On February 6, 2020 Kleo Pharmaceuticals, Inc., an immuno-oncology company developing next-generation, fully synthetic bispecific compounds designed to emulate or enhance the activity of biologics, reported that it has received Investigational New Drug (IND) authorization to proceed from the U.S. Food and Drug Administration (FDA) to initiate a safety and tolerability clinical study combining KP1237, a CD38-targeting antibody recruiting molecule (ARM), with patients’ own Natural Killer (NK) cells to treat multiple myeloma (MM) in post-transplant patients (Press release, Kleo Pharmaceuticals, FEB 6, 2020, View Source [SID1234553969]).

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The single-arm study will be conducted in 25-30 patients with exploratory endpoints that assess the MRD (minimal residual disease) conversion rate at 90-100 days after transplantation. Recent clinical trials have identified MRD negativity post-transplant as a potential surrogate of long-term remission in MM. The trial is expected to begin enrollment in the first half of 2020, and topline data are expected in the second half of 2021.

"We are excited to have clearance to initiate a clinical trial in the US that addresses a significant unmet medical need in newly diagnosed, post-transplant multiple myeloma patients," said Kleo CEO Doug Manion, MD. "Approximately 30,000 individuals are diagnosed with multiple myeloma in the United States each year, with at least 1/3 of those patients undergoing autologous stem cell transplants."

In this trial, KP1237 is being investigated as a "cell homing" molecule to target the patient’s activated NK cells to the CD38-expressing tumor. Current anti-CD38 therapeutic antibodies kill NK cells and are not approved for use in this clinical settingi.

Nonclinical efficacy data presented at the 2019 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated that CD38-ARMs are able to kill multiple myeloma cells by antibody-dependent cellular cytotoxicity without depleting CD38-expressing immune cells. Nonclinical data also demonstrated that the CD38-ARM molecule did not induce complement-dependent cytotoxicity (CDC) suggesting it is not likely to cause CDC in humans. Kleo’s 2019 ASH (Free ASH Whitepaper) posters can be viewed here and here.

On February 6, 2020 The University of Texas MD Anderson Cancer Center reported that it has developed a slightly different approach using a different type of immune cell called Natural Killer (NK) cells (Press release, MD Anderson, FEB 6, 2020, View Source [SID1234554065]). These cells target the cell-surface antigen CD19 and in a Phase I/IIa clinical trial, showed eight of 11 had a therapeutic response, with seven showing complete responses.

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The research was published in the New England Journal of Medicine. The seven patients showed no evidence of disease at a median follow-up of 13.8 months. There were no major toxicities observed.

"We are encouraged by the results of the clinical trial, which will launch further clinical studies to investigate allogeneic cord blood-derived CAR NK cells as a potential treatment option for patients in need," said Katy Rezvani, professor of Stem Cell Transplant & Cellular Therapy at MD Anderson and corresponding author of the study.

The new approach was led by Rezvani with MD Anderson’s CAR NK platform, support of the adoptive cell therapy (ACT) platform, Chronic Lymphocytic Leukemia Moon Shot and B-Cell Lymphoma Moon Shot, which are all part of MD Anderson’s Moon Shots Program.

CAR NK cells are allogeneic, which means they can be drawn from healthy patients rather than the cancer patient. This would be an off-the-shelf approach that would allow CAR NK cells to be manufactured and stored for immediate use. The current CAR-T therapies are individualized and take several weeks from drawing the patient’s T-cells to when they are prepared to be reinfused.

The NK cells in their technique are cultured from donated umbilical cord blood, then genetically engineered to express the appropriate CAR, which recognizes the specific cancers. They say these particular cells are "armored" with IL-15, a type of immune signaling molecule that is engineered to improve growth and survival of the cells. In this case, they were designed to target B-cell cancers.

In the trial, 11 patients received a single dose of the CD19 CAR NK cells, one of three different dose levels. Five patients had CLL and six had NHL. All the patients in the trial and received at least three previous therapies, with the maximum being 11 previous therapies. The first nine patients treated were partially matched by human leukocyte antigen (HLA), but the final two patients were treated with no HLA match.

"Due to the nature of the therapy, we’ve actually been able to administer it in an outpatient said," Rezvani said. "We look forward to building upon these results in larger multi-center trials as we work with Takeda to make this therapy available more broadly."

The CAR NK platform was licensed to Takeda Pharmaceutical Company in 2019. Takeda has exclusive rights to develop and commercialize up to four of the programs, including the CDK19 CAR NK cell therapy (TAK-007) and B-cell maturation antigen (BCMA)-targeted CAR NK cells.

MD Anderson and Takeda are now collaborating on launching a pivotal clinical trial for TAK-007 in 2021.

On February 6, 2020 Athenex, Inc. (Nasdaq: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer, reported that it will release fourth quarter and full year 2019 financial results on Thursday, February 27, 2020, before the market opens, and host a conference call and live audio webcast 8:00am Eastern Time to discuss the financial results and provide a business update (Press release, Athenex, FEB 6, 2020, View Source [SID1234573883]).

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To participate in the call, dial 877-407-0784 (domestic) or 201-689-8560 (international) fifteen minutes before the conference call begins and reference the conference passcode 13698005. The live conference call and replay can also be accessed via audio webcast at the Investor Relations section of the Company’s website, located at View Source

On February 6, 2020 eFFECTOR Therapeutics, Inc., a leader in the development of selective translation regulators (STRs) for the treatment of cancer, reported that Steve Worland, Ph.D., president and chief executive officer of eFFECTOR, will present a company overview during the BIO CEO & Investor Conference on Tuesday, February 11 at 2:00 p.m. ET (Press release, eFFECTOR Therapeutics, FEB 6, 2020, View Source [SID1234553938]). The conference will take place February 10-11, in New York, N.Y.

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On February 6, 2020 CEL-SCI Corporation (NYSE American: CVM), a Phase 3 cancer immunotherapy company, reported that Geert Kersten, Chief Executive Officer, will be presenting at the 2020 BIO CEO & Investor Conference in New York, NY on Monday, February 10, 2019 at 3:45 p.m (Press release, Cel-Sci, FEB 6, 2020, View Source [SID1234553954]).

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A live audio webcast of the presentation will be available on CEL-SCI’s website at View Source A replay of the presentation will be available approximately 1 hour after the presentation and is accessible until May 12, 2020.

Now in its 22nd year, the BIO CEO & Investor Conference is one of the largest independent investor conferences focused on established and emerging publicly traded and select private biotech companies. Experience the best of biotech with two days of productive partnering meetings with institutional and early-stage investors, industry analysts, and senior biotechnology executives, in one location.