On June 10, 2015 Intrexon Corporation (NYSE: XON), a leader in synthetic biology, and Oragenics (NYSE:MKT – OGEN) reported a new Exclusive Channel Collaboration (ECC) to pursue development of biotherapeutics for oral mucositis (OM) and other diseases and conditions of the oral cavity, throat, and esophagus, including clinical advancement of the ActoBiotic AG013 for the treatment of OM (Press release, Intrexon, JUN 10, 2015, View Source [SID:1234514823]). OM results in the painful inflammation and ulceration of the membranes lining the oral cavity, throat, and esophagus and is among the most frequently reported adverse events associated with cancer therapy affecting up to 500,000 patients annually. At present there is no drug approved to prevent the condition broadly and therapies are primarily palliative, alleviating symptoms without addressing the underlying pathology, resulting in a significant unmet medical need.

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"We are eager to advance therapeutic programs through the established clinical foundations of the ActoBiotics platform, and moving forward in the clinic with AG013 is a natural fit for our focus on the treatment of oral cavity diseases with innovative biopharmaceuticals," said Frederick Telling, Ph.D., Chairman of the Board of Oragenics. "Work continues under our current collaboration with Intrexon centered on the development of a novel class of antibiotics known as lantibiotics, and we are excited to build on this relationship in the field of oral health to realize the promise of microbial approaches in the generation of efficacious new medicines."

"I am pleased that this new collaboration provides an opportunity for continued development of AG013 as an intervention for oral mucositis," said Stephen T. Sonis, DMD, DMSc, Senior Surgeon, Divisions of Oral Medicine, Brigham and Women’s Hospital and the Dana-Farber Cancer Institute. "AG013’s successful attenuation of chemotherapy-induced oral mucositis in the Phase 1B trial suggests that this innovative delivery platform may offer a new approach to the treatment of this important unmet clinical need."

Through the molecular engineering of food-grade microbes (Lactococcus lactis), biologically-contained ActoBiotics allow for in situ expression and secretion of novel biotherapeutic proteins and peptides. AG013 is formulated as a convenient oral rinsing solution and designed to deliver the therapeutic molecule Trefoil Factor 1 (TFF1) to the mucosal tissues in the oral cavity. TFFs are a class of peptides that are involved in protection of the gastrointestinal tissues against mucosal damage and have an important role in subsequent repair.

A phase 1B clinical trial with AG013 in 25 cancer patients with OM across 5 cancer referral centers showed that AG013 was safe and well tolerated. Data published in the journal Cancer showed a 35% reduction of the duration of ulcerative OM in the AG013-treated patients versus the placebo-treated patients. Furthermore, close to 30% of the patients treated with AG013 were full responders while all placebo-treated patients developed ulcerative OM. Additionally, a phase 1 pharmacokinetic (PK) study in 10 healthy volunteers showed that live AG013 bacteria adhere to the buccal mucosa and actively secrete protein locally, resulting in homogeneous exposure to the entire mucosal surface up to 24 hours after administration of a rinse. AG013 has already been granted Orphan Drug status in the European Union and has potential eligibility for Biologic License Application exclusivity as well as Fast Track designation with the United States Food and Drug Administration.

Under the terms of the agreement, Oragenics will have access to Intrexon’s technologies and expertise to develop products for the treatment of oral mucositis or products containing genetically modified Lactococcus lactis expressing trefoil factors in the oral cavity, throat, and esophagus for a technology access fee and will reimburse Intrexon for the research and development costs. The agreement also provides for commercial and regulatory milestone payments to Intrexon, as well as a low double-digit percentage royalty based on the net sales from collaboration products.

"AG013 represents a promising product from a truly innovative platform for the prevention of oral mucositis in cancer patients," commented Gregory Frost, Ph.D., Senior Vice President and Head of Intrexon’s Health Sector. "We are very pleased with the expanded collaboration with Oragenics, especially given their unique experience in the field of oral health using novel approaches. This ECC will further the clinical development of AG013 in the hope of providing cancer patients with a much needed adjunctive therapy for oral mucositis, a challenging side effect that can become a treatment-limiting toxicity for effective cancer therapy."

On June 10, 2015 CTI BioPharma and Baxter International reported that Patient Reported Outcomes (PROs) data from the Phase 3 PERSIST-1 trial, evaluating pacritinib in patients with myelofibrosis, will be highlighted in a late-breaking oral presentation at the upcoming 20th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), June 11-14, Vienna (Press release, CTI BioPharma, JUN 10, 2015, View Source;p=RssLanding&cat=news&id=2057957 [SID:1234505406]). Pacritinib is an investigational oral multikinase inhibitor with specificity for JAK2 and FLT3. These data were also selected for inclusion in the official EHA (Free EHA Whitepaper) Press Briefing on Friday, June 12, 2015 at 08:30 CEST.

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Myelofibrosis is associated with significantly reduced quality of life and shortened survival. As the disease progresses, the body slows production of important blood cells and within one year of diagnosis the incidence of disease-related thrombocytopenia (very low blood platelet counts), severe anemia, and red blood cell transfusion requirements increase significantly. Among other complications, most patients with myelofibrosis present with enlarged spleens (splenomegaly) as well as many other potentially devastating physical symptoms such as: abdominal discomfort, bone pain, feeling full after eating little, severe itching, night sweats, and tiredness.

Full details for the abstracts involving pacritinib in this year’s EHA (Free EHA Whitepaper) program are below:

Title: Patient-Reported Outcomes (PROS) in PERSIST-1: A Randomized, Multi-Country Phase III Trial of the JAK2 Inhibitor Pacritinib (PAC) VS. Best Available Therapy (BAT) in Myelofibrosis (MF)
First Author: Ruben Mesa, M.D., Deputy Director, Mayo Clinic Cancer Center, Chair of the Division of Hematology & Medical Oncology, Mayo Clinic Cancer Center, Scottsdale, AZ and one of the principal investigators for PERSIST-1
Date/Time: Sunday, June 14 at 12:15 CEST
Location: Room A7
Presentation Type: Oral Presentation
Abstract #: LB2072
This abstract is under a press embargo until Friday, June 12 at 09:30 CEST.

Title: PERSIST-1: A Phase III Study of Pacritinib (PAC) vs Best Available Therapy (BAT) in Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (PPV-MF) or Post-Essential Thrombocythemia MF (PET-MF)
First Author: Claire Harrison, M.D., Consultant Hematologist, Guy’s and St. Thomas’ NHS Foundation Trust, Guy’s Hospital, London, United Kingdom and one of the principal investigators for PERSIST-1
Date/Time: Friday, June 12 at 17:15 to 18:45 CEST
Location: Poster area (Hall C)
Presentation Type: Poster
Abstract #: LB314
The full abstract can be viewed here.

About Pacritinib

Pacritinib is an oral multikinase inhibitor with specificity for JAK2 and FLT3. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia, and lymphoma. The kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL) due to its potent inhibition of c-fms, IRAK1, JAK2, and FLT3.1

On June 10, 2015 Curis reported that data for CUDC-907 will be presented at the 20th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), being held from June 11 – June 14, 2015 in Vienna, Austria as well as at the 13th International Congress on Malignant Lymphoma (ICML), being held from June 17 – June 20, 2015 in Lugano, Switzerland (Press release, Curis, JUN 10, 2015, View Source [SID:1234505383]).

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Curis and its study investigators will present data from the Phase 1 study of CUDC-907, Curis’ proprietary oral, dual histone deacetylase (HDAC) and phosphoinositide 3-kinase (PI3K) inhibitor that is being studied in the ongoing expansion stage of a Phase 1 trial, with a focus on patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). The U.S. FDA recently granted Orphan Drug Designation to CUDC-907 for the treatment of DLBCL.

Additional information on the presentations can be found below.

CUDC-907 Presentations:

20th Congress of EHA (Free EHA Whitepaper):
Date/Time: Friday, June 12, 2015, 17:15 – 18:45 CEST
Abstract Number: P325 (Poster)
Presentation Title: A Phase 1 trial of CUDC-907, an oral, first-in-class, dual inhibitor of HDAC and PI3K, in patients with refractory or relapsed lymphoma and multiple myeloma.

Presenter: Jaye Viner, MD

13- ICML:
Date/Time: Thursday, June 18, 2015, 15:35 – 16:55 CEST (Session 6)
Abstract No: 080 (Oral)
Presentation Title: A first-in-human trial of CUDC-907, an oral, first-in-class, dual inhibitor of HDAC and PI3K, in patients with refractory/ relapsed lymphoma and multiple myeloma.

Presenter: Anas Younes, MD

On JUNE 9, 2015 Cellectis reported in order to comply with NASDAQ financial practices, is publishing for the first time its 1st quarter results (Filing, 6-K, Cellectis, JUN 9, 2015, View Source [SID:1234505385]).

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Cellectis previously published consolidated financial statements for the first six months of 2014 and for the full year 2014. The Company did not publish financial statements for first half-year and full year 2014. Cellectis did not have consolidated financial statements for the 1st and 3rd quarters 2014. Therefore, no comparative 2014 figures will be presented for 1st and 3rd quarters in 2015. Cellectis will publish full quarterly comparative figures starting 2016.

Consolidated financial statements have been prepared in accordance with International Financial Reporting Standards, or IFRS, as issued by the International Accounting Standards Board ("GAAP").

First Quarter 2015 Financial Results

Revenues and Other Income: Total revenues and other income were of €9.2 million for the first quarter 2015 (€26.5 million for FY2014) and mainly comprised €7.9 million of collaboration revenues (€11.9 million for FY2014) and €0.4 million of license revenues (€7.3 million for FY2014).

Total Operating Expenses and Other Operating Income: Total operating expenses and other operating income for the first quarter of 2015 were of €12.8 million (€31.7 million for FY2014).
R&D Expenses: Research and development expenses for the first quarter of 2015 were of €5.6 million (€14.4 million for FY2014). These expenses mainly consisted of personnel expenses (€3.5 million), external purchases and other expenses (€2.1 million).

Research and development expenses for the first quarter also reflect the impact of social charges related to stock-options and free shares granted during the first quarter (€1.9 million).

SG&A Expenses: Selling, general and administrative expenses were of €7.2 million for the first quarter 2015 (€13.1 million for FY2014). These expenses mainly related to personnel expenses (€4.9 million), and to external purchases and other expenses (€2.3 million). Selling, general and administrative expenses for the first quarter also reflect the impact of social charges related to stock-options and free shares granted during the first quarter (€3.3 million). For the first three months 2015, the non–recurring IPO expenses amounted to €0.5 million.

Financial Gain: Financial gain was of €9.9 million for the first quarter 2015 (€7.1 million for FY2014), which is mainly attributable to a favorable Euro-Dollar exchange rate applied to U.S. dollar-denominated cash and cash equivalents during the first quarter of 2015.

Net Income (Loss): Net income was of €6.3 million, or €0.23 per share, for the first quarter of 2015 (net loss of €1.0 million, or €0.00 per share, for FY2014). Adjusted net income for the first quarter of 2015 was €7.1 million, or €0.23 per share. Adjusted net income for the first quarter of 2015 excludes a non-cash stock-based compensation expense of €0.8 million. Please see "Note Regarding Use of Non-GAAP Financial Measures" for a reconciliation of GAAP net income to adjusted net income.

Cash Position: Cash and cash equivalents include cash, bank accounts, money market funds and fixed bank deposits that meet the definition of a cash equivalent. As of March 31, 2015, Cellectis had €118.4 million in cash and cash equivalents compared to €112.3 million as of December 31, 2014. Our initial public offering closed on March 30, 2015 and as of March 31, 2015, the €196.8 million of net proceeds from the initial public offering are classified under Other Current Assets. We believe that our cash and cash equivalents, together with the net proceeds from our initial public offering and our cash flow from operations (including payments we expect to receive pursuant to our collaboration agreements) and government funding of research programs will be sufficient to fund our operations through at least 2017. However, we may require additional capital for the further development of our existing product candidates and may also need to raise additional funds sooner to pursue other development activities related to additional product candidates.

On June 9, 2015 Cellular Biomedicine Group reported that it has entered into a definitive agreement to acquire from Blackbird Bio Finance ("BB") University of South Florida’s ("Licensor") next generation GVAX vaccine’s ("CD40LGVAX") related technologies and know-how for an initial consideration of $2.5 million in cash and $1.75 million in shares of the Company’s Common Stock (Filing, 8-K, Cellular Biomedicine Group, JUN 9, 2015, View Source [SID:1234505412]). The per share price will be based on the 20-day volume weighted average price ("VWAP") of the Company’s Common Stock upon the closing of the acquisition. CBMG will pay potentially more than $25 million in future milestones and royalty payments. As part of the transaction, CBMG will be the exclusive global licensee of the Licensor’s related technologies and know-how, the progeny manufacturing rights with access to a master vaccine bank originating from the University of South Florida ("USF").

The inventor of CD40LGVAX, Scott Antonia, MD, Ph.D. is currently the Department Chair of Thoracic Oncology and Program Leader of the Immuno-oncology Program at the Moffitt Cancer Center. Dr. Antonia ranks among the foremost experts in the world of immuno-oncology and is an active collaborator with large pharmaceutical companies. He is recognized as one of the world’s leading authorities in the treatment of lung cancer with immunotherapeutics and has recently joined the Company’s Scientific Advisory Board. Given the positive Phase I results of CD40LGVAX alone in non-small cell lung cancer (NSCLC), Dr. Antonia plans to combine the CD40LGVAX with a checkpoint inhibitor, anti-PD1 monoclonal antibody, Nivolumab, in a three patient lead-in Phase I clinical trial followed by a randomized Phase II clinical trial in the U.S. to evaluate the safety and efficacy of the combination in patients with Stage 4 unresectable non-small cell lung cancer. The clinical trials are expected to commence in the second half of 2015.

Dr. William (Wei) Cao, Chief Executive Officer of Cellular Biomedicine Group, commented: "This strategic acquisition will enable CBMG to broaden its product portfolio based on the acquired related technologies and know-how to augment our immuno-oncology platform portfolio, particularly cancer immunotherapy vaccine and combination technology platform. Fundamentally, it would transform CBMG into a global player with leading comprehensive cancer treatment programs. It should be noted that Dr. Antonia’s NSCLC advanced clinical programs using proprietary "off-the-shelf" vaccines, would be conducted in the United States with CBMG funding the vaccine supply. We are committed to deploying resources in the U.S. to support the clinical trials, serve U.S. patients and obtain eventual FDA regulatory approval. This represents a major milestone in our foray into the U.S. market. In addition, upon receiving the requisite regulatory approval, we will seek approval to conduct clinical trials in China with leading medical centers to serve China’s patients with lung cancer. We look forward to continuing to explore other international partnerships and licensing opportunities."

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ABOUT NSCLC

Based on the latest data available from NCCN Clinical Practice Guidelines in Oncology Non-Small Cell Lung Cancer (Version 4. 2014), an estimated 224,210 people in the United States were diagnosed with lung cancer in 2014, with an estimated 159,260 deaths occurring because of the disease. In China, 728,552 people were diagnosed with lung cancer in 2012, and 592,410 people in China died from lung cancer in 2012 (source: Chinese Cancer Registry annual report 2012 & GMCD40L Study Synopsis). NSCLC is relatively insensitive to chemotherapy and radiation therapy. Despite the advances of targeted therapies and recent breakthroughs with immune checkpoint inhibitors, such as anti-PD1 or PDL1 monoclonal antibody treatments, there are still significant unmet medical needs in NSCLC. CD40LGVAX vaccine, in combination with an anti-PD1 monoclonal antibody, may provide synergistic and improved clinical benefits in both PDL1 positive and negative patients.

ABOUT CD40LGVAX

CD40LGVAX is a vaccine in which irradiated allogeneic lung adenocarcinoma cells are combined with a bystander cell line transfected with hCD40L and hGM-CSF. The key differentiator is the transfection of the bystander cell line with GM-CSF and CD40L. Both GM-CSF and CD40L can activate dendritic cells (DC). Nivolumab, an anti-PD-1 monoclonal antibody, enhances cytotoxic T cell activity by blocking the interaction between PD-1 and its receptors. The vaccine has previously been tested in a Phase I trial, and has shown encouraging efficacy and toxicity profile. In the United States, the Food and Drug Administration ("FDA") has approved Nivolumab for treatment of patients with melanoma and advanced squamous NSCLC who have progressed on or after platinum-based chemotherapy. About 25% to 30% of all NSCLC are squamous. By combining CD40LGVAX with an anti-PD1 monoclonal antibody, the approach is expected to further boost the body’s immune system to kill cancer cells. Given the strength of both products and potential synergistic mechanism of action, this potential combination may provide more clinical benefit to NSCLC than either product alone.