On March 6, 2019 Idera Pharmaceuticals, Inc. ("Idera") (NASDAQ: IDRA), a clinical-stage biopharmaceutical company focused on the development, and ultimately the commercialization, of therapeutic drug candidates for both oncology and rare disease indications characterized by small, well-defined patient populations with serious unmet medical needs, reported its financial and operational results for the fourth quarter and year ended December 31, 2018 (Press release, Idera Pharmaceuticals, MAR 6, 2019, View Source [SID1234534076]).

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"2018 ended with incredibly positive momentum for tilsotolimod which carried into 2019 and continues today," stated Vincent Milano, Idera’s chief executive officer. "Despite the challenges we faced in the first half of 2018, we emerged as a tightly focused organization with a clear understanding of our mission to bring tilsotolimod to the market in our initial indication of anti-PD-1 refractory metastatic melanoma. We also are exploring tilsotolimod as an investigational therapy for patients with other tumor types that have not been well-served by available immunotherapies to date," continued Milano. "The ILLUMINATE development program is making excellent progress across the board and I am incredibly proud of our team for their continued dedication and execution."

"As it pertains to Idera overall, we will continue business development efforts to identify and pursue assets and opportunities with the purpose of further growing our company for long-term success and value creation for our loyal shareholders. However, I cannot be any clearer that the advancement and future success of tilsotolimod is our highest priority, passion and focus."

ILLUMINATE (tilsotolimod) Clinical Development Update

ILLUMINATE 301 – Randomized phase 3 trial of tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with PD-1 refractory metastatic melanoma:

• Overall Response Rate (ORR) and Overall Survival (OS) as primary endpoints;

• Trial initiated in the first quarter of 2018;

• Sites planned in 12 countries: 78 sites activated;

• Planned enrollment of approximately 300 patients; and

• Completion of enrollment expected during the fourth quarter of 2019.

ILLUMINATE 206 – Phase 2, multi-center trial to test the safety and effectiveness of tilsotolimod in combination with ipilimumab and nivolumab in treating patients with Squamous Cell Carcinoma of the Head and Neck (SCCHN) and Microsatellite Stable Colorectal Cancer (MSS-CRC).

• Received notice from the U.S. Food and Drug Administration that the company can proceed to implement the ILLUMINATE-206 clinical trial under a new Investigational New Drug (IND) application; and

• Both trial cohorts of SCCHN and MSS-CRC expected to initiate in the second quarter of 2019.

ILLUMINATE 204 – Phase 1/2 trial of tilsotolimod in combination with ipilimumab or pembrolizumab in patients with PD-1 refractory metastatic melanoma:

• Completed enrollment with 52 patients in Phase 2 expansion at 8 mg (RP2D) dose with ipilimumab;

• Completed target enrollment of at least 40 patients in the primary enrollment population constituting patients who are naïve to prior ipilimumab treatment in the metastatic setting;

• Presented an interim data update in December 2018 which showed:

32.4% ORR of the first 34 patients evaluable for efficacy including 9% (N=3) achieve Complete Response (CR); 24% (N=8) achieving Partial Response (PR); and 76.5% (N=26) achieving disease control (CR, PR or Stable Disease [SD]); and
• Data from ILLUMINATE-204 expected in the fourth quarter of 2019.

ILLUMINATE 101 – Phase 1b trial of tilsotolimod monotherapy in patients with refractory solid tumors:

• Completed enrollment in all dose cohorts of the trial; and

• Abstract accepted for presentation of translational data from this trial at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2019 Annual Meeting, being held March 29 – April 3, 2019 in Atlanta, GA.

Corporate Updates:
The company today is announcing the planned retirement of Senior Vice President and Chief Medical Officer Joanna Horobin, M.B. Ch.B, given the consolidation of Idera’s business operations and team in Pennsylvania. The retirement will be effective July 31, 2019. Dr. Horobin joined Idera in November 2015 and notably has led the advancement of the tilsotolimod ILLUMINATE program, advancing the program from pre-clinical into exploratory early phase clinical and translational evaluation through to the ongoing ILLUMINATE 301 phase 3 trial in anti-PD-1 refractory metastatic melanoma and most recently expanding into a study in patients SCCHN and MSS-CRC.

Dr. Horobin will assist Idera in the search to fill the role of Chief Medical Officer and will remain with the company in an advisory capacity following her July 31 retirement.

"I’m incredibly appreciative to Joanna for the critical role she has played as we transitioned Idera to a focused clinical development organization with a clear eye towards ultimate commercialization of products for patients suffering from life-threatening rare diseases," offered Milano. "We all wish Joanna the utmost happiness as she transitions to the next phase of her life following a tremendously productive and rewarding 35-year career developing critical medicines that have made a positive impact on countless patients’ lives."

"I am tremendously proud of the progress we’ve made at Idera over the past several years. We have shown clinical proof-of-concept of tilsotolimod to prime the immune system to better respond to checkpoint inhibition which may enable tilsotolimod to benefit more patients," stated Dr. Horobin. "It’s been a great honor to work with so many talented individuals during my time at Idera and I look forward to continuing to assist in an advisory capacity as the opportunities for tilsotolimod expand."

Additionally, since September 30, 2018, the following corporate updates were announced:

• The company entered into an at the market offering (ATM) agreement with JMP Securities LLC under which the company may elect to sell shares of its common stock having an aggregate offering price of up to $50 million;

• The company entered into a common stock purchase agreement and registration rights agreement with Lincoln Park Capital (LPC) Fund, LLC, under which the company has the right to sell an aggregate of up to $35 million of its common stock at the company’s discretion; and

• Carol A. Schafer was appointed to Idera’s Board of Directors on December 18, 2018, filling the seat of Mr. William Reardon, who will be resigning from the Board effective March 10, 2019.

Upcoming Investor Conference:
The company will participate in the 2019 Barclays Global Healthcare Conference on Tuesday, March 12, 2019 at 11:15 a.m. Eastern Time at the Loews Miami Beach Hotel in Florida.

Live audio webcast of Idera’s presentations will be accessible in the Investors and Media section of Idera’s website at View Source Archived versions will also be available on the Company’s website after the event for 90 days.

Financial Results
Fourth Quarter Results
Net loss applicable to common stockholders for the three months ended December 31, 2018 was $12.2 million, or $0.45 per basic and diluted share, compared to net loss applicable to common stockholders of $14.9 million, or $0.66 per basic and diluted share, for the same period in 2017. Revenue in the fourth quarter of 2018 was nominal. Research and development expenses for the three months ended December 31, 2018 totaled $8.9 million compared to $10.4 million for the same period in 2017. General and administrative expense for the three months ended December 31, 2018 totaled $3.6 million compared to $3.7 million for the same period in 2017.

Full Year Results
Net loss applicable to common stockholders for the year ended December 31, 2018 was $59.9 million or $2.25 per basic and diluted share, compared to net loss applicable to common stockholders of $66.0 million, or $3.35 per basic and diluted share, for the same period in 2017. Revenue for the year ended December 31, 2018 was $0.7 million compared to revenue of $0.9 million for the same period in 2017. Research and development expenses for the year ended December 31, 2018 totaled $41.8 million compared to $50.7 million for the same period in 2017. General and administrative expenses for the year ended December 31, 2018 totaled $15.4 million compared to $15.6 million for the same period in 2017. Merger-related costs, net for the year ended December 31, 2018 totaled $1.2 million compared to $1.1 million for the same period in 2017. Restructuring costs for the year ended December 31, 2018 totaled $3.1 million and related to our decision in July 2018 to wind-down our discovery operations. No such costs were incurred in 2017.

As of December 31, 2018, our cash and cash equivalents totaled $71.4 million compared to $112.6 million as of December 31, 2017. We currently anticipate that, based on our current operating plan, our existing cash, cash equivalents and investments will fund our operations into the first quarter of 2020.

On March 6, 2019 Clovis Oncology, Inc. (Nasdaq: CLVS) reported that its Chief Executive Officer and President, Patrick J. Mahaffy, will present at the Barclays Global Healthcare Conference on Wednesday, March 13, 2019 at 11:15 a.m. Eastern time (Press release, Clovis Oncology, MAR 6, 2019, View Source [SID1234534037]). The conference will be held at the Loews Miami Beach Hotel in Miami.

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A live webcast of the presentation can be accessed through the investor relations section of the Company’s website at www.clovisoncology.com. Following the live presentation, a replay of the webcast will be available on the Company’s website for 30 days.

On March 6, 2019 Trovagene, Inc. (Nasdaq: TROV), a clinical-stage oncology therapeutics company, using a precision medicine approach to develop drugs that target cell division (mitosis) for the treatment of leukemias, lymphomas and solid tumor cancers, reported company highlights and financial results for the fourth quarter and full-year ended December 31, 2018 (Press release, Trovagene, MAR 6, 2019, View Source [SID1234534038]). The company is issuing this press release in lieu of conducting a conference call.

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"We are pleased with the progress we are making in the clinical development of onvansertib for the treatment of cancers and indications where there is a significant need to bring new therapeutic options to physicians and their patients – Acute Myeloid Leukemia (AML), metastatic Castration-Resistant Prostate Cancer (mCRPC) and metastatic Colorectal Cancer (mCRC)," said Dr. Thomas Adams, Chief Executive Officer and Chairman of Trovagene. "Our AML trial continues to advance and data shows that the combination of onvansertib and standard-of-care chemotherapy is demonstrating both a favorable safety profile and showing activity in greater than 88% of evaluable patients treated to-date, which is very encouraging. In January, we received a "study may proceed" notification from the FDA for our Phase 1b/2 trial in mCRC and expect that enrollment in this trial, which is being funded by PoC Capital, will begin mid-year. We anticipate having data readouts from our Phase 2 trial in mCRPC throughout 2019, and recently presented a poster at the Genitourinary Cancers Symposium (ASCO-GU), overviewing the trial and confirming the safety of the combination of onvansertib and Zytiga. We will also be presenting safety and preliminary clinical data from our Phase 1b/2 trial in AML and Phase 2 trial in mCRPC at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual conference in early April, 2019."

Dr. Adams added, "We achieved a number of key milestones in 2018, including: Successful completion of the first three dose levels, without any dose-limiting toxicities, in the Phase 1b segment of our AML trial; granting of Orphan Drug Designation in Europe for the treatment of AML; presentation of preliminary clinical data from our AML trial at the American Society of Hematology (ASH) (Free ASH Whitepaper) conference in December; opening of our mCRPC Phase 2 trial to full enrollment, following confirmation that the combination of onvansertib and Zytiga is safe and well tolerated in the safety lead-in phase; submission of a new IND and protocol for our Phase 1b/2 trial in mCRC in December; strengthening of our patent portfolio around our drug candidate, onvansertib, with the issuance of two new patents; and entering into an exclusive license agreement with MIT to develop combination therapies that include anti-androgen or androgen antagonist and a Polo-like Kinase (PLK) inhibitor (onvansertib) for the treatment of cancer."

The Company has advanced its business in 2018, and to-date in 2019, with the following activities and milestone achievements:

Announced Presentation Update on Phase 2 Study of Onvansertib in Combination with Zytiga in Patients with mCRPC at ASCO (Free ASCO Whitepaper)-GU
On February 14, 2019, Trovagene announced the presentation of a poster reviewing its ongoing Phase 2 study evaluating onvansertib in combination with Zytiga (abiraterone acetate)/prednisone in patients with metastatic Castration-Resistant Prostate Cancer (mCRPC) at the Genitourinary Cancers Symposium (ASCO-GU) in San Francisco, CA. The data featured demonstrates the safety and tolerability of onvansertib in combination with Zytiga which was confirmed in the safety lead-in portion of the trial that was completed prior to opening the trial to full enrollment. In addition, a second arm is planned with the goal of maximizing clinical activity by reducing the dosing schedule from the current 21-day cycle to a 14-day cycle.

Announced that Trovagene and PoC Capital Entered into an Agreement to Fund Clinical Development of Onvansertib in metastatic Coloreactal Cancer (mCRC)
On January 29, 2019, Trovagene announced an agreement with PoC Capital to fund its Phase 1b/2 trial of onvansertib in combination with FOLFIRI and Avastin in patients with mCRC with a KRAS mutation. Trovagene submitted an Investigational New Drug (IND) application and protocol to the FDA on December 19, 2018, and received a "study may proceed" notification from the FDA, 28-days later, on January 16, 2019. The trial will be conducted at two prestigious cancer centers in the U.S.; USC Norris Comprehensive Cancer Center and The Mayo Clinic.

Announced New Patent Issued for Combination of Onvansertib and Ant–Androgen Drugs to Treat Non-Metastatic and Metastatic Prostate Cancer
On January 23, 2019, Trovagene announced the issuance of a new patent (10,155,006), entitled Combination Therapies and Methods of Use Thereof for Treating Cancer, by the U.S. Patent and Trademark Office (USPTO). This patent broadens previously issued patent (9,566,280), by expanding the use of Onvansertib to encompass combination therapies with any anti-androgen and androgen antagonist drug, such as Zytiga, Xtandi and Erleada for the treatment of metastatic and non-metastatic castrate-resistant prostate cancer. The issuance of this patent further strengthens Trovagene’s existing intellectual property portfolio obtained with the licensing of exclusive global development and commercialization rights to onvansertib from Nerviano Medical Sciences in March, 2017.

Announced New Data from Phase 1b/2 Study of Onvansertib in Combination with LDAC or Decitabine Demonstrated Response to Treatment in Relapsed/Refractory AML
On December 3, 2018, Trovagene announced presentation of data from its ongoing Phase 1b/2 study evaluating onvansertib in combination with standard-of-care chemotherapy in Acute Myeloid Leukemia (AML) at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting. The date presented in a poster demonstrated that onvansertib, in combination with LDAC or decitabine is benefiting patients who have relapsed/refractory AML and that the combination is safe and well tolerated, with no serious adverse events (SAEs) reported to-date.

Announced New Patent Claim Allowances Affirming Broad Patent Portfolio Coverage of NPM1 Mutations by U.S. Patent and Trademark Office
On October 24, 2018, Trovagene announced that the U.S. Patent and Trademark Office (USPTO) has allowed claims that affirms broad coverage of NPM1 mutation testing; Patent Application 14/750331, entitled "Nucleophosmin Protein (NPM) Mutants, Corresponding Gene Sequences and Uses Thereof." This patent encompasses broad claims around the assessment of NPM1 mutational status in any cancer type, including acute myeloid leukemia (AML). This not only aligns with the Company’s current biomarker strategy and clinical development of onvansertib in AML, but also strengthens the revenue generating potential for Trovagene.

Announced Exclusive License Agreement with MIT for Combination Therapy of Anti-Androgens and Polo-like Kinase Inhibitors in Prostate Cancer
On October 3, 2018, Trovagene announced that it has entered into an exclusive patent license agreement with the Massachusetts Institute of Technology (MIT). Under the agreement, Trovagene has exclusive rights to develop combination therapies that include anti-androgen or androgen antagonist and a Polo-like Kinase (PLK) inhibitor for the treatment of cancer. The exclusive license agreement is part of Trovagene’s strategy to explore the efficacy of Onvansertib, its first-in-class, 3rd generation, highly-selective, oral PLK1 inhibitor, in combination with anti-androgen drugs in cancers including prostate, breast, pancreatic, lung and gastrointestinal. There is a need for new therapies that effectively treat cancers that depend on internal androgen signaling, such as castration-resistant prostate cancer, as well as cancers which over-express androgen receptor (AR), or are otherwise dependent on the synthesis of steroid hormones for their growth, such as some breast cancers. In-vitro and in-vivo preclinical research demonstrates a unique synergistic effect with the combination of PLK inhibitors and anti-androgens, which was the precursor that led to the current Phase 2 trial of onvansertib in combination with Zytiga (abiraterone acetate)/prednisone that is being conducted at the three Harvard Medical Cancer Centers.

Announced Completion of Dosing Cohort of Patients Treated with Onvansertib in Combination with Decitabine in Ongoing Phase 1b/2 AML Trial
On September 27, 2018, Trovagene announced completion of the second dosing cohort of onvansertib, its first-in-class, 3rd generation, highly-selective oral Polo-like Kinase 1 (PLK1) Inhibitor, in combination with standard-of-care decitabine, in its Phase 1b/2 clinical trial in patients with Acute Myeloid Leukemia (AML). All three patients in the cohort successfully completed treatment with onvansertib at 18mg/m2, administered orally, once daily, on days 1-5 of the treatment cycle, in combination with decitabine and the combination was well tolerated. The Safety Review Committee (SRC) has recommended escalating to the next dose level of onvansertib at 27mg/m2 (approximately a 50% increase) in combination with decitabine.

Announced Predictive Clinical Biomarker Approach to Identify Acute Myeloid Leukemia (AML) Patients Most Likely to Respond to Onvansertib
On September 5, 2018, Trovagene announced it has developed a method for predicting response to treatment by measuring the ability of onvansertib, a first-in-class, 3rd generation, oral and highly-selective Polo-like Kinase 1 (PLK1) inhibitor, to inhibit PLK1 in patients with Acute Myeloid Leukemia (AML). PLK1 uniquely phosphorylates translational control tumor protein (TCTP) to form pTCTP and inhibition of this enzymatic activity by onvansertib appears to be predictive of patient response to treatment. In the ongoing Phase 1b/2 open label clinical trial in AML, PLK1 inhibition is being assessed 3-hours following administration, at the approximate peak concentration (Cmax) of onvansertib. In the first six patients treated, the greatest target engagement, or inhibition of PLK1, was observed in the three patients who showed a response to treatment.

Announced European Commission Grants Orphan Drug Designation to Onvansertib (PCM-075) for Treatment of Acute Myeloid Leukemia in Europe
On August 29, 2018, Trovagene announced that the European Commission (EC) has endorsed the positive opinion of the Committee for Orphan Medicinal Products (COMP) and has granted Orphan Drug Designation (ODD) for onvansertib, a first-in-class, 3rd generation, oral and highly-selective Polo-like Kinase 1 (PLK1) inhibitor, for the treatment of patients with Acute Myeloid Leukemia (AML). Orphan drug designation by the EC provides regulatory and financial incentives to Trovagene, including reduced fees during the product development phase, direct access to centralized marketing authorization in the EU, and 10-year market exclusivity following product approval.

Announced Completion of Second Dosing Cohort of Patients Treated with Onvansertib (PCM-075) in Ongoing Phase 1b/2 AML Trial
On August 16, 2018, Trovagene announced completion of the second dosing cohort of onvansertib, a first-in-class, 3rd generation, highly-selective oral Polo-like Kinase 1 (PLK1) Inhibitor, in combination with standard-of-care low-dose cytarabine (LDAC), in its Phase 1b/2 clinical trial in patients with Acute Myeloid Leukemia (AML). All three patients in the cohort successfully completed treatment with onvansertib at 18 mg/m2, administered orally, once daily, on days 1-5 of the treatment cycle, in combination with LDAC and the combination was well tolerated. The Safety Review Committee (SRC) has recommended escalating to the next dose level of onvansertib at 27 mg/m2 (approximately a 50% increase) in combination with LDAC. Additionally, two patients in the three-patient cohort of onvansertib at 18 mg/m2 in combination with decitabine have also successfully completed at least one cycle of treatment and recruitment of the third patient to complete this cohort is in process. Four of the eleven patients treated to-date remain on treatment, three are currently receiving a second cycle of treatment and one patient is scheduled to start a fifth cycle of treatment.

Fourth Quarter 2018 Financial Results

Total operating expenses were approximately $4.2 million for the three months ended December 31, 2018, an increase of million $0.2 million from $4.0 million for the same period in 2017. The increase in operating expenses is attributed to advancing the onvansertib clinical development programs.

Net cash used in operating activities in the fourth quarter of 2018 was $3.6 million, compared to $3.3 million in the fourth quarter of 2017. The year-over-year increase of $0.3 million can be attributed primarily to progress made with the clinical development of its drug candidate, onvansertib.

Research and development expenses increased by approximately $1.3 million to $2.5 million for the three months ended December 31, 2018 from $1.2 million for the same period in 2017. The overall increase in research and development expenses was primarily due to the increased outside service costs for clinical studies related to the development of our drug candidate, onvansertib. We expect increases in research and development costs to continue as we advance the onvansertib clinical development programs in AML, mCRPC and mCRC.

Selling, general and administrative expenses decreased by approximately $0.2 million to $1.7 million for the three months ended December 31, 2018 from $1.9 million for the same period in 2017. The reduction is primarily due to a decrease in stock-based compensation.

The weighted average diluted shares of common stock outstanding used to calculate per share results for the three months ended December 31, 2018 was 3.8 million.

As of December 31, 2018, Trovagene had approximately $11.5 million of cash and cash equivalents.

On March 6, 2019 Castle Biosciences, Inc., a skin cancer diagnostics company providing personalized genomic information to improve cancer management decisions, reported publication of an independent, prospective study of patients with Stage IB and II cutaneous melanoma showing that the DecisionDx-Melanoma gene expression profile (GEP) test accurately identifies risk of melanoma recurrence (Press release, Castle Biosciences, MAR 6, 2019, View Source [SID1234535089]). The study was published in the Journal of the European Academy of Dermatology and Venereology. Results are consistent with the eight previously published prospective and retrospective performance studies showing the clinical value of the DecisionDx-Melanoma test to predict patient outcomes, which can help inform management decisions.

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Study Details:

A total of 86 patients diagnosed with cutaneous melanoma between April 2015 and December 2016 were prospectively enrolled across five tertiary melanoma referral centers in Spain.
Patients received the DecisionDx-Melanoma test as part of their initial work-up.
Median age was 59.2 years, mean tumor Breslow thickness was 2.5 mm and 70% of patients did not have ulcerated tumors.
62 patients (72%) had American Joint Committee on Cancer (AJCC 7th edition) Stage IB/IIA melanoma (considered "low risk"), and 24 patients (28%) had Stage IIB/C melanoma. Patients who had a positive sentinel lymph node biopsy result were excluded.
Overall median follow-up time was 26 months.
Key Results:

53 patients had a Class 1 (low risk) DecisionDx-Melanoma test result; 33 patients were Class 2 (high risk).
All 7 patients who experienced a recurrence were Class 2; no Class 1 patients experienced a recurrence (p<0.001). Two of the patients who experienced a recurrence were classified as low risk by AJCC staging but high risk (Class 2) by their DecisionDx-Melanoma test result.
Disease-free survival rate was significantly higher for patients with a Class 1 test result compared to those with a Class 2 result based on Kaplan-Meier survival curve analysis (p<0.001).
When AJCC staging was combined with the DecisionDx-Melanoma test result, the significant difference in disease-free survival between Class 1 and Class 2 results was maintained (p=0.001).
Multivariate analysis that included age and AJCC staging showed the DecisionDx-Melanoma test result to be an independent predictor of recurrence (Class 2 hazard ratio=18.82, p=0.01).
"For patients with early stage melanoma, this prospective, multicenter study shows that the DecisionDx-Melanoma test can accurately predict risk of recurrence both independently and when combined with traditional AJCC staging factors," commented Sebastian Podlipnik, M.D., Hospital Clinic of Barcelona, University of Barcelona, Spain. "These results are consistent with previous retrospective and prospective studies, and show that the DecisionDx-Melanoma test can improve identification of high-risk patients and inform decisions on surveillance and follow-up strategies."

The full published study results can be accessed at the journal’s website.

About DecisionDx-Melanoma

DecisionDx-Melanoma is a gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as sentinel lymph node positivity, independent of traditional staging factors and has been studied in over 2,900 patients. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in three multicenter studies that have included 690 patients and have demonstrated consistent results. Performance has also been confirmed in five prospective studies including over 780 patients. The consistent high performance and accuracy demonstrated in these studies, which combined have included over 1,470 patients, provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results.

Prediction of the likelihood of sentinel lymph node positivity has also been validated in two prospective multicenter studies that included over 1,400 patients. Impact on patient management plans for one of every two patients tested has been demonstrated in multicenter and single-center studies. More information about the test and disease can be found at www.SkinMelanoma.com.

On March 6, 2019 GlycoMimetics, Inc. (Nasdaq:GLYC) reported its financial results for the year and fourth quarter ended December 31, 2018, highlighted recent company achievements and commented on milestone achievements anticipated in 2019 (Press release, GlycoMimetics, MAR 6, 2019, View Source [SID1234534007]). Cash and cash equivalents at December 31, 2018 were $209.9 million.

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"In 2018, GlycoMimetics delivered achievements on many fronts: in clinical development, preclinical research and discovery and in financial resource management. We advanced our comprehensive clinical program for uproleselan that, if successful, could position uproleselan as a foundational therapy across the spectrum of AML. We believe this accomplishment reflects the enthusiasm of clinicians who have seen the Phase 1/2 uproleselan data at top oncology congresses and uproleselan’s impact on patient outcomes in clinical trials. In terms of our discovery and preclinical research during 2018, our team, utilizing our specialized chemistry expertise, produced new drug candidates to expand our pipeline opportunities into indications that go beyond sickle cell disease and hematologic cancers. Looking forward to 2019, we are in a strong financial position to pursue the clinical and preclinical opportunities ahead. Importantly, we look to the rivipansel top-line readout expected late in the second quarter that could represent both the first potential commercial success from our pipeline and a key financial resource going forward," noted Rachel King, Chief Executive Officer.

2018 and Recent Highlights

The GlycoMimetics-sponsored pivotal Phase 3 trial of uproleselan in relapsed/refractory AML enrolled its first patient; multiple investigative sites have now been initiated; work continues to expand to clinical sites across the US, Europe, Canada and Australia
The National Cancer Institute (NCI) collaborative study of uproleselan in newly diagnosed patients fit for chemotherapy has opened and is recruiting patients at multiple sites
Planning continues for the collaborative Haemato Oncology Foundation for Adults in the Netherlands (HOVON) European study of uproleselan in newly diagnosed patients unfit for chemotherapy with a goal of trial initiation in 2019
At the ASH (Free ASH Whitepaper) Annual Meeting in December 2018, key new data on clinical outcomes from the Phase 1/2 relapsed/refractory AML trial of uproleselan underscored opportunities to position this drug candidate, if approved, as a potential foundational therapy across the spectrum of AML
Our Japanese patent for uproleselan was granted in August 2018, complementing patents already issued in the United States and Europe
Preclinical data for several existing and new pipeline programs, including GMI-1687 and GMI-1757, were presented at key scientific meetings, including AACR (Free AACR Whitepaper) in March 2018 and ASH (Free ASH Whitepaper) in December 2018
GlycoMimetics’ collaborator Pfizer advised that top-line results of the rivipansel Phase 3 clinical trial would be announced by late second quarter 2019
Scott Jackson, veteran biopharma executive, joined the Board of Directors
Chairman of the Board, M. James Barrett, Ph.D., GlycoMimetics’ founding venture investor and John Magnani, Ph.D., GlycoMimetics’ Co-founder and Chief Scientific Officer, notified the company that they will not run for reelection to the Board. At the Annual Meeting of Stockholders on May 17, 2019, the Board Chair position will be taken by Tim Pearson, a GlycoMimetics Director since 2014, and until recently, Chief Financial Officer and Executive Vice President for TESARO, Inc., a publicly held oncology-focused biopharmaceutical company recently acquired by GlaxoSmithKline
Fourth Quarter and Year-end 2018 Financial Results:

Cash position: As of December 31, 2018, GlycoMimetics had cash and cash equivalents of $209.9 million as compared to $123.9 million as of December 31, 2017. In March 2018, the Company completed a public offering of 8,050,000 shares of common stock yielding net proceeds of $128.4 million.
R&D Expenses: The Company’s research and development expenses increased to $12.0 million for the quarter ended December 31, 2018 as compared to $6.7 million for the fourth quarter of 2017. Research and development expenses increased by $16.0 million to $40.1 million for the year ended December 31, 2018, from $24.1 million in the year ended December 31, 2017. These increases were primarily the result of higher manufacturing costs to scale up production of uproleselan clinical supplies for the Company’s Phase 3 clinical trial and for clinical trials conducted by or in collaboration with third parties. Personnel-related and stock-based compensation increased due to an increase in clinical headcount.
G&A Expenses: The Company’s general and administrative expenses increased to $2.9 million for the quarter ended December 31, 2018 as compared to $2.8 million for the fourth quarter of 2017. General and administrative expenses for the year ended December 31, 2018 increased to $11.4 million as compared to $9.8 million in the prior year. These increases were primarily due an increase in legal and patent expenses as well as labor-related costs and stock-based compensation expense. Patent expenses were higher due to an increase in the number of patent applications filed. Personnel-related and stock-based compensation expenses increased due to additional headcount in 2018, annual salary adjustments and annual stock option awards granted in the first quarter of 2018.
Shares Outstanding: Shares of common stock outstanding as of December 31, 2018 were 43,160,751.
The company will host a conference call and webcast today at 8:30 a.m. ET. The dial-in number for the conference call is (844) 413-7154 for domestic participants and (216) 562-0466 for international participants, with participant code 5072004. A webcast replay will be available via the "Investors" tab on the GlycoMimetics website for 30 days following the call. A dial-in phone replay will be available for 24 hours after the close of the call by dialing (855) 859-2056 for domestic participants and (404) 537-3406 for international participants, participant code 5072004.

About Uproleselan (GMI-1271)

uproleselan (yoo’ pro le’ sel an) is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed/refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better than expected remission rates and overall survival compared to historical controls, which have been derived from results from third party clinical trials evaluating standard chemotherapy, as well as lower than expected induction-related mortality rates. Treatment in these patient populations was generally well tolerated, with fewer than expected adverse effects. The U.S. Food and Drug Administration (FDA) has granted uproleselan Breakthrough Therapy Designation for the treatment of adult AML patients with relapsed/refractory (R/R) disease. GlycoMimetics is implementing a comprehensive development program across the clinical spectrum of AML. This includes the company-sponsored Phase 3 trial in R/R AML that is currently enrolling patients and two consortia-sponsored trials in newly diagnosed patients. One consortium trial is being sponsored by the NCI and will enroll newly diagnosed patients fit for intensive chemotherapy. The other trial is sponsored by the HOVON group in Europe and will enroll newly diagnosed patients unfit for intensive chemotherapy.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules that keep cancer cells in the bone marrow. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers.

About Rivipansel

rivipansel, the most advanced drug candidate in the GlycoMimetics pipeline, is a glycomimetic drug candidate that acts as a pan-selectin antagonist, meaning it binds to all three members of the selectin family – E-, P- and L-selectin. The first potential indication for rivipansel is vaso-occlusive crisis (VOC) of sickle cell disease (SCD), one of the most severe complications of SCD which can result in acute ischemic organ injury at one or more sites. By reducing cell adhesion, activation and inflammation that are believed to contribute to reduced blood flow through the microvasculature during VOC, GlycoMimetics believes that rivipansel could be the first drug to interrupt the underlying cause of VOC, thereby potentially enabling patients to leave the hospital more quickly. Pfizer is conducting a Phase 3 clinical trial for rivipansel in SCD.