AmpliPhi Biosciences Reports Second Quarter 2018 Financial Results and Business Highlights

On August 9, 2018 AmpliPhi Biosciences Corporation (NYSE American: APHB), a clinical-stage biotechnology company focused on precisely targeted bacteriophage therapeutics for antibiotic-resistant infections, reported financial results for the second quarter ended June 30, 2018 (Press release, AmpliPhi Biosciences, AUG 9, 2018, View Source [SID1234528749]). AmpliPhi Biosciences will not be conducting a conference call in conjunction with this financial release.

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"I’m delighted to report that we have made substantial progress with our expanded access program for AB-SA01 and AB-PA01," said Paul C. Grint, M.D., CEO of AmpliPhi Biosciences. "As of today, we have treated a total of nineteen patients at seven different hospitals and we are encouraged by the results. We are analyzing the data and plan to share a more detailed update in the near future. We look forward to our two meetings with the FDA over the next two months, the goal of which is to obtain important clinical development feedback for Phase 2 and potentially pivotal trials in one or more indications."

Recent Business Highlights

Two meetings are scheduled with the FDA for August and September to discuss Phase 2 trial plans and the path forward to regulatory approval for investigational drug candidates AB-SA01, targeting Staphylococcus aureus (S. aureus), and AB-PA01, targeting Pseudomonas aeruginosa (P. aeruginosa).
North America’s first Center for Innovative Phage Applications and Therapeutics (IPATH) was launched by UC San Diego (UCSD) in June 2018 with AmpliPhi as an industry partner. As part of the IPATH launch, UCSD announced the successful case of a patient treated with AmpliPhi’s AB-SA01. Prior to phage therapy, the patient had a persistent S. aureus ventricular assist device infection that was not eradicated for three years despite antibiotic treatment.
Presented four case studies of critically ill patients suffering from severe S. aureus bloodstream infections, who received treatment with AB-SA01, at the American Society for Microbiology (ASM) Microbe 2018 annual meeting in Atlanta in June 2018. In all cases, standard medical and surgical therapy was considered inadequate before starting bacteriophage therapy. AB-SA01 was well-tolerated and bacterial elimination was demonstrated in three out of four patients.
Presented a successful case study of a patient with cystic fibrosis (CF), suffering from recurrent multi-drug resistant (MDR) P. aeruginosa pneumonia and multiple CF exacerbations, who received treatment with AB-PA01, at the 41st European Cystic Fibrosis Conference in Belgrade, Serbia, in June 2018. Prior to treatment with AB-PA01, the patient received multiple courses of antibiotics, including colistin, but due to renal failure, colistin administration was discontinued. Treatment with AB-PA01 was well tolerated and the patient’s infection resolved. No recurrence of pneumonia or CF exacerbation was reported during the two-month follow-up period after the completion of treatment with AB-PA01. The patient’s renal failure resolved.
Presented a successful case study of a lung transplant recipient suffering from recurrent episodes of MDR P. aeruginosa pneumonia who received treatment with bacteriophage therapeutics, including AB-PA01, at the International Society of Heart and Lung Transplant Annual Meeting in Nice, France, in April 2018. The patient clinically responded to bacteriophage and antibiotic therapy with resolution of pneumonia and improved respiratory status.
Utilized the Therapeutic Development Services funded by the National Institute of Allergy and Infectious Disease (NIAID), part of the National Institutes of Health (NIH), to conduct further preclinical studies of AB-SA01. The Therapeutic Development Services program funds the provision of preclinical services for selected companies and researchers in order to advance development of promising interventional agents.
Second Quarter and Six Months Ended June 30, 2018 Financial Results

Research and development (R&D) expenses for the second quarter of 2018 were $1.7 million compared to $1.1 million for the second quarter of 2017, primarily attributable to a $0.2 million increase in professional and consulting fees and a $0.2 million increase in clinical costs.
R&D expenses for the six months ended June 30, 2018 increased by $0.6 million to $3.2 million from $2.6 million for the six months ended June 30, 2017, primarily due to an increase in clinical costs.
General and administrative (G&A) expenses were $1.4 million for the second quarter of 2018 compared to $2.8 million for the second quarter of 2017. The decrease was primarily due to lower payroll-related costs, lower legal and professional fees, as well as a $0.6 million decrease in non-cash stock-based compensation and other non-cash charges.
G&A expenses for the first six months of 2018 decreased by $1.7 million to $3.0 million from $4.7 million for the first six months of 2017. The decrease was primarily attributable to a decrease in the items described above for the second quarter comparison.
Net cash used in operating activities for the six months ended June 30, 2018 was $5.7 million compared to $6.2 million for the six months ended June 30, 2017.
Cash and cash equivalents as of June 30, 2018 totaled $5.8 million.
In July 2018, the Company received $1.2 million of tax rebate incentive payments in cash from the Australian tax authority. The incentive payments are based on R&D activities in Australia in 2017.
As of August 6, 2018, there were 16.5 million shares of common stock outstanding.

BioCardia, Inc. Reports Second Quarter 2018 Financial Results and Business Highlights

On August 9, 2018 BioCardia, Inc. [OTC: BCDA], a leader in the development of comprehensive solutions for cardiovascular regenerative therapies, reported financial results and business highlights for the second quarter 2018 and filed its quarterly report on Form 10-Q for the three and six months ended June 30, 2018 with the Securities and Exchange Commission (Press release, Biocardia, AUG 9, 2018, View Source [SID1234528586]).

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Second Quarter 2018 Financial Results:

Revenues increased to $239,000 for the second quarter of 2018 compared to $112,000 for the second quarter of 2017.
Net loss was $3.2 million for the second quarter of 2018, compared to $2.9 million for the second quarter of 2017.
Research and development expenses increased to $2.0 million in the second quarter of 2018, compared to $1.3 million in the second quarter of 2017, primarily due to expenses associated with the pivotal CardiAMP Heart Failure Trial and development of the Company’s second cell therapy candidate, the CardiALLO Cell Therapy System.
Selling, general and administrative expenses decreased to $1.3 million in the second quarter of 2018, compared to $1.6 million in the second quarter of 2017, primarily due to lower stock compensation expense.
Net cash used in operations in the second quarter of 2018 was $2.8 million, as compared to $2.1 million in the second quarter of 2017.
2018 Corporate Highlights:

Enrollment in the pivotal CardiAMP Heart Failure Trial has expanded to 17 world class centers in the United States as detailed on ClinicalTrials.Gov.
Two abstracts have been accepted for presentation at the Transcatheter Cardiovascular Therapeutics (TCT) conference in San Diego, one of the world’s largest and most important educational meeting specializing in interventional cardiovascular medicine. Both presentations are scheduled for September 24, 2018:
"The CardiAMP Heart Failure Study: First Results from Unblinded Roll-In Phase", to be presented at 9:06 am PDT.
"Performance of Helix Transendocardial Biotherapeutic Delivery System after 300 Cases", to be presented at 9:18 am PDT.
The U.S. Food and Drug Administration approved a second pivotal Investigational Device Exemption trial for the CardiAMP Cell Therapy System for the treatment of chronic myocardial ischemia with refractory angina, the "CardiAMP Chronic Myocardial Ischemia Trial". The Centers for Medicare and Medicaid Services (CMS) has approved this trial for national reimbursement coverage.
The Company received a new US patent related to its CardiAMP cell potency assay, providing further protection to its autologous cell therapy programs in both heart failure and chronic myocardial ischemia indications.
Anticipated Upcoming Milestones:

12-month data from the CardiAMP Heart Failure Trial 10-patient roll-in cohort in the fourth quarter 2018.
Filing of the Investigational New Drug (IND) for the CardiALLO Cell Therapy System, an investigational allogenic culture expanded "off the shelf" mesenchymal cell therapy for the treatment of ischemic heart failure, in the fourth quarter 2018.
Filing for FDA approval of an advanced bidirectional steerable guide system in the fourth quarter 2018.
Dosing of the first patient in the pivotal CardiAMP Chronic Myocardial Ischemia Trial in the first quarter 2019.
Completion of enrollment in the pivotal CardiAMP Heart Failure Trial in the third quarter 2019.

argenx to Present at 2018 Wedbush PacGrow Healthcare Conference

On August 9, 2018 argenx (Euronext & Nasdaq: ARGX) a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported that the Company will present on Tuesday, August 14th at 1:20 p.m. ET at the 2018 Wedbush PacGrow Healthcare Conference in New York (Press release, argenx, AUG 9, 2018, View Source;p=RssLanding&cat=news&id=2363190 [SID1234528607]).

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A live webcast of the presentation will be available on the Company’s website at www.argenx.com. A replay of the webcast will be available for 90 days following the presentation.

Entry into a Material Definitive Agreement.

On August 6, 2018, PRA Health Sciences, Inc. (the "Company") entered into an Underwriting Agreement (the "Underwriting Agreement") by and among the Company, the selling stockholder named therein (the "Selling Stockholder"), and Morgan Stanley & Co. LLC and Goldman Sachs & Co. LLC (the "Underwriters"), relating to an underwritten offering (the "Offering") of 6,500,000 shares (the "Shares") of the Company’s common stock, par value $0.01 per share, pursuant to the Company’s Registration Statement on Form S-3 (File No. 333-209883), filed on March 2, 2016, as supplemented by the prospectus supplement dated August 6, 2018 (Filing, 8-K, PRA Health Sciences, AUG 9, 2018, View Source [SID1234528791]). All of the Shares are being sold by the Selling Stockholder. Pursuant to the Underwriting Agreement, the Underwriters purchased the Shares at a price of $101.01 per share in a transaction that was completed on August 9, 2018.

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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.