Soligenix Announces Presentations at Prominent Scientific Conferences

On April 13, 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 planned conference presentations scheduled through April and May (Press release, Soligenix, APR 13, 2021, View Source [SID1234578028]). The presentations will focus on the HyBryte (SGX301 or synthetic hypericin) cutaneous T-cell lymphoma (CTCL) program, including elaboration on previously released efficacy and safety outcomes in the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study and an update on the CiVax (COVID-19 subunit vaccine) program. Registration links for each conference are included below.

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Planned Oral Presentations:

Visible Light Activated Topical Hypericin Ointment in CTCL: Phase 3 FLASH Study Results presented by Dr. Ellen Kim, Lead FLASH Investigator, at the American Academy of Dermatology Association Virtual Meeting Experience, April 23-25, 2021. As a Top 12 Late-Breaking Research Abstract, Dr. Kim will also be present at a live Q&A session on April 24 from 2-3 pm Central Time. Attendees can register here.

Progress towards a Thermostabilized, Single-Vial, COVID-19 Subunit Vaccine with a Nano-emulsion Adjuvant presented by Dr. Oreola Donini, Chief Scientific Officer, at the Annual Conference on Vaccinology Research, April 26-27, 2021. Attendees can register here.

Topical hypericin ointment photodynamic therapy is effective and safe in CTCL (FLASH study) presented by Dr. Ellen Kim, Lead FLASH Investigator, at the Society for Investigative Dermatology Virtual Meeting, May 3-8, 2021. Attendees can register here.

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. 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 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 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. 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 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.

About CiVax

CiVax is the Company’s heat stable subunit vaccine candidate for the prevention of COVID-19, the infection caused by SARS-CoV-2. Under the Company’s Public Health Solutions business segment, ongoing collaborations with Axel Lehrer, PhD of the Department of Tropical Medicine, Medical Microbiology and Pharmacology, JABSOM, UHM have demonstrated the feasibility of developing heat stable subunit filovirus vaccines, including hemorrhagic disease caused by Zaire ebolavirus, Sudan ebolavirus as well as Marburg marburgvirus, with both monovalent and bivalent vaccine combinations. Formulation conditions have been identified to enable heat stabilization of each antigen, alone or in combination, for at least 12 weeks at 40 degrees Celsius (104 degrees Fahrenheit). In March 2020, Soligenix and its collaborators expanded the technology platform to assess compatibility with vaccine candidates targeting SARS-CoV-2, the cause of COVID-19.

The vaccine platform includes three essential components:

a protein antigen, specifically a viral surface glycoprotein, which mediates entry and fusion of the virus with host cells and is manufactured with a proprietary insect cell expression system coupled with protein-specific affinity purification;
an adjuvant which has been shown to enhance both cell mediated and humoral immunity; and
a formulation which enables thermostabilization of the resulting mixture, avoiding the need for cold chain storage and shipping.
The resulting vaccine is broadly applicable, including to individuals often excluded from common viral vector vaccine approaches such as children, the elderly and the immunocompromised. The protection of elderly and immunocompromised populations are particularly important in the context of COVID-19. The ability to provide a thermostabilized, single vial vaccine, is particularly important in the context of rapid and broad vaccine distribution.

These same components are now being applied to coronavirus vaccine, using the well-defined surface glycoprotein, known as the Spike protein, as the antigen. Nonclinical work in mice with a prototype vaccine recently have been made available, demonstrating the ability of the CoVaccine adjuvant in combination with a prototype antigen, to:

stimulate immunity within 14 days after the first vaccination;
induce a balanced Th1 response, believed to be critical to inducing immunity without aggravating disease pathology;
induce a neutralizing antibody response; and
induce a cell mediated immune response.
CiVax development is being funded through an SBIR grant from NIAID (grant number 1 R44 AI157593-01).

BridgeBio Pharma, Inc. and GlycoNet Initiate Collaboration to Discover Potential Treatments for Genetic Diseases through Glycomics Research

On April 13, 2021 BridgeBio Pharma, Inc. (NASDAQ: BBIO) and the Canadian Glycomics Network (GlycoNet), a pan-Canadian Network of Centres of Excellence, reported a collaboration to translate scientific research in glycomics into potential treatments for patients with genetic diseases (Press release, BridgeBio, APR 13, 2021, View Source [SID1234578024]).

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"We are thrilled to be partnering with GlycoNet, an institution at the forefront of developing carbohydrate-based drugs to address areas of unmet need," said BridgeBio founder and CEO Neil Kumar, Ph.D. "We understand the clinical benefit research in glycomics could have for certain genetic diseases and by partnering together, we hope to advance potentially life-changing medicines as rapidly as possible."

BridgeBio will work alongside GlycoNet researchers to identify research programs that may have the potential to become treatments for genetic diseases. BridgeBio will potentially sponsor research programs and support clinical investigation through its licensing and affiliate development model. Genetic disease research has benefitted substantially from the study of glycomics, which relates to the functions of glycans, or sugar, in biological systems.

"The opportunity to partner with BridgeBio is invaluable," said GlycoNet CEO Elizabeth Nanak, Ph.D., MBA. "Our joint effort could potentially address areas of unmet needs and get treatments to patients more efficiently. We are hopeful that our network’s expertise in glycomics coupled with BridgeBio’s vigor to advance therapies into the clinic, will improve the quality of life of patients with genetic disorders."

BridgeBio partners with top academic and research institutions throughout the world, including GlycoNet, to support early, promising research. Today BridgeBio also announced formal partnerships with Brown University, The Lundquist Institute, Oregon Health & Science University, Roswell Park Comprehensive Cancer Center, University of California, Davis and University of California, San Diego – for a total of 20 partnerships between BridgeBio and leading academic and research institutions to-date. For a list of some of the institutions BridgeBio is partnered with, please visit Our Partners page.

As one of its guiding principles for drug development, BridgeBio believes in the importance of developing long-term partnerships through trust, respect and science in order to pioneer critical medicines for patients with genetically driven conditions as quickly and safely as possible.

Harbour BioMed Presents Novel Antibody for Cancer Immunotherapy at 2021 American Association for Cancer Research Annual Meeting

On April 13, 2021 Harbour BioMed ("HBM"; HKEX: 02142.HK), a global clinical-stage biopharmaceutical company, reported its newly discovered fully human anti-B7H7 monoclonal antibody at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, Harbour BioMed, APR 13, 2021, View Source [SID1234578000]). This meeting is being held virtually from April 10-15, 2021. The anti-B7H7 antibody is a novel immune-oncology antibody with potent anti-tumor activity generated using HBM’s proprietary H2L2 Harbour Mice platform.

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Details of the presentation:

Harbour BioMed’s presentation
By leveraging its innovative discovery engine based on the Harbour Mice antibody platforms, HBM has built a sustained capability to deliver novel antibody therapeutics in the field of oncology and immunology. Following the previously reported monoclonal antibody against a novel target CCR8, HBM now reports another first-in-class therapeutic antibody targeting a novel immune checkpoint – B7H7. B7H7 is mainly expressed in some PD-L1 negative/low tumors and may serve as an alternative immune escape pathway for these tumors. This antibody generated from H2L2 Harbour Mice demonstrates excellent preclinical activity and anti-tumor efficacy.

About B7H7

B7H7 is a novel immune checkpoint of B7 family, which is highly expressed on a variety of human cancers including colon, pancreatic, kidney, breast, bladder, lung cancers, etc. and is associated with metastatic disease and poorer survival rate of these patients. B7H7 antibody may present a novel anti-tumor therapy complementary to PD-L1/PD1 based therapy. B7H7 antibodies show significant T cell activation effect in vitro and tumor growth inhibition in several mouse tumor models.

Biomica Announces Positive Pre-Clinical Results, Demonstrating Efficacy of BMC128 in Melanoma

On April 13, 2021 Biomica Ltd., an emerging biopharmaceutical company developing innovative microbiome-based therapeutics and a subsidiary of Evogene Ltd. (NASDAQ: EVGN) (TASE: EVGN), reported additional positive pre-clinical results in its immuno-oncology program demonstrating efficacy of its live biotherapeutic product (LBP) consortium BMC128, this time in melanoma (Press release, Biomica, APR 13, 2021, View Source [SID1234577998]). In these studies, Biomica tested BMC128, which consists of four live bacterial strains, in a mouse model of melanoma.

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Dr. Elran Haber, CEO of Biomica, stated: "We are very excited with the results of this study demonstrating the effectiveness of BMC128 in treating additional types of solid cancer tumors. These positive pre-clinical results indicate the potential of BMC128 to become best-in-class in the treatment of solid cancer tumors, and help validate Biomica’s computational-based drug design approach. We look forward to providing incremental updates as we work towards a first-in-human, proof of concept clinical trial."

Treatment with BMC128 in combination with Immune Checkpoint Inhibitors (ICI) immunotherapy significantly enhanced anti-tumor activity, resulting in an increased response of melanoma tumors to anti-PD1, as demonstrated in an improved Objective Response Rate (ORR) and Percent Tumor Growth Inhibition (%TGI). The group treated with only anti-PD1 showed no response (ORR = 0%) as measured by the Response Evaluation Criteria in Solid Tumors (RECIST), while the group treated with a combination of BMC128 and anti-PD1 demonstrated a total of 13% response (ORR = 13%). The %TGI was increased by 100% in the BMC128 and anti-PD1 combination treated group compared to the group treated by anti-PD1 alone. Response to BMC128 was correlated with a desired anti-tumor immunological profile. BMC128 changed the course of response to ICI, leading to stimulation of the immune system which shifted cold-tumors into hot-tumors.

These positive results supplement previous pre-clinical data using BMC128 in combination with ICI in a breast cancer mouse model that demonstrated pronounced anti-tumor activity as manifested in an increase of almost 50% in ORR. The current results demonstrate the potential applicability of BMC128 and its relevance to treating multiple types of solid tumors.

Biomica’s immuno-oncology program is based on the premise that the gut microbiome affects the efficacy of cancer immunotherapy, specifically that of the ICI involving the blockade of PD-1 or PD-L1 and CTLA-4 as suggested in scientific literature.[1],[2] Fecal microbial transplantation has been recently reported to increase response in patients resistant to immune-checkpoint therapy[3],[4], however the specific microbial entities driving this response are currently unknown. BMC128 is a rationally-designed microbial consortium identified and selected through a detailed functional microbiome analysis using PRISM, a proprietary high-resolution microbiome analysis platform powered by Evogene’s MicroBoost AI platform.

As previously reported, Biomica has initiated scale-up processes and Good Manufacturing Practice (GMP) production of its drug candidate in its immuno-oncology program in preparation for the first-in-human proof-of-concept clinical trial expected later this year.

Mr. Ofer Haviv, Chairman of Biomica and Evogene President & CEO, stated: "We are proud of the results that Biomica reported today. These results support the computational biology capabilities developed by Evogene and Biomica which predicted that the microbes that make up BMC128 can be utilized to improve the efficacy of ICI in solid tumors. We look forward to validating the same computational forecasting capabilities in additional successful programs led by Biomica such as IBD and IBS."

[1] Zitvogel et al. 2018, Science 359 (6382)
[2] Thompson J, et al. Microbiome & immunotherapy: Antibiotic use is associated with inferior survival for lung cancer patients receiving PD-1 inhibitors. J Thorac Oncol 12(suppl 2):S1998, 2017
[3] Baruch E, et al. 2021. Fecal microbiota transplant promotes response in immunotherapy-refractory melanoma patients. Science, 371 (6529)
[4] Davar D, et al. 2021. Fecal microbiota transplant overcomes resistance to anti–PD-1 therapy in melanoma patients. Science, 371 (6529)

Iterion Therapeutics Confirms Safety of Tegavivint Following Completion of Enrollment in Phase 1/2a Expansion Study in Patients with Desmoid Tumors

On April 13, 2021 Iterion Therapeutics, Inc., a venture-backed, clinical stage biotechnology company developing novel cancer therapeutics, reported that it has confirmed the safety of Tegavivint, a novel, potent and selective nuclear beta-catenin inhibitor, after completing enrollment and dosing the final patient in a multicenter Phase 1/2a dose expansion clinical study of Tegavivint in patients with desmoid tumors (Press release, Iterion Therapeutics, APR 13, 2021, View Source [SID1234577997]).

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Nuclear beta-catenin is a highly-studied oncology target associated with numerous cancer types. Tegavivint is unique among nuclear beta-catenin inhibitors in that it binds to TBL1 (Transducin Beta-like Protein One), a novel downstream target in the Wnt-signaling pathway. As such, Tegavivint enables silencing of Wnt-pathway gene expression without affecting other Wnt/beta-catenin functions in the cell membrane, thus avoiding toxicity issues common to other drugs in this pathway.

The Phase 1/2a clinical trial of Tegavivint in patients with progressive desmoid tumors was designed as an open-label, non-randomized dose-finding study. The primary objectives of the study were to evaluate the safety and tolerability of Tegavivint. Secondary objectives were to determine the durability of response (DOR) to Tegavivint after the achievement of best response. The total study enrolled 24 patients. During the dose expansion portion of the trial 16 of these patients were treated with a recommended Phase 2 dose (RP2D) that was established based on pharmacokinetic exposure levels and clinical responses in a recently completed Phase 1 study.

Data from patients treated in the dose expansion portion of the trial reaffirmed Tegavivint’s safety at the RP2D level. No dose-limiting toxicities or significant adverse events were observed. This data will enable Iterion to accelerate clinical activity in additional cancer indications where nuclear beta-catenin signaling has been identified as a potential therapeutic target, including AML, NSCLC, and certain pediatric cancers. Iterion expects to initiate clinical programs investigating Tegavivint for these indications in 2021.

"We have seen very good tolerability with no dose-limiting toxicities and no significant adverse events in escalating clinical doses," said Casey Cunningham, Chief Medical Officer of Iterion. "We are seeing a very strong safety signal in patients who have been on Tegavivint for over a year and are also observing tumor activity in patients. We continue to follow the patients that are still receiving treatment and look forward to sharing efficacy results at an upcoming medical conference."

Rahul Aras, CEO of Iterion, stated: "The completion of enrollment in the dose expansion phase of our desmoid tumor clinical trial and demonstration of safety and clinical activity at the RP2D represent important milestones in our clinical development of Tegavivint. We look forward to advancing the clinical development of Tegavivint in desmoid tumors as this disease target is greatly underserved. The results of this study also provide a ‘green light’ to initiate clinical development of Tegavivint in additional, high-value cancer settings, including AML, NSCLC, and certain pediatric cancers, that are characterized by nuclear beta-catenin overexpression."

About Desmoid Tumors

Desmoid tumors are rare, non- metastasizing sarcomas that overexpress nuclear beta-catenin, a historically "undruggable" oncology target implicated in cell proliferation, differentiation and immune evasion. An estimated 1,500 patients in the US are newly diagnosed with desmoid tumors each year. Desmoids are most commonly diagnosed in young adults between 30-40 years of age and are associated with significant morbidities, including severe pain, disfigurement, internal bleeding and organ damage, range of motion loss and, in rare cases, death. Iterion has received Orphan Drug Designation for Tegavivint to treat desmoid tumors, a disease for which there are no FDA approved therapies.