1stOncology™: Cancer Intelligence Service – Page 13050 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

AVEO Reports First Quarter 2019 Financial Results and Provides Business Update

On May 9, 2019 AVEO Oncology (NASDAQ: AVEO) reported financial results for the first quarter ended March 31, 2019 and provided a business update (Press release, AVEO, MAY 9, 2019, View Source [SID1234536077]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

"With a successful recent equity offering, together with the triggering of a FOTIVDA (tivozanib) milestone from EUSA, AVEO’s strengthened balance sheet provides us with a cash runway that we expect will take us into the fourth quarter of 2020," said Michael Bailey, president and chief executive officer of AVEO. "We remain committed to our goal of improving outcomes and patient experience in renal cell carcinoma (RCC), and look forward to reporting more mature interim OS results from our TIVO-3 study in advanced or metastatic RCC, which we expect will occur in the fourth quarter of 2019, as well as the subsequent decision regarding a potential NDA filing in the U.S. We also continue to make progress with the balance of our programs and pipeline, most notably the ongoing clinical collaborations combining FOTIVDA with Bristol Myers Squibb’s OPDIVO (nivolumab) for the TiNivo study in RCC and with AstraZeneca’s IMFINZI (durvalumab) in first-line hepatocellular carcinoma, ongoing studies of ficlatuzumab in multiple oncology indications, and the emerging potential of a new ocular formulation of tivozanib for the treatment of age-related macular degeneration."

Recent Highlights

$2 Million Milestone Payment from EUSA Pharma Triggered. In April 2019, AVEO announced the triggering of a $2 million milestone payment from EUSA Pharma related to the February 2019 reimbursement approval and subsequent commercial launch of FOTIVDA (tivozanib) in Spain as a first-line treatment of adult patients with RCC.

Closing of Public Offering of Common Stock and Warrants. In April 2019, AVEO completed an underwritten public offering of 21,739,131 shares of common stock and 25,000,000 warrants to purchase common stock at the public offering price of $1.14 per share and $0.01 per warrant. The warrants have a two-year term and a strike price of $1.25 per share. Gross proceeds of the offering were approximately $25.0 million and are expected to be used for ongoing clinical and preclinical development of AVEO’s product candidates, as well as for working capital and other general corporate purposes.

Announced Positive Results from Phase 1b Ficlatuzumab-Cytarabine Trial (CyFi) in Patients with Relapsed and Refractory AML. In April 2019, AVEO announced the presentation of positive data from an investigator-sponsored Phase 1b expansion cohort of ficlatuzumab, AVEO’s potent hepatocyte growth factor (HGF) inhibitory antibody in combination with cytarabine in patients with relapsed and refractory acute myeloid leukemia (AML), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, held March 29 – Apr 3, 2019 in Atlanta. Of 18 AML patients enrolled in the study, all had disease that was refractory to initial treatment, 17 were evaluable and 9 achieved a complete response. The most frequent grade 3/4 treatment emergent adverse events observed were febrile neutropenia, LFT abnormalities, and electrolyte disturbance.

There was one death from sepsis and multi-organ failure that was determined to be disease related, and one patient withdrew from the study due to grade 4 gastrointestinal bleed, determined to be likely ficlatuzumab related. A copy of the presentation is currently available in the Publications & Presentation section of AVEO’s website.

Based on these results, the Company is evaluating potential next steps for this program in collaboration with its ficlatuzumab development and commercialization partner, Biodesix, Inc.

Appointed Gregory T. Mayes to Board of Directors. In February 2019, the Company announced the appointment of Gregory T. Mayes to its Board of Directors. Mr. Mayes brings to the AVEO Board over 20 years of experience as a biopharmaceutical executive with deep expertise in public company governance, business strategy and the commercialization of life sciences products.

Presented Topline Results from TIVO-3 in an Oral Presentation at the 2019 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium and Announced Updated NDA Timing. In February 2019, AVEO presented topline results from the TIVO-3 trial, AVEO’s Phase 3 randomized, controlled, multi-center, open-label study to compare tivozanib to sorafenib in 350 subjects with refractory advanced or metastatic RCC at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (GU) Cancers Symposium held February 14-16, 2019 in San Francisco. The results were presented during an oral presentation titled "TIVO-3: A Phase 3, Randomized, Controlled, Multi-Center, Open-Label Study to Compare Tivozanib to Sorafenib in Subjects with Refractory Advanced Renal Cell Carcinoma (RCC)." A copy of the presentation is currently available in the Publications & Presentation section of AVEO’s website.

The presentation noted that the TIVO-3 trial met its primary endpoint of demonstrating a statistically significant benefit in progression-free survival (PFS) versus sorafenib. There was also a significant PFS improvement demonstrated for tivozanib both in the subgroups of patients who received prior PD-1 therapy and those who received two prior VEGF TKI therapies. The secondary endpoint of overall response rate demonstrated a statistically significant improvement for patients receiving tivozanib compared to sorafenib. The analysis of the secondary endpoint of overall survival (OS) was not mature at the time of the final PFS analysis, but the hazard ratio at the time of the analysis favored sorafenib. Tivozanib was generally well-tolerated, with grade 3 or higher adverse events consistent with those observed in previous tivozanib trials. Infrequent but severe adverse events reported in greater number in the tivozanib arm were thrombotic events similar to those observed in previous tivozanib studies. The most common adverse event in patients receiving tivozanib was hypertension, an adverse event known to reflect effective VEGF pathway inhibition.

AVEO intends to initiate an additional interim OS analysis in August 2019, the results of which are expected to be reported in the fourth quarter of 2019 and would be the first planned update since the prior OS analysis was initiated in the fourth quarter of 2018. At the recommendation of the U.S. Food and Drug Administration, AVEO plans to make a

New Drug Application (NDA) filing decision following the availability of more mature OS results.

First Quarter 2019 Financial Results

AVEO ended Q1 2019 with $23.5 million in cash, cash equivalents and marketable securities as compared with $24.4 million at December 31, 2018.

Total revenue for Q1 2019 was approximately $1.6 million compared with $1.0 million for Q1 2018.

Research and development expense for Q1 2019 was $6.9 million compared with $5.4 million for Q1 2018.

General and administrative expense for Q1 2019 was $2.5 million compared with $2.6 million for Q1 2018.

Net income for Q1 2019 was $0.6 million, or net income of $0.01 and net loss of $0.06 per basic and diluted share, respectively, compared with a net loss of $9.0 million for Q1 2018, or net loss of $0.08 per basic and diluted share.

The Q1 2019 net income was driven by an approximate $8.8 million non-cash gain attributable to the decrease in the fair value of the 2016 PIPE warrant liability that principally resulted from the decrease in the stock price that occurred during the fiscal quarter. In Q1 2018, the non-cash loss attributable to the increase in the fair value of such warrant liability was $1.5 million.

Financial Guidance

AVEO believes that our approximate $23.5 million in cash, cash equivalents and marketable securities at March 31, 2019, along with approximately $24.2 million in additional net funding received in the second quarter of 2019 to-date, as described above, would allow us to fund our planned operations into the fourth quarter of 2020. This estimate excludes possible additional clinical trials we may sponsor and, subject to our decision whether to submit an NDA for tivozanib to the FDA following the availability of more mature OS results, remaining costs to prepare and filing fees in connection with a possible NDA submission, any related drug manufacturing and drug supply distribution, and pre-commercialization activities that we may undertake. This estimate also assumes no receipt of additional milestone payments from our partners, no funding from new partnership agreements, no additional equity financings, no debt financings, no additional sales of equity under our Leerink Sales Agreement and no additional sales of equity through the exercise of our outstanding warrants. Accordingly, the timing and nature of activities contemplated for the remainder of 2019 and thereafter will be conducted subject to the availability of sufficient financial resources.

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI) discovered by Kyowa Hakko Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union plus Norway and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF

receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC. Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal and breast cancers. In addition, a new formulation of tivozanib is in pre-clinical development for the treatment of age-related macular degeneration.

About Ficlatuzumab

Ficlatuzumab (formerly known as AV-299) is a potent hepatocyte growth factor (HGF) inhibitory antibody that binds to the HGF ligand with high affinity and specificity to inhibit HGF/c-Met biological activities. AVEO and Biodesix, Inc. have a worldwide agreement to develop and commercialize ficlatuzumab. Ficlatuzumab is currently being evaluated in investigator-sponsored trials in squamous cell carcinoma of the head and neck (HNSCC), metastatic pancreatic ductal cancer (PDAC), and acute myeloid leukemia (AML).

Immunomedics Reports First Quarter 2019 Results and Provides Corporate Update

On May 9, 2019 Immunomedics, Inc., (NASDAQ: IMMU) ("Immunomedics" or the "Company"), a leading biopharmaceutical company in the area of antibody-drug conjugates (ADC), reported financial results for the first quarter of 2019 (Press release, Immunomedics, MAY 9, 2019, View Source [SID1234536076]). Please refer to the Company’s Quarterly Report on Form 10-Q for more details on the Company’s financial results.

"We have made meaningful progress in all business areas during the past three months. Significantly, we have received clarity from the FDA on the CRL and plan to resubmit the BLA in early fourth quarter of 2019. In the meantime, our salesforce is working closely with Janssen Biotech Inc. to co-promote Balversa, allowing our team to further build on its extensive experience in the oncology community. To enhance shareholder value and to continue on our path to becoming a global biopharmaceutical company, we are partnering with China-based Everest Medicines to develop and commercialize sacituzumab govitecan in Greater China, the world’s fastest growing pharmaceutical market. Finally, we have launched the registrational Phase 3 TROPICS-02 study in late-line HR+/HER2 metastatic breast cancer to potentially address a large unmet market," said Behzad Aghazadeh, Executive Chairman of Immunomedics.

The randomized global TROPICS-02 study is expected to enroll approximately 400 patients with hormonal receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2–) metastatic breast cancer (mBC) who have failed at least two prior chemotherapy regimens for metastatic disease. Patients are randomized to receive either sacituzumab govitecan or physician’s choice of eribulin, capecitabine, gemcitabine or vinorelbine.

The primary endpoint will be progression-free survival with overall survival serving as secondary endpoint. The protocol also allows for an interim analysis of overall response rate (ORR) and duration of response (DoR), the results of which could support a potential accelerated approval submission.

Recent Company Highlights

Phase 2 data with sacituzumab govitecan in metastatic triple-negative breast cancer (mTNBC) were published in the New England Journal of Medicine.1

The Company’s sales force is co-promoting Balversa (erdafitinib) in the U.S. with Janssen Biotech Inc. until the end of March 2020. Should sacituzumab govitecan be approved for mTNBC in the U.S. before that time, Immunomedics is only required to support Balversa in second position detail.

The Company entered into an exclusive license agreement with Everest Medicines, the largest single-asset in-licensing deal for regional China to-date, to support development, registration, and commercialization of sacituzumab govitecan for key cancer indications in Greater China, South Korea and certain Southeast Asian countries. The Company will receive a $65 million upfront payment and an additional $60 million based on the FDA approval of sacituzumab govitecan in mTNBC in the U.S., and has the potential to receive an additional $710 million, if certain milestones are achieved.

The Company met with the FDA to discuss issues raised in the Complete Response Letter (CRL) it received in January 2019. After receiving clarity from the regulatory agency, the Company plans to resubmit its Biologics License Application in early fourth quarter of 2019 seeking the approval of sacituzumab govitecan for the treatment of patients with mTNBC who have received two prior therapies for metastatic disease.

A registrational Phase 3 TROPICS-02 study of sacituzumab govitecan was launched in late-line HR+/HER2‒ mBC. This study could support a potential accelerated approval submission with interim results of ORR and DoR.
Results for the First Quarter of 2019
The Company had no revenues for the three months ended March 31, 2019, due primarily to the discontinued sale of LeukoScan during the quarter ended March 31, 2018 in order for the Company to focus on its ADC business. Revenues in the comparable quarter ended March 31, 2018, were approximately $0.5 million.

Total costs and expenses were $79.6 million for the three months ended March 31, 2019, compared to $38.1 million for the comparable quarter ended March 31, 2018, due primarily to a $29.3 million increase in research and development expenses, a $6.7 million increase in general and administrative expenses, and a $5.5 million increase in sales and marketing expenses. Most of these increases were attributable to activities related to preparations for the potential approval and commercial launch of sacituzumab govitecan for patients with at least two prior lines of treatment for metastatic TNBC in the United States, and to expanded clinical development of sacituzumab govitecan into other indications.

The Company had no non-cash income or expense for the three months ending March 31, 2019, compared to a $9.8 million non-cash gain for the comparable quarter ended March 31, 2018, due to a decrease in the fair value of outstanding warrants. There were no warrants outstanding as of March 31, 2019.

Interest expense was $10.0 million for the three months ended March 31, 2019, compared to $10.9 million for the comparable quarter March 31, 2018. The decrease was due primarily to changes in the fair value of our debt balances as a result of the agreement with RPI Finance Trust.

Net loss attributable to stockholders was $87.3 million, or $0.46 per share, for the three months ended March 31, 2019, compared to $35.5 million, or $0.21 per share, for the comparable quarter ended March 31, 2018.

As of March 31, 2019, the Company had $442.7 million in cash, cash equivalents, and marketable securities, which it believes is adequate to support its clinical development plan for sacituzumab govitecan; further build its clinical and manufacturing infrastructure and fund its operations through 2020.

Conference Call
The Company will host a conference call and live audio webcast today at 8:00 a.m. Eastern Time to discuss first quarter 2019 financial results and provide a corporate update. To access the conference call, please dial (877) 303-2523 or (253) 237-1755 using the Conference ID 5357619. The conference call will be webcast via the Investors page on the Company’s website at View Source Approximately two hours following the live event, a webcast replay of the conference call will be available on the Company’s website for approximately 30 days.

Reference

View Source

UroGen Pharma Reports First Quarter 2019 Financial Results and Recent Corporate Developments

On May 9, 2019 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in uro-oncology, reported financial results for the first quarter ended March 31, 2019 and provided an overview of the Company’s recent developments (Press release, UroGen Pharma, MAY 9, 2019, View Source [SID1234536075]).

"The accomplishments and performance of UroGen during the first quarter have set the stage for a pivotal and exciting year ahead as we prepare for the potential approval and commercialization of UGN-101, our first investigational candidate. Our top priority remains completion of our rolling New Drug Application (NDA) for UGN-101 for the treatment of patients with low-grade upper tract urothelial cancer (LG UTUC) and, with our commercial preparations well underway, we are confident in our readiness to deliver on a strong launch in the first half of 2020," said Liz Barrett, President and Chief Executive Officer of UroGen. "Our company sees great possibilities for the RTGel platform within uro-oncology, especially around new therapeutic modalities. Our goal is to leverage that and continue to build a company with sustainable growth via a robust pipeline as well as assessing opportunities via external partnerships."

Recent Highlights

UGN-101 Clinical Development:

At the 114th American Urological Association (AUA) Annual Meeting in Chicago, Seth Lerner, M.D. delivered a presentation during the plenary session that highlighted the unmet need and potential for UGN-101 to change the treatment paradigm for patients with LG UTUC.

The updated analysis demonstrated that in the OLYMPUS intent-to-treat population, 71 patients had undergone primary disease evaluation (PDE) at the time of the analysis and 42 of the 71 patients (59 percent) achieved a complete response (CR). Forty-one patients entered follow-up. At the time of the analysis, 27 patients underwent a six-month evaluation, and 24 out of 27 patients (89 percent) have remained disease free at six months. The most common adverse events observed were urinary tract infection, ureteral narrowing and stricture formation. The majority of ureteral events were reported as mild to moderate and have resolved. Full Phase 3 data from the OLYMPUS trial is anticipated for 2H 2019.

The Company remains on track to complete its rolling NDA for UGN-101 in 2H 2019 and is planning for approval in 1H 2020. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC. Full data is planned for 2H 2019.

Pipeline Advancement:

UGN-102:

The Company continues to enroll patients in its Phase 2b OPTIMA II clinical trial of UGN-102 (mitomycin gel) for intravesical instillation as a first-line chemoablation agent in the treatment of patients with intermediate risk low-grade non-muscle invasive bladder cancer (LG NMIBC), a form of disease associated with a high risk of recurrence.

Initial data from the OPTIMA II trial of UGN-102 is expected in 2H 2019.

There are currently no drugs approved by the FDA as first-line treatment for LG NMIBC. UGN-102 represents a substantial opportunity in UroGen’s pipeline and has the potential to be a treatment option for up to approximately 80,000 patients for whom repetitive surgical resection via Transurethral Resection of Bladder Tumor (TURBT) remains the standard of care.

UGN-201:

UroGen is currently evaluating the optimal pathway to advance UGN-201, a TLR7/8 immunomodulatory agent for the treatment of high-grade bladder disease.

Commercial Preparations:

The Company continues to accelerate its pre-commercial activities and infrastructure build-out to support the anticipated U.S. approval and launch of UGN-101 targeted for 1H 2020. The focus is on building awareness of our RTGel technology and unmet needs in UTUC to support rapid adoption and seamless integration of UGN-101 into the urologist practice following regulatory approval.

A strong team of seven medical science liaisons (MSLs) have been strategically deployed across the U.S. to engage in scientific exchange and clinical support.

The Company launched www.UTUC.com, the first resource designed to address a void in the urology space by educating patients about UTUC and available treatment options.

Business Development:

UroGen recently entered into an agreement with Janssen Research & Development, LLC (Janssen) to conduct an early-stage feasibility evaluation in a therapeutic area of mutual interest. UroGen and Janssen will each conduct certain activities under the terms of the agreement.

First Quarter 2019 Financial Results; 2019 Guidance

As of March 31, 2019, cash and cash equivalents totaled $246.7 million. This includes net proceeds of approximately $161.4 million from a public offering of ordinary shares in January 2019.

Research and development expenses for the three months ended March 31, 2019 were $9.7 million, including non-cash share-based compensation expense of $2.3 million.

General and administrative expenses for the three months ended March 31, 2019 were $12.7 million, including non-cash share-based compensation expense of $5.1 million.

The Company reported a net loss of $21.4 million, or basic and diluted net loss per ordinary share of $1.11, for the three months ended March 31, 2019.

The 2019 financial guidance set forth during the Company’s year-end earnings call on February 28th remains the same based on current business goals and anticipated activities.

Conference Call & Webcast Information

Members of UroGen’s management team will host a live conference call and webcast today at 8:30 am Eastern Time to review the Company’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (888) 771-4371 (U.S.) or (847) 585-4405 (International) to listen to the live conference call. The conference ID number for the live call will be 48486174. An archive of the webcast will be available for two weeks on the Company’s website.

Magenta Therapeutics Reports First Quarter 2019 Financial Results and Recent Business Highlights

On May 9, 2019 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported financial results for the first quarter ended March 31, 2019 and recent business highlights (Press release, Magenta Therapeutics, MAY 9, 2019, View Source [SID1234536074]).

"In 2019 we are continuing to advance our portfolio of programs toward our vision of curing more patients with autoimmune diseases, blood cancers and genetic diseases. This momentum was reflected in the recent start of our Phase 1 study of MGTA-145, our first-line therapy for stem cell mobilization and second clinical program, as well as in the extended evidence of disease benefit we see in our Phase 2 study of our MGTA-456 cell therapy in patients with inherited metabolic disorders," said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. "We are positioned to build on this momentum through additional important milestones across each of our programs and to deliver value for patients and shareholders."

Upcoming Anticipated Milestones:

The Company plans to achieve the following key milestones in 2019:

Present preclinical data on C100 anti-CD45 targeted conditioning program in autoimmune disease and declare a development candidate

Present preclinical data on C200 anti-CD117 targeted conditioning program in gene therapy

Present clinical data from the Phase 1 study of MGTA-145

Present additional clinical data from the Phase 2 study of MGTA-456 in inherited metabolic disorders (IMDs)

Recent Business Highlights:

Dosed first subjects in Phase 1 clinical trial of MGTA-145 first-line stem cell mobilization product candidate: In April 2019, Magenta announced that it had dosed the first subjects in a Phase 1 study of MGTA-145. Magenta intends to develop MGTA-145 in autoimmune diseases, blood cancers and genetic diseases. The Phase 1 study will investigate the safety and tolerability of MGTA-145 alone and in combination with plerixafor in healthy volunteers and establish recommended Phase 2 doses. The study will also measure the number of hematopoietic stem cells in the blood after dosing with MGTA-145 alone and in combination with plerixafor. Magenta expects to present data from the study in the second half of 2019. Depending on the Phase 1 data, the Company plans to move MGTA-145 into a Phase 2 study in multiple myeloma and non-Hodgkin lymphoma in 2020.

Updated clinical data for MGTA-456 cell therapy showed continued signs of durable clinical benefit in patients with IMDs: Magenta presented updated data from the Phase 2 clinical study of MGTA-456 in patients with IMDs at the American Academy of Neurology (AAN) annual meeting in May 2019. Patients with cerebral adrenoleukodystrophy (cALD) treated with MGTA-456 in the study showed stable neurological function scores and persistent resolution of brain inflammation by MRI at 6 months post-transplant, suggesting that the progression of disease has been halted. Magenta expects to update these results in the second half of 2019.

Preclinical data on E478 stem cell gene therapy expansion program show significant increase in gene-modified stem cells: At the American Society of Gene and Cell Therapy annual meeting in May 2019, Magenta presented data showing that E478 increased the number of human hematopoietic stem cells modified with either CRISPR/Cas9 or lentiviral vector by 10-fold compared to standard culture methods. Magenta is developing E478 to achieve high doses of gene-modified stem cells for better outcomes in patients with genetic disorders, including sickle cell disease and beta-thalassemia, where gene editing or viral vector technologies are used to correct stem cells. Magenta intends to develop E478 in partnership with gene therapy companies.

Presented nine abstracts at Transplant and Cellular Therapies Conference: Magenta presented data covering the breadth of the Company’s integrated portfolio of programs at the Transplant and Cellular Therapy (TCT) annual meeting in February 2019.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2019, were $127.3 million compared to $142.6 million on December 31, 2018. In addition, earlier this week Magenta announced that it completed a public offering of common stock and raised gross proceeds of $64.8 million. Magenta anticipates that its cash, cash equivalents and marketable securities, including the proceeds from this recent financing, will be sufficient to fund operations and capital expenditures into the second half of 2021.

Research and Development Expenses: Research and development (R&D) expenses were $10.5 million in the first quarter of 2019, compared to $7.8 million in the first quarter of 2018. The increase was driven by investments related to the IND filing and clinical activities for MGTA-145, as well as the on-going clinical development of MGTA-456.

General and Administrative Expenses: General and administrative (G&A) expenses were $5.8 million for the first quarter of 2019, compared to $3.5 million for the first quarter in 2018. The increase was primarily due to increased personnel and facility costs associated with the growth of the Company.

Net Loss: Net loss was $14.8 million for the first quarter of 2019, compared to net loss of $11.2 million for the first quarter of 2018.

Ionis reports first quarter 2019 financial results

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]).

"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.