On May 7, 2019 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, reported its financial results for the first quarter ended March 31, 2019 and provided an update on the company’s recent achievements and anticipated upcoming milestones (Press release, Audentes Therapeutics, MAY 7, 2019, View Source [SID1234535866]).

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!

"We are excited by the continued strong momentum across our business," stated Matthew R. Patterson, Chairman and Chief Executive Officer. "We recently presented new positive data from ASPIRO, the Phase 1/2 study of AT132 for the treatment of XLMTM, at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper). These data further support the compelling profile of this important product candidate and help further our progress toward the goal of making AT132 globally available to patients living with XLMTM as rapidly as possible."

Mr. Patterson continued, "In our Pompe disease program, this quarter we successfully completed an initial NHP safety study of our product candidate AT845 and the encouraging results give us added confidence as we complete GLP toxicology and dose-ranging studies to support a third quarter IND submission. Finally, we are thrilled to have recently announced the expansion of our technology platform and development pipeline with the addition of programs utilizing vectorized antisense to address Duchenne muscular dystrophy and myotonic dystrophy, two devastating neuromuscular diseases."

Recent Achievements & Upcoming Key Events

AT132 for X-Linked Myotubular Myopathy (XLMTM):

Presented new positive data from ASPIRO at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper).
The ASGCT (Free ASGCT Whitepaper) data set included safety and efficacy assessments out to 48 weeks of follow-up for six treated patients in dose Cohort 1 (1×1014 vg/kg) and 24 weeks for three treated patients in dose Cohort 2 (3×1014 vg/kg).
Patients receiving AT132 achieved reductions in ventilator dependence not previously observed in chronically ventilated patients with neuromuscular disorders, including four patients achieving ventilator independence and all other treated patients demonstrating sustained and clinically meaningful reductions in ventilator use.
The data also demonstrated sustained improvements in neuromuscular function in both dose cohorts with corresponding achievement of clinically meaningful motor milestones. Additionally, muscle biopsy data continued to show robust tissue transduction, protein expression, and histological improvements in 24 and 48-week muscle biopsies in all patients, with evidence of more rapid pathological improvement by week 24 in the Cohort 2 biopsy samples.
AT132 has been generally well-tolerated and has shown a manageable safety profile across both dose cohorts with no clinically meaningful safety differences between doses to date.
Enrollment of an additional three to five patients in Cohort 2 (3×1014 vg/kg) of ASPIRO is ongoing.
On track to select optimal dose of AT132 in the second quarter of 2019. Plan to provide an updated data package to FDA and on track to provide update on the license application submission plans for AT132 in the third quarter of 2019.
Next clinical data presentation planned at the 24th International Annual Congress of the World Muscle Society (WMS) in Copenhagen, Denmark, October 1-5, 2019.
AT845 for Pompe Disease:

Successfully completed an initial non-GLP NHP study, which showed a clean safety profile.
Previously planned GLP toxicology and dose-ranging studies in progress.
On track to file IND in the third quarter of 2019.
AT702/AT751/AT753 for Duchenne Muscular Dystrophy (DMD):

Announced exclusive license agreement with the Research Institute at Nationwide Children’s Hospital to develop AT702, an AAV-antisense candidate designed to induce exon 2 skipping for DMD caused by duplications of exon 2 and mutations in exons 1-5 of the dystrophin gene. Conducting additional preclinical work of AT702 and plan to commence a Phase 1/2 study at Nationwide Children’s in the fourth quarter of 2019.
Separate from the Nationwide Children’s collaboration, conducting preclinical work to advance AT751 and AT753, additional vectorized exon skipping candidates designed to treat DMD patients with genotypes amenable to exon 51 and exon 53 skipping. Both AT751 and AT753 utilize the same vector construct backbone as AT702, enabling a potentially accelerated path into clinical development.
Initial programs target more than 25% of patients with DMD. Plan to leverage vectorized exon skipping platform to develop further product candidates to address up to 80% of DMD patients over time.
AT466 for Myotonic Dystrophy (DM1):

Collaboration announced with Nationwide Children’s to evaluate vectorized RNA knockdown and vectorized exon skipping for DM1.
Preclinical studies are underway, and IND planned for 2020.
AT342 for Crigler-Najjar Syndrome and AT307 for CASQ2-CPVT:

Completed a strategic review of our product candidates and plan to focus on those programs that have the potential to create the greatest long-term value for patients and shareholders.
Plan to explore outlicensing opportunities to continue to advance these important and promising gene therapy programs.
Manufacturing:

Announced new internal cGMP plasmid manufacturing facility, further establishing the company’s leadership in AAV manufacturing and enabling the rapid advancement of neuromuscular programs from preclinical development to commercialization.
Made continued progress on chemistry, manufacturing, and controls (CMC) BLA and MAA-readiness efforts for AT132 based on FDA and EMA feedback on the company’s preliminary filing strategy.
Corporate:

Expanded leadership team with the additions of Mark Meltz, Senior Vice President, General Counsel, to lead the legal, compliance, and corporate governance functions and Sarah Spencer, Vice President, Corporate Communications, to lead internal and external communications to build global awareness of the company, its mission, culture, and innovative product portfolio.
First Quarter 2019 Financial Results

Cash Position: At March 31, 2019, Audentes had cash, cash equivalents and marketable securities of $375.0 million, which is expected to fund operations into 2021.
Research and Development Expenses: Research and development expenses were $39.8 million for the first quarter of 2019 compared to $19.9 million for the same period in 2018, an increase of $19.9 million. The increase in research and development expense was primarily attributable to increased R&D headcount and related facility costs, a one-time licensing fee of $7.0 million related to the AT702 program, increased internal manufacturing costs, increased clinical trial expenses for our AT132 program, increased pre-clinical and clinical trial planning costs for our AT845 program, and an increase in stock compensation expense.
General and Administrative Expenses: General and administrative expenses were $12.0 million for the first quarter of 2019 compared to $6.5 million for the same period in 2018, an increase of $5.5 million. The increase in general and administrative expenses was primarily attributable to increased headcount and related facility costs, increased professional service and consulting fees, higher costs related to public company regulatory compliance and increase in stock compensation expense.
Net Loss: Net loss was $49.4 million for the first quarter of 2019 compared to $25.6 million for the same period in 2018.
Conference Call
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its first quarter 2019 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investors + Media section of the Audentes website at www.audentestx.com. Alternatively, please call (833) 659-8620 (U.S.) or (409) 767-9247 (international) and dial the conference ID# 6839198 to access the call.

A replay of the webcast will be available on the Audentes website for approximately 30 days.

On May 7, 2019 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL), reported financial results for the first quarter ended March 31, 2019, including sales of TAVALISSE (fostamatinib disodium hexahydrate), for the treatment of thrombocytopenia in adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment (Press release, Rigel, MAY 7, 2019, View Source [SID1234535865]).

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!

"Since the U.S. launch of TAVALISSE one year ago, a growing number of physicians are utilizing our product in earlier lines of therapy and with an increasing number of their chronic ITP patients," stated Raul Rodriguez, president and CEO. "Internationally, the recent collaboration with Grifols in Europe enhances our potential access to a majority of the ex-U.S. ITP market. Meanwhile, our pipeline is expanding with the initiation of our Phase 3 trial in warm autoimmune hemolytic anemia and additional ongoing clinical and research projects."

Financial Update

For the first quarter of 2019, Rigel reported a net loss of $17.6 million, or $0.11 per share, compared to a net loss of $24.4 million, or $0.17 per share, in the same period of 2018.

For the first quarter of 2019, Rigel reported total revenues of $12.6 million, consisting of $8.1 million in net product sales of TAVALISSE and $4.6 million in contract revenues from collaborations. The increase in net product sales of TAVALISSE reflects the continuing growth of its business since commercial launch in May 2018. There were no net product sales nor contract revenues from collaborations in the first quarter of 2018.

Contract revenues from collaborations of $4.6 million during the three months ended March 31, 2019 primarily related to a portion of the $30.0 million upfront fee recognized as revenue upon delivery of license rights to Grifols, S.A. and Rigel’s performance of certain research and development services. There were no contract revenues from collaborations during the three months ended March 31, 2018.

Rigel reported total costs and expenses of $31.0 million in the first quarter of 2019, compared to $24.7 million for the same period in 2018. The increase in costs and expense was primarily due to the increases in personnel costs, including its customer-facing and medical affairs teams, and third-party costs to support Rigel’s commercialization of TAVALISSE.

As of March 31, 2019, Rigel had cash, cash equivalents and short-term investments of $127.9 million, compared to $128.5 million as of December 31, 2018.

Business Update

Rigel’s launch of TAVALISSE in the U.S. continued to build upon its early success with growth driven by expansion of the prescriber base, steady use of the product as an early treatment option in steroid refractory patients, physicians prescribing to an increased number of patients, and strong reimbursement from the payer community.
Rigel advanced the regulatory review of fostamatinib in chronic ITP by responding to day-120 questions from the European Medicines Agency (EMA) and remains on track for potential approval in the EU by the end of 2019.
In January, Rigel entered into a collaboration with Grifols, S.A. for the rights to fostamatinib in all indications in Europe and Turkey enabling a commercial launch preparation well ahead of the anticipated approval date.
Kissei Pharmaceuticals, Rigel’s collaborator in Japan, has initiated discussions with the Pharmaceuticals and Medical Devices Agency (PMDA) to define a path towards fostamatinib approval in Japan.
In warm AIHA, Rigel opened clinical trial sites for its pivotal Phase 3 clinical study of fostamatinib. The clinical trial protocol calls for approximately 80 patients in a 24-week study with enrollment of the first patient expected this month. Topline trial results are projected in early 2021, positioning fostamatinib to potentially be the first FDA-approved treatment for this indication.
The company’s research and development team continues to investigate potential molecules that modulate the immune system, including R835, Rigel’s IRAK 1/4 inhibitor currently in Phase 1 development. In addition, Rigel has four partnered programs that are in Phase 1 and 2 of their clinical development and continue to advance.
About ITP
In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPOs) and splenectomy. However, not all patients are adequately treated with existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
AIHA is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 40,000 adult patients in the U.S. and can be a severe, debilitating disease. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients.

About R8351
The investigational candidate, R835, is an orally available, potent and selective inhibitor of IRAK1 and IRAK4 that has been shown preclinically to block inflammatory cytokine production in response to toll-like receptor (TLR) and the interleukin-1 (IL-1R) family receptor signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to a variety of inflammatory conditions. R835 is active in multiple rodent models of inflammatory disease including psoriasis, arthritis, lupus, multiple sclerosis and gout. The safety and efficacy of R835 has not been established by the FDA or any healthcare authority.

Conference Call and Webcast with Slides Today at 4:30PM Eastern Time
Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing 855-892-1489 (domestic) or 720-634-2939 (international) and using the Conference ID number 3999559. The webcast, with slide presentation, can be accessed from Rigel’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (e.g., rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (e.g., digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.
Please see www.TAVALISSE.com for full Prescribing Information.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

On May 7, 2019 Genomic Health, Inc. (NASDAQ: GHDX) reported financial results and business progress for the quarter ended March 31, 2019 (Press release, Genomic Health, MAY 7, 2019, View Source [SID1234535864]).

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!

"In the first quarter of 2019, we delivered more than 17 percent overall revenue growth and $13.0 million in profit driven by significant growth across all key product areas," said Kim Popovits, chairman of the board, chief executive officer and president of Genomic Health. "Our first quarter performance was very strong, based in part on the impact of the landmark TAILORx trial results, which are continuing to drive increased Oncotype DX Breast Recurrence Score test usage both in the United States and globally. We expect to achieve double-digit revenue growth for the year with continued growth across key products and look forward to the national reimbursement decision in Germany for the Oncotype DX breast cancer test."

First Quarter Financial Results

Total revenue for the first quarter of 2019 was $108.8 million, compared with $92.6 million for the first quarter of 2018, an increase of 17.4 percent, and an increase of 18.0 percent on a non-GAAP constant currency basis. U.S. product revenue was $91.0 million for the first quarter of 2019, compared with $78.9 million for the first quarter of 2018, an increase of 15.4 percent.

Revenue delivered across key product areas was as follows:

U.S. invasive breast revenue from Oncotype DX Breast Recurrence Score tests was $79.8 million for the first quarter of 2019, compared with $71.0 million for the first quarter of 2018, an increase of 12.5 percent.
U.S. prostate test revenue from Oncotype DX Genomic Prostate Score (GPS) tests was $8.5 million for the first quarter of 2019, compared with $5.8 million for the first quarter of 2018, an increase of 46.6 percent.
International product revenue for the first quarter of 2019 was $17.8 million, compared with $13.8 million for the first quarter of 2018, an increase of 29.0 percent, and an increase of 32.9 percent on a non-GAAP constant currency basis.
Net income was $13.0 million, or $0.35 and $0.34 per share on a basic and diluted basis, respectively, for the first quarter of 2019, an improvement of $16.8 million, compared with a net loss of $3.8 million, or $0.11 per share on a basic and diluted basis, for the first quarter of 2018. Operating income was $11.5 million for the first quarter of 2019, an improvement of $15.9 million, compared with an operating loss of $4.4 million for the first quarter of 2018.

More than 37,580 Oncotype test results were delivered in the first quarter of 2019, an increase of 15.9 percent, compared with more than 32,440 test results delivered in the same period in 2018. Tests delivered across key product areas was as follows:

Oncotype DX Breast Recurrence Score tests delivered in the U.S. grew 9.4 percent in the first quarter of 2019, compared with the same period in 2018.
Oncotype DX Genomic Prostate Score tests delivered in the U.S. grew 24.5 percent in the first quarter of 2019, compared with the same period in 2018.
Oncotype DX international tests delivered grew 28.1 percent in the first quarter of 2019, compared with the same period in 2018 and represented approximately 25 percent of total test volume in the first quarter of 2019.
Recent Business Highlights

Presented results from five studies at the 16th St. Gallen International Breast Cancer Conference, including real-world evidence that reinforces the treatment paradigm established by the TAILORx trial. Additionally, two decision impact studies from the UK and the Czech Republic highlighted the value of Oncotype DX in personalizing and improving the quality of clinical decisions in patients with early-stage breast cancer, including those with node positive disease .
Multiple private insurers established new coverage for the Oncotype DX Genomic Prostate Score test, bringing the total number of U.S. covered lives to more than 114 million, including Medicare.
First private insurer established reimbursement for the Oncotype DX AR-V7 Nucleus Detect test, bringing the total number of U.S. covered lives to more than 61 million, including Medicare.
The National Comprehensive Cancer Network (NCCN) strengthened its prostate cancer guidelines, recommending the consideration of AR-V7 testing in metastatic castrate resistant prostate cancer (mCRPC) patients following abiraterone/enzalutamide to help guide the selection of further therapy.
Conference Call Details
To access the live conference call today, May 7, 2019, at 4:30 p.m. Eastern Time via phone, please dial (877) 303-7208 from the United States and Canada, or +1 (224) 357-2389 internationally. The conference call ID is 6535029. Please dial in approximately 10 minutes prior to the start of the call. To access the live and subsequently archived webcast of the conference call, go to the Investor Relations section of the company’s website at View Source Please connect to the website at least 15 minutes prior to the presentation to allow for any software download that may be necessary.

On May 7, 2019 Penumbra, Inc. (NYSE: PEN), a global healthcare company focused on innovative therapies, reported financial results for the first quarter ended March 31, 2019 (Press release, Penumbra, MAY 7, 2019, View Source [SID1234535863]).

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!

Revenue of $128.4 million in the first quarter of 2019, an increase of 25.1%, or 27.2% in constant currency1, over the first quarter of 2018.
First Quarter 2019 Financial Results
Total revenue grew to $128.4 million for the first quarter of 2019 compared to $102.7 million for the first quarter of 2018, an increase of 25.1%, or 27.2% on a constant currency basis. The United States represented 64% of total revenue and international represented 36% of total revenue for the first quarter of 2019. Revenue from sales of neuro products grew to $81.5 million for the first quarter of 2019, an increase of 14.1%, or 16.5% on a constant currency basis. Revenue from sales of vascular products grew to $47.0 million for the first quarter of 2019, an increase of 50.2%, or 51.8% on a constant currency basis.

Gross profit was $83.9 million, or 65.3% of total revenue, for the first quarter of 2019, compared to $66.6 million, or 64.8% of total revenue, for the first quarter of 2018.

Total operating expenses for the first quarter of 2019 were $72.8 million, or 56.6% of total revenue. This compares to total operating expenses of $62.5 million, or 60.9% of total revenue, for the first quarter of 2018. R&D expenses were $11.7 million for the first quarter of 2019, compared to $8.0 million for the first quarter of 2018. SG&A expenses were $61.1 million for the first quarter of 2019, compared to $54.5 million for the first quarter of 2018.

Operating income for the first quarter of 2019 was $11.2 million, compared to operating income of $4.0 million for the first quarter of 2018.

Webcast and Conference Call Information
Penumbra, Inc. will host a conference call to discuss the first quarter 2019 financial results after market close on Tuesday, May 7, 2019 at 4:30 PM Eastern Time. The conference call can be accessed live over the phone by dialing (866) 393-4306 for domestic callers or (734) 385-2616 for international callers (conference id: 6096838), or the webcast can be accessed on the "Events" section under the "Investors" tab of the Company’s website at: www.penumbrainc.com. The webcast will be available on the Company’s website for at least two weeks following the completion of the call.

On May 7, 2019 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the first quarter of 2019 and reaffirmed 2019 financial guidance (Press release, Jazz Pharmaceuticals, MAY 7, 2019, Jazz Pharmaceuticals Announces First Quarter 2019 Financial Results [SID1234535862]).

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!

"In the first quarter of 2019, we delivered strong top- and bottom-line growth and continued our efforts to bring innovative and life-changing medicines to patients, with FDA approval of Sunosi for EDS associated with narcolepsy or OSA, launch of Xyrem in pediatric narcolepsy and announcement of positive top-line results from our Phase 3 study of JZP-258," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "As the year progresses, we are continuing to invest in our business to support the successful launch of Sunosi in the U.S. and pre-launch activities in the EU, to generate data for our existing products and to fuel further advancement and diversification of our pipeline."

Financial Highlights

GAAP net income for the first quarter of 2019 was $85.2 million, or $1.47 per diluted share, compared to $46.0 million, or $0.75 per diluted share, for the first quarter of 2018.

Adjusted net income for the first quarter of 2019 was $213.2 million, or $3.67 per diluted share, compared to $182.4 million, or $2.98 per diluted share, for the first quarter of 2018. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.

Key Regulatory/R&D Updates

In March 2019, the company announced that the U.S. Food and Drug Administration (FDA) approved Sunosi (solriamfetol) to improve wakefulness in adult patients with EDS associated with narcolepsy or OSA. Sunosi is the first and only dual-acting dopamine and norepinephrine reuptake inhibitor approved by the FDA for this indication and was approved with strengths of 75 mg and 150 mg for patients with narcolepsy and 37.5 mg, 75 mg, and 150 mg for patients with OSA.

In March 2019, the company announced positive top-line results from the global, double-blind, placebo-controlled, randomized-withdrawal, multi-center Phase 3 study evaluating the efficacy and safety of JZP-258 for the treatment of cataplexy and EDS in adult patients with narcolepsy. JZP-258 is a novel oxybate product candidate with a 92% reduction in sodium content compared to Xyrem.

In March 2019, positive results from DEFIFrance, an observational, multi-center, post-marketing study in adult and pediatric patients treated with defibrotide at hematopoietic stem cell transplant centers in France, were presented at the European Society for Blood and Marrow Transplant (EBMT) meeting.

Select 2019 Milestones

Programs

2019 Milestones*

Xyrem (sodium oxybate) oral solution

Launched in March for the treatment of cataplexy or EDS in pediatric narcolepsy

JZP-258

Announced positive top-line results in March from the Phase 3 narcolepsy study

Expect to submit top-line results from the Phase 3 narcolepsy study to a fall medical meeting

Pre-New Drug Application (NDA) meeting with FDA

Goal to submit NDA as early as year-end

Sunosi (solriamfetol)

FDA approval on March 20 for EDS in narcolepsy or OSA

Drug Enforcement Administration (DEA) scheduling decision by late second quarter

Initiate Sunosi launch following DEA scheduling decision

Announce new Phase 3 development program mid-year

Obtain EU approval for EDS in narcolepsy or OSA as early as year-end

Vyxeos (daunorubicin and cytarabine) liposome for injection

Presentation by Children’s Oncology Group at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (relapsed/refractory pediatric acute myeloid leukemia (AML) study data)

Potential interim combination data results from MD Anderson collaboration

Finalized protocol for Phase 1/2 study (low-dose Vyxeos in combination with venetoclax); patient enrollment is expected to begin in the second half of the year

Defitelio (defibrotide sodium) / defibrotide

Presentation of positive results from DEFIFrance study at EBMT in March

Conduct interim analysis of the Phase 3 study for prevention of hepatic veno-occlusive disease (VOD)

Complete enrollment in prevention of acute graft-vs-host disease Phase 2 study

Initiate exploratory Phase 2 study in chimeric antigen receptor t-cell therapy associated neurotoxicity

Initiate Phase 2 study in transplant-associated thrombotic microangiopathy

Asparaginase

Provide informational update on early-stage recombinant crisantaspase program later this year

CombiPlex

Continue Investigational New Drug enabling activities for one solid tumor combination and progress exploratory activities for other hematology/oncology candidates

* Milestones denoted as • have been completed; all other milestones are planned or expected in 2019.

Other Developments

In March 2019, the company launched Xyrem to treat cataplexy and EDS in pediatric narcolepsy patients following receipt of FDA approval in October 2018 after completing implementation of the Risk Evaluation and Mitigation Strategy to include pediatric patients and their caregivers.

In April 2019, the company announced the finalization of the settlement agreement with the U.S. Department of Justice (DOJ) related to the company’s support of charitable organizations that provide financial assistance to Medicare patients. In 2018, the company had announced an agreement in principle and recorded a total expense of $58.2 million related to this matter, including related interest. Under the settlement agreement, in April 2019, the company paid $57.0 million plus interest and entered into a five-year corporate integrity agreement with the Office of Inspector General of the Department of Health and Human Services.

Total revenues increased 14% in the first quarter of 2019 compared to the same period in 2018.

Xyrem net product sales increased 16% in the first quarter of 2019 compared to the same period in 2018.

Erwinaze/Erwinase net product sales increased 20% in the first quarter of 2019 due to an increase in product availability compared to the same period in 2018. The company continues to expect supply disruptions throughout 2019 which will cause inter-quarter variability in Erwinaze net sales.

Defitelio/defibrotide net product sales increased 18% in the first quarter of 2019 compared to the same period in 2018 due to increased use by transplant centers that treat adult and pediatric patients. VOD is an ultra-rare disease and, as a result, the company continues to expect inter-quarter variability in Defitelio net sales.

Vyxeos net product sales increased 10% in the first quarter of 2019 compared to the same period in 2018 primarily due to the rolling launch in the EU initiated in September 2018. The company continues its education and outreach initiatives and its efforts to generate data to support Vyxeos’ potential use across broader patient populations in AML and other hematological malignancies.

Operating expenses changed over the prior year period primarily due to the following:

Selling, general and administrative (SG&A) expenses on a GAAP basis decreased in the first quarter of 2019 compared to the same period in 2018 primarily due to a $57.0 million loss contingency recorded in 2018 related to the DOJ matter described above. SG&A expenses on a GAAP basis, excluding the impact of the loss contingency, and on a non-GAAP adjusted basis increased in the first quarter of 2019 compared to the same period in 2018 primarily due to higher expenses related to the planned launch of Sunosi in the U.S. and an increase in headcount and compensation-related expenses to support expansion of the business.
Research and development (R&D) expenses on a GAAP basis decreased in the first quarter of 2019 compared to the same period in 2018 primarily due to milestone payments of $11.0 million related to FDA acceptance for filing of the company’s solriamfetol NDA recorded in 2018. R&D expenses on a GAAP basis, excluding the impact of milestone payments, and on a non-GAAP adjusted basis increased in the first quarter of 2019 compared to the same period in 2018 primarily due to expenses related to the company’s pre-clinical and clinical development programs, including partner programs, regulatory activities and related headcount increases to support these efforts.
Cash Flow and Balance Sheet

As of March 31, 2019, cash, cash equivalents and investments were $832.5 million and the outstanding principal balance of the company’s long-term debt was $1.8 billion. During the first quarter of 2019, the company generated $202.3 million of cash from operations, made an upfront payment of $56.0 million to Codiak BioSciences, Inc. under a collaboration agreement and used $111.2 million to repurchase shares.

In the first quarter of 2019, the company repurchased approximately 858,000 ordinary shares under the company’s share repurchase program at an average cost of $129.66 per ordinary share. As of March 31, 2019, the remaining amount authorized for share repurchases was $267.9 million.

2019 Financial Guidance

Includes minimal net sales contribution from Sunosi in the U.S., assuming launch in mid-2019.

Excludes $6-$8 million of share-based compensation expense from estimated GAAP gross margin.

Excludes $82-$90 million of share-based compensation expense from estimated GAAP SG&A expenses.

Excludes $0-$34 million of milestone payments and $22-$27 million of share-based compensation expense from estimated GAAP R&D expenses.

Excludes the income tax effect of adjustments between GAAP reported and non-GAAP adjusted net income.

See "Non-GAAP Financial Measures" below. Reconciliations of non-GAAP adjusted guidance measures are included above and in the table titled "Reconciliation of GAAP to Non-GAAP Adjusted 2019 Net Income Guidance" at the end of this press release.

Conference Call Details

Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. EDT (9:30 p.m. IST) to provide a business and financial update and discuss its 2019 first quarter results. The live webcast may be accessed from the Investors section of the company’s website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 6667859.

A replay of the conference call will be available through May 14, 2019 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 6667859. An archived version of the webcast will be available for at least one week in the Investors section of the company’s website at www.jazzpharmaceuticals.com.