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ISA Pharmaceuticals and Scancell enter collaboration agreement for the manufacturing, development and commercialisation of Modi-1 / AMPLIVANT® combination

On 15 February, 2018 ISA Pharmaceuticals B.V. (‘ISA’), a clinical-stage immunotherapy company, and Scancell Holdings plc, (‘Scancell’ or the ‘Company’), the developer of novel immunotherapies for the treatment of cancer, reported that they have entered into a worldwide licensing and collaboration agreement to use ISA’s AMPLIVANT adjuvant technology for the manufacturing, development and commercialisation of Scancell’s first Moditope development candidate, Modi-1 (Press release, ISA Pharmaceuticals, FEB 15, 2018, https://www.isa-pharma.com/isa-pharmaceuticals-and-scancell-enter-collaboration-agreement-for-the-manufacturing-development-and-commercialisation-of-modi-1-amplivant-combination/ [SID1234612392]). This partnership has the potential to provide a new treatment option for patients with triple negative breast cancer, ovarian cancer, sarcomas, and other solid tumours.

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Under the terms of this agreement, ISA has granted Scancell an exclusive worldwide license to manufacture, develop and commercialise the AMPLIVANT:Modi-1 conjugate therapy and will contribute know-how and expertise related to AMPLIVANT. Clinical studies will be conducted by Scancell and are expected to commence in H1 2019.

In return, ISA will receive an upfront payment from Scancell and is entitled to milestone and royalty fees following achievement of certain criteria as defined in the agreement. Financial details were not disclosed.

Previous pre-clinical data demonstrated that conjugation of the Modi-1 peptides to AMPLIVANT enhances anti-tumour immune responses 10-100 fold and resulted in highly efficient tumour eradication, including protection against tumour re-challenge.

ISA’s AMPLIVANT technology can be applied to any type of targeted immunotherapy, significantly enhancing its efficacy. It is based on a proprietary and synthetic small molecule TLR1/2 ligand with enhanced immunostimulatory activity that can be chemically coupled to the respective immunotherapeutic. AMPLIVANT conjugates allow lower dosing with higher efficacy through better dendritic cell antigen processing and presentation, as well as enhanced T cell priming.

Scancell’s Moditope platform acts by stimulating the production of CD4+ T cells using citrullinated tumour-associated peptide epitopes. This technology overcomes the immune suppression induced by tumours themselves, allowing activated T cells to seek out and kill tumour cells that would otherwise be hidden from the immune system.

"This collaboration is an important step to advance new adjuvant technologies such as AMPLIVANT to clinical-stage programmes and bring patients better treatments," said Ronald Loggers, CEO of ISA Pharmaceuticals. "The partnership will further validate the power of AMPLIVANT conjugates for use in therapeutic cancer vaccines that carry a variety of epitopes, including post-translationally modified epitopes such as Scancell’s Moditope products."

Cliff Holloway, CEO of Scancell, commented: "This collaboration with ISA Pharmaceuticals is an important step in the continued development and commercialisation of our first Moditope immunotherapy, Modi-1, which has the potential to treat patients with triple negative breast cancer, ovarian cancer and sarcoma who are resistant to other immunotherapies. Our pre-clinical studies have demonstrated Modi-1 induces potent anti-tumour responses and significant improvements in survival. We believe that combining Modi-1 with an enabling adjuvant technology such as AMPLIVANT has the potential to significantly enhance its efficacy in patients and we are looking forward to moving this important and novel therapy into the clinic in the first half of 2019."

This announcement contains inside information for the purposes of Article 7 of Regulation (EU) 596/2014 (MAR).

GT BIOPHARMA (GTBP: OTCQB) ANNOUNCES APPOINTMENT OF SHAWN M CROSS AS CHAIRMAN AND CHIEF EXECUTIVE OFFICER

On February 15, 2018 GT Biopharma, Inc. (OTCQB: GTBP) (GTBP.PA) ("GT" or the "Company"), a clinical-stage biopharmaceutical company focused on the development and commercialization of novel cancer immunotherapy products as well as central nervous system treatments, reported that Shawn M. Cross will become Chairman and Chief Executive Officer effective February 15, 2018 (Press release, GT Biopharma , FEB 15, 2018, View Source [SID1234539533]).

Mr. Cross has held the position of President and Chief Operating Officer of GTBP since November 16, 2017. The plan to add The Chairman of the Board and CEO positions to Mr. Cross’ duties as President/COO was initiated by the Company’s board of directors to prepare the company for its next phase of growth. Mr. Cross will lead GTBP through NASDAQ capital markets listing process, attract institutional investment and analyst coverage to the company, and guide development of its neurology portfolio and its first TriKE into human clinical testing in 2018.

Dr. Kathleen Clarence-Smith will become Vice Chairwoman of the Board of Directors and President of the Neurology Division to focus her efforts on creating value in GTBP’s central nervous system portfolio and drive the company’s intellectual property developments.

Anthony J. Cataldo will remain as a member of the Board of Directors and become a consultant to the company.

Mr. Cross is an internationally recognized healthcare investment banker specializing in the biopharmaceutical sector with over 20 years of experience and has completed dozens of capital markets and strategic transactions for growth-oriented biopharmaceutical companies. He joined GTBP as President and Chief Operating Officer of the Company. Previously he was Managing Director, Head of Biotechnology Investment Banking at Deutsche Bank Securities Inc. and Managing Director and Head of Biopharmaceutical Investment Banking at Wells Fargo Securities, LLC where he completed dozens of capital markets and strategic transactions for growth-oriented biopharmaceutical companies.

GT Biopharma Executive Chairman Anthony J. Cataldo said, "It is now time to move GT Biopharma to an institutional quality biotech company. I created GTBP using the same model I used when creating, what is now known as, Iovance Biotherapeutics, Inc. (IOVA); an approximately $1.5 billion market cap company. The leadership provided by Shawn Cross, Dr. Kathleen Clarence-Smith and Dr. Ray Urbanski, our Chief Medical Officer will be invaluable to moving these assets towards clinical and commercial success. I am excited for our shareholders and look forward to helping the board and management progress GT Biopharma to becoming a significant biotech company."

GT Biopharma Chief Executive Officer Dr. Kathleen Clarence-Smith said, "Having known Shawn for over a decade, I’m confident that Shawn will be a very effective CEO for GTBP while I focus on maximizing the value of our neurology portfolio. I look forward to moving the company to the next level."

GT Biopharma President and COO Shawn M. Cross said, "I continue to see a great deal of promise in GT Biopharma’s pipeline of immuno-oncology and neurology therapeutics and am honored to become Chairman and Chief Executive Officer of such a dynamic company. I look forward to announcing additional experienced members to our management team and board in the coming weeks. I also am excited to work closely with Dr. Urbanski as we prepare to enter our first TriKE into human clinical testing as well as with Dr. Clarence-Smith in creating value from our neurology portfolio."

Celularity Announces $250M in Funding to Deliver Treatments for Cancer, Inflammatory and Degenerative Diseases, and Functional Regeneration

On February 15, 2018 Celularity reported that it has been created through the contribution and acquisition of extensive intellectual property, clinical-stage assets, basic and clinical research, and product development expertise including (Press release, Celularity, FEB 15, 2018, View Source [SID1234530906]):

First proprietary allogeneic "off-the-shelf" immunotherapeutic platform
Proprietary clinical stage Placental Natural Killer (PNK) Cell Program
IND-ready CD38 CAR-T program
Innovative allogeneic CD38 CAR NK program
Phase 3-ready placental adherent cell products for serious Crohn’s Disease
Sorrento Therapeutics G-MAB Library of 50+ fully-human antibody-CAR constructs
Unparalleled IP position: 800+ issued patents in cell therapy and regenerative medicine; dominating position worldwide for placental stem cells
World-class cell and biomaterials manufacturing capabilities
Commercial-stage functional regeneration products Biovance and Interfyl, and late-stage pipeline assets across broad disease indications
LifeBankUSA, pioneering stem cell biobanking business with two decades’ operating expertise
Celularity, a biotechnology company founded by stem cell pioneer Robert Hariri, MD, PhD, is announcing the formation of the company with $250M in funding with contributions from Celgene (NSDQ: CELG), United Therapeutics (NSDQ: UTHR), Sorrento Therapeutics (NSDQ: SRNE), Human Longevity, Inc., Genting Group, the Dreyfus Family Office, Section 32, and Heritage Group. Celularity is co-founded with Vice Chairman, Peter H. Diamandis, MD, alongside Dr. Hariri, and has a board of industry luminaries including Vice Chairman, John Sculley, formerly of Apple and Pepsi-Cola, Bill Maris of Section 32 (previously founder and CEO of Google Ventures), and Andrew von Eschenbach, former commissioner of the US Food and Drug Administration.

Celularity sources, develops, and deploys transformative therapies derived from the placenta for treatment of complex medical conditions including hematological and solid tumors, autoimmune disease, diabetes, as well as degenerative effects of aging. By combining synergistic assets from Celgene, United Therapeutics, Sorrento Therapeutics, and Human Longevity Inc., Celularity is accelerating the development of cell and tissue regenerative products to address unmet medical needs. These treatments have the potential to reverse life-threatening diseases and extend the healthy human lifespan.

"My goal is to make it so the next generation grows up in a world where cancer is managed just like the common cold, and the body’s natural regenerative engine remains empowered throughout our lives," said Dr. Hariri, Founder, Chairman and Chief Executive Officer of Celularity. "Celularity is a new biotechnology company model founded to harness the placenta as a platform for discovery and therapeutics, ultimately with a goal of amplifying the body’s ability to fight disease, restore function and extend the healthy lifespan. It is my vision that the cellular medicines we derive from the placenta will lead to abundant and affordable treatments."

Celularity has assembled an industry-leading intellectual property portfolio of more than 800 issued patents, which attain a dominating position around placental-derived stem and progenitor cells. Celularity is also the first to own and deploy the full value chain ranging from sourcing placental stem cells to delivering patient treatment. The company is built on three key pillars: Cell Therapy, Functional Regeneration, and Biosourcing:

Cell Therapy: Celularity has developed five clinical stage cell therapy candidates currently being evaluated for cancer, and immunological and degenerative diseases
Functional Regeneration: Celularity is addressing serious wounds, burns, orthopedic, and other surgical indications including reconstructive and aesthetic applications with a broad range of placental biomaterials. Derived from placental tissue, Celularity’s BIOVANCE and Interfyl were the first biomaterial products introduced to the market to enhance the body’s regenerative processes
Biosourcing: Celularity owns and operates LifeBankUSA, the world’s only repository that allows families to bank their newborn’s placental cells and biomaterials for future therapeutic and regenerative use
Celularity is the only company with an allogeneic placental cell platform. Placental stem cells are uniquely immunoprivileged, therefore treatments do not require cells to be engineered or matched for each individual patient. Celularity is focused on this highly-scalable platform of technology to optimize economics and access to these cutting-edge therapies, including immuno-oncology. These placental stem cells allow for unprecedented scalability for Celularity’s CAR-T and CAR-NK platforms.

"Celularity’s technology has the potential to augment human immunity and longevity," said Dr. Peter H. Diamandis, Co-Founder and Vice-Chairman of Celularity. "The company’s ultimate vision is to make 100 years old the new 60, providing people with maximal cognition, mobility and aesthetics as they age. The 20 years of science, research, and intellectual property pioneered by Dr. Bob Hariri has the highest potential to become the cornerstone for this vision."

Andrew von Eschenbach, MD, among the founding members of the Celularity Board of Directors, and the former United States Food and Drug Administration (FDA) Commissioner and Director of the National Cancer Institute, said, "The pioneering work of Celularity founder Bob Hariri has unleashed the unique properties of placental derived stem cells, which have renewed hope for creating safe and effective therapies for the most challenging degenerative diseases." Dr. von Eschenbach added, "Celularity, with its focus on accelerating innovation in regenerative medicine, can become the leading catalyst for cell therapy to address many of the world’s unmet medical needs."

"Our investment in Celularity brings together its proprietary allogeneic placental platform and Sorrento’s best in class CAR-T products and cGMP manufacturing capabilities," said Henry Ji, another founding member of Celularity’s Board of Directors and Chairman, President and CEO of Sorrento Therapeutics. "Together, we can deliver these health solutions at scale to treat exponentially more people than we ever thought possible."

Kura Oncology Provides Update on Phase 2 Trial of Tipifarnib in HRAS Mutant Head and Neck Cancer

On February 15, 2018 Kura Oncology, Inc. (Nasdaq:KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported the presentation of updated preliminary results from its Phase 2 clinical trial of its lead product candidate, tipifarnib, in patients with head and neck squamous cell carcinomas (HNSCC) with HRAS mutations (Press release, Kura Oncology, FEB 15, 2018, View Source [SID1234524150]).

The results are being presented by Alan Ho, M.D., Ph.D., of Memorial Sloan Kettering Cancer Center at the 2018 Multidisciplinary Head and Neck Cancers Symposium, taking place today in Scottsdale, Arizona. A copy of the poster is available online at www.kuraoncology.com.

Nine patients with HRAS mutant HNSCC had been enrolled in the Phase 2 trial as of the February 8, 2018 data cutoff date. Five out of the six evaluable patients achieved a confirmed, partial response as defined by standard RECIST criteria for an overall response rate of 83% (36-99.6%, 95%CI), including durable responses of more than 18 months in two patients. The sixth evaluable patient experienced tumor shrinkage and prolonged disease stabilization. Three additional patients were enrolled since the last update in October 2017, however, none of the three additional patients was evaluable as of the February 8th data cutoff date; two are off study and one was still too early for disease assessment.

"We continue to be very encouraged by the high level of clinical activity of tipifarnib observed in patients with HRAS mutant HNSCC," said Antonio Gualberto, M.D., Ph.D., Head of Development and Chief Medical Officer of Kura Oncology. "In addition to rapid responses in patients who do not appear to benefit from current standards-of-care, we are also seeing dramatic resolution of disfiguring and often painful lesions. We are continuing to enroll patients in the ongoing Phase 2 trial while we gather input from regulatory agencies on the design of a potential registrational trial of tipifarnib in HRAS mutant HNSCC anticipated to be initiated later this year."

All patients joined the trial upon progression from at least one line of therapy, including chemotherapy, cetuximab or immune therapy, with patients having experienced a median of two prior therapies (range, 1-4). Response rates for the three agents approved for treatment of HNSCC in the second line are in the range of 13-16%, with a median progression-free survival of approximately 2 months and a median overall survival of up to 7.5 months.

Tipifarnib was generally well-tolerated in the trial. Adverse events observed are consistent with the known safety profile of tipifarnib.

In September 2017, Kura announced that the Phase 2 trial in HRAS mutant HNSCC had achieved its primary efficacy endpoint prior to the completion of enrollment. The trial protocol required four confirmed, partial responses, per RECIST 1.1 criteria, out of 18 patients to meet its primary endpoint.

About Tipifarnib

Kura Oncology’s lead candidate, tipifarnib, is an inhibitor of farnesylation, a key cell signaling process implicated in cancer initiation and development. In extensive clinical trials, tipifarnib has shown a well-established safety profile and compelling and durable anti-cancer activity in certain patient subsets. Preclinical and clinical data suggest that, in the appropriate context, tipifarnib has the potential to provide significant benefit to cancer patients with limited treatment options. Leveraging advances in next-generation sequencing as well as emerging information about cancer genetics and tumor biology, Kura Oncology is seeking to identify patients most likely to benefit from tipifarnib. In addition to its development program in solid tumors with HRAS mutations, Kura has identified potential biomarkers of activity for tipifarnib in hematologic malignancies, including peripheral T-cell lymphomas (PTCL), myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML).

Selumetinib Granted Orphan Drug Designation by the U.S. FDA for Neurofibromatosis Type 1

On February 15, 2018 -AstraZeneca and Merck (NYSE:MRK), known as MSD outside the U.S. and Canada, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1) (Press release, Merck & Co, FEB 15, 2018, View Source [SID1234524008]).

NF1 is an incurable genetic condition that affects one in 3,000 births with highly-variable symptoms including cutaneous (skin), neurological (nervous system) and orthopedic (skeletal) manifestations. NF1 can cause secondary complications including learning difficulties, visual impairment, pain, disfigurement, twisting and curvature of the spine, high blood pressure and epilepsy. Plexiform neurofibromas (PNs) are tumors that arise from nerve fascicles and tend to grow along the length of the nerve. PNs, a neurological manifestation of NF1,occur in approximately 20-50 percent of NF1 patients causing pain, motor dysfunction and disfigurement.

Sean Bohen, executive vice president, global medicines development and chief medical officer, AstraZeneca, said, "Neurofibromatosis type 1 is a devastating condition that can lead to life-threatening complications. There is no known cure for neurofibromatosis and there are limited treatment options to manage symptoms."

Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories, said, "This is an important collaborative effort with our colleagues at AstraZeneca addressing an area of significant unmet medical need to potentially benefit patients with neurofibromatosis type 1."

The potential benefit of selumetinib in NF1 is being explored in the U.S. National Cancer Institute-sponsored phase 1/2 SPRINT trial in pediatric patients with symptomatic NF1-related PNs. Phase II trial results are expected later in 2018.

The FDA’s ODD program provides orphan status to medicines that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S.

In addition to NF1, selumetinib is being investigated in the phase 3 ASTRA trial of patients who are diagnosed with differentiated thyroid cancer (DTC) following surgery and treatment with radioactive iodine. Selumetinib was granted ODD by the US FDA for the adjuvant treatment of stage 3/4 DTC in 2016. It is also being explored as a monotherapy and in combination with other treatments in phase 1 trials.

NOTES TO EDITORS

About neurofibromatosis type 1 (NF1)

The NF1 gene provides instructions for making a protein called Neurofibromin. The disease is associated with many symptoms, including soft lumps on and under the skin (subcutaneous neurofibromas), skin pigmentation (cafe au lait spots) and, in 20-50 percent of patients, tumors on the nerve sheaths (plexiform neurofibromas). These plexiform neurofibromas can cause morbidities such as pain, motor dysfunction and disfigurement. Patients with NF1 may experience a number of other complications such as learning difficulties, visual impairment, twisting and curvature of the spine, high blood pressure, and epilepsy. People with NF1 also have an increased risk of developing other cancers, including malignant brain and peripheral nerve sheath tumors, and leukaemia. Symptoms begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.

About selumetinib

Selumetinib, is an investigational MEK 1/2 inhibitor licensed by AstraZeneca from Array BioPharma Inc. in 2003.

The NF1 gene codes for a protein called Neurofibromin. This protein negatively regulates the RAS/MAPK pathway, which helps to control cell growth, differentiation and survival. Mutations in the NF1 gene may result in dysregulation in RAS/RAF/MEK/ERK signaling, which can cause cells to grow, divide and copy themselves in an uncontrolled manner, and may result in tumor growth. Selumetinib inhibits the MEK enzyme in this pathway, potentially leading to inhibition of tumor growth.