On December 13, 2017 Eleven Biotherapeutics, Inc. (NASDAQ:EBIO), a late-stage clinical oncology company advancing novel product candidates based on its Targeted Protein Therapeutics (TPTs) platform, reported that Dr. Gregory Adams, Chief Scientific Officer, will chair a session focused on antibody drug conjugates and fusion proteins at the Antibody Engineering and Therapeutics Meeting in San Diego, CA (Press release, Eleven Biotherapeutics, DEC 13, 2017, View Source [SID1234522620]). As part of the session, Dr. Adams will give a talk on the potential for tumor-targeted payloads to prime the immune system to facilitate more effective therapy in combination with immuno-oncology agents, including checkpoint inhibitors.

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"Checkpoint inhibitors are a significant step forward in the quest to engage the patient’s immune system in the fight against cancer. Checkpoint inhibitors function by blocking the signals that cancers use to inhibit the immune system. However, for checkpoint inhibitors to be effective, they require the presence of an active immune response against the cancer. There is broad recognition of the potential for tumor-targeted payloads, such as Eleven’s Targeted Protein Therapeutics (TPTs), to provide this spark to the immune system and act synergistically with checkpoint inhibitors," said Dr. Gregory Adams, Chief Scientific Officer of Eleven Biotherapeutics. "Earlier this year at the American Association of Cancer Research Conference, we presented data which supports this potential, demonstrating that TPTs induce immunogenic cell death as evidenced by the presence of damage-associated molecular patterns. Our collaboration with the National Cancer Institute and AstraZeneca aims to build on this evidence."

Session Details

Title: Antibody-Drug Conjugates & Fusion Proteins
Date: Wednesday, December 13, 2017
Time: 8:10 am – 12:00 pm PT
Location: Manchester Grand Hyatt; San Diego, CA

About Vicinium

Vicinium is manufactured as a single protein anti-epithelial cell adhesion molecule (anti-EpCAM) fusion protein fused with Pseudomonas Exotoxin A (ETA) designed to specifically target and deliver a potent anti-cancer payload directly into tumor cells. It is constructed with a stable, genetically-engineered linker to ensure its potent protein payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues thereby improving the compound’s safety. Vicinium’s one-step manufacturing process offers significant cost advantages and results in the production of a homogenous product, with less batch-to-batch variability than most antibody drug conjugates. Vicinium is currently in a Phase 3 registration clinical trial for the treatment of high-grade non-muscle invasive bladder cancer (NMIBC) in patients who have previously received two courses of Bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Eleven Biotherapeutics intends to enroll 134 subjects in the trial, including 77 subjects with carcinoma in situ (CIS), at over 70 centers in the United States and Canada. Primary and secondary endpoints include complete response (CR) in CIS subjects, time to disease recurrence and event free survival. The Company expects to complete patient enrollment in the first quarter of 2018 and to report topline three-month data in mid-2018.

On December 13, 2017 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi reported positive topline results from a pivotal Phase 2 clinical study of cemiplimab in 82 patients with advanced cutaneous squamous cell carcinoma (CSCC), the second deadliest skin cancer after melanoma (Press release, Regeneron, DEC 13, 2017, View Source [SID1234522608]). Cemiplimab, an investigational human antibody targeting PD-1 (programmed cell death protein 1), demonstrated an overall response rate (ORR) of 46.3%, as determined by independent review. The median duration of response (DOR) had not yet been reached at the data cut-off point (32 of 38 responses are ongoing). At the time of this analysis, all patients had a minimum follow up of 6 months. The safety profile in the study was generally consistent with approved anti-PD-1 agents.

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These pivotal data will form the basis of a rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA), which has been initiated and is expected to be completed in the first quarter of 2018. A rolling BLA submission allows for portions of the regulatory application to be submitted to the FDA as they are completed. A submission to the European Medicines Agency (EMA) is also expected to be completed in the first quarter of 2018. These data confirm the positive Phase 1 clinical trial expansion cohort results reported at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, which led to a Breakthrough Therapy Designation for cemiplimab in advanced CSCC in September 2017.

"For patients with CSCC that cannot be cured by surgery or radiation, there are no FDA-approved treatment options, and advanced CSCC is responsible for 3,900 to 8,800 deaths per year in the U.S.," said Israel Lowy, MD, PhD, Vice President of Global Clinical Development and Head of Translational Science and Clinical Oncology, Regeneron. "This is the largest prospective study ever conducted in this disease, and we are pleased that many patients were able to achieve deep and durable responses with cemiplimab monotherapy. The high and durable response rates seen in this study are particularly notable given that the study enrolled patients regardless of biomarker status."

The efficacy data reported today include results from 82 patients in the Phase 2 EMPOWER-CSCC 1 study. Approximately two-thirds of patients had progressed after prior systemic chemotherapy or radiation.

"EMPOWER-CSCC 1 was initiated in 2016 and has enrolled rapidly, underscoring the serious unmet need in advanced CSCC," said Elias Zerhouni, MD, President, Global R&D, Sanofi. "We look forward to working with regulatory agencies globally to bring this important therapy to advanced CSCC patients as quickly as possible. We continue to rapidly advance a broad development program to evaluate cemiplimab both as monotherapy and combination across a number of solid tumor and blood cancers."

EMPOWER-CSCC 1 is a single-arm, open-label clinical trial and remains active. Enrollment is complete in the study arm of patients with metastatic CSCC receiving a 3 mg/kg dose of cemiplimab every two weeks. Enrollment continues in the remaining two study arms of patients with metastatic CSCC receiving a 350 mg flat dose of cemiplimab every three weeks and patients with locally advanced and unresectable CSCC receiving a 3 mg/kg dose of cemiplimab every two weeks.

Updated results from both the EMPOWER-CSCC 1 and the Phase 1 clinical trial will be submitted for presentation at a 2018 medical congress.

Cemiplimab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement, and was invented by Regeneron using the company’s proprietary VelocImmune technology that yields optimized fully-human antibodies. Cemiplimab is currently under clinical development, and its safety and efficacy has not been fully evaluated by any regulatory authority.

About Cutaneous Squamous Cell Carcinoma (CSCC)
CSCC is the second most common type of skin cancer in the United States. Although CSCC has a good prognosis when caught early, it can prove especially difficult to treat when it progresses to advanced stages. Patients at this stage can be disfigured due to multiple surgeries to remove CSCC tumors on the head, neck and other parts of the body. CSCC is the second deadliest skin cancer after melanoma and is responsible for the most deaths among non-melanoma skin cancer patients.

On Decmber 13, 2017 Stemline Therapeutics presented 2017 ASH (Free ASH Whitepaper) Conference Presentation (Presentation, Stemline Therapeutics, DEC 13, 2017, View Source [SID1234522636]).

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On December 13, 2017 MolMed S.p.A. (MLM.MI) reported that it obtained its first national marketing authorization for its proprietary product, Zalmoxis: the Board of Directors of AIFA (Agenzia Italiana del Farmaco) approved the agreement negotiated between AIFA’s Prices and Reimbursement Committee (CPR) and MolMed, in which the price and reimbursement for Zalmoxis medicinal product were defined (Press release, MolMed, DEC 13, 2017, View Source [SID1234595072]).

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Zalmoxis is MolMed’s first patient specific cell therapy product, based on genetically engineering of the immune system, administered following haploidentical haematopoietic stem-cell transplantation (HSCT) from partially compatible donors to adult patients with leukaemia and other high-risk haematologic malignancies. Zalmoxis is administered from the 21st day post-transplantation and foresees up to a maximum of 4 infusions per patient based on the achievement of immune-reconstitution.

The terms of the agreement provide for an ex-factory price, excluding VAT, of 149,000 EUR per infusion, gross of discounts foreseen by law and of selective reductions foreseen by AIFA Resolutions ("Determinazioni") of 3 July 2006 and 27 September 2006. Furthermore the agreement also set a flat fee per patient and a safeguard clause on sales for the first 24 months. Given the nature of the product, it will be a hospital-only dispensed therapy.

The agreement signed with AIFA will be effective from the fifteenth day subsequent to its publication in the Gazzetta Ufficiale of the Italian Republic.

Riccardo Palmisano, MolMed’s CEO, commented: "This is truly a turning point for a research and development company like MolMed: both Zalmoxis eligibility for reimbursement and the "Aifa granted price" acknowledge the value of our therapy and, at the same time, the successful completion of a top level Italian research, development and manufacturing journey. In addition, this result paves the way to the marketing of this sophisticated genetically engineered cell therapy. This first national authorization allows us to look with increasing confidence to the introduction of Zalmoxis in the other European countries covered by the Dompé agreement, from which we expect relevant results, considering our partner’s value, expertise and successful track record. Zalmoxis commercialization will allow MolMed to increase its revenues from proprietary products on top of those deriving from GMP development and production for third party."

Professor Claudio Bordignon, Founder and Chairman of MolMed, commented: "The inclusion of Zalmoxis among the medicinal products reimbursable by the National Health Service makes this revolutionary therapy available to those patients for whom the allogeneic stem cell transplantation is the best treatment option for high-risk leukaemia and other blood malignancies. In absence of a fully compatible donor, Zalmoxis makes the treatment from a haploidentical family donor safer, by eliminating post-transplant immunosuppression and, at the same time, resolving the potential occurrence of Graft versus Host Disease. Furthermore, Zalmoxis accelerates immune-reconstitution by protecting the patient against infections and leukaemia relapse. Zalmoxis has proven to significantly increase the one-year survival rate in the treated population."

"We are very pleased with the outcome of MolMed’s negotiation with AIFA. By stipulating this agreement, it is stated that Zalmoxis, the first ex-cell cellular therapy based on the immune system engineering for the PRESS RELEASE 2 treatment of adult patients with high risk blood malignancies, responds to a major clinical need", commented Eugenio Aringhieri, Chief Executive Officer of Dompé. "The synergy of our distinctive competences and shared value priorities, on which this alliance with MolMed is founded, will lead us in the interaction with the scientific community and the regulatory authorities aimed at bringing Zalmoxis to all Patients eligible for this therapy."

About Zalmoxis
Zalmoxis is an innovative therapy based on genetically engineering donor immune system T cells to carry an inducible "suicide gene". Administered to patients following HSCT from partially compatible donors (haploidentical HSCT), these cells foster an anti-leukaemia effect by eliminating post-transplant immunosuppression prophylaxis and inducing a rapid immune reconstitution. The suicide gene allows to readily control Graft versus Host Disease (GvHD), the most significant and serious adverse event in haploidentical transplantation, caused by the genetic disparity between patient and donor. Zalmoxis significantly increases long-term survival, regardless of disease status at transplant, thus making HSCT from partially compatible donors safer and more effective. On August 18th, 2016 the European Commission granted a Conditional Marketing Authorisation (CMA) for Zalmoxis, the first immunogene therapy, as patient-specific adjunctive treatment in haplo-identical haematopoietic stem-cell transplantation for adult patients with leukaemia and high-risk haematological malignancies. Following this authorization, which allows MolMed to market Zalmoxis in the 28 EU Member States and in the European Economic Area, activities aimed at its introduction on the European markets have increased. The marketing authorization issued by AIFA is therefore only the first out of the ones on which MolMed has been working since the date of the European CMA.

With the aim of successfully marketing Zalmoxis, on July 26th, 2017 MolMed signed an exclusive license and distribution agreement with Dompé Farmaceutici S.p.A., one of the leading Italian biopharmaceutical companies, granting Dompé the exclusive right and obligation to conduct all activities aimed at promoting, marketing, exploiting, distributing and selling Zalmoxis in all member countries of the current European Economic Area (EEA) and an option right for Switzerland, Turkey and Australia.

In parallel, preparation of the dossier to obtain price and reimbursement of Zalmoxis from the German and French authorities continued through direct interactions. Furthermore activities aimed at starting negotiations with authorities of other countries have been speeded up. In regard to distribution and marketing beyond the European borders, contracts have already been signed with Megapharm Ltd for Israel and with TTY Biopharm Company Ltd for Taiwan and a number of countries in South-East Asia.

On December 13, 2017 Iovance Biotherapeutics, Inc. presented , Analyst Presentation-December 2017 (Presentation, Iovance Biotherapeutics, DEC 13, 2017, View Source [SID1234522633]).

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