Feliza Mirasol is the science editor for 

Feliza Mirasol

Univercells Technologies, a Donaldson company, has expanded its evaluation agreement with the University of Pennsylvania's Gene Therapy Program (GTP). The collaborators will determine the scalability of GTP’s gene therapy product manufacturing with bioreactors provided by Univercells Technologies.

Under the expanded agreement, GTP will evaluate Univercells Technologies’ commercial- series bioreactor (scale-X nitro 600 m2 bioreactor). The evaluation is aimed at significantly increasing production per batch and potentially reducing cost of goods sold, according to a July 16, 2024 company press release.

“We are honored to extend our collaborative work with GTP in evaluating the scalability of gene therapy production,” said Mathias Garny, general manager, Univercells Technologies, in the press release. “Our mission aligns closely with GTP’s vision of making gene therapies more accessible globally, and we are committed to supporting their efforts with our innovative bioprocessing technologies.”

Under the Bill and Melinda Gates Foundation’s Global Grand Challenge initiative, Univercells Technologies developed the scale-X bioreactor, which was originally designed to lower the cost of viral vaccine production for critical public health vaccines. Through the GTP collaboration, this bioreactor will now be applied to viral vector manufacturing to enable accessibility of gene therapies.

“Our hope is that Univercells Technologies will improve the efficiency of AAV vector manufacturing and help play a role in decreasing costs and enabling access through more affordable prices in the future,” said James M. Wilson, MD, PhD, the Rose H. Weiss professor and director of the Orphan Disease Center, and a professor of Medicine and Pediatrics at the Perelman School of Medicine, in the press release.

GTP is an academic program focused on genetic medicines. It is led by Wilson and is well known for its pioneering work in gene therapy. The program specializes in increasing the accessibility of gene therapies for rare diseases to patients worldwide.

Univercells Technologies became a part of Donaldson through an 

 in June 2023. Through a €136 million (US$148 million at the time of acquisition) transaction, Donaldson gained Univercells Technologies, including that company’s product line, which includes the scale-X single-use structured fixed-bed bioreactor as well as NevoLine, Univercells Technologies’ automated upstream platform.

In the June 29, 2023 press release, Tod Carpenter, chairman, president, and CEO, Donaldson said, “The acquisition of Univercells Technologies represents an important next step in Donaldson’s life sciences strategy, as we continue to expand our product portfolio aimed at providing customers with a comprehensive, differentiated offering. Univercells Technologies’ engineered systems provide close adjacencies with our previous life sciences acquisitions as well as with our growing organic product suite.”

Donaldson provides filtration products and solutions for complex filtration challenges. It comprises three primary segments: Mobile Solutions, Industrial Solutions, and Life Sciences.

1. Univercells Technologies. Donaldson Acquires Univercells Technologies, Broadens Life Sciences Reach. Press Release, June 29, 2023.

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Univercells and the University of Pennsylvania are expanding their collaboration to evaluate certain bioreactors with the aim to scale up gene therapy manufacturing.

Univercells and University of Pennsylvania Expand Agreement for Gene Therapy Program

GSK’s marketing authorization application (MAA) for Blenrep (belantamab mafodotin) combination therapies has been accepted by the European Medicines Agency (EMA). In the MAA, GSK is seeking approval of Blenrep in combination with bortezomib plus dexamethasone (BorDex) or pomalidomide plus dexamethasone (PomDex) for treating relapsed or refractory multiple myeloma (RRMM). Following EMA’s acceptance, the Committee for Medicinal Products for Human Use will begin formal review of the MAA and will make a recommendation to the European Commission, GSK announced in a July 19, 2024 company press release.

The MAA is based on interim results from two Phase III trials (DREAMM-7 and DREAMM-8), which both met their respective primary endpoints. The trials showed statistically significant and clinically meaningful improvements in progression-free survival for the belantamab mafodotin combinations compared to standard-of-care combinations in RRMM. The DREAMM-7 trial is evaluating the combination of belantamab mafodotin with BorDex versus daratumumab plus BorDex. The DREAMM-8 trial is evaluating the combination of belantamab mafodotin with PomDex versus bortezomib plus PomDex.

“[This] milestone reinforces the potential for Blenrep to redefine outcomes for patients with multiple myeloma at or after first relapse. We are working to bring Blenrep to patients as quickly as possible given the high unmet need and the clinically robust effects of the Blenrep combinations in the DREAMM-7 and DREAMM-8 [P]hase III head-to-head trials,” said Hesham Abdullah, senior vice-president, global head Oncology, R&D, GSK, in the press release.

Both Phase III trials also showed a positive overall survival (OS) trend, but the trend was not statistically significant at the time the interim analysis was conducted, according to the press release. Follow-up for OS is ongoing. Results did show clinically meaningful improvements across all other secondary efficacy endpoints, however, including deeper and more durable responses compared to respective standard-of-care therapy combinations. “The safety and tolerability profiles of the belantamab mafodotin combinations in DREAMM-7 and DREAMM-8 trials were broadly consistent with the known profiles of the individual agents,” GSK stated in the press release.

Multiple myeloma, the third most common blood cancer globally, is considered treatable but not curable (1,2). Approximately more than 180,000 new cases are diagnosed annually on a global basis. The diagnosis includes approximately 50,000 new cases in Europe (3).

Blenrep is an antibody-drug conjugate. The drug consists of a humanized B-cell maturation antigen monoclonal antibody (mAb) that is conjugated to auristatin F (cytotoxic agent) by a non-cleavable linker. According to GSK, the drug linker technology was licensed from Seagen. The mAb is produced using POTELLIGENT Technology, which was licensed from BioWa, a member of the Kyowa Kirin Group.

1. Sung, H.; Ferlay, J.; Siegel, R.; et al. Global Cancer Statistics 2020: GLOBOCAN Estimates of Incidence and Mortality Worldwide for 36 Cancers in 185 Countries. 

 (3), 209–249. DOI:10.3322/caac.21660
2. Kazandjian, D. Multiple Myeloma Epidemiology and Survival: A Unique Malignancy. 

 (6), 676–681. DOI:10.1053/j.seminoncol.2016.11.004
3. Ferlay, J.; Ervik, M.; Lam, F.; et al. Multiple Myeloma. In 

; International Agency for Research on Cancer, 2024 [accessed July 23, 2024].

GSK's MAA is supported by an interim analyis of Phase III trials, which demonstrated significant progression-free survival benefit and positive overall survival trends from Blenrep combination therapies.

GSK’s Application Seeking Blenrep Combinations for Treating Multiple Myeloma Accepted by EMA

Pluri, an Israel-based biotechnology company, announced that it has entered into a tech transfer and manufacturing agreement with Kadimastem, an Israel-based clinical stage biotechnology company developing therapeutic cells to treat amyotrophic lateral sclerosis (ALS) and diabetes. According to a July 18, 2024 company press release, Pluri’s contract development and manufacturing organization (CDMO) business, PluriCDMO, which was launched in January 2024 (1), will manufacture two cell therapy product candidates for Kadimastem at its 47,000-ft

 good manufacturing practice (GMP) cell production facility at its MATAM Advanced Technology Park site in Haifa, Israel (2). 

The two cell therapy product candidates from Kadimastem are AstroRx, clinical-grade human astrocytes (nervous system supporting cells) for the treatment of ALS, and IsletRx, clinical-grade pancreatic islet cells that produce and secrete insulin and glucagon in response to blood glucose levels. The material PluriCDMO produces for AstroRx will supply an upcoming FDA Phase II study. Meanwhile, IsletRx is in development for treating diabetes.

“Working with Pluri marks a pivotal milestone, enhancing Kadimastem’s capacity to manufacture our products under GMP conditions,” said Ronen Twito, executive chairman and president of Kadimastem, in a company press release. “This collaboration is integral to our strategy as we prepare for clinical trials and expand into the US market with our AstroRx product candidate.”

Kadimastem’s decision in selecting PluriCDMO is based on Pluri’s expertise and experience in developing and manufacturing GMP-grade cell-based products for clinical use. The CDMO has the capacity to scale up from initial clinical trial batches to mass-scale commercial production, according to Pluri in the press release.

Pluri’s CDMO division offers access to its patented bioreactor system, which enables 3D cell expansion at mass scale using a fully controlled, automated, and validated process. The company’s 3D cell expansion technology supports the large-scale growth of cells and offers unique batch-to-batch consistency in a scalable, cost-effective manner that can support late-stage clinical trials in key jurisdictions, including FDA, the European Medicines Agency, Israeli’s Ministry of Health, Japan’s Pharmaceuticals and Medical Devices Agency, and the Ministry of Food and Drug Safety of the Republic of Korea. In addition, the company’s PluriMatrix technology enables industrial-scale production of cell-based products (1).

“We are excited to work with Kadimastem and support their development of cell therapies, potentially improving the lives of patients with ALS and diabetes,” said Yaky Yanay, chief executive officer and president of Pluri, in the press release. “This collaboration underscores the versatility of our PluriCDMO platform and our commitment to aiding innovative companies in advancing their life-saving therapies. We look forward to a successful collaboration with Kadimastem as they make progress with their clinical development programs.”

Kadimastem specializes in developing allogeneic (i.e., “off-the-shelf”) proprietary cell products based on its technology platform, which enables the expansion and differentiation of human embryonic stem cells (hESCs) into functional cells.

1. Pluri. Pluri Launches Advanced Global Cell Therapy Contract Development and Manufacturing Organization. Press Release, Jan. 8, 2024.
2. Pluri. 10K–Annual 2023 Financial Report. 

PluriCDMO, Pluri's newly launched CDMO division, has been selected by Kadimastem to manufacture two novel cell therapy product candidates.

Kadimastem and Pluri Enter Cell Therapy Manufacturing Deal

AstraZeneca has completed its acquisition of Amolyt Pharma, a clinical-stage biotechnology company focused on treatments for rare endocrine diseases, in a deal valued at $1.05 billion. Through the acquisition, AstraZeneca gains a Phase III investigational therapeutic peptide, eneboparatide (AZP-3601), which is in development for treating hypoparathyroidism. The addition of this drug candidate boosts the AstraZeneca–Alexion Pharmaceuticals Rare Disease late-stage pipeline and bone metabolism franchise, according to a July 15, 2024 press release. AstraZeneca previously acquired Alexion in 2021 (1,2). 

Under the Amolyt Pharma acquisition, which AstraZeneca 

 in March 2024 (3), AstraZeneca acquired all of Amolyt Pharma’s outstanding shares for a total consideration of up to $1.05 billion, which includes $800 million upfront at the closing of the deal plus the right for Amolyt Pharma’s shareholders to receive an additional $250 million contingent payment, based on a specified regulatory milestone.

Eneboparatide has a novel mechanism of action; it is designed to bind with high affinity to a specific conformation of the parathyroid hormone (PTH) receptor 1 (4). The therapeutic goals for this drug candidate include regulating and maintaining serum calcium levels in a normal range to manage symptoms of hypoparathyroidism; limiting the excretion of urine calcium by restoring the kidney’s ability to reabsorb calcium; and potentially preventing progressive kidney function decline and the onset of chronic kidney disease (5). Eneboparatide has also been designed to have a short plasma half-life to enable the restoration of bone turnover to a more physiologic state and to preserve bone integrity (4).

Hypoparathyroidism, a rare condition, is caused by a PTH deficiency, which leads to decreased calcium levels and elevated phosphorus levels in the blood (6). “Despite available treatments, patients experience persistent, life-altering symptoms and often develop complications and comorbidities that diminish quality of life and create segments of the patient population with specific clinical needs. Clinical manifestations of hypoparathyroidism impact many tissues and organ systems, in particular, the kidneys and bone,” AstraZeneca stated in a company press release.

The Amolyt Pharma acquisition is the latest in a series of billion-dollar-level acquisitions that AstraZeneca has completed so far this year. In February 2024, AstraZeneca completed its $1.1 billion acquisition of 

, a clinical-stage biopharmaceutical company, and its $1.2 billion acquisition of 

, a clinical-stage biopharmaceutical company specializing in cell therapies for cancer and autoimmune diseases (7,8). With the Icosavax acquisition, AstraZeneca gained IVX-A12, Icosavax’s lead investigational vaccine candidate that targets respiratory syncytial virus and human metapneumovirus. IVX-A12 is a combination protein virus-like particle platform vaccine that is Phase III-ready and has been granted fast track designation by FDA (7). Through the Gracell acquisition, AstraZeneca gained GC012F, a clinical-stage chimeric antigen receptor T-cell therapy. GC012F is, potentially, a new treatment for multiple myeloma and other hematologic malignancies and autoimmune diseases, including systemic lupus erythematosus (8).

1. AstraZeneca. AstraZeneca to Acquire Alexion, Accelerating the Company's Strategic and Financial Development. Press Release, Dec. 12, 2020.
2. AstraZeneca. Acquisition of Alexion Completed. Press Release, July 21, 2021.
3. AstraZeneca. AstraZeneca to acquire Amolyt Pharma, Expanding Late-Stage Rare Disease Pipeline. Press Release, March 14, 2024.
4. Takacs, I.; Mezosi, E.; Soto, A.; et al. An Open-Label Phase 2 Study of Eneboparatide, a Novel PTH Receptor 1 Agonist, in Hypoparathyroidism. 

. DOI: 10.1210/clinem/dgae121
5. Khan, A.A.; Bilezikian, J. P.; Brandi, M.L.; et al. Evaluation and Management of Hypoparathyroidism Summary Statement and Guidelines from the Second International Workshop. 

 (12), 2568–2585. DOI: 10.1002/jbmr.4691
6. Clarke, B.L.; Brown, E.M.; Collins, M.T.; et al. Epidemiology and Diagnosis of Hypoparathyroidism. 

, 101 (6), 2284–2299. DOI: 10.1210/jc.2015-3908
7. AstraZeneca. Acquisition of Icosavax Completed. Press Release, Feb. 19, 2024.
8. AstraZeneca. Acquisition of Gracell Completed. Press Release, Feb. 22, 2024.

With this acquisition, AstraZeneca gets a Phase III therapeutic peptide candidate that adds to the company's rare disease pipeline.

AstraZeneca Completes Amolyt Pharma Acquisition and Boosts Rare Disease Pipeline

Eyebiotech Limited (EyeBio) has become a wholly owned subsidiary of Merck, known as MSD outside of the United States and Canada, following the completion of Merck's acquisition of the company, according to a July 12, 2024 press release.

Under the agreement, Merck acquired all outstanding shares of EyeBio through a Merck subsidiary for approximately $0.50 per share, or $1.3 billion, which Merck will record as a charge in the third quarter of 2024 (to be included in non-generally accepted accounting principles results). The acquisition adds EyeBio’s lead candidate, Restoret (EYE103), an investigational, potentially first-in-class tetravalent, tri-specific antibody, to Merck’s eye disease therapy pipeline.

“The EyeBio acquisition further diversifies our late-stage pipeline with the addition of a promising candidate based on novel biology and genetics for the treatment of certain retinal diseases,” said Dean Y. Li, PhD, president, Merck Research Laboratories, in the press release. “We are excited to welcome the EyeBio team and look forward to working together to advance Restoret for the patients that need it.”

Restoret acts as an agonist of the Wingless-related integration site (Wnt) signaling pathway, which is part of innate immunity. The Wnt signaling pathway is believed to have a major role in the regulation of inflammation (1). Following positive results from a Phase Ib/IIa study (AMARONE) in patients with diabetic macular edema (DME) and neovascular age-related macular degeneration (NVAMD), Restoret will enter a pivotal Phase IIb/III trial in the second half of 2024 to evaluate its potential in treating patients with DME.

In addition to Restoret, Merck gains other pipeline candidates through the acquisition, including clinical and preclinical assets in development for preventing and treating vision loss associated with retinal vascular leakage, which is a known risk factor for retinal diseases, according to the press release.

Restoret is administered as an intravitreal injection. Its aim is to eliminate vascular leakage in retinal diseases by agonizing the Wnt pathway, and in so doing, restore and maintain the blood-retinal barrier. “Preclinical evidence indicates that agonizing the Wnt pathway in the retina may reduce vascular leakage,” Merck stated in the press release.

The completion of the EyeBio acquisition follows Merck’s other 

 for Harpoon Therapeutics, a US-based clinical-stage immuno-oncology company, which Merck announced in January 2024 (2) and completed in March 2024 (3). This approximately $680 million transaction adds Harpoon’s lead candidate, HPN328 (now designated as MK-6070), a T-cell engager targeting delta-like ligand 3 (DLL3) to Merck’s pipeline. DLL3 is an inhibitory canonical Notch ligand expressed at high levels in small cell lung cancer (SCLC) and neuroendocrine tumors. HPN328 is in clinical study as a monotherapy for advanced cancers associated with the expression of DLL3 and is being studied in combination with atezolizumab for treating SCLC. FDA granted the drug candidate orphan drug designation in March 2022 for the treatment of SCLC.

The Harpoon acquisition also gives Merck additional pipeline candidates, including HPN217, a T-cell engager targeting B-cell maturation antigen currently in Phase I clinical development for relapsed/refractory multiple myeloma treatment and several preclinical stage candidates, including HPN601, a conditionally activated targeting epithelial cell adhesion molecule (EpCAM) in development for treating certain patients with EpCAM-expressing tumors (3).

1. Krüger, B. D.; Hofer, G.E.; Rudiger, A.; et al. Wingless-Related Integration Site (WNT) Signaling is Activated During the Inflammatory Response Upon Cardiac Surgery: A Translational Study. 

, 997350. DOI: 10.3389/fcvm.2022.997350
2. Merck. Merck to Acquire Harpoon Therapeutics, Further Diversifying Oncology Pipeline. Press Release, Jan. 8, 2024.
3. Merck. Merck Completes Acquisition of Harpoon Therapeutics, Inc. Press Release, March 11, 2024.

Merck gains a late-phase antibody drug candidate for diabetic macular edema and neovascular age-related macular degeneration through its acquisition of EyeBio.

Merck Gains Late-Stage Eye Disease Drug Candidate with EyeBio Acquisition

Johnson & Johnson boosts its bispecific antibody pipeline with the addition of NM26, a novel, investigational bispecific antibody in development for treating atopic dermatitis (AD), to its portfolio following the completion of its $1.25 billion acquisition of Yellow Jersey, a demerged subsidiary of Numab Therapeutics, according to a July 11, 2024 press release.

NM26 is ready to enter Phase II studies in AD. It targets interleukin 4 receptor (IL4R)-alpha subunit (IL-4Rα) and IL-31, two clinically proven pathways that trigger T helper type 2 (Th2)-mediated skin inflammation and skin itch, respectively. By targeting these two pathways, NM26 has the potential to transform the standard of care for AD and could also be effective in treating other inflammatory skin diseases in which Th2 inflammation and itch are prevalent (1).

“NM26 is designed to help different subpopulations of patients by targeting two disease-driving pathways, which is key when treating a heterogeneous disease like AD,” said David Lee, Global Immunology Therapeutic Area head, Johnson & Johnson Innovative Medicine, in the press release. “We are excited about the potential this represents to transform the standard of care for AD, as well as other inflammatory skin diseases involving Th2 inflammation and itch.”

The NM26 program was included as part of the acquisition agreement for Yellow Jersey Therapeutics, which was formed through a demerger with Numab. Separately, Johnson & Johnson has entered an agreement with Kaken Pharmaceutical to secure rights to the drug candidate in the Asia Pacific Region. In 2021, Numab had formed a global co-development and regional licensing agreement with Kaken for NM26, under which Kaken has commercial rights to the drug candidate in Japan, China, South Korea, Taiwan, Singapore, and Hong Kong, while Numab kept the commercial rights in the United States, Europe, and the rest of the world (2).

AD, the most common inflammatory skin disease also referred to as eczema, causes itching and inflammation. The symptoms are made worse by scratching, which can lead to increased risk of skin infections, skin pain, difficulty sleeping, anxiety, stress, depression and even an increased risk of suicide. It affects more than 9.6 million children and 16.5 million adults in the US, according to the press release.

“Nearly three-quarters of people with AD are not achieving remission with currently available treatments. The unmet need is high, and we must do better for patients,” said Candice Long, worldwide vice-president, Immunology, Johnson & Johnson, in the press release. “Our investment in NM26, and other bispecific antibodies, is another important step towards our mission of durable, symptom-free remission for all patients living with immune-mediated diseases.”

 in May 2024 with an expected close of transaction in the the second half of 2024 (1). “To deliver durable, symptom-free remission for the millions of people living with AD, our medicines need to be tailored to target multiple disease-driving pathways in different patient subpopulations,” said Lee in the May press release. “That’s why we are committed to developing differentiated bispecifics that combine the targeting of two distinct disease-driving pathways. NM26 has the potential to deliver a treatment specifically for patients who have inflamed skin associated with intense itching.”

1. Johnson & Johnson. Johnson & Johnson to Obtain Rights to a Clinical-Stage Bispecific Antibody to Address Distinct Patient Needs in Atopic Dermatitis. Press Release, May 28, 2024.
2. Numab Therapeutics. Numab Therapeutics and Kaken Pharmaceutical Form Co-Development Alliance for a Next-Generation Atopic Dermatitis Treatment Option. Press Release, Jan. 13, 2021.

Johnson & Johnson gains NM26, a Phase II-ready bispecific antibody in development for atopic dermatitis, boosting its immune-mediated and inflammatory disease portfolio.