After its bumpy road with a thrice-failed Duchenne drug, the team at PTC Therapeutics has been busy putting new irons in its fire. In its latest effort, the New Jersey company announced Thursday that it’s acquiring a gene therapy startup with a few promising drugs in its pipeline.
PTC is paying $50 million in cash and about $150 million in stock to buy up Agilis Biotherapeutics, a Cambridge, MA-based company that has a lead gene therapy candidate in late-stage trials. The drug is being developed to treat a rare CNS disorder called Aromatic L-Amino Acidd Decarboxylase, or ADDC. It’s already been tested in two prospective clinical studies, which showed it boosted dopamine production — a key goal for treating ADDC.
“Data from trials that began in 2010 have demonstrated long-term evidence of durable clinical benefit,” the company stated. “Based on multi-year data from initial clinical studies with the AADC gene therapy treatment candidate, PTC plans to submit a Biologics License Application (BLA) to the FDA in 2019.”
Agilis also has other gene therapy candidates, including a Friedreich ataxia program nearing IND, and an Angelman syndrome program.
“The addition of the gene therapy platform transforms PTC and aligns with our vision of being a leader in the treatment of rare disorders,” said Stuart Peltz, CEO of PTC, in a statement. “We look forward to advancing the Friedreich ataxia and Angelman syndrome programs into the clinic in the next two years.”
The acquisition is good news for PTC, a company that’s been plagued with bad news for years due to the failure (after failure after failure) of its Duchenne muscular dystrophy drug ataluren. That drug, approved in Europe, has so far failed to please regulators in the US. Although PTC still isn’t giving up on DMD, they’re certainly shoring up risk by putting more products in the pipeline.
And this isn’t the first time a Duchenne company veered into gene therapy land. Sarepta, the maker of the controversial Exondys 51 treatment for DMD, has made gene therapy a huge area of new focus for the company. I met with Sarepta’s CEO Doug Ingram at BIO this year, where he updated me on the company’s widespread efforts in the space. Sarepta now has 8 new gene therapy programs, he says, and just recruited Louise Rodino-Klapac, a former Nationwide Children’s Hospital exec, to lead its new gene therapy unit.
“There is no doubt that gene therapy — if it is successful — is a massive, massive opportunity,” Ingram said at the time. “I mean billions of dollars. The financial reason for that is that gene therapy is agnostic to mutation; it spans all mutations. In that sense, there’s a lot of big financial opportunity for gene therapy.”
PTC has clearly boarded that train. The Agilis acquisition deal includes up to $535 million in success-based milestones in connection with regulatory approvals on the three most advanced programs and receipt of a priority review voucher, as well as tiered commercial milestones of $150 million, and 2-6% of annual net sales for Friedreich ataxia and Angelman syndrome, the company said in a statement.
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