On November 10, 2015 FUJIFILM Corporation (President: Shigehiro Nakajima) reported to conduct a clinical trial of its anti-cancer drug FF-10101 in patients with relapsed or refractory acute myeloid leukemia (AML) in the United States next year (Press release, Fujifilm, NOV 10, 2015, View Source [SID:1234508186]). FF-10101 is a new drug candidate discovered by Fujifilm, tapping into its advanced technology to synthesize and design chemical compounds nurtured through the photographic film business.

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AML is a type of hematological malignancy and considered as a refractory disease. The malignant transformation of hematopoietic stem cells into leukemia cells followed by their abnormal growth in bone marrow inhibits normal hematopoietic functions, leading to cytopenia. Organ dysfunctions are also caused by infiltration of the leukemia cells out of the bone marrow. Fms-like tyrosine kinase 3 (FLT3)* is a protein involved in proliferation of hematopoietic cells. Approximately 30% of AML patients harbor FLT3 mutations such as internal tandem duplications (ITD)** and tyrosine kinase domain (TKD)*** mutations, which induce abnormal growth of leukemia cells. Currently, FLT3 inhibitors resulting to inhibit leukemia cell proliferation are being developed, some of which show a favorable efficacy against leukemia cells with FLT3-ITD mutation. However, the TKD mutations are generally known to cause drastic decrease of efficacies of the FLT3 inhibitors.

FF-10101 is a FLT3 inhibitor which binds to an amino acid in FLT3 irreversibly. FF-10101 demonstrated a high efficacy in reducing leukemia cells with the ITD or TKD mutation in a preclinical mouse model, hence the promising efficacy in clinical trials is expected. The results of the preclinical study will be presented at the 57th annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), the world’s largest hematology meeting, in Orlando, Florida the United States in December this year.

The development of FF-10101 was adopted as Next generation Technology Program (NexTEP) by the Japan Science and Technology Agency, which is in collaboration with Professor Hitoshi Kiyoi, M.D., Ph.D., Nagoya University, to investigate the efficacy and the safety profile of FF-10101. Findings from the research will be applied to the clinical study in an effort to further accelerate the development of FF-10101.

Fujifilm is defining oncology as its focal area and promoting the R&D of anti-cancer drugs by combining the technologies and experiences including chemical synthesis capacity, design ability, analysis technology accumulated through the development and production of photographic film. Fujifilm has initiated a Phase I clinical trial of FF-10501 in patients with relapsed or refractory myelodysplastic syndromes (MDS)*4. The company will keep focusing on unmet medical needs in fields including oncology and actively promote R&D to expand business deployment and supply of innovative pharmaceutical products so as to contribute to resolving social issues.

On November 10, 2015 Delcath Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, reported financial results for the three and nine months ended September 30, 2015 (Press release, Delcath Systems, NOV 10, 2015, View Source;p=RssLanding&cat=news&id=2110968 [SID:1234508184]).

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Financial and other highlights for the third quarter of 2015 and recent weeks included:

84% increase in total revenue compared with the third quarter of 2014
23% decrease in total operating expenses compared with the third quarter of 2014
Establishment of national reimbursement coverage for CHEMOSAT procedures in Germany
Treatment of first patient in the intrahepatic cholangiocarcinoma (ICC) cohort of the Global Phase 2 HCC/ICC trial program
Acceptance for publication of prior Melanoma Phase 3 trial results in the Annals of Surgical Oncology
Presentation of data supporting Delcath’s Hepatic CHEMOSAT Delivery System at the CIRSE, ECCO and EADO annual meetings
Appointment of Jennifer Simpson, Ph.D., MSN, CRNP, President and CEO of Delcath to the Company’s Board of Directors
"Our performance in the third quarter and recent weeks was strong, with significant achievements in all of our commercial and clinical priorities," said Dr. Simpson. "Highlighting the period was the establishment of ZE reimbursement for CHEMOSAT procedures in Germany beginning in 2016, which represents our first national reimbursement mechanism and an important step towards increasing commercialization of CHEMOSAT in Europe. We also continued to drive commercial adoption of CHEMOSAT in other European markets, as evidenced by a nearly 84% increase in third quarter revenue compared with last year. In fact, our year-to-date revenue has already exceeded full-year 2014 revenue. This performance was achieved while maintaining disciplined expense management, which allowed us to beat our operating expense guidance of $4-5 million per quarter."

"During the period we also continued to advance our clinical development program, with the first treatment performed in the ICC cohort of our Global Phase 2 HCC/ICC trial program in October and data supporting CHEMOSAT presented at three major medical conferences. Adding to this growing body of evidence will be data from our prior U.S. Phase 3 clinical trial, which will published in the Annals of Surgical Oncology in the coming weeks. Publication of these results will be an important tool that will enhance our efforts to expand reimbursement in certain European countries, and will also help increase awareness of the value of this therapy in Europe."

"We held a productive meeting with the U.S. Food and Drug Administration regarding our plans for a global pivotal Phase 3 clinical trial in patients with ocular melanoma (OM) that has metastasized to the liver, with overall survival as the primary endpoint. The dialogue was constructive and the FDA is working with Delcath to advance the initiation of this important trial."

"Our team is executing our plan and entirely focused on delivering value for shareholders. We look forward to continuing this momentum through to the end of 2015 and beyond," concluded Dr. Simpson.

Third Quarter Financial Results

Total revenue for the third quarter of 2015 of $0.4 million increased 83.9% from $0.2 million for the third quarter of 2014. Selling, general and administrative expenses during the third quarter were $2.3 million, a decrease of $2.2 million or 48.9% from $4.5 million for the same period in 2014.

Total operating expenses for the third quarter of 2015 decreased by 23.1% to $4.0 million from $5.2 million for the same period in 2014. This decrease reflects a reduction in severance and compensation-related expenses following significant workforce restructurings throughout 2014 and into 2015, as well as a reduction in facility expenses.

The Company recorded a net loss for the third quarter of 2015 of $2.4 million, or $0.12 per share, a decrease of $2.2 million or 47.8%, compared with a net loss of $4.6 million, or $0.48 per share, for the same period in 2014. This decrease is primarily due to a $1.2 million reduction in operating expenses, a $0.1 million improvement in gross profit and a $0.8 million change in the fair value of the warrant liability, a non-cash item.

Nine Month Financial Results

Total revenue for the first nine months of 2015 of $1.3 million increased 62.5% from $0.8 million for the first nine months of 2014. Selling, general and administrative expenses during the first nine months of 2015 were $7.8 million, a decrease of $5.2 million or 40.0% from $13.0 million for the same period in 2014.

Total operating expenses for the first nine months of 2015 decreased by 27.7% to $12.0 million from $16.6 million for the same period in 2014. This decrease reflects a reduction in severance and compensation-related expenses following significant workforce restructurings throughout 2014 and into 2015, as well as a reduction in facility expenses.

The Company recorded a net loss for the first nine months of 2015 of $9.6 million, or $0.67 per share, a decrease of $4.8 million or 33.3% compared with a net loss of $14.4 million, or $1.54 per share, for the same period in 2014. This decrease is primarily due to a $4.7 million reduction in operating expenses, a $0.4 million improvement in gross profit and a $0.2 million change in the fair value of the warrant liability, a non-cash item.

Balance Sheet Highlights

Cash and cash equivalents as of September 30, 2015 were $16.7 million, compared with $20.5 million as of December 31, 2014. During the first nine months of 2015, net cash used in operating activities was $12.2 million.

On November 10, 2015 The U.S. Food and Drug Administration reported that it approved Cotellic (cobimetinib) to be used in combination with vemurafenib to treat advanced melanoma that has spread to other parts of the body or can’t be removed by surgery, and that has a certain type of abnormal gene (BRAF V600E or V600K mutation) (Press release, , NOV 10, 2015, View Source [SID:1234508187]).
Melanoma is the most aggressive and dangerous form of skin cancer in the United States. It forms in the skin cells that develop the skin’s pigment and if not diagnosed early, the cancer is likely to spread to other parts of the body. The National Cancer Institute estimates that 73,870 Americans will be diagnosed with melanoma and 9,940 will die from the disease this year.

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"As we continue to advance our knowledge of tumor biology, we have learned that cancer cells have a remarkable ability to adapt and become resistant to targeted therapies. Combining two or more treatments addressing different cancer-causing targets may help to address this challenge," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "Today’s approval provides a new targeted treatment that, when added to vemurafenib, demonstrates greater benefit than vemurafenib alone in patients with BRAF mutation-positive melanoma."

Cotellic works by blocking the activity of an enzyme known as MEK, which is part of a larger signaling pathway. Abnormal activity of signaling pathways can lead to cancer. Cotellic prevents or slows cancer cell growth. Vemurafenib, marketed in the U.S. as Zelboraf, is a BRAF inhibitor that affects a different part of the same pathway and was approved in 2011 to treat patients with melanoma that has spread to other parts of the body or cannot be removed by surgery, whose tumors express a gene mutation called BRAF V600E, as detected by an FDA approved test. Health care providers should confirm the presence of BRAF V600 E or V600K mutation in their patients’ tumor specimens using one of the available FDA approved tests prior to starting treatment with Cotellic in combination with vemurafenib.

The safety and efficacy of Cotellic taken in combination with vemurafenib were demonstrated in a randomized clinical study of 495 patients with previously untreated, BRAF V600 mutation-positive melanoma that is advanced or cannot be removed by surgery. All study participants received vemurafenib and were then randomly selected to also take either Cotellic or a placebo. On average, patients taking Cotellic plus vemurafenib experienced a delay in the amount of time it took for their disease to worsen (approximately 12.3 months after starting treatment) compared to approximately 7.2 months after starting treatment for those taking vemurafenib only. In addition, patients taking Cotellic plus vemurafenib lived longer, with approximately 65 percent of patients alive 17 months after starting treatment as compared to half of those taking vemurafenib only. Additionally, 70 percent of those taking Cotellic plus vemurafenib experienced complete or partial shrinkage of their tumors, compared to 50 percent among those taking vemurafenib plus placebo.

The most common side effects of treatment with Cotellic in combination with vemurafenib are diarrhea, sensitivity to ultraviolet (UV) light (photosensitivity reaction), nausea, fever (pyrexia) and vomiting.

Cotellic may cause severe side effects including damage to the heart muscle (cardiomyopathy) or to other muscles (rhabdomyolysis), new skin tumors (primary cutaneous malignancies), eye disease (retinal detachment), severe skin rash, liver damage (hepatotoxicity), hemorrhage and severe skin rash due to increased sensitivity to sunlight (photosensitivity). People taking Cotellic should avoid sun exposure, wear protective clothing, and a broad spectrum ultraviolet A/ultraviolet B sunscreen to protect against sunburn. Women taking Cotellic should use effective contraception, as the medication can cause harm to a developing fetus.

Cotellic was reviewed under the FDA’s priority review program that provides for an expedited six-month review of drugs that, at the time the application was submitted, have the potential to be a significant improvement in safety or effectiveness in the treatment of a serious condition. Cotellic also received orphan drug designation, which provides incentives such as tax credits, user fee waivers and eligibility for orphan drug exclusivity to assist and encourage the development of drugs for rare diseases.

Cotellic and Zelboraf are both marketed by Genentech of San Francisco, California.

On November 10, 2015 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the third quarter of 2015 and provided an update on progress toward delivering upon its key 2015 corporate objectives and clinical development milestones (Press release, Exelixis, NOV 10, 2015, View Source;p=RssLanding&cat=news&id=2111224 [SID:1234508190]).

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Key Priorities and Corporate Updates

Following release of positive results from the pivotal METEOR trial, Exelixis is focused on expediting its regulatory submissions and augmenting its commercial infrastructure to support the potential launch of its lead compound, cabozantinib, in advanced renal cell carcinoma (RCC) in the United States. At the same time, in support of its collaboration partner, Genentech, a member of the Roche Group, Exelixis is rolling out its portion of the U.S. sales force promoting COTELLICTM (cobimetinib), a second Exelixis-discovered compound, following recent regulatory approvals for the compound in combination with vemurafenib for the treatment of BRAF V600 mutation-positive unresectable or metastatic melanoma in the United States and Switzerland.

Cabozantinib Highlights

METEOR Trial Delivers Positive Results in Advanced RCC; Cabozantinib Granted Breakthrough Therapy Designation. In July 2015, Exelixis announced that METEOR met its primary endpoint, demonstrating a statistically significant improvement in progression-free survival (PFS) for cabozantinib versus everolimus in a population of patients with advanced RCC who have experienced disease progression following treatment with at least one prior VEGFR tyrosine kinase inhibitor. Based on these data, the FDA granted Breakthrough Therapy Designation to cabozantinib as a potential treatment for patients with advanced RCC who have received one prior therapy. In September 2015, detailed data from METEOR were published in The New England Journal of Medicine and also presented during the Presidential Session I at the European Cancer Congress in Vienna, Austria.

Progress on U.S. and EU Regulatory Filings for Cabozantinib in Advanced RCC. Based on the data from the METEOR trial, in October 2015, Exelixis initiated the rolling submission of its New Drug Application (NDA) in the United States. Exelixis expects to complete the U.S. filing before the end of 2015. In the European Union, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has granted accelerated assessment to cabozantinib for advanced RCC. As a result, when filed, the company’s Marketing Authorization may be eligible for a 150-day review, versus the standard 210 days (excluding clock stops when written or oral information is requested from CHMP). Exelixis expects to complete the EU filing in early 2016.

Results from a Randomized Phase 2 Trial in First-Line RCC Expected in the First Half 2016. The randomized phase 2 trial comparing cabozantinb versus sunitinib in the treatment of first-line intermediate or poor risk RCC patients, CABOSUN, completed enrollment in early 2015 and results for the primary endpoint of PFS are now expected in the first half of 2016. CABOSUN is being conducted by The Alliance for Clinical Trials in Oncology as part of Exelixis’ collaboration with the National Cancer Institute’s Cancer Therapy Evaluation Program (NCI-CTEP).

Trial Underway Evaluating Cabozantinib with Immunotherapies. In July 2015, Exelixis’ collaborators at NCI-CTEP initiated a phase 1 trial of cabozantinib in combination with nivolumab alone, or in combination with nivolumab plus ipilimumab, in patients with genitourinary tumors, including bladder cancer and RCC. The primary endpoint of the trial is the determination of dose-limiting toxicities and a recommended phase 2 dose for the combinations. Exelixis believes that there is a strong rationale for combining cabozantinib with immunotherapies, including clinical evidence of cabozantinib’s ability to create a more immune-permissive environment, as well as preclinical data suggesting cabozantinib increases T-cell infiltration into tumors. Data from this trial could have relevance in other disease settings, including non-small cell lung cancer (NSCLC).

Cobimetinib Highlights

Regulatory Progress for COTELLIC in Europe, Including Approval in Switzerland and Positive Opinion Issued by the European Medicines Agency’s CHMP. On August 27, 2015, Exelixis announced that Swissmedic, the Swiss licensing and supervisory authority, approved COTELLIC for use in combination with vemurafenib as a treatment for patients with advanced melanoma. In September 2015, the European Medicines Agency’s CHMP adopted a positive opinion of Roche’s Marketing Authorization Application for COTELLIC in combination with vemurafenib for the treatment of BRAF V600 mutation-positive unresectable or metastatic melanoma. The European Commission is expected to release its final opinion by the end of 2015. Under the terms of the collaboration agreement with Roche and Genentech, Exelixis will receive royalties on sales of COTELLIC outside of the United States.

Positive Overall Survival Data for Cobimetinib in Combination with Vemurafenib in Advanced Melanoma. In October 2015, Exelixis announced that the phase 3 coBRIM trial of cobimetinib in combination with vemurafenib met its secondary endpoint of demonstrating a statistically significant and clinically meaningful increase in overall survival for patients with unresectable locally advanced or metastatic melanoma carrying the BRAF V600 mutation. These data will be the subject of a presentation at the Society for Melanoma Research 2015 Congress taking place in San Francisco, November 18-21, 2015.

Regulatory Approval for COTELLIC in the United States. Today, Exelixis announced that the U.S. FDA approved cobimetinib for use in combination with vemurafenib as a treatment for patients with BRAF V600 mutation-positive advanced melanoma. COTELLIC is expected to be available within two weeks. Exelixis is entitled to an initial equal share of U.S. profits and losses, which will decrease as sales increase, and shares in the U.S. sales and marketing costs, including co-promoting COTELLIC in the U.S.

Corporate Highlights

Key Hires in Medical Affairs, Sales, and Marketing. In September 2015, the company announced three high-level appointments to support the commercialization of cabozantinib and cobimetinib: William Berg, M.D. joined the company as Senior Vice President of Medical Affairs, Jonathan Berndt as Vice President of Sales, and Gregg Bernier as Vice President of Marketing.

Public Offering of Stock Raises Net Proceeds of Approximately $145.6 Million. In late July 2015, Exelixis launched and completed a public offering of common stock. The company issued 28,750,000 shares, including 3,750,000 shares issued under the underwriters’ 30-day option to buy shares, at a price to the public of $5.40 per share, receiving approximately $145.6 million in net proceeds after deducting the underwriting discount and other estimated offering expenses payable by Exelixis. Exelixis currently expects to use the net proceeds from the offering for general corporate purposes, including for clinical trials, build-out of commercial infrastructure, research and development, capital expenditures and working capital.

"Over the last few months, we have made significant strides with our lead development program, cabozantinib in advanced RCC, including receiving Breakthrough Therapy Designation from the FDA, initiating our rolling NDA submission and obtaining accelerated assessment status from the European Medicines Agency’s CHMP," said Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis. "At the same time, we have significantly strengthened our organization’s capabilities, including the addition of high-level personnel in medical affairs, sales, and marketing in advance of the potential commercialization of cabozantinib in advanced RCC."

Dr. Morrissey continued: "Moreover, after the third quarter closed, Exelixis achieved a major milestone when COTELLIC became the second medicine to emerge from our research and development organization that has received FDA approval. We are excited to embark on the launch of COTELLIC in the U.S., working closely with our partners Genentech and Roche to commercialize the product, including fielding one quarter of the U.S. sales force."

2015 Financial Guidance. The company is refining its operating expense guidance for the second six months of 2015. We expect second half operating expenses to be at the upper end of the previously indicated $80 million to $90 million range including approximately $10 million of incremental non-cash stock-based compensation expense related to the vesting of performance stock options tied to the read-out of METEOR top-line results. As a result, we anticipate that our full year 2015 operating expenses will be near the upper end of the previously-indicated $150 million to $160 million range.

Third Quarter 2015 Financial Results

Net revenues for the quarter ended September 30, 2015 were $9.9 million, compared to $6.3 million for the comparable period in 2014. Net revenues for the third quarter of 2015 consisted of $6.9 million net product revenue related to the sale of COMETRIQ and $3.0 million of contract revenues for a milestone payment received from Merck related to their worldwide license of our PI3K-delta program.

Research and development expenses for the quarter ended September 30, 2015 were $26.1 million, compared to $43.6 million for the comparable period in 2014. The decrease was primarily related to a net decrease in clinical trial costs related to COMET and METEOR, the company’s phase 3 trials in metastatic castration-resistant prostate cancer and advanced RCC, and to a lesser degree, decreases in personnel related expenses resulting from an overall reduction in headcount. Those decreases were partially offset by an increase in stock-based compensation expense due to performance-based stock-options that vested as a result of the positive top-line data received from METEOR.

Selling, general and administrative expenses for the quarter ended September 30, 2015 were $17.8 million, compared to $9.9 million for the comparable period in 2014. The increase was primarily related to stock-based compensation expense due to the vesting of performance-based stock-options as a result of the positive top-line data received from the METEOR trial and higher marketing expenses, including expenses for cobimetinib under the company’s collaboration agreement with Genentech. Those increases were partially offset by a decrease in facilities costs and consulting and outside services.

Other income (expense), net for the quarter ended September 30, 2015 was a net expense of ($11.8) million compared to ($11.0) million for the comparable period in 2014. The net expense is comprised primarily of interest expense which includes $6.9 million of non-cash expense related to the accretion of the discounts on both the 4.25% Convertible Senior Subordinated Notes due 2019 and the company’s indebtedness under the Deerfield Notes for the quarter ended September 30, 2015, as compared to $7.5 million for the comparable period in 2014.

Net loss for the quarter ended September 30, 2015 was ($47.6) million, or ($0.22) per share, basic, compared to ($62.6) million, or ($0.32) per share, basic, for the comparable period in 2014. The decreased net loss for the quarter was primarily due to decreases in research and development expenses and an increase in net revenues, partially offset by an increase in selling, general and administrative expenses.

Cash and cash equivalents, short- and long-term investments and short- and long-term restricted cash and investments totaled $282.1 million at September 30, 2015 compared to $242.8 million at December 31, 2014.

On November 10, 2015 Aptose Biosciences Inc. (Nasdaq:APTO) (TSX:APS), a clinical-stage company developing new therapeutics and molecular diagnostics that target the underlying mechanisms of cancer, reported two collaborations that will provide exclusive access to new epigenetic therapeutics for the Company’s oncology pipeline (Press release, Aptose Biosciences, NOV 10, 2015, View Source;p=irol-newsArticle&ID=2110973 [SID:1234508436]). These partnerships have been strategically formed to leverage Aptose’s scientific and clinical expertise in cancer and hematologic diseases to develop mechanistically differentiated and high-value epigenetic drug candidates.

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Strategic Collaboration with Moffitt Cancer Center

Aptose has entered into a definitive agreement with Moffitt Cancer Center for exclusive global rights to potent, multi-targeting, single-agent inhibitors for the treatment of hematologic and solid tumor cancers. These small molecule agents are highly differentiated inhibitors of the Bromodomain and Extra-Terminal motif (BET) protein family members, which simultaneously target specific kinase enzymes. The molecules developed by Moffitt exhibit single-digit nanomolar potency against the BET family members and specific oncogenic kinases which, when inhibited, are synergistic with BET inhibition. Under the agreement, Aptose will gain access to the drug candidates developed by Moffitt and the underlying intellectual property covering the chemical modifications enabling potent bromodomain (BRD) inhibition on the chemical backbone of a kinase inhibitor. Aptose expects lead clinical candidates to emerge from the collaboration by late 2016.

Transcriptional dysregulation in cancer cells may occur through various means, including chromatin remodeling, histone modification and super-enhancer formation. The bromodomain proteins play a critical role in this dysregulation, and hence targeting specific bromodomains represents a validated treatment approach for various cancers. Aptose is committed to developing a pipeline of molecules that inhibit key epigenetic targets with the potential to intervene in oncogenesis and induce remission.

"We’ve built an oncology drug development organization with valuable ties to leading clinical centers and thought leaders," said William G. Rice, Ph.D., Chairman, President and CEO, "and we are exceptionally pleased to partner with Moffitt on advancing new epigenetic inhibitors, specifically bromodomain inhibitors that simultaneously inhibit specific kinases in key regulatory pathways."

"Aptose views a multi-targeting approach, which incorporates bromodomain inhibition, as an exciting means to enhance efficacy and diminish therapeutic resistance relative to the current landscape in cancer treatment. This is even more beneficial when inhibition of the pathways is highly synergistic. The researchers at Moffitt have made unprecedented progress in this field," continued Dr. Rice.

"We view the advancement of epigenetic multi-inhibitors as a highly promising strategy in the treatment of cancer," said the principal investigators Ernst Schonbrunn, Ph.D. and Nicholas Lawrence, Ph.D., members of the Drug Discovery Program at Moffitt, "and targeting broad-acting epigenetic regulators of transcription like bromodomain proteins is needed to suppress the induction of gene expression that results when cancer cells respond to kinase inhibitors."

"We are excited to work with an organization as scientifically driven to develop novel therapeutics as Aptose," said Haskell Adler, Ph.D., MBA, Senior Licensing Manager at Moffitt.

Exclusive Agreement with Laxai Avanti Life Sciences

Aptose today also announced an exclusive drug discovery partnership with Laxai Avanti Life Sciences (LALS) for their expertise in next generation epigenetic-based therapies. Under the agreement, LALS will be responsible for developing multiple clinical candidates, including optimizing candidates derived from Aptose’s relationship with the Moffitt Cancer Center. Aptose will own global rights to all newly discovered candidates characterized and optimized under the collaboration, including all generated intellectual property.

"We have identified LALS as an organization with high-caliber medicinal chemistry and with robust, and highly efficient drug discovery capabilities that complement our capabilities at Aptose," said Dr. Rice. "These collaborations are designed to build upon insights into the epigenetic field that were informed by the mechanism of APTO-253. As we continue to advance APTO-253 into late-stage clinical development, we are committed to creating and acquiring additional differentiated agents and building a staged oncology pipeline behind APTO-253."

About APTO-253

Epigenetic suppression of the KLF4 gene has been reported in the scientific literature as a key transforming event in AML and high-risk myelodysplastic syndromes. APTO-253, Aptose’s lead drug candidate, is a first-in-class inducer of the Krüppel-like factor 4 (KLF4) tumor suppressor gene, and the only clinical-stage compound targeted for patients with suppressed KLF4 levels. APTO-253 has demonstrated a favorable safety profile with no evidence of bone marrow suppression. Preclinical studies have shown potent single-agent activity and an opportunity for combination therapy with a variety of anti-cancer therapeutics. The drug candidate is in a Phase 1b clinical study in patients with relapsed or refractory hematologic malignancies.

About Moffitt Cancer Center

Located in Tampa, Moffitt is one of only 45 National Cancer Institute-designated Comprehensive Cancer Centers, a distinction that recognizes Moffitt’s excellence in research, its contributions to clinical trials, prevention and cancer control. Moffitt is the top-ranked cancer hospital in Florida and has been listed in U.S. News & World Report as one of the "Best Hospitals" for cancer care since 1999. With more than 4,600 team members, Moffitt has an economic impact in the state of $1.9 billion. For more information, visit MOFFITT.org, and follow the Moffitt momentum on Facebook, Twitter and YouTube.

About Laxai Avanti Life Sciences

Laxai Avanti Life Sciences (LALS) was established in 2007 with a vision to innovate and accelerate the drug discovery campaigns of global pharmaceutical companies. The goal of LALS is to provide intelligent solutions to global pharmaceutical and biotechnological companies by providing high quality services with accelerated timelines. LALS provides a one-stop service for pharmaceutical and biotechnology companies around the globe to accelerate drug discovery programs. LALS current client base includes Biopharmaceutical, Agrochemical and Specialty Chemical Companies in Europe and the US.

About Aptose Biosciences

Aptose Biosciences is a clinical-stage biotechnology company committed to discovering and developing personalized therapies addressing unmet medical needs in oncology. Aptose is advancing new therapeutics focused on novel cellular targets on the leading edge of cancer research, coupled with companion diagnostics to identify the optimal patient population for our products. The Company’s small molecule cancer therapeutics pipeline includes products designed to provide enhanced efficacy with existing anti-cancer therapies and regimens without overlapping toxicities. Aptose Biosciences Inc. is listed on NASDAQ under the symbol APTO and on the TSX under the symbol APS.