2seventy bio Sells Hemophilia A Program and Gene Editing Tech to Novo Nordisk for Up to $40 Million, Files Prospectus Amid Strategic Streamlining

On June 26 local time, 2seventy bio announced the completion of an Asset Purchase Agreement (APA) with Novo Nordisk. According to the terms of the APA, Novo Nordisk has acquired the rights to the Hemophilia A project and 2seventy bio's in vivo gene editing technology, which can be used to develop autologous or allogeneic cell therapies for immune cells to treat autoimmune diseases.

According to the terms of the agreement, 2seventy bio may receive payments of up to $40 million.2seventy bio to Transfer Hemophilia A Program to Novo Nordisk, Existing Collaboration Agreement to be Terminated. Additionally, the divestment will include the transfer of 2seventy bio's megaTAL technology and underlying intellectual property license.

According to the announcement by 2seventy bio, the employees of 2seventy bio currently involved in this project will also join Novo Nordisk to continue advancing the research. The project is based on the original research agreement established in 2019, which focuses on gene-editing therapy for patients with hemophilia A.

In fact, as early as January this year, the company spun off its preclinical and clinical cell therapy programs to Regeneron to form a new division.On January 30, 2024, Regeneron announced an agreement with 2seventy bio, under which Regeneron will obtain full development and commercialization rights to 2seventy bio's entire pipeline of novel investigational immunocyte therapies, as well as its research and clinical manufacturing capabilities, and will assume ongoing project, infrastructure, and personnel costs associated with these programs.

As 2seventy bio sells its pipeline, 160 of its employees, including Chief Scientific Officer Philip Gregory and Chief Medical Officer Steve Bernstein, will join Regeneron. After the transaction is completed, Philip Gregory will lead Regeneron's newly established R&D division, Regeneron Cell Medicines, to advance the development of the acquired projects as well as its own cell therapy programs.

Under the terms of the agreement at that time, Regeneron will pay 2seventy bio a $5 million upfront payment and make a milestone payment to it after the first project generated from the deal receives its first market approval.Through the collaboration with Regeneron, 2seventy bio has strategically adjusted its development, expecting to save approximately $150 million and $200 million in 2024 and 2025 respectively, including about $8 million in one-time restructuring costs.

It is not difficult to see that, compared with the biopharmaceutical transactions that often amount to hundreds of millions, the two collaborations by 2seventy bio this year can be said to offer maximum cost-effectiveness.

However, the original intention of 2seventy bio was not to rely on partnerships with large pharmaceutical companies or multinational corporations (MNCs) for lucrative returns or to leverage their resources to quickly advance product commercialization. Instead, they aimed to completely hand over pipelines, technologies, and relevant personnel to their partners, all in pursuit of "lightening the load" to go further.

After selling two R&D pipelines, 2seventy bio will partner with Bristol-Myers Squibb (BMS) to focus on the clinical development and commercialization of Abecma (idecabtagene vicleucel).Abecma is an anti-B-cell maturation antigen (anti-BCMA) directed CAR-T therapy. In March 2021, Abecma was approved by the U.S. FDA for marketing to treat relapsed/refractory multiple myeloma (r/r MM).

Not only is 2seventy bio keen on "selling off" and "dismantling" projects, but the company itself also originated from a spin-off.

In April 2021, 2seventy bio was spun off from gene therapy pioneer bluebird.Incorporated in Delaware, focusing on the development of innovative cell therapies and gene-editing treatments. The spun-off 2seventy bio not only inherited bluebird’s most valuable asset, Abecma, but also received a $441.5 million capital injection from bluebird.

As the world's first BCMA-CAR-T, Abecma once garnered significant attention. Priced at $419,500 per bag, it achieved sales of $164 million within just ten months of its launch and reached $297 million in sales in 2022. BMS/2seventy bio anticipates that Abecma will reach a peak sales range of $2 to $3 billion.

But with the arrival of Johnson & Johnson/Legend's Carvykti, the sales growth of Abecma has slowed down. 2seventy bio encountered survival difficulties less than two years after its spin-off. Faced with this predicament, 2seventy bio has placed all its hopes on Abecma, and whether it succeeds or fails is left to be seen over time.

Compared to the ups and downs experienced in recent years by gene therapy leader Bluebird and its spin-off 2seventy bio, Novo Nordisk has raked in substantial profits with semaglutide, emerging as the "new top influencer" in the pharmaceutical industry.

According to Novo Nordisk's 2023 financial report, its revenue last year was approximately $33.7 billion, a year-on-year increase of 31%, and its net profit was approximately $12.1 billion, a year-on-year increase of 52%. Among them, the combined sales of the three products of semaglutide were $21.157 billion. The sales of the injectable hypoglycemic drug Ozempic were $13.889 billion, a year-on-year increase of 60%; the sales of the oral hypoglycemic drug Rybelsus were $2.72 billion, a year-on-year increase of 66%. This product was recently approved for marketing in China; the sales of the weight loss version Wegovy were $4.548 billion, a year-on-year increase of 407%.

Abundant funds naturally provide a foundation for Novo Nordisk to layout various niche tracks.However, compared with other large multinational pharmaceutical companies, Novo Nordisk has been more focused since its establishment about a hundred years ago, without involving the entire industrial chain or multiple disease areas.

Currently, Novo Nordisk's R&D pipeline mainly focuses on four strategic areas: diabetes, obesity, rare diseases, cardiovascular and other severe chronic diseases. Projects entering Phase III clinical trials are primarily centered around the diabetes treatment field and the expansion of semaglutide indications.

Among them, Novo Nordisk has been frequently active in the niche field of blood diseases. According to incomplete statistics from Asymchem, Novo Nordisk has a total of 21 drugs in the blood disease sector, with the approved products mainly focusing on coagulation factors.

In addition, Novo Nordisk has been particularly active in the field of hemophilia A, a rare disease. In fact, this is not the first collaboration between Novo Nordisk and 2seventy bio. As early as 2019, 2seventy signed an agreement with its then-parent company Bluebird, based on which 2seventy spun off new operations to Novo Nordisk. In January 2022, Novo Nordisk expanded its cooperation agreement with 2seventy bio, introducing the latter's megaTAL technology platform to continue developing gene-editing therapies for hemophilia A.

In November last year, Novo Nordisk also entered into a collaboration and non-exclusive licensing agreement with Genevant Sciences to combine proprietary lipid nanoparticles (LNPs) with an innovative mRNA-based megaTAL technology for the development of an in vivo gene-editing therapy for hemophilia A.

We look forward to Novo Nordisk building on its strengths in metabolic diseases and achieving further success in new fields in the next century.

References:

1. "The Code of Novo Nordisk's Century Blueprint: The Power of Ultimate Focus"

2. "The Biotech Surviving Solely on BCMA-CAR-T"