On April 24, 2019 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that Fate scientists and collaborators will present data highlighting the Company’s induced pluripotent stem cell (iPSC) product platform and its iPSC-derived cell product candidates at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 22nd Annual Meeting being held from April 28 – May 2, 2019 in Washington, D.C (Press release, Fate Therapeutics, APR 24, 2019, View Source [SID1234535357]).

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"Our strong scientific presence at ASGCT (Free ASGCT Whitepaper) demonstrates the unique value of our proprietary iPSC product platform for gene editing and the development of universal, off-the-shelf, engineered cell therapies," said Bob Valamehr, Ph.D., Chief Development Officer of Fate Therapeutics. "We look forward to showcasing the critical importance of characterizing and selecting a single clone from a heterogeneous population of engineered cells, our unmatched expertise in creating large numbers of clinical-grade NK cells and T cells from clonal master engineered iPSC lines, and the breadth of our highly-differentiated off-the-shelf cancer immunotherapy pipeline."

Three oral and two poster presentations will highlight several product candidates from the Company’s universal, off-the-shelf, iPSC-derived cancer immunotherapy pipeline, including NK cell product candidates FT500 for the treatment of advanced solid tumors and FT516 for the treatment of advanced hematologic malignancies. FT500 is the first-ever iPSC-derived cell therapy to be administered to patients in the U.S., and FT516 is the first-ever engineered iPSC-derived cell therapy allowed for clinical investigation in the world. The Company’s iPSC-derived, dual-targeted CD16-CAR19 NK cell product candidate FT596 and its iPSC-derived, TCR-less, TRAC-targeted, 1XX CAR19 T-cell product candidate FT819, which is being developed under a collaboration with Memorial Sloan Kettering Cancer Center led by Michel Sadelain, M.D., Ph.D., will also be featured.

2019 ASGCT (Free ASGCT Whitepaper) Oral Presentations

Workshop – CAR T and Related Immune Effector Cell Therapies
Title: iPSC-Derived Immune Effector Cells
Presenter: Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center
Date and Time: Sunday, April 28, 2019, 3:30 PM – 6:00 PM
Location: Jefferson East
Session 132 – Induced Pluripotent Stem Cells
Title: Pluripotent Cell-Derived T and NK Cells as a Cornerstone Approach for Off-the-Shelf Cancer Immunotherapy
Presenter: Bob Valamehr, Ph.D., Chief Development Officer, Fate Therapeutics
Date and Time: Monday, April 29, 2019, 1:30 PM – 3:00 PM
Location: Monroe Room
Session 134 – NK Cells versus NKT Cells
Title: Clinical Development of NK Cell Cancer Therapy
Presenter: Sarah Cooley, M.D., Senior V.P., Clinical Translation, Fate Therapeutics
Date and Time: Monday, April 29, 2019, 1:30 PM – 3:00 PM
Location: Georgetown Room
2019 ASGCT (Free ASGCT Whitepaper) Poster Presentations

Poster 877 – iPSC Product Platform
Title: A Versatile Platform for Generation of Single-Cell Derived, Genetically Engineered, Fully Characterized Pluripotent Master Cell Lines as a Renewable Source for Off-the-Shelf Cell Therapy
First Author: Mochtar Pribadi, Ph.D., Scientist, Molecular Biology, Fate Therapeutics
Date and Time: Wednesday, May 1, 2019, 5:00 PM – 6:00 PM
Location: Columbia Hall
Poster 882 – CRISPR-based iPSC Generation
Title: A Novel Platform Combining Chemical and CRISPRa Approaches for the Generation of Human iPSCs
First Author: Jason Dinella, Ph.D., Scientist, Reprogramming Biology, Fate Therapeutics
Date and Time: Wednesday, May 1, 2019, 5:00 PM – 6:00 PM
Location: Columbia Hall
About FT500
FT500 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line. FT500 is being investigated in an open-label, repeat-dose Phase 1 clinical trial for the treatment of advanced solid tumors in up to 64 patients, both as a monotherapy and in combination with FDA-approved checkpoint inhibitor therapy. Despite the favorable response rates observed with checkpoint inhibitor therapy, the majority of patients do not respond and many responders relapse. One common mechanism of resistance to checkpoint inhibitor therapy is associated with loss-of-function mutations in genes critical for antigen presentation. A potential strategy to overcome resistance is through the administration of allogeneic NK cells, which have the inherent capability to recognize and directly kill tumor cells with these mutations.

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. CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. CD16 occurs in two variants, either with high (158V) or low (158F) affinity for 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 CD16 high-affinity variant (approximately 15% of patients) have improved clinical outcomes. In addition, the expression of CD16 can undergo considerable down-regulation in cancer patients, which significantly inhibits the immune system’s anti-tumor response. FT516 incorporates a novel CD16 Fc receptor, which has been modified to prevent its down-regulation and augment its binding to tumor-targeting antibodies for enhanced ADCC. The Company announced in February 2019 that the FDA allowed its FT516 IND application for the treatment of relapsed / refractory hematologic malignancies including in combination with certain FDA-approved monoclonal antibody therapies.

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 in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of 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 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 to treat many patients. 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 fraught with batch-to-batch and cell-to-cell variability that can affect safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 100 issued patents and 100 pending patent applications.

On April 24, 2019 Biogen Inc. (Nasdaq: BIIB) reported first quarter 2019 financial results (Press release, Biogen, APR 24, 2019, View Source [SID1234535356]).

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"Biogen delivered strong financial results in the first quarter driven by the solid operational performance of our MS, SMA, and biosimilars franchises," said Michel Vounatsos, Biogen’s chief executive officer. "However, we are deeply disappointed with the discontinuation of aducanumab for Alzheimer’s disease. We followed the science, and unfortunately the outcome was not as we had hoped. We continue to believe we can create value for patients and investors by capitalizing on opportunities in neuroscience, and we remain focused on allocating capital to the areas we believe have the highest potential return for shareholders."

Financial Results

First quarter total revenues were $3.5 billion, an 11% increase versus the first quarter of 2018.
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Multiple sclerosis (MS) revenues, including $112 million in royalties on the sales of OCREVUS, were relatively stable in the first quarter versus the prior year at $2.1 billion.
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Revenue growth was driven in part by the continued global launch of SPINRAZA, which contributed $518 million in revenues in the first quarter compared to $364 million in the first quarter of 2018.

Biosimilars revenues increased to $175 million compared to $128 million in the first quarter of 2018, driven by the launch of IMRALDITM.

Other revenues in the first quarter increased to $292 million compared to $164 million in the first quarter of 2018, primarily due to the sale of most of the remaining hemophilia inventory on hand to Bioverativ Inc.

First quarter GAAP net income and diluted earnings per share (EPS) attributable to Biogen Inc. were $1.4 billion and $7.15, respectively, compared to $1.2 billion and $5.54, respectively, in the first quarter of 2018.

First quarter Non-GAAP net income and diluted EPS attributable to Biogen Inc. were $1.4 billion and $6.98, respectively, compared to $1.3 billion and $6.05, respectively in the first quarter of 2018.

A reconciliation of GAAP to Non-GAAP quarterly financial results can be found in Table 3 at the end of this news release.

Mr. Vounatsos added, "We continued to make meaningful progress diversifying our pipeline. We are building depth in neuromuscular diseases and movement disorders, and our proposed acquisition of Nightstar Therapeutics would provide us with two potentially first-in-class mid- to late-stage clinical assets in specialty ophthalmology. By the end of 2020 we expect readouts across our clinical programs in MS, progressive supranuclear palsy, ALS, Parkinson’s disease, pain, cognitive impairment associated with schizophrenia, epilepsy, stroke, and lupus."

In the first quarter of 2019 channel inventory levels in the U.S. decreased by approximately $170 million for TECFIDERA, AVONEX, PLEGRIDY, and TYSABRI combined. This compares to an increase in inventory levels of approximately $105 million in the fourth quarter of 2018 and a decrease of approximately $140 million in the first quarter of 2018.

In the first quarter of 2019 SPINRAZA revenues comprised $223 million in sales in the U.S. and $295 million in sales outside the U.S. The number of commercial patients receiving SPINRAZA grew approximately 5% in the U.S. and approximately 24% outside the U.S. versus the fourth quarter of 2018. In the first quarter of 2019 Biogen recorded SPINRAZA revenues in over 40 countries.

R&D expense in the first quarter of 2019 included $39 million related to Biogen’s agreement with Skyhawk Therapeutics, Inc.

R&D expense in the first quarter of 2019 included approximately $45 million in net closeout costs for the Phase 3 studies of aducanumab in Alzheimer’s disease.

For the remainder of 2019 Biogen expects a reduction in operating expenses of approximately $125 million related to the discontinuation of aducanumab, with a net savings of approximately $80 million for the full year 2019.

Other Financial Highlights

In the first quarter of 2019 Biogen booked a pre-tax GAAP loss of $116 million related to its share purchase agreement with FUJIFILM Corporation (FUJIFILM) under which FUJIFILM will acquire all of the outstanding shares of Biogen’s subsidiary that owns its biologics manufacturing operations in Hillerød, Denmark.

For the first quarter of 2019 pre-tax GAAP other income was $357 million, including $376 million in net gains on investments, principally driven by an increase in the fair value of our equity investment in Ionis Pharmaceuticals, Inc. Non-GAAP other expense was $19 million.

For the first quarter of 2019 the Company’s effective GAAP tax rate was 23%, and the Company’s effective non-GAAP tax rate was 18%.

In the first quarter of 2019 Biogen repurchased approximately 2.4 million shares of the Company’s common stock for a total value of approximately $656 million. From April 1, 2019 through April 24, 2019, Biogen repurchased an additional approximately 2.1 million shares of the Company’s common stock at a cost of approximately $492 million.
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In the first quarter of 2019 Biogen’s Board of Directors authorized a program to repurchase up to $5.0 billion of the Company’s common stock. This is in addition to the approximately $1.0 billion remaining as of April 24, 2019, under the share repurchase program authorized in August 2018.
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As of March 31, 2019, Biogen had cash, cash equivalents, and marketable securities totaling approximately $5.3 billion, and approximately $5.9 billion in notes payable.

In the first quarter of 2019 the Company generated $1.5 billion in net cash flows from operations.

For the first quarter of 2019 the Company’s weighted average diluted shares were 197 million.

Recent Events

In April 2019 Biogen presented data on the rapidly progressive nature of spinal muscular atrophy (SMA) and the benefits of treatment with SPINRAZA at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida (April 13-17, 2019). Presentations included data on the severity of disease progression in adults, adolescents, and older children, as well as data from the NURTURE study highlighting the benefits of pre-symptomatic treatment and findings on the role of neurofilament as a potential biomarker for predicting motor function in SMA.

In March 2019 the first patient was dosed in the Phase 3 VALOR study of BIIB067 (tofersen), an antisense oligonucleotide for amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations. VALOR is a continuation of the Phase 1/2 single- and multiple-ascending dose study and is designed to assess the efficacy and safety of BIIB067 versus placebo. The primary endpoint of this study is an analysis based on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Score. Biogen is collaborating with regulators to further define the scope of the clinical data package required to support the registration of BIIB067.

In March 2019 Biogen and Eisai Co., Ltd. announced the decision to discontinue the global Phase 3 studies, ENGAGE and EMERGE, designed to evaluate the efficacy and safety of aducanumab in patients with mild cognitive impairment due to Alzheimer’s disease and mild Alzheimer’s disease dementia. The decision to stop the studies was based on results of a futility analysis conducted by an independent data monitoring committee, which indicated the studies were unlikely to meet their primary endpoint upon completion. The recommendation to stop the studies was not based on safety concerns. Biogen has also decided not to initiate a Phase 3 secondary prevention study to evaluate whether early use of aducanumab can prevent or delay the clinical onset of Alzheimer’s disease at this time.

In March 2019 Biogen announced that it has entered into a share purchase agreement with FUJIFILM under which FUJIFILM will acquire all of the outstanding shares of Biogen’s subsidiary that owns its biologics manufacturing operations in Hillerød, Denmark, for up to $890 million in cash, subject to working capital adjustments and other contractual terms. Following the completion of the proposed transaction, FUJIFILM would use the Hillerød facility to produce commercial products for Biogen, such as TYSABRI, as well as other third-party products. The proposed transaction remains subject to customary closing conditions, including filings and clearances under the Danish Competition Act. The closing of the proposed transaction is expected to occur in the second half of 2019, after which Biogen will operate manufacturing facilities in Research Triangle Park, North Carolina and Solothurn, Switzerland, which Biogen expects to be operational by the end of 2020.

In March 2019 Biogen announced that it had entered into an agreement to acquire Nightstar Therapeutics plc (NST), a clinical-stage gene therapy company focused on adeno-associated virus treatments for inherited retinal disorders. Under the terms of the proposed acquisition, Biogen would pay NST shareholders $25.50 in cash for each issued and outstanding NST share. This offer represents a total transaction value of approximately $800 million on a fully diluted basis, after taking into account expected transaction expenses and anticipated cash acquired at closing. NST has two potentially first-in-class mid- to late-stage clinical assets as well as preclinical programs. NST’s lead asset NSR-REP1 is in Phase 3 development for choroideremia, a rare degenerative disorder that leads

to blindness and has no approved treatment options. The proposed acquisition of NST is planned to be funded through available cash and accounted for as an acquisition of a business. The closing of the proposed acquisition remains subject to customary closing conditions, including the approval by NST shareholders and the issuance of an order by the U.K. Court. Biogen expects to complete the acquisition by mid-year 2019.

In February 2019 Biogen announced that SPINRAZA was approved by the China National Medical Products Association for the treatment of 5q SMA, expanding the Company’s presence in China. Approximately 95% of all SMA cases are 5q SMA, making it the most common form of the disease. SPINRAZA is the first approved treatment in China for SMA. SPINRAZA will initially be available in China for self-paying patients as the Company works to secure provincial and national reimbursement.

In February 2019 Alkermes plc and Biogen announced that the U.S. Food and Drug Administration (FDA) had accepted for review the New Drug Application (NDA) for diroximel fumarate (BIIB098), a novel oral fumarate in development for the treatment of relapsing forms of MS. If approved, Biogen intends to market diroximel fumarate under the brand name VUMERITY, which has been conditionally accepted by the FDA and will be confirmed upon approval. The NDA has been assigned a PDUFA (Prescription Drug User Fee Act) target action date in the fourth quarter of 2019.

Conference Call and Webcast
The Company’s earnings conference call for the first quarter will be broadcast via the internet at 8:00 a.m. ET on April 24, 2019, and will be accessible through the Investors section of Biogen’s website, www.biogen.com. Supplemental information in the form of a slide presentation is also accessible at the same location on the internet and will be subsequently available on the website for at least one month.

On April 24, 2019 Bausch Health Companies Inc. (NYSE/TSX: BHC) reported that the Company will participate in four investor conferences in May (Press release, Valeant, APR 24, 2019, View Source [SID1234535355]).

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Paul S. Herendeen, executive vice president and chief financial officer, Arthur J. Shannon, senior vice president and head of Investor Relations and Communications, and William Woodfield, vice president and treasurer, are scheduled to participate at the Goldman Sachs Leveraged Finance Conference in Rancho Palos Verdes, CA on May 7, 2019 at 10:40 a.m. PDT (1:40 p.m. EDT).

Joseph C. Papa, chairman and chief executive officer, and Sam Eldessouky, senior vice president and corporate controller, are scheduled to participate in investor meetings at the SunTrust Robinson Humphrey Life Sciences Summit in New York, NY on May 8, 2019.

Mr. Papa, Mr. Eldessouky and Mr. Shannon are scheduled to participate at the Bank of America Merrill Lynch Global Healthcare Conference in Las Vegas, NV on May 15, 2019 at 11:20 a.m. PDT (2:20 p.m. EDT).

Mr. Herendeen and Mr. Shannon are scheduled to participate at the RBC Capital Markets Healthcare Conference in New York, NY on May 22, 2019 at 10:00 a.m. EDT.

Live webcasts and audio archives of the presentations will be available on the Investor Relations page of the Bausch Health Companies Inc. web site at: View Source

On April 24, 2019 Syros Pharmaceuticals presented the corporate presentation (Presentation, Syros Pharmaceuticals, APR 24, 2019, View Source [SID1234535354]).

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On April 24, 2019 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported its financial results for the first quarter of the fiscal year ended December 31, 2019 (Press release, Chugai, APR 24, 2019, View Source [SID1234535351]).

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Chugai reported record high revenues and profits for the first quarter of 2019 driven by contribution from new products including the hemophilia A treatment Hemlibra and the immune checkpoint inhibitor Tecentriq, and strong growth of mainstay products in Japan and overseas. Revenues increased by a little less than 5% as the double-digit sales growth driven mainly by the contribution of new products and strong exports outweighed the remarkable decrease in royalties and other operating income in the absence of one-time income from the transfer of long-term listed products reported last year. Operating profit achieved a double-digit increase due to a better cost to sales ratio as the proportion of in-house products increased in the total product mix.

Domestic sales increased to ¥99.3 billion (+6.9%). Contribution from new products including the hemophilia A treatment Hemlibra created by Chugai and the immune checkpoint inhibitor Tecentriq, and the strong growth of mainstay products chiefly in bone and joint diseases area have exceeded the negative impact from the NHI drug price revisions last year and generic competition.
Oncology: Sales increased driven by the contribution from new products such as Tecentriq and Gazayva as well as solid growth of Perjeta and Alecensa, despite the negative impact from last year’s NHI drug price revisions and generic competition facing Rituxan.
Bone and joint diseases: Sales recorded a double-digit growth due to strong sales of mainstay products such as the anti-rheumatic agent Actemra and the osteoporosis agent Edirol.
Renal diseases: Sales were almost flat while sales of the renal anemia agent Mircera and the secondary hyperparathyroidism treatment Oxarol approximated the previous year’s level.
Others: Sales increased primarily due to favorable market penetration of new products such as the hemophilia A treatment Hemlibra, despite the negative impact from the transfer of long-term listed products last year.
Overseas sales increased to ¥38.4 billion (+20.4%) due to increase in exports of Alecensa and Actemra to Roche.
Royalties and other operating income decreased to ¥16.6 billion (-26.9%) due to a decrease in other operating income in the absence of one-time income from the transfer of long-term listed products reported last year, despite royalties and profit sharing income related to Hemlibra increased.
Billion JPY Q1 2019 Q1 2018 % change
Sales 137.7 124.7 +10.4%
Domestic sales 99.3 92.9 +6.9%
Oncology 52.0 48.6 +7.0%
Bone and joint diseases 24.2 21.6 +12.0%
Renal diseases 7.9 8.0 -1.3%
Others 15.2 14.6 +4.1%
Overseas sales 38.4 31.9 +20.4%
[Core operating profit]
Core gross profit increased to ¥90.6 billion (+8.0%) due to sales growth and the better cost to sales with a higher proportion of in-house products in the total product mix, despite the remarkable decrease in royalties and other operating income.
Core operating expenses grew by a low single-digit percentage to ¥42.7 billion (+3.9%), more modestly than the growth in Core gross profit. As a result, Core operating profit totaled ¥47.9 billion (+11.9%).