On March 23, 2018 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small-molecule drugs that address cancer, liver disease and inflammatory diseases, reported it has filed its 2017 Annual Report on Form 20-F with the U.S. Securities and Exchange Commission (Press release, Can-Fite BioPharma, MAR 23, 2018, View Source [SID1234524956]).

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Clinical Development Program and Corporate Highlights Include:

Piclidenoson (CF101)

A multi-million dollar agreement has been signed with Gebro Holdings for the distribution of piclidenoson in 3 European Countries

Under the terms of the distribution agreement, Gebro made a total upfront and milestone payment of approximately $2,200,000 to Can-Fite. In addition, the agreement provides that additional payments of up to approximately $7,000,000 will be received by Can-Fite upon the achievement of certain regulatory, launch and sales milestones plus double-digit percentage royalty payments on net sales.

Patient enrolment for the ACRobat Phase III Trial in Rheumatoid Arthritis is ongoing

During the fourth quarter of 2017, Can-Fite commenced enrollment in the pivotal Phase III ACRobat trial of approximately 500 patients through clinical sites in Europe, Israel and Canada. The study aims to evaluate Piclidenoson (CF101) as a first line treatment and replacement for the current standard of care, Methotrexate (MTX), the most widely used drug for rheumatoid arthritis. The primary endpoint of ACRobat is low disease activity after 12 weeks of treatment in patients dosed with Piclidenoson compared to those dosed with MTX. Piclidenoson at 1 mg and 2 mg, or placebo, will be administered twice daily, and MTX or placebo will be administered once weekly. The total study duration will be 24 weeks.

The rheumatoid arthritis market is forecast to reach $34.6 billion by 2020.

A patent for Psoriasis has been approved in Korea

The Korean Intellectual Property Office issued patent No. 10-1741281 titled, "Pharmaceutical Composition Comprising A3 Adenosine Receptor Agonist (IB-MECA/CF-101) For Treatment of Psoriasis" for the Company’s lead drug candidate Piclidenoson in its psoriasis indication. Can-Fite has two distribution agreements in Korea, including one with Kwang Dong Pharmaceutical for Piclidenoson in the treatment of rheumatoid arthritis and another with Chong Kun Dang for Namodenoson in the treatment of liver cancer.

Namodenoson (CF102)

Progress of the Phase II Liver Cancer Namodenoson (CF102) Study in the treatment of advanced HCC – Data to be released H2/2018

Current data indicate potentially favorable drug safety profile. The global Phase II study is being conducted in the U.S., Europe and Israel. Patients with advanced HCC, Child Pugh B, who failed Nexavar (sorafenib) as a first line treatment are being treated twice daily with 25 mg of oral Namodenoson or placebo using a 2:1 randomization. The primary endpoint of the Phase II study is Overall Survival (OS). Secondary endpoints include Progression Free Survival (PFS), safety, and the relationship between outcomes and A3AR expression. A total of approximately 78 patients have been enrolled in the Phase II study. As of December 2017, 15 subjects have completed at least 12 cycles of treatment (each cycle is 28 days of treatment), of which two completed 24 cycles. The Company anticipates data release to occur in 2H 2018.

The market for hepatocellular carcinoma drugs is expected to generate $1.4 billion in sales in 2019.

Phase II NAFLD/NASH Study is enrolling patients; Data Release Expected in H1 2019

Can-Fite is currently enrolling patients in a Phase II study in the treatment of non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH) with Namodenoson which plans to enroll 60 patients. Can-Fite’s 12-week study is being led by key opinion leaders in the area of NASH and liver diseases. Clinical trial sites are some of the most prestigious medical institutions in Israel, including Hadassah Medical Center and Rabin Medical Center. Patients are being enrolled in three arms, including two different dosages of oral Namodenoson twice daily versus placebo. The study’s primary endpoints are the mean percent change from baseline in serum alanine aminotransferase (ALT) levels and safety. The secondary endpoint is percent change from baseline in hepatic steatosis measured by magnetic resonance imaging-determined by proton-density fat-fraction (MRI-PDFF) and additional metabolic parameters.

The Phase II trial design was based on preclinical studies showing Namodenoson’s efficacy in reducing liver steatosis, inflammation and fibrosis in experimental NASH models.

There is currently no U.S. FDA approved drug for the treatment of NASH, which is an addressable pharmaceutical market estimated to reach $35-40 billion by 2025.

"We’ve made several notable accomplishments during this past year which validate the strength of our drug portfolio and demonstrate our commitment in execution. We believe these events position us well for the year ahead in which we anticipate a marked progress with our Piclidenoson and Namodenoson drug candidates. We are pleased with the progress thus far and recently announced the submission of safety reports for Piclidenoson and Namodenoson to the FDA and other regulatory authorities so that we may continue with the various clinical studies. Our drug candidates are highly unique due to our favorable safety profile and the specific anti-inflammatory and anti-cancer effects. In the coming year 2018, we remain focused on execution with the goal of providing our patients a better alternative option and treatment for autoimmune inflammatory, oncology and NASH/NAFLD," stated Can-Fite CEO Dr. Pnina Fishman.

Financial Results

Revenues for the year ended December 31, 2017 were NIS 2.9 million (U.S. $0.85 million), an increase of NIS 2.3 million (U.S. $0.66 million), or 350%, compared to NIS 0.65 million (U.S. $0.2 million) for the year ended December 31, 2016. The revenues during 2017 were mainly due to a portion of the NIS 0.76 million (U.S. $0.22 million or CAD 0.2 million) advance payment received in March 2015 under the distribution agreement with Cipher and from the recognition of the milestone payment of NIS 1.8 million (U.S. $0.5 million) and the recognition of a portion of the NIS 0.4 million (U.S. $0.1 million) advance payment received in December 2016 under the distribution agreement with CKD.

Research and development expenses for the year ended December 31, 2017 were NIS 18.3 million (U.S. $5.28 million), a decrease of NIS 5 million (U.S. 1.44 million), or 22%, compared to NIS 23.4 million (U.S. $6.74 million) for the year ended December 31, 2016. Research and developments expenses for the year ended 2017 comprised primarily of expenses associated with the Phase II studies for Namodenoson as well as expenses for ongoing studies of Piclidenoson. The decrease is primarily due to costs associated with CF602 expenses that decreased since the postponement of a planned IND submission for this indication and decreased costs associated with the ongoing clinical trial of Namodenoson for treatment in liver cancer. The Company expects that the research and development expenses will increase through 2018 and beyond.

General and administrative expenses were NIS 10.2 million (U.S. $2.94 million) for the year ended December 31, 2017 a decrease of NIS 0.2 million (U.S. $0.06 million), or 2%, compared to NIS 10.5 million (U.S. $3.02 million) for the year ended December 31, 2016. The minor decrease is primarily due to a decrease in investor and public relations expenses. The Company expects that general and administrative expenses will remain at the same level through 2018.

Financial income, net for the year ended December 31, 2017 aggregated NIS 6.6 million (U.S. $1.89 million) compared to financial income, net of NIS 6.31 million (U.S. $1.82 million) for the same period in 2016. The slight increase in financial income, net in the year ended December 31, 2017 was mainly due to issuance expenses recorded in 2017 which were not recorded in 2016, a decrease in net change in fair value of warrants exercisable into shares and a decrease in exchange rate difference expenses.

Can-Fite’s net loss for the year ended December 31, 2017 was NIS 17.3 million (U.S. $4.99 million) compared with a net loss of NIS 27 million (U.S. $7.78 million) for the year ended December 31, 2016. The decrease in net loss for the year ended December 31, 2017 was primarily attributable to a decrease in research and development expenses.

As of December 31, 2017, Can-Fite had cash and cash equivalents of NIS 12.1 million (U.S. $3.51 million) as compared to NIS 31.2 million (U.S. $8.99 million) at December 31, 2016. The decrease in cash during the year ended December 31, 2017 is due to use of cash to fund operating expenses.

For the convenience of the reader, the reported NIS amounts have been translated into U.S. dollars, at the representative rate of exchange on December 31, 2017 (U.S. $1 = NIS 3.467).

The Company’s consolidated financial results for the year ended December 31, 2017 are presented in accordance with International Financial Reporting Standards.

The 2017 Annual Report can be found on the Company’s website at www.canfite.com as well as on the SEC website at www.sec.gov. In addition, security holders may request a hard copy of the Annual Report, which includes the Company’s complete audited financial statements, free of charge. Requests can be made by contacting Can-Fite Investor Relations at 10 Bareket Street, Kiryat Matalon, Petah-Tikva 4951778, Israel or by phone at +972-3-9241114.

On March 22, 2018 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) reported that it filed a Registration Statement on Form F-1 with the U.S. Securities and Exchange Commission (SEC) for a proposed offering of ordinary shares in the form of American Depositary Shares ("ADSs") in the United States (Press release, MorphoSys, MAR 22, 2018, View Source [SID1234556339]). The final number of ADSs to be offered and the price for the offering have not yet been determined.

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MorphoSys’s ordinary shares are listed on the Prime Standard Segment in Frankfurt, Germany. Application has been made to list the ADSs to be offered in the proposed offering on the Nasdaq Global Market in the United States under the ticker symbol "MOR".

Goldman Sachs & Co. LLC, J.P. Morgan Securities LLC and Leerink Partners LLC, are acting as lead book-running managers, and Berenberg Capital Markets, LLC and JMP Securities LLC are acting as co-managers for the proposed ADS offering.

A Registration Statement relating to these securities has been filed with the U.S. Securities and Exchange Commission but has not yet become effective. The securities may not be sold, nor may offers to buy be accepted, prior to the time the Registration Statement becomes effective.

The securities referred to in this release are to be offered only by means of a prospectus. A copy of the preliminary prospectus, when available, can be obtained from Goldman Sachs & Co. LLC, Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526, facsimile: 1-212-902-9316 or by e-mailing [email protected]; J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, telephone: 1-866-803-9204; Leerink Partners LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at 1-800-808-7525, ext. 6132, or by emailing [email protected].

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

On March 22, 2018 Astellas Pharma Inc. (President and CEO: Yoshihiko Hatanaka, "Astellas" ) reported that the Japanese Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation to gilteritinib for the treatment of FLT3mut+ Acute Myeloid Leukemia (AML), the drug’s prospective indication (Press release, Astellas, MAR 22, 2018, View Source [SID1234536693]).

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Orphan drug designation system in Japan aims to support the development of drugs for diseases that, despite there being a significant medical need for treatments, affect only a small number of patients, and for which research and development is virtually nonexistent. As stipulated in Article 77 of the Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act of Japan, the designation is granted by the minister of Health, Labour and Welfare for drugs that meet the designation criteria which include the following: the number of patients who may use the drug is less than 50,000 in Japan; there is no alternative appropriate drug or treatment in Japan; high efficacy or safety is expected compared to existing products. Specific measures to support the development of orphan drugs include subsidies for research and development expenditures, prioritized consultation regarding clinical development, reduced consultation fees, tax incentives, priority review of applications, reduced application fees, and extended registration validity period1

AML is a cancer that impacts the blood and bone marrow, and its incidence increases with age. In Japan, approximately 5,500 patients are diagnosed with AML each year2. Gilteritinib is an investigational compound that has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) as well as FLT3 tyrosine kinase domain (TKD), two common types of FLT3 mutations that are seen in approximately one-third of patients with AML. Further, gilteritinib has also demonstrated inhibition of the AXL receptor in AML cell lines.

Astellas will continue to develop gilteritinib to provide it to FLT3mut+ AML patients as early as possible.

(1): View Source
(2): KantarHealth. TREATMENT ARCHITECTURE: JAPAN LEUKEMIA, ACUTE MYELOID. CancerMPact Japan, February 2017.

On March 22, 2018 Rheos Medicines, Inc. was launched with $60 million in Series A financing backed by Third Rock Ventures, LLC. Rheos is pioneering immunometabolism as a novel approach to tune metabolic pathways in immune cells to treat disease (Press release, Rheos Medicines, MAR 22, 2018, View Source [SID1234532790]). The company will translate recent breakthroughs in the understanding of immune cell metabolism to develop precision medicines for immune-mediated diseases.

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The number of patients with immune-mediated diseases is large and growing in Western society, with an estimated 7-10 percent of the population with autoimmune diseases1 and 5-7 percent of the population with immune-mediated inflammatory diseases.2 Despite recent advances in treatment, there remains a significant unmet need. Understanding how variations in immune cell metabolism underlie patient heterogeneity provides an opportunity to bring precision treatments to patients.

New, in-depth understanding of immune cell physiology based upon research by leading academic scientists, including Rheos’s founders, points to cellular metabolism as a key driver of immune cell response. Rheos’s product engine takes a fundamentally novel approach to immune-mediated diseases by encompassing the full range of immune cells and their functions, and how they are regulated by cellular metabolism. In addition, by identifying new therapeutic targets and related biomarkers, the Rheos engine will address patient heterogeneity inherent to many immune-mediated diseases.

"The emerging field of immunometabolism offers a tremendous opportunity to set a higher standard for how immune-mediated diseases are treated. This opens an opportunity for Rheos to direct our medicines to a new aspect of disease pathogenesis by targeting the underlying cellular metabolism of immune cells," said Abbie Celniker, PhD, Chief Executive Officer of Rheos. "By building on the discoveries of our founders, Rheos is developing a biomarker and drug discovery engine that will allow us to address disease biology and patient variability by ‘tuning’ immune cells in select patient populations with precision medicines."

Rheos Product Engine and Pipeline

Rheos has developed a product engine that offers unprecedented insight into the drivers of immune-mediated diseases by characterizing how different immune cell types impact disease progression in different patient subpopulations. By simultaneously identifying new drug targets and characterizing biomarkers of disease, the Rheos product engine enables a precision medicine approach to treatment of immune-mediated diseases. Using the latest technological advances– including DNA sequencing, transcriptional and metabolomic profiling – along with incorporating primary patient samples, the Rheos product engine is an integrated immune cell metabolism and physiology platform.

Central to this product engine is a proprietary Immune Cell Encyclopedia (ICE), which maps the metabolic pathways used by different types of immune cells to regulate their fate and function in disease and in health. The initial focus of Rheos’s product pipeline is on therapeutics that target CD4 and CD8 T cell subtypes, which are involved in diseases such as inflammatory bowel disease, psoriasis, vitiligo and in immuno-oncology applications. These immune cell subtypes are critical to disease pathogenesis, and Rheos’s product engine is providing insight into how these cells drive immune response in disease.

"I have firsthand experience treating patients with immune-mediated diseases based on my years in clinical practice, and it is exhilarating to apply the wealth of expertise and technology at Rheos to create new treatments and address these patient needs," said Larry Turka, MD, Chief Scientific Officer and co-founder of Rheos. "I am excited to work with the talented team at Rheos and use our product engine to translate the powerful science of immunometabolism into the reality of new treatments that can make a lasting difference for the millions of patients with immune-mediated diseases."

Expert team of immunometabolism scientists and immune disease clinicians

The Rheos leadership team includes recognized leaders in immunometabolism, target discovery, translational medicine and company building. Company leaders include Abbie Celniker, PhD, interim Chief Executive Officer; Cary Pfeffer, MD, interim Chief Business Officer; Laurence Turka, MD, Chief Scientific Officer; Edward Driggers, PhD, Chief Technology Officer; Ryan Cohlhepp, PharmD, Senior Vice President, R&D Strategy and Operations; Brian Albrecht, PhD, Vice President, Drug Discovery; and Hozefa Bandukwala, PhD, Senior Director, Head of Discovery Biology.

Rheos’s internal team is working alongside a founding team of leading scientists whose discoveries opened the field of immunometabolism and clinicians with deep understanding of immune-mediated diseases. The company’s scientific founders are:

Richard Flavell, PhD, Sterling Professor of Immunobiology, Yale University; Investigator, Howard Hughes Medical Institute. Flavell is a world-renowned immunologist who pioneered the use of transgenic mouse models to study autoimmune and inflammatory diseases. Richard is a member of the National Academy of Sciences, National Academy of Medicine (USA), and a Fellow of the Royal Society.
Edward Pearce, PhD, Senior Group Leader, Max Planck Institute of Immunobiology and Epigenetics; Faculty of Biology, University of Freiburg. Edward Pearce is a pioneer in understanding how macrophage and dendritic cell metabolism influences the function of those cells.
Erika Pearce, PhD, Director, Max Planck Institute of Immunobiology and Epigenetics. Erika Pearce is a world leader in T cell metabolism and understanding how different metabolic pathways tune T cell function and fitness.
Ken Smith, MD, PhD, Professor of Medicine and Head of the Department of Medicine, University of Cambridge. Smith is an international expert in identifying biomarkers which predict prognosis in patients with autoimmune disease.
William St. Clair, MD, Professor of Medicine and Immunology, Duke University Medical Center. Dr. St. Clair is a former President of the American College of Rheumatology, he has extensive experience in translational immunology and the design and implementation of trials in autoimmune diseases.
Laurence Turka, MD, CSO, Rheos Medicines. Turka is former President of the American Society of Transplantation, a leader in the fields of T cell costimulation and regulatory T cell biology. Prior to joining Rheos, Laurence was the Harold and Ellen Danser Professor of Surgery and Professor of Medicine at Harvard Medical School and Massachusetts General Hospital.

On March 22, 2018 Celyad Reported 2017 Financial and OperatingResults and Expected Key Milestones for 2018 (Press release, Celyad, MAR 22, 2018, View Source(3).pdf [SID1234532496]).

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