On February 28, 2019 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported its fourth quarter business update and full year results for 2018 (Press release, argenx, FEB 28, 2019, View Source [SID1234533796]).

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The full year results will be discussed during a conference call and webcast presentation today at 3 pm CET/9 am EST. To participate in the conference call, please select your phone number below and use the confirmation code 9795988. The webcast may be accessed on the homepage of the argenx website at www.argenx.com or by clicking here.

"By translating fundamental immunology breakthroughs into novel product candidates, we have built a robust pipeline of differentiated antibodies with the potential to truly impact patients’ lives," commented Tim Van Hauwermeiren, CEO of argenx. "We made great progress in 2018 with our wholly owned lead asset efgartigimod, advancing development across four severe autoimmune indications, in both intravenous and subcutaneous formulations. We signed a transformational global collaboration and license agreement with Janssen to develop cusatuzumab in AML while retaining key commercial rights to the product in the U.S. We also announced that AbbVie exercised its option to in-license ARGX-115 targeting GARP.

"Based on our deep and diversified product candidate portfolio, a solid cash position and an ambitious business plan we are facing an abundance of opportunity ahead in 2019. We will continue to build out our novel pipeline through our Innovative Access Program and our commercial capabilities as we drive enrollment of the Phase 3 ADAPT trial of efgartigimod in gMG and plan for the start of a second Phase 3 trial in ITP."

FOURTH QUARTER 2018 AND RECENT HIGHLIGHTS

argenx continues to build a deep pipeline of differentiated antibody-based product candidates, including wholly-owned efgartigimod for the treatment of severe autoimmune diseases and cusatuzumab for the treatment of acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) in collaboration with Janssen. Through its Innovative Access Program, argenx grows its proprietary and partnered pipeline by leveraging its antibody expertise to match fundamental biology breakthroughs at leading research institutions.

Efgartigimod (ARGX-113) Program

· Full Phase 2 immune thrombocytopenia (ITP) trial data presented during American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting

o Correlation between IgG antibody reduction, platelet count increase and reduction of bleeding events following efgartigimod treatment.

o Increasing separation between treatment arms and placebo at increasing response thresholds observed, with response rates of 46% and 67% in primary trial and first dosing cycle of open-label extension trial, respectively.

o Primary endpoint analysis revealed efgartigimod to be well-tolerated in all patients consistent with tolerability analysis from Phase 1 healthy volunteer study.

· Orphan drug designation granted in February 2019 by U.S. Food and Drug Administration (FDA) for use for primary ITP.

o argenx additionally received orphan drug designation for efgartigimod in generalized myasthenia gravis (gMG) from FDA and the European Medicines Agency, which was announced, respectively, in September 2017 and March 2018.

· By mid-2019, argenx expects to close its ongoing open-label extension study as part of the completed Phase 2 proof-of-concept trial in ITP in anticipation of the start of a more chronic dosing regimen in its Phase 3 trial.

Cusatuzumab (ARGX-110) Program

· Updated Phase 1/2 AML trial data in combination with azacitidine presented during ASH (Free ASH Whitepaper) Annual Meeting.

o Overall response rate (ORR) of 92% in 12 newly diagnosed AML patients unfit for intensive chemotherapy, including 10 patients (91%) with a complete remission with or without hematologic recovery (CR/CRi) and 1 patient (9%) with partial remission (PR).

o Five patients (42%) achieved minimal residual disease (MRD) negativity. Translational data demonstrated that cusatuzumab monotherapy and in combination with azacitidine significantly reduced leukemic stem cells in the bone marrow of AML patients.

o Well-tolerated with no exacerbation of azacitidine toxicity.

· Orphan drug designation granted by FDA in January 2019 for use of cusatuzumab for AML.

Collaborations

· Entered global collaboration and license agreement with Halozyme for ENHANZE technology (February 2019).

· Gained access to ENHANZE subcutaneous delivery technology for up to three targets, including exclusive rights to develop therapeutic products targeting human neonatal Fc receptor FcRn.

· Upfront payment of $30 million paid to Halozyme with potential future payments up to $160 million per selected target subject to achievement of specified development, regulatory and sales-based milestones.

· Entered exclusive global collaboration and license agreement with Janssen for cusatuzumab

· Deal totaling up to $1.6 billion, plus royalties ranging from the low double digits to the high teens, to develop cusatuzumab in AML, MDS and other hematological malignancies.

Received $300 million upfront cash payment, and Johnson & Johnson Innovation made equity investment of €176.7 million ($200.0 million based on exchange rate on date of signing) in argenx, representing 4.68% of then-outstanding share capital.

· argenx retains right to co-promote cusatuzumab in the U.S. and share such royalties with Janssen on a 50-50 basis.

Corporate Updates

· Awarded €2.6 million grant from Flanders Innovation and Entrepreneurship (VLAIO) agency to explore new applications and modes of action of proprietary ABDEG technology, the Fc engineering technology used in the design of efgartigimod to augment clearance of disease-causing autoantibodies.

· Selected for BEL 20 Index on Euronext Brussels and awarded ‘Best BEL 20 Company’ for 2018.

· Intellectual property portfolio (which includes both owned and in-licensed patent rights) now includes 105 granted and 106 pending patents, covering lead assets and core technologies.

· Company expanded to 132 employees and consultants in support of growing business needs.

OUTLOOK 2019

In 2019, argenx expects to execute on its business plan towards the below milestones:

· Continue enrollment of efgartigimod in ongoing Phase 3 ADAPT and ADAPT+ trials in gMG

· Launch Phase 3 clinical trial with efgartigimod in ITP in second half of 2019

· Launch Phase 2 clinical trial with subcutaneous formulation of efgartigimod in ITP in first half of 2019

· Launch cohort 3 of Phase 2 proof-of-concept trial of efgartigimod in pemphigus vulgaris in first half of 2019

· Launch Phase 2 clinical trial with efgartigimod in chronic inflammatory demyelinating polyneuropathy before end of 2019

· Launch Phase 1 clinical trial with subcutaneous ENHANZE formulation of efgartigimod in healthy volunteers

· Host R&D day (May 22, 2019) to present new pipeline programs ARGX-117 and ARGX-118

Details of the Financial Results

Cash, cash equivalents and current financial assets totaled €564.6 million as of December 31, 2018, compared to €359.8 million on December 31, 2017. The increase in the year-end cash balance on December 31, 2018 resulted primarily from €240.9 million of net proceeds received from the follow-on public offering of American Depositary Shares on the Nasdaq Global Select Market in September 2018.

Operating income decreased by €12.1 million for the year ended December 31, 2018 to €29.2 million, compared to €41.3 million for the year ended December 31, 2017. The decrease in 2018 was primarily related to a €11.5 million decrease in the recognition of upfront payments.

Research and development expenses totaled €83.6 million and €51.7 million for the years ended December 31, 2018 and 2017, respectively. The increase in 2018 resulted primarily from higher external research and development expenses and personnel expenses, reflecting higher clinical trials costs and manufacturing expenses related to the development of the argenx product candidate portfolio and the recruitment of additional employees to support its research and development activities. argenx employed 75 employees in its research and development functions on December 31, 2018, compared to 58 employees on December 31, 2017.

Selling, general and administrative expenses totaled €27.5 million and €12.4 million for the years ended December 31, 2018 and 2017, respectively. The increase of €15.1 million in selling, general and administrative expenses for the year ended December 31, 2018 primarily resulted from higher personnel expenses and consulting fees related to the preparation for potential future commercialization of lead product candidate efgartigimod. On December 31, 2018, argenx employed 30 employees in its selling, general and administrative functions, compared to 15 employees on December 31, 2017.

For the year ended December 31, 2018, financial income amounted to €3.7 million compared to €1.3 million for the year ended December 31, 2017. The increase of €2.4 million in 2018 related primarily to an increase in the interest received on cash, cash equivalents and current financial assets.

Exchange gains totaled €12.3 million for the year ended December 31, 2018 compared to the €5.8 million exchange losses incurred for the year ended December 31, 2017. The increase was mainly attributable to unrealized exchange rate gains on the cash and current financial assets position in U.S. dollars due to the favorable fluctuation of the EUR/USD exchange rate in 2018.

The total comprehensive loss for the year ended December 31, 2018 was €66.6 million, compared to €28.1 million for the year ended December 31, 2017.

U.S. SEC and statutory financial reporting

argenx’s primary accounting standard for quarterly earnings releases and annual reports is International Financial Reporting Standards (IFRS) as issued by the International Accounting Standards Board (IASB). Quarterly summarized statements of profit and loss based on IFRS as issued by the IASB are available on www.argenx.com.

In addition to reporting financial figures in accordance with IFRS as issued by the IASB, argenx also reports financial figures in accordance with IFRS as adopted by the European Union (EU) for statutory purposes. The consolidated statement of financial position, the consolidated statements of profit and loss, the consolidated statements of cashflow, and the consolidated statement of changes in equity are not affected by any differences between IFRS as issued by the IASB and IFRS as adopted by the EU.

The consolidated statement of profit and loss data of argenx SE as of December 31, 2018 presented in this press release is unaudited.

Annual Report 2018

argenx expects to publish its 2018 Annual Report based on IFRS as issued by the IASB and its 2018 Annual Report for statutory purposes based on IFRS as adopted by the EU on March 26, 2019. These Annual Reports will be available on www.argenx.com.

EXPECTED 2019 FINANCIAL CALENDAR

· May 7, 2019: Annual General Meeting

· May 9, 2019: Q1 2019 business update and financial results

· August 1, 2019: HY 2019 business update and financial results

· October 24, 2019: Q3 2019 business update and financial results

Dial-in numbers:

Please dial in 5–10 minutes prior to 3 p.m. CET/ 9 a.m. ET using the number and conference ID below.

Confirmation Code: 9795988

Belgium

+32 (0)2 400 9874

Belgium

0800 48740

France

+33 (0)1 767 00794

France

0805 103028

Netherlands

+31 (0)20 714 3545

Netherlands

0800 0249557

United Kingdom

+44 (0)844 571 8892

United Kingdom

0800 376 7922

United States

+1 (631) 510 7495

United States

+1 (866) 966 1396

A question and answer session will follow the presentation of the results. Go to www.argenx.com to access the live audio webcast. The archived webcast will also be available (90 days) for replay shortly after the close of the call from the "Downloads" section of the argenx website.

on February 28, 2019 Intrexon Corporation (NASDAQ: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported its fourth quarter and full year financial results for 2018 (Press release, Intrexon, FEB 28, 2019, View Source [SID1234533838]).

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Recent Business Highlights:

Precigen, Inc., a wholly owned subsidiary of Intrexon, announced the US Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for PRGN-3006, an investigational drug for patients with relapsed or refractory acute myeloid leukemia (AML) and higher risk myelodysplastic syndrome (MDS). PRGN-3006 is an autologous chimeric antigen receptor T-cell (CAR-T) therapeutic candidate utilizing Precigen’s transformative UltraCAR-T platform, which reduces manufacturing time to within two days following non-viral gene transfer;

Precigen announced the FDA cleared the IND application for PRGN-3005 UltraCAR-T, an investigational drug using CAR-T cells to treat advanced-stage platinum-resistant ovarian cancer patients and the first UltraCAR-T candidate targeting solid tumors to enter the clinic;

Following the previously reported reacquisition of oncology rights from Ziopharm in October 2018, Precigen and Intrexon entered into an agreement with Merck KGaA, Darmstadt, Germany, a leading science and technology company, and its wholly owned subsidiary Ares Trading, pursuant to which Intrexon assumed rights from Ares Trading under the existing agreement among Precigen, Ares Trading and Ziopharm relating to the development of CAR-T therapies. In addition to receiving 20,640,119 shares of Intrexon common stock, the agreement also included a further $25 million investment in the Company. In return, Merck KGaA, Darmstadt, Germany, received a $25 million convertible note, providing the option to receive either Precigen or Intrexon common stock;

Xogenex, a majority owned subsidiary of Intrexon, has completed an evaluation of three advanced heart failure patients who were administered INXN-4001, an investigational, non-viral, plasmid-based gene drug candidate designed to drive expression of three cardiac effector genes involved in heart failure, in a Phase 1 clinical trial. The data reflected the patients’ status six-months after being given the INXN-4001 and appears to indicate that the drug material and the delivery process were both well tolerated by the patients. Preliminary review of the data suggests improvements in several cardiac performance parameters;

Intrexon’s methane bioconversion platform is being employed to produce 2,3 BDO from natural gas and has achieved 80% of the goal for the first small-scale plant operations;

Detailed engineering design for Intrexon’s first-of-a-kind small-scale methane bioconversion facility to 2,3 BDO is currently being bid out, discussions with partners for sites are ongoing. The overall schedule is still consistent as the sites under consideration are brownfield which require less engineering time than the original greenfield concept;

Exemplar Genetics, a wholly owned subsidiary of Intrexon, announced the launch of a joint venture with the Mayo Clinic, which is focused on the development of a high-quality source of human liver cells or hepatocytes (HHCs);

Trans Ova Genetics, a wholly owned subsidiary of Intrexon, continues to expand and improve its herd genetics with two Jersey heifers ranking 2nd and 9th in the world and 15 bulls that rank at the top of the global Holstein bull population;

EnviroFlight, Intrexon’s joint venture with Darling Ingredients Inc., opened the very first commercial Black Soldier Fly (BSFL) facility, in Maysville, Kentucky in November 2018. The production plant has the ability to produce 900 tonnes/year of dried BSFL, and orders for product from the new facility account for one-third of the anticipated annual output;

Okanagan Specialty Fruits, a wholly owned subsidiary of Intrexon, harvested more than 2,100 bins of Arctic apples in their 2018 harvest, which are available at select retailers as fresh sliced apples and ApBitz dehydrated apple snacks, and is planning to plant up to 1,000,000 trees in the spring of 2019;

Intrexon entered into a strategic licensing agreement with Next Green Wave Holdings Inc. to utilize Intrexon’s Botticelli next generation plant propagation platform to enable rapid production of Next Green Wave’s proprietary cannabis cultivars for the California market;

Oxitec, Ltd., a wholly owned subsidiary of Intrexon, announced that it will be transitioning from its 1st generation self-limiting Friendly Aedes aegypti mosquito (OX513A) to a new Friendly Aedes mosquito (OX5034) that uses Oxitec’s 2nd generation technology, allowing the company to focus on advancing its entire mosquito and crop pest portfolios using this next-generation platform; and

Oxitec has secured two multi-year development agreements with a partner to develop solutions for pest problems beyond the mosquito, which have the potential for application in key markets globally.

Fourth Quarter 2018 Financial Highlights:

Total revenues of $43.2 million, a decrease of 44% from the fourth quarter of 2017;

Net loss of $340.5 million attributable to Intrexon, or $(2.59) per basic share, including non-cash charges of $311.0 million;

Adjusted EBITDA of $(27.2) million, or $(0.21) per basic share; and

Cash, cash equivalents, and short-term investments totaled $222.5 million and the value of common equity securities totaled $2.2 million at December 31, 2018.

Full Year 2018 Financial Highlights:

Total revenues of $160.6 million, a decrease of 31% from the full year ended December 31, 2017;

Net loss of $509.3 million attributable to Intrexon, or $(3.93) per basic share, including non-cash charges of $420.0 million; and

Adjusted EBITDA of $(102.5) million, or $(0.79) per basic share.

"With several of the most ambitious stated objectives at the time of our IPO now achieved, most notably with our work in UltraCAR-T and in natural gas upgrading, our team is more energized than ever to establish its tangible value in the world," commented Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon. "We expect to achieve this initially through independent equitization and/or monetization events at Precigen and from our Methane Bioconversion Platform, while other business units of our company may also find higher values independently of our company."

Mr. Kirk concluded, "It has always been our purpose to be an enabler of enterprise that is built on engineered biology and not to create an industrial conglomerate. In this we have succeeded greatly so we look forward to seeing some of our bold enterprises making their marks in the world. We are confident in our prospects to achieve this and that we have adequate cash resources to fuel us to these realizations."

Fourth Quarter 2018 Financial Results Compared to Prior Year Period

Total revenues decreased $33.8 million, or 44%, from the quarter ended December 31, 2017. Collaboration and licensing revenues decreased $30.9 million from the quarter ended December 31, 2017 due to (i) the mutual termination in 2017 of the Company’s second exclusive channel collaboration (ECC) with Ziopharm for the treatment of graft-versus-host disease, (ii) a decrease in research and development services for certain of the Company’s ECCs as the Company redeployed certain resources towards supporting prospective new platforms and partnering opportunities and began to focus more on the further development of relationships and structures that will provide the Company with more control and ownership over the development process and commercialization path, including programs where the Company reacquired the previously licensed technology rights in 2018, and (iii) a decrease in research and development services performed by the Company for collaborators upon the transition of program execution to its collaborators. Product revenues decreased $2.8 million or 36% primarily due to lower milk prices which resulted in lower customer demand for cows and cloned products. Gross margin on products declined in the current period as a result of decreased sales. Gross margin on services improved in the current period as a result of pricing changes and an increase in the number of embryos produced per bovine in vitro fertilization cycle due to improved production results.

Research and development expenses increased $242.0 million, or 628%, and include $228.0 million of expenses related to in-process research and development reacquired from former collaborators. Selling, general and administrative (SG&A) expenses decreased $7.9 million, or 24%. This decrease was primarily due to lower compensation expenses due to adjustments to previously accrued compensation expenses for performance and retention incentives for SG&A employees in 2018. The Company recorded an impairment charge of $60.5 million in the fourth quarter of 2018 due to a change in the Company’s business strategy for commercializing the Oxitec technology targeting the Aedes aegypti mosquito.

Full Year 2018 Financial Results Compared to Prior Year Period

Total revenues decreased $70.4 million, or 31%, from the year ended December 31, 2017. Collaboration and licensing revenues decreased $68.7 million from the year ended December 31, 2017 primarily due to (i) the mutual termination in 2017 of the Company’s second ECC with Ziopharm for the treatment of graft-versus-host disease, (ii) a decrease in research and development services for certain of the Company’s ECCs as the Company redeployed certain resources towards supporting prospective new platforms and partnering opportunities and began to focus more on the further development of relationships and structures that provide the Company with more control and ownership over the development process and commercialization path, including programs where the Company reacquired the previously licensed technology rights in 2018, and (iii) a decrease in research and development services performed by the Company for collaborators upon the transition of program execution to its collaborators. Product revenues decreased $5.1 million or 15% primarily due to lower milk prices which in turn resulted in lower customer demand for live calves, cows previously used in production, and cloned products. Gross margin on products declined in the current period as a result of lower product sales and increased operating costs associated with new product offerings and cloned products. The increase in service revenues of $1.8 million, or 4%, as well as the gross margin thereon relates to pricing changes and an increase in the number of embryos produced per bovine in vitro fertilization cycle due to improved production results.

Research and development expenses increased $261.4 million, or 183%, and include $236.7 million of expenses related to in-process research and development reacquired from former collaborators. SG&A expenses decreased $8.3 million, or 6%, from the prior period. Legal and professional fees decreased $7.5 million primarily due to (i) decreased legal fees associated with ongoing litigation and (ii) decreased fees incurred for regulatory and other consultants. The Company recorded an impairment charge of $60.5 million in the fourth quarter of 2018 due to a change in the Company’s business strategy for commercializing the Oxitec technology targeting the Aedes aegypti mosquito.

Total other income (expense), net, decreased $41.5 million, or 185%. This decrease was primarily attributable to losses on the Company’s investment in Ziopharm preferred stock prior to returning this investment to Ziopharm in the fourth quarter of 2018, as well as an increase in interest expense related to the 3.5% convertible notes issued by the Company in the third quarter of 2018.

Based on Intrexon’s financial position, including its cash, cash equivalents and short-term investments of $224 million at December 31, 2018, in connection with issuing its financial statements Intrexon expects to include a conclusion in its Form 10-K that there is substantial doubt about its ability to continue as a going concern.

Conference Call and Webcast

The Company will host a conference call today Thursday, February 28th, at 5:30 PM ET to discuss the fourth quarter and full year 2018 financial results and provide a general business update. The conference call may be accessed by dialing 1-888-317-6003 (Domestic US), 1-866-284-3684 (Canada), and 1-412-317-6061 (International) and providing the number 4443860 to join the Intrexon Corporation Call. Participants may also access the live webcast through Intrexon’s website in the Investors section at View Source

On February 28, 2018 Pacira Pharmaceuticals, Inc. (NASDAQ: PCRX) reported financial results for the fourth quarter and full year of 2018 and its outlook for 2019 (Press release, Pacira Pharmaceuticals, FEB 28, 2019, View Source;p=RssLanding&cat=news&id=2389460 [SID1234533797]).

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2018 Financial Highlights

Fourth quarter EXPAREL net product sales of $94.4 million

Full year EXPAREL net product sales of $331.1 million

Fourth quarter GAAP net income of $8.3 million or $0.20 per diluted share

Fourth quarter non-GAAP net income of $19.8 million or $0.47 per diluted share

Full year GAAP net loss of $0.5 million or $0.01 per basic and diluted share

Full year non-GAAP net income of $43.5 million or $1.04 per diluted share
"Pacira had a landmark 2018 with record revenues underscoring the growing marketplace adoption of EXPAREL as a fundamental component of opioid-sparing pain management protocols at influential academic centers and also the success of our brachial plexus nerve block launch," said Dave Stack, chairman and chief executive officer of Pacira. "There is great enthusiasm among a growing number of anesthesiologists who are using regional nerve blocks and field blocks to revolutionize postsurgical pain management and improve patient outcomes. We are also seeing robust growth in new accounts driven primarily by ambulatory, plastic, and oral surgery centers seeking to differentiate their practices by providing patients with an effective, long-acting non-opioid option for managing postsurgical pain. Finally, our significant partnership with Johnson and Johnson continues to drive uptake with EXPAREL being integrated across its world class educational platforms and comprehensive offering of orthopedic procedural solutions. Altogether this strong growth trajectory provides us with a solid operating foundation to support the strategic expansion of our commercial and clinical offering within the non-opioid pain management and regenerative health space."

"This substantial momentum has continued into 2019 and we plan to build on our success by executing on multiple growth opportunities, including expanding the role of EXPAREL as a catalyst for shifting inpatient procedures to the ambulatory setting, and building out our differentiated non-opioid portfolio with innovative products that address the need for improving patients’ journeys along the neural pain pathway. We believe this strategy provides the greatest opportunities to build value in both the near- and long-term," concluded Mr. Stack.

Recent Highlights

EXPAREL manufacturing capacity expansion to meet growing demand. In February 2019, Pacira announced that commercial production of EXPAREL is now underway at a custom suite in Swindon, UK created under the company’s partnership with Thermo Fisher Scientific Pharma Services (formerly Patheon UK Limited). This suite was designed to mirror the company’s existing facility at the Pacira Science Center Campus in San Diego, CA and is expected to double the company’s manufacturing capacity. Through the partnership, the companies are developing a second dedicated suite that is expected to enable another doubling of EXPAREL manufacturing capacity in approximately two years.

Phase 4 EXPAREL study achieves statistically significant reductions in opioid consumption and pain scores in C-Section patients. In January 2019, Pacira announced that its Phase 4 study of EXPAREL in patients undergoing Cesarean section (C-section) achieved its primary endpoint with a statistically significant reduction in total postsurgical opioid consumption through 72 hours (P<0.05). EXPAREL also achieved statistical significance for reduction in pain intensity scores through 72 hours (P<0.05). The full study results will be submitted for publication in peer-reviewed medical literature later this year.

Centers for Medicare and Medicaid Services establish new reimbursement for EXPAREL. In November 2018, the Centers for Medicare and Medicaid Services (CMS) finalized a policy to provide separate Medicare reimbursement for EXPAREL when administered in ambulatory surgical centers (ASCs) through establishment of the product-specific billing code of C9290. This code, which provides payment for EXPAREL at $1.22 per milligram, sets national Medicare reimbursement rates for EXPAREL administered in ASCs. In addition, the American Dental Association published a CDT code (D9613) to report infiltration of a sustained-release therapeutic drug in oral surgery procedures. Both codes became effective on January 1, 2019.

New data show EXPAREL significantly reduces opioid use, hospital length of stay and total hospitalization costs following TKA. In November 2018, Pacira announced new data were published in The Journal of Medical Economics on the use of EXPAREL to manage postsurgical pain following total knee arthroplasty (TKA). The findings showed that patients receiving EXPAREL had a significant reduction in opioid use, hospital length of stay (LOS), and total hospitalization costs compared to TKA patients who did not receive EXPAREL. Patients receiving EXPAREL also had increased likelihood to be discharged home rather than a skilled nursing facility.
Fourth Quarter 2018 Financial Results

EXPAREL net product sales were $94.4 million in the fourth quarter of 2018, a 20% increase over the $78.7 million reported for the fourth quarter of 2017.

Total revenues were $95.1 million in the fourth quarter of 2018, a 20% increase over the $79.1 million reported for the fourth quarter of 2017.

Total operating expenses were $82.9 million in the fourth quarter of 2018, compared to $70.6 million in the fourth quarter of 2017.

GAAP net income was $8.3 million, or $0.20 per share (basic and diluted) in the fourth quarter of 2018, compared to GAAP net income of $4.6 million, or $0.11 per share (basic and diluted), in the fourth quarter of 2017.

Non-GAAP net income was $19.8 million, or $0.48 per share (basic) and $0.47 per share (diluted), in the fourth quarter of 2018, compared to non-GAAP net income of $16.0 million, or $0.39 per share (basic) and $0.38 per share (diluted), in the fourth quarter of 2017.

Pacira had 41.1 million basic weighted average shares of common stock outstanding in the fourth quarter of 2018.

Pacira had 42.2 million diluted weighted average shares of common stock outstanding in the fourth quarter of 2018.
Full-Year 2018 Financial Results

EXPAREL net product sales were $331.1 million in 2018, a 17% increase over the $282.9 million reported in 2017.

Total revenues were $337.3 million in 2018, an 18% increase over the $286.6 million reported in 2017.

Total operating expenses were $321.4 million in 2018, compared to $311.6 million in 2017.

GAAP net loss was $0.5 million, or $0.01 per share (basic and diluted) in 2018, compared to a GAAP net loss of $42.6 million, or $1.07 per share (basic and diluted) in 2017.

Non-GAAP net income was $43.5 million, or $1.06 per share (basic) and $1.04 per share (diluted), in 2018, compared to non-GAAP net income of $8.6 million, or $0.22 per share (basic) and $0.21 per share (diluted), in 2017.

Pacira ended 2018 with cash, cash equivalents, short-term and long-term investments ("cash") of $409.3 million.

Pacira had 40.9 million basic weighted average shares of common stock outstanding in 2018.

For non-GAAP measures, Pacira had 41.9 million diluted weighted average shares of common stock outstanding in 2018.
2019 Outlook

Pacira announces its full year 2019 financial guidance as follows. Pacira expects:

EXPAREL net product sales of $400 million to $410 million.

Non-GAAP gross margins of 75% to 76%.

Non-GAAP research and development (R&D) expense of $60 million to $70 million.

Non-GAAP selling, general and administrative (SG&A) expense of $165 million to $175 million.

Stock-based compensation of $30 million to $35 million.
See "Non-GAAP Financial Information" and "Reconciliations of GAAP to Non-GAAP 2019 Financial Guidance" below.

Today’s Conference Call and Webcast Reminder

The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Thursday, February 28, 2019, at 8:30 a.m. ET. The call can be accessed by dialing 1-877-845-0779 (domestic) or 1-720-545-0035 (international) ten minutes prior to the start of the call and providing the Conference ID 6050757.

A replay of the call will be available approximately two hours after the completion of the call and can be accessed by dialing 1-855-859-2056 (domestic) or 1-404-537-3406 (international) and providing the Conference ID 6050757. The replay of the call will be available for two weeks from the date of the live call.

The live, listen-only webcast of the conference call can also be accessed by visiting the "Investors & Media" section of the company’s website at investor.pacira.com. A replay of the webcast will be archived on the Pacira website for two weeks following the call.

Non-GAAP Financial Information

This press release contains financial measures that do not comply with U.S. generally accepted accounting principles (GAAP), such as non-GAAP net income (loss), non-GAAP net income (loss) per share, non-GAAP cost of goods sold, non-GAAP gross margins, non-GAAP research and development (R&D) expense and non-GAAP selling, general and administrative (SG&A) expense, because such measures exclude milestone revenue, stock-based compensation, amortization of debt discount, loss on early extinguishment of debt, exit costs related to the discontinuation of DepoCyt(e) production and loss on an unexercised investment purchase option.

These measures supplement the company’s financial results prepared in accordance with GAAP. Pacira management uses these measures to better analyze its financial results, estimate its future cost of goods sold, gross margins, R&D expense and SG&A expense outlook for 2019 and to help make managerial decisions. In management’s opinion, these non-GAAP measures are useful to investors and other users of our financial statements by providing greater transparency into the operating performance at Pacira and the company’s future outlook. Such measures should not be deemed to be an alternative to GAAP requirements or a measure of liquidity for Pacira. Non-GAAP measures are also unlikely to be comparable with non-GAAP disclosures released by other companies. See the tables below for a reconciliation of GAAP to non-GAAP measures, and a reconciliation of our GAAP to non-GAAP 2019 financial guidance for gross margins, R&D expense and SG&A expense.

on February 28, 2019 Heat Biologics, Inc. (NASDAQ: HTBX), a biopharmaceutical company developing immunotherapies designed to activate a patient’s immune system against cancer, reported updated interim results from its ongoing Phase 2 study investigating HS-110 in combination with Bristol-Myers Squibb’s anti-PD-1 checkpoint inhibitor, nivolumab (Opdivo), in patients with advanced non-small cell lung cancer (NSCLC) (Press release, Heat Biologics, FEB 28, 2019, View Source [SID1234533839]). The results were presented today at the ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium by Daniel Morgensztern, M.D., Associate Professor of Medicine and Director of Thoracic Oncology, Washington University School of Medicine, and Lead Investigator in the trial. Data were presented on both Cohort A and Cohort B of the trial. Cohort A enrolls only previously treated patients who have never received a checkpoint inhibitor (CPI), while Cohort B enrolls patients who received a minimum of 4 months of treatment with a CPI as part of their prior therapy, but subsequently had documented progressive disease.

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"The treatment landscape for NSCLC has fundamentally changed as the number of patients who receive first line checkpoint inhibitor therapy is rapidly increasing," said COL(ret) George E Peoples, MD, FACS, Heat’s Chief Medical Advisor. "The preliminary data from our Cohort B is increasingly relevant and potentially exciting as it suggests that the addition of HS-110 to nivolumab may restore anti-tumor activity in patients whose disease has progressed after treatment with a CPI."

Jeff Hutchins, Ph.D., Chief Scientific and Operating Officer of Heat said, "The observed response rates and durability of disease stabilization support our mechanistic hypothesis that the broad, T-cell mediated immune response activated by HS-110 may improve patient survival when administered in combination with a CPI. The Cohort B data suggest that HS-110 may improve clinical outcomes for patients who have lost the benefit of treatment with a checkpoint inhibitor. We look forward to completing enrollment in this trial in Q2 and releasing additional results later this year as the data matures."

Highlights for both cohorts are presented below:

Cohort B (patients who progressed after ≥ 4 months of prior treatment with a checkpoint inhibitor)

Of first 20 patients enrolled in this cohort:

Partial response (PR) in 3 patients (15%) per RECIST 1.1 and 4 patients (20%) per investigator assessment

Disease control rate (DCR) of 55%

The 3 RECIST 1.1 PR patients had documented progression on CPI monotherapy immediately preceding study entry

Median progression free survival (mPFS) was 2.7 months (95% CI; 1.8 – 4.0 months)

Cohort A (patients who have never received a CPI prior to study entry)

Of 42 patients enrolled by the cutoff date:

PR in 9 patients (21%) per RECIST 1.1

DCR of 50%

Median overall survival not yet reached (60% still alive with a median follow-up of 14.4 months)

Responses and disease stabilization are durable and long-lasting

Subgroup analyses, predefined in the clinical protocol, were performed for levels of tumor-infiltrating lymphocytes (CD8+ TILs) present in tumors at baseline. A survival benefit [hazard ratio (HR) = 0.39] was observed in patients with levels CD8+ TIL <10% (i.e. "cold" tumors), a population that typically responds poorly to checkpoint inhibitors. The treatment benefit appeared to be independent of PD-L1 status (HR = 0.85)

Immune reactivity to HS-110 was measured via ELISPOT assay (high vs. low compared to median) on patient peripheral blood mononuclear cells obtained before and during treatment with a median overall survival benefit of 6.2 months in the high ELISPOT group

Overall survival was significantly higher in patients that experienced at least one dermal injection site reaction to HS-110 at any time during study treatment, supporting HS-110’s mechanism of action (HR = 0.15 [95% CI: 0.05-0.45], p=0.0001)

For both cohorts, treatment with HS-110 in combination with nivolumab was well tolerated, with no additional toxicities beyond those observed with single agent CPI therapy.

Importantly, data from Cohort B suggest that HS-110 in combination with nivolumab reduced tumor burden in patients whose disease progressed after treatment with a checkpoint inhibitor at any time prior to study entry. Additionally, the deepest responses were observed in three patients whose last treatment immediately preceding enrollment was checkpoint inhibitor monotherapy.

Also of interest is the data regarding overall survival (OS) in patients with low CD8+ TIL (tumor infiltrating lymphocytes) at baseline. Protocol-defined subgroup analysis of patients categorized as ‘high’ or low’ TIL, based on levels of CD8+ cells present in the stroma of their tumor tissue at baseline, demonstrate a survival advantage for the ‘low TIL’ group as compared to the ‘high TIL’ group (not reached vs. 13.8 months; HR = 0.39 [95% CI; 0.06-2.31]. These data are very encouraging as prior studies with nivolumab alone suggest that "cold" tumor patients with lower levels of baseline CD8+ TILs have lower response rates compared to "hot" tumor patients with high levels of CD8+ TILs1.

Trial results are summarized in the company’s updated corporate presentation, along with the official ASCO (Free ASCO Whitepaper)-SITC poster.

Trial Design

The Phase 2 trial is designed to evaluate the safety and efficacy of HS-110 combined with an immune checkpoint inhibitor for the treatment of advanced non-small cell lung cancer. Patients receive weekly HS-110 (1 x 107 cells) administered as 5 intradermal 0.1 mL injections for 18 weeks in combination with bi-weekly nivolumab 240 mg IV administered until confirmed disease progression or unacceptable toxicity, whichever occurs first. The primary endpoint is objective response rate (ORR); secondary endpoints include overall survival (OS), progression-free survival (PFS), disease control rate (DCR) and duration of response (DOR). Exploratory endpoints include correlation of clinical outcomes to baseline CD8+ TILs, PD-L1 expression, peripheral blood tumor mutation burden and ELISPOT analysis.

For further details about the trial and the results presented at ASCO (Free ASCO Whitepaper)-SITC, refer to Heat Bio’s updated corporate presentation, which can be found on the Investors tab of the corporate website View Source

On February 28, 2019 G1 Therapeutics, Inc. (Nasdaq: GTHX), a clinical-stage oncology company, reported financial results for the fourth quarter and full-year ended December 31, 2018 (Press release, G1 Therapeutics, FEB 28, 2019, View Source [SID1234533798]). The company also highlighted 2018 operational results and upcoming 2019 milestones.

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"Data from four randomized Phase 2 trials showed the benefits of trilaciclib across different indications, lines of therapy and chemotherapy regimens," said Raj Malik, M.D., Chief Medical Officer and Senior Vice President, R&D. "We have scheduled meetings with U.S. and European regulatory authorities in the first half of 2019 to discuss the totality of data and next steps for the development of trilaciclib. We will provide an update on these meetings in the second quarter."

"We made substantial progress across our three clinical-stage product candidates in 2018. We reported positive results from all four Phase 2 trilaciclib trials, presented proof-of-concept data on lerociclib in breast cancer, initiated a trial of lerociclib in non-small cell lung cancer, and brought our oral selective estrogen receptor degrader G1T48 into the clinic," said Mark Velleca, M.D., Ph.D., Chief Executive Officer. "These accomplishments will drive a number of important clinical and regulatory milestones in 2019 in the advancement of our pipeline."

Corporate Highlights

Reported positive multi-lineage myelopreservation data from three randomized, double-blind, placebo-controlled Phase 2 trials of trilaciclib in small cell lung cancer (SCLC): In the fourth quarter, the company presented additional data from its Phase 2 trial of trilaciclib in combination with chemotherapy in first-line SCLC at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress, and reported preliminary data from Phase 2 trials in first-line SCLC in combination with chemotherapy/Tecentriq (atezolizumab) and second/third-line SCLC in combination with chemotherapy. In these trials, patients receiving trilaciclib showed statistically significant improvements in duration and occurrence of severe neutropenia (primary endpoints) and clinically meaningful reductions in G-CSF administrations and red blood cell transfusions. Treatment was well tolerated and the safety profile of trilaciclib was consistent across the three trials.

Presented preliminary improved progression-free survival data from randomized Phase 2 trial of trilaciclib in combination with chemotherapy in patients with metastatic triple-negative breast cancer (mTNBC): In December, the company presented data from its Phase 2 trial of trilaciclib in patients with mTNBC at the 2018 San Antonio Breast Cancer Symposium. Preliminary median progression-free survival (PFS) was 5.4 months in the chemotherapy arm, 8.8 months in the chemotherapy and trilaciclib (dosed the day of chemotherapy) arm (hazard ratio 0.52, p=0.0669), and 7.3 months in the chemotherapy and trilaciclib (dosed the day prior to and day of chemotherapy) arm (hazard ratio 0.49; p=0.0546). A combined analysis of trilaciclib-treated patients showed PFS of 5.4 months for the chemotherapy arm and 7.9 months for chemotherapy and trilaciclib (hazard ratio 0.50, p=0.0189). Patients on trilaciclib received more chemotherapy cycles than those in the control arm. The safety profile of trilaciclib was consistent with previously reported trials; no trilaciclib-related serious adverse events were reported.

Anticipated Milestones for 2019

Meet with U.S. and European regulatory authorities in the first half of 2019 and announce the next steps for trilaciclib development in the second quarter of 2019.

Initiate additional randomized trials for trilaciclib in the second half of 2019, pending feedback from regulatory authorities.

Report additional data from all four randomized Phase 2 trilaciclib clinical trials.

Present additional data from the Phase 1b clinical trial of lerociclib/Faslodex (fulvestrant) in ER+, HER2- breast cancer in the second half of 2019.

Present preliminary dose-escalation data from the Phase 1b clinical trial of lerociclib/Tagrisso (osimertinib) in non-small cell lung cancer in the second half of 2019.

Present preliminary dose-escalation data from the Phase 1 clinical trial of G1T48, an oral selective estrogen receptor degrader (SERD), in ER+ breast cancer in the second half of 2019.

Fourth Quarter and Full-Year 2018 Financial Highlights

Cash Position: Cash, cash equivalents and short-term investments totaled $369.3 million as of December 31, 2018, compared to $390.5 million as of September 30, 2018, and $103.8 million as of December 31, 2017.

Operating Expenses: Operating expenses were $26.1 million for the fourth quarter of 2018, compared to $17.3 million for the fourth quarter of 2017. GAAP operating expenses include stock-based compensation expense of $3.3 million for the fourth quarter of 2018, compared to $1.0 million for the fourth quarter of 2017. Operating expenses for the full-year 2018 were $89.3 million, compared to $61.0 million for the prior year. Stock-based compensation expense for the full-year 2018 was $10.2 million, compared to $3.4 million for the prior year.

Research and Development Expenses: Research and development (R&D) expenses for the fourth quarter of 2018 were $19.1 million, compared to $15.1 million for the fourth quarter of 2017. The increase in expense was due to an increase in clinical program costs, drug manufacturing costs to support clinical programs, external research studies and personnel costs due to additional headcount. R&D expenses for the full-year 2018 were $70.7 million, compared to $53.9 million for the prior year.

General and Administrative Expenses: General and administrative (G&A) expenses for the fourth quarter of 2018 were $7.0 million, compared to $2.2 million for the fourth quarter of 2017. The increase in expense was largely due to an increase in professional fees and personnel-related costs. G&A expenses for the full-year 2018 were $18.6 million, compared to $7.1 million for the prior year.

Net Loss: G1 reported a net loss of $24.1 million for the fourth quarter of 2018, compared to $17.0 million for the fourth quarter of 2017. Net loss for the full-year 2018 was $85.3 million, compared to a net loss of $60.1 million for the prior year.

Webcast and Conference Call

The management team will host a webcast and conference call at 4:30 p.m. ET today to provide a financial update for the fourth quarter and full-year of 2018. The live call may be accessed by dialing 866-763-6020 (domestic) or 210-874-7713 (international) and entering the conference code: 2698949. A live and archived webcast will be available on the Events & Presentations page of the company’s website: www.g1therapeutics.com. The webcast will be archived on the same page for 90 days following the event.

Clinical program updates will be provided at the Investor Day 2019 meeting on March 6.