On January 9, 2020 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported preliminary unaudited Andexxa global net revenues for the fourth quarter and full year 2019 (Press release, Portola Pharmaceuticals, JAN 9, 2020, View Source [SID1234552930]). For the fourth quarter, the Company expects Andexxa global net revenues to be approximately $28 million. For the full year 2019, the Company expects Andexxa global net revenues to be approximately $111 million.

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In the U.S., net revenues of Andexxa in the fourth quarter 2019 were approximately $24 million, which compares to net revenues of $14.0 million in the same period a year ago and $33.0 million in third quarter 2019.

In Europe, net revenues of Ondexxya were approximately $4 million, which compares to net revenues of $2.7 million in the third quarter of 2019. Fourth quarter 2019 was Ondexxya’s first full quarter of sales following its launch in July 2019 in Europe, where it is now marketed in Germany, Austria, the U.K., the Netherlands, Sweden, Denmark and Finland.

During the fourth quarter, approximately 90 new accounts, and over 425 new accounts in 2019, ordered Andexxa in the U.S. This brings the total number of accounts now ordering Andexxa to approximately 640 at the end of 2019. There continues to be a significant hospital penetration opportunity within the Company’s 2,100 target accounts in 2020 and beyond. Also in the fourth quarter, re-ordering accounts contributed 80% of U.S. revenues, compared to 76% of revenues in the third quarter.

Fourth quarter Andexxa net sales in the U.S. were impacted primarily by two factors:

A $5 million gross to net adjustment due to a return reserve for short-dated product. The Company expects this to be mitigated going forward by its current longer-dated, 36-month product, which began shipping in November 2019.

Flat quarter over quarter demand due to a decrease in utilization, primarily in tier 1 accounts. While physician demand remains strong, the Company believes that in certain of these accounts, hospital pharmacies curtailed use of Andexxa following drug utilization reviews in an effort to manage pharmacy budgets. Following this reduction, re-ordering patterns are stabilizing in many of these accounts.

"While we are disappointed in the fourth quarter net revenues for Andexxa in the U.S., 2019 was a year of significant growth. We exceeded $100 million in revenues, including encouraging contributions from our global launch of Ondexxya, and Andexxa remains on track as one of the top five hospital drug launches in the U.S. in the last 30 years," said Scott Garland, Portola’s president and chief executive officer. "Our belief in the long-term potential of Andexxa remains

strong, and we maintain our projection that this highly innovative, orphan drug has an over $2 billion opportunity in the rapidly growing FXa-inhibitor market in the U.S. and Europe. We look forward to continuing to build long-term revenue growth and value for our shareholders as we establish Andexxa as the standard for care for doctors addressing the urgent needs of patients facing serious, life-threatening bleeds."

"Physician support for Andexxa and Ondexxya in the U.S. and Europe has been tremendous, and we continue to receive outstanding feedback from members of the medical community who have seen first-hand the benefits of Andexxa in treating DOAC-related bleeds," said Sheldon Koenig, Portola’s chief commercial officer. "The drug has excellent efficacy, works fast and is the only FDA-approved agent available. In 2020, we are moving aggressively to execute on our launch strategy and build greater awareness of the life-saving benefits of Andexxa by further educating hospitals on its value."

2020 Growth Drivers

The Company expects continued strong demand of Andexxa, driven by the growth of the Factor Xa inhibitor market in the U.S. and Europe. In 2019, the Factor Xa inhibitor market volume grew by 27% globally to reach $24 billion. In addition, growth in new hospital customers and deeper hospital utilization of Andexxa is expected in 2020 driven by:

Presentation and publication of newly generated health economics and outcomes research data at multiple medical meetings starting with three abstracts at the American College of Cardiology’s Annual Scientific Session together with the World Congress of Cardiology, showing the impact of Andexxa on inpatient mortality, burden of illness, budget impact model and cost effective analysis

Presentation and publication of data demonstrating 4-factor PCC’s, which are only approved to reverse warfarin, are not effective for reversing Factor Xa related bleeds

The launch of key educational initiatives to raise awareness of best practices for the various access and reimbursement pathways available, including: The New Technology Add-on Payment (NTAP) for Medicare beneficiaries in the inpatient setting; a pass through C-code for Medicare and commercial patients in the outpatient setting; participation in both Medicaid and the 340B drug pricing program; and consignment opportunities

Initiation of a single arm study in patients taking Factor Xa inhibitors that require urgent surgery, providing experience to inform the design of a randomized clinical trial

Conclusion of reimbursement discussions in European Wave 1 countries

Expansion of European launch into Wave 2 countries

The Company has a strong cash position to continue to invest in the launch of Andexxa and Ondexxya. As of December 31, 2019, the Company’s cash position totaled $464 million, compared with $317 million as of December 31, 2018.

These preliminary results are based on management’s initial analysis of operations for the quarter ended December 31, 2019. The Company expects to issue full financial results for the fourth quarter and fiscal year 2019 in late February.

Conference Call Details

The Company will host a live conference call on Thursday, January 9, 2020, at 5:00 p.m. ET, which can be accessed by phone by calling (844) 452-6828 from the United States and Canada or 1 (765) 507-2588 internationally and using the passcode 3995797. The webcast can also be accessed live on the Investor Relations section of the Company’s website at View Source

Corporate Update on January 14, 2020

As previously announced, Portola will provide a corporate update on Tuesday, January 14, at 7:00 a.m. PT (10:00 a.m. ET). The live webcast will be available on the Company’s website at View Source A replay of that presentation will remain available on the website after the live webcast.

On January 9, 2019 Pacira BioSciences, Inc. (NASDAQ: PCRX), a leading provider of innovative non-opioid pain management options, reported preliminary unaudited net revenue of $421.0 million for 2019, a 25% increase compared with net revenue of $337.3 million reported for 2018 (Press release, Pacira Pharmaceuticals, JAN 9, 2020, View Source [SID1234552929]). Preliminary fourth quarter net revenues were $122.4 million in 2019, as compared with $95.1 million in 2018.

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"2019 was a year of outstanding results led by strong EXPAREL (bupivacaine liposome injectable suspension) revenue growth as it becomes a critical mainstay of anesthesiologists’ opioid-sparing strategies for a wide range of soft tissue and orthopedic procedures. In addition, we successfully advanced the initial commercial rollout of iovera°, which we believe positions us to become the preferred provider of opioid-sparing pain management for both inpatient and ambulatory surgeries," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "As we look ahead to 2020 and beyond, we expect continued topline growth to drive substantial operating leverage and cash flow, providing us with significant financial flexibility to capitalize on internal and external growth opportunities that support our global leadership position in innovative, non-opioid pain management."

2019 Full Year & Fourth Quarter Revenue Highlights

• Fourth quarter net product sales of EXPAREL/bupivacaine liposome injectable suspension were $118.6 million in 2019, compared to $94.7 million in 2018.
• Fourth quarter EXPAREL net product sales were $116.9 million in 2019, compared to $94.4 million in 2018. Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $1.7 million in 2019, compared to $0.3 million in 2018.
• Full-year EXPAREL net product sales were $407.9 million in 2019, compared to $331.1 million in 2018. Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $3.2 million in 2019, compared to $1.3 million in 2018.
• Fourth quarter iovera° net product sales were $3.2 million in 2019. Full-year iovera° net product sales were $7.9 million in 2019. Pacira began recognizing sales of iovera° in April 2019 after completing its acquisition of MyoScience, Inc., a privately held medical technology company.
• Fourth quarter royalty revenue was $0.6 million and full-year was $2.1 million; compared to $0.4 million and $1.9 million in 2018, respectively.
• The company’s 2019 financial guidance was as follows: EXPAREL net product sales in the range of $400 million to $410 million and iovera° net product sales in the range of $8 million to $10 million.

The financial information included in this press release is preliminary, unaudited and subject to adjustment. It does not present all information necessary for an understanding of the company’s fourth

quarter and full-year financial results for 2019. Pacira expects to report its complete financial results for the fourth quarter and full-year 2019, along with financial guidance for 2020, in the first quarter of 2020.

On January 9, 2020 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company developing next-generation antisense therapies using its scalable PATrOL platform to address genetic diseases, reported its financial results for the fiscal year ended September 30, 2019 (Press release, Ohr Pharmaceutical, JAN 9, 2020, View Source [SID1234552928]).

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"The successful execution of multiple platform milestones in 2019 has built a solid foundation to accelerate the advancement of our pipeline of PATrOL-enabled therapies in 2020 and beyond. As a result, we expect to complete and report data from preclinical studies evaluating the pharmacokinetic ("PK") and pharmacodynamic ("PD") properties of our PATrOL platform and PATrOL-enabled candidates beginning in the first quarter and in the second quarter of calendar year 2020. We believe the results from these studies will inform the development of our first indications, which include Huntington’s disease and Myotonic Dystrophy type 1, as well as allow us to expand our pipeline across a range of targets and tissues," said Dietrich Stephan, Ph.D., chief executive officer of NeuBase Therapeutics.

"As we enter this exciting period in NeuBase’s history, we’ve garnered increased industry recognition for our platform’s unique capabilities to address genetic diseases. Over the past several months, we’ve recruited renowned scientists and leading industry experts in their respective fields to several executive and research positions at NeuBase, as well as appointments to our board of directors and scientific advisory committee. The team we now have in place is positioned to quickly advance the development of our pipeline of PATrOL-enabled therapies," concluded Dr. Stephan.

Fiscal Fourth Quarter of 2019 and Recent Operating Highlights

·U.S. Patent and Trademark Office (USPTO) issued NeuBase U.S. Patent No. 10,370,415, a foundational patent that covers its proprietary DNA and RNA binding technology, which enables PATrOL-based therapies to target the secondary structures of DNA and RNA
·The Scientist magazine recognized NeuBase’s Janus Base technology as a top 10 innovation of 2019
·Management held a KOL meeting for the investment community on Huntington’s disease and NeuBase’s peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform
·Announced the appointment of several preeminent scientists to NeuBase’s Scientific Advisory Board in the fourth quarter of FY2019 and in the months following, including George Church, Ph.D., Samuel Broder, M.D., and Steven Dowdy, Ph.D.
·Management rang the closing bell at the NASDAQ on August 7, 2019 to celebrate the listing of the Company’s stock on NASDAQ

Financial Results for the Fiscal Year Ended September 30, 2019:

Note for Fiscal Year 2019: The period of July 12 to September 30, 2019 includes the combined financial results for Ohr Pharmaceutical and NeuBase post-merger, which closed on July 12, 2019. The period prior to July 12, 2019 includes only the financials of NeuBase prior to the merger. Therefore, Fiscal Year 2019 is not directly comparable to prior periods.

·At September 30, 2019, the Company had cash and cash equivalents of approximately $10.3 million, compared to cash and equivalents of approximately $0.2 million at September 30, 2018. The Company believes that its current cash balance will provide sufficient capital to fund operations through the end of fiscal year 2020.
·For the fiscal year ended September 30, 2019, the Company reported a net loss of approximately $27.0 million, or ($3.26) per share, compared to a net loss of approximately $0.04 million, or ($0.01) per share, for the period from inception (August 28, 2018) to September 30, 2018. Non-GAAP adjusted net loss was $3.7 million, or ($0.45) per share, for fiscal year 2019, and $0.04 million, or ($0.01) per share, for the period from inception (August 28, 2018) to September 30, 2018. The non-GAAP adjusted net loss for fiscal year 2019 excludes certain non-cash items. Loss per share amounts have been retroactively adjusted for the reverse stock split effected on February 4, 2019.
·For the fiscal year ended September 30, 2019, total operating expenses were approximately $26.3 million, consisting of approximately $9.1 million in general and administrative expenses, $4.3 million of research and development expenses, and $13.0 million in research and development – licenses acquired – expense. This compares to total operating expenses of $0.04 million for the period from inception (August 28, 2018) to September 30, 2018, comprised of approximately $0.03 million in general and administrative expenses, and $0.01 million in research and development expenses.

Non-GAAP Financial Measures

The non-GAAP financial measures contained herein are a supplement to the corresponding financial measures prepared in accordance with U.S. GAAP. The non-GAAP financial measures presented exclude the following non-cash items: share-based compensation expense, non-cash research and development expense-licenses acquired, the change in fair value of warrant liabilities, depreciation and amortization expense and non-cash interest and amortization expense on convertible notes. Management believes that because these non-cash items have no impact on the cash position of the Company and most of these expenses and liabilities occurred as a result of the merger with Ohr Pharmaceutical, adjustments for these items will assist investors in making comparisons of period-to-period operating results. Furthermore, management believes that the excluded items are not indicative of the Company’s on-going core operating performance.

These non-GAAP financial measures have certain limitations in that they do not reflect all of the costs associated with the operations of the Company’s business as determined in accordance with GAAP. Therefore, investors should consider non-GAAP financial measures in addition to, and not as a substitute for, or as superior to, measures of financial performance prepared in accordance with GAAP. The non-GAAP financial measures presented by the Company may be different from the non-GAAP financial measures used by other companies.

For more information on our non-GAAP financial measures and a reconciliation of GAAP to non-GAAP measures, please see the "Reconciliation of GAAP to Non-GAAP Results" table in this press release.

On January 9, 2020 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported plans to report 2019 fourth quarter and full year financial results on February 6, 2020 (Press release, Ligand, JAN 9, 2020, View Source [SID1234552927]). Ligand’s CEO John Higgins, President and COO Matt Foehr and Executive Vice President and CFO Matt Korenberg will host the conference call.

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2019 Fourth Quarter and Full Year Earnings Conference Call

What:

Ligand conference call to discuss financial results and provide general business updates

Date:

Thursday, February 6, 2020

Time:

4:30 p.m. Eastern time (1:30 p.m. Pacific time)

Conference Call:

Dial (833) 591-4752 within the U.S.

Dial (720) 405-1612 outside the U.S.

Conference ID is 1150048

Webcast:

Live conference call webcast and replay accessible at www.ligand.com

On January 9, 2020 Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported an update on recent progress and the Company’s 2020 priorities and expected milestones (Press release, Intellia Therapeutics, JAN 9, 2020, View Source [SID1234552925]).

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"2020 will be a significant year for Intellia, as we execute on our full-spectrum strategy. With milestones anticipated across our pipeline, we are making important progress towards the development of curative treatments for severe diseases. In particular, we expect to dose ATTR patients with the first-ever systemically delivered CRISPR/Cas9-based therapy this year, and we are beginning IND-enabling activities for our newly announced development candidate, NTLA-5001, a WT1-TCR-directed engineered cell therapy, for treatment of AML," said Intellia President and Chief Executive Officer, John Leonard, M.D. "We are focused on developing a robust platform with modular genome editing capabilities that enable a fast and reproducible path to development. Today’s update reflects this strategy, and it also features the announcement of our third development program, an in vivo knockout approach for HAE. Importantly, this program leverages the infrastructure and insights from NTLA-2001 and underscores our ability to produce a rapid succession of new clinical candidates. We are excited by the strong momentum across our diverse pipeline and look forward to providing updates on our development programs in the upcoming year."

Program Updates and Anticipated 2020 Milestones:

ATTR Program: Intellia remains on track to submit an investigational new drug (IND) application in mid-2020 for its lead in vivo candidate, NTLA-2001, for treatment of

transthyretin amyloidosis (ATTR). NTLA-2001 is anticipated to be the first systemically delivered CRISPR/Cas9 therapy to enter the clinic, and Intellia anticipates dosing the first patients in the second half of 2020. In addition, Intellia completed a 12-month durability study of its lead lipid nanoparticle (LNP) formulation in support of NTLA-2001, maintaining an average reduction of >95% of serum transthyretin (TTR) protein and sustained liver genome editing after a single dose in non-human primates (NHPs). NTLA-2001 is part of a co-development/co-promotion (Co/Co) agreement between Intellia, which is the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc. (Regeneron). Intellia and Regeneron have a 75% and 25% share of worldwide development costs and profits, respectively.

AML Program: Intellia reported NTLA-5001 as its first engineered T cell therapy development candidate, utilizing its T cell receptor (TCR)-directed approach to target the Wilms’ Tumor 1 (WT1) intracellular antigen for the treatment of acute myeloid leukemia (AML). Intellia’s WT1-TCR-directed approach aims to develop a broadly applicable treatment for AML patients, regardless of mutational background of a patient’s leukemia. This approach employs CRISPR/Cas9 to efficiently knock out and replace the endogenous TCR with a natural, high affinity therapeutic TCR. The resulting cells are capable of specific and potent killing of AML blasts, and have no detectable bone marrow cell toxicity. The Company expects to present preclinical data in support of NTLA-5001 at an upcoming scientific meeting in the first quarter of 2020 and plans to submit an IND application in the first half of 2021. Additional efforts are underway to evaluate the potential use of the WT1-TCR construct to treat other tumor types, including solid tumors.

HAE Program: Today, Intellia announced that the Company is committed to developing a CRISPR/Cas9-based therapy for hereditary angioedema (HAE) as its third development program. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. The disease is caused by increased levels of bradykinin, a protein which leads to swelling. Most patients with HAE have a C1 esterase inhibitor (C1-INH) protein deficiency, which normally prevents the unregulated release and buildup of bradykinin. Using its modular LNP-based CRISPR/Cas9 delivery system, Intellia aims to knock out the kallikrein B1 (KLKB1) gene, which is part of a biological pathway that results in release of bradykinin. Knocking out this gene should reduce the undesired bradykinin activity in HAE patients. The Company plans to present preclinical data at an upcoming scientific meeting in the first quarter of 2020. In addition, Intellia is evaluating several potential guide RNAs and expects to nominate a development candidate in the first half of 2020. Intellia’s KLKB1 HAE program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party.

Cash Position and Financial Guidance:

Intellia ended the fourth quarter of 2019 with approximately $284.5 million in cash, cash equivalents and marketable securities. Intellia expects that its cash, cash equivalents and marketable securities as of December 31, 2019 will enable the Company to fund its anticipated operating expenses and capital expenditure requirements at least through the end of 2021. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.