Feliza Mirasol is the science editor for 

Feliza Mirasol

Takeda has received FDA approval for HYQVIA (immune globulin infusion 10% [Human] with recombinant human hyaluronidase) for treating chronic inflammatory demyelinating polyneuropathy (CIDP), a rare, acquired, immune-mediated neuromuscular disorder. A subcutaneous immunoglobulin (IG), HYQVIA is meant for the maintenance treatment of CIDP to prevent the relapse of neuromuscular disability and impairment in adults.

According to a Jan. 16, 2024 company press release, HYQVIA is the only FDA-approved combination of IG and hyaluronidase, making it a facilitated subcutaneous IG (SCIG) infusion. With the hyaluronidase component, the therapy can be infused for adults with CIDP up to once monthly (every two, three, or four weeks). The hyaluronidase component facilitates the dispersion and absorption of large IG volumes in the subcutaneous space between the skin and the muscle, according to the press release.

Because of its subcutaneous delivery method, HYQVIA can be administered by a healthcare professional in a medical office, infusion center, or in the home. It can also be self-administered with proper training of the patient or caregiver.

HYQVIA first received US approval in 2014 to treat primary immunodeficiency in adults. This indication has since been expanded to include children two to 16 years old.

“With the FDA approval of HYQVIA for CIDP, which builds on our expertise in rare neuroimmunological and neuromuscular disorders, we can now offer a personalized maintenance treatment option for adults with this debilitating disease,” said Giles Platford, president of Takeda’s Plasma-Derived Therapies Business Unit, in the press release. “Research and clinical experience have shown that IG therapy is effective as maintenance treatment in adults with CIDP, and we hope that this approval for HYQVIA is the first of several around the world as we strive to deliver our broad and diverse IG portfolio to more people with complex neuroimmunological diseases.”

“While it is considered the standard-of-care for maintenance treatment of adults with CIDP, IVIG [intravenous immunoglobulin] infusions may be challenging for some patients and their caregivers,” said Lisa Butler, executive director, GBS-CIDP Foundation International, in the release. “We’re excited that this therapy could offer some adults with CIDP an alternative subcutaneous option that may address some of these challenges and help personalize treatment.”

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Takeda has received FDA approval for HYQVIA, a subcutaneous immunoglobulin for the maintenance treatment of chronic inflammatory demyelinating polyneuropathy.

Takeda’s Subcutaneous Immunoglobulin Receives FDA Approval for Maintenance Therapy of Rare Neuromuscular Disorder

KBI Biopharma (KBI), a JSR Life Sciences company, and Argonaut Manufacturing Services (Argonaut), announced on Jan. 16, 2024 that they have entered into a strategic alliance with the goal of offering end-to-end drug substance and drug product manufacturing solutions. The arrangement is expected to provide integration of drug substance and drug product manufacturing by combining KBI’s drug development and biologics manufacturing services with Argonaut’s expertise in sterile fill/finish.

“At KBI Biopharma, we have always been committed to delivering innovative solutions that accelerate the development and manufacturing of life-saving therapeutics,” said J.D. Mowery, president and CEO of KBI Biopharma, in a company press release. “Our … facilities and … team have consistently pushed the boundaries of what is possible in biopharmaceutical development. Similarly, Argonaut has established itself as a trusted provider in the industry, known for its cutting-edge technologies and … track record in drug product manufacturing. Their commitment to quality and efficiency aligns perfectly with our own values and vision. This collaboration represents a new era of collaboration and innovation in the biopharma industry.”

“We believe this strategic alliance resolves a key issue for biopharmaceutical organizations as it greatly improves the alignment and integration between two complex and critical aspects of drug development and manufacturing,” said Wayne Woodard, founder and CEO of Argonaut, in a company press release. “Biopharmaceutical companies will benefit from an integrated solution that is faster, more flexible, and leverages the unique expertise of our two organizations.”

“Argonaut’s fill/finish capabilities are scalable from first-in-human clinical material for rare diseases through commercial batches of approved products. With this collaboration, biopharmaceutical organizations can now seamlessly move from development to full-scale manufacturing, saving both time and capital expenditures,” Woodard also said in the release.

Argonaut had previously announced a capital investment in January 2023 for the significant expansion of its Carlsbad, Calif., manufacturing facilities. The expansion includes increased capacity and scale.

Under a new alliance, KBI Biopharma and Argonaut Manufacturing Services will combine their strengths to offer end-to-end biopharma development and CGMP manufacturing solutions.

KBI Biopharma and Argonaut Manufacturing Services Team Up on Drug Substance and Drug Product Manufacturing Solutions

Abzena announced on Jan. 16, 2023 the launch of AbZelect and AbZelectPRO, its new cell line development (CLD) platforms, designed to speed up the generation of production cell lines for the manufacture of antibodies and recombinant proteins. The platforms are intended to enable the rapid progress of complex biologic drug programs towards clinical trials and investigational new drug (IND) application filing.

According to a company press release, the CLD platforms enable the rapid generation and selection of high-yielding mammalian Chinese hamster ovary cell lines for producing therapeutic proteins and recombinant vaccines. AbZelect was designed for traditional protein programs, such as antibodies. Based on this design, it is optimized across three key areas: vector, host cell line, and process.

AbZelectPRO combines AbZelect with ProteoNic Biosciences’ 2G UNic vector technology, which boosts expression levels and generates higher-producing, stable cell lines that express proteins such as bispecifics, fusion proteins, and other novel modalities, according to the press release. These platforms are expected to shorten timelines from DNA to research cell banks to 15 weeks.

“The higher productivity that’s achievable with the AbZelect and AbZelectPRO platforms means we can produce more drug substances per manufacturing run. These efficiencies will not only expand our capacity and help to reduce the cost of goods for our customers but also reduce the time it takes to get new and critical therapies to patients in need,” said Matthew Stober, CEO, Abzena, in the press release.

Abzena has launched its AbZelect platforms, designed for improving cell line development.

Abzena Launches New Cell Line Development Platforms

Charles River Laboratories International announced the launch of its off-the-shelf replication/capsid (rep/cap) plasmids for the manufacture of adeno-associated virus (AAV) vectors during Advanced Therapies Week, held Jan. 16–19, 2024 in Miami Beach, Fla. These plasmids are designed to streamline AAV-based gene therapy programs, according to a company press release.

The addition of AAV rep/cap (RC2, 5, 6, 8, 9) follows a previously announced launch of the company’s lentiviral packaging and AAV helper plasmid products. According to Charles River, the ready-to-use plasmid products are produced and released according to batch production records. The products include chemistry, manufacturing, and controls guidance and a certificate of analysis to support investigational new drug and clinical trial application filing.

Standard off-the-shelf plasmids, such as helper and rep/cap plasmids required for AAV production, are immediately available, which can lower development costs, risks, and timelines for gene therapy developers, according to the press release. Charles River’s rep/cap plasmids have been used to support the production of AAV serotypes carrying various therapeutic transgenes at multiple development and good manufacturing practice production scales.

“The reduction in production time and improved efficacy of the supply chain will help accomplish our ultimate goal of delivering safe, effective therapies to patients, faster,” said Kerstin Dolph, senior vice-president, Manufacturing, Charles River, in the release.

Charles River’s off-the-shelf rep/cap plasmids are intended to simplify gene therapy supply chains.

Charles River Launches Off-the-Shelf Rep/Cap Plasmids for AAV Vector Manufacturing

Merck, known as MSD outside of the United States and Canada, and Harpoon Therapeutics, a US-based clinical-stage immuno-oncology company, announced on Jan. 8, 2024 that they have entered into a definitive agreement for Merck to acquire Harpoon via a Merck subsidiary. Merck will acquire Harpoon for $23.00 per share in cash, or a total equity value of approximately $680 million. The transaction is expected to close in the first half of 2024.

Under the agreement, Merck will gain Harpoon’s lead candidate, HPN328, a T-cell engager targeting delta-like ligand 3 (DLL3). DLL3 is an inhibitory canonical Notch ligand that has been shown to be expressed at high levels in small cell lung cancer (SCLC) and neuroendocrine tumors. HPN328 is currently being studied in a Phase I/II clinical trial as a monotherapy in patients with advanced cancers associated with the expression of DLL3. It is also being studied in combination with atezolizumab in patients with SCLC. Harpoon announced positive interim tolerability and response data in certain patients with SCLC and neuroendocrine tumors last October 2023.

Harpoon has developed a portfolio of novel T-cell engagers using the company’s proprietary Tri-specific T cell Activating Construct (TriTAC) platform. The TriTAC platform is an engineered protein technology that has been designed to direct a patient’s own immune cells to kill tumor cells. In addition, the company’s ProTriTAC platform involves the application of a prodrug concept to the TriTAC platform to create a therapeutic T-cell engager that is designed to remain inactive until it reaches the tumor.

Additional candidates in the company’s pipeline include HPN217, which targets B-cell maturation antigen (BCMA). HPN217 is currently in Phase I clinical development for treating patients with relapsed/refractory multiple myeloma. The company also has several preclinical-stage candidates, including HPN601, which is a conditionally activated targeting epithelial cell adhesion molecule (EpCAM) for treating certain patients with EpCAM-expressing tumors.

“At Merck, we continue to enhance our oncology pipeline through strategic acquisitions that complement our current portfolio and advance breakthrough science to help address the needs of people with cancer worldwide,” said Dean Y. Li, president, Merck Research Laboratories, in a company press release. “This agreement reflects the creativity and commitment of scientists and clinical development teams at Harpoon. We look forward to further evaluating HPN328 in innovative combinations with other pipeline candidates.”

“At Harpoon, we have always been committed to advancing our cancer immunotherapy candidates to improve the lives of patients. With Merck’s recognized leadership in oncology clinical development and global commercial footprint, our lead candidate, HPN328, is well positioned moving forward,” said Julie Eastland, president and chief executive officer, Harpoon Therapeutics, in the press release.

Through the acquisition of Harpoon Therapeutics, Merck will gain an investigational delta-like ligand 3-targeting T-cell engager under development for cancer treatment.

Merck to Acquire Harpoon Therapeutics in Deal Valued at $680 Million

On Jan. 11, 2024, Boehringer Ingelheim announced that it is investing €120 million (US$131 million) to further expand and upgrade its production facility in Koropi, Greece. With the investment, the company plans to increase manufacturing capacity of new and existing medications, some of which are in late-stage development in the company’s pipeline. The company is investigating several main disease areas, including cardio-renal-metabolic diseases, mental health, and pulmonary fibrosis.

According to a company press release, the expansion will boost medicine exports from Greece, in particular Jardiance (empagliflozin) for treating type 2 diabetes, chronic (long-term) heart failure, and chronic kidney disease, to the US market. The expansion will also create 110 additional jobs. The company had originally announced multi-year investments in 2020 to expand the Koropi site and its production capacity.

The expansion falls in line with the company’s goal of implementing innovative production technologies to support its R&D activities, the company stated in its press release. In 2022, the company spent €4.6 billion (US$5 billion) for its Human Pharma R&D unit, with 25% of Human Pharma net sales being reinvested to advance science and cutting-edge research for innovative therapeutics.

“This expansion investment will bring some of our most innovative therapies to an increasing number of patients globally,” said Hubertus von Baumbach, chairman of the board of managing directors at Boehringer Ingelheim, in the press release.

Boehringer Ingelheim is investing €120 million (US$131 million) into its Koropi, Greece site to expand production for new therapeutics.