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Feliza Mirasol is the science editor for 

On Aug. 21, 2023, Gilead Sciences announced that FDA has placed a partial clinical hold on the company initiating new patients into US studies for magrolimab, an investigational anti-CD47 immunotherapy, to treat acute myeloid leukemia (AML). FDA’s decision follows a previous announcement in which a Phase III clinical study was discontinued that was studying magrolimab in higher-risk myelodysplastic syndromes (1).

According to a Gilead company press release, screening and enrollment of new study participants under US investigational new drug application (IND) 147229 and a US expanded access program will be paused, effective immediately. However, patients who are already enrolled in AML clinical studies may continue to be treated and monitored, according to study protocol. Meanwhile, studies of magrolimab in solid tumors may continue and are not impactd by this FDA action.

Global regulatory authorities and clinical trial investigators involved in the studies have been informed of FDA’s decision. Gilead is working with regulatory authorities to determine the next steps needed to release the partial clinical hold.

Magrolimab is an investigational molecule that has not been approved anywhere globally, and Its safety and efficacy have not yet been established. In addition to AML, magrolimab is being developed as a potential treatment for myeloid malignancies, lymphoma, myeloma, head and neck cancer, colorectal cancer, lung cancer, and breast cancer.

1. Gilead Sciences, Gilead To Discontinue Phase 3 ENHANCE Study of Magrolimab Plus Azacitidine in Higher-Risk MDS. Press Release, July 21, 2023.

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FDA has halted enrollment in clinical studies for Gilead Sciences’ magrolimab, a biologic treatment in development for acute myeloid leukemia.

FDA Halts Clinical Trial Enrollment for Gilead’s Magrolimab in AML Studies

Akari Therapeutics, a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, announced on Aug. 18, 2023 its plan to establish its US headquarters in Boston, Mass. According to a company press release, the office will support expansion efforts in preparation for the beginning of enrollment in two registrational Phase III clinical trials for the recombinant protein therapeutic, nomacopan, in pediatric and adult hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). The expanded operations will also support the start of proline/alanine/serine (PAS)-nomacopan clinical trials in geographic atrophy (GA).

Nomacopan has been tested in other clinical trials, including one for treating bullous pemphigold. The study findings suggested that a larger randomized clinical trial is needed to confirm the safety profile of the molecule to further be considered as a therapeutic option for bullous pemphigold (1).

“Boston is a vibrant epicenter of biotech innovation in the [United States] providing Akari access to … talent and other resources as we continue to build our US capabilities, advance our pivotal clinical trials, and work to realize the promise of nomacopan on behalf of patients and our investors,” said Rachelle Jacques, president and CEO, Akari, in the press release.

Enrollment for the Phase III clinical trials of nomacopan in pediatric and adult HSCT-TMA is expected to begin in late 2023 and 2024, respectively. The clinical trials of long-acting PAS-nomacopan as a potential treatment for GA is expected to start in 2024.

1. Sadik, C.D.; Rashid, H.; Hammers C. M.; et al. Evaluation of Nomacopan for Treatment of Bullous Pemphigoid: A Phase 2a Nonrandomized Controlled Trial. 

 (6), 641–649. DOI:10.1001/jamadermatol.2022.1156

The expanded operations will also support the start of proline/alanine/serine (PAS)-nomacopan clinical trials in geographic atrophy (GA).

Akari Therapeutics Establishes US Headquarters in Boston

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On Aug. 16, 2023, the European Medicines Agency (EMA) released a press statement to announce that their Pharmacovigilance Risk Assessment Committee (PRAC) is reviewing data on the possible risk of neurodevelopmental disorders (NDDs) developing in children because of paternal use of valproate medicines. The data being assessed is from a retrospective observational study conducted by companies as an obligation after a previous review of valproate use during pregnancy. The study compared the development of NDDs (including autism spectrum disorder) in children from men taking valproate with the development in children from men taking lamotrigine or levetiracetam (other epilepsy treatments). It was conducted in multiple registry databases in Denmark, Sweden, and Norway.

The results may indicate an increased rate of NDD development in children born to men taking valproate three months from conception, but PRAC found several important limitations with the data from the study. It had questions about the definition of NDDs used in the study, and the specific type of epilepsy the fathers had, as higher dosages of valproate can be prescribed more frequently for certain types of epilepsy associated with NDDs. Additionally, after the results were submitted, the companies also informed PRAC on errors in the Norwegian database.

Because of these limitations, PRAC requested the companies to submit analyses of corrected data as well as more information as soon as possible. PRAC will review the new data when it is available before it makes an EU-wide recommendation. In the meantime, some Member States are using interim national recommendations. EMA reminded male patients taking valproate to not stop taking their medicine without consulting their doctor, as symptoms from their epilepsy or bipolar disorder could worsen, and sudden discontinuation could trigger seizures. Previous recommendations of women during pregnancy to avoid exposure to valproate remain in place.

The assessment aims to determine whether valproate use in men could lead to neurodevelopmental disorders in their children.  

EMA Reviews Data on Paternal Exposure to Valproate

Stuart Malcolm, head of Standards, Efficiency, and Automation at Veramed, spoke with Grant Playter, associate editor, on-site at the REV4 data conference in New York City. Stuart spoke at length on the intersection of statistical programming and clinical trials and how the industry plans to leverage data, automation, visualization, and artificial intelligence technologies going forward.

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Stuart Malcolm, head of Standards, Efficiency, and Automation at Veramed, speaks on how advancing technologies are shaping clinical trials.

How Breakthrough Technologies are Shaping Clinical Trials with Stuart Malcolm

Amicus Therapeutics announced on Aug. 15, 2023 that the Medical and Healthcare Products Regulatory Agency (MHRA) of the United Kingdom has granted marketing authorizations for Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for adults living with late-onset Pompe disease. The indication for Pombiliti is a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD), according to the press release.

Pompe disease is described as an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Further, reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is assumed to lead to the clinical manifestations of Pompe disease. According to the release, late-onset Pompe disease can be severe and debilitating with progressive muscle weakness throughout the body that worsens over the time.

Pombiliti + Opfolda is a two-component therapy that consists of cipaglucosidase alfa, which is a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining capacity for processing into the most active form of the enzyme. Clinical studies demonstrated that Pombiliti + Opfolda was associated with improvements in both musculoskeletal and respiratory measures, according to the press release.

“The MHRA approvals for Pombiliti and Opfolda are a major step forward for adults in the UK living with late-onset Pompe who are seeking new treatments. We are grateful to the global Pompe community who have helped advance this therapy, especially the patients, families, and physicians who participated in our clinical studies,” said Bradley Campbell, president and chief executive officer of Amicus Therapeutics, in a press release. “The speed in which NICE recommended reimbursement of Pombiliti and Opfolda is reflective of the UK’s Innovative Licensing and Access Pathway, the data behind Pombiliti and Opfolda, the strong collaboration with the reimbursement authorities, and the Amicus commitment to bring this therapy to those living with Pompe disease as quickly as possible.”

The decision was based off clinical data from the Phase III study, PROPEL, which is known as the only trial in LOPD to analyze both ERT-naïve and ERT-experienced participants in a controlled setting.

“From the positive uptake of the Early Access to Medicines Scheme, we have already seen the impact that this treatment is having for patients,” said Professor Mark Roberts, consultant neurologist at the Greater Manchester Neurosciences Unit at Salford Royal NHS Foundation Trust, in a press release. “Having widespread access to this treatment is an exciting development for the Pompe community, giving HCPs and patients a new option that exhibits a novel mode of action.”

Additionally, Pombiliti + Opfolda has demonstrated fulfillment of the Great Britain orphan designation criteria and will be added to the Orphan Register held by the MHRA, according to the press release.

The indication for Pombiliti is a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD).

Amicus Therapeutics Launches Pompe Disease Therapy in United Kingdom

On Aug. 15, 2023, US-based Chiesi Global Rare Diseases, a business unit of the Chiesi Group, announced that it has entered into a co-development agreement with Aliada Therapeutics, a US-based biotechnology company focused on developing a novel blood-brain barrier (BBB)-crossing platform technology. Under the collaboration, the companies will focus on multiple enzyme cargoes that have been modified using Aliada's Modular Delivery (MODEL) platform. The goal is to establish drug delivery systems that will allow large-molecule therapeutics to cross the BBB, particularly for the treatment of lysosomal storage disorders (LSDs).

Aliada’s MODEL platform harnesses the endogenous cell transport mechanisms in brain endothelial cells to efficiently move large-molecule therapeutics across the BBB. The technology enables high therapeutic exposure in the brain, according to a company press release. According to the release, the MODEL platform demonstrates advantages over competing approaches as it offers a broad design landscape that confers an ability to optimize therapeutics for both central nervous system (CNS) delivery and downstream functionality.

"Our commitment to the development of new treatment options for people living with [LSDs] is global, as evidenced by recent regulatory approvals," said Giacomo Chiesi, head of Chiesi Global Rare Diseases, in the press release. "Many LSDs have CNS involvement. With this collaboration, we are expanding our strategy and presence in BBB-crossing technologies and hope to leverage our know-how in LSDs to support the development of an effective and differentiated drug delivery platform. We are especially proud to advance this important research with Aliada, a partner with vast experience in neuro drug development and biologics delivery."

"We are excited to partner with Chiesi to develop improved therapeutics for patients living with LSDs, who currently lack treatments that can readily access the brain," said Adam Rosenberg, CEO, Aliada Therapeutics, in the release. "We admire Chiesi's continued commitment to patients, exemplified by their two recent FDA approvals. This collaboration will allow Aliada to demonstrate the diverse capabilities of our MODEL platform, which enables us to efficiently transport a diverse range of therapeutic cargoes into the brain."

With this agreement, Chiesi Global Rare Diseases will co-develop drug delivery systems with Aliada Therapeutics for large-molecule therapeutics that can cross the blood-brain barrier.

Chiesi Global Rare Diseases and Aliada Therapeutics Partner to Advance Blood-Brain Barrier-Crossing Platform Technology for Drug Delivery

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On Aug. 10, 2023, drug creation company Absci Corporation (ABSI) announced that its collaborative effort with the California Institute of Technology (Caltech) has received a grant from the Bill & Melinda Gates foundation. Led by Dr. Pamela Bjorkman, the collaboration combines research capabilities and advanced technological expertise in structural biology, protein design, synthetic biology, and generative AI to discover affordable HIV vaccinations. The teams will work to develop a novel HIB therapy that “that first exposes and then binds to a highly conserved epitope binding site on HIV-1 to potentially both treat and protect against infection from all strains of the virus,” as described in a press release by Absci.

While Antiretroviral therapies have helped those with the HIV virus live longer and healthier lives, in more than forty years after the AIDS pandemic began no vaccine or cure exists to completely cure HIV. The cost and inaccessibility of these drugs, which must be taken for life, also mean that treatment is often out of reach from millions of people from low-income and marginalized communities. Absci and Caltech aim to focus on the affordability, scalability, and accessibility of HIV therapeutic vaccinations.

“We are thrilled to receive this grant from the Bill & Melinda Gates Foundation,” said Dr. Stephen Mayo,Bren Professor of Biology and Chemistry and Merkin Institute Professor at Caltech and project co-leader. “We’re committed to making transformative contributions to society through research and innovation, and we are excited to partner with Absci, who has developed a powerful de novo AI antibody platform that is helping to unlock new therapeutic possibilities. This collaboration with Absci allows us to combine our expertise and work towards a common goal of developing affordable HIV therapeutic vaccinations that can save lives and bring hope to millions.”

A major grant from the Bill & Melinda Gates Foundation will bolster efforts to fight the global HIV/AIDS epidemic.

Absci and Caltech Collaborate to Accelerate Affordable HIV Therapeutic Vaccine

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

Chris Spivey, editorial director, hosts a wide ranging discussion on the COVID-19 pandemic response, future government communications, and healthcare funding for mRNA with experts Dr. Kate Broderick, Chief Innovation Officer, Maravai LifeSciences and Dr. Tom Madden

President & CEO at Acuitas Therapeutics. 

Highlights include supply chain modalities, ensuring a fully trained manufacturing workforce, combining delivery with gene editing innovations, epigenetic approaches to modulate gene expression moving toward the clinic. We conclude with the huge potential for RNA expression of monoclonal antibodies and protein replacement applications.

, chief innovation officer at Maravai LifeSciences has extensive experience and expertise in leading and liaising with multi-disciplinary groups from discovery and R&D to engineering and clinical teams. She brings strong and broad scientific expertise to TriLink Biotechnologies, which covers multiple areas, including gene delivery, medical devices, gene therapies for the treatment of various infectious diseases, cancer immunotherapies and vaccine development. Additionally, she has extensive experience with non-viral delivery systems for a wide range of vaccine targets and cancer immunotherapies.

, president & CEO of Acuitas Therapeutics, is a world-renowned expert in the area of nanotechnology. Dr. Madden co-founded Acuitas Therapeutics in February 2009 and has guided the company into its position as a global leader through the development and application of lipid nanoparticle (LNP) technology. Acuitas Therapeutics partners with leading pharmaceutical and biotechnology companies and prestigious academic institutions around the world, providing its proprietary LNP delivery technology to enable new drugs based on nucleic acid therapeutics.

 presents the Drug Solutions podcast, where the editors will chat with industry experts from across the pharmaceutical and biopharmaceutical supply chain. Join us as experts share insights into your biggest questions—from the technologies, to strategies, to regulations related to the development and manufacture of drug products.

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An mRNA conversation about the recent past, current hot topics, and probable future.

Drug Solutions Podcast: mRNA Past, Present, and Future

In this excerpt from the most recent episode of the 

, Chris Spivey, editorial director, hosts a panel on advances in mRNA. There is promise of personalized cancer vaccines through the inherent nimbleness of mRNA platform. From a manufacturers point of view, how can we best to automate, miniaturize and speed up performance?

For the complete discussion, check out the full podcast episode 

Experts discuss the promise of personalized cancer vaccines through the inherent nimbleness of mRNA platform.

Miniaturing Personalized Cancer Vaccines Through mRNA

Parexel, a clinical research organization (CRO), and Partex, a biopharmaceutical company that has developed a digital pharma platform, announced a preferred strategic alliance on Aug. 15, 2023. According to a company press release, the goal of the alliance is to leverage artificial intelligence (AI)-powered solutions to accelerate drug discovery and development. According to a company press release, Parexel’s experience in Phase I to IV clinical development and Partex’s big data and AI capabilities are expected to complement one another in this endeavor.

Under the agreement, clinical trial execution through Partex will be managed by Parexel as the company’s preferred CRO partner. The parties will collaborate on improving clinical trial execution through the Partex-validated AI platform, which is designed to add to and expand Parexel’s existing AI tools and capabilities. According to the release, the companies ultimately hope to further advance drug developers’ ability to understand the probability of clinical success of assets within their portfolio, as well as recommend other disease indications for which their assets may be clinically viable.

"One of the biggest and most complex challenges in drug development is anticipating the investigational therapies that will be safe and effective treatment options,” said Jamie Macdonald, CEO, Parexel, in the release. “Our innovative alliance with Partex helps to address this challenge by bringing to the forefront those assets with the strongest probability of clinical success, in turn enabling customers to focus their time and resources where it is most beneficial to patients.”

"By combining Partex's … AI capabilities with Parexel's renowned clinical research expertise, we aim to accelerate the delivery of safe and effective therapies to patients worldwide, ultimately transforming the biopharmaceutical landscape,” said Gunjan Bhardwaj, CEO, Partex, in the release. “We eagerly anticipate the unveiling of new value-generating approaches and groundbreaking innovations resulting from this collaboration.”

The collaboration between Parexel and Partex is designed to leverage artificial intelligence-powered solutions to accelerate drug discovery and development.

Featured Content

Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Behind the Headlines Episode 28

Ask the Expert

Drug Digest December 2025

A “Plan, Prevent, Prove” Approach