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.

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]).

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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."

The prestigious Fierce 15 biotech award has come to symbolize novelty and being at the forefront of biotechnology development amongst privately held businesses. The winners of this award are aiming at breakthroughs and big things, not at being ‘me-too’.

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For years, 1stOncology has taken special interest in those awardees with positions in oncology or whose technologies we deem beneficial in the oncology area. With oncology being one of the fastest growing therapeutic fields, it comes as no surprise that eleven out of the fifteen 2017 Fierce 15 Biotech Award winners are oncology companies. The majority are U.S. based (8), originating from Massachusetts (4), California (2), New Jersey (1) and Texas (1). Two companies are based in Canada and one in the United Kingdom.

Awardee	Founded	Country
PsiOxus Therapeutics	2006	United Kingdom
PMV Pharma	2013	USA
Rubius Therapeutics	2014	USA
Vividion Therapeutics	2014	USA
Gritstone Oncology	2015	USA
Turnstone Biologics	2015	Canada
Aravive Biologics	2016	USA
Magenta Therapeutics	2016	USA
Relay Therapeutics	2016	USA
Repare Therapeutics	2016	Canada
Tango Therapeutics	2017	USA

These companies were all founded between 2006 and as recent as this year. PsiOxus Therapeutics is the oldest company with Tango Therapeutics being the new kid on the block. Most of the companies were founded in 2016.

Many of them have recently secured financing, like Magenta Therapeutics ($50 million (2017)), PMV Pharma ($74 million (2017)), Rubius Therapeutics ($120 million (2017)), Tango Therapeutics ($55 million (2017)) and Relay Therapeutics ($57 million (2016)).

Among the technologies and discovery engines of the above companies we find gene editing, oncolytic viruses, synthetic lethality and neoantigen-based immunotherapies etc. Target interest falls on traditional hot areas such as tumor adoptive responses, oncogenic drivers, tumor suppressor gene loss and immune evasion, but also new avenues are being explored like the Unfolded Protein Response (UPR) pathway etc.

Morover, Rubius Therapeutics is developing Red-Cell Therapeutics (RCTs) as a new class of medicines for the treatment of cancer and albeit not specifically talking about oncology, Vividion Therapeutics is developing drugs based on technology that radically expands  the druggability of the human proteome.

Interested in understanding and surveying the pipelines and progress of these best and brightest award winners, not only from this year, but back to 2010? We would like to welcome you to join us for a free 30 minute 1stOncology demo to show you the difference 1stOncology can make in your day to day work.

On October 3, 2017 Kancera reported that the company’s ROR inhibitor KAN0441571 effectively eliminates ROR1-bearing leukemic cells in a mouse model of human chronic lymphocytic leukemia (Press release, Kancera, OCT 3, 2017, View Source;releaseID=1373958 [SID1234520782]).

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The study was conducted in mice into which leukemia cells from a patient with chronic lymphocytic leukemia were introduced. Human leukemia cells were allowed to infiltrate the lymphatic system in mice for one week, as also occurs in humans. Thereafter treatment began, consisting of two doses of KAN0441571 given every 3 days. This dose range was chosen in view of the high effect of KAN0441571 measured in laboratory studies and the long circulation time of the substance that has been measured in blood (over 10 hours).

After 13 days (and four treatment days), the number of ROR1-bearing human cancer cells in the lymphatic system (spleen sample) decreased by about 50% compared to control-treated animals. This effect is statistically significant (P < 0.0005) as is also the desired decrease in spleen weight (P < 0.0002). The results also indicate that KAN0441571 was well tolerated and gave a long-acting effect against chronic lymphocytic leukemia in this disease model that closely resembles human disease.

Kanceras KAN0441571 acts on cancer cells mainly by causing cell death by self-destruction, so called apoptosis. The research that has resulted in the production of KAN440571 is based on an assay method that identifies ROR1 inhibitory substances. Thereafter, an optimization of the cell kill effect has been carried out using leukemic cells from patients (and corresponding cells from healthy donors). This optimization process has meant that the effect of substances like KAN0441571 has been spread to several target cells in the cancer cell. Kancera has been able to verify that the total effect of KAN0441571, in addition to an inhibition of ROR1, also includes regulation of mechanisms that control cell division and inflammation signals. This pattern of effects can help Kancera identify cancers that are particularly sensitive to the substance.

About the ROR project
ROR is a family of receptors: ROR-1 and ROR-2. The ROR receptors convey signals for growth and survival. Initially, ROR was linked to fetal development, but nowadays it is known that they also contribute to the development and spread of cancer cells. Kancera and Professor Håkan Mellstedt and his colleagues at Karolinska Institutet have shown that Kancera’s ROR inhibitor is capable of killing tumor cells from tumors in the pancreas, lungs, leukemia cells and multiple myeloma cells. Researchers have also shown that ROR is active and a target in prostate, breast and ovarian cancer.

Kancera and Professor Mellstedt have shown that inhibition of ROR causes cancer cells to eliminate themselves via cellular suicide. The efficiency of eliminating cancer cells has also been optimized against cells in patient samples. Thus, the ROR inhibitors have been directed at several targets in the cancer which together add up to the desired effect. In addition to effect against ROR1, Kancera has shown that the company’s ROR inhibitor also works by controlling cell division and inflammation signals. A more effective activity against cancer cells compared to healthy cells has been ensured by continuously investigating the pattern of effects in samples from patients and healthy donors. Against this background, there is reason to believe that a ROR-targeted drug is both safer and more effective than non-selective chemotherapeutic agents that are currently used to treat cancer.

On October 3, 2017 Vium reported the selection of OncoSynergy, a UCSF oncology therapeutics spinout, as the first recipient of its "Next Generation Disease Model Grant" of $100,000 worth of research services (Press release, OncoSynergy, OCT 3, 2017, View Source [SID1234520773]). The companies will collaborate on a study of OncoSynergy’s lead drug candidate OS2966 to facilitate development of an optimized and clinically relevant experimental model of malignant ascites (MA), a complication of metastatic ovarian cancer (OC) associated with poor prognosis. The study will be the first to collect in-depth quality of life (QOL) metrics using an established experimental model of MA.

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OncoSynergy has already demonstrated efficacy of OS2966, its lead monoclonal antibody therapeutic candidate designated as an Orphan Drug by the U.S. Food and Drug Administration (FDA), in multiple models of OC, including alleviation of clinical signs of MA. The new study will therefore use Vium’s Smart Housing to increase the therapeutic window to more closely model disease progression in humans, thereby yielding more accurate measurement of disease metrics and improved tracking of QOL measures.

"We are pleased to collaborate with OncoSynergy and to fund this innovative study in a disease area with high unmet research and treatment needs," said Vium Chief Scientific Officer David Hutto, Ph.D, DVM, DACVP. "It’s a great match for Vium, as it allows us to apply our fully automated, low-touch technology and novel digital metrics to continuously assess changes in ovarian cancer-related malignant ascites — signs that would be difficult to evaluate using traditional methods."

"We believe the ability to continuously collect multimodal data noninvasively will be a game-changer for oncology studies, in which the current standard for decades has been binary," commented W. Shawn Carbonell, MD, Ph.D, co-founder and CEO of OncoSynergy. "We are excited to work with Vium to develop this novel and more humane approach to studying animal models of orphan malignancies such as ovarian cancer and glioblastoma, as these diseases are our main focus."