On August 7, 2018 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the second quarter 2018 and provided an overview of recent accomplishments and clinical development plans for selinexor, its lead, novel, oral SINE compound and its other pipeline programs (Press release, Karyopharm, AUG 7, 2018, View Source [SID1234528491]).

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"We have made tremendous progress toward advancing our lead drug candidate, selinexor, as we strive to improve the lives of patients with myeloma and other forms of cancer. Most notably, we have now completed the submission of our first New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for selinexor as a potential new treatment for patients with penta-refractory myeloma," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "The positive results from the Phase 2b STORM study announced during the quarter demonstrated that treatment with selinexor may result in an important clinical benefit in this patient population and we look forward to working with the FDA during their review of the application. We are making excellent progress in advancing commercial preparation for the potential launch of selinexor in the U.S., as well as preparing a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), which we expect to submit in early 2019, with a request for conditional approval, also in penta-refractory myeloma. Finally, while completing our first-ever NDA submission is clearly a landmark event for Karyopharm, we remain committed to fulfilling the full potential of selinexor as we advance our clinical strategy in earlier lines of treatment in myeloma and in additional tumor types."

Second Quarter 2018 and Recent Events

Selinexor in Multiple Myeloma

Submitted NDA in Penta-Refractory Myeloma. Karyopharm completed its submission of an NDA to the FDA seeking accelerated approval for selinexor, its lead, novel, oral SINE compound, as a new treatment for patients with penta-refractory multiple myeloma. Patients with penta-refractory myeloma have previously received the two proteasome inhibitors (PIs), Velcade (bortezomib) and Kyprolis (carfilzomib), the two immunomodulatory drugs (IMiDs), Revlimid (lenalidomide) and Pomalyst (pomalidomide), and the anti-CD38 monoclonal antibody Darzalex (daratumumab) as well as alkylating agents, and their disease is refractory to at least one PI, at least one IMiD, Darzalex and their most recent therapy. The Company also plans to submit an MAA to the EMA in early 2019 with a request for conditional approval in the same indication.

Reported Positive Top-line Data from the Phase 2b STORM Study in Patients with Penta-Refractory Myeloma. Karyopharm reported positive top-line results from Part 2 of the Phase 2b STORM study evaluating selinexor plus low dose dexamethasone (Sd) in patients with penta-refractory multiple myeloma. The STORM study’s primary endpoint of overall response rate (ORR) was 25.4%, which included two stringent complete responses (sCRs) and 29 partial or very good partial responses. The two sCRs were negative for minimal residual disease, one at 1:10-6 and one at 1:10-4, which is particularly significant in this penta-refractory population. The median duration of response, a key secondary objective, was 4.4 months. Across the relevant patient population, side effects of oral selinexor were generally predictable and often managed with dose adjustments or supportive care. Safety results were consistent with those previously reported from Part 1 of this study and from other selinexor studies and no new safety signals were identified. Karyopharm plans to submit detailed STORM study results for presentation at an upcoming medical oncology meeting.

Presented Updated Data from the Phase 1b/2 STOMP Study at the European Hematology Association (EHA) (Free EHA Whitepaper) 2018 Annual Meeting. At EHA (Free EHA Whitepaper) 2018, Karyopharm presented three posters with updated data from STOMP evaluating selinexor and dexamethasone in combination with standard approved therapies, Velcade (SVd), Pomalyst (SPd) or Darzalex (SDd), in patients with previously treated multiple myeloma. The SVd arm demonstrated progression-free survival (PFS) of 17.8 months, an ORR of 83% in the same patient population eligible for the pivotal Phase 3 BOSTON study. The SDd arm demonstrated an ORR of 82% in patients with heavily pretreated Darzalex-naïve disease. The all oral SPd arm demonstrated an ORR of 55% in patients with Pomalyst-naïve and Revlimid-relapsed or -refractory disease, with a PFS of 11.6 months. Adverse events across all three arms were consistent with those reported previously with selinexor and the combination therapies, with no new safety signals specific to the combinations identified. These results suggest that selinexor can be combined with other anti-myeloma agents and induce durable responses in patients with previously treated MM.
Initiated New STOMP Arm Evaluating All Oral Regimen of Selinexor, Revlimid and Dexamethasone (SRd) in Patients with Newly Diagnosed Myeloma. Based on the positive STOMP results reported to date evaluating SRd in patients with relapsed myeloma, the Company initiated a new, all oral STOMP arm to investigate the combination of SRd in the front-line setting. Given the observed synergistic activity of selinexor with standard approved multiple myeloma therapies, Karyopharm believes oral selinexor has the potential to be a future backbone therapy in multiple myeloma.
Pivotal Phase 3 BOSTON Study in Progress. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is underway and enrolling patients in 14 countries. BOSTON is evaluating 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (once weekly) and low-dose dexamethasone (SVd), compared to standard twice weekly Velcade and low-dose dexamethasone (Vd) in patients with multiple myeloma who have had one to three prior lines of therapy. The primary endpoints of the study are PFS and ORR. Data from the BOSTON study, if positive, would be used to support regulatory submissions to the FDA and EMA requesting full approvals for use of selinexor in second line multiple myeloma, following the Company’s requests for accelerated and conditional approvals, respectively, using data from the Phase 2b STORM study. The Company expects to enroll approximately 360 patients at over 100 clinical sites internationally and expects to complete enrollment by the end of 2018, with top-line data anticipated in 2019.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)

Ongoing Phase 2b SADAL Study in DLBCL. Karyopharm is also investigating oral selinexor as a single-agent for the treatment of patients with relapsed or refractory DLBCL who are not eligible for stem cell transplantation. The SADAL study is expected to enroll up to a total of 130 patients in the single-arm cohort evaluating single-agent selinexor dosed 60mg twice weekly in patients who received two to five lines of prior therapy. Karyopharm plans to report top-line results by the end of 2018. Assuming the results from the SADAL study are positive, Karyopharm plans to submit an NDA to the FDA with a request for accelerated approval, and an MAA to the EMA with a request for conditional approval, for oral selinexor in this relapsed/refractory DLBCL patient population.
Selinexor in Solid Tumors

Presented Data from the Phase 2 Portion of the Phase 2/3 SEAL Study in Liposarcoma at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2018 Annual Meeting. At ASCO (Free ASCO Whitepaper) 2018, Karyopharm presented positive results from the successful Phase 2 portion of the blinded, randomized Phase 2/3 SEAL study evaluating single-agent selinexor versus placebo in patients with previously treated, advanced unresectable dedifferentiated liposarcoma. For the primary endpoint of PFS, oral selinexor showed superiority over placebo, achieving a median PFS of 5.5 months, compared to 2.7 months for placebo with a hazard ratio (HR) of 0.67, representing a 33% reduction in the risk of progression or death. Across the relevant patient population, side effects of oral selinexor were generally predictable and often managed with dose adjustments or supportive care, with the most frequent events being nausea, fatigue, anorexia and weight loss, with low levels of Grade 3/4 cytopenias, and no new or unexpected safety signals identified. The Phase 3 portion of the SEAL study is underway and, assuming a positive outcome on the primary end point of PFS, the Company intends to use the data from the SEAL study to support an NDA and an MAA submission requesting full approval for oral selinexor for patients with advanced unresectable dedifferentiated liposarcoma. Top-line data from the Phase 3 portion of the SEAL study are anticipated by the end of 2019.
Ongoing Investigator Sponsored Phase 2/3 Trial as Maintenance Therapy in Endometrial Cancer Underway. A randomized Phase 2/3 study of selinexor versus placebo as maintenance therapy in patients with one or two prior platinum-based treatments for advanced endometrial cancer lead by Dr. Ignace Vergote, Head of the Department of Obstetrics and Gynaecology and Gynaecologic Oncology at the Catholic University of Leuven, Belgium, is currently ongoing. In the U.S., endometrial cancer is the most common gynecological cancer with approximately 58,000 cases expected to be diagnosed and an estimated 10,000 women expected to die from this cancer in 20181, revealing a meaningful patient population in need of novel therapies.
Corporate Updates

Anand Varadan Appointed Chief Commercial Officer. Karyopharm announced the appointment of Anand Varadan as Executive Vice President, Chief Commercial Officer. Mr. Varadan brings over 25 years of commercial operations and strategy experience with a proven track record of building and leading commercial and cross-functional teams and successfully launching and marketing new therapeutics at publicly-traded, healthcare-focused companies. Mr. Varadan leads the Company’s commercial strategy and operations, including for the launch of selinexor.

Ian Karp Appointed Vice President, Investor and Public Relations. The Company also appointed Ian Karp as Vice President, Investor and Public Relations. Mr. Karp brings over 20 years of investor relations, corporate development, and commercial experience and has successfully led global teams in achieving key corporate communications objectives, including in the areas of oncology and orphan diseases. Mr. Karp leads all of the Company’s corporate communications activities, including corporate visibility, financial communications, and media and investor relations.
Exclusive License Agreement Executed with Antengene to Develop and Commercialize Selinexor, Eltanexor, Verdinexor and KPT-9274 in China and Other Regions in Asia. The agreement includes the development of selinexor and eltanexor for the diagnosis, treatment and/or prevention of all human oncology indications in China and Macau. The agreement also includes the development and commercialization of KPT-9274 in all human oncology indications and verdinexor in human non-oncology indications in mainland China, Macau, Taiwan, Hong Kong, South Korea, and the ASEAN countries. The transaction carries a total deal value of up to $162 million, plus royalties.
Second Quarter Ended June 30, 2018 Financial Results

Cash, cash equivalents and investments as of June 30, 2018, including restricted cash, totaled $250.5 million, compared to $176.4 million as of December 31, 2017.

On May 7, 2018, Karyopharm completed an underwritten public offering of 10,525,424 shares of its common stock at a price to the public of $14.75 per share, resulting in net proceeds of approximately $145.7 million after deducting the underwriting discounts and commissions and other offering expenses.

For the quarter ended June 30, 2018, Karyopharm recognized $19.9 million in revenue, compared to a small amount of grant revenue for the three months ended June 30, 2017. The increase in revenue was primarily the result of recognizing $19.7 million of revenue related to fulfilling an obligation under our license agreement with Ono Pharmaceutical Co., LTD. The cash related to this revenue was received as part of the upfront payment received from Ono in October 2017.

For the quarter ended June 30, 2018, research and development expense was $44.7 million compared to $23.1 million for the quarter ended June 30, 2017. For the quarter ended June 30, 2018, general and administrative expense was $9.5 million compared to $6.6 million for the quarter ended June 30, 2017.

Karyopharm reported a net loss of $33.7 million, or $0.60 per share, for the quarter ended June 30, 2018, compared to a net loss of $29.4 million, or $0.64 per share, for the quarter ended June 30, 2017. Net loss includes stock-based compensation expense of $4.4 million and $5.1 million for the quarters ended June 30, 2018 and June 30, 2017, respectively.

Financial Outlook

Karyopharm expects its operating cash burn, including research and development and general and administrative expenses, for the year ending December 31, 2018 to be in the range of $175 to 185 million. Based on current operating plans, Karyopharm expects that its existing cash, cash equivalents and investments will be sufficient to fund its operations for at least the next twelve months. These plans include the continued clinical development of selinexor in the Company’s lead indications with a focus on preparing the commercial infrastructure and hiring a sales force for the potential launch of selinexor in the U.S. Additional key activities expected in 2018 include preparing for a potential MAA submission to the EMA requesting conditional approval for selinexor in multiple myeloma, topline data from the SADAL study and completion of enrollment in the Phase 3 BOSTON study.

Further Information About Potential Accelerated Approval for Selinexor in Multiple Myeloma

The FDA instituted its Accelerated Approval Program to allow for expedited approval of drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint or an intermediate clinical endpoint thought to predict clinical benefit, like overall response rate (ORR). Accelerated approval is available only for drugs that provide a meaningful therapeutic benefit over existing treatments at the time of consideration of the application for accelerated approval, which the FDA has reiterated in its feedback to the Company. Particularly in disease areas with multiple available and potential new therapies, such as multiple myeloma, accelerated approval carries a high regulatory threshold. Consistent with its general guidance, the FDA has noted to the Company its preference for randomized studies geared toward full approval, which the Company has undertaken with the ongoing pivotal, Phase 3 BOSTON study, and has reminded the Company that accelerated approval requires patients to have exhausted all available approved therapies. FDA’s Fast Track designation is available to therapeutics treating an unmet medical need in a serious condition; the Company has received Fast Track designation from the FDA specifically for the population treated in the STORM trial. In light of this recognition that the STORM patient population represents an unmet medical need and the positive top-line data reported in April 2018, the Company believes that the STORM study should support its request to the FDA for accelerated approval.

Conference Call Information

Karyopharm will host a conference call today, Tuesday, August 7, 2018, at 8:30 a.m. Eastern Time, to discuss the second quarter 2018 financial results, recent accomplishments, clinical developments and business plans. To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference ID 3084449. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.

On August 7, 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 second quarter ended June 30, 2018 and provided pipeline updates (Press release, AnaptysBio, AUG 7, 2018, View Source [SID1234528509]).

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"We made significant advances during the second quarter of 2018 in the clinical development of our first-in-class wholly-owned antibody therapeutics for patients with severe inflammatory conditions," said Hamza Suria, president and chief executive officer of AnaptysBio. "We are excited to advance the clinical development of etokimab in large atopic disease markets, including our ongoing Phase 2b ATLAS trial in moderate-to-severe atopic dermatitis, our ongoing Phase 2a trial in severe eosinophilic asthma and our upcoming Phase 2 ECLIPSE trial in adult chronic rhinosinusitis with nasal polyps. Development of ANB019 in orphan diseases has been initiated with our Phase 2 GALLOP trial in generalized pustular psoriasis and upcoming Phase 2 POPLAR trial in palmoplantar pustulosis. We look forward to the five clinical efficacy readouts anticipated from our etokimab and ANB019 programs by the end of 2019, starting with our upcoming etokimab Phase 2a top-line data in eosinophilic asthma during the third quarter of 2018, as key milestones in our mission to bring novel treatments to patients with severe inflammatory diseases."

Etokimab (ANB020 Anti-IL-33 Program)

• In July 2018, etokimab (pronounced ee-toh-key-mab) was adopted as the nonproprietary name by the United States Adopted Names (USAN) Council, in consultation with the World Health Organization (WHO) International Nonproprietary Names Expert Committee, for AnaptysBio’s anti-IL-33 antibody drug candidate previously referred to as ANB020.

• In May 2018, updated data from the company’s Phase 2a trial of etokimab, AnaptysBio’s wholly-owned anti-IL-33 antibody program, in adult patients with moderate-to-severe atopic dermatitis were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2018 in Munich by the principal investigator of the trial, Dr. Graham Ogg, professor of dermatology at Oxford University in Oxford, England. Key observations presented by Dr. Ogg during the aforementioned presentation included:

Biomarker data demonstrated that reduction of circulating blood eosinophil was consistent with clinical efficacy measures in this Phase 2a trial, with a maximum reduction of 40 percent at day 29 after a single dose of etokimab relative to baseline, which is aligned with genotypic studies that associate lower eosinophil counts with human IL-33 loss-of-function mutations. In addition, clinical efficacy data in this Phase 2a trial were consistent with an ex vivo pharmacodynamic assay measuring IL-33 mediated interferon-gamma release, where 98 percent inhibition was observed within 72 hours following etokimab administration and 86 percent inhibition was sustained at day 57 post-ANB020 administration, which is consistent with the pharmacodynamic activity observed using the same assay in a prior Phase 1 trial of etokimab in healthy volunteers.
A single dose of etokimab resulted in achievement of EASI-50 by all 12 patients enrolled in this trial on or before day 57 post-etokimab administration. Rapid clinical response was observed by day 15 post-etokimab administration and day 29 results exceeded the primary efficacy objective of the trial with 10 of 12 patients (83%) achieving EASI-50, of which four patients (33%) also achieved EASI-75. EASI-50 results were sustained through day 140 following single dose administration of etokimab, five of 12 patients (42%) achieved EASI-50, of which three patients (25%) also achieved EASI-75. Other atopic dermatitis efficacy endpoints, including the five-point Investigator’s Global Assessment (IGA) scale, the SCORing Atopic Dermatitis (SCORAD) scale, Dermatology Life Quality Index (DLQI) and the five-dimensional pruritus scale, demonstrated rapid and sustained single dose etokimab efficacy results in a similar manner to the aforementioned EASI results.
Etokimab was generally well-tolerated by all patients and no drug-related safety signals were observed. The most frequent adverse events reported were dizziness in 17 percent of patients post-placebo and headache in 25 percent of patients post-etokimab administration. A single serious adverse event of depression was reported on day 140 post-etokimab administration, which was consistent with the patient’s pre-trial history of depression and was deemed not drug-related.
• 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 follow-up period, with data expected in the second half of 2019. Sixty patients are being randomized into each of the five arms in the ATLAS trial where dosing will occur as follows: (i) initial 600mg loading dose followed by 300mg monthly doses of etokimab, (ii) initial 300mg loading dose followed by 150mg monthly doses of etokimab, (iii) initial 300mg loading dose followed by 150mg doses of etokimab every eight weeks, (iv) monthly 20mg doses of etokimab and (v) monthly doses of placebo.

• AnaptysBio expects to report top-line efficacy and safety data, including improvement in Forced Expiratory Volume in One Second (FEV1), from its ongoing double-blinded, placebo-controlled severe eosinophilic asthma trial Phase 2a trial, where approximately 24 adult severe eosinophilic asthma patients are treated with a 300mg intravenous single dose of etokimab versus placebo, each in combination with inhaled corticosteroids and long-acting beta agonists as background therapy, in the third quarter of 2018.

• The Company has expanded development of etokimab into adult chronic rhinosinusitis with nasal polyps (CRSwNP), which is a debilitating atopic disorder associated with elevated IL-33 pathway signaling, affecting approximately 1.3 million adults in the U.S., and AnaptysBio estimates approximately 400,000 of these patients are inadequately controlled with standard-of-care. The Company plans to initiate a randomized, placebo-controlled Phase 2 trial, also referred to as the ECLIPSE trial, in approximately 100 adult CRSwNP patients treated with two different multi-dosing frequencies of subcutaneously-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 follow-up period. The Company plans to initiate the ECLIPSE trial by the end of 2018, and anticipates top-line data will be available in the second half of 2019.

• As a result of market assessment regarding the adoption of the peanut oral food challenge in future commercial usage of etokimab in peanut allergy patients, AnaptysBio has decided to deprioritize further company-sponsored clinical development of etokimab in moderate-to-severe baseline adult peanut allergy patients. At this time, AnaptysBio does not intend to utilize its clinical development resources to pursue a Phase 2b clinical trial of etokimab in peanut allergy, however the Company may pursue potential investigator-sponsored trials related to this indication.

ANB019 (Anti-IL-36 Receptor Program)

• Data from the company’s Phase 1 healthy volunteer trial of ANB019, its wholly-owned anti-interleukin-36 receptor, or IL-36R therapeutic antibody, were presented during May in a poster session at the EAACI Congress 2018 in Munich. In the double-blind, placebo-controlled healthy volunteer Phase 1 trial, 36 subjects were administered a single subcutaneous or intravenous dose of ANB019 ranging between 10 mg and 750 mg, 18 subjects were administered multiple ascending doses of ANB019 intravenously ranging between 40 mg and 300 mg weekly for four consecutive weeks and 18 subjects were dosed with placebo. ANB019 was well-tolerated by all subjects and no dose-limiting toxicities were observed. The most frequent treatment-emergent adverse events observed in the single ascending dose cohorts were upper respiratory tract infections in 10 of 36 (28%) subjects dosed with ANB019 versus six of 12 (50%) subjects dosed with placebo, and headache in 10 of 36 (28%) subjects dosed with ANB019 versus three of 12 (25%) subjects dosed with placebo. In the multiple ascending dose cohorts, the most frequent treatment-emerging adverse events observed were headache in seven of 18 (39%) subjects dosed with ANB019 versus one of six (17%) subjects dosed with placebo. No serious adverse events were reported among any subjects in the trial. The in vivo half-life of ANB019 was approximately 28 days for both subcutaneous and intravenous routes of administration, with bioavailability of approximately 90 percent. A single dose of ANB019 at certain dose levels was able to completely suppress IL-36 cytokine function for 85 days, as measured by IL-36 cytokine-mediated release of IL-8 using an ex vivo pharmacodynamic assay. The favorable safety, pharmacokinetics and pharmacodynamic properties of ANB019 demonstrated by this Phase 1 trial support advancement of ANB019 into Phase 2 studies for GPP and PPP.

• AnaptysBio has initiated a 10-patient open-label Phase 2 trial of ANB019 in GPP, also known as the GALLOP trial and top-line data are anticipated by early 2019. Patients are dosed with a 750mg intravenous loading dose of ANB019 upon enrollment, followed by 100mg subcutaneously-administered monthly doses of ANB019 for a treatment period of up to 16 weeks post enrollment and followed an eight-week follow-up period. The company plans to initiate a placebo-controlled 50-patient multi-dose Phase 2 trial in PPP, also known as the POPLAR trial, where top line data is anticipated in the second half of 2019.

Second Quarter Financial Results

• Cash, cash equivalents and investments totaled $300.6 million as of June 30, 2018 compared to $324.3 million as of December 31, 2017, for a decrease of $23.7 million. The decrease primarily relates to operating cash outflow for clinical and manufacturing related expenses, as well as personnel costs.

• Research and development expenses were $10.6 million for the three months ended June 30, 2018, as compared to $7.2 million for the three months ended June 30, 2017. The increase was primarily due to continued advancement of the Company’s ANB020 and ANB019 clinical programs and additional personnel-related expenses, including share based compensation, as well as the recognition of lower research and development tax incentives during the three months ended June 30, 2018.

• General and administrative expenses were $3.8 million for the three months ended June 30, 2018, as compared to $2.4 million for the three months ended June 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 through the end of 2019.

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 we believe is broadly applicable to the treatment of atopic inflammatory disorders, such as moderate-to-severe adult atopic dermatitis, severe adult eosinophilic asthma, adult CRSwNP and potentially other allergic conditions. Following completion of a healthy volunteer Phase 1 trial of etokimab, AnaptysBio has continued clinical development of etokimab into a Phase 2a trial for moderate-to-severe adult atopic dermatitis and a 24-patient placebo-controlled Phase 2a trial in severe adult eosinophilic asthma patients where top-line data are anticipated in the third quarter 2018. AnaptysBio is enrolling its ATLAS trial, a 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. AnaptysBio also plans to initiate its ECLIPSE trial, a randomized, placebo-controlled Phase 2 trial of etokimab in approximately 100 adult patients with CRSwNP by the end of 2018 with data anticipated in the second half of 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 (GPP) and palmoplantar pustulosis (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 early 2019, and plans to initiate its POPLAR trial, a Phase 2 study in PPP in 2018 where data is anticipated in the second half of 2019.

On August 7, 2018 Johnson & Johnson (NYSE: JNJ) reported that it will participate in the 2018 Wells Fargo 13th Annual Healthcare Conference on Thursday, Sept. 6th, at The Westin Boston Waterfront in Boston, MA (Press release, Johnson & Johnson, AUG 7, 2018, View Source [SID1234528660]). Alex Gorsky, Chairman and Chief Executive Officer will represent the Company in a session scheduled at 9:45 a.m. (Eastern Time).

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This webcast will be available to investors and other interested parties by accessing the Johnson & Johnson website at www.investor.jnj.com.

A webcast replay will be available approximately two hours after the live webcast.

On August 7, 2018 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its second quarter 2018 financial results and provided an update on the company, including its progress with IPI-549, a first-in-class oral immuno-oncology product candidate that selectively inhibits phosphoinositide-3-kinase-gamma (PI3K-gamma) and targets immune-suppressive tumor macrophages/myeloid-derived suppressor cells (MDSCs) (Press release, Infinity Pharmaceuticals, AUG 7, 2018, View Source [SID1234528757]).

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"We are particularly pleased to welcome Sam Agresta to the Infinity team as Chief Medical Officer. Sam brings extensive experience in novel oncology drug development, having led the recent approvals of Agios’s two targeted therapies based on compelling Phase 1 data," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "We continue to be encouraged by our progress in all aspects of our business. Building on the datasets we presented at the recent American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, including showing that IPI-549 was well-tolerated, clinically active and on-mechanism as a monotherapy and in combination with nivolumab, we look forward to presenting more mature data from the combination expansion cohorts in the second half of this year. We are also enthusiastic about partnering with Arcus and testing IPI-549 in combination with its very promising novel agents, in a new collaboration we established in June."

Infinity is evaluating IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in collaboration with Bristol-Myers Squibb, in the MARIO-1 Phase 1/1b study in approximately 200 patients with advanced solid tumors. In addition, Arcus Biosciences will initiate two triple combinations investigating IPI-549 with their dual adenosine receptor antagonist, AB928, anti-PD-1 antibody, AB122, and chemotherapy in triple negative breast cancer and ovarian cancer. One triple combination therapy will evaluate IPI-549 in combination with AB928 and AB122 and the second will evaluate IPI-549 in combination with AB928 and chemotherapy, with topline data expected in 2019.

Recent developments include the following:

IPI-549

Continued Progress with the MARIO-1 Phase 1/1b Study of IPI-549: The monotherapy and combination dose-escalation portions of this study have been completed. Enrollment is complete in the mesothelioma combination expansion cohort and is ongoing for the other five disease-specific combination expansion cohorts currently underway, as well as for the seventh combination expansion cohort of patients pre-selected for having high baseline blood levels of myeloid derived suppressor cells (MDSCs), which began enrolling patients in May 2018.
Second Quarter 2018 Financial Results

At June 30, 2018, Infinity had total cash, cash equivalents and available-for-sale securities of $49.2 million, compared to $47.8 million at March 31, 2018.
R&D expense for the second quarter of 2018 was $3.7 million, compared to $3.9 million for the same period in 2017. The decrease in R&D expense was primarily due to a reduction in bonus and stock compensation offset by an increase in clinical development expense for IPI-549.
General and administrative expense was $3.4 million for the second quarter of 2018, compared to $6.2 million for the same period in 2017. The decrease in G&A expense was primarily due to a reduction in bonus and stock compensation.
Net loss for the second quarter of 2018 was $7.0 million, or a basic and diluted loss per common share of $0.12, compared to a net loss of $17.0 million, or a basic and diluted loss per common share of $0.34 for the same period in 2017.
Financial Outlook
Infinity’s 2018 financial guidance is:

Net Loss: Infinity expects net loss for 2018 to range from $35 million to $45 million.
Cash and Investments: Infinity expects to end 2018 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $15 million to $25 million.
Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities will be adequate to satisfy the company’s capital needs through the third quarter of 2019. Infinity’s financial guidance excludes additional funding or business development activities and does not include the potential $22 million payment from Verastem upon the first regulatory approval of duvelisib, or a potential $2 million payment from PellePharm, a private company, upon initiation of a Phase 3 study for the hedgehog inhibitor program, which Infinity licensed to PellePharm in 2013. Verastem announced that its New Drug Application for duvelisib was accepted by the U.S. Food and Drug Administration (FDA) and that it was given priority review with an FDA action date of October 5, 2018. With the potential Verastem payment, Infinity expects that its cash runway would extend into 2020.
Conference Call Information
Infinity will host a conference call today, August 7, 2018, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial 1-877-316-5293 (domestic) or 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 8037589. An archived version of the webcast will be available on Infinity’s website for 30 days.

About IPI-549 and the Ongoing Phase 1/1b Study
IPI-549 is an investigational first-in-class, oral, immuno-oncology product candidate targeting tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition, thereby reducing pro-tumor macrophage function and increasing anti-tumor macrophage function. In preclinical studies, IPI-549 demonstrated the ability to reprogram macrophages from a pro-tumor (M2), immune suppressive function, to an anti-tumor (M1) immune activating function and enhance the activity of, and overcome resistance to, checkpoint inhibitors.1,2 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially additive or synergistic approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

The ongoing Phase 1/1b study being conducted by Infinity is designed to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo in approximately 200 patients with advanced solid tumors.3 The study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The monotherapy dose-escalation and expansion components are complete. The combination dose-escalation component is also complete, and combination expansion cohorts are enrolling.

The combination expansion component of the study includes multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma and head and neck cancer whose tumors show initial resistance or initially respond to but subsequently develop resistance to immune checkpoint blockade therapy. The combination expansion component also includes a cohort of patients with triple negative breast cancer (TNBC) who have not been previously treated with immune checkpoint blockade therapy, a cohort of patients with mesothelioma, a cohort of patients with adrenocortical carcinoma and a cohort of patients with high baseline blood levels of MDSCs.

IPI-549 is an investigational compound and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On August 7, 2018 Novelion Therapeutics Inc. (NASDAQ: NVLN), a biopharmaceutical company dedicated to developing and commercializing therapies for individuals living with rare diseases ("Novelion" or the "Company"), reported financial results for the second quarter and six months ended June 30, 2018 and provided an overview of business activities (Press release, QLT, AUG 7, 2018, View Source [SID1234528932]).

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Interim Chief Executive Officer Jeff Hackman commented, "Under our new leadership team, we are laser focused on maximizing the potential of our two commercial assets, enhancing our operational efficiencies by reducing costs, and fixing our capital structure issues so we can invest in the long-term opportunity of our valuable rare disease therapies.

"On the commercial front, we expect sequential revenue growth in the second half of this year, bolstered by stabilizing JUXTAPID sales in the U.S., and continued penetration of the Japan market, where there are more than 200 registered HoFH patients. We also expect initial contribution from the European launch of MYALEPTA (metreleptin) for both generalized lipodystrophy (GL) and partial lipodystrophy (PL), which represents the largest market for metreleptin in terms of treatable patients, a number of whom have already been identified through our pre-approval compassionate use program and are expected to be converted onto therapy, subject to pricing and reimbursement approvals, where required. Further, we believe that we can leverage the EU approval data package to support our plans to expand the U.S. label to include the PL indication and to file in additional markets, including Brazil. We want to thank Murray Stewart, M.D., our head of R&D, and his team, along with all the employees that supported the filing and approval, for achieving this important approval. We look forward to their work on expanding the metreleptin opportunity."

Business Highlights

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On July 31, Novelion announced that the European Commission (EC) granted marketing authorization for MYALEPTA, as an adjunct to diet, as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients with confirmed congenital GL or acquired GL in adults and children 2 years of age and above; or with confirmed familial PL or acquired PL, in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. MYALEPTA is the first and only licensed treatment in Europe in these indications. Pricing and reimbursement negotiations with healthcare authorities have commenced and will be pursued on a country-by-country basis.

•
In August, the Company submitted a marketing application for JUXTAPID in Brazil. While the Company currently recognizes sales for JUXTAPID in Brazil via the pre-approval named patient sales program, the Company is seeking formal marketing approval which, if successful, will open the door for product promotion and should also result in more predictable sales trends.

•
JUXTAPID: Novelion reported net revenues of JUXTAPID of $16.3 million in the second quarter of 2018, $10.5 million, or 64%, of which were from prescriptions written in the U.S. and $0.8 million of which was royalty revenue from Amryt’s sales of JUXTAPID in the EMEA region.

•
MYALEPT: Novelion reported net revenues of MYALEPT of $15.6 million in the second quarter of 2018, $12.5 million, or 80%, of which were from prescriptions written in the U.S.

•
In June, clinical data from a metreleptin study assessing weight loss in overweight and obese adults with low leptin levels were featured in a poster presentation at the American Diabetes Association’s (ADA) 78th Scientific Sessions. These data support potential metreleptin pipeline opportunities.

•
In July, Novelion’s Board of Directors appointed Mark Corrigan, M.D. as Executive Chair. Dr. Corrigan serves in a supervisory role to the Company’s management team and will continue to perform the traditional duties of Board Chair. In addition, Jeff Hackman was appointed interim chief executive officer.

Dr. Corrigan commented, "The first half of 2018 has been filled with important milestones. Having finalized Aegerion’s legal settlements earlier this year, our organization was pleased with the strong market receptivity of our continued launch of JUXTAPID in Japan, as well as the stabilization of our U.S. JUXTAPID franchise. More importantly, the MYALEPTA European approval creates a larger revenue opportunity for us than in the U.S. In addition to top-line growth, we intend to further our operational improvements with the goal of creating a pathway to sustainable positive cash flow and robust EBITDA growth. Given our progress on these initiatives, we intend to re-engage with the investor and analyst community, and we also plan to resume revenue guidance for 2019."

Second Quarter 2018 Financial Results

GAAP total net revenues for the second quarter of 2018 were $31.9 million compared to $40.9 million for the same period of 2017, primarily as a result of the timing of orders for both products in Brazil. The second quarter of 2017 benefitted from $8.1 million of Brazil revenues derived from the named patient sales program, whereas the second quarter of 2018 does not reflect any named patient sales in Brazil. The Company expects that marketing approval of its products, if achieved, in Brazil will support more predictable sales. Revenue growth of $2.0 million in other foreign markets more than offset the $0.7 million decrease in U.S. revenues. The second quarter of 2017 also benefitted from a one-time recognition of previously deferred revenue totaling $2.3 million, related to a change in method of revenue recognition for MYALEPT.

GAAP net revenues for JUXTAPID in the second quarter of 2018 were $16.3 million compared to $20.7 million for the same period in 2017. JUXTAPID revenues representing named patient sales in Brazil totaled $4.3 million in the second quarter of 2017, while there were no named patient sales

of JUXTAPID in Brazil in the second quarter of 2018. Growth of 8% in other foreign markets nearly offset the 5% decrease in U.S. revenues, where we continue to see stabilization of sales.

GAAP net revenues for MYALEPT in the second quarter of 2018 were $15.6 million compared to $20.2 million for the same period in 2017. MYALEPT revenues representing named patient sales in Brazil totaled $3.8 million in the second quarter of 2017, while there were no named patient sales of MYALEPT in Brazil in the second quarter of 2018. As mentioned previously, MYALEPT revenues of $20.2 million in the second quarter of 2017 included a one-time recognition of previously deferred revenue of $2.3 million. Excluding this effect, U.S. revenues were virtually unchanged compared to the comparable period of the prior year, while MYALEPT revenues in foreign markets other than Brazil more than doubled from $1.5 million to $3.2 million in the same timeframe.

GAAP total operating expenses for the second quarter of 2018 were $34.1 million compared to total operating expenses of $38.4 million for the same period in 2017. GAAP SG&A expenses were $23.7 million in the second quarter of 2018 compared to $26.5 million for the same period in 2017. GAAP R&D expenses were $10.4 million in the second quarter of 2018 compared to $10.8 million for the same period in 2017.

On a pro forma basis, during the second quarter of 2018, SG&A expenses were $21.7 million compared to $25.1 million for the same period in 2017. The 14% decrease in pro forma SG&A expenses in the second quarter of 2018 compared with the same period in 2017 was primarily related to cost reduction programs initiated in early 2018. Additionally, there were no restructuring charges incurred during the second quarter of 2018, compared to $1.0 million in restructuring charges incurred during the second quarter of 2017, related to the consolidation of similar positions during the integration of the business subsequent to the acquisition of Aegerion.

On a pro forma basis, during the second quarter of 2018, R&D expenses decreased 5% to $10.2 million compared to $10.7 million for the same period in 2017, reflecting cost savings initiatives.

GAAP net loss in the second quarter of 2018 was $31.3 million compared to GAAP net loss of $21.4 million during the same period in 2017.

On a pro forma basis, net loss in the second quarter of 2018 was $11.1 million, compared to a net loss of $1.4 million for the same period in 2017.

First Six Months of 2018 Financial Results

GAAP total net revenues for the first six months of 2018 were $59.4 million compared to $70.9 million for the same period of 2017, primarily as a result of timing of orders for both products in Brazil. The first six months of 2017 benefitted from $10.9 million of Brazil sales derived from the named patient sales program, whereas named patient sales in Brazil totaled $1.2 million in the first six months of 2018. Growth of $5.2 million, or 45%, in other foreign markets offset the $4.7 million, or 10% decrease in U.S. revenues, excluding the impact of the one-time deferred revenue recognition in 2017.

GAAP net revenues for JUXTAPID for the first six months of 2018 were $29.6 million compared to $36.7 million in same period in 2017. JUXTAPID revenues totaled $5.9 million in Brazil in the first half of 2017, whereas there were no named patient sales of JUXTAPID in the first half of 2018. Revenue growth of $1.7 million, or 19%, in other foreign markets helped offset the $2.8 million, or

13%, decrease in U.S. revenues in the most recent six month period compared to the comparable period of 2017.

GAAP net revenues for MYALEPT for the first six months of 2018 were $29.8 million compared to $34.1 million for the same period in 2017. MYALEPT revenues in Brazil totaled $5.0 million in the first half of the prior year compared to $1.2 million in the first half of 2018. Excluding the impact of the $2.3 million deferred revenue recognition in 2017, U.S. sales of MYALEPT declined $1.8 million, or 7%, which was more than offset by the growth in foreign markets other than Brazil where MYALEPT revenues more than doubled from $2.8 million in the first half of 2017 to $6.3 million in the same period of 2018.

GAAP total operating expenses for the first six months of 2018 were $69.6 million compared to total operating expenses of $73.6 million for the same period in 2017. GAAP SG&A expenses were $47.4 million in the first six months of 2018 compared to $51.0 million for the same period in 2017. GAAP R&D expenses were $22.1 million for the first six months of 2018 compared to $20.1 million for the same period in 2017.

On a pro forma basis, for the first six months of 2018, SG&A expenses decreased 10% to $43.3 million compared to $48.0 million for the same period in 2017 primarily as a result of cost reduction programs initiated in early 2018. There were no restructuring charges in the first six months of 2018, compared with restructuring charges of $2.5 million for the same period in 2017 which were related to the consolidation of similar positions during the integration of the business subsequent to the acquisition of Aegerion.

On a pro forma basis, for the first six months of 2018, R&D expenses increased 11% to $21.8 million compared to $19.7 million for the same period in 2017 due to increased clinical activity, partially offset by cost savings initiatives.

GAAP net loss for the first six months of 2018 was $64.1 million compared to GAAP net loss of $52.4 million during the same period in 2017.

Net loss on a pro forma basis for the first six months of 2018 was $24.7 million, compared to $10.1 million for the same period in 2017.

As of June 30, 2018, the Company’s consolidated unrestricted cash balance was $40.0 million, compared to $55.4 million at December 31, 2017. As of June 30, 2018, there were 18.9 million shares outstanding. Consolidated debt principal is $345.0 million, reflecting the amount of aggregate convertible and term loan debt issued by subsidiary Aegerion.