On October 5, 2016 SYNCONA LLP and CANCER RESEARCH TECHNOLOGY (CRT) reported the formation of Achilles Therapeutics Ltd (Press release, Achilles Therapeutics, OCT 5, 2016, View Source [SID1234523124]).

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The new private company will bring together world-class research from UCL (University College London) and the Francis Crick Institute, funded by Cancer Research UK and the National Institute for Health Research (NIHR).

Achilles Therapeutics will design therapies to target truncal tumour neo-antigens – unique flags to the immune system present on the surface of every cancer cell*, which were first discovered by Cancer Research UK and the NIHR University College London Hospitals (UCLH) Biomedical Research Centre (BRC) funded scientists at the Francis Crick Institute and UCL Cancer Institute.

Truncal tumour neo-antigens are present on all cancer cells in an individual patient’s tumour but not on healthy cells, so could allow scientists to target and destroy tumours without harming healthy tissues.

Syncona and CRT, with the support of UCL Business (UCLB) and the Crick, formed Achilles Therapeutics with a successful financing round of £13.2 million ($17.5 million) led by Syncona with the CRT Pioneer Fund and the UCL Technology Fund.

The company founders bring together world-class capability from three prestigious institutions. They are:

Professor Charles Swanton, Group Leader and Royal Society Napier Professor at the Francis Crick and UCL Cancer Institute working on cancer evolution and genome instability and a consultant at UCLH
Professor Karl Peggs, Group Leader of the Stem Cell Transplantation and Cellular Immunotherapy Group at UCL Cancer Institute and a consultant at UCLH
Dr Sergio Quezada, Group Leader of the Immune Regulation and Tumour Immunotherapy Group at UCL Cancer Institute
Professor Mark Lowdell, Director of the Centre for Cell, Gene & Tissue Therapeutics at the Royal Free Hospital.

CRT will receive equity milestones and royalties from products developed and commercialised by Achilles Therapeutics. Any such financial reward from the company will be shared with UCLB and the Crick.

The company has exclusive rights to develop and commercialise neo-antigen technologies arising from Cancer Research UK’s £14million TRACERx study**. This clinical study, involving 850 people with non-small cell lung cancer, tracks the evolution of patients’ cancers over time, in different parts of their tumours and in response to treatment. It receives infrastructure support from the NIHR University College London Hospitals BRC and is being carried out at the Clinical Research Facility at UCLH.

Professor Charles Swanton, scientific founder of Achilles Therapeutics and a Group Leader at the Francis Crick Institute, said: "Our research could provide a truly personalised approach to lung cancer therapy by targeting cell surface markers that are specific to each patient and present on all cancer cells rather than just a subset of cells. We’re delighted to be able to bring this exciting science closer to the clinic. We hope to create a new and kinder treatment for this hard-to-treat disease that results in around 36,000 patient deaths each year in the UK ***."

Iraj Ali, Partner with Syncona LLP and Director of Achilles Therapeutics, said: "In founding Achilles we believe we are working with the world leaders capable of exploiting the confluence of two of the most exciting and innovative fields in healthcare today: cancer bioinformatics and immuno-therapy. Our ambition is to build a company to deliver personalised therapies with transformative potential for cancer patients with the greatest need."

Chris Ashton, CEO of Achilles Therapeutics, said: "This company is underpinned by world-leading science, committed investors and leading health institutes. Bringing all of these major players together holds great promise for non-small cell lung cancer patients and I hope that working alongside one another we will see great successes in the future."

On October 5, 2016 BioLineRx Ltd. (NASDAQ/TASE: BLRX), a clinical-stage biopharmaceutical company dedicated to identifying, in-licensing and developing promising therapeutic candidates, reported that data on BL-8040, the Company’s leading oncology platform, have been accepted for presentations at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exhibition in San Diego, California, taking place December 3-6, 2016 (Filing, 6-K, BioLineRx, OCT 5, 2016, View Source [SID:SID1234515592]).

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Information on the BL-8040 abstracts presented at ASH (Free ASH Whitepaper) is included below:

· (Abstract #767) Oral Presentation: The High Affinity CXCR4 Inhibitor, BL-8040, Induces Apoptosis of AML Blasts and their Terminal Differentiation by Blocking AKT/ERK Survival Signals and Downregulating BCL-2, MCL-1 and Cyclin-D1 through Regulation of Mir-15a/16-1 Expression; Monday, December 5, 2016, 11:30 am PST; Marriott Marquis San Diego Marina Hotel, San Diego, Ballroom AB

· (Abstract #2745) Poster Presentation: The Selective Anti-Leukemic Effect of BL-8040, a Peptidic CXCR4 Antagonist, is Mediated by Induction of Leukemic Blast Mobilization, Differentiation and Apoptosis: Results of Correlative Studies from a Ph2a Trial in Acute Myeloid Leukemia; Sunday, December 4, 2016, 6:00-8:00 pm PST; San Diego Convention Center, Hall GH.

About BL-8040
BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and certain hematological indications. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells from the bone marrow, thereby sensitizing these cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing apoptosis. In addition, BL-8040 has also demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, and T, B and NK cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

On October 4, 2016 EUSA Pharma (EUSA), a specialty pharmaceutical company with a focus on oncology and oncology supportive care, reported the acquisition of exclusive global commercialization rights to the oncology product dinutuximab beta from Apeiron Biologics (Press release, EUSA Pharma, OCT 4, 2016, View Source [SID1234527664]). Dinutuximab beta is currently used as part of the regimen for the treatment of high risk neuroblastoma in Europe and is available under a managed access program. The immunotherapy has orphan drug designation in the US and EU and is currently under review for marketing authorization by the EMA. EUSA expects to file the product for registration in the US and Japan in 2017.

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Under the terms of the agreement, EUSA Pharma will pay Apeiron an upfront fee, with a portion contingent on EU approval. EUSA will also pay regulatory milestone payments in other key territories and royalties on future product sales.

Neuroblastoma is an orphan oncology indication with significant unmet medical need. It accounts for up to 10% of childhood tumors and affects approximately 1,200 children in the EU5 and US each year. Consequently, EUSA Pharma intends to continue dinutuximab beta’s managed access program, and once approved in Europe will promote the immunotherapy to oncologists through its specialty sales team. In the United States, the company plans to submit a regulatory filing in 2017, and once approved will commercialize the product directly through its established US infrastructure. In other territories, including Japan, EUSA plans to bring the product to market through its international network of partners.

"We are delighted to acquire the global rights to dinutuximab beta, which is a perfect fit with our strategic focus in the specialty oncology field and will allow us to leverage our commercial infrastructure in the EU and expand our presence in the US," said Lee Morley, EUSA Pharma’s Chief Executive Officer. "Dinutuximab beta is already used extensively across Europe, where it is included in a number of treatment protocols, and we look forward to bringing this life saving therapy to patients around the world. As a rapidly growing specialty pharma company we have made great progress since our launch 18 months ago, and we plan to continue this through further product acquisition and in- licensing."

"EUSA Pharma is the ideal partner to bring dinutuximab beta to market, with its strong focus on oncology and specialty commercial expertise in Europe, the US and further afield," said Dr. Hans Loibner, Apeiron Biologic’s Chief Executive Officer. "Dinutuximab beta is an important treatment in an area of significant unmet need, which we have developed together with our academic partners, in particular with the cooperative group SIOPEN, and we look forward to working with EUSA to make this product available around the world."

On October 4 , 2016 – Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company focused on discovering and developing highly targe ted cancer immunotherapies, announced today the presentation of preclinical data on the Company’s lead candidate AFM13 , a bispecific NK – cell – engaging TandAb targeting CD30/CD16A , at the 16th Annual Meeting of the Society for Natural Immunity in Taormina, Italy (Press release, Affimed, OCT 4, 2016, View Source [SID1234515989]).

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The data, generated in Affimed’s collaboration with the Innate Immunity Group of Dr. Adelheid Cerwenka at the German Cancer Research Center (DKFZ) in Heidelberg, Germany, were presented in a poster ti tled " The bispecific CD3 0/CD16A TandAb AFM13 amplifies the cytolytic and proliferative potential of NK – cells " yesterday, October 3, 2016 .

In this preclinical study, the specific phenotype and functionality of human NK – cells when redirected to AFM13 – coated tumor cells, as well as their responsiveness to cytokines , were analyzed. The results show that AFM13 improves NK – cell cytotoxicity against CD30+ tumor cells that are resistant to naïve NK – cells. Using CFSE – labelled NK – cells, the researchers demonstrated that AFM13 amplifies cytokine – mediated NK – cell proliferation and expansion by enhancing the NK – cells’ sensitivity to IL – 2 and IL – 15. These data indicate that cytokine administration in combination with AFM13 might potentially enhance NK – cell activity in the tumor microenvironment.

AFM13 is a tetravalent bispecific TandAb antibody that binds bivalently to both CD30 on tumor cells and CD16A on NK – cells. The molecule has been shown to engage NK – cells through CD16A with high affinity and specificity, resulting in strong NK – cell – mediated cytotoxicity. AFM13 is currently being tested in Hodgkin lymphoma patients as a monotherapy (Phase 2) and in combination with Merck’s Keytruda (Phase 1b).

On October 4, 2016 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that it has received a second Breakthrough Therapy Designation (BTD) from the United States Food and Drug Administration (FDA) for its ALK inhibitor, Alecensa (alectinib) (Press release, Hoffmann-La Roche , OCT 4, 2016, View Source [SID:SID1234515583]). The latest BTD was granted for the treatment of adult patients with advanced anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) who have not received prior treatment with an ALK inhibitor.

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"The J-ALEX study that supports the second Breakthrough Designation for Alecensa showed superior efficacy versus the standard of care, crizotinib, in Japanese people with advanced ALK-positive disease," said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development. "The decision by the FDA to grant a second breakthrough therapy designation is recognition of the clinically meaningful improvement in efficacy and safety that Alecensa brings to the care of people with advanced ALK-positive lung cancer who have not received prior treatment with an ALK inhibitor."

This second breakthrough therapy designation is based on the results of the open-label, randomised phase III J-ALEX study, which were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2016 Annual Meeting in June. J-ALEX compared the efficacy and safety of Alecensa with crizotinib in 207 Japanese people with ALK-positive, advanced or recurrent NSCLC who either had not been treated with chemotherapy or had received one prior line of chemotherapy. Results from the study demonstrated that Alecensa reduced the risk of disease worsening or death (progression-free survival, PFS) by 66% compared to crizotinib, whilst maintaining a favourable tolerability and safety profile consistent with that observed in previous studies.

The FDA’s Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat serious diseases and to help ensure patients have access to them through FDA approval as soon as possible. Alecensa received its first FDA BTD in June 2013 for people with ALK-positive NSCLC whose disease progressed on treatment with crizotinib.

Alecensa is currently available in the US and Israel to ALK-positive metastatic NSCLC patients who have progressed on or are intolerant to crizotinib, and in Japan to ALK-positive unresectable, recurrent or advanced NSCLC patients. In addition, an ongoing global, randomised phase III trial called ALEX is comparing Alecensa to Xalkori as an initial (first-line) treatment for people with advanced ALK-positive NSCLC.

About J-ALEX
The J-ALEX study conducted by Chugai is an open-label, randomised Phase III study that compared the efficacy and safety of Alecensa to crizotinib in Japanese people. The J-ALEX study enrolled 207 people with ALK-positive, advanced or recurrent NSCLC who had not been previously treated with an ALK inhibitor. People were randomised to the Alecensa group or the crizotinib group in a one-to-one ratio. Results include:

Alecensa reduced the risk of disease worsening or death (PFS) by 66 percent compared to crizotinib (HR=0.34, 99 percent CI: 0.17-0.70, p<0.0001).

Median PFS was not reached in the Alecensa arm (95 percent CI: 20.3 months-not estimated) versus 10.2 months in the crizotinib arm (95 percent CI: 8.2-12.0).

Grade 3-4 adverse events (AE) occurred with lesser frequency in the Alecensa arm compared to the crizotinib arm (27 percent vs. 51 percent).

The most common AE occurring with > 30 percent frequency with Alecensa was constipation (36 percent). The most common AEs for crizotinib were nausea (74 percent), diarrhoea (73 percent), vomiting (59 percent), visual disturbance (55 percent), alteration in taste (dysgeusia, 52 percent), constipation (46 percent), and an elevation in liver enzymes called alanine transaminase (ALT, 32 percent) and aspartate transaminase (AST, 31 percent).

About Alecensa
Alecensa (RG7853/AF-802/CH5424802) is an oral medicine created at Chugai Research Laboratories and is being developed for people with NSCLC whose tumours are identified as ALK-positive. ALK-positive NSCLC is often found in younger people who have a light or non-smoking history. It is almost always found in people with a specific type of NSCLC called adenocarcinoma. Alecensa is currently approved in the United States for the treatment of people with advanced (metastatic) ALK-positive NSCLC whose disease has worsened after, or who could not tolerate treatment with, crizotinib.

In two key phase II studies, NP28761 and NP28673, Alecensa shrank tumours in up to 44% of people with ALK-positive NSCLC who progressed on crizotinib. Alecensa also demonstrated activity in brain metastases, indicating that the drug may be taken up in the brain. The brain is protected by the Blood-Brain
Barrier, a network of tightly joined cells that line the inside of the blood vessels in the brain and spinal cord.

One of the ways the Blood-Brain Barrier prevents molecules from affecting the brain is to actively eject them from the barrier through a process known as ‘active efflux’. The active efflux system does not recognise
Alecensa, which means that it may travel into and throughout brain tissue.

The global phase III studies of Alecensa include a companion test developed by Roche Diagnostics. Alecensa is marketed in Japan by Chugai Pharmaceutical, a member of the Roche Group.

About ALK-positive non-small cell lung cancer
Lung cancer is the biggest cancer killer, causing 1.59 million deaths globally each year. NSCLC is the most common type of lung cancer, and is the leading cause of cancer-related deaths in Europe and across the world, accounting for approximately 85% of lung cancer cases. ALK-positive NSCLC occurs in approximately 5% of patients with advanced NSCLC, translating to about 75,000 patients being diagnosed with the disease annually. It is almost always found in people with a specific type of NSCLC called adenocarcinoma, and is more common in light or non-smokers.