July 17, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Thousand Oaks, Calif.-based Amgen announced yesterday the topline results of its Phase II clinical trial of Blincyto (blinatumomab) in adults with relapsed or refractory Philadelphia chromosome-positive (Ph+) B-cell precursor acute lymphoblastic leukemia (ALL).
The study showed the drug included complete remission or complete remission with partial hematological recovery within two treatment cycles in a clinically meaningful number of patients. Results were consistent with the drug’s safety profile.
“These top-line results are encouraging and support blinatumomab as a potential treatment option for patients with relapsed or refractory Philadelphia chromosome-positive B-cell precursor ALL,” said Sean Harper, executive vice president of Research and Development at Amgen in a statement. “We are hopeful that our comprehensive ALL development program for blinatumomab, the first clinical and regulatory validation of the BiTE platform, will continue to demonstrate clinical effectiveness for patients with this serious disease.”
About 25 percent of adult patients with ALL have what is known as the Philadelphia chromosome, in which a piece of chromosome 9 changes places with a piece of chromosome 22. The small remainder of chromosome 22 is known as the Philadelphia chromosome because it was the city where the translocation was first observed.
The chromosome breakage occurs in two genes, BCR and ABL1, and results in a fusion gene, BCR-ABL. The fused gene codes for a protein that is continuously activated and results in unregulated cell division. The chromosome abnormality is most commonly associated with chronic myelogenous leukemia (CML), but is observed in ALL and sometimes in acute myelogenous leukemia (AML).
Blincyto is an antibody construct product that is bispecific CD19-directed CD3 T cell engager (BiTE). The drug was granted breakthrough therapy and priority review designations by the U.S. Food and Drug Administration (FDA). It is a type of onco-immunology therapy, which uses part of the patient’s own immune system to attack cancer cells.
On Dec. 3, 2014, the FDA approved Blincyto to treat patients with Philadelphia chromosome-negative (Ph-) precursor B-cell acute lymphoblastic leukemia (B-cell ALL), a rare form of ALL.
Blincyto is an innovative, breakthrough therapy for an ultra-orphan that affects approximately 900 patients with Philadelphia chromosome-negative relapsed/refractory ALL in the US. These patients have a median overall survival of just three to five months and BLINCYTO is the first major treatment advance for these patients in more than two decades. In the study single-agent study leading to approval, 41.6 percent of patients achieved complete remission or complete remission with partial hematologic recovery (CR/CRh) within two cycles of treatment with BLINCYTO. Among patients who achieved CR/CRh, 39 percent went on to hematopoietic stem cell transplantation, the only potential cure for these patients.
The drug carries a so-called “boxed” warning because there have been cases of low blood pressure and difficulty breathing, also known as cytokine release syndrome. When this occurs, it’s typically at the beginning of the first treatment, and may also include difficulty thinking (encephalopathy) or other nervous system side effects.
Blincyto is priced at $178,000 for two courses of treatment.
In a statement to Medical Daily in December 2014, Amgen said, “We believe the price reflects the significant clinical, economic and humanistic value of the product to patients and the healthcare system, for an ultra-orphan population with a dramatic impact on a serious illness.”
Amgen picked up Bincyto when it acquired Micromet, a German biotech company, in 2012 for $1.2 billion.
As New Jersey Biotech Booms, Will It Overtake Other States As Prime Location?
A week after Celgene Corporation announced it is officially the mystery buyer of Merck & Co. ’s former 1 million-square-foot R&D site in Summit, N.J., it quickly became our most popular story last week.
The company announced last Wednesday that it is buying the space, ending months of speculation about what Big Pharma company might move into the neighborhood.
The Summit, N.J. site is zoned research/office. The New Jersey site would put operations closer to some of the major biotech and pharmaceutical hubs on the East Coast.
But, by far, the most tempting part of doing business in the state remains New Jersey’s operating tax credit, which allows companies to sell their net operating losses to the New Jersey Treasury. One of the state’s most recognizable biotechs, Celgene, used the program until it became profitable, which was key to it staying in the state, said local officials.
That has BioSpace is wondering if New Jersey is becoming the new face of biotech. What do you think? Can the Garden State compete with other longtime stalwarts like California or Boston?
