Clear registration pathway and FDA agreement on the ORALTO Phase 3 trial for MAT2203 represent critical steps forward and supports partnership discussions

Continued success of Compassionate/Expanded Use Access Program demonstrates potential of MAT2203 in treating multiple severe invasive fungal infections, including invasive aspergillosis

Successful in vivo LNC platform studies demonstrating (a) the oral delivery of small oligonucleotides with biological activity and (b) the dramatically improved safety of LNC-docetaxel over IV-docetaxel, with similar efficacy, supports the future use of the LNC platform in inflammation and oncology

Conference call begins at 4:30 p.m. Eastern time today

BEDMINSTER, N.J., March  27, 2024  (GLOBE NEWSWIRE) -- Matinas BioPharma Holdings, Inc. (NYSE American: MTNB), a clinical-stage biopharmaceutical company focused on delivering groundbreaking therapies using its lipid nanocrystal (LNC) platform delivery technology, reports 2023 financial results and provides a business update.

"A clear regulatory approval pathway for oral MAT2203 is a critical step toward future commercialization in its initial indication of the treatment of invasive aspergillosis in patients with limited treatment options," said Jerome D. Jabbour, Chief Executive Officer of Matinas. "Reaching agreement with FDA on the design of the ORALTO registration trial and the consistent successful treatment outcomes in our ongoing Compassionate/Expanded Use Access Program have elevated our confidence that, if approved, MAT2203 could represent a new treatment paradigm for addressing the unmet medical need in the treatment of a variety of difficult to treat invasive fungal infections. With these important elements secured, we are actively pursuing partnership opportunities to advance MAT2203 into Phase 3 as quickly as possible.

"We have also advanced the application of our LNC platform into exciting areas, including oncology and inflammation. In the oncology space, our in vivo data demonstrate that treatment with oral LNC-docetaxel at dosages substantially higher than those proven effective in targeting melanoma tumors resulted in tumor size reductions comparable to IV-docetaxel and was associated with none of the toxicity (body weight loss) observed with conventional IV-docetaxel. In the field of inflammation, we have demonstrated the successful oral delivery of biologically active – and potentially therapeutic – small oligonucleotides in several inflammatory disease models.

"Overall, we are pleased with all of the progress made by our team throughout 2023 and so far in 2024, and our focus remains on executing our strategic plan to make the LNC platform the preferred next-generation orally available intracellular drug delivery technology, facilitating an internal and external pipeline of drug candidates."

Key Program Updates

MAT2203 (Oral Amphotericin B) Program

Phase 3 ORALTO Registration Trial

• Matinas reached alignment with the FDA on the design of a single Phase 3 registration trial of oral MAT2203 in patients with invasive aspergillosis who have limited treatment options. This is a serious and life-threatening invasive fungal infection that occurs primarily in severely immunocompromised patients, including those with hematological malignancies and in transplant recipients. In 2022, the World Health Organization released its Fungal Priority Pathogen List that designated the most common invasive aspergillosis, Aspergillus fumigatus, to be in the Critical Priority group, which is designated as the highest perceived public health threat. Aspergillus fumigatus is also included in the FDA qualified designation list of pathogens that pose a serious and life-threatening risk.

• The Phase 3 randomized, multicenter, open-label, adjudicator-blinded ORALTO trial will evaluate the efficacy and safety of MAT2203 as an oral step-down treatment following two days of treatment with AmBisome (liposomal IV-amphotericin B) compared with the standard of care in patients with invasive aspergillosis who have limited treatment options. The primary efficacy endpoint in this non-inferiority study is all-cause mortality at study day 42. Key secondary objectives include demonstration of superiority for treatment-related toxicities leading to changes in treatment, long-term survival benefit of MAT2203 using all-cause mortality at study day 84 and the impact of MAT2203 on healthcare resource utilization and quality of life.

• The Phase 3 ORALTO trial is expected to include approximately 65 investigator sites in the U.S., Europe, South America, the Middle East and Asia Pacific. Enrollment is expected to include approximately 216 adults with recently diagnosed probable or proven invasive aspergillosis who are being treated with AmBisome due to their inability to receive an IV mold-active azole and with limited alternative treatment options. Following up to two days of treatment with AmBisome, eligible participants will be randomized 2:1 to receive either oral MAT2203 or continued AmBisome treatment followed by standard of care. All study participants will receive up to 12 weeks of treatment starting from the first day of treatment with AmBisome. All study participants are expected to be hospitalized during the initial AmBisome treatment period. After step-down to oral MAT2203, study participants may be discharged to continue treatment on an outpatient basis, as clinically appropriate. An independent Data Review Committee, which will be blinded to treatment, will adjudicate primary and secondary endpoints, including clinical, radiological, and mycological responses.

• Once approximately 75% of participants are enrolled, an independent Data Safety Monitoring Board will review the pooled all-cause mortality rate in a blinded fashion to ensure sample size assumptions are reasonable and the study is adequately powered. Should the pooled event differ substantially from expected levels, a sample size adjustment can be made to the trial.

• The Company is engaged in active dialogues with potential partners and is seeking to finalize a partnership as soon as possible in order to commence the Phase 3 ORALTO trial.

MAT2203 Compassionate/Expanded Use Access Program

• A total of 19 patients with serious/life-threatening invasive fungal infections have been enrolled in the program to date, with others being evaluated. The infections treated include a variety of micro-organisms (including Aspergillus, Mucorales species, Candidiasis, Fusarium and suspected Coccidioides) at multiple sites of infection including brain, bladder/colon, bone, lung, sinus, and skin. The majority of enrolled patients are post-transplant or are undergoing treatment for underlying malignancies.

• Patients have been enrolled in the Program at prestigious institutions including the University of Michigan, Johns Hopkins, Nationwide Children's Hospital, City of Hope, Vanderbilt University Medical Center, the National Institutes of Health, Children's Hospital of Philadelphia, Memorial Sloan Kettering Cancer Center and the University of California, San Diego School of Medicine.

• Most patients were receiving AmBisome prior to enrollment but developed treatment-limiting nephrotoxicity, and most also required treatment for azole-resistant organisms or had failed azole therapy and had no other treatment options. All patients who transitioned to MAT2203 after developing renal toxicity following treatment with AmBisome experienced a reversal of renal impairment with a return to baseline renal function and no subsequent renal issues. In addition, most patients to date were able to be discharged from the hospital setting and effectively treated at home, supporting the potential significant pharmacoeconomic impact of MAT2203.

• Eight patients who completed the desired course of treatment with oral MAT2203 had complete clinical resolution of their infection and patients with ongoing treatment continue to experience significant clinical improvement.

LNC Platform Updates

Internal Oral LNC Oncology Program

• Conventional docetaxel, a well-known chemotherapeutic agent used in the management of multiple metastatic and unresectable tumors, is only administered intravenously, and is associated with significant side effects and toxicities.

• LNC's crystalline structure encapsulates and protects the body from the docetaxel cargo and selectively delivers drug to tumor cells. This markedly reduces the amount of free drug circulated systemically, avoiding one of the primary drivers of toxicity.

• LNC-docetaxel is an effective targeting vehicle and an efficient delivery platform for oncology applications due to its unique phospholipid composition that allows for targeting and delivering docetaxel to tumor cells that express phosphatidylserine on their surface.

• In vivo study data reported in November 2023 demonstrated that oral LNC-docetaxel effectively targeted melanoma tumors and was able to reduce tumor sizes to a degree comparable to that of IV-docetaxel with no apparent toxicity.

• Additional in vivo study data reported in March 2024 corroborated the lack of toxicity in a more comprehensive safety study with a longer treatment duration and higher doses of oral LNC-docetaxel. Healthy mice administered oral LNC-docetaxel at doses more than 8x greater than IV-docetaxel showed no weight loss, versus an average 20% peak weight loss in mice treated with IV-docetaxel. Mice treated with oral LNC-docetaxel maintained their body weight, which was statistically no different than the weight of control mice treated with oral saline.

Internal Oral LNC Small Oligonucleotide Inflammation Program

• In vivo studies documented the successful oral delivery and biological activity of two different LNC-formulated small oligonucleotides targeting inflammatory cytokines IL-17A and TNFα with reductions in tissue cytokine mRNA in both colitis and psoriasis, along with significant reductions in serum TNFα levels in colitis.

• The LNC-formulated small oligonucleotides evaluated in these studies interfered with cytokine synthesis rather than simply targeting the cytokine itself, which creates additional opportunities for potential future applications of LNC-delivered therapeutics.

Corporate Development

• The Company received notification from the NYSE American LLC that it has regained compliance with the NYSE American LLC continued listing standards by resolving the continued listing deficiency with respect to the low selling price of its common stock as described in Section 1003(f)(v) of the NYSE American Company Guide.

2023 Financial Results

Revenue for 2023 was $1.1 million, which was generated from the Company's research collaborations with BioNTech SE and Genentech Inc. This compares with revenue for 2022 of $3.2 million, which was generated from the Company's research collaboration with BioNTech SE.

Total costs and expenses for 2023 were $24.9 million compared with $27.8 million for 2022. The decrease was primarily due to lower clinical trial expenses and lower professional and consulting fees. Income from selling unused New Jersey net operating losses (NOLs) and research and development tax credits was $0.5 million and $3.5 million for 2023 and 2022, respectively.

The net loss for 2023 was $22.9 million, or $0.11 per share, compared with a net loss for 2022 of $21.0 million, or $0.10 per share.

Cash, cash equivalents and marketable securities as of December 31, 2023 were $13.8 million compared with $28.8 million as of December 31, 2022. Based on current projections, the Company believes its cash position is sufficient to fund planned operations through the third quarter of 2024.

Conference Call and Webcast

Matinas will host a conference call and webcast today beginning at 4:30 p.m. Eastern time. To participate in the call, please dial 877-484-6065 or 201-689-8846. The live webcast will be accessible on the Investors section of the company's website and archived for 90 days.

[Financial Tables to Follow]

Source: Matinas BioPharma Holdings, Inc.

Released March 27, 2024