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argenx reports third quarter 2019 financial results and provides business update

On October 24, 2019 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported its financial results for the third quarter ended September 30, 2019 and provided a business update (Press release, argenx, OCT 24, 2019, View Source [SID1234542472]).

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"We have had another strong quarter of execution across our development pipeline and look forward to a busy remainder of the year that should set the stage for an exciting 2020. With efgartigimod, we are planning for three upcoming readouts, including Phase 3 ADAPT data in generalized myasthenia gravis, which remains on track with enrollment and for data readout in the second half of 2020; Phase 2 data in pemphigus vulgaris in the first half of 2020; and Phase 1 healthy volunteer data from the ENHANZE SC formulation of efgartigimod by the end of this year, which will guide our subcutaneuous development path forward. In tandem, we plan to initiate new trials of efgartigimod, cusatuzumab and ARGX-117 as we continue to build a multitude of opportunities across our pipeline and strengthen our focus within important therapeutic franchises," commented Tim Van Hauwermeiren, CEO of argenx.

THIRD QUARTER 2019 AND RECENT HIGHLIGHTS

argenx is executing on its "argenx 2021" vision to become a fully integrated, global immunology company, which includes its anticipated first commercial launch of efgartigimod in generalized myasthenia gravis (gMG) in 2021 and the building of two potential commercial franchises in neuromuscular and hematology with its three core assets: efgartigimod, cusatuzumab and ARGX-117.

Efgartigimod: First-in-class opportunity with potential across range of high-value autoimmune indications

Efgartigimod is a human IgG1 Fc fragment engineered for optimal blocking of FcRn. Treatment with efgartigimod is intended to result in a targeted reduction of IgG autoantibodies. argenx is evaluating efgartigimod across four indications where IgG autoantibodies are directly pathogenic, including:

· Generalized Myasthenia Gravis

· Global, multi-center Phase 3 ADAPT clinical trial, including ADAPT+ one-year open-label extension study, currently ongoing

· Based on current enrollment, argenx continues to expect topline data from ADAPT in second half of 2020

· Primary Immune Thrombocytopenia (ITP)

· Global Phase 3 program to include two registrational trials to be run concurrently

· First trial, ADVANCE, on track to start in second half of 2019 and will evaluate 10mg/kg intravenous (IV) efgartigimod on top of standard of care medication

· Second trial to evaluate 10mg/kg IV efgartigimod to induce IgG antibody reduction and clinical response followed by fixed dose 330mg subcutaneous (SC) injectable efgartigimod to maintain clinical benefit

· Pemphigus Vulgaris (PV)

· Interim data from ongoing Phase 2 proof-of-concept clinical trial expected in first half of 2020

· Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

· Phase 2 proof-of-concept clinical trial on track to start in second half of 2019

· Key opinion leader (KOL) event planned for December 5, 2019 to present Phase 2 trial design and market opportunity in CIDP

argenx is developing three formulations of efgartigimod to address the needs of patients, physicians and payors across indications and geographies, including IV efgartigimod and two SC formulations.

· A standalone SC formulation of efgartigimod as part of argenx’s collaboration with Halozyme enabling co-formulation of efgartigimod with Halozyme’s proprietary ENHANZE drug delivery technology

· Data from Phase 1 healthy volunteer (HV) trial expected by end of 2019

· After Phase 1 HV trial data are available, argenx to disclose path forward for testing in patients for ENHANZE SC formulation of efgartigimod, including potential bridging strategy in gMG

· argenx has exclusive access to ENHANZE technology for FcRn target

·A fixed 330mg SC injection to be dosed as maintenance treatment following IV induction; this formulation to be evaluated in a second Phase 3 ITP trial

Cusatuzumab: First-in-class opportunity with potential in hematological malignancies

Cusatuzumab is an anti-CD70 monoclonal antibody being developed under an exclusive global collaboration and license agreement with Janssen for the treatment of acute myeloid leukemia (AML), high-risk myelodysplastic syndromes and other hematological malignancies.

·Phase 2 and registration-directed clinical trial of cusatuzumab currently enrolling up to 150 patients with previously untreated AML who are not eligible for intensive chemotherapy

· In two-part trial, patients will first be randomized to receive one of two dose levels of cusatuzumab (10mg/kg and 20mg/kg) in combination with azacytidine (75mg/m2) followed by an expansion cohort to evaluate efficacy of the selected dose of cusatuzumab

ARGX-117: First-in-class anti-C2 antibody expected to enter clinic in first quarter 2020

ARGX-117 is a complement-targeting antibody against C2 with potential therapeutic applications in multiple autoimmune diseases. A Clinical Trial Application (CTA) is on track to be filed by end of 2019 with first-in-human trial expected to start in first quarter of 2020.

UPCOMING MILESTONES

·Before the end of 2019, argenx expects:

Data from Phase 1 HV trial of ENHANZE SC formulation of efgartigimod after which argenx will communicate on path forward for testing in patients with this co-formulation

· Start of first Phase 3 ADVANCE trial of efgartigimod in primary ITP

· Start of Phase 2 proof-of-concept trial of efgartigimod in CIDP

· In 2020, argenx expects:

· Data from Phase 2 proof-of-concept trial of efgartigimod in PV in first half

· First-in-human Phase 1 trial to start with ARGX-117 in first quarter

· Data from global Phase 3 ADAPT trial of efgartigimod in gMG in second half

·Announcement of fifth indication with efgartigimod and new pipeline asset ARGX-119

Update from ongoing cusatuzumab development

FINANCIAL OUTLOOK

Based on current development plan, argenx expects that its cash, cash equivalents and investments will fund planned operating and capital expense requirements up to the expected launch of efgartigimod in gMG in 2021. This development plan excludes any potential upcoming milestone payments under existing collaborations.

With the advancement of development of efgartigimod across indications, build-out of the argenx commercial organization, commitment to fund 40% of the development plan for cusatuzumab, and the expansion of its business plan, argenx expects operating and capital expense requirements to continue to increase year-over-year.

Actinium to Announce Interim Results from Pivotal Phase 3 SIERRA Trial on Conference Call Scheduled for Monday, October 28th

On October 24, 2019 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") reported that it will a host a conference call and webcast to highlight interim results from the pivotal Phase 3 SIERRA trial for its lead program, Iomab-B (Press release, Actinium Pharmaceuticals, OCT 24, 2019, View Source [SID1234542471]). The SIERRA trial (Study of Iomab-B in Elderly Relapse Refractory Acute Myeloid Leukemia) is a 150-patient, randomized 1:1 pivotal Phase 3 trial that is studying Iomab-B compared to physician’s choice of salvage chemotherapy in patients age 55 and above with active, relapsed or refractory AML or acute myeloid leukemia. The SIERRA trial is the only randomized Phase 3 trial to offer BMT, the only potentially curative treatment option, to this patient population.

The SIERRA trial reached 50 percent enrollment in July 2019. The call will highlight findings from the first 50 percent of patients in the SIERRA trial.

Conference Call and Webcast Details:
Time and Date: 12:00 PM ET on Monday, October 28th
Webcast link: View Source
U.S./Canada Toll Free Dial-in: (855) 698-6739
Participant Dial-in: (646) 402-9440
Conference ID: 0417

About Iomab-B

Iomab-B is an ARC or Antibody Radiation-Conjugate comprised of the anti-CD45 antibody apamistamab and the radioisotope iodine-131 that is intended to be a targeted conditioning agent prior to a BMT or bone marrow transplant. Iomab-B was developed at the Fred Hutchinson Cancer Research Center and has been studied in over 300 patients in multiple hematologic indications across 12 clinical trials in addition to the ongoing SIERRA study in older patients with active, relapsed or refractory AML or Acute Myeloid Leukemia prior to patients receiving an allogeneic BMT or bone marrow transplant. Iomab-B is Actinium’s lead targeting conditioning ARC in its multi-target, multi-indication targeted conditioning pipeline that includes the Iomab-B and Actimab-MDS programs for BMT and the Iomab-ACT program that will study a lower dose of Iomab-B for lymphodepletion prior to CAR-T and other cellular therapies.

Cambrex Releases Selected Preliminary Third Quarter 2019 Financial Results

On October 24, 2019 Cambrex Corporation (NYSE: CBM), the leading small molecule company providing drug substance, drug product and analytical services across the entire drug lifecycle, reported that in connection with the debt portion of the financing for the pending acquisition by an affiliate of the Permira Funds, they are making the following third quarter 2019 preliminary unaudited financial information available to prospective lenders (Press release, Cambrex, OCT 24, 2019, View Source [SID1234542469]).

The financial results presented above are unaudited and preliminary estimates that (i) represent the most current information available to management as of the date of this press release, (ii) are subject to completion of financial closing and procedures that could result in significant changes to the estimated amounts, and (iii) do not present all information necessary for an understanding of Cambrex’s financial condition as of, and its results of operations for the quarter ended, September 30, 2019. Accordingly, undue reliance should not be placed on such preliminary estimates.

Use of Non-GAAP Financial Measures
EBITDA and Adjusted EBITDA are non-GAAP financial measures ("non-GAAP financial measures"). Other companies may have different definitions of these non-GAAP financial measures, and as a result they may not be comparable with non-GAAP financial measures provided by other companies.

EBITDA and Adjusted EBITDA should not be considered alternatives to measurements required by U.S. GAAP, such as Operating profit, and should not be considered a measure of Cambrex’s liquidity.

Cambrex uses these non-GAAP financial measures, among several other metrics, to assess and analyze its operational results and trends. Cambrex also believes these measures are useful to investors because they are common operating performance metrics as well as metrics routinely used to assess potential enterprise value. Cambrex has provided a reconciliation of U.S. GAAP amounts to non-GAAP amounts within this press release.

Chugai Announces 2019 3rd Quarter Results

On October 24, 2019 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported its financial results for the third quarter of the fiscal year ended December 31, 2019 (Press release, Chugai, OCT 24, 2019, View Source [SID1234542468]).

Chugai reported record high revenues, operating profit and net income for the third consecutive quarter (Core-basis). Revenues increased by 19.3% due to a double-digit sales growth driven by the contribution of new products including the hemophilia A treatment Hemlibra and the immune checkpoint inhibitor Tecentriq, mainstay products and strong exports as well as large increases in royalties and other operating income related to Hemlibra. Operating profit increased by 65.6% due to a better cost to sales ratio as the proportion of in-house products increased in the total product mix.

Chugai raised forecasts for FY2019 following the strong nine-month results. The revenues forecast was raised by ¥87.5 billion to ¥680.0 billion from the original forecast reflecting a strong progress in both domestic sales and export to Roche. The increase in domestic sales was mainly driven by mainstay products and new products in the oncology area as well as Hemlibra. Royalties related to Hemlibra and one-time income also progressed well-beyond the original expectations, resulting in the raised forecast for revenues. Forecast for Core operating profit was also raised by ¥75.0 billion to ¥218.0 billion from the original forecast due to reasons including a better cost to sales ratio expected due to changes in the product mix from the original forecast.

In addition, the dividend forecast was revised to undecided. Reflecting the significant changes in the profit structures, year-end dividends will be decided based on the Company’s dividend policy* after the fiscal year ends.

R&D activities also progressed well. Satralizumab, the anti-interleukin-6 (IL-6) receptor recycling antibody created by Chugai, was filed for regulatory approval in EU and the U.S.. Satralizumab is a growth driver candidate following Actemra and Hemlibra, and a regulatory filing in Japan is also planned this year. Clinical development for another in-house project NXT007 commenced for the treatment of hemophilia A. In addition, Tecentriq achieved line extensions as the first immune checkpoint inhibitor in Japan approved for the treatment of extensive-stage small cell lung cancer and PD-L1-positive triple negative breast cancer.

"We had a very good third quarter as sales of new products and mainstay products, including Hemlibra, Tecentriq and Perjeta, grew beyond our expectations. At the same time, the start of global regulatory filings for satralizumab, a future growth driver, is proceeding as planned. Although we revised the full-year forecast upward based on the strong nine-month performance, the negative growth of the prescription drug market in Japan will likely continue, increasing the severity of the environment in the future. We remain focused in our efforts to archive the targets of the mid-term business plan IBI 21," said Tatsuro Kosaka, Chugai’s President and CEO.

*Regarding income distribution, taking into account the strategic funding needs and earning prospects, Chugai aims for a consolidated dividend payout ratio of 50% on Average in comparison with Core EPS to provide a stable allocation of profit to all shareholders.

Brain Cancer Patients Display Appropriate Immune Responses and Decreasing Tumor Biomarkers in AIVITA Biomedical’s Phase 2 Clinical Trial

On October 24, 2019 AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, reported updated data from its ongoing glioblastoma Phase 2 clinical trial investigating AIVITA’s platform immunotherapy targeting tumor-initiating cells (Press release, AIVITA Biomedical, OCT 24, 2019, View Source [SID1234542467]). In approximately 80% of treated patients, sequential blood plasma biomarker analyses have identified the induction of a type II hypersensitivity response, followed by a dendritic cell-mediated antibody response. This immunological response is accompanied by decreased tumor load in approximately 81% of those same patients, equivalent to 65% of the total number of treated patients.

Blood was collected from subjects at multiple time points, one week after each dose administration, and assayed for 450 different immune and tumor biomarkers. A robust type II hypersensitivity response was noted as an acute development at 2-3 weeks post-treatment, followed by dendritic cell maturation and the induction of an antibody-mediated response by 8 weeks post-treatment. Furthermore, the response is proportional to tumor load. The oncology literature associates this immunological profile with survival; indeed, 81% of the treated patients in the AIVITA study exhibiting this immunological profile also show a decrease of tumor markers.

"Blood analyses indicate that the AIVITA immunotherapy, in the presence of tumor, triggers an acute cytotoxic hypersensitivity followed by an antibody-mediated response and tumor marker reduction," said Dr. Gabriel Nistor, Chief Science Officer at AIVITA. "While very encouraging, only time will truly validate whether the appropriate immune response and decreasing tumor markers translate into patient survival."

AIVITA is currently conducting three distinct clinical studies in the USA investigating its platform immunotherapy, in patients with ovarian cancer, glioblastoma and melanoma. AIVITA is also seeking conditional commercial approval of its melanoma treatment in Japan. AIVITA uses 100% of proceeds from the sale of its ROOT of SKIN skincare line to support the treatment of women with ovarian cancer.

CLINICAL TRIAL DETAIL

OVARIAN CANCER

AIVITA’s ovarian Phase 2 double-blind study is active and enrolling approximately 99 patients who are being randomized in a 2:1 ratio to receive either the autologous tumor-initiating cell-targeting immunotherapy or autologous monocytes as a comparator.

Patients eligible for randomization and treatment will be those (1) who have undergone debulking surgery, (2) for whom a cell line has been established, (3) who have undergone leukapheresis from which sufficient monocytes were obtained, (4) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), and (5) who have completed primary therapy. The trial is not open to patients with recurrent ovarian cancer.

For additional information about AIVITA’s AVOVA-1 trial patients can visit: www.clinicaltrials.gov/ct2/show/NCT02033616

GLIOBLASTOMA

AIVITA’s glioblastoma Phase 2 single-arm study is active and is enrolling approximately 55 patients to receive the tumor-initiating cell-targeting immunotherapy.

Patients eligible for treatment will be those (1) who have recovered from surgery such that they are about to begin concurrent chemotherapy and radiation therapy (CT/RT), (2) for whom an autologous tumor cell line has been established, (3) have a Karnofsky Performance Status of > 70 and (4) have undergone successful leukapheresis from which peripheral blood mononuclear cells (PBMC) were obtained that can be used to generate dendritic cells (DC). The trial is not open to patients with recurrent glioblastoma.

For additional information about AIVITA’s AV-GBM-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03400917

MELANOMA

AIVITA’s melanoma Phase 1B open-label, single-arm study will establish the safety of administering anti-PD1 monoclonal antibodies in combination with AIVITA’s tumor-initiating cell-targeting immunotherapy in patients with measurable metastatic melanoma. The study will also track efficacy of the treatment for the estimated 14 to 20 patients. This trial is not yet open for enrollment.

Patients eligible for treatment will be those (1) for whom a cell line has been established, (2) who have undergone leukapheresis from which sufficient monocytes were obtained, (3) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), (4) who have either never received treatment for metastatic melanoma or were previously treated with enzymatic inhibitors of the BRAF/MEK pathway because of BRAF600E/K mutations and (5) are about to initiate anti-PD1 monotherapy.