On February 27, 2018 Sierra Oncology, Inc. (Nasdaq: SRRA), a clinical stage drug development company focused on advancing next generation DNA Damage Response (DDR) therapeutics for the treatment of patients with cancer, reported the signing of a clinical supply agreement with Janssen Research & Development, LLC. (Janssen) for access to TESARO’s ZEJULA (niraparib), an orally administered poly (ADP-ribose) polymerase (PARP) inhibitor undergoing clinical development by Janssen for patients with prostate cancer (Press release, Sierra Oncology, FEB 27, 2018, View Source [SID1234524261]). Sierra intends to evaluate its Chk1 inhibitor, SRA737, in combination with niraparib in patients with metastatic castration-resistant prostate cancer (mCRPC). The scientific rationale for the combination was supported by translational research conducted at The Institute of Cancer Research, London.

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Professor Johann de Bono, Regius Professor of Cancer Research at The Institute of Cancer Research, London, and Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust, will be the Chief Investigator of the new study. He noted: "I look forward to supporting Sierra’s investigation of this combination. Research has demonstrated the potential benefit of PARP inhibitor therapy in prostate cancer patients with defects in HRR, and clinical trials are under way evaluating niraparib in these patients. However, the combination of Chk1 inhibition with PARP inhibition potentially may expand the application of niraparib to patients with HRR proficient tumors or re-sensitize patients who have developed PARP inhibitor resistance."

"Preclinical studies have demonstrated synergy between PARP inhibitors and SRA737, including in contexts where PARP inhibitors have minimal activity such as HRR proficient and PARP inhibitor resistant settings," added Dr. Nick Glover, President and CEO of Sierra Oncology. "PARP inhibitors impede the repair of single-strand DNA breaks, resulting in stalled DNA replication forks and double strand breaks that make the cell highly reliant on HRR, which is regulated by Chk1. The combined inhibition of both pathways is the basis for Sierra’s drug combination strategy of SRA737 with niraparib."

"This supply agreement with Janssen is significant strategically in the continued advancement of SRA737 as it enables the exploration of a novel and independent development path for our Chk1 inhibitor, supported by both a promising scientific and a compelling commercial rationale," added Dr. Angie You, Chief Business & Strategy Officer and Head of Commercial at Sierra.

Sierra plans to conduct an open-label, multicenter Phase 1b/2 dose-ranging study to assess the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of SRA737 in combination with niraparib in patients with mCRPC. The study is intended to determine the maximum tolerated dose and schedule of SRA737 in combination with niraparib and to propose a recommended Phase 2 dose (RP2D) and schedule of the combination. Data will be analyzed to examine specific hypotheses regarding the relationship between clinical response and alterations in genes regulating cell cycle progression and DNA damage response.

Janssen will provide Sierra with niraparib, while Sierra will conduct and control the study, which is anticipated to commence in the fourth quarter of 2018. Preclinical studies evaluating the combination of SRA737 and niraparib are ongoing.

About Sierra’s Chk1 inhibitor, SRA737
SRA737, is a potent, highly selective, orally bioavailable small molecule inhibitor of Checkpoint kinase 1 (Chk1), a key regulator of cell cycle progression and the DDR Replication Stress response. SRA737 is currently being investigated in two Phase 1/2 clinical trials in patients with advanced cancer: SRA737-01, a monotherapy study evaluating SRA737 in patients with tumors identified to have genetic aberrations hypothesized to confer sensitivity to Chk1 inhibition via synthetic lethality; and SRA737-02, a drug combination study evaluating SRA737 potentiated by low-dose gemcitabine. Sierra is also preparing for potential clinical studies of SRA737 in combination with other agents where there is a strong biological rationale for synergy with Chk1 inhibition, such as immune oncology therapeutics and other DDR inhibitors including PARP inhibitors.

About niraparib
Janssen Biotech, Inc. maintains a worldwide collaboration, license and supply agreement with TESARO, Inc., for exclusive rights to the investigational compound niraparib in prostate cancer for all geographies except Japan. Janssen is conducting a Phase 2 study (NCT02854436) of niraparib in patients with mCRPC who have DNA-repair anomalies and a Phase 1 study (NCT02924766) of niraparib in combination with an androgen receptor-targeted therapy in mCRPC.

Niraparib (ZEJULA) was approved by the Food and Drug Administration (FDA) on March 27, 2017 and is marketed by TESARO in the United States and Europe. ZEJULA is an oral, once-daily poly (ADP-ribose) polymerase (PARP) 1/2 inhibitor that is indicated in the U.S. for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. The National Comprehensive Cancer Network (NCCN) added ZEJULA to the NCCN Clinical Practice Guidelines in Oncology Ovarian Cancer version 1.2017—April 12, 2017—as maintenance therapy for patients with platinum-sensitive disease who are in partial or complete response after completion of two or more lines of platinum-based chemotherapy. In preclinical studies, ZEJULA concentrates in the tumor relative to plasma, delivering greater than 90% durable inhibition of PARP 1/2 and a persistent antitumor effect.

On February 27, 2018 Seattle Genetics, Inc. (NASDAQ:SGEN) reported that management will present at the Cowen and Company 38th Annual Health Care Conference on Tuesday, March 13, 2018 at 9:20 a.m. Eastern Time (Press release, Seattle Genetics, FEB 27, 2018, View Source;p=RssLanding&cat=news&id=2334859 [SID1234524248]). The presentation will be webcast live and available for replay from Seattle Genetics’ website at www.seattlegenetics.com in the Investors section.

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On February 27, 2018 Actinium Pharmaceuticals, Inc. (NYSE American:ATNM) ("Actinium" or "the Company"), reported that the Company has successfully activated sixteen clinical trial sites in the pivotal Phase 3 SIERRA (Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia) trial (Press release, Actinium Pharmaceuticals, FEB 27, 2018, View Source [SID1234524189]). The SIERRA trial is planned to enroll 150 patients with relapsed or refractory acute myeloid leukemia (AML) who are age 55 and above and will compare Iomab-B and a BMT to physician’s choice of salvage chemotherapy. The primary endpoint is durable complete remission (dCR) of at least six months. Iomab-B is intended to provide safer myeloablation of the bone marrow prior to a bone marrow transplant, thus providing a potentially curative treatment option for this patient population and for patients with other leukemias, lymphomas, myelomas and other blood disorders.

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With Stony Brook, New York-based Stony Book University, the sixteen clinical trial sites in the Phase 3 SIERRA trial represent over one-third of bone marrow transplant volume in the U.S., which bodes well for reaching the 150-patient enrollment goal. The following medical institutions are clinical trial sites in the Iomab-B Phase 3 clinical trials:

Center Location
MD Anderson Cancer Center Houston, Texas
Memorial Sloan Kettering Cancer Center New York, New York
Mayo Clinic Rochester, Minnesota
Mayo Clinic Jacksonville, Florida
Washington University School of Medicine Saint Louis, Missouri
Yale Cancer Center New Haven, Connecticut
Baylor Charles A. Sammons Cancer Center Dallas, Texas
The University of Kansas Cancer Center Westwood, Kansas
Roswell Park Cancer Institute Buffalo, New York
University Hospitals Cleveland Medical Center Cleveland, Ohio
The Ohio State University Comprehensive Cancer Center Columbus, Ohio
Penn State Hershey Cancer Institute Hershey, Pennsylvania
Loyola University Medical Center Maywood, Illinois
Banner MD Anderson Cancer Center Gilbert, Arizona
Fred Hutchinson Cancer Research Center Seattle, Washington
Stony Book University Stony Brook, New York
Dr. Mark Berger, Actinium’s Chief Medical Officer said, "We are optimistic that working with these renowned scientific institutions will move Iomab-B closer to realization as an accepted treatment to improve the therapy and prospects of bone marrow transplant patients."

Actinium also announced that it expects to provide updates on the Iomab-B SIERRA trial in line with previously stated objectives for 2018 and 2019. The SIERRA trial will have three safety analyses by an independent Data Monitoring Committee when 25%, 50% and 75% patient enrollment has been reached. Also, two ad-hoc efficacy analyses may be requested by Actinium after 70 and/or 110 patients have engrafted and given enough time to achieve the primary endpoint of durable complete remission at six months post-treatment.

Sandesh Seth, Actinium’s Chairman and CEO said, "Participation of these leading U.S. transplant centers are in the SIERRA trial reflects strongly on the prospects for our leading drug candidate, Iomab-b. We expect to provide several enrollment and Data Monitoring Safety Board related updates during 2018 and topline results next year and believe we are making solid progress toward meeting these goals with the addition of additional sites and participation of these prestigious institutions."

About Iomab-B

Iomab-B is Actinium’s lead product candidate that is currently being studied in a 150-patient, multicenter pivotal Phase 3 clinical trial in patients with relapsed or refractory acute myeloid leukemia who are age 55 and above. Upon approval, Iomab-B is intended to prepare and condition patients for a bone marrow transplant, also referred to as a hematopoietic stem cell transplant, which is often considered the only potential cure for patients with certain blood-borne cancers and blood disorders. Iomab-B targets cells that express CD45, a pan-leukocytic antigen widely expressed on white blood cells with the monoclonal antibody, BC8, labeled with the radioisotope, iodine-131. By carrying iodine-131 directly to the bone marrow in a targeted manner, Actinium believes Iomab-B will avoid the side effects of radiation on most healthy tissues while effectively killing the patient’s cancer and marrow cells. In a Phase 2 clinical study in 68 patients with advanced AML or high-risk myelodysplastic syndrome (MDS) age 50 and older, Iomab-B produced complete remissions in 100% of patients and patients experienced transplant engraftment at day 28. Iomab-B was developed at the Fred Hutchinson Cancer Research Center where it has been studied in almost 300 patients in a number of blood cancer indications, including acute myeloid leukemia (AML), chronic myeloid leukemia (CML), acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), Hodgkin’s disease (HD), Non-Hodgkin lymphomas (NHL) and multiple myeloma (MM). Iomab-B has been granted Orphan Drug Designation for relapsed or refractory AML in patients 55 and above by the U.S. Food and Drug Administration and the European Medicines Agency.

On February 27, 2018 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of autoimmune disease, asthma and allergic diseases, and cancer, reported financial results for the fourth quarter and full year ended December 31, 2017 and provided a review of 2017 and recent business and clinical highlights (Press release, Xencor, FEB 27, 2018, View Source [SID1234524270]).

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"Our 2017 accomplishments, including the announcement of final results from our Phase 2 trial of XmAb5871 in IgG4-RD and the expansion of our bispecific antibody pipeline in oncology, demonstrate the potential of our XmAb antibody engineering technology to deliver new drug candidates for patients with a range of severe or life-threatening diseases," said Bassil Dahiyat, Ph.D., president and chief executive officer of Xencor. "In 2018, we expect continued progress from our wholly-owned and partnered pipeline, which now includes 11 clinical-stage antibody programs. Specifically, we look forward to topline results from our Phase 2 trial of XmAb5871 in SLE and initial data from our Phase 1 trial of bispecific antibody XmAb14045 in AML, and to Phase 3 results from our partner Alexion’s trial of ALXN1210.

"We are also committed to advancing and broadening our clinical-stage efforts. We recently initiated a Phase 1 trial for XmAb18087, our first bispecific antibody targeting solid tumors. Later this year, we plan to initiate a Phase 3 trial of XmAb5871 in IgG4-RD and a Phase 1 trial of XmAb20717, our lead TME activator, while filing investigational new drug (IND) applications for two additional bispecific TME activators. We are also expanding our TME pipeline with XmAb24306, an IL-15/IL-15Ra-Fc candidate that has tuned CD122 activation and is engineered for longer half-life, and for which we expect to file an IND in 2019."

Recent Business Highlights and Upcoming Clinical Plans

XmAb5871: XmAb5871 is a first-in-class monoclonal antibody that targets CD19 with its variable domain, and uses Xencor’s XmAb immune inhibitor Fc domain to target FcyRIIb, a receptor that inhibits B-cell function. Xencor presented final data from a Phase 2 trial in IgG4-RD in November 2017, in which all 12 patients who completed the study achieved the primary endpoint of at least a two-point reduction in the IgG4-RD Responder Index and eight patients achieved disease remission. Xencor completed enrollment in a Phase 2 trial in SLE in December 2017.

Initiation of Phase 3 trial in IgG4-RD expected in 2H18. Following a Type B End of Phase 2 meeting with the U.S. Food and Drug Administration (FDA), Xencor expects this Phase 3 trial to be a randomized, placebo-controlled, double-blinded study, evaluating the addition of XmAb5871 to standard-of-care in approximately 200 to 250 patients with IgG4-RD.
Engagement with the European Medicines Agency to discuss a path forward for Phase 3 development in IgG4-RD expected in early 2018.
Topline data from Phase 2 trial in SLE expected in 4Q18.

Bispecific Oncology Pipeline: 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. Their XmAb Fc domains confer long circulating half-lives, stability and ease of manufacture.

Initial data from Phase 1 study of XmAb14045 for the treatment of AML and other CD123-expressing hematologic malignancies expected in 2018, pending alignment on timing with Novartis.
Initial data from Phase 1 study of XmAb13676 for the treatment of B-cell malignancies expected in 2018, pending alignment on timing with Novartis.
Initial data from Phase 1 study of XmAb18087 for the treatment of neuroendocrine tumors (NET) and gastrointestinal stromal tumors (GIST) expected in 2019.

In February 2018, Xencor announced that it has dosed the first patient in its Phase 1 dose-escalation study of XmAb18087, targeting somatostatin receptor 2 and CD3 (SSTR2 x CD3). The trial is a multiple ascending dose study to determine the safety and tolerability, pharmacokinetics and immunogenicity, and preliminary anti-tumor activity of weekly intravenous administration of XmAb18087 and to determine the maximally tolerated dose and regimen in patients with advanced NET or GIST.

Xencor is also expanding its bispecific pipeline to include a suite of tumor microenvironment activators that engage multiple targets, such as T-cell checkpoints or agonists, with three IND applications scheduled to be filed over the next 12 months:

Initiation of Phase 1 trial evaluating XmAb20717, a PD-1 x CTLA-4 dual checkpoint inhibitor for the treatment of multiple oncology indications, expected in 2018.
IND filing for XmAb23104, a PD-1 x ICOS bispecific antibody for the treatment of multiple oncology indications, expected in 2018 and initiation of Phase 1 trial expected in 2019.
IND filing for XmAb22841, a CTLA-4 x LAG-3 dual checkpoint inhibitor for the treatment of multiple oncology indications, expected in 2018 and initiation of Phase 1 trial expected in 2019.
IND filing for XmAb24306, an IL-15/IL-15Ra-Fc bispecific antibody for the treatment of multiple oncology indications, expected in 2019.

Today, Xencor announces XmAb24306 as an IL-15/IL-15Ra-Fc candidate for the treatment of multiple oncology indications. XmAb24306 is designed to create sustained T-cell expansion via modulated CD122 activation and an XmAb bispecific Fc domain. IL-15/IL-15Ra naturally targets CD122 without targeting CD25, and Xencor uses its XmAb Fc scaffold to create a stable ligand-receptor complex. Xencor plans to present detailed preclinical data for XmAb24306 at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2018.

At the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2017 Annual Meeting in November 2017, Xencor presented preclinical data supporting the development of XmAb20717 and XmAb23104 for the treatment of human malignances. Both antibodies are selective for their target pairs and show superior T-cell activation compared to anti-PD-1 antibodies alone, and are well tolerated in cynomolgus monkeys with antibody-like pharmacokinetics. XmAb22841 is also active in vivo, and combines with anti-PD1 antibodies to achieve highly active triple checkpoint blockade.

XmAb7195: XmAb7195 is a first-in-class monoclonal antibody that targets IgE with its variable domain and uses Xencor’s XmAb immune inhibitor Fc domain to target FcyRIIb, resulting in three distinct mechanisms of action for reducing IgE. Data from Xencor’s Phase 1b study of subcutaneously-administered XmAb7195 were announced in November 2017 and showed potent IgE reduction with improved tolerability. Xencor is currently seeking a development partner for XmAb7195.

Partnered XmAb Programs: Eight pharmaceutical companies and the National Institutes of Health are advancing novel drug candidates either discovered at Xencor or that rely on Xencor’s proprietary XmAb technology. Six such programs are currently undergoing clinical testing, including two in Phase 3 studies.

Initial data from Alexion’s Phase 3 trial comparing intravenously-administered ALXN1210 to Soliris in complement inhibitor treatment-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) and from Alexion’s Phase 3 PNH Switch study of intravenously-administered ALXN1210 compared to patients currently treated with Soliris are expected in 2Q18. ALXN1210 uses Xencor’s XmAb Xtend technology.
MorphoSys received Breakthrough Therapy designation for XmAb5574/MOR208 in relapsed and refractory diffuse large B-cell lymphoma (r/r DLBCL) in combination with lenalidomide in November 2017 and is currently running a Phase 2 trial for that combination, in addition to a Phase 3 trial in r/r DLBCL in combination with bendamustine.
In December 2017, Amgen submitted an IND application for AMG 424, a novel humanized T cell-recruiting bispecific antibody targeting CD38 and CD3, which uses Xencor’s Bispecific XmAb technology. Pursuant to Xencor’sSeptember 2015 licensing agreement with Amgen, this IND filing triggered a milestone payment to Xencor of $10.0 million.

Corporate:

In December 2017, Xencor announced the appointment of Richard Ranieri to its Board of Directors. Mr. Ranieri is currently Executive Vice President of Human Resources at BioMarin.

Fourth Quarter and Full Year Ended December 31, 2017 Financial Results:

Cash, cash equivalents and marketable securities totaled $363.3 million as of December 31, 2017, compared to $403.5 million on December 31, 2016. The 2017 year-end cash balance reflects operation spending net of $31.0 million in milestone payments received during the year. The 2016 year-end cash balance reflects the upfront proceeds of $150.0 million received from Xencor’s Novartis Collaboration and net proceeds of $119.3 million received from a financing in excess of spending on operations in 2016.

Revenues for the fourth quarter ended December 31, 2017 were $10.9 million, compared to $6.4 million for the same period in 2016. Revenues for full year 2017 were $35.7 million, compared to $87.5 million in 2016. Revenues in the three-month period ended December 31, 2017 were earned primarily from a milestone payment from Amgen, compared to revenues from the same period in 2016, which were earned primarily from a milestone payment received from Alexion. Total revenues earned in 2017 were lower than 2016, primarily due to revenue earned from the Amgen collaboration in 2017 compared to revenue earned from the Novartis collaboration in 2016.

Research and development expenditures for the fourth quarter ended December 31, 2017 were $20.4 million, compared to $13.4 million for the same period in 2016. Research and development expenditures were $71.8 million for the full year ended December 31, 2017, compared to $51.9 million in 2016. Research and development spending for the fourth quarter and full year ended December 31, 2017 was greater than expenditures incurred over comparable periods in 2016, primarily due to increased spending on Xencor’s bispecific oncology pipeline.

General and administrative expenses for the fourth quarter ended December 31, 2017 were $4.4 million, compared to $3.1 million in the same period in 2016. General and administrative expenses were $17.5 million in the full year 2017, compared to $13.1 million in 2016. Additional spending on general and administration for the full year ended December 31, 2017 over the comparable period in 2016 reflects increased stock based compensation charges.

Non-cash, share based compensation expense for the year ended December 31, 2017 was $13.7 million, compared to $7.8 million for the year ended December 31, 2016.

Net loss for the fourth quarter ended December 31, 2017 was $11.8 million, or $(0.25) on a fully diluted per share basis, compared to a net loss of $9.1 million, or $(0.21) on a fully diluted per share basis, for the same period in 2016. For the full year ended December 31, 2017, net loss was $48.9 million, or $(1.05) on a fully diluted per share basis, compared to a net income of $23.6 million, or $0.56 on a fully diluted per share basis, for the full year ended December 31, 2016. The higher loss for the three months ended December 31, 2017 over the loss reported for the same period in 2016 is primarily due to increased research and development spending, while the loss reported for the year ended December 31, 2017 compared to the income earned over the same period in 2016 is primarily due to the Novartis collaboration revenue reported in 2016 and increased expenses in 2017.

The total shares outstanding was 47,002,488 as of December 31, 2017, compared to 46,567,978 as of December 31, 2016.

Financial Guidance:

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations beyond 2020. Xencor expects to end 2018 with approximately $240 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 fourth quarter and full year 2017 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: 3991218. A live webcast of the conference call will be available online from the investor relations section of the company’s website at www.xencor.com. The webcast will be archived on the company’s website for 90 days.

On February 27, 2018 vTv Therapeutics Inc. (Nasdaq:VTVT) reported a corporate update and financial and operational results for the fourth quarter and full year that ended December 31, 2017 (Press release, vTv Therapeutics, FEB 27, 2018, View Source;p=RssLanding&cat=news&id=2335134 [SID1234524269]).

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"We made tremendous progress across the spectrum of our Alzheimer’s and diabetes programs this past year, and look forward to continuing this momentum in 2018 as we anticipate reporting topline results in April from Part A of our Phase 3 STEADFAST study of azeliragon in patients with mild Alzheimer’s disease," said Steve Holcombe, president and CEO of vTv Therapeutics. "Our unique and holistic approach to targeting Alzheimer’s through the receptor for advanced glycation endproducts (RAGE) antagonist addresses three major pathologies believed to contribute to the disease: transport of a-beta to the brain, inflammation and the phosphorylation of tau protein. We’re hopeful that results from the study will be a step toward finding a much-needed therapy for the Alzheimer’s community and the millions of people suffering from this devastating disease."

Fourth Quarter 2017 Highlights

vTv Therapeutics Initiates Phase 1b/2 Study as Part of Industry Partnership with the Juvenile Diabetes Research Foundation (JDRF)

Conducted with support from JDRF, the leading global organization funding type 1 diabetes (T1D) research, vTv Therapeutics initiated simplici-T1, an adaptive Phase1b/2 study assessing the pharmacokinetics, pharmacodynamics, safety and tolerability of vTv Therapeutics’ liver-selective glucokinase activator, TTP399, in type 1 diabetes. The study will evaluate whether TTP399 is well-tolerated when administered as an add-on to insulin therapy and can improve daily glucose profiles and HbA1c in people living with T1D.

vTv Therapeutics and Hangzhou Zhongmei Huadong Pharmaceutical Co. Enter Licensing Agreement for GLP-1r Diabetes Program

vTv Therapeutics successfully entered into a licensing agreement with Hangzhou Zhongmei Huadong Pharmaceutical Co., one of the largest pharmaceutical companies in China, for rights to develop and commercialize vTv Therapeutics’ GLP-1r agonist program, including TTP273, in China and other Pacific Rim countries. vTv Therapeutics received an $8 million upfront payment and is eligible for up to an additional $75 million in milestone payments related to development, regulatory and commercial milestones. In addition, vTv Therapeutics will be eligible to receive royalty payments on sales of commercialized products in the licensed territories. vTv will conduct a Phase 2 multi-region clinical trial, including sites in both the United States and China, to investigate the safety and efficacy of a lower dose of TTP273 in patients with type 2 diabetes.

vTv Therapeutics and Reneo Pharmaceuticals Enter Licensing Agreement for PPAR-delta Program

vTv Therapeutics granted Reneo Pharmaceuticals exclusive worldwide rights to research, develop and commercialize vTv Therapeutics’ selective peroxisome proliferator-activated receptor delta (PPAR-delta) program, including HPP593, in a global licensing agreement. Under the terms of the agreement, vTv Therapeutics received an upfront payment and is eligible to receive future development and commercialization milestones as well as royalties on sales of approved products. vTv Therapeutics also received shares of Reneo Pharmaceuticals’ common stock.

vTv Therapeutics Hosts Key Opinion Leader (KOL) Event on Current State of Clinical Development in Alzheimer’s Disease

vTv Therapeutics hosted a KOL presentation focused on the current state of clinical development in Alzheimer’s disease. The event featured two speakers with extensive experience in Alzheimer’s research and care: Dr. Howard Fillit, founding executive director and chief scientific officer of Alzheimer’s Drug Discovery Foundation, clinical professor of geriatric medicine, palliative care and neuroscience at Mt. Sinai School of Medicine; and Dr. Mary Sano, associate dean for clinical research, professor of psychiatry, founding member and director of the Alzheimer’s Disease Research Center at Mt. Sinai School of Medicine. vTv Therapeutics provided a brief overview of the company’s ongoing Phase 3 clinical development program for azeliragon, an orally bioavailable small molecule RAGE antagonist for patients with mild Alzheimer’s disease.

Upcoming Anticipated Milestones

vTv Therapeutics anticipates reporting topline data from Part A of the company’s Phase 3 STEADFAST Study in April 2018. Data from Part B are expected to read out in early 2019. The STEADFAST study is a single protocol within which vTv Therapeutics is conducting two statistically independent, identical, randomized, double-blind, placebo-controlled trials investigating the efficacy of azeliragon as a potential treatment of mild Alzheimer’s disease.

Shelf Registration on Form S-3

Today the Company filed a S-3 Registration Statement with the Securities and Exchange Commission to register Class A Common Stock. The Company has no current plans to issue securities under the Registration Statement.

Fourth Quarter 2017 Financial Results

Cash Position: Cash and cash equivalents as of December 31, 2017, were $11.8 million compared to $20.5 million as of September 30, 2017.
R&D Expenses: Research and development expenses were $10.1 million in the fourth quarter of 2017, compared to $9.0 million in the third quarter of 2017. The increase in research and development expense was primarily driven by increased enrollment in the open-label extension trial and higher consulting costs incurred related to the STEADFAST Study.
G&A Expenses: General and administrative expenses were $2.9 million and $2.6 million, for the fourth and third quarters of 2017, respectively. The increase in general and administrative cost was primarily due to the higher professional service fees incurred in the fourth quarter of 2017 related to our license agreements entered into in December 2017.
Net Loss Before Non-Controlling Interest: Net loss before non-controlling interest was $14.6 million for the fourth quarter of 2017 compared to net loss before non-controlling interest of $12.4 million for the third quarter of 2017.
Net Loss per Share: GAAP net loss per share was $0.44 and $0.38 for the three months ended December 31, 2017 and September 30, 2017, respectively, based on weighted-average shares of 9.7 million in each period. Non-GAAP net loss per fully exchanged share was $0.44 and $0.38 for the three months ended December 31, 2017 and September 30, 2017, respectively, based on non-GAAP fully exchanged weighted-average shares of 32.8 million in each period.

Full Year 2017 Financial Results

R&D Expenses: Research and development expenses were $39.6 million in 2017, compared to $45.7 million in 2016. The decrease in research and development expense was primarily driven by decreases in clinical trial costs for TTP399 and TTP273 as both the AGATA and LOGRA studies were completed in 2016. Additionally, we saw decreases in the expense for azeliragon as a result of the completion of drug-drug interaction and other supporting studies in 2016 which were partially offset by increases in cost related to continuing enrollment in the open-label extension trial and increased cost of consultants engaged to assist with the STEADFAST Study.
G&A Expenses: General and administrative expenses were $11.3 million and $9.9 million, for the 2017 and 2016, respectively. The increase in general and administrative cost was primarily due to the higher professional service fees incurred in 2017 related to our license agreements entered into in December 2017 and increased compensation cost related to the grant of additional share-based compensation awards as well as the impact of additional personnel hired in both years.
Net Loss Before Non-Controlling Interest: Net loss before non-controlling interest was $54.6 million for 2017 compared to net loss before non-controlling interest of $55.4 million for 2016.
Net Loss per Share: GAAP net loss per share was $1.67 and $1.71 for 2017 and 2016, respectively, based on weighted-average shares of 9.7 million and 9.5 million in each period, respectively. Non-GAAP net loss per fully exchanged share was $1.67 and $1.69 for 2017 and 2016, respectively, based on non-GAAP fully exchanged weighted-average shares of 32.8 million in each period.

vTv Therapeutics Inc.
Condensed Consolidated Balance Sheets
(in thousands)

December 31, September 30,
2017 2017
(Unaudited)
Assets
Current assets:
Cash and cash equivalents $ 11,758 $ 20,488
Restricted cash and cash equivalents 162 281
Accounts receivable, net 8,000 —
Prepaid expenses and other current assets 442 725
Total current assets 20,362 21,494
Restricted cash and cash equivalents, long-term 2,500 —
Property and equipment, net 283 310
Long-term investments 2,480 —
Long-term deposits 2,292 2,251
Total assets $ 27,917 $ 24,055
Liabilities, Redeemable Noncontrolling Interest and Stockholders’ Deficit
Current liabilities:
Accounts payable and accrued expenses $ 13,901 $ 10,120
Deferred revenue 8,757 —
Current portion of notes payable 4,271 2,083
Total current liabilities 26,929 12,203
Notes payable 15,316 17,228
Deferred revenue, net of current portion 4,497 —
Warrant liability, related party 492 —
Other liabilities 290 285
Total liabilities 47,524 29,716
Commitments and contingencies
Redeemable noncontrolling interest 131,440 130,642
Stockholders’ deficit:
Class A Common Stock 97 97
Class B Common Stock 232 232
Additional paid-in capital 127,682 127,036
Accumulated deficit (279,058 ) (263,668 )
Total stockholders’ deficit attributable to vTv Therapeutics Inc. (151,047 ) (136,303 )
Total liabilities, redeemable noncontrolling interest and stockholders’ deficit $ 27,917 $ 24,055


vTv Therapeutics Inc.
Condensed Consolidated Statements of Operations – Unaudited
(in thousands, except per share data)

Three Months Ended
September 30,
December 31, 2017 2017
Revenue $ 233 $ 15
Operating expenses:
Research and development 10,068 8,989
General and administrative 2,937 2,567
Total operating expenses 13,005 11,556
Operating loss (12,772 ) (11,541 )
Interest income 22 35
Interest expense (852 ) (849 )
Other expense, net (190 ) —
Loss before income taxes and noncontrolling interest (13,792 ) (12,355 )
Income tax provision 800 —
Net loss before noncontrolling interest (14,592 ) (12,355 )
Less: net loss attributable to noncontrolling interest (10,281 ) (8,705 )
Net loss attributable to vTv Therapeutics Inc. $ (4,311 ) $ (3,650 )

Net loss per share of vTv Therapeutics Inc. Class A Common Stock, basic and diluted
$ (0.44 ) $ (0.38 )

Weighted-average number of vTv Therapeutics Inc. Class A Common Stock, basic and diluted
9,693,254 9,693,254


vTv Therapeutics Inc.
Condensed Consolidated Statements of Operations
(in thousands, except per share data)

Three Months Ended For the Year Ended
December 31, December 31,
(Unaudited)
2017 2016 2017 2016
Revenue $ 233 $ 38 $ 291 $ 634
Operating expenses:
Research and development 10,068 11,099 39,640 45,748
General and administrative 2,937 2,252 11,333 9,906
Total operating expenses 13,005 13,351 50,973 55,654
Operating loss (12,772 ) (13,313 ) (50,682 ) (55,020 )
Interest income 22 20 117 87
Interest expense (852 ) (394 ) (3,092 ) (398 )
Other expense, net (190 ) (24 ) (190 ) (22 )
Loss before income taxes and noncontrolling interest (13,792 ) (13,711 ) (53,847 ) (55,353 )
Income tax provision 800 — 800 —
Net loss before noncontrolling interest (14,592 ) (13,711 ) (54,647 ) (55,353 )
Less: net loss attributable to noncontrolling interest (10,281 ) (9,661 ) (38,503 ) (39,001 )
Net loss attributable to vTv Therapeutics Inc. $ (4,311 ) $ (4,050 ) $ (16,144 ) $ (16,352 )

Net loss per share of vTv Therapeutics Inc. Class A Common Stock, basic and diluted
$ (0.44 ) $ (0.42 ) $ (1.67 ) $ (1.71 )

Weighted-average number of vTv Therapeutics Inc. Class A Common Stock, basic and diluted
9,693,254 9,693,254 9,693,254 9,545,527