Array BioPharma to Report Financial Results for the Fourth Quarter and Full Year of Fiscal 2018 on August 14, 2018

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

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Ionis Reports Second Quarter 2018 Financial Results

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.

ADC Therapeutics Doses First Patient in Pivotal Clinical Trial of ADCT-402 in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma

On August 7, 2018 ADC Therapeutics, an oncology drug discovery and development company that specializes in the development of proprietary antibody drug conjugates (ADCs), reported that the first patient has been dosed in its Phase II clinical trial intended to support the submission of Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) (Press release, ADC Therapeutics, AUG 7, 2018, View Source [SID1234596075]). The clinical trial is evaluating the efficacy and safety of ADCT-402 (loncastuximab tesirine) in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

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At the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, the Company presented interim Phase I data on ADCT-402 in 138 evaluable, heavily pre-treated lymphoma patients who had failed, or were intolerant to, any established therapy known to provide clinical benefit, with a median of three prior therapies. At the time, for the 49 response-evaluable patients in Part 1 of the study (dose escalation) with DLBCL who received ADCT-402 at doses greater than or equal to 120 μg/kg, the overall response rate (ORR) was 55 percent (27/49), with 18 patients achieving a complete response (37 percent) and 9 patients achieving a partial response (18 percent).

The primary endpoint of the Phase II, multi-center, open-label, single-arm trial is the ORR in patients treated with ADCT-402, as confirmed by central review. Secondary endpoints include assessments of duration of response, complete response rate, relapse-free survival, progression-free survival and overall survival, as well as safety, pharmacokinetics and health-related quality of life. The trial will enroll approximately 140 patients with relapsed or refractory DLBCL at multiple centers in the USA and Europe.

"We are pleased to have dosed the first patient in our registrational Phase II clinical trial evaluating ADCT-402 in patients with DLBCL who have relapsed and have refractory disease after two or more multi-agent treatment regimens. Our Phase I clinical trial of ADCT-402 in non-Hodgkin lymphoma showed significant activity in patients with DLBCL and an acceptable safety profile," said Jay Feingold, MD, PhD, Chief Medical Officer and Senior Vice President of Clinical Development at ADC Therapeutics. "Unfortunately, there is no effective treatment for patients with multiple relapsed and refractory DLBCL, so we are excited about the potential to improve outcomes in these patients with ADCT-402 in a single-arm trial. We anticipate reporting results from the Phase II trial in the third quarter of 2019 and are hopeful that the data will support our submission of a BLA to the FDA."

Alex Spira, MD, PhD, FACP, Director of Virginia Cancer Specialists Research Institute and Clinical Assistant Professor of Oncology at Johns Hopkins School of Medicine, added, "Patients with DLBCL who have relapsed or are refractory after second-line chemotherapy face a very poor prognosis. There is a

significant unmet need for an effective new treatment option for this patient population, and we believe ADCT-402 has the potential to help impact patient outcomes in this disease."

For more information about the Phase II clinical trial, please visit www.clinicaltrials.gov (identifier NCT03589469).

ADC Therapeutics also plans to initiate multiple combination studies with ADCT-402 in the fourth quarter of 2018.

About Diffuse Large B-Cell Lymphoma (DLBCL)

Non-Hodgkin lymphoma (NHL) is the seventh most common type of cancer in the U.S., and accounted for an estimated 4.3 percent of new cancer cases in 2017.1 Diffuse large B-cell lymphoma (DLBCL) accounts for nearly one-third (32.5 percent) of NHL.2 The most common initial treatment for patients with DLBCL is chemo-immunotherapy. Response to initial treatment is high, but more than half of patients do not have long-term disease control.3 The current standard of care for relapsed DLBCL is additional chemotherapy, which can be followed by stem cell transplantation (SCT). The prognosis for relapsed patients is poor, especially for those with chemotherapy-refractory disease with a short interval between remission and relapse or those who relapse after high-dose therapy and SCT. There is a significant unmet need for an effective treatment for patients with relapsed or refractory DLBCL.

About ADCT-402

ADCT-402 is an antibody drug conjugate (ADC) composed of a humanized monoclonal antibody that binds to human CD19, conjugated through a linker to a pyrrolobenzodiazepine (PBD) dimer toxin. Once bound to a CD19-expressing cell, ADCT-402 is internalized into the cell where enzymes release the PBD-based warhead. CD19 is a clinically validated target for the treatment of B-cell malignancies. The PBD-based warhead has the ability to form highly cytotoxic DNA interstrand cross-links, blocking cell division and resulting in cell death. Preliminary data from a Phase I clinical trial in relapsed or refractory B-cell non-Hodgkin lymphoma demonstrate ADCT-402 has significant activity in patients with diffuse large B-cell lymphoma (DLBCL). ADCT-402 is also being evaluated in an ongoing Phase I clinical trial in patients with relapsed or refractory B-cell lineage acute lymphoblastic leukemia (B-ALL). The U.S. Food and Drug Administration has granted orphan drug designation to ADCT-402 for the treatment of DLBCL and mantle cell lymphoma.

GT BIOPHARMA ANNOUNCES COMPLETION OF $5.1 MILLION CONVERTIBLE DEBT FINANCING

On August 7, 2018 GT Biopharma Inc. (OTCQB: GTBP; Euronext Paris: GTBP) an immuno-oncology biotechnology company focused on innovative treatments based on the company’s proprietary platforms, reported that on August 2, 2018 it completed a private placement of convertible debentures for gross proceeds of $5,140,000 (Press release, GT Biopharma , AUG 7, 2018, View Source [SID1234539526]).

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”It is a testament to the potential of our immuno-oncology platforms and the promise for patients that our well informed and successful financing partners continue to support GT Biopharma as we move this company to the next level. Meeting our capital requirements is just one of many critical steps to advancing our drug development programs,” said Dr. Raymond W. Urbanski, Chairman and Chief Executive Officer of GT Biopharma.

The interest rate on the convertible debentures is 10%. The convertible debentures are convertible into the Company’s common stock, par value $0.001 per share, at an initial exercise price of $2 per share, subject to adjustment. The convertible debentures mature after one year.

Arrowhead Pharmaceuticals Reports Fiscal 2018 Third Quarter Results

On August 7, 2018 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2018 third quarter ended June 30, 2018. The company is hosting a conference call at 4:30 p.m. EDT to discuss results (Press release, Arrowhead Pharmaceuticals, AUG 7, 2018, View Source [SID1234528496]).

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 8452059.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 8452059.

Selected Fiscal 2018 Third Quarter and Recent Events

Made presentations at the EASL International Liver Congress, including:
Preclinical data on ARO-AAT, the second generation candidate for the treatment of alpha-1 antitrypsin deficiency liver disease
Preclinical data on ARO-HBV, the third generation clinical candidate for the treatment of chronic hepatitis B virus infection
Clinical data on ARC-520, a prior generation compound for HBV
Presented preclinical data at various medical meetings on the growing pipeline, including data on two cardiometabolic candidates ARO-APOC3 and ARO-ANG3 and the first candidate targeting the lung, ARO-ENaC
Completed enrollment and dosing of the single ascending dose portion of the ongoing Phase 1/2 study of ARO-HBV and began dosing HBV patients in the multiple ascending dose portion of the study
Completed enrollment of the Phase 1 study of ARO-AAT
Received a positive EMA opinion on orphan designation for ARO-AAT, this follows orphan drug designation that was previously granted by the US FDA
Presented initial clinical data on ARO-AAT at the Alpha-1 National Education Conference, representing this was the first clinical data presented on the proprietary Targeted RNAi Molecule (TRiMTM) platform
Announced that Amgen had administered the first dose of AMG 890, formerly ARO-LPA, in a Phase 1 clinical study, which earned Arrowhead a $10 million milestone payment