On December 22, 2022 SNIPR Biome ApS ("SNIPR" or "the Company"), the company pioneering CRISPR-based microbial gene therapy, reported the grant of a new patent by the US Patent and Trademark Office (USPTO) (Press release, SNIPR Biome, DEC 22, 2022, View Source [SID1234625559]). This patent represents a further addition to the Company’s extensive intellectual property portfolio, comprising more than 60 granted patents worldwide covering SNIPR’s technology platform, which enables editing of prokaryotes using CRISPR/Cas.

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The USPTO has granted patent number US11,517,582, which covers the use of CRISPR/Cas systems to achieve selective killing of bacteria by at least 1000-fold in situations where bacteria are growing in a mixed population. This patent covers the use of any type of CRISPR/Cas.

In natural environments, such as in gut microbiomes, bacteria are found growing in mixed populations and it has been difficult to selectively target individual bacterial species with conventional broad-spectrum antibiotics. SNIPR’s CRISPR technology provides a highly selective way to target individual bacterial strains for killing which can be useful for the prevention and treatment of any indication where a specific bacterial strain is the underlying cause of a disease, such as an infection.

SNIPR has also received an allowance by the USPTO indicating that it will shortly grant a US patent broadly covering methods of using CRISPR lytic phage, by use of any CRISPR/Cas system and for any application (US 15/817,144). This expands the Company’s portfolio protecting CRISPR lytic phage. In August 2022, SNIPR was awarded patent number US11,400,110 which covers lytic phage armed with CRISPR gene editing systems. CRISPR and phage lysis of target bacteria is a potent combination for therapeutics, with potential for broad application, including the targeting of any bacteria for any medical use.

Dr Christian Grøndahl, Co-founder and CEO of SNIPR Biome, commented: "This expansion of our patent estate strengthens our already extensive intellectual property portfolio covering the use of CRISPR/Cas to edit prokaryotes. SNIPR Biome has exclusive, worldwide rights to this patent estate for medical applications, which supports our pipeline and lead program SNIPR001, a CRISPR-armed bacteriophage cocktail that targets the prevention of antibiotic-resistant E. coli infections in hematological cancer patients. We will continue our pioneering work in this field as we advance our mission of developing CRISPR-based medicines for the benefit of patients suffering from life-threatening diseases."

Earlier this year, the Company made its IP available for academic and non-profit research use without a written license. Parties interested in licensing SNIPR’s intellectual property should contact the Company at [email protected].

Acknowledgement and disclaimer:

Research reported in this communication is supported by CARB-X. CARB-X’s funding for this project is sponsored by the Other Transaction Agreement Number 75A50122C00028 from ASPR/BARDA and by an award from Wellcome. The content is solely the responsibility of the authors and does not necessarily represent the official views of CARB-X or any of its funders.

About CARB-X

Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) is a global non-profit partnership dedicated to accelerating early development antibacterial R&D to address the rising global threat of drug-resistant bacteria. CARB-X is led by Boston University and funding is provided by the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response (ASPR) in the U.S. Department of Health and Human Services, Wellcome, a global charity working to improve health worldwide, Germany’s Federal Ministry of Education and Research (BMBF), the U.K. Department of Health and Social Care’s Global Antimicrobial Resistance Innovation Fund (GAMRIF), the Bill & Melinda Gates Foundation, and with in-kind support from National Institute of Allergy and Infectious Diseases (NIAID), part of the U.S. National Institutes of Health (NIH). CARB-X supports the world’s largest and most innovative pipeline of preclinical and early-stage development products against antibiotic-resistant infections. CARB-X is headquartered at Boston University School of Law. For more information, view a fact sheet on CARB-X’s first five years and the 2020-2021 annual report. Visit carb-x.org and follow CARB-X on Twitter @CARB_X.

On December 22, 2022 Menarini Silicon Biosystems, a pioneer of liquid biopsy and single cell technologies, reported promising long-term results of the STIC trial, on OS (overall survival) in patients with ER+/HER2- MBC (Press release, Menarini Silicon Biosystems, DEC 22, 2022, View Source [SID1234625558]). This robust (n=755), randomized, multicenter phase III study aimed to compare the efficacy of a clinician- vs CTC-driven treatment choice. After meeting its primary endpoint on PFS (progression free survival) in 2018[1], follow-up data presented orally by Professor Francois-Clément Bidard at the 2022 San Antonio Breast Cancer Symposium (SABCS) on December 8th, demonstrates that a single assessment of CELLSEARCH CTC count before the start of treatment, may lead to increased OS in a metastatic setting2.

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According to FC Bidard, MD, PhD, Professor of Medical Oncology & Coordinator of Breast Cancer research at the Institut Curie hospitals, "we were particularly impressed by the subgroup of patients with a low clinical risk profile and CTC count ≥ 5, for whom physicians, in line with guidelines, selected endocrine therapy (ET). When treated with chemotherapy (ChT) instead, these patients had a superior median OS of 16 months". The trial further showed that, when escalated to ChT, these patients also experienced a 47% reduction in their risk of death. "This is the first ever trial to demonstrate the clinical utility of CTC count in MBC. It paves the way to integrating CTC count into treatment decision algorithms for patients who previously received CDK4/6 inhibitors as first line or adjuvant therapy, and for whom the decision between either a further line of ET or ChT is difficult, heterogeneous and controversial when based only on clinicians’ judgment" – he added.

Breast cancer is the most commonly diagnosed malignancy in women worldwide. Among various subtypes, HR+/HER2- is the most prevalent3 and improvement of long-term survival in MBC still remains a challenge.

CELLSEARCH is the first and only clinically validated blood test cleared by the U.S. Food & Drug Administration (FDA) for detecting and counting CTCs in metastatic breast cancer when used in conjunction with other clinical monitoring methods. Because it can capture and enumerate CTCs from just 7.5 ml of peripheral blood, this assay represents a minimally invasive method, compared to more burdensome tumor biopsies, for informing treatment strategies. "We are thrilled by the positive results of our study on the more direct clinical benefit endpoint of OS" said Fabio Piazzalunga, President and CEO of Menarini Silicon Biosystems. "The fact that our CELLSEARCH CTC assay* had such a significant impact on this measure is quite promising. We believe this trial will open new doors to support treatment decisions that optimize patient care for other life-threatening tumor types, considering our desire to be the indisputable leader in non-invasive cell-based applications for easier, faster, and more precise diagnostic and therapeutic approaches."

About CELLSEARCH

CELLSEARCH is the first and only clinically validated blood test cleared by the U.S. Food & Drug Administration (FDA) for detecting and counting CTCs to aid physicians in managing patients with metastatic breast, prostate, and colorectal cancers when used in conjunction with other clinical methods of monitoring. The test is also approved by the China National Medical Products Administration (NMPA) for use in monitoring patients with Metastatic Breast Cancer. The CELLSEARCH System is the most extensively studied CTC technology, with research published in more than 800 peer-reviewed publications.

CELLSEARCH Circulating Tumor Cell Test is not cleared or approved for guiding treatment decisions. For more information on the full intended use and limitations of CELLSEARCH system, please refer to the Instructions for Use at View Source

CELLSEARCH Circulating Tumor Cell Test is available as a laboratory developed test (LDT) in MSB’s CLIA/CAP/ ISO 15189 accredited laboratory in Huntingdon Valley, Pa.

On December 22, 2022 IASO Biotherapeutics (IASO Bio), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products,, reported that the Investigational New Drug (IND) application for its in-house developed BCMA CAR-T CT103A (equecabtagene autoleucel) has been approved by the U.S. Food and Drug Administration (FDA) for use in U.S. clinical trials for relapsed/refractory multiple myeloma (R/R MM) (Press release, IASO Biotherapeutics, DEC 22, 2022, View Source [SID1234625557]).

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Multiple myeloma is a deadly blood cancer that often infiltrates the bone marrow causing anemia, kidney failure, immune problems, and bone fractures. For multiple myeloma patients, common first-line drug treatments include proteasome inhibitors, immunomodulatory drugs, and alkylating agents. While treatment may result in remission, most patients will inevitably enter the relapsed or refractory stage as there’s currently no cure. As a result, there is a significant unmet need for patients with relapsed/refractory multiple myeloma. In the United States, MM accounts for nearly 2% of new cancer cases, and more than 2% of all cancer-related deaths.

According to Frost & Sullivan, the number of new MM cases in the United States rose from 30,300 in 2016 to 32,300 in 2020 and is expected to increase to 37,800 by 2025. The total number of patients diagnosed with MM in the United States increased from 132,200 in 2016 to 144,900 in 2020 and is expected to rise to 162,300 by 2025.

CT103A, a CAR-T cell therapy targeting the B-cell maturation antigen (BCMA), has a chimeric antigen receptor (CAR) structure containing fully human single-chain variable fragments (scFvs), allowing it to bypass potential anti-CAR immunogenicity of the host while retaining anti-tumor activity. Results from a clinical phase I/II (NCT05066646) study in China showed excellent safety and efficacy of CT103A.

The data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Research Conference 2022 showed that as of January 21, 2022, 79 subjects have received CT103A infusion at the recommended Phase II dose (RP2D) of 1.0×106 CAR-T cells/kg. The median follow-up time for the 79 subjects was 9 months (1.2, 19.6), and the objective response rate (ORR) was 94.9%. The median time to response (mTTR) was only 16 days. 73 (92.4%) subjects achieved at least once negative minimal residual disease (MRD) status after cell infusion. All subjects who achieved complete response (CR) or better achieved MRD-negative. Subjects who had previously received CAR-T therapy continued to benefit from CT103A infusion. Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) that occurred after cell infusion were only grade 1~2. No subject developed grade ≥3 CRS or ICANS, and all CRS and ICANS resolved after supportive treatment.

CT103A’s regulatory milestones:

In February 2021, CT103A was granted the "breakthrough therapy designation (BTD)" by China’s National Medical Products Administration (NMPA) for the treatment of relapsed/refractory multiple myeloma.
In February 2022, CT103A was granted the "orphan drug designation (ODD)" by the FDA for the treatment of multiple myeloma.
In June 2022, CT103A was accepted by the NMPA for the NDA application for the treatment of relapsed/refractory multiple myeloma and included in the priority review.
In August 2022, a new IND was approved by NMPA for CT103A with an expanded indication of AQP4-IgG-positive neuromyelitis optica spectrum disorder (NMOSD).
Dr. Wen (Maxwell) Wang, Chief Executive Officer of IASO Bio, said, "The IND approval of CT103A in the U.S. is an important milestone of IASO Bio and a new starting point outside China. IASO Bio will accelerate its overseas clinical trials and the development and implementation of cellular immunotherapy drugs to benefit more patients globally."

About CT103A

Equecabtagene autoleucel (CT103A) is a BCMA chimeric antigen receptor autologous T cell injection, a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3ζ activation domains. Based on strict selection and screening, utilizing a proprietary in-house optimization platform, and integrated in-house manufacturing process improvement, the construct of the BCMA CAR-T is potent and equecabtagene autoleucel shows prolonged persistency in patients. The NMPA accepted the New Drug Application for equecabtagene autoleucel for the treatment of relapsed/refractory multiple myeloma (R/R MM). Equecabtagene autoleucel also received Breakthrough Therapy Designation by the NMPA in February 2021 and Orphan Drug Designation (ODD) by the U.S. FDA in February 2022. In addition to multiple myeloma, the NMPA has received IND application of equecabtagene autoleucel for the new expanded indication of Neuromyelitis Optica Spectrum Disorder (NMOSD).

On December 22, 2022 Tetra Bio-Pharma Inc. ("Tetra" or the "Company") (TSX: TBP) (OTCQB: TBPMF) (FRA: JAM1) a leader in cannabinoid-derived drug discovery and development reported that it is providing its shareholders with another regulatory update on the REDUVO New Drug Submission (NDS) (Press release, Tetra Bio Pharma, DEC 22, 2022, View Source [SID1234625556]). The last update was provided on November 18, 2022.

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On December 21, 2022, the Company received a second Clarification Request (Clarifax) from Health Canada regarding the product’s Risk Management Plan, which is not atypical for a controlled substance drug. The Company will be submitting the response to Health Canada within the allowed timeframe. The Company cannot comment on Health Canada’s response timelines. Questions/feedback from Health Canada are received as the review of the dossier progresses through different review streams.

About REDUVO

REDUVO is a soft gel capsule used to treat chemotherapy-induced nausea and vomiting (CINV). It is also used to treat weight loss and severe nausea in people living with HIV infection. The active pharmaceutical ingredient in REDUVO is dronabinol, also known as THC, a synthetic form of the active natural substance in cannabis. REDUVO 5-year cumulative gross sales expected to reach $121M.

On December 22, 2022 Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products reported its business and financial results for the fiscal full year ended September 30, 2022 (Press release, Citius Pharmaceuticals, DEC 22, 2022, View Source [SID1234625555]).

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Fiscal Full Year 2022 Business Highlights and Subsequent Developments

Completed Pivotal Phase 3 trial of I/ONTAK (E7777) and submitted biologics license application (BLA) to the U.S. Food and Drug Administration (FDA);
FDA confirmed Prescription Drug User Fee Act (PDUFA) target action date of July 28, 2023;
Advanced Mino-Lok Phase 3 trial:
Expanded trial to additional sites in India for an anticipated total of 35 clinical trial locations globally,
Enrolled 169 patients to date, exceeding recruitment goal of 144 patients,
Observed 72 of 92 required catheter failure events, with 17 patients in active treatment or pending study completion data review (which may contribute additional failure events);
Initiated Phase 2b trial of Halo-Lido for the treatment of hemorrhoids in April 2022; patient enrollment ongoing with data readout expected 2H 2023;
Initiated clinical collaboration with the University of Pittsburgh to evaluate regulatory T-cell (T-reg) depletion with I/ONTAK (E7777) in combination with pembrolizumab in recurrent or metastatic solid cancer tumors in a Phase 1 investigator-initiated trial, with first patient enrolled in November 2022; and,
Approved for $3.6 million in non-dilutive capital through the New Jersey Economic Development Program to support ongoing research and development efforts.
Financial Highlights

Cash and cash equivalents of $41.7 million as of September 30, 2022;
R&D expenses were $17.7 million for the full year ended September 30, 2022, compared to $12.2 million for the full year ended September 30, 2021;
G&A expenses were $11.8 million for the full year ended September 30, 2022, compared to $9.8 million for the full year ended September 30, 2021;
Stock-based compensation expense was $3.9 million for the full year ended September 30, 2022, compared to $1.5 million for the full year ended September 30, 2021; and,
Net loss was $33.6 million, or ($0.23) per share for the full year ended September 30, 2022 compared to a net loss of $23.1 million, or ($0.23) per share for the full year ended September 30, 2021.
"In 2022, we focused on execution across our key development programs: I/ONTAK, Mino-Lok and Halo-Lido. These efforts, combined with a prudent use of funds, enabled us to meaningfully advance our pipeline. We believe we have sufficient runway through December 2023 to realize additional value-creating milestones, including a potential FDA approval and two anticipated trial completions in the coming calendar year," stated Leonard Mazur, Chairman and CEO of Citius.

"Our Phase 3 Mino-Lok trial is now significantly closer to completion. While we expected to achieve 92 catheter failure events with 144 patients by the end of 2022, the trial’s observed catheter failure event rate has proven to be lower than anticipated. Consequently, we must continue recruiting patients. By successfully re-engaging with our U.S. trial sites as they recovered from the impact of Covid, we were able to drive patient recruitment. We have now exceeded our targeted enrollment and have achieved 72 of the required catheter failure events, with additional patients under review. To augment our recruitment efforts and continue the positive momentum in enrollment, we expanded the Mino-Lok trial to include sites in India. Once all new trial sites are fully activated, we will have nearly doubled our clinical site footprint. With these additional sites helping to drive incremental enrollment, we anticipate that the 92-event threshold required to complete the trial is achievable in the coming months," added Mazur.

"During the year, we also completed a Phase 3 trial and submitted a BLA for I/ONTAK, an oncology asset we in-licensed just over a year ago. Upon further discussion with the FDA, the PDUFA target date has been set for July 28, 2023. We remain committed to establishing a robust commercial infrastructure to support I/ONTAK’s successful product launch, if approved. In the second half of 2022, we also extended our support for a Phase 1 investigator-initiated study of I/ONTAK in combination with pembrolizumab (Keytruda1) to treat patients with recurrent or metastatic solid tumors. This study has begun recruiting patients and is the second investigator-initiated trial to explore I/ONTAK’s potential as a combination therapy in much larger immuno-oncology markets. We continue to believe I/ONTAK’s value extends beyond a potential initial indication in persistent or recurrent cutaneous T-cell lymphoma. Earlier in the year, we announced our intention to spin off I/ONTAK. Given broader market conditions, we continue to evaluate opportunities to further unlock this asset’s value," continued Mazur.

"In addition to advancing our Phase 3 trials, we initiated a Phase 2b trial for Halo-Lido, our prescription strength topical formulation for hemorrhoids. The trial began enrolling patients with symptomatic Grade II or III hemorrhoids in the second quarter of 2022. Recent recruitment has accelerated and we expect complete trial data available in the second half of 2023," added Mazur.

"As financial stewards, we continuously evaluate the optimal capital structure for the company. We believe our anticipated catalysts, along with a healthy cash position, provide us with several strategic and financial options with which to continue advancing our pipeline. This may include the previously announced potential spinoff of I/ONTAK into a standalone oncology company, pending market conditions, and other standalone financing alternatives available to us. We are encouraged by the multiple value-driving catalysts anticipated in calendar 2023, including a potential drug approval and two trial completions, and look forward to extending our positive momentum in the months ahead," concluded Mazur.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA

FULL YEAR 2022 FINANCIAL RESULTS:

Liquidity

As of September 30, 2022, the Company had $41.7 million in cash and cash equivalents.

As of September 30, 2022, the Company had 146,211,130 common shares outstanding.

The Company estimates that its available cash resources will be sufficient to fund its operations through December 2023.

Research and Development (R&D) Expenses

R&D expenses were $17.7 million for the full year ended September 30, 2022, compared to $12.2 million for the full year ended September 30, 2021. The increase of $5.5 million is primarily associated with the completion of the I/ONTAK (E7777) Phase 3 trial and the preparation and submission of the related Biologics License Application to the FDA, incremental Mino-Lok Phase 3 trial costs related to the addition of a global clinical research organization, Biorasi, and the opening of international sites in India, as well as costs associated with the initiation of the Halo-Lido Phase 2 study. The increase was offset primarily by a one-time $5 million license fee paid to Novellus in the year ended September 30, 2021, which did not recur.

We expect that research and development expenses will continue to increase in fiscal 2023 as we continue to focus on the anticipated commercialization of E7777, our Phase 3 trial for Mino-Lok, our Phase 2b trial for Halo-Lido, and accelerate our research and development efforts related to Mino-Wrap and ARDS.

General and Administrative (G&A) Expenses

G&A expenses were $11.8 million for the full year ended September 30, 2022, compared to $9.8 million for the full year ended September 30, 2021. The increase was primarily due to additional compensation costs for new employees and investor relations expenses. General and administrative expenses consist primarily of compensation costs, consulting fees for our financing activities and corporate development services, and investor relations expenses.

Stock-based Compensation Expense

For the full year ended September 30, 2022, stock-based compensation expense was $3.9 million as compared to $1.5 million for the prior year. The increase reflects expenses related to new grants made by Citius and the NoveCite stock option plan. In fiscal year 2022, we granted options to our new employees and additional options to other employees, directors, and consultants. Stock-based compensation expense includes options granted to directors, employees, and consultants.

At September 30, 2022, unrecognized total compensation cost related to unvested options for Citius common stock of $5.3 million is expected to be recognized over a weighted average period of 1.9 years and unrecognized total compensation cost related to unvested options for NoveCite common stock of $0.2 million is expected to be recognized over a weighted average period of 1.5 years

Net loss

Net loss was $33.6 million, or ($0.23) per share for the year ended September 30, 2022, compared to a net loss of $23.1 million, or ($0.23) per share for the year ended September 30, 2021. The increase in net loss is primarily due to the $5.4 million increase in our research and development expenses, a $1.9 million increase in general and administrative expenses, and a $2.4 million increase in stock-based compensation expense.