Pharmaceutical Technology Editors

Biogen UK has announced, in an April 15, 2021 press release, that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) and the European Commission (EC) have granted marketing authorization for the subcutaneous (SC) formulation of Tysabri (natalizumab).

Both regulatory bodies’ approvals mean that adults patients living with highly active relapsing-remitting multiple sclerosis (MS) across the European Union (EU), in the United Kingdom, and Ireland now have access to natalizumab SC. Data from the DELIVER (Phase I) and REFINE (Phase II) trials were used in the regulatory review processes. In the clinical trials, the efficacy and safety of the SC formulation of natalizumab were found to be generally consistent with the intravenous (IV) formulation.

“People have such varied experiences of living with MS, which is why patient choice is a key factor. This treatment option offers flexibility and minimizes the time spent receiving treatment, while still providing continuity of care through access to MS healthcare teams,” said David Martin, CEO, MS Trust, in the press release.

Similarly, as with the IV formulation, the SC formulation of natalizumab is administered once every four weeks by a healthcare professional in a clinical setting. The nature of the SC route of administration means that the clinical setting for the new formulation of natalizumab can be expanded out beyond clinical centers, allowing for the potential of care closer to home.

“This unprecedented year has put the NHS [National Health Service] under significant resource pressures and created new challenges for those living with long-term conditions, like MS, when accessing vital, life impacting treatments,” added Dr Mihaela Vlaicu, head of Medical Affairs UK and Ireland, Biogen UK and Ireland, in the press release. “As our healthcare and everyday environment evolves, we must continue to provide solutions to address capacity and resource concerns within the health service, whilst addressing patient needs. Reinforced by nearly 15 years of real-world evidence and post marketing experience with natalizumab IV, SC offers a new method of delivery that can help to reduce patient time in a hospital setting and increase convenience in clinical practice.”

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Biogen UK has announced that the UK’s MHRA and the EC have granted marketing authorization for the subcutaneous (SC) formulation of Tysabri (natalizumab).

MHRA and EC Grant Marketing Authorization for Biogen’s MS Treatment

Thermo Fisher Scientific announced on April 15, 2021 that it has acquired PPD, a clinical research services provider, for $17.4 billion.

The acquisition, which is expected to be completed by the end of 2021, will give Thermo Fisher access to PPD’s drug development platform, patient recruitment capabilities, and laboratory services, Thermo Fisher said in a company press release. The collaboration is expected to offer new solutions for customers, while reducing the time and cost of the drug development process.

"Pharma and biotech is our largest and fastest growing end market, and our customers value us as a strategic partner and an industry leader. The acquisition of PPD is a natural extension for Thermo Fisher and will enable us to provide these customers with important clinical research services and partner with them in new and exciting ways as they move a scientific idea to an approved medicine quickly, reliably, and cost effectively," said Marc N. Casper, chairman, president, and CEO, Thermo Fisher Scientific, in the press release. "Longer term, we plan to continue to invest in and connect the capabilities across the combined company to further help our customers accelerate innovation and drive productivity, while driving further value for our shareholders."

"This is a very exciting announcement for our shareholders and will provide customers with an even better opportunity to bring meaningful innovation to the market faster and more efficiently,” added David Simmons, chairman and CEO, PPD, in the press release. “Thermo Fisher is a world-class company with a very similar culture and values and will provide a great foundation for our colleagues to continue to deliver for our customers and to develop their own skills and careers."

The acquisition, which is expected to be completed by the end of 2021, will give Thermo Fisher access to PPD’s drug development platform, patient recruitment capabilities, and laboratory services.

Thermo Fisher to Acquire PPD for $17.4 Billion

Abzena, a US-based global partner research organization, announced on April 15, 2021 that it has chosen Sanford, NC, as the location for its new good manufacturing practices (GMP) facility.

The new facility, which will be designed specifically for phase III and commercial manufacturing of biologics, will initially include four modular suites with up to two 2000-L bioreactors, followed by the addition of two more 2000 L suites, Abzena said in a company press release. The facility will also handle continuous manufacturing and perfusion, with manufacturing beginning in 2022.

“We are delighted to announce the investment of our newest site in Sanford, NC,” said Jonathan Goldman, MD, CEO of Abzena, in the press release. “Our goal is to support our partners and the patients they serve with this expansion in [C]GMP [current good manufacturing practice] biologics manufacturing services. We are pleased to provide integrated asset development with a full suite of in-house services from discovery through clinical and commercial phases without the need for technology transfer. We are very pleased to be welcomed by North Carolina.”

The new Abzena facility will initially include four modular suites with up to two 2000-L bioreactors, followed by the addition of two more 2000 L suites.

Abzena Names North Carolina Location for New GMP Manufacturing Facility

Catalent announced on April 15, 2021 that it has expanded its services at its clinical supply services facility in Philadelphia to include cryogenic capabilities for the support of cell and gene therapy development.

With the added cryogenic storage, cell therapies and other biological materials will be preserved in liquid nitrogen vapor at temperatures of around minus -180°C, Catalent said in a company press release. Additionally, the facility will have the ability to package, label, and distribute cryogenic materials.

“Establishing robust clinical supply chain services for cell and gene therapies is complex and challenging, and Catalent has undertaken an in-depth strategic review to evaluate how it can establish a safe, efficient, and flexible approach to support this fast-growing area of the industry,” said Ricci Whitlow, president, Catalent Clinical Supply Services, in the press release. “The solution we have implemented at Philadelphia not only meets current needs, but also provides a template for us to easily replicate at other facilities in our global network, allowing incremental capacity expansion within the new infrastructure as demand grows.”

Catalent plans to add cryogenic capabilities to its facility in Philadelphia, which will strengthen its ability to cryogenically preserve cell therapies and other biological materials.

Catalent to Add Cryogenic Capabilities to Support Cell and Gene Therapy Development

Hyalo Technologies, a US-based biopharmaceutical company has unveiled a new patented technology, called Sterilization with the Hyalo Matrix. This new method adds a matrix to enhance stability, allow for sterilization without degradation, and allow maintenance of drug efficacy at room temperature. The ability to remain stable at room temperature will ease up global distribution and reduce dependency on cold chain distribution of pharmaceuticals and biopharmaceuticals, including genetic materials, attenuated viruses, viral vectors, biologics, antibodies, biosimilars, protein subunits, nanosuspensions, and small and large molecules, the company reported in a March 29, 2021 press release.

Furthermore, when added to the drug molecule, the matrix can also effectively be sterilized, preventing degradation and preserving the integrity of temperature-sensitive materials.

The company's latest studies yielded positive results, which showed that the Hyalo Matrix is able to protect and enhance the stability of various biologics, antibodies, and biosimilars. After the matrix was added to each of these types of drug molecules and sterilized, the drugs were analyzed, with ≥ 95% of the drug molecules recovered. These results show the Hyalo Matrix helps preserve efficacy and enhance stability of each drug molecule.

Currently under the COVID-19 pandemic, vaccine manufacturers have been having issues with scaling up manufacturing as well as delayed distribution, bringing cold chain logistics into the spotlight. Hyalo Technologies reported in its press release that the current stats for cold chain logistics are as follows:

Roughly 80% of drugs require temperature-controlled transportation, and that trend is on the rise.

Cold chain requires close consideration of factors, including shipping modes, climate zones, storage capabilities, and healthcare settings.

Pharmaceutical manufacturers, distributors, logistics providers, and others risk the loss of temperature-sensitive products and, therefore, the loss of billions of dollars as evidenced by following:

30% of discarded pharmaceuticals can be attributed solely to cold chain logistics issues

25% of vaccines reach their destination degraded because of incorrect storage and transport during shipping

20% of temperature-sensitive products are damaged during transport because the cold chain has been broken at some point in the process.

"This new technology could eradicate the need for cold chain storage and transport as a drug can now be stable at room temperature, which will make global distribution quicker and more eficient," said Shalabh Jain, chairman and CEO of Hyalo Technologies, in the press release.

"This game-changing technology has the potential to enhance drug delivery and distribution," said Bob Oliver, executive advisor and board member of Hyalo Technologies, in the press release.

This new technology could eliminate the need for cold chain storage, transport, and distribution of temperature-sensitive molecules.

Hyalo Technologies Unveils Room Temperature Drug-Stabilizing Technology

On April 14, 2021, ATMPS, a UK-based provider of blockchain-based “vein-to-vein” cell orchestration platforms for advanced therapies, and Ori Biotech, an innovator in cell and gene therapy (CGT) manufacturing platforms, announced that they have formed a global non-exclusive collaboration to create an integrated digital data platform to support the development of advanced therapies.

Under the agreement, the partnership will integrate Ori’s proprietary CGT manufacturing platform’s data architecture with ATMPS’ Hataali, a secure data sharing platform that is specially designed to be a scalable system for advanced therapies. The combination of the two proprietary platforms is expected to provide improved product tracking, scheduling, and live monitoring of the manufacturing status across the entire CGT supply chain from vein-to-vein.

Hataali works by encrypting and storing data using distributed ledger technology through a process known as hashing. This process ensures data security is protected through a system of private keys and cryptography. The data platform was the first cell orchestration platform installed in a hospital globally, and ATMPS was the first blockchain company to integrate with the UK National Health Service for the delivery of advanced therapies, the company stated in a press release.

“Ori selected ATMPS’ Hataali technology for integration with our platform to allow the secure sharing of relevant process data with therapy developers, contract development and manufacturing organizations, and even clinicians or patients in real-time. This collaboration will provide increased visibility into the treatment process for those involved at each stage of the treatment process,” said Jason C. Foster, CEO, Ori Biotech, in the press release. “The addition of established technologies from companies like ATMPS allows Ori to further its mission of enabling partners across the CGT industry to bring therapies as safely, cost effectively, and quickly as possible to large numbers of patients with few alternative therapeutic options.”

“Ori is an ideal partner for ATMPS, as both companies share a common mission to lower the costs and improve processes for cell and gene therapies. Ori’s work in helping innovators scale from preclinical to commercial is a massive step towards reducing complexity and bringing these vital therapies to patients more quickly,” said Raja Sharif, CEO, ATMPS, in the press release. “We all know how challenging and expensive the development and manufacturing of CGTs can be. With the help of Hataali, Ori can securely leverage vein-to-vein data to advance their manufacturing solutions much more efficiently. This will enable real-time therapy tracking, improved patient trust, and expediated clinical development.”

The ATMPS and Ori Biotech collaboration aims to improve visibility and reporting of cell and gene therapy manufacturing through a pre-integrated data solution.