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 16, 2016 Bio-Path Holdings, Inc., (NASDAQ: BPTH) ("Bio-Path"), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery technology to develop a portfolio of targeted nucleic acid cancer drugs, reported operational and financial results for the year ended December 31, 2015 (Filing, Annual, Bio-Path Holdings, 2015, MAR 16, 2016, View Source [SID:1234509584]).

"2015 was a year of significant progress for Bio-Path," said Peter Nielsen, President and Chief Executive Officer of Bio-Path. "With two full remissions achieved in the safety segment of our Phase II trial of lead candidate BP1001 in advanced AML patients, the formation of an inaugural Scientific Advisory Board, and receiving orphan drug designation for BP1001 in AML, we are looking forward to a successful 2016."

2015 Operational Highlights:

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· Finalized the data package for the monotherapy portion of the Phase I clinical trial of Bio-Path’s lead product candidate, BP1001 (Liposomal Grb2 antisense), in blood cancers during the fourth quarter of 2015. BP1001 was well tolerated and showed signs of anti-leukemia activity and no drug-related toxicities. Among 21 evaluable patients, more than half experienced at least a 50 percent reduction in peripheral or bone marrow blasts from baseline. Additionally, several patients demonstrated transient improvement and/or stable disease. Notably, one patient with chronic myelogenous leukemia (CML) blast phase showed a significant reduction in blasts. Patient data from the Phase I clinical trial also demonstrated significant reductions in the target Grb2 protein and its downstream proteins, providing positive evidence that Bio-Path’s DNAbilizeTM neutral lipid delivery with proprietary antisense technology successfully delivers an antisense drug substance to a diseased cell to knock down the target protein.

· Received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BP1001 for the treatment of acute myeloid leukemia (AML) in the second quarter of 2015. Orphan drug status provides Bio-Path with seven years of exclusivity after receiving formal marketing approval, as well as additional development incentives.

· Performed preclinical testing of BP1001 in two additional indications—triple negative breast cancer (TNBC) and inflammatory breast cancer (IBC), two cancers characterized by formation of aggressive tumors and relatively high mortality rates. Bio-Path is rolling this initiative into a broader solid tumor testing program, including advanced ovarian cancer. The preclinical program may be expanded to include combination therapy evaluations.

· Added a second drug manufacturer, strengthening Bio-Path’s manufacturing process while increasing capability and capacity.

· Continued preclinical evaluation of a third DNAbilizeTM product. Bio-Path’s product candidate screening and development program has validated the next promising candidate, which will diversify the Company’s product pipeline. Potential indications include diffuse large B-cell lymphoma, non-small cell lung cancer, pancreatic cancer and disease candidates outside of oncology, such as autoimmune disorders.

· Formed a Scientific Advisory Board to support the advancement of Bio-Path’s clinical and preclinical therapeutic candidates. Jorge Cortes, M.D., renowned leukemia expert from The University of Texas MD Anderson Cancer Center, joined as Chairman. Amy P. Sing, M.D., a member of Bio-Path’s board of directors and Senior Director of Medical Affairs at Genomic Health, Inc., joined as a founding member.

· Presented Bio-Path’s proprietary technology and clinical trial results at an international meeting. Jorge Cortes, M.D. of The University of Texas MD Anderson Cancer Center and Chair of Bio-Path’s Scientific Advisory Board presented a poster at the 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 7, 2015 in Orlando, FL. Dr. Cortes discussed data from the Phase I and safety segment of the Phase II clinical trials of BP1001 in blood cancers. These data included the complete remission of two evaluable patients receiving BP1001 in combination with low-dose cytarabine (LDAC) chemotherapy.

· Presented to the medical and scientific community at the IBC’s 17th Annual TIDES: Oligonucleotide and Peptide Therapeutics Conference in San Diego. The presentation featured Bio-Path’s DNAbilizeTM technology for delivering liposome/antisense drugs and highlighted BP1001. The TIDES Summit is prominently known as the premier conference for the oligonucleotide and peptide discovery, development and manufacturing industries.

· Continued enhancement of Bio-Path’s public profile within the investment community and biopharmaceutical industry. Chief Executive Officer Peter Nielsen delivered company presentations at the 17th Annual Rodman & Renshaw Global Investment Conference in September 2015, the 14th Annual BIO Investor Forum in October 2015, Biotech Showcase 2015 Conference in San Francisco, CA in January 2016 and the 18th Annual BIO CEO & Investor Conference in New York City in February 2016.

· Established an "at-the-market" ("ATM") program during the second quarter of 2015, through which it may offer and sell up to $25 million of common stock from time to time, at Bio-Path’s discretion, through an investment banker, acting as sales agent. Sales of Bio-Path common stock under the ATM program may be made directly on or through the Nasdaq Capital Market, among other methods. As of December 31, 2015, the Company has not offered or sold any shares of common stock under the ATM program.

Recent / First Quarter 2016 Operational Highlights:

· Completed the safety segment of the Phase II clinical trial of BP1001, in combination with low-dose cytarabine (LDAC) chemotherapy, in patients with advanced AML. Of the six evaluable patients, two had a complete response and two had a partial response, with one patient continuing treatment. Bio-Path saw no adverse events attributable to BP1001 treatment.

· Entered into a sponsored research agreement with The University of Texas MD Anderson Cancer Center to evaluate Bio-Path’s clinical pipeline for its ability to modulate pancreatic cancer.

Expected Upcoming Milestones:

· BP1001 in Acute Myeloid Leukemia (AML): Bio-Path is finalizing steps to commence a multi-site Phase II clinical trial assessing the efficacy of BP1001 in combination with low-dose cytarabine (LDAC) chemotherapy, which is expected to commence in the second quarter of 2016.

· BP1001 in Chronic Myelogenous Leukemia (CML): Bio-Path commenced development of a protocol for a Phase II clinical trial evaluating BP1001 in combination with frontline chemotherapy in CML patients in blast crisis, an area of unmet medical need. This clinical trial is expected to start in the second quarter of 2016.

· BP1002 (Liposomal Bcl2; L-Bcl2): Bio-Path is finalizing a preclinical package of toxicity, tissue distribution, pharmacokinetics and efficacy studies for its second product candidate, BP1002. An Investigational New Drug (IND) application will be filed with the FDA upon finalizing drug batch required for the Chemistry, Manufacturing and Controls section of the IND. Bio-Path expects that the favorable toxicity profile of BP1001 will allow for a Phase I clinical trial of BP1002 to begin at a higher dose, thus reducing the number of patients required to complete the safety phase of the trial.

2015 Financial Highlights:

· Bio-Path reported a net loss of $5.5 million for the year ended December 31, 2015, compared to a net loss of $4.5 million for the year ended December 31, 2014. The increase was primarily due to increased clinical trial expenses, manufacturing development, preclinical study costs and personnel costs associated with the addition of research and development support staff in the second half of 2014. The Company reported a net loss of $0.06 per share for the year ended December 31, 2015, compared to a net loss of $0.05 per share for the year ended December 31, 2014.

· Research and development expenses for the year ended December 31, 2015 increased to $3.0 million, compared to $1.8 million for the year ended December 31, 2014.

· General and administrative expenses for the year ended December 31, 2015 decreased to $2.5 million, compared to $2.7 million for the year ended December 31, 2014.

· As of December 31, 2015, the Company had a cash balance of $8.9 million, compared to $13.9 million at December 31, 2014. Net cash used in operating activities for the year ended December 31, 2015 was $5.0 million, compared to $3.8 million for the comparable period in 2014.

About Bio-Path’s Delivery Technology

Bio-Path’s drug delivery technology, called DNAbilize, involves microscopic-sized liposome particles that distribute nucleic acid drugs systemically and safely throughout the human body, via simple intravenous infusion. The delivery technology is applied to proprietary, single stranded (antisense) nucleic acid compounds with the potential to revolutionize the treatment of cancer and other diseases where druggable targets of disease are well characterized. The Company is currently focused on developing liposomal antisense drug candidates. Bio-Path also anticipates developing liposome tumor targeting technology, representing next-generation enhancements to the Company’s core liposome delivery technology.

About BP1001 (Liposomal Grb2 antisense)

BP1001 (Liposomal Grb2 antisense) is a neutral-charge, liposome-incorporated antisense drug substance designed to inhibit Grb2 protein expression. The protein Grb2 is essential to cancer cell signaling because it is utilized by oncogenic tyrosine kinases to induce cancer progression. Suppressing the function or expression of Grb2 should interrupt its vital signaling function and have a therapeutic application in cancer.