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]).

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"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.

On May 7, 2019 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) reported its financial and operating results for the first quarter ended March 31, 2019 (Press release, AEterna Zentaris, MAY 7, 2019, View Source [SID1234535890]).

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All Amounts are in U.S. Dollars

Highlights

In March 2019, the Board formed a Special Committee to review strategic options available to the Company and engaged Torreya Partners to assist in that process
In January 2019, European Medicines Agency granted marketing authorization for macimorelin for diagnosis of adult growth hormone deficiency
Net loss for the first quarter of 2019 $4.9 million, compared to net income of $14.4 million for the same period in 2018
As of March 31, 2019, we had $11.4 million of unrestricted cash and cash equivalents
Summary of First Quarter Results

For the three-month period ended March 31, 2019, we reported a consolidated net loss of $4.9 million, or $0.30 loss per common share, as compared with a consolidated net income of $14.4 million, or $0.88 income per common share, for the three-month period ended March 31, 2018. The $19.3 million decline in net loss results primarily from a reduction of $24.6 million in total revenues and $3.9 million increase in net finance loss, offset by a reduction of tax expense of $6.9 million and of operating expenses of $2.3 million. In January 2018, the Company received a $24.0 million cash payment under a license and assignment agreement for Macrilen (macimorelin).

Condensed Interim Consolidated Financial Statements and Management’s Discussion and Analysis

For reference, the condensed interim consolidated financial statements as at March 31, 2019 and for the three-month periods ending March 31, 2019 and 2018 and management’s discussion and analysis of financial condition and results of operations for the first quarter ended March 31, 2019, will be found at www.zentaris.com in the "Investors" section and at the Company’s profile at www.sedar.com and www.sec.gov.

The following tables set out summary consolidated financial information for the periods indicated. The results of operations for interim periods are not necessarily indicative of the results to be expected for a full year or any future period. The information presented herein does not contain disclosures required by IFRS for consolidated financial statements and should be read in conjunction with the Company’s audited annual consolidated financial statements for the year ended December 31, 2018.

On May 7, 2019 AstraZeneca reported positive results from the Phase III ASCEND trial of Calquence (acalabrutinib) in previously-treated patients with chronic lymphocytic leukaemia (CLL) (Press release, AstraZeneca, MAY 7, 2019, View Source [SID1234535787]). Results showed a statistically-significant and clinically-meaningful improvement in progression-free survival (PFS) with Calquence monotherapy compared to a combination regimen of rituximab plus physician’s choice of idelalisib or bendamustine. Importantly, the safety and tolerability of Calquence was consistent with the known profile.

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José Baselga, Executive Vice President, R&D Oncology said: "Calquence is the first BTK inhibitor to show benefit in a Phase III trial as a monotherapy compared to current standard-of-care combinations used in relapsed or refractory chronic lymphocytic leukaemia. We look forward to presenting detailed results at a forthcoming medical meeting."

Within AstraZeneca’s robust development programme for Calquence, ASCEND is the first of two Phase III CLL trials expected to read out in 2019. The second is ELEVATE-TN (ACE-CL-007) in treatment-naïve, front-line CLL. Calquence is currently approved for the treatment of adults with relapsed or refractory mantle cell lymphoma (MCL) in the US, Brazil, the UAE, and Qatar, and is being developed for the treatment of CLL and other blood cancers.

About ASCEND

ASCEND (ACE-CL-309) is a global, randomised, multicentre, open-label Phase III trial evaluating the efficacy of Calquence in previously-treated patients with CLL. In the trial, 310 patients were randomised (1:1) into two groups. Patients in the first group received Calquence monotherapy (100mg twice daily until disease progression). Patients in the second group received rituximab plus physician’s choice of idelalisib or bendamustine.1

The primary endpoint is PFS assessed by an independent review committee (IRC), and key secondary endpoints include physician-assessed PFS, IRC- and physician-assessed overall response rate and duration of response, as well as overall survival, patient reported outcomes and time to next treatment (TTNT).1

About Calquence

Calquence (acalabrutinib) was granted accelerated approval by the US Food and Drug Administration (FDA) in October 2017 for the treatment of adult patients with MCL who have received at least one prior therapy. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials

Calquence is an inhibitor of Bruton tyrosine kinase (BTK). Calquence binds covalently to BTK, thereby inhibiting its activity2. In B-cells, BTK signalling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis, and adhesion.

As part of an extensive clinical development programme, AstraZeneca and Acerta Pharma are currently evaluating Calquence in 26 clinical trials that are company-sponsored. Calquence is being developed for the treatment of multiple B-cell blood cancers including CLL, MCL, diffuse large B-cell lymphoma, Waldenstrom macroglobulinaemia, follicular lymphoma, and multiple myeloma and other haematologic malignancies. Several Phase III clinical trials in CLL are ongoing, including ASCEND, ELEVATE-TN, ELEVATE-RR (ACE-CL-006) evaluating Calquence vs. ibrutinib in previously-treated CLL and ACE-CL-311 evaluating Calquence in combination with venetoclax and obinutuzumab in previously-untreated fit patients with CLL.

About chronic lymphocytic leukaemia (CLL)

CLL is the most common type of leukaemia in adults3. In the US, it accounts for approximately one in four new cases of leukaemia, with the average age at the time of diagnosis being approximately 70 years of age4. In CLL, too many blood stem cells in the bone marrow become abnormal lymphocytes and these abnormal cells have difficulty fighting infections3. As the number of abnormal cells grows there is less room for healthy white blood cells, red blood cells and platelets3. This could result in anaemia, infection and bleeding3. B-cell receptor signalling through BTK is one of the essential growth pathways for CLL.

About AstraZeneca in haematology

Leveraging its strength in oncology, AstraZeneca has established haematology as one of four key oncology disease areas of focus. The Company’s haematology franchise includes two US FDA-approved medicines and a robust global development programme for a broad portfolio of potential blood cancer treatments. Acerta Pharma serves as AstraZeneca’s haematology research and development arm. AstraZeneca partners with like-minded science-led companies to advance the discovery and development of therapies to address unmet need.

In October 2018, AstraZeneca and Innate Pharma announced a global strategic collaboration that included Innate Pharma licensing the US commercial rights of Lumoxiti (moxetumomab pasudotox-tdfk), and with support from AstraZeneca, will continue EU development and commercialisation, pending regulatory submission and approval.

About AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance Oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On May 7, 2019 Advaxis, Inc. (NASDAQ: ADXS), a late-stage biotechnology company focused on the discovery, development and commercialization of immunotherapy products, reported participation in five upcoming industry conferences (Press release, Advaxis, MAY 7, 2019, View Source [SID1234535814]):

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BioNJ 9th Annual BioPartnering Conference

Date and Time: Wednesday, May 8, 2019 at 2:00pm ET
Venue: The Palace at Somerset Park – Gatsby Suite, Somerset, New Jersey
Presenter: Molly Henderson, Executive Vice President and Chief Financial Officer
Presentation: Corporate overview
Frontiers in Cancer Immunotherapy

Date: Tuesday, May 14, 2019
Venue: The New York Academy of Sciences, New York, New York
Presenter: Robert Petit, Ph.D., Chief Scientific Officer
Two Poster Presentations: Safety and Immunogenicity of a Personalized Neoantigen-Listeria Vaccine in Cancer Patients; and Effects of ADXS-PSA With or Without Pembrolizumab on Survival and Antigen Spreading in Metastatic, Castration-Resistant Prostate Cancer Patients (Results from KEYNOTE-046)
Immuno-Oncology Xchange

Date and Time: Wednesday, May 15, 2019 at 12:30pm ET
Venue: Seaport World Trade Center, Boston, Massachusetts
Presenter: Andres A. Gutierrez, M.D. Ph.D., Chief Medical Officer
Presentation: Challenges of First-in-Human Phase I Dose-Escalating Trials in Patients with Advanced Cancers
BIO International Convention

Date: June 3-6, 2019
Venue: Pennsylvania Convention Center, Philadelphia, Pennsylvania
Management holding partnering meetings: Ken Berlin, President and Chief Executive Officer, and Molly Henderson, Executive Vice President and Chief Financial Officer
IO Combinations 360°

Date and Time: June 20-21, 2019 at 12:00pm ET
Venue: Wyndham Historic District Hotel, Philadelphia, Pennsylvania
Presenter: J. Randolph Hecht M.D., Professor of Clinical Medicine, David Geffen School of Medicine at UCLA and Director of the UCLA Gastrointestinal Oncology Program
Presentation: A Phase 1 Dose-Escalation Study of ADXS-NEO Expressing Personalized Tumor Antigens in Subjects with Advanced Solid Tumors: Updated Results

On May 7, 2019 MannKind Corporation (NASDAQ:MNKD) reported financial results for the quarter ended March 31, 2019 (Press release, Mannkind, MAY 7, 2019, View Source [SID1234535830]).

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"In the first quarter of 2019, we executed against the United Therapeutics License and Collaboration agreement, achieving the first of four milestone payments of $12.5 million. We also continued to grow Afrezza net revenue by 49% compared to 1Q 2018 and we released new clinical data at scientific meetings that continue to differentiate Afrezza from other rapid acting insulins," said Michael Castagna, Chief Executive Officer of MannKind Corporation.

Total revenues were $17.4 million for the first quarter of 2019, reflecting Afrezza net revenue of $5.1 million and collaboration and services revenue of $12.4 million. Afrezza net revenue increased 49% compared to $3.4 million in the first quarter of 2018, primarily driven by higher product demand, a more favorable mix of cartridges and price. Collaboration and services revenue increased $12.4 million, primarily due to the United Therapeutics licensing and research agreements.

Afrezza cost of goods sold was $4.0 million for the first quarters of both 2019 and 2018. Afrezza gross profit for the first quarter of 2019 was $1.1 million, the second consecutive quarter that gross profit was recognized for Afrezza. Afrezza gross profit for the first quarter of 2018 was negative $0.6 million. The increase was primarily driven by higher Afrezza sales.

Research and development expenses for the first quarter of 2019 were $1.7 million compared to $2.6 million for the first quarter of 2018. This 37% decrease was primarily attributable to $0.4 million decreases in both lower clinical trial spending and lower personnel costs.

Selling, general and administrative expenses for the first quarter of 2019 were $25.7 million compared to $20.6 million for the first quarter of 2018. This 25% increase was primarily due to $9.3 million spent on direct-to-consumer television advertising offset by a $1.2 million decrease in personnel costs, a $0.9 million decrease in stock-based compensation expense, and a $0.7 million decrease in professional fees.

Interest expense on notes (facility financing obligation and senior convertible notes) for the first quarter of 2019 was $0.6 million compared to $1.8 million for the first quarter of 2018. This $1.2 million decrease was primarily due to a reduction in debt principal balances.

The net loss for the first quarter of 2019 was $14.9 million, or $0.08 per share compared to a $30.4 million net loss in the first quarter of 2018 or $0.25 per share. The lower net loss is mainly attributable to a $14.0 million increase in total revenues.

Cash, cash equivalents, restricted cash, and short-term investments at March 31, 2019 was $59.8 million compared to $71.7 million at December 31, 2018. The decrease was primarily due to net cash used in operating activities of $11.6 million in the first quarter of 2019, which included the receipt of a $12.5 million milestone payment from United Therapeutics.

Conference Call

MannKind will host a conference call and presentation webcast to discuss these results today at 9:00 a.m. Eastern Time. To participate in the live call by telephone, please dial (800) 289-0438 or (323) 794-2423 and use the participant passcode: 6329706. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at View Source under News & Events.

A telephone replay of the call will be accessible for approximately 14 days following completion of the call by dialing (844) 512-2921 or (412) 317-6671 and use the participant passcode: 6329706#. A replay will also be available on MannKind’s website for 14 days.