On April 18, 2022 Shionogi & Co., Ltd. (Head Office: Osaka, Japan; President & CEO: Isao Teshirogi, Ph.D.; hereafter "Shionogi") and NEC Corporation (Head Office: Tokyo, Japan; President & CEO: Takayuki Morita; hereafter "NEC"), reported the execution of a strategic research collaboration agreement for the development of a novel hepatitis B therapeutic vaccine (Press release, NEC, APR 18, 2022, View Source [SID1234612393]). NEC OncoImmunity (Head Office: Oslo, Norway, CEO: Richard Stratford Ph.D.), an NEC subsidiary that specializes in artificial intelligence-driven biotechnology, is also participating in this research collaboration.

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Hepatitis B is a potentially life-threatening liver disease caused by the hepatitis B virus (HBV). Chronic infection with HBV results in a high risk of death from cirrhosis and liver cancer. About 300 million people are living with chronic hepatitis B infection worldwide, and hepatitis B resulted in an estimated 820,000 deaths in 20191. Although the widespread use of hepatitis B vaccines in infants has considerably reduced the incidence of new chronic HBV infections under the age of 5, the number of new infections by other routes continues to increase2.

Interferon (IFN) and nucleotide analog therapy are currently used in the treatment of hepatitis B. However, treatment with IFN has a high frequency of side effects, and nucleotide analog therapy has a high recurrence rate if treatment is interrupted, so it is necessary to take drugs for a lifetime3. Therefore, the unmet medical need for safe and highly effective drugs that can finally achieve a complete cure for hepatitis B is high.

On April 18, 2022 The Kuraray Group reported that the Kuraray Foundation, a general incorporated foundation established to promote the Group’s social contribution activities, launched a website on April 18 to introduce said activities (Press release, Kuraray, APR 18, 2022, View Source [SID1234612378]).

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The Kuraray Foundation was established in April 2016 in order to realize the Kuraray Group’s desire to increase the scope and scale of its social contributions in line with global expansion in its business and presence.

On April 15, 2022 Brooklyn ImmunoTherapeutics, Inc. (Nasdaq:BTX) ("Brooklyn"), a biopharmaceutical company focused on exploring the role that cytokine, gene editing, and cell therapy can have in treating patients with cancer, blood disorders, and monogenic diseases, reported financial results for the fourth quarter and year ended December 31, 2021 (Press release, Brooklyn ImmunoTherapeutics, APR 15, 2022, View Source [SID1234612348]).

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Financial and corporate highlights for the quarter and year months ended December 31, 2021 and subsequently include the following:

Fourth quarter ended December 31, 2021:

Appointed Susan McClatchey as Vice President and Head of Quality
Named to both the Nasdaq Biotechnology Index and the ICE Biotechnology Index
Transitioned from the NYSE American to the Nasdaq Global Market to better align with industry peers
The period January 2021 – September 2021:

Appointed Howard J. Federoff, M.D., Ph.D., as Chief Executive Officer and President; Kevin D’Amour, Ph.D. as Chief Scientific Officer; Jay Sial as Chief Administrative Officer and Roger Sidhu, M.D. as Chief Medical Officer
Completed the acquisition of Novellus, Inc. ("Novellus") in July 2021, which developed next-generation engineered mesenchymal stem cell ("MSC") therapies using patented mRNA-based cell reprogramming and gene editing technologies licensed from Factor Bioscience ("Factor")
Raised over $52 million in net proceeds through equity line sales of common stock for general corporate purposes, including working capital to be used to enhance the development of the mRNA gene editing and cell therapies technology licensed from Factor
Published results of IRX-2 (a human-derived mixed cytokine product) monotherapy in early-stage breast cancer in Breast Cancer Research
Acquired an exclusive license for mRNA gene editing and cell therapies technology of Factor Bioscience Limited and Novellus Therapeutics
Became a public company pursuant to our merger transaction with NTN Buzztime, Inc. ("NTN")
Howard Federoff, M.D., Ph.D., Brooklyn’s President and Chief Executive Officer, commented, "2021 was a transformational year for Brooklyn ImmunoTherapeutics. I am proud to be working with our new team of experts and seasoned professionals, Roger Sidhu, M.D., Jay Sial and Kevin D’Amour, Ph.D. Together with our board of directors and scientific advisors, we are committed to our mission to discover, develop and deliver innovative products to patients who need them. We believe that we have created a leading platform company in cell, gene-editing and cytokine therapies, with a broad and deep pipeline. Our accomplishments in 2021 reflect our continued progress towards our vision of transforming patients’ lives through science.

Dr. Federoff continued: "We began 2022 with the launch of our new research and development facilities in San Diego, California. Another sign of our growth is the transfer of our common stock listing from the NYSE American to the Nasdaq Global Market in late 2021, being named to represent the biotech sector as part of the Nasdaq Biotechnology Index (Nasdaq:NBI) and being added to the ICE Biotechnology Index (NYSE:ICEBIO). We believe that our Nasdaq listing better aligns Brooklyn with industry peers, is more in line with the innovation we are pursuing and acknowledges and further validates our approach and technology."

"We believe that we are well positioned for growth. As we await the readout of our Phase 2b trial for neoadjuvant head and neck cancer with our original asset, IRX-2, during the second quarter of 2022, we continue investigator-driven trials in a number of additional cancer types and look forward to sharing these outcomes as appropriate. I want to thank the team for all of their hard work and commitment and our shareholders, partners and investors for their continued support," concluded Dr. Federoff.

Financial Results
Operating expenses for the three and twelve months ended December 31, 2021 were $9.1 million and $113.6 million, respectively, compared to $21.4 million and $26.5 million of operating expenses for the same periods in 2020.

Research and development expenses were $4.2 million and $93.2 million for the three and twelve months ended December 31, 2021, respectively, compared to $1.7 million and $4.0 million for the same periods in 2020, respectively. Research and development expenses increased during the fourth quarter primarily due to increased license fees and non-cash stock-based compensation resulting from the issuance of equity awards. Research and development expenses increased for the full year of 2021 due to license fees, non-cash stock-based compensation and $80.5 million of acquired in-process research and development expense related to the Novellus acquisition in July 2021.

General and administrative expenses were $4.4 million and $14.7 million for the three and twelve months ended December 31, 2021, respectively, compared to $0.6 million and $3.3 million during the same periods in 2020. The quarter-over-quarter and year-over-year increases in general and administrative expense were primarily related to increased legal, accounting and consulting fees, costs associated with being a publicly traded company, increased headcount, increased insurance expense and increased non-cash stock-based compensation.

The change in the fair value of contingent consideration was $0.6 million of expense for the three months ended December 31, 2021 and a credit of $0.2 million for the twelve months ended December 31, 2021 compared to expense of $19.2 million for both the three and twelve months ended December 31, 2020.

The Company recognized transaction costs of $5.8 million during the twelve months ended December 31, 2021 related to the value of shares issued to its banker for the merger transaction with NTN in March 2021, as well as a loss on the subsequent sale of the NTN assets of $9.6 million.

Net loss for the three and twelve months ended December 31, 2021 was $8.5 million and $122.3 million, respectively, compared to a net loss of $21.5 million and $26.5 million for the same period in 2020.

As of December 31, 2021, Brooklyn had approximately $17.0 million in cash, compared to $1.6 million as of December 31, 2020.

On April 15, 2022 GC Genome, a leading company in Genomic Diagnostics, reported that the company presented the deep learning algorithm that detects and classifies multi-cancer using cf-WGS (cell free DNA whole genome sequencing), summarized in a poster session held at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 in New Orleans, Louisiana (Press release, GC Genome, APR 15, 2022, View Source [SID1234612320]).

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The data from the presentation show that it detects abnormal patterns of cancer more sensitively and accurately than original liquid biopsy as it applies deep learning algorithm on whole genome sequencing data, which uncovers structural variants with distinct tumor type.

In addition, this biopsy can detect the existence of 9 major cancers (△ lung cancer △ colorectal cancer △ breast cancer △liver cancer △ pancreatic cancer △ cholangiocarcinoma △ head and neck cancer △ ovarian cancer △ esophageal cancer) through whole genome sequencing (WGS) from circulating tumor DNA (ctDNA) in the blood. Further, its ‘Multi-cancer prediction’ model can predict the tumor-derived tissues related to the 6 specific types of cancers. (△ lung cancer △ breast cancer △ liver cancer △ pancreatic cancer △ ovarian cancer △ esophageal cancer)

"We are excited to introduce the AI-based liquid biopsy with GC Genome’s original technology applied," said Eun-Hae Cho, Chief Technology Officer at GC Genome Research Center, "The data to be presented at this year’s AACR (Free AACR Whitepaper) meeting demonstrates a major step forward in our commitment to offering clinicians and patients a highly sensitive multi-cancer screening test where we believe cancer screening can save lives."

On April 15, 2022 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that the Company has voluntarily withdrawn the pending Biologics License Application (BLA)/supplemental New Drug Application (sNDA) for the combination of ublituximab and UKONIQ (umbralisib) (combination referred to as U2) for the treatment of adult patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) (Press release, TG Therapeutics, APR 15, 2022, View Source [SID1234612319]). The decision to withdraw was based on recently updated overall survival (OS) data from the UNITY-CLL Phase 3 trial that showed an increasing imbalance in OS. Additional details are included below in the section entitled "ABOUT UNITY-CLL PHASE 3 TRIAL AND THE WITHDRAWAL OF THE BLA/sNDA SUBMISSION."

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In addition, the Company announced that it has voluntarily withdrawn UKONIQ from sale for the approved indications of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based regimen and for the treatment of adult patients with follicular lymphoma (FL) who have received at least three prior systemic therapies. UKONIQ was granted accelerated approval in these indications in February 2021. The Company’s decision to withdraw UKONIQ from sale was primarily based on the withdrawal of the BLA and sNDA for U2 in CLL.

Michael S. Weiss, Chairman and Chief Executive Officer of TG Therapeutics stated, "We were very disappointed to see that the recently updated overall survival data showed an increasing survival imbalance in favor of the control arm. Accordingly, we and our advisors determined that we should withdraw the BLA/sNDA for U2 in CLL. Additionally, we made the difficult decision to withdraw UKONIQ from sale for the approved indications in MZL/FL. We want to thank the patients, families and practitioners who worked with us in our search for novel treatment options for patients with B-cell malignancies."

Mr. Weiss continued, "While we had hoped to bring U2 to patients with CLL, this will now permit us to focus our attention, passion and energy to building out our multiple sclerosis and autoimmune platform. With our ublituximab BLA pending for patients with relapsing forms of multiple sclerosis and a PDUFA goal date of September 28, 2022, we are excited about the possibility of bringing ublituximab to patients with RMS. If approved, we believe the differentiated profile of ublituximab with its one-hour infusion will be welcomed by the MS community."

ABOUT UNITY-CLL PHASE 3 TRIAL AND THE WITHDRAWAL OF THE BLA/sNDA SUBMISSION
UNITY-CLL, a global, Phase 3, randomized, controlled clinical trial, compared the U2 combination, to an active control arm of obinutuzumab plus chlorambucil in patients with both treatment-naïve and relapsed or refractory chronic lymphocytic leukemia (CLL). The trial met its primary endpoint, with U2 significantly prolonging independent review committee (IRC) assessed progression-free survival (PFS) vs. the control arm. The UNITY-CLL Phase 3 trial was conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA). Based on the results of the UNITY-CLL trial, a BLA and sNDA were submitted to the FDA for U2 to treat patients with CLL/SLL.

In November 2021, the FDA notified the Company that it planned to host an Oncologic Drug Advisory Committee (ODAC) meeting in connection with its review of the pending BLA/sNDA and to discuss the benefit risk of UKONIQ in its approved indications. While the FDA identified a number of concerns, the FDA’s desire to host an ODAC appeared to stem from an early ad hoc analysis of overall survival (OS) from the UNITY-CLL trial.

OS was designated as a secondary efficacy endpoint in the UNITY-CLL protocol but was not part of the primary analysis in accordance with the study’s statistical analysis plan agreed upon via a SPA, and therefore, was not analyzed or included in the BLA/sNDA. Additionally, the study was not powered for overall survival. As part of the ongoing review of the BLA/sNDA, the FDA requested an early analysis of OS from the UNITY-CLL trial. In a first analysis of OS using a cut-off date of September 2021, there was an imbalance in favor of the control arm (HR: 1.23). However, based on the ad hoc nature of the analysis, approximately 15% of patients had missing or outdated survival data. Further, when excluding deaths related to COVID-19, the two arms were approximately balanced (HR: 1.04). In February 2022, the Company submitted updated OS data with the same September 2021 cut-off date, but with reduced missing data and additional OS events, which showed an improvement from the previously reported OS data. Neither the original preliminary OS results nor the updated preliminary OS results were statistically significant.

Pursuant to a recent information request made by the FDA, updated OS data were collected that showed an increasing imbalance in favor of the control arm, differing from the improved results provided to the FDA in February 2022. Based on these new data, the Company decided to withdraw the pending BLA/sNDA for U2 to treat CLL/SLL and accordingly the April 22, 2022, ODAC meeting will be canceled.

In addition, based on the Company’s decision to withdraw UKONIQ from sale, we anticipate that the FDA will withdraw the accelerated approval for the product.

The FDA also has scheduled an ODAC meeting for April 21, 2022, in which it plans to discuss the appropriate approach for phosphatidylinositol-3-kinase inhibitors under development for treatment of hematologic malignancies. UKONIQ is within this class of drugs and may be discussed during this
meeting.

CONFERENCE CALL INFORMATION
The Company will host a conference call Monday, April 18, 2022, at 8:30 AM ET, to discuss the regulatory updates.

To participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Update Call. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for a period of 30 days after the call.