On March 28, 2024 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotech company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to improving survival outcomes for patients with cancer, reported the full-year 2023 annual results and provided a business overview (Press release, MaaT Pharma, MAR 28, 2024, View Source [SID1234641570]).

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"As we reflect on the achievements of 2023, I am proud to announce that we have successfully delivered on milestones across our entire pipeline, while being cash conscious. This past year has been pivotal, laying the foundations for the next phase of development for our lead asset MaaT013 with completion of the Phase 3 and readiness for commercialization. We look forward to reaching new milestones in 2024 and anticipate a major inflection point with the publication of the primary endpoint of our Phase 3. By relying on our team and our network of physicians, and continuing to work closely with regulators, we anticipate continuing to create value for our shareholders and make significant progress in bringing new therapeutic options for patients with cancer," states Hervé Affagard, CEO and co-founder of MaaT Pharma.

Pipeline highlights

MET-N

MaaT013

In hemato-oncology:

In April 2023, the U.S. Food and Drug Administration (FDA) lifted the clinical hold and cleared the Investigational New Drug (IND) application for MaaT013 in patients with aGvHD[4]. The Company has engaged active discussions with prominent US clinicians in the field of stem cell transplantation to explore the most efficient path forward to introduce MaaT013 to patients in the United States.
In July 2023, the Company announced that clinical data from its Phase 2 study assessing MaaT013 as a treatment for aGvHD was published in eClinicalMedicine, one of the Lancet Discovery Science suite of journals.
In October 2023, the Company announced that the DSMB[5] unanimously recommended that the open-label, single arm pivotal Phase 3 ARES clinical trial (NCT04769895), evaluating MaaT013 in aGvHD, continue without modification. The Overall Response Rate (ORR) was superior to pre-defined protocol assumptions. Therefore, the DSMB concluded that the benefit/risk ratio with "high efficacy and low toxicity" was favorable in this patient population. Primary endpoint, gastrointestinal overall response rate (GI-ORR), is now expected mid Q4 2024.
In December 2023, the Company presented positive results from the Early Access Program (EAP) in Europe involving 111 patients with aGvHD treated with MaaT013, at the 2023 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The GI-ORR was 54% at day 28, positively and significantly impacting overall survival (OS) with 47% OS at 12 months for all patients with aGvHD and 52% for a patient subgroup from the EAP with a similar profile to those in the ARES trial with GI-ORR of 61% and 58% of complete responses observed at day 28.
As a post period event, in March 2024, the Company announced that it will present for the first time extended results that include OS data after 12 months in more aGvHD patients from its EAP that were treated with MaaT013. The data will be shared during an oral presentation at the 50th Annual Meeting of the European Society for Blood and Marrow Transplantation held in Glasgow, UK, from April 14-17, 2024.
As a post period event, the Company announces the launch of a retrospective multicenter trial called CHRONOS in Europe. Its objective is to provide the Company efficacy data for 3rd-line therapies for patients not receiving MaaT013 or any Microbiome intervention. This retrospective study does not impact cash projections as funding is already secured.
In immuno-oncology:

As a post period event, in March 2024, the Company informed on the completion of patient recruitment for the Phase 2a clinical trial (NCT04988841) sponsored by AP-HP and in collaboration with INRAe[6] and Institut Gustave Roussy[7], evaluating MaaT013, the Company’s lead product candidate, in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy) and nivolumab (Opdivo) in ICI naïve patients with metastatic melanoma. Having reached this key recruitment milestone, the first publication will be submitted at the end of 2024 or in the first quarter of 2025.
MaaT033

In hemato-oncology:

In April 2023, data from the Phase 1b study (CIMON) with MaaT033, previously communicated during the 64th annual ASH (Free ASH Whitepaper) meeting, were also presented at the EBMT 2023 conference.
In September 2023, the Company announced that the European Medicines Agency (EMA) had granted orphan drug designation (ODD) to MaaT033, which is aimed at improving overall survival in patients undergoing allo-HSCT[8]. The EMA recognized the significant benefit that MaaT033 could therefore bring to this patient population. The status offers key benefits including market exclusivity, clinical protocol assistance, waivers or reductions in regulatory fees.
In November 2023, the Company announced that the first patient was treated in the Phase 2b trial (PHOEBUS) investigating the efficacy of MaaT033 in improving OS at 12 months in patients with blood cancer that are receiving allo-HSCT. The international, multi-center, randomized, double-blind, placebo-control study (NCT05762211), will be conducted in up to 56 clinical investigation sites and is expected to enroll 387 patients. It is, to date, the largest randomized controlled trial assessing a microbiome therapy in oncology.
In November 2023, the Company announced that its ongoing Phase 2b trial, PHOEBUS has been selected for funding of 7.4 million EUR as part of the France 2030 Health Innovation plan, in response to the ‘Innovation in Biotherapy and Bioproduction’ call for projects from the ‘Biotherapies and Bioproduction of Innovative Therapies’ acceleration strategy, operated by Bpifrance (project code: METALLO). Post period the first tranche of 1.8 million EUR was received.

In neurodegenerative diseases:

In September 2023, the Company announced that the first patient was dosed in the IASO Phase 1b pilot study (NCT05889572) in Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease in the U.S. and Charcot’s disease in French-speaking countries. As a post period event, in February 2024, the Company announced that the DSMB reviewed safety data in the IASO trial in the first 8 patients with ALS treated with MaaT033. The DSMB recommended that the trial continue without modifications.
MET-C

MaaT034

In immuno-oncology:

In November 2023, the Company had two presentations at the 38th Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting highlighting in vitro results for its new Artificial Intelligence (AI)-generated lead product, MaaT034, designed to improve responses to immunotherapy for patients with solid tumors. MaaT034 is the first member of the
MET-C platform. Data presented at SITC (Free SITC Whitepaper) 2023 shows that MaaT034 replicates, at large industrial scale, the richness, and diversity of healthy native-based microbiome ecosystems. The first clinical batches are expected to be produced in 2024 and the first-in-human study is planned for 2025.
As a post period event, in March 2024, the Company announced that it will present new in vitro data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024, taking place on April 5-10 in San Diego, California.
Corporate update

In February 2023, the Company completed a successful capital increase of approximately €12.7 million with the support of current shareholders.
In July 2023, MaaT Pharma joined the Microbiome Therapeutics Innovation Group (MTIG). MTIG is a coalition of companies focused on developing FDA-approved microbiome therapeutics and products to improve medical care, outcomes, and cost-effectiveness.
In September 2023, the Company and Skyepharma announced completion of the cGMP manufacturing facility and the transfer of MaaT Pharma’s Production and Development teams to the new site.
During 2023 and Q1 2024, MaaT Pharma reinforced its board of directors, executive team, and key functions:
Karim Dabbagh as Chairman and Nadia Kamal as Director, both independent.
Jonathan Chriqui, as Chief Business Officer and member of the executive management team.
Guilhaume Debroas, as Head of Investor Relations.
Financial highlights

The key financial results for the 2023 full year are as follows:

Income Statement

In thousands of euros 31 December 2023 31 December 2022
Revenue 2 228 1 430
Cost of Goods Sold (573) (339)
Gross Margin 1 655 1 091
Other Income 4 667 4 122
Sales and distribution costs (449) (347)
General and administrative costs (4 965) (4 111)
Research and development costs (20 851) (14 311)
Operating income (expense) (19 943) (13 557)
Financial Income 639 45
Financial Expense (413) (201)
Net financial income (expense) 226 (156)
Income (loss) before income tax (19 717) (13 713)

Income tax expense – –
Net Income (loss) for the period (19 717) (13 713)
Prepared in accordance with international standards, IFRS.
Revenues totaled €2.2 million for the year ended December 31, 2023, the highest revenues generated thus far by the Company, which includes compensation invoiced from the Early Access Program in France and for which data was presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2023. The gross margin generated by the compassionate access program amounts to €1.7 million.

Operating expense amounted to €19.9 million compared with €13.6 million for 2022, an increase of €6.3 million. This increase reflects the growth of research and development costs which have risen from €14.3 million in 2022 to €21.2 million in 2023, representing an overall increase of €6.9 million and consistent with the advancement of clinical and operational activities as detailed in the pipeline highlights’ section above.

Other income of €4.7 million includes the R&D tax credit of €3.6 million, an increase of €0.4 million compared with prior year, which amounted to €3.2 million and in line with the growth of research and development activities and eligible expenses.

General and administrative expenses amounted to €5.0 million compared with €4.1 million in 2022 reflecting the increase in regulatory advisory costs and expenses to support the early access program.

The net loss amounts to €19.7 million for the year ended December 31, 2023, compared with €13.7 million for the year ended December 31, 2022.

Average annual employees evolved from 43 in 2022 to 53 in 2023 following the strengthening of the clinical and production and supply chain and to a lesser extent administrative teams.

Cash Position

As of December 31, 2023, total cash and cash equivalents were €24.3 million, as compared to €31.7 million as of September 30, 2023, and €35.2 million as of December 31, 2022.

The net decrease in cash position of €11.0 million between December 31, 2022, and December 31, 2023, is due to the financing of operations for a total of €18.7 million, offset by cash inflows from financing of €8.1 million. Cash inflows from financing reflects the share capital increase in February 2023 of approximately €12.7 million, offset by debt repayments over 2023 €4.2 million. Total financial debt totaled €14.1 million as of December 31, 2023, of which €0.4 million relates to state-backed loans ("PGE") and €6.1 million of lease liabilities, which includes future contractual payments due to Skyepharma for the use of the manufacturing facility completed in 2023.

Based on the development plans and corresponding cash needs, the Company believes it has sufficient cash to finance operations to the end of Q3.2024 extending the cash runway by three months compared to prior communications as a result of a voluntary slowdown in headcount growth, optimization of its manufacturing plan and prioritizing expenses to support on the roll out of the Phase 2b trial PHOEBUS in France and Germany (approved countries) and finalizing recruitment into the Phase 3 ARES trial in Europe. The Company has active discussions ongoing to finance operations beyond the end of Q3.2024 and remains confident in extending its cash runway.

Note: This press release contains financial data approved by the Board of Directors on March 27, 2024, based on the financial statements for the year ended December 31, 2023. The audit is in progress at the date of this communication.

Upcoming financial communication*

May 14, 2024: Publication of revenues for Q1 2024
May 28, 2024: Annual General Shareholders Meeting
September 19, 2024: Publication of half year financial results H1
November 5, 2024: Publication of revenues for Q3 2024
*Indicative calendar that may be subject to change.

Upcoming investor conferences participation

April 15-17, 2024 – Kempen Life Sciences Conference, Amsterdam
June 11-12, 2024 – Portzamparc Mid & Small Caps 2024 Conference
June 25-27, 2024 – Stifel European Healthcare Summit Lyon
Upcoming investor conferences participation

April 5-10, 2024 – American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024, San Diego
April 14-17, 2024 – 50th Annual meeting of the European Bone Marrow Transplant (EBMT), Glasgow
The Company’s universal registration document, which includes the annual financial report, will be available on MaaT Pharma’s website on 02/04/2024: www.maatpharma.com

On March 28, 2024 Larkspur Biosciences, a company pioneering a new wave in cancer therapy by targeting cancer-intrinsic drivers of immune evasion, reported that it will present positive preclinical efficacy data for LRK-A, Larkspur’s novel, lead investigational therapy targeting the lipid kinase PIP4K2C, in primary human tumor samples and an in vivo model of colorectal cancer (CRC) (Press release, Larkspur Biosciences, MAR 28, 2024, View Source [SID1234641568]). The data will be featured in a poster presentation at the 115th Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (AACR 2024) in San Diego, CA.

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"Larkspur Biosciences pursues novel targets inside the cancer, like PIP4K2C, that prevent the immune system from seeing and attacking it. Our data at AACR (Free AACR Whitepaper) 2024 demonstrate the potential of targeted PIP4K2C degradation to uncloak the tumor and support the advancement of our PIP4K2C degrader program as a monotherapy to treat CRC and other solid tumors," said Catherine Sabatos-Peyton, PhD, CEO of Larkspur Biosciences. "Using LarkXCRC, our proprietary bioinformatics platform, Larkspur developed a patient biomarker strategy to select the patients most likely to respond to LRK-A. This strategy enables the design of clinical trials that are smaller, faster, and have a higher probability of success."

CRC is the second leading cause of cancer-related deaths in the U.S.; in people under the age of 50, CRC is the top cause of cancer mortality in men and second cause in women 1. Larkspur aims to shift the treatment paradigm – which hasn’t meaningfully changed in decades – by effectively activating the immune system against tumors by targeting PIP4K2C.

"PIP4K2C is part of the ‘dark kinome’ – the approximately 25% of kinases with unknown or poorly understood functions," said Nathanael Gray, PhD, professor of chemical and systems biology at Stanford University and co-founder of Larkspur. "My co-founders and I jointly discovered that PIP4K2C acts in both cancer cells and immune cells to get tumor antigens presented to the right immune cells. Lew Cantley discovered that uniquely among the family of PI5P4K isoforms, mice that were germline knockouts of PIP4K2C broke tolerance, a hallmark of key targets that drive cancer immune evasion. Larkspur’s AACR (Free AACR Whitepaper) 2024 presentation sheds further light on PIP4K2C by offering key evidence for its role as a cancer-intrinsic driver of immune evasion in both tumor and immune cells, and demonstrating the therapeutic promise of degrading the target in CRC and other solid tumors."

Presentation details:

Published Abstract Number: 5574

Title: "Degradation of PIP4K2C by novel bivalent functional degrader LRK-A induces tumor regression in CRC"

Session Category / Session Title: Tumor Biology / Tumor-Immune System Cross-Talk

Session Date and Time: Tuesday, April 9, 1:30-5:30 pm PDT

Location / Poster Board Number: Poster Section 12 / Number 30

Presenter: Eva d’Hennezel, PhD, Director of Immunology, Larkspur Biosciences

On March 28, 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 year ended December 31, 2023 (Press release, IMUNON, MAR 28, 2024, View Source [SID1234641567]). 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 the treatment of first-line, locally advanced-stage 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 in preclinical studies for the development of next-generation vaccines.

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Highlights of 2023 and recent weeks include the following:

Reported interim progression-free survival (PFS) and overall survival (OS) data with IMNN-001 in the Phase 2 OVATION 2 Study in patients with advanced ovarian cancer. Interim data from the intent-to-treat (ITT) population showed an approximate 30% delay in disease progression or death in the treatment arm compared with the control arm, and preliminary OS data showed an approximate nine-month improvement in the treatment arm over the control arm.
Enrolled four patients in a Phase 1/2 clinical trial evaluating IMNN-001 in combination with bevacizumab in patients with advanced ovarian cancer at the University of Texas MD Anderson Cancer Center, and recently added Memorial Sloan Kettering Cancer Center as a clinical site for this study.
Announced results from a non-human primate study confirming PlaCCine as a viable modality for the development of the next generation of prophylactic vaccines.
Reported pre-clinical data demonstrating PlaCCine vaccines elicit robust and more durable T cell responses than commercial mRNA vaccines in animal models, signaling that PlaCCine vaccines may provide greater protection against reinfection, hospitalization or death.
Submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) in the first quarter of 2024 for a Phase 1/2 proof-of-concept clinical trial with a seasonal COVID-19 booster vaccine.
Launched a new current Good Manufacturing Practices production facility to efficiently support R&D with significantly lower costs for infectious disease vaccines, and DNA-based immuno-oncology therapies.
"IMUNON has a dedicated management team to advance our two platform technologies and execute our strategic plan," said Mr. Michael Tardugno, IMUNON’S Executive Chairman. "We remain on track to report topline results mid-year from the OVATION 2 Study with IMNN-001 in advanced ovarian cancer. If the interim data are confirmed in the final readout, the observed PFS benefit would represent a clinically meaningful outcome. We also remain on track to begin a Phase 1 proof-of-concept clinical study in the second quarter of 2024 with a seasonal COVID-19 booster vaccine, following FDA clearance of our IND application. Our objective is to confirm the safety and immunogenicity of our clinical vaccine in humans. Based on these results, we will advance discussions with potential partners to continue development of the platform."

"IMUNON made significant progress during 2023, in particular with advancing our clinical programs in immuno-oncology with IMNN-001, our gene-mediated IL-12 immunotherapy. In September we reported interim PFS and OS data in our OVATION 2 Study suggesting a delay in disease progression or death in the treatment arm of approximately 30% 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 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 PlaCCine modality continues to advance with very encouraging data. We demonstrated the validity of this proprietary technology in prophylactic vaccines, with impressive preclinical proof-of-concept data not only in COVID-19, but also in a multiple of other pathogenic viruses. We also completed the evaluation of our vaccines in non-human primates. The final data from these studies show excellent immunological response and viral clearance. In a recent mouse study, we demonstrated that a single dose of our PlaCCine vaccine 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 4C for more than 12 months, representing a significant commercial advantage over commercial mRNA-based vaccines.

Given the high costs and long lead times of third party CMOs, we have strategically invested in, and completed development of in-house pilot manufacturing capabilities for DNA plasmids and synthetic delivery systems. Our scientists can now select any protein from the human or pathogen proteomes to be engineered. Our labs also can conduct testing and run experiments in a variety of animal disease models independently supporting bench-to-bedside development of our novel therapies and vaccines. These capabilities are expected to allow us to realize our goal of attracting strategic partners while minimizing dependence on vendors so that we control both the costs and the development timelines.

"We are excited about the key value-creating milestones we face in 2024. The potential for advances in treating late-stage ovarian cancer may be within reach, while a better vaccine platform technology holds tremendous commercial promise. We look forward to continuing to create value for our stockholders and for patients," Mr. Tardugno concluded.

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 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 interval debulking surgery, followed by three additional cycles of chemotherapy to treat any residual tumor.

The open-label study is directional and designed with an 80% confidence interval to show an approximate 33% improvement in PFS, when comparing the treatment arm (NACT + IMNN-001) with the control arm (NACT only). The secondary endpoints include OS, objective response rate, pathological response, surgical response and serologic response. The final readout of this study is expected in mid-2024. A positive readout would inform next development steps.

Interim data from the ITT 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 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.
Continued benefits were seen in other secondary endpoints including an approximately 25% higher R0 tumor resection score and a doubling of the CRS 3 chemotherapy response score to approximately 30% in the treatment arm, versus 14% in the control arm. A complete tumor resection (R0) is a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed. Chemotherapy response score is considered a good prognostic indicator in ovarian cancer. Safety analyses continue to show good tolerability of IMNN-001 in this setting.

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 has joined MD Anderson Cancer Center in enrolling patients in this clinical trial. A total of four patients have been enrolled in the study to date.

PlaCCine: Developing the Prophylactic Vaccines of the Future

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.
IMUNON’s Vice President of R&D Presented at the Vaccines Summit-2023. In November 2023, Jean Boyer, Ph.D. delivered a presentation titled "Robust Immunogenicity and Protection with PlaCCine: A Novel DNA Vaccine Delivered with a Functionalized Polymeric Delivery System" during the "New Vaccine Development" session at the Vaccines Summit-2023 in Boston. The presentation included updated data related to IMUNON’s PlaCCine SARS-CoV-2 DNA vaccine, including studies showing PlaCCine expresses spike proteins in mice and primates demonstrating induction of spike-specific neutralizing antibody responses and CD8 and CD4 spike-specific cellular responses. The induced immune responses in vaccinated mice were maintained for up to 14 months after vaccination. The presentation also showed that in both primates and mice, the induced immune responses reduced lung viral loads by more than 90%. In mouse studies, robust immune responses were observed following a single intramuscular injection of either PlaCCine SARS-CoV-2 DNA vaccine or a novel PlaCCine Lassa Virus DNA vaccine.

IMUNON’s Chief Science Officer Presented at the 3rd International Vaccines Congress. In October 2023, Khursheed Anwer, Ph.D. delivered a presentation titled "A DNA-based Vaccine Technology Independent of Virus or Device" at the 3rd International Vaccines Congress in Boston. The presentation described the multiple advantages of the PlaCCine modality over current commercial vaccine platforms. The presentation also described the versatility of the PlaCCine modality, demonstrating activity against Marburg and influenza viruses in collaboration with the Wistar Institute, and activity against Lassa virus being evaluated at the NIH/NIAID.

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 Year Ended December 31, 2023

IMUNON reported a net loss for 2023 of $19.5 million, or $2.16 per share, compared with a net loss for 2022 of $35.9 million, or $5.03 per share. Operating expenses were $21.0 million for 2023, a decrease of $4.4 million or 17% from $25.4 million for 2022. The Company recognized tax benefits from the sale of its New Jersey NOLs of $1.3 million and $1.6 million in 2023 and 2022, respectively.

Research and development (R&D) expenses were $11.3 million for 2023, a decrease of $0.4 million from $11.7 million for 2022. Costs associated with the OVATION 2 Study were $1.2 million and $1.5 million for 2023 and 2022, respectively. Costs associated with the Phase 3 OPTIMA Study were de minimis for 2023 compared with $1.0 million for 2022. Other clinical and regulatory costs were $1.8 million for 2023 compared with $1.9 million for 2022. 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 $6.0 million for 2023 compared with $6.1 million for 2022. CMC costs increased to $2.3 million for 2023 compared with $1.2 million for 2022 due to the development of in-house pilot manufacturing capabilities for DNA plasmids and nanoparticle delivery systems in 2023.

General and administrative expenses were $9.7 million for 2023 compared with $13.7 million for 2022. This decrease was primarily attributable to lower non-cash stock compensation expense ($1.3 million), lower employee-related costs ($0.8 million), lower legal costs ($1.0 million), lower insurance costs ($0.6 million) and lower public company expenses ($0.2 million), offset by higher consulting fees ($0.2 million).

Other non-operating income was $0.2 million for 2023 compared with other non-operating expenses of $12.5 million for 2022. This increase was attributable to the following:

Investment income from the Company’s short-term investments was $1.2 million for 2023 and $0.5 million for 2022.
In June 2021, the Company entered into a $10.0 million loan facility with Silicon Valley Bank (SVB). The Company immediately used $6.0 million from this facility to retire all outstanding indebtedness with Horizon Technology Finance Corporation. In connection with the loan facility, the Company incurred $0.2 million in interest expense in 2023 compared with $0.5 million in 2022. In the second quarter of 2023, the Company terminated the SVB Loan Facility, paid early termination and end-of-term charges and recognized $0.3 million as a loss on debt extinguishment.
In 2022, the Company recognized (i) an impairment charge of $13.4 million due to the write off of In-Process Research & Development (IPR&D) assets and (ii) a non-cash gain of $5.4 million due to the write-off of the earnout milestone liability because of the requirements not being achieved.
In 2022, the Company incurred additional interest expense attributable to the one-time payment of $4.5 million in interest and offering expenses resulting from the sale and subsequent redemption of $28.5 million of Series A & B convertible redeemable preferred stock.
Net cash used for operating activities was $19.0 million for 2023 compared with $23.1 million for 2022. This decrease was primarily due to the one-time payment of $4.5 million in interest expense resulting from the sale and subsequent redemption of $28.5 million of Series A & B convertible redeemable preferred stock in the first quarter of 2022. Cash used in financing activities was $3.6 million for 2023 from the pay-off of the SVB loan ($6.4 million), offset by sales under the Company’s At-the-Market Equity Facility ($2.8 million). This compares with cash provided by financing activities of $6.7 million for 2022 resulting from an at-the-market equity offering ($6.2 million) and sales under the Company’s At-the-Market Equity Facility ($0.5 million).

The Company ended 2023 with $15.7 million in cash, investments and accrued interest receivable. Along with future planned sales of the Company’s remaining $1.3 million of New Jersey NOLs, the Company believes it has sufficient capital resources to fund its operations into the fourth quarter of 2024.

Conference Call and Webcast

The Company is hosting a conference call to provide a business update, discuss 2023 financial results and answer questions at 10:00 a.m. Eastern time today. To participate in the call, please dial 866-777-2509 (Toll-Free/North America) or 412-317-5413 (International/Toll) and ask for the IMUNON 2023 Earnings Call. A live webcast of the call will be available here.

The call will be archived for replay until April 11, 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 8601697. A webcast of the call will be available here for 90 days.

On March 28, 2024 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM:​HCM; HKEX:​13) reported that the supplemental New Drug Application ("sNDA") for savolitinib, in adult patients with locally advanced or metastatic non-small cell lung cancer ("NSCLC") with mesenchymal epithelial transition factor ("MET") exon 14 skipping alteration, has been accepted for review by the China National Medical Products Administration (NMPA) (Press release, HUTCHMED, MAR 28, 2024, View Source [SID1234641566]). If approved, the new label indication for savolitinib will be expanded to include treatment-naive patients in China.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Savolitinib was previously granted conditional approval in China for the treatment of patients with NSCLC with MET exon 14 skipping alterations who have progressed following prior systemic therapy or are unable to receive chemotherapy. Savolitinib was launched and is marketed under the brand name ORPATHYS by our partner, AstraZeneca for this patient population, representing the first selective MET inhibitor approved in China. More than a third of the world’s lung cancer patients are in China and, among those with NSCLC globally, approximately 2-3% have tumors with MET exon 14 skipping alterations.

Preliminary efficacy and safety data from the first-line cohort of the confirmatory Phase IIIb clinical trial (NCT04923945) were presented during the IASLC World Conference on Lung Cancer (WCLC) in September 2023. Final data from the confirmatory Phase IIIb trial were presented at the European Lung Cancer Congress on March 20, 2024.

The data from this study provide confirmatory evidence for savolitinib as a targeted treatment option for treatment-naïve or previously treated patients with MET exon 14 skipping alteration NSCLC. In treatment-naïve patients, objective response rate ("ORR") was 62.1% (95% CI: 51.0% to 72.3%), disease control rate ("DCR") was 92.0% (95% CI: 84.1% to 96.7%) and median duration of response ("DoR") was 12.5 months (95% CI: 8.3 months to 15.2 months), as assessed by an independent review committee. Median progression free survival ("PFS") was 13.7 months (95% CI: 8.5 months to 16.6 months) and median overall survival ("OS") was not reached with median follow-up of 20.8 months. In previously treated patients, ORR was 39.2% (95% CI: 28.4% to 50.9%), DCR was 92.4% (95% CI: 84.2% to 97.2%) and median DoR was 11.1 months (95% CI: 6.6 months to not reached), as assessed by an independent review committee. Median PFS was 11.0 months (95% CI: 8.3 months to 16.6 months) and median OS was not mature with median follow-up of 12.5 months. Responses occurred early (time to response 1.4-1.6 months) in both treatment-naïve and previously treated patients. The safety profile was tolerable and no new safety signals were observed. The most common drug-related treatment-emergent adverse events of Grade 3 or above (5% or more of patients) were abnormal hepatic function (16.9%), increased alanine aminotransferase (14.5%), increased aspartate aminotransferase (12.0%), peripheral oedema (6.0%) and increased gamma-glutamyltransferase (6.0%).

About NSCLC and MET aberrations

Lung cancer is the leading cause of cancer death among men and women, accounting for about one-fifth of all cancer deaths.1 Lung cancer is broadly split into NSCLC and small cell lung cancer, with 80-85% classified as NSCLC.2 The majority of NSCLC patients (approximately 75%) are diagnosed with advanced disease, and approximately 10-15% of NSCLC patients in the U.S. and Europe and 30-40% of patients in Asia have EGFRm NSCLC.

MET is a tyrosine kinase receptor that has an essential role in normal cell development.7 MET overexpression and/or amplification can lead to tumor growth and the metastatic progression of cancer cells, and is one of the mechanisms of acquired resistance to EGFR TKIs for metastatic EGFR-mutated NSCLC.7,8 Approximately 2-3% of NSCLC patients have tumors with MET exon 14 skipping alterations, a targetable mutation in the MET gene.9 Among patients who experience disease progression post-osimertinib treatment, approximately 15-50% present with MET aberration.10,11,12,13,14 The prevalence of MET depends on the sample type, detection method and assay cut-off used.15

About Savolitinib (ORPATHYS in China)

Savolitinib is an oral, potent and highly selective MET tyrosine kinase inhibitor that has demonstrated clinical activity in advanced solid tumors. It blocks atypical activation of the MET receptor tyrosine kinase pathway that occurs because of mutations (such as exon 14 skipping alterations or other point mutations), gene amplification or protein overexpression.

Savolitinib is marketed in China under the brand name ORPATHYS for the treatment of patients with non-small cell lung cancer with MET exon 14 skipping alterations who have progressed following prior systemic therapy or are unable to receive chemotherapy. It is currently under clinical development for multiple tumor types, including lung, kidney and gastric cancers, as a single treatment and in combination with other medicines. Starting on March 1, 2023, ORPATHYS was included in the National Reimbursement Drug List (NRDL) for the treatment of locally advanced or metastatic NSCLC adult patients with MET exon 14-skipping alterations who have progressed after or unable to tolerate platinum-based chemotherapy.

In 2011, AstraZeneca and HUTCHMED entered a global licensing and collaboration agreement to jointly develop and commercialize savolitinib. Joint development of savolitinib in China is led by HUTCHMED, while AstraZeneca leads development outside of China. HUTCHMED is responsible for the marketing authorization, manufacturing and supply of savolitinib in China. AstraZeneca is responsible for the commercialization of savolitinib in China and worldwide. Sales of savolitinib are recognized by AstraZeneca.

On March 28, 2024 Heron Therapeutics, Inc. (Nasdaq: HRTX), a commercial-stage biotechnology company, reported that Craig Collard, Chief Executive Officer of Heron, will participate in a fireside chat on Wednesday, April 10, 2024, at 11:00 AM ET at the 23rd Annual Needham Virtual Healthcare Conference (Press release, Heron Therapeutics, MAR 28, 2024, View Source [SID1234641565]). The conference is being held in a virtual format, April 8-11, 2024.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the fireside chat will be available on the Company’s website at www.herontx.com in the Investor Resources section.