On November 5, 2019 A2 Biotherapeutics (www.a2bio.com), a biotechnology company developing innovative cell therapies for cancer patients, reported its official launch (Press release, A2 Biotherapeutics, NOV 5, 2019, View Source [SID1234550323]). The company, founded in 2018 by veteran biotechnology leaders, has a unique and potentially broad solution for the central problem of cancer research – targeted killing of tumor cells vs. normal cells. A2 Biotherapeutics employs a powerful discovery and optimization technology to identify antibody and T-cell receptor fragments that bind to targets that are technically challenging but definitively distinguish tumor cells. The target binder platform is seamlessly combined with proprietary cell engineering, modular vector designs, and novel quantitative assays to extend the reach of cell therapies into solid tumors.

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Investors in the $57 million Series A financing include The Column Group, Vida Ventures, Samsara BioCapital and Nextech Invest.

Tumor cells exhibit only subtle differences from normal cells, making it challenging to create cancer therapies that recognize and kill only tumor cells. A2 Biotherapeutics is overcoming this challenge, having already demonstrated targeted killing of tumor cells in its research. The company aims to beat cancer via tumor-specific targeting using a novel approach that considers targets gained or lost in cancer cells. "This approach has the promise of being utilized for multiple tumor types, providing solutions for many cancer patients. A2 Biotherapeutics has potent, highly selective binders that we combine into molecular constructs to integrate multiple signals and potentially provide a large therapeutic window," said Alexander Kamb, Co-Founder and chief scientific officer. Kamb was formerly SVP of Research at Amgen.

To unequivocally distinguish tumor cells from healthy cells, A2 Biotherapeutics is pursuing two target classes. First, the company addresses peptide MHC targets, which are generated inside the cell and presented on the cell surface, providing the potential to access intracellular targets gained in tumors (e.g., neo-antigens). The second target class is tackled through modular cell engineering to deploy a more powerful version of the mechanism used by natural killer cells. This latter approach addresses targets that are irreversibly lost in tumor cells via loss of heterozygosity.

To advance its product candidates, A2 Biotherapeutics has a fully integrated discovery, development and manufacturing organization in Agoura Hills, California with a team that already includes more than 40 staff. Efforts are led by an experienced management team aggregating the experience of ex-Amgen and ex-Kite Pharma drug and cell therapy developers. Scott Foraker, an executive with 25 years of experience at Amgen, was recently hired as president and chief executive officer. "Our unique approach to targeted killing of tumor cells has the potential to create breakthrough cancer therapies that we look forward to bringing to patients, particularly those in need of new approaches," said Foraker.

The company also has the support of distinguished academic experts in cell therapy and T-cell biology, including:

Mark Davis, PhD, Director, Stanford Institute for Immunity, Transplantation and Infection and The Burt And Marion Avery Family Professor of Microbiology and Immunology at Stanford University

Robert Schreiber, PhD, AM Bursky & JM Bursky Distinguished Professor of Pathology & Immunology at the Washington University School of Medicine in St. Louis

David Maloney, MD, PhD, Medical Director of Cellular Immunotherapy at the Immunotherapy Integrated Research Center (IIRC) and Leonard and Norma Klorfine Endowed Chair for Clinical Research at Fred Hutch

George Tsokos, MD, Professor of Medicine at the Harvard Medical School and Chief of Rheumatology at the Beth Israel Deaconess Medical Center in Boston

A2 Biotherapeutics currently has four programs in development with the first clinical candidate expected next year and is open to collaborations to rapidly advance these programs. The company’s plans also include innovations in manufacturing; it will launch an autologous cell manufacturing facility in 2020 with technology scalable for commercial launch. "A2 Biotherapeutics aspires to increase speed to patients and reduce cost of goods by leveraging its cell therapy manufacturing expertise and advances in technology," said Madhu Balachandran, former EVP of Operations at Amgen and member of A2 Biotherapeutics Board of Directors.

On November 5, 2019 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported business highlights and financial results for the third quarter ended September 30, 2019 (Press release, Fate Therapeutics, NOV 5, 2019, View Source [SID1234550322]).

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"We achieved several significant clinical milestones over the past three months including treating the first patients with FT516, the first-ever engineered iPSC-derived cellular immunotherapy, and securing FDA clearance to initiate clinical investigation of FT596, the first-ever cellular immunotherapy engineered to express three active anti-tumor modalities. We also successfully opened our new cGMP facility specifically designed to enable consistent, large-scale, and cost-effective manufacture of allogeneic NK cell and CAR-T cell products using clonal master iPSC lines as a starting cell source," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We look forward to the ASH (Free ASH Whitepaper) annual meeting in December, where we have had six abstracts accepted and will be sharing our first-in-human insights into the clinical safety and tolerability of FT500, the first-ever iPSC-derived cell therapy to be administered off-the-shelf in multiple doses over multiple cycles. With the completion of our recent common stock offering in September, we are well-positioned to generate clinical data across our iPSC-derived, cell-based cancer immunotherapy pipeline in 2020."

Clinical Programs

First-ever Patient Treatment with Engineered iPSC-derived Cell Product. In October 2019, two patients at the M Health Fairview University of Minnesota Medical Center were treated with FT516, the Company’s off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity, non-cleavable CD16 Fc receptor. FT516 is the first-ever cell product in the world derived from a genetically engineered pluripotent stem cell to be administered to patients. The first patient received FT516 in combination with rituximab for the treatment of diffuse large B-cell lymphoma, and the second patient received FT516 as a monotherapy for the treatment of acute myeloid leukemia.

Initiated Enrollment at 300M Cell Dose Level in FT500 ICI Combination Arm. The Company is conducting an open-label, multi-dose Phase 1 clinical trial of FT500, an off-the-shelf NK cell cancer immunotherapy derived from a clonal master iPSC line, for the treatment of advanced solid tumors. The dose-escalating stage of the Phase 1 study is designed to assess the safety and tolerability of administering up to six doses of FT500 as a monotherapy and in combination with immune checkpoint inhibitor (ICI) in an outpatient setting. In the monotherapy arm of the study, three patients have been treated at 100 million cells per dose and five patients have been treated at 300 million cells per dose, with no reported dose-limiting toxicities (DLTs) or FT500-related serious adverse events. Additionally, in the combination arm of the study in patients that have failed prior ICI therapy, three patients have been treated at 100 million cells per dose in combination with ICI therapy, with no reported DLTs or FT500-related serious adverse events. The Company is currently enrolling patients at 300 million cells per dose in the combination arm of the study.

Received FDA Clearance of IND Application for FT596. In September, the U.S. Food and Drug Administration (FDA) allowed the Company’s Investigational New Drug (IND) application for FT596, the Company’s first off-the-shelf chimeric antigen receptor (CAR) NK cell cancer immunotherapy, which is uniquely designed to engage multiple tumor-associated antigens expressed on cancer cells for best-in-class activity. FT596 is derived from a clonal master iPSC line engineered with three anti-tumor functional modalities: a proprietary CAR optimized for NK cell biology that targets the B-cell antigen CD19; a novel high-affinity, non-cleavable CD16 Fc receptor that is designed to augment antibody dependent cellular cytotoxicity (ADCC); and an IL-15 receptor fusion (IL-15RF), a potent cytokine complex that promotes NK cell activation without the need for systemic cytokine support. Study initiation is underway at multiple clinical sites for the first-in-human Phase 1 clinical trial of FT596 as a monotherapy, in combination with rituximab for the treatment of advanced B-cell lymphoma, and in combination with obinutuzumab for the treatment of chronic lymphocytic leukemia.

Completed Enrollment in Phase 2 PROTECT Study of ProTmune. In October, the Company completed enrollment in the randomized, controlled and double-blinded Phase 2 PROTECT study of ProTmune, the Company’s first-in-class, allogeneic hematopoietic cell graft for the prevention of acute graft-versus-host disease (GvHD) in patients undergoing hematopoietic cell transplantation (HCT) for the treatment of hematologic malignancies. ProTmune has been granted Orphan Drug and Fast Track Designations by the FDA, and Orphan Medicinal Product Designation by the European Commission.

Corporate Highlights

Opened State-of-the-Art cGMP Manufacturing Facility Dedicated to iPSC-derived Cell Therapies. In September 2019, the Company opened its current Good Manufacturing Practice (cGMP) manufacturing facility for the clinical production of its off-the-shelf NK cell and CAR T-cell product candidates. The Company’s facility, located in San Diego, California, is custom designed to use clonal master iPSC lines as a renewable cell source for the manufacture of off-the-shelf allogeneic cell products. The new state-of-the-art facility has been commissioned and qualified, the Company has been issued a drug manufacturing license by the State of California, Department of Health Services, Food and Drug Branch, and the Company has commenced manufacture of certain of its product candidates.

Foundational U.S. Patent Issued Covering iPSC-derived CAR T Cells. In August 2019, the U.S. Patent and Trademark Office issued U.S. Patent No. 10,370,452 covering compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell including an iPSC. The claims of this newly-issued patent are not restricted by the signaling domain of the CAR construct nor by the antigen to which the CAR targets and binds. The patent is expected to expire in 2034, and is owned by Memorial Sloan Kettering Cancer Center (MSK) and is licensed exclusively to Fate Therapeutics for all human therapeutic uses. The Company is currently conducting IND-enabling activities for FT819, its first off-the-shelf, iPSC-derived CAR T-cell product candidate, under its collaboration with MSK.

Completed $173 Million Common Stock Offering. In September 2019, the Company closed an underwritten public offering of 9.9 million shares of its common stock at a public offering price of $17.50 per share.

Third Quarter 2019 Financial Results

Cash & Short-term Investment Position: Cash, cash equivalents and short-term investments as of September 30, 2019 were $302.8 million, compared to $201.0 million as of December 31, 2018. The increase was driven primarily by $162.4 million in net cash proceeds received by the Company from its September 2019 public offering of common stock. These proceeds were offset by the Company’s use of cash to fund operating activities.

Total Revenue: Revenue was $2.4 million for the third quarter of 2019, compared to $1.0 million for the same period in 2019. Revenue for the third quarter of 2019 was derived from the Company’s collaboration with Ono Pharmaceutical.

R&D Expenses: Research and development expenses were $23.2 million for the third quarter of 2019, compared to $13.6 million for the same period in 2018. The increase in R&D expenses was attributable primarily to an increase in employee compensation, including share-based compensation, and in expenses associated with the clinical development and manufacture of the Company’s product candidates and the conduct of research activities including under the collaboration with Ono Pharmaceutical.

G&A Expenses: General and administrative expenses were $6.3 million for the third quarter of 2019, compared to $4.1 million for the same period in 2018. The increase in G&A expenses was attributable primarily to an increase in employee compensation, including share-based compensation.

Shares Outstanding: Common shares outstanding were 75.4 million as of September 30, 2019 and 64.7 million as of December 31, 2018. Preferred shares outstanding as of September 30, 2019 and December 31, 2018 were 2.8 million, each of which is convertible into five shares of common stock.

Today’s Conference Call and Webcast

The Company will conduct a conference call today, Tuesday, November 5, 2019 at 5:00 p.m. ET to review financial and operating results for the quarter ended September 30, 2019. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 4748666. The live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.

On November 5, 2019 ExCellThera Inc., an advanced biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, reported the publication of full data from the first clinical trial using ECT-001 (single UM171-expanded cord blood) in patients with haematological malignancies (Press release, ExCellThera, NOV 5, 2019, View Source [SID1234550321]). The data were published in the peer-reviewed medical journal, The Lancet Haematology.

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The clinical trial findings indicate that ECT-001 cell therapy is feasible, safe (as suggested by the low transplant-related mortality, low incidence of severe acute graft-vs-host disease (GVHD), and absence of moderate to severe chronic GVHD) and allows for the use of small cords without compromising engraftment. In addition, ECT-001 has shown potential to overcome the disadvantages of unexpanded cord blood transplants while maintaining their benefits of low risk of chronic GVHD and relapse. The Lancet Haematology paper provides the first detailed analysis of the study results presented at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH 2018) in December 2018 and supports the recent advancement of the ECT-001 clinical program.

"We’re pleased that these important results from the first clinical trial using ECT-001 in haematological malignancies are now fully available to the broader bone marrow transplant community," said Dr. Guy Sauvageau, CEO and founder of ExCellThera, and co-senior author of the paper. "These results indicate that ECT-001 transplants combine the advantages of conventional grafts using bone marrow (low treatment-related mortality), peripheral blood (fast engraftment) and cord blood (greater accessibility, low relapse and chronic GvHD) in a single, low cost, easy to produce 7-day culture product, which could lead to a paradigm shift in bone marrow transplantation."

The FDA granted ECT-001 Orphan Drug Designation for the prevention of graft-versus-host disease in 2018 and Regenerative Medicine Advanced Therapy Designation in the treatment of hematologic malignancies in 2019. ECT-001 is currently being used for the treatment of blood disorders in other ongoing and approved clinical trials in the United States and Canada. ExCellThera also plans to initiate a European clinical trial as well as a pivotal, multi-centre clinical trial in the coming months.

On November 5, 2019 DiaMedica Therapeutics Inc. (Nasdaq: DMAC) reported that Rick Pauls, President & Chief Executive Officer, will meet with institutional investors at the 10th Annual Craig-Hallum Alpha Select Conference on Tuesday, November 12, 2019, at the Sheraton New York Times Square Hotel in New York City (Press release, DiaMedica, NOV 5, 2019, https://ir.diamedica.com/news/detail/1582/diamedica-therapeutics-to-participate-in-10th-annual-craig-hallum-alpha-select-conference [SID1234550320]).

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Interested institutional investors should contact their Craig-Hallum sales representative to schedule a meeting.

On November 5, 2019 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" or the "Company"), a biopharmaceutical company focused on the development of new cancer therapies, reported it will host a cocktail reception featuring a panel discussion of Delmar’s glioblastoma multiforme (GBM) trial status with highly recognized thought leaders in GBM during the 2019 Society for NeuroOncology Annual Meeting in Phoenix, Ariz (Press release, DelMar Pharmaceuticals, NOV 5, 2019, View Source [SID1234550319]).

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The event will be held on November 22, 2019 at 4:15-5:30 PM MT at the JW Marriott Desert Ridge, 5350 E. Marriott Drive in Phoenix Ariz. in the Grand Sonoran Room (H-I). Panel participants to include:

John de Groot, M.D., professor and chairman ad interim, Department of Neuro-Oncology at The University of Texas M.D. Anderson Cancer Center
David Reardon, M.D., clinical director of the Center for Neuro-Oncology at the Dana Farber Cancer Institute and professor of Medicine at Harvard Medical School
Timothy Cloughesy, M.D., professor of neurology at the David Geffen School of Medicine at the University of California, Los Angeles and member of the UCLA Brain Research Institute and Jonsson Comprehensive Cancer Center.
Nicholas Butowski, M.D., neuro-oncologist practicing at UCSF Medical Center in San Francisco, Calif. and director of translational research in neuro-oncology at the Brain Tumor Center
Zhong-ping Chen, M.D., founder chairman of the Department of Neurosurgery/Neuro-oncology at Sun Yat-sen University Cancer Center, China and lead investigator of the Company’s Phase 2 clinical trial of VAL-083 in first-line treatment of MGMT-unmethylated GBM
Naureen Quibria, Ph.D., Equity Research Associate, Maxim Group – Moderator
For information or to sign up to attend this event, please send an email request to [email protected].

A recording of the event’s proceedings will be available shortly following on the Company’s website, www.delmarpharma.com.