Fresh off finally winning an FDA nod for its first product, Amicus Therapeutics is getting in on the gene therapy renaissance.
This morning, Cranbury, NJ-based Amicus (NASDAQ: FOLD) agreed to pay $100 million up front to buy a privately held company, Celenex, spun out of Nationwide Children’s Hospital in Ohio. The acquisition gives Amicus a portfolio of 10 experimental gene therapies—treatments that may offer a long-lasting, if not permanent, effect through a single infusion—originally discovered at Nationwide and The Ohio State University.
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These gene therapies are all for lysosomal storage disorders (LSDs), Amicus’s focus. LSDs are caused by mutations in the genetic instructions for important enzymes that normally help our cells clear out waste like extra fats and sugar. The most advanced Celenex gene therapies are in early-stage clinical trials aiming to treat two genetic subsets of patients with Batten disease, a rare and fatal inherited disorder. Additional human data on one of the treatments—for Batten patients with a mutation to the CLN6 gene—should be presented next year.
Other programs for disorders including Niemann-Pick disease type C, Wolman Disease, and Tay-Sachs haven’t been tested in humans yet.
Amicus could pay another $277 million in downstream payments if all of these programs successfully advance. Celenex shareholders could also get up to $75 million in payouts if the gene therapies hit certain sales targets. Amicus financed the deal with a $150 million credit line.
Amicus shares ticked up about 6 percent in pre-market trading. The company will hold a conference call this morning to discuss the acquisition.
The move makes Amicus the latest to place a bet on gene therapy, a field of research that has finally come of age after years of ups and downs. In 2017, the FDA approved three treatments that modify a patient’s genes to treat disease—two for blood cancers, and one for a rare form of inherited blindness. Others are making clinical progress in blood diseases like hemophilia and beta thalassemia, rare disorders such as Duchenne muscular dystrophy, spinal muscular atrophy (SMA), and more. Two gene therapies were approved earlier in Europe. These developments are the culmination of decades of scientific research on how to effectively and safely deliver genes to cells.
Nationwide has been at the center of a few of the programs progressing through clinical testing. One program, licensed to Sarepta Therapeutics (NASDAQ: SRPT), has shown early promise in its first human test. Another tested at Nationwide and developed by Chicago-based AveXis, for SMA, showed such encouraging results in clinical testing that Novartis bought the company for $8.7 billion in April.
Amicus noted that the Celenex gene therapies use a similar approach to the SMA gene therapy. A modified adeno-associated virus—a workhorse gene therapy delivery tool—is loaded up with genetic instructions and infused into a patient’s spinal fluid. Brian Kaspar, the chief scientific officer and scientific founder of AveXis, co-founded Celenex with Nationwide gene therapy expert Kathrin Meyer. Both will be scientific advisors to Amicus.
Amicus cut the deal just a month after winning FDA approval of migalastat (Galafold), for Fabry disease—the company’s first marketed drug. A second drug, for Pompe disease, is in human testing. Here’s more on Amicus and its ups and downs over the years.