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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

On June 10, 2016 Acceleron Pharma Inc. (NASDAQ:XLRN) and Celgene Corporation (NASDAQ: CELG), reported 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) (Free EHA Whitepaper) in Copenhagen, Denmark (Press release, Acceleron Pharma, JUN 10, 2016, View Source [SID:1234513176]).

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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 (Free EHA Whitepaper)

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) (Free ASH Whitepaper) 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.

Acceleron EHA (Free EHA Whitepaper) Conference Call Information

Acceleron will host a conference call and live webcast from EHA (Free EHA Whitepaper) today at 8:00 a.m. EDT (2:00 p.m. CEST). To participate by teleconference, please dial 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the Acceleron EHA (Free EHA Whitepaper) Congress Review.

To access the live webcast, please select "Events & Presentations" in the Investor section on Acceleron’s website (www.acceleronpharma.com) at least 10 minutes beforehand to ensure time for any downloads that may be required.

An archived webcast recording will be available on the Acceleron website beginning approximately two hours after the event.

About Luspatercept

Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Acceleron and Celgene are enrolling Phase 3 clinical trials that are designed to evaluate the safety and efficacy of luspatercept in patients with myelodysplastic syndromes (the "MEDALIST" study) and in patients with beta-thalassemia (the "BELIEVE" study). For more information, please visit www.clinicaltrials.gov.

Aptose Bio inks license deal with CrystalGenomics for cancer candidate

On June 9, 2016 Aptose Biosciences (APTO +8.1%) reported that it enters into an exclusive global option and license agreement with Seoul, South Korea-based CrystalGenomics to develop CG026806, a small molecule inhibitor of a group of enzymes called Aurora kinases, as well as Bruton’s tyrosine kinase (BTK) and FMS-like tyrosine kinase (FLT3) (Press release, CrystalGenomics, JUN 9, 2016, View Source;id=1463&page=4&num=93&nowpos=512&type=&sermun=&qu=&tb_name=eng_news&rt_page=/en/news/news.php [SID1234539168]).

CrystalGenomics could earn up to $303M, including milestones, and will receive single-digit royalties on worldwide net sales exclusive of Korea and China.

The product candidate, expected to enter early-stage development in mid-2017, has potential efficacy in a range of cancers, including blood cancers, and certain autoimmune diseases. BTK plays a key role in B cell blood cancers, FLT3 occurs in about a third of acute myeloid leukemia and Aurora kinases are principal drivers of certain blood cancers and solid tumors.

Aptose Biosciences, CrystalGenomics Launch Cancer Drug Collaboration

On June 9, 2016 Aptose Biosciences reported it will co-develop CrystalGenomics’ preclinical cancer drug candidate CG026806, the companies said, under an exclusive global option and license agreement that could generate up to $303 million-plus for CrystalGenomics (Press release, CrystalGenomics, JUN 9, 2016, View Source;id=1465&page=4&num=94&nowpos=510&type=&sermun=&qu=&tb_name=eng_news&rt_page=/en/news/news.php [SID1234539167]).

Aptose said it expects to undertake IND-enabling studies immediately, and, if it exercises an option under the agreement, to launch a Phase I clinical trial by mid-2017. Upon exercise of that option, Aptose will own global rights to develop and commercialize CG026806 outside of South Korea and China.

"We are impressed by the ability of once-daily oral dosing of CG026806 to demonstrate tumor eradication in the absence of toxicity in murine xenograft models of human hematologic malignancies," William G. Rice, Ph.D., Aptose’s Chairman, President and CEO, said in a statement.

The companies said their deal was valued at $303 million, with Aptose agreeing to pay CrystalGenomics payments tied to achieving development, regulatory, and commercial-based milestones. CrystalGenomics is also eligible to receive a single-digit royalty on sales worldwide except China and South Korea.

CG026806 is a first-in-class, noncovalent, small molecule inhibitor of Bruton’s tyrosine kinase (BTK), FMS-like tyrosine kinase 3 (FLT3), and Aurora kinases (AURK). The candidate is CrystalGenomics’ lead compound among BTK/FLT3/AURK inhibitors.

Oncology is one of CrystalGenomics’ therapeutic areas, along with infectious disease and inflammation. The company won approval in February from the Korean Ministry of Food and Drug Safety (MFDS) for its osteoarthritis drug Acelex (polmacoxib). Aptose specializes in cancer drugs and diagnostics.

The companies envision CG026806 fighting multiple forms of cancer, particularly those resistant to current BTK inhibitors or those that possess the FLT3-internal tandem duplication (ITD) alteration.

BTK plays a key role in B-cell hematologic malignancies, such as chronic lymphocytic leukemia and mantle cell lymphoma, as well as some autoimmune diseases. FLT3, including the ITD, a mutation of the FLT3 gene, occurs in approximately 30–35% of patients with acute myeloid leukemia. AURK participate in the epigenetic phosphorylation of histones and are key drivers in a series of hematologic malignancies and solid tumors.

argenx announces presentation of updated ARGX-110 phase 1 data in TCL at EHA 2016

On June 9, 2016 argenx (Euronext Brussels: ARGX), a clinical-stage biopharmaceutical company focused on creating and developing differentiated therapeutic antibodies for the treatment of cancer and severe autoimmune diseases, reported the presentation of efficacy and safety data from its Phase 1 expansion study of ARGX-110 in patients with T-cell lymphoma (TCL) during an e-poster session at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress (Copenhagen, Denmark) (Press release, arGEN-X, JUN 9, 2016, View Source [SID:1234513200]).

The data from the Phase 1 expansion study show evidence of clinical and/or biological anti-tumor activity with ARGX-110 in highly refractory cutaneous TCL & peripheral TCL patients with confirmed overexpression of CD70. The abstract can be accessed here.

"The data presented today during EHA (Free EHA Whitepaper) 2016 are very encouraging and further support the important role of CD70 in the TCL patient population. We are seeing signs of biological activity in both CTCL and PTCL patients that have failed standard therapy, as well as favourable safety profiles, so we are eager to present the full data set later this year in this critical indication," said Tim van Hauwermeiren, Chief Executive Officer of argenx. "We remain on track with patient recruitment, which we expect to complete by mid 2016."

About ARGX-110

ARGX-110 is a SIMPLE Antibody targeting CD70, an immune checkpoint target involved in hematological malignancies, several solid tumors and severe autoimmune diseases. ARGX-110 works in three ways: i) blocks growth of tumor cells, ii) kills cancer cells and iii) restores immune surveillance against tumors (Silence K. et al. mAbs 2014; 6 (2):523-532). ARGX-110 is currently being evaluated in both hematological and solid tumors.

Blue Earth Diagnostics Announces AxuminTM (Fluciclovine F 18) Presentations at Upcoming Society of Nuclear Medicine and Molecular Imaging (SNMMI) Annual Meeting

On June 0, 2016 Blue Earth Diagnostics, a molecular imaging diagnostics company, reported the presentation of results from Axumin (fluciclovine F 18) injection studies in biochemically recurrent prostate cancer at the Society of Nuclear Medicine and Molecular Imaging Annual Meeting (SNMMI), from June 11-15, 2016 in San Diego, Ca Blue Earth Diagnostics Announces AxuminTM (Fluciclovine F 18) Presentations at Upcoming Society of Nuclear Medicine and Molecular Imaging (SNMMI) Annual Meeting
(Press release, Blue Earth Diagnostics, JUN 9, 2016, View Source [SID:1234513170]). The Company will also participate in a panel presentation on new imaging agents. Details of Axumin presentations by Blue Earth Diagnostics and its collaborators are listed below.

Date: Sunday, June 12, 2016
Presentation:
Fluciclovine F18 (FACBC): An Amino Acid Tracer for the Staging of Recurrent Prostate Cancer
Session Title: New Imaging and Therapeutic Agents on the Horizon for Routine Clinical Use
Presenter: Jonathan Allis, D. Phil.
Presentation Time: 12:30 PM – 2:00 PM PT
Location: 25 A

Date: Monday, June 13, 2016
Poster Title:
Evidence of the effectiveness of reader training for the staging of biochemically recurrent prostate cancer using fluciclovine F18 PET-CT
Session Title: Prostate/GU: poster session
Presenter: Matthew P. Miller, Ph.D.
Presentation Time:
3:00 PM – 4:30 PM PT
Location: Exhibit Hall G
Publication No.: 1556

In addition, the following presentation will be part of an independent continuing education program at SNMMI.

Date: Tuesday, June 14, 2016
Presentation:
Fluciclovine F18: A New Option for Biochemical Recurrence in Prostate Cancer
Session Title: CE79 Imaging Prostate Cancer
Presenter: Trond V. Bogsrud, M.D., Ph.D.
Presentation Time: 2:45 PM – 4:15 PM PT
Location: 20 BC

Blue Earth Diagnostics invites participants at this year’s SNMMI Annual Meeting to visit the Company at Exhibit Booth 337.

About AxuminTM (fluciclovine F 18)

Axumin (fluciclovine F 18) injection is a novel product indicated for use in positron emission tomography (PET) imaging to identify suspected sites of prostate cancer recurrence in men. Recurrence of prostate cancer is suspected by an increase in prostate specific antigen (PSA) levels following initial therapy. PET imaging with Axumin may identify the location and extent of such recurrence. Axumin was developed to enable visualization of the increased amino acid transport that occurs in many cancers, including prostate cancer. It consists of a synthetic amino acid that is preferentially taken up by prostate cancer cells compared with surrounding normal tissues, and is labeled with the radioisotope F18 for PET imaging. Axumin was approved by the U.S. Food and Drug Administration on May 27, 2016 following Priority Review, and is the first product commercialized by Blue Earth Diagnostics, which licensed the product from GE Healthcare. The molecule is being investigated by Blue Earth Diagnostics for other potential cancer indications, such as glioma.

Indication and Important Safety Information About Axumin

INDICATION

Axumin (fluciclovine F 18) injection is indicated for positron emission tomography (PET) imaging in men with suspected prostate cancer recurrence based on elevated blood prostate specific antigen (PSA) levels following prior treatment.

IMPORTANT SAFETY INFORMATION

Image interpretation errors can occur with Axumin PET imaging. A negative image does not rule out recurrent prostate cancer and a positive image does not confirm its presence. The performance of Axumin seems to be affected by PSA levels. Axumin uptake may occur with other cancers and benign prostatic hypertrophy in primary prostate cancer. Clinical correlation, which may include histopathological evaluation, is recommended.
Hypersensitivity reactions, including anaphylaxis, may occur in patients who receive Axumin. Emergency resuscitation equipment and personnel should be immediately available.
Axumin use contributes to a patient’s overall long-term cumulative radiation exposure, which is associated with an increased risk of cancer. Safe handling practices should be used to minimize radiation exposure to the patient and health care providers.
Adverse reactions were reported in ≤ 1% of subjects during clinical studies with Axumin. The most common adverse reactions were injection site pain, injection site erythema and dysgeusia.