After scoring big Pfizer buyout, a Therachon co-founder turns once again to dwarfism with $24M Series A
Less than two years after selling her first biotech to Pfizer for a hefty $810 million, a French researcher has launched a new gene therapy outfit that’s focusing on the same target — dwarfism — but taking a different path to treatment.
The scientist is Elvire Gouze and the company is InnoSkel, which launched with €20 million in Series A financing (about $24.2 million) early Monday. Though InnoSkel is aiming to treat a whole host of rare skeletal diseases that affect the body’s collagen, it’s first going after what’s known as spondyloepiphyseal dysplasia congenita (SEDc), the second-leading cause of dwarfism that stunts the growth of a patient’s cartilage.
Monday’s fundraise was co-led by Jeito Capital and Arie Belldegrun’s team at Vida Ventures. Région Sud Investissement and The Turenne Group also participated in the round.
Gouze had been working on skeletal dysplasia at Inserm, the French equivalent of the NIH, and helped launch the newly Pfizer-owned Therachon. She kept her academic lab while working there and came up with the idea for a gene therapy in type 2 collagenopathies, which includes SEDc.
“That’s how we started InnoSkel, it’s a spin-out of my Inserm lab,” Gouze told Endpoints News.
There are two main causes of dwarfism, Gouze said, the first of which was a leading target for Therachon. While the biotech’s research centers also revolve around rare musculoskeletal disorders, Therachon’s mission was to treat the complications of achondroplasia like those in the body’s cardiovascular, neurological and metabolic systems.
Achondroplasia affects bone growth and arises from a separate genetic mutation, resulting in a completely different mechanism but similar characteristics of dwarfism as SEDc. Therachon’s experimental drug is an FGFR3 decoy, which they believe will correct a signaling pathway that triggers the abnormal growth.
Though both conditions result in individuals with dwarfism having the same physical appearance, Gouze says the complications in this cartilage-based disorder are quite different. Rather than their bones growing abnormally, patients with SEDc see effects in their growth plates — the cartilage “template” of bones in children that haven’t formed yet — essentially necessitating treatment before puberty.
This can lead to retinal detachments and patients having trouble breathing, Gouze said. Further difficulties holding up their heads and necks can result in paralysis.
“In this case, the patients are very short and have severe complications,” Gouze said. “What we’re trying to achieve is to repair the exocyclic matrix of the cartilage.”
InnoSkel isn’t ready to divulge how their program treats the condition, nor can Gouze predict when they’ll first hit the clinic as the company is still awaiting the publication of a peer-reviewed paper. Monday’s funding will go toward advancing to human trials, however, and any product will look like a typical gene-therapy injection to provide proteins that help the cartilage behave normally, she said.
Trying to treat dwarfism is a cause that’s drawn significant opposition from some corners of the community. It’s one Gouze is all too familiar with given her previous role as scientific founder of Therachon, and now she’s back for more with InnoSkel. There have also been efforts by a prominent pharma, BioMarin, to get an achondroplasia drug across the finish line. The FDA accepted BioMarin’s pitch for the program just last month, with an action date coming next August.
But Gouze says she “fully respects” the argument from advocates that dwarfism is not something meant to be “cured.” Making everyone taller is not Gouze’s main goal, she says, rather it’s trying to prevent the complications that result from achondroplasia and now SEDc.
“When you’re an adult with dwarfism you have to live with your condition anyway, and it’s not to make them like everybody; the treatments are not trying to ‘standardize’ them,” Gouze said. “It’s not so much to make everybody look the same, which would be useless and absolutely not interesting anyway. It’s trying to prevent the complications and decrease the pain.”