On March 30, 2023 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, reported financial results for the fourth quarter and year ended December 31, 2022 and provided a corporate update (Press release, Ocuphire Pharma, MAR 30, 2023, View Source [SID1234629612]).

"2022 proved to be a year of significant clinical, regulatory, patent, and strategic partner milestones, positioning the company for further success in 2023," said Mina Sooch, MBA, founder and CEO of Ocuphire Pharma. "Our first NDA for Nyxol in the reversal of pharmacologically-induced mydriasis has a PDUFA action date the FDA this September, and we look forward to working with the FDA through the regulatory review process. For APX3330, our oral candidate for diabetic eye disease, the ZETA-1 Phase 2 trial achieved statistical significance on a potential registration endpoint of slowing progression of diabetic retinopathy, and we are preparing for an End-of-Phase 2 meeting. Across the Nyxol and APX3330 programs, we are poised for potential late-stage clinical, regulatory, and product approval catalysts in 2023."

Cam Gallagher, Ocuphire’s Chairman of the Board added, "Our collaboration with Viatris is transformative for the company, and we are excited with Viatris having selected the Nyxol portfolio of indications as one of the key elements of its plan to create a global eye care leader. The partnership provides an externally financed pathway to completing development and regulatory activities for Nyxol and to executing successful US and global commercial launches, if approved. The financial elements of the agreement have greatly strengthened the balance sheet and provides capital to advance the APX3330 program to a pivotal stage.

Key Anticipated Future Milestones

Partner or
OCUP
Name
Study
Milestone
Anticipated
Indication
1H 2023
2H
2023
APX3330
ZETA-1
End of Phase 2 FDA meeting
Diabetic Retinopathy

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•
MR-140
(Nyxol)
MIRA program
PDUFA date Sept 28, 2023
Reversal of
Pharmacologically-
induced Mydriasis

•
MR-141
(Nyxol and Nyxol+LDP)
VEGA-3
Initiate 2nd Phase 3 trial
Presbyopia
•
VEGA-2
Report 1st Phase 3 topline data
Presbyopia

•
MR-142
(Nyxol)
LYNX-2
Initiate 2nd Phase 3 trial
Night Vision or Dim
Light Disturbances
(DLD)
•
MR-141 and MR-142
(Nyxol)
LYRA-1
Initiate long-term safety trial
Presbyopia and DLD
•

Recent Business Highlights

Advances in Clinical and Regulatory Development

•
In February 2023, the Company announced that U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) for Nyxol in RM and set a PDUFA date of September 28, 2023. The NDA was submitted in November 2022 and was supported by positive results from the comprehensive MIRA clinical programs (MIRA-1, MIRA-2, MIRA-3 and MIRA-4) collectively involving over 600 subjects, including pediatric subjects over 3 years old.

•
In January 2023, the Company announced topline results from the ZETA-1 Phase 2 trial of oral APX3330 for the treatment of diabetic retinopathy (DR). Oral APX3330 achieved statistical significance on a key pre-specified secondary endpoint of binocular ≥ 3-step worsening of DRSS and demonstrated favorable safety and tolerability after 24 weeks of treatment. This binocular secondary endpoint is a potential Phase 3 registration endpoint which the Company plans a meeting with the FDA to formally agree on this endpoint and Phase 3 development in an End-of-Phase 2 meeting in 2H 2023. The safety and tolerability of APX3330 were favorable in diabetic patients and consistent with prior trials in non-ophthalmic indications. Prevention of progression is a clinically meaningful outcome that may change the treatment paradigm for a large number of diabetic retinopathy (DR) patients.

•
In January 2023, the Company announced the initiation of the VEGA-2 Phase 3 pivotal trial, with the first patient enrolled in late December. VEGA-2 is evaluating treatment efficacy and safety for two labels for presbyopia: Nyxol alone and Nyxol with adjunctive Low Dose Pilocarpine (LDP) therapy. The Company plans to initiate a second Phase 3 pivotal trial in presbyopia (VEGA-3), and a one-year safety study (LYRA-1) in 2023.

Presentations, Publications, and Conferences

•
From January 2022 through March 2023, more than 20 papers, posters, and panel talks were presented at over 30 medical and industry conferences with updates on Nyxol in RM by Justin Schweitzer, OD, Leslie O’Dell, OD and Shane Foster, OD, presbyopia by Mitch Jackson, MD and Mitch Ibach, OD, and night vision disturbances by Shane Kannarr, OD and on APX3330 in DR by Douglas Devries, OD, Peter Kaiser, MD and Rishi Singh, MD.

•
In November 2022, the Company announced publication of an earlier Phase 2 clinical trial in patients with severe night vision disturbances in the BMC Ophthalmology peer-reviewed journal. The publication can be accessed here.

•
In October 2022, the Company held a key opinion leader (KOL) webinar on oral APX3330 featuring presentations by KOLs Peter Kaiser, MD, from the Cleveland Clinic, Caroline Baumal, MD, from Tufts Medical Center, and David Lally, MD, from New England Retina Consultants. A replay of the event, including slides, can be found on the corporate website here.

Intellectual Property

•
For the Nyxol Program:

o
The United States Patent & Trademark Office (USPTO) issued U.S. Patent No. 11,400,077 in August 2022 with claims directed to methods for treating mydriasis using phentolamine mesylate, which has a term extending into year 2039.

o
The USPTO issued U.S. Patent No. 11,566,005 in January 2023 with claims directed polymorphic forms of phentolamine mesylate and methods using and preparing the same, which has a term extending into year 2042.

•
For the APX3330 Program:

o
The USPTO issued U.S. Patent No. 11,351,130 in June 2022 with claims directed to methods of treating inflammation and chronic pain using APX3330 in combination with certain additional therapeutic agents, which has a term extending into year 2038, not including any patent term extension.

o
The European Patent Office issued European Patent No. 3725309 in November 2022 with claims directed to APX3330 for use in inhibiting advanced macular degeneration, which has a term extending to year 2028, not including any supplementary protection certificate.

Corporate

•
In November, the Company entered into an exclusive license agreement with FamyGen Life Sciences, Inc. (Famy), which was subsequently acquired by Viatris Inc. (Nasdaq: VTRS), for the development and commercialization of Nyxol across three indications in US, Europe, Japan, India, China and other global markets. Viatris, a leading global healthcare company will be responsible for the commercialization of Nyxol following each regulatory approval. Under the terms of the agreement, Ocuphire received an upfront payment of $35 million. Ocuphire has the potential to receive a $10 million milestone payment upon FDA approval for the RM indication later in 2023 and thereafter to receive additional regulatory milestone payments for presbyopia and night vision disturbances indications. Upon commercialization, Ocuphire will receive tiered double-digit royalties on worldwide net sales through 2040 and is eligible to receive sales milestone payments upon achievement of certain annual sales thresholds.

Fourth Quarter Ended December 31, 2022 Financial Highlights

As of December 31, 2022, Ocuphire had cash and cash equivalents of approximately $42.6 million. The Company has no debt. Based on current projections, management believes the present cash on hand will be sufficient to fund operations into 2025.

License and collaborations revenue was $39.9 million for the quarter and year ended December 31, 2022, compared to zero and $0.6 million for the quarter and year ended December 31, 2021, respectively. Revenue during 2022 was derived from the Nyxol License Agreement signed with Famy in the fourth quarter of the year.

General and administrative expenses for the quarter and year ended December 31, 2022 were $2.1 million and $7.3 million, respectively, compared to $1.4 million and $8.1 million for the quarter and year ended December 31, 2021, respectively. The decrease in the year over year period was primarily attributable to a non-cash settlement cost in 2021, decreases in stock-based compensation and other operating expenses. Partially offsetting the expense decreases from the prior year were increases in administrative employee headcount costs, legal fees and professional service costs. General and administrative expenses included $1.1 million in stock-based compensation expense in each of the years ended December 31, 2022, and 2021.

Research and development expenses for the quarter and year ended December 31, 2022 were $3.6 million and $14.4 million, respectively, compared to $4.7 million and $15.2 million for the quarter and year ended December 31, 2021, respectively. The decrease in the year over year period was primarily attributable to decreases in contract research organization expenses along with an associated decrease in manufacturing activities, offset in part by cost increases attributable to staff headcount and consulting services, as well as increases attributable to regulatory and other research and development efforts. Research and development expenses also included $0.7 million and $0.8 million in stock-based compensation expense during the years ended December 31, 2022 and 2021, respectively.

Net income for year ended December 31, 2022 was $17.9 million compared to a net loss of $56.7 million for the year ended December 31, 2021. The $56.7 million net loss in 2021 included a non-cash expense of $33.8 million related to the fair value change in warrant liabilities. Net income for the fourth quarter ended December 31, 2022 was $33.9 million compared with a net loss of $6.3 million in the comparable period of 2021.

Basic net income per share for the quarter and year ended December 31, 2022 was $1.63 and $0.90 per share, respectively, compared to a basic and diluted net loss per share of ($0.35) and ($3.82) per share, respectively, for the comparable periods in 2021. Diluted net income per share for the quarter and year ended December 31, 2022 was $1.58 and $0.87 per share, respectively.

For further details on Ocuphire’s financial results, refer to the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 to be filed with the Securities and Exchange Commission.

On March 30, 2023 Pulse Biosciences, Inc. (Nasdaq: PLSE), a company leveraging its novel and proprietary nanosecond pulsed field ablation (nsPFA) technology for electrophysiology and the treatment of atrial fibrillation, reported financial results for the fourth quarter and full year ended December 31, 2022 (Press release, Pulse Biosciences, MAR 30, 2023, View Source [SID1234629610]).

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Company Updates

●

Announced the progression of its corporate strategy elevating the focus on the development of nsPFA delivery devices, including a clamp and catheter, for cardiac ablation to treat atrial fibrillation (AF) in both the surgical and electrophysiology lab settings. Current company focus:
○ Develop and advance the cardiac clamp through the appropriate FDA regulatory path
○ Complete all catheter product and regulatory milestones required to treat patients

●

Completed cardiac ablation clamp commercial design. Pre-submission meeting with FDA planned for 2Q23 to discuss the regulatory requirements.

●

Advanced development of the nsPFA cardiac ablation catheter system intending to pursue a first-in-human safety feasibility study.

●

AF Symposium poster presentation demonstrated the ability of the Company’s nsPFA catheter system to perform cardiac ablation and mapping with a single catheter in preclinical studies.

●

Reduced fourth quarter 2022 cash usage to $8.0 million dollars, a reduction of $2.6 million compared to the third quarter of 2022 and in-line with prior guidance, and cash on hand is expected to be sufficient to fund planned operations into the third quarter of 2024.

● Extended the maturity date of the loan facility from Robert Duggan to September 2024.

"As a bioelectric medicine company committed to health innovation, shifting our focus to the treatment of atrial fibrillation is intended to maximize the benefit our technology can provide patients and the value we can create as a company. Based on our preclinical evidence, we believe the delivery of nsPFA utilizing the CellFX System with our clamp and catheter devices will offer a differentiated and improved safety and efficacy profile for cardiac ablation," said Kevin Danahy, Chief Executive Officer of Pulse Biosciences. "In 2023 we will remain laser focused on advancing the development and regulatory progress for both devices."

Fourth Quarter 2022 Results

As a result of the discontinuation of commercial dermatology activity there was no revenue recognized in the fourth quarter of 2022.

Total GAAP cost and expenses, representing cost of revenues, research and development, sales and marketing, and general and administrative expenses, for the three months ended December 31, 2022 were $8.7 million, compared to $16.3 million for the prior year period. Non-GAAP cost and expenses for the three months ended December 31, 2022 were $7.7 million compared to $12.3 million in the prior year period.

GAAP net loss for the three months ended December 31, 2022 was ($9.2) million compared to ($15.4) million for the three months ended December 31, 2021. Non-GAAP net loss for the three months ended December 31, 2022 was ($8.1) million compared to ($11.5) million for the three months ended December 31, 2021.

Full Year 2022 Results

Revenue for 2022 was $700 thousand driven mainly by commercial dermatology activity in the first half of the year.

Total GAAP costs and expenses, representing cost of revenues, research and development, sales and marketing and general and administrative expenses, for 2022 were $58.8 million, compared to $64.4 million in 2021. Non-GAAP cost and expenses for 2022 were $51.3 million compared to $48.7 million in the prior year.

GAAP net loss for the full year of 2022 was ($58.5) million compared to ($63.7) million for 2021. Non-GAAP net loss for 2022 was ($51.1) million compared to ($47.9) million in 2021.

Cash, cash equivalents and investments totaled $61.1 million as of December 31, 2022 compared to $28.6 million as of December 31, 2021 and $69.2 million as of September 30, 2022. Cash used in the fourth quarter of 2022 totaled $8.0 million compared to $13.4 million used in the same period in the prior year and $10.6 million used in the third quarter of 2022. Excluding net proceeds from financing, cash used in the full year of 2022 totaled $47.3 compared to $52.9 million used in 2021.

Reconciliations of GAAP to non-GAAP cost and expenses and net loss have been provided in the tables following the financial statements in this press release. An explanation of these measures is also included below under the heading "Non-GAAP Financial Measures."

Webcast and Conference Call Information

Pulse Biosciences’ management will host a conference call today, March 30, 2022, beginning at 1:30pm PT. Investors interested in listening to the conference call may do so by dialing 1-877-704-4453 for domestic callers or 1-201-389-0920 for international callers. A live and recorded webcast of the event will be available at View Source

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