FDA accepts IND app for Epizyme’s mesothelioma drug

FDA sign

After a slew of failures in mesothelioma, there has been a small bright spot today after the FDA accepted an IND for Epizyme’s ($EPZM) experimental treatment for the hard-to-treat cancer.

The U.S. regulator has allowed tazemetostat’s Investigational New Drug (IND) application in patients with mesothelioma characterized by BAP1 loss-of-function. In the third quarter of this year, the company added that it also plans to start a Phase II study in patients with mesothelioma.

“We are moving quickly to expand the tazemetostat clinical program into mesothelioma, adding to our ongoing studies in non-Hodgkin lymphoma and certain genetically defined solid tumors,” said Robert Bazemore, president and CEO of Epizyme.

“We believe that tazemetostat has the potential to treat multiple types of cancer in patients who have limited treatment options. We look forward to starting the mesothelioma Phase II study later this year.”

Tazemetostat is a first-in-class small molecule inhibitor of EZH2 created by Cambridge, MA-based Epizyme using its drug development platform. Aberrant EZH2 activity results in misregulation of genes that control cell proliferation and has been associated with a diverse set of human cancers.

Emerging preclinical findings from published reports suggest that mesothelioma, and particularly mesothelioma characterized with BAP1 loss of function, may be sensitive to EZH2 inhibition, according to the company--with this affecting around half of the 12,000 new mesothelioma cases each year.

Mesothelioma is associated with exposure to asbestos and affects a number of organs, although typically its hits the lungs. Only around 10% of people diagnosed with this aggressive cancer are alive after 5 years.

A number of biotechs and Big Pharma have been looking to develop new drugs for this area, but with poor results. Earlier this year AstraZeneca ($AZN) reported that its CTLA-4 drug tremelimumab flunked a midstage test in mesothelioma, while Verastem ($VSTM) posted data last September that showed its lead drug defactinib (VS-6063) also failed to help patients with the disease.

In March, GlaxoSmithKline ($GSK) said it was cutting ties with Five Prime Therapeutics' ($FPRX) in-development cancer therapy and backed out in the middle of a mesothelioma trial. Five Prime did however say that it plans to work with GSK to complete enrollment in the study, adding that it "continues to be encouraged" by the drug's potential in this cancer.

Tazemetostat is also being studied in ongoing Phase II programs in both non-Hodgkin lymphoma, and certain genetically defined solid tumors--including INI1-negative and SMARCA4-negative tumors and synovial sarcoma.

The company has in addition announced plans to initiate additional clinical evaluations of tazemetostat in 2016--including a combination with R-CHOP in patients with diffuse large B-cell lymphoma and a combination with an immune checkpoint inhibitor in NHL.

Last year--3 years after an initial tie-up--Celgene ($CELG) remapped its partnership with Epizyme, extending their collaboration by 3 more years. Under this new deal, the biotech gained $10 million cash with up to $610 million in milestones for three histone methyltransferase (HMT) inhibitor targets.

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