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

Felicity Thomas

Clinical-stage UK-based pharmaceutical company, PharmaKure, has been granted Clinical Trial Authorization (CTA) from the United Kingdom’s regulatory body, the Medicines and Healthcare Regulatory Agency, to start a Phase IIa study to assess an oral combined drug for the treatment of mild cognitive impairment (MCI) as a result of Alzheimer’s Disease.

According to a Jan. 4, 2024 press release, the CTA has been granted for PharmaKure’s PK051 oral combined drug that has been designed to target disaggregation of amyloid-β proteins—overproduction of which is being increasingly accepted as the initial event in Alzheimer’s Disease pathology. In the multi-ascending dose Phase IIa study, the company will evaluate the safety and tolerability of PK051 in patients.

"The MHRA authorization marks a major step forward in our mission to develop PK051 as a disease modifying therapy for MCI due to Alzheimer’s Disease,” said Dr. Farid Khan, CEO, PharmaKure, in the press release. “This authorization follows successful study results recently announced by the company for a novel whole blood test to quantify Alzheimer’s Disease biomarkers. PharmaKure’s proprietary ALZmetrix blood test can identify blood-based biomarkers in patients with Alzheimer’s Disease to provide early warning of cognitive decline. Used as a companion diagnostic, this could enable treatments such as PK051 to be offered earlier to provide better population-based health outcomes.”

“We are delighted to have approval to begin clinical testing of PK051,” added Dr. Bob Smith, chief clinical director, PharmaKure, in the press release. “This Phase IIa study is intended to confirm safety, tolerability, and to help us determine an appropriate dose for future efficacy studies. The trial will involve 40 patients with MCI due to Alzheimer’s Disease at a single site in the UK. The first patient is expected to be dosed in early to mid 2024, with preliminary clinical data emerging within 12 months of first dose.”

Articles

MHRA has granted a Clinical Trial Authorization to PharmaKure for its oral combined drug for the treatment of mild cognitive impairment.

PharmaKure Gets Nod from MHRA to Trial Drug Targeting Amyloid Deposits

Finnish medicine performance enhancing company, Nanoform, has announced, in a Jan. 5, 2024 press release, the completion of the First Subject First Visit (FSFV) in a trial evaluating the relative bioavailability of its nanotechnology-enhanced enzalutamide.

The single-dose, randomized, comparative bioavailability study, which is being performed by a North American contract research organization, will compare Nanoform’s nanocrystalline enabled alternative to an amorphous solid dispersion (ASD) formulation of nanoenzalutamide and the FDA-approved prostate cancer androgen receptor inhibitor, Xtandi, in prostate cancer patients. The trial will compare the bioavailability of one enzalutamide 160 mg film-coated tablets (Bluepharma Farmaceutica S.A.) with that of four Xtandi 40 mg film-coated tablets (Astellas Pharma Europe B.V.). As pill burden and dysphagia are documented as common challenges for prostate patients, the development of a single tablet per day regimen could be a preferable option.

“We are pleased to have achieved [FSFV] as planned. The initiation of this trial represents a key milestone as we advance the development of nanoenzalutamide in prostate cancer for patients,” said Dr. Edward Haeggström, CEO of Nanoform, in the press release. “We continue our discussions with specialty pharma and value-added medicine partners to ensure this advance reaches the patients that need it the most. We see significant interest in nanocrystalline alternatives for [ASDs] from pharma and value-added medicine partners, for whom this platform technology offers a differentiated and improved formulation that could reduce pill burden, provide a preferred route of administration, or improve patient experience, acceptance, and adherence.”

A readout from the clinical trial is expected in the first quarter of 2024. In the event that the results of the trial are positive, Nanoform and partners have reported that they will seek one or more license and/or commercial supply agreements for nanoenzalutamide during 2024, under the proviso of maintaining 25% share of net-income.

Nanoform has completed the First Subject First Visit in a trial evaluating the relative bioavailability of its nanotechnology-enhanced enzalutamide.

Relative Bioavailability Trial of Nanotechnology-Enhanced Enzalutamide is Underway

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At the beginning of 2023, inflation represented a significant thorn in the side of global economies and exacerbated the challenges raised by the tumultuous preceding 12 months—encompassing the tail end of the COVID-19 pandemic, the war in Ukraine, and skyrocketing energy prices. Despite inflation moving in the right (downward) direction across Europe, it is still a lingering issue for the continent and is negatively impacting European economies (1,2).

For the bio/pharma industry—an industry well known for its ability to weather economic turmoil with reasonable safety—the latest global economic challenges have raised significant concerns. In Europe, moving into 2024 there will be additional factors at play, such as an increased drive to digitalization, a focus on sustainability, and a shortage of skilled personnel, meaning that bio/pharma companies will need to adapt over the coming months.

“The pandemic, inflation, geopolitics, new therapeutic modalities, evolving ways of working, and EU [European Union] regulatory changes, have conspired to have major implications for the pharma industry,” reports Romuald Braun, managing partner, MAIN5, a European consulting firm specializing in organizational and digitally enabled change in Life Sciences.

“The most notable fallout,” Braun says, “has been rising operational complexity (e.g., the need to manage diverse product portfolios and supply chains); increasing risk around balancing global demand, supply chain disruptions, and new modalities; the need to adapt to technological changes; rising capital costs (investing in new technologies and facilities); variable cost increases (addressing inflation and supply chain challenges); and the need to seize every opportunity for savings through optimized operations.”

In concurrence, Jordan Schmitting, senior research analyst, The Josh Bersin Company—research, advisory, and education on the trends, technologies, and best practices for the new world of work—confirms that the evolving geopolitical, economic, and regulatory conditions have put European pharma companies’ supply chains and profit margins under pressure throughout 2023.

“However,” Schmitting adds, “digitization and biotechnology were perhaps the most defining trends within the industry, shaping the future of not only how treatments are discovered, developed, manufactured, and distributed, but the types of treatments being explored.”

An increased integration of digital technologies is being seen within the bio/pharma industry, agrees Braun. “This [integration] includes the use of AI [artificial intelligence], big data analytics, and digital health platforms to improve efficiency and outcomes,” he says. “Digital transformation now extends to adoption of ‘Generative AI’, as companies strive to be more data and insight driven, moving away from dependence on static documents.”

Furthermore, the use of cloud-based systems to ease information exchange and data management will be seen, Braun continues. “Breaking down data silos is essential to enable advanced analytics and faster decision-making—as long as data governance and data quality are prioritized so that teams can trust the insights being surfaced,” he states.

Along with changes in data collection, however, comes an increased focus on data privacy and the general data protection regulation, adds Kate Coleman, vice president of Quality and Compliance at Arriello—a consultancy and solutions provider of risk management and compliance services to the pharma industry. “Again, these topics were raised repeatedly in 2023, not just in the context of medicine but also in the broader social context, and the importance of data protection is a conversation that will continue into 2024,” she notes.

“AI has gathered pace and is a consistent talking point as it is now recognized as a powerful tool in many areas across the pharmaceutical industry including drug development, writing, data analysis, translations, and pharmacovigilance activity to name a few,” remarks Coleman. “AI has the potential to radically change how we do things in the industry, and everyone will need to increase their understanding of this powerful technology to understand both its strengths and weaknesses.”

For Beena Wood, senior vice president of Safety and Medical, ArisGlobal—the creator of the LifeSphere technology platform and provider of global patient treatment solutions—there has been crucial technological innovation that is now providing all bio/pharma companies access to AI and machine learning (ML). “AI/ML can now be applied to a broad range of connected data—including a broader range of standardized, reliable real‑world data—to enable advanced analytics and powerful, timely insights,” she says. “This will inspire new treatment/care innovation with the interests of patients right at the heart.”

Patient-centricity will become an intense focus for bio/pharma companies in 2024 because of several converging factors, according to Wood. “First, the status quo is being challenged increasingly by empowered patients who now know and expect more,” she specifies. “Second, [there is] accelerated delivery of personalized therapies, geared to the needs of smaller patient populations and individuals. Meanwhile, regulatory mandates are now prioritizing patient concerns and considerations right from development phase and throughout the treatment lifecycle.”

Braun concurs that patient centricity will be a huge topic for companies moving forward and points out that patients’ needs and outcomes will remain important for companies. “Personalized medicine, patient engagement, and real-world evidence will guide decision-making,” he states.

“The increased focus on personalized medicine, gene therapies, and other advanced therapeutics combined with discussions on treatment costs have raised questions that will need to be considered for the viability of future innovative pharmaceutical products,” emphasizes Coleman. “Many high-profile agreements have been reported in 2023, and the topic of cost benefit analysis for innovative therapies will be ongoing in the next year.”

Another focus for bio/pharma companies in the future will be embracing more sustainable practices, something that is being expected from customers and patients. “Companies will be increasingly investing in green initiatives, implementing eco-friendly manufacturing practices, and adopting sustainable packaging solutions,” says Preeya Beczek, principal consultant at Beczek.com, a regulatory affairs and compliance consultancy.

“Increasing efforts will need to be made to reduce waste generation, minimize water consumption, and decrease greenhouse gas emissions,” Beczek continues. “These changes will need to be measurable so that achievements against targets can be demonstrated through key performance indicators over time.”

As the climate crisis is becoming more apparent to the world, an issue that will only be more heightened in the coming years, the bio/pharma industry is finding itself in a difficult position, states Coleman. Simply due to the nature of the bio/pharma industry—manufacturing and distributing products around the world—it is a major contributor to the global carbon footprint, she stresses.

“The pharma industry is also highly regulated, which means significant change is neither easy nor fast,” Coleman says. “To really combat the impact that the pharma industry has on the environment, the risks and impacts from the industry need to be fully identified across all activities from sourcing of raw materials, shipment, transport, manufacture, distribution, and management of waste to identify a full and holistic plan for change that can reduce the impact on the environment and on the planet. When mitigation actions are identified that will improve the carbon footprint, such as reverting to stainless steel rather than single use plastics, the regulators will need to assist companies with balancing the risks between impact on patients, impact on employees, and impact on the environment as a whole.”

There are numerous environmental, social, and corporate governance (ESG) challenges facing the bio/pharma industry, confirms Braun. “These [challenges] include reducing carbon footprint and promoting sustainable practices; ensuring ethical sourcing and minimizing environmental impact across the supply chain; balancing profit with patient needs to ensure patient access and affordability; more responsible drug development (ethical research and innovation); diversity and inclusion considerations, promoting a diverse workforce and equitable practices,” he notes.

“Moreover, there will be changes to what needs to be reported on, and how, under the EU’s Corporate Sustainability Reporting Directive (CSRD),” Braun stresses. “From 2024, almost 50,000 companies are subject to mandatory sustainability reporting, including non-EU companies with subsidiaries operating within the EU or listed on EU-regulated markets.”

An increasing role of technology and innovation, such as AI, robotics, and automated processes, combined with newer therapeutic modalities and personalized medicines in the bio/pharma industry is changing the skills and expertise required, emphasizes Coleman. “For example, roles such as the Qualified Person have changed significantly when considering ATMPs [advanced therapy medicinal products] where the QC [quality control] tests and timelines for batch review and release to market have been either significantly reduced or removed completely,” she says.

Schmitting also alludes to a misalignment of roles and skills across all talent segments of the bio/pharma industry caused by rapid evolution of the industry’s requirements as a result of trends like AI, automation, digitization, and big data. “These forces are changing the nature of work not only in manufacturing and distribution, but across the entire pharma value chain, including R&D,” she says. “Take AI-driven platforms, for example, which is a research innovation fundamentally changing the methods and timescale of specialized therapy discovery and development.”

However, the technological innovation being seen within the bio/pharma industry could also help to attract the younger generation to the sector, Beczek remarks. The skills gained with a more technologically advanced bio/pharma industry can also be transferrable should the potential employee wish to change their role, she adds. “Harnessing the tech-savvy power of this younger generation could help the industry see new possibilities and pathways to drive change forward,” Beczek states.

Additionally, as biologicals and molecular technologies become more present within industry, the skillsets required to deal with these are changing and are not necessarily easily acquired, Braun stresses. To manage this issue, Braun anticipates there will be an increase in M&A activity and a rise in the use of external services to plug gaps.

“Lower-skill labour in production facilities is a challenge too, not least as 24/7 continuous production increases,” Braun says. “To address these shortages and contain costs, pharma companies will need to continue streamlining processes, harnessing automation and applying AI as appropriate.”

Potential options for bio/pharma companies to deal with the skills shortage is to collaborate with academic institutions, industry associations, and research centres to pool talent, Braun continues. To attract and retain skilled professionals, companies should develop a more compelling employer value proposition and offer continuous learning opportunities for existing employees, such as upskilling in digital technologies, and so forth, he adds.

Beczek believes in a two-pronged approach to tackle the skills shortage. “Start at the higher education level and actively recruit seasoned medical experts in the key therapeutic areas to join innovative companies as clinical development leaders, medical experts to drive product strategy at a disease level,” she explains.

There also needs to be different pathways offered in addition to the standard three- to four-year full-time qualifications pursued at an academic institution, according to Beczek. “There are a handful of settings that offer young adults a life sciences path via apprenticeships with an opportunity to balance hands-on work and study including a salary. This is the way forward,” she says.

“The academic institutions do appear to recognize the change in the industry and proactive measures have been taken at this level to ensure graduates are more equipped to deal with the demands of the reality of working in a high technology environment,” specifies Coleman. “However, one element that this cohort lack is the practical experience needed to develop into quality resources that can support this ever-changing industry.”

Moving into 2024, Braun expects that bio/pharma companies will continue to be cost-conscious and the industry transformation with new modalities gaining prominence will persist. “Pharma companies will need to invest in R&D to harness these [therapeutic] innovations effectively,” he says.

Adoption of digital technologies will accelerate, and companies will need to invest in digital capabilities to keep up with opportunities, Braun adds. “AI/ML will move from a prototyping or pilot phase towards larger use cases, presenting new challenges for validation and data integrity,” he says. “As the pace of change accelerates, IT must be much closer to the business than previously.”

There is heightened pressure on bio/pharma companies to control costs, maximize the value of investments, and optimize their strategic focus, while also maintaining the highest levels of patient safety and stakeholder confidence, adds Wood. “In pharmacovigilance and agency reporting, large language models/ChatGPT-like Generative AI tools are poised to deliver up to 80% productivity gains for certain work processes, compared with 20–50% improvements with previous-generation technologies,” she states. “No company can afford to let that kind of opportunity pass them by. The appetite is there, and proof-of-concepts are well underway now.”

However, with the various therapeutic and technological advancements, companies will also need to ensure they keep abreast of regulatory updates. “The EU’s regulatory changes will continue to impact the industry. Companies must stay informed about evolving regulations related to clinical trials, medical devices, and AI applications. Compliance and adaptation are key,” Braun reports.

Coleman also points to the regulatory landscape, which will be a focus in 2024 as the existing pharmaceutical regulation will be replaced. “The main objectives of the revision are aligned with the points mentioned previously, and it is intended that the revised EU pharmaceutical regulation will address innovation and technological advancements in the industry, align manufacturing of medicinal products with the European Green Deal related to climate change, and make medicines in Europe more affordable, accessible, and available to patients that need them,” she summarizes. “The changes are intended to make the industry and its regulation more agile, and more appropriate for the world we live in now.”

The regulatory updates are aligned with the rise in biotech innovation, drive to reduce time-to-market, and increased focus on patient needs, meaning that the bio/pharma industry has no choice but to change the way it operates, asserts Wood. “Leading players have realized that if they want different outcomes, they can’t keep doing things the way they’ve always done,” she says. “They need to be smarter, and more informed by the latest data, if they want to honour their commitment to patient centricity, and to safe, agile innovation.

All the trends discussed have a massive impact on the bio/pharma industry because of the many moving parts involved, adds Beczek. “The crucial part is that teams will have to increasingly work across functions not just in R&D but also closely with commercial, medical affairs, in country affiliates, with industry experts and healthcare professionals, expanding the end-to-end value chain of strategy and operations,” she concludes.

1. Guerrera, F. Europe Faces Dirtier Inflation Fight than US. 

, 6 Sep. 2023.
2. Smith-Meyer, B. High Interest Rates Have Hit Growth More than Expected, EU Says. 

Felicity Thomas is the European/senior editor for Pharmaceutical Technology Group.


Vol. 36, No. 1
January 2024
Pages: 10–12, 16

When referring to this article, please cite it as Thomas, F. Bridging Gaps and Investing in Change. 

European bio/pharma companies have no choice but to invest and adapt to the evolving industry needs.

Bridging Gaps and Investing in Change

3D Rendering of earth from space with run rising and ray light flare at horizon among glowing stars in galaxy. For wallpaper, sci fi, science or technology background | Image Credit: © knssr – Stock.adobe.com

A key political decision from 2023 that will undoubtedly impact the scientific communities of both the United Kingdom and European Union has been the allowance of the UK to re-enter the Horizon Europe research programme (see 

) (1). After voting to depart from the EU in 2016 and finally severing from the union in 2020, the UK has been excluded from full membership of certain EU projects, such as Horizon Europe. However, in March 2023, after the implementation of the Windsor framework (2,3), discussions commenced between the regions with the aim of getting the UK reinstated into the Horizon Europe research programme (4).

On 7 Sep. 2023, the UK and EU agreed upon a new bespoke deal, allowing the UK to participate in the Horizon Europe programme as well as the EU’s Earth observation programme, Copernicus (5,6). UK scientists were able to apply for grants and could bid to participate in projects from the date of the deal announcement (7 Sep. 2023), and from 1 Jan. 2024 are able to formally participate in Horizon Europe on par with EU counterparts with access to funding (6).

“The EU and UK are key strategic partners and allies, and [this] agreement proves that point. We will continue to be at the forefront of global science and research,” specified Ursula von der Leyen, president of the European Commission (EC) in a press release about the deal (6).

Horizon Europe is the European Union’s (EU’s) seven-year research and innovation funding programme that has succeeded the Horizon 2020 programme. Across seven years, researchers and innovators can apply to receive a grant from the programme’s budget which is worth a total of €95.5 billion, making it the largest civil research and innovation programme.

Consideration of funding for the programme is split into three main pillars: excellent science; global challenges and European industrial competitiveness; and innovative Europe. Any EU member state and associated countries can participate in the programme.

More information can be found on the European Commission’s website: 

https://research-and-innovation.ec.europa.eu/funding

Many in the scientific research communities of both the UK and Europe have been grateful to see this deal finalized. “[The] announcement on the UK’s association to Horizon Europe and Copernicus is a cause for celebration on both sides of the channel,” said Dr. Michael Spence, president and provost of University College London, in a news release (7). “Association to Horizon Europe will enable research, which makes a positive difference to people’s lives, supporting vital research and innovation networks and strengthening the UK’s position as an attractive destination for talent.”

The deal is a particular boon to those in the UK’s scientific community who were facing uncertainty due to limited funding opportunities for research. “The lack of agreement to allow the UK to continue to play a full role in the EU Horizon funding programme severely threatened [UK science] continued success,” explained Professor Liam Smeeth, director of the London School of Hygiene and Tropical Medicine, in a news release (8). “My concern was particularly for the long term. With no new partnerships and collaborations being formed, there was a very real risk of the UK becoming isolated and insular.”

Mayer Schreiber, CEO of Discovery Park, emphasized the importance of collaboration in the science community. “Collaboration is vital to successful science and innovation and the funding and partnership opportunities the programme offers will bring considerable benefits to the UK and Europe,” he said in a press release (9).

Now that the UK is fully reinstated into the Horizon Europe programme, reforging research relationships that have been lost in the interim will be vital. According to Thomas Jørgensen, director for Policy Coordination and Foresight at the European University Association, trying to get the level of cooperation back to where it was prior to the UK’s removal from the programme will be the biggest challenge (10).

Adding to this challenge will be issues such as the UK and EU’s divergence in regulations and standards, such as the rules around digital platforms, and the European view on certain sensitive subjects, such as the development and sharing of strategically important technologies, asserted Jørgensen (10).

However, on a positive note, the hiatus from the programme has finally come to an end, and UK-based researchers are once again able to receive funding and lead research projects involving multinational consortia. “Joining the Horizon Europe programme is a huge win for the scientific research community, who have been pushing for resolution over the past few years,” specified Janet Valentine, executive director of the Association of the British Pharmaceutical Industry, in a response to the deal. “UK innovation and research depends on international collaborations, which are crucial for driving advancements in all areas of science, including the discovery and early development of new medicines and vaccines. The UK accession to Horizon enables the two sides to reinvigorate their longstanding partnership in R&D, and directly contributes to UK growth and competitiveness in the life sciences sector by making the UK an attractive destination for talented researchers” (11).

. Policy Paper, 27 Feb. 2023.
3. European Commission. Protocol on Ireland and Northern Ireland: The Windsor Framework. 

 (accessed 10 Jan. 2024).
4. Gallardo, C. Britain and EU Agree Draft Horizon Deal. 

, 5 July 2023.
5. UK Gov. UK Joins Horizon Europe Under a New Bespoke Deal. 

, Press release, 7 Sep. 2023.
6. European Commission. EU-UK Relations: Commission and UK Reach Political Agreement on UK Participation in Horizon Europe and Copernicus. 

, Press Release, 7 Sep. 2023.
7. UCL. Rejoining Horizon Europe is a ‘Cause for Celebration’. News Release, 7 Sep. 2023.
8. LSHTM. Expert Comment—UK Rejoins Horizon Programme. News Release, 8 Sep. 2023.
9. Discovery Park. Discovery Park Welcomes Decision to Re-Join EU Horizon Science Programme. 

, 7 Sep. 2023.
10. Jørgensen, T. So the UK is in Horizon Europe—Now What? Research Professional News, 19 Sep. 2023.
11. ABPI. ABPI Response: UK Joins Horizon Europe. News Release, 7 Sep. 2023.

Felicity Thomas is the European/senior editor, and Jill Murphy was the former editor for Pharmaceutical Technology Group


Vol. 36, No. 1
January 2024
Pages: 32–33

When referring to this article, please cite it as Thomas, F.; Murphy, J. Bringing Collaboration to the Horizon Once More. 

UK scientists are once again able to participate in the Horizon European research programme thanks to a deal struck in 2023.

Bringing Collaboration to the Horizon Once More

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Across the Western world, the number 13 is commonly considered to be unlucky; so much so that in many aspects of life, such as hotel floors, aeroplane seat rows, and even house numbers, the use of 13 is avoided completely. Popular theories about the basis for this superstitious belief include the arrival of Judas Iscariot at the Last Supper and the Norse god Loki at a dinner party in Valhalla, as both were the 13th guest, and both are associated with negative outcomes (1).

However, in 2024, for the bio/pharma industry, 13 may prove to be lucky, certainly for the companies whose drugs have been revealed as ‘ones to watch’ in Clarivate’s latest annual report (2). Each year, analysts from Clarivate evaluate drugs in development and discern, using certain factors, which ones they believe will be blockbusters (reaching $1 billion in annual sales) or will be therapeutic game changers.

“The fundamentals underpinning the biopharma sector have never been stronger, with new technologies fuelling medical advancements and providing treatment options to patients with previously unmet needs,” said Mike Ward, global head of Thought Leadership, Life Sciences and Healthcare, Clarivate, in a press release (3). “Leveraging deep industry expertise and comprehensive therapeutic area differentiated data, this year’s 

 report identifies innovative medicines based on recent scientific breakthroughs poised to have extraordinary impacts on patient outcomes.”

From their analysis for 2024, the Clarivate experts created a list of 13 late-stage therapies that cover a wide number of conditions, including breast cancer, multiple myeloma, sickle cell disease, and respiratory syncytial virus (RSV). The ‘Drugs to Watch’ for 2024 are: 

aflibercept (high dose; EYLEA HD)—developed by Bayer and Regeneron Pharmaceuticals Inc. for the treatment of we age-related macular degeneration, diabetic macular oedema, or diabetic retinopathy

budesonide (TARPEYO/Kinpeygo/Nefecon)—developed by Calliditas Therapeutics AB, Everest Medicines and STADA Arzneimittel AG to reduce proteinuria in adults with IgA nephropathy

datopotamab deruxtecan (Dato-DXd)—developed by AstraZeneca and Daiichi Sankyo to treat metastatic non-small cell lung cancer or metastatic HR-positive/HER2-negative breast cancer

efanesoctocog alfa (ALTUVIIIO/BIVV001)—developed by Sanofi (Bioverativ Therapeutics Inc) and Swedish Orphan Biovitrum AB (Sobi) to reduce therapy burden for haemophilia A patients

ensifentrine (RPL554)—developed by Verona Pharma to reduce exacerbations in moderate to severe chronic obstructive pulmonary disease without systemic side effects

exagamglogene autotemcel (CASGEVY/exa-cel)—developed by CRISPR Therapeutics and Vertex Pharmaceuticals Inc. to treat severe sickle cell disease

lovotibeglogene autotemcel (Lyfgenia/lovo-cel/formerly LentiGlobin)—developed by CRISPR Therapeutics and Bluebird Bio to treat severe sickle cell disease and transfusion-dependent beta-thalassemia

mirikizumab (Omvoh/LY-3074828)—developed by Eli Lilly and Company to treat Crohn’s disease and moderately to severely active ulcerative colitis

niraparib + abiraterone acetate (AKEEGA)—developed by Johnson & Johnson Innovative Medicine to treat deleterious or suspected deleterious BRCA-mutated, metastatic castration-resistant prostate cancer

RSVpreF (ABRYSVO/PF-06928316)—developed by Pfizer Inc. as a vaccine to prevent lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV)

RSVpreF3 (AREXVY/GSK-3844766A)—developed by GSK plc as a vaccine to prevent LRTD caused by RSV

talquetamab (TALVEY)—developed by Johnson & Johnson Innovative Medicine to treat relapsed or refractory multiple myeloma

zolbetuximab (IMAB362)—developed by Astellas Pharma Inc. to treat HER2-negative, CLDN18.2-positive unresectable locally advanced or metastatic gastric or gastroesophageal junction adenocarcinoma.

If prior year’s predictions and analyses by Clarivate experts are anything to go by, then 13 might very well be considered lucky in bio/pharma for 2024.

Maranzani, B. What’s So Unlucky About the Number 13? 

Clarivate. Clarivate Identifies Thirteen Potential Blockbuster Drugs and Gamechangers in Annual Drugs to Watch Report. Press Release, 8 Jan. 2024.

When referring to this article, please cite it as Thomas, F. Unlucky for Some. 

Clarivate has released its 2024 Drugs to Watch report, highlighting 13 therapies with great potential.

Unlucky for Some

Adapting to Solid Dosage Development and Manufacturing Needs

In this exclusive Drug Digest video interview, Felicity Thomas, Europe/senior editor, 

Group, interviews experts about trends shaping solid dosage drug development and manufacturing. During this episode, Felicity chats with Jan Vertommen from Lonza at CPHI Barcelona about key trends, such as advances to help overcome solubility issues and the increasing importance of sustainability, as well as the role of a CDMO in solid dosage development and manufacturing. Additionally, Dave DiProspero from CRB Group details the progression of oral solid dosage manufacturing and the transformation of continuous processes in the sector.

Jan Vertommen, Vice President, Head of Commercial Development, Small Molecules at Lonza.

Jan Vertommen currently serves as Vice President, Head of Commercial Development, Small Molecules at Lonza. Jan has more than 25 years of experience in operational and commercial roles in the pharmaceutical industry. He joined Lonza in 2003 and held operational positions in product development as Head of Product Development and as Site Leader of manufacturing sites, as well as business-oriented positions in business and commercial development. Jan received his degree of pharmacist and made his PhD thesis on pharmaceutical delivery technologies at the Catholic University of Leuven, Belgium. He has authored several publications in scientific journals and has given numerous presentations at scientific symposia on pharmaceutical dosage form development and manufacturing.

Dave DiProspero, Senior Fellow—Director of Pharmaceutical Process Technology, CRB Group.

Dave DiProspero has 25 years of pharmaceutical engineering experience with a specialty in oral solid dose (OSD) form manufacturing operations/facilities/equipment/technology. His primary expertise is in internationally regulated, CGMP operations with a strong engineering, process, containment, equipment, material handling and facility systems integration background. Dave is experienced in site master planning, conceptual/preliminary/detailed facility design, engineering, procurement, qualification, project management and facility assessments. He is a long-term member of the International Society for Pharmaceutical Engineering and has chaired numerous committees–many related to education. Dave is currently the chair of the Facility of the Year Committee and steering committee advisor of the OSD Community of Practice. He is the steering committee manager for the OSD Baseline Guide Volume 3 revision effort.

This episode of Drug Digest is sponsored by:

Drug Digest is a tech talk video series with the 

 editors, who interview industry experts to discuss the emerging opportunities, obstacles, and advances in the pharmaceutical and biopharmaceutical industry for the research, development, formulation, analysis, upstream and downstream processing, manufacturing, supply chain, and packaging of drug products.

 APIs, Excipients, and Formulation Advances

Videos

In this episode of Drug Digest, Pharmaceutical Technology’s European/Senior Editor, Felicity Thomas, chats about the trends shaping solid dosage drug development and manufacturing with Jan Vertommen from Lonza and Dave DiProspero from CRB Group.