On January 8, 2024 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported Pascal Touchon, President and Chief Executive Officer of Atara, will present the Company’s 2023 accomplishments across strategic priorities and key upcoming milestones at the 42nd Annual J.P. Morgan Healthcare Conference on Thursday, January 11 at 9:45 a.m. PST / 12:45 p.m. EST (Press release, Atara Biotherapeutics, JAN 8, 2024, View Source [SID1234639055]).

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"Our off-the-shelf, allogeneic CAR EBV T cell pipeline now spans both oncology and autoimmune indications and is designed to overcome current limitations of autologous CAR T and other allogeneic cell therapy approaches. With preliminary clinical data expected later this year for ATA3219 in lymphoma and a planned IND in Lupus Nephritis in Q1, we enter 2024 with multiple opportunities for a potential best-in-class allogeneic product," said Pascal Touchon, President and Chief Executive Officer of Atara. "Meanwhile, we are encouraged by our latest pivotal study data for tab-cel supporting our plan to file a BLA in Q2 2024, while our global commercial partner Pierre Fabre is starting to prepare the U.S. launch."

Tabelecleucel (tab-cel or EBVALLOTM) for Post-Transplant Lymphoproliferative Disease (PTLD)

Atara is advancing toward filing a Biologics License Application (BLA) in Q2 2024, which will include the latest pivotal ALLELE study data-cut that demonstrated a statistically significant 49% Objective Response Rate (ORR) (p<0.0001) and favorable safety profile consistent with previous analyses
This new data set augments the extensive database of pivotal and supportive data as part of the upcoming BLA filing package, collectively consisting of approximately 450 patients treated with tab-cel across multiple life-threatening diseases
The expanded global partnership with Pierre Fabre Laboratories for the U.S. and remaining global commercial markets for tab-cel closed on December 20, 2023
Under the agreement, Atara received approximately USD 27 million in cash upfront at the closing of the deal, with the potential to receive up to a total of USD 640 million in milestone payments, development funding, and significant double-digit tiered royalties on net sales
Tab-cel for Potential Indication Expansion

Positive new clinical data from a combined analysis, including the first reported data from the multicohort Phase 2 EBVision trial, were presented during an oral session at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Annual Congress
In the pooled analysis, an ORR of 77.8% was observed in 18 central nervous system (CNS) EBV+ PTLD patients including 1 CNS EBV+ PTLD patient with no prior treatment, who achieved a complete response
One- and two-year overall survival rates were higher in responders (85.7% and 66.7%, respectively) versus non-responders (0% and 0%, respectively)
Tab-cel was well tolerated, with no reports of serious treatment-related fatal or life-threatening treatment-emergent adverse events (TEAEs), and no reports of serious treatment-related TEAEs of neurotoxicity, organ rejection, graft versus host disease, or tumor flare reaction of any grade
Enrollment is continuing at sites in the potential label expansion multi-cohort Phase 2 EBVision trial evaluating new patient populations, including 1L EBV+ PTLD and EBV+ immunodeficiency-associated lymphoproliferative diseases (IA-LPDs)
CAR-T Programs (Hematological Malignancies and Autoimmune Conditions)

ATA3219

Atara is progressing development of ATA3219, an allogeneic, off-the-shelf CAR T targeting CD19, optimized for a memory phenotype and incorporating a next generation 1XX signaling domain
Pre-clinical data support a potential best-in-class profile with longer persistence and superior anti-tumor efficacy compared to an autologous CD19 CAR T benchmark
Site selection and activation is ongoing for the Phase 1 study in relapsed/refractory B-cell non-Hodgkin’s lymphoma (NHL) and progressing toward enrolling the first patient in Q1 2024
Preliminary clinical data in lymphoma anticipated H2 2024
Planned Q1 2024 IND submission in Lupus Nephritis following compelling clinical results from autologous CD19 CAR T academic clinical study showing 8/8 patients attaining remission1
Atara’s EBV CAR T cells may offer a differentiated therapeutic approach—off-the-shelf accessibility, no requirement for gene editing, and a less differentiated phenotype driving cellular fitness—with the potential for rapid and deep B-cell depletion
ATA3219 autoimmune development is building upon the favorable safety profile of Atara’s allogeneic EBV T cells in autoimmune disease
ATA3431

Positive preclinical data presented at ASH (Free ASH Whitepaper) for ATA3431, an allogeneic, dual-targeted CAR directed against CD20 and CD19 to mitigate CD19 antigen escape, built on Atara’s EBV T-cell platform with novel 1XX stimulation for enhanced persistence
Data showed superior in vivo anti-tumor activity, survival, and functional persistence of ATA3431 compared to an autologous CD20- CD19 CAR-T benchmark
Atara is advancing ATA3431 into IND-enabling studies
Strategic Restructure and Financial Impact

Atara is undertaking a strategic restructuring and reducing its current workforce of 225 by approximately 25% reflecting its evolving corporate strategy and pipeline focus to progress its potential best-in-class allogeneic CAR-T portfolio for cancer and autoimmune diseases
Atara will focus on executing its remaining responsibilities under the tab-cel collaboration with Pierre Fabre Laboratories, including filing the BLA in Q2 2024, and advancing its differentiated allogeneic CAR-T (AlloCAR-T) ATA3219 and ATA3431 programs to key milestones in 2024
The strategic restructuring, combined with anticipated payments upon successful filing and approval of tab-cel BLA from our expanded global partnership, and the Company’s existing cash, cash equivalents and short-term investments as of September 30, 2023, is expected to fund the Company’s planned operations into 2027
A live audio webcast of the presentation will be available by visiting the Investors & Media – News & Events section of atarabio.com on Thursday, January 11, at 9:45 a.m. PST / 12:45 p.m. EST. An archived replay of the webcast will be available on the Company’s website for 30 days following the live presentation. A new corporate presentation will be available on Monday, January 8 at 8:00 a.m. EST / 5:00 a.m. PST.

Next-Generation Allogeneic CAR-T Approach

Atara is focused on applying Epstein-Barr virus (EBV) T-cell biology, featuring experience in over 500 patients treated, and novel chimeric antigen receptor (CAR) technologies to meet the current limitations of autologous and allogeneic CAR therapies head-on by advancing a potential best-in-class CAR-T pipeline in oncology and autoimmune disease. Unlike gene-edited approaches aimed at inactivating T-cell receptor (TCR) function to reduce the risk for graft-vs-host disease, EBV T cells maintain expression of native TCRs that promote in vivo functional persistence while also demonstrating inherently low alloreactivity due to their recognition of defined viral antigens and partial human leukocyte antigen (HLA) matching. A molecular toolkit of clinically-validated technologies—including the 1XX costimulatory domain designed for better cell fitness and less exhaustion while maintaining stemness— offers a differentiated approach to addressing significant unmet need with the next generation CAR T.

On January 8, 2024 Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") reported the U.S. Food and Drug Administration (FDA) issued a complete response letter on January 4, 2024, regarding the Biologics License Application (BLA) for zolbetuximab, an investigational agent for the treatment of patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors are claudin (CLDN) 18.2 positive (Press release, Astellas, JAN 8, 2024, View Source [SID1234639054]).

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The FDA stated that the agency cannot approve the BLA by the Prescription Drug User Fee Act (PDUFA) action date of January 12, 2024, due to unresolved deficiencies following its pre-license inspection of a third-party manufacturing facility for zolbetuximab. The FDA has not raised any concerns related to the clinical data, including efficacy or safety, of zolbetuximab, and is not requesting additional clinical studies. Astellas is working closely with the FDA and the third-party manufacturer to establish a timeline to quickly resolve the agency’s feedback. No other Astellas products are affected.

Moitreyee Chatterjee-Kishore, Ph.D., M.B.A., Senior Vice President and Head of Immuno-Oncology Development, Astellas
"We remain confident in zolbetuximab’s clinical profile and potential to fill a significant therapeutic gap for those diagnosed with advanced gastric or GEJ cancer whose tumors are CLDN18.2 positive. Astellas is committed to working with the FDA and the third-party manufacturer to address the agency’s feedback, and to bringing zolbetuximab to U.S. patients in need, as soon as possible."

Regulatory applications for zolbetuximab are also under review in several other countries and regions, including Japan, Europe, and China.

The impact of this matter on Astellas’ financial results in the fiscal year ending March 31, 2024, will be limited.

For more information, please see the press release "Astellas Announces U.S. FDA Grants Priority Review for Zolbetuximab Biologics License Application" issued on July 6, 2023.

About Zolbetuximab
Zolbetuximab is an investigational, first-in-class chimeric IgG1 monoclonal antibody (mAb) that targets and binds to claudin 18.2 (CLDN18.2), a transmembrane protein. Zolbetuximab acts by binding to CLDN18.2 on the cancer cell surface of gastric epithelial cells. In pre-clinical studies, this binding interaction then induces cancer cell death by activating two distinct immune system pathways — antibody-dependent cellular cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC).1 Zolbetuximab has not been approved by any regulatory bodies for the treatment of patients with gastric and GEJ cancers, and there is no guarantee the agent will receive regulatory approval or become commercially available for the uses being investigated.

On January 8, 2024 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported preliminary financial results for the full-year 2023, including global net product sales (inclusive of both VYVGART and VYVGART Hytrulo), and announced its strategic priorities for 2024 (Press release, argenx, JAN 8, 2024, View Source [SID1234639053]).

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"In 2023, we reached more than 6,000 patients globally, making VYVGART available to gMG patients around the world," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "We continued to invest in and demonstrate the sustainability of our business by successfully launching our subcutaneous VYVGART product, and are poised for continued expansion in gMG and beyond in 2024. argenx is delivering on its promise to transform how the world understands autoimmunity. It is with this commitment in mind that we submitted our sBLA for VYVGART Hytrulo in CIDP and, if approved, expect to launch in the U.S. in mid-2024. We will continue to be aggressive in advancing our pipeline this year and expect to report efgartigimod data from six Phase 2 studies in 2024, and to further develop empasiprubart in MMN. Through sustained investment in our IIP, we expect to see repeat value creation, and plan to submit four new INDs by the end of 2025."

"Two years ago, argenx’s key goal was to launch efgartigimod in the U.S. Today, we have built a formidable global commercial organization with product approvals in over 30 countries worldwide and a vibrant pipeline of promising new therapeutics to address immune-mediated diseases. We have forged important partnerships that support our mission to advance the human understanding of immunology to best benefit patients. argenx is well positioned for sustained growth throughout 2024 and well into the future."

2024 Strategic Priorities

argenx will focus on three strategic priorities in 2024 to drive sustainable long-term growth, including:

· Reach more patients with VYVGART by building upon its strong commercial foundation to address ongoing unmet patient need, broaden the MG opportunity, and expand into CIDP
· Advance its extensive pipeline through new data readouts, creating multiple opportunities to demonstrate transformative clinical benefit
· Leverage its repeatable innovation engine, driving pipeline growth through its Immunology Innovation Program

Reach More Patients with VYVGART

VYVGART (efgartigimod alfa fcab) is a first-in-class antibody fragment targeting the neonatal Fc receptor (FcRn) and is now approved in more than 30 countries globally. VYVGART subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) is approved in the U.S. (as VYVGART Hytrulo) and Europe, making VYVGART the only gMG treatment available as both an IV and simple SC injection. argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts. argenx will work to reach patients earlier in the MG treatment paradigm and improve the lives of new MG patient populations through additional global regulatory approvals, and the expansion of uses to treat additional autoimmune indications.

· Regulatory approval decisions of VYVGART for gMG expected in Switzerland, Australia, Saudi Arabia and South Korea by end of 2024
· Through strategic collaboration with Zai Lab, VYVGART to be included on China’s 2023 National Reimbursement Drug List (NRDL), starting in January 2024
· Decision on approval of VYVGART SC for gMG in Japan expected in first quarter of 2024 and in China through Zai Lab by end of 2024
· Decision on approval of VYVGART for primary immune thrombocytopenia (ITP) in Japan expected in first quarter of 2024
· Supplemental Biologics License Application (sBLA) submitted to FDA for VYVGART Hytrulo for CIDP with priority review voucher (PRV); if approved, launch expected mid-2024
· Regulatory submissions of VYVGART SC for CIDP in Japan, Europe, China and Canada expected in 2024
· Registrational studies to expand VYVGART label into broader MG populations, including in seronegative patients, to start in 2024
· Update on pre-filled syringe development expected in first half of 2024; ongoing studies to support potential approval in gMG and CIDP in 2024

Advance Current Pipeline through Upcoming Data Readouts

argenx continues to demonstrate breadth and depth within its immunology pipeline and is advancing multiple pipeline-in-a-product candidates. With efgartigimod, argenx is solidifying its leadership in FcRn and is on track to be approved or in development in 15 autoimmune indications by 2025. Beyond efgartigimod, argenx is advancing its earlier stage pipeline programs, including empasiprubart (C2 inhibitor) with Phase 2 studies ongoing in multifocal motor neuropathy (MMN), delayed graft function and dermatomyositis (DM). In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024.

Today, argenx reported positive clinical data from the first cohort of the Phase 2 ARDA study of empasiprubart, establishing proof-of-concept in MMN. After confirming IVIg dependence, 27 patients were withdrawn from IVIg treatment and randomized 2:1 to either empasiprubart or placebo for 16 weeks. Patients were monitored for clinical deterioration that required IVIg retreatment, which was the main efficacy endpoint of the study.

· Empasiprubart demonstrated a 91% reduction in the need for IVIg rescue compared to placebo [HR: 0.09 95% CI (0.02; 0.044)]

· According to the Patient Global Impression of Change scale, 94% (17/18) of empasiprubart-treated patients rated their condition as improved since study start, including 55% (10/18) who were much or very much improved. Of placebo patients, 89% (8/9) worsened or had no change.

· Empasiprubart demonstrated improvement on all six efficacy measurements compared to baseline

· Safety and tolerability profile were consistent with Phase 1 results

· Cohort 2 is ongoing to determine dose response ahead of a Phase 3 study start

argenx is on track to report topline data from five additional proof-of-concept studies in 2024, including:

· Phase 2 RHO study evaluating efgartigimod in primary Sjogren’s syndrome expected in first half of 2024

· Phase 2 ALPHA study evaluating efgartigimod in post-COVID-19 postural orthostatic tachycardia syndrome (PC-POTS) expected in first half of 2024

· Seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM), antisynthetase syndrome (ASyS), and DM) expected in the second half of 2024

Leverage Repeatable Innovation Playbook to Drive Long-Term Pipeline Growth

argenx continues to invest in its discovery engine, the Immunology Innovation Program (IIP), to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX-213 targeting FcRn and further solidifying argenx’s leadership in this new class of medicine; ARGX-121 and ARGX-220, which are first-in-class targets broadening argenx’s focus across the immune system; and ARGX-109, targeting IL-6, which plays an important role in inflammation. Preclinical work is ongoing in each candidate and argenx is on track to file four investigational new drug (IND) applications by the end of 2025.

Preliminary* Fourth Quarter and Full-Year 2023 Financial Results

Today, argenx also announced preliminary* global net VYVGART revenues for the fourth quarter and full-year 2023 of approximately $374 million and $1.2 billion, respectively.

As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*. Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion. The projected 2024 cash burn will be 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 February 2024.

42nd Annual J.P. Morgan Healthcare Conference Presentation and Webcast

Mr. Van Hauwermeiren will highlight these updates in a corporate presentation at the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, 2024, 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.

Phase 2 ARDA Study Design

The Phase 2 ARDA study is a randomized, double-blinded, placebo-controlled multicenter study to evaluate the safety and tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of two dose regimens of empasiprubart in adults with multifocal motor neuropathy (MMN). The study consists of an IVIg dependency and monitoring period and two 16-week treatment cohorts of 24 MMN patients receiving empasiprubart or placebo in a 2×1 randomization. The dosing for Cohort 2 was established after a planned interim analysis of the first nine patients to complete the 16-week treatment period from Cohort 1. The primary endpoint is safety and tolerability. Additional endpoints include time to IVIg retreatment, biomarker analyses of C2 levels, and changes in measurements on key functional scores (modified medical research council (mMRC)-10 sum score, grip strength, MMN-RODS) as well as several patient-reported quality of life outcome measures (fatigue severity score (FSS), chronic acquired polyneuropathy patient-reported index (CAP-PRI), and values of the patient global impression change (PGIC) scale).

About Multifocal Motor Neuropathy

Multifocal motor neuropathy (MMN) is a rare, chronic autoimmune disease of the peripheral nervous system. The disease is characterized by slowly progressive, asymmetric muscle weakness mainly of the hands, forearms and lower legs. MMN is often associated with anti-GM1 IgM autoimmunity, leading to activation of the classical complement pathway, driving subsequent axon damage. High-dose IVIg is the only approved treatment for MMN and patients typically experience disease progression despite therapy, indicating an unmet need for efficacious and better tolerated therapeutic options.

About Empasiprubart

Empasiprubart (ARGX-117) is a first-in-class humanized sweeping antibody that binds specifically to C2 thereby blocking both the classical and lectin pathways of the complement cascade. By blocking upstream complement activity, empasiprubart has the potential to reduce tissue inflammation representing a broad pipeline opportunity across multiple severe autoimmune indications. In addition to multifocal motor neuropathy, argenx is evaluating empasiprubart in delayed graft function following kidney transplant and dermatomyositis.

About VYVGART and VYVGART SC

VYVGART is a human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG autoantibodies. It is the first approved FcRn blocker globally for the treatment of adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive and in Japan for the treatment of adults with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs).

VYVGART SC is a subcutaneous combination of efgartigimod alfa and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE drug delivery technology to facilitate subcutaneous injection delivery of biologics. It is marketed as VYVGART Hytrulo in the U.S. and VYVGART SC in Europe, and may be marketed under different proprietary names following approval in other regions.

On January 8, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported its anticipated 2024 milestones and value-driving catalysts through 2026 that support the company’s mission to transform patient outcomes in rare diseases (Press release, Agios Pharmaceuticals, JAN 8, 2024, View Source [SID1234639052]). Agios will present at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10, 2024, at 7:30 a.m. PT, and a live webcast will be available at investor.agios.com.

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"We were pleased to announce positive topline data from the Phase 3 study of our lead PK activator, mitapivat, in non-transfusion-dependent alpha- or beta-thalassemia last week, a segment of the population with no currently approved therapeutic options in the U.S. We look forward to data readouts from four additional Phase 3 studies across our industry-leading PK activator franchise by the end of 2025," said Brian Goff, chief executive officer at Agios. "This robust series of near-term catalysts positions Agios for potential launches of a first- and best-in-class therapy in thalassemia in 2025 and in sickle cell disease in 2026, and we look forward to maximizing the commercial opportunities ahead of us. Supported by our strong cash position, Agios is poised for significant progress in the next 12-24 months, and we look forward to the opportunity to deliver a novel oral treatment option for two additional hematologic diseases with high unmet need."

2023 Highlights

Thalassemia: Completed enrollment in the Phase 3 ENERGIZE and ENERGIZE-T studies of mitapivat in non-transfusion-dependent and transfusion-dependent thalassemia, respectively
Sickle Cell Disease: Announced positive data from the Phase 2 portion of the RISE UP study of mitapivat and dosed the first patients in the Phase 3 portion
Pediatric PK Deficiency: Completed enrollment in the Phase 3 ACTIVATE kids-T study of mitapivat in children with PK deficiency who are regularly transfused. Enrolled more than half of patients in the Phase 3 ACTIVATE-kids study of mitapivat in children with pediatric PK deficiency who are not regularly transfused
Lower-risk Myelodysplastic Syndromes (LR-MDS): Announced clinical proof-of-concept in Phase 2a study of AG-946, supporting continued development in Phase 2b
Earlier-stage Pipeline: Filed an Investigational New Drug Application (IND) for PAH stabilizer for the treatment of phenylketonuria (PKU)
Business Development: Announced exclusive worldwide license agreement with Alnylam for novel siRNA targeting TMPRSS6 for the potential treatment of polycythemia vera (PV)
Data Presentations: Presented broad set of clinical and translational data at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, including positive data from the Phase 2 portion of the RISE UP study of mitapivat in sickle cell disease
Anticipated 2024 Milestones

Thalassemia: Following the announcement of topline data from the Phase 3 ENERGIZE study last week, Agios plans to report topline data from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year) and submit a New Drug Application (NDA) for mitapivat in thalassemia (year-end)
Sickle Cell Disease: Complete enrollment in the Phase 3 portion of the RISE UP study of mitapivat (year-end)
Pediatric PK Deficiency: Complete enrollment in the Phase 3 ACTIVATE-kids study of mitapivat (mid-2024). Report topline data from Phase 3 ACTIVATE kids-T study (year-end)
Lower-risk Myelodysplastic Syndromes (LR-MDS): Dose first patient in Phase 2b study of AG-946 (mid-year)
Earlier-stage Pipeline: Dose the first patient in the Phase 1 study of PAH stabilizer for the treatment of PKU (H1 2024)
Four Additional Phase 3 Readouts and Two Potential New Indication Approvals Expected by End of 2026

2024

Data readout from Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia (announced January 3, 2024)
Data readout from Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year)
Data readout from Phase 3 ACTIVATE kids-T study of mitapivat in pediatric PK deficiency (year-end)
2025

Data readout from Phase 3 portion of the RISE UP study of mitapivat in sickle cell disease
Data readout from Phase 3 ACTIVATE kids study of mitapivat in pediatric PK deficiency
Potential FDA approval for mitapivat in thalassemia
2026

Potential FDA approval for mitapivat in sickle cell disease
Potential FDA approval for mitapivat in pediatric PK deficiency
Presentation at 42nd Annual J.P. Morgan Healthcare Conference

Agios will webcast its corporate presentation from the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10 at 7:30 a.m. PT. A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On January 8, 2024 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P (Press release, Abeona Therapeutics, JAN 8, 2024, View Source [SID1234639051]). The credit agreement, which has a term of three and a half years, includes a first tranche of $20 million at closing, a second tranche of $10 million of committed capital, and an additional accordion option to upsize the credit facility by an additional $20 million upon satisfaction of certain terms and conditions.

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Proceeds from the facility are intended to support the Company’s ongoing preparations for launch and commercialization in anticipation of a potential approval for marketing in the U.S. by the Food and Drug Administration (FDA) of pz-cel (prademagene zamikeracel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB), and for general corporate purposes. The FDA has accepted and granted Priority Review with a PDUFA target action date of May 25, 2024 for the Biologics License Application for pz-cel.

"We are excited to enter into this relationship with Avenue Venture Fund and secure additional financial resources to further support our launch and commercialization efforts for pz-cel," said Joe Vazzano, Chief Financial Officer of Abeona. "We expect the proceeds from this term loan facility will meaningfully extend Abeona’s cash runway even further beyond value creating milestones, including the potential approval of pz-cel."

A.G.P./Alliance Global Partners served as financial advisor and sole lead arranger to the Company on this transaction.

About Avenue Venture Opportunities
The Avenue Venture Debt Fund seeks to provide creative financing solutions to high-growth, venture capital-backed technology and life science companies. The Avenue Venture Debt Opportunities Fund focuses generally on companies within the underserved segment of the market created by the widening financing gap between commercial banks and larger debt funds. The Avenue Venture Debt fund is part of the larger group of funds of Avenue Capital Group. For additional information on Avenue Capital Group, which is a global investment firm with assets estimated to be approximately $12.6 billion as of November 30, 2023, visit www.avenuecapital.com.