On August 2, 2018 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the second quarter of 2018 and provided a business update (Press release, Regeneron, AUG 2, 2018, View Source [SID1234528311]).

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"Regeneron made important commercial progress in the second quarter with continued strong U.S. sales growth for EYLEA in retinal diseases and Dupixent in atopic dermatitis. We are particularly pleased by U.S. launch progress with Dupixent for adults with moderate-to-severe atopic dermatitis, driven by a positive experience in the marketplace by patients and physicians in this serious disease; we anticipate continued robust growth as more physicians increase their experience with the product," said Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron. "In the second half of the year, we anticipate two significant U.S. regulatory approvals: cemiplimab for advanced cutaneous squamous cell carcinoma and Dupixent for uncontrolled asthma. We also plan to submit regulatory applications for Dupixent in adolescent atopic dermatitis and to report Phase 3 results in nasal polyps, in addition to other advances across our innovative portfolio for serious diseases."

Financial Highlights

Bayer records net product sales of EYLEA outside the United States and Sanofi records global net product sales of Dupixent, Praluent, Kevzara, and ZALTRAP. Refer to Table 4 below for the Company’s share of profits/losses recorded in connection with sales of EYLEA outside the United States and global sales of Dupixent, Praluent, and Kevzara. Sanofi pays the Company a percentage of aggregate net sales of ZALTRAP.

Second Quarter 2018 Business Highlights

Key Pipeline Progress
Regeneron has nineteen product candidates in clinical development, which consist of EYLEA and fully human antibodies generated using the Company’s VelocImmune technology, including eight in collaboration with Sanofi. Updates from the clinical pipeline include:

EYLEA (aflibercept) Injection

The Company recently submitted a supplemental Biologics License Application (sBLA) for EYLEA for the treatment of diabetic retinopathy.
In the second quarter of 2018, the Company submitted an sBLA for EYLEA in a pre-filled syringe.
Dupixent (dupilumab) Injection

Dupixent, an antibody that blocks signaling of IL-4 and IL-13, is being studied in asthma, adolescent and pediatric atopic dermatitis, nasal polyps, eosinophilic esophagitis (EoE), and grass immunotherapy, with additional studies planned in 2018.
In May 2018, the Company and Sanofi reported that a Phase 3 trial evaluating Dupixent to treat moderate-to-severe atopic dermatitis in adolescents (12-17 years of age) met its primary and key secondary endpoints.
In May 2018, the Company and Sanofi announced that the New England Journal of Medicine published detailed, positive results from two Phase 3 trials of Dupixent in moderate-to-severe asthma.
In the second quarter of 2018, a Phase 2 study of Dupixent in grass immunotherapy was initiated.
Praluent (alirocumab) Injection

In May 2018, the Company and Sanofi announced they will lower the net price of Praluent in exchange for straightforward, more affordable patient access from Express Scripts. Praluent has been chosen as the exclusive PCSK9 inhibitor therapy on the Express Scripts national formulary. The agreement took effect on July 1, 2018.
An sBLA and a Marketing Authorization Application (MAA) for Praluent for cardiovascular risk reduction have been recently submitted.
An sBLA for first-line treatment of hyperlipidemia has also been recently submitted.
In the second quarter of 2018, a Phase 3 pediatric study in heterozygous familial hypercholesterolemia (HeFH) was initiated.
Cemiplimab, an antibody to PD-1, is being studied in patients with cancer.

In April 2018, the FDA accepted for priority review the BLA for cemiplimab for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or patients with locally advanced CSCC who are not candidates for surgery. The target action date for the FDA decision is October 28, 2018.
In April 2018, the European Medicines Agency (EMA) also accepted for review the MAA for cemiplimab in patients with metastatic CSCC or patients with locally advanced CSCC who are not candidates for surgery.
In June 2018, the Company and Sanofi announced that pivotal data from two trials evaluating cemiplimab in advanced CSCC were published in the New England Journal of Medicine.
In May 2018, the Company and Sanofi announced positive interim results from a Phase 1 study assessing cemiplimab as a potential treatment for advanced non-small cell lung cancer (NSCLC).
Fasinumab, an antibody targeting Nerve Growth Factor (NGF), is being studied in patients with osteoarthritis of the knee or hip.

In April 2018, an independent Data Monitoring Committee monitoring the ongoing safety and efficacy of the fasinumab clinical trials recommended that the higher dose-regimens be discontinued based on the risk benefit assessment and that the program may continue with the lower dose-regimens of fasinumab; the ongoing osteoarthritis trials have been modified accordingly. Since the Phase 3 clinical study in chronic low back pain in patients with concomitant osteoarthritis was only using higher doses, the Company is no longer actively dosing patients in this study.
Evinacumab is an antibody to ANGPTL3. In the second quarter of 2018, a Phase 3 study in severe hypertriglyceridemia was initiated.

REGN3500 is an antibody to IL-33. In the third quarter of 2018, a Phase 2 study in chronic obstructive pulmonary disease (COPD) was initiated.

REGN3918 (pozelimab) is an antibody to C5. The Company expects to report full data from its Phase 1 study in paroxysmal nocturnal hemoglobinuria (PNH) in the second half of 2018, and plans to initiate a Phase 2 study in PNH in early 2019.

REGN4018 is a bi-specific antibody targeting MUC16 and CD3. In the second quarter of 2018, a Phase 1 study in platinum-resistant ovarian cancer was initiated.

REGN4659 is an antibody against CTLA4. In the second quarter of 2018, a Phase 1 study in advanced NSCLC was initiated.

Select Upcoming 2018 Milestones

Programs

Milestones

EYLEA

FDA decision on sBLA for every 12-week dosing interval in wet AMD (target action date of August 11, 2018)
Report one-year data from Phase 3 PANORAMA study for the treatment of non-proliferative diabetic retinopathy in patients without diabetic macular edema (DME)
Dupixent (dupilumab)

FDA decision on sBLA for asthma in adult/adolescent patients (target action date of October 20, 2018)
Additional regulatory agency decisions on applications for atopic dermatitis in adults outside the United States
Submit sBLA and MAA for expanded indication in adolescent patients with atopic dermatitis (12-17 years of age)
Report data from Phase 3 studies in nasal polyps
Initiate Phase 3 study in EoE
Initiate Phase 2 study in peanut allergy
Praluent (alirocumab)

FDA decision on sBLA for use with apheresis (target action date of August 24, 2018)
Initiate Phase 3 pediatric study in homozygous familial hypercholesterolemia (HoFH)
Kevzara (sarilumab)

Initiate Phase 3 study in giant cell arteritis
Initiate Phase 3 study in polymyalgia rheumatica
Cemiplimab (PD-1 Antibody)

FDA decision on BLA for advanced CSCC (target action date of October 28, 2018)
Continue Phase 3 patient enrollment for the treatment of non-small cell lung cancer, as well as various other studies
Fasinumab (NGF Antibody)

Report data from first Phase 3 efficacy study in osteoarthritis pain
Continue patient enrollment in Phase 3 long-term safety and efficacy studies in osteoarthritis
REGN3500 (IL-33 Antibody)

Initiate Phase 2 study in atopic dermatitis
Bispecific Antibodies

Initiate Phase 2 study for REGN1979 (CD20xCD3 Antibody) in follicular lymphoma
Submit Investigational New Drug Application (IND) for BCMAxCD3 antibody

Financial Results

Product Revenues: Net product sales were $996 million in the second quarter of 2018, compared to $924 million in the second quarter of 2017. EYLEA net product sales in the United States were $992 million in the second quarter of 2018, compared to $919 million in the second quarter of 2017. Overall distributor inventory levels remained within the Company’s one- to two-week targeted range.

Total Revenues: Total revenues, which include product revenues described above, increased by 9% to $1.608 billion in the second quarter of 2018, compared to $1.470 billion in the second quarter of 2017. Total revenues include Sanofi and Bayer collaboration revenues of $501 million in the second quarter of 2018, compared to $432 million in the second quarter of 2017. Sanofi collaboration revenue in the second quarter of 2018 increased primarily due to the Company’s share of higher net sales of Dupixent and an increase in reimbursable expenses in connection with late-stage clinical development activities for cemiplimab. These increases were partly offset by the Company’s Discovery and Preclinical Development Agreement with Sanofi ending on December 31, 2017, lower reimbursement for Dupixent development activities, and an increase in the Company’s share of the collaboration’s Dupixent commercialization expenses. Bayer collaboration revenue increased in the second quarter of 2018 primarily due to an increase in the Company’s share of net profits in connection with higher sales of EYLEA outside the United States.

The Company adopted Accounting Standard Codification (ASC) 606, Revenue from Contracts with Customers, as of January 1, 2018. The Company adopted the standard using the modified retrospective method; prior period amounts have not been adjusted and the adoption of the new standard did not have a material impact on the Company’s total revenues in the second quarter of 2018.

Refer to Table 4 for a summary of collaboration and other revenue.

Research and Development (R&D) Expenses: GAAP R&D expenses were $529 million in the second quarter of 2018, compared to $510 million in the second quarter of 2017. The higher R&D expenses in the second quarter of 2018 were principally due to an increase in cemiplimab and fasinumab clinical trial costs and higher R&D headcount and facilities-related costs, partly offset by a decrease in Dupixent development expenses and the discontinuation of certain development programs. In the second quarter of 2018, R&D-related non-cash share-based compensation expense was $60 million, compared to $70 million in the second quarter of 2017.

Selling, General, and Administrative (SG&A) Expenses: GAAP SG&A expenses were $365 million in the second quarter of 2018, compared to $307 million in the second quarter of 2017. The higher SG&A expenses in the second quarter of 2018 were primarily due to higher headcount and headcount-related costs and an increase in commercialization-related expenses for EYLEA and Dupixent, and, to a lesser extent, for cemiplimab. In the second quarter of 2018, SG&A-related non-cash share-based compensation expense decreased to $40 million, compared to $45 million in the second quarter of 2017.

Income Tax Expense: In the second quarter of 2018, GAAP income tax expense was $105 million and the effective tax rate was 16.0%, compared to $138 million and 26.3% in the second quarter of 2017. The Company’s effective tax rate for the second quarter of 2018 was significantly impacted by the bill known as the Tax Cuts and Jobs Act (the "U.S. Tax Reform Act"), which reduced the U.S. federal corporate income tax rate from 35% to 21% effective January 1, 2018. The effective tax rate for the second quarter of 2018 was positively impacted, compared to the U.S. federal statutory rate, primarily by the tax benefit associated with stock-based compensation, income earned in foreign jurisdictions with tax rates lower than the U.S. federal statutory rate, the foreign-derived intangible income deduction, and the federal tax credit for research activities.

Other income (expense), net: GAAP other income in the second quarter of 2018 included the recognition of $17 million of net unrealized gains on equity securities. In the first quarter of 2018, the Company adopted Accounting Standards Update ("ASU") 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities, as of January 1, 2018, which requires the Company to measure equity investments at fair value with changes in fair value recognized in net income; previously, such changes in fair value were recognized in Other comprehensive income (loss). Refer to Table 3 for the non-GAAP adjustment related to gains and losses on investments in equity securities.

GAAP other expenses in the second quarter of 2017 included a $30 million loss on debt extinguishment related to the 2017 Tarrytown lease transaction.

GAAP and Non-GAAP Net Income(2): GAAP net income was $551 million, or $5.12 per basic share and $4.82 per diluted share, in the second quarter of 2018, compared to GAAP net income of $388 million, or $3.66 per basic share and $3.34 per diluted share, in the second quarter of 2017.

Non-GAAP net income was $624 million, or $5.79 per basic share and $5.45 per diluted share, in the second quarter of 2018, compared to non-GAAP net income of $487 million, or $4.59 per basic share and $4.17 per diluted share, in the second quarter of 2017.

A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

2018 Financial Guidance(3)

Regeneron records net product sales of EYLEA in the United States. Outside the United States, EYLEA net product sales comprise sales by Bayer in countries other than Japan and sales by Santen Pharmaceutical Co., Ltd. in Japan under a co-promotion agreement with an affiliate of Bayer. The Company recognizes its share of the profits (including a percentage on sales in Japan) from EYLEA sales outside the United States within "Bayer collaboration revenue" in its Statements of Operations.

This press release uses non-GAAP net income, non-GAAP net income per share, non-GAAP unreimbursed R&D, and non-GAAP SG&A, which are financial measures that are not calculated in accordance with U.S. Generally Accepted Accounting Principles ("GAAP"). These non-GAAP financial measures are computed by excluding certain non-cash and other items from the related GAAP financial measure. Non-GAAP adjustments also include the estimated income tax effect of reconciling items.

The Company makes such adjustments for items the Company does not view as useful in evaluating its operating performance. For example, adjustments may be made for items that fluctuate from period to period based on factors that are not within the Company’s control (such as the Company’s stock price on the dates share-based grants are issued or changes in the fair value of the Company’s equity investments) or items that are not associated with normal, recurring operations (such as changes in applicable laws and regulations). Management uses these non-GAAP measures for planning, budgeting, forecasting, assessing historical performance, and making financial and operational decisions, and also provides forecasts to investors on this basis. Additionally, such non-GAAP measures provide investors with an enhanced understanding of the financial performance of the Company’s core business operations. However, there are limitations in the use of these and other non-GAAP financial measures as they exclude certain expenses that are recurring in nature. Furthermore, the Company’s non-GAAP financial measures may not be comparable with non-GAAP information provided by other companies. Any non-GAAP financial measure presented by Regeneron should be considered supplemental to, and not a substitute for, measures of financial performance prepared in accordance with GAAP. A reconciliation of the Company’s historical GAAP to non-GAAP results is included in Table 3 of this press release.

The Company’s 2018 financial guidance does not assume the completion of any significant business development transactions not completed as of the date of this press release.

Conference Call Information

Regeneron will host a conference call and simultaneous webcast to discuss its second quarter 2018 financial and operating results on Thursday, August 2, 2018, at 8:30 AM. To access this call, dial (800) 708-4539 (U.S.) or (847) 619-6396 (International). A link to the webcast may be accessed from the "Investors and Media" page of Regeneron’s website at www.regeneron.com. A replay of the conference call and webcast will be archived on the Company’s website and will be available for 30 days.

On August 2, 2018 Acorda Therapeutics, Inc. (Nasdaq: ACOR) reported its financial and pipeline update for the quarter ended June 30, 2018 (Press release, Acorda Therapeutics, AUG 2, 2018, View Source [SID1234528357]).

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"Our outstanding quarter reflected the continued excellence of our specialty neurology sales force and commercial, patient advocacy and affiliated teams. Our primary focus now is on the approval and launch of INBRIJA, which will benefit from these same capabilities," said Ron Cohen, M.D., Acorda’s President and CEO. "We expect INBRIJA, if approved, to help address the large unmet medical need for the approximately 350,000 people in the U.S. who are challenged by OFF periods related to Parkinson’s disease. Based on our continued market research, we believe the market opportunity for INBRIJA in the U.S. is greater than $800 million."

"The company’s strong execution year to date is fueling our ability to launch INBRIJA, to invest in the ARCUS pipeline and remain well capitalized throughout the INBRIJA launch," Dr. Cohen continued.

Second Quarter 2018 Financial Results

AMPYRA (dalfampridine) Extended Release Tablets, 10 mg – For the quarter ended June 30, 2018, the Company reported AMPYRA net revenue of $150.3 million compared to $131.6 million for the same quarter in 2017.

Research and development (R&D) expenses for the quarter ended June 30, 2018 were $25.9 million, including $1.5 million of share-based compensation compared to $51.2 million, including $3.0 million of share-based compensation for the same quarter in 2017.

Sales, general and administrative (SG&A) expenses for the quarter ended June 30, 2018 were $44.3 million, including $3.7 million of share-based compensation compared to $49.3 million, including $7.8 million of share-based compensation for the same quarter in 2017.

Provision for income taxes for the quarter ended June 30, 2018 was $8.4 million compared to a provision for income taxes of $5.5 million for the same quarter in 2017.

The Company reported GAAP net income of $46.2 million for the quarter ended June 30, 2018, or $0.98 per diluted share. GAAP net loss in the same quarter of 2017 was $8.2 million, or $0.18 per diluted share.

Non-GAAP net income for the quarter ended June 30, 2018 was $65.9 million, or $1.40 per diluted share. Non-GAAP net income in the same quarter of 2017 was $13.3 million, or $0.29 per diluted share. This quarterly non-GAAP net income measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, changes in the fair value of acquired contingent consideration, and restructuring costs. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At June 30, 2018, the Company had cash, cash equivalents and short-term investments of $391.7 million.

Guidance for 2018

The Company reiterates AMPYRA 2018 net revenue guidance of $330-$350 million.
R&D expenses for the full year 2018 are expected to be $100-$110 million and include manufacturing expenses associated with INBRIJA. This guidance is a non-GAAP projection that excludes share-based compensation, as more fully described below under "Non-GAAP Financial Measures."
SG&A expenses for the full year 2018 are expected to be $170-$180 million. This guidance is a non-GAAP projection that excludes share-based compensation, as more fully described below under "Non-GAAP Financial Measures."
The Company expects to end 2018 with a year-end cash balance in excess of $300 million.
This guidance may be revised with a positive outcome of the pending appeal.
Second Quarter 2018 Updates

INBRIJA (levodopa inhalation powder)
The Company’s Marketing Authorization Application (MAA) for INBRIJA was validated by the European Medicines Agency (EMA) and the application currently is under review. After the adoption of an opinion on the application by the Agency’s Committee for Medicinal Products for Human Use (CHMP), a final decision regarding the MAA will be issued by the European Commission.
In June, the Company presented four INBRIJA abstracts at the 2nd Pan American Parkinson’s Disease and Movement Disorders Congress in Miami. These data were previously presented at the American Academy of Neurology Annual Meeting in April 2018.
AMPYRA Patent Appeal
In June, the oral argument in the AMPYRA patent litigation took place at the U.S. Court of Appeals for the Federal Circuit. The Company is awaiting the Court’s decision.
On July 24, the Federal Circuit denied the Company’s motion for a preliminary injunction to prevent generic at risk launch pending the Court’s decision.
Webcast and Conference Call

Acorda will host a conference call and webcast to review its 2Q18 update and financial results on Thursday, August 2 at 8:30 a.m. ET. To participate in the conference call, dial (866) 393-4306 (domestic) or (734) 385-2616 (international) and reference the access code 4898766. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 11:30 a.m. ET on August 2, 2018 until 11:59 p.m. ET on September 1, 2018. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international); reference code 4898766. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain historical and forward-looking non-GAAP financial measures. In particular, Acorda has provided non-GAAP net income, adjusted to exclude the items below, and has provided 2018 guidance for R&D and SG&A expenses on a non-GAAP basis. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, the Company believes the presentation of non-GAAP net income, when viewed in conjunction with our GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because this measure excludes (i) non-cash compensation charges and benefits that are substantially dependent on changes in the market price of our common stock, (ii) non-cash interest charges related to the accounting for our outstanding convertible debt which are in excess of the actual interest expense owing on such convertible debt as well as non-cash interest charges related to the Fampyra royalty monetization, the asset based loan which was terminated in 2017 and acquired Biotie debt, (iii) changes in the fair value of acquired contingent consideration which do not correlate to our actual cash payment obligations in the relevant periods, (iv) acquisition related expenses and related foreign currency gains that pertain to a non-recurring event, and (v) expenses that pertain to non-routine restructuring events. The Company believes its non-GAAP net income measure helps indicate underlying trends in the Company’s business and is important in comparing current results with prior period results and understanding projected operating performance. Also, management uses this non-GAAP financial measure to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

In addition to non-GAAP net income, we have provided 2018 guidance for R&D and SG&A expenses on a non-GAAP basis. Due to the forward looking nature of this information, the amount of compensation charges and benefits needed to reconcile these measures to the most directly comparable GAAP financial measures is dependent on future changes in the market price of our common stock and is not available at this time. The Company believes that these non-GAAP measures, when viewed in conjunction with our GAAP results, provide investors with a more meaningful understanding of our ongoing and projected R&D and SG&A expenses. Also, management uses these non-GAAP financial measures to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

On August 2, 2018 Idera Pharmaceuticals, Inc. (NASDAQ: IDRA), a pharmaceutical company focused on the development and commercialization of its proprietary immune modulator, tilsotolimod, for the treatment of cancer, reported its financial and operational results for the second quarter ended June 30, 2018 (Press release, Idera Pharmaceuticals, AUG 2, 2018, View Source [SID1234528376]).

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"As we concluded the first half of 2018, we also arrived at a point of inflection for our company. Since my arrival at Idera, the company has undergone necessary significant changes as we evaluated numerous components of our portfolio, none of which were certain for success. After these nearly four years, it is now clear that any future success for Idera will be driven by our TLR9 agonist, tilsotolimod," stated Vincent Milano, Idera’s Chief Executive Officer.

"The body of data that we have generated with tilsotolimod continues to demonstrate the potential positive difference this drug can make in patients who have not benefited from existing immuno-therapy. Our mission from here is to explore the entirety of the opportunity both in melanoma and additional tumor types in order that we can offer hope to as many patients as possible," continued Milano.

"As it relates to the proposed merger that was recently terminated, we made an aggressive attempt to pursue a strategy that we believe would have strengthened our company, provided additional diversification and ultimately create more value for our shareholders. Throughout that process, my faith in the value of Idera and its future prospects has never wavered, nor will my belief in the approach of growing our company through business development activities. This will remain core for us moving forward as we continue to advance tilsotolimod, and at the same time search for additional assets to bring into Idera for our long-term success."

Milano further added, "To our long-term shareholders, I understand that these have been challenging times. I’ve worked through these moments more than once during my career. I appreciate the loyalty you’ve shown our company and I assure you that your loyalty serves as a great motivator for our entire team to deliver success in the future."

Clinical Development Program Updates:

ILLUMINATE (tilsotolimod) Clinical Development

ILLUMINATE 301 – Randomized Phase 3 trial of intratumoral tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with PD-1 refractory metastatic melanoma:

Trial initiated in the first quarter of 2018;
26 of the planned 80 sites across 12 countries have been activated for the randomization of patients into the trial;
Planned enrollment of approximately 300 patients with Overall Response Rate ("ORR") and Overall Survival as primary endpoints; and
U.S. Food and Drug Administration granted Fast Track Designation for tilsotolimod in combination with ipilimumab for the treatment of patients with unresectable or metastatic melanoma following failure of PD-1 inhibitor treatment in fourth quarter of 2017.
ILLUMINATE 204 – Phase 1/2 trial of intratumoral tilsotolimod in combination with ipilimumab or pembrolizumab in patients with PD-1 refractory metastatic melanoma:

Ipilimumab Combination Arm – Phase 2 Expansion Ongoing at RP2D of 8 mg

Enrollment (up to 60 patients) completion expected by year end 2018;
Recently increased trial sites open to enrollment to 8 (2 additional planned);
Abstract accepted for the upcoming ESMO (Free ESMO Whitepaper) 2018 Congress meeting in Munich, Germany, October 20, 2018.
ILLUMINATE-204 Key Findings Presented at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting (ASCO 2018) (Date cut-off for safety: 4/09/18; Data cut-off for efficacy: 5/09/18):

21 patients treated with the 8 mg dose of tilsotolimod in combination with ipilimumab have had disease evaluations;
Confirmed RECIST v1.1 responses (including 2 Complete Response [CR]) were observed in 8 of these 21 subjects (38.1%);
Six of 8 responses are ongoing (1 CR ongoing for nearly 2 years); median duration of response for these 8 has not yet been reached;
Overall 15 patients out of 21 evaluable for efficacy (71.4%) experienced disease control (CR, PR, or SD);
The combination regimen is generally well tolerated. 6/26 subjects (23%) had immune-related toxicities indicating that IMO-2125 + ipilimumab does not appear to add toxicity versus ipilimumab alone;
Injection-related toxicities were grade 1-2 transient fever and flu-like symptoms lasting <48 hours; and
15/26 patients (57.7%) with lesions accessible only by image-guided injection (5 deep visceral lesions and 10 lymph nodes) were included.
Pembrolizumab Combination Arm – Phase 1 Dose Escalation Ongoing – Update

Enrollment in the last dosing cohort (32 mg) ongoing; and
The previously reported partial response (PR) in 1 of the first 6 patients in the 16 mg cohort of intratumoral tilsotolimod in combination with pembrolizumab has evolved into a confirmed complete response (CR).
ILLUMINATE 101 – Phase 1b trial of intratumoral tilsotolimod monotherapy in patients with refractory solid tumors:

Completed enrollment in first three dosing cohorts [11 patients treated with 8 mg dose of tilsotolimod, 8 patients treated with 16 mg dose of tilsotolimod, 8 patients treated with 23 mg dose of tilsotolimod; and enrollment continues in the final cohort (32 mg)];
One patient in cohort 1 (8 mg) continues in follow-up; one patient in cohort 2 (16 mg) continues tilsotolimod monotherapy and one patient continues in follow-up; two patients in cohort 3 (23 mg) continue tilsotolimod therapy and two patients continue in follow-up. 6 of 8 planned patients for cohort 4 (32 mg) enrolled; and
First patient enrolled into the refractory melanoma cohort continues at a dose of 8 mg of tilsotolimod as monotherapy.
Investigator Sponsored Trials (IST)

During the second quarter of 2018, the company announced that it had entered into a clinical development support agreement with Pillar Partners Foundation. Under the terms of the agreement, Pillar has agreed to provide direct funding to support three investigator initiated clinical trials to further strategically expand the clinical research of tilsotolimod. The three trials are:

A Phase 1/2 open label study of intratumoral tilsotolimod in combination with intratumoral ipilimumab and IV nivolumab in a protocol open to multiple tumor types including non-small cell lung cancer (NSCLC), melanoma, squamous cell carcinoma of the head and neck and urothelial carcinoma. The principal investigator initiating this trial is Aurélien Marabelle, MD, PhD, Clinical Director of the Cancer Immunotherapy Program at Institut Gustave Roussy, Villejuif, France;
A Phase 2 study of intratumoral tilsotolimod in combination with IV pembrolizumab in patients with NSCLC. The principal investigator initiating this trial is Arafat Tfayli, MD, Professor of Clinical Medicine, Director of Hematology/Oncology Fellowship Program at the American University of Beirut Medical Center (AUBMC), Lebanon; and
A Phase 2 placebo-controlled study of intradermal administration of tilsotolimod in patients with T3/T4 primary melanoma scheduled to undergo a combined re-excision and sentinel node biopsy (SNB) procedure. The principal investigators initiating this are Bas Koster, MD and Tanja de Gruijl, PhD at The VU University Medical Center, Amsterdam, the Netherlands.
Corporate Updates:
In July 2018, following an analysis of its gene-silencing technology platform and our research portfolio and the termination of the merger agreement, the company decided to suspend its rare disease and discovery programs as part of its overall strategy to more narrowly focus its capital resources on the development and commercialization of tilsotolimod. In connection with this focused strategy, it will be closing its facility at 167 Sidney Street in Cambridge, Massachusetts, with its Exton, Pennsylvania, location serving as its new headquarters.

On July 27, 2018, the Company implemented a 1-for-8 reverse split of its issued and outstanding shares of common stock (the Reverse Stock Split) and set the number of its authorized shares of common stock at 70,000,000. The Reverse Stock Split became effective on July 27, 2018 at 5:00 pm and the Company’s common stock began trading on The Nasdaq Capital Market on a post-split basis at the open of trading on July 30, 2018. The Reverse Stock Split affected all of the company’s stockholders uniformly and did not alter any stockholder’s percentage interest in the company’s equity, except to the extent that the Reverse Stock Split resulted in any of the Company’s stockholders owning a fractional share, which will be settled in cash.

On January 21, 2018, the company entered into an Agreement and Plan of Merger (the Merger Agreement) with BioCryst Pharmaceuticals, Inc. (BioCryst), Nautilus Holdco, Inc., a direct, wholly owned subsidiary of BioCryst (Holdco), Island Merger Sub, Inc., a direct, wholly owned subsidiary of Holdco, and Boat Merger Sub, Inc., a direct, wholly owned subsidiary of Holdco. The board of directors of each of Idera and BioCryst unanimously approved the Merger Agreement and the transactions contemplated thereby and the required regulatory approvals were received. However, the proposed merger was subject to approval by the stockholders of Idera and BioCryst, and satisfaction of other customary closing conditions, as specified in the Merger Agreement.

At a special meeting of BioCryst stockholders held on July 10, 2018, BioCryst’s stockholders voted against the adoption of the Merger Agreement. Following such vote and in accordance with the terms of the Merger Agreement, BioCryst terminated the Merger Agreement on July 10, 2018.

In accordance with the Merger Agreement, BioCryst paid the company a fixed expense reimbursement amount of $6 million in connection with the termination of the Merger Agreement.

Financial Results
Second Quarter Results
Net loss applicable to common stockholders for the three months ended June 30, 2018 was $16.0 million, or $0.59 per basic and diluted share, compared to net loss applicable to common stockholders of $21.5 million, or $1.15 per basic and diluted share, for the same period in 2017. Revenue in each of the three months ended June 30, 2018 and 2017 was nominal. Research and development expenses for the three months ended June 30, 2018 totaled $10.9 million compared to $17.9 million for the same period in 2017. General and administrative expense for the three months ended June 30, 2018 totaled $5.6 million compared to $3.9 million for the same period in 2017.

As of June 30, 2018, the company’s cash and cash equivalents totaled $94.0 million. The company currently anticipates that, based on its current operating plan, its existing cash and cash equivalents, including the expense reimbursement proceeds received in July 2018 in connection with the termination of the Merger Agreement, will be sufficient to enable it to fund company operations into the first quarter of 2020.

On August 2, 2018 Aileron Therapeutics (Nasdaq:ALRN), the leader in the field of stapled peptide therapeutics for cancers and other diseases, reported that Manuel Aivado, SVP and Chief Medical Officer, will present at the Canaccord Genuity 38th Annual Growth Conference being held in Boston, MA (Press release, Aileron Therapeutics, AUG 2, 2018, View Source;p=RssLanding&cat=news&id=2361619 [SID1234528619]).

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

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Presentation Details:

Event: Canaccord Genuity 38th Annual Growth Conference
Date: Thursday, August 9, 2018
Time: 1:00 – 1:25 p.m. EDT
Location: Intercontinental Boston Hotel, Boston, MA

A live webcast of the presentation may be accessed from the Events & Presentations section of Aileron’s website. An archived version of the webcast will be available for replay following the event.

On August 2, 2018 Arbutus Biopharma Corporation (Nasdaq: ABUS) reported its 2018 second quarter financial results and provides corporate update (Press release, Arbutus Biopharma, AUG 2, 2018, View Source [SID1234528635]). "With the advancement of AB-506 and AB-452 into clinical development, we are laying the groundwork for a potentially similar paradigm shift in HBV as what has been seen in HCV," said Dr. Mark J Murray, Arbutus’ President and Chief Executive Officer. "Once the Phase 1a/1b studies are completed, our goal is to rapidly initiate an all-oral combination clinical trial using AB-506 and AB-452 with an approved nucleoside analogue drug. We expect this study to begin by the end of 2019 and move us closer to developing a curative treatment for people with HBV."

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

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

                  Schedule Your 30 min Free Demo!

Recent Accomplishments and Upcoming Clinical Milestones

Phase 1a/1b study initiated for AB-506, Arbutus’ second generation, and potentially best-in-class, oral capsid inhibitor. The healthy volunteer portion of the study will be followed by dosing cohorts of HBV patients, with top-line results expected in the second quarter of 2019.

The regulatory filing for AB-452, Arbutus’ novel and proprietary RNA destabilizer, is on track for submission in the third quarter, with subject dosing to follow in the fourth quarter of 2018.

Pending completion of the monotherapy Phase 1a/1b studies for AB-506 and AB-452, Arbutus expects to begin an all-oral combination study with AB-506 and AB-452 with an approved nucleoside analogue by the end of 2019.

HBsAg data from the six-week treatment point of patients in the ARB-1467 Phase 2b combination study (with nucleoside analogue), qualifying patients for PEG-IFN add on therapy will be available in Q4.

Partnership with Alnylam

Patisiran is an RNAI therapeutic targeting transthyretin that is being developed as a treatment for hereditary ATTR (hATTR) amyloidosis. Patisiran is currently under Priority Review as a Breakthrough Therapy with the U.S. Food and Drug Administration. The PDUFA date for patisiran is August 11, 2018.

Successful approval of patisiran will trigger a royalty entitlement to Arbutus for the proprietary LNP technology licensed by Arbutus to Alnylam for patisiran.

Genevant

Genevant, a company formed in the second quarter of 2018 and jointly owned by Arbutus and Roivant Sciences, recently announced that it has entered into a strategic partnership with BioNTech AG, an industry leader in mRNA therapy development. BioNTech and Genevant will develop five mRNA products for rare diseases with high unmet medical need under a 50/50 co-development and co-commercialization collaboration.

Genevant and BioNTech have also agreed to a series of exclusive licenses covering the application of Genevant’s proprietary delivery technology for five oncology targets, for which Genevant is eligible to receive significant commercial milestones. This partnership advances Genevant’s goal of having 5-10 programs in the clinic by 2020 across RNAi, mRNA, and gene editing modalities and positions Genevant as a leader in the development of RNA-based therapeutics. Arbutus is entitled to royalties on any product sales by Genevant.

Financial Results

Cash, Cash Equivalents and Investments

As at June 30, 2018, Arbutus had cash, cash equivalents and short-term investments totaling $154.9 million, as compared to $139.0 million in cash and cash equivalents, short-term investments, and restricted investments at December 31, 2017. The increased cash balance was the result of $66.4 million of gross proceeds received in Q1 2018 from the second tranche Preferred Shares issued to Roivant, offset by cash used in operations.

For further details with respect to the Preferred Shares, please refer to Arbutus’ Proxy materials filed on Schedule 14A with the U.S. Securities and Exchange Commission on December 6, 2017.

Net Income (Loss)

For Q2 2018, net income attributable to common shares was $0.6 million ($0.01 basic income per common share) as compared to a net loss of $18.3 million ($0.33 basic loss per common share) for Q2 2017. The Company recorded a non-cash gain of $24.9 million in the second quarter of 2018 related to the formation of Genevant. See discussion below in Gain on Investment in Genevant.

Revenue

Revenue was $1.2 million in Q2 2018 as compared to $1.0 million in Q2 2017.

In October 2017, Arbutus entered into a license agreement with Gritstone that entitles Gritstone to research, develop, manufacture and commercialize products with the Company’s LNP technology in exchange for an upfront license payment and potential future milestone and royalty payments. In April 2018, as part of the license agreement for Arbutus’ delivery technologies, Genevant gained the right to receive 50% of future revenues from Gritstone. Revenue recognized in Q2 2018 relates to Arbutus’ share of the earned portion of the upfront license fee, as well as services provided to Gritstone.

In addition, Arbutus has ongoing license agreements with Alnylam and Spectrum, under which Arbutus is eligible to receive royalties on sales.

Research, Development, Collaborations and Contracts Expenses

Research, development, collaborations and contracts expenses increased to $16.4 million in Q2 2018 from $15.4 million in Q2 2017. Program R&D expenses increased as the Company’s HBV pipeline expands and progresses further into the clinic. In the first half of 2017, Arbutus initiated a Phase 1 clinical trial for AB-423. In the first half of 2018 the Company initiated a Phase 1a/1b clinical trial POS AB-506 (capsid inhibitor) which has shown striking potency and improved PK over AB-423 in preclinical studies. In the first half of 2018 Arbutus has progressed AB-452 (RNA destabilizer) through IND/CTA enabling work to prepare for a regulatory filing in Q3 2018. Arbutus also continues to incur costs related to its other programs pre-IND/CTA work on AB-729 (GalNAc-RNAi). The increase in program R&D expenses in Q2 2018 was offset by a decrease of $1.2 million related to non-cash compensation expense related to the expiry of repurchase rights.

General and Administrative

General and administrative expenses were $3.8 million in Q2 2018, as compared to $4.6 million in Q2 2017.

General and administrative expenses decreased in Q2 2018 compared to Q2 2017 primarily due to a decrease in non-cash compensation expense related to the expiry of repurchase rights in Q3 2017, offset by professional fees incurred in Q2 2018 related to the launch of Genevant Sciences.

Gain on Investment in Genevant

As previously announced, on April 11, 2018 Arbutus entered into an agreement with Roivant to form Genevant Sciences, a jointly-owned company focused on the discovery, development, and commercialization of a broad range of RNA-based therapeutics enabled by Arbutus’ proprietary lipid nanoparticle (LNP) and ligand conjugate delivery technologies. Initially, Arbutus held a 50% ownership interest in Genevant and after additional funding received by Genevant at a stepped-up valuation, at June 30, 2018 Arbutus holds 41% of the outstanding equity of Genevant. As a result of the equity interest received by Arbutus in exchange for the license of its delivery technologies and other contributed assets, Arbutus has recognized a non-cash gain of $24.9 million during Q2 2018.

Site Consolidation

In February 2018, we announced a site consolidation and organizational restructuring to better align our HBV business in Warminster, PA, by reducing our global workforce and closing our Burnaby facility. In Q1 2018 we began executing our site consolidation plan and substantially completed these activities in Q2 2018. We recorded expenses of $2.6 million in Q2 2018 related to the site consolidation. We expect related total cash expenditures to be approximately $5.0 million and have recognized $4.2 million to June 30, 2018.

Outstanding Shares

The Company had 55.3 million common shares issued and outstanding at June 30, 2018. In addition, the Company had 6.8 million options outstanding and 1.164 million Series A participating convertible preferred shares outstanding, which (including the annual 8.75% coupon) will be mandatorily convertible into 22.6 million common shares on October 18, 2021. Assuming the outstanding options and convertible preferred shares were fully converted, the Company would have had 84.7 million common shares outstanding at June 30, 2018.

The Company’s consolidated financial statements are prepared in accordance with generally accepted accounting principles in the United States (U.S. GAAP) on a basis consistent for all periods presented. In addition to the results reported in accordance with U.S. GAAP, the Company provides additional measures that are considered "non-GAAP" financial measures under applicable SEC rules. These non-GAAP financial measures should not be viewed in isolation or as a substitute for GAAP net loss and basic and diluted net loss per common share.

The Company evaluates items on an individual basis, and considers both the quantitative and qualitative aspects of the item, including (i) its size and nature, (ii) whether or not it relates to the Company’s ongoing business operations, and (iii) whether or not the Company expects it to occur as part of its normal business on a regular basis. In the three months ended June 30, 2018, the Company’s non-GAAP net loss and non-GAAP net loss attributable to common shares per common share excludes the gain on investment related to the launch of Genevant. The Company believes that the exclusion of this item provides management and investors with supplemental measures of performance that better reflect the underlying economics of the Company’s business. In addition, the Company believes the exclusion of this item is important in comparing current results with prior period results and understanding projected operating performance.

Conference Call Today

Arbutus will hold a conference call and webcast today, Thursday, August 2, 2018 at 1:30 PM Pacific Time (4:30 PM Eastern Time) to provide a corporate update. You can access a live webcast of the call through the Investor section of Arbutus’ website at www.arbutusbio.com. Alternatively, you can dial 1-866-393-1607 or 1-914-495-8556 and reference conference ID 6966479.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling 1-855-859-2056 or 1-404-537-3406 and reference conference ID 6966479.