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

On August 9, 2018 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported financial results for the three months ended June 30, 2018 and provided a corporate update (Press release, Portola Pharmaceuticals, AUG 9, 2018, View Source;p=RssLanding&cat=news&id=2363072 [SID1234528581]).

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"The second quarter of 2018 brought a significant addition to our product suite with the U.S. Food and Drug Administration’s Accelerated Approval of Andexxa and the initiation of our Early Supply Program. Together with Bevyxxa, we now have two FDA-approved, first-and-only medicines for their indications in the field of thrombosis with the potential to impact public health," said Mardi Dier, interim co-president and chief financial officer of Portola. "In the second half of the year, we remain focused on continuing to lay the foundation for the U.S. launch of Bevyxxa and preparing for a number of significant milestones, including our regulatory submission in the U.S. for the Generation 2 Andexxa product, review and potential approval for andexanet alfa in Europe, and determining the development and regulatory path forward for cerdulatinib, our Syk/JAK inhibitor and the third novel compound discovered in our labs."

Second Quarter 2018 Financial Results
Total revenue for the second quarter of 2018 was $4.0 million, compared with $3.8 million for the second quarter of 2017. This includes $1.7 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 $2.2 million from initial sales of Andexxa in the U.S. under the Company’s Early Supply Program launched in May 2018, and $33,000 in product revenue from sales of Bevyxxa, which launched in the U.S. in January 2018.

Total operating expenses for the second quarter of 2018 were $107.7 million, compared with $69.6 million for the same period in 2017. Total operating expenses for the second quarter of 2018 included $13.2 million in stock-based compensation expense, compared with $13.3 million for the same period in 2017.

Research and development expenses were $66.4 million for the second quarter of 2018, compared with $49.3 million for the second quarter of 2017. The increase is due to the second Generation 2 Andexxa commercial manufacturing campaign. Selling, general and administrative expenses for the second quarter of 2018 were $40.2 million, compared with $20.3 million for the same period in 2017. The increase is due to the build-out of the field force and marketing spend for the Andexxa Early Supply Program and the Bevyxxa launch.

For the second quarter of 2018, Portola reported a net loss of $106.2 million, or $1.61 net loss per share, compared with a net loss of $69.7 million, or $1.22 net loss per share, for the same period in 2017. Shares used to compute net loss per share attributable to common stockholders were 65.9 million for the second quarter of 2018 compared with 57.1 million for the same period in 2017.

Cash, cash equivalents and investments at June 30, 2018 totaled $456.7 million, compared with $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 arrangement with Health Care Royalty Partners.

Recent Achievements and Events

Received Accelerated Approval from the FDA for Andexxa and initiated commercial launch under the Early Supply Program.
Completed the second successful manufacturing campaign of Generation 2 Andexxa product.
Continued progress with the Bevyxxa U.S. commercial launch, including formulary wins, reimbursement coverage and physician education.
New interim results from the ongoing Phase 2a study of cerdulatinib presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).
Four abstracts presented at the International Society on Thrombosis and Haemostasis (ISTH) meeting.
Received New Technology Add-on Payment for Andexxa from the Centers for Medicare and Medicaid Services.
Upcoming Milestones

Eight abstracts to be presented at the European Society of Cardiology (ESC) meeting.
Submit Prior Approval Supplement (PAS) for Generation 2 Andexxa product by the end of August, positioning the Company for a broader commercial launch in early 2019, upon FDA approval.
On track to deliver additional data to European regulatory authorities in the fourth quarter, with potential for European approval of andexanet alfa in the first half of 2019.
Ongoing discussion with the FDA on the potential regulatory pathway for cerdulatinib.
Conference Call Details
Portola will host a conference call today, Thursday, August 9, 2018, at 8:30 a.m. ET, during which time management will discuss the second quarter 2018 financial results, updates on the U.S. launches of Andexxa and 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 6650817. 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.

Newly Published Independent Study on a Prospective Cohort Highlights DecisionDx-Melanoma Prognostic Test’s Ability to Accurately Identify High-Risk Melanoma Patients

On August 9, 2018 Castle Biosciences, Inc., the skin cancer diagnostics company providing molecular diagnostics to improve cancer management decisions, reported the publication of an independent study from a dermatology practice, in a prospectively collected cohort, showing that the DecisionDx-Melanoma prognostic test can accurately identify patients at high and low risk of metastasis and death from melanoma (Press release, Castle Biosciences, AUG 9, 2018, View Source [SID1234528575]). Patients identified as high risk were found to be 22 times more likely to develop metastasis than those with a low-risk test result. Overall this 256-patient study is consistent with previous prospective and retrospective studies and confirms the prognostic value of the DecisionDx-Melanoma test. The study was authored by Dr. Bradley Greenhaw and colleagues and published in Dermatologic Surgery.

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Study Findings:

High negative predictive value of 98.6% provides a high degree of confidence in a low risk of metastasis for Class 1 patients.
Class 2 patients were 22 times more likely to develop metastasis than patients with a Class 1 result.
The 5-year melanoma-specific survival (MSS) rate was also significantly different between groups with 99% survival for Class 1 patients and 79% survival for Class 2 patients (p<0.0001).
The authors indicate that they now offer the DecisionDx-Melanoma test to all patients with invasive cutaneous melanoma and provide more intensive follow-up for Class 2 patients to support timely detection of metastasis and earlier intervention with therapy.
"These robust findings from an independent study demonstrating that the DecisionDx-Melanoma test can identify early-stage melanoma patients who are at high risk for metastasis and death are consistent with previous retrospective and prospective studies," said lead author Bradley Greenhaw, M.D., Dermatology Center of North Mississippi, Tupelo, Mississippi. "In this dermatology practice, as in many others across the country, the DecisionDx-Melanoma test has been incorporated into practice protocols to improve patient management decisions based on recurrence risk beyond clinicopathologic staging alone."

Study Details:

The study was conducted at a single dermatology center and included 256 patients who were identified using the center’s prospectively collected melanoma registry. Patients were diagnosed with cutaneous melanoma during the previous 5 years and were prospectively tested with DecisionDx-Melanoma as part of their initial diagnostic work-up or tested as part of their follow-up care. Retrospective chart review was used in cases of missing data in the registry.
86% of patients had American Joint Committee on Cancer (AJCC) Stage I melanoma, and 14% had Stage II disease.
214 patients had a Class 1 DecisionDx-Melanoma test result; 42 patients were Class 2. Mean follow-up time was 23 months.
The paper can be accessed at the Dermatologic Surgery website.

About DecisionDx-Melanoma

The DecisionDx-Melanoma test uses tumor biology to predict individual risk of melanoma recurrence and sentinel lymph node positivity independent of traditional factors. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in three multi-center studies that have included 690 patients and have demonstrated consistent results. Performance has also been confirmed in four prospective studies including 702 patients. The consistent high performance and accuracy demonstrated in these studies, which combined have included over 1,300 patients, provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results.

Prediction of the likelihood of sentinel lymph node positivity has also been validated in two prospective multicenter studies that included over 1,400 patients. Impact on patient management plans for one of every two patients tested has been demonstrated in multi-center and single-center studies. More information about the test and disease can be found at www.SkinMelanoma.com.

Vaxart Announces Second Quarter 2018 Financial Results and Corporate Update

On August 9, 2018 Vaxart, Inc., a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, reported financial results for the second quarter ended June 30, 2018 and provided a corporate update (Press release, Aviragen Therapeutics, AUG 9, 2018, View Source [SID1234528574]).

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"We are encouraged by the recent publications in Vaccine and the Journal of Clinical Investigation Insight recognizing the unique ability of our oral recombinant vaccines to elicit both systemic and mucosal immune responses, further validating the value of our proprietary platform," said Wouter Latour, M.D., chief executive officer of Vaxart. "Our team is fully focused on advancing our vaccine programs, and we are on track to initiate our Phase 1 norovirus bivalent study and Phase 2 norovirus challenge study later this year."

Second Quarter 2018 and Recent Highlights:

Corporate:

· On May 31, 2018, the Company presented additional data on the previously disclosed Phase 1 norovirus vaccine trial in a poster presentation at the American Society of Microbiology 2018. As described in the poster, the Vaxart GI.1 norovirus tablet vaccine generated IgG and IgA antibodies in serum that were highly cross-reactive against other GI genotypes, specifically GI.3 and GI.4.

· On June 4, 2018, Vaxart reported the topline results from a Phase 2 clinical trial evaluating teslexivir, a small-molecule antiviral for the treatment of condyloma that Vaxart obtained in the acquisition of Aviragen earlier in 2018, in which the primary efficacy endpoint was not achieved.

· On June 27, 2018, the Company announced the publication of preclinical results from its oral F-protein based Respiratory Syncytial Virus (RSV-F) vaccine in Vaccine. As described in the article, the oral RSV-F vaccine candidate provided complete sterilizing protection against RSV infection in the cotton rat challenge model at the target dose.

· On July 12, 2018, Vaxart announced the publication of the comprehensive results of the previously disclosed Phase 1 clinical trial with its norovirus oral tablet vaccine in the Journal of Clinical Investigation Insight. As reported in the article, the vaccine generated robust systemic and mucosal immune responses, including mucosal IgA, memory B cells, and serum blocking antibody titers (BT50), all potential correlates of protection.

Second Quarter 2018 Financial Results

· Vaxart ended the quarter with cash and cash equivalents of $23.9 million compared to $17.5 million at March 31, 2018. The increase was primarily due to $12.0 million received from royalty payments offset by cash used in operations.

· Revenue for the quarter was $0.6 million compared to $1.9 million in the second quarter of 2017. The decrease was due to lower revenues from the contract with HHS BARDA, where activities are winding down.

· Research and development expenses were $5.0 million for the quarter compared to $4.3 million for the second quarter of 2017. The increase was due to additional personnel, amortization of intangibles and clinical trial costs resulting from the merger with Aviragen, offset by lower expenditures on the HHS BARDA activities and Vaxart’s norovirus Phase 1 clinical trial.

· General and administrative expenses were $1.8 million for the quarter compared to $0.7 million for the second quarter of 2017. The increase was due to the additional costs of being a public company, including the costs of additional personnel and higher legal and other services.

· During the quarter, Vaxart recognized an impairment charge of $1.6 million resulting from the write-off of the teslexivir intangible asset acquired in the merger with Aviragen.

Loxo Oncology Reports Second Quarter 2018 Financial Results

On August 9, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company developing highly selective medicines for patients with genomically defined cancers, reported second quarter 2018 financial results (Press release, , AUG 9, 2018, View Source [SID1234528569]).

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"We made significant progress across our pipeline in the second quarter," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "In May, our NDA for larotrectinib was accepted by FDA and granted Priority Review. In June, initial clinical data from the ongoing LIBRETTO-001 Phase 1/2 study of LOXO-292 were presented at ASCO (Free ASCO Whitepaper). These data enabled a subsequent FDA meeting, which established a path forward for the program. And lastly, we completed important enabling studies and manufacturing activities for the LOXO-305 IND. In the second half of the year, we are focused on continuing to advance our pipeline, as well as aiding and advising our partners at Bayer in anticipation of a possible larotrectinib regulatory approval. Operationally, we aim to lay the groundwork for the long-term adoption of tumor genomic testing capable of detecting TRK fusions alongside other clinically actionable alterations."

LOXO-292 Regulatory Update

Loxo Oncology recently conducted a meeting with the U.S. Food and Drug Administration (FDA) for LOXO-292. Based on written minutes from FDA, Loxo Oncology expects to submit a new drug application (NDA) for LOXO-292 in late 2019, utilizing data generated from the ongoing LIBRETTO-001 clinical trial. This timeline integrates standard NDA activities which are ongoing and planned, including clinical pharmacology studies, non-clinical studies, and manufacturing. Loxo Oncology expects to file for separate potential indications for two populations of patients: those with RET fusion-positive solid tumors such as lung and thyroid cancer, and those with RET-mutant medullary thyroid cancer (MTC). In both cases, Loxo Oncology expects that patients will be required to have received systemic therapy, progressed following prior treatment and have no satisfactory alternative treatment options.

Recent Highlights

Larotrectinib

Abstract Accepted for Oral Presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress: Clinical data for larotrectinib will be presented at the ESMO (Free ESMO Whitepaper) 2018 Congress to be held October 19-23, 2018, in Munich, Germany. The presentation will provide updated clinical follow-up for the 55 patients who comprise the primary efficacy analysis population that has supported global regulatory filings. The presentation will also include new data for TRK fusion patients subsequently enrolled.
Abstract Accepted for Poster Presentation at the Molecular Analysis for Personalised Therapy 2018 Congress: On September 15, 2018, Ventana Medical Systems, Inc., a member of the Roche Group, and Loxo Oncology, will present a co-authored poster on the analytical validation of Ventana’s pan-TRK IHC assay at the Molecular Analysis for Personalised Therapy 2018 Congress, to be held September 14-15, 2018 in Paris, France.
Acceptance of NDA by FDA: On May 29, 2018, Loxo Oncology announced FDA acceptance of the larotrectinib NDA and granting of Priority Review for the treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring an NTRK gene fusion. The FDA has set a target action date of November 26, 2018, under the Prescription Drug User Fee Act (PDUFA). More information can be found here.
International Symposium on Pediatric Neuro-Oncology (ISPNO) Poster Presentation: On July 1, 2018, a poster presentation at ISPNO 2018 detailed a case report of a pediatric patient with TRK fusion high-grade glioma treated with larotrectinib on a single patient protocol. The poster can be found here.
World Congress on Gastrointestinal Cancer Oral Presentation: On June 22, 2018, an oral presentation at the ESMO (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer detailed larotrectinib data in patients with TRK fusion gastrointestinal (GI) cancers enrolled to the pivotal larotrectinib program. The presentation can be found here.
Journal of Clinical Investigation Publication: On June 19, 2018, an article was published online in the peer-reviewed Journal of Clinical Investigation detailing the occurrence of TRK fusions in hematologic malignancies, including a case report of a patient with TRK fusion acute myeloid leukemia (AML) treated with larotrectinib on a single patient protocol. The publication can be found here.
Pediatric Blood & Cancer Publication: On June 12, 2018, a case report was published in the online edition of the peer-reviewed journal Pediatric Blood & Cancer detailing a pediatric patient with metastatic TRK fusion congenital mesoblastic nephroma (CMN) treated with larotrectinib on the SCOUT trial. The publication can be found here.
LOXO-292

Interim Clinical Data Presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting: In June, interim clinical data from the LOXO-292 global LIBRETTO-001 trial were presented in an oral presentation at ASCO (Free ASCO Whitepaper). The presentation included 82 patients enrolled across eight dose escalation cohorts and employed an April 2, 2018 data cut-off. The data demonstrated a 77 percent (95% CI: 61-89%) overall response rate (ORR) in RET fusion cancers and a 45 percent (95% CI: 24-68%) ORR in RET mutated MTC as evaluated by RECIST 1.1 criteria. Patients had received a median of three prior treatments with two-thirds having been treated with at least one prior multi-kinase inhibitor. All responding patients across all tumor types remained on therapy as of the data cut-off. LOXO-292 was well tolerated with most treatment-emergent adverse events Grade 1 in severity. The treatment-emergent adverse events observed in ≥10% of patients, regardless of relationship to LOXO-292, were fatigue, diarrhea, constipation, dry mouth, nausea, and dyspnea. Phase 2 cohorts are open and enrolling at the 160mg BID dose. See the presented data here.
LOXO-305

Presentation Accepted at the Society of Hematologic Oncology (SOHO) Annual Meeting: In September 2018, Loxo Oncology authors will present LOXO-305 preclinical characterization data in oral and poster presentations at the SOHO Annual Meeting taking place September 12-15 in Houston, Texas.
Upcoming Milestones

Larotrectinib (TRK)
Presentation of updated clinical data at the ESMO (Free ESMO Whitepaper) Congress
Submission of a Marketing Authorisation Application in the European Union, by Bayer, is expected in the second half of 2018
NDA PDUFA date of November 26, 2018
LOXO-195 (next-generation TRK)
Updated clinical data is now expected in the first half of 2019
LOXO-292 (RET)
Updated clinical data is expected in the second half of 2018
LOXO-305 (BTK)
Presentation of preclinical data at the SOHO Annual Meeting
Initiation of a Phase 1 clinical trial is expected in the fourth quarter of 2018
Second Quarter 2018 Financial Results

As of June 30, 2018, Loxo Oncology had aggregate cash, cash equivalents and investments of $706.4 million, compared to $626.2 million as of December 31, 2017.

Revenue from the collaboration agreement was $42.6 million for the second quarter of 2018, compared to none for the second quarter of 2017. This represents $51.2 million in revenue recognized from the $400 million upfront payment from the Bayer collaboration offset by $8.6 million, Loxo Oncology’s share of the joint larotrectinib co-promotion costs.

Revenue from the collaboration agreement was $81.0 million for the six months ended June 30, 2018, compared to none for the six months ended June 30, 2017. This represents $94.1 million in revenue recognized from the $400 million upfront payment from the Bayer collaboration offset by $13.1 million, Loxo Oncology’s share of the joint larotrectinib co-promotion costs. Loxo Oncology recognizes revenue from the upfront payment on a proportional performance basis utilizing a calculation based on quarterly research and development spending associated with larotrectinib and LOXO-195, relative to cumulative and forecasted research and development spending on larotrectinib and LOXO-195 over the course of the collaboration agreement. As a result, the quarterly revenue recognized for the upfront payment varies from quarter to quarter. A supporting schedule that shows the different components of revenue from the collaboration agreement is included with the attached financial statements.

Research and development expenses were $41.6 million for the second quarter of 2018 compared to $24.4 million for the second quarter of 2017. This increase was primarily due to expanded development expenses across our LOXO-292 and LOXO-305 programs and higher employment costs primarily due to increased headcount. These numbers are net of 50/50 cost-sharing with Bayer for larotrectinib and LOXO-195 development costs. Loxo Oncology recognized research and development-related stock-based compensation expense of $5.8 million during the second quarter of 2018 as compared to $3.5 million for the second quarter of 2017.

Research and development expenses were $73.5 million for the six months ended June 30, 2018 compared to $44.6 million for the six months ended June 30, 2017. This increase was primarily due to expanded development expenses across our LOXO-292 and LOXO-305 programs and higher employment costs primarily due to increased headcount. These numbers are net of 50/50 cost-sharing with Bayer for larotrectinib and LOXO-195 development costs. Loxo Oncology recognized research and development-related stock-based compensation expense of $10.1 million during the six months ended June 30, 2018 as compared to $5.9 million for the six months ended June 30, 2017.

General and administrative expenses were $15.7 million for the second quarter of 2018 compared to $6.5 million for the second quarter of 2017. The increase was primarily due to additional headcount and associated employment costs and general and administrative professional fees. Loxo Oncology recognized general and administrative-related stock-based compensation expense of $6.5 million during the second quarter of 2018 compared to $2.0 million for the second quarter of 2017.

General and administrative expenses were $27.9 million for the six months ended June 30, 2018 compared to $11.3 million for the six months ended June 30, 2017. The increase was primarily due to additional headcount and associated employment costs and general and administrative professional fees. Loxo Oncology recognized general and administrative-related stock-based compensation expense of $11.9 million during the six months ended June 30, 2018 compared to $3.5 million for the six months ended June 30, 2017.

Net loss was $11.7 million and $15.3 million for the three and six months ended June 30, 2018, respectively, compared to $30.4 million and $54.9 million for the three and six months ended June 30, 2017, respectively. This decrease in net loss is primarily driven by the revenue recognized from the $400.0 million upfront payment from the Bayer collaboration, the larotrectinib and LOXO-195 development reimbursement from the Bayer collaboration, offset by increases in operating expenses.

Non-GAAP net loss was $50.6 million and $87.4 million for the three and six months ended June 30, 2018, respectively, compared to $25.0 million and $45.5 million for the three and six months ended June 30, 2017, respectively. This non-GAAP net loss measure, more fully described below under "Non-GAAP Financial Measures," excludes the recognition of collaboration revenue related to the Bayer upfront payment and share-based compensation expenses. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

Earnings Conference Call and Webcast Information
Loxo Oncology will host a conference call today at 8:00 a.m. ET to discuss the second quarter 2018 financial results and company updates. A live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the company’s website at www.loxooncology.com. The conference call can be accessed by dialing (877) 930-8065 (domestic) or (253) 336-8041 (international) and referring to conference ID 7291605. The webcast will be archived and made available for replay on the company’s website beginning approximately two hours after the event.

Nordic Nanovector ASA: Invitation to Second Quarter and First Half 2018 Results Presentation and Webcast

On August 8, 2018 Nordic Nanovector ASA (OSE: NANO) reported its second quarter and first half 2018 results on Wednesday, 22 August 2018 (Press release, Nordic Nanovector, AUG 8, 2018, View Source [SID1234553496]).

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Second Quarter and First Half 2018 Results Presentation and Webcast

A presentation by Nordic Nanovector’s senior management team in English will take place at 8:30 am CEST on 22 August at:

Thon Hotel Vika Atrium, Munkedamsveien 45, 0250 Oslo

Meeting Room: AKER

The presentation will be recorded as a webcast and will be available at www.nordicnanovector.com in the section: Investors & Media

The results report and the presentation will be available at www.nordicnanovector.com in the section: Investors & Media/Reports and Presentations/Interim Reports/2018 from 7:00 am CEST the same day.

Results presentation in Norwegian

A separate presentation of the results in Norwegian, to be hosted by Nordic Nanovector’s CFO, and its VP IR & Corporate Communications, will take place on Thursday, 23 August 2018 at 8:30 am CEST at:

Thon Hotel Vika Atrium, Munkedamsveien 45, 0250 Oslo

Meeting Room: VIPPETANGEN

To attend this meeting please email – [email protected]

The presentation will NOT be recorded as a webcast