible for approved precision therapies or clinical trials. These data suggest that broader testing criteria and greater access to testing leads to better informed care for patients and their families.

The consensus conference noted the need for additional research into the associations between genetics and prostate cancer in African-American men, who are 1.8 times more likely to be diagnosed with and 2.2 times more likely to die from prostate cancer. Importantly, this study included 16% participation among African-Americans, which is greater participation than previous similar studies, aligning to the consensus conference research priorities.

The full consensus statement can be found in the Journal of Clinical of Oncology.