On August 12, 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 second quarter 2024, recent business highlights, and key upcoming milestones for 2024 (Press release, Atara Biotherapeutics, AUG 12, 2024, View Source [SID1234645722]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Building on the recent BLA acceptance with Priority Review for tab-cel, we are making significant progress with the agency towards the target action date of January 15, 2025, while supporting our partner Pierre Fabre with their U.S. launch preparation," said Pascal Touchon, President and Chief Executive Officer of Atara. "During the quarter we continued to advance the clinical development of our lead CAR T program, ATA3219, and remain on track to deliver key value creating milestones within the next 12 months. This is highlighted by initial data from our non-Hodgkin’s lymphoma study anticipated in the first quarter of 2025, which we believe will provide a read-through for ATA3219’s potential in autoimmune disease. On that front, we plan to initiate our ATA3219 Systemic Lupus Erythematosus trial in the fourth quarter, including the cohort without lymphodepletion, with initial data expected in mid-2025."

Dr. Touchon continued, "Following the landmark milestone of the world’s first-ever approval of an allogeneic T-cell therapy and with the potential first U.S. approval approaching, we are advancing our differentiated allogeneic CAR-T programs into the clinic. With the Company in a strong position, I have decided to move into the role of Chairman for personal reasons to dedicate more time to my family. I look forward to having a very active and strategic advisory role as board chair and continuing to help Atara create value with the potential tab-cel U.S. approval and initial clinical data with ATA3219. I believe the future of Atara is bright under the leadership of Cokey Nguyen, Ph.D. who will be promoted to the role of President and CEO. Cokey is a visionary leader in the cell therapy field, and our Board of Directors values his deep commitment to our staff and to patients as well as his expertise across the breadth of our business."

"I admire the strong foundation we built under Pascal’s leadership. I am honored to serve as Atara’s CEO at this pivotal time to continue our journey to get tab-cel approved in the U.S. and to unlock the disruptive potential of our allogeneic CAR-T platform," said Cokey Nguyen, Ph.D. "I look forward to working alongside our world-class and innovative teams to rapidly innovate and strive to develop better cell therapy treatment options for patients."

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

●
U.S. Food and Drug Administration (FDA) accepted the filing of Atara’s Biologics License Application (BLA) for tabelecleucel (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 BLA has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of January 15, 2025
●
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 submission is supported by 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
●
Atara received a $20 million milestone payment from Pierre Fabre Laboratories in August 2024, following the acceptance of the tab-cel BLA, with the potential to receive a $60 million milestone payment from Pierre Fabre contingent upon FDA approval of the tab-cel BLA

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 mid-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 subjects
●
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 was presented in poster presentation at the International Society for Cell & Gene Therapy meeting. The data demonstrated 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 presented in the poster showed that ATA3219 CAR T cells, which incorporate the next-generation 1XX costimulatory domain, released lower levels of pro-inflammatory cytokines while maintaining cytotoxic function and potency in response to stimulation with CD19+ target cells when compared to autologous CAR T controls. 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 mid-2025
o
Eligible subjects with severe SLE will receive ATA3219 at a dose of 40, 80, or 240 × 106 CAR+ T cells
•
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 continues opening sites and initiating 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 Q1 2025
●
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

Leadership Updates

•
Effective September 9, 2024:
o
Cokey Nguyen, Ph.D., the Company’s current Chief Scientific and Technical Officer, will be promoted to the role of President and CEO and Pascal Touchon, the Company’s current President and CEO, will transition to the role of Chairman of the Board of Directors
o
Cokey Nguyen, Ph.D., will be appointed to the Company’s Board of Directors
o
Carol Gallagher, Pharm.D., current Chair of the Board, will transition to become Independent Lead Director

Second Quarter 2024 Financial Results

●
Cash, cash equivalents and short-term investments as of June 30, 2024 totaled $35.3 million, as compared to $46.2 million as of March 31, 2024
●
Q2 2024 accounts receivable totaled $2.4 million; however, this amount does not include the $20 million milestone payment owed by Pierre Fabre related to the tab-cel BLA acceptance, which was received in August 2024
●
On August 9, 2024, Pierre Fabre and Atara entered into an agreement for Pierre Fabre to purchase certain existing tab-cel intermediate inventory from Atara for $15.5 million, which is expected to be received from Pierre Fabre in September 2024
●
Together, cash, cash equivalents, short-term investments, accounts receivable as of June 30, 2024, the $20 million tab-cel BLA acceptance milestone payment, and the $15.5 million tab-cel intermediate inventory purchase amount total $73.2 million
●
Net cash used in operating activities was $10.6 million for the second quarter 2024, as compared to $52.8 million in the same period in 2023
○
Q2 2024 net cash used in operating activities included a $20 million cash payment received from Pierre Fabre for a milestone payment achieved in March 2024, whereas Q2 2023 had no such cash receipts

●
Total revenues were $28.6 million for the second quarter 2024, as compared to $1.0 million for the same period in 2023. Total revenues increased by $27.6 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 $7.7 million for the second quarter 2024, as compared to $13.8 million for the same period in 2023
●
Research and development expenses were $33.3 million for the second quarter 2024, as compared to $56.1 million for the same period in 2023
○
Research and development expenses include $3.3 million of non-cash stock-based compensation expenses for the second quarter 2024, as compared to $7.2 million for the same period in 2023
●
General and administrative expenses were $8.9 million for the second quarter 2024, as compared to $13.3 million for the same period in 2023
○
General and administrative expenses include $3.0 million of non-cash stock-based compensation expenses for the second quarter 2024, as compared to $5.4 million for the same period in 2023
●
Atara reported net losses of $19.0 million, or $3.10 per share, for the second quarter 2024, as compared to $71.1 million, or $16.91 per share, for the same period in 2023

2024
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 began in Q2 2024 and is expected to continue for the remainder of the year
●
Atara expects that cash, cash equivalents, short-term investments, and accounts receivable as of June 30, 2024, plus the items noted below, in total will enable funding of planned operations into 2027:
○
$20 million milestone payment for the acceptance of the tab-cel BLA received from Pierre Fabre in August 2024 and $60 million contingent upon the approval of the tab-cel BLA;
○
$15.5 million purchase by Pierre Fabre of tab-cel intermediate inventory to be received in September 2024 and additional 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. 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 August 12, 2024 Syros Pharmaceuticals (NASDAQ:SYRS), a biopharmaceutical company committed to advancing new standards of care for the frontline treatment of hematologic malignancies, reported that it will discontinue enrollment in the SELECT-AML-1 Phase 2 clinical trial evaluating the triplet regimen of tamibarotene in combination with venetoclax and azacitidine compared to the doublet regimen of venetoclax and azacitidine in newly diagnosed, unfit patients with acute myeloid leukemia (AML) and RARA gene overexpression (Press release, Syros Pharmaceuticals, AUG 12, 2024, View Source [SID1234645738]). This decision is based on the results of a prespecified interim analysis of the trial.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Data from 51 patients enrolled in SELECT-AML-1 were reviewed on August 9, 2024. This review included a prespecified non-binding futility analysis conducted on the first 40 randomized patients after the fortieth randomized patient received approximately three months of study drug or discontinued treatment. A similar complete response (CR)/complete response with incomplete hematologic recovery (CRi) rate was observed in the triplet arm (n=20; 65%, CI: 40.8-84.6) and the doublet arm (n=20; 70%, CI: 45.7-88.1). As a result, the probability for success of the SELECT-AML-1 study to demonstrate superiority at the final analysis in 80 randomized patients was considered low, and Syros made the decision to discontinue further enrollment. There were no new safety signals associated with the use of tamibarotene in combination with venetoclax and azacitidine. Patients currently enrolled in SELECT-AML-1 will have the opportunity to remain on study at the discretion of study investigators. Syros plans to present data from SELECT-AML-1 at the 12th Annual Meeting of the Society of Hematologic Oncology (SOHO) in September 2024.

"We are disappointed by this unexpected outcome, especially for people living with AML," said David A. Roth, M.D., Chief Medical Officer of Syros. "In our prior Phase 2 clinical trial, the doublet combination of tamibarotene and azacitidine delivered a 61% CR/CRi rate in newly diagnosed AML patients with RARA overexpression. This supports our conviction in pursuing a doublet strategy in higher-risk MDS, where we are comparing tamibarotene and azacitidine to azacitidine alone. We remain steadfast in our commitment to delivering tamibarotene for the treatment of HR-MDS and look forward to sharing pivotal data from SELECT-MDS-1 by mid-fourth quarter."

Syros continues to evaluate tamibarotene, an oral, selective, retinoic acid receptor alpha (RARα) agonist, in combination with azacitidine in the SELECT-MDS-1 Phase 3 clinical trial in newly diagnosed higher-risk myelodysplastic syndrome (MDS) patients with RARA gene overexpression. The SELECT-MDS-1 trial passed a prespecified futility analysis in the first quarter of 2024 and is continuing as planned, with pivotal CR data expected by the middle of the fourth quarter of 2024.

On August 12, 2024 Lisata Therapeutics, Inc. (Nasdaq: LSTA) ("Lisata" or the "Company"), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, reported business update and announced financial results for the second quarter ended June 30, 2024 (Press release, Lisata Therapeutics, AUG 12, 2024, View Source [SID1234645723]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The second quarter generated strong momentum for Lisata as we continued to advance multiple ongoing and planned clinical studies centered around our novel investigational product, certepetide," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Lisata. "We have a lot to look forward to with multiple key data readouts projected over the next 18 months, including topline results from the Phase 2b ASCEND trial. These results have transformative potential for the Company as we plan to explore conditional approvals with various regulatory agencies and/or to design an optimized Phase 3 program in metastatic pancreatic ductal adenocarcinoma, an aggressive, often fatal, form of pancreatic cancer. In just the first half of 2024, certepetide has received U.S. FDA Orphan Drug and Rare Pediatric Disease designation in osteosarcoma, and a waiver for evaluating certepetide in a pediatric population with pancreatic cancer in Europe (EMA). These agency recognitions further validate and support our excitement and the broad therapeutic potential of this innovative therapy."

Dr. Mazzo added, "Our continued prudent, strategic financial management allows us to reaffirm our projection that available cash will fund current operations into early 2026, providing the necessary capital for all planned trials through to completion."

Development Portfolio Highlights

Certepetide as a treatment for solid tumors in combination with other anti-cancer agents

Certepetide (formerly LSTA1) is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to penetrate solid tumors more effectively. Certepetide actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor. Certepetide has also been shown to modify the tumor microenvironment, diminishing its immunosuppressive nature and inhibiting the metastatic cascade. Along with our collaborators, we have amassed significant non-clinical data demonstrating enhanced delivery of various existing and emerging anti-cancer therapies, including immunotherapies and RNA-based therapeutics. To date, certepetide has also demonstrated favorable safety, tolerability, and clinical activity in completed and ongoing clinical trials designed to test its ability to enhance the effectiveness of standard-of-care ("SoC") chemotherapy for pancreatic cancer. Lisata is exploring the potential of certepetide to enable a variety of treatment modalities to treat a range of solid tumors more effectively. Certepetide has been awarded Fast Track designation (U.S.) and Orphan Drug Designation for pancreatic cancer (U.S. and E.U.) as well as Orphan Drug Designation for glioma (U.S.) and osteosarcoma (U.S.). Additionally, certepetide has received Rare Pediatric Disease Designation for osteosarcoma (U.S.). Currently, certepetide is the subject of multiple ongoing or planned Phase 2a and 2b clinical studies being conducted globally in a variety of solid tumor types in combination with a variety of anti-cancer regimens, including:

•ASCEND: Phase 2b double-blind, randomized, placebo-controlled clinical trial evaluating two dosing regimens of certepetide in combination with SoC chemotherapy (gemcitabine/nab-paclitaxel) in patients with metastatic pancreatic ductal adenocarcinoma ("mPDAC"). Cohort A of the study receives a single dose of 3.2 mg/kg certepetide essentially simultaneously with SoC, while Cohort B is identical to Cohort A, but with a second dose of 3.2mg/kg of certepetide given four hours after the first. The trial is being conducted at 25 sites in Australia and New Zealand led by the Australasian Gastro-Intestinal Trials Group in collaboration with the University of Sydney and with the National Health and Medical Research Council Clinical Trial Centre at the University of Sydney as the Coordinating Centre. The conclusion of a planned interim futility analysis in 2023 by the Independent Data Safety Monitoring Committee was that the conditions for futility were not met and that the study should proceed to completion. With trial enrollment completed in the fourth quarter of 2023, Lisata expects topline data from the 95 patients assigned to Cohort A of the study to be reported in the fourth quarter of 2024 and the complete data set of all 158 patients from the study to be available by mid-2025.

•BOLSTER: Phase 2a double-blind, placebo-controlled, multi-center, randomized trial in the U.S. evaluating certepetide in combination with SoC in first- and second-line cholangiocarcinoma ("CCA"). The Company achieved complete enrollment in first-line CCA nearly six months ahead of plan, accelerating anticipated topline data readout to mid-2025. Based on this rapid enrollment rate and the pressing need to improve treatment outcomes in patients that have progressed after first-line CCA treatment, a second cohort has been added to the BOLSTER trial evaluating subjects in second-line CCA. Lisata expects to enroll the first patient by the fourth quarter of 2024.
•CENDIFOX: Phase 1b/2a open-label trial in the U.S. of certepetide in combination with neoadjuvant FOLFIRINOX based therapies in pancreatic, colon and appendiceal cancers. The trial has completed enrollment in the pancreatic cohort and expects to complete enrollment in the remaining two cohorts by the end of 2024.
•Qilu Pharmaceutical, the licensee of certepetide in the Greater China territory, is currently evaluating certepetide in combination with gemcitabine and nab-paclitaxel as a treatment for mPDAC. During the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting, Qilu Pharmaceutical presented an abstract sharing preliminary data from the study which corroborated previously reported findings from the Phase 1b/2a trial of certepetide plus gemcitabine and nab-paclitaxel conducted in Australia in patients with mPDAC. As previously reported, Qilu has begun treating patients in their Phase 2 placebo-controlled trial in mPDAC.
•iLSTA: Phase 1b/2a randomized, single-blind, single-center, safety and pharmacodynamic trial in Australia evaluating certepetide in combination with the checkpoint inhibitor, durvalumab, plus SoC gemcitabine and nab-paclitaxel chemotherapy versus SoC alone in patients with locally advanced non-resectable PDAC. Enrollment completion is expected in the second half of 2024.

•A Lisata-funded Phase 2a, double-blind, placebo-controlled, randomized, proof-of-concept study evaluating certepetide in combination with SoC temozolomide versus temozolomide alone in patients with newly diagnosed GBM is being conducted across multiple sites in Estonia and Latvia and is targeted to enroll 30 patients with a randomization of 2:1 in favor of the certepetide treatment group.

•FORTIFIDE: Phase 1b/2a, double-blind, placebo-controlled, three-arm, randomized study in the U.S. to evaluate the safety, tolerability, and efficacy of a 4-hour continuous infusion of certepetide in combination with SoC in subjects with second-line mPDAC who have progressed on FOLFIRINOX. As part of this study, Lisata has engaged Haystack Oncology to use its MRD technology to measure circulating tumor DNA levels at multiple timepoints in patients throughout the study as an exploratory endpoint for analyzing the early therapeutic effect of certepetide. The Company expects to enroll the first patient in the study by the first half of 2025.

Second Quarter 2024 Financial Highlights

For the three months ended June 30, 2024, operating expenses totaled $5.5 million, compared to $6.9 million for the three months ended June 30, 2023, representing a decrease of $1.4 million or 19.7%.
Research and development expenses were approximately $2.6 million for the three months ended June 30, 2024, compared to $3.2 million for the three months ended June 30, 2023, representing a decrease of $0.6 million or 17.7%. This was primarily due to a reduction in expenses associated with the Phase 2b ASCEND trial which completed enrollment in the prior year, lower spend on chemistry, manufacturing and control ("CMC") related expenses and lower equity expense partially offset by an increase in expenses associated with our enrollment activities in the current year for our BOLSTER trial.
General and administrative expenses were approximately $2.9 million for the three months ended June 30, 2024, compared to $3.7 million for the three months ended June 30, 2023, representing a decrease of $0.8 million or 21.3%. This was primarily due to one-off related severance costs in the prior year associated with the elimination of the Chief Business Officer position on May 1, 2023, a reduction in equity expense and a decrease in directors and officers insurance premiums in the current year.
Benefit from income taxes was $0.0 million for the three months ended June 30, 2024, compared to $2.3 million for the three months ended June 30, 2023. In April 2023, we received net proceeds of $2.2 million from the sale of tax benefits to a qualified and approved buyer pursuant to the New Jersey Economic Development Authority’s Technology Business Tax Certificate Transfer Program.
Overall, net losses were $5.0 million for the three months ended June 30, 2024, compared to $4.0 million for the three months ended June 30, 2023.

Balance Sheet Highlights

As of June 30, 2024, Lisata had cash, cash equivalents, and marketable securities of approximately $38.3 million. Based on its current expected capital needs, the Company believes that its projected capital will fund its current proposed operations into early 2026, encompassing anticipated data milestones from all its ongoing and planned clinical trials.

Conference Call Information
Lisata will hold a live conference call today, August 12, 2024, at 4:30 p.m. Eastern Time to discuss financial results, provide a business update and answer questions.
Those wishing to participate must register for the conference call by way of the following link: CLICK HERE TO REGISTER. Registered participants will receive an email containing conference call details with dial-in options. To avoid delays, we encourage participants to dial into the conference call 15 minutes ahead of the scheduled start time.
A live webcast of the call will also be accessible under the Investors & News section of Lisata’s website and will be available for replay beginning two hours after the conclusion of the call for 12 months.

On August 12, 2024 Theralase Technologies Inc. ("Theralase" or the "Company") (TSXV: TLT) (OTCQB: TLTFF), a clinical stage pharmaceutical company dedicated to the research and development of light and/or radiation activated small molecules for the safe and effective destruction of various cancers, bacteria and viruses reported the Company’s unaudited condensed consolidated interim financial statements for the six-month period ended June 30, 2024. ("Financial Statements") (Press release, Theralase, AUG 12, 2024, View Source [SID1234645739]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Theralase will be hosting a conference call on Wednesday August 21st, 2024 at 11:00 am ET, which will include a presentation of the financial and operational results for the six-month period ended June 30, 2024. Questions are welcome. To ensure Theralase has time to review and properly address them during the call, please send them in advance to [email protected].

Zoom Meeting Link: View Source

Conference Call in: 1-647-558-0588 (Canada) / 1-646-558-8656 (US) – not required for those attending by Zoom.

An archived version will be available on the website following the conference call.

Financial Summary:

For the six-month period ended June 30th:

1 Other represents foreign exchange, interest accretion on lease liabilities and / or interest income

Financial Highlights

For the six-month period ended June 30, 2024;

Total revenue decreased 35%, year over year.
Cost of sales was $186,324 (67% of revenue) resulting in a gross margin of $90,077 (33% of revenue). In comparison, the cost of sales for the same period in 2023 was $224,947 (53% of revenue) resulting in a gross margin of $201,140 (47% of revenue). The gross margin decrease, as a percentage of sales year over year, is attributed to an increase in material costs.
$145,915, from $147,304 for the same period in 2023, a 1% decrease.
The decrease is a result of reduced spending on general and administrative expenses (59%) and stock-based compensation (28%) (due to the cumulative effect of accounting for the vesting of stock options granted in the current and previous years).
for the Drug Division decreased to $1,368,333 from $1,594,676 for the same period in 2023, a 10% decrease. The decrease is primarily attributed to a decrease in costs for Study II patient enrollment and treatment.
The increase is attributed to development of a new software program for the TLC-2000 Cool Laser Therapy system.
Net loss was $2,400,461, which included $374,445 of net non-cash expenses (i.e.: amortization, stock-based compensation expense and foreign exchange gain/loss). This compared to a net loss in 2023 of $2,564,187, a 6% year-over-year reduction, which included $474,558 of net non-cash expenses. The Drug Division represented $1,938,024 of this loss (81%). The decrease in net loss is primarily attributed to decreased spending on research and development expenses in Study II.
Operational Highlights:

Non-Brokered Private Placement:

On February 5, 2024, the Company closed a non-brokered private placement of units. On closing, the Company issued an aggregate of 6,666,670 units at a price of $CAN 0.18 per Unit for aggregate gross proceeds of approximately $CAN 1,200,000 of which 1,310,502 units were purchased by certain insiders of the Corporation, representing gross proceeds of $235,890. Each Unit consisted of one common share of the Company and one non-transferable warrant. Each Warrant entitles the holder to acquire an additional Common Share at a price of $CAN 0.25 for a period of 5 years following the date of issuance.

On April 24, 2024, the Company closed a non-brokered private placement of units. On closing, the Company issued an aggregate of 4,167,778 units at a price of $0.18 per Unit for aggregate gross proceeds of approximately $750,200. Each Unit consisted of one common share of the Company and one non-transferable common share purchase warrant. Each Warrant entitles the holder to acquire an additional Common Share at a price of $0.25 for a period of 5 years following the date of issuance.

On July 8, 2024, the Company closed a non-brokered private placement of units. On closing, the Company issued an aggregate of 3,522,729 units at a price of $0.22 per Unit for aggregate gross proceeds of approximately $775,000. Each Unit consisted of one common share of the Company and one non-transferable common share purchase warrant. Each Warrant entitles the holder to acquire an additional Common Share at a price of $0.30 for a period of 5 years following the date of issuance.

In 2024, the Company plans to secure funding through various equity and debt instruments to allow the Company the ability to become base shelf eligible. This will allow the Company sufficient funding to complete enrollment into Study II by year end, data lock in mid 2026 and position the Company for FDA and Health Canada approval by the end of 2026, subject to achieving FDA Priority Review."

Study II Update:

On February 8th, 2024, Dr. Michael Jewett joined the Company in the role of an independent consultant, to assist the Company in the accruement of patients into Study II. Under the terms of the consulting agreement, Dr. Jewett will be responsible for working with existing clinical study sites and helping to onboard new clinical study sites to assist Theralase to complete enrollment and provide the primary study treatment to 75 to 100 patients in Study II, preferably by December 31, 2024.

To date, Theralase has enrolled and treated 72 patients in Study II, who have been provided the primary Study II Procedure. The clinical study sites have screened an additional 3 patients, who they are planning to enroll and treat over the next 4 to 6 weeks, bringing the total to 75 treated patients.

Theralase plans to add up to 5 new CSSs in 2024, as well as increase enrollment at the existing 10 Clinical Study Sites ("CSSs") to complete Study II accruement by the end of 2024 / beginning of 2025.

90% (65/72) of treated patients have been evaluated at the 90 days assessment visit for treatment safety and efficacy according to the clinical study protocol.

For the primary endpoint of Study II (Complete Response ("CR") at any point in time) 63% (41/65) of treated patients achieved a CR.

For the secondary endpoint of Study II (duration of CR) 44% (18/41) of treated patients, who achieved a CR, maintained their CR response for at least 12 months.

For the tertiary endpoint of Study II (safety of Study Procedure) 100% (65/65) experienced no Serious Adverse Events ("SAEs") directly related to the Study Drug or Study Device.

Break Through Designation Update:

In 2020, the FDA granted Theralase Fast Track Designation ("FTD") for Study II. As a Fast Track designee, Theralase has access to early and frequent communications with the FDA to discuss Theralase’s development plans and ensure the timely collection of clinical data to support the approval process. The accelerated communication with the FDA potentially allows, the Study Procedure, to be the first intravesical, patient-specific, light-activated, Ruthenium-based small molecule for the treatment of patients diagnosed with BCG-Unresponsive NMIBC CIS, (with or without recurrent / resected papillary Ta/T1 tumours). FTD can also lead to Break Through Designation ("BTD"), Accelerated Approval ("AA") and/or Priority Review, if certain criteria are met, which the FDA previously defined to the Company for BTD as clinical data on approximately 20 to 25 patients enrolled and provided the primary Study Procedure, who demonstrate significant safety and efficacy clinical outcomes.

To this list, the FDA has added: Post Study II Monitoring of Response and Central Pathology Laboratory Review.

The Company is currently working with the CSSs, a biostatistics organization and a regulatory organization to update the pre-BTD submission with clinical data clarifications, identified by the FDA. The Company plans to resubmit the pre-BTD submission to the FDA in 3Q2024 for FDA review of these clarifications. Once the pre-BTD submission has been accepted by the FDA, the Company plans to compile a BTD submission for review by the FDA in 3Q2024 in support of the grant of a BTD approval.

Theralase has commenced receiving clinical data from the CSSs with a significant number of patients, who achieved CR, continuing to experience a duration of their CR beyond 450 days, with some patients demonstrating CR for up to 3 years and counting, post the primary Study Procedure.

Study II Preliminary Clinical Data:

Performance to Primary, Secondary and Tertiary Objectives:

The interim clinical data above demonstrates that:

For the primary objective, 63% of patients provided the Study Procedure (Study Drug activated by the Study Device) demonstrated a Complete Response ("CR") (negative cystoscopy and negative urine cytology, among other definitions). Including patients, who demonstrated an Indeterminate Response ("IR") (negative cystoscopy and positive or suspicious urine cytology), the Total Response ("TR") increases to 71%. This represents almost 3 out of 4 Bacillus Calmette Guérin ("BCG")-Unresponsive Non-Muscle Invasive Bladder Cancer ("NMIBC") Carcinoma In-Situ ("CIS") patients treated with Theralase’s unique Study Procedure are demonstrating complete destruction of their CIS bladder cancer within their bladders.

For the secondary objective, 44% (almost 1 out of 2) patients, who demonstrated a CR at any point in time continued to demonstrate a CR at 15 months from date of first treatment with 46% of patients demonstrating a TR.

> 90% of patients who demonstrated a CR at 450 days continue to demonstrate this response beyond 450 days.

For the tertiary objective, no patients have been diagnosed with a Serious Adverse Event ("SAE") directly related to the Study Drug or Study Device 100% (65/65).

Note:

For patients to be included in the statistical clinical analysis they must be enrolled in Study II, provided the primary Study Procedure and evaluated by a Principal Investigator ("PI") at the 90 days assessment visit (cystoscopy and urine cytology)
One patient passed away prior to their 90 days assessment and is therefore not included in the efficacy statistical analysis, only in the safety statistical analysis; therefore, there are 65 patients that have been statistically analyzed for efficacy.
Evaluable Patients are defined as patients who have been evaluated by a PI and thus excludes a patient’s clinical data at specific assessment days, if that clinical data is pending.
7 patients have been enrolled and provided the primary Study Procedure but, have not been evaluated at their 90 day assessment; therefore, 65 patients are considered Evaluable Patients at 90 days, with 41 patients considered Evaluable Patients at 450 days.
The data analysis presented above, should be read with caution, as the clinical data is interim in its presentation, as Study II is ongoing and new clinical data collected may or may not continue to support the current trends, with clinical data still pending.
Patient Response Chart:

The Swimmer’s plot below is a graphical representation of the interim clinical results (n=41) for patients who achieved a CR at any point in time and their response over 1080 days, graphically demonstrating a patient’s response to a treatment over time. As can be seen in the plot, clinical data is still pending for patients, who have demonstrated an initial CR at 90 days and continue to demonstrate a duration of that response.

The Swimmer’s Plot illustrates:

63% (41/65) Evaluable Patients achieved CR at any point in time, with 44% (18/41) patients, who demonstrated CR, continuing to demonstrate CR at 450 days and thus achieving the primary and secondary objectives of Study II.
41% (17/41) Evaluable Patients demonstrate CR beyond 450 days.
Note: This is interim clinical data and clinical data is still being collected, but all indications demonstrate that the study has achieved its primary, secondary and tertiary objectives.

Kaplan-Meier Curve:

The Kaplan-Meier ("KM") Curve illustrates graphically, for patients who have achieved a CR, the duration of CR and probability of that CR continuing in the future.

Note: The information on the time-to-outcome event is not available for all patients in this analysis, as not all patients have been assessed at all available assessment visits. Only patients that achieved the primary objective (CR at any point in time) have been analyzed and data is plotted relative to the date at which their first CR was observed. The "X" denotes censored observations (subjects who achieved CR at their last assessment visit and are currently on-study or have been removed from study). Thus, the KM Curve estimates the risk of a patient failing to maintain a CR over time, according to currently available interim data.

In summary, the interim clinical data demonstrates that patients consenting to participate in Study II have a 63% chance of achieving CR.

If CR is obtained, then the patient has a 48.3%, 42.3% and 33.8% chance of remaining cancer free for 1, 2 and 3 years, respectively.

Serious Adverse Events

For 72 patients treated in Study II, there have been 14 Serious Adverse Events ("SAEs") reported:

3 – Grade 2 (resolved within 1, 1 and unknown days, respectively)
7 – Grade 3 (resolved within 1, 2, 3, 4, 4, 82 and unknown days, respectively)
3 – Grade 4 (resolved within 3, 6 and 8 days, respectively)
1 – Grade 5
Theralase believes all SAEs reported to date are unrelated to the Study II Drug or Study II Device.

Note: A SAE is defined as any untoward medical occurrence that at any dose: Is serious or life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or results in death.

Dr. Arkady Mandel, M.D., Ph.D., D.Sc., Chief Scientific Officer of Theralase stated, "The interim clinical data of Study II, to date, has proven to be world-class. Study II has demonstrated an ability to destroy urothelial cell carcinoma in a patient’s bladder for a Total Response ("TR") of 71% and a duration of that TR of 46%, at 450 days. The primary benefits of the Theralase technology versus competitive technologies are: a urologist-led treatment, a single out-patient procedure, high efficacy rates (patients achieve a CR in 63% of the cases with a 44% duration of that CR at 450 days), high probability of the ongoing duration of that complete response (34% ≥ 3 years, based on the Kaplan-Meier Curve analysis of the interim clinical data) and high safety profile (no SAEs directly associated with the Study Drug or Study Device); therefore, the Theralase technology presents a safe, effective alternative therapy for patients, who are at high risk of having their bladder removed."

Roger DuMoulin-White, B.E.Sc., P.Eng., Pro.Dir., President and Chief Executive Officer of Theralase stated, "Based on the interim clinical data accumulated to date, Study II has achieved its primary, secondary and tertiary endpoints and requires only a few additional patients enrolled and follow-up on all patients to complete the study. Theralase expects to complete patient follow-up by mid 2026 with review by Health Canada and the FDA on a marketing approval by end of 2026. The Theralase bladder cancer treatment has been proven clinically to be safe and effective in the treatment of BCG-Unresponsive NMIBC CIS, fulfilling an unmet need of the medical community."

About Study II:

Study II utilizes the therapeutic dose of the patented Study II Drug ("RuvidarTM" or "TLD-1433") (0.70 mg/cm2) activated by the proprietary Study II Device (TLC-3200 Medical Laser System or "TLC-3200"). Study II is focused on enrolling and treating approximately 75 to 100 BCG-Unresponsive NMIBC Carcinoma In-Situ ("CIS") patients in up to 15 Clinical Study Sites ("CSS") located in Canada and the United States.

About RuvidarTM:

RuvidarTM is a peer reviewed, patented PDC currently under investigation in Study II.

On August 12, 2024 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the second quarter ended June 30, 2024, and recent corporate updates (Press release, Checkpoint Therapeutics, AUG 12, 2024, View Source [SID1234645724]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "We’ve made significant recent progress as we seek approval of cosibelimab as a potential new treatment for patients with metastatic or locally advanced cutaneous squamous cell carcinoma (‘’cSCC’’) who are not candidates for curative surgery or curative radiation. We are pleased to have reached alignment with the U.S. Food and Drug Administration (‘‘FDA’’) on our strategy to potentially address the deficiencies identified in the complete response letter (‘‘CRL’’) received last December. Shortly thereafter, we resubmitted our Biologics License Application ("BLA"), which was accepted by the FDA for review as a complete response to the CRL. We look forward to working with the FDA in advance of the Prescription Drug User Fee Act (‘‘PDUFA’’) goal date of December 28, 2024, to potentially deliver this important therapeutic option to cutaneous squamous cell carcinoma patients and their families."

Recent Corporate Updates:

· Checkpoint submitted a BLA to the FDA in January 2023 seeking approval of cosibelimab as a potential new treatment for patients with metastatic or locally advanced cSCC who are not candidates for curative surgery or curative radiation. In December 2023, the FDA issued a CRL for the cosibelimab BLA. The CRL only cited findings that arose during a multi-sponsor inspection of Checkpoint’s third-party contract manufacturing organization ("CMO") as approvability issues to address in a BLA resubmission. The CRL did not state any concerns about the clinical data package, safety, or labeling for the approvability of cosibelimab.

· In June 2024, Checkpoint reached alignment with the FDA on its BLA resubmission strategy for cosibelimab and resubmitted the BLA shortly thereafter.

· In July 2024, Checkpoint announced that the FDA accepted for review the resubmission of its BLA for cosibelimab as a complete response to the CRL issued in December 2023 and set a PDUFA goal date of December 28, 2024.

· Also in July 2024, Checkpoint announced a collaboration to explore the combined therapeutic potential of cosibelimab, its anti-PD-L1 antibody with dual mechanism of action, with GC Cell’s Immuncell-LC, an innovative autologous Cytokine Induced Killer T cell therapy composed of cytotoxic T lymphocytes and natural killer T cells.

· Also in July 2024, Checkpoint completed a registered direct offering priced At-the-Market under Nasdaq rules and a concurrent private placement of warrants to purchase Checkpoint common stock, for total gross proceeds of approximately $12.0 million.

Financial Results:

· Cash Position: As of June 30, 2024, Checkpoint’s cash and cash equivalents totaled $5.0 million, compared to $11.2 million at March 31, 2024 and $4.9 million at December 31, 2023, a decrease of $6.2 million for the quarter and an increase of $0.1 million, year-to-date. After the end of the second quarter, Checkpoint raised gross proceeds of approximately $12.0 million in a registered direct offering completed in July 2024.

· R&D Expenses: Research and development expenses for the second quarter of 2024 were $4.5 million, compared to $13.9 million for the second quarter of 2023, a decrease of $9.4 million. Research and development expenses for the second quarter of 2024 included $0.6 million of non-cash stock expenses, compared to $0.2 million for the second quarter of 2023.

· G&A Expenses: General and administrative expenses for the second quarter of 2024 were $2.2 million, compared to $2.3 million for the second quarter of 2023, a decrease of $0.1 million. General and administrative expenses for the second quarter of 2024 included $0.6 million of non-cash stock expenses, compared to $0.8 million for the second quarter of 2023.

· Net Loss: Net loss attributable to common stockholders for the second quarter of 2024 was $6.7 million, or $0.18 per share, compared to a net loss of $16.5 million, or $1.05 per share, in the second quarter of 2023. Net loss for the second quarter of 2024 included $1.2 million of non-cash stock expenses, compared to $1.0 million for the second quarter of 2023.