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Tetraphase Pharmaceuticals to Present at Upcoming Investor Conferences

On November 7, 2018 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel antibiotics to treat life-threatening multidrug-resistant (MDR) infections, reported that President and Chief Executive Officer Guy Macdonald will present a corporate overview at the Stifel 2018 Healthcare Conference on Wednesday, November 14, 2018 at 8:00 a.m. ET at Lotte New York Palace Hotel in New York City. Mr. Macdonald also will participate in a fireside chat at the Piper Jaffray 30th Annual Healthcare Conference on Tuesday, November 27, 2018 at 2:30 p.m. ET at the Lotte New York Palace Hotel in New York City.

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A live audio webcast of the Stifel 2018 Healthcare Conference presentation will be available on the Company’s website at View Source The archived presentation will be available for 30 days.

NantHealth to Report 2018 Third-Quarter Financial Results and Host Conference Call on Thursday, November 15

On November 7, 2018 NantHealth, Inc. (NASDAQ-GS: NH), a next-generation, evidence-based, personalized healthcare company, reported that it will report financial results for its 2018 third quarter on Thursday, November 15, 2018, after market close (Press release, NantHealth, NOV 7, 2018, View Source;p=RssLanding&cat=news&id=2375793 [SID1234530850]). NantHealth management will host a conference call that same day at 1:30 p.m. PT (4:30 p.m. ET) to review the company’s performance. The company intends to file its Quarterly Report on Form 10-Q for the three and nine months ended September 30, 2018 on or before November 23, 2018.

The company’s normal process for compilation and review of its financial statements was delayed due to the effects of Hurricane Michael on accounting personnel and accounting operations in the company’s offices in Panama City, Florida.

The conference call will be available to interested parties by dialing 844-309-3709 from the U.S. or Canada, or 281-962-4864 from international locations, passcode 9992769. The call will be broadcast via the Internet at www.nanthealth.com.

Bellicum Pharmaceuticals to Present at the Jefferies 2018 London Healthcare Conference

On November 7, 2018 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported that Rick Fair, President and CEO, is scheduled to present a corporate overview at the Jefferies 2018 London Healthcare Conference on Wednesday, November 14 at 5:20 p.m. GMT in London, UK (Press release, Bellicum Pharmaceuticals, NOV 7, 2018, View Source [SID1234530849]).

A live webcast of the presentation may be accessed from the News & Events section of the Bellicum website. An archived version of the webcast will be available for replay for at least two weeks following the event.

Pieris Pharmaceuticals Reports Third Quarter 2018 Financial Results and Provides Corporate Update

On November 7, 2018 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory, cancer and other diseases, reported financial results for the third quarter of 2018 ended September 30, 2018 and provided an update on the Company’s recent and future developments (Press release, Pieris Pharmaceuticals, NOV 7, 2018, View Source [SID1234530848]).

"We are pleased to announce that PRS-060, an IL-4 receptor alpha antagonist for moderate-to-severe asthma, was safe and well-tolerated by healthy volunteers in a single ascending dose Phase I study. This is the first clinical use of an inhaled Anticalin protein and marks an important development milestone for the Company. We continue to enroll subjects with mild asthma in a multiple ascending dose Phase I study. Along with our partner AstraZeneca, we anticipate reporting the data from the Phase I studies at an upcoming medical meeting. For PRS-343, our 4-1BB/HER2 bispecific, we now intend to report initial data from a Phase I study in the first half of next year, after completing the dose escalation, to provide a more comprehensive data set," said Stephen S. Yoder, President and CEO of Pieris. "We look forward to providing updates on our progress as we head into 2019."

PRS-060: PRS-060, an inhaled IL-4 receptor alpha antagonist for moderate-to-severe asthma, was tested in 48 healthy volunteers at dose levels ranging from 0.25 mg to 400 mg in a single ascending dose Phase I study. The drug candidate was safe and well-tolerated in this study. Pieris continues to enroll subjects with mild asthma and elevated levels of fractional exhaled nitric oxide (FeNO) in a multiple ascending dose Phase I study. This study will evaluate the safety, tolerability and FeNO-reducing potential of PRS-060 versus placebo. The data from the PRS-060 Phase I studies will be presented at an upcoming medical meeting. PRS-060 is the lead candidate in Pieris’ respiratory collaboration with AstraZeneca. Pieris is sponsoring the Phase I studies and AstraZeneca is funding the costs. AstraZeneca will conduct and fund the Phase IIa study, after which Pieris will have separate options to co-develop and co-commercialize the drug candidate.

PRS-343: The Company continues to enroll and treat patients in a Phase I dose-escalation study of PRS-343, a 4-1BB/HER2 bispecific for HER2-positive solid tumors, and now intends to report initial data from the study in the first half of 2019, after completing the dose escalation, in order to provide a more comprehensive data set. Our objective for the remaining enrollment will be to favor patient selection across a range of immunotherapy-responsive tumor types. The Company is screening patients for the 11th cohort of the study, a dose level comparable to approved dose levels of trastuzumab. In August, Pieris initiated a trial with PRS-343 in combination with atezolizumab, and the Company intends to report data from this trial in 2019.

PRS-080: Pieris is planning to enroll and complete dosing of the final patient in its Phase IIa multiple ascending dose study of PRS-080, a half-life-optimized hepcidin antagonist for anemia, by year end. In the ongoing trial, PRS-080 at both doses (4mg/kg and 8mg/kg) has not generated any observed drug-related serious adverse events and has demonstrated substantial iron mobilization and transferrin saturation (TSAT) increases versus placebo. To date, there has been no conclusive change in hemoglobin at either dose versus placebo. We remain committed to completing the study and sharing the data with ASKA and other potential partners for continued development. We intend to report the full data set from the study in the first half of 2019.

PRS-344: Pieris will present preclinical data for PRS-344, a PD-L1/4-1BB antibody-Anticalin bispecific in IND-enabling studies, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2018 Annual Meeting in Washington, D.C. The poster, P375, will be presented on November 9–10, 2018. PRS-344 is one of five development programs in Pieris’ immuno-oncology alliance with Servier.

Board Appointments: In September 2018, Pieris appointed Peter Kiener, D.Phil. to the Company’s Board of Directors. Dr. Kiener served in several executive roles, most recently as the Chief Scientific Officer at Sucampo. In October 2018, Pieris also appointed Matthew L. Sherman, M.D. to the Company’s Board of Directors. Dr. Sherman most recently served as Executive Vice President and Chief Medical Officer at Acceleron Pharma.

Preclinical Pipeline: Pieris remains committed to advancing several early-stage programs into the clinic and is on track to file two immuno-oncology INDs, one for a proprietary drug candidate and one as part of its collaboration with Servier, in 2019. The Company has also initiated two proprietary respiratory programs.
Third Quarter Financial Update:

Cash Position – Cash, cash equivalents and investments totaled $137.3 million as of September 30, 2018, compared to a cash, cash equivalents and investments balance of $82.6 million as of December 31, 2017. The increase was driven primarily by the $47.2 million in net proceeds from the Company’s February 2018 equity financing, the $30.0 million in upfront payments received as part of the Seattle Genetics immuno-oncology collaboration, and the $12.5 million milestone payment from AstraZeneca that was triggered during the fourth quarter of 2017 and received during the first quarter of 2018. The increase was partially offset by $35.7 million of operating cash expenditures during the year.

R&D Expense – R&D expenses were $11.4 million for the three months ended September 30, 2018, compared to $6.3 million for the three months ended September 30, 2017. R&D expenses were $28.5 million for the nine months ended September 30, 2018, compared to $17.0 million for the nine months ended September 30, 2017. The Company’s increase in R&D expenses reflects preparation for and advancement of clinical studies as well as advancement across its pipeline of preclinical programs.

G&A Expense – G&A expenses were $4.7 million for the three months ended September 30, 2018, compared to $2.9 million for the three months ended September 30, 2017. G&A expenses were $13.9 million for the nine months ended September 30, 2018, compared to $11.2 million for the nine months ended September 30, 2017. The Company’s increase in G&A expenses reflects higher personnel costs and professional services costs for audit and legal, as well as an increase in general administrative costs to support the growing business of the Company. On a nine-month basis, the increase was partially offset by lower transaction fees for license and collaboration agreements compared to amounts recorded in the first half of 2017.

Interest Income – Interest income was $0.5 million and $1.5 million for the three and nine months ended September 30, 2018, respectively, compared to no interest income earned in the comparable 2017 periods. The Company began investing cash received from the collaboration agreements signed in 2017 in the fourth quarter of 2017.

Other Income – Other income was $1.1 million and $1.5 million for the three and nine months ended September 30, 2018, respectively, compared to other expense of $1.7 million and $3.1 million for the three and nine months ended September 30, 2017, respectively. The Company began investing cash received from the collaboration agreements signed in 2017 in the fourth quarter of 2017. The increase in income for the three- and nine-month periods is a result of net foreign currency transaction gains due to the strengthening of the U.S. dollar against the euro, positively impacting the remeasurement of U.S. dollar denominated monetary assets held in Germany.

Net Loss – Net loss was $6.2 million or $(0.11) per share for the three months ended September 30, 2018, compared to a net loss of $7.1 million or $(0.16) per share for the three months ended September 30, 2017. Net loss was $15.1 million or $(0.29) per share for the nine months ended September 30, 2018, compared to a net loss of $25.1 million or $(0.58) per share for the nine months ended September 30, 2017.

Conference Call:

Pieris management will host a conference call beginning at 8:00 AM Eastern Standard Time on Wednesday, November 7, 2018, to discuss the third quarter of 2018 financial results and provide a corporate update. Individuals can join the call by dialing +1-877-407-8920 (US & Canada) or +1-412-902-1010 (International). An archived replay of the call will be available by dialing +1-877-660-6853 (US & Canada) or +1-201-612-7415 (International) and providing the Conference ID #: 13661472.

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]).

"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.