On May 16, 2022 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that it will present its first research data on the development of a novel universal CAR T-cell with immune-evasive properties using TALEN-gene editing, at the American Society of Cell and Gene Therapy Annual Meeting (ASGCT) (Free ASGCT Whitepaper) being held on May 16-19, 2022 (Press release, Cellectis, MAY 16, 2022, View Source [SID1234614584]). This novel immune-evasive CAR T-cell scaffold evades NK (Natural Killer) cell and alloresponsive T-cell attacks and imparts efficient antitumor activity in vitro and in vivo.

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Cellectis’ novel immune-evasive CAR T-cell (ΔTRACCARΔB2MHLAE), was developed using a combination of TALEN-mediated gene editing and adeno-associated virus (AAV) dependent gene insertion. ΔTRACCARΔB2MHLAE is devoid of TCRαβ and human leukocyte antigen (HLA) Class I expression and endowed with an engineered surface-exposed HLA-E. These three features could enable CAR T-cells to prevent graft versus host (GvH) reaction and evade the cytolytic activities from alloresponsive T-cells and NK cells.

"Universal CAR T-cell therapies are poised to revolutionize cancer treatment and to improve patient outcomes. Realizing these advantages in an allogeneic setting requires universal CAR T cells that can kill target tumor cells, avoid depletion by the host immune system, and proliferate without attacking host tissues. Cellectis’ research suggested that ΔTRACCARΔB2MHLAE T-cells evade NK cell and alloresponsive T-cell attacks and showed prolonged antitumor activity in the presence of cytotoxic levels of NK cells. This new cellular scaffold could enable the broad use of universal CAR T-cells in allogeneic settings and holds great promise for clinical applications," said Julien Valton, Ph.D., Vice President Gene Therapy at Cellectis.

Research data showed that:

ΔTRACCARΔB2MHLAE overcame alloresponsive T-cell and NK cells attacks.

The immune-evasive property of ΔTRACCARΔB2MHLAE was similar toward NK cells from healthy donors, acute myeloid leukemia (AML) patients and acute lymphocytic leukemia (ALL) patients.

ΔTRACCARΔB2MHLAE T-cells exhibit prolonged antitumor activity in the presence of cytotoxic levels of NK cells.
Title: Endowing Universal CAR T-cell with Immune-Evasive Properties Using TALEN-Gene Editing

Session Date: May 16, 2022
Presentation Time: 3:45pm – 4:00pm ET
Location: Walter E. Washington Convention Center
Session title: Cell-Based Cancer Immunotherapies I
Room: 207
Final abstract number: 99

The research data will be presented today in an oral presentation. The abstract can be accessed on the ASGCT (Free ASGCT Whitepaper) website, and the oral presentation will be posted on Cellectis’ website during the conference.

On May 16, 2022 Vyant Bio, Inc. ("Vyant Bio", "Company") (Nasdaq: VYNT) is an innovative biotechnology company reinventing drug discovery for complex neurodevelopmental and neurodegenerative disorders (Press release, Cancer Genetics, MAY 16, 2022, View Source [SID1234614583]). The Company’s central nervous system ("CNS") drug discovery platform combines human-derived organoid models of brain disease, scaled biology, and machine learning. Today, Vyant Bio expects to file its Form 10-Q for the First Quarter 2022 with the Securities and Exchange Commission and will report its First Quarter 2022 highlights and business updates this afternoon in a conference call and webcast scheduled for 4:30 pm ET.

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"During the first quarter of 2022, we progressed our scientific work by using our proprietary drug discovery platform centered on human iPSC-derived neurospheroids to unveil novel targets and potentially disease-modifying therapeutics to treat patients suffering from severe neurodevelopmental and neurodegenerative diseases. We are validating important data that we believe will demonstrate our ability to de-risk the selection of therapeutics that can rescue a disease phenotype. We believe current preclinical models are not sufficiently predictive, and many current therapies for CNS disorders only address the symptoms and do not reverse the effects of neurological diseases. Our focus during the first 90 days of 2022 was to progress our platform validation efforts to advance our CNS programs that are designed to identify disease-modifying therapeutics, consistent with our plans and prior disclosures to our shareholders," stated Jay Roberts, the Company’s Chief Executive Officer.

"As we highlighted in our last earnings conference call, we believe our focus on complex neurodevelopmental and neurodegenerative disorders addresses significant unmet medical needs, and will lead to the identification of important, disease modifying therapies for major causes of death and disability worldwide. Our Rett patient-derived neural spheroids show a robust and reproducible disease-specific phenotype that can be quantified in an unbiased manner across dozens of endpoints. By phenotypic screening of our Rett neural spheroids, we identified VYNT-0126 which has consistently shown a dose-dependent unique rescue of the RTT functional phenotype with a differentiated mechanism of action from other Rett syndrome clinical candidates. Our ongoing work on CDKL5 and familial Parkinson’s disease has further established that our discovery platforms represent a new, unique robust model for human-first CNS drug discovery" stated Dr. Robert Fremeau, Chief Scientific Officer of Vyant Bio.

FIRST QUARTER 2022 FINANCIAL RESULTS

Cash and cash equivalents totaled $16.4 million as of March 31, 2022. The Company implemented two new vehicles to facilitate the raising of additional equity capital at the Company’s option with the finalization of the Lincoln Park Equity line of credit allowing access to raise up to $15 million, as well as signing a $14.5 million ATM with Canaccord Genuity. The Company’s current cash balances, future proceeds from the sale of vivoPharm and future proceeds from the equity line of credit and ATM are expected to fund operations well into 2023.

During the first quarter of 2022, the Company continued the process of divesting the vivoPharm business which is expected to complete in 2022. Therefore, the vivoPharm business is classified as a "held-for-sale" asset, and its financial information as "discontinuing operations".

The Company’s loss from continuing operations aggregated $4.4 million in the first quarter of 2022 and included non-cash depreciation and amortization as well as stock-based compensation expenses of $98 thousand and $278 thousand, respectively, and one-time severance charges of $437 thousand. Discontinuing operations net loss for the March 31, 2022 quarter aggregated $4.8 million and included a non-cash impairment charge of $4.3 million resulting from changed market conditions for contract research organizations from December 31, 2021 to March 31, 2022.

Total revenue from continuing operations increased 49.3%, or $100 thousand, to $303 thousand for the three months ended March 31, 2022, as compared with $222 thousand for the three months ended March 31, 2021.

Cost of goods sold – service from continuing operations totaled $38 thousand and $64 thousand, respectively, for the three months ended March 31, 2022 and 2021, resulting in a cost of goods sold of 40% and 66%, respectively, of service revenue.

Cost of goods sold – product costs decreased by 12%, or $48 thousand to $348 thousand for the three months ended March 31, 2022, as compared with $396 thousand for the three months ended March 31, 2021.

Research and development expenses increased by 89%, or $731 thousand, to $1.6 million for the three months ended March 31, 2022 from $820 thousand for the three months ended March 31, 2021. This increase is principally due a $336 thousand increase in payroll-related and consulting expenses, a $315 thousand increase in research and development activities at our Maple Grove facility, and $48 thousand related to moving to a new facility in California.

Selling, general and administrative expenses increased by 128%, or $1.5 million, to $2.8 million for the three months ended March 31, 2022, as compared with $1.2 million for the three months ended March 31, 2021. The 2021 period reflects the Company as a privately-held company whereas the 2022 period reflect the Company as a publicly-held company. The quarter ended March 31, 2022 includes incremental $564 thousand of payroll-related expenses, including one-time contractual severance benefits for two former employees of $437 thousand. The Company incurred incremental professional services fees of $472 thousand in the first quarter of 2022 as compared with the same prior-year period related to accounting, audit and other professional services and incurred $418 thousand of additional insurance expense.

Vyant Bio’s Conference Call and Webcast and Information

Vyant Bio’s management will host a conference call on Monday, May 16, 2022 at 4:30pm ET to discuss the first quarter 2022 results and provide strategic business updates as well as answer questions.

On May 16, 2022 AIM ImmunoTech Inc. (NYSE: American AIM) ("AIM" or the "Company"), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases, including COVID-19, the disease caused by the SARS-CoV-2 virus, reported its financial results for the first quarter 2022 and provided a business update (Press release, AIM ImmunoTech, MAY 16, 2022, View Source [SID1234614582]).

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"The first quarter was marked by the rollout of important clinical data across our pipeline which gives us added confidence in the potential of Ampligen as an important treatment in high-value indications," commented Thomas Equels, Chief Executive Officer of AIM. "We published clinical efficacy data in our three lead indications, pancreatic, ovarian and triple negative breast cancers in a prestigious peer-reviewed journal and/or at leading oncology scientific congresses. These data were consistent with prior clinical data seen with Ampligen and we believe validate our conviction of its potential in these difficult to treat cancers, including data indicating potentially important effects in combination with checkpoint inhibitors. We remain committed to continue working with preeminent scientific institutions to advance our rich oncology pipeline towards commercialization that we believe will drive the execution of clinical, regulatory and operational catalysts that have the potential to build shareholder value in the near and long-term."

Recent Highlights

Provided a summary of clinical data that support the potential of Ampligen with checkpoint blockade therapies (see AIM’s 4/21/22 press release).
Engaged world-renowned CRO, Amarex Clinical Research LLC to conduct upcoming Phase 2 study of Ampligen as a therapy for locally advanced pancreatic cancer (AMP-270).
Presented data at the prestigious American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022:
Negative impact of paclitaxel on human breast tumor microenvironment and its reversal by the combination of interferon-α with TLR3 agonist rintatolimod
Initial results of a phase II study evaluating a chemokine-modulatory (CKM) regimen in patients with colorectal cancer metastatic to the liver
Systemic Rintatolimod and Interferon-α2b selectively reprogram local tumor microenvironment in patients with metastatic triple negative breast cancer for enhanced influx of cytotoxic T-lymphocytes but not regulatory T-cells
Combined loco-regional and systemic, triple agent chemoimmunotherapy increases biomarkers of T cell chemotaxis in ovarian cancer
Received notification from the U.S. Food and Drug Administration ("FDA") that the FDA’s Clinical Hold on AIM’s investigational new drug ("IND") application for a Phase 2 study of Ampligen as a therapy for locally advanced pancreatic cancer (AMP-270) has been lifted and the Company may proceed with the study.
Announced the publication of positive data from a single-center, named-patient program treating advanced and metastatic pancreatic cancer patients (see: Rintatolimod (Ampligen) enhances numbers of peripheral B cells and is associated with longer survival in patients with locally advanced and metastasized pancreatic cancer pre-treated with FOLFIRINOX: a single-center named patient program)
Announced a contract for the strategic sale of its facility located in New Brunswick, New Jersey for a purchase price of $3.9 million.
Appointed biotech and finance industry veteran Robert Dickey IV as Chief Financial Officer, effective April 4, 2022.
Announced the publication of positive results from Phase 1/2 study of intraperitoneal chemo-immunotherapy in advanced recurrent ovarian cancer.
Clinical Program Update

Ampligen (rintatolimod): dsRNA being developed for globally important cancers, viral diseases and disorders of the immune system

Ampligen has demonstrated in the clinic a potential for standalone efficacy in a number of solid tumors. Additionally, Ampligen has shown therapeutic synergy with checkpoint inhibitors, including increasing survival rates and efficacy, in the treatment of animal tumors when used in combination with checkpoint blockade therapies. The first detection of Ampligen’s synergistic potential with checkpoint blockade therapeutics was witnessed in pre-clinical mouse models of melanoma and pancreatic cancers. Additionally, the Company now has data from two clinical studies – in advanced recurrent ovarian cancer and triple negative breast cancer – that indicate that the drug may have similar anti-tumor activity in humans. (See AIM’s 8-K financial filing on 6/14/21 regarding a patent in the Netherlands)

Ampligen is being evaluated as a combinational therapy for the treatment of a variety of solid tumor types in multiple clinical trials – both underway and planned – at major cancer research centers. Ampligen is also being used as a monotherapy to treat pancreatic cancer patients in an Early Access Program (EAP) approved by the Inspectorate of Healthcare in the Netherlands at Erasmus Medical Center.

Immuno-Therapy Targeting Multiple Cancers with High Unmet Need

Locally Advanced Pancreatic Cancer – Phase 2 study IND cleared by the U.S. Food and Drug Administration ("FDA"). Study will compare the efficacy of Ampligen versus a no treatment control group following FOLFIRINOX for subjects with locally advanced pancreatic adenocarcinoma. Approximately 90 subjects expected to be enrolled across up to 30 centers in the U.S. and Europe. Study is on track to commence in Q3 2022.
Advanced Recurrent Ovarian Cancer – Phase 1/2 study of intraperitoneal chemo-immunotherapy in advanced recurrent ovarian cancer. Phase 1 portion was completed. The Phase 2 portion of the study is planned to be conducted in the future. ClinicalTrials.gov: NCT02432378
Advanced Recurrent Ovarian Cancer – A follow-up Phase 2 study of advanced recurrent ovarian cancer using cisplatin and pembrolizumab, plus Ampligen; up to 45 patients to be enrolled; numerous patients have commenced treatment. ClinicalTrials.gov: NCT03734692
Stage 4 Colorectal Cancer Metastatic to the Liver – Phase 2a study of Ampligen as a component of a chemokine modulatory regimen on colorectal cancer metastatic to liver was completed and met primary endpoint, evidenced by increased CD8a expression post-treatment (p=0.046); 19 patients were enrolled and 12 patients were evaluable. Data suggest that chemokine modulatory (CKM) regimen with Ampligen may be useful to enhance effectiveness of immunotherapies. Data from the Phase 2a study were presented in April 2022 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022. ClinicalTrials.gov: NCT03403634
Stage 4 Metastatic Triple Negative Breast Cancer – a Phase 1 study of metastatic triple-negative breast cancer using CKM therapy, including Ampligen and pembrolizumab, successfully met its primary endpoint. Eight patients were enrolled and 6 patients were evaluable. Positive data from this proof-of-concept study indicate that short-term systemic CKM followed by pembrolizumab is well-tolerated and selectively enhances local cytotoxic T-lymphocyte (CTL) infiltration in the tumor microenvironment (TME). The data from the Phase 1 study was presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 in April 2022. ClinicalTrials.gov: NCT03599453
Early-Stage Prostate Cancer – Phase 2 study investigating the effectiveness and safety of aspirin and Ampligen with or without interferon-alpha 2b (Intron A) compared to no drug treatments in a randomized three-arm study of patients with prostate cancer before undergoing radical prostatectomy. Patient enrollment has been initiated in this study designed for up to 45 patients. ClinicalTrials.gov: NCT03899987
Early-Stage Triple Negative Breast Cancer – Phase 1 study of chemokine modulation plus neoadjuvant chemotherapy in patients with early-stage triple negative breast cancer has received FDA authorization. The objective of this study is to evaluate the safety and tolerability of a combination of Ampligen and celecoxib with or without Intron A, when given along with chemotherapy. The goal of this approach is to increase survival. This study is recruiting patients and is designed for up to 24 patients. ClinicalTrials.gov: NCT04081389
Refractory Melanoma – Phase 2 study that will evaluate polarized dendritic cell vaccine, interferon alpha-2, Ampligen and celecoxib for the treatment of HLA-A2+ refractory melanoma at Roswell Park. Up to 24 patients to be enrolled. ClinicalTrials.gov: NCT04093323
Advanced Ovarian Cancer – AIM plans to develop a Phase 2 Cisplatin Resistant Advanced Recurrent Ovarian Cancer Clinical Study utilizing Ampligen at the University of Pittsburgh.
Broad-Spectrum Immune System Response Against SARS-CoV-2 (COVID-19)

Previous animal studies yielded positive results utilizing Ampligen to treat Western Equine Encephalitis Virus, Ebola and SARS-CoV-1. The Company has also conducted laboratory experiments in SARS-CoV-2 showing Ampligen has a powerful impact on viral replication. The prior studies of Ampligen in SARS-CoV-1 animal experimentation may predict similar protective effects against SARS-CoV-2. AIM is currently evaluating the safety and effectiveness of intravenous Ampligen to reduce replication of SARS-CoV-2 virus from upper airway in patients in an ongoing Phase 1/2 study for the treatment of COVID-19 cancer patients. The Company also plans to conduct an intranasal study of Ampligen to potentially enhance and expand natural immunity.

Immune System Disorders (ISD): Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) / COVID-19 Long Hauler

The Company is currently sponsoring an expanded access program (EAP) for ME/CFS patients in the United States, and in 2021 AIM dosed its first "Long Hauler" patient with Ampligen in its post-COVID-19 "Long Hauler" portion of the active AMP-511 EAP in the United States. Patients receiving Ampligen in the ongoing AMP-511 EAP with Long Hauler/Post-COVID conditions (such as fatigue and cognitive function deficiency) have reported improvements after receiving Ampligen.

Recent Ampligen Data Publications

Positive data from a single-center named patient program was published in March 2022. The manuscript titled, "Rintatolimod (Ampligen) enhances numbers of peripheral B cells and is associated with longer survival in patients with locally advanced and metastasized pancreatic cancer pre-treated with FOLFIRINOX: a single-center named patient program1," was published in the peer-reviewed journal, Cancers Special Issue: Combination and Innovative Therapies for Pancreatic Cancer.
Positive results of a Phase 1/2 study of intraperitoneal chemo- immunotherapy in advanced recurrent ovarian cancer were published in January 2022. The manuscript titled, "Phase I trial combining chemokine-targeting with loco-regional chemo-immunotherapy for recurrent, platinum-sensitive ovarian cancer shows induction of CXCR3 ligands and markers of type 1 immunity2" was published in the American Association for Cancer Research (AACR) (Free AACR Whitepaper) publication, Clinical Cancer Research.
Summary of Financial Highlights for First Quarter 2022

As of March 31, 2022, AIM reported cash and cash equivalents of $44.5 million. Based on management’s current expectation, the Company’s cash runway is expected to fund operations through 2023.
Research and development expenses for the three months ended March 31, 2022 were $1.0 million, compared to $1.4 million for the same period in 2021.
General and administrative expenses were $2.1 million for the three months ended March 31, 2022 and 2021.
The net loss from operations for the three months March 31, 2022 was $3.8 million, or $0.08 per share, compared to $3.6 million, or $0.08 per share, for the three months ended March 31, 2021.
Please refer to the full 10-Q for complete details.

On May 16, 2022 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported that Dr. Neal Walker, President and CEO of Aclaris, will virtually present a company overview at the H.C. Wainwright Global Investment Conference, which will be available beginning on Tuesday, May 24, 2022 at 7:00 a.m. ET (Press release, Aclaris Therapeutics, MAY 16, 2022, View Source [SID1234614581]). Management will be available May 23rd throughout the day for virtual 1×1 meetings.

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A webcast of the presentation may be accessed through the "Events" page of the "Investors" section of Aclaris’ website, www.aclaristx.com. The webcast will be archived for at least 30 days on the Aclaris website.

On May 16, 2022 Scandion Oncology (Scandion) reported that will publish its Q1 2022 interim report on Thursday, May 19, 2022 before 09:00 CET (Press release, Scandion Oncology, MAY 16, 2022, View Source;2022,c3567316 [SID1234614574]).

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Scandion Oncology’s Executive Management will host a webcast and conference call the same day at 10:00 CET presenting the results and a company update.

At the end of the presentation there will be a Q&A session.

The information was provided by the contact person above for publication on May 16, 2022, at 08.30 CET.