On November 12, 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 its recent accomplishments and financial results for the quarter ended September 30, 2021 (Press release, Soligenix, NOV 12, 2021, View Source [SID1234595487]).

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Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix stated, "There continues to be a great deal of activity across our Specialized BioTherapeutics and Public Health Solutions business segments. We continue to work diligently to prepare the new drug application (NDA) for HyBryte (SGX301 or synthetic hypericin) following the positive pivotal Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study in cutaneous T-cell lymphoma (CTCL). The Office of Orphan Products Development of the United States (U.S.) Food and Drug Administration (FDA) granted orphan drug designation to the active ingredient hypericin for the treatment of T-cell lymphoma, expanding the treatment population beyond CTCL. Additionally, we announced expansion of synthetic hypericin development into psoriasis, under the research name SGX302, where we plan to initiate a Phase 2a clinical study in the second half of 2022. This decision follows validation of synthetic hypericin’s biologic activity in the FLASH study, as well as positive proof-of-concept (PoC) demonstrated in a small Phase 1/2 pilot study in mild-to-moderate psoriasis patients, a large and underserved market with a significant unmet medical need. Under our Public Health Solutions business segment, we remain focused on developing heat-stable vaccines for global use. Most notably, we announced publication of compelling pre-clinical immunogenicity studies for CiVax (heat stable COVID-19 vaccine program) demonstrating durable broad-spectrum neutralizing antibody responses in non-human primates (NHPs), including against the Beta, Gamma and Delta variants."

Dr. Schaber continued, "With approximately $29 million in cash, not including our non-dilutive government funding, we anticipate having the necessary capital to achieve our upcoming milestones, including NDA filing and expansion into psoriasis with the conduct of the Phase 2a clinical trial. We are also continuing to actively assess various strategic options, including but not limited to, partnership and merger and acquisition opportunities."

Soligenix Recent Accomplishments

On November 8, 2021, the Company announced it had been granted a Pediatric Investigation Plan (PIP) product-specific waiver in the United Kingdom from the Medicines and Healthcare products Regulatory Agency for HyBryte in the treatment of CTCL. To view this press release, please click here.
On November 4, 2021, the Company announced the publication of pre-clinical immunogenicity studies for RiVax (heat stable ricin toxin vaccine) demonstrating enduring protection for at least 12 months post-vaccination. The article titled "Durable Immunity to Ricin Toxin Elicited by a Thermostable, Lyophilized Subunit Vaccine" has been accepted for publication in the journal mSphere. To read the publication, please click here. To view this press release, please click here.
On September 28, 2021, the Company announced the publication of pre-clinical immunogenicity studies for CiVax demonstrating durable broad-spectrum neutralizing antibody responses, including against the Beta, Gamma and Delta variants of concern. The article, titled "Protein Vaccine Induces a Durable, More Broadly Neutralizing Antibody Response in Macaques than Natural Infection with SARS-CoV-2 P.1", has been posted as an accelerated preprint on bioRxiv. To view the publication, please click here. To view this press release, please click here.
On September 16, 2021, the Company announced that following the validation of synthetic hypericin’s biologic activity in the positive pivotal Phase 3 FLASH study in CTCL, as well as PoC demonstrated in a small Phase 1/2 pilot study in mild-to-moderate psoriasis patients, the Company will be expanding this novel therapy under the research name SGX302 into psoriasis, a large and underserved market affecting between 60-125 million people worldwide. To view this press release, please click here.
On September 9, 2021, the Company announced that the Office of Orphan Products Development of the U.S. FDA had granted orphan drug designation to the active ingredient hypericin for the treatment of T-cell lymphoma, extending the target population beyond CTCL as previously granted. To view this press release, please click here.
On August 23, 2021, the Company announced a publication describing the formulation of single-vial platform presentations of monovalent (single antigen), bivalent (two antigens) and trivalent (three antigens) combinations of filovirus vaccine candidates. In collaboration with University of Hawaiʻi at Mānoa (UHM) and University of Colorado co-authors, the manuscript titled "Single-Vial Filovirus Glycoprotein Vaccines: Biophysical Characteristics and Immunogenicity after Co-lyophilization with Adjuvant", has been published in Vaccine. To read the publication, please click here. To view this press release, please click here.
On August 18, 2021, the Company announced positive data demonstrating the efficacy of multiple filovirus vaccine candidates in NHPs, including thermostabilized multivalent vaccines in a single vial platform presentation. Collaborators at UHM describe the potent efficacy of vaccine candidates protecting against three life-threatening filoviruses, Zaire ebolavirus, Sudan ebolavirus and Marburg Marburgvirus in an article titled "Recombinant Protein Filovirus Vaccines Protect Cynomolgus Macaques from Ebola, Sudan, and Marburg Viruses", published in Frontiers in Immunology. To read the article, please click here. To view this press release, please click here.
Financial Results – Quarter Ended September 30, 2021

Soligenix’s revenues for the quarter ended September 30, 2021 were $0.2 million as compared to $0.6 million for the quarter ended September 30, 2020. Revenues primarily included payments on grants received to support the development of: SGX943 for treatment of emerging and/or antibiotic-resistant infectious diseases; ThermoVax, our thermostabilization platform technology; and CiVax, our vaccine candidate for the prevention of COVID-19.

Soligenix’s basic net loss was $3.6 million, or ($0.09) per share, for the quarter ended September 30, 2021, as compared to $1.8 million, or ($0.06) per share, for the quarter ended September 30, 2020. This increased net loss was primarily due to a reduction in revenue from the expiration of government contracts, an increase in research and development expenses associated with the continued development of HyBryte and additional interest expense on convertible debt.

Research and development expenses were $2.5 million as compared to $1.3 million for the quarters ended September 30, 2021 and 2020, respectively. The increase in research and development spending for the quarter ended September 30, 2021 was primarily attributable to the increased expenses associated with the continued development of HyBryte.

General and administrative expenses were $0.9 million and $0.8 million for the three months ended September 30, 2021 and 2020, respectively.

As of September 30, 2021, the Company’s cash position was approximately $29 million.

On November 12, 2021 Intensity Therapeutics, Inc. ("Intensity"), a clinical-stage biotechnology company focused on the discovery and development of proprietary immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, reported that data from its open-label Phase 1/2 study of novel lead asset, INT230-6, as a monotherapy or in combination with ipilimumab in adult subjects with metastatic sarcomas, is being presented today, in an oral presentation, at the Connective Tissue Oncology Society Virtual Annual Meeting (CTOS) (Press release, Intensity Therapeutics, NOV 12, 2021, View Source [SID1234595486]).

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"Preliminary data suggests that INT230-6, as a monotherapy, demonstrates direct tumor killing in soft tissue sarcoma subjects (STS) and elicits an anti-cancer immune response within the injected tumor," stated poster presenter and study investigator, Matthew A. Ingham, MD, Assistant Professor of Medicine in the Division of Hematology and Oncology at Columbia University Vagelos College of Physicians and Surgeons and first author of the study. "To date, though in a limited number of patients, INT230-6 treatment related adverse events are mostly low grade and the drug is well-tolerated either as a monotherapy or in combination with checkpoint inhibitor, ipilimumab. Additionally, an exploratory analysis showed an increase in survival with dosing a volume of INT230-6 above forty percent of the total tumor burden, as compared to survival from historical sarcoma studies. These results provide further evidence to continue studying this novel therapeutic drug approach."

Lewis H. Bender, President and Chief Executive Officer of Intensity Therapeutics, added, "The promising data supports our belief that INT230-6, a locally-delivered anti-cancer product candidate, may potentially lead to clinical benefit with lower levels of off target side effects compared to chemotherapies for patients with advanced soft tissue sarcomas. Given these results, earlier this year we met with and reached alignment with FDA of the design of a randomized Phase 3 trial, subject to review by the Agency of the final protocol, to evaluate INT230-6 versus standard of care in patients with advanced soft tissue sarcomas."

Title: Safety and Efficacy from a Phase 1/2 Study of Intratumoral INT230-6 Alone or In Combination with Ipilimumab [INTENSITY# IT-01; BMS# CA184-592] in Adult Subjects with Metastatic Sarcomas (NCT 03058289)

The presentation will be accessible on the "Publications, Papers and Posters" section of Intensity’s website at: View Source

The data presented included results from 19 patients with different metastatic sarcomas treated with INT230-6 either as a monotherapy (n=10), or in combination with a checkpoint inhibitor (n=8) primarily ipilimumab. The enrolled patients’ cancer progressed following a median of three prior lines of therapy (range 0 to 9) including all approved, appropriate therapies for a subject’s particular cancer. Demographics were similar in subjects enrolled in monotherapy and checkpoint inhibitor combination arms. The cumulative dose of INT230-6 IT injections ranged from 20 to 530mL (265mg cisplatin, 53mg vinblastine), with repeated intratumoral injections in multiple tumors. Pharmacokinetic (PK) profile analysis from 18 STS subjects in study IT-01 were analyzed for cisplatin, SHAO and vinblastine and show less than 5% of the drugs enter the blood stream.

A retrospective exploratory analysis of subjects was assessed by comparing INT230-6 dosed at greater than or equal to 40% of their incoming total tumor burden, suggesting promising survival. For sarcoma subjects dosed to >40% of their total tumor burden, approximately 60% of subjects will be alive at 1 year. These results compare favorably to those seen in historical Phase 1/2 studies of sarcoma subjects where approximately 50% of subjects are deceased at 3 to 8 months depending on certain prognostic factors (ECOG, LDH, # of metastatic sites). Abscopal effects were seen in visceral lesions in two subjects, which may be an underestimation, as no tumors under 1 cm and not all larger lesions were followed.

Immunohistochemistry analysis of INT230-6 monotherapy paired (pre- and 28 days post-dose) biopsy of injected lesions from 3 INT230-6 monotherapy STS subjects showed a median of 60% reduction in tumor cell content and a 72% reduction in Ki67 staining (proliferation marker).

Hematoxylin and Eosin assessments demonstrated substantial reductions of cancer cells in biopsies from monotherapy subjects after two injections of INT230-6 when compared to baseline. Local delivery of INT230-6 as monotherapy into tumors induced an immune response with increases of activated CD4+ and CD8+ T-cells in the tumor with a few low grade immune-related adverse events. These clinical results are consistent with immune findings from in vivo models. Most other adverse events were low grade and transient, consisting mostly of pain at the injection site (68.4% of patients), fatigue (42.1%) or decreased appetite (36%). There were no grade 4 or 5 treatment emergent adverse events and no events that were dose limiting.

About INT230-6

INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug is composed of two proven, potent anti-cancer agents, cisplatin and vinblastine, and a penetration enhancer molecule that helps disperse the drugs throughout tumors for diffusion into cancer cells. In addition to local disease control, direct killing of the tumor by INT230-6 creates neoantigens leading to engagement of the immune system and systemic anti-tumor effects in the tumor. Importantly, these effects are mediated without the immunosuppression of concomitant systemic chemotherapy.

INT230-6 is currently being evaluated in several Phase 2 cohorts (NCT03058289) in patients with various advanced solid tumors as part of Study IT-01. In 2019, the Company signed a clinical collaboration agreement with Merck Sharpe & Dohme (Merck) to evaluate the combination of INT230-6 and KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 (programmed death receptor-1) therapy, in patients with advanced pancreatic, colon, squamous cell and bile duct malignancies. In 2020, the Company executed a clinical collaboration agreement with Bristol-Myers Squibb Company to evaluate the combination of INT230-6 with Bristol-Myers Squibb’s anti-CTLA-4 antibody, Yervoy (ipilimumab), in patients with advanced liver, breast and sarcoma cancers. In 2021, the Company executed agreements with the Ottawa Hospital Research Institute and the Ontario Institute of Cancer Research to study INT230-6 in a randomized controlled neoadjuvant Phase 2 study in women with early stage breast cancer (the INVINCIBLE study) (NCT04781725).

On November 12, 2021 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that Walter V. Kemp, Founder, President, CEO and Chairman and Dr. John Paul Waymack, Senior Chief Medical Officer will participate in the Virtual Investor Roundtable Event on Thursday, November 18, 2021 at 11:00 AM ET (Press release, Moleculin, NOV 12, 2021, View Source [SID1234595485]).

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Joining the Company’s management team for the roundtable discussion will be Sant P. Chawla, MD, Director, Sarcoma Oncology Center, Director, Cancer Center of Southern California.

As part of the virtual event, the Company will provide a brief presentation, followed by a moderated Roundtable discussion and an interactive Q&A session. In addition to the moderated portion of the event, all investors and interested parties will have the opportunity to submit questions live during the event. Interested parties may also pre-submit questions in advance of the live event, which can be sent via the conference website at virtualinvestorco.com. The Company will answer as many questions as possible during the event.

A live video webcast of the Roundtable Event will be available on the Events page of the Investors section of the Company’s website (moleculin.com). A webcast replay will be available two hours following the live presentation and will be accessible for one year.

On November 12, 2021 Actym Therapeutics reported that ACTM-838 has been selected as a lead clinical development candidate for the treatment of solid tumors (Press release, Actym Therapeutics, NOV 12, 2021, View Source [SID1234595484]). ACTM-838 is based on the company’s immunotherapy platform called STACT (S. Typhimurium-Attenuated Cancer Therapy), which delivers multiplexed immuno-modulatory payloads directly to tumor-resident immune cells . ACTM-838 is a STACT strain that encodes engineered IL-15 (IL-15plex) and STING (eSTING). In preclinical studies, the IL-15plex + eSTING payload combination delivered by STACT resulted in primary human and mouse M2 macrophage production of type I interferon, and repolarization into a novel hybrid M1/M2 phenotype capable of tumor cell phagocytosis and priming of CD8+ T-cells. Complete tumor responses were observed in metastatic, checkpoint refractory tumors (IV dosing), that were entirely dependent on CD8+ T-cells. In primates, the therapy was well tolerated, rapidly cleared, and elicited minimal cytokine responses after IV dosing.

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"The nomination of a lead candidate is a tremendous accomplishment for our company, and we’re excited about the possibility of bringing a desperately needed treatment option to cancer patients," said Christopher Thanos, Ph.D., Cofounder and Chief Executive Officer of Actym Therapeutics.

Actym’s data will be presented on Friday, November 12, 2021, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in Washington D.C. (Presentation #853, Novel Single Agent Immunotherapies).

On November 12, 2021 PathAI, a global provider of AI-powered technology applied to pathology reported that results from a recent exploratory biomarker analysis on digitized slides to apply AI-predicted CD8 topology assessment of patients with advanced melanoma will be presented at the annual Meeting of Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), November 10-14 2021 (Press release, PathAI, NOV 12, 2021, View Source [SID1234595483]).

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The results will be shared in two posters, Lee et al. The utility of AI-powered spatial classification of intratumoral CD8+ immune-cell topology in predicting overall survival in patients with melanoma as part of the CheckMate 067 clinical trial, and Glass et al. Machine Learning Models Can Quantify CD8 Positivity in Melanoma Clinical Trial Samples.

PathAI developed Machine Learning (ML) models to quantify CD8+ T cells in digitized whole slide images (WSI) of melanoma patient samples, and validated their performance against a consensus of pathologists on a held-out data set. These models were deployed on CD8-stained biopsy samples collected from patients with previously untreated advanced melanoma in the CheckMate 067 clinical trial. The AI-based predictions of CD8 positivity were used by ML models created by Bristol Myers Squibb to categorize each WSI by spatial pattern and density of CD8+ T cell infiltration (CD8 topology) as desert (deficient in CD8+ T cells), excluded (CD8+ T cells at the tumor boundaries and surrounding stroma), or inflamed (CD8+ T cells within the tumor parenchyma).

PD-L1 stained tumor cell positivity scores of either PD-L1 <1% or PD-L1 >1%, collected previously during the CheckMate 067 clinical trial, were combined with the CD8+ spatial phenotype scores to create a composite biomarker. Correlations between overall survival of patients in CheckMate 067 and CD8+ spatial phenotype alone, or the composite biomarker identified a subpopulation of patients with PD-L1 expression < 1%, and a CD8+-excluded tumor spatial pattern that benefited significantly from the drug combination treatment (nivolumab and ipilimumab) compared with PD-L1 negative patients with a CD8+-inflamed tumor spatial pattern (P = 0.002). No difference in survival between excluded and inflamed phenotypes was observed for patients treated with monotherapy (nivolumab alone) (P = 0.41), nor with any patients with PD-L1 >1%.

The data presented here suggest that in addition to PD-L1 positive patients, PD-L1 negative CheckMate 067 patients who were also CD8+ in specific subregions of tumors had overall survival benefit when treated with ipilimumab plus nivolumab combinations compared to those that were treated with ipilimumab monotherapy.

Together, these publications highlight the promise of ML-based CD8+ phenotype scoring to reveal populations of patients that might have the greatest benefit from existing treatments.