On August 7, 2018 AVEO Oncology (NASDAQ: AVEO) reported financial results for the second quarter ended June 30, 2018 and provided a business update (Press release, AVEO, AUG 7, 2018, View Source [SID1234528477]).

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"Our U.S. registration strategy remains a key focus for AVEO, with topline readout of the Phase 3 TIVO-3 study expected in the fourth quarter of this year," said Michael Bailey, president and chief executive officer of AVEO. "TIVO-3 is the first randomized Phase 3 study in advanced kidney cancer to stratify for prior immunotherapy and, as a result, it has the potential to serve as a benchmark study for the sequencing of therapies in advanced disease."

Mr. Bailey added: "The next two pillars of our tivozanib strategy also continue to make progress. The recent launch of FOTIVDA in Scotland adds to ongoing commercial efforts in Germany, the U.K., and Austria by our partner EUSA Pharma. We anticipate additional potential reimbursement approvals for France, Germany, Italy, and Spain in the coming months, triggering up to $8 million in milestone payments due to AVEO in addition to double-digit royalty payments on net sales of FOTIVDA in Europe. EUSA has the option to access TIVO-3 data in the event of a positive outcome in exchange for a $20 million R&D reimbursement payment to AVEO. Finally, we look forward to presenting additional data at the ESMO (Free ESMO Whitepaper) meeting in October from the Phase 2 portion of the TiNivo study of tivozanib and nivolumab (OPDIVO) in aRCC, a study which to date has demonstrated promising activity and a favorable safety profile."

Tivozanib TIVO-3 Study North America Update

Topline Data from Phase 3 TIVO-3 Study Anticipated in the Fourth Quarter of 2018. As previously announced, the Company expects to report topline results from the TIVO-3 study, AVEO’s Phase 3 trial of tivozanib as a third-line treatment for advanced renal cell carcinoma (aRCC), in the fourth quarter of 2018, approximately 6-8 weeks after the trial records 255 progression free survival (PFS) events. AVEO plans to announce when 255 PFS events have occurred and the topline data analysis for the trial has been initiated. Together with the TIVO-1 study, TIVO-3 is designed to serve as the basis for a potential U.S. approval of tivozanib (FOTIVDA) as a first- and third-line treatment for aRCC.

Tivozanib (FOTIVDA) European Union Updates

Tivozanib (FOTIVDA) Launched in Scotland for the Treatment of aRCC. In July 2018, FOTIVDA was launched in Scotland for the first-line treatment of adult patients with aRCC after the Scottish Medicines Consortium approved its use. FOTIVDA is now available in Germany, Scotland, the U.K., and Austria. FOTIVDA was granted European

Medicines Association approval in August 2017 for the treatment of adult patients with aRCC in the European Union plus Norway and Iceland.

Tivozanib (FOTIVDA) Expanded Access Program Launched in Italy. In July 2018, The Program of Therapeutic Use Tivozanib (Expanded Access Program) for renal cell carcinoma (RCC) was initiated, allowing patients in Italy to have access to tivozanib as front-line therapy. In addition, EUSA pharma is effecting the reimbursement procedure with the Italian Drug Agency (AIFA), a process which is expected to be finalized in the coming months.

Additional Tivozanib Updates

Updated Phase 2 Results from the TiNivo Trial of Tivozanib and Nivolumab (OPDIVO) in aRCC to be Presented at the 2018 ESMO (Free ESMO Whitepaper) Annual Meeting. Updated Phase 2 data from the Phase 1b/2 TiNivo study of tivozanib in combination with nivolumab (OPDIVO, Bristol-Myers Squibb), an immune checkpoint, or PD-1, inhibitor, will be presented at the 2018 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Meeting in Munich. The data will be presented during a poster presentation titled, "TiNivo: Tivozanib combined with nivolumab: safety and efficacy in patients with metastatic renal cell carcinoma (mRCC)" (Presentation Number 878P). Previously presented results support the potential advantages of a combination therapy using a high-specificity VEGF inhibitor TKI in connection with an immune checkpoint therapy in renal cancer.

Ficlatuzumab Update

Trials in Progress Poster for Phase 2 Study of Ficlatuzumab in Combination with Cetuximab in HNSCC to be Presented at the 2018 ESMO (Free ESMO Whitepaper) Annual Meeting. Data from an ongoing, investigator-sponsored Phase 2 trial of ficlatuzumab and cetuximab (ERBITUX), an EGFR-targeted antibody, in patients with cetuximab-resistant, metastatic head and neck squamous cell carcinoma (HNSCC) will be presented as a trials in progress poster at the 2018 ESMO (Free ESMO Whitepaper) Annual Meeting (Presentation Number 1124TiP). This randomized multi-center study, which is being conducted under the direction of Julie E. Bauman, MD, MPH, Professor of Medicine, Chief, Division of Hematology/Oncology, Associate Director of Translational Research, University of Arizona Cancer Center, is expected to enroll approximately 60 patients randomized to receive either ficlatuzumab alone or ficlatuzumab and cetuximab.

Trials in Progress Poster for Phase 1b Study of Ficlatuzumab in Combination with Gemcitabine and Nab-paclitaxel in Pancreatic Cancer Presented at the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting. Data from an ongoing, investigator-sponsored Phase 1b study to test the safety and tolerability of ficlatuzumab when combined with nab-paclitaxel and gemcitabine in previously untreated metastatic pancreatic ductal cancer (PDAC) was presented as a trials in progress poster (Poster Board: #330b, Abstract TPS4152) at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL in June 2018. The study, which is being conducted under the direction of Kimberly Perez,

MD at the Dana-Farber Cancer Institute, is currently enrolling, with an expected total enrollment of approximately 30 patients.

Corporate Update

Added to the Russell 2000, Russell 3000, and Russell Microcap Indexes. In June 2018, AVEO announced that it had been added to the Russell 2000, Russell 3000, and Russell Microcap Indexes as part of FTSE’s annual reconstitution. Russell U.S. Indexes are widely used by investment managers and institutional investors as the basis for index funds and as benchmarks for active investment strategies. Approximately $9 trillion in assets are benchmarked against Russell U.S. Indexes.

Second Quarter 2018 Financial Highlights

AVEO ended Q2 2018 with $18.1 million in cash, cash equivalents and marketable securities as compared with $33.5 million at December 31, 2017.

Total revenue for Q2 2018 was approximately $0.4 million compared with $0.4 million for Q2 2017.

Research and development expense for Q2 2018 was $4.9 million compared with $6.9 million for Q2 2017.

General and administrative expense for Q2 2018 was $2.8 million compared with $2.3 million for Q2 2017.

Net income for Q2 2018 was $4.0 million, or income of $0.03 per basic share and a loss of $0.06 per diluted share, compared with net loss of $33.3 million for Q2 2017, or a loss of $0.30 per basic and diluted share. Approximately $11.1 million of Q2 2018 net income was a non-cash gain attributable to the decrease in the fair value of the 2016 private placement warrant liability that principally resulted from the decrease in the stock price that occurred within the quarter. In Q2 2017, the non-cash loss attributable to the increase in the fair value of such warrant liability was $23.9 million.

Financial Guidance

We believe that our $18.1 million in cash resources would allow us to fund our planned operations into the first quarter of 2019. This estimate assumes no receipt of additional milestones from our partners, no additional funding from new partnership agreements, no additional equity or debt financings, and no sales of equity through the exercise of our outstanding warrants issued in connection with our 2016 private placement or outstanding warrants issued in connection with the recent settlement of our securities class action litigation.

On August 7, 2018 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that Scott Wolchko, President and Chief Executive Officer, will present at the 2018 Wedbush PacGrow Healthcare Conference in New York on Tuesday, August 14, 2018 at 2:30 p.m. ET (Press release, Fate Therapeutics, AUG 7, 2018, View Source [SID1234528495]).

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A live webcast of the presentation will be available through the investor relations section of the Company’s website at www.fatetherapeutics.com. Following the live webcast, an archived replay will be available on the Company’s website.

On August 7, 2018 Array BioPharma Inc. (Nasdaq: ARRY) reported that it will report financial results for the fourth quarter and full year of fiscal 2018 and hold a conference call to discuss those results on Tuesday, August 14, 2018 (Press release, Array BioPharma, AUG 7, 2018, View Source [SID1234528515]). Ron Squarer, Chief Executive Officer, will lead the call.

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Date: Tuesday, August 14, 2018

Time: 9:00 a.m. Eastern Time

Toll-Free: (844) 464-3927

Toll: (765) 507-2598

Pass Code: 1766079

On August 7, 2018 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported financial results for the second quarter of 2018 and highlighted its recent business and pipeline successes (Press release, Ionis Pharmaceuticals, AUG 7, 2018, View Source [SID1234528559]).

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"With the approval of TEGSEDI in Europe, Ionis is entering a new chapter as a multi-product, sustainably profitable company. We anticipate the approval and launch of TEGSEDI and WAYLIVRA in multiple markets this year," said Stanley T. Crooke, M.D., Ph.D., chairman of the board and chief executive officer of Ionis. "Adding TEGSEDI and WAYLIVRA product sales to growing revenues from SPINRAZA positions Ionis for continued growth. In addition, we expect at least three of our drugs to advance into pivotal trials by the end of 2019, representing our next wave of near-term commercial opportunities. Importantly, we have achieved these successes while generating operating profits due to the combination of our efficient technology platform and business strategy."

Second Quarter 2018 Financial Highlights

Revenues increased by 15 percent

For the second quarter and year-to-date 2018 revenue was $118 million and $262 million, compared to $112 million and $228 million for the same periods in 2017

Commercial revenue from SPINRAZA for year-to-date 2018 was $98 million, a three-fold increase over year-to-date 2017

Commercial revenue was more than 35 percent of Ionis’ total revenue in the first half of 2018 compared to less than 15 percent for the same period in 2017, reflecting Ionis’ transition to a commercial company

On track for third consecutive year of pro forma operating profitability while investing in the launch of two drugs

GAAP operating results were a loss of $50 million and $54 million for the second quarter and year-to-date 2018, respectively, compared to income of $6 million and $26 million for the same periods in 2017

Pro forma operating results were a loss of $16 million and income of $9 million for the second quarter and year-to-date 2018, respectively, compared to income of $28 million and $68 million for the same periods in 2017

Operating expenses increased primarily due to higher SG&A expenses related to the commercialization of TEGSEDI and WAYLIVRA

Substantial cash position of $2 billion enabling investment in commercial products and pipeline

During the first half of 2018, Ionis received more than $1.2 billion in payments from partners, including $1 billion from Ionis’ expanded collaboration with Biogen

"Our strong financial results were driven by a more than three-fold increase in commercial revenue from SPINRAZA compared to last year. Looking ahead to the second half of this year, we expect to continue to strengthen our financial performance as we add product sales from TEGSEDI and potentially WAYLIVRA to our growing SPINRAZA royalties. We also have the potential to earn numerous milestone payments from our partnered programs. In addition, we will have two full quarters of amortization from our expanded Biogen collaboration, providing further revenue growth," said Elizabeth L. Hougen, chief financial officer of Ionis. "We are on track to achieve our third consecutive year of pro forma operating income even as we prepare to launch two new drugs this year. In addition, we expect to end 2018 with more than $1.8 billion in cash."

All pro forma amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of pro forma and GAAP measures, which is provided later in this release. Additionally, Ionis has labeled its prior period financial statements "as revised" to reflect the new revenue recognition accounting standard the Company adopted on January 1, 2018.

Business Highlights

TEGSEDI (inotersen) – approved in the EU for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR)

On track for post-summer launch in the EU

On track for approval and launch in the U.S. in 2018

License agreement with PTC Therapeutics accelerates access to TEGSEDI in Latin America

Results from the TEGSEDI pivotal study published in the New England Journal of Medicine

Akcea’s commercial organization staffed; patient support program and supply chain in place

WAYLIVRA (volanesorsen) – potential first treatment for people with FCS

U.S. FDA Division of Metabolism and Endocrinology Products Advisory Committee voted in favor of approving WAYLIVRA

On track for approval and launch in the U.S. and EU in 2018

License agreement with PTC Therapeutics accelerates access to WAYLIVRA in Latin America

Akcea’s commercial organization staffed; patient support program and supply chain in place

SPINRAZA – the first and only approved treatment for people with spinal muscular atrophy

SPINRAZA, commercialized by Biogen, continues to generate growth, with global sales of $423 million in the second quarter of 2018, a 250 percent increase from the second quarter of 2017

10 percent of adults with SMA in the U.S. are currently on SPINRAZA treatment, a 20 percent increase from last quarter. Adult patients represent 60 percent of the U.S. SMA patient population

More than 5,000 people with SMA are now on SPINRAZA, representing a 28 percent increase from last quarter

Access outside the U.S. is expanding with reimbursement in 24 countries; Biogen expects reimbursement in at least four more countries by the end of 2018

Pipeline Progress

European Union granted PRIME designation to IONIS-HTTRx (RG6042), potentially providing accelerated assessment for the treatment of people with Huntington’s disease

European Medicines Agency granted Orphan Drug Designation to IONIS-MAPTRx for the treatment of people with frontotemporal dementia

Ionis earned a $7.5 million milestone payment when the FDA approved Achaogen’s ZEMDRI (plazomicin) for the treatment of people with complicated urinary tract infections.

Key Upcoming Events

TEGSEDI EU launch

TEGSEDI U.S. approval and launch

WAYLIVRA U.S. and EU approval and launch

Pivotal study of IONIS-HTTRx in patients with Huntington’s disease initiation by Roche

Results from a Phase 2 clinical study of AKCEA-APO(a)-LRx in patients with high Lp(a) and cardiovascular disease

Results from a Phase 1/2 study of IONIS-SOD1Rx in patients with ALS and mutations in SOD1

Phase 1 clinical study of IONIS-AZ4-2.5-LRx, Ionis’ first Generation 2.5 LICA drug to enter clinical development

The increase in revenue in the first half of 2018 compared to the same period in 2017 was primarily due to the increase in commercial revenue from SPINRAZA royalties, which increased over 250%. Revenue from the amortization of upfront payments increased due to Ionis’ expanded strategic neurology collaboration with Biogen. Ionis expects that the quarterly amortization from this collaboration will be nearly $14 million beginning in the third quarter. In the second quarter and year-to-date 2017, revenue included the $50 million milestone payment from Biogen for SPINRAZA approval in the EU. License fees in the first half of 2018 were $63 million, primarily from AstraZeneca for the license of IONIS-AZ5-2.5Rx and IONIS-AZ6-2.5-LRx compared to $65 million in 2017, primarily from Bayer for the license of IONIS-FXI-LRx.

Operating Expenses

Operating expenses for the three and six months ended June 30, 2018 on a GAAP basis were $168.0 million and $315.7 million, respectively, and on a pro forma basis were $134.2 million and $253.4 million, respectively. These amounts compare to GAAP operating expenses for the three and six months ended June 30, 2017 of $105.8 million and $202.1 million, respectively, and pro forma operating expenses of $84.6 million and $160.0 million, respectively. Operating expenses increased in the first half of 2018, compared to the same period in 2017, principally due to higher SG&A expenses as Akcea, Ionis’ commercial affiliate, prepares to commercialize TEGSEDI and WAYLIVRA. The Company’s SG&A expenses also increased in the first half of 2018 compared to the first half of 2017 due to an increase in fees the Company owed under its in-licensing agreements related to SPINRAZA. R&D expenses accounted for a smaller portion of the increase in operating expenses. R&D expenses increased primarily from medical affairs expenses related to the planned launch of TEGSEDI and WAYLIVRA.
Net Income (Loss)

Ionis reported a net loss of $56.6 million and $67.4 million for the three and six months ended June 30, 2018, respectively, compared to a net loss of $3.1 million and net income of $5.9 million for the same periods in 2017, all according to GAAP. On a pro forma basis, Ionis reported a net loss of $22.7 million and $5.1 million for the three and six months ended June 30, 2018, respectively, compared to net income of $18.2 million and $48.0 million for the same periods in 2017. Ionis’ GAAP net loss increased in the first half of 2018 primarily due to increased operating expenses related to the commercialization of TEGSEDI and WAYLIVRA.

Net Loss Attributable to Noncontrolling Interest in Akcea Therapeutics, Inc.

From the closing of Akcea’s IPO in July 2017 through mid-April 2018, Ionis owned 68 percent of Akcea. In April 2018, Ionis received an additional 18.7 million shares of Akcea’s stock from the license of TEGSEDI and AKCEA-TTR-LRx to Akcea, increasing Ionis’ ownership percentage to approximately 75 percent. Ionis’ second quarter and year-to-date 2018 financial results reflect this increased ownership. The shares held by third parties represent an interest in Akcea’s equity that Ionis does not control. However, because Ionis continues to maintain overall control of Akcea, Ionis reflects the assets, liabilities and results of operations of Akcea in Ionis’ consolidated financial statements. Ionis reflects the noncontrolling interest attributable to other holders of Akcea’s common stock in a separate line called "Net loss attributable to noncontrolling interest in Akcea" on Ionis’ statement of operations. Ionis’ net loss attributable to noncontrolling interest in Akcea for the three and six months ended June 30, 2018, was $16.2 million and $25.6 million, respectively. Ionis also added a corresponding account in its stockholders’ equity section on its balance sheet called "Noncontrolling interest in Akcea Therapeutics, Inc."

Net Income (Loss) Attributable to Ionis Common Stockholders

Ionis reported a net loss attributable to Ionis’ common stockholders of $40.4 million and $41.8 million for the three and six months ended June 30, 2018, respectively, compared to a net loss of $3.1 million and net income of $5.9 million for the same periods in 2017. For the three months ended June 30, 2018 and 2017, basic and diluted net loss per share were $0.29 and $0.02, respectively. For the six months ended June 30, 2018, basic and diluted net loss per share were each $0.30. For the six months ended June 30, 2017, basic and diluted net income per share were each $0.05. All amounts are on a GAAP basis.

Balance Sheet

As of June 30, 2018, Ionis had cash, cash equivalents and short-term investments of $2.0 billion compared to $1.0 billion at December 31, 2017. During the first half of 2018, Ionis received over $1.2 billion in payments from its partners, primarily from Biogen.

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 August 7, 2018 Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR), reported its financial results for the quarter ended June 30, 2018 and provided an update on the Company’s recent achievements (Press release, Eidos Therapeutics, AUG 7, 2018, View Source [SID1234576275]).

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"With the proceeds from our IPO and Series B financing, we are well positioned to continue the momentum of developing AG10 as a disease-modifying therapy for ATTR," said Neil Kumar, PhD, chief executive officer of Eidos. "Specifically, we plan to complete our ongoing Phase 2 trial in ATTR-CM patients by the end of 2018 and initiate Phase 3 studies in ATTR-CM and ATTR-PN patients in 2019."

Recent Achievements and Upcoming Milestones

Initiated and completed enrollment in the ongoing Phase 2 trial in ATTR-CM patients.
Completed Series B preferred stock financing raising $64 million.
Completed initial public offering, with total gross proceeds of $122.2 million including exercise of underwriters’ option to purchase additional shares, from the sale of 7.2 million shares of common stock.
Complete data from Phase 1 study of AG10 in healthy volunteers to be presented at poster presentation at Heart Failure Society of America 22nd Annual Scientific Meeting (September 15-18).
Top-line results from ongoing Phase 2 study of AG10 in symptomatic ATTR-CM patients to be announced by the end of 2018.
Financial Results for the Second Quarter 2018

Cash and cash equivalents totaled $176.7 million at June 30, 2018 compared with $5.5 million at December 31, 2017, reflecting the $112.2 million of net proceeds from our initial public offering in June 2018 and $64.0 million related to the Series B preferred stock financing.

Research and development expenses were $7.4 million for the second quarter of 2018, compared to $1.3 million for the same period of 2017, an increase of $6.1 million. The increase was primarily due to increased expenses for contract consultants, contract manufacturing and other activities for AG10 clinical trials and increases in headcount and related salaries and expenses.

General and administrative expenses were $1.9 million for the second quarter of 2018 compared to $0.5 million for the same period in 2017, an increase of $1.4 million. The increase was primarily due to increased salaries and employee-related expenses and increases in professional fees and services in connection with becoming a public company.

Net loss for the quarter ended June 30, 2018 was $10.6 million or $1.38 per common share, compared to a net loss of $1.7 million or $0.49 per common share for the same period in 2017.

About AG10

AG10 is an orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to amyloidosis, or ATTR. AG10 is currently being examined in a Phase 2 clinical trial in patients with ATTR cardiomyopathy. Top-line results from this trial are expected to be reported by the end of 2018.

AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a "rescue mutation" because it has been shown to prevent ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the "super-stabilizing" properties of this rescue mutation.