On February 14, 2018 Amgen (NASDAQ:AMGN) reported that the Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review data supporting the BLINCYTO (blinatumomab) supplemental Biologics License Application (sBLA) for the treatment of patients with minimal residual disease (MRD)-positive B-cell precursor acute lymphoblastic leukemia (ALL) at a meeting on March 7, 2018 (Press release, Amgen, FEB 14, 2018, View Source;p=RssLanding&cat=news&id=2332494 [SID1234523963]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

MRD refers to the presence of a small amount of detectible cancer cells that remain in the patient after treatment.

"After achieving remission, the presence of MRD is the strongest prognostic factor for relapse in acute lymphoblastic leukemia. However, today up to half of patients remain MRD-positive after induction treatments and receive limited clinical benefit from treatments like chemotherapy or stem cell transplantation as a result of failure to identify and treat this residual disease," said David M. Reese, M.D., senior vice president of Translational Sciences and Oncology at Amgen. "Currently there are no approved therapies for patients with MRD-positive ALL, representing a significant unmet need. This sBLA for BLINCYTO is the first application to ever be submitted for an MRD positive indication, and we look forward to discussing the supporting data with members of the Committee."

The Committee will review results from clinical studies in support of this potential new indication, including results from the Phase 2 BLAST study evaluating patients with B-cell precursor ALL and persistent or recurrent MRD after at least three cycles of intensive chemotherapy.

BLINCYTO is the first-and-only approved bispecific CD19-directed CD3 T cell engager (BiTE) immunotherapy. It is also the first bispecific antibody construct from Amgen’s BiTE platform, which helps the body’s immune system target cancer cells and represents an entirely new area of oncology research.

The sBLA for BLINCYTO was accepted by the FDA for priority review, and a Prescription Drug User Fee Act (PDUFA) target action date of March 29, 2018 has been set.

About BLINCYTO (blinatumomab)
BLINCYTO is a bispecific CD19-directed CD3 T cell engager (BiTE) antibody construct that binds specifically to CD19 expressed on the surface of cells of B-lineage origin and CD3 expressed on the surface of T cells. BLINCYTO was granted breakthrough therapy and priority review designations by the FDA in 2014 for the treatment of relapsed or refractory B-cell precursor ALL in adults and children, and is now approved in the U.S. for this indication.

In November 2015, BLINCYTO was granted conditional marketing authorization in the European Union for the treatment of adults with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor ALL. Additional regulatory applications for BLINCYTO are underway and have been submitted to health authorities worldwide.

About the BLAST Study
The BLAST study is the largest prospective trial in patients with MRD-positive ALL. It is an open-label, multicenter, confirmatory single-arm, Phase 2 study evaluating the efficacy, safety and tolerability of BLINCYTO in adult patients with MRD-positive B-cell precursor ALL in complete hematologic remission after three or more cycles of intensive chemotherapy treatments and a presence of MRD. Patients received continuous IV infusion of 15 μg/m2/d for four weeks, followed by two weeks off. Patients received up to four cycles of treatment, or could undergo a hematopoietic stem cell transplantation (HSCT) at any time after the first cycle, if eligible. The primary endpoint was the rate of complete MRD response within the first treatment cycle. The key secondary endpoint was relapse-free survival at 18 months. Additional secondary endpoints include incidence and severity of adverse events, overall survival (OS), time to hematological remission and duration of complete MRD response. Results from the BLAST study were presented at the 57th Annual Meeting and Exposition of the American Society of Hematology (ASH) (Free ASH Whitepaper) in 2015.

About ALL and MRD
ALL is a rare and rapidly progressing cancer of the blood and bone marrow that occurs in both adults and children.1,2 Many adult patients with ALL relapse, often within one year of their diagnosis, from which the median survival is only 4 to 8 months.3 Poor outcomes have been observed in patients who relapse after achieving a complete response but have persistent MRD, with 5-year OS rates as high as 75 percent for patients that achieve MRD-negative status, compared with 33 percent among patients that remain MRD-positive.4 In pediatric patients, MRD-positive status after treatment is associated with a 15-times higher risk of relapse compared with those with undetectable residual disease.5

About BiTE Technology
Bispecific T cell engager (BiTE) antibody constructs are a type of immunotherapy being investigated for fighting cancer by helping the body’s immune system to detect and target malignant cells. The modified antibodies are designed to bridge T cells to tumor cells, using the patient’s own immune system to eradicate their cancer. BiTE antibody constructs help place the T cells within reach of the targeted cell, with the intent of allowing T cells to inject toxins and trigger the cancer cell to die (apoptosis). BiTE antibody constructs are currently being investigated for their potential to treat a wide variety of cancers. For more information, visit www.biteantibodies.com.

BLINCYTO U.S. Product Safety Information

Indication and Important Safety Information, including Boxed WARNINGS, for BLINCYTO (blinatumomab) for injection, for intravenous use

INDICATION

BLINCYTO is indicated for the treatment of relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) in adults and children.

IMPORTANT SAFETY INFORMATION

WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGICAL TOXICITIES

Cytokine Release Syndrome (CRS), which may be life-threatening or fatal, occurred in patients receiving BLINCYTO. Interrupt or discontinue BLINCYTO as recommended.
Neurological toxicities, which may be severe, life-threatening or fatal, occurred in patients receiving BLINCYTO. Interrupt or discontinue BLINCYTO as recommended.
Contraindications
BLINCYTO is contraindicated in patients with a known hypersensitivity to blinatumomab or to any component of the product formulation.

Warnings and Precautions

Cytokine Release Syndrome (CRS): CRS, which may be life-threatening or fatal, occurred in patients receiving BLINCYTO. Infusion reactions have occurred and may be clinically indistinguishable from manifestations of CRS. Closely monitor patients for signs and symptoms of serious events such as pyrexia, headache, nausea, asthenia, hypotension, increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), increased total bilirubin (TBILI), disseminated intravascular coagulation (DIC), capillary leak syndrome (CLS), and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS). Interrupt or discontinue BLINCYTO as outlined in the Prescribing Information (PI).
Neurological Toxicities: Approximately 65% of patients receiving BLINCYTO in clinical trials experienced neurological toxicities. The median time to the first event was within the first 2 weeks of BLINCYTO treatment and the majority of events resolved. The most common (≥ 10%) manifestations of neurological toxicity were headache and tremor. Severe, life-threatening, or fatal neurological toxicities occurred in approximately 13% of patients, including encephalopathy, convulsions, speech disorders, disturbances in consciousness, confusion and disorientation, and coordination and balance disorders. Manifestations of neurological toxicity included cranial nerve disorders. Monitor patients for signs or symptoms and interrupt or discontinue BLINCYTO as outlined in the PI.
Infections: Approximately 25% of patients receiving BLINCYTO experienced serious infections, some of which were life-threatening or fatal. Administer prophylactic antibiotics and employ surveillance testing as appropriate during treatment. Monitor patients for signs or symptoms of infection and treat appropriately, including interruption or discontinuation of BLINCYTO as needed.
Tumor Lysis Syndrome (TLS): TLS, which may be life-threatening or fatal, has been observed. Preventive measures, including pretreatment nontoxic cytoreduction and on-treatment hydration, should be used during BLINCYTO treatment. Monitor patients for signs and symptoms of TLS and interrupt or discontinue BLINCYTO as needed to manage these events.
Neutropenia and Febrile Neutropenia, including life-threatening cases, have been observed. Monitor appropriate laboratory parameters during BLINCYTO infusion and interrupt BLINCYTO if prolonged neutropenia occurs.
Effects on Ability to Drive and Use Machines: Due to the possibility of neurological events, including seizures, patients receiving BLINCYTO are at risk for loss of consciousness, and should be advised against driving and engaging in hazardous occupations or activities such as operating heavy or potentially dangerous machinery while BLINCYTO is being administered.
Elevated Liver Enzymes: Transient elevations in liver enzymes have been associated with BLINCYTO treatment with a median time to onset of 3 days. In patients receiving BLINCYTO, although the majority of these events were observed in the setting of CRS, some cases of elevated liver enzymes were observed outside the setting of CRS, with a median time to onset of 19 days. Grade 3 or greater elevations in liver enzymes occurred in 7% of patients outside the setting of CRS and resulted in treatment discontinuation in less than 1% of patients. Monitor ALT, AST, gamma-glutamyl transferase (GGT), and TBILI prior to the start of and during BLINCYTO treatment. BLINCYTO treatment should be interrupted if transaminases rise to > 5 times the upper limit of normal (ULN) or if TBILI rises to > 3 times ULN.
Pancreatitis: Fatal pancreatitis has been reported in patients receiving BLINCYTO in combination with dexamethasone in clinical trials and the post-marketing setting. Evaluate patients who develop signs and symptoms of pancreatitis and interrupt or discontinue BLINCYTO and dexamethasone as needed.
Leukoencephalopathy: Although the clinical significance is unknown, cranial magnetic resonance imaging (MRI) changes showing leukoencephalopathy have been observed in patients receiving BLINCYTO, especially in patients previously treated with cranial irradiation and antileukemic chemotherapy.
Preparation and administration errors have occurred with BLINCYTO treatment. Follow instructions for preparation (including admixing) and administration in the PI strictly to minimize medication errors (including underdose and overdose).
Immunization: Vaccination with live virus vaccines is not recommended for at least 2 weeks prior to the start of BLINCYTO treatment, during treatment, and until immune recovery following last cycle of BLINCYTO.
Risk of Serious Adverse Reactions in Pediatric Patients due to Benzyl Alcohol Preservative: Serious and fatal adverse reactions including "gasping syndrome," which is characterized by central nervous system depression, metabolic acidosis, and gasping respirations, can occur in neonates and infants treated with benzyl alcohol-preserved drugs including BLINCYTO (with preservative). When prescribing BLINCYTO (with preservative) for pediatric patients, consider combined daily metabolic load of benzyl alcohol from all sources including BLINCYTO (with preservative) and other drugs containing benzyl alcohol. The minimum amount of benzyl alcohol at which serious adverse reactions may occur is not known. Due to the addition of bacteriostatic saline, 7-day bags of BLINCYTO solution for infusion with preservative contain benzyl alcohol and are not recommended for use in any patients weighing < 22 kg.
Adverse Reactions

The most common adverse reactions in Philadelphia chromosome-negative relapsed or refractory B-cell precursor ALL (TOWER Study) (≥ 20%) in the BLINCYTO arm were infections (bacterial and pathogen unspecified), pyrexia, headache, infusion-related reactions, anemia, febrile neutropenia, thrombocytopenia, and neutropenia. Serious adverse reactions were reported in 62% of patients. The most common serious adverse reactions (≥ 2%) included febrile neutropenia, pyrexia, sepsis, pneumonia, overdose, septic shock, CRS, bacterial sepsis, device related infection, and bacteremia.
Adverse reactions that were observed more frequently (≥ 10%) in the pediatric population compared to the adult population were pyrexia (80% vs. 61%), hypertension (26% vs. 8%), anemia (41% vs. 24%), infusion-related reaction (49% vs. 34%), thrombocytopenia (34% vs. 21%), leukopenia (24% vs. 11%), and weight increased (17% vs. 6%).
In pediatric patients less than 2 years old (infants), the incidence of neurologic toxicities was not significantly different than for the other age groups, but its manifestations were different; the only event terms reported were agitation, headache, insomnia, somnolence, and irritability. Infants also had an increased incidence of hypokalemia (50%) compared to other pediatric age cohorts (15-20%) or adults (17%).
Dosage and Administration Guidelines

BLINCYTO is administered as a continuous intravenous infusion at a constant flow rate using an infusion pump which should be programmable, lockable, non-elastomeric, and have an alarm.
It is very important that the instructions for preparation (including admixing) and administration provided in the full Prescribing Information are strictly followed to minimize medication errors (including underdose and overdose).
Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide, for BLINCYTO.

About Amgen’s Commitment to Oncology
Amgen Oncology is committed to helping patients take on some of the toughest cancers, such as those that have been resistant to drugs, those that progress rapidly through the body and those where limited treatment options exist. Amgen’s supportive care treatments help patients combat certain side effects of strong chemotherapy, and our targeted medicines and immunotherapies focus on more than a dozen different malignancies, ranging from blood cancers to solid tumors. With decades of experience providing therapies for cancer patients, Amgen continues to grow its portfolio of innovative and biosimilar oncology medicines.

On February 14, 2018 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2017 (Press release, Agios Pharmaceuticals, FEB 14, 2018, View Source [SID1234523961]). In addition, Agios highlighted select 2018 corporate milestones and data presentations for its clinical development programs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2017 was an extraordinary year for Agios with the U.S. approval of IDHIFA, our first internally discovered and developed drug, the NDA submission for our wholly owned medicine ivosidenib and our sixth IND submission since the company’s inception," said David Schenkein, M.D., chief executive officer at Agios. "It was a data-rich year where we set the stage for building long-term value across our cancer and rare disease portfolios. Execution in 2018 will be equally critical as we ready our organization for the potential approval and launch of ivosidenib, initiate our pivotal program for AG-348 in pyruvate kinase deficiency and advance our robust discovery portfolio."

KEY UPCOMING MILESTONES

The company plans to achieve the following key milestones in 2018:

Cancer:

Potential approval and commercialization of ivosidenib in the United States for relapsed/refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation in the third quarter of 2018.
Potential submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for ivosidenib for IDH1m R/R AML in the fourth quarter of 2018.
Support, in conjunction with Celgene, the initiation of an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.
Initiate a perioperative ‘window’ trial with ivosidenib and AG-881 in low-grade glioma in the first quarter of 2018 to further investigate their effects on brain tumor tissue.
Initiate a Phase 1 dose-escalation trial for AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in patients with methylthioadenosine phosphorylase (MTAP)-deleted tumors in the first quarter of 2018.
Rare Genetic Diseases:

Initiate two global pivotal trials for AG-348 in pyruvate kinase (PK) deficiency in the first half of 2018:
ACTIVATE-T: A single arm trial of approximately 20 regularly transfused patients is expected to initiate in the first quarter of 2018.
ACTIVATE: A placebo-controlled trial of approximately 80 patients who do not receive regular transfusions is expected to initiate in the second quarter of 2018.
Initiate a global registry, known as PEAK, for adult and pediatric patients with PK deficiency in the first quarter of 2018.
Initiate a Phase 2 proof of concept trial of AG-348 in thalassemia in the fourth quarter of 2018.
Research:

Submit an investigational new drug (IND) application for our newest development candidate, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
ANTICIPATED KEY 2018 DATA PRESENTATIONS

Updated data from the expansion phase of the ongoing Phase 1 study of ivosidenib in IDH1m R/R AML has been submitted to the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Updated data from the ongoing Phase 1/2 combination trial of enasidenib or ivosidenib with VIDAZA in patients with newly diagnosed AML with an IDH2 or IDH1 mutation ineligible for intensive chemotherapy has been submitted to ASCO (Free ASCO Whitepaper).
First clinical data from the Phase 1 study of AG-881 in advanced IDHm positive solid tumors, including glioma, has been submitted to ASCO (Free ASCO Whitepaper).
Updated data from the ongoing Phase 1 combination trial of enasidenib or ivosidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation to be submitted to the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
FOURTH QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS

Completed an underwritten public offering in January of 8,152,986 shares of common stock at the offering price of $67.00 per share, resulting in proceeds, net of underwriting discounts and commissions, of approximately $516.2 million.
Submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for ivosidenib for the treatment of patients with R/R AML with an IDH1 mutation.
Received FDA clearance of an IND application for AG-270, a MAT2A inhibitor, for the treatment of MTAP-deleted tumors.
Presented new and updated data from the IDH and PKR programs at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper):
First data from the expansion phase of the ongoing Phase 1 trial of ivosidenib in IDH1m R/R AML and advanced hematologic malignancies
First data from the ongoing Phase 1 combination trial of ivosidenib or enasidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH1 or IDH2 mutation
First data from the ongoing Phase 1/2 combination trial of ivosidenib or enasidenib with VIDAZA in patients with newly diagnosed AML with an IDH1 or IDH2 mutation ineligible for intensive chemotherapy
Updated data from the AG-348 Phase 2 DRIVE PK study in PK deficiency
Appointed Jacqualyn "Jackie" Fouse, Ph.D., former president and chief financial officer of Celgene, to Agios’ board of directors.
Presented updated data from the glioma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors at the 2017 Society for NeuroOncology Annual Meeting.
FULL YEAR 2017 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of December 31, 2017 were $567.8 million, compared to $573.6 million as of December 31, 2016. The decrease in cash was driven by expenditures to fund operations of $306.8 million during the year ended December 31, 2017. These expenditures were offset by an increase in cash driven by net proceeds of $270.2 million from the April follow on offering, $17.0 million of cost reimbursements under our collaboration agreements with Celgene and $14.2 million received from employee stock transactions.

Revenue for the year ended December 31, 2017 was $43.0 million, which includes $41.1 million of collaboration revenue and $1.9 million of royalty revenue from net sales of IDHIFA. Revenue for the year ended December 31, 2016 was $69.9 million, which included a $25.0 million milestone payment related to the initiation of the Phase 3 IDHENTIFY trial with IDHIFA under the 2010 Agreement.

Research and development (R&D) expenses were $292.7 million, including $30.8 million of stock-based compensation expense, for the year ended December 31, 2017, compared to $220.2 million, including $25.4 million in stock-based compensation expense, for the year ended December 31, 2016. The increase in R&D expense was primarily attributable to the ivosidenib program, including manufacturing and regulatory activities to prepare the NDA submission, start-up costs for the Phase 3 AGILE clinical trial, and on-going site activation and patient enrollment of the Phase 3 ClarIDHy clinical trial. R&D expense also increased compared to the prior year due to IND enabling activities for AG-270.

General and administrative (G&A) expenses were $71.1 million, including $17.0 million of stock-based compensation expense, for the year ended December 31, 2017, compared to $50.7 million, including $16.7 million of stock-based compensation expense, for the year ended December 31, 2016. The increase in G&A expense was primarily attributable to an increase of $21.1 million to support our growing commercial organization for the launch of IDHIFA and the potential launch of ivosidenib in 2018.

Net loss for the year ended December 31, 2017 was $314.7 million, compared to a net loss of $198.5 million for the year ended December 31, 2016.

CASH GUIDANCE

In January, Agios completed an underwritten public offering of 8,152,986 shares of common stock, which includes the full exercise of the underwriters’ option to purchase an additional 1,063,433 shares, at the offering price of $67.00 per share, resulting in proceeds, net of underwriting discounts and commissions, of approximately $516.2 million.

The company expects that its cash, cash equivalents and marketable securities as of December 31, 2017, together with the net proceeds from the recent financing, anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss fourth quarter and full year 2017 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 3198522. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On February 14, 2018 Athenex, Inc. (Nasdaq:ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported early completion of patient enrollment for both Phase III clinical studies of KX2-391 Ointment for actinic keratosis indications months ahead of schedule (Press release, Athenex, FEB 14, 2018, View Source;p=RssLanding&cat=news&id=2332390 [SID1234523964]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Actinic keratosis is a common skin condition that is induced through ultra-violet light damage, resulting in patches of thick, scaly, or crusty skin. Left untreated, the lesions have risk of progression to squamous cell carcinoma and consequently treatment by a dermatologist is recommended. Actinic keratosis is the most common pre-cancerous condition in dermatology and it affects more than 55 million Americans. Actinic keratosis constitutes between 14-29% of dermatologist visits in the USA.1

KX2-391, also known as KX-01, is a dual Src kinase and tubulin polymerization inhibitor and a first-in-class topical treatment of actinic keratosis. Phase I and Phase II clinical studies showed excellent efficacy and safety results. After the FDA end of Phase II meeting, two randomized double-blind controlled Phase III trials totaling 600 patients were initiated in September 2017 in the United States and completed patient recruitment in February 2018.

Dr. Rudolf Kwan, Athenex’s Chief Medical Officer, commented, "KX2-391 ointment, given its excellent clinical efficacy and safety profile compared to existing medical therapy, has the potential to change the paradigm of topical therapy for actinic keratosis. We are delighted to see that our investigators and patients are also seeing the potential of this new treatment as indicated by the rapid completion of patient enrollment in around four months which is extremely fast, and three months ahead of our original schedule. Such rapid patient recruitment rates in clinical trials underscores the excitement of the clinical and scientific communities."

Dr. Johnson Lau, Athenex’s Chief Executive Officer and Chairman of the Board, stated, "This excellent milestone highlights the quality of our clinical team members and operation. Our previous commitment to build an internal clinical operation will also ensure that the clinical trial monitoring and management, database management, and biostatistics will not be dependent on outside vendors. As such, we are confident that all aspects of treatment, follow up, monitoring, and database will be completed on time. We are now expecting topline data to be available in third quarter of 2018. We also look forward to presenting our Phase II data to the public in the upcoming American Academy of Dermatology meeting on February 17, 2018."

As previously announced on December 11, 2017, Athenex and Almirall, a leading skin-health focused global pharmaceutical company and one of the leaders in the field of actinic keratosis treatment, entered into a License Agreement in which Athenex granted Almirall an exclusive license under the Athenex Intellectual Property to research, develop and commercialize KX2-391 in the United States of America and European countries, including Russia. Athenex will receive an upfront fee and near-term payments of up to USD $55 million, and additional indications milestones payment and a royalty payment starting at 15% based on annual net sales, with incremental increases in royalty rates with increased sales. Athenex retains certain co-promotion rights in the USA and retains the rights for other parts of the world including Canada, Central and South America, Japan, Asia and China, Australia and New Zealand, and Africa including South Africa. Almirall will employ its expertise to support the development in Europe and also to commercialize the product in the defined territories. Milestones were established to encourage the joint effort of Athenex and Almirall to develop additional indications and additional formulations.

Peter Guenter, Chief Executive Officer at Almirall, said, "We are impressed by the Athenex team in their execution of drug development. KX2-391, given its efficacy and very good safety profile compared to existing medical therapies, has the potential to change the standard of care for actinic keratosis, as well as expand the market since many patients remain undiagnosed or untreated. Almirall’s experience in Europe, currently as leaders in actinic keratosis, and our presence in the United States will help to develop and commercialize this product. We are now expecting top line Phase III data to be available in the third quarter of 2018. We also look forward to the presentation of the Phase II data to the public in the upcoming American Academy of Dermatology meeting on February 17, 2018."

References
1. E. Stockfleth et al. Physician perceptions and experience of current treatment in actinic keratosis. JEADV 2015, 29, 298–306

On February 14, 2018 Alkermes plc (Nasdaq: ALKS) today reported financial results for the twelve months ended Dec. 31, 2017 and provided financial expectations for 2018 (Press release, Alkermes, FEB 14, 2018, View Source [SID1234523962]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Quarter Ended Dec. 31, 2017 Financial Highlights

•Total revenues for the quarter were $275.4 million. This compared to $213.5 million for the same period in the prior year, representing an increase of 29%.

•Net loss according to generally accepted accounting principles in the U.S. (GAAP) was $9.8 million, or a basic and diluted GAAP loss per share of $0.06. This compared to GAAP net loss of $21.1 million, or a basic and diluted GAAP loss per share of $0.14 for the same period in the prior year.

•Non-GAAP net income was $50.3 million, or a non-GAAP basic earnings per share of $0.33 and non-GAAP diluted earnings per share of $0.31, for the quarter. This compared to non-GAAP net income of $23.3 million, or a non-GAAP basic and diluted earnings per share of $0.15, for the same period in the prior year.

Quarter Ended Dec. 31, 2017 Financial Results

Revenues

•Net sales of VIVITROL were $75.6 million, compared to $62.1 million for the same period in the prior year, representing an increase of 22%.

•Net sales of ARISTADA were $28.3 million, compared to $17.3 million for the same period in the prior year, representing an increase of 64%.

•Manufacturing and royalty revenues from RISPERDAL CONSTA, INVEGA SUSTENNA/XEPLION and INVEGA TRINZA/TREVICTA were $78.2 million, compared to $74.0 million for the same period in the prior year.

•Manufacturing and royalty revenues from AMPYRA/FAMPYRA1 were $38.1 million, compared to $32.3 million for the same period in the prior year.

•Revenues from the collaboration with Biogen for BIIB098 (formerly ALKS 8700) included $28.0 million of license revenue and $2.3 million of R&D reimbursement.

Costs and Expenses

•Operating expenses were $269.5 million, compared to $237.1 million for the same period in the prior year.

•Income tax provision of $20.6 million included a $21.5 million tax charge related to the reduction in the value of our deferred tax assets due to the enactment of the Tax Cuts and Jobs Act of 2017. This compared to an income tax benefit of $3.2 million for the same period in the prior year.

Calendar Year 2017 Financial Highlights

•Total revenues increased 21% to $903.4 million in 2017, which included VIVITROL net sales of $269.3 million and ARISTADA net sales of $93.5 million. This compared to total revenues of $745.7 million for 2016, which included VIVITROL net sales of $209.0 million and ARISTADA net sales of $47.2 million. Please see the tables at the end of this press release for a detailed breakdown of the revenues from our key commercial products.

•GAAP net loss was $157.9 million, or a basic and diluted GAAP loss per share of $1.03, for 2017. This compared to a GAAP net loss of $208.4 million, or a basic and diluted GAAP loss per share of $1.38, for 2016.

•Non-GAAP net income was $27.8 million, or a non-GAAP basic earnings per share of $0.18 and non-GAAP diluted earnings per share of $0.17, for 2017. This compared to non-GAAP net loss of $10.3 million, or a non-GAAP basic and diluted loss per share of $0.07, for 2016.

•At Dec. 31, 2017, Alkermes recorded cash, cash equivalents and total investments of $590.7 million, compared to $619.2 million at Dec. 31, 2016. At Dec. 31, 2017, the company’s total debt outstanding was $281.4 million, compared to $283.7 million at Dec. 31, 2016.

"Our financial results in 2017 were driven by the strong year-over-year growth of our proprietary products, VIVITROL and ARISTADA, our base royalty and manufacturing business and our strategic alliance with Biogen for BIIB098. We exited the year well positioned to execute on our key strategic initiatives in 2018," commented James Frates, Chief Financial Officer of Alkermes. "Looking ahead, 2018 will be a transformative year for Alkermes. Our financial expectations for 2018 reflect important investments that will drive future value as we advance our late-stage pipeline and prepare for the anticipated launch of ALKS 5461, with the planned expansion of our commercial organization midyear. We also expect solid growth for VIVITROL and ARISTADA, and remain committed to driving awareness and advancing the treatment paradigm for these important medicines."

Recent Events:

•ALKS 5461: In January 2018, Alkermes submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ALKS 5461 for the adjunctive treatment of major depressive disorder (MDD). ALKS 5461 was granted Fast Track status by the FDA in October 2013 for the adjunctive treatment of MDD in patients with an inadequate response to standard antidepressant therapies.

•BIIB098: Biogen and Alkermes announced a license and collaboration agreement for the development and commercialization of BIIB098 for the treatment of relapsing forms of Multiple Sclerosis (MS). Under the terms of the agreement, Alkermes granted Biogen an exclusive, worldwide license to develop and commercialize BIIB098 and Biogen will pay Alkermes a mid-teens royalty on worldwide net sales of BIIB098. Biogen is responsible for all development and commercialization expenses, effective Jan. 1, 2018. Alkermes may also receive milestone payments for BIIB098 with a maximum aggregate value of $200 million upon certain clinical and regulatory achievements.

•ARISTADA: A NDA was filed with the FDA for Aripiprazole Lauroxil NanoCrystal Dispersion (ALNCD), a novel, investigational product designed for initiation onto ARISTADA. A target action date of June 30, 2018 was assigned to the ALNCD NDA under the Prescription Drug User Fee Act (PDUFA).

•VIVITROL: Results from the National Institute on Drug Abuse (NIDA)-funded X:BOT study, comparing extended-release naltrexone (VIVITROL) and buprenorphine-naloxone, were published in The Lancet. Data from the study demonstrated that, once treatment was initiated, both medications were equally safe and effective in preventing relapse to opioid dependence.

•ALKS 4230: Preclinical data on ALKS 4230 was presented at the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. The data showed that treatment with ALKS 4230 significantly delayed tumor growth and led to accumulation of tumor-killing T cells in the tumor microenvironment in individualized and humanized melanoma xenograft models of tumor immunology.

2

"We are making significant progress in advancing our pipeline of late-stage CNS development candidates, highlighted by the recent submission of the ALKS 5461 NDA, the recently announced license and collaboration agreement with Biogen to develop and commercialize BIIB098, and as we near completion of enrollment in the six-month pivotal weight study for ALKS 3831," said Richard Pops, Chief Executive Officer of Alkermes. "Alkermes has worked for many years toward this moment. With a diversified CNS business and significant news flow expected across our pipeline of development candidates in 2018, we are focused on executing on our business strategy to bring these important potential new medicines to patients and creating value for our shareholders."

2018 Expected Milestones

The following outlines the company’s expected milestones for 2018. The following statements are forward-looking, and actual results may differ materially. Please see "Note Regarding Forward-Looking Statements" at the end of this press release for risks that could cause results to differ materially from these forward-looking statements.

•VIVITROL

oPublication and presentation of detoxification protocols for induction onto VIVITROL from recent clinical trials (H1)

•ARISTADA

oALNCD for initiation onto ARISTADA PDUFA target action date (June 30, 2018)

•ALKS 5461

oAssignment of PDUFA target action date, following NDA acceptance (Q2)

oPotential Advisory Committee meeting and FDA action (H2)

•ALKS 3831

oENLIGHTEN-2 six-month weight study enrollment completion (Q1)

oPhase 1 human metabolic study data presentation (H1)

oENLIGHTEN-2 topline results (Fall)

•BIIB098 (formerly ALKS 8700)

oPotential receipt of $50 million payment following initial data from EVOLVE-MS-2 gastrointestinal head-to-head study (mid-2018)

oPlanned NDA submission for treatment of MS (H2)

•ALKS 4230

oDose escalation data and dose expansion initiation (H2)

oPlanned submission of Investigational New Drug (IND) application for subcutaneous dosing phase 1 study (H2)

Financial Expectations for 2018

The following outlines the company’s financial expectations for 2018, which include investments in commercial infrastructure in preparation for the expected launch of ALKS 5461 and in the company’s pipeline of late-stage development candidates. The following statements are forward-looking, and actual results may differ materially. Please see "Note Regarding Forward-Looking Statements" at the end of this press release for risks that could cause results to differ materially from these forward-looking statements.

•Revenues: The company expects total revenues to range from $975 million to $1.025 billion, driven by continuing growth of VIVITROL and ARISTADA. Included in this total revenue expectation, Alkermes expects VIVITROL net sales to range from $300 million to $330 million, and ARISTADA net sales to range from $140 million to $160 million.

•Cost of Goods Manufactured and Sold: The company expects cost of goods manufactured and sold to range from $180 million to $190 million.

•Research and Development (R&D) Expenses: The company expects R&D expenses to range from $415 million to $445 million.

•Selling, General and Administrative (SG&A) Expenses: The company expects SG&A expenses to range from $555 million to $585 million. This increase reflects important planned investment in the

3

expansion of our commercial organization in preparation for the anticipated launch of ALKS 5461.

•Amortization of Intangible Assets: The company expects amortization of intangibles to be approximately $65 million.

•Net Interest Expense: The company expects net interest expense to be approximately $10 million.

•Income Tax Expense: The company expects income tax expense of up to $10 million.

•GAAP Net Loss: The company expects GAAP net loss to range from $250 million to $280 million, or a basic and diluted loss per share of $1.61 to $1.81, based on a weighted average basic and diluted share count of approximately 155 million shares outstanding.

•Non-GAAP Net Loss: The company expects non-GAAP net loss to range from $5 million to $35 million, or a non-GAAP basic and diluted loss per share of $0.03 to $0.23, based on a weighted average basic and diluted share count of approximately 155 million shares outstanding.

•Capital Expenditures: The company expects capital expenditures to range from $80 million to $90 million.

Conference Call

Alkermes will host a conference call at 8:30 a.m. ET (1:30 p.m. GMT) on Wednesday, Feb. 14, 2018, to discuss these financial results and provide an update on the company. The conference call may be accessed by visiting Alkermes’ website or by dialing +1 888 424 8151 for U.S. callers and +1 847 585 4422 for international callers. The conference call ID number is 6037988. In addition, a replay of the conference call will be available from 11:00 a.m. ET (4:00 p.m. GMT) on Wednesday, Feb. 14, 2018, through 5:00 p.m. ET (10:00 p.m. GMT) on Wednesday, Feb. 21, 2018, and may be accessed by visiting Alkermes’ website or by dialing +1 888 843 7419 for U.S. callers and +1 630 652 3042 for international callers. The replay access code is 6037988.

On February 14, 2018 DelMar Pharmaceuticals, Inc. (NASDAQ: DMPI) ("DelMar" or the "Company"), a biopharmaceutical company focused on the development of new cancer therapies, reported its financial results for the second quarter ended December 31, 2017 (Press release, DelMar Pharmaceuticals, FEB 14, 2018, View Source [SID1234523988]). DelMar executive management will host a business update conference call for investors, analysts and other interested parties on Tuesday, February 20, 2018 at 4:30 p.m. Eastern Standard Time.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This quarter has been an exciting period for DelMar. Our priority is to leverage VAL-083’s unique mechanism of action to efficiently advance it into the most promising indications, including MGMT-unmethylated glioblastoma and platinum-resistant ovarian cancer. We now have a revised VAL-083 development strategy that is focused on MGMT methylation status in glioblastoma, which has become routine in clinical practice as a biomarker which correlates with resistance to the standard-of-care chemotherapy with temozolomide (Temodar "TMZ"), and patient outcomes. We believe using this biomarker will allow us to optimize patient selection for treatment with our lead drug candidate, VAL-083, thereby streamlining development and enhancing opportunities for success in our clinical development programs," commented Saiid Zarrabian, Interim President and Chief Executive Officer.

KEY DEVELOPMENTS AND UPDATED STRATEGIC PLAN

● Evaluation of MGMT promoter methylation status has increasingly become common practice in the diagnostic assessment of glioblastoma multiforme (GBM). DelMar believes that this provides it with an enhanced ability to leverage MGMT methylation as a biomarker to optimize patient selection for DelMar’s novel DNA-targeting agent in the treatment of GBM.

● The National Comprehensive Cancer Network (NCCN), provided updated guidelines for the standard treatment of GBM based on MGMT methylation status. DelMar believes these recently published guidelines may allow the Company to capitalize on VAL-083’s unique mechanism of action and activity in the estimated 60 percent of GBM patients whose tumors are MGMT-unmethylated.

● The U.S. Food and Drug Administration (FDA) allowed a second Investigational New Drug Application (IND) to enable DelMar to study its lead drug candidate, VAL-083, as a potential treatment for ovarian cancer.

● In November 2017, at the annual meeting of the Society for NeuroOncology (SNO), DelMar presented a positive interim update from its ongoing open label Phase 2 clinical trial in patients with MGMT-unmethylated recurrent GBM (rGBM) whose tumors have recurred following treatment with temozolomide (Avastin naïve). This study, which was initiated in February 2017, is being conducted at the University of Texas MD Anderson Cancer Center.

● In December 2017, the FDA fully approved Avastin (bevacizumab) which may impact our ability to recruit suitable patients for our STAR-3 Phase 3 clinical trial.

● In December 2017, the FDA granted Fast Track designation for VAL-083, in recurrent glioblastoma.

● Based on the above developments, and other factors as stated in DelMar’s Quarterly Report on Form 10-Q for the quarter ended December 31, 2017 (10-Q) filed with the Securities and Exchange Commission (SEC) on February 14, 2018, DelMar has decided to put the STAR-3 program on hold for up to 12 months and will suspend further site or patient enrollment. This will allow DelMar to fully evaluate the possible impact of Avastin’s recent approval by the FDA on patient enrollment for this study, and possible protocol amendments, non-dilutive financing sources, as well as to increase focus on the MGMT-unmethylated clinical studies currently underway as further described in the SEC filings. During this interim evaluation period, DelMar will continue to provide treatment to patients already enrolled in the STAR-3 trial, and consider, on a case-by-case basis, and subject to required institutional and regulatory approvals, providing VAL-083 to patients in accordance with our expanded access policy

● Based on this updated strategy, DelMar believes it has cash available into the second quarter of calendar 2019.

For further details on the Company’s operating and financial results, as well as more detail about its updated strategy, refer to DelMar’s 10-Q filed with the SEC on February 14, 2018, View Source

CONFERENCE CALL DETAILS

DelMar plans to host a conference call to discuss its financial results for the quarter ended December 31, 2017 and provide a corporate update on Tuesday, February 20, 2018, at 4:30 p.m. Eastern Time. For both "listen-only" participants and those who wish to take part in the question and answer portion of the call, the telephone Dial-in Number is 1 888 632 3384 (toll free) with Conference ID DELMAR.

A replay of the conference call will be available on the IR Calendar of the Investors section of the Company’s website at www.delmarpharma.com and will be archived for 30 days.

SUMMARY OF FINANCIAL RESULTS FOR THE PERIOD ENDED DECEMBER 31, 2017

At December 31, 2017, the Company had cash and clinical trial deposits on hand of approximately $12.0 million (unaudited).

For the three months ended December 31, 2017, the Company reported a net loss of $3,161,598 or $0.14 per share, compared to a net loss of $1,321,973, or $0.13 per share, for the three months ended December 31, 2016. For the six months ended December 31, 2017, the Company reported a net loss of $5,828,004 or $0.31 per share, compared to a net loss of $3,612,312, or $0.36 per share, for the six months ended December 31, 2016.

-2-

The following represents selected financial information as of December 31, 2017. The Company’s financial information has been prepared in accordance with U.S. GAAP and this selected information should be read in conjunction with DelMar’s consolidated financial statements and management’s discussion and analysis ("MD&A"), as filed.

DelMar’s financial statements as filed with the U.S. Securities Exchange Commission can be viewed on the company’s website at: View Source

Selected Balance Sheet Data

December 31,
2017
$

June 30,
2017
$

Cash 11,021,568 6,586,014
Working capital 9,959,948 6,566,371
Total assets 12,216,116 7,911,021
Derivative liability 5,549 61,228
Total stockholders’ equity 9,983,574 6,578,524

Selected Statement of Operations Data

For the three months ended:

December 31, December 31,
2017 2016
$ $

Research and development 2,141,945 1,120,910
General and administrative 1,011,879 571,286
Change in fair value of stock option and derivative liabilities 889 (361,668 )
Foreign exchange loss (gain) 7,120 (8,495 )
Interest income (235 ) (60 )
Net and comprehensive loss for the period 3,161,598 1,321,973
Series B Preferred stock dividend 54,066 159,756
Net and comprehensive loss available to common stockholders 3,215,664 1,481,729
Basic weighted average number of shares outstanding 22,559,234 11,424,485
Basic and fully diluted loss per share 0.14 0.13

Excluding the impact of non-cash expense, research and development expenses increased to $2,015,570 during the current quarter compared to $1,186,637 for the same period in the prior year. The increase was largely attributable to an increase in clinical development costs related to the three clinical studies ongoing for VAL-083, as well as personnel, and preclinical research costs. Excluding the impact of non-cash expenses, general and administrative expenses increased in the quarter ended December 31, 2017 to $909,747 from $580,761 for the quarter ended December 31, 2016.

-3-

For the six months ended:

December 31, December 31,
2017 2016
$ $

Research and development 4,076,588 1,853,639
General and administrative 1,756,500 1,887,925
Change in fair value of stock option and derivative liabilities (55,679 ) (135,980 )
Foreign exchange loss 50,986 6,829
Interest income (391 ) (101 )
Net and comprehensive loss for the period 5,828,004 3,612,312
Series B Preferred stock dividend 95,732 467,054
Net and comprehensive loss available to common stockholders 5,923,736 4,079,366
Basic weighted average number of shares outstanding 18,882,259 11,363,237
Basic and fully diluted loss per share 0.31 0.36

Excluding the impact of non-cash expense, research and development expenses increased to $3,955,187 during the six months ended December 31, 2017 compared to $1,863,529 for the same period in the prior year. The increase was largely attributable to VAL-083 clinical development and manufacturing costs related to the Company’s STAR-3 refractory-GBM clinical trial and two Phase 2 clinical trials in MGMT-unmethylated GBM.

Excluding the impact of non-cash expenses, general and administrative expenses increased in the current six months to $1,586,005 compared to $1,307,175 for the six months ended December 31, 2016.

We believe, based on our current estimates, that we will be able to fund our operations into the second quarter of calendar year 2019.