New biotherapeutics both drive and benefit from innovative development technologies.

Innovation Demands and Spurs Invention

FDA releases some J&J vaccines from Emergent facility; EU puts release on hold

Novavax Reports 90% Efficacy for Vaccine

Complex formulations, personalized medicines, and more are driving innovations in drug packaging.

Injecting Innovation into Drug Packaging

A task force establishes scientific data for integrity test activities. 

Reducing Sterile Filtration Risk

Automation offers benefits for sterile manufacturing in 503B facilities. 

Robotics for Drug Compounding

CureVac announced results on June 17, 2021 of its second interim analysis of its Phase IIB/III study for its first-generation COVID-19 vaccine candidate, CVnCoV. The vaccine candidate demonstrated an interim vaccine efficacy of 47% against COVID-19 disease of any severity and did not meet prespecified statistical success criteria, reported the company, which pointed to the context of at least 13 variants of the virus in the study population subset that was assessed. The company communicated available data to the European Medicines Agency (EMA), and the Data Safety Monitoring Board (DSMB) confirmed a favorable safety profile for CVnCoV. CureVac noted that at the conclusion of the study, all the data will be assessed to determine the most appropriate regulatory pathway.

In the HERALD study, which CureVac conducted with Bayer, approximately 40,000 participants in 10 countries in Latin America and Europe were enrolled. The second interim analysis included 134 cases, occurring at least two weeks after administration of the second dose. These cases were due to a range of variants, with only one case due to the original SARS-CoV-2 virus. Interim results suggest efficacy in younger participants.

“While we were hoping for a stronger interim outcome, we recognize that demonstrating high efficacy in this unprecedented broad diversity of variants is challenging. As we are continuing toward the final analysis with a minimum of 80 additional cases, the overall vaccine efficacy may change,” said Dr. Franz-Werner Haas, Chief Executive Officer of CureVac, in the press release. “In addition, the variant-rich environment underlines the importance of developing next-generation vaccines as new virus variants continue to emerge.”

CureVac said it remains committed to COVID-19 vaccine development and pointed to partnership with GlaxoSmithKline to develop second-generation COVID-19 vaccine candidates based on new mRNA backbones. The newer candidates will include potential variants in multivalent vaccine formats as well as combination vaccines for potential protection against multiple infectious diseases in one vaccine. CureVac and GSK expect to enter clinical testing in the third quarter of 2021 with a second-generation vaccine candidate, with the goal of introducing the vaccine in 2022, subject to regulatory approval.

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CureVac’s first-generation COVID-19 vaccine candidate, CvnCoV, did not meet efficacy targets in a second interim analysis. 

CureVac Releases Interim Results from COVID-19 Vaccine Candidate Trial

The US National Institute of Allergy and Infectious Diseases (NIAID) 

 on June 17, 2021 that the Biden administration will be investing more than $3 billion for the development and manufacturing of antiviral medicines. The National Institutes of Health (NIH), NIAID, and the Biomedical Advanced Research and Development Authority (BARDA) will collaborate to respond to the urgent need for COVID-19 antivirals through the Antiviral Program for Pandemics. The plan will spur the availability of lifesaving COVID-19 medicines and prepare for future viral threats by building discovery and development platforms for antivirals for other viruses with the possibility of becoming a pandemic.

NIH will evaluate, prioritize, and advance antiviral candidates to Phase II clinical trials as part of the plan. More than $300 million will be provided for research and lab support, almost $1 billion will be provided for preclinical and clinical evaluation, and nearly $700 million will be provided for development and manufacturing through NIAID and BARDA. Additionally, $1.2 billion will support the creation of Antiviral Drug Discovery (AViDD) Centers for Pathogens of Pandemic Concern for the discovery and development of antiviral drugs. The centers will first target coronaviruses and then expand to other viruses with pandemic potential.

“New antivirals that prevent serious COVID-19 illness and death, especially oral drugs that could be taken at home early in the course of disease, would be powerful tools for battling the pandemic and saving lives,” said Dr. Anthony Fauci, chief medical adviser to the President and NIAID Director, in a 

. “Through multidisciplinary collaborations among leading scientists in academia and industry, this investment from the American Rescue Plan to create the Antiviral Program for Pandemics will help inspire medical innovation and build on the extraordinary success we have seen in developing the COVID-19 vaccines.”

“This plan builds on the long, successful partnership between BARDA and NIH to develop vaccines, therapeutics and diagnostics, from combatting Ebola, anthrax and smallpox, to now tackling COVID-19,” said BARDA Director Gary Disbrow, PhD, in the release. “Throughout this public health emergency, we have worked with industry to develop treatments to reduce severe illness, hospitalizations, and deaths, and this proposal will help us go even further to accelerate and deliver more safe and effective treatments that are easy to administer.”

The US government will invest $3 billion from the American Rescue Plan into a COVID-19 antiviral development strategy.

Biden Administration Invests in Antiviral Medicines

GlaxoSmithKline (GSK) and iTeos Therapeutics announced on June 14, 2021 that they have signed an agreement to co-develop and co-commercialize EOS-448, an anti-T-cell immunoreceptor with immunoglobulin (Ig) and immunoreceptor tyrosine-based inhibitory motif domains (TIGIT) monoclonal antibody (mAb) in a deal worth up to nearly $2.08 billion.

Under the agreement, iTeos will receive an upfront payment of $625 million and will be eligible to receive up to an additional $1.45 billion in milestone payments if certain development and commercial milestones are achieved. GSK and iTeos will share responsibility and costs for the global development of EOS-448 and will jointly commercialize and equally split profits in the United States. Outside of the US, GSK will receive an exclusive license for commercialization, and iTeos will receive tiered royalty payments.

EOS-448 is currently in Phase I development as a potential treatment for patients with cancer. GSK and iTeos plan to start combination studies of EOS-448 with dostarlimab in 2022. TIGIT, which is part of the CD226 checkpoint axis, has demonstrated potential as a promising target for the next generation of immuno-oncology therapies based on preclinical data and data from a Phase II clinical trial. With this collaboration, GSK now has access to antibodies that synergistically target all three known CD226 checkpoints—TIGIT, CD96, and poliovirus receptor-related immunoglobulin domain-containing (PVRIG).

“Immuno-oncology has transformed cancer care but unfortunately less than 30 percent of patients respond to treatment with the current leading immune checkpoint inhibitors. Based on the underlying science, we believe that combinations of a PD-1 [programmed death-1], TIGIT, CD96 and PVRIG inhibitor could become transformative medicines for many patients with cancer. We are excited to collaborate with the team at iTeos and, together, we can play a leading role in the next generation of immuno-oncology therapies,” said Hal Barron, chief scientific officer and president R&D, GSK, in a company press release.

“Through this transformative collaboration, iTeos now has access to GSK’s best-in-class resources, which will provide us with a significant advantage in a highly competitive, global market. We have chosen GSK because of their commercial capabilities, experience in immuno-oncology, and their commitment to invest in the rapid advancement of our TIGIT program and create a clear path forward for EOS-448. Inspired by the multifaceted mechanism of action of EOS-448 and promising early results in clinical trials, this collaboration allows us to accelerate and expand the clinical development of EOS-448. We are more confident than ever in our ability to succeed. This collaboration validates our science and provides a catalyst for the future of iTeos. The collaboration with GSK will allow our team to continue to develop next-generation immunotherapies starting with inupadenant, our highly differentiated clinical-stage A2A adenosine receptor antagonist, and to drive scientific innovation with our expertise in tumor immunology to build our pipeline,” said Michel Detheux, president and CEO, iTeos, in the press release.

GlaxoSmithKline and iTeos Therapeutics enter into a development and commercialization collaboration worth up to $2.08 billion for a new therapeutic anti-cancer mAb candidate.

GSK and iTeos Therapeutics Enter mAb Collaboration

Analysis by Public Health England (PHE) has shown that two doses of COVID-19 vaccines are highly effective against hospitalization as a result of infection with the Delta variant of COVID-19.

The real-world data, which were announced in a June 14, 2021 press release, have shown that two doses of the Pfizer-BioNTech vaccine are 96% effective at preventing hospitalization and two doses of the Oxford-AstraZeneca vaccine are 92% effective against hospitalization due to the Delta variant of COVID-19. These results are comparable with the efficacy found against hospitalization from the Alpha variant of the virus.

As of yet, the level of protection offered by the vaccines against mortality from the Delta variant is not known, but further work is underway to establish these data also. However, it is anticipated that the vaccines’ level of protection against mortality will also be high, as has been the case with other variants.

“These hugely important findings confirm that the vaccines offer significant protection against hospitalization from the Delta variant,” said Mary Ramsay, head of Immunisation at PHE, in the press release. “The vaccines are the most important tool we have against COVID-19. Thousands of lives have already been saved because of them.”

Recent analysis has shown that two doses of COVID-19 vaccines are highly effective against hospitalization as a result of infection with the Delta variant of COVID-19.

PHE Analysis Demonstrates Efficacy of COVID Vaccines Against Delta Variant

The UK BioIndustry Association (BIA) has published a report presenting the findings of a survey on the public attitudes toward equal access to medicines for those living with rare diseases, it was announced in a June 17, 2021 press release.

Results of the survey, which was conducted by YouGov, have shown that the public strongly believe that patients living with rare diseases should have equal access to medicines via the National Health Service (NHS) as those living with more common conditions. Additionally, the majority of survey respondents agreed that patients with rare diseases should have access to medicines assured by the NHS on a basis of clinical need, irrespective of cost.

The survey findings follow recent assertions made by the National Institute for Health and Care Excellence (NICE), specifying that there isn’t an appetite among the general public for specific measures to tackle rare disease. BIA’s report, 

Public Attitudes to Rare Diseases: The Case for Equal Access

, recommends that NICE revises its position on rare conditions and medicines access, and that the body consider the value of a rarity modifier when performing health technology assessments.

“As scientific discovery delivers new and extraordinary treatments, it is essential that patients are able to benefit from them and that must include people living with rare diseases. Securing patient access to rare diseases face very specific issues, including small patient populations, relatively poor understanding of the epidemiology of these conditions, a significant diagnostic odyssey and the absence of a ‘standard of care’ to act as a benchmark,” said Steve Bates, CEO of the BIA, in the press release. “This survey once again demonstrates that there is broad public support for measures to ensure access to medicines for rare diseases based on clinical need even if that would entail higher costs. We would therefore encourage NICE and the Department of Health and Social Care to revisit their assumptions on public appetite for rarity-specific measures.”

BIA has published a report presenting the findings of a survey on the public attitudes toward equal access to medicines for those living with rare diseases.

Survey Reveals Attitudes of Public Toward Rare Diseases and Medicines Access

Horiba, a manufacturer of measurement and analytical systems, has joined a consortium headed by the Cell and Gene Therapy Catapult (CGT Catapult) aimed at accelerating the development of process analytical technologies (PAT) for cell and gene therapy manufacturing, Horiba announced in a June 17, 2021 press release.

According to the press release, Horiba’s A-TEEM fluorescence technology, which has shown promise for fast quality control/quality assurance (QC/QA) of complex biological systems, will be assessed by the consortium as a way of reducing batch failures and manufacturing costs. The fluorescence technology powers the company’s Aqualog Industrial QC/QA Analyzer and is a “column-free” spectroscopic technique that measures absorbance, transmittance, and a fluorescence excitation emission matrix simultaneously.

“This consortium offers a unique blend of expertise and capability covering technology, pharmaceutics, therapy, and patient need. Our continued spirit of addressing new scientific challenges with an ever-evolving suite of core technologies comes together perfectly within this consortium, where the power of A-TEEM Molecular Fingerprinting will be truly explored and harnessed,” said Simon FitzGerald, technical manager at Horiba UK, in the press release. “Data and knowledge in context are key to progress—this consortium will deliver both, helping to bring advanced therapies to patients more quickly.”

Matthew Durdy, CEO at CGT Catapult added, in the press release, “The industry needs to make a giant leap in terms of analytical capability and the dynamic use of information to control and improve processes, product, and costs. The coming together of these leaders in the field is a very important first step towards achieving this.”

Horiba has joined a consortium aimed at accelerating the development of process analytical technologies for cell and gene therapy manufacturing

Horiba Joins CGT Catapult Consortium to Accelerate Development of PAT

FDA’s Center for Biologics Evaluation and Research has given approval to Sanofi Pasteur, the vaccines global business unit of Sanofi, for an additional influenza manufacturing facility located at its existing site in Swiftwater, Pa., the company announced in a June 17, 2021 press release. The newly completed facility further expands production and distribution of Sanofi's Fluzone High-Dose Quadrivalent for the upcoming 2021–2022 influenza season in the United States. The company invested $425 million to build the capacity, which will create up to 200 additional manufacturing jobs.

"We are experiencing fast-growing demand for our vaccine in the US and globally, given the 10 years of data demonstrating protection from flu and its related complications," said Elaine O'Hara, head of North America Commercial Operations for Sanofi Pasteur, in the press release. "Our new facility will complement our existing capacities to produce enough high-dose vaccine for all people 65 and older in as many countries as possible this flu season and beyond, supporting the needs of healthcare providers and patients."

The US approved the quadrivalent form of the high-dose vaccine in November 2019 for adults age 65 and older, and it replaces the trivalent form of the vaccine. Outside of the US, the vaccine has earned recommendations for use by the National Advisory Committee on Immunization (NACI) in Canada and a recommendation for priority use in older adults by Germany's Standing Committee on Vaccination (STIKO).

, Sanofi announced that it would invest in a new vaccine manufacturing facility for Fluzone High-Dose Quadrivalent influenza vaccine at its existing site in Toronto, Canada; this facility is expected to become operational in 2026. The company is also building a facility for this vaccine in Val-de-Reuil, France.

Sanofi’s new Pennsylvania facility expands production of Fluzone High-Dose Quadrivalent flu vaccine.

Sanofi Facility for Flu Vaccine Licensed by FDA

Evonik and Stanford University have entered into a three-year sponsored research collaboration to develop a new polymer-based drug delivery system. The new technology can deliver messenger RNA (mRNA) to tissues and organs in a manner that goes beyond the capabilities of lipid nanoparticles (LNPs), Evonik announced on June 10, 2021. Evonik will license and commercialize the polymer-based platform, which complements its existing portfolio of lipid-based drug delivery, including LNPs.

Under the collaboration, Evonik will work together with Stanford University scientists to scale up the synthesis and formulation of the drug delivery platform and further develop the technology for organ-selective delivery based on a non-animal-derived, synthetic degradable polymer. Evonik aims to make this technology good manufacturing practice-compliant for use in clinical-stage developments and, ultimately, for commercial-scale manufacturing. The new polymer-based delivery platform, known as CART (Charge Altering Releasable Transporters), was developed by Professor Robert Waymouth, Professor Paul Wender, and Professor Ronald Levy of Stanford University.

“We are proud to collaborate with Stanford University and combine our innovative power in advanced drug delivery. Through this project we look forward to enabling the next generation of mRNA-based medicine,” said Dr. Thomas Riermeier, head of Evonik’s Health Care business line, in a company press release.

“If we are to harness the full potential of mRNA therapeutics, we will need a toolbox of drug delivery technologies to target an expanded range of tissues and organs. Therefore, it is a great pleasure to collaborate with Stanford University and bring our expertise in advanced drug delivery to commercialize the new platform,” said Stefan Randl, vice-president of Research, Development & Innovation for Evonik Health Care, in the press release.

Evonik and Stanford University have signed a research collaboration to develop and market a new drug delivery platform for mRNA and gene therapy.

Evonik and Stanford University Partner to Develop mRNA Drug Delivery Technology

Yposkesi, a contract development and manufacturing organization (CDMO) specializing in cell and gene therapies, announced on June 14, 2021 the launch of project SKY, which includes the construction of its second commercial cell and gene therapy manufacturing facility on its campus in Corbeil-Essonnes, France. Project SKY is intended to be a new global resource for drug developers of biologics.

To accelerate innovation, SK Pharmteco, a global CDMO, became a new majority equity shareholder in Yposkesi in March 2021. With financial backing from SK Pharmteco, Yposkesi is investing €58 million (US$71 million) in the new 5000 m2 (50,000 ft²) site, which makes it one of the largest advanced therapy medicinal product (ATMP) facilities in Europe, Yposkesi said in a company press release. The new facility is scheduled to be qualified and operational in conformance with European Medicines Agency (EMA) and FDA regulations in 2023. Project SKY will create approximately 80 new jobs by 2023.

The new state-of-the-art facility, designed for European and American good manufacturing practice (GMP) compliance, will double Yposkesi’s production surface to 10,000 m2 (100,000 ft²), equipping the company to meet the shortage in manufacturing capacity for drug developers seeking to advance to clinical trials and commercialize new therapeutic drugs.

The new building will house two additional production lines with several 1000-L scale bioreactors, supporting projects from early process development phases up to large-scale

manufacturing of approved drugs. Construction on the facility began on May 25, 2021 on a site adjacent to Yposkesi’s current 5000-m2 commercial facility. Equipment consisting of several 1000-L bioreactors, chromatography, and purification skids will be installed by 2023. The new facility will also incorporate a centralized warehouse, capacity for media, and solutions and mountings preparation. It will also house drug substance production suites and storage and quality control lab resources.

“Yposkesi is proud and excited to embark full speed on this new phase in our development. SKY is the culmination of our industrial strategy, guaranteeing clients a range of quality services from early clinical drug development through all phases to the manufacture of large-scale commercial batches of ATMPs,” said Alain Lamproye, executive chairman of Yposkesi, in the press release. “This project will also showcase our outstanding technological know-how in bioproduction for gene therapy drugs.”

“Manufacturing is an essential part of any drug developer’s success in delivering its product to patients,” said Morad El Gueddari, production director and head of project SKY, in the press release. “Yposkesi has the world-class skills and expertise, ambition, and drive to be among the global leaders in gene therapy manufacturing. SKY is the key to getting us there.”

Yposkesi is building its second commercial facility for cell and gene therapy manufacturing at its campus on Corbeil-Essonnes, France.

Yposkesi Launches Project SKY for its Second Commercial Bioproduction Site

Optima, a Germany-based special machine manufacturer, announced on June 10, 2021 that its Pharma Division has partnered with the Robert-Bosch-Krankenhaus (RBK), a charitable hospital in Stuttgart, Germany, and the Universitätsklinikum Heidelberg (UKHD) to develop a unit for decentralized, automated production of chimeric antigen receptor T cell (CAR-T) cell therapies under the ProCell for Patient project, a collaboration launched by the three organizations in late 2020.

The ProCell for Patient system prototype is scheduled to be installed at the RBK in the summer of 2022 and is expected to cut the amount of work needed to manufacture CAR-T cell therapies in half, Optima stated in a company press release. The manufacturing system should deliver benefits in terms of quality, cost, and time, according to Optima.

As part of a forerunner project with Charité Hospital in Berlin, Optima Pharma has already taken the first steps toward developing a suitable production unit. Roll-out to additional treatment centers and pharmaceutical contract developers and manufacturers is planned once testing and clinical trials have been completed. Clinics that are involved in decentralized manufacturing can also contribute in the future to optimizing and developing new cell therapies, the company said in its press release.

"The automation of CAR-T cell therapy production is long overdue. Not just for reasons of cost and quality. In the future, it will facilitate the innovation process," said Walter E. Aulitzky, chief physician of the Department of Oncology, Hematology, and Palliative Medicine at the RBK, in the press release.

"With the help of the ProCell for Patient system, it will probably be possible to reduce the amount of work, i.e., the number of hours that qualified staff today are spending on producing CAR-T cells, by at least 50 percent," said Andrea Traube, director, Market Development Pharma, Optima Pharma, in the press release.

The project is funded by the Ministry of Economic Affairs, Labour and Housing Baden-Württemberg as part of the "Forum Health Region Baden-Württemberg" initiative.

Optima has teamed up with the Robert-Bosch-Krankenhaus and Heidelberg University to develop a unit for decentralized, automated production of CAR-T cell therapies.

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