On July 2, 2024 Full-Life Technologies (Full-Life), a fully integrated global radiotherapeutics company, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for 225Ac-FL-020, the company’s lead radiopharmaceutical, which targets prostate-specific membrane antigen (PSMA), for the treatment of metastatic castration-resistant prostate cancer (mCRPC) (Press release, Full-Life Technologies, JUL 2, 2024, View Source [SID1234644657]).

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The Fast Track program is designed to facilitate the development and regulatory review of novel potential therapies intended to treat serious conditions and fill an unmet need. This designation highlights the potential of 225Ac-FL-020 to provide a novel therapeutic option for patients with mCRPC, a condition for which there are currently limited effective treatments.

"The FDA Fast Track Designation for 225Ac-FL-020 underscores the critical need for innovative and effective treatments for mCRPC", said Steffen Heeger, M.D., M.Sc., Chief Medical Officer of Full-Life. "This designation will enable us to collaborate more closely with the FDA throughout the development process, potentially accelerating the availability of 225Ac-FL-020 to patients."

225Ac-FL-020 employs targeted alpha-radiotherapy designed to selectively attack cancer cells, reducing the damage to healthy tissues. In preclinical models, radiolabeled FL-020 displayed a very promising in vivo biodistribution profile, with high and sustained tumor uptake and fast systemic clearance. 225Ac-FL-020 exhibited robust anti-tumor activity in LNCaP xenograft mice, with a favorable safety profile. The Phase I clinical trial will evaluate the safety, tolerability, and anti-tumor activity of 225Ac-FL-020. In May 2024, Full-Life received clearance of its Investigational New Drug (IND) Application from the FDA for clinical trials of 225Ac-FL-020.

About 225Ac-FL-020
225Ac-FL-020 is a novel, potential best-in-class, next-generation PSMA-targeting radionuclide drug conjugate (RDC) that entered global Ph1 clinical studies in 2024. Its targeting vector, FL-020, was discovered using Full-Life’s proprietary UniRDC platform, which enables significant improvement of drug uptake in the tumor while maintaining fast systemic clearance. In pre-clinical models, 225Ac-FL-020 has demonstrated potent anti-tumor activity and a favorable safety profile.

On July 2, 2024 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage biopharmaceutical company, reported that members of the Company’s management team will be hosting one-on-one meetings with institutional investors at the Leerink Partners Therapeutics Forum: I&I and Metabolism, being held on July 9 and 10, 2024 in Boston, MA (Press release, Altimmune, JUL 2, 2024, View Source [SID1234644655]). The event will feature analyst-hosted panel discussions with leading physicians in a range of autoimmune and metabolic indications, as well as meetings between participating companies and investors.

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On July 2, 2024 Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the "Company"), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, reported that it has entered into a securities purchase agreement for a private investment in public equity ("PIPE") financing that is expected to result in gross proceeds of approximately $130.0 million to the Company, before deducting placement agent fees and other offering expenses (Filing, 8-K, Selecta Biosciences, JUL 2, 2024, View Source [SID1234644653]). The PIPE financing is expected to close on or about July 3, 2024, subject to satisfaction of customary closing conditions.

The PIPE financing included participation from both new and existing investors, including HBM Healthcare Investments (Cayman) Ltd., Invus, Schooner Capital, Surveyor Capital (a Citadel company), Timothy A. Springer, Ph.D., a leading mutual fund manager, and other institutional investors.

Pursuant to the terms of the securities purchase agreement, the Company is selling an aggregate of 3,563,247 shares of its common stock ("Common Stock"), and 2,937,903 shares of its Series B Non-Voting Convertible Preferred Stock ("Series B Preferred Stock"), each at a purchase price of $20.00 per share. Each share of Series B Preferred Stock is convertible into one share of Common Stock, subject to Cartesian stockholder approval thereof and certain beneficial ownership limitations set by the purchasers of the Series B Preferred Stock.

The Company intends to use the net proceeds from the PIPE financing, together with the Company’s existing cash, cash equivalents, and marketable securities, to fund its pipeline programs, and for general corporate purposes and working capital.

Leerink Partners and TD Cowen are acting as lead placement agents for the PIPE financing and Needham & Company is acting as placement agent for the PIPE financing.

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, and 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. Concurrently with the execution of the securities purchase agreement, the Company and the investors entered into a registration rights agreement pursuant to which the Company has agreed to file a registration statement with the Securities and Exchange Commission (the "SEC") registering the resale of the shares of Common Stock underlying the Series B Preferred Stock and the shares of Common Stock sold in the PIPE financing.

This press release shall not constitute an offer to sell or a 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.

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On July 2, 2024 Biodexa Pharmaceuticals PLC ("Biodexa" or the "Company") (Nasdaq: BDRX), an acquisition-focused clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, reported data from a Phase 1 study of MTX110 in Diffuse Midline Glioma ("DMG") f/k/a Diffuse Intrinsic Pontine Glioma, or DIPG, an orphan pediatric brain cancer were presented over the weekend at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) in Philadelphia, PA (Press release, Midatech Pharma, JUL 2, 2024, View Source [SID1234644652]).

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Results of the Phase 1 study
Overall, the treatment was well tolerated by patients. There was one Grade 4 adverse event assessed by the investigators as unrelated to the drug but related to the infusion and tumor anatomy. Most other adverse events were related to infusion and were deemed Grade 2 to 3.

Although the study was not powered to reliably demonstrate efficacy, median progression free survival (PFS) was 10 months (range 8 to 20 months) and overall survival (OS) of patients in the study was 16.5 months (range 12 to 35 months). This compares favourably with median OS in a cohort of 316 cases of 10.0 months (Jansen et al, 2015. Neuro-Oncology 17(1):160-166).

Design of the Phase 1 study
The open label investigator-initiated study was conducted by Columbia University Irving Medical Center in patients newly diagnosed with DMG. Patients were administered MTX110 via convection enhanced delivery ("CED") using a subcutaneous pump connected to a catheter directly implanted into the pons in a 3+3 dose-escalating design (NCT 04264143).

As this was the first ever study of repeated infusions to the pons via an implanted CED catheter, the primary endpoint of the study was to evaluate the safety and maximum tolerated dose with secondary endpoints of Progression Free Survival and Overall Survival.

The number of infusions was limited to two, each of 48 hours, seven days apart. Nine patients were treated in the study (30 M group, n=3; 60 M group, n=4; 90 M group (optimal dose), n=2). Although the study was not powered to reliably demonstrate efficacy, median overall survival (OS) of patients in the study was 16.5 months. This compares favourably with median survival rate in a cohort of 316 cases of 10.0 months (Jansen et al, 2015. Neuro-Oncology 17(1):160-166).

MTX110 in DMG
In October 2020, the Company announced headline results from a Phase I study at the University of California, San Francisco ("UCSF") in patients with DMG (the "UCSF study" NCT03566199).

The primary endpoint of the study was to determine the dosage regimen to be used in a proposed Phase II study of the safety and efficacy of MTX110 in patients with DIPG. Preliminary high-level data from the UCSF study supports a dose of between 60µM and 90µM of MTX110, depending upon patient tolerance over the course of 12 infusions in Phase II.

In total, seven patients were recruited into the UCSF study. Patients were newly diagnosed with DMG and received focal external beam radiation therapy four to 14 weeks before commencement of MTX110 treatment. MTX110 was administered directly into the tumour via a micro-catheter using CED with gadolinium-enhanced intra-operative MRI to guide and track drug distribution to the tumour. Patients could receive up to 12 cycles of treatment every four to eight weeks. The dose was escalated between and within patients as tolerated initially by increasing the infusion volume at a concentration of 30µM MTX110 and then with higher drug concentrations of 60µM and 90µM as the sixth and seventh dose increments, respectively.

Median overall survival based on Kaplan Meier analysis was 26.06 months. Survival was not an endpoint of the UCSF study nor was the study powered for statistical significance.

About MTX110
MTX110 is a water-soluble form of panobinostat free base, achieved through complexation with hydroxypropyl-β-cyclodextrin (HPBCD), that enables CED at potentially chemotherapeutic doses directly to the site of the tumour. Panobinostat is a hydroxamic acid and acts as a non-selective histone deacetylase inhibitor (pan-HDAC inhibitor). The currently available oral formulation of panobinostat lactate (Farydak) is not suitable for treatment of brain cancers owing to poor blood-brain barrier penetration and inadequate brain drug concentrations. Based on favourable translational science data, MTX110 is being evaluated clinically as a treatment for DMG (NCT03566199, NCT04264143) and recurrent medulloblastoma (NCT04315064), and recurrent glioblastoma (NCT 05324501). MTX110 is delivered directly into and around the patient’s tumor via a catheter system (e.g. CED or fourth ventricle infusions) to bypass the blood-brain barrier. This technique exposes the tumor to very high drug concentrations while simultaneously minimising systemic drug levels and the potential for toxicity and other side effects. Panobinostat has demonstrated high potency against DMG tumor cells in in vitro and in vivo models, and in a key study it was the most promising of 83 anticancer agents tested in 14 patient-derived DMG cell lines (Grasso et al, 2015. Nature Medicine 21(6), 555-559).

On July 2, 2024 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotech company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to improving survival outcomes for patients with cancer, reported that the DSMB completed its first safety assessment of the Phase 2b trial PHOEBUS, the largest one to date for a microbiome therapy in oncology, and recommended continuation of the trial without modification (Press release, MaaT Pharma, JUL 2, 2024, View Source [SID1234644651]). The trial is an international, multi-center, randomized, double-blind, testing MaaT033, an oral freeze-dried formulation against placebo, set to be conducted in up to 56 clinical investigation sites and is expected to enroll 387 patients (NCT05762211).

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The DSMB, composed of 5 independent experts, reviewed safety data on the first 20 patients (cutoff date as of April 30th, 2024) and concluded that safety was acceptable and well tolerated. Since its first clinical entry in 2020, MaaT033, a drug candidate produced by combining the microbiota from multiple donors using a patented "pooling" process, has continuously displayed a good safety profile.

"The positive outcome of this first DSMB review of the PHOEBUS study significantly builds on the favorable safety and tolerability profile exhibited by MaaT033," said Gianfranco Pittari, M.D., PhD, Chief Medical Officer of MaaT Pharma. "We are very enthusiastic about MaaT033’s potential to ensure an optimal microbiome ecosystem and enhance clinical outcomes in patients undergoing allogeneic stem cell transplantation."

"MaaT033 is designed for ambulatory use and chronic treatment; it will address a substantial market of approximately 11,000 patients annually, and with its freeze-dried capsule formulation it will significantly drive our growth," highlighted Hervé Affagard, Chief Executive Officer and co-founder of MaaT Pharma. "With the GMP manufacturing facility at full capacity, we will produce up to 1,300,000 capsules annually, meeting patient demand and supporting our innovative microbiome therapies".