Behind the Headlines Episode 27

Ask the Expert

Small Molecules, APIs, and Excipients—Trends, Challenges, and Opportunities

Drug Digest September 2025

Artificial intelligence, machine learning, and novel instruments are helping drug developers.

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

J.P. Morgan Report Lists Biologics, Obesity, China

Top Trends Reshaping Pharma R&D

TOP NEWS on grunge world map | Image Credit: © Sean K - stock.adobe.com

On Oct 8, 2023, Bristol Myers Squibb and Mirati Therapeutics announced that Bristol Myers Squibb has acquired Mirati for a total equity value of $4.8 billion. Mirati stockholders will receive one non-tradable Contingent Value Right for each Mirati share held, possibly representing another $1 billion. Both boards unanimously agreed on the merger.

Mirati is a commercial-stage targeted oncology company designed to create breakthrough therapies for cancer patients, making it a good fit with Bristol Myers Squibb’s oncology portfolio. One of Mirati’s most attractive assets, KRAZATI (adagrasib),is a lung cancer medicine that Bristol Myers Squibb will now add to its commercial portfolio. Adagrasib was given accelerated approval by FDA for the treatment of KRAS-mutated Non-Small Lung Cancer (NSCLC) in patients who have gone through one prior systemic therapy.

Other assets include MRTX1719, and a leading KRAS and KRAS enabling program. MRTX1719 is “a potential first-in-class MTA-cooperative PRMT5 inhibitor” currently in phase one development. It targets MTAP-deleted tumors, which make up around 10% of all cancers. The second phase of development is planned to start in the first half of 2024. The KRAS and KRAS enabling program include MRTX1133 and MRTX0902. MRTX113 targets the KRAS mutation, which contributes to key tumor types, and is implicated in over 30% of pancreatic cancer patients. MRTX0902 is a phase one SOS1 inhibitor that may have use in combination with other agents targeting the MAPK/RAS pathway, including Adagrasib.

“Mirati strengthens and complements our current portfolio by adding assets focused on intrinsic tumor targets in the MTAP and MAPK pathways,” said Samit Hirawat, M.D., Bristol Myers Squibb’s chief medical officer and head of Global Drug Development, in a press release. “We believe Mirati’s assets have the potential to change the standard of care in multiple cancers, both as standalone therapies and in combination with Bristol Myers Squibb’s existing pipeline. We are excited about the significant potential that this transaction creates to transform patients’ lives through science around the world.”

Articles

Mirati’s focus on breakthrough cancer treatments will bolster Bristol Myers Squibb’s oncology portfolio.

Bristol Myers Squibb Acquires Mirati Therapeutics for up to $5.8 Billion

eXmoor pharma, the full-service cell and gene therapy (CGT) manufacturing partner, launched its new Cell and Gene Therapy Centre good manufacturing practice (GMP) facility on Oct. 11, 2023 to support the production of multiple therapies for clinical trials. According to the press release, the 65,000-square-foot facility is designed with the capacity and capability to help scale the next generation of CGTs for human trials and beyond. It is also the final step in eXmoor’s expansion into a full-service CGT contract development and manufacturing organization (CDMO).

“The opening of this facility marks a landmark achievement for us, as we bring our 19 years of translation consulting and process development experience to directly enable the rapid development of life changing therapies in the UK [United Kingdom] and beyond,” said Angela Osborne, CEO of eXmoor, in a press release. “To date, we have completed more than 500 projects, including the design of 37 CGT manufacturing facilities. Our Cell and Gene Therapy Centre represents the culmination of all that experience.”

The new facility based in Bristol was constructed in 14 months to fully expedite more GMP manufacturing facilities in the CGT space, according to the release. The new Cell and Gene Therapy Center is specialized in labs for clients’ process and analytical development with the capability for technology transfer to GMP production on the same site.

Four large, Grade C processing modules with airlocks

Process-agnostic design for both cell and gene therapy manufacturing

Complete segregation between processing modules

Up to two-times 200L bioreactor scale for viral vector or allogeneic cell therapy manufacture or space for up to five autologous cell therapy stations

Fully serviced with all GMP gas supplies for any cell/gene therapy process

Uninterruptable power supply and emergency generator for electrical back up

Expansion space of 12,000 sq ft for another nine suites to grow with eXmoor’s clients into scaled-up and -out manufacturing for pivotal trials and in-market supply

6500 sq ft for process development, quality control, and analytical labs, with ‘same again’ expansion potential for additional manufacturing and commercialization partnerships.

The facility has also been designed with no fossil fuel usage on site, relying instead on solar technology and heat pumps, according to the press release.

The 65,000-square-foot facility is designed with the capacity and capability to help scale the next generation of CGTs for human trials and beyond. 

eXmoor pharma Expands into Full-Service Cell and Gene Therapy CDMO Partner with Opening of GMP Facilities 

Feliza Mirasol is the science editor for 

Sweden-based Salipro Biotech and Estonia-based Icosagen, a contract research, development, and manufacturing organization, announced on Oct. 10, 2023 that they have entered into a multi-target antibody research agreement to advance drug discovery programs. The companies intend to discover and characterize monoclonal antibodies (mAbs) against several challenging membrane proteins, including G protein-coupled receptors (GPCRs) and solute carrier (SLC) transporters.

Under the agreement, Icosagen will leverage its expertise in protein production and analytics. The company’s knowledge in antibody discovery encompasses high-throughput antibody screening and a variety of lead optimization techniques that the company expects will support the collaboration and form a solid basis for future biologic drug development, according to a company press release.

The partnership between the companies aims to build on Salipro's strength in establishing multiple collaboration and licensing agreements with leading pharma and biotech companies that enable drug discovery against complex membrane protein targets. The company’s experience includes small-molecule drug discovery programs as well as structural biology and antibody discovery.

“Our Salipro technology provides an enabling platform for the discovery of therapeutics against challenging drug targets. We are excited to unlock the potential of these targets with Icosagen and develop novel drugs that can improve patient outcomes," said Jens Frauenfeld, CEO of Salipro Biotech, in the press release.

"We are delighted to apply our QMCF technology and protein analytics capabilities in this project. Both teams in Icosagen and Salipro Biotech are always finding ways to push the boundaries in novel technologies of biologics development. It will be interesting to see what we’ll be able to achieve as we put our heads together for this project,” said Mart Ustav Jr., chief scientific officer, Icosagen, in the release.

Salipro Biotech and Icosagen have entered into a multi-target collaboration to discover and characterize monoclonal antibodies.

Salipro Biotech and Icosagen to Collaborate on Antibody Discovery

Felicity Thomas is the former Associate Editorial Director of Pharmaceutical Technology, Pharmaceutical Technology Europe, BioPharm International.

Science laboratory test tubes, laboratory equipment | Image Credit: © BillionPhotos.com - © BillionPhotos.com - stock.adobe.com

Global biopharma company, GSK, has entered into an exclusive strategic partnership with Chongqing Zhifei Biological Products Co. (Zhifei)—a fully-integrated biotechnology company—to co-promote GSK’s shingles vaccine, Shingrix, to healthcare professionals and points of vaccination in China.

According to an Oct. 9, 2023 press release, the partnership, which has been set to last for an initial three-year period with the option to extend in the future, will broaden the availability of Shingrix in China significantly. Additionally, the partnership is expected to support future co-development and commercialization in China of GSK’s respiratory syncytial virus (RSV) vaccine, Arexvy, for adults aged 60 years and older.

The agreement is set to begin on Jan. 1, 2024 and will see Zhifei gain the exclusive rights for the importation and distribution of Shingrix within China, while GSK will co-promote the vaccine in the country through raising awareness of the importance of shingles vaccination for healthcare professionals. Under the terms of the agreement, Zhifei will buy agreed upon volumes of Shingrix that will equate to a total value of £2.5 billion ($3.1 billion) to GSK over the course of three years. The volumes of the vaccine purchased will be phased over time to accommodate the expected increase in demand as the vaccine and its importance are promoted. Further, as a part of the terms of the partnership, Zhifei has given GSK the right of first refusal to be the exclusive partner for a similar arrangement for GSK’s Arexvy vaccine once it has been approved in China.

“This partnership is consistent with our focus on products with a high and durable level of differentiation,” said Luke Miels, chief commercial officer, GSK, in the press release. “It materially expands the number of Chinese adults who can benefit from Shingrix and includes the option to extend the collaboration to include our novel RSV vaccine Arexvy.”

The companies will co-promote GSK’s shingles vaccine, Shingrix, to healthcare professionals and points of vaccination in China. 

GSK and Zhifei Partner to Extend the Availability of Shingrix

Text sign showing Industry News. Business photo text delivering news to the general public or a target public | Image Credit: © Artur - © Artur - stock.adobe.com

FDA published draft guidance on Oct. 11, 2023, 

Quality Considerations for Topical Ophthalmic Drug Products

, that discusses quality considerations for topical ophthalmic drug products used in and around the eye. These types of products include solutions, suspensions, emulsions, gels, ointments, and creams.

The document specifically outlines approaches for evaluating visible particulate matter, extractables and leachables, and impurities and degradation products. It also discusses 

 drug release/dissolution testing for certain ophthalmic dosage forms. FDA also offers recommendations for design, delivery, and dispensing features of container closure systems (CCSs) and stability studies.

Regarding visible particulate matter, the guidance states, “For topical ophthalmic drug products packaged in opaque containers, appropriate technologies (e.g., X-ray spectroscopy) or destructive testing should be used to identify particulates within the accepted visible size range.”

In discussing extractables and leachables, the document states, “Leachables have the potential to interact with the formulated drug product, which could compromise product quality and therapeutic effect. The assessment of extractables and leachables should consider the primary, secondary, and tertiary packaging components of the CCS, including the labeling components.”

 drug release and dissolution testing, FDA states, “One approach that applicants can consider as part of the quality control strategy for certain ophthalmic dosage forms (e.g., suspensions, emulsions, semi-solids) is the use of 

 drug release/dissolution testing. Other approaches are also acceptable, such as using one or more critical quality attributes (CQAs) that are sensitive to the formulation and process variants. The applicant should provide scientific justification for how the control strategy will ensure consistent product quality.”

In addition, the guidance document provides information about United States Pharmacopeia criteria and recommendations for documentation of chemistry, manufacturing, and controls information for new drug applications, abbreviated new drug applications, and biologics license applications. However, the guidance does not apply to biological products regulated by the Center for Biologics Evaluation and Research.

The draft guidance document discusses quality considerations for topical ophthalmic drug products.

FDA Issues Guidance for Topical Ophthalmic Drugs

Humberto Vega, PhD, Executive Director, Bristol Myers Squibb, discusses the journey from clinical to commercial cell therapies as a part of his session, "Successfully Navigating Global Cell Therapy Requirements."

 The case of cell therapies is one word data is now being generated. And the different companies are learning about processes and products, as they are in development. It is not a new journey, it began back in the 70s and even earlier, but the learning about sales and the immune system really grew during the last 20 years with the advantage of technologies and that is one challenge that is being overcome. Now, the regulatory space and the compliance space is something that is now being developed, is growing, and each country has various specific requirements, looking not only on having a product that is safe, but also protecting the patient from the use of the cells and how those products are now develop use, and in the future, they will become the mainstream. So, the challenge is making sure that all the regulatory aspects are properly addressed during clinical stage and development all the way into the routine commercial promotion.


 One key element is understanding what is expected from the regulatory space, having a close communication with the agency, having clear, a change of information with those regulatory bodies to ensure their expectations, their regulatory space, is very clear to the company and the group developing and transitioning into a commercial space in particular products. So, there are no surprises at the time, the product is fine, and all the information required by the regulatory group is provided and explain and justify, and then once approved, how to maintain the quality and the regulatory posture as a production is now in a commercial stage.

Videos

The Challenges of the Journey from Clinical to Commercial Cell Therapies

FDA announced on Oct. 11, 2023 that it has established the Digital Health Advisory Committee to advise the agency on the scientific and technical issues surrounding digital health technologies (DHTs) such as artificial intelligence (AI), machine learning (ML), augmented reality, virtual reality, digital therapeutics, wearables, and remote patient monitoring. The committee, which expects to be fully operational in 2024, will provide expertise and perspective on FDA’s understanding of the benefits and challenges of DHTs.

The development of the committee is in response to the rapidly evolving DHT space that covers a range of technologies. These technologies also include decentralized trials, patient-generated health data, and cybersecurity, according to FDA.

“As one of our strategic priorities, our goal is to advance health equity in part through expanding access by bringing prevention, wellness, and healthcare to all people where they live—at home, at work, in big cities, and rural communities,” said Jeff Shuren, director of FDA’s Center for Devices and Radiological Health, in a press release. “Digital health technologies are critical for achieving this transformation in care delivery. As digital health technologies advance, [FDA] must capitalize on knowledge from inside and outside of the agency to help ensure we appropriately apply our regulatory authority in a way that protects patient health while continuing to support innovation.”

“Technology moves at an incredible pace, and we’re excited to have a committee of experts throughout the field who can help ensure our regulation of these exciting tools maintains an appropriate pace while working within parameters of safety and effectiveness standards,” said Troy Tazbaz, director of FDA’s Digital Health Center of Excellence. “Many of these technologies are novel and tend to rapidly change; it’s our duty to seek as much knowledge on them as possible as we determine and implement appropriate regulation to encourage innovation while protecting public health.”

There will be nine core voting members on the committee including the committee chair. FDA is requesting that those interested in serving on the committee submit nominations electronically to the 

FDA Advisory Committee Membership Nomination Portal 

The new Digital Health Advisory Committee will advise the agency on issues related to technologies such as AI, virtual reality, and machine learning.

FDA Creates Advisory Committee on Digital Health Technologies

BIOVECTRA and Acuitas Therapeutics have entered into a technology transfer service agreement, through which Acuitas’ lipid nanoparticle (LNP) technology platform for manufacturing messenger RNA (mRNA)-based therapeutics will be transferred to BIOVECTRA’s new biomanufacturing center currently being built at its Charlottetown, Prince Edward Island site in Canada.

Under the agreement, BIOVECTRA will formulate Acuitas’ LNP for licensed therapeutic partners for clinical and commercial products using a variety of payloads. Acuitas’ LNP technology is currently used in multiple vaccines and therapeutics in clinical development. The technology is also used in the Pfizer-BioNTech COVID-19 vaccine, COMIRNATY, according to an Oct. 5, 2023 company press release.

The tech transfer will occur in BIOVECTRA’s biomanufacturing center in Charlottetown, which is expected to be completed in late 2023. Upon completion, the facility will support the good manufacturing practice manufacturing of plasmid DNA, mRNA, and LNP formulations. The 36,000 additional ft2 will be able to produce the equivalent of 160 million doses of mRNA vaccines and will have the capability to fill/finish 70 million final vaccine doses per year for commercial distribution, according to the press release.

“We are thrilled about this partnership with Acuitas Therapeutics … ,” said Marc Sauer, chief science officer, BIOVECTRA, in the press release. “Our work together will provide their licensed partners with faster market access to CGMP [current good manufacturing practice]-quality LNPs for early and late-stage products. BIOVECTRA looks forward to continuing to make a difference in patients’ lives by supporting GMP and commercial-ready LNP formulation at our new mRNA biomanufacturing facility in Atlantic Canada.”

“LNP-based mRNA medicines provide the opportunity to address an incredibly wide range of health conditions, from infectious diseases such as COVID-19, flu, and malaria to therapeutic areas related to heart disease, or cancer, as well as many rare diseases,” said Chris Barbosa, vice-president, Technology Development, Acuitas Therapeutics, in the release. “As we move forward in the development process in all of these areas with our partners, the demand for Acuitas’ clinical-grade LNP for our partners increases exponentially. We look forward to working with the BIOVECTRA team to expand the CGMP manufacturing capacity for Acuitas’ LNP so that we can continue to meet the growing needs of our global partners.”

Under this agreement, Acuitas Therapeutics’ LNP technology platform will be transferred to BIOVECTRA for use in manufacturing mRNA-based therapies.

BIOVECTRA in Tech Transfer Agreement with Acuitas Therapeutics for LNP Delivery System

Gritgen Therapeutics, a biotechnology company focused on gene therapy products, announced the successful validation and official launch of operations at its commercial good manufacturing practice (GMP) facility located in Suzhou Industrial Park. According to the press release, the milestone sets the stage for the development of Gritgen’s Phase III clinical and commercial product for hemophilia A (HA). Further, the construction of Gritgen’s GMP facility strictly adheres to the global current GMP (CGMP) standards and complies with the regulatory guidelines of authoritative agencies including the National Medical Products Administration (NMPA), FDA, and the European Medicines Agency (EMA).

The new facility is 8600 square meters and covers plasmid production and AAV production lines. In addition, the production lines are based on a mature and reliable upstream process with mammalian cell suspension, and a downstream process with chromatography platform technology, equipped with disposable bioreactors of different scales (the largest scale reaches 1000 liters).

According to the press release, the facility is equipped with pilot filling lines and commercial fill/finish lines to meet the need of drug product manufacture, and has a quality control laboratory covering biochemical, physicochemical, molecular biological, and microbiological tests. The production lines in the facility also are completely physically separated, which comprehensively controls the risk of cross-contamination and is a solid foundation for the global supply chain guarantee of innovative gene therapy products.

“The validation and operational launch of our GMP facility represents a pivotal juncture for Gritgen, which provides an important vector foundation for the company to realize commercialization,” said Dr. Wu Fenglan, co-founder and CEO of Gritgen, in a press release. “At the present, a bottleneck of gene therapy is the manufacturing process, a challenge resonating globally. The production capacity of large-scale, high-quality, cost controllable vectors is an important driving force for the development of gene therapy industry."

Further, the construction of Gritgen’s GMP facility strictly adheres to the global current GMP (CGMP) standards and complies with the regulatory guidelines of authoritative agencies including the National Medical Products Administration (NMPA), FDA, and the European Medicines Agency (EMA).

Gritgen Therapeutics Launches Commercial GMP Facility in China

Roche has entered into an agreement with Ionis Pharmaceuticals for two undisclosed early stage programs for RNA-targeting investigational therapeutics for treating Alzheimer's disease (AD) and Huntington's disease (HD). According to a Sept. 27, 2023 company press release, Roche gains exclusive worldwide rights and will be responsible for clinical development, manufacturing, and commercialization of the candidates worldwide, the latter of which is dependent upon regulatory approval.

Under the agreement, Ionis' expertise in discovering medicines that target the root cause of central nervous system diseases will work in tandem with Roche's global experience developing and commercializing therapies for nervous system disorders. Ionis will receive a $60 million upfront payment and will be eligible for development, regulatory, and commercial milestone payments as well as tiered royalties. Ionis will be responsible for advancing the two programs through pre-clinical studies, after which Roche will take over. Roche will have sole responsibility for clinical development and, if approved, commercialization worldwide.

This agreement builds upon a 10-year-long partnership between the companies, which started in 2013. In 2018, the companies entered another collaboration to develop an Ionis therapeutic candidate for immunoglobulin A nephropathy and geographic atrophy treatment.

"We are excited to expand our partnership with Roche … ," said Brett P. Monia, CEO, Ionis, in the company press release. "With this new collaboration, we are joining forces to accelerate the discovery and development of novel therapies for people living with Alzheimer's disease and Huntington's disease globally. Collaborating on these two programs enables Ionis to advance our wholly owned programs, including those in neurology, aligned with our strategic priorities."

"Our lasting partnership with Ionis … is a great example of two collaborators mutually benefiting from their relationship by complementing and learning from each other," said James Sabry, global head of Roche Pharma partnering, in the press release. "By expanding our alliance, we bring together the companies' combined knowledge of the science in Alzheimer's disease and Huntington's disease with Roche's proven capabilities in the development and commercialization of innovative treatments in neuroscience."

Roche will gain exclusive worldwide rights to develop, manufacture, and commercialize Ionis’ investigational RNA-based therapeutic candidates for Alzheimer's and Huntington's disease.

Featured Content

Behind the Headlines Episode 27

Ask the Expert

Drug Digest September 2025

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

Top Trends Reshaping Pharma R&D