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US FDA accepts Biologics License Application for moxetumomab pasudotox in hairy cell leukaemia

On April 3, 2018 AstraZeneca and MedImmune, its global biologics research and development arm, reported that the US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for moxetumomab pasudotox, an investigational anti-CD22 recombinant immunotoxin and a potential new medicine for the treatment of adult patients with hairy cell leukaemia (HCL) who have received at least two prior lines of therapy (Press release, AstraZeneca, APR 3, 2018, View Source [SID1234525473]). The FDA has granted the moxetumomab pasudotox BLA Priority Review status with a Prescription Drug User Fee Act date set for the third quarter of 2018.

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The Phase III (‘1053’) moxetumomab pasudotox clinical trial met its primary endpoint of durable complete response in adult patients with relapsed or refractory HCL, for which there is currently no established standard of care and few treatments available.[i],[ii] Results from the 1053 Phase III trial will be presented at a forthcoming medical meeting.

Priority Review is granted by the FDA to applications for medicines that, if approved, would offer a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.[iii]

NOTES TO EDITORS
About Moxetumomab Pasudotox

Moxetumomab pasudotox (formerly CAT-8015 or HA22) is an investigational anti-CD22 recombinant immunotoxin and a potential new medicine with the opportunity to be a first-in-class treatment in the US for patients with relapsed or refractory HCL who have received at least two prior lines of therapy. Immunotoxins are a class of anticancer agents that combine the selectivity of antibodies to target drug delivery and the potency of toxins to kill target cancer cells.[iv] Moxetumomab pasudotox is composed of a binding portion of an anti-CD22 antibody fused to a toxin. CD22 is a B-lymphocyte restricted transmembrane protein with a higher receptor density in HCL cells relative to normal B cells, making it an attractive therapeutic target for the treatment of this cancer.[v] After binding to CD22, the molecule is internalised, processed and releases its modified protein toxin that inhibits protein translation, leading to apoptotic cell death. Moxetumomab pasudotox has been granted Orphan Drug Designation by the FDA for the treatment of HCL.

About Hairy Cell Leukaemia

HCL is a rare, incurable slow-growing leukaemia in which the bone marrow overproduces abnormal B cells or lymphocytes.[vi] HCL can result in serious and life-threatening conditions, including infections, bleeding and anaemia.[vii] Approximately 1,000 people are diagnosed with HCL in the US each year.[viii],[ix],[x] While many patients initially respond to treatment, up to 40% will relapse.[xi] With no established standard of care and very few treatments available, there remains significant unmet medical need for people with relapsed or refractory HCL.1,2

About the ‘1053’ Phase III Trial

The ‘1053’ trial is a single-arm, multicentre Phase III clinical trial assessing the efficacy, safety, immunogenicity and pharmacokinetics of moxetumomab pasudotox monotherapy in patients with relapsed or refractory HCL who have received at least two prior therapies. The trial is being conducted in 80 patients across 34 sites in 14 countries.[xii]

About AstraZeneca in Haematology

Leveraging its collective heritage in oncology, AstraZeneca has established haematology as one of four key oncology disease areas of focus, and is accelerating development of a broad portfolio of potential blood cancer treatments. AstraZeneca and Acerta Pharma, its haematology research and development centre of excellence, recently received US FDA approval for Calquence (acalabrutinib), the first medicine in this franchise.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that have the potential to transform patients’ lives and the Company’s future. With at least six new medicines aimed to be launched between 2014 and 2020 and a broad pipeline of small molecules and biologics in development, we are committed to advance New Oncology as one of AstraZeneca’s five Growth Platforms focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

About MedImmune

MedImmune is the global biologics research and development arm of AstraZeneca, a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialisation of small molecule and biologic prescription medicines. MedImmune is pioneering innovative research and exploring novel pathways across Oncology; Respiratory, Cardiovascular & Metabolic Diseases; and Infection and Vaccines. The MedImmune headquarters is located in Gaithersburg, MD, one of AstraZeneca’s three global R&D centres, with additional sites in Cambridge, UK, and Mountain View, CA. For more information, please visit www.medimmune.com.

EMA to review cemiplimab as a potential treatment for advanced cutaneous squamous cell carcinoma

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).

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

Delcath’s PHP Therapy to be Featured in Video Training Session at European Conference of Interventional Oncology

On April 3, 2018 Delcath Systems, Inc. (OTCQB:DCTH), an interventional oncology company focused on the treatment of primary and metastatic liver cancers, announces that Dr. Brian Steadman, Interventional Oncologist at the Southampton University Hospital will present a video training session at the Annual Meeting of the European Conference of Interventional Oncology (ECIO) (Press release, Delcath Systems, APR 3, 2018, View Source;p=RssLanding&cat=news&id=2340763 [SID1234525453]). Dr. Steadman will present an overview of the Percutaneous Hepatic Perfusion (PHP) procedure and potential indications of PHP Therapy, as well as review recent research. The session will be held Tuesday, April 24, 2018 at 3pm local time.

The ECIO will be held in Vienna, Austria, April 22-25, 2018.

Pfizer and Allogene Therapeutics Enter into Asset Contribution Agreement for Pfizer’s Allogeneic CAR T Immuno-oncology Portfolio

On April 3, 2018 Pfizer Inc. (NYSE:PFE) and Allogene Therapeutics, Inc. (Allogene) reported that the two companies have entered into an asset contribution agreement for Pfizer’s portfolio of assets related to allogeneic chimeric antigen receptor T cell (CAR T) therapy, an investigational immune cell therapy approach to treating cancer (Press release, Pfizer, APR 3, 2018, https://www.pfizer.com/news/press-release/press-release-detail/pfizer_and_allogene_therapeutics_enter_into_asset_contribution_agreement_for_pfizer_s_allogeneic_car_t_immuno_oncology_portfolio [SID1234525452]).

Pfizer Inc. (NYSE:PFE) and Allogene Therapeutics, Inc. (Allogene) reported that the two companies have entered into an asset contribution agreement for Pfizer’s portfolio of assets related to allogeneic chimeric antigen receptor T cell (CAR T) therapy, an investigational immune cell therapy approach to treating cancer.

Pfizer views this agreement as an attractive opportunity to support the continued development of allogeneic CAR T therapy in a highly focused and skilled manner. Pfizer will continue to participate financially in the development of the CAR T portfolio through a 25 percent ownership stake in Allogene. Separately, Pfizer continues to have an 8 percent ownership stake in Cellectis through an equity agreement entered into in 2014. Allogene, a Two River portfolio company, was formed with Series A financing of $300 million from an investment consortium that includes TPG, Vida Ventures, BellCo Capital, the University of California Office of the Chief Investment Officer and Pfizer, among others. TPG, Vida Ventures, BellCo Capital and Pfizer will be represented on the Allogene Board of Directors. Closing is expected in the second quarter of 2018, subject to closing conditions.

"The allogeneic CAR T platform represents a potentially transformative approach to treating cancer, and we are very excited about what the future may hold for this area of research," said Robert Abraham, Senior Vice President and Group Head, Oncology Research & Development, Pfizer. "We believe that under the strong scientific, clinical development and regulatory expertise of Allogene’s leadership team, the portfolio of CAR T assets contributed by Pfizer will be well-positioned to rapidly advance into potential innovative new therapies, and ultimately to reach patients in need more quickly."

"While there is important work underway across the industry for next-generation autologous cell therapy, Allogene hopes to bring about the next revolution in the field with the successful development of allogeneic cell therapy and the potential for greater and faster patient access," said Belldegrun. "Under the direction of David Chang, an extraordinary scientist, physician and life sciences business executive with over 30 years of unprecedented experience in developing cancer treatments, Allogene is poised to potentially lead the development of one of the most exciting opportunities in our industry today."

"Last year, Kite’s anti-CD19 CAR T therapy became the first autologous CAR T treatment to be approved by the U.S. Food and Drug Administration for adult patients with aggressive non-Hodgkin lymphoma. Many believed the idea was rooted in science fiction, but science fiction became a reality," said Chang. "We believe that this partnership among leaders in the field – visionaries, industry forerunners, venture capitalists and researchers – has the potential to accelerate the development of allogeneic T cell therapy, making it a reality and forever changing how cancer is treated."

"Investing in innovation and R&D has long been a hallmark of who we are as investors, and for many years, we’ve been partnering with dynamic companies that are driving meaningful change in healthcare," said Todd Sisitsky, Managing Partner, TPG Capital. "We believe CAR T is one of the most exciting spaces within the pharmaceutical landscape today, and we are thrilled to partner with a best-in-class management team and industry leaders to invest in this potentially groundbreaking opportunity."

"As a pioneer of the allogeneic approach and expert in gene editing, the Cellectis team is excited by this agreement and eager to continue this groundbreaking work with Allogene’s experienced team, striving to accelerate the development of the portfolio and to continue along the path of making these treatments available to patients as soon as possible," said Dr. André Choulika, Cellectis CEO.

"I believe that the recognized expertise of the Allogene team in the field of CAR T will be of benefit to the development of UCART19, for which Servier is the sponsor of two clinical studies," commented Olivier Laureau, President of Servier Group. "The development of off-the-shelf allogeneic CAR T therapy in the field of oncology initiates a revolution that could potentially expand access of such innovative treatment to a larger number of oncologists and their patients."

Centerview Partners is acting as financial advisor to Pfizer, with Ropes & Gray LLP acting as its legal advisor. Cooley LLP is serving as legal counsel to Allogene, Vida Venture and TPG. Gibson Dunn & Crutcher LLP are also serving as legal counsel to TPG.

Allogeneic CAR T Cell Therapies

Allogeneic CAR T cell therapies have the potential to become the next advancement in one of the most powerful anti-cancer agents, eliminating the need to create personalized therapy from a patient’s own cells. These therapies are developed from cells of healthy donors and stored for "off-the-shelf" use in patients, simplifying manufacturing process and reducing waiting time for patients.

Allogene will receive from Pfizer the rights to 16 preclinical CAR T assets licensed from Cellectis and Servier and one clinical asset licensed from Servier, UCART19, an allogeneic CAR T therapy that is being developed for treatment of CD19-expressing hematological malignancies. In partnership with Servier, UCART19 is initially being developed in acute lymphoblastic leukemia (ALL) and is currently in Phase I. UCART19 utilizes TALEN gene editing technology pioneered and owned by Cellectis.

Allogene and Servier intend to initiate Phase 2 studies in 2019. Under the terms of the original development agreement, Allogene will have exclusive rights to develop and commercialize UCART19 in the United States, while Servier will retain exclusive rights for all other countries.

Pfizer’s Commitment to Immuno-Oncology

Immuno-Oncology (IO) is a key area of focus within Pfizer’s broad oncology portfolio, with research and development efforts spanning diverse modalities and mechanisms of action that tap into key immune system functions, harnessing the natural ability of the immune system to fight cancer. We believe that the future of IO lies in novel, biologically rational combinations based on unique tumor characteristics. We believe that Pfizer Oncology’s pipeline is in a strong position to help advance the next wave of IO science by developing new targeted therapies and IO combinations – areas in which Pfizer has a robust and proven legacy. We know that great science comes through collaboration, and we actively team up with strategic partners in IO who we believe will strengthen our portfolio and help speed innovative treatments to benefit more patients.

About Allogene Therapeutics

Allogene Therapeutics is a biotechnology company with a mission to catalyze the next revolution in cancer treatment through the development of allogeneic chimeric antigen receptor T-cell (CAR T) therapy directed at blood cancers and solid tumors. Founded and led by former Kite Pharma executives who bring unrivaled clinical development acumen in cell therapy, Allogene is well-positioned to further the potential of allogeneic cell therapy for patients.

Allogeneic CAR T therapies are engineered from cells of healthy donors and stored for "off-the-shelf" use in patients. This approach eliminates the need to create personalized therapy from a patient’s own cells, simplifies manufacturing, and reduces the time patients must wait for CAR T treatment. The Allogene portfolio includes 16 pre-clinical T cell therapy assets and UCART19, an allogeneic CAR T therapy currently in Phase 1 development for the treatment of acute lymphoblastic leukemia (ALL). Through its notable partnerships, Allogene leverages pioneering technology platforms, including TALEN gene editing technology, to progress its portfolio of immuno-oncology therapies. Allogene, with headquarters in San Francisco, California, is a Two River portfolio company formed with one of the largest Series A financings in biotechnology from an investment consortium that includes TPG, Vida Ventures, BellCo Capital, the University of California Office of the Chief Investment Officer and Pfizer, among others. For more information, please visit www.allogene.com, follow @AllogeneTx on Twitter and LinkedIn.

Preliminary Data from Phase 3 VISTA Trial in Bladder Cancer to Be Presented in a Plenary Session at American Urological Association Annual Meeting

On April 3, 2018 Eleven Biotherapeutics, Inc. (NASDAQ: EBIO), a late-stage clinical company developing next-generation antibody-drug conjugate (ADC) therapies for the treatment of cancer, reported that preliminary efficacy and safety data from its ongoing Phase 3 VISTA trial of Vicinium in patients with non-muscle invasive bladder cancer (NMIBC) who have been previously treated with bacillus Calmette-Guérin (BCG) have been selected for presentation during a plenary session currently scheduled for Monday, May 21, 2018 at 11:00 a.m. PT at the American Urological Association Annual Meeting taking place in San Francisco (Press release, Eleven Biotherapeutics, APR 3, 2018, View Source [SID1234525376]). Vicinium is a fusion protein, designed to be a next-generation ADC, that targets the epithelial cell adhesion molecule (EpCAM) antigens on the surface of bladder cancer cells to deliver a potent cytotoxin into those cells.

Data, as reported in the abstract published online today, are from a subgroup of the first 75 evaluable carcinoma in situ patients. Within these patients, preliminary findings show that greater than 95 percent expressed EpCAM on the surface of tumor cells, the target of Vicinium. Efficacy data as measured by complete response rates at three-months will be reported during the plenary session. Vicinium has been well-tolerated. Treatment-related adverse events (AEs) were reported in 46 percent of patients, the majority of which were Grade 1 or 2. The most common of these were dysuria (12%), UTI or pollakiuria (10%) and hematuria (7%), which are often associated with catheter-delivered treatments into the bladder and bladder cancer itself. As of the December 2017 data cut off, three treatment-related serious AEs were reported, including renal failure (CTCAE Grade 5) with cholestatic hepatitis (Grade 4) in one patient and acute kidney injury (Grade 3) in a second patient who recovered.

In March 2018, recruitment was completed in the VISTA trial, a single-arm registration study designed in accordance with U.S. Food and Drug Administration’s final guidance for developing treatments for BCG-unresponsive NMIBC.
"When treatment with today’s standard of care, BCG, is no longer an option, the next treatment option is typically removal of the patient’s bladder, a challenging, life-altering procedure that many patients elect not to undergo," said Stephen Hurly, president and chief executive officer of Eleven Biotherapeutics. "Vicinium has demonstrated that it is a well-tolerated and active agent in patients with BCG unresponsive NMIBC in studies to-date. We are excited to be presenting the first preliminary data from our Phase 3 VISTA trial of Vicinium for patients with NMIBC at this year’s AUA meeting. During our plenary presentation, we will share initial efficacy findings and data supporting the favorable safety we have observed so far with Vicinium. We believe Vicinium holds tremendous potential as a treatment for bladder cancer, and we look forward to sharing these and additional data later in the year."
The company plans to host a conference call in conjunction with the data presentation, with details to follow.
About the VISTA Clinical Trial
The VISTA trial is an open-label, multicenter, single-arm Phase 3 clinical trial evaluating the efficacy and tolerability of Vicinium in patients with high-risk non-muscle invasive bladder cancer (NMIBC) that is carcinoma in situ (CIS, cancer found on the inner lining of the bladder that has not spread into muscle or other tissue) or papillary (cancer that has grown from the bladder lining out into the bladder but has not spread into muscle or other tissue), who have been previously treated with bacillus Calmette-Guérin (BCG). The primary endpoint of the trial is the complete response rate in patients with CIS with or without papillary disease and durability of that response. Patients in the study receive locally administered Vicinium twice a week for six weeks, followed by once-weekly treatment for another six weeks, then treatment every other week for up to two years. Topline data assessing responses and durability of responses at three-months on treatment are expected in mid-2018, with 12-month data anticipated in mid-2019.

About Vicinium
Vicinium, Eleven Biotherapeutics’ lead product candidate, is a next-generation antibody-drug conjugate developed using the company’s proprietary Targeted Protein Therapeutics platform. Vicinium is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A (ETA). Vicinium is constructed with a stable, genetically engineered linker to ensure the 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 its safety. In prior clinical studies conducted by Eleven Biotherapeutics, EpCAM has been shown to be overexpressed in non-muscle invasive bladder cancer (NMIBC) cells with minimal to no EpCAM expression observed on normal bladder cells. Eleven Biotherapeutics is currently conducting the Phase 3 VISTA trial, designed to support the registration of Vicinium for the treatment of NMIBC in patients who have previously received two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Topline, three-month data from the trial are expected in mid-2018. Additionally, Eleven Biotherapeutics believes that Vicinium’s cancer cell-killing properties promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. The activity of Vicinium in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.