Which bio/pharma companies may be under the industry’s radar but are executing better-than expected performance and winning strategies for their companies? What products—development and commercial—are delivering? DCAT Value Chain Insights takes an inside look.

By Patricia Van Arnum, Editorial Director, DCAT, pvanarnum@dcat.org

Inside key deals
Deal-making among the large bio/pharmaceutical companies and smaller companies is an important source of product innovation in the industry. These deals are important to know what pipeline contenders or technologies that the large companies see value for their drug-development activities and in turn, puts these smaller companies on the industry’s map. Below is a roundup of some interesting deals thus far in 2024.

AbbVie (Gilgamesh Pharmaceuticals, FutureGen Pharmaceuticals, Medincell, Umoja Biopharma). AbbVie has made several interesting deals thus far in 2024. In May (May 2024), AbbVie and Gilgamesh Pharmaceuticals, a New York-based bio/pharmaceutical company, announced a collaboration and option-to-license agreement to develop therapies for psychiatric disorders, in a deal worth up to $2 billion ($65 million upfront and $1.95 billion in milestone payments). The collaboration will use AbbVie’s expertise in psychiatry disorders and Gilgamesh’s research platform to discover neuroplastogens.

In June (June 2024), AbbVie and FutureGen Biopharmaceutical, a Beijing-based bio/pharmaceutical company, entered a license agreement to develop FutureGen’s FG-M701, a TL1A antibody for treating inflammatory bowel disease currently in preclinical development, in a deal worth up to $1.71 billion ($150 million upfront and $1.56 billion in milestone payments). Under the agreement, AbbVie receives an exclusive global license to develop, manufacture, and commercialize FG-M701. FutureGen receives $150 million in upfront and near-term milestone payments and will be eligible to receive up to an additional $1.56 billion in clinical development, regulatory, and commercial milestones, as well as tiered royalties up to low-double digits on net sales.

Also, earlier this year (2024), AbbVie and Medincell, a French clinical- and commercial-stage bio/pharmaceutical licensing company developing long-acting injectable drugs, formed a collaboration to co-develop and commercialize up to six therapeutic products across multiple therapeutic areas and indications, in a deal worth up to $1.9 billion ($35 million upfront and $1.9 billion in milestone payments). 

AbbVie also partnered with Umoja Biopharma, a Louisville, Colorado-based bio/pharmaceutical company, under two exclusive option and license agreements to develop multiple in-situ generated CAR-T cell therapy candidates in oncology using Umoja’s VivoVec platform, in deals worth up to $1.44 billion. Umoja’s VivoVec gene-delivery platform combines lentiviral vector gene delivery with a T-cell targeting and activation surface complex. This enables T cells in the body to manufacture their own cancer-fighting CAR-T cells in vivo, thereby providing the potential to eliminate a number of challenges associated with traditional CAR-T approaches, including reliance on gathering a patient’s own or donor cells that are modified externally before being delivered back to the patient, the associated time lag and manufacturing challenges of ex vivo cell modification, and the need for patient’s lymphodepletion, according to information from AbbVie. 

The first agreement provides AbbVie an exclusive option to license Umoja’s CD19-directed in-situ generated CAR-T cell therapy candidates. This includes UB-VV111, Umoja’s lead clinical program for hematologic malignancies currently at the investigational new drug-enabling phase. Under the second agreement, AbbVie and Umoja will develop up to four additional in-situ generated CAR-T cell therapy candidates for discovery targets selected by AbbVie.

Boehringer Ingelheim (Ochre Bio, Ribocure Pharmaceuticals). In May (May 2024), Boehringer Ingelheim (BI) and Ochre Bio, an Oxford, UK-based bio/pharmaceutical company developing mRNA medicines for chronic liver diseases, formed a partnership focused on the discovery and development of regenerative treatments for chronic liver diseases (CLDs), such as late-stage metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis, in a deal worth up to $1 billion ($35 million upfront and $1 billion in milestone payments). 

Within the partnership, Ochre Bio will use its proprietary discovery platform that combines machine learning with human data, including advanced imaging and genomic phenotyping, as well as in-house RNA chemistry, and the ability to employ ex-vivo human-organ perfusion models. This will enable the identification, characterization, and validation of multiple regenerative targets for CLDs. BI aims to develop new treatments modulating regenerative targets with the potential to enhance the liver’s self-repair capabilities to prevent or reverse disease progression. 

In January (January 2024), BI, Suzhou Ribo Life Science, and its subsidiary, Ribocure Pharmaceuticals, entered a collaboration to develop small interfering RNA (siRNA) therapeutics for treating nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), in a deal worth up to $2 billion. 

NASH is an inflammatory liver disease that is caused by accumulation of fat in the liver. Over time, NASH causes scar-tissue formation, which in many cases leads to liver cirrhosis and related serious complications, including liver failure or liver cancer, according to information from the companies. 

Suzhou Ribo Life Science is a Kunshan, China-based clinical-stage bio/pharma company focused on the development of nucleic acid drugs and related products based on RNA interference (RNAi) technology, and Ribocure Pharmaceuticals is its subsidiary based in Mölndal, Sweden, focused on developing siRNA drugs. Ribo’s RIBO-GalSTAR platform enables the development of RNAi therapeutics targeting disease-causing genes specifically in hepatocytes by silencing their messenger RNAs (mRNAs). This approach has the potential to treat diseases addressing previously inaccessible drug targets, according to the companies. Under the agreement, Ribo will receive an upfront payment. In this multi-target collaboration, Ribo is entitled to receive success-based milestones for clinical, regulatory, and commercial success and tiered royalties with an overall deal value that exceeds $2 billion.

Bristol-Myers Squibb (Repertoire Immune Medicines). Bristol Myers Squibb (BMS) and Repertoire Immune Medicines, a Cambridge, Massachusetts-based bio/pharmaceutical company, entered a multi-year strategic collaboration to develop tolerizing vaccines for up to three autoimmune diseases, in a deal worth up to $1.9 billion ($65 million upfront and $1.8 billion in milestone payments). Repertoire Immune Medicines is involved in discovering the immune codes to design T cell receptor (TCR) bispecific molecules and mRNA cancer vaccines that selectively eliminate tumors in cancer patients and mRNA tolerizing vaccines that restore immune balance in patients with autoimmune disease. It develops mRNA tolerizing vaccines that are comprised of disease-relevant, tissue-specific epitopes that have been discovered using its proprietary DECODE platform. These epitopes are encoded by mRNA and encapsulated in a proprietary lipid nanoparticle together with an mRNA-encoded immune modulator. The company was founded in 2019 by Flagship Pioneering and operates from sites in Cambridge, Massachusetts and Zurich, Switzerland.  

Eli Lilly and Company (Haya Therapeutics). In September (September 2024), Eli Lilly and Company and Haya Therapeutics, a Lausanne, Switzerland-based bio/pharma company focused on developing RNA-based therapeutics, entered a multi-year agreement to apply Haya’s RNA-guided regulatory genome platform to support Lilly’s preclinical drug discovery efforts in obesity and related metabolic conditions, in a deal worth up to $1 billion. Haya is a precision medicines company developing programmable therapeutics targeting regulatory RNAs derived from the dark genome, a cell information processing unit, to reprogram pathological cell states for a broad range of diseases, including cardiovascular and metabolic diseases and cancer. The company is using its technology platforms to gain insights into the biology of disease cell states and the long non-coding RNAs (IncRNA) that regulate them. HAYA’s lead therapeutic candidate is HTX-001, in development for the treatment of heart failure. HAYA is also developing a pipeline of lncRNA-targeting precision therapies for the cell-specific treatment of diseases in other tissues.

Gilead Sciences (Nurix Therapeutics). Earlier this year (2024), Gilead Sciences extended its research pact with Nurix Therapeutics, a San Francisco, California-based clinical-stage bio/pharmaceutical company using small-molecule drugs to activate or inhibit the natural process of protein degradation, in a deal worth up to $1.8 billion ($15 million upfront and $1.8 billion in milestone payments).

Under the agreement, Nurix is deploying its proprietary drug-discovery platform to identify agents that use E3 ligases to induce degradation of specified drug targets. Gilead has an option to license drug candidates resulting from the work, and Nurix retains co-development and co-detail options on up to two programs in the US, subject to certain restrictions. For those programs that Nurix opts in to co-develop and co-detail, the parties will split development costs as well as profits and losses 50/50 for the US, and Nurix will be eligible to receive royalties on ex-US sales and reduced milestone payments. The companies originally partnered in 2019, when, Gilead made an upfront payment of $45 million. In extending the agreement,  Nurix will receive a $15-million extension fee and remains eligible for up to $73.5 million in preclinical milestones and potential future licensing payments, and up to a total of $1.7 billion in potential future development, regulatory, and sales milestones as well as royalties on future products.

GlaxoSmithKline (Flagship Pioneering). Earlier this year (2024), GSK and Flagship Pioneering, a private equity firm that funds new bio/pharmaceutical companies (bioplatform companies), entered a collaboration with the goal of discovering and developing a portfolio of medicines and vaccines, starting in respiratory and immunology, in a deal worth up to $870 million ($150 million upfront and $720 million in milestone payments).

GSK and Flagship will initially fund up to $150 million upfront to support an exploration phase to identify the most promising concepts for further research and development with Flagship’s bioplatform companies. From these explorations, the collaboration aims to identify a portfolio of up to 10 novel medicines and vaccines that will each be subject to an exclusive option by GSK for further clinical development. Under the agreement, Flagship and its bioplatform companies will be eligible to receive up to $720 million in upfront, development,  and commercial milestones from GSK, as well as preclinical funding and tiered royalties, for each acquired program.

Merck KGaA (Caris Discovery). In April (April 2024), Merck KGaA and Caris Discovery, the therapeutic research arm of Caris Life Sciences, an Irving, Texas-based bio/pharmaceutical company, formed a multi-year strategic partnership to accelerate the discovery and development of antibody-drug conjugates (ADCs) for treating cancer, in a deal worth up to $1.4 billion. Caris Discovery applies the scale of Caris’ core molecular-profiling business to discover druggable targets that would be otherwise inaccessible through more traditional approaches. Under the agreement, Merck KGaA will provide Caris with an upfront payment as well as research funding. In addition, Caris will be eligible for discovery, development, regulatory and sales-based milestone payments that may total up to $1.4 billion along with tiered royalties. Merck KGaA will receive an exclusive global license to develop, manufacture and commercialize ADC therapeutics for selected targets.

Novartis (Borealis Biosciences, Dren Bio, PeptiDream, Voyager Therapeutics, Arvinas). Novartis has been involved with several noteworthy deals. In August (August 2024), Novartis partnered with Versant Ventures, a venture-capital firm, to form Borealis Biosciences, an independent, discovery-stage biotechnology company based in Vancouver, Canada, focused on developing xRNA-based medicines for kidney disease.

Borealis stems from Novartis’ $3.5-billion acquisition of Chinook Therapeutics, a Seattle, Washington-based clinical-stage bio/pharmaceutical focused on developing therapeutics for kidney disease. Chinook was founded by Versant in 2019 and acquired by Novartis in 2023 for $3.2-billion upfront and up to $300 million in a contingent value right achievable upon certain milestones. Following the acquisition, Novartis leveraged the scientific potential of legacy Chinook early research talent, site facilities, and capabilities, with financing from Versant, to launch Borealis. With its acquisition of Chinook in 2023, Novartis gained two late-stage drug candidates (atrasentan and zigakibart) to treat immunoglobulin A nephropathy (IgAN), a type of kidney disease where antibodies build up in the kidneys and cause damage to the glomeruli (small filters inside the kidneys).

Borealis received combined Series A financing of $150 million from Novartis and Versant, and Novartis has transferred certain employees and the former Chinook site in Vancouver to Borealis to enable the continuation of drug-discovery efforts by certain former researchers of Chinook. An additional part of this deal includes a near-term research funding agreement, which provides Novartis the option to acquire two future development-ready programs under pre-agreed terms to complement Novartis’ core strategic priorities in renal medicine and xRNA technology platforms. Borealis is eligible to receive up to $750 million in total consideration, including clinical and regulatory milestones.

In July (July 2024), Novartis and Dren Bio, a Foster, California-based bio/pharmaceutical company focused on antibody therapeutics for cancer, autoimmune, and other diseases, entered into a strategic collaboration worth up to $3 billion ($150 million upfront, including an equity investment by Novartis in Dren Bio of $25 million, and $2.85 billion in milestone payments). The collaboration will focus on the discovery and development of therapeutic bispecific antibodies for cancer using Dren Bio’s proprietary Targeted Myeloid Engager and Phagocytosis Platform. Dren Bio and Novartis will collaborate to advance selected targeted myeloid engager programs in oncology through clinical candidate selection, at which point Novartis will assume full responsibility for all remaining development, manufacturing, regulatory, and commercialization activities.

In May (May 2024), Novartis and PeptiDream, a Kanagawa, Japan-based bio/pharmaceutical company, announced an expansion of their peptide discovery collaboration, in a deal worth up to $2.9 billion ($180 million upfront and $2.7 billion in milestone payments). Under the agreement, PeptiDream will use its proprietary Peptide Discovery Platform System technology to identify and optimize macrocyclic peptides against targets selected by Novartis, for potential conjugation to radionuclides or other applications for both therapeutic and diagnostic purposes. 

Also, earlier this year (2024), Novartis and Arvinas, a New Haven, Connecticut-based clinical-stage biotechnology company developing drugs based on targeted protein degradation, entered into an exclusive strategic license agreement for the worldwide development and commercialization of ARV-766, a prostate cancer drug, in a deal worth up to $1.1 billion ($150 million upfront and $1 billion in milestone payments). 

ARV-766 is an investigational orally bioavailable protein degrader designed to selectively target and degrade the androgen receptor (AR). The androgen receptor is a protein that binds male hormones called androgens. Androgen receptors are found inside the cells of male reproductive tissue, some other types of tissue, and some cancer cells. In prostate cancer, androgens bind to androgen receptors inside the cancer cells, which causes the cancer cells to grow. Arvinas uses its proprietary Protac  discovery-engine platform to engineer proteolysis targeting chimeras, or Protac-targeted protein degraders, which are designed to use the body’s own natural protein disposal system to selectively degrade and remove disease-causing proteins.

In January (January 2024), Novartis and Voyager Therapeutics, a Lexington, Massachusetts-based bio/pharmaceutical company, entered a strategic collaboration and capsid license agreement to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA), in a deal worth up to $1.3 billion ($100 million upfront and $1.2 billion in milestone payments). Voyager will provide Novartis with a target-exclusive license to access Voyager’s Tracer capsids and other intellectual property for the respective diseases, and Voyager and Novartis will collaborate to advance a preclinical gene-therapy candidate for HD.

Under the agreement, Novartis agreed to pay Voyager $100 million of consideration upfront, including a $20-million purchase of newly issued equity in Voyager. Voyager is eligible to receive up to $1.2 billion in preclinical, development, regulatory, and sales milestones, as well as tiered royalties on global net sales of products incorporating Voyager’s Tracer capsids. Novartis will obtain target-exclusive access to Voyager’s Tracer capsids related to SMA for the duration of the agreement and will be responsible for all development and commercialization. Novartis will also receive worldwide rights to Voyager’s adeno-associated virus gene therapy for HD using Voyager’s capsids and proprietary payloads. Voyager will be responsible for preclinical advancement, and Novartis will be responsible for all clinical development and commercialization for the HD program. Novartis previously exercised options to license novel capsids generated from Voyager’s Tracer capsid discovery platform for use in gene-therapy programs against two undisclosed neurological disease targets.

Novo Nordisk (Neomorph, Omega Therapeutics, Cellarity, Flagship Pioneering Medicines). In February (February 2024), Novo Nordisk and Neomorph, a San Diego, California-based bio/pharmaceutical company, entered into a collaboration and licensing agreement to discover, develop, and commercialize molecular glue degraders for treating cardiometabolic and rare diseases in a deal worth up to $1.46 billion. Neomorph is focused on advancing targeted protein degradation to destroy undruggable proteins in the development of therapeutics. 

Under the agreement, Neomorph receives an upfront and near-term milestone payments, plus R&D funding. Neomorph is also eligible to receive future clinical, commercial, and sales milestone payments to bring the total potential deal value for multiple targets to $1.46 billion, plus tiered royalties. Neomorph will lead discovery and preclinical activities against selected targets with Novo Nordisk having the right to exclusively pursue further clinical development and commercialization of the compounds.

Also in 2024, Novo Nordisk formed two separate research pacts, respectively with Omega Therapeutics, a Cambridge, Massachusetts-based bio/pharma company, and Cellarity, a Somerville, Massachusetts-based bio/pharmaceutical company, worth up to $532 million with each company (approximately $1.1 billion combined). 

These are the first two programs signed under the framework collaboration between Flagship Pioneering Medicines and Novo Nordisk to use Flagship’s bioplatform companies (Omega, Cellarity) to develop treatment approaches for cardiometabolic diseases. Under the agreement with Omega, the companies will use Omega’s platform technology to develop an epigenomic controller designed to enhance metabolic activity as a part of a potential new treatment approach for obesity management. Under the agreement with Cellarity, the companies will aim at biological drivers of metabolic dysfunction-associated steatohepatitis (MASH), a chronic and progressive liver disease, and will use Cellarity’s platform to develop a small-molecule therapy against this disease.

Each company, Novo Nordisk, and Flagship Pioneering Medicines will jointly advance these respective programs through preclinical development and conduct foundational activities, after which point, Novo Nordisk could advance the programs into clinical studies. Under the terms of the respective agreements, Novo Nordisk will reimburse R&D costs. Additionally, each agreement may pay up to $532 million in upfront, development and commercial milestone payments, as well as tiered royalties on annual net sales of a licensed product, to be shared between the respective companies and Flagship’s Pioneering Medicines.

Pfizer (Quotient Therapeutics). In September (September 2024), Pfizer also partnered with Flagship Pioneering and Quotient Therapeutics, a Nottingham, UK-based bioplatform company of Flagship Pioneering, in a strategic partnership under which the companies will analyze somatic mutations that occur in diseased patient tissue to inform the discovery and development of therapies for cardiovascular and renal diseases.

Pioneering Medicines, Flagship’s in-house drug-discovery and development unit, is responsible for leading the strategic partnership with Pfizer, including driving the exploration process for potential drug development programs built on Flagship’s bioplatforms and modalities. The Quotient agreement is the most recent program initiated under Flagship’s strategic partnership with Pfizer that was announced in July 2023.

Takeda (AC Immune, Ascentage Pharma, Degron Therapeutics) Takeda has partnered in several interesting deals this year, including two deals with China-based bio/pharmaceutical companies. In May (May 2024), Takeda and AC Immune, a Cambridge, Massachusetts-based bio/pharmaceutical company, announced an exclusive, worldwide option and license agreement for AC Immune’s active immunotherapies targeting toxic forms of amyloid beta, including AC Immune’s ACI-24.060 for the treatment of Alzheimer’s disease, in a deal worth up to $2.2 billion ($100 million upfront and $2.1 billion in milestone payments).

ACI-24.060 is an anti- amyloid beta (Abeta) active immunotherapy candidate in Phase Ib/II development, designed to induce an antibody response against the toxic forms of Abeta believed to drive plaque formation and Alzheimer’s disease progression. By inducing plaque clearance and inhibiting plaque formation in the brain, ACI-24.060 has the potential to delay or slow Alzheimer’s disease progression. 

In June (June 2024), Takeda announced a deal with Ascentage Pharma, a bio/pharmaceutical company based in Rockville, Maryland, and Suzhou, China, for an exclusive license option agreement for Ascentage’s olverembatinib, for treating chronic myeloid leukemia (CML) and other hematological cancers, in a deal worth up to $1.3 billion ($100 million upfront, $1.2 billion in potential milestones, plus an equity investment by Takeda in Ascentage Pharma).

Olverembatinib is a BCR-ABL tyrosine kinase inhibitor (TKI) and is currently approved and marketed in China for the treatment of adult patients with TKI-resistant chronic-phase CML (CP-CML) or accelerated-phase CML (AP-CML) harboring the T315I mutation and in adult patients with CP-CML resistant to and/or intolerant of first- and second-generation TKIs. Olverembatinib has been granted orphan drug designation and fast-track designation by the US Food and Drug Administration and orphan designation by the European Medicines Agency. 

In May (May 2024), Takeda and Degron Therapeutics, a Shanghai-based bio/pharmaceutical company, entered into an agreement to discover and develop molecular glue degraders for multiple targets in oncology, neuroscience, and inflammation, in a deal worth up to $1.2 billion.

Degron and Takeda will collaborate to use Degron’s GlueXplorer platform to identify, validate, and optimize molecular glue degraders for specific therapeutic targets selected by Takeda. Upon reaching a certain stage of advancement, the projects would be transitioned to Takeda for further development and commercialization. Under the agreement, Degron Therapeutics will receive an upfront payment and is eligible to receive potential future preclinical, clinical development and commercial milestone payments that could total $1.2 billion if all related milestones are achieved over the course of the agreement. Degron is also eligible to receive tiered royalty payments on sales of any potential commercialized products. The parties have the option to expand the collaboration to include more targets. Additionally, Takeda will make an equity investment in Degron. Degron Therapeutics will retain full ownership of its pipeline programs.