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

Chris Spivey is director of Industry Relations and Strategic Partnership, MJH Life Sciences.

E27 THumbnail.jpg

In episode 27, Christopher Hopkins, PhD; Omkar Kawalekar, PhD; Barnaby Pickering; and Jerry Rosenbaum, MD, go behind the headlines.

Behind the Headlines Episode 27: Unpacking Mega Deals and R&D Directions

Behind the Headlines bi-weekly episodes examine the ongoing trends, factors, and fashions that cause pharmaceutical news to happen. Panelists are a mix of consultants, venture capitalists, scientists, patient advocates, journalists, and editors. Each episode talks about the current headlines of the preceding weeks that are on everyone’s lips but seeks to highlight the more enduring lessons that hide behind these headlines.

, director, Center for the Neuroscience of Psychedelics, Massachusetts General Hospital, and co-founder, Psy Therapeutics and Entheos Labs—take a 30,000 foot look at multiple recent multi-billion dollar deals. On October 15, 2025, J.P. Morgan released its Q3 2025 report showing biopharma licensing valued at $63.7 billion, while M&A transactions reached $30.8 billion (1).

Our panel singled out Novo Nordisk buying Akero Therapeutics for up to $5.2B (2), AstraZeneca’s construction of a $4.5B API plant in Virginia (3), the Bristol Myers Squib (BMS) $5B Orbital buyout that extended the 

 cell therapy deal streak (including late-breaking news from Kite Pharma, a part of Gilead, partnering with Chinese company Pregene in a deal worth up to $1.2B) (4). Of note, the orbital acquisition provides BMS an experimental autoimmune disease treatment that could begin clinical testing in 2026.

All this, however, while both Novo and Takeda withdrawal from cell therapy R&D, despite myriad news like Regeneron’s gene therapy restores hearing in children with OTOF genetic hearing loss (5). Behind these headlines, the panel discusses to what direction all this activity leads.

Of particular note, we examine progress in central nervous system therapies, especially the chief opportunities and challenges of psychedelics, as anchored by the 50-year informed perspective of Dr. Rosenbaum. The panel also covered the relative strengths of several life science clusters, especially in Northern Europe.

Biopharma, Medtech Activity Diverges in Q3 2025

Akero Therapeutics to be Acquired by Novo Nordisk for up to $5.2 Billion

AstraZeneca Breaks Ground at $4.5 Billion Virginia Plant as Drugmakers Court Trump

Gilead's Kite Signs $1.64B In Vivo Cell Therapy Pact With Pregene Biopharma

Latest DB-OTO Results Demonstrate Clinically Meaningful Hearing Improvements in Nearly All Children with Profound Genetic Hearing Loss in CHORD Trial

Videos

Industry collaboration remains critical to addressing the challenges of modern drug manufacturing, regulation, and global supply resilience as bio/pharmaceutical development increasingly becomes complex. Informa Markets' announced agenda and speaker lineup for 

 reflects this need as the company launches a new Leadership Summit designed to unite decision-makers from across the global bio/pharmaceutical value chain (1).

Taking place Oct. 28–30, 2025, at Messe Frankfurt, the event will feature more than 260 expert speakers from regulatory bodies, biotech startups, manufacturing organizations, and supply chain leaders. The program will focus on the future of drug development, artificial intelligence (AI), digital transformation, and sustainability—key themes shaping how bio/pharmaceutical technologies are developed, scaled, and delivered worldwide (1–4).

“The 2025 event reflects not only the scale of innovation across pharma but also the sector’s need for connected leadership and diverse perspectives,” said Tara Dougal, event director for Informa Markets, in a press release (1). “Our speaker line-up represents a balance of experience and forward thinking, tackling the core challenges and opportunities facing pharma today.”

What key industry issues will the leadership summit address?

The Leadership Summit, sponsored by Ensera (formerly SteriPack), is a major addition to this year’s event. The summit will serve as a strategic forum for executive dialogue on topics such as pharmaceutical resilience, mergers and acquisitions, access to medicines, and the impact of geopolitical shifts on global supply chains.

These discussions are expected to resonate across the bio/pharmaceutical industry, for which managing risk and maintaining manufacturing continuity are central concerns. By emphasizing long-term strategy and policy alignment, the summit aims to bridge the gap between regulatory goals, commercial strategy, and the operational realities of global drug production.

What hot topics will the specialized tracks explore?

The conference’s seven content tracks will explore specialized areas critical to the evolution of pharmaceutical manufacturing and development and will include:

Packaging and Device Innovation (Hall 8.0): Patient-centric packaging, connected devices, and regulatory strategies for combination products

Future of Pharma and Ingredients (Hall 8.0): Advances in API design, digital chemistry, and modernization of regulatory pathways

Next-Gen Bio (Hall 4.1): Challenges and solutions in scaling up biologics, cell and gene therapies, and messenger RNA platforms

Clinical Innovation (Hall 6.1): Use of AI, digital endpoints, and decentralized trial models to improve patient recruitment and data reliability

Manufacturing 5.0 (Hall 4.1): Smart factories, machine learning integration, and operational excellence in production

Sustainable Futures (Hall 4.0): Strategies for low-emission manufacturing, circular pharma, and responsible sourcing

Spotlight Stage (Hall 4.0): Focused updates on cleanroom technologies and sustainability in production.

According to Informa, each track was developed in collaboration with global partners, including IQVIA, Medicines for Europe, CEPI, Environmental Financial Consulting Group, and PSCI. The collaboration aims to ensure that the content reflects the latest data-driven insights and industry priorities (1).

The diversity of speakers includes subject matter experts from government, academia, and multinational biopharma enterprises and underscores CPHI Frankfurt’s role as a global forum for advancing bio/pharmaceutical technology. Through technical sessions and leadership discussions, the event aims to shape the dialogue around efficiency, sustainability, and digitalization across the full drug development lifecycle (1).

CPHI Frankfurt 2025 Unveils Dynamic Speaker Line-Up and Launches New Leadership Summit

. Press Release. Oct. 21, 2025.
2. Fu, C.; Chen, Q. The Future of Pharmaceuticals: Artificial Intelligence in Drug Discovery and Development. 


3. Mustoe, C. L.; Turner, A. J.; Urwin, S. J.; et al. Quality by Digital Design to Accelerate Sustainable Medicines Development. 

The Rise of AI in Biopharmaceuticals: Uncovering the Environmental Cost and the Promise of Sustainable AI

Articles

The upcoming CPHI Frankfurt 2025 event will address the complexities of modern drug manufacturing, regulation, and global supply resilience through a new Leadership Summit. This summit will unite decision-makers from the bio/pharmaceutical value chain to discuss key industry issues such as pharmaceutical resilience, mergers and acquisitions, and geopolitical impacts on supply chains. The event will feature over 260 expert speakers and seven specialized content tracks focusing on themes like AI, digital transformation, and sustainability. The goal is to align regulatory goals with commercial strategies and operational realities in global drug production.

Spotlighting global biopharma innovation, the event will unite leaders to discuss digitalization, sustainable manufacturing, and future-ready development.

CPHI Frankfurt 2025 to Showcase Advances in Biopharma Manufacturing, Digital Transformation, and Global Industry Collaboration

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

The first needle-free adrenaline treatment launches in the UK.

Nasal Spray Approval

ALK announced on Oct. 20, 2025, that its adrenaline nasal spray (EURneffy) is now available for prescription by healthcare professionals in the United Kingdom, making it the UK’s first needle-free emergency anaphylaxis treatment for adults and children weighing 30 kg (66 lbs.) or more (1).

a significant regulatory and formulation milestone,

 providing a portable, ready-to-use alternative to traditional adrenaline auto-injectors (AAIs) (1,2). 

 of this non-invasive product, which is also approved in the US (under the brand name Neffy), 

validates an innovative administration route

 for critical, acute care medication traditionally reliant exclusively on injection (3,4).

The robust clinical and real-world data supporting this intranasal epinephrine product underscore the growing importance of delivery system innovation and human factors engineering (2). Extensive pharmacological studies demonstrated that the spray achieves outcomes comparable to AAIs across various patient scenarios, including when significant nasal congestion is present (1). Furthermore, the first analysis of real-world treatment outcomes reinforces this effectiveness, showing that nearly 9 in 10 patients (89.2%) experiencing allergic reactions were successfully treated with a single dose, matching historic injection response rates (3).

These results are poised to influence development strategies across the pharmaceutical value chain by challenging established practices for acute emergency treatments and demanding greater focus on intuitive design and logistical resilience in drug manufacturing and distribution (2).

Is non-invasive delivery comparable to injection?

Pharmacological data from more than 700 participants and real-world evidence confirm rapid absorption via the nasal mucosa is comparable to intramuscular injection, validating the non-invasive route for life-threatening emergencies like anaphylaxis. This success sets a strong precedent for pursuing alternative delivery methods for other critical, fast-acting therapies.

How does delivery innovation improve adherence?

The needle-free, pocket-sized design directly addresses the practical and psychological barriers—such as needle anxiety and hesitation—that historically caused patients and caregivers to delay or avoid administering life-saving AAIs. Future development programs must integrate simple, intuitive design early in the process to overcome these adherence challenges and potentially improve outcomes.

The new formulation boasts a significantly extended shelf life (up to 30 months total) and requires no special storage conditions, tolerating temperature changes better than traditional AAIs. This breakthrough reduces reliance on the cold chain, simplifies distribution, and has the potential to minimize device waste from expiration, reducing the need for replacement devices by an estimated 32% per year.

1. ALK. EURneffy Needle-Free Nasal Adrenaline Spray for Emergency Anaphylaxis Treatment Now Available for Prescription in the UK. Press Release. Oct. 20, 2025.
2. Cole, C. 

UK Anaphylaxis Nasal Spray Approval Helps Reshape Emergency Allergy Treatment.

Epinephrine Nasal Spray Comparable to Injection in Real-World Data of Patients Experiencing Anaphylaxis.

First Nasal Adrenaline Spray Among EMA Approvals in June.

ALK's adrenaline nasal spray, EURneffy, is now available in the UK, marking the first needle-free emergency anaphylaxis treatment for individuals over 30 kg. This innovation offers a portable alternative to traditional adrenaline auto-injectors, with comparable efficacy demonstrated in clinical and real-world settings. The nasal spray's design addresses barriers like needle anxiety, potentially improving adherence. Its extended shelf life and lack of special storage requirements reduce cold chain dependency, simplifying distribution and minimizing waste. This development challenges traditional acute treatment methods and emphasizes delivery system innovation.

The first needle-free adrenaline treatment launches in the UK, validating non-invasive delivery with real-world data comparable to injection effectiveness.

How ALK's UK-Approved Nasal Spray Changes the R&D Model for Acute Emergency Treatment

Prescription medicine bottle and oral syringe for baby isolated on blue background | Image Credit: © nd700 - stock.adobe.com

 eBook, authors Laura Owens and Yeli Zhang of Roquette explore 

"Enhancing Patient-Centric Care Through Oral Liquid Drug Delivery Formulations."

The following 10 questions address key points in the article. See how much you learned from reading the piece, and click through at the end for the answers!

1. According to the article, what is a primary objective of patient-centric drug development?

A. To streamline the regulatory approval process by prioritizing formulations with the longest shelf life.

B. To improve treatment adherence and health outcomes by focusing on patient needs and preferences.

C. To increase the bioavailability of active pharmaceutical ingredients (APIs) above all other factors.

D. To reduce the overall cost of manufacturing by using more common excipients.

2. For which patient population are thickened liquid suspensions particularly beneficial as a safety measure?

A. Patients with chronic conditions requiring extended-release formulations.

B. Pediatric patients who are sensitive to the taste of medications.

C. Patients with dysphagia who are at risk of aspiration.

D. Geriatric patients experiencing polypharmacy.

3. In the development of an oral suspension, omitting a suspension aid like xanthan gum could most likely lead to what consequence?

A. Oxidative degradation of the active pharmaceutical ingredient.

B. Rapid microbial growth throughout the product's shelf life.

C. Inconsistent dosing due to precipitation or sedimentation of the API.

D. An unpleasantly sweet taste, leading to poor patient compliance.

4. What key rheological property, demonstrated by colloidal MCC in the case studies, allows a suspension to flow easily when poured but regain its structure during storage?

5. In Case Study 1, how did the stability of the paracetamol suspension formulated with NaCMC compare to the others after four weeks, as measured by the TURBISCAN stability index (TSI)?

A. It showed moderate stability, comparable to the xanthan gum formulations ranked 'C'.

B. It demonstrated moderately good stability with a rank of 'B', similar to the reconstitutable suspension.

C. It was the most stable formulation, with the lowest TSI and a rank of 'A+'.

D. It was not stable and received the lowest stability ranking of 'D'.

6. According to Case Study 2, what was the primary challenge when formulating an ibuprofen suspension with pre-made liquid colloidal MCC at a low pH (approximately 2.7)?

A. Poor taste that could not be masked by flavoring agents.

B. Degradation of the ibuprofen active ingredient.

C. A significant and unmanageable increase in viscosity.

D. Flocculation of the colloidal MCC, leading to instability.

7. In Case Study 3, what strategy was successfully employed to stabilize a pre-made liquid colloidal MCC suspension containing a high concentration of calcium chloride?

A. Adjusting the formulation to a much lower pH.

B. Increasing the concentration of colloidal MCC in the formulation.

C. Replacing the pre-made liquid colloidal MCC with NaCMC.

D. Adding a protective colloid, such as xanthan gum.

8. Which type of oral liquid drug delivery system is defined as a homogeneous mixture where the drug is completely dissolved in a solvent?

9. The article projects significant growth for the pharmaceutical liquid suspensions market, identifying a key demographic driver. What is this driver?

A. A projected increase in childhood diseases requiring long-term medication.

B. The near doubling of the world's population older than 60 by 2050.

C. Increased regulatory preference for liquid formulations over tablets.

D. A rise in demand for on-the-go medications for young adults.

10. What is the primary function of excipients categorized as stabilizers, such as sodium metabisulfite or sorbic acid?

A. To enhance the solubility of poorly water-soluble drugs.

B. To protect the formulation from chemical or microbial degradation.

C. To mask unpleasant tastes and improve the medication's palatability.

D. To maintain uniform distribution of insoluble particles.

Enhancing patient-centric care through oral liquid drug delivery formulations focuses on improving treatment adherence and health outcomes by addressing patient needs and preferences. Thickened liquid suspensions are particularly beneficial for patients with dysphagia to prevent aspiration. Omitting suspension aids like xanthan gum can lead to inconsistent dosing due to API sedimentation. Colloidal MCC demonstrates thixotropic behavior, allowing easy flow and structural recovery. The pharmaceutical liquid suspensions market is projected to grow significantly, driven by the increasing population over 60 years old.

Take this quiz to test your comprehension of one of our recent feature articles.

Test Your Knowledge: Patient-Centric Care Through Oral Liquid Delivery

Pharmaceutical Vibrating Sorting Machine Aligning Capsules for Blister Packaging | Image Credit: © guteksk7 - stock.adobe.com

CPHI Frankfurt is scheduled to convene from Oct. 28–30, 2025. As a key highlight in the industry’s annual calendar, this event serves as a crucial forum for the entire pharmaceutical supply chain, offering key insights into technological advancements designed to enhance process control, patient safety, and efficiency.

The industry is rapidly embracing solutions for complex challenges, including the need for greater automation in biopharmaceutical production, reliable material performance in continuous manufacturing environments, and stricter regulatory compliance. Concurrently, efforts to meet growing sustainability mandates are driving innovation in both packaging and formulation.

These pivotal developments highlight a trajectory toward greater scalability and integrity in modern drug production. The following is a compilation of recent company news detailing innovations and exhibits to be shown at the conference.

BENEO’s excipient supports continuous manufacturing

The pharmaceutical-grade isomalt excipient galenIQ™ will be presented as a compelling binder solution for continuous manufacturing, delivering robust granule formation and high-strength tablets while maintaining rapid disintegration (1). This non-synthetic, plant-based filler-binder also provides formulators with process control and consistency, while its pleasant taste enhances patient compliance for oral dosage forms.

Eckelmann enhances biopharma automation and scalability

Eckelmann is offering customized control systems and specialized artificial intelligence vision capabilities from a single source, aimed at accelerating biopharmaceutical production capacity and reducing operating costs (2). The utilization of digital twins for simulating test runs and commissioning saves time and money, enabling maximum plant availability and optimization of key performance indicator monitoring.

IMA advances aseptic filling and continuous OSD lines

IMA Life is introducing TILE-X, a fully gloveless solution for filling small-batch ready-to-use high-value products, such as cell and gene therapies, ensuring maximum sterility and operator safety compliant with Annex 1 good manufacturing practice (GMP) guidelines (3). Separately, IMA Active is cultivating expertise, including a partnership with CONTINUUS Pharmaceuticals, to advance continuous manufacturing lines for the compression and coating of oral solid dosage forms.

BD and partners debut unit-level syringe traceability

A joint demonstration will showcase an end-to-end workflow for unit-level traceability in injectable drug manufacturing using the BD iDFill RFID Integration Tour, tracking syringes from filling through assembly (4). This radio frequency identification-enabled system supports improved efficiency, deviation management, and adherence to increasingly strict regulatory expectations for product integrity and quality.

SÜDPACK medica unveils recyclable blister concepts

The PharmaGuard blister concept is a pioneering polypropylene-based mono-material solution that achieves high recyclability and a significantly lower climate impact compared with conventional multilayer structures. This innovation aligns with growing industry pressure for sustainable plastic film solutions while meeting necessary current GMP requirements for oral solid doses.

1. BENEO. CPHI 2025: BENEO to Present galenIQ As the Granulation Binder of Choice. Press Release. Sept. 24, 2025.
2. Eckelmann Group. 

From a Single Source: Hardware and Software for Efficiency in Biopharmaceutical Production Capacities.

 Press Release. Oct. 13, 2025.
3. IMA Group. 

IMA Pharma and IMA MED-TECH at CPHI Frankfurt 2025.

 Press Release. Oct. 16, 2025.
4. ten23 health. 

BD, ten23 health, and Partners to Demo Full RFID Syringe Traceability at CPHI Frankfurt.

 Press Release. Oct. 15, 2025.
5. SÜDPACK Medica. 

Sustainable Innovations and Valuable Insights: SÜDPACK Medica at CPHI.

CPHI Frankfurt 2025 will serve as a pivotal event for the pharmaceutical supply chain, focusing on technological advancements in process control, patient safety, and efficiency. The industry is addressing challenges like automation in biopharmaceutical production, material performance in continuous manufacturing, and regulatory compliance. Sustainability is driving innovation in packaging and formulation. Key innovations include BENEO's galenIQ™ excipient for continuous manufacturing, Eckelmann's biopharma automation solutions, IMA's aseptic filling advancements, BD's syringe traceability system, and SÜDPACK's recyclable blister concepts.

As the conference approaches, scientists are reviewing new technologies that enhance manufacturing process control, product integrity, and regulatory compliance through automation and sustainable materials.

CPHI Frankfurt to Show Innovations Driving Pharma Manufacturing Efficiency and Compliance

We highlight the week’s industry news in an easy-to-consume, fun format.

PharmTech Weekly News Roundup — Week of October 13, 2025

In this PharmTech video feature, we highlight the week’s industry news in an easy-to-consume, fun format. New roundups will drop every Friday, so be sure to come back each week.

Collectively, the week’s coverage discusses the pharmaceutical industry’s recent developments and current regulatory landscape. Read on for recaps of each story we’ve covered.

 selectively favoring later-stage Phase II programs, with median values rising to $63 million. R&D partnerships focused on obesity and diabetes treatments have reached record values totaling $18.2 billion in announced potential deal value this year.

The success of antibody-drug conjugates (ADCs) is catalyzing a transformation 

 which use radio ligands conjugated to an antibody for targeted systemic delivery. Development challenges persist in optimizing the chemistry for linking the radio ligand to the antibody and identifying the molecular cohorts most sensitive to this new therapy.

 for 75% of life sciences CEOs, with targeted funding focused on operational areas like intelligent manufacturing and agentic AI. While 85% of leaders cite workforce upskilling as critical, 66% also view ethical challenges as a barrier to AI implementation.

The global biotechnology equipment market is projected to 

 driven by increased R&D funding and the industry shift toward customized therapies. Analytical instruments are forecasted to lead this market expansion, underscoring the critical necessity of robust measurement and testing capabilities across the entire drug lifecycle.

 include a fully gloveless filling system for cell and gene therapies, which ensures maximum sterility compliant with Annex 1 good manufacturing practice guidelines. 

 for animal-derived ingredients requires rigorous traceability and regulatory harmonization to secure the supply chain, while unit-level syringe traceability via RFID improves efficiency.

Nearly 100 abstracts featuring AstraZeneca medications or pipeline molecules will be shared 

 demonstrating advancements in cancer care. These presentations include pivotal Phase III trial data for ADCs like trastuzumab deruxtecan and datopotamab deruxtecan in high-risk breast cancer.

AstraZeneca plans a $4.5 billion investment in a new US manufacturing facility to support a broader range of medicines, including cancer treatments. This major infrastructure commitment coincides with 

 to offer drugs at lower, most-favored-nation prices through the federal government’s forthcoming TrumpRx.gov platform.

Biopharma R&D strategy pivots: M&A favors late-stage assets, obesity deals surge, AI accelerates manufacturing, and policy drives strategic US facility investment.

Targeted Alpha Therapy as a cancer treatment in clinical trials as a oncology therapies targeting malignant tumors with radioactive particles destroying cells | Image Credit: ©freshidea -stock.adobe.com

Radiopharmaceuticals have seen a surge in research and clinical trials as a treatment for cancer due to their ability to target specific cancer cells. Studies suggest that targeted radiation therapy may reduce the risk of side effects, according to the National Cancer Institute at the National Institutes for Health (1).

“Radiopharmaceuticals also work best when the drugs can get inside cells. But that’s not necessary for them to be effective. Once a radiopharmaceutical has stuck to a cancer cell, the radioactive compound naturally breaks down. This decay releases energy that damages the DNA of nearby cells. And when a cell’s DNA is irreparably damaged, that cell dies. Cancer cells are particularly sensitive to radiation-induced DNA damage” (1).

Read this article in the Trends in Formulation October 2025 eBook.

Michael Ritchie, chief commercial officer at Champions Oncology

, to find out what makes radiopharmaceuticals unique for the treatment of cancer.

also talks about FDA’s draft guidance document

Oncology Therapeutic Radiopharmaceuticals: Dosage Optimization During Clinical Development

, which the agency published on Aug. 18, 2025 (2). The guidance was created to assist drug sponsors with identifying an optimized dosage for radiopharmaceutical therapies for oncology indications during clinical development.

PharmTech: What is unique about the use of radiopharmaceuticals to treat cancer?

 Actually, radio pharmaceuticals have been around for the past 100 years or so. They were used early on to treat more superficial tumors [such as] melanoma and using large machines to direct focused radiation to the tumors. And as that technology evolved, we were able to treat more internal tumors.

So, it’s been around for a very long time, but we’re in the middle of a transformation right now in radiopharmaceuticals, and much of that is owed to a field called antibody-drug conjugates [ADCs]. [ADCs] have been around for quite a while. That’s that magic bullet idea that you can take a chemotherapeutic or something that’s otherwise very toxic to every cell in a patient and direct that to the tumor, specifically, so that you impact only the tumor and nothing else. The first of those drugs was approved in 2000. It’s a drug called Mylotarg, and it really took us 25 years to have a good understanding of what makes a good ADC. Where can they be effective? From a therapeutic point of view, what do we need to do to make this drug long lasting in the patient?

Now we have [seen] really strong, durable responses in patients with ADCs. Now that we have a good understanding of that, we can take these radio ligands, which essentially [are] an element on the periodic table that introduces radiation in a focused way, and we can conjugate that to an antibody in the same way that these ADCs work. And so, with the advent of success in ADCs, we now have radio drug conjugates, and we’re starting to see people employ radio ligands in a similar way to a chemotherapeutic and direct it to a patient administered systemically. It’s really an exciting period, because it’s a new mechanism of action where we can potentially tackle unmet needs.

PharmTech: What are some of the specific challenges associated with the development of radiopharmaceuticals?

 There [are] real challenges across the board. There are therapeutic development challenges as well as the biology of treating these tumors. From a therapeutic development perspective, we’re still in our infancy as far as what we understand makes a good drug.

There’s so much [put] into pharmaceutical development [to make] something that can be manufactured at scale. It can sit on a shelf for a while. We can administer it to a patient, and it behaves in the way that we want. All of that chemistry, all of that biologics development, we’re just learning how to apply what we learn from ADCs into radio drug conjugates, so the chemistry of how to link this radio ligand to an antibody, [is] something that will continue to evolve, and there’s a real opportunity there to improve the therapeutic activity.

From the biology perspective, again, we’re in our infancy and understanding [of] what are the tumor types or the molecular cohorts within a tumor that are more sensitive to radio ligands. Like any chemotherapy or any therapy, certain tumors will respond. Others will not. They’ll develop resistance. And so really understanding the right cohorts of patients to treat is something that we’re still learning, and I think we’ll continue to learn as the clinical development of these programs evolves.

PharmTech: Can you please explain how clinically annotated patient-derived xenograft (PDX) models are used in radiopharmaceutical testing?

 PDXs are used from a couple different perspectives in radiopharma testing. It really comes back to that second hurdle that we need to overcome in understanding what tumor types or molecular cohorts respond. It’s really important that you do your drug testing and models that represent the tumors that your drugs [are] going to be up against in the clinic. Patients tend to be heavily pretreated. When they take an experimental medicine, their tumor has metastasized to multiple organs, and those are, unfortunately, the lesions that ultimately kill the tumor. Those are the lesions you need your drug to combat effectively. And so, using models that are representative of that biology are key. We simulate clinical trials in animals where we will take our tumor bank, we’ll grow them in animals, and then you’ll test your drug across a broad library of tumor[s] [that] simulate that clinical trial. Then, when you see where you have an effect and where you do not have an effect, we’ll look deeply at the molecular nature of these tumors and try to predict in the clinic which patients should receive the drug because they’re most likely to benefit versus those that are likely to benefit from a different type of therapy.

PharmTech: Why is it important to use real-world data in this type of testing?

 Real-world data are a real important data set, and they’ve been around for a while. And the way I think of real-world data is, is there information on patients? What’s the stage of their tumor, the metastatic nature of it? What were they treated with? How did they respond?

That’s a very important layer. But I think there’s another side to the coin that we really need to think about, and that is, what’s the molecular nature of the tumors associated with that patient? What makes those tumors tick? What are the vulnerabilities and the liabilities within those tumors that you could potentially exploit in therapeutic development? I think Champions is actually one of the unique companies that have both of those sides of the coin. We have a large data set of real-world evidence linked to very deep molecular data of the tumor, whole cell exome sequencing, RNA [sequencing], proteomics, phospholipids, cell surface proteomics. This really allows researchers to dive deep [and] understand molecular characteristics of the tumors that potentially impact survival [and] impacts resistance to the therapeutic. It just gives them real insight early on [about] how to design their clinical trials. As companies come to Champions and they simulate these clinical trials, they utilize these data for prediction purposes and for driving greater insights.

PharmTech: How does FDA’s new draft guidance on identifying dosages for clinical trials of oncology radiopharmaceuticals impact the development and approval of these treatments?

 Classical radiopharma relied on something called EBRT, or external beam radiotherapy, and that is essentially delivering a radio beam to the patient from an external source. But we’re now looking at a new wave of therapies which are injected systemically, and quite frankly, we just don’t know enough about them. And so, FDA has released new guidance that has said, ‘Hey, we want you to come to the table with a better understanding of pharmacodynamics potential liabilities from a toxicity perspective, therapeutic window. But we also want you to design your clinical trials in such a way that we can get an idea on dosimetry. We can get a good idea on toxicity from an acute perspective, but we want you to monitor these patients for a longer period of time to look for more chronic toxicities that could arise. And we want you to try different therapeutic doses, different therapeutic schedules. We really want you to throw quite a lot at this, so that if we consider approving these, we have a better understanding, or the best understanding, of what these patients are receiving’.

What are some of the challenges involved with clinical trials for radiopharmaceuticals to treat cancer?

 I think there’s both scientific and logistic challenges here. From the scientific side, we really just don’t know what we don’t know. These haven’t been around for very long, so we’re really unaware of the tumor types that respond to these types of therapies. We’re unaware of the types of toxicities.

If you think about ADCs, which are kind of a second cousin to these therapies, after more than two decades of research, we have a really good understanding of what organ types are. You can easily sustain those types of therapies, and we know how to manage the patient care for different classes. For [radiopharmaceuticals], we just, we just don’t know. And so that creates a challenge. We also really don’t understand, I think, quite yet, what makes the best possible radiotherapy from pharmaceutical sciences perspective.

From the drug development side, the logistics are very, very different. This is a highly regulated space. We’re dealing with nuclear medicine. The nucleotides themselves aren’t so readily available, so that brings challenges. And then obviously not every clinical site that will receive patients for a clinical trial is set up or equipped to offer this type of therapy, so patient enrollment can be a challenge.

And then finally, these nucleotides have a short half-life and a short shelf life. And so, it’s not like drugs can be manufactured and stuck in a refrigerator for months as they’re waiting to be used for a trial. They kind of have to be made and then used within a week, so there’s some logistical hurdles as well.

NCI Staff. Radiopharmaceuticals: Radiation Therapy Enters the Molecular Age. National Cancer Institute. 

https://www.cancer.gov/news-events/cancer-currents-blog/2020/radiopharmaceuticals-cancer-radiation-therapy

Draft Guidance for Industry, Oncology Therapeutic Radiopharmaceuticals: Dosage Optimization During Clinical Development

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/oncology-therapeutic-radiopharmaceuticals-dosage-optimization-during-clinical-development

When referring to this article, please cite it as Haigney, S. Why Radiopharmaceuticals Are Ideal for Treating Cancer. 

Radiopharmaceuticals are gaining traction in cancer treatment due to their ability to target specific cancer cells, minimizing side effects. The integration of antibody-drug conjugates (ADCs) has advanced the field, allowing for precise delivery of radiation to tumors. Challenges remain in optimizing dosage, understanding tumor sensitivity, and overcoming logistical hurdles in clinical trials. The FDA's draft guidance emphasizes the need for comprehensive understanding of pharmacodynamics and toxicity. Real-world data and patient-derived xenograft models are crucial for predicting treatment efficacy and designing effective clinical trials.

Michael Ritchie, chief commercial officer at Champions Oncology, explains what makes radiopharmaceuticals unique in the treatment of cancer.

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