On November 14, 2024 Curis, Inc. ("Curis") (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 inhibitor, reported its business update and financial results for the quarter ended September 30, 2024 (Press release, Curis, NOV 14, 2024, View Source [SID1234648395]).

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"We continue to make excellent progress across our clinical programs. We are especially excited about the recent R/R PCNSL data released in September which continue to demonstrate the activity of emavusertib in combination with ibrutinib in salvage-line patients and that the CR/CRu responses appear to be durable," said James Dentzer, Curis Chief Executive Officer. "We are also excited to present additional clinical data in our TakeAim Leukemia study next month at ASH (Free ASH Whitepaper). We believe the positive momentum as we finish the year sets us up well for 2025."

Third Quarter 2024 and Recent Operational Highlights

Emavusertib (IRAK4 Inhibitor)

TakeAim Lymphoma

In September, at the 3rd Annual IRAK4 Symposium in Cancer, the Company released preliminary efficacy data in 10 response-evaluable patients who had progressed on treatment with a BTKi. The data showed 3 complete responses (CR), 1 unconfirmed complete response (CRu) and 2 partial responses (PR). The duration of response for 3 of the 4 patients with a CR/CRu was greater than 6 months.
The Company is actively engaged in discussions with regulatory authorities to gain alignment on the registrational path in PCNSL.
TakeAim Leukemia

The Company will have both an oral presentation and a poster presentation at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December.

Oral Presentation:
Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: New Treatment Approaches for AML
Session Date: Monday, December 9, 2024
Presentation Time: 11:30 AM
Room: Manchester Grand Hyatt San Diego, Grand Hall B
Publication Number: 737
Title: Preliminary Safety, Efficacy, and Molecular Characterization of Emavusertib (CA-4948) in Relapsed/Refractory Acute Myeloid Leukemia Patients

Poster:
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster II
Session Date: Sunday, December 8, 2024
Presentation Time: 6:00 PM – 8:00 PM
Location: San Diego Convention Center, Halls G-H
Publication Number: 3225
Title: Preliminary Safety, Efficacy and Molecular Characterization in Patients with Higher-Risk Myelodysplastic Syndrome Treated with Single Agent Emavusertib (CA-4948)

Corporate

In October 2024, Curis completed a registered direct offering and concurrent private placement of unregistered warrants ("October 2024 Offerings") with net proceeds of approximately $10.8 million.

Third Quarter 2024 Financial Results

For the third quarter of 2024, Curis reported a net loss of $10.1 million or $1.70 per share on both a basic and diluted basis as compared to $12.2 million or $2.13 per share on both a basic and diluted basis, for the same period in 2023. Curis reported a net loss of $33.8 million or $5.77 per share on both a basic and diluted basis, for the nine months ended September 30, 2024 as compared to a net loss of $35.7 million or $6.96 per share on both a basic and diluted basis for the same period in 2023.

Revenues for the third quarter of 2024 were $2.9 million as compared to $2.8 million for the same period in 2023. Revenues for both periods consist of royalty revenues from Genentech and Roche’s sales of Erivedge. Revenues for the nine months ended September 30, 2024 and 2023 were $7.6 million and $7.3 million, respectively.

Research and development expenses were $9.7 million for the third quarter of 2024, as compared to $10.4 million for the same period in 2023. The decrease was primarily attributable to lower consulting and employee related costs. Research and development expenses were $29.6 million for the nine months ended September 30, 2024, as compared to $29.5 million for the same period in 2023.

General and administrative expenses were $3.8 million for the third quarter of 2024, as compared to $4.8 million for the same period in 2023. The decrease was primarily attributable to lower legal and employee related costs. General and administrative expenses were $13.4 million for the nine months ended September 30, 2024, as compared to $13.8 million for the same period in 2023.

Other income, net was $0.5 million for the third quarter of 2024, as compared to $0.2 million for the same period in 2023. The increase was primarily attributable to a decrease in the non-cash expense related to the sale of future royalties. Other income, net was $1.8 million for the nine months ended September 30, 2024, as compared to $0.4 million for the same period in 2023.

Including the impact of the October 2024 Offerings, Curis’s cash and cash equivalents totaled $31.6 million, and the Company had approximately 8.5 million shares of common stock outstanding. Curis expects its existing cash and cash equivalents will enable its planned operations into mid-2025.

Conference Call Information

Curis management will host a conference call today, November 14, 2024, at 8:30 a.m. ET, to discuss the business update and these financial results.

To access the live conference call, please dial 800-836-8184 from the United States or 1-646-357-8785 from other locations, to access the webcast login to View Source shortly before 8:30 a.m. ET. The webcast can also be accessed via the Curis website in the ‘Investors’ section.

On November 14, 2024 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells for the treatment of cancer and autoimmune disease, reported a business and financial update for the third quarter of 2024 (Press release, Cue Biopharma, NOV 14, 2024, View Source [SID1234648394]).

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Recent Business Highlights

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Presented positive updated data from the Phase 1 trials of CUE-101 and CUE-102 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 39th Annual Meeting (SITC 2024) November 6-10
Updated data from Phase 1 trial of CUE-101 in combination with KEYTRUDA (pembrolizumab) continued to demonstrate enhanced benefit versus historical studies with pembrolizumab alone. Key findings included an objective response rate (ORR) of 46%, 12-month overall survival (OS) of 91.3% and a median overall survival (mOS) of 21.8 months in first line (1L) HPV+ R/M HNSCC patients, as well as an ORR of 50% in the subset of 1L patients with low PD-L1 expression (combined positive score (CPS) 1-19)
Updated data from Phase 1 monotherapy trial of CUE-102 included evidence of selective expansion of WT1-specific T cells and anti-tumor activity, as well as a favorable tolerability profile with no dose limiting toxicities (DLTs) in patients with Wilms’ Tumor 1 (WT1)-expressing colorectal, gastric, ovarian and pancreatic cancers
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Demonstrated disease control rate (DCR) of 67% in late-stage pancreatic cancer patients treated with CUE-102 monotherapy, including an unconfirmed partial response (PR) with a 40% decrease in tumor burden
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Announced pricing of $12.0 million public offering
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Appointed industry veteran Lucinda Warren as Chief Business Officer
•
Continued advancement of preclinical programs, CUE-401 for induction and expansion of regulatory T cells, in collaboration with Ono Pharmaceutical, and CUE-501 for B cell depletion, positioning both programs towards drug candidate selection

"We are very pleased with the validating updated clinical data from our Phase 1 trials for both CUE-101 and CUE-102," said Daniel Passeri, chief executive officer of Cue Biopharma. "Importantly, we believe the maturing data further supports and strengthens our competitive differentiation and positioning for selective modulation of disease-specific T cells. This data further bolsters our confidence that the CUE-100 series, exemplified by CUE-101 and CUE-102, represents the potential of establishing a new standard of care for cancer patients. We are also very pleased with the continued progress of our preclinical autoimmune programs, both of which have moved closer towards drug candidate selection."

On November 14, 2024 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, reported financial results for the third quarter ended September 30, 2024 and other recent business developments (Press release, Celcuity, NOV 14, 2024, View Source [SID1234648393]).

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"Enrollment in our VIKTORIA-1 study remains robust and on-track. The PIK3CA wild-type cohort is 100% enrolled, and enrollment in the PIK3CA mutant cohort is on plan," said Brian Sullivan, CEO and co-founder of Celcuity. "Based on our current forecast of reaching the event thresholds that will trigger primary analysis in both the PIK3CA wild-type and mutant cohorts, we expect to report topline data for the PIK3CA wild-type cohort sometime in late Q1 2025 or during Q2 2025 and to report topline data for the PIK3CA mutant cohort in the second half of 2025."

Third Quarter 2024 Business Highlights and Other Recent Developments

● The VIKTORIA-1 Phase 3 clinical trial expects to provide topline data for the PIK3CA wild-type cohort in late Q1 2025 or during Q2 2025 and for the PIK3CA mutant cohort in the second half of 2025.

○ VIKTORIA-1 is evaluating gedatolisib in combination with fulvestrant with and without palbociclib in adults with HR+, HER2- advanced breast cancer who have received prior treatment with a CDK4/6 inhibitor.
○ The PIK3CA wild-type cohort is 100% enrolled and enrollment of the PIK3CA mutant cohort is on-track relative to plan.

● The VIKTORIA-2 Phase 3 clinical trial remains on track to enroll its first patient in Q2 2025.

○ The VIKTORIA-2 study is a global Phase 3 open-label randomized clinical trial evaluating the efficacy and safety of gedatolisib in combination with fulvestrant plus a CDK4/6 inhibitor, either ribociclib or palbociclib, in comparison to fulvestrant plus a CDK4/6 inhibitor as a first-line treatment for patients with HR+/HER2- advanced breast cancer who are endocrine therapy resistant.
○ Prior to the initiation of the Phase 3 portion of the trial, a safety run-in study will be conducted in 12-36 participants to assess the safety profile of gedatolisib in combination with ribociclib and fulvestrant.
○ Site qualification activities to support activation of up to 200 sites across North America, Europe, Latin America, and Asia are on track.

● The Phase 1b/2 clinical trial, evaluating gedatolisib in combination with darolutamide for the treatment of patients with metastatic castration resistant prostate cancer (mCRPC), is ongoing and expected to report preliminary data in Q2 2025.

● Overall survival data from the B2151009 Phase 1b clinical trial will be presented at the San Antonio Breast Cancer Symposium (SABCS), taking place December 10-13, 2024. Details of the poster presentation are as follows:

○ Abstract Title: Overall survival in patients with HR+/HER2- advanced breast cancer treated in a phase 1b trial evaluating gedatolisib in combination with palbociclib and endocrine therapy (SESS-1510)
○ Presentation Number: P4-08-25
○ Date/Time: Thursday, December 12, 5:30 PM CST

● Additional nonclinical data further characterizing the mechanism of action of gedatolisib and its effect on breast cancer cell metabolic functions will also be presented at the SABCS.

○ Abstract Title: Mechanism of action of gedatolisib in combination with fulvestrant and/or palbociclib in estrogen receptor positive breast cancer models (SESS-989)
○ Abstract Title: Different effects of gedatolisib versus single-node PI3K/AKT/mTOR pathway inhibitors on breast cancer cell metabolic functions (SESS-997)
● In October, Cancers published results of nonclinical studies in gynecological cancer cell line models highlighting the differences between single-node inhibitors of the PI3K/AKT/mTOR pathway and gedatolisib. The published manuscript is available online and on the publications section of Celcuity’s website.

Third Quarter 2024 Financial Results

Unless otherwise stated, all comparisons are for the third quarter ended September 30, 2024, compared to the third quarter ended September 30, 2023.

Total operating expenses were $30.1 million for the third quarter of 2024, compared to $18.9 million for the third quarter of 2023.

Research and development (R&D) expenses were $27.6 million for the third quarter of 2024, compared to $17.5 million for the prior-year period. Of the approximately $10.1 million increase in R&D expenses, $6.3 million primarily related to activities supporting the VIKTORIA-1 Phase 3 trial, the Phase 1b/2 trial and the initiation of the VIKTORIA-2 Phase 3 trial, and $3.8 million was related to increased employee and consulting expenses.

General and administrative (G&A) expenses were $2.5 million for the third quarter of 2024, compared to $1.4 million for the prior-year period. Employee and consulting related expenses accounted for $0.9 million of the increase. Professional fees and other administrative expenses accounted for the remaining increase of approximately $0.2 million.

Net loss for the third quarter of 2024 was $29.8 million, or $0.70 loss per share, compared to a net loss of $18.4 million, or $0.83 loss per share, for the third quarter of 2023. Non-GAAP adjusted net loss for the third quarter of 2024 was $27.6 million, or $0.65 loss per share, compared to non-GAAP adjusted net loss of $17.3 million, or $0.78 loss per share, for the third quarter of 2023. Non-GAAP adjusted net loss excludes stock-based compensation expense, non-cash interest expense, and non-cash interest income. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States (GAAP) to non-GAAP financial measures, please see the financial tables at the end of this press release.

Net cash used in operating activities for the third quarter of 2024 was $20.6 million, compared to $12.7 million for the third quarter of 2023.

At September 30, 2024, Celcuity reported cash, cash equivalents and short-term investments of $264.1 million.

On November 14, 2024 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported financial results for the third quarter ended September 30, 2024, and provided a corporate update (Press release, Candel Therapeutics, NOV 14, 2024, View Source [SID1234648392]).

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"We remain on track for phase 2b and phase 3 topline data in non-metastatic, localized prostate cancer for CAN-2409 in the fourth quarter of 2024, and hope we will deliver the data and regulatory approvals to enable a paradigm shift in how these patients will be treated in the future. We continue to advance our clinical and pre-clinical candidates, while leveraging our robust enLIGHTENTM Discovery Platform to identify new and innovative assets that may be impactful in cancer immunotherapy," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "We are encouraged by the first clinical and biomarker activity data after repeated injection of CAN-3110 from our ongoing phase 1b clinical trial of CAN-3110 in recurrent high-grade glioma, which suggests a long tail of survival. We are also excited about the data that supports potential expansion of CAN-3110 from recurrent high-grade glioma into melanoma, where we observed antitumor activity in pre-clinical models."

Dr. Tak continued, "As to the future, we are also looking forward to reporting updated overall survival data from both our ongoing CAN-2409 phase 2 NSCLC and pancreatic cancer clinical trials in Q1 2025."

Third Quarter 2024 & Recent Highlights

o
CAN-3110 – Recurrent High-Grade Glioma
▪
Received orphan drug designation from the U.S. Food and Drug Administration (FDA) for CAN-3110 for the treatment of recurrent high-grade glioma (rHGG).
▪
Presented a Trial-in-Progress poster at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on the ongoing phase 1b clinical trial exploring multiple doses of CAN-3110 in patients with rHGG.
▪
Presented clinical and biomarker activity data from the ongoing phase 1b clinical trial at the 16th Annual International Oncolytic Virotherapy Conference (IOVC).
▪
In the oral presentation, the investigators reported ongoing improved survival compared to historical controls, with 3 out of 6 patients still alive after more than one year (12.2, 13.0, and 18.7 months, respectively) after initiation of experimental treatment with repeated CAN-3110 injections.
▪
The data also show discrepancies between imaging and histologic findings, suggesting radiologic pseudo-progression: there was a near absence of tumor cells alongside dense lymphocyte infiltrates in biopsies obtained after CAN-3110 administration, especially in patients with enhancement on post-treatment magnetic resonance imaging (MRI) scans.
o
CAN-3110 – Melanoma
▪
Presented preclinical results on the therapeutic potential of CAN-3110 in the Ras-Raf pathway altered melanoma model at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 39th Annual Meeting.
▪
CAN-3110 exhibited potent, tumor-specific cytotoxicity in human and murine melanoma cell lines with varied CDKN2A pathway alterations and Nestin expression. In vivo mouse studies showed dose-dependent inhibition of tumor growth, with regression observed in a subset (3 of 8) of tumors treated with a high dose of CAN-3110.

▪
Cytotoxic activity in melanoma-bearing mice was associated with systemic immune activation, including increased activation and proliferation of circulating T cells.
▪
Findings mirror those from rHGG patients treated with CAN-3110 reported last year in Nature. The therapy was well-tolerated in preclinical models based on body weight and histopathological analysis following intratumoral administration.
o
enLIGHTEN Discovery Platform
▪
Presented poster titled "A first-in-class multimodal immunotherapy for enhanced immune activation in the tumor microenvironment as a novel therapeutic strategy for solid tumors" at IOVC.
▪
The presentation focused on the latest asset from the enLIGHTEN Discovery Platform, a multimodal viral therapeutic candidate encoding IL-12 and IL-15. Data showed the ability of the asset to induce expansion and activation of natural killer and CD8+ T cell populations, resulting in significant tumor growth inhibition and regression in two different models.
Anticipated Milestones

•
Phase 2b topline data for CAN-2409 in low-to-intermediate-risk, localized, non-metastatic prostate cancer expected in Q4 2024.
•
Phase 3 topline disease-free survival data for CAN-2409 in localized intermediate/high-risk prostate cancer expected in Q4 2024.
Financial Results for Third Quarter Ended September 30, 2024

Research and Development Expenses: Research and development expenses were $5.4 million for the third quarter of 2024 compared to $5.8 million for the third quarter of 2023. The decrease was primarily due to lower payroll-related expenses following the corporate restructuring in the fourth quarter of 2023 and a decrease in depreciation, impairment, and loss on the sale of fixed assets. These decreases were partially offset by clinical development costs driven by increased manufacturing costs for CAN-2409 programs. Research and development expenses included non-cash stock compensation expense of $0.6 million for the third quarter of 2024 compared to $0.3 million for the third quarter of 2023.

General and Administrative Expenses: General and administrative expenses were $3.3 million for the third quarter of 2024 compared to $3.0 million for the third quarter of 2023. The increase was primarily due to increased professional and consulting fees. The increase was partially offset by lower insurance costs. General and administrative expenses included non-cash stock compensation expense of $0.5 million for the third quarter of 2024 compared to $0.4 million for the third quarter of 2023.

Net Loss: Net loss for the third quarter of 2024 was $10.6 million, compared to a net loss of $8.4 million for the third quarter of 2023, and included other expense, net of $1.9 million for the third quarter of 2024 and other income, net of $0.4 million for the third quarter of 2023, primarily due to the change in the fair value of the Company’s warrant liability.

Cash Position: Cash and cash equivalents, as of September 30, 2024, were $16.6 million, as compared to $35.4 million as of December 31, 2023. Based on current plans and assumptions, the Company expects that its existing cash and cash equivalents will be sufficient to fund its current operating plan to the end of the first quarter of 2025.

On November 14, 2024 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported third quarter results and business highlights for the quarter ended September 30, 2024, including recent commercial and operational progress (Press release, bluebird bio, NOV 14, 2024, View Source [SID1234648391]).

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"Patient starts more than doubled from our second to third quarter update, providing clear evidence that our commercial launches continued to accelerate," said Andrew Obenshain, chief executive officer. "This momentum, coupled with steps we took in the third quarter to increase manufacturing capacity for ZYNTEGLO and optimize our cost structure, is propelling bluebird forward on our path to becoming a sustainable commercial gene therapy company. We remain focused on securing additional cash resources to extend our runway, which we believe would enable us to achieve this vision and reach cash flow break-even in the second half of 2025."

COMMERCIAL LAUNCH UPDATES

Continued commercial momentum across the portfolio

57 patient starts completed to date in 2024 (35 ZYNTEGLO, 17 LYFGENIA, 5 SKYSONA).
17 additional starts scheduled through the remainder of 2024.
Evidence of strong commercial demand, with 30 patient starts already scheduled in 2025, supporting the potential for cash flow breakeven in the second half of 2025.
More than 70 activated QTCs, with 40% having initiated or completed treatment for at least one patient.
Validated access and reimbursement strategy is driving favorable coverage landscape

To date, more than half of all states have affirmed coverage for LYFGENIA through a preferred drug list or published coverage criteria.
Nearly 50% of Medicaid-insured individuals with sickle cell disease in the U.S. live in a state that has already completed prior authorization approval for the use of LYFGENIA for at least one patient.
Multiple outcomes-based agreements are published and in place for LYFGENIA with national commercial payer organizations, representing more than 200 million U.S. lives.
DATA PRESENTATIONS AT ASH (Free ASH Whitepaper) 2024

Updated data from the Company’s lentiviral vector (LVV) gene addition programs in patients with sickle cell disease who have a history of vaso-occlusive events and patients with beta-thalassemia who require regular blood transfusions will be presented at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. The meeting will take place December 7-10, 2024 at the San Diego Convention Center and online.

SICKLE CELL DISEASE DATA

Oral Presentation [#511]: An Update on Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-cel
Presenting Author: Dr. Stacey Rifkin-Zenenberg (Hackensack)
Date/Time: Sunday, December 8, 2024, 9:30 a.m. – 11:00 a.m. PT
Poster Presentation [#3576]: Participants with a History of Stroke in Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials
Presenting Author: Dr. Jen Jaroscak (The Medical University of South Carolina)
Date/Time: Sunday, December 8, 2024, 6:00 p.m. – 8:00 p.m. PT
BETA-THALASSEMIA DATA

Poster Presentation [#2194]: Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy results in durable Hemoglobin A (HbA) Production with up to 10 Years of Follow-Up in Participants with Transfusion-Dependent β-Thalassemia
Presenting Author: Dr. Alexis A Thompson (Children’s Hospital of Philadelphia)
Date/Time: Saturday, December 7, 2024, 5:30 p.m. – 7:30 p.m. PT
Abstracts outlining bluebird bio’s accepted data at ASH (Free ASH Whitepaper) 2024 are available on the ASH (Free ASH Whitepaper) conference website.

THIRD QUARTER FINANCIAL HIGHLIGHTS

Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately $118.7 million, including restricted cash of approximately $48.0 million, as of September 30, 2024.

bluebird and Hercules are engaging collaboratively as bluebird works to secure adequate cash runway to obtain additional financing and reach cash flow break-even. Based on current forecasts, which assume continued cost-saving initiatives, successfully renegotiating key contracts, and continued collaborative engagement from Hercules, we expect our existing cash and cash equivalents will enable us to fund our operations into the first quarter of 2025.

The Company anticipates quarterly cash flow break-even in the second half of 2025, assuming it scales to approximately 40 drug product deliveries per quarter and obtains additional cash resources to extend its runway.
Revenue, net: Total revenue, net was $10.6 million for the three months ended September 30, 2024, compared to $12.3 million for the three months ended September 30, 2023, driven by quarter-to-quarter variability in drug product infusions.

Revenue for the third quarter includes revenue from LYFGENIA, following the completion of the first infusion for sickle cell disease.

bluebird previously guided to an anticipated reduction of net revenue in the third quarter; the Company now anticipates net revenue of at least $25 million in the fourth quarter 2024, as previously reported patient starts are infused.
Cost of Product Revenue: Cost of product revenue was $11.8 million for the three months ended September 30, 2024, compared to $9.1 million for the three months ended September 30, 2023.
SG&A Expenses: Selling, general and administrative expenses were $39.8 million for the three months ended September 30, 2024, compared to $40.8 million for the three months ended September 30, 2023. The decrease of $1.0 million was primarily driven by decrease in employee compensation, benefit, and other headcount related expenses, commercial expenses, and facility fees, partially offset by increased professional services fees.
R&D Expenses: Research and development expenses were $23.2 million for the three months ended September 30, 2024, compared to $58.5 million for the three months ended September 30, 2023. The decrease of $35.3 million was primarily driven by material production shift to inventory and cost of product revenue as well as decreased employee compensation, benefit, and other headcount related expenses, consulting fees, and facility and information technology fees.
Net income (loss): Net loss was $60.8 million for the three months ended September 30, 2024, compared to a net loss of $87.2 million for the three months ended September 30, 2023.
CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss its third quarter 2024 results and business updates today, Wednesday, November 14, 2024, at 8:00 am ET.

To access the live conference call via telephone, please register at this link to receive a dial in number and unique PIN.

To access the live webcast, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source A replay of the webcast will be available on the bluebird bio website for 90 days following the event.