Vertex Enlists Orum to Find Improved 'Preconditioning' Drugs

Vertex Pharmaceuticals is actively seeking to enhance patient preparation for intensive therapies, such as its sickle cell treatment Casgevy. On Tuesday, the company announced a collaboration with Orum Therapeutics to develop a new type of conditioning regimen that deviates from the traditional chemotherapies currently in use. According to the agreement, Vertex will invest $15 million to explore Orum's "degrader-antibody conjugates" (DACs) as a preconditioning agent for gene editing therapies. Following the research period, Vertex will have the option to license a DAC for each target included in the collaboration. Orum could potentially receive up to $310 million in development and commercial milestone payments for each of the three targets.

In December, Vertex and its partner CRISPR Therapeutics gained approval from the Food and Drug Administration for Casgevy, marking the first CRISPR gene editing-based treatment to reach the market. Casgevy is approved for treating sickle cell disease and severe beta thalassemia, with each patient treatment costing $2.2 million. Although Casgevy offers substantial, potentially life-long benefits, it requires patients to undergo chemotherapy first. The chemotherapy regimen can result in painful mouth sores, low blood cell counts, and organ damage. These side effects can be particularly challenging for older patients or those with already compromised organs. Furthermore, chemotherapy poses a high risk of infertility, complicating decisions for patients who wish to start a family.

These significant risks pose barriers to the widespread adoption of Casgevy, which Vertex anticipates will see a gradual rollout. Currently, the company provides fertility preservation services for commercially insured patients but is prohibited from offering these services to those covered by Medicaid. On Monday, Vertex took legal action against the U.S. government to change this restriction.

Both Vertex and other companies in the gene editing field are keen on developing new conditioning agents that are more tolerable than existing chemotherapy regimens. Orum Therapeutics perceived Vertex's interest in this area as an opportunity to expand its focus. Although primarily centered on oncology and immuno-oncology, Orum found it strategic to partner with Vertex, a more established leader in the gene editing space. Sung Joo Lee, Orum's CEO, remarked that they recognized an unmet need and believed they could address it effectively through this partnership.

A Vertex spokesperson emphasized that improving conditioning regimens could expand patient access to treatments like Casgevy. A safer and gentler conditioning process would minimize both the immediate side effects and long-term risks associated with the current chemotherapy approach, thereby enhancing the patient experience.

Orum's innovative research is part of a broader interest within the biotech industry in antibody-drug conjugates (ADCs), treatments that combine a targeting antibody with a toxic payload to precisely target and eliminate tumor cells. However, Orum's approach involves merging ADCs with targeted protein degradation. Instead of utilizing toxins, Orum pairs antibodies with compounds that degrade specific proteins. Lee expressed optimism that their approach would significantly improve the targeted therapy landscape.

This partnership marks Orum's second major deal within a year. In November, the biotech company entered into a collaboration with Bristol Myers Squibb, receiving $100 million upfront to advance its cancer research. Orum is currently developing a drug called ORM-5029, which is in a Phase 1 clinical trial targeting HER2-expressing solid tumors in breast cancer patients. The study is expected to be completed by 2025.