Mining Biopharma's IP Storeroom: The Quest for Hidden Rare Disease Treatments

In a groundbreaking development for the rare disease community, SpringWorks Therapeutics has successfully brought a once-discontinued asset to market, showcasing the potential hidden within the pharmaceutical industry's intellectual property archives. This achievement not only highlights the value of rescuing shelved drug candidates but also sets a precedent for addressing unmet needs in rare disease treatment.

The SpringWorks Success Story

SpringWorks Therapeutics recently secured approval for Gomekli, a drug originally developed by Pfizer but later abandoned. This success story has ignited hope for patients with neurofibromatosis and other rare diseases. Annette Bakker, CEO of the Children's Tumor Foundation (CTF), estimates that there could be 30 more drugs currently in limbo that might benefit neurofibromatosis patients alone.

The journey of Gomekli from Pfizer's storeroom to FDA approval serves as a perfect case study for the industry. Jim Cassidy, SpringWorks' Chief Medical Officer, and Bakker believe this process can be replicated for numerous rare diseases. With Gomekli projected to generate approximately $760 million by 2030, the financial incentive for companies to explore dormant assets is significant.

The Challenge of "Ghost Assets"

The pharmaceutical landscape is littered with what Carl Schoellhammer, associate partner at DeciBio, calls "ghost assets." These are drug candidates developed by smaller biotechs that never reach the market, often due to financial constraints rather than scientific failures. The problem is particularly acute among platform companies and is expected to worsen as the industry shifts focus and companies restructure.

Schoellhammer predicts that areas like mRNA and cell therapy are likely to produce more of these abandoned assets in the coming years. For instance, CD19 CAR T programs may be deprioritized as the market becomes saturated, leaving potentially valuable treatments undeveloped.

Strategies for Rescuing Discontinued Assets

Industry experts are calling for a systematic approach to salvage promising drug candidates. Bakker suggests several strategies:

1. Creating an "investor club" willing to sustain companies through to the finish line
2. Developing biotech accelerators focused on discontinued assets
3. Establishing holding companies to acquire and advance shelved drug candidates
4. Collaborating with the FDA to cluster rare diseases by type, broadening treatment opportunities
5. Building infrastructure for public-private collaboration in drug development
6. Forming clinical trial consortia to facilitate the movement of drugs into the clinic

Policy solutions, such as tax incentives for companies willing to develop shelved assets and loosening FDA policies on old investigational new drug (IND) filings, could also play a crucial role in incentivizing the rescue of discontinued drugs.

The success of Gomekli demonstrates that with persistence, the right partnerships, and a patient-centric approach, valuable treatments can be brought out of retirement and into the hands of those who need them most. As the industry grapples with the challenge of abandoned assets, the SpringWorks model offers a beacon of hope for rare disease patients and a potential roadmap for unlocking the hidden potential within biopharma's intellectual property vaults.