On November 7, 2023 Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS), a clinical-stage biopharmaceutical company, reported a business update and announced financial results for the third quarter ended September 30, 2023 (Press release, Corvus Pharmaceuticals, NOV 7, 2023, View Source [SID1234637126]).

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"We continue to make progress towards initiating our Phase 3 registrational clinical trial for soquelitinib, including finalizing the study protocol and submitting it to the FDA," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "From a regulatory standpoint, we are clear to initiate the trial. We are delighted by the strong interest from leading academic centers in the U.S. in participating in the clinical trial, which is anticipated to begin enrolling patients by the second quarter 2024. We also plan to initiate a Phase 2 trial in early 2024 with soquelitinib monotherapy in recurrent renal cell cancer and have been encouraged by recent work from other academic groups demonstrating the potential of ITK inhibition in solid tumor animal models. In addition, our internal research team continues to reveal the therapeutic potential of ITK inhibition in several preclinical models of autoimmune and allergic disease, including the inhibition of Th17 cells and secretion of IL-17, which are validated mediators of inflammatory disease."

Business Update and Strategy

Prioritized Program: Soquelitinib (formerly CPI-818, Corvus’ selective ITK inhibitor)

Soquelitinib for T Cell Lymphoma

Corvus continues to enroll patients with relapsed PTCL in a Phase 1/1b clinical trial evaluating single agent therapy with soquelitinib. The latest data from the trial was reported at the International Conference on Malignant Lymphoma, which took place June 13-17, 2023 in Lugano, Switzerland. As of the May 18, 2023 cut-off date, the data showed that a majority of the patients treated with the optimal dose of 200 mg twice per day of soquelitinib experienced tumor regression.
New interim data from the Phase 1/1b clinical trial, along with complementary preclinical data, will be presented in a poster presentation at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2023.
Soquelitinib Preclinical Data in Hematologic and Solid Tumors

In July 2023, Corvus announced the publication of preclinical data on soquelitinib as a preprint at bioRxiv, which highlighted the selective inhibition of ITK being able to potentially enhance anti-tumor immune response to hematologic and solid tumors, indicating its potential as a novel approach to cancer immunotherapy.
In September 2023, a paper was published by an independent academic group in Scientific Reports validating the potential of ITK inhibition for treatment of solid tumors. The preclinical data demonstrated a reduction and reversal of T cell exhaustion markers and an increase in the infiltration of killer T cells into tumors, consistent with soquelitinib’s proposed mechanism of action. The paper highlights the potential of selective ITK inhibition for the treatment of cancers and helps to confirm preclinical and clinical results generated by Corvus.
Soquelitinib Preclinical Data in Autoimmune/Allergy

On November 1, 2023, 2023, Corvus announced the publication of preclinical data on soquelitinib as a preprint at bioRxiv that demonstrated ITK’s selective inhibition which produced therapeutic benefits in several autoimmune and allergy preclinical models including psoriasis, asthma, pulmonary fibrosis, scleroderma and graft versus host disease. The mechanism of action involves the inhibition of Th2 and Th17 cells and the subsequent production of cytokines such as IL-17, IL-4, IL-5 and other cytokines involved in these diseases. The novel mechanism is a result of ITK inhibition and blockade of formation of Th2 and Th17 cells.
Partner Led Programs: Ciforadenant (adenosine 2a receptor inhibitor) and Mupadolimab (anti-CD73)

The Kidney Cancer Research Consortium is enrolling a Phase 1b/2 clinical trial evaluating ciforadenant as a potential first line therapy for metastatic renal cell cancer (RCC) in combination with ipilimumab (anti-CTLA-4) and nivolumab (anti-PD-1). The Phase 1b portion of this trial has been completed and patients are now being enrolled in the Phase 2 portion. The clinical trial is expected to enroll up to 60 patients and initial data is anticipated in early 2024.
Angel Pharmaceuticals, Corvus’ partner in China, is enrolling patients in a Phase 1/1b clinical trial of mupadolimab in patients with non-small cell lung cancer (NSCLC) and head and neck squamous cell cancers. In this clinical trial, patients will receive mupadolimab monotherapy or in combination with pembrolizumab.
Financial Results
As of September 30, 2023, Corvus had cash, cash equivalents and marketable securities of $32.2 million as compared to $42.3 million as of December 31, 2022. During the nine months ending September 30, 2023, the Company sold 2,461,903 shares of its common stock through its at-the-market program, generating net proceeds to the Company of approximately $7.8 million. Corvus expects full year 2023 net cash used in operating activities to be between approximately $22 million and $23 million, resulting in a projected cash balance of between $27 million and $28 million as of December 31, 2023. Based on its current plans, Corvus expects its cash to fund operations into late 2024.

Research and development expenses for the three months ended September 30, 2023 totaled $4.0 million compared to $10.4 million for the same period in 2022. The decrease of $6.4 million was primarily due to lower clinical trial and manufacturing costs associated with the development of mupadolimab.

The net loss for the three months ended September 30, 2023 was $6.0 million compared to a net loss of $14.8 million for the same period in 2022. Total stock compensation expense for the three months ended September 30, 2023 was $0.5 million compared to $0.7 million for the same period in 2022 and the non-cash loss from Corvus’ equity method investment in Angel Pharmaceuticals was $0.9 million for the three months ended September 30, 2023 compared to $2.7 million in the same period in 2022.

Conference Call Details
Corvus will host a conference call and webcast today, Tuesday, November 7, 2023, at 4:30 p.m. ET (1:30 p.m. PT), during which time management will provide a business update and discuss the third quarter 2023 financial results. The conference call can be accessed by dialing 1-855-327-6837 (toll-free domestic) or 1-631-891-4304 (international) or by clicking on this link for instant telephone access to the event. The live webcast may be accessed via the investor relations section of the Corvus website. A replay of the webcast will be available on Corvus’ website for 90 days.

About Corvus Pharmaceuticals
Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company pioneering the development of ITK inhibition as a new approach to immunotherapy for a broad range of cancer and immune diseases. The Company’s lead product candidate is soquelitinib, an investigational, oral, small molecule drug that selectively inhibits ITK. Corvus plans to initiate a Phase 3 registrational clinical trial for soquelitinib in patients with relapsed peripheral T cell lymphoma. Its other clinical-stage candidates are being developed for a variety of cancer indications. For more information, visit www.corvuspharma.com.

About Soquelitinib
Soquelitinib (CPI-818) is an investigational small molecule drug given orally designed to selectively inhibit ITK (interleukin-2-inducible T cell kinase), an enzyme that is expressed predominantly in T cells and plays a role in T cell and natural killer (NK) cell immune function. The immunologic effects of soquelitinib lead to what is known as Th1 skewing and is made possible by the high selectivity of soquelitinib for ITK. Research on soquelitinib’s mechanism of action suggests that it has the potential to control differentiation of normal T helper cells and enhance immune responses to tumors by augmenting the generation of cytotoxic killer T cells and the production of cytokines that inhibit cancer cell survival. Soquelitinib has also been shown to prevent T cell exhaustion, a major limitation of current immunotherapy and CAR-T therapies. Optimal doses of soquelitinib have been shown to affect T cell differentiation and induce the generation of Th1 helper cells while blocking the development of both Th2 and Th17 cells and production of their secreted cytokines. Th1 T cells are required for immunity to tumors, viral infections and other infectious diseases. Th2 and Th17 helper T cells are involved in the pathogenesis of many autoimmune and allergic diseases. The Company believes the inhibition of specific molecular targets in T cells may be of therapeutic benefit for patients with cancers, including solid tumors, and in patients with autoimmune and allergic diseases. Based on interim results from a Phase 1/1b clinical trial in patients with refractory T cell lymphomas, which demonstrated tumor responses in very advanced, refractory, difficult to treat T cell malignancies, the Company plans to initiate a registrational Phase 3 clinical trial of soquelitinib in patients with relapsed peripheral T cell lymphoma (PTCL).

About Peripheral T Cell Lymphoma
Peripheral T cell lymphoma is a heterogeneous group of malignancies accounting for about 10% of non-Hodgkin’s lymphomas (NHL) in Western populations, reaching 20% to 25% of NHL in some parts of Asia and South America. The most common subtypes are PTCL-not otherwise specified (PTCL-NOS) and T follicular helper cell lymphoma. First line treatment for these diseases is typically combination chemotherapy, however, approximately 75% of patients either do not respond or relapse within the first two years. Patients in relapse are treated with various chemotherapy agents but have poor overall outcomes with median progression-free survival in the three to four month range and overall median survival of six to 12 months. There are no approved drugs in relapsed PTCL based on randomized trials.

PTCL is a disease of mature helper T cells that express ITK, often containing numerous genetic mutations and frequently associated with viral infection. Most often the malignant cells of PTCL express a Th2 phenotype.

About Ciforadenant
Ciforadenant (CPI-444) is an investigational small molecule, oral, checkpoint inhibitor designed to disable a tumor’s ability to subvert attack by the immune system by blocking the binding of adenosine to immune cells present in the tumor microenvironment. Adenosine, a metabolite of ATP (adenosine tri-phosphate), is produced within the tumor microenvironment where it may bind to the adenosine A2A receptor present on immune cells and block their activity. Ciforadenant has been shown to block the immunosuppressive effects of myeloid cells present in tumors and preclinical studies published in 2018 demonstrated synergy with combinations of anti PD1 and anti-CTLA4 antibodies.

About Mupadolimab
Mupadolimab (CPI-006) is an investigational, potent humanized monoclonal antibody that is designed to react with a specific site on CD73. In preclinical studies, it has demonstrated immunomodulatory activity resulting in activation of lymphocytes, induction of antibody production from B cells and effects on lymphocyte trafficking. While there are other anti-CD73 antibodies and small molecules in development for treatment of cancer, such agents react with a different region of CD73. Mupadolimab is designed to react with a region of the molecule that acts to stimulate B cells and block production of immunosuppressive adenosine. Mupadolimab is being studied in combination with pembrolizumab in a Phase 1b/2 clinical trial in patients with advanced head and neck cancers and in patients with NSCLC that have failed chemotherapy and anti-PD(L)1 therapy. It is postulated that the activation of B cells will enhance immunity within the tumors of these patients, leading to improved clinical outcomes.

On November 7, 2023 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported financial results for the third quarter of 2023 and reviewed recent business updates (Press release, Caribou Biosciences, NOV 7, 2023, View Source [SID1234637125]).

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"In 2023, we have advanced our programs to build value across the pipeline and position Caribou as a leader in the allogeneic CAR-T cell therapy space," said Rachel Haurwitz, PhD, Caribou’s president and chief executive officer. "We plan to meet with the FDA to discuss a potential pivotal trial in second-line LBCL patients for our lead program, CB-010, and we intend to share the agency’s feedback by the end of this year. Patient enrollment is ongoing in the ANTLER trial, and we expect to report initial dose expansion data in the first half of 2024. Additionally, we are enrolling patients in our CaMMouflage trial for CB-011 and plan to initiate enrollment in the AMpLify Phase 1 clinical trial for CB-012 by mid-2024. With two years of cash and continued financial discipline, we are well positioned to execute on our current programs and continue Caribou’s momentum."

Accomplishments and highlights

Pipeline and technology
•CB-010: Caribou continues to enroll second-line LBCL patients in the dose expansion portion of the ongoing ANTLER Phase 1 clinical trial based on positive data from the dose escalation portion of the trial. CB-010 is an allogeneic anti-CD19 CAR-T cell therapy in development for relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL). The dose expansion data will be used to determine the recommended Phase 2 dose (RP2D).
•CB-011: Caribou continues to enroll patients at dose level 2 (150×106 CAR-T cells) in the dose escalation portion of the ongoing CaMMouflage Phase 1 trial of CB-011, an allogeneic anti-BCMA CAR-T cell therapy, for relapsed or refractory multiple myeloma (r/r MM). Caribou has concluded dose level 1 (50×106 CAR-T cells, N=3) without any dose-limiting toxicities (DLTs) and received Study Steering Committee clearance to proceed with dosing patients at dose level 2.
•CB-012: Caribou recently announced clearance of the company’s investigational new drug (IND) application from the U.S. Food and Drug Administration (FDA) for CB-012, an allogeneic anti-CLL-1 CAR-T cell therapy, for relapsed or refractory acute myeloid leukemia (r/r AML). Clinical site activation is underway for the AMpLify Phase 1 clinical trial and Caribou plans to initiate patient enrollment by mid-2024 to evaluate CB-012 at ascending dose levels starting at dose level 1 (25×106 CAR-T cells).

Anticipated milestones
•CB-010: Caribou plans to meet with the FDA to discuss a potential pivotal clinical trial in second-line LBCL patients and plans to share FDA feedback by year-end 2023. The Company also plans to report initial dose expansion data in second-line LBCL patients from the ongoing ANTLER trial in H1 2024.
•CB-011: Caribou plans to provide updates on dose escalation as the CaMMouflage Phase 1 clinical trial in r/r MM advances.
•CB-012: Caribou plans to initiate patient enrollment in the AMpLify Phase 1 clinical trial in r/r AML by mid-2024.

Corporate updates
•Appointed two multiple myeloma experts to Caribou’s scientific advisory board: Caribou appointed two experts in multiple myeloma drug development to its scientific advisory board [www.cariboubio.com/about/#sab] in the third quarter of 2023.
◦Sundar Jagannath, MD, director of the Center of Excellence for Multiple Myeloma and professor of medicine at the Tisch Cancer Institute of Mount Sinai.
◦Sriram Krishnaswami, PhD, vice president and development head for multiple myeloma at Pfizer Oncology’s Global Product Development division.
•Appointed chief people officer: In September 2023, Caribou appointed Reigin Zawadzki [View Source to the newly created position of chief people officer. Ms. Zawadzki brings over 20 years of experience leading human resources in the biotechnology industry and will lead Caribou’s people strategy.
•Completed successful $134.4 million follow-on financing: In the third quarter of 2023, Caribou completed an underwritten public offering of 22,115,384 shares of its common stock, which included the full exercise of the underwriters’ option to purchase additional shares. The net proceeds to Caribou were $134.4 million.
•Received $25.0 million Pfizer investment: In July 2023, Caribou announced that Pfizer invested [View Source $25.0 million in Caribou common shares on June 30, 2023. Caribou will use the proceeds from this investment to advance CB-011. Caribou maintains full ownership and control of CB-011 and its other allogeneic CAR-T and CAR-NK cell therapies.

Third quarter 2023 financial results
Cash, cash equivalents, and marketable securities: Caribou had $396.7 million in cash, cash equivalents, and marketable securities as of September 30, 2023, compared to $317.0 million as of December 31, 2022. This amount includes the approximately $134.4 million in net proceeds from the Company’s underwritten public offering and the $25.0 million equity investment from Pfizer. Caribou expects its cash, cash equivalents, and marketable securities will be sufficient to fund its current operating plan into Q4 2025.

Licensing and collaboration revenue: Revenue from Caribou’s licensing and collaboration agreements was $23.7 million for the three months ended September 30, 2023, compared to $3.3 million for the same period 2022. The increase was primarily due to $21.5 million in revenue recognized under the AbbVie Collaboration and License Agreement, including $20.8 million of revenue recognized upon termination of this agreement as previously disclosed, which was the remaining deferred revenue balance from AbbVie’s $30 million upfront payment in February 2021.
R&D expenses: Research and development expenses were $28.6 million for the three months ended September 30, 2023, compared to $20.0 million for the same period in 2022. The increase was primarily due to costs to advance pipeline programs, including the CB-010 ANTLER and CB-011 CaMMouflage Phase 1 clinical trials; personnel-related expenses, including stock-based compensation, due to headcount increases; and facilities and other allocated expenses.

G&A expenses: General and administrative expenses were $9.7 million for the three months ended September 30, 2023, compared to $9.8 million for the same period in 2022. The decrease was primarily due to lower patent prosecution and maintenance costs, and lower insurance, accounting, and other service-related expenses. This decrease was partially offset by an increase in personnel-related expenses, including stock-based compensation, due to headcount increases.
Net loss: Caribou reported a net loss of $10.0 million for the three months ended September 30, 2023, compared to $26.6 million for the same period in 2022.

About CB-010
CB-010 is the lead product candidate from Caribou’s allogeneic CAR-T cell therapy platform and is being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL). In the ongoing ANTLER Phase 1 trial, Caribou is enrolling second-line patients with large B cell lymphoma (LBCL) comprised of different subtypes of aggressive r/r B-NHL (DLBCL NOS, PMBCL, HGBL, tFL, and tMZL). CB-010 is an allogeneic anti-CD19 CAR-T cell therapy engineered using Cas9 CRISPR hybrid RNA-DNA (chRDNA) genome-editing technology. To Caribou’s knowledge, CB-010 is the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knockout, a genome-editing strategy designed to improve antitumor activity by limiting premature CAR-T cell exhaustion. To Caribou’s knowledge, CB-010 is the first anti-CD19 allogeneic CAR-T cell therapy to be evaluated in the second-line LBCL setting and it has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations by the FDA. Additional information on the ANTLER trial (NCT04637763) can be found at clinicaltrials.gov.

About CB-011
CB-011 is a product candidate from Caribou’s allogeneic CAR-T cell therapy platform and is being evaluated in patients with relapsed or refractory multiple myeloma (r/r MM) in the CaMMouflage Phase 1 trial. CB-011 is an allogeneic anti-BCMA CAR-T cell therapy engineered using Cas12a chRDNA genome-editing technology. To Caribou’s knowledge, CB-011 is the first allogeneic CAR-T cell therapy in the clinic that is engineered to improve antitumor activity through an immune cloaking strategy with a B2M knockout and insertion of a B2M–HLA-E fusion protein to blunt immune-mediated rejection. CB-011 has been granted Fast Track designation by the FDA. Additional information on the CaMMouflage trial (NCT05722418) can be found at clinicaltrials.gov.

About CB-012
CB-012 is a product candidate from Caribou’s allogeneic CAR-T cell therapy platform and will be evaluated in the AMpLify Phase 1 clinical trial in patients with relapsed or refractory acute myeloid leukemia (r/r AML). CB-012 is an anti-CLL-1 CAR-T cell therapy engineered with five genome edits, enabled by Caribou’s patented next-generation CRISPR technology platform, which uses Cas12a chRDNA genome editing to significantly improve the specificity of genome edits. To Caribou’s knowledge, CB-012 is the first allogeneic CAR-T cell therapy with both checkpoint disruption, through a PD-1 knockout, and immune cloaking, through a B2M knockout and B2M–HLA-E fusion protein insertion; both armoring strategies are designed to improve antitumor activity.

On November 7, 2023 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the third quarter ended September 30, 2023, including recent commercial and operational progress, and regulatory updates (Press release, bluebird bio, NOV 7, 2023, View Source [SID1234637124]).

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"We continue to see strong, linear growth for both the ZYNTEGLO and SKYSONA launches, with patients completing the treatment process, and significant demand across our established qualified treatment center network," said Andrew Obenshain, chief executive officer, bluebird bio. "Launch preparations for lovo-cel for sickle cell disease are well underway in anticipation of our December 2023 PDUFA date. In the third quarter, we accelerated the growth of our QTC network, which is one hundred percent synergistic across ZYNTEGLO and lovo-cel, and advanced conversations with payers who recognize the value of lovo-cel and have responded favorably to our innovative contract offerings. We are confident in our commercial gene therapy experience, committed to continued partnership with the sickle cell community, and excited about the opportunity to bring gene therapy to individuals living with SCD."

RECENT HIGHLIGHTS

Commercial launches of ZYNTEGLO (betibeglogene autotemcel) and SKYSONA (elivaldogene autotemcel)

Continued strong, linear growth for ZYNTEGLO with 16 patient starts (cell collections) since launch. Cell collection has been completed for 6 patients for SKYSONA to date.
In September, bluebird announced an amendment to its agreement with Lonza, which manufactures drug product for ZYNTEGLO and SKYSONA. The amended agreement enables increased manufacturing capacity for both therapies. The agreement also included modified payment terms intended to better align with the Company’s business objectives.
Value of ZYNTEGLO and SKYSONA continues to be recognized among commercial and government payers. Recently, bluebird signed outcomes-based agreements for ZYNTEGLO with Michigan and Massachusetts state Medicaid agencies. To date, the Company has received zero ultimate denials for both therapies and prior authorization approvals for drug product remain consistent at approximately two weeks across all payers.
bluebird’s qualified treatment center (QTC) network has scaled to 29 activated centers (defined as a signed master service agreement or MSA) including both adult and pediatric centers across 16 states. Of these 29 activated QTCs, four have been activated to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD) in addition to ZYNTEGLO. The Company remains on track to scale to 40-50 activated QTCs by the end of 2023.
lovo-cel FDA Review

On August 16, 2023, bluebird bio announced that the U.S. Food and Drug Administration (FDA) communicated that an advisory committee meeting will not be scheduled for lovotibeglogene autotemcel (lovo-cel).
The lovo-cel Biologics Licensing Application (BLA) review remains on track. The Agency previously accepted the lovo-cel BLA for Priority Review and set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023.
Advance Agreement to Sell PRV

On October 30, 2023, bluebird entered into an agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV), if received, in connection with the potential approval of lovo-cel for sickle cell disease. Under the terms of the agreement, rights to the PRV will transfer to the buyer and the Company will receive $103 million upon closing of the sale, which is contingent upon the FDA’s approval of the BLA for lovo-cel and granting of the PRV. bluebird anticipates receipt of a potential PRV should lovo-cel be approved for patients with sickle cell disease ages 12 and older. Proceeds from the potential sale of the PRV are not yet reflected in the Company’s cash runway.
Data Presentations at ASH (Free ASH Whitepaper) 2023

Updated long-term follow-up data from the Company’s gene therapy programs in sickle cell disease and beta-thalassemia will be presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 9-12, 2023 at the San Diego Convention Center and online. bluebird continues to have the most mature gene therapy programs for both SCD and beta-thalassemia, presenting up to five years and up to nine years of long-term data, respectively.

SICKLE CELL DISEASE DATA
Oral Presentation [#1051]: Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received lovotibeglogene autotemcel (lovo-cel) Gene Therapy: Up to 60 Months of Follow-up
Presenting Author: Julie Kanter, M.D., director of the UAB Adult Sickle Cell clinic, associate professor in the Division of Hematology and Oncology, and co-director of the UAB Comprehensive Sickle Cell Disease Center at the University of Alabama in Birmingham
Date/Time: Monday, December 11, 2023, 4:30 p.m. PT
BETA-THALASSEMIA DATA
Poster Presentation [#1102]: Sustained, Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with betibeglogene autotemcel (beti-cel)
Presenting Author: Alexis A. Thompson, M.D., M.P.H., professor of pediatrics (hematology), Perelman School of Medicine, University of Pennsylvania, Philadelphia, and chief, Division of Hematology, Children’s Hospital of Philadelphia
Date/Time: Saturday, December 9, 2023, 5:30 p.m. PT
Poster Presentation [#2480]: Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with betibeglogene autotemcel (beti-cel) Gene Therapy: Up to 9 Years of Follow-up
Presenting Author: Janet L. Kwiatkowski, M.D., MSCE, professor of pediatrics (hematology), Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, and director, Thalassemia Center, Children’s Hospital of Philadelphia
Date/Time: Sunday, December 10, 2023, 6:00 p.m. PT
Abstracts outlining bluebird bio’s accepted data at ASH (Free ASH Whitepaper) 2023 are available on the ASH (Free ASH Whitepaper) conference website.

UPCOMING ANTICIPATED MILESTONES

For lovo-cel, the FDA has set a PDUFA goal date for December 20, 2023, and if approved, the Company anticipates commercial launch in early 2024. bluebird estimates approximately 20,000 individuals living with SCD (or one-fifth of the U.S. SCD population) may be eligible for gene therapy.
The Company is on track to scale to 40-50 activated QTCs by the end of 2023. bluebird’s QTC network is designed to maximize its commercial opportunity in beta-thalassemia and to prioritize proximity to individuals with living with SCD in anticipation of a 2024 commercial launch for lovo-cel, if approved by the FDA.
For SKYSONA, the Company continues to anticipate 5-10 patient starts this year as previously guided.
THIRD QUARTER 2023 FINANCIAL RESULTS

Cash Position: The Company’s cash, cash equivalents, marketable securities and restricted cash balance was approximately $227 million, as of September 30, 2023. bluebird anticipates full-year 2023 net cash burn to be in the range of $270-$300 million, as previously guided. Based on current operating plans, bluebird expects its cash, cash equivalents and marketable securities including anticipated cash flows from operations, and excluding $53 million of restricted cash, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the second quarter of 2024.
Revenue, net: Total revenue, net was $12.4 million for the three months ended September 30, 2023, compared to $0.1 million for the three months ended September 30, 2022. The increase of $12.3 million was primarily due to SKYSONA and ZYNTEGLO product revenue.
SG&A Expenses: Selling, general and administrative expenses were $40.7 million for the three months ended September 30, 2023, compared to $33.4 million for the three months ended September 30, 2022. SG&A includes lease expense related to 50 Binney Street; however, the associated sublease income is presented in other income (expense), net. Excluding the lease expense for 50 Binney St., SG&A expenses were $31.1 million for the three months ended September 30, 2023, compared to $24.1 million for the three months ended September 30, 2022. This increase is mainly attributable to commercial costs driven by marketing activities for ZYNTEGLO and SKYSONA in the United States and the performance of commercial readiness activities in the United States for lovo-cel, in anticipation of potential approval, as well as increased employee compensation, benefit and other headcount-related expenses, professional fees, and information technology and facility related costs in 2023. These increased costs were partially offset by decreased consulting fees.
R&D Expenses: Research and development expenses were $45.5 million for the three months ended September 30, 2023, compared to $53.1 million for the three months ended September 30, 2022. The decrease of $7.6 million was primarily due to manufacturing costs related to SKYSONA and ZYNTEGLO now being included in inventory and cost of product revenue, as well as decreased employee compensation, benefit and other headcount-related expenses, license and milestone fees, and information technology and facility related costs in 2023. These decreased costs were partially offset by increased clinical costs, lab expenses, and consulting fees.
Net income (loss): Net loss was $71.7 million for the three months ended September 30, 2023, compared to a net loss of $76.5 million for the three months ended September 30, 2022.
CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss third quarter financial results and commercial launch progress on Tuesday, November 7 at 8:00 am ET.

To access the call via telephone, please register at this link https://register.vevent.com/register/BIee363f414c2f4fd19da314c2b328d635 to receive a dial in number and unique PIN to access the live conference call.

The live webcast of the call may be accessed by visiting the "Events & Presentations" page within the Investors & Media section of the bluebird website at View Source A replay of the webcast will be available on the bluebird website for 90 days following the event.

On November 7, 2023 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with genetically defined cancers, reported that its President and Chief Executive Officer, Mark A. Velleca, M.D., Ph.D., will participate in fireside chats during two upcoming investor conferences (Press release, Black Diamond Therapeutics, NOV 7, 2023, View Source [SID1234637123]):

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The Stifel 2023 Healthcare Conference at 2:25pm ET on Tuesday, November 14, 2023, in New York, NY.
The Piper Sandler 35th Annual Healthcare Conference at 4:30pm ET on Wednesday, November 29, 2023, in New York, NY.

Webcasts will be available at the start of the presentations on the investor relations section of the Company’s website, www.blackdiamondtherapeutics.com. Replays of the presentations will also be available and archived on the site for 90 days.

On November 7, 2023 Biodesix, Inc. (Nasdaq: BDSX), a leading diagnostic solutions company with a focus in lung disease, reported its financial and operating results for the third quarter ended September 30, 2023 and provided a corporate update (Press release, Biodesix, NOV 7, 2023, View Source [SID1234637122]).

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"I am pleased to announce yet another impressive quarter of growth at Biodesix," said Scott Hutton, President and Chief Executive Officer. "The team delivered over 10,400 Lung Diagnostics tests, growing 60% over the 6,500 tests delivered in the third quarter of 2022, the fifth straight quarter of at least 60% year-over-year growth, and 6% improvement over second quarter 2023. Our Biopharma Services business also had a strong quarter, growing 79% year-over-year and 181% over the second quarter. I am thrilled with Biodesix’s ability to consistently grow the business while maintaining a strong cost disciplined approach. Starting this quarter, we are now providing Adjusted EBITDA to provide additional insight into our progress toward profitability. Our third quarter Adjusted EBITDA was ($5.4) million, a 40% improvement over both the third quarter of 2022 and the second quarter of 2023. In the fourth quarter we are continuing to improve our balance sheet with the final closing of our private placement announced in August and plan to draw down an additional $10 million from the second tranche of our term loan with Perceptive Advisors. I am exceptionally pleased with our progress as we continue to increase test volumes, drive revenue growth, advance operational effectiveness, and improve Adjusted EBITDA as we strive to impact more patients with lung disease than ever before."

Third Quarter 2023 Financial Results

For the three-month period ended September 30, 2023, as compared to the same period of 2022 (where applicable):

Total revenue of $13.5 million, an increase of 21%, including COVID testing revenue in third quarter 2022, driven primarily by strong year-over-year growth in lung diagnostics, and a 37% year-over-year increase excluding COVID testing revenues from the prior year comparison;
Lung diagnostic revenue of $12.3 million reflected a year-over-year increase of 34% driven primarily by the continued adoption of Nodify Lung nodule risk assessment tests, and approximately 60% when excluding one-time cash revenue from tests performed in prior periods primarily from Medicare coverage of the Nodify CDT test;
Biopharma Services and other revenue of $1.2 million increased 79% year-over-year, a result of both delivering against our increasing book of business and new agreements;
COVID-19 testing revenue decreased by 100% year-over-year, as the Company no longer provides COVID-19 diagnostic testing services;
Third quarter 2023 gross profit of $10.3 million, or 76% gross margin compared to 67% gross margin in the comparable prior year period, primarily driven by growth in Lung Diagnostic Testing and optimization of testing workflows that resulted in improvements in costs per test, the ongoing recovery of our Biopharma Services business, and the commercial discontinuation of our lower-margin COVID-19 diagnostic testing;
Operating expenses (excluding direct costs and expenses) of $17.4 million, a decrease of approximately $0.7 million, or 4% as compared to the third quarter 2022 (includes non-cash stock compensation expense of $1.0 million as compared to $1.2 million). This decrease is primarily attributable to a decrease in research and development costs, partially offset by increased sales and marketing costs to support lung diagnostic sales growth to enhance product awareness and drive adoption;
Net loss of $10.9 million, a decrease of approximately $2.8 million, or 20%;
Adjusted EBITDA was a loss of $5.4 million, an improvement of $3.6 million, or 40%;
Cash and cash equivalents of $19.8 million as of September 30, 2023, an increase of $2.4 million from June 30, 2023;
The Company plans to draw down an additional $10 million from the second tranche of its $50 million term loan facility with Perceptive Advisors in the fourth quarter of 2023, subject to the terms and conditions of such facility;
Cash balance includes $15.3 million of the $27.5 million private placement announced in August. The remaining $12.2 million will be received in the fourth quarter of 2023.
2023 Financial Outlook

Third quarter revenues were impacted by a change in the timing and assumptions regarding the anticipated collection of revenue from a backlog of previously unrecognized Medicare Advantage claims. Based on these new assumptions, the Company is adjusting its 2023 financial outlook to remove the backlog and is not making any change to core business expectations. The Company expects to generate $50 to $52 million in total revenue in 2023.

Conference call and webcast information

Listeners can register for the webcast via this link. Analysts wishing to participate in the question-and-answer session should use this link. A replay of the webcast will be available via the Company’s investor website approximately two hours after the call’s conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.

For a full list of Biodesix’s press releases and webinars, please visit biodesix.com.