Start-up aims to make personalized medicines. Is it scalable?

Julia Vitarello is in the unenviable role of having outlived her child. Her daughter, Mila, had a rare and fatal disorder called Batten disease. With Batten—best described as a collection of neurological symptoms, which can include blindness and seizures—the earlier symptoms appear, the shorter the person’s lifespan. Mila began to show symptoms at 3 years, was diagnosed at age 6, and died at the age of 10.

But for a while, it looked as if Mila would beat the odds. A fierce advocate for her daughter, Vitarello worked with a group of doctors, led by Timothy Yu at Boston Children’s Hospital, who developed an antisense oligonucleotide tailor-made for Mila’s specific genetic mutation. They called it milasen and got permission from the US Food and Drug Administration to use it on Mila through an expanded-access investigational clinical protocol. Milasen didn’t save her life, but it did appear to stabilize certain symptoms and reduce the frequency and severity of her seizures (N. Engl. J. Med. 2019, DOI: 10.1056/NEJMoa1813279).

Since then, Vitarello has dedicated her life to getting the medical world to repeat the experiment and create personalized medicines for individual patients, especially those suffering from rare and incurable illnesses like Batten. Much of Vitarello’s advocacy has been through her nonprofit, Mila’s Miracle Foundation, which helped fund the New England Journal of Medicine study.

Now, for-profit players have joined her effort.

Khosla Ventures, GV (formerly Google Ventures), Third Rock Ventures, and an unnamed health-care investment fund have all backed a new start-up Vitarello cofounded called EveryONE Medicines. The firm says it aims to develop “personalized genetic medicines” and, perhaps more ambitiously, to make such a venture scalable by setting up new testing and treatment protocols with regulators and insurers. “Julia’s done a fantastic job, over the last few years, carrying the torch for this,” says Kent Rogers, CEO of EveryONE.

“Technology is no longer the limiting factor,” Vitarello says in a statement. “We can now find the underlying genetic cause of disease in children like Mila and develop a medicine that targets it, even if unique to one or just a few patients. As patients with rare and common diseases are increasingly being categorized in smaller groups, there is a willingness to significantly change regulatory oversight and reimbursement allowing for an entirely new business model for many highly targeted medicines each for a few or single patients.”

Vitarello is not the first patient advocate to turn to the private sector for solutions. Companies including Solid Biosciences, Envoy Therapeutics, Tevard Biosciences, and Cerevance were started by relatives of people with the diseases they hope to treat: Duchenne muscular dystrophy, Dravet syndrome, and ataxia-telangiectasia, among others.

But drug development for rare diseases can be tricky. Scientific challenges often collide with bureaucratic ones. Stealth Biotherapeutics, which has been working on a drug for the ultrarare Barth syndrome, has struggled with shifting guidance from the FDA that has affected its business, including its ability to file for approval. The biotech isn’t expecting a decision from the agency until January—years after it completed clinical trials.

Drugs for rare diseases are also some of the most expensive, partly because of their potential markets are small. Orchard Therapeutics’ gene therapy for the neurodegenerative disease metachromatic leukodystrophy, which affects only about 40 children a year in the US, is priced at $4.25 million wholesale, making it the costliest drug in the world.

Rogers joined EveryONE largely to help solve that pricing problem. He was previously chief commercial officer of EQRx, which sought to bring cancer drugs to market at a fraction of the price of existing medicines primarily by licensing assets from Chinese biopharmas that had already done large-scale clinical trials. (EQRx folded last year after the FDA changed its stance on trials run in single countries, which dealt a serious blow to the company’s pipeline.) Rogers also previously managed United Health Group’s pharmacy benefit manager, Optum Rx, and held various market-access roles at Acorda Therapeutics, Schering-Plough, and Merck & Co.

“A lot of the cost in traditional drug development is that long stage of getting from the lab into the clinic and the process of going through the various phases of development,” Rogers says.

With EveryONE, he’s hoping to reduce the cost by creating a set of blueprints that can be quickly paired with individual patients, largely based on whole-genome sequencing. In theory, those genomic reports should reveal what toxicology studies need to be done, what type of antisense oligonucleotide—or eventually, another type of drug—might work best, and how long and how frequently the patient should be dosed, among other considerations.

“If we can do that, hopefully that yields a much lower cost of development, which will translate into a more scalable approach for a personalized medicine company,” Rogers says.

It’s a tall order. EveryONE Medicines currently has fewer than 10 full-time employees and an undisclosed amount of seed funding. Rogers is working on raising a series A round, whose target he declined to comment on. He also would not comment on whether Arch Venture Partners, where he is a partner, would be investing in the start-up.

Rogers plans to lean on his board of directors, as well as investors, for their resources and expertise. He says US, UK, and European regulators have already shown support for new accelerated pathways for individual therapies. The UK recently launched a pilot program, the Rare Therapies Launch Pad, in which Mila’s Miracle Foundation is playing a central role.

“There’s no real model right now for a scalable solution,” he says. “The interesting part about this is that the science and clinical odds of antisense oligonucleotides isn’t our challenge. It’s bringing a timely and collaborative approach to the research, approval, and access.”