On May 12, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported that it is presenting three oral presentations and two poster presentations at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), taking place virtually from May 12-15, 2020 (Press release, Intellia Therapeutics, MAY 12, 2020, View Source [SID1234557567]). Intellia researchers are presenting new data in support of NTLA-5001, the company’s engineered cell therapy candidate for the treatment of acute myeloid leukemia (AML). Intellia is also providing an update on NTLA-2002, its newest development candidate for the treatment of hereditary angioedema (HAE).

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"At Intellia, we are applying our CRISPR/Cas9 technology to develop new processes that can produce enhanced engineered cell therapies to treat severe cancers, such as AML, that traditional approaches cannot address. Our proprietary platform provides a powerful tool to generate more potent TCR-directed cells, that can treat blood cancers initially and potentially solid tumors. The data being presented today validate Intellia’s approach of reducing AML tumor cell blasts, and our plans to enter the clinic with NTLA-5001 next year," said Intellia President and CEO John Leonard, M.D. "We are also pleased to present data that support our recently announced HAE development candidate, NTLA-2002, Intellia’s second systemic therapy employing our in vivo knockout approach and modular delivery platform."

Data Presentations on Intellia’s First Engineered Cell Therapy Development Candidate, NTLA-5001 for the Treatment of AML, and Proprietary Cell Engineering Process

NTLA-5001 is Intellia’s first engineered T cell receptor (TCR) T cell therapy development candidate, which targets the Wilms’ Tumor 1 (WT1) intracellular antigen for the treatment of AML. NTLA-5001 is being developed in collaboration with Chiara Bonini’s team at IRCCS Ospedale San Raffaele to treat AML patients regardless of the genetic subtype of a patient’s leukemia. AML is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment and is the most common type of acute leukemia in adults (Source: NIH SEER Cancer Stat Facts: Leukemia – AML).

Intellia’s proprietary process is a significant improvement over standard engineering processes commonly used to introduce nucleic acids into cells. Intellia’s process enabled multiple gene edits using CRISPR/Cas9, while maintaining cell products with high expansion potential and minimal undesirable chromosomal translocations. CRISPR/Cas9 was used to insert a WT1-directed TCR in locus, while eliminating the expression of the endogenous TCRs, with the goal of producing homogeneous T cell therapies like NTLA-5001.

Intellia’s novel approach with NTLA-5001 can overcome the challenges of standard T cell therapy, including risks of reduced specificity associated with mixed expression and mispairing of endogenous and transgenic TCRs (tgTCRs); graph-versus-host disease (GvHD) risks, which could lead to an attack on the patient’s healthy cells; and reduced efficacy tied to lower tgTCR expression per T cell. Intellia’s unprecedented process is expected to streamline cell engineering and manufacturing, yielding a homogenous product comprising WT1-targeted T cells with high anti-tumor activity. Data highlights from today’s presentation include the following:

Developed a sequential genome editing process in primary human T cells that lead to the knockout of three genes with up to >98% efficiency and no detectable target-to-target translocations. Applied sequential editing approach to achieve knockout of the endogenous TCR with up to >99% efficiency, along with insertion of the tgTCR targeting WT1 into 50-70% of the cells.
Improved T cell viability post-editing that resulted in a significant increase in T cell expansion relative to cells engineered by standard methods, and which is expected to shorten the time required for T cell manufacturing and to increase the yield of therapeutic cells.
Increased proportion of cells having a desirable early stem cell memory phenotype with improved reactivity against WT1-expressing tumor cell lines and higher long-term proliferative capacity, which may be associated with better persistence in patients.
Intellia’s cell engineering efforts are focused on its initial clinical investigation of NLTA-5001 on AML, while continuing preclinical studies exploring the potential for targeting WT1 in solid tumors. The company confirmed plans last week to submit an IND or IND-equivalent for NTLA-5001 for the treatment of AML in the first half of 2021.

The presentation titled, "Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering," will be made today by Aaron Prodeus, Ph.D., senior scientist, Cell Therapy, and can be found here, on the Scientific Publications & Presentations page of Intellia’s website. These data were a follow-on to the study presented at Keystone Symposia’s Engineering the Genome Conference from this past February.

In Vivo Data Supports Intellia’s Novel TCR Candidate

A second presentation on engineered cell therapy progress, in collaboration with IRCCS Ospedale San Raffaele, showed in vivo data demonstrating the potential of TCR-edited T cells to effectively target WT1 tumor cells in AML. In addition to the previously disclosed results of effective in vitro recognition of primary AML tumor cells by edited WT1-specific cytotoxic T cells (CD8 T cells), new data indicate that the selected TCR also enables T helper cells (CD4 T cells) to react to WT1-expressing tumor cells, providing cytokine support. This distinguishes Intellia’s TCR from other therapeutic TCR candidates, which either exclusively activate toxic CD8 T cells or require the co-transfection of CD8 into CD4 T cells to render them functional.

Using a mouse model carrying disseminated human primary AML, researchers observed a significant therapeutic effect, including decreased AML tumor burden. In addition, no signs of GvHD were observed in mice treated with the WT1-specific T cells. The data show that tgTCR-engineered cells have targeted anti-cancer activity in a challenging model of systemic AML, demonstrating the therapeutic potential of Intellia’s engineered TCR T cell approach.

The presentation titled, "Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia," will be given today by Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy. Notably, ASGCT (Free ASGCT Whitepaper) meeting organizers selected this presentation as one of six to receive the ASGCT (Free ASGCT Whitepaper) Excellence in Research Award this year.

Continued Progress on Intellia’s Second In Vivo Development Candidate, NTLA-2002 for the Treatment of HAE

Intellia is presenting development data updates on its potential HAE therapy, NTLA-2002, which utilizes the company’s systemic in vivo knockout approach, including its proprietary lipid nanoparticle (LNP) system. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. NTLA-2002 aims to prevent unregulated production of bradykinin by knocking out the prekallikrein B1 (KLKB1) gene through a single course of treatment to ameliorate the frequency and intensity of these swelling attacks.

The KLKB1 gene knockout in an ongoing non-human primate (NHP) study resulted in a sustained 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates (Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least six months, demonstrating the same high level of efficacy and durability seen in earlier rodent studies.

The short talk titled, "CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema," will be given by Jessica Seitzer, director, Genomics, Intellia on Fri., May 15, 2020, when it will be made available here, on the Scientific Publications & Presentations page of Intellia’s website. The presented data include results from ongoing collaborations with researchers at Regeneron, and the program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party. Intellia expects to submit an IND or IND-equivalent to initiate a Phase 1 trial for NTLA-2002 in the second half of 2021.

On May 12, 2020 VBL Therapeutics (Nasdaq: VBLT), reported that it has closed the previously announced registered direct offering of 6,349,208 ordinary shares of the Company, at a purchase price of $1.575 per share, in a registered direct offering priced at-the-market under Nasdaq rules (Press release, VBL Therapeutics, MAY 12, 2020, View Source [SID1234557562]). VBL has also issued to the investors, in a concurrent private placement, unregistered warrants to purchase up to an aggregate of 6,349,208 of VBL’s ordinary shares. The warrants have an exercise price of $1.45 per ordinary share, are immediately exercisable and will expire on November 11, 2020. The registered direct offering closed on May 11, 2020.

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H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.

The gross proceeds from this offering were approximately $10.0 million, before deducting the placement agent’s fees and other estimated offering expenses. The Company intends to use the net proceeds from this offering for working capital and general corporate purposes.

The ordinary shares (but not the warrants or the ordinary shares underlying the warrants) were offered by VBL pursuant to a "shelf" registration statement on Form F-3 (File No. 333-222138) previously filed with the Securities and Exchange Commission (the "SEC") on December 18, 2017 and declared effective by the SEC on January 4, 2018. The offering of the ordinary shares was made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the ordinary shares offered have been filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (646) 975-6996 or e-mail at [email protected].

The warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder and, along with the ordinary shares underlying the warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the warrants and underlying ordinary shares may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws.

$8.1 Million Registered Direct Offering to Key Current Shareholders

VBL also announced today that it has entered into definitive agreements with two of the Company’s largest current shareholders (the "Second Offering") for the purchase and sale of 5,142,857 ordinary shares of the Company, at a purchase price of $1.575 per share, in a registered direct offering priced at-the-market under Nasdaq rules. VBL has also agreed to issue to the investors, in a concurrent private placement, unregistered warrants to purchase up to an aggregate of 5,142,857 of VBL’s ordinary shares. The warrants have an exercise price of $1.45 per ordinary share, will be immediately exercisable and will expire on November 11, 2021.

This Second Offering enables the participating shareholders to maintain, or increase, their relative holdings at the Company, under the same terms of the offering that was concluded on May 11, 2020.

The Second Offering is expected to close on or about May 12, 2020, subject to the satisfaction of customary closing conditions.

H.C. Wainwright & Co. is acting as the exclusive placement agent for the Second Offering.

The gross proceeds from the Second Offering are expected to be approximately $8.1 million, before deducting the placement agent’s fees and other estimated offering expenses. The Company intends to use the net proceeds from this offering for working capital and general corporate purposes.

The ordinary shares (but not the warrants or the ordinary shares underlying the warrants) to be issued in the Second Offering are being offered by VBL pursuant to a "shelf" registration statement on Form F-3 (File No. 333-222138) previously filed with the Securities and Exchange Commission (the "SEC") on December 18, 2017 and declared effective by the SEC on January 4, 2018. The Second Offering of the ordinary shares will be made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the ordinary shares being offered in the Second Offering will be filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained, when available, on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (646) 975-6996 or e-mail at [email protected].

The warrants described above are being offered in the Second Offering in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder and, along with the ordinary shares underlying the warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the warrants and underlying ordinary shares may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On May 12, 2020 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported financial results and corporate highlights for the first quarter 2020, as well as anticipated corporate milestones for 2020 (Press release, Surface Oncology, MAY 12, 2020, View Source [SID1234557561]).

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"I am pleased to report that Surface has made substantial clinical progress to date in 2020; achieving important milestones with both of our highly differentiated lead programs, SRF617 and SRF388, while rapidly working through a safe transition to a largely virtual operation due to the current pandemic," said Jeff Goater, chief executive officer. "Over the past few months, we announced the dosing of the first patients in the Phase 1 dose ascending trials for both of our lead product programs. In partnership with our clinical sites, we are following the latest FDA guidance regarding safe conduct of clinical trials during this time. We look forward to providing our initial clinical update for both programs before the end of this year."

Recent Corporate Highlights:

In March, initiated a Phase 1/1b clinical trial of SRF617, which targets CD39, an important component of the adenosine axis. This trial is designed to provide rapid evaluation of SRF617 via multiple arms, including as a monotherapy and in combination with both chemotherapy and other immuno-oncology agents

In April, initiated a Phase 1 clinical trial of SRF388, which targets the immunosuppressive cytokine IL-27. Surface Oncology is the first company to advance an IL-27 targeted therapy into clinical development. The development of SRF388 is informed by a compelling translational hypothesis in which hepatocellular and renal cell carcinoma are prioritized, both of which are characterized by high levels of circulating EBI3, a subunit of IL-27

Disclosed our development candidate SRF114, a monoclonal antibody targeting the chemokine receptor CCR8. SRF114 is a highly specific antibody that is designed to deplete immuno-suppressive cells present in the tumor microenvironment

Entered into a clinical collaboration with Arcus Biosciences (NYSE: RCUS) in January 2020, to evaluate SRF617 in combination with AB928 (a dual A2a/A2b adenosine receptor antagonist) in clinical trials

Continued progression of the ongoing Phase 1/1b trial of NZV930 (targeting CD73) by Surface Oncology’s partner Novartis

Promoted Lisa McGrath to senior vice president, human resources

Selected Anticipated 2020 Corporate Milestones:

Anticipated preclinical data presentations at multiple key medical and scientific conferences throughout 2020, including the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Annual Meeting in May and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II in June

Anticipated initial clinical updates for both SRF617 and SRF388 by the end of 2020

Financial Results:

As of March 31, 2020, cash, cash equivalents and marketable securities were $90.1 million, compared to $105.2 million on December 31, 2019.

Revenue recognized in the three months ended March 31, 2020 was $38.6 million, compared to $14.4 million for the same period in 2019. The increase was a result of the expiration of the final Novartis option purchase period in January 2020 and the corresponding recognition of the remaining deferred revenue under the collaboration.

Research and development (R&D) expenses were $11.3 million for the three months ended March 31, 2020, compared to $14.3 million for the same period in 2019. This decrease was primarily driven by a reduction in expenses associated with contract manufacturing and other IND enabling activities, as a result of the SRF617 and SRF388 IND filings in 2019. R&D expenses included $0.7 million in stock-based compensation expense for the three months ended March 31, 2020.

General and administrative (G&A) expenses were $4.8 million for the three months ended March 31, 2020, compared to $5.1 million for the same period in 2019. This decrease was primarily due to decreased personnel costs and professional fees. G&A expenses included $1.2 million in stock-based compensation expense for the three months ended March 31, 2020.

For the three months ended March 31, 2020, net income was $22.6 million, or basic net income per share attributable to common stockholders of $0.81, and diluted net income per share attributable to common stockholders of $0.74. Net loss was $4.2 million for the same period in 2019, or basic and diluted net loss per share attributable to common stockholders of $0.15.

Financial Outlook:

Surface Oncology continues to project that current cash and cash equivalents are sufficient to fund the Company into 2022. Anticipated milestones under the NZV930 collaboration with Novartis and additional capital potentially available under the K2 HealthVentures debt financing, in aggregate, would extend Surface Oncology’s cash runway into the second half of 2022.

On May 12, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an innovation-driven pharmaceutical company, reported that Michael Kauffman, MD, PhD, Chief Executive Officer, will participate in a fireside chat at the 2020 RBC Capital Markets Global Healthcare Virtual Conference on Tuesday, May 19, 2020 at 8:35 a.m. ET (Press release, Karyopharm, MAY 12, 2020, View Source [SID1234557560]).

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A live webcast of the fireside chat can be accessed on the "Events & Presentations" in the Investor section of the Company’s website, View Source A replay of the webcast will be archived on the Company’s website for 90 days following the fireside chat.

On May 12, 2020 Sysmex Corporation (HQ: Kobe, Japan; Chairman and CEO: Hisashi Ietsugu) reported certain
differences between its financial forecast on November 6, 2019, for the fiscal year ended March 31, 2020 (April 1, 2019, to March 31, 2020) and the actual results announced today (Press release, Sysmex, MAY 12, 2020, View Source [SID1234557556]). Furthermore, at a meeting of the Managing Board on May 12, 2020, Sysmex resolved to award dividends from surplus as described below, with a record date of March 31, 2020.

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1. Change from Financial Forecasts

(1) Consolidated Financial Results for Fiscal Year from April 1, 2019, to March 31, 2020

(2) Reason

In addition to the impact of the spreading COVID-19 pandemic, major reasons for the change in consolidated net sales were lower-than-expected sales in the hemostasis and urinalysis fields in the United States and sales to distributors in Latin America and the EMEA region, which were lower than forecast. Despite efforts to reduce selling, general and administrative expenses, operating profit, profit before tax and profit attributable to owners of the parent were also below forecast, due to lower sales and a foreign exchange valuation loss.

2. Dividend from Surplus

(2) Reason

In terms of returns to shareholders, we intend to provide a stable dividend on a continuous basis and aim for a consolidated payout ratio of 30% under our basic policy of sharing the successes of our operations in line with business performance. In accordance with this policy, we have set the ordinary year-end dividend for the fiscal year ended March 31, 2020, at ¥36 per share. Accordingly, annual total dividends will be ¥72 and the consolidated payout ratio will be 43.1%. This amounts to an increase of ¥2 in the total dividend for the year, from ¥70 in the fiscal year ended March 31, 2019.