On April 14, 2022 MaaT Pharma (EURONEXT: MAAT – the "Company"), a French clinical-stage biotech and a pioneer in the development of microbiome-based ecosystem therapies dedicated to improving survival outcomes for patients with cancer reported full-year 2021 revenues and provided a business overview for 2022 (Press release, MaaT Pharma, APR 14, 2022, View Source [SID1234612197]).

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Hervé Affagard, CEO and co-founder of MaaT Pharma stated,

"We are proud of our accomplishments regarding our clinical programs and of the success of our IPO on Euronext last November. With the support of our historical shareholders and new institutional and individual investors, we have already achieved key milestones in our clinical activities (MaaT013 in pivotal Phase 3 and MaaT033 in Phase 1b) and expect to continue this growth path in 2022 with the ongoing patient enrollment for our pivotal Phase 3, the initiation of Phase 2/3 for MaaT033, building our manufacturing facility and preparing the entry of MaaT03X for clinical study in 2023. In line with the strategic roadmap presented during the IPO, we have a good financial visibility up to the end of the third quarter of 2023."

Key Financial Results

The key audited financial results for the 2021 full-year are as follows:

Income Statement

Prepared in accordance with international standards, IFRS

Revenues totaled €1.0 million for the year ended December 31, 2021, which includes compensation invoiced since the first half of 2021 from the ATUn program (Temporary Authorization for Use, now known as compassionate access following legislative changes). The gross margin generated by the compassionate access program amount to €0.8 million.

Operating expense amounted to €8.9 million compared with €5.3 million for 2020, an increase of €3.6 million. This increase reflects the growth of research and development costs which have risen from €6.1 million in 2020 to €9.1 million in 2021, representing an overall increase of €3.0 million and consistent with the advancement of activities:

MaaT013: conclusion of the Phase 2 clinical trial, HERACLES, for the treatment of acute Graft-versus-Host Disease (aGvHD), for which an oral presentation of additional data was conducted at the American Society of Hematology (ASH) (Free ASH Whitepaper) Conference in December 2021. Preparation and initiation of the pivotal clinical trial, ARES, for which the first patient was dosed in March 2022
MaaT033: continuation of the Phase 1b clinical trial, CIMON, for which positive interim engraftment data and a satisfactory safety were announced in January 2022
MaaT03x: conduct of pre-clinical trials
Conclusion of a partnership with Skyepharma to establish cGMP manufacturing facility dedicated to ecosystem microbiome-based therapeutics which will be operational in 2023 and for which an initial down payment was made in 2021
General and administrative expenses amounted to €2.7 million compared with €1.3 million in 2020 reflecting the structuring of the Company to meet the needs of a public listing and to support the different clinical and development programs.

The net loss amounts to €9.0 million for the year ended 31 December 2021 compared with €5.3 million for the year ended 31 December 2020, reflecting the roadmap presented during the Company’s IPO.

Average annual employees evolved from 24 in 2020 to 33 in 2021 following the strengthening of the Medical Affairs team, along with the R&D and Technical departments as well as Business development. Dr John Weinberg, CMO, has resigned from his position to pursue other activities and will leave the Company in early May. Following the recruitment of several experienced personnel within the clinical and regulatory teams, no impact is expected on the ongoing clinical trials because of this change.

Cash Position

As of December 31, 2021, total cash and cash equivalents were €43.3 million, as compared to €15.3. million as of June 30, 2021, and €19.9 million as of December 31, 2020.

The net increase in cash position of €23.4 million between December 31, 2020 and December 31, 2021 is due to the Company’s IPO on Euronext and corresponding capital raise generating net cash inflow of €32.4 million, offset by the financing of operations, including R&D and general & administrative costs, for a total of €7.9 million. Net cash outflows from debt repayments over 2021 amounted to €1.0 million and total financial debt (including lease liabilities) totaled €6.5 million as of December 31, 2021, of which €1.0 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 up until the end of the third quarter of 2023.

Major milestones achieved in 2021 and at the beginning of 2022

March 2021: Release of positive topline results for Phase 2 trial evaluating MaaT013 in a high-risk patient population with grade III-IV steroid-refractory, gastrointestinal-predominant acute graft-versus-host disease.
July 2021: Non-dilutive funding of €1.9 million for its "MEPA" project through the France Relance "Resilience" program to support the industrialization of manufacturing processes for a new generation of microbiome ecosystem therapies in immuno-oncology.
November 2021:
Initial Public Offering on Euronext: MaaT Pharma became the first company developing microbiome-based drugs to be listed on the regulated market of Euronext Paris and raised €35.7 million
As part of the France Relance plan, the Company was a successful candidate to the 4th Investment for the Future Program ("4ème Programme Investissements d’Avenir" or PIA4) for its METIO project ("Development of the first European innovative Microbiome Ecosystem Therapies in Immuno-Oncology"), which makes it eligible to €4.26 million in funding, for which a first payment of €1.1 million was received in January 2022.
December 2021 : Presentation of additional promising results from Phase 2 trial in 24 patients with steroid-resistant Grade III-IV gastrointestinal aGvHD (GI-aGvHD) and from a compassionate use program (Early Access Program or EAP) for MaaT013 in France in 52 patients with Grade II-IV GI-aGvHD. Safety signals were consistent with the adverse events profile expected in this patient
January 2022: Release of positive preliminary and interim engraftment data for MaaT033 allowing early termination of Phase 1b study investigating the maximum tolerated dose of MaaT033 in patients with acute myeloid leukemia (AML) who have undergone intensive chemotherapy.
February 2022: Announcement of the partnership with Skyepharma providing MaaT Pharma with a dedicated +16,150 square foot site, with the potential to go up to 32, 300 sq ft if needed, to increase its cGMP manufacturing capacities to support clinical and then commercial development of its most advanced assets MaaT013 and MaaT033, as well as to expand R&D manufacturing capacities for its new drug generation (MaaT03x).
March 2022:
Initiation of clinical pivotal Phase 3 trial in Europe, a world first for a microbiome-based therapy in onco-hematology: an open-label, single-arm study (NCT04769895), evaluating the safety and efficacy of MaaT013, the high-richness, high-diversity lead Microbiome Ecosystem Therapy (MET) as a third-line therapy in patients with gastrointestinal aGvHD.
Implementation of a liquidity contract with Kepler Cheuvreux to enhance the liquidity of the MaaT Pharma shares admitted to trading on Euronext Paris in conformity with applicable legislation. A total of 200,000 euros will be allocated to the liquidity contract.
April 2022: Initiation of a randomized, placebo-controlled Phase 2a proof of concept clinical trial, sponsored by AP-HP, evaluating MaaT013 in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy) and nivolumab (Opdivo), which are standard first line treatments for patients with metastatic melanoma.
Key milestones targeted in 2022

First semester 2022: The Company expects to publish the full topline results of the Phase 1b clinical trial of MaaT033, its second drug candidate, performed in patients with acute myeloid leukemia.

End of second semester 2022: A pivotal Phase 2/3 may start to evaluate MaaT033 as a prophylactic treatment in patients with liquid tumors receiving allo-HSCT.

Ongoing: In line with 2021, the Company continues its early access program in France, allowing patients to benefit from early access to MaaT013 therapy, mainly for the treatment of acute Graft-vs-host-Disease. Outside of France, the Company has responded positively to individual requests for compassionate access in other European countries.

Upcoming financial communication*

May 05, 2022 – Revenues and Cash Position Quarter 1
May 31, 2022 – Annual General Meeting
July 28, 2022 – Revenues and Cash Position Quarter 2
September 29, 2022 – Half-year Results 2022
*Indicative calendar that may be subject to change.

Upcoming investor conference participation

April 21, 2022 – 14th Kempen Life Sciences Conference, Amsterdam
June 30, 2022 – 9th Portzamparc Annual conference, Paris
September 15 and 16, 2022 – KBCS Life Sciences Conference

On April 14, 2022 ISA Pharmaceuticals reported that a clinical trial using ISA’s novel AMPLIVANT adjuvant technology has started (Press release, ISA Pharmaceuticals, APR 14, 2022, View Source [SID1234612196]). The trial, run by partner Scancell Holdings plc (AIM: SCLP, "Scancell") is a multicentre clinical trial, testing the Moditope vaccine Modi1. This first-in-human clinical trial brings Modi-1 to patients with triple negative breast cancer, ovarian cancer, head and neck cancer, and renal cancer using the AMPLIVANT adjuvant technology to boost immune response to the therapy.

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The main study is an open label clinical trial. The initial part of the trial will assess the safety and immunogenicity of Modi-1. In addition, the effect of Modi-1 in promoting T-cell infiltration into the tumour will be assessed in a neoadjuvant cohort of patients with head and neck cancer treated with Modi-1, randomised to treatment with or without a checkpoint inhibitor, prior to the first surgical resection.

The Modi-1 peptides are linked to AMPLIVANT, a potent adjuvant and toll-like receptor (TLR) 1/2 agonist, which enhances the immune response 10-100 fold, resulting in highly efficient tumour clearance, including protection against tumour recurrence, in preclinical models. AMPLIVANT is the subject of a worldwide non-exclusive licensing and collaboration agreement with Scancell for the manufacturing, development, and commercialisation of Modi-1.

Professor Kees Melief, Chief Scientific Officer, ISA Pharmaceuticals, commented: "Adjuvants are crucial components of vaccines which boost efficacy, however there is lack of new, innovative, and effective adjuvants in development to add to a limited armamentarium. As such this is an important achievement and highlights the productive collaboration, we have with Scancell. The trial provides a further opportunity to demonstrate the potent adjuvant properties that AMPLIVANT confers on therapeutic vaccines to potentially benefit patients with a broad range of solid tumours."

Professor Lindy Durrant, Chief Executive Officer, Scancell, commented: "This is the first time we have taken a product from our Moditope platform into cancer patients and is a major step forward for Scancell and our collaboration with ISA Pharmaceuticals. We are very excited about the prospects for Modi-1 based on the dramatic regression of large tumours in our preclinical models."

Amplivant is a registered trademark in Europe

On April 13, 2024 Ingenium Therapeutics reported the company had attracted ‘Pre-Series A’ investment worth 5.7 billion won (Press release, Ingenium Therapeutics, APR 13, 2022, View Source [SID1234643520]).

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Venture capital (VC) companies such as Capstone Partners, K-Ground, Kairos Investment, Maple, and Laplace participated in this investment.

Ingenium is developing an anti-cancer natural killer (NK) cell therapy. The patent was transferred from the Korea Research Institute of Bioscience and Biotechnology. It is explained that clinical results (data), management’s expertise, and potential as a next-generation anticancer drug led to this investment.

Ingenium plans to apply for phase 1 and 2 clinical trials to the Ministry of Food and Drug Safety at the end of this month. It is planned to be conducted on patients with refractory acute myeloid leukemia (refractory AML) at three domestic medical institutions, including Asan Medical Center, Samsung Medical Center, and Seoul St. Mary’s Hospital.

For lymphocytic leukemia, chimeric antigen receptor T cell (CAR-T) treatment is commercially available. On the other hand, there is currently no specific treatment for incurable acute myeloid leukemia. Even after receiving an allogeneic hematopoietic stem cell transplant, the recurrence rate within one year is 60-70%. Also, the 5-year survival rate is less than 10%.

Ingenium also reported that it succeeded in mass producing NK cells. The company’s NK cells are expected to have strong anti-cancer abilities due to their high expression of surface active receptors and high levels of ‘interferon gamma (IFN-r)’. It was said that cell viability was also high, overcoming the problem of ‘short survival period in the body’, which was known to be a limitation of existing NK cell treatments.

A company official said, "As a result of researchers’ clinical trials conducted by Korea Life Insurance Co., Ltd. on about 100 patients with incurable leukemia between 2009 and 2018, the survival rate increased more than three times compared to existing hematopoietic stem cell transplantation," adding, "The goal is to obtain conditional approval in 2024 when phase 2 is completed." "He said.

Ingenium is also preparing for global clinical trials with the goal of entering next year. We plan to pursue clinical trials not only for intractable leukemia, but also for lung cancer, liver cancer, and colon cancer, which have previously shown effectiveness in preclinical trials.

On April 13, 2022 Blue Water Vaccines Inc. ("BWV" or "Blue Water Vaccines" or "the Company"), a biopharmaceutical company developing transformational vaccines to address significant global health challenges, reported that it has entered into definitive agreements with several institutional investors for the purchase of 1,180,812 shares of common stock (or common stock equivalents in lieu thereof) in a private placement priced at-the-market under Nasdaq rules (Press release, Onconetix, APR 13, 2022, View Source [SID1234641107]). The Company will also issue to the investors unregistered preferred investment options (the "investment options") to purchase up to an aggregate of 1,180,812 shares of common stock. The purchase price for one share of common stock (or common stock equivalent) and one investment option to purchase one share of common stock is $6.775. The investment options have an exercise price of $6.65 per share, will be exercisable immediately upon issuance, and will have a term equal to four years following the issuance date.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The gross proceeds from the private placement are expected to be approximately $8.0 million, before deducting placement agent fees and other offering expenses. The Company intends to use the net proceeds from the private placement for the research and development of its pipeline as well as for working capital and other general corporate purposes. The private placement is expected to close on or about April 18, 2022, subject to the satisfaction of customary closing conditions.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and the securities have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws. Accordingly, the securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. Pursuant to a registration rights agreement with investors, BWV has agreed to file a resale registration statement covering the securities described above within twenty days.

This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On 13 April 2022 Oxford BioTherapeutics (OBT), a clinical stage oncology company with a pipeline of immuno-oncology and Antibody Drug Conjugate (ADC)-based therapies, reported that Boehringer Ingelheim has exercised its option to receive exclusive rights to a third novel oncology target from an ongoing discovery collaboration (Press release, Oxford BioTherapeutics, APR 13, 2022, View Source [SID1234626469]). The target was discovered using OBTs proprietary OGAP drug discovery platform, which incorporates one of the world’s largest proteomic databases, integrating clinical, experimental and expression data. The option triggers another milestone payment from Boehringer Ingelheim to OBT.

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Under the terms of the agreement, Boehringer Ingelheim will be responsible for the future development and commercialization of antibody products to the target. OBT will receive development and regulatory milestone payments as well as royalties on any future product sales. This represents the third asset to be optioned under the longstanding partnership between OBT and Boehringer Ingelheim, with the first two programs already in clinical development.

"Boehringer Ingelheim’s decision to exercise its option on a third oncology drug target, developed under our collaboration, is further validation of our proprietary OGAP drug discovery platform," said OBT’s Chief Executive Officer, Dr. Christian Rohlff. "Selecting the right target is fundamental for the successful development of a first-in-class antibody drug product. OBT’s platforms are designed to discover and engineer antibody constructs to novel therapeutic targets – including bi-specific, Chimeric Antigen Receptor T Cell (CAR-T), other T- cell and NK cell-mediated cytotoxicity (ADCC) and ADC therapeutics – to best address difficult-to-treat cancers. We believe that the advancement of the first two Boehringer Ingelheim compounds, directed to oncology targets identified by us, into the clinic, further validates our approach."

In addition to the programs in the partnership with Boehringer Ingelheim, OBT’s own clinical assets have also been enabled through the OGAP discovery platform. A major differentiator between OBT’s discovery platform and other approaches is the retention of the link between individual patient samples through to the design of therapeutic antibodies and diagnostic patient selection tools, which increases the overall success rate of these novel compounds transitioning into clinical development.