Drug Development

Backed by a biotech legend, two young entrepreneurs tackle one of the Holy Grails of R&D

Joshua Cohen

Joshua Cohen

It’s not often that two recent college grads can come up with enough cash and connections to run a Phase II study of a new combination drug for a tough and deadly disease like ALS. But with some backing by biotech legend Henri Termeer and $8 million in venture cash and grant money, Justin Klee and Josh Cohen say they’re ready to turn what started out as an undergraduate science project at Brown into a clinical reality in a matter of months at a startup dubbed Amylyx.

The key component in all this, the money to start dosing patients, comes from a $5 million Series A led by Morningside Venture with contributions from the ALS Investment Fund and former Genzyme CEO Termeer. Combined with a $3 million grant from the ALS Accelerated Therapeutics Initiative and some discount pricing from a network of hospitals engaged in ALS work, and Klee and Cohen say they’ve reached the threshold of a mid-stage trial that will look for both efficacy and safety data.

Justin Klee

Justin Klee

“It is atypical,” Klee allows in our telephone interview this morning. But the two young entrepreneurs are preparing to take a shot at ALS on a budget that most Big Pharmas would spend on preclinical work. And they say their work could have applications for Alzheimer’s, another devastating disease that has so far burned billions of dollars in largely wasted research efforts.

ALS, or more formally Amyotrophic Lateral Sclerosis, has defied researchers for years now. The disease first cripples and then typically kills its victims in just a few years. Much of the work has been stymied by a fundamental lack of understanding of what causes the disease. But the two newly minted biotech entrepreneurs say that they’re taking two different drugs and will look to address two different manifestations of the disease in an attempt to disrupt the crippling cascade of events that afflicts patients.

“We thought, why don’t we look with what we know is correlating to patients’ clinical decline,” says Klee. “In Alzheimer’s you lose neurons in your memory center, the brain regions involved in memory. The same in ALS, you lose neurons in the motor cortex and spinal cord, and you lose movement ability. The other thing,” he adds, is that as neurons die, the brain immune system responds and kills more neurons, causing a toxic cycle that speeds the course of the disease.

Amylyx is focusing on the energy crisis in the mitochondria and the unfolded protein problem that causes a lot of the cell death, says Cohen.

The two have honed in on a new treatment, AMX0035, that combines two drugs, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA), aimed at tackling both those problems at once. They have some clinical data from earlier studies on both and enough backing now to test their idea that they can synergistically address ALS.

It’s a tall order concerning one of the Holy Grails of biotech R&D; something akin to first-timer climbers tackling Mt. Everest on a shoestring budget. But they say that the nonprofit groups, especially involving the hospitals like Mass General where the experienced investigators reside, have made it all possible with some key pricing breaks. And now, with some high profile connections like Termeer, the virtual biotech with a staff of three full timers is starting out to see if they can get close to a peak that has eluded so many before.

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