1stOncology™: Cancer Intelligence Service – Page 9994 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Ultragenyx Reports Fourth Quarter and Full Year 2020 Financial Results and Corporate Update

On February 11, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter and full year ended December 31, 2020 and reaffirmed its financial guidance for 2021 (Press release, Ultragenyx Pharmaceutical, FEB 11, 2021, View Source [SID1234574923]).

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!

"Last year was a transformative year as we received two approvals, generated clinically meaningful data in our gene therapy and antisense oligonucleotide programs, and completed several strategic business development transactions," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In addition to growing our global commercial footprint in 2021, we will also have a strong, diverse, late-stage clinical development portfolio. We will initiate three pivotal gene therapy studies, a fourth pivotal study with our recently in-licensed biologic, setrusumab, for osteogenesis imperfecta, make additional progress in the Angelman syndrome study, and bring our first mRNA program to the clinic for glycogen storage disease type III."

Fourth Quarter and Full Year 2020 Financial Results

Net Revenues
For the fourth quarter of 2020, Ultragenyx reported $91.5 million in total revenue. Ultragenyx recognized $40.4 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $37.5 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $2.9 million. Total royalty revenue related to European Crysvita sales were $3.6 million. Dojolvi (triheptanoin) product sales in the fourth quarter of 2020 were $6.4 million. Mepsevii (vestronidase alfa) product sales for the fourth quarter of 2020 were $3.7 million. Total revenue for the fourth quarter also includes $37.5 million of revenue related to the collaboration and license agreement with Daiichi Sankyo that was executed in March 2020.

Revenue for the year ended December 31, 2020 was $271.0 million, including $138.9 million in Crysvita revenue in the Ultragenyx territories. Crysvita collaboration revenue in the North American profit share territory was $128.6 million and net Crysvita product sales in other regions were $10.4 million. Total royalty revenue related to European Crysvita royalties was $14.5 million, which includes $1.5 million recognized on sales that occurred prior to January 1, 2020. Dojolvi product revenue for the year ended December 31, 2020 was $13.0 million. Mepsevii product revenue was $15.3 million. For the year ended December 31, 2020, revenue related to our strategic manufacturing partnership with Daiichi Sankyo was $89.2 million.

Operating Expenses
Total operating expenses for the fourth quarter of 2020 were $187.6 million, including non-cash stock-based compensation of $22.8 million. This compares to total operating expenses for the fourth quarter of 2019, which were $130.0 million and total non-cash stock-based compensation of $19.7 million.

Total operating expenses for the year ended December 31, 2020 were $601.1 million, which includes $25.0 million to maintain the option to acquire GeneTx, $13.2 million from a one-time in-process R&D expense related to the strategic collaboration with Solid Biosciences, $7.0 million to license certain vectors from REGENXBIO, and non-cash stock-based compensation of $85.7 million. This is compared with $527.9 million for the same period in 2019, which includes a $15.6 million research and development expense from the Arcturus collaboration amendment, $20.0 million for the upfront payment on the GeneTx agreement, and non-cash stock-based compensation of $82.0 million. The increase in total operating expenses was due to the increase in commercial, on-going clinical and preclinical development, and general and administrative costs as the company commercializes, grows, and advances its portfolio.

For the fourth quarter of 2020, Ultragenyx reported net loss of $24.0 million, or $0.37 per share basic and diluted, compared with a net loss for the fourth quarter of 2019 of $93.8 million, or $1.62 per share, basic and diluted. For the year ended December 31, 2020, net loss was $186.6 million, or $3.07 per share, basic and diluted, compared with a net loss for the same period in 2019 of $402.7 million, or $7.12 per share, basic and diluted. The net loss for the fourth quarter of 2020 and the net loss for the year ended December 31, 2020 includes a $79.1 million and $170.4 million increase in the fair value of investments in equity securities, respectively. Net cash used in operations for the year ended December 31, 2020 was $132.2 million, compared to net cash used of $345.4 million for the same period in 2019.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were $1.2 billion as of December 31, 2020.

2021 Financial Guidance

Crysvita Guidance in Ultragenyx Territories
The company reaffirms the 2021 guidance range for Crysvita that was provided at the beginning of the year. This range is $180 million to $190 million for the North American profit share region and the other regions where product sales are recognized (Latin America and Turkey).

Program Updates and Upcoming Milestones

Dojolvi for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): Launched on July 22, 2020

Five months into the United States launch of Dojolvi for LC-FAOD, the company has received approximately 190 completed start forms from approximately 90 unique prescribers. This has led to approximately 130 patients on reimbursed therapy as of the end of December, a 117% increase over the prior quarter.
In Canada, Dojolvi has been granted priority review by Health Canada and a new drug submission was filed in August 2020, with a decision expected soon. Dojolvi has been submitted to the Brazilian Health Regulatory Agency (ANVISA) seeking marketing authorization. Discussions with the European Medicines Agency (EMA) are ongoing. Responses to named patient treatment requests continue in the EU.
Three global, pivotal stage gene therapy clinical studies are expected in 2021

DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Following Scientific Advice with the EMA and an End of Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA), the company currently expects to initiate a Phase 3 study in the first half of 2021.
DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Based on initial Scientific Advice from the EMA and following a planned EOP2 meeting with the FDA, the company currently expects to initiate a Phase 3 study in the second half of 2021.
UX701 for Wilson disease: The Investigational New Drug (IND) application has cleared and the program recently received Fast Track Designation from the FDA. The company currently expects to initiate a seamless, single-protocol Phase 1/2/3 study in the first half of 2021.
GTX-102 for Angelman Syndrome, partnered with GeneTx Biotherapeutics: Study expected to resume in first half 2021 with additional data anticipated in second half of 2021

GeneTx, with Ultragenyx support, submitted to the FDA a substantial information amendment to the IND including follow-up safety information for the five patients dosed and toxicology data in nonhuman primates that demonstrate no evidence of this safety issue at higher repeat dosing. GeneTx has received feedback and questions from the FDA based on this submission and GeneTx and Ultragenyx have filed a response to FDA and are working on an amendment to the protocol.
A Clinical Trial Application (CTA) has been submitted in Canada and a protocol and information amendment similar to what has been proposed to FDA will be submitted. GeneTx, with Ultragenyx support, is in the process of expanding the study to other countries using the amended dosing and administration plan.
The companies currently expect the Phase 1/2 study to resume enrollment and dosing in the first half of 2021, following resolution of FDA requests and approval to proceed.

UX143 (setrusumab) for Osteogenesis Imperfecta, in collaboration with Mereo BioPharma: Phase 2/3 in pediatric patients anticipated in second half of 2021

The companies expect to conduct a Phase 2/3 study in pediatric patients that first focuses on determining the optimal dose based on increases in bone production via changes in serum P1NP levels over two months and an acceptable safety profile. Following determination of the dose, the study is intended to transition into a pivotal Phase 3 study, evaluating fracture reduction over an estimated 15 to 24 months. Final study design and endpoints are pending regulatory review.
UX053 for Glycogen Storage Disease Type III or Debrancher deficiency: IND on track in the first half of 2021

An IND for the company’s first mRNA program, UX053, for the treatment of GSDIII debrancher deficiency is on track for the first half of 2021, with a Phase 1/2 study expected to initiate in the second half of 2021.
1: Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, February 11, 2021, at 2 p.m. PT/ 5 p.m. ET to discuss the fourth quarter and year 2020 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 6391931. The replay of the call will be available for one year.

Myriad Genetics to Participate in Multiple Upcoming Health and Technology Conferences

On February 11, 2021 Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, reported that it will participate at multiple upcoming health and technology conferences, sharing insights on how the company is intensifying its focus on serving patients and healthcare providers in Women’s Health, Oncology and Mental Health (Press release, Myriad Genetics, FEB 11, 2021, View Source [SID1234574922]).

Paul J. Diaz, president and CEO at Myriad Genetics, and R. Bryan Riggsbee, CFO, will participate in a fireside chat at the BTIG Virtual MedTech, Digital Health, Life Science & Diagnostic Tools Conference on February 19 at 10:30 a.m. EST.

On February 24, 2021, Mr. Riggsbee will participate in a fireside chat at the Leerink Global Healthcare Conference at 5:00 p.m. EST.

On March 2, 2021, Mr. Diaz will participate in a fireside chat at the Cowen Annual Healthcare Conference at 9:50 a.m. EST.

The presentations will be available through a live audio webcast link in the investor information section of Myriad’s website at www.myriad.com.

Dynavax to Report Fourth Quarter and Full Year 2020 Financial Results and Host Conference Call on February 25, 2021

On February 11, 2021 Dynavax Technologies Corporation (Nasdaq: DVAX), a biopharmaceutical company focused on developing and commercializing vaccines, reported fourth quarter and full year 2020 financial results on Thursday, February 25, 2021, after the U.S. financial markets close (Press release, Dynavax Technologies, FEB 11, 2021, View Source [SID1234574921]).

Dynavax will host a conference call and live audio webcast on Thursday, February 25, 2021 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 View Source Alternatively, participants may dial (866) 420-4066 (domestic) or (409) 217-8237 (international) and refer to conference ID 8380559. A replay of the webcast will be available for 30 days following the live event.

Veracyte to Present at BTIG Virtual MedTech, Digital Health, Life Science & Diagnostic Tools Conference

On February 11, 2021 Veracyte, Inc. (Nasdaq: VCYT), a global genomic diagnostics company, reported that Bonnie Anderson, chairman and chief executive officer, will participate in a fireside chat at the BTIG Virtual MedTech, Digital Health, Life Science & Diagnostic Tools Conference on Thursday, February 18, at 1:30 p.m. Eastern Time (Press release, Veracyte, FEB 11, 2021, View Source [SID1234574920]).

The link to the live audio webcast of the company’s presentation will be available by visiting Veracyte’s website at View Source A replay of the webcast will be available for 90 days following the conclusion of the live presentation broadcast.

Seagen Reports Fourth Quarter and Full Year 2020 Financial Results

On February 11, 2021 Seagen Inc. (Nasdaq:SGEN) reported financial results for the fourth quarter and year ended December 31, 2020 (Press release, Seagen, FEB 11, 2021, View Source [SID1234574919]). The Company also highlighted ADCETRIS (brentuximab vedotin), PADCEV (enfortumab vedotin-ejfv) and TUKYSA (tucatinib) commercial and development accomplishments, as well as progress with its lead pipeline programs to treat cancer.

"In 2020, we generated record product revenues of more than $1 billion driven by successful launches of PADCEV and TUKYSA, as well as continued growth of ADCETRIS," said Clay Siegall, Ph.D., President and Chief Executive Officer of Seagen. "These strong results are in addition to several significant business, regulatory and development accomplishments during the year. Looking ahead, we are focused on maximizing the global potential of our approved medicines, advancing our late-stage programs towards securing approvals of new products, and expanding our innovative early-stage pipeline. In collaboration with our partner Genmab we submitted a BLA for tisotumab vedotin to the FDA, positioning it to be our fourth commercial product. We are on track for several other milestones across our pipeline over the course of 2021, and are in a strong financial position to continue executing against our goals."

COMMERCIAL PRODUCT HIGHLIGHTS

ADCETRIS

Presented 5-year Follow-up Results for ECHELON-1 and ECHELON-2 Trials at the ASH (Free ASH Whitepaper) Annual Meeting: Key long-term updates for two phase 3 ADCETRIS trials were presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting. Notably, the five-year update of the phase 3 ECHELON-1 clinical trial showed treatment with ADCETRIS in combination with AVD (Adriamycin [doxorubicin], vinblastine and dacarbazine) resulted in superior long-term outcomes when compared to ABVD, which includes bleomycin, in frontline advanced Hodgkin lymphoma.
PADCEV

Reporting Phase 3 EV-301 and EV-201 Cohort 2 Data in Oral Presentations at ASCO (Free ASCO Whitepaper) GU; Regulatory Submissions Planned: Full results from the phase 3 EV-301 trial and the second cohort of the pivotal phase 2 EV-201 trial will be presented at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Genitourinary Cancers Symposium (ASCO-GU) being held February 11-13. Both trials were conducted in patients with previously treated locally advanced or metastatic urothelial cancer. The EV-301 data are intended to support global registration submissions planned for the first quarter of 2021. The results from the second cohort of EV-201 will support a supplemental Biologics License Application (BLA) submission with the U.S. Food and Drug Administration (FDA) planned for the first quarter of 2021.
Initiated Second Phase 3 Trial in Muscle Invasive Bladder Cancer (MIBC): In collaboration with Astellas and Merck, the randomized phase 3 EV-304/KEYNOTE-B15 trial was initiated to evaluate PADCEV in combination with KEYTRUDA (pembrolizumab) in cisplatin-eligible MIBC. This is in addition to the ongoing randomized phase 3 study, EV-303/KEYNOTE-905, in cisplatin-ineligible MIBC patients.
TUKYSA

Received Positive CHMP Opinion for TUKYSA: In December 2020, the European Medicines Agency adopted a positive opinion recommending the approval of TUKYSA in combination with trastuzumab and capecitabine for the treatment of adult patients with HER2-positive locally advanced or metastatic breast cancer who have received at least 2 prior anti-HER2 treatment regimens. The CHMP positive opinion is now being considered by the European Commission, which has the authority to approve medicines in the European Union.
Presented Multiple TUKYSA Abstracts at SABCS: In December 2020, eight abstracts highlighted Seagen’s commitment to addressing unmet needs in breast cancer at the San Antonio Breast Cancer Symposium (SABCS). One presentation provided new exploratory analyses from the pivotal HER2CLIMB trial in metastatic HER2-positive breast cancer that demonstrated progression-free survival, overall survival and objective response rate improvements with TUKYSA were observed consistently across hormone receptor status subgroups, including in patients with brain metastases.
PIPELINE HIGHLIGHTS

Submitted Tisotumab Vedotin BLA to FDA: In February 2021, Seagen and Genmab submitted a BLA to the FDA seeking accelerated approval of tisotumab vedotin for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. Results from the innovaTV 204 trial formed the basis of the submission.
Initiated Phase 3 Trial of Tisotumab Vedotin: A phase 3 trial, called innovaTV 301, was initiated to evaluate tisotumab vedotin compared to chemotherapy in patients with recurrent or metastatic cervical cancer who have received one or two prior lines of therapy. innovaTV 301 is intended to support global regulatory applications and potentially serve as a confirmatory trial in the U.S.
Treated First Patient in Phase 1 Trial of SGN-STNV: In January 2021, the first patient was dosed in a phase 1 trial of SGN-STNV, a novel antibody-drug conjugate (ADC) targeting Sialyl Thomsen-nouveau (STN). STN is highly expressed across multiple solid tumors.
For additional information on Seagen’s pipeline, visit www.seagen.com/science/pipeline.

FOURTH QUARTER AND FULL YEAR 2020 FINANCIAL RESULTS

Revenues: Total revenues in the fourth quarter and year ended December 31, 2020 increased to $601.3 million and $2.2 billion, respectively, compared to $289.8 million and $916.7 million for the same periods in 2019. Revenues were comprised of the following three components:

Note: Sum of product sales may not equal total net product sales due to rounding.

Royalty Revenues: Royalty revenues in the fourth quarter were $39.2 million, compared to $72.3 million in the fourth quarter of 2019. The fourth quarter of 2019 included a $40 million milestone from Takeda triggered by annual net sales exceeding $400 million in Takeda’s territory during 2019. Royalty revenues were $126.8 million for the full year in 2020, compared to $138.5 million in 2019. Royalty revenues are primarily driven by sales of ADCETRIS outside the U.S. and Canada by Takeda and, to a lesser extent, also include royalties from sales of POLIVY (polatuzumab vedotin) by Roche and BLENREP (belantamab mafodotin) by GlaxoSmithKline, which are ADCs that use Seagen technology.
Collaboration and License Agreement Revenues: Amounts earned under the Company’s collaboration and license agreements were $267.9 million in the fourth quarter and $1.0 billion for the full year in 2020, compared to $51.1 million and $150.2 million, respectively, for the same periods in 2019. The fourth quarter of 2020 included $250.1 million in revenue related to the premium over market price paid by Merck under a $1 billion stock purchase that closed in October 2020 as part of the ladiratuzumab vedotin collaboration agreement. The full year in 2020 included $975.1 million in license revenue related to the Merck collaborations for ladiratuzumab vedotin and TUKYSA that were entered into in September 2020.
Cost of Sales: Cost of sales in the fourth quarter were $61.8 million, compared to $11.9 million in the fourth quarter of 2019. Cost of sales were $217.7 million for the full year in 2020, compared to $44.0 million for the same period in 2019. The increases in 2020 were primarily due to the gross profit share payment to Astellas based on PADCEV sales, which were $32.1 million and $104.6 million in the fourth quarter and full year, respectively. Cost of sales for the periods in 2020 also reflect amortization of acquired in-process technology costs that began with the approval of TUKYSA in April 2020, and third-party royalties owed for ADCETRIS, PADCEV and TUKYSA net product sales. Cost of sales for the year in 2020 included a payment owed to a third-party technology licensor related to the TUKYSA license agreement with Merck reflected in the third quarter of the year.

Research and Development (R&D) Expenses: R&D expenses in the fourth quarter were $216.2 million, compared to $201.1 million in the fourth quarter of 2019. R&D expenses were $827.1 million for the full year in 2020, compared to $719.4 million in 2019. The increase in 2020 primarily reflected continued investment in the Company’s pipeline.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses in the fourth quarter were $158.4 million, compared to $115.2 million in the fourth quarter of 2019. SG&A expenses were $533.8 million for the full year in 2020, compared to $373.9 million for the same period in 2019. The increases were primarily attributed to increased field sales personnel in the U.S. for Seagen’s recently commercialized products, PADCEV and TUKYSA, as well as higher infrastructure costs to support the Company’s continued growth and international expansion.

Non-cash share-based compensation cost for the full year in 2020 was $147.2 million, compared to $127.3 million for the same period in 2019.

Net Income / Loss: Net income for the fourth quarter of 2020 was $167.1 million, or $0.90 per diluted share, compared to net income of $25.8 million, or $0.14 per diluted share, for the fourth quarter of 2019. For the full year in 2020, net income was $613.7 million, or $3.37 per diluted share, compared to a net loss of $158.7 million, or $0.96 per diluted share, for the year in 2019. Net income for 2020 was driven by the revenue recognized in the third and fourth quarters under the Merck collaborations. Seagen utilized federal net operating loss carryforwards as allowed, however the Company incurred income taxes in certain states which resulted in an income tax provision of $2.0 million for the year in 2020.

Cash and Investments: As of December 31, 2020, Seagen had $2.7 billion in cash and investments. This includes proceeds from a $1.0 billion equity investment by Merck, which closed in October 2020.

2021 FINANCIAL OUTLOOK

Seagen anticipates 2021 revenues, operating expenses and other costs to be in the ranges shown in the table below.

Revenues

1. Non-cash costs include share-based compensation, depreciation and amortization of intangible assets.

Conference Call Details

Seagen management will host a conference call and webcast with supporting slides to discuss its fourth quarter and full year 2020 financial results and provide an update on business activities. The event will be held today at 1:30 p.m. Pacific Time (PT); 4:30 p.m. Eastern Time (ET). The live event and supporting slides will be simultaneously webcast and available for replay from the Seagen website at www.seagen.com, under the Investors section. Investors may also participate in the conference call by calling 844-763-8274 (U.S.) or 412-717-9224 (international). The conference ID is 10150708. A webcast replay will be archived on the Company’s website www.seagen.com, under the Investors section.