On May 6, 2021 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that the research collaboration by Chugai (Forerunner Pharma Research), Department of Allergy and Rheumatology at the University of Tokyo Hospital, and RIKEN on a functional genome database was published online in the U.S. scientific journal Cell on April 29, 2021 (Press release, Chugai, MAY 6, 2021, View Source [SID1234579314]).

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"Dynamic landscape of immune cell-specific gene regulation in immune-mediated diseases"

"Chugai’s innovation is generated by its own scientific and technological capabilities. For the maintenance and improvement of state-of-the-art sciences, open innovation including collaboration with academia is essential. I am very proud that one of these efforts to understand disease biology has obtained the world’s highest level of scientific appraisal through the publication in Cell," said Dr. Osamu Okuda, Chugai’s President and CEO. "TOP I 2030, our growth strategy announced in February, places open innovation as one of its key drivers. We will continue expanding our collaboration with external networks to create innovative new drugs."

This study constructed a large-scale functional genome database "ImmuNexUT" (Immune cell gene expression atlas from the University of Tokyo) that significantly outperforms previous reports in the number of cases of immune-mediated diseases, the variety of clinical cases, and the types of immune cells, and clarified the relationship between gene polymorphisms and gene expression levels in each cell type involved in the onset of various immune-mediated diseases. A total of 9,852 samples of 28 immune cells were obtained from 416 peripheral blood samples from 10 representative patients with immunological diseases and healthy individuals, and these samples were used to quantify gene expression and to analyze the association between gene expression and gene polymorphisms. As valuable data showing the genomic functions of Asians, this database is expected to be applied to genomic studies of various disease states involving immunity, leading to the elucidation of disease states and the identification of therapeutic target molecules and biomarkers for pharmaceuticals.

Chugai participated in this collaborative study with the purpose of elucidating the disease state of autoimmune and autoinflammatory diseases and creating therapies through an integrated understanding of immunology and functional genomics. In promoting research, Chugai’s research subsidiary Forerunner Pharma Research which terminated its operation in March 2021 made significant contributions to building ImmuNexUT through acquisition by utilizing its strong immunology research infrastructure with clinical samples.

Chugai, which aims at becoming a top innovator in the healthcare industry, will promote open innovation including collaboration with academia to contribute to patients around the world through innovative drugs.

On May 6, 2021 Genprex, Inc. ("Genprex" or the "Company") (Nasdaq: GNPX), a clinical-stage gene therapy company focused on developing life-changing technologies for patients with cancer and diabetes, reported that the Company and a major cancer research center in Houston, Texas, in March 2021, entered into an amendment (the "Amendment") to their May 2020 License Agreement (the "License Agreement") to grant to Genprex an exclusive worldwide license to an additional portfolio of six patents and one patent application and related technology ("Newly Licensed IP") (Press release, Genprex, MAY 6, 2021, View Source [SID1234579313]). The Newly Licensed IP includes methods for treating non-small cell lung cancer (NSCLC) by administration of a TUSC2 therapeutic in conjunction with EGFR inhibitors or other anti-cancer therapies, in patients who are predicted to be responsive to TUSC2 therapy. A TUSC2 gene-expressing plasmid is the active agent in REQORSA immunogene therapy, Genprex’s lead drug candidate.

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"We are pleased to continue optimizing and expanding our world-class intellectual property portfolio with the addition of these technologies," said Rodney Varner, President and Chief Executive Officer of Genprex. "These new technologies further add to our arsenal of combination therapies for REQORSA, and may enable us to improve patient outcomes through the advancement of multiple therapeutic approaches."

On May 6, 2021 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 operational highlights and financial results for the first quarter ended March 31, 2021 (Press release, Intellia Therapeutics, MAY 6, 2021, View Source [SID1234579269]).

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"Intellia continues to build momentum as we progress our full-spectrum pipeline towards key upcoming milestones this year. In mid-2021, we anticipate reporting initial clinical data for NTLA-2001 for ATTR, the first-ever systemically delivered CRISPR-based therapy. The company also plans on submitting first-in-human regulatory filings for NTLA-5001 for AML and NTLA-2002 for HAE, and remains on track to nominate at least one additional development candidate from our research efforts," said Intellia President and Chief Executive Officer John Leonard, M.D. "During the first quarter, we shared important advancements that expanded the breadth of our modular platform, such as introducing our proprietary base editor and establishing preclinical proof-of-concept for systemic in vivo editing in bone marrow. We believe our platform innovations will drive future pipeline growth and expand our ability to develop potentially curative treatments for patients with a range of severe diseases."

First Quarter 2021 and Recent Operational Highlights

NTLA-2001 for ATTR: NTLA-2001 is the first systemically delivered CRISPR-based therapy dosed in a patient and could potentially be a curative treatment for ATTR. By applying the Company’s in vivo lipid nanoparticle (LNP) delivery technology, NTLA-2001 offers the possibility of halting and reversing the disease with potent, lifelong transthyretin (TTR) protein reduction after a single administration. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party, and Regeneron Pharmaceuticals, Inc. (Regeneron).

The Company anticipates reporting interim clinical data from the ongoing single ascending dose portion of the Phase 1 study evaluating NTLA-2001 in adults with hereditary ATTR with polyneuropathy (hATTR-PN) at a scientific or medical meeting in mid-2021. The data are expected to characterize the emerging safety and activity profile of NTLA-2001 at the initial dose levels.
In March, Intellia announced that the European Commission (EC) granted NTLA-2001 orphan drug designation. Orphan drug designation is granted to therapies that are intended for the treatment, prevention, or diagnosis of life threatening or chronically debilitating rare diseases where there are either no treatments or no satisfactory therapeutic options in the European Union.
Intellia intends to evaluate NTLA-2001 in a broader ATTR population of both polyneuropathy and cardiomyopathy patients following its Phase 1 safety assessment and dose optimization.
NTLA-5001 for AML: NTLA-5001 is a potential best-in-class engineered T cell therapy designed to treat all genetic subtypes of AML. This investigational candidate is an autologous T cell receptor (TCR)-T cell therapy targeting the Wilms’ Tumor 1 (WT1) antigen utilizing Intellia’s proprietary cell engineering process.

Intellia expects to submit an Investigational New Drug (IND) application or equivalent regulatory application for NTLA-5001 in mid-2021. This first-in-human trial is expected to evaluate the safety and activity of NTLA-5001 in patients with persistent or recurrent AML who have previously received first-line therapies.
The Company is evaluating the potential of NTLA-5001 to treat WT1-positive solid tumors in preclinical studies.
NTLA-2002 for HAE: NTLA-2002 aims to prevent attacks for people living with HAE after treatment consisting of a single administration. Intellia is applying its modular LNP delivery system to develop NTLA-2002 to knock out the KLKB1 gene in the liver to permanently reduce plasma kallikrein activity. This approach is expected to provide continuous suppression of kallikrein activity and eliminate the significant treatment burden associated with currently available therapies for HAE patients.

The Company expects to submit an IND or equivalent regulatory application for NTLA-2002 in the second half of 2021. Intellia is leveraging insights gained from the development of NTLA-2001 to expedite clinical development of NTLA-2002. The first-in-human trial is expected to evaluate safety, tolerability and activity in patients with HAE.
In March, Intellia presented preclinical results confirming greater reductions in serum kallikrein protein levels and activity versus the current standard of care for HAE, sustained over 17 months following a single dose in an ongoing non-human primate study of its cyno-specific LNP formulation for NTLA-2002. Additionally, the Company presented data from a humanized KLKB1 mouse model of bradykinin-mediated vascular permeability, establishing that a single administration of NTLA-2002 prevented captopril-induced vascular leakage. These results, which affirm NTLA-2002’s therapeutic hypothesis of preventing HAE attacks, were presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) 2021 Annual Meeting.
Modular Platform and Pipeline Expansion: Intellia is advancing its modular platform technologies to broaden the in vivo and ex vivo applications of genome editing. This includes progressing capabilities for innovative CRISPR/Cas9-mediated targeted transgene insertion, in vivo editing in multiple tissue types and an allogeneic approach for the development of "off-the-shelf" T cell therapies. These efforts will support new therapeutic candidates for genetic diseases, requiring removal and/or restoration of a protein, and next-generation engineered cell therapies for cancers and autoimmune diseases.

Intellia remains on track to nominate at least one additional development candidate in 2021.
The Company plans to present preclinical data at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 24th Annual Meeting, taking place virtually May 11 – 14, 2021, highlighting research advancements and platform innovations.
In March, the Company presented preclinical data introducing Intellia’s novel, proprietary cytosine deaminase base editing technology. The data demonstrated the technology’s potential for enhanced cell engineering, with multiple simultaneous gene knockouts achieving >90% T cell editing efficiency and no detectable increase in translocation above background levels. These results, which expand Intellia’s modular ex vivo capabilities, were presented at the seventh Cold Spring Harbor Laboratory (CSHL) virtual scientific meeting on Nucleic Acid Therapies.
In March, the Company presented preclinical data establishing proof-of-concept for systemic, in vivo genome editing in a tissue outside the liver. Intellia’s non-viral delivery platform achieved dose-dependent, therapeutically meaningful editing of bone marrow and hematopoietic stem cells (HSCs), lasting one year following a single dose in mice. These results, which extend Intellia’s modular in vivo capabilities to treat inherited blood disorders such as sickle cell disease, were presented at the Keystone eSymposium: Precision Engineering of the Genome, Epigenome and Transcriptome.
Board of Directors:

In April, Intellia appointed Georgia Keresty, Ph.D., M.P.H., to the Company’s Board of Directors. Dr. Keresty has held key global roles in pharmaceutical research and development, operations, manufacturing and distribution, quality, compliance and regulatory affairs. Most recently, Dr. Keresty served as chief operating officer and global head, medical sciences and development operations for Takeda Research and Development, a division of Takeda Pharmaceuticals USA, Inc.
Upcoming Events

The Company will participate in the following events during the second quarter of 2021:

ASGCT 24th Annual Meeting, May 11–14, Virtual
Jefferies Healthcare Conference, June 2, Virtual
Upcoming Milestones

The Company has set forth the following for pipeline progression:

ATTR: Report initial clinical data from Phase 1 study of NTLA-2001 in mid-2021
AML: Submit an IND or IND-equivalent for NTLA-5001 in mid-2021
HAE: Submit an IND or IND-equivalent for NTLA-2002 in 2H 2021
Nominate at least one new development candidate in 2021
First Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $600.8 million as of March 31, 2021, compared to $597.4 million as of December 31, 2020. The increase was driven by $45.3 million of net equity proceeds raised from the Company’s "At the Market" (ATM) agreement, $13.3 million in proceeds from employee-based stock plans and $2.4 million from the Regeneron collaboration. These increases were offset in part by cash used to fund operations of approximately $57.6 million.
Collaboration Revenue: Collaboration revenue decreased by $6.5 million to $6.4 million during the first quarter of 2021, compared to $12.9 million during the first quarter of 2020. The decrease was primarily driven by the $5.0 million milestone payment earned from Novartis Institutes for BioMedical Research, Inc. (Novartis) for the IND submission of OTQ923 in 2020.
R&D Expenses: Research and development expenses increased by $4.6 million to $39.3 million during the first quarter of 2021, compared to $34.7 million during the first quarter of 2020. This increase was primarily driven by the advancement of our lead programs, research personnel growth to support these programs, and expansion of the development organization.
G&A Expenses: General and administrative expenses increased by $2.3 million to $13.6 million during the first quarter of 2021, compared to $11.3 million during the first quarter of 2020. This increase was primarily related to employee related expenses, including stock-based compensation of $0.9 million.
Net Loss: The Company’s net loss was $46.2 million for the first quarter of 2021, compared to $31.8 million during the first quarter of 2020.
Financial Guidance

Intellia expects that its cash, cash equivalents and marketable securities as of March 31, 2021 will enable the Company to fund its robust R&D plans, anticipated operating expenses and capital expenditure requirements at least through the next 24 months. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.

Conference Call to Discuss First Quarter Earnings

The Company will discuss these results on a conference call today, May 6, 2021, at 8 a.m. E.T.

To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call.
All participants should ask to be connected to the Intellia Therapeutics conference call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com beginning on May 6, 2021 at 12 p.m. E.T.

On May 6, 2021 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases with significant unmet needs, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Protara Therapeutics, MAY 6, 2021, View Source [SID1234579268]).

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"We remain on track to file an Investigational New Drug (IND) application and initiate our Phase 1 clinical study for TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC) by the end of the year. Patients suffering from NMIBC have limited approved treatment options, and with the current standard-of-care facing a long-term supply shortage, there is an urgent need for effective therapies for these patients," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We look forward to completing GMP scale up and comparability and then aligning with the Food and Drug Administration (FDA) on the design of a clinical study for TARA-002 in patients with Lymphatic Malformations (LMs). This new study, together with the extensive data for OK-432, the originator compound for TARA-002, should provide a robust data package for TARA-002 in this rare pediatric indication for which there is no U.S. FDA-approved therapy."

Recent Highlights and Upcoming Milestones

TARA-002 in NMIBC

Protara remains on track to submit an IND application in the second half of 2021. Subject to the successful completion of select non-clinical studies, which are expected to be completed in the first half of 2021, and FDA acceptance of the IND application, the Company plans to commence a Phase 1 study by the end of 2021 to assess the safety and tolerability of TARA-002 in patients with NMIBC, including patients with carcinoma in situ (CIS).
TARA-002 in LMs

Based on feedback from the FDA, the Company intends to complete confirmatory, large-scale, GMP manufacturing comparability in the second half of 2021 and, upon alignment with FDA on study design, subsequently initiate a clinical study in pediatric LM patients.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)

The Company is currently undertaking a prevalence study in partnership with a large home health organization in the U.S. to enhance understanding of the appropriate patient population and will use this information to define the next steps for the development program.
Corporate Update

In April 2021, the Company announced the appointment of Martín Sebastian Olivo, M.D. as Chief Medical Officer. Dr. Olivo brings to Protara more than 15 years of experience in oncology translational and clinical research and global drug development, most recently serving as Vice President, Breast Cancer Clinical Development Lead at Gilead Sciences, Inc. (formerly Immunomedics, Inc.).
First Quarter 2021 Financial Results

As of March 31, 2021, cash, cash equivalents and investments totaled $155 million.

Research and development expenses for the first quarter of 2021 increased to $7.0 million from $3.1 million during the first quarter of 2020. The increased R&D expenses were primarily due to increases in manufacturing and regulatory expenses associated with TARA-002.

General and administrative expenses for the first quarter of 2021 decreased to $6.5 million from $7.1 million for the prior year period. The decrease was primarily due to one-time expenses related to the reverse merger, which occurred in the first quarter of 2020, off-set by an increase in personnel and related costs supporting the Company’s growth.

For the first quarter of 2021, Protara reported a net loss of $13.5 million, or $1.20 per share, compared with a net loss of $10.1 million, or $1.81 per share, for the same period in 2020. Net loss for the first quarter of 2021 included approximately $2.7 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd. Protara successfully demonstrated initial manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-6, IL-8, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, and vascular endothelial growth factor (VEGF) are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the 6th most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle. The current standard of care for high-grade NMIBC includes intravesical Bacillus Calmette-Guerin (BCG).

About Lymphatic Malformations

Lymphatic malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of 3 years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride and Intestinal Failure-associated Liver Disease (IFALD)

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN) who have IFALD. Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. If approved, IV Choline Chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.

On May 6, 2021 Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, reported that it will issue financial results for its fiscal quarter ended March 31, 2021 on Thursday, May 13, 2021 (Press release, Anavex Life Sciences, MAY 6, 2021, View Source [SID1234579267]).

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Management will host a conference call on Thursday, May 13, 2021 at 4:30 pm Eastern Time to review financial results and provide an update on its clinical programs and corporate highlights. Following management’s formal remarks, there will be a question-and-answer session with equity analysts.

Conference Call / Webcast Information:

The live webcast of the conference call can be accessed online at View Source

To join the conference call, live via telephone, interested parties within the U.S. should dial, toll-free, 1 (866) 866-1333 and international callers should dial 1 (404) 260-1421. Please use confirmation number 50162864, followed by the pound sign (#).

A replay of the conference call will also be available on www.anavex.com.