Feliza Mirasol is the science editor for 

Feliza Mirasol

On March 27, 2023, Takeda Pharmaceutical announced that the Japanese Ministry of Health, Labour and Welfare has approved Entyvio (vedolizumab) in 108-mg pens for subcutaneous (SC) injection and 108-mg syringes for SC injection as maintenance therapy for moderate to severe ulcerative colitis.

Entyvio SC provides an alternative dosing option that allows for patients to choose the method of administration that best meets individual needs. According to a company press release, SC delivery may reduce the number of personnel, equipment, facilities, and time necessary to prepare an intravenous formulation. This reduction may minimize errors encountered in the administration of the drug. The SC delivery method is also intended to provide ease of handling and convenience and can reduce the time required per administration.

“We are delighted that the subcutaneous formulation of Entyvio has been approved. We believe that this approval will provide a wider range of dosing options for ulcerative colitis patients and healthcare professionals and will help meet their diverse needs and improve quality of life,” said Naoyoshi Hirota, general manager of Takeda Development Center Japan in the press release.

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Takeda received approval in Japan for a subcutaneous injection formulation of Entyvio (vedolizumab) for treating ulcerative colitis.

Takeda Gets Approval in Japan for Entyvio Subcutaneous Injection 

Exothera, a Belgium-based contract development and manufacturing organization (CDMO), and Remedium Bio, a US-based biotechnology company, have entered into an agreement under which the two companies will collaborate to scale-up manufacturing and demonstrate the industrialization potential of Remedium’s lead gene therapy drug candidate, AAV2-FGF18, the companies announced on March 22, 2023.

Remedium’s lead candidate is a disease-modifying gene therapy that is based on the clinically proven regenerative mechanism of fibroblast growth factor 18 (FGF18), according to Remedium in a company press release. The company is developing the gene therapy to treat osteoarthritis, a sector of rheumatology that has the largest unmet clinical need and which currently has no approved disease-modifying treatments, according to the press release.

 manufacturing center of excellence in Jumet, Belgium, Exothera, is well-positioned to supply clinical- and commercial-scale supplies of AAV2-FGF18, the company stated in the release. The CDMO will execute a proof-of-concept demonstration for transient expression of AAV2-FGF18 with suspension culture using a human embryonic kidney cells 293 cell line. Exothera aims to identify a high yield production process and reduce cost-of-goods for the lead candidate’s manufacturing process.

CDMO Exothera and biotech company Remedium are joining forces to scale up the manufacturing process for Remedium’s lead gene therapy candidate for treating osteoarthritis.

Exothera Partners with Remedium to Scale-Up Manufacturing of Gene Therapy Drug Candidate

Takeda Pharmaceutical announced on March 23, 2023 that it plans to invest approximately ¥‎100 Billion (US$754 million) into building a new manufacturing facility in Osaka, Japan, for plasma-derived therapies (PDTs). According to a company press release, this move represents Takeda’s largest investment in expanding its manufacturing capacity in Japan.

The investment is part of Takeda’s strategy to establish end-to-end manufacturing capability for PDTs at Osaka. According to the company in its press release, the new state-of-the-art facility will be the largest of its kind in the country. The facility is expected to be operational by 2030 and expands Takeda’s capacity for plasma manufacturing by almost five-fold. The company also has an existing plasma facility in Narita, Japan.

The facility will be built with automation and advanced digital technologies and will be a fully integrated plant, comprising teardown, fractionation, purification, and fill/finish capabilities as well as a cold storage warehouse. Takeda will use an environmentally friendly design to support its goal of achieving net-zero greenhouse gas emissions related to operations, including scopes 1 and 2, before the year 2035. The company’s existing Narita plasma manufacturing site will continue day-to-day operations until at least the end of the decade, during which time the company will make continued necessary investments for maintenance.

“Takeda has delivered plasma therapies to meet the needs of patients in Japan for more than 70 years. This new investment will strengthen our ability to continuously and reliably bring high-quality PDTs to a growing number of patients in Japan and worldwide. We will engage in discussions with the Ministry of Health, Labor, and Welfare to ensure we are contributing to sustainable patient care in an area of high unmet need in Japan,” said Milano Furuta, president of the Japan Pharma Business Unit at Takeda, in the company press release.

“We are pleased to make this investment in Japan, demonstrating our commitment to our fast-growing PDT business and to our home country,” said Thomas Wozniewski, global manufacturing and supply officer at Takeda, in the release. “Japan will play an even more critical role in our global manufacturing network, contributing to supply chain resilience globally. The new facility will provide a plasma fractionation capacity of more than [two] million liters per year. The company will unleash the power of automation and digitalization to deliver high quality products to patients reliant on PDTs, including those with diseases for which there are no alternative treatments.”

Takeda plans to build a new facility in Osaka, Japan, for the manufacture of plasma-derived therapies.

Takeda to Invest ¥‎100 Billion (US$754 Million) into New Plasma Therapy Manufacturing Facility

On March 22, 2023, Invitae, a US-based medical genetics company, and Deerfield Management, a US-based healthcare investment firm, announced that they are partnering to advance genetics-based drug discovery and development in rare diseases. Under the agreement, the partnership will harness genetics and clinical data from millions of patients and apply that data to Deerfield's drug discovery and development expertise.

The two parties aim to address research gaps in the drug discovery process by drawing insights from Invitae's data platform. The platform includes sizable cohorts of rare disease patients. Through collaboration, the companies will analyze data from the more than 3.6 million genetic tests delivered by Invitae that are linked to longitudinal clinical data, which will strictly follow patients' preferences for sharing of their data. The companies believe that by combining Invitae’s data with Deerfield’s analytics expertise, they will be well-suited to uncover insights needed to discover and validate new rare disease targets, according to a company press release.

"Invitae's core objectives include expanding access to genetic information to improve healthcare for patients. Through this partnership, we intend to serve millions of families affected by rare diseases with high unmet needs," said Farid Vij, general manager of patient network and data at Invitae, in the release. "Our genetic and clinical data platform is highly enriched with patients possessing rare disorders, enabling a deeper understanding of the natural history and genetic basis of these diseases, and improving the success rate of new clinical programs."

"Despite significant research efforts, there are thousands of rare diseases without a line of sight toward a viable therapeutic intervention," said Matt Nelson, head of genetics and genomics at Deerfield Management, in the release. "Invitae's unique data and analytics capabilities position it to interrogate genetic effects on rare and many other diseases. With Deerfield's extensive experience in rare diseases and drug discovery, we believe there is strong potential for this partnership to uncover valuable patient insights and create important therapies for a range of currently untreatable conditions."

Under a new partnership, Invitae and Deerfield Management will harness genetic and clinical testing data from patients to discover potential novel therapeutics for treating rare diseases.

Invitae and Deerfield Management Team Up on Novel Therapeutics for Rare Diseases

Sanofi announced on March 16, 2023 that it will cut the US list price of Lantus (insulin glargine injection) 100 units/mL, a widely prescribed insulin, by 78%. The company will also establish a $35 per month cap on out-of-pocket costs for Lantus, which will apply to all patients with commercial insurance.

The price changes will go into effect on Jan. 1, 2024, and follows decisions made in June 2022 to lower diabetes medicines costs, such as the launch of an unbranded Lantus biologic at 60% less than the Lantus list price and a cap on out-of-pocket costs on insulin to $35 for all people without insurance. In addition, Sanofi will also cut the list price of its short-acting Apidra (insulin glulisine injection) 100 Units/mL by 70%.

In a similar move, Eli Lilly and Company announced in early March 2023 that 

it is capping its out-of-pocket cost for insulin

 to $35 per month for patients with commercial insurance and set lower list prices for its other insulin products (1).

“Sanofi believes that no one should struggle to pay for their insulin and we are proud of our continued actions to improve access and affordability for millions of patients for many years. We launched our unbranded biologic for Lantus at 60 percent less than the Lantus list price in June 2022 but, despite this pioneering low-price approach, the health system was unable to take advantage of it due to its inherent structural challenges,” said Olivier Bogillot, head of US General Medicines, Sanofi, in a company press release. “We are pleased to see others join our efforts to help patients as we now accelerate the transformation of the US insulin market. Our decision to cut the list price of our lead insulin needs to be coupled with broader change to the overall system to actually drive savings for patients at the pharmacy counter.”

1. Eli Lilly and Company. Lilly Cuts Insulin Prices by 70% and Caps Patient Insulin Out-of-Pocket Costs at $35 Per Month. Press Release, March 1, 2023.

Sanofi intends to cut its US list price for Lantus (insulin glargine injection) by 78% and cap out-of-pocket costs at $35 for all patients with commercial insurance.

Sanofi Drops Lantus US List Price by 78%, Capping Out-of-Pocket Costs at $35

On March 20, 2023, BioNTech and OncoC4, a US-based clinical-stage biopharmaceutical company, announced that they have entered into an exclusive worldwide license and collaboration agreement for the development and commercialization of OncoC4’s next-generation anti-cytotoxic T-lymphocyte–associated antigen 4 (CTLA-4) monoclonal antibody (mAb) candidate, ONC-392. The companies aim to co-develop and commercialize ONC-392 as a monotherapy or in combination therapy for various solid tumor cancer indications.

Under the agreement, OncoC4 will receive $200 million in an upfront payment. The company is also eligible to receive development, regulatory, and commercial milestone payments in addition to double-digit tiered royalties. The two companies will jointly develop ONC-392 as monotherapy and in combination with anti-programmed cell death (PD)-(L)-1 antibodies. The companies will be aiming for a range of solid tumor indications, including non-small cell lung cancer (NSCLC). The joint effort will continue until approval of the candidate is achieved. Both parties will share equally in the development costs for studies. The transaction is expected to close in the first half of 2023, dependent on customary closing conditions and regulatory clearances.

BioNTech will solely develop all combinations outside of the PD-1 inhibition indication—in particular all combinations with a compound in BioNTech’s pipeline. BioNTech will hold exclusive worldwide commercialization rights for any products it develops from these candidates, with OncoC4 participating in certain markets, which are to be negotiated in the future.

ONC-392 has received fast track designation from FDA as a monotherapy for immunotherapy-resistant NSCLC. It is also currently being evaluated in an additional Phase II trial as a combination therapy with pembrolizumab in platinum-resistant ovarian cancer.

“Despite being a prime target for more than a decade, we believe that targeting CTLA-4 has not reached its full potential in cancer immunotherapy," said Prof. Ugur Sahin, CEO and co-founder of BioNTech, in a company press release. "The data presented by OncoC4 on their ONC-392 antibody indicate a differentiated safety profile and encouraging clinical activity in various types of tumors. We believe that this antibody is a valuable addition to our immuno-oncology portfolio, whether used alone or in combination with our personalized immunotherapies.”

“Because of its specific mechanism of action, we believe ONC-392 has the potential to broaden the reach of CTLA-4-targeting immunotherapy,” said Yang Liu, co-founder, CEO and chief scientific officer of OncoC4, in the release. “We very much look forward to working hand-in-hand with BioNTech in developing ONC-392 for cancer indications with unmet medical needs.”

BioNTech will receive an exclusive worldwide license from OncoC4 to develop and commercialize its anti-CTLA-4 mAb candidate for solid tumor indications.