Moderna surfaces as the first partner for Jim Wilson's rare disease nonprofit, donating an mRNA program at no cost
A week after Jim Wilson and Alex Karnal debuted their Tachi Yamada-inspired nonprofit aimed at treating ultra-rare diseases, the partner providing the Institute for Life Changing Medicines with its first pipeline program has emerged from the shadows.
Moderna jumped into the spotlight Tuesday, announcing it had donated an mRNA-based therapy for Crigler-Najjar syndrome free of charge. In a press release, CEO Stéphane Bancel said Moderna would have had to charge a fortune if the company had gone on to develop it for profit given the ultra-rare nature of the disease.
But that’s exactly the kind of program ILCM wants, Karnal told Endpoints News.
“They share our recognition that there’s a market failure in the for-profit world leading to Crigler-Najjar patients being left behind,” Karnal said of Moderna. “It’s understandable from an economic perspective for that to happen … it speaks to the market today where these amazing technologies are not readily available for the people who need them.”
The candidate is called mRNA-3351, and Wilson says it originally emerged from a partnership he had with Alexion several years ago. Alexion ultimately exited the mRNA space and gave the full slate of programs to Moderna, with whom Wilson continued the collaboration.
What piqued Wilson’s interest here was the use of an mRNA therapy in the event of an acute crisis. Crigler-Najjar patients tend to need about 12 hours of phototherapy every day, largely characterized by sleeping under bright blue lights, in order not to develop jaundice. Sometimes, if a patient gets less of the therapy than needed, emergency intervention may be needed.
The Institute’s new program can step in here, Wilson says, as one potential application could be to stock pharmacies with an approved drug, allowing patients to receive treatment should the need arise. If patients also need to travel long distances on a plane, or through another method where bringing along their phototherapy beds is cumbersome, they can head to the pharmacy ahead of time and not have to worry about their disease.
“This would be in the pharmacy of hospitals or available quickly in stores, and if a patient gets in trouble or requires a major surgery or needs to travel, they get their infusion and they would then be maintained on their standard of care, which is blue lights,” Wilson said.
The other use Wilson envisions for the drug is as a maintenance therapy, replacing the blue lights altogether. Data from the program have shown in mouse models the therapy could prove effective for almost three weeks, he said, possibly allowing patients to visit a clinic at about the same rate.
Karnal also shed some more light on the priority review voucher system the Institute will utilize to bring in funds. The nonprofit plans on raising money to develop the Crigler-Najjar program solely through philanthropic donations, and because the candidate has received the FDA’s rare pediatric disease designation, Moderna and the Institute are eligible to receive a voucher should it be approved.
Whether or not Moderna decides to use or pass on the voucher, the Institute will receive funds tied to the value of the PRV or its proxy, which will then be used to bring in more pipeline programs eligible for the same designation as the Crigler-Najjar treatment. Once the Institute has seen multiple approvals, it will start selling contracts tied to the vouchers to continue bringing in more programs, Karnal said, creating a cycle of “self-sustaining independence.”
Ultimately, Karnal and Wilson are hopeful the Crigler-Najjar program is just the start of things to come. Wilson has researched this specific disease for a while and believes the trial design can win over regulators by using bilirubin — the compound implicated in jaundice — as a biomarker.
“We think there’s a good chance we could get the product registered by demonstrating a decrease in bilirubin without the need for clinical endpoints, as it’s as close to a validated clinical biomarker as cholesterol would be for familial hypercholesterolemia because it’s bilirubin that actually causes the toxicity in the brain,” Wilson said.
“We are poised to begin toxicology studies and submit briefing docs as we speak,” he added.