On August 5, 2020 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the second quarter of 2020 and recent business highlights (Press release, Ionis Pharmaceuticals, AUG 5, 2020, View Source [SID1234562896]).

"The first half of this year was marked by numerous important achievements. Our Phase 3 programs progressed, we advanced our cardio-renal, metabolic and neurological disease franchises and added new medicines to our Ionis-owned pipeline. Together, these catalysts moved us closer to delivering 10 or more marketing applications through 2025, which we expect to result in a number of new commercial medicines. In the second half of this year, we are expanding the reach of our technology in neurological and pulmonary diseases, as studies in sporadic ALS and COPD get underway. We also look forward to proof-of-concept data from additional mid-stage programs," said Brett P. Monia, Ph.D., chief executive officer at Ionis. "Our achievements, together with our significant financial resources, position us to realize my vision for Ionis – to lead in the delivery of transformational medicines for patients around the world."

Second Quarter 2020 Financial Results and Highlights

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On track to achieve financial guidance of being meaningfully profitable this year

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Net income of $8 million on a non-GAAP basis and a net loss of $32 million on a GAAP basis

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Achieved quarter over quarter growth in commercial and R&D revenues

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Commercial revenue from SPINRAZA (nusinersen) royalties increased to $72 million

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Product sales from TEGSEDI (inotersen) and WAYLIVRA (volanesorsen) increased to $16 million

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R&D revenue increased to more than $55 million, including $26 million from Ionis’ neurological disease franchise, $13 million from the oncology franchise and more than $10 million from the cardio-renal franchise

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Cash position of more than $2.3 billion provides substantial financial resources to continue executing on strategic goals

"We ended the second quarter with net income on a non-GAAP basis, an increase compared to our first quarter results. Looking ahead, we are maintaining our 2020 financial guidance and expect revenue and earnings growth in the second half of this year. With our substantial resources, we are well positioned to continue executing on our ambitious agenda and to deliver increasing value near-term and into the future," said Elizabeth L. Hougen, chief financial officer of Ionis.

All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of non-GAAP and GAAP measures, which is provided later in this release.

Commercial Medicine Highlights

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SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages

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$495 million in worldwide sales in the second quarter of this year

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More than 11,000 patients were on SPINRAZA treatment worldwide at the end of the second quarter, including patients across commercial, expanded access and clinical trial settings

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The Phase 4 RESPOND study to evaluate SPINRAZA benefit in patients with a suboptimal clinical response to Zolgensma (onasemnogene abeparvovec) is expected to begin early next year

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The DEVOTE study evaluating a higher dose of SPINRAZA with the potential to deliver even greater efficacy in SMA patients of all ages is progressing

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New clinical data from the NURTURE and SHINE studies, as well as new real-world data, further support SPINRAZA’s durable efficacy and established safety profile across SMA patients of all ages

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TEGSEDI: the only approved at-home subcutaneous therapy for the treatment of hereditary transthyretin amyloidosis (hATTR) with polyneuropathy in adult patients

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Commercially available in 15 countries

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Reimbursement approved in Portugal, Spain, Italy and Austria

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Expanding commercial availability in additional EU countries and in Latin America this year

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WAYLIVRA: the only approved treatment in the EU for adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis

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Launch progressing in Germany, Austria, Greece and through the ATU in France; launching in additional EU countries this year

Filed for marketing approval in Brazil; refiling new drug application for U.S. marketing authorization

Second Quarter 2020 and Recent Pipeline Highlights

Completed enrollment in the global GENERATION HD1 Phase 3 study of tominersen in patients with Huntington’s disease

Progressed multiple neurological disease medicines under Ionis’ broad Biogen collaboration

Published data from the Phase 1/2 study of tofersen in the New England Journal of Medicine

Progressed the IONIS-MAPTRx long-term extension study in patients with Alzheimer’s disease and achieved a $12 million milestone payment

Advanced ION464 into a Phase 1/2 study in patients with multiple system atrophy and achieved an $18 million milestone payment

Advanced medicines for the treatment of cancer and immune-mediated GI disease

Licensed ION736 to AstraZeneca for the treatment of cancer and achieved a $13 million license fee

Initiated a Phase 1 study of ION253 for the treatment of immune-mediated GI disease and achieved a $5 million milestone payment from Janssen
Expanded the Ionis-owned pipeline with the addition of ION363 for the treatment of FUS-ALS

Upcoming Catalysts

Report clinical proof-of-concept results for four or more programs

Present positive Phase 2 results from vupanorsen and AKCEA-APOCIII-LRx

Initiate a Phase 3 study of AKCEA-APOCIII-LRx in patients with FCS

Initiate a registration study of ION363 in patients with FUS-ALS

Initiate a Phase 1/2 study of ION541 in patients with sporadic ALS

Initiate a Phase 2 study of IONIS-ENaC-2.5Rx in patients with chronic obstructive pulmonary disease (COPD)

Initiate a Phase 2 study of IONIS-FXI-LRx in patients with clotting disorders

Initiate a Phase 2 study of IONIS-HBVRx in patients with hepatitis B virus infection

Advance additional new medicines into development

Operating Expenses

Ionis’ operating expenses for the second quarter of 2020 increased compared to the same period in 2019 driven by its investments in advancing its strategic priorities, including advancing the Phase 3 program for AKCEA-TTR-LRx and other medicines in its Ionis-owned pipeline.

Net Loss Attributable to Noncontrolling Interest in Akcea

At June 30, 2020, Ionis owned approximately 76 percent of Akcea. The Company’s net loss attributable to noncontrolling interest in Akcea for the second quarter of 2020 was consistent with the same period last year. The line titled "Net loss attributable to noncontrolling interest in Akcea" on Ionis’ statement of operations reflects the portion of Akcea’s net income or loss attributable to the other owners of Akcea’s common stock.

Net Income (Loss) Attributable to Ionis Common Stockholders

Ionis’ net loss attributable to Ionis’ common stockholders for the second quarter of 2020 was larger compared to the same period in 2019 primarily due to higher revenue and lower operating expenses in the same period last year.

Balance Sheet

Ionis ended June 2020 with cash, cash equivalents and short-term investments of more than $2.3 billion, compared to $2.5 billion at December 31, 2019.

Webcast

Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast to discuss this earnings release and related activities. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address.

On August 5, 2020 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that its Chief Financial Officer, Joseph J. Ferra, will present a corporate overview at the 2020 Wedbush PacGrow Healthcare Virtual Conference. Details are as follows (Press release, Syros Pharmaceuticals, AUG 5, 2020, View Source [SID1234562894]):

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2020 Wedbush PacGrow Healthcare Virtual Conference:
Date: Wednesday, August 12
Time: 8:00 a.m. ET

A live webcast of the presentation will be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

On August 5, 2020 RGENIX, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, reported that it has been selected to present at multiple virtual healthcare investor conferences taking place during the month of August (Press release, Rgenix, AUG 5, 2020, View Source [SID1234562893]).

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These conferences include:

LifeSci Partners Summer Symposium
August 5, 2020 at 11:30 A.M EDT

BTIG Virtual Biotechnology Conference 2020
August 11, 2020 at 3:30 P.M. EDT

2020 Wedbush PacGrow Healthcare Conference
August 12, 2020 at 9:10 A.M. EDT

Canaccord Genuity 40th Annual Growth Conference
August 13, 2020 at 8:00 A.M. EDT

Links to the live and archived versions of these presentations are available on Rgenix’s website within the News section.

On August 5, 2020 Veracyte, Inc. (Nasdaq: VCYT) reported the publication of new data demonstrating that the Percepta classifier significantly reduces invasive procedures in lung cancer diagnosis by classifying nearly 40 percent of patients as "low risk" when bronchoscopy is inconclusive (Press release, Veracyte, AUG 5, 2020, View Source [SID1234562877]). The findings, from a large, prospective clinical study involving 35 centers, also suggest that these results are durable over a one-year follow-up and that the classifier results do not delay time to diagnosis among those patients with lung cancer. The study appears online in CHEST, the journal of the American College of Chest Physicians.

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"Lung nodules are often difficult to diagnose when bronchoscopy results are indeterminate following initial work-up of the patient. As a result, physicians must determine whether to direct patients to further, invasive procedures or just monitor them, at the risk of missing a cancer and delaying necessary treatment," said Giulia C. Kennedy, Ph.D., Veracyte’s chief scientific officer and chief medical officer. "The real-world findings published in CHEST show that use of the Percepta classifier significantly alters physicians’ pre-test clinical plans for an invasive procedure, enabling some patients to safely avoid the potential morbidity, mortality and cost associated with unnecessary, invasive procedures without delaying or shifting the stage of diagnosis among those who have lung cancer."

The real-world registry study prospectively evaluated physicians’ management of lung nodules, as well as resulting clinical outcomes, at 35 academic and community-based U.S. medical centers. Researchers evaluated the impact of Percepta results on clinical decision-making for 213 patients with a low or intermediate pre-test risk of malignancy and a non-diagnostic bronchoscopy.

Comparing physicians’ pre-test to post-test plans for these patients, researchers observed a major shift following the Percepta classifier result. Among 67 patients for whom physicians had initially planned a subsequent invasive procedure, the Percepta test down-classified the risk of malignancy in 34.3 percent. Of these down-classified patients, physicians changed management plans for 73.9 percent – from an invasive procedure to surveillance – and 61 percent avoided a procedure for up to 12 months after initial evaluation.

In addition, researchers observed a larger absolute reduction in procedure rates among down-classified patients (61 percent vs. 52 percent, p<0.01), compared to those patients with unchanged risk.

Lastly, the time to diagnosis was not significantly delayed when comparing Percepta test down-classified patients to patients who were not down-classified (p=0.58), among all patients in the pre-test low and intermediate risk group who had confirmed lung cancers.

"The findings from this real-world multicenter study reinforce the large and growing body of evidence demonstrating that the Percepta classifier can reduce unnecessary invasive procedures for lung nodule patients when bronchoscopy findings are inconclusive," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "These data are particularly important as physicians seek ways to limit their patients’ exposure to invasive procedures. We believe they will help support physician adoption of and payer reimbursement for the Percepta classifier."

About the Percepta Classifier

The Percepta classifier is a novel genomic test that helps physicians determine individual patients’ lung cancer risk when results from bronchoscopy – a non-surgical procedure standardly used to assess lung nodules for cancer – are inconclusive. Veracyte estimates that approximately 545,000 bronchoscopies are performed each year to evaluate suspicious lung nodules and that up to 60 percent of these produce inconclusive results. The Percepta classifier was developed using advanced genomic technology and machine learning, and is based on novel "field of injury" science – which identifies genomic changes associated with lung cancer in current or former smokers using a simple brushing of the person’s airway. The test is covered by Medicare.

On August 5, 2020 Clovis Oncology, Inc. (NASDAQ: CLVS) announced today treatment of the first patient in the Phase 2 portion of the LIO-1 trial evaluating the combination of lucitanib, Clovis’ investigational angiogenesis inhibitor, including vascular endothelial growth factor receptors 1 through 3 (VEGFR1-3), and Opdivo (nivolumab), Bristol Myers Squibb’s PD-1 inhibitor, for the treatment of gynecologic cancers (Press release, Clovis Oncology, AUG 5, 2020, View Source [SID1234562876]). The LIO-1 trial is sponsored by Clovis as part of its broad clinical collaboration with Bristol Myers Squibb.

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"The Phase 2 part of the LIO-1 trial will advance our scientific understanding of the potential for an inhibitor of multiple tyrosine kinases, including VEGF, such as lucitanib, to be combined with a PD-1 inhibitor for the treatment of gynecologic cancers," said Dr. Erika Hamilton, Director of the Breast and Gynecologic Research Program, Sarah Cannon Research Institute at Tennessee Oncology. "It is estimated that nearly 100,000 women will be diagnosed with a gynecologic cancer in the U.S. this year alone, and it is vital that we identify new treatment options, in particular new combinations, for these women."

The Phase 2 part of LIO-1 is an open-label study to evaluate the safety and efficacy of lucitanib and Opdivo in patients with advanced gynecological solid tumors, including a broad spectrum of ovarian and endometrial subtypes including clear cell disease and patients with cervical cancer. The primary endpoint is confirmed best overall response rate based on investigator assessment according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. The study will be conducted in the U.S. and Europe, in collaboration with the European Network for Gynaecological Oncological Trial groups (ENGOT) for European study sites.

The Phase 2 dosing regimen for the LIO-1 study is based on results from the recently completed Phase 1b dose-escalation portion of the LIO-1 study. Abstracts describing the initial results of the Phase 1b portion of the LIO-1 study, as well as a trials-in-progress description of the Phase 2 study design of LIO-1, have been accepted as ePosters at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020 in September.

"The initiation of the Phase 2 stage of the LIO-1 clinical trial is an important milestone for the lucitanib development program, and I am grateful to our team and our investigators for their commitment to initiating this study safely and expeditiously in this new COVID-19 era," said Patrick J. Mahaffy, President and Chief Executive Officer of Clovis Oncology. "Importantly, we look forward to sharing initial Phase 1b data from LIO-1 at the upcoming virtual ESMO (Free ESMO Whitepaper) Congress, as well as data for each of our commercial and development-stage products. We are committed to pursue innovative clinical studies, both monotherapy and in combination, that are supported by a strong scientific rationale and offer the potential to provide additional treatment options with meaningful clinical benefit to a broad group of cancer patients."

More information about the LIO-1 trial (NCT04042116) is available here.

About Lucitanib

Lucitanib is an oral, potent inhibitor of the tyrosine kinase activity of vascular endothelial growth factor receptors 1 through 3 (VEGFR1-3), platelet-derived growth factor receptors alpha and beta (PDFGRα/β) and fibroblast growth factor receptors 1 through 3 (FGFR1-3). Emerging clinical data support the combination of angiogenesis inhibitors and immunotherapy to increase effectiveness in multiple cancer indications. Angiogenic factors, such as vascular endothelial growth factor (VEGF), are frequently up-regulated in tumors and create an immunosuppressive tumor microenvironment. Use of antiangiogenic drugs may reverse this immunosuppression and augment response to immunotherapy.

Lucitanib is an unlicensed medical product.