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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

On October 4, 2017 Neon Therapeutics, an immuno-oncology company developing neoantigen-based therapeutic vaccines and T cell therapies to treat cancer, reported that the company has entered into an exclusive license agreement with the Netherlands Cancer Institute (NKI) for technology to be utilized in Neon Therapeutics’ personalized neoantigen T cell therapy program, NEO-PTC-01 (Press release, Neon Therapeutics, OCT 4, 2017, View Source [SID1234527449]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Under a collaborative research agreement established in 2016, Neon Therapeutics and the NKI have developed an ex vivo induction protocol which produces autologous T cells targeting each patient’s own neoantigens. This collaborative research has been led at the NKI by John Haanen, M.D., Ph.D., professor and head of the Medical Oncology Division at NKI, and Joost van den Berg, Ph.D., director of the Cell Therapy facility, with support from Neon co-founder Ton Schumacher, Ph.D., professor and senior member of the Division of Molecular Oncology & Immunology. Neon Therapeutics has now licensed intellectual property and know-how from NKI to support further development of NEO-PTC-01 into clinical development.

NEO-PTC-01 is an autologous T cell therapy, where a proprietary induction protocol is used to induce multiple T cell populations specific for multiple neoantigen targets. Neon Therapeutics will continue to work with the NKI to prepare for the initiation of a phase 1 clinical study by the end of 2018.

"Our collaboration with the Netherlands Cancer Institute has been a productive one, bringing in complementary capabilities to accelerate the development of Neon Therapeutics’ personalized T cell program," said Richard Gaynor, president of research and development at Neon Therapeutics. "We are excited to enter the next phase of NEO-PTC-01 development, and look forward to continuing to work with the NKI to scale-up the GMP manufacturing process."

"We are very happy to see our collaboration with Neon Therapeutics entering the next phase. It nicely confirms that our efforts to exploit new opportunities for immunotherapy are quickly moving in the right direction," said Prof. Dr. Rene Medema, director of research at the NKI.

"We have made great progress working with Neon Therapeutics to develop this technology to induce de novo neoantigen-based T cell responses," said Prof. Dr. Haanen. "We are pleased that Neon Therapeutics will continue to develop these technologies to bring the promise of fully personalized cell therapies to cancer patients."

A stealth start-up called 3T Biosciences has raised more than $12 million in a massive seed round led by early Facebook investor Sean Parker and including Peter Thiel, according to a person familiar with the company’s financing (External Source, 3T Biosciences, OCT 4, 2017, View Source [SID1234525823]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Little is known about 3T Biosciences, but this person said the company is led by cancer biology PhD student and investor, Asset Management Ventures’ principal Luke Lee, as well as a group of academics from Christopher Garcia’s biology lab at Stanford.

3T Biosciences is in the T-cell therapy space, the person said, meaning that it is among a growing group of new start-ups working to bolster the body’s own immune system to fight cancer. That’s a different approach than traditional medicines like chemotherapy, which kill off both cancerous and healthy cells.

Sean Parker speaking at the 2015 CGI Annual Meeting in New York.
Adam Jeffery | CNBC
Sean Parker speaking at the 2015 CGI Annual Meeting in New York.
These approaches have not proved successful for all cancer patients and are still early. Juno Therapeutics, a Seattle-based company in the space, needed to halt development for its drug therapy after three patient deaths.

Still, Silicon Valley’s venture investors see an opportunity to make a return with Gilead Sciences’ recently acquiring cancer immunotherapy company Kite Pharma for $11.9 billion. That follows Abbvie’s $10.2 billion acquisition of Founders Fund-backed cancer drug start-up Stemcentrx in 2016.

These venture firms also see potential to combine traditional drug therapeutics with new technologies like machine learning in the hopes of accelerating the timeline and reducing the cost of bringing a new drug to market.

The round was led by Parker through his venture fund, the person said, with participation from Thiel Capital and several other firms.

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.