Behind the Headlines Episode 27

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Feliza Mirasol is the science editor for 

Abzena announced on Jan. 16, 2023 the launch of AbZelect and AbZelectPRO, its new cell line development (CLD) platforms, designed to speed up the generation of production cell lines for the manufacture of antibodies and recombinant proteins. The platforms are intended to enable the rapid progress of complex biologic drug programs towards clinical trials and investigational new drug (IND) application filing.

According to a company press release, the CLD platforms enable the rapid generation and selection of high-yielding mammalian Chinese hamster ovary cell lines for producing therapeutic proteins and recombinant vaccines. AbZelect was designed for traditional protein programs, such as antibodies. Based on this design, it is optimized across three key areas: vector, host cell line, and process.

AbZelectPRO combines AbZelect with ProteoNic Biosciences’ 2G UNic vector technology, which boosts expression levels and generates higher-producing, stable cell lines that express proteins such as bispecifics, fusion proteins, and other novel modalities, according to the press release. These platforms are expected to shorten timelines from DNA to research cell banks to 15 weeks.

“The higher productivity that’s achievable with the AbZelect and AbZelectPRO platforms means we can produce more drug substances per manufacturing run. These efficiencies will not only expand our capacity and help to reduce the cost of goods for our customers but also reduce the time it takes to get new and critical therapies to patients in need,” said Matthew Stober, CEO, Abzena, in the press release.

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Abzena has launched its AbZelect platforms, designed for improving cell line development.

Abzena Launches New Cell Line Development Platforms

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The European Medicines Agency (EMA) published 

on Jan. 16, 2024, which gives an overview of the medicines that received recommendations for marketing authorization in 2023. The agency recommended 77 medicines, 39 of which were new active substances to the European Union. Two vaccines were recommended to protect respiratory syncytial virus (RSV). In addition, a new drug that uses clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 was recommended to treat two rare blood disorders.

Recommended approvals of specific note include the following:

Nine cancer treatments including Omjjara (momelotinib), the first treatment for myelofibrosis; Columvi (glofitamab) and Tepkinly (epcoritamab) for the treatment of diffuse large B-cell lymphoma; and Elrexfio (elranatamab) for the treatment of adult patients with relapsed or refractory multiple myeloma

Aqumeldi (enalapril maleate), which treats heart failure in children up to 18 years

Skyclarys (omaveloxolone) for the treatment of Friedreich’s ataxia

Arexvy (recombinant, adjuvanted) to protect adults aged 60 years and older against lower respiratory tract disease caused by RSV.

Seventeen orphan medicines were recommended for authorization that included treatments for cancer, chronic hypoparathyroidism, sickle cell disease, Duchenne muscular dystrophy, and a variety of other conditions.

Three medicines were recommended under the agency’s Priority Medicines (PRIME) program: cancer treatments, Elrexfio and Talvey; and Casgevy for the treatment of sickle cell disease.

There were 77 extensions for indications, which included 38 drugs for pediatrics.

Three medicines received negative approval from the agency in 2023: Albrioza (sodium phenylbutyrate/ursodoxicoltaurine) for the treatment of amyotrophic lateral sclerosis; Lagevrio (molnupiravir) for the treatment of COVID-19 in adults; and Sohonos (palovarotene), to treat fibrodysplasia ossificans progressive.

The agency monitors authorized medications after approval for quality and safety and provides industry with advice. Agency advice given in 2023 includes, but is not limited to, the following:

It was recommended that Adakveo (crizanlizumab) no longer be used to “prevent painful crises in patients aged 16 years and older with sickle cell disease, a genetic condition in which the red blood cells become rigid and sticky and change from being disc-shaped to being crescent-shaped (like a sickle).”

EMA reminded industry to use measures to reduce the risk involved in the use of fluoroquinolone antibiotics.

A lower dose of Olumiant (baricitinib) was recommended for patients “at higher risk of blood clots, cardiovascular conditions, and cancer in line with the dosing recommendations for other JAK [Janus kinase] inhibitors used to treat several chronic inflammatory disorders.”

EMA also suspended marketing authorization for more than 350 generic drugs that were tested by the contract research organization, Synapse Labs Pvt. Ltd., after a good clinical practice inspection found that supporting data were lacking or insufficient to show bioequivalence.

The agency recommended 77 drugs for marketing authorization in 2023, including 39 new APIs.

EMA Looks Back at Medicines Approved in 2023 

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On Jan. 8, 2024, Calypso Biotech, a European immunotherapy biotech company focused on the research and development of novel biologics, announced it had agreed to be acquired by Novartis. Calypso’s shareholders are set to receive an upfront payment of $250 million and are eligible to receive development milestones of up to $175 million, based on reaching certain milestones.

Calypso, a spin-out from Merck, is engaged in the research and development of monoclonal antibodies for a range of autoimmune indications, with a specialization in IL-15 biology. IL-15, according to Calypso, is a broad, untapped immune axis that controls barrier function and downstream immune cascades in many chronic autoimmune diseases. Calypso’s lead product candidate. CALY-002 is a potential best-in-class therapeutics antibody that binds to and neutralizes Interleukin-15.

This acquisition will give Novartis full rights to CALY-002, which it intends to explore further across a wide range of autoimmune indications with unmet medical need. CALY-002 is currently in a Phase Ib trial in patients with celiac disease and eosinophilic esophagitis.

“We are excited for this transaction with Novartis, a company with relentless commitment to the development of innovative therapies for autoimmune conditions. As part of the Novartis portfolio, CALY-002 is in the best position to be developed effectively, so that it can promptly address unmet medical needs in multiple indications,” said Alain Vicari, chief executive officer and co-founder of Calypso.

Bernard Coulie, chairman, Calypso, added, “The transaction with Novartis constitutes the high point in the development path of CALY-002 for the Calypso team. Calypso has established a significantly de-risked profile for CALY-002 as a potential best-in-class therapeutic anti-IL-15 antibody.”

“Novartis is committed to bringing innovative treatment options forward for patients living with immunological diseases,” said Richard Siegel, head of Immunology Research at Novartis in a Calypso press release. “We’re thrilled to add Calypso’s potential best-in-class antibody to our Immunology pipeline and explore it in a spectrum of autoimmune indications.”

The acquisition will give Novartis full rights to CALY-002, Calypso’s lead product candidate.

Novartis Acquires Calypso Biotech for up to $425 Million

FDA’s Center for Drug Evaluation and Research (CDER) published its 

 report on Jan. 16, 2024 highlighting new drugs approved in 2023. CDER approved 55 novel drugs during the year and expanded indications or patient populations for previously approved products.

The approved drugs treat a variety of diseases including COVID-19, respiratory syncytial virus (RSV), HIV, neurological conditions, anemia, weight management, type 2 diabetes, and endocrine disorders. Drugs were also approved that treat opioid addiction, three of which were over-the-counter opioid overdose reversal drugs.

More than half of the novel drugs approved were done so under the orphan drug designation program for rare diseases. Rare disease treatments include those for Friedreich’s ataxia, paroxysmal nocturnal hemoglobinuria, and activated phosphoinositide 3-kinase delta. Treatments were also approved for rare cancers such as mantle cell lymphoma and nasopharyngeal carcinoma.

There were five biosimilars approved in 2023 that included three for reference products that did not yet have a biosimilar. This brings the total of biosimilars approved by the agency to 45.

CDER also details in the report initiatives taken to create efficiencies in its review process. The center met or exceeded its Prescription Drug User Fee Act goal dates for89% of drug approvals. It also approved 84% of approvals on the “first cycle” and approved 35 of the 55 novel drugs before other countries. Thirty-six of the 55 drugs were approved using one of the agency’s expedited programs.

 CDER Director Patrizia Cavazzoni stated, “Deciding whether to approve a drug is a collaborative, well-coordinated process. It often involves expertise from throughout our center and agency, as well as external parties. We take our regulatory decision-making seriously, because we know our drug approval actions affect the health and well-being of patients and consumers nationwide. I want to thank my colleagues for their dedication to reviewing and approving these drugs, which will benefit many people in years to come.”

FDA’s Center for Drug Evaluation and Research released its report on new drugs approved in 2023.

CDER Approved 55 Novel Drugs in 2023

Charles River Laboratories International announced the launch of its off-the-shelf replication/capsid (rep/cap) plasmids for the manufacture of adeno-associated virus (AAV) vectors during Advanced Therapies Week, held Jan. 16–19, 2024 in Miami Beach, Fla. These plasmids are designed to streamline AAV-based gene therapy programs, according to a company press release.

The addition of AAV rep/cap (RC2, 5, 6, 8, 9) follows a previously announced launch of the company’s lentiviral packaging and AAV helper plasmid products. According to Charles River, the ready-to-use plasmid products are produced and released according to batch production records. The products include chemistry, manufacturing, and controls guidance and a certificate of analysis to support investigational new drug and clinical trial application filing.

Standard off-the-shelf plasmids, such as helper and rep/cap plasmids required for AAV production, are immediately available, which can lower development costs, risks, and timelines for gene therapy developers, according to the press release. Charles River’s rep/cap plasmids have been used to support the production of AAV serotypes carrying various therapeutic transgenes at multiple development and good manufacturing practice production scales.

“The reduction in production time and improved efficacy of the supply chain will help accomplish our ultimate goal of delivering safe, effective therapies to patients, faster,” said Kerstin Dolph, senior vice-president, Manufacturing, Charles River, in the release.

Charles River’s off-the-shelf rep/cap plasmids are intended to simplify gene therapy supply chains.

Charles River Launches Off-the-Shelf Rep/Cap Plasmids for AAV Vector Manufacturing

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

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Taiwan’s largest pharmaceutical manufacturer, Bora Pharmaceuticals, has announced the approval by its Board of Directors for the acquisition of generics manufacturer, Upsher-Smith, which is based in Minnesota, USA, for a total of $210 million.

According to a Jan. 16, 2024 press release, this acquisition—the inaugural one for Bora in the US—marks a pivotal expansion for the company into the US market. Additionally, the acquisition creates additional capacity for Bora’s global contract development and manufacturing organization (CDMO) business and also completes the company’s capabilities to serve its customers throughout the entire value chain.

Upsher-Smith has two manufacturing facilities in Minnesota, one located in Plymouth and the other in Maple Grove. In combination, the two facilities are able to commercialize a broad range of dosage forms, including oral solid, powders, and liquids, in addition to packaging. Furthermore, the acquisition provides opportunities for Bora’s commercial sales operations in the US, TWi, through enhanced product development and marketing capabilities.

“This is a significant milestone for Bora Group, marking the most critical expansion of Bora’s presence in the US market and significantly enhancing Bora’s position as a global competitor with regard to its CDMO business and commercial Rx business,” said Bobby Sheng, chairman and CEO of Bora Group, in the press release. “Upsher-Smith’s competitiveness is a perfect match to Bora’s long-term growth—its newly-expanded, USFDA-approved manufacturing facilities, with a total capacity of 3.5 billion doses, will be Bora’s first local facility in the US, establishing a crucial stronghold for our global CDMO business.”

Closure of the acquisition is subject to the usual regulatory approvals. Once the deal has been officially closed, Bora will acquire 100% of Upsher-Smith through its wholly owned US subsidiary.

The acquisition marks a pivotal expansion for the company into the US market. 

Bora Pharmaceuticals Fuels Global Expansion with Acquisition in US

The European Medicines Agency (EMA) announced on Jan. 12, 2024 that its Pharmacovigilance Risk Assessment Committee (PRAC) was beginning a review of data on secondary malignancies related to T-cells for six approved chimeric antigen receptor (CAR) T-cell medicines. The committee is reviewing data from 23 cases of T-cell lymphoma or leukemia in the European Union’s EudraVigilance adverse event database.

According to EMA, secondary malignancies are those that develop when a cancer patient develops a second cancer that is different from the original cancer. This risk was considered at the time of authorization of the impacted drugs and included in risk management plans. The drugs have been monitored, and the marketing authorization holders are required to submit interim results from long-term and efficacy studies.

The impacted drugs—Breyanzi, Carvykti, Kymriah, Tecartus, and Yescarta—are approved in the EU and are used to treat blood cancers such as B-cell leukemia, B-cell lymphoma, follicular lymphoma, multiple myeloma, and mantle cell lymphoma in patients whose cancer has relapsed or has stopped responding to previous treatment. They are “personalized cancer immunotherapies where patients' white blood cells (T-cells) are reprogrammed and reinjected to attack the cancer.”

The agency is reviewing data on secondary malignancies related to T-cells for approved CAR T-cell medicines.

EMA Starts Safety Review of CAR T-Cell Medicines

Merck, known as MSD outside of the United States and Canada, and Harpoon Therapeutics, a US-based clinical-stage immuno-oncology company, announced on Jan. 8, 2024 that they have entered into a definitive agreement for Merck to acquire Harpoon via a Merck subsidiary. Merck will acquire Harpoon for $23.00 per share in cash, or a total equity value of approximately $680 million. The transaction is expected to close in the first half of 2024.

Under the agreement, Merck will gain Harpoon’s lead candidate, HPN328, a T-cell engager targeting delta-like ligand 3 (DLL3). DLL3 is an inhibitory canonical Notch ligand that has been shown to be expressed at high levels in small cell lung cancer (SCLC) and neuroendocrine tumors. HPN328 is currently being studied in a Phase I/II clinical trial as a monotherapy in patients with advanced cancers associated with the expression of DLL3. It is also being studied in combination with atezolizumab in patients with SCLC. Harpoon announced positive interim tolerability and response data in certain patients with SCLC and neuroendocrine tumors last October 2023.

Harpoon has developed a portfolio of novel T-cell engagers using the company’s proprietary Tri-specific T cell Activating Construct (TriTAC) platform. The TriTAC platform is an engineered protein technology that has been designed to direct a patient’s own immune cells to kill tumor cells. In addition, the company’s ProTriTAC platform involves the application of a prodrug concept to the TriTAC platform to create a therapeutic T-cell engager that is designed to remain inactive until it reaches the tumor.

Additional candidates in the company’s pipeline include HPN217, which targets B-cell maturation antigen (BCMA). HPN217 is currently in Phase I clinical development for treating patients with relapsed/refractory multiple myeloma. The company also has several preclinical-stage candidates, including HPN601, which is a conditionally activated targeting epithelial cell adhesion molecule (EpCAM) for treating certain patients with EpCAM-expressing tumors.

“At Merck, we continue to enhance our oncology pipeline through strategic acquisitions that complement our current portfolio and advance breakthrough science to help address the needs of people with cancer worldwide,” said Dean Y. Li, president, Merck Research Laboratories, in a company press release. “This agreement reflects the creativity and commitment of scientists and clinical development teams at Harpoon. We look forward to further evaluating HPN328 in innovative combinations with other pipeline candidates.”

“At Harpoon, we have always been committed to advancing our cancer immunotherapy candidates to improve the lives of patients. With Merck’s recognized leadership in oncology clinical development and global commercial footprint, our lead candidate, HPN328, is well positioned moving forward,” said Julie Eastland, president and chief executive officer, Harpoon Therapeutics, in the press release.

Through the acquisition of Harpoon Therapeutics, Merck will gain an investigational delta-like ligand 3-targeting T-cell engager under development for cancer treatment.

Merck to Acquire Harpoon Therapeutics in Deal Valued at $680 Million

Pharmapack Europe 2024 survey results have been released just prior to the event kicking off in France—the event will take place between Jan. 24 and 25 in the Paris Expo—revealing general consensus on growth for contract packaging. The results, which were announced in a Jan. 11, 2024 press release, show a slight improvement on those from 2023, with 94% of respondents feeling “highly positive” or “moderately positive” about growth prospects for the coming 12 months.

“2024 will see an increasing number of manufacturers launching specialty and biologic medicines, all requiring specialist packaging solutions to help them differentiate their offerings,” said Aurelio Arias, director, Thought Leadership at IQVIA, in the press release. “Moreover, this year it will be recognized that policy solutions are required to increase resilience, including an [European Union] Critical Medicines Act and the [World Health Organization’s] draft pandemic accord. The crucial point is that these changes to legislation seek to track supply and incentivise diversity of supply, which will necessitate greater packaging services to meet this increased demand. So, I concur that 2024 is likely to be a good growth year for the industry.”

The full survey results will be published in the Pharmapack Annual report along with the Sustainability Index, which has improved significantly in 2024 rising by 8.6%, indicating that the industry has shifted its focus back to sustainability as was the priority prior to the COVID-19 pandemic. Additionally, the report expects that those companies investing in greener innovations will experience higher revenue streams.

“All across drug delivery and packaging, we are seeing positive signs and an increased desire to do business, with green technology standing out as a notable trend,” Laura Indriksone, brand manager at Pharmapack Europe, in the press release. “This is shown by our sustainability index, with newer device and packaging innovations helping deliver increased revenue opportunities for early movers.”

Full survey results and the Sustainability Index will be published in the Pharmapack Annual Report during the 2024 event and on 

According to the Pharmapack Europe 2024 survey results, contract packaging will see growth over the coming 12 months.

Contract Packaging Set for High Growth in 2024

CellVoyant—an artificial intelligence (AI) first biotechnology company spun out of the University of Bristol—has raised £7.6 million in seed funding to speed up the development of novel cell therapies. According to a Jan. 16, 2024 press release, the funding was led by Octopus Ventures and included Horizon Ventures, Verve Ventures, and Air Street Capital as participants of the funding round.

The biotech’s primary aim is to use its platform, which combines advanced AI with live cell imaging, to develop novel stem cell therapies in a cost-effective way while also improving reliability and speed of development. While there are promising applications of stem cell therapies, the science behind these options is complex and unpredictable and development tends to have high attrition rates.

“Cell therapies have the potential to revolutionize the way we treat diseases that affect millions of people every year,” said Rafael E. Carazo Salas, CEO and founder of CellVoyant, in the press release. “By combining the latest advances in AI and live cell imaging, we can help bring these transformative treatments to the market quickly, reliably, and cost-effectively. [This funding] milestone validates the potential of our approach and will help us to accelerate our R&D capabilities.”

“University spin-outs like CellVoyant are at the heart of the UK’s thriving biotech ecosystem,” added Uzma Choudry, lead biotech investor at Octopus Ventures, in the press release. “CellVoyant sets a new standard in precision and reliability for predicting and controlling stem cell behavior, which will make cell therapies more accessible to those who need them. We are thrilled to invest into a company that is transforming how patients can benefit from life-changing treatments.”

With the seed funding raised, CellVoyant will scale up its operations, including doubling the team size over the next two years, expanding laboratory and experimental infrastructure, and advancing AI capabilities.

CellVoyant has raised £7.6 million in seed funding to speed up the development of novel cell therapies.

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