On September 30, 2021 Immix Biopharma, Inc. ("ImmixBio"), a biotechnology company pioneering Tissue Specific Therapeutics (TSTx)TM for oncology and inflammation, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to IMX-110 for the treatment of soft tissue sarcoma (Press release, Immix Biopharma, SEP 30, 2021, View Source [SID1234590560]).

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The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including financial incentives, to support clinical development and the potential for up to 7 years of market exclusivity in the U.S. upon regulatory approval.

"We are pleased to receive FDA’s orphan drug designation in soft tissue sarcoma for IMX-110, the first clinical-stage product of our SMARxT Tissue-Specific Platform," said Ilya Rachman MD PhD, ImmixBio Founder & CEO. "We are thrilled to potentially contribute to expanding therapeutic options for oncology patients, beginning with soft tissue sarcoma."

Gabriel Morris, ImmixBio Chief Financial Officer, added: "Orphan drug designation for IMX-110 represents a substantial value creating step along our path of building a strong, independent biopharmaceutical organization."

About IMX-110

IMX-110 is a Tissue-Specific TherapeuticTM built on ImmixBio’s TME NormalizationTM Technology encapsulating a poly-kinase inhibitor and apoptosis inducer delivered deep into the tumor micro-environment, or TME. ImmixBio’s TME Normalization Technology enables IMX-110 to circulate in the bloodstream, then exit through porous tumor blood vessels, and accumulate in the TME. IMX-110 then simultaneously attacks all 3 components of the TME (cancer associated fibroblasts, or CAFs; tumor-associated macrophages/immune cells, or TAMs, and cancer itself), severing the critical lifelines between the tumor and its metabolic and structural support. IMX-110’s TME Normalization Technology causes tumor apoptosis, a non-inflammatory tumor-cell death (vs. necroptosis, which results in repeat reignition of the inflammatory cascade leading to tumor progression).

IMX-110 is currently being evaluated in a phase 1b/2a open-label, dose-escalation/dose-expansion safety, tolerability and pharmacokinetic study in patients with advanced solid tumors in the United States and Australia.

On September 30, 2021 Amplia Therapeutics Limited (ASX: ATX), ("Amplia" or the "Company"), a company developing new drugs for the treatment of cancer and fibrosis, reported that publication of a key paper from the Garvan Institute of Medical Research ("Garvan") (Press release, Amplia Therapeutics, SEP 30, 2021, View Source;[email protected] [SID1234590579]). The paper, which describes the fundamental biology underpinning Amplia’s planned Phase 2 clinical trial in pancreatic cancer patients, further highlightsthe potential benefits of using a focal adhesion kinase (FAK) inhibitor prior to administration of standard chemotherapy.

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Entitled "Intravital imaging technology guides FAK-mediated priming in pancreatic cancer precision medicine according to Merlin status" the paper has been published in the high impact peer-reviewed journal Science Advances. 1 Professor Paul Timpson, a leading researcher in FAK biology at Garvan and a member of Amplia’s Scientific Advisory Board, led the research program which has shown that in mice that have been implanted with human pancreatic cancer tissue, pre-treatment with a FAK inhibitor (‘priming’) increased the responsiveness of the cancer to subsequently administered gemcitabine/Abraxane chemotherapy. Furthermore, FAK-priming reduced the metastatic spread of tumour cells to secondary sites such as the liver.

"There have been several publications over the last two years that have highlighted the potential of FAK inhibitors in pancreatic cancer, including their ability to work synergistically with chemotherapy agents" said John Lambert, CEO of Amplia: "This latest study from our collaborators at the Garvan Institute is particularly exciting as its replicates the approach that we are taking to treat first line pancreatic cancer patients in our recently announced Phase 2 clinical trial. We believe that making an established standard of care, namely chemotherapy with gemcitabine/Abraxane, more effective offers a very promising approach for improving the outcomes for these patients".

On September 30, 2021 IMV Inc. (NASDAQ: IMV; TSX: IMV), a clinical-stage biopharmaceutical company pioneering a novel class of immunotherapies against difficult-to-treat cancers, reported that two abstracts featuring two DPX-based immunotherapies have been accepted for virtual poster presentation at the upcoming AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) on October 7-10, 2021 (Press release, IMV, SEP 30, 2021, View Source [SID1234590597]).

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Poster #1:

Survivin peptides formulated in the DPX delivery platform rather than standard emulsions, elicit a robust, sustained T cell response to survivin in advanced and recurrent ovarian cancer patients.

Presenter:
Yogesh Bramhecha, Ph.D.,
Director of Translational Research, IMV Inc.

Poster Number:
LBA026

Poster #2:

DPX-SurMAGE, a novel dual-targeted immunotherapy for bladder cancer, induces target-specific T cells with a favorable safety profile in preclinical model.

Presenter:
Yves Fradet, M.D.

Professor, Department of Surgery

Faculty of Medicine, Université Laval, Quebec City

Poster Number:
LBA030

Full abstracts and e-posters will be available on demand on the conference platform on October 7, 2021 at 9am ET. Both e-posters will be available under the Scientific Publications & Posters section on IMV’s website.

On September 30, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported that David Baltimore, Ph.D., a director of Regulus, is the recipient of the 2021 Lasker-Koshland Special Achievement Award in the category of Medical Science (Press release, Regulus, SEP 30, 2021, View Source [SID1234590618]).

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Dr. Baltimore’s recognition is based on the breadth and substance of his discoveries in virology, immunology, and cancer as well as his leadership within the academic and scientific communities and his extensive work as a public advocate for science.

The Lasker Awards program honors discoveries and clinical advances that improve human health and highlights the importance of public support for science. The awards program is widely considered one of the most prestigious awards programs in medical science and numerous recipients, including Dr. Baltimore, also hold Nobel Prizes.

"On behalf of the Regulus team, we congratulate David for this recognition of his extensive and impressive body of work," said Jay Hagan, President and Chief Executive Officer of Regulus. "As a 1975 Nobel Prize winner in Physiology or Medicine, David has made multiple foundational scientific discoveries over the course of his career, including the discovery of reverse transcriptase, as well as his additional research, which has contributed broadly to our understanding of cancer, AIDS, and the underlying biology of the immune response. He has been a powerful advocate for science over the years, advancing work that helped America respond to the AIDS crisis, and has played a significant role in the development of the biotechnology industry in America. We are proud that he is a part of Regulus and continually benefit from his guidance as we advance our pipeline of microRNA therapeutics."

More information about the foundation and the Special Achievement Award can be found here.

About David Baltimore, Ph.D.

Dr. David Baltimore is a Director of Regulus and President Emeritus and Distinguished Professor of Biology at Caltech. Awarded the Nobel Prize in 1975, Dr. Baltimore has profoundly influenced national science policy on such issues as recombinant DNA research and the AIDS epidemic. Dr. Baltimore graduated from Swarthmore College with a degree in chemistry. He took graduate courses at Massachusetts Institute of Technology and received his Ph.D. from Rockefeller University. He was a postdoctoral fellow at MIT and Albert Einstein College of Medicine. He was a Research Associate at The Salk Institute in 1965 and joined the faculty at MIT in 1968. He has served as Director of the Whitehead Institute for Biomedical Research, President of Rockefeller University, and President of Caltech. He was awarded the 1999 National Medal of Science and 2000 Warren Alpert Foundation Prize. He is a member of the National Academy of Sciences, fellow of the American Academy of Arts and Sciences, and a foreign member of the Royal Society of London and the French Academy of Sciences. He has also been President and Chair of the American Association of the Advancement of Science.

On September 30, 2021 Nimbus Therapeutics, a biotechnology company designing breakthrough medicines through structure-based drug discovery and development, reported that research in the Proceedings of the National Academy of Sciences describing the structural basis for isoform-specific inhibition of human CTPS1 (CTP synthase 1) (Press release, Nimbus Therapeutics, SEP 30, 2021, View Source [SID1234590561]). Selective inhibition of CTPS1 is a promising approach for the treatment of autoimmune and other T cell-driven diseases, but little is known about the mechanisms underlying selective versus non-selective inhibition.

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Nimbus scientists, in partnership with researchers from the University of Washington and Schrödinger, used cryo-electron microscopy (cryo-EM) to characterize the activity of CTPS inhibitors in binding to, inhibiting and distinguishing between CTPS1 and its isoform CTPS2. The cryo-EM research was funded by Nimbus and led by professor Justin Kollman at the University of Washington.

"We’re proud to have conducted this impactful research in partnership with our valued colleagues at University of Washington and Schrödinger, further building on Nimbus’ long history of fruitful academic collaborations," said Peter Tummino, Ph.D., Chief Scientific Officer at Nimbus. "The insights we have published will be instrumental in our efforts to develop selective inhibitors of CTPS1 that can potentially offer a powerful new means of treating autoimmune diseases and T cell-driven cancers."

"A defining feature of Nimbus’ structure-based drug discovery approach is our use of leading-edge computational technology, including cryo-EM, to characterize drug targets in unprecedented detail," said Scott Edmondson, Ph.D., Senior Vice President and Head of Chemistry at Nimbus. "The discoveries made in this research, together with Nimbus’ expertise in computational chemistry, molecular sciences and disease biology, will inform our ongoing development of highly selective small-molecule CTPS1 inhibitors."

The paper, entitled "Structural basis for isoform-specific inhibition of human CTPS1," published online in the Proceedings of the National Academy of Sciences this week: View Source