On May 11, 2023 TCR2 Therapeutics Inc. (Nasdaq: TCRR) (TCR2 or the Company), a clinical-stage cell therapy company with a pipeline of novel next-generation T cell therapies for patients suffering from solid tumors, reported financial results for the first quarter ended March 31, 2023 and provided a corporate update (Press release, TCR2 Therapeutics, MAY 11, 2023, View Source [SID1234631538]).

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"We are pleased with our continued progress during the last quarter which included the announcement of the strategic combination of TCR2 with Adaptimmune. We believe that the integration of the two companies’ complimentary technology platforms and pipelines focused on treating solid tumors has the potential to fundamentally change the war on cancer," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics.

Recent Developments

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TCR2 announced a strategic combination with Adaptimmune Therapeutics plc (Adaptimmune) to create a preeminent cell therapy company for solid tumors. The two companies entered into a definitive agreement under which Adaptimmune will combine with TCR² in an all-stock transaction. The transaction is expected to close in the second quarter of 2023, subject to approval by TCR2 stockholders and Adaptimmune shareholders and satisfaction or waiver of other closing conditions. Following the closing of the transaction, Adaptimmune shareholders will own approximately 75% and TCR² stockholders will own approximately 25% of the combined company. As a result, and following the closing of the transaction, it is anticipated that the combined company’s cash runway will extend into 2026. A special meeting of TCR2 stockholders to approve the merger with Adaptimmune will be held on May 30, 2023. Proxy materials and voting instructions have been made publicly available. Please call TCR2’s proxy solicitor, Innisfree M&A Incorporated, at (877) 750-8233, with any questions.
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TCR2 presented Phase 1 gavo-cel data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) meeting and published preclinical gavo-cel data in OncoImmunology.

Financial Highlights

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Cash Position: TCR2 ended the first quarter of 2023 with $110.3 million in cash, cash equivalents, and investments compared to $149.2 million as of December 31, 2022. Net cash used in operations was $40.0 million for the first quarter of 2023 compared to $31.1 million for the first quarter of 2022.
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R&D Expenses: Research and development (R&D) expenses were $29.2 million for the first quarter of 2023 compared to $22.3 million for the first quarter of 2022. The increase in R&D expenses was primarily due to an increase in clinical trial expenses associated with patient treatment and product manufacturing.
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Impairment and Restructuring Expenses: Impairment expenses were $4.0 million for the first quarter of 2023 compared to $0.6 million for the first quarter of 2022. The increase in impairment expenses for the first quarter includes $2.1 million in severance and related costs and $1.9 million in charges related to construction in progress and certain laboratory equipment.
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G&A Expenses: General and administrative expenses were $8.2 million for the first quarter of 2023 compared to $6.3 million for the first quarter of 2022. The increase in general and administrative expenses for the first quarter includes $3.6 million in transactions costs related to the proposed transaction with Adaptimmune.
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Net Loss: Net loss was $40.4 million for the third quarter of 2023 compared to $29.1 million for the first quarter of 2022.

About gavo-cel, TC-510, and TC-520

Our most advanced program, gavo-cel, targets tumors that express the protein mesothelin.

TC-510 is an enhanced version of gavo-cel that co-expresses a PD-1:CD28 chimeric switch receptor that the Company believes may lead to deeper responses and more durable benefit.

TC-520 is the Company’s first TRuC-T cell targeting CD-70-expressing solid and liquid tumors which incorporates IL-15 pathway enhancements designed to improve T-cell persistence. TCR2 is currently advancing TC-520 to Investigational New Drug (IND) status.

On May 11, 2023 Synlogic, Inc. (Nasdaq: SYBX), the leading company advancing therapeutics based on synthetic biology, reported its financial results for the first quarter ended March 31, 2023, and provided an update on its pipeline programs (Press release, Synlogic, MAY 11, 2023, View Source [SID1234631537]).

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"We continue to advance the SYNB1934 program towards initiation of our pivotal study, Synpheny-3, in the first half of the year, focused on our opportunity to transform the medical management of PKU," said Aoife Brennan, M.B. Ch.B., Synlogic President and Chief Executive Officer. "The quarter also included recognition of Synthetic Biotics as novel biotherapeutics for rare metabolic diseases more broadly, as both our PKU and homocystinuria (HCU) programs were spotlighted at the leading medical congress for inborn errors of metabolism."

First Quarter 2023 and Recent Business Highlights

Received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for SYNB1934 for PKU
Received positive opinion on orphan designation from the European Medicines Agency (EMA) for SYNB1934 for PKU
Announced full data from both the Phase 2 Synpheny-1 study in PKU and the Phase 1 study in HCU at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting
Anticipated Upcoming Milestones

Initiation of Phase 3 clinical trial of SYNB1934 for PKU in the first half of 2023
Advancing SYNB1353 to Phase 2 study in patients with HCU
Progression of preclinical pipeline programs, including partnerships
First Quarter 2023 Financial Results

As of March 31, 2023, Synlogic had cash, cash equivalents and short-term investments of $57.4 million.

Revenue was $0.2 million for each of the three months ended March 31, 2023 and March 31, 2022. Revenue in both periods was primarily associated with the ongoing research collaboration with Roche for the discovery of a novel Synthetic Biotic for the treatment of inflammatory bowel disease.

For the three months ended March 31, 2023, Synlogic reported a consolidated net loss of $15.6 million, or $0.23 per share, compared to a consolidated net loss of $15.7 million, or $0.22 per share, for the corresponding period in 2022.

Research and development expenses were $12.5 million for the three months ended March 31, 2023, compared to $11.7 million for the corresponding period in 2022.

General and administrative expenses were $4.0 million for the three months ended March 31, 2023, compared to $4.3 million for the corresponding period in 2022.

Financial Outlook

Based upon its current operating plan and balance sheet as of March 31, 2023, Synlogic expects to have sufficient cash to be able to fund operations into the second half of 2024.

Upcoming Investor and Industry Conference Participation

Synlogic leadership will attend the Jefferies Global Healthcare Conference being held June 7-9, 2023, in New York City.

On May 11, 2023 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported that data from two collaborator-sponsored clinical studies evaluating nirogacestat, an investigational oral gamma secretase inhibitor, in combination with B-cell maturation agent (BCMA) therapies in patients with relapsed or refractory multiple myeloma (RRMM) will be presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2023 Congress, taking place in Frankfurt, Germany from June 8-11, 2023 (Press release, SpringWorks Therapeutics, MAY 11, 2023, View Source [SID1234631536]). Updated clinical data from the Phase 1/2 study sponsored by GSK plc (LSE/NYSE: GSK) evaluating nirogacestat in combination with low-dose belamaf (belantamab mafodotin-blmf), GSK’s antibody-drug conjugate targeting BCMA, in patients with RRMM will be presented in a poster presentation. In addition, new data from the Phase 1b clinical trial sponsored by Janssen Research & Development, LLC (Janssen) evaluating nirogacestat in combination with teclistamab, Janssen’s bispecific antibody targeting BCMA and CD3, will be presented in an oral presentation.

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"These data provide further validation of the mechanistic approach supporting nirogacestat’s ability to enhance the activity of BCMA-directed therapies across modalities. We are pleased that updated data from the GSK-sponsored trial continue to support our thesis that the combination with nirogacestat may further optimize the benefit-risk profile of belamaf monotherapy. We are also encouraged that the Janssen-sponsored trial establishes an initial tolerability, safety and efficacy profile for combining nirogacestat with a BCMA bispecific antibody," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our goal is to improve the outcomes for patients with multiple myeloma and we believe that developing a robust clinical data set across BCMA modalities and treatment lines can help us demonstrate where within the multiple myeloma treatment landscape nirogacestat has the greatest opportunity to maximize clinical benefit."

Poster Presentation at the EHA (Free EHA Whitepaper) 2023 Congress

Low-dose belantamab mafodotin (belamaf) in combination with nirogacestat vs belamaf monotherapy in patients with relapsed/refractory multiple myeloma (RRMM): Phase 1/2 DREAMM-5 Platform Sub-study 3

Abstract #: P913

Session Date and Time: Friday, June 9, 18:00-19:00 CEST (12:00-1:00 p.m. ET)

This ongoing Phase 1/2 trial, which is sub-study 3 of GSK’s DREAMM-5 platform trial (NCT04126200), aims to determine if low-dose belamaf in combination with nirogacestat results in similar efficacy with an improved ocular toxicity profile compared to belamaf alone at a higher dose. Patients were randomized 1:1 to 0.95 mg/kg belamaf every three weeks (Q3W, low-dose) combined with 100 mg twice daily nirogacestat or belamaf 2.5 mg/kg Q3W monotherapy.

Initial results from the pre-planned interim analysis were presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Updated data from a randomized cohort expansion in which 34 patients received low-dose belamaf plus nirogacestat and 37 patients received belamaf monotherapy will be presented at EHA (Free EHA Whitepaper). Patients had a median of 5 (range 3-14) prior lines of therapy. As of the December 9, 2022 data cut-off, patients received a median of 4 (range 1-20) cycles of the combination and a median of 3 (range 1-9) monotherapy cycles.

Overall response rate (ORR) in the low-dose belamaf (0.95 mg/kg Q3W) plus nirogacestat arm was 29%, with 1 patient (3%) achieving a complete response (CR), 5 patients (15%) achieving a very good partial response (VGPR), and 4 patients (12%) achieving a partial response (PR). ORR in the belamaf monotherapy arm (2.5 mg/kg Q3W) was 38%, with no patient achieving a CR, 5 patients (14%) achieving a VGPR, and 9 patients (24%) achieving a PR. Per the prespecified analysis plan, ORR was also calculated incorporating prior ORR for low-dose belamaf plus nirogacestat from the dose exploration cohort of this DREAMM-5 sub-study 3 and from the DREAMM-2 belamaf monotherapy study; ORR across these studies was 36% in the low-dose belamaf plus nirogacestat combination arm and 33% in the belamaf monotherapy arm.

Safety results showed a substantial reduction of high-grade ocular events in the low-dose belamaf plus nirogacestat arm compared to the monotherapy arm of belamaf at a higher dose. Specifically, Grade 3 ocular events were 29% for low-dose belamaf + nirogacestat versus 59% for belamaf monotherapy (per the KVA scale); no Grade 4 ocular events or new toxicities occurred in either arm.

"These clinical data suggest that combining nirogacestat with a low dose of belamaf may result in comparable efficacy to a higher monotherapy belamaf dose, while simultaneously substantially reducing the frequency high-grade ocular adverse events," said Jim Cassidy, M.D., Ph.D., Chief Medical Officer of SpringWorks. "We are encouraged by these results and we look forward to the further evaluation of this combination with standard of care agents in relapsed refractory multiple myeloma as well as its potential in earlier lines of therapy."

Oral Presentation at the EHA (Free EHA Whitepaper) 2023 Congress

Teclistamab (tec) + nirogacestat (niro) in relapsed/refractory multiple myeloma (RRMM): the Phase 1b MajesTEC-2 study

Session Title: MM Clinical: New combinations and novel targets

Abstract #: S194

Session Date and Time: Saturday, June 10, 12:30-12:45 CEST (6:30-6:45 a.m. ET)

This ongoing Phase 1b trial (NCT04722146), which is part of a multi-arm trial being conducted by Janssen, aims to evaluate the safety, tolerability and preliminary efficacy of nirogacestat in combination with teclistamab in patients with RRMM. Patients in the study had received ≥3 prior lines of therapy or were double refractory to a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD) and triple exposed to a PI, an IMiD, and an anti-CD38 antibody, with progressive disease within 12 months of their last line of therapy. Three dose levels were evaluated: 1) teclistamab 720 μg/kg weekly plus concurrent nirogacestat 100 mg twice daily starting with the first dose of teclistamab (n=8); 2) teclistamab 720 μg/kg weekly plus once daily nirogacestat 100 mg starting after teclistamab step-up dosing (n=7); and 3) 1500 μg/kg (which is the FDA-approved dose) weekly plus once daily nirogacestat 100 mg starting after teclistamab step-up dosing (n=13).

As of the December 16, 2022 data cut-off, 28 patients received the combination of teclistamab and nirogacestat across three different dose levels. Median prior lines of therapy was 4 (range 2-12) and median duration of treatment was 9.4 months (range 0.13-19.7) for teclistamab and 4.7 months for nirogacestat (range 0.16-13.0).

The most frequent (>20%) treatment-emergent adverse events (TEAEs) for all doses were neutropenia (82%), cytokine release syndrome, or CRS (75%), diarrhea (64%), injection-site erythema (54%), decreased appetite (50%), fatigue (42.9%), and anemia (35%). Of eight patients who received teclistamab 720 μg/kg plus concurrent nirogacestat, two dose-limiting toxicities were reported (one Grade 3 GI bleed and Grade 3 diarrhea; one Grade 3 immune effector cell-associated neurotoxicity syndrome, or ICANS). In addition, one patient had Grade 3 CRS and one patient had Grade 3 confusional state. These events led to the decision to delay the starting dose of nirogacestat in subsequent cohorts. In the dose level 2 and dose level 3 cohorts, when nirogacestat was added after teclistamab step-up dosing and reduced to once daily, no dose-limiting toxicities or Grade 3 CRS or neurologic adverse events were reported.

The overall response rate was 71% for dose level 1, 57% for dose level 2, and 92% for dose level 3. The total ORR across the three dose levels was 78% and all responses were VGPR or better. The percentage of patients experiencing complete response (CR) or stringent CR (sCR) was 43%, 57% and 54%, respectively (total percentage of CR or sCR across the three cohorts was 52%).

"This is the first clinical data set of nirogacestat in combination with a BCMA bispecific agent. We believe these data provide important insights into a tolerable treatment schedule and early evidence of an encouraging ORR, including promising CR and sCR rates, when combining nirogacestat with this BCMA modality," commented Dr. Cassidy. "Nirogacestat is being evaluated in combination with three other bispecific agents and we look forward to generating more data with these combinations."

About Nirogacestat

Nirogacestat is an oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors and in Phase 2 clinical development for ovarian granulosa cell tumors. Nirogacestat is an investigational drug for which safety and efficacy have not been established.

Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to growth of desmoid and ovarian granulosa cell tumors. Gamma secretase has also been shown to directly cleave membrane-bound B cell maturation antigen (BCMA), resulting in the release of the BCMA extracellular domain (ECD) from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat’s ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has several collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities. SpringWorks has also formed research collaborations with Fred Hutchinson Cancer Research Center and Dana-Farber Cancer Institute to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA-directed therapies using a variety of preclinical multiple myeloma models.

The U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for nirogacestat for the treatment of adults with desmoid tumors, which is being reviewed under the FDA’s Real-Time Oncology Review program. The NDA was granted Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of August 27, 2023. The FDA also granted Fast Track and Breakthrough Therapy Designations to nirogacestat for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. In addition, nirogacestat has received Orphan Drug Designation from the FDA for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma.

On May 11, 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 the United States (U.S.) Food and Drug Administration (FDA) has granted a Type A meeting to discuss the design of a second, Phase 3 pivotal study evaluating HyBryte (hypericin sodium) in the treatment of early stage cutaneous T-cell lymphoma (CTCL), a rare cancer, where it has successfully demonstrated statistically significant results in the first Phase 3 clinical trial (Press release, Soligenix, MAY 11, 2023, View Source [SID1234631535]). The Type A Meeting is the highest priority classification of meeting the FDA grants companies.

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"Responding to FDA feedback, Soligenix has submitted a confirmatory Phase 3 draft study protocol retaining the key aspects of the first Phase 3 trial," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "We look forward to discussing the protocol in detail with FDA. We intend to provide a further update once we have received the minutes from the meeting or when we have more clarity on next steps, which we anticipate having by or before the end of June."

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 a prestigious academic institution 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 May 11, 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 provided its financial results for the quarter ended March 31, 2023 (Press release, Sellas Life Sciences, MAY 11, 2023, View Source [SID1234631534]).

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"We are pleased with the continued advancement of both our galinpepimut-S (GPS) and GFH009 clinical programs during the first quarter of 2023, which positions us for near-term execution of several key value-driving milestones," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "Positive topline data for the patient group with acute myeloid leukemia (AML) from the GFH009 Phase 1 dose-escalation trial in relapsed/refractory myeloid malignancies suggests strong evidence of anti-tumor activity and survival benefit in this group with no significant safety issues. This supports advancement to a Phase 2a trial in patients with AML in combination with azacitidine and venetoclax, which we are initiating this quarter, with topline data expected in Q4 2023. Last month, the Independent Data Monitoring Committee (IDMC) also recommended that our GPS Phase 3 REGAL study continue without any modifications and is expected to meet again in Q3 to provide further guidance."

Dr. Stergiou continued "The key objectives for us this quarter and next, among others, will include:

the initiation of the GFH009 Phase 2a study in AML patients in combination with azacitadine and venetoclax;
completion of, and report of, data from the lymphoma patient group from the GFH009 Phase 1 study;
report of topline data from the GPS investigator-sponsored trial (IST) in malignant pleural mesothelioma (MPM);
abstract acceptance of our GPS ovarian cancer data from a major medical gynecological conference;
3D Medicines participation in the REGAL study; and
meeting of, and feedback from, the IDMC for the REGAL study."
"It was a great pleasure meeting our SELLAS colleagues last month at our Shanghai headquarters where we had a chance to discuss and further strengthen our strategic partnership," said John Gong, M.D., Ph.D., Chairman and Chief Executive Officer of 3D Medicines. "The blinded pooled analysis of GPS in AML conducted at the end of last year is indeed very exciting and may have the potential to improve AML patients’ quality of life and extend survival. We eagerly anticipate joining the REGAL trial very soon and to enroll about 20 patients in China to satisfy Chinese regulatory requirements towards a potential market approval. In addition, we will work together to explore solid tumor indications for GPS by combination with our subcutaneous anti-PD-L1, envafolimab, and other products."

Pipeline Update:

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

Phase 3 REGAL study in AML: Enrollment continued in the global Phase 3 REGAL registrational clinical trial in patients with AML who have achieved complete remission following second-line salvage therapy (CR2 patients), with interim analysis continuing to be expected to occur in late 2023 or early 2024. The participation of 3D Medicines in the REGAL study through the enrollment of patients from the Greater China territory will trigger milestone payments totaling $13.0 million to SELLAS, which the Company expects to receive by the end of the third quarter of 2023. The IDMC performed a routine, prespecified risk-benefit assessment of unblinded data from the REGAL study in April 2023 and recommended that the trial continue without modifications. The IDMC endorsed all clinical trial initiatives undertaken in REGAL to advance GPS, including the addition of clinical sites in China. The next routine IDMC meeting is scheduled for the third quarter of 2023.

3D Medicines Phase 1 clinical trial in China: 3D Medicines continued enrollment in China for their open-label, single-arm, multi-center Phase 1 clinical trial in patients with AML, multiple myeloma, non-Hodgkin’s lymphoma, or higher-risk myelodysplastic syndrome.

Phase 1/2 Study in combination with pembrolizumab (Keytruda) in ovarian cancer: Top-line data released in November 2022 showed clinical benefit of GPS in combination with pembrolizumab anti-PD-1 therapy in WT1 positive relapsed or refractory platinum resistant advanced metastatic ovarian cancer patients. The study was conducted under a Clinical Trial Collaboration and Supply Agreement with Merck & Co., Inc., Rahway, N.J., USA (known as MSD outside the United States and Canada), and SELLAS plans to present final data at a medical conference during the fourth quarter of 2023.

Phase 1 Study in combination with nivolumab (Opdivo) for MPM: Patient enrollment was completed at the end of 2022 for an investigator sponsored open-label Phase 1 trial for GPS combination therapy with checkpoint inhibitor nivolumab (Opdivo) for treatment of MPM in patients who were either refractory to or relapsed after at least one line of the standard of care therapy. SELLAS expects to report topline data during the first half of 2023.

Phase 1 Study in combination with nivolumab (Opdivo) in ovarian cancer: Final analysis was published in the peer-reviewed journal Cancers on previously reported data from the Phase 1 clinical trial showing clinical benefit of GPS combination with anti-PD-1 antibody nivolumab (Opdivo) in patients with relapsed WT1-expressing ovarian cancers (NCT02737787).

GFH009: highly selective CDK9 inhibitor

Phase 1 clinical trial in hematological malignancies: In the Phase 1 trial of GFH009, dose escalation was successfully completed in the group of patients with AML, while dose escalation continues in the lymphoma group at the highest dose level for that group (75 mg). Positive topline data for the group of patients with AML showed evidence of anti-tumor activity increasing with higher doses and no significant safety issues even at the highest dose levels. The recommended Phase 2 dose (RP2D) has been established for AML. SELLAS plans to commence a Phase 2a trial with GFH009 in combination with venetoclax and azacitidine (aza/ven) in patients with AML during the second quarter of 2023 with topline data expected in the fourth quarter of 2023.

Corporate Updates:

Underwritten Public Offering: On February 28, 2023, the Company closed an underwritten public offering providing gross proceeds of $20.0 million, before deducting underwriting discounts and commissions and offering expenses.

Appointment of Vice President, Head of Regulatory Affairs: Andrew Elnatan joined the leadership team in January 2023 as Vice President, Regulatory Affairs, CMC and Quality. He brings nearly three decades of global regulatory experience with successful breakthrough therapy designation and global drug approvals.

Financial Results for the First Quarter 2023:

Licensing Revenue: There was no licensing revenue for the first quarter of 2023, as compared to $1.0 million for the same period in 2022.

Cost of Revenue: There was no cost of revenue for the first quarter of 2023, as compared to $0.1 million for the same period in 2022.

R&D Expenses: Research and development expenses for the first quarter of 2023 were $7.2 million, compared to $4.6 million for the same period in 2022. The increase was primarily due to the ongoing Phase 3 REGAL clinical trial of GPS in AML patients and the Phase 1 clinical trial of GFH009 in hematological malignancies.

G&A Expenses: General and administrative expenses for the first quarter of 2023 were $4.1 million, as compared to $3.0 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 quarter of 2023, compared to $10.0 million for the same period in 2022 from the in-licensing of GFH009.

Net Loss: Net loss was $11.1 million for the first quarter of 2023, or a basic and diluted loss per share of $0.47, compared to a net loss of $16.7 million for the same period in 2022, or a basic and diluted loss per share of $1.05.

Cash Position: As of March 31, 2023, cash and cash equivalents totaled approximately $23.9 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.