Daria G. Husni

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Editor's note: this story was originally published on 

On March 14, 2024 AstraZeneca announced its acquisition of Amolyt Pharma, a clinical-stage biotechnology company focused on developing novel treatments for rare endocrine diseases, in a deal worth potentially $1.05 billion. The transaction includes an $800 million upfront payment and a potential milestone payout of $250 million. With this acquisition, AstraZeneca will obtain eneboparatide (AZP-3601), Amolyt’s Phase III investigational therapeutic peptide that operates with a new mechanism of action intended to treat hypoparathyroidism.

Hypoparathyroidism is characterized by a deficiency in a patient’s parathyroid hormone (PTH) production, leading to severe dysregulation of calcium and phosphate. Patients suffer from life-altering symptoms and complications, including chronic kidney disease. An estimated 115,000 people in the United States and 107,000 people in the European Union, approximately 80% of whom are women, are affected by the disease, making it one of the largest rare diseases being studied. 

Amolyt’s clinical pipeline also includes other therapeutic peptides under development to treat rare endocrine diseases. Among them is AZP-3813, a peptide growth hormone receptor antagonist in Phase I clinical development. AZP-3813 is intended for the potential treatment of acromegaly. With these peptides, especially eneboparatide, AstraZeneca aims to increase its rare disease pipeline, especially in bone metabolism and rare endocrinology.

“Chronic hypoparathyroid patients face a significant need for an alternative to current supportive therapies, which do not address the underlying hormone deficiency. As leaders in rare disease, Alexion is uniquely positioned to drive the late-stage development and global commercialization of eneboparatide, which has the potential to lessen the often debilitating impact of low parathyroid hormone and avoid the risks of high-dose calcium supplementation. We believe this program, together with Amolyt’s talented team, expertise and earlier pipeline, will enable our expansion into rare endocrinology,” said Marc Dunoyer, CEO, Alexion, AstraZeneca Rare Disease, in a company press release.

This move follows other recent acquisitions that AstraZeneca has been making. In January, 

© reported on the pharma company’s acquisitions of Icosavax (1) and Gracell (2), both of which were acquired for over a billion dollars. Icosavax’s acquition, which was finalized in February 2024 (3), marked another addition to AstraZeneca’s growing pipelines. In addition to these transactions, AstraZeneca also recently poured $828 million into its Liverpool and Cambridge manufacturing sites in the United Kingdom. The investement is to be used in research, development, manufacturing, and expansion. With the strengthening of these facilities and the continued acquisition of promising drug candidates, AstraZeneca is expected to boost its positioning in the UK pharmaceutical market.

AstraZeneca to Acquire Icosavax in Deal Valued at Approximately $1.1 Billion

AstraZeneca Poised to Acquire Cell Therapy Developer, Gracell, in Deal Valued at $1.2 Billion

AstraZeneca Completes Acquisition of Icosavax

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AstraZeneca’s acquisition of Amolyt Pharma includes eneboparatide, a Phase III therapeutic peptide for the treatment of hypoparathyroidism.

AstraZeneca to Acquire Amolyt Pharma in Deal Worth Potentially $1.05 Billion 

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Cellares, a self-titled integrated development and manufacturing organization (IDMO), has announced that it has completed its first current Good Manufacturing Practice (cGMP)–compliant Cell Shuttle, an automated cell therapy manufacturing platform that can purportedly significantly reduce costs and process failures associated with the production of cell therapies.

The Cell Shuttle’s adherence to cGMP qualifies that it has been built to strict regulatory guidelines, with patient safety in mind. “By adhering to cGMP standards, Cellares ensures that cell therapies produced on the Cell Shuttle are safe, effective, and are consistently of the highest quality which is critical for patient care and therapeutic outcomes,” stated Fabian Gerlinghaus, CEO of Cellares, in a press release.

Challenges to the cell therapy sphere include high costs, high process failure rates, and an inability for manual manufacturing processes to scale effectively. Cellares hopes to address these issues with the Cell Shuttle, which is designed to cut down on labor and facility space requirements by 90%. Because of this, Cellares states that its IDMO Smart Factories can produce ten times the cell therapy batches per year as conventional CDMO facilities, with the same workforce and footprint.

, Fabian Gerlinghaus, chief executive officer and co-founder of Cellares, spoke on the catalyst that drove Cellares’ creation. “I was attending a lot of cell therapy conferences. And if you listened, people were shouting from the rooftops, we need closed highly automated, reliable cell-type manufacturing technologies in order for this industry to be viable in the long term, and in order for us to meet the patient demand. It is outrageous that there are patients dying on the waitlist, even though they're eligible for FDA-approved cell therapies on the market because the industry can't meet demand,” Gerlinghaus said. “[In 2018], I was walking around on the exhibition floors of the typical cell therapy conferences… and there was just nothing on the market that I thought would actually be able to solve the scalability and the cost and the quality problems. And at that point, Omar and I teamed up to start Cellares, and build a technology platform that actually does solve all three of these issues.”

With the completion of the cGMP-compliant Cell Shuttle, Cellares draws closer to making that goal a reality. The company is preparing to rapidly advance their manufacturing capabilities, recently adding a commercial-scale IDMO Smart Factory, located in Bridgewater, New Jersey, to its existing facility in San Francisco.

part one of Pharmaceutical Technology's interview with Gerlinghaus

 to learn more about Cellares and the capabilities of its Cell Shuttle platform.

The Cell Shuttle platform will manufacture cell therapies for its first clinical trials by the end of the year.

Cellares Set to Launch Good Manufacturing Practice–Compliant Cell Shuttle Manufacturing Platform

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BioCina, a biologics contract development and manufacturing organization (CDMO) and NovaCina, a global fill-and-finish CDMO, have announced a partnership to offer integrated drug substance and drug product solutions for biologics developers. This will allow BioCina to provide fill/finish services while converting drug substances produced at its Adelaide, South Australia, facilities into drug product at NovaCina’s facilities in Perth, Australia. This collaboration aims to provide end-to-end biopharmaceutical manufacturing solutions.

BioCina specializes in CDMO services such as cell line development, process development, and current good manufacturing practice clinical and commercial manufacturing for microbial, plasmid DNA, and messenger RNA modalities. It plans to establish a second site alongside its Adelaide facility, which was once a Pfizer facility, that will focus on mammalian Parkinson’s disease and large-scale manufacturing. NovaCina focuses on clinical and commercial fill/finish experience, working alongside regulatory bodies for its services, which include development, manufacturing, packaging, labeling, and stability studies.

“BioCIna is excited to partner with NovaCina, integrating our globally recognized expertise in clinical and commercial process development and drug substance manufacturing with their … drug product services,” said Mark W. Womack, BioCina’s CEO, in a press release,

The partnership will allow for both companies to provide end-to-end biopharmaceutical manufacturing solutions.

BioCina and NovaCina to Partner on Drug Substance and Product Solutions for Biologics

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Affinity ligands play an exciting role in the ever-growing sphere of downstream processing. In speaking with Pharmaceutical Technology®, Phillip Elliott, PhD, associate director of Process Development, BioCina; Jan Bekker, PhD, director, Business Development, Commercial and Technical Operations, BioCina; and Parviz Shamlou, PhD, senior vice-president of Science & Technology, Abzena illuminate the newest innovations with affinity ligands and provide a deeper understanding of the role affinity ligands play in the future of downstream processing.

Pharmaceutical Technology/Pharmaceutical Technology Europe Outsourcing Innovation eBook February 2024

Pharmaceutical Technology/Pharmaceutical Technology Europe Outsourcing Innovation

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Issue 2
February 2024
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When referring to this article, please cite it as Husni, D. A Closer Look at Affinity Ligands. 

A look at the newest innovations offers a deeper understanding of affinity ligands and their role in the future of downstream processing.

A Closer Look at Affinity Ligands

Revolo Biotherapeutics announced on Jan. 30, 2024 that FDA has granted Orphan Drug Designation to ‘1104, a first-in-class immune-resetting peptide being developed as a potential treatment for eosinophilic esophagitis (EoE). Revolo focuses on developing therapies that reset the immune system to achieve long-term remission for patients with autoimmune and allergic diseases. The original request for ‘1104 was submitted before its Phase IIa EoE study, and the request was amended following the study’s positive results.

The Orphan Drug Designation is granted by FDA to support the development and evaluation of new therapies that are intended for the treatment, diagnosis, or prevention of rare diseases and disorders affecting less than 200,000 people in the United States. EoE is a chronic, inflammatory disease in which eosinophils, specialized cells in the immune system, accumulate in the lining of the esophagus. This causes inflammation, resulting in progressive tissue injury. Untreated, patients can have difficulty swallowing, chronic pain, malnutrition, and weight loss. According to a study 

published in the American Journal of Gastroenterology

, EoE affected around 23,000 patients in 2018, 6000 more than in 2015.

“We are excited that the FDA has granted ODD for ‘1104 in EoE, as there remains a significant unmet need for new treatment innovations for people living with this disease,” said Woody Bryan, president and chief executive officer of Revolo Biotherapeutics, in a Revolo press release. “We look forward to initiating a Phase IIb study of ‘1104 in EoE later this year.”

Evan Dellon, M.D., MPH, gastroenterologist, professor of Medicine and adjunct professor of Epidemiology at the University of North Carolina Chapel Hill, and Principal Investigator for the Phase IIa clinical trial, added, “’1104 is a promising EoE therapeutic with a novel mechanism of action.Based on the proof-of-concept data from the Phase IIa study, I am eager to see this molecule move forward into the next stage of development.”

The immune-resetting peptide, developed by Revolo Biotherapeutics, received positive results from its Phase IIb study.

FDA Grants Orphan Drug Designation to First-in-Class Treatment for Eosinophilic Esophagitis

On Jan, 23, 2024, Sanofi announced its planned acquisition of Inhibrx, a clinical-stage biopharmaceutical, in a deal valued at approximately $1.7 billion with a potential additional cash consideration of around $296 million. The acquisition will take place after the spin-off of non-INBRX-101 assets, such as its immune-oncology pipeline, into New Inhibrx. New Inhibrx will also retain its employees.

INBRX-101 is a human recombinant protein that can potentially normalize serum alpha-1-antitrypsin (AAT) levels in AAT deficiency (AATD) patients with less frequent doses. AATD is a genetic rare disease defined by low levels of AAT protein, which primarily affects the lung with progressive deterioration of the tissue. INBRX-101 works by inhibiting neutrophil elastase, an enzyme that causes lung tissue damage in AATD patients. INBRX-101 has the potential to reduce inflammation and prevent further deterioration of lung function in affected patients, according to a company press release.

INBRX-101 has finished a Phase I trial, where it demonstrated positive results in terms of safety and pharmacokinetics. It is being enrolled in a Phase II clinical trial to continue evaluating its potential as a treatment for AATD.

“The addition of INBRX-101 as a high-potential asset to our rare disease portfolio reinforces our strategy to commit to differentiated and potential best-in-class products,” said Houman Ashrafian, head of Research and Development at Sanofi, in the press release. “With our expertise in rare diseases and growing presence in immune-mediated respiratory conditions, INBRX-101 will complement our approach to deploy R&D efforts in key areas of focus and address the needs of the underserved AATD patients and communities.”

The acquisition adds a potential rare disease asset to Sanofi’s alpha-1 antitrypsin deficiency pipeline.