On May 9, 2019 BioTime, Inc. (NYSE American and TASE: BTX), a clinical-stage biotechnology company developing cellular therapies for unmet medical needs, reported financial and operating results for the first quarter ended March 31, 2019 (Press release, BioTime, MAY 9, 2019, View Source;p=RssLanding&cat=news&id=2398106 [SID1234536039]). BioTime management will host a conference call and webcast today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its first quarter 2019 financial results and to provide a business update.

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"We have been transforming BioTime into what we believe is one of the foremost cell therapy companies, with a pipeline which now consists of three innovative and promising clinical-stage product candidates, each with the potential to significantly and positively impact serious diseases or degenerative conditions," stated Brian M. Culley, Chief Executive Officer of BioTime. "We will remain focused on progressing our clinical programs in a thoughtful and cost-effective manner throughout 2019. As of March 31, 2019, the value of our cash, marketable securities, equity positions in our affiliate companies, and the balance of the promissory note due to us in August 2020 was more than $100 million, and we believe we are well-positioned to advance our programs. Our goal is to build awareness and support for our reinvigorated and repositioned company with the investment, medical, and patient communities, and advance our objective of bringing cell therapies to patients who can most benefit from their extraordinary potential."

Recent Highlights

Presented positive results from the Company’s ongoing Phase I/IIa clinical study of OpRegen for the treatment of dry-age-related macular degeneration (AMD) with geographic atrophy (GA), at the 2019 Association for Research in Vision and Ophthalmology Annual Meeting. Data from the study demonstrate that treatment with OpRegen continues to be well tolerated and in some patients, signs of structural improvement in the treated areas of the retina have been observed. Of note, early data from Cohort 4 patients with earlier-stage dry-AMD and smaller areas of GA remain encouraging, with indications of the continued presence of the transplanted OpRegen cells and improvements in visual acuity.
Presented SCiStar Clinical Study Top-Line Results at the 26th Annual American Society for Neural Therapy and Repair Annual Conference. The primary goals of the SCiStar Clinical Study, which were to observe the safety of OPC1 in cervical spinal cord injury patients and other important metrics related to the optimal timing of OPC1 injection, the tolerability of the immunosuppression regimen, the engraftment of OPC1 cells, and rates of motor recovery observed among different study subpopulations, were all achieved.
Announced the issuance of a patent from the United States Patent and Trademark Office for a method of reducing spinal cord injury (SCI)-induced parenchymal cavitation in patients who suffered an acute SCI. The issued patent would have a term that expires no earlier than 2036.
Announced exclusive agreement with Orbit Biomedical Ltd. (Orbit) under which BioTime and Orbit will collaborate on the use of Orbit’s proprietary U.S. Food and Drug Administration 510(k) approved injection technology to enhance the sub-retinal delivery of OpRegen RPE cells for the treatment of dry-AMD in BioTime’s ongoing Phase I/IIa clinical study.
The ongoing transfer of assets acquired in the Asterias merger to BioTime’s existing GMP manufacturing facility in Jerusalem in preparation for the hand off of Asterias’s Fremont facility to Novo Nordisk in the third quarter of 2019. These actions are expected to lead to significant cost savings via headcount and facility reductions, as well as support BioTime’s innovative and diversified clinical-stage pipeline.
BioTime affiliate OncoCyte Corporation (NYSE American: OCX) recently reported successful completion of its Analytical Validation study and the commencement of a CLIA Validation study of DetermaVu, its non-invasive liquid biopsy test intended to facilitate clinical decision making in lung cancer diagnosis. BioTime owns approximately 28% of OncoCyte’s common stock, or 14.7 million shares, as of May 8, 2019. As of that same date, the value of BioTime’s OncoCyte share position was approximately $65.7 million, based on the closing price of OncoCyte’s common stock on that date.
Plans for 2019

Pursuant to an exclusive collaboration with Orbit, initiate dosing of the first patient with the Orbit device and a new thaw and inject formulation in the ongoing Phase I/IIa clinical study of OpRegen for the treatment of dry-AMD, anticipated in the second quarter of 2019.
Announce decision on BioTime’s CE Mark application for Renevia, an investigational medical device being developed as an alternative for whole adipose tissue transfer procedures, now expected in the second quarter of 2019.
Continue advancement of the OPC1 program and meet with the U.S. Food and Drug Administration (FDA) to discuss plans for next steps in the clinical development of the program, anticipated by year end 2019.
Strengthen and expand existing partnerships with the California Institute for Regenerative Medicine and Cancer Research UK for the ongoing support of the development of the OPC1 and VAC2 programs.
Complete patient enrollment in the ongoing Phase I/IIa clinical study of OpRegen for the treatment of dry-AMD, anticipated by year end 2019.
Evaluate the development of OPC1 as a candidate for the potential treatment of multiple sclerosis (MS) and ischemic stroke through ongoing research collaborations with major universities.
Increase presence and engagement within the patient, physician, and advocacy communities.
Balance Sheet Highlights

Cash, cash equivalents and marketable securities totaled $27.1 million as of March 31, 2019.

BioTime’s investment in OncoCyte was valued at $58.0 million as of March 31, 2019 and at $65.7 million as of May 8, 2019, under the equity method of accounting, and based on the closing stock price of OncoCyte as of such dates.

Intangible assets, net increased during the first quarter of 2019 due to the Asterias merger and the acquisition of OPC1 (fair value of $31.7 million) and VAC2 (fair value of $14.8 million).

BioTime’s promissory note due from Juvenescence Limited had an outstanding balance (principal plus accrued interest) of $22.5 million as of March 31, 2019. Unless earlier converted into Juvenescence common shares, the promissory note is payable in cash, plus accrued interest at 7% per year, at maturity in August 2020. If Juvenescence completes an initial public offering (IPO) resulting in gross proceeds of not less than $50.0 million, the promissory note automatically converts into the Juvenescence securities issued in the IPO based on the per-share price to the public in the IPO, subject to an upward adjustment in the number of shares that would be issued to BioTime upon such conversion if the 20-day volume-weighted average trading price of one share of common stock of AgeX Therapeutics, Inc. (AgeX) before the IPO is priced above $3.00. If the promissory note is converted, the Juvenescence ordinary shares will be a marketable security that BioTime may use to supplement its liquidity, as needed and as market conditions allow.

First Quarter Operating Results

Revenues: BioTime’s revenue is generated primarily from research grants, licensing fees and royalties. Total revenues for the three months ended March 31, 2019 were $0.9 million, an increase of $0.2 million, compared to $0.7 million for the same period in 2018. The increase was primarily related to a $0.4 million increase in grant revenues, offset by a $0.2 million decrease in subscriptions and advertisement revenues attributable to the deconsolidation of AgeX. AgeX was deconsolidated from BioTime on August 30, 2018, and beginning on that date, AgeX’s revenues are not included in BioTime revenues.

Operating Expenses: Operating expenses are comprised of research and development ("R&D") expenses and general and administrative ("G&A") expenses. Total operating expenses for the three months ended March 31, 2019 were $13.6 million, as reported, and $7.9 million, as adjusted. AgeX was deconsolidated from BioTime on August 30, 2018, and beginning on that date, AgeX’s operating expenses are not included in BioTime’s operating expenses.

As adjusted operating expenses is a non-generally accepted accounting principles (non-GAAP) financial measure. The reconciliation between operating expenses determined in accordance with GAAP and non-GAAP operating expenses, by entity, is provided in the financial tables included at the end of this press release.

R&D Expenses: Beginning on August 30, 2018, BioTime ceased recognizing R&D expenses related to AgeX and its programs due to the AgeX deconsolidation on that date.

R&D expenses for the three months ended March 31, 2019 were $5.0 million, a decrease of $0.9 million, compared to $5.9 million for the same period in 2018. The decrease was primarily related to a $1.6 million decrease from the AgeX deconsolidation and the absence of AgeX R&D expenses incurred after August 30, 2018, offset by a net increase of $0.6 million in BioTime programs primarily related to: (1) an increase of $0.8 million in OpRegen related expenses, (2) an increase of $0.6 million in OPC1 and VAC2 expenses (these programs were acquired in the Asterias merger) offset by (3) decreases of $0.8 million in Renevia and HyStem related expenses.

G&A Expenses: Beginning on August 30, 2018, BioTime ceased recognizing G&A expenses related to AgeX and its subsidiaries due to the AgeX deconsolidation on that date.

G&A expenses for the three months ended March 31, 2019 were $8.7 million, an increase of $2.7 million, compared to $6.0 million for the same period in 2018. The increase was primarily attributable to a $3.5 million increase in severance, legal, accounting and other expenses related to the Asterias merger and a $0.5 million increase in stock-based compensation, offset by a $1.3 million decrease in AgeX related G&A expenses.

Other Income/(Expenses), Net: Other income/(expenses), net for the three months ended March 31, 2019 reflected other income, net of $47.7 million, compared to other expense, net of ($51.5) million for the same period in 2018. The variance was primarily related to changes in the value of equity investments in OncoCyte and Asterias for the applicable periods.

Net income/(loss) attributable to BioTime: The net income/(loss) attributable to BioTime for the three months ended March 31, 2019 was net income of $39.3 million, or $0.30 per share (basic and diluted), compared to a net loss attributable to BioTime of ($63.5) million, or ($0.50) per share (basic and diluted), for the same period in 2018.

Conference Call and Webcast

BioTime will host a conference call and webcast today, at 1:30pm PT/4:30pm ET to discuss its first quarter 2019 financial results and to provide a business update. Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from outside the U.S. and Canada and should request the "BioTime Inc. Call". A live webcast of the conference call will be available online in the Investors section of BioTime’s website. A replay of the webcast will be available on BioTime’s website for 30 days and a telephone replay will be available through May 16th, 2019, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from outside the U.S. and Canada and entering conference ID number 9155549.

On May 9, 2019 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported its financial results for the quarter ended March 31, 2019 (Press release, Corcept Therapeutics, MAY 9, 2019, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-first-quarter-2019-financial [SID1234536056]).

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Financial Highlights

Revenue of $64.8 million, a 12 percent increase from first quarter 2018
Fully-diluted GAAP net income of $0.15 per share, compared to $0.14 per share in first quarter 2018
Fully-diluted non-GAAP net income of $0.20 per share, compared to $0.19 per share in first quarter 2018
Cash and investments of $215.7 million, a $9.0 million increase from year-end 2018
Reaffirmed 2019 revenue guidance of $285 – $315 million
Corcept reported quarterly revenue of $64.8 million, compared to $57.7 million in the first quarter of 2018. First quarter GAAP net income was $18.3 million, compared to $17.5 million in the same period last year. Excluding non-cash expenses related to stock-based compensation, utilization of deferred tax assets and related income tax effects, non-GAAP net income in the first quarter was $24.3 million, compared to $24.5 million in the first quarter of 2018. A reconciliation of GAAP to non-GAAP net income is included below.

First quarter operating expenses were $45.9 million, compared to $36.7 million in the first quarter of 2018, primarily due to an increase in research and development personnel and increased spending to advance new compounds towards the clinic and to formulate and manufacture relacorilant, CORT118335 and CORT125281.

Cash and investments were $215.7 million at March 31, 2019, an increase of $9.0 million from year-end. This increase was after the expenditure of $13.6 million in the first quarter to repurchase 1.2 million shares of common stock. At March 31, 2019, $62.8 million remained available under Corcept’s stock repurchase program.

The company reaffirmed 2019 revenue guidance of $285 – $315 million.

"As in prior years, our first quarter revenue was reduced as payors forced patients to secure reauthorization of their insurance," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "Our first quarter revenue was also reduced by the requirement that we cover a portion of the ‘donut hole’ in Medicare Part D insurance plans – a portion that, by statute, increased significantly this year. No patient’s care was interrupted, because we provide Korlym at no cost until coverage is restored. The number of patients receiving Korlym continues to grow.

"Awareness of Cushing’s syndrome and the importance of screening more widely for the disorder is increasing," Dr. Belanoff added. "Our goal is to educate physicians about the benefits of cortisol modulation with Korlym and, should it continue to generate positive clinical data and we secure FDA approval, relacorilant, Korlym’s planned successor. We believe relacorilant has the potential to offer significant benefits – even beyond what we see with Korlym."

Clinical Highlights

Dosing continues in Phase 3 trial of relacorilant in patients with Cushing’s syndrome ("GRACE")
European Medicines Agency ("EMA") recommends orphan designation for relacorilant to treat patients with Cushing’s syndrome
Dosing initiated in double-blind, placebo-controlled, Phase 1b trial of CORT118335 for prevention of antipsychotic-induced weight gain; Phase 2 trials in reversal of antipsychotic-induced weight gain planned in second half of 2019
Placebo-controlled, Phase 2 trial of CORT118335 to treat non-alcoholic steatohepatitis ("NASH") planned in second half of 2019
Data from Phase 1/2 trial of relacorilant plus Abraxane in solid tumors to be presented at 2019 ASCO (Free ASCO Whitepaper) Meeting, May 31 – June 4
Dosing continues in controlled, Phase 2 trial of relacorilant plus Abraxane in metastatic ovarian cancer
Dosing continues in Phase 1/2 trial of CORT125281 plus Xtandi in castration-resistant prostate cancer
"It is an exciting time to join Corcept," said Andreas Grauer, MD, who became Corcept’s Chief Medical Officer in March 2019. "Three of our proprietary, selective cortisol modulators have entered the clinic and we are conducting five clinical trials – in Cushing’s syndrome, solid tumors and antipsychotic-induced weight gain. Additional trials – in antipsychotic-induced weight gain and NASH – are planned for later in the year."

"Relacorilant’s GRACE trial continues to dose patients with Cushing’s syndrome and has begun opening sites in Europe, where many of our Phase 2 patients were enrolled. The EMA’s recommendation that relacorilant receive orphan drug designation is especially gratifying. Unlike in the United States, orphan designation in Europe requires the authorities to find plausible evidence of a drug’s efficacy and potential to confer significant clinical benefit compared to already-approved treatments – findings we hope GRACE will confirm for relacorilant." (For more about GRACE, go to cushingresearch.com.)

Corcept presented data from relacorilant’s Phase 2 trial at the 2019 annual meeting of the American Association of Clinical Endocrinologists ("AACE") in April. Subjects in the trial exhibited clinically meaningful improvements in the trial’s key endpoints – hypertension and glucose control – and in a variety of secondary endpoints, including weight loss, liver function, coagulopathy, insulin resistance, cognitive function, mood and quality of life. Relacorilant was well-tolerated, with the most common adverse events being those associated with a reduction in excess cortisol activity. These side effects usually resolve with continued treatment. There were no instances of abnormal vaginal bleeding, even in women who had previously experienced abnormal vaginal bleeding with Korlym. Importantly, there were no instances of hypokalemia, an adverse event experienced by 44 percent of patients in Korlym’s pivotal trial and a leading cause of Korlym discontinuation. (Corcept’s AACE presentation is available at ir.corcept.com/events.)

"Our oncology program is also progressing," said Dr. Grauer. "Enrollment has begun in our 180-patient, controlled, Phase 2 trial of relacorilant plus Abraxane in patients with metastatic ovarian cancer. At the ASCO (Free ASCO Whitepaper) congress this June, we will present data from our Phase 1/2 trial of relacorilant plus Abraxane in a variety of solid tumors and discuss our plans in metastatic pancreatic cancer."

"Finally, our most promising compound for metabolic disorders, CORT118335, has entered the clinic. We are dosing healthy subjects in a placebo-controlled Phase 1b trial in the prevention of antipsychotic-induced weight gain and plan to start two placebo-controlled, Phase 2 trials in patients later this year, as well as a placebo-controlled, Phase 2 trial in patients with NASH – a precursor of cirrhosis. These disorders afflict millions of patients and there are no good treatment options."

Conference Call
We will hold a conference call on May 9, 2019, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, dial 1-800-347-6311 from the United States or 1-323-994-2132 internationally approximately ten minutes before the start of the call. The passcode will be 4773625. A replay will be available through May 23, 2019 at 888-203-1112 in the United States and 719-457-0820 internationally. The passcode will be 4773625.

Hypercortisolism
Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients being diagnosed each year. Symptoms vary, but most people experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system in the body and can be lethal if not treated effectively.

On May 9, 2019 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the first quarter of 2019 and recent business highlights (Press release, Ionis Pharmaceuticals, MAY 9, 2019, View Source [SID1234536073]).

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"Our strong first quarter results put us on track to achieve our 2019 goals. We added commercial revenue from the first full quarter of the TEGSEDI launch to our growing commercial revenue from SPINRAZA. WAYLIVRA is now our third antisense medicine approved in just over two years, and we look forward to launching in Europe next quarter through our affiliate, Akcea," said Stanley T. Crooke, M.D., Ph.D., chairman of the board and chief executive officer of Ionis. "This week, data presented from our medicines targeting Huntington’s disease and SOD1-ALS once again demonstrate the potential for our antisense technology to provide benefit in disease measures for patients with serious and previously untreatable diseases. Both medicines are in Phase 3 clinical trials with potential to support rapid paths to patients. Novartis licensed our most advanced LICA medicine, AKCEA-APO(a)-LRx, targeting the millions of patients worldwide with Lp(a)-driven cardiovascular disease. Novartis’ decision to advance AKCEA-APO(a)-LRx into a Phase 3 cardiovascular outcomes study further validates the potential of our rapidly expanding LICA pipeline to treat a broad range of diseases, including those for large patient populations. We plan to advance our next LICA medicine, AKCEA-TTR-LRx targeting TTR amyloidosis, into Phase 3 development in the second half of this year. We believe our accomplishments in the first quarter of 2019 position us for continued success. The power of our efficient technology and business strategy give us confidence that we can continue to deliver sustainable financial growth while aggressively investing in our commercial medicines and advancing our pipeline and our technology."

First Quarter 2019 Financial Results and Highlights

Revenues more than doubled compared to Q1 2018

Total revenue was $297 million compared to $144 million in Q1 2018.

Commercial revenue from SPINRAZA (nusinersen) was $60 million compared to $41 million in Q1 2018.

TEGSEDI (inotersen) product sales were $7 million in its first full quarter on the market and $9 million since launching in Q4 2018.

R&D revenue included $150 million from Novartis for its license of AKCEA-APO(a)-LRx and $35 million from Roche when it enrolled the first patient in the Phase 3 study of IONIS-HTTRx (RG6042) in patients with Huntington’s disease.

Achieved substantial operating income and net income

Operating income and net income were $121 million and $84 million, respectively, compared to an operating loss and net loss of $3 million and $1 million, respectively, in Q1 2018, all on a GAAP basis.

Non-GAAP operating income and net income were $167 million and $126 million, respectively, compared to $25 million for both non-GAAP operating income and net income in Q1 2018.

Operating expenses increased in the first quarter primarily due to Ionis’ investment in commercializing TEGSEDI.

Substantial cash position grew to $2.3 billion enabling aggressive investment broadly across Ionis’ business

"We achieved another quarter of strong financial performance with both operating income and net income in the first quarter of 2019, substantially outperforming the same quarter in 2018. Our revenues in the first quarter were composed of growing commercial revenues from SPINRAZA royalties and TEGSEDI product sales, on top of substantial R&D revenues, driven in large part by the one-time $150 million license fee from Novartis for AKCEA-APO(a)-LRx. Looking ahead, we expect growing revenues this year from SPINRAZA, TEGSEDI and our partnered programs. And we also look forward to adding revenue following the EU launch of WAYLIVRA," said Elizabeth L. Hougen, chief financial officer of Ionis. "We are on track to achieve our 2019 financial guidance of net income and more than $100 million in operating income, both on a non-GAAP basis. Our goal is to continue to be profitable while investing in our commercial products, our pipeline and our technology."

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.

Recent Business Highlights

SPINRAZA – the worldwide standard-of-care for the treatment of people with all forms of spinal muscular atrophy

Biogen reported worldwide sales of SPINRAZA of $518 million in the first quarter of 2019, a 42 percent increase compared to Q1 2018, driven primarily by increased penetration in existing markets, new country launches and continued uptake in the U.S. by children and adult patients.

There were more than 7,500 SMA patients from over 40 countries on SPINRAZA treatment at the end of the first quarter of 2019, including commercial patients and patients in the expanded access program and clinical trials.

SPINRAZA data from the ongoing NURTURE and SHINE open-label extension studies demonstrated continued durable efficacy and reinforced the safety profile of SPINRAZA in patients treated for up to 6 years, as presented by Biogen at the 2019 AAN Annual Meeting.

TEGSEDI – launch underway in multiple markets for the treatment of polyneuropathy of hereditary transthyretin amyloidosis (hATTR) in adult patients

TEGSEDI product sales were $7 million in its first full quarter on the market and $9 million since launching in Q4 2018.

TEGSEDI received a positive Final Evaluation Document (FED) from the National Institute for Health and Care Excellence (NICE) authorizing reimbursement for the treatment of patients with polyneuropathy due to hATTR amyloidosis in England.

Data presented at AAN from the TEGSEDI NEURO-TTR open-label extension study demonstrated long-term efficacy and safety in patients with hATTR.

WAYLIVRA (volanesorsen) – approved in the EU for the treatment of adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis

Akcea’s preparations to launch in the EU are underway, beginning in Germany in Q3 2019.

Launch in additional EU countries is planned in 2020.

Earned a $6 million milestone payment from PTC Therapeutics for the EU approval of WAYLIVRA.

Roche presented nine-month data from the ongoing Phase 1/2 open-label extension study of IONIS-HTTRx (RG6042) in patients with Huntington’s disease at AAN, demonstrating continued and sustained reductions in mutant huntingtin protein with bi-monthly dosing.

Based on these data, Roche amended the dosing regimen in the Phase 3 study of IONIS-HTTRx (RG6042) in patients with Huntington’s disease to replace the monthly dosing regimen with a tri-annual (every four months) dosing regimen.

Biogen presented data from the Phase 1/2 study of tofersen (IONIS-SOD1Rx) in ALS patients with SOD-1 mutations (SOD1-ALS) at AAN, demonstrating benefit in clinical measures of ALS disease progression after three months of treatment.

Tofersen is in a Phase 3 clinical study that could support a rapid path to patients.

Biogen is collaborating with regulators to further define the scope of the clinical data package required to support registration.

Ionis generated a $7.5 million milestone payment for advancing a new target for an unidentified neurological disease under its 2018 strategic neurology collaboration with Biogen.

Brett P. Monia, Ph.D., chief operating officer of Ionis was appointed to the Ionis board of directors.

Key Upcoming Data Events

Open-label extension study of IONIS-HTTRx (RG6042) in patients with Huntington’s disease

Phase 1/2 study of AKCEA-TTR-LRx in healthy volunteers

BROADEN study of WAYLIVRA in patients with familial partial lipodystrophy (FPL)

Development program targeting FXI for the treatment of patients with clotting disorders

Development program for the treatment of patients with HBV infection

Phase 2 study of IONIS-GHR-LRx in patients with acromegaly

Phase 1 study of IONIS-ENAC-2.5Rx in healthy volunteers

In the first quarter of 2019, Ionis significantly increased both commercial revenue and R&D revenue. Commercial revenue from SPINRAZA royalties increased more than 45 percent. The Company also added growing TEGSEDI product sales to its commercial revenue.

Ionis’ R&D revenue substantially increased in the first quarter of 2019 due to the $150 million and $35 million the Company earned from Novartis and Roche, respectively.

In the second quarter of 2019, Alnylam announced it licensed Ionis’ technology to Regeneron. Once the transaction closes, Ionis expects to earn $20 million in sublicensing revenue.

Operating Expenses

Operating expenses for the first quarter of 2019 on a GAAP basis were $176 million and on a non-GAAP basis were $130 million. These amounts compare to GAAP operating expenses for the first quarter of 2018 of $147 million and non-GAAP operating expenses of $119 million. The increase in operating expenses was principally due to Ionis’ investments in the global launch of TEGSEDI.

Income Tax Expense

Ionis recorded income tax expense of $31 million for the three months ended March 31, 2019, compared to $15,000 for the same period in 2018. The increase in its income tax expense was primarily due to Ionis’ expectation that it will generate U.S. federal and state taxable income in 2019. Ionis’ 2019 income tax expense has two components. The first component relates to federal income taxes. Ionis expects to utilize its deferred tax assets to offset its U.S. federal taxable income. The other component of Ionis’ income tax expense relates to the estimated cash taxes it will pay for its state income taxes. Although Ionis is recording the expense for its state income taxes in 2019, Ionis will not have to make the majority of the payment for this liability until the first quarter of 2020.

Net (Income) Loss Attributable to Noncontrolling Interest in Akcea

At March 31, 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 income attributable to noncontrolling interest in Akcea for the first quarter of 2019 was $6 million, compared to a net loss attributable to noncontrolling interest in Akcea of $9 million for the same period in 2018.

Net Income (Loss) Attributable to Ionis Common Stockholders

Ionis reported net income attributable to Ionis’ common stockholders of $84 million for the first quarter of 2019 compared to a net loss of $1 million for the same period in 2018, both on a GAAP basis. On a non-GAAP basis, Ionis reported net income attributable to Ionis’ common stockholders of $126 million for the first quarter of 2019 compared to $25 million for the same period in 2018. The increase was primarily due to increases in revenue.

For the first quarter of 2019, basic and diluted net income per share were $0.63 and $0.62, respectively, compared to basic and diluted net loss per share of $0.01 for the same period in 2018. All amounts are on a GAAP basis.

Balance Sheet

Ionis added to its strong balance sheet, ending the first 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.

On May 9, 2019 Vaxart, Inc., a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, reported financial results for the first quarter ended March 31, 2019 (Press release, Vaxart, MAY 9, 2019, View Source [SID1234536090]).

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"With the Phase 1b study of our bivalent norovirus tablet vaccine underway and two successful financings behind us, we are well positioned to advance our lead product candidate, the first oral vaccine against norovirus, the leading cause of food-borne illness in the U.S.," said Wouter Latour, M.D., chief executive officer of Vaxart. "We expect to initiate the Phase 2 monovalent norovirus challenge study in the first half of 2019, and we expect topline results from both the Phase 1b and Phase 2 studies in the second half of 2019. In addition, we are advancing our first therapeutic vaccine for the treatment of HPV associated cervical dysplasia and cancer toward the clinic."

2019 Highlights:

Corporate:

On March 8, Vaxart announced the initiation of the open label lead-in portion of its Phase 1b bivalent norovirus vaccine clinical trial. On March 27, the Company announced it had completed dosing of the lead-in cohort, and on April 16 the first patient in the randomized cohort of the clinical trial was dosed.
On March 19, Vaxart announced the pricing of a registered direct offering of 1,200,000 shares of its common stock at a price of $2.50 per share. Total gross proceeds from the offering were $3.0 million.
On April 1, at the International Society for Influenza and other Respiratory Virus Diseases conference in Siena, Italy, and on April 3, at the Influenza Vaccines for the World 2019 conference in Edinburgh, Scotland, Vaxart presented new data from its Phase 2 influenza challenge study further solidifying the evidence that its oral tablet vaccine protects against influenza infection, primarily through mucosal immunity.
On April 9, Vaxart announced the pricing of an underwritten public offering of a total of 925,455 shares of common stock and 8,165,455 pre-funded warrants with an exercise price of $0.10 per share, as well as common warrants for 10,454,546 shares with an exercise price of $1.10 per share. The gross proceeds of the offering at close were $9.3 million. As of May 8, 2019, 6,519,091 pre-funded warrants had been exercised, bringing the aggregate gross proceeds up to $10.0 million, and 1,646,364 pre-funded warrants remained outstanding.
On April 13, at the 29th European Congress of Clinical Microbiology and Infectious Diseases in Amsterdam, the Netherlands, Vaxart presented preclinical data showing that Vaxart’s oral quadrivalent seasonal influenza vaccine conferred 100% protection against a lethal H5N1 pre-pandemic influenza challenge in ferrets, while in the Fluzone group only 62% of the animals survived.
Financial Results for the Three Months Ended March 31, 2019

Vaxart reported a net loss of $1.3 million for the first quarter of 2019 compared to net income of $2.3 million for the first quarter of 2018. The principal reason for the decrease was the absence of a $7.0 million one-off non-cash bargain purchase gain recorded in the first quarter of 2018, partially offset by an increase in revenue of $3.9 million, primarily from royalties on Inavir and Relenza.
Vaxart ended the quarter with cash and cash equivalents of $8.4 million compared to $11.5 million at December 31, 2018. The decrease was primarily due to cash used in operations, partially offset by the $2.5 million net raised in the registered direct offering in March 2019.
Revenue for the quarter was $5.4 million compared to $1.5 million in the first quarter of 2018. The $3.9 million increase was principally due to royalty revenue resulting from our merger with Aviragen being recorded for the full quarter in 2019, while in 2018 the majority of this revenue was earned pre-merger.
Research and development expenses were $3.8 million for the quarter compared to $3.4 million for the first quarter of 2018. The increase was mainly due to higher clinical and manufacturing costs incurred in the Company’s norovirus program and amortization of intangible assets acquired in the merger with Aviragen, partially offset by the discontinuation of the teslexivir program and completion of the BARDA contract.
General and administrative expenses were $2.0 million for the quarter, substantially unchanged from the first quarter of 2018.

On May 9, 2019 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health" or the "Company") reported that it has launched an offering of $750,000,000 aggregate principal amount of senior notes due 2028 (the "2028 Notes") and $750,000,000 aggregate principal amount of senior notes due 2029 (the "2029 Notes" and, together with the 2028 Notes, the "Notes") (Press release, Valeant, MAY 9, 2019, View Source [SID1234536040]). Bausch Health intends to use the net proceeds from the offering of the Notes, along with cash on hand, to repurchase $1,500 million aggregate principal amount across the Company’s outstanding 5.50% Senior Notes due 2023 (the "5.50% Notes") and 5.875% Senior Notes due 2023 (the "5.875% Notes" and, together with the 5.50% Notes, the "Existing Notes") pursuant to tender offers announced today, and to pay related fees and expenses. This announcement does not constitute an offer to purchase or the solicitation of an offer to sell the Existing Notes.

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The Notes will be guaranteed by each of the Company’s subsidiaries that are guarantors under the Company’s credit agreement and existing senior notes. Consummation of the offering of the Notes is subject to market and other conditions, and there can be no assurance that the Company will be able to successfully complete this transaction on the terms described above, or at all.

The Notes will not be registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state securities law and may not be offered or sold in the United States absent registration or an applicable exemption from registration under the Securities Act and applicable state securities laws. The Notes will be offered in the United States only to persons reasonably believed to be qualified institutional buyers pursuant to Rule 144A under the Securities Act and outside the United States to non-U.S. persons pursuant to Regulation S under the Securities Act. The Notes have not been and will not be qualified for sale to the public by prospectus under applicable Canadian securities laws and, accordingly, any offer and sale of the Notes in Canada will be made on a basis which is exempt from the prospectus requirements of such securities laws.

This news release is being issued pursuant to Rule 135c under the Securities Act and shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction.