On May 9, 2024 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported first quarter results and business highlights for the quarter ended March 31, 2024, including recent commercial and operational progress (Press release, bluebird bio, MAY 9, 2024, View Source [SID1234642980]).

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"bluebird has built a solid commercial gene therapy foundation, with an unparalleled network of qualified treatment centers (QTCs), proven access and reimbursement for patients, and demonstrated demand from both patients and providers," said Andrew Obenshain, chief executive officer, bluebird bio. "Following the completion of the first LYFGENIA patient start earlier this month, and with the continued momentum behind our ongoing launches, we believe we are poised for accelerated growth through the remainder of 2024."

COMMERCIAL LAUNCH UPDATES

Continued commercial momentum for LYFGENIA (lovotibeglogene autotemcel), ZYNTEGLO (betibeglogene autotemcel) and SKYSONA (elivaldogene autotemcel)

First LYFGENIA patient start completed in May 2024.
14 patient starts completed for ZYNTEGLO and SKYSONA since the beginning of 2024 (11 ZYNTEGLO, 3 SKYSONA).
Validated access and reimbursement strategy is driving favorable coverage landscape

Successfully confirmed prior authorization approval for commercial and Medicaid-insured patients for LYFGENIA.
Multiple outcomes-based agreements in place for LYFGENIA with national commercial payer organizations; published coverage policies are in place for more than 200 million U.S. lives.
Discussions ongoing with Medicaid agencies representing 80% of Medicaid-insured individuals with sickle cell disease in the U.S.
Timely access to ZYNTEGLO and SKYSONA has continued, with zero ultimate denials for either therapy across both Medicaid and commercial payers.
Substantial QTC footprint established

bluebird has activated 64 QTCs for LYFGENIA and ZYNTEGLO (defined as a signed MSA).
Six centers are also activated to administer SKYSONA for patients with cerebral adrenoleukodystrophy (CALD).
2024 GUIDANCE

The Company anticipates 85 to 105 patient starts (cell collections) combined across all three of its FDA approved therapies (LYFGENIA, ZYNTEGLO, SKYSONA) in 2024. Consistent with previous quarters, bluebird plans to provide quarterly updates on patient starts for each of its therapies.
Gross-to-net discounts across all three products are expected to be in the range of 20% to 25% of gross revenue in 2024 and will fluctuate based on product and payer mix, as well as utilization of outcomes-based agreements for LYFGENIA and ZYNTEGLO.
Based on projected timelines from cell collection to infusion, the Company anticipates recognizing revenue from its first infusion of LYFGENIA in the third quarter of 2024.
FIRST QUARTER FINANCIAL HIGHLIGHTS

Cash Position: The Company’s cash, cash equivalents and restricted cash balance was approximately $264 million, including restricted cash of approximately $52 million, as of March 31, 2024

Based on current business plans, which assumes the Company’s ability to achieve certain commercial revenue targets, bluebird expects its cash and cash equivalents, excluding restricted cash and assuming the remaining two tranches totaling $50 million in proceeds from its term loan facility with Hercules Capital are executed, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements through Q1 2026.

Revenue, net: Total revenue, net was $18.6 million for the three months ended March 31, 2024, compared to $2.4 million for the three months ended March 31, 2023. The increase of $16.2 million was due to increased ZYNTEGLO product revenue.
On March 26, 2024, bluebird announced that it will restate its consolidated financial statements as of and for the year ended December 31, 2022, and for each of the first three quarters of 2022 and 2023 in its Annual Report on Form 10-K for the year ended December 31, 2023 (the "2023 Form 10-K"). The restatements relate to the identification of embedded leases and the treatment of non-lease components contained in lease agreements. The restatement is not expected to impact the Company’s cash position or revenue. As a result of the restatement, the Company is delayed in filing its 2023 Form 10-K and its Quarterly Report on Form 10-Q for the quarter ended March 31, 2024 (the "Q1 2024 Form 10-Q"). The Company is continuing to work expeditiously to complete these filings.

The financial results included in this press release represent the most current information available to the Company’s management. The Company expects that its actual results to be reported in its Q1 2024 Form 10-Q will not differ materially from the results included herein, however, these results are subject to change following the completion of the Company’s financial close procedures and the review of its consolidated financial statements for the quarter ended March 31, 2024.

CONFERENCE CALL DETAILS

bluebird will hold a conference call to discuss its first quarter 2024 results and business updates today, Thursday, May 9, 2024, at 8:00 am ET.

To participate in the conference call, please dial +1 (800) 715-9871 (U.S. and Canada) and ask to be joined into the bluebird call or provide the Conference ID 9768329.

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 May 9, 2024 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer, reported financial results for the first quarter ended March 31, 2024, and provided a corporate update (Press release, Black Diamond Therapeutics, MAY 9, 2024, View Source [SID1234642979]).

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"We reached an important inflection in BDTX-1535 development this past quarter, as we initiated dosing of patients with non-classical EGFR mutant NSCLC in the first-line setting, and are on track to release initial results from the ongoing Phase 2 second/third-line cohorts in the third quarter of this year ", said Mark Velleca, M.D., Ph.D., Chief Executive Officer of Black Diamond Therapeutics. "Our recent AACR (Free AACR Whitepaper) presentation on the evolving EGFR mutation landscape highlights the major unmet need in EGFR mutant NSCLC, and the differentiated BDTX-1535 profile as the potential first- and best-in-class fourth-generation oral TKI in clinical development addressing the full spectrum of classical, non-classical, and C797S resistance mutations in NSCLC."

Recent Developments & Upcoming Milestones:
BDTX-1535:
•Following receipt of End of Phase 1 feedback from the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2023, Black Diamond initiated dosing of a Phase 2 first-line cohort in patients with non-classical epidermal growth factor receptor mutation positive (EGFRm) non-small cell lung cancer (NSCLC) during the first quarter of 2024. (NCT05256290)
•In April 2024, Black Diamond described real world evidence of the evolving EGFR mutation landscape in patients with NSCLC and the potential of BDTX-1535 to address a broader range of mutations compared to existing therapies at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting. The oral presentation titled "BDTX-1535 – A MasterKey EGFR Inhibitor Targeting Classical, Non-Classical and the C797S Resistance Mutation to Address the Evolved Landscape of EGFR Mutant NSCLC," evaluated more than 235,000 sequenced cases of NSCLC sourced from Guardant Health (GuardantINFORM) and Foundation Medicine (FoundationInsights). The analyses revealed a broad spectrum of non-classical mutations, as well as an increased prevalence of the acquired resistance mutation, C797S. These non-classical EGFR mutations were present in 20-30% of newly diagnosed EGFRm NSCLC patients.

•Black Diamond anticipates the following upcoming key milestones for BDTX-1535:
◦Poster presentations at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 1, 2024, of Phase 1 dose escalation data in patients with relapsed/recurrent GBM, and initial intratumoral pharmacokinetic results from a "window of opportunity" (also known as a Phase 0/1 "Trigger") trial sponsored by the Ivy Brain Tumor Center, in patients with recurrent high-grade glioma (HGG).
◦Disclosure of initial Phase 2 data in 2L/3L EGFRm NSCLC patients with non-classical mutations or the acquired resistance C797S mutation remains on track for Q3 2024.
BDTX-4933:
•Enrollment of patients with BRAF and select RAS/MAPK mutation-positive cancers, with an emphasis on patients with KRAS mutant NSCLC is ongoing in a Phase 1 dose escalation trial of BDTX-4933, a brain-penetrant oral inhibitor of oncogenic alterations in KRAS, NRAS and BRAF (NCT05786924). An update from this trial is anticipated in Q4 2024.
Corporate
•In April 2024, the Company announced changes to its Board of Directors. Industry veterans Shannon Campbell and Prakash Raman, Ph.D., have been appointed to the Company’s Board of Directors. In addition, Wendy Dixon, Ph.D., and Alex Mayweg, Ph.D., have stepped down as members of the Board of Directors.
Financial Highlights
•Cash Position: Black Diamond ended the first quarter of 2024 with approximately $115.2 million in cash, cash equivalents, and investments compared to $131.4 million as of December 31, 2023. Net cash used in operations was $21.2 million for the first quarter of 2024 compared to $20.0 million for the first quarter of 2023.
•Research and Development Expenses: Research and development (R&D) expenses were $13.5 million for the first quarter of 2024, compared to $14.8 million for the same period in 2023. The decrease in R&D expenses was primarily due to reduced spending on early discovery projects.
•General and Administrative Expenses: General and administrative (G&A) expenses were $6.7 million for the first quarter of 2024, compared to $6.8 million for the same period in 2023. The decrease in G&A expenses was primarily due to a decrease in personnel related costs and legal and other professional fees.
•Net Loss: Net loss for the first quarter of 2024 was $18.2 million, as compared to $20.9 million for the same period in 2023.
Financial Guidance
•Black Diamond ended the first quarter of 2024 with approximately $115.2 million in cash, cash equivalents and investments which the Company believes is sufficient to fund its anticipated operating expenses and capital expenditure requirements into the third quarter of 2025.

On May 9, 2024 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the first quarter of 2024 (Press release, BioXcel, MAY 9, 2024, View Source [SID1234642978]).

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"The fundamentals of our business are strong as we look to continue advancing and expanding our agitation portfolio," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "We have designed two Phase 3 registrational programs for BXCL501 in addition to strengthening our balance sheet and intellectual property. We are intensely focused on the BXCL501 journey into the at-home setting and expansion into Alzheimer’s-related agitation with the goal of bringing new treatment options to larger numbers of patients while expanding the market potential of our lead neuroscience asset."

TRANQUILITY and SERENITY Clinical Programs

· Plans for two late-stage programs are advancing following recently announced designs of pivotal Phase 3 trials:

o TRANQUILITY In-Care trial: designed to evaluate the efficacy and safety of a 60 mcg dose of BXCL501 for agitation associated with Alzheimer’s dementia (AAD).

o SERENITY At-Home* safety trial: designed to evaluate the safety of a 120 mcg dose of BXCL501 in the at-home setting for agitation associated with bipolar disorders or schizophrenia.

· Study protocol submitted to FDA.

IGALMI Post-Marketing Requirement (PMR) Study

· Study was designed to evaluate whether tolerance, tachyphylaxis, or withdrawal occur following repeat dosing of IGALMI following seven days of repeated treatment.

o Completed enrollment of approximately 20 patients with frequent episodes of agitation for bipolar disorders or schizophrenia in an open-label study.

o Patients self-administered 180 mcg of IGALMI for repeated agitation episodes over the treatment period.

o Initiated data cleaning to enable database lock.

Corporate Updates

IGALMI Commercialization

· Net revenue grew 55% in Q1 2024 over the prior quarter driven largely by volume contracting, new customer acquisition, increased utilization among existing customers, and the permanent J-Code for IGALMI that became effective January 1, 2024.

Patent Portfolio

The Company continues to strengthen its intellectual property portfolio with over 30 granted or allowed patents and more than 140 additional patent applications in prosecution as of April 2024.

· Recently granted two new patents for BXCL501, in Japan and the U.S., with patent protection to 2039 and 2043, respectively.

· Eight currently listed U.S. patents for IGALMI in the United States Food and Drug Administration’s ("FDA") Approved Drug Products with Therapeutic Equivalence Evaluations (commonly known as the "Orange Book"), with two additional recently allowed patents that will be submitted for listing in the Orange Book once issued by the USPTO.

OnkosXcel Therapeutics

· Announced late-breaking abstract selected for presentation at 2024 ASCO (Free ASCO Whitepaper) Annual Meeting on preliminary findings from a Phase 2 investigator-sponsored trial of BXCL701 and KEYTRUDA (pembrolizumab) in metastatic pancreatic ductal adenocarcinoma (PDAC).

First Quarter 2024 Financial Results

Net Revenue: Net revenue from IGALMI was $582,000 for the first quarter of 2024, compared to $206,000 for the same period in 2023, representing a 182% increase. Sequential quarterly revenue increased 55% in Q1 2024 from the fourth quarter of 2023. The increased revenue for both periods was primarily attributable to increasing demand with existing customers, new customer orders, and volume-based contracting.

Research and Development (R&D) Expenses: R&D expenses were $11.4 million for the first quarter of 2024, compared to $27.8 million for the same period in 2023. The decreased expenses were primarily attributable to the wind-down of the SERENITY III and TRANQUILITY II and III trials, as well as decreased professional fees, personnel, and related costs.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $13.3 million for the first quarter of 2024, compared to $23.6 million for the same period in 2023. The reduced expenses were primarily attributable to a decrease in personnel and costs associated with the commercialization of IGALMI compared to the first quarter of 2023. The reduced expenses were partially offset by increased professional fees in the first quarter of 2024.

Net Loss: BioXcel Therapeutics had a net loss of $26.8 million for the first quarter of 2024, compared to a net loss of $52.8 million for the same period in 2023. The Company used $17.7 million in operating cash during the first quarter of 2024.

Cash and cash equivalents totaled $74.1 million as of March 31, 2024. This includes the $25 million from the registered direct offering announced on March 25, 2024.

Conference Call and Webcast

BioXcel Therapeutics will host a conference call and webcast today, May 9, 2024, at 8:00 a.m. ET to discuss its first quarter 2024 financial results. To access the call, please dial 877-407-5795 or 201-689-8722. A live webcast will be available on the Investors section of the corporate website, bioxceltherapeutics.com, and a replay will be available through August 9, 2024.

BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may sign up to automatically receive email alerts and other information about the Company by visiting the "Email Alerts" option under the News/Events section of the Investors & Media website section and submitting your email address.

*SERENITY At-Home represents the redesigned SERENITY III trial.

About IGALMI (dexmedetomidine) sublingual film

INDICATION

IGALMI (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, that is placed under the tongue or behind the lower lip and is used for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults. The safety and effectiveness of IGALMI has not been studied beyond 24 hours from the first dose. It is not known if IGALMI is safe and effective in children.

IMPORTANT SAFETY INFORMATION

IGALMI can cause serious side effects, including:

· Decreased blood pressure, low blood pressure upon standing, and slower than normal heart rate, which may be more likely in patients with low blood volume, diabetes, chronic high blood pressure, and older patients. IGALMI is taken under the supervision of a healthcare provider who will monitor vital signs (like blood pressure and heart rate) and alertness after IGALMI is administered to help prevent falling or fainting. Patients should be adequately hydrated and sit or lie down after taking IGALMI and instructed to tell their healthcare provider if they feel dizzy, lightheaded, or faint.

· Heart rhythm changes (QT interval prolongation). IGALMI should not be given to patients with an abnormal heart rhythm, a history of an irregular heartbeat, slow heart rate, low potassium, low magnesium, or taking other drugs that could affect heart rhythm. Taking IGALMI with a history of abnormal heart rhythm can increase the risk of torsades de pointes and sudden death. Patients should be instructed to tell their healthcare provider immediately if they feel faint or have heart palpitations.

· Sleepiness/drowsiness. Patients should not perform activities requiring mental alertness, such as driving or operating hazardous machinery, for at least 8 hours after taking IGALMI.

· Withdrawal reactions, tolerance, and decreased response/efficacy. IGALMI was not studied for longer than 24 hours after the first dose. Physical dependence, withdrawal symptoms (e.g., nausea, vomiting, agitation), and decreased response to IGALMI may occur if IGALMI is used longer than 24 hours.

The most common side effects of IGALMI in clinical studies were sleepiness or drowsiness, a prickling or tingling sensation or numbness of the mouth, dizziness, dry mouth, low blood pressure, and low blood pressure upon standing.

These are not all the possible side effects of IGALMI. Patients should speak with their healthcare provider for medical advice about side effects.

Patients should tell their healthcare provider about their medical history, including if they suffer from any known heart problems, low potassium, low magnesium, low blood pressure, low heart rate, diabetes, high blood pressure, history of fainting, or liver impairment. They should also tell their healthcare provider if they are pregnant or breastfeeding or take any medicines, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Patients should especially tell their healthcare provider if they take any drugs that lower blood pressure, change heart rate, or take anesthetics, sedatives, hypnotics, and opioids.

Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201- 1088 or [email protected].

Please see full Prescribing Information at igalmi.com.

About BXCL501

In indications other than those approved by the U.S. Food and Drug Administration (FDA) as IGALMI (dexmedetomidine) sublingual film, BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. BioXcel Therapeutics believes that BXCL501 potentially targets an important mediator of agitation, and the Company has observed anti-agitation results in multiple clinical trials across several neuropsychiatric disorders. BXCL501 is under investigation by BioXcel Therapeutics for the acute treatment of agitation associated with dementia due to probable Alzheimer’s disease and for the acute treatment of agitation associated with bipolar I or II disorder or schizophrenia in the at-home setting. The safety and efficacy of BXCL501 for these investigational uses have not been established. BXCL501 has been granted Breakthrough Therapy designation by the FDA for the acute treatment of agitation associated with dementia and Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders, and dementia.

About BXCL701

BXCL701 is an investigational, oral innate immune activator designed to initiate inflammation in the tumor microenvironment. Approved and experimental immunotherapies often fail to address cancers that appear "cold." Therefore, BXCL701 is being evaluated to determine if it can render "cold" tumors "hot," making them more detectable by the adaptive immune system and thereby facilitating the development of a strong anticancer immune response. OnkosXcel Therapeutics’ preclinical data support BXCL701’s potential synergy with both current checkpoint inhibitors and emerging immunotherapies directed to activate T-cells. BXCL701 is currently being developed as a potential therapy for the treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors. BXCL701 has received Orphan Drug Designation from the U.S. Food and Drug Administration in four indications: acute myelogenous leukemia, pancreatic cancer, stage IIb to IV melanoma, and soft tissue sarcoma. The U.S. Food and Drug Administration (FDA) designated as a Fast Track development program the investigation of BXCL701 in combination with a checkpoint inhibitor for treatment of patients with metastatic small cell neuroendocrine prostate cancer (SCNC) with progression on chemotherapy and no evidence of microsatellite instability. An 800+-subject clinical database, with data collected by the Company and others, supports the ongoing development of BXCL701.

On May 9, 2024 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported financial results for the first quarter 2024, recent business highlights, and key upcoming milestones for 2024 (Press release, Atara Biotherapeutics, MAY 9, 2024, View Source [SID1234642977]).

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"Our lead CAR T program, ATA3219, is advancing as a promising new therapeutic option for oncology and autoimmune diseases, where it is positioned to benefit from the unique characteristics of our proven allogeneic EBV T-cell platform," said Pascal Touchon, President and Chief Executive Officer of Atara. "This is anticipated to provide multiple near-term clinical milestones for ATA3219, including initial non-Hodgkin’s lymphoma data expected in the fourth quarter 2024, and initial lupus nephritis data in the first half of 2025, with plans to expand into a new SLE cohort without lymphodepletion. In addition, our partnership with Pierre Fabre continues to generate value, as we received our first milestone payment related to tab-cel regulatory progress in the U.S. in April, with the potential for additional near-term payments related to the BLA acceptance and approval."

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

Atara plans to submit a Biologics License Application (BLA) in Q2 2024 for tab-cel indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate
The data package for the filing includes pivotal and supportive data covering more than 430 patients treated with tab-cel across multiple life-threatening diseases
The BLA will include the latest pivotal ALLELE study data-cut that demonstrated a statistically significant 48.8% Objective Response Rate (ORR) (p<0.0001) and favorable safety profile consistent with previous analyses
Ebvallo received regulatory approval from Swissmedic, the regulatory authority in Switzerland, further expanding its potential in Europe
Atara continues to advance the Pierre Fabre Laboratories expanded global partnership, which included approximately $27 million in cash upfront at the closing of the deal and the potential to receive up to $640 million in additional payments and significant double-digit tiered royalties on net sales, including up to $100 million in potential regulatory milestones through BLA approval
ATA3219: CD19 Program in Lupus Nephritis (LN)

Atara expects to initiate a Phase 1 study of ATA3219 as a monotherapy for the treatment of systemic lupus erythematosus (SLE) with kidney involvement (lupus nephritis [LN]) in Q4 2024 with initial clinical data anticipated in H1 2025
The Phase 1 open-label, dose-escalation study is designed to evaluate safety, preliminary efficacy, pharmacokinetics, and biomarkers of a single dose of ATA3219 administered to LN subjects refractory to one or more lines of treatment. Subjects will receive lymphodepletion treatment followed by ATA3219 at a dose of 40, 80, or 160 x 106 CAR+ T cells. Each dose level is designed to enroll 3-6 patients
Atara is positioned to potentially expand ATA3219 Phase 1 study into additional autoimmune indications via the same Investigational New Drug (IND) application previously cleared for the LN study
Preclinical data supporting the potential of ATA3219 in SLE will be presented in poster presentation at the International Society for Cell & Gene Therapy meeting, which is being held May 29-June 1, 2024. The data demonstrate that ATA3219 CAR T cells led to complete CD19-specific B-cell depletion against SLE or multiple sclerosis patient peripheral blood mononuclear cells
Additional preclinical data also presented in the poster show that ATA3219 CAR T cells, which use the next-generation 1XX costimulatory domain, release lower levels of pro-inflammatory cytokines while maintaining cytotoxic function as potent as observed with autologous CAR T controls in response to stimulation with CD19+ target cells. Mitigating inflammatory cytokine release that is typically seen with standard CD19 CAR T signaling may lead to reduced toxicity and better tolerability if confirmed in clinical trials
ATA3219: CD19 Program in Severe Systemic Lupus Erythematosus (SLE) Without Lymphodepletion

Atara plans to expand the Phase 1 LN study of ATA3219 and add a new cohort in severe SLE without lymphodepletion (LD) in Q4 2024 with initial clinical data anticipated in H2 2025
The elimination of LD is designed to further simplify the treatment regimen and to potentially provide a differentiated safety profile to patients without comprising efficacy which may improve patient access
There is compelling clinical and scientific rationale supporting the potential to eliminate the need for LD based on the EBV T-cell backbone and unique features of ATA3219, including: 1) low alloreactivity risk and favorable safety in over 600 patients treated without LD, due to T-cell receptor EBV specificity and partial human leukocyte antigen matching; 2) expansion and persistence data without LD correlating to efficacy in patients treated with tab-cel; and 3) the inclusion of clinically validated features into ATA3219 such as the 1XX costimulatory domain and memory phenotype that increase potency and persistence
ATA3219: CD19 Program in Non-Hodgkin’s Lymphoma (NHL)

Atara initiated enrollment of a multi-center, Phase 1 open-label, dose-escalation clinical trial of ATA3219 in NHL, including large B-cell lymphomas, follicular lymphoma, and mantle cell lymphoma, with initial clinical data anticipated in Q4 2024
Study designed to evaluate safety, preliminary efficacy, pharmacokinetics, and biomarkers. Subjects will receive LD treatment followed by ATA3219 at a dose of 40, 80, 240, or 480 x 106 CAR+ T cells. Each dose level is designed to enroll 3-6 patients
Previously presented preclinical data demonstrated superior in vivo persistence and CD19-specific anti-tumor efficacy compared to an autologous CD19 CAR T benchmark with no observed toxicity or alloreactivity
ATA3431: CD19/CD20 Program for B-Cell Malignancies

Preclinical data presented at ASH (Free ASH Whitepaper) 2023 demonstrated early evidence of potent antitumor activity, long-term persistence, and superior tumor growth inhibition compared to an autologous CD19/CD20 CAR T benchmark
Dual CD19 and CD20 targeting designed to address CD19 escape and tumor variability and may provide additional efficacy in lymphoma
Atara is progressing toward an IND submission in H2 2025
First Quarter 2024 Financial Results

Cash, cash equivalents and short-term investments as of March 31, 2024 totaled $46.2 million, as compared to $51.7 million as of December 31, 2023
Q1 2024 accounts receivable totaled $35.8 million and include a $20 million milestone payment related to the positive tab-cel pre-BLA meeting and approximately $12 million for the reimbursement of tab-cel global development costs from Pierre Fabre. The $20 million milestone payment was received in April 2024 and the approximate $12 million payment is expected to be received in May 2024
Together, cash, cash equivalents, short-term investments, and accounts receivable as of March 31, 2024 totaled $82.1 million
Net cash used in operating activities was $29.6 million for the first quarter 2024, as compared to $38.4 million in the same period in 2023
Q1 2024 net cash used in operating activities included 2023 annual employee bonus payments and cash disbursements related to the November 2023 and January 2024 reductions in force of approximately $13 million in aggregate
Total revenues were $27.4 million for the first quarter 2024, as compared to $1.2 million for the same period in 2023. Total revenues increased by $26.2 million year over year, primarily due to revenue recognized as a result of additional obligations for the expanded partnership with Pierre Fabre and accelerated recognition of existing deferred revenue due to the planned transition of substantially all activities relating to tab-cel at the time of BLA approval and transfer to Pierre Fabre
Total costs and operating expenses include non-cash stock-based compensation, depreciation and amortization expenses of $9.8 million for the first quarter 2024, as compared to $13.0 million for the same period in 2023
Total costs and operating expenses include restructuring expense of $4.8 million for the first quarter 2024 related to the reduction in force Atara announced in January 2024, and which reduced its headcount at that time by approximately 25% to 170 employees
Research and development expenses were $45.5 million for the first quarter 2024, as compared to $62.2 million for the same period in 2023
Research and development expenses include $4.7 million of non-cash stock-based compensation expenses for the first quarter 2024, as compared to $6.8 million for the same period in 2023
General and administrative expenses were $11.1 million for the first quarter 2024, as compared to $13.9 million for the same period in 2023
General and administrative expenses include $3.7 million of non-cash stock-based compensation expenses for the first quarter 2024, as compared to $5.0 million for the same period in 2023
Atara reported net losses of $31.8 million, or $0.23 per share, for the first quarter 2024, as compared to $74.8 million, or $0.72 per share, for the same period in 2023
Outlook and Cash Runway

Atara expects full year 2024 operating expenses to decrease by approximately 35% from 2023
The large majority of the year-over-year operating expense reduction is expected to begin in Q2 2024 and continue for the remainder of the year
Atara expects that cash, cash equivalents, short-term investments, and accounts receivable as of March 31, 2024, plus the items noted below, in total will enable funding of planned operations into 2027:
anticipated payments of $20 million and $60 million from Pierre Fabre contingent upon the successful acceptance and approval of the tab-cel BLA, respectively;
anticipated purchases of tab-cel inventory through the manufacturing transfer date by Pierre Fabre;
anticipated reimbursement for tab-cel global development costs through the BLA transfer by Pierre Fabre;
operating efficiencies resulting from completed workforce reductions;
the planned transition of substantially all activities relating to tab-cel at the time of the BLA transfer to Pierre Fabre potentially as early as Q1 2025, which will further reduce quarterly operating expenses; and
anticipated royalties from sales of tab-cel by Pierre Fabre in the U.S. post BLA approval
About ATA3219

ATA3219 combines the natural biology of unedited T cells with the benefits of an allogeneic therapy. It consists of allogeneic Epstein-Barr virus (EBV)-sensitized T cells that express a CD19 CAR construct for the treatment of CD19+ relapsed or refractory B-cell malignancies, including B-cell non-Hodgkin’s lymphoma and B-cell mediated autoimmune diseases including systemic lupus erythematosus (SLE) with kidney involvement (lupus nephritis [LN]). ATA3219 has been optimized to offer a potential best-in-class profile, featuring off-the-shelf availability. It incorporates multiple clinically validated technologies including a modified CD3ζ signaling domain (1XX) that optimizes expansion and mitigates exhaustion, enrichment during manufacturing for a less differentiated phenotype for robust expansion and persistence and retains the endogenous T-cell receptor without gene editing as a key survival signal for T cells contributing to persistence.

About ATA3431

ATA3431 is an allogeneic, bispecific CAR directed against CD20 and CD19, built on Atara’s EBV T-cell platform. The design consists of a tandem CD20-CD19 design, with binders oriented to optimize potency. Dual targets address the limitations of single antigen loss and tumor variability. ATA3431 features a novel 1XX costimulatory domain, memory phenotype, and retained, unedited T-cell receptor. Preclinical data have demonstrated early evidence of antitumor activity, long-term persistence, and superior tumor growth inhibition compared to an autologous CD19/CD20 CAR T benchmark.

Next-Generation Allogeneic CAR T Approach

Atara is focused on applying Epstein-Barr virus (EBV) T-cell biology, featuring experience in over 600 patients treated with allogeneic EBV T cells, and novel chimeric antigen receptor (CAR) technologies to meet the current limitations of autologous and allogeneic CAR therapies head-on by advancing a potential best-in-class CAR T pipeline in oncology and autoimmune disease. Unlike gene-edited approaches aimed at inactivating T-cell receptor (TCR) function to reduce the risk for graft-vs-host disease, Atara’s allogeneic platform maintains expression of the native EBV TCR that promote in vivo functional persistence while also demonstrating inherently low alloreactivity due to their recognition of defined viral antigens and partial human leukocyte antigen (HLA) matching. A molecular toolkit of clinically-validated technologies—including the 1XX costimulatory domain designed for better cell fitness and less exhaustion while maintaining stemness—offers a differentiated approach to addressing significant unmet need with the next generation CAR T.

On May 9, 2024 Arvinas, Inc. (Nasdaq: ARVN) reported that two posters, including updated clinical trial data, for vepdegestrant will be presented at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Breast Cancer Annual Congress held from May 15-17, 2024, in Berlin, Germany (Press release, Arvinas, MAY 9, 2024, View Source [SID1234642976]). Vepdegestrant is a novel investigational PROTAC estrogen receptor (ER) degrader that is being jointly developed by Arvinas and Pfizer for the treatment of patients with early and locally advanced or metastatic ER positive/human epidermal growth factor receptor 2 (HER2) negative (ER+/HER2-) breast cancer.

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Poster session details are as follows:
Date: Thursday, May 16, 2024
Time: 12:00 – 1:00 p.m. CET/ 6:00 – 7:00 a.m. ET
Category: Metastatic Breast Cancer

Poster Title and Number: Vepdegestrant, a PROteolysis TArgeting Chimera (PROTAC) estrogen receptor (ER) degrader, plus palbociclib (palbo) in ER+/human epidermal growth factor receptor 2 (HER2)- advanced breast cancer: updated phase 1b cohort results, Poster 218P
Presentation Type and Abstract Number: Abstract, 453
Poster Title and Number: TACTIVE-K: phase 1b/2 study of vepdegestrant, a PROteolysis TArgeting Chimera (PROTAC) estrogen receptor (ER) degrader, in combination with PF-07220060, a cyclin-dependent kinase (CDK)4 inhibitor, in ER+/human epidermal growth factor receptor 2 (HER2)- advanced breast cancer, Poster 264
Presentation Type and Abstract Number: Trial-in-Progress (TiP), 488
Abstracts are now available via the official ESMO (Free ESMO Whitepaper) Breast Cancer Annual Congress website here.

About Vepdegestrant
Vepdegestrant is an investigational, orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with ER positive (ER+)/human epidermal growth factor receptor 2 (HER2) negative (ER+/HER2-) breast cancer. Vepdegestrant is being developed as a potential monotherapy and as part of combination therapy across multiple treatment settings for ER+/HER2- metastatic breast cancer.

In July 2021, Arvinas announced a global collaboration with Pfizer for the co-development and co-commercialization of vepdegestrant; Arvinas and Pfizer will share worldwide development costs, commercialization expenses, and profits.

The U.S. Food and Drug Administration (FDA) has granted vepdegestrant Fast Track designation as a monotherapy in the treatment of adults with ER+/HER2- locally advanced or metastatic breast cancer previously treated with endocrine-based therapy.