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More signs of alignment between startups, pharma, and payers. 

The Shrinking Disconnect in Digital Therapeutics

Pharma companies are increasingly turning to tech enabled behavioral-based interventions to help improve patient outcomes through their diagnosis, treatment, and coordination of care.


Digital Therapeutics Can Break Down Barriers to Innovation to Benefit Patients

AbbVie Pedro Valencia ADCs

PE: How is AbbVie using novel cancer biomarkers to develop potential first-in-class ADCs for the treatment of advanced solid tumors?

We're constantly looking for biomarkers that can help us to do two key things. The first is to tackle cancer cells. and spare normal cells. We’re looking for specific antigens that are over-expressing tumor cells, and you see limited expression in normal cells. We want to make sure that target that’s over-expressed is critical for the survival of the cancer cell. Once you block it, you can actually drive a cancer cell death. That’s what we're looking for. In that perspective, c-Met is one of those biomarkers that is over expressing multiple tumor types such as lung, colorectal, gastric, and other GA tumors. We as an industry have tried to tackle c-Met in the past but have been unsuccessful. It would actually be successful to tackle what we call met, which is a gene inside the cell.

There’s this thing called met amplification, where the cancer cell produces multiple copies of the gene, and around 30% of patients have it. We can actually tackle that with small molecules. But once the gene becomes a protein and gets over-expressed in the cell, that over-expression is something we haven’t been able to target. For example, we know that on lung cancer, c-Met over-expression is about 25% to 50% in colorectal cancer, and it could be anywhere from 30% to 70% depending how you measure it, so it’s quite prevalent.

With ADCs, we’re trying to tackle c-Met over-expression on the surface of cancer cells. That was novel, and we can do it through ADCs. 706 has a target called SEZ6, and it’s a target that is over expressed in neuroendocrine cells or neuroendocrine neoplasms. When you think about it, multiple tumor types kind of have a subset of cells that are neuroendocrine driven. Small cell lung cancer is one of the few that has everything driven by neuroendocrine cells. The beauty of SEZ6 is over-expressing those cells and limited expression in normal cells. So, we can tackle it with an ADC and drive the results that we're seeing today.

PE: ADCs have redefined the treatment of cancer in recent years. Can you please discuss the promise of this emerging class and what the future may hold?

At the end of the day, ADCs work like a trojan horse. You have a good antibody that cancer cells are happy to receive, but once you hook into the cancer cell receptor, you deliver the payload, which is pretty much chemotherapy. It’s actually a highly potent version of the regular chemotherapy cancer patients are given on a regular basis. So, from that perspective, our goal is to replace chemotherapy. Our grand goal with ADCs is to replace good old, decades old chemotherapy that is really toxic for some patients. That’s how we see the redefinition of cancer treatment in the future with ADCs. So, what do we see in the future? Well, if you think about it, chemotherapy is being given in combination with other agents. In the future, we see ADCs being given in combination with chemotherapy. There will be one with other agents such as immuno-oncology agents. So, if you combine the ADC and the PD1s, you can start seeing that synergy come together and kind of start really dreaming about cures for the patients. All in all, we're super excited about ADCs replacing chemotherapy. 

In an interview with Pharm Exec Associate Editor Don Tracy, Pedro Valencia, VP, Solid Tumor Pipeline Strategy & Execution, AbbVie, discusses using novel cancer biomarkers to develop potential first-in-class antibody drug conjugates (ADCs) and the promise that they offer. 

Utilizing Biomarkers to Develop First-in-Class Antibody Drug Conjugates to Treat Advanced Solid Tumors

The FDA has approved Samsung Bioepis’ Ospomyv (denosumab-dssb; SB16) and Xbryk (denosumab-dssb; SB16), biosimilars referencing Amgen’s Prolia and Xgeva, respectively. Ospomyv is indicated for osteoporosis treatment in postmenopausal women and men at high risk of fracture, as well as for patients undergoing androgen deprivation or aromatase inhibitor therapy. Additionally, Xbryk is approved for preventing skeletal-related events in patients with multiple myeloma and solid tumor bone metastases, treating unresectable giant-cell tumors of bone, and addressing bisphosphonate-refractory hypercalcemia of malignancy.

“The FDA approval of Ospomyv and Xbryk marks a key step in improving patient access and alleviating treatment cost for patients with osteoporosis and cancer-related bone loss in the US. By providing quality-proven biosimilars, we are helping to address a critical healthcare need and reduce the burden of skeletal fractures that impact patients’ quality of life,” said Byoungin Jung, VP, regulatory affairs team leader, Samsung Bioepis, in a press release. “This achievement underscores our commitment to healthcare innovation through biosimilars and our mission to meet the growing needs in critical therapeutic areas.”

Approval was based on results from Phase I and Phase III clinical trials, including analytical data, non-clinical data, and clinical data. This included a Phase I randomized, double-blind, three-arm, parallel group, single-dose trial, which demonstrated the pharmacokinetic (PK) equivalence between SB16, EU-sourced denosumab, and US-sourced denosumab in healthy male participants. Results of the study met all primary PK endpoints, including area under the concentration-time curve (AUC) from time zero to infinity, AUC from time zero to the last quantifiable concentration, and maximum serum concentration.

Additionally, a Phase III randomized, double-blind, multi-center study demonstrated equivalent efficacy and comparable safety, immunogenicity, PK, and pharmacodynamics. The primary endpoint was the percent change from baseline in lumbar spine bone mineral density at one year and a follow up of 18 months.

Samsung Bioepis warns that Ospomyv and Xbryk are contraindicated in patients with known hypersensitivity to denosumab products and hypocalcemia. Ospomyv is also contraindicated in patients who are pregnant. Adverse events (AEs) of Ospomyv include dermatologic reactions, severe joint, bone, and muscle pain, and serious skin infections. AEs of Xbryk include embryo-fetal toxicity, hypercalcemia, osteonecrosis of the jaw, and fractures to the vertebrae and femoral bone.

According to the International Osteoporosis Foundation, osteoporosis affects one in three women and one in five men over 50 years of age globally. In Europe, the United States, and Japan, it is estimated that a total of 75 million people are currently living with osteoporosis. For the United States, this accounts for around 10.2 million people, with 54 million currently at risk. Additionally, 80% of people with osteoporosis in the United States are women.

According to the Johns Hopkins Arthritis Center, 30% of Caucasian postmenopausal women in the United States have osteoporosis. Additionally, the prevalence of osteoporosis in Caucasian postmenopausal women is around 14% between 50 and 59 years of age, 22% in women between 60 and 69 years of age, 39% in women between 70 and 79 years of age, and 70% in women over 80 years of age.3 In men, the numbers decrease significantly, with 3.3% of men between 50 and 64 years of age developing osteoporosis and 5.7% in men over 65 years of age.

According to Samgsung Bioepsis, the approvals mark the company’s first FDA approval for endocrinology biosimilars.

1. FDA Approves Samsung Bioepis’ OSPOMYV™, XBRYK™ (denosumab-dssb), a Biosimilar to Prolia and Xgeva. 

February 15, 2025. Accessed February 18, 2025. https://www.businesswire.com/news/home/20250215814020/en/FDA-Approves-Samsung-Bioepis%E2%80%99-OSPOMYV%E2%84%A2-XBRYK%E2%84%A2-denosumab-dssb-a-Biosimilar-to-Prolia-and-Xgeva

Accessed February 18, 2025. https://www.osteoporosis.foundation/health-professionals/about-osteoporosis/epidemiology

Accessed February 18, 2025. https://www.hopkinsarthritis.org/arthritis-info/osteoporosis-info/

Accessed February 18, 2025. https://www.healthline.com/health/osteoporosis/prevalence-of-osteoporosis

Ospomyv, a biosimilar to Prolia, is indicated for postmenopausal women and men at high risk of fracture, while Xbryk, a biosimilar to Xgeva, is indicated for preventing skeletal-related events in multiple myeloma and solid tumor bone metastases. 

FDA Approves Samsung Bioepis Biosimilars Ospomyv and Xbryk 

Nitrosamines have become a significant concern in the pharmaceutical industry due to their potential carcinogenic risks. Regulatory agencies such as the FDA, EMA, and ICH have imposed strict guidelines to monitor and control nitrosamine impurities in drug products. Traditional analytical techniques like LC-MS/MS, while effective, can be time-consuming, expensive, and reliant on organic solvents. A more efficient alternative is emerging: Headspace-Selected Ion Flow Tube Mass Spectrometry (Headspace-SIFT-MS). This powerful method enables real-time, high-throughput screening of volatile nitrosamines in pharmaceuticals, offering a streamlined approach that reduces complexity and environmental impact.

Why Headspace-SIFT-MS is Transforming Nitrosamine Analysis

Headspace-SIFT-MS is a direct mass spectrometry technique that provides fast, highly sensitive, and selective quantification of nitrosamines in pharmaceutical samples. Unlike conventional chromatographic methods, it requires minimal sample preparation and no organic solvents, making it a more sustainable and practical alternative. By using soft chemical ionization with multiple reagent ions (H₃O⁺, NO⁺, and O₂⁺), this technique ensures accurate detection of volatile nitrosamines while minimizing interferences from complex sample matrices.

 – Provides results in minutes, significantly reducing batch release and regulatory compliance timelines.

 – Eliminates hazardous solvents such as methanol and acetonitrile, improving safety and sustainability.

 – Achieves low limits of quantitation (LOQ <10% of regulatory limits) with excellent linearity (R² > 0.99) for six nitrosamines.

 – Reduces manual labor through automated workflows, increasing laboratory productivity.

 – Meets performance criteria outlined in USP 〈1469〉, supporting industry standards.

Proven Performance in Nitrosamine Screening

Recent studies have shown that Headspace-SIFT-MS effectively detects six commonly regulated nitrosamines, including NDMA, NDEA, NDBA, NEIPA, NDIPA, and NMPA. The method has been assessed against United States Pharmacopeia (USP) 〈1469〉 guidelines, demonstrating very high repeatability, precision, and accuracy.

Reliable Results in Complex Drug Matrices

A study evaluating a finished drug product found that headspace-SIFT-MS produced consistent results, with average recovery rates of 115% for NDMA and 86% for NDEA, based on nine replicates. The technique successfully identified nitrosamines even in complex pharmaceutical formulations, reinforcing its reliability.

LOQ of 0.06 ppm (NDMA) and 0.03 ppm (NDEA)

Consistent recoveries across multiple replicates

Minimal impact from complex drug formulations

These findings confirm that headspace-SIFT-MS provides reliable and reproducible nitrosamine detection, even in challenging sample types.

How It Works: A Straightforward and Effective Process

Unlike LC-MS/MS, which requires extensive sample preparation, headspace-SIFT-MS streamlines the workflow with a direct approach:

 – Pharmaceutical samples are mixed with potassium carbonate to promote nitrosamine transfer into the headspace.

 – Samples are incubated at 80°C for 15 minutes, and the headspace is extracted.

 – The extracted gas is analyzed using reagent ions that selectively react with nitrosamines.

 – Results are processed using SyftAuditTracer software, delivering quantification in under four minutes.

 – The method supports rapid batch analysis, optimizing laboratory workflows.

Expanding Applications Beyond Pharmaceuticals

While primarily used for pharmaceutical impurity testing, headspace-SIFT-MS is also valuable in other industries:

 – Detecting nitrosamines in processed foods and beverages to ensure consumer safety.

 – Monitoring nitrosamine contamination in water sources and air emissions.

 – Identifying volatile nitrosamines in cosmetics, tobacco, and e-cigarette products.

Revolutionizing Pharmaceutical Quality Control

The Syft Tracer Pharm11 solution is changing the landscape of pharmaceutical quality control. By offering a fast, solvent-free, and user-friendly approach, it provides an effective alternative to conventional chromatographic techniques. This solution is ideal for routine impurity testing, ensuring that manufacturers stay ahead of regulatory requirements while improving efficiency.

As regulatory scrutiny of nitrosamines intensifies, the pharmaceutical industry must adopt faster, more sustainable, and cost-effective testing solutions. Headspace-SIFT-MS offers an advanced, environmentally responsible approach that enhances drug safety and streamlines laboratory workflows. With its rapid turnaround, automated analysis, and solvent-free operation, this technology is set to become a cornerstone of modern pharmaceutical impurity testing.

For a smarter, safer, and more efficient approach to nitrosamine detection, headspace-SIFT-MS is the way forward.

Nitrosamine Detection in Pharmaceuticals: A Faster, Greener, and More Effective Approach

Jen Butler, Chief Commercial Officer of Pleio, discusses the significant threat of misinformation to public health and how emotional appeal plays a part.

The Threat of Misinformation on Public Health

 video interview, Jen Butler, Chief Commercial Officer for Pleio, discussed the significant threat of misinformation to public health, citing a 2021 report by former Surgeon General Murthy. She highlighted that 70% of false news is shared more often than true news due to its emotional appeal. Butler emphasized the importance of verifying health information by directly contacting trusted sources like Cleveland Clinic, Mayo Clinic, and Kaiser Permanente. She also noted the role of pharma in supporting patient communities and the need for media and health literacy. Butler also suggested that peer-to-patient platforms can enhance patient engagement and adherence, particularly for chronic conditions.

What kind of threat is misinformation to public health?

I have to say, nowadays, it's a huge threat. Back in 2021, former Surgeon General Murthy published a report about confronting health misinformation, and this was four years ago. He acknowledged at that point, and this was coming off of COVID and the pandemic, and the role of misinformation and causing confusion and anxiety and growing mistrust with patients and the population, and the impact that that can have over public health efforts. He raised concern in this report about the changing information environment, and at the time, four years ago, he was really focusing on the social media outlets that we have in place, and the fact that engaging content isn't always it. I should say engaging content is really emotionally driven, and therefore there's opinions behind it. In this report, there is a reference to a study that 70% of false news is shared more often than true news because it has that appeal. And while this is not about fake news, I think that can lend itself to health information also, because it's really important for patients when they're feeling vulnerable to be able to find information and not question. And as you're questioning information, you start to get more anxious and overwhelmed about what's coming next.

Fast forward from where we were four years ago to today with the impact and prevalence of TikTok and AI and we've just ignited where former Surgeon General Murthy was starting with his view of this, of this surge of misinformation for patients. It's really tricky. It's too easy for patients to go into their feeds and start finding patients like them in their feeds or hearing influencers. And no matter what you Google or search for in these in the in these forums, you're going to find opinions, and that it's the tricky part is, it's comforting. It's comforting to see patients like yourself offering opinions. And I think there is a great wealth of awareness that can be raised, but information and validated information can't be trusted upon opinions, and I think that's where it's a slippery slope.

When you add in TikTok and AI — and AI again, shows amazing opportunity for information collection, information seekers, but we know that AI also has quote, unquote hallucinations that you don't know what information you're getting back is credible. So, it's wonderful to have access to all this information. I mean, at least patients aren't in the dark like we were maybe 10, 15, 20 years ago, but we also have to make sure that we know how to handle the information that's coming our way and be able to separate fact from opinion.

In this Pharmaceutical Executive video interview, Jen Butler, Chief Commercial Officer of Pleio, discusses the significant threat of misinformation to public health and how emotional appeal plays a part.

The biopharmaceutical industry has long been at the forefront of innovation. From the rise of precision medicine, to artificial intelligence technologies accelerating drug development, to the advent of small-molecule drugs, recent revolutions in pharmaceuticals are delivering better results for patients and more options for drug manufacturers. 

Now, emerging developments in antibody-drug conjugates (ADCs) for oncology; hopeful treatments on the rise for idiopathic pulmonary fibrosis (IPF), a long-elusive disease to target; the transforming—and overarching—promise of GLP-1 agonists; growing investment around advanced therapy medicinal products (ATMPs); and a concerted push for new approaches in antibiotics are challenging the boundaries of what’s possible. 

’s 20th Annual Pipeline Report profiles the most promising activity and efforts in development across these five therapeutic areas.

Cancer therapy has undergone a significant amount of research in recent years, with the global oncology drugs market expected to reach a value of $532.91 billion by 2031.

One new emerging oncology development is the rise of ADCs, which oncologists view as very promising.

Used in treating blood cancers and solid tumors, ADCs combine targeted therapy with chemotherapy to attack cancer cells while leaving healthy cells alone.

As the name implies, an antibody-drug conjugate consists of a monoclonal antibody that attaches to tumor antigens and a chemotherapy drug. A linker protein binds the two compounds together, keeping them connected as they travel to the tumor and releasing the chemotherapy drug where it needs to be.

Darja Irdam, PhD, is a partner at global insights agency Hall & Partners in London, a part of the Escalent Group. Irdam says that ADCs are an extremely promising new development in oncology that is opening new frontiers for targeting tumors and treating diseases that were previously untreatable. She explains that ADCs were the topic of the day at the 2024 annual American Society of Clinical Oncology meeting last spring in Chicago.

“One of the key trends that was discussed and that we’ll definitely see this year is better linkers for ADCs,” notes Irdam. “One of the core goals for linkers is stability. The stability of the linker is critical because it prevents the cytotoxic payload from being released too early. If the cytotoxic payload is released into the bloodstream, then the cancer cells aren’t targeted properly and the rest of the body is exposed to unnecessary toxicity.”

Irdam predicts that in addition to their oncology applications, ADCs will also become a major treatment for autoimmune conditions and infectious diseases. First, though, researchers will need to improve the compounds’ tolerability. Irdam says that the efficacy of an ADC depends on its dose, and patients often cannot tolerate high ADC doses.

Furthermore, Irdam predicts that the manufacturing process for ADCs will become simplified over time. The current process of manufacturing these types of treatments is quite complex, which means developing ADCs is prohibitively expensive. As manufacturers improve their processes and global supply chains adjust, Irdam predicts that ADCs will become more economically viable.

There are currently several ADCs in various stages of clinical development. In January 2024, BioNTech and DualityBio announced the beginning of a Phase III trial of their ADC candidate, BNT323/DB-1303. Designed to target metastatic breast cancer, the agent has shown promising results in treating HER-2 positive metastatic breast cancer in a combined Phase I/II study.

 A Phase III study, the DYNASTY-Breast02 study, is currently underway and is expected to finish in 2028.

Another ADC hopeful, AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan, recently finished Phase II trials. The TROPION-Lung05 trial examined the effects of the ADC in patients with locally advanced or metastatic non-small cell lung cancer.

 Based on the results of that study, AstraZeneca and Daiichi Sankyo have submitted a new biologics license application to the FDA for accelerated approval.

IPF is a difficult-to-treat condition because, as an idiopathic disease, its cause is unknown. Sujith Eramangalath, vice president of life sciences at Escalent, says the IPF space mostly focuses on anti-fibrotic treatments that are designed to reduce inflammation and halt the advancement of the disease.

“There are about 15 drugs in the pipeline, but only two are on the market,” notes Eramangalath. “The activity in this space is fairly low, but where I do see activity in the future is a lot of diagnosis happening via biomarkers. One of the challenges with IPF is that there’s no genetic-based research or integration happening. There’s no extensive gene-based or microbiome-based therapy that’s been happening at this point, at least not in a commercial sense.”

Eramangalath says that one of the most recent IPF drugs in development was pamrevlumab by FibroGen. He points out that the drug, a monoclonal antibody, failed to meet its Phase III endpoints, and the trial showed mixed results. Still, other companies are pushing ahead with their own takes on IPF drugs.

For example, taladegib, also known as ENV-101, is a hedgehog signaling pathway inhibitor being developed by Endeavor BioMedicines. The hopeful treatment is currently in Phase IIB clinical trials.

“The most positive thing about it is that it shows reversal of lung fibrosis,” says Eramangalath. “So far, all of the drugs on the market reduce symptoms; in Phase IIA, this drug showed reversal of lung fibrosis and improved lung function, which is very positive.”

Eramangalath also points to products by Insilico Medicine and Blade Therapeutics, which are designed to slow the advancement of IPF. However, it’s still too early to determine if these drugs improve lung capacity.

Across Phases I, II, and III, Eramangalath says there are about 15 IPF drugs currently in development.

One emerging IPF drug in the pipeline is showing promise in Phase III trials. In September 2024, Boehringer Ingelheim announced late-stage results of the company’s PDE4B inhibitor, nerandomilast. Topline data showed that the agent successfully improved forced vital capacity in IPF patients after 52 weeks. Ioannis Sapountzis, head of global therapeutic areas at Boehringer Ingelheim, said in a press release that this is the first IPF Phase III trial in a decade to meet its primary endpoint.

 Eramangalath says that the study has shown significant positive results, and Boehringer Ingelheim is on track to file for regulatory submission this year.

Slightly further back in the pipeline, Bristol Myers Squibb’s investigational drug, BMS-986278, posted Phase II results in September 2023.The drug, a lysophosphatidic acid receptor 1 agonist, was found to reduce the rate of decline in forced vital capacity among IPF patients by 69% relative to placebo.

Meanwhile, United Therapeutics’ inhaled treprostinil is in Phase III development; Eramangalath says the drug has shown great results so far. The TETON 2 trial, a Phase III clinical study on treprostinil for IPF, is currently underway, with topline data expected in the second half of 2025.

GLP-1s, short for glucagon-like peptide-1 receptor agonists, have gained significant popularity among patients and a great deal of attention from the media. Originally designed as medications for type 2 diabetes, GLP-1 drugs, such as Novo Nordisk’s Wegovy and Ozempic (semaglutide) and Eli Lilly’s Mounjaro (tirzepatide), have since been found to be highly effective at treating obesity (with tirzepatide branded as Zepbound for weight loss).

Sara Mallatt, director of healthcare research at AlphaSense in New York City, says now that GLP-1s have been proven in the obesity sphere, companies in the market are creating different combinations of weight loss drugs to better treat obesity. Roche, Viking Therapeutics, and Amgen, for instance, are all working on a GLP-1/GIP combination drug, while Zealand Pharma is developing a GLP-1/glucagon agonist drug. Lilly, meanwhile, is pursuing a triple agonist drug that targets GLP-1, GIP, and glucagon all in a single medication.

Mallatt also notes that there’s significant interest among biopharma manufacturers in expanding the indications that GLP-1s can treat. She explains that in order to get insurers on board, it’s important to provide research around other indications beyond obesity.

“We already had diabetes and obesity indications,” says Mallatt. “In March 2024, Novo’s Wegovy was approved for cardiovascular disease. The other indications that are being pursued are fatty liver disease, chronic kidney disease, obstructive sleep apnea, and Alzheimer’s disease. Beyond those, there’s speculation that GLP-1s could be used for substance abuse disorders and arthritis.”

Overall, according to projections from Towards Healthcare, theGLP-1 receptor agonist market will total $62.86 billion in 2025, a figure it predicts will skyrocket to $268.37 billion by 2034 (a compound annual growth rate of 17.5 % during that period).

In December, the FDA approved Zepbound for the treatment of moderate-to-severe obstructive sleep apnea in adults with obesity.The approval was based on two randomized, double-blind, placebo-controlled studies of 469 adults without type 2 diabetes.

 According to the results, Zepbound was about five times more effective than placebo in reducing breathing disruptions in adults not on positive airway pressure (PAP) therapy, leading to 25 fewer breathing disruptions per hour with Zepbound and five with placebo. In adults on PAP therapy, Zepbound led to 29 fewer breathing disruptions per hour compared to six with placebo.

Back on the cardiovascular front, one 2024 systematic review and meta-analysis examined the results of 13 randomized clinical trials of GLP-1 receptor agonists and tirzepatide. In total, 65,878 patients with type 2 diabetes mellitus were included in the meta-analysis. The review found that relative to placebo, GLP-1s reduced incidents of major adverse cardiovascular events, all-cause mortality, and cardiovascular mortality while simultaneously reducing the risk of a stroke.

Meanwhile, other studies are underway to examine the effects of GLP-1s on Parkinson’s disease, Alzheimer’s disease, drug and alcohol addiction, and sleep apnea. Novo Nordisk is currently running two Phase III clinical trials, the evoke and evoke+ studies, to assess the safety and efficacy of oral semaglutide in managing the symptoms of Alzheimer’s disease. Readouts of the two trials are expected in 2025.

Last month, results of an observational study conducted by Washington University in St. Louis, published in

, found that the use of semaglutide and tirzepatide may reduce the risk of Alzheimer’s disease and substance abuse disorders.

 At the same time, the study, which is based on health records from the US Department of Veterans Affairs (covering 215,970 diabetes patients taking the new drugs), also reported a spike in the risk of digestive issues and arthritis.

Other biopharma companies active in R&D and expansion pursuits in the GLP-1 space include Pfizer, AstraZeneca,Boehringer Ingelheim, Merck,Sanofi, Amneal Pharmaceuticals, Ascendis Pharma,Hansoh Pharma, Innovent Biologics, and Metsera, Inc.

ATMPS offer breakthroughs for autoimmune diseases

ATMPs are medical products that are made from cells, tissues, or genes. These products typically fall into one of three categories: gene therapies, somatic-cell therapies, and tissue-engineered medicines.

 While ATMPs have historically been studied for their oncological applications,

 Mallatt says there’s now significant investment in CAR T-cell therapy for autoimmune indications.

“There was a breakthrough study from a group in Germany in February 2024,” notes Mallatt. “They had results from a case series in which 15 patients with various autoimmune conditions all achieved complete remission after receiving a single infusion of CAR T. It was also found that after infusion, patients could stop taking their immunosuppressants.”

Mallatt says that since then, other studies have confirmed the benefits of ATMPs in treating autoimmune conditions, leading to significant interest and enthusiasm. She notes that as of December 2023, there are over 30 companies pursuing or planning to pursue drugs in this area; as of May 2024, there were over 50 clinical trials for ATMPs for autoimmune diseases listed on ClinicalTrials.gov. While the results of these trials are still pending, companies such as Novartis have published encouraging updates.

“CAR T researchers are focusing on B-cell-mediated diseases,” explains Mallatt. “That includes lupus, rheumatoid arthritis, and multiple sclerosis. In this disease group, B-cells start attacking healthy cells. These B-cells contain a specific surface protein that CAR T can recognize and target. B-cell targets employed in autoimmune CAR T-cell therapy happen to be the same targets employed in CAR T-cell therapy for blood cancers.”

Mallatt says that there is significant interest in CAR T for autoimmune disease treatment; she points to AstraZeneca’s acquisition of Graycell Biotech and Regeneron’s purchase of 272 Bio as evidence of the growing focus in that area.

In just the UK alone, there were over 175 clinical trials for ATMPs in 2023. Oncology remains the largest therapeutic area being investigated, with 41% of clinical trials studying ATMPs for cancer. Meanwhile, hematological conditions account for 10% of clinical trials, and ophthalmological conditions account for 9%.

One ATMP in development, axicabtagene ciloleucel (axi-cel), was studied as a second-line treatment in a Phase III trial published in 2023. In this trial, 359 patients were randomly assigned to receive either axi-cel or standard care for refractory large B-cell lymphoma. The study found that axi-cel administration increased four-year survival rates by 8% relative to standard care.

Other ATMPs in development include RNA- and mRNA-based drugs and vaccines such as CTX001 and rhBMP-2. An investigational CRISPR gene-edited stem cell therapy for sickle cell disease under development by CRISPR Therapeutics and Vertex Pharmaceuticals, CTX001 works by genetically modifying stem cells to increase fetal hemoglobin production. A Phase III clinical trial published in the 

 in April 2024 found that out of 30 patients who were evaluated, 29 patients were free from vaso-occlusive crises for at least 12 consecutive months. Furthermore, all 30 patients stayed out of the hospital for vaso-occlusive crises for at least 12 consecutive months.

Meanwhile, recombinant human bone morphogenetic protein-2 (rhBMP-2) is a growth factor that potentiates stem cells to become osteocytes. rhBMP-2’s potential applications include treating open tibia fractures, tibia shaft nonunions, and other orthopedic trauma.

 study examined the effects of rhBMP-2 and human bone marrow mesenchymal stromal cell (hBM-MSC) administration on bone formation and differentiation. The clinical study found that when combined with other differentiation signals, rhBMP-2 potentiates multilineage differentiation of osteogenesis, adipogenesis, and chondrogenesis.

Pharm Exec’s 20th Annual Pipeline Report examines the emerging drug development trends and surging investments in five expanding therapeutic areas.

Leo Grimaldi, MD

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