Pharmaceutical Technology Editors

Ipsen, a global biopharmaceutical company, and Albireo, a rare disease company focused on the development of novel bile acid modulators, announced a definitive merger agreement on Jan. 9, 2023. The deal, expected to close by the end of Q1 2023, will see Ipsen acquire Albireo for approximately $952 million.

According to a company press release, Albireo’s lead medicine is Bylvay (odevixibat), a once-daily, oral, non-systemic ileal bile acid transport inhibitor. FDA has approvedodevixibat as a treatment of pruritus in patients ages three months and older with progressive familial intrahepatic cholestasis (PFIC), while E.U. has approved it for treatment of PFIC in patients ages six months and older. Odevixibat is being considered for other rare diseases; it was the subject of supplementary regulatory filings for Alagille syndrome in December 2022 and is also in late-stage development as a treatment for biliary atresia.

As part of the transaction, Ipsen will also acquire Albireo’s clinical stage asset A3907, a novel oral systemic apical sodium-dependent bile acid transporter inhibitor currently in development for adult cholestatic liver disease. Albireo’s pipeline also includes A2342, an oral systemic sodium-taurocholate co-transporting peptide inhibitor currently being evaluated for viral and cholestatic diseases.

“We are excited about the potential of Albireo’s assets and scientific expertise, which we gain through this acquisition, and we believe this is a compelling growth opportunity for Ipsen,” said David Loew, CEO, Ipsen, in the press release. “Our rare disease franchise is strengthened with [odevixibat], which, in addition to being the first-approved treatment in PFIC, has two further indications being investigated in rare liver conditions that are underserved. Additionally, [odevixibat] and the clinical and preclinical novel bile acid transport inhibitors in Albireo’s portfolio complement our own pipeline in liver disease.”

“Unwavering dedication to patients and commitment to science have always been the north star for Albireo. This focus has driven us to develop and gain approval for [odevixibat] as the first drug treatment for PFIC,” said Ron Cooper, president and CEO, Albireo, in the press release. “Our talented team at Albireo have advanced the first Phase III studies in three different pediatric liver diseases while discovering two promising new clinical stage bile acid modulators. We believe that Ipsen is well positioned to apply its global R&D and commercial capabilities to make these medicines available to more cholestatic liver disease patients and accelerate the mission of providing hope for families. ”

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Ipsen’s $952 million acquisition of Albireo is designed to bolster the company's rare disease portfolio.

Ipsen to Bolster Liver Disease Portfolio with Albireo Acquisition

Sartorius, a life sciences research group, and RoosterBio, a biotechnology company focused on human mesenchymal stem/stromal cell (hMSC) and extracellular vesicle (EV) product and process development, announced the extension of a strategic collaboration on Jan. 5, 2023. This extension builds on an existing two-year collaboration that focused on the industrialization of cell-based regenerative cures; the goal of the new partnership is to work together to address purification challenges and establish scalable downstream manufacturing processes for exosome-based therapies.

According to a company press release, the partnership is attempting to produce a hMSC-based exosome production platform that can deliver industry-leading yield, purity, and potency. The collaboration will also focus on the development of comprehensive analytical techniques for exosome characterization, as well as the entire spectrum of EVs, to establish quantitative strategies for quality control of exosome purification intermediates and final drug product.

“Sartorius is delighted to enter into a collaboration with RoosterBio to develop a robust process for Exosome production,” said Michaela Pischke, head of Business Area Separation Technologies, Sartorius, in the press release. “Together we merge our technologies, RoosterBio’s clinical MSC & EV solution portfolio and Sartorius’ regulatory-compliant bioprocess manufacturing solutions, and industry expertise to deliver the bundled solutions for enabling fast-track early drug development and robust, cost-effective, scalable manufacturing process for exosomes.”

“We are delighted to continue our highly impactful partnership with Sartorius, as this collaboration builds upon our previous process development initiative to establish scalable stirred tank bioreactor expansion processes for hMSCs,” said Tim Kelly, CEO, RoosterBio, in the press release. “Exosomes offer extremely high utility as a new therapeutic modality with the potential to address unmet medical needs. The cell and gene therapy industry will greatly benefit from access to robust, scalable, end-to-end solutions for the production and characterization of native and engineered exosome therapeutics.”

Sartorius and RoosterBio will work together to address purification challenges and establish scalable downstream manufacturing processes for exosome-based therapies.

Sartorius and RoosterBio Strike Downstream Collaboration Deal

Getinge, a Swedish medical technology company, announced the launch of the LivitFlex bioprocess control system. The system is designed to help pharmaceuticals and biotechnology products reach market faster.

According to a Dec. 15, 2022 press release, the LivitFlex speeds up development through system flexibility, smart sensor technology, and easy-to-use software functionalities. Additionally, by accessing and controlling multiple controllers from a PC or tablet in a laboratory, users can set up parallel experiments and monitor all data from one central location.

“Combining the flexibility of the Livit Flex controller, the intuitive Livit software, and Getinge bioreactors enables acceleration of R&D processes to make sure that pharmaceuticals and biotech products will ultimately end up faster where they are most needed – with the patients,” said Matthijs Niemeijer, head, Product Management Bioreactor Systems, Getinge, in the release.

Getinge is launching Livit Flex, a bioprocess control system designed to help pharmaceutical and biotech products get to market faster.

Getinge Announces New BioProcess Control System

Moderna and CytomX, a clinical-stage oncology-focused biopharmaceutical company, announced a strategic collaboration to develop investigational messenger RNA (mRNA)-based conditionally activated therapeutics on Jan. 5, 2022. CytomX will receive $35 million in upfront payment from Moderna, with an additional $1.2 billion, plus tiered royalties contingent on achieving various development, regulatory, and commercial milestones.

According to a company press release, the collaboration will combine Moderna’s mRNA technologies with CytomX’s therapeutic platform Probody. Probody is designed to enable local activation of proteins in diseased tissue while remaining masked in systemic circulations, allowing for the encoding of potent masked biologics via mRNA. Both companies will collaborate on discovery and preclinical development, while Moderna will lead clinical development and commercialization of therapeutics resulting from the agreement.

"We are excited to enter this collaboration with CytomX to combine our technologies and to potentially bring mRNA-based conditionally activated therapies to patients," said Rose Loughlin, senior vice-president, Research and Early Development, Moderna, in the release. "Moderna and CytomX have a shared vision of investing at the intersection of biology and technology to transform the lives of patients, and this collaboration will expand applications of our growing therapeutics pipeline."

"At CytomX, we have always embraced bold science in building the potential of Probody therapeutics, and we are thrilled to be joining forces with Moderna in oncology as well as expanding our technology to areas outside oncology where we believe there is great potential," said Sean McCarthy, CEO and chairman, CytomX, in the release. "Moderna's global impact has shown the enormous power of mRNA and we look forward to working closely with our newest collaborator to bring novel, mRNA-based conditionally activated therapeutics to patients with unmet medical needs."

The collaboration will see Moderna and CytomX work together to develop mRNA-based conditionally activated therapeutics.

Moderna and CytomX Announce mRNA Partnership

FDA announced the approval of Leqembi (lecanemab-irmb) for the treatment of Alzheimer’s disease on Jan. 6, 2023. This move follows recent controversy surrounding the agency’s 2021 approval of another Alzheimer’s disease treatment, Aduhelm (aducanumab), developed by Eisai and BioGen, which was the subject of a congressional report from the House Energy and Commerce Committee and House Committee on Oversight and Reform in late 2022 (1).

Lecanemab-irmb was approved using the Accelerated Approval pathway, which allows FDA to approve drugs for serious conditions with unmet medical need; this can only be done when a drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. It also received Fast Track, Priority Review, and Breakthrough Therapy designations.

According to an agency press release, FDA based its approval on results from a double-blind, placebo-controlled, parallel-group, dose-finding study of 856 participants with Alzheimer’s disease, wherein patients receiving the treatment had significant dose- and time-dependent reduction of amyloid beta plaque, a marker of the disease. FDA is also anticipating results from a Phase III randomized, controlled clinical trial to confirm the drug's clinical benefit.

“Alzheimer’s disease immeasurably incapacitates the lives of those who suffer from it and has devastating effects on their loved ones,” said Billy Dunn, director, office of Neuroscience, Center for Drug Evaluation and Research, FDA, in the release. “This treatment option is the latest therapy to target and affect the underlying disease process of Alzheimer’s, instead of only treating the symptoms of the disease.”

1. Belluck, P, Congressional Inquiry into Alzheimer’s Drug Faults Its Maker and FDA 

Eisai’s Alzheimer’s treatment, Leqembi (lecanemab-irmb), received approval from FDA via the Accelerated Approval pathway.

FDA Grants Accelerated Approval for Eisai Alzheimer’s Disease Treatment

Astra Bioseparations (Astrea Bio), a provider of bioseparation and purification technologies, announced on Jan. 9, 2023 that it has acquired Delta Precision, a manufacturer of advanced chromatography columns for the biotechnology and pharmaceutical industries.

The acquisition will allow Astrea Bio to expand its product offerings to include a full range of high-performance chromatography columns for biomanufacturing. This will add to its existing portfolio of bioprocessing resins, reusable columns, and prepacked single-use columns. Delta Precision’s products are used in a variety of applications, including protein purification, vaccine production, and drug development.

“We are delighted to welcome the Delta team into the fold. The acquisition of Delta is another step forward in Astrea Bio’s rapid expansion to support the biopharmaceutical manufacturing field,” said Terry Pizzie, CEO of Astrea Bio. “This is a highly strategic move for us, allowing us to offer a full range of industry-leading purification and separation tools.”

“We look forward to joining the Astrea Bio family, further cementing the long-standing relationship between the two companies,” added Geoff Parnell, o-founder and managing director at Delta Precision. “We are confident that our expertise in column manufacturing will complement and accelerate the Astrea Bio business and customer reach.”

Astrea Bioseparations has acquired Delta Precision, a manufacturer of chromatography columns for biomanufacturing.