On May 4, 2018 Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, reported an overview of its corporate activities and milestones, as well as revenues and cash position for the Nicox Group as of March 31, 2018 (Press release, NicOx, MAY 4, 2018, View Source [SID1234526113]).

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"The early months of 2018 marked a time of growth for Nicox, with the expansion and relocation of our U.S. development office to Research Triangle Park in North Carolina in preparation for the NCX 470 and NCX 4251 programs advancing to Phase 2 clinical testing," said Michele Garufi, Chairman and Chief Executive Officer of Nicox. "With respect to VYZULTATM, our partner Bausch + Lomb announced successfully securing the first reimbursement coverage with a key payor, a crucial step in expanding the reach of this innovative, much needed therapy. Through the efforts of our partner and the recently renegotiated financial terms of our global licensing agreement, VYZULTATM is a key meaningful value driver for Nicox."

First Quarter 2018 and Recent Operational Highlights

NCX 667 scientific data presented at the Association for Research in Vision and Ophthalmology (ARVO) 2018 Annual Meeting (April 29 – May 3, 2018). Yesterday, Nicox announced the presentation of strong preclinical data at ARVO 2018 demonstrating that NCX 667, a lead molecule among the Company’s future generation of stand-alone NO donors, lowers intraocular pressure (IOP) in a dose-dependent manner in various normotensive and hypertensive ocular models. The Company also presented novel mechanism of action data supporting the effect of NCX 667 on classical aqueous humor outflow, generated in an in vitro bioengineered human trabecular meshwork/Schlemm’s canal system.
U.S. development office relocated to Research Triangle Park. In April, Nicox announced the opening of its U.S. development office in Research Triangle Park (RTP) in North Carolina, USA. The decision to relocate from its prior development office in Fort Worth, Texas, and to expand the Company’s presence at RTP, is driven by the anticipated progress of NCX 470 for patients with glaucoma and NCX 4251 for patients with blepharitis towards Phase 2 clinical testing.
Secured improved financial terms for VYZULTATM licensing agreement with Bausch + Lomb. In March, the Company announced an amendment to its global licensing agreement with Bausch + Lomb Incorporated, a leading global eye health company and wholly owned subsidiary of Valeant Pharmaceuticals International Inc., for VYZULTATM (latanoprostene bunod ophthalmic solution), 0.024%. Under the amended terms, entered into in consideration of final resolution and release relating to certain alleged issues between the parties, royalties paid to Nicox on worldwide net sales of VYZULTATM will increase by 1% over the original royalty on Net Sales above $300 million per year. In addition, the potential milestones payable to Nicox by Bausch + Lomb have been increased by $20 million.
Tomas Navratil, Ph.D. appointed Vice President, Head of Development. In January, Nicox appointed Tomas Navratil, Ph.D. as Vice President, Head of Development. Tomas is responsible for leading all of the Company’s non-clinical, CMC, and clinical development activities.
Key Upcoming Milestones
ZERVIATETM expected to be launched in the U.S. by Eyevance Pharmaceuticals for the 2018 fall allergy season. Indicated for the treatment of ocular itching associated with allergic conjunctivitis, ZERVIATETM is the first and only topical ocular formulation of cetirizine. ZERVIATETM was approved by the U.S. FDA in May 2017. In September 2017, Nicox licensed exclusive U.S. commercial rights for ZERVIATETM to Eyevance Pharmaceuticals, LLC.
NCX 470 U.S. Investigational New Drug (IND) submission enabling Phase 2 clinical study in glaucoma patients planned in Q3 2018. NCX 470 is a novel second generation NO-donating prostaglandin analog in development for the reduction of IOP in patients with open-angle glaucoma or ocular hypertension.
NCX 4251 U.S. IND submission enabling Phase 2 clinical study in blepharitis patients planned in Q1 2019. NCX 4251 is a novel, patented ophthalmic formulation of fluticasone propionate being developed for the first time as a targeted topical treatment of the eyelids for patients with acute exacerbation of blepharitis. Blepharitis is a common ocular condition in which the edges of the eyelids become red and swollen, and may contain dandruff-like matter.
First Quarter 2018 Financial Highlights
As of March 31, 2018, the Group had cash and cash equivalents of €36.3 million as compared with €41.4 million at December 31, 2017. Net revenue for the first quarter of 2018 was €0.075 million, comprised exclusively of royalty revenue from early sales of VYZULTATM by global partner Bausch + Lomb, after deduction of Nicox’s royalty payments to Pfizer under a previous agreement signed in 2009. The Group recorded no revenues for the first quarter 2017.

On May 4, 2018 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported that it will present results from five studies at the Seventh International Symposium on Hereditary Breast and Ovarian Cancer annual meeting to be held May 8 to 11, 2018 in Montréal, Canada (Press release, Myriad Genetics, MAY 4, 2018, View Source [SID1234526136]). More information about the symposium can be found here: View Source

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"We look forward to presenting important new research on risk associated with breast and ovarian cancer gene mutations," said Johnathan Lancaster, M.D., Ph.D., chief medical officer, Myriad Genetics. "Our presentations will highlight Myriad’s ongoing commitment to research, advances to state-of-the-art hereditary cancer risk assessment and the highest quality of clinical testing."

About Myriad myRisk Hereditary Cancer

The Myriad myRisk Hereditary Cancer test uses an extensive number of sophisticated technologies and proprietary algorithms in an 850 step laboratory process to evaluate 28 clinically significant genes associated with eight hereditary cancer sites including: breast, colon, ovarian, endometrial, pancreatic, prostate and gastric cancers and melanoma. For more information visit: View Source

About riskScore

riskScore is a clinically validated personalized medicine tool that enhances Myriad’s myRisk Hereditary Cancer test. riskScore helps to further predict a women’s lifetime risk of developing breast cancer using clinical risk factors and genetic-markers throughout the genome. The test incorporates data from greater than 80 single nucleotide polymorphisms identified through 20 years of genome wide association studies in breast cancer and was validated in our laboratory to predict breast cancer risk in women of European descent. This data is then combined with a best-in-class family and personal history algorithm, the Tyrer-Cuzick model, to provide more patients with individualized breast cancer risk.

On May 4, 2018 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), reported a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, is hosting an Investor Day in New York City today (Press release, Agios Pharmaceuticals, MAY 4, 2018, View Source [SID1234526117]). During the event, the company will provide a comprehensive business update and report financial results for the first quarter ended March 31, 2018. The presentations will highlight how Agios’ drug discovery platform and broad clinical portfolio set Agios on the path to become a sustainable, multi-product biopharmaceutical company. The event will be webcast today starting at 8:00 a.m. ET at investor.agios.com.

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"As we prepare to launch our second Agios-discovered and first wholly owned medicine later this year, we continue to invest in our productive drug discovery engine and advance a robust pipeline of first-in-class medicines," said David Schenkein, M.D., chief executive officer at Agios. "Our first quarter progress against that objective was highlighted by the NDA acceptance of TIBSOVO in IDH1m relapsed or refractory AML and multiple clinical trial initiations, including dosing the first patient with our MAT2A inhibitor AG-270 and the start of our AG-348 pivotal program in PK deficiency."

HIGHLIGHTS FROM INVESTOR DAY PRESENTATIONS

Communicated a robust research pipeline consisting of nine advanced drug discovery programs against novel targets across oncology, rare genetic diseases and metabolic immuno-oncology with the potential to deliver multiple INDs over the next 24 months.
Expanded rare genetic disease portfolio:
The company disclosed active research programs in three rare genetic diseases: phenylketonuria (PKU), erythroid porphyria and Friedreich’s ataxia
The most advanced research program is in PKU, where Agios has developed a novel approach to stabilize the mutant phenylalanine hydroxylase (PAH) protein and has demonstrated significantly decreased blood phenylalanine levels in a severe pre-clinical model of the disease. PKU is an autosomal recessive disease caused by mutations in the PAH gene affecting approximately 16,000 patients in the U.S.1
Updated clinical milestones to advance the development of isocitrate dehydrogenase (IDH) 1 inhibitors in solid tumors:
Glioma pivotal development strategy expected to be finalized by year-end 2018
Completion of enrollment of ClarIDHy, a global, registration-enabling randomized Phase 3 study for ivosidenib in IDH1m positive advanced cholangiocarcinoma, accelerated to the first half of 2019
Announced acceptance of the following presentations at 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting:
Updated data from the expansion phase of the ongoing Phase 1 study of ivosidenib in IDH1m relapsed or refractory (R/R) acute myeloid leukemia (AML)
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
First clinical data from the Phase 1 study of AG-881 in advanced IDHm positive solid tumors, including glioma
Completed commercial infrastructure build, including the deployment of an expanded sales force, to successfully launch TIBSOVO (ivosidenib) within 48 hours of potential FDA approval.
FIRST QUARTER 2018 HIGHLIGHTS & RECENT PROGRESS

Initiated ACTIVATE-T, a single-arm pivotal trial for AG-348, in adult pyruvate kinase (PK) deficiency patients who receive regular blood transfusions.
Initiated PEAK, a global registry, for adult and pediatric patients with PK deficiency.
Initiated a perioperative ‘window’ trial with ivosidenib and AG-881 in IDHm low-grade glioma to further investigate their effects on brain tumor tissue.
Initiated 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.
Announced FDA acceptance, priority review and a Prescription Drug User Fee Act (PDUFA) action date of August 21, 2018 for the new drug application (NDA) for TIBSOVO (ivosidenib) for the treatment of patients with R/R AML with an IDH1 mutation.
Completed an underwritten public offering of 8,152,986 shares of common stock at the offering price of $67.00 per share, resulting in proceeds to the company, net of underwriting discounts and commissions, of approximately $516.2 million.
UPCOMING 2018 MILESTONES & EXPECTED DATA PRESENTATIONS

The company expects to achieve the following additional milestones in 2018:

Cancer:

Potential approval and commercialization of TIBSOVO (ivosidenib) in the United States for R/R AML with an IDH1 mutation in the third quarter of 2018.
Submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TIBSOVO (ivosidenib) for the treatment of patients with R/R AML and an IDH1 mutation in the fourth quarter of 2018.
Support, in collaboration with Celgene, the initiation of HO150, 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.
Present 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 the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
Rare Genetic Diseases:

Initiate ACTIVATE, a global, placebo-controlled, pivotal trial for AG-348 in approximately 80 adults with PK deficiency who do not receive regular blood transfusions in the second 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, AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
FIRST QUARTER 2018 FINANCIAL RESULTS & CASH GUIDANCE

Revenue for the quarter ended March 31, 2018 was $8.8 million, which includes $7.4 million of collaboration revenue and $1.4 million of royalty revenue from net sales of IDHIFA. Revenue for the quarter ended March 31, 2017 was $10.5 million and consisted solely of collaboration revenue. The decrease in collaboration revenue recognized for the quarter ended March 31, 2018 compared to the comparable period in 2017 was primarily driven by adoption of the new revenue recognition standard.

Research and development (R&D) expenses were $78.2 million, including $8.6 million of stock-based compensation expense, for the quarter ended March 31, 2018, compared to $62.7 million, including $7.0 million in stock-based compensation expense, for the comparable period in 2017. The increase in R&D expense was primarily attributable to start-up costs for the AG-348 pivotal program in PK deficiency, including the initiation of the ACTIVATE-T trial. R&D expense also increased as a result of the initiation of a Phase 1 dose-escalation study of AG-270, our first-in-class MAT2A inhibitor, and IND enabling activities for AG-636, our DHODH inhibitor.

General and administrative (G&A) expenses were $24.6 million, including $5.9 million of stock-based compensation expense, for the quarter ended March 31, 2018, compared to $14.8 million, including $3.7 million of stock-based compensation expense, for the quarter ended March 31, 2017. The increase in G&A expense was primarily attributable to the growth in our U.S. commercial organization in order to support the expected launch of TIBSOVO (ivosidenib) in the third quarter of 2018.

Net loss for the quarter ended March 31, 2018 was $90.8 million, compared to a net loss of $66.2 million for the quarter ended March 31, 2017.

Cash, cash equivalents and marketable securities as of March 31, 2018 were $994.7 million, compared to $567.8 million as of December 31, 2017. The increase in cash was driven by the net proceeds of $516.2 million from the January follow on offering, $4.4 million of cost reimbursements under our collaboration agreements with Celgene and $12.3 million received from employee stock transactions. This was offset by expenditures to fund operations of $104.8 million during the quarter ended March 31, 2018.

The company expects that its cash, cash equivalents and marketable securities as of March 31, 2018, together with the 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.

WEBCAST INFORMATION

The live webcast from today’s event 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 after the event.

About Agios
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has an approved oncology precision medicine and multiple first-in-class investigational therapies in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company’s website at www.agios.com.

On May 4, 2018 Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that it is scheduled to speak at the Deutsche Bank 43rd Annual Health Care Conference in Boston (Press release, Quest Diagnostics, MAY 4, 2018, View Source [SID1234526138]). Mark Guinan, Executive Vice President and CFO will discuss the company’s vision, goals and two-point strategy to accelerate growth and drive operational excellence. The presentation is scheduled for Wednesday, May 9, 2018 at 12:50 p.m. Eastern Time.

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The presentation and Q&A session will be webcast live during the conference and will be available on the company’s investor relations page which can be accessed at ir.QuestDiagnostics.com. In addition, the archived webcast will be available within 24 hours after the conclusion of the live event and will remain available until June 6, 2018.

On May 3, 2018 Coherus BioSciences, Inc. (NASDAQ:CHRS), reported the re-submission of its biologics license application (BLA) for CHS-1701, a pegfilgrastim (Neulasta) biosimilar candidate, to the U.S. FDA under the 351(k) pathway (Press release, Coherus Biosciences, MAY 3, 2018, View Source [SID1234531702]).

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The BLA is supported by similarity data from analytical, pharmacokinetic, pharmacodynamics, and immunogenicity studies comparing CHS-1701 and Neulasta and integrates new immunogenicity data obtained from using a more revised immunogenicity assay.

"The CHS-1701 BLA re-submission marks a significant milestone in our ongoing transition to a commercial company as we tightly focus on execution of our strategic plan," said Denny Lanfear, President and CEO of Coherus BioSciences. "Pegfilgrastim is the largest selling oncology product in the U.S., and CHS-1701 is the cornerstone of our oncology franchise. We believe we have a strong competitive position with this product, exemplified by our comprehensive clinical immunogenicity data as well as our excellent analytical biosimilarity data."