On May 13, 2024 iBio, Inc. (NYSEA:IBIO) ("iBio" or the "Company"), an AI-driven innovator of precision antibody immunotherapies, reported financial results for the third quarter ended March 31, 2024 and provided a corporate update (Press release, iBioPharma, MAY 13, 2024, View Source [SID1234643142]).

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"In our third fiscal quarter, we continued to advance toward our goal of becoming a leading antibody discovery company with a differentiated machine-learning platform," said Dr. Martin Brenner, CEO and Chief Scientific Officer. "Along with our recent equity financing, we secured a transformative partnership with AstralBio to discover and develop novel antibodies to treat obesity and other cardiometabolic diseases, with a lead program focused on targeting the transforming growth factor beta (TGFb) superfamily for treating muscle wasting and obesity. Our enhanced financial position has permitted us to accelerate the AstralBio collaboration, and we’ve already begun recruiting for key positions to support the process of identifying new molecules that are expected to simultaneously expand and diversify our pipeline. We believe we are well-positioned to advance our internal pipeline priorities in immuno-oncology and cardiometabolics, and drive partnerships in new therapeutic areas."

Fiscal Third Quarter 2024 & Recent Corporate Updates:

Entered into a securities purchase agreement for a fully subscribed private investment in public equity ("PIPE") financing with several institutional investors and an accredited investor in March, and consummated the financing in April for gross proceeds of approximately $15.0M before deducting placement agent fees and offering expenses.
Entered into a collaboration with AstralBio, Inc. to provide an exclusive license in the cardiometabolic and obesity space. iBio will develop four targets of interest, with the Company having the rights to license up to three of these targets prior to entering the clinic.
In February, iBio closed the sale of its early-stage PD-1 asset to Otsuka Pharmaceutical Co., Ltd. ("Otsuka") for $1M in upfront cash with contingent downstream payments of up to $52.5M, a pivotal moment that showcased the power of iBio’s platform to discover best-in-class assets.
Strengthened iBio’s cash position after previously issued warrants were exercised for proceeds of approximately $4.3M.
Appointed iBio’s CEO and Chief Scientific Officer, Dr. Martin Brenner, to the Board of Directors, effective June 1, 2024.
Presented data on the company’s technology stack, demonstrating how its machine learning (ML)-driven epitope steering and mammalian-display antibody libraries efficiently discover diverse T-cell engager arms tuned for potency, toxicity, developability, and cyno cross-reactivity at the 24th annual PepTalk conference.
Fiscal Third Quarter 2024 Financial Results:

No revenue was reported for the third quarter ended March 31, 2024.
R&D and G&A expenses for the third quarter of fiscal 2024 decreased approximately 66% and 23%, respectively, over the comparable period in fiscal 2023, reflecting the Company’s cash preservation strategy and focus on collaboration partnerships. Net loss from continuing operations for the third quarter ended March 31, 2024, was approximately $2.6 million, or $0.71 per share, compared to a net loss of approximately $6.3 million, or $9.53 per share, in the same period of 2023.
Cash, cash equivalents and restricted cash as of March 31, 2024 was approximately $6.4 million, inclusive of $1.1 million of restricted cash. On April 1, 2024, iBio received net proceeds of approximately $14.1 million after consummation of the PIPE financing. As of today’s filing, the Company has approximately $17.9 million in cash, cash equivalents and restricted cash.

On May 13, 2024 Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, reported financial results for the first quarter ended March 31, 2024, and provided an overview of recent operational highlights (Press release, TONIX Pharmaceuticals, MAY 13, 2024, View Source [SID1234643159]).

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"Our near-term priority continues to be the submission of our New Drug Application (NDA) for Tonmya (cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia, while continuing to build out our commercial strategy for the anticipated product launch in the event of FDA approval, which we currently estimate to occur in the second half of 2025," said Seth Lederman, M.D., Chief Executive Officer of Tonix.

Dr. Lederman added, "The well-known treatment-limiting side effects of the three currently approved drugs have led to widespread patient dissatisfaction, creating what we believe is a significant opportunity for a new therapeutic. Tonmya has a differentiated mechanism of action and is generally free of common side effects associated with the currently approved products, including weight gain, fatigue, insomnia, increased blood pressure, gastrointestinal issues or sexual dysfunction. As such, we believe Tonmya, if approved, could become the treatment of choice for the approximately 10 million people in the U.S. suffering the debilitating effects of fibromyalgia."

The Company is also advancing other key pipeline programs including those for immunology, obesity, eating disorders, infectious and rare diseases, many through a capital efficient strategy involving partnerships, grants and in-kind contributions.

Recent Highlights – Key Product Candidates*

Central Nervous System (CNS) Pipeline

Tonmya (also known as TNX-102 SL; cyclobenzaprine HCl sublingual tablets): a centrally-acting, non-opioid, small molecule analgesic taken once-daily at bedtime for the management of fibromyalgia (FM).

In January 2024, Tonix presented additional safety and tolerability data from the pivotal Phase 3 RESILIENT study that showed Tonmya treatment was not associated with increases in systolic or diastolic blood pressure or body weight, nor were there any reported sexual side effects. The Company had previously announced in December 2023 that the Phase 3 RESILIENT study, a registration-quality, double-blind, placebo-controlled study evaluating Tonyma met its pre-specified primary endpoint in the second of two positive Phase 3 clinical trials, significantly reducing daily pain compared to placebo (p-value=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results were also seen in all pre-specified key secondary endpoints including those related to improving sleep quality, reducing fatigue, and improving patient global ratings and overall fibromyalgia symptoms and function. Tonmya was well tolerated with an adverse event profile comparable to prior studies and no new safety signals observed. In addition, Tonmya therapy showed activity on improving female sexual function relative to placebo with a nominal p-value=0.010 by the Changes in Sexual Functioning Questionnaire short-form, female version.
Tonix plans to submit an NDA to the FDA in the second half of 2024 for Tonmya for the management of fibromyalgia. In February 2024, Tonix announced the engagement of Rho, Inc. as our contract research organization (CRO) to support NDA submission.
In February 2024, Tonix announced statistically significant results from its clinical pharmacokinetic (PK) bridging study of Tonmya in healthy adult male and female ethnic Japanese and Chinese volunteers. Results indicate that key PK parameters of cyclobenzaprine are comparable in ethnic Japanese and Chinese volunteers to Caucasian volunteers from a prior PK study. Tonmya was generally well tolerated in the ethnic Japanese and Chinese healthy volunteers. The company expects these data to fulfill the requirement for a bridging study, and enables Tonix to rely on Phase 3 studies RESILIENT and RELIEF results to support regulatory filings for clinical studies in Japan and China where cyclobenzaprine is a new chemical entity (NCE). Tonix holds issued patents for market exclusivity rights of Tonmya in Japan, China, Hong Kong and Taiwan.
In March 2024, Tonix announced the selection of two contract manufacturing organizations (CMOs), including Almac Pharma Services, as dual supply sources for the potential launch and commercialization of Tonmya in the U.S.
In March 2024, Tonix selected EVERSANA, a leading provider of commercialization services to the global life sciences industry, to support the launch strategy and commercial planning of Tonmya in the U.S.
Tonix presented additional efficacy data from RESILIENT at the 6th International Congress on Controversies in Fibromyalgia in Brussels, Belgium, March 7-8, 2024. The data showed that Tonmya treatment resulted in an improvement in cognitive dysfunction, or ‘brain fog’, measured by the change in the Fibromyalgia Impact Questionnaire-Revised (FIQ-R) memory item. The FIQ-R cognitive item showed nominal improvement in Tonmya-treated patients vs placebo-treated patients with a nominal p-value=0.001 and effect size of 0.31.
TNX-102 SL for the treatment of acute stress reaction (ASR) and acute stress disorder (ASD), and prophylaxis against development of posttraumatic stress disorder (PTSD)

In February 2024, the Company announced the FDA cleared the Investigational New Drug (IND) application for the Phase 2 investigator-initiated OASIS trial to evaluate TNX-102 SL in reducing the severity of ASR and the frequency of ASD and PTSD. The trial is sponsored by the University of North Carolina Institute for Trauma Recovery and supported by a $3 million grant from the U.S. Department of Defense, which was awarded in September 2023. The proposed Phase 2, Optimizing Acute Stress Reaction Interventions with TNX-102 SL (OASIS) study will examine the safety and efficacy of TNX-102 SL to reduce adverse posttraumatic neuropsychiatric sequelae among patients presenting to the emergency department (ED) after a motor vehicle collision. The study will enroll approximately 180 trauma survivors at ED study sites in the U.S. Participants will be randomized in the ED to receive a two-week course of either TNX-102 SL 5.6 mg or placebo.
Tonix anticipates the Phase 2 OASIS trial will initiate in the second quarter of 2024.
TNX-102 SL for the treatment of Fibromyalgia-Type Long COVID, also known as Post-Acute Sequelae of COVID-19 (PASC)

In January 2024, the Company announced the online publication of a research paper in the Journal Pain. The article titled, "Chronic Overlapping Pain Conditions Increase the Risk of Long COVID Features, Regardless of Acute COVID Status," by Bergmans, et al.1, found that patients with pre-existing chronic overlapping pain conditions (COPCs) had an increased risk of being diagnosed with symptoms of Long COVID1. Faculty at the University of Michigan directed the research. Commentary on the article titled, "A step towards better understanding chronic overlapping pain conditions" by Fitzcharles, et al,2 is in the same issue of the journal. COPCs include fibromyalgia, chronic fatigue syndrome, migraine headache, irritable bowel syndrome, endometriosis and low back pain. These results contribute to a growing body of evidence that common symptoms of Long COVID in many patients are at least partly driven by central nervous system mechanisms.
TNX-1300 (recombinant double mutant cocaine esterase): biologic for life-threatening cocaine intoxication

Tonix expects to initiate a Phase 2 clinical study of TNX-1300 for the treatment of cocaine intoxication in emergency rooms in the second quarter of 2024. In 2022, Tonix was awarded a Cooperative Agreement grant from the National Institutes of Health (NIH)’s National Institute of Drug Abuse (NIDA) to support development of TNX-1300.
TNX-1300 has been granted Breakthrough Therapy designation by the FDA.
TNX-1900 (intranasal potentiated oxytocin): small peptide in development through investigator-initiated studies for adolescent obesity, binge eating disorder, bone health in autism and social anxiety disorder (SAD).

TNX-1900 continues to be studied in four ongoing investigator-initiated Phase 2 studies as follows: Massachusetts General Hospital (MGH): (1) Phase 2 study for binge-eating disorder (BED); (2) Phase 2 study for adolescent obesity; (3) Phase 2 study for improving bone health in children with autism spectrum disorder (BOX); and at University of Washington, (4) Phase 2 study for social anxiety disorder (SAD). The BED study and the adolescent obesity study will investigate whether TNX-1900 has effects on eating behaviors in specialized populations.
Rare Disease Pipeline

TNX-2900 (intranasal potentiated oxytocin): small peptide for the treatment of Prader-Willi syndrome (PWS)

In March 2024, Tonix announced that it received Rare Pediatric Disease designation from the FDA for TNX-2900 for the treatment of PWS. Tonix has an IND to support clinical development of TNX-2900 to treat PWS in children and adolescents. The planned Phase 2 study is a dose-finding study involving approximately 36 PWS patients divided into four groups with approximately nine per group. One group will receive placebo and three groups will receive different dosage regimens of TNX-2900. TNX-2900 for the treatment of PWS was granted Orphan Drug designation by the FDA in 2022. PWS is a genetic disorder that affects several body systems, with cognitive and behavioral symptoms including pathological over-eating beginning in childhood and leading to severe metabolic sequelae.
Immunology Pipeline

TNX-1500 (anti-CD40L Fc-modified humanized monoclonal antibody): third generation anti-CD40L monoclonal antibody for prophylaxis of organ transplant rejection and treatment of autoimmune disorders.

The first proposed indication for TNX-1500 is prophylaxis of organ rejection in adult patients receiving a kidney transplant; but multiple additional indications are possible, including autoimmune diseases. Two peer reviewed publications described the work with TNX-1500 at the Massachusetts General Hospital (MGH) on allogeneic transplants in animals.3,4
Preclinical studies have shown that TNX-1500 maintains the activity of first-generation monoclonal antibodies (mAbs), yet with reduced risk of thrombotic complications.3-5 Modeling studies from animal pharmacokinetic data3 predict a half-life of greater than three weeks for TNX-1500 in humans, which supports a monthly i.v. dosing regimen. This analysis together with TNX-1500’s activity and tolerability in animals, suggests that the protein engineering of TNX-1500’s Fc region has achieved its design goals.
In February 2024, Tonix announced the completion of the clinical stage of its Phase 1 single ascending dose study of TNX-1500 in healthy volunteers. The primary objectives of the study are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of intravenous TNX-1500. This first-in-human study is intended to support dosing in a planned Phase 2 trial in kidney transplant recipients.
In March of 2024, the MGH announced the first transplant of a genetically modified pig kidney into a living patient in collaboration with eGenesis, which produced the pig donors and used an anti-CD40L mAb from another company.5 Some of the pre-clinical work that supported the living human transplant was performed in collaboration with Tonix and used TNX-1500.6 The patient was able to return home after the transplant, but died after approximately two months.7
Marketed Products – Recent Highlights

As of April 1, 2024, Tonix completed the transition to becoming a fully integrated pharmaceutical company. Tonix Pharmaceuticals has implemented personnel, systems and contracts required to support a commercial organization and has assumed responsibility for distribution, selling and marketing of Zembrace SymTouch and Tosymra, as well as supply chain, regulatory and quality control of the two products.
Facilities – Recent Highlights

In the fourth quarter of 2023, Tonix engaged CBRE, an international real estate brokerage firm, to potentially find a strategic partner for, or buyer of, its Advanced Development Center (ADC) to align with the Company’s current business objectives and priorities. At this time, the Company does not have a commitment in place to sell the building. ADC, located in the New Bedford business park in Dartmouth, Massachusetts, is an approximately 45,000 square foot BSL-2 facility intended for clinical scale manufacturing of live-virus vaccines and biologics.
*All of Tonix’s product candidates are investigational new drugs or biologics and none have been approved for any indication.

Tonmya is conditionally accepted by the U.S. Food and Drug Administration (FDA) as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.

1 Bergmans RS, et al. PAIN. 2023. DOI: 10.1097/j.pain.0000000000003110.

2 Fitzcharles M-A, et al. PAIN. 2023. DOI: 10.1097/j.pain.0000000000003129.

3 Lassiter G., et al. Am J Transplantation. 2023. View Source

4 Miura S., et al. Am J Transplantation. 2023. View Source

5 Massachusetts General Hospital press release. March 21, 2024. "World’s First Genetically Edited Pig Kidney Transplant into Living Recipient Performed at Massachusetts General Hospital." www.massgeneral.org/news/press-release/worlds-first-genetically-edited-pig-kidney-transplant-into-living-recipient (accessed March 29, 2024)

6 Anand, R.P., et al Nature. 622, 393–401 (2023). View Source

7 Stoico, N. Boston Globe. May 11, 2023. "Mass Man who received first kidney transplant from genetically engineered pig has died, family says".

Recent Highlights – Financial

As of March 31, 2024, Tonix had $7.0 million of cash and cash equivalents, compared to $24.9 million as of December 31, 2023. Net cash used in operations was approximately $17.6 million for first quarter 2024, compared to net cash used in operations of $32.9 million for the same period in 2023.

On April 1, 2024, the Company closed a financing with existing healthcare-focused institutional investors for upfront gross proceeds of approximately $4.4 million through a registered direct offering.

First Quarter 2024 Financial Results

Net product revenue for the first quarter 2024 was approximately $2.5 million. Net product revenue consisted of combined net sales of Zembrace SymTouch and Tosymra, which were acquired from Upsher-Smith Laboratories, LLC on June 30, 2023. Cost of Sales for the first quarter 2024 was approximately $1.7 million.

Research and development expenses for the first quarter 2024 were $12.9 million, compared to $26.5 million for the same period in 2023. This decrease is predominantly due to decreased clinical, non-clinical and manufacturing expenses.

General and administrative expenses for the first quarter 2024 were $9.3 million, compared to $7.4 million for the same period in 2023. The increase was primarily due to sales and marketing and the transition services expenses associated with the Company’s recently acquired marketed products offset by a decrease in financial reporting expenses.

Net loss was $(14.9) million, or $(0.18) per share, basic and diluted, for the first quarter 2024, compared to net loss of $(33.0) million, or $(3.21) per share, basic and diluted, for the same period in 2023. The basic and diluted weighted average common shares outstanding for the first quarter 2024 was 80,879,108 compared to 10,268,500 shares for the same period in 2023.

On May 13, 2024 Akoya Biosciences, Inc. (Nasdaq: AKYA) ("Akoya"), The Spatial Biology Company, reported its financial results for the first quarter ending March 31, 2024 (Press release, Akoya Biosciences, MAY 13, 2024, View Source [SID1234643127]).

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Business Highlights

● Revenue was $18.4 million in the first quarter of 2024, compared to $21.4 million in the prior year period; a decrease of 14%. Reagents and services revenue continued to increase.
● Akoya announced the establishment of a new Manufacturing Center of Excellence in Marlborough, Massachusetts to scale internal reagent manufacturing to meet the accelerating demand for reagents.
● Akoya’s partner Acrivon Therapeutics presented initial positive Phase 2b clinical data for the ACR-368 therapeutic in patients positive for the ACR-368 OncoSignature Assay in ovarian and endometrial cancer, deployed on the PhenoImager HT platform.
● Akoya and NeraCare, a leading developer of laboratory tests for the prognosis of melanoma patients, announced an exclusive partnership to enable personalized therapy selection for early-stage melanoma patients at high risk of relapse and death.
● Akoya and Shanghai KR Pharmtech announced that the KR-HT5 instrument, based on the PhenoImager HT, has received premarket approval from China’s National Medical Products Administration (NMPA).

"While we made meaningful progress in advancing both our operational and clinical objectives, our first quarter results fell short of expectations due to three main factors. First, systemic pressure on capital expenditures persisted. Second, certain pharmaceutical partner lab services revenue were deferred to the second half of 2024 due to revised clinical trial milestones. Third, the completion and launch of our fully operational Manufacturing Center of Excellence temporarily impacted reagent fulfillment times, delaying instrument purchases," said Brian McKelligon, CEO of Akoya Biosciences. "With the industry’s leading installed base, normalization of pharmaceutical partner revenue in the second half of 2024, and now resolved reagent availability issues, we believe that our foundational initiatives will lead us back to strong top-line growth and achievement of our profitability objectives."

First Quarter 2024 Financial Results

● Revenue was $18.4 million in the first quarter of 2024, compared to $21.4 million in the prior year period; a decrease of 14%. Reagents and services revenue continued to increase.
● For the first quarter of 2024, reported gross margin was 46% while non-GAAP adjusted gross margin was 57% when excluding the write off from discontinued legacy instruments. Both GAAP and non-GAAP adjusted gross margin were 57% for the first quarter of 2023.
● For the first quarter of 2024, operating expenses were $30.0 million and non-GAAP operating expenses were $25.6 million when excluding an impairment charge for facility consolidation and restructuring associated with a reduction in force in January. Both GAAP and non-GAAP operating expenses were $29.7 million for the first quarter of 2023.
● For the first quarter of 2024, loss from operations was $21.6 million and non-GAAP loss from operations was $15.2 million, excluding the items noted above. Both GAAP and non-GAAP loss from operations were $17.4 million for the first quarter of 2023.
● Ended the quarter with an instrument installed base of 1,213 (354 PhenoCyclers, 859 PhenoImagers), a year-over-year increase of 22% compared to an installed base of 992 in the prior year period (273 PhenoCyclers, 719 PhenoImagers).
● 1,307 total publications citing Akoya’s technology as of March 31, 2024, compared to 860 total publications in the prior year period: an increase of 52%.
● $61.6 million of cash, cash equivalents and marketable securities as of March 31, 2024.

2024 Financial Outlook

Akoya is updating its revenue outlook for the full year 2024 while maintaining its commitment to achieving operating cash flow breakeven by year end. The Company now expects full year 2024 revenue to be in the range of $104.0-$112.0 million.

Webcast and Conference Call Details

Akoya will host a conference call today, May 13, 2024, at 5:00 p.m. Eastern Time to discuss its first quarter 2024 financial results. Investors interested in listening to the conference call are required to register online. A live webcast of the conference call will be available on the "Investors" section of the Company’s website at View Source The webcast will be archived on the website following the completion of the call for three months.

On May 13, 2024 IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage drug-development company focused on developing DNA-mediated immuno-oncology therapies and next-generation vaccines, reported financial results for the three months ended March 31, 2024 (Press release, IMUNON, MAY 13, 2024, View Source [SID1234643143]). The Company also provided an update on its clinical development programs with IMNN-001, a DNA-based interleukin-12 (IL-12) immunotherapy in Phase 2 clinical development for first-line treatment of locally advanced ovarian cancer, and on its PlaCCine modality, a proprietary mono- or multi-cistronic DNA plasmid and a synthetic DNA delivery technology for the expression of pathogen antigens for the development of next-generation vaccines.

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"Potential key value-creating milestones are upon us. We expect that this summer will be rewarding and busy as we look to improve the treatment paradigm in late-stage ovarian cancer and to offer an "mRNA-better" vaccine platform technology with excellent commercial promise," said Mr. Michael H. Tardugno, IMUNON’s Executive Chairman.

"We remain on track to report topline results from the OVATION 2 Study with IMNN-001 in advanced ovarian cancer in mid-2024. If interim data are confirmed, the observed progression-free survival (PFS) benefit would represent a clinically meaningful outcome. In September, we reported interim PFS and overall survival (OS) data suggesting an approximate 30% delay in disease progression or death in the treatment arm compared with the control arm, with the hazard ratio nearing the study objective. Preliminary OS data followed a similar trend, showing an approximate nine month improvement in the treatment arm over the control arm. Subgroup analyses suggest patients treated with a PARP inhibitor (PARPi) as maintenance therapy had longer PFS and OS if they were also treated with IMNN-001, compared with patients treated with neoadjuvant chemotherapy (NACT) only," he added.

"Our Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for our seasonal COVID-19 booster vaccine (IMNN-101) was accepted by the Agency. The Company has begun a Phase 1 proof-of-concept study in two investigational centers. Our goal is to confirm the safety and immunogenicity of this DNA-based vaccine as an annual booster with long-lasting protection. The first patients are expected to be enrolled during the current quarter, and based on the results, we intend to advance discussions with potential partners for further development. Our optimism is based, in part, on final data from non-human primate studies that showed excellent immunological response and viral clearance. In a recent mouse study, we demonstrated that a single dose of IMNN-101, without a booster dose, produced longer duration of IgG responses and higher T cell activation than an mRNA vaccine. We have also demonstrated continued drug stability at standard refrigerated temperature of 4°C for more than 12 months, representing a significant advantage over commercial mRNA-based vaccines."

Stacy Lindborg, PhD appointed President and Chief Executive Officer

With great pleasure, the Company announced the appointment of Stacy R. Lindborg, Ph.D. as President and Chief Executive Officer, effective May 13, 2024. Dr. Lindborg has served on IMUNON’s board of directors since 2021 and was most recently Co-Chief Executive Officer of BrainStorm Cell Therapeutics, where she remains a director.

"We are delighted that Dr. Lindborg has agreed to deepen her ties with IMUNON as President and CEO," said Mr. Tardugno. "We have benefited significantly from her counsel as a director, where she has played an integral role in establishing our strategic priorities. Stacy joins the Company at a particularly important time. We now look forward to benefiting from her expertise in a more meaningful way, especially as our near-term data readouts will require important decisions with respect to advancing various programs and assets."

Dr. Lindborg, a globally recognized biostatistician, has nearly 30 years of pharmaceutical and biotech industry experience with a particular focus on R&D, regulatory affairs, executive management and strategy development. She has worked with biologics, small molecules and cell therapies to address a range of diseases and disorders. She has extensive experience in early-stage development, having taken molecules from first-in-human studies into the clinic, through regulatory approval and commercial launch.

RECENT DEVELOPMENTS

IMNN-001 Immunotherapy

Reported Interim PFS and OS Data in OVATION 2 Study in Advanced Ovarian Cancer. In September 2023, the Company announced interim PFS and OS data with IMNN-001 in its OVATION 2 Study. This study is evaluating the dosing, safety, efficacy and biological activity of intraperitoneal IMNN-001 in combination with chemotherapy prior to tumor reduction surgery (known as: NACT) in patients newly diagnosed with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer. NACT is designed to shrink the tumors as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo tumor debulking surgery, followed by three additional cycles of chemotherapy to treat any remaining tumor tissue.

The open-label study is directional and is designed to show an approximate 33% improvement in PFS when comparing the treatment arm with the control arm. Key secondary endpoints include OS, and the objective response rate. The final readout of this study is expected in mid-2024. A positive readout would inform the Phase 3 study design.

Interim data from the intent-to-treat population showed efficacy trends in PFS, demonstrating a delay in disease progression in the treatment arm of approximately three months compared with the control arm, with the hazard ratio nearing the study objective. Preliminary OS data followed a similar trend, showing an approximate nine-month improvement in the treatment arm over the control arm.
Non-prespecified subgroup analyses, commissioned as a result of the evolving standard of care for this population, suggest that patients treated with a PARPi as maintenance therapy had longer PFS and OS if they were also treated with IMNN-001, compared with patients treated with NACT only.
The median PFS in the PARPi + NACT group and the PARPi + NACT + IMNN-001 group was 15.7 months and 23.7 months, respectively.
The median OS in the PARPi + NACT group was 45.6 months and has not yet been reached in the PARPi + NACT + IMNN-001 group.
Began Treatment in a Phase 1/2 Clinical Trial Evaluating IMNN-001 in Combination with Bevacizumab (Avastin) in Advanced Ovarian Cancer. In October 2023, the first patient was enrolled in this trial at the University of Texas MD Anderson Cancer Center. This trial is expected to enroll 50 patients with Stage III/IV ovarian cancer. Patients undergoing frontline neoadjuvant therapy will be randomized 1:1 to receive standard chemotherapy plus bevacizumab, or standard chemotherapy plus bevacizumab and IMNN-001. The trial’s primary endpoint is detection of minimal residual disease (MRD) by second-look laparoscopy and the secondary endpoint is PFS. This trial will also include a wealth of translational endpoints aimed at understanding the clonal evolution and immunogenomic features of the MRD phase of ovarian cancer that is currently undetectable by imaging or tumor markers. In February 2024, the Company announced that Memorial Sloan Kettering Cancer Center joined MD Anderson Cancer Center in enrolling patients in this clinical trial.

PlaCCine: Developing the Prophylactic Vaccines of the Future

IND Application Cleared by the FDA to Begin Human Testing of IMNN-101. In April 2024, the Company announced receipt of FDA clearance to begin a Phase 1 proof-of-concept clinical trial with IMNN-101, a seasonal COVID-19 booster vaccine. Pending resolution of limited comments from the FDA, IMUNON expects to commence patient enrollment in the second quarter of 2024.

IMNN-101 utilizes the company’s PlaCCine platform, a proprietary mono- or multi-valent DNA plasmid that regulates the expression of key pathogen antigens and is delivered via a unique synthetic DNA delivery system. The primary objectives of the Phase 1 study are to evaluate safety, tolerability, neutralizing antibody response and the vaccine’s durability (duration of immunogenicity) in healthy adults. Secondary objectives include evaluating the ability of IMNN-101 to elicit binding antibodies and cellular responses and their associated durability. Based on reported preclinical data, durability of immune protection is expected to be superior to published mRNA vaccine data.

As currently planned, the Phase 1 study will enroll 24 subjects evaluating three escalating doses of IMNN-101 at two U.S. clinical trial sites. For this study, IMMN-101 has been designed to protect against the SARS-CoV-2 Omicron XBB1.5 variant, in accordance with the FDA’s Vaccines and Related Biological Products Advisory Committee’s June 2023 announcement of the framework for updated COVID-19 doses.

Preclinical Data for IMUNON’s PlaCCine DNA-Based Vaccine in SARS-CoV-2 Published in Peer-Reviewed Journal Vaccine. In February 2024, the Company announced that an article titled "Strong immunogenicity & protection in mice with PlaCCine: A COVID-19 DNA vaccine formulated with a functionalized polymer" was published in the peer-reviewed journal Vaccine, by Elsevier.

The article is available at View Source(24)00077-X.

The study described in the article used IMUNON’s proprietary formulation against the spike proteins from two SARS-CoV-2 variants, both alone and in combination. Data from the study show:

IMUNON’s proprietary formulation of functionalized polymer protected DNA from degradation and enhanced protein expression, while the combination with an adjuvant led to an increase in immunogenicity.
PlaCCine vaccines are stable for up to one year at 4°C and at least one month at 37°C.
Vaccination with PlaCCine resulted in the induction of spike-specific neutralizing antibodies and cytotoxic T cells.
In the in vivo challenge model, the vaccine-induced immune response was capable of suppressing viral replication.
Multiple inserts can be cloned into the PlaCCine backbone (a plug-and-play strategy), therefore allowing for an immune response with broader protection.
Corporate Developments

Received $1.3 Million in Non-Dilutive Funding from the Sale of New Jersey Net Operating Losses. In March 2024, the Company received $1.3 million in net cash proceeds from the sale of approximately $1.4 million of its unused New Jersey net operating losses (NOLs). The NOL sales cover the tax year 2022 and are administered through the New Jersey Economic Development Authority’s Technology Business Tax Certificate Transfer (NOL) program. This non-dilutive funding further strengthened the Company’s balance sheet.

FINANCIAL RESULTS FOR THE THREE MONTHS ENDED MARCH 31, 2024

IMUNON reported a net loss for the first quarter of 2024 of $4.9 million, or $0.52 per share, compared with a net loss of $5.6 million, or $0.68 per share, for the first quarter of 2023. Operating expenses were $5.0 million for the first quarter of 2024, a decrease of $0.7 million or 12% from $5.7 million for the first quarter of 2023.

Research and development (R&D) expenses were $3.3 million for the first quarter of 2024, an increase of $0.7 million from $2.6 million for the comparable period in 2023. Costs associated with the OVATION 2 Study were $0.3 million for both the first quarters of 2024 and 2023. Other clinical and regulatory costs were $1.1 million for the first quarter of 2024 compared with $0.3 million for the prior-year period. R&D costs associated with the development of IMNN-001 to support the OVATION 2 Study, as well as development of the PlaCCine DNA vaccine technology platform, were $1.6 million for the first quarter of 2024, compared with $1.4 million for the same period last year. CMC costs were $0.3 million for the first quarter of 2024, compared with $0.6 million for 2023 due to the development of in-house pilot manufacturing capabilities for DNA plasmids and nanoparticle delivery systems.

General and administrative expenses were $1.7 million for the first quarter of 2024, compared with $3.1 million for the comparable prior-year period. This decrease was primarily attributable to lower non-cash stock compensation expense ($0.3 million), lower legal costs ($0.5 million), lower employee-related costs ($0.2 million), lower consulting fees ($0.2 million) and lower insurance costs ($0.1 million).

Other non-operating income was $81,921 for the first quarter of 2024, compared with $93,085 for the comparable prior-year period. Investment income decreased $0.2 million due to lower balances of short-term investments in the current quarter. Interest expense decreased $0.2 million due to the repayment of the Company’s loan facility with Silicon Valley Bank in the second quarter of 2023.

Net cash used for operating activities was $5.9 million for the first quarter of 2024, compared with $4.0 million for the comparable prior-year period. This increase was primarily due to the final payment of CRO costs associated with the Phase III OPTIMA Study.

The Company ended the first quarter of 2024 with $9.8 million in cash, investments and accrued interest receivable. The Company believes it has sufficient capital resources to fund its operations to the end of 2024.

Conference Call and Webcast

The Company is hosting a conference call at 11:00 a.m. Eastern time today to provide a business update, discuss first quarter 2024 financial results and answer questions. To participate in the call, please dial 833-816-1132 (Toll-Free/North America) or 412-317-0711 (International/Toll) and ask for the IMUNON First Quarter 2024 Earnings Call. A live webcast of the call will be available here.

The call will be archived for replay until May 27, 2024. The replay can be accessed at 877-344-7529 (U.S. Toll-Free), 855-669-9658 (Canada Toll-Free) or 412-317-0088 (International Toll), using the replay access code 9343581. A webcast of the call will be available here for 90 days.

On May 13, 2024 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer, reported financial results for the first quarter ended March 31, 2024, and provided a corporate update (Press release, TScan Therapeutics, MAY 13, 2024, View Source [SID1234643160]).

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"During the first quarter we advanced our clinical pipeline, most recently marked by the dosing of the first patient in our Phase 1 solid tumor program. This is a significant milestone on the way to developing enhanced, customized, multiplex TCR-T therapy. We continue to prioritize screening patients for this study to enable rapid enrollment, and I am pleased to announce that we are on track to sharing initial data later this year," said Gavin MacBeath, Ph.D., Chief Executive Officer. "At the same time, we remain focused on enrolling and following patients in our heme malignancies study. We plan to complete Phase 1 enrollment and open expansion cohorts at the proposed recommended Phase 2 dose in the third quarter of 2024 and provide a data update near the end of 2024. With the recent closing of our public offering, TScan is well-funded to execute on anticipated milestones into the fourth quarter of 2026."

Recent Corporate Highlights

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The Company recently announced that the first patient has been dosed in its Phase 1 clinical trial evaluating TCR-T therapy for the treatment of various solid tumors. The Company is initially dosing patients with singleplex therapy to establish safety prior to initiating dosing with multiplex TCR-T (T-Plex). Patients are prospectively assigned to a treatment cohort based on expression of cancer-associated antigens and human leukocyte antigens (HLAs) in their tumor samples. The first patient, who has metastatic melanoma, was dosed with TSC-203-A0201, a TCR-T targeting PReferentially expressed Antigen in MElanoma (PRAME) on HLA-A*02:01. The Company expects robust patient enrollment as over 60 patients have completed all biomarker testing in the screening protocol. Of these patients, approximately 55% qualify for at least one TCR-T in the ImmunoBank and approximately 30% could qualify for multiplex therapy.

•
The Company recently provided an update on its Phase 1 heme malignancies program designed to treat residual disease and prevent relapse in patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), or myelodysplastic syndromes (MDS). The update included additional follow-up on all eight treatment-arm patients as well as data on two additional control-arm patients. With a median follow-up of >10 months, all eight patients treated with TSC-100 or TSC-101 remain relapse-free with no detectable disease. No dose-limiting toxicities were observed. In contrast, two control-arm patients relapsed approximately six months post-transplant and one of these patients died approximately three months later. A third control-arm patient required clinical intervention because of concerns of impending relapse, and a fourth control-arm patient died post-transplant.

•
In April 2024, the Company announced the closing of an upsized $167.8 million underwritten public offering with participation from new and existing high-quality healthcare investors. The financing extends the Company’s cash runaway into the fourth quarter of 2026. The Company intends to use the net proceeds to

advance its heme and solid tumor programs and expand and optimize its manufacturing capabilities, as well as for general corporate purposes.

•
The Company announced two upcoming poster presentations at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held May 31–June 4 both in Chicago and virtually. Details on the respective presentations can be found here.

•
The Company recently presented at two major medical meetings:

o
American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting
o
American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024

Presentation materials from the meetings can be found on the "Publications" tab of TScan’s website at tscan.com.

Upcoming Anticipated Milestones

Heme Malignancies Program: TScan’s two lead TCR-T therapy candidates, TSC-100 and TSC-101, are designed to treat residual disease and prevent relapse in patients with AML, ALL, or MDS undergoing allogeneic hematopoietic cell transplantation (HCT) (NCT05473910).

•
Opening of expansion cohorts at the proposed recommended Phase 2 dose level to further characterize safety and evaluate translational and efficacy endpoints is planned for the third quarter of 2024.
•
Completion of Phase 1 enrollment and reporting of one-year clinical and translational data on initial patients is anticipated in the second half of 2024.
•
Expects to initiate registration trial pending feedback from regulatory authorities and report two-year clinical and translational data in 2025.

Solid Tumor Program: TScan continues to expand the ImmunoBank, a collection of therapeutic TCR-Ts that target different cancer-associated antigens presented on diverse HLA types. TScan’s strategy is to treat patients with multiple TCR-Ts to overcome tumor heterogeneity and prevent resistance that may arise from either target or HLA loss (screening protocol: NCT05812027; treatment protocol: NCT05973487).

•
Phase 1 solid tumor clinical study has been initiated; first patient dosed in early May, with three additional patients enrolled and manufacturing underway.
•
Initial data expected in the second half of 2024.
•
Additional investigational new drug (IND) filings planned to continue to expand the ImmunoBank.
•
Long-term duration of response data for multiplex therapy anticipated in 2025.

First Quarter 2024 Financial Results

Revenue: Revenue for the first quarter of 2024 was $0.6 million, compared to $6.8 million for the first quarter of 2023. The decrease was primarily due to the timing of research activities pursuant to the Company’s collaboration agreements. Revenue for the first quarter of 2024 was related solely to the collaboration agreement with Amgen which commenced in May 2023. Revenue for the first quarter of 2023 was related solely to the collaboration agreement with Novartis, which concluded in March 2023.

R&D Expenses: Research and development expenses for the first quarter of 2024 were $24.9 million, compared to $21.8 million for the first quarter of 2023. The increase of $3.1 million was primarily driven by an increase in personnel expenses due to additional headcount in support of expanded research and development activities, as well as an increase in clinical studies expense associated with the ongoing enrollment of our Phase 1 heme study and start-up activities for the Phase 1 solid tumor clinical trial. Research and development expenses included non-cash stock compensation expense of $1.1 million and $0.4 million for the first quarter of 2024 and 2023, respectively.

G&A Expenses: General and administrative expenses for the first quarter of 2024 were $7.1 million, compared to $7.8 million for the first quarter of 2023. The decrease of $0.7 million was primarily driven by lower facility-related and personnel costs. General and administrative expenses included non-cash stock compensation expense of $0.9 million and $0.7 million for the first quarter of 2024 and 2023, respectively.

Net Loss: Net loss was $30.1 million for the first quarter of 2024, compared to $22.6 million for the first quarter of 2023, and included net interest income of $1.2 million and $0.2 million, respectively.

Cash Position: Cash, cash equivalents, and marketable securities as of March 31, 2024, were $162.8 million, excluding $5.0 million of restricted cash. The Company believes that its existing cash resources along with the $161.4 million net proceeds received from its April 2024 underwritten public offering will be sufficient to fund its current operating plan into the fourth quarter of 2026.

Share Count: As of March 31, 2024, the Company had issued and outstanding shares of 47,904,737, which consists of 43,628,149 shares of voting common stock and 4,276,588 shares of non-voting common stock, and outstanding pre-funded warrants to purchase 47,010,526 shares of voting common stock at an exercise price of $0.0001 per share.

Subsequent to the end of the first quarter of 2024, in connection with its April 2024 underwritten public offering, the Company issued an additional 4,958,068 shares of voting common stock, and pre-funded warrants to purchase up to 18,577,419 shares of voting common stock with an exercise price of $0.0001 per share.