On April 6, 2021 Ribon Therapeutics, a clinical stage oncology company developing therapeutics targeting stress support pathways, reported that it will present one oral and four poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2021 Virtual Annual Meeting (Week 1), taking place from April 10 to 15, 2021 (Press release, Ribon Therapeutics, APR 6, 2021, View Source [SID1234577618]). Abstracts are available at: www.aacr.org.

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"The breadth of new pre-clinical data that we are presenting this year at AACR (Free AACR Whitepaper) further validates our BEACON+ platform targeting novel cellular stress pathways," said Heike Keilhack, Ph.D., Senior Vice President of Biological Sciences, Ribon Therapeutics. "We are particularly encouraged by our research further elucidating the mechanism of action of our PARP7 inhibitor and lead asset, RBN-2397, and its potential for efficacy in numerous types of cancer."

Ribon Therapeutics will present the following from its development program and platform:

Abstract Title: RBN-2397: A potent and selective small molecule inhibitor of PARP7 that induces tumor-derived antitumor immunity dependent on CD8 T cells
Presenter: Joseph M. Gozgit, Ph.D., Director, Biological Sciences, Ribon Therapeutics
Date & Time: Sunday, April 11, 2021 at 2:00 PM ET
Session Type: Minisymposium
Session Title: New Therapeutics Targeting Molecular Drivers in Cancer
Abstract ID: 48
Summary:

RBN-2397 restores Type I interferon (IFN) signaling in cancer cells and researchers demonstrate that this is an on-target effect of inhibiting the catalytic activity of PARP7 and not PARP1. Researchers further show that the adaptive immune response was required for the antitumor effects of RBN-2397.
Abstract Title: Elevated PARP7 expression in select cancers identifies a target population for RBN-2397 therapy
Presenter: Jodie Wong, Research Associate, Ribon Therapeutics
Date & Time: Available for online viewing starting at 8:30 AM on Saturday, April 10
Session Type: E-Poster Session
Session Title: Biomarkers Predictive of Therapeutic Benefit
Abstract ID: 381
Summary:

RBN-2397 is a PARP7 inhibitor that induces cancer cell autonomous and immune stimulatory effects in preclinical models through enhanced Type I IFN signaling in cancer cells. Elevated PARP7 expression or amplification may identify cancer patients who could derive benefit from treatment with RBN-2397. Researchers showed the presence of PARP7 amplifications as well as high expression levels in several tumor types including non-small cell lung carcinoma, breast, and pancreatic ductal adenocarcinoma, providing evidence for the therapeutic relevance of PARP7 inhibition and highlighting potential patient selection strategies to identify those patients more likely to benefit from RBN-2397 treatment.
Abstract Title: Investigating the mechanism of PARP7 inhibition in Type I interferon signaling by arrayed CRISPR screening
Presenter: Bin Gui, Ph.D., Senior Scientist, Ribon Therapeutics
Date & Time: Available for online viewing starting at 8:30 AM on Saturday, April 10
Session Type: E-Poster Session
Session Title: Cellular Responses to Anticancer Drugs
Abstract ID: 1021
Summary:

To investigate the underlying mechanism of PARP7 inhibition and to determine the drivers of the differential sensitivity across cell lines, researchers performed arrayed CRISPR knockout screens, targeting approximately 240 genes in the nucleic acid sensing and IFN signaling pathways, in the presence and absence of PARP7 inhibition. The arrayed screens confirmed multiple hits from a previous genome-wide pooled synthetic/lethal CRISPR dropout screen, shedding light on the mechanism by which PARP7 acts as a critical suppressor of the innate immune response in tumor cells and demonstrating both redundancy and crosstalk between different nucleic acid-sensing pathways.
Abstract Title: Targeted Degradation of PARP14 Using a Heterobifunctional Small Molecule
Presenter: Tim J. Wigle, Ph.D., Senior Director, Biochemical & Cellular Pharmacology, Ribon Therapeutics
Date & Time: Available for online viewing starting at 8:30 AM on Saturday, April 10
Session Type: E-Poster Session
Session Title: Novel Targets and Pathways
Abstract ID: 1348
Summary:

RBN012811 is a heterobifunctional small molecule based on a catalytic inhibitor of PARP14 that binds in the enzyme’s NAD+-binding site and recruits the E3 ligase cereblon to ubiquitinate PARP14 and selectively target it for degradation. Researchers found that in PARP14 expressing cells, RBN012811 has a half-maximal degradation concentration (DC50) of 0.005 μM and it does not cause degradation of other PARP enzymes. In human primary macrophages, PARP14 degradation by RBN012811 led to a dose-dependent decrease of IL-10 release induced by IL-4 stimulation.
Abstract Title: Small molecule inhibitor of CD38 modulates its intra- and extracellular functions leading to antitumor activity
Presenter: Prashant B. Shambharkar, Ph.D., Senior Scientist, Ribon Therapeutics
Date & Time: Available for online viewing starting at 8:30 AM on Saturday, April 10
Session Type: E-Poster Session
Session Title: Novel Targets and Pathways
Abstract ID: 1344
Summary:

Inhibition of CD38 with a small molecule affects both intra- and extra-cellular CD38 activity and modulates key metabolites playing an important role in immunomodulation. Further, data indicate that CD38 is expressed at baseline in cancer and further increased by immune checkpoint inhibitor treatment. Finally, catalytic inhibition of CD38 can lead to antitumor activity in mouse cancer models.
Following its AACR (Free AACR Whitepaper) presentations, Ribon Therapeutics expects to make the poster presentations available on its corporate website via the following link: View Source

About RBN-2397

RBN-2397, is an orally available small molecule inhibitor of PARP7 that we are developing for the treatment of solid tumors. PARP7 is upregulated in response to cellular stress, including genomic instability in cancers, and acts as a brake on the cellular stress response by negatively regulating the Type I interferon response. By inhibiting PARP7 in tumor cells, RBN-2397 has been shown to directly inhibit cellular proliferation and restore interferon signaling to stimulate an innate and adaptive antitumor immune response. RBN-2397 is currently in a Phase 1 clinical trial as a monotherapy in patients with advanced solid tumors. PARP7 is overexpressed in a number of tumors, including squamous cell carcinoma of the lung, or SCCL, which represents approximately 30% of all non-small cell lung cancers.

On April 6, 2021 Volastra Therapeutics reported it will collaborate with Microsoft to develop tools that help detect drivers of cancer metastasis (Press release, Volastra Therapeutics, APR 6, 2021, View Source [SID1234577634]). These unique digital pathology tools will accelerate the development of promising new therapies for cancer and help identify the patients most likely to benefit.

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"Metastasis is one of the biggest unsolved challenges in cancer treatment, but new insights into tumor biology from our founders have laid the groundwork for major advancements in this field," said Charles Hugh-Jones, M.D., Chief Executive Officer at Volastra. "We are excited to work with Microsoft to apply data science to the challenges of cancer research. The digital tools we develop will fuel our drug discovery efforts and bring new hope to people living with cancer."

"We have seen the tremendous promise of advanced computation in the fields of medical diagnostics and drug discovery," said Desney Tan, Ph.D., Managing Director, Microsoft Health Futures. "The collaboration with Volastra will lead to potential advances in the development of therapies to prevent and treat cancer metastasis. We look forward to continued collaboration with Volastra to deliver solutions for the computational life sciences community."

The teams will work together on algorithms to identify markers that correlate with tumor metastatic behaviors. The collaboration will develop automated machine learning tools capable of rapidly and accurately integrating insights across multiple datasets, including pathology slides and three-dimensional tumor-derived organoids.

On April 6, 2021 MyBiotics Pharma Ltd., a microbiome therapeutics company, and Hadasit Medical Research Services and Development Ltd., the technology transfer office of Hadassah Medical Center, reported that they have entered into a research collaboration and licensing agreement for the identification of microbiome-based therapeutics that will enhance the response to and reduce adverse effects of anti-PD-1 and anti-PD-L1 in melanoma patients (Press release, MyBiotics, APR 6, 2021, View Source [SID1234577652]).

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The collaboration will combine Hadassah’s knowhow and expertise in immunotherapy treatment of melanoma patients with MyBiotics’ proprietary microbiome technologies enabling development of novel therapeutics. The two-year joint research project will be conducted by researchers from MyBiotics together with a team of researchers at Hadassah Cancer Research Institute headed by Prof. Michal Lotem, MD., Head of the Center for Melanoma and Cancer Immunotherapy, Department of Oncology at Hadassah Medical Center. It will be funded by MyBiotics, which has an exclusive license for all data and inventions stemming from the collaboration.

The research is aimed at assessing the composition of the gut microbiome and secondary metabolites (organic compounds produced by the gut bacteria) in up to 100 melanoma patients treated with PD-1/PD-L1 checkpoint inhibitors, some of which already exhibited long-term response to the treatment. Stool and blood samples will be collected at various time points along the treatment, for the purpose of identifying microbiome components that promote treatment success.

"Recent research supports the important role played by the microbiome in promoting the success of cancer immunotherapies, and points to the possibility of influencing the composition of the microbiome as an adjunct treatment," stated David Daboush, CEO of MyBiotics Pharma. "In this collaboration with Hadassah we will leverage Mybiotics’ SuperDonor whole microbiome recovery technology in combination with the MyLiveIn computational and predictive tools to advance research in order to unravel new layers of understanding and findings that will enable the design of effective microbiome-based therapeutics."

"We look forward to the joint research with the team at Hadassah Cancer Research Institute, who bring excellent clinical capabilities combined with innovative thinking and expect this will be a significant partnership that will lead to better treatment options for oncology patients within the next few years," Daboush added.

"For years I have strived to study what was driving long-term survival of melanoma patients who did well beyond expectations," stated Prof Michal Lotem, and added, "This collaboration gives us advanced molecular and genomic tools to analyze treatment success. After years of studying how cancer deceives us, I cannot wait to translate lessons of the past to therapies of the future."

"This collaboration is an excellent example of the kind of partnerships we strive to create between medical companies and hospital-based research centers. I trust that the unique combination of scientific excellence and clinical expertise will work for the benefit of patients in Israel and worldwide", stated Dr. Tamar Raz, the CEO of Hadasit.

MyBiotics has developed breakthrough and robust culturing, fermentation and delivery technologies for generating a highly stable and diverse bacterial community that can be efficiently delivered to the gut and can reliably restore microbiome equilibrium. These technologies are effective for single microbes, complex microbial consortia and whole microbiome products, and are integrated with a computational AI platform which enables the design of unique microbial consortia and whole microbiome profiles. The technologies are highly potent and suitable for patients with microbiome-related medical conditions or for those who use antibiotics. Preclinical studies have shown that MyBiotics’ products deliver enhanced durability in various gastrointestinal and manufacturing conditions, enable targeted release in different gastrointestinal locations and exhibit robust colonization in the gut.

About the Microbiome and Cancer Immunotherapy

In the last 5 years, studies elucidating the possible contribution of the microbiome to cancer development and response to treatments has been at the forefront of scientific research. Findings have shown, for example, that broad-spectrum antibiotics can reduce the efficiency of checkpoint inhibitors. In addition, studies have pointed to differences between microbiomes of patients responding to immunotherapy and those who do not respond to treatment. In animal models, tweaking the microbiome or adding secondary metabolites thereof, influenced the efficiency of cancer immunotherapy. These findings and others support the potential of changing the microbiome composition as a tool for improving the efficacy and reducing the toxicity of checkpoint inhibitor immunotherapy in cancer patients.

On April 6, 2021 PCI Biotech (OSE: PCIB), a clinical-stage biopharma company developing innovative therapeutics that address significant unmet medical needs in cancer reported that it will present at the European Biotech Investor Days 2021, a US based online event taking place April 7-8, 2021 and hosted by Goodwin, Solebury Trout, Deutsche Bank and Nasdaq (Press release, PCI Biotech, APR 6, 2021, View Source [SID1234585154]).

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On Thursday, April 8, 2021 at 15:00 (CEST), Dr. Per Walday, CEO, will present an overview of PCI Biotech’s proprietary platform technology via a general company presentation.

The presentation can be accessed live through this link View Source

The presentation slides will be made available on PCI Biotech’s website (www.pcibiotech.com) under "Other presentations" after the event.

On April 6, 2021 ORPHELIA Pharma and Biocodex reported the execution of an exclusive distribution and marketing agreement for Kigabeq (vigabatrin) for most of the European Union territory, including France (Press release, ORPHELIA Pharma, APR 6, 2021, View Source [SID1234577603]).

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Kigabeq, the first pediatric form of vigabatrin developed by ORPHELIA Pharma, is notably indicated in the treatment of infantile spasms (West syndrome). Kigabeq is approved in Europe, where this medicine, intended exclusively for children, benefits from a centralized marketing authorization (Pediatric Use Marketing authorization, PUMA).

"We are very pleased to release this collaboration agreement with Biocodex", says Hugues BIENAYME, Founder and CEO of ORPHELIA Pharma, "With their long-standing experience in the field of pediatric epilepsy, Biocodex is certainly the best partner for Kigabeq distribution in Europe".

"With Kigabeq, the only pediatric presentation of vigabatrin, Biocodex is expanding its portfolio of drugs intended for rare and serious pathologies in children" adds Nicolas Coudurier, CEO of Biocodex, " Kigabeq and Diacomit (stiripentol, developed and marketed by Biocodex) are two drugs which address unmet medical needs for young patients affected by severe and resistant to treatment epilepsies".

"Our objective is to make Kigabeq available to all European clinicians, so that children affected by West syndrome can benefit." concludes Gilles ALBERICI, President of ORPHELIA Pharma, "Thanks to this agreement with Biocodex, Kigabeq will soon be prescribed in most of the European territory."

About Kigabeq

Kigabeq is the first pediatric formulation of vigabatrin, an essential anti-epileptic drug. Presented as soluble and scored tablets of 100 mg and 500 mg for oral or nasogastric administration, Kigabeq is notably indicated for the treatment of infantile spasms (West syndrome), an extremely serious epileptic encephalopathy of the infant. Kigabeq was developed exclusively for children and has been granted European Pediatric Marketing Authorization (PUMA).

About infantile spasms

West syndrome, or infantile spasms, is an extremely serious epileptic encephalopathy in infants that combines epileptic spasms, psychomotor deterioration and a hypsarrhythmic type electroencephalogram. It is a rare disease, with an estimated incidence of 5 per 10,000 births. It can occur in an infant with previously normal development or with a pre-existing delay; in all cases, infantile spasms severely hamper the psychomotor development. Pharmacological treatment should be started quickly to allow the spasms to stop and to improve the prognosis.