On January 4, 2018 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, and Riken Genesis, a subsidiary of Sysmex Corporation (TOKYO, 6869), reported that they have entered into a partnership to introduce nCounter-based diagnostic assays in Japan (Press release, NanoString Technologies, JAN 4, 2018, View Source [SID1234522864]).

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Under this agreement, NanoString and Riken Genesis will collaborate to register, obtain reimbursement and commercialize companion diagnostic assays in Japan, including NanoString’s Lymphoma Subtyping Test which will be marketed as the nCounter Dx LymphMarkTM assay.

LymphMark is a 20-gene signature that classifies cell-of-origin subtypes of Diffuse Large B-cell Lymphoma (DLBCL) tumors. The initial indication for the LymphMark assay is expected to be a potential companion diagnostic to aid in identifying DLBCL patients for treatment. The LymphMark assay is based on the Lymph2Cx gene signature that was originally developed by the Lymphoma/Leukemia Molecular Profiling Project (LLMPP) and has demonstrated analytical robustness and potential clinical utility.

The LymphMark assay was used to select patients in a Phase III clinical trial. The LymphMark assay is being evaluated in more than 40 research studies that are being conducted with 23 companies.

"Through this collaboration, we will work closely with Riken Genesis to launch novel diagnostic products on the nCounter platform that have the potential to guide decision making in the Japanese oncology community," said Brad Gray, chief executive officer of NanoString.

"We are pleased to partner with NanoString to bring new highly multiplexed molecular assays to clinicians and patients in Japan," said Dr. Naoto Kondo, president and chief executive officer of Riken Genesis. "These innovative assays will be an important addition to our portfolio of diagnostic tests."

On January 4, 2018 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported that it has initiated a Phase 3 clinical study to evaluate the safety and efficacy of DCC-2618, a pan-KIT and PDGFRα inhibitor, in patients with advanced gastrointestinal stromal tumors (GIST) (Press release, Deciphera Pharmaceuticals, JAN 4, 2018, View Source [SID1234522871]).

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"We are extremely pleased to initiate the INVICTUS study with DCC-2618 in heavily pretreated GIST patients, specifically fourth-line and fourth-line plus patients," said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. "We expect to report top-line results in 2019 and, if successful, this pivotal Phase 3 study could serve as the basis for a New Drug Application (NDA), providing a much-needed therapeutic option for these patients for whom there are no approved treatments. We also plan to initiate a second Phase 3 study later this year evaluating DCC-2618 in second-line GIST patients who have progressed or are intolerant to front-line therapy with imitanib."

"While effective treatments are available for patients with early-stage GIST, in 9 out 10 patients the disease will eventually progress due to the development of secondary drug resistance mutations," said Professor Jean-Yves Blay, Medical Oncologist, General Director Centre Léon Bérard, Comprehensive cancer Centre of Lyon, France. "A therapy with the potential to provide broad coverage across the full spectrum of KIT and PDGFRα mutations would represent a much-needed improvement over currently approved treatment options for patients with later-stage GIST."

Initiation of the INVICTUS study follows results from the ongoing Phase 1 clinical trial presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in September 2017, in which durable disease control by DCC-2618 was observed in heavily pretreated patients with GIST.

INVICTUSPhase 3 Study
The INVICTUS Phase 3 clinical study is a randomized, double-blind, placebo-controlled, international, multicenter trial to evaluate the safety, tolerability, and efficacy of DCC-2618 compared to placebo in patients with advanced GIST patients whose previous therapies have included imatinib, sunitinib, and regorafenib. The trial is expected to enroll approximately 120 patients randomized 2:1 to either 150 mg once daily of DCC-2618 or placebo. The primary efficacy endpoint is median progression free survival (PFS) as determined by independent radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST). Secondary endpoints as determined by independent radiologic review using modified RECIST include Objective Response Rate (ORR); Time to Tumor Progression (TTP); and Overall Survival (OS). See www.clinicaltrials.gov for further information (NCT03353753).

About DCC-2618
DCC-2618 is a pan-KIT and PDGFRα kinase switch control inhibitor in clinical development for the treatment of KIT and/or PDGFRα-driven cancers, including gastrointestinal stromal tumors, glioblastoma multiforme and systemic mastocytosis. DCC-2618 was specifically designed to improve the treatment of GIST patients by inhibiting the full spectrum of mutations in KIT and PDGFRα. DCC-2618 is a pan-KIT and pan-PDGFRα inhibitor that blocks initiating KIT mutations in exons 9, 11, 13, 14, 17, and 18, known to be present in GIST patients and the D816V exon 17 mutation known to be present in ASM patients. DCC-2618 inhibits PDGFRα mutations in exon 18, including the D842V mutation that drives a subset of GIST.

On January 4, 2017 Rocket Pharmaceuticals, Ltd., a leading U.S.-based multi-platform gene therapy company addressing challenging rare diseases, reported the completion of its merger with Inotek Pharmaceuticals Corporation ("Inotek") (Press release, Rocket Pharmaceuticals, JAN 4, 2018, View Source [SID1234522921]). The combined company ("Company") will be named Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket") and will focus on advancing a pipeline of gene therapy programs targeting rare and undertreated diseases. Rocket’s common stock will be listed on the NASDAQ Global Market under the symbol "RCKT" and is expected to be begin trading on January 5, 2018. Rocket is based in New York City and led by President and Chief Executive Officer Gaurav Shah, M.D., who previously was a Global Program Head in the Cell & Gene Therapies Unit at Novartis.

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"The support for this transaction by both Inotek and Rocket shareholders was evident today, underscoring support for our long-term growth strategy to become a fully-integrated, multi-platform gene therapy company"

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"The support for this transaction by both Inotek and Rocket shareholders was evident today, underscoring support for our long-term growth strategy to become a fully-integrated, multi-platform gene therapy company," said Dr. Shah. "The closing of this merger provides immediate value to grow our operations, execute on our clinical development goals, and expand our in-house manufacturing and analytics capabilities. As we enter this next stage of growth as a publicly traded company, we are focused on driving Company value by bringing our current pipeline of five programs to major value inflection points as rapidly as possible, and achieving first mover advantage in these markets."

Rocket’s Pipeline

Rocket utilizes a multi-platform development approach that leverages the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene delivery methods and is initially targeting devastating rare diseases in children that lead to early mortality in the absence of bone marrow transplant or other invasive procedures.

The Company’s lead program, a Phase 1/2 LVV-based gene therapy for Fanconi Anemia (FA), is currently in clinical trials with academic partners in the U.S. and Europe. FA causes genetic instability due to mutations in DNA repair genes resulting in early bone marrow failure and malignancy. Early results in FA patients have demonstrated clinical engraftment of ex vivo-transduced autologous hematopoietic stem cells (HSCs). The proportion of gene-corrected cells increases over time, confirming the selective advantage of gene-corrected cells in the bone marrow without requiring conditioning (i.e. destruction of bone marrow prior to transplant). Functional correction and clinical proof of concept have also been observed. Both blood and marrow cells demonstrate resistance to DNA-damaging agents (sensitivity to DNA-damaging agents is a diagnostic feature of FA). Patients demonstrated stable or improving blood cell counts during the months following treatment despite decreases noted during the months and years preceding gene therapy. Additional patient data are expected in 2018, with a registration study anticipated to start in 2019.

Three additional LVV-based programs are currently in preclinical development and target Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and Infantile Malignant Osteopetrosis (IMO). The LAD-I program is expected to advance into the clinic in 2018, with the PKD and IMO programs to follow in 2019.

An undisclosed AAV-based gene therapy program is expected to enter the clinic in the next year and has demonstrated encouraging histological correction of the disease phenotype. This program targets a monogenic pediatric disease with early mortality and represents the first gene therapy being developed for this large class of indications.

Rocket’s Management

The combined Company’s executive management team will be led by Dr. Shah and will consist of: Jonathan Schwartz, M.D., Chief Medical Officer, who led several biologics approvals as Vice President of Clinical Development at ImClone Systems/Eli Lilly and Company; Kinnari Patel, Pharm.D., MBA, newly appointed Chief Operating Officer, who led regulatory filings for six rare disease agents as well as for Opdivo while at Bristol-Myers Squibb; and Brian Batchelder, MBA, Vice President of Finance, who previously served as Chief Financial Officer of ImClone Systems, a subsidiary of Eli Lilly and Company.

In addition, Rocket appointed Claudine Prowse, Ph.D., as Head of Corporate Development and Investor Relations Officer. Previously, she was Head of Strategy at Inotek, where she was integral to the merger transaction with Rocket Pharmaceuticals, Ltd. Prior to that, she was Vice President of Investor Relations at Biogen.

About the Merger

Prior to the closing of the merger, Inotek effected a 1 for 4 reverse split of its common stock. Following the reverse stock split and closing of the merger, there will be approximately 33.1 million shares of the combined company’s common stock outstanding with prior Rocket shareholders owning approximately 79.4% and prior Inotek shareholders owning approximately 20.6%. Based on the reverse stock split, the conversion rate of the combined Company’s $52.0 million of 5.75% Convertible Senior Notes due 2021 will automatically be adjusted from 124.7505 shares of common stock per $1,000 principal amount of the notes to 31.1876 shares of common stock per $1,000 principal amount of the notes. Cash, cash equivalents and short-term investments for the combined Company at closing were approximately $117.2 million.

On January 4, 2018 Intrexon Corporation (NYSE: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported that Randal J. Kirk, Chairman and Chief Executive Officer will present at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 10th at 9:30 a.m. Pacific Time (Press release, Intrexon, JAN 4, 2018, View Source [SID1234522882]).

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A live webcast of the presentation will be available on the Investors section of Intrexon’s website at View Source Replay of the webcast will be available for 30 days following the event.

On January 4, 2018 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that the first patient has been treated with IMGN632 in a Phase 1 clinical trial of patients with CD123-positive hematological malignancies, including acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN) (Press release, ImmunoGen, JAN 4, 2018, View Source [SID1234522879]).

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IMGN632 uses ImmunoGen’s novel indolino-benzodiazepine payload, DGN549, which alkylates DNA without crosslinking, as well as novel linker technology with a CD123-targeting antibody. In preclinical studies with IMGN632, ImmunoGen has reported potent and selective activity against AML cells with lower cytotoxicity to normal myeloid progenitor cells than an ADC designed to crosslink DNA.1 Supporting preclinical data for IMGN632 have also shown compelling activity in AML and acute lymphoblastic leukemia (ALL) models with single and multi-dose regimens.2,3 These data suggest that IMGN632 has the potential to be a highly effective, yet tolerable ADC.

"We continue to rapidly advance our novel IGN portfolio in a number of hematological malignancies and are pleased to be moving our second IGN ADC, IMGN632, into the clinic," said Anna Berkenblit, M.D., VP and Chief Medical Officer of ImmunoGen. "Our IGN payloads were developed to meet the dual challenges of achieving high potency against target cells, while enabling continued patient treatment. We believe IMGN632 has the potential to be a highly effective therapy with favorable tolerability for the treatment of patients with CD123-positive hematologic malignancies, including AML and BPDCN, cancers where new therapies are desperately needed."

The Phase 1 trial in AML and BCPDN will follow a once every three week dosing schedule while in its dose-finding stage. The selected dose will then be used in expansion cohorts assessing IMGN632 in patients with BPDCN, AML, ALL, and other CD123-positive hematologic malignancies.

"We are excited to be leading off the clinical evaluation of IMGN632, a potential new treatment option for patients with CD123-positive hematologic malignancies," said Hagop M. Kantarjian, M.D., professor and chair of the Department of Leukemia at the University of Texas MD Anderson Cancer Center and principal investigator of the trial of IMGN632.

Data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper) 2017 demonstrated promising activity and safety with IMGN632 in preclinical models of B-cell ALL (B-ALL).4 CD123 expression is prevalent across ALL subtypes, including 90% of B-ALL and nearly half of T-cell acute lymphoblastic leukemia. IMGN632 demonstrated promising activity against B-ALL cell lines and patient samples in vitro, including the elimination of more than 90% of B-ALL blasts in 6 out of 8 patient samples. Normal cells were not affected by IMGN632 at 100-fold higher concentrations.

This is the second clinical trial using IGNs, a new class of cancer-killing agents developed by ImmunoGen for use in ADCs. ImmunoGen recently reported findings from the Company’s ongoing Phase 1 study of IMGN779 in patients with relapsed or refractory adult AML whose tumors express CD33.5 The data demonstrate that IMGN779 is well-tolerated with no dose-limiting toxicities, pharmacokinetic exposures and pharmacodynamic CD33 saturation increasing with dose, and anti-leukemia activity observed in patients with poor prognostic features.

About IMGN632
IMGN632 is a humanized anti-CD123 ADC that is a potential treatment for AML, BPDCN, myelodysplastic syndrome, B-cell ALL and other CD123-positive malignancies. IMGN632 uses a novel IGN payload, linker and antibody technology, and has demonstrated potent and selective activity, with minimal cytotoxic effects, in preclinical models of AML and ALL.6,7

About IGNs
Indolino-benzodiazepine agents, or IGNs, are a new class of cancer-killing agent developed by ImmunoGen for use in ADCs. IGN payloads were designed to meet the dual challenges of achieving high potency against target cells, while having a tolerability profile that can enable continued patient treatment. These ultra-potent, DNA-acting IGNs alkylate DNA without crosslinking, which preclinically has resulted in potent anti-leukemia activity with relative sparing of healthy cells.8,9

About Acute Myeloid Leukemia (AML)
AML is a cancer of the bone marrow cells that produce white blood cells. It causes the marrow to increasingly generate abnormal, immature white blood cells (blasts) that do not mature into effective infection-fighting cells. The blasts quickly fill the bone marrow, impacting the production of normal platelets and red blood cells. The resulting deficiencies in normal blood cells leave the patient vulnerable to infections, bleeding problems and anemia.

It is estimated that, in the U.S. alone, 21,380 patients will be diagnosed with AML this year and 10,590 patients will die from the disease.10

About Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

BPDCN is a disease of the bone marrow and blood that affects multiple organs, including the lymph nodes and the skin. It often presents as leukemia or lymphoma. There are little data about BPDCN and there is no established treatment. The average age at diagnosis is 60 to 70 years. There are more men than women who are diagnosed with BPDCN.11,12