Amgen makes regenerative medicine play, backing $25M Fortuna round to move neural stem cells into the clinic

Amgen’s VC wing has invested in Fortuna Fix to help move autologous neural stem cells into the clinic. The $25 million series B sets regenerative medicine startup Fortuna up to move candidates against Parkinson’s disease and spinal cord injury to clinical trials next year.

Fortuna’s pipeline is built on the concept of direct cell reprogramming. Rather than start with induced pluripotent stem cells and tackle the challenge of converting them into neural cells, Fortuna’s process begins with fully differentiated cells taken from the patient. Fortuna then forces the expression of genes associated with being a neural stem cell before locking the cell, known as a drNPC, into this new position.

“There's a whole chromatin remodeling that takes place. And that chromatin remodeling, the epigenetic part of it, is done chemically. There's no genetic manipulation, no viral vectors, no animal components, no human embryos, no fetuses in the process,” Masha Le Gris, VP of corporate development at Fortuna, said.

The process uses a patient’s own cells. Initially, these will be taken from bone marrow but Fortuna thinks eventually it could use any harvestable cell, even those from the hair. The potential benefit of the approach is Fortuna should be able to administer its cells without immunosuppression.

Fortuna, under its previous name of Ophiuchus, has already tested its autologous neural stem cells in a clinical trial in Russia. Full details of the outcomes of the 30-patient, investigator-led spinal cord injury clinical trial are being kept under wraps pending their formal publication. But the fact spinal cord injury is one of two indications in which Fortuna is moving forward tells the broad story. 

“We're moving forward with our clinical plans and therefore things were positive, the outcome was positive,” Le Gris said.

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The broad approach to the generation of drNPCs is the same regardless of the targeted indication. But Fortuna is tweaking the details to create cells tailored to the biology of each condition. 

“What we have done [in spinal cord injury] is actually push the drNPCs a little bit down the linage of making more oligodendrocytes and neurons proportionally to the astrocytes. We are going with a product which is still a neural precursor cell, a neural stem cell, but will be a little bit differentiated in that lineage so that we get optimal myelination, remyelination and grafting,” Le Gris said. 

For Parkinson’s, Fortuna is pushing the starting material toward the state of A9 dopaminergic precursor cells. Researchers have wanted to use dopaminergic cells to offset cell death in Parkinson’s patients for years and multiple groups are working toward the goal. 

The advance of these groups toward the clinic has taken place against a backdrop of discussion about when such cells are ready for human testing and which patients should be treated. Fortuna is still figuring out which patients it will target in its trial.

“It's an ongoing dialogue between the key opinion leaders and of course we are discussing with the FDA,” Le Gris said.

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Backed by Amgen Ventures and other investors, Fortuna can now gear up for the end of those regulatory discussions and start of its clinical trials. The healthy bank balance is a new experience for Fortuna CEO Jan-Eric Ahlfors, who has toiled for more than a decade on some of the technology in development at the company.  

Ahlfors’ efforts began with Total ReCord, a biotech that spun out of his research think tank New World Laboratories (NWL). Total ReCord talked up its prospects of moving a “new class of blockbuster therapeutics” into the clinic in 2005 but five years later Ahlfors folded the technology back into NWL.

In 2015, some of the technology reemerged as part of Ophiuchus. Under that moniker, Ahlfors got the cell therapy into the Russian clinical trial but funding remained a challenge. At one stage, Ahlfors sought donations through his non-profit Novagenesis Foundation—which owned 100% of the shares in Ophiuchus—as an alternative to venture capital. But ultimately a lack of cash held things back.     

“It was the lack of manpower and resources which have really stalled the CEO and the company,” Le Gris said. 

In toiling to reach this point, Fortuna laid the groundwork for its future by investing in automated manufacturing. The result is a system designed to eliminate some of the complexity—and associated costs—typically tied to the production of autologous cell therapies.  

“You take a sample of cells from a patient, you put them into this robot and then six weeks later you have 100 million neural stem cells from that patient,” Le Gris said.

Fortuna has capacity for up to 10,000 cell lines a year at a GMP plant in Montreal, Canada, Le Gris said, and can scale up by building more robots when needed. If the robots work as well as claimed, that puts Fortuna on a strong footing with regard to one of the challenges associated with cell therapies.

The other, more critical problem faced by many cell therapies is a lack of efficacy. StemCells canned its spinal cord injury program and shut down last year after coming up short in phase 2. 

Precedent suggests a similar fate may await Fortuna. But having persuaded a big biotech, or at least its VC wing, to make a rare move into neurodegenerative cell therapies, Fortuna now finally has the resources to start finding out either way.