On September 5, 2017 GT Biopharma Inc. reported the closing of the merger of GT Biopharma, Inc. (formerly Oxis International Inc.) (OTCQB: OXISD) and Georgetown Translational Pharmaceuticals Inc (Press release, GT Biopharma, SEP 5, 2017, View Source [SID1234539564]). The merger brings GT Biopharma, Inc a new Chief Executive Officer, a robust intellectual property portfolio, and a multi-million dollar financing that leaves the Company well-positioned to continue pursuing its pipeline of oncology and Central Nervous System (CNS) drugs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As part of the transaction, GTP shareholders were issued 16,927,878 of GT Biopharma (OTCQB: OXISD) common stock. Also, as a requirement of Closing, GT Biopharma completed a financing of over $4.5 million and retired 100% of the company’s debt. The new funding provides capital for GT Biopharma to continue to pursue regulatory approval of the drugs in its oncology pipeline and newly acquired CNS pipeline. Completion of the merger and elimination of all debt is a key milestone accomplishment which positions GT Biopharma to pursue its strategy for acceptance to the Nasdaq Exchange.

The merger closed just days after Gilead Sciences announced it would pay $11.9 billion to acquire Kite Pharma (symbol: KITE) and its CAR-T cancer treatment, shining a spotlight on companies pursuing immunotherapy cancer treatment – including GT Biopharma’s platform targeted immunotherapy BiKE and TriKE technologies.

Anthony J. Cataldo, GT Biopharma’s Executive Chairman said, "This is a major accomplishment for our shareholders. We have now positioned the company to be a significant player in the expanding fight against cancer. In this calendar year we corrected our balance sheet, advanced our product pipeline (OXS-1550 phase 2 FDA trial), signed a partnership agreement with Altor Biosciences, Inc., chaired by Dr. Patrick Soon-Shiong, and advanced our highly valued TriKE platform, which is anticipated to initiate clinical trials in the US in the first half of 2018. GT Biopharma research partners at the University of Minnesota were awarded the NIH REACH award in 2016 to further pursue TriKE technology. This is given by the NIH for technologies judged likely to achieve commercial success. We also brought in an accomplished CEO (Kathleen Clarence-Smith, MD, PhD). She has already demonstrated at several major pharmaceutical companies and at VC-backed start-up companies (most recently Chase Pharmaceuticals) her ability to efficiently and rapidly develop drugs through the FDA process. Additionally, she brings to GT Biopharma a team of highly experienced, seasoned professionals in drug development, and a suite of products which are close to filing an application with the FDA to obtain approval (NDA) to market the products."

"I am pleased to be part of GT Biopharma. Our pipelines in oncology and CNS are bulging and our knowledge and expertise are in place. We are fully prepared to execute on our development plans to shepherd our drugs to commercialization and meet the urgent and expanding needs of patients with oncological and neurological disorders," said Dr. Kathleen Clarence-Smith.

Prior to founding GTP, Dr. Clarence-Smith co-founded Chase Pharmaceuticals Corporation in Washington D.C. and served as Chairman of the company’s Board from 2008 to 2014. Chase Pharmaceuticals was acquired by Allergan, PLC (AGN) in 2016 in a deal that, with significant up-front payment and milestones could reach $1 billion.

Dr. Clarence-Smith also held executive management positions with Sanofi, Roche, Otsuka Pharmaceutical and Prestwick Pharmaceuticals. She is co-founder and a managing member of KM Pharmaceutical Consulting in Washington, D. C.

GT Biopharma shares will trade under the symbol OXISD for before shifting to GTBP later this month.

Georgetown’s CNS pipeline includes Pain Brake for the treatment of chronic neuropathic pain and a drug candidate GTP-004 for the treatment of myasthenia gravis, a rare muscular disease. The only approved drug for this disease (pyridostigmine) carries significant GI side effects, limiting its achievable efficacy. GTP-004 combines pyridostigmine with another approved treatment. The goal is to reduce side effects, providing greater safety, and to improve efficacy over existing protocols involving treatment with pyridostigmine.

A third drug candidate, GTP-011, is a treatment for motion sickness. This is a repurposed version of an existing drug. It was designed to be as effective as the scopolamine patch, but to have fewer side effects, especially fewer memory problems. Reducing or even eliminating memory disturbances is particularly important in elderly patients.

About GT Biopharma, Inc.: GT Biopharma, Inc (formerly known as Oxis International, Inc.) is an immuno-oncology focused company developing innovative drugs focused on the treatment of cancer and other unmet medical needs. Oxis’ lead drug candidate, OXS-1550 (DT2219ARL) is a novel bispecific scFv recombinant fusion protein-drug conjugate composed of the variable regions of the heavy and light chains of anti-CD19 and anti-CD22 antibodies and a diphtheria toxin as its cytotoxic drug payload. OXS-1550 targets and binds to cancer cells expressing the CD19 receptor or CD22 receptor or both receptors. When OXS-1550 binds to cancer cells they internalize the drug and are killed due to the cytotoxic payload. OXS-1550 has demonstrated encouraging results in early human clinical trials in patients with relapsed/refractory B-cell lymphoma or leukemia. OXS-3550 TriKE technology was developed by researchers at the University of Minnesota Masonic Cancer Center. As demonstrated in non-clinical models, this targeted immunotherapy directs NK cells to kill cancer cells while diminishing drug-related toxicity, and is anticipated to be to NK cells what CAR-T is to T-cells.

On September 4, 2017 Takeda Pharmaceutical Company Limited (TSE: 4502) and Noile-Immune Biotech Inc. reported that they have entered into a collaboration to develop next generation chimeric antigen receptor T cell therapy (CAR-T) (Press release, Noile-Immune Biotech, SEP 4, 2017, View Source [SID1234533339]). The next generation CAR-T cell therapy was developed by Professor Koji Tamada at Yamaguchi University and Noile-Immune has exclusive license for this platform technology. The CAR-T therapy produces cytokines, chemokines, and other molecules, which is expected to potentially influence or alter the tumor microenvironment of solid tumor tissues to enhance the anti-tumor effect of the therapy. The companies intend to use this technology to discover and develop new CAR-T cell immunotherapies, with the aim of treating a broad range of cancers.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The collaboration between Takeda and Noile-Immune Biotech will accelerate research and development of CAR-T cell therapy. In addition to providing resources required for implementation, Takeda will make a technology access payment to Noile-Immune Biotech and additionally Takeda will make an equity investment. Takeda will have exclusive options to obtain licensing rights for the development and commercialization of Noile-Immune’s pipeline and products resulting from this partnership on pre-agreed

terms. Additional terms of this agreement are not disclosed.

"This technology forms the basis for developing potentially transformational treatments for solid tumors," said Dr. Hidenobu Ishizaki, President of Noile-Immune. "The platform was developed by our founder, director, and CSMO, Professor Koji Tamada at the Department of Immunology at Yamaguchi University Graduate School of Medicine. We believe our collaboration with Takeda is a significant step towards rapidly delivering therapies that use this technology to cancer patients."

"We recognize the enormous potential of next-generation CAR-T cell therapy technology to deliver transformative medicines in oncology, one of our core therapeutic areas," said Chris Arendt, Head of the Oncology Drug Discovery Unit, Takeda. "This collaboration is another example of our commitment to invest in highly innovative technologies and to work with top external scientific and clinical teams as we seek to deliver therapies that address the needs of patients with cancer. We are especially excited that our

collaboration with the outstanding team at Noile-Immune will be located at our cutting-edge Shonan Research Center in Japan, allowing our Takeda scientists to work side-by-side with the Noile-Immune team to accelerate the advancement of innovative cellular immunotherapies to the clinic." Takeda signed an agreement with Noile-Immune through its wholly-owned subsidiary, Millennium Pharmaceuticals, Inc

IO101 is a peptide vaccine encompassing an HLA-A2-restricted, 9 amino acid IDO-derived CD8+ T-cell epitope. IO101 is currently in phase II clinical trial.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A first-in-man clinical phase I vaccination trial using IO101 was conducted. The study comprised 15 HLA-A2+ patients with advanced NSCLC who were vaccinated with IO101 in Montanide adjuvant following pre-treatment with topical Imiquimod (Aldara Cream). The vaccine was well tolerated with manageable side effects and no CTCAE grade 3/4 toxicities.

One patient obtained an objective partial response after demonstrating continuous regression of liver metastases on vaccine treatment for 1 year.

Presently a small phase II study is ongoing in melanoma patients. In this study, IO101 together with an A2 Survivin peptide is used for vaccination in combination with temozolomide chemotherapy treatment.

More information can be found in this recent publication: Long-lasting disease stabilization in the absence of toxicity in metastatic lung cancer patients vaccinated with an epitope derived from indoleamine 2,3 dioxygenase

On September 4, 2017 Cellectis (Paris:ALCLS) (NASDAQ:CLLS) a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported having received notice from the U.S. Food and Drug Administration (FDA) that a clinical hold was placed on both UCART123 ongoing Phase 1 studies, respectively in acute myeloid leukemia (AML) and in blastic plasmacytoid dendritic cell neoplasm (BPDCN) (Press release, Cellectis, SEP 4, 2017, View Source [SID1234520368]).

Cellectis is working closely with the investigators and the FDA in order to resume the trials with an amended protocol including a lowered dosing of UCART123.

The clinical hold was initiated after Cellectis reported one fatality in the BPDCN clinical trial (ABC study). This was the first patient treated in the BPDCN study, a 78-year-old male treated with one prior therapy, who presented with relapsed/refractory BPDCN with 30% blasts in his bone marrow and cutaneous lesions (biopsy-proven BPDCN) at baseline prior to conditioning regimen. He received 30mg/m2/day fludarabine for 4 days and 1g/m2/day cyclophosphamide for 3 days, as a preconditioning regimen. On August 16, 2017 (Day 0), he received 6.25×105 UCART123 cells per kilogram, the first dose level explored in the protocol, without complication. At Day 5, the patient experienced a grade 2 Cytokine Release Syndrome (CRS)1, and a grade 3 lung infection, which quickly improved after a first dose of tocilizumab and institution of anti-infective therapy (broad spectrum intravenous antibiotics). He then experienced at Day 8 a grade 5 CRS, together with a grade 4 Capillary Leak Syndrome2. Despite a treatment in keeping with CRS management including administration of corticosteroids and tociluzumab x 2 as well as intensive care unit support, the patient died on Day 9.

The first patient treated in the AML study was a 58-year-old woman, with 84% blasts in her bone marrow at baseline prior to conditioning regimen. On June 27, 2017 (Day 0), the patient received the same preconditioning regimen and the same dose of UCART123 as the BPDCN patient, without complication. She experienced an initial grade 2 CRS at Day 8, worsening to a grade 3 at Day 9 and resolving at Day 11 with treatment management in intensive care unit. She also experienced a grade 4 Capillary Leak Syndrome at Day 9, resolved at Day 12.

No GvHD3 was reported for any of these patients.

The DSMB (Data Safety Monitoring Board) met on August 28 and recommended lowering the dose to 6.25×104 UCART123 cells per kilogram in both studies and capping cyclophosphamide to a total dose of 4g over 3 days.

On September 1, 2017 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs that address inflammatory and cancer diseases, reported financial results for the six months ended June 30, 2017 and provided clinical and corporate updates (Press release, Can-Fite BioPharma, SEP 1, 2017, View Source [SID1234520359]).

Clinical Development Program and Corporate Highlights Include:

Namodenoson (CF102): Progress in Clinical Development and Receipt of Milestone Payment

● Patient Enrollment Completed in Phase II Liver Cancer Trial of Namodenoson

Can-Fite enrolled and randomized all 78 patients for its global Phase II study of Namodenoson in the treatment of hepatocellular carcinoma (HCC), the most common form of liver cancer. Patients with advanced HCC, Child Pugh B, were enrolled in the U.S., Europe and Israel. The primary endpoint of the Phase II study is overall survival. Can-Fite is following the survival data closely and plans to perform the survival analysis at the earliest possible opportunity. The HCC market is expected to generate $1.4 billion in sales in 2019.

● Milestone Payment Received for Distribution of Namodenoson in Korea for the Treatment of Liver Cancer

In August 2017, Can-Fite received a milestone payment of $500,000 from Chong Kun Dang Pharmaceuticals (CKD), which licensed the exclusive right to distribute Namodenoson for the treatment of liver cancer in Korea upon receipt of regulatory approvals. The payment is part of a deal worth up to $3,000,000 in upfront and milestone payments plus 23% royalties.

● Namodenoson to Commence Phase II Trial in the Treatment of NAFLD/NASH

During the second quarter of 2017, Can-Fite conducted a successful Clinical Investigator Meeting with principal investigators, researchers, and doctors participating as clinical investigators in the Company’s Phase II trial of Namodenoson in the treatment of non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). Namodenoson drug supply for the trial has been paid for and is ready to be administered to patients. The trial protocol has been approved by two leading Institutional Review Boards in Israel. Can-Fite estimates the cost of this Phase II trial to be less than $1 million. Patient enrollment is expected to begin in the third quarter of 2017. By 2025, the addressable pharmaceutical market for NASH is estimated to reach $35-40 billion.

● Established Clinical Advisory Board for NASH Indication

Can-Fite established a Clinical Advisory Board comprised of Key Opinion Leaders to provide advice and steer Namodenoson’s clinical development program in the treatment of NAFLD and NASH. Board members include researchers and medical practitioners in the field of liver disease from institutions including Mount Sinai in New York, Virginia Commonwealth University School of Medicine, and Hadassah University in Israel.

Piclidenoson (CF101): ACRobat Phase III Trial in Rheumatoid Arthritis to Commence

In August 2017, Can-Fite successfully concluded a cardiodynamic trial for its lead drug candidate Piclidenoson, which showed favorable safety data required by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) prior to initiation of Phase III studies.

During the second quarter of 2017, Can-Fite conducted a successful Clinical Investigator Meeting with approximately 100 rheumatologists, and their staff, who are participating as clinical investigators in the Company’s global pivotal Phase III ACRobat study, which is set to commence patient enrollment in the third quarter of 2017.

ACRobat is a Phase III trial that will evaluate Piclidenoson as a first line treatment and replacement for the current standard of care, Methotrexate (MTX), the most widely used drug for rheumatoid arthritis. The trial will enroll approximately 500 patients in Europe, Canada and Israel. The estimated cost of the entire Phase III study is approximately $5 million. Rheumatoid arthritis is a treatment market forecast to reach $34.6 billion by 2020.

Piclidenoson is also being developed as a treatment for psoriasis. Can-Fite is preparing for an upcoming Phase III trial that will investigate the efficacy and safety of Piclidenoson compared to placebo as its primary endpoint and as compared to apremilast (Otezla) as its secondary endpoint in approximately 400 patients with moderate-to-severe plaque psoriasis. Can-Fite expects to submit its clinical protocol to Institutional Review Boards in the fourth quarter of 2017. The psoriasis market is forecast to be $8.9 billion in 2018 and Otezla sales are estimated to be $2.35 billion by 2020.

"Completion of patient enrollment in our Phase II study is a significant advancement in the development of Namodenoson to treat liver cancer. We will evaluate results as soon as possible based on survival data. There is a clear unmet medical need for HCC patients with Child-Pugh Class B cirrhosis. Our Orphan Drug status in both the U.S. and Europe, as well as Fast Track status in the U.S. in this indication, we believe will accelerate our path towards applying for market approval for Namodenoson for liver cancer," stated Can-Fite CEO Dr. Pnina Fishman. "In the current quarter, we look forward to commencing patient enrollment in our Phase II NAFLD/NASH study for Namodenoson and our Phase III rheumatoid arthritis study for Piclidenoson."

Financial Results

Revenues for the six months ended June 30, 2017 were NIS 0.53 million (U.S. $0.15 million) compared to NIS 0.43 million (U.S. $0.12 million) in the first six months of 2016. The increase in revenue was mainly due to the recognition of a portion of the NIS 1.9 million (U.S. $0.5 million) advance payment received in December 2016 under the distribution agreement with CKD.

Research and development expenses for the six months ended June 30, 2017 were NIS 8.84 million (U.S. $2.53 million) compared with NIS 9.97 million (U.S. $2.85 million) for the same period in 2016. Research and development expenses for the first half of 2017 comprised primarily of expenses associated with the Phase II study for Namodenoson, the preclinical study of CF602, as well as expenses for ongoing studies of Piclidenoson. The decrease is primarily due to a reduction in preclinical studies of CF602 conducted during the six months ended June 30, 2017.

General and administrative expenses were NIS 5.0 million (U.S. $1.43 million) for the six months ended June 30, 2017, the same as the general and administrative expenses for the same period in 2016.

Financial income, net for the six months ended June 30, 2017 aggregated NIS 1.57 million (U.S. $0.45 million) compared to financial income, net of NIS 3.19 million (U.S. $0.91 million) for the same period in 2016. The decrease in financial income, net in the first half of 2017 was mainly from exchange rate differences as compared to the same period in 2016 and from issuance expenses, offset by a larger decrease in the fair value of warrants that are accounted for as financial liability as compared to the same period in 2016.

Can-Fite’s net loss for the six months ended June 30, 2017 was NIS 11.72 million (U.S. $3.35 million) compared with a net loss of NIS 11.35 million (U.S. $3.25 million) for the same period in 2016. The slight increase in net loss for the first half of 2017 was primarily attributable to a decrease in financial income, net.

As of June 30, 2017, Can-Fite had cash and cash equivalents of NIS 23.98 million (U.S. $6.86 million) as compared to NIS 31.2 million (U.S. $8.92 million) at December 31, 2016. The decrease in cash during the six months ended June 30, 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 June 30, 2017 (U.S. $1 = NIS 3.496).

The Company’s consolidated financial results for the six months ended June 30, 2017 are presented in accordance with International Financial Reporting Standards.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!