On April 25, 2019 Dynavax Technologies Corporation (NASDAQ: DVAX), a fully-integrated biopharmaceutical company focused on discovering and developing novel vaccines and immuno-oncology therapeutics, reported that it will report its first quarter 2019 financial results on Wednesday, May 8, 2019, after the U.S. financial markets close (Press release, Dynavax Technologies, APR 25, 2019, View Source [SID1234535612]).

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Dynavax will host a conference call and live audio webcast on Wednesday, May 8, 2019, at 4:30 p.m. (ET)/1:30 p.m. (PT).

The live audio webcast may be accessed through the "Events & Presentations" page on the "Investors" section of the company’s website at www.dynavax.com. Alternatively, participants may dial (855) 327-6837 (domestic) or (631) 891-4304 (international) and refer to conference ID 10006654.

The archived conference call will be available on Dynavax’s website beginning approximately two hours after the event and will be archived and available for replay for at least 30 days after the event.

On April 25, 2019 AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), a specialty pharmaceutical company, reported that it will release first quarter financial results after market close on Wednesday, May 8th, 2019 (Press release, AcelRx Pharmaceuticals, APR 25, 2019, View Source [SID1234535406]). AcelRx management will host a live webcast and conference call at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) on May 8, 2019 to discuss the financial results and provide an update on the company’s business.

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The webcast is accessible by visiting the Investors page of the company’s website at www.acelrx.com and clicking on the webcast link on the Investors home page. The webcast will be accompanied by a slide presentation. A webcast replay will be available on the AcelRx website for 90 days following the call by visiting the Investor page of the company’s website at www.acelrx.com.

To access the conference call dial (888) 317-6003 for domestic callers or (412) 317-6061 for international callers and use passcode 5289662.

On April 25, 2019 Cerus Corporation (Nasdaq:CERS) reported that its first quarter 2019 results will be released on Tuesday, May 7, 2019, after the close of the stock market (Press release, Cerus, APR 25, 2019, View Source [SID1234535391]). The company will host a conference call and webcast at 4:30 P.M. ET that afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook.

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To access the live webcast, please visit the Investor Relations page of the Cerus website at View Source Alternatively, you may access the live conference call by dialing (866) 235-9006 (U.S.) or (631) 291-4549 (international).

A replay will be available on the company’s website, or by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and entering conference ID number 3932269. The replay will be available approximately three hours after the call through May 21, 2019.

On April 25, 2019 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the first quarter ended March 31, 2019 (Press release, BioMarin, APR 25, 2019, View Source [SID1234535407]).

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Total Net Product Revenues for the first quarter 2019 increased to $394.5 million, compared to $369.1 million for the first quarter of 2018. Net Product Revenues Marketed by BioMarin increased to $349.2 million, compared to $303.0 million for the first quarter of 2018. The increase in Net Product Revenues Marketed by BioMarin was attributed to the following:

Vimizim: increased $8.7 million, or 7%, primarily driven by government ordering patterns in certain Latin American, Middle Eastern and European countries. Patient growth in the quarter was robust with net patients growth increasing 12% year over year;
Kuvan: increased $7.8 million, or 8%, primarily due to new patients initiating therapy in the U.S. and sales volume in Europe;
Naglazyme: increased $11.9 million, or 16%, primarily due to increased sales volume driven by government ordering patterns from Brazil;
Palynziq: received approval from the U.S. Food and Drug Administration (FDA) in May 2018, with commercial sales launching in the third quarter of 2018. Palynziq Net Product Revenues during the first quarter of 2019 totaled $12.3 million driven primarily by the conversion of clinical patients to commercial Palynziq in the U.S.; and,
Brineura: increased $5.3 million, or 77%, primarily attributed to new patients initiating therapy in Germany and the U.S.
Aldurazyme Net Product Revenues decreased $20.8 million, or 31%, due to timing of shipments to Genzyme. Under the new revenue standards adopted in 2018, the Company records Aldurazyme Net Product Revenues when the product is released and control is transferred to Genzyme based on the estimated variable consideration payable it expects to earn when the product is sold through by Genzyme. Aldurazyme net product sales reported by Genzyme increased to $75.7 million, or up 20% in the first quarter of 2019, compared to $62.9 million the same period in 2018.

The increase in GAAP Net Loss for the first quarter of 2019, compared to the same period in 2018 was primarily due to the following:

higher selling, general and administrative (SG&A) expense in support of continued U.S. commercial launch of Palynziq and European Union (EU) pre-launch activities, other administrative expenses, consulting fees and employee-related costs to support our operations;
increased contingent consideration expense related to the progression of the Palynziq development program towards approval of the European Marketing Authorization Application, as a result of the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in the first quarter; partially offset by,
increased gross profits of $20.4 million driven by increased sales revenue from products marketed by BioMarin.
Non-GAAP Income for the first quarter of 2019 increased $3.5 million, or 16%, to $24.8 million, compared to $21.3 million for the same period in 2018. The increase in Non-GAAP Income for the quarter was attributed to increased gross profit from sales partially offset by higher SG&A expenses as described above.

As of March 31, 2019, BioMarin had cash, cash equivalents and investments totaling approximately $1.2 billion, as compared to $1.3 billion on December 31, 2018.

Commenting on first quarter results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "We begin 2019 well-positioned for potential significant achievements across our late-stage product pipeline, as well as record Total Revenues for the full year. Starting with valoctocogene roxaparvovec gene therapy for severe hemophilia A, our pivotal study is on track to complete enrollment in the third quarter. Based on draft guidance from the FDA for hemophilia gene therapy products published in 2018, we have the opportunity to pursue a potential accelerated approval path forward based on Factor VIII activity results from a subset of Phase 3 subjects. Based on the results from the subset of Phase 3 subjects, we will make and communicate our decision on whether or not to pursue an accelerated approval before the end of the year. We have observed a high level of interest and enthusiasm from the hemophilia community in support of valoctocogene roxaparvovec and are hopeful that it will be a potential treatment option for these patients in the very near future."

Mr. Bienaimé continued, "We have a number of other exciting catalysts on the horizon including the potential approval and launch of Palynziq in Europe later this year. We have been thrilled with the pace of the U.S. launch, as we ended the first quarter with 414 patients on reimbursed Palynziq. Building on this success and as part of our strategy to increase our leadership in the PKU market, we anticipate filing an IND for BMN 307, our gene therapy product for PKU, in the second half of 2019. BMN 307 demonstrated lifetime normalization of Phe in a validated PKU mouse model, and as a result we believe it has the potential to be an important new treatment and market expander as part of our PKU franchise. Finally, we look forward with great anticipation to the results of our global Phase 3 program with vosoritide for the treatment of achondroplasia. Our ongoing Phase 2 study has so far demonstrated an average additional cumulative height gain of 5.7 centimeters over 42 months. Based on data observed to date, we are very encouraged that vosoritide could potentially be the first approved treatment option for children with achondroplasia."

*All Financial Guidance items are calculated based on U.S. GAAP with the exception of Non-GAAP Income/Loss. Refer to Non-GAAP Information beginning on page 8 of this press release for a complete discussion of the Company’s Non-GAAP financial information and reconciliations to the comparable GAAP reported information.

Key Program Highlights

Valoctocogene roxaparvovec gene therapy for hemophilia A: During the quarter, the Company announced that it had enrolled the subset of subjects from the ongoing Phase 3 study that could potentially be used to support submission of a marketing application through the accelerated approval pathway. In addition, BioMarin has completed the process qualification manufacturing campaigns required as part of a BLA submission for accelerated approval. The Company intends to make a decision on whether or not to pursue a potential accelerated approval path and communicate that decision before the end of 2019. In 2018, the Company updated the protocol for the Phase 3 GENEr8-1 study evaluating the 6e13 vg/kg dose and has statistically powered the study results to evaluate superiority to the current standard of care, Factor VIII prophylaxis. The complete Phase 3 GENEr8-1 study will include 130 participants, and is expected to be fully enrolled in the third quarter of 2019.
Palynziq for PKU: Palynziq, an injection to reduce blood Phe concentrations in adult patients with PKU, was added to BioMarin’s commercial product portfolio upon its U.S. approval last May. As of March 31, 2019, 414 patients were on reimbursed Palynziq, with an additional 140 patients enrolled and awaiting their first treatment with commercial Palynziq. Of the 414 patients on therapy at the end of the first quarter, 278 were formerly naïve patients and 136 transitioned from clinical studies. Of the 125 PKU clinics in the U.S., 89 had at least one complete patient enrollment in the REMS program as of March 31, 2019.BioMarin received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), on Palynziq on March 1, 2019. Based on this positive opinion, the Company anticipates marketing authorization in the European Union by the end of the second quarter of this year.
Vosoritide for children with achondroplasia: The Company expects top line results from the ongoing global, Phase 3 study by year-end 2019. The vosoritide development program includes four distinct areas of focus to support global approval, including a large contemporaneous natural history study which is underway. The global Phase 3 study, which is fully enrolled, is a randomized, placebo-controlled study of vosoritide in approximately 110 children with achondroplasia between the ages of 5 to 14 years. The Company most recently updated its ongoing global Phase 2 study in children ages 5 to 14, which demonstrated an average of 5.7 centimeters of cumulative additional height gained at 42 months. BioMarin expects to have over 5 years of clinical data from this study to corroborate maintenance of effect at the time of anticipated marketing authorization submissions.In 2018, BioMarin began a global Phase 2 study with vosoritide in infants and young children (less than 60 months old) with achondroplasia, to determine the impact of treatment in this age group. Three cohorts, segmented by age, are being enrolled in this study. Cohort 1 includes children ages 24 to 60 months old and will complete enrollment this year. Cohort 2 includes children ages 6 to 24 months old and is currently enrolling. Following evaluation of safety and pharmacokinetics in sentinel subjects in cohort 2, cohort 3 will begin enrolling infants up to 6 months old.
Tralesinidase alfa (formerly referred to as BMN 250) for MPS IIIB (Sanfilippo Syndrome, Type B): Tralesinidase alfa is currently being evaluated in ongoing natural history and clinical trials. Previously, encouraging signs of biochemical and clinical efficacy have been suggested. Trials are ongoing to collect further data in regard to the untreated natural history of the condition, as well as biochemical and clinical outcomes of therapy.
BMN 307 gene therapy product candidate for phenylketonuria (PKU): As previously announced, the Company expects to submit an investigational new drug application (IND) and/or a clinical trial application (CTA) for a gene therapy product for the treatment of PKU in the second half of 2019. At R&D Day 2018, BioMarin shared data with BMN 307 that demonstrated a lifetime Phe correction sustained at 80 weeks in preclinical mouse models. BMN 307 is an AAV vector containing the DNA sequence that codes for the phenylalanine hydroxylase enzyme that is deficient in people with PKU. Product to support clinical evaluation will be produced at BioMarin’s Leveroni facility using a commercial scale manufacturing process to facilitate rapid clinical development.
BMN 290 for Friedreich’s Ataxia: BMN 290 is a selective chromatin modulation therapy intended for the treatment of Friedreich’s ataxia. Currently, there are no approved disease modifying therapies for Friedreich’s ataxia. The Company is currently conducting additional pre-clinical work on BMN 290 and will decide in the first half of 2019 whether to file an IND based on the outcome of those data.
BioMarin will host a conference call and webcast to discuss first quarter 2019 financial results today, Thursday, April 25, 2019 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.

U.S. / Canada Dial-in Number: 866.502.9859

Replay Dial-in Number: 855.859.2056

International Dial-in Number: 574.990.1362

Replay International Dial-in Number: 404.537.3406

Conference ID: 9996637

Conference ID: 9996637

On April 25, 2019 Bristol-Myers Squibb Company (NYSE:BMY) reported results for the first quarter of 2019 which were highlighted by strong demand for Opdivo (nivolumab) and Eliquis (apixaban) and a robust operating performance across the portfolio (Press release, Bristol-Myers Squibb, APR 25, 2019, View Source [SID1234535392]).

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"We had a very good first quarter during which the company remained focused on delivering strong sales growth of our prioritized brands and continuing to advance the science in our disease areas of focus," said Giovanni Caforio, M.D., chairman and chief executive officer, Bristol-Myers Squibb. "We also achieved approval from Bristol-Myers Squibb and Celgene shareholders to move forward with the acquisition. Looking forward, we are focused on our integration planning with Celgene and creating a leading biopharma company, with potential first-in- and best-in-class medicines, to address the unmet needs of our patients and create long-term substantial growth."

FIRST QUARTER FINANCIAL RESULTS

Bristol-Myers Squibb posted first quarter 2019 revenues of $5.9 billion, an increase of 14% compared to the same period a year ago. Revenues increased 18% when adjusted for foreign exchange impact.
U.S. revenues increased 24% to $3.4 billion in the quarter compared to the same period a year ago. International revenues increased 2%. When adjusted for foreign exchange impact, international revenues increased 10%.
Gross margin as a percentage of revenue decreased from 69.5% to 68.9% in the quarter primarily due to product mix and higher excise tax, partially offset by favorable foreign exchange.
Marketing, selling and administrative expenses increased 3% to $1.0 billion in the quarter.
Research and development expenses increased 8% to $1.4 billion in the quarter.
The effective tax rate was 13.3% in the quarter, compared to 16.0% in the first quarter last year.
The company reported net earnings attributable to Bristol-Myers Squibb of $1.7 billion, or $1.04 per share, in the first quarter, compared to net earnings of $1.5 billion, or $0.91 per share, for the same period in 2018. The results for the first quarter of 2019 include $187 million of Celgene-related acquisition and integration expenses.
The company reported non-GAAP net earnings attributable to Bristol-Myers Squibb of $1.8 billion, or $1.10 per share, in the first quarter, compared to net earnings of $1.5 billion, or $0.94 per share, for the same period in 2018. An overview of specified items is discussed under the "Use of Non-GAAP Financial Information" section.
Cash, cash equivalents and marketable securities were $10.0 billion, with a net cash position of $4.0 billion, as of March 31, 2019.
ACQUISITION OF CELGENE CORPORATION

In April, the company announced its shareholders voted to approve the company’s pending acquisition of Celgene Corporation. The company continues to expect to close the acquisition in the third quarter. (link)

FIRST QUARTER PRODUCT AND PIPELINE UPDATE

Product Sales/Business Highlights

Global revenues for the first quarter of 2019, compared to the first quarter of 2018, were driven by:

Eliquis , which grew by $419 million or 28% increase
Opdivo , which grew by $290 million or 19% increase
Yervoy , which grew by $135 million or 54% increase
Orencia , which grew by 8%
Sprycel , which grew by 5%
Opdivo

Clinical

The company reported topline results from the Phase 2 CheckMate -714 trial evaluating Opdivo versus Opdivo plus Yervoy (ipilimumab) in patients with recurrent or metastatic squamous cell carcinoma of the head and neck. The study did not meet its primary endpoints.
In April, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019, the company announced four-year survival results from pooled analyses of four studies (CheckMate -017, -057, -063 and -003) in patients with previously-treated advanced non-small cell lung cancer who were treated with Opdivo. (link)
In February, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Genitourinary Cancers Symposium, the company announced new data and analysis from studies evaluating Opdivo plus Yervoy:
CheckMate -650: Results from the Phase 2 study evaluating Opdivo in combination with Yervoy in patients with metastatic castration-resistant prostate cancer. (link)
CheckMate -214: Results from the Phase 3 study evaluating Opdivo plus low-dose Yervoy in patients with previously untreated advanced or metastatic renal cell carcinoma. (link)
Eliquis

Clinical

In March, at the American College of Cardiology’s 68th Annual Scientific Session 2019, the company and its alliance partner Pfizer announced results from the Phase 4 AUGUSTUS trial evaluating Eliquis versus vitamin K antagonists in patients with non-valvular atrial fibrillation and recent acute coronary syndrome and/or undergoing percutaneous coronary intervention. The data was simultaneously published in the New England Journal of Medicine. (link)
Sprycel

Regulatory

In February, the company announced the European Commission approved Sprycel (dasatinib) in combination with chemotherapy for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia.
2019 FINANCIAL GUIDANCE

Bristol-Myers Squibb is increasing its 2019 GAAP EPS guidance range to $3.84 – $3.94 and confirming its non-GAAP EPS guidance range of $4.10 – $4.20. Both GAAP and non-GAAP guidance assume current exchange rates. Key 2019 GAAP and non-GAAP line-item guidance assumptions are:

Worldwide revenues increasing in the mid-single digits.
Gross margin as a percentage of revenue to be approximately 70% for both GAAP and non-GAAP.
Marketing, selling and administrative expenses decreasing in the mid-single digit range for both GAAP and non-GAAP.
Research and development expenses decreasing in the high-single digits for GAAP and increasing in the high-single digits for non-GAAP.
An effective tax rate of approximately 14% for GAAP and approximately 16% for non-GAAP.
The financial guidance for 2019 excludes the impact of any potential future strategic acquisitions and divestitures, including any impact of the Celgene acquisition other than expenses incurred in the first quarter of 2019, and any specified items that have not yet been identified and quantified. The non-GAAP 2019 guidance also excludes other specified items as discussed under "Use of Non-GAAP Financial Information." Details reconciling adjusted non-GAAP amounts with the amounts reflecting specified items are provided in supplemental materials available on the company’s website.

Guidance inclusive of the Celgene acquisition will be provided after the close of the transaction. The company’s previously announced sale of the UPSA consumer health business to Taisho Pharmaceutical Holdings Co., Ltd. for $1.6 billion is anticipated to be completed in July 2019.

Use of Non-GAAP Financial Information

This earnings release contains non-GAAP financial measures, including non-GAAP earnings and related EPS information, that are adjusted to exclude certain costs, expenses, gains and losses and other specified items that are evaluated on an individual basis. These items are adjusted after considering their quantitative and qualitative aspects and typically have one or more of the following characteristics, such as being highly variable, difficult to project, unusual in nature, significant to the results of a particular period or not indicative of future operating results. Similar charges or gains were recognized in prior periods and will likely reoccur in future periods, including acquisition and integration expenses, restructuring costs, accelerated depreciation and impairment of property, plant and equipment and intangible assets, R&D charges or other income resulting from up-front or contingent milestone payments in connection with the acquisition or licensing of third-party intellectual property rights, divestiture gains or losses, pension, legal and other contractual settlement charges and debt redemption gains or losses, among other items. Deferred and current income taxes attributed to these items are also adjusted for considering their individual impact to the overall tax expense, deductibility and jurisdictional tax rates. Non-GAAP information is intended to portray the results of the company’s baseline performance, supplement or enhance management, analysts and investors overall understanding of the company’s underlying financial performance and facilitate comparisons among current, past and future periods. For example, non-GAAP earnings and EPS information is an indication of the company’s baseline performance before items that are considered by us to not be reflective of the company’s ongoing results. In addition, this information is among the primary indicators that we use as a basis for evaluating performance, allocating resources, setting incentive compensation targets and planning and forecasting for future periods. This information is not intended to be considered in isolation or as a substitute for net earnings or diluted EPS prepared in accordance with GAAP and may not be the same as or comparable to similarly titled measures presented by other companies due to possible differences in method and in the items being adjusted.

Company and Conference Call Information

Bristol-Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol-Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube and Facebook. For more information about Bristol-Myers Squibb’s proposed acquisition of Celgene, please visit View Source

There will be a conference call on April 25, 2019 at 10:30 a.m. ET during which company executives will review financial information and address inquiries from investors and analysts. Investors and the general public are invited to listen to a live webcast of the call at View Source or by calling the U.S. toll free 888-254-3590 or international 720-543-0302, confirmation code: 7211894. Materials related to the call will be available at the same website prior to the conference call. A replay of the call will be available beginning at 1:45 p.m. ET on April 25, 2019 through 1:45 p.m. ET on May 9, 2019. The replay will also be available through View Source or by calling the U.S. toll free 888-254-3590 or international 720-543-0302, confirmation code: 7211894.