Pharmaceutical Technology Editors

The FDA amended its emergency use authorization (EUA) for the Pfizer-BioNTech COVID-19 vaccine on Sept. 23, 2021. The new guidelines allow for a single booster dose to be given to:

Individuals 18 through 64 years of age at high risk of severe COVID-19;

Individuals 18 through 64 years of age whose frequent institutional or occupational exposure to SARS-CoV-2 puts them at high risk of serious complications of COVID-19, including severe COVID-19.

While there has been a heated battle about the need for booster doses for COVID-19, this is the first vaccine to officially be granted booster status in the US. While many argued that boosters would be needed very soon, others, such as 

World Health Organization’s Director-General Tedros Adhanom Ghebreyesus

former FDA scientists Marion Gruber and Phillip Krause

, felt the global community might be moving too fast.

This decision was made following an analysis of several key data points concerning the long-term efficacy of the vaccine, numbers of breakthrough cases, and the emergence of the Delta variant. Pfizer’s own original clinical trial, which 

 days before this announcement, was among the data sets that led to the EUA amendment.

“Today’s action demonstrates that science and the currently available data continue to guide the FDA’s decision-making for COVID-19 vaccines during this pandemic,” said Acting FDA Commissioner Janet Woodcock in a FDA press release. “After considering the totality of the available scientific evidence and the deliberations of our advisory committee of independent, external experts, the FDA amended the EUA for the Pfizer-BioNTech COVID-19 Vaccine to allow for a booster dose in certain populations such as health care workers, teachers and day care staff, grocery workers, and those in homeless shelters or prisons, among others.”

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The FDA has amended the emergency use authorization for the Pfizer-BioNTech COVID-19 vaccine to allow a single booster dose to several high-risk populations.

FDA Authorizes Booster Dose of Pfizer-BioNTech COVID-19 Vaccine for Certain Populations

Johnson & Johnson (J&J) announced new data that reinforced the long-lasting protection of the company’s COVID-19 vaccine in a Sept. 21, 2021, press release. Moreover, this data showed increased protection against COVID-19 following a booster shot of the Johnson & Johnson vaccine. The safety profile of the vaccine remained consistent and was generally well-tolerated when administered as a booster.

The real-world evidence study for a COVID-19 vaccine demonstrated stable vaccine effectiveness of 79% for COVID-19-related infections and 81% for COVID-19-related hospitalizations. There was no evidence of reduced effectiveness over the study duration, including when the Delta variant became dominant in the US.

Included in the study were 390,000 people who received the J&J COVID-19 vaccine versus approximately 1.52 million unvaccinated people, who were matched on age, sex, time, three-digit zip code, and comorbidities and predictors for COVID-19 infection severity. The study was conducted from March to late July 2021. The data was consistent with the Phase III ENSEMBLE trial.

In the Phase III ENSEMBLE 2 study, another J&J COVID-19 vaccine (booster) given at 56 days following the first shot provided:

100% protection against severe/critical COVID-19 (at least 14 days post-final vaccination)

75% protection against symptomatic (moderate to severe/critical) COVID-19 globally and 94% in the United States

Additionally, when a J&J booster was given two months after the first shot, antibody levels were four to six times higher than observed after the single shot.

“Our large real-world evidence and Phase III studies confirm that the single-shot Johnson & Johnson vaccine provides strong and long-lasting protection against COVID-19-related hospitalizations. Additionally, our Phase III trial data further confirm protection against COVID-19-related death,” said Mathai Mammen, global head, Janssen research and development, J&J, in the press release. “Our single-shot vaccine generates strong immune responses and long-lasting immune memory. And, when a booster of the Johnson & Johnson COVID-19 vaccine is given, the strength of protection against COVID-19 further increases.”

“A single-shot COVID-19 vaccine that is easy to use, distribute and administer, and that provides strong and long-lasting protection is crucial to vaccinating the global population,” said Paul Stoffels, vice chairman of the executive committee and chief scientific officer, J&J, in the press release. “At the same time, we now have generated evidence that a booster shot further increases protection against COVID-19 and is expected to extend the duration of protection significantly.”

New data finds J&J COVID-19 vaccine and booster to provide increased protection against COVID-19. 

Real-World Evidence Shows Strong Protection from COVID-19 with J&J Vaccine and Booster

Two clinical-stage biotechnology companies, Celularity and Oncternal Therapeutics, announced on Sept. 20, 2021, that they would begin a research collaboration to evaluate placental-derived cellular therapies targeting receptor-tyrosine kinase-like Orphan Receptor 1 (ROR1). ROR1 is highly expressed in many solid tumors and hematological malignancies, conferring both an aggressive phenotype and survival advantage to tumor cells.

In the collaboration, Celularity will explore the use of Oncternal’s cirmtuzumab, a ROR1-targeted monoclonal antibody, in combination with Celularity’s natural killer cells, CYNK-101. They will also explore ROR1-targeted chimeric antigen receptor gene modification in both the natural killer cell and CyCART T cell platforms.

Cirmtuzumab binding to ROR1 (for both leukemia and lymphoma cells) decreases tumor cell proliferation and survival by blocking Wnt5a-induced activation, according to a Celularity press release. CYNK-101 is a placental-derived allogeneic cell therapy that has been engineered to synergize with therapeutic antibodies such as cirmtuzumab.

“We believe that targeted cellular therapies have the potential to extend the clinical benefit of our research and improve the standard of care for patients,” said James Breitmeyer, founder, president, and CEO of Oncternal, in a press release. “However, the current limitations in efficacy, safety, and availability of cellular therapies hinders their broader use. Celularity’s approach, leveraging the ability of placental-derived cells to differentiate and expand, has the potential to overcome these obstacles and could potentially offer more potent, tolerable, and accessible cellular medicines and, in combination with our ROR1 targeting antibodies, address the significant unmet needs of patients.”

“We are thrilled to enter this partnership with Oncternal to forge new therapeutic strategies for both solid tumors and hematological malignancies using our allogeneic placental-derived cell therapy product candidates with their innate stemness,” said Robert J. Hariri, founder, chairperson, and CEO of Celularity, in a press release. “Oncternal’s work has established ROR1 as an exciting target that could be utilized for the development of new and novel cellular medicines, and there is an immense potential for synergy combining two novel approaches to create exciting new pipeline candidates targeting a wide range of cancers.”

Celularity and Oncternal’s collaboration will focus on therapeutics targeting Orphan Receptor 1, which is linked to aggression and survival in solid tumor cells.

Celularity and Oncternal Therapeutics to Research Placental-Derived Cellular Therapies

Lonza announced on Sept. 22, 2021, that they will invest in additional drug manufacturing capabilities at their Stein site in Switzerland. The investment will fund an additional aseptic flexible filling line for clinical supply of drug product.

The new line will support liquid and lyophilized vial filling and processing as well as cartridges and pre-filled syringes. This line will allow Lonza to process various modalities, including monoclonal antibodies, bioconjugates, viral vectors, and various other gene therapy products. According to a company press release, the filling line will implement state-of-the-art robotic technology and implement innovative freeze-drying technology with an air-cooling system. Expected to be completed in 2023, the installation will create more than 70 new positions at the Stein site.

In tandem with the global drug product capacity increase, Lonza will extend its drug product services footprint for parenteral dosage forms at its Basel site. This will increase process development and characterization capabilities offered at the site, supporting both early and late-stage clinical stage pharmaceutical development. It’s expected to be operational in 2024.

"The drug product capability expansions in Switzerland provide our customers with access to a flexible, integrated offering comprising the development, testing and manufacturing services for mammalian, microbial, bioconjugate products, cell and gene therapies, and small molecules,” said Jean-Christophe Hyvert, president of biologics and cell and gene, Lonza, in a press release. “Enhancing our global drug product capacity underlines Lonza's continuing commitment to providing our customers with an end-to-end solution across various modalities and scales."

"Our drug product services offering in Basel is a world-class capability and represents a center of excellence for Lonza's global drug product network,” said Peter Droc, head of drug product services at Lonza, in a press release. “Alongside the investment in the filling line in Stein, expanding the footprint and capabilities of our drug product services allows us to answer customer demand for integrated solutions."

Lonza’s investment in its Stein and Basel facilities in Switzerland aims to expand its overall drug product development and manufacturing services.

Lonza to Expand Capabilities at Manufacturing Sites in Switzerland

Novartis announced that it had acquired Arctos Medical on Sept. 21, 2021, giving them access to Arctos’ preclinical optogenetics-based adeno-associated viral vector gene therapy program and proprietary technology for its ophthalmology portfolio. Novartis hopes to use the technology gained in this acquisition to develop gene therapies for patients with vision loss.

Acrtos’ technology was developed as a method for treating conditions that involve photoreceptor loss, such as inherited retinal dystrophies (IRDs) or age-related macular degeneration. Because it does not target a specific gene, it could potentially address many forms of IRD independent of the underlying mutation. This is significant because, according to a Novartis press release, there are over 100 genes whose mutations could lead to IRDs.

The technology is designed to deliver proprietary, light-sensitive optogenes to specific retinal cells using gene therapy. This turns the targeted cells into replacement photoreceptor-like cells. If successful, a therapy derived from the technology could be used to treat diseases that cause blindness as a result of photoreceptor death.

“Optogenetics is emerging as a promising therapeutic approach that might restore sight to patients who are legally blind,” said Jay Bradner, president of the Novartis Institutes for BioMedical Research, in a company press release. “The Arctos technology builds on our conviction that optogenetic gene therapies may meaningfully help patients battling devastating eye diseases.”

“We’ve watched this technology develop and mature into a therapeutic program that complements our existing portfolio and gives us new optogenetics technology to wield in our efforts to bring desperately needed therapeutic options to patients for these blinding diseases,” said Cynthia Grosskreutz, global head of ophthalmology at the Novartis Institutes for BioMedical Research, in the press release.

Novartis’ acquisition of Arctos Medical could lead to significant advancements in optogenetic therapies for patients with vision loss.

Novartis Acquires Arctos Medical, Expanding Optogenetics Profile

Metrion Biosciences, a contract research and drug discovery company, announced on Sept. 22, 2021, that it was relocating to a new, custom-designed headquarters at Grant Park in Cambridge. This move was done as a means of significantly expanding its range of services and cell lines.

The 12,000-ft² facility more than doubled Metrion’s previous laboratory space. The new laboratories have significant additional capacity for client projects, with dedicated space for 384-well format automated electrophysiology and enhanced liquid handling. It was specifically designed to support the addition of good laboratory practice compliant cardiac safety services and expanded cell biology facilities.

“Our new headquarters has been specifically designed to support Metrion’s ambitious business plan for global growth, with a particular focus on the USA in 2022. Following the recent appointment of Nick Foster as Chief Commercial Officer, the additional space will also allow for further growth of Metrion’s team of expert laboratory scientists,” said Andrew Southan, chief executive of Metrion Biosciences, in a company press release. “Metrion continues to build its reputation as a leading provider of drug discovery research services for ion channel targets across multiple therapeutic areas and I look forward to working with the team during this next phase of growth for our company.”

Metrion’s new 12,000 ft² headquarters will significantly expand its range of services and cell lines.