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 Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL) reported its second quarter 2018 financial results and described recent clinical and corporate accomplishments including (Press release, Synta Pharmaceuticals, AUG 7, 2018, View Source [SID1234528529]):

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· Positive Phase 2 Clinical Results – MGL-3196 demonstrated highly statistically significant results for the key 36-week endpoints in its Phase 2 clinical trial in non-alcoholic steatohepatitis (NASH), including statistically significant reductions of liver fat and resolution of NASH

· Madrigal’s abstract "In a Placebo-Controlled 36-Week Phase 2 Trial, Treatment with MGL-3196 Compared to Placebo Results in Significant Reductions in Hepatic Fat (MRI-PDFF), Liver Enzymes, Fibrosis Biomarkers, Atherogenic Lipids, and Improvement in NASH on Serial Liver Biopsy" has been selected for oral presentation at The Liver Meeting 2018 in San Francisco. The presentation will take place on Monday, November 12, 2018, at 8:15 AM during the Presidential Plenary — Clinical Science

· Increased Capital Position — Madrigal completed an underwritten registered public offering in which the gross proceeds to Madrigal from the offering, before deducting the underwriting discounts and commissions and other estimated offering expenses, were approximately $329 million

· Progress in out-licensed legacy oncology programs — Madrigal’s licensee for its Hsp90 inhibitor program, Aldeyra Therapeutics, Inc., recently announced that, ADX-1612, its lead Hsp90 compound, is being advanced in multiple indications: mesothelioma, ovarian cancer and lymphoproliferative immune disease

"We believe the 36-week data from our recently completed Phase 2 clinical study of MGL-3196 in patients with NASH suggest a high likelihood of success in a similarly designed Phase 3 study, for which we are actively preparing, pending regulatory agreement," stated Paul Friedman, M.D., Chief Executive Officer of Madrigal. "With our financial raise completed, which provided more than $300 million of additional capital, we are in a strong position to expedite the MGL-3196 development program in NASH and dyslipidemias."

Becky Taub, M.D., CMO and Executive VP, Research & Development of Madrigal added, "We believe the totality of data from our clinical and preclinical studies to date, including the consistency of the various parameters related to clinical benefits and safety, demonstrate the potential of MGL-3196 to resolve NASH and improve multiple atherogenic lipids. We are pleased our abstract was selected by AASLD as an oral presentation in the presidential plenary clinical session and look forward to presenting these encouraging clinical outcomes in November at The Liver Meeting 2018."

Financial Results for the Three Months and Six Months Ended June 30, 2018

As of June 30, 2018, Madrigal had cash, cash equivalents and marketable securities of $490.3 million, compared to $191.5 million at December 31, 2017. The increase in cash and marketable securities resulted primarily from the net proceeds of $311.8 million from Madrigal’s public offering of common stock in June 2018, partially offset by cash used in operations of $14.0 million.

Operating expenses were $7.8 million and $14.9 million, respectively, for the three month and six month periods ended June 30, 2018, compared to $8.4 million and $14.5 million in the comparable prior year periods.

Research and development expenses for the three month and six month periods ended June 30, 2018 were $5.1 million and $10.3 million, respectively, as compared to $6.8 million and $11.2 million in the comparable prior year periods. The decreases are primarily attributable to completion of treatment in our Phase 2 clinical studies in 2018.

General and administrative expenses for the three month and six month periods ended June 30, 2018 were $2.7 million and $4.6 million, respectively, as compared to $1.6 million and $3.3 million in the comparable prior year periods. The increases are due primarily to higher non-cash stock compensation expense from stock option awards.

Interest income for the three month and six month periods ended June 30, 2018 was $1.2 million and $1.9 million, respectively, as compared to $92 thousand and $168 thousand in the comparable prior year periods. The change in interest income was due primarily to a higher average principal balance in our investment account in 2018.

Out-Licensed Legacy Oncology Programs

Madrigal’s licensee for its Hsp90 inhibitor program, Aldeyra Therapeutics, Inc., provided a pipeline update during its June 2018 Research Day. ADX-1612 is a novel Hsp90 inhibitor in development for the treatment of post-transplant lymphoproliferative disorder and cancer. Hsp90 is a protein that facilitates cell replication, which is excessive and uncontrolled in certain inflammatory diseases and cancer. ADX-1612 is currently being studied in investigator-sponsored trials for mesothelioma, with clinical results expected in the second half of 2018, and ovarian cancer, with Phase 2 clinical trial initiation expected in the second half of 2018. Aldeyra is further developing ADX-1612 for the

treatment of lymphoproliferative immune disease, with Phase 2 clinical testing expected to start in 2019.

Clinical Program Summaries for MGL-3196

NASH

Non-alcoholic Steatohepatitis (NASH) is a common liver disease in the United States and worldwide, unrelated to alcohol use, that is characterized by a build-up of fat in the liver, inflammation, damage (ballooning) of hepatocytes and increasing fibrosis. Although people with NASH may feel well and often do not know they have the disease, NASH can lead to permanent damage, including cirrhosis and impaired liver function in a high percentage of patients.

In October 2016, the first patient was treated in the ongoing Phase 2 trial of MGL-3196 for the treatment of NASH. The randomized, double-blind, placebo-controlled, multi-center Phase 2 study enrolled 125 patients 18 years of age and older with liver biopsy-confirmed NASH and included approximately 25 clinical sites in the United States. Patients were randomized to receive either MGL-3196 or placebo in a 2:1 ratio.

The primary endpoint of the study was the reduction of liver fat at 12 weeks compared with baseline (relative change), assessed by MRI-PDFF. Key secondary endpoints at 36 weeks included: reduction in liver fat compared with baseline (relative change), also assessed by MRI-PDFF; a two-point reduction in NAS (NALFD activity score) on biopsy; resolution of NASH on biopsy; and, safety and tolerability based on adverse events and changes in laboratory values.

The primary endpoint of the study at 12 weeks was achieved. Liver fat was reduced by 36.3% in all MGL-3196 treated patients (78) and 42.0% in a pre-specified group of high exposure MGL-3196 treated patients (44/78), as compared with 9.6% median reduction in liver fat in 38 placebo treated patients. These results were statistically significant (p<0.0001) for both MGL-3196 treatment groups. Further, 75% of the high-exposure MGL-3196 treated patients showed liver fat reductions of >30%.

At 36 weeks, MGL-3196 achieved multiple key secondary endpoints including a sustained highly significant (p<0.001) reduction in liver fat compared to placebo as measured by MRI-PDFF; mean relative fat reduction for MGL-3196 was 37% versus 8.9% for placebo. MGL-3196 was associated with a greater percentage of subjects with a 2-point improvement in NAS (56% of 73 patients vs 32% of 34 placebo subjects, p=0.02). NASH resolution (NR) was seen in 27% of MGL-3196 compared with 6% of placebo subjects, p=0.02. MGL-3196 patients with > 30% fat reduction on Week 12 MRI-PDFF demonstrated a higher percentage of 2-point improvement in NAS (70%, p=0.001) and NR (39%, p=0.001) compared with placebo, demonstrating a strong relationship between early reduction in liver fat as demonstrated by week 12 MRI-PDFF and NASH improvement on liver biopsy at Week 36. In patients with NASH Resolution,

35% of the MGL-3196 treated patients and no placebo patients had more advanced NASH (baseline NAS >5).

At Week 36, MGL-3196 treated patients showed sustained reduction of fibrosis biomarkers. In MGL-3196 patients with NASH resolution, fibrosis also resolved in 50% of patients and was decreased statistically significantly reduced relative to all placebo patients.

There were statistically significant reductions in liver enzymes in MGL-3196 treated patients compared to placebo treated patients; reductions of greater magnitude were achieved with longer duration of MGL-3196 treatment. Statistically significantly more MGL-3196 treated patients than placebo treated patients had normalization of ALT (alanine transaminase).

Similar to week 12, at week 36 there were sustained, statistically significant reductions in low-density lipoprotein cholesterol (LDL-C), triglycerides, ApoB and lipoprotein(a).

MGL-3196 was well tolerated in this trial with mostly mild and a few moderate AEs which were balanced between drug treated and placebo patients. An increase in incidence of mild transient diarrhea in MGL-3196-treated, often a single episode, at the start of treatment. Diarrhea incidence was not increased later in the study.

Based on liver enzyme inclusion criteria, some patients are receiving extended treatment beyond 36 weeks for up to 36 additional weeks. All patients in this extension study will receive MGL-3196 and only non-invasive assessments will be made, including serial MRI-PDFF, safety labs, and circulating biomarkers.

Additional information about the study [NCT02912260] can be obtained at www.ClinicalTrials.gov.

Heterozygous familial hypercholesterolemia (HeFH), and a much rarer form called homozygous familial hypercholesterolemia (HoFH), are severe genetic dyslipidemias typically caused by inactivating mutations in the LDL receptor. Both forms of FH lead to early onset cardiovascular disease. HeFH, the most common dominantly inherited disease, is present in up to 1 in 200 people; the disease is found in higher frequencies in certain more genetically homogenous populations. Treatments exist for both HeFH and HoFH but many patients (as many as 40 percent of HeFH patients) are not able to reach their cholesterol (LDL-C) reduction goals on these therapies, reflecting the lifetime burden of cholesterol buildup in their bodies. Based on evidence of impressive LDL cholesterol lowering in Phase 1, and data suggesting that MGL-3196 has a mechanism of action that is different from and complementary to statins, Madrigal initiated a Phase 2 proof-of-concept trial in HeFH in February 2017 and enrolled 116 patients.

In this Phase 2 HeFH trial, patients who were not at their LDL-C goal were randomized in a 2:1 ratio to receive either MGL-3196 or placebo, in addition to their current cholesterol lowering regimen, which included approximately 75% taking high intensity statins (20/40 mg rosuvastatin or 80 mg atorvastatin), and about 2/3 of patients also taking ezetimibe. MGL-3196 treated patients (placebo corrected) achieved highly significant (p< 0.0001) LDL-C lowering of 18.8%, and 21% LDL-C lowering in those on an optimal dose of MGL-3196. LDL-C lowering was 28.5% in MGL-3196 treated compared to placebo in a prespecified group of patients who did not tolerate high intensity statin doses. Highly significant reductions (p<0.0001) relative to placebo were also observed with ApoB, triglycerides (TG) (25-31%), apolipoprotein CIII (Apo CIII) and Lp(a) (25-40%) in all MGL-3196 treated patients and prespecified subgroups, irrespective of statin treatment.

MGL-3196 was well-tolerated with primarily mild and some moderate AEs, the numbers of which were balanced between placebo and drug-treatment groups.

About MGL-3196

Among its many functions in the human body, thyroid hormone, through activation of its beta receptor, plays a central role in controlling lipid metabolism, impacting a range of health parameters from levels of serum cholesterol and triglycerides to the pathological buildup of fat in the liver. Attempts to exploit this pathway for therapeutic purposes in cardio-metabolic and liver diseases have been hampered by the lack of selectivity of older compounds for the thyroid hormone receptor (THR)-β, chemically-related toxicities and undesirable distribution in the body.

Madrigal recognized that greater selectivity for thyroid hormone receptor (THR)-β and liver targeting might overcome these challenges and deliver the full therapeutic potential of THR-β agonism. Madrigal believes that MGL-3196 is the first orally administered, small-molecule, liver- directed, truly β-selective THR agonist. MGL- 3196 has now demonstrated in two Phase 2 double-blind, placebo-controlled trials in NASH and HeFH the potential for a broad array of therapeutically beneficial effects, improving components of both metabolic syndrome, such as insulin resistance and dyslipidemia, and fatty liver disease, including lipotoxicity and inflammation. Based on evidence of these pleiotropic actions, coupled with an excellent safety profile, Madrigal plans to initiate a Phase 3 clinical program in NASH and dyslipidemias.

On August 7, 2018 Sunesis Pharmaceuticals, Inc. (Nasdaq: SNSS) reported financial results for the quarter ended June 30, 2018 (Press release, Sunesis, AUG 7, 2018, View Source [SID1234528528]). Loss from operations for the three and six months ended June 30, 2018 was $6.6 million and $13.7 million. As of June 30, 2018, cash, cash equivalents and marketable securities totaled $20.4 million. This capital is expected to fund the company into the first quarter of 2019.

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"During the second quarter, we continued to focus on advancing our Phase 1b/2 trial evaluating our lead program, the non-covalent BTK inhibitor vecabrutinib, to help patients who have developed resistance to covalent BTK inhibitors such as ibrutinib," said Dayton Misfeldt, Interim Chief Executive Officer of Sunesis. "Specifically, we have added clinical sites, including the recent additions of Memorial Sloan Kettering Cancer Center and Moffitt Cancer Center, and strengthened our development team. We also entered into a financing facility with Aspire Capital to provide us with another flexible financing tool to support our development programs. We expect to present an update on our ongoing vecabrutinib trial at the annual American Society of Hematology (ASH) (Free ASH Whitepaper) meeting."

Recent Highlights

Strengthened Senior Management Team. In July 2018, the Company announced three key new management appointments as part of its expanded development team. Deepali Suri, a former Executive Director at Pharmacyclics (an AbbVie Company), was appointed Vice President, Clinical Operations and Sean Gharpurey joined as Executive Director, Project Management. Stephen Nava was promoted to Vice President, Quality Assurance, Compliance and Regulatory Affairs.

Entered into $15.5 Million Common Stock Purchase Agreement with Aspire Capital Fund, LLC. In June 2018, Sunesis entered into a Common Stock Purchase Agreement (CSPA) of up to $15.5 million with Aspire Capital. Under the terms of the Agreement, Aspire made an initial investment via purchase of $500,000 worth of SNSS common shares at a price of $2.19 per common share. In addition, Aspire committed to purchasing up to an additional $15 million of common shares of Sunesis at Sunesis’ request from time to time during a 24-month period, at prices based on the market price at the time of each sale. As consideration for Aspire’s obligations under the CSPA, Sunesis also issued 212,329 shares of common stock to Aspire as a commitment fee.

Presented Pre-clinical Data Demonstrating Activity of Vecabrutinib at EHA (Free EHA Whitepaper) Annual Meeting. In June, the laboratory of Professor Gilles Salles at the Université Claude Bernard de Lyon presented preclinical validation of vecabrutinib activity at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Stockholm, Sweden. The data demonstrated the activity of Sunesis’ non-covalent BTK inhibitor vecabrutinib in BTK-dependent lymphomas, including lymphoma cell lines overexpressing mutated BTK C481S. In addition, a Sunesis-supported study led by Professor Paolo Ghia for the European Research Initiative on CLL (ERIC) assessed the

real-world prevalence of BTK C481 and PLCγ2 mutations in CLL patients relapsing under ibrutinib. Approximately half of the relapsed patients had BTK C481S mutations.

Financial Highlights

Cash, cash equivalents, and marketable securities totaled $20.4 million as of June 30, 2018, as compared to $31.8 million as of December 31, 2017. This capital is expected to fund the company into the first quarter of 2019. The 6-month decrease of $11.4 million was primarily due to $12.4 million of net cash used in operating activities, partially offset by $1.1 million in net cash flows from financing activities.

Net cash flows from financing activities comprised $0.8 million in proceeds from the issuance of common stock and $0.3 million in proceeds from stock option exercises and ESPP stock purchases.

Subsequent to June 30, 2018, the Company raised an additional $2.6 million in net cash proceeds from a combination of the Aspire CSPA and the Cantor Fitzgerald Controlled Equity Offering facility.

Research and development expense was $3.8 million and $7.7 million for the three and six months ended June 30, 2018, as compared to $4.9 million and $11.1 million for the same periods in 2017, primarily relating to the vecabrutinib and the vosaroxin development program in each period. The decreases of $1.1 million and $3.4 million between the comparable periods from last year was primarily due to decreases in salary and personnel expenses due to lower headcount, and decrease in professional services and clinical trials expenses related to higher expenses incurred in the second quarter of 2017 due to the MAA with the EMA.

General and administrative expense was $2.8 million and $6.2 million for the three and six months ended June 30, 2018, as compared to $3.7 million and $7.6 million for the same periods in 2017. The decreases of $0.9 million and $1.4 million between the comparable periods in 2017 were primarily due to reduced personnel and commercial expenses.

Interest expense was $0.3 million and $0.6 million for the three and six months ended June 30, 2018, as compared to $0.3 million and $0.8 million for the same periods in 2017. The decreases were primarily due to the decrease in the outstanding notes payable.

Cash used in operating activities was $12.4 million for the six months ended June 30, 2018, as compared to $20.5 million for the same period in 2017. Net cash used in the 2018 period resulted primarily from the net loss of $14.1 million, partly offset by net adjustments for non-cash items of $1.6 million and changes in operating assets and liabilities of $0.1 million. Net cash used in the six months ended June 30, 2017, resulted primarily from the net loss of $18.7 million and changes in operating assets and liabilities of $3.9 million, partly offset by net adjustments for non-cash items of $2.1 million.

Sunesis reported loss from operations of $6.6 million and $13.7 million for the three and six months ended June 30, 2018, as compared to $8.6 million and $18.0 million for the same periods in 2017. Net loss was $6.8 million and $14.1 million for the three and six months ended June 30, 2018, as compared to $8.8 million and $18.7 million for the same periods in 2017.

Conference Call Information

Sunesis will host a conference today at 4:30 p.m. Eastern Time. The call can be accessed by dialing (844) 296-7720 (U.S. and Canada) or (574) 990-1148 (international) and entering passcode 5198125. To access the live audio webcast, or the subsequent archived recording, visit the "Investors and Media – Calendar of Events" section of the Sunesis website at www.sunesis.com. The webcast will be recorded and available for replay on the company’s website for two weeks.

On August 7, 2018 Protagonist Therapeutics, Inc. (Nasdaq:PTGX) reported its financial results for the second quarter ended June 30, 2018 (Press release, Protagonist, AUG 7, 2018, View Source;p=RssLanding&cat=news&id=2362550 [SID1234528527]).

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"Protagonist continues to advance well-differentiated therapeutic candidates discovered through its novel peptide technology platform," commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "We very recently reported results from a comprehensive review of the data from the Phase 2 PROPEL study of PTG-100 for the treatment of moderate to severe active ulcerative colitis. We are pleased to conclude that this drug candidate showed signals of clinical efficacy, has no safety concerns, and warrants further clinical development for potential treatment of ulcerative colitis. We are planning to discuss next steps in the clinical development of PTG-100 with the U.S. Food and Drug Administration (FDA) and other global regulatory authorities in the second half of this year. We are glad to report on the continued progress of the clinical development of PTG-200 in collaboration with our partner Janssen for potential treatment of Crohn’s disease. In addition, we look forward to initiating an open label Phase 2 study in beta-thalassemia patients in the fourth quarter of 2018 with PTG-300, our product candidate for rare blood disorders."

Product Development Update:

PTG-100

In March 2018, Protagonist had announced discontinuation of the Phase 2 PROPEL study following a planned interim analysis conducted by an independent Data Monitoring Committee. The interim data had revealed an unusually high placebo rate of clinical remission (24 percent, approximately four times higher than historical norms for similar UC studies) that led to a futility decision and discontinuation of the trial. A re-read of the endoscopies by the subcontractor of the contract research organization (CRO) and a subsequent fully blinded re-read of the endoscopies by an independent third party, Robarts Clinical Trials, confirmed that a subset of the initial endoscopy reads provided by the CRO were in error. If the re-read of endoscopy results had been utilized for the interim futility analysis, the trial would have continued. Based on this entire analysis, the Company plans to discuss the next steps in advancing the clinical development of PTG-100 with the FDA and other global regulatory authorities in the second half of 2018.
PTG-200

In the fourth quarter of 2018, Protagonist expects a U.S. IND filing by its partner Janssen Biotech that will support the initiation of a global Phase 2 study of PTG-200, an oral, gut-restricted interleukin-23 receptor antagonist peptide in Crohn’s disease patients. This IND filing would trigger a milestone payment from Janssen of $25 million under the existing exclusive license and collaboration agreement between Janssen and Protagonist.
PTG-300

The results of a Phase 1 study in healthy volunteers and supportive pre-clinical data for PTG-300, an injectable hepcidin mimetic peptide, were the subject of oral presentations in June 2018 at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).
Protagonist completed discussions with U.S. and global regulatory agencies and filed a U.S. IND in the second quarter of 2018. The Company plans other global regulatory submissions to support the initiation of a global Phase 2 trial in patients with beta-thalassemia in the fourth quarter of 2018.
Preclinical Programs

The Company presented data related to preclinical product candidates, oral peptide agonists targeting the delta/mu opioid receptors, in a podium presentation at the Digestive Diseases Week conference in June 2018.
Corporate Update – Financing:

Protagonist recently announced the completion of an equity financing with investors including BVF Partners L.P. and their affiliates for gross proceeds of $22 million. Proceeds from the financing will be used to advance development of drug candidate PTG-100.
Financial Results

Protagonist reported a net loss of $8.7 million and $16.3 million, respectively, for the second quarter and first six months of 2018, as compared to a net loss of $15.0 million and $29.1 million, respectively, for the same periods of 2017. The decrease in net loss for both the second quarter and year-to-date periods was driven primarily by license and collaboration revenue recognized during the first and second quarters of 2018, partially offset by increased research and development (R&D) and general and administrative (G&A) expenses. The net loss for the second quarter and first six months of 2018 includes non-cash stock-based compensation of $1.6 million and $2.8 million, respectively, as compared to $1.0 million and $1.8 million, respectively, for the same periods of 2017.

License and collaboration revenue was $11.7 million and $22.5 million for the second quarter and first six months of 2018, respectively, and consisted of revenue from activities performed under the Janssen Collaboration Agreement. Protagonist did not recognize any license and collaboration revenue for the second quarter or first six months of 2017.

R&D expenses for the second quarter and first six months of 2018 were $17.7 million and $33.1 million, respectively, as compared to $12.0 million and $23.3 million, respectively, for the same periods of 2017. The increases in R&D expenses were primarily due to costs related to contract manufacturing and the preparation for and conduct of clinical trials for our product candidates. R&D expenses for the quarter also included an increase in salaries and employee-related expenses due to an increase in R&D personnel.

G&A expenses for the second quarter and first six months of 2018 were $3.2 million and $6.8 million, respectively, as compared to $3.1 million and $6.1 million, respectively, for the same periods of 2017. The increases in G&A expenses were primarily due to increases in salaries and employee-related expenses primarily due to an increase in headcount to support the growth of our operations, partially offset by a decrease in legal expenses.

Protagonist ended the second quarter with $125.2 million in cash, cash equivalents and investments. With the financing announced yesterday, the company expects to have sufficient financial resources to fund operations to mid-2020.

On August 7, 2018 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health" or the "Company" or "we") reported its second-quarter 2018 financial results (Press release, Valeant, AUG 7, 2018, View Source [SID1234528518]).

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"For a second consecutive quarter, the Company delivered overall organic growth2, driven by solid results in our Salix and Bausch + Lomb/International segments, which together represented approximately 78% of our business in the quarter," said Joseph C. Papa, chairman and CEO, Bausch Health. "Growth in the Salix segment reflects higher sales of our promoted brands, most notably XIFAXAN and RELISTOR, while organic growth2 in the Bausch + Lomb/International segment was primarily due to volume increases and strong growth across Europe and China."

"Due to our strong results in the quarter, we are raising our full-year Adjusted EBITDA (non-GAAP) guidance range, and despite significant foreign exchange headwinds, we are maintaining our full-year revenue guidance range," continued Mr. Papa.

Company Highlights

Executing on Core Businesses and Advancing Pipeline

Completed Company’s name change to Bausch Health Companies Inc.
Realigned into four reporting segments3 (Bausch + Lomb/International, Salix, Ortho Dermatologics and Diversified Products) as part of the Company’s ongoing transformation; this provides greater clarity into the performance of each of our businesses
Reported revenue in the Bausch + Lomb/International segment decreased by 1% compared to the second quarter of 2017 primarily due to divestitures and discontinuations; revenue in this segment grew organically2 by 4% compared to the second quarter of 2017, driven primarily by volume increases and strong growth in Europe and China
Successfully launched LUMIFY, which achieved a weekly market share of 21% in the Redness Reliever category4
Launched Soothe Xtra Protection Preservative Free lubricant eye drops
Launched Ocuvite Blue Light eye vitamins, a nutritional supplement that helps protect eyes from blue light emitted from digital devices
Launched PreserVision AREDS 2 Formula Chewable eye vitamins
The U.S. Food and Drug Administration accepted the New Drug Application for loteprednol etabonate ophthalmic gel, 0.38% with a PDUFA action date of Feb. 25, 2019
Grew revenue in the Salix segment by 14% compared to the second quarter of 2017
XIFAXAN revenue increased by 26% compared to the second quarter of 2017
RELISTOR franchise revenue increased by 43% compared to the second quarter of 2017
APRISO revenue increased by 3% compared to the second quarter of 2017
UCERIS franchise revenue increased by 3% compared to the second quarter of 2017
Entered into an exclusive agreement with US WorldMeds to co-promote LUCEMYRA, the first and only non-opioid medication for the mitigation of withdrawal symptoms to facilitate abrupt discontinuation of opioids in adults, and have now launched the product
Continued to stabilize the Ortho Dermatologics segment
Revenue in the Global Solta business increased by 14% compared to the second quarter of 2017
Addressing Debt and Extending Maturities

Refinanced Company’s credit facility
Extended the maturity date of the revolving credit facility to June 1, 2023
Replaced previous Term B loans with new Term B loans that have a maturity date of June 1, 2025
Modified facility covenants to provide the Company with enhanced operating flexibility
Lowered interest rates applicable to the credit facility
Issued $750 million aggregate principal amount of 8.500% unsecured Senior Notes due 2027
On June 1, 2018, a portion of the net proceeds from the new Term B loans and the 8.500% unsecured Senior Notes due 2027, along with cash on hand, were put on deposit with a trustee and subsequently used to redeem, on July 2, 2018, approximately $2 billion of unsecured Senior Notes, including all remaining unsecured Senior Notes due in 2020, and to pay related fees and expenses
Resolving Legal Issues

Achieved dismissals or other positive outcomes in resolving litigation, disputes and investigations in approximately 40 matters since Jan. 1, 2018
The Company continues to achieve positive resolutions in the Shower to Shower cases, with legal fees and costs being reimbursed by Johnson & Johnson
Second-Quarter 2018 Revenue Performance

Total reported revenues were $2.128 billion for the second quarter of 2018, as compared to $2.233 billion in the second quarter of 2017, a decrease of $105 million, or 5%. Excluding the impact of the 2017 divestitures and discontinuations of $183 million and the favorable impact of foreign exchange of $25 million, revenue grew organically2 by 3% compared to the second quarter of 2017, primarily driven by growth in the Salix segment and organic growth2 in the Bausch + Lomb/International segment. Organic revenue growth2 was partially offset by declines in the Ortho Dermatologics segment and lower volumes in the Diversified Products segment, attributed to the previously reported loss of exclusivity for a basket of products.

Bausch + Lomb/International segment revenues were $1.209 billion for the second quarter of 2018, as compared to $1.223 billion for the second quarter of 2017, a decrease of $14 million, or 1%. Excluding the impact of divestitures and discontinuations of $84 million, and the favorable impact of foreign exchange of $25 million, the Bausch + Lomb/International segment grew organically2 by approximately 4% compared to the second quarter of 2017. Organic growth2 in the quarter was primarily driven by volume increases.

Salix Segment

Salix segment revenues were $441 million for the second quarter of 2018, as compared to $387 million for the second quarter of 2017, an increase of $54 million, or 14%. Growth was largely driven by higher sales of XIFAXAN, RELISTOR and other promoted products across the segment.

Ortho Dermatologics Segment

Ortho Dermatologics segment revenues were $142 million for the second quarter of 2018, as compared to $162 million for the second quarter of 2017, a decrease of $20 million, or 12%. Compared to the second quarter of 2017, revenues in the Global Solta business grew by 14%.

Diversified Products Segment

Diversified Products segment revenues were $336 million for the second quarter of 2018, as compared to $461 million for the second quarter of 2017, a decrease of $125 million, or 27%. The decline was primarily driven by the impact of the 2017 divestitures and discontinuations of $97 million and by decreases attributed to the previously reported loss of exclusivity for a basket of products.

Operating Loss

Operating loss was $245 million for the second quarter of 2018, as compared to an operating income of $175 million for the second quarter of 2017, a decrease of $420 million. The decrease in operating results for the second quarter of 2018 primarily reflects an asset impairment associated with the loss of exclusivity of a certain product, an increase in amortization of intangible assets and a decrease in product contribution driven by the impact of divestitures and discontinuations.

Net Loss

Net loss for the three months ended June 30, 2018 was $873 million, as compared to net loss of $38 million for the same period in 2017, a decrease of $835 million. The decrease is primarily attributed to an increase in operating loss, as noted above, and an increase in the provision for income taxes of $343 million, which is primarily due to internal tax reorganizational efforts that the Company began in the fourth quarter of 2016 and completed in the third quarter of 2017.

Adjusted net income (non-GAAP) for the second quarter of 2018 was $327 million, as compared to $362 million for the second quarter of 2017, a decrease of 10%.

Operating Cash

The Company delivered $222 million in operating cash in the second quarter of 2018, as compared to $268 million in the second quarter of 2017, a decrease of $46 million. Cash flows in the second quarter of 2018 were negatively affected by approximately $70 million due to divestitures in 2017, $57 million of accelerated interest payments in connection with the refinancings in June 2018 and approximately $50 million in payments for legal settlements. In the second quarter of 2017, operating cash also included $190 million of net payments made in the resolution of the Salix securities class action litigation.

EPS

GAAP Earnings Per Share (EPS) Diluted for the second quarter of 2018 were $(2.49), as compared to $(0.11) for the second quarter of 2017.

Adjusted EBITDA(non-GAAP)

Adjusted EBITDA (non-GAAP) was $868 million for the second quarter of 2018, as compared to $951 million for the second quarter of 2017, a decrease of $83 million. Adjusted EBITDA (non-GAAP) in the second quarter of 2018 reflects the impact of 2017 divestitures of approximately $70 million, transactional foreign exchange and other of $51 million, the impact of the loss of exclusivity of certain products of $59 million and favorable translational foreign exchange of $10 million.

2018 Financial Outlook

Bausch Health has maintained its full-year revenue guidance range for 2018 and has raised its full-year Adjusted EBITDA (non-GAAP) guidance range for 2018:

Full-Year Revenues in the range of $8.15 – $8.35 billion
Full-Year Adjusted EBITDA (non-GAAP) in the range of $3.20 – $3.35 billion from $3.15 – $3.30 billion
Other than with respect to GAAP Revenues, the Company only provides guidance on a non-GAAP basis. The Company does not provide a reconciliation of forward-looking Adjusted EBITDA (non-GAAP) to GAAP net income (loss), due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation. In periods where significant acquisitions or divestitures are not expected, the Company believes it might have a basis for forecasting the GAAP equivalent for certain costs, such as amortization, which would otherwise be treated as non-GAAP to calculate projected GAAP net income (loss). However, because other deductions (such as restructuring, gain or loss on extinguishment of debt and litigation and other matters) used to calculate projected net income (loss) vary dramatically based on actual events, the Company is not able to forecast on a GAAP basis with reasonable certainty all deductions needed in order to provide a GAAP calculation of projected net income (loss) at this time. The amount of these deductions may be material and, therefore, could result in projected GAAP net income (loss) being materially less than projected Adjusted EBITDA (non-GAAP). The guidance provided in this section represents forward-looking information, and actual results may vary materially. Please see the risks and assumptions referred to in the Forward-looking Statements section of this news release.

Additional Highlights

Bausch Health’s cash and cash equivalents were $838 million at June 30, 2018
The Company’s availability under the Revolving Credit Facility was approximately $725 million at June 30, 2018

Conference Call Details

Date: Tuesday, Aug. 7, 2018

Time: 8:00 a.m. EDT

Web cast: View Source

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Replay Passcode: 1378128 (replay available until Oct. 7, 2018)