On August 10, 2023 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that patient enrollment has been opened for the investigator-initiated study (IIS) at the University of Pennsylvania, supported by a $2.6 million Orphan Products Development grant award by the U.S. Food and Drug Administration (FDA) (Press release, Soligenix, AUG 10, 2023, View Source [SID1234634216]). The IIS will evaluate the expanded treatment, including up to 12 months of treatment, with HyBryte (synthetic hypericin) in patients with early-stage cutaneous T-cell lymphoma (CTCL). The trial is sponsored by Ellen Kim, MD, Director, Penn Cutaneous Lymphoma Program, Vice Chair of Clinical Operations, Dermatology Department, and Professor of Dermatology at the Hospital of the University of Pennsylvania who was a leading enroller in the recently published positive Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study in the treatment of early-stage CTCL.

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"In the Phase 3 FLASH study, HyBryte was shown to be efficacious with a promising safety profile. CTCL patients are often searching for alternative treatments, with limited options especially for early-stage disease. HyBryte offers a distinct treatment option which patients found extremely useful and continue to specifically request. We look forward to demonstrating the expanded use of HyBryte in a "real world" setting and thank both the FDA and Soligenix for their support of this study," noted Dr. Kim, principal investigator of the IIS, RW-HPN-MF-01.

"We are pleased the FDA is supporting the HyBryte program and giving patients an opportunity to access the therapy in an open-label setting," stated Christopher J. Schaber, President and CEO of Soligenix, Inc. "CTCL is an incredibly difficult to treat orphan disease and remains an area of unmet medical need with a very limited number of safe and effective treatment options. With the demonstration of statistical significance in the primary endpoint and continued improvement in treatment response with extended treatment in our published Phase 3 FLASH study, we are continuing discussions with FDA on the design of a second confirmatory Phase 3 study. This IIS has the potential to augment the expanding safety database for synthetic hypericin, as well as provide further real-world evidence into the practical use of HyBryte once commercially available."

The clinical study RW-HPN-MF-01, "Assessment of Treatment with Visible Light Activated Synthetic Hypericin Ointment in Mycosis Fungoides Patients" is designed as an open-label, multicenter clinical trial enrolling approximately 50 patients at select high enrolling clinical centers that participated in the Phase 3 FLASH study. Patients have the potential to be treated for up to 12 months with twice a week dosing (visible light activation to follow ointment application by 24 ± 6 hours). The study also allows for potential transition to the home-use setting. The primary endpoint for the study will be evaluating the number of treatment successes defined as ≥50% reduction in the cumulative CAILS (Composite Assessment of Index Lesion Severity) score from baseline to end of the treatment.

About HyBryte

HyBryte (research name SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light approximately 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).

The recently published Phase 3 FLASH trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a positive treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte’s mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With very limited systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc. In addition, the FDA awarded an Orphan Products Development grant to support the evaluation of HyBryte for expanded treatment in patients with early-stage CTCL, including in the home use setting. The grant, totaling $2.6 million over 4 years, was awarded to the University of Pennsylvania that was a leading enroller in the Phase 3 FLASH study.

About Cutaneous T-Cell Lymphoma (CTCL)

CTCL is a class of non-Hodgkin’s lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as patches and may progress to raised plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas.

CTCL constitutes a rare group of NHLs, occurring in about 4% of the approximate 700,000 individuals living with the disease. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects over 25,000 individuals in the U.S., with approximately 3,000 new cases seen annually.

On August 10, 2023 Shattuck Labs, Inc. (Shattuck) (NASDAQ: STTK), a clinical-stage biotechnology company pioneering the development of bi-functional fusion proteins as a new class of biologic medicine for the treatment of patients with cancer and autoimmune disease, reported financial results for the quarter ended June 30, 2023, and provided recent business highlights (Press release, Shattuck Labs, AUG 10, 2023, View Source [SID1234634215]).

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"We are pleased with our continued progress in enrollment in our ongoing trials in the second quarter of 2023, and in particular to now have four different expansion cohorts well underway in our AML/HR-MDS and PROC trials," said Taylor Schreiber, M.D., Ph.D., Chief Executive Officer of Shattuck. "We believe that data from our dose-escalation study in PROC presented at ASCO (Free ASCO Whitepaper) demonstrated that SL-172154 may be a differentiated CD47 inhibitor due to the integrated CD40 agonist function, and we look forward to sharing initial combination data across multiple tumors and lines of therapy by the end of the year."

2023 Anticipated Milestones

ARC Platform
SL-172154 (SIRPα-Fc-CD40L)
•Complete enrollment and initial data from the ongoing Phase 1B clinical trial of SL-172154 in combination with liposomal doxorubicin in PROC expected in the fourth quarter of 2023.
•Initial data from the ongoing Phase 1B clinical trial of SL-172154 in combination with mirvetuximab soravtansine in PROC expected in the fourth quarter of 2023.
•Complete dose-escalation data, as monotherapy and in combination with azacitidine, for Phase 1A clinical trial of SL-172154 in primarily relapsed/refractory AML and HR-MDS expected in the fourth quarter of 2023.
•Completion of enrollment in the frontline TP53 mutant AML dose-expansion cohort and frontline HR-MDS dose-expansion cohort from our ongoing Phase 1A/B clinical trial of SL-172154 and initial data expected in the fourth quarter of 2023.
Second Quarter 2023 Recent Business Highlights and Other Recent Developments
ARC Clinical-Stage Pipeline
SL-172154 (SIRPα-Fc-CD40L)

•Presented Complete Dose-Escalation Data from Phase 1A Monotherapy Clinical Trial of SL-172154 in PROC at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting: This open-label, multi-center, dose-escalation clinical trial evaluated the safety, tolerability, pharmacokinetics, anti-tumor activity, and pharmacodynamic effects of SL-172154 administered intravenously in patients with PROC. SL-172154 had near-full CD47 and CD40 target engagement and CD40-dependent pharmacodynamic effects observed at the 3 mg/kg dose. SL-172154 had a favorable safety and tolerability profile across doses. The best response per RECIST 1.1 was stable disease in six (22%) patients.
•Completed Enrollment in Dose-escalation Portion of Phase 1A/B Clinical Trial of SL-172154 in AML and HR-MDS: This trial is evaluating the safety, tolerability, pharmacokinetics, anti-tumor activity, and pharmacodynamic effects of SL-172154 as both monotherapy and in combination with azacitidine. In the dose-escalation portion of this trial, enrollment is complete. We are now enrolling patients in the dose expansion cohorts, evaluating SL-172154 in combination with azacitidine in both frontline HR-MDS patients and in frontline TP53 mutant AML. We expect to complete enrollment for the two expansion cohorts in the fourth quarter of 2023. We expect to share complete data from the dose-escalation portion of the trial and initial data from the frontline expansion cohorts in the fourth quarter of 2023.
•Enrollment Progressing in Phase 1B Clinical Trial of SL-172154 in Combination with Liposomal Doxorubicin in PROC: Enrollment is continuing in this trial, which is evaluating the safety, tolerability, pharmacokinetics, anti-tumor activity, and pharmacodynamic effects of SL-172154, using the selected dose of 3 mg/kg, in combination with liposomal doxorubicin in patients with PROC. We completed enrollment in the safety run in portion of this trial in the second quarter of 2023 and expect to complete enrollment in the expansion cohort and present initial data from the trial in the fourth quarter of 2023.
•Enrollment Progressing in Phase 1B Clinical Trial of SL-172154 in Combination with Mirvetuximab Soravtansine in PROC: This trial is evaluating the safety, pharmacokinetics, pharmacodynamic effects, and preliminary anti-tumor activity of SL-172154 administered in combination with mirvetuximab soravtansine in patients with PROC. Mirvetuximab soravtansine is an antibody-drug conjugate targeting folate receptor alpha (FRα), which provides for both direct tumor cell killing as well as enhanced macrophage phagocytosis through binding with Fc gamma receptors and has received accelerated approval for PROC patients whose tumors are shown to be FRα positive, defined as ≥75%, as determined by the VENTANA FOLR1 (FOLR1-2.1) RxDx Assay. Preclinical studies have shown that both of these killing mechanisms are complementary to the mechanism of SL-172154 by enhancing the activity of macrophages to phagocytose FRα- expressing ovarian cancer cells, and that SL-172154 may broaden the activity of mirvetuximab, particularly in patients with tumors that express lower levels of FRα. We intend to enroll patients with broader FRα expression, including those with "high" (greater than ≥75%), "medium" (≥50% to <75%), and "low" (≥25% to <50%) expression of FRα, as determined by the VENTANA FOLR1 (FOLR1-2.1). We expect to present initial data from the trial in the fourth quarter of 2023.
Gamma Delta T Cell Engager (GADLEN) Platform
GADLEN Preclinical Compounds
•In an initial non-human primate toxicology study, presented at the 2023 Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), we observed dose-dependent B cell depletion following administration of a CD20-directed GADLEN. Subsequently, in the second quarter of 2023, we performed an additional toxicology study in non-human primates that was designed to expand upon the initial non-human primate study. In this second study, the depth and durability of B cell depletion were inferior to that reported in published studies with other B cell depleting agents. We are working to determine the underlying scientific cause for these differences in B cell response, and whether these are due to species differences between humans and cynomolgus macaques, or due to aspects inherent to gamma delta T cell biology. We do not currently plan to file an Investigational New Drug application for any GADLEN compounds until these data are better understood.
Second-Quarter 2023 Financial Results
•Cash and Cash Equivalents and Investments: As of June 30, 2023, cash and cash equivalents and investments were $117.2 million, as compared to $214.2 million as of June 30, 2022.

•Research and Development (R&D) Expenses: R&D expenses were $18.2 million for the quarter ended June 30, 2023, as compared to $23.0 million for the quarter ended June 30, 2022. This decrease was primarily driven by a decrease in expense associated with the manufacture of clinical trial materials to support our ongoing clinical trials.
•General and Administrative (G&A) Expenses: G&A expenses were $4.7 million for the quarter ended June 30, 2023, as compared to $4.7 million for the quarter ended June 30, 2022.
•Net Loss: Net loss was $21.3 million for the quarter ended June 30, 2023, or $0.50 per basic and diluted share, as compared to a net loss of $27.4 million for the quarter ended June 30, 2022, or $0.65 per basic and diluted share.
2023 Financial Guidance
Shattuck believes its cash and cash equivalents and investments will be sufficient to fund its operations through year-end 2024, beyond results from its Phase 1 clinical trials of SL-172154. This cash runway guidance is based on the Company’s current operational plans and excludes any additional capital that may be received, proceeds from business development transactions, and/or additional costs associated with clinical development activities that may be undertaken.

About SL-172154

SL-172154 (SIRPα-Fc-CD40L) is an investigational ARC fusion protein designed to simultaneously inhibit the CD47/SIRPα checkpoint interaction and activate the CD40 costimulatory receptor to bolster an anti-tumor immune response in patients with advanced cancer. Multiple Phase 1 clinical trials are ongoing for patients with PROC (NCT04406623, NCT05483933) and patients with AML and HR-MDS (NCT05275439).

On August 10, 2023 Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a clinical-stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, reported financial results for the quarter ended June 30, 2023, and highlighted recent business updates (Press release, Carisma Therapeutics, AUG 10, 2023, View Source [SID1234634214]).

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"In the second quarter, the Carisma team successfully achieved the important clinical milestone of dosing the first patient in the Phase 1 clinical trial of CT-0508 in combination with KEYTRUDA, which represents the next step in our progression of engineering macrophages for the treatment of solid tumors," said Steven Kelly, President and Chief Executive Officer of Carisma. "We continue to execute on our overall strategic plan and we are excited to use this momentum to reach our upcoming potential value inflection points within our HER2 franchise and across our pipeline. We believe we are well-positioned to drive innovation as the leader in engineered macrophages and deliver value to our stakeholders."

Pipeline Updates

· CT-0508
o Announced the first patient has been dosed in the Company’s Phase I clinical trial that will test the safety and tolerability of the Company’s lead candidate, CT-0508, a human epidermal growth factor receptor 2 (HER2) targeted chimeric antigen receptor macrophage (CAR-M) in combination with Merck’s anti-PD1 therapy KEYTRUDA (pembrolizumab) for the treatment of HER2 overexpressing solid tumors.

· In Vivo Oncology Program (Moderna Collaboration)
o Nominated and initiated research on an additional oncology target under the Moderna research collaboration to develop in-vivo targeted CAR-M therapies. The goal of the program, which now has five nominated targets, is to create novel in vivo CAR-M therapies with an approach that uses Moderna’s mRNA/LNP technology together with Carisma’s CAR-M platform technology.

Recent Business Highlights

· Expanded Leadership Team to support legal and human resources functions. The Company appointed Eric Siegel as General Counsel and Corporate Secretary and Terry Shields as Senior Vice President of Human Resources. Mr. Siegel and Ms. Shields each bring to Carisma more than 25 years of experience in their respective fields.

· Added to the Russell 2000, Russell 3000, and Russell Microcap Indexes. Following the conclusion of the 2023 Russell indexes annual reconstitution, the Company joined three Russell indexes that capture the 4,000 largest U.S. stocks as of April 28, 2023, ranked by total market capitalization.

Anticipated Upcoming Milestones

· Data from Group 2 of Phase 1 clinical trial of CT-0508 expected in the second half of 2023

· Initial data from Phase 1 clinical trial of CT-0508 in combination with KEYTRUDA (pembrolizumab) expected in the second half of 2023

· Submission of Investigational New Drug (IND) application to the U.S. Food and Drug Administration for CT-0525, Carisma’s first anti-HER2 CAR-Monocyte product candidate, expected in the second half of 2023

· Selection of next-generation candidate for CT-1119 expected in the first half of 2024

· Proof-of-concept data for the Company’s initial non-oncology program expected in the first half of 2024

Second Quarter 2023 Financial Results

· Cash, cash equivalents and marketable securities as of June 30, 2023 were $117.1 million, compared to $139.0 million as of March 31, 2023.

· Research & development expenses were $18.5 million for the second quarter of 2023, compared to $14.2 million for the same period in 2022. The increase of $4.3 million was primarily due to a $1.5 million increase in direct costs associated with the preclinical development of CT-0525, a $1.3 million increase in direct costs associated with CT-0508, a $1.0 million increase in personnel costs due to growth in research and development employee headcount, a $0.5 million increase in direct costs related to CT-1119, and a $0.2 million increase in facilities and other expenses due to an increase in rent expense, partially offset by a $0.2 million decrease of other clinical and pre-clinical development expenses associated with tracking CT-0525 and CT-1119 separately.

· General & administrative expenses were $6.0 million for the second quarter of 2023, compared to $2.4 million for the same period in 2022. The increase of $3.6 million was attributable to $1.9 million increase in legal and professional fees in support of our expanding infrastructure and patent portfolio, a $1.5 million increase of higher personnel costs as a result of an increase in headcount, as well as a $0.3 million increase in other expenses due to an increase in travel and other administrative costs, partially offset by a $0.1 million decrease in facilities and supplies due to a decline in office expenditures.

· Net loss was $19.9 million for the second quarter of 2023, compared to net loss of $14.8 for the same period in 2022, primarily due to increased research and development expenses to support CT-0508 and CT-0525 as well as increase in expanding headcount and infrastructure, which was partially offset by Moderna collaboration revenue.

Outlook

Carisma believes that its cash, cash equivalents and marketable securities of $117.1 million as of June 30, 2023, are sufficient to sustain Carisma’s planned operations through the end of 2024.

About CT-0508

CT-0508 is a human epidermal growth factor receptor 2 (HER2) targeted chimeric antigen receptor macrophage (CAR-M). It is being evaluated in a landmark Phase 1 multi-center clinical trial that focuses on patients with recurrent or metastatic HER2-overexpressing solid tumors whose cancers do not have approved HER2-targeted therapies or who do not respond to treatment. Carisma is selecting participants who have tumors of any anatomical origin, but with the commonality of overexpressing the HER2 receptor on the cell surface, which is the target for its CAR-M. The Phase 1 clinical trial is first-of-its-kind, marking the first time that engineered macrophages are being studied in humans. The trial continues to enroll patients at seven clinical sites in the U.S., including (i) the University of Pennsylvania Abramson Cancer Center, (ii) the University of North Carolina Lineberger Comprehensive Cancer Center, (iii) the City of Hope National Medical Center, (iv) the MD Anderson Cancer Center, (v) the Sarah Cannon Cancer Research Institute, (vi) Oregon Health & Science University and (vii) Fred Hutchinson Cancer Center.

On August 10, 2023 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported a business update and highlighted its financial results for the quarter ended June 30, 2023 (Press release, Sellas Life Sciences, AUG 10, 2023, View Source [SID1234634213]).

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"We are pleased with the solid progress across our clinical programs in the first half of the year. We expect to complete enrollment in our Phase 3 REGAL study of galinpepimut-S (GPS) in the second half of this year. In addition to our REGAL study, following a strong safety and efficacy profile for SLS009, our CDK9 inhibitor, as a monotherapy in acute myeloid leukemia (AML) in our Phase 1 study, we are particularly excited about the commencement of the Phase 2a study of SLS009 in combination with azacitidine and venetoclax in relapsed and refractory AML, for which we expect topline data in the fourth quarter of 2023," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "The focus of our clinical programs is to potentially deliver treatments to impact the lives of patients with AML, from tackling active disease to prolonging patients’ lives in the maintenance setting, and we look forward to our upcoming milestones across the AML treatment landscape."

Pipeline Update:

Galinpepimut-S (GPS): Wilms Tumor-1 (WT1) targeting immunotherapeutic

Phase 3 REGAL study in AML:

The interim analysis of the ongoing REGAL global Phase 3 registrational clinical trial of GPS in patients with AML who have achieved complete remission following second-line salvage therapy (CR2 patients) is expected in late 2023 or early 2024.

In April, the Independent Data Monitoring Committee (IDMC) recommended that REGAL continue as planned, following a routine, prespecified risk-benefit assessment of unblinded data from the study. The next routine IDMC meeting is scheduled for the third quarter of 2023.

A trial in progress poster titled "A randomized, open-label study of the efficacy and safety of galinpepimut-S (GPS) maintenance monotherapy compared to investigator’s choice of best available therapy (BAT) in patients with acute myeloid leukemia (AML) who have achieved complete remission (CR) after second-line salvage therapy" was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June.

Phase 1/2 Study in combination with pembrolizumab (Keytruda) in ovarian cancer:

SELLAS plans to present final data at the International Gynecologic Cancer Society Annual Meeting in November 2023.The topline data showed clinical benefit of GPS and anti-PD-1 pembrolizumab combination therapy in WT1 positive relapsed or refractory platinum resistant advanced metastatic ovarian cancer patients.

Phase 1 Study in combination with nivolumab (Opdivo) in MPM:

SELLAS reported topline data from the investigator sponsored open-label Phase 1 trial for GPS combination therapy with checkpoint inhibitor nivolumab (Opdivo) for treatment of malignant pleural mesothelioma (MPM) in patients who were either refractory to or relapsed after at least one line of the standard of care therapy. Safety and efficacy endpoints were met with clinical activity and increased survival observed. Additional immune response data is expected in the fourth quarter of 2023.

SLS009: highly selective CDK9 inhibitor

Phase 1 clinical trial in relapsed/refractory (r/r) hematological malignancies:

The safety evaluation stage of the Phase 1 dose escalation clinical trial of SLS009 was completed for the highest dose cohort of AML patients who relapsed after or were refractory to available antileukemic therapies. No further dose escalations are planned in the AML group, while dose escalation continues in the lymphoma group with the addition of 75 mg and 100 mg once-a-week dose cohorts. The 100 mg cohort is planned to likely be the highest dose level for the lymphoma group. Observed clinical activity in the lymphoma group will be announced after the last dose level is complete and is expected during the third quarter of 2023.

SELLAS reported positive topline data in the second quarter of 2023 for the AML cohort as a monotherapy which supported advancement to a Phase 2a trial in AML with SLS009 in combination with azacitidine and venetoclax (aza/ven).

Phase 2a clinical trial in AML:

A Phase 2a trial was initiated studying SLS009 combination treatment with aza/ven in r/r AML patients who did not respond or stopped responding to venetoclax-based therapies. The Phase 2a clinical trial is an open label, single arm, multi-center study that is designed to evaluate safety, tolerability, and efficacy at two dose levels of SLS009. The first patients were dosed in June and topline data are expected in the fourth quarter of 2023.

Hosted a virtual expert panel discussion:

SELLAS hosted a virtual expert panel discussion on SLS009 in AML in May featuring hematology-oncology specialists Tapan Kadia, MD (The University of Texas MD Anderson Cancer Center), Joshua Zeidner, MD (University of North Carolina Lineberger Comprehensive Cancer Center), and Omer Jamy, MD (O’Neal Comprehensive Cancer Center at the University of Alabama) who discussed the treatment landscape for AML and the potential for SLS009 to address unmet medical needs for patients with relapsed and/or refractory AML. A replay of the event is available on SELLAS’ website.

Corporate Updates:

SELLAS expects to receive milestone payments totaling $13.0 million from 3D Medicines by the end of the third quarter of 2023.

Financial Results for the Second Quarter 2023:

R&D Expenses: Research and development expenses for the second quarter of 2023 were $5.9 million, compared to $5.5 million for the same period in 2022. Research and development expenses for the first half of 2023 were $13.1 million, compared to $10.1 million for the same period in 2022.The increase was primarily due to an increase in clinical trial expenses related to the ongoing Phase 3 REGAL clinical trial of GPS in AML patients and the ongoing Phase 2a and Phase 1 clinical trials of SLS009 in hematological malignancies, increased clinical and regulatory consulting, and personnel-related expenses due to increased headcount.

G&A Expenses: General and administrative expenses for the second quarter of 2023 were $3.1 million, as compared to $3.1 million for the same period in 2022. General and administrative expenses for the first half of 2023 were $7.2 million, as compared to $6.1 million for the same period in 2022. The increase was primarily due to personnel-related expenses due to increased headcount.

Acquired In-Process Research and Development: There was no acquired in-process research and development for the first half of 2023 and second quarter of 2022. Acquired in-process research and development was $10.0 million for the first half of 2022, resulting from the in-licensing of SLS009.

Net Loss: Net loss was $8.8 million for the second quarter of 2023, or a basic and diluted loss per share of $0.31, compared to a net loss of $8.4 million for the same period in 2022, or a basic and diluted loss per share of $0.41. Net loss was $19.9 million for the first half of 2023, or a basic and diluted loss per share of $0.77, compared to a net loss of $25.2 million for the same period in 2022, or a basic and diluted loss per share of $1.39.

Cash Position: As of June 30, 2023, cash and cash equivalents totaled approximately $13.8 million.

Keytruda is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA and is not a trademark of SELLAS. Opdivo is a registered trademark of Bristol-Myers Squibb Company, New York, NY, USA and is not a trademark of SELLAS. The manufacturers of these brands are not affiliated with and do not endorse SELLAS or its products.

On August 10, 2023 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company using protein inhibition and protein degradation to develop cancer therapies for patients in need of new treatment options, reported financial results for the three and six months ended June 30, 2023 and provided a business update (Press release, Salarius Pharmaceuticals, AUG 10, 2023, View Source [SID1234634212]).

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Financial and Business Highlights

•Cash and cash equivalents were $11.5 million as of June 30, 2023, compared with $12.1 million as of December 31, 2022
•Net loss for the second quarter of 2023 was $3.9 million, or $1.43 per share, compared with net loss for the second quarter of 2022 of $4.7 million, or $2.20 per share, reflecting lower spending on seclidemstat and lower general and administrative expenses
•Raised gross proceeds of $6.0 million from a private placement of common stock and warrants, and an additional $1.7 million from an At the Market facility
•Announced plans to explore strategic alternatives and implement measures to extend resources

"While the second quarter and recent weeks were highlighted by significant advancements in both of our development programs, after a review of each program’s future funding needs and the current financial markets, the Board of Directors has made the difficult decision to limit further drug development while we explore strategic alternatives for Salarius," said David Arthur, president and chief executive officer of Salarius Pharmaceuticals.

"It was an exceptionally difficult decision to initiate our cost-savings plans and explore strategic alternatives in light of the promising early seclidemstat Ewing sarcoma clinical data, seclidemstat hematological clinical data and the recent FDA clearance to begin the SP-3164 Phase 1 trial. Unfortunately, we believe the current public financial markets make it extremely challenging to raise sufficient capital to continue meaningful clinical development activities on our own," concluded Mr. Arthur.

The Company is conducting a comprehensive review of strategic alternatives focused on maximizing shareholder value including, but not limited to, an acquisition, merger, reverse merger, divestiture of assets, licensing or other strategic transactions involving the Company. However, there is no set timetable for this process and there can be no assurance that this process will result in the Company pursuing a transaction or that any transaction, if pursued, will be completed on attractive terms. If the Company is unable to complete a transaction it may be necessary to seek other alternatives for restructuring and resolving its liabilities, including an orderly wind-down. Salarius does not expect to disclose developments with respect to this process unless and until the evaluation of strategic alternatives has been completed or the Board of Directors has concluded that disclosure is appropriate or legally required.

In connection with the evaluation of strategic alternatives and in order to extend its resources, Salarius is implementing a cost-savings plan that includes a reduction in workforce by over 50% of its positions, with remaining employees focusing primarily on limited drug-development activities, completing the U.S. Food and Drug Administration (FDA) process to determine the clinical trial registration

requirements for the seclidemstat Ewing sarcoma program and supporting the exploration of strategic alternatives.

Second Quarter Financial Results

Research and development expenses declined to $2.4 million for the second quarter of 2023 from $2.9 million for the second quarter of 2022, primarily due to lower spending on seclidemstat offset by higher spending on SP-3164. Spending associated with seclidemstat and SP-3164 for the second quarter of 2023 was $1.1 million and $1.3 million, respectively, compared with $2.1 million and $0.8 million, respectively, for the second quarter of 2022. General and administrative expenses were $1.6 million for the second quarter of 2023, compared with $1.8 million for the second quarter of 2022, with the decline due to lower annual shareholder meeting expenses and overall compensation and benefit costs.

Year-to-Date Financial Results

Research and development expenses declined to $6.1 million for the first half of 2023 from $7.4 million for the prior-year period, primarily due to lower spending on seclidemstat offset by higher spending on SP-3164. Spending associated with seclidemstat and SP-3164 for the first half of 2023 was $2.4 million and $3.7 million, respectively, compared with $4.4 million and $3.0 million, respectively, for the prior-year period.

Net cash used for operating activities during the first half of 2023 was $7.6 million, an increase of $0.4 million from the same period a year ago. The increase is primarily due to a decrease in accounts payable.

As of June 30, 2023, Salarius had cash, cash equivalents and restricted cash of $11.5 million, compared with $12.1 million as of December 31, 2022. Current cash and cash equivalents are expected to fund the company’s planned operations through the fourth quarter of 2023 and enable the evaluation and implementation of strategic alternatives.

Seclidemstat Highlights

•The FDA removed its partial clinical hold on Salarius’ Phase 1/2 trial evaluating seclidemstat in combination with topotecan and cyclophosphamide as a potential treatment for patients with Ewing sarcoma
•The Company initiated the process with the FDA to determine the clinical trial registration requirements for the seclidemstat Ewing sarcoma program
•Previously reported interim data showed a 60% confirmed disease control rate and 7.4 months median time to tumor progression for first-relapse Ewing sarcoma patients, with no disease progression observed in either first- or second-relapse patients who achieved confirmed disease control
•The Company continues to monitor patients in the Ewing sarcoma trial and plans to release updated survival data in the coming months
•The FDA previously granted seclidemstat Fast Track, Orphan Drug and Rare Pediatric Disease designations for Ewing sarcoma
•University of Texas MD Anderson Cancer Center (MDACC) is working to restart their investigator initiated Phase 1/2 study with seclidemstat in combination with azacytidine in patients with myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML)
•Researchers at MDACC previously reported interim clinical trial results in patients who relapsed or progressed after hypomethylating agent therapy. Of eight evaluable patients, four (50%) had an objective response. These researchers reported a 90% probability of patient survival for 11 months in patients receiving seclidemstat plus azacitidine versus an expected survival of four to six months

SP-3164 Highlights

•On July 11, 2023 Salarius announced that the FDA had cleared the IND application to treat relapsed/refractory non-Hodgkin lymphoma patients with SP-3164.
•Presented NHL preclinical data at the European Hematology Association (EHA) (Free EHA Whitepaper) 2023 Hybrid Congress in June that showed:
oPotent degradation of Ikaros and Aiolos (I/A) in peripheral blood mononuclear cells (PBMC) within 2 hours of dosing, which increased in a dose- and time-dependent manner over 24 hours
oSP-3164 does not negatively impact PBMC at clinically relevant concentrations up to 96 hours post-treatment
oIn addition to having direct antitumor effects, SP-3164 also induces an anticancer immunomodulatory effect as demonstrated through the induction of cytokine secretion in human T cells following treatment
•Presented two abstracts at the AACR (Free AACR Whitepaper) Annual Meeting in April:
oOne presentation demonstrated the robust protein degradation effects of SP-3164 and its anticancer activity in NHL animal models as well as SP-3164’s compelling antitumor activity in animal models of follicular lymphoma, a type of NHL, as a single agent and in combination with venetoclax (Venclexta) or tazemetostat (Tazverik)
oThe other presentation demonstrated SP-3164’s compelling anticancer activity in cell lines and animal models of multiple myeloma. In animal models, SP-3164 demonstrated superior single-agent activity compared with the approved agents lenalidomide (Revlimid) and pomalidomide (Pomalyst), and the combination of SP-3164 and bortezomib (Velcade) was shown to be superior to the combination of pomalidomide and bortezomib.