On August 1, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, and Orano Med (formerly AREVA Med) a subsidiary of Orano, a nuclear biotech company developing innovative therapies in oncology, reported that the two companies have entered into an agreement to combine certain proprietary technologies from each company to create a novel oncologic therapy (Press release, Cellectar Biosciences, AUG 1, 2018, View Source [SID1234528397]). The collaboration will focus on the development of novel phospholipid drug conjugates (PDC) utilizing Orano Med’s unique alpha emitter, lead-212 (212Pb), conjugated to Cellectar’s phospholipid ether (PLE). The companies intend to evaluate the new PDC in up to three oncology indications.

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"We believe that the combination of Cellectar’s targeted delivery with Orano Med’s powerful alpha emitter offers the potential to create a novel and highly potent cancer therapy," said James Caruso, chief executive officer of Cellectar Biosciences. "This collaboration is an ideal strategic fit and provides an excellent opportunity to expand our radiotherapeutic portfolio beyond CLR 131, a highly potent beta emitter, and establish one of the most complete oncology-focused radiotherapeutic portfolios."

Cellectar’s proprietary PLE and PLE analogs provide targeted delivery of various molecules, including radioisotopes, to malignant tumor cells with up to 30-fold more payload delivered to the tumor versus normal tissues. Orano Med’s 212Pb is a unique alpha emitter that provides high energy delivery over a shorter distance than other radioisotopes. The higher energy associated with alpha particles causes non-repairable double stranded DNA breaks. As a result, enhanced tumor targeting of the construct may allow the 212Pb to provide greater efficacy at lower doses with less side effects.

Orano Med has partnered 212Pb with other companies to create a broad pipeline of tumor targeting 212Pb therapies. These other collaborations are using diverse biological targeting vectors or pursuing indications separate from those planned in this collaboration. Many of these approaches utilize antibodies or peptides; the most advanced of these approaches has recently entered a Phase 1 clinical trial.

Cellectar and Orano Med believe that the PLE conjugated to 212Pb could be an ideal drug candidate and provide improved anti-cancer effects beyond those seen with some of the other delivery technologies.

"This collaboration with Cellectar is an exciting opportunity for Orano Med. Our 212Pb is a powerful radioactive isotope that at low doses kills cancer cells and has limited impact on nearby healthy cells. We believe that 212Pb conjugated to Cellectar’s PLE has great potential to improve patient outcomes by having a better efficacy and safety profile than other technologies," said Julien Dodet, CEO of Orano Med.

Under the terms of the agreement, early preclinical costs will be shared equally between the organizations with both parties having an option to advance and commercialize the PDC alone or in collaboration with each other. The option is exercisable after establishment of early proof of concept data.

About Phospholipid Drug Conjugates

Cellectar’s product candidates are built upon a patented delivery and retention platform that utilizes optimized phospholipid ether-drug conjugates (PDCs) to target cancer cells. The PDC platform selectively delivers diverse oncologic payloads to cancerous cells and cancer stem cells, including hematologic cancers and solid tumors. This selective delivery allows the payloads’ therapeutic window to be modified, which may maintain or enhance drug potency while reducing the number and severity of adverse events. This platform takes advantage of a metabolic pathway utilized by all tumor cell types in all cell cycle stages. Compared with other targeted delivery platforms, the PDC platform’s mechanism of entry does not rely upon specific cell surface epitopes or antigens. In addition, PDCs can be conjugated to molecules in numerous ways, thereby increasing the types of molecules selectively delivered. Cellectar believes the PDC platform holds potential for the discovery and development of the next generation of cancer-targeting agents.

About 212Pb

212Pb is a promising agent for use in the field of alpha particle radiotherapy that has been tested in clinical trials. Alpha particle emitting radiotherapies, like 212Pb, cause double stranded DNA breaks by releasing high energy particles over a short distance. 212Pb represents one of the more powerful alpha emitters and has a half-life of 10.6 hours.

On August 1, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported it will report its second quarter 2018 financial results and business progress on Tuesday, August 7, 2018, after the close of the U.S. financial markets (Press release, Tocagen, AUG 1, 2018, View Source;p=RssLanding&cat=news&id=2361441 [SID1234528395]).

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On August 1, 2018 The Medicines Company (NASDAQ:MDCO) reported its financial results for the second quarter ended June 30, 2018 (Press release, Medicines Company, AUG 1, 2018, View Source;p=RssLanding&cat=news&id=2361238 [SID1234528394]).

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"During the second quarter of 2018, we continued to advance inclisiran’s development programs, including the accumulation of further promising safety data from 3,660 patients in Phase III trials," said Clive Meanwell, M.D., Ph.D., Chief Executive Officer of The Medicines Company. "We were also able to present consistent phase II subset efficacy data in various demographic and disease populations for LDL-C and other atherogenic lipoproteins. We look forward to further progress in 2018 and Phase III data read-outs in 2019."

Second quarter 2018 highlights included the following:

In April, the Company presented new data and analyses from multiple studies in the ORION development program for inclisiran at the National Lipid Association 2018 Scientific Sessions. The data demonstrated that inclisiran likely has a "one-size-fits-all" dosing regimen, without the necessity of dose adjustments, across a wide range of dyslipidemia patient populations, including those hard-to-treat patients with homozygous familial hypercholesterolemia (HoFH) and other sub-groups, such as patients with renal impairment and diabetes. The data showed that inclisiran lowered low-density lipoproteins cholesterol (LDL-C) by more than 50% for a wide range of dyslipidemia patient populations and sub-groups, and by up to 44% in HoFH patients.
In May, the Company presented the results of a pre-specified analysis of secondary endpoints from the ORION-1 Phase II trial at the 86thEuropean Atherosclerosis Society Congress. The results, which were published in Circulation, the journal of the American Heart Association, showed that, beyond its powerful effect on LDL-C, inclisiran also reduced atherogenic lipoproteins in a profound and sustained manner. Atherogenic lipoproteins – non-HDL-C, ApoB, VLDL-C and Lp(a) – have been associated with an increased risk of heart attacks and strokes, particularly in high-risk patients. The reductions, which were generally dose-dependent, were achieved most clearly with a 300 mg dose of inclisiran given on Day-1 and Day-90, and were sustained to the pre-specified time of assessment (180 days) and beyond (at least 210 days). This is the same starting dose of inclisiran being utilized in the Phase III trials (the Phase III dose of inclisiran is 300 mg given on Day-1 and Day-90, and then every six months thereafter).
In June, the Company presented new data from a pre-specified, subgroup analysis of dosing, efficacy and safety of inclisiran in patients with diabetes from the ORION-1 Phase II trial at the American Diabetes Association 78th Scientific Sessions. The data demonstrated that a subcutaneous injection of 300 mg of inclisiran given at Day-1 and Day-90 lowered LDL-C at Day-180 by more than 50% in patients with atherosclerotic cardiovascular disease (ASCVD) and those considered ASCVD-risk equivalents, regardless of whether those patients had diabetes. Importantly, the data showed that patients with and without diabetes experienced similar adverse event profiles, including no effects on control of blood glucose levels over six months.
In June, the Independent Data Monitoring Committee (IDMC) for the ongoing inclisiran Phase III clinical trials conducted its third, planned review of safety and efficacy data from the trials and recommended that they continue without modification. At the time of the review, substantially all patients in trials had received two doses of inclisiran or placebo, and more than 1,550 patient-years of safety data for inclisiran had been accumulated – with an additional 5 patient-years of safety data continuing to accumulate every day.
During the second quarter, the Company substantially completed the implementation of its previously-announced restructuring, as anticipated.
Commenting further, Dr. Meanwell said, "We continued to deliver against our 2018 objectives during the second quarter, demonstrating strong execution on all fronts. We remain sharply focused on tightening expense management and advancing the inclisiran development program efficiently."

Second-Quarter 2018 Financial Summary from Continuing Operations

On a GAAP basis, loss from continuing operations in the second quarter of 2018 was $54.5 million, or $0.74 per share, compared to a loss of $370.1 million, or $5.15 per share, in the second quarter of 2017. Included in loss from continuing operations for the second quarter of 2018 were restructuring charges of $6.1 million. On a non-GAAP basis, adjusted loss(1) from continuing operations in the second quarter of 2018 was $46.3 million, or $0.63(1) per share, compared to a loss of $52.0 million, or $0.72(1) per share, in the second quarter of 2017.

First Half 2018 Financial Summary from Continuing Operations

On a GAAP basis, loss from continuing operations in the first half of 2018 was $139.3 million, or $1.89 per share, compared to a loss of $441.1 million, or $6.17 per share, in the first half of 2017. Included in loss from continuing operations for the first half of 2018 was a non-cash, mark-to-market change in fair value of approximately $31.1 million associated with the Company’s common stock ownership in Melinta, guaranteed repayments and restructuring charges of $11.4 million. On a non-GAAP basis, adjusted loss(1) from continuing operations in the first half of 2018 was $102.6 million, or $1.40(1) per share, compared to a loss of $105.3 million, or $1.47(1) per share, in the first half of 2017.

First Half 2018 Financial Summary from Discontinued Operations

In the first quarter of 2018, the Company completed the sale of its infectious disease business, consisting of the products Vabomere, Orbactiv and Minocin IV, as well as line extensions of those products, for $270 million in upfront consideration and guaranteed payments, tiered royalty payments of between 5% to 25% on worldwide net sales of Vabomere, Orbactiv and Minocin IV, and the assumption by Melinta of all royalty, milestone and other payment obligations relating to those products.

(1) Adjusted net loss and adjusted loss per share from continuing operations are non-GAAP financial performance measures with no standardized definitions under U.S. GAAP. For further information and a detailed reconciliation, refer to the "Non-GAAP Financial Performance Measures" and "Reconciliations of GAAP to Adjusted Loss From Continuing Operations and Adjusted Loss per Share" sections of this press release.

Net income from discontinued operations in the first half of 2018 was $114.2 million, compared to a net loss of $58.9 million in 2017. Net income from discontinued operations in the first half of 2018 included a pre-tax gain of approximately $169.0 million from the sale of the Company’s infectious disease business to Melinta.

At June 30, 2018, the Company had $162.5 million in cash and cash equivalents, compared to $151.4 million at the end of 2017.

Second-Quarter 2018 Conference Call and Webcast Information

The Company will host a conference call and webcast today, August 1, 2018, at 8:30 a.m., Eastern Daylight Time, to discuss its second-quarter 2018 financial results and provide clinical and operational updates. The dial-in information to access the call is as follows:

U.S./Canada: (877) 359-9508
International: (224) 357-2393
Conference ID: 5847059
A taped replay of the conference call will be available from 11:30 a.m., Eastern Daylight Time, today until 11:30 p.m., Eastern Daylight Time, on August 8, 2018. The replay may be accessed as follows:

U.S./Canada: (855) 859-2056
International: (404) 537-3406
Conference ID: 5847059
The webcast can be accessed in the "Investors" section of The Medicines Company website. A replay of the webcast will also be available.

About Inclisiran

Inclisiran is an investigational GalNAc-conjugated RNA interference therapeutic, which inhibits the synthesis of PCSK9 protein in liver cells, thereby reducing liver cell LDL receptor turnover, and lowering plasma LDL-C.

The Medicines Company and Alnylam Pharmaceuticals, Inc. are collaborating in the advancement of inclisiran pursuant to their 2013 agreement. Under the terms of the agreement, Alnylam completed certain pre-clinical studies and the Phase I clinical study, with The Medicines Company leading and funding the development of inclisiran from Phase II forward, as well as potential commercialization.

On August 1, 2018 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology, reported its financial results and operational progress for the second quarter of 2018 (Press release, Intellia Therapeutics, AUG 1, 2018, View Source [SID1234528392]).

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"In the first half of 2018, we focused on executing against our priorities, including initiating IND-enabling studies for our lead in vivo liver program, demonstrating that our lipid nanoparticle delivery platform has broad utility, and advancing our ex vivo R&D efforts across our key collaborations. We are excited about our progress, and we remain focused on achieving our 2018 goals," said Intellia President and Chief Executive Officer John Leonard, M.D.

Second Quarter 2018 Operational Highlights

The Company achieved several key operational milestones during the second quarter of 2018, including the following:

Intellia continued progress in non-human primate (NHP) dose-ranging studies that support the Company’s lead in vivo program in transthyretin amyloidosis (ATTR).

At the 21st Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in Chicago, Intellia and research collaborator, Ospedale San Raffaele, announced the identification of T cell receptors (TCRs) targeting a Wilms’ Tumor 1 protein (WT1) epitope, important for the treatment of acute myeloid leukemia as Intellia’s first ex vivo program. TCRs that target WT1 may also have applicability in solid tumors and other

hematological malignancies, as WT1 is over-expressed on many tumor types. The Company plans to develop these TCRs as part of its first ex vivo product candidate. Intellia anticipates that this program will benefit from ongoing work to develop a truly allogeneic approach for engineered cellular therapy.

In the second quarter, Intellia progressed ex vivo multiplexing efforts to achieve triple knockout edits with greater than 80 percent efficiency in human cells, and observed insertion efficacy of ~50 percent with simultaneous double knockout edits. Capabilities in multiplexing will support and advance ex vivo efforts within the Company’s expanding engineered cell therapy pipeline.

Expanding on the in vivo liver editing achievements announced earlier this year, the Company progressed its primary hyperoxaluria type I (PH1) program utilizing a phenotypic mouse model of the disease. In PH1, excess oxalate produced in the liver crystallizes and accumulates in various organs eventually causing kidney failure. A knockout of the HAO1 gene reduces levels of glyoxylate, a precursor to urinary oxalate, and thereby reduces oxalate accumulation. In a mouse model of the disease, the Company achieved 74 percent editing of HAO1 leading to a ~90 percent protein reduction and a ~55 percent reduction in urinary oxalate after a single dose. This progress reinforces the value and speed of Intellia’s modular lipid nanoparticle delivery platform, including efficient and effective delivery to hepatocytes in the liver.

Intellia continued to expand its fully automated, next-generation sequencing and bioinformatics platform to support the identification of highly active guides with limited to no off-target cutting or unforeseen deletions. In the second quarter, Intellia increased throughput capacity to process greater than 30,000 sequencing samples per week. This capability enables measuring of on- and off-target genome editing, including indels, translocations, excisions and inversions.

Intellia, along with other licensees, announced in June that the U.S. Patent and Trademark Office (USPTO) granted U.S. Patent No. 10,000,772 ("the ’772 patent") to The Regents of the University of California, the University of Vienna and Emmanuelle Charpentier, Ph.D. (collectively, "UC"), co-owners of foundational intellectual property relating to CRISPR/Cas9 genome editing technology. The ’772 patent covers the use of single- and dual-guide RNA formats having certain structural motifs in a region that interacts with the Cas9 enzyme. These guide RNA formats are widely used in the CRISPR/Cas9 field. Intellia anticipates this is the first of many patents to be granted in the U.S. to UC for the CRISPR/Cas9 genome editing intellectual property. Outside of the U.S., UC continues to hold a strong global intellectual property position with applications from this patent estate issued in Europe, the United Kingdom, China, Japan, Australia and various other countries worldwide. The ’772 patent is not involved in the appeal to the U.S. Court of Appeals for the Federal Circuit (CAFC) relating to the February 2017 interference decision from the USPTO’s Patent Trial and Appeal Board. UC’s appeal was heard on April 30, 2018 by the CAFC, and a decision is still pending.

Upcoming Milestones

For the remainder of 2018, Intellia’s expected milestones include the following:

Advance a second liver knockout target in NHPs;

Advance candidates for a second liver indication;

Present additional editing data supporting more complex edits such as insertion and repair;

Prepare for a pre-Investigational New Drug meeting with the U.S. Food and Drug Administration for ATTR;

Expand preclinical data in support of Intellia’s first proprietary ex vivo autoimmune program; and

Identify Intellia’s first hematopoietic stem cell target from the collaboration with Novartis.

Second Quarter 2018 Financial Results

Collaboration Revenue

Collaboration revenue was $7.7 million for the second quarter of 2018, compared to $5.9 million during the second quarter of 2017. The increase in collaboration revenue in 2018 was primarily driven by amounts recognized under Intellia’s collaboration agreement with Regeneron.

Since inception through June 30, 2018, the Company has received $114.1 million in funding from the collaborations with Novartis and Regeneron, excluding amounts received for equity investments, and had an accounts receivable balance of $8.6 million on June 30, 2018.

Operating Expenses

Research and development expenses increased by $7.9 million to $23.5 million during the second quarter of 2018, compared to $15.6 million during the second quarter of 2017. This increase was driven primarily by the advancement of Intellia’s research programs, research personnel growth to support these programs, as well as the expansion of the development organization, and includes laboratory supplies and research materials such as reagents.

General and administrative expenses increased by $1.4 million to $7.8 million during the second quarter of 2018, compared to $6.4 million during the second quarter of 2017. This increase was driven primarily by increased salary and related headcount-based expenses to support Intellia’s larger research and development organization, public company compliance, and administrative obligations.

The Company’s net loss was $22.2 million for the second quarter of 2018, compared to $15.6 million during the second quarter of 2017.

Cash and cash equivalents at June 30, 2018, were $305.5 million, compared to $241.0 million for second quarter in 2017.

Financial Guidance

The Company’s primary uses of capital will continue to be for research and development programs, laboratory and related supplies, compensation costs for current and future employees, consulting, intellectual property related costs, and general operating costs.

As of June 30, 2018, the Company had an accumulated deficit of $159.3 million. The Company expects losses to increase as it continues to incur significant research and development expenses related to the advancement of Intellia’s therapeutic programs and ongoing operations. Based on Intellia’s research and development plans and expectations related to the progress of the Company’s programs, the Company expects that the cash and cash equivalents as of June 30, 2018, as well as technology access and research funding from Novartis and Regeneron, will enable Intellia to fund operating expenses and capital expenditures through mid-2020, excluding any potential milestone payments or extension fees that could be earned and distributed under the collaboration agreements with Novartis and Regeneron or any strategic use of capital not currently in the base-case planning assumptions.

Upcoming Events During the Third Quarter 2018

The Company expects to make presentations at the following upcoming investor conferences:

B. Riley FBR Health Care Conference, Sept. 4, New York City

Citi Biotech Conference, Sept. 5, Boston

Wells Fargo Health Care Conference, Sept. 6, Boston

Jefferies Gene Therapy Summit, Sept. 27, New York City

On August 1, 2018 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to quizartinib, an investigational FLT3 inhibitor, for the treatment of adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML) (Press release, Daiichi Sankyo, AUG 1, 2018, View Source [SID1234528390]).

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"There have been limited advances over the past several decades for the treatment of relapsed/refractory FLT3-ITD AML, a very aggressive form of the disease associated with poor prognosis. Quizartinib is the first FLT3 inhibitor to significantly improve overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory AML with FLT3-ITD, an underlying driver of this subtype of AML," said Arnaud Lesegretain, Vice President, Oncology Research and Development and Head, AML Franchise, Daiichi Sankyo. "We are excited that quizartinib has received Breakthrough Therapy designation and we look forward to working closely with the FDA to bring this potential new treatment option to patients as quickly as possible."

Breakthrough Therapy designation is designed to expedite the development and regulatory review of medicines that may demonstrate substantial benefit over currently approved treatments, in order to more quickly bring new treatment options to patients with serious diseases. Significant unmet medical need exists in relapsed/refractory AML, as available treatment options are limited and there are no approved targeted therapies for patients with relapsed/refractory FLT3-ITD AML.

The designation was granted based on the results of the pivotal phase 3 QuANTUM-R study of quizartinib, which were presented during the plenary program at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June 2018. QuANTUM-R is the first randomized phase 3 study to show that a FLT3 inhibitor, quizartinib, prolongs overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory FLT3-ITD AML.

The safety profile observed in QuANTUM-R appears consistent with that observed at similar doses in the quizartinib clinical development program. Incidence of treatment-emergent adverse events was comparable between patients who received single agent quizartinib (n=241) and those who received salvage chemotherapy (n=94). The most common adverse events (>30 percent, any Grade) in patients treated with quizartinib included nausea, thrombocytopenia, fatigue, musculoskeletal pain, pyrexia, anemia, neutropenia, febrile neutropenia, vomiting and hypokalemia.

About Quizartinib
Quizartinib, the lead investigational agent in the AML Franchise of the Daiichi Sankyo Cancer Enterprise, is an oral selective FLT3 inhibitor currently in phase 3 development for relapsed/refractory (QuANTUM-R) and newly-diagnosed (QuANTUM-First) FLT3-ITD AML in the U.S., EU and Japan, and phase 2 development for relapsed/refractory FLT3-ITD AML in Japan.

In addition to Breakthrough Therapy designation, quizartinib has been granted Fast Track designation by the FDA for the treatment of relapsed/refractory AML. Quizartinib also has been granted Orphan Drug designation by both the FDA and the European Medicines Agency (EMA) for the treatment of AML. Quizartinib is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.

About FLT3-ITD Acute Myeloid Leukemia
AML is an aggressive blood and bone marrow cancer that causes uncontrolled growth and accumulation of malignant white blood cells that fail to function normally and interfere with the production of normal blood cells.1 In the U.S. this year, it is estimated that there will be more than 19,000 new diagnoses of AML and more than 10,000 deaths from AML.2 The five-year survival rate of AML reported from 2005 to 2011 was approximately 26 percent, which was the lowest of all leukemias.1

FLT3 gene mutations are one of the most common genetic abnormalities in AML.3 FLT3-ITD is the most common FLT3 mutation, affecting approximately one in four patients with AML.4,5,6,7 Patients with FLT3-ITD AML have a worse overall prognosis, including an increased incidence of relapse, an increased risk of death following relapse and a higher likelihood of relapse following hematopoietic stem cell transplantation as compared to those without this mutation.8,9