Pharma Giant AstraZeneca PLC Scores Another FDA Breakthrough

August 1, 2017
By Alex Keown, BioSpace.com Breaking News Staff

WASHINGTON – AstraZeneca is on a roll. This morning the company announced it won its second Breakthrough Therapy Designation from the U.S. Food and Drug Administration this week. This time the company won the designation for its blood cancer drug acalabrutinib.

Acalabrutinib is a Bruton tyrosine kinase (BTK) inhibitor in development for the treatment of multiple B-cell cancers in patients who have received at least one prior therapy. It’s the second AstraZeneca drug this week to win the breakthrough designation. On Monday the company announced its lung cancer drug Imfinzi earned the breakthrough status. The FDA’s Breakthrough Therapy Designation will expedite the regulatory review of the two drugs.

Acalabrutinib won the designation based on data from the Phase II ACE-LY-004 clinical trial in patients with relapsed or refractory MCL. In addition to MCL, acalabrutinib is being investigated as a monotherapy and in combination with other treatments for several blood cancers as well as solid tumors. In addition to MCL, acalabrutinib is also being investigated as a therapy for chronic lymphocytic leukemia. In February, AstraZeneca announced positive preliminary results from a Phase I/II trial in using acalabrutinib to treat CLL.

Mantle cell lymphoma is an aggressive form of B-cell non-Hodgkin lymphoma. It accounts for approximately 3 percent to 6 percent of new NHL cases annually. It has a poor prognosis for patients.

Flavia Borellini, chief executive officer of Acerta Pharma, a subsidiary of AstraZeneca focused on hematological treatments, called the designation an “exciting regulatory milestone for our work in hematology.” Borellini said acalabrutinib has a high degree of specificity for its target.

“If approved, it could be a clinically-meaningful treatment option for patients with this devastating disease,” Borellini said in a statement.

Sean Bohen, AstraZeneca’s chief medical officer, agreed that new treatments are needed for people with mantle cell lymphoma. He said acalabrutinib is a treatment that can be beneficial for patients who have relapsed or have not responded to other therapies.

Snagging two Breakthrough Therapy Designations from the FDA is a welcome shot in the arm for AstraZeneca following the failure of its critical Phase III Mystic trial. In that trial Imfinzi, a PD-L1 inhibitor, was being tested in combination with tremelimumab, a CTLA-4 inhibitor, for the treatment of patients with lung cancer whose tumors express PD-L1 on 25 percent or more of their cancer cells. The failure caused shares of AstraZeneca to plunge, resulting in an approximately $10 billion loss in market value. Shares of AstraZeneca are down slightly this morning, trading at $29.95 as of 11:04 a.m.