On November 15, 2021 iBio, Inc. (NYSEA:IBIO) ("iBio" or the "Company"), a developer of next-generation biopharmaceuticals and pioneer of the sustainable FastPharming Manufacturing System, reported its financial results for the fiscal quarter ended September 30, 2021 and provides a corporate update (Press release, iBioPharma, NOV 15, 2021, View Source [SID1234595599]).

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"We are continuing to accelerate the execution of our growth strategy as we start our fiscal year 2022," said Tom Isett, Chairman & CEO of iBio. "Having only just launched our oncology initiative six months ago, we have already created six active programs, including the two new ones announced today. In addition, we have advanced our novel COVID-19 vaccine candidate to pre-IND, while concurrently building out our vaccine platform with new drug delivery technology partnerships. We have also acquired our cGMP manufacturing facility and taken sole ownership of our CDMO subsidiary, along with the attendant rights to manufacture biologics using our FastPharming System in the United States. We are confident that our investments over the past year in talent, technology, and infrastructure will return value to patients and shareholders as we go forward."

Fiscal First Quarter and Recent Business Developments:

BIOPHARMACEUTICALS

Therapeutics

In August 2021, iBio added to its pipeline by in-licensing an IL-2 sparing anti-CD25 antibody (IBIO-101) from RubrYc Therapeutics. Pre-clinical data shows that the molecule can deplete regulatory T cells from the tumor microenvironment and restore anti-tumor immunity, potentially representing an important new tool in the fight against cancer.
The Company continues to advance its endostatin E4 molecule for fibrotic diseases (IBIO-100) toward the clinic. Meanwhile, leveraging its investment in endostatin E4, iBio announced earlier today that it will separately explore the molecule’s usefulness in the treatment of solid tumors as part of a research collaboration with the University of Texas Southwestern Medical Center.
Pursuant to its new partnership with RubrYc Therapeutics wherein artificial intelligence ("AI")-based antibody discovery technologies are deployed, iBio announces today that the first new target is now being optimized using the platform. This result follows just two months after the joint discovery collaboration was initiated.
Vaccines

The Company recently conducted a preclinical dose ranging study of IBIO-202, its nucleocapsid ("N") antigen-based, intramuscularly ("IM")-delivered COVID-19 vaccine candidate. The results confirmed generation of a robust, antigen-specific, memory T-cell response and provided data that further informed iBio’s product formulation strategy.
The Company submitted a pre-IND package for IBIO-202 to the U.S. Food and Drug Administration in September 2021 and is anticipating a response in the coming months.
Today, iBio announces that is has entered a collaboration with a leading innovator of microarray patch systems, which are a painless alternative to IM injections. The first objective of the collaboration is to evaluate feasibility of intradermal delivery of a COVID-19 vaccine antigen. If successful, the partnership has the potential to drive improved access to vaccines by avoiding cold chain issues and possibly enabling self-administration. The collaboration is not expected to impact the current development timeline of IBIO-202.
The Company continues to advance its Classical Swine Fever ("CSF") vaccine candidate, IBIO-400, through the U.S. Department of Agriculture’s Centers for Veterinary Biologics’ regulatory process. Additionally, iBio announces today that studies are now underway at Texas A&M University System to evaluate alternatives to IM injection of its CSF vaccine candidate, including an oral dose option.
BIOPROCESS

In November 2021, iBio purchased the Bryan, Texas, manufacturing facility it previously operated under a lease from two affiliates of Eastern Capital Limited (the "Eastern Affiliates"). The Company also acquired the equity interest in iBio CDMO, LLC, that was formerly held by the Eastern Affiliates. As a result, the subsidiary and its intellectual property are now wholly-owned by iBio.
Fiscal First Quarter and Recent Corporate Developments:

iBio made progress in government relations during the quarter by engaging with senior-level U.S. policymakers to build awareness of the FastPharming System and to underscore the benefits of rapid, sustainable, domestic bioproduction.
iBio is encouraging shareholders who held common stock at the close of business on October 15, 2021 to vote "FOR" all proposals put forth in the proxy statement prior to its December 9, 2021, Annual Meeting of Stockholders to help the Company continue to grow its pipeline, services, and value.
"Over the course of the next year, we are focused on delivering shareholder value across multiple fronts, including announcing new oncology targets and two possible IND filings," said Mr. Isett. "We are also excited about the partnership opportunities that we see ahead for our bioprocess business as industry awareness of the need to implement sustainable, ESG-based practices continues to grow. With sole ownership of our FastPharming Facility and CDMO subsidiary now secured, we have additional strategic and operational flexibility to deploy our rapid, scalable biologics development system for our own pipeline, as well as for clients, so that we may enable more potentially life-saving therapeutics to enter the clinic."

Financial Results:

Revenues for the first fiscal quarter ended September 30, 2021, were approximately $211,000, a decrease of 49% from approximately $410,000 in the same period of 2020. As noted previously, significant quarter-to-quarter revenue variability is commonplace for early-stage pharma services companies given the timing of revenue recognition. Consistent with these business dynamics, iBio continues to expect a sequential decline in revenue during the first half of fiscal 2022 compared to the second half of fiscal 2021, followed by higher growth in the second half of fiscal 2022.

R&D and G&A expenses for the first quarter of fiscal 2022 increased 35% and 24%, respectively, over the comparable period in fiscal 2020. This reflects the Company’s growing investments in its pipeline, platform technologies, employees, and related infrastructure. iBio anticipates this trend continuing, however, the rate of growth is expected to moderate over time.

iBio’s consolidated net loss for the first quarter ended September 30, 2021, was approximately $8.9 million, or $0.04 per share, compared to a net loss of approximately $7.5 million, or $0.05 per share, in the same period of 2020.

iBio had $82.3 million dollars in cash and cash equivalents and investments in debt securities as of September 30, 2021. The Company used $9.3 million in cash during the quarter for operating activities and $5.1M for investing activities which included elements of the RubrYc transaction and for capital expenditures. Subsequent to the end of the first quarter of fiscal 2021, iBio used approximately $6.0 million in cash to help fund the purchase of its manufacturing facility in Bryan, Texas, and to secure full control of iBio CDMO, LLC, which holds the exclusive rights to manufacture using the FastPharming System in the United States. Taking into account potential long-term financing options, combined with the approximate 67% savings in facility-related cash requirements expected to be achieved through this transaction, the Company continues to believe that its current cash position is sufficient to fund its operations through Q3-FY23. If the Company cannot take advantage of the additional financial flexibility, and based on the Company’s working capital on September 30, 2021, management has concluded that there is sufficient liquidity to fund normal operations through at least Q2-FY23.

Webcast and Conference Call

iBio management will host a webcast and conference call at 4:30 p.m. Eastern Time today, November 15, 2021, to discuss these results and provide a corporate update.

The live and archived webcast may be accessed on the Company’s website at www.ibioinc.com under "News and Events" in the Investors section. The live call can be accessed by dialing (833) 672-0651 (domestic) or (929) 517-0227 (international) and referencing conference code: 7779281.

On November 15, 2021 TransCode Therapeutics, Inc. (Nasdaq: RNAZ), an RNA oncology company created on the belief that cancer can be defeated through the intelligent design and effective delivery of RNA therapeutics, reported recent business progress and third quarter 2021 financial results (Press release, TransCode Therapeutics, NOV 15, 2021, View Source [SID1234595598]).

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"TransCode has made further progress across our corporate infrastructure and bourgeoning portfolio, advancing our mission to design RNA therapeutics that can effectively treat cancer. Reflecting the results of an important microdosing study recently reported in Cancer Nanotechnology, TransCode believes it has an opportunity in its planned First-in-Human (FIH) Phase 0 clinical trial of its lead therapeutic candidate, TTM-MC138, to obtain results that will better inform designs of planned follow-on trials than the original FIH trial design. The reported study was conducted by Dr. Zdravka Medarova, a scientific co-founder of TransCode Therapeutics who was appointed Chief Technology Officer effective October 1," said Michael Dudley, co-founder, president and CEO of TransCode Therapeutics. Dr. Medarova noted, "We believe a microdosing study offers the potential to more definitively establish proof-of-mechanism for our delivery platform, upon which we hope to build a broad and diverse pipeline of therapeutics and diagnostics with the potential to reach previously undruggable genetic targets."

Dudley added, "We are striving to file our exploratory Investigational New Drug Application (eIND) required for the FIH trial by the end of the first quarter 2022, although because of COVID-19-related backlogs at contract research organizations, the filing may be later in the first half. Regardless, we believe the proposed microdosing study should, if successful, demonstrate the power and versatility of our TTX platform in solving the challenges of RNA delivery in oncology. In addition, we have continued IND-enabling work for TTX-MC138 and remain on track to file an IND for a Phase I clinical trial for treatment of metastatic disease by year end 2022."

Third Quarter 2021 and Recent Highlights

Expanded the Company’s executive team with the appointment of TransCode’s scientific co-founder, Zdravka Medarova, Ph.D., as Chief Technology Officer effective October 1. Dr. Medarova is a leader in the field of non-coding RNA delivery to cancer and has authored multiple high-impact publications on the topic of RNA delivery, nanotechnology, and the biology of cancer metastasis.
Continued eIND-enabling activities for TTX-MC138, the Company’s lead program, targeting micoRNA-10b (miR-10b) for treatment of metastatic solid tumors. Due to continued COVID-19-related backlogs at contract research organizations, the planned eIND filing may be in the second quarter 2022. Concurrently, IND-enabling efforts remain on track to support an IND expected to be filed in 2H 2022 for a planned Phase 1 study of TTX-MC138.
Announced publication of preclinical results supporting TTX-MC138 in Cancer Nanotechnology, based on research conducted at the Athinoula A. Martinos Center for Biomedical Imaging in the Department of Radiology at Massachusetts General Hospital and Harvard Medical School. The key results of the study demonstrated that a radiolabeled derivative of TTX-MC138, when injected intravenously, accumulated in metastatic lesions, confirming its intended pharmacokinetic profile.
Based on joint research conducted at Michigan State University and the Athinoula A. Martinos Center for Biomedical Imaging in the Department of Radiology and Massachusetts General Hospital and Harvard Medical School, additional preclinical data were published in Oncotarget highlighting the potential for microRNA-based therapies in glioblastoma multiforme (GBM). The publication included in vivo animal studies confirming the inhibitory effect of TTX-MC138 on the growth of stem cell-derived orthotopic GBM xenografts, suggesting miR-10b may represent a useful target in GBM therapy.
Conversion of the Company’s convertible promissory notes into common stock upon completion of the initial public offering (IPO) in July.
Key Planned Upcoming Milestones

TransCode’s goals to continue to advance its portfolio include:

TTX-MC138
Submission to FDA of an eIND application by second quarter 2022.
Initiation of a FIH Phase 0 clinical study evaluating TTX-MC138 for treatment of metastatic solid tumors later in 2022.
Concurrent completion of IND-enabling studies to support a second half 2022 filing of an IND application for a Phase I clinical trial of TTX-MC138.
Publication in the first half of 2022 of preclinical results supporting the TTX delivery platform in pancreatic cancer and glioblastoma multiforme.
Filing of additional patents related to new and current technologies.
Continuation of pre-clinical studies for therapeutic candidates TTX-RIGA, TTX-siPDL1 and TTX-siLin28b
Third Quarter Financial Highlights

Cash and Cash Equivalents: As of September 30, 2021, cash and cash equivalents totaled approximately $22.5 million largely reflecting the net proceeds from the July IPO.
R&D Expenses: Research and development expenses were approximately $993 thousand in the third quarter of 2021, compared to approximately $54 thousand in the third quarter of 2020. The increase was primarily due to purchases of materials, compensation costs which the Company did not have prior to the third quarter of 2021 except for stock-compensation expenses, license fees, intellectual property expenses, and lab facility expenses.
G&A Expenses: General and administrative expenses were approximately $1.4 million in the third quarter of 2021, compared to approximately $186 thousand in the third quarter of 2020. The increase was primarily due to increased liability insurance costs, compensation and related personnel costs which the Company did not have in the 2020 period except for stock- compensation expenses, investor relations and other costs of being a public company, and expenses for legal, accounting and tax services.
Operating Loss: Operating loss was approximately $2.4 million in the third quarter of 2021, compared to approximately $239 thousand in the third quarter of 2020. Net loss for the 2021 third quarter was approximately $2.3 million, or $0.20 per basic and diluted share, compared to a net loss of approximately $1.4 million, or $0.30 per basic and diluted share, for the third quarter of 2020. The Company expects that operating losses will increase substantially in the foreseeable future.
Financial Guidance

TransCode expects that its cash and cash equivalents as of September 30, 2021, together with additional funding expected from an April 2021 SBIR award, are sufficient to fund planned operations through year-end 2022.

On November 15, 2021 Phosplatin Therapeutics Inc., a clinical stage pharmaceutical company focused on oncology therapeutics, reported that it has entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the National Institutes of Health (Press release, Phosplatin, NOV 15, 2021, View Source [SID1234595597]). Under the terms of the agreement, Phosplatin and NCI will collaborate on the clinical development of Phosplatin’s lead therapeutic candidate, PT-112, for patients with recurrent thymic epithelial tumors (TETs), specifically thymoma and thymic carcinoma, which are rare cancers with no FDA approved therapy.

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Phosplatin and NCI anticipate opening the Phase 2 study soon, under a recently approved investigational new drug (IND) application. The study is designed to further assess the safety and efficacy of PT-112 in patients with thymoma and thymic carcinoma, and to use correlative studies to explore molecular profiles, examine parameters of immune activation and analyze immune cell infiltration in response to PT-112 treatment, as PT-112 is known to promote immunogenic cell death (ICD) in relevant cancer models. Phosplatin will provide NCI with PT-112 drug supply and support correlative research, and NCI will oversee enrollment and care of the study’s intended 49 patients.

"Thymoma and thymic carcinoma are rare forms of cancer for which there is currently no FDA approved treatment. There remains a pressing need for novel therapies for relapsed and refractory TETs – induction of immunogenic cell death is an approach of great interest for this population with no approved immunotherapy treatment," said Arun Rajan, MD, Senior Clinician in the Thoracic and GI Malignancies Branch at the National Cancer Institute. "This clinical trial will evaluate PT-112 as a potential treatment option for our patients with TETs, and we hope to better understand PT-112’s therapeutic potential through this collaborative research program."

The collaboration is based upon early experience with PT-112 in a number of thymoma patients. "In our first Phase 1 trial of PT-112, we observed a durable clinical response in a thymoma patient with advanced metastatic disease, which deepened over many months with symptomatic improvement for the patient," said Joseph F. O’Donnell, MD, Chief Medical Officer at Phosplatin. "Along with our Phase I safety data, and our demonstration of PT-112’s ICD effects in validated models, this case helped establish the interest in this collaboration. We’re proud to be working with the exceptional colleagues at the NCI in evaluating PT-112 in a patient population that currently has no approved therapeutic options for metastatic disease."

Phosplatin holds an FDA Orphan Drug Designation for PT-112 in thymoma and thymic carcinoma. TETs, including thymomas and thymic carcinomas, are uncommon tumors of the thymus for which there is no approved FDA-approved drug. In cases of relapse following surgical intervention, TETs have the potential to metastasize and there are limited options for treatment.

Further information on this clinical trial (NCT05104736) can be found at clinicaltrials.gov.

About PT-112

PT-112 is the first small-molecule conjugate of pyrophosphate in oncology, and possesses a unique pleiotropic mechanism of action that promotes immunogenic cell death (ICD), through the release of damage associated molecular patterns (DAMPs) that bind to dendritic cells and lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 may represent the best-in-class inducer of this immunological form of cancer cell death. Further, PT-112 harbors a property known as osteotropism, or the propensity of the drug to reach its highest concentrations in certain areas of the bone, making it a candidate for treatment of patients with cancers that originate in, or metastasize to, the bone. The first in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses among heavily pre-treated patients and won "Best Poster" within the Developmental Therapeutics category at the ESMO (Free ESMO Whitepaper) 2018 Annual Congress. The combination Phase 1b dose escalation study of PT-112 with PD-L1 checkpoint inhibitor avelumab in solid tumors was reported in an oral presentation at the ESMO (Free ESMO Whitepaper) 2020 Virtual Congress. The Phase 1 study in patients with relapsed or refractory multiple myeloma presented at ASH (Free ASH Whitepaper) is the third completed Phase 1 study of PT-112. Monotherapy Phase 2 development is ongoing in mCRPC, and the PD-L1 combination study is ongoing in a dose confirmation cohort of non-small cell lung cancer (NSCLC) patients, and will soon include the Phase 2 proof of concept study in thymic epithelial tumors under the company’s collaboration with the NCI.

On November 15, 2021 iBio, Inc. (NYSEA:IBIO) ("iBio" or the "Company"), a developer of next-generation biopharmaceuticals and pioneer of the sustainable FastPharming Manufacturing System, reported a research collaboration with the University of Texas Southwestern Medical Center ("UT Southwestern") to explore in solid tumors the anti-cancer potential of the molecule that is part of the IBIO-100 program (Press release, iBioPharma, NOV 15, 2021, View Source [SID1234595596]).

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Among all the stromal cells that present in the tumor microenvironment, cancer-associated fibroblasts ("CAFs") are one of the most abundant and critical components of tumor tissue, which provide physical support for tumor cells and can promote or retard tumorigenesis in a context-dependent manner. CAFs are also involved in the modulation of many components of the immune system, and recent studies have revealed their roles in immune evasion and poor responses to cancer immunotherapy.1 In addition, CAF response to chemotherapy is highly variable.2

Through a series of planned in vitro and in vivo studies, the collaboration will evaluate the potential of the anti-fibrotic effects of iBio’s endostatin E4 molecule to improve the efficacy of concomitant treatments, such as chemotherapy and immunotherapy, in cancer models with a fibrotic component. The Company is currently developing endostatin E4 as IBIO-100 for fibrotic diseases.

"We are thrilled to combine our efforts with one of the world’s leading fibrotic tumor cancer research labs at one of the premier academic medical centers in the nation," said Tom Isett, Chairman and Chief Executive Officer at iBio. "IBIO-100 has shown strong therapeutic potential in preclinical models of two major fibrotic diseases, systemic scleroderma and idiopathic pulmonary fibrosis, and we look forward to exploring these same potentially transformative benefits in the treatment of solid tumors."

"Through many years of research, we have come to understand that an overabundant fibrotic tumor microenvironment is associated with poor cancer treatment outcomes," commented Martin Brenner, DVM. Ph.D., iBio’s Chief Scientific Officer. "We have also learned that destruction of CAFs can lead to worse prognosis, not better.3 We believe that our endostatin E4 molecule has the potential to normalize fibrosis without the detrimental effects of CAF destruction, thereby improving responses to current standard of care treatments such as chemotherapy and immunotherapy. We look forward to exploring this potential with our partners at UT Southwestern."

On November 15, 2021 NKGen Biotech, a subsidiary of NKMax, harnessing the power of the body’s immune system through the development of Natural Killer (NK) cell therapies, reported that data from a Phase 1/2a clinical trial, sponsored by NKMax, evaluating SNK01 (autologous natural killer cells) plus pembrolizumab in Non-Small Cell Lung Cancer (NSCLC) patients in Korea was presented at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) held November 10 – 14, 2021, in Washington, D.C (Press release, NKMax America, NOV 15, 2021, View Source [SID1234595595]).

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The poster "Phase I/IIa randomized trial evaluating safety and efficacy of SNK01 plus pembrolizumab in patients with Stage IV Non-Small Cell Lung Cancer (NSCLC) who have failed first-line platinum-based therapy" was presented by NKMax on November 10th at the Industry Program Reception.

"We are extremely pleased to share promising clinical data showing safety and superior efficacy of our SNK01 autologous NK cell therapy in combination with pembrolizumab," said Sangwoo Park, Chief Executive Officer of NKGen Biotech. "The results of the trial demonstrate that SNK01 can be safely combined with checkpoint inhibitors, resulting in meaningfully improved clinical outcomes, especially compared to the checkpoint inhibitor alone. We look forward to seeing similar results in other solid tumor settings as we continue the clinical development of SNK01."

In this trial, eighteen patients with advanced Stage IV NSCLC who failed prior frontline platinum-based therapy were randomized 2:1 to received pembrolizumab every three weeks with +/- 6 weekly infusions of SNK01. Two dosing groups for SNK01 were evaluated in combination with pembrolizumab: 2×109 cells/dose and 4×109 cells/dose. The primary endpoint was safety and secondary endpoints were objective response rate (ORR), progression-free survival (PFS), overall survival, and quality of life.

The median PFS was superior in the SNK01 combination group and results were statistically significant (6.2 months vs. 1.6 months; p = 0.001). Furthermore, a dose response was noted as PFS in the higher 4×109 cells/dose SNK01+ pembrolizumab combination arm was 9.4 months compared to 1.6 months for the pembrolizumab only group. No new safety signals were seen when SNK was added to pembrolizumab.

In this trial, combination of SNK01 and pembrolizumab enhanced antitumor activity in advanced NSCLC patients, and suggests possible utilization of combining SNK01 with pembrolizumab. NKGen Biotech/NKMax will continue to explore SNK01 in various tumor settings, including the combination approach with different modalities.