On May 10,2018 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported business and financial highlights for the first quarter ended March 31, 2018 (Press release, Celldex Therapeutics, MAY 10, 2018, View Source [SID1234526471]).

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"Celldex has made considerable progress on an important strategic prioritization of our pipeline, following announcement in April of the METRIC study results in triple-negative breast cancer and discontinuation of the glembatumumab program across all indications," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "In 2018, we will focus primarily on continued clinical development of two company-sponsored programs—CDX-1140, a promising CD40 agonist, and CDX-3379, which blocks ErbB3, a receptor thought to play an important role in regulating cancer cell growth and survival. Development of varlilumab and CDX-301 will also continue externally through investigator-sponsored initiatives and internally through inclusion in combination studies."

"In line with this, to extend our financial resources and direct them to the advancement of the programs we believe can bring the most value to both patients and shareholders, we made significant cuts to our business operations, including executing a corporate restructuring in late April. Based on our progress to date, we believe our cash on hand combined with proceeds from our established ATM will support the continued development of our pipeline through 2020. This extended runway will provide for multiple inflection points, and we are solely focused on executing along these lines."

Pipeline Prioritization:

Celldex is focusing its efforts and resources on the continued research and development of:

CDX-1140, an agonist human monoclonal antibody targeted to CD40, a receptor expressed on dendritic cells and a key activator of immune response, currently in a Phase 1 dose-escalation study in multiple types of solid tumors. CD40 agonist antibodies have shown encouraging results in early clinical studies, but systemic toxicity associated with broad CD40 activation has limited their dosing. CDX-1140 is differentiated by potent agonist activity that is independent of Fc receptor interaction, allowing for more consistent, controlled immune activation without promoting cytokine production. Additionally, CD40 ligand binding is not blocked, allowing for potential synergistic effects near activated T cells in lymph nodes and tumors. Celldex is currently focusing its efforts on executing the Phase 1 dose-escalation activities and advancing to combination cohorts. The combination cohorts will include CDX-301, which as a dendritic cell growth factor can increase the number of cells responding to CDX-1140. In addition, combination with varlilumab, especially in lymphomas which co-express these receptors, could have significant potential.

CDX-3379, a human monoclonal antibody designed to block the activity of ErbB3 (HER3), currently in an early Phase 2 study in advanced head and neck squamous cell cancer in combination with Erbitux. The proposed mechanism of action for CDX-3379 sets it apart from other drugs in development in this class due to its ability to block both ligand-independent and ligand-dependent ErbB3 signaling by binding to a unique epitope. It has a favorable pharmacologic profile, including a longer half-life and slower clearance relative to other drug candidates in this class. CDX-3379 also has potential to enhance anti-tumor activity and/or overcome resistance in combination with other targeted and cytotoxic therapies to directly kill tumor cells. Tumor cell death and the ensuing release of new tumor antigens have the potential to serve as a focus for combination therapy with immuno-oncology approaches, even in refractory patients. Celldex intends to complete enrollment to the first stage of the Phase 2 study and will use this data to inform next decisions. In line with this, the Company continues to explore potential other opportunities in additional indications where ErbB3 is believed to play a role.

Varlilumab, an immune modulating antibody targeting CD27 designed to enhance a patient’s immune response against cancer, being studied in multiple investigator initiated research studies and currently completing a Phase 1/2 study across multiple solid tumors in combination with Opdivo. Celldex is conducting the study in collaboration with Bristol-Myers Squibb Company (BMS) and plans to present data at various medical meetings in 2018, including in an oral presentation at the ASCO (Free ASCO Whitepaper) 2018 Annual Meeting in June. The Company intends to explore varlilumab externally through several investigator-initiated studies and internally through inclusion in combination studies.

CDX-301, a dendritic cell growth factor, currently being evaluated in an investigator-initiated pilot study with radiation therapy in patients with advanced non-small cell lung cancer (NSCLC) and planned for combination study with CDX-1140. Celldex believes CDX-301’s potential as a dendritic cell mobilizer could play an important role in immuno-oncology regimens. The Company will continue to support investigator initiated research and will seek to combine CDX-301 with CDX-1140 in its ongoing Phase 1 study of CDX-1140 in the future.

Celldex’s preclinical pipeline includes CDX-0159, which is planned to enter the clinic in 2019; the TAM program, comprised of the targets Tyro3, AXL and MerTK; and a bispecific antibody (BsAb) program. Celldex’s initial BsAb candidate couples CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway using novel, highly active anti-PD-L1 antibodies. Data from this program were presented in a poster at the AACR (Free AACR Whitepaper) 2018 Annual Meeting. The BsAb was more potent in human T cell activation and anti-tumor activity compared to the combined CD27 and PD-L1 antibodies. Enhanced efficacy has been attributed to more efficient cross-linking of the CD27 receptor, resulting in stronger T cell activation.
To conserve resources, Celldex is discontinuing development of:

Glembatumumab vedotin, a targeted antibody-drug conjugate (ADC), across all indications, as previously disclosed;
CDX-014, an ADC (which are typically more costly to develop than other therapeutics), in early Phase 1 development in renal cell and clear cell ovarian carcinomas; and
CDX-1401, an NY-ESO-1-antibody fusion protein, that was being explored in investigator-sponsored and collaborative studies.
Recent Program Highlights Presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2018 Annual Meeting in April

Data from the CDX-1140 program were presented in a poster session. Building off previously presented preclinical work, CDX-1140 was further characterized showing tumor shrinkage and prolonged survival in several xenograft models. These preclinical studies support the potential of CDX-1140 having direct anti-tumor effects on CD40-positive tumors that may supplement its activity as an immune activating agent.

Data from the CDX-3379 program were presented in two poster sessions.
Data were presented from a preoperative "window of opportunity" study in 12 patients with head and neck squamous cell carcinoma (HNSCC). The study was designed to evaluate the effect of CDX-3379 on phosphorylated ErbB3 (pErbB3) and other potential biomarkers in patients with HNSCC. Patients with newly diagnosed HNSCC received two doses of CDX-3379, at a two-week interval prior to tumor resection. CDX-3379 reduced pErbB3 levels in 83% (10/12) of patient samples, with greater than or equal to 50% decreases in 58% of patients (7/12), which met the primary study objective. Stable disease was observed in 92% (11/12) of patients prior to surgery, and a patient with HPV-negative disease experienced significant tumor shrinkage (92% in primary tumor; 26% in metastatic lesion). CDX-3379 was well-tolerated, and no treatment-related adverse events were observed.
Data were presented from a study that explored the reduction of PD-L1 expression by simultaneous blockade of EGFR and ErbB3 in HNSCC. Investigators examined the effects of combining CDX-3379 and cetuximab, a monoclonal antibody targeting EGFR, in xenograft models of HNSCC. Combining CDX-3379 and cetuximab inhibited tumor growth more potently than cetuximab alone. Mechanistic studies demonstrated a reduction of PD-L1 expression from the combination.

Early promising data (n=9) from an ongoing, investigator-initiated pilot study of CDX-301 were presented in a plenary session. This Phase 2 study is evaluating the combination of CDX-301 and stereotactic body radiotherapy (SBRT) in up to 29 patients with advanced non-small cell lung cancer (NSCLC). The presentation included data from nine patients, seven of whom were previously treated with anti-PD(L)1 checkpoint inhibitors. The one-week course of treatment included subcutaneous injections of CDX-301 and SBRT directed to a single lung tumor lesion. Non-irradiated tumors were evaluated for response. Enrollment is ongoing.

Progression-free survival at four months (PFS4), the primary endpoint of the study, was achieved in 56% (5/9) of patients overall and in 100% (5/5) of patients who experienced partial responses (PRs) by PERCIST.
Notably, PRs were observed in non-irradiated tumors in 56% (5/9) of patients at two months; 3 PRs (3/9) were confirmed by immune-related response criteria (irRC).
In the patients previously treated with immune checkpoint inhibitors, 71% (5/7) experienced PRs and PFS4 versus 0% (0/2) in patients not treated with an anti-PD(L)1 therapy.
SBRT in combination with CDX-301 induced and reactivated anti-tumor immune responses in patients who had progressive disease on checkpoint inhibitors.
No dose-limiting toxicities were observed.
First Quarter 2018 Financial Highlights and Updated 2018 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2018 were $123.2 million compared to $139.4 million as of December 31, 2017. The decrease was primarily driven by first quarter cash used in operating activities of approximately $28.0 million and partially offset by the receipt of $11.7 million from sales of common stock under our Cantor agreement. At March 31, 2018, Celldex had 143.4 million shares outstanding.

Revenues: Total revenue was $4.1 million in the first quarter of 2018, compared to $1.5 million for the comparable period in 2017. The increase in revenue was primarily due to the contract manufacturing and research and development agreements with International AIDS Vaccine Initiative and Frontier Biotechnologies, Inc. signed in the second quarter of 2017.

R&D Expenses: Research and development (R&D) expenses were $21.9 million in the first quarter of 2018, compared to $25.8 million for the comparable period in 2017. The decrease in R&D expenses was primarily due to lower varlilumab, CDX-3379 and anti-KIT program product development expenses of $0.9 million, $0.7 million and $0.3 million, respectively, and lower personnel and facility costs of $1.4 million.

G&A Expenses: General and administrative (G&A) expenses were $5.6 million in the first quarter of 2018, compared to $7.2 million for the comparable period in 2017. The decrease in G&A expenses was primarily due to lower personnel expenses of $0.7 million, lower commercial planning costs of $0.4 million and lower legal, consulting and professional services expense of $0.3 million.

Changes in Fair Value Remeasurement of Contingent Consideration: The $13.6 million gain on the fair value remeasurement of contingent consideration in the first quarter of 2018 was primarily due to updated assumptions for glemba-related milestones and discount rates. The $3.4 million loss on fair value remeasurement of contingent consideration in the first quarter of 2017 was primarily due to changes in discount rates and the passage of time.

Intangible Asset and Goodwill Impairments: The Company recorded $18.7 million in non-cash impairment charges related to fully impaired glemba-related intangible assets and $91.0 million in goodwill impairment charges as the carrying value of the Company’s net assets exceeded the Company’s fair value by an amount in excess of the goodwill asset in the first quarter of 2018.

Income Tax Benefit: The Company recorded a $0.8 million non-cash income tax benefit related to the impaired glemba in-process research and development (IPR&D) assets in the first quarter of 2018.

Net Loss: Net loss was $118.1 million, or ($0.84) per share, for the first quarter of 2018, compared to a net loss of $34.3 million, or ($0.28) per share, for the comparable period in 2017.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at March 31, 2018, combined with the anticipated proceeds from future sales of our common stock under the Cantor agreement, are sufficient to meet estimated working capital requirements and fund planned operations through 2020. This could be impacted if Celldex elects to pay Kolltan contingent milestones, if any, in cash.

Opdivo is a registered trademark of Bristol-Myers Squibb. Erbitux is a registered trademark of Eli Lilly & Co.

On May 10, 2018 Cancer Genetics, Inc. (Nasdaq:CGIX), a leader in enabling precision medicine for oncology through molecular markers and diagnostics, reported that it will release its financial results for the first quarter ended March 31, 2018 on Tuesday, May 15, 2018 during pre-market hours (Press release, Cancer Genetics, MAY 10, 2018, View Source [SID1234526470]). The Company will hold a conference call at 8:30 a.m. Eastern on Tuesday, May 15, 2018 to discuss the financial results and provide an update on its strategic direction and key organizational improvements being made by the Company.

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CONFERENCE CALL & WEBCAST
Tuesday, May 15, 2018, 8:30 a.m. Eastern Time
Domestic: 800-289-0438
International: 323-794-2423
Conference ID: 4878415
Webcast: View Source
Replay – Available through May 29, 2018
Domestic: 844-512-2921
International: 412-317-6671
Conference ID: 4878415

On May 10, 2018 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage pharmaceutical company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, reported its financial results for the first quarter ended March 31, 2018 (Press release, Calithera Biosciences, MAY 10, 2018, View Source [SID1234526468]). As of March 31, 2018, cash, cash equivalents and investments totaled $171.2 million.

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"In the first quarter we continued to work towards our goal of developing CB-839 as a potential new treatment option for advanced renal cell carcinoma," said Susan Molineaux, PhD, President and Chief Executive Officer of Calithera. "We are currently enrolling a registration-enabling randomized double-blind placebo controlled trial of CB-839 with cabozantinib for the treatment of renal cell carcinoma and have received Fast Track designation from the FDA for this trial."

First Quarter 2018 and Recent Highlights

CB-839

Preclinical Combination Data Demonstrate Synergy of CB-839 with CDK4/6 and PARP inhibitors. In April 2018, we presented results at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting demonstrating that CB-839 has synergistic anti-proliferative activity when combined with a CDK4/6 inhibitor in colorectal carcinoma (CRC), triple negative breast cancer (TNBC), and ER+ breast cancer cell lines. CB-839 treatment in combination with PARP inhibitors has synergistic anti-proliferative activity in TNBC, CRC, non-small cell lung carcinoma, ovarian and prostate cancer cells. In vivo, the combination of CB-839 with PARP inhibitors or the CDK4/6 inhibitor each enhanced anti-tumor activity in animal models.

Initiated Randomized Phase 2 of CB-839 in Combination with Cabozantinib in Renal Cell Carcinoma. At the 2018 Genitourinary Cancer Symposium in February, we presented preliminary results of the Phase Ib trial of CB-839 in combination with cabozantinib, an oral tyrosine kinase inhibitor, showing that the combination demonstrated a 40% overall response rate in advanced clear cell RCC patients and a 100% disease control rate, with the safety profile of CB-839 plus cabozantinib generally consistent with that of cabozantinib monotherapy. On the basis of this efficacy and safety data, we initiated a randomized double-blind placebo controlled trial, known as CANTATA, comparing patients treated with cabozantinib and CB-839 to patients treated with cabozantinib alone. This trial will enroll approximately 300 clear cell renal cell carcinoma patients who have previously received one or two prior lines of therapy. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for CB-839 in combination with cabozantinib for the treatment of this patient population. In parallel, the ENTRATA trial, a randomized double-blind placebo-controlled study of later line patients, is enrolling approximately 66 patients to receive either everolimus and CB-839 or everolimus alone.

Abstracts Accepted for Presentation at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). A phase 1 Investigator sponsored clinical trial of CB-839 plus capecitabine has been accepted for poster presentation at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).1 In addition, Calithera and clinical collaborators will present two trials-in-progress abstracts, which describe the design of ongoing studies.
INCB001158

Enrolling INCB001158 Clinical Trials. INCB001158 is being evaluated in multiple clinical trials for the treatment of patients with solid tumors both as a monotherapy, and in combination with immunotherapies and chemotherapy. INCB001158 is being developed as part of a collaboration and license agreement with Incyte.
Selected First Quarter 2018 Financial Results

Cash, cash equivalents and investments totaled $171.2 million at March 31, 2018.

Collaboration revenue for the first quarter of 2018 was $5.2 million, compared with $4.2 million for the same period in the prior year, and represents the portion of deferred revenue recognized from our collaboration and license agreement with Incyte. The increase of $1.0 million was primarily due to a full quarter of activity in 2018 verses a partial quarter in 2017, partially offset by differences in accounting due to our adoption of the accounting standard related to revenue from contracts with customers, or ASC 606, on January 1, 2018.

Research and development expenses were $15.4 million for the three months ended March 31, 2018, compared with $6.6 million for the same period in the prior year. The increase of $8.8 million was primarily due to a $7.6 million increase in our CB-839 program to support our new and ongoing clinical trials, including our three Phase 2 trials, as well as an increase of $1.0 million from investment in our early stage research programs, and an increase of $0.2 million from our INCB001158 program.

General and administrative expenses were $3.5 million for the three months ended March 31, 2018, compared with $3.3 million for the same period in the prior year. The increase of $0.2 million was primarily due to $1.0 million in higher personnel-related costs, partially offset by $0.4 million of lower costs associated with entering into the Incyte agreement and $0.4 million lower expenses due to the execution of a sublease agreement for office and laboratory space, both in the first quarter of 2017.

Net loss for the three months ended March 31, 2018 was $13.2 million, or $0.37 per share.

On May 10, 2018 Athersys, Inc. (NASDAQ:ATHX) reported its financial results for the three months ended March 31, 2018 (Press release, Athersys, MAY 10, 2018, View Source [SID1234526466]).

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Highlights of the first quarter of 2018 and recent events include:

Completed a $21.1 million equity investment and announced plans to expand the HEALIOS K.K. ("Healios") collaboration by June 1, 2018. This proposed expansion includes a $10 million license fee funded in an escrow account and a potential additional $25 million of committed payments over time, as well as additional possible payments including milestones and royalties, if the collaboration is fully expanded in accordance with the letter of intent disclosed in March 2018;
Advanced our preparations for the MASTERS-2 Phase 3 registration study for ischemic stroke to enable initiation of this important study and supported the continued enrollment of Healios’ TREASURE study;
Progressed Phase 1/2 study evaluating MultiStem therapy in acute respiratory distress syndrome (ARDS) patients;
Announced plans with The University of Texas Health Science Center at Houston to conduct a Phase 2 clinical trial evaluating MultiStem cell therapy for early treatment and prevention of complications after severe traumatic injury;
Established a new equity facility with Aspire Capital Fund LLC, as a follow-on to a prior facility, providing the Company with access to additional capital as needed to support its ongoing operations;
Recognized revenues of $1.1 million for the quarter ended March 31, 2018 and a net loss of $10.2 million, or $(0.08) per share; and
Ended the 2018 first quarter with $49.7 million of cash and cash equivalents, excluding the $10 million in escrow from Healios related to the proposed collaboration expansion.
"We finished the first quarter in a strong financial position, with approximately $50 million on the balance sheet and look forward to building on that as we work to complete the expansion of our partnership with Healios," commented Dr. Gil Van Bokkelen, Chairman & CEO at Athersys. "As we and Healios recently disclosed, we have extended the timeline to complete the agreements to expand our collaboration until the end of May, and we are working together to achieve that goal.

"We are also focused on preparing for the upcoming initiation of our MASTERS-2 trial, while we continue to support the ongoing TREASURE trial in Japan," added Dr. Van Bokkelen. "In addition, we have been advancing our other clinical programs and broadening our portfolio of new opportunities, as evidenced by the recent announcement related to a clinical study in trauma. This represents another significant area of unmet clinical need that we are now well-positioned to pursue with funding support from the Medical Technology Enterprise Consortium and our collaborator, UTHealth," concluded Dr. Van Bokkelen.

First Quarter Results

Revenues decreased to $1.1 million for the three months ended March 31, 2018 compared to $1.5 million for the three months ended March 31, 2017 . Our revenues are comprised of revenue from manufacturing-related activities for Healios, royalty and related contract revenue from our collaboration with RTI Surgical, Inc. and grant revenue. Our revenue from Healios increased during the first quarter of 2018 compared to the prior year first quarter by approximately $0.3 million as we continue to supply clinical product to Healios and provide other manufacturing-related services, and we expect these revenues will be higher for the 2018 annual period as compared to the 2017 year. Regarding our royalty revenue, excluding a $1.0 million milestone payment from RTI in the 2017 first quarter, royalty revenues increased by approximately $0.2 million in the first quarter of 2018 as a result of an increase in the royalty rate that became effective late 2017 associated with our technology license to RTI. Grant revenue varies from period-to-period with new and completed grants, and the timing of grant-funded activities. Absent new grant awards, we expect our annual grant revenue to decline in 2018 from 2017 with the expiration of certain grant-funded programs.

Research and development expenses increased to $8.9 million for the three months ended March 31, 2018 from $5.6 million in the comparable period in 2017. The $3.3 million increase is primarily comprised of an increase in preclinical and clinical development costs of $2.7 million, an increase in personnel costs of $0.3 million, and an increase in internal research supplies and other research costs of $0.3 million. The increase in our clinical and preclinical costs is primarily a result of increased process development activities to support large-scale manufacturing and clinical product manufacturing costs during the period.

General and administrative expenses increased to $2.7 million for the three months ended March 31, 2018 from $2.1 million in the comparable period in 2017. The $0.6 million increase was due primarily to an increase of $0.3 million in professional fees and increases in personnel costs, stock-based compensation costs and other administrative costs compared to the same period last year.

Net loss was $10.2 million in 2018 compared to $5.6 million in 2017. The difference of $4.6 million reflects the above variances, as well as $0.4 million in insurance proceeds that we received this quarter, which were offset by a $0.7 million non-cash gain related to the fair value of our warrant liabilities recorded in the first quarter of 2017 for warrants that expired in 2017. The warrant issued in March 2018 to Healios in connection with its equity investment was treated as equity with its value recorded as additional paid-in capital with an offset in other current assets.

Net cash used in operating activities was $5.7 million for the three months ended March 31, 2018 and $5.4 million for the three months ended March 31, 2017, reflecting the increased net loss in the first quarter of 2018 (i.e., cash used to fund preclinical and clinical development activities) compared to the prior year period, as offset in part by proceeds received from Healios for our cost-share arrangement for clinical product supply and an increase in accounts payable due to service providers, such as contract manufacturers, under longer-term contracts. As of March 31, 2018, we had $49.7 million in cash and cash equivalents, compared to $29.3 million at December 31, 2017, which includes, among other things, the investment made by Healios in March 2018 and excludes the $10 million that Healios has funded into an escrow account to be released to us upon execution of the expansion agreements.

Conference Call

Gil Van Bokkelen, Chairman and Chief Executive Officer, and William (BJ) Lehmann, President and Chief Operating Officer, will host a conference call today to review the results as follows:

Date Thursday, May 10, 2018
Time 4:30 p.m. (Eastern Time)
Telephone access: U.S. and Canada 800-273-1254
Telephone access: International 973-638-3440
Access code 1189915
Live webcast www.athersys.com, under the Investors section
A replay will be available for on-demand listening shortly after the completion of the call until 11:59 PM Eastern Time on May 24, 2018 at the aforementioned URL, or by dialing (800) 585-8367 or (855) 859-2056 in the U.S. and Canada, or from abroad (404) 537-3406, and entering access code 1189915.

About MultiStem

MultiStem cell therapy is a patented regenerative medicine product in clinical development that has shown the ability to promote tissue repair and healing in a variety of ways, such as through the production of therapeutic factors produced in response to signals of inflammation and tissue damage. MultiStem therapy’s potential for multidimensional therapeutic impact distinguishes it from traditional biopharmaceutical therapies focused on a single mechanism of benefit. The therapy represents a unique "off-the-shelf" stem cell product that can be manufactured in a scalable manner, may be stored for years in frozen form, and is administered without tissue matching or the need for immune suppression. Based upon its efficacy profile, its novel mechanisms of action, and a favorable and consistent safety profile demonstrated in clinical studies, MultiStem therapy could provide a meaningful benefit to patients, including those suffering from serious diseases and conditions with unmet medical need.

On May 10,2018 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported financial results for the three months ended March 31, 2018 and reported on corporate developments (Press release, Aptose Biosciences, MAY 10, 2018, View Source;p=RssLanding&cat=news&id=2348536 [SID1234526465]). Unless specified otherwise, all amounts are in US dollars.

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The net loss for the quarter ended March 31, 2018 was $6.8 million ($0.23 per share) compared with $3.3 million ($0.19 per share) in the quarter ended March 31, 2017 and total cash used in operating activities was $4.1 million compared with $2.7 million in the quarter ended March 31, 2018. Total cash and cash equivalents and investments as of March 31, 2018 were $16.2 million (which, based on current operations, provide the Company with sufficient resources to fund research and development and operations into Q2 2019). In the quarter ended March 31, 2018, Aptose raised $8.9 million from the Common Shares Purchase Agreement with Aspire Capital and subsequent to March 31, we raised a further $6.1 million with Aspire Capital under this same agreement.

"CG-806, our highly potent non-covalent pan-FLT3/pan-BTK inhibitor, continues on track with IND-enabling studies," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "In parallel, new preclinical data on CG-806 presented at AACR (Free AACR Whitepaper) demonstrate its breadth and potency of activity against patient-derived AML and CLL cells and superiority to other kinase inhibitors. For our clinical-stage compound APTO-253, we made notable progress in manufacturing and qualification studies required to return the new drug product to the clinic. We also presented new mechanistic data for APTO-253 at AACR (Free AACR Whitepaper), demonstrating heightened sensitivity of BRCA1 or BRCA2 mutated cancer cells to APTO-253 and opening the door to genetically-defined solid tumor indications for the molecule."

Corporate Highlights

CG-806 preclinical data presented at AACR (Free AACR Whitepaper) – At the 2018 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Conference held in April, researchers from Oregon Health & Science University (OHSU) Knight Cancer Institute and Aptose presented preclinical data demonstrating that CG-806, a highly potent pan-FLT3/pan-BTK inhibitor, kills malignant cells in samples freshly collected from patients with various hematologic malignancies and demonstrates superiority to other kinase inhibitors. CG-806 was shown to have greater potency against a broader set of AML samples relative to other FLT3 inhibitors, including midostaurin, gilteritinib, quizartinib, sorafenib, crenolanib, and dovitinib. Separately, CG-806 was also shown to have greater potency and range of activity than ibrutinib on patient-derived CLL samples.

An additional study presented by Aptose explored the potency of CG-806 in hematologic malignancies relative to other FLT3 or BTK inhibitors commercialized or in development. CG-806 induced apoptosis through inhibition of key multiple signaling pathways that were specific to each sample of AML or B-cell cancer cells. CG-806 was also superior to other FLT3 inhibitors against FLT3-wild type (WT) AML cells, as well as AML cells housing various mutant forms of FLT3. Collectively, the data demonstrated the ability of CG-806 to target all WT and mutant forms of FLT3 and BTK and to inhibit multiple signaling pathways, producing killing of diverse subtypes of hematologic malignancies driven by different genomic aberrations.
New mechanistic data on APTO-253 presented at AACR (Free AACR Whitepaper) – Also at AACR (Free AACR Whitepaper), the Aptose research team presented data showing that APTO-253 stabilizes a G-quadruplex DNA structure in the MYC gene promoter, leading to suppression of MYC expression and induction of programmed cell death in AML cells. The action of APTO-253 on separate G-quadruplexes induces the DNA damage response in cancer cells and exhibits synthetic lethality comparable to olaparib – an FDA-approved targeted therapy that acts against cancers in people with hereditary BRCA1 or BRCA1 mutations, including some ovarian, breast and prostate cancers – albeit through a different mechanism. Unlike other drugs for which loss of this DNA repair function results in hypersensitivity, APTO-253 does not produce myelosuppression, even at the maximum tolerated dose. These findings open the window to how APTO-253 might be used clinically to treat certain solid tumor patients with tumors harboring deficiencies in DNA repair.

Completed manufacture of APTO-253 cGMP clinical supply – The Company completed the manufacture of the cGMP clinical supply that will be required for the potential return of APTO-253 to the clinic, and the new clinical supply has completed and passed stability, sterility, mock infusion, animal bridging and blood compatibility studies. Aptose plans to submit these findings to the FDA during the 2nd Quarter of 2018 to seek release of the CMC-related clinical hold and allow return of APTO-253 to dosing in the open Phase 1b trial in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

CG-806 pre-IND progress – Aptose successfully manufactured CG-806 drug substance and formulated drug product, and then performed animal dose range finding preclinical studies in rodents and dogs, and successfully dosed up to 1000 mg/kg/day, the maximum feasible dose. The Company is currently manufacturing a drug batch that will be used for IND-enabling GLP animal toxicology studies that are planned to begin during the 2nd quarter of 2018.

Appointment of New Board Member – In April, Aptose announced the appointment of Caroline M. Loewy to its Board of Directors. Ms. Loewy is an accomplished and respected executive leader with more than 25 years of experience in accelerating biotechnology product development and growth. She currently provides strategic advisory services to life science companies on a variety of high-impact matters including funding strategies, product pipeline evaluation, and assessing business development opportunities. Prior, she held numerous executive roles, including that of Chief Financial Officer at several public and private biopharmaceutical companies, and senior biotechnology research analyst at Morgan Stanley and Prudential Securities. Aptose’s Board of Directors now includes seven members with extensive biotechnology and pharmaceutical experience.

Early Exercise of Option for CG-806 License – In May, Aptose exercised its option under the 2016 Option Agreement to exclusively license CG-806 from CrystalGenomics, Inc. With the early exercise of the option, Aptose owns global rights to develop and commercialize CG-806 for all indications outside of Korea and China – the Licensed Territory. The exercise triggered a payment of $2.0 million to CrystalGenomics, and CrystalGenomics is eligible for regulatory and sales milestone payments, as well as royalties on product sales in the Licensed Territory.
New Base Shelf Prospectus Filing and New At-The-Market Facility – As previously announced in February, Aptose filed a new Base Shelf Prospectus in Canada, and a registration statement with the SEC, which allows the Company to offer up to $100,000,000 of common shares, warrants to purchase common shares or units comprised of one or more of these securities, for a period of 25 months. This provides Aptose with flexibility to raise capital to fund operations, R&D and potential upcoming clinical trials. Subsequently in March, Aptose entered into a new At-The-Market (ATM) Facility to replace the ATM Facility that expired in December 2017.