On April 30, 2020 UroGen Pharma Ltd. (Nasdaq: URGN) reported The Lancet Oncology published results from the pivotal Phase 3 OLYMPUS trial, reporting that 59% of low-grade upper tract urothelial cancer (LG-UTUC) patients treated with UGN-101, now referred to as Jelmyto (mitomycin) for pyelocalyceal solution, achieved a Complete Response (CR) (Press release, UroGen Pharma, APR 30, 2020, View Source [SID1234556855]). Additionally, in the publication, durability at 12 months (based on interim data) was estimated to be 84% by Kaplan-Meier analysis.1

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!

Jelmyto consists of mitomycin, an established chemotherapy, and sterile hydrogel, using UroGen’s proprietary sustained release RTGel technology. It has been designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is indicated for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC). It is contraindicated in patients with perforation of the bladder or upper urinary tract.

"The current approach to treating low-grade upper tract urothelial cancer includes multiple endoscopic surgeries, high risk of disease recurrence and for many patients, the eventual removal of a kidney, which can present a whole new set of challenges that can adversely impact long-term health," said Seth Lerner, M.D., FACS, Professor of Urology at Baylor College of Medicine in Houston, TX and Principal Investigator of the OLYMPUS trial. "Data from the OLYMPUS trial suggest UGN-101 is associated with a higher than expected initial complete response rate and strong durability without additional endoscopic surgery, which represents significant progress for patients living with this rare and difficult-to-treat type of cancer."

LG-UTUC is a rare cancer that develops in the lining of the upper urinary tract, ureters and kidneys. In the U.S., there are approximately 6,000 – 7,000 new or recurrent LG-UTUC patients annually. It is a challenging condition to treat due to the complex anatomy of the urinary tract system. The current standard of care includes multiple surgeries, and most patients require a radical nephroureterectomy, which includes the removal of the renal pelvis, kidney, ureter and bladder cuff.2 Treatment is further complicated by the fact that LG-UTUC is most commonly diagnosed in patients over 70 years of age, who may already have compromised kidney function and may suffer further complications as a result of major surgery.

In the pivotal OLYMPUS trial, participants received six once-weekly instillations of Jelmyto via retrograde catheter in the renal pelvis and calyces. The intent-to-treat (ITT) population included the seventy-one patients who received at least one dose of Jelmyto; 48% of these patients had tumors that were deemed endoscopically unresectable. As reported in The Lancet Oncology publication, Jelmyto achieved a Complete Response (CR) in 59% of the ITT population (a CR was defined as negative 3-month ureteroscopic evaluation, negative cytology and negative for cause biopsy) and durability at 12 months (at the time of the data cutoff) was estimated to be 84% by Kaplan-Meier analysis.

"The positive results of the OLYMPUS trial demonstrate that Jelmyto has the potential to help fulfill a significant unmet need for patients with low-grade upper tract urothelial cancer," said Dr. Mark P. Schoenberg, Chief Medical Officer at UroGen. "We are committed to addressing treatment challenges for this underserved patient population and to improving the standard of care for those who need it most."

As reported in The Lancet Oncology publication, in the trial, 67 patients (94%) experienced adverse events (AEs) and 60 patients (85%) had AEs that were considered to be related to the study treatment or procedure. No treatment-related deaths occurred. Overall, the most frequently reported AEs were ureteric stenosis in 31 (44%) of 71 patients, urinary tract infection in 23 (32%), haematuria in 22 (31%), flank pain in 21 (30%), and nausea in 17 (24%).

The U.S. Food and Drug Administration (FDA) approved Jelmyto on April 15, 2020. The FDA-approved labeling for Jelmyto reports Complete Response (CR) (primary endpoint) of 58% in the intent-to-treat population. The product labeling also reports that at the 12-month time point for assessment of durability, 19 patients remained in CR, seven had experienced recurrence of disease, nine patients continued to be followed for the 12-month duration of response, and median duration of response was not reached as of the FDA-approval date.

About the Phase 3 OLYMPUS Trial

OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is an open-label, single-arm Phase 3 clinical trial of UGN-101, Jelmyto (mitomycin) for pyelocalyceal solution, to evaluate the safety, tolerability and tumor ablative effect of Jelmyto in patients with low-grade UTUC. Seventy-one patients were treated at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of Jelmyto administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine Complete Response (CR), the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer and for cause biopsy. Patients who achieved a CR at the PDE timepoint were then followed for up to 12 months to determine the durability of response with Jelmyto.

About Jelmyto

Jelmyto (mitomycin) for pyelocalyceal solution, is a drug formulation of mitomycin indicated for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, Jelmyto is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is delivered to patients using standard ureteral catheters or nephrostomy tube. The U.S. FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG-UTUC. On April 15, 2020, the FDA approved Jelmyto, making it the first drug approved for the treatment of LG-UTUC in adult patients.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as upper tract urothelial cancers (UTUC). In the U.S., there are approximately 6,000 – 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can lead to a high rate of recurrence and relapse.

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose.
Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: side pain, urinary tract infection, blood in your urine, kidney problems, tiredness, nausea, stomach pain, trouble with urination, vomiting, low red blood cell count, frequent urination, itching, chills, and fever.

On April 30, 2020 Pacira BioSciences, Inc. (NASDAQ:PCRX) reported that it will report its first quarter financial results before the open of the U.S. markets on Thursday, May 7, 2020 (Press release, Pacira Pharmaceuticals, APR 30, 2020, View Source [SID1234556854]). Following the release, the company will host a live conference call and webcast at 8:30 a.m. ET.

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!

To participate in the conference call, dial 1-877-845-0779 and provide the passcode 8568879. International callers may dial 1-720-545-0035 and use the same passcode. In addition, a live audio of the conference call will be available as a webcast. Interested parties can access the event through the "Events" page on the Pacira website at investor.pacira.com.

For those unable to participate in the live call, a replay will be available at 1-855-859-2056 (domestic) or 1-404-537-3406 (international) using the passcode 8568879. The replay of the call will be available for one week from the date of the live call. The webcast will be available on the Pacira website for approximately two weeks following the call.

On April 30, 2020 Bio-Techne Corporation (NASDAQ:TECH) reported its financial results for the third quarter ended March 31, 2020 (Press release, Bio-Techne, APR 30, 2020, View Source [SID1234556853]).

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!

Third Quarter FY2020 Snapshot

Third quarter organic revenue increased by 6% (5% reported) to $194.7 million and 8% (8% reported) in the first three quarters of fiscal 2020 to $562.9 million.
GAAP EPS was $0.92 versus $1.15 one year ago. Delivered adjusted earnings per share (EPS) of $1.39 versus $1.21 one year ago.
Adjusted Operating Margin increased to 36.4% in the third quarter of fiscal 2020 compared to 35.2% in the third quarter of fiscal 2019.
Maintained strong operating cash flows with an increase of over 27% in year-to-date operating cash flow from the prior fiscal year.
Bio-Techne is utilizing our deep product portfolio and expertise as a trusted partner in the fight against COVID-19. Our automated immunoassay platform EllaTM, is providing front-line support through real-time monitoring of an individual’s immune responses to COVID-19 while we deploy our other market-leading technologies to provide critical support for researchers further advancing the development of potential therapeutics and vaccinations worldwide.
Continued execution of long-term Exosome strategy with coverage expansion through a 10-year agreement with General Services covering approximately 9 million lives, product enhancements with an ExoDx at-home specimen collection kit, and certification of the ExoDx CLIA lab for virology testing expanding our CLIA lab capabilities.
The Company’s financial statements are prepared in accordance with accounting principles generally accepted in the United States (GAAP). Adjusted EPS, adjusted earnings, adjusted gross margin, adjusted operating income, and adjusted operating margin are non-GAAP measures that exclude certain items detailed later in this press release under the heading "Use of non-GAAP Adjusted Financial Measures." A reconciliation of GAAP to non-GAAP financial measures is included in this press release.

"Our Q3 performance is indicative of the resolve and energy our teams around the world have to succeed in these trying times. I want to thank all of our employees for their commitment and hard work," said Chuck Kummeth, President and Chief Executive Officer of Bio-Techne. "A finish of 6% organic growth is a great accomplishment for the company, given the unprecedented challenges we all faced with the COVID-19 virus pandemic during the second half of the quarter."

Kummeth added, "Going forward, we have all the necessary processes in place to service our customers remotely as their labs come back online. In addition, we have pivoted a significant part of the company to innovate for COVID-19 solutions. We are currently working on many COVID-19 related projects, and I am extremely excited about how our company can participate on a global level to help eradicate this horrible virus. As a premier Proteins, Antibody, Assay and Diagnostics producer, we are in a unique position to provide reagents for all aspects of COVID-19 research and diagnostics. Our mission thrives on helping solve these types of problems."

COVID-19 Business Update

COVID-19 negatively impacted sales growth during the third quarter of fiscal year 2020 due to numerous customer site shutdowns that occurred in our academia and Bio-Pharma end-markets, particularly in the last several weeks of the quarter. We estimate these customer site shutdowns negatively impacted our third quarter fiscal year 2020 sales by approximately 3%. Customer site shutdowns will continue to have a negative impact on sales while they remain in effect, and we are currently unable to forecast the impact given uncertainty over the duration of the COVID-19 pandemic. We are anticipating a positive long-term outlook for future sales growth resulting from expected future funding increases within life-science research in response to the current pandemic.

The Company has responded to COVID-19 by developing resources for SARS-CoV-2 detection, cytokine monitoring, and drug discovery, all which provide critical support in understanding the mechanisms of infection and developing effective treatments. The Company continues to pursue available opportunities to partner in the fight against COVID-19, which may partially offset the impact of our customer site closures.

Adjusted EPS was negatively impacted by COVID-19 by an estimated $0.07-$0.09 in the third quarter of fiscal 2020. The COVID-19 impact on adjusted EPS was primarily due to the sales impacts described above. We anticipate the short- and long-term impacts of COVID-19 on adjusted EPS to be similar to that of sales growth.

The Company remains in a strong financial position with sufficient available cash as well as access to additional funding if necessary, through our long-term debt agreement. We did not experience any material changes to our March 31, 2020 Balance Sheet resulting from COVID-19 for items such as additional reserves or asset impairments resulting from the pandemic.

The Company remains fully operational as we abide by local "stay at home" orders across the world. To achieve this, the Company is utilizing a remote workforce where possible. For employees who are unable to work remotely, the Company adopted significant protective measures at our sites, including staggered shifts, and distancing and hygiene best practices recommended by the Center for Disease Control (CDC). In addition, the Company has taken additional steps to monitor and strengthen our supply chain to ensure we can maintain an uninterrupted supply of our critical products and services.

Third Quarter Fiscal 2020

Revenue

Net sales for the third quarter increased 5% to $194.7 million. Organic growth was 6% compared to the prior year, with foreign currency exchange having an unfavorable impact of 1% and acquisitions having an immaterial impact on revenue.

GAAP Earnings Results

GAAP EPS decreased to $0.92 per diluted share, versus $1.15 in the same quarter last year. GAAP operating income for the third quarter of fiscal 2020 increased 8% to $47.8 million, compared to $44.1 million in the third quarter of fiscal 2019. GAAP operating margin was 24.5%, compared to 23.9% in the third quarter of fiscal 2019. GAAP operating margin compared to prior year was positively impacted by volume leverage on sales growth.

Non-GAAP Earnings Results

Adjusted EPS increased to $1.39 per diluted share, versus $1.21 in the same quarter last year, an increase of 15%. Adjusted EPS increased primarily due to revenue growth in the quarter. Adjusted operating income for the third quarter of fiscal 2020 increased 17% compared to the third quarter of fiscal 2019. Adjusted operating margin was 36.4%, compared to 35.2% in the third quarter of fiscal 2019. Adjusted operating margin compared to the prior year was favorably impacted by volume leverage on sales growth.

Segment Results

Management uses adjusted operating results to monitor and evaluate performance of the Company’s business segments, as highlighted below. Since these results are used for this purpose, they are also considered to be prepared in accordance with GAAP.

Protein Sciences Segment

The Company’s Protein Sciences segment is one of the world’s leading suppliers of specialized proteins such as cytokines and growth factors, immunoassays, antibodies and reagents, to the biotechnology community. Additionally, the segment provides an array of platforms useful in various areas of protein analysis. Protein Sciences segment’s third quarter fiscal 2020 net sales were $145.5 million, an increase of 6% from $137.9 million for the third quarter of fiscal 2019. Organic growth for the segment was 6%, with foreign currency exchange having an unfavorable impact of 1% on revenue growth and acquisitions contributing 1% to revenue growth. Protein Sciences segment’s operating margin was 44.7% in the third quarter of fiscal 2020 compared to 45.1% in the third quarter of fiscal 2019. The segment’s operating margin compared to the prior year was negatively impacted by sales mix and the acquisition of B-Mogen in the fourth quarter of fiscal 2019.

Diagnostics and Genomics Segment

The Company’s Diagnostics and Genomics segment provides blood chemistry and blood gas quality controls, hematology instrument controls, immunoassays and other bulk and custom reagents for the in vitro diagnostic market. The Diagnostics and Genomics segment also develops and provides in situ hybridization products as well as exosome-based diagnostics for various pathologies, including prostate cancer. The Diagnostics and Genomics segment’s third quarter fiscal 2020 net sales were $49.4 million compared to $47.1 million for the third quarter of fiscal 2019. Organic growth for the segment was 5%, with foreign currency exchange having an immaterial impact on revenue. The Diagnostics and Genomics segment’s operating margin was 14.3% in the third quarter of fiscal 2020 compared to 7.6% in the third quarter of fiscal 2019. The segment’s operating margin was favorably impacted by volume leverage.

Conference Call

Bio-Techne will host an earnings conference call today, April 30, 2020 at 8:00 a.m. CST. To listen, please dial 1-877-407-9208 or 1-201-493-6784 for international callers, and reference conference ID 13701798. A live webcast of the earnings conference call is also available on the Bio-Techne website (View Source). A recorded rebroadcast will be available for interested parties unable to participate in the live conference call by going to:

View Source

The replay will be available from 11:00 a.m. CDT on Thursday, April 30, 2020 until 11:00 p.m. CDT on Sunday, May 30, 2020.

On April 30, 2020 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported financial results for the first quarter ended March 31, 2020, and provided a business update (Press release, Insmed, APR 30, 2020, View Source [SID1234556850]).

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!

"The start of 2020 saw an unprecedented global event with the COVID-19 pandemic. We are proud to be fighting this disease alongside many in the biopharmaceutical industry by supporting an investigator-initiated study of brensocatib, formerly known as INS1007, in patients with severe COVID-19. While we continue to advance this novel treatment candidate in bronchiectasis, we are hopeful that Insmed can contribute in the fight against COVID-19," commented Will Lewis, Chairman and Chief Executive Officer of Insmed. "I am also pleased that in an incredibly challenging environment, we have been able to support the NTM lung disease community remotely and provide an uninterrupted supply of ARIKAYCE while working to ensure the safety of all stakeholders, including our employees. With meaningful advances in our pipeline; important growth catalysts for ARIKAYCE; and a strong balance sheet, we believe we are well-positioned to stay the course through these challenging times and continue to serve patients in need."

First Quarter 2020 Financial Results

·Total revenue for the first quarter ended March 31, 2020, was $36.9 million, comprising U.S. net sales of $35.2 million and ex-U.S. net sales of $1.7 million. This compares to total revenue of $21.9 million for the first quarter of 2019.
·Cost of product revenues (excluding amortization of intangible assets) was $8.4 million for the first quarter of 2020, compared to $4.2 million for the first quarter of 2019.
·Research and development (R&D) expenses were $36.2 million for the first quarter of 2020, compared to $31.2 million for the first quarter of 2019. The increase in R&D expenses is primarily attributable to an increase in compensation and benefit-related expenses, including stock-based compensation expense, due to an increase in headcount and increases in manufacturing and regulatory, quality assurance, and medical affairs costs.
·Selling, general, and administrative (SG&A) expenses for the first quarter of 2020 were $51.3 million, compared to $54.8 million for the first quarter of 2019. The decrease in SG&A expenses is primarily due to a decrease in compensation and benefit-related costs and external costs related to ARIKAYCE (amikacin liposome inhalation suspension).
·For the first quarter of 2020, Insmed reported a GAAP net loss of $66.4 million, or $0.74 per share, compared to a GAAP net loss of $74.2 million, or $0.96 per share, for the first quarter of 2019.

Recent Corporate Developments & Program Highlights

Brensocatib Program Updates:

As previously announced:

·Phase 3 Study in Bronchiectasis: Insmed plans to initiate a Phase 3 program with brensocatib in patients with bronchiectasis in the second half of 2020, following alignment with the U.S. Food and Drug Administration (FDA) on trial design.

·Investigator-Initiated Study in Severe COVID-19: Insmed is supporting an investigator-initiated study of brensocatib in patients with severe COVID-19. The randomized, double-blind, placebo-controlled trial is expected to enroll up to 300 patients hospitalized with confirmed COVID-19 and will be led by the University of Dundee in the UK.

·AstraZeneca Option Exercise: In March 2020, AstraZeneca AB exercised an option pursuant to the companies’ October 2016 license agreement under which AstraZeneca can advance clinical development of brensocatib in the indication of chronic obstructive pulmonary disease (COPD) or asthma up to and including Phase 2b clinical trials. Insmed retains full development and commercialization rights for brensocatib in all other indications and geographies.

ARIKAYCE Label Expansion

Insmed continues to advance the post-approval confirmatory clinical trial for ARIKAYCE in a front-line setting of patients with Mycobacterium avium complex (MAC) lung disease as well as the development of an appropriate patient reported outcome (PRO) tool that will enable the assessment of ARIKAYCE for the treatment of nontuberculous mycobacterial (NTM) lung disease. Insmed plans to initiate both the confirmatory study and a study to validate the PRO in the second half of 2020 and to run these studies in parallel, pending alignment with the FDA.

Global Growth

Insmed remains on track in preparing for potential approvals and commercial launches of ARIKAYCE in both Japan and Europe. In Japan, the Company announced in March that it had submitted a new drug application to the Ministry of Health, Labour and Welfare for ARIKAYCE for the treatment of patients with NTM lung disease caused by MAC who did not sufficiently respond to prior treatment. In Europe, pending approval of the marketing authorization application for ARIKAYCE, the Company anticipates a potential launch in Germany by the end of 2020, followed shortly thereafter by the UK.

Treprostinil Palmitil (Formerly INS1009) Advancement

Insmed is advancing treprostinil palmitil, formerly known as INS1009, a dry powder, inhaled treprostinil prodrug formulation, for the potential treatment of pulmonary arterial hypertension. The Company plans to file an Investigational New Drug application and initiate a Phase 1 study of treprostinil palmitil in the second half of 2020.

COVID-19 Response

In March 2020, Insmed implemented, and continues to maintain, a number of corporate initiatives in response to the global COVID-19 pandemic. These include a remote working policy for all employees, including field-based therapeutic specialists and employees who support ARIKAYCE prescribers, to aid the global containment effort. The Company is providing remote support and engagement options to both healthcare professionals and patients prescribed ARIKAYCE.

Importantly, Insmed has observed no disruptions to date in its supply chain for the production of ARIKAYCE. The Company believes it has enough active pharmaceutical ingredient used in ARIKAYCE to meet anticipated global requirements, including commercial, clinical and compassionate use, through the end of 2022.

Balance Sheet

As of March 31, 2020, Insmed had cash and cash equivalents of $428.9 million. The Company’s total operating expenses for the first quarter of 2020 were $88.8 million. Adjusted operating expenses, as defined below, for the first quarter of 2020 were $76.3 million.

The Company plans to continue investing in the following key activities in 2020:

(i)U.S. commercialization of ARIKAYCE;
(ii)inical trial activities, including (a) the development of a PRO for NTM lung disease, the initiation of a study to validate the PRO and, in parallel, a confirmatory clinical study of ARIKAYCE, (b) the advancement of brensocatib into a Phase 3 program in patients with bronchiectasis, and (c) the advancement of treprostinil palmitil and our earlier-stage research pipeline; and
(iii)expansion in Japan and Europe to support pre-commercial activities for ARIKAYCE in those regions.

Conference Call

Insmed will host a conference call beginning today at 8:30 AM Eastern Time. Shareholders and other interested parties may participate in the conference call by dialing (888) 317-6003 (domestic) or (412) 317-6061 (international) and referencing conference ID number 2121512. The call will also be webcast live on the Company’s website at www.insmed.com.

A replay of the conference call will be accessible approximately one hour after its completion through May 14, 2020 by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and referencing replay access code 10142785. A webcast of the call will also be archived for 90 days under the Investors section of the Company’s website at www.insmed.com.

Non-GAAP Financial Measures

In addition to the U.S. generally accepted accounting principles (GAAP) results, this earnings release includes adjusted operating expenses, a non-GAAP financial measure, which Insmed defines as total operating expenses less stock-based compensation expense, depreciation, amortization of intangibles and certain milestones related to ARIKAYCE, which are payable under our amended agreements with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT). A reconciliation of this non-GAAP financial measure to its most directly comparable GAAP financial measure is presented in the table attached to this press release.

Management believes that this non-GAAP financial measure is useful to both management and investors in analyzing our ongoing business and operating performance. Management believes that providing this non-GAAP information to investors, in addition to the GAAP results, allows investors to view our financial results in the way that management views financial results. Management does not intend the presentation of this non-GAAP financial measure to be considered in isolation or as a substitute for results prepared in accordance with GAAP. In addition, this non-GAAP financial measure may differ from similarly named measures used by other companies.

About ARIKAYCE (amikacin liposome inhalation suspension)

ARIKAYCE is the first and only FDA-approved therapy indicated for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney function. Insmed’s proprietary PULMOVANCE liposomal technology enables the delivery of amikacin directly to the lungs, where liposomal amikacin is taken up by lung macrophages where the infection resides. This approach prolongs the release of amikacin in the lungs while limiting systemic exposure. ARIKAYCE is administered once daily using the Lamira Nebulizer System manufactured by PARI Pharma GmbH (PARI).

About PARI Pharma and the Lamira Nebulizer System

ARIKAYCE (amikacin liposome inhalation suspension) is delivered by a novel inhalation device, the Lamira Nebulizer System, developed by PARI. Lamira is a quiet, portable nebulizer that enables efficient aerosolization of liquid medications, including liposomal formulations such as ARIKAYCE, via a vibrating, perforated membrane. Based on PARI’s 100-year history working with aerosols, PARI is dedicated to advancing inhalation therapies by developing innovative delivery platforms and new pharmaceutical formulations that work together to improve patient care.

About Brensocatib (Formerly INS1007)

Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase I (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs.

IMPORTANT SAFETY INFORMATION FOR ARIKAYCE IN THE U.S.

WARNING: RISK OF INCREASED RESPIRATORY ADVERSE REACTIONS

ARIKAYCE has been associated with an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease that have led to hospitalizations in some cases.

Hypersensitivity Pneumonitis has been reported with the use of ARIKAYCE in the clinical trials. Hypersensitivity pneumonitis (reported as allergic alveolitis, pneumonitis, interstitial lung disease, allergic reaction to ARIKAYCE) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (3.1%) compared to patients treated with a background regimen alone (0%). Most patients with hypersensitivity pneumonitis discontinued treatment with ARIKAYCE and received treatment with corticosteroids. If hypersensitivity pneumonitis occurs, discontinue ARIKAYCE and manage patients as medically appropriate.

Hemoptysis has been reported with the use of ARIKAYCE in the clinical trials. Hemoptysis was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (17.9%) compared to patients treated with a background regimen alone (12.5%). If hemoptysis occurs, manage patients as medically appropriate.

Bronchospasm has been reported with the use of ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma, bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea exertional, prolonged expiration, throat tightness, wheezing) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (28.7%) compared to patients treated with a background regimen alone (10.7%). If bronchospasm occurs during the use of ARIKAYCE, treat patients as medically appropriate.

Exacerbations of underlying pulmonary disease has been reported with the use of ARIKAYCE in the clinical trials. Exacerbations of underlying pulmonary disease (reported as chronic obstructive pulmonary disease (COPD), infective exacerbation of COPD, infective exacerbation of bronchiectasis) have been reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (14.8%) compared to patients treated with background regimen alone (9.8%). If exacerbations of underlying pulmonary disease occur during the use of ARIKAYCE, treat patients as medically appropriate.

Anaphylaxis and Hypersensitivity Reactions: Serious and potentially life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients taking ARIKAYCE. Signs and symptoms include acute onset of skin and mucosal tissue hypersensitivity reactions (hives, itching, flushing, swollen lips/tongue/uvula), respiratory difficulty (shortness of breath, wheezing, stridor, cough), gastrointestinal symptoms (nausea, vomiting, diarrhea, crampy abdominal pain), and cardiovascular signs and symptoms of anaphylaxis (tachycardia, low blood pressure, syncope, incontinence, dizziness). Before therapy with ARIKAYCE is instituted, evaluate for previous hypersensitivity reactions to aminoglycosides. If anaphylaxis or a hypersensitivity reaction occurs, discontinue ARIKAYCE and institute appropriate supportive measures.

Ototoxicity has been reported with the use of ARIKAYCE in the clinical trials. Ototoxicity (including deafness, dizziness, presyncope, tinnitus, and vertigo) were reported with a higher frequency in patients treated with ARIKAYCE plus background regimen (17%) compared to patients treated with background regimen alone (9.8%). This was primarily driven by tinnitus (7.6% in ARIKAYCE plus background regimen vs 0.9% in the background regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background regimen vs 2.7% in the background regimen alone arm). Closely monitor patients with known or suspected auditory or vestibular dysfunction during treatment with ARIKAYCE. If ototoxicity occurs, manage patients as medically appropriate, including potentially discontinuing ARIKAYCE.

Nephrotoxicity was observed during the clinical trials of ARIKAYCE in patients with MAC lung disease but not at a higher frequency than background regimen alone. Nephrotoxicity has been associated with the aminoglycosides. Close monitoring of patients with known or suspected renal dysfunction may be needed when prescribing ARIKAYCE.

Neuromuscular Blockade: Patients with neuromuscular disorders were not enrolled in ARIKAYCE clinical trials. Patients with known or suspected neuromuscular disorders, such as myasthenia gravis, should be closely monitored since aminoglycosides may aggravate muscle weakness by blocking the release of acetylcholine at neuromuscular junctions.

Embryo-Fetal Toxicity: Aminoglycosides can cause fetal harm when administered to a pregnant woman. Aminoglycosides, including ARIKAYCE, may be associated with total, irreversible, bilateral congenital deafness in pediatric patients exposed in utero. Patients who use ARIKAYCE during pregnancy, or become pregnant while taking ARIKAYCE should be apprised of the potential hazard to the fetus.

Contraindications: ARIKAYCE is contraindicated in patients with known hypersensitivity to any aminoglycoside.

Most Common Adverse Reactions: The most common adverse reactions in Trial 1 at an incidence ≥5% for patients using ARIKAYCE plus background regimen compared to patients treated with background regimen alone were dysphonia (47% vs 1%), cough (39% vs 17%), bronchospasm (29% vs 11%), hemoptysis (18% vs 13%), ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%), musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs 10%), exacerbation of underlying pulmonary disease (15% vs 10%), diarrhea (13% vs 5%), nausea (12% vs 4%), pneumonia (10% vs 8%), headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash (6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs 1%), and chest discomfort (5% vs 3%).

Drug Interactions: Avoid concomitant use of ARIKAYCE with medications associated with neurotoxicity, nephrotoxicity, and ototoxicity. Some diuretics can enhance aminoglycoside toxicity by altering aminoglycoside concentrations in serum and tissue. Avoid concomitant use of ARIKAYCE with ethacrynic acid, furosemide, urea, or intravenous mannitol.

Overdosage: Adverse reactions specifically associated with overdose of ARIKAYCE have not been identified. Acute toxicity should be treated with immediate withdrawal of ARIKAYCE, and baseline tests of renal function should be undertaken. Hemodialysis may be helpful in removing amikacin from the body. In all cases of suspected overdosage, physicians should contact the Regional Poison Control Center for information about effective treatment.

U.S. INDICATION

LIMITED POPULATION: ARIKAYCEÒ is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients.

This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Limitation of Use: ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.

Patients are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. You can also call the Company at 1-844-4-INSMED.

On April 30, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported that it will present its first quarter 2020 financial results and operational highlights in a conference call on May 7, 2020 at 8 a.m. ET (Press release, Intellia Therapeutics, APR 30, 2020, View Source [SID1234556849]).

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!

To join the call:

U.S. callers should dial 1-877-317-6789 and use conference ID# 10141541, approximately five minutes before the call.
International callers should dial 1-412-317-6789 and use conference ID# 10141541, approximately five minutes before the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section of the company’s website at www.intelliatx.com, beginning on May 7, 2020 at 12 p.m. ET.