On May 20, 2021 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company developing potential new medicines for patients with pediatric cancers, solid tumors, and other cancers, reported the publication by the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) of three abstracts accepted for poster presentations during the ASCO (Free ASCO Whitepaper) 2021 Annual Meeting (Press release, Salarius Pharmaceuticals, MAY 20, 2021, View Source [SID1234580429]). ASCO (Free ASCO Whitepaper) 2021 will take place June 4-8, 2021 via a virtual platform.

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Salarius submitted three abstracts disclosing clinical research involving its lead drug candidate, seclidemstat. Details on the presentations can be found below. Key findings include data showing that seclidemstat has a manageable safety profile with no significant hematological toxicities, which can be a limitation for other LSD1 inhibitors. In addition, seclidemstat showed proof-of-concept preliminary drug activity in relapsed/refractory Ewing sarcoma and other advanced cancer patients, including FET-rearranged sarcomas, at or below the recommended phase 2 dose (RP2D), which was established to be 900 mg BID.

"We are excited to have the opportunity to present clinical data regarding safety, dosing, and initial efficacy signals for seclidemstat during ASCO (Free ASCO Whitepaper) 2021," said David Arthur, President and Chief Executive of Salarius Pharmaceuticals. "For the first time, we will report full data and the recommended Phase 2 dose from the recently completed dose-escalation stage of our Phase 1/2 clinical trial in Ewing sarcoma. We will also discuss in more detail the preliminary drug activity data observed in FET-rearranged sarcoma patients from our Advanced Solid Tumor (AST) trial that supports our continued development of seclidemstat as single-agent therapy in select sarcomas."

The full abstracts are available on ASCO (Free ASCO Whitepaper)’s 2021 Meeting Library. Details from the Salarius abstracts are as follows:

Abstract #11514: Phase 1 trial of seclidemstat (SP-2577) in patients with relapsed/refractory Ewing sarcoma
Session Type & Title: Poster Discussion Session, Sarcoma
Presenting Author: Damon R. Reed, M.D., H. Lee Moffitt Cancer Center & Research Institute, Tampa, Florida.
Date & Time: June 4, 2021, 9 a.m. ET
Key Information & Findings: Seclidemstat has a manageable safety profile with proof-of-concept preliminary activity in heavily pretreated patients with relapsed or refractory Ewing sarcoma.

900 mg BID established as the RP2D
A patient dosed at 600 mg BID achieved a reduction in target lesions after 58 days (cycle 2) with further tumor shrinkage after 112 days (cycle 4) and 168 days (cycle 6) for a maximum 76% tumor shrinkage; Coincident new non-target lesion appearance at end of cycle 2
Two additional patients dosed at 600 mg BID and 900 mg BID had overall stable disease
No treatment-related deaths; The most common (>5%) Grade 3 treatment-related adverse events (TRAEs) were vomiting (15%), abdominal pain (11%), and hypokalemia (11%); One patient with Grade 3 pancreatitis reported elevated lipase, a Grade 4 adverse event (AE); No significant hematological TRAES (Grade 3 occurred in <5% of patients)
Abstract #3073: Preliminary efficacy from an ongoing phase 1 dose escalation study of seclidemstat (SP-2577) in patients (pts) with advanced solid tumors (AST)
Session Type & Title: Poster Session, Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Presenting Author: Sant P. Chawla, M.D., Sarcoma Oncology Research Center, Santa Monica, California
Date & Time: Friday, June 4, 2021, 9 a.m. ET
Key Information & Findings: Seclidemstat has shown activity among advanced sarcoma patients with a manageable safety profile. Dose escalation is ongoing and preliminary clinical data supports further exploration in FET-translocated sarcomas as single agent and in combination therapy. Safety data will be presented after completion of Phase 1 dose-escalation.

As of December 30, 2020, 19 patients were enrolled in AST trial, including patients with prostate ovarian, pancreatic, renal, cervical and breast cancer, as well as sarcomas; Patients received a median of four prior systemic treatments
13 patients were evaluable for response after 58 days of treatment (cycle 2); 7 patients had best response of stable disease (SD) with median time to progression (TTP) of 4.3 months, including 3 patients with advanced FET-rearranged sarcomas
All 7 patients were dosed at 300 mg BID or 600 mg BID
The most common Grade 3 TRAEs were gastrointestinal-related and included diarrhea (5.3%) and abdominal pain (5.3%)
No treatment-related deaths and no Grade 4 TRAEs have been reported
Abstract #TPS11577: Phase 1 expansion trial of the LSD1 inhibitor seclidemstat (SP-2577) with and without topotecan and cyclophosphamide (TC) in patients (pts) with relapsed or refractory Ewing sarcoma (ES) and select sarcomas
Session Type & Title: Poster Session, Sarcoma
Presenting Author: Damon Reed, M.D., H. Lee Moffitt Cancer Center & Research Institute, Tampa, Florida.
Date & Time: June 4, 2021, 9 a.m. ET
Key Information: This is an ongoing dose-expansion study assessing seclidemstat at the RP2D (900 mg BID) in two cohorts: a single-agent expansion in select sarcoma patients and a safety lead-in dose escalation and expansion of seclidemstat in combination with topotecan and cyclophosphamide (TC) in Ewing sarcoma patients.

The sarcoma cohort will enroll patients with myxoid liposarcoma or other select sarcomas with FET family translocations, including desmoplastic small round cell tumor (DSRCT); The trial will allow patients treated with one to three prior lines of therapy
The Ewing sarcoma cohort will allow patients treated with up to two prior lines of therapy
Primary objective is safety and tolerability, and secondary objective is efficacy
Recruiting patients across eight U.S. locations
This year, ASCO (Free ASCO Whitepaper) received and reviewed more than 5,400 abstracts for the 2021 Annual Meeting, the world’s largest gathering of physicians, biopharmaceutical companies, researchers, and investors to discuss cancer research and therapeutics. Information about the Annual Meeting may be accessed at View Source

On May 20, 2021 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 Japan Patent Office has allowed the patent application titled "Systems and Methods for Producing Synthetic Hypericin" (Press release, Soligenix, MAY 20, 2021, View Source [SID1234580424]). The allowed claims are directed to unique, proprietary methods to produce a novel, highly purified form of synthetic hypericin, and are similar to those previously allowed in the United States (U.S.). Synthetic hypericin is the active pharmaceutical ingredient in HyBryte (SGX301), the Company’s photodynamic therapy, for which positive primary endpoint results in a pivotal Phase 3 study for the treatment of cutaneous T-cell lymphoma (CTCL) were recently announced. This new patent is the first allowed in Japan covering the proprietary methods developed by the Company and further expands the comprehensive HyBryte patent estate, which includes protection on the composition of the purified synthetic hypericin, methods of synthesis and therapeutic methods of use in both CTCL and psoriasis, and is being pursued worldwide.

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HyBryte (synthetic hypericin) is a novel first-in-class photodynamic therapy for first-line treatment of early stage CTCL. In the recently completed pivotal Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial, HyBryte achieved a statistically significant treatment response rate (p=0.04) in the primary endpoint after just 6 weeks (Cycle 1) of therapy when compared to placebo. This positive treatment response continued to significantly improve with extended HyBryte treatment in the open-label treatment cycles after 12 weeks (Cycle 2) and 18 weeks (Cycle 3) total treatment, reinforcing the positive HyBryte primary endpoint treatment response demonstrated in Cycle 1. In addition, HyBryte has demonstrated a statistically significant response in both patch and plaque lesions through 12 weeks of treatment (Cycle 2), highlighting the unique benefit of using visible light with its deeper skin penetration. HyBryte was well tolerated throughout the study and no mutagenic risks have been identified, unlike other second-line or off-label treatments, including other phototherapies, which utilize ultraviolet light. The Company believes HyBryte has compelling competitive advantages over existing therapies for early stage CTCL and represents a significant commercial opportunity.

"This recently issued patent continues to expand, strengthen and protect our synthetic hypericin patent estate," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "With the support of key patient advocacy organizations, such as the Cutaneous Lymphoma Foundation, and key opinion leaders, we are moving towards marketing approval and commercialization of HyBryte, with the initial focus on the U.S. market, with partnership opportunities being explored to leverage ex-U.S. markets."

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.

About HyBryte

HyBryte (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 16 to 24 hours later which triggers apoptosis of the cell. 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 U.S. Food and Drug Administration (FDA), as well as orphan designation from the European Medicines Agency (EMA) and Promising Innovative Medicine (PIM) and "Innovation Passport" under the Innovative Licensing and Access Pathway (ILAP) from the Medicines and Healthcare Products Regulatory Agency (MHRA) of the United Kingdom.

The Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) 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 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. Follow-up visits were completed in Q4 2020, and the clinical study report to support the NDA is in the process of being finalized.

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. Its 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 no 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.

On May 20, 2021, Aclaris Therapeutics, Inc. (the "Company") reported that it entered into a Sales Agreement (the "Agreement") with SVB Leerink LLC ("SVB Leerink") and Cantor Fitzgerald & Co. ("Cantor") under which the Company may offer and sell, from time to time at its sole discretion, shares of its common stock, par value $0.00001 per share (the "Common Stock"), having an aggregate offering price of up to $150,000,000 through SVB Leerink and Cantor as its sales agents (Filing, 8-K, Aclaris Therapeutics, MAY 20, 2021, View Source [SID1234580423]). The issuance and sale, if any, of Common Stock by the Company under the Agreement will be made pursuant to a registration statement on Form S-3.

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SVB Leerink and Cantor may sell the Common Stock by any method permitted by law deemed to be an "at the market offering" as defined in Rule 415 of the Securities Act of 1933, as amended. SVB Leerink and Cantor will use commercially reasonable efforts to sell the Common Stock from time to time, based upon instructions from the Company (including any price, time or size limits or other customary parameters or conditions the Company may impose). The Company will pay SVB Leerink and Cantor a commission equal to three percent (3.0%) of the gross sales proceeds of any Common Stock sold through SVB Leerink and Cantor under the Agreement. The Company has provided customary representations, warranties and covenants and the parties have agreed to customary indemnification rights.

The Company is not obligated to make any sales of Common Stock under the Agreement. The offering of shares of Common Stock pursuant to the Agreement will terminate upon the earlier of (i) the sale of all Common Stock subject to the Agreement or (ii) termination of the Agreement in accordance with its terms.

The foregoing description of the Agreement is not complete and is qualified in its entirety by reference to the full text of the Agreement, a copy of which is filed herewith as Exhibit 10.1 to this Current Report on Form 8-K and is incorporated herein by reference.

This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy the securities discussed herein, nor shall there be any offer, solicitation, or sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state.

On May 20, 2021 Processa Pharmaceuticals, Inc. ("Processa") presented the corporate presentation (Presentation, Processa Pharmaceuticals, MAY 20, 2021, View Source [SID1234580422]).

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On May 20, 2021 NovalGen Ltd ("NovalGen"), a biopharmaceutical company developing breakthrough cancer therapies, reported the company will give a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual meeting 2021, taking place June 4-8, 2021 (Press release, UCLB, MAY 20, 2021, View Source [SID1234580421]).

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NovalGen has developed an ROR1xCD3 bispecific antibody T cell engager, NVG-111, that is currently entering Phase 1/2 development for patients with non-Hodgkin lymphoma; initially focused on Mantle Cell Lymphoma (MCL) and Chronic Lymphocytic Leukemia (CLL), but with the potential to target >20 different hard-to-treat solid and liquid cancers. NVG-111 is a first in class bispecific antibody T cell engager targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1). ROR1 is a tumor-associated antigen that is overexpressed in a broad range of cancers, but has negligible expression in healthy adult tissues, making it an ideal candidate for novel, targeted cancer therapies.

"These data presented in our poster at ASCO (Free ASCO Whitepaper) show preclinical data supporting our Phase 1/2 first in human study for NVG-111," said Professor Amit Nathwani, CEO of NovalGen. "The data demonstrates NVG-111 eliciting potent killing at low concentrations of the drug. Additionally, cytokine release appears lower than other T cell engagers. Overall, it advances the scientific understanding of the potential of NVG-111 in a range of hard-to-treat cancers."

Poster Presentation Details:

Abstract Number for Publication: 7549

Abstract Title: NVG-111, a novel ROR1xCD3 bispecific antibody for non-Hodgkin lymphoma

Session Title: Poster Session: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia

The abstract is available on the ASCO (Free ASCO Whitepaper) meeting library.