On May 8, 2018 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the first quarter ended March 31, 2018 (Press release, Syndax, MAY 8, 2018, View Source [SID1234526234]). In addition, the Company provided a clinical and business update. As of March 31, 2018, Syndax had $113.2 million in cash, cash equivalents and short-term investments.

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"Syndax is off to a strong start in 2018 and we believe this momentum will carry us through the balance of what we expect will be a milestone-rich and potentially transformative year. This includes the progression free survival readout from our ongoing pivotal Phase 3 E2112 trial, for which results are expected in the third quarter," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We also look forward to sharing additional data from multiple cohorts of the ENCORE 601 program, including biomarker analyses, later this quarter at the ASCO (Free ASCO Whitepaper) Annual Meeting. The ENCORE program represents a key pillar of our clinical strategy and is supported by an extensive correlative science program designed to identify biomarkers that could predict which patients will respond to our combination therapies."

Pipeline Updates

The Phase 3 registration trial of entinostat plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer, E2112, is 92% enrolled as of the end of April. ECOG-ACRIN Cancer Research Group, the trial sponsor, has notified the Company that the Data Safety Monitoring Committee (DSMC) completed the final progression free survival (PFS) analysis and the first interim analysis for overall survival in November 2017. Earlier this quarter, the DSMC also notified Syndax that it conducted a subsequent interim overall survival analysis. The trial is proceeding as planned, and Syndax continues to anticipate that enrollment will be complete in the third quarter of 2018, at which time the result of the PFS analysis will be released to the Company.

Enrollment in the PD-(L)1 refractory melanoma ENCORE 601 cohort is now complete. The Company will present Phase 2 data from all evaluable patients at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting in June. Later this quarter, the Company plans to communicate a registration strategy for entinostat in this indication.

Enrollment in the PD-(L)1 refractory non-small cell lung cancer (NSCLC) ENCORE 601 cohort is now complete. Phase 2 data from all evaluable patients in this cohort will be presented at ASCO (Free ASCO Whitepaper) next month, including updated results from the Company’s biomarker analyses.

Initial enrollment in the first stage of the ENCORE 601 cohort of patients with microsatellite stable colorectal cancer (MSS-CRC) completed in the third quarter of 2017. The Company expects to share preliminary data from this cohort at ASCO (Free ASCO Whitepaper). The ENCORE 601 trial is being conducted in collaboration with Merck, through a subsidiary. The two companies recently agreed to expand this cohort, and expect to continue enrolling patients to the first stage later this quarter. A decision on whether to continue to the second stage of this cohort is expected in the first half of 2019.

Enrollment of the Phase 2 portion of ENCORE 602, the Phase 1b/2 clinical trial evaluating the combination of entinostat plus Genentech’s PD-(L)1 inhibitor atezolizumab (TECENTRIQ) in patients with triple negative breast cancer, remains on track to complete later this quarter. Topline results are now anticipated in the first half of 2019.

Enrollment is now complete in the Phase 2 portion of ENCORE 603, the Phase 1b/2 clinical trial evaluating entinostat in combination with Pfizer/Merck KGaA’s BAVENCIO in patients with ovarian cancer. Topline results are expected in the first half of 2019.

Dosing of patients with solid tumors in the Phase 1 multiple ascending dose (MAD) clinical trial of SNDX-6352 is ongoing. The Company anticipates presenting data from this trial and disclosing a Phase 2 strategy in the second half of 2018. In February, the Company entered into a clinical collaboration with AstraZeneca to evaluate the efficacy and safety of SNDX-6352 in combination with durvalumab (IMFINZI), AstraZeneca’s human monoclonal antibody directed against PD-(L)1, in multiple solid tumors. Initial work focusing on establishing the safety of this combination is expected to begin this quarter.

Development of the Company’s portfolio of Menin-Mixed Lineage Leukemia (MLL) inhibitors, in-licensed from Vitae Pharmaceuticals, Inc., a subsidiary of Allergan plc, is ongoing. Data from this program were recently presented in both oral and poster presentations at the 2018 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The Company expects to initiate clinical trials for this program in the first half of 2019.

First Quarter 2018 Financial Results

As of March 31, 2018, Syndax had cash, cash equivalents and short-term investments of $113.2 million and 24,697,944 shares issued and outstanding.

First quarter 2018 research and development expenses increased to $15.3 million from $9.6 million for the comparable period in the prior year. The increases were primarily due to an increase in development activities of $2.8 million, legal and professional fees of $1.7 million and increased employee compensation expense of $1.3 million. The increase in development activities was primarily due to increases in spending related to the increased CMC costs for SNDX-6352, and development of the Menin program, partially offset by completion of the Phase 1 clinical pharmacology trials and decrease in E2112 costs. The increase in employee compensation costs was primarily due to increased headcount.

General and administrative expenses totaled $4.8 million during the first quarter of 2018, compared to $3.9 million for the comparable period in the prior year. The increase in general and administrative expenses was primarily due to an increase in professional fees of $0.7 million as well as an increase in legal fees of $0.2 million. The increase in professional fees was primarily due to pre-commercialization activities. The increase in legal fees was primarily due to an increase in patent-related legal expenses.

For the three months ended March 31, 2018, Syndax reported a net loss attributable to common stockholders of $19.4 million or $0.79 per share compared to $13.0 million or $0.71 per share for the comparable prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the second quarter and full year 2018. For the second quarter and full year 2018, research and development expenses are expected to be $15 to $18 million and $62 to $70 million, respectively, and total operating expenses are expected to be $20 to $23 million and $82 to $90 million, respectively.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Tuesday, May 8, 2018.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 7087078
Domestic Dial-in Number: 1-855-251-6663
International Dial-in Number: 281-542-4259
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

On May 8, 2018 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI), a clinical-stage biotechnology company developing IPI-549, a first-in-class immuno-oncology product candidate that selectively inhibits phosphoinositide-3-kinase gamma (PI3K-gamma), reported that data from the ongoing clinical trial of IPI-549 will be presented in three events on June 4th during the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Infinity Pharmaceuticals, MAY 8, 2018, View Source [SID1234526233]).

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Investor Event 6:30 a.m. CT – 7:30 a.m. CT
Infinity Pharmaceuticals will host an investor event on Monday, June 4, 2018, from 6:30 a.m. CT – 7:30 a.m. CT, with Dr. Ryan Sullivan from Massachusetts General Hospital, an investigator on the IPI-549 Phase 1/1b study, to review the IPI-549 data being presented at ASCO (Free ASCO Whitepaper). The event will be webcast live and can be accessed on the Investors/Media section of Infinity’s website at www.infi.com for 30 days following the event.

Poster Session 8:00 a.m. CT – 11:00 a.m. CT
Title: Initial results from first-in-human study of IPI-549, a tumor macrophage-targeting agent, combined with nivolumab in advanced solid tumors.
Session Title: Developmental Therapeutics – Immunotherapy
Session Date and Time: Monday, June 4, 2018, 8:00 a.m. CT – 11:00 a.m. CT
Poster Board: 227
Abstract Number: 3013
First Author:Ryan J. Sullivan, MD, Massachusetts General Hospital
Location: Hall A, McCormick Place convention center

Poster Discussion Session 11:30 a.m. CT – 12:45 p.m. CT
Title: Initial results from first-in-human study of IPI-549, a tumor macrophage-targeting agent, combined with nivolumab in advanced solid tumors.
Session Title: Developmental Therapeutics – Immunotherapy
Session Date and Time: Monday, June 4, 2018, 11:30 a.m. CT – 12:45 p.m. CT
Abstract Number: 3013
First Author:Ryan J. Sullivan, MD, Massachusetts General Hospital
Location: Hall B1, McCormick Place convention center

About IPI-549 and the Ongoing Phase 1/1b 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 demonstrated the ability to reprogram macrophages from a pro-tumor (M2), immune suppressive function, to an anti-tumor (M1) immune activating function and enhance the activity of, and overcome resistance to, checkpoint inhibitors.[i], [ii] 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 1/1b 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.[iii] The study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The monotherapy dose-escalation and expansion components are complete. 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 cancer 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 May 8, 2018 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its first quarter 2018 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 macrophages (Press release, Infinity Pharmaceuticals, MAY 8, 2018, View Source [SID1234526232]). 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 continue to make important progress with IPI-549 and look forward to reporting clinical and translational data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June from the combination dose escalation portion of the trial, in addition to providing an update on the monotherapy portion of the trial and initial data from combination expansion cohorts initiated in early 2018," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "We expect that these data will provide additional insights into the activity, durability, safety and mechanism of action of IPI -549, including in combination with nivolumab."

Recent developments include the following:

IPI-549

Continued Progress with the Phase 1/1b Study: The monotherapy portions of the study and combination dose escalation portion of the study have been completed. Enrollment, which began in 2018, is ongoing for six disease-specific combination expansion cohorts, along with a seventh combination expansion cohort of patients pre-selected for having high baseline blood levels of myeloid derived suppressor cells (MDSCs).
2018 Program Goals for IPI-549

At ASCO (Free ASCO Whitepaper) report:
Data from the monotherapy expansion component of the study
Data from the combination dose-escalation component of the study
Initial data from six disease-specific combination expansion cohorts
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
First Quarter 2018 Financial Results

At March 31, 2018, Infinity had total cash, cash equivalents and available-for-sale securities of $47.8 million, compared to $57.6 million at December 31, 2017.
R&D expense for the first quarter of 2018 was $5.9 million, compared to $4.0 million for the same period in 2017. The increase in R&D expense was primarily due to higher clinical development expenses for IPI-549.
General and administrative expense was $3.6 million for the first quarter 2018, compared to $6.4 million for the same period in 2017. The decrease in G&A expense was primarily due to a reduction in bonus and stock compensation.
Net loss for the first quarter of 2018 was $9.5 million, or a basic and diluted loss per common share of $0.18, compared to a net loss of $10.5 million, or a basic and diluted loss per common share of $0.21 for the same period in 2017.
Financial Outlook
Infinity’s 2018 financial guidance remains unchanged:

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 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 its New Drug Application for duvelisib was accepted by the U.S. Food and Drug Administration (FDA) and that it was given priority review with an FDA action date of October 5, 2018. With the potential Verastem payment, Infinity expects that its cash runway would extend into 2020.
Conference Call Information
Infinity will host a conference call today, May 8, 2018, at 4:30 p.m. ET 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) or 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 2479309. An archived version of the webcast will be available on Infinity’s website for 30 days.

About IPI-549 and the Ongoing Phase 1/1b 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 demonstrated the ability to reprogram macrophages from a pro-tumor (M2), immune suppressive function, to an anti-tumor (M1) immune activating function and enhance the activity of, and overcome resistance to, checkpoint inhibitors.1, 2 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 1/1b 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.3 The study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The monotherapy dose-escalation and expansion components are complete. 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 cancer 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 May 8, 2018 SCYNEXIS, Inc. (NASDAQ:SCYX), a biotechnology company delivering innovative anti-infective therapies for difficult-to-treat and often life-threatening infections, reported financial results for the quarter ended March 31, 2018, and provided an update on recent operational and clinical developments (Press release, Scynexis, MAY 8, 2018, View Source [SID1234526231]).

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"The first quarter of 2018 was one of significant clinical progress in what has the potential to be a transformative year for SCYNEXIS with multiple anticipated milestones," said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. "We remain on track to report top-line data from the Phase 2b, dose-finding study evaluating oral SCY-078 for the treatment of vulvovaginal candidiasis (VVC) by July 2018, with Phase 3 initiation planned for the fourth quarter. We also anticipate continued progress toward expanding the therapeutic utility of SCY-078 with the planned initiation of clinical trials in invasive candidiasis and invasive aspergillosis in the second half of the year, as well as the advancement of our programs targeting refractory invasive fungal infections. We are well-positioned to realize the promise of SCY-078 as a potent and versatile antifungal agent."

Clinical and Regulatory Update for Lead Program – SCY-078 as a Treatment for VVC

In May 2018, SCYNEXIS announced it has completed enrollment in the Phase 2b, dose-finding study of oral SCY-078 for the treatment of VVC (the DOVE study). SCYNEXIS is on-track to announce top-line data by July 2018. Following successful dose-identification from this trial, SCYNEXIS plans to initiate a Phase 3 registration program of oral SCY-078 in VVC in the fourth quarter of 2018, with potential NDA filing for VVC expected in 2020.
In May 2018, SCYNEXIS announced the receipt of Qualified Infectious Disease Product (QIDP) and Fast Track designations from the U.S. Food and Drug Administration (FDA) for the treatment of VVC and prevention of recurrent VVC. The QIDP designation allows SCYNEXIS to have priority review and an additional five years of market exclusivity in the U.S. for SCY-078. The FDA’s Fast Track Drug Development Program is a process designed to facilitate the development and expeditious review of drugs to treat serious conditions and fill unmet medical needs.
Continued Advancement of IV SCY-078 Program with Liposomal Formulation

Pre-clinical work on the liposomal IV formulation of SCY-078 continues, and SCYNEXIS remains on track to initiate a Phase 1 trial evaluating the safety and tolerability of this formulation in healthy volunteers in the third quarter of 2018.
If successful, following completion of the Phase 1 study and pending FDA review, SCYNEXIS plans to initiate a Phase 2b, IV-oral step-down study of SCY-078 in invasive candidiasis patients with the liposomal IV and oral formulations of SCY-078 in the fourth quarter of 2018.
Pre-clinical Data Support Continued Development of SCY-078 for Invasive Fungal Infections

In April 2018, at the 28thEuropean Congress of Clinical Microbiology and Infectious Disease (ECCMID), SCYNEXIS presented pre-clinical data demonstrating SCY-078’s potent antifungal activity against Aspergillus spp., including azole-resistant isolates, as well as synergistic activity with approved antifungals against Aspergillus strains. Additionally, SCYNEXIS presented in vivo data in a mouse model of Pneumocystis pneumonia that demonstrated the activity of SCY-078 as determined by improved survival and reduction of fungal burden, supporting future development for prophylaxis indications.
In March 2018, at Superbugs and Superdrugs 2018, SCYNEXIS presented pre-clinical data demonstrating the inhibitory effect of SCY-078 against Candida auris biofilms, as well as SCY-078’s ability to affect the ultrastructure of C. auris cells and interrupt cell division.
In February 2018, at the 8th Advances Against Aspergillosis, SCYNEXIS presented new pre-clinical data demonstrating synergistic in vivo activity and improved outcomes of SCY-078 in combination with isavuconazole for the treatment of invasive pulmonary aspergillosis. The Company plans to initiate a Phase 2 study in the third quarter of 2018 to test the clinical efficacy of oral SCY-078 in combination with standard of care for the treatment of invasive aspergillosis.
All posters and presentations are available on the Scientific Publications page of the SCYNEXIS website.
Corporate Update

In April 2018, SCYNEXIS received confirmation of the renewal of its Small and Medium Enterprise (SME) designation by the European Medicines Agency (EMA). With this designation, SCYNEXIS is eligible to receive financial incentives, regulatory fee reductions and waivers, and European Union funding. SCYNEXIS remains committed to the European market and has designed its registration programs such that concurrent FDA and EMA approvals would be possible. European sites are actively enrolling in the FURI study, a global, open-label study, designed to evaluate oral SCY-078 for the treatment of fungal infections that are refractory to or intolerant of standard therapies.
On March 8, 2018, SCYNEXIS raised $30.0 million in gross proceeds by issuing 17,751,500 shares of the Company’s common stock and two series of warrants to purchase up to an aggregate of 21,301,800 shares of the Company’s common stock. The offering resulted in approximately $27.9 million of net proceeds after deducting the underwriting discount and estimated offering expenses.
First Quarter 2018 Financial Results

Cash and cash equivalents and short-term investments totaled $63.7 million as of March 31, 2018, with net working capital of $53.5 million.

Research and development expenses increased to $5.3 million in the first quarter of 2018, compared to $4.0 million in the first quarter of 2017. The increase of $1.3 million, or 33%, was primarily driven by an increase of $0.7 million in pre-clinical development expense, a $0.7 million increase in clinical development expense, a $0.4 million increase in chemistry, manufacturing, and controls (CMC), and a net increase of $0.2 million in other research and development costs; offset by a decrease in consulting expense of $0.7 million.

Selling, general and administrative expenses of $2.0 million in the first quarter of 2018 were consistent compared to the selling, general and administrative expenses of $2.1 million in the first quarter of 2017.

Total other income increased to $3.2 million in the first quarter of 2018 due to a $3.6 million non-cash gain recorded on the fair value adjustment of the warrant liabilities.

Net loss for the first quarter of 2018 was $4.0 million, or $0.12 basic net loss per share. This compares to a net loss for the first quarter of 2017 of $4.9 million, or $0.19 basic net loss per share.

On May 8, 2018 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2018 second quarter ended March 31, 2018 (Press release, Arrowhead Research Corporation, MAY 8, 2018, View Source [SID1234526230]). The company is hosting a conference call at 4:30 p.m. EDT to discuss results.

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 2895628.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 2895628.

Selected Fiscal 2018 Second Quarter and Recent Events

Strengthened the balance sheet with an equity financing yielding gross proceeds of $60.4 million

Received orphan drug designation from the United States Food and Drug Administration (FDA) for ARO-AAT, Arrowhead’s second-generation investigational medicine for the treatment of a rare genetic liver disease associated with alpha-1 antitrypsin deficiency

Initiated dosing in AROAAT1001 (NCT03362242), a Phase 1 single- and multiple-ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and effect of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers

Initiated dosing in AROHBV1001 (NCT03365947), a Phase 1/2 study to evaluate the safety, tolerability, and pharmacokinetic effects of single-ascending doses (SAD) of ARO-HBV in healthy adult volunteers, and to evaluate the safety, tolerability, and pharmacodynamic effects of multiple-ascending doses (MAD) of ARO-HBV in patients with chronic HBV

Presented clinical data on ARC-520, the company’s prior generation investigational medicine for the treatment of chronic hepatitis B infection, at The International Liver Congress 2018 (ILC), the annual meeting of the European Association for the Study of the Liver (EASL), including the following key results:

Multiple doses of ARC-520 resulted in s-antigen reductions in all patients by as much as 5.3 Log10

Where measurable, multi-log reductions were also seen in e-antigen, core-related antigen, DNA and HBV RNA

One e-antigen negative patient, while remaining on entecavir, serocleared for all measurable viral markers including s-antigen, core-related antigen, HBV RNA, and HBV DNA. We believe this will represent a functional cure

2 out of 3 e-antigen positive and 2 out of 5 e-antigen negative patients, or half of the patients in the study, achieved productive and sustained host responses. These were characterized by mild ALT elevations coinciding with continued reductions in various viral markers which persisted after ARC-520 therapy was removed

Two patients that experienced sustained host responses but had not yet serocleared, appear poised to potentially seroclear if the trends in the decrease of viral markers continues

Presented preclinical data on both ARO-AAT and ARO-HBV at EASL

Made continued progress on the emerging pipeline of RNAi therapeutics developed using the Targeted RNAi Molecule (TRiMTM) platform including:

The cardiometabolic pipeline, which includes ARO-APOC3, targeting apolipoprotein C-III (ApoC3), and ARO-ANG3, targeting angiopoietin-like protein 3 (ANGPTL3)

The pulmonary pipeline, which includes ARO-ENaC, formerly called ARO-Lung1, which is an inhaled RNAi therapeutic targeting the epithelial sodium channel alpha subunit (aENaC) for the treatment of cystic fibrosis

ARO-HIF2 for the treatment of clear cell renal cell carcinoma