Rocket Pharmaceuticals to Participate in the Chardan 7th Annual Genetic Medicines Conference

On September 26, 2023 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to participate in a fireside chat at the Chardan 7th Annual Genetic Medicines Conference in New York City on Tuesday, October 3, at 1:00 p.m. ET (Press release, Rocket Pharmaceuticals, SEP 26, 2023, View Source [SID1234635436]).

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A webcast of the fireside chat will be available under "Events" in the Investors section of the Company’s website at View Source

OS Therapies Announces Full Enrollment in PhIIb Clinical Trial AOST-2121 in Recurred, Resected Osteosarcoma with OST-HER2 (Listeria monocytogenes) for Delay or Prevention of Recurrence

On September 26, 2023 OS Therapies, a research and clinical stage biopharmaceutical company, reported Full Enrollment for its lead OST-HER2 (OST31-164) program in its ongoing clinical trial AOST-2121 (NCT04974008) (Press release, OS Therapies, SEP 26, 2023, View Source [SID1234635435]). The trial is being conducted at 21 Children’s Oncology Group (COG) sites across the country for patients with Osteosarcoma that had metastasized to the lungs requiring surgical removal. Historically, 80-90% of these patients would develop additional metastasis within the period of the trial. The planned number of patients have now received treatment and the few remaining slots will be allocated to patients that have already been identified. Patients continue OST-HER2 treatment for 48 weeks and then are followed for survival 2 additional years. The primary endpoints of the trial are Event Free Survival and Overall Survival.

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"Several patients have completed the treatment phase of the trial and many more are undergoing treatment with OST-HER2. We hope that this treatment will reduce additional recurrences and extend the lives of all osteosarcoma patients and look forward to the outcome once all patients have completed treatment," said Dr. Robert Petit, CMO/CSO of OS Therapies. Osteosarcoma is a rare but challenging disease for patients and their physicians, occurring primarily in teens and young adults. "Reaching thirty-nine patients enrolled is a major milestone in this trial, occurring more quickly than projected. A sincere thank you to the patients, families and healthcare providers – the Children’s Oncology Group, and the outstanding leadership of Principle Investigators Damon Reed, MD, and Kelly Bailey, MD," said Paul Romness, CEO of OS Therapies.

About Osteosarcoma

Osteosarcoma is a solid tumor of the bone that predominantly occurs in adolescents and young adults (AYA). Standard treatment includes surgery and chemotherapy. For patients with metastatic osteosarcoma or have recurrence after chemotherapy, the prognosis is poor.

Elpiscience Announces First Patient Dosed for Phase 1 Clinical Trial of Anti-LILRB2 Antibody ES009 in Australia

On September 26, 2023 Elpiscience Biopharmaceuticals ("Elpiscience"), a clinical-stage biopharmaceutical company dedicated to discovering and developing next-generation cancer immunotherapies, reported that the first patient has been dosed in a Phase 1 clinical trial of its anti-LILRB2 monoclonal antibody ES009 in Australia (Press release, Elpiscience, SEP 26, 2023, View Source [SID1234635434]). The objective of the trial is to evaluate its safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary clinical activity.

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LILRB2, also known as ILT4, is an inhibitory receptor widely expressed on the surface of myeloid cells that also contributes significantly to immune suppression in the tumor microenvironment (TME). ES009 specifically binds to a unique epitope on human LILRB2 and potently blocks LILRB2 binding to multiple ligands. By blocking LILRB2-mediated inhibitory signaling, ES009 can reprogram myeloid cells from anti-inflammatory phenotype into pro-inflammatory phenotype, and reinvigorate T cell functionalities. ES009 has demonstrated exceptional potential to reverse immune suppression in the TME and promote anti-tumor immunity in preclinical studies.

"LILRB2 is a key immune checkpoint for tumor immunotherapy whose suppression of immune response is proved as one mechanism of anti-PD(L)1 resistance. With a keen interest in revolutionizing therapies for cancer patients non-responsive or resistant to PD(L)1 treatment, we developed ES009 which has shown best-in-class potential. We believe in the power of enhanced innate immunity in treating cancer and have developed a highly differentiated myeloid cell focused therapeutics portfolio. We will work relentlessly to bring these highly promising therapies to cancer patients with unmet medical needs," said Dr. Hongtao Lu, Co-founder and CSO of Elpiscience.

CEL-SCI Submits Scientific Advice Filing to European Medicines Agency (EMA) for Multikine in the Treatment of Head & Neck Cancer

On September 26, 2023 CEL-SCI Corporation (NYSE American: CVM) reported that it has filed a request for Scientific Advice regarding Multikine* (Leukocyte Interleukin, Injection) immunotherapy for the treatment of newly diagnosed locally advanced squamous cell carcinoma of the head and neck (SCCHN) with the European Medicines Agency’s (EMA’s) Scientific Advice Working Group (Press release, Cel-Sci, SEP 26, 2023, View Source [SID1234635433]).

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Europe is a high priority market for CEL-SCI, as Europe has 150,000 new annual cases of head and neck cancer, more than twice the 68,000 cases diagnosed each year in the U.S.

"We look forward to discussing with the Scientific Advice Working Group the positive clinical data from our completed Phase 3 study of Multikine in head and neck cancer. The goal is to obtain the Working Group’s opinion regarding this information and reach an agreement with them on a plan which will allow us to file for marketing authorization in the EU as soon as possible" stated CEL-SCI’s CEO Geert Kersten. "This is part of our global regulatory approval strategy. We concurrently plan to pursue filings for marketing authorization in multiple countries. There is no question about the dire need for a new and effective treatment for newly diagnosed SCCHN patients, and we are hopeful that regulators will appreciate the robust data and results from our Phase 3 study that demonstrate Multikine’s efficacy and safety."

MaaT Pharma Publishes its Half Year 2023 Results and Provides a Business Update

On September 26, 2023 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer, reported its half year financial results for the six-month period ended June 30, 2023 and provided a business overview (Press release, MaaT Pharma, SEP 26, 2023, View Source [SID1234635432]).

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"Over the first half of 2023, we laid a solid foundation for clinical development and manufacturing scale-up. Regarding MaaT013, our onco-hematology lead product, the lifting of the FDA hold and the upcoming DSMB review for our Phase 3 trial represent pivotal moments in our development. In addition, our second asset, MaaT033, has received orphan drug designation from the EMA, highlighting the pressing medical need for improved treatments in HSCT. Achieving these significant milestones reflects MaaT Pharma’s unwavering commitment to progress and innovation." stated Siân Crouzet, Chief Financial Officer of MaaT Pharma. "These achievements also contribute to the overall positive trends in the global microbiome landscape, as demonstrated by the industry’s latest positive clinical data and the recent approval of the third microbiome drug by regulatory agencies."

Pipeline Highlights

MET-N platform

MaaT013

In onco-hematology:
In April 2023, MaaT013 clinical results in its early access program for 81 patients, previously communicated during the 64th annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), were presented during the 49th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2023).
In April 2023, the U.S. Food and Drug Administration (FDA) lifted the clinical hold and cleared the Investigational New Drug (IND) application for MaaT013 in patients with aGvHD3. MaaT Pharma intends to consult with the FDA on the next steps of the regulatory process to bring MaaT013 to US patients in the most expeditious way possible while the Company continues the late-stage clinical development of MaaT013 in Europe with the ongoing international multicenter, open-label, single arm, pivotal Phase 3 trial (ARES).
As a post-period event, in July 2023, the Company announced that clinical data on MaaT013 as a treatment for aGvHD was published in eClinicalMedicine, one of the Lancet Discovery Science suite’s journals.
As a post period event, the Company announces that the ARES study has recruited the required number of patients and that the DSMB is planned for early Q4.23.
In immuno-oncology:
The PICASSO study, sponsored by APHP, is largely on track for data readout now expected by end of 2024/early 2025. This is the only double-blind randomized clinical trial in the field evaluating a microbiome approach (MaaT013) to enhance the efficacy of Immune Checkpoints Inhibitors (ICI) treatments in patients with metastatic melanoma.
With more than half of the patients now having completed their week 9 visit, the Company is in a position to receive biomarker data from its partner.
MaaT033

In onco-hematology:
In April 2023, MaaT033 data of Phase 1b study CIMON, previously communicated during the 64th annual ASH (Free ASH Whitepaper) meeting, were also presented at the EBMT 2023.
As a post period event, in September 2023, the Company announced that the European Medicines Agency (EMA) had granted MaaT033 orphan drug designation aiming to improve overall survival in patients undergoing HSCT and had recognized the significant benefit that MaaT033 could therefore bring to this patient’s population. The status offers key benefits including market exclusivity, clinical protocol assistance, waivers or reductions in regulatory fees.
In neurodegenerative diseases:
As a post period event, the Company announces that the first patient was dosed in the IASO Phase 1b pilot study (NCT05889572) in ALS (also known as Lou Gehrig’s disease in the US and Charcot’s disease in French-speaking countries). The Company has developed the clinical trial in partnership with the French patients’ association Tous en Selles contre la SLA.
MET-C platform

MaaT034

In combination with immune checkpoint inhibitors in solid tumors
MaaT034 is the first member of the MaaT03X co-culture family from the MET-C platform. First in human is scheduled for 2025, with the manufacture of the first clinical batch in 2024.
As a post period event, the Company announces that two posters have been accepted for the 38th Annual Meeting of Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), Nov 1-5, 2023 in San Diego, CA USA.
Corporate update

In February 2023, the Company announced the successful completion of a capital increase of approximately €12.7 million supported by its existing shareholders including Seventure Partners, PSIM Fund represented by Bpifrance Investissement, Biocodex, Invus, Céleste Management, Skyviews Life Sciences and Tocqueville.
In June 2023, MaaT Pharma announced new appointments to the Board of Directors and Executive team, to align with the Company’s long-term vision and goals:
Karim Dabbagh as Chairman and Nadia Kamal as Director, both independent.
Philippe Moyen as Chief Operating Officer.
In June 2023, MaaT Pharma also announced the appointment of Guilhaume Debroas as Head of Investor Relations.
As a post period event, in July 2023, MaaT Pharma announced joining the Microbiome Therapeutics Innovation Group (MTIG).
As a post period event, in September 2023, the Company and Skyepharma announced completion of the cGMP manufacturing facility and the transfer of MaaT Pharma’s Production and Development teams to the new site.
As a post period event and with deep sorrow, MaaT Pharma announces the sudden passing of Professor Gervais Tougas, who served as our part time acting Chief Medical Officer. The Company has initiated a search for a full-time replacement.
Key Financial Results

The key unaudited financial results for the first half of 2023 are as follows:

Income Statement

In thousands of euros

2023.06

2022.06

Revenue

1 378

494

Cost of Goods Sold

(284 )

(72 )

Gross Margin

1 095

422

Other Income

2 659

1 793

Sales and distribution costs

(541 )

(140 )

General and administrative costs

(2 097 )

(2 115 )

Research and development costs

(9 650 )

(7 328 )

Operating Income (loss)

(8 534 )

(7 368 )

Financial Income

258

Financial Expense

(159 )

(50 )

Net financial income (expense)

99

(49 )

Income (loss) before income tax

(8 435 )

(7 417 )

Income tax expense

Net Income (loss) for the period

(8 435 )

(7 417 )

Prepared in accordance with international standards, IFRS

Revenues totaled €1.4 million for the half year ended June 30, 2023, compared with €0.5 million in the half year ended June 30, 2022, reflecting the increase in demand from healthcare professionals and the treatment of an increased number of patients.

Operating loss amounted to €8.5 million in the first half of 2023 compared with €7.4 million in the first half of 2022, an increase of €1.2 million. This increase reflects the growth of research and development costs which have risen from €7.3 million in the first half of 2022 to €9.7 million in 2023, representing an overall increase of €2.3 million and fully consistent with the advancement of the Company’s research programs, offset in part by the R&D tax credit of €2.7 million included in "Other Income".

The net loss amounts to €8.4 million as of June 30, 2023, compared with €7.4 million as of June 30, 2022, reflecting the growth of the Company and in particular the investment in R&D.

The average number of employees has increased from 39 over the first half of 2022 to 47 for the same period of 2023. As of June 30, 2023, there were 51 employees, of which 39 were dedicated to research and development.

Cash Position

As of June 30, 2023, total cash and cash equivalents were €35.1 million, as compared to €35.2 million as of December 31, 2022.

Over the first half of 2023, the net decrease in cash position amounted to €0.2 million compared with €4.9 million in the first half of 2022. Nonetheless, cash used to finance operations increased by €5.9 million for compared with the first half of 2022 due to the increased in operating expenses, particularly associated with research and development. Cash inflows related to financing activities amounted to €13.2 million as a result of the capital increase of approximately €12.7 million supported by its existing shareholders in February 2023, in addition to financing of the 2022 R&D tax credit for a total of €3.1 million offset by loan repayments totaling €1.5 million.

Total financial debt (including lease liabilities) totaled €13.0 million as of June 30, 2023, of which €0.6 million relates to state-backed loans ("PGE").

Based on the development plans and corresponding cash needs, the Company believes it has sufficient cash to finance its activities into the second quarter of 2024.

The Company has updated its corporate presentation, which can be downloaded here: View Source

Upcoming financial communication*

November 9, 2023 – Q3 2023 Results
*Indicative calendar that may be subject to change.

Upcoming investor and scientific conferences participation

September 27, 2023 – 6th edition – Forum LPB Valeurs Régionales
October 4, 2023 – KBC Securities Life Sciences Conference
October 4-5, 2023 – Portzamparc Seminar Biotech & Health
October 9-10, 2023 – Investor Access Event
November 1-5, 2023 – 38th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)