On April 3, 2018 The European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for cemiplimab for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or patients with locally advanced CSCC who are not candidates for surgery (Press release, Sanofi Genzyme, APR 3, 2018, View Source [SID1234525463]). Advanced CCSC is the deadliest non-melanoma skin cancer. Cemiplimab is an investigational human monoclonal antibody targeting the checkpoint inhibitor PD-1 (programmed cell death protein-1).

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The MAA for cemiplimab is based on a Phase 2 pivotal, single-arm, open-label clinical trial of cemiplimab for advanced CSCC (EMPOWER-CSCC 1) in addition to Phase 1 data from two advanced CSCC expansion cohorts. Both clinical trials enrolled patients with metastatic CSCC and patients with locally advanced CSCC who were not candidates for surgery. Topline results from EMPOWER-CSCC 1 were previously announced in December 2017, and Phase 1 expansion cohort results were presented at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Updated results from both clinical trials are being submitted for presentation at upcoming medical congresses.

Cemiplimab is being jointly developed by Sanofi and Regeneron under a global collaboration agreement.

Cemiplimab is currently under clinical development, and its safety and efficacy have not been fully evaluated by any regulatory authority.

About CSCC

Cutaneous squamous cell carcinoma (CSCC) is one of the most common cancers worldwide, with the number of newly diagnosed cases expected to rise annually. Although CSCC has a good prognosis when caught early, the cancer can prove especially difficult to treat effectively when it is advanced, and patients can experience reduced quality of life due to the impact of the disease as it progresses. Advanced CSCC is the deadliest non-melanoma skin cancer, and there are no EMA-approved treatments for advanced CSCC.

About Regeneron Pharmaceuticals, Inc.

Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to six FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye disease, heart disease, allergic and inflammatory diseases, pain, cancer, infectious diseases and rare diseases.

Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite technologies, such as VelocImmune which produces optimized fully-human antibodies, and ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.

For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitte

On April 3, 2018 Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted treatments for cancer, reported that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. P150207US03, entitled "Alkylphosphocholine analogs for multiple myeloma imaging and therapy (Press release, Cellectar Biosciences, APR 3, 2018, View Source [SID1234525803])." The claims in this patent cover a method of use for CLR 131, the company’s proprietary lead Phospholipid Drug Conjugate (PDC) in multiple myeloma (MM). Cellectar and the Wisconsin Alumni Research Foundation (WARF) are joint owners of the patent and Cellectar has licensed exclusive rights to it from WARF.

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"The issuance of this patent and its associated claims expands the protection for CLR 131 in our most advanced indication, multiple myeloma," stated Jim Caruso, chief executive officer of Cellectar Biosciences. "Multiple myeloma remains an incurable disease with reduced survival benefits seen for patients with each successive line of therapy. We hope to demonstrate that CLR 131’s unique mechanism of action could enable extended survival among patients in this population, even those in later lines of treatment."

About CLR 131

CLR 131 is Cellectar’s investigational PDC under development for several orphan- designated cancers. CLR 131 utilizes the company’s patented phospholipid ether tumor targeting delivery platform to deliver the cytotoxic radioisotope iodine-131 directly to tumor cells. CLR 131 is currently being evaluated in a Phase 2 clinical trial for relapsed or refractory MM and select relapsed or refractory lymphomas as well as a Phase 1b dose escalation clinical trial in patients with relapsed or refractory MM. The U.S. Food and Drug Administration has granted orphan drug designation for CLR 131 in the treatment of MM and pediatric neuroblastoma.

About Phospholipid Drug Conjugates

Cellectar’s product candidates are built upon a patented delivery and retention platform that utilizes optimized PDCs to target cancer cells. The PDC platform is used to selectively deliver diverse oncologic payloads to cancerous cells and cancer stem cells, including hematologic cancers and solid tumors. This selective delivery allows a payloads’ therapeutic window to be modified, which may maintain or enhance drug potency while reducing the number and severity of adverse events. This platform takes advantage of a metabolic pathway utilized by all tumor cell types in all cell cycle stages. Compared with other targeted delivery platforms, the PDC platform’s mechanism of entry does not rely upon specific cell surface epitopes or antigens. In addition, PDCs can be conjugated to molecules in numerous ways, thereby increasing the types of molecules selectively delivered. Cellectar believes the PDC platform holds potential for the discovery and development of the next generation of cancer-targeting agents.

On April 3, 2018 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, reported that it will present new data on Arginase 1 Deficiency patients at the 2018 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics (ACMG) in Charlotte, North Carolina on Thursday, April 12 (Press release, Aeglea BioTherapeutics, APR 3, 2018, View Source [SID1234525152]). The new data will include additional clinical insights on short-term treatment with repeat doses of pegzilarginase, including baseline standardized assessments of neuromotor function. The Company will conduct a clinical update conference call at 8:30 a.m. ET on April 12.

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Title: Weekly Pegzilarginase Produces Marked and Sustained Reductions in Guanidino Compounds in Adults with Arginase 1 Deficiency: Early Phase 1/2 Results

Presenting Author: Roberto Zori, M.D., University of Florida, Gainesville, FL

Poster Number: 803

Presentation Date and Time: Thursday, April 12, 10:00 a.m. to 11:30 a.m. ET

An electronic version of the presentation will be available for download from the Presentations & Events section of the Company’s investor relations website beginning on the day of the their presentation at the 2018 ACMG Annual Clinical Genetics Meeting.

Conference Call & Webcast Details
Aeglea will hold a clinical update conference call on Thursday, April 12, 2018 at 8:30 a.m. ET. To access the live conference call via phone, please dial 1-877-709-8155 (toll free) within the United States, or 1-201-689-8881 internationally. A replay of the call will be available through April 19, 2018 by dialing 1-877-660-6853 within the United States or 1-201-612-7415 internationally. The conference ID is 13678293.

To access the live and archived webcast of the presentation, please visit the Presentations & Events section of the Aeglea BioTherapeutics investor relations website. Please connect to the website at least 15 minutes prior to the presentation to allow for any software download that may be necessary.

About Pegzilarginase (AEB1102) in Arginase 1 Deficiency
Pegzilarginase is an enhanced human arginase that enzymatically degrades the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a debilitating urea cycle disorder caused by deficiency of a key arginine metabolizing enzyme that leads to severe and progressive hyperargininemia-related neurological abnormalities, hyperammonemia and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. The Company’s Phase 1 data demonstrated that pegzilarginase reduced blood arginine levels into the normal range, supporting its mechanism of action.

On April 3, 2018 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported that William G. Rice, Ph.D., Chairman, President and Chief Executive Officer, and Gregory K. Chow, Senior Vice President and Chief Financial Officer, will participate at the H.C. Wainwright Annual Global Life Sciences Conference in Monte Carlo, Monaco on Monday, April 9, 2018 at 11:30 a.m. CEST (Press release, Aptose Biosciences, APR 3, 2018, View Source [SID1234525154]):

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Time: 11:30 a.m. CEST
Date: Monday, April 9, 2018
Location: Le Meridien Beach Plaza Hotel, Monte Carlo, Monaco
Live webcast: View Source
The audio webcasts can also be accessed through the Aptose website at www.aptose.com and will be archived shortly after the live event and available for 90 days.

On April 2, 2018 SELLAS Life Sciences Group Inc., (Nasdaq:SLS) (SELLAS), a clinical-stage biopharmaceutical company focused on novel cancer immunotherapies for a broad range of cancer indications, reported positive interim data from the prospective, randomized, single-blinded, controlled Phase 2b independent investigator-sponsored clinical trial (IST) of trastuzumab (Herceptin) +/- nelipepimut-S (NeuVax) in HER2 1+/2+ breast cancer patients in the adjuvant setting to prevent recurrences (Press release, Sellas Life Sciences, APR 2, 2018, View Source [SID1234525120]).

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A pre-specified interim analysis, conducted by an independent Data Safety Monitoring Board (DSMB) of the efficacy and safety data for the study in an overall population of 275 patients as well as the two primary study target patient populations (node-positive and TNBC) after a median follow-up of 19 months, demonstrated a clinically meaningful difference in median disease-free survival (DFS) in favor of the active arm (NeuVax + Herceptin), a primary endpoint of the study, with hazard ratios of 0.67 and 0.61 in the intent to treat (ITT) and modified ITT (mITT) populations (i.e., those who received at least one dose of vaccine or control) as well as a 34.9% and 39.5% reduction in relative risk of recurrence in the active versus control arms in the ITT and mITT populations, respectively.

A clinically meaningful and also statistically significant difference was found between the two arms in the cohort of patients (n= 98) with triple-negative breast cancer (TNBC), with a hazard ratio of 0.26 and a p-value of 0.023 in favor of the NeuVax + Herceptin combination with a 70.4% reduction in relative risk of recurrence in the active arm versus control. Similarly, a clinically meaningful and statistically significant difference was found between the two arms in favor of the combination in the cohort of patients not receiving hormonal therapy (n = 110), with a hazard ratio of 0.24 and a p-value of 0.009 with a 74.1% reduction in relative risk of recurrence in the active arm versus control. This pre-specified interim analysis also showed an adverse event profile with no notable differences between treatment arms. The addition of NeuVax to Herceptin did not result in any additional cardiotoxicity compared to Herceptin alone.

"We are indeed excited about these compelling results and believe NeuVax + Herceptin has the potential to become an important therapeutic option for TNBC patients. The positive NeuVax phase 2b data underscores the innovative science and approach we have taken to investigate this agent’s potential to address this persistent therapeutic challenge. We plan to immediately engage with the FDA and EMA, as per the recommendation of the DSMB, to identify the optimal path forward in this particular patient group, while advancing the drug through a partnership or other strategic collaboration," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "These are indeed unique and exciting clinical data for TNBC patients, and I would like to extend my sincere gratitude to all patients who have participated in this clinical trial, as well as the study teams."

The NeuVax + Herceptin combination was found to be generally well-tolerated. The majority of treatment-emergent adverse events (TEAE) were of mild or moderate (G1/G2) severity and the majority of G3 systemic TEAEs were unrelated to NeuVax. Treatment-related adverse events consisted primarily of manageable local injection site reactions, skin induration, pruritus and fatigue.

Additionally, in the NeuVax + Herceptin arm, in vivo HER2-specific T-cell immune responses (IRs), assessed by delayed type hypersensitivity (DTH) skin testing, showed a time-dependent increase in IR potency compared to the earliest tested datapoint (p=0.000023), while no such increase was observed in the control arm.

Based on the results above, the DSMB has recommended to expeditiously seek regulatory guidance by the FDA for further development of the combination of NeuVax + Herceptin in TNBC, considering the statistically significant benefit of the combination therapy seen in this population with large unmet medical need.

"We are very pleased with these findings, which suggest that NeuVax + Herceptin may provide a clinically meaningful benefit to breast cancer patients with low-to-intermediate HER2-expression, especially given the recent report of the NSABP B-47 trial showing no benefit in these patients with Herceptin alone. Furthermore, our trial has shown a significantly improved disease-free survival in women with TNBC. The favorable findings for this cohort are particularly promising, given the limited treatment options for these patients with high risk of recurrence and death," commented COL (ret) George E. Peoples, MD, FACS, the study director and sponsor-investigator of the IST. "We look forward to presenting these data at an upcoming major medical conference and to supporting SELLAS in the regulatory and developmental pathway for NeuVax."

Herceptin is a registered trademark of Genentech, Inc. and is not a trademark of SELLAS. The manufacturer of this brand is not affiliated with and does not endorse SELLAS or its products.

About the NeuVax + Herceptin study

This Phase 2b trial is a multi-center, randomized, single-blinded, placebo-controlled trial in 275 HER2 1+/2+ breast cancer patients with positive nodes and/or TNBC. The study combines NeuVax and trastuzumab (Herceptin) in the adjuvant setting aiming to prevent recurrence or death. Tumors in these women show low levels of expression of HER2, as measured by immunohistochemistry (IHC), i.e., at a level of either 1+ or 2+ and, hence, these patients are not considered candidates for Herceptin. Patients who are hormone receptor-negative and HER2 1+/2+ by IHC are currently defined as ‘triple-negative’ breast cancer (TNBC) patients. NeuVax (nelipepimut-S) is a potentially first-in-class, HER2-directed cancer immunotherapy and is the immunodominant peptide derived from the extracellular domain of the HER2 protein, a well-established target for therapeutic intervention in breast carcinoma. The nelipepimut-S sequence stimulates specific CD8+ cytotoxic T lymphocytes (CTLs) following binding to specific HLA molecules on antigen presenting cells (APC) and destroy HER2 expressing cancer cells.