Full-day virtual event exploring the latest developments in formulation, manufacturing, innovative drug dosage forms and other crucial topics impacting the industry

2020 Bio/Pharma Virtual Congress: November 11, 2020

Remdesivir approved; Vaccine development agreements

COVID-19 Therapy Developments

More complex and challenging compounds require a tailored formulation approach.

Unique Molecules Push Formulation Boundaries

on Oct. 30, 2020 that it has identified a list of essential medicines, medical countermeasures, and critical inputs that should always be available in adequate amounts to protect against outbreaks of infectious diseases. The list was developed in response to an executive order issued in August 2020 to “ensure sufficient and reliable, long-term domestic production of these products, and to minimize potential shortages by reducing our dependence on foreign manufacturers of these products,” according to FDA.

The list, which was created in consultation with other federal FDA partners, includes 223 essential medicines and medical countermeasures and 96 device medical countermeasures. FDA identified essential medicines most needed for patients in acute care medical facilities. “The medical countermeasures we identified are FDA-regulated products (biologics, drugs, and devices) that meet the definition of a ‘medical countermeasure’ provided in the executive order and that we anticipate will be needed to respond to future pandemics, epidemics, and chemical, biological, and radiological/nuclear threats. When identifying essential medicines and medical countermeasures, we focused on including those that are medically necessary to have available in adequate supply which can be used for the widest populations to have the greatest potential impact on public health,” the agency stated in a press release. The agency identified APIs of essential medicines and medical countermeasures as critical inputs, “as well as ingredients or components that possess unique attributes essential in assessing the safety and effectiveness of such products.”

The agency will also coordinate with federal partners to develop strategies for acquiring products on the list, accelerate domestic manufacturing of these products, and investigate supply chain vulnerabilities. “The FDA believes that the adoption of innovative technologies, such as advanced manufacturing techniques could enable US-based pharmaceutical manufacturing to bolster its competitiveness with foreign countries and help ensure a stable supply of drugs critical to the health of US patients. As companies look to increase domestic manufacturing of the products on this list, we encourage them to consider adopting advanced manufacturing technologies,” FDA stated in the 

Articles

The agency, in response to an executive order, published a list of essential medicines, medical countermeasures, and critical inputs that should be available at all times in an adequate amount to serve patient needs.

FDA Identifies Essential Medicines

on Oct. 28, 2020 that researchers at the Center of Drug Evaluation and Research (CDER) have studied factors that might predict the possible submission of an abbreviated new drug application (ANDA). CDER researchers used proprietary data related to ANDAs, internal or public information, and information on drug sales to see if a drug’s characteristics, regulatory history, or market sales could predict the submission of an ANDA. The study separately analyzed drugs that were originally approved as new chemical entities (NCEs) and those that did not have NCE exclusivity.

The study found that ANDAs were more likely to occur in NCEs and non-NCEs that had higher revenues. “By contrast, designation of a brand-name product as a complex drug (for example, due to its structure, composition, or its mode of delivery) was negatively associated with ANDA submission for both NCEs and non-NCE drugs,” FDA stated in a press release. Challenges associated with complex drug substances or drug products may be a factor, according to the researchers.

Data showed that the availability of a published product-specific guidance (PSG) for non-NCE drugs was a positive factor in ANDA submissions. “Non-NCE drugs for which there was a PSG were approximately 3.8 times more likely to result in an ANDA submission than non-NCE drug products for which no PSG had been published (data not shown),” FDA stated. The study also found that patents did not have a significant impact on ANDA submissions. “…in the case of large-revenue drugs, that sponsors will challenge the patent even when they are unlikely to win, meaning that higher sales are associated with more pre-generic approval patent challenges (commonly referred to as ‘Paragraph IV’ challenges).”

The Center for Drug Evaluation and Research is studying factors that influence the development of generic versions of brand-name drugs.

Predicting Generic Drug Marketing Applications

on Oct. 30, 2020 has published clinical data supporting the marketing authorization application for Veklury (remdesivir) and a list of COVID-19 medicines and vaccines that have received scientific advice or informal guidance from the agency’s pandemic Task Force. The agency is releasing this information as part of its dedication to transparency of its activities in relation to COVID-19 vaccines and treatments.

There are 64 documents available on EMA’s clinical data website that give a clinical overview, summaries, and the final reports from pharmacokinetic and Phase I clinical studies. Interim study reports from Phase III clinical studies, clinical data from the compassionate use program, and the data anonymization report are also available.

“The publication of clinical data for Veklury is in line with EMA’s landmark policy to proactively publish clinical data supporting marketing authorization applications. The agency had to suspend the publication of clinical data at the end of 2018 as a result of its move from London to Amsterdam. It currently remains suspended due to ongoing business continuity linked to the COVID-19 pandemic and human resource constraints. EMA has decided to exceptionally publish clinical data for COVID-19 medicines given the unprecedented public interest for this information in the context of the ongoing pandemic,” EMA stated in a press release.

Information on the medicines that received scientific advice or guidance includes the stage of development in which the advice was given to help the developer prepare for a marketing authorization application. This advice may include “best methods and study designs for generating robust data on a medicine’s safety and effectiveness. It can also focus on quality aspects, such as manufacturing and testing, or on laboratory studies,” according to EMA.

The agency published clinical data on remdesivir and information about COVID-19 treatments that have received scientific advice or informal guidance from EMA’s pandemic Task Force.

EMA Implements Transparency Measures for COVID-19 Medicines

in October 2020 describing the requirements for electronically submitting standardized clinical and nonclinical study data under section 745A(a) of the Federal Food, Drug, and Cosmetic Act for new drug applications, abbreviated new drug applications, biologics license applications, and investigational new drug applications to the Center for Drug Evaluation and Research or the Center for Biologics Evaluation and Research. “Submissions that are not submitted electronically and electronic submissions that are not in a format that FDA can process, review, and archive will not be filed or received, unless exempt from the electronic submission requirements or if FDA has granted a waiver,” the guidance states.

The guidance discusses the requirements for electronic submission of study data including exchange format standards, study data standards, and controlled terminology standards. It also discusses the agency’s policy on waivers of electronic submission requirements.

FDA published guidance for submitting standardized study data in electronic format.

Guidance on Submitting Standardized Study Data 

The Research Center Pharmaceutical Engineering (RCPE) in Graz, Austria has been awarded FDA funding for two projects, set to begin in the fourth quarter of 2020, that will focus on using advanced technologies to improve pharmaceutical production processes, RCPE announced in an Oct. 29, 2020 press release. RCPE noted that FDA typically only awards five to 10 projects a year to European organizations, which demonstrates that these projects have the potential for high impact within the global pharma industry.

In the first project, RCPE will partner with global pharma companies, including MSD (known as Merck in the US and Canada) and Pfizer, to investigate the use of optical coherence tomography (OCT) for real-time monitoring and control of the drug tablet coating process. Researchers will seek to integrate OCT with artificial intelligence (AI), such as machine learning approaches, to prevent errors in tablet coating before they occur.

In the second project, RCPE will partner with MSD, Pfizer, and other collaborators to continue previous work with digital simulation tools for drug product manufacturing and process development. A digital twin-based platform will model products, processes, and conditions, using these simulations for evaluation, optimization, and scale-up. Replacing extensive lab-based experiments is expected to reduce drug development and manufacturing time and reduce energy consumption and carbon footprint for next-generation pharmaceutical production.

Professor Johannes Khinast, RCPE CEO and scientific director, said in the press release that the awards are an endorsement “of our pioneering approach to solving drug manufacturing challenges. Having achieved this important milestone, we look forward to working with our partners to improve their manufacturing technology, ultimately benefitting patients around the globe.”

Two FDA-funded projects at RCPE will look at real-time monitoring and control of drug tablet coating and developing a digital twin for drug production control.

RCPE Awarded FDA Funding to Develop Next-Generation Technology

On Oct. 27, 2020, the United States filed a civil complaint against Med-Pharmex (MPX), a Pomona, CA-based animal health company, regarding its manufacturing and distribution of adulterated animal drugs.

According to a press release from the Department of Justice, the complaint aims to stop the company from manufacturing and distributing animal drugs in conditions unsatisfactory to the minimum regulatory requirements to ensure safety and quality. In the complaint, it is noted that FDA inspected the company site several times, which resulted in a warning letter in 2017 that noted deficiencies at the company including the failure to properly clean and disinfect areas used to manufacture sterile drugs and the failure to conform to current good manufacturing practices. Additionally, the complaint also mentions MPX’s failure to sufficiently examine reports of the death or illness of animals receiving MPX drugs, the press release said.

“Americans depend on animal drugs being safe and effective,” said Acting Assistant Attorney General Jeffrey Bossert Clark of the Justice Department’s Civil Division, in the press release. “We will continue working with FDA to ensure all drug manufacturers abide by public safety requirements.”

“Ensuring FDA-approved animal medications are safe, effective, and manufactured using current good manufacturing practices is an important part of the FDA’s mission to protect human and animal health,” added FDA Chief Counsel Stacy Amin, in the press release. “The FDA will continue to pursue actions against those who put animal patients in harm’s way by manufacturing and distributing adulterated animal drug products.”

The complaint aims to stop the company from manufacturing and distributing animal drugs in conditions unsatisfactory to the minimum regulatory requirements to ensure safety and quality.

The Department of Justice Files a Complaint Against Animal Health Company for the Distribution of Adulterated Animal Drugs

Takeda and Moderna announced on Oct. 29, 2020 that they are entering into a three-way agreement with the Government of Japan’s Ministry of Health, Labor, and Welfare to import and distribute 50 million doses of Moderna’s COVID-19 vaccine candidate, mRNA-1273, beginning in the first half of 2021.

Through the agreement, Takeda will handle securing the regulatory approvals before distributing the doses, while Moderna will supply the finished product, a Takeda press release said. Currently, a Phase III clinical trial for the vaccine candidate in the United States is fully enrolled.

This collaboration follows Takeda’s recent announcement that it is planning to manufacture Novavax’s COVID-19 vaccine candidate at its facilities in Japan to support the long-term supply of the treatment to the Japanese population.

“Takeda is collaborating with the Japanese Government and vaccine developers to provide rapid and sustained access to COVID-19 vaccines in Japan,” said Rajeev Venkayya, MD, president of the Global Vaccine Business Unit, Takeda, in the press release. “We have chosen to work with Novavax and Moderna, both of which have promising vaccine candidates, and will continue to support the global response to COVID-19 through R&D efforts across Takeda.”

Through the agreement, Takeda will handle securing the regulatory approvals before distributing the doses, while Moderna will supply the finished product.

Takeda, Moderna, and the Government of Japan to Collaborate to Distribute 50 Million COVID-19 Vaccine Doses

AstraZeneca and Daiichi Sankyo announced on Oct. 28, 2020 that their supplemental biologics license application (sBLA) for Enhertu (trastuzumab deruxtecan), an antibody drug conjugate (ADC) for treating HER2-positive metastatic gastric or gastroesophageal junction adenocarcinoma, was accepted by FDA and granted priority review.

FDA’s acceptance of the sBLA was based on positive results from a Phase II trial (Destiny-Gastric01), which showed improvement in objective response rate, the primary endpoint, and overall survival in patients treated with the therapeutic, according to an AstraZeneca press release.

“Once patients with HER2-positive metastatic gastric cancer progress following initial treatment with an anti-HER2 regimen, there are no approved HER2-directed medicines,” said José Baselga, executive vice-president, Oncology R&D, AstraZeneca, in the press release. “The prognosis for these patients is poor, as available treatment options offer only limited clinical benefit. This milestone brings us one step closer to delivering a potentially practice-changing medicine to patients with gastric cancer in the [United States].”

“The results of the Destiny-Gastric01 trial are unprecedented as they represent the first time a HER2-directed medicine has demonstrated an improvement in survival following chemotherapy and HER2 treatment in the metastatic setting,” added Antoine Yver, executive vice-president and global head, Oncology R&D, Daiichi Sankyo, in the press release. “Building on the recent [b]reakthrough [t]herapy [d]esignation, the filing of the application, and [p]riority [r]eview by [FDA] for this potential new indication for Enhertu reflects the importance of the data and the significant unmet need for patients with previously treated HER2-positive metastatic gastric cancer.”

FDA has granted priority review for the companies’ sBLA for Enhertu, an ADC for treating gastric cancers.

AstraZeneca and Daiichi Sankyo Granted Priority Review for Gastric Cancer sBLA

Catalent has signed an agreement with Bone Therapeutics for the acquisition of Skeletal Cell Therapy Support SA (SCTS), which is a subsidiary of Bone Therapeutics. The acquisition, which is expected to close in November 2020, was announced in an Oct. 29, 2020 press release.

Under the terms of the agreement, Catalent will acquire the subsidiary company, and all of its assets located in Gosselies, Belgium, through the purchase of all SCTS shares that are currently owned by Bone Therapeutics. The deal will include a purpose-built current good practice (CGxP) facility of approximately 41,000 ft2, which incorporates quality control and product development laboratories, warehouse, grade C and B cleanrooms and equipment, and land for further development. Additionally, Catalent will take on the manufacturing of clinical material for Bone Therapeutics’ ALLOB—an allogenic osteoblastic cell therapy product derived from 

The acquired facility is located next to Catalent’s existing cell therapy site, which will enable Catalent to expand its cell therapy capabilities and advanced clinical and commercial supply, as well as create an integrated European center of excellence in cell therapy. All the SCTS manufacturing employees will remain with the company and become part of the Catalent workforce.

“Catalent is committed to supporting the development and manufacture of next-generation therapies and bringing life-changing treatments to patients in need,” said Manja Boerman, president, Catalent Cell & Gene Therapy, in the press release. “Following the acquisition of MaSTherCell, the additional manufacturing capacity and technical expertise that this deal with Bone Therapeutics gives us will immediately expand our functional clinical and commercial capacity for current late-stage customers. This addition also creates a strong center of cell therapy excellence for Catalent in Europe and will serve as a hub for the growing cell therapy pipeline.”

“This agreement with Catalent is an important strategic step for Bone Therapeutics and is the result of our continuous effort to optimize our operations,” added Miguel Forte, CEO, Bone Therapeutics, in the press release. “The integration of our manufacturing subsidiary into Catalent, one of the premier contract manufacturers in the cell therapy space, significantly increases our operational flexibility. It enables Bone Therapeutics to maximize the efficient use of our manufacturing assets by combining the manufacturing facility and specialist operators with Catalent’s global network of clinical and commercial manufacturing facilities. This will ensure the most efficient ongoing global production of ALLOB.”

Catalent has signed an agreement with Bone Therapeutics for the acquisition of Skeletal Cell Therapy Support SA (SCTS).

Catalent Acquires Bone Therapeutics’ Cell Manufacturing Facility 

Contract development and manufacturing organization (CDMO), 3P Biopharmaceuticals, and Oxford University spin-out, SpyBiotech, have signed a vaccine contract manufacturing agreement, it was announced in an Oct. 26, 2020 press release.

SpyBiotech’s SpyCatcher/SpyTag platform technology enables antigens to be displayed onto virus-like particles with a covalent, irreversible bond in a stable and effective way with specific orientation/epitope presentation and high density. The company states that the technology has broad applicability in vaccine development and has established proof of concept data in viral, bacterial, parasitic diseases, chronic diseases, and cancer.

In June 2019, SpyBiotech selected 3P Biopharmaceuticals for the manufacture of a current good manufacturing practice (CGMP) batch of its virus-like particle carrier, with the aim for production to be completed in 2021. Through this latest agreement, 3P Biopharmaceuticals will demonstrate its capacity to perform processes and manufacturing of molecules developed for diverse therapeutic applications.

“It is amazing being part of a project which presents an opportunity to develop treatments for a broad range of diseases,” said Dámaso Molero, CEO of 3P Biopharmaceuticals, in the press release. “We are helping to support manufacturing which we believe will open the door for pioneering vaccines which could offer a hope for improving the lives of many people. That is extremely exciting.”

Genevieve Labbe, senior scientist and GMP project manager for SpyBiotech added in the press release, “It’s critical to have a trusted partner in place for cell line and CGMP production. SpyBiotech is looking forward to advancing this product and we are delighted to be working with 3P Biopharmaceuticals.”

3P Biopharmaceuticals, and Oxford University spin-out, SpyBiotech, have signed a vaccine contract manufacturing agreement.

3P Biopharmaceuticals and SpyBiotech Sign Vaccine Manufacturing Agreement

A United Kingdom consortium of biotech companies, charities, and academia, the UK BIA Antibody Taskforce, has identified differentiated antibody combinations that will be taken into development as COVID-19 antibody therapy candidates.

According to an Oct. 29, 2020 press release, the taskforce developed an accelerated and rigorous multifaceted approach that allowed them to create a pool of more than 600 novel candidates and identify a set of antibodies with the greatest potential to treat and protect against SARS-CoV-2. These antibodies are the first to be selected for the next stage of development and assessment of their efficacy as a ‘cocktail’ is on-going.

“We have accelerated the standard timelines for antibody discovery, taking seven months rather than the industry standard 18 months, establishing a pathway that can be applied to future pandemics,” said Dr. Jane Osbourn OBE, chief scientific officer at Alchemab and leader of the taskforce, in the press release. “We believe that the most effective tool against COVID-19 will most likely be a defined mixture of two to three antibodies—so the effectiveness of different combinations must also be assessed.”

“This ground-breaking taskforce has brought together UK-based industry experts who share a joint commitment to help fight the COVID-19 pandemic,” added Steve Bates OBE, CEO of the UK BioIndustry Association (BIA), in the press release. “Contributing their expertise and specialist resources, the team has worked tirelessly to identify the most promising antibodies with the potential to positively impact treatment of those affected by this devastating virus. The next stage will involve securing external funding to facilitate the further development and manufacture of the candidates.”

The UK BIA Antibody Taskforce has identified differentiated antibody combinations that will be taken into development as COVID-19 antibody therapy candidates.

UK BIA Antibody Taskforce Identifies Novel COVID-19 Antibody Therapy Candidates

Privately held biopharmaceutical company, NanoMab Technology, has signed heads of terms with a supplier of Tungsten-188/Rhenium-188 generators, OncoBeta, it was revealed in an Oct. 27, 2020 press release.

The heads of terms outline the route toward a strategic alliance and supply agreement between the companies that will target the development of Rhenium-188 API solutions. Rhenium-188 API solutions are suitable for NanoMab’s range of nanobodies for the production of therapeutic radiopharmaceuticals.

“This is an important step for us in our development of cancer therapies,” said Dr. Hong Hoi Ting, NanoMab’s chief scientific officer, in the press release. “Ensuring that we have a global supply of Rhenium-188 is critical to our development plan and we are therefore delighted to be forming a partnership with such a high-quality company as OncoBeta.”

Nicholas H. Vetter, OncoBeta’s chief operating officer, added in the press release, “We have been impressed with NanoMab’s ambitious program to develop a range of radiopharmaceuticals for the treatment of a number of cancers. We’re delighted to work towards forming a partnership with NanoMab around the use of Rhenium-188 for further therapeutic purposes.”

NanoMab Technology has signed heads of terms with a supplier of Tungsten-188/Rhenium-188 generators, OncoBeta.

NanoMab Technology Signs Heads of Terms with OncoBeta

Pfizer has announced that the US Food and Drug Administration (FDA) has accepted for filing and granted priority review designation for a new drug application and the European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for abrocitinib.

According to an Oct. 27, 2020 press release, both regulatory agencies are expected to make decisions in 2021. Abrocitinib is an investigational oral once-daily Janus kinase 1 (JAK1) inhibitor to treat atopic dermatitis (AD) in patients who are 12 years old and older. The filings with both FDA and EMA were based on Phase III clinical trial results, in which abrocitinib demonstrated statistically superior improvements in skin clearance, disease extent, and severity, as well as improvements in itch, when compared with placebo.

“AD is a serious, unpredictable, and often debilitating condition that can have a significant impact on the daily lives of patients and their families,” said Michael Corbo, PhD, chief development officer, Inflammation & Immunology, Pfizer Global Product Development, in the press release. “We are grateful to those who participated in our clinical studies supporting these regulatory filings and proud that the FDA has granted abrocitinib both Breakthrough Therapy and Priority Review designations. We are working diligently with the regulatory authorities to bring abrocitinib to patients in the US and the EU [European Union], where, if approved, it may provide an effective and convenient new option.”

“Many patients with moderate to severe atopic dermatitis have poorly controlled disease. They need additional treatment options that alleviate the symptoms most important to them,” said Jonathan Silverberg, MD, PhD, Department of Dermatology, The George Washington University School of Medicine and Health Sciences. “Abrocitinib has demonstrated strong efficacy at relieving the signs and symptoms of AD, including rapid reduction of itch, across multiple clinical trials. If abrocitinib is approved, it could make a meaningful difference in real-world clinical practice.”

Pfizer has announced that FDA has accepted for filing and granted priority review designation for a new drug application and EMA has accepted the MAA for abrocitinib.

Pfizer Gains FDA and EMA Acceptance for Abrocitinib

Novartis and Molecular Partners announced on Oct. 28, 2020 that they have entered into a collaboration for the development, manufacture, and commercialization of Molecular Partners’ anti-COVID-19 DARPin program, which comprises two therapeutic candidates, MP0420 and MP0423.

As per the terms of the agreement, during the option period, Molecular Partners will perform Phase I clinical trials for MP0420, expected to begin in November 2020, and perform all remaining preclinical work for MP0423. Novartis will be charged with conducting the Phase II and Phase III clinical trials, which will be sponsored by Molecular Partners.

Upon option exercise, Novartis will be responsible for all further development and commercialization activities. During the clinical development stage, Molecular Partners will provide clinical supply. Manufacturing capacity scale-up will be a collaborative effort of Novartis and Molecular Partners, along with Sandoz, the generics and biosimilar Novartis division, to provide worldwide supply.

“Novartis remains unwavering in its support for tackling COVID-19 and it is clear that this pandemic calls for not just scientific solutions, but also for collaboration between companies to provide treatments in an area of high unmet need,” said Vas Narasimhan, CEO of Novartis, in the press release. “This Swiss led partnership, which could deliver both prophylactic and treatment options at scale for COVID-19 patients across the globe, is another demonstration of our sustained commitment to addressing one of the greatest health challenges of our time.”

“Our team rapidly mobilized to deliver a unique DARPin-based approach to address the overwhelming need for effective therapeutics against COVID-19. As a class, DARPins have demonstrated over years of clinical research a number of characteristics that enhance their profile as antiviral therapeutics for a global pandemic. We have built on this long-term research with these two candidates, which have demonstrated extremely potent neutralization of the virus through inhibiting multiple viral mechanisms,” added Patrick Amstutz, CEO of Molecular Partners, in the press release.

Novartis and Molecular Partners are collaborating on the development, manufacture, and commercialization of Molecular Partners’ anti-COVID-19 DARPin program.

Novartis Collaborates with Molecular Partners on Two Anti-COVID-19 Therapies 

Sanofi and GSK announced on Oct. 28, 2020 that they have signed a Statement of Intent with Gavi, the legal administrator of the COVAX Facility, a global risk-sharing mechanism for the distribution of future COVID-19 vaccines, for the availability of 200 million doses of their adjuvanted recombinant protein-based COVID-19 vaccine pending regulatory approval.

“To address a global health crisis of this magnitude, it takes unique partnerships. The commitment we are announcing today for the COVAX Facility can help us together stand a better chance of bringing the pandemic under control,” said Thomas Triomphe, executive vice-president and Global Head of Sanofi Pasteur, in an Oct. 28, 2020 press release. “This moment also reflects our long-term commitment to global health and ensures our COVID-19 vaccines are affordable and accessible to those most at risk, everywhere in the world.”

"Since we started working on the development of COVID-19 vaccines, GSK has pledged to make them available to people around the world,” added Roger Connor, president of GSK Vaccines, in the press release. “We are proud to be working with Sanofi to make this adjuvanted recombinant protein-based vaccine available to the countries signed up to the COVAX Facility as soon as possible—this has the potential to be a significant contribution to the global fight against COVID-19.”

The companies have signed a Statement of Intent with Gavi to make 200 million doses of their adjuvanted recombinant protein-based COVID-19 vaccine available for distribution. 

Sanofi and GSK Agree to Support COVAX with 200 Million COVID-19 Vaccines Doses

Eli Lilly and Company announced on Oct. 28, 2020 that it has entered into an agreement with the US government to supply 300,000 vials of bamlanivimab (LY-CoV555), an investigational neutralizing monoclonal antibody for the treatment of mild to moderate COVID-19 in high-risk patients, for $375 million.

Under the terms of the agreement, the government will accept the vials if Emergency Use Authorization (EUA) is granted by FDA, an Eli Lilly press release said. Delivery will commence over the course of two months following the EUA, and the government will have the ability to purchase up to 650,000 additional vials of the treatment through June 30, 2021 based on product availability and medical need. The company said in the release that it is partnering globally with governments and philanthropic organizations around the world to allocate the limited supply of antibody therapies.

"Lilly has leveraged our deep scientific capability to fight this pandemic, and we are proud of our efforts to develop potential medicines to combat COVID-19. Supply agreements with governments—such as this one with the US government to meet Operation Warp Speed goals—are fundamental to enable the most widespread and equitable access to our potential therapy," said David A. Ricks, Lilly's chairman and CEO, in the press release. "The US is experiencing a surge in COVID-19 cases and associated hospitalizations, and we believe bamlanivimab could be an important therapeutic option that can bring value to the overall healthcare system, as it has shown a potential benefit in clinical outcomes with a reduction in viral load and rates of symptoms and hospitalizations."

Lilly noted in the release that it has been investing in large-scale manufacturing at risk and anticipates producing up to one million doses of bamlanivimab 700 mg by the end of 2020, with 100,000 doses ready to ship globally after authorization. The company expects to continue to bring more manufacturing resources online throughout the year, and expects the therapy’s supply to increase significantly in the first quarter of 2021.

Delivery will commence over the course of two months following the EUA and the government will have the ability to purchase up to 650,000 additional vials of the treatment through June 30, 2021 based on product availability and medical need.

Eli Lilly to Provide the US Government with 300,000 vials of its COVID-19 Treatment for $375 Million

Clarke Solutions, Covex LLC, and Acquire Automation announced on Oct. 21, 2020 that they are combining and will rebrand as Verista, a pharmaceutical, life sciences, food and beverage, and manufacturing solutions provider.

The new company will house more than 350 experts and will offer compliance and validation solutions for pharmaceuticals and medical devices; manufacturing and packaging; sterilization and traceability consulting and solutions; manufacturing and packaging engineering; and automated systems, a company press release said.

“The Verista brand signifies the expertise, trust, and process [for which] our talented team has been known for several decades,” said Manny Soman, CEO, Verista, in the press release. “Verista will continue to uphold the commitment to quality and industry-specific solutions established by Clarke Solutions, Covex, and Acquire Automation and allow our customers to focus on core competencies.”

​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 

The new company will house more than 350 experts and will offer pharmaceutical, life sciences, food and beverage, and manufacturing solutions.

Clarke Solutions, Covex LLC, and Acquire Automation Combine to Form Verista

Novavax’ Phase III clinical trial of NVX-CoV2373, its COVID-19 vaccine candidate, is expanding enrollment in the United Kingdom (UK) and will begin in the US by the end of November, the company reported in an Oct. 27, 2020 press release. The company has enrolled over 5500 participants in the UK trial and plans expanded enrollment to 15,000 volunteers by the end of November. The larger numbers of participants are expected to allow assessment of safety and efficacy in a shorter time period. Interim data in this event-driven trial may be available as soon as early first quarter 2021.

“We are pleased with the significant progress made in our Phase 3 clinical trial since it began in the United Kingdom at the end of September,” said Gregory M. Glenn, president of R&D at Novavax. “We are grateful for the support of the UK Government’s Vaccines Taskforce (VTF) and the National Institute for Health Research (NIHR) for our pivotal trial. Recognizing the recent, large increase in the number of COVID-19 cases in the UK, as well as the rapid progress in participant enrollment for our trial, in consultation with the VTF and NIHR, we adjusted our plans and increased enrollment.”

The company plans to begin its Phase III clinical trial in the US, with support from the US government’s Operation Warp Speed, and in Mexico by the end of November. The company said in the press release that although it experienced delays compared to original timing estimates, it has made significant progress in large-scale manufacturing. Large-scale manufacturing is being developed at multiple sites around the world. Production from contract manufacturer Fujifilm Diosynth Biotechnologies in Morrisville, NC will be used in the Phase III trials in North America.

NVX‑CoV2373 is a stable, prefusion protein made using Novavax’ nanoparticle technology and includes Novavax’ proprietary Matrix‑M adjuvant.

The Phase III clinical trial of NVX-CoV2373 is expected to be fully enrolled in the UK and will begin in the US and Mexico by the end of November 2020.

Novavax COVID-19 Vaccine Trial Expands Enrollment

Catalent and Stevanato Group, an producer of glass primary packaging and integrated systems headquartered in Italy, announced on Oct. 27, 2020 that they are expanding their longstanding collaboration to accelerate the delivery of advanced visual inspection systems to help with potential solutions for the COVID-19 pandemic.

Under the partnership, Catalent has ordered Plus 400 LKD automatic and PWL semi-automatic visual inspection machines from Stevanato Group to install at its Bloomington, IN, and Anagni, Italy facilities to support customer programs, a Stevanato press release said. Stevanato will accelerate the delivery of the equipment by several months to meet the demands of the pandemic.

“Catalent appreciates Stevanato Group’s commitment to delivering these vital inspection machines to our sites ahead of schedule,” said Dominick Marino, vice-president of Global Procurement at Catalent, in the press release. “This added capability will increase the speed in which we package and supply important medicines for our customers and patients.”

“We understand what is at stake, and our engineers are committed to delivering high-performance inspection machines ahead of schedule to meet Catalent’s needs and expectations,” added Raffaele Pace, engineering vice-president of Operations, Stevanato Group, in the press release.

Under the partnership, Catalent has ordered visual inspection machines from Stevanato Group to install at its Bloomington, IN and Anagni, Italy facilities to support customer programs.

Catalent and Stevanato Expand Partnership for the Accelerated Delivery of COVID-19 Solutions

CMIC, a Japanese contract research organization (CRO), and SUSMED, a research and development firm also located in Japan, announced on Oct. 21, 2020 that they have formed a strategic partnership to offer digital therapeutics (DTx) development services from app development to post-launch support.

According to a CMIC press release, the companies will work to provide development strategy consultations, system-building, and clinical trial operations, while lessening development time and reducing risk by centralizing project tasks between companies. The services will be available to pharmaceutical and information technology companies in Japan and worldwide.

“DTx will be an important field in the Japanese healthcare industry going forward, and it is a new market for which regulations and other infrastructure are just starting to be established,” said Toru Fujieda, president of CMIC, in the press release. “SUSMED shares the same pioneering spirit as CMIC to work outside the box. With this partnership we are now able to combine our knowledge in various areas to build and offer an integrated DTx service platform that covers all steps from development to post-launch support. It will support the continuous growth of the DTx market and help advance the healthcare industry in Japan.”

“Leveraging this partnership with CMIC, we can provide our technology to a larger number of companies and help accelerate the overall DTx market growth in Japan,” said Taro Ueno, CEO of SUSMED, in the press release. “We believe that building this one-stop service platform will allow us to deliver more treatment approaches to healthcare providers in Japan.”

​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​  

The companies will work to provide development strategy consultations, system-building, and clinical trial operations, while lessening development time and reducing risk by centralizing project tasks between companies.

CMIC and SUSMED to Collaborate on Providing Digital Therapeutics Development Services

On Oct. 27, 2020, Evonik introduced the Eudragit IR ReadyMix White, a new ready-to-use powder premix blend of its Eudragit functional polymers used for oral products with an immediate release profile.

The new premix can be used with tablets and a range of other solid oral dosage forms and can be processed at half the time of HPMC or PVA-based ready-to-use alternatives because of its high solid content and low processing temperature, a company press release said. It also features a high-gloss coating and can be used to optimize the processability, appearance, taste, and swallowability of drug products.

The new premix can be used with tablets and a range of other solid oral dosage forms.

Evonik Launches New Ready-To-Use Powder Premix Blend

Thermo Fisher Scientific announced on Oct. 27, 2020 that it has expanded its Direct-to-Patient service offerings to support decentralized clinical trials with the goal of lessening clinical trial lengths and costs, while increasing patient enrollment and participation rates.

"Decentralized clinical trials have been on the rise even before the COVID-19 pandemic due to the location of study centers and the amount of time required for study visits being barriers to patient enrollment and ongoing participation," said Chris Armstrong, president, clinical trials business, Thermo Fisher Scientific, in a company press release. "As patients increasingly reach out to clinical trials sponsors to request receiving medication at home, it's critical to have a solution that delivers patients' medications safely and on time, without compromising trial quality and speed."

Pharmacy-to-Patient and Depot-to-Patient services for centralized control and supply chain planning to manage high-value or limited supply agents.

Clinical Site-to-Patient services that deliver medications directly to patients' homes or study sites.

Direct-to-Patient solutions for ancillary sourcing, blinding, patient kit production, clinical supply optimization, and decentralized clinical trial setups.

The Direct-to-Patient services include Pharmacy-to-Patient, Depot-to-Patient, and Clinical Site-to-Patient services. 


Thermo Fisher Expands its Direct-to-Patient Service Offerings

The CountQ system from Verista is an inspection and reconciliation machine for tray-loaded parts that increases accuracy of count and throughput versus manual methods. The machine handles vials, syringes, ampoules, and other tray-loaded parts. The system uses deep learning, which is a type of artificial intelligence (AI), to inspect products that cannot be counted consistently with traditional machine vision technologies due to variation in products and presentation to the camera, such as the inspection of ampoules.

“How quickly COVID-19 vaccine production ramps up will depend on technology like CountQ for vial count verification and reconciliation,” says Jim Evans, director of the Vision, Connectivity, and Automation Division at Verista (a company formed in 2020 by unifying Clarke Solutions, Covex LLC, and Acquire Automation). “The CountQ system provides vaccine manufacturers increased efficiency and count accuracy, while reducing operational costs. Our unique AI-based counting system works with an operator to verify everything on a tray of vials is accounted for and correct.”

The system enables a trained operator to accurately inspect a tray in 15 seconds. Software provides 100% electronic verification of product counts and eliminates count deviations that can occur during the filling, inspection, and packaging processes. The stainless-steel systems are available in multiple portable forms, including full-sized units with conveyor transport and smaller manual feed and inline options.

The CountQ system from Verista automates accurate counts of tray-loaded parts, including vaccine vials. 

Inspection and Reconciliation Machine Accurately Counts Tray Loads

Bayer announced on Oct. 26, 2020 that it will acquire Asklepios BioPharmaceutical (AskBio), a Research Triangle Park, NC-based biopharmaceutical company focused on the manufacturing of gene therapies, for up to $4 billion.

Under the terms of the acquisition, Bayer will pay $2 billion upfront and will provide up to $2 billion more in success-based milestone payments, a Bayer press release said.

Bayer will now own the rights to AskBio’s adeno-associated virus (AAV)-based gene therapy platform, its intellectual property portfolio, and an established contract development and manufacturing organization, the press release said. Additionally, Bayer will acquire AskBio’s gene therapy technology platform and its existing gene therapy manufacturing platform through the agreement.

“In line with our purpose ‘science for a better life’, we are committed to bringing significant improvements for patients through innovation,” said Werner Baumann, chairman of the board of management and CEO of Bayer, in the press release. “With this acquisition, Bayer significantly advances the establishment of a cell and gene therapy platform that can be at the forefront of breakthrough science, contributing to preventing or even curing diseases caused by gene defects and further driving company growth in the future.”

“With Bayer‘s worldwide reach and translational expertise, especially in pathway diseases, our combined cultures of scientific advancement and commitment to patients, along with the retention of AskBio’s independent structure, Bayer and AskBio are positioned to provide accelerated development of gene therapies to treat more patients who can benefit from them,” said Sheila Mikhail, CEO and co-founder of AskBio, in the press release.

Bayer will own the rights to AskBio’s AAV-based gene therapy platform, its intellectual property portfolio, and an established CDMO.

Bayer to Acquire AskBio for up to $4 Billion

FDA announced on Oct. 22, 2020 the approval of Gilead’s antiviral drug Veklury (remdesivir) as the first treatment for patients with COVID-19 that require hospitalization. The approval was designated for adult and pediatric patients 12 years of age and older, weighing at least 40 kilograms (88 pounds) and must be administered in a hospital or in a healthcare setting that provides acute care comparable to inpatient hospital care.

The agency also issued a new Emergency Use Authorization for Veklury for hospitalized pediatric patients under 12 years of age weighing at least 3.5 kg or hospitalized pediatric patients weighing 3.5 kg to less than 40 kg with suspected or laboratory confirmed COVID-19 for whom use of an intravenous agent is clinically appropriate. Clinical trials to assess the safety and efficacy for the pediatric patient group are ongoing.

In announcing the approval, FDA noted that the decision was based on analysis of three randomized, controlled clinical trials that included patients hospitalized with mild-to-severe COVID-19.

In one study, the median time to recovery from COVID-19 was 10 days for the Veklury group compared to 15 days for the placebo group. The odds of clinical improvement at Day 15 were also statistically significantly higher in the Veklury group when compared to the placebo group, FDA reported.

A second trial showed the odds of a subject’s COVID-19 symptoms improving were statistically significantly higher at day 11 for a five-day Veklury treatment group when compared to those receiving only standard of care. For patients in a 10-day treatment group, odds of symptom improvement when compared to those receiving only standard of care were numerically favorable, but not statistically significantly different, FDA reported.

A third trial of hospitalized adults with severe COVID-19 compared treatment with Veklury for five days and treatment with Veklury for 10 days, with clinical status evaluated on day 14. The study found the odds of a subject’s symptoms improving were similar for those in the five-day Veklury group as those in the 10-day Veklury group; there were no statistically significant differences in recovery rates or mortality rates between the two groups, FDA reported.

FDA granted Veklury Fast Track and Priority Review designations and a Material Threat Medical Countermeasure Priority Review Voucher.

Gilead’s antiviral drug Veklury is the first approved treatment in the US for COVID-19.

FDA Approves Remdesivir for Hospitalized Patients

Vision artificial intelligence (AI) software company Neurala announced a new strategic partnership on October 20, 2020, with IMA Group, which designs and manufactures automation equipment for processing and packaging of pharmaceuticals and in other markets. The partnership will focus on field testing of AI used for monitoring and analyzing data collected through industrial internet of things (IIoT) systems.

“As the Industrial Internet of Things (IIoT) becomes commonplace—with dozens of sensors and cameras gathering product data, as well as basic diagnostics from industrial equipment—manufacturers critically need human-level AI that can extract actionable insights from that data at the compute edge,” said Max Versace, CEO and cofounder of Neurala, in the press release. “Data without AI is not useful, and off-the-shelf cloud-based AI is not cut out for the manufacturing floor. Unlike traditional AI solutions, Neurala’s technology is trained at the edge, enabling it to continuously learn based on manufacturers’ data as it varies across specific machines and production runs. We are incredibly excited to bring this technology together with IMA’s industry-leading solutions and expertise. Our new partnership will demonstrate just how far edge AI can go in solving industrial monitoring challenges on a global scale.”

“Today, factory floors are more complex than ever, and manufacturers are challenged when it comes to managing the extremely high volume of data they’re amassing,” said Dario Rea, director of Corporate Research and Innovation, IMA Group, in the press release. “To become even more competitive on the global stage, and to help manufacturers overcome this complexity, IMA must improve not only the quality of our products, but also, internal visibility on equipment health and productivity.”

Neurala will work with IMA to install the technology directly on industrial machines, at the compute edge, allowing operators to quickly and independently set up advanced AI systems without requiring specialized expertise. As part of the partnership, IMA has made a targeted investment in Neurala.

Edge-AI technology will enable Industry 4.0 automation in pharmaceutical manufacturing.

Neurala Partners with IMA Group on AI for Manufacturing Automation

The third part of the CPhI annual report, which was released during the Festival of Pharma virtual event, has predicted a significant amount of pressure will be placed on bioprocessing networks should COVID-19 biologics gain approval.

According to an Oct. 19, 2020 press release, Dawn M. Ecker, managing director of bioTRAK Database Services at BDO, provided her expertise in the report and specified that although short-term capacity issues for biologics have been eased, there may be challenges encountered beyond 2024. Primarily, future commercial capacity may be impacted by the progress of Alzheimer’s drugs, PDL/PDL-1 checkpoint inhibitors, and COVID-19 therapeutics currently in late stage development.

“Should indications, such as treatments for Alzheimer’s disease or broad cancers obtain regulatory approval, a significant increase in demand for manufacturing capacity could occur, potentially leading to a serious capacity shortage,” Ecker said in the press release. “We also face an additional challenge of incorporating demand for recombinant proteins to combat COVID-19. We conservatively estimate that the demand for these products could require approximately 30 metric tonnes per year—so this potential demand will add significant pressure to manufacturing networks with large-scale capacity.”

In the report, Ecker has highlighted that the global capacity for biomanufacturing is expected to increase to 6500 kL by 2024, yet the demand for capacity will increase at a faster compound annual growth rate of approximately 12% by 2024. Additionally, in her analysis of manufacturing trends, Ecker reports that capacity is moving from big pharma to contract manufacturing organizations and hybrids.

“Over the last five years there has been modest capacity growth in North America and Europe, with significantly greater growth in Asia. The next few years, however, will see significant growth rates projected in both Asia (~7%) and Europe (~12%), and by 2024 Europe will have equivalent capacity to the US for the first time,” added Ecker, in the press release. “Looking deeper at the country level, particularly Korea, Singapore, and Ireland, we see that growth here reflects government incentives and tax advantages, amongst other factors.”

The third part of the CPhI annual report has predicted a significant amount of pressure will be placed on bioprocessing networks should COVID-19 biologics gain approval.

Biomanufacturing Capacity Facing Huge Pressure, Notes CPhI Annual Report

California-based JSR Life Sciences has increased its European footprint with a new expanded facility in Geneva, Switzerland, that will combine the company’s two affiliates, KBI Biopharma and Selexis SA, in one campus. The expansion was announced in an Oct. 20, 2020 press release.

The Stellar 32 campus will provide 8700 m

 of space for the affiliate companies to co-locate and will provide the opportunity for European customers of JSR Life Sciences to receive current good manufacturing practice (CGMP) biologics bulk drug substance manufacturing and highly specialized mammalian cell line development services. It is anticipated that the new campus will create more than 250 new highly technical jobs.

“JSR Life Sciences is proud to have best-in-class companies focused on translating customers’ ideas into novel biologics that are improving patients’ lives worldwide,” said Tim Lowery, president of JSR Life Sciences, in the press release. “This strategic investment in our European operations creates new options by linking the core strengths of our affiliates, increases efficiencies, and ultimately accelerates our customers’ ability to bring innovative life science products into the marketplace. In addition, KBI and Selexis will benefit from the extensive biopharma industrial base, advanced technical resources, and specialized education offerings in this region.”

Part of the expansion will comprise a 5600 m

 biologic bulk drug substance manufacturing facility for KBI Biopharma, which is expected to be operational by mid-2022. Through this facility, KBI Biopharma will be able to supply customers with CGMP bulk drug substance for clinical trials. Selexis’ expanded footprint will include new standard cell line development suites and two separate cell culture suites to accommodate quarantined client-specific cell lines or client-dedicated cell line spaces, which are expected to be operational by mid-2021.

“This expansion is a cornerstone of our European growth strategy, which builds on Selexis’ innovative cell line technologies and KBI’s strong expertise in biologics manufacturing to meet the needs of our clients’ clinical trial requirements. The new facility allows us to increase engagement with a broader client base with whom we can partner more effectively due to our local presence,” added Dirk Lange, president and CEO of KBI Biopharma and CEO of Selexis SA, in the press release. “As science progresses, research and treatments become increasingly complex and sophisticated, and we are fully prepared to keep pace with our clients’ needs by providing the latest life science discovery and development technology. Furthermore, by co-locating these operations, the synergies for rapid cell line and process development and introduction to manufacturing will be tremendous, which we see as a true advantage for our client base.”

JSR Life Sciences has increased its European footprint with a new expanded facility in Switzerland, that will combine the company’s two affiliates in one campus.

JSR Life Sciences Expands European Footprint

The European Commission (EC) has granted orphan drug designation to Pharming Group for its treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), leniolisib, based on the positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicine Agency (EMA).

“We are pleased to have received orphan drug designation from the EC, an important milestone in the development of leniolisib for the treatment for APDS, an ultra-rare and debilitating disease,” said Sijmen de Vries, CEO of Pharming, in an Oct. 21, 2020 press release. “With no currently approved treatment, leniolisib has the potential to address a significant unmet need for patients with APDS. Leniolisib is currently being studied in a registration-enabling Phase II/III trial and remains, subject to regulatory approval, on track to launch in H2 2022.”

Leniolisib is a small molecule phosphoinositide 3-kinase delta inhibitor with immunomodulating and potentially anti-neoplastic activities. It has been granted orphan drug designation by the US Food and Drug Administration in January 2018 for the treatment of APDS or p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency.

EC has granted orphan drug designation to Pharming Group for its treatment of activated phosphoinositide 3-kinase delta syndrome, leniolisib.

EC Grants Orphan Drug Designation to Pharming’s Leniolisib

The Swiss marketing and distribution partner of Diurnal Group, EffRx Pharmaceuticals, has submitted a marketing authorization application (MAA) for Alkindi to the Swiss regulatory body, Swissmedic.

According to an Oct. 21, 2020 press release, the MAA has been based on the European regulatory dossier and published clinical trial data on Alkindi—hydrocortisone granules in capsules for opening. EffRx anticipates a potential market launch for Alkindi in Switzerland by 2022 and will receive the exclusive rights to market and sell the product in Switzerland once the product has been registered.

“We are pleased to see the submission of a MAA for Alkindi in Switzerland by EffRx,” said Martin Whitaker, CEO of Diurnal, in the press release. “[This] announcement is yet further validation of the quality of our products and broadens the future availability of Alkindi outside our core markets in line with our global strategy. We look forward to working with EffRx to make Alkindi available to patients suffering from pediatric adrenal insufficiency (AI) in Switzerland.”

Alkindi is already approved and marketed in Europe and is the first preparation of hydrocortisone that has been specifically designed for pediatric use in the treatment of AI and the related condition, congenital adrenal hyperplasia.

The Swiss marketing and distribution partner of Diurnal Group, EffRx Pharmaceuticals, has submitted a MAA for Alkindi to Swissmedic.

Featured Content

2020 Bio/Pharma Virtual Congress: November 11, 2020

COVID-19 Therapy Developments

Unique Molecules Push Formulation Boundaries