Feliza Mirasol is the science editor for 

Feliza Mirasol

On Jan. 13, 2025, Pharmapack Europe and CPHI announced the key trends they have identified for the pharmaceutical industry going into 2025. High on the list of those trends is the long sought-after return of funding liquidity, primarily from increased venture capital investments and private equity capital for mergers and acquisitions. Enhanced supply chain resilience is also anticipated in the year.

Conversely, the entities have identified insecurities that are expected to plague the 2025 year, including ongoing uncertainty surrounding the intentions and practical implications of the BIOSECURE ACT in the United States as well as Donald Trump’s return as president of the country, which Pharmapack has termed “the Great Known Unknown”.

Yet, despite the uncertainties facing US pharma companies, both Pharmapack and CPHI experts emphasize that strong fundamentals underlining the industry have nurtured widespread optimism. These experts are predicting a landmark year in 2025, which they expect will be driven by a boom in biologics, robust pipelines, and growing profits from glucagon-like peptide-1 (GLP-1) drugs for treating diabetes and related obesity. In addition, new manufacturing hubs are expected to be established, which will accelerate supply chains. Outsourcing will be a key driver of drug discovery, development, and commercial supply, the experts predict (1).

The uncertainties tied to Trump’s return to office have led many industry analysts to caution an "expect the unexpected" attitude, but the pharma market has responded with “surprising optimism,” notes Pharmapack. “There is even a measured ‘wait-and-see’ attitude toward the controversial appointment of Robert F. Kennedy Jr. Remarkably, eight years after our initial New Year’s prediction release on a Trump presidency as the ‘great unknown’ (2), this 2.0 iteration has been met with a surprisingly composed response from the pharma sector. The takeaway? While changes are anticipated and the direction remains difficult to forecast, the industry appears confident that there will be more winners than losers, regardless of the path taken during the next administration,” Pharmapack stated in a press release (1).

The practical implications of the BIOSECURE ACT are expected to continue to evolve until the bill is either passed or rejected in some form. The industry, in the meantime, is already preparing for medium-term changes, such as switching discovery chemistry services to Europe and to Indian contract research organizations. In comparison, development and commercial contracts are being more widely shared, opening up new opportunities, particularly for companies investing in advanced biologics manufacturing with an eye toward lowering costs per gram.

On the Europe front, European markets are expected to drive innovation in drug delivery and packaging forward over the next 12-months. Pharmapack identified Île-de-France as a growing innovation hub for the MedTech sector in 2025, and the region will host Pharmapack Europe 2025 on January 22–23. “[The region]’s highlighted as the ideal location for start-up device and MedTech companies, thanks to its combination of funding opportunities, availability of R&D staff and, crucially, a strong base of pharma manufacturing and packaging resources,” according to the press release.

Pharmapack also emphasizes that the Île-de-France region is expected to play a significant role in both biologic device design and contract filling, based largely on the ongoing demand for GLP-1 drugs. Aseptic filling capacity is expected to be tied up in the clinical filling needs of GLP-1s, especially with generic GLP-1s soon to enter the market in 2025, which may cause delays in the clinical filling of other therapies with smaller volumes, such as oncology-targeted therapies, according to Pharmapack. “The result is that we will see biotechs widening supplier networks and potentially even Big Pharma searching for new partners for GLP-1s,” Pharmapack states in the press release. “Consequentially, contract and device filling is predicted to be a rapid growth area at Pharmapack.”

More than 6000 attendees and more than 370 exhibiting companies are expected to attend Pharmapack Europe this year. Pharmapack is seeing significant growth in participation from early stage companies as well; the conference’s Start-Up Hub is expected to host more than 20 emerging companies. In particular, there has been increasing interest from German and Swiss executives who are seeking new opportunities in development and licensing deals for device and packaging innovations.

The full results of Pharmapack’s report can be accessed on 

. More information about Pharmapack Europe can be found on 

1. Pharmapack Europe. Pharmapack Predictions 2025: the Good the Bad and…. the Donald 2.0. Press Release. Jan. 13, 2025.
2. Wright, T. The Good, the Bad, and the Donald. 

Articles

Pharmapack identifies biologic approvals, drug delivery advancements, GLP-1 generics, and the return of funding flow as drivers for a record year in 2025.

Pharmapack Reports on Key Trends for 2025

On Jan. 13, 2025, Lonza announced that it has partnered with Iconovo, a Swedish company specializing in developing inhalable drugs based on proprietary inhalers and dry powder formulations, to develop spray-dried formulations of an intranasal biologic. The biologic will be administered through Iconovo’s proprietary intranasal device, ICOone Nasal. The companies aim to use a reformulated substance for treating obesity, which follows Iconovo’s strategy to reformulate known pharmaceuticals and replace injections and oral treatments with inhalation dose forms.

Under the agreement, Lonza will develop spray-dried formulations to be administered in the ICOone Nasal device at its site in Bend, Ore., in the United States. At the Bend site, Lonza works on addressing bioavailability challenges and modulating pharmacokinetics to meet target product profiles. The collaboration will identify ideal product properties related to particle size, stability, and device compatibility while preserving biological activity.

“Lonza is a world leader in bioavailability enhancement and renowned for its spray-drying capabilities of biologics. Combined with Iconovo’s unique nasal inhaler, we expect the result to be an unprecedented patient-friendly product, and we hope that this cooperation will lead to a fruitful, long-term collaboration that goes beyond this specific project,” said Johan Wäborg, CEO, Iconovo, in a company press release (1).

ICOone Nasal is a proprietary nasal device that releases a dose when the user inhales. An inhale through each nostril from one inhaler administers a standard dose of the biologic compound, according to the press release.

“Our team at Lonza’s respiratory center of excellence in Bend is pleased to work with Iconovo on its innovative applications, combining Lonza’s particle engineering expertise with Iconovo’s state-of-the-art devices. Delivering non-invasive and accessible therapies to patients in need is a focus area for both companies,” said Kim Shepard, director of Advanced Drug Delivery, Lonza, in the release.

“This collaboration, leveraging Iconovo’s innovative intranasal delivery technology alongside our expertise in spray-dried formulations, underscores our commitment to advancing patient care by developing innovative products. We believe that combining our strengths can lead to the development of patient-friendly products and a long-lasting, successful collaboration,” said Jan Vertommen, vice-president, head of Commercial Development, Small Molecules, Lonza, in the release.

In addition to this collaboration for an inhaled biologic, Lonza expanded its services to support smart capsule companies who are working to develop 

 in December 2024 (2). Smart capsules use electronic or mechanical elements to deliver drugs to the stomach or intestine. Promising applications for smart capsules include the treatment of diabetes, obesity, and other therapeutic fields.

To support these smart capsule developers, Lonza now offers a bespoke development and manufacturing service that focuses on their specific needs. Lonza can tailor its service to certain requirements, such as size, design, and lock mechanisms, and offers a variety of functionalities, including targeted release using a bi-layer manufacturing technology.

1. Lonza. Iconovo and Lonza to Collaborate on the Formulation Development of an Intranasal Biologic. Press Release. Jan. 13, 2025.
2. Lonza. Lonza Launches Tailored Offering for Smart Capsules Companies Developing Oral Delivery Solutions for Biologic Drugs. Press Release. Dec. 3, 2024.

Lonza will work with Iconovo to develop spray-dried formulations for an intranasally delivered biologic using a reformulated biologic drug candidate for obesity.

Lonza and Iconovo Team Up on Intranasal Biologic Candidate

US contract research organization and contract development and manufacturing organization PackGene Biotech has entered into a collaboration with Genetic Cures for Kids (GC4K), an Australian non-profit group, and the Belfer Gene Therapy Core Facility at Weill Cornell Medicine, to deliver an accessible, custom-tailored gene therapy solution for treating hereditary spastic paraplegia type 56 (SPG56), an ultra-rare neurological disease, PackGene announced on Jan. 8, 2025.

Under the alliance, the team will use adeno-associated virus serotype 9 (AAV9) to deliver the healthy gene directly into patient cells. PackGene specializes in providing comprehensive services, including plasmid DNA, viral vector, and messenger RNA production as well as supporting programs from research stage to commercialization. PackGene has pledged to subsidize costs and fast-track the development of a clinical-grade gene therapy for SPG56. Meanwhile, Weill Cornell Medicine’s Belfer Gene Therapy Core Facility has tailored its AAV manufacturing capabilities to serve small, patient-driven projects.

“Our work at the Belfer Gene Therapy Core Facility is driven by a commitment to making gene therapy accessible to all patients, especially those with rare diseases [such as] SPG56,” said Stephen M. Kaminsky, professor of Research in Genetic Medicine at Weill Cornell Medicine, in a press release (1).

SPG56 is a progressive, degenerative disorder that typically begins in early childhood (2). This global alliance was prompted by the parents of Tallulah Moon, a young child who suffers from the disease. The parents founded GC4K in an effort to find a cure for their daughter. Children lose motor and cognitive abilities as the disease progresses. From the start, diagnosing SPG56 can be an uphill battle, especially considering that genetic testing is limited in its ability to detect the disease due to its ultra-rare nature. According to the press release, fewer than one in a million people worldwide are diagnosed, and most families are left with no treatment options (1).

This collaboration between PackGene, Weill Cornell Medicine, and GC4K is meant to show that ultra-rare diseases can draw international attention, funding, and breakthrough innovations in a pharmaceutical industry where large patient populations and high-return therapies are often prioritized, according to the press release. This project uses “top-tier science” to deliver tailored solutions for such a small population of children and is an approach that can be replicated for thousands of other conditions that are currently “too rare” to draw significant investment (1).

“Together with my colleague Professor Dolan Sondhi, our long-established track record in supporting therapeutic development programs, combined with our expertise in AAV vector manufacturing, is being harnessed to potentially deliver life-changing treatments for Tallulah. This effort also serves as an example for addressing thousands of other rare genetic conditions,” Kaminsky said in the release.

This initiative has potential to go beyond a single disease such as SPG56. GC4K aims to create a replicable framework to address more than 7000 other genetic conditions that currently have no cure.

“Our collaboration with Genetic Cures for Kids and Weill Cornell Medicine’s Belfer Gene Therapy Core Facility is a testament to the power of compassion paired with scientific excellence,” said Xin Swanson, PhD, chief scientific officer, PackGene Biotech, in the release. “We are leveraging our expertise not just to create plasmid DNA and AAV vectors, but to reshape the future landscape of gene therapy—making it affordable, accessible, and deeply human.”

“As the mother of a child living with SPG56, I’ve come to understand that families like ours are not just fighting diseases—we’re fighting for the attention, resources, and urgency that our children’s lives deserve,” said Golden Whitrod, founder of Genetic Cures for Kids and mother to Tallulah Moon, in the release. “Until governments seriously invest in the credible and worthwhile market of personalized medicine, family foundations like ours will continue to lead the charge. But we cannot do it alone. The generosity and philanthropy of companies [such as] PackGene and institutions [such as] Weill Cornell Medicine remind us that compassion and innovation can change the world, one life at a time.”

A Global Alliance to Save Lives: How Two Parents and PackGene Are Redefining What’s Possible in Gene Therapy with Help from Weill Cornell Medicine

. Press Release. Jan. 8, 2025.
2. Wong, S. S-N.; Yuen, L. Y-P.; Kan, E.; et al. CYP2U1: An Emerging Treatable Neurometabolic Disease with Cerebral Folate Deficiency in 2 Chinese Brothers. 

, 101023. DOI: 10.1016/j.ymgmr.2023.101023

Through this global alliance, PackGene, Weill Cornell Medicine, and GC4K, an Australian non-profit, intend to deliver a custom-tailored gene therapy solution to treat hereditary spastic paraplegia type 56, a particularly rare neurological disease.

PackGene and GC4K Team Up Along with Weill Cornell Medicine for Gene Therapy to Treat Rare Neurological Disease

FDA has granted fast track designation to Johnson & Johnson (J&J) for posdinemab, an investigational phosphorylated tau-directed monoclonal antibody (mAb) for treating patients with early Alzheimer’s disease (AD), J&J announced on Jan. 8, 2025. Discovered by J&J, posdinemab is in a Phase IIb study (AuTonomy) and has shown potential in targeting disease-associated phosphorylated tau in cerebrospinal fluid (CSF) from AD patients who are being treated. The mAb has also shown an ability to block the development and spread of tau aggregates in non-clinical models of disease.

FDA’s decision on posdinemab marks the second fast track designation granted to J&J in 2024 for its AD portfolio. In July 2024, FDA granted fast track designation to J&J’s anti-tau active immunotherapy, JNJ-2056, which represents the first active immunotherapy that targets tau in a preclinical AD population (1). JNJ-2056 is in a Phase IIb trial (ReTain), which is currently actively enrolling patients. This Phase IIb trial will evaluate the potential of JNJ-2056 to activate the immune system so that it generates antibodies against pathological phosphorylated tau. The goal with this mode of action is to delay or prevent the onset of AD symptoms and, ultimately, progression of the disease.

“As the average age of the global population increases, the number of people impacted by this debilitating progressive disease continues to rise. Alzheimer’s disease places a substantial emotional and financial burden on patients and their loved ones and has a significant economic and societal impact. At J&J, we envision a future where we can help stop the progression of AD the moment it’s detected,” said Bill Martin, PhD, global therapeutic area head, Neuroscience, Johnson & Johnson Innovative Medicine, in a company press release (1).

Posdinemab targets the mid-domain of AD-specific phosphorylated tau. The mAb is designed to bind to pathological phosphorylated tau when released from neurons and to neutralize it before it can seed or spread to another neuron. Tau, which is abundant in neurons, has a notorious reputation, which is due to the fact that it is found deposited in cells as fibrillar lesions in many neurodegenerative diseases, especially AD (2). Posdinemab has shown promise in reducing tau seeding, which is the process by which toxic tau spreads through the brain, in both 

“Posdinemab has the potential to slow the spread of tau pathology in the brain—which may slow cognitive decline. [FDA’s] [f]ast [t]rack designation reflects the urgent unmet need for new treatment options for the millions living with AD,” said Martin in the press release.

Meanwhile, JNJ-2056 (formerly ACI-35.030, in collaboration with AC Immune) is an investigational anti-tau active immunotherapy that targets the c-terminal region of pathological phosphorylated tau. This therapy is designed to inhibit the seeding and spreading of pathological tau as well, also with the goal of delaying or preventing the onset of clinical symptoms in preclinical AD. The Phase IIb in which JNJ-2056 is being evaluated includes participants with preclinical AD who have tau pathology in their brains but have not yet developed cognitive impairment.

Johnson & Johnson’s Posdinemab and Tau Active Immunotherapy Receive US FDA Fast Track Designations for the Treatment of Alzheimer’s Disease

. Press Release. Jan. 8, 2025.
2. Stoothoff, W. H.; Johnson, G. V. W. Tau Phosphorylation: Physiological and Pathological Consequences. 

Biochimica et Biophysica Acta (BBA)—Molecular Basis of Disease

 (2–3), 280-297. DOI: 10.1016/j.bbadis.2004.06.017

Posdinemab is the second tau-directed investigational therapy by J&J that has received fast track designation from FDA this year for Alzheimer’s disease.

J&J Gets FDA Fast Track Designation for Alzheimer’s mAb, Posdinemab

Argobio announced on Jan. 7, 2025 that it has launched Inverna Therapeutics, a new, Denmark-based biotech company specializing in innovative RNA therapeutics, with the University of Southern Denmark as co-founder. The new company will focus on severe genetic diseases, starting with Huntington’s disease, for which the company has a lead program.

Inverna’s technology platform comes from research conducted by Professor Brage S. Storstein Andresen, an expert in RNA splicing and antisense technology who holds the position of co-founder and chief scientific officer at Inverna (1). Inverna’s goal is to overcome the limitations of conventional RNA therapeutics, and it will do so by harnessing the potential of sequence-based splice modulation, which will allow the company to create safer and more effective treatments for a wide range of debilitating diseases, according to a press release.

“We are thrilled to introduce Inverna to the world,” said Poul Sørensen, CEO of Inverna and partner and entrepreneur-in-residence at Argobio, in the release (1). “Our team, combined with the strong support of Argobio and the University of Southern Denmark, is uniquely positioned to develop transformative therapies that address significant unmet medical needs. Leveraging our proprietary insights into RNA splicing modulation, our pseudo-exon targeting technology offers a potential solution to limitations of current RNA therapeutics by enabling precise, sequence-based and allele-specific targeting with minimal off-target effects.”

Argobio is a European biotech start-up studio, based in Paris, that has played an important role in establishing Inverna. The studio sources early-stage therapeutic projects from leading European academic research institutions and focuses on rare diseases, neurological disorders, oncology, and immunology. Argobio selects and incubates these projects with the goal of developing them into fully-fledged biotech companies and advancing them to significant Series A financing. The studio offers extensive expertise in novel drug discovery and development with the help of a team of highly experienced biotech entrepreneurs. With an initial investment, strategic guidance, and operational expertise, Argobio empowered Inverna to accelerate the development of its innovative RNA pipeline.

“It is fantastic to have the opportunity to translate our many years of research on pre-mRNA [messenger RNA] splicing regulation in human disease into new RNA precision medicines,” said Andresen in the release. “We are excited and eager to unleash the potential of our new discovery engine to develop new RNA therapeutics for untreatable diseases.”

Argobio builds companies to address critical medical needs. The companies that it establishes are based on breakthrough scientific discoveries and innovative approaches, according to Thierry Laugel, chairman at Argobio and managing partner at Kurma Partners.

“We are very pleased to transform another outstanding academic project into a promising industry player. Inverna fits perfectly into our model, with access to cutting-edge science and our unique expertise in building and running biotech companies,” Laugel said in the release.

The University of Southern Denmark was also instrumental in laying the scientific groundwork for Inverna’s innovative approach.

“The launch of Inverna Therapeutics exemplifies the University of Southern Denmark’s commitment to transforming world-class research into real-world impact," said Thomas Schmidt, head of Technology Transfer at the university, in the press release. “Through our collaboration with Argobio and the visionary leadership of Professor Brage Storstein Andresen, we are proud to contribute to the development of groundbreaking therapies that address critical unmet medical needs.”

Next-generation RNA Company Inverna Therapeutics Launched by Argobio and University of Southern Denmark

Using splicing technology, the new company aims to innovate safer and more effective RNA therapies for severe genetic diseases.

Argobio and University of Southern Denmark Launch New Next-Gen RNA Company, Inverna Therapeutics

Denmark-based biotech startup Orbis Medicines recently won major funding to support its pursuit of developing oral alternatives to current biologic drugs, which are primarily administered parenterally.

The company announced on Jan. 6, 2025 that it closed a Series A funding round of $94 million (€90 million), which will go toward development of Orbis’ pipeline of next-generation oral macrocycle drugs, which the company has termed “

Cycles”. This latest funding round brings the total amount raised by the company to $121 million (€116 million).

Macrocycles bind to targets with higher affinity and selectivity than small-molecule drugs, but they have been historically challenging to develop as oral drugs. Orbis Medicines’ technology platform, 

Gen, systematically delivers macrocycle candidates—

Cycles—that are optimized for oral bioavailability. It has been this lack of oral bioavailability that has hindered therapeutic development of this class of molecules in the past (1). The 

Gen platform comprises multiple proprietary integrated elements in a “lab in a loop” system. It starts with hit-finding libraries containing 100 billion compounds. Using automated chemistry, the platform can synthesize and analyze up to 100,000 distinct synthetic macrocycles in weeks, which allows the company to discover candidates with the right properties to enable oral dosing and intracellular targeting.

The company’s pipeline is initially focused on 

Cycle candidates aimed against targets validated by blockbuster biologic drugs, which are delivered by injection, a route of administration that is not favorable with patients. The biopharma industry has been bending its efforts toward developing 

 for biologics that bypass the parenteral route, such as oral and inhaled biologic formulations (2). However, because biologics have more structural sensitivities than small-molecule drugs to factors such as pH and temperature, there are limits to their delivery, especially orally, such as degradation in the alimentary tract and an inability to overcome intestinal cell or mucosal barriers and first-pass metabolism (2). These limitations therefore open a path to alternative orally bioavailable drugs, such as 

“The Orbis Medicines team has impressed us with the data supporting 

Gen, which we believe is the world’s leading discovery engine for oral macrocycle drugs. We are thrilled to support not only their journey to the clinic, but also their longer-term vision as a leader in their field,” said Ed Mathers, partner, New Enterprise Associates (NEA), in the press release (1). NEA led the financing.

In addition to NEA as the lead financer, others that participated in the funding round include new investors Eli Lilly and Company, Cormorant, and the Export and Investment Fund of Denmark as well as founding investors Novo Holdings and Forbion. Morten Graugaard has been appointed as CEO of Orbis Medicines after having served nearly three years as executive chair of its board of directors.

“The company is advancing the potential of macrocycles by developing orally available 

Cycles—a format preferred by patients, physicians, and healthcare systems, particularly for chronic diseases,” said Graugaard in the release. “The interest we have received demonstrates the market appetite for 

Cycles as an oral approach to both validated biologic targets and targets undruggable by other modalities. We have a clear strategy for rapid value creation that leverages the unique capabilities of our 

Gen platform and are excited to advance this important new class of drugs for patients.”

Orbis Medicines Raises EUR 90 Million Series A Funding Round to Develop Pipeline of Oral Macrocycles

. Press Release. Jan. 6, 2025.
2. Mirasol, F. 

Exploring Innovative Means for Biologics Delivery

With the new $94 million (€90 million) funding, the company will develop its pipeline of oral macrocycle drugs, nCycles, against validated biologic targets.

