on its deal, that drug—as well as two other drug candidates involved in the collaboration, a pre-clinical one known as ACY-738 targeting neurological diseases, and another that hasn’t been named—must all make it through clinical studies, win approval from regulators, and begin earning cash.
The $100 million will fund Acetylon’s operations “across the board,” according to Ogier, but largely will help propel ACY-1215 through clinical development. None of it will be paid out to the company’s shareholders, he says.
Acetylon was formed in 2008 out of work at Harvard University and the Dana-Farber Cancer Institute looking into drug candidates that target one of a class of enzymes known as histone deacetylases, or HDACs, which help regulate gene expression and are implicated in a number of cancers. HDACs are a well-known molecular target: Merck’s lymphoma drug vorinostat (Zolinza) is an HDAC inhibitor, and Celgene itself agreed to pay as much as $640 million in 2009 for Gloucester Pharmaceuticals because of its HDAC inhibitor for both cutaneous and peripheral T-Cell lymphoma, romidepsin (Istodax). That drug generated about $50 million in sales in 2012.
Earlier versions of HDAC inhibitors, however, attacked several HDAC enzymes at once—Acetylon’s drugs are homing in on those enzymes more precisely. ACY-1215 only blocks a specific enzyme known as HDAC6; ACY-738 targets both HDAC1 and 2. This type of specificity is important, because it is meant to keep Acetylon’s drug from messing with healthy cells and producing nasty side effects like nausea, vomiting, and diarrhea, and even severe ones like brain hemorrhages.
Celgene was attracted to Acetylon because of ACY-1215’s ability to potentially provide the cancer-fighting punch of an HDAC inhibitor without those toxicity issues. It jumped in with a $15 million investment in February 2012 and took an advisory role, nabbing a seat at the company’s board as a non-voting observer. Their relationship evolved as Acetylon began testing ACY-1215 in an early-stage clinical trial in combination with Celgene’s blockbuster lenalidomide (Revlimid), in patients with relapsed/treatment-resistant multiple myeloma. Acetylon is also testing the drug in combination with Takeda’s myeloma drug bortezomib (Velcade).
“That trial’s gone well, it’s been a collaborative, comfortable process for both companies,” Ogier says. “So that definitely helped open the doors to discussions.”
In June, Acetylon reported that none of the patients in the lenalidomide trial reported any serious adverse events, though it said one case of neutropenia—a depletion of infection-fighting white blood cells—was “possibly” related to the drug. Acetylon knows it will have to produce these types of safety results on a much larger scale to reach its potential, and ultimately saddle up to Celgene.
Acetylon’s plan is to position ACY-1215 as a combination therapy with either lenalidomide or bortezomib in multiple myeloma. It also plans to test it out as either a monotherapy or part of a combination regimen in non-Hodgkin’s lymphoma in patients who have failed prior therapy, according to Ogier.
As Xconomy readers well know, meanwhile, the deal fits right in with Celgene’s flexible style of early-stage business development, through which it tailors specific deal structures for specific companies based on their development needs. Celgene, for instance, already has nailed down a few other option-to-acquire transactions over the past few years, among them deals to buy South San Francisco, CA-based Quanticel Pharmaceuticals and Seattle-based VentiRx Pharmaceuticals.
