On January 9, 2023 Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, reported that it has signed a multi-year research collaboration and option agreement with ReCode Therapeutics (Press release, Bayer, JAN 9, 2023, View Source [SID1234626033]). Under this agreement, the companies will work together to potentially discover precision genetic medicines through the development of a novel platform for full gene insertion by single vector delivery of gene editing and DNA cargoes. This represents another step forward for AskBio in developing its gene editing and other nonviral delivery technologies and expertise.

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"The collaboration pairs AskBio’s expertise in synthetic DNA and CRISPR applications with ReCode’s novel SORT LNP delivery technology to potentially discover new genetic medicines," said AskBio’s Chief Executive Officer Sheila Mikhail. "Through this collaboration, we have an opportunity to advance AskBio’s gene editing and nonviral delivery efforts, which support and align with our commitment to serve as an industry-leading engine of gene therapy innovation."

Under the agreement, AskBio will combine its synthetic DNA and gene editing nucleases with ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) technology to potentially create an all-in-one solution that enables full gene insertion by delivering with precision both the gene editing tool and DNA as mixed cargo in a single LNP to desired targets. This collaboration could extend the reach of gene editing for liver and lung disease targets.

"AskBio’s pioneering gene editing technology is a powerful complement to our modular SORT LNP genetic medicines platform, which enables the precise delivery of a wide variety of genetic cargoes to specific organs and cells, including large, complex genetic cargoes and mixed payloads," said Shehnaaz Suliman, M.D., MBA, M.Phil., Chief Executive Officer of ReCode Therapeutics. "We are excited to combine these unique platforms to develop next-generation genetic therapies with the potential to transform the lives of patients suffering from a wide range of debilitating genetic diseases."

In June 2022, ReCode announced the closing of a $200 million Series B extension financing round, co-led by Leaps by Bayer, the impact investment unit of Bayer AG, to further strengthen Leaps by Bayer’s innovation portfolio in gene therapies. The funds raised are being used to advance the development of ReCode’s platform and pipeline to selectively deliver genetic medicines to target organ and cell types in a predictable and programmable fashion.

On January 9, 2023 Selecta Biosciences, Inc. (NASDAQ: SELB, President and CEO: Carsten Brunn, Ph.D., "Selecta"), and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas" or "Astellas Gene Therapies") reported an exclusive licensing and development agreement for IdeXork (Xork). Xork is being studied as a potential next generation immunoglobulin G (IgG) protease that will be developed by Astellas for use with AT845, an investigational, adeno-associated virus (AAV)-based treatment for Late-Onset Pompe disease (LOPD) in adults (Press release, Astellas, JAN 9, 2023, View Source [SID1234626032]).

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"Currently many patients are ineligible for clinical trials with investigational AAV gene therapy products due to the presence of naturally occurring antibodies against AAV gene therapy capsids," said Carsten Brunn, Ph.D., President and Chief Executive Officer of Selecta. "Xork has the potential to expand access to life-changing gene therapies by addressing pre-existing immunity to AAV. Most other IgG proteases in development are derived from common human pathogens, and as a result there is a high prevalence of pre-existing antibodies against these proteases that can restrict their use. Xork is differentiated by its low cross-reactivity to pre-existing antibodies in human serum. We are thrilled to partner with Astellas as they advance their robust gene therapy portfolio through the clinic."

Naoki Okamura, Chief Strategy Officer of Astellas added, "We are looking forward to partnering with Selecta as we strive to expand our therapies to a broader range of patients living with debilitating diseases, who have limited treatment options. This agreement provides an opportunity to deliver potentially transformative gene therapy treatments to a specific population of LOPD adult patients who might otherwise be ineligible for clinical trials or treatment with Astellas’ investigational product."

Under the terms of the agreement, Selecta will receive a $10M upfront payment and is eligible to receive up to $340M for certain additional development and commercial milestones plus royalties on any potential commercial sales where Xork is used as a pre-treatment for AT845. Selecta is responsible for the development and manufacturing of Xork and will maintain the rights for the development of additional indications beyond Pompe disease. Astellas would have the sole and exclusive right to commercialize Xork for use in Pompe disease with an Astellas gene therapy investigational or authorized product, with a current focus on AT845.

On January 9, 2023 Ascendis pharma presented its corporate presentation (Presentation, Ascendis Pharma, JAN 9, 2023, View Source [SID1234626031]).

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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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