On May 11, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported first quarter 2020 financial results and recent business highlights (Press release, Sangamo Therapeutics, MAY 11, 2020, View Source [SID1234557503]).

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"I’m proud of how the Sangamo team has worked together through the unusual circumstances presented by the COVID-19 pandemic," said Sandy Macrae, CEO of Sangamo. "While adhering to governmental workplace guidelines, we have sought to minimize disruptions to the progress of our research in the labs, our clinical trials, and our business development discussions. Importantly, Pfizer continues to target dosing a first patient in the Phase 3 hemophilia A gene therapy study in the second half of 2020, and we are working together to identify an opportunity to present additional data from the Phase 1/2 Alta Study. Our AAV manufacturing facility in Brisbane is expected to be operational by year end, and our cell therapy manufacturing units in Brisbane and in France in 2021. With the $350 million received from the recently closed Biogen collaboration, in addition to the $363 million in cash resources reported as of March 31st, we are moving ahead with significant balance sheet strength, and we are beginning to plan for resuming normal operations as shelter-in-place orders are lifted."

Recent Highlights
•Closed collaboration agreement with Biogen for development of gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurological diseases, receiving $225 million in stock sale proceeds and an additional $125 million upfront license fee.
•Executed a collaboration and exclusive global license agreement with UK cell conversion company Mogrify Ltd for Sangamo to develop allogeneic cell therapies from Mogrify’s proprietary induced pluripotent stem cells (iPSCs) cell conversion technology using Sangamo’s zinc finger protein (ZFP) gene-engineered chimeric antigen receptor regulatory T cell (CAR-Treg) platforms. This collaboration may have the potential to accelerate the development of scalable and accessible CAR-Treg cell therapies for the treatment of inflammatory and autoimmune diseases, diversifying Sangamo’s options and complementing current programs.
•Pfizer continues to target dosing the first patient in the Phase 3 hemophilia A gene therapy study in the second half of 2020. Details of the Phase 3 trial protocol were recently posted to clinicaltrials.gov, and Pfizer continues to recruit patients into the Phase 3 lead-in study. Pfizer and Sangamo are working together to identify an appropriate opportunity this year to provide the next clinical data update of results from the ongoing Phase 1/2 Alta Study.
•Successfully screened and enrolled the first several patients into the Phase 1/2 STAAR study evaluating ST-920 gene therapy for the treatment of Fabry disease and expect to initiate patient dosing at the earliest appropriate and safe opportunity in light of the COVID-19 pandemic.
•Appointed D. Mark McClung as Executive Vice President and Chief Business Officer leading commercial strategic planning, alliance management and corporate and business development.

First Quarter 2020 Financial Results
Cash, cash equivalents and marketable securities were $363.1 million as of March 31, 2020, compared to $384.3 million as of December 31, 2019. Since the end of the first quarter, the Company has received from Biogen $225.0 million for the issuance of Sangamo stock and a $125.0 million upfront license fee.

Consolidated net loss attributable to Sangamo for the first quarter ended March 31, 2020 was $42.9 million, or $0.37 per share, compared to a net loss of $42.2 million, or $0.41 per share, for the same period in 2019. Revenues

for the first quarter ended March 31, 2020 were $13.1 million, compared to $8.1 million for the same period in 2019.
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Non-GAAP operating expenses, which excludes stock-based compensation expense, were $52.0 million for the first quarter ended March 31, 2020, compared to $47.4 million for the same period in 2019. The increase in operating expenses reflects the Company’s headcount growth and facilities expansion to support the advancement of Sangamo’s therapeutic pipeline and manufacturing capabilities. These increases were partially offset by a decrease in clinical and manufacturing supply expenses.

Financial Guidance for 2020 reiterated (initially provided on February 28, 2020)
•On a GAAP basis, we continue to expect operating expenses in the range of $270 million to $285 million, including stock-based compensation expense of approximately $25 million.
•We continue to expect non-GAAP operating expenses, which excludes stock-based compensation expense, in the range of $245 million to $260 million.

Conference Call
Sangamo will host a conference call today, May 11, 2020, at 5:00 p.m. Eastern Time, which will be open to the public. The call will also be webcast live and can be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 6043504. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 6043504.

On May 11, 2020 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that Shalini Sharp, the company’s Chief Financial Officer, will hold a virtual presentation at the BofA Securities Global Health Care Conference on Thursday, May 14, 2020 at 10:20 AM ET (Press release, Ultragenyx Pharmaceutical, MAY 11, 2020, View Source [SID1234557491]).

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The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On May 11, 2020 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported financial results for the three months ended March 31, 2020, and provided a corporate update, including the Company’s actions to continue to support public health efforts and the health and safety of employees, patients and healthcare providers during the COVID-19 pandemic (Press release, Portola Pharmaceuticals, MAY 11, 2020, View Source [SID1234557490]).

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"2020 started strong with January representing our highest month of Andexxa demand in the U.S. since launch, driven in part by a return of growth in our tier one accounts," said Scott Garland, Portola’s president and chief executive officer. "While encouraging, the emergence of the COVID-19 pandemic impacted our revenue in March and the quarter due to three main factors. First, market research and customer feedback indicate that shelter-in-place restrictions issued by over 40 states have led to fewer patients coming into emergency departments. Second, most healthcare systems shifted their focus to preparing for and addressing COVID-19 patients, which impacted the number of new accounts added in the quarter. Finally, on March 13, we suspended face-to-face field interactions with healthcare providers. Despite these challenges, we continue to execute on our growth drivers and make progress with virtual meetings and education programs to highlight the value proposition of Andexxa, including recently presented clinical data."

As previously announced on May 5, 2020, Portola entered into a definitive merger agreement to be acquired by Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN). Under the terms of the merger agreement, a subsidiary of Alexion will commence a tender offer to acquire all of the outstanding shares of Portola common stock at a price of $18 per share in cash. The tender offer is subject to customary conditions, including the tender of a majority of the outstanding shares of Portola common stock, the expiration or termination of the waiting period under the Hart-Scott Rodino Antitrust Improvements Act of 1976 and receipt of certain other regulatory approvals. Following successful completion of the tender offer, Alexion will acquire all remaining shares not tendered in the offer at the same price of $18 per share in cash through a merger. The transaction is expected to close in the third quarter of 2020.

Quarter Ending March 31, 2020, and Related Financial Results

•Total global revenues for the first quarter of 2020 were $26.4 million compared with $22.2 million for the same period in 2019. This includes $25.6 million in net product revenues from sales of Andexxa/Ondexxya [coagulation factor Xa (recombinant), inactivated-zhzo], and $0.8 million in collaboration and license revenues.

•Net loss attributable to Portola for the first quarter of 2020 was $68.8 million, or $0.88 net loss per share, compared with $78.2 million, or $1.17 net loss per share, for the same period in 2019.

•Total operating expenses for the first quarter of 2020 were $84.8 million, including $9.5 million in stock-based compensation, compared with $95.8 million for the same period in 2019. Included in total operating expenses was a stock-based compensation expense of $9.5 million for the first quarter of 2020, compared with $17.9 million, including one-time equity valuation for a manufacturer of $5.8 million, for the same period in 2019.

•Research and development (R&D) expenses for the first quarter of 2020 were $26.1 million compared with $35.6 million for the same period in 2019. Included in R&D expenses was a stock-based compensation expense of $2.6 million for the first quarter of 2020, compared with $10.1 million, including one-time equity valuation for a manufacturer of $5.8 million, for the same period in 2019.

•Selling, general and administrative (SG&A) expenses for the first quarter of 2020 were $54.4 million compared with $53.0 million for the same period in 2019. Included in SG&A expenses was a stock-based compensation expense of $6.9 million for the first quarter of 2020, compared with $7.8 million for the same period in 2019.

•Cost of sales (COS) for the first quarter of 2020 was $4.3 million compared with $7.2 million for the same period in 2019.

Cash, Cash Equivalents and Investments

Cash, cash equivalents and investments at March 31, 2020, totaled $394.1 million compared with $466.2 million as of December 31, 2019.

Updated 2020 Annual Financial Guidance

Portola is reducing its 2020 operating expenses by approximately $50 million. For the fiscal year 2020, Portola is updating its guidance for R&D and SG&A expenses. Portola now expects GAAP R&D expenses to be between $90 million and $105 million, including stock-based compensation expenses of approximately $15 million, a decrease of $15 million from the prior guidance range of $105 million and $120 million. Portola now expects GAAP SG&A expenses to be between $200 million and $215 million, including stock-based compensation expenses of approximately $38 million, a decrease of $35 million from the prior guidance range of $235 million and $250 million. This updated guidance reflects near-term cost containment measures to extend the cash runway as Portola navigates the COVID-19 pandemic.

With the uncertainty around the duration of COVID-19 impacts, Portola is suspending prior guidance provided during its fourth quarter 2019 call for approximately 350 new hospital adds in 2020.

COVID-19 Response

As the global pandemic continues to evolve rapidly, Portola’s first priority is the health and safety of employees, patients and healthcare providers. Effective March 13, 2020, the Company suspended face-to-face field activity and instituted a mandatory work-from-home policy for all employees, including those in the Company’s South San Francisco and European headquarters. The Company shifted to a virtual field force to continue engaging customers with digital tools and remote meetings where possible. Portola’s plan to present and publish data throughout the year remains intact.

At this time, the global Andexxa supply chain and distribution structure is intact for customers to continue to use and re-order Andexxa. Portola has adequate supply of this important medicine on hand for the next couple of years in the United States and Europe.

Recent Achievements and Events

·Added 68 new accounts in the first quarter of 2020. Andexxa is now stocked in over 700 U.S. hospitals.

·Establishment of a permanent J-code for Andexxa by the Centers for Medicare & Medicaid Services, effective July 1, 2020, ensuring greater patient access by providing hospitals with a clearer reimbursement pathway when administering Andexxa in the outpatient hospital setting.

·Presented the budget impact model demonstrating that using Andexxa may provide a net cost reduction for the treatment of intracranial hemorrhage (ICH) associated with oral Factor Xa inhibitors. The analysis, presented at the Emergencies on Medicine conference, projects that hospital use of Andexxa with NTAP reimbursement can reduce cost per hospitalization by up to $5,400 compared to 4F-PCC’s.

·Presented new data at ACC demonstrating that Andexxa was associated with a lower rate of in-hospital and 30-day mortality in patients with multiple types of life-threatening Factor Xa inhibitor-related bleeds compared with other treatment options. Propensity matched data from the ANNEXA-4 and ORANGE studies showed 30-day mortality was 15% with Andexxa versus 34% with 4F-PCC across all bleed types. Additionally, in a real-world database analysis, in-hospital mortality was 4% with Andexxa and 10% with 4F-PCC across all bleed types.

·Agreed to terminate the collaboration and license agreement with Bristol-Myers Squibb Company and Pfizer, Inc. regarding the development and commercialization of andexanet alfa in Japan. Portola will regain full Japanese rights for andexanet alfa. Japan represents the third largest market for Factor Xa inhibitors after the United States and the EU 5 countries. Portola will have exclusive rights to develop and commercialize andexanet alfa in the United States, Europe, Japan and rest of the world markets.

Planned Upcoming Milestones

·Present and publish comparative clinical, mechanistic and economic data supporting the adoption of Andexxa at medical meetings and in peer-reviewed journals throughout the year including:

oThe ANNEXA-4 study ICH subgroup

oRetrospective comparisons of Andexxa vs. 4F-PCC’s on clinical outcomes using data from the ANNEXA-4, ORANGE and RETRACE studies

·Advance reimbursement discussions in the United Kingdom (2H 2020), Germany (2H 2020) and other Wave 1 European countries.

·Submit a supplemental Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by year end for inclusion of the additional Factor Xa inhibitors edoxaban and enoxaparin in the Andexxa label.

On May 11, 2020 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported financial results for the first quarter ended March 31, 2020 and provided an update on its recent corporate activities and outlook (Press release, Halozyme, MAY 11, 2020, View Source [SID1234557489]).

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"This has been a very exciting start to the year for Halozyme as we have achieved multiple value-creating events in our ENHANZE business, including U.S. FDA approval for DARZALEX FASPROTM with a broad set of label indications, and the receipt of a positive CHMP opinion in the EU, also recommending a broad set of label indications. The positive CHMP opinion is typically a precursor to marketing clearance," said Dr. Helen Torley, president and chief executive officer. "As we look ahead, we are excited about the launches of subcutaneous DARZALEX in the U.S. and E.U. as well as the potential FDA approval of the subcutaneous fixed-dose combination of Perjeta and Herceptin later this year, and what they may mean for patients."

"I want to express my gratitude to the Halozyme team, our partners and suppliers for our continued progress in spite of the challenges posed by COVID-19," continued Dr. Torley. "In light of these challenges, it is obviously difficult to predict how the pandemic recovery will unfold in the coming quarters. However, based on the latest information from our partners and suppliers, and our team’s commitment to maintaining a lean operating structure, we feel confident maintaining our 2020 financial guidance at this time."

First Quarter 2020 and Recent Highlights Include:

On May 1, the Company announced that The Janssen Pharmaceutical Companies of Johnson and Johnson received U.S. FDA approval of DARZALEX FASPROTM in four regimens across five indications in multiple myeloma patients, including newly diagnosed, transplant-ineligible patients as well as relapsed or refractory patients. As a fixed-dose formulation, DARZALEX FASPROTM can be administered subcutaneously over three to five minutes, significantly less time than IV DARZALEX which requires multi-hour infusions.
On April 30, the Company announced that Janssen-Cilag International NV (Janssen) received a Committee for Medicinal Products for Human Use (CHMP) Positive Opinion from the European Medicines Agency (EMA) recommending approval of a DARZALEX (daratumumab) subcutaneous (SC) formulation for the treatment of adult patients with multiple myeloma in frontline and relapsed/refractory settings. The CHMP’s Positive Opinion for daratumumab SC formulation applies to multiple current daratumumab indications including newly diagnosed and transplant-ineligible patients, as well as relapsed or refractory patients.
In April, the Company announced the submission of a New Drug Application (NDA) to Japan’s Ministry of Health, Labour and Welfare (MHLW) by Janssen Pharmaceutical K.K. (Janssen) seeking approval of a new subcutaneous (SC) formulation of daratumumab, an intravenous (IV) treatment approved for patients with multiple myeloma.
During the first quarter, the Company repurchased 3.2 million shares of its common stock at a weighted average price of $16.15 per share. These repurchased shares were in addition to shares repurchased as part of an Accelerated Share Repurchase plan that was completed in mid-February. To date the Company has repurchased over $250 million in shares as part of its three-year share repurchase authorization of up to $550 million approved by the Board in November 2019.
In February, the Company announced that the FDA has accepted a Biologics License Application (BLA) from Genentech, a member of the Roche Group, for the fixed-dose combination of pertuzumab (Perjeta) and trastuzumab (Herceptin ) for subcutaneous administration using ENHANZE technology in combination with IV chemotherapy for the treatment of eligible patients with HER2-positive breast cancer, with an action date of October 18, 2020.
First Quarter 2020 Financial Highlights

Revenue for the first quarter was $25.4 million compared to $56.9 million for the first quarter of 2019. The year-over-year decrease was primarily driven by a $30 million upfront payment from argenx in the prior year period. Revenue for the quarter included $16.8 million in royalties, which compared to $18.0 million in the prior year period.
Research and development expenses for the first quarter were $10.2 million, compared to $31.3 million for the first quarter of 2019. The decrease in expenses was due to a decrease in clinical trial activities-related costs as a result of the Company halting its oncology drug development efforts and related restructuring as announced in November 2019.
Selling, general and administrative expenses for the first quarter were $12.6 million, compared to $18.0 million for the first quarter of 2019. The decrease was due to lower compensation and commercial-related expenses related to the corporate restructuring announced in November 2019.
Net loss for the first quarter was $6.1 million, or $0.04 per share, compared to a net income in the first quarter of 2019 of $1.8 million, or $0.01 per share.
Cash, cash equivalents and marketable securities were $368.2 million at March 31, 2020, compared to $421.3 million at December 31, 2019.
Financial Outlook for 2020

The Company continues to monitor the impact of the COVID-19 pandemic on its business and receive updates from its partners and suppliers on how their businesses are affected. Based on this information and Halozyme’s planned expenditures for the year, the Company’s 2020 financial guidance remains unchanged from that first provided on January 14, 2020. For 2020 Halozyme continues to expect:

Revenues of $230 million to $245 million, representing growth of 17% to 25%;
Earnings per share on a GAAP basis of $0.60 to $0.75 with the first quarter of sustainable profitability beginning in Q2 2020.
The guidance on earnings per share does not reflect any potential impact from the Company’s plans to repurchase any additional number of shares, up to an additional $98 million worth, during the remainder of 2020. The amount and timing of shares repurchased during 2020 will be subject to a variety of factors including market conditions, other business considerations and applicable legal requirements.

Webcast and Conference Call

Halozyme will webcast its Quarterly Update Conference Call for the first quarter of 2020 today, Monday, May 11, 2020 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Torley will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website and a replay will be available following the close of the call. To access the webcast and additional documents related to the call, please visit halozyme.com approximately fifteen minutes prior to the call to register, download and install any necessary audio software. The call may also be accessed by dialing (833) 968-2181 (domestic callers) or (825) 312-2108 (international callers). A telephone replay will be available after the call by dialing (800) 585-8367 (domestic callers) or (416) 621-4642 (international callers) using replay ID number 3199114.

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.