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.

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 MabVax Therapeutics Holdings, Inc. (NASDAQ: MBVX), a clinical-stage oncology drug development company, reported that it will present three posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting being held April 14-18, 2018 in Chicago, Illinois at McCormick Place (Press release, MabVax, MAR 15, 2018, View Source [SID1234524812]). The first poster session features MVT-1075 (177Lu-CHX-A″-DTPA-HuMab5B1), the Company’s novel fully human antibody-based radioimmunotherapy (RIT) currently being evaluated in clinical development for the treatment of pancreatic cancer and other CA19-9 positive malignancies. The second presentation will feature preclinical investigations on the novel fully human antibody targeting the Thomsen-nouveau (Tn) and the sialyl Tn (sTn) cancer antigens which are highly overexpressed on ovarian and breast cancer tissues. The third poster features the Company’s immunoPET imaging agent MVT-2163 (89Zr-DFO-HuMab5B1), as a companion diagnostic for use in pancreatic cancer and CA19-9 positive malignancies.

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MabVax Therapeutics Logo (PRNewsfoto/MabVax Therapeutics Holdings, I)

Paul Maffuid, Ph.D., Executive Vice President of Research and Development of MabVax, stated, "We look forward to sharing the significant progress we have made through these clinical and preclinical studies that continue to establish our growing body of data supporting the development of MVT-1075 for the treatment of pancreatic cancer and other CA19-9 cancers, and our most advanced research program focused on the Tn and sTn cancer antigens."

Dr. Maffuid continued, "MVT-1075 potentially represents a more potent analog of our fully human HuMab-5B1 therapeutic antibody and establishing these safety and early response data bring us an important step closer in providing a much-needed treatment option for patients who have these devastating cancers. Equally important, our anti-Tn/sTn antibody program has made significant progress over the last few months and these data are the subject of partnering interest."

MabVax Abstracts and Poster Presentations

Sunday April 15, 2018 from 1:00 PM – 5:00 PM CDT:
Title: A fully human antibody binds Tn and sTn carbohydrate antigens specifically on serine residues, without need for polypeptide interaction
Session Location: Poster Section 43
Abstract Number: LB-002, Poster Board Number 2
Presenting Author: Jonah Rainey, Ph.D., Executive Director, Antibody Research MabVax Therapeutics

Tuesday April 17, 2018 from 8:00 AM – 12:00 PM CDT:
Title: Phase I dose escalation study of 177Lu-HuMab-5B1 (MVT-1075) in combination with MVT-5873 as radioimmunotherapy (RIT) in subjects with relapsed / refractory pancreatic cancer or other CA19-9+ malignancies
Session Location: McCormick Place South, Hall A, Poster Section 42
Abstract Number: CT140, Poster Board Number 23
Presenting Author: Paul Maffuid, Ph.D., Executive Vice President, Research & Development MabVax Therapeutics

Tuesday April 17, 2018 from 8:00 AM – 12:00 PM CDT:
Title: PEGylated Hyaluronidase Increases Tumor Uptake of 89Zr-DFO-HuMab-5B1 (MVT-2163) in a CA19-9 Positive Hyaluronan-Accumulating Pancreatic Cancer Model
Session Location: McCormick Place South, Hall A, Poster Section 1
Abstract Number: 3036, Poster Board Number 9
Co-presenting Authors: Paul Maffuid, Ph.D., Executive Vice President, Research & Development and Jonah Rainey, MabVax Therapeutics Executive Director, Antibody Research MabVax Therapeutics

About MVT-1075

MVT-1075 is a radioimmunotherapy product that combines established efficacy of radiation therapy with tumor specific targeting. It has the potential to deliver a more potent HuMab-5B1 based product. MVT-1075 uses small doses of the Company’s MVT-5873 antibody, coupled to a radioisotope to target pancreatic cancer cells and kill them.

About the HuMab-Tn Antibody Targeting Tn and sTn

HuMab-Tn is a fully human antibody derived from a patient vaccinated with a pool of cancer glycans, including Tn. The antibody has been affinity-matured and demonstrates highly selective Tn/sTn glycan binding. Further, the antibody recognizes a wide array of cancers, particularly ovarian and breast including approximately 90% of triple negative breast cancers tested.

About MVT-2163

MVT-1075 is an immunoPET imaging agent product that combines the established PET imaging capabilities of 89Zr with 5B1 tumor specific targeting. It has the potential to aid in identifying the best surgical treatment options for patients with pancreatic cancer and as a potential companion diagnostic with treatment options.

On March 15, 2017 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its fourth quarter 2017 financial results and provided an update on the company, including its progress with IPI-549, a first-in-class oral immuno-oncology product candidate that selectively inhibits phosphoinositide-3-kinase-gamma (PI3K-gamma) and targets immune-suppressive tumor microphages (Press release, Infinity Pharmaceuticals, MAR 15, 2018, View Source [SID1234524810]). Infinity is evaluating IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in a Phase 1/1b study in approximately 200 patients with advanced solid tumors.

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"We made significant progress with IPI-549 over the past year, and 2018 will be an important year for us as we report data from the monotherapy expansion and combination dose escalation components of our study and from seven combination expansion cohorts, which will help define our development and regulatory strategy for this first-in-class product candidate. Based on our initial clinical data, IPI-549 has a favorable tolerability profile and has demonstrated clinical and biological activity, both as a monotherapy and in combination with nivolumab. We believe that IPI-549 has the potential to increase the number of patients who respond to immunotherapies as well as to increase the duration of those responses," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "In addition, we’ve continued to position Infinity for success by expanding our board and clinical leadership team and establishing a scientific advisory board with thought leaders in the field of immuno-oncology."

Recent developments include the following:

IPI-549

Advanced and expanded clinical development of IPI-549: The Phase 1/1b monotherapy and combination dose escalation components of the study have been completed, and the monotherapy expansion component has been fully enrolled. In addition, six disease-specific combination expansion cohorts are enrolling at the recommended Phase 2 dose of 40 mg once daily of IPI-549 plus Opdivo (nivolumab) at 240 mg every two weeks in patients with non-small cell lung cancer, melanoma, head and neck cancer, triple-negative breast cancer, mesothelioma, and adrenocortical carcinoma. An additional combination expansion cohort of patients pre-selected for having high baseline blood levels of myeloid derived suppressor cells (MDSCs) is expected to open for enrollment in the next few weeks. Studies have shown that poor response to checkpoint inhibitor therapy is correlated with the presence of high baseline blood levels of MDSCs in cancer patients.1,2,3 Preliminary translational data from the study demonstrated an association between high baseline blood levels of MDSCs and clinical responses. Preselecting patients with high blood MDSCs could lead to improved clinical activity for patients treated with the combination of IPI-549 and anti-PD1.
Presented Phase 1/1b clinical and translational data at SITC (Free SITC Whitepaper): In November, Infinity announced updated data from the monotherapy dose-escalation component of the Phase 1/1b study of IPI-549 in a late-breaking presentation at the SITC (Free SITC Whitepaper) Annual Meeting 2017. These data demonstrated that IPI-549 dosed once daily was well tolerated and clinically active. Among 18 patients evaluable for activity, there was a 44 percent clinical benefit rate, defined as patients who had remained on treatment for at least 16 weeks, including one partial response in a patient with advanced peritoneal mesothelioma. Initial translational data from patient blood samples demonstrated that IPI-549 treatment results in a reduction in immune suppression and increased immune stimulation, with upregulation of interferon-gamma responsive factors and reinvigoration/proliferation of exhausted T cells across multiple tumor types and dose levels. Additionally, those patients who showed a clinical benefit had increased numbers of activated monocytes, suggesting a biologic correlate can be identified in patients who remain on treatment longer.
Corporate

David Beier appointed to Board of Directors: David Beier, J.D., is a Managing Director of Bay City Capital and brings a wealth of experience to the Infinity Board. David serves as an advisor to the Parker Institute for Cancer Immunotherapy, as a Senior Fellow at the USC Schaeffer Center for Health Policy & Economics and as a member of the Board of Directors of Arcus Biosciences, UCSF Benioff Children’s Hospitals and the California Life Sciences Association. He also serves as an appointee of Governor Brown on the California State Government Organization and the Economy Commission. Having spent two decades as part of the senior management teams for Amgen and Genentech, he brings invaluable perspective regarding strategy for entrepreneurial biotechnology firms and the industry in general as a globally recognized leader in health care policy, regulatory affairs, healthcare economics, and pricing. Mr. Beier also previously served in the White House during the Clinton Administration as a Chief Domestic Policy Advisor to the Vice President.
Clinical leadership team expanded: Marie-Louise Fjällskog M.D., Ph.D., has been appointed as Vice President of Clinical Development and will play an integral role in the expanded clinical development of IPI-549. Dr. Fjällskog is an Associate Professor of Oncology at Uppsala University, Sweden, and has over twenty-five years of experience in clinical oncology, translational research, and drug development. She joins Infinity from the Novartis Institute for Biomedical Research, where she served as a Clinical Program Leader, Translational Clinical Oncology, and as the global lead for several immuno-oncology programs, including those targeting CSF-1 and PD-1.

In addition, Suresh Mahabhashyam, M.D., M.P.H., was promoted to Vice President of Safety and Risk Management. Dr. Mahabhashyam has over 20 years of experience in medical practice and epidemiology with the last decade focused on drug development at Infinity, Alexion and Allergan.
Scientific Advisory Board established with four thought leaders in immuno-oncology:
Dmitry Gabrilovich, M.D., PhD., a leader in myeloid cell biology and the Christopher M. Davis Professor in Cancer Research and Program Leader, Immunology, Microenvironment, and Metastasis at the Wistar Institute in Philadelphia and Wistar Professor at the Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania;
Roy Herbst, M.D., Ph.D., a leader in lung cancer treatment and research and the Ensign Professor of Medicine (Medical Oncology) and Professor of Pharmacology; Chief of Medical Oncology, Yale Cancer Center and Smilow Cancer Hospital;
Stephen Hodi, M.D., a leader in developing immune therapy and melanoma therapeutics and the Director of the Melanoma Center and the Center for Immuno-Oncology at Dana-Farber/Brigham and Women’s Cancer Center, the Sharon Crowley Martin Chair in Melanoma at Dana-Farber Cancer Institute and Professor of Medicine at Harvard Medical School; and
David H. Munn, M.D., a pioneer in T cell activation and indoleamine 2,3-dioxygenase (IDO) research and a Professor of Pediatric Hematology-Oncology at the Medical College of Georgia, Augusta University.
2018 Program Goals for IPI-549

Report data from the monotherapy expansion component of the study in the second quarter of 2018
Report data from the combination dose-escalation component of the study in the second quarter of 2018
Report initial data from six disease-specific combination expansion cohorts in the second quarter of 2018
Report more mature clinical and translational data (including insights from paired tumor biopsies) from the six disease-specific cohorts and initial data from the cohort of patients pre-selected for having high baseline blood levels of myeloid derived suppressor cells (MDSCs) in the combination expansion component of the study in the second half of 2018
Full Year 2017 Financial Results

At December 31, 2017, Infinity had total cash, cash equivalents and available-for-sale securities of $57.6 million, compared to $92.1 million at December 31, 2016.
Revenue during 2017 was $6.0 million, all of which related to the amount received from Verastem for the DUO study meeting the pre-specified criteria at completion. Revenue during 2016 was $18.7 million related to Infinity’s previous collaboration agreement with AbbVie Inc.
R&D expense for 2017 was $20.8 million, compared to $119.6 million for 2016. The decrease in R&D expense was primarily related to the company’s 2016 restructuring activities and out-licensing of duvelisib to Verastem.
General and administrative expense was $21.6 million for 2017, compared to $42.2 million for 2016. The decrease in G&A expense was primarily due to the company’s 2016 restructuring activities.
Net loss for 2017 was $41.8 million, or a basic and diluted loss per common share of $0.83, compared to a net loss of $30.1 million, or a basic and diluted loss per common share of $0.61 for 2016.
Financial Outlook
Infinity’s updated 2018 financial guidance is as follows:

Net Loss: Infinity expects net loss for 2018 to range from $35 million to $45 million.
Cash and Investments: Infinity expects to end 2018 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $15 million to $25 million.
Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities will be adequate to satisfy the company’s capital needs into the third quarter of 2019. Infinity’s financial guidance excludes additional funding or business development activities and does not include the potential $22 million payment from Verastem upon the first regulatory approval of duvelisib, or a potential $2 million milestone payment from PellePharm, a private company, upon initiation of a Phase 3 study for the hedgehog inhibitor program, which Infinity licensed to PellePharm in 2013. Verastem announced that it submitted a New Drug Application for duvelisib to the U.S. Food and Drug Administration on February 7, 2018. With the potential Verastem milestone payment, Infinity expects to extend its cash runway into 2020.
Conference Call Information
Infinity will host a conference call today, March 15, 2018, at 4:30pm EDT to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial 1-877-316-5293 (domestic) and 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 5558799. An archived version of the webcast will be available on Infinity’s website for 30 days.

About IPI-549 and the Ongoing Phase 1b Study
IPI-549 is an investigational first-in-class, oral, immuno-oncology product candidate targeting tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition, thereby reducing pro-tumor macrophage function and increasing anti-tumor macrophage function. In preclinical studies, IPI-549 reprograms macrophages from a pro-tumor (M2), immune suppressive function, to an anti-tumor (M1) immune activating function and can enhance the activity of, and overcome resistance to, checkpoint inhibitors. 4,5 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially additive or synergistic approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

The ongoing Phase 1b study being conducted by Infinity is designed to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo in approximately 200 patients with advanced solid tumors.6 The study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The monotherapy dose-escalation component is complete, and the monotherapy expansion component has been fully enrolled. The combination dose-escalation component is also complete, and combination expansion cohorts are enrolling.

The combination expansion component of the study includes multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma and head and neck squamous cell carcinoma (HNSCC) whose tumors show initial resistance or initially respond to but subsequently develop resistance to immune checkpoint blockade therapy. The combination expansion component also includes a cohort of patients with triple negative breast cancer (TNBC) who have not been previously treated with immune checkpoint blockade therapy, a cohort of patients with mesothelioma, a cohort of patients with adrenocortical carcinoma and a cohort of patients with high baseline blood levels of MDSCs.

IPI-549 is an investigational compound and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On March 15, 2018 Incyte Corporation (Nasdaq:INCY) reported that 15 abstracts from its research and development portfolio will be presented at the upcoming 2018 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in Chicago, Illinois from April 14-18, 2018 (Press release, Incyte, MAR 15, 2018, View Source;p=RssLanding&cat=news&id=2338255 [SID1234524809]). These abstracts include the first pre-clinical data from the Company’s recently announced AXL/MER, TIM-3 and LAG-3 antibody programs in patients with advanced solid tumors.

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"We are excited to present a broad collection of abstracts from our robust development portfolio at this year’s AACR (Free AACR Whitepaper) Annual Meeting," said Reid Huber, Ph.D., Incyte’s Chief Scientific Officer. "The acceptance of these abstracts and their results represent the progress Incyte continues to make in the pursuit of R&D excellence and ultimately innovation in the treatment of cancer and other serious diseases."

Key abstracts include:

Immuno-Therapy

Combination of a T Cell Activating Immunotherapy with Immune Modulators Alters the Tumor Microenvironment and Promotes More Effective Tumor Control in Preclinical Models (Abstract #1761, Immunology – Modifiers of the Tumor Microenvironment 2)

Monday, April 16, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 33
Identification of p-AKT as a PD marker for MER Kinase in Human G361 Cells (Abstract #2367, Molecular and Cellular Biology/Genetics – Kinases and Phosphatases)

Monday, April 16, 2018, 1:00 p.m. – 5:00 p.m. CDT, Chicago, Poster Section 17
Characterization of INCB081776, a Potent and Selective Dual AXL/MER Kinase Inhibitor (Abstract #3759, Immunology – Immunomodulatory Agents and Interventions 1)

Tuesday, April 17, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 32
INCAGN02390, a Novel Antagonist Antibody that Targets the Co-Inhibitory Receptor TIM-3 (Abstract #3825, Immunology – Therapeutic Antibodies, Including Engineered Antibodies 3)

Tuesday, April 17, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 34
INCAGN02385 is an Antagonist Antibody Targeting the Co-Inhibitory Receptor LAG-3 for the Treatment of Human Malignancies (Abstract #3819, Immunology – Therapeutic Antibodies, Including Engineered Antibodies 3)

Tuesday, April 17, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 34
Epacadostat plus Durvalumab in Patients with Advanced Solid Tumors: Preliminary Results of the Ongoing, Open-Label, Phase 1/2 ECHO-203 Study (Abstract #CT177, Session CTMS03 – Biomarkers in Immuno-Oncology [minisymposium])

Tuesday, April 17, 2018, 2:45 p.m. – 5:00 p.m. CDT, Chicago, N Hall C – McCormick Place North (Level 1)
Targeted Therapy

INCB052793, a JAK1 Selective Inhibitor, is Highly Efficacious in PDX and Xenograft Models of Acute Myeloid Leukemia (AML) Expressing Elevated Endogenous pSTAT3/pSTAT5 (Abstract #1867, Experimental and Molecular Therapeutics – Experimental Agents and Combinations for Hematologic Malignancies 2)

Monday, April 16, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 38
The Evaluation of INCB059872, an FAD-directed Inhibitor of LSD1, in Preclinical Models of T-ALL (Abstract #1893, Experimental and Molecular Therapeutics – Experimental Agents and Combinations for Hematologic Malignancies 2)

Monday, April 16, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 38
The FAD-directed LSD1 Specific Inhibitor, INCB059872, is a Promising Epigenetic Agent for AML Therapy by Inducing Differentiation of Leukemic Stem/Progenitor Cells (Abstract #1888, Experimental and Molecular Therapeutics – Experimental Agents and Combinations for Hematologic Malignancies 2)

Monday, April 16, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 38
INCB059872, a Novel FAD-directed LSD1 Inhibitor, is Active in Prostate Cancer Models and Impacts Prostate Cancer Stem-like Cells (Abstract #1379, Molecular and Cellular Biology/Genetics – Epigenetic Therapy)

Monday, April 16, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 16
In Vivo Assessment of the Combination of the JAK1-Selective Inhibitor Itacitinib with First- and Second-Generation EGFR Inhibitors in Models of Non-Small Cell Lung Cancer (Abstract #2938, Experimental and Molecular Therapeutics – Novel Experimental Combinations)

Monday, April 16, 2018, 1:00 p.m. – 5:00 p.m. CDT, Chicago, Poster Section 41
The FAD-Directed LSD1 Specific Inhibitor, INCB059872, Inhibits Cell Migration and Metastasis by Suppressing Premetastatic Niche Formation in a Spontaneous Metastasis Mouse Model (Abstract #3929, Experimental and Molecular Therapeutics – Resistance and Biology)

Tuesday, April 17, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 39
The BET Inhibitor INCB057643 Suppresses ALDH Activity by Targeting the ALDH1A1 Super-Enhancer in High-Grade Serous Ovarian Cancer (Abstract #3685, Clinical Research – Molecular Classification of Tumors 1: Epigenetic Therapy, Functional and Molecular Imaging, and Tumor Heterogeneity)

Tuesday, April 17, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 28
Effect of JAK/STAT or PI3Kδ Plus PD-1 Inhibition on the Tumor Microenvironment: Biomarker Results From a Phase 1b Study in Patients with Advanced Solid Tumors (Abstract #CT176, Session CTMS03 – Biomarkers in Immuno-oncology [minisymposium])

Tuesday, April 17, 2018, 2:45 p.m. – 5:00 p.m. CDT, Chicago, N Hall C – McCormick Place North (Level 1)
Anti-Tumor Efficacy of INCB057643, a Novel BET Bromodomain Inhibitor, in Castration-Resistant Prostate Cancer as Single Agent and in Combination Therapy (Abstract #5793, Experimental and Molecular Therapeutics – Canonical Targets 2)

Wednesday, April 18, 2018, 8:00 a.m. – 12:00 p.m. CDT, Chicago, Poster Section 36
Full session details and data presentation listings for AACR (Free AACR Whitepaper) 2018 can be found at: View Source!/4562