On November 12, 2019 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in uro-oncology, reported financial results for the third quarter ended September 30, 2019 and provided an overview of the Company’s recent developments (Press release, UroGen Pharma, NOV 12, 2019, View Source [SID1234551005]).

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"On behalf of the UroGen team, we are very pleased to announce that we have completed the New Drug Application (NDA) submission for UGN-101 to the FDA. This is a very important milestone for UroGen and for patients with low-grade UTUC as UGN-101 has the potential to bring the first primary chemoablation drug to these patients. I am proud of the UroGen team and investigators who have helped to make this key milestone a reality," said Liz Barrett, President and Chief Executive Officer of UroGen. "Beyond UGN-101, we have taken important steps to build a company that can deliver long-term sustainable growth through our focus on high unmet need areas utilizing both local delivery and novel medicines. We’ve demonstrated that relentless commitment with our recently announced positive complete response data for investigational agent UGN-102 in patients with intermediate risk LG NMIBC, and our exclusive collaboration with Agenus to advance therapies for high-grade urinary tract cancers. We look forward to a strong future for UroGen."

Recent Highlights and Upcoming Milestones

UGN-101 (mitomycin gel) for instillation for patients with low-grade upper tract urothelial cancer (LG UTUC):
The Company completed its NDA submission to the FDA, and the FDA has a 60-day filing review period to determine whether the NDA submission is complete. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of LG UTUC. UGN-101 is eligible for Priority Review and the Company is planning for anticipated approval and launch in 1H 2020. UGN-101 plans for launch readiness are on track for January 2020.
The NDA submission is supported by positive data from the pivotal Phase 3 OLYMPUS clinical trial.
UroGen also initiated a retreatment protocol as an extension to OLYMPUS to evaluate the efficacy and safety of retreatment with UGN-101 in patients from the trial who initially achieved a CR and were subsequently found to have a documented recurrence of LG UTUC during follow-up.
UGN-102 (mitomycin gel) in development for intravesical instillation for patients with intermediate risk low-grade non-muscle invasive bladder cancer (LG NMIBC):
UroGen recently presented interim results from the single-arm, open-label Phase 2b OPTIMA II trial of investigational UGN-102 for patients with intermediate risk LG NMIBC. LG NMIBC is defined as those with one or two of the following criteria: multifocal disease, large tumors and rapid rates of recurrence.
In an interim analysis, 63% (20/32) of patients from the Phase 2b OPTIMA II trial achieved a CR. Patients will continue to be followed with 12-month durability to be reported at a later date.
The most common treatment emergent adverse events (TEAEs) included dysuria, pollakiuria, fatigue, hematuria and urinary tract infection. The majority were characterized as mild or moderate in severity and transient.
The trial completed enrollment of 63 patients ahead of schedule at clinical sites across the U.S. and Israel. The Company intends to initiate a pivotal Phase 3 trial in 2020 following a planned discussion with the FDA in 1Q 2020.
This is a patient population whereby the current standard of care, transurethral resection of bladder tumor, or TURBT, is used repeatedly to address chronic recurrence of disease. These patients experience what can be viewed as a form of surgical failure and many undergo multiple surgical procedures during life to "manage" bladder cancer recurrences.
There are no drugs currently approved by the FDA as first-line treatment for intermediate risk LG NMIBC. UGN-102 has the potential to provide a non-surgical alternative for the treatment of chronic relapse for approximately 80,000 patients characterized as intermediate risk.
Pipeline Advancement:
UGN-201 (TLR 7/8 agonist) for patients with high-grade NMIBC:
UroGen is exploring the utility of UGN-201 in the context of novel combinatorial immunotherapy for NMIBC. Nonclinical data suggests an efficacy signal when UGN-201 is administered locally with anti-CTLA4 antibody in a murine model of high-grade bladder cancer. The Company plans to optimize combinations and advance into human studies.
Exclusive Worldwide License Agreement with Agenus Inc. to Advance Treatment of High-Grade Urinary Cancers
UroGen entered into an exclusive worldwide license agreement with Agenus to develop and commercialize zalifrelimab (AGEN1884, anti-CTLA-4 antibody) in combination with UGN-201 for the treatment of urinary tract cancers via intravesical delivery. Zalifrelimab (AGEN1884, anti-CTLA-4 antibody) is currently being evaluated by Agenus as a monotherapy in PD-1 refractory patients.
The initial indication for development will be high-grade NMIBC.
UroGen will be responsible for all development and commercialization activities.
Third Quarter 2019 Financial Results; 2019 Guidance

As of September 30, 2019, cash, cash equivalents and marketable securities totaled $221.7 million.
Research and development expenses for the three months ended September 30, 2019 were $9.5 million, including non-cash share-based compensation expense of $2.1 million. Research and development expenses for the nine months ended September 30, 2019 were $29.2 million, including non-cash share-based compensation expense of $6.4 million.
General and administrative expenses for the three months ended September 30, 2019 were $14.0 million, including non-cash share-based compensation expense of $5.2 million. General and administrative expenses for the nine months ended September 30, 2019 were $40.5 million, including non-cash share-based compensation expense of $15.5 million.
The Company reported a net loss of $22.3 million, or basic and diluted net loss per ordinary share of $1.06, for the three months ended September 30, 2019. The Company reported a net loss of $66.2 million, or basic and diluted net loss per ordinary share of $3.25, for the nine months ended September 30, 2019.
Including the recently announced Agenus deal, the Company is still on track to end the year with a net loss for the year in the range of $100 to $110 million, which is expected to include non-cash stock-based compensation expense in the range of $28 to $30 million, subject to market conditions.
Conference Call & Webcast Information

Members of UroGen’s management team will host a live conference call and webcast today at 8:30 am Eastern Time to review the Company’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (888) 771-4371 (U.S.) or (847) 585-4405 (International) to listen to the live conference call. The conference ID number for the live call will be 49159339. An archive of the webcast will be available for two weeks on the Company’s website.

On November 12, 2019 Gossamer Bio, Inc. (Nasdaq:GOSS), a clinical-stage biopharmaceutical company, reported that it has entered into a clinical collaboration agreement with Merck (known as MSD outside the US and Canada) to evaluate the combination of Gossamer’s investigational product candidate GB1275, the first-in-class oral modulator of CD11b, and Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in patients with selected advanced solid tumors (Press release, Gossamer Bio, NOV 12, 2019, View Source [SID1234551004]).

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Collaboration with Merck

Under the terms of the agreement, Gossamer will conduct a Phase 1/2 study, KEYNOTE-A36, in advanced solid tumors, with the Phase 1 consisting of dose escalation of three GB1275 regimens including one with KEYTRUDA. Additionally, the Phase 2 consists of expansion cohorts evaluating GB1275 in combination with KEYTRUDA or chemotherapy. Gossamer owns worldwide commercial rights to GB1275.

"We are very excited to collaborate with Merck, an established leader in cancer immunotherapy, on our fourth clinical asset and first oncology agent, GB1275, as we work to improve the lives of cancer patients," said Sheila Gujrathi, M.D., Chief Executive Officer of Gossamer.

"Immunotherapies are revolutionizing the treatment of patients with certain types of cancers, but there remain many tumor types in which existing immunotherapies have not demonstrated sufficient efficacy," said Jakob Dupont, M.D., Chief Medical Officer of Gossamer. "Based on the preclinical data and the novel mechanism targeting the CD11b receptor, we believe GB1275’s effect on the immunosuppressive mechanisms in the tumor microenvironment will lead to enhanced anti-tumor activity in combination with KEYTRUDA or chemotherapy."

GB1275 is an oral modulator of CD11b, a receptor broadly expressed on immunosuppressive myeloid cells found within the tumor microenvironment (TME). In preclinical studies, GB1275 has been shown to reduce the recruitment of these cells into the TME, while also converting the cells within the TME from an immunosuppressive to an active state. The FDA has granted GB1275 orphan drug designation for the treatment of pancreatic cancer.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp, a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Phase 1/2 Trial Update

The KEYNOTE-A36 Phase 1/2 trial is underway, enrolling patients at a number of centers of excellence in the United States and United Kingdom for the treatment of selected solid tumor types, including pancreatic, gastric, esophageal, colorectal, prostate and triple negative breast. The Phase 1 portion of the study consists of dose escalation of GB1275 monotherapy. Subsequently, after clearing several monotherapy dose levels, dose escalation combinations will be initiated with KEYTRUDA or chemotherapy. The primary endpoints of the dose escalation phase of the trial are to evaluate the safety, tolerability, and the pharmacokinetics / pharmacodynamics of all three regimens. Biomarkers of immunohistochemistry and other immune monitoring methodologies will also be evaluated.

Gossamer expects to report initial data from the Phase 1 portion of the study in the second half of 2020. An abstract detailing the scientific background for the trial and the study design was presented at the 34th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper). Additional information about the trial can be found at clinicaltrials.gov (identifier: NCT04060342).

Science Translational Medicine Publication

GB1275 preclinical data generated by labs at the Washington University of St. Louis and Rush University were published as the cover article of the July 3rd edition of Science Translational Medicine. The article, "Agonism of CD11b reprograms innate immunity to sensitize pancreatic cancer to immunotherapies," contains data demonstrating the efficacy of GB1275 monotherapy and combination therapies with anti-PD-1 and chemotherapy in multiple pancreatic tumor models. Of note, GB1275 and anti-PD-1 combination therapy demonstrated statistically superior efficacy as compared to anti-PD-1 combinations with other macrophage or granulocyte depletion mechanisms, including CSF1R neutralization, CCR2i inhibition, and anti-αLy6G.

On November 12, 2019 bluebird bio, Inc. (Nasdaq: BLUE) and Forty Seven, Inc. (Nasdaq:FTSV) reported that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Seven’s novel antibody-based conditioning regimen, FSI-174 (anti-cKIT antibody) plus magrolimab (anti-CD47 antibody), with bluebird’s ex vivo lentiviral vector hematopoietic stem cell (LVV HSC) gene therapy platform (Press release, bluebird bio, NOV 12, 2019, View Source [SID1234551003]). This collaboration will focus on a conditioning approach aimed to deliver reduced toxicity and will initially target diseases that have the potential to be corrected with transplantation of autologous gene-modified blood-forming stem cells. If successful, the new conditioning regimen could allow for more patients to undergo gene therapy.

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Autologous hematopoietic stem cell transplantation (HSCT) and most ex vivo LVV HSC gene therapies require that a patient’s own stem cells first be depleted from the bone marrow to facilitate the engraftment of the new (or gene-modified) HSCs through a process called conditioning. Conditioning is performed using chemotherapy or radiation, which can place patients at risk for infection and require hospitalization until bone marrow cells have recovered. In addition, conventional conditioning can place patients at risk for secondary malignancy and infertility. As a result, the overall toxicity profile of current conditioning regimens limits the types of patients who are eligible for gene therapy. It is hoped that novel antibody based conditioning regimens could avoid these toxicities.

"We are excited about this collaboration, combining our industry-leading LVV HSC gene therapy platform with Forty Seven’s novel antibody-based conditioning regimen," said Philip Gregory, chief scientific officer, bluebird bio. "We believe that, if successful, this novel conditioning modality could not only increase the number of patients and physicians who may consider gene therapy but also improve the overall risk benefit profile for stem cell-based gene therapy, as well as potentially reduce time and costs associated with hospital visits."

"Forty Seven is advancing the pioneering work on CD47 and cKIT from our scientific founder, Irv Weissman’s lab. We have shown that antibody blockade of CD47 can synergize with other antibodies targeting cancer to promote tumor engulfment. Based on this experience, coupled with the results of preclinical studies, we are eager to explore this dual-antibody approach for the potential treatment of non-malignant diseases," says Jens Peter Volkmer, M.D., Founder and Vice President of Research and Development at Forty Seven.

Forty Seven’s President and Chief Executive Officer, Mark McCamish, M.D., Ph.D., commented, "bluebird is a leading gene therapy company and we are excited to collaborate with them. Stem cell transplantation is potentially curative for a variety of blood diseases, including genetic blood disorders like sickle cell disease and beta-thalassemia. If successful, we believe our chemo- and radiation-free, all-antibody approach could expand transplantation beyond genetic blood disorders to a range of indications for which current transplantation approaches are suboptimal. In 2020, we plan to evaluate FSI-174 in healthy volunteers, before initiating a combination study of Forty Seven’s novel all-antibody conditioning regimen and bluebird’s gene therapy product."

Under the terms of the agreement, bluebird bio will provide its ex vivo LVV HSC gene therapy platform and Forty Seven will contribute its innovative antibody-based conditioning regimen for the collaboration.

About FSI-174 and Magrolimab
FSI-174 is a humanized monoclonal antibody targeting cKIT, which is a receptor that is highly expressed on hematopoietic stem cells. Magrolimab is a humanized monoclonal antibody targeting CD47, which is a "don’t eat me" signal to macrophages and is expressed on all cells. Magrolimab is currently being investigated in Phase 2 clinical trials to treat cancer and has established clinical efficacy in four indications, including myelodysplastic syndrome, acute myeloid leukemia, diffuse large B cell lymphoma and follicular lymphoma, with a favorable safety profile in over 350 patients treated, including some patients treated continuously for over two years. When combined, FSI-174 sends a positive signal to macrophages to target blood forming stem cells for removal and magrolimab disengages inhibitory signals that block phagocytosis. Combination of these antibodies has shown efficient removal of blood forming stem cells, allowing for transplantation in pre-clinical models.

On November 12, 2019 AVEO Oncology (NASDAQ: AVEO) reported financial results for the third quarter ended September 30, 2019 and provided a business update (Press release, AVEO, NOV 12, 2019, View Source [SID1234551002]).

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"This quarter we have made meaningful progress toward our goal of bringing effective and better tolerated therapies to patients battling kidney and other cancers," said Michael Bailey, president and chief executive officer of AVEO. "We are working to submit our New Drug Application (NDA) in the first quarter of 2020 for tivozanib as a treatment for relapsed/refractory renal cell carcinoma (RCC) and to provide a final overall survival (OS) update for the TIVO-3 study in June 2020. We also continue to make progress in evaluating tivozanib-immunotherapy combinations, with recently reported positive results from the TiNivo study of tivozanib and OPDIVO (nivolumab) in RCC and the initiation of the Phase 1b/2 DEDUCTIVE study with IMFINZI (durvalumab) in hepatocellular carcinoma. Ficlatuzumab also continues to advance in the clinic, with the initiation of CyFi-2, a Phase 2 randomized study in patients with relapsed and refractory acute myeloid leukemia (AML). Critical to this progress, we continue to maintain a strong balance sheet that we believe provides us with a cash runway into the second quarter of 2021."

Tivozanib North America Regulatory and Phase 3 TIVO-3 Study Updates

Announced Plans for TIVO-3 Final OS Analysis and NDA Submission for Tivozanib. Last week, AVEO provided a regulatory update following a meeting with the U.S. Food and Drug Administration (FDA) to discuss results from the August 2019 OS analysis of the TIVO-3 trial and the Company’s proposal to proceed with an NDA for tivozanib in relapsed/refractory RCC.
The Company intends to submit an update to the TIVO-3 statistical analysis plan to the FDA allowing for the final OS analysis to be conducted, followed by an NDA submission in the first quarter of 2020, and expects to report results from the final OS analysis of the TIVO-3 trial in June 2020. The FDA and the Company agreed that if, during the review, the final analysis yields an OS hazard ratio (HR) above 1.00, the Company will withdraw its NDA application. The FDA informed the Company that an Oncologic Drugs Advisory Committee panel would likely be convened to review the final tivozanib data package.

TIVO-3 is the Company’s Phase 3 randomized, controlled, multi-center, open-label study to compare tivozanib, the Company’s vascular endothelial growth factor receptor tyrosine kinase inhibitor (VEGFR-TKI), to sorafenib in 350 subjects with highly refractory metastatic RCC.

Announced Updated Overall Survival HR of 0.99 in Phase 3 TIVO-3 Trial of Tivozanib in RCC. In September 2019, AVEO announced results from the second prespecified analysis of OS in the TIVO-3 trial. These results included an OS HR, which assesses the relative risk of death for the entirety of the dataset, that was below 1.00 (HR=0.99; 95% CI: 0.76-1.29; p=0.95).
The data cutoff date for the second prespecified analysis was August 15, 2019, two years from the last patient enrolled and approximately ten months from the data cutoff date for the first prespecified analysis. Between the two data cutoff dates, 16 additional OS events were reported on the tivozanib arm and 28 on the sorafenib arm, resulting in a total of 114 OS events on the tivozanib arm and 113 on the sorafenib arm. Median OS, a point in time value of the OS when half of the patients within each arm are still alive, was 16.4 months for tivozanib (95% CI: 13.4-22.2) and 19.7 months for sorafenib (95% CI: 15.0-24.2). As of the second data cutoff date, twenty patients remained progression free on the tivozanib arm and two on the sorafenib arm, with a median duration on study of 32.5 months.

Updated Data from TIVO-3 Trial to be Presented at the 18th International Kidney Cancer Symposium. Updated data from the TIVO-3 trial will be presented during an oral session at the 18th International Kidney Cancer Symposium being held November 15-16, 2019 in Miami. The presentation, titled "Overall Survival from Phase 3 TIVO-3 Study in Advanced Renal Cell Carcinoma", will be presented on Saturday, November 16 at 9:42 a.m. Eastern Time. The presentation will include the recently announced OS data, as well as new data from important subgroups. A copy of the slides will be available in the Publications & Presentations Section of AVEO’s website following the presentation.
Additional Tivozanib Updates

Announced Initiation of Enrollment in Phase 1b/2 DEDUCTIVE Study of Tivozanib in Combination with IMFINZI (durvalumab) in Previously Untreated Metastatic HCC. In September 2019, AVEO announced the initiation of enrollment in the DEDUCTIVE trial, an open-label, multi-center Phase 1b/2 clinical trial evaluating tivozanib in combination with IMFINZI (durvalumab), AstraZeneca’s human monoclonal antibody directed against programmed death-ligand 1 (PD-L1), in patients with HCC who have not received prior systemic therapy. The trial is being conducted as part of a clinical collaboration between AVEO and AstraZeneca. AVEO is serving as the study sponsor, with study costs shared equally by both parties and clinical drug supplied by each respective company.
Presented Final PFS Results from Phase 2 Portion of the TiNivo Study of Tivozanib and OPDIVO (nivolumab) in Advanced or Metastatic RCC. In September 2019, AVEO and EUSA Pharma announced the presentation of final progression free survival (PFS) results from the Phase 2 portion of the TiNivo study, a Phase 1b/2 multicenter trial of tivozanib in combination with OPDIVO (nivolumab), Bristol-Myers Squibb’s immune checkpoint, or PD-1, inhibitor, for the treatment of advanced or metastatic RCC. The data were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Annual Congress in Barcelona, Spain.
The combination required few dose reductions and showed additive or synergistic activity for objective response rate and PFS in both treatment naïve and previously treated patients with metastatic RCC. Overall median PFS for the 25 patients treated at the study’s full dose and schedule was 18.9 months (95% CI: 16.4; NR). Median PFS for previously untreated patients (n=12) was 18.5 months, while median PFS for previously treated patients (n=13) had not yet been reached as of the August 27, 2019 data cutoff date. An objective response rate was observed in 56% of patients (complete responses + partial responses), including one treatment naïve patient (1/12) achieving a complete response, and disease control (complete response + partial response + stable disease) was observed in 96% of patients. The most common treatment-related Grade 3/4 adverse event was hypertension, and only 17% of patients required a dose reduction.

A copy of the presentation is available in the Publications & Presentations Section of AVEO’s website.

Announced Kyowa Kirin Buy Back of Tivozanib Non-Oncology Rights from AVEO. In August 2019, AVEO and Kyowa Kirin Co., Ltd. announced that the companies’ license agreement for tivozanib has been amended to allow Kyowa Kirin to buy back the non-oncology rights of tivozanib in AVEO’s territories, which includes the U.S. and EU. Under the terms of the amended license agreement, AVEO received a $25 million upfront payment and a waiver of the $18 million milestone payment due to Kyowa Kirin upon AVEO obtaining U.S. market approval for tivozanib. In addition, AVEO will be eligible to receive up to $391 million in milestone payments upon the successful achievement of certain development and commercial objectives related to tivozanib formulations for the treatment of non-oncology indications. AVEO is also eligible to receive tiered royalty payments on net sales in these indications, which range from a high single-digit to low double-digit percent.
Ficlatuzumab Update

Initiation of the CyFi-2 Study of Ficlatuzumab in Relapsed and Refractory AML. Last week, AVEO and Biodesix, Inc. announced the initiation of the CyFi-2 study, a randomized Phase 2 clinical study evaluating ficlatuzumab, AVEO’s potent hepatocyte growth factor (HGF) inhibitory antibody product candidate, in combination with high-dose cytarabine vs. high-dose cytarabine alone in patients with relapsed and refractory AML.
AVEO will sponsor the CyFi-2 study, which is expected to enroll approximately 60 patients with AML who failed induction chemotherapy or who achieved a complete response but relapsed within one year. The CyFi-2 study is being conducted as part of the companies’ worldwide partnership to develop and commercialize ficlatuzumab. Under the terms of this agreement, AVEO and Biodesix equally share all development costs.

Recent Corporate Update

Appointment of Key Regulatory, Commercial and Medical Affairs Leadership Roles. AVEO announced today the appointment of three individuals to key leadership roles during the fourth quarter:

Darlene Noci, Interim Head of Regulatory Affairs. Ms. Noci brings to AVEO over 20 years of leadership experience in global regulatory affairs and strategic drug development. She received a Bachelor’s degree in Political Science from Adelphi University and a Master’s degree in Government from Harvard University.
Kevin Peacock, Vice President of Marketing. Mr. Peacock brings over 15 years of commercial leadership experience in oncology marketing, strategic planning, and business analytics. He received his Bachelor’s degree in Business Administration from Temple University.
Daniel Powers, D.O., Vice President of Medical Affairs. Dr. Powers brings to AVEO over 20 years of clinical, academic, and biopharmaceutical medical affairs experience. He received his Bachelor’s degree in Chemistry from the University of Massachusetts Boston and a Doctor of Osteopathic Medicine degree from Rowan University. He completed his internship and residency in Internal Medicine at Yale-New Haven Hospital and National Navy Medical Center.
Third Quarter 2019 Financial Results

AVEO ended Q3 2019 with approximately $57.7 million in cash, cash equivalents and marketable securities as compared with $24.4 million at December 31, 2018.
Total revenue for Q3 2019 was approximately $25.7 million compared with $2.5 million for Q3 2018.
Research and development expense for Q3 2019 was $4.0 million compared with $5.2 million for Q3 2018.
General and administrative expense for Q3 2019 was $2.9 million compared with $2.7 million for Q3 2018.
Net income for Q3 2019 was $16.4 million, or net income of $0.10 per basic and diluted share, respectively, compared with a net loss of $22.2 million for Q3 2018, or a loss of $0.18 per basic and diluted share, respectively.
On August 1, 2019, as scheduled and included in the Company’s cash guidance below, the Company resumed principal payments of approximately $0.8 million per month on the $20.0 million Hercules loan that matures on July 1, 2021.
Financial Guidance

AVEO believes that its cash, cash equivalents and marketable securities of approximately $57.7 million at September 30, 2019 would allow the Company to fund its planned operations into the second quarter of 2021. This estimate is a change from the Company’s prior quarter guidance as a result of the Company’s plan to file an NDA for tivozanib, reflecting additional costs related to the NDA filing, as well as limited commercial launch-readiness activities.

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI) discovered by Kyowa Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union plus Norway, New Zealand and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC4. Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal, ovarian and breast cancers.

About Ficlatuzumab

Ficlatuzumab (formerly known as AV-299) is a potent hepatocyte growth factor (HGF) inhibitory antibody that binds to the HGF ligand with high affinity and specificity to inhibit HGF/c-Met biological activities. AVEO and Biodesix, Inc. have a worldwide agreement to develop and commercialize ficlatuzumab. Ficlatuzumab is currently being evaluated in squamous cell carcinoma of the head and neck (SCCHN), metastatic pancreatic ductal cancer (PDAC), and acute myeloid leukemia (AML).

On November 12, 2019 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") reported financial results for the three- and nine-month periods ended September 30, 2019 (Press release, Eagle Pharmaceuticals, NOV 12, 2019, View Source [SID1234551001]). Third quarter and recent highlights include:

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Invested $12 million in research and development and external legal costs to advance Eagle’s pipeline.
Eagle’s Japanese marketing partner, SymBio Pharmaceuticals Limited, submitted a New Drug Application ("NDA") for TREAKISYM, bendamustine ready-to-dilute liquid formulation, in Japan in September. Approval is expected in Q4 2020, which would trigger a $5 million milestone payment to Eagle. Potential payments to Eagle could reach $10 to $25 million per year in royalties and milestones.
Advanced clinical development plans for Eagle’s innovative fulvestrant program, which has the potential to change the treatment of estrogen receptor-positive breast cancer. The program aims to determine if the unique properties of Eagle’s product will result in greater inhibition of estrogen receptors and better patient outcomes compared to currently available treatment options. The Company expects to dose the first subject in December.
Enrolled additional patients in its controlled clinical study of RYANODEX (dantrolene sodium for injectable suspension) for the treatment of exertional heat stroke ("EHS") patients during the 2019 Hajj pilgrimage held from August 9-14 in Saudi Arabia. The Company has recruited a total of 41 patients at the 2015, 2018 and 2019 Hajj pilgrimages. Eagle has submitted a plan to the U.S. Food and Drug Administration ("FDA") that proposes reviewing the data collectively for all 41 patients. If FDA agrees with this plan, Eagle plans to resubmit the NDA for EHS in response to the Complete Response Letter received in 2017.
The Company, in dialogue with FDA, has received further clarity regarding RYANODEX for the treatment of brain damage secondary to nerve agent exposure. FDA has recommended that, under the animal rule, an additional study be conducted in a second species. Eagle expects to file a supplement to the current NDA in the second half of 2020.
Total revenue for Q3 2019 was $41.1 million, compared to $51.3 million in Q3 2018, primarily reflecting lower BENDEKA royalty revenue and lower product sales of BELRAPZO and RYANODEX, partially offset by higher product sales of BENDEKA.
Q3 2019 net loss was $2.4 million, or $0.17 per basic and diluted share, compared to net income of $14.0 million, or $0.94 per basic and $0.91 per diluted share in Q3 2018.
Q3 2019 adjusted non-GAAP net income was $3.7 million, or $0.27 per basic and $0.26 per diluted share, compared to adjusted non-GAAP net income of $18.3 million, or $1.22 per basic and $1.18 per diluted share, in Q3 2018.
Cash and cash equivalents were $117.2 million, net accounts receivable was $44.8 million, and debt was $40.0 million as of September 30, 2019.
"In the third quarter, we invested over $12 million to further advance our pipeline. This includes $9 million in non-GAAP R&D expense as well as $3 million in external legal expense related to the pemetrexed and vasopressin litigations. We are advancing our pipeline, as evidenced by the news today on EHS, nerve agent and the planned initiation of our next clinical trial for fulvestrant. We are also pleased that our bendamustine program is expanding to Japan, and we are expecting $10-$25 million in annual royalty and milestone payments beginning in 2021. This is an exciting time for Eagle as we move closer to realizing the full potential of many of our late-stage products," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.

Third Quarter 2019 Financial Results

Total revenue for the three months ended September 30, 2019 was $41.1 million, as compared to $51.3 million for the three months ended September 30, 2018.

Royalty revenue was $26.5 million in the third quarter of 2019, compared to $35.2 million in the third quarter of 2018. BENDEKA royalties were $26.2 million in the third quarter of 2019, compared to $33.8 million in the third quarter of 2018. A summary of total revenue is outlined below:

Gross Margin was 64% during the third quarter of 2019, as compared to 75% in the third quarter of 2018. The compression in gross margin in the third quarter of 2019 was primarily driven by an increase in BENDEKA product sales to our marketing partner, on which Eagle earns no profit, and the decrease in BENDEKA royalty revenue.

R&D expense was $10.2 million for the third quarter of 2019, compared to $6.0 million in the third quarter of 2018. The increase is largely attributable to spending on fulvestrant and vasopressin. Excluding stock-based compensation and other non-cash and non-recurring items, R&D expense during the third quarter of 2019 was $9.0 million.

SG&A expense in the third quarter of 2019 increased to $18.5 million compared to $13.9 million in the third quarter of 2018. External legal spend associated with litigation on pemetrexed and vasopressin as well as higher stock compensation expense account for the year-over-year increase. Excluding stock-based compensation and other non-cash and non-recurring items, third quarter 2019 SG&A expense was $13.4 million.

Net loss for the third quarter of 2019 was $2.4 million, or $0.17 per basic and diluted share, compared to net income of $14.0 million, or $0.94 per basic and $0.91 per diluted share, in the third quarter of 2018, due to the factors discussed above.

Adjusted non-GAAP net income for the third quarter of 2019 was $3.7 million, or $0.27 per basic and $0.26 per diluted share, compared to adjusted non-GAAP net income of $18.3 million or $1.22 per basic and $1.18 per diluted share in the third quarter of 2018. For a full reconciliation of adjusted non-GAAP net income to the most comparable GAAP financial measures, please see the tables at the end of this press release.

2019 Expense Guidance

R&D spend in 2019, on a non-GAAP basis, is expected to be $32.0-$36.0 million, as compared to $38.0 million in 2018.
SG&A spend in 2019, on a non-GAAP basis, is expected to be $51.0-$54.0 million, as compared to $43.0 million in 2018.
The guidance provided in this section represents forward-looking information, and actual results may vary. Please see the risks and assumptions referred to in the Forward-Looking Statements section of this press release.

Liquidity

As of September 30, 2019, the Company had $117.2 million in cash and cash equivalents plus $44.8 million in net accounts receivable, $34.4 million of which was due from Teva Pharmaceutical Industries Ltd. The Company had $40.0 million in outstanding debt. Therefore, at September 30, 2019, the Company had net cash plus receivables of $122.0 million.

Conference Call

As previously announced, Eagle management will host its third quarter 2019 conference call as follows:

Date

Tuesday, November 12, 2019

Time

8:30 A.M. EST

Toll free (U.S.)

866-342-8591

International

203-518-9713

Webcast (live and replay)

www.eagleus.com, under the "Investor + News" section

A replay of the conference call will be available for one week after the call’s completion by dialing 800-839-4577 (US) or 402-220-2682 (International) and entering conference call ID EGRXQ319. The webcast will be archived for 30 days at the aforementioned URL.