Pharmaceutical Technology Editors

FDA announced on June 25, 2021, the addition of warnings about myocarditis and pericarditis to the fact sheets for healthcare providers administering messenger RNA-based vaccines from Moderna and Pfizer-BioNTech; fact sheets for patients also were revised to provide information about potential reactions and when to seek medical attention.

The Centers for Disease Control and Prevention (CDC) reported on June 23, 2021, that 

more than 1000 cases of myocarditis and pericarditis in patients receiving the mRNA vaccine had been reported to the Vaccine Adverse Event Reporting System

. The cases primarily involved male adolescents and young adults after receiving a second dose of the vaccine. CDC calls the reports to be rare and patients typically recover quickly. More than 300 million doses of mRNA vaccines have been administered.

The vaccine fact sheet revisions were implemented following review of data presented at a meeting of CDC’s Advisory Committee on Immunization Practices. FDA said the agency and CDC will continue to monitor reports, gather information, and assess longer-term outcomes.

Articles

Reports of myocarditis and pericarditis following Pfizer-BioNTech and Moderna vaccinations prompts warnings.

FDA Adds Warning to mRNA Vaccine Fact Sheets

On June 24, 2021, AbbVie and Teneobio, an affiliate of Teneobio, Inc., announced that AbbVie has exercised its exclusive right to acquire TeneoOne, and TNB-383B, a B-cell maturation antigen (BCMA)-targeting immunotherapeutic for the potential treatment of relapsed or refractory multiple myeloma (R/R MM).

AbbVie and TeneoOne had entered a strategic transaction in February 2019 to develop and commercialize TNB-383B, a bispecific antibody that simultaneously targets BCMA and CD3 and is designed to direct the body's own immune system to target and kill BCMA-expressing tumor cells. AbbVie exercised its acquisition right based on an interim analysis of an ongoing Phase I study.

The interim results from the ongoing Phase I study demonstrated an objective response rate of 79%, very good partial response or better of 63%, and complete response of 29% at doses ≥40 mg.

"Since the beginning of this partnership, we have been encouraged by the potential of TNB-383B as a promising new therapy for multiple myeloma, and our analysis of the Phase [I] data to date has allowed us to make this decision with confidence," said Michael Severino, MD, vice-chairman and president, AbbVie, in a company press release. "While other BCMA and CD3 bispecific therapies require weekly administration, the recommended Phase [II] dose of TNB-383B will investigate infrequent dosing of every [three] weeks for intravenous administration, which is an important treatment factor for people living with multiple myeloma."

"Our aim in developing TNB-383B and our T-cell redirecting anti-CD3 platform is to maximize the therapeutic window of a class of therapeutic molecules that have been clinically challenged by dose-limiting toxicities," said Roland Buelow, CEO of Teneobio, Inc., in the press release. "AbbVie recognized the potential of Teneobio's platform and shared our vision to assess its clinical validation. The clinical data support the unique features of TNB-383B and our T-cell redirecting CD3 platform. We are confident that AbbVie is the right partner to rapidly develop TNB-383B with the ultimate goal to bring this potential new therapy to myeloma patients in need."

AbbVie has exercised its right to acquire TeneoOne, along with its lead immunotherapeutic asset for the potential treatment of multiple myeloma.

AbbVie to Acquire TeneoOne and Lead Asset for Multiple Myeloma Treatment

On June 23, 2021, Amgen announced that the Japanese Ministry of Health, Labor and Welfare granted marketing approval for Aimovig (erenumab) to suppress the onset of migraine attacks in adults, making it the first and only approved treatment in Japan to block the calcitonin gene-related peptide receptor (CGRP-R) believed to play a critical role in migraine. The approval also marks the first independent submission and approval for Amgen K.K., a wholly owned affiliate of Amgen Inc., headquartered in Tokyo, the company said in a press release.

Aimovig's approval in Japan is based on results from a Phase II study evaluating the safety and efficacy of the monoclonal antibody in adult Japanese patients with episodic migraine, and a Phase III study evaluating its efficacy and safety in adult Japanese patients with episodic and chronic migraine. In both studies, Aimovig significantly reduced monthly migraine days from baseline over months four, five, and six of the double-blind treatment period. The safety and tolerability of Aimovig was also shown to be consistent with previously available global data.

"Today's approval further strengthens Amgen's commitment to the migraine community, and we continue to look for ways to expand the availability of Aimovig to help more patients," said Murdo Gordon, executive vice-president of Global Commercial Operations at Amgen in the company press release. "We've seen how much Aimovig has already helped many people living with migraine around the world. Having this treatment approved in Japan will enable us to ultimately serve more patients and help them find the right treatment for this disabling, neurological disease."

"We were impressed by the Japanese Phase III study (20170609) that showed patients treated with Aimovig saw a reduction from baseline in their monthly migraine days," said Koichi Hirata, MD, vice-president, Dokkyo Medical University, in the press release. "We believe Aimovig will bring renewed hope to patients by enabling fewer monthly migraine days, and that it will become one of the preferred treatments given its long-term safety and efficacy data."

"Migraine is a serious neurological disease, yet is poorly understood in Japan as a condition that requires proper treatment," said Steve Sugino, general manager, Amgen K.K., in the press release. "We want patients to know they don't need to push through this disease alone. If properly treated with a therapy [such as] Aimovig that has proven efficacy and has an established tolerability profile, they may be able to take on day-to-day tasks and challenges they had previously been forced to give up."

Aimovig (erenumab-aooe) was approved as a preventive treatment for migraine in adults in the United States on May 17, 2018. As of May 2021, the drug has been approved in 71 countries or territories, including the European Union, the United Kingdom, Canada, and Australia, and is approved by many regulatory authorities worldwide.

Amgen’s Aimovig has become the first and only approved treatment in Japan for treating migraine attacks in adults by blocking the CGRP-R.

Amgen’s Aimovig Approved in Japan for Treating Migraine

FDA has granted breakthrough therapy designation for donanemab, Eli Lilly and Company's (Lilly’s) investigational antibody therapy for Alzheimer's disease (AD), Lilly announced on June 24, 2021. The designation is based on clinical evidence for the antibody, which targets a modified form of beta amyloid called N3pG.

A Phase II trial studied the efficacy and safety of donanemab in patients with early, symptomatic AD, and data were presented at the 15th International Conference on Alzheimer's & Parkinson Diseases 2021 as well as published simultaneously in the 

Lilly intends to submit a biologics license application for donanemab under the accelerated approval pathway later this year based on data from this Phase II trial. The company is also conducting a Phase III study to evaluate the safety, tolerability, and efficacy of donanemab.

Breakthrough therapy designation aims to expedite the development and review of drugs that are intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over already available therapies that have received full FDA approval.

FDA has granted breakthrough therapy designation to donanemab, Eli Lilly and Co.’s investigational antibody treatment for Alzheimer’s disease.

Lilly's Investigational Alzheimer's Therapeutic Gets Breakthrough Therapy Designation

SP Scientific Products (SP) launched its SP Hull LyoStar 4.0 R&D and process development freeze dryer is based on a full-scale production freeze dryer to support rapid lyophilization cycle development, optimization, and scale-up. The pilot-scale lyophilizer offers shelf mapping and rapid shelf freezing, as well as a suite of process analytical technology (PAT) tools. In addition, the equipment uses a more eco-friendly refrigerant gas that reduces the carbon footprint involved in lyophilization.

PAT tools used by the equipment include LyoFlux TDLAS sensors for vapor mass flow measurement; AutoM/SMART Freeze Dryer Technology, a primary drying cycle optimization tool; ControLyo Nucleation Technology, which gives pinpoint control of freezing point; a three-dimensional modeling package for computational fluid dynamics and process monitoring; and Tempris wireless sensors for real-time product temperature measurement.

The equipment was designed to both scale up to production and down to R&D as part of SP’s Line of Sight approach across all its products. The LyoStar 4.0 scales up to the SP Hull LyoConstellation line of production lyophilizers and uses the same efficient refrigeration technology. Line of Sight also enables continuous process monitoring and improvement, in line with Pharma 4.0.

SP Scientific Products’ SP Hull LyoStar 4.0 R&D freeze dryer supports rapid freeze-dry cycle development, optimization, and process scale-up.


New Pilot-Scale Lyophilizer Aids Drug Development

Lonza announced on June 23, 2021 that the company has a manufacturing agreement with CellPoint, a clinical-stage biopharmaceutical company, to develop T-cell therapies for treating various cancers. The collaboration will use Lonza’s proprietary Cocoon Platform, an automated, closed patient-scale cell therapy manufacturing platform.

“We look forward to collaborating with CellPoint to accelerate and enable bringing novel cancer immunotherapies to the clinic and patients. The use of the Cocoon Platform, coupled with the array of CellPoints’ therapies and technologies, and Lonza’s manufacturing capabilities, will help to accelerate the path to the clinic and provide a smoother path to commercial approval. This collaboration also aims to illustrate the promise and feasibility of manufacturing autologous immunotherapies at the point-of-care by leveraging Cocoon’s unique platform capabilities,” said Eytan Abraham, head of Personalized Medicine at Lonza, in a 

“CellPoint has selected Lonza’s Cocoon Platform as the next-generation state-of-the-art closed and automated manufacturing device to develop its point-of-care strategy to make CAR-T affordable and more readily accessible. Over the past year, we have been working with Lonza to develop a manufacturing process that will allow a six to seven day vein-to-vein time to treat cancer patients,” said Tol Trimborn, Chief Executive Officer, CellPoint, in the release.

The companies will utilize the Cocoon Platform for clinical point-of-care manufacturing.