On January 13, 2025 BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a new type of biopharmaceutical company focused on genetic diseases, reported updates on its commercial progress for Attruby (acoramidis), status of late-stage pipeline programs, and anticipated 2025 milestones (Press release, BridgeBio, JAN 13, 2025, View Source [SID1234649722]).

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"With the FDA’s approval of Attruby, we marked an important moment for both our organization and the broader ATTR-CM patient community in need of new treatment options. We’re grateful for the enthusiasm surrounding the product and the associated initial commercial momentum, with 430 prescriptions written by 248 unique physicians, and we look forward to continued progress," said Neil Kumar, Ph.D., Founder and CEO of BridgeBio. "Additionally, we are excited to share that we have completed enrollment of all three of our major market Phase 3 clinical trials. I look forward to continuing to work with this stellar team to serve patients with genetic disease in 2025."

Business Update
On November 22, 2024, the U.S. Food and Drug Administration (FDA) approved Attruby (acoramidis), a near-complete TTR stabilizer (≥90%), to reduce cardiovascular death and cardiovascular-related hospitalization in adult patients with ATTR-CM, a progressive fatal disease presenting as an infiltrative, restrictive cardiomyopathy resulting in heart failure.

Since the approval, BridgeBio has seen remarkable momentum with 430 patient prescriptions written by 248 physicians.

Pipeline Updates

BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):

FORTIFY is a Phase 3 clinical trial of BBP-418 in LGMD2I/R9, a rare genetic disorder caused by variants in the fukutin‑related protein (FKRP) gene that result in progressive muscle degeneration and damage, and eventual loss of functional independence. The trial is fully enrolled with 112 patients.
The Company expects Last Patient – Last Visit (LPLV) and topline readout of the interim analysis cohort in second half 2025.
If successful, BBP-418 would be the first approved therapy for individuals living with LGMD2I/R9.

Encaleret – Calcium-sensing receptor (CaSR) antagonist for autosomal dominant hypocalcemia type 1 (ADH1):

CALIBRATE, the Phase 3 clinical trial of encaleret in ADH1, a rare, genetic form of hypoparathyroidism, is fully enrolled with 70 patients. The trial is designed to evaluate the efficacy and safety of encaleret compared to standard of care in adult patients with ADH1.
The Company expects Last Patient – Last Visit and topline readout in second half 2025.
If successful, encaleret would be the first approved therapy for individuals living with ADH1.

Infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:

PROPEL 3, the Phase 3 clinical trial of infigratinib in achondroplasia, the most common form of disproportionate short stature, is fully enrolled with 114 participants.
The Company expects Last Participant – Last Visit in second half 2025.
If successful, infigratinib would be the first approved oral therapy for children living with achondroplasia.

2025 Milestones

Program Status Anticipated 2025 Milestone
Acoramidis for ATTR-CM US FDA approval on November 22, 2024 EU and Japan approvals in 1H 2025
BBP-418 for LGMD2I/R9 FORTIFY, Phase 3 study enrollment completed Last Patient – Last Visit and Topline readout in 2H 2025
Encaleret for ADH1 CALIBRATE, Phase 3 study enrollment completed Last Patient – Last Visit and Topline readout in 2H 2025
Infigratinib for achondroplasia PROPEL 3, Phase 3 study enrollment completed Last Participant – Last Visit in 2H 2025

About Attruby (acoramidis)

INDICATION
Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization.

On January 13, 2025 Daiichi Sankyo reported AstraZeneca’s Biologics License Application (BLA) for datopotamab deruxtecan (Dato-DXd) has been accepted and granted Priority Review in the U.S. for the treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFR-mutated) non-small cell lung cancer (NSCLC) who have received prior systemic therapies, including an EGFR-directed therapy (Press release, Daiichi Sankyo, JAN 13, 2025, View Source [SID1234649721]).

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Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed by Daiichi Sankyo and AstraZeneca.

The U.S. Food and Drug Administration (FDA) grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance. The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for its regulatory decision, is July 12, 2025. Datopotamab deruxtecan was previously granted Breakthrough Therapy Designation (BTD) by the FDA for this patient population.

The BLA and BTD are based on data from the TROPION-Lung05 phase 2 trial and supported by data from the TROPION-Lung01 phase 3 trial. In addition, the BLA is supported by the TROPION-PanTumor01 phase 1 trial. In a pooled analysis of patients with previously treated advanced or metastatic EGFR-mutated NSCLC in the TROPION-Lung05 and TROPION-Lung01 trials presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Asia 2024 Congress, datopotamab deruxtecan demonstrated clinically meaningful tumor response. The safety profile of datopotamab deruxtecan was consistent with previous reports from the TROPION-Lung05 and TROPION-Lung01 trials, with no new safety concerns identified.

"Treating advanced EGFR-mutated non-small cell lung cancer presents a significant challenge due to the limited efficacy of available treatments once the disease has progressed following front-line therapies, including the use of an EGFR tyrosine kinase inhibitor," said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. "If approved, datopotamab deruxtecan could become the first TROP2 directed antibody drug conjugate for lung cancer, providing a promising option for patients."

"Acquired resistance to front-line therapies and, ultimately, disease progression are unfortunate realities for most patients with advanced EGFR-mutated non-small cell lung cancer," said Susan Galbraith, MBBChir, PhD, Executive Vice President, Oncology R&D, AstraZeneca. "This Priority Review, and the previously granted Breakthrough Therapy Designation, recognize the potential for datopotamab deruxtecan to provide a much-needed option to patients whose disease has become resistant to current treatments."

Daiichi Sankyo and AstraZeneca are evaluating datopotamab deruxtecan alone and in novel combinations as treatment for patients with NSCLC in seven phase 3 trials including the TROPION-Lung14 and TROPIONLung15 trials of datopotamab deruxtecan alone and with osimertinib, AstraZeneca’s EGFR tyrosine kinase inhibitor (TKI), as treatment for patients with advanced or metastatic EGFR-mutated nonsquamous NSCLC.

About TROPION-Lung05

TROPION-Lung05 is a global, multicenter, single-arm, open-label phase 2 trial evaluating the efficacy and safety of datopotamab deruxtecan in patients with locally advanced or metastatic NSCLC with actionable genomic alterations who have progressed on at least one TKI (with or without other systemic therapies) and on or after one regimen of platinum-based chemotherapy. Patients receiving up to four prior lines of treatment with tumors with one or more genomic alterations including EGFR, ALK, ROS1, NTRK, BRAF, RET or MET were eligible for the trial.

The primary trial endpoint of TROPION-Lung05 is objective response rate (ORR) as assessed by blinded independent central review (BICR). Secondary efficacy endpoints include duration of response (DoR), disease control rate (DCR), clinical benefit rate, progression-free survival (PFS), time to response (TTR), overall survival (OS) and safety

TROPION-Lung05 enrolled 137 patients globally in Asia, Europe and North America. For more information visit ClinicalTrials.gov

About TROPION-Lung01

TROPION-Lung01 is a global, randomized, multicenter, open-label phase 3 trial evaluating the efficacy and safety of datopotamab deruxtecan versus docetaxel in adult patients with locally advanced or metastatic NSCLC with and without actionable genomic alterations who require systemic therapy following prior treatment. Patients with actionable genomic alterations were previously treated with an approved targeted therapy and platinum-based chemotherapy. Patients without known actionable genomic alterations were previously treated, concurrently or sequentially, with platinum-based chemotherapy and a PD-1 or PD-L1 inhibitor

The dual primary endpoints of TROPION-Lung01 are PFS as assessed by BICR and OS. Key secondary endpoints include investigator-assessed PFS, ORR, DoR, TTR, and DCR as assessed by both BICR and investigator, and safety.

TROPION-Lung01 enrolled approximately 600 patients in Asia, Europe, North America, Oceania and
South America. For more information visit ClinicalTrials.gov.

About TROPION-PanTumor01

TROPION-PanTumor01 is a first-in-human, open-label, two-part, multicenter phase 1 trial evaluating the safety and preliminary efficacy of datopotamab deruxtecan in patients with advanced solid tumors that have relapsed or are refractory to standard treatment or for which no standard treatment is available. The dose escalation portion of the trial enrolled patients with NSCLC to assess the safety and tolerability of datopotamab deruxtecan to determine the recommended dose for expansion (6 mg/kg). The dose expansion part of TROPION-PanTumor01 enrolled several different cohorts including patients with NSCLC, triple negative breast cancer (TNBC), HR positive, HER2 low or negative breast cancer, small cell lung cancer, urothelial, gastric, pancreatic, castration resistant prostate and esophageal cancer. Safety endpoints include dose-limiting toxicities and serious adverse events. Efficacy endpoints include ORR, DoR, TTR, PFS and OS. Pharmacokinetic, biomarker and immunogenicity endpoints also are being evaluated. TROPION-PanTumor01 enrolled approximately 900 patients in Asia and North America. For more information visit ClinicalTrials.gov.

About Advanced Non-Small Cell Lung Cancer
Nearly 2.5 million lung cancer cases were diagnosed globally in 2022.1 Lung cancer is broadly split into small or non-small cell lung cancer, the latter accounting for about 80% of cases.2 Approximately 10% to 15% of patients with NSCLC in the U.S. and Europe, and 30% to 40% of patients in Asia have an EGFR mutation.3,4 The majority of EGFR mutations occur in tumors of nonsquamous histology.5 For patients with tumors that have an EGFR mutation, the established first-line treatment in the metastatic setting is an EGFR TKI.6 While EGFR TKIs have improved outcomes in the first-line setting, most patients eventually experience disease progression and receive subsequent therapies, such as chemotherapy. 7,8,9,10 TROP2 is a protein broadly expressed in the majority of NSCLC tumors.11 There is currently no TROP2 directed ADC approved for the treatment of lung cancer.6,12

About Datopotamab Deruxtecan (Dato-DXd)

Datopotamab deruxtecan (Dato-DXd) is an investigational TROP2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, datopotamab deruxtecan is one of six DXd ADCs in the oncology pipeline of Daiichi Sankyo, and one of the most advanced programs in AstraZeneca’s ADC scientific platform. Datopotamab deruxtecan is comprised of a humanized anti-TROP2 IgG1 monoclonal antibody, developed in collaboration with Sapporo Medical University, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. Datopotamab deruxtecan is approved in Japan under the brand name DATROWAY for the treatment of adult patients with HR positive, HER2 negative (IHC 0, IHC 1+ or IHC 2+/ISH-) unresectable or recurrent breast cancer after prior chemotherapy based on the results of the TROPION-Breast01 trial. Datopotamab deruxtecan is an investigational medicine in all countries outside of Japan.

On January 13, 2025 Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine the treatment of solid tumor cancers with cell therapy, reported that letetresgene autoleucel (lete-cel), has been granted breakthrough therapy designation by the U.S. FDA for the treatment of patients with unresectable or metastatic myxoid/round cell liposarcoma (MRCLS) who have received prior anthracycline-based chemotherapy, are positive for HLA-A*02:01, HLA-A*02:05, or HLA-A*02:06, and whose tumor expresses the NY-ESO-1 antigen (Press release, Adaptimmune, JAN 13, 2025, View Source [SID1234649720]).

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More details about the Company’s sarcoma franchise, including the lete-cel clinical program and launch progress for TECELRA (afamitresgene autoleucel), the Company’s first commercial product and the first FDA approved engineered cell therapy for a solid tumor, will be provided during the Company’s presentation at the Annual J.P. Morgan Healthcare Conference, taking place in San Francisco, California, on Tuesday, January 14th, 2025, 4:30-5:10 PM PST (Webcast access here).

Breakthrough therapy designation for lete-cel in MRCLS was based on the results in this indication from the Phase II IGNYTE-ESO trial. The Company previously received breakthrough therapy designation for lete-cel for the treatment of synovial sarcoma in 2016. In the Phase II analysis, 27/64 (42%) people with synovial sarcoma or MRCLS had RECISTv1.1 responses by independent review, with 6 complete responses and 21 partial responses. The response rate was 14/34 (41%) for people with synovial sarcoma and 13/30 (43%) for people with MRCLS. The median duration of response (DoR) was 12.2 months (95% CI 6.8, 19.5). In synovial sarcoma, the median duration of response was 18.3 months (95% CI 3.3, -). In MRCLS, the median duration of response was 12.2 months (95%, CI 5.3, -). Safety findings were consistent with the known profile of lete-cel from previous data. All patients experienced treatment-emergent adverse events: cytopenias, cytokine release syndrome (CRS) and rash were the most common adverse events. Overall, toxicities were manageable, and consistent with an acceptable benefit to risk profile. Data from this trial were presented at the Connective Tissue Oncology Society (CTOS) 2024 annual meeting (Link to press release HERE; Presentation HERE).

Adrian Rawcliffe, Adaptimmune’s Chief Executive Office: "This designation by the FDA highlights the potential of lete-cel to address a critical need for new treatment options for patients with MRCLS. This is another important milestone in building out our sarcoma franchise, as we aim to bring lete-cel to market in 2026 for the treatment of synovial sarcoma and MRCLS. We look forward to initiating a rolling Biologics License Application for lete-cel later this year for the treatment of both sarcoma indications."

The breakthrough therapy designation is designed to expedite drug development and review processes. The criteria for this designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. With the designation, lete-cel will receive incentives, such as additional interaction and guidance from the FDA, the potential for a rolling submission, and potential priority review of the biologics license application, as well as other opportunities to expedite the development.

In addition to the Company’s presentation at J.P. Morgan, Adaptimmune will present the Company’s Allo-T program as a spin-out opportunity at the Biotech ShowCase and the Wuxi Global Forum 2025 Investor Roundtable. Details can be found below:

Biotech Showcase | Investor conference | Co-produced by Demy-Colton and EBD Group
Presentation at 2 p.m. PST on Tuesday, January 14th, Yosemite C, Hilton Hotel, Union Square.
WuXi Global Forum 2025 – WXPress: for WuXi news and R&D insights
Round Table Presentation at 5 p.m. PST on Tuesday, January 14th, Tower 3, Hilton Hotel, Union Square

On Jan. 13, 2025 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported highlights for the upcoming company presentation at the 43rd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2025 at 9:00 AM PT/12:00 PM ET (Press release, Precigen, JAN 13, 2025, View Source [SID1234649719]). Participants may view details for Precigen’s company presentation at Precigen’s website in the Events & Presentations section at investors.precigen.com/events-presentations.

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"Our recent BLA submission for PRGN-2012 in RRP puts our commercial readiness activities in high gear and we are well underway to ensure full readiness in anticipation of a potential launch in the second half of this year. We have made tremendous progress in the past six months on payer and prescriber analysis, clarifying the market opportunity, building out our commercial and distribution infrastructure, and other activities to ensure we fully coordinate the payor, provider, and patient journeys for launch," said Phil Tennant, Chief Commercial Officer of Precigen. "Our updated analysis of the market using claims data and electronic health records underscores the significant potential for PRGN-2012 in RRP with approximately 27,000 adult patients now identified in the US and we expect more than 125,000 patients ex-US. We are confident that we will be ready to commercialize in the US as soon as we receive approval so that patients can gain access to this game-changing treatment."

"With 100% manufacturing success rate demonstrated to date, we anticipate our in-house commercial drug substance cGMP manufacturing facility will be ready to meet the projected demand to support potential commercial launch in the second half of this year," said Rutul Shah, Chief Operating Officer of Precigen.

"We continue to de-risk our PRGN-2012 asset and now that we have submitted the BLA in RRP, we are a step closer to completing our transition to a commercial-stage company. As we start 2025, we are laser focused on working with the FDA and advancing commercial readiness efforts in anticipation of a potential launch in the second half of this year. In addition, we are preparing for submissions to other health authorities in our prioritized global markets. Beyond RRP, we see immense potential for the AdenoVerse platform in other HPV6/11-driven indications, such as genital warts, and HPV16/18-driven indications, such as cervical cancer and head and neck cancers," said Helen Sabzevari, PhD, President and CEO of Precigen. "As communicated previously, we plan to pursue strategic partnerships to advance our UltraCAR-T programs, which deliver autologous, antigen-specific CAR-T cells overnight to a patient at the patient’s medical center. In conjunction, we are preparing for an end of Phase 1b meeting with the FDA for PRGN-3006 to share results for this highly promising program, including new clinical biomarker data that may further enable patient stratification and positively impact efficacy."

Realizing Precigen’s Commercial Vision for PRGN-2012 AdenoVerse Gene Therapy in RRP

PRGN-2012 (INN: zopapogene imadenovec†) is an investigational off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP), a rare and devastating chronic disease for which the current standard-of-care is repeated surgeries. If approved, PRGN-2012 has the potential to be the first US Food and Drug Administration (FDA)-approved therapeutic for the treatment of RRP.
PRGN-2012 received Breakthrough Therapy Designation from the FDA. PRGN-2012 also received Orphan Drug Designation from the FDA and Orphan Drug Designation from the European Commission.
In December 2024, the Company announced that it had completed the submission of a biologics license application (BLA) with a request for priority review for PRGN-2012 for the treatment of adults with RRP to the FDA. The submission is in the initial 60 day review period, during which time the FDA will decide whether to accept the BLA for further review and set the Prescription Drug User Fee Act (PDUFA) action date. The BLA included a request for priority review, which, if granted, would reduce the review timeline from the standard 10-month to a priority 6-month review from the date the submission is accepted by the FDA.
The BLA, under an accelerated approval pathway, is supported by data from the Phase 1/2 pivotal study in which more than 50% of patients achieved Complete Response and more than 85% of patients had a decrease in surgical interventions in the year after PRGN-2012 treatment compared to the year prior to treatment. PRGN-2012 was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2.
The confirmatory clinical trial of PRGN-2012 was initiated and is enrolling patients in accordance with the guidance from the FDA to initiate the study prior to submission of the BLA.
The Company continues to rapidly advance its commercial and manufacturing readiness campaign in anticipation of a potential 2025 launch.
Maximizing the Potential of PRGN-2009 AdenoVerse Gene Therapy in HPV-associated Cancers

PRGN-2009 Phase 2 clinical trials under a cost-effective cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer are ongoing.
Enrollment was paused in the cervical cancer Phase 2 clinical trial at non-NCI sites as part of strategic reprioritization activities in 2024.
Maximizing the Value of the UltraCAR-T Platform through Strategic Partnerships

Enrollment was completed for the Phase 1b trial for PRGN-3006, which received Fast Track designation from the FDA for the treatment of relapsed or refractory (r/r) acute myeloid leukemia (AML).
Based on the results of correlative studies of the patient samples from the Phase 1/1b study, the Company has identified clinical biomarkers that correlate to objective responses after PRGN-3006 treatment in r/r AML patients. This advancement may further enable patient stratification and positively impact efficacy.
The Company is preparing for an end of Phase 1b meeting with the FDA to discuss results and next steps.
The Company plans to focus on strategic partnership opportunities to advance this promising UltraCAR-T program in AML.
*Cash on-hand is preliminary and unaudited and reflects preliminary financial information as of December 31, 2024. In preparing this information, the Company’s actual financial position as of December 31, 2024 has not yet been finalized by management or reviewed or audited by the Company’s independent registered public accounting firm. This information is also not a comprehensive statement of financial position or results of operations as of or for the year-ended December 31, 2024. Subsequent information or events may lead to material differences between the foregoing preliminary financial information and those reported in the Company’s subsequent SEC filings. Accordingly, investors should not place undue reliance on this preliminary financial information.

†zopapogene imadenovec is the international nonproprietary name (INN) for the investigational therapeutic known as PRGN-2012. Zopapogene imadenovec has not been approved by any health authority in any country for any indication.

On January 13, 2025 Autolus Therapeutics plc (Nasdaq: AUTL), a commercial stage biopharmaceutical company developing, manufacturing and delivering next-generation T cell therapies to patients, reported business updates and an overview of 2025 milestones (Press release, Autolus, JAN 13, 2025, View Source [SID1234649718]).

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"We enter 2025 with good momentum following the recent U.S. Food and Drug Administration (FDA) approval of AUCATZYL (obecabtagene autoleucel, or "obe-cel") and publication of the FELIX trial data in the New England Journal of Medicine. In addition, the inclusion of AUCATZYL in the NCCN Guidelines, noting the lack of REMS program associated with our therapy, provides further validation of the demonstrated clinical benefit, favorable safety profile and the potential impact of the therapy for patients in need," said Dr. Christian Itin, Chief Executive Officer of Autolus. "We are executing to plan with the commercial launch and are encouraged to enter the year with 24 authorized treatment centers out of the 30 we anticipated by end of Q1."

"With the AUCATZYL launch on track, we look forward to updating the market on additional clinical pipeline programs that we believe will drive future growth," Dr. Itin continued. "In the second half of 2024, we undertook an R&D portfolio review to evaluate the highest priority areas for strategic investment. In our upcoming R&D investor event we will provide an outlook and plan to share initial data from the ongoing CARLYSLE Phase 1 trial in systemic lupus erythematosus (SLE)."

AUCATZYL was approved by the FDA on November 8, 2024. Autolus expects to complete authorization of the first 30 treatment centers, covering approximately 60% of the identified target patient population, by the end of January 2025. The Company expects to complete authorization of 60 treatment centers, covering approximately 90% of the target patient population, by the end of 2025.

In December 2024, the National Comprehensive Cancer Network (NCCN) added AUCATZYL (obecabtagene autoleucel) to its Clinical Practice Guidelines in Oncology (NCCN Guidelines) for the treatment of adult patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). The NCCN is a not-for-profit alliance of 30 leading cancer centers devoted to patient care, research, and education. The NCCN Guidelines are a comprehensive set of guidelines detailing the sequential management decisions and interventions that currently apply to 97% of cancers affecting patients in the U.S and are intended to ensure that all patients receive preventive, diagnostic, treatment, and supportive services that reflect the latest evidence in oncological patient care.

Obe-cel is also under regulatory review in both the European Union and the United Kingdom, with marketing authorization submissions accepted by the European Medicines Agency (EMA) in April 2024, and the UK Medicines and Healthcare products Regulatory Agency (MHRA) in August 2024. Assuming precedent regulatory timelines, Autolus expects potential marketing approvals from the MHRA and EMA in the second half of 2025.

2025 Events & Milestones

Obe-cel in autoimmune disease: initial data from SLE Phase 1 study Q1 2025
R&D Investor Event, New York City April 23, 2025
Initial data from PY01 trial of obe-cel in pediatric ALL H2 2025
MHRA & EMA approvals for obe-cel in ALL H2 2025
SLE Phase 1 trial presentation at medical conference H2 2025
Additional details regarding the R&D investor event, including registration link, will be communicated in the near future.