On March 12, 2020 EXUMA Biotech Corp. (formerly F1 Oncology), a clinical-stage biotechnology company discovering and developing CAR-T therapies for solid and liquid tumors, reported a $19M Series B round of financing that includes new investments by MSD Partners and F1 BioVentures, as well as conversion of notes held by individual investors (Press release, EXUMA Biotechnology, MAR 12, 2020, View Source [SID1234555463]).

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EXUMA Biotech Corp. and its affiliates will be utilizing these proceeds to advance new logic gated CAR-T therapies for solid tumor malignancies as well as for further development and partnering of its rapid point-of-care (or "rPOC"), same-day CAR-T platform technology.

Additionally, the company has now aligned management with its international subsidiaries under one cohesive global organization (www.exumabio.com), to better reflect its new business model. "We are now moving forward together as one company. The timing was right for us to create a unified global platform to leverage our synergistic capabilities, which we believe will ultimately allow us to produce and deliver CAR-T products even more efficiently for the millions of people worldwide in need of cutting-edge treatments in their fight against cancer," said Gregory Frost, Ph.D., Chairman and CEO of EXUMA Biotech Corp.

The name change coincides with the addition of two new directors, Scott Segal and Frank McCormick, who join existing directors David Ramsay and Gregory Frost. "We are pleased to welcome these new Directors to the EXUMA board; their collective experience will be invaluable as we advance through several key inflection points in 2020," said Dr. Frost. "This will be a tremendous year for the company as we continue development of these exciting new platforms, especially rPOC, which can help make cellular therapies more readily accessible to patients around the world."

Scott Segal is a Managing Director at MSD Partners ("MSD"), where he invests across a range of sectors, in both debt and structured equity. He joined MSD in 2006 from the Boston Consulting Group, where he provided consulting services to Fortune 500 executives. Prior to the Boston Consulting Group, Mr. Segal worked at HarbourVest Partners and Salomon Brothers.

Frank McCormick, Ph.D., F.R.S., D.Sc. (Hon) currently serves as a Professor at the UCSF Helen Diller Family Comprehensive Cancer Center. Prior to this, Dr. McCormick pursued cancer-related work and held positions with several Bay area biotechnology firms, including Cetus and Chiron Corporations. In 1992, he founded Onyx Pharmaceutics, where as CSO he initiated drug discovery efforts that led to the approval of Sorafenib in 2005 for treatment of renal cell cancer, and for liver cancer in 2007. Currently, he leads the NCI’s Ras Initiative, where he oversees developmental therapies against Ras-driven cancers, which include many pancreatic, colorectal, and lung cancers. Dr. McCormick is the author of over 300 scientific publications.

On March 12, 2020 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the fourth quarter and full year 2019 and provided an operational update (Press release, Bellicum Pharmaceuticals, MAR 12, 2020, View Source [SID1234555491]).

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"Bellicum is at an exciting inflection point as we continue to validate the GoCAR platform and explore its utility more broadly," said Rick Fair, President and Chief Executive Officer of Bellicum. "We recently presented promising new translational data from the BPX-601 Phase 1 study, and we intend to present an update later this year on safety and preliminary activity in pancreatic cancer using repeated BPX-601 GoCAR-T activation with rimiducid. We have also expanded utilization of our GoCAR platform by initiating our first off-the-shelf GoCAR-NK program. In 2020, we look forward to the progress we expect to make applying our platform to solid tumor and off-the-shelf cell therapies."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES
BPX-601 GoCAR-T

Bellicum presented new Phase 1 translational data for BPX-601 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI) in San Francisco in January 2020. Primary observations included tumor infiltration, GoCAR-T mediated immunomodulation, survival and persistence of cells for up to nine months, and changes in the tumor microenvironment gene expression consistent with a productive CAR-T cell immune response.

Bellicum is currently enrolling cohort 5C of this trial to collect data to evaluate the safety of repeat rimiducid dosing to re-activate GoCAR-T cells over time, the first-in-human experience using the GoCAR platform as intended. Initial results from Cohort 5C are expected to be presented at a medical meeting by the end of 2020.

BPX-603 GoCAR-T

In response to Bellicum’s IND application for BPX-603, the FDA requested additional nonclinical data to further characterize this product candidate. Non-clinical experiments to generate the data are underway. Management expects to provide an update on its progress for this program in the third quarter of 2020.

BCMA GoCAR-NK Program

Bellicum recently initiated formal preclinical development activities for its GoCAR-NK program targeting B-cell maturation antigen, or BCMA, for the treatment of multiple myeloma. Bellicum presented a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November 2019 that included preclinical data that suggest the GoCAR platform may enhance NK cell proliferation, persistence, and cytotoxicity, potentially improving their utility as an off-the-shelf cancer therapy. Specifically, Bellicum believes that GoCAR-NK may improve the durability of clinical responses while offering the anticipated advantages that an allogeneic, off-the-shelf product may provide, including faster and more certain time to treatment, greater scalability and convenience, and potentially lower cost.

Management expects to present additional preclinical data for this program by the end of 2020.

Corporate Highlights

In January 2020, Bellicum entered into an asset purchase agreement under which The University of Texas MD Anderson Cancer Center will acquire Bellicum’s approximately 60,000-square-foot Houston facility, including manufacturing, office and laboratory space, for $15.0 million. As part of the transaction, Bellicum will also enter into a master services agreement with MD Anderson. Following completion of the transaction, MD Anderson will operate the Houston facility for its own internal programs as well as to manufacture Bellicum’s GoCAR and other cellular therapy programs for clinical trials and potentially early commercial supply.

In December 2019, Bellicum licensed its CaspaCIDe safety switch to MD Anderson for use in its CD19 CAR-NK program. Under terms of the original license agreement, MD Anderson exercised its option to non-exclusively license the technology for this construct and subsequently sublicensed it to a third party for future development. These actions entitled Bellicum to receive an upfront payment of $5 million and undisclosed future milestone payments and royalties on sales.

Fourth Quarter and Full Year 2019 Financial Results and Outlook
Revenue: Bellicum reported revenue of $5.1 million and $7.1 million for the fourth quarter and year ended December 31, 2019, respectively compared to $0.3 million and $1.1 million during the comparable periods in 2018. The increase in revenues in the fourth quarter and full year 2019 compared to the respective periods in 2018 were primarily due to a $5.0 million license fee received from MD Anderson for the non-exclusive license to the CaspaCIDe safety switch.
R&D Expenses: Research and development expenses were $13.3 million and $64.5 million for the fourth quarter and year ended December 31, 2019, respectively, compared to $19.9 million and $71.6 million during the comparable periods in 2018. The reduction in expenses in the fourth quarter and full year 2019 compared to respective periods in 2018 were primarily due to reduced expenses related to rivo-cel, reductions in general R&D expenses, and reduced employee salary-related charges from the reduction in force that was implemented during the second half of 2019, partially offset by the impairment of the intangible asset previously recorded from the Miltenyi Supply Agreement, increased expenses related to our GoCAR-T program, and employee severance costs arising from the reduction in force.
G&A Expenses: General and administrative expenses were $5.7 million and $30.0 million for the fourth quarter and year ended December 31, 2019, respectively, compared to $7.0 million and $25.0 million during the comparable periods in 2018. The reduction in expenses in the fourth quarter of 2019 relative to the comparable period in 2018 was primarily due to a decrease in personnel costs and share-based compensation from the reduction in force that was implemented during the second half of 2019. The increase in G&A expenses for the year ended December 31, 2019, compared to the year ended December 31, 2018, was primarily due to an increase in personnel costs and commercialization activities during the first half of 2019, partially offset by a reduction in rivo-cel related commercialization activities as well as the effects of the reduction in force that reduced employee salary-related charges.

Loss from Operations: Bellicum reported a loss from operations of $13.9 million and $87.4 million for the fourth quarter and year ended December 31, 2019, respectively, compared to a loss from operations of $26.6 million and $95.5 million for the comparable periods in 2018.
Cash used in operating activities was $12.7 million and $77.6 million for the fourth quarter and year ended December 31, 2019, respectively, compared to cash used in operating activities of $20.4 million and $74.8 million for the comparable periods in 2018.
Net Loss: Bellicum reported a net loss of $29.0 million and $112.5 million for the fourth quarter and year ended December 31, 2019, respectively, compared to a net loss of $27.2 million and $98.0 million for the comparable periods in 2018. The results included non-cash expense of $14.3 million and $19.2 million related to the change in fair value of warrant liability in the fourth quarter and year ended December 31, 2019, respectively.
Shares Outstanding: In February, Bellicum effected a reverse stock split of its issued and outstanding common stock, at a ratio of 1-for-10. As of February 28, 2020, Bellicum had 5,047,892 shares of common stock outstanding and 534,200 shares of preferred stock outstanding. Each preferred share can be converted into 10 shares of common stock.
Cash Position and Guidance: Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements into the second half of 2021. Management expects cash utilization of $55 to $65 million in 2020. Bellicum reported cash and cash equivalents, restricted cash and investments totaling $93.8 million as of December 31, 2019, compared to $106.9 million as of September 30, 2019.

Conference Call and Webcast
Bellicum’s management will host a webcast and conference call today at 5 p.m. ET / 2 p.m. PT, March 12, 2020, to discuss the financial results for the fourth quarter 2019 and provide a corporate update. The live call may be accessed by dialing (877) 407-3103 for domestic callers and (201) 493-6791 for international callers. A live webcast of the call will be available from the Investors and Media section of the company’s website at www.bellicum.com and a replay will be available shortly after the live event.

On March 12, 2020 Merck & Co reported that the rejection, which would have been for adults who have had platinum-containing chemotherapy, comes after a review of new evidence collected while the MSD blockbuster was available via the Cancer Drugs Fund.

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NICE says that there is still uncertainty surrounding the long-term benefit of the second-line immunotherapy treatment in comparison with other options, meaning it is not a "cost-effective use of NHS resources at its current price."

The organisation says that the drug will be withdrawn from the Cancer Drugs Fund once final guidance has been published, meaning that no new patients will be offered the treatment, although it will not affect those whose treatment was started before the final guidance was published.

As it stands, the list price for Keytruda is £2,630 per 100mg vial, which would be given as 200mg intravenously every three weeks. Even when the drug is offered with its agreed discount, the most plausible cost-effectiveness estimate remains above what NICE normally considers acceptable, even for end-of-life treatments.

The rejection marks the second NICE ‘no’ for MSD and Keytruda in the last month or so, as the organisation released a draft guidance rejecting the drug in combo with Pfizer’s Inlyta (axitinib) to treat advanced renal cell carcinoma in February.

Urothelial cancer begins in the lining of the bladder and is thought to be caused by prolonged exposure to harmful substances such as those found in tobacco smoke. It is the most common form of bladder cancer and accounts for nine in 10 of all cases.

On March 12, 2020 AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck & Co., Inc. inside the US and Canada) reported high-level results from the Phase III GY004 trial, led by NRG Oncology and sponsored by the US National Cancer Institute (NCI), that examined primarily the efficacy and safety of the potential new medicine cediranib added to Lynparza (olaparib) versus platinum-based chemotherapy in patients with platinum-sensitive relapsed ovarian cancer (Press release, AstraZeneca, MAR 12, 2020, View Source [SID1234555443]). Ovarian cancer is the eighth most common cause of death from cancer in women worldwide.

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The trial did not meet the primary endpoint in the intent-to-treat (ITT) population of a statistically significant improvement in progression-free survival (PFS) with cediranib added to Lynparza versus platinum-based chemotherapy. Cediranib is an oral vascular endothelial growth factor receptor (VEGFR) inhibitor, which blocks the growth of blood vessels supporting tumour growth.

José Baselga, Executive Vice President, Oncology R&D, said: "Despite these disappointing results, we remain committed to expanding on the benefits already demonstrated with Lynparza for patients with advanced ovarian cancer. We will work closely with NRG Oncology and the NCI to review the full results to inform our ongoing research."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: "Ovarian cancer is one of the most difficult tumours to diagnose and treat early. AstraZeneca, MSD and our partners will continue to explore ways to help patients through our joint clinical trial development programme."

The safety and tolerability profiles observed in GY004 were generally consistent with those known for each medicine.

The NCI and NRG Oncology will present the full data at a forthcoming medical meeting.

Ovarian cancer

Ovarian cancer is the eighth most common cause of death from cancer in women worldwide.1 In 2018, there were nearly 300,000 new cases diagnosed2 and around 185,000 deaths.1 Most women are diagnosed with advanced (Stage III or IV) ovarian cancer and have a five-year survival rate of approximately 30%.3 The primary aim of treatment in relapsed ovarian cancer is to delay progression of the disease for as long as possible, therefore increasing the time to re-initiation of chemotherapy with its associated toxicities, and maintaining quality of life for these patients.4,5,6

GY004

GY004 is an open-label, randomised, multicentre, Phase III trial testing the efficacy and safety of the potential new medicine cediranib added to Lynparza, versus Lynparza monotherapy versus standard platinum-based chemotherapy in patients with recurrent platinum-sensitive ovarian cancer, fallopian tube, or primary peritoneal cancer patients, with or without a BRCA mutation.

The GY004 trial is led by NRG Oncology, a non-profit research organisation funded by the NCI, in collaboration with AstraZeneca. The NCI is part of the National Institutes of Health. AstraZeneca provided both cediranib and Lynparza to support the trial through a Cooperative Research and Development Agreement with the NCI.

Lynparza

Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination repair, such as mutations in BRCA1 and/or BRCA2. Inhibition of PARP with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. Lynparza is being tested in a range of PARP-dependent tumour types with defects and dependencies in the DDR pathway.

Lynparza is currently approved in 73 countries, including those in the EU, for the maintenance treatment of platinum-sensitive relapsed ovarian cancer. It is approved in the US, the EU, Japan, China and several other countries as 1st-line maintenance treatment of BRCA-mutated advanced ovarian cancer following response to platinum-based chemotherapy. It is also approved in 58 countries, including the US and Japan, for germline BRCA-mutated, HER2-negative, metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer. In 2019, Lynparza was additionally approved in the US for the treatment of germline BRCA-mutated metastatic pancreatic cancer. Regulatory reviews are underway in other jurisdictions for ovarian, breast, pancreatic and prostate cancers.

Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, is approved for the treatment of advanced ovarian cancer, metastatic breast cancer and pancreatic cancer. It has been used to treat over 30,000 patients worldwide. Lynparza has the broadest and most advanced clinical-trial development programme of any PARP inhibitor, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

Cediranib

Cediranib is an oral vascular endothelial growth factor (VEGF) receptor inhibitor that has demonstrated efficacy as a monotherapy and in combination in various cancers. Cediranib has shown anti-tumour activity in many cancers, including ovarian, breast, colorectal, renal, lung, sarcoma and glioblastoma. Cediranib is being evaluated in combination with Lynparza in advanced ovarian cancer in the Phase II CONCERTO trial, Phase II/III GY005 trial (sponsored by the NCI) and Phase III ICON9 trial (sponsored by University College, London).

The AstraZeneca and MSD strategic oncology collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and selumetinib in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and selumetinib in combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With six new medicines launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to AstraZeneca’s main capabilities, the Company is actively pursuing innovative partnerships and investment that accelerate the delivery of our strategy, as illustrated by the investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.

On March 12, 2020 Harbour BioMed (HBM) reported successful completion of its Series B+ round financing of $75 million to accelerate the advancement of its clinical-stage compounds and growing portfolio of next generation biotherapeutics for treating cancer and immunological diseases (Press release, Harbour BioMed, MAR 12, 2020, View Source [SID1234555477]). New investors participating in the financing – SK Holdings, Greater Bay Area Fund, Efung Capital, Zheshang Venture Capital and Zhejiang University Future Capital and JT New Century – joined existing investors, including Legend Capital, AdvanTech and GIC Pvt. Ltd. The company previously completed an $85 million Series B financing in August 2018.

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The funds from the Series B+ will help drive these programs towards key clinical and development milestones. This round of financing further underscores both company’s existing and new investors’ confidence in HBM, and the team’s ability to execute with conviction and commitment.

"Over the past several months, HBM has made great progress in advancing two leading assets towards mid-late stage clinical trials in China, and we began international clinical studies with our first internally-discovered biotherapeutic, a next generation, fully-human anti-CTLA-4 antibody (HBM4003) for the treatment of advanced solid tumors," said Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed. "Our discovery efforts have also been extremely productive, both internally and in collaboration with leading organizations around the world. In addition to HBM4003, our innovative discovery platform HBICETM has rapidly generated a robust portfolio of differentiated, fully human antibodies, including next generation bi-and multi-specifics. More recently, we have leveraged the power of our antibody discovery platform in select other areas of great unmet medical needs, such as in coronavirus disease (COVID-19), where we have entered a collaboration with Mount Sinai Heath System in New York".

The Company recently reported on major progress in its clinical development programs, including:

· Start of Phase 1 clinical studies with HBM4003 for the treatment of advanced solid tumors. HBM4003, a next generation anti-CTLA 4 therapeutic, is the first fully human antibody based on HBM’s heavy chain only (HCAb) antibody discovery technology. The trial, underway in Australia, is the first in an international development program that will inaugurate trials in the US, EU and China. HBM recently received IND approval from the FDA to initiate its trial in the US. HBM4003 has shown potential in preclinical studies for increased anti-tumor activity based on enhanced antibody dependent cell toxicity mediated Treg depletion and a favorable safety profile.

· Start of Phase 2/3 clinical trials of HBM 9161, an anti-FcRn antibody, for the treatment of multiple severe, autoimmune diseases including myasthenia gravis, adult immune thrombocytopenia, Graves’ ophthalmology and other related indications. Phase 2/3 studies for several of these indications are expected to begin in the first half of 2020 in Greater China.

· Successful completion of a Phase 2 trial of HBM 9036 for Dry Eye Disease. HBM is preparing to start a Phase 3 registration trial in Q2 2020 in China.

· Start of Phase 2 clinical trials of HBM9167, its humanized IgG1 monoclonal antibody targeting programmed death-ligand 1 (PD-L1), for the treatment of nasopharyngeal cancer (NPC). The US FDA granted HBM9167 Orphan Drug Designation (ODD) for the development in treating NPC.

HBM has built a powerful drug discovery engine based on its patented HCAb platform for generating novel, fully human heavy chain only antibodies, including bi- and multi-specifics. The technology is integrated with other advanced approaches – e.g., single cell analysis and deep profiling— to dramatically reduce the time to candidate selection from months to days.