BerGenBio Reports First-in-Patient Phase 1 Data for BGB324 in Patients with Myeloid Malignancies at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting

Promising clinical activity with biologic activity, tolerability, and durability

Jun 07, 2016, 02:00 ET from BerGenBio

BERGEN, Norway, June 7, 2016 /PRNewswire/ --

BerGenBio AS ("BerGenBio" or the "Company"), a clinical stage biotechnology company focused on the development of novel cancer therapeutics, announced that clinical data from its lead product candidate BGB324, a first-in-class selective Axl kinase inhibitor, was presented at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago. The data demonstrated the clinical activity, tolerability and unique mechanism-of-action of BGB324 in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

"We are very encouraged by the promising clinical activity and prolonged tolerability of BGB324 as a single agent in elderly patients with AML and high-risk MDS," said Richard Godfrey, Chief Executive Officer of BerGenBio. "We look forward to progressing into Phase 2 trials as the data indicate that BGB324 could be a potential future treatment option for patients suffering from AML and MDS."

Dr. Sonja Loges, attending oncologist at the University Medical Center Hamburg-Eppendorf, presented a poster entitled: "A first-in-patient phase I study of BGB324, a selective Axl kinase inhibitor in patients with refractory/relapsed AML and high-risk MDS" on Sunday June 5, 2016, as part of the session on Developmental Therapeutics - Clinical Pharmacology and Experimental Therapeutics.

The presented data demonstrate that BGB324 can be safely administered for prolonged periods at doses that inhibit Axl activation and exhibit anti-leukemic activity, and therefore could be a potential treatment option for patients with AML and MDS.

A dose escalation study was performed to identify the recommended Phase 2 dose of BGB324 in patients with previously treated AML or high risk MDS. Three daily maintenance dose levels (100mg, 200mg and 300mg) were explored. Twenty patients with AML and four patients with MDS were treated; seven patients remain on treatment.

The data demonstrated encouraging signals of anti-leukemic activity. One AML patient reported complete remission, with incomplete blood count recovery at five months; another patient reported partial remission demonstrated by clearance of circulating leukemic blasts accompanied by peripheral blood count recovery for more than three months. Two of the four MDS patients showed objective responses. A further six patients in the study showed disease stabilisation for more than four months. Complete inhibition of Axl activation and signal transduction was demonstrated in leukaemia cells from patient bone marrow samples, validating the unique mechanism-of-action of BGB324.

Reversible mild or moderate gastrointestinal disorders were the most common observed adverse events. These occurred mostly during the loading dose period and resolved during chronic dosing.  

"It is very encouraging that BGB324 was well tolerated and showed clinical activity as monotherapy in this elderly population with many comorbidities and no further treatment options. In addition, we could demonstrate that BGB324 treatment at these doses completely inhibits the target Axl," commented Dr. Loges. "I am looking forward to further advance the monotherapy cohort and combine BGB324 with chemotherapy. BGB324 could become a new treatment option in AML and MDS."

The full abstract can be accessed through the ASCO website at:

About BerGenBio AS 

BerGenBio AS is a clinical stage biopharmaceutical company focused on developing first-in-class drugs for aggressive cancers. The company is a world leader in understanding the biology of epithelial-mesenchymal transition (EMT), a widely recognised key pathway in immune evasion, acquired cancer drug-resistance and metastasis. BerGenBio AS is founded on proprietary platform technology, CellSelect™, to identify and validate novel drug targets and biomarkers. The company has progressed its lead drug candidate BGB324, a potent highly selective orally bioavailable Axl inhibitor: Axl is an essential mediator of EMT, into clinical trials in AML and NSCLC. And is preparing for multiple phase 2 trials as a single agent and in combination with standard of care cancer drugs in several diffident cancer indications. The Company is also pursuing the pre-clinical development of additional proprietary EMT inhibitors.

About BGB324 

BGB324 is a first-in-class, highly selective small molecule inhibitor of the Axl receptor tyrosine kinase. It blocks the epithelial-mesenchymal transition (EMT), which is a key driver in immune evasion, drug-resistance and metastasis.