On August 7, 2019 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat age-related diseases and extend healthy lifespan, reported its financial results for the second quarter ended June 30, 2019 (Press release, CohBar, AUG 7, 2019, View Source [SID1234538366]).

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"During the quarter, we were pleased to resume the dosing of subjects in the Phase 1a/1b clinical trial of our lead candidate CB4211 for NASH and obesity, while continuing to progress our evaluation of additional novel peptides targeting fibrotic diseases, cancer, and type 2 diabetes," said Steven Engle, Chief Executive Officer. "We believe that CB4211 is the first of a number of candidates that our technology platform will identify for advancement into the clinic. Our seminar in June also provided strong validation from world class researchers and CohBar scientists of the potential of mitochondrial based therapeutics, as well as CohBar’s position as a leader in this emerging field. It’s an exciting time to be at CohBar."

Business Highlights

●Resumed CB4211 clinical trial: Dosing of subjects for the company’s Phase 1a/1b clinical trial of CB4211 resumed in June, and has continued in the current quarter for its lead MBT candidate for the potential treatment of nonalcoholic steatohepatitis (NASH) and obesity. The trial design is available at www.clinicaltrials.gov.

●Advanced new potential disease targets: During the second quarter, the company advanced its evaluation and optimization of novel analogs of mitochondrial-derived peptides for the potential treatment of fibrotic diseases. Preclinical studies provided preliminary evidence of anti-fibrotic activity in cell-based and animal models of idiopathic pulmonary fibrosis. Fibrotic disease is a major unmet medical need contributing to approximately one-third of deaths worldwide. The company also advanced its evaluation of novel analogs for the potential treatment of cancer, where in vitro data demonstrated the potential for CohBar peptides to enhance the killing of cancer cells by human immune cells. The market for cancer immunotherapy is projected to grow significantly to over $100 billion by 2023.

●Presented a novel scientific discovery at the American Diabetes Association annual conference: In June, CohBar presented its recent discovery of a mechanism of action of a family of novel peptides with positive effects on glucose tolerance in animal models of type 2 diabetes. The studies showed novel peptide analogs of CB5064 are effective in diet induced obese (DIO) mice, a widely used model of type 2 diabetes. Additional studies also demonstrated that CB5064 interacts with the apelin receptor, a key cell surface receptor involved in the regulation of glucose utilization, fluid homeostasis and cardiovascular function. Click link to ADA poster.

●Appointed new CEO and expanded Board of Directors: In May, Steven Engle was appointed as the company’s Chief Executive Officer. Mr. Engle has over two decades of executive leadership experience with public biotech companies developing breakthrough products in metabolic, autoimmune, oncologic and infectious disease areas. In April, the company expanded its Board with the addition of David Greenwood, who brings more than 30 years of financial and operational experience in biotechnology and investment banking.

●Hosted Mitochondria Based Therapeutics Seminar with key opinion leaders: In May, the company hosted a seminar, entitled "Mitochondria, a Source for Novel Therapeutics," which featured CohBar’s founders, Dr. Pinchas Cohen and Dr. David Sinclair, and CohBar’s Chief Science Officer, Dr. Ken Cundy, discussing the role of mitochondria in health and aging, and mitochondrial-derived peptides (MDPs) as a novel source of potential therapeutics for a host of major age-related diseases. A recording of the webcast is available at www.cohbar.com or by clicking on this link.

●Held Annual General Meeting and review: In June, the company held its Annual General meeting and provided a review and update on the company’s progress and plans. The slides are available on the company’s website at www.cohbar.com.

During the second quarter and more recently, Dr. Pinchas Cohen and Dr. Nir Barzilai continued to be recognized as international leaders in the study of mitochondrial science, aging and age-related diseases:

●Dr. Cohen delivered a keynote presentation on "Systems Biology of the Mitochondria in Aging" at the American Aging Association Annual Meeting in San Francisco, in May 2019; and "Addressing the Challenges of an Aging Society Through Innovation" at a panel entitled "Forum on Aging," at the G20 Meeting in Japan in June 2019. In addition, Dr. Cohen co-authored "Metabolomic profile of diet-induced obesity mice in response to humanin and small humanin-like peptide 2 treatment," published in Metabolomics, and "The mitochondrial-derived peptide MOTS-c is a regulator of plasma metabolites and enhances insulin sensitivity," published in Physiological Reports. He also authored an editorial published in The Journal of Molecular Medicine entitled "MOTS-c: an equal opportunity insulin sensitizer."

●Dr. Barzilai delivered a keynote presentation, entitled "Advancements in Genomics," at the Glenn Center Harvard Symposium in May 2019, and the keynote address at the Nutrition and Obesity Research Center (NORC) at the Harvard Annual Symposium in July 2019. In addition, Dr. Barzilai presented data at the Biology of Aging, Gordon Research Conference on Molecular, Cellular and Physiological Determinants of Lifespan and Healthspan. More recently, Dr. Barzilai co-authored three papers on "Creating the Next Generation of Translational Geroscientists," "The Development of Clinical Trials to Extend Healthy Lifespan," and a highly publicized paper in Nature on exome sequencing in type 2 diabetes.

Financial Highlights

●Cash and Investments. CohBar had cash and investments of $16.8 million as of June 30, 2019, compared to $22.2 million as of December 31, 2018. The cash burn for the quarter ended June 30, 2019, was approximately $2.9 million.

●R&D Expenses. Research and development expenses were $1.4 million in the three months ended June 30, 2019, compared to $1.8 million in the prior year quarter. The decrease was primarily due to the timing of certain preclinical and clinical costs incurred in the prior year period partially offset by an increase in costs associated with our research programs focused on the continuing development of peptides.

●G&A Expenses. General and administrative expenses were $1.5 million for the three months ended June 30, 2019, compared to $1.3 million in the prior year quarter. The increase in general and administrative expenses was primarily due to legal expenses related to the protection of our intellectual property, recruiting costs, and increased director fees.

●Net Loss. For the three months ended June 30, 2019, net loss, which included $0.8 million of non-cash expenses, was $3.1 million, or $0.07 per basic and diluted share. For the three months ended June 30, 2018, net loss, which included $0.9 million of non-cash expenses, was $3.3 million, or $0.08 per basic and diluted share.

Second Quarter Investor Call and Slide Presentation:

Date: August 7, 2019

Time: 5:00 p.m. EDT (2:00 p.m. PDT)

Conference Audio

-Dial-in U.S. and Canada: (800) 239-9838
-Dial-in International: (323) 794-2551
-Conference ID No.: 8671787

Slide Presentation

-Go to www.webex.com, click on the ‘Join’ button and enter meeting number 923 119 468 and Password CWBR, or
-Go to www.cohbar.com and click on Q2 2019 Shareholder Presentation at top of homepage.

For individuals participating in the Investor Call and Slide Presentation, please call into the conference audio and log into Webex approximately 10 minutes prior to its start.

An audio recording of the call will be available beginning at 8:00 p.m. (EDT) on August 7, 2019, through August 28, 2019. To access the recording please dial 1-844-512-2921 in the U.S. and Canada, or 1-412-317-6671 internationally, and reference Conference ID No. 8671787. The audio recording along with the slide presentation will also be available at www.cohbar.com during the same period.

About CB4211

CohBar’s lead clinical program is based on CB4211, a first-in-class mitochondria based therapeutic, that has demonstrated significant therapeutic potential in preclinical models of nonalcoholic steatohepatitis (NASH) and obesity. CB4211 is a novel and improved analog of MOTS-c, a naturally occurring mitochondrial-derived peptide, which has been shown to play a significant role in the regulation of metabolism, and was discovered in 2012 by CohBar founder Dr. Pinchas Cohen and his academic collaborators. In July 2018, CB4211 entered a Phase 1a/1b clinical trial which includes an evaluation of biological activity relevant to NASH and obesity. NASH is a chronic and silent disease in its early stages that can progress to more serious disease stages, such as advanced fibrosis, cirrhosis, liver failure or liver cancer. NASH has been estimated to affect as many as 12% of adults in the U.S., and there is no approved treatment for the disease.

On August 7, 2019 OPKO Health, Inc. (NASDAQ: OPK) reported business highlights and financial results for the three months ended June 30, 2019 (Press release, Opko Health, AUG 7, 2019, View Source [SID1234538365]).

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Business Highlights

RAYALDEE total prescriptions reported by IQVIA increased 92% in 2Q 2019 compared with 2Q 2018: Total prescriptions for the three months ended June 30, 2019 increased to approximately 12,700, compared with approximately 6,600 (as adjusted by IQVIA) during the comparable period of 2018.

4Kscore receives a new proposed local coverage determination from Novitas Solutions and utilization remained strong during 2Q 2019 with approximately 18,800 tests performed: Novitas Solutions, Inc. has issued a new proposed local coverage determination (LCD) for the 4Kscore test, with defined coverage criteria. Under the LCD, Medicare proposes reimbursing the test for patients who meet the defined criteria. The LCD is subject to a comment period ending August 11, 2019.

OPKO Plans to Refile 4Kscore De Novo Submission as PreMarket Approval Application (PMA): The Company plans to refile its 4Kscore test submission as a PMA application based on FDA feedback to the Company’s de novo request for this test, which has now been withdrawn. The FDA did not request any additional clinical data or any additional clinical trials in connection with a PMA submission. The Company currently anticipates submitting the PMA application in August 2019.

BioReference Laboratories formed a strategic collaboration with SOMOS, New York City’s largest multi-cultural physician led network: BioReference is now SOMOS’ preferred provider of diagnostic testing and will assist with data analytics for its patients. This collaboration is designed to streamline patient care and offer communication efficiencies between BioReference and the SOMOS network. SOMOS will leverage its relationship with BioReference to improve patient outcomes among its participants while decreasing the overall cost of care.

BioReference and its GeneDx subsidiary expand access to commercially insured lives: BioReference and GeneDx have been selected for inclusion in the UnitedHealthcare Preferred Lab Network beginning July 1, 2019. This was a comprehensive process that evaluated service

standards, turnaround time, quality and accreditation. More than 300 laboratories were invited to apply; only 100 submitted applications due to the complexity of the requirements, and BioReference and GeneDx, along with five other laboratories, earned a place in the Preferred Lab Network. In addition, effective April 1, 2019, BioReference and GeneDx are now in-network providers with Humana, which provides access to 11 million additional lives.

Pharmaceutical pipeline continues to progress. Last patient, last visit for our pivotal global Phase 3 clinical trial for hGH-CTP in growth hormone deficient children is expected this month. Topline data from the trial are expected to be announced by the end of 2019. The ongoing open-label phase 2 trial for RAYALDEE in hemodialysis patients is progressing well and initial data are expected by the end of 2019.

RAYALDEE Marketing Authorization Application filed by Vifor Fresenius in Switzerland. With several Marketing Authorization Applications already accepted and under review in other European countries, Vifor expects to receive approvals in several European countries to market RAYALDEE for the treatment of secondary hyperparathyroidism in adult non-dialysis patients with chronic kidney disease during 2020.

Financial Highlights

Consolidated revenues for the second quarter of 2019 were $226.4 million, compared with $263.7 million for the comparable period of 2018. During the 2019 quarter, revenue from services was $178.5 million, revenue from products was $28.7 million, including RAYALDEE net revenue of $5.7 million, and revenue from licensing and intellectual property was $19.2 million.

Operating expenses for the second quarter of 2019 were $273.6 million. This included continued investment in the company’s pharmaceutical pipeline, with R&D expense of $28.3 million, principally for pediatric trials with the hGH-CTP long-acting human growth hormone product.

The net loss for the three months ended June 30, 2019 was $59.8 million, compared with a net loss of $6.2 million for the comparable period of 2018 principally as a result in the decrease in revenue from services as wells as the comparable period of 2018 including a $15.4 million reversal of contingent consideration expense compared to $3.8 million for the 2019 period.

Cash, cash equivalents and marketable securities were $111.1 million as of June 30, 2019, which includes the repayment of a line of credit during the second quarter of approximately $40 million.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today. The conference call dial-in and webcast information is as follows:

DOMESTIC DIAL-IN: 866-634-2258
INTERNATIONAL DIAL-IN: 330-863-3454
PASSCODE: 7984069
WEBCAST: OPKO 2Q19 Results Conference Call

For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 7984069. The replay can be accessed for a period of time on OPKO’s website at OPKO 2Q19 Results Conference Call.

On August 7, 2019 G1 Therapeutics, Inc. (Nasdaq: GTHX), a clinical-stage oncology company, reported a corporate and financial update for the second quarter ended June 30, 2019 (Press release, G1 Therapeutics, AUG 7, 2019, View Source [SID1234538364]).

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"Our most advanced investigational therapy, trilaciclib, has demonstrated significant benefits for people being treated with chemotherapy for small cell lung cancer and triple-negative breast cancer. We are pleased that the FDA has granted Breakthrough Therapy Designation based on myelopreservation data in small cell lung cancer, an important step toward making trilaciclib available to these patients. We look forward to working with the FDA during our pre-NDA meeting next month. We have also initiated parallel discussions with the FDA regarding promising data in metastatic triple-negative breast cancer, which showed improved overall survival," said Mark Velleca, M.D., Ph.D., Chief Executive Officer. "In addition, we continue to make rapid progress across our pipeline, with emerging data suggesting that all three investigational therapies – trilaciclib, lerociclib and G1T48 – have the potential to improve outcomes for women with breast cancer and be used in early stages of their disease."

Raj Malik, M.D., Chief Medical Officer and Senior Vice President, R&D, added, "We will present new data on trilaciclib, lerociclib and G1T48 at the upcoming ESMO (Free ESMO Whitepaper) congress. Of note, we will report the first clinical data from approximately 25 patients in a Phase 1 trial of G1T48, our oral selective estrogen receptor degrader. Based on data from this trial, we are planning to initiate a pivotal trial in 2020 with G1T48 for the treatment of ER+, HER2- breast cancer in combination with a CDK4/6 inhibitor."

Clinical, Regulatory and Corporate Updates

Breakthrough Therapy Designation (BTD) granted for trilaciclib based on myelopreservation data in small cell lung cancer (SCLC) patients; U.S. and European regulatory filings on track for 2020: The company has received BTD from the U.S. Food and Drug Administration (FDA) based on positive myelopreservation data in small cell lung cancer patients from three randomized Phase 2 clinical trials. The BTD program is designed to expedite development and review of drugs intended for serious or life-threatening conditions. The company expects to submit marketing applications in the U.S. and Europe in 2020.

Preliminary overall survival (OS) results from randomized Phase 2 trial demonstrated women with metastatic triple-negative breast cancer (mTNBC) lived significantly longer when receiving trilaciclib and chemotherapy compared with women receiving chemotherapy alone: Myelopreservation results, objective response rate (ORR), progression-free survival (PFS) and safety data from this trial were presented at the 2018 San Antonio Breast Cancer Symposium (SABCS) (press release here). In June 2019, the company reported updated anti-tumor efficacy results that showed women receiving trilaciclib and a chemotherapy regimen of gemcitabine/carboplatin had a statistically significant improvement in OS compared with those receiving gemcitabine/carboplatin alone (press release here). Detailed data from this trial will be presented at a medical meeting later this year.

Data on all three clinical-stage programs accepted for presentation at ESMO (Free ESMO Whitepaper) 2019 Congress: New clinical data on trilaciclib, lerociclib and G1T48 have been accepted for presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress, being held Sept. 27-Oct. 1. Presentations include the first clinical data on G1T48, an oral selective estrogen receptor degrader (SERD), myelopreservation and efficacy data from the Phase 2 trilaciclib + chemotherapy + Tecentriq (atezolizumab) small cell lung cancer trial, and safety and tolerability data from the Phase 1b/2a lerociclib + Tagrisso (osimertinib) non-small cell lung cancer trial. The company will host a webcast on Sunday, Sept. 29 to review the data and provide an overview of development and commercial plans across the pipeline.

Additional data on trilaciclib reported at ASCO (Free ASCO Whitepaper) and MASCC/ISOO annual meetings: The company reported additional data from trilaciclib SCLC clinical trials at both the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the Multinational Association of Supportive Care in Cancer (MASCC)/International Society of Oral Oncology (ISOO) 2019 annual meetings. Pooled myelopreservation and patient-reported outcomes (PRO) data from all three trilaciclib SCLC trials presented at MASCC 2019 showed significant multilineage benefits across neutrophils, red blood cells and platelets, and significantly improved symptoms and function across multiple parameters over time compared to placebo.

Executive team update: In July, the company announced the appointment of Mark Avagliano as Chief Business Officer. Prior to joining G1, Mr. Avagliano was Vice President, Corporate Development at Pfizer Inc., where he was responsible for the evaluation, planning and execution of significant corporate level transactions and oversaw the Mergers and Acquisitions, Transactions and Valuations, and Out-licensing groups (press release here).

Board of Directors update: In June, current board member Garry Nicholson was named board chair, succeeding former chair Seth Rudnick, M.D. Additionally, Dr. Rudnick, Sir Andrew Witty and Fredric Eshelman, Pharm.D. were re-elected to the company’s Board of Directors.

Second Quarter 2019 Financial Highlights

Cash Position: Cash, cash equivalents and short-term investments totaled $324.9 million as of June 30, 2019, compared to $369.3 million as of December 31, 2018.

Operating Expenses: Operating expenses were $32.6 million for the second quarter of 2019, compared to $21.7 million for the second quarter of 2018. GAAP operating expenses include stock-based compensation expense of $3.7 million for the second quarter of 2019, compared to $2.1 million for the second quarter of 2018.

Research and Development Expenses: Research and development (R&D) expenses for the second quarter of 2019 were $23.5 million, compared to $18.4 million for the second quarter of 2018. The increase in R&D expense was primarily due to an increase in clinical program costs and personnel costs due to additional headcount.

General and Administrative Expenses: General and administrative (G&A) expenses for the second quarter of 2019 were $9.1 million, compared to $3.3 million for the second quarter of 2018. The increase in G&A expense was largely due to an increase in compensation due to additional headcount, increase in pre-commercialization activities, and an increase in professional fees and other administrative costs necessary to support our operations as a public company.

Net Loss: G1 reported a net loss of $30.7 million for the second quarter of 2019, compared to $20.9 million for the second quarter of 2018.

2019 Guidance: the company expects to end the year with $260-$270 million in cash and cash equivalents.

Anticipated Milestones for 2H 2019

Present new clinical results for trilaciclib, lerociclib and G1T48 at the ESMO (Free ESMO Whitepaper) 2019 Congress, being held Sept. 27-Oct. 1. The company will host an onsite event/webcast on Sunday, Sept. 29 to review the data.

Complete meetings with the FDA and provide regulatory update for trilaciclib, including NDA filing timeline.

Present preliminary OS findings from trilaciclib mTNBC trial at a medical meeting in 2H19

Present additional data from the Phase 1b/2a clinical trial of lerociclib + Faslodex (fulvestrant) in ER+, HER2- breast cancer in 4Q19.

In 4Q19, identify dose and schedule of lerociclib and G1T48 for pivotal trials in breast cancer in 2020.

Webcast and Conference Call

The management team will host a webcast and conference call at 4:30 p.m. ET today to provide a corporate and financial update for the second quarter 2019 ended June 30, 2019. The live call may be accessed by dialing 866-763-6020 (domestic) or 210-874-7713 (international) and entering the conference code: 7989125. 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.

On August 7, 2019 Sierra Oncology, Inc. (SRRA), a late-stage drug development company focused on advancing targeted therapeutics for the treatment of patients with significant unmet needs in hematology and oncology, reported that Dr. Nick Glover, President and Chief Executive Officer, will present an overview of the company at 2:30 pm ET on Wednesday, August 14 at the Wedbush PacGrow Healthcare Conference being held in New York (Press release, Sierra Oncology, AUG 7, 2019, View Source [SID1234538351]).

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A live audio webcast and archive of the presentation will be accessible through the Sierra Oncology website at www.sierraoncology.com.

On August 7, 2019 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the second quarter and year-to-date 2019 and recent business highlights (Press release, Ionis Pharmaceuticals, AUG 7, 2019, View Source [SID1234538350]).

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"We enter the second half of 2019 in a position of substantial financial strength driven by revenue growth of more than 75 percent. SPINRAZA’s blockbuster performance, with over $1 billion in net sales in the first half, contributed significantly to our strong financial results. There are approximately 8,400 patients on SPINRAZA, an increase of approximately 12 percent compared to last quarter. Product sales from TEGSEDI’s second full quarter on the market also contributed to our strong first half results. Additionally, WAYLIVRA is on track to launch in Europe this quarter. With three medicines commercialized in the last three years, an advancing technology and a pipeline of over 40 medicines, we are positioned to continue delivering value to patients and shareholders through this year and beyond," said Stanley T. Crooke, M.D., Ph.D., chairman of the board and chief executive officer of Ionis.

"Our strong year-to-date financial results were driven by continued revenue growth across both commercial and R&D revenues. These results put us on track to meet or potentially improve upon our 2019 guidance. We are also on track to achieve our fourth consecutive year of operating income and our third consecutive year of net income, both on a non-GAAP basis. We believe our ability to be profitable while investing in commercial activities, fully exploiting our pipeline and advancing our technology, clearly sets us apart from our peers," said Elizabeth L. Hougen, chief financial officer of Ionis.

Year-to-Date 2019 Financial Results and Highlights

Year-to-date revenues increased more than 75 percent
Commercial revenue from SPINRAZA (nusinersen) royalties increased more than 30 percent to $130 million for the first half of 2019 compared to 2018.
TEGSEDI (inotersen) product sales were $17 million in the first half of 2019, reflecting a more than 40 percent increase in the second quarter compared to the first quarter.
R&D revenue nearly doubled in the first half of 2019 compared to 2018.
Significant net income for the first half of 2019 puts Ionis on track for its third consecutive year of net income
Operating income and net income significantly improved to $103 million and $84 million, respectively, in the first half of 2019 compared to 2018.
Non-GAAP operating income increased by more than 20-fold in the first half of 2019 compared to 2018.
Non-GAAP net income increased by approximately 10-fold in the first half of 2019 compared to 2018.
Maintained substantial cash position of $2.3 billion while aggressively investing broadly across Ionis’ business
All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of non-GAAP and GAAP measures, which is provided later in this release.

"In addition to our commercial medicines, we have a broad and advancing pipeline of potentially transformational medicines we are moving toward the market. In our late-stage pipeline, our medicines targeting Huntington’s disease and SOD1-ALS are progressing in Phase 3 studies, both with the potential to support registration. Our LICA medicines targeting Lp(a)-driven cardiovascular disease and TTR amyloidosis are on track to initiate Phase 3 programs this year. Positive results from our HBV program, our advancing FXI program, which is nearing completion, and the initiation of multiple Phase 2 studies demonstrate the potential value in our mid-stage pipeline. All of these potentially transformational medicines demonstrate the ability of our technology to address a broad range of diseases from rare diseases to those affecting millions of patients," said Brett P. Monia, chief operating officer at Ionis.

Recent Business Highlights

SPINRAZA – a worldwide foundation-of-care for the treatment of patients of all ages with spinal muscular atrophy (SMA)
Worldwide sales of SPINRAZA increased by nearly 30 percent to $1 billion in the first half of 2019 compared to 2018.
Patients on SPINRAZA treatment increased by approximately 12 percent, compared to last quarter, to approximately 8,400 patients across global commercial, clinical and expanded access settings.
Biogen believes the global SMA opportunity to be significantly greater than previous estimates, with over 45,000 patients in markets where Biogen has a direct presence.
TEGSEDI – launch underway in multiple markets for the treatment of polyneuropathy of hereditary transthyretin amyloidosis (hATTR) in adult patients
Product sales were $17 million in the first half of 2019 and $10 million in the second quarter of 2019.
On track to launch in England this month following a positive reimbursement recommendation from NICE.
Launch in additional EU countries planned; launch in Latin America planned with PTC Therapeutics.
WAYLIVRA (volanesorsen) – the only medicine approved in the EU for the treatment of adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis
On track to launch in the EU this month, beginning in Germany, with additional EU country launches planned in 2020.
Encouraging discussions continued with the U.S. Food and Drug Administration to clarify a path forward in the U.S.
Received $6 million milestone payment from PTC Therapeutics for the EU approval of WAYLIVRA in the second quarter.
Achieved the primary endpoint of a statistically significant reduction in triglyceride levels and an important secondary endpoint of a statistically significant reduction in liver fat with good safety and tolerability in patients with familial partial lipodystrophy (FPL) in the BROADEN study.
Ionis achieved positive results in its program to treat patients with hepatitis B viral infection as reported by GSK.
Ionis initiated a Phase 2 study of IONIS-FB-LRx in patients with geographic atrophy secondary to age-related macular degeneration.
Ionis initiated a Phase 2 study of IONIS-PKK-LRx in patients with hereditary angioedema.
Ionis appointed Joan E. Herman, MBA, MS, president and chief executive officer of Herman and Associates, LLC to the Ionis board of directors.
Key Upcoming Events

Ionis and Akcea plan to initiate the Phase 3 program for AKCEA-TTR-LRx in patients with TTR amyloidosis before the end of 2019.
Novartis plans to initiate the Phase 3 HORIZON cardiovascular outcomes study of AKCEA-APO(a)-LRx (TQJ230) in patients with elevated Lp(a)-driven cardiovascular disease before the end of 2019.
Ionis and Akcea plan to present data from the Phase 1/2 study of AKCEA-TTR-LRx in healthy volunteers in September 2019 at the European ATTR Amyloidosis meeting and at the Heart Failure Society of America.
Roche plans to present data from the open label extension portion of the Phase 1/2 study of IONIS-HTTRx (RG6042) in patients with Huntington’s disease.
Ionis plans to initiate a Phase 2 study of IONIS-FB-LRx in a second indication under Ionis’ collaboration with Roche to develop medicines for the treatment of complement-mediated diseases.
Ionis and its partners plan to report data from the HBV and FXI clinical programs at future medical conferences.
Ionis and Akcea plan to report top line results from Phase 2 studies of AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx in 1H 2020.
Revenue

Ionis’ revenue in the three and six months ended June 30, 2019 was $164 million and $461 million, respectively, compared to $118 million and $262 million for the same periods in 2018 and was comprised of the following (amounts in millions):

For the first half of 2019, Ionis significantly increased both commercial revenue and R&D revenue compared to the same period in 2018. Commercial revenue from SPINPRAZA royalties increased over 30 percent primarily due to increased SPINRAZA product sales. TEGSEDI product sales increased more than 40 percent over the first quarter of this year and were another important source of commercial revenue for Ionis.

Ionis’ R&D revenue substantially increased in the first half of 2019 compared to the same period in 2018 primarily due to the following:

$150 million the Company earned from Novartis when Novartis licensed AKCEA-APO(a)-LRx;
$35 million the Company earned from Roche when Roche enrolled the first patient in the Phase 3 study of IONIS-HTTRx in patients with Huntington’s disease; and
$20 million the Company earned from Alnylam when Alnylam licensed Ionis’ technology to Regeneron.
Operating Expenses

Operating expenses for the three and six months ended June 30, 2019 on a GAAP basis were $183 million and $358 million, respectively, and on a non-GAAP basis were $141 million and $271 million, respectively. These amounts compare to GAAP operating expenses for the three and six months ended June 30, 2018 of $168 million and $316 million, respectively, and non-GAAP operating expenses of $134 million and $253 million, respectively. The increase in operating expenses was principally due to Ionis’ investment in the global launch of TEGSEDI and preparing to launch WAYLIVRA in the EU.

Income Tax Expense

Ionis recorded an income tax benefit of $7 million for the three months ended June 30, 2019 and income tax expense of $24 million for the six months ended June 30, 2019. Ionis’ income tax expense in the first half of this year was primarily due to Ionis’ expectation that it will generate U.S. federal and state taxable income in 2019.

Net Loss Attributable to Noncontrolling Interest in Akcea

At June 30, 2019, Ionis owned approximately 76 percent of Akcea. The shares of Akcea third parties own represent an interest in Akcea’s equity that Ionis does not control. However, because Ionis continues to maintain overall control of Akcea through its voting interest, Ionis reflects the assets, liabilities and results of operations of Akcea in Ionis’ consolidated financial statements. Ionis reflects the noncontrolling interest attributable to other owners of Akcea’s common stock in a separate line called "Net loss attributable to noncontrolling interest in Akcea" on Ionis’ statement of operations. Ionis’ net loss attributable to noncontrolling interest in Akcea for the three and six months ended June 30, 2019 was $9 million and $3 million, respectively, compared to $16 million and $25 million for the three and six months ended June 30, 2018, respectively.

Net Income (Loss) Attributable to Ionis Common Stockholders

On a GAAP basis, Ionis reported net loss and net income attributable to Ionis’ common stockholders of $1 million and $84 million for the three and six months ended June 30, 2019, respectively, compared to a net loss of $40 million and $42 million for the same periods in 2018. On a non-GAAP basis, Ionis reported net income attributable to Ionis’ common stockholders of $38 million for the three months ended June 30, 2019, compared to a net loss attributable to Ionis’ common stockholders of $10 million for the same period in 2018. For the six months ended June 30, 2019, Ionis reported net income attributable to Ionis’ common stockholders of $162 million, compared to $15 million for the same period in 2018, all on a non-GAAP basis. The increase was primarily due to increases in revenue.

For the three months ended June 30, 2019, basic and diluted net loss per share were $0.01, compared to $0.29 for the same period in 2018. For the six months ended June 30, 2019, basic and diluted net income per share were $0.62 and $0.61, respectively, compared to basic and diluted net loss per share of $0.30 for the same period in 2018. All amounts are on a GAAP basis.

Balance Sheet

Ionis maintained its strong balance sheet, ending the second quarter of 2019 with cash, cash equivalents and short-term investments of $2.3 billion, compared to $2.1 billion at December 31, 2018.

Webcast and Conference Call

Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast conference call to discuss this earnings release and related activities. Interested parties may listen to the call by dialing 877-443-5662 or access the webcast at www.ionispharma.com. A webcast replay will be available for a limited time.