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.

On January 9, 2020 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) ("Infinity") reported a $20 million non-dilutive asset-backed financing with BVF Partners L.P. ("BVF"), Infinity’s largest shareholder (Press release, Infinity Pharmaceuticals, JAN 9, 2020, View Source [SID1234552924]). This investment by BVF entails no equity to be issued by Infinity and has its sole recourse in potential royalty payments due on future sales of patidegib, a hedgehog pathway inhibitor discovered by Infinity and licensed to PellePharm in 2013. Infinity has the right to repay the $20 million plus interest to repurchase the right to future patidegib royalties during the next three years under certain conditions.

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"This non-dilutive financing is representative of our very collaborative relationship with BVF and their tremendous support as a value-added investor. Leveraging our financial interest in patidegib royalties enables us to preserve significant upside for all of Infinity’s shareholders," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "Importantly, with this financing, we now have over $60 million cash on hand to fund all of our current IPI-549 trials to key data readouts throughout 2020 and into 2H 2021. These data readouts include studies of IPI-549 in a randomized, controlled Phase 2 study in bladder cancer and in front line settings with novel triple therapy combinations, including in indications for which we have seen clinical activity, as part of a thoughtful clinical development strategy designed to reveal the potentially transformative impact of reprogramming macrophages with IPI-549."

Mark Lampert, President of BVF, Inc., commented, "In light of our longstanding association with Infinity, the enormous potential of IPI-549 to help cancer patients, which is not currently reflected in the company’s stock price, and our admiration for management’s dilution sensitivity in advance of data, we wanted to help Infinity raise capital without equity dilution. BVF’s large existing ownership stake in the company was fundamental in aligning our interests with the company to preserve IPI-549 upside for all shareholders, and we believe this innovative financing structure accomplishes the objective in a win-win manner."

Within this extended cash runway into 2H 2021, Infinity expects to generate data on approximately 525 patients from the following trials:

MARIO-275, our global randomized Phase 2 study in collaboration with Bristol-Myers Squibb (BMS), evaluating IPI-549 in combination with Opdivo in patients with advanced urothelial cancer.
MARIO-3, our Phase 2 study in collaboration with Roche/Genentech evaluating IPI-549 in combination with Tecentriq and Abraxane as a front-line treatment in patients with triple negative breast cancer (TNBC) and in combination with Tecentriq and Avastin as a front-line treatment for patients with renal cell cancer (RCC).
MARIO-1, our Phase 1/1b study in collaboration with BMS evaluating IPI-549 in combination with Opdivo in patients with advanced solid tumors.
Arcus Biosciences’ Phase 1 collaboration study evaluating IPI-549 in a novel, checkpoint inhibitor free regimen that includes their dual adenosine receptor inhibitor, AB928, and Doxil in patients with relapsed/refractory TNBC.
In addition to the initial $20 million payment, Infinity is eligible to receive from BVF an additional $5 million payment upon positive data from PellePharm’s Phase 3 trial in patients with Gorlin Syndrome. PellePharm announced the completion of enrollment in a Phase 3 trial of a topical formulation of patidegib in patients with Gorlin Syndrome in December 2019. FDA granted Breakthrough Therapy Designation and Orphan Drug Designation to PellePharm for a topical formulation of patidegib in patients with Gorlin Syndrome in November 2017. Infinity retains rights to all patidegib milestone payments from PellePharm of up to $9 million in regulatory and first commercial sale milestones and $37.5 million in sales threshold milestones.

Infinity has the option to repurchase the rights to future patidegib royalties by paying BVF an amount equal to the principal amount received by Infinity plus interest at any time when the 20-day volume weighted average price per share of Infinity’s common stock exceeds $5.00 during the next three years.

Furthermore, Infinity retains its approximately 1% equity interest in PellePharm. PellePharm has previously announced that LEO Pharmaceuticals has the right to acquire PellePharm following Phase 3 data for total potential consideration of $690 million.

If, during the period ending three years from the date of the agreement or earlier in the event Infinity has exercised its repurchase option for future patidegib royalties from BVF, Infinity completes future equity financings above a specified share quantity threshold and below a specified price threshold, then Infinity has agreed to provide BVF with 50% warrant coverage at a 50% premium to the price at which such shares in excess of the share quantity threshold were sold.

The terms and conditions of the transaction are described in more detail in a Form 8-K filed by Infinity with the Securities and Exchange Commission on January 9, 2020.