Crescendo Biologics Announces Humabody® Evaluation in CAR-T by Takeda

On January 3, 2019 Crescendo Biologics Ltd (Crescendo), the drug developer of novel, targeted T-cell enhancing therapeutics, reported that Takeda Pharmaceutical Company Limited (Takeda) is planning to evaluate the application of recently licensed Humabodies from Crescendo for the development of novel CAR-T therapeutics (Press release, Crescendo Biologics, JAN 3, 2019, View Source [SID1234553819]).

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This research will investigate the unique properties of the Humabody VHs for tumour targeting of CAR-Ts. Unlike single-chain variable fragments (scFvs), Humabody VHs do not require a light chain for high specificity and affinity, and can be easily configured for multi-specific target binding. Takeda’s decision to license these Humabodies provides an opportunity to evaluate a large number of different VHs directed to the same target, in order to identify a format that delivers both enhanced safety and functionality.

Crescendo’s global, strategic, multi-target collaboration and license agreement with Takeda was first announced in October 2016. Under this agreement, Crescendo’s proprietary transgenic platform and engineering expertise is being used to identify and configure Humabody-based therapeutics against certain targets selected by Takeda.

Dr Peter Pack, CEO of Crescendo, commented:
"We’re pleased to see the research Takeda is undertaking with our Humabody technology. Their desire to explore the Humabody technology in a CAR-T setting presents an exciting opportunity to evaluate whether Humabodies can address the issues that exist with other CAR-T targeting approaches."

AbbVie and Tizona Therapeutics Announce Strategic Collaboration to Develop First-in-Class Immunotherapy for Cancer Targeting CD39

On January 3, 2019 AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, and Tizona Therapeutics, Inc. ("Tizona"), a privately held immunotherapy company, reported that they have entered into a global, strategic collaboration to develop and commercialize CD39-targeted therapeutics, including TTX-030, a first-in-class antibody for the treatment of cancer (Press release, AbbVie, JAN 3, 2019, View Source [SID1234532450]).

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The ATP-adenosine axis has recently emerged as a key immune regulatory switch in the tumor microenvironment (TME) by controlling the inflammatory and suppressive activities of immune cells. CD39 is the enzyme responsible for the initial steps in the conversion of immune stimulatory extracellular ATP to immune suppressive adenosine in the TME. Inhibition of CD39 with TTX-030 represents a novel and differentiated approach to targeting this pathway.

"Immuno-oncology is one of AbbVie’s key focus areas in our mission to discover and develop medicines that drive transformational improvements in cancer treatment," said Mo Trikha, Ph.D., Vice President, Head of Oncology Early Development, AbbVie. "Exploring the tumor microenvironment as a source of targets that can be modulated to inhibit cancer growth holds tremendous promise. The Tizona team has generated compelling preclinical data for their TTX-030 program, and we look forward to a productive collaboration focused on rapidly advancing this novel first-in-class antibody."

"Tumors employ various strategies to create a tolerogenic microenvironment, which reduces the immune system’s ability to detect and fight cancer," said Courtney Beers, Ph.D., Vice President, Immunology, Tizona. "Preclinical research shows that inhibiting CD39 may hold the key to restoring and bolstering immune responses against tumors. In AbbVie, we have a partner who shares our passion for science and commitment to delivering breakthrough innovation to patients with cancer. We look forward to advancing this exciting program."

Under the terms of the agreement, Tizona has received an upfront payment of $105 million for the exclusive option to license the CD39 program including TTX-030. In addition, AbbVie has made an equity investment in Tizona. Tizona will lead clinical development through completion of Phase 1b studies, after which AbbVie has an exclusive option to lead global development and commercial activities. Tizona retains an option to co-develop and co-promote in the United States and is eligible for success-based development and commercial milestones and tiered royalties on net sales.

An investigational new drug application for TTX-030 has been accepted by the US Food and Drug Administration.

TTX-030, the Adenosine Axis, and the Tumor Microenvironment

TTX-030 is a monoclonal antibody that inhibits the activity of CD39, a cell surface enzyme upregulated on tumors, exhausted T cells, as well as many suppressive cell types as an immune evasion strategy. It catalyzes the conversion of ATP to AMP, the first step in the generation of adenosine. By blocking the action of CD39, TTX-030 prevents the formation of immune suppressive extracellular adenosine, which would otherwise inhibit effector cells in the tumor microenvironment (TME). In addition to preventing the formation of suppressive adenosine, TTX-030 prevents the degradation of ATP, preserving its ability to stimulate dendritic and myeloid-derived cells responsible for innate immunity and immune cell priming necessary for adaptive immunity.

Ohr Pharmaceutical, Inc. Announces Merger Agreement with NeuBase Therapeutics, Inc.

On January 3, 2019 Ohr Pharmaceutical, Inc. ("Ohr") (Nasdaq: OHRP) reported that it has entered into a definitive merger agreement with NeuBase Therapeutics, Inc. ("NeuBase"), under which the stockholders of NeuBase would become the majority holders of the combined company (Press release, Ohr Pharmaceutical, JAN 3, 2019, View Source [SID1234556172]). The proposed merger will create a public company focused on advancing NeuBase’s peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) technology platform for the development of therapies to address severe and currently untreatable diseases caused by genetic mutations.

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Upon closing of the transaction, the combined company will change its name to "NeuBase Therapeutics, Inc." and propose its trading symbol on the NASDAQ be changed to "NBSE". The executive team of NeuBase will serve as the executive team of the combined company, led by Dietrich A. Stephan, Ph.D. as Chief Executive Officer.

"We are excited to enter into a definitive merger agreement with NeuBase, a company with a powerful technology and pipeline that has the potential to address multiple unmet medical needs across a range of serious genetic diseases," said Jason Slakter, M.D., Chief Executive Officer of Ohr Pharmaceutical. "Following a comprehensive review of strategic alternatives, Ohr’s Board of Directors concluded that the proposed transaction with NeuBase is in the best interest of our stockholders. The proposed merger will provide an opportunity to create value as an innovative, science-driven company with a proprietary technology platform utilizing advanced gene silencing techniques. We intend to hold a special meeting of Ohr shareholders in the first half of 2019 to vote on this merger."

"The proposed merger with Ohr signals the next stage of growth for NeuBase," added Dr. Dietrich Stephan, Chief Executive Officer of NeuBase Therapeutics. "The company’s new therapeutic modality has the potential to address a wide range of germline and somatic diseases caused by inappropriate expression and change-of-function mutations of genes. Our technology has significant potential advantages over currently available antisense and small molecule approaches to gene silencing, including high selectivity for targets, cell membrane and blood brain barrier permeability, early data indicating no immune response and a low cost of goods. These characteristics are essential for scalability in addressing a wide range of genetic diseases, including cancer. We are initially developing this exciting platform for RNA gene silencing in Huntington’s disease and myotonic dystrophy, with additional future, high value RNA silencing indications."

NeuBase’s modular PATrOL technology platform is being developed to treat a multitude of rare genetic diseases. The systemically-deliverable PATrOL therapies have the potential to improve upon current gene silencing treatments by combining the advantages of synthetic small molecule approaches with the precision of antisense technologies. NeuBase’s development is currently focused on severe neurological disorders such as Huntington’s disease and myotonic dystrophy, where blood-brain barrier penetration and broad tissue distribution are critical. In some cases, such as Huntington’s disease, systemic administration may ameliorate both CNS and non-CNS pathology, a benefit that current intrathecally administered therapies cannot achieve.

About the transaction
On a pro forma basis and based upon the number of shares of Ohr common stock to be issued in the merger, current Ohr stockholders will own approximately 20% of the combined company and NeuBase stockholders will own approximately 80% of the combined company, after accounting for the additional NeuBase financing transaction. The actual allocation will be subject to adjustment based on Ohr’s and NeuBase’s cash balance at the time of closing and the amount of the additional financing consummated by NeuBase at or before the closing of the merger. Certain members and affiliates of the board of directors and management of Ohr and Neubase have indicated an intent to invest in the additional NeuBase financing transaction.

The proposed transaction has been approved by the board of directors of both companies. The merger is subject to the approval of Ohr shareholders at a special meeting of shareholders, which is expected to occur in the first half of 2019, along with the satisfaction or waiver of other customary conditions.

This communication does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.

Roth Capital Partners, LLC is acting as financial advisor to Ohr for the transaction and Troutman Sanders LLP is serving as legal counsel to Ohr. Allele Capital Partners, LLC at Tribal Capital Markets, LLC is acting as financial advisor and Paul Hastings LLP is serving as legal counsel to NeuBase.

Additional Information about the Merger and Where to Find It
In connection with the Merger, the Company intends to file relevant materials with the Securities and Exchange Commission (the "SEC"), including a registration statement on Form S-4 that will contain a prospectus, joint proxy and information statement. Investors and security holders of the Company and NeuBase are urged to read these materials when they become available because they will contain important information about the Company, NeuBase and the Merger. The joint proxy statement, information statement, prospectus, and other relevant materials (when they become available), and any other documents filed by the Company with the SEC, may be obtained free of charge at the SEC web site at www.sec.gov. In addition, investors and security holders may obtain free copies of the documents filed with the SEC by the Company by directing a written request to: Ohr Pharmaceutical, Inc., 800 Third Avenue, 11th Floor, New York, NY, Attention: Corporate Secretary. Investors and security holders are urged to read the joint proxy statement, prospectus and the other relevant materials when they become available before making any voting or investment decision with respect to the Merger.

This communication shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offering of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended.

Participants in the Solicitation
The Company and its directors and executive officers and NeuBase and its directors and executive officers may be deemed to be participants in the solicitation of proxies from the stockholders of the Company in connection with the proposed transaction. Information regarding the special interests of these directors and executive officers in the merger will be included in the joint proxy statement/prospectus referred to above. Additional information regarding the directors and executive officers of the Company is also included in the Company’s Annual Report on Form 10-K for the year ended September 30, 2018 and the proxy statement for the Company’s 2018 Annual Meeting of Stockholders. These documents are available free of charge at the SEC web site (www.sec.gov) and from the Company, Attn: Corporate Secretary, at the address described above.

Ohr Pharmaceutical financial results for the fiscal year ended September 30, 2018:

For the fiscal year ended September 30, 2018, the Company reported a net loss of approximately $13.2 million, or ($0.23) per share, compared to a net loss of approximately $23.8 million, or ($0.53) per share in the fiscal year ended September 30, 2017.
For the fiscal year ended September 30, 2018, total operating expenses were approximately $13.9 million, consisting of $3.6 million in general and administrative expenses, $4.3 million of research and development expenses, $1.1 million in depreciation and amortization, $0.7 million in loss on impairment of goodwill, $5.3 million in loss on impairment of intangible assets, and $1.2 million in gain on settlement of liabilities. This compares to total operating expenses of $23.8 million in the fiscal year ended September 30, 2017, comprised of approximately $5.3 million in general and administrative expenses, $17.4 million in research and development expenses, $1.2 million in depreciation and amortization, and $0.1 million in gain on settlement of liabilities.
At September 30, 2018, the Company had cash and cash equivalents of approximately $3.8 million, compared to cash and equivalents of approximately $12.8 million at September 30, 2017.

Lilly to Participate in J.P. Morgan Healthcare Conference

On January 3, 2019 Eli Lilly and Company (NYSE: LLY) reported that it will participate in the 37th Annual J.P. Morgan Healthcare Conference on Tuesday, January 8, 2019 (Press release, Eli Lilly, JAN 3, 2019, View Source [SID1234532392]). David A. Ricks, Lilly’s chairman and chief executive officer, will participate in a fireside chat from 6:00 p.m. to 6:25 p.m. Eastern time.

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

AbbVie to Host Fourth-Quarter and Full-Year 2018 Earnings Conference Call

On January 3, 2019 AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, reported that it will announce its fourth-quarter and full-year 2018 financial results on Friday, January 25, 2019 before the market open (Press release, AbbVie, JAN 3, 2019, View Source [SID1234532409]). AbbVie will host a live webcast of the earnings conference call at 8 a.m. Central time (9 a.m. Eastern). It will be accessible through AbbVie’s Investor Relations website investors.abbvie.com. An archived edition of the session will be available later that day.

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