On May 2, 2018 Endocyte, Inc. (Nasdaq:ECYT), a biopharmaceutical company developing targeted therapeutics for personalized cancer treatment, reported that the company will host a conference call on Wednesday, May 9th, at 8:30 a.m. EDT to discuss its first quarter financial results and provide an operational update (Press release, Endocyte, MAY 2, 2018, View Source [SID1234525959]).

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Investors and the general public are invited to listen to a live webcast of the call, which can be accessed in the Investors & News section of the Company’s website at www.endocyte.com or by dialing (877) 845-0711 (U.S./Canada) or (760) 298-5081 (International).

The webcast will be recorded and available on the Company’s website for 90 days following the call.

Website Information
Endocyte routinely posts important information for investors on its website, www.endocyte.com, in the "Investors & News" section. Endocyte uses this website as a means of disclosing material information in compliance with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the "Investors & News" section of Endocyte’s website, in addition to following its press releases, SEC filings, public conference calls, presentations and webcasts. The information contained on, or that may be accessed through, Endocyte’s website is not incorporated by reference into, and is not a part of, this document..

Investors and the general public are invited to listen to a live webcast of the call, which can be accessed in the Investors & News section of the Company’s website at www.endocyte.com or by dialing (877) 845-0711 (U.S./Canada) or (760) 298-5081 (International).

The webcast will be recorded and available on the Company’s website for 90 days following the call.

Website Information
Endocyte routinely posts important information for investors on its website, www.endocyte.com, in the "Investors & News" section. Endocyte uses this website as a means of disclosing material information in compliance with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the "Investors & News" section of Endocyte’s website, in addition to following its press releases, SEC filings, public conference calls, presentations and webcasts. The information contained on, or that may be accessed through, Endocyte’s website is not incorporated by reference into, and is not a part of, this document.

On May 2, 2018 Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported that the Company has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) requesting approval to initiate a Phase 1 clinical trial for UCART22, Cellectis’ second wholly controlled TALEN gene-edited product candidate, for the treatment of B-cell acute lymphoblastic leukemia (B-ALL) in adult patients (Press release, Cellectis, MAY 2, 2018, View Source;utm_medium=feed&utm_campaign=Feed%3A+cellectis+%28Cellectis+RSS+Feed%29#When:20:30:00Z [SID1234525958]).

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Pending regulatory clearance, Cellectis plans to initiate a Phase I clinical trial in the third quarter of 2018. The clinical research will be led by Dr. Nitin Jain, Assistant Professor, and Prof. Hagop Kantarjian, Chairman in the Department of Leukemia and University Chair in Cancer Medicine at The University of Texas MD Anderson Cancer Center in Houston.

"This IND application for UCART22 is an important regulatory milestone for the Company," said Stephan Reynier, Chief Regulatory and Compliance Officer, Cellectis. "The first ever FDA approval for a CAR T-cell therapy, directed against CD19, for pediatric and young patients with R/R B-ALL occurred in 2017. However, further CART approaches are needed, as some limitations of the CD19-CART treatment appear to be due to the expansion of CD19-negative leukemia clones.[1] The UCART22 product candidate will be evaluated in relapsed or refractory CD22 B-ALL, including relapses after CD19 CAR-T administration."

Acute lymphoblastic leukemia (ALL) is a rapidly progressing form of leukemia that is characterized by the presence of a large number of immature white blood cells in the blood and bone marrow. In 2016, an estimated 6,590 new cases were diagnosed in the U.S., with over 1,400 deaths due to ALL.[2] Approximately 85 percent of ALL cases involve precursor B-cells (B-ALL).

UCART22 is an allogeneic, off-the-shelf gene-edited T-cell product candidate designed for the treatment of B-ALL. Like CD19, CD22 is a cell surface antigen expressed from the pre B-cell stage of development through mature B-cells and CD22 expression occurs in more than 90 percent of patients with B-ALL.[3]

"Given the high unmet medical need for patients who suffer from B-ALL, filing the IND is the first vital step to potentially creating a treatment to be manufactured on an industrial scale, allowing these patients to get the help that they need much faster," added Prof. Stéphane Depil, Senior Vice President Research & Development and Chief Medical Officer. "We are dedicated to making this a reality as soon as possible and look forward to hitting the ground running with the clinical trial once we obtain regulatory clearance."

The manufacturing process of Cellectis’ allogeneic CAR T-cell product line, Universal CARTs or UCARTs, yields frozen, off-the-shelf, non-alloreactive engineered CAR T-cells. UCARTs are meant to be readily available CAR T-cells for a large patient population. Their production is industrialized with defined pharmaceutical release criteria.

On May 2, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation (RPDD) to the company’s lead phospholipid drug conjugate, CLR 131, for the treatment of neuroblastoma (Press release, Cellectar Biosciences, MAY 2, 2018, View Source [SID1234525957]).

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"Neuroblastoma is a devastating cancer most often found in infants and young children. The grant of RPDD for CLR 131 in conjunction with the orphan drug designation we received in March highlight the critical need for new treatments in the fight against this disease," said John Friend, M.D., chief medical officer of Cellectar. "We look forward to working with the FDA to bring this potential therapy to pediatric patients and expect to begin a clinical study in neuroblastoma during the second half of 2018."

The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children from birth to 18 years old, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. If CLR 131 is approved by the FDA for neuroblastoma, the rare pediatric disease designation may enable Cellectar to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive Priority Review for a future NDA or BLA submission which would reduce the FDA review time from twelve months to six months. Currently, these vouchers can also be transferred or sold to another entity. Over the last 16 months, five priority review vouchers were sold for between $110 million to $150 million each.

About Neuroblastoma

Neuroblastoma, a neoplasm of the sympathetic nervous system, is the most common extracranial solid tumor of childhood, accounting for approximately 7.8% of childhood cancers in the United States and is recognized by the FDA as an orphan disease. The incidence is about 10.54 cases per 1 million per year in children younger than 15 years and 90% are younger than 5 years at diagnosis. Approximately 50% of patients present with metastatic disease requiring systemic treatment. Although the prognosis is favorable in children under one year of age with an 86% to 95% 5-year survival, in children aged one to 14 years the 5-year survival ranges from 34% to 68%.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131, is in a Phase 2 clinical study in relapsed or refractory (R/R) MM and a range of B-cell malignancies and a Phase 1 clinical study in patients with (R/R) MM exploring fractionated dosing. In the second half of 2018 the company plans to initiate a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma, as well as a Phase 1 study in combination with external beam radiation for head and neck cancer.

On May 2, 2018 bluebird bio, Inc. (Nasdaq: BLUE) reported financial results and business highlights for the first quarter ended March 31, 2018 (Press release, bluebird bio, MAY 2, 2018, View Source [SID1234525956]).

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"Early in 2018, we’ve made important progress against our stated regulatory and commercial goals for the year, including preparing and investing in our team and infrastructure to bring us closer to providing our therapies to patients," said Nick Leschly, chief bluebird. "Our plans remain on-track for our first regulatory filing in Europe for TDT, we are rapidly advancing development of bb2121 for patients, and we are focused on future innovation platforms."

Recent Highlights

COMPLETED NORTHSTAR (HGB-204) STUDY – In February 2018, the final patient to be treated in Northstar, the company’s Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin for the treatment of patients with TDT, reached two years of follow-up. The study, along with data from the HGB-205 study, and available data from the Northstar-2 Study (HGB-207), will form the basis of the European Marketing Authorization Application (MAA) submission, which is planned for the second half of 2018.
ENTERED INTO AGREEMENT WITH CELGENE TO CO-DEVELOP AND CO-PROMOTE bb2121 – In March 2018, bluebird and Celgene Corporation (Celgene) announced that bluebird has exercised its option to co-develop and co-promote bb2121, an investigational anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell therapy for the potential treatment of patients with relapsed/refractory multiple myeloma in the United States. Under the terms of the agreement, bluebird and Celgene have joint responsibility for development, manufacturing and commercialization in the United States. Celgene will assume sole responsibility for drug product manufacturing and commercialization outside the United States.
PUBLISHED LENTIGLOBIN TDT DATA IN NEJM – In April 2018, interim data was published in the New England Journal of Medicine (NEJM) from two separate two-year clinical studies investigating the potential for LentiGlobin gene therapy to eliminate or reduce chronic blood transfusions in patients with TDT. The interim results from the Northstar (HGB-204) and HGB-205 studies showed that a majority of the 22 patients in the two Phase 1/2 studies followed for two years or longer remained free from transfusions.
PRESENTED PRE-CLINICAL GENE EDITING RESEARCH AT AACR (Free AACR Whitepaper) – In April 2018, bluebird researchers presented a poster at the American Academy of Cancer Research entitled "Enhancing CAR T cell activity by simultaneous checkpoint gene knock-out and PDCD1 promoter driven IL-12 expression." The poster outlined the development of an IL-12 delivery system driven by the PDCD1 promoter whose expression depends on tumor specific CAR T activation, and concluded that the approach could improve safety and pharmacokinetics of IL-12, as well as reduce T cell exhaustion to enhance CAR T functions.
Upcoming Anticipated Milestones

TDT
Filing for European approval of LentiGlobin in patients with TDT and non-β0/β0 genotypes in the second half of 2018
Submission of LentiGlobin clinical data from the Northstar-2 (HGB-207) clinical study in patients with TDT and non- β0/β0 genotypes to the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting and at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting
Submission of LentiGlobin clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and the β0/β0 genotype to the ASH (Free ASH Whitepaper) Annual Meeting
SCD
Update on the clinical development plan and registration strategy for LentiGlobin in SCD by year end 2018
Initiation of an investigator-led Phase 1 clinical study of a lentiviral gene therapy targeting BCL11a suppression and fetal hemoglobin upregulation in patients with SCD
Submission of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD to the ASH (Free ASH Whitepaper) Annual Meeting
Multiple Myeloma
Submission of bb2121 clinical data from the CRB-401 study to the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting
Submission of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma to the ASH (Free ASH Whitepaper) Annual Meeting
Initiation by Celgene of a Phase 3 clinical study of bb2121 in third line multiple myeloma
CALD
Presentation of Lenti-D clinical data from the ongoing Starbeam clinical study in patients with CALD in the second half of 2018
First Quarter 2018 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2018 and December 31, 2017 were $1.57 billion and $1.61 billion, respectively.
Revenues: Total revenues were $16.0 million for the first quarter of 2018 compared to $6.8 million for first quarter of 2017. Effective January 1, 2018, bluebird adopted Accounting Standards Codification, Topic 606, Revenue from Contracts with Customers ("Topic 606"), using the modified retrospective transition method. The increase was primarily attributable to the adoption of Topic 606 and, to a lesser extent, increased manufacturing services under the company’s agreement with Celgene. Total revenues under the previous revenue recognition standard would have been $9.8 million for the first quarter of 2018.
R&D Expenses: Research and development expenses were $97.1 million for the first quarter of 2018 compared to $55.0 million for the first quarter of 2017. The increase in research and development expenses was driven by costs incurred to advance and expand the company’s pipeline and is attributable to increased clinical trial-related costs and manufacturing costs for our development programs, as well as increased employee-related costs due to headcount growth supporting overall research and development activities, and increased license milestones and fees under the company’s strategic collaboration and license agreements.
G&A Expenses: General and administrative expenses were $34.9 million for the first quarter of 2018 compared to $20.3 million for the first quarter of 2017. The increase in general and administrative expenses was attributable to increases in employee-related costs due to increased headcount to support overall growth, commercial-readiness activities, and professional and consulting fees.
Net Loss: Net loss was $115.1 million for the first quarter of 2018 compared to $68.7 million for the first quarter of 2017.

On May 2, 2018 Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, reported that it has entered into a definitive agreement under which it will acquire BeneVir Biopharm, Inc. (BeneVir), a privately-held, biopharmaceutical company specializing in the development of oncolytic immunotherapies (Press release, Janssen Pharmaceutica, MAY 2, 2018, View Source [SID1234525955]). BeneVir utilizes a proprietary T-Stealth Oncolytic Virus Platform to engineer oncolytic viruses, tailored to infect and destroy cancer cells. Johnson & Johnson Innovation LLC facilitated the transaction.

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"Oncolytic viral immunotherapy holds exciting potential in the treatment of solid tumors through the priming and augmenting of an anti-tumor immune response," said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. "BeneVir’s unique technology platform complements our immuno-oncology research, which is focused on bringing forward an array of novel immunotherapies and combinations that may improve treatment outcomes for patients."

BeneVir engineers oncolytic viruses through the T-Stealth platform to overcome the barrier of the body’s immune system. Janssen intends to advance pre-clinical candidates as standalone therapies and in combination with other immunotherapies for the treatment of solid tumor cancers (e.g., lung, prostate, colorectal, etc.).

"We are delighted to add the scientific caliber of the BeneVir team and their oncolytic immunotherapy platform to Janssen’s robust immuno-oncology efforts," said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, LLC. "We are committed to pursue transformational science from our own laboratories and those of others, as we continue to advance our focus on treating some of the world’s most devastating diseases."

BeneVir will maintain a research presence in Rockville, Maryland and become part of the Janssen Oncology Therapeutic Area. The team will remain focused on the optimization of next generation T-Stealth oncolytic viruses in solid tumors and the execution of pre-clinical activities.

The closing of the transaction is subject to customary closing conditions, including clearance under the Hart-Scott-Rodino Antitrust Improvements Act, and is expected to close in the second quarter of 2018.