GLPG-5101

iStock, cyano66 SpringWorks Therapeutics is the perfect case study for rescuing a discontinued assets. It’s time to repeat the process for every rare disease, experts say. The biopharma industry has never been better at making drug candidates than it is in this moment. But not everything that comes out of the drug discovery engine will get developed, even if curative. It’s hard to comprehend the scope of the drugs, research and intellectual property sitting on pharma shelves, or being lost as biotechs shut down. Annette Bakker, CEO of the Children’s Tumor Foundation (CTF) that supported the development of SpringWorks Therapeutics’ newly approved rare disease drug Gomekli, estimates that there are probably 30 more drugs out there right now that could help patients with neurofibromatosis if they weren’t in limbo. “This is one drug, but it’s definitely not the last one,” Bakker said. “And what we actually really hope is that this may trigger a motivation for some other companies.” Gomekli’s journey from Pfizer’s storeroom to approval is a perfect case study that Bakker and SpringWorks Chief Medical Officer Jim Cassidy say could be replicated over and over. “What we’re building now can actually be copied to every rare disease under the sun,” Bakker said. Gomekli is now estimated to bring in about $760 million by 2030, according to Bakker, so the opportunity is real for companies willing to wade into the dust and cobwebs of pharma’s attic. “There is just a whole energy here that I think is worth exploring now that we have a success, Bakker said. “How can we make this a sustainable endeavor going forward? Because for [Gomekli], this . . . was a fantastic experience, but now we need to think about making this a scalable opportunity.” The Ghosts of Biotechs Past Smaller biotechs also churn out assets that never see the light of the day. Carl Schoellhammer, associate partner at consulting firm DeciBio, calls these “ghost assets.” Myriad reasons can drive a biotech out of business, but it’s not always because a drug failed. A company may simply be unable to secure a new round of fundraising. This leaves them attempting a fire sale of the assets, or they simply fade away and the science is never seen again, Schoellhammer explained. The issue is particularly prominent among platform companies, which were all the rage a few years ago but have petered out as the industry turned to focus on single assets with data behind them. Bakker thinks the problem is getting worse. “You see that a lot of companies are restructuring these days, and I think we’re going to have even a more pronounced wave of what I call shell drugs that actually will never see the patient. And for a rare disease, that is a problem.” Schoellhammer predicts that areas like mRNA or cell therapy are likely to create these poltergeists in the years to come. Companies that launched in the heyday of COVID to advance mRNA have not really figured out the next phase of this technology. Even Moderna, the most famous name in the mRNA game, is struggling to find the right indications for its tech. For cell therapy, Schoellhammer predicts that CD19 CAR T programs will likely be cast off in the years to come. Several of these therapies have already been approved, leaving dozens still in development. Galapagos just deprioritized CD19 CAR T candidate GLPG5201 after years of development, he noted. For a lot of smaller cell therapy companies, they raised funds when the industry was at a high. “Everyone thought that cell therapies are gonna be the end all be all,” Schoellhammer said. “There’s some question marks [now], and they couldn’t raise again.” Galapagos is well capitalized with about €500 million ($525.8 million) in the bank to last into 2028 and can afford to move onto the next candidate in line, in this case GLPG5101. A smaller company cannot. When facing this problem, biotechs can try and sell the assets off. If they can’t find a buyer, they could pay to maintain the IP, but that’s a huge expense that a failing biotech probably does not have the cash to cover. A venture capital firm that had backed the company is unlikely to want the hassle of trying to form a new company around an old asset, Schoellhammer said. If the IP came from a research organization, it can be transferred back there. But often the assets drift off never to be seen again. According to Bakker, drugs from small biotechs can get lost in an acquisition focused on another drug. If the smaller company had filed an investigational new drug application for another asset with the FDA prior to being acquired, no one can access those data because the smaller company no longer exists after it’s absorbed into a bigger one. The data are simply lost. “That, I think, is a huge problem for the patients, but also it’s pretty unethical that we would have to do these toxicology studies on cats and dogs because we can’t access that data,” Bakker said. Policy solutions could help incentivize development of shelves assets, too. Bakker suggested tax incentives for companies and that the FDA loosen its policies on old IND filings to ensure that data are not lost if a company goes away. According to Cassidy, the key for getting Gomekli across the FDA’s finish line was CTF. And if you ask Bakker, it was having a company like Pfizer that was willing to spin out the intellectual property, and then the volunteers to make it happen. Cassidy said SpringWorks was very lucky that Pfizer was willing, but that’s not always the case. “It’s all very well making a fuss over a drug that’s been discontinued. . . . Most drug companies, they’ll be deaf to that from the outside world. You have to be really persistent, really tenacious and really go for the right people in the company . . . and influence them in the right way,” Cassidy said. “We can’t do that. That’s got to be a CTF-like organization.” Investors also need to be identified who would be willing to take a chance and form new entities around these orphaned assets. Bakker also thinks that biotech accelerators could focus on them. “What I’m working on now is creating almost like an investor club of people that are willing to sustain these companies to make sure that they make it to the finish line,” Bakker said. Both Cassidy and Schoellhammer said that another strategy to capturing dismissed assets is via a holding company, which would pick up assets being sold off as companies shut down and assemble the resources to advance them. Now that Gomekli is approved, Bakker is eager to share her expertise and move on to the next potential asset. She is working with the FDA, Sanofi and the Myhre Syndrome Foundation to see if rare diseases can be clustered together by type to broaden the treatment opportunities. CTF is also developing an infrastructure to help public and private institutions collaborate to develop drugs efficiently and a clinical trials consortium to facilitate the movement of drugs into the clinic. “Our approach is, let us build a prototype that actually can be expanded to other rare disease,” Bakker said. “So it’s not just for NF.” Editor’s note: The following story is part one in a two part story looking at discontinued assets in biopharma. You can read Part One, How SpringWorks Took Pfizer’s Rare Disease Ghost Drug And Got it to Patients , here .