Feinstein Institutes | November 29, 2022
Peptides, short sections of proteins made up of chains of amino acids, are an important area of new drug development due to their potential for increased specificity and fewer side effects. Their main drawback is the rapid degradation by the body’s own enzymes, which has led to the need to develop longer-lasting peptide alternatives. Azapeptides are one type of peptide alternative that has shown great potential as therapeutics, exemplified by the HIV drug, Atazanavir.
A new paper published in the journal Nature Communications by researchers at The Feinstein Institutes for Medical Research details proprietary reagents and methods that can selectively replace only those amino acids known to be targeted by enzymes with more stable versions to create azapeptides.
The paper, authored by Yousef Al-Abed, PhD, co-director of the Institute of Bioelectronic Medicine at the Feinstein Institutes, describes the proprietary building blocks and method used to conduct systematic and robust peptide editing, replacing targeted amino acids with aza-amino acids to form more stable and efficient azapeptides. The azapeptides created could expedite the novel therapies to treat many diseases and conditions, including metabolic disease, influenza, pulmonary arterial hypertension, Crohn’s disease, arthritis, and irritable bowel diseases, among others.
The paper, titled “Thiocarbazate building blocks enable the construction of azapeptides for rapid development of therapeutic candidates,” provides multiple examples of azapeptide construction, screening, and selection using a known peptide inhibitor of inflammation and bradykinin.
“Peptides as drug candidates are easy to discover yet difficult to develop as final drugs because of their fast degradation in our body. A technology that circumvents these problems inherent to native peptides will change the future landscape of drug discovery. Our technology provides a platform of tools that enable minimal modification of any peptide at highly prone degradation sites, thereby increasing its life span in our body, allowing it more time to find its target and neutralize it.”
Through peptide editing, going from peptide to azapeptide, using methods that Dr. Al-Abed and his team detail in the new paper, scientists can create potential therapies that have enhanced characteristics compared to natural peptides, like longer stabilities in a more efficient manner. Dr. Al-Abed and his team have precisely engineered thiocarbazates (a novel functional group) in the proprietary process to achieve an almost universal and more efficient means to selectively replace amino acids in a peptide chain.
“Peptide drugs are a crucial resource for patients and the global pharmaceutical industry,” said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes and Karches Family Distinguished Chair in Medical Research. “Dr. Al-Abed and his team invented a fully automated chemical strategy using novel chemistry to make new peptide drugs which is an important new avenue for peptide drug discovery.”
About the Feinstein Institutes
The Feinstein Institutes for Medical Research is the research arm of Northwell Health, the largest health care provider and private employer in New York State. Home to 50 research labs, 3,000 clinical research studies and 5,000 researchers and staff, the Feinstein Institutes raises the standard of medical innovation through its five institutes of behavioral science, bioelectronic medicine, cancer, health innovations and outcomes, and molecular medicine. We make breakthroughs in genetics, oncology, brain research, mental health, autoimmunity, and are the global scientific leader in bioelectronic medicine – a new field of science that has the potential to revolutionize medicine.
VIEWS AND ANALYSIS, PHARMA TECH
Anaqua and PatSnap | December 08, 2022
Anaqua, the leading provider of innovation and intellectual property management technology announced it is partnering with innovation intelligence platform PatSnap. The collaboration will deliver an enhanced IP management solution to provide Anaqua’s pharmaceutical industry clients with a more detailed and informed view of the market and competitive landscape for key use cases. The combined Anaqua AQX® Pharma and PatSnap Synapse solutions will help AQX Pharma clients further enhance competitiveness, keep up with the fast-paced requirements of the current market, eliminate duplicated work across functions, as well as identify and realize new areas of investment.
PatSnap is a platform for innovation intelligence, utilizing AI technology – including machine learning, computer vision and Optical Character Recognition that facilitates access to an unrivaled breadth of data that is connected in a meaningful way. PatSnap’s Synapse solution will give AQX Pharma users access to millions of data points, sourced utilizing best-in-class algorithms and expert manual curation, providing a comprehensive 360-degree market view and adding strategic value to drug development processes and strategy.
The new integration is in response to client requests to access more pharmaceutical market data within the AQX platform to improve internal team efficiency and streamline processes. The integration will support sharing deeper intelligence between clients and partners and simplify the route of administration through features such as docket personalization that will allow teams to focus on their most strategic initiatives. Specifically, the combined system will address the use case of conducting stage gate reviews and automatically kick off relevant workflows.
“AQX Pharma helps clients manage the business aspects of pharmaceutical IP management, while Synapse powers the external aspects of drug discovery through external and competitor drug discovery processes. Together, we’re using our unique capabilities to enable best practices in Life Science lifecycle stage gate reviews. We’re pleased to offer a complete pharma innovation solution, which can be further complemented by leveraging Anaqua’s strategic integrations, such as its innovation management solution, ideaPoint.”
Vincent Brault, SVP of Product & Innovation at Anaqua
Ray Chohan, Co-founder and VP of Corporate Development at PatSnap, said, “PatSnap is thrilled to team up with Anaqua, who are true innovators in life sciences IP management. Synapse will empower users of AQX Pharma to comb through millions of data points to discover and commercialize new therapies.”
Anaqua, Inc. is a premier provider of integrated intellectual property management technology solutions and services for corporations and law firms. Its IP management software solutions, AQX® and PATTSY WAVE®, both offer best practice workflows with big data analytics and tech-enabled services to create an intelligent environment designed to inform IP strategy, enable IP decision-making, and streamline IP operations, tailored to each segment’s need. Today, nearly half of the top 100 U.S. patent filers and global brands, as well as a growing number of law firms worldwide, use Anaqua’s solutions. Over one million IP executives, attorneys, paralegals, administrators, and innovators use the platform for their IP management needs. The company’s global operations are headquartered in Boston, with offices across the U.S., Europe, Australia, and Asia.
PatSnap’s AI-powered innovation intelligence platform transforms billions of innovation data points into actionable intelligence for over 10,000 global customers in more than 50 countries. With an unrivaled breadth of data, including patents, drug information, technology news, market reports, scientific literature, and more, PatSnap breaks barriers to connect innovators with insights.
BUSINESS INSIGHTS, PHARMACY MARKET
Phio Pharmaceuticals Corp. | December 22, 2022
Phio Pharmaceuticals Corp., today announced it expects to file an IND in the US in the first half of 2023 for a Phase 1b clinical trial of its INTASYL™ compound, PH-762. Phio is a clinical stage biotechnology company whose proprietary INTASYL self-delivering RNAi technology is designed to make immune cells more effective in killing tumor cells. PH-762 has been shown to reduce the expression of PD-1, a protein that inhibits T cells' ability to kill cancer cells. When administered intratumorally in preclinical models PH-762 primes an anti-tumor immune response, and inhibits tumor growth.
Phio is currently conducting a Phase 1b clinical trial of PH-762 for the treatment of advanced melanoma at the Gustave Roussy Institute, one of the largest cancer centers in Europe. Phio expects to commence a US Phase 1b clinical trial early in the 2nd half of 2023. The initial US trial is expected to focus on the treatment of cutaneous squamous cell carcinoma and other selected cutaneous malignancies, following successful regulatory review of the IND.
"Therapeutic interventions for cSCC are limited, and there is increasing unmet medical need. As cSCC tumors comprise approximately 51% of the total incidence of solid tumors in the US, excluding basal cell cancers, we recognize the growing need for alternative therapies for cSCC," said Robert Bitterman, Phio's Principal Executive Officer and Executive Chairman. Mr. Bitterman has over 25 years of executive leadership experience in the pharmaceutical and biologic life science industry with a proven track record in operations, finance and investor relations.
"While monoclonal antibody therapies are available for the treatment of cSCC, mechanistically they only block the interaction between PD-1 and PD-L1 on the cell surface. PH-762 also has the potential to address PD-1 inside the T cell, essentially further enhancing the activity of the T cell to kill the tumor cells," said Dr. James Cardia, Phio's Vice President of Scientific Operations. Dr. Cardia led the team that discovered INTASYL.
"PH-762 has the potential to meet a significant medical need in patients who have failed to respond to mAbs, as well as those with resectable and metastatic solid tumors. Phio is committed to advancing the study of PH-762 to provide a meaningful therapy for patients with a broad range of solid tumors."
Dr. Mary Spellman, Phio's Medical and Clinical Development Advisor
INTASYL compounds are chemically modified siRNAs that are designed to provide efficient, spontaneous cellular uptake and potent, long lasting intracellular activity targeting a broad range of cell types and tissues. INTASYL drugs precisely target specific proteins that reduce the body's ability to fight cancer, without the need for specialized formulations or drug delivery systems. INTASYL has demonstrated preclinical efficacy in both Direct-to-Tumor and Adoptive Cell Therapy applications. In comparison to biologics and cell and gene therapies, INTASYL has a favorable pre-clinical toxicity and safety profile, and a streamlined chemical synthesis that reduces costs and offers substantial convenience to the prescriber and patient. Phio believes that INTASYL is the only self-delivering RNA interference technology focused on immuno-oncology therapeutics.
About Phio Pharmaceuticals
Phio Pharmaceuticals Corp. is a clinical stage biotechnology company whose proprietary INTASYL™ RNAi technology is designed to make immune cells more effective in killing tumor cells. Phio believes that INTASYL is the only self-delivering RNAi technology focused on immuno-oncology therapeutics. INTASYL drugs precisely target specific proteins that reduce the body's ability to fight cancer, without the need for specialized formulations or drug delivery systems.
VIEWS AND ANALYSIS, PHARMA TECH
Virpax Pharmaceuticals | December 15, 2022
Virpax® Pharmaceuticals, Inc. a company specializing in developing non-addictive products for pain management, post-traumatic stress disorder, central nervous system disorders and viral barrier indications, today announced that it has received pre-Investigational New Drug application guidance from the U.S. Food and Drug Administration for NobrXiol™, the Company’s product candidate for the delivery of cannabidiol in the management of epilepsy in children and adults. NobrXiol utilizes the Nanomerics Molecular Envelope Technology as its delivery system to cross the blood brain barrier, propelling the cannabidiol nanoparticles through the nose to the brain via the olfactory nerve.
The main purpose of a pre-IND submission is to obtain FDA guidance on the overall development plan for a new drug and to identify any need for further data prior to submitting the IND.
“Virpax now has guidance on how to proceed with the IND enabling studies and possible regulatory pathways to pursue for NobrXiol. Based on the written responses from the FDA and its recommendations, we believe that we can proceed with the next steps in the process towards an IND application for this product candidate.”
Dr. Sheila A. Mathias, Chief Scientific Officer for Virpax
“This is a significant step forward for the NobrXiol project and we are very pleased with the outcome of the pre-IND meeting with the FDA,” said Anthony P. Mack, Chairman and CEO of Virpax. “We believe this product candidate has potential benefits over existing oral CBD treatments for epilepsy including Dravet Syndrome and Lennox-Gastaut Syndrome. We believe that by using the MET delivery system there may be significant advantages for patients including fewer side effects, avoidance of drug-to-drug interaction and lower dosing of CBD required,” concluded Mr. Mack.
Virpax has acquired the exclusive worldwide rights from Nanomerics to use Nanomerics' MET platform for the nasal delivery of cannabidiol or the management of epilepsy in children and adults. As part of this agreement, Nanomerics is developing an investigational formulation delivered via the nasal route to enhance CBD transport to the brain which could potentially eliminate any drug interaction issues and bypass the digestive system, possibly eliminating many of the side effects associated with the product currently in use on the market. Nanomerics demonstrated the ability of its platform technology to deliver CBD directly to the brain in an animal model.
About Virpax Pharmaceuticals
Virpax is developing branded product candidates for non-addictive pain management and neurological disorders using its proprietary technologies that optimize and target drug delivery. Virpax is initially seeking FDA approval of its three different patented drug delivery platforms. Epoladerm™ is a topical diclofenac spray film formulation being developed to manage pain associated with osteoarthritis of the knee. Probudur™ is a single injection long-acting liposomal bupivacaine formulation being developed to manage post-operative pain. Envelta™ is an intranasal Molecular Envelope Technology enkephalin formulation being developed for the management of acute and chronic pain, including pain associated with cancer, as well as post-traumatic stress disorder under the name PES200. MET technology is also used in AnQlar™, a candidate to inhibit viral replication caused by influenza or SARS-CoV-2. Virpax acquired global rights to NobrXiol™, a product candidate for the nasal delivery of a pharmaceutical-grade cannabidiol for the management of epilepsy in children and adults.