Pharmaceutical Technology Editors

FDA announced in a Nov. 19, 2021, press release that it has officially amended its Emergency Use Authorization (EUA) for both Pfizer-BioNTech’s and Moderna’s COVID-19 single-shot booster vaccinations to now include all adults. To qualify, individuals must be 18 or older and be at least six months removed from completion of their primary vaccination series of the Pfizer-BioNTech or Moderna shots. If they received the Janssen single-shot vaccine, this limit is reduced to two months.

Prior to this announcement, booster vaccinations were only made available to individuals aged 65 or older, individuals aged 18 through 64 at high risk of severe COVID-19, and individuals aged 18 through 64 with frequent institutional or occupational exposure to SARS-CoV-2.

“Throughout the course of the COVID-19 pandemic, the FDA has worked to make timely public health decisions as the pandemic evolves. COVID-19 vaccines have proven to be the best and highly effective defense against COVID-19,” said Janet Woodcock, acting FDA commissioner, in an agency press release. “Authorizing the use of a single booster dose of either the Moderna or Pfizer-BioNTech COVID-19 vaccine for individuals 18 years of age and older helps to provide continued protection against COVID-19, including the serious consequences that can occur, such as hospitalization and death.”

According to the release, this decision was made following an evaluation of immunity data from clinical trials of both the Pfizer-BioNTech and Moderna vaccines. The Pfizer-BioNTech trial evaluated 200 participants aged 18 through 55 who received a single booster dose at least six months after their second dose relative to peers who only completed the two-dose series, while the Moderna trial did a similar evaluation of 149 individuals aged 18 or older. Both studies found an increased immune response in the booster group one month after vaccination.

“The FDA has determined that the currently available data support expanding the eligibility of a single booster dose of the Moderna and Pfizer-BioNTech COVID-19 vaccines to individuals 18 years of age and older,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, in the press release. “Streamlining the eligibility criteria and making booster doses available to all individuals 18 years of age and older will also help to eliminate confusion about who may receive a booster dose and ensure booster doses are available to all who may need one.”

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FDA has amended its EUA for Pfizer-BioNTech’s and Moderna’s COVID-19 booster to include all individuals aged 18 or older.

FDA Authorizes COVID-19 Boosters for All Adults

Novo Nordisk announced that it would acquire Dicerna, a biopharmaceutical company focusing on ribonucleic acid interference (RNAi) technology, for approximately $3.3 billion on Nov. 18, 2021. This blockbuster deal will give Novo Nordisk rights to Dicerna’s GalXC RNAi platform, which is designed to enable access to intracellular disease targets across hepatic and extrahepatic cell and tissue types.

Novo Nordisk and Dicerna entered into a research collaboration in 2019 to develop RNAi therapies via GalXC. The companies explored more than 30 liver cell targets with the potential to deliver clinical candidates for conditions like chronic liver disease, nonalcoholic steatohepatitis, type 2 diabetes, obesity, and various rare diseases. Novo Nordisk expects to initiate clinical development of the first investigational RNAi therapeutic born from this collaboration in 2022.

According to a Dicerna press release, RNAi is a disease inhibition technology that works by silencing genes via specific destruction of the messenger RNA (mRNA) made from the gene. Rather than inhibiting the protein, this is intended to prevent its creation. GalXC technology uses a proprietary N-acetyl-D-galactosamine (GalNAc)-mediated structure of double-stranded RNA molecules designed to bind specifically to receptors on liver cells. This is designed to lead to selective hepatocyte internalization and permit access to the RNAi machinery within the cells.

“The acquisition of Dicerna accelerates Novo Nordisk’s research within RNAi and expands the usage of the RNAi technology,” said Marcus Schindler, executive VP and chief scientific officer of Novo Nordisk, in the press release. “We build on our successful collaboration and by combining Dicerna’s state-of-the-art RNAi drug engine and intracellular delivery with our deep capabilities in disease biology understanding and tissue targeting through peptides and proteins, we have the potential to expand our pipeline and deliver life-changing precision medicines for people living with chronic diseases such as diabetes, obesity, cardiovascular disease and NASH, as well as rare diseases like endocrine disorders and bleeding disorders.”

“Since the start of our collaboration two years ago, the Dicerna and Novo Nordisk teams have established a strong rapport built on a foundation of mutual respect for one another’s capabilities, culture, and expertise,” said Douglas Fambrough, founder, president, and CEO of Dicerna, in the press release. “The combination of Dicerna’s expertise in RNAi and oligonucleotide therapeutics and highly skilled employees with Novo Nordisk’s industry leadership in developing and commercializing medicines to treat serious chronic diseases, has the potential to significantly accelerate and expand our mission to deliver GalXC RNAi therapies for the benefit of patients and all our stakeholders.”

Novo Nordisk’s 3.3 billion acquisition of Dicerna gives them access to their RNAi technology.

Novo Nordisk Acquires Dicerna for $3.3 Billion

Secarna Pharmaceuticals, a Germany-based biopharmaceutical company focused on next-generation antisense oligonucleotide (ASO) therapies, announced on Nov. 10, 2021 that it has entered into a research, option, and license agreement with Achilles Therapeutics, a member of the Achilles Therapeutics plc group of companies, to optimize the development of T cell therapies in Achilles’ pipeline.

Under the agreement, Secarna will receive an undisclosed upfront payment, a target-based technology access fee, and option exercise fees. The company is also eligible to receive development and commercial milestone payments and tiered royalties on net sales of personalized T cell therapies developed and commercialized under the collaboration.

In the collaboration, Secarna will apply its discovery and development platform, LNAplus, to generate ASO candidates against selected targets. These targets potentially play a central role in the 

 optimization of personalized T cell therapies under development by Achilles. Secarna will also perform 

 testing, after which Achilles will conduct production feasibility and optimization testing.

“We are excited to collaborate with Achilles and jointly work on a highly innovative approach to optimize T cell therapies with our LNAplus-based ASOs,” said Alexander Gebauer, CEO of Secarna Pharmaceuticals, in a company press release. “By combining our industry-leading antisense oligonucleotide platform with Achilles’ expertise in the field of T cell therapies, we see the potential to break new ground in the treatment of cancer patients.”

Secarna Pharmaceuticals and Achilles Therapeutics have entered into an agreement to optimize the development of T cell therapies in Achilles’ pipeline.

Secarna Pharmaceuticals and Achilles Therapeutics Partner on T Cell Therapy Development

The Cell and Gene Therapy Catapult (CGT Catapult) and Deep Science Ventures, a London-based venture creator, have collaboratively launched a partnership to generate innovative approaches that address barriers in the advanced therapy medicinal products (ATMP) industry. The partnership’s goals are to create new companies and deliver better patient outcomes, CGT Catapult announced in a Nov. 11, 2021 press release.

Under the agreement, CGT Catapult and Deep Science Ventures will spend the next 12 months identifying an area that is ripe for innovation, hire a founding analyst to evaluate potential approaches for scientific and commercial viability, build teams around optimal approaches, and create one or more high-impact ventures. Furthermore, in addition to improving patient outcomes, the partners aim to ultimately increase job opportunities and innovation in the ATMP sector.

Major hurdles for ATMP development include the challenges of ensuring target specificity and achieving effective manufacturing at-scale. Under this collaboration Deep Science Ventures will use its outcome-focused approach to venture creation, in which it will combine available scientific knowledge with founder-type scientists into high-impact venture companies. CGT Catapult will provide its cell and gene therapy-specific technical, non-clinical, and regulatory expertise to apply Deep Science Ventures’ approach to the ATMP sector.

“Joining forces with the CGT Catapult enables us to bring our unique approach to innovation to the cell and gene therapy ecosystem. Bringing together [Deep Science Ventures’] structured approach to venture creation and the ability to tap into the [CGT] Catapult’s network of expertise will allow us to create high-impact solutions overcoming some of the barriers holding back the ATMP space,” said Kerstin Papenfuss, associate director Pharma, Deep Science Ventures in a company press release.

“The innovative and collaborative concept used by [Deep Science Ventures] fits well with the CGT Catapult’s ethos, and, by addressing challenges within the cell and gene therapy industry, our activities have the potential to provide exceptional outcomes in driving innovation forward and creating opportunities in employment. We very much look forward to initiating our work together and creating new partnerships between scientists and investors,” said Matthew Durdy, CEO of CGT Catapult, in the press release.

CGT Catapult and Deep Science Ventures have partnered to encourage innovative approaches to overcoming barriers in delivering advance therapies to patients.

CGT Catapult and Deep Science Ventures Team Up to Boost Innovation in Advanced Therapies

GlaxoSmithKline (GSK) has been granted approval by the European Commission (EC) for its monoclonal antibody therapy, Nucala (mepolizumab), which targets interleukin-5 (IL-5) as a treatment for three additional eosinophil-driven diseases.

According to a Nov. 17, 2021 press release, the approval follows on from the positive opinion granted by the Committee for Medicinal Products for Human Use (CHMP) and means that Nucala can be used as an add on treatment in hypereosinophilic syndrome, eosinophilic granulomatosis with polyangiitis, and chronic rhinosinusitis with nasal polyps. The commission’s decision has been based on data from pivotal trials that have investigated the role of targeted IL-5 inhibition with mepolizumab in the three aforementioned eosinophil-driven diseases.

“With millions of patients across Europe affected by eosinophil-driven diseases, we recognize the urgency in delivering the first approved targeted treatment for use in four of these conditions,” said Hal Barron, chief scientific officer and president R&D, GSK, in the press release. “Today’s approvals reinforce the important role treatments such as mepolizumab can play in helping to improve the lives of patients with these debilitating diseases.”

“The lives of patients affected by an eosinophil-driven disease are often impacted by what can be severe or life-threatening symptoms. They may rely on both intermittent or continuous oral steroids to manage their condition or be left feeling they have no option but to endure ongoing symptoms and possible flare-ups,” added Tonya Winders, CEO & president, Allergy and Asthma Network and president of Global Allergy and Airways Patient Platform, in the press release. “The availability of mepolizumab, a targeted biologic therapy, provides patients and their healthcare professionals with a new option in their armamentarium to treat hypereosinophilic syndrome, eosinophilic granulomatosis with polyangiitis, and chronic rhinosinusitis with nasal polyps.”

GSK has been granted approval by the EC for its monoclonal antibody therapy, Nucala (mepolizumab), as a treatment for three additional eosinophil-driven diseases.

GSK Receives European Approval for Three Additional Indications of Nucala

AbbVie announced on Nov. 17, 2021 that the European Commission (EC) has approved SKYRIZI (risankizumab) for the treatment of active psoriatic arthritis in adults, which is the therapy’s second indication in the European Union.

The latest approval covers the use of the therapy either alone or in combination with methotrexate to treat adults with active psoriatic arthritis who have had an inadequate response or have been intolerant to one or more disease-modifying anti-rheumatic drugs. The treatment regimen approved for the second indication is 150 mg of risankizumab to be administered via subcutaneous injection at week 0, week 4, and every 12 weeks thereafter.

"People living with psoriatic arthritis struggle with psoriatic lesions and joint inflammation that causes swelling and pain. Reducing these symptoms may give people the ability to resume their daily activities and improve their quality of life," said Michael Severino, vice chairman and president, AbbVie, in a company press release. "We are excited by the EC approval of SKYRIZI for the treatment of adults with active psoriatic arthritis."

The approval for the second indication was supported by data from two Phase III studies, in which a statistical significance was achieved for the primary endpoint for efficacy and multiple secondary endpoints. Additionally, the safety profile of the therapy was consistent in psoriatic arthritis as was seen with plaque psoriasis (the initial indication for the therapy).

"Millions of people living with psoriatic arthritis are impacted by psoriatic lesions, joint pain, stiffness and fatigue," added Lars Erik Kristensen, consultant and head of science at the Parker Institute in Copenhagen Denmark, associate professor, Lund Sweden, SUS University Hospital. "As seen in this Phase [III] clinical trial program in psoriatic arthritis, SKYRIZI has the potential to be a valuable new treatment option, helping to improve the signs and symptoms of the disease."

SKYRIZI forms part of a collaboration between Boehringer Ingelheim and AbbVie, where AbbVie is responsible for leading global development and commercialization.

AbbVie's SKYRIZI (risankizumab) has gained approval for the treatment of active psoriatic arthritis in adults, which is the therapy’s second indication in the European Union.