On Februuary 21, 2019 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and wet age-related macular degeneration, reported that it will report its fourth quarter and full year 2018 financial and operating results after the close of U.S. financial markets on Thursday, February 28, 2019 (Press release, Tracon Pharmaceuticals, FEB 21, 2019, View Source [SID1234533582]). In addition, management will host a conference call to provide an update on corporate activities and discuss the quarterly financial results.

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Conference call and webcast:
Date: February 28, 2019
Time: 4:30 pm Eastern Time (1:30 pm Pacific Time)
Dial-in: (855) 779-9066 (Domestic) or (631) 485-4859 (International)
Passcode: 7886794
Via web: www.traconpharma.com; "Events and Presentations" section within the "Investors" section
A replay of the webcast will be available for 60 days on the website.

On February 21, 2019 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported financial results for the fourth quarter and full year ended December 31, 2018 and provided an update on recent corporate activities (Press release, Halozyme, FEB 21, 2019, View Source [SID1234533533]).

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"Our latest collaboration with argenx demonstrates the broad applicability of our ENHANZE drug delivery technology and is a great way to start the year," said Dr. Helen Torley, president and chief executive officer. "We anticipate multiple additional key milestones in our ENHANZE business including potential approval of Herceptin SC later this quarter and potential regulatory submissions for a subcutaneous formulation of Darzalex in the second half of the year. With a total of nine ENHANZE collaborations spanning from established products to new, innovative therapies addressing unmet needs, we remain confident in the potential for $1 billion in royalty revenue in 2027."

"Our PEGPH20 oncology program achieved a critical milestone during the fourth quarter with the completion of enrollment in our pivotal HALO-301 pancreas cancer study with approximately 500 patients. We also reached agreement with the FDA to change the primary endpoint for HALO-301 to a single primary endpoint of overall survival (OS), which we believe incrementally de-risked the study. We look forward with excitement to topline results from HALO-301, which we currently project in the second half of 2019."

Fourth Quarter 2018 and Recent Highlights Include:

In February 2019, Halozyme licensed its ENHANZE drug delivery technology to argenx providing exclusive access to ENHANZE for any product targeting the human neonatal Fc receptor FcRn, including argenx’s lead asset efgartigimod (ARGX-113), and up to two additional targets. Under the terms of the agreement, argenx paid an upfront payment of $30 million to Halozyme, and will pay Halozyme $10 million per target for future target nominations and potential future payments of up to $160 million per selected target subject to achievement of specified development, regulatory and sales-based milestones. Halozyme will also receive mid-single digit royalties on sales of commercialized products.

In January 2019, the U.S. Food and Drug Administration completed its review of the submitted clinical study protocol amendment and statistical analysis plan for HALO-301, which included a change in the primary endpoint to a single primary endpoint of overall survival (OS), with no additional questions or comments.

In December, enrollment in HALO-301, the company’s Phase 3 study evaluating PEGPH20 in metastatic pancreas cancer, was completed with approximately 500 subjects enrolled. The company projects the study will achieve its target of 330 OS events between August and November of 2019. Based on achieving this timeline, the company projects topline results will be available in the second half of 2019.

In December, Roche dosed the first patient in a Phase 1b/2 study of Tecentriq (atezolizumab) with ENHANZE triggering a $5 million milestone payment to Halozyme.

During the fourth quarter, BMS began recruitment for a Phase 1 study of OPDIVO (nivolumab) with ENHANZE.

ENHANZE partner Janssen continued to make progress in clinical studies for a subcutaneous co-formulation of Darzalex (daratumumab) with the recent initiation of two additional Phase 3 trials. Janssen is planning regulatory filings in the second half of 2019.

In October, Halozyme expanded its collaboration with Roche by licensing its ENHANZE drug-delivery technology for exclusive development of a new undisclosed clinical stage therapeutic target resulting in an upfront payment of $25 million.

Fourth Quarter and Full Year 2018 Financial Highlights

Revenue for the fourth quarter was $60.2 million compared to $189.6 million for the fourth quarter of 2017. The year-over-year decrease was driven by $141.4 million upfront license fees for the BMS and Alexion agreements and a $15.0 million milestone payment from Janssen recognized in 2017, compared to $30.0 million in upfront and milestone revenue for the Roche collaboration recognized in 2018. The decrease was offset by a 9 percent growth in royalties on a reported basis from partner sales. Revenue for the fourth quarter included $19.3 million in royalties and $4.2 million in HYLENEX recombinant (hyaluronidase human injection) product sales.
Revenue for the full year was $151.9 million, compared to $316.6 million in 2017.
Revenue from royalties for the full year was $79.0 million, up 24% on an as-reported basis compared to $63.5 million in 2017.

Research and development expenses for the fourth quarter were $36.7 million, compared to $41.4 million for the fourth quarter of 2017.
Research and development expenses for the full year were $150.3 million, compared to $150.6 million in 2017.

Selling, general and administrative expenses for the fourth quarter were $18.0 million, compared to $14.8 million for the fourth quarter of 2017.
Selling, general and administrative expenses for 2018 were $60.8 million, compared to $53.8 million in 2017.

Net loss for the fourth quarter was $2.1 million, or $0.01 per share, compared to net income in the fourth quarter of 2017 of $123.9 million, or $0.85 per share.
Net loss for the full year was $80.3 million, or $0.56 per share, compared to net income of $63.0 million in 2017, or $0.45 per share.

Cash, cash equivalents and marketable securities were $354.5 million at December 31, 2018, compared to $469.2 million at December 31, 2017.

Financial Outlook for 2019

Halozyme updated its 2019 financial guidance, first provided on January 9, 2019, to reflect the recent argenx collaboration and license agreement:

Net revenue of $205 million to $215 million, excluding revenue from any additional, new ENHANZE global collaboration and licensing agreements;

Operating expenses of $265 million to $275 million, or $225 million to $235 million excluding an expected increase in cost of goods sold. Excluding the cost of goods sold the modest increase in expenses is driven by ENHANZE partner support, and support of the potential commercialization of PEGPH20;

Operating cash burn of $45 million to $55 million;

Debt repayment of approximately $90 million, the company expects to pay off the remainder of the royalty-backed debt by the end of the first quarter of 2020;

Year-end cash, cash equivalents and marketable securities balance of $210 million to $220 million.

Webcast and Conference Call
Halozyme will webcast its Quarterly Update Conference Call for the fourth quarter of 2018 today, Thursday, February 21 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Torley will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website and a replay will be available following the close of the call. To access the webcast and additional documents related to the call, please visit halozyme.com approximately fifteen minutes prior to the call to register, download and install any necessary audio software. The call may also be accessed by dialing (877) 410-5657 (domestic callers) or (334) 323-7224 (international callers) using passcode 387156. A telephone replay will be available after the call by dialing (877) 919-4059 (domestic callers) or (334) 323-0140 (international callers) using replay ID number 68892505.

On February 21, 2019 MorphoSys AG (FSE: MOR; Prime Standard Segment, MDAX & TecDAX; NASDAQ: MOR) reported that it will present at the following conference (Press release, MorphoSys, FEB 21, 2019, View Source [SID1234533550]):

8th Annual Leerink Partners Global Healthcare Conference
Date: February 28, 2019, 2:30 pm EST (8:30 pm CET, 7:30 pm GMT)
Venue: New York, NY, USA
Presenter: Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG
Dr. Julia Neugebauer, Director Corporate Communications & IR

PDF versions of the presentations will be provided at www.morphosys.com. The link to the webcasts will be filed under www.morphosys.com/conference-calls.

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On February 21, 2019 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the full year and fourth quarter of 2018 (Press release, BioMarin, FEB 21, 2019, View Source [SID1234533562]). Net Loss for 2018 decreased $39.8 million or 34%, to $77.2 million, compared to $117.0 million in 2017. Net Loss for the quarter ended December 31, 2018 decreased to $3.6 million, compared to Net Loss of $51.4 million, for same period in 2017.

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The change in Net Loss for the full year and fourth quarter of 2018, compared to the same periods in 2017 was primarily due to the following:

a year over year increase in gross profits of $104.0 million driven by increased sales volume across all of our products, a $4.1 million quarter over quarter decrease in gross profits driven by a decrease in Aldurazyme sales volume; and
an increase in the benefit from income taxes. During 2018, the Company recognized a $65.5 million benefit from income taxes primarily attributed to Orphan Drug Credits earned in the year, whereas in 2017 it recognized income tax expense of $81.2 million primarily driven by U.S. tax reform; partially offset by
higher research and development (R&D) expense for the expansion of BioMarin’s clinical programs related to valoctocogene roxaparvovec, vosoritide and tralesinidase alfa and higher selling, general and administrative (SG&A) expense in support of the U.S. commercial launch of Palynziq and European (EU) pre-launch activities, the continued commercial expansion of Brineura and market preparation activities related to the Company’s valoctocogene roxaparvovec product candidate; and
a decrease in the gain on the sale of intangible assets. During the third and fourth quarters of 2018, the Company received milestone payments of $20.0 million and $30.0 million, respectively. The milestone payments were triggered by a third-party’s attainment of development and regulatory approval milestones related to a previously sold intangible asset. In 2017, BioMarin sold the Priority Review Voucher it received in connection with the FDA approval of Brineura and recognized the $125.0 million of proceeds as a gain on the sale of intangible assets.
Non-GAAP Income for 2018 increased $16.9 million, or 23%, to $90.9 million, compared to $74.0 million in 2017. Non-GAAP Loss for the quarter ended December 31, 2018 was $10.8 million, compared to Non-GAAP Income of $5.2 million in the quarter ended December 31, 2017. The change in Non-GAAP Income/Loss for the full year 2018 was attributed to increased gross profit from sales partially offset by higher expenses as described above. The change in Non-GAAP Income/Loss quarter to quarter was attributed to decreased gross profit and higher expenses as described above.

Net product revenues for 2018 increased 14% to $1.5 billion, compared to $1.3 billion in 2017. The increase in net product revenues for the full year, is attributed to increased sales across all of our products despite quarter to quarter volatility driven by central government ordering patterns. The increase by product was:

Vimizim: increased $68.7 million, or 17%, primarily driven by new patients initiating therapy and government ordering patterns;
Aldurazyme: increased $45.1 million, or 50%, of which $20.2 million is due to the different revenue recognition principles applied as a result of BioMarin’s adoption of Accounting Standards Codification 606, Revenue from Contracts with Customers, (ASC 606), and $24.9 million due an increase in sales volume;
Brineura: contributed $31.3 million to increased net product revenues, primarily attributed to new patients initiating therapy as the product was commercially launched in mid-2017;
Kuvan: increased $26.1 million, or 6%, primarily due to an increase in patients initiating therapy in North America;
Naglazyme: increased $13.7 million, or 4%, primarily driven by new patients initiating therapy in Turkey and North America and government ordering patterns in the Middle East, partially offset by a decrease due to the impact of government ordering patterns from certain Latin American countries; and
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 in 2018 totaled $12.2 million primarily driven by the conversion of clinical patients to commercial Palynziq in the U.S.
Net product revenues for the fourth quarter of 2018 were $347.2 million, compared to $353.6 million in the fourth quarter of 2017. The decrease in net product revenues in the fourth quarter of 2018 compared to the fourth quarter of 2017 was primarily attributed to decreased Naglazyme net product revenues driven by the volatility of central government ordering patterns and lower Aldurazyme sales volume driven by timing of shipments to Genzyme, partially offset by the first full quarter of Palynziq commercial sales and increased Brineura net product revenues.

As of December 31, 2018, BioMarin had cash, cash equivalents and investments totaling approximately $1.3 billion, as compared to $1.8 billion on December 31, 2017. On October 15, 2018, our 0.75% senior subordinated convertible notes matured and were settled with a combination of $375.0 million in cash for the full principal amount and cash in lieu of fractional shares plus the issuance of 190,220 common stock for the conversion value in excess of the principal.

Commenting on 2018 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "BioMarin’s achievements over the last 12 months have prepared us for a number of key catalysts across the product portfolio in 2019. In the second quarter of 2018, we received FDA approval of Palynziq, an important new therapy that helps address a significant unmet need in adults with phenylketonuria (PKU). As of February 15, 2019, 335 PKU patients were being treated in the U.S. with reimbursed Palynziq. Looking forward, we expect to hear the status of our European marketing authorization application in the first quarter of 2019. We are hopeful that PKU patients in the European Union will have the opportunity to benefit from Palynziq should we receive approval in that region later this year."

"In May, we provided two years of clinical data from the Phase 2 study with the 6e13 vg/kg dose of valoctocogene roxaparvovec gene therapy for severe hemophilia A that demonstrated the elimination of need for prophylaxis and no spontaneous bleeds. In addition, we amended the protocol of the global GENEr8-1 (Phase 3) pivotal study by increasing the number of participants from 40 to 130 in order to evaluate superiority compared to the current standard of care. We now anticipate completing enrollment during the third quarter of 2019. Based on draft guidance from the FDA for hemophilia gene therapy products published in 2018, we communicated our interest in exploring a potential accelerated approval pathway with valoctocogene roxaparvovec. We plan to decide in the second half of 2019 whether we will pursue an accelerated approval path."

Mr. Bienaimé continued, "In November of 2018, we showcased a number of our other pipeline and research programs at BioMarin’s annual Research and Development Day. Specifically, we provided a 42-month update on vosoritide for the treatment of achondroplasia that our ongoing Phase 2 study demonstrated an average additional cumulative height gain of 5.7 centimeters. Based on these results, we are encouraged that vosoritide could potentially be the first approved treatment option for children with achondroplasia. Finally, we were pleased to share initial pre-clinical data for BMN 307, our gene therapy product for PKU, which demonstrated lifetime normalization of Phe in a validated PKU mouse model. We plan to complete preclinical studies in the first half of 2019 with an anticipated IND filing planned for the second half of 2019."

Full-Year 2019 Financial Guidance (in millions, except %)

*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 9 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

Palynziq for PKU: With the approval in May 2018 of Palynziq in the United States, an injection to reduce blood Phe concentrations in adult patients with PKU, BioMarin added its seventh commercial product to its portfolio. As of February 15, 2019, 335 patients were on reimbursed Palynziq, with an additional 131 patients enrolled and awaiting their first treatment with commercial Palynziq. Of the 125 PKU clinics in the U.S., 80 had at least one complete patient enrollment in the REMS program as of February 15, 2019. BioMarin anticipates an opinion from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), on Palynziq in the first quarter of 2019. If the CHMP provides a positive opinion in the first quarter of 2019, then in the second quarter of 2019, it is possible that the European Commission (EC) could provide marketing authorization in the European Union.
Valoctocogene roxaparvovec gene therapy for hemophilia A: In May 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 Phase 3 GENEr8-1 study will include 130 participants and is expected to be fully enrolled in the third quarter of 2019.Draft guidelines published by the FDA in 2018 on the development of gene therapy products for the treatment of hemophilia outlined a potential accelerated approval path forward applicable to valoctocogene roxaparvovec. The Company announced in January 2019 that it had completed enrollment of the initial cohort of patients in its Phase 3 program that would be included in a potential accelerated submission. The Company plans to decide in the second half of 2019 whether it will submit a Biologics License Application through an accelerated approval pathway.
Vosoritide for children with achondroplasia: On November 7, 2018, the Company provided a 42-month update for vosoritide at R&D Day 2018. Data from the children in the ongoing Phase 2 study 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 possibly filing for marketing authorization. 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. BioMarin expects top line results from the 52-week Phase 3 study by year end 2019. Also in 2018, BioMarin began its global Phase 2 study with vosoritide in infants and young (less than 60 months old) children with achondroplasia, to determine the impact of treatment in this age group.
Tralesinidase alfa (formerly referred to as BMN 250) for MPS IIIB (Sanfilippo Syndrome, Type B): In February 2019, the Company provided an update at the Society for the Study of Inborn Errors of Metabolism (SSIEM) meeting from the Phase 1/2 trial with tralesinidase alfa. Of the seven subjects who have been treated with the 300 mg/kg weekly dose, heparan sulfate levels were normalized in the brain fluid. All subjects also experienced normalization of the enlargement of their liver and spleen. Development Quotient (DQ), a measure of cognitive function normalized to age, was also monitored. Five of the seven subjects have experienced encouraging trends in brain function based on DQ measures.
BMN 307 gene therapy product candidate for phenylketonuria (PKU): 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.
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 fourth quarter 2018 financial results today, Thursday, February 21, 2019 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.

On February 21, 2019 Alder BioPharmaceuticals, Inc. (NASDAQ:ALDR), a biopharmaceutical company focused on developing novel therapeutic antibodies for the treatment of migraine, reported that Bob Azelby, Alder’s president and chief executive officer, will provide a business overview and update at the 8th Annual SVB Leerink Global Healthcare Conference at 11:00 am ET on Thursday, February 28, 2019 in New York, NY (Press release, Alder Biopharmaceuticals, FEB 21, 2019, View Source [SID1234533583]).

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The presentation will be webcast live on the Events & Presentations page of the Investors section of Alder’s website at View Source, or by following the link below in your web browser. An archived replay of the webcast will be available on Alder’s website for at least 30 days after the live event concludes.