On October 5, 2017 Apogenix AG, a biopharmaceutical company developing next-generation immuno-oncology therapeutics, reported that its lead product candidate, asunercept (APG101), has been granted orphan designation from the European Commission (EC) for the treatment of myelodysplastic syndromes (MDS) (Press release, Apogenix, OCT 5, 2017, View Source [SID1234524529]). MDS is a bone marrow disorder characterized by ineffective hematopoiesis (blood cell formation) and can lead to severe anemia. Patients often suffer from life-threatening infections and are at risk of developing acute myeloid leukemia.

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Orphan designation includes access to a centralized marketing authorization procedure for the European Union, ten years of protection from market competition with similar medicines in similar indications and fee reductions for consultations with the EMA. Earlier, asunercept received Orphan Drug Designation for MDS from the US Food and Drug Administration (FDA).

Dr. Harald Fricke, Chief Medical Officer of Apogenix, commented: "The vast majority of patients suffering from MDS are anemic and dependent on frequent regular blood transfusions. Asunercept prevents premature death of red blood cells in the bone marrow and thus reduces the need of blood transfusions, even making them superfluous in many patients. We are highly encouraged by the data from our clinical phase I trial with asunercept in these patients and are currently preparing to initiate a clinical phase II proof-of-concept trial to further evaluate the efficacy of asunercept in MDS."

Asunercept has been evaluated in an open label, single-arm phase I clinical trial in 20 patients with low to intermediate risk MDS, in which treatment with asunercept was well tolerated and led to a significant decrease in transfusion frequency. In addition, investigation of parameters involved in erythropoiesis delineated how asunercept stimulates the production of red blood cells in these patients.

Asunercept binds to the CD95 ligand (CD95L) and blocks the activation of the CD95 receptor. Excessive stimulation of the CD95 receptor on hematopoietic precursor cells in the bone marrow of MDS patients inhibits erythropoiesis. As a result, transfusion-dependent anemia develops, which is refractory to erythropoiesis-stimulating agents. Treatment with asunercept, which inhibits the CD95 system, addresses this major cause of the disorder.

About Myelodysplastic Syndromes (MDS)
MDS is a bone marrow disorder that is characterized by ineffective hematopoiesis and can lead to severe anemia. In most cases, the anemia is treated with blood transfusions that eventually result in an iron overload, which can damage the liver and other organs. At the same time, the number of thrombocytes that are responsible for coagulation and the number of leucocytes that are responsible for immune defense significantly decreases in patients with this disorder. As a result, MDS patients frequently suffer from sudden bleeding and life-threatening infections. In addition, they are at risk of developing acute myeloid leukemia, a type of blood cancer.

About asunercept (APG101)
Apogenix’ lead immuno-oncology candidate asunercept is a fully human fusion protein that consists of the extracellular domain of the CD95-receptor and the Fc domain of an IgG1 antibody. Asunercept is being developed for the treatment of solid tumors and malignant hematological diseases. The World Health Organization (WHO) has assigned the international nonproprietary name (INN) "asunercept" for APG101.

On October 5, 2017 the National Institute for Health and Care Excellence(NICE) reported a final appraisal determination (FAD) recommending that AbbVie’s VENCLYXTO(venetoclax) is made available to NHS patients with difficult-to-treat types of chronic lymphocyticleukaemia (CLL) via the Cancer Drugs Fund (CDF), providing conditions of the managed accessagreement are followed (Press release, PharmaTimes, OCT 5, 2017, View Source [SID1234520814]). Venetoclax will now be available on the NHS to adult patients in Englandwith CLL in the absence of 17p deletion or TP53 mutation who have failed bothchemo-immunotherapy and a B-cell receptor (BCR) inhibitor. Venetoclax has also beenrecommended for the treatment of adult CLL patients in the presence of 17p deletion or TP53mutation who are either unsuitable for or have failed a BCR inhibitor.2 Please see the NICE websitefor the eligibility criteria: View Source appraisal-determination-document.

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Today’s recommendation marks the continuation of patient access across the UK, following therecent acceptance of venetoclax for use across NHS Scotland this August.

The immediate inclusion of venetoclax in the Cancer Drugs Fund is a positive step forward forpatients with CLL in England" commented David Innes, Chair of the CLL Support Association. "Accessto new treatment options is vital for patients with challenging forms of CLL, who have a short lifeexpectancy after exhausting current treatment options. We are pleased to see AbbVie and NICEworking together to expedite patient access and are hopeful that this will ultimately translate intolonger-term routine prescribing on the NHS, providing an essential treatment option for those livingwith CLL and their families."

Venetoclax, is a first-in-class, oral, once-daily medicine that selectively inhibits the function of theBCL-2 protein, restoring the body’s ability to trigger cancer cell self-destruction.2 For those patientsliving with CLL requiring treatment, the majority will eventually have their disease recur,3 with one intwo patients failing current treatments facing survival as short as three months.4,5 Venetoclax isbeing developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, amember of the Roche Group, in the U.S. and by AbbVie outside of the U.S.

Dr Peter Hillmen, Professor of Experimental Haematology and Honorary Consultant Haematologistat Leeds Teaching Hospitals NHS Trust, commented, "Today’s recommendation is great news forpatients with CLL who have failed existing treatments, and provides clinicians with an important newtreatment option. The studies that NICE has assessed to reach this positive decision represent a 4UB 2 milestone in the management of relapsed/refractory CLL. The early clinical data is compelling,showing survival benefits for this challenging group of patients, including some who achievedcomplete remission. I would anticipate that collection of further data through the CDF will confirmthese extremely promising early findings."

CLL affects the blood and immune system and is the most common form of adult leukaemia withalmost 3,500 people affected in the UK each year, with over 3,000 cases in England alone.6,7 Forpeople who develop or harbour gene mutations, such as 17p deletion and/or TP53 mutation,treatment is particularly challenging and these are associated with poorer quality of life and amedian life expectancy of less than two to three years with current standard-of-care regimens.

In a Phase 2 study (M13-982) of 158 patients with relapsed and/or refractory CLL with a 17pdeletion, the overall response rate was 77.2% (122/158) according to investigator assessment.10,11Based on Kaplan-Meier estimations, 86.7% of patients were estimated to be alive following 12months of treatment.11 In a separate Phase 2 two arm study (M14-032) of venetoclax in 64 CLLpatients who relapsed or were refractory to BCR inhibitors (ibrutinib or idelalisib), the primaryendpoint, overall response rate, was 67% and 57% respectively, according to investigatorassessment.11 Venetoclax has also demonstrated early and sustained improvements in fatigue, adebilitating symptom of CLL, with reductions observed at just 4 weeks.

A recent study supports the use of Minimal Residual Disease (MRD) negativity as a prognosticmarker for long-term progression-free survival and as a potential therapeutic goal in CLL. MRDnegativity describes the presence of a small number of leukaemic cells that remain followingtreatment and is defined as <1 CLL cell detectable per 10,000 leukocytes.13,14 In a Phase 2 study inpatients with relapsing and refractory CLL with the del(17p) gene mutation, a high risk prognosticfactor, MRD was used as an exploratory endpoint. Of 158 patients who were treated withvenetoclax, 24% of patients (38/158) achieved MRD negativity in the peripheral blood, including 16patients who were also MRD negative in the bone marrow.

Venetoclax was the first blood cancer medicine to be given positive scientific opinion through theEarly Access to Medicines Scheme (EAMS), following its designation as a Promising InnovativeMedicine (PIM) by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA)designation.

4UB 3As part of AbbVie’s ongoing focus on delivering breakthrough medicines, it has worked with theMHRA and NHSE to provide 50 patients in the UK with early access to venetoclax via EAMS. OnceEAMS ceased, AbbVie made a commitment to providing the treatment free of charge untilreimbursement. Through a combination of EAMS and free of charge supply, approximately 100patients with a high unmet need have benefitted from early access to venetoclax.

Xspray Pharma har fått godkännande för två sökta patent i Japan (Press release, Xspray, OCT 4, 2017, View Source [SID1234523283]). Patenten gäller dels komposition, dels metod, avseende produktkandidaten HyNap-Dasa. Det är Xsprays första godkännande på en av de tre strategiska huvudmarknaderna USA, Europa och Japan. Bolaget har pågående ansökningsärenden för motsvarande patent för samtliga tre produktkandidater i USA, Europa och Japan.

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"Att vi nu får patent beviljade bekräftar att de finns en unik och skyddsvärd innovationshöjd i vår teknologi och i våra produktkandidater," säger Per Andersson, vd för Xspray Pharma.

Xspray Pharma har erhållit godkännande ("patent allowance") för två sökta patent i Japan avseende produktkandidaten HyNap-Dasa som är tänkt för behandling av vissa cancerformer. Det är de två första patenten som beviljas för bolagets produktkandidater på en viktig och kommersiellt intressant marknad. Beskedet kommer i enlighet med bolagets plan att söka patent för komposition och metod för samtliga tre produktkandidater under utveckling på de tre viktigaste marknaderna, USA, Europa och Japan.

"Vi satsar primärt på att lansera våra produktkandidater på den amerikanska marknaden, men vi vill samtidigt bygga upp en kommersiell beredskap för att dra nytta av möjligheter som kan öppna sig på andra viktiga marknader, främst Japan och Europa. Där fyller vår patentstrategi en viktig funktion," kommenterar Xsprays vd Per Andersson.

Xspray Pharmas aktier introducerades den 26 september på Nasdaq First North, efter en lyckosamt genomförd nyemission som tillförde bolaget 132 miljoner kronor före emissionskostnader. Planen är nu att använda kapitalet för att utveckla tre produktkandidater och blivande cancerläkemedel baserade på bolagets egenutvecklade teknologi, samt att introducera de första produkterna på den amerikanska marknaden under perioden 2020-2023.

On October 4, 2017 Selexis SA and Pelican Therapeutics ("Pelican"), a subsidiary of Heat Biologics, Inc. ("Heat") (Nasdaq: HTBX), reported that they have entered into a service agreement to advance the development of Pelican’s proprietary immunotherapy clinical candidates (Press release, Selexis, OCT 4, 2017, View Source [SID1234520784]). Under the agreement, Pelican intends to leverage Selexis’ proprietary SUREtechnology PlatformTM to rapidly develop high-performance research cell banks (RCBs) expressing two of Pelican’s clinical candidates, including PTX-35, a humanized monoclonal antibody that is a functional agonist of human TNFRSF25, and PTX-15, a human TL1A-Ig fusion protein. Both candidates have the potential to improve clinical response when used in combination with Heat’s ImPACT therapeutic platform and other immunotherapy drugs.

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"With immunotherapies dominating the treatment landscape, we continually find that our technology can help our partners, such as Pelican, advance candidates for unmet medical needs including certain cancers," said Yemi Onakunle, PhD, MBA, Selexis vice president, licensing and business development. "By combining our ability to rapidly produce high-expressing and stable research cell banks with KBI Biopharma’s strong development, production and analytical capabilities, we are able to reliably and predictably support Pelican’s clinical development and provide the fastest timelines to the eventual commercialization of both clinical candidates."
In June 2017, JSR Corporation acquired Selexis SA to bring best-in-class cell line development technology to JSR Life Sciences, its life sciences division. JSR Life Sciences is now offering industry partners best-in-class contract development manufacturing organization (CDMO) services by complementing its commercial subsidiary KBI Biopharma with Selexis technologies.

Rahul Jasuja, CEO of Pelican, added: "We chose Selexis for their expertise in cell line expression technologies for recombinant protein therapeutics for the CMC development of our two molecules, PTX-35 and PTX-15. We look forward to advancing our TNFRSF25 agonist program with both KBI Biopharma and Selexis as we prepare for our first-in-human clinical trial."
PTX-35, Pelican’s lead product candidate, is a novel monoclonal antibody against TNFRSF25, an emerging costimulatory receptor on T cells. The agent provides highly selective and potent stimulation of ‘memory’ CD8+ cytotoxic T cells, a class of T cell that is responsible for eliminating tumor cells in patients. PTX-35 has the potential to enhance durability of antigen-specific immune responses in combination with other immunotherapies.

Selexis’ proprietary SUREtechnology Platform facilitates the rapid, stable, and cost-effective production of virtually any recombinant protein and provides seamless integration of the biologics development continuum, spanning discovery to commercialization.

On October 4, 2017 — Palleon Pharmaceuticals, a company focused on developing the first Glycoimmune Checkpoint Inhibitors to treat cancer, reported the completion of a $47.6 million Series A financing from leading biotech venture investors SR One, Pfizer Ventures, Vertex Ventures HC, Takeda Ventures, and AbbVie Ventures (Press release, Palleon Pharmaceutical, OCT 4, 2017, View Source [SID1234520792]).

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The Series A funding will be used to establish and advance a first-in-class pipeline of drug candidates targeting Glycoimmune Checkpoints — receptors on immune cells that normally distinguish "self" versus "non-self" but get exploited in cancer to create immunosuppression that allows tumors to thrive. Glycoimmune Checkpoints function in ways similar to T cell checkpoints, with a few important differences: they are activated by binding to cell-surface glycans — the sugar molecules found on cell surfaces — rather than to other proteins, and they are expressed in a wider range of immune cells involved in the anti-cancer response, including both innate and adaptive immune cells. In addition, Glycoimmune Checkpoints appear to be exploited by most types of cancer, and a tumor’s unique glycan signature offers the prospect of selecting patients who are likely to respond to therapy.

Palleon has exclusive licenses to the intellectual property of discoveries made by its scientific co-founders, Carolyn Bertozzi, PhD, the Anne T. and Robert M. Bass Professor of Chemistry at Stanford University and an Investigator of the Howard Hughes Medical Institute, and Paul Crocker, PhD, Professor of Glycoimmunology and Head of the Division of Cell Signaling and Immunology at the University of Dundee, Scotland. Bertozzi’s and Crocker’s scientific discoveries in the separate fields of tumor glycoscience and human immunology were brought together to create a unique platform which enables Palleon to develop cancer drugs targeting Glycoimmune Checkpoints.

Jim Broderick, MD, Chief Executive Officer and Founder of Palleon, commented, "The most meaningful breakthroughs often occur at the intersection of diverse and seemingly unrelated scientific disciplines. Palleon was spawned by bringing together new findings in glycoscience and human immunology, which resulted in unexpected implications for oncology. The convergence of these two fields has enabled us to develop a novel class of medicines that could have a significant impact on the lives of cancer patients."

"We have known for decades that certain glycan patterns such as hypersialylation appear on the surface of tumors, and that these patterns are correlated with poor clinical outcomes; however, we did not understand the functional role of these tumor-specific glycans in immunosuppression," said Dr. Bertozzi. "We now know that tumors evolve in such a way that their cell surface glycans "trick" the immune system, which prevents many types of immune cells from detecting and destroying cancer cells. We are using this knowledge to develop new and innovative cancer therapies for patients."

Palleon’s Convergence Platform is driven by the combined expertise of its world-class founding team in tumor glycoscience and human immunology. The company has incorporated a range of technologies to overcome the historic scientific barriers that have made glycoscience a challenging area of study. Armed with a unique understanding of the differences in immune pattern recognition between humans and other species, the company has integrated human biology into every step of the drug development process, including target validation, in vitro models, in vivo models, and translational research. The company has assembled the core expertise needed to pioneer a new field in this complex area of biology, and its proprietary knowledge will define Palleon’s leadership in this rapidly emerging area.

Palleon was originally incubated in the Cambridge offices of SR One, where Dr. Broderick served as the firm’s first Entrepreneur-in-Residence, working closely with SR One President Jens Eckstein. "We are very excited about the launch of Palleon. Within the dynamic field of immuno-oncology, Palleon has forged an entirely new pathway for targeting the immune system. The unique features of Glycoimmune Checkpoints will make possible a much wider range of rational combination therapies to treat cancer," said Dr. Eckstein. "Palleon is built on the discoveries of its exceptional scientific founders who have identified Glycoimmune Checkpoints as a means to transform the treatment of cancer and improve and extend the lives of patients throughout the world."
About Palleon Pharmaceuticals

Palleon Pharmaceuticals is the leading biotechnology company focused on developing Glycoimmune Checkpoint inhibitors to treat cancer. The company’s proprietary Convergence Platform integrates technologies and insights from world-renowned scientific leaders in the fields of glycoscience and human immunology to create a novel approach to treating cancer. By targeting multiple immune cell types, Glycoimmune Checkpoint inhibitors will tackle resistance to first-generation immuno-oncology agents, and make possible a wider range of rational combination therapies to treat cancer. While Palleon is focused primarily on oncology, the Convergence Platform is applicable to other therapeutic areas including infectious diseases, neurodegeneration, inflammation, and fibrosis. The company is advancing its pipeline and development programs with a $47.6 million Series A financing from leading biotech venture investors SR One, Pfizer Ventures, Vertex Ventures HC, Takeda Ventures, and AbbVie Ventures. Learn more at www.palleonpharma.com.