On May 14, 2019 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported its financial results for the first quarter ended March 31, 2019 (Press release, Curis, MAY 14, 2019, View Source [SID1234536294]).

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"2019 has been a transformational year for Curis as we demonstrate the results of our heightened focus on clinical execution. We are currently on track or ahead of schedule in the execution of all three of our clinical trials," said James Dentzer, President and Chief Executive Officer of Curis. "We look forward to discussing clinical data from all three trials later this year: this summer for CA-4948 and later in the second half for fimepinostat and CA-170."

"Furthermore, with this quarter’s agreement with Oberland Capital, for $65 million upfront and up to $70.7 million in additional milestones, we secured the financial flexibility needed to ensure we can continue to move forward aggressively in our clinical execution of all three programs," he concluded.

First Quarter 2019 Financial Results

Curis reported a net loss of $9.9 million, or $0.30 per share on both a basic and diluted basis for the first quarter of 2019, as compared to a net loss of $10.7 million, or $0.33 per share on both a basic and diluted basis for the same period in 2018.

Revenues for the first quarter of 2019 were $1.8 million, as compared to $2.5 million for the same period in 2018. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses were $7.3 million for the first quarter of 2019, as compared to $12.4 million for the same period in 2018, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.1 million for both the first quarter of 2019 and 2018.

Research and Development Expenses. Research and development expenses were $4.1 million for the first quarter of 2019 as compared to $8.3 million for the same period in 2018. The decrease was primarily due to decreased costs related to ongoing clinical and manufacturing activities for fimepinostat and CA-170. Employee-related expenses decreased from the prior quarter primarily due to a reduction in headcount that occurred in the fourth quarter of 2018. These changes reflect our shift in focus toward clinical development, while down-sizing the in-house discovery research organization. We have also focused the CA-170 program toward the VISTA-expressing mesothelioma indication, which allowed substantial reduction in the number of sites and regions required.

General and Administrative Expenses. General and administrative expenses were $3.1 million for the first quarter of 2019 as compared to $4.0 million for the same period in 2018. The decrease in general and administrative expenses was driven primarily by lower personnel, legal, and stock-based compensation for the period.

Other expense, net was $4.3 million for the first quarter of 2019, as compared to $0.8 million for the same period in 2018. Other expense, net primarily consisted of the loss on extinguishment of $3.5 million and interest expense of $0.8 million related to Curis Royalty’s (a wholly owned subsidiary of Curis) debt obligations.

As of March 31, 2019, Curis’s cash, cash equivalents and investments totaled $42.8 million and there were approximately 33.2 million shares of common stock outstanding.

Recent Operational Highlights

Precision oncology, fimepinostat (HDAC/PI3K inhibitor):

Curis is initiating a study of fimepinostat (a MYC suppressor) with venetoclax (a BCL-2 inhibitor) combination regimen in diffuse large B-cell lymphoma (DLBCL), including patients with Double-Hit/Double-Expressor Lymphoma. In preclinical models, fimepinostat administered in combination with venetoclax resulted in an enhanced benefit relative to each agent alone.

Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):

In April 2019, Curis advanced to the 200mg BID cohort in the CA-4948 study for treatment of patients with non-Hodgkin lymphoma, including those with MYD88 alterations.

Immuno-oncology, CA-170 (VISTA / PDL1 antagonist; Aurigene collaboration):

In January 2019, Curis dosed the first mesothelioma patient in its ongoing Phase 1 CA-170 trial following evidence supporting high levels of VISTA expression in mesothelioma tumor samples. Recent publications have identified VISTA as a possible resistance mechanism to treatment with anti-PD1 antibodies in several cancer indications.

In May 2019, Curis announced completion of its target enrollment of mesothelioma patients in the ongoing Phase 1 CA-170 trial.

First Quarter 2019 and Recent Corporate Highlights

In March 2019, Curis announced that it sold a portion of its Erivedge royalties to Oberland Capital for up to $135.7 million, including $65 million upfront.

2019 Data Catalysts

For the remainder of the year, Curis expects to:

Report initial data on the combination of fimepinostat and venetoclax regimen in patients with R/R DLBCL, including patients with DH/DE Lymphoma, in the second half of 2019.

Report initial efficacy data from its CA-4948 dose escalation study in patients with NHL in mid-year 2019.

Report initial efficacy data from its CA-170 Phase 1 trial in patients with mesothelioma (high VISTA expressors) in the second half of 2019.

Conference Call Information

Curis management will host a conference call today, May 14, 2019, at 4:30 p.m. EDT, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412 317-5252 from other locations, shortly before 4:30 p.m. EDT. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.

On May 14, 2019 Agilent Technologies, Inc. (NYSE: A) reported revenue of $1.24 billion for the second quarter ended April 30, 2019, up 3 percent year-over-year (up 4 percent on a core basis(1)) (Press release, Agilent, MAY 14, 2019, https://www.agilent.com/about/newsroom/presrel/2019/14may-gp19011.html [SID1234536293]).

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On a GAAP basis, second-quarter net income was $182 million or $0.57 per share. This compares with $205 million and $0.63 per share in the second quarter of 2018. Non-GAAP net income(2) was $228 million or $0.71 per share during the quarter, compared with $212 million and $0.65 per share during the second quarter a year ago.

"While overall revenues were below our expectations, the story of our second quarter results is one where we demonstrated the resilience of Agilent’s business model," said Mike McMullen, Agilent president and CEO. "Two of our three business units continued to deliver strong growth while the third was affected by soft market conditions. We generated EPS of $0.71, representing 9 percent growth, which was at the midpoint of our guidance."

Financial Highlights

Life Sciences and Applied Markets Group

Second-quarter revenue of $529 million from Agilent’s Life Sciences and Applied Markets Group (LSAG) was down 1 percent year over year (down 1 percent on a core basis(1)). Demand in the environmental and forensics markets was strong, offset by weakness in the pharma and food markets. LSAG’s operating margin for the quarter was 20.3 percent.

Agilent CrossLab Group

Second-quarter revenue of $455 million from the Agilent CrossLab Group (ACG) grew 7 percent year over year (up 9 percent on a core basis(1)). Growth was broad-based across all regions, led by China. ACG’s operating margin for the quarter was 25.2 percent.

Diagnostics and Genomics Group

Second-quarter revenue of $254 million from Agilent’s Diagnostics and Genomics Group (DGG) grew 5 percent year over year (up 6 percent on a core basis(1)). Strength in the company’s pathology-related businesses and NASD led the group’s results. DGG’s operating margin for the quarter was 19.3 percent.

Third-Quarter and Full-Year Outlook

Agilent expects third-quarter 2019 revenue in the range of $1.225 billion to $1.245 billion. Third-quarter 2019 non-GAAP earnings are expected to be in the range of $0.71 to $0.73 per share(3).

For fiscal year 2019, the company is revising its full-year revenue guidance to a range of $5.085 billion to $5.125 billion while maintaining non-GAAP earnings guidance in the range of $3.03 to $3.07 per share(3).

Conference Call

Agilent’s management will present more details about its second-quarter fiscal year 2019 financial results on a conference call with investors today at 1:30 p.m. (Pacific Time). This event will be webcast live in listen-only mode. Listeners may log on at www.investor.agilent.com and select "Q2 2019 Agilent Technologies Inc. Earnings Conference Call" in the "News & Events — Calendar of Events" section. The webcast will remain available on the company’s website for 90 days.

Additional information regarding financial results can be found at www.investor.agilent.com by selecting "Financial Results" in the "Financial Information" section.

A telephone replay of the conference call will be available at approximately 4:30 p.m. (Pacific Time) after the call on May 14 and through May 21 by dialing (855) 859-2056 (or (404) 537-3406 from outside the United States) and entering passcode 3086626.

On May 14, 2019 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that the U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) voted (Vote: 12 yes, 3 no, zero abstained) that the demonstrated benefit of pexidartinib outweighs the risks in the treatment of adult patients with symptomatic TGCT, which is associated with severe morbidity or functional limitations, and which is not amenable to improvement with surgery (Press release, Daiichi Sankyo, MAY 14, 2019, View Source [SID1234536292]).

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"Today’s vote in favor of pexidartinib marks a significant step toward delivering the first approved systemic therapy for select TGCT patients whose disease is not amenable to improvement with surgery," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. "Some people living with TGCT experience debilitating symptoms and need innovative treatment options. We believe that pexidartinib has the potential to help address this need by offering carefully selected TGCT patients an important treatment advancement, and we look forward to working with the FDA as it completes its review of our application."

The New Drug Application (NDA) for pexidartinib is currently under Priority Review in the U.S., and the FDA is expected to decide whether to approve the application by the PDUFA date of August 3, 2019. The FDA will consider today’s vote as it reviews the NDA, although it is not obligated to follow the Committee’s recommendation. The NDA submission is based on the results of the pivotal phase 3 ENLIVEN study of oral pexidartinib, the first placebo-controlled study of a systemic investigational therapy in patients with TGCT.

The ENLIVEN study met its primary endpoint of tumor response rate by RECIST, which was 39 percent in pexidartinib-treated patients and zero percent for placebo-treated patients at week 25 (p <0.0001). In the ENLIVEN study, hepatic toxicities were more frequent with pexidartinib versus placebo (aspartate aminotransferase [AST] or alanine aminotransferase [ALT] ≥3X the upper limit of normal [ULN]: 33 percent, total bilirubin ≥2X ULN: 5 percent, N=61). In the randomized Part 1 of the study, eight (13%) patients discontinued pexidartinib due to adverse events (AEs); one discontinuation was due to hypertension and seven were due to liver-related AEs occurring within the first two months of treatment. Of the liver-related AEs, three were serious nonfatal AEs with increased bilirubin, one lasting ~7 months. In non-TGCT development studies using pexidartinib, two severe liver toxicity cases (one required liver transplant, one was associated with death) were observed.

About TGCT (PVNS/GCT-TS)
Tenosynovial giant cell tumor (TGCT), also referred to as pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS), is a rare, non-malignant tumor that can be locally aggressive. TGCT affects the synovium-lined joints, bursae, and tendon sheaths, resulting in swelling, pain, stiffness and reduced mobility in the affected joint or limb.[1],[2],[3]

While the exact incidence of TGCT is not known, it is estimated that the incidence of TGCT is 11 to 50 cases per million person-years, based on studies from three countries.[4],[5],[6] TGCT is subcategorized into two types: localized, which is more common and accounts for 90 percent of cases, and diffuse, which accounts for 10 percent of cases.5,6 The current standard of care for TGCT is surgical resection.1,2 However, in patients with a recurrent, difficult to treat, or diffuse form where the tumor can wrap around bone, tendons, ligaments and other parts of the joint, it is more difficult to remove or might not be amenable to improvement with surgery due to the risk of morbidity and potential recurrence. Additional surgeries for more severe cases can lead to significant joint damage, debilitating functional impairments, and reduced quality of life and amputation may be considered.[7],[8],[9]

Recurrence rates for localized TGCT are estimated to be up to 15 percent following complete resection.2,[10],[11],[12] Diffuse TGCT recurrence rates are estimated to be about 20 percent to 50 percent following complete resection.3,10,[13] TGCT affects all age groups; the diffuse type on average occurs most often in people below the age of 40 and the localized type typically occurs in people between 30 and 50 years old.1,4,5,6

About Pexidartinib
Pexidartinib is an investigational, novel, oral small molecule that potently inhibits CSF1R (colony stimulating factor-1 receptor), which is a primary growth driver of abnormal cells in the synovium that cause TGCT. Pexidartinib also inhibits c-kit and FLT3-ITD. Pexidartinib was discovered by Plexxikon Inc., the small molecule structure-guided R&D center of Daiichi Sankyo.

Pexidartinib is currently under regulatory review with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT), which is associated with severe morbidity or functional limitations, and which is not amenable to improvement with surgery. In addition to Priority Review designation, pexidartinib has been granted Breakthrough Therapy designation for the treatment of patients with pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS), where surgical resection may result in potentially worsening functional limitation or severe morbidity, and Orphan Drug designation for PVNS/GCT-TS by the FDA. Pexidartinib also has received Orphan Drug designation from the European Commission for the treatment of TGCT. Pexidartinib is an investigational compound that has not been approved for any indication in any country. Safety and efficacy have not been established.

About Daiichi Sankyo Cancer Enterprise
The mission of Daiichi Sankyo Cancer Enterprise is to leverage our world-class, innovative science and push beyond traditional thinking to create meaningful treatments for patients with cancer. We are dedicated to transforming science into value for patients, and this sense of obligation informs everything we do. Anchored by three pillars including our investigational Antibody Drug Conjugate Franchise, Acute Myeloid Leukemia Franchise and Breakthrough Science, we aim to deliver seven distinct new molecular entities over eight years during 2018 to 2025. Our powerful research engines include two laboratories for biologic/immuno-oncology and small molecules in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in Berkeley, CA. Compounds in pivotal stage development include: [fam-] trastuzumab deruxtecan, an antibody drug conjugate (ADC) for HER2 expressing breast, gastric and other cancers; quizartinib, an oral selective FLT3 inhibitor, for newly-diagnosed and relapsed/refractory FLT3-ITD acute myeloid leukemia (AML); and pexidartinib, an oral CSF1R inhibitor, for tenosynovial giant cell tumor (TGCT). For more information, please visit: www.DSCancerEnterprise.com.

On May 14, 2019 Phio Pharmaceuticals Corp. (NASDAQ: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported its financial results for the first quarter ended March 31, 2019 and provided a business update (Press release, Phio Pharmaceuticals, MAY 14, 2019, View Source [SID1234536286]).

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"Our financial results during the first quarter of the year continue to remain consistent and in line with our projections for 2019. With an approximate $2 million quarterly cash burn expected to continue for the remainder of the year, the Company expects to have funding into the second half of 2020 while also providing us with the ability to significantly advance and expand our pipeline projects. In parallel with progressing our lead pipeline products, we have expanded our collaborations to include a collaboration with Glycostem Therapeutics and have increased our data output supporting the broad applicability of our self-delivering RNAi platform in various immune cell types and immuno-oncology applications, such as adoptive cell therapy and direct intra-tumoral use of our products," said Dr. Gerrit Dispersyn, President and CEO of Phio Pharmaceuticals Corp. "By bringing new talent on board and implementing improved R&D processes, we look forward to further accelerating our R&D efforts within the financial expectations and projected cash runway."

Quarter in Review and Recent Corporate Updates

Leadership:
Appointed Gerrit Dispersyn, Dr. Med. Sc. as the Company’s President and Chief Executive Officer. Dr. Dispersyn succeeded Geert Cauwenbergh, Dr. Med. Sc., who retired as CEO of the Company and remains as a member of the Company’s Board of Directors.
Appointed John A. Barrett, Ph.D. as the Company’s Chief Development Officer. Dr. Barrett joins Phio from Ziopharm Oncology, Inc. where he served as the company’s Vice President of R&D and Translational Medicine. He has accumulated over 25 years of experience working in research and development and is an expert in developing cell-based immuno-oncology therapies. Dr. Barrett will lead the development and execution of the Company’s preclinical and clinical strategy for our product candidates.
Entered into a research collaboration with Glycostem Therapeutics BV to explore the potential synergies of using our sd-rxRNA in combination with Glycostem’s proprietary Natural Killer-cell (NK-cell) generation technology (oNKord). The goal of the collaboration is to develop cellular immunotherapies for cancer treatment with enhanced efficacy and/or safety, resulting in further improvement of Glycostem’s cellular immunotherapies for the treatment of cancer patients.
Presented a poster titled "Feasibility and efficacy using self-delivering RNAi against TGFB1 to reduce TME immunosuppression" at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019.
Data from the poster demonstrated that an sd-rxRNA targeting TGFB1 was efficiently taken up by cancer and immune cells resulting in downregulation of target gene expression. The data further showed that intra-tumoral injection of sd-rxRNA targeting TGFB1 can reduce the immunosuppressive tumor microenvironment and potentially boost immune effector cell activity. Future clinical development activities will explore the possible synergistic effects of this approach in combination with adoptive cell therapy or other oncology therapies.
On May 14, 2019, the Nasdaq Stock Market provided written notice and granted the Company an additional 180 calendar days, or until November 11, 2019, to regain compliance with the minimum bid price requirements set forth in the Nasdaq listing rules. The written notice has no effect on the listing of the Company’s common stock at this time.
Select Financial Results

Cash Position

At March 31, 2019, the Company had cash of $12.7 million as compared with $14.9 million at December 31, 2018. The Company expects its cash to provide funding into the second half of 2020.

Research and Development Expenses

Research and development expenses for the quarter ended March 31, 2019 were $1.1 million as compared with $1.4 million for the quarter ended March 31, 2018. The decrease was primarily due to a reduction in headcount and the payroll-related expenses, as well as the completion of the Company’s drug manufacture of RXI-762 in the prior year period.

General and Administrative Expenses

General and administrative expenses for the quarter ended March 31, 2019 were $1.1 million as compared with $0.9 million for the quarter ended March 31, 2018. The increase was primarily due to an increase in stock-based compensation expense related to the restricted stock issued to the Company’s former Chief Executive Officer in lieu of cash compensation.

Net Loss

Net loss for the quarter ended March 31, 2019 was $2.1 million, compared with $2.2 million for the quarter ended March 31, 2018. The decrease was primarily due to changes in operating expenses, as discussed above

On May 14, 2019 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported the outcome of the U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) to discuss the company’s New Drug Application (NDA) for quizartinib for the treatment of adults with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML) (Press release, Daiichi Sankyo, MAY 14, 2019, https://www.prnewswire.com/news-releases/daiichi-sankyo-announces-outcome-of-fda-oncologic-drugs-advisory-committee-meeting-to-review-flt3-inhibitor-quizartinib-for-the-treatment-of-patients-with-relapsedrefractory-flt3-itd-aml-300850274.html [SID1234536285]).

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Three committee members voted yes and 8 committee members voted no when asked if the results from the pivotal phase 3 QuANTUM-R study demonstrated that treatment with quizartinib provides a benefit that outweighs the safety risks for patients with relapsed/refractory FLT3-ITD AML.

"While we are disappointed by the outcome of today’s ODAC vote, we will work closely with the FDA as it completes the review of our submission," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. "Patients with relapsed/refractory FLT3-ITD AML are facing a very aggressive disease with poor prognosis, and we continue to believe that quizartinib could offer an important additional treatment option that specifically targets FLT3-ITD, a driver mutation in AML."

The FDA is not bound by the recommendations of the advisory committee. The NDA for quizartinib is currently under Priority Review in the U.S., and the FDA is expected to make a decision on approval by the Prescription Drug User Fee Act (PDUFA) date of August 25, 2019. The NDA submission of quizartinib is based on the results of the pivotal phase 3 QuANTUM-R study, which was the first randomized phase 3 study to show that a FLT3 inhibitor prolonged overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory FLT3-ITD AML.

In the quizartinib QuANTUM-R study, the median treatment duration with quizartinib was 4 cycles of 28 days each versus 1 cycle in the salvage chemotherapy arm. Incidence of treatment-emergent adverse events was comparable between patients who received single agent quizartinib and those who received salvage chemotherapy. The most common adverse drug reactions (>30 percent, any Grade) in patients treated with quizartinib included infections, bleeding, nausea, asthenic conditions, pyrexia, febrile neutropenia, and vomiting, and the most common Grade ≥ 3 adverse drug reactions (>20 percent) were infection and febrile neutropenia. The most common laboratory adverse reactions (incidence >50 percent) were decreased white blood cell count, decreased lymphocyte count, decreased hemoglobin, decreased neutrophil count, and decreased platelet count. QTcF >500 msec occurred in 8 patients (3.3 percent) and 2 out of 241 patients discontinued quizartinib due to QTcF prolongation. There were no reported events of Grade 4 QTcF prolongation (Torsades de Pointes, sudden death or cardiac arrest) in the quizartinib arm. The safety profile observed in QuANTUM-R appears consistent with that observed at similar doses in the quizartinib clinical development program.

About FLT3-ITD AML
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 2007 to 2013 was approximately 27 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 FLT3-ITD is a driver mutation that presents with high leukemic burden and has poor prognosis and a significant impact on disease management for patients with AML.5,8 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.9,10

About Quizartinib
Quizartinib, the lead investigational agent in the AML Franchise of the Daiichi Sankyo Cancer Enterprise, is an oral selective type II FLT3 inhibitor currently under regulatory review with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Japan Ministry of Health, Labour and Welfare (MHLW) for the treatment of adult patients with relapsed/refractory AML which is FLT3-ITD positive. Quizartinib has been granted Priority Review and Breakthrough Therapy designation for the treatment of adult patients with relapsed/refractory FLT3-ITD AML, and Fast Track designation for the treatment of relapsed/refractory AML by the FDA. Quizartinib also has been granted Orphan Drug designation by both the FDA and the European Commission (EC) for the treatment of AML and by the Japan MHLW for the treatment of FLT3-mutated AML.

Quizartinib is also in phase 3 development in combination with standard chemotherapy in newly-diagnosed FLT3-ITD AML (QuANTUM-First) in the U.S., EU and Japan; phase 1/2 development for pediatric and young adult relapsed/refractory FLT3-ITD AML in North America and the EU; and phase 1 development in combination with an investigational MDM2 inhibitor, milademetan, for relapsed/refractory FLT3-ITD AML and newly-diagnosed FLT3-ITD AML unfit for intensive chemotherapy in the U.S. and Japan.

Quizartinib and milademetan are investigational agents that have not been approved for any indication in any country. Safety and efficacy have not been established.