On March 15, 2019 Molecular Partners AG (SIX: MOLN), a clinical-stage biotech company pioneering the use of DARPin therapeutics* to treat serious diseases, reported its audited Financial Results for 2018 and the company’s 2018 Annual Report (Press release, Molecular Partners, MAR 15, 2019, View Source [SID1234534327]).

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The Audited Financial Results for 2018 and the company’s 2018 Annual Report are available on the investors section of the company’ website.

Financial Calendar
March 19, 2019 – Expected Publication of Invitation to Annual General Meeting
April 16, 2019 – Annual General Meeting
May 9, 2019 – Interim Management Statement Q1 2019
August 27, 2019 – Publication of Half-year Results 2019 (unaudited)
October 31, 2019 – Interim Management Statement Q3 2019
View Source

About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics opening an extra dimension of multi-specificity and multi-functionality. DARPin candidates are potent, specific, safe and very versatile. They can engage more than 5 targets at once, offering potential benefits over those offered by conventional monoclonal antibodies or other currently available protein therapeutics.
The DARPin technology is a fast and cost-effective drug discovery engine, producing drug candidates with ideal properties for development and very high production yields.

With their good safety profile, low immunogenicity and long half-life in the bloodstream and the eye, DARPin therapeutics have the potential to advance modern medicine and significantly improve the treatment of serious diseases, including cancer and sight-threatening disorders. Molecular Partners is partnering with Allergan to advance clinical programs in ophthalmology, and is advancing a proprietary pipeline of DARPin drug candidates in oncology and immuno-oncology. The most advanced global product candidate is abicipar, a molecule currently in phase 3, in partnership with Allergan. Several DARPin molecules for various ophthalmic indications are also in development. The most advanced DARPin therapeutic candidate wholly owned by Molecular Partners, MP0250, is in phase 2 clinical development for the treatment of solid tumors and hematological tumors. MP0274, the second-most advanced DARPin drug candidate owned by Molecular Partners, has broad anti-HER activity; it inhibits HER1, HER2 and HER3-mediated downstream signaling via Her2, leading to induction of apoptosis. MP0274 is currently in phase 1. Molecular Partners is also advancing a growing preclinical pipeline that features several immuno-oncological development programs. DARPin is a registered trademark owned by Molecular Partners AG.

On March 15, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported financial results for the quarter ended December 31, 2018. The Company also reviewed recent milestones (Press release, Stemline Therapeutics, MAR 15, 2019, View Source [SID1234534361]):

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ELZONRIS (tagraxofusp) – US Approval and Commercial Launch

ELZONRIS was FDA-approved on December 21, 2018 for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients, two years and older.

ELZONRIS has been commercially available in the U.S. since the end of January 2019, and patients are currently being treated with ELZONRIS in the commercial setting.

A Marketing Authorization Application (MAA) for ELZONRIS seeking marketing approval in Europe was submitted to, and subsequently validated by, the European Medicines Agency (EMA) in January 2019. The MAA has been granted accelerated assessment and is currently under review.
ELZONRIS – Market Expansion Efforts

ELZONRIS is being evaluated in clinical trials in additional indications, including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

Based on the clinical results observed in CMML and MF thus far, we are evaluating potential registrational pathways in these indications. For CMML, we intend to discuss registration-directed plans with the FDA mid-year.

We are also working towards expansion opportunities within the BPDCN universe, including maintenance therapy after stem cell transplant.

In parallel, we plan to expand our clinical efforts later this year and next into subsets of AML patients enriched for CD123+ expression.

We expect to provide periodic updates on these initiatives throughout this year and next at scientific conferences.
ASH Conference

In December 2018, ELZONRIS data were selected for four presentations at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) conference. Presentations included results of the BPDCN pivotal trial, delivered via oral presentation, and updated clinical trial data in patients with CMML and MF.

Additionally, we had an active clinical, medical affairs and pre-commercial presence at ASH (Free ASH Whitepaper) focused on BPDCN disease awareness.
SL-801

In October 2018, data from the ongoing Phase 1 trial of SL-801 in patients with advanced solid tumors were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress 2018. We expect to provide further updates at upcoming conferences.
SL-701

In November 2018, data from the Phase 2 trial of SL-701 in patients with second-line glioblastoma (GBM) were delivered via oral presentation at the 23rd Annual Meeting of the Society of Neuro-Oncology (SNO). We expect to provide further updates on this program later this year.
SL-901

SL-901 is a novel kinase inhibitor that was evaluated in an abbreviated Phase 1 trial of solid tumor patients in Europe. Neither a dose limiting toxicity nor maximum tolerated dose was identified, and there was one partial response (PR) in a patient with advanced non-small cell lung cancer. We plan to re-initiate a Phase 1 study by early 2020.
SL-1001

In March 2019, we announced the in-licensing of SL-1001, a novel, selective RET kinase inhibitor that demonstrated potent preclinical in vitro and in vivo activity. We expect to begin IND-enabling studies this year, with a Phase 1 clinical trial targeted for 2020.
Robert Francomano, SVP and Global Head of Commercial, commented, "2018 was a transformational year for Stemline as we invested in building out a focused, world class, commercial organization ahead of the FDA approval of ELZONRIS. We are excited to bring this important new treatment to patients, and our highly talented team is in the field, excited, engaged, and poised for success in 2019."

Ivan Bergstein, M.D., CEO of Stemline Therapeutics, commented, "We have built a solid foundation for growth in 2019 and beyond, driven by the launch of ELZONRIS in BPDCN. ELZONRIS is the first drug ever approved for BPDCN and brings to patients with BPDCN a new treatment option and hope. In parallel, our team is working diligently with the EMA in an effort to make ELZONRIS potentially available to patients in Europe. We continue to aggressively pursue market expansion efforts with ELZONRIS, while also building out our overall pipeline, with the goal of improving the lives of patients with cancer."

Fourth Quarter 2018 Financial Results Review
Stemline ended the fourth quarter of 2018 with $60.1 million in cash, cash equivalents and investments, as compared to $78.5 million as of September 30, 2018, which reflects cash expenditures of $18.4 million for the quarter. Subsequent to year-end 2018, Stemline completed a follow-on public offering during January 2019 raising $86.1 million in net cash proceeds bringing total cash, cash equivalents and investments to $125.2 million as of March 15, 2019.

For the fourth quarter of 2018, Stemline had a net loss of $26.6 million, or $0.92 per share, compared with a net loss of $21.7 million, or $0.93 per share, for the same period in 2017.

Research and development expenses were $12.1 million for the fourth quarter of 2018, which reflects a decrease of $4.6 million compared with $16.7 million for the fourth quarter of 2017. The higher costs in 2017 were primarily due to manufacturing and regulatory expenses in support of our BLA filing for ELZONRIS.

General and administrative expenses were $14.9 million for the fourth quarter of 2018, which reflects an increase of $9.7 million compared with $5.2 million for the fourth quarter of 2017. The increase in costs were primarily attributable to pre-launch expenses in support of the commercialization of ELZONRIS and compensation costs related to an increase in headcount to support the commercial launch.

On March 14, 2019 AstraZeneca will share pioneering research and development across its successful Oncology portfolio and extensive next-generation pipeline at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Atlanta, USA, 29 March to 3 April 2019 (Press release, AstraZeneca, MAR 14, 2019, View Source [SID1234534328]).

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The new research will showcase AstraZeneca’s potent and selective DNA-dependent protein kinase (DNA-PK) inhibitor, taking the DNA Damage Response (DDR) pipeline beyond PARP inhibition. Additional highlights include new insights from the MYSTIC and TATTON trials for Imfinzi (durvalumab) and Tagrisso (osimertinib) in predicting response and addressing treatment resistance in lung cancer.

In total, data from 84 presentations will illustrate the progress of AstraZeneca’s Oncology pipeline, with 28 abstracts reporting new Immuno-Oncology (IO) data, 33 focused on complementary biological pathways exploring the DDR mechanism, and 20 on tumour drivers and resistance mechanisms.

José Baselga, Executive Vice President, Research & Development, Oncology, said: "AstraZeneca is continuing to strengthen its portfolio of innovative cancer medicines by exploring new indications and developing a pioneering next-generation pipeline. We will be sharing some of our latest research at the 2019 AACR (Free AACR Whitepaper) Annual Meeting, including 28 new molecular entities and six combinations, highlighting an exciting new phase of scientific discovery from our Oncology R&D."

IO pipeline explores new pathways to enhance immune response

Key data from AstraZeneca’s early-stage clinical and preclinical IO pipeline include potential new medicines targeting multiple pathways, providing insight into novel mechanisms to boost current immune response and modify the tumour microenvironment both alone and in combination with checkpoint inhibition.

Presentations will highlight AstraZeneca’s growing portfolio in small molecules and antisense oligonucleotides (ASOs) targeting immunosuppressive mechanisms in cancer, and the Company’s exploration of the adenosine pathway, which is increasingly recognised as critical to tumour suppression and represents a new frontier within IO.

Phase I and preclinical data demonstrating AZD4635, a small molecule A2AR antagonist, prevents adenosine-mediated immunosuppression. Early clinical activity has been observed with AZD4635 monotherapy or in combination with Imfinzi in patients with metastatic castration-resistant prostate cancer (Abstracts #LB-192/10, #CT026/21)
Preclinical research on the activity of ASO AZD8701 (ION-AZ7), showing that inhibiting FOXP3 may limit immunosuppressive functions and induce tumour regression (Abstract #2713)
Preclinical pharmacodynamic and mechanistic data illustrating how danvatirsen (AZD9150), a STAT3 ASO reverses immunosuppression to produce significant anti-tumour effects, both as monotherapy and in combination with anti-PDL1 (Abstract #3215/25)
Preclinical data for MEDI5083, a novel fusion protein which activates the CD40 pathway, indicating robust immune activation and anti-tumour activity (Abstract #1534/3)
Preclinical data for MEDI1191, a novel IL12-based treatment designed for injection directly into tumours, showing potential to drive anti-tumour response for patients with solid tumours both as monotherapy and in combination with anti-PDL1 (Abstract #5017/11)
Next wave of DDR targets

New research will showcase AstraZeneca’s DDR pipeline beyond PARP inhibition, including on the discovery and first structural disclosure of AZD7648, a potent and selective DNA-PK inhibitor (Abstract #DDT01-02). DNA-PK is critical in repairing DNA double strand breaks through the non-homologous end joining (NHEJ) pathway and has also been linked to the replication stress response (RSR), a type of DDR.

Additional new data will be shared on reversing PARP inhibitor resistance by targeting alternative DDR dependencies, like the RSR (Abstract #932).

Predicting response and addressing treatment resistance in lung cancer: new insights from MYSTIC and TATTON trials for Imfinzi and Tagrisso

Exploratory analyses of blood and tissue tumour mutational burden (TMB) from the Phase III MYSTIC trial assess TMB as a potential biomarker of survival in 1st-line use of Imfinzi with or without tremelimumab vs. chemotherapy in metastatic non-small cell lung cancer (NSCLC) (Abstract #CT074). The trial did not meet the primary endpoints, but Imfinzi monotherapy demonstrated clinical activity in the primary analysis. The MYSTIC trial provides the most comprehensive data set to date evaluating blood TMB and is the only Phase III randomised, controlled trial to demonstrate an association between high TMB and overall survival benefit with immunotherapy treatment. This new analysis will further evaluate the use of TMB as a potential biomarker predictive of survival benefit with IO treatment.

In addition, several abstracts will be presented from the TATTON Phase Ib trial testing the combination of Tagrisso with potential new medicines savolitinib or selumetinib in NSCLC patients who have progressed on prior epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) treatment (Abstracts #CT032, #CT033, #CT034). The TATTON trial supports SAVANNAH, an ongoing Phase II clinical trial exploring the combination of Tagrisso and savolitinib to overcome MET-driven EGFR-TKI resistance following treatment with Tagrisso in EGFR-mutated (EGFRm) NSCLC. Characterisation of detection methods for identifying MET-driven EGFR-TKI resistance will be presented (Abstract #4897/20). TATTON will also inform the trial design of ORCHARD, a Phase II platform trial exploring potential new treatment options to address resistance mechanisms in patients with EGFRm NSCLC who have experienced disease progression following 1st-line treatment with Tagrisso.

Key AstraZeneca presentations at AACR (Free AACR Whitepaper) 2019

Lead author

Abstract title

Presentation details

Immuno-Oncology

Peters, S

Tumour mutational burden (TMB) as a biomarker of survival in metastatic non-small cell lung cancer (mNSCLC): blood and tissue TMB analysis from MYSTIC, a Phase 3 study of first-line durvalumab ± tremelimumab vs chemotherapy

Abstract #CT074

Session CTPL03 – Optimizing PD-1/PD-L1 Immune Checkpoint Inhibitor Therapy: Dedicated to the Memory of Waun Ki Hong

Monday 1 April

10:30am-12:45pm

Marcus Auditorium- Bldg A-GWCC

Srinivasan, S

STAT3 ASO reverses immunosuppression and enhances cytotoxic cell function to enhance PDL1 blockade

Abstract #3215/25

Session PO.IM02.01 – Combination Immunotherapies 2

Tuesday 2 April

8:00am-12:00pm

Section 23

Barbon, CM

The A2AR antagonist AZD4635 prevents adenosine-mediated immunosuppression of CD103+ dendritic cells

Abstract #LB-192/10

Session LBPO.IM02 – Late-Breaking Research:
Immunology 2

Tuesday 2 April

8:00am-12:00pm

Section 42

Merchant, MS

Evidence of Immune Activation in the first-in-human Phase 1a dose escalation study of the Adenosine 2a Receptor Antagonist, AZD4635, in patients with advanced solid tumors

Abstract #CT026/21

Session PO.CT01 – Phase I Clinical Trials: Part 1

Sunday 31 March

1:00pm-5:00pm

Section 16

Sinclair, C

Discovery and characterization of AZD8701, a high affinity antisense oligonucleotide targeting FOXP3 to relieve immunosuppression in cancer

Abstract #2713

Session MS.IM02.01 – Rational Combinations of Immunotherapy

Monday 1 April

4:35pm-4:50pm

Room A411-Georgia World CC

Wang, Y

MEDI5083, a novel CD40L-Fc fusion protein, activates the CD40 pathway on antigen presenting cells and promotes a robust anti-tumor immune response in a B16F10 murine tumor model

Abstract #1534/3

Session PO.IM02.17 – Therapeutic Antibodies 2

Monday 1 April

8:00am-12:00pm

Section 25

Luheshi, N

MEDI1191, a novel IL-12 mRNA therapy for intratumoral injection to promote TH1 transformation of the patient tumor microenvironment

Abstract #5017/11

Session PO.IM02.10 – Tumor Immune Microenvironment

Wednesday 3 April

8:00am-12:00pm

Section 25

DNA damage response

Goldberg, FW

Discovery and first structural disclosure of AZD7648, a potent and selective DNA-PK inhibitor

Abstract #DDT01-02

Session DDT01 – New Drugs on the Horizon: Part 1

Sunday 31 March

1:24pm-1:48pm

Room A305 – Georgia World CC

Cadogan, EB

AZD7648: A potent and selective inhibitor of DNA-PK with pharmacodynamic and monotherapy anti-tumour activity

Abstract #3505/16

Session PO.MCB07.03 – DNA Damage and Repair 3

Tuesday 2 April

8:00am-12:00pm

Section 35

Fok, J

AZD7648, a potent and selective inhibitor of DNA-PK, potentiates the activity of ionising radiation and doxorubicin in vitro and causes tumour regression in xenograft models

Abstract #3512/23

Session PO.MCB07.03 – DNA Damage and Repair 3

Tuesday 2 April

8:00am-12:00pm

Section 35

O’Connor, M

Reversing PARP inhibitor resistance by targeting the replication stress response

Abstract #932

Session MS.ET03.01 – Drug Resistance

Sunday 31 March

4:35pm-4:50pm

Room B304 – Georgia World CC

Tumour drivers and resistance

Yu, H

TATTON Phase Ib expansion cohort: osimertinib plus savolitinib for patients (pts) with EGFR-mutant, MET amplified NSCLC after progression on prior first/second-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI)

Abstract #CT032

Session CTPL02 – Can the Challenge of NSCLC Resistance be MET or Will We Not MEK It?

Sunday 31 March

3:00pm-5:15pm

Marcus Auditorium- Bldg A-GWCC

Sequist, L

TATTON Phase Ib expansion cohort: osimertinib plus savolitinib for patients (pts) with EGFR-mutant, MET-amplified NSCLC after progression on prior third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI)

Abstract #CT033

Session Clinical Trials Plenary, Can the Challenge of NSCLC Resistance be MET or Will We Not MEK it?

Sunday 31 March

3:00pm-5:15pm

Marcus Auditorium- Bldg A-GWCC

Ramalingam, S

Osimertinib plus selumetinib for patients (pts) with EGFR-mutant (EGFRm) NSCLC following disease progression on an EGFR-TKI: results from the Phase Ib TATTON study

Abstract #CT034

Session Clinical Trials Plenary, Can the Challenge of NSCLC Resistance be MET or Will We Not MEK it?

Sunday 31 March

3:00pm-5:15pm

Marcus Auditorium- Bldg A-GWCC

Hartmaier, RJ

Detection of MET-mediated EGFR tyrosine kinase inhibitor (TKI) resistance in advanced non-small cell lung cancer (NSCLC): biomarker analysis of the TATTON study

Abstract #4897/20

Session PO.CL11.07 – Novel Strategies for Biomarker Identification and Use in Cancer 3

Wednesday 3 April

8:00am-12:00pm

Section 19

Liquid biopsies

Barrett, JC

Regulatory Considerations for Utilizing Liquid Biopsies in Drug and Diagnostic Development

Major Symposium: Regulatory Science and Policy

Session SYPOL07 – Regulatory Considerations for Utilizing Liquid Biopsies in Drug and Diagnostic Development

Tuesday 2 April

3:00pm-5:00pm

Room A402 – Georgia World CC

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance Oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On March 14, 2019 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel tetracyclines to treat serious and life-threatening conditions, reported financial results for the fourth quarter and year ended December 31, 2018, provided an overview of recent achievements, and highlighted key milestones for 2019 (Press release, Tetraphase, MAR 14, 2019, View Source [SID1234534362]).

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"We capped off 2018 with a strong finish, with the regulatory approvals of XERAVA (eravacycline) for the treatment of complicated intra-abdominal infections (cIAI) in both the U.S. and Europe, followed by the commercial launch of XERAVA in the U.S. in mid-October," said Guy Macdonald, President and Chief Executive Officer of Tetraphase. "With the U.S. launch off to a solid start, XERAVA is now available for use in U.S. hospitals and healthcare institutions for the treatment of a range of patients with empiric and confirmed cIAI. We are encouraged by the progress we have made during our first few months of launch, with more than 400 formulary reviews already completed or scheduled to occur by mid-year and a re-ordering rate for XERAVA above 70 percent. Further, as we look to the balance of 2019, we have made the decision to delay launching XERAVA in the EU5 independently and will instead continue to focus our resources on building momentum and supporting a successful launch in the U.S."

Mr. Macdonald continued, "With respect to our pipeline, we expect to complete our bronchopulmonary disposition study for TP-6076, targeted against Acinetobacter baumannii and other multidrug-resistant pathogens, later this year and look forward to presenting preclinical data on our new acute myeloid leukemia (AML) pipeline candidate, TP-2846, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Atlanta."

Key Milestones for 2019

Complete 400 formulary reviews for XERAVA by mid-year
Present preclinical data on TP-2846 at the AACR (Free AACR Whitepaper) Annual Meeting – 2Q 2019
Everest Medicines to begin Phase 3 clinical trial of eravacycline in cIAI in China – 2Q 2019
Complete bronchopulmonary disposition study for TP-6076 – 2H 2019
Fourth Quarter and Recent Highlights

Commercially Launched XERAVA for cIAI in the U.S.
The Company commercially launched XERAVA in the U.S. in mid-October 2018. XERAVA is now available for use in hospitals and healthcare institutions for the treatment of a range of patients with empiric and confirmed cIAI. The salesforce is focusing on institutions responsible for treating 90 percent of patients with Gram-negative infections. Currently, three antimicrobial susceptibility tests (ASTs) have been approved for commercial use including the Eravacycline Mast Disk by Hardy Diagnostics; the Eravacycline MIC Strip by Liofilchem, Inc.; and the Sensititre Plate by Thermo Fisher Scientific, Inc. Previously, more than 200 healthcare institutions ordered ASTs for research-use-only purposes.
Received Marketing Authorization from the European Commission (EC) for XERAVA in the European Union (EU)
In September, the EC adopted the July 2018 positive opinion issued by the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) to grant marketing authorization for XERAVA for the treatment of cIAI in adults in all EU member states as well as Iceland, Liechtenstein and Norway.
Entered into Loan and Security Agreement with Solar Capital Limited
In November 2018, the Company entered into a loan agreement with Solar Capital Limited, providing up to $75 million, with $30 million funded at closing, which is being used to support the commercial launch of XERAVA and for general corporate purposes.
Announced New Preclinical Data on TP-2846 for AML to be Presented at the 2019 AACR (Free AACR Whitepaper) Annual Meeting
In February 2019, Tetraphase announced it will present three posters on TP-2846, the Company’s newly revealed pipeline candidate for AML, at the 2019 AACR (Free AACR Whitepaper) Annual Meeting taking place March 29 – April 3 at the Georgia World Congress Center in Atlanta. The poster presentations will include in vitro and in vivo data supporting TP-2846’s potential as a novel tetracycline antileukemia agent.
Approval of Everest Medicines’ Investigational New Drug (IND) Application for Eravacycline in cIAI by the China National Medical Products Administration (NMPA)
In June 2018, Everest Medicines, a C-Bridge Capital-backed biopharmaceutical company, which has the exclusive license to develop and commercialize eravacycline in China, Taiwan, Hong Kong, Macau, South Korea and Singapore, submitted an IND application to the China NMPA, formerly the China FDA. The IND was approved by China NMPA, and Everest expects to begin enrolling patients in its Phase 3 study of eravacycline in cIAI in the second quarter of 2019.
Presented XERAVA and TP-6076 Data at the Infectious Disease Society of America’s (IDSA) 2018 IDWeek
In October 2018, the Company presented data related to XERAVA and TP-6076 at IDSA’s IDWeek. Among the data presented were results from a post-hoc analysis of XERAVA Phase 3 data, which showed high clinical cure and microbiological eradication rates with XERAVA among patients with cIAI and concurrent bacteremia. Data from a Phase 1 randomized, placebo-controlled, double-blind, multiple-ascending-dose study demonstrating positive safety, tolerability and pharmacokinetic results for the Company’s novel, fully synthetic tetracycline, TP-6076, were also presented.
Presented XERAVA Data at the American College of Clinical Pharmacy (ACCP) 2018 Global Conference
At the ACCP 2018 Global Conference in October, the Company announced positive data from a post-hoc analysis of two Phase 3 trials of XERAVA in cIAI in higher-risk populations – obese patients and those with altered renal function. Similar clinical cure rates were observed for XERAVA in these populations across all classifications of renal function, further supporting that the drug is an effective empiric treatment for cIAI and may provide an alternative to antibiotics that require dosing modification in patients with altered renal function. XERAVA was also effective regardless of body mass index when dosed 1mg/kg IV every 12 hours, based on total body weight when compared to carbapenems.
Fourth Quarter and Full-Year 2018 Financial Results

As of December 31, 2018, Tetraphase had cash and cash equivalents of $107.8 million and 53.7 million shares outstanding. The Company expects that its cash and cash equivalents, as well as expected revenue, will be sufficient to fund operations into the third quarter of 2020.

For the fourth quarter of 2018, Tetraphase reported a net loss of $21.5 million, or $0.40 per share, compared to a net loss of $23.5 million, or $0.46 per share, for the same period in 2017. For the year ended December 31, 2018, Tetraphase reported a net loss of $72.2 million, or $1.37 per share, compared to a net loss of $114.8 million, or $2.63 per share, for the same period in 2017.

XERAVA product revenue for the fourth quarter and year ended December 31, 2018 was $178,000, reflecting sales of the product which was commercially introduced in the U.S. in mid-October 2018.

Total revenues were $4.3 million for the fourth quarter of 2018, compared to $2.5 million for the same period in 2017. Total revenues were $18.9 million for the year ended December 31, 2018, compared to $9.7 million for the same period in 2017. Total revenues for the fourth quarter and year ended December 31, 2018 consisted of XERAVA product revenue, license and collaboration revenue from the Company’s relationship with Everest Medicines and government contract revenue. The increases in total revenues for the fourth quarter and year ended December 31, 2018 compared to the same prior-year periods were primarily due to the collaboration and XERAVA revenue, neither of which existed in 2017.

Research and development (R&D) expenses for the fourth quarter of 2018 were $10.7 million, compared to $18.5 million for the same period in 2017. R&D expenses for the year ended December 31, 2018 were $54.9 million, compared to $101.7 million for the same period in 2017. The decreases in R&D expenses for the fourth quarter and year ended December 31, 2018 compared to the same prior-year periods were primarily due to lower clinical trial costs associated with the IGNITE Phase 3 clinical trials, which concluded in the first quarter of 2018, and a decrease in chemistry, manufacturing and controls (CMC) expenses for XERAVA.

Selling, general and administrative (SG&A) expenses for the fourth quarter of 2018 were $14.7 million, compared to $7.9 million for the same period in 2017. SG&A expenses for the year ended December 31, 2018 were $37.1 million, compared to $23.7 million for the same period in 2017. The increases in SG&A expenses for the fourth quarter and year ended December 31, 2018 compared to the same prior-year periods were primarily due to an increase in commercial launch-related expenses for XERAVA and related G&A infrastructure investments.

Conference Call and Webcast Information

Tetraphase will host a conference call today at 4:30 p.m. ET to discuss its financial results and provide an update on the Company. The call can be accessed by dialing 844-831-4023 (U.S. and Canada) or 731-256-5215 (international) and entering conference ID number 4187876. To access the live audio webcast, visit the "Investors — Events & Presentations" section of the Tetraphase website at www.tphase.com.

A replay of the conference call will be available from 7:30 p.m. ET on Thursday, March 14, 2019, through 7:30 p.m. ET on Thursday, March 21, 2019 and by dialing 855-859-2056 (U.S. and Canada) or 404-537-3406 (international). The conference ID number is 4187876. A replay of the webcast will be available on Tetraphase’s website for 90 days.

About XERAVATM

XERAVA (eravacycline for injection) is a tetracycline class antibacterial indicated for the treatment of complicated intra-abdominal infections (cIAI) in patients 18 years of age and older. It is approved for use in the U.S. and Europe. XERAVA was investigated for the treatment of cIAI as part of the Company’s IGNITE (Investigating Gram-Negative Infections Treated with Eravacycline) Phase 3 program. In the first pivotal Phase 3 trial in patients with cIAI, twice-daily intravenous (IV) eravacycline met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to ertapenem and was well-tolerated. In the second Phase 3 clinical trial in patients with cIAI, twice-daily IV eravacycline met the primary endpoint by demonstrating statistical non-inferiority of clinical response compared to meropenem and was well-tolerated. In both trials, XERAVA achieved high cure rates in patients with Gram-negative pathogens, including resistant isolates.

XERAVATM Important Safety Information

XERAVA is a tetracycline class antibacterial indicated for the treatment of complicated intra-abdominal infections in patients 18 years of age and older.

XERAVA is not indicated for the treatment of complicated urinary tract infections.

To reduce the development of drug-resistant bacteria and maintain the effectiveness of XERAVA and other antibacterial drugs, XERAVA should be used only to treat or prevent infections that are proven or strongly suspected to be caused by susceptible bacteria.

XERAVA is contraindicated for use in patients with known hypersensitivity to eravacycline, tetracycline-class antibacterial drugs or to any of the excipients. Life-threatening hypersensitivity (anaphylactic) reactions have been reported with XERAVA.

The use of XERAVA during tooth development (last half of pregnancy, infancy and childhood to the age of eight years) may cause permanent discoloration of the teeth (yellow-gray-brown) and enamel hypoplasia.

The use of XERAVA during the second and third trimester of pregnancy, infancy and childhood up to the age of eight years may cause reversible inhibition of bone growth.

Clostridium difficile associated diarrhea (CDAD) has been reported with use of nearly all antibacterial agents and may range in severity from mild diarrhea to fatal colitis.

The most common adverse reactions observed in clinical trials (incidence ≥ 3%) were infusion site reactions, nausea, and vomiting.

XERAVA is structurally similar to tetracycline-class antibacterial drugs and may have similar adverse reactions. Adverse reactions including photosensitivity, pseudotumor cerebri, and anti-anabolic action which has led to increased blood urea nitrogen, azotemia, acidosis, hyperphosphatemia, pancreatitis, and abnormal liver function tests, have been reported for other tetracycline-class antibacterial drugs, and may occur with XERAVA. Discontinue XERAVA if any of these adverse reactions are suspected.

To report SUSPECTED ADVERSE REACTIONS, contact Tetraphase Pharmaceuticals Inc., at 1-833-7-XERAVA (1-833-793-7282) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full prescribing information for XERAVA.

About Tetraphase Pharmaceuticals, Inc.

Tetraphase Pharmaceuticals, Inc., is a biopharmaceutical company using its proprietary chemistry technology to create novel tetracyclines for serious and life-threatening conditions, including bacterial infections caused by many of the multidrug-resistant bacteria highlighted as urgent public health threats by the World Health Organization and the Centers for Disease Control and Prevention. The Company has created more than 3,000 novel tetracycline compounds using its proprietary technology platform. Tetraphase’s lead product XERAVATM is approved for the treatment of complicated intra-abdominal infections by the U.S. Food and Drug Administration and the European Medicines Agency. The Company’s pipeline also includes TP-271 and TP-6076, which are in Phase 1 clinical trials, and TP-2846, which is in preclinical testing for acute myeloid leukemia. Please visit www.tphase.com for more company information.

On March 14, 2019 Novelion Therapeutics Inc. (NASDAQ: NVLN), a biopharmaceutical company dedicated to developing and commercializing therapies for individuals living with rare diseases ("Novelion" or the "Company"), today reported financial results for the fourth quarter and year ended December 31, 2018 (Press release, QLT, MAR 14, 2019, View Source [SID1234534329]).

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Novelion’s Interim Chief Executive Officer Ben Harshbarger commented, "We are pleased with our fourth quarter results which reflect strong revenue performance and show meaningful impact from the cost reduction initiatives executed throughout 2018. We remain focused on undertaking a comprehensive capital restructuring and also on delivering our two very important rare disease therapies to indicated patients in need."

Business Update

Following marketing authorization of MYALEPTA (metreleptin) for generalized lipodystrophy (GL) and partial lipodystrophy (PL) by the EMA in July, our subsidiary Aegerion Pharmaceuticals commenced the pricing and reimbursement processes in key EU markets. Reimbursement decisions in many of the key EU markets are anticipated throughout 2019. MYALEPTA sales growth in 2018 was supported by named patient sales programs, which allow for sales on an unsolicited basis prior to regulatory approval and/or pricing and reimbursement decisions, as well as the launch of MYALEPTA in Germany in the fourth quarter.

In February 2019, Aegerion entered into an exclusive licensing agreement with Recordati Rare Diseases Inc. ("Recordati") for the commercialization of JUXTAPID (lomitapide) in Japan. The agreement includes exclusive rights in Japan for Recordati to commercialize JUXTAPID for the current approved indication, homozygous familial hypercholesterolemia (HoFH). The terms of, and use of proceeds from, the Recordati licensing agreement are described more fully in Novelion’s Form 8-K filed on February 6, 2019.

In January 2019, Aegerion held a meeting with the U.S. Food and Drug Administration (FDA) to obtain feedback on the design of the placebo-controlled study that will be required in order to pursue the PL indication for MYALEPT in the U.S. The Company is assessing feedback on the study design and integrating it into the Phase 3 study protocol.

Aegerion plans to file for regulatory approvals for metreleptin in GL and PL in certain key markets outside the U.S. and EU, including Brazil, in 2019.

As previously announced, Novelion and Aegerion have each engaged advisors to independently explore and advise them on all available strategic alternatives regarding the Company’s capital structure, such as a restructuring of Aegerion’s Convertible Notes due August 2019 (including a restructuring that would likely involve a debt for equity swap), a sale or merger of Novelion or Aegerion, or the sale or other disposition of certain businesses or assets. The implementation of one or more of such transactions (or the failure to complete any such transaction or transactions) will likely require Aegerion, and could require Novelion, to seek the protections of applicable bankruptcy laws allowing for corporations to seek to restructure their debts and other affairs under a reorganization.
Fourth Quarter 2018 Financial Results

JUXTAPID: Novelion reported net revenues of JUXTAPID of $15.2 million in the fourth quarter of 2018, compared to $20.1 million for the same period in 2017, $8.2 million, or 53.9%, of which was from prescriptions written in the U.S. and $0.8 million of which was royalty revenue from sales of JUXTAPID in the EMEA region. The JUXTAPID revenue decline in the fourth quarter of 2018 compared to the same period in 2017 was primarily due to product competition for JUXTAPID, restrictions on reimbursement, and patient discontinuation from therapy.

MYALEPT: Novelion reported net revenues of MYALEPT of $25.5 million in the fourth quarter of 2018, compared to $18.8 million for the same period in 2017, $13.4 million, or 52.5%, of which was from prescriptions written in the U.S. MYALEPT revenue growth in the fourth quarter was driven by increased sales in all key markets.

GAAP total net revenues for the fourth quarter of 2018 were $40.7 million compared to $38.9 million for the same period in 2017.

Cost of product sales for the fourth quarter of 2018 was $18.0 million compared to $17.0 million for the same period in 2017, resulting in stable year-over-year fourth quarter gross margins.

GAAP total operating expenses for the fourth quarter of 2018 were $21.7 million compared to total operating expenses of $35.9 million, a 39.6% reduction compared to the same period in 2017. GAAP SG&A expenses were $14.1 million in the fourth quarter of 2018 compared to $24.1 million for the same period in 2017. GAAP R&D expenses were $7.7 million in the fourth quarter of 2018 compared to $11.8 million for the same period in 2017.

On a non-GAAP basis, during the fourth quarter of 2018, SG&A expenses were $13.0 million compared to $22.5 million for the same period in 2017. The 42.2% decrease in non-GAAP SG&A expenses in the fourth quarter of 2018 compared with the same period in 2017 was primarily related to cost reduction initiatives executed throughout 2018.

On a non-GAAP basis, during the fourth quarter of 2018, R&D expenses decreased 33.6% to $7.7 million compared to $11.6 million for the same period in 2017, reflecting cost reduction initiatives executed throughout 2018.

GAAP net loss in the fourth quarter of 2018 was $19.4 million, an improvement of approximately 21.1% compared to GAAP net loss of $24.6 million during the same period in 2017.

On a non-GAAP basis, net income was $2.9 million in the fourth quarter of 2018 compared to a net loss of $3.3 million for the same period in 2017.

A full reconciliation of the GAAP financial results to non-GAAP financial results is included in the financial information tables below.

Full Year 2018 Financial Results

JUXTAPID: Novelion reported net revenues of JUXTAPID of $59.1 million for the year ended December 31, 2018, compared to $72.1 million for 2017. The decline in sales resulted from product competition for JUXTAPID, restrictions on reimbursement, and patient discontinuation from therapy. Named patient sales of JUXTAPID in Brazil totaled $0.4 million in 2018, compared to $6.7 million of JUXTAPID named patient sales in Brazil in 2017. Revenue growth in Japan in 2018 helped offset the sales decline in the U.S. and Brazil.

MYALEPT: Novelion reported net revenues of MYALEPT of $71.4 million for the year ended December 31, 2018, compared to $66.3 million for 2017. The increase was primarily attributable to revenues in France, Germany and Turkey. In addition, 2017 MYALEPT sales benefited from $2.3 million of one-time deferred revenue recognition in the U.S.

GAAP total net revenues for the year ended December 31, 2018 were $130.4 million compared to $138.4 million for 2017.

Cost of product sales for the year ended December 31, 2018 was $59.7 million compared to $77.2 million for 2017, resulting in improved gross margin. The improvement in 2018 was primarily a result of higher reserves in 2017 for excess and obsolete inventory which were charged to cost of product sales, partially offset by a higher royalty rate on U.S. sales of metreleptin in 2018.

GAAP total operating expenses for the year ended December 31, 2018 were $120.8 million compared to total operating expenses of $148.0 million for 2017. GAAP SG&A expenses were $79.8 million for the year ended December 31, 2018 compared to $96.5 million for 2017. GAAP R&D expenses were $38.8 million for the year ended December 31, 2018 compared to $49.0 million for 2017.

On a non-GAAP basis, for the year ended December 31, 2018, SG&A expenses decreased 20.0% to $72.6 million compared to $90.7 million for 2017, primarily as a result of cost reduction initiatives executed throughout 2018. Restructuring charges for 2018 were $2.2 million, compared with restructuring charges of $2.5 million for 2017.

On a non-GAAP basis, for the year ended December 31, 2018, R&D expenses decreased 20.5% to $38.3 million compared to $48.2 million for 2017 due primarily to cost reduction initiatives executed throughout 2018.

GAAP net loss for the year ended December 31, 2018 was $108.3 million compared to GAAP net loss of $126.7 million for 2017.

Net loss on a non-GAAP basis for the year ended December 31, 2018 was $25.3 million, compared to $30.0 million for 2017.

As of December 31, 2018, the Company’s consolidated unrestricted cash balance was $45.2 million, compared to $55.4 million at December 31, 2017. Novelion’s consolidated cash as of December 31, 2018 includes $13.3 million at Novelion and $31.9 million at the Aegerion subsidiary level.

Debt and Government Settlement Payments

As of December 31, 2018, Aegerion’s debt liabilities and government settlement payments included $302.5 million in outstanding principal under Aegerion’s Convertible Notes due August 15, 2019, $75.9 million in outstanding principal (including paid in kind fees and interest) under Aegerion’s secured term loans having a maturity date of June 30, 2019, $37.1 million outstanding under Aegerion’s secured intercompany term loan with Novelion, as lender, which has a maturity date of July 1, 2019 (which term loan amounts were subsequently reduced by repayments received from the Recordati license transaction described above), as well as $31.1 million owed under Aegerion’s settlements with the Department of Justice and the U.S. Securities and Exchange Commission (the "Commission"), payable in prescribed installments until the first quarter of 2021.

Financial Guidance

Novelion expects total net revenues in 2019 to be between $160.0 and $175.0 million, including $30.0 million of licensing revenues, in the form of the $25.0 million upfront licensing payment and $5.0 payment upon transfer of the marketing authorization to Recordati, resulting from the Recordati transaction.