On November 8, 2018 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on unmet medical needs in inflammation, reported operating results for the third quarter ended September 30, 2018 and provided pipeline updates (Press release, AnaptysBio, NOV 8, 2018, View Source [SID1234531162]).

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"We continued to advance the clinical development of our wholly-owned etokimab and ANB019 programs for severe inflammatory disease indications during the third quarter of 2018," said Hamza Suria, president and chief executive officer of AnaptysBio. "Top-line data from our etokimab Phase 2a trial in severe adult eosinophilic asthma patients demonstrated rapid and sustained improvement in Forced Exhaled Volume In One Second versus placebo, with corresponding reduction in blood eosinophil levels. We look forward to further advancement of our wholly-owned pipeline with four additional readouts from ongoing clinical trials of etokimab and ANB019 during 2019."

Etokimab (ANB020 Anti-IL-33 Program)
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In September, the Company announced positive topline proof-of-concept data for etokimab, its investigational anti-IL-33 therapeutic antibody, in an ongoing single dose Phase 2a clinical trial in adult patients with severe eosinophilic asthma. Patients administered with etokimab rapidly improved their Forced Exhaled Volume In One Second, or FEV1, which is a measure of lung function, with an eight percent FEV1 improvement over placebo at Day 2. FEV1 improvement was sustained through Day 64, with an 11 percent increase over placebo. Blood eosinophil reduction was sustained through the interim analysis period, with a 31 percent reduction at Day 2 and a 46 percent reduction at Day 64 over placebo, which was consistent with FEV1 improvement observed in this trial. Etokimab was generally well tolerated in all patients and no serious adverse events were reported as of this interim analysis. This Phase 2a trial is currently ongoing and the company plans to report full data from this trial at a medical conference in 2019 following trial completion. AnaptysBio plans to continue development of etokimab in eosinophilic asthma with a multi-dose Phase 2b randomized, double-blinded, placebo-controlled trial, which is expected to be initiated in 2019.
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The Company is enrolling a Phase 2b randomized, double-blinded, placebo-controlled, multi-dose study in 300 adult patients with moderate-to-severe atopic dermatitis, also referred to as the ATLAS clinical trial, to assess different dose levels and dosing frequencies of subcutaneously-administered etokimab for a 16-week treatment period followed by an eight-week monitoring period, with data expected in the second half of 2019.
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The Company has cleared an IND and initiated a randomized, placebo-controlled Phase 2 trial, also referred to as the ECLIPSE trial, in approximately 100 adult patients with chronic rhinosinusitis with nasal polyps. Patients will be treated with two multi-dosing frequencies of subcutaneous-administered etokimab or placebo, each in combination with mometasone furoate nasal spray as background therapy, for a treatment period of 16 weeks followed by an eight-week monitoring period. The Company anticipates top-line data from the ECLIPSE trial will be available in the second half of 2019.

ANB019 (Anti-IL-36 Receptor Program)
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AnaptysBio has initiated a 10-patient, single arm, open-label Phase 2 trial of ANB019 in generalized pustular psoriasis, or GPP, also known as the GALLOP trial, and top-line data are anticipated in mid-2019.

All patients will be treated with an intravenous loading dose of ANB019 upon enrollment, followed by subcutaneously-administered monthly doses of ANB019 for a treatment period of up to 16 weeks post enrollment and followed by an eight-week monitoring period.
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AnaptysBio has cleared an IND with the FDA and has initiated a randomized, placebo-controlled 50-patient multi-dose Phase 2 trial in palmoplantar pustulosis, or PPP, also known as the POPLAR trial, where top line data are anticipated in the second half of 2019. Patients will be treated with a (i) a subcutaneously-administered loading dose of ANB019 upon enrollment, followed by subcutaneously-administered monthly doses of ANB019, or (ii) placebo, each for a treatment period of 16 weeks post enrollment and followed an eight-week monitoring period.
Corporate Highlights
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On September 28, 2018, the Company completed an underwritten public offering of 2,530,000 shares of common stock at a price to the public of $94.46, which included the exercise by the underwriters of their option to purchase an additional 330,000 shares of common stock. AnaptysBio, received net proceeds from the offering of $227.5 million, after deducting underwriting discounts and commissions.
Third Quarter Financial Results
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Cash, cash equivalents and investments totaled $512.4 million as of September 30, 2018 compared to $324.3 million as of December 31, 2017, for an increase of $188.1 million. The increase primarily relates to net proceeds received by the Company of $227.5 million from the public offering, offset by operating cash outflow for clinical and manufacturing related expenses, as well as personnel costs.
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Collaboration revenue was $5.0 million for the three and nine months ended September 30, 2018, related to a milestone for the first Phase 3 trial of TSR-042 by TESARO compared to no revenue and $7.0 million for two TESARO milestones for the three and nine months ended September 30, 2017, respectively.
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Research and development expenses were $17.9 million for the three months ended September 30, 2018, as compared to $6.7 million for the three months ended September 30, 2017. The increase was primarily due to continued advancement of the Company’s etokimab and ANB019 clinical programs and additional personnel-related expenses including share based compensation during the three months ended September 30, 2018.
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General and administrative expenses were $4.0 million for the three months ended September 30, 2018, as compared to $2.4 million for the three months ended September 30, 2017. The increase was primarily attributable to additional personnel-related expenses, including share based compensation, to support the Company’s growth.

Financial Guidance
AnaptysBio expects that its cash, cash equivalents and investments will fund its current operating plan at least through the end of 2020.
About Etokimab
Etokimab, previously referred to as ANB020, is an antibody that potently binds and inhibits the activity of interleukin-33, or IL-33, a pro-inflammatory cytokine that multiple studies have indicated is a central mediator of atopic diseases, which AnaptysBio believes is broadly applicable to the treatment of atopic inflammatory disorders, such as atopic dermatitis, eosinophilic asthma, chronic rhinosinusitis with nasal polyps, or CRSwNP, and potentially other allergic conditions. Following completion of a healthy volunteer Phase 1 trial of etokimab, AnaptysBio continued clinical development of etokimab into a Phase 2a trial for moderate-to-severe adult atopic dermatitis and a placebo-controlled Phase 2a trial in severe adult eosinophilic asthma patients. AnaptysBio is enrolling its ATLAS trial, a randomized, double-blinded, placebo-controlled multi-dose Phase 2b clinical trial of etokimab in 300 moderate-to-severe adult atopic dermatitis patients where data is anticipated in the second half of 2019. The company has also initiated its ECLIPSE trial, a randomized, double-blinded, placebo-

controlled Phase 2 trial of etokimab in approximately 100 adult patients with CRSwNP with data anticipated in the second half of 2019. AnaptysBio also plans to initiate a randomized, double-blinded, placebo-controlled, multi-dose Phase 2b trial of etokimab in patients with eosinophilic asthma in 2019.
About ANB019
ANB019 is an antibody that inhibits the function of the interleukin-36-receptor, or IL-36R, which AnaptysBio plans to initially develop as a potential first-in-class therapy for patients suffering from generalized pustular psoriasis, or GPP and palmoplantar pustulosis, or PPP. AnaptysBio conducted a Phase 1 clinical trial in healthy volunteers, where 54 subjects are dosed with ANB019 and 18 are dosed with placebo in single and multi-dose cohorts at various subcutaneous and intravenously administered dose levels. In May 2018, AnaptysBio presented data from this Phase 1 clinical trial, which demonstrated favorable safety, pharmacokinetics and pharmacodynamic properties that support advancement of ANB019 into Phase 2 studies. AnaptysBio is enrolling its GALLOP trial, a Phase 2 study of ANB019 in GPP where data is anticipated in the second quarter of 2019, and have initiated its POPLAR trial, a Phase 2 study in PPP where data is anticipated in the second half of 2019.

On November 8, 2018 Merus N.V. (Nasdaq:MRUS), a clinical-stage immuno-oncology company developing innovative bispecific antibody therapeutics (Biclonics), reported that Ton Logtenberg, Ph.D., President and Chief Executive Officer of Merus, will present a company overview at the Jefferies 2018 London Healthcare Conference on Thursday, November 15, 2018, at 3:20 p.m. GMT (Press release, Merus, NOV 8, 2018, View Source;p=RssLanding&cat=news&id=2376165 [SID1234532116]).

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A live webcast of the presentation will be available on the Investors and Media page of the Company’s website, View Source A replay of the presentation will be archived and available on the Merus website site for a limited time following the event.

On November 8, 2018 Actym Therapeutics, a privately held biotechnology company focused on therapeutic targeting of intractable immune pathways in the tumor microenvironment, reported that it will be presenting data at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in Washington, D.C. from November 9-11, 2018 (Press release, Actym Therapeutics, NOV 8, 2018, View Source [SID1234530898]). The presentation is titled "STACT-TREX1: A novel tumor-targeting systemically-delivered STING pathway agonist demonstrates robust anti-tumor efficacy in multiple murine cancer models."

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"We look forward to sharing data demonstrating the potent anti-tumor activity of our first-in-class STING pathway agonist therapy, STACT-TREX1, in preclinical models. We are encouraged by the data showing that single-agent treatment with STACT-TREX1 generates tumor antigen-specific CD8+ T cell responses, suggesting induction of adaptive anti-tumor immunity after intravenous administration in mice. We believe this mechanism for activating the STING pathway in the tumor microenvironment represents a novel and superior approach for the treatment of cancer," said Dr. Christopher Thanos, President and CEO of Actym Therapeutics.

Details of the presentation are as follows:

Abstract

P235:

STACT-TREX1: A novel tumor-targeting systemically-delivered STING pathway agonist demonstrates robust anti-tumor efficacy in multiple murine cancer models

Date:

November 9, 2018

Location:

Hall E, Walter E. Washington Convention Center

For those unable to attend the conference who would like a copy of the presentation, please send an email request to [email protected]. Please include your name, company, title and phone number. The presentation will be emailed to those making proper requests after November 10, 2018.

On November 8, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the National Institute for Health and Care Excellence (NICE) has published a Final Appraisal Determination (FAD) recommending Vyxeos 44 mg/100 mg powder for concentrate for solution for infusion for routine use on the National Health Service (NHS) in England and Wales for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC)1 – two types of secondary AML (Press release, Jazz Pharmaceuticals, NOV 8, 2018, View Source;p=RssLanding&cat=news&id=2376093 [SID1234530972]).

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"This is the first new chemotherapy in forty years for adults with specific types of newly diagnosed secondary AML, a particularly aggressive cancer that typically affects older people and has a high mortality rate," said Dr. Nigel Russell, Professor of Haematology, Faculty of Medicine & Health Sciences at the University of Nottingham. "I am pleased that NICE has recognised the value of this medicine for adults with secondary AML. In time, it is expected to become the standard of care for this specific group of older AML patients."

Patients diagnosed with t-AML or AML-MRC have a very poor prognosis and have the poorest survival of all AML diagnostic subgroups.2,3 Thus they are classified as having high risk disease because of these poor outcomes. In the UK, the expected number of these high-risk AML cases is 680 per year.4 Its incidence increases with age and accounts for approximately 25% of all AML cases in the UK.4

"A diagnosis of acute myeloid leukaemia can have a huge emotional impact on the lives of patients, as well as their family and friends," said Zack Pemberton-Whiteley, Patient Advocacy Director at Leukaemia Care. "We welcome the decision to recommend this treatment for adults with high-risk AML. Leukaemia Care has been working with NICE to enable patients to access this important new medicine, where options have been previously limited."

"Jazz is delighted that Vyxeos will now be made routinely available on the NHS in England and Wales as people with therapy-related AML or AML with myelodysplasia-related changes have had limited treatment options until now," said Iain McGill, senior vice president, Europe and Rest of World at Jazz Pharmaceuticals. "We believe that it is a meaningful medicine for patients with this rapidly progressing and life-threatening blood cancer."

Vyxeos is an advanced liposomal formulation that delivers a synergistic molar ratio of daunorubicin and cytarabine. It is the first chemotherapy to demonstrate a significant overall survival advantage versus the current treatment standard, 7+3 chemotherapy, in a Phase 3 study of older adult patients with newly diagnosed t-AML or AML-MRC,5 and the first chemotherapy treatment option specifically for people who have these types of high-risk AML.

Notes to Editors

About Vyxeos
It is an advanced liposomal formulation that has been shown in vitro to deliver a synergistic combination of daunorubicin and cytarabine to leukaemia cells for a prolonged period of time. Based on data in animals, the liposomes accumulate and persist in high concentration in the bone marrow, where they are preferentially taken up intact by leukaemia cells.6 It is the first product developed with the company’s proprietary CombiPlex platform, which enables the consideration and design of various combinations of therapies.

It received Orphan Drug Designation (ODD) by the European Commission in January 2012 with retention of the ODD reaffirmed in July 2018 following assessment by the Committee for Orphan Medicinal Products (COMP), and by the U.S. Food and Drug Administration (FDA) in September 2008 for the treatment of AML. It received Promising Innovative Medicine (PIM) designation from the Medicines and Healthcare Products Regulatory Agency in the United Kingdom. In August 2018, the European Commission approved it for the treatment of adults with newly diagnosed t-AML or AML-MRC in all European Union Member States, as well as Iceland, Norway and Liechtenstein.

View the Summary of Product Characteristics here.

About CombiPlex
The CombiPlex proprietary technology enables the design and rapid evaluation of various combinations of therapies to deliver enhanced anti-cancer activity. The CombiPlex technology seeks to identify the most synergistic ratio of drugs in vitro and fix this ratio in a nano-scale delivery complex that maintains the synergistic combination after administration. CombiPlex utilizes two proprietary nano-scale delivery platforms: liposomes to control the release and distribution of water-soluble drugs and drugs that are both water- and fat-soluble (amphipathic), and nanoparticles to control the release and distribution of non-water-soluble (hydrophobic) drugs.

About AML
Acute myeloid leukaemia (AML) is a blood cancer that begins in the bone marrow, which produces most of the body’s new blood cells. AML cells crowd out healthy cells and move into the bloodstream to spread cancer to other parts of the body.7 The median age at diagnosis is 72 years old, with rising age associated with a progressively worsening prognosis.4 There is also a reduced tolerance for intensive chemotherapy as patients age.8 Patients with t-AML or AML-MRC have the lowest survival rates compared with people with other forms of leukaemia.2,3 A haematopoietic stem cell transplant (HSCT) may be a curative treatment option for patients.9

On November 8, 2018 Dova Pharmaceuticals, Inc. (NASDAQ: DOVA), a pharmaceutical company focused on acquiring, developing, and commercializing drug candidates for diseases where there is a high unmet need, reported its operating and financial results for the third quarter ended September 30, 2018 (Press release, Dova Pharmaceuticals, NOV 8, 2018, View Source [SID1234531030]).

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"We continued to make steady progress in the third quarter with both our launch efforts for DOPTELET for patients with thrombocytopenia associated with chronic liver disease as well as the potential expansion of DOPTELET for additional indications," said Alex C. Sapir, President and Chief Executive Officer of Dova. "We were also delighted to have finalized an agreement with Salix Pharmaceuticals, one of the world’s premier gastrointestinal pharmaceutical companies, in order to expand our reach to target gastroenterologists. We continue to hear very positive feedback from the market on how DOPTELET is performing in patients and because of that experience, we are consistently adding more new prescribers. In addition, we are also excited that FDA accepted the avatrombopag supplemental New Drug Application (sNDA) for review for the treatment of chronic ITP, which provides additional long-term growth potential for DOPTELET."

DOPTELET Launch Highlights
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From launch through September 30, 2018, a total of 335 health care professionals have prescribed DOPTELET to their patients with an increasing number using DOPTELET for multiple patients within their practice.

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During the third quarter, for prescriptions that completed the adjudication process with payers, Dova has seen 81% of those prescriptions approved by the payer with an average approval time of 7.9 days.

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Dova has made significant progress in its outreach efforts having reached 67% of target prescribers an average of 2.8 times from launch through September 30, 2018.

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Inventory held by specialty pharmacies increased by approximately 65% from July 1, 2018 to September 30, 2018 as certain specialty pharmacies increased their inventory levels and stocking locations based on increased patient shipments.

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In September, Dova entered into an exclusive co-promotion agreement with Salix Pharmaceuticals to co-promote DOPTELET in the United States. Salix is deploying approximately 100 sales specialists who will promote DOPTELET to gastroenterology healthcare professionals. Dova will continue its commercial efforts targeting primarily hepatologists and interventional radiologists and certain other specialties.

Other Important Highlights for the Quarter
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The U.S. FDA accepted for review Dova’s sNDA for DOPTELET for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to a previous treatment. The sNDA for ITP is supported by safety and efficacy data from two Phase 2 clinical trials and one randomized, placebo-controlled Phase 3 clinical trial that met its primary and secondary efficacy endpoints with high statistical significance. The Prescription Drug User Fee Act (PDUFA) goal date for an FDA decision is June 30, 2019.

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Following the submission in April of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for DOPTELET for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure, the Company expects a target decision date for approval in Europe in the second or third quarter of 2019.

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Enrollment remains ongoing for the Company’s Phase 3 clinical trial for the treatment of patients with chemotherapy-induced thrombocytopenia (CIT), the pre-surgery study treating thrombocytopenia, and the CLD registry study.

Third Quarter and Financial Results

Dova reported a net loss of $19.5 million for the third quarter of 2018, compared to a net loss of $9.7 million for the same period in 2017.

For the third quarter of 2018, Dova reported net product sales from DOPTELET of $2.9 million. The Company recognizes revenue when products are delivered to its specialty pharmacy partners.

Cost of product sales for the third quarter were $0.4 million, which consisted of the cost of inventory, royalty payments due to Astellas and certain distribution and overhead costs.

Research and development expenses were $4.8 million in the third quarter of 2018, compared to $5.4 million for the same period in 2017. The decrease was primarily driven by the $1.0 million

milestone payment that Dova became obligated to pay Astellas upon submission of the NDA and the completion of the clinical trials in 2017 of DOPTELET, partially offset by the initiation of clinical trials to evaluate DOPTELET for the treatment of a broader population of surgical patients as well as for CIT.

Selling, general and administrative expenses were $17.0 million in the third quarter of 2018, compared to $4.2 million for the same period in 2017. The increase was primarily driven by increased headcount and sales and marketing activities to support the commercial launch of DOPTELET, increased corporate infrastructure, and additional costs associated with operating as a public entity.

As of September 30, 2018, Dova had $122.0 million in cash and equivalents compared to $94.8 million as of December 31, 2017.

Company to Host Conference Call

Dova will host a conference call today, November 8, 2018 at 4:30 p.m. ET to discuss third quarter 2018 financial results and recent operational highlights. A question-and-answer session will follow Dova’s remarks.

To participate on the live call, please dial 866-550-8145 (domestic) or +1-430-775-1344 (international) and provide the conference ID 2574148 five to 10 minutes before the start of the call.

A live audio webcast of the call will also be available via the "Investor Relations" page of the Dova website, www.dova.com. Please log on through Dova’s website approximately 10 minutes before the scheduled start time. A replay of the webcast will be archived on Dova’s website for 90 days following the call.

Indication and Important Safety Information

INDICATION

DOPTELET (avatrombopag) is indicated for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic and thromboembolic complications in patients with chronic liver disease. Portal vein thrombosis has been reported in patients with chronic liver disease treated with TPO receptor agonists. In the ADAPT-1 and ADAPT-2 clinical trials, there was 1 treatment-emergent

event of portal vein thrombosis in a patient (n=1/430) with chronic liver disease and thrombocytopenia treated with DOPTELET.

Consider the potential increased thrombotic risk when administering DOPTELET to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions (Factor V Leiden, Prothrombin 20210A, Antithrombin deficiency or Protein C or S deficiency).

DOPTELET should not be administered to patients with chronic liver disease in an attempt to normalize platelet counts.

CONTRAINDICATIONS:

None

ADVERSE REACTIONS:

Most common adverse reactions (≥ 3%) were: pyrexia, abdominal pain, nausea, headache, fatigue, and edema peripheral.

Please see full Prescribing Information for DOPTELET (avatrombopag) www.doptelet.com