1stOncology™: Cancer Intelligence Service – Page 12699 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Acceleron Reports First Quarter 2020 Operating and Financial Results

On May 11, 2020 Acceleron Pharma Inc. (Nasdaq:XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the first quarter ended March 31, 2020 (Press release, Acceleron Pharma, MAY 11, 2020, View Source [SID1234557505]).

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!

"Acceleron is off to a strong start in 2020, propelled by one of the most eventful time periods in the Company’s history," said Habib Dable, President and Chief Executive Officer of Acceleron. "The breadth and depth of our hematology program has been on full display, owing to the publication of the MEDALIST and BELIEVE Phase 3 luspatercept trial results in the prestigious New England Journal of Medicine and the recent FDA approval of REBLOZYL for the treatment of anemia in a population of patients with lower-risk MDS. We’re thrilled that this first-in-class erythroid maturation agent, having received approvals in two distinct indications over a span of just five months, is now available in the US to patients who have long needed a new option for treating their chronic anemias."

Added Mr. Dable: "Our scientific expertise in leveraging the therapeutic potential of the TGF-beta superfamily of proteins is delivering equally exciting results in pulmonary disease. With great enthusiasm, we announced that the PULSAR Phase 2 trial of sotatercept in patients with PAH met its primary and key secondary endpoints-underscoring our belief that sotatercept could eventually alter the current treatment paradigm. Following the results, sotatercept was granted Breakthrough Therapy designation by the FDA and PRIME designation by the EMA. We look forward to presenting topline results from PULSAR in a virtual meeting of the ATS as well as interactions with health authorities as we prepare for future Phase 3 development of sotatercept on our path to potential global registration."

Program Highlights

Hematology

REBLOZYL (luspatercept-aamt): Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
REBLOZYL is the first and only U.S. FDA approved erythroid maturation agent designed to promote red blood cell production through a novel mechanism. Luspatercept-aamt is being developed to treat anemia in patients with beta-thalassemia, MDS, and MF. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.

In April, Acceleron and partner Bristol Myers Squibb announced the FDA approved REBLOZYL for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

The New England Journal of Medicine published results from the pivotal Phase 3 MEDALIST and BELIEVE trials.

In April, REBLOZYL received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the treatment of adult patients with:

transfusion-dependent anemia due to very low-, low- and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy; and

transfusion-dependent anemia associated with beta thalassemia

Bristol Myers Squibb expects to initiate the Phase 3 INDEPENDENCE trial in patients with MF on concomitant JAK 2 inhibitor therapy who require RBC transfusions by year-end 2020.

The BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta-thalassemia is ongoing, with topline results expected by year-end 2020 or early 2021.

Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with treatment-naïve, lower-risk MDS, with topline results expected in 2022.

Pulmonary

Sotatercept: Pulmonary Arterial Hypertension (PAH)
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept (RAP-011) reversed pulmonary vessel muscularization and improved indicators of right heart failure.

In January, Acceleron reported that the PULSAR Phase 2 trial in patients with PAH met its primary and key secondary endpoints. Sotatercept was generally well tolerated in the trial and adverse events observed were generally consistent with previously published data on sotatercept in other diseases.

PULSAR Phase 2 topline results are expected to be presented in an American Thoracic Society (ATS) 2020 web-based session by the end of June 2020.

Acceleron plans to host a webcast and conference call for investors and analysts following the ATS 2020 virtual presentation to discuss highlights from the PULSAR trial results.

In April, sotatercept was granted Breakthrough Therapy designation by the FDA for the treatment of patients with PAH (World Health Organization Group 1).

The EMA recently granted Priority Medicines (PRIME) designation to sotatercept for the treatment of patients with PAH.

Enrollment is ongoing in the exploratory SPECTRA trial in patients with PAH, with preliminary results expected in 2020.

Financial Results

Cash Position – Cash, cash equivalents and investments as of March 31, 2020 were $415.6 million, as compared to $453.8 million as of December 31, 2019. Based on the Company’s current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from REBLOZYL sales.

Revenue – Revenue for the first quarter of 2020 was $4.3 million, which includes $2.8 million of cost share revenue, and $1.5 million of royalty revenue from net U.S. sales of REBLOZYL. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.

Costs and Expenses – Total costs and expenses for the first quarter of 2020 were $55.9 million. This includes R&D expenses of $37.7 million and SG&A expenses of $18.3 million.

Net Loss – The Company’s net loss for the first quarter of 2020 was $50.9 million.

Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first quarter 2020 financial results and provide an update on recent corporate activities on May 11, 2020, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron First Quarter 2020 Earnings Call."

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

Castle Biosciences Announces First Quarter 2020 Results

On May 11, 2020 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported its financial results for the first quarter ended March 31, 2020 (Press release, Castle Biosciences, MAY 11, 2020, View Source [SID1234557504]).

"The Castle Biosciences team continued to perform at an exceptional level in the first quarter, with significant growth in revenue and DecisionDx-Melanoma test report volume," said Derek Maetzold, president and chief executive officer. "We are making investments in our business intended to put us in a position of strength as we continue to move through the current COVID-19 situation and execute on our strategy in the latter half of 2020 and 2021. This includes filling key positions and the initiation of clinical studies to drive additional evidence development for DecisionDx-Melanoma as well as to support the commercial launch of our two pipeline tests.

"With the recent publication of three peer-reviewed articles that document the clinical validity and impact of DecisionDx-SCC in patients diagnosed with high-risk cutaneous squamous cell carcinoma (SCC), we plan to launch this pipeline test in the third quarter of 2020. Our proprietary tests are used to inform important treatment plan decisions in early stage cancers and having the opportunity to expand our service to patients diagnosed with high-risk SCC is an important step. The planned launch of our gene expression profile test for suspicious pigmented lesions, our other near-term pipeline test, remains on track for the second half of 2020.

"The COVID-19 situation continues to evolve and brings along with it a high level of uncertainty surrounding potential future impacts. The pandemic has caused disruptions to patient flow and a significant reduction in biopsies and the number of patients with a diagnosis of cutaneous melanoma. During the first quarter, our business was not materially affected. However, from April 1, 2020 to May 6, 2020, orders for our lead product, DecisionDx-Melanoma, declined 43% from the same period in 2019, as a result of these patient flow disruptions due to COVID-19.

"In the midst of the COVID-19 pandemic, I would like to personally thank our employees for their dedication and tremendous efforts during these difficult times. We have implemented steps that focus on the health of our employees and their families and serving our customers. As a result, we have maintained access to our tests with our standard turnaround time, continuing to be, on average, less than five days from receipt of specimen to issuance of report, so that patients and their clinicians can incorporate our test results into their cancer treatment plan decisions without interruption. It is important to understand that we are able to meet the needs of the patients we serve, due to the dedication and effort of our team."

First Quarter Ended March 31, 2020, Financial Highlights

•Revenue was $17.4 million in the first quarter of 2020, compared to $8.7 million in the first quarter of 2019.

•Delivered 4,574 DecisionDx-Melanoma test reports in the first quarter of 2020, which represents 42% growth compared to the 3,232 reports delivered during the first quarter of 2019.
•New ordering clinicians for DecisionDx-Melanoma grew 43% in the first quarter, compared to the same period in 2019.
•Delivered 361 DecisionDx-UM test reports in the first quarter of 2020, which is consistent with the 360 reports during the first quarter of 2019.
•Gross margin in the first quarter of 2020 was 86%.
•Operating cash flow was $(0.3) million in the first quarter of 2020, compared to $1.3 million in the first quarter of 2019.

Cash and Cash Equivalents

As of March 31, 2020, the Company’s cash and cash equivalents totaled $98.7 million, and the outstanding principal balance on the Company’s bank term loan was $26.7 million.

COVID-19 Response and Impact

•The Company made adjustments to its laboratory operations in March designed to keep employees safe and provide uninterrupted access to its proprietary DecisionDx-Melanoma test and DecisionDx-UM test. The Company has maintained its specimen receipt to report turnaround time to on average less than 5 days.
•Based upon the analysis of the Company’s supply channel and inventory levels, the Company believes it has adequate access to reagents and consumables needed for testing patient samples and expects to continue providing normal turnaround times for delivery of its test reports.
•The Company observed a significant reduction in clinician orders of its DecisionDx-Melanoma test. Based upon results from its proprietary customer study, the Company believes that this is due to 1) an approximate 60% reduction in the number of patients seen, 2) the subsequent impact on reduced biopsies performed and 3) a reduction in melanomas diagnosed. A reduction in orders was first observed in the back half of March, and for the period of April 1 – May 6, 2020, the Company saw an overall decrease of 43% in Decision-Dx Melanoma orders, compared to the same period in 2019. The Company has observed a stabilization in the decline of orders, beginning in the second week of April.
•The DecisionDx-Melanoma dermatological clinician customers that participated in this proprietary customer study, indicated that they expect to expand office hours during the next two to three months, as they catch up on these rescheduled appointments, and that they intend to prioritize biopsies.
•In April 2020, the Company received $1.9 million in relief funds automatically allocated to Medicare providers under the Coronavirus Aid, Relief and Economic Security Act (CARES Act) and an advance payment of $8.3 million from the Centers for Medicare & Medicaid Services (CMS), which will be applied against future Medicare claims the Company submits for reimbursement later this year.

Cash and Cash Equivalents as of April 30, 2020

As of April 30, 2020, the Company had cash and cash equivalents of approximately $110 million. This amount is preliminary, subject to adjustment and based solely upon information available to the Company as of the date of this press release. It includes the $8.3 million advance payment received from CMS, which will increase the Company’s liabilities when recorded in the second quarter of 2020. This amount of cash and cash equivalents is not a comprehensive statement of the Company’s results of operations, liquidity or financial condition as of April 30, 2020, including with respect to the Company’s liabilities as of April 30, 2020, and has not been audited, reviewed or compiled by the Company’s independent registered public accounting firm. This amount is not intended to be indicative of expected cash and cash equivalents as of June 30, 2020, or other future fiscal periods. Accordingly, undue reliance should not be placed on this preliminary information, and it should be viewed in the context of all other available information regarding the Company’s results of operations, liquidity and financial condition.

Suspension of 2020 Revenue Guidance

Due to uncertainties regarding the duration and impact of the COVID-19 pandemic, the Company is suspending its previously announced annual revenue guidance for 2020.

Supplemental Revenue Information

Included in revenues for the quarters ended March 31, 2020 and 2019, were positive revenue adjustments related to tests delivered in prior periods of $3.2 million and $0.6 million, respectively. The additional positive revenue adjustments in the current year primarily relate to recognition of revenue for certain tests delivered in prior periods for which no revenue was recognizable originally but was recognized upon cash collection of payments for the tests in the current-year period.

First Quarter and Recent Clinical Evidence Updates

•Data from a systematic review and meta-analysis of the DecisionDx-Melanoma test was published in the Journal of the American Academy of Dermatology (JAAD). This meta-analysis included four study cohorts and demonstrated the strength and consistency of the test as an independent, significant predictor of recurrence and metastatic risk in patients with invasive cutaneous melanoma. Under the Strength of Recommendation Taxonomy (SORT) system, a systematic review and meta-analysis provide for the highest level of evidence for a prognostic biomarker (Level 1 evidence). The SORT system is used by the American Academy of Dermatology and other organizations to evaluate the quality, quantity and consistency of evidence supporting tests, such as DecisionDx-Melanoma.
•The development and validation of the Company’s cutaneous squamous cell carcinoma (SCC) prognostic test, DecisionDx-SCC, for patients diagnosed with high-risk cutaneous SCC, was recently published in JAAD. The results demonstrate that DecisionDx-SCC is the strongest, as well as an independent, predictor of metastatic risk relative to current SCC staging systems and can complement clinicopathologic risk factors to better stratify risk of metastasis in patients with high-risk SCC.
•Study data supporting a framework for integration of DecisionDx-SCC into risk-appropriate management of 300 high-risk cutaneous SCC patients (as defined by

NCCN), were recently published in Current Medical Research & Opinion (CMRO) and found combining DecisionDx-SCC class with American Joint Committee on Cancer (AJCC) T stage identified a group of 159 low-risk patients (Class 1, T1-T2) with a 7.5% rate of metastasis. Similarly, combining test results with Brigham and Women’s Hospital (BWH) staging identified 173 patients with a metastasis rate of 8.1%. Rates in both groups approached the rate observed for the general cutaneous SCC patient population. By comparison, Class 2B patients in the study had rates of metastasis surpassing 50%, regardless of the staging system with which it was combined, a rate that may warrant a high intensity plan with the NCCN management recommendations. Incorporation of DecisionDx-SCC results with T stage for these 300 patients with NCCN high risk features, more than 50% would have been recommended a low intensity management plan, while 34-39% would be recommended for a moderate intensity plan, and only 8% for a high intensity plan.
•Results from an intended use study conducted at the 2020 Winter Clinical Dermatology Conference involving 162 clinicians was also published as a companion article in CMRO. Using the established pre-test post-test vignette methodology, clinicians determined the treatment plan they would employ for patients with high-risk SCC. Treatment plan modalities included follow-up schedule, sentinel lymph node biopsy, nodal imaging, adjuvant radiation and adjuvant chemotherapy. Clinicians were then asked to determine the treatment plan with the addition of DecisionDx-SCC test results. The addition of a DecisionDx-SCC Class 1 test score resulted in more than a 60% reduction in treatment plan modality intensity, while a Decision-SCC Class 2B test score resulted in more than a 90% escalation in treatment plan modality intensity. Importantly, more than 95% of the changes were made in a risk appropriate manner within national guidelines for patient management.
•Another peer-reviewed manuscript demonstrating that DecisionDx-UM test results significantly impacted treatment plan recommendations for patients with uveal melanoma (UM) in a multicenter, prospective study was published in Melanoma Management. The multicenter CLEAR II study (Clinical Application of DecisionDx-UM Gene Expression Assay Results II) was designed to prospectively evaluate patterns of physician referral and metastatic surveillance regimens for UM patients who were tested with DecisionDx-UM as part of their diagnostic work up, and to compare management plans between DecisionDx-UM low-risk (Class 1) and DecisionDx-UM high-risk (Class 2) patients.

Conference Call and Webcast Details

Castle Biosciences will hold a conference call on Monday, May 11, 2020, at 4:30 p.m. Eastern time to discuss its first quarter 2020 results and provide a corporate update.

A live webcast of the conference call can be accessed here: View Source or via the webcast link on the Investor Relations page of the Company’s website (www.castlebiosciences.com). Please access the webcast at least 10 minutes before the conference call start time. An archive of the webcast will be available on the Company’s website until June 1, 2020.

To access the live conference call via phone, please dial 877-282-2581 from the United States and Canada, or +1 470-495-9479 internationally, at least 10 minutes prior to the start of the call, using the conference ID 5699079.

There will be a brief Question & Answer session following management commentary.

SANGAMO THERAPEUTICS REPORTS BUSINESS HIGHLIGHTS AND
FIRST QUARTER 2020 FINANCIAL RESULTS

On May 11, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported first quarter 2020 financial results and recent business highlights (Press release, Sangamo Therapeutics, MAY 11, 2020, View Source [SID1234557503]).

"I’m proud of how the Sangamo team has worked together through the unusual circumstances presented by the COVID-19 pandemic," said Sandy Macrae, CEO of Sangamo. "While adhering to governmental workplace guidelines, we have sought to minimize disruptions to the progress of our research in the labs, our clinical trials, and our business development discussions. Importantly, Pfizer continues to target dosing a first patient in the Phase 3 hemophilia A gene therapy study in the second half of 2020, and we are working together to identify an opportunity to present additional data from the Phase 1/2 Alta Study. Our AAV manufacturing facility in Brisbane is expected to be operational by year end, and our cell therapy manufacturing units in Brisbane and in France in 2021. With the $350 million received from the recently closed Biogen collaboration, in addition to the $363 million in cash resources reported as of March 31st, we are moving ahead with significant balance sheet strength, and we are beginning to plan for resuming normal operations as shelter-in-place orders are lifted."

Recent Highlights
•Closed collaboration agreement with Biogen for development of gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurological diseases, receiving $225 million in stock sale proceeds and an additional $125 million upfront license fee.
•Executed a collaboration and exclusive global license agreement with UK cell conversion company Mogrify Ltd for Sangamo to develop allogeneic cell therapies from Mogrify’s proprietary induced pluripotent stem cells (iPSCs) cell conversion technology using Sangamo’s zinc finger protein (ZFP) gene-engineered chimeric antigen receptor regulatory T cell (CAR-Treg) platforms. This collaboration may have the potential to accelerate the development of scalable and accessible CAR-Treg cell therapies for the treatment of inflammatory and autoimmune diseases, diversifying Sangamo’s options and complementing current programs.
•Pfizer continues to target dosing the first patient in the Phase 3 hemophilia A gene therapy study in the second half of 2020. Details of the Phase 3 trial protocol were recently posted to clinicaltrials.gov, and Pfizer continues to recruit patients into the Phase 3 lead-in study. Pfizer and Sangamo are working together to identify an appropriate opportunity this year to provide the next clinical data update of results from the ongoing Phase 1/2 Alta Study.
•Successfully screened and enrolled the first several patients into the Phase 1/2 STAAR study evaluating ST-920 gene therapy for the treatment of Fabry disease and expect to initiate patient dosing at the earliest appropriate and safe opportunity in light of the COVID-19 pandemic.
•Appointed D. Mark McClung as Executive Vice President and Chief Business Officer leading commercial strategic planning, alliance management and corporate and business development.

First Quarter 2020 Financial Results
Cash, cash equivalents and marketable securities were $363.1 million as of March 31, 2020, compared to $384.3 million as of December 31, 2019. Since the end of the first quarter, the Company has received from Biogen $225.0 million for the issuance of Sangamo stock and a $125.0 million upfront license fee.

Consolidated net loss attributable to Sangamo for the first quarter ended March 31, 2020 was $42.9 million, or $0.37 per share, compared to a net loss of $42.2 million, or $0.41 per share, for the same period in 2019. Revenues

for the first quarter ended March 31, 2020 were $13.1 million, compared to $8.1 million for the same period in 2019.
table1.gif

Non-GAAP operating expenses, which excludes stock-based compensation expense, were $52.0 million for the first quarter ended March 31, 2020, compared to $47.4 million for the same period in 2019. The increase in operating expenses reflects the Company’s headcount growth and facilities expansion to support the advancement of Sangamo’s therapeutic pipeline and manufacturing capabilities. These increases were partially offset by a decrease in clinical and manufacturing supply expenses.

Financial Guidance for 2020 reiterated (initially provided on February 28, 2020)
•On a GAAP basis, we continue to expect operating expenses in the range of $270 million to $285 million, including stock-based compensation expense of approximately $25 million.
•We continue to expect non-GAAP operating expenses, which excludes stock-based compensation expense, in the range of $245 million to $260 million.

Conference Call
Sangamo will host a conference call today, May 11, 2020, at 5:00 p.m. Eastern Time, which will be open to the public. The call will also be webcast live and can be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 6043504. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 6043504.

Ultragenyx to Present at BofA Securities Global Health Care Conference

On May 11, 2020 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that Shalini Sharp, the company’s Chief Financial Officer, will hold a virtual presentation at the BofA Securities Global Health Care Conference on Thursday, May 14, 2020 at 10:20 AM ET (Press release, Ultragenyx Pharmaceutical, MAY 11, 2020, View Source [SID1234557491]).

The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

Portola Pharmaceuticals Reports First Quarter 2020 Financial Results and Provides Corporate Update

On May 11, 2020 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported financial results for the three months ended March 31, 2020, and provided a corporate update, including the Company’s actions to continue to support public health efforts and the health and safety of employees, patients and healthcare providers during the COVID-19 pandemic (Press release, Portola Pharmaceuticals, MAY 11, 2020, View Source [SID1234557490]).

"2020 started strong with January representing our highest month of Andexxa demand in the U.S. since launch, driven in part by a return of growth in our tier one accounts," said Scott Garland, Portola’s president and chief executive officer. "While encouraging, the emergence of the COVID-19 pandemic impacted our revenue in March and the quarter due to three main factors. First, market research and customer feedback indicate that shelter-in-place restrictions issued by over 40 states have led to fewer patients coming into emergency departments. Second, most healthcare systems shifted their focus to preparing for and addressing COVID-19 patients, which impacted the number of new accounts added in the quarter. Finally, on March 13, we suspended face-to-face field interactions with healthcare providers. Despite these challenges, we continue to execute on our growth drivers and make progress with virtual meetings and education programs to highlight the value proposition of Andexxa, including recently presented clinical data."

As previously announced on May 5, 2020, Portola entered into a definitive merger agreement to be acquired by Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN). Under the terms of the merger agreement, a subsidiary of Alexion will commence a tender offer to acquire all of the outstanding shares of Portola common stock at a price of $18 per share in cash. The tender offer is subject to customary conditions, including the tender of a majority of the outstanding shares of Portola common stock, the expiration or termination of the waiting period under the Hart-Scott Rodino Antitrust Improvements Act of 1976 and receipt of certain other regulatory approvals. Following successful completion of the tender offer, Alexion will acquire all remaining shares not tendered in the offer at the same price of $18 per share in cash through a merger. The transaction is expected to close in the third quarter of 2020.

Quarter Ending March 31, 2020, and Related Financial Results

•Total global revenues for the first quarter of 2020 were $26.4 million compared with $22.2 million for the same period in 2019. This includes $25.6 million in net product revenues from sales of Andexxa/Ondexxya [coagulation factor Xa (recombinant), inactivated-zhzo], and $0.8 million in collaboration and license revenues.

•Net loss attributable to Portola for the first quarter of 2020 was $68.8 million, or $0.88 net loss per share, compared with $78.2 million, or $1.17 net loss per share, for the same period in 2019.

•Total operating expenses for the first quarter of 2020 were $84.8 million, including $9.5 million in stock-based compensation, compared with $95.8 million for the same period in 2019. Included in total operating expenses was a stock-based compensation expense of $9.5 million for the first quarter of 2020, compared with $17.9 million, including one-time equity valuation for a manufacturer of $5.8 million, for the same period in 2019.

•Research and development (R&D) expenses for the first quarter of 2020 were $26.1 million compared with $35.6 million for the same period in 2019. Included in R&D expenses was a stock-based compensation expense of $2.6 million for the first quarter of 2020, compared with $10.1 million, including one-time equity valuation for a manufacturer of $5.8 million, for the same period in 2019.

•Selling, general and administrative (SG&A) expenses for the first quarter of 2020 were $54.4 million compared with $53.0 million for the same period in 2019. Included in SG&A expenses was a stock-based compensation expense of $6.9 million for the first quarter of 2020, compared with $7.8 million for the same period in 2019.

•Cost of sales (COS) for the first quarter of 2020 was $4.3 million compared with $7.2 million for the same period in 2019.

Cash, Cash Equivalents and Investments

Cash, cash equivalents and investments at March 31, 2020, totaled $394.1 million compared with $466.2 million as of December 31, 2019.

Updated 2020 Annual Financial Guidance

Portola is reducing its 2020 operating expenses by approximately $50 million. For the fiscal year 2020, Portola is updating its guidance for R&D and SG&A expenses. Portola now expects GAAP R&D expenses to be between $90 million and $105 million, including stock-based compensation expenses of approximately $15 million, a decrease of $15 million from the prior guidance range of $105 million and $120 million. Portola now expects GAAP SG&A expenses to be between $200 million and $215 million, including stock-based compensation expenses of approximately $38 million, a decrease of $35 million from the prior guidance range of $235 million and $250 million. This updated guidance reflects near-term cost containment measures to extend the cash runway as Portola navigates the COVID-19 pandemic.

With the uncertainty around the duration of COVID-19 impacts, Portola is suspending prior guidance provided during its fourth quarter 2019 call for approximately 350 new hospital adds in 2020.

COVID-19 Response

As the global pandemic continues to evolve rapidly, Portola’s first priority is the health and safety of employees, patients and healthcare providers. Effective March 13, 2020, the Company suspended face-to-face field activity and instituted a mandatory work-from-home policy for all employees, including those in the Company’s South San Francisco and European headquarters. The Company shifted to a virtual field force to continue engaging customers with digital tools and remote meetings where possible. Portola’s plan to present and publish data throughout the year remains intact.

At this time, the global Andexxa supply chain and distribution structure is intact for customers to continue to use and re-order Andexxa. Portola has adequate supply of this important medicine on hand for the next couple of years in the United States and Europe.

Recent Achievements and Events

·Added 68 new accounts in the first quarter of 2020. Andexxa is now stocked in over 700 U.S. hospitals.

·Establishment of a permanent J-code for Andexxa by the Centers for Medicare & Medicaid Services, effective July 1, 2020, ensuring greater patient access by providing hospitals with a clearer reimbursement pathway when administering Andexxa in the outpatient hospital setting.

·Presented the budget impact model demonstrating that using Andexxa may provide a net cost reduction for the treatment of intracranial hemorrhage (ICH) associated with oral Factor Xa inhibitors. The analysis, presented at the Emergencies on Medicine conference, projects that hospital use of Andexxa with NTAP reimbursement can reduce cost per hospitalization by up to $5,400 compared to 4F-PCC’s.

·Presented new data at ACC demonstrating that Andexxa was associated with a lower rate of in-hospital and 30-day mortality in patients with multiple types of life-threatening Factor Xa inhibitor-related bleeds compared with other treatment options. Propensity matched data from the ANNEXA-4 and ORANGE studies showed 30-day mortality was 15% with Andexxa versus 34% with 4F-PCC across all bleed types. Additionally, in a real-world database analysis, in-hospital mortality was 4% with Andexxa and 10% with 4F-PCC across all bleed types.

·Agreed to terminate the collaboration and license agreement with Bristol-Myers Squibb Company and Pfizer, Inc. regarding the development and commercialization of andexanet alfa in Japan. Portola will regain full Japanese rights for andexanet alfa. Japan represents the third largest market for Factor Xa inhibitors after the United States and the EU 5 countries. Portola will have exclusive rights to develop and commercialize andexanet alfa in the United States, Europe, Japan and rest of the world markets.

Planned Upcoming Milestones

·Present and publish comparative clinical, mechanistic and economic data supporting the adoption of Andexxa at medical meetings and in peer-reviewed journals throughout the year including:

oThe ANNEXA-4 study ICH subgroup

oRetrospective comparisons of Andexxa vs. 4F-PCC’s on clinical outcomes using data from the ANNEXA-4, ORANGE and RETRACE studies

·Advance reimbursement discussions in the United Kingdom (2H 2020), Germany (2H 2020) and other Wave 1 European countries.

·Submit a supplemental Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by year end for inclusion of the additional Factor Xa inhibitors edoxaban and enoxaparin in the Andexxa label.