On April 27, 2021 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, reported that two oral and two digital presentations of the Company’s induced pluripotent stem cell (iPSC) product platform were accepted for presentation at the 24th American Society of Gene & Cell Therapy Annual Meeting (ASGCT) (Free ASGCT Whitepaper) being held virtually from May 11-14, 2021 (Press release, Fate Therapeutics, APR 27, 2021, View Source [SID1234578561]).

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In addition to the Company’s presentations at ASGCT (Free ASGCT Whitepaper), its iPSC-derived natural killer (NK) cell product pipeline is expected to be featured in a meeting symposium on May 11 by Jeffrey S. Miller, M.D., Professor of Medicine, University of Minnesota and Deputy Director of the Masonic Cancer Center and scientific advisor and collaborator of the Company, and its iPSC-derived CAR T-cell product platform is expected to be highlighted during the meeting’s plenary session on May 12 by Michel Sadelain, M.D., Ph.D., Stephen and Barbara Friedman Chair and Director, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center and collaborator of the Company.

The Company also plans to host a virtual investor event on May 13 to highlight interim Phase 1 clinical data from its FT516 and FT538 programs for the treatment of relapsed / refractory acute myeloid leukemia (AML). The Phase 1 clinical trial of FT516 has enrolled the first and second dose cohorts (90 million and 300 million cells per dose, respectively), and dose escalation is ongoing in the third dose cohort (900 million cells per dose). The Phase 1 clinical trial of FT538 is ongoing in the first dose cohort (100 million cells per dose).

ASGCT Oral Presentations

Sequential CRISPR-mediated Engineering and Clonal Banking for the Generation of Multiplexed Engineered Master Pluripotent Cell Lines for the Mass Manufacture of Off-the-Shelf Immune Cells Targeting Solid Cancers
Room 9 – Advances in Ex Vivo Modified Cell Therapies; Abstract 5; May 11, 2021; 6:30pm – 6:45pm EDT
Enhanced Generation of T-cell Derived Naïve Pluripotent Cells as a Renewable Cell Source for the Mass Manufacture of Off-the-shelf CAR T-cell Therapies
Room 5 – CAR Modified Cellular Therapies; Abstract 76; May 12, 2021; 6:45pm – 7:00pm EDT
ASGCT Digital Presentations

Development and Application of a Pluripotent Stem Cell Platform to Generate Precision-Engineered Single Cell-derived Renewable Clonal Master Cell Lines for Therapeutic Use
Abstract # 793
Temporal Gene Regulation of T-cell Enhancers by Locus Targeted Engineering Enables Cytokine Autonomy and Augments Anti-tumor Efficacy of iPSC-derived Off-the-shelf CAR-T Therapy
Abstract #784
About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 350 issued patents and 150 pending patent applications.

About FT516
FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies. CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. ADCC is dependent on NK cells maintaining stable and effective expression of CD16, which has been shown to undergo considerable down-regulation in cancer patients. In addition, CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity, respectively, to the Fc domain of IgG1 antibodies. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. FT516 is being investigated in a multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-targeted monoclonal antibodies for the treatment of advanced B-cell lymphoma (NCT04023071). Additionally, FT516 is being investigated in a multi-dose Phase 1 clinical trial in combination with avelumab for the treatment of advanced solid tumor resistant to anti-PDL1 checkpoint inhibitor therapy (NCT04551885).

About FT538
FT538 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three functional components: a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; an IL-15 receptor fusion (IL-15RF) that augments NK cell activity; and the deletion of the CD38 gene (CD38KO), which promotes persistence and function in high oxidative stress environments. FT538 is designed to enhance innate immunity in cancer patients, where endogenous NK cells are typically diminished in both number and function due to prior treatment regimens and tumor suppressive mechanisms. In preclinical studies, FT538 has shown superior NK cell effector function, as compared to peripheral blood NK cells, with the potential to confer significant anti-tumor activity to patients through multiple mechanisms of action. FT538 is being investigated in a multi-dose Phase 1 clinical trial for the treatment of acute myeloid leukemia (AML) and in combination with daratumumab, a CD38-targeted monoclonal antibody therapy, for the treatment of multiple myeloma (NCT04614636).

On April 27, 2021 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that the Company has submitted its Marketing Authorization Application ("MAA") to the European Medicines Agency for omburtamab for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma (Press release, Y-mAbs Therapeutics, APR 27, 2021, View Source [SID1234578560]). Omburtamab is an investigational, monoclonal antibody that targets B7-H3 and is radiolabeled before intraventricular central nervous system ("CNS") administration. B7-H3 is an immune checkpoint molecule that is widely expressed in tumor cells of several cancer types.

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"We believe omburtamab is on track to potentially become the first EMA approved targeted therapy for pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma," said Thomas Gad, founder, Chairman and President at Y-mAbs. "With this submission to EMA, Y-mAbs is continuing to advance the omburtamab program to potentially provide access to this innovative therapy to pediatric patients globally as quickly as possible."

In addition, Y-mAbs recently concluded a Type B meeting with the U.S. Food and Drug Administration ("FDA") regarding omburtamab and the Company continues to be in close dialog with the FDA and maintains its aim of resubmitting the Biologics License Application ("BLA") to the FDA late in the second quarter or in the third quarter of 2021.

Dr. Claus Moller, the Company’s Chief Executive Officer, continued, "We are excited to submit the MAA for omburtamab, and very pleased about the progress we are making. We believe omburtamab can potentially address a significant unmet medical need for children with CNS/leptomeningeal metastasis from neuroblastoma. We also are continuing to work closely with the FDA to resubmit the omburtamab BLA."

Researchers at Memorial Sloan Kettering Cancer Center ("MSK") developed omburtamab, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interest related to the compound and Y-mAbs.

On April 27, 2021 Perrigo Company plc (NYSE; TASE: PRGO), a leading provider of Quality, Affordable Self-Care Products, reported that it will release its fiscal first quarter financial results on Tuesday, May 11, 2021 (Press release, Perrigo Company, APR 27, 2021, View Source,-2021 [SID1234578559]). The Company will also host a conference call beginning at 8:00 a.m. (EDT).

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The conference call will be available live via webcast to interested parties in the investor relations section of the Perrigo website at View Source or by phone at 888-317-6003, International 412-317-6061, and reference ID # 5211930. A taped replay of the call will be available beginning at approximately 12:00 p.m. (EDT) Tuesday, May 11, until midnight Tuesday, May 18, 2021. To listen to the replay, dial 877-344-7529, International 412-317-0088, and use access code 10155879.

On April 27, 2021 Illumina, Inc. (NASDAQ: ILMN) reported its financial results for the first quarter of fiscal year 2021 (Press release, Illumina, APR 27, 2021, View Source [SID1234578558]).

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"Illumina achieved its first billion-dollar revenue quarter in company history and delivered a very strong start to 2021, exceeding our expectations"

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First quarter results reflect record revenue:

Revenue of $1,093 million, a 27% increase compared to the prior year period
GAAP net income for the quarter of $147 million, or $1.00 per diluted share, compared to $173 million, or $1.17 per diluted share, for the prior year period
Non-GAAP net income for the quarter of $278 million, or $1.89 per diluted share, compared to $243 million, or $1.64 per diluted share, for the prior year period. Non-GAAP net income excludes acquisition-related expenses, primarily the Continuation Payments paid to GRAIL (see the "Reconciliation Between GAAP and Non-GAAP Net Income" table for a reconciliation of these GAAP and non-GAAP financial measures)
Cash flow from operations of $282 million compared to $281 million in the prior year period
Free cash flow (cash flow from operations less capital expenditures) of $240 million for the quarter compared to $241 million in the prior year period
"Illumina achieved its first billion-dollar revenue quarter in company history and delivered a very strong start to 2021, exceeding our expectations," said Francis deSouza, Chief Executive Officer. "Orders during the first quarter of 2021 reached an all-time high demonstrating strength in our core business across all regions, reflecting growth in both clinical and research customers. We are seeing tremendous progress in clinical market access and reimbursement for genomic applications increasing access to genomic testing for patients worldwide. We are proud of the significant contributions our customers, partners, and employees are making to the creation of a genomic epidemiology infrastructure to combat COVID-19, as well as monitor for future pathogen outbreaks for the benefit of global public health."

Gross margin in the first quarter of 2021 was 69.9% compared to 72.1% in the prior year period. Excluding amortization of acquired intangible assets and expenses related to COVID-19, non-GAAP gross margin was 70.5% for the first quarter of 2021 compared to 73.0% in the prior year period.

Research and development (R&D) expenses for the first quarter of 2021 were $197 million compared to $156 million in the prior year period. Non-GAAP R&D expenses as a percentage of revenue were 18.0% compared to 18.3% in the prior year period.

Selling, general and administrative (SG&A) expenses for the first quarter of 2021 were $374 million compared to $274 million in the prior year period. Excluding acquisition-related expenses and expenses and income related to COVID-19, non-GAAP SG&A expenses as a percentage of revenue were 20.4% compared to 21.1% in the prior year period.

Depreciation and amortization expenses were $48 million and capital expenditures for free cash flow purposes were $42 million during the first quarter of 2021. At the close of the quarter, the company held $4.6 billion in cash, cash equivalents and short-term investments, compared to $3.5 billion as of January 3, 2021.

Updates since our last earnings release:

Announced TSO 500 partnership with Kartos Therapeutics to advance comprehensive genomic profiling for blood cancers
Published positive economic study results in partnership with Harvard Pilgrim Health Care demonstrating the cost-effectiveness of offering non-invasive prenatal testing (NIPT) to all pregnant women
Illumina was selected among the top 100 influential global companies by Time magazine
Published our second annual Corporate Social Responsibility report that highlights our commitment to society, our employees and the environment
Completed a senior unsecured investment grade bond offering for an aggregate principal amount of $1 billion and finalized a $750 million 5-year unsecured revolving credit facility
Announced Jay Flatley will step down from the Board of Directors and John W. Thompson will be appointed as the new Chair of the Board effective May 26, 2021
Received medical device registration in Russia for NextSeq 550Dx and associated reagent kits
Financial outlook and guidance

The non-GAAP financial guidance discussed below reflects certain pro forma adjustments to assist in analyzing and assessing our core operational performance. Please see our Reconciliation of Non-GAAP Financial Guidance included in this release for a reconciliation of the GAAP and non-GAAP financial measures.

For fiscal 2021, the company expects year-over-year revenue growth in the range of 25% to 28%, and now expects GAAP earnings per diluted share of $4.72 to $4.97 and non-GAAP earnings per diluted share of $5.80 to $6.05. Except for acquisition-related expenses and bridge facility fees incurred during Q1 2021, this guidance excludes the potential impact from the pending acquisition of GRAIL, which we expect to close in the second half of 2021.

Conference call information

The conference call will begin at 2:00 pm Pacific Time (5:00 pm Eastern Time) on Tuesday, April 27, 2021. Interested parties may access the live teleconference through the Investor Info section of Illumina’s website under the "Company" tab at www.illumina.com. Alternatively, individuals can access the call by dialing 1 (866) 211-4597 or 1 (647) 689-6853 outside North America, both using conference ID 4359912.

A replay of the conference call will be posted on Illumina’s website after the event and will be available for at least 30 days following.

On April 27, 2021 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that it has received notification of formal grant of the patent titled, "Phospholipid-Ether Analogs as Cancer-Targeting Drug Vehicles" by the Eurasian, Australian and Mexican patent authorities (Press release, Cellectar Biosciences, APR 27, 2021, View Source [SID1234578557]). This patent provides composition of matter and use protection for the company’s proprietary phospholipid-ether (PLE) analogs as a targeted delivery vehicle in combination with a broad range of commonly used chemotherapeutic classes such as alkaloids, nucleoside analogs, as well as other classes of small molecule chemotherapeutic agents.

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"These granted patents further strengthen the global IP coverage for our phospholipid drug conjugate (PDC) platform that can enable selective delivery of key small molecules to cancer cells," said James Caruso, president and CEO of Cellectar. "Our PDC platform has a wide range of functionality, including the potential to improve the efficacy and tolerability of certain drugs, and is applicable to multiple classes of molecules. We look forward to continuing our development and exploring potential partnerships to leverage this impressive technology for the benefit of patients."

The cancer-targeting PLE delivery vehicle serves as the foundation for the company’s research, development and pipeline including CLR 131, our lead product candidate. CLR 131 is currently in multiple clinical studies, including a pivotal registrational study in Waldenstrom’s macroglobulinemia, a Phase 2b study in multiple myeloma, and a Phase 1 study in multiple pediatric solid tumor indications.

About Phospholipid Drug Conjugates

Cellectar’s product candidates are built upon a patented delivery platform that utilizes optimized phospholipid ether-drug conjugates (PDCs) to target cancer cells. The PDC platform selectively delivers diverse oncologic payloads to hematologic cancers and solid tumors including cancer stem cells. This selective delivery allows the payloads’ concentration within tumor cells to be increased while reducing the concentration in normal tissue, which may enhance drug potency while reducing adverse events. This platform takes advantage of a metabolic pathway utilized by all tumor cell types. Compared with other targeted delivery platforms, the PDC platform’s mechanism of entry relies on targeting a change in cancer cell membranes that occurs due to the metabolic needs of the cancer which is very different than normal tissue. In addition, PDCs can be conjugated to molecules in numerous ways, thereby increasing the types or classes of molecules that can be selectively delivered. Cellectar believes the PDC platform holds potential for the discovery and development of the next generation of cancer-targeting agents.