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FDA Grants Fast Track Designation to Tocagen’s Toca 511 & Toca FC for Treatment of Recurrent High Grade Glioma

On July 9, 2015 Tocagen reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead immuno-oncology product, Toca 511 & Toca FC, for the treatment of recurrent high grade glioma, which includes glioblastoma and anaplastic astrocytoma (Press release, Tocagen, JUL 9, 2015, View Source [SID:1234506191]). This treatment will enter a registrational study called Toca 5 later this year in patients with recurrent glioblastoma or anaplastic astrocytoma.

“The granting of Fast Track designation for Toca 511 & Toca FC underscores the urgent need for new treatments for high grade glioma, an extremely deadly and aggressive cancer,” said Harry Gruber, M.D., chief executive officer of Tocagen. “In addition to the excellent safety profile and encouraging median survival we have seen in two separate studies evaluating Toca 511 & Toca FC in patients with recurrent high grade glioma, this designation will help accelerate our development plans to address this unmet need.”

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need, with the goal of getting important new drugs to patients earlier. Fast Track designation allows for more frequent interactions with the FDA review team, including meetings to discuss the drug’s development plan to support potential drug approval and the opportunity to submit sections of an NDA on a rolling basis as data become available.

About Toca 511 & Toca FC
Toca 511 & Toca FC is an immuno-oncology therapy that will be evaluated in a registrational study to be initiated in 2015 in patients with recurrent glioblastoma or anaplastic astrocytoma. Toca 511 & Toca FC are designed to selectively transform the tumor into a chemotherapy factory while also activating the immune system against the tumor both locally and systemically. Toca 511 is a retroviral replicating vector (RRV) that selectively delivers a gene for the enzyme cytosine deaminase to the tumor. The patient then takes oral cycles of Toca FC, a novel formulation of an antifungal drug, which is converted into the FDA-approved chemotherapy drug, 5-fluorouracil (5 FU). As result, infected cancer cells are selectively killed, subsequently activating the immune system to recognize cancer cells, while leaving healthy cells unharmed. Immune activation locally in the tumor occurs through a combination of mechanisms which leads to breaking of immune tolerance and activation of the immune system selectively against the cancer cells.

Epizyme Announces Extension of Celgene Research Collaboration

On July 9, 2015 Epizyme, reported that it has amended and restated its agreement with Celgene Corporation to extend the research collaboration between the two companies for at least three additional years (Press release, Epizyme, JUL 9, 2015, View Source [SID:1234506189]).

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Under the collaboration, Celgene will have the option to license histone methyltransferase (HMT) inhibitors being developed by Epizyme against three predefined targets.

Under the terms of the revised agreement:

Epizyme will receive a $10 million extension fee from Celgene in return for an option to individually license global rights for two of the targets and ex-US rights for the third target.
Celgene may exercise its option with respect to each of the targets at the time of the IND filing for an additional pre-specified license payment.
Epizyme will be responsible for leading and funding development for each target candidate through phase 1 clinical trials.
Following the completion of phase 1, if Celgene chooses to continue its license for a specific target, it may do so by making an additional pre-specified payment.
Epizyme may earn total potential milestones of up to $610 million on the three targets, including up to $75 million in development milestones and license fees, $365 million in regulatory milestones, and $170 million in sales milestones
Epizyme also may earn a royalty of up to a low double-digit percentage on worldwide net sales for two of the product candidates, and on ex-US net sales for the third product candidate.
Epizyme will retain global rights to the remainder of its pipeline, as Celgene’s option to license ex-US rights for any other preclinical programs will terminate.
In addition, Celgene will retain its ex-US license to, and the companies will continue their ongoing clinical collaboration on, pinometostat (EPZ-5676), a HMT inhibitor targeting DOT1L. Pinometostat is in phase 1 development for the treatment of patients with acute leukemia with alterations in the MLL gene (MLL-r).

"We believe that the extension of our agreement with Celgene will accelerate our goal of developing new therapies that have the potential to help many patients with epigenetically driven cancers," said Robert Gould, Ph.D., President and Chief Executive Officer, Epizyme. "Celgene is a leading company in oncology development and commercialization and we are pleased to continue our partnership on pinometostat and these three exciting novel targets."

The term of this agreement is based on specific development milestones, including the timing of IND filings and completion of phase 1 studies, but will extend for a minimum of three years. In addition, Celgene will no longer have the right of first negotiation on a business combination with Epizyme.

Financial Update

The Company also announced today that, based on its current operating plans, it projects that its cash and cash equivalents will be sufficient to fund operations through at least the end of the second quarter of 2017, prior to including any potential option exercise fees or future milestone payments. This new cash outlook reflects a significant reallocation of resources, implementation of cost savings initiatives, the additional capital provided from the Celgene extension fee payment and the partial exercise of the overallotment option in April from the Company’s March public financing.

"We have increased investment in tazemetostat development, both as a single agent and in future studies in combination with other agents," said Andrew Singer, Executive Vice President and Chief Financial Officer at Epizyme. "This required reprioritizing our pipeline development plans and reducing operating costs. We are excited about the updated data from our dose escalation and dose expansion studies presented at the International Congress on Malignant Lymphoma in Lugano, Switzerland on June 20. We look forward to presenting additional data at the European Society for Medical Oncology’s European Cancer Congress in Vienna, Austria on September 26."

About Tazemetostat (EPZ-6438)

Epizyme is developing tazemetostat for the treatment of non-Hodgkin lymphoma patients and patients with INI1-deficient solid tumors. Tazemetostat is a first-in-class small molecule inhibitor of EZH2 developed by Epizyme. In many human cancers, aberrant EZH2 enzyme activity results in misregulation of genes that control cell proliferation resulting in the rapid and unconstrained growth of tumor cells. Tazemetostat is the WHO International Non-Proprietary Name (INN) for compound EPZ-6438.

Tazemetostat is the second HMT inhibitor to enter human clinical development (following Epizyme’s DOT1L inhibitor, pinometostat).

Epizyme is conducting a five-arm, multi-center international phase 2 clinical trial that will assess the safety and activity of tazemetostat in patients with relapsed or refractory non-Hodgkin lymphoma. A phase 1 dose escalation and dose expansion trial of tazemetostat is also ongoing, with additional data expected to be reported later in 2015. Additional information about this program, including clinical trial information, may be found here: View Source

About Pinometostat (EPZ-5676)

Epizyme is developing pinometostat, a small molecule inhibitor of DOT1L created with Epizyme’s proprietary product platform, for the treatment of patients with acute leukemia in which the MLL gene is rearranged due to a chromosomal translocation (MLL-r). Due to these rearrangements, DOT1L is misregulated, resulting in the increased expression of genes causing leukemia. Pinometostat is the WHO International Non-Proprietary Name (INN) for compound EPZ-5676.

Epizyme believes that pinometostat was the first HMT inhibitor to enter human clinical development. Epizyme is currently conducting a two-stage Phase 1 study in adult MLL-r patients and in May 2014, initiated a Phase 1b study of pinometostat in pediatric patients with rearrangements of the MLL gene. The adult dose escalation cohorts have completed enrollment, and an adult MLL-r dose expansion cohort is now enrolling patients. Additional information about these ongoing Phase 1 studies can be found here: View Source

Pinometostat has been granted orphan drug designation for the treatment of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) by the Food and Drug Administration in the U.S. and by the European Commission in Europe.

Epizyme retains all U.S. rights to pinometostat and has granted Celgene an exclusive license to pinometostat outside of the U.S.

Xenetic Biosciences Completes $3,000,000 Bridge Note Financing

On July 8, 2015 Xenetic Biosciences, Inc. ("Xenetic" or the "Company") (OTCQB:XBIO), a biopharmaceutical company focused on developing next-generation biologic drugs and novel orphan oncology therapeutics, reported that it has completed a $3,000,000 bridge note financing (Press release, Xenetic Biosciences, JUL 8, 2015, View Source [SID1234537814]). The financing was concluded with OJSC Pharmsynthez ("Pharmsynthez"), a collaborative partner of Xenetic and an affiliate of Xenetic’s largest shareholder SynBio LLC ("Synbio") and was arranged directly between the managements of Xenetic and Pharmsynthez on a commission free basis.

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The $3,000,000 was received from the sale of a ten percent (10%) senior secured collateralized convertible promissory note due July 1, 2016 (the "Note"). The Note is convertible, in whole or in part, into shares of Common Stock of the Company at the option of Pharmsynthez. In addition, in connection with the sale of the Note, Pharmsynthez received certain five year warrants to purchase shares of Common Stock of the Company. Please refer to SEC form 8-K filed on July 8, 2015 for further material information regarding the bridge note financing.

"We are very pleased to have completed this important bridge note financing with a company that has been associated with Xenetic for fifteen years," said Scott Maguire, Chief Executive Officer of Xenetic. "Pharmsynthez is an important collaborator with Xenetic. The partnership covers six drug candidates currently in development in Russia which include indications for cystic fibrosis and obesity with the intention that the Company will eventually seek approval of these drug candidates in the global markets."

"These funds will allow us to continue with our plan to seek a listing of the Company’s shares on a national U.S. securities exchange that offers greater liquidity and will allow Xenetic to trade alongside its peers, therefore allowing greater exposure to the life science investment community. We believe that this financing, which was concluded with an affiliate of our single largest shareholder, reflects a belief in the potential of the Company’s core technologies. Our primary focus remains to improve the human condition by developing and offering safer and more effective treatments via the development of our portfolio of proprietary products. We are looking forward to the future with great confidence."

Xenetic Biosciences, Inc. ("Xenetic" or the "Company") (OTCQB:XBIO), a biopharmaceutical company focused on developing next-generation biologic drugs and novel orphan oncology therapeutics, reported that it has completed a $3,000,000 bridge note financing. The financing was concluded with OJSC Pharmsynthez ("Pharmsynthez"), a collaborative partner of Xenetic and an affiliate of Xenetic’s largest shareholder SynBio LLC ("Synbio") and was arranged directly between the managements of Xenetic and Pharmsynthez on a commission free basis.

The $3,000,000 was received from the sale of a ten percent (10%) senior secured collateralized convertible promissory note due July 1, 2016 (the "Note"). The Note is convertible, in whole or in part, into shares of Common Stock of the Company at the option of Pharmsynthez. In addition, in connection with the sale of the Note, Pharmsynthez received certain five year warrants to purchase shares of Common Stock of the Company. Please refer to SEC form 8-K filed on July 8, 2015 for further material information regarding the bridge note financing.

"We are very pleased to have completed this important bridge note financing with a company that has been associated with Xenetic for fifteen years," said Scott Maguire, Chief Executive Officer of Xenetic. "Pharmsynthez is an important collaborator with Xenetic. The partnership covers six drug candidates currently in development in Russia which include indications for cystic fibrosis and obesity with the intention that the Company will eventually seek approval of these drug candidates in the global markets."

"These funds will allow us to continue with our plan to seek a listing of the Company’s shares on a national U.S. securities exchange that offers greater liquidity and will allow Xenetic to trade alongside its peers, therefore allowing greater exposure to the life science investment community. We believe that this financing, which was concluded with an affiliate of our single largest shareholder, reflects a belief in the potential of the Company’s core technologies. Our primary focus remains to improve the human condition by developing and offering safer and more effective treatments via the development of our portfolio of proprietary products. We are looking forward to the future with great confidence."

CEL-SCI IS CLEARED TO START PATIENT ENROLLMENT FOR ITS PHASE 3 CANCER IMMUNOTHERAPY TRIAL IN THAILAND

On June 8, 2015 CEL-SCI Corporation (NYSE MKT: CVM)("CEL SCI" or the "Company") reported that CEL-SCI is now cleared to start patient enrollment in Thailand in its ongoing Phase 3 trial with its investigational cancer immunotherapy Multikine* (Leukocyte Interleukin, Injection) in patients with advanced primary (not yet treated) head and neck cancer (Press release, Cel-Sci, JUL 8, 2015, View Source [SID:1234506548]). Thailand is the 24th country to authorize CEL-SCI’s Phase 3 trial for patient enrollment.

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"We have now completed enrollment of over 50% of the anticipated 880 patients in our Phase 3 trial and continue to expand the trial into more sites to increase the rate of enrollment. In the past month alone, we have added Spain and Italy as participating countries in our trial. We plan to have approximately 100 clinical centers around the world in about 25 countries screening and treating patients," stated CEL-SCI Chief Executive Officer Geert Kersten.

As of May 31, 2015, 463 patients had been enrolled in the global Phase 3 study.

About the Multikine Phase 3 Study

The Multikine Phase 3 study is enrolling patients with advanced primary squamous cell carcinoma of the head and neck. The objective of the study is to demonstrate a statistically significant improvement in the overall survival of enrolled patients who are treated with the Multikine treatment regimen plus standard of care ("SOC") vs. subjects who are treated with SOC only.

About Multikine

Multikine (Leukocyte Interleukin, Injection) is an investigational immunotherapeutic agent that is being tested in an open-label, randomized, controlled, global pivotal Phase 3 clinical trial as a potential first-line (right after diagnosis, before surgery) treatment for advanced primary squamous cell carcinoma of the head and neck. Multikine is designed to be a different type of therapy in the fight against cancer: one that appears to have the potential to work with the body’s natural immune system in the fight against tumors.

Multikine is also being tested in a Phase 1 study under a Cooperative Research and Development Agreement ("CRADA") with the U.S. Naval Medical Center, San Diego, as a potential treatment for peri-anal warts in HIV/HPV co-infected men and women. CEL-SCI has also entered into two co-development agreements with Ergomed Clinical Research Limited to further the development of Multikine for cervical dysplasia/neoplasia in women who are co-infected with HIV and HPV and for peri-anal warts in men and women who are co-infected with HIV and HPV.

Cellectis Reaches Milestone in Servier Collaboration

On July 8, 2015 Cellectis reported the achievement of a significant milestone under the Company’s collaboration agreement with Servier, in the preclinical development of two next-generation product candidates in solid tumors (Press release, Cellectis, JUL 8, 2015, View Source [SID:1234506188]).

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Under the terms of the Company’s collaboration agreement with Servier, Cellectis is eligible to an undisclosed payment.

The collaboration announced in February 2014, is focused on research, development, and potentially commercialization of five product candidates targeting leukemia and solid tumors.

"We are very pleased with the productivity of this alliance enabling us to accelerate our development in the field of solid tumors", commented Mathieu Simon, MD, EVP Chief Operating Officer of Cellectis.

"We believe that immunotherapy will dramatically change the management of metastatic cancers. Our goal at Servier is to make these new technologies available for the largest number of cancer patients", commented Jean-Pierre Abastado, MD, Director Oncology Innovation Therapeutic Pole of Servier.