On November 29, 2017 Vedanta Biosciences, an affiliate of PureTech Health (LSE: PRTC) developing a new category of therapies for immune-mediated and infectious diseases based on rationally designed consortia of human microbiome-derived bacteria, reported new translational medicine collaborations in cancer immunotherapy with Leiden University Medical Center and the University of South Alabama (USA) Mitchell Cancer Institute (Press release, Vedanta Biosciences, NOV 29, 2017, View Source [SID1234522340]). The Company also reported the expansion of its translational medicine collaborate on in cancer immunotherapy with NYU Langone Health and its Perlmutter Cancer Center. Researchers at these institutions have been collaborating with Vedanta Biosciences to analyze microbiome clinical data from interventional checkpoint inhibitor studies to identify microbiome signatures associated with response to immunotherapy and key mechanisms through which the gut microbiota modulate immunotherapeutic responses.

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"Data from our ongoing clinical collaborations in melanoma show that gut bacteria signatures could help determine if a cancer immunotherapy will work," said Bruce Roberts, Ph.D., Chief Scientific Officer of Vedanta Biosciences. "We’re pleased to expand our research collaborations into others forms of cancer, with the ultimate goal of identifying ways to change the microbiome to increase the proportion of patients and types of cancer patients who respond to immunotherapies."

Under the agreement with Leiden University Medical Center, Vedanta Biosciences will work in collaboration with Ellen Kapiteijn, M.D., Ph.D., and Ed Kuijper, M.D., Ph.D., to understand the role of the microbiome in immunotherapeutic responses against a variety of cancers, including melanoma, head and neck, and bladder. The new collaboration with the USA Mitchell Cancer Center, led by Art Frankel, M.D., will analyze associations between the gut microbiome and responses to checkpoint inhibitor treatment in melanoma and cancers of the bladder and kidneys. Building on the existing translational work with NYU Langone in melanoma led by Jeffrey S. Weber, M.D., Ph.D., and Melissa Wilson, M.D., Ph.D., the expanded agreement adds collaborations in bladder cancer and lung cancer, led, respectively, by Arjun V. Balar, M.D., and Leena Gandhi, M.D., Ph.D.

Vedanta Biosciences’ immuno-oncology programs include lead product candidate, VE800, which has been shown in preclinical models to activate CD8+ T cells, a type of white blood cell that is the predominant effector in cancer immunotherapy, improve CD8+ T cell tumor infiltration, and improve survival in several cancer models in combination with checkpoint inhibitors. Vedanta anticipates filing an investigational new drug application (IND) for this candidate in 2018.

On November 29, 2017 Kitov Pharmaceuticals presented Company Presentation (Presentation, Kitov Pharmaceuticals , NOV 29, 2017, View Source [SID1234522305]).

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On November 29, 2017 Amphivena Therapeutics Inc., a privately held biotechnology company developing a novel CD33/CD3 T cell engager for the treatment of Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS), reported that it has received Orphan Drug Designation from the U.S. Food and Drug Administration for its lead compound AMV564 for the treatment of AML (Press release, Amphivena Therapeutics, NOV 29, 2017, View Source [SID1234522341]).

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"The FDA’s designation of AMV564 as an orphan drug is an important milestone for us that will provide marketing protections and economic benefits at drug approval. Given the unique safety and efficacy profile that is emerging in the clinic, we believe our CD33-targeted T cell engager will be an important drug in the armamentarium for leukemia patients who have limited treatment options today," said Eric J. Feldman, M.D., Amphivena’s Senior Vice President of Clinical Development.

Amphivena is conducting a Phase 1 clinical study of AMV564 in relapsed or refractory AML. Amphivena plans to launch a Phase 1 clinical study in patients with MDS in early 2018. The company is also exploring the utility of AMV564 in solid tumors. In preclinical studies, this novel CD33/CD3 bispecific antibody demonstrated potent activity against AML patient samples that was independent of CD33 expression level, disease stage and cytogenetic risk. The antibody eliminated nearly all blasts from bone marrow and spleen in a stringent AML patient-derived xenograft murine model. In addition, Amphivena established a therapeutic window for AMV564 in cynomolgus monkeys, with rapid and sustained elimination of CD33-expressing cells during AMV564 dosing and rapid hematopoietic recovery following dosing.

Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to products that treat rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides the sponsor certain benefits and incentives, including a period of marketing exclusivity for the first marketing application, if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.

On November 29, 2017 Mavupharma (Mavu) reported that it has secured $20 million in a Series A financing led by Frazier Healthcare Partners and joined by Alpine BioVentures. Mavu is developing orally bioavailable, non-nucleotide modulators of the STING (stimulator of interferon genes) pathway to treat cancer and infectious diseases, and the Series A funding will be used primarily to advance Mavu’s lead drug candidates into the clinic (Press release, Mavupharma, NOV 29, 2017, View Source [SID1234537642]).

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"Targeting the STING pathway holds great potential for harnessing the body’s immune system to fight cancer and viral diseases," said Michael Gallatin, Ph.D., president of Mavupharma. "Mavu’s novel approach uses non-nucleotide small molecules to indirectly and conditionally modulate the pathway, and we are leveraging this technology to develop orally bioavailable STING activators with first-in-class potential."

Mavupharma was started by a team of industry veterans including Dr. Gallatin, who co-founded Calistoga Pharmaceuticals and Stromedix and served as vice president and scientific director at ICOS, and Greg Dietsch, Ph.D., who was vice president of research at VentiRx and vice president of preclinical research at ICOS. The company has assembled an experienced scientific, drug development and management team and recently hired Clayton Knox, M.D., as chief operating officer. Dr. Knox was formerly senior vice president for corporate development and strategy at Acerta Pharma, prior to which he held clinical research and business development positions at Merck & Co.

As part of the financing, Mavu announced members of its board of directors. Bob Baltera, chief executive officer of Cirius Therapeutics will serve as executive chairman of the board. Other members of the board are Jamie Topper, M.D., Ph.D., of Frazier and Rich Heyman Ph.D., formerly chief executive officer of Seragon Pharmaceuticals and Aragon Pharmaceuticals.

"Frazier has had great success by assembling experienced teams to build value around innovative assets," stated Dr. Topper. "Mavu, with its differentiated approach to an exciting pathway, is a natural investment for us."

ABX196, a first-in-class iNKT agonist boosting the immune response in cancer

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ABIVAX is currently developing ABX196, a state of the art immune enhancer candidate based on iNKT activation. This product is largely derived from the technology and exclusive patent rights transferred to ABIVAX by the Scripps Research Institute (La Jolla, CA), the University of Chicago (Chicago, IL) and the Brigham Young University (Salt Lake City, UT)

A phase I clinical trial in healthy volunteers has been completed and showed activation of iNKT cells. Based on these data, new immuno-oncology pre-clinical studies were conducted that demonstrated the potential of the product in oncology, in particular in turning tumors not responsive to anti-PD-1 (cold) to tumors responsive to anti-PD-1 (hot). As ABIVAX is focusing on the antiviral and anti-inflammatory therapeutic spaces and does not intend to play a role in the immune-oncology field, ABIVAX is seeking to out-license this interesting product candidate.

The characteristics of ABX196 are as follows:

ABX196 has been developed from a platform technology proprietary to ABIVAX that identifies "iNKT Agonists" that demonstrate immune enhancing effects in cancer models
ABX196 is a synthetic agonist (glycolipid) of iNKT (invariant Natural Killer T) cells, in a liposomal formulation
ABX196 is well characterized, with stability studies and full toxicity package (including non-human primate studies) conducted prior to the phase I clinical trial in healthy volunteers
Phase I showed ABX196 is well tolerated and triggered both humoral as well as iNKT immune responses in human volunteers
Pre-clinical data show that ABX196 enhances anti-tumoral activity when used alone and in combination with anti-PD-1 antibody, doxorubicin or sorafenib
ABX196 turns a cold (i.e. non-responding to anti-PD-1 antibody) tumor into a hot, responsive tumor in the mouse melanoma model, corresponding to an increase in survival time
ABX196 potentiates the anti-tumoral response to chemotherapy (doxorubicin) in the mouse melanoma model, corresponding to an increase in survival time
ABX196 controls tumor progression in an orthotopic hepatocellular cancer model in mice, again showing a prolongation of survival
In total, POC of anti-tumoral activities has been established in four preclinical cancer models
Easy-to-use liposome formulation, with scaled-up and controlled manufacturing
Strong IP protection and FTO: 5 patent families