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).

On January 9, 2020 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported plans to report 2019 fourth quarter and full year financial results on February 6, 2020 (Press release, Ligand, JAN 9, 2020, View Source [SID1234552927]). Ligand’s CEO John Higgins, President and COO Matt Foehr and Executive Vice President and CFO Matt Korenberg will host the conference call.

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2019 Fourth Quarter and Full Year Earnings Conference Call

What:

Ligand conference call to discuss financial results and provide general business updates

Date:

Thursday, February 6, 2020

Time:

4:30 p.m. Eastern time (1:30 p.m. Pacific time)

Conference Call:

Dial (833) 591-4752 within the U.S.

Dial (720) 405-1612 outside the U.S.

Conference ID is 1150048

Webcast:

Live conference call webcast and replay accessible at www.ligand.com

On January 9, 2020 Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, reported the expansion of its Series A financing with an additional investment of $14.1 million led by Roche Venture Fund and with participation from other investors, bringing the total company financing to more than $50 million to date (Press release, Jasper Therapeutics, JAN 9, 2020, View Source [SID1234552943]). The initial Series A round was led by Abingworth LLP and Qiming Venture Partners USA, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC.

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Jasper plans to use the proceeds to advance and expand the study of its lead clinical asset, JSP191. A humanized antibody targeting CD117 on hematopoietic stem cells, JSP191 is designed to replace toxic chemotherapy and radiation therapy as conditioning regimens to prepare patients for curative stem cell and gene therapy. JSP191 is the only antibody of its kind in clinical development as a single conditioning agent for people undergoing curative hematopoietic cell transplantation.

This investigational agent is currently being evaluated in a Phase 1/2 dose-escalation and expansion study as a conditioning agent to enable stem cell engraftment in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that resulted in poor outcome. Initial positive results from this ongoing clinical trial were presented in an oral session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2019. Jasper plans to expand the Phase 1/2 clinical study to include patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) receiving hematopoietic cell transplant. The development of JSP191 is supported by a collaboration with the California Institute for Regenerative Medicine (CIRM).

About Hematopoietic Cell Transplantation
Blood-forming, or hematopoietic, stem cells are rare cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Replacement of the defective or malignant hematopoietic stem cells in the patient’s bone marrow by transplantation and engraftment of healthy stem cells is the only cure for most of these life-threatening conditions. Successful transplantation is currently achieved by subjecting patients to toxic treatment with radiation and/or chemotherapy followed by transplantation of a donor or gene-corrected hematopoietic cell graft. These toxic regimens cause DNA damage and lead to short- and long-term toxicities, including unwanted damage to organs and prolonged hospitalization. As a result, many patients who could benefit from a hematopoietic cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191
JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant. IND-enabling studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

On January 9, 2020 American Brain Tumor Association (ABTA) reported a new partnership with the Uncle Kory Foundation to accelerate brain tumor research (Press release, American Brain Tumor Association, JAN 9, 2020, View Source [SID1234552960]). Through this partnership, the Uncle Kory Foundation will invest $100,000 in the ABTA research program to fund two glioblastoma research projects. For more than 46 years, the ABTA has served the brain tumor community by funding research and delivering patient programs and services.

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"Through collaboration with the Uncle Kory Foundation, we aim to leverage the strengths of each organization to maximize impact and drive change within this underserved disease population," said Ralph DeVitto, president and chief executive officer of the ABTA.

With an established and sophisticated mechanism to evaluate and foster brain tumor research, the ABTA has earned recognition as a trusted and committed research conduit for organizations and foundations who aim to advance brain tumor research. Since 1976, the ABTA has awarded more than $32 million in research grants to scientists from around the world to discover more about the causes, effects, diagnosis and treatment of pediatric and adult brain tumors.

The Uncle Kory Foundation was founded in 2014 in honor of Kory Hunter, with the mission to advance innovative and collaborative brain cancer research to specifically improve the survival rate and treatment of those diagnosed with glioblastoma.

"This collaboration is the key to success," said Renee Vachon, director of operations of Uncle Kory Foundation. "By leveraging the ABTA research platform we can adhere to our mission of funding innovative research for glioblastoma in a concise and effective effort."

The Uncle Kory Foundation is an important new partner for the ABTA’s research initiatives. The ABTA is grateful to them and other established research collaborators within the brain tumor community, including the Joel A. Gingras, Jr. Memorial Foundation and Humor to Fight the Tumor.

On January 9, 2020 ADC Therapeutics SA, a clinical-stage oncology-focused biotechnology company pioneering the development and commercialization of highly potent antibody drug conjugates (ADCs) for patients suffering from hematological malignancies and solid tumors, reported positive results from the pivotal 145-patient Phase 2 clinical trial of loncastuximab tesirine (ADCT-402) for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) (Press release, ADC Therapeutics, JAN 9, 2020, View Source [SID1234596050]).

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To date, loncastuximab tesirine has achieved an overall response rate (ORR) of 45.5% (66/145 patients), including 20% complete responses and 25.5% partial responses, across a broad population of relapsed or refractory DLBCL patients, even those who are difficult to treat. Comparably, the ORR in the 183-patient Phase 1 clinical trial of loncastuximab tesirine at the initial dose used in Phase 2 was 41.4% (29/70 patients), including 21.4% complete responses and 20% partial responses. Loncastuximab tesirine has demonstrated manageable toxicity in patients with relapsed or refractory DLBCL. The most common grade ≥3 treatment-emergent adverse events in the Phase 2 clinical trial were neutropenia, thrombocytopenia and increased gamma-glutamyltransferase.

Jay Feingold, MD, PhD, Senior Vice President and Chief Medical Officer at ADC Therapeutics, said, "These data exceeded our primary endpoint target and reinforce the significant single-agent anti-tumor activity and manageable toxicity profile of loncastuximab tesirine in patients with relapsed or refractory DLBCL who have failed established therapies. Loncastuximab tesirine has demonstrated its potential to fill a critical unmet need for a new therapy and become a key part of the treatment paradigm for all heavily pretreated patients with DLBCL. We plan to present final data from the pivotal Phase 2 clinical trial at a future scientific meeting."

Chris Martin, Chief Executive Officer of ADC Therapeutics, said, "We look forward to submitting a BLA to the U.S. Food and Drug Administration for accelerated approval of loncastuximab tesirine for the treatment of relapsed or refractory DLBCL patients who have failed two or more treatment regimens later this year and we are building our commercial organization in anticipation of a launch in the second quarter of 2021."

The single-arm, multi-center, open-label Phase 2 clinical trial evaluated the safety, efficacy and pharmacokinetics of loncastuximab tesirine as a monotherapy in patients with relapsed or refractory DLBCL. Patients received 30-minute intravenous infusions of loncastuximab tesirine once every three weeks at a dose of 150 μg/kg for the first two cycles, followed by 75 μg/kg for subsequent cycles for up to one year or until disease progression, unacceptable toxicity, or other discontinuation criteria, whichever occurred first.

About Loncastuximab Tesirine
Loncastuximab tesirine (formerly ADCT-402) is an antibody drug conjugate (ADC) composed of a humanized monoclonal antibody directed against human CD19 and conjugated through a linker to a pyrrolobenzodiazepine (PBD) dimer cytotoxin. Once bound to a CD19-expressing cell, loncastuximab tesirine is designed to be internalized by the cell, following which the warhead is released. The warhead is designed to bind irreversibly to DNA to create highly potent interstrand cross-links that block DNA strand separation, thus disrupting essential DNA metabolic processes such as replication and ultimately resulting in cell death. CD19 is a clinically validated target for the treatment of B-cell malignancies. Loncastuximab tesirine is being evaluated in a pivotal Phase 2 clinical trial in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) (NCT03589469), a Phase 1b trial in combination with ibrutinib in patients with R/R DLBCL or mantle cell lymphoma (MCL) (NCT03684694) and a Phase 1b trial in combination with durvalumab in patients with R/R DLBCL, MCL or follicular lymphoma (NCT03685344). The U.S. Food and Drug Administration granted orphan drug designation to loncastuximab tesirine for the treatment of R/R DLBCL and MCL.