On January 9, 2023 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported its strategic priorities for 2023 and provided preliminary financial results for the full year 2022, including global net product sales of VYVGART (efgartigimod alfa-fcab) (Press release, argenx, JAN 9, 2023, View Source [SID1234626030]).

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"We had a landmark year in 2022, marking our first as a fully integrated immunology company transforming the treatment paradigm of generalized myasthenia gravis, and one which will stand as only the very beginning of what we expect to achieve as an organization," said Tim Van Hauwermeiren, Chief Executive Officer, argenx. "Looking forward, we will be expanding our patient reach both geographically and through the anticipated U.S. approval and launch of subcutaneous efgartigimod in March. By the end of 2023, we will be active in 15 IgG- and complement-mediated autoimmune diseases as we work to uncover the full breadth of our differentiated pipeline with key data readouts from additional indications of efgartigimod, as well as ARGX-117 and ARGX-119."

2023 Strategic Priorities

argenx will focus on four strategic priorities in 2023 to drive sustained growth and value creation as part of its ‘argenx 2025’ vision and a path to profitability.

Reach More Patients with VYVGART

argenx is planning for multi-dimensional expansion to reach more patients with VYVGART, its first-in-class neonatal Fc receptor blocker. This includes generalized myasthenia gravis (gMG) patients through regulatory approvals in new regions and the launch of its subcutaneous (SC) product offering, as well as a new autoimmune indication with the VYVGART regulatory submission for ITP in Japan.

Prescription Drug User Fee Act (PDUFA) target action date of March 20, 2023, for U.S. Food and Drug Administration approval decision on SC efgartigimod for gMG

Regulatory approval decision on SC efgartigimod for gMG expected in EU in fourth quarter of 2023

Submission for marketing approval of SC efgartigimod for gMG expected in Japan in first quarter of 2023

Regulatory approval decisions of VYVGART for gMG expected in Canada in third quarter of 2023 and in China and Israel by end of 2023

gMG launch in France, United Kingdom and Italy expected by year-end 2023 following review of respective reimbursement dossiers

Submission for Japan marketing approval of VYVGART for ITP expected in mid-2023

Pioneer Development of FcRn Class with New Clinical and Translational Data

argenx aims to solidify its FcRn leadership by expanding the scope of IgG-mediated autoimmune diseases in development and further demonstrating the potential of FcRn blockade with three Phase 3 trial readouts, one proof-of-concept trial readout, and a commitment to a ‘bedside-to-bench’ translational biology effort. By the end of 2023, efgartigimod is expected to be approved, in regulatory review or in development in 13 severe autoimmune diseases.

ADHERE: Topline data in chronic inflammatory demyelinating polyneuropathy (CIDP) now expected in second quarter of 2023; Stage B enrollment has surpassed the initial projected target of 130 patients

ADDRESS: Topline data in pemphigus expected in second half of 2023

ADVANCE-SC: Topline data from SC trial in ITP expected in second half of 2023

Proof-of-concept data in post-COVID-19 postural orthostatic tachycardia syndrome (PC-POTS) expected in fourth quarter of 2023

Registrational trial to start in TED in fourth quarter of 2023

Proof-of-concept trials to start in ANCA-associated vasculitis and AMR in kidney transplant in fourth quarter of 2023; AAV trial to be run through IQVIA collaboration

Externally sponsored research studies to launch in IgG-mediated neuromuscular autoimmune diseases in 2023

Translational work ongoing to understand potential disease-modifying effect of FcRn mechanism

Drive Earlier-Stage Immunology Opportunities Towards Clinical Proof-of-Concept

Beyond efgartigimod, argenx is advancing a robust portfolio of innovative clinical programs, including ARGX-117 (C2 inhibitor) and ARGX-119 (muscle-specific kinase (MuSK) agonist). Both programs have the potential to be first-in-class opportunities for multiple severe autoimmune indications.

ARDA: Interim data from proof-of-concept trial of ARGX-117 in multifocal motor neuropathy expected mid-2023

Proof-of-concept trial of ARGX-117 expected to start following regulatory discussions for prevention of delayed graft function after kidney transplantation

Dermatomyositis selected as third autoimmune indication for development of ARGX-117

Phase 1 dose-escalation trial of ARGX-119 in healthy volunteers to start in first quarter of 2023 with subsequent Phase 1b trial to assess early signal detection in patients

Build Immunology Innovation Ecosystem to Drive Long-Term Pipeline Growth

argenx continues to invest in its discovery engine, the Immunology Innovation Program, to foster a robust innovation ecosystem and drive early-stage pipeline growth. argenx expects to nominate one new development candidate in 2023.

Preliminary* Fourth Quarter and Full Year 2022 Financial Results

argenx also announced today preliminary* global net VYVGART revenues for the fourth quarter and full-year 2022 of approximately $175 million and $402 million, respectively.

As of December 31, 2022, argenx had approximately $2.2 billion in cash, cash equivalents and current financial assets*. Based on its current operating plans and a projected 2023 cash burn of approximately $500 million, argenx expects its existing cash, cash equivalents and current financial assets, together with anticipated future product revenues, to fund the company to profitability.

The preliminary selected financial results are unaudited, subject to adjustment, and provided as an approximation in advance of the Company’s announcement of complete financial results in March 2023.

41st Annual J.P. Morgan Healthcare Conference Presentation and Webcast

Mr. Van Hauwermeiren will highlight these updates in a corporate presentation at the 41st Annual J.P. Morgan Healthcare Conference today, Monday, January 9, 2023, at 9:00 a.m. PT. The live webcast of the presentation may be accessed under Investors on the argenx website. A replay will be available for 30 days following the presentation.

On January 9, 2023 Aprea therapeutics presented its corporate presentation (Presentation, Aprea, JAN 9, 2023, View Source [SID1234626029]).

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On January 9, 2023 Alloy Therapeutics, a biotechnology ecosystem company, reported its 2023 roadmap of new discovery technologies and services across a range of biologic modalities (Press release, Alloy Therapeutics, JAN 9, 2023, View Source [SID1234626027]). Alloy will support its community of 140+ partners with deepened capabilities in its core antibodies offering, as well as substantial expansion into new modalities such as genetic medicines, leveraging an existing partnership announced with Dr. Sudhir Agrawal, a pioneer of gapmer antisense oligonucleotide (ASO) technology.

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Alloy launched in 2017 with a mission to centralize and democratize foundational technologies and expertise for biologics discovery to enable the global scientific community to advance the best, most innovative medicines more quickly. It started with the ATX-Gx transgenic humanized mouse platform for monoclonal antibody discovery and rapidly built antibody and bispecific discovery services around that foundational technology, along with novel protocols to serve collaborators with bespoke campaigns to discover the best therapeutic antibodies. Alloy reinvests 100% of its revenue into innovation and access to innovation, a commitment that enables the organization to continuously improve upon and develop new technologies to support its partners’ ambitious drug discovery goals.

In 2022, Alloy advanced into its second modality for T cell receptor-based (TCR-based) therapeutics, with the launch of Keyway TCR Discovery. Keyway is the first, and only, fully integrated discovery service provider and proprietary platform to discover therapeutic soluble proteins binding peptide-MHC complexes. Keyway is already collaborating in the discovery of highly specific TCR-mimic antibodies, and in 2023 will expand its end-to-end discovery offering to include soluble TCRs (alpha and beta chains). Keyway pairs TCR therapeutic pioneers with Alloy’s flagship bispecific antibody discovery and optimization services for the discovery of highly specific, next-generation soluble TCR-based therapeutics. Alloy’s ability to amalgamate shared capabilities such as antigen generation, bioinformatics, library design, protein engineering, antibody discovery, specialized specificity testing, and protein analytics across modalities enables it to quickly mobilize its scientists and collaborators into new areas for an expansive 2023 innovation roadmap.

"Our 2023 service and technology innovations are a reflection of years of thoughtful R&D work to give the drug discovery community access to the technologies and expertise that allow them to efficiently advance innovative therapeutics," said Alloy Therapeutics CEO and Founder Errik Anderson. "For some companies, ‘access’ to innovation is best served through working with our discovery services team in a collaborative and highly engaged process. Alloy is eager to serve our collaborators with bespoke discovery campaigns that allow them to discover the best drug against their therapeutic target, regardless of format."

2023 Innovations will span Alloy’s modalities:

Antibodies: Alloy’s foundational modality will advance new hyperimmune strains for its ATX-Gx monoclonal antibody discovery platform, available for license in partners’ labs or available as an end-to-end service as part of Alloy’s DeepImmune Antibody Discovery and Daedamab Antibody Discovery services. These groups will continue to strengthen their mission to find the best therapeutic antibodies with new capabilities such as machine learning-aided repertoire sequencing and protein optimization.
Bispecific Antibodies: Alloy will launch ATX-CLC, a new suite of transgenic common light chain mice to enhance the discovery of bispecifics, which will be integrated into bespoke discovery campaigns through DeepImmune Discovery at Alloy, as well as available for license.
Cell Therapy: Extending Alloy’s services in CAR-T generation, Alloy will launch the ATX-SiD transgenic humanized mouse for the discovery of single domain antibodies to enable development of bispecifics and chimeric antigen receptors. New discovery services supporting engineering into final CAR-T constructs as well as in vitro and in vivo functional screening will launch in 2023.

Keyway TCR Discovery: Keyway TCRm encompasses proprietary antibody-like bispecific formats for simultaneous effector cell engagement and target engagement and will launch new industry-leading specificity screening workflows to improve the safety profiles and drive the success of TCR-mimics in clinical trials.

Genetic Medicines: Alloy will launch Genetic Medicines Discovery services as its third fully integrated discovery services organization, leveraging proprietary platforms and world-class, modality-specific expertise. Through the collaboration with Dr. Agrawal, Alloy has developed proprietary technologies and oligonucleotide formats with differentiated properties compared to existing formats. Genetic Medicines will first partner with collaborators to convert existing gapmer antisense leads into these new formats for augmented activity and then launch its end-to-end de novo development of ASOs. This modality will also span immunostimulatory and immunosuppressive oligos, enabling collaborators to access unique sequences as proprietary adjuvant and autoimmunity drugs.

82VS: Alloy’s affiliated venture studio, 82VS, will be launching new companies advancing medicines discovered from the breadth of Alloy platforms and services. These startups are the first to validate and iterate on Alloy’s novel platform innovations and serve as great examples of what is possible for collaborators who would like to work with Alloy Discovery Services. In 2023, 82VS will launch new companies from stealth spanning TCR-mimics, bispecific antibodies, novel monoclonal antibodies, and genetic medicines.

"We are grateful to our partners who trust us with their biggest drug discovery challenges and provide the insights that lead to these technology advances," said Alloy Therapeutics President and Chief Scientific Officer Piotr Bobrowicz. "This type of collaboration is the reason Alloy started and will continue to drive the invention of foundational technologies to support sophisticated discovery campaigns across modalities to the benefit of the entire industry. We encourage drug discovery teams everywhere to look to Alloy as a partner to advance the technologies they need to be successful, through Alloy Research Services collaborations that can launch the discovery innovations of tomorrow."

On January 9, 2023 Alkermes presented its corporate presentation (Presentation, Alkermes, JAN 9, 2023, View Source [SID1234626026]).

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On January 9, 2023 Alentis Therapeutics ("Alentis"), the biotechnology company developing breakthrough treatments for organ fibrosis and CLDN1+ tumors, reported positive results from a single ascending dose Phase 1 study of its lead program, ALE.F02, currently in development for the treatment of advanced kidney, lung and liver fibrosis (Press release, Alentis Therapeutics, JAN 9, 2023, View Source [SID1234626025]). The study found ALE.F02 to be well tolerated in healthy volunteers at all doses with a good safety profile and demonstrated initial evidence of on-target biological activity.

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CLDN1, one of the claudin family of transmembrane proteins like Claudin-18.2, is a previously unexploited target that plays a key role in the pathology of tumors with immune evasive properties and fibrotic diseases across multiple organs. Alentis is the only company developing potential treatments for solid cancers and fibrosis targeting CLDN1.

ALE.F02 is a highly selective anti-CLDN1 mAb that recognizes pathological overexpressed and conformation-dependent CLDN1 epitopes on transformed epithelial cells and is being investigated for the treatment of fibrotic disease in the kidney, lung and liver.

The Phase 1 clinical study was initiated in January 2022 to look at the safety and tolerability of ALE.F02 in 40 individuals comprising of five dose cohorts with eight individuals in each cohort. Dosing ranged from a minimum of 0.3mg/kg to a maximum of 20 mg/kg. Pharmacokinetic results predict an optimal dose for full receptor occupancy in humans within the range. No serious or severe adverse events were recorded. A multiple ascending dose Phase 1 study is ongoing, the results of which will be reported in Q1 2023.

Dr. Roberto Iacone, CEO at Alentis Therapeutics, commented: "These important clinical data for our lead program ALE.F02 are very encouraging, have enabled us to identify an optimal dose for further Phase 1 testing, and demonstrated encouraging on-target biological activity.

"As we continue to develop breakthrough treatments for solid cancers and fibrosis targeting CLDN1, we look forward to reporting the full Phase 1 results for ALE.F02 in Q1 2023. In addition, we expect to initiate a first-in-human clinical study of our lead oncology asset ALE.C04 in cancer patients in H2 2023."

Dr. Markus Meyer, VP R&D Operations at Alentis Therapeutics, added: "There are currently limited treatment options available for patients with fibrotic associated cancers and kidney, lung and liver fibrosis. CLDN1 is a novel, previously unexplored target with a unique mechanism of action in the pathology of CLDN1+ solid tumors and fibrosis. We continue to gather further clinical data as we develop a potential new treatment option for patients in the future."