On March 30, 2023 Pulmatrix (NASDAQ: PULM), a clinical-stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and central nervous system disease using its patented iSPERSE technology, reported fourth quarter and year-end financial results for 2022 and provided a corporate update on its development programs (Press release, Pulmatrix, MAR 30, 2023, View Source [SID1234629609]).

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Ted Raad, Chief Executive Officer of Pulmatrix commented, "2022 was a year of significant progress for all of our development programs, setting the stage for significant milestone accomplishments in 2023. In 2022, we completed the Phase 1b study of PUR1800 for acute exacerbations in chronic obstructive pulmonary disease (AECOPD), for which the data was presented this year. We also initiated and completed a Phase 1 study of PUR3100, our orally inhaled formulation of dihydroergotamine (DHE) for acute migraine, allowing us to begin 2023 by announcing data that we believe illustrates a potentially positive pharmacokinetic and pharmacodynamic profile for PUR3100 – including a rapid systemic exposure within the targeted therapeutic range, and fewer side effects compared to intravenous (IV) dosing. Finally, we prepared for a Phase 2b study of PUR1900 in allergic bronchopulmonary aspergillosis (ABPA) and announced the first patient dosed in Q1 2023. We are grateful for the potential opportunity to positively impact patients with these programs and are proud of the Company’s accomplishments throughout last year and into this year."

2022 and Recent Program and Corporate Highlights

PUR1900

In February 2023, Pulmatrix began dosing patients for its Phase 2b study of PUR1900. This Phase 2b trial is designed as a randomized, double-blind, multi-center, placebo-controlled study to evaluate the efficacy and duration of treatment with itraconazole, administered as a dry powder for inhalation (PUR1900). The goal of the study is to provide data on potential registrational endpoints in ABPA in patients with asthma. The multi-center study is being conducted in the United States, United Kingdom, Australia and France. Endpoints include safety, tolerability, and potential efficacy outcomes in adult patients with asthma and ABPA. Pulmatrix expects to report topline data from this study in mid-2024.
PUR3100

On January 4, 2023, Pulmatrix achieved positive topline results from the Phase 1 study for PUR3100, a novel pulmonary inhaled formulation of DHE for the treatment of acute migraine. The study found that PUR3100 was well-tolerated and there was a lower incidence of nausea in PUR3100 dose groups compared to IV DHE. No vomiting was observed in any of the PUR3100 dose groups. Oral inhalation of PUR3100 achieved peak exposures in the targeted therapeutic range at all doses and the Tmax occurred at five minutes after dosing.
The Phase 1 study was designed as a double-dummy, double-blinded trial to assess the safety, tolerability, and pharmacokinetics of three dose levels of single doses of inhaled PUR3100 with IV placebo, as compared to IV DHE (DHE mesylate injection) with inhaled placebo. Twenty-six healthy subjects were enrolled and each of the four groups contained at least six subjects.
Pulmatrix believes its PUR3100 formulation of DHE is highly differentiated from other DHE products already approved or in development, can be immediately self-administered and has a pharmacokinetic profile that may potentially advance the treatment of patients with acute migraine. Given the positive Phase 1 study results, the Company plans to pursue further clinical studies for PUR3100, including a potential Phase 2 clinical study.
PUR1800

In March 2022, Pulmatrix reported topline data from its Phase 1b clinical study of PUR1800 for AECOPD. The Company has presented the analyzed data results of the completed Phase 1b clinical study at the American Academy of Allergy, Asthma and Immunology medical conference in February 2023. The Company is continuing to analyze these data to inform the design for a potential Phase 2 efficacy and safety study in subjects with AECOPD.
2022 Corporate Highlights

On February 28, 2022, the Company completed a reverse stock-split at a ratio of 1-for-20 which reduced the number of outstanding shares of the Company’s common stock from approximately 65.9 million shares to approximately 3.3 million shares. The number of authorized shares of the Company’s common stock remains at 200,000,000 shares.
On March 1, 2022, the Company announced the hiring of Dr. Margaret Wasilewski as the Company’s Chief Medical Officer. Dr. Wasilewski leverages over 25 years of experience in pharmaceutical drug development.
On March 17, 2022, the Company announced that it regained compliance for its listing on Nasdaq, allowing continued access to capital markets and liquidity for its investors.
Fourth Quarter and Year-End Financial Results

Revenue was $6.1 million for the year ended December 31, 2022, as compared to $5.2 million for the year ended December 31, 2021; an increase of $0.9 million. The increase was related to $4.6 million more revenues under the collaboration with Cipla Technologies LLC during 2022, which was partially offset by a $3.7 million decrease in license-related revenues under a former collaboration with Johnson & Johnson Enterprise Innovation, Inc.

Research and development expense was $18.2 million for the year ended December 31, 2022, as compared to $15.4 million for the year ended December 31, 2021; an increase of $2.8 million. The increase was primarily due to an increased spend of $2.9 million in costs related to the Company’s PUR1900 program and $2.6 million of employment and operating costs, partially offset by decreased spend of $2.7 million in costs primarily related to Pulmatrix’s PUR1800 program.

General and administrative expense was $6.8 million for the year ended December 31, 2022, as compared to $6.4 million for the year ended December 31, 2021; an increase of $0.4 million. The increase was primarily due to increased professional services costs of $0.4 million.

Pulmatrix’s total cash and cash equivalents balance as of December 31, 2022 was $35.6 million. The Company anticipates that its cash position is sufficient to fund operations into Q2 2024.

PULMATRIX, INC.

Consolidated Balance Sheets

(in thousands, except share and per share data)

December 31,

2022

December 31,

2021

Assets

Current assets:

Cash and cash equivalents

$

35,628

$

53,840

Restricted cash

153

–

Accounts receivable

1,298

67

Prepaid expenses and other current assets

1,068

871

Total current assets

38,147

54,778

Property and equipment, net

235

321

Operating lease right-of-use asset

710

2,093

Long-term restricted cash

1,472

1,625

Other long-term assets

389

–

Total assets

$

40,953

$

58,817

Liabilities and stockholders’ equity

Current liabilities:

Accounts payable

$

1,188

$

839

Accrued expenses and other current liabilities

1,638

1,233

Operating lease liability

857

1,431

Deferred revenue

1,339

939

Total current liabilities

5,022

4,442

Deferred revenue, net of current portion

4,822

6,069

Operating lease liability, net of current portion

–

857

Total liabilities

9,844

11,368

Stockholders’ equity:

Preferred Stock, $0.0001 par value — 500,000 shares authorized;
6,746 shares designated Series A convertible preferred stock; no and
1,830 shares issued and outstanding at December 31, 2022 and 2021,
respectively

–

1,081

Common stock, $0.0001 par value — 200,000,000 shares authorized;
3,639,185 and 3,222,037 shares issued and outstanding at December
31, 2022 and 2021, respectively

–

–

Additional paid-in capital

304,585

301,008

Accumulated deficit

(273,476)

(254,640)

Total stockholders’ equity

31,109

47,449

Total liabilities and stockholders’ equity

$

40,953

$

58,817

PULMATRIX, INC.

Consolidated Statements of Operations

(in thousands, except share and per share data)

Year ended December 31,

2022

2021

Revenues

$

6,071

$

5,169

Operating expenses:

Research and development

18,240

15,382

General and administrative

6,778

6,377

Impairment of goodwill

–

3,577

Total operating expenses

25,018

25,336

Loss from operations

(18,947)

(20,167)

Other income/(expense):

Interest income

309

7

Other expense, net

(198)

(11)

Total other income/(expense), net

111

(4)

Net loss

(18,836)

(20,171)

Less: Deemed dividend – beneficial conversion feature of preferred stock

–

(3,197)

Net loss attributable to common stockholders

$

(18,836)

$

(23,368)

Net loss per share attributable to common stockholders – basic and diluted

$

(5.46)

$

(8.63)

Weighted average common shares outstanding – basic and diluted

3,447,701

2,708,558

On March 30, 2023 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported its first-quarter 2023 sales and earnings conference call with institutional investors and analysts at 9:00 a.m. ET on Thursday, April 27 (Press release, Merck & Co, MAR 30, 2023, View Source [SID1234629608]). During the call, company executives will provide an overview of Merck’s performance for the quarter.

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Investors, journalists and the general public may access a live audio webcast of the call via this weblink. A replay of the webcast, along with the sales and earnings news release, supplemental financial disclosures, and slides highlighting the results, will be available at www.merck.com.

All participants may join the call by dialing (888) 769-8514 (U.S. and Canada Toll-Free) or (517) 308-9208 and using the access code 8206435.

On March 30, 2023 MaaT Pharma (EURONEXT: MAAT – the "Company"), a French clinical-stage biotech and a pioneer in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to improving survival outcomes for patients with cancer, reported the full-year 2022 annual results and provided a business overview (Press release, MaaT Pharma, MAR 30, 2023, View Source [SID1234629607]).

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Hervé Affagard, CEO and co-founder of MaaT Pharma states, "in 2022, we have successfully executed on key clinical and manufacturing objectives despite a difficult economic environment. With our lead asset, MaaT013, currently in a Phase 3 clinical trial in hemato-oncology, it is our ambition to make it quickly accessible to all patients fighting acute Graft-versus-Host Disease in need of a safe and effective therapeutic option. Our clinical development pipeline continues to progress with positive Phase 1b data for MaaT033 announced in 2022, the commencement of a Phase 2b trial in the first half of 2023 as well was the ongoing Early Access Program in Europe for MaaT013. With the construction of our manufacturing facility to be completed by mid-2023 and the roadmap we shared at the beginning of the year, we are excited with the progress we have made to advance our microbiome therapies that we believe could become a new pillar in treating cancer."

Key Financial Results

The key audited financial results for the 2022 full year are as follows:

Income statement

In thousands of euros 31 December 2022 31 December 2021
Revenue 1 430 972
Cost of Goods Sold (339) (166)
Gross Margin 1 091 806
Other Income 4 122 2 390
Sales and distribution costs (347) (217)
General and administrative costs (4 111) (2 727)
Research and development costs (14 311) (9 145)
Operating income (expense) (13 557) (8 893)
Financial Income 45 0
Financial Expense (201) (126)
Net financial income (expense) (156) (126)
Income (loss) before income tax (13 713) (9 019)

Income tax expense – –
Net Income (loss) for the period (13 713) (9 019)
Prepared in accordance with international standards, IFRS.

Revenues totaled €1.4 million for the year ended December 31, 2022, 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 2022. The gross margin generated by the compassionate access program amounts to €1.1 million.

Operating expense amounted to €13.6 million compared with €8.9 million for 2021, an increase of €4.7 million. This increase reflects the growth of research and development costs which have risen from €9.1 million in 2021 to €14.3 million in 2022, representing an overall increase of €5.2 million and consistent with the advancement of clinical and operational activities as detailed in the 2022 key achievements’ section below.

Other income of €4.1 million includes the R&D tax credit of €3.2 million, an increase of €1.2 million compared with prior year, which amounted to €2.0 million and in line with the growth of research and development activities.

General and administrative expenses amounted to €4.1 million compared with €2.7 million in 2021 reflecting the recurring costs and structuring of the Company to meet the needs of its listing on Euronext Paris regulated market and to support the different clinical and development programs.

The net loss amounts to €13.7 million for the year ended 31 December 2022 compared with €9.0 million for the year ended 31 December 2021.

Average annual employees evolved from 33 in 2021 to 43 in 2022 following the strengthening of the clinical and scientific teams, along with the R&D, technical and regulatory departments.

Cash Position

As of December 31, 2022, total cash and cash equivalents were €35.2 million, as compared to €38.4 million as of June 30, 2022, and €43.3 million as of December 31, 2021.

In thousands of euros 31 December 2022 31 December 2021
Net cash used in operating activities (12 605 ) (7 929 )
Net cash used in investing activities (815 ) (238 )
Net cash used in financing activities 5 364 31 558
Net change in cash and cash equivalents (8 057 ) 23 391
The net decrease in cash position of €8.1 million between December 31, 2021, and December 31, 2022, is due to the financing of operations for a total of €12.6 million, offset by cash inflows from financing of €5.4 million. Cash inflows from financing reflects new financial debt, offset by debt repayments over 2022 €1.9 million. Total financial debt (including lease liabilities) totaled €11.4 million as of December 31, 2022, of which €0.9 million relates to state-backed loans ("PGE").

Based on the development plans, corresponding cash needs and following the capital increase in February 2023, the Company believes it has sufficient cash to finance operations into the second quarter of 2024.

2022 Key achievements

Pipeline highlights

MaaT013

In March 2022, the Company announced the initiation of its Phase 3 open label, single arm, pivotal trial, called ARES, evaluating MaaT013 in treating patients with acute Graft-versus-Host Disease (aGvHD). The trial is ongoing in six European countries including France, Austria, Spain, Belgium, Germany, and Italy. In the US, interactions with the FDA remain active regarding MaaT013, for which US development is currently on clinical hold following an FDA communication received in August 2022. In February 2023, the Company announced new discussions with the FDA which have been detailed in the section below "First half of 2023".
In April 2022, the Company announced the initiation of a randomized, placebo-controlled Phase 2a, proof of concept, clinical trial in France sponsored by AP-HP, evaluating MaaT013’s impact on the efficacy of immune checkpoints inhibitors (ICI) treatments in patients with metastatic melanoma.
In 2022, the Company continued the Early Access Program in Europe allowing patients to benefit from early access to the MaaT013 drug candidate, mainly for the treatment of aGvHD. As of today, the Company has safely treated over 160 patients with MaaT013 in Europe.
In December 2022, the Company presented compelling consolidated data from 81 patients in the Early Access Program in France at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2022.
MaaT033

In the first half of 2022, the Company announced positive topline results for its Phase 1b open-label, dose-ranging clinical trial, called CIMON, investigating the maximum tolerated dose of MaaT033 in patients with acute myeloid leukemia or high-risk myelodysplastic syndrome who have undergone intensive chemotherapy and confirming clinical potential of MaaT Pharma’s oral drug candidate. These promising results support the launch of the Phase 2b clinical trial, called PHOEBUS, to improve overall survival and to prevent complications in patients with blood cancers receiving allo-HSCT.
In December 2022, the Company presented the Phase 1b clinical data in a poster format at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
MaaT03X

In 2022, the Company has been consolidating in vivo and in vitro data with MaaT03X and continuing its product development characterization.
Financial highlight

In February 2022, the Company announced the construction of its cGMP manufacturing facility in France, dedicated to ecosystem microbiome-based therapeutics, in partnership with Skyepharma. A total down payment of €1.1 million has been made in 2022 for a total of €1.4 million cumulatively. The facility is expected to be operational by mid-2023.
First half of 2023

In January 2023, the Company announced the expansion of its scientific research to neurodegenerative diseases with a first trial in Amyotrophic Lateral Sclerosis (ALS). The Company is now preparing to launch a Phase 1b pilot study to evaluate MaaT033 in ALS, following ANSM regulatory authorizations received in March 2023 – the inclusion of the first patient is expected in H1 2023.
In February 2023, the Company completed a successful capital increase of approximately €12.7 million with the support of current shareholders.
The ongoing international multicenter open-label, single arm pivotal Phase 3 trial (ARES) evaluating MaaT013 in aGvHD is ongoing in Europe – the Data and Safety Monitoring Board (DSMB) review is expected to take place at the end of the first half of 2023, if half of the patients have been recruited.
In February 2023, the Company announced the receipt of a letter from the FDA indicating that the Agency agreed to a defined list of conditions that could enable clinical evaluation of MaaT013 in the U.S. These measures have since been included by the Company and submitted to the FDA. The communication from the FDA therefore provides a path forward regarding MaaT Pharma’s pooling technology for this trial.
The Phase 2a proof-of-concept clinical trial evaluating MaaT033 in association with ICI in metastatic melanoma, sponsored by AP-HP, in France is ongoing – biological biomarker data are expected in H1 2023 after half of the patients have been enrolled and achieved their evaluation 9 weeks after randomization.
The preparations are ongoing to initiate the randomized placebo-controlled Phase 2b trial (PHOEBUS) evaluating MaaT033 in improving overall survival and to prevent complications in allo-HSCT patients and the Company has received French and German regulatory authorizations in March 2023– study is expected to start in Q2 2023.
The Company’s universal registration document, which includes the annual financial report, will be available on MaaT Pharma’s website: www.maatpharma.com

Upcoming financial communication*

May 9, 2023 – Revenues and Cash Position Quarter 1
June 19, 2023 – Annual General Meeting
July 27, 2023 – Revenues and Cash Position Quarter 2
September 26, 2023 – Half-year Results 2023
November 9, 2023 – Revenues and Cash Position Quarter 3
*Indicative calendar that may be subject to change.
Upcoming investor conferences participation

April 4, 2023 – Investor Access Conference, Paris
April 26, 2023 – Kempen Life Sciences Conference, Amsterdam
Upcoming scientific conference participation

April 23-26, 2023 – 49th Annual meeting of the European Bone Marrow Transplant, Paris
June 28-30, 2023 – 8th Microbiome Movement – Drug Development Summit, Boston

On March 30, 2023 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company discovering and developing innovative gamma-delta T cell therapies, reported financial results and operational highlights for the fourth quarter and full-year ended December 31, 2022 (Press release, In8bio, MAR 30, 2023, View Source [SID1234629604]). In addition, the Company provided an overview of recent corporate developments.

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"We are extremely pleased with the outstanding progress IN8bio has made in the past year and the encouraging clinical results we have observed across our gamma-delta T cell platform in both solid and liquid tumors," remarked William Ho, CEO and co-founder of IN8bio. "Our lead clinical programs, INB-100 and INB-200, continue to deliver promising outcomes with longer than expected relapse free and overall survival rates. Last year, we showcased our manufacturing, regulatory and clinical capabilities by filing and receiving clearance for our first corporate-sponsored IND from the FDA for INB-400. Additionally, our team remains committed to leveraging our profound knowledge of gamma-delta T cell biology to drive innovation in next-generation chimeric antigen receptor (CAR) technology. Most recently, we disclosed new preclinical data from our INB-300 platform, demonstrating a CAR construct that can differentiate between tumor and healthy tissue when both express the targeted antigen. We eagerly anticipate releasing additional clinical updates and unveiling our continued progress throughout this year."

Business Highlights and Recent Developments

•
Presented initial preclinical data demonstrating how INB-300, the Company’s novel non-signaling gamma-delta CAR-T platform (nsCAR) has the unique ability to distinguish between tumor and healthy tissue. Using the well-established CD-19 target, the proof-of-concept study revealed that INB-300 (ns19CAR) eliminated 80% of the B cell leukemia cell line and only 5% of healthy B cells when both expressed CD19. These results demonstrating the wider therapeutic index support the potential expansion of CAR technology for previously "undruggable" cancer targets in indications including acute myeloid leukemia (AML) and solid tumor cancers.
•
Provided a clinical update from the ongoing INB-200 Phase 1 trial in patients with newly diagnosed glioblastoma (GBM), highlighting encouraging progression free survival results. As of December 31, 2022, eight patients with newly diagnosed glioblastoma (GBM) had been dosed with INB-200: three in Cohort 1 (single dose), four in Cohort 2 (three doses) and one in Cohort 3 (six doses). Two Cohort 2 patients died of unrelated causes with no evidence of progression before death, while two other patients in the same cohort remained alive, progression-free and clinically asymptomatic at 1.5 and 1.2 years, respectively.
•
Presented new data at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrating the potential of INB-100 to achieve long-term, durable responses in patients with high-risk or relapsed hematologic malignancies. The findings were based on the ongoing Phase 1 trial of INB-100, a single-dose allogeneic gamma-delta T cell therapy, administered to patients with hematologic malignancies undergoing haploidentical stem cell transplantation (HSCT). All trial participants remained progression-free as of December 9, 2022, with the longest response durations extending beyond 2.7 years.
•
Obtained U.S. Food and Drug Administration (FDA) clearance for the Company-sponsored INB-400 Investigational New Drug (IND) application. This will enable the initiation of a Phase 2 clinical trial for INB-400, a genetically modified autologous gamma-delta T cell therapy targeting newly diagnosed GBM. The study will evaluate safety, efficacy, and tolerability at leading medical centers across the United States.

Upcoming Pipeline Milestones and Events

•
INB-100: Report updated Phase 1 trial data from leukemia patients undergoing HSCT at the European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting in April 2023; define recommended Phase 2 dose for INB-100.

•
INB-200: Complete enrollment of Cohort 3 in the Phase 1 trial; report updated data and results with longer-term follow-up at medical meetings throughout 2023.

•
INB-300: Present additional preclinical data demonstrating proof-of-concept for the nsCAR platform at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2023.

•
INB-400: Initiate patient enrollment in the company-sponsored Phase 2 trial of INB-400, a genetically modified autologous gamma-delta T cell therapy, targeting newly diagnosed GBM in the second half of 2023.

•
INB-410: Submit a new IND to the FDA for a Phase 1b trial of INB-410, a genetically modified allogeneic gamma-delta T cell therapy in newly diagnosed and relapsed GBM in late 2023.

•
New solid tumor indications: Announce and present relevant data at a scientific conference in the first half of 2023.

Fourth Quarter and Full Year 2022 Financial Highlights

•
Cash position: As of December 31, 2022, the Company had cash of $18.2 million, compared to $37.0 million as of December 31, 2021. The decrease in cash was primarily due to cash used by the Company in R&D and continued operations to advance its programs along with ongoing construction of a state-of-the-art ~10,000 sq. ft. R&D facility in Birmingham, AL.
•
Research & Development (R&D) expenses: R&D expenses were $4.0 million for the three months ended December 31, 2022, compared to $2.7 million for the comparable prior year period. R&D expenses were $14.1 million for the year ended December 31, 2022, compared to $7.3 million in the prior year. The increase in R&D expenses was primarily due to contract research organization expenses related to the INB-400 program and IND, increased third-party clinical trial and IND-related activities, increased facility-related costs from opening our new laboratory space in Birmingham, Alabama and increased personnel-related costs, including salaries, benefits, and stock-based compensation due to increased headcount.
•
General and administrative (G&A) expenses: G&A expenses were $3.9 million for the three months ended December 31, 2022, compared to $3.2 million for the comparable prior year period. G&A expenses were $14.5 million for the year ended December 31, 2022, compared to $7.3 million in the prior year. The increase was primarily due to increased personnel-related costs, including salaries, benefits, and stock-based compensation reflecting an increased headcount, facility-related costs, insurance costs, and expenses associated with operating as a public company.
•
Net loss: The Company reported a net loss of $7.8 million, or $0.32 per basic and diluted common share, for the three months ended December 31, 2022, compared to a net loss of $5.9 million, or $0.44 per basic and diluted common share, for the comparable prior year period. For the full year, net loss was $28.5 million, or $1.36 per basic and diluted common share, compared to a net loss of $14.7 million, or $1.47 per basic and diluted common share, for the comparable prior year.

On March 30, 2023 IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage drug-development company focused on developing DNA-mediated immunotherapy and next-generation vaccines, reported financial results for the year ended December 31, 2022, and provided an update on its clinical development programs with IMNN-001 (formerly GEN-1), a DNA-based interleukin-12 (IL-12) immunotherapy in Phase 2 clinical development for the treatment of advanced-stage ovarian cancer, and with PLACCINE, a proprietary, multivalent DNA plasmid technology utilizing synthetic, non-viral vaccine delivery vectors being evaluated in preclinical studies for superiority over current generation of nucleic acid vaccines (Press release, IMUNON, MAR 30, 2023, View Source [SID1234629590]).

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Highlights of 2022 and recent weeks include:

Completed enrollment in the Phase 1/2 OVATION 2 Study with IMNN-001 in advanced ovarian cancer.
Phase 1/2 Clinical Study of IMNN-001 in combination with Avastin in advanced ovarian cancer was opened to enrollment in the first quarter of 2023.
Reported compelling results from a non-human primate (NHP) study confirming PLACCINE as a viable modality for the development of the next generation of prophylactic vaccines. PLACCINE is IMUNON’s non-viral, non-device plasmid DNA-based vaccine modality targeting multiple antigens from a single vector.
Signed new research collaborations with The Wistar Institute to develop new vaccine formulations utilizing the Company’s PLACCINE modality for the development of vaccines for infectious diseases, and with Acuitas Therapeutics to evaluate IMUNON’s plasmid DNA with Acuitas’ lipid nanoparticle delivery system.
Made strategic investment in Transomic Technologies to strengthen IMUNON’s development capabilities of the PLACCINE DNA vaccine modality.
Reported cash and cash equivalents of $38.9 million as of December 31, 2022, which is expected to fund operations into 2025.
"I am pleased to report that IMUNON made significant progress during 2022 in advancing our clinical programs in immuno-oncology with IMNN-001, our IL-12 gene-mediated immunotherapy. Earlier in the year we reported data from 46 patients in the experimental arm of our OVATION 2 Phase 1/2 study who had undergone interval debulking surgery, showing an improvement in R0 surgical resection rates and CRS 3 chemotherapy response scores over the 41 patients in the control arm. In September we reached full enrollment of 110 patients in this study and expect to report an additional set of interim, more mature data in the second half of 2023 and topline results by mid-2024," said Dr. Corinne Le Goff, IMUNON’s President and Chief Executive Officer.

"Our PLACCINE modality continues to advance with very promising data. We demonstrated the validity of this proprietary technology in prophylactic vaccines, with impressive proof-of-concept data in a COVID-19 model. We also completed the evaluation of our vaccines in non-human primates. I am pleased to report that the final data are consistent with the earlier data and show excellent immunological response and viral clearance. We demonstrated in a recent mouse study 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 are now nine months into a 12-month PLACCINE stability study and have demonstrated continued drug stability at the standard refrigerated temperature of 4⁰C, representing a significant commercial advantage over mRNA-based vaccines," she added. "With the continuing volatility of the public markets, our decision to raise capital earlier this year to strengthen our balance sheet and extend our operating runway into 2025 was well timed. We expect to report several value-creating developments during this period."

Dr. Le Goff continued, "This year we anticipate filing an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for our seasonal COVID-19 booster vaccine. Our objective is to confirm in a Phase 1 clinical study the safety of our PLACCINE modality. In the first half of 2023, we intend to apply for a pre-IND consultation with the FDA to receive guidance on our proposed program prior to submitting the IND.

"We also will select our next pathogen target for our PLACCINE modality. It is likely that we will choose a pathogen among the list of priority pathogens established by the Coalition for Epidemic Preparedness Innovations. Our vaccine program objective is to establish the safety and efficacy of our platform in a Phase 1 human study, and then seek to out-license this powerful technology to pharmaceutical companies for the utilization of our platform and/or to establish non-dilutive partnerships to develop vaccines for pathogens of interest."

"We have developed in-house pilot manufacturing capabilities for DNA plasmids and nanoparticle delivery systems. Our scientists can select any protein from the human or pathogen proteomes to be engineered. In combination, during recent months we made an investment in Transomic Technologies, which offers a comprehensive array of CRISPR, RNAi and gene expression tools and services. Our existing labs also have the ability to conduct testing and to run experiments in a variety of animal disease models. These capabilities are expected to allow us to realize our goal of attracting corporate partners while minimizing dependence on vendors so that we control both the costs and the development timelines. Our progress to date is evidence that IMUNON is a fully integrated clinical development company with expertise in running global mid-stage clinical programs," Dr. Le Goff concluded.

RECENT DEVELOPMENTS

PLACCINE: Developing the Prophylactic Vaccines of the Future

Presentation at Vaccine Technology Summit 2023 Describes Compelling Preclinical Data Supporting Continued development of PLACCINE as a Differentiated, Next-Generation Vaccine. In March 2023, Khursheed Anwer, Ph.D., the Company’s Executive Vice President and Chief Science Officer, presented data on the company’s PLACCINE platform at the Vaccine Technology Summit 2023 in Boston. Dr. Anwer’s presentation is titled "A Novel DNA Vaccine Platform with Potential to Create Next Generation Vaccines," and can be found on the company’s website here.

Dr. Anwer reviewed the Company’s work in advancing its PLACCINE modality and the promising preclinical data generated to date. Among topics presented was the ability of this multi-valent technology to achieve broad spectrum immunity from a single DNA plasmid with a synthetic delivery system. This ability is independent of virus, device or liquid nanoparticle formulations. The data presented showed:

Robust immunogenicity and protection in SARS-CoV-2 models
Durable cellular or humoral responses detectable for more than 12 months
Comparable protection activity to a commercial mRNA vaccine in a booster-dose comparison
Superior immune quality versus the mRNA vaccine in a single-dose comparison
In addition, the PLACCINE modality had important distinguishing advantages for a commercial vaccine, including a shelf-life at 4⁰C for greater than nine months, and the ability for simple, rapid and scalable manufacturing.

Presentation at World Vaccine & Immunotherapy Congress Highlights PLACCINE Preclinical Proof of Concept and Key Competitive Advantages. In December 2022, Khursheed Anwer, Ph.D., the Company’s Chief Science Officer, presented at the World Vaccine & Immunotherapy Congress. Dr. Anwer highlighted the PLACCINE modality and proof-of-concept rodent and non-human primate data in SARS-CoV-2. Slides from Dr. Anwer’s presentation are available here.

Final Results from NHP Study and Additional Preclinical Studies Support PLACCINE as a Viable Prophylactic Vaccine Development Modality. In October 2022 the Company reported partial results from an ongoing NHP study designed to examine the immunogenicity of its proprietary DNA-based vaccine in support of PLACCINE as a viable alternative to commercial mRNA vaccines. The study examined a single plasmid DNA vector containing the SARS-CoV-2 Alpha variant spike antigen formulated with a synthetic DNA delivery system and administered by intramuscular injection.

In the study, cynomolgus monkeys were vaccinated with the PLACCINE vaccine or a commercial mRNA vaccine on Day 1, 28 and 84. Analysis of blood samples for IgG and neutralizing antibodies showed evidence of immunogenicity both in PLACCINE and mRNA vaccinated subjects. Analysis of bronchoalveolar lavage for viral load by quantitative PCR showed viral clearance by more than 90% of the non-vaccinated controls. Viral clearance from nasal swab followed a similar pattern in a majority of vaccinated animals and a similar clearance profile was observed when viral load was analyzed by the tissue culture infectious dose method. In a head-to-head comparison, the protection efficiency as measured by viral clearance following challenge with the SARS-CoV-2 virus was similar between PLACCINE and a commercial mRNA vaccine.

On March 1, 2023, IMUNON’s CEO issued a Letter to Shareholders announcing the final results from the Company’s evaluation of its vaccines in NHP. Dr. Le Goff reported that the final data are consistent with the earlier data and show excellent immunological response and viral clearance. The Company reported results from a recent mouse study that demonstrated a single dose of PLACCINE vaccine without a booster dose produced longer duration of IgG responses and higher T-cell activation than an mRNA vaccine as well as nine-month data from a 12-month PLACCINE stability study that demonstrates continued drug stability at 4⁰C (standard refrigerated temperature), representing a significant commercial advantage over mRNA-based vaccines.

IMNN-001 Immunotherapy

Phase 1/2 Clinical Study of IMNN-001 in Combination with Bevacizumab in Advanced Ovarian Cancer was opened to enrollment. In February 2023 the Company announced a collaboration to evaluate IMNN-001 in a Phase 1/2 clinical trial in combination with bevacizumab in ovarian cancer in the frontline, neoadjuvant setting. Working with four of the foremost comprehensive cancer centers in the world, the goal of this project is to transform the care of women with ovarian cancer by developing unprecedented capabilities for understanding and targeting persistent minimal residual disease (MRD), as explained here.

The trial is open to enrollment at the University of Texas MD Anderson Cancer Center with expected additional participation at The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins and Memorial Sloan Kettering Cancer Center. The Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology will provide artificial intelligence services throughout the trial, including biomarker and genomic analyses, which is expected to expand the Company’s knowledge of the treatment paradigm.

This new Phase 1/2 study, titled "A Phase I/II Study Evaluating the Effect of IMNN-001 (IL-12 Plasmid Formulated with PEG-PEI-Cholesterol Lipopolymer) on Minimal Residual Disease (MRD) as determined by Second Look Laparoscopy when Administered in Combination with Bevacizumab and Neoadjuvant Chemotherapy in Subjects Newly Diagnosed with Advanced Ovarian, Fallopian Tube or Primary Peritoneal Cancer," is expected to enroll 50 patients with Stage III/IV advanced ovarian cancer and is being led by principal investigator Amir Jazaeri, M.D., Professor of Gynecologic Oncology and Reproductive Medicine at MD Anderson. The study will be partially funded by a third party.

Partnerships and Collaborations

Collaborative Research Agreement with The Wistar Institute’s Vaccine & Immunotherapy Center, Acuitas Therapeutics and Transomic Technology. In January 2023 the Company announced a collaborative research agreement with The Wistar Institute, a global leader in biomedical research, through its Vaccine & Immunotherapy Center, to research and develop new vaccine formulations utilizing the Company’s PLACCINE modality for the development of vaccines for infectious diseases. The Wistar Institute Vaccine & Immunotherapy Center possesses world-renowned expertise in cancer, immunology, infectious diseases and vaccine creation. They are uniquely positioned to advance new vaccine formulations and will facilitate further expansion and development of PLACCINE with the goal of expanding vaccine targets ideally matched for the Company’s novel formulated DNA delivery platform.

During the fourth quarter of 2022 the Company entered into an agreement with Acuitas Therapeutics to evaluate PLACCINE Plasmid DNA with Acuitas’ lipid nanoparticle delivery system. Under this agreement, Acuitas will evaluate the administration of IMUNON’s vector constructs formulated in various LNP formulations for gene expression and immunogenicity in murine models. The Company also announced a strategic investment in Transomic Technology to utilize its custom vector construction services to continue to generate plasmids that are being developed and evaluated by IMUNON as part of the Company’s DNA vaccine program. As a condition of the investment, Michael H. Tardugno, IMUNON’s executive chairman, has joined the Transomic board of directors.

Corporate Developments

Received $1.6 Million in Non-Dilutive Funding from the Sale of New Jersey Net Operating Losses. In January 2023, the Company announced it received $1.6 million in net cash proceeds from the sale of approximately $1.7 million of its unused New Jersey net operating losses (NOLs). The NOL sales cover the tax year 2021 and are administered through the New Jersey Economic Development Authority’s (NJEDA) Technology Business Tax Certificate Transfer (NOL) program. This non-dilutive funding further strengthened the Company’s balance sheet. The Company plans to sell an additional $1.9 million of unused New Jersey NOLs available to the Company under the program in 2023.

Financial Results for the Year Ended December 31, 2022

IMUNON reported a net loss for 2022 of $35.9 million, or $5.03 per share, compared with a net loss for 2021 of $20.8 million, or $3.83 per share. Operating expenses were $25.4 million for 2022, an increase of $3.9 million or 18% from $21.5 million for 2021. The Company recognized tax benefits from the sale of its New Jersey NOLs of $1.6 million and $1.4 million in tax in 2022 and 2021, respectively.

Research and development (R&D) expenses were $11.7 million for 2022, an increase of $1.1 million from $10.6 million for 2021. Costs associated with the OVATION 2 Study were $1.5 and $1.3 million for 2022 and 2021, respectively. Costs associated with the Phase 3 OPTIMA Study decreased to $0.5 million for 2022, compared with $1.0 million for 2021. Other clinical and regulatory costs were $2.3 million for 2022, compared with $2.6 million for 2021. 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 increased to $6.1 million for 2022, compared with $4.3 million for 2021. CMC costs decreased to $1.2 million for 2022, compared with $1.5 million for 2021 due to the discontinuation of the ThermoDox clinical development program in primary liver cancer.

General and administrative expenses were $13.7 million for 2022, compared with $10.9 million for 2021. This $2.8 million increase was primarily attributable to higher professional fees including legal fees to defend various lawsuits filed after the announcement in July 2020 of the OPTIMA Phase 3 study results, higher compensation expenses related to the CEO succession plan and higher staffing costs, which were partially offset by lower non-cash stock compensation expense.

Other non-operating expenses were $12.5 million for 2022, compared with $1.1 million for 2021. This increase was attributable to the following:

Due to the continuing deterioration of the public capital markets in the biotech industry in 2021 and 2022 and its impact on the market capitalization of companies in this sector, the Company reviewed its In-Process Research & Development (IPR&D) asset for impairment. After conducting a detailed analysis, the Company determined that the IPR&D asset was impaired. As of December 31, 2022, the Company wrote off the $13.4 million carrying value of this asset, thereby recognizing a non-cash charge of $13.4 million.
The Company wrote off the earn-out milestone liability because of the requirements not being achieved and recognized a non-cash gain of $5.4 million during 2022 as a result of the change in the fair value of the earn-out milestone liability.
The Company recognized interest expense of $5.0 million for 2022, compared with $0.6 million for 2021. 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 SVB and Horizon loan facilities, the Company incurred $0.5 million in interest expense in 2022, compared with $0.6 million in 2021. In connection with the termination of the Horizon loan facility in 2021, the Company paid early termination and end-of-term charges to Horizon and recognized $0.2 million as a loss on debt extinguishment.
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 $30.0 million of Series A & B convertible redeemable preferred stock.
Investment income from the Company’s short-term investments was $0.5 million for 2022. Investment income was insignificant for 2021.
Net cash used for operating activities was $23.1 million for 2022, compared with $16.2 million for 2021. This increase was primarily due to the one-time payment of $4.5 million in interest expense resulting from the sale and subsequent redemption of $30.0 million of Series A & B convertible redeemable preferred stock, as well as higher operating costs attributable to the development of IMNN-001 and the PLACCINE DNA technology platform and higher legal and professional fees. Cash provided by financing activities of $6.7 million during 2022 resulted from an at-the-market equity offering with no warrants and sales under the Company’s At-the-Market Equity Facility.

The Company ended 2022 with $38.9 million in cash, investments, accrued interest receivable and restricted cash. Along with future planned sales of the Company’s remaining New Jersey NOLs, the Company believes it has sufficient capital resources to fund its operations into 2025.

Conference Call and Webcast

The Company is hosting a conference call to provide a business update, discuss 2022 financial results and answer questions at 11:00 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 2022 Earnings Call. A live webcast of the call will be available here.

The call will be archived for replay until April 13, 2023. 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 5236742. A webcast of the call will be available here for 90 days.