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Puma Biotechnology Reports First Quarter 2018 Financial Results

On May 9, 2018 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported financial results for the first quarter ended March 31, 2018 (Press release, Puma Biotechnology, MAY 9, 2018, View Source [SID1234526389]).

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Unless otherwise stated, all comparisons are for the first quarter 2018 compared to the first quarter 2017.

On July 17, 2017, Puma Biotechnology received approval from the U.S. Food and Drug Administration (FDA) for NERLYNX (neratinib) for the treatment of early stage HER2-positive breast cancer following adjuvant trastuzumab-based therapy, and the Company began shipment to wholesalers at the end of July 2017. Prior to the launch of NERLYNX the Company had no product revenue. Net product revenue from sales of NERLYNX in the first quarter of 2018 amounted to $36.0 million, compared to net product revenue of $6.1 million and $20.1 million in the third and fourth quarters of 2017, respectively.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss applicable to common stock of $24.3 million, or $0.65 per share, for the first quarter of 2018, compared to a net loss applicable to common stock of $72.9 million, or $1.97 per share, for the first quarter of 2017.

Non-GAAP adjusted net income was $1.1 million, or $0.03 per basic share and $0.02 per diluted share, for the first quarter of 2018, compared to non-GAAP adjusted net loss of $43.1 million, or $1.16 per basic and diluted share, for the first quarter of 2017. Non-GAAP adjusted net income (loss) excludes stock-based compensation expense, which represents a significant portion of overall expense and has no impact on the cash position of the Company. For a reconciliation of GAAP net loss to non-GAAP adjusted net income (loss) and GAAP net loss per share to non-GAAP adjusted net income (loss) per share, please see the financial tables at the end of this news release.

Net cash used in operating activities for the first quarter of 2018 was $6.3 million. At March 31, 2018, Puma had cash and cash equivalents of $78.6 million, compared to cash and cash equivalents of $81.7 million at December 31, 2017.

"We made substantial progress in the commercialization of our lead product, NERLYNX (neratinib), during the first quarter of 2018," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "We quickly built momentum in the U.S. market, with net sales steadily rising since our launch. Our exclusive licensing agreements to date, with Pint Pharma in Latin America, CANbridge in mainland China and Taiwan, Medison Pharma in Israel, and Specialised Therapeutics Asia in South East Asia, demonstrate our commitment to also make NERLYNX accessible to patients globally while we continue to grow the U.S. market.

"We are also pleased with the updated National Comprehensive Cancer Network (NCCN) guidelines, which designate NERLYNX as a recommended combination treatment option for breast cancer patients with brain metastases. In addition, data on neratinib were published in the journal Nature, which included initial results from Puma’s ongoing SUMMIT Phase II ‘basket’ clinical trial in patients with tumors harboring HER2 or HER3 mutations. SUMMIT is designed to evaluate the contributions of both genetic mutation and cancer type on individual patient response to neratinib. Information generated from the trial will help guide neratinib-based targeted therapy across a broad spectrum of tumor types with HER2 or HER3 mutations, including patients with rare tumors who may not otherwise have access to investigational therapies. We believe the publication of the initial SUMMIT data in this prestigious journal reflects the novelty and quality of this precision-medicine trial design, as well as the growing understanding that both tumor type and gene mutations play an important role in individual patients’ response to cancer therapies such as neratinib."

Mr. Auerbach added, "During 2018, we anticipate the following key milestones: (i) reporting updated Phase I/II data from neratinib plus Kadcyla (T-DM1) in the HER2-positive metastatic breast cancer trial in the second quarter of 2018; (ii) re-assessment of the Marketing Authorisation Application for neratinib by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in mid-2018; (iii) reporting data from the Phase III trial in third-line metastatic breast cancer patients in the second half of 2018; (iv) submitting for regulatory approval for the extended adjuvant HER2-positive early stage breast cancer indication in select countries in the second half of 2018; and (v) reporting additional data from the Phase II CONTROL trial in the fourth quarter of 2018."

Revenue

Total revenue consists of net product revenue from sales of NERLYNX, Puma’s first and only commercial product to date, and license revenue. The FDA approved NERLYNX for commercial sale in the United States in July 2017 and the Company commenced shipment to wholesalers in late July. For the first quarter of 2018, total revenue was $66.5 million, of which $36.0 million was net product revenue and $30.5 million was license revenue received from Puma’s sub-licensees.

Operating Expenses

Operating expenses were $89.9 million for the first quarter of 2018, compared to $73.2 million for the first quarter of 2017.

Cost of Sales:

Cost of sales was $6.4 million for the first quarter of 2018. The Company had no product sales prior to the third quarter of 2017.

Selling, General and Administrative Expenses:

Selling, general and administrative expenses were $36.6 million for the first quarter of 2018, compared to $18.4 million for the first quarter of 2017. The $18.2 million increase resulted primarily from increases of approximately $7.8 million in payroll and related costs, $6.6 million in marketing, market access, and legal expenses, $1.7 million in travel and related costs, and $1.7 million in stock-based compensation. These increases reflect the commercial launch of NERLYNX and overall corporate growth.

Research and Development Expenses:

Research and development (R&D) expenses were $46.9 million for the first quarter of 2018, compared to $54.8 million for the first quarter of 2017. The $7.9 million decrease resulted primarily from decreases of approximately $6.1 million for stock-based compensation and $4.0 million for clinical trial expenses, partially offset by an increase of $2.2 million for payroll and related costs in medical affairs and commercial quality assurance. For our existing clinical trials, we expect R&D expenses to decrease in subsequent quarters as clinical trials continue to wind down.

Conference Call

Puma Biotechnology will host a conference call to report its first quarter 2018 financial results and provide an update on the company’s business and outlook at 1:30 p.m. PDT/4:30 p.m. EDT on Wednesday, May 9, 2018. The call may be accessed by dialing 1-877-709-8150 (domestic) or 1-201-689-8354 (international) at least 10 minutes prior to the start of the call and referencing the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available approximately one hour after completion of the call and will be archived on the company’s website for 90 days.

8-K – Current report

On May 9, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported financial results for the first quarter ending March 31, 2018 (Press release, PTC Therapeutics, MAY 9, 2018, View Source [SID1234526388]).

"Over the past twenty years it has been our mission to bring clinically differentiated therapies to patients with rare disorders," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "As we look forward, we are executing on that mission and consolidating our position as a leading rare disorder biotech company. We intend to continue to leverage our deep scientific expertise and world class commercial capabilities."

First Quarter 2018 Financial Highlights:

Translarna net product sales were $36.8 million for the first quarter of 2018, representing 39% growth over $26.4 million reported in the first quarter of 2017.

Emflaza net product sales were $19.2 million for the first quarter of 2018.

Total revenues for the first quarter of 2018 were $56.1 million compared to $26.5 million in the same period in 2017. The change in total revenue was a result of Emflaza sales and the expanded commercial growth of Translarna.

GAAP R&D expenses were $31.4 million for the first quarter of 2018 compared to $27.4 million for the same period in 2017. Non-GAAP R&D expenses were $27.6 million for the first quarter of 2018, excluding $3.7 million in non-cash, stock-based compensation expense, compared to $22.9 million for the same period in 2017, excluding $4.5 million in non-cash, stock-based compensation expense. The increase in R&D expenses for the first quarter of 2018 as compared to the prior year period was primarily due to increased investment in research programs and advancement of the clinical pipeline.

GAAP SG&A expenses were $33.0 million for the first quarter of 2018 compared to $25.5 million for the same period in 2017. Non-GAAP SG&A expenses were $29.0 million for the first quarter of 2018, excluding $4.0 million in non-cash, stock-based compensation expense, compared to $20.9 million for the same period in 2017, excluding $4.6 million in non-cash, stock-based compensation expense. The increase in SG&A expenses for the first quarter of 2018 as compared to the prior year period was primarily due to the continued commercial support for the Emflaza launch and continued growth of Translarna marketing activities.

Net interest expense for the first quarter of 2018 was $3.3 million compared to $2.2 million in the same period in 2017. The increase in net interest expense for the first

quarter of 2018 as compared to the prior year period was primarily due to increased interest expense related to the $40 million secured loan facility which we closed during the second quarter of 2017 partially offset by interest income from investments.

Net loss for the first quarter of 2018 was $19.3 million compared to a net loss of $29.1 million for the same period in 2017.

Cash, cash equivalents, and marketable securities totaled approximately $178.3 million at March 31, 2018 compared to approximately $191.2 million at December 31, 2017.

Shares issued and outstanding as of March 31, 2018 were 41.8 million.

In April 2018, PTC completed a public offering of 4,600,000 shares of common stock, resulting in net offering proceeds of $117.9 million.
2018 Guidance

Full year 2018 net product revenues to be between $260 and $295 million. PTC anticipates Translarna net product revenue for the full year 2018 to be between $170 and $185 million. PTC projects a 5-year (December 31, 2022) compound annual growth rate of 15% for net product revenues representing continued strong growth year-over-year by increasing penetration in current countries and pursuing opportunities for label expansion. PTC anticipates Emflaza net product revenue for the full year 2018 to be between $90 and $110 million.

GAAP R&D and SG&A expense for the full year 2018 to be between $280 and $290 million.

Non-GAAP R&D and SG&A expense for the full year 2018 to be between $250 and $260 million, excluding estimated non-cash, stock-based compensation expense of approximately $30 million.

Key 2018 Corporate Highlights:

Analyst Day highlighting PTC’s 20-year commitment to developing therapeutics for rare disorders. PTC’s management and research teams provided an in-depth update on the Company’s commercial products, Translarna and Emflaza, its scientific platforms including alternative splicing with Spinal muscular atrophy in pivotal trials and two indications, Huntington’s disease and Familial dysautonomia which the company anticipates will enter the clinic in 2020. In addition to these indications, PTC also provided updates on our niche oncology pipeline. There are currently two advanced candidates, PTC596 which is currently in the clinic, and PTC299, which PTC anticipates will re-enter the clinic later this year. In 2018, PTC is expecting to initiate clinical trials in two solid tumor indications for PTC596. PTC299, a DHODH inhibitor, is planned to enter clinical trials in hematological tumors in the third quarter of this year. PTC also has a second DHODH inhibitor fast follower currently in late-stage chemical optimization. A replay of the presentations and panel discussions can be found on the Investors page of the website.

Encouraging early data from open-label FIREFISH study in Type 1 SMA babies. Data recently presented at the American Academy of Neurology 2018 Annual Meeting from Part 1 of the FIREFISH study, the dose finding portion, showed that there have been no drug-related safety findings leading to withdrawal at any dose level of the

investigational molecule RG7916. In addition, no babies have required a tracheostomy or permanent ventilation since study initiation and no baby has lost the ability to swallow. The median age of first dose was 6.7 months and the 21 babies in the study received RG7916 for a duration of up to 14.8 months. It was reported in January that two babies had died from causes related to disease progression and the deaths were determined not to be drug related. The primary endpoint of Part 2, the confirmatory part of the FIREFISH study, is the proportion of patients sitting without support after 12 months on RG7916 treatment. Recruitment is ongoing globally for FIREFISH Part 2. The SMA program is a collaboration between PTC, Roche, and the SMA Foundation.

Review of the Translarna Pediatric Label Expansion by the Committee for Medicinal Products for Human Use (CHMP) in progress. The application to expand the label of Translarna for the treatment of nonsense mutation DMD patients who are 2-5 years is currently under review by the CHMP in Europe and a decision is expected mid-year.

Dystrophin study for US NDA for ataluren (Translarna) in DMD to begin by the end of 2018. The Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway. This would involve a re-submission of an NDA containing the current data on effectiveness of ataluren with new data to be generated on dystrophin production. PTC is working to design such a study and expects to initiate such a study by the end of 2018.

Successful closing of public offering. PTC offered 4,600,000 shares of common stock, successfully raising $117.9 million in the public market. PTC intends to use the net proceeds from this offering to fund its research and development efforts, including clinical trials and studies with respect to its products and product candidates and potential additional indications, including its programs for alternative splicing for the treatment of rare disorders and oncology, commercialization activities for Translarna for the treatment of nmDMD outside of the United States and Emflaza for the treatment of DMD in the United States and for working capital and other general corporate purposes.

Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude stock-based compensation expense. This non-GAAP financial measure is provided as a complement to financial measures reported in GAAP because management uses this non-GAAP financial measure when assessing and identifying operational trends. In management’s opinion, this non-GAAP financial measure is useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Quantitative reconciliations of non-GAAP financial measures to their closest equivalent GAAP financial measures are included in the tables below.

Today’s Conference Call and Webcast Reminder:

Today’s conference call will take place at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 3757748. A live, listen-only webcast of the conference call can be accessed on the Investor Relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for two weeks.

Protagonist Therapeutics Reports First Quarter 2018 Financial Results

On May 9, 2018 Protagonist Therapeutics, Inc. (Nasdaq: PTGX) reported its financial results for the first quarter ended March 31, 2018 (Press release, Protagonist, MAY 9, 2018, View Source;p=RssLanding&cat=news&id=2348218 [SID1234526387]).

"Protagonist is continuing to build a broad and diverse pipeline of potentially transformative drug candidates discovered through its innovative peptide technology platform," said Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "PTG-200, the IL-23 receptor antagonist being developed in collaboration with Janssen Biotech, Inc., is currently in a Phase 1 study, and we expect a U.S. IND filing by the end of 2018 to support a Phase 2 global study in Crohn’s disease. With our hepcidin mimetic PTG-300, we plan to commence a Phase 2 study in beta-thalassemia patients in the fourth quarter of 2018."

"In addition to developing PTG-200 and PTG-300, we are conducting a comprehensive review of the PTG-100 dataset from the Phase 2b PROPEL study and expect to report our findings in the third quarter of 2018. As previously announced, we discontinued the trial based on a planned interim analysis by an independent Data Monitoring Committee and noted that it also showed an unexpectedly high placebo response rate," continued Dr. Patel.

Product Development Update:

PTG-100

A planned interim analysis was conducted during the first quarter for the Phase 2b PROPEL study of PTG-100, the company’s investigational oral GI-restricted alpha-4-beta-7 integrin antagonist peptide, in patients with moderate to severe ulcerative colitis. The Data Monitoring Committee reported that the interim analysis met prespecified futility criteria on the primary endpoint of clinical remission, and as a result the study was discontinued. It was also noted that the study experienced an unusually high placebo response rate.
Protagonist is now conducting an extensive review of the complete dataset from all patients enrolled in the Phase 2b trial. The Company will determine next steps for PTG-100 and for the general therapeutic approach of oral, GI-restricted alpha-4-beta-7-integrin antagonists after completing this review which is expected to be in the third quarter of 2018.
PTG-200

Dosing has been completed for all cohorts in the Phase 1 study of PTG-200, a first-in-class oral, GI restricted, IL-23 receptor antagonist peptide, which is partnered with Janssen Biotech, Inc. (Janssen). The Phase 1 study in normal healthy volunteers was initiated in Australia in November 2017 and involves single-ascending doses and multiple ascending doses of PTG-200.
In the second half of 2018 we expect a U.S. IND filing and other regulatory filings for a global Phase 2 study of PTG-200 in Crohn’s disease to be conducted in collaboration with Janssen.
PTG-300

PTG-300, an injectable hepcidin mimetic, is in development for the potential treatment of anemia and iron overload related to rare blood disorders. We have completed discussions with U.S. and global regulatory agencies, and the Company plans to file a U.S. IND and other regulatory filings in the third quarter of 2018 with the intent to commence a global Phase 2 trial in Q4 2018 in patients with beta-thalassemia. Furthermore, treatment of anemia and transfusion-dependence in myelodysplastic syndromes and exaggerated erythropoiesis in polycythemia vera represent additional opportunities for further development of PTG-300.
The U.S. Food and Drug Administration granted Orphan Drug Designation to PTG-300 for the treatment of beta-thalassemia in March of 2018.
Preclinical programs

The Company’s oral peptide agonist targeting the mu/delta opioid receptors will be discussed in a podium presentation at the Digestive Diseases Week conference, on Saturday, June 2, from 5:00 PM to 5:15 PM ET at the Walter E. Washington Convention Center, Room 140 in Washington, D.C.
The company will present preclinical data supporting the superiority of mu/delta dual agonist activities when compared to eluxadoline in treating diarrhea or abdominal pain.
Financial Results

Protagonist reported a net loss of $7.7 million for the first quarter of 2018, as compared to a net loss of $14.1 million for the same period of 2017. The decrease in net loss was driven primarily by license and collaboration revenue recognized during the first quarter of 2018, which partially offset increased research and development (R&D) expenses and increased general and administrative (G&A) expenses. The net loss for the first quarter of 2018 includes non-cash stock-based compensation of $1.2 million, as compared to $0.8 million for the same period of 2017.

License and collaboration revenue was $10.8 million for the first quarter of 2018 and consisted of revenue from activities performed under the Janssen Collaboration Agreement. Protagonist did not recognize any license and collaboration revenue for the first quarter of 2017.

R&D expenses for the first quarter of 2018 were $15.4 million, as compared to $11.3 million for the same period of the prior year. The increase in R&D expenses was primarily due to costs related to contract manufacturing, and the preparation for and conduct of PTG-100, PTG-200 and PTG-300 clinical trials. R&D expenses for the quarter also included an increase in salaries and employee-related expenses due to an increase in R&D personnel.

G&A expenses for the first quarter of 2018 were $3.6 million, as compared to $3.0 million for the same period in the prior year. The increase in G&A expense was due primarily to increases in salaries and employee-related expenses primarily due to an increase in headcount to support the growth of our operations, franchise and business related taxes and other administrative expenses.

Protagonist ended the first quarter of 2018 with $140.5 million in cash, cash equivalents and investments. The company expects current capital resources to be sufficient to fund its operations through 2019.

Protagen AG collaborates with UCSF to study better immuno-profiling of cancer patients receiving immunotherapy

On May 9, 2018 Protagen AG has reported the start of a collaboration with the University of California, San Francisco (UCSF) to utilize Protagen’s SeroTag technology to investigate the immuno-profiling of prostate cancer patients treated with checkpoint inhibitors and therapeutic vaccines (Press release, Protagen, MAY 9, 2018, View Source [SID1234526386]).

Cancer immunotherapies can be very powerful and provide novel opportunities for the treatment of cancer. However, they currently work for a limited number of indications and patients. In addition, as these therapies re-activate the immune system to fight the cancer, they sometimes can cause severe immune-related Adverse Events (irAEs). Through the collaboration, Protagen and UCSF intend to provide further insight into utilizing immune system profiling to predict treatment response and monitor prostate cancer patients for irAEs, specifically, a so-called cold tumor that it is difficult to target with immuno-therapies.

Dr. Peter Schulz-Knappe, Protagen’s Chief Scientific Officer, commented: "Our proprietary SeroTag technology has enabled patient stratification and immuno-profiling of patients into homogenous disease subgroups for several autoimmune indications. The strong link between immuno-oncology and autoimmune disease, confirmed by the observed irAEs under immunotherapy, provides us with an opportunity to improve immuno-profiling of cancer patients. We feel honored that Dr. Fong and UCSF share this view and we are excited about our collaboration, especially in an indication like prostate cancer that has shown to be difficult to target with immuno-therapies."

Dr. Lawrence Fong, the UCSF Efim Guzik Distinguished Professor in Cancer Biology and leader of the Cancer Immunotherapy Program in the UCSF Helen Diller Family Comprehensive Cancer Center added: "Although cancer immunotherapies can be effective in many different cancers, success in prostate cancer has been more limited. Nevertheless, we know that a small proportion of prostate cancer patients can respond to monotherapies. Immunologic profiling of these patients could enable approaches to patient selection. This collaboration could provide opportunities to accomplish this goal."

Disclaimer

The information stated above was prepared by Protagen AG and reflects solely the opinion of Protagen AG. Nothing in this statement shall be construed to imply any support or endorsement of Protagen AG, or any of its products, by the Regents of the University of California, its officers, agents and employees.

About UC San Francisco (UCSF)

UCSF is a leading university dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. It includes top ranked graduate schools of dentistry, medicine, nursing and pharmacy; a graduate division with nationally renowned programs in basic, biomedical, transitional and population sciences; and a preeminent biomedical research enterprise.

It also includes UCSF Health, which comprises three top-ranked hospitals, UCSF Medical Center and UCSF Benioff Children’s Hospitals in San Francisco and Oakland, and other partner and affiliated hospitals and healthcare providers throughout the Bay Area.

Please visit www.ucsf.edu/news.

Portola Pharmaceuticals Reports First Quarter 2018 Financial Results and Provides Corporate Update

On May 9, 2018 Portola Pharmaceuticals, Inc. (Nasdaq:PTLA) reported financial results for the three months ended March 31, 2018 and provided a corporate update (Press release, Portola Pharmaceuticals, MAY 9, 2018, View Source;p=RssLanding&cat=news&id=2348250 [SID1234526385]).

"We achieved several major manufacturing and regulatory milestones this past year, including last week’s U.S. approval of Andexxa. We are now concentrating the Company’s efforts on the successful launch of Andexxa and Bevyxxa, which both have the potential to impact public health and become standards of care in the field of thrombosis," said Bill Lis, chief executive officer of Portola. "We are also encouraged by interim data for our next compound for hematologic cancers, cerdulatinib, which will be presented next month at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Together, these three compounds comprise a leading thrombosis and hematology portfolio, all with global rights."

Recent Achievements, Upcoming Events and Milestones

Andexxa [coagulation factor Xa (recombinant), inactivated-zhzo] – antidote for the reversal of the Factor Xa inhibitors rivaroxaban and apixaban.

Andexxa received Accelerated Approval from the FDA on May 3, 2018 for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.
Earned an additional $100 million milestone payment from the Company’s royalty-based financing with Health Care Royalty Partners based on the FDA approval of Andexxa in May 2018.
Early Supply Program to launch in June with broader commercial launch anticipated in early 2019, upon FDA approval of the Generation 2 product.
Presented interim data from the ongoing Phase 3b/4 ANNEXA-4 study in a late-breaking clinical trial presentation at the American College of Cardiology’s 67th Annual Scientific Session & Expo (ACC.18). Enrollment on track for completion this summer.
Received a positive CHMP trend vote and working with regulatory authorities to address their accompanying request for additional data.
Built significant Generation 2 product inventory to meet broad demand upon regulatory approval in the U.S. and Europe.
Bevyxxa (betrixaban) – oral, once-daily Factor Xa inhibitor approved for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for VTE.

Initiated U.S. commercial launch and continued to expand the field force and market access teams.
Additional results from the APEX trial published in the American Heart Journal and the American Journal of Medicine continue to highlight betrixaban’s effect on symptomatic VTE and VTE-related deaths.
Eight abstracts accepted at the upcoming International Society on Thrombosis and Haemostasis (ISTH) and European Society of Cardiology (ESC) meetings.
Cerdulatinib – an oral, dual-spleen tyrosine kinase (SYK) and janus kinase (JAK) inhibitor in development for the treatment of relapsed/refractory B-cell and other T-cell malignancies in patients who have failed multiple therapies.

Completed enrollment in two of four cohorts of the ongoing Phase 2a study evaluating the safety and efficacy of cerdulatinib in patients with relapsed/refractory B-cell and T-cell malignancies who have failed multiple therapies.
Interim results from the ongoing Phase 2a study accepted for presentation at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Received initial feedback from the FDA on the potential regulatory pathway.
First Quarter 2018 Financial Results
Total revenue for the first quarter of 2018 was $6.6 million, compared with $5.1 million for the first quarter of 2017. This includes $6.0 million in collaboration and license revenue earned under Portola’s collaboration and license agreements with Bristol-Myers Squibb Company, Pfizer, Bayer Pharma, Janssen Pharmaceuticals and Daiichi Sankyo, as well as $0.6 million in product revenue from initial sales of Bevyxxa, which was launched in the U.S. in January 2018.

Total operating expenses for the first quarter of 2018 were $91.9 million, compared with $45.7 million for the same period in 2017. Total operating expenses for the first quarter of 2018 included $11.0 million in stock-based compensation expense, compared with $9.0 million for the same period in 2017.

Research and development expenses were $60.1 million for the first quarter of 2018, compared with $30.6 million for the first quarter of 2017. The increase is due to the second Generation 2 commercial manufacturing campaign. Selling, general and administrative expenses for the first quarter of 2018 were $31.5 million, compared with $15.0 million for the same period in 2017. The increase is due to the build-out of the field force and marketing spend for the Bevyxxa launch.

For the first quarter of 2018, Portola reported a net loss of $84.2 million, or $1.28 net loss per share, compared with a net loss of $41.7 million, or $0.74 net loss per share, for the same period in 2017. Shares used to compute net loss per share attributable to common stockholders were 65.5 million for the first quarter of 2018 compared with 56.7 million for the same period in 2017.

Cash, cash equivalents and investments at March 31, 2018 totaled $451.1 million, compared with cash, cash equivalents and investments of $534.2 million as of December 31, 2017.

Based on the FDA approval of Andexxa in May 2018, the Company earned an additional $100 million milestone payment from its royalty-based financing with Health Care Royalty Partners.

Conference Call Details
Portola will host a conference call today, Wednesday, May 9, 2018, at 4:30 p.m. ET, during which time management will provide first quarter 2018 financial results, updates on Andexxa, the U.S. launch of Bevyxxa and other matters. The live call can be accessed by phone by dialing (844) 452-6828 from the U.S. and Canada or 1 (765) 507-2588 internationally and using the passcode 7068059. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.