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argenx Provides Strategic Outlook Advancing Late-Stage Pipeline Towards ‘argenx 2021’ Vision

On January 9, 2020 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 a strategic outlook for 2020 outlining key priorities for its broad pipeline and path towards achieving its ‘argenx 2021’ integrated commercial vision (Press release, argenx, JAN 9, 2020, View Source [SID1234552916]).

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"We begin 2020 in an exciting position, having met all our objectives for our clinical programs. This includes the completion of enrollment of our Phase 3 ADAPT trial of efgartigimod in gMG, the launch of key efgartigimod clinical trials in ITP and CIDP, and the initiation of cusatuzumab clinical trials in two AML settings with Janssen. In addition, we’re announcing today positive proof-of-concept data for efgartigimod in PV, our third ‘beachhead’ indication, further demonstrating our initial development strategy of targeting pathogenic autoantibodies and creating commercial opportunities in several therapeutic areas. Looking forward to the remainder of 2020, we plan up to five registrational efgartigimod trials and further expansion of the cusatuzumab global development plan with Janssen," said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

"Most importantly, we are continuing to execute on the ‘argenx 2021’ vision to become a global, integrated immunology company with our first launch of efgartigimod in gMG expected in 2021. At the core of this growth strategy is a commitment to expanding our early-stage pipeline with immunology breakthroughs and advancing our late-stage candidates while extending our reach to bring first-in-class medicines to patients," continued Mr. Van Hauwermeiren.

argenx 2021 Vision

argenx continues to execute on its plan to become a fully integrated immunology company through its "argenx 2021" vision, including building two initial commercial franchises in neuromuscular indications and hematology/oncology and expanding its global presence encompassing Boston, Ghent and Tokyo. As part of this vision, argenx highlights:

·Leadership in FcRn and its therapeutic immunology potential:

· On track to launch first FcRn antagonist with efgartigimod in generalized myasthenia gravis (gMG) in 2021

· Up to five registrational trials expected to be ongoing in 2020 across four targeted indications (gMG, immune thrombocytopenia (ITP), chronic inflammatory demyelinating polyneuropathy (CIDP) and pemphigus vulgaris (PV))

· Fifth efgartigimod indication expected to be announced in 2020

· Further research underway exploring therapeutic potential of FcRn modulation

· Launch of MyRealWorld MG:

· First-of-its-kind in MG, real-world evidence study launched; aiming to enroll approximately 2000 patients globally with support from regional patient advocacy organizations

· Disease and treatment burden to be documented to support proposed value of efgartigimod

· Strong financial foundation:

·Following November 2019 equity offering, argenx expects to report 2019 year-end cash and cash equivalents of approximately $1.5 billion

Pipeline Updates and 2020 Priorities

argenx today is reporting positive proof-of-concept data in PV, the third beachhead indication as part of the broad efgartigimod development strategy:

· 23 patients (10mg/kg or 25mg/kg efgartigimod) were evaluated for efficacy in an adaptive Phase 2 trial aiming to establish optimal treatment regimen

· Clear correlation demonstrated between pathogenic IgG reduction and Pemphigus Disease Area Index score improvement

· 78% (18/23) of patients achieved rapid disease control; median time to disease control for both monotherapy and combination therapy is 14-15 days

·Fast clinical remission (CR) observed in 70% (5/7) of patients receiving optimized dosing regimen

·CR achieved within 2-10 weeks

· Optimized dosing regimen determined to be at least biweekly dosing of efgartigimod in combination with oral prednisone (0.25-0.5mg/kg)

·Potential of corticosteroid sparing demonstrated

·Independent data review committee concluded safety and tolerability to be favorable

· Patients still in trial in extended dosing cohort; detailed results of Phase 2 data to be presented during medical meeting in 2020

· Data support advancing to registrational trial expected to start in second half of 2020

Within its neuromuscular franchise, argenx is evaluating:

· Efgartigimod in gMG:

· Enrollment completed of 167 gMG patients in Phase 3 ADAPT trial with 10mg/kg IV efgartigimod; topline results from ADAPT now expected in mid-2020

· Biologics License Agreement (BLA) for gMG expected to be filed in fourth quarter of 2020

· Plan to engage with U.S. Food and Drug Administration (FDA) on potential bridging strategy for 1000mg subcutaneous (SC) ENHANZE-efgartigimod in gMG

· Efgartigimod in CIDP:

· Phase 2 ADHERE trial initiated in CIDP with SC ENHANZE-efgartigimod

Within its hematology/oncology franchise, argenx is evaluating:

· Efgartigimod in ITP:

· Phase 3 ADVANCE registrational trial initiated evaluating approximately 150 primary ITP patients dosed with 10mg/kg IV efgartigimod for both induction and maintenance of platelet response

· ADVANCE SC trial expected to initiate in second half of 2020 evaluating 10mg/kg IV efgartigimod for induction of platelet response and fixed dose of SC efgartigimod for maintenance

· Additional small confirmatory IV trial as part of registrational ITP program expected to initiate in first half of 2020

· Orphan designation granted by European Medicines Agency (EMA) for ITP; designation granted by FDA in February 2019

· Cusatuzumab in collaboration with Janssen:

· CULMINATE trial in combination with azacitidine to continue enrollment of newly diagnosed "unfit" AML patients

· Phase 1b platform trial underway in various AML subpopulations and settings with initial trial evaluating combinations of cusatuzumab, venetoclax and azacitadine; additional trials expected to launch under platform trial in first half of 2020

· Randomized Phase 2 trial in higher-risk myelodysplastic syndromes (MDS) expected to launch in first half of 2020

·Data update from cusatuzumab development expected in 2020

argenx continues to expand its early-stage pipeline with first-in-class antibodies against immunologic targets:

· Phase 1 trial of ARGX-117 in healthy volunteers expected to begin in first quarter of 2020

· Multiple doses and formulations (IV and SC with Halozyme ENHANZE technology) to be evaluated as part of dose-finding work

· Following analysis of Phase 1 data in fourth quarter of 2020, argenx expects to launch Phase 2 program in multifocal motor neuropathy (MMN) within its neuromuscular franchise and develop in additional indications

· Lead optimization work on ARGX-118 for airway inflammation to continue in 2020

· New product candidate ARGX-119 expected to be announced in 2020

JP Morgan Conference Presentation and Webcast

Tim Van Hauwermeiren, Chief Executive Officer, will present at the 38th Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 2:00 p.m. PST in San Francisco, followed by a breakout question and answer session.

The live webcast of both the presentation and question and answer session that follows may be accessed on the homepage of the argenx website at www.argenx.com. Shortly after the presentation, a replay of the webcast will be available for 90 days on the argenx website.

About efgartigimod

Efgartigimod is an IgG Fc fragment engineered to optimally antagonize the neonatal Fc Receptor (FcRn) for the treatment of IgG-mediated autoimmune diseases. FcRn plays a central role in rescuing IgG from degradation in the lysosome through a recycling pathway. Through inhibition of FcRn, efgartigimod leads to fast depletion of the disease-causing IgG autoantibodies. Efgartigimod binds in the same way as endogenous IgG, the natural ligand of FcRn, and has been engineered with ABDEG mutations to increase its affinity for FcRn while preserving the characteristic pH-dependent binding, contributing to its long serum half-life, pharmacodynamic effect and potentially enhanced tissue penetration. The development work on efgartigimod is conducted in close collaboration with Prof. E. Sally Ward (Department of Molecular and Cellular Medicine, Texas A&M University Health Science Center, College Station, TX; Center for Cancer Immunology, University of Southampton, Southampton, UK).

AMAG Pharmaceuticals Announces Leadership Transition, Results of Strategic Review to Unlock Shareholder Value and Financial Update

On January 9, 2020 AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) reported the initiation of a leadership transition, the decision to divest Intrarosa and Vyleesi and financial updates (Press release, AMAG Pharmaceuticals, JAN 9, 2020, View Source [SID1234552915]).

Leadership Transition & Business Updates
AMAG announced that William Heiden plans to step down as AMAG’s President and Chief Executive Officer, and that the Company’s Board of Directors will immediately initiate a search for his successor, which it expects to complete by mid-2020. Mr. Heiden will remain in his role as President and CEO until a successor is appointed.

AMAG also recently completed a robust review of its product portfolio and strategy and engaged Goldman Sachs and Co., LLC as its financial advisor to assist in the review. The guiding principles for this strategic review included driving near- and long-term profitability and enhancing shareholder value. As a result of this review, AMAG will divest Intrarosa (prasterone) and Vyleesi (bremelanotide). The Company has received preliminary expressions of interest to acquire/sub-license the rights to these products. AMAG’s 2020 financial guidance indicates a return to positive adjusted EBITDA, reflecting the continued growth of Feraheme (ferumoxytol injection) and a significant reduction in operating expenses, primarily associated with the divestiture of Intrarosa and Vyleesi. The 2020 revenue guidance reflects a range of potential revenue scenarios for Makena (hydroxyprogesterone caproate injection) given the uncertainty caused by the FDA Advisory Committee meeting and soft fourth quarter results.

"The strategic decisions announced today will allow AMAG to leverage its commercial strengths and proven development and regulatory capabilities while focusing on core value drivers: developing ciraparantag and AMAG-423; driving the continued growth of Feraheme, which funds our two pipeline assets; and continuing our work to retain patient access to Makena," said Mr. Heiden. "It has been an honor and a privilege to lead the team at AMAG. We’ve achieved numerous successes and overcome many challenges. As we implement the strategic shift announced today, my fellow directors and I believe that this is the right time for the board to identify a new CEO for the next leg of AMAG’s journey."

Mr. Heiden added, "We continue to believe in the significant long-term potential of Intrarosa and Vyleesi. However, the uncertainty around the long-term durability of Makena revenues makes it challenging to invest in both our promising pipeline and in the physician and consumer marketing required to support these two new products. Given the significant future commercial potential of ciraparantag and AMAG-423 to fulfill unmet medical needs of patients, AMAG made the difficult decision to divest Intrarosa and Vyleesi. The Company is seeking a transaction with a party that can make the investments necessary to maximize the value of these two important women’s healthcare products."

In addition to the announced CEO transition, Chief Financial Officer Ted Myles will assume the additional role of Chief Operating Officer, effective immediately, expanding his responsibilities to include technical operations, global supply chain and quality. General Counsel Joseph Vittiglio will assume the additional role of Chief Business Officer, focusing on managing the divestiture of Intrarosa and Vyleesi and out-licensing opportunities in ex-U.S. territories, primarily for AMAG’s development-stage programs.

Gino Santini, Chairman of AMAG’s Board, said, "The Board is committed to pursuing strategies that will unlock value for AMAG shareholders and better leverage the Company’s strengths and proven capabilities. We are confident that taking the strategic actions announced today will position AMAG for future growth and enable the Company to better serve patients."

Mr. Santini continued, "The Board would like to thank Bill for his visionary leadership, transforming AMAG from a single-product company to one with an in-house clinical development team that has gained three FDA product approvals in the last two years, and is progressing two late-stage development products through the regulatory process. For nearly eight years, Bill developed and executed on strategies to invest in products that have benefited hundreds of thousands of patients and which hold the potential of reaching many more in the future. The Board looks forward to working with Bill on a seamless transition, as well as continuing to work with AMAG’s outstanding executive leadership team to drive near- and long-term results."

Preliminary 2019 Financial Results and 2020 Financial Guidance
AMAG announced preliminary unaudited fourth quarter and full year 2019 financial results and provided 2020 financial guidance, reflecting the Company’s focus on profitability in 2020. The Company expects to report final financial results for the fourth quarter and audited results for the full year of 2019 in early March.

Preliminary, Unaudited Fourth Quarter Financial Results
____________________
1 Includes the recognition of $16M of collaboration revenue due to the termination and settlement agreement entered into with Daiichi Sankyo, Inc. (DSI) in December 2019 related to a clinical trial collaboration agreement that AMAG acquired as part of the Perosphere acquisition. As part of the settlement, AMAG received $10M in cash from DSI in December 2019.
2 Operating loss does not include the impact of material impairment charges or the associated acceleration of amortization, which are likely to be recognized in the 2019 audited financial statements.
3 See reconciliation of GAAP to non-GAAP preliminary financial results at the conclusion of this press release.

The Company ended 2019 with approximately $170 million in cash and investments and $320 million of 2022 convertible notes (principal amount outstanding).

Key priorities for 2020 include:

Complete successful CEO transition

Divest Intrarosa and Vyleesi to align with the new strategic direction

Drive continued Feraheme growth

Work with the FDA to maintain patient access to Makena

Advance ciraparantag and AMAG-423 development programs

Pursue ex-US portfolio partnering opportunities

Meet/exceed financial guidance
2020 Financial Guidance4

Mr. Myles stated, "With the divestiture of Intrarosa and Vyleesi and the associated expense reductions, the AMAG of 2020 will be more streamlined and focused to drive Feraheme growth, support the development of our two pipeline assets and deliver positive adjusted EBITDA. While we remain committed to working with the FDA to maintain patient access to Makena, we will be managing Makena-related expenses so that the product is cash flow positive. Our financial guidance includes a reduction of operating expenses of more than $100 million in 2020 relative to 2019. We believe our 2020 plan, including the revised capital allocation strategy, maintains our strong commitment to patients and best positions AMAG to generate sustainable, long-term shareholder value."

INFORMATION FOR LIVE AUDIO WEBCAST AT THE 38TH J.P. MORGAN HEALTHCARE CONFERENCE
The Company will provide a live update at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco on Thursday, January 16, 2020 at 9:30 a.m. PT (12:30 p.m. ET). A live audio webcast of the presentation and the

4 See reconciliations of 2020 GAAP to non-GAAP financial guidance at the conclusion of this press release. 2020 financial guidance reflects management’s current assumptions about the potential impact of multiple scenarios across our product portfolio, including (i) various potential regulatory outcomes related to Makena and (ii) that the divestitures of Intrarosa and Vyleesi will be reported in discontinued operations for accounting purposes in 2020. Therefore, 2020 financial guidance excludes revenue and expenses related to Intrarosa and Vyleesi.

following breakout session will be accessible in the Investors section of AMAG’s website at www.amagpharma.com. on January 16, 2020 at 9:30 a.m. PT (12:30 p.m. ET). Following the conference, the webcast will be archived on the Company’s website until February 17, 2020.

USE OF NON-GAAP FINANCIAL MEASURES
AMAG has presented certain non-GAAP financial measures, including non-GAAP adjusted EBITDA (earnings before income taxes, depreciation and amortization). These non-GAAP financial measures exclude certain amounts, expenses or income, from the corresponding financial measures determined in accordance with accounting principles generally accepted in the U.S. (GAAP). Management believes this non-GAAP information is useful for investors, taken in conjunction with AMAG’s GAAP financial statements, because it provides greater transparency regarding AMAG’s operating performance. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of AMAG’s operating results as reported under GAAP, not as a substitute for GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures are included in the tables accompanying this press release.

Agenus Announces the Appointment of Dr. Jennifer Buell to the position of President and COO

On January 9, 2020 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of agents that activate immune response to cancers, is reported the appointment of Dr. Jennifer Buell to President and COO (Press release, Agenus, JAN 9, 2020, View Source [SID1234552914]).

Dr. Jennifer Buell, President & COO of Agenus

"Jen’s tenure with Agenus, her exceptional leadership and cultural attributes, and her deep understanding of our science and operations makes her the top candidate for this newly created position," said Garo H. Armen, Ph.D., Chairman and CEO of Agenus. He added, "I am very excited to partner with Jen as we set to realize Agenus’ vision of defeating cancer in the coming years."

After her tenure at Harvard Clinical Research Institute, Dr. Buell rejoined Agenus in 2013 as the Head of Global R&D operations. She was subsequently appointed as Chief Communications and External Affairs Officer, and then, to the position of Chief Operating Officer.

Dr. Buell brings over 20 years of biopharmaceutical industry experience and knowledge. Her efforts in the rapid advancement of discovery candidates through development has resulted in Agenus’ record of advancing 13 I-O candidates to the clinic in the past four years. Her experience also includes extensive communication with internal and external constituencies, regulators, investors, and collaborators. Dr. Buell’s previous operational and research experience include her tenures at Bristol-Myers Squibb and Harvard Clinical Research Institute. Dr. Buell obtained her PhD in Cellular, Biochemical, and Molecular Biochemistry with an MS in Biostatistics from Tufts University in Boston.

Grey Wolf completes £2.5 million Series A2 financing for development of therapies targeting ERAP2

On January 9, 2020 Grey Wolf Therapeutics, a drug discovery biotechnology company focused on developing first-in-class therapies for immuno-oncology (IO), reported it has completed a £2.5 million ($3.3 million) Series A2 financing round with existing leading international healthcare investors Andera Partners and Canaan (Press release, Grey Wolf Therapeutics, JAN 9, 2020, View Source [SID1234552910]).

The new funding – all from existing investors – will allow the company to accelerate development of therapies targeting endoplasmic reticulum aminopeptidase 2 (ERAP2), following many positive signals of its potential. Funds will also be used to continue to drive the lead endoplasmic reticulum aminopeptidase 1 (ERAP1) modulator program. For context, both of Grey Wolf’s novel ERAP approaches are aimed at directly altering tumor cells, illuminating them for attack and destruction by the immune system. The goal is to exploit this increased tumor visibility in monotherapy and to extend the therapeutic benefit of already approved immunotherapies to many more cancers. The company is developing small molecule modulators of ERAP1 and ERAP2, two key proteins in the antigen presentation pathway, to change the antigen repertoire of tumors and thereby increase the number and range of cancer-related antigens, including neoantigens, presented on tumor cells available to engage an immune response.

"We are delighted to have the continued support of Andera and Canaan." said Peter Joyce, Chief Executive Officer and Co-Founder. "The financing reflects growing potential in both our ERAP1 and ERAP2 approaches. We continue to see momentum, both in our own work and in the broader scientific community. These funds will allow us to further capitalize on this opportunity and expand our leadership position in the discovery and development of both ERAP1 and ERAP2 modulators."

Grey Wolf is expanding efforts around ERAP2 for two reasons. First, clinical data continues to demonstrate that tumors which are more visible to the immune system show improved responses to checkpoint inhibitors. One such example is the recent November 2019 paper published in Nature Medicine by the Chan group at MSKCC. The results are consistent across patients which either have a higher tumor mutational burden, heterozygosity of the Class I HLA locus or greater structural / sequence divergence at the Class I HLA locus. Second, the company has developed unique insight into the targeting of the ERAP enzymes through the lead program ERAP1 and validated the role for ERAP inhibition in modulating the cancer-related antigen repertoire. These in-house data provide compelling evidence that the therapies could have a real impact in the treatment of oncology.

"We have continued to generate data showing that modulation of both ERAP pathways drives change to the cancer-related antigen repertoire," said Tom McCarthy, Executive Chairman and Co-Founder of Grey Wolf Therapeutics. "Data clearly demonstrates that modulation of ERAP2 drives an altogether different change to the antigen repertoire, when compared with ERAP1 modulation, due to ERAP2’s clearly differentiated peptide substrate specificities. With this investment and the prior knowledge base within Grey Wolf we will be able to accelerate the ERAP2 program quickly through optimization, building on our leading position in ERAP disease-related biology."

The additional funds represent further confidence from investors. "Grey Wolf have already provided compelling insight into the potential of ERAP1 modulation," said Raphaël Wisniewski, Partner at Andera Partners. "This timely investment further underlines our excitement in these approaches. Grey Wolf are fast becoming true experts in modulation of ERAP biology for treatment of cancer." Brent Ahrens, General Partner at Canaan, added: "Canaan continues to be excited by the potential of targeting the ERAP pathway and are impressed with the progress the team has made in such a short space of time since closing Series A. The additional A2 investment enables the Company to push forward ERAP2 whilst maintaining the momentum on the lead ERAP1 program."

Oncolytics Biotech® Announces Key Opinion Leader Call Conducted by ROTH Capital Partners to Discuss Multiple Myeloma

On January 9, 2020 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported that ROTH Capital Partners (ROTH) will conduct a Key Opinion Leader call today at 10:30 am ET for their institutional clients (Press release, Oncolytics Biotech, JAN 9, 2020, View Source [SID1234552909]). The call, "Proteasome Inhibitors Augment Pelareorep in Multiple Myeloma" will feature Dr. Flavia Pichiorri Ph.D. and Dr. Craig Hofmeister M.D., both of whom are working on ongoing multiple myeloma studies with Oncolytics’ oncolytic virus, pelareorep.

The equity research team at ROTH will host and moderate the call for their clients, with a focus on recent data presented at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, and how it could impact the design of future multiple myeloma trials with oncolytic viruses versus the standard of care.

Management plans to provide a recap of the call next week, including key messages and insights provided by Dr. Pichiorri and Dr. Hofmeister.

Dr. Flavia Pichiorri Ph.D., Judy and Bernard Briskin Center for Multiple Myeloma Research, City of Hope, Duarte, CA and Department of Hematologic Malignancies Translational Science, Beckman Research Institute, City of Hope, Duarte, CA.

Dr. Craig Hofmeister M.D., Winship Cancer Institute /Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA.

About Pelareorep

Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.