Audentes Therapeutics to Participate in Upcoming Investor Conferences

On November 7, 2018 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, reported that it will participate in the following investor conferences in November (Press release, Audentes Therapeutics, NOV 7, 2018, View Source [SID1234530902]):

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Credit Suisse 27th Annual Healthcare Conference Natalie Holles, President and Chief Operating OfficerFormat: 1×1 Investor MeetingsWednesday, November 14, 2018Scottsdale, Arizona
Evercore ISI HealthConX Conference Matthew R. Patterson, Chairman and Chief Executive OfficerFormat: Fireside ChatTuesday, November 27, 2018, 10:35am ESTBoston, Massachusetts
Piper Jaffray 30th Annual Healthcare Conference Matthew R. Patterson, Chairman and Chief Executive OfficerFormat: Fireside ChatWednesday, November 28, 2018, 11:30am ESTNew York, New York
Barclays Gene Editing & Gene Therapy SummitMatthew R. Patterson, Chairman and Chief Executive OfficerFormat: Corporate PresentationThursday, November 29, 2018, 10:15am ESTNew York, New York
To access live webcasts of the fireside chats and presentation please visit the Events & Presentations page within the Investors + Media section of the Audentes website. Following each conference, a replay of the live webcast will be available on the Audentes website for approximately 30 days.

Novavax Reports Third Quarter 2018 Financial Results

On November 7, 2018 Novavax, Inc., (Nasdaq: NVAX) reported its financial results and operational highlights for the third quarter and nine months ended September 30, 2018 (Press release, Novavax, NOV 7, 2018, View Source [SID1234530919]).

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"We remain on track to meet our 2018 objectives, as we continue to execute on our clinical milestones for ResVax and NanoFlu," said Stanley C. Erck, President and Chief Executive Officer of Novavax, Inc. "In Prepare, our Phase 3 trial of ResVax, we will complete monitoring of the efficacy endpoints by year-end, which would allow us to announce top-line efficacy results in the first quarter of 2019. For NanoFlu, we have completed enrollment of our Phase 2 clinical trial and anticipate top-line results in the first quarter of 2019. Based on this progress, we expect to conduct an End-of-Phase 2 meeting with the FDA in the first half of 2019 and to initiate a pivotal Phase 3 clinical trial later in the year."

Third Quarter 2018 Operational Highlights

ResVax Program

Novavax expects to report top-line results from its final efficacy analysis in the first quarter of 2019 and, assuming successful results, expects to submit the Biologics License Application (BLA) and Marketing Authorization Application (MAA) by the first quarter of 2020.
NanoFlu Program

In September 2018, Novavax initiated a Phase 2 clinical trial in older adults of quadrivalent formulations of NanoFlu, its nanoparticle seasonal influenza vaccine candidate, and in October 2018, Novavax completed enrollment of approximately 1,375 healthy older adults across clinical sites in the U.S. This randomized, observer-blinded, active-controlled trial will assess the safety and tolerability of different doses and formulations of NanoFlu, both adjuvanted with Matrix-M and unadjuvanted, as compared to two U.S.-licensed comparators. Phase 2 clinical trial top-line results are expected in the first quarter of 2019.

During a pre-IND meeting in 2018, the FDA acknowledged and agreed that the accelerated approval pathway for seasonal influenza vaccines could be available for NanoFlu. Novavax plans to discuss the Phase 2 data and the proposed Phase 3 study design, and reach agreement on the use of accelerated approval, with the FDA during an End-of-Phase 2 meeting in the first half of 2019. Novavax will bring forward the selected dose/formulation of the Phase 2 clinical trial into its future pivotal Phase 3 immunogenicity clinical trial.
Corporate

Novavax announced the appointment of Rachel King, Co-Founder and Chief Executive Officer of GlycoMimetics, Inc., to its Board of Directors. In addition to extensive experience as an executive in the biotechnology industry, Mrs. King has also worked in the venture capital side of life sciences, and has held executive positions within a global multinational pharmaceutical company.
Key Upcoming Anticipated Events

Final efficacy results of the Prepare trial are expected in the first quarter of 2019.

Top-line data from the Phase 2 clinical trial of NanoFlu are expected in the first quarter of 2019.

An End-of-Phase 2 meeting for NanoFlu is expected in the first half of 2019.
Financial Results for the Three and Nine Months Ended September 30, 2018

Novavax reported a net loss of $44.6 million, or $0.12 per share, for the third quarter of 2018, compared to a net loss of $44.6 million, or $0.15 per share, for the third quarter of 2017. For the nine months ended September 30, 2018, the net loss was $135.4 million, or $0.37 per share, compared to a net loss of $132.9 million, or $0.47 per share, for the same period in 2017.

Novavax revenue in the third quarter of 2018 was $7.7 million, compared to $8.4 million in the same period in 2017. This 7% decrease was driven by the completion of enrollment of the Prepare trial in the second quarter of 2018.

Research and development expenses decreased 1% to $41.3 million in the third quarter of 2018, compared to $41.9 million for the same period in 2017.

General and administrative expenses increased 2% to $8.3 million in the third quarter of 2018, compared to $8.1 million for the same period in 2017.

Interest income (expense), net for the third quarter of 2018 was ($2.7) million, compared to ($3.0) million for the same period of 2017.

As of September 30, 2018, Novavax had $145.6 million in cash, cash equivalents, marketable securities and restricted cash, compared to $186.4 million as of December 31, 2017. Net cash used in operating activities for the third quarter of 2018 was $33.5 million, compared to $44.2 million for same period in 2017. The decrease in cash usage was primarily due to receiving a $15 million payment under the BMGF grant in the third quarter of 2018, whereas no payment was received in the same period of 2017.

Conference Call

Novavax will host its quarterly conference call today at 4:30 p.m. ET. The dial-in numbers for the conference call is (877) 212-6076 (Domestic) or (707) 287-9331 (International), passcode 3639227. A replay of the conference call will be available starting at 7:30 p.m. ET on November 7, 2018 until 7:30 pm ET on November 14, 2018. To access the replay by telephone, dial (855) 859-2056 (Domestic) or (404) 537-3406 (International) and use passcode 3639227.

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 February 7, 2019.

About RSV in Infants

Globally, RSV (respiratory syncytial virus) is the leading viral cause of severe lower respiratory tract disease in infants and young children.1 It is the second leading cause of death in children under one year of age.2 Estimated annual hospitalizations of 1.4 million and an estimated 27,300 in-hospital deaths were due to RSV acute lower respiratory infection in children under six months of age.3 RSV results in a total global economic burden of $6.2 billion annually.

In the U.S., RSV is the leading cause of hospitalization of infants, with estimated annual hospitalizations of up to 76,000.4,[5],[6] While RSV can impact all infants, babies under six months of age are among those at highest risk, as approximately 77% of all first-year RSV infections occur before six months.7 In the U.S., the total economic burden is $2.7 billion annually.

About ResVax

ResVax is an RSV fusion (F) protein recombinant nanoparticle vaccine with aluminum phosphate as an adjuvant. It is being developed to protect infants from RSV disease via maternal immunization, which may offer the best method of protection from RSV disease in infants through the first months of life. Currently, ResVax is being evaluated in Prepare, a global Phase 3 clinical trial in 4,636 pregnant women, at least 3,000 of whom have received the vaccine, and their infants. Prepare is supported by an $89.1 million grant from the Bill & Melinda Gates Foundation (BMGF).

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 globally each year are attributed to influenza.8 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 2012-13 season to $5.3 billion by the 2021-22 season.9

About NanoFluand Matrix M

NanoFlu is a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine produced by Novavax in its SF9 insect cell baculovirus system. NanoFlu uses HA amino acid protein sequences that are the same as the recommended wild-type circulating virus HA sequences. NanoFlu 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. In October 2018, Novavax completed enrollment in its Phase 2 clinical trial in older adults of quadrivalent formulations of NanoFlu in 1,375 healthy older adults across clinical sites in the U.S.

About Accelerated Approval

Accelerated approval may be granted for certain biological products that have been studied for their safety and effectiveness in treating serious or life-threatening illnesses and that provide meaningful therapeutic benefit over existing treatments. Such an approval will be based on adequate and well-controlled clinical trials establishing that the biological product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit. For seasonal influenza vaccines, the hemagglutination inhibition (HAI) antibody response may be an acceptable surrogate marker of activity that is reasonably likely to predict clinical benefit. To be considered for accelerated approval, a biologics license application for a new seasonal influenza vaccine should include results from one or more well-controlled studies designed to meet immunogenicity endpoints and a commitment to conduct confirmatory post-marketing studies of clinical effectiveness in preventing influenza.

Protagonist Therapeutics to Present at the Stifel 2018 Healthcare Conference Wednesday, Nov. 14

On November 7, 2018 Protagonist Therapeutics, Inc. (NASDAQ:PTGX) reported that Dinesh V. Patel, Ph.D., President and Chief Executive Officer, will provide a corporate overview at the Stifel 2018 Healthcare Conference at 8:45 a.m. EST at the Lotte New York Palace Hotel (Press release, Protagonist, NOV 7, 2018, View Source;p=RssLanding&cat=news&id=2376046 [SID1234531167]).

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Presentation details:

Event:

Stifel 2018 Healthcare Conference

Date:

Wednesday, Nov. 14

Time:

8:45 a.m. EST

A live and archived audio webcast of the presentation can be accessed by visiting the Investors page of the Protagonist Therapeutics website at View Source

Horizon Pharma plc Reports Third-Quarter Net Sales Growth of 20 Percent Driven by Orphan and Rheumatology Net Sales Growth of 25 Percent; Increases Full-Year 2018 Adjusted EBITDA Guidance

On November 7, 2018 Horizon Pharma plc (NASDAQ: HZNP) reported its third-quarter 2018 financial results, confirmed its full-year 2018 net sales guidance range and increased its adjusted EBITDA guidance range (Press release, Horizon Pharma, NOV 7, 2018, View Source [SID1234530843]).

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"We generated record quarterly net sales for the Company and for our orphan and rheumatology segment, driven by accelerating KRYSTEXXA growth and continued strong performance from our rare disease medicines," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc.

"Our clinical programs continue to advance, with the recent presentation of new teprotumumab Phase 2 data that underscore the durable efficacy observed in thyroid eye disease," continued Walbert. "We are also working to maximize the role of KRYSTEXXA to help more patients, including adapting our MIRROR immunomodulation clinical trial based on promising recent data to support the potential for registration. These advancements, in addition to potential asset acquisition opportunities, support our strategy to build a robust pipeline enabling sustainable long-term growth."

Third-Quarter and Recent Company Highlights

New Phase 2 Teprotumumab Data Presented at American Thyroid Associationand American Academy of Ophthalmology: At Week 24, 71.4 percent (30 of 42) of patients responded with reductions in proptosis (eye bulging) of 2mm or more and 61.9 percent of patients (26 of 42) responded with improvement of at least one grade in diplopia (double vision), which is considered a clinically meaningful change. At Week 72, 48 weeks following the study completion and nearly a year off therapy, 53.3 percent of the Week 24 proptosis responders maintained the reductions and 69.2 percent of patients with diplopia improvement maintained the benefit. These results demonstrate that teprotumumab has the potential to be a disease-modifying therapy.

Teprotumumab is a fully human monoclonal antibody IGF-1R inhibitor in Phase 3 development for the treatment of thyroid eye disease (TED), in which the muscles and fatty tissue behind the eye become inflamed, which can lead to proptosis and diplopia as well as quality-of-life issues.
New KRYSTEXXA Immunomodulation Data Presented at American College of Rheumatology/Association of Rheumatology Health Professionals (ACR): At the 2018 ACR meeting in October, investigators shared results from a case series of nine sequential patients with uncontrolled gout (chronic gout that is refractory to conventional therapies), evaluating the administration of KRYSTEXXA with the immunomodulator methotrexate to improve the durability of KRYSTEXXA response. The study states that of the nine patients followed out to five months, all nine were responders as defined by >80 percent of serum uric acid levels being maintained at goal <6.0 mg/dL during the observation period.

In its clinical trial MIRROR (Methotrexate to Increase Response Rates in Patients with UncontrolledGOut Receiving KRYSTEXXA), the Company is evaluating the administration of KRYSTEXXA with methotrexate to potentially improve the durability of response rate. Following the external case series data, the Company is adapting its MIRROR trial to support the potential for registration. Methotrexate has been shown to reduce anti-drug antibodies when combined with biologics and is the immunomodulator most commonly used by rheumatologists.
Uncontrolled Gout and KRYSTEXXA Data Presented at ACR and American Society of Nephrology (ASN): The Company participated in both the ACR and ASN medical meetings, all in October. At ACR, multiple studies were shared demonstrating the extensive burden of uncontrolled gout and its impact on patients. At ASN, multiple studies were shared demonstrating that people who have undergone a kidney transplant experience higher rates of uncontrolled gout compared to other renal disease patients and mortality rates were higher in kidney transplant recipients diagnosed with gout.
Intellectual Property Update:The Company settled litigation with Par Pharmaceutical (part of Endo International), the first generic filer on RAVICTI. Par’s license to enter the market with a generic version of RAVICTI would begin on July 1, 2025.
Research and Development Programs

Orphan Candidates and Programs:

Teprotumumab: The pivotal Phase 3 confirmatory study, OPTIC, is evaluating teprotumumab for the treatment of moderate-to-severe active TED, which has no FDA-approved treatments. OPTIC completed enrollment on Sept. 4, 2018, and topline results are expected in the second quarter of 2019. OPTIC-X, a 48-week open-label extension study in which participants may receive up to eight additional infusions of teprotumumab, continues to enroll patients. The objective of OPTIC-X is to evaluate the potential for retreatment and longer treatment with teprotumumab.
Rheumatology Pipeline Candidates and Programs:

KRYSTEXXA Immunomodulation Studies: The evaluation of the use of immunomodulation therapies to enhance the response rate to KRYSTEXXA is being studied in MIRROR, as well as two investigator-initiated trials. The three trials are evaluating different immunomodulators, each of which are used by rheumatologists.
MIRROR: a Horizon Pharma-sponsored multicenter, efficacy and safety study for KRYSTEXXA co-administered with methotrexate to evaluate the impact of methotrexate on response rate. Enrollment began in the fourth quarter of 2018. The Company is currently adapting the MIRROR clinical trial design to support the potential for registration and expects to begin enrolling patients into the adapted protocol in the second quarter of 2019
Two additional investigator-initiated trials co-administerKRYSTEXXA withmycophenolate mofetil (MMF) and with azathioprine.
Next-generation Biologic Programs for Uncontrolled Gout: The Company is pursuing two development programs for next-generation biologics for uncontrolled gout, HZN-003 and PASylated uricase technology, to support and sustain the Company’s market leadership in uncontrolled gout. The programs are exploring the use of optimized uricase technology as well as optimized PEGylation and PASylation technology.
Third-Quarter Financial Results

Note:For additional detail and reconciliation of non-GAAP financial measures to the most directly comparable GAAP financial measures, please refer to the tables at the end of this release.

Net Sales: Third-quarter 2018 net sales were $325.3 million, an increase of 20 percent, driven by continued strong growth of the Company’s orphan and rheumatology medicines.
Gross Profit: Under U.S. GAAP in the third quarter of 2018, the gross profit ratio was 69.6 percent compared to 53.8 percent in the third quarter of 2017. The non-GAAP gross profit ratio in the third quarter of 2018 was 91.2 percent compared to 89.6 percent in the third quarter of 2017.
Operating Expenses: R&D expenses were 6.5 percent of net sales and selling, general and administrative (SG&A) expenses were 49.7 percent of net sales. Non-GAAP R&D expenses were 5.9 percent of net sales and non-GAAP SG&A expenses were 39.3 percent of net sales.
Income Tax Rate: In the third quarter of 2018, the income tax benefit rate on a GAAP basis was 7.0 percent and the income tax expense rate on a non-GAAP basis was 10.1 percent.
Net Income: On a GAAP basis in the third quarter of 2018, net income was $26.0 million. Third-quarter 2018 non-GAAP net income was $112.6 million.
Adjusted EBITDA: Third-quarter 2018 adjusted EBITDA was $149.9 million.
Earnings (Loss) per Share: On a GAAP basis in the third quarter of 2018, diluted earnings per share was $0.15; in the third quarter of 2017, diluted loss per share was $0.39. Non-GAAP diluted earnings per share in the third quarter of 2018 and 2017 were $0.65 and $0.26, respectively. Weighted average shares outstanding used for calculating GAAP diluted earnings per share and non-GAAP diluted earnings per share in the third quarter of 2018 were 167.0 million and 172.5 million, respectively.
Third-Quarter Segment Results

Management uses net sales and segment operating income to evaluate the performance of the Company’s two segments. While segment operating income contains certain adjustments to the directly comparable GAAP figures in the Company’s consolidated financial results, it is considered to be prepared in accordance with GAAP for purposes of presenting the Company’s segment operating results.

(1) On June 23, 2017, Horizon Pharma completed the divestiture of a European subsidiary that owned the marketing rights to PROCYSBI and QUINSAIR in Europe, the Middle East and Africa (EMEA) to Chiesi Farmaceutici S.p.A. Horizon Pharma retains marketing rights for the two medicines in the United States, Canada, Latin America and Asia. Year-to-date 2017 net sales of PROCYSBI and QUINSAIR in EMEA were $9.5 million.
Third-quarter 2018 net sales of the orphan and rheumatology segment were $219.9 million, an increase of 25.3 percent over the prior year’s quarter, driven by continued strong KRYSTEXXA growth as well as growth of RAVICTI and PROCYSBI. Third-quarter 2018 orphan and rheumatology segment operating income was $91.5 million, an increase of 39.5 percent.

Third-quarter 2018 net sales of the primary care segment were $105.4 million and operating income was $58.0 million.
Cash Flow Statement and Balance Sheet Highlights

On a GAAP basis in the third quarter of 2018, operating cash flow was $84.9 million. Non-GAAP operating cash flow was $95.6 million.
The Company had cash and cash equivalents of $807.0 million as of Sept. 30, 2018.
As of Sept. 30, 2018, the total principal amount of debt outstanding was $1.993 billion, which consists of $818 million in senior secured term loans due 2024; $300 million senior notes due 2024; $475 million senior notes due 2023 and $400 million exchangeable senior notes due 2022. As of Sept. 30, 2018, net debt was $1.186 billion.
Full-Year 2018 Guidance

The Company confirmed its full-year 2018 net sales guidance of $1.170 billion to $1.200 billion and continues to project full-year 2018 net sales growth for KRYSTEXXA of more than 65 percent. The Company increased its full-year 2018 adjusted EBITDA guidance range to $420 million to $430 million, from $400 million to $420 million.

Webcast

At 8 a.m. EST / 1 p.m. IST today, the Company will host a live webcast to review its financial and operating results and provide a general business update. The live webcast and a replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay of the webcast will be available approximately two hours after the live webcast.

Incyte to Present at Upcoming Investor Conference

On November 7, 2018 Incyte Corporation (Nasdaq:INCY) reported that it will present at Evercore ISI HEALTHCONx on Wednesday, November 28, 2018 at 3:30 pm EST in Boston (Press release, Incyte, NOV 7, 2018, View Source [SID1234530904]).

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The presentation will be webcast live and can be accessed at www.incyte.com in the Investors section under "Events and Presentations." Investors interested in listening to the live webcast should log on before the start time in order to download any software required.