On January 12, 2020 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address significant unmet medical needs, reported that it will provide an outlook for 2020 and review progress towards Vision 3×3, the company’s strategic roadmap through 2025 to achieve sustainable growth at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Ascendis Pharma, JAN 12, 2020, View Source [SID1234553048]).

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"2019 was a transformative year for Ascendis Pharma. For our endocrinology rare disease portfolio, we were successful in our first phase 3 trial for a TransCon product candidate, TransCon hGH, and we advanced two other programs globally into phase 2. In addition, we established oncology as our second therapeutic area with a highly-differentiated pipeline leveraging the TransCon technology," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "As groundbreaking as 2019 was for Ascendis, it was only the beginning of our path to build a leading fully-integrated global biopharma company. We are on track with our Vision 3×3 goals to deliver multiple sources of sustainable growth as we seek to make a meaningful difference in patients’ lives."

Pipeline Updates and 2020 Outlook

TransCon hGH: TransCon hGH is an investigational long-acting prodrug of human growth hormone (hGH) in phase 3 development as a once-weekly treatment for growth hormone deficiency (GHD). TransCon hGH releases unmodified somatropin and has demonstrated a statistically significant increase in height velocity compared to a daily hGH in the phase 3 heiGHt Trial:

Ascendis Pharma recently held two pre-BLA meetings with the U.S. Food and Drug Adminstration (FDA) to review its Chemistry, Manufacturing and Controls (CMC), and clinical/non-clinical packages for TransCon hGH as a potential treatment for pediatric GHD. The company is on track to file a Biologics License Application with the FDA in the second quarter. A Marketing Authorisation Application to the European Medicines Agency is planned to follow in the fourth quarter.
Long-term data presented from the ongoing enliGHten Trial (long-term extension) continued to demonstrate statistically superior growth of subjects treated with once-weekly TransCon hGH in the heiGHt Trial who continued into enliGHten, compared to those who started treatment with daily Genotropin and switched to TransCon hGH after one year. The adverse event profile of TransCon hGH, which was comparable to Genotropin in the phase 3 heiGHt Trial, was consistent across the phase 3 clinical trials.
The company plans to submit regulatory filings to initiate a global, phase 3 clinical trial in adult GHD during the first quarter, and to initiate a trial in pediatric GHD in Japan during the fourth quarter.

TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a once-daily replacement therapy for hypoparathyroidism (HP) designed to replace PTH at physiologic levels for 24 hours each day and fully address all aspects of the disease:

Following completion of screening of subjects in the recently expanded phase 2 PaTH Forward Trial, Ascendis intends to enroll approximately 55 subjects in the trial. The company expects to report top-line results from the trial around the end of March 2020, with six-month data from the open-label extension phase expected in the third quarter.
Preliminary data presented from the first eight subjects who completed four weeks of follow-up in the open-label extension portion of the phase 2 PaTH Forward Trial reinforce the company’s target product profile for TransCon PTH as a promising new potential therapy for HP in the absence of standard of care.
The company plans to submit regulatory filings to initiate a global, phase 3 clinical trial in adults with HP during the fourth quarter.

TransCon CNP: TransCon CNP is an investigational long-acting prodrug of CNP in development as a therapy for children with achondroplasia, the most common form of dwarfism, for which there is no FDA-approved treatment. TransCon CNP is designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose:

Ascendis is conducting the phase 2 ACcomplisH Trial of TransCon CNP in children (ages 2-10 years) with achondroplasia and plans to escalate sequential dose cohorts throughout 2020.
The company is expanding the TransCon CNP program in China through its strategic investment in VISEN Pharmaceuticals, with initiation of a second phase 2 trial in children with achondroplasia during the fourth quarter.

Oncology: Ascendis continues to advance a pipeline of multiple pre-clinical programs in oncology by applying both systemic and sustained localized TransCon technologies for clinically validated pathways:

Additional data from non-human primate studies demonstrated that a single dose of TransCon IL-2 b/g provided biased receptor binding and prolonged enhancement of lymphocyte counts, suggesting feasibility of every three week dosing and reduced risk of toxicity.
Ascendis Pharma’s innovative TransCon technology for sustained localized release intratumorally (IT) was accepted to participate in the FDA’s Emerging Technology Program. The program provides for enhanced interactions and dialogue with the FDA to discuss, identify and resolve potential technical and CMC regulatory questions related to the TransCon sustained IT programs prior to filing regulatory submissions.
The company plans to file an IND or equivalent for its first oncology program in 2020, furthering the goal to create best-in-class oncology therapeutics.
Presentation at J.P. Morgan Healthcare Conference on Monday, January 13

Live webcasts of the J.P. Morgan presentation and associated Question & Answer session will be available in the Investors and News section of the Ascendis Pharma website at:

View Source

The presentation will begin at 8:00 a.m. Pacific Time, followed by the Question & Answer session at 8:30 a.m. A webcast replay will also be available for 30 days.

The company’s corporate investor presentation and slides from the J.P. Morgan presentation are also available in the Investors and News section.

On January 12, 2020 Luminex Corporation (Nasdaq: LMNX) reported 2019 highlights and 2020 guidance (Press release, Luminex, JAN 12, 2020, View Source [SID1234553041]).

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All amounts in this release are in conformity with U.S. generally accepted accounting principles ("GAAP").

Preliminary results, contained herein, are based on management’s initial analysis of operations for the quarter and year ended December 31, 2019 and are subject to further internal review and audit by the company’s external auditors.

Current Highlights

Total fourth quarter revenue of $90.4 million, an 11% increase over Q4 2018.
Total full year revenue of $334.6 million, up 6%; and up 19% without consideration of LabCorp revenue and including the effect of the Flow Cytometry acquisition.
Sample-to-answer full year revenue of $75.7 million, up more than 20% vs. 2018, including contracting 200 additional systems.
Licensed Technologies Group full year revenue was relatively unchanged from 2018, as expected; however, end user sales of our technology increased by 12% year over year.
Flow Cytometry full year revenue of $45 million, up 11% vs. 2018.
Anticipate being profitable in the fourth quarter of 2019.
Continued $0.09 per share dividend in the fourth quarter of 2019.
2020 Guidance

The company expects 2020 consolidated revenue to be between $352 and $362 million, up approximately 7% to the mid-point relative to 2019, which includes approximately 2 to 3 percent headwind attributable to the departure of certain remaining sales to LabCorp.

CEO Commentary

"2019 was a transition year for Luminex as we adjusted to the departure of certain sales to LabCorp and the integration of the Flow Cytometry acquisition," said Nachum "Homi" Shamir, President & CEO. "I am very pleased with the significant progress we are making. Luminex is now a more diversified company, with opportunities for strong organic growth, profitability, and cash flow, and exciting new platforms being launched across each of our major product lines. We are well positioned to achieve our mid-term target of $500 million of annual revenue in the next 4 to 5 years."

Luminex Presentation / Webcast at 38th Annual J.P. Morgan Healthcare Conference on January 14, 2020

Luminex President and CEO, Homi Shamir, will present at the 38th Annual J.P. Morgan Healthcare Conference to be held at The Westin St. Francis hotel in San Francisco, CA on Tuesday, January 14 at 12:00 p.m. Pacific Time. A live webcast of the Luminex presentation may be accessed at the company’s website at investor.luminexcorp.com. The presentation will be archived for six months on the website using the ‘replay’ link.

Fourth Quarter and Full Year 2019 Earnings Release on February 10, 2020

The company plans to report results for the fourth quarter and full year ended December 31, 2019 and provide details around the 2020 revenue guidance after the market close on Monday, February 10, 2020. Management will hold a conference call to discuss the operating highlights and financial results on that date, at 5:00 p.m. Eastern Time.

On January 12, 2020 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported an update on its business performance, including preliminary net product sales results for 2019, and key commercial and clinical development milestones for 2020 (Press release, Neurocrine Biosciences, JAN 12, 2020, View Source [SID1234553040]). Kevin Gorman, Chief Executive Officer of Neurocrine Biosciences, will discuss these updates as part of a webcast presentation at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, Jan. 13 at 11:30 a.m. Pacific Time (2:30 p.m. Eastern Time).

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Preliminary Fourth Quarter and Full-Year 2019 INGREZZA (valbenazine) Net Product Sales and Financial Results (Unaudited)

Based on preliminary unaudited financial information, the Company expects INGREZZA net product sales for the three months and full-year ended December 31, 2019 to be approximately $238 million and $753 million, respectively, compared to $130 million and $410 million for the same periods in 2018. Preliminary analysis of INGREZZA net product sales and TRx for the fourth quarter 2019 compared to third quarter 2019 suggests:

– Continued strength in new patient additions
– End of fourth quarter days-on-hand channel inventory increased relative to the third quarter due to timing of quarter-end purchases resulting in an approximate $11 million benefit to net product sales. Given the increase to year-end channel inventory, the Company anticipates this may lead to a reduction to channel inventory in the first quarter of 2020.
– INGREZZA net product sales per prescription of $5,650 for the fourth quarter of 2019

Preliminary unaudited cash and marketable securities balance as of December 31, 2019 is $970 million.

"Our results reflect our team’s dedication to educating healthcare providers and patients about tardive dyskinesia as seen in the growing number of patients receiving treatment with INGREZZA. We still have a lot of work to do as a majority of people suffering from tardive dyskinesia remain undiagnosed and untreated," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "In 2020, we aim to have three FDA-approved medicines in four indications and will remain focused on helping more tardive dyskinesia patients with INGREZZA, preparing for the anticipated approval of opicapone in the U.S., and advancing our growing pipeline, including three pivotal clinical trial programs. We are well positioned as we evolve towards becoming a leading global neuroscience-focused biopharmaceutical company."

2020 INGREZZA Net Product Sales Outlook

During the first quarter of 2020, the Company expects INGREZZA net product sales to be impacted as a result of payer-related seasonal dynamics, gross-to-net discount increases from payer and Medicare Part D rebates, and potential reduction in channel inventory. The Company does not intend to provide formal INGREZZA net product sales guidance for 2020 as the tardive dyskinesia market continues to evolve.

2020 Expected Milestones and Key Activities

INGREZZA (valbenazine) for Tardive Dyskinesia

– "Talk About TD" disease state awareness campaign
– Continued execution of post-marketing clinical studies, including RE-KINECT, the largest real-world study in patients with possible tardive dyskinesia
– Presentations at key scientific annual meetings, including American Academy of Neurology (AAN), American Psychiatric Association (APA), International Parkinson and Movement Disorder Society (MDS)
– Regulatory submissions for approval by Mitsubishi Tanabe in ASEAN territory

Valbenazine for Chorea in Huntington Disease

– Advance the current Phase III study recruitment and initiate open label portion of study during 1H 2020

Elagolix in Collaboration with AbbVie

– Prescription Drug User Fee Act (PDUFA) date in Q2 2020 of elagolix for uterine fibroids
– Continued launch of ORILISSA (elagolix) to treat moderate to severe pain associated with endometriosis by AbbVie

Opicapone for Parkinson’s Disease

– PDUFA date of April 26, 2020
– Preparation for commercial launch
– Presentations at key scientific annual meetings, including AAN, MDS

Crinecerfont (NBI-74788) for Congenital Adrenal Hyperplasia (CAH)

– Phase IIa data (adults) oral presentation in March at ENDO 2020
– Global registrational study initiation (adults) in mid-2020
– Advancement of pediatric plan including Phase IIa study

NBIb-1817 (VY-AADC) for Parkinson’s Disease

– Present 3yr follow up data from study PD-1101 at appropriate medical conference
– Implement amended protocol for RESTORE-1 registration trial by mid-year based upon feedback from the U.S. Food and Drug Administration (FDA)
– Initiate RESTORE-II registration trial in 2H 2020

NBI-921352 (XEN901) for Epilepsy

– File Investigational New Drug (IND) application with the FDA in mid-2020 in order to start a Phase II trial in SCN8A developmental and epileptic encephalopathy patients in 2H 2020

ACT-709478 for Epilepsy

– Upon IND application acceptance by the FDA, expected in mid-2020, the Company will have 30 days to exercise the option to license ACT-709478
– A Phase II study in a rare pediatric epilepsy is planned in 2H 2020

About INGREZZA (valbenazine) Capsules
INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso, and/or other body parts.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release in presynaptic neurons. INGREZZA, developed in Neurocrine’s laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

Important Safety Information

Contraindications
INGREZZA is contraindicated in patients with a history of hypersensitivity to valbenazine or any components of INGREZZA. Rash, urticaria, and reactions consistent with angioedema (e.g., swelling of the face, lips, and mouth) have been reported.

Warnings & Precautions
Somnolence
INGREZZA can cause somnolence. Patients should not perform activities requiring mental alertness such as operating a motor vehicle or operating hazardous machinery until they know how they will be affected by INGREZZA.

QT Prolongation
INGREZZA may prolong the QT interval, although the degree of QT prolongation is not clinically significant at concentrations expected with recommended dosing. INGREZZA should be avoided in patients with congenital long QT syndrome or with arrhythmias associated with a prolonged QT interval. For patients at increased risk of a prolonged QT interval, assess the QT interval before increasing the dosage.

Parkinsonism
INGREZZA may cause Parkinsonism in patients with tardive dyskinesia. Parkinsonism has also been observed with other VMAT2 inhibitors. Reduce the dose or discontinue INGREZZA treatment in patients who develop clinically significant parkinson-like signs or symptoms.

Adverse Reactions
The most common adverse reaction (≥5% and twice the rate of placebo) is somnolence. Other adverse reactions (≥2% and >placebo) include: anticholinergic effects, balance disorders/falls, headache, akathisia, vomiting, nausea, and arthralgia.

On January 12, 2020 Exact Sciences Corp. (Nasdaq: EXAS) reported that the company expects to report revenue between $294 million and $296 million for the fourth quarter ended Dec. 31, 2019 (Press release, Exact Sciences, JAN 12, 2020, View Source [SID1234553038]).

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"Building off a transformative 2019, Exact Sciences is uniquely positioned to change the way cancer is diagnosed and treated," said Kevin Conroy, chairman and CEO of Exact Sciences. "Following our combination with Genomic Health, we have the leading scientific minds, an experienced commercial team with deep relationships, and the global footprint necessary to support the growth of current and future cancer diagnostics. We’ve never been more excited about the future of Exact Sciences."

Preliminary, Unaudited Fourth-Quarter 2019 Financial Results

For the three-month period ended December 31, 2019, as compared to the same period of 2018 (where applicable):

Expected total revenue between $294 million and $296 million
Expected Screening revenue between $229 million and $230 million, an increase of 61 percent
Expected Precision Oncology revenue between $65 million and $66 million for the period Nov. 8, 2019 through Dec. 31, 2019, following the close of the Genomic Health acquisition
Expected Precision Oncology proforma revenue for the full fourth quarter between $118 million and $119 million, an increase of 13 percent, assuming Genomic Health were a standalone entity
Cologuard test volume was 477,000, an increase of 63 percent
Oncotype DX test volume was 41,000, an increase of 14 percent
Preliminary, Unaudited 2019 Financial Results

For the twelve-month period ended December 31, 2019, as compared to the same period of 2018 (where applicable):

Expected total revenue between $874.5 million and $876.5 million, including Precision Oncology revenue between $65 million and $66 million for the period Nov. 8, 2019 through Dec. 31, 2019, following the close of the Genomic Health acquisition
Expected Screening revenue between $809.5 million and $810.5 million, an increase of 78 percent
Expected Precision Oncology proforma revenue between $455 million and $456 million, an increase of 16 percent, assuming Genomic Health were a standalone entity
Cologuard test volume was 1.68 million, an increase of 80 percent
Oncotype DX test volume was 156,000, an increase of 14 percent
For the fourth quarter and 2019, Screening includes revenue from Cologuard and Biomatrica products. Precision Oncology includes global Oncotype DX product revenue. Proforma revenue figures for Precision Oncology for the three and twelve-month periods ended Dec. 31, 2019 include results prior to the acquisition of Genomic Health by Exact Sciences which closed on Nov. 8, 2019.

Exact Sciences has not completed preparation of its financial statements for the fourth quarter or full year of 2019. The revenue ranges presented in this news release for the fourth quarter of 2019 and for the year ended Dec. 31, 2019 are preliminary and unaudited and are thus inherently uncertain and subject to change as we complete our financial results for the fourth quarter of 2019. We are in the process of completing our customary year-end close and review procedures as of and for the year ended Dec. 31, 2019, and there can be no assurance that our final results for this period will not differ from these estimates. During the course of the preparation of our consolidated financial statements and related notes as of and for the year ended Dec. 31, 2019, we or our independent registered public accountants may identify items that could cause our final reported results to be materially different from the preliminary financial estimates presented herein.

Exact Sciences plans to report 2019 financial results and provide guidance during its February 2020 earnings call.

About Cologuard
Cologuard was approved by the FDA in August 2014, and results from Exact Sciences’ prospective 90-site, point-in-time, 10,000-patient pivotal trial were published in the New England Journal of Medicine in March 2014. Cologuard is included in the American Cancer Society’s (2018) colorectal cancer screening guidelines and the recommendations of the U.S. Preventive Services Task Force (2016) and National Comprehensive Cancer Network (2016). Cologuard is indicated to screen adults 45 years of age and older who are at average risk for colorectal cancer by detecting certain DNA markers and blood in the stool. Do not use Cologuard if you have had precancer, have inflammatory bowel disease and certain hereditary syndromes, or have a personal or family history of colorectal cancer. Cologuard is not a replacement for colonoscopy in high risk patients. Cologuard performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older. Cologuard performance in repeat testing has not been evaluated.

The Cologuard test result should be interpreted with caution. A positive test result does not confirm the presence of cancer. Patients with a positive test result should be referred for diagnostic colonoscopy. A negative test result does not confirm the absence of cancer. Patients with a negative test result should discuss with their doctor when they need to be tested again.

Medicare and most major insurers cover Cologuard. For more information about Cologuard, visit www.cologuardtest.com. Rx Only.

On January 12, 2020 Mundipharma reported a partnership with Samsung Bioepis to commercialize Samsung Bioepis’ first-wave biosimilar candidates in Taiwan and Hong Kong (Press release, Mundipharma, JAN 12, 2020, View Source [SID1234553037]).

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The partnership covers Samsung Bioepis’ biosimilar candidates in the field of immunology and oncology, including SB5 (adalimumab), SB4 (etanercept), SB3 (trastuzumab), and SB8 (bevacizumab).

Through the partnership, Mundipharma will be the exclusive commercialization partner, while Samsung Bioepis will remain the Marketing Authorisation Holder (MAH), responsible for clinical development, regulatory registration, and manufacture of the biosimilars.

"This partnership brings together Samsung Bioepis’ proven biosimilar development platform with Mundipharma’s commercial acumen and market insight and ability to increase patient access to proven treatments," said Mundipharma CEO, Raman Singh. "This partnership will help to address the patient need in two important territories in Asia for immunology and oncology treatments," he added.