On March 15, 2018 Molecular Templates, Inc., (Nasdaq:MTEM) a clinical stage biopharmaceutical company focused on the discovery and development of Engineered Toxin Bodies, a new class of targeted biologic therapies that possess unique mechanisms of action in oncology, reported that new data on two of its pipeline programs will be presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2018, to be held April 14-18 at McCormick Place North/South in Chicago, Illinois (Press release, Molecular Templates, MAR 15, 2018, View Source [SID1234524813]).

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Molecular Templates has utilized the ability of its Engineered Toxin Bodies (ETBs) to route to the endoplasmic reticulum and cytoplasm to deliver cytomegalovirus (CMV) foreign antigen for presentation in the context of MHC class I molecules on tumor cells. This Antigen Seeding Technology (AST) allows for the targeted delivery, intracellular processing, and surface MHC-I presentation of CMV antigen and subsequent destruction of tumors via a CMV-specific T lymphocyte response.

Preclinical data on the company’s PD-L1 targeted ETB incorporating AST will be presented at the AACR (Free AACR Whitepaper) meeting. This molecule has been engineered to both destroy PD-L1-expressing tumor cells via ribosomal destruction and to deliver the immunodominant HLA:A02 restricted cytomegalovirus (CMV) protein-pp65 for MHC-I presentation on these cells. The dual mechanisms of action against the PD-L1 target allow for a more potent and complete destruction of tumor cells. Molecular Templates expects this program to enter the clinic in the first half of 2019.

"We are excited by this wholly novel approach to immuno-oncology," said Eric Poma, Ph.D., CEO and CSO of Molecular Templates. "Antigen seeding is a way to target an immense, activated T-cell repertoire to the tumor without having to overcome T-cell exhaustion or stimulate antigen presentation from APCs. We are looking forward moving to this approach into the clinic."

Date: Monday, April 16
Time: 1:00pm – 5:00pm Central Time
Session: PO.IMO2.11 – Therapeutic Antibodies, Including Engineered Antibodies 2
Location: Section 34
Poster Title: Antigen Seeding Technology by Engineered Toxin Bodies Provides a Targeted Immuno-Oncology Approach for Treatment of Cancers
Authors: Brigitte Brieschke, Sangeetha Rajagopalan, Garrett L. Robinson, Erin K. Willert, Hilario J. Ramos

Molecular Templates will also present on its HER2 engineered toxin body program. MT-5111, a HER2-targeted ETB with picomolar potency against HER2 expressing cells, was designed to overcome mechanisms of resistance to current HER2 targeting modalities such as escape from antibody dependent cell-mediated cytotoxicity (ADCC), alterations in signal transduction, epitope masking, and enhanced small molecule efflux. Additionally, MT-5111 was genetically engineered to reduce the anti-drug antibody response and signaling through innate receptors, allowing for repeat dosing.

In vitro and in vivo data will be presented at the AACR (Free AACR Whitepaper) meeting, highlighting the potential for MT-5111 as a novel agent in development for treatment of breast carcinomas and other malignancies overexpressing the HER2 receptor. Molecular Templates intends to file an IND with MT-5111 in 2018.

"Antibody, small molecule, and ADC approaches to HER2 have all shown meaningful benefit in patients with HER2+ cancers but ultimately cease to work in most patients even though HER2 expression persists," said Eric Poma, Ph.D., CEO and CSO of Molecular Templates. "We believe the unique mechanism of action of our HER2 ETB may circumvent mechanisms of resistance to the other HER2 modalities."

Date: Wednesday, April 18
Time: 8:00am – 12:00pm Central Time
Session: PO.ET01.02 – Antibodies, Fusion Proteins, and Related Biologics
Location: Section 35
Poster Title: Targeted Engineered Toxin Bodies Provide a Novel Mechanism of Action Against HER2 Positive Cancers
Authors: Brigitte Brieschke, Garrett L. Robinson, Sangeetha Rajagopalan, Hilario J. Ramos, Jensing Liu, Jack P. Higgins, Erin K. Willert

On March 15, 2018 Exelixis, Inc. (NASDAQ:EXEL) reported it has completed the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for CABOMETYX (cabozantinib) tablets as a treatment for patients with previously treated advanced hepatocellular carcinoma (HCC) (Press release, Exelixis, MAR 15, 2018, View Source;p=RssLanding&cat=news&id=2338226 [SID1234525492]).The sNDA submission is based on results from the CELESTIAL randomized pivotal phase 3 trial of CABOMETYX in patients with advanced HCC who received prior sorafenib.

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"We look forward to working closely with regulatory authorities through the review process in anticipation of bringing CABOMETYX to people diagnosed with advanced hepatocellular carcinoma, an underserved patient community that urgently needs new treatment options," said Gisela Schwab, M.D., President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis. "We would sincerely like to thank the study patients and clinicians who participated in the CELESTIAL trial as well as our dedicated clinical development, medical and regulatory teams for bringing us another step closer to our goal of fully exploring the potential of CABOMETYX and making it accessible to every patient who may benefit from its use."

On October 16, 2017, Exelixis announced that the independent data monitoring committee for the study recommended that the CELESTIAL trial be stopped for efficacy following review at the second planned interim analysis, with cabozantinib providing a statistically significant and clinically meaningful improvement in OS compared with placebo in patients with previously treated advanced HCC (pre-specified critical p-value ≤ 0.021). In March 2017, the FDA granted orphan drug designation to cabozantinib for the treatment of advanced HCC.

An sNDA is an application to the FDA that, if approved, will allow a drug sponsor to make changes to a previously approved product label, including modifications to the indication.

About the CELESTIAL Study
CELESTIAL is a randomized, double-blind, placebo-controlled study of cabozantinib in patients with advanced HCC conducted at more than 100 sites globally in 19 countries. The trial was designed to enroll 760 patients with advanced HCC who received prior sorafenib and may have received up to two prior systemic cancer therapies for HCC and had adequate liver function. Enrollment of the trial was completed in September 2017. Patients were randomized 2:1 to receive 60 mg of cabozantinib once daily or placebo and were stratified based on etiology of the disease (hepatitis C, hepatitis B or other), geographic region (Asia versus other regions) and presence of extrahepatic spread and/or macrovascular invasion (yes or no). No cross-over was allowed between the study arms during the blinded treatment phase of the trial. The primary endpoint for the trial is OS, and secondary endpoints include objective response rate and progression-free survival. Exploratory endpoints include patient-reported outcomes, biomarkers and safety.

About HCC
Liver cancer is the second-leading cause of cancer death worldwide, accounting for more than 700,000 deaths and nearly 800,000 new cases each year.1 In the U.S., the incidence of liver cancer has more than tripled since 1980.2 HCC is the most common form of liver cancer, making up about three-fourths of the estimated nearly 42,000 new cases in the U.S. in 2018.2 HCC is the fastest-rising cause of cancer-related death in U.S.3 Without treatment, patients with advanced HCC usually survive less than 6 months.4

About CABOMETYX (cabozantinib)
CABOMETYX tablets are approved in the United States for the treatment of patients with advanced RCC. CABOMETYX tablets are also approved in the European Union, Norway, Iceland, Australia, Switzerland and South Korea for the treatment of advanced RCC in adults who have received prior vascular endothelial growth factor (VEGF)-targeted therapy. Ipsen also submitted to European Medicines Agency (EMA) the regulatory dossier for cabozantinib as a treatment for first-line advanced RCC in the European Union on August 28, 2017; on September 8, 2017, Ipsen announced that the EMA validated the application. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan, including RCC.

CABOMETYX is not indicated for previously treated advanced HCC.

Please see Important Safety Information below and full U.S. prescribing information at View Source

U.S. Important Safety Information

Hemorrhage: Severe and fatal hemorrhages have occurred with CABOMETYX. In two RCC studies, the incidence of Grade ≥ 3 hemorrhagic events was 3% in CABOMETYX-treated patients. Do not administer CABOMETYX to patients that have or are at risk for severe hemorrhage.
Gastrointestinal (GI) Perforations and Fistulas: In RCC studies, fistulas were reported in 1% of CABOMETYX-treated patients. Fatal perforations occurred in patients treated with CABOMETYX. In RCC studies, gastrointestinal (GI) perforations were reported in 1% of CABOMETYX-treated patients. Monitor patients for symptoms of fistulas and perforations, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a fistula which cannot be appropriately managed or a GI perforation.
Thrombotic Events: CABOMETYX treatment results in an increased incidence of thrombotic events. In RCC studies, venous thromboembolism occurred in 9% (including 5% pulmonary embolism) and arterial thromboembolism occurred in 1% of CABOMETYX-treated patients. Fatal thrombotic events occurred in the cabozantinib clinical program. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or any other arterial thromboembolic complication.
Hypertension and Hypertensive Crisis: CABOMETYX treatment results in an increased incidence of treatment-emergent hypertension, including hypertensive crisis. In RCC studies, hypertension was reported in 44% (18% Grade ≥ 3) of CABOMETYX-treated patients. Monitor blood pressure prior to initiation and regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume CABOMETYX at a reduced dose. Discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy. Discontinue CABOMETYX if there is evidence of hypertensive crisis or severe hypertension despite optimal medical management.
Diarrhea: In RCC studies, diarrhea occurred in 74% of patients treated with CABOMETYX. Grade 3 diarrhea occurred in 11% of patients treated with CABOMETYX. Withhold CABOMETYX in patients who develop intolerable Grade 2 diarrhea or Grade 3-4 diarrhea that cannot be managed with standard antidiarrheal treatments until improvement to Grade 1; resume CABOMETYX at a reduced dose.
Palmar-Plantar Erythrodysesthesia (PPE): In RCC studies, palmar-plantar erythrodysesthesia (PPE) occurred in 42% of patients treated with CABOMETYX. Grade 3 PPE occurred in 8% of patients treated with CABOMETYX. Withhold CABOMETYX in patients who develop intolerable Grade 2 PPE or Grade 3 PPE until improvement to Grade 1; resume CABOMETYX at a reduced dose.
Reversible Posterior Leukoencephalopathy Syndrome (RPLS), a syndrome of subcortical vasogenic edema diagnosed by characteristic finding on MRI, occurred in the cabozantinib clinical program. Perform an evaluation for RPLS in any patient presenting with seizures, headache, visual disturbances, confusion or altered mental function. Discontinue CABOMETYX in patients who develop RPLS.
Embryo-fetal Toxicity may be associated with CABOMETYX. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during CABOMETYX treatment and for 4 months after the last dose.
Adverse Reactions: The most commonly reported (≥25%) adverse reactions are: diarrhea, fatigue, nausea, decreased appetite, hypertension, PPE, weight decreased, vomiting, dysgeusia, and stomatitis.
Strong CYP3A4 Inhibitors: If concomitant use with strong CYP3A4 inhibitors cannot be avoided, reduce the CABOMETYX dosage.
Strong CYP3A4 Inducers: If concomitant use with strong CYP3A4 inducers cannot be avoided, increase the CABOMETYX dosage.
Lactation: Advise women not to breastfeed while taking CABOMETYX and for 4 months after the final dose.
Hepatic Impairment: In patients with mild to moderate hepatic impairment, reduce the CABOMETYX dosage. CABOMETYX is not recommended for use in patients with severe hepatic impairment.
Please see accompanying full Prescribing Information View Source

On March 15, 2018 Novavax, Inc., (Nasdaq: NVAX) reported its financial results for the fourth quarter and twelve months ended December 31, 2017 (Press release, Novavax, MAR 15, 2018, View Source [SID1234524815]).

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"During this last quarter we collected our most significant results to date from our two lead vaccine programs," said Stanley C. Erck, President and CEO, Novavax, Inc. "This progress gives us enhanced focus and momentum to execute under these programs for the remainder of 2018 and beyond."

NanoFlu Program Update:

In late February 2018, the Company announced positive top-line results from its Phase 1/2 clinical trial in 330 older adults of its NanoFlu recombinant influenza vaccine, which includes its proprietary Matrix-M adjuvant, compared to the leading licensed egg-based, high-dose influenza vaccine for older adults (IIV3-HD). Key findings from the trial show that Novavax’ trivalent NanoFlu vaccine induced significantly higher hemagglutination inhibition (HAI) antibody responses against homologous A-type strains as well as against historic and forward-drifted H3N2 strains. Based on the strength of these trial results, the Company submitted a related manuscript to a peer-reviewed medical journal and is planning to present the data at the World Vaccine Congress meeting on April 4, 2018.

"This influenza season in the Northern Hemisphere has resulted in a serious public health epidemic, largely because of the H3N2 flu strain and the inability of current vaccines to provide adequate protection, particularly to older adults and other vulnerable populations," said Gregory M. Glenn, M.D., President of Research and Development. "Our NanoFlu vaccine’s head-to-head performance against IIV3-HD demonstrated that it has the potential to address two primary confounding factors related to poor vaccine efficacy: virus drift and vaccine mutation resulting from egg-based manufacturing. With these findings, we are able to initiate manufacturing and clinical operations activities to support our next step, a Phase 2 trial of a quadrivalent formulation of our NanoFlu vaccine, scheduled to begin in the third quarter of this year."

RSV F Vaccine Maternal Immunization Program Update:

In December 2017, the Company completed a successful informational analysis of the Phase 3 Prepare clinical trial of its RSV F Vaccine for infants via maternal immunization. The analysis of data from 1,307 infants in the per-protocol population indicate an observed vaccine efficacy in the range of between 45% and 100%. The Company anticipates that it will reach approximately 4,600 participants, including approximately 3,000 actively vaccinated mothers, in the second quarter of 2018, which will enable an interim efficacy analysis with results reported in early 2019. This program continues to be funded under an $89 million grant from the Bill and Melinda Gates Foundation (BMGF), and has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA).

"The results of our informational analysis this December significantly increased the likelihood of success of our RSV F Vaccine program for infants via maternal immunization," added Mr. Erck. "With over 4,000 current participants, we are very close to triggering the interim efficacy analysis, the positive results of which would form the basis of our Biologics License Application filing with the FDA. Providing protection to newborns from respiratory syncytial virus, one of the most prevalent and damaging diseases to which they are exposed during their first months of life, has important global public health implications."

Financial Results for the Three and Twelve Months Ended December 31, 2017

Novavax reported a net loss of $50.8 million, or $0.16 per share, for the fourth quarter of 2017, compared to a net loss of $57.1 million, or $0.21 per share, for the fourth quarter of 2016. For the twelve months ended December 31, 2017, the net loss was $183.8 million, or $0.63 per share, compared to a net loss of $280.0 million, or $1.03 per share, for the same period in 2016.

Novavax revenue in the fourth quarter of 2017 was $10.4 million, compared to $5.4 million in the same period in 2016. This 93% increase was driven by higher revenue recorded under the BMGF grant of $89 million.

Research and development expenses decreased 3% to $49.7 million in the fourth quarter of 2017, compared to $51.1 million for the same period in 2016. The decrease was primarily due to reduced development activities of our RSV F Vaccine for older adults, partially offset by increased development activities of our RSV F Vaccine for infants via maternal immunization.

Interest income (expense), net for the fourth quarter of 2017 and 2016 was ($3.1) million.

As of December 31, 2017, the company had $157.3 million in cash, cash equivalents and marketable securities, compared to $235.5 million as of December 31, 2016. Net cash used in operating activities for the full year 2017 was $138.7 million, compared to $255.5 million for same period in 2016. The decrease in cash usage was primarily due to decreased costs relating to our RSV F Vaccine and lower overall employee-related costs.

Conference Call

Novavax management will host its quarterly conference call today at 5:00 p.m. ET. The dial-in number for the conference call is (877) 212-6076 (Domestic) or (707) 287-9331 (International), passcode 6472939. A replay of the conference call will be available starting at 7:30 p.m. ET on March 14, 2018 until 8:30 pm ET on March 21, 2018. To access the replay by telephone, dial (855) 859-2056 (Domestic) or (404) 537-3406 (International) and use passcode 6472939.

A webcast of the conference call can also be accessed via a link on the home page of the Novavax website (novavax.com) or through the "Investor Info"/"Events" tab on the Novavax website. A replay of the webcast will be available on the Novavax website until June 14, 2018.

About Influenza

Influenza is a world-wide infectious disease that causes illness in humans with symptoms ranging from mild to life-threatening or even death. Serious illness occurs not only in susceptible populations such as infants, young children and older adults, but also in the general population largely because of infection by continuously evolving strains of influenza which can evade the existing protective antibodies in humans. An estimated one million deaths each year are attributed to influenza.1 Current estimates for seasonal influenza vaccine growth in the top seven markets (U.S., Japan, France, Germany, Italy, Spain and UK), show a potential increase from approximately $3.2 billion in 2015 to $5.3 billion by 2025.2

About the Phase 1/2 Clinical Trial

Novavax conducted a randomized, observer-blind, comparator-controlled trial of NanoFlu vaccine (in two trivalent formulations: 45µg or 180µg total HA) against IIV3-HD in 330 healthy adults aged 60 years or older. Vaccine immunogenicity was measured by HAI and neutralization antibody responses against a panel of vaccine-homologous, and historically and forward-drifted, influenza virus strains.

About NanoFlu and Matrix M

NanoFlu vaccine is a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine candidate produced by Novavax in its SF9 insect cell baculovirus system. NanoFlu vaccine uses HA amino acid protein sequences that are substantially the same as wild-type circulating virus HA sequences. NanoFlu vaccine contains Novavax’ patented saponin-based Matrix-M adjuvant, which has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes.

About RSV

RSV is the most common cause of lower respiratory tract infections and the leading viral cause of severe lower respiratory tract disease in infants and young children worldwide, with estimated annual infection and mortality rates of 64 million and 160,000, respectively.3 In the US, RSV is the leading cause of hospitalization of infants.4 Despite the induction of post-infection immunity, repeat infection and lifelong susceptibility to RSV is common.5 Currently, there is no approved RSV vaccine available.

1 Resolution of the World Health Assembly (2003) WHA56.19.28
2 Influenza Vaccines Forecasts. Datamonitor (2013)
3 View Source
4 Leader S. Pediatr Infect Dis J. 2002 Jul;21(7):629-32
5 PLOS. "How immunity to respiratory syncytial virus develops in childhood, deteriorates in adults." ScienceDaily. 21 April 2016. View Source

About RSV F Vaccine for Infants via Maternal Immunization

Novavax is developing a vaccine that targets the fusion protein, or F-protein, of the RSV virus. The F-protein has highly conserved amino acid sequences, called antigenic sites, which are the target of neutralizing antibodies and are believed to be ideal vaccine targets. Novavax’ genetically engineered novel F-protein antigen exposes a range of these antigenic sites, and can evoke immune responses to them in human vaccine recipients. In a previous Phase 2 clinical trial of the RSV F Vaccine, which assessed the transplacental transfer of maternal antibodies induced by the vaccine, immunized women demonstrated meaningful fold rises in anti-F IgG, palivizumab-competing antibodies and microneutralization titers. In addition, infants’ antibody levels at delivery averaged 90-100% of the mothers’ levels, indicating efficient transplacental transfer of antibodies from mother to infant.

About the U.S. Food and Drug Administration’s (FDA) Fast Track Program

The Fast Track Drug Development Program was established under the FDA Modernization Act of 1997. A Fast Track designation is intended for products that treat serious or life-threatening diseases or conditions, and that demonstrate the potential to address unmet medical needs for such diseases or conditions. The program is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously. Specifically, Fast Track designation facilitates meetings to discuss all aspects of development to support licensure and it provides the opportunity to submit sections of a BLA on a rolling basis as data become available, which permits the FDA to review modules of the BLA as they are received instead of waiting for the entire BLA submission. In addition, priority review (six month review versus standard ten month review) is a potential benefit that may be available to Novavax’ RSV F vaccine in the future.

On March 15, 2018 ArQule, Inc. (Nasdaq: ARQL) reported that pre-clinical and clinical data on the company’s pipeline of drug candidates will be presented at the 2018 AACR (Free AACR Whitepaper) Annual Meeting taking place in Chicago from April 14-April 18 (Press release, , MAR 15, 2018, View Source [SID1234524785]). Data will be presented in one oral and nine poster sessions on trials conducted by ArQule and its collaborators for BTK inhibitor, ARQ 531, AKT inhibitors, miransertib and ARQ 751, as well as FGFR inhibitor, derazantinib.

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The oral presentation will highlight data from a phase 1b trial for miransertib in combination with anastrozole in PIK3CA or AKT1-mutant endometrial and ovarian cancers conducted by Dr. David M. Hyman and colleagues at Memorial Sloan Kettering.

Oral Presentation Details

April 15, 2018

Title: A phase 1b study of Miransertib (ARQ 092) in combination with anastrozole in patients with PIK3CA or AKT1-mutant ER+ endometrial and ovarian cancer
Time: 3:00 – 5:00 p.m. CT
Location: Room N427 – McCormick Place North, Level 4
Sponsor: Memorial Sloan Kettering, New York, NY

Poster Presentation Details

April 15, 2018

Title: ARQ 531, a Novel and Reversible Inhibitor of Bruton’s Tyrosine Kinase, Displays Favorable Oral Bioavailability and Exposure in patients with B-cell malignancies
Time: 1:00 – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 43, Poster Board 18
Sponsor: ArQule, Inc.

Title: The novel Bruton’s tyrosine kinase inhibitor ARQ 531 disrupts survival signaling and triggers apoptosis in AML cells
Time: 1:00 – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 37, Poster Board 3
Sponsor: University of Genoa, Genova, Italy

Title: Results of A Phase 1 Dose Escalation Study of ARQ 751 in Adult Subjects with Advanced Solid Tumors with AKT1, 2, 3 Genetic Alterations, Activating PI3K Mutations, PTEN-null, or other known actionable PTEN mutations
Time: 1:00 – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 42, Poster Board 17
Sponsor: MD Anderson Cancer Center, Houston, TX

April 16, 2018

Title: ARQ 531, a potent reversible BTK inhibitor exhibits potent antitumor activity in ibrutinib resistant diffuse large B-cell lymphoma
Time: 8:00 a.m. – 12:00 p.m. CT
Location: Exhibit Hall A, Poster Section 41, Poster Board 8
Sponsor: ArQule, Inc.

Title: Preclinical Evaluation of the Tyrosine Kinase Inhibitor ARQ531 in AML
Time: 8:00 a.m. – 12:00 p.m. CT
Location: Exhibit Hall A, Poster Section 38, Poster Board 13
Sponsor: The Ohio State University, Columbus OH

Title: In vivo combination of Miransertib (ARQ 092) with anti-PD-1 antibody, Trametinib, Lapatinib, Trastuzumab and Paclitaxel
Time: 1:00 p.m. – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 41, Poster Board 23
Sponsor: ArQule, Inc.

April 17, 2018

Title: Derazantinib (ARQ 087) Pharmacodynamics: Alterations in FGF19/21/23 and phosphate in Patients with Cholangiocarcinoma
Time: 8:00 a.m. – 12:00 p.m. CT
Location: Exhibit Hall A, Poster Section 43, Poster Board 22
Sponsor: ArQule, Inc.

Title: In vitro and in vivo effect of ARQ 531 on Trk family kinases
Time: 1:00 p.m. – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 36, Poster Board 26
Sponsor: ArQule, Inc.

Title: Combinations of imatinib mesylate with AKT inhibitor (Miransertib, ARQ 751) or FGFR inhibitor (Derazantinib) show synergy in GIST cell lines and pre-clinical models
Time: 1:00 p.m. – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 37, Poster Board 7
Sponsor: Fox Chase Cancer Center, Philadelphia, PA

About Miransertib and ARQ 751

Miransertib (ARQ 092) and ARQ 751 are orally bioavailable, selective small molecule inhibitors of the AKT serine/threonine kinase. The AKT pathway when abnormally activated is implicated in multiple oncogenic processes such as cell proliferation and apoptosis. This pathway has emerged as a target of potential therapeutic relevance for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications.

Miransertib, the lead compound in ArQule’s AKT program, has completed phase 1a clinical testing and has advanced into phase 1b expansion testing in cohorts of patients with endometrial cancer, lymphomas and tumors harboring either AKT or PI3K mutations. A company sponsored phase 1/2 trial is being conducted in the U.S. and E.U. for Overgrowth Diseases, including PROS and Proteus syndrome. Miransertib is also in a phase 1 trial being conducted by the NIH for Proteus syndrome.

On March 15, 2018 Pfenex Inc. (NYSE American: PFNX) is a clinical-stage development and licensing biotechnology company focused on leveraging its Pfenex Expression Technology to improve protein therapies for unmet patient needs. Using the patented Pfenex Expression Technology platform, the Company has created an advanced pipeline of therapeutic equivalents, vaccines, biologics and biosimilars. Today Pfenex Inc. reported financial results for the fourth quarter and full year ended December 31, 2017 and provided a business update. (Press release, Pfenex, MAR 15, 2018, View Source2018-03-15-Pfenex-Reports-Fourth-Quarter-and-Full-Year-2017-Results-and-Provides-Business-Update" target="_blank" title="View Source2018-03-15-Pfenex-Reports-Fourth-Quarter-and-Full-Year-2017-Results-and-Provides-Business-Update" rel="nofollow">View Source [SID1234524816]).

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Pfenex logo (PRNewsFoto/Pfenex) (PRNewsfoto/Pfenex Inc.)

"Throughout 2017 we made considerable progress in advancing our lead product candidates as well as achieving substantial development milestones on our partnered programs," said Eef Schimmelpennink, chief executive officer of Pfenex. "I joined Pfenex in August as the company’s chief executive officer because I saw a company with a unique and validated protein expression platform, a broad differentiated portfolio and a very talented team, but, was in need of change. From day-one the team and I focused on defining our priorities and setting a new course. We ended the year with a clear and concise strategic focus that leverages the strength of the Pfenex Expression Technology platform through the development of our own key assets in our pipeline, while selectively collaborating with strong partners. Pfenex is at a transformational point in our development, and I look forward to leading the team and continuing to build Pfenex into an industry success."

Business Updates and 2017 Highlights

PF708 therapeutic equivalent to Forteo (teriparatide). The Company completed the last patient visit in mid-February 2018 from its on-going PF708-301 trial which compares the effects of PF708 and Forteo in osteoporosis patients. The Company expects top-line immunogenicity data results in the second quarter of 2018. Pfenex believes that results from its PF708-301 trial, along with the bioequivalence findings from its PF708-101 trial in healthy subjects announced previously, should satisfy the clinical filing requirements for PF708. Pfenex expects to submit the new drug application (NDA) to the FDA in the third quarter of 2018, with a potential commercial launch possible in the US as early as the third quarter of 2019 upon expiration of the relevant patents and subject to receipt of US FDA marketing authorization.

Jazz Collaboration Agreement. In December 2017, Pfenex announced that under the amended collaboration agreement with Jazz Pharmaceuticals, Pfenex will be eligible to receive an additional $43.5 million in amendment fee and development milestone payments bringing the total value of payments and potential payments associated with the collaboration to $224.5 million. Upon signing the amended and restated agreement, Pfenex received a total of $18.5 million, consisting of an upfront payment of $5.0 million and a payment of $13.5 million for development achievement. Pfenex may also be eligible to receive tiered royalties on worldwide sales of any products resulting from the collaboration at rates reduced from those under the 2016 agreement. Finally, in the third quarter of 2017, we completed a process development milestone associated with this collaboration.

Px563L and RPA563. The development of the Company’s novel anthrax vaccine candidates is funded through an advanced development contract with the Department of Health and Human Services through the Biomedical Advanced Research and Development Authority (BARDA) valued at up to approximately $143.5 million. In January of 2017, BARDA exercised additional phases of the development contract, allowing for the continuing development of both Px563L and RPA563. In the second quarter of 2017, Pfenex completed its long-term follow-up of the Phase 1 study subjects, and the results showed no change to the interim safety or immunogenicity results. In October 2017, Pfenex completed a meeting with the FDA in which the Agency provided guidance for the proposed clinical development and licensure plans for post-exposure prophylaxis indication. Over the course of 2017, the Company continued to collect favorable stability data for both products. Potential next milestones in 2018 are triggering of analytical and non-clinical animal study options leading to potential Phase 2 study in 2019, subject in each case to continued funding by BARDA.

Financial Highlights for the Fourth Quarter and Full Year 2017

Total Revenue increased to $17.9 million in the three-month period ended December 31, 2017 compared to $5.5 million in same period in 2016. The increase in the three month period was due primarily to development achievements related to the Jazz collaboration. Total revenue decreased to $28.8 million in the year ended December 31, 2017 compared to $60.2 million in 2016. The year over year decrease in revenue was primarily due to the termination of the development and license agreement with Pfizer in the third quarter of 2016, which accelerated the recognition of revenue that had been previously deferred. In addition, in 2016 Pfenex recognized revenue of $4.9 million attributable to amortization of a development and license fee. The decrease in revenue was partially offset by $21.5 million of revenue recognized in 2017 from Jazz for development achievement and achievement of certain development milestones, development services and amortization of license fees.

Cost of revenue increased to $1.7 million in the three-month period ended December 31, 2017 compared to $1.3 million in same period in 2016. The change was due primarily to a net increase in costs for the Company’s proprietary novel vaccine program Px563L, which is funded by BARDA. Cost of revenue decreased to $5.2 million in the year ended December 31, 2017 compared to $5.3 million in 2016. The change was primarily due to a net decrease of costs for the Company’s proprietary novel vaccine program Px563L, which is funded by BARDA.

Research and development expenses decreased to $7.2 million in the three-month period ended December 31, 2017 compared to $10.7 million in same period in 2016. The decrease was primarily due to the Company’s decision to pause the Company’s development activities on certain product candidates. Research and development expenses decreased to $31.9 million in the year ended December 31, 2017 compared to $32.4 million in 2016. The decrease was primarily due to the Company’s decision to pause the Company’s development activities on certain product candidates. The decrease was partially offset by increased costs related to clinical trials for PF708, which began in the first quarter of 2017, and research and development expenses of hematology/oncology products related to the Company’s collaboration with Jazz, including PF743, a recombinant crisantaspase, and PF745, a recombinant crisantaspase with half-life extension technology, as well as other biosimilar product candidates.

Selling, general and administration expenses decreased to $3.7 million in the three-month period ended December 31, 2017 compared to $4.4 million in same period in 2016. This decrease in costs was primarily due to the departure of the Company’s former CFO during the fourth quarter of 2017. In addition, in the fourth quarter of 2016, Pfenex incurred costs associated with the Audit Committee’s investigation. Selling, general and administration expenses increased to $17.7 million in the year ended December 31, 2017 compared to $17.3 million in 2016. The year over year increase was primarily due to costs incurred in connection with the separation of former officers.

Cash and cash equivalents as of December 31, 2017 was $57.7 million. Pfenex believes it has sufficient cash to meet the Company’s anticipated cash needs for at least the next 12 months. The Company also believes it has sufficient cash resources to fund all necessary activities leading up to and including the submission of a new drug application (NDA) for PF708 to the FDA.

Cautionary Note Regarding Forward-Looking Statement –

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements generally relate to future events or Pfenex’s future financial or operating performance. In some cases, you can identify forward-looking statements because they contain words such as "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "target," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these words or other similar terms or expressions that concern Pfenex’s expectations, strategy, plans or intentions. Forward-looking statements in this press release include, but are not limited to, statements regarding the future potential of Pfenex’s product candidates and the company in general, including future plans to advance, develop, manufacture and commercialize its product candidates; Pfenex’s expectation to receive data from the PF708 clinical program in second quarter of 2018 and its expectation to submit an NDA in the third quarter of 2018, and the possibility of the potential commercial US launch of PF708 in the third quarter of 2019; Pfenex’s expectations with respect to the sufficiency of its cash resources; Pfenex’s expectations regarding the timing of the release of additional clinical trial data for its product candidates; Pfenex’s expectations regarding the timing and advancement of clinical trials and studies and the types of future clinical trials and studies for its product candidates, including PF708 and Px563L/RPA563; Pfenex’s expectations regarding the sufficiency of its clinical trials to satisfy regulatory requirements; Pfenex’s expectation for potential partnership opportunities for its product candidates; expectations with regard to future milestone and royalty payments from Pfenex’s collaboration with Jazz Pharmaceuticals; Pfenex’s expectations regarding potential payments from BARDA; Pfenex’s expectation that it will potentially initiate a phase 2 study for Px563L/RPA563 in 2019; and Pfenex’s future projections related to fluctuations and increases in revenue and expenses, including increases in research and development costs as Pfenex advances its product candidates. Pfenex’s expectations and beliefs regarding these matters may not materialize, and actual results in future periods are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Actual results may differ materially from those indicated by these forward-looking statements as a result of the uncertainties inherent in the clinical drug development process, including, without limitation, Pfenex’s ability to successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates or may require Pfenex to conduct additional clinical trials or modify ongoing clinical trials or regulatory pathways; challenges related to commencement, patient enrollment, completion, and analysis of clinical trials; difficulties in achieving and demonstrating biosimilarity in formulations; Pfenex’s ability to manage operating expenses; Pfenex’s ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives; Pfenex’s dependence on third parties for development, manufacture, marketing, sales and distribution of products; unexpected expenditures; and difficulties in obtaining and maintaining intellectual property protection for its product candidates. Information on these and additional risks, uncertainties, and other information affecting Pfenex’s business and operating results is contained in Pfenex’s Annual Report on Form 10-K for the period ended December 31, 2017 and in its other filings with the Securities and Exchange Commission. The forward-looking statements in this press release are based on information available to Pfenex as of the date hereof, and Pfenex disclaims any obligation to update any forward-looking statements, except as required by law.

Pfenex investors and others should note that we announce material information to the public about the Company through a variety of means, including our website (View Source), our investor relations website (View Source), press releases, SEC filings, public conference calls, corporate Twitter account (View Source), Facebook page (View Source), and LinkedIn page (View Source) in order to achieve broad, non-exclusionary distribution of information to the public and to comply with our disclosure obligations under Regulation FD. We encourage our investors and others to monitor and review the information we make public in these locations as such information could be deemed to be material information. Please note that this list may be updated from time to time.