On March 16, 2016 Amrita Therapeutics ("the Company"), a biopharmaceutical company developing innovative technologies from the microbiome, reported that it is pleased to announce its submission to the FDA of a pre-INDType B Meeting request for discussion of development plans for the Company’s lead oncology peptide AT-01C (Press release, Amrita Therapeutics, MAR 16, 2016, View Source [SID:1234512730]).

Amrita Therapeutics’ AT-01C peptide demonstrates p53 ("guardian of the genome") tumor suppression and binds to the SMAR1* protein for down-regulation of oncogenes with little or no toxicity to healthy tissues, with effectiveness against solid tumors including genitourinary, gastrointestinal (GI), liver and CNS cancers. In parallel to clinical development of AT-01C, the Company is focusing on the ‘master regulator’ SMAR1 biomarker as a companion diagnostic test to identify patients most likely to benefit from AT-01C therapy.

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Susan K. Finston, Amrita Therapeutics’ Chief Executive Officer notes, "we continue to gain insights into the extraordinary therapeutic potential of peptides from the microbiome and look forward to clinical development of AT-01C to address unmet needs of cancer patients."

With less toxicity and fewer side effects, naturally occurring peptide drugs have a greater likelihood of regulatory approval double that of small molecule new chemical entities (NCEs) for a range of indications including infectious diseases, metabolic disorders and advanced cancer therapies. Amrita’s first peptide drug has the potential to reach sales in excess of $ 1 billion within the first years of launch.

On March 16, 2016 Kite Pharma, Inc., (Nasdaq:KITE) ("Kite") a clinical-stage biopharmaceutical company focused on developing engineered autologous T cell therapy (eACT) products for the treatment of cancer, reported that two oral presentations and two poster presentations to be delivered at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in New Orleans, Louisiana (Press release, Kite Pharma, MAR 16, 2016, View Source [SID:1234509601]). The oral presentations will address KTE-C19, Kite’s lead chimeric antigen receptor (CAR) product candidate, and, separately, an engineered T cell receptor (TCR) product candidate targeting the cancer testis antigen MAGE-A3. The TCR product candidate is currently being studied as part of a Cooperative Research and Development Agreement (CRADA) between Kite and the National Cancer Institute.

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Oral Presentations:

Title: Updated Phase 1 Results from ZUMA-1: A Phase 1-2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-C19 (Anti-CD19 CAR T Cells) in Subjects with Refractory Aggressive Non-Hodgkin Lymphoma (NHL)

Date: Tuesday, April 19, 2016 3:00-5:00PM Central Time
Session: Early Clinical Trials Evaluating Cell-based, Checkpoint Inhibitors, and Novel Immunotherapeutics
Abstract Number: CT135
Location: Room 343, Morial Convention Center
Presenter: Armin Ghobadi, M.D., Washington University, St. Louis, MO

Title: A Phase 1 Study of an HLA-DPB1*0401-restricted T Cell Receptor Targeting MAGE-A3 for Patients with Metastatic Cancer

Date: Sunday, April 17, 2016 2:15-4:00PM Central Time
Session: Immuno-Oncology Clinical Trials I
Abstract Number: CT003
Location: La Nouvelle Ballroom, Morial Convention Center
Presenter: Yong-Chen W. Lu, Ph.D., Surgery Branch, National Cancer Institute

Poster Presentations:

Title: Manufacturing and Characterization of KTE-C19 in a Multicenter Trial of Subjects with Refractory Aggressive Non-Hodgkin’s Lymphoma (NHL) (ZUMA-1)

Date: Monday, April 18, 2016 1:00-5:00PM Central Time
Session: Adoptive Cell Therapy
Abstract Number: 2308
Location: Poster Hall, Section 25, Poster Board 20
Presenter: Marc Better, Ph.D., Kite Pharma, Santa Monica, CA

Title: Comparative Evaluation of Peripheral Blood T cells and Resultant Engineered Anti-CD19 CAR T Cell Products from Relapsed/Refractory Non-Hodgkin’s Lymphoma (NHL) Patients

Date: Monday, April 18, 2016 1:00-5:00PM Central Time
Session: Adoptive Cell Therapy
Abstract Number: 2305
Location: Poster Hall, Section 25, Poster Board 17
Presenter: Timothy J. Langer, Kite Pharma, Santa Monica, CA

About KTE-C19

KTE-C19 is an investigational therapy in which a patient’s T cells are genetically modified to express a CAR designed to target the antigen CD19, a protein expressed on the cell surface of B cell lymphomas and leukemias. Kite is currently enrolling four pivotal studies (also known as ZUMA studies) for KTE-C19 in patients with various B cell malignancies. The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation status to KTE-C19, for the treatment of patients with refractory diffuse large B cell lymphoma, primary mediastinal B cell lymphoma, and transformed follicular lymphoma. KTE-C19 has also secured Orphan Drug Designation in the U.S. for DLBCL and in the EU for various hematological indications.

On March 16, 2016 GSK and Miltenyi Biotec reported a strategic collaboration that will bring together GSK’s expertise in developing cell and gene therapy based treatments with Miltenyi Biotec’s global leadership in cell processing and related technologies in cell therapy (Press release, GlaxoSmithKline, MAR 16, 2016, View Source [SID:1234509747]). The collaboration seeks to optimise the manufacture and delivery of these personalised therapies using increased automation and leading edge processing technology.

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GSK is building a cell and gene therapy R&D platform to underpin development of novel therapies in oncology and rare diseases – two of its core research areas. This reflects the company’s belief in cell and gene therapy’s potential as an important treatment approach for tackling the underlying cause of serious disease.

Through the collaboration, Miltenyi Biotec will engage with GSK to integrate greater automation and high-tech processing technology into GSK’s current cell and gene therapy R&D manufacturing capabilities. The goal is to use this increased automation to further industrialise cell and gene therapy, overcoming the manufacturing and scale-up constraints associated with current, more manual cell and gene therapy processes. This could reduce the costs and geographical barriers associated with this treatment approach, speed development of therapies and support their potential beyond rare diseases and limited populations.

The collaboration will also bring together the technology and expertise of both companies to advance the discovery of new CAR (chimeric antigen-receptor) T-cell based therapeutics – cells that have been engineered to target and destroy cancer cells by strengthening a patient’s natural T-cell response. GSK and Miltenyi Biotec will collaborate on defined CAR-T oncology targets and on the development of advancements in technologies in this space that may be further applied by both companies. This collaboration supplements GSK’s existing CAR-T preclinical portfolio.

Patrick Vallance, President of Pharmaceuticals R&D at GSK, said: "Cell based gene therapies are living treatments, unique to individual patients and complex to manufacture. We see tremendous potential for the cell and gene therapy platform we are building within GSK, however the complexity of current manufacturing processes limits their use to local treatment of small patient populations. Working with Miltenyi Biotec, our vision is to transform current technology so that we can expand the possibilities for cell and gene therapy treatment to wider patient populations with broader geographical reach."

Stefan Miltenyi, President and CEO at Miltenyi Biotec, said: "For more than 20 years we have been developing and providing cell therapy solutions to patients worldwide. Working together with the global experts at GSK, we will accelerate innovation to broaden patient access to future personalised cell and gene therapy."

About Cell and Gene therapies
Cell and gene therapies are potentially powerful disease modifying experimental treatments that focus on genetically engineering living cells to either repair the direct cause of a genetic defect or equip them with genes that enhance their functions.

To make cell and gene therapy treatments, selected populations of cells are extracted from the body and genetically-engineered to produce the desired therapeutic effect. This can mean replacing a faulty gene in a stem cell or changing immune cells so they can recognise tumours. The transformed cells are then re-introduced into a patient’s body where they exert their effect by replacing faulty genes or educating the immune system to recognise and kill cancer cells

On March 16, 2016 Cellectis (Alternext: ALCLS – Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells (UCART), and MabQuest SA, a biotech company focused on the development of antibody-based therapeutic interventions, reported that they have entered into a research collaboration and license agreement pertaining to the development of a new class of monoclonal antibodies targeting PD-1 (Press release, Cellectis, MAR 16, 2016, View Source [SID1234645116]).

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The action of these PD-1 antibodies is to promote the recovery of T-cells from exhaustion through a new mechanism of action. This new class of antibodies differs from currently approved anti-PD-1 mAbs in that they do not block the PD-1-PD-L1 interaction. These anti-PD-1 mAbs have potential uses for multiple indications in immunotherapy, including notably treatments for a variety of cancers. Cellectis plans to use this new class of anti-PD-1 antibodies either in combination therapy with its gene-edited UCART product candidates or single-agent or in combination with other already approved immunotherapy drugs.

In vitro studies have shown that the combination of these novel PD-1 mAbs with currently approved anti-PD-1 mAbs enhances the recovery of T-cells from exhaustion. Due to their new mechanism of action, these anti-PD-1 mAbs may be used in combination with other PD-1/PD-L1 inhibitors, such as Nivolumab and Pembrolizumab, or other checkpoints inhibitors and immunotherapy approaches for boosting the therapeutic effects of single therapy. Furthermore, this novel class of anti-PD-1 mAbs may represent an alternative and effective therapeutic intervention in those cancer patients with tumors expressing low levels of PD-L1, with respect to the currently approved anti-PD-1 mAbs. In addition, Cellectis intend to combine these PD-1 mAbs with its gene-edited UCART product candidates to enhance their activity and increase their half-life.

The agreement includes a collaboration phase funded by Cellectis whereby Cellectis and MabQuest will jointly pursue preclinical research on several candidate antibodies; and a clinical development and commercialization phase of the best selected antibodies which will be led by Cellectis.

Under the agreement, MabQuest has granted an exclusive option to Cellectis. Upon exercise of the option, Cellectis would be granted worldwide exclusive rights over the family of PD-1 antagonist antibodies developed under the collaboration for all fields, and further potential derivatives of these antibodies.

"We are very pleased to have signed this agreement with MabQuest, with founders and lead scientists who have great expertise in the field of immunology and monoclonal antibodies," said André Choulika, Chairman and Chief Executive Officer of Cellectis. "This collaboration is an important building block for our gene-edited UCART product candidates and for our immunotherapy franchise. This new partnership fits perfectly into Cellectis’ strategy of expanding our focus in the cancer immunotherapy space with our CAR T-cell based approaches."

"The collaboration agreement with Cellectis is a tremendous opportunity for MabQuest to move into clinical development with this new class of anti-PD-1 mAbs. This collaboration will also boost MabQuest’s discovery program to develop additional antibody-based strategies to modulate the host immune system," said Dr. Giuseppe Pantaleo, President of MabQuest and Professor of Medicine and Chief of the Service of Immunology and Allergy at the Lausanne University Hospital, Lausanne, Switzerland.

On March 15, 2016 BIND Therapeutics, Inc. (NASDAQ: BIND), a biotechnology company developing targeted and programmable therapeutics called ACCURINS, reported financial results for the fourth quarter and full year 2015 (Press release, BIND Therapeutics, MAR 15, 2016, View Source [SID:1234509533]). Additionally, the Company announced a shift in its research and discovery strategy to focus on the development of innovative medicines, primarily in cancer. Moving forward, BIND plans to focus on the development of therapeutics that leverage the ability of ACCURINS to incorporate novel combinations of targeting ligands and unique payloads including oligonucleotides and potent kinase inhibitors, creating synergistic properties in a single particle.

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"Our new strategy is to build a pipeline of innovative medicines that address challenges that small molecule chemistry or antibody engineering have not been able to overcome," said Andrew Hirsch, president and chief executive officer, BIND Therapeutics. "Historically, nanoparticle-based therapies have used previously approved medicines as therapeutic payloads to provide equivalent efficacy with improved safety. Our new strategy leverages the unique attributes of ACCURINS, specifically their ability to precisely target cells and tissues with ligand-mediated binding while containing high concentrations of novel therapeutic payloads. We believe this approach will lead to innovative medicines with the potential to achieve therapeutic outcomes unachievable through existing therapeutic modalities."

ACCURINS possess several advantageous properties. The surface of ACCURINS can be functionalized with ligands that can achieve tissue localization, cellular internalization or biological activity, thereby increasing the number of therapeutic strategies available. In addition, the modular nature of ACCURINS enables the functionalization of more than one type of ligand on the surface. ACCURINS are also able to incorporate novel therapeutic payloads with diverse physical and chemical properties, including oligonucleotides and potent kinase inhibitors. The release rate of Accurin payloads can also be controlled to optimize the amount of drug getting to the target tissue over the optimal amount of time.

"Our internal discovery efforts leverage the modular nature of ACCURINS, allowing us to efficiently engineer ideal combinations of tumor-directed targeting ligands and new classes of therapeutic payloads," said Jonathan Yingling, Ph.D., chief scientific officer of BIND. "When encapsulating oligonucleotide-based payloads, ACCURINS have the potential to protect against enzymatic degradation and clearance in the plasma, target tissues beyond the liver, and concentrate them in target cells through ligand-mediated binding that results in tumor cell death. By incorporating small molecule kinase inhibitors, we believe ACCURINS can control the biological activity of tumor-protective immune cells and reverse the immunosuppressed tumor microenvironment. Following optimization of lead product candidates and completion of preclinical studies, we anticipate initiation of clinical testing for one or more of our proprietary innovative product candidates as early as 2018."

BIND remains committed to its ongoing collaborations and clinical-stage programs. The Company’s collaborations with AstraZeneca and Pfizer are intended to enable greater inhibition of important cellular pathways with our collaborators’ proprietary kinase inhibitors. Additionally, the Company expects to communicate topline data from the ongoing iNSITE 1 and 2 trials with BIND-014 in squamous cell non-small cell lung cancer (NSCLC), advanced cervical cancer and head and neck cancer in April 2016. As previously announced, further development of BIND-014 is contingent upon data from these trials.

Anticipated 2016 Milestones:

Report topline data on the full 40 patients in iNSITE 1 squamous NSCLC trial with BIND-014

Report Stage 1 data from iNSITE 2 trial with BIND-014 in advanced cervical and head and neck cancers

Report in vivo proof-of-concept (POC) data for targeting tumor associated macrophages

Identify first ACCURINS-based immuno-oncology product concept

Report initial in vivo POC data for discovery programs, with preclinical pharmacokinetic and efficacy data expected in second half of 2017

In vivo POC data for targeting guanylate cyclase-C (GC-C) receptors expressed on tumors, specifically GI malignancies

Further in vivo POC data in delivering single and double stranded RNA fragments to target cells and achieving target knock-down

Demonstrate in vitro and in vivo POC for achieving endosomal escape with double stranded RNA

2015 Business and Pipeline Highlights:

Continued preclinical work, and generated promising initial data, on Accurin versions of anti-infective and oligonucleotide-based therapies

Continued preclinical work related to targeting of tumor-associated macrophages

Strengthened scientific leadership and corporate governance with appointment of Jonathan Yingling, Ph.D., as chief scientific officer for BIND, and appointment of Arthur Tzianabos, Ph.D., to Board of Directors

Completed preclinical development for AZD2811, an Accurin containing AstraZeneca’s Aurora B Kinase inhibitor, for which AstraZeneca initiated a phase 1 clinical study in the fourth quarter of 2015

Advanced collaboration with Pfizer following its exercise of its option to obtain an exclusive license to develop and commercialize an Accurin drug candidate

Achieved $4.0 million milestone with AstraZeneca for dosing first patient in Accurin AZD2811 phase 1 clinical trial

Received $2.5 million option exercise fee from Pfizer to acquire an exclusive license for the development and commercialization of first compound covered by the agreement

Recognized $14.3 million in 2015 in revenue from milestones, option exercises and reimbursable expenses related to our collaborations with AstraZeneca, Pfizer and Roche

Enrolled patients in phase 2 iNSITE 1 and iNSITE 2 trials allowing the Company to report clinical data in April 2016

Completed build out of dedicated manufacturing space, through an arrangement with a large contract manufacturing organization, that is capable of producing ACCURINS at the double-digit kilogram scale

Fourth Quarter and Full Year 2015 Financial Results

Fourth quarter and full year 2015 revenue was $6.4 million and $15.4 million, compared to $3.0 million and $10.4 million for the same periods in 2014, respectively. The increase in revenue for the fourth quarter and full year 2015 was primarily due to the achievement of the $4 million milestone upon dosing of the first patient in Accurin AZD2811 phase 1 clinical trial.

Fourth quarter and full year 2015 research and development (R&D) expenses were $11.1 million and $37.3 million compared to $8.0 million and $28.9 million for the same periods in 2014, respectively. The increase in R&D expenses for the fourth quarter of 2015 compared to the fourth quarter of 2014 was primarily due to higher clinical development expenses of BIND-014 and reimbursable manufacturing expenses related to Accurin AZD2811. The increase in R&D expenses for the full year 2015 compared to the full year 2014 was primarily due to headcount growth to support the development of BIND’s internal programs and our collaborations, which led to an increase in salaries and benefits, higher reimbursable manufacturing expenses related to Accurin AZD2811 and increased clinical development expenses of BIND-014.

Fourth quarter and full year 2015 general and administrative expenses were $4.6 million and $17.6 million, respectively, compared to $4.2 million and $15.1 million for the fourth quarter and full year 2014, respectively.

Fourth quarter net loss was $7.6 million, or $0.37 per basic and diluted share, compared to a net loss of $8.5 million, or $0.51 per basic and diluted share for the fourth quarter of 2014. 2015 net loss was $36.6 million, or $1.81 per basic and diluted share, compared to a net loss of $32.5 million, or $1.97 per basic and diluted share for 2014.

Cash, cash equivalents and short-term investments were $36.9 million as of December 31, 2015. The Company continues to expect its cash, cash equivalents and short-term investments will fund anticipated operations into the fourth quarter of 2016.