Genmab Announces Phase II Study of Daratumumab in Smoldering Multiple Myeloma

On November 24, 2014 Genmab reported that its collaboration partner, Janssen Biotech, Inc. (Janssen) plans to start a Phase II study of daratumumab in smoldering multiple myeloma (Press release Genmab, NOV 24, 2014, View Source [SID:1234501009]). The study (SMM2001) will evaluate three different dose schedules of daratumumab for the treatment of smoldering multiple myeloma. The study is expected to start enrolling patients in 2015.

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"We are pleased to announce this study, which illustrates that the development plan for daratumumab encompasses all stages of multiple myeloma. Smoldering multiple myeloma is a challenging indication, as physicians will evaluate treating patients at an early stage of the disease, with the intent to extend the period before the disease transitions to symptomatic multiple myeloma," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

ARIAD and STA Announce Approval of Iclusig (Ponatinib) in Australia

On November 24, 2014 ARIAD and Specialised Therapeutics Australia reported the marketing approval of IclusigTM (ponatinib) in Australia by the Therapeutic Goods Administration (TGA) (Press release Ariad, NOV 24, 2014, View Source;p=RssLanding&cat=news&id=1992387 [SID:1234501007]).

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The Australian Product Information for Iclusig states that it is indicated for the treatment of adult patients with:

Chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) whose disease is resistant to, or who are intolerant of at least two prior tyrosine kinase inhibitors; or where there is a T315I mutation.
Philadelphia-chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) whose disease is resistant to, or who are intolerant of dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or where there is a T3151 mutation.

Therapy should be initiated and monitored by a haematologist with expertise in managing adult leukaemias.

"Up to thirty percent of patients with CML become resistant to current therapies, and patients with resistant disease eventually run low on treatment options," said Professor Timothy Hughes, Consulting Haematologist at the Royal Adelaide Hospital and one of the PACE trial investigators. "Iclusig will be a valuable new therapy for refractory leukaemia patients and treating clinicians in Australia."

ARIAD submitted its marketing application for Iclusig in the third quarter of 2013 to the Therapeutics Goods Administration (TGA), in Australia. Commercial launch of Iclusig is expected to occur early in 2015.

"We are very pleased with the approval of Iclusig in Australia and will work closely with STA to make Iclusig available to appropriate Philadelphia-positive leukaemia patients as quickly as possible," stated Harvey J. Berger, M.D., chairman and chief executive officer of ARIAD. "We look forward to continuing our strong collaboration with STA to provide this important treatment option to refractory CML patients in Australia."

"Iclusig provides a new treatment option for patients with difficult-to-treat CML or Ph+ ALL who previously had limited therapies available to them," said Carlo Montagner, chief executive officer at STA. "We look forward to the Pharmaceutical Benefit Advisory Committee’s decision on Iclusig’s reimbursement for Australian patients under the Pharmaceutical Benefits Scheme."

The TGA decision was based on results from the pivotal Phase 2 PACE (Ponatinib Ph+ ALL and CML Evaluation) trial in patients with CML or Ph+ ALL who were resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy, or who had the T315I mutation of BCR-ABL. Iclusig demonstrated anti-leukemic activity achieving a major cytogenetic response (MCyR) in 54 percent of chronic-phase CML patients and in 70 percent of patients with the T315I mutation.1, 2 MCyR within the first 12 months was the primary endpoint of the PACE trial for chronic-phase patients.1, 2

In patients with advanced disease, 57 percent of accelerated-phase CML patients and 34 percent of blast-phase CML patients achieved a major hematologic response (MaHR) with Iclusig. MaHR within the first 6 months was the primary endpoint in the trial for patients with advanced disease.1, 2

The most common (>1%) serious adverse reactions for Iclusig were pancreatitis, abdominal pain, decrease in platelet count, lipase increased, anaemia, cardiac failure, coronary artery disease, diarrhoea, decreased neutrophil count, febrile neutropenia, pancytopenia, and pyrexia.2 The most common (≥20%) adverse reactions of any severity were decrease in platelet count, rash, dry skin, and abdominal pain.2

CML is a cancer of the white blood cells that is diagnosed in approximately 330 patients each year in Australia.3 CML and Ph+ ALL patients treated with TKIs can develop resistance or intolerance over time to these therapies. Iclusig is a targeted cancer medicine discovered and developed at ARIAD. It was designed by ARIAD scientists using ARIAD’s platform of computational chemistry and structure-based drug design to inhibit BCR-ABL, including drug-resistant mutants that arise during treatment. Iclusig is the only TKI that has received an approval in Australia for an indication that includes CML and Ph+ ALL patients with the T315I mutation.

For further information, please consult the full Iclusig Product Information, available at View Source

Pipeline

RAD1901 is a selective estrogen receptor down-regulator/degrader, or SERD, that crosses the blood-brain barrier and is being evaluated by Radius Health for the potential treatment of breast cancer brain metastases (BCBM). (Company Pipeline Radius, NOV 24, 2014, View Source [SID:1234501002]).

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Pipeline

SRX-1177 is a peptide conjugated to commercially available radionuclides to target the melanocortin-1 receptor (MC1-R) for the treatment of melanoma (Company Pipeline SolaranRx, NOV 24, 2014, View Source [SID:1234501001]).

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Transposagen Biopharmaceuticals Enters Into Research Collaboration and Worldwide License Agreement with Janssen for Allogeneic Chimeric Antigen Receptor (CAR) T-Cell Therapies

On November 24, 2014 Transposagen Biopharmaceuticals reported that they have entered into a research collaboration and worldwide license agreement with Janssen Biotech, Inc. (Janssen) to develop allogeneic Chimeric Antigen Receptor T-cells (CAR-T) (Press release Transposagen, NOV 24, 2014, View Source [SID:1234501015]). To date, CAR-T therapies have shown promise in early human clinical trials for the treatment of blood cancers and allogeneic CAR-Ts have the potential for use as off-the-shelf cancer treatments without the need of matching donor with recipient.

Transposagen will be using its proprietary genome editing technologies, including the piggyBacTM Footprint-FreeTM Gene Editing System, to create the allogeneic CAR-T therapies. Under the agreement, Janssen has exclusive rights to any allogeneic CAR-T therapy that is jointly developed by Transposagen and Janssen. In addition, Janssen has received a non-exclusive research license to utilize Transposagen’s proprietary gene editing technologies for gene and cell therapy solutions for treating diseases with significant unmet medical need. Transposagen will retain the rights to develop autologous CAR-T therapies and CAR-T therapies using Natural Killer (NK) cells or NK-like cells.

Janssen will pay Transposagen up to $292 million per CAR-T therapeutic, which includes an up-front fee and potential development, regulatory, and commercial milestone payments. Transposagen will also receive tiered royalties on net sales of any allogeneic CAR-T products that are commercialized by Janssen.

Transposagen will enter into a 3-year research collaboration with Janssen whereby both companies will work together on preclinical research. Janssen will be responsible for manufacturing and commercialization of allogeneic CAR-T therapies.

“The research collaboration with Janssen will pair Transposagen’s cutting-edge gene editing and gene delivery technology and expertise with Janssen industry-leading technologies in the antibody and antibody alternative areas to create what may be the ideal CAR-T therapy,” said Eric Ostertag, President and CEO of Transposagen.