Susan Haigney

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Bespak, a contract development and manufacturing organization that specializes in inhalation products, announced on Nov. 6, 2024 that it is partnering with Orbia Advance Corporation, S.A.B. de C.V (BMV: ORBIA) Four & Energy Materials (Orbia Fluor & Energy Materials) to transition to climate-friendly inhalers. As part of the collaboration, Orbia, a supplier of fluoroproducts and technologies, will provide Bespak with its low global warming potential (GWP) propellant, Zephex 152a for use in Bespak’s pressurized metered dose inhalers (pMDIs).

Bespak is making the transition to the GWP propellant as part of the overall pMDI industry’s commitment to sustainable solutions. The company verified the use of producing pMDI formulations with GWP propellants with the assistance of its network of partners and its good laboratory practice (GLP) lab-scale testing, as well as its clinical- and commercial-scale good manufacturing practice (GMP) capabilities. Bespak will also utilize Orbia’s clinical-scale GLP and GMP capabilities.

The two companies will work closely at their locations in North West, England, with support from Bespak’s King’s Lynn, United Kingdom, location, which has conducted research on a range of valves and actuators.

“We are committed to leading the transition to low GWP propellants in pMDIs, and in collaboration with Orbia Fluor & Energy Materials, we will enable customers to progress their inhaler portfolios utilizing Zephex 152a,” said Chris Hirst, CEO of Bespak, in a press release (1). “Our goal is to convert as many formulations as possible to maintain patient access to pMDIs as global regulation evolves, and we look forward to working with Orbia Fluor & Energy Materials to provide customers with a pathway to commercialization at our Holmes Chapel site.”

“This is another step forward in bringing pMDIs using our low GWP medical propellant Zephex 152a to the market,” said Markus Laubscher, general manager of Orbia Fluor & Energy Materials Pharma Business Unit, in the release. “We are excited to extend our industry collaborations to Bespak who bring the needed capabilities to the table, from their expertise in valves and actuators, to their demonstrated commitment to clinical and commercial-scale product manufacturing.”

Bespak has made recent investments in its development services through its market approval services for all pMDI formulations and propellants. It has also increased capacity for commercial supply. In October 2024, the company announced that, in connection with H&T Presspart, it had added a GMP pilot-scale filling line for pMDIs to produce GWP propellants HFDA-152a and HFO-1234ze (2). The new filling line provide access to small-scale GMP capabilities for development programs and clinical trials.

“This new low GWP pilot-scale filling capability addresses the short-term market need for clinical trial supply and will help pharma companies to advance their low GWP development programs towards commercialization,” Hirst said in the press release. “When added to our rapidly expanding low GWP commercial-scale capability, it is clear that we can now offer customers a complete transition pathway.”

“H&T Presspart is committed to supporting the industry in transitioning to low GWP propellants,” said Christian Krätzig, president of H&T Presspart, in the release. “In our partnership with Bespak, we are now leveraging our combined expertise to create the infrastructure needed for small-scale manufacturing with low GWP propellants. The launch of a GMP pilot-scale filling line is an important step forward, and we are excited about future collaborations that will help to create a more sustainable pMDI platform for the next generation.”

Bespak. Bespak and Orbia Fluor & Energy Materials Unite to Accelerate Transition to Climate-friendly Inhalers. Press Release. Nov. 5, 2024.

Bespak. Bespak & H&T Presspart Announce Availability of GMP Pilot Line for Low GWP pMDIs. Press Release. Oct. 8, 2024.

Articles

The companies will work to create climate-friendly pMDIs using Orbia’s low global warming potential propellant.

Bespak and Orbia Fluor & Energy Materials Collaborate on Climate-Friendly Inhalers

Aptamer Group announced on Nov. 7, 2024 that one of its customers has advanced use of the company’s Optimer binders for the commercial phase of its product. The customer has used the Optimer delivery vehicle for the product’s development phase and Aptamer shipped test materials to the customer in October 2024.

According to Aptamer, the customer has validated the performance of the delivery vehicles, indicating the vehicles can target the required cell type. The vehicles have the potential to be used as part of a precision therapy. This would create downstream licensing revenue for Aptamer.

The customer also found that Optimer showed binding to the target cells from a panel of animal species, which makes the delivery vehicle desirable for preclinical development. “Due to the targeting specificity, multiple species binding, and reproducibility of the results, the customer has committed to progressing to the final commercial development stage for the therapeutic delivery vehicle,” Aptamer stated in a press release (1).

According to Aptamer, targeting genetic medicines to specific cell types is a challenge. Highly selective delivery vehicles address this challenge by targeting these cell types, which has been difficult in the past. The company is developing delivery vehicles that target fibrotic liver cells, creating progress for genetic medicine payloads.

“We are delighted with our customer’s decision to advance to the final stage of Optimer development. The customer’s results align with those seen within our own laboratories and validate the hard work of our scientific team to develop novel, high-performance delivery vehicles,” said Arron Tolley, chief executive officer of Aptamer Group, in the press release. “As this Optimer advances through the final development stages, we continue to move closer to demonstrating the strength of our Optimer platform in generating high-value, therapeutic targeting assets. This represents our second drug delivery asset with licensing potential, alongside our liver fibrosis targeting Optimer, which has already shown good effectiveness in delivering functional genetic medicines to specific cells. These could enable new approaches to precise drug delivery across the field of genetic medicines whilst underpinning our business model with passive income from licensing. We look forward to updating shareholders on future progress in due course.”

Earlier in 2024, the company announced an agreement with AstraZeneca to evaluate the use of Optimer for targeted delivery of small interfering RNA (siRNA). Aptamer will conduct in-house experiments to assess the effectiveness of the delivery vehicle with AstraZeneca’s siRNA as part of the agreement.

“Delivery of siRNA to precise cell types and tissues with successful cell internalization remains a significant challenge for the wider therapeutic application of the technology. Despite this limitation, the siRNA market was still valued at over $13 billion in 2023. Optimer technology could represent a paradigm shift in the targeted delivery of siRNA molecules, due to the high levels of selectivity, high affinity, and simple conjugation of siRNA payloads, offered using Optimer delivery systems as non-viral vectors. If successful, Optimer-enabled delivery of siRNA could lead to the development of novel compounds that have significant advantages over current cell and tissue-targeting methods,” the company stated in a press release (2).

“We are really excited to work with AstraZeneca to evaluate and optimize our Optimer delivery vehicles,” said Tolley in the release. “Partnerships such as this will enable Aptamer Group to make rapid progress in this area, which is a key focus for Optimer technology. Targeted delivery to specific cell types remains a critical unmet need in many applications within the tissue targeting space and, as such, has been part of our strategic focus to support the development of binders with the potential for long-term, high value. Our initial dataset in Optimer targeted delivery has raised significant interest from multiple parties. We are eager to advance to animal model testing and delighted to continue collaborating with AstraZeneca in this endeavor. Reaching proof of principle in animal models will derisk the Optimer delivery platform and bring us closer to delivering targeted and effective gene therapies for patients.”

Aptamer Group. Aptamer Extends Agreement with Genetic Medicines Customer. Press Release. Nov. 7, 2024.

Aptamer Group. Agreement to Explore Optimer Vehicles for Targeted Delivery of siRNA. Press Release. July 3, 2024.

A genetic medicines customer has selected the company’s binders for commercial development of the customer’s product.

Aptamer Extends Agreement for Drug Delivery Vehicles

Avid Bioservices, a biologics contract development and manufacturing organization (CDMO), announced on Nov. 6, 2024 that it has entered into a merger agreement, valued at approximately $1.1 billion, with GHO Capital Partners and Ampersand Capital Partners.

Under the terms of the agreement, GHO and Ampersand will acquire all outstanding shares held by Avid’s stakeholders for $12.50 a share in cash, which is a 13.8% premium to the Nov. 6, 2024 closing price of $10.98 and a 21.9% premium to Avid’s 20-day volume-weighted average share price. The transaction is expected to close in the first quarter of 2025, subject to customary closing conditions, approval by Avid’s stockholders, and receipt of required regulatory approvals.

“Since our founding, Avid Bioservices’ business has grown by evolving to meet our customers' broad range of development and manufacturing needs. After years of investment and expansion, now is the right time to move forward as a private company with new owners that will support our next phase,” stated Nick Green, president and CEO of Avid Bioservices, in a press release (1). “In evaluating this transaction, our [b]oard considered a range of alternatives and determined that it provides our stockholders significant, immediate, and certain cash value for their shares. Partnering with GHO Capital and Ampersand Capital Partners allows us to build on our strong foundation by accessing their significant knowledge base, network and capital to position the business for the future with our customers.”

“We are excited to announce this recommended cash acquisition of Avid,” said Alan MacKay and Mike Mortimer, managing partners of GHO, in the release. “As experienced CDMO industry investors, GHO brings deep expertise and experience to support Avid's management team going forward. Our mission at GHO is to make healthcare better, faster, and more accessible and at the heart of this is enabling efficient, high-quality manufacturing of innovative treatments. Avid exemplifies this perfectly—the [c]ompany operates in high-growth markets, producing complex biologics for leading pharmaceutical and biotech innovators at both the clinical and commercial stages. Avid’s recent investments, both in capacity and its exemplary team, position it strongly for future growth. We look forward to working with the Avid team to unlock the [c]ompany’s full potential through our established playbook of expanded offerings, talent investment, and greater geographic reach.”

“Avid has long been a trusted provider of biopharmaceutical development and manufacturing services, and we have tremendous respect for its team’s expertise, its broad spectrum of customized services, and its strong regulatory track record. We look forward to leveraging our deep industry experience, focused strategy, and collaborative approach to drive growth,” said,David Anderson, general partner ofAmpersand, in the release.

Avid offers a range of biopharmaceutical process development, current good manufacturing practice (CGMP) clinical, and commercial manufacturing services, which include CGMP clinical and commercial drug substance manufacturing, bulk packaging, release and stability testing, and regulatory submissions support. It also provides process development activities for early stage programs, such as cell line development, upstream and downstream development and optimization, analytical methods development, testing, and characterization.

1. Avid Bioservices. Avid Bioservices to be Acquired by GHO Capital Partners and Ampersand Capital Partners in $1.1 Billion Transaction. Press Release. Nov. 6, 2024.

The merger agreement is valued at approximately $1.1 billion.

Avid Bioservices Acquired by GHO Capital Partners and Ampersand Capital Partners

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FDA’s Patrizia Cavazzoni, director, Center for Drug Evaluation and Research (CDER), and Peter Marks, director, Center for Biologics Evaluation and Research (CBER), provided an update on the agency’s 

 (the Hub) in a Nov. 1, 2024 FDA Voices blog post (1). The Hub was created in July 2024 (2) as a cross-center program that operates as a single point of engagement with stakeholders in the development of treatments for rare diseases. The Hub is the agency’s way of creating a community that can share new approaches for drug development for rare diseases.

In establishing the Hub, FDA gathered feedback from stakeholders through a public meeting, held on Oct. 16, 2024 in collaboration with the Reagan-Udall Foundation. The meeting included advocates from rare disease patient groups, academics, regulators, and other stakeholders who discussed how the Hub can work with the community to prioritize work.

A Hub director was also appointed. Amy Comstock Rick will be the Director of Strategic Coalitions for the Hub and be accountable to both Cavazzoni and Marks. Previously, Ms. Rick worked as a consultant at Leavitt Partners and was president and CEO of the Food & Drug Law Institute.

“Ms. Rick brings to this new role more than 20 years of experience as a leader and trusted ally in advocating for policies throughout healthcare, biomedical research, and nonprofit communities, which includes extensive engagement with patient communities and government agencies on issues related to rare diseases. We are excited to have her join and begin creating the important partnerships needed to strengthen the Hub’s engagement throughout the rare disease community,” stated Cavazzoni and Marks in the blog post.

As part of their work with the Hub, Marks and Cavazzoni plan on collaborating with other FDA centers that work with rare diseases, such as the Center for Devices and Radiological Health, the Oncology Center of Excellence, and the Office of Orphan Products Development. In addition, a Rare Disease Innovation Hub Steering Committee has been established to bring the groups together, and a new CBER-CDER Rare Disease Policy and Portfolio Council has been created. The council will bring experts from the different FDA centers together to discuss scientific, regulatory, and policy issues.

“The collaborative model of the Hub will build upon the knowledge and skills of staff within their respective centers, promote adoption of best practices, and avoid confusion over decision-making authorities within those organizations. Meetings between the FDA and sponsors relating to specific drug or biological products will remain the same, as will the way decisions are made about specific programs and how advisory committee meetings are structured and run,” Cavazzoni and Marks stated. “By working together, we believe that we can further facilitate the development, evaluation, and availability of safe and effective therapies. We are committed to ongoing and open communication and look forward to continuing this dialogue with the rare disease community.”

Cavazzoni, P. and Marks, P.FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub. 

https://www.fda.gov/news-events/fda-voices/fda-takes-exciting-steps-toward-establishing-rare-disease-innovation-hub

FDA. FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients.

CDER Director Patrizia Cavazzoni and CBER Director Peter Marks provided an update on the rare disease innovation hub in a new FDA Voices blog post.

FDA Rare Disease Innovation Moves Forward

Abzena, an end-to-end contract development and manufacturing organization, is expanding its quality control testing capabilities at its San Diego, Calif., biologics development and current good manufacturing practice site, the company announced on Oct. 30, 2024. The expansion is part of the company’s strategy to meet market demand for quality testing. According to Abzena, the additional laboratory space will enhance its existing analytical toolkit for antibodies with microbiology release testing. The expansion will also improve scalability with advanced materials separation.

The standalone microbiology lab offers separate product testing from other areas of the facility, in alignment with industry and regulatory standards. Product microbiological testing is segregated from utility and environmental microbiological testing in order to minimize cross-contamination. Production capacity is optimized by the separation of high volumes of clinical materials, which results in yields up to 99%, according to the company. “This design methodology offers superior operational efficiency and improved scalability while minimizing overall ecological impact,” the company stated in a press release (1).

“This announcement comes at a very timely moment with World Quality Month quickly approaching. As an organization focused on quality excellence, increasing our QC [quality control] footprint is a testament to our commitment to continuous improvement and innovation,” said Troy Wright, senior vice-president and global head of Quality at Abzena, in the release. “This expansion provides even greater sample integrity in the management of our microbiological testing capabilities. We are now positioned to deliver faster results and better support for our customer network, ultimately providing quality and compliant products to patients around the world.”

In other Abzena news, the company announced on Sept. 26, 2024 that its mammalian cell line development platform, AbZelectPRO, reduces timelines by more than 30% and improves overall protein production levels. The platform can bridge from DNA to research cell bank in 10 weeks and produces up to 8 g/L of high-performing titres before process development, according to the company (2).

Abzena recently attended CPHI Milan in October 2024, and 

caught up with Nicolas Camper, senior director—Bioconjugation Chemistry at Abzena, to talk about advancements in conjugation and linker technologies in antibody-drug conjugate (ADC) development and manufacturing (3).

“There have been a lot of new developments in terms of conjugation and linker technologies. There is a definitive move towards site-specific conjugation technologies and away from stochastic conjugation approaches that allows [us] to prepare better-defined antibiotic-[drug] conjugates,” Camper said in the interview.

Another new development that has been observed over the past few years is that linkers are becoming more sophisticated. “Quite often, when you conjugate cytotoxic drugs, they are quite hydrophobic, and that can impact the performance of your ADC. So, there has been a lot of effort put into developing more hydrophilic linkers,” Camper noted.

Abzena. Abzena Announces Major Expansion in Microbiology Laboratory Space in Suppor of Biologic Manufacturing. Press Release. Oct. 30, 2024.

Abzena. AbZelectPRO Enables Biopharma Customers to Reach their Research Cell Bank in 10 weeks with 8g/L. Press Release. Sept. 26, 2024.

Mirasol, F. CPHI Milan 2024: New Developments in ADC Technologies. 

The company will be expanding its quality control testing capabilities at its San Diego, Calif. biologics development and manufacturing site.

Abzena Expands Microbiology Laboratory Space

The World Health Organization (WHO) announced on Oct. 29, 2024 that it has activated the Global Health Emergency Corp (GHEC) in collaboration with Member States and other partners in response to mpox outbreaks (1). This is the first time GHEC, which was established by WHO in 2023 as a response to COVID-19, has been activated to support countries dealing with mpox. Leaders from affected and other countries met on October 22 as part of the GHEC activation to discuss effect control measures, best practices, and the coordination of efforts.

Mpox was declared as a public health emergency by WHO in August of 2024 (2). “The emergence of a new clade of mpox, its rapid spread in eastern DRC [Democratic Republic of the Congo], and the reporting of cases in several neighboring countries are very worrying. On top of outbreaks of other mpox clades in DRC and other countries in Africa, it’s clear that a coordinated international response is needed to stop these outbreaks and save lives,” WHO Director-General Tedros Adhanom Ghebreyesus, PhD, said at the time of the announcement (2).

“WHO and partners are supporting the government of the Democratic Republic of the Congo and other countries to implement an integrated approach to case detection, contact tracing, targeted vaccination, clinical and home care, infection prevention and control, community engagement and mobilization, and specialized logistical support,” said Mike Ryan, MD, executive director of WHO’s Health Emergencies Programme, in the October 29 press release (1). “The GHEC enhances the ability of the many effective responders at national and regional levels to collaborate and ensure the success on the ground in interrupting transmission and reducing suffering.”

GHEC’s objective is to strengthen the response to health emergencies and provide a platform in which countries and health networks can collaborate the response, including deployment of experts and leaders. GHEC is assessing workforce capacities in eight countries including DRC and Burundi, which have been the most impacted by the mpox outbreak. There have been 22 areas identified by GHEC that are in need of epidemiology, surveillance, laboratory capacity, infection prevention, risk communication, and community engagement. WHO has deployed 55 experts to the impacted countries, as of October 17, that include WHO staff through the Global Outbreak Alert and Response Network (GOARN) and the African Volunteers Health Corps. GOARN is also mapping partner support on a bilateral basis to impacted countries and the coordination structure in the region, including providing experts, supplies, financial support, and strengthening capacity.

“By mobilizing trained professionals from within the continent, we ensure that responses are not only timely but also contextually relevant,” said Abdou Salam Gueye, MD, PhD, regional emergency director for the WHO Regional Office for Africa, in the press release (1). “The dedication and expertise of these responders are essential in saving lives and building resilient health systems capable of withstanding future threats.”

In other mpox news, a report based on outcomes of a workshop held on Oct. 2, 2024 by the International Coalition of Medicines Regulatory Authorities was announced by the European Medicines Agency on Oct. 25, 2024. The current epidemiological status and the availability of mpox vaccines in African countries was discussed during the workshop. Discussion at the workshop was focused on the prioritization of treatments for children because children are the most vulnerable group in the DRC and have the highest fatality ratio (3).

WHO. WHO and Partners Activate Global Health Emergency Corps for the First Time in Response to Mpox Outbreak. Press Release. Oct. 29, 2024.

WHO. WHO Director-General Declares Mpox Outbreak a Public Health Emergency of International Concern. Press Release. Aug. 14, 2024.

EMA. Fostering Regulatory Collaboration to Improve Access to Mpox Medicines. Press Release. Oct. 25, 2024.

WHO and its partners are providing support to countries dealing with mpox outbreaks.