Eli Lilly Pipeline Analysis: Gene Therapy Products Accounts for 1/3, What Diseases Are Targeted?

On January 8, 2024, the FDA announced a pilot program called “CoGenT Global”, which stands for “Gene Therapy Global Pilot Cooperation.” The program mentioned that the FDA will collaborate with other global regulatory partners, including the World Health Organization and members of the International Council for Harmonisation (ICH), which includes the European Union (EU), Japan, Canada, and Switzerland, to explore a common approval process to provide a more favorable regulatory environment for the field of gene therapy.

At an industry conference in July this year, Peter Marks, the director of the Center for Biologics Evaluation and Research (CBER) at the FDA, indicated that the program may be expanded in the future.

As a relatively new technology that started late, gene therapy has developed rapidly in recent years, with many products already on the market, showing an excellent application prospect. At the same time, driven by policy and the industry’s desire for new tracks, many pharmaceutical companies continue to enter this field.

Recently, the opening of Eli Lilly’s Boston research and development center, which is mainly used for gene medicine research and development, has once again made the industry realize the weight of gene therapy in the eyes of MNCs (Multinational Corporations).

Eli Lilly Pipeline: Gene Therapy’s Share Rises from 1/4 to 1/3

Recently, the foreign media outlet Endpoints News published an interview article with Eli Lilly’s Chief Scientific Officer Dan Skovronsky and Andrew Adams, Senior Vice President of Neurodegenerative Disease Research and Co-Director of the Lilly Institute for Genetic Medicine.

The article points out that compared to last year’s pipeline distribution, the number of gene therapy pipelines at Eli Lilly has significantly increased, with nearly 1/3 of the current Eli Lilly pipeline being gene therapies. Most of these additional gene therapy pipelines are still in the early clinical stages.

According to the pipeline information available on Eli Lilly’s official website, the company is currently focusing on five major disease areas: cancer, metabolic diseases, autoimmune diseases, neurodegenerative diseases, and pain. Among them, the layout of its gene therapy is mainly concentrated in the fields of metabolic diseases and neurodegenerative diseases.

In the field of metabolic diseases, Eli Lilly’s gene therapy pipeline mainly consists of small nucleic acid drugs, used for the treatment of some cardiovascular diseases and chronic liver diseases.

1. LY3819469 (Lepodisiran), a GaINAc-conjugated siRNA, is currently in Phase III clinical trials and is the most advanced siRNA drug in Eli Lilly’s pipeline, developed for the treatment of atherosclerotic cardiovascular disease (ASCVD).

2. LY3561774 (Solbinsiran), an siRNA drug targeting liver ANGPTL3 expression, is currently in Phase II clinical trials and is also being developed for ASCVD.

3. APOC3 siRNA, developed for the treatment of cardiovascular diseases.

4. PNPLA3 siRNA, developed for the treatment of metabolic dysfunction-related steatohepatitis (MASH).

5. SCAP siRNA, also developed for the treatment of MASH.

In the field of degenerative diseases, Eli Lilly’s cell and gene therapy (CGT) products are mainly gene therapy products:

1. GBA1 Gene Therapy: Parkinson’s disease and Gaucher’s disease.

2. GRN Gene Therapy: Frontotemporal dementia.

3. OTOF Gene Therapy: Hearing loss.

Eli Lilly’s layout in the field of gene therapy has not always been a focus of attention for the media and industry insiders, but judging from the current share of gene therapy in Eli Lilly’s pipeline, this will be one of the key directions for Eli Lilly’s future efforts.

In fact, Eli Lilly has not been inactive in the field of gene therapy in previous years.

In January 2021, Eli Lilly completed the acquisition of Prevail Therapeutics, which played a key role in the early layout of Eli Lilly’s gene therapy products.

In February 2022, Eli Lilly announced the establishment of the Institute for Genetic Medicine and announced an investment of about $700 million in the construction of the Boston Seaport. Its Boston R&D center has recently been officially opened. Also in 2022, Eli Lilly acquired Akouos.

In addition, in June 2023, Eli Lilly reached a cooperation agreement with Verve Therapeutics for the first time, which advanced Eli Lilly’s gene therapy projects in the field of cardiovascular diseases.

When Gene Therapy Moves Toward Common Diseases

In addition to Eli Lilly, Novo Nordisk also expressed its preference for gene therapy products earlier this year.

Jacob Sten Petersen, Senior Vice President of Global Nucleic Acid Therapies at Novo Nordisk, said in an interview with Endpoints News that Novo Nordisk is investing profits from Ozempic and Wegovy into the research of gene therapy drugs to achieve “greater weight loss effects.” Petersen introduced that by blocking, editing, or fine-tuning the output of genes, Novo Nordisk hopes to develop therapies that induce the body to burn fat and counteract muscle loss caused by existing drugs.

The pipeline layout updates of these two leading pharmaceutical companies, Eli Lilly and Novo Nordisk, are confirming the vision of gene therapy proposed by Peter Marks, the director of CBER: many years later, perhaps many basic diseases can also be treated with gene therapy. For example, patients can choose to use gene therapy to reduce the level of cholesterol in the blood.

In the past, most gene therapies approved by the FDA were used for the treatment of patients with rare diseases (click “Read the Original” at the end of the article to see which CGT therapies have been approved by the FDA).

The Orphan Drug Act enacted in the United States in 2002 defines rare diseases as diseases or conditions affecting fewer than 200,000 people in the United States. According to FDA data, there are currently more than 7,000 rare diseases in the United States, affecting more than 30 million Americans.

In the fields of degenerative and metabolic diseases that Eli Lilly is currently laying out, the incidence and number of patients of most diseases are far higher than those of rare diseases. Taking ASCVD as an example, this disease is the disease with the highest morbidity and mortality rate in the world.

When gene therapy is developed for diseases with higher incidence rates, on the one hand, a larger patient base will directly disperse the costs of product research and development and production; on the other hand, to meet market demand, suppliers will inevitably optimize the production process and efficiency of such products, and the cost of the product will be reduced accordingly. With the joint action of other factors such as policies, the price of gene therapy will also drop significantly.

And as more research on gene therapy is initiated, the development of this field of technology will also accelerate. Perhaps one day, even gene therapies for diseases with very low incidence rates will no longer be out of reach.

【Editor’s note】The above content (~6092 words) is a quick translation of a Chinese article by DrugTimes team. To read the original article, please click here. All comments are warmly welcome. Many thanks!