Startup profile: Mitochon Pharmaceuticals
- Founded by: Robert Alonso,John Geisler
- Year founded: 2015
- Headquarters: Blue Bell, PA
- Sector: Pharmaceuticals
- Funding and valuation: $16 million
- Key ecosystem partners: Ben Franklin Technology Partners
For patients with ALS, even a small gain can be enormous. In an early-stage trial, a new once-a-day drug has shown promising signs.
Blue Bell-based Mitochon Pharmaceuticals completed the clinical study in Europe, involving 18 patients over two weeks, with the goal of testing whether its treatment can help slow the disease by stabilizing nerve cells. During that time, one person was able to button their shirt for the first time in months.
“What our drug does is basically restore the energy to the cell.”
Roboert Alonso, Mitochon
“What our drug does is basically restore the energy to the cell, and by doing that, it prevents the cells from dying,” Mitochon cofounder Robert Alonso told Technical.ly.
ALS, or amyotrophic lateral sclerosis, progressively destroys motor neurons, stripping patients of the ability to move, speak and eventually breathe. Only a small number of drugs have been approved, and many experimental therapies have failed in late-stage trials. That track record has made investors cautious.
The disease’s progression can be rapid and severe, with many patients dying within three years of diagnosis. In the US, around 30,000 people currently live with ALS, with around 5,000 people receiving a diagnosis each year.
In the initial study of the once-daily 2.5mg oral tablet, the company also observed biomarkers suggesting cells were “not dying as much as in a placebo control group,” Alonso said. The company is still reviewing the full data.
“There’s no such thing as a cure right now,” Alonso said. “But if you could slow progression down dramatically, if someone could sustain the place they’re at when they start the drug, that would be meaningful.”
If confirmed in larger trials, that kind of stabilization could represent a shift in how ALS is managed.
Turning mitochondrial research into a startup
Before its founding in 2015, Mitochon traces back to research conducted at Johnson & Johnson’s facility in Spring House, PA.
Co-founder and chief scientific officer John Geisler had been working on mitochondrial-targeted compounds there before leaving to pursue the idea independently. He later connected with Alonso, a self-described serial entrepreneur who had recently sold another biotech company.
The startup initially explored obesity and diabetes before shifting its focus to neurodegenerative diseases, including ALS, Parkinson’s disease and Alzheimer’s disease, which Alonso said presented a greater unmet need.
State-backed venture fund Ben Franklin Technology Partners invested in the early days, after previously backing Alonso’s prior company. Once BFTP committed, Alonso said, he brought in other investors who had supported him before.
To date, Mitochon has raised roughly $16 million, per Alonso. About half has come from private investors, and about half from non-dilutive funding sources (meaning they don’t take equity), including the National Institutes of Health, the US Department of Defense and the Michael J. Fox Foundation.
“The Department of Defense gave us a lot of money for traumatic brain injury,” Alonso said. “They’re interested in it for soldiers.”
Alonso said that when the drug is given within hours of injury in preclinical work, it appears to spare tissue from being destroyed by stabilizing cellular energy production.
Beyond the first 18 patients
Next up is a larger, longer trial.
Mitochon plans to begin a 90-day study involving about 60 ALS patients, which would run into 2027. The study will include an open-label extension, which allows patients in the placebo arm to switch to the active drug, pending FDA guidance.
Clinical trials in ALS are expensive. Alonso said the upcoming study is expected to cost about $5 million. Earlier studies cost roughly $1 million each. Without revenue, the company relies on investor support and grants to continue advancing the program.
“Although we’ve got really exciting early data, everybody wants to see more,” Alonso said. “That’s been the challenge, trying to continue to drive the company forward and continue to get new data.”
