On May 8, 2018 BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported financial results for the first quarter ended March 31, 2018 (Press release, BioCryst Pharmaceuticalsa, MAY 8, 2018, View Source [SID1234526199]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are off to a strong start in 2018 as we continue to make progress advancing all our HAE development programs and remain on track to read out top line data from APeX-2 in the first half of next year," said Jon P. Stonehouse, BioCryst’s President and Chief Executive Officer. "We remain excited about our proposed merger with Idera Pharmaceuticals, Inc., which we believe will create greater and more sustainable value for the benefit of our stockholders and our patients. We look forward to positive data readouts from Idera in early June, which would reinforce the value creation potential in combining our synergistic discovery engines and creating a more robust and diversified late-stage pipeline."

First Quarter 2018 Financial Results

For the three months ended March 31, 2018, total revenues were $4.0 million, compared to $9.4 million in the first quarter of 2017. The decrease in revenue was primarily associated with infrequent revenue events that occurred in 2017 that did not recur in 2018. Those 2017 events were the recognition of $4.1 million of royalty revenue from Japanese government stockpiling of RAPIACTA and a $2.0 million payment for the Canadian regulatory approval of RAPIVAB.

Research and Development ("R&D") expenses for the first quarter of 2018 increased to $18.4 million from $16.8 million in the first quarter of 2017, primarily due to additions in R&D personnel and increased spending on our hereditary angioedema ("HAE") and preclinical programs. These increases were partially offset by a decrease in the Company’s peramivir and galidesivir development spending in 2018.

General and administrative ("G&A") expenses for the first quarter of 2018 increased to $7.6 million, compared to $3.1 million in the first quarter of 2017. The increase was primarily due to approximately $4.7 million of merger-related costs associated with the Company’s pending merger with Idera Pharmaceuticals, Inc. ("Idera").

Interest expense was $2.2 million in the first quarter of 2018, compared to $2.1 million in the first quarter of 2017. Also, a $1.8 million mark-to-market loss on the Company’s foreign currency hedge was recognized in the first quarter of 2018, as compared to a $1.5 million mark-to-market loss in the first quarter of 2017. These changes result from periodic changes in the U.S. dollar/Japanese yen exchange rate.

Net loss for the first quarter of 2018 was $25.8 million, or $0.26 per share, compared to a net loss of $14.2 million, or $0.19 per share, for the first quarter 2017.

Cash, cash equivalents and investments totaled $137.5 million at March 31, 2018, and reflect a decrease from $159.0 million at December 31, 2017. Net operating cash use for the first quarter 2018 was $22.9 million.

Clinical Development Update & Outlook

On February 28, 2018, BioCryst announced the dosing of the first patient in APeX-S, a long-term safety trial evaluating two dosage strengths of BCX7353 administered orally once-daily as a preventive treatment in patients with HAE. APeX-S is an open label two-arm trial to evaluate the safety of two dose levels of BCX7353 (110 mg once daily and 150 mg once daily) over 48 weeks in patients with Type I and II HAE. The trial will enroll approximately 160 patients.

On March 15, 2018, BioCryst announced the dosing of the first patient into APeX-2, a Phase 3 clinical trial evaluating two dosage strengths of BCX7353 administered orally once-daily as a preventive treatment to reduce the frequency of attacks in patients with HAE. APeX-2 is a randomized, double-blind, placebo-controlled, three-arm trial testing two doses of BCX7353 (110 mg and 150 mg) for prevention of angioedema attacks. The trial is expected to enroll approximately 100 patients with Type I and II HAE in the United States, Canada and Europe. The primary efficacy endpoint of APeX-2 is the rate of angioedema attacks over 24 weeks of study drug administration.

Enrollment in both the 750 mg and 500 mg cohorts of the ZENITH-1 proof-of-concept Phase 2 clinical trial liquid formulation of BCX7353 for treatment of acute angioedema attacks in HAE have been completed, and the 250 mg cohort is enrolling. We expect to report top-line results from the first cohort in the second half of 2018.

On May 1, 2018, BioCryst announced that the European Medicines Agency ("EMA") has approved peramivir with the brand name of ALPIVABTM, a single intravenous infusion for the treatment of uncomplicated influenza in adults and children from the age of 2 years. The EMA approval of ALPIVAB under the centralized licensing procedure provides marketing authorization for all 28-member states of the European Union, Norway and Iceland.

BioCryst has a license agreement with Seqirus regarding peramivir. As previously disclosed, BioCryst and Seqirus are engaged in a formal dispute resolution process involving many items under the contract including, but not limited to, the EMA approval milestone of $5 million, which BioCryst maintains is due.

In April 2018, the Therapeutic Goods Administration approved RAPIVAB (peramivir injection), an intravenous treatment for acute influenza, for commercial sale in Australia.
Financial Outlook for 2018

Based upon development plans and awarded government contracts, on a stand-alone basis, BioCryst continues to expect its 2018 net operating cash use to be in the range of $67 to $90 million, and its 2018 operating expenses to be in the range of $85 to $110 million. With merger-related costs and the aggressive advancement of programs thus far, it is expected the Company will trend to the upper-end of both ranges. The Company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the Company’s stock, as well as by the vesting of the Company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst’s leadership team will host a conference call and webcast Tuesday, May 8, 2018 at 11:00 a.m. Eastern Time to discuss these financial results and recent corporate developments. To participate in the conference call, please dial 1-877-303-8027 (United States) or 1-760-536-5165 (International). No passcode is needed for the call. The webcast can be accessed live or in archived form in the "Investors" section of the Company’s website at www.BioCryst.com. An accompanying slide presentation may also be accessed via the BioCryst website. Please connect to the website at least 15 minutes prior to the start of the conference call to ensure adequate time for any software download that may be necessary.

Special Meetings of Stockholders

On April 10, 2018, BioCryst and Idera jointly announced that they have each rescheduled their respective Special Meetings of Stockholders to vote on the proposed merger of BioCryst and Idera to July 10, 2018 at 10:00 a.m. Eastern Time.

The BioCryst Board of Directors unanimously recommends that BioCryst stockholders vote "FOR" the proposed merger at the BioCryst Special Meeting.

About BCX7353

Discovered by BioCryst, BCX7353 is a novel, oral, once-daily, selective inhibitor of plasma kallikrein currently in development for the prevention and treatment of angioedema attacks in patients diagnosed with HAE. BCX7353 has been generally safe and well tolerated in the Phase 2 APeX-1 clinical trial. BioCryst is currently conducting the Phase 3 APeX-2 clinical trial and the long-term safety APeX-S clinical trial, both evaluating two dosage strengths of BCX7353 administered orally once-daily as a preventive treatment to reduce the frequency of attacks in patients with HAE. BioCryst is also conducting the ZENITH-1 clinical trial. ZENITH-1 is a proof-of-concept Phase 2 clinical trial testing an oral liquid formulation of BCX7353 for the treatment of acute angioedema attacks.

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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

On May 8, 2018 TESARO, Inc., an oncology-focused biopharmaceutical company, reported results from a TESARO-sponsored Europe-wide literature review examining the views of both patients and physicians on the communication and treatment needs in the care of women with ovarian cancer (Press release, TESARO, MAY 8, 2018, View Source [SID1234526246]). The comprehensive review confirms that the psychosocial effects of ovarian cancer play a significant part in a woman’s quality of life. The review, ‘Our Way Forward – Ovarian Cancer in Europe’ draws data from approximately 65 publications and patient surveys, spanning the last 15 years.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The ‘Our Way Forward – Ovarian Cancer in Europe’ literature review is part of TESARO’s commitment to ovarian cancer as exemplified by the Our Way Forward program, an initiative that was launched in the U.S. last year. The review is the first step towards supporting the development of patient-focused information tools and bringing the debate in ovarian cancer higher up the medical and political agenda. The review was conducted with guidance and input from European and global patient advocacy organizations, with support by TESARO, under its Our Way Forwardprogram. Our Way Forward was created by TESARO, Inc. with input from the National Ovarian Cancer Coalition (NOCC) and the Ovarian Cancer Research Fund Alliance (OCRFA). The program was launched in June of 2017 and is a call-to-action for ovarian cancer patients, their loved ones and healthcare providers to rethink how they talk about advanced ovarian cancer and ways to partner together to navigate the physical and emotional challenges that the disease brings.

Orlando Oliveira, Senior Vice President and General Manager, TESARO International, explains "TESARO is committed to offering resources to educate, empower and support those affected by ovarian cancer. There is clearly a need for better information and enhanced dialogue around the care of women with ovarian cancer in Europe, as well as new treatments that don’t impair quality of life. Through Our Way Forward and strong collaboration with the patient advocacy community, we aim to help support women with ovarian cancer to speak out and bring their needs to the attention of those that can make a difference, so both their psychological and physical needs can be met, every step of the way."

According to the findings from the literature review, ovarian cancer is frequently diagnosed late,3 leading to urgency in treatment and little time for healthcare professionals (HCPs) to consider the psychological aspects of care.4 The psychological impact of cancer can have a significant impact on treatment outcomes as 80% of women experienced poor mental health2 and patients with depression and anxiety are at a significantly greater risk of mortality, hospitalization and poorer treatment outcomes.5

Despite this, women feel they receive information about the tangible and practical aspects of treatment, but much less so for the psychosocial aspects of the disease and how to cope.4 Women are also less likely to bring the topic up with their HCPs as most women feel uncomfortable raising psychological and emotional concerns during their appointment as they are concerned about taking up too much time.1 As a result, women with ovarian cancer feel isolated, with little support, and feel the need for more information.4

Elisabeth Baugh, Chair, World Ovarian Cancer Coalition, explains that "As the leading global ovarian cancer patient advocacy organization, the World Ovarian Cancer Coalition is pleased to support the release today of this important research undertaken as part of TESARO’s Our Way Forward program. The focus of this work on improving both physical and psychological outcomes for women with ovarian cancer, in Europe and around the world is welcomed, especially as it complements our own World Ovarian Cancer Coalition’s Every Woman Study findings. As we celebrate World Ovarian Cancer Day 2018, the Coalition is pleased to work with all partners on this important day to highlight the struggles that ovarian cancer patients face."

The review also revealed that the ovarian cancer patient journey is fragmented and unpredictable, leading to considerable fear for disease recurrence. Ovarian cancer recurs in approximately 85% of women with advanced ovarian cancer6 and this is at the very top of mind for ovarian cancer survivors. The review also revealed women with recurrent ovarian cancer are not receiving the same level of support as those who are newly diagnosed, with one study showing that for 53% of patients, no one had discussed symptoms of recurrence, and 63% of nurses claim they don’t have the time do so.2

Recurrence of ovarian cancer also takes a psychological toll with 60% of women with recurrence reporting they can do less than they wanted to do because of their emotional status (versus 16% of women without recurrence) and 66% of women with recurrence reporting having trouble concentrating (versus 26% of women without recurrence).7

An important psychological aspect of treatment in relation to allaying fears of recurrence is a discussion around treatment options. Patients need to make a critical trade-off between efficacy and quality of life. This, however, is often under-addressed by their healthcare team. The review revealed that 86% of patients said they would be willing to try a drug that could improve the quality of their life even if it may not prolong it.8

This is the first phase of the ‘Our Way Forward – Ovarian Cancer in Europe’ review, with a second-phase review of the literature planned with healthcare providers to provide a 360-degree picture of the current treatment journey. The full review will offer fresh insights that will support advocacy groups in amplifying the voice of women with ovarian cancer by helping in the development of resources and tools for these women.

On May 8, 2018 Athenex, Inc. (Nasdaq:ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported that management will present at the Deutsche Bank’s 43rd Annual Health Care Conference in Boston, MA, on Tuesday, May 8th, at 2:10 pm EDT (Press release, Athenex, MAY 8, 2018, View Source;p=RssLanding&cat=news&id=2347628 [SID1234526264]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The presentation will be webcasted, and can be accessed at the Investor Relations section of the Company’s website, located at www.athenex.com. An archive will be available at this website until August 6, 2018.

On May 8, 2018 Endo International plc (NASDAQ: ENDP) reported first-quarter 2018 financial results, including (Press release, Endocrine Pharmaceuticals, AUG 8, 2018, View Source [SID1234526280]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Revenues of $701 million, a 32 percent decrease compared to first-quarter 2017 revenues of $1,038 million.
Reported net loss from continuing operations of $498 million compared to first-quarter 2017 reported net loss from continuing operations of $165 million.
Reported diluted loss per share from continuing operations of $2.23 compared to first-quarter 2017 reported diluted loss per share from continuing operations of $0.74.
Adjusted income from continuing operations of $151 million compared to first-quarter 2017 adjusted income from continuing operations of $275 million.
Adjusted diluted EPS from continuing operations of $0.67 compared to first-quarter 2017 adjusted diluted EPS from continuing operations of $1.23.
Adjusted EBITDA of $334 million compared to first-quarter 2017 adjusted EBITDA of $478 million.
"Endo delivered strong first-quarter operating results in our core focus areas as revenue growth accelerated for both our XIAFLEX franchise and Sterile Injectable business. We are currently on target to meet the full-year financial guidance we provided earlier this year," said Paul Campanelli, President and CEO of Endo. "Importantly, we continue to execute our multi-year turnaround plan and build our portfolio for the future by advancing our CCH cellulite treatment development program with the initiation of and recruitment for two pivotal Phase 3 trials. In addition, as we recently announced, we expect to significantly enhance our Sterile Injectables pipeline through the acquisition of Somerset Therapeutics."