On September 8, 2017 Sanofi and Regeneron Pharmaceuticals, Inc. reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation status to cemiplimab (REGN2810) for the treatment of adults with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC, the second deadliest skin cancer after melanoma[i], Anchor[ii] (Press release, Sanofi, SEP 8, 2017, View Source [SID1234520415]). Cemiplimab is an investigational human, monoclonal antibody targeting PD-1.
Sanofi and Regeneron previously reported positive, preliminary results for cemiplimab from two expansion cohorts involving 26 advanced CSCC patients in a Phase 1 study of nearly 400 patients, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2017. EMPOWER-CSCC 1, a Phase 2, potentially pivotal, single-arm, open label clinical trial of cemiplimab is currently enrolling patients for metastatic CSCC and locally advanced and unresectable CSCC. Cemiplimab is being jointly developed by Sanofi and Regeneron under a global collaboration agreement. Pending data results, the companies anticipate submitting a biologics license application for cemiplimab with the FDA in the first quarter of 2018.
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.[iii] CSCC is responsible for the most deaths among non-melanoma skin cancer patients.ii
Breakthrough Therapy designation serves to expedite the development and review of drugs that target serious or life-threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy. The designation includes all of the Fast Track program features, as well as more intensive FDA guidance and discussion. The Breakthrough Therapy designation is distinct from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.[iv]
Cemiplimab is currently under clinical development, and its safety and efficacy has not been fully evaluated by any regulatory authority.

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SurVaxM is a first-of-its-kind, patented peptide mimic immunotherapeutic vaccine (immunotherapy) that targets survivin, a cell-survival protein present in 95 percent of glioblastomas and many other cancers. It is engineered to recognize survivin-expressing cancer cells as foreign and stimulate patients’ own immune response to control tumor growth and recurrence.

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While vaccines are typically thought of as ways to prevent diseases, vaccines can also be used in a therapeutic mode (e.g., to treat cancer). SurVaxM is delivered through simple subcutaneous injection.
Malignant Glioma (GBM):

SurVaxM demonstrated safety and tolerability in a Phase I study in patients with recurrent or progressive malignant glioma (brain tumors). View press release

SurVaxM has now entered a Phase II clinical trial in adults with newly diagnosed glioblastoma. at leading cancer centers: Roswell Park Cancer Institute, Cleveland Clinic and Dana-Farber Cancer Institute. Please visit the Clinical Trials page for more information.

Although SurVaxM was first tested in brain cancer, survivin is present in most cancers, including multiple myeloma, melanoma, ovarian, renal, lymphoma, prostate and breast cancers. Thus SurVaxM could have broad applicability to these cancers.
Multiple Myeloma:

A Phase I clinical trial evaluating SurVaxM in combination with REVLIMID(lenalidomide) as a maintenance therapy for adults with multiple myeloma has been initiated with support from Celgene (marketer of REVLIMID). Please visit the Clinical Trials page for more information.

MimiVax has an exclusive license to globally commercialize SurVaxM, as well as an extensive worldwide patent portfolio for SurVaxM and other products in development.

On September 8, 2017 CytomX Therapeutics, Inc. (Nasdaq:CTMX), a biopharmaceutical company developing investigational Probody therapeutics for the treatment of cancer, reported upcoming Trials in Progress poster presentations for its two lead product candidates, CX-072, a PD-L1 targeting Probody therapeutic and CX-2009, a Probody-drug conjugate targeting CD166, at the European Society for Medical Oncology Annual Meeting from September 8-12 in Madrid, Spain (Press release, CytomX Therapeutics, SEP 8, 2017, View Source;p=RssLanding&cat=news&id=2299537 [SID1234520427]).

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Abstract Information:

Title: The First-in-Human, Dose-Finding PROCLAIM-CX-072 Trial to Assess the Antitumor Activity and Tolerability of the Probody
Therapeutic CX-072 as Monotherapy and in Combination With Ipilimumab or Vemurafenib in Solid Advanced Tumors and Lymphomas
Author: Valentina Boni, M.D., Ph.D., START Madrid-CIOCC, Madrid, Spain
Date: Monday, September 11, 2017
Time: 1:15 p.m. – 2:15 p.m.
Location: Hall 8
Abstract: 423TiP
About the PROCLAIM-CX-072 Trial:

The PROCLAIM-CX-072 (PRObody CLinical Assessment In Man) study is designed to evaluate tolerability, and preliminary antitumor activity and translational biomarkers of multiple doses of CX-072 as monotherapy or as combination therapy with ipilimumab or vemurafenib in patients with advanced, unresectable solid tumors or lymphoma. This first-in-human, open-label, multicenter, dose-escalation, phase 1/2 study of CX-072 includes 4 dose-escalation groups (monotherapy, Part A; 2 ipilimumab combination schedules, Parts B1 and B2; vemurafenib combination, Part C), a stage testing biomarkers and efficacy in PD-L1+ tumors (Part A2), and a dose-expansion phase (Part D).

Abstract Information:

Title: PROCLAIM-CX-2009: A First-in-Human Trial to Evaluate CX-2009 in Adults With Metastatic or Locally Advanced Unresectable Solid Tumors
Author: Javier Garcia-Corbacho M.D., Hospital Clinic Barcelona, Barcelona, Spain
Date: Monday, September 11, 2017
Time: 1:15 p.m. – 2:15 p.m.
Location: Hall 8
Abstract: 422TiP
About the PROCLAIM-CX-2009 Trial:

The objectives of the ongoing PROCLAIM-CX-2009 (PRObody CLinical Assessment In Man) module are to determine the maximum tolerated dose (MTD), recommended phase 2 dose (RP2D), dose-limiting toxicities, and preliminary antitumor activity of CX-2009 as monotherapy in the following 7 selected tumor types with high CD166 expression: breast carcinoma, castration-resistant prostate carcinoma (CRPC), cholangiocarcinoma, endometrial carcinoma, epithelial ovarian carcinoma, head and neck squamous cell carcinoma (HNSCC), and non–small cell lung carcinoma (NSCLC).

CellCeutix has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, CellCeutix, SEP 8, 2017, View Source [SID1234520459]).

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On September 7, 2017 AstraZeneca and MedImmune, its global biologics research and development arm, reported that they have been informed by partner Celgene that the US Food and Drug Administration (FDA) has placed a partial clinical hold on five trials and a full clinical hold on one trial in the Celgene FUSION programme (Press release, AstraZeneca, SEP 7, 2017, View Source [SID1234520401]). The trials are testing Imfinzi (durvalumab), an anti-PD-L1 agent, in combination with immunomodulatory agents, with or without chemotherapy, in blood cancers such as multiple myeloma, chronic lymphocytic leukaemia and lymphoma.

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The decision by the FDA was based on risks identified in other trials for an anti-PD-1 agent, pembrolizumab, in patients with multiple myeloma in combination with immunomodulatory agents. No imbalance has been observed in the FUSION programme; however, the clinical holds allow for additional information to be collected to further understand the risk benefit profile of the programme. The FDA has taken similar action with other combination trials in patients with multiple myeloma.

Patients enrolled in the trials on partial clinical hold who are receiving clinical benefit from treatment may remain on treatment. Patients enrolled in the trial on full clinical hold will be discontinued from treatment. No new patients will be enrolled into the listed trials.

Other trials with Imfinzi in haematological malignancies and other tumour types continue unchanged.

The trials placed on partial clinical hold are:

MEDI4736-MM-001: A Phase Ib multicenter, open-label study to determine the recommended dose and regimen of durvalumab either as monotherapy or in combination with pomalidomide with or without low-dose dexamethasone in patients with relapsed and refractory multiple myeloma
MEDI4736-MM-003: A Phase II, multicenter, open-label study to determine the safety and efficacy for the combination of durvalumab and daratumumab in patients with relapsed and refractory multiple myeloma
MEDI4736-MM-005: A Phase II, multicenter, single-arm study to determine the efficacy for the combination of durvalumab plus daratumumab in patients with relapsed and refractory multiple myeloma that have progressed while on current treatment regimen containing daratumumab
MEDI4736-NHL-001: A Phase I/II, open-label, multi-center study to assess the safety and tolerability of durvalumab as monotherapy and in combination therapy in subjects with lymphoma or chronic lymphocytic leukaemia. The only arm in this trial for which enrolment is suspended is the arm with the durvalumab, REVLIMID and rituximab combination
MEDI4736-DLBCL-001: A Phase II, open-label, multicenter study to evaluate the safety and clinical activity of durvalumab in combination with rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone (R-CHOP) or with lenalidomide plus R-CHOP (R2 CHOP) in patients with previously untreated, high risk diffuse large B Cell lymphoma
The trial placed on full clinical hold is:

MEDI4736-MM-002: A Phase Ib multicenter, open-label study to determine the recommended dose and regimen of durvalumab in combination with lenalidomide with and without low-dose dexamethasone in subjects with newly diagnosed multiple myeloma
The trials that will continue to enrol are:

MEDI4736-MDS-001: A randomised, multicenter, open-label, Phase II trial evaluating the efficacy and safety of azacitidine subcutaneous in combination with durvalumab in previously untreated subjects with higher-risk myelodysplastic syndromes or in elderly (>= 65 Years) acute myeloid leukaemia subjects not eligible for haematopoietic stem cell transplantation
CC-486-MDS-006: A Phase II, international, multicenter, randomised, open-label, parallel group to evaluate the efficacy and safety of CC-486 alone in combination with durvalumab in subjects with myelodysplastic syndromes who fail to achieve an objective response to treatment with azacitidine for injection or decitabine
In April 2015, Celgene entered into a strategic collaboration with MedImmune to develop and commercialise durvalumab for haematologic malignancies. The use of durvalumab in combination with other agents for the treatment of patients with haematologic malignancies is not approved by the FDA, and the safety and efficacy of those combinations have not been established.