On August 21, 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 its recent accomplishments and financial results for the quarter ended June 30, 2023 (Press release, Soligenix, AUG 21, 2023, View Source [SID1234634599]).

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"During the second quarter we advanced multiple clinical trials, while continuing our collaborative discussions with the U.S. Food and Drug Administration (FDA) regarding the design of a second, confirmatory Phase 3 pivotal study evaluating HyBryte (synthetic hypericin sodium) in the treatment of cutaneous T-cell lymphoma (CTCL), where we successfully demonstrated statistically significant results in the first Phase 3 clinical trial," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "A HyBryte expanded treatment CTCL trial at the University of Pennsylvania was recently opened for patient enrollment, supported by a $2.6 million Orphan Products Development grant award from the FDA. We are continuing to actively enroll patients in the Phase 2a trial of SGX302 (synthetic hypericin sodium) for the treatment of mild-to-moderate psoriasis after demonstration of a clear biological signal in all five of the initial patients, with the majority recording an improvement in their PASI (psoriasis area and severity index) score. Additionally, we announced positive clinical results from a compatibility study evaluating HyBryte in the treatment of CTCL that confirmed and extended response results from the first Phase 3 study conducted."

Dr. Schaber continued, "During the second quarter of 2023, we completed a public offering with gross proceeds of approximately $8.5 million, which has allowed us to continue to move our rare disease pipeline forward. With approximately $13.2 million in cash at June 30, 2023, we are managing cash burn very carefully in order to achieve our near-term milestones, as we continue to assess all strategic options, including but not limited to, partnership and merger and acquisition opportunities."

Soligenix Recent Accomplishments

On August 10, 2023, the Company announced that patient enrollment had been opened for the HyBryte expanded treatment investigator-initiated CTCL study at the University of Pennsylvania, supported by a $2.6 million Orphan Products Development grant award from the FDA. To view this press release, please click here.
On July 11, 2023, the Company announced the expansion of its Phase 2a trial of SGX302 for the treatment of mild-to-moderate psoriasis after demonstration of biological effect in all five of the initial subjects. To view this press release, please click here.
On May 16, 2023, the Company announced the European Patent Office had granted a patent entitled "Novel Peptides and Analogs for Use in the Treatment of Oral Mucositis". To view this press release, please click here.
Financial Results – Quarter Ended June 30, 2023

Soligenix’s revenues for the quarters ended June 30, 2023 and 2022 were $0.2 million. Revenues primarily relate to third party licensing and the government contracts and grants awarded in support of HyBryte, our therapeutic candidate for early-stage CTCL; SGX943, our therapeutic candidate for treatment of emerging and/or antibiotic-resistant infectious diseases; ThermoVax, our thermostabilization technology; and CiVax, our vaccine candidate for the prevention of COVID-19.

Soligenix’s net loss was $1.6 million, or ($0.22) per share, for the quarter ended June 30, 2023, as compared to $2.4 million, or ($0.83) per share, for the quarter ended June 30, 2022. The decrease in net loss was primarily due to a decrease in operating expenses.

Research and development expenses were $0.8 million as compared to $2.0 million for the quarters ended June 30, 2023 and 2022, respectively. The decrease was primarily due to the decrease in manufacturing and regulatory costs associated with the HyBryte new drug application filing.

General and administrative expenses were $0.9 million and $1.4 million for the quarters ended June 30, 2023 and 2022, respectively. This decrease in general and administrative expenses is primarily attributable to a reduction in legal and consulting expenses associated with the arbitration against Emergent BioSolutions, Inc. and certain of its subsidiaries.

As of June 30, 2023, the Company’s cash position was approximately $13.2 million.

On August 21, 2023 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL RNAi platform technology is designed to make immune cells more effective in killing tumor cells, reported that its clinical development partners, AgonOx, Inc. and Providence Cancer Institute of Oregon, have dosed their first patient in an Adoptive Cell Therapy clinical trial (Press release, Phio Pharmaceuticals, AUG 21, 2023, View Source [SID1234634598]).

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This first-in-human trial is investigating the safety and the potential for enhanced therapeutic benefit from the administration of AgonOx’s AGX148 ‘double positive’ (DP) CD8 tumor-infiltrating lymphocytes (TIL) alone and in combination with Phio’s PD-1 silencing PH-762 in patients with melanoma and other advanced solid tumors. More information about this clinical trial is available at clinicaltrials.gov. The clinical trial is one of two studies cleared by the FDA which will utilize Phio’s lead product candidate, PH-762.

The trial is being conducted at Providence Cancer Institute in Portland, Oregon, by Principal Investigator Brendan Curti, M.D., medical oncologist and Robert W. Franz Endowed Chair for Clinical Research at the Earle A. Chiles Research Institute, a division of Providence. "Our institute has been at the leading edge of immunotherapy clinical research including checkpoint immunotherapy, novel co-stimulatory agents and cancer vaccines," said Dr. Curti. "Our vision of the future is to select and engineer immune cells with enhanced abilities to attack tumors. We have a strong record of accomplishment in translating laboratory ideas into clinical practice. The work with Dr. Weinberg is another example of exciting first-in-human translational science from our Institute and its many ongoing collaborations."

"TIL therapies hold the promise of clinical benefit in patients with solid malignancies. This product is different from current TIL therapies as we are selectively expanding the tumor-reactive T cells and potentially increasing their potency by reducing PD-1 suppression," said Andrew Weinberg, Ph.D., Founder and CSO of AgonOx, Inc., and Judith Ann Hartmann Endowed Chair for the Laboratory of Basic Immunology at the Earle A. Chiles Research Institute.

"This is an important collaborative milestone between these organizations in the advancement of treatments for solid tumors," said Robert Bitterman, President and CEO of Phio Pharmaceuticals.

On August 21, 2023 Moleculin presented its corporate presentation (Presentation, Moleculin, AUG 21, 2023, View Source [SID1234634597]).

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On August 21, 2023 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported that the United States Food and Drug Administration (FDA) has accepted the filing of Geron’s New Drug Application (NDA) for imetelstat, its first-in-class telomerase inhibitor, for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS) (Press release, Geron, AUG 21, 2023, View Source [SID1234634596]).

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"The FDA’s acceptance of our New Drug Application is an important landmark along our steadfast journey to bring telomerase inhibition with imetelstat to the market," said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. "We look forward to continuing our collaboration with the FDA toward the goal of bringing imetelstat to the many patients for whom we believe this treatment could make a significant difference."

"FDA acceptance of our NDA is a significant milestone for both Geron and the MDS community, as there remain few treatment options and significant unmet needs, particularly for patients with difficult-to-treat subtypes of this cancer," said Faye Feller, M.D., Executive Vice President, Geron’s Chief Medical Officer. "We believe that the IMerge Phase 3 data reflect the truly unique attributes of imetelstat, and, if approved, we expect imetelstat will change the standard of care in lower risk MDS."

The NDA submission is based on results from IMerge Phase 3, in which the primary endpoint of 8-week transfusion independence (TI) was significantly higher with imetelstat vs. placebo (p<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders. Mean hemoglobin levels in imetelstat-treated patients increased significantly (p<0.001) over time compared to placebo patients. Statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category. Patient-reported outcomes (PRO) data reported a sustained meaningful improvement in fatigue for imetelstat-treated patients vs. placebo. Safety results were consistent with prior imetelstat clinical experience.

As allowed under the 21st Century Cures Act, the FDA has up to 74 days from the NDA submission date to notify Geron of the Prescription Drug User Fee Act (PDUFA) action date for the NDA. Upon receipt of this notification, Geron plans to disclose the timeline for the NDA review.

Additionally, Geron expects to submit a Marketing Authorization Application (MAA) in the European Union (EU) in the fourth quarter of 2023.

About IMerge Phase 3

The Phase 3 portion of the IMerge Phase 2/3 study is a double-blind, 2:1 randomized, placebo-controlled clinical trial to evaluate imetelstat in patients with IPSS Low or Intermediate-1 risk (lower risk) transfusion dependent MDS who were relapsed after, refractory to, or ineligible for, erythropoiesis stimulating agent (ESA) treatment, had not received prior treatment with either a HMA or lenalidomide and were non-del(5q). To be eligible for IMerge Phase 3, patients were required to be transfusion dependent, defined as requiring at least four units of packed red blood cells (RBCs), over an eight-week period during the 16 weeks prior to entry into the trial. The primary efficacy endpoint of IMerge Phase 3 is the rate of red blood cell transfusion independence (RBC-TI) lasting at least eight weeks, defined as the proportion of patients without any RBC transfusion for at least eight consecutive weeks since entry to the trial (8-week TI). Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks (24-week TI), the duration of TI and the rate of hematologic improvement erythroid (HI-E), which is defined under 2006 IWG criteria as a rise in hemoglobin of at least 1.5 g/dL above the pretreatment level for at least eight weeks or a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden. A total of 178 patients were enrolled in IMerge Phase 3 across North America, Europe, Middle East and Asia.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic malignancies. Data from non-clinical studies and clinical trials of imetelstat provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the U.S. Food and Drug Administration for both the treatment of adult patients with transfusion dependent anemia due to Low or Intermediate-1 risk MDS that is not associated with del(5q) who are refractory or resistant to an erythropoiesis stimulating agent, and for adult patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment. Imetelstat is currently not approved by any regulatory authority.

On August 21, 2023 AskGene Pharma Inc. reported that the first patient has been dosed at Columbia University Irving Medical Center in New York City in the phase I clinical trial of ASKG915 (Press release, AskGene Pharmaceuticals, AUG 21, 2023, View Source [SID1234634595]). The study is an open label, multi-regional phase I clinical trial evaluating the safety, tolerability, and pharmacokinetics of ASKG915 as a single agent in patients with selected advanced solid tumors. ASKG915 is the first anti-PD-1/IL-15 prodrug in clinical development in the world.

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Barbara Hickingbottom, J.D., M.D., Chief Medical Officer of AskGene commented: "Cytokines are natural immune stimulants, which cannot be used widely due to their short half-lives and narrow therapeutic windows. AskGene uses its proprietary technology of SmartKine to overcome these challenges. We already have on-going clinical studies of ASKG315 (an IL-15 prodrug) and an IL-2 prodrug through a partnership. With ASKG915, we expand our platform to include a molecule that should produce both full anti-PD-1 and IL-15 effects. Such molecules may be able to treat both patients who have failed anti-PD-1 treatment, as well as those who need the extra boost of IL-15 to achieve a response to checkpoint inhibitor therapy. FIH dosing of ASKG915 is an important milestone towards making this next-generation immunotherapy available to patients in need. We are very excited about the progress made so far and look forward to the upcoming POC data from our clinical studies in the future."

About ASKB915

ASKG915 is the world’s first anti-PD-1 antibody / IL-15 prodrug fusion molecule to enter clinical development. The molecule can achieve tumor targeting through the anti-PD-1 antibody and be locally activated at the tumor site. It is also designed to allow a high enough dose so that the anti-PD-1 antibody has full PD-1 blockage functionality. Preclinical data showed that ASKG915 has extended PK, significantly better efficacy than anti-PD-1 antibody monotherapy, and an expanded therapeutic window. ASKG915 is expected to benefit patients who are not responding to current anti-PD-1 antibody therapies. A multi-regional phase I clinical trial in the US and China was started in Aug 2023.