On July 9, 2015 Cancer Research UK and Cancer Research Technology (CRT) reported they have reached an agreement with Monopar Therapeutics LLC, an emerging biopharmaceutical company focused on developing orphan oncology compounds*, to take forward Monopar’s experimental antibody treatment HuATN-658 into clinical trials in cancer patients with advanced solid tumours (Press release, Cancer Research Technology, JUL 9, 2015, View Source [SID1234523204]).

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!

HuATN-658 is an antibody that targets the cell surface protein uPAR, which is found in high levels in some of the most deadly cancers.

Under the agreement, Cancer Research UK’s Centre for Drug Development (CDD) will finance and complete preclinical development of HuATN-658 and conduct a Phase 1 clinical trial in cancer patients. Although drug efficacy will be monitored, the trial’s main objective will be to evaluate the safety of the antibody.

The trial will be managed and run by the CDD through the Experimental Cancer Medicine Centre (ECMC) network, a UK-wide initiative funded by Cancer Research UK and the UK’s four Health Departments.

Upon completion of the clinical trial, Monopar has the right to acquire the clinical trial data. If Monopar declines the option, CRT may continue the development and commercialisation of HuATN-658 in exchange for a share to Monopar of any future revenues from the drug.

Dr Chandler D. Robinson, co-founder and CEO of Monopar Therapeutics, said: "While these are still the early days of the company, we have created a very strong team and development strategy.

"HuATN-658, our lead compound, was discovered by our chief scientific officer, Dr Andrew Mazar, one of the world’s experts on the uPAR pathway.

"The development partnership with Cancer Research UK allows Monopar to leverage a strong platform for the clinical advancement of this compound as it brings long established clinical, regulatory, and manufacturing expertise and resources that would be difficult for an emerging company to amass quickly."

Dr Nigel Blackburn, Cancer Research UK’s director of drug development, said, "We’re excited to be working with Monopar Therapeutics to take this promising new treatment a step further on its journey towards the clinic. The collaboration forms part of our Clinical Development Partnerships scheme, which aims to help progress promising treatments from drug company pipelines and bring them into early phase trials sooner. Thanks to this, six new treatments are now in clinical trials that may otherwise never have moved beyond the lab."

On July 9, 2015 Eli Lilly reported the U.S. Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) met today to discuss the data supporting Eli Lilly and Company’s necitumumab in combination with gemcitabine and cisplatin for use in first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) (Press release, Eli Lilly, JUL 9, 2015, View Source [SID:1234506198]).

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!

Squamous NSCLC is a devastating and difficult-to-treat form of lung cancer. The five-year survival rate for patients with metastatic disease is less than five percent.[1] Necitumumab in combination with gemcitabine and cisplatin is the first regimen to show a significant improvement in overall survival over chemotherapy alone, specifically in the first-line setting.

"We are encouraged by the Committee’s constructive discussion on the benefit-risk profile of necitumumab as few advances have been made over the past two decades in the first-line treatment of advanced squamous NSCLC, leaving a significant unmet medical need," said Richard Gaynor, M.D., senior vice president, product development and medical affairs for Lilly Oncology. "We believe necitumumab with gemcitabine and cisplatin represents a meaningful advance in the search for a new first-line treatment option and look forward to working closely with the FDA as they continue their review."

The FDA is expected to make a decision on Lilly’s biologics license application for necitumumab later this year.

About Necitumumab
Necitumumab is a recombinant human IgG1 monoclonal antibody that is designed to block the ligand binding site of the human epidermal growth factor receptor 1 (EGFR). Activation of EGFR has been correlated with malignant progression, induction of angiogenesis and inhibition of apoptosis or cell death.

About Squamous Non-Small Cell Lung Cancer (NSCLC)
NSCLC is the most common type of lung cancer, and accounts for about 85 percent of all lung cancer cases.[2] Squamous NSCLC, which represents about 30 percent of all people affected by NSCLC,[3] is a devastating, difficult-to-treat form of the disease. Patients face an imposing disease and symptom burden with very poor prognosis; the five-year survival rate for patients with metastatic disease is less than five percent.[4] Little progress has been made over the last two decades, particularly in the first-line setting, leaving a significant unmet medical need. In order to address the unique and complex needs of individual patients with advanced squamous NSCLC, more first-line treatment options are needed.

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 (Filing, 8-K, Epizyme, JUL 9, 2015, View Source [SID:1234506203]). Under the collaboration, Celgene will have the option to license histone methyltransferase (HMT) inhibitors being developed by Epizyme against three predefined targets.

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!

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.

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.

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!

"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.

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]).

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!

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.