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Press Release: Absorption Systems Awarded 5-year IDIQ Contract by the FDA

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To conduct preclinical testing of locally-acting topical dermal and ophthalmic drug product formulations

EXTON, PA – October 09, 2019 – Absorption Systems, a global leader in translational medicine, pharmacokinetic testing of drugs, and cell and gene therapy, announces that it has been awarded a 5 year IDIQ (Indefinite Delivery Indefinite Quantity) contract, for a total amount of $3.5 million, by the U.S. Food and Drug Administration (FDA). The objective is to support FDA’s development and refinement of drug product approval standards for locally-acting topical dermal and ophthalmic drug product formulations.

Under the contract, Absorption Systems will develop and conduct appropriate in vitro release tests (IVRT) and in vitro permeability test (IVPT), in accordance with published FDA guidance documents. The company will also perform in vivo studies to evaluate the pharmacokinetics, distribution, and efficacy of topically administered drugs that act locally on the skin or in the eye.  Ultimately, fulfillment of this contract will help ensure that safe, effective, and high-quality complex generic drugs are available to the public.

Locally acting complex drugs are particularly challenging for drug regulators to evaluate because they may not be absorbed or may be absorbed in such small amounts that it is not feasible to simply compare plasma levels to determine whether two products are bioequivalent, as can be done with orally administered drugs. This award recognizes the core strength of Absorption Systems as a developer of biopharmaceutics tools that can predict human outcomes. These next-generation technologies improve our fundamental understanding of how the critical quality attributes (CQAs) of formulations affect the local and/or systemic pharmacokinetics and pharmacodynamics of drugs. This, in turn, contributes to the establishment of in vitro-in vivo correlation (IVIVC) or in vitro-in vivo relationship (IVIVR) models to help ensure the bioequivalence of complex drug products.

Vatsala Naageshwaran, Senior Vice President of Operations of Absorption Systems, commented “As a testing facility we have focused on the increased utilization of biopharmaceutics principles, advances in non-clinical tools for guiding formulation strategy and predicting in-vivo performance, and demonstrating product equivalence without the need for clinical studies. The FDA has prioritized research to investigate reliable and reproducible scientific tools to help understand chemical and biological complexity and establish surrogate models that are predictive of clinical performance. Our goal is to support these efforts, to enable significant regulatory outcomes in categorizing appropriate paths for development and approval thereby enhancing patient access to generic ophthalmic and topical drug products.”

About Absorption Systems

Absorption Systems, founded in 1996, assists pharmaceutical, biotechnology and medical device companies in identifying and overcoming ADMET (Absorption, Distribution, Metabolism, Excretion and Toxicity) barriers in the development of drugs, biologics and medical devices. The company’s mission is to continually develop innovative research tools that can be used to accurately predict human outcomes or to explain unanticipated human outcomes when they occur. IDAS2, a novel in vitro test system for simultaneous screening of drug absorption and dissolution, exemplifies Absorption Systems’ commitment to innovation and is soon to be an industry standard for formulation assessment. Absorption Systems, with facilities near Philadelphia, PA, in San Diego, CA, and in Medford, MA, serves customers throughout the world. For information on the company’s comprehensive contract services, applied research programs, and proprietary test systems, please visit www.absorption.com.

Gene Therapy in Combination: A Promising Avenue for Cancer Treatment

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Gene therapies (GTs) are a rapidly growing field of cancer treatment, with almost 2000 active clinical trials worldwide representing 65% of total gene therapies. There is particular interest in the use of GTs in combination with existing drug treatments for cancers to provide more effective therapies. Some of the reasons behind this optimism include:

Improving response rates… Immune checkpoint inhibitors (IHIs) like PD-1 work by blocking the shutdown of the normal immune response to cancer cells, promoting tumor clearance by the immune system. IHI drugs like pembrolizumab and nivolumab have relatively high response rates (33 and 40% respectively), but remain ineffective in over half of patients. Gene therapies that work in parallel with IHIs to boost immune response offer attractive targets. A recent Phase Ib study has shown that pembrolizumab in combination with an oncolytic viral therapy (T-VAC) boosted response rate to 62% in metastatic melanoma patients. Psioxus Therapeutics currently has a Phase I trial for Enadenotucirev in combination with PD-1 inhibitors to treat Advanced Epithelial tumors.

Tackling redundancy… A major barrier to effective cancer treatment is the ability for tumors to use multiple redundant signalling pathways to the same end. For example, both EGFR and AKT signalling pathways can help cancer cells escape apoptotic cell death signals, meaning blocking only one pathway can be ineffective. Genprex has developed a non-viral means to deliver the TUSC2 gene into tumors. TUSC2 is a tumor suppressor gene that promotes apoptosis by targeting EGFR and AKT pathways. Phase I/II trials of Oncoprex in combination with EGFR inhibitors in lung cancer patients are ongoing, but initial data suggests a 78% control rate of the disease. In another example, a recent publication in Gene Therapy showed that p53 expression in combination with chemotherapy led to 100% survival rate in a preclinical model of prostate cancer.

More bang for your buck… New gene delivery vectors are being developed that can introduce multiple genes into tumors. In a multifaceted disease like cancer, having the ability to introduce as many interventions as possible is critiThis approach has a long history, with the first trial in 1995 for two interleukin-12 (IL-12) genes to treat kidney cancer. More recently, IL-12 was added in combination with a suicide gene and showed improved response in a preclinical model of prostate cancer.

Shared learnings… Advances in genes therapies in other disease fields have important implications for future cancer treatment. Last year, the FDA approved the first ever siRNA therapy for the treatment of a rare hereditary neuropathy. siRNAs offer the ability to silence gene expression. Many cancers are driven by expression of mutated or over-active ‘oncogenes’. siRNAs are a promising way to enable inhibition of targets that are not amenable to pharmacological intervention and could provide attractive therapies when combined with other GTs or drugs.

Looking forward, gene therapies are only going to become more commonplace in the treatment of cancer. Absorption Systems’ ACF Bioservices platform is supporting these therapies to reach market safely and expediently through a wide range of in vitro and in vivo services.

Webinar: Challenges in Adapting Neutralizing and Anti-Drug Antibody Assays to Gene Therapies

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Webinar Download

Originally Hosted:
Thursday, October 10, 2019
11:00am – 12:00pm EDT

Challenges in Adapting Neutralizing and Anti-Drug Antibody Assays to Gene Therapies

This presentation summarizes a recently published whitepaper by Absorption Systems.

We will discuss the process of developing and validating tests for detecting ADAs and NAbs. Given that the host immune response may be triggered by the AAV vector capsid, the therapeutic gene/ RNA, and the translated protein, a multi-tiered approach is required.  There are numerous challenges in developing ADA tests. These include:

  • Selection of the optimal methodology
  • Choice of cell line for NAb assays
  • Determining the appropriate cut-off point
  • Utilizing appropriate positive controls

Here we share our experience in overcoming these challenges and set out best-practice criteria.

Download Recording

NAbbed Again: Challenges in Developing Neutralizing and Anti-drug Antibody Assays for Gene Therapy

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Abstract

Gene Therapy is one of the most recent and fast-growing fields of medicine, which promises to change the way we approach and treat different diseases, including many which were considered incurable even a decade ago.  Recombinant adeno-associated virus (rAAV) is the most commonly used vector for therapeutic gene/RNA delivery because of its safety profile, limited immunogenicity and broad tissue tropism. Despite being the least immunogenic therapeutic viral vector AAV can evoke anti-drug antibodies (ADAs), which may be either pre-existing or developed after the onset of treatment. The absence of ADAs, especially neutralizing antibodies (NAbs), is often a prerequisite for enrolment in gene therapy trials, therefore, developing sensitive and robust assays for these antibodies is crucial.

We discuss the process of developing and validating tests for detecting ADAs and NAbs. Given that the host immune response may be triggered by the AAV vector capsid, the therapeutic gene/ RNA, and the translated protein, a multi-tiered approach is required.  There are numerous challenges in developing ADA tests. These include: selection of the optimal methodology, choice of cell line for NAb assays, determining the appropriate cut-off point, and utilizing appropriate positive controls. Here we share our experience in overcoming these challenges and set out best-practice criteria.

Demand Exceeds Supply in Cell & Gene Therapy Workforce

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The recent rapid growth of cell and gene therapies (CGTs) has led to a gap in the talent market that represents both a challenge and opportunity for the sector.

Whilst the research base for CGTs is well-established, the last five years have seen a ramping up of therapies being brought to market and treating patients. In the US over half of FDA approved CGTs gained authorization since 2014, bringing the current total up to 17. With almost 300 CGTs currently in development, the UK Cell and Gene Therapy Catapult predicts tens of thousands of patients to be benefiting from CGTs in the coming decade.

Figure 1. Number of CGTs with market authorization (MA) from 2001-2018. Taken from Cuende et al. 2018

The increasing demand for current and upcoming therapies is placing significant strain on parts of the CGT pipeline. As the industry is young and highly specialized, this growth has exposed gaps in the talent pool. Several key areas include:

  • Manufacturing… Mass production of critical components of cell and gene therapies, such as viral vectors, is a critical bottleneck for CGT companies. Production of CGTs is complex and highly regulated, with sites having to comply with strict “current Good Manufacturing Practice” (cGMP) standards set by national regulators. Scientists with experience working in these environments are highly sought after, and decisions about production facility locations often hinge on the availability of manufacturing expertise. Allogene Therapeutics’ recent plans to build a new manufacturing site in the biotech hub in San Francisco’s Bay area highlight this. The UK (a world-leader in CGT manufacturing), has announced apprenticeship schemes to help meet the need for these skills.
  • Senior management… The correct senior management is critical to the success of CGT companies. A recent report by the RSA concluded that fast delivery times and technical challenge of CGTs required management teams with a broader skills-base than in other drug discovery companies. Analyzing 182 CGT companies, the report finds that the majority (72%) of founders have scientific/medical expertise with a significant minority (24%) having managerial experience. Ensuring that operational and commercial management are on-board early in development was highlighted as critical to company success.
  • Regulatory expertise… The regulatory environment for CGTs is constantly developing. In 2018, the FDA and EMA released new regulatory guidelines for design, manufacture and testing of gene therapies. Whilst only 4% of CGT company founders have regulatory experience, this skill set becomes critical in commercialization and/or exit phases of growth.

As the CGT market continues to expand, there will only be an increasing demand for those with the scientific, manufacturing and commercial experience to tackle the unique challenges facing the CGT sector. In the interim, many CGT companies are using CROs like Absorption Systems to outsource expertise in specialist areas such as analytical testing. These services are crucial to bridge the talent gap whilst training schemes, attractive career paths and continued investment help the market reach its full potential in the coming decade and beyond.