Aeterna Zentaris: Phase 2 Data Demonstrate Perifosine's Promising Efficacy in the Treatment of Advanced Leukemia, Lymphoma and Multiple Myeloma

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BlankAeterna Zentaris: Phase 2 Data Demonstrate Perifosine's Promising Efficacy

in the Treatment of Advanced Leukemia, Lymphoma and Multiple Myeloma

In two Phase 2 studies, perifosine achieves objective responses and high rates

of stable disease as a single-agent in the treatment of Chronic Lymphocytic

Leukemia (CLL), and in combination with sorafenib in the treatment of

relapsed/refractory Hodgkin's Lymphoma (HL)

QUÉBEC CITY, Dec. 6 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS,TSX: AEZ)

today announced Phase 2 data presented for the first time on perifosine, its

lead novel oral anti-cancer compound, showing promising clinical activity,

safety and tolerability in patients with advanced chronic lymphocytic leukemia

(CLL) and Hodgkin's lymphoma (HL). The data were presented over the weekend at

the 52nd Annual Meeting of the American Society of Hematology (ASH) in Orlando,

Florida.

Juergen Engel, Ph.D., President and CEO of Aeterna Zentaris stated, " The

encouraging data in advanced leukemia and Hodgkin's lymphoma presented at the

ASH meeting, confirm perifosine's efficacy and safety as a novel, oral

anti-cancer agent not only in combination therapy with approved anti-cancer

agents but also as a single agent therapeutic. Data also showcase perifosine's

potential beyond the current Phase 3 registration trials in metastatic

colorectal cancer and multiple myeloma. "

Key highlights from the two Phase 2 poster presentations are as follows:

Abstract # 2861: " Clinical Activity and Safety of the Combined Therapy with the

AKT Inhibitor Perifosine and the Multikinase Inhibitor Sorafenib In Heavily

Pretreated Patients with Relapsed/Refractory Lymphomas: Preliminary Results of a

Phase II Trial "

Study Background

In this Phase 2 study, 26 patients were enrolled with advanced lymphoma (6 NHL,

4 CLL, 1 Waldenstrom's Macroglobulinemia and 15 Hodgkin's lymphoma). 73% of

patients were previously refractory to their prior therapy, with 85% of patients

having had 4 or more prior therapies. Perifosine (50 mg BID) was started as a

single agent for 28 days; after 28 days, patients achieving partial response

(PR) or better were continued on single agent perifosine. Patients achieving

less than a PR were given the combination of perifosine (50 mg BID) plus

sorafenib (Nexavar®) at 400 mg BID.

Results

All of the 4 CLL patients in the study achieved a partial response on

single-agent perifosine within one month of treatment and remained on perifosine

single agent. Response durations for each of the 4 patients were 4, 8, 9+ and

12 months. The remaining 22 patients were administered the combination with

sorafenib, where 5 of the 15 (33%) Hodgkin's lymphoma patients achieved a

partial response with a median response duration of 9 months. An additional 6

patients receiving the combination (40%) achieved stable disease. The

combination was well tolerated with no unexpected safety events.

The investigators concluded that perifosine in combination with sorafenib has

significant anti-lymphoma activity in relapsed/refractory HL, and that

perifosine as a single agent induced prolonged responses in high-risk, heavily

pretreated CLL patients.

Abstract # 1842: " Pre-Clinical and Interim Results of a Phase II Trial of

Perifosine In Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia

(CLL) "

Study Background:

In this Phase 2 study, 12 patients with advanced CLL began treatment with single

agent perifosine at 50 mg BID. Patients on study were heavily pre-treated having

had a median of 4 prior lines of therapy with 75% of patients classified as Rai

stage IV.

Results

1 patient achieved a partial response (5 months on treatment) and 5 additional

patients achieved stable disease (median duration of 4.25 months), for an

overall 50% clinical benefit rate (PR + SD). Perifosine was well tolerated with

minimal dose modifications.

Abstract # 3064: " Final Phase I Results of Perifosine In Combination with

Lenalidomide and Dexamethasone In Patients with Relapsed or Refractory Multiple

Myeloma (MM) "

Additionally, the final data set from the Phase 1 study of perifosine +

lenalidomide (Revlimid®) + dexamethasone were also presented during the ASH

meeting. The final data showed a 73% objective response rate (minimal response

or better) with a 50% PR or better, a median Progression-Free Survival of 10.8

months, and a median duration for Overall Survival of 30.6 months. The myeloma

investigators concluded that perifosine in combination with lenalidomide +

dexamethasone was well tolerated even at the highest doses used, and

demonstrated encouraging clinical activity and survival.

A copy of the above referenced abstracts can be viewed online through the ASH

website at www.hematology.org

About Perifosine

Perifosine is a novel, oral anticancer treatment that inhibits Akt activation in

the phosphoinositide 3-kinase (PI3K) pathway. The product works by interfering

with membranes of cancer cells thereby inhibiting Akt signaling which then

affects cell death, growth, differentiation and survival. Perifosine, in

combination with chemotherapeutic agents, is currently being studied for the

treatment of multiple myeloma, colorectal cancer and other cancers, and is the

most advanced anticancer compound of its class in late-stage development.

Perifosine as monotherapy, is being explored in other indications. The US Food &

Drug Administration ( " FDA " ) has granted perifosine orphan-drug designation in

multiple myeloma and neuroblastoma, and Fast Track designations in both multiple

myeloma and refractory advanced colorectal cancer. Additionally, an agreement

was reached with the FDA to conduct the Phase 3 trials in both of these

indications under a Special Protocol Assessment. Perifosine has also been

granted orphan medicinal product designation from the European Medicines Agency

( " EMA " ) in multiple myeloma. Furthermore, perifosine has received positive

Scientific Advice from the EMA for both the multiple myeloma and advanced

colorectal cancer programs, with ongoing Phase 3 trials for these indications

expected to be sufficient for registration in Europe. Perifosine rights have

been licensed to Keryx Biopharmaceuticals for North America and to Handok for

Korea.