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 Acceleron Pharma Inc. (Nasdaq:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the first quarter ended March 31, 2019 (Press release, Acceleron Pharma, MAY 9, 2019, View Source [SID1234536072]).

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"With the submission of marketing applications for luspatercept in the U.S. and E.U. last month, we and our global collaboration partner, Celgene, are excited about the potential to bring a new therapy to patients with myelodysplastic syndromes and beta-thalassemia within the next year," said Habib Dable, President and Chief Executive Officer of Acceleron. "At the same time, our pulmonary program remains on track, with enrollment ongoing in two Phase 2 trials of sotatercept in PAH, and we are anticipating topline results from the placebo-controlled part of the Phase 2 trial of our locally-acting muscle agent, ACE-083, in FSHD during the second half of this year."

Development Program Highlights

Hematology

Luspatercept: Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is an investigational first-in-class erythroid maturation agent (EMA) designed to address a late-stage erythroid maturation defect that results in chronic anemia and the need for regular red blood cell transfusions in adults with serious hematologic diseases. Luspatercept is part of the global collaboration between Acceleron and Celgene.

Celgene recently submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for luspatercept in patients with MDS- and beta-thalassemia-associated anemia based on the safety and efficacy results of the pivotal Phase 3 studies MEDALIST and BELIEVE.

The Companies expect to announce preliminary topline results from the Phase 2 trial of luspatercept in patients with MF in the second half of 2019.

Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS and the BEYOND Phase 2 trial in patients with non-transfusion-dependent beta-thalassemia, with preliminary results expected from the BEYOND trial in 2020.

Neuromuscular Disease

ACE-083: Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie-Tooth Disease (CMT)
ACE-083 is an investigational locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause focal muscle weakness. ACE-083 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta superfamily ligands involved in muscle formation.

Previously presented results from Part 1 of the Phase 2 trial evaluating ACE-083 in patients with FSHD were highlighted in an encore presentation at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in April 2019.

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Preliminary results from Part 2 of the Phase 2 trial in patients with FSHD are expected in the second half of 2019.

Previously presented results from Part 1 of the Phase 2 trial evaluating ACE-083 in patients with CMT will be highlighted in a platform presentation at the American Academy of Neurology (AAN) 71st Annual Meeting on May 10, 2019.

Enrollment is ongoing in Part 2 of the Phase 2 trial in patients with CMT, with preliminary results expected in the first quarter of 2020.

Pulmonary Disease

Sotatercept: Pulmonary Arterial Hypertension (PAH)
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure.

Enrollment is ongoing in the PULSAR Phase 2 trial in patients with PAH, with topline results expected in the first half of 2020.

Enrollment is ongoing in the exploratory SPECTRA trial in patients with PAH, with preliminary results expected in 2020.

A preclinical abstract of sotatercept in PAH has been accepted for presentation at the American Thoracic Society (ATS) 2019 International Conference on May 21, 2019.

Financial Results

Cash Position – Cash, cash equivalents and investments as of March 31, 2019 were $513.1 million. As of December 31, 2018, the Company had cash, cash equivalents and investments of $291.3 million. Based on the Company’s current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from luspatercept sales.

Revenue – Collaboration revenue for the first quarter was $2.8 million. The revenue is all from the Company’s partnership with Celgene and is primarily related to expenses incurred by the Company in support of luspatercept.

Costs and Expenses – Total costs and expenses for the first quarter were $43.6 million. This includes R&D expenses of $32.8 million and G&A expenses of $10.8 million.

Net loss – The Company’s net loss for the first quarter ended March 31, 2019 was $38.1 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first quarter 2019 financial results and provide an update on recent corporate activities on May 9, 2019, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron First Quarter 2019 Earnings Call."

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

On May 9, 2019 PDL BioPharma, Inc. ("PDL" or "the Company") (NASDAQ: PDLI) reported financial results for the three months ended March 31, 2019 (Press release, PDL BioPharma, MAY 9, 2019, View Source [SID1234536071]):

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First Quarter and Recent Financial Highlights

Total revenues of $38.9 million.

GAAP net income of $6.7 million or $0.05 per diluted share.

Non-GAAP net income attributable to PDL’s shareholders of $11.9 million. A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 at the end of this news release.

Cash and cash equivalents of $366.3 million as of March 31, 2019.

Repurchased 13.1 million shares of common stock in the open market during the first quarter of 2019 for $44.4 million, or an average price of $3.38 per share.

Invested $30.0 million in Evofem Biosciences, Inc. in April 2019.

"This is a very exciting time at PDL as we report strong first quarter financial results while we consider expanding our strategic transaction with Evofem Biosciences, as announced on April 11," said Dominique Monnet, president and CEO of PDL. "The elements of this transaction fit with our commitment to creating shareholder value by entering into strategic collaborations with pharmaceutical companies with innovative products and technologies. Evofem Biosciences provides us with an attractive opportunity to make a contribution to women’s healthcare, which presents many unmet medical needs that have been largely underserved by large pharmaceutical companies. Evofem’s lead investigational drug product, Amphora, offers a novel non-hormonal approach to contraception for women. Additionally, we are confident that Evofem’s team has the talent, expertise and dedication to execute successfully its commercial plan for Amphora.

"Should we make the second $30 million tranche of our proposed investment in Evofem, our team at PDL would bring significant value to the Amphora launch by contributing our capital and expertise in commercializing products in the U.S. and internationally," he added.

First Quarter Revenue Highlights

Total revenues of $38.9 million included:

Product revenue of $26.7 million, which consisted of $20.0 million from the sales of our branded prescription medicine products Tekturna and Tekturna HCT in the U.S. and Rasilez and Rasilez HCT in the rest of the world and revenue generated from the sale of an authorized generic form of Tekturna in the United States (collectively, the Noden Products), and $6.7 million of product revenue from the LENSAR Laser System.

Product revenue from the Noden Products was $12.2 million in the U.S. and $7.8 million in the rest of the world.

Net royalty payments from acquired royalty rights and a change in fair value of the royalty rights assets of $12.3 million, primarily related to the Assertio royalty asset.

Total revenues of $38.9 million, compared with $38.5 million for the first quarter of 2018.

Product revenue of $26.7 million, increased 14.4%, compared with $23.3 million for the prior-year period. The increase is primarily due to the initial inventory stocking related to the launch of an authorized generic form of Tekturna in the United States in March 2019.

PDL recognized $12.3 million in revenue from royalty rights – change in fair value, compared with $11.1 million in the prior-year period. The increase is related to higher royalties from the Assertio royalty asset.

PDL received $12.6 million in net cash royalties from its royalty rights in the first quarter of 2019.

Royalties from PDL’s licensees to the Queen et al. patents were less than $0.1 million in the first quarter of 2019, compared with $2.8 million for the first quarter of 2018 as royalties on the sales of Tysabri are nearing completion.

Interest revenue decreased by $0.7 million from the prior-year period due to CareView Communications not making its interest payment in the first quarter of 2019.

First Quarter Operating Expense Highlights

Operating expenses were $28.4 million, a $5.8 million decrease from $34.2 million for the first quarter of 2018. The variance was primarily a result of:

a $4.7 million decline in amortization expense for the Noden intangible assets as a result of the impairment recorded for these intangible assets in the second quarter of 2018,

a $1.2 million, or 10%, decline in general and administrative expenses primarily due to lower professional fees,

a $2.8 million, or 50%, decline in sales and marketing expenses, reflecting the cost savings from the change in our marketing strategy for the Noden Products,

offset by a $2.2 million increase in Noden Products and LENSAR cost of product revenue, due to higher sales in both segments,

a $0.6 million favorable adjustment to the fair value of the contingent consideration recorded in the first quarter of 2018 with no corresponding adjustment in the first quarter of 2019, and

higher research and development expenses in our Medical Devices segment.

Stock Repurchase Programs

In November 2018, PDL began repurchasing shares of its common stock pursuant to the $100.0 million share repurchase program. During the first quarter of 2019, the Company repurchased 13.1 million shares for an aggregate purchase price of $44.4 million, or an average cost of $3.38 per share, including trading commission.

Subsequent to the close of the first quarter of 2019, the Company repurchased 2.8 million shares at an average price of $3.77 per share, for a total of $10.4 million.

To date, the Company has repurchased 24.5 million shares for a total of $80.3 million in the $100.0 million program leaving $19.7 million available to be repurchased.

Since initiating its first stock repurchase program in March 2017, the Company has used $135.3 million to repurchase a total of 46.6 million shares of its common stock.

Other Financial Highlights

PDL had cash and cash equivalents of $366.3 million as of March 31, 2019, compared with cash and cash equivalents of $394.6 million as of December 31, 2018.

The reduction in cash and cash equivalents was primarily the result of common stock repurchases of $44.4 million, partially offset by the proceeds from operations and royalty rights.

Conference Call and Webcast Details

PDL will hold a conference call to discuss financial results and provide a business update at 4:30 p.m. Eastern time today. Slides to accompany the conference call will be available in the Investor Relations section of www.pdl.com.

To access the live conference call via phone, please dial 844-535-4071 from the U.S. and Canada or 706-679-2458 internationally. The conference ID is 1099595. A telephone replay will be available beginning approximately one hour after the call through one week following the call and may be accessed by dialing 855-859-2056 from the U.S. and Canada or 404-537-3406 internationally. The replay passcode is 1099595.

To access the live and subsequently archived webcast of the conference call, go to the Investor Relations section of www.pdl.com and select "Events & Presentations."

On May 9, 2019 Array BioPharma Inc. (Nasdaq: ARRY) reported that its Chief Executive Officer, Ron Squarer, will speak at the Bank of America Merrill Lynch 2019 Health Care Conference in Las Vegas (Press release, Array BioPharma, MAY 9, 2019, View Source [SID1234536070]). The public is welcome to participate in the conference through a webcast on the Array BioPharma website.

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Event:

Bank of America Merrill Lynch 2019 Health Care Conference

Presenter:

Ron Squarer, Chief Executive Officer, Array BioPharma

Date:

Thursday, May 16, 2019

Time:

10:00 a.m. Pacific Time / 1:00 p.m. Eastern Time

Webcast:

http://www.veracast.com/webcasts/baml/healthcare2019/id83306305205.cfm

On May 9, 2019 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company focused on the development and commercialization of therapeutics for patients with kidney disease, reported financial results for the first quarter ended March 31, 2019 (Press release, Akebia, MAY 9, 2019, View Source [SID1234536069]). The company also announced full enrollment of its global Phase 3 INNO2VATE studies for vadadustat, that it has bolstered its commercial capabilities with the addition of Dell Faulkingham as Senior Vice President, Chief Commercial Officer, and appointed Steven K. Burke, M.D., as Senior Vice President, Chief Medical Officer.

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"The first quarter marked the achievement of another important milestone for Akebia with the announcement of positive top-line results from two Phase 3, active-controlled, pivotal studies evaluating vadadustat in Japanese patients with anemia due to chronic kidney disease (CKD)," stated John P. Butler, President and Chief Executive Officer of Akebia Therapeutics. "We are excited by these results as they increase our level of confidence in the hypoxia inducible factor (HIF) pathway and more specifically, the direction of our clinical program for vadadustat. These results are expected to serve as the basis for a New Drug Application in Japan (JNDA) by our collaboration partner, Mitsubishi Tanabe Pharma Corporation (MTPC), in 2019."

Butler continued, "We are also pleased to announce the completion of enrollment in our global Phase 3 INNO2VATE studies evaluating vadadustat for the treatment of anemia due to CKD in dialysis-dependent CKD subjects. With the addition of Dell Faulkingham to our executive team, we have strengthened our commercial capabilities and believe we are well positioned to execute on our revenue growth strategies for Auryxia. Lastly, we look forward to welcoming Dr. Steven Burke as our new Chief Medical Officer as we continue to execute on the multiple catalysts expected with vadadustat’s Phase 3 program over the next 12 to 18 months."

Auryxia Highlights

•
Auryxia net product revenue for the first quarter of 2019 was $23.1 million, representing 12.1 percent growth over the first quarter of 2018.

•
Total Auryxia prescriptions were 40,080, representing 22.5 percent growth over the first quarter of 2018.

Vadadustat Highlights

•
Announced positive top-line results from two Phase 3, active-controlled, pivotal studies evaluating vadadustat in Japanese subjects with anemia due to CKD in March 2019. Data from these two pivotal studies as well as from two additional single-arm studies in peritoneal dialysis and hemodialysis subjects, also recently announced, are expected to serve as the basis for a JNDA submission by MTPC in 2019.

•
Expanded license agreement with Vifor Pharma announced in April 2019 creates opportunity for accelerated introduction of vadadustat, if approved by the U.S. Food and Drug Administration (FDA), in up to 60 percent of U.S. dialysis patients.

•
Enrollment in the global Phase 3 INNO2VATE studies evaluating the safety and efficacy of vadadustat in dialysis-dependent CKD subjects with anemia due to CKD, has been completed. Enrollment in the smaller of the two INNO2VATE studies (the "Correction Study"), was completed in April 2019, with a total of 369 subjects enrolled. Enrollment in the larger INNO2VATE study (the "Conversion Study") was completed in February 2019, with a total of 3,554 subjects enrolled. The company continues to expect to report top-line data from both INNO2VATE studies in the second quarter of 2020, subject to the accrual of major adverse cardiovascular events (MACE).

•
The company expects enrollment in the global Phase 3 PRO2TECT studies evaluating the safety and efficacy of vadadustat in non-dialysis dependent CKD subjects with anemia due to CKD, to be completed in 2019, with up to approximately 3,700 subjects expected to be enrolled. The company continues to expect to report top-line results in mid-2020, subject to the accrual of MACE.

Financial Results

Total revenue for the first quarter of 2019 was $72.7 million, compared to $45.9 million in the first quarter of 2018.

Auryxia net product revenue for the first quarter of 2019 was $23.1 million, compared to $20.6 million, as reported by Keryx Biopharmaceuticals, Inc. ("Keryx") prior to its merger with the company, during the same period in 2018. This represents a 12.1 percent increase in net product revenue from the first quarter of 2018. Auryxia is the company’s FDA approved oral iron tablet to treat non-dialysis dependent adult CKD patients for iron deficiency anemia (IDA) and dialysis-dependent adult CKD patients for hyperphosphatemia.

"As we previously discussed, the Centers for Medicare & Medicaid Services’ (CMS) new prior authorization requirement for Auryxia caused delays in approvals of prescriptions and negatively impacted Auryxia product revenue for the first quarter. We believe our efforts to help patients and prescribers navigate this process are working and we’re encouraged with the growth in weekly prescriptions that we are now seeing. In fact, the prescription demand we’ve seen in the first four weeks of the second quarter exceeded the first four weeks of any quarter since Auryxia was launched," stated Butler. "Looking ahead, we believe continued execution on our growth strategy and underlying market demand, will drive increased revenue for Auryxia in the second quarter and across the year."

Collaboration revenue for the first quarter of 2019 was $49.6 million, compared with $45.9 million in the first quarter of 2018. The increase was primarily due to increased collaboration revenue in the first quarter of 2019 from the company’s cost sharing arrangement under its Otsuka collaboration agreements. The company expects Otsuka to begin funding 80 percent of its development costs for vadadustat in the second quarter of 2019.

Cost of goods sold was $31.3 million for the first quarter of 2019, consisting of $7.6 million of costs associated with the manufacture of Auryxia and $23.7 million related to the application of purchase accounting as a result of the merger with Keryx, including $14.6 million of inventory step-up and $9.1 million of amortization of intangibles.

Research and development expenses were $82.4 million for the first quarter of 2019 compared to $61.4 million for the first quarter of 2018. The increase was primarily attributable to an increase in external costs related to the continued advancement of the PRO2TECT and INNO2VATE Phase 3 studies.

Selling, general and administrative expenses were $34.3 million for the first quarter of 2019 compared to $9.0 million for the first quarter of 2018. The increase in selling, general and administrative expenses was primarily attributable to commercialization costs associated with Auryxia, as there were no comparable commercialization costs in the first quarter of 2018.

The company reported a net loss for the first quarter of 2019 of $72.4 million, or ($0.62) per share, as compared to a net loss of $23.4 million, or ($0.48) per share, for the first quarter of 2018. The company’s net loss for the first quarter of 2019 includes the impact of merger-related accounting charges totaling $23.7 million, offset by a $2.8 million deferred tax benefit.

The company ended the quarter with cash, cash equivalents and available-for-sale securities of $168.0 million. The decrease from the fourth quarter of 2018 was primarily related to the timing of cash flows between quarters, including reimbursement amounts from the company’s collaboration partners and payments related to its Phase 3 program for vadadustat, including $13.0 million of advanced purchases of comparator drug inventory in anticipation of Brexit. Additionally, cash was impacted by one-time payments of certain previously accrued, merger-related liabilities totaling $30.0 million. The company continues to expect its cash resources, including the prepaid quarterly committed cost-share funding from its collaboration partners, to fund its current operating plan into the third quarter of 2020.

Leadership Team Additions

Steven K. Burke, M.D. will succeed Rita Jain, M.D., who informed the company of her plans to step down from her position as Senior Vice President, Chief Medical Officer effective June 17, 2019 to pursue other opportunities. Dr. Jain has been a valued member of the company’s leadership team, and during her tenure made important contributions to the vadadustat development program and enhanced the development organization. She has advised the company that she is committed to supporting the company during a transitional period and ensuring a seamless and successful transition to her successor, Dr. Burke.

Dr. Burke will join the company from Proteon Therapeutics, Inc., where he has been Senior Vice President and Chief Medical Officer since 2006. Prior to joining Proteon, Dr. Burke served as Senior Vice President of Medical and Regulatory Affairs at Genzyme Corporation, where he worked from 2001 to 2006. From 1994 to 2001, Dr. Burke held roles at GelTex Pharmaceuticals, Inc. including Vice President of Clinical Research and Medical Director, and before that he held positions at Glaxo, Inc.. Dr. Burke received an A.B. from Harvard College and an M.D. from Cornell University Medical College. He completed a medical residency and fellowship at Brigham and Women’s Hospital and is certified by the American Board of Internal Medicine.

Dell Faulkingham joins the company with more than 20 years of commercial experience across a broad range of specialty pharmaceutical categories. His experience includes commercial leadership roles with Biogen Inc., where he held multiple positions of increasing responsibility, most recently serving as Senior Vice President and Head, U.S. Multiple Sclerosis (MS) Franchise. Mr. Faulkingham also recently served as Vice President, Head of U.S. MS Marketing and Field Operations at Biogen. Prior to joining Biogen, Mr. Faulkingham held several roles with Takeda Pharmaceuticals. Mr. Faulkingham began his career in sales at Forest Pharmaceuticals, Inc. and received a B.S. in biology from the University of Georgia.

Conference Call:

Akebia will host a conference call today, Thursday, May 9, 2019, at 9:00 a.m. Eastern Time to discuss its first quarter financial results. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 4271217. The call will also be webcast LIVE and can be accessed via the Investors section of the company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through May 15, 2019. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 4271217. An online archive of the conference call can be accessed via the Investors section of the company’s website at View Source