On May 11, 2020 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 first quarter ended March 31, 2020 (Press release, Fate Therapeutics, MAY 11, 2020, View Source [SID1234557488]).

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"We are encouraged by the resilience of our employees, our clinical trial investigators and participating patients, and our collaboration partners in the face of the challenge posed by the global pandemic. Like others, we have been affected by COVID-19, which has impacted clinical site initiation, slowed the cadence of new patient enrollment, and changed how we conduct our day-to-day business," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "Nevertheless, we have continued to enroll patients across our three Phase 1 clinical programs, expanded the clinical footprint of our FT596 program into relapse prevention following autologous HSCT, and submitted our IND application to the FDA for FT538, the first-ever CRISPR-edited, iPSC-derived cell therapy, in multiple myeloma. Additionally, we entered into a transformative collaboration with Janssen that leverages our iPSC product platform and Janssen’s proprietary tumor-targeting antigen binders to develop novel CAR NK and CAR T-Cell product candidates for hematologic malignancies and solid tumors, supporting our fundamental goal of bringing off-the-shelf, iPSC-derived cell-based cancer immunotherapies to patients."

Clinical Programs

First Patient Treated with FT596 Monotherapy for Advanced Diffuse Large B-cell Lymphoma. FT596 is the industry’s first investigational cellular immunotherapy engineered with three active anti-tumor modalities to be evaluated in patients. The Company is currently conducting an open-label Phase 1 clinical trial of FT596 in patients with relapsed / refractory B-cell malignancies and chronic lymphocytic leukemia. FT596 is an off-the-shelf, multi-antigen targeted, chimeric antigen receptor (CAR) natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a proprietary CD19-targeting CAR, a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, and a unique interleukin-15 receptor fusion (IL-15RF). The hnCD16 Fc receptor enables concurrent targeting of additional tumor-associated antigens bound by therapeutic antibodies to overcome antigen escape and promote multifaceted tumor cell killing, and IL-15RF is a potent cytokine complex that promotes survival, proliferation and trans-activation of NK cells and CD8 T cells without the need for exogenous cytokine support.
Second FT596 IND Application Allowed by FDA for Relapse Prevention after Autologous HSCT. The open-label Phase 1 study, which is sponsored by investigators from the Masonic Cancer Center, University of Minnesota, is intended to assess the potential of FT596 to prevent relapse in patients undergoing autologous hematopoietic stem cell transplant (HSCT) for the treatment of non-Hodgkin lymphoma. The clinical trial is expected to enroll up to 18 patients considered high risk for early relapse based on failure to achieve complete or partial remission on, or relapse within twelve months of, prior therapy. Up to three dose levels of FT596 will be administered in combination with rituximab approximately 30 days following HSCT.
FT500 Phase 1 Dose-Escalation Stage Successfully Completed for Advanced Solid Tumors. Three patients have been treated in the dose-escalation stage of the FT500 Phase 1 study at 300M cells per dose in combination with checkpoint inhibitor therapy. There were no dose-limiting toxicities, no FT500-related Grade ≥3 adverse events (AEs) or serious adverse events (SAEs), and no incidents of cytokine release syndrome, neurotoxicity, or graft-versus-host disease reported by investigators. Enrollment into the dose-expansion stage of the FT500 Phase 1 study is proceeding in patients with non-small cell lung cancer (NSCLC) who are refractory to, or have relapsed following, checkpoint inhibitor therapy. The Company intends to treat up to 15 patients in the outpatient setting, administering three once-weekly doses of FT500 at 300M cells per dose over up to two 30-day cycles with IL-2 cytokine support and the same checkpoint inhibitor on which the patient failed or progressed.
FT516 Clinical Investigation Expanded to Solid Tumors. In January, the Company announced that the U.S. Food & Drug Administration (FDA) allowed its second IND application for FT516, the Company’s off-the-shelf NK cell cancer immunotherapy derived from a clonal master iPSC line engineered to express hnCD16 Fc receptor, enabling clinical investigation in combination with PDL1-, PD1-, EGFR- and HER2-targeting monoclonal antibody (mAb) therapies across a broad range of solid tumors. The Company intends to initially evaluate FT516 in combination with avelumab in patients with advanced solid tumors who are refractory to, or have relapsed following, at least one line of anti-PDL1 mAb therapy. The multi-dose treatment course consists of three once-weekly doses of FT516 over up to two 30-day cycles. The Company is currently conducting an open-label, multi-dose Phase 1 clinical trial of FT516 as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-directed monoclonal antibodies for the treatment of advanced B-cell lymphoma.
IND Application Submitted for FT538, the First CRISPR-edited, iPSC-derived Cellular Immunotherapy. FT538 is derived from a clonal master iPSC line engineered with hnCD16 and IL-15RF and edited for elimination of CD38 expression (CD38KO) to mitigate anti-CD38 antibody-mediated fratricide. The Company has recently submitted an IND application to the FDA for the clinical investigation of FT538 in combination with anti-CD38 monoclonal antibody therapy for the treatment of multiple myeloma.
FT516 Investigator-initiated Clinical Trial for COVID-19 Opened to Enrollment. Sponsored by investigators from the Department of Medicine, Division of Infectious Diseases and International Medicine, University of Minnesota, the open-label Phase 1 study is designed to assess the clinical safety and tolerability of FT516 for the treatment of Coronavirus Disease 2019 (COVID-19) (NCT04363346). The study is evaluating three FT516 dose-escalating strategies (90M cells on Day 1; 90M cells on Day 1 and 300M cells on Day 4; and 90M cells on Day 1, 300M cells on Day 4, and 900M cells on Day 7) in up to 20 patients at a high risk of developing critical life-threatening illness. Secondary objectives of the study include time to elimination of viral shedding, to discontinuation of supplemental oxygen support, and to hospital discharge.
Corporate Highlights

Strategic Collaboration Formed with Janssen for Novel iPSC-derived Cell-based Cancer Immunotherapies. In April, the Company entered into a global collaboration and option agreement with Janssen Biotech, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop iPSC-derived chimeric antigen receptor (CAR) NK and CAR T-cell product candidates targeting up to four tumor-associated antigens for which Janssen is contributing proprietary antigen binding domains. The Company is eligible to receive payments of up to $3.0 billion upon the achievement of certain development, regulatory and commercial milestones, plus tiered double-digit royalties on worldwide net sales of products targeting the antigens. In addition, the Company has the right to elect to co-commercialize each product candidate in the U.S. and share equally in profits and losses in the U.S., subject to its payment of certain clinical development costs and adjustments in milestone and royalty payments. The Company received $100 million upon entering into the collaboration, including $50 million in an upfront cash payment and $50 million from the purchase by Johnson & Johnson Innovation – JJDC, Inc. of newly issued shares of the Company’s common stock at a price per share of $31.00.
Lease Executed for New Corporate Headquarters with cGMP Cell Manufacturing Facility. In January, the Company entered into a lease agreement for the entirety of the Scripps Northridge Corporate Center, a 200,000-square-foot life sciences complex in San Diego, CA that is designed to include a 40,000 square foot cGMP cell manufacturing facility. The Company intends to move its corporate headquarters to the campus in the middle of 2021.
Upcoming Scientific Presentations

IND-enabling Data for FT819 iPSC-derived CAR T-cell to be Presented at AACR (Free AACR Whitepaper) II. IND-enabling data for FT819, which is derived from a clonal master iPSC line engineered with a novel 1XX CAR targeting CD19 inserted into the T-cell receptor alpha constant (TRAC) locus and edited for elimination of T-cell receptor (TCR) expression, is scheduled to be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II (June 22-24). In the second quarter of 2020, the Company intends to submit an IND application to the FDA for clinical investigation of FT819, the Company’s first off-the-shelf, iPSC-derived CAR T-cell product candidate.
FT576 Master iPSC Line Engineered with Four Anti-Tumor Modalities to be Highlighted at ASGCT (Free ASGCT Whitepaper). FT576 is the Company’s off-the-shelf, iPSC-derived, multi-antigen targeted CAR-BCMA NK cell product candidate for multiple myeloma that is currently undergoing preclinical development. At the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Virtual Annual Meeting (May 12-15), the Company will highlight an innovative paradigm to generate new clonal master iPSC lines for next-generation product development. Starting with an existing clonal master iPSC line engineered with three anti-tumor modalities (hnCD16; IL15-RF; and CD38KO) as a cell backbone, a CAR-BCMA transgene was engineered into the backbone, followed by single-cell selection and generation of a new clonal master iPSC line incorporating CAR-BCMA as a fourth functional element.
New iPSC-derived CAR MICA/B Solid Tumor Program to be Presented at ASGCT (Free ASGCT Whitepaper). The Company plans to present initial preclinical data for its new off-the-shelf, iPSC-derived CAR MICA/B cancer immunotherapy program at ASGCT (Free ASGCT Whitepaper). MICA and MICB are pan-tumor associated stress proteins induced by malignant transformation, and proteolytic shedding of MICA/B by cancer cells is a common mechanism of immune cell evasion broadly observed across solid tumors. The Company’s proprietary CAR MICA/B program targets a specific protein region which prevents shedding to overcome tumor escape.
First Quarter 2020 Financial Results

Cash & Investment Position: Cash, cash equivalents and investments as of March 31, 2020 were $219.4 million. The Company’s cash, cash equivalents and investments exclude the receipt of $100.0 million in April 2020 in connection with entering into the Janssen collaboration.
Total Revenue: Revenue was $2.5 million for the first quarter of 2020, which was derived from the Company’s collaboration with Ono Pharmaceutical.
R&D Expenses: Research and development expenses were $29.3 million for the first quarter of 2020, which includes $4.3 million of non-cash stock-based compensation expense.
G&A Expenses: General and administrative expenses were $7.7 million for the first quarter of 2020, which includes $2.7 million of non-cash stock-based compensation expense.
Shares Outstanding: Common shares outstanding were 76.0 million, and preferred shares outstanding were 2.8 million, as of March 31, 2020. Each preferred share is convertible into five common shares. Common shares outstanding excludes the issuance of 1.6 million shares in April 2020 in connection with entering into the Janssen collaboration.
Today’s Conference Call and Webcast

The Company will conduct a conference call today, Monday, May 11, 2020 at 5:00 p.m. ET to review financial and operating results for the quarter ended March 31, 2020. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 8623509. 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 May 11, 2020 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the first quarter ended March 31, 2020 (Press release, CymaBay Therapeutics, MAY 11, 2020, View Source [SID1234557487]).

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Sujal Shah, President and CEO of CymaBay, stated, "Last week a panel of some of the most experienced and distinguished liver pathologists and hepatologists in the world completed an independent review analyzing findings from our Phase 2b study of seladelpar in patients with NASH. I am pleased to report that the panel unanimously concluded there was no clinical, biochemical or histological evidence of seladelpar-induced liver injury in the study, and as a result they also unanimously supported the lifting of the clinical hold and re-initiation of clinical development.

While we have not yet discussed full results from our investigation nor any of the panel’s conclusions with the FDA, we are planning to re-engage with the agency as quickly as possible. At this point we cannot guarantee what the next steps or timelines will be, but we are confident that we have conducted a truly rigorous, independent review to help us definitively support the conclusion that seladelpar did not cause drug-induced liver injury in our NASH phase 2b study."

Dr. Paul Watkins, Howard Q Ferguson Distinguished Professor, Schools of Medicine, Pharmacy, and Public Health, Director, Institute for Drug Safety Sciences at the University of North Carolina, Chapel Hill, said, "I was pleased to chair this esteemed independent panel of liver experts. The panel conducted a comprehensive, systematic review and discussion of all of the clinicopathological data from the seladelpar NASH Phase 2b study. In my experience, no other drug in development for NASH has been through such rigorous scrutiny of safety data at this stage of development. As we have stated, the features noted by study pathologists at end of treatment were confirmed on this review. However, these did not differ qualitatively between baseline and end of treatment. We suspect these histologic features are underreported; however, in the experience of the pathology review subcommittee, these features may be observed in patients with NASH. The panel unanimously concluded that the data in aggregate, including the lack of significant differences in histologic features or their changes across the placebo and treatment groups, do not support injury related to seladelpar."

Dr. Stephen Harrison, Medical Director, Pinnacle Clinical Research, Visiting Professor of Hepatology at Radcliffe Department of Medicine, University of Oxford, and principal investigator of the seladelpar Phase 2b study in NASH, added, "I believe CymaBay and the FDA did the right thing in putting patient safety first when development of seladelpar was halted at the end of last year until an in-depth investigation was conducted into the findings identified by study pathologists in the NASH study. At this point, the findings and additional data collected have been thoroughly investigated by leading experts in the areas of drug-induced liver injury and hepatopathology. Given the benefit observed on both NASH resolution and fibrosis with seladelpar in the NASH Phase 2b study as well as data presented at multiple medical meetings from studies of seladelpar in PBC, I am pleased that the independent review panel is supportive of restarting clinical development pending approval from the FDA."

Recent Corporate Highlights

At the end of last week, a panel of eight of the world’s foremost expert liver pathologists and hepatologists, whose collective experience relevant to CymaBay’s investigation includes drug-induced liver injury, NASH and cholestatic liver diseases, completed a four-day independent review analyzing findings from CymaBay’s NASH Phase 2b study. The panel unanimously supported lifting the clinical hold for seladelpar and re-initiation of clinical development. In addition to the chair, Dr. Paul Watkins, the panel included:
° Pierre Bedossa, MD, PhD, Professor of Pathology at the University Paris-Diderot, France, and Medical Director and CEO of LIVERPAT
° Michael Charlton, MD, Chief of Hepatology, Director of the Center for Liver Diseases and Medical Director of the Transplant Institute at the University of Chicago
° Zachary Goodman, MD, PhD, Director of Hepatic Pathology Consultation and Research, Center for Liver Disease, Inova Healthcare Services
° Neil Kaplowitz, MD, Professor of Medicine and Thomas H. Brem Chair in Medicine, Budnick Chair of Liver Disease, Keck School of Medicine, University of Southern California
° David Kleiner, MD, PhD, Head of Histopathology and Autopsy Pathology at the NIH and the Reference Pathologist for the Drug-Induced Liver Injury Network
° Willis Maddrey, MD, Professor Emeritus of Internal Medicine at The University of Texas Southwestern Medical Center
° John Vierling, MD, Professor of Medicine and Surgery, Baylor College of Medicine

CymaBay intends to reach out to the FDA to discuss all of the data it has collected to date and the results of the panel review meetings. Once initial feedback is gathered, CymaBay intends to submit a complete response to the seladelpar clinical hold to the FDA. The CymaBay Board of Directors has worked closely with management throughout the investigation and panel review and is in support of next steps to re-engage with the FDA.

As a reminder, during the fourth quarter of 2019, management implemented a restructuring program following the placement of the seladelpar program on clinical hold pending further investigation of the histologic observations noted by study pathologists in CymaBay’s Phase 2b NASH study and pending completion of its review of strategic options.

Late in the first quarter of 2020, the need for sustained cost containment was further underscored by the unexpected and rapid onset of the coronavirus pandemic and the associated travel restrictions and shelter-in-place orders issued by governmental authorities in jurisdictions where CymaBay, its partners, investigators, and vendors, conduct operations. In response to these measures, CymaBay has taken steps, such as enabling remote operations for all employees, which have allowed operating activities to continue as seamlessly as possible.

CymaBay will continue to closely monitor pandemic developments and their associated risks to the business, and will take actions available to mitigate them where possible. Further, all of CymaBay’s actions will be guided by a commitment to taking all steps possible to ensure the health and safety of its employees.

Held $176.2 million in cash, cash equivalents and short-term investments at March 31, 2020.
Mr. Shah continued, "As the next steps in our seladelpar investigation process become clear we will continue to keep our shareholders updated as appropriate, while also continuing to evaluate potential strategic alternatives. Further, we remain focused on cost containment and will look at additional steps we can take into fiscal year 2020 in order to closely control the Company’s operating expenses and associated cash burn."

First Quarter Ended March 31, 2020 Financial Results

Research and development expenses for the three months ended March 31, 2020 were $9.5 million, compared to $18.6 million for the three months ended March 31, 2019. Research and development expense in the first quarter of 2020 was significantly lower than the corresponding period in 2019 primarily due to declining clinical trial activities related to the Phase 3 PBC, Phase 2b NASH, and Phase 2 PSC clinical trials, and other studies, as efforts continue to scale back and shut down these studies as a result of the clinical hold on the seladelpar development program.

General and administrative expenses for the three months ended March 31, 2020 were $4.3 million, compared to $5.7 million for the three months ended March 31, 2019. General and administrative expenses in the first quarter of 2020 was lower than the corresponding period in 2019 due to lower continuing labor costs and other administrative expenses following restructuring efforts undertaken in the fourth quarter of 2019.

Net loss for the three months ended March 31, 2020 was $13.1 million, or ($0.19) per diluted share, compared to a net loss of $23.1 million, or ($0.37) per diluted share in the three months ended March 31, 2019. Net loss was lower in the first quarter of 2020 compared to the corresponding period in 2019 primarily due to a decrease in operating expenses, including clinical trial and labor related expenses.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss first quarter 2020 financial results and provide a corporate update. To access the live conference call, please dial 855-327-6837 from the U.S. and Canada, or 631-891-4304 internationally, Conference ID# 10009543. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

On May 11, 2020 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the first quarter ended March 31, 2020 and recent company developments (Press release, TG Therapeutics, MAY 11, 2020, View Source [SID1234557486]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "The first few months of 2020 have undoubtedly been the most impactful and exciting in our Company’s history. We kicked off the year with the initiation of our first rolling regulatory submission for umbralisib in both MZL and FL and most recently reported positive topline results from our UNITY-CLL Phase 3 trial evaluating our proprietary U2 combination in patients with CLL. This positive outcome marks a major step forward in our mission of developing the best possible combination treatment options for patients with B-cell diseases." Mr. Weiss continued, "We now have three successful pivotal data sets which we believe have the potential to support regulatory approvals across MZL, FL and CLL. With more than $150 million proforma in cash on our balance sheet, we are well funded through and beyond our next set of key milestones, including the release of topline data from the ULTIMATE MS Phase 3 program, submission of an NDA/BLA for U2 in CLL, and hopefully, our first approval for umbralisib in MZL and FL, all of which are targeted to occur over approximately the next 9 months."

Recent Developments and Highlights

Chronic Lymphocytic Leukemia:

In May 2020, reported positive topline results from the Company’s UNITY-CLL Phase 3 trial evaluating U2 (the combination of umbralisib and ublituximab) in patients with previously untreated and relapsed/refractory chronic lymphocytic leukemia (CLL). The trial met its primary endpoint of improved progression-free survival (PFS) (p<.0001), as determined by an Independent Review Committee (IRC) and will be stopped early for superior efficacy. Regulatory submission and full data presentation targeted by year-end 2020.
Marginal Zone Lymphoma & Follicular Lymphoma:

In January 2020, received guidance from the U.S. Food and Drug Administration (FDA) allowing submission of a single New Drug Application (NDA) for marginal zone lymphoma (MZL) and follicular lymphoma (FL) indications. A rolling NDA submission for umbralisib to treat adult patients with previously treated MZL and FL was initiated, with completion of submission targeted in the first half of 2020.
In March 2020, received orphan drug designation for umbralisib from the FDA for the treatment of FL.
Multiple Sclerosis:

In May 2020, announced the publication of results from the multicenter Phase 2 trial evaluating ublituximab in patients with relapsing forms of multiple sclerosis (RMS) in the Multiple Sclerosis Journal.
Awaiting topline data from the Company’s Phase 3 ULTIMATE I & II trials evaluating ublituximab in patients with RMS, targeted in second half 2020.
Board of Directors & Management:

In May 2020, appointed Sagar Lonial, MD, FACP, Professor and Chair of the Department of Hematology and Medical Oncology at the Emory University School of Medicine, as well as the Chief Medical Officer at Winship Cancer Institute of Emory University, to the Company’s Board of Directors.
In May 2020, strengthened executive team with the addition of Owen A. O’Connor, MD, PhD as Chief Scientific Officer. Dr. O’Connor most recently served as a Professor of Medicine and Experimental Therapeutics, the Director of the Center for Lymphoid Malignancies, and Co-Program Director of the Lymphoid Development and Malignancy Program in the Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center.
Bolstered Balance Sheet:

In May 2020, strengthened balance sheet with more than $75 million in gross proceeds through the Company’s At-the-Market (ATM) facility, $40 million of which came from a longtime shareholder.

Key Objectives for 2020

Complete rolling NDA submission for umbralisib in patients with previously treated MZL and FL, in the first half of 2020.
Report topline results from the Phase 3 ULTIMATE I & II trials in RMS, in the second half of 2020.
Present full data from the UNITY-CLL Phase 3 trial and present full data from the FL and MZL umbralisib monotherapy cohorts of the UNITY-NHL trial at a major medical meeting, by year-end 2020.
Target an NDA/Biologics Licensing Application (BLA) submission of U2 for the treatment of patients with CLL (including both previously untreated and relapsed/refractory patients), by year end 2020.
Continue to advance our early pipeline candidates including our anti-PD-L1 monoclonal antibody, cosibelimab (TG-1501), our covalently-bound Bruton’s Tyrosine Kinase (BTK) inhibitor, TG-1701, and our anti-CD47/CD19 bispecific antibody, TG-1801.

Financial Results for the Three Months Ended March 31, 2020

R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation) was $34.0 million for the three months ended March 31, 2020, compared to $30.9 million for the three months ended March 31, 2019. The modest increase in R&D expense is primarily attributable to costs associated with the preparation of the Company’s NDA filing for umbralisib in MZL and FL. We expect our R&D expenses to decrease during 2020 as costs associated with our main pivotal clinical trials continue to decline over the remainder of the year, partially offset by expenses associated with the expected NDA/BLA filing for U2 in CLL.

G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $5.2 million for the three months ended March 31, 2020, as compared to $1.9 million for the three months ended March 31, 2019. The increase in other G&A expenses is primarily due to the build out of our commercial team and infrastructure in anticipation of the potential commercialization of umbralisib and ublituximab. We expect G&A expenses to increase modestly during the remainder of 2020.

Net Loss: Net loss was $51.1 million for the three months ended March 31, 2020, compared to a net loss of $35.2 million for the three months ended March 31, 2019. Excluding non-cash compensation, the net loss for the three months ended March 31, 2020 was approximately $40.0 million, compared to a net loss of $33.3 million for the three months ended March 31, 2019.

Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $78.3 million as of March 31, 2020. Pro forma cash, cash equivalents and investment securities as of March 31, 2020 are approximately $154.3 million, after giving effect to $76.0 million of net proceeds from the utilization of the Company’s ATM sales facility during the second quarter of 2020 at an average price of $17.07. The Company believes its cash, cash equivalents and investment securities on hand as of March 31, 2020, inclusive of the proceeds raised from the ATM facility, as well as future availability under the Company’s debt and ATM facility, will be sufficient to fund the Company’s planned operations through the end of 2021.

On May 11, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported that it has amended its existing co-promotion/co-development agreement with Bristol Myers Squibb (BMS) to enable the companies to focus their efforts on efficient commercialization of idecabtagene vicleucel (ide-cel; bb2121) in the U.S., the companies’ lead investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, currently in review with the FDA (Press release, bluebird bio, MAY 11, 2020, http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-amended-bcma-car-t-collaboration [SID1234557484]).

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"Under our amended collaboration, we and BMS are redoubling our commitment to ide-cel and optimizing the relationship as we work together to bring this critical treatment to patients in the commercial setting," said Joanne Smith-Farrell, PhD, Chief Business Officer and Oncology Franchise Leader, bluebird bio. "With bluebird exiting the passive participation as supplier outside the U.S., we and BMS are taking steps to ensure an efficient and robust supply chain for this program. This, together with the monetization of our ex-U.S. royalties and milestones will allow bluebird to continue to participate in co-developing and co-commercializing ide-cel within the U.S. and to refocus resources on our internal programs and pipeline."

"Our collaboration with bluebird has resulted in the first CAR T cell therapy submitted for regulatory approval to target the B-cell maturation antigen and for multiple myeloma," said Krishnan Viswanadhan, Pharm.D., Senior Vice President, Global Cell Therapy Franchise Lead for Bristol Myers Squibb. "This amended partnership allows Bristol Myers Squibb to leverage our global manufacturing capabilities and consolidate all responsibilities outside the United States."

The companies will continue to share equally profits and losses in the U.S. Under the terms of the amended agreement, BMS will buy out its obligations to pay bluebird bio future ex-U.S. milestone and royalty payments for ide-cel and bb21217, the companies’ second BCMA-directed CAR T immunotherapy, for a one-time upfront payment of $200 million. bluebird bio is currently in the process of building out and qualifying its wholly-owned manufacturing facility in Durham, North Carolina for the production of lentiviral vector (LVV) to support the U.S. commercial market for ide-cel and for bluebird bio’s pipeline. Over time, BMS will assume responsibility for manufacturing of LVV outside the U.S.

On May 11, 2020 Portage Biotech Inc. ("Portage" or "the Company") (Canadian Securities Exchange: PBT.U, OTC Markets : PTGEF), reported that It has made an additional €900k investment in its associate, Stimunity, the Paris-based cancer immunotherapy company focused on STING (Press release, Portage Biotech, MAY 11, 2020, View Source [SID1234557483]). Stimunity has reached a major milestone in its preclinical development plan and the additional financing will enable it to start the manufacturing of its biologic cGAMP-VLP (STI-001) lead compound.

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"We knew since the beginning of the company that STING-activating cGAMP Virus-Like Particle (cGAMP-VLP) technology has a unique property enabling its payload to preferentially target immune cells, which is different from other chemical STING approaches. This has an impact on the stimulation of the immune system and the quality of the anti-tumoral response. We have determined that this targeting mechanism is able to deliver the cGAMP via systemic route of administration and that it leads to induction of systemic anti-tumor T-cell response" says Nicolas Manel, co-founder and Scientific Advisor of the company.

"These new data demonstrate that picking the right approach to modulate STING is key. The use of VLP carrier enables a simple systemic approach to deliver STING modulation directly to immune cells. Portage is pleased with the work performed by Stimunity and this new round of financing will support the progress toward clinical trials" says Dr. Ian Walters, Chairman of Stimunity’s Supervisory Board and CEO of Portage.