Can-Fite To Harness Artificial Intelligence To Develop Novel Anti-Cancer Drugs

Can-Fite signed an agreement with Collaborations Pharmaceuticals, a leading expert in Artificial Intelligence and Machine Learning

PETACH TIKVA, Israel--(BUSINESS WIRE)-- Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncology and inflammatory diseases, today announced that it entered into an agreement with Collaborations Pharmaceuticals, Inc. (CPI) to develop anti-cancer drugs utilizing artificial intelligence (AI) and machine learning (ML) techniques. This project will aim to develop a next-generation A3 adenosine receptor drug agonists that significantly reduce the development time and cost of bringing such drugs to market.

CPI will utilize, apply and use AI and ML tools, including their MegaSyn generative AI method, to design new molecules with high affinity and selectivity to the A3AR Can-Fite target. CPI will also perform the chemical synthesis of the newly designed molecules with the ultimate goal of developing novel and robust anti-cancer drug candidates. Can-Fite will perform the testing of the biological anti-cancer effects and validate the molecular mechanism of the novel, chemically synthesized drug candidates.

“Our vision is to deliver in silico small molecule drug candidates in a better and faster way to patients via a collaboration with Collaborations Pharmaceuticals. Our accumulated experience of bringing anti-cancer drugs which target the A3AR from lab to patients will be implemented into this AI drug development project,” stated Prof. Pnina Fishman, Executive Chairman and CSO at Can-Fite.

“We are delighted that Can-Fite chose our team of experts for this AI-led drug discovery collaboration and look forward to demonstrating what our technology can do,” said Sean Ekins, PhD, DSc., CEO and Founder of Collaborations Pharmaceuticals, Inc. We also look forward to complementing their outstanding scientific approach with our integrated technology platform and ability to generate novel and selective molecules."

About Can-Fite BioPharma Ltd.
Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF) is an advanced clinical stage drug development Company with a platform technology that is designed to address multi-billion dollar markets in the treatment of cancer, liver, and inflammatory disease. The Company’s lead drug candidate, Piclidenoson recently reported topline results in a Phase III trial for psoriasis and is expected to commence a pivotal Phase III. Can-Fite’s cancer and liver drug, Namodenoson, is being evaluated in a Phase IIb trial for the treatment of steatotic liver disease (SLD), a Phase III pivotal trial for hepatocellular carcinoma (HCC), and the Company is planning a Phase IIa study in pancreatic cancer. Namodenoson has been granted Orphan Drug Designation in the U.S. and Europe and Fast Track Designation as a second line treatment for HCC by the U.S. Food and Drug Administration. Namodenoson has also shown proof of concept to potentially treat other cancers including colon, prostate, and melanoma. CF602, the Company’s third drug candidate, has shown efficacy in the treatment of erectile dysfunction. These drugs have an excellent safety profile with experience in over 1,600 patients in clinical studies to date. For more information please visit: www.can-fite.com.

About Collaborations Pharmaceuticals:
Collaborations Pharmaceuticals, Inc. developed MegaSyn for generative drug design. In addition they have developed Assay Central® software for data curation and machine learning as well as curated model collections such as MegaTox®, MegaTrans® and MegaPredict®. Collaborations Pharmaceuticals, Inc. performs research and development on innovative therapeutics for multiple rare and neglected diseases and consults for pharmaceutical and consumer product companies. For more information, http://www.collaborationspharma.com/

Forward-Looking Statements
This press release may contain forward-looking statements, about Can-Fite’s expectations, beliefs or intentions regarding, among other things, its product development efforts, business, financial condition, results of operations, strategies or prospects. All statements in this communication, other than those relating to historical facts, are “forward looking statements”. Forward-looking statements can be identified by the use of forward-looking words such as “believe,” “expect,” “intend,” “plan,” “may,” “should” or “anticipate” or their negatives or other variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. Forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to known and unknown risks, uncertainties and other factors that may cause Can-Fite’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Important factors that could cause actual results, performance or achievements to differ materially from those anticipated in these forward-looking statements include, among other things, our history of losses and needs for additional capital to fund our operations and our inability to obtain additional capital on acceptable terms, or at all; uncertainties of cash flows and inability to meet working capital needs; the initiation, timing, progress and results of our preclinical studies, clinical trials and other product candidate development efforts; our ability to advance our product candidates into clinical trials or to successfully complete our preclinical studies or clinical trials; our receipt of regulatory approvals for our product candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of our product candidates; our ability to establish and maintain strategic partnerships and other corporate collaborations; the implementation of our business model and strategic plans for our business and product candidates; the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and our ability to operate our business without infringing the intellectual property rights of others; competitive companies, technologies and our industry; risks related to the COVID-19 pandemic and the Russian invasion of Ukraine; risks related to not satisfying the continued listing requirements of NYSE American; and statements as to the impact of the political and security situation in Israel on our business. More information on these risks, uncertainties and other factors is included from time to time in the “Risk Factors” section of Can-Fite’s Annual Report on Form 20-F filed with the SEC on March 30, 2023 and other public reports filed with the SEC and in its periodic filings with the TASE. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Can-Fite undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws.

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Collaborations Pharmaceuticals and UTMB awarded a $295,000 grant for Preclinical Development of a Nipah Virus Treatment

RALEIGH, NORTH CAROLINA, USA, September 20, 2023/EINPresswire.com/ -- Collaborations Pharmaceuticals, Inc. (CPI) and the Texas Medical Branch (UTMB) are pleased to announce that they were awarded a $295,000 Phase I Small Business Innovation Research (SBIR) grant from the National Institute of Allergy and Infectious Diseases (NIAID), to perform preclinical in vivo studies on a recently identified Nipah virus (NiV) inhibitor.

NiV is a bat-borne pathogen that results in acute and often fatal (recent outbreaks in India have demonstrated case fatality ~70%) respiratory and neurological disease for which there is currently no FDA approved treatment. There have been very few small molecule antivirals that have demonstrated activity against NiV in vitro or in vivo. We have recently identified a potential antiviral against Nipah and now propose to perform preclinical studies at UTMB’s Robert E. Shope and the Galveston National Lab (GNL) BSL-4 laboratories. Our ultimate aim is to bring a treatment to market for NiV which can be stockpiled by the USA and other countries in preparation for future outbreaks.

Sean Ekins, CEO of CPI, added, "We are excited to continue our collaboration with Dr. Alexander Freiberg and colleagues at GNL at UTMB with whom we have previously published widely on developing antiviral small molecules with activity against other BSL-4 pathogens including Ebola and Marburg. We are looking forward to generating the preclinical data needed to translate this molecule and move it closer to the clinic."

About Collaborations Pharmaceuticals:
Collaborations Pharmaceuticals, Inc. has an active pipeline of molecules for infectious and rare diseases with considerable expertise in antiviral drug discovery. This work is aided by our inhouse software including Assay Central® software for data curation and machine learning as well as curated model collections such as MegaTox®, MegaTrans® and MegaPredict®. Most recently we have developed MegaSyn for generative drug design. For more information, http://www.collaborationspharma.com/

About UTMB
UTMB is an internationally recognized academic institution with strong expertise and resources in the global fight against infectious diseases. The GNL is an anchor lab of the NIAID Biodefense Laboratory Network with containment capabilities for biodefense research. More than 120 UTMB faculty and principal investigators from different departments conduct research with their teams in the GNL.

https://www.einpresswire.com/article/656529579/collaborations-pharmaceuticals-and-utmb-awarded-a-295-000-grant-for-preclinical-development-of-a-nipah-virus-treatment

PsychoGenics and Collaborations Pharmaceuticals awarded a $1M grant to use AI platforms to design new drugs for mental health disorders

PsychoGenics Inc. (PsychoGenics) and Collaborations Pharmaceuticals, Inc. (CPI) are pleased to announce that they were awarded a $1million Phase I Small Business Innovation Research (SBIR) grant, to use their combined AI tools to design new drugs for mental health disorders. This grant will enable both companies to collaborate on a groundbreaking project that responds to the National Institute of Mental Health (NIMH) at the National Institutes of Health (NIH) strategic plan aimed at the "Development and validation of new methods and technologies to advance the treatment of mental health disorders". The companies will develop and apply machine learning (ML) tools to explore new areas of chemistry, with the ultimate aim of identifying treatments that target novel or multiple aspects of complex neuropsychiatric disorders.

Statistics reveal that approximately one in four individuals experiences a mental disorder at some point in their lifetime. These disorders encompass a wide spectrum, including major depressive disorder, bipolar disorder, schizophrenia, and substance use disorders, impacting people of all age groups. Alarmingly, suicide ranks as the second leading cause of death among 15 to 29-year-olds, with 10 to 20 million depressed individuals attempting suicide annually and approximately 1 million completing the act. Current treatment modalities inadequately address the intricate challenges posed by mental illness, contributing to nearly one-third of adult disability on a global scale. Hence, there exists an urgent and compelling need for enhanced treatments that unlock new avenues of pharmacological intervention.

Acknowledging the colossal burden of mental illness and the glaring gap in improved treatments, PsychoGenics and CPI will harness their AI-enabled phenotypic drug discovery approach along with their ML tools to explore uncharted areas of chemistry to discover new classes of drugs. PsychoGenics has been a trailblazer in the use of ML for drug discovery and the exploration of structure-activity relationships (SAR) through its innovative SmartCube® technology. SmartCube® employs ML classifiers trained on reference CNS drugs to predict a compound's therapeutic and side-effects profiles. On the other hand, CPI has developed a suite of ML software including Assay Central®, MegaTox®, MegaTrans®, MegaPredict®, and MegaSyn®, which generate new molecules exploring uncharted chemistry space while optimizing essential properties.

Dr. Daniela Brunner, Chief Innovation Officer at PsychoGenics, expressed her enthusiasm about this collaboration, stating, "We are honored to have been awarded this SBIR grant and delighted to collaborate with CPI on this groundbreaking project. By synergizing our strengths in behavioral pharmacology, drug discovery, and advanced ML techniques, we hold the potential to unlock new treatments, ultimately making a positive impact on the lives of patients."

Sean Ekins, CEO of CPI, added, "We eagerly anticipate this collaboration with Dani and her team at PsychoGenics to explore uncharted areas of chemistry. Our AI tools, combined with PsychoGenics' Cube platforms and collective experience, render the ambitious goal of discovering new classes of drugs feasible."

About PsychoGenics:

PsychoGenics is a leading biotechnology company dedicated to revolutionizing drug discovery through PsychoGenics Inc. and its discovery arm, PGI Drug Discovery LLC (collectively known as PsychoGenics), have pioneered the translation of rodent behavioral and physiological responses into robust, high-throughput and high-content phenotyping. PsychoGenics' drug discovery platforms SmartCube®, NeuroCube®, PhenoCube®, and eCube™, have been used in shared-risk partnerships with major pharmaceutical companies, including Sunovion, Roche and Karuna, resulting in the discovery of several novel compounds now in advanced preclinical development and clinical trials. PsychoGenics' capabilities also include standard behavioral testing, electrophysiology, translational EEG, molecular biology, microdialysis, and quantitative immunohistochemistry. In addition, the Company offers a variety of in-licensed transgenic mouse models to support research in areas such as Huntington's disease, autism spectrum disorders, psychosis/schizophrenia, depression, PTSD, Alzheimer's disease, Parkinson's disease, muscular dystrophy, ALS and seizure disorders.

About Collaborations Pharmaceuticals:

Collaborations Pharmaceuticals, Inc. has developed Assay Central® software for data curation and machine learning as well as curated model collections such as MegaTox®, MegaTrans® and MegaPredict®. Most recently they have developed MegaSyn for generative drug design. Collaborations Pharmaceuticals, Inc. performs research and development on innovative therapeutics for multiple rare and neglected diseases. For more information, http://www.collaborationspharma.com/

View source version on businesswire.com: https://www.businesswire.com/news/home/20230905116595/en/

Collaborations Pharmaceuticals Announces Publication of New Enterovirus-D68 and Coxsackie B5 inhibitor

A new paper published in Antiviral Research describes a new inhibitor of Enterovirus-D68 and Coxsackie B5.

RALEIGH, NC, USA, June 15, 2023/EINPresswire.com/ -- Dr. Thomas Lane and Dr. Sean Ekins from Collaborations Pharmaceuticals, Inc. (CPI) with Dr. Vadim Makarov and collaborators at the Research Center of Biotechnology RAS, Moscow; and collaborators at Purdue University, North Carolina State University, Utah State University, University of Colorado Anschutz Medical Campus and Saint Petersburg Pasteur Institute are pleased to announce their new publication in Antiviral Research "Efficacy of an isoxazole-3-carboxamide analog of pleconaril in mouse models of Enterovirus-D68 and Coxsackie B5".

Enteroviruses (EV) and coxsackieviruses (CVs) cause a wide range of acute and chronic diseases, such as aseptic meningitis, encephalitis, hand-foot-and-mouth disease, conjunctivitis, diarrhea, herpetic angina, acute and chronic myocarditis, etc. These viruses represent the main cause of death in low-income countries and the third largest cause of death worldwide. EV-D68 is known to cause respiratory illness in children that can lead to acute flaccid myelitis, a polio like illness, while Coxsackievirus B5 (CVB5) is commonly associated with hand-foot-mouth disease. There is no antiviral treatment available for either. The most promising results against EVs to date have been obtained with the capsid-binding inhibitors.

“We have previously developed several new pleconaril-based molecules which displayed potent in vitro activity against a panel of CVs and rhinoviruses. We have now discovered that one of these compounds, 3-(3-methyl-4-(3-(3-N,N-dimethylcarbamoyl-isoxazol-5-yl)propoxy)phenyl)-5-trifluoromethyl-1,2,4-oxadiazole (11526092) displays potent in vitro inhibition across various EVs. This molecule demonstrated promising antiviral activity against EV-D68 in a mouse respiratory model of EV-D68 infection. Cryo-electron microscopy (cryo-EM) was used to demonstrate that 11526092 binds to the EV-D68 VP1 from the MO strain. We have additionally determined that 11526092 inhibits CVB5 in vitro and in vivo, and therefore may represent a promising new lead compound” said CPI CEO Dr. Sean Ekins.

Dr. Thomas Lane, Associate Director at CPI remarked “The CDC has recognized the reemergence of EV-D68 infections causing hospitalization of children and adolescents in the US based on a report published in November 2022. As these only capture instances that required hospitalization, this likely represents a small fraction of the total cases of EV-D68 in the US, reemphasizing the need for antivirals for EV-D68 and related viruses following the 2014 EV-D68 outbreak. This new compound has improved properties over its analog pleconaril, where it shows higher activity against multiple viruses, including some pleconaril-resistant strains, as well as reduced CYP3A4 upregulation, both of which likely contributed to pleconaril’s clinical failure. Our compound shows significant in vivo efficacy in an EV-D68 respiratory model, including viral reduction and a favorable cytokine response. This compound also showed in vivo antiviral activity against CVB5, one of the most common causes of hand-foot-and-mouth disease worldwide, expanding its potential use as a broad spectrum therapeutic.”

“11526092 is a new antiviral compound for EVs which we have studied in detail. It binds to EV-D68 in a similar manner to pleconaril, and we have now described the unique mechanism of action for compounds binding to different strains of the EV-D68 virus. 11526092 is well-tolerated in mice and demonstrates promising antiviral efficacy in in vivo mouse models. Based on this study and in the absence of any suitable antiviral treatments for these viruses, further testing of 11526092 may be warranted against these and other related EV’s We look forward to partnering with other companies to further develop this molecule.”

Publication information:
Efficacy of an isoxazole-3-carboxamide analog of pleconaril in mouse models of Enterovirus-D68 and Coxsackie B5.
Authors:
Thomas R. Lane a†, Jianing Fu b†, Barbara Sherry c, Bart Tarbet d,e, Brett L. Hurst d,e, Olga Riabova f, Elena Kazakova f, Anna Egorova f, Penny Clarke g, J. Smith Leser g, Joshua Frost h, Michael Rudy i, Kenneth L. Tyler g-i, Thomas Klose b, Alexandrina S. Volobueva j, Svetlana V. Belyaevskaya j, Vladimir V. Zarubaev j, Richard J. Kuhn b, Vadim Makarov f and Sean Ekins a#

Affiliations:
aCollaborations Pharmaceuticals Inc.; Raleigh, NC, USA.

bDepartment of Biological Sciences, Purdue University; West Lafayette, IN, USA.

cDepartment of Molecular Biomedical Sciences, North Carolina State University, College of Veterinary Medicine; Raleigh, NC, USA.

dInstitute for Antiviral Research, Utah State University; Logan, UT, USA.

eDepartment of Animal, Dairy and Veterinary Sciences, Utah State University; Logan, UT, USA

fResearch Center of Biotechnology RAS, 33-1 Leninsky prospect, 119071 Moscow, Russia.

gDepartment of Neurology, University of Colorado Anschutz Medical Campus; Aurora, CO, USA.

hDepartment of Immunology and Microbiology, Infectious Disease, Medicine and Neurology, University of Colorado Anschutz Medical Campus; Aurora, CO, USA.

iDepartment of Veterans Affairs; Aurora, CO, USA.

jSaint Petersburg Pasteur Institute, 14 Mira Street, 197101 Saint Petersburg, Russia.


† Co-first authors.
https://www.sciencedirect.com/science/article/abs/pii/S0166354223001328


Funding
National Institutes of Health (National Institute of Neurological Disorders and Stroke (NINDS) grant 1R01NS102164-01 (SE)
National Institutes of General Medical Sciences grant: R44GM122196-02A1 (SE)
National Institute of Allergy and Infectious Diseases (NIAID) program for non-clinical and pre-clinical services (SE)
This work was supported R01-AI011219 (R.J.K), and by NIH/NIAID contract HHSN272201700060C (R.J.K; PI: K. Satchell).
Funding was provided by the National Institute of Health contract number HHSN27220100041I, Task Order A16, from the Virology Branch, Division of Microbiology and Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institute of Health, USA.

https://www.einpresswire.com/article/639469598/collaborations-pharmaceuticals-announces-publication-of-new-enterovirus-d68-and-coxsackie-b5-inhibitor

Collaborations Pharmaceuticals Announces Publication of New HIV Reverse Transcriptase inhibitors to address HAND

RALEIGH, NC, USA, May 2, 2023/EINPresswire.com/ -- Collaborations Pharmaceuticals, Inc. (CPI) with collaborators at the Research Center of Biotechnology RAS, Moscow (Dr. Vadim Makarov); the University of North Carolina, Chapel Hill (Dr. Julie Nelson); as well as collaborators at the University of Cagliari are pleased to announce their NIH/NINDS funded work resulting in new peer reviewed publication entitled "N-phenyl-1-(phenylsulfonyl)-1H-1,2,4-triazol-3-amine as a new class of Human immunodeficiency virus (HIV) non-nucleoside reverse transcriptase inhibitors (NNRTI)."

HIV infection is one of the most important pathogens affecting mankind. There were approximately 1.5 million new cases in 2021 and 38.4 million living with HIV, amongst them 2.73 million were children. The disease can be managed with a cocktail of antiretroviral drugs including NNRTI. It has been reported that there is resistance against NNRTIs as well as side effects such as HIV associated neurocognitive disorders (HAND), which results in symptoms from minor problems with memory to severe dementia-like symptoms. We therefore need to have more diversity in the types of chemical structures assessed, in order to stand the best chance of addressing both drug resistance and HAND in future.

“We have identified the N-phenyl-1-(phenylsulfonyl)-1H-1,2,4-triazol-3-amine scaffold as possessing potent activity as an NNRTI. In the process we have developed over 100 analogs using a classical medicinal chemistry structure activity relationship (SAR) to optimize activity against wild-type, A17 mutant (K103N/Y181C) as well as other common mutant strains resistant to approved NNRTIs. This has led to the discovery of a series of optimized compounds with picomolar activity against wild-type HIV that retain activity against these clinically relevant mutants” said CPI CEO Dr. Sean Ekins.

“HIV remains a global threat and as such the need for additional treatments remains of utmost importance. Current therapies have yet to eradicate the prevalence of HAND; therefore, this still represents an important therapeutic goal that we have addressed. These molecules are a new class of HIV NNRTI and without likely toxicity seen with older drugs. We also look forward to partnering these molecules from our pipeline with other companies to further develop this molecule.”


Publication information:
N-phenyl-1-(phenylsulfonyl)-1H-1,2,4-triazol-3-amine as a new class of HIV-1 non-nucleoside reverse transcriptase inhibitor

Thomas Lane1#, Vadim Makarov2#*, Julie A. E. Nelson3#, Rick B. Meeker4, Giuseppina Sanna5, Olga Riabova2, Elena Kazakova2, Natalia Monakhova2, Andrey Tsedilin2, Fabio Urbina1, Thane Jones1, Ashley Suchy3 and Sean Ekins1*

1Collaborations Pharmaceuticals Inc., 840 Main Campus Drive, Lab, 3510, Raleigh, NC 27606, USA.; 2Research Center of Biotechnology RAS, Leninsky Prospekt 33-2, 119071, Moscow 119071, Russia; 3Department of Microbiology and Immunology, University of North Carolina, Chapel Hill, NC 27514, USA.; 4Department of Neurology, University of North Carolina, NC 27514, USA; 5Department of Biomedical Science, University of Cagliari, Monserrato, 09042, Italy.
# co-first authors


Funding
Research reported in this publication was supported by the National Institute of Neurological Disorders and Stroke of the National Institutes of Health under Award Number 1R01NS102164-01. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
We also kindly acknowledge NIH funding: R44GM122196-02A1 from NIGMS and 1R43ES031038-01 from NIEHS (PI – Sean Ekins). “Research reported in this publication was supported by the National Institute of Environmental Health Sciences of the National Institutes of Health under Award Number R43ES031038. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.” This work was supported by the University of North Carolina at Chapel Hill Center for AIDS Research (P30 AI050410).

https://www.einpresswire.com/article/629045104/collaborations-pharmaceuticals-announces-publication-of-new-hiv-reverse-transcriptase-inhibitors-to-address-hand

Collaborations Pharmaceuticals launches a new software product to predict acetylcholinesterase inhibitors

Collaborations Pharmaceuticals, Inc. announces a new commercial software product for predicting acetylcholinesterase (AChE) inhibitors that is now available.

RALEIGH, NORTH CAROLINA, USA, February 9, 2023 /EINPresswire.com/ -- AChE is an important enzyme and target for human therapeutics (for Alzheimer’s disease for example), environmental safety and global food supply. Inhibitors of this enzyme are widely utilized for pest elimination and can also be used as chemical weapons. AChE targeting pesticides can also act on non-target organisms, such as fish, amphibians and humans, and lead to toxicity.

We have recently exhaustively curated the public data for AChE inhibition which has enabled the development of machine learning models for seven different species. This work was recently published inChemical Research in Toxicology. We have subsequently created a software productMegaAChEthat can be used to score molecules for their potential inhibition of this enzyme in different species.

We have also made a website available to the scientific community so users can sample this capability megaache.collaborationspharma.com.

These models could be used to design new AChE inhibitors for CNS diseases as well as for the prediction of off-target toxicity as it is frequently included in in vitro panels and utilized by companies to access potential toxicity. As there are pesticides that target AChE inhibition as their mechanism, these models could also be used to help develop species specific molecules without human toxicity. Such models may therefore be useful for pharmaceutical, agrochemical, consumer product and chemical companies.

Funding
This work was funded by the National Institute of Environmental Health Sciences of the National Institutes of Health under Award Number 1R43ES033855-01 and a matching grant award under the FY 21-22 One North Carolina SBIR/STTR Matching Funds Program Solicitation.

Statement on dual-use:
The AChE machine learning models in MegaAChE have potential dual-use capabilities, and we therefore propose to implement restrictions to control who has access to these models and limit the number of molecules predicted when used on the website. We believe such precautions are necessary and these will evolve over time as we integrate software features to control this dual-use.

Collaborations Pharmaceuticals, Inc. and The University of Arizona Awarded A Phase II STTR award from NIGMS/NIH

Raleigh – The National Institute of General Medical Sciences (NIGMS) has awarded $1,729,532.72 to Collaborations Pharmaceuticals, Inc. (CPI) and the University of Arizona to generate in vitro data and develop MegaTransÒ machine learning software for drug discovery.

“The objective of MegaTrans® is to develop a new computational system and tools for integrating human transporter data into drug discovery pipelines, as well as enabling its analysis and visualization. It will also assist with developing computational tools for quantitatively modeling drug-drug interactions of xenobiotics. We will generate data for transporters which currently have a paucity of data in the public domain, use validated machine learning algorithms, access the latest curated datasets and develop a user-intuitive interface and visualization system to enable predictions. MegaTrans® will be developed as commercial quality software and then commercialized by licensing to companies, as well as used in fee-for-service consulting projects. In Phase I of this project, we have collaborated with Dr. Steven Wright and Dr. Nathan Cherrington at the University of Arizona to generate numerous models for important human drug transporters. CPI have also developed the prototype for MegaTransÒ, curating numerous transporter datasets then generating and tested the resulting models. This Phase II award will allow us to perform additional in vitro testing (at the University of Arizona) to expand our models and develop MegaTransÒ further as a commercial product available for licensing by pharmaceutical and other groups so they can benefit from our expertise.” said Sean Ekins, CEO, CPI.

Drs. Stephen Wright and Nathan Cherrington of the University of Arizona are long-term collaborators with Collaborations Pharmaceuticals, Inc. This joint effort builds upon extensive experience and extends the mechanistic understanding of drug disposition to the whole patient by accurately predicting functional disposition. This collaboration is now poised at the forefront of understanding xenobiotic disposition and predicting the mechanistic features that determine human exposure and response to drugs.

We are not aware of any other company pursuing such an approach to both generate new high value datasets or models, performing testing of their own models and creating a wide array of transporter models. MegaTrans® can be applied in these various companies or organizations to aid in decision making or in regulatory submissions.

Disclaimer

The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

About Collaborations Pharmaceuticals, Inc.

Collaborations Pharmaceuticals, Inc.® has developed software for data curation and machine learning called Assay Central® (www.assaycentral.org) as well as curated model bundles in MegaTox®, MegaTrans® and MegaPredict®. Most recently we have developed MegaSyn and UV-adVISor. CPI performs research and development on innovative therapeutics for multiple rare and neglected diseases and is located in laboratories in the NC State Incubator at Centennial campus. We have considerable experience with preclinical and computational approaches to drug discovery and toxicity prediction. For more information, please visit http://www.collaborationspharma.com/

About The University of Arizona

The University of Arizona, a land-grant university with two independently accredited medical schools, is one of the nation's top 50 public universities, according to U.S. News & World Report. Established in 1885, the university is widely recognized as a student-centric university and has been designated as a Hispanic Serving Institution by the U.S. Department of Education. The university ranked in the top 20 in 2020 in research expenditures among all public universities, according to the National Science Foundation, and is a leading Research 1 institution with $761 million in annual research expenditures. The university advances the frontiers of interdisciplinary scholarship and entrepreneurial partnerships as a member of the Association of American Universities, the 66 leading public and private research universities in the U.S. It benefits the state with an estimated economic impact of $4.1 billion annually. 

https://www.einpresswire.com/article/589055970/collaborations-pharmaceuticals-inc-and-the-university-of-arizona-awarded-a-phase-ii-sttr-award-from-nigms-nih

Collaborations Pharmaceuticals, Inc. and collaborators announce publication on the efficacy of a treatment for CLN1

A new paper published in the Journal of Clinical Investigation describes the efficacy of an enzyme replacement therapy in multiple species for Batten disease.

Collaborations Pharmaceuticals, Inc. (CPI) and collaborators at Washington University in St Louis, The Roslin Institute, Amicus Therapeutics, Edinburgh University, NHS Lothian, and University of Texas Southwestern Medical Center are pleased to announce a publication led by the laboratory of Dr. Jonathan Cooper and colleagues in this prestigious journal (https://www.jci.org/articles/view/163107) that describes the development of an enzyme replacement therapy for CLN1 Batten disease and provides a foundation for future development.

CLN1 disease is a fatal neurodegenerative lysosomal storage disorder resulting from mutations in the CLN1gene encoding the soluble lysosomal enzyme, palmitoyl-protein thioesterase-1 (PPT1). This neurodegenerative storage disorder primarily affects the brain and the retina of children and young adults, leading to dementia, blindness, epilepsy, and early death. There are currently no treatments available (other than palliative therapies) for this disease. Even gene therapy has proven less effective for this disease than for similar lysosomal storage diseases.

With substantial funding from NINDS we have now been able to demonstrate how monthly intracerebroventricular administration of human PPT1 produced statistically significant treatment effects in enzyme palmitoyl-protein thioesterase-1 (PPT1) deficient mice (Ppt1-/-), such as rescue of PPT1 enzyme activity, decreased secondary enzyme levels, decreased the loss of neurons in all regions of brain and spinal cord and improved gait and rotarod results. Additional data is also presented that shows a similar efficacy in a sheep model of the disease.

“These results suggest the efficacy and feasibility of the repeated ICV delivery of the recombinant enzyme and are an important next step before clinical testing. We were recently funded by the NIH/NINDS with a nearly $3M Phase II SBIR to manufacture the recombinant human PPT1 and conduct IND enabling toxicologystudies. This work will set the stage for future clinical studies. CPI holds the Orphan Drug and rare disease designations from the Food and Drug Administration for this potential treatment which we are focused on commercializing. We welcome discussions with other rare disease companies and potential partners that would be interested in learning more about this or other treatments in our rare disease pipeline” said CPI CEO Dr. Sean Ekins.

The ERT was originally developed in the laboratory of Dr. Sandra Hofmann at the University of Texas Southwestern Medical Center and the team involved in this multi-year collaborative project included Hemanth R. Nelvagal, Samantha L. Eaton, Sophie H. Wang, Elizabeth M. Eultgen, Keigo Takahashi, Steven Q. Le, Rachel Nesbitt, Joshua T. Dearborn, Nicholas Siano, Ana C. Puhl, Patricia I. Dickson, Gerard Thompson, Fraser Murdoch, Paul M Brennan, Mark Gray, Stephen N Greenhalgh, Peter Tennant, Rachael Gregson, Eddie Clutton, James Nixon, Chris Proudfoot, Stefano Guido, Simon G. Lillico, C. Bruce A. Whitelaw, Jui-Yun Lu, Sandra L Hofmann, Sean Ekins, Mark S Sands, Thomas M Wishart, Jonathan D. Cooper.

Funding for various members of the team was provided by NINDS National Institutes of Health grants R43 NS107079, R43NS107079-01S1, 3R43NS107079-01S2, R56 NS117635 and R01 NS124655, R01 NS100779, Hailey’s Heroes project grant, Biotechnology and Biological Sciences Research Council BB/J004316/1 and BB/P013732/1 ISP support to The Roslin Institute, The RS Macdonald Charitable Trust, Institutional Support from the Department of Pediatrics, Washington University in St Louis, School of Medicine and McDonnell International Scholars Academy award.

https://www.einpresswire.com/article/588874440/collaborations-pharmaceuticals-inc-and-collaborators-announce-publication-on-the-efficacy-of-a-treatment-for-cln1

Collaborations Pharmaceuticals, Inc. Awarded A Phase II SBIR award from NIEHS/NIH to Develop MegaTox software

Raleigh – The National Institute of Environmental Health Sciences (NIEHS) has awarded $1,710,006 to Collaborations Pharmaceuticals, Inc. (CPI) to develop new capabilities in their MegaTox® machine learning software that will be used for environmental, consumer product and drug discovery applications.

“CPI developed MegaTox® software to provide a comprehensive collection of ADME/Tox machine learning models. Pharmaceutical, consumer products, agrochemical and other chemistry focused companies possess structure-activity data generated over many decades of screening that is not in the public domain, and this data is primarily only accessible to the cheminformatics experts in each company. Outside of these companies small pharmaceutical, biotech companies and academics must rely on data from public databases, commercial databases and their own data. Integrating such data from diverse sources and processing with algorithms to build machine learning models that can help to enable predictions for new compounds is an important undertaking. In Phase I of this project, we have developed the prototype for MegaTox®, we curated toxicity datasets then generated and tested hundreds of models. We also collaborated with numerous academic laboratories and performed fee-for-service work with multiple commercial companies to apply these models. We are excited by this Phase II award as it will enable us to integrate novel graph-based and other machine learning models, apply read across and adverse outcome pathway methods as well as generate validated models for in vivo data for non-mammalian species that can be licensed by other companies so they can benefit from our expertise.” said Sean Ekins, CEO, CPI.

We expect to develop thousands of additional ADME/Tox models over the course of this project.  We are not aware of any other company pursuing such an approach to both generate new high value datasets or models, performing testing of their own models and creating a wide array of toxicity models. MegaTox® is a product available for licensing by pharmaceutical, consumer product, agrochemical and regulatory groups as well as used in our fee-for-service consulting. The models generated could also be used for generative AI approaches such as those applied in our MegaSyn product. With such a wide array of models and approaches to address customer needs MegaTox® can be applied in various companies or organizations to aid in decision making or in regulatory submissions.

Disclaimer

“Research reported in this publication was supported by the NATIONAL INSTITUTE OF ENVIRONMENTAL HEALTH SCIENCES of the National Institutes of Health under Award Number 2R44ES031038-02A1. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.”

About Collaborations Pharmaceuticals, Inc.

Collaborations Pharmaceuticals, Inc.® has developed software for data curation and machine learning called Assay Central® (www.assaycentral.org) as well as curated model bundles in MegaTox®, MegaTrans® and MegaPredict®. Most recently we have developed MegaSyn and UV-adVISor. CPI performs research and development on innovative therapeutics for multiple rare and neglected diseases and is located in laboratories in the NC State Incubator at Centennial campus. We have considerable experience with preclinical and computational approaches to drug discovery and toxicity prediction. For more information, please visit http://www.collaborationspharma.com/

https://www.einpresswire.com/article/584008956/collaborations-pharmaceuticals-inc-awarded-a-phase-ii-sbir-award-from-niehs-nih-to-develop-megatox-software?r=pa7NljPWpYYrBoBI3Y

Collaborations Pharmaceuticals, Inc. Awarded A Phase II SBIR From NIH/NINDS To Develop Batten Disease Treatment

June 9th, 2022

Raleigh – The National Institute of Neurological Disorders and Stroke has awarded a $2,999,846 SBIR grant to Collaborations Pharmaceuticals, Inc.

A Phase II SBIR awarded to Collaborations Pharmaceuticals, Inc. by the National Institutes of Health / National Institute of Neurological Disorders and Stroke.

We are excited to be awarded this phase II SBIR grant for “Manufacture of an intracerebroventricular Enzyme Replacement Therapy for CLN1 Batten Disease” said Collaborations Pharmaceuticals, Inc. (CPI) CEO Dr. Sean Ekins.

The neuronal ceroid lipofuscinoses (NCLs) are a group of incurable neurodegenerative storage disorders primarily affecting the brain and the retina of children and young adults, leading to dementia, blindness, epilepsy, and early death. There are currently no treatments available (other than palliative therapies) for this fatal disease.

With substantial funding from NINDS with academic collaborators we have been able to demonstrate how monthly intracerebroventricular administration of human PPT1 produced statistically significant treatment effects in enzyme palmitoyl-protein thioesterase-1 (PPT1) deficient mice (Ppt1-/-), such as rescue of PPT1 enzyme activity, decreased secondary enzyme levels, decreased the loss of neurons in all regions of brain and spinal cord and improved gait and rotarod results. We have also been supported by NINDS to participate in NIH I-Corps which provided valuable experience in commercialization. In addition, a diversity supplement has also assisted us in hiring personnel and further work on this disease.

“We now propose in this Phase II SBIR to manufacture recombinant human PPT1 and conduct IND enabling toxicology studies. This work marks key steps in the further development of this enzyme replacement therapy (ERT) that is critical for CLN1 patients, setting the stage for future clinical studies and the development of a potential treatment. CPI already holds the Orphan Drug and rare disease designations from the Food and Drug Administration for this approach. This project fulfils our goal to pursue treatments for ultra-rare diseases and this new funding will catalyze the development of this ERT. We are now well positioned to continue the development of this potential treatment for a devastating disease in children. We welcome discussions with other rare disease companies and potential partners to help bring this to the families and children awaiting a treatment”.

This grant was awarded on the same day as International Batten Awareness Day which aims to honor and support all those affected by Batten disease and to raise global awareness.

About Batten Disease

The infantile onset form CLN1 disease is caused by mutations in the CLN1/PPT1 gene, which codes for the lysosomal enzyme palmitoyl-protein thioesterase-1 (PPT1) resulting in a reduction or absence of enzyme activity. CLN1 disease usually presents between 6 and 24 months of age and there are 2-3 children with this form identified each year and there are over 20 known children with CLN1 in the US and 11 in Brazil (with likely many more undiagnosed globally).

About Collaborations Pharmaceuticals, Inc.

Collaborations Pharmaceuticals, Inc.® (CPI) performs research and development on innovative therapeutics and has a preclinical pipeline of treatments for rare and neglected diseases. In addition, CPI has developed software for data curation and machine learning called Assay Central® (www.assaycentral.org) as well as curated model bundles in MegaTox®, MegaTrans® and MegaPredict®. Most recently we have developed generative software called MegaSyn and UV-adVISor for predicting UV-Vis spectra for small molecules. CPI is located in laboratories in the NC State Incubator at the Centennial campus. We have considerable experience with preclinical and computational approaches to drug discovery and toxicity prediction. For more information, please visit http://www.collaborationspharma.com/

https://www.einpresswire.com/article/576063309/collaborations-pharmaceuticals-inc-awarded-a-phase-ii-sbir-from-nih-ninds-to-develop-batten-disease-treatment