On August 6, 2019 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported its financial results for the second quarter ended June 30, 2019 (Press release, Curis, AUG 6, 2019, View Source [SID1234538187]).

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"Today, we are excited to announce initial data from our ongoing Phase 1 study of CA-4948 in patients with non-Hodgkin lymphoma, demonstrating anti-cancer activity for CA-4948 with tumor reduction observed in multiple patients across multiple dose levels," said James Dentzer, President and Chief Executive Officer of Curis. "We are especially pleased to see such encouraging results, as we are still in the middle of the dose optimization phase of the study. As a result of this positive readout, we are announcing today our decision to advance CA-4948 into the clinic in a new study of patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). We look to provide more detailed guidance on the timing of that study later this year."

"I am very pleased with the initial data we are seeing in our ongoing Phase 1 trial of CA-4948," said Robert Martell, Head of R&D of Curis. "As we increase dosing, we have observed continued evidence of pharmacodynamic target inhibition with dose-proportional increases in pharmacokinetic exposure. With the caveats of small cohort sizes and low initial dosage levels, we found patients experienced anti-tumor activity at multiple doses, including two of the patients at the 200mg BID dose level. And, importantly, all of these findings have been observed at tolerable dose levels. We look forward to reporting additional data in this ongoing study at an upcoming medical conference."

Separately, ongoing clinical studies of fimepinostat and CA-170 remain on track to provide initial data, for both studies, later this year.

Second Quarter 2019 and Recent Operational Highlights

Precision oncology, fimepinostat (HDAC/PI3K inhibitor):

Curis has initiated a Phase 1 study of fimepinostat (a MYC suppressor) with venetoclax (a BCL-2 inhibitor) combination regimen in diffuse large B-cell lymphoma (DLBCL), including patients with double-hit/double-expressor (DH/DE) lymphoma, or High-Grade B-Cell Lymphoma (HGBL). In preclinical models, fimepinostat administered in combination with venetoclax resulted in an enhanced benefit relative to each agent alone.
Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):

Today, Curis announced initial safety and efficacy data from its Phase 1 dose escalation study of CA-4948 in patients with non-Hodgkin lymphoma, including those with MYD88 alterations. As previously reported, the Phase 1 study of CA-4948 has advanced through five dose levels and is now being studied at exposures that resulted in anticancer efficacy in preclinical animal models. In the ongoing Phase 1 study, CA-4948 has demonstrated pharmacokinetic activity linearly-associated with dosing and clear signs of pharmacodynamic inhibition of the target. In addition, tumor burden reduction was observed in multiple patients across multiple dose levels with an acceptable safety profile. Treatment with CA-4948 at 200mg twice-daily (BID) is generally safe and well-tolerated, with no dose-limiting toxicities (DLTs) observed. The Company is currently enrolling patients in the study at 400mg BID and plans to continue dose escalation in the study to determine the optimal dose for clinical development.

Today, Curis announced that it will initiate a separate Phase 1 trial of CA-4948 in patients with AML and MDS. In June 2019, the Company highlighted a publication in Nature Cell Biology showing that a cancer-causing splicing variant of IRAK4 (IRAK4-L) is dominant in the majority of cases of AML and MDS. In addition, specific mutations of the U2AF1 splicing factor induce IRAK4-L, which has potential therapeutic targetability by CA-4948. The findings present the inhibition of IRAK4 as a potential treatment option for patients with myeloid malignancies expressing IRAK4-L and with U2AF1 mutations.
Immuno-oncology, CA-170 (VISTA / PDL1 antagonist; Aurigene collaboration):

Phase 1 study of CA-170 in mesothelioma patients (high VISTA expressors) is ongoing. The trial completed enrollment in May 2019.
Upcoming 2019 Milestones

Report update of initial safety and efficacy data from the Phase 1 dose escalation study of CA-4948 in patients with NHL at an upcoming medical conference.

Report initial safety data from the Phase 1 study of the combination of fimepinostat and venetoclax regimen in patients with R/R DLBCL, including patients with DH/DE lymphoma, or HGBL, in the second half of 2019.

Report initial efficacy data from the Phase 1 study of CA-170 in patients with mesothelioma in the second half of 2019.
Second Quarter 2019 Financial Results

Curis reported a net loss of $7.2 million, or $0.22 per share on both a basic and diluted basis for the second quarter of 2019, as compared to a net loss of $8.7 million, or $0.26 per share on both a basic and diluted basis for the same period in 2018. Curis reported a net loss of $17.1 million, or $0.52 per share, on both a basic and diluted basis for the six months ended June 30, 2019, as compared to a net loss of $19.4 million, or $0.59 per share on both a basic and diluted basis for the same period in 2018.

Revenues for the second quarter of 2019 were $2.1 million, as compared to $2.4 million for the same period in 2018. Revenues for the six months ended June 30, 2019 were $3.9 million, as compared to $4.8 million for the same period in 2018. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses were $8.2 million for the second quarter of 2019, as compared to $10.2 million for the same period in 2018. Operating expenses for the six months ended June 30, 2019 were $15.6 million, as compared to $22.6 million for the same period in 2018, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.1 million for both the second quarter of 2019 and 2018. Cost of royalty revenues for the six months ended June 30, 2019 were $0.2 million, as compared to $0.3 million for the same period in 2018.

Research and Development Expenses (R&D). R&D expenses were $5.6 million for the second quarter of 2019, as compared to $6.5 million for the same period in 2018. The decrease was primarily driven by lower employee related expenses partially offset by an overall increase in direct research and development expenses. R&D expenses were $9.7 million for the six months ended June 30, 2019 as compared to $14.7 million for the same period in 2018.

General and Administrative Expenses (G&A). G&A expenses were $2.5 million for the second quarter of 2019 as compared to $3.6 million for the same period in 2018. The decrease was primarily driven by lower personnel, legal services and stock-based compensation during the period. G&A expenses were $5.7 million for the six months ended June 30, 2019, as compared to $7.6 million for the same period in 2018.

Other Expenses. Net other expense for the second quarter 2019 was $1.1 million, as compared to $0.8 million for the same period in 2018. Net other expense for the second quarter 2019 primarily consisted of non-cash imputed interest expense related to future royalty payments, whereas in 2018 the expense related to interest accrued on Curis Royalty’s debt obligations. Other expense, net was $5.4 million and $1.6 million for the six months ended June 30, 2019 and 2018, respectively.

As of June 30, 2019, Curis’s cash, cash equivalents, marketable securities and investments totaled $35.3 million and there were approximately 33.2 million shares of common stock outstanding.

Conference Call Information

Curis management will host a conference call today, August 6, 2019, at 4:30 p.m. EDT, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412-317-5252 from other locations, shortly before 4:30 p.m. EDT. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.

On August 6, 2019 BioXcel Therapeutics, Inc. ("BTI" or "Company") (Nasdaq: BTAI), reported its quarterly results for the second quarter ended June 30, 2019 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, AUG 6, 2019, View Source [SID1234538204]). BTI is a clinical-stage biopharmaceutical development company utilizing novel artificial intelligence approaches to identify the next wave of medicines across neuroscience and immuno-oncology.

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Second Quarter 2019 and Recent Highlights

(BXCL501)-Neuroscience Program-

·BXCL501 adaptive Phase 1b, randomized, double blind, placebo-controlled, multi-center, U.S. trial reported positive topline data as a potential therapy for acute treatment of agitation in schizophrenia patients;

·BXCL501 met primary endpoint and demonstrated statistically significant and clinically meaningful rapid mean reduction in PEC (PANSS or the Positive and Negative Syndrome Scale, Excitatory Component) score at two hours compared to placebo following a single dose of 80 mcg (p=0.0152), 120 mcg (p=0.0003) and 180 mcg (p<0.0001);

· Results from secondary analyses showed statistically significant calming as measured by the ACES (Agitation-Calmness Evaluation Scale) at two hours compared to placebo

following a single dose of 80 mcg (p=0.0156), 120 mcg (P=0.0005) and 180 mcg (P<0.0001);

· Well tolerated with no serious or severe adverse events across the entire dose range;

· Trial results support the potential to advance the BXCL501 program to Phase 3 pivotal studies in agitated schizophrenia and bipolar patients, subject to a pre-Phase 3 meeting with the FDA;

·Phase 3 Pivotal trials are anticipated to enroll approximately 600 to 700 patients (300-350 each in schizophrenia and bipolar disorder), designed to measure reduction in PEC at two hours as the primary endpoint, as used in clinical trials of other approved agents. A data readout is expected in 1H 2020;

·BXCL501 adaptive Phase 1b trial in agitated Alzheimer’s disease/dementia patients is expected to begin in Q4 2019;

·Development plans for the acute treatment of agitation with BXCL501 in hyperactive delirium and opioid withdrawal are underway

(BXCL701)-Immuno-Oncology Program-

· Currently enrolling patients in the U.S. in the double combination of BXCL701 and Keytruda clinical trial for treatment emergent Neuroendocrine Prostate Cancer (tNEPC). Multiple patients have been treated in the safety and escalation portion of the trial which will be followed by a two-stage efficacy portion of the clinical program. A data read out is expected in 2H 2019;

· Clinical Trial Application (CTA) was accepted by the U.K. Medicines and Healthcare products Regulatory Agency ("MHRA") for the double combination trial of BXCL701 and Keytruda in tNEPC patients. Expected to activate clinical sites, subject to approval from local U.K. authorities. This approval is the first step in our plan to expand our clinical trials globally;

·FDA authorized the IND application for the triple combination of BXCL701, bempegaldesleukin (produced by Nektar Therapeutics, Inc., or Nektar) and BAVENCIO (avelumab, Merck KGaA, Darmstadt, Germany and Pfizer) in pancreatic cancer. The safety escalation portion of the trial is ongoing and will be followed by a two-stage efficacy portion. A data read-out is expected in 1H 2020;

· Pursuing a clinical proof of mechanism study with BXCL701 in pancreatic cancer patients to characterize immune cell infiltration and activation and the circulating cytokines elicited in order to validate it’s mechanism of action;

· Continuing to explore additional indications for BXCL701 with synergistic combinations

"We are excited by the clinical achievements we made during this quarter by advancing both our lead neuroscience program, BXCL501, and our immuno-oncology program, BXCL701," commented Vimal Mehta, President and Chief Executive Officer of BTI.

"In our BXCL501 program, we recently announced positive top-line data from our adaptive Phase 1b, randomized, double-blind, placebo-controlled, multi-center, U.S. trial demonstrating statistically significant reductions in the PEC score at two hours compared to placebo for multiple dosages and displayed rapid and durable calming effect without excessive sedation for such dosages. The drug was also well tolerated across all doses tested. We are pleased by these results which reflect significant progress in developing a non-invasive, easy to administer therapy, and which provide evidence supporting BXCL501’s potential as a rapid-onset treatment for acute agitation. Based on these results, we intend to have discussions with the FDA to determine the path forward for the BXCL501 program. We anticipate moving into the pivotal Phase 3 trial in the fourth quarter of 2019 and expect a data readout during the first half of 2020. Additionally, we are also planning to begin a Phase 1b trial in agitated Alzheimer’s disease/dementia patients that we expect to initiate in the fourth quarter of 2019. With all these positive developments, we remain confident in our strategic plan to submit our first NDA for BXCL501 during the second half of 2020."

Dr. Mehta added, "We are also committed to driving progress in our immuno-oncology program for BXCL701 with two ongoing Phase 1b/2 trials. We are evaluating the combination of BXCL701 and Keytruda in tNEPC, an aggressive form of prostate cancer with limited treatment options. We are enrolling patients in this Phase 1b/2 study of BXCL701 combined with Keytruda in the United States, and multiple patients have been treated in the safety and escalation portion of the trial. A data readout for this study is anticipated in the second half of 2019. Further, the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) accepted our clinical trial authorization to evaluate BXCL701 plus Keytruda in tNEPC which allows us to expand the development of this double combination into other geographies. Following the FDA authorization of our IND application for the combination of BXCL701, bempegaldesleukin and BAVENCIO as a second line therapy for pancreatic cancer, we initiated a clinical study for which data readouts are expected in the first half of 2020. Our collaboration with Nektar Therapeutics, Merck KGaA, Darmstadt, Germany and Pfizer enables us to pursue our mutual goal of advancing the triple combination of immuno-oncology agents in pancreatic cancer to bring an effective therapeutic option in this large, underserved indication."

"With the achievement of these value driving milestones in both of our lead programs, we are extremely pleased with our performance during the second quarter. We also raised our visibility in the financial community through BTI’s addition to the Russell 2000 and 3000 Indexes. As we continue to effectively manage our cash position, we believe it will be sufficient

to fund key milestones and operations to mid-2020. We remain confident in our strategy and believe we are positioned for continued growth."

Second Quarter 2019 Financial Results

BTI reported a net loss of $8.5 million for the second quarter of 2019, compared to a net loss of $3.0 million for the same period in 2018.

Research and development expenses were $6.5 million for the second quarter of 2019, as compared to $1.8 million for the same period in 2018. The increase was primarily due to an expansion of research and development activities, including increased personnel costs, clinical trials expenses, manufacturing costs, and professional fees, associated with BTI’s two lead product candidates.

General and administrative expenses were $2.1 million for the second quarter of 2019, as compared to $1.5 million for the same period in 2018. The increase was primarily due to additional payroll and payroll-related expenses, professional fees and costs associated with operating as a public company.

These results include approximately $1.0 million in non-cash stock based compensation.

As of June 30, 2019, cash and cash equivalents totaled approximately $30.0 million. BTI is well positioned to execute on key milestones with sufficient cash to fund operations through mid-2020.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call please dial (800) 239-9838 (domestic) and (323) 994-2093 (international) and provide the passcode 2198753. A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The archived webcast will be available through September 6, 2019.

About BXCL501:

BXCL501 is a potential first-in-class, proprietary sublingual thin film of dexmedetomidine, a selective alpha-2a receptor agonist for the treatment of acute agitation. BTI believes that BXCL501 directly targets a causal agitation mechanism and the Company has observed anti-agitation effects in multiple clinical studies across multiple neuropsychiatric indications. BXCL501 is currently being developed for agitation associated with schizophrenia and bipolar disorders followed by Alzheimer’s/dementia.

About BXCL701:

BXCL701 is an investigational orally-available systemic innate immunity activator with dual mechanisms of action. It has demonstrated single agent activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients. Designed to stimulate both the innate and acquired immune systems, BXCL701 inhibits dipeptidyl peptidase (DPP) 8/9 and blocks immune evasion by targeting Fibroblast Activation Protein (FAP). BXCL701, is currently being developed for treatment of a rare form of prostate cancer and for pancreatic cancer in combination with other immuno-oncology agents

On August 6, 2019 Quanterix Corporation (NASDAQ: QTRX), a company digitizing biomarker analysis to advance the science of precision health, reported financial results for the three and six months ended June 30, 2019 (Press release, Quanterix, AUG 6, 2019, View Source [SID1234538220]).

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"We are pleased with our execution and yet another quarter of record results, with strong revenue growth and gross margins, and material progress in our quest to lead today’s biomarker revolution," said Kevin Hrusovsky, Chief Executive Officer, President and Chairman, Quanterix. "Our first half and Q2 growth continues to outpace expectations based on stronger adoption momentum and a small level of favorable timing in our consumables business. We believe our growth trajectory in all segments of our business continues to advance productively opposite our longer-term growth target propelled by recent product launches into oncology, expansion of our assay menu, and strong progress in our neurodegeneration portfolio and associated pharma services supporting drug trials and approvals. In addition, we are enthusiastic about our strategic acquisition of UmanDiagnostics, which further accelerates many opportunities for neurofilament light (Nf-L) as a transformative biomarker for neurodegenerative diseases and protects our supply of this important Nf-L antibody. We remain confident in our growth prospects and our ability to pioneer disruption in healthcare."

Second Quarter 2019 Financial Highlights

Key financial results for the second quarter of 2019 are shown below:

Q2 revenue of $13.5M versus prior year Q2 of $8.6M, an increase of 57%.
Q2 product revenue was $8.8M versus prior year Q2 of $5.2M, an increase of 69%.
Q2 service and other revenue totaled $4.8M versus prior year Q2 of $3.2M, an increase of 50%.
Q2 gross margin at 51.2% versus prior year Q2 of 46.0%, an increase of 520 bps
1H 2019 Financial Highlights

Key financial results for the first half of 2019 are shown below:

1H revenue of $25.9M versus prior year 1H of $16.2M, an increase of 60%.
1H product revenue was $18.3M versus prior year 1H of $9.9M, an increase of 84%.
1H service and other revenue totaled $7.5M versus prior year 1H of $5.7M, an increase of 33%.
1H gross margin at 50.0% versus prior year 1H of 44.2%, an increase of 580 bps
Second Quarter 2019 Business Highlights

Entered into definitive agreement to acquire UmanDiagnostics AB, the world’s leading Nf-L antibody supplier, with the acquisition completed after quarter end. The acquisition allows Quanterix to supply researchers globally with the "best-in-class" Simoa Nf-L assays, while continuing to innovate new digital biomarkers to advance the field of research in diagnostics for neurological disorders. The deal also positions Quanterix to capitalize on the growth fueled by the momentum in Nf-L as a tremendously promising brain biomarker.
Successfully raised nearly $50 million through an at-the-market equity (ATM) facility, attracting marquee investors, and further enhancing Quanterix’ liquidity and potential for value creation.
Announced launch of the Simoa HD-X Analyzer, Quanterix’ new, fully automated Simoa bead-based immunoassay instrument model, with commercial shipments beginning in Q4. The HD-X replaces the HD-1 and has been designed to deliver significant productivity improvements, greater user flexibility, unparalleled sensitivity, and best-in-class assay performance across a broad assay menu to empower biomarker research and accelerate drug development.
Completed the move to its expanded headquarters in Billerica, Mass. The 92,000 square-foot state-of-the-art facility allows the Company to unite its CLIA-certified laboratory with its expanding workforce to accelerate entry into pharmaceutical drug services and further increase its impact on the transformation of precision healthcare.
Took part in major industry presence at the Alzheimer’s Association International Conference (AAIC), the largest and most influential international meeting dedicated to dementia science, where Quanterix presented to partners and customers at a standing-room-only dinner and hosted a panel discussion featuring several of the world’s foremost researchers in Alzheimer’s Disease.
Received EY’s Entrepreneur Of The Year 2019 Award in New England recognizing Hrusovsky’s achievements in healthcare technology innovation, Quanterix’ outstanding financial performance, his commitment to powering precision health (PPH) and the founding of the PPH foundation.
Continued to strengthen its leadership team with the appointment of Amol Chaubal as CFO, John Fry to General Counsel and several key commercial leaders.
Powered more than 85 percent of the Nf-L biomarker research presented at the American Academy of Neurology’s Annual Meeting. The 36 abstracts used Quanterix’ Simoa technology to validate Nf-L as a potential diagnostic and prognostic biomarker for neurodegeneration.
Hrusovsky was invited to speak at BIO International Convention, a leading healthcare event featuring world leaders in biotech and pharma, on the company’s vision for continuing to pioneer disruption in healthcare with digital biomarkers.
Conference Call

In conjunction with this announcement, Quanterix Corporation will host a conference call on Aug. 6, 2019, at 10 a.m., EDT to discuss the Company’s financial results and business outlook. To access this call, dial (833) 686-9351 for domestic callers, or (612) 979-9890 for international callers. Please reference the following conference ID: 7499384.

A live webcast will be accessible on the Investors section of Quanterix’ website: View Source The webcast will be available on the Company’s website for one year following completion of the call.

On August 6, 2019 Lixte Biotechnology Holdings, Inc. (OTCQB: LIXT) reported that it signed a clinical trial agreement with the Spanish Sarcoma Group (Grupo Español de Investigación en Sarcomas, GEIS) to support a Phase 1b/randomized Phase 2 study of doxorubicin, the global standard for initial treatment of advanced soft tissue sarcomas (ASTS), versus doxorubicin plus LB-100 (Press release, Lixte Biotechnology, AUG 6, 2019, View Source [SID1234538246]).

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Dr. John S. Kovach, CEO of Lixte, said, "We are pleased to support this investigator-initiated trial proposed by GEIS, a leader for many years in seeking improved therapies for ASTS. GEIS has a network of referral centers in Spain and across Europe that has an impressive track record of efficiently conducting innovative studies in ASTS. We believe GEIS and their EU collaborators are an excellent team to evaluate the potential benefit of adding Lixte’s non-cytotoxic inhibitor of DNA damage repair to standard treatment for this challenging disease. The goal is to enter the first patient in the last quarter of this year and to complete enrollment of approximately 170 patients over two years."

Dr. Kovach continued, "Doxorubicin alone has been the mainstay of first line treatment of ASTS for over 40 years with little therapeutic gain from adding cytotoxic compounds to or substituting other cytotoxic compounds for doxorubicin. In animal models, LB-100, an inhibitor of protein phosphatase2A (PP2A), consistently enhances the antitumor activity of doxorubicin without apparent increases in toxicity and, in a Phase 1 clinical trial, LB-100 alone was associated with stabilization of an advanced chondrosarcoma and fibrosarcoma for 6 and 9 months, respectively, without toxicity. LB-100 potentiates the effectiveness of agents like doxorubicin by inhibiting multiple steps required to repair chemotherapy induced DNA damage. We are excited to learn if the combination of LB-100 and doxorubicin will finally advance the effectiveness of ASTS therapy."

Dr. Javier Martin-Broto, Coordinating Investigator of the trial and medical oncologist at Virgen del Rocío University Hospital (Seville) commented, "Although there has been an increase in overall survival in advanced sarcoma in recent years, this gain has not been accompanied by advances in first line therapy. Anthracyclines, and specifically doxorubicin, is still the standard initial treatment. The growing list of negative phase III trials indicates to us that sarcoma therapy is in crisis. It is true that sarcoma encompasses more than 60 different subtypes and, for some of them, substantial advances have emerged. But it is also true that the most frequent sarcoma subtypes desperately need a turning point. One promising topic of research is the combination of doxorubicin with drugs that are able to impair the mechanisms of DNA repair. LB-100 has demonstrated synergistic action in in vivo preclinical mesenchymal tumors. GEIS will lead a European initiative to conduct a phase I/randomized II trial exploring the combination of doxorubicin plus LB-100 in first line of advanced soft tissue sarcomas".

On August 6, 2019 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer, autoimmune diseases, asthma and allergic diseases, reported financial results for the second quarter ended June 30, 2019 and provided a review of recent business and clinical highlights (Press release, Xencor, AUG 6, 2019, View Source [SID1234538188]).

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"Over the last two years, Xencor has initiated six Phase 1 clinical studies evaluating XmAb bispecific antibodies—both T cell engagers and tumor microenvironment (TME) activators—in patients with many types of advanced cancers, and we have also entered into strategic collaborations to advance select programs with our partners," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "We anticipate several initial data presentations from these programs in the second half of 2019 and first half of 2020, and our strong financial position supports our broad pipeline and research into novel antibodies and cytokines engineered with XmAb bispecific technologies."

Dr. Dahiyat added, "In the second quarter, we reopened the Phase 1 study of XmAb14045 to enrollment, and patients have since begun treatment under the amended protocol. We also advanced our second and third TME activating bispecific antibodies into Phase 1 clinical studies. TME bispecific antibodies are designed with tuned dual checkpoint or checkpoint-agonist mechanisms to generate anti-tumor immune responses in a targeted manner. Finally, we continued to strengthen our senior leadership with appointments in business development and regulatory affairs."

Recent Business and Clinical Highlights and Anticipated Upcoming Milestones

CD3 Bispecific Antibodies: Xencor’s initial bispecific antibody programs are tumor-targeted antibodies that contain both a tumor antigen binding domain and a cytotoxic T-cell binding domain (CD3). These bispecific antibodies activate T cells for highly potent and targeted killing of malignant cells.

XmAb14045 (CD123 x CD3): Clinical sites participating in the Phase 1 study in patients with relapsed/refectory acute myeloid leukemia have resumed enrollment, and in early July the first patient was dosed under the study’s amended protocol. Amendments to the protocol included guidance on the monitoring and clinical management of cytokine release syndrome.
XmAb13676 (CD20 x CD3): A Phase 1 dose-escalation study began dosing patients with B-cell malignancies in February 2017. Enrollment in dose-escalation cohorts is ongoing, and initial data are expected in the fourth quarter of 2019.
XmAb18087 (SSTR2 x CD3): A Phase 1 dose-escalation study began dosing patients with neuroendocrine tumors or gastrointestinal stromal tumors in February 2018. Enrollment in dose-escalation cohorts is ongoing, and initial data are expected in the first half of 2020.
Tumor Microenvironment (TME) Activating Bispecific Antibodies: Xencor’s bispecific pipeline includes a suite of TME activators that engage multiple, different targets, such as T-cell checkpoint or agonist receptors. Xencor’s TME activators are designed to promote tumor-selective T-cell activation.

XmAb20717 (PD-1 x CTLA-4):A Phase 1 dose-escalation study in patients with advanced solid tumors began dosing patients in July 2018. Enrollment in dose-escalation cohorts is ongoing, and initial data are expected in the first half of 2020.
XmAb22841 (CTLA-4 x LAG-3): In May 2019, the first patient was dosed in a Phase 1 study evaluating XmAb22841 as a monotherapy and in combination with pembrolizumab in patients with select advanced solid tumors.
XmAb23104 (PD-1 x ICOS):In May 2019, the first patient was dosed in a Phase 1 study in patients with select advanced solid tumors.
Cytokines: Xencor uses its bispecific Fc domain and Xtend technology to engineer cytokines, which are immune signaling proteins, that have potency tuned to improve therapeutic index and have longer half-life.

XmAb24306 (IL15/IL15Rα-Fc fusion protein): The Company will support Genentech’s efforts to submit an IND application for this candidate, which is anticipated in the second half of 2019.
Partnered XmAb Programs: Xencor has eight licensing partnerships for XmAb technology. The most advanced program where the Company has licensed its technology is Alexion’s Ultomiris, which has received marketing authorizations for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) in the U.S. (December 2018), Japan (June 2019) and Europe (July 2019).

Corporate: In June 2019, Xencor announced the planned October 31, 2019 retirement of Paul Foster, M.D., Chief Medical Officer. As the Company searches for Dr. Foster’s successor, Xencor strengthened its senior management team in the second quarter with the appointments of Jeremy Grunstein, Ph.D., as vice president, business development, and Kirk Rosemark, as vice president, regulatory affairs and quality assurance.

Ultomiris is a registered trademark of Alexion Pharmaceuticals, Inc.

Second Quarter Ended June 30, 2019 Financial Results

Cash, cash equivalents and marketable securities totaled $626.1 million as of June 30, 2019, compared to $530.5 million at December 31, 2018. The increase reflects upfront proceeds of $135 million received in the first half of 2019 from the Genentech and Astellas collaborations, offset by cash used to fund operating activities in the first six months of 2019.

Total revenue for the second quarter ended June 30, 2019 was $19.5 million, which was primarily revenue recognized under the Astellas collaboration and a milestone and royalties earned from Alexion. Total revenue for the six months ended June 30, 2019 was $131.4 million and includes revenue earned from the Genentech and Astellas collaborations and the milestone and royalty revenue from Alexion. No revenue was reported for the same periods in 2018.

Research and development expenses for the second quarter of 2019 were $33.3 million, compared to $23.3 million for the same period in 2018. Total research and development expenses for the six months ended June 30, 2019 were $61.5 million, compared to $49.4 million for the same period in 2018. The increased research and development spending for the three and six months ended June 30, 2019 reflects increased stock-based compensation expense and additional spending on Xencor’s CD3 bispecific antibody and cytokine development candidates and technologies.

General and administrative expenses for the second quarter of 2019 were $5.8 million, compared to $5.0 million for the same period in 2018. Total general and administrative expenses for the six months ended June 30, 2019 were $11.3 million, compared to $9.5 million for the same period in 2018. The increased general and administrative spending for the three and six months ended June 30, 2019 reflects additional spending on intellectual property including patents and licensing and additional expenses related to personnel and professional services.

Non-cash, stock-based compensation expense for the six months ended June 30, 2019 was $15.2 million, compared to $9.4 million for the same period in 2018.

Net loss for the second quarter ended June 30, 2019 was $16.0 million, or $(0.28) on a fully diluted per share basis, compared to a net loss of $25.9 million, or $(0.46) on a fully diluted per share basis, for the same period in 2018. For the six months ended June 30, 2019, net income was $64.0 million, or $1.10 on a fully diluted per share basis, compared to a net loss of $55.4 million, or $(1.07) on a fully diluted per share basis, for the same period in 2018. The lower net loss reported for the three months ended June 30, 2019 over the same period in 2018 is primarily due to revenue from our Astellas and Alexion collaborations in 2019. The net income reported for the six months ended June 30, 2019 over the net loss reported for the same period in 2018 is primarily due to revenue recognized from our Genentech collaboration.

The total shares outstanding were 56,529,398 as of June 30, 2019, compared to 55,821,310 as of June 30, 2018.

Financial Guidance

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations beyond 2024. Xencor expects to end 2019 with between $575 million and $600 million in cash, cash equivalents and marketable securities.

Conference Call and Webcast

Xencor will host a conference call today at 4:30 p.m. ET (1:30 p.m. PT) to discuss these second quarter 2019 financial results and provide a corporate update. The live call may be accessed by dialing (877) 359-9508 for domestic callers or (224) 357-2393 for international callers and referencing conference ID number 1597118. A live webcast of the conference call will be available under "Events & Presentations" in the Investors section of the Company’s website located at www.xencor.com. The webcast will be archived on the company website for 30 days.