Phase 3 Luspatercept Trial

Thank you for sharing CrazyPharm,

I'm very happy to hear that.  Praying for the best.  

Thank you Sharin....its a great news..

Thanks Sharmin.

All of we are eagerly waiting for the drug..

Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Myelodysplastic Syndromes at the 21st Congress of the European Hematology Association

http://investor.acceleronpharma.com/releasedetail.cfm?ReleaseID=975231

June 10, 2016
Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Myelodysplastic Syndromes at the 21st Congress of the European Hematology Association
- Preliminary results show that treatment with investigational drug luspatercept results in clinically meaningful increases in hemoglobin and durable transfusion independence in patients with lower risk myelodysplastic syndromes -

- Acceleron to host conference call and live webcast today at 8:00 a.m. EDT (2:00 p.m. CEST) -

CAMBRIDGE, Mass. & SUMMIT, N.J.--(BUSINESS WIRE)-- Acceleron Pharma Inc. (NASDAQ:XLRN) and Celgene Corporation (NASDAQ: CELG), today announced preliminary results from an ongoing long-term Phase 2 extension study with luspatercept in patients with lower risk myelodysplastic syndromes (MDS) at the 21st Congress of the European Hematology Association (EHA) in Copenhagen, Denmark. Results highlighted in an oral presentation showed that 51% of patients with lower risk MDS treated with luspatercept (n=49) achieved increased hemoglobin levels and 35% of patients achieved transfusion independence in the 3-month base study. In the ongoing extension study, 81% (26/32) of patients had increased hemoglobin levels and of the patients eligible for transfusion independence (TI), 50% achieved TI with luspatercept treatment. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

"The results for luspatercept in lower risk MDS patients are increasingly encouraging as we gain longer term safety and efficacy experience with this agent," said Uwe Platzbecker, M.D., Professor of Hematology and Head of the MDS program at the University Hospital in Dresden, Germany. "There is a significant unmet need for new therapies that reduce the number of or eliminate the need for blood transfusions."

Highlights of the Luspatercept MDS Phase 2 Data Presented at EHA

Study Design

Data from two Phase 2 studies were presented at the conference: the completed dose-escalation study in which patients received treatment with luspatercept for three months and the ongoing long-term extension study in which patients receive treatment with luspatercept for an additional 24 months. In both the 3-month base study and the long-term extension study, high transfusion burden patients (≥ 4 units RBC / 8 weeks) and low transfusion burden patients ( < 4 units RBC / 8 weeks) were enrolled and treated with open-label luspatercept, dosed subcutaneously once every 3 weeks. The primary outcome measure for the 3 month study was the proportion of patients who had an erythroid response. Erythroid response was defined as hemoglobin ≥ 1.5 g/dL from baseline for ≥ 14 days in non-transfusion dependent patients or a reduction of either ≥ 4 units or ≥ 50% of units of RBCs transfused compared to pretreatment in transfusion-dependent patients. The primary outcome for the long-term extension study is to evaluate the long-term safety and tolerability of luspatercept with low or intermediate-1 risk MDS who were previously enrolled in the 3-month study.

Efficacy

              
        Response rate (% of patients)   
    
3-month base study
(n=49, higher dose
levels)
    
Long-term extension
study
(n=32)
International Working Group Hematologic
Improvement-Erythroid (IWG HI-E)
Response Rate (reduction of ≥4 units RBC / 8
weeks or a hemoglobin increase ≥1.5 g/dL ≥ 8
weeks)
    51% (25/49)       81% (26/32)   
RBC Transfusion Independence (RBC-TI)
Response Rate (Transfusion free ≥ 8 weeks for
patients with ≥ 2 units RBC / 8 weeks prior to
treatment)
    35% (14/40)       50% (11/22)   
Duration of RBC-TI               Range: 9-80+ weeks   
       
For reference, results presented six months ago at the American Society of Hematology (ASH) annual meeting in December 2015 were as follows:

IWG HI-E response rate was 69% (22/32)
RBC-TI response rate was 50% (11/22)
Duration of RBC-TI ranged from 9 to 50+ weeks
Safety

There were three grade 3 adverse events possibly/probably related to study drug (blast cell count increase, myalgia and worsening of general condition).
Adverse events at least possibly related to study drug that occurred in at least 2 patients during studies were fatigue, bone pain, diarrhea, myalgia, headache, hypertension and injection site erythema.
Luspatercept is an investigational product that is not approved for use in any country.

The MEDALIST Trial, a global Phase 3 study in patients with very low, low, or intermediate risk, MDS with ring sideroblasts who require red blood cell transfusions, is currently enrolling.

The slides from this oral presentation are available on Acceleron's website (www.acceleronpharma.com) under the Science tab.

The Phase 3 trials are currently recruiting. More info can be found at https://clinicaltrials.gov/ct2/show/NCT02604433?term=luspatercept&rank=1

Friends
If everything comes out good, will it open doors for kids/infants? Or it's limited to adult only?

Is it really going to take as long as the 2020's or something until Luspatercept will be available? That's a damn long time.

The phase 3 trials will answer a lot of questions. I think it will be available to children once approved.

Acceleron and Celgene Announce Updated Results from Phase 2 Studies of Luspatercept in Beta-Thalassemia Presented at the 58th Annual Meeting of the American Society of Hematology

"Beta-thalassemia is a severe, chronic disease with no pharmaceutical treatment options to correct or improve the underlying anemia in patients," said Michael Pehl, President, Hematology and Oncology for Celgene. "These longer term luspatercept Phase 2 data are encouraging, and we are continuing to enroll patients in the Phase 3 BELIEVE study in transfusion dependent beta-thalassemia patients."

Luspatercept Beta-Thalassemia Data Presented at ASH

Results in Transfusion Dependent (TD) Beta-Thalassemia Patients

    
RBC transfusion reduction
over any 12 weeks versus 12
weeks pre-treatment
        Response rate (% of patients)
            3-month base study
(n=31)
        Long-term extension study
(n=24)
    ≥ 20%           81% (25/31)           96% (23/24)
    ≥ 33%           71% (22/31)           83% (20/24)
    ≥ 50%           55% (17/31)           71% (17/24)
               
Results in Non-Transfusion Dependent (NTD) Beta-Thalassemia Patients

    
Hemoglobin (Hb) response over
any 12 weeks versus 12 weeks
pre-treatment
        Response rate (% of patients)
in patients treated with ≥ 0.6 mg/kg
            3-month base study
(n=21)
        Long-term extension study
(n=27)
    Increase in mean Hb ≥ 1.0 g/dL           62% (14/21)           78% (21/27)
    Increase in mean Hb ≥ 1.5 g/dL           33% (7/21)           52% (14/27)
               
In the long-term extension study, the median duration of a hemoglobin increase ≥ 1.0 g/dL maintained for at least 12 weeks in responders is 13.5 months (N=21) with treatment still ongoing.

for how long phase 3 wi
ll continue. Andy Sir what will u suggest the tentative time for its approval.

Trials will be taking place in many different areas over a period of time, as patients are recruited. My educated guess is that we will see this drug on the market within 5 years.

I have tried to enroll into the trials but was rejected the day it opened in my city because quota is already full. unbelievable... .. i've been watching this drug ready to jump on the trial since its early phase 2 days. bummer i cant be a part i hope for a fast approval

Check the recent post by Parin.

bluebird bio is also moving forward with plans to initiate Northstar-3 (HGB-212), a Phase 3 trial of LentiGlobin drug product in patients with transfusion-dependent β-thalassemia with the β0/β0 genotype. This study will also be conducted under the new manufacturing process, and is expected to begin enrolling patients in 2017. The primary endpoint of this planned study is transfusion reduction.

Thank you Andy for valuable information.

Short question that came up: why are they never releasing detailed blood test results? Do they only mention the 'transfusion reduction' because that is also the current goal for their studies? I think it would be very helpful to see which effects the drug has on many other blood values and therefore on the body as a whole.