On August 17, 2021 AIM ImmunoTech Inc. (NYSE American: AIM) reported financial results for the second quarter ended June 30, 2021 and provides a business update (Press release, AIM ImmunoTech, AUG 17, 2021, View Source [SID1234586680]).

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Second Quarter 2021 Financial Highlights:

As of June 30, 2021, AIM had cash, cash equivalents and marketable securities of $57.3 million, compared to $54.4 million as of December 31, 2020.

Research and development expenses for the three months ended June 30, 2021 were $1.3 million, compared to $1.5 million for the three months ended June 30, 2020.

General and administrative expenses for the three months ended June 30, 2021 were $2.1 million, compared to $1.7 million for the three months ended June 30, 2020.

The net loss from operations for the three months ended June 30, 2021 was $5.9 million, or $0.12 per share, compared to $3.4 million, or $0.11 per share, for the three months ended June 30, 2020.

Please refer to the full 10-Q for complete details.

2021 Clinical and Business Highlights

After several years of success in various studies and trials, AIM has decided to strategically narrow its focus to trials and activities that have the shortest path to potential FDA and EMA drug approval, thus putting energy into the tasks where clinical results suggest an opportunity for expedited success. AIM anticipates that these planned trials primarily will be AIM-sponsored and AIM-funded.

Immuno-oncology

Analyses of data collected in the Early Access Program at Erasmus Medical Center (Erasmus MC) in the Netherlands found a statistically significantly positive survival benefit when using Ampligen in patients with locally advanced/metastatic pancreatic cancer after systemic chemotherapy. Based on these encouraging data, AIM is developing a protocol design and schema – in consultation with Amarex Clinical Research, Buffet Cancer Center at the University of Nebraska and Erasmus MC – to seek regulatory approval to initiate a Phase 2/3 clinical trial of Ampligen in the United States and/or the European Union.

Multiple additional Ampligen clinical trials are underway at university cancer centers testing whether tumor microenvironments can be reprogrammed to increase the effectiveness of cancer immunotherapy, including with checkpoint inhibitors:

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; enrollment has commenced, and 17 subjects have now commenced treatment. View Source
Stage 4 Metastatic Triple Negative Breast Cancer – Phase 1/2 study of metastatic triple-negative breast cancer using chemokine modulation therapy, including Ampligen and pembrolizumab. Eight patients were enrolled and treated. We await publication of data. View Source
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; 15 patients were enrolled and treated. We await publication of data. View Source
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. View Source
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. View Source
COVID-19

In January, AIM entered into a Sponsor Agreement with the Centre for Human Drug Research (CHDR), a foundation located in Leiden in the Netherlands, to manage a Phase 1 randomized, double-blind study to evaluate the safety and activity of repeated intranasal administration of Ampligen. AIM funded and sponsored the study. This study was designed to assess the safety, tolerability and biological activity of repeated administration of Ampligen intranasally. A total of 40 healthy subjects received either Ampligen or a placebo in the trial, with the Ampligen given at four escalating dosages across four cohorts, to a maximum level of 1,250 micrograms. All patients had completed treatment by June 2021 and the interim results reported no Severe Adverse Events at any dosage level. The object was to establish safety for intranasal Ampligen as a potential broad spectrum prophylaxis for respiratory viruses, including SARS-CoV-2. Following the completion of the Phase 1 dosing, and based on its positive interim results, in July 2021 AIM signed a Reservation and Start-Up Agreement with hVIVO, reserving space in hVIVO’s quarantine facility to sponsor a Phase 2a Human Challenge Trial (HCT) to test Ampligen as a potential intranasal antiviral therapy using a human Rhinovirus hRV (a common cold virus) and Influenza as challenge viruses. This antiviral study will be conducted by hVIVO, a subsidiary of Open Orphan plc, a rapidly growing specialist pharmaceutical services clinical research organization and world leader in vaccine and antiviral testing using human challenge clinical trials. The trial is expected to commence in Q4 2021. The object is to establish Ampligen’s potential as a broad spectrum prophylaxis for respiratory viruses, including SARS-CoV-2.

In an HCT, subjects are intentionally exposed to particular disease pathogens to test how the diseases will respond to the test treatment. An HCT will allow the Company to expedite the development process for Ampligen by ensuring that all subjects in both the control group and the Ampligen group are exposed to the target pathogen, so as to assess whether there is a prophylactic effect. A successful Phase 2a HCT study could also establish Ampligen’s potential as a prophylaxis against future viral variants and future novel respiratory viruses for which there are no current vaccines or therapies, as well as known viruses such as SARS-CoV-2, which causes COVID-19.

Immuno-oncology / COVID-19

In June, Ampligen was featured in a publication containing state-of-the-art methodologies in the peer-reviewed medical journal Cancers as a potential treatment option for cancer patients who are infected with SARS-CoV-2. The study’s authors stated that Ampligen has the potential to reduce the severity of the deadly respiratory disease COVID-19, which has so far caused more than 4.3 million deaths globally. The full journal article is titled: "Rintatolimod Induces Antiviral Activities in Human Pancreatic Cancer Cells: Opening for an Anti-COVID-19 Opportunity in Cancer Patients?" Cancers is a peer-reviewed, open access journal of oncology published semimonthly online by MDPI. The study’s authors include Prof. C.H.J. van Eijck, MD, PhD, the lead investigator for an Early Access Program at Erasmus Medical Center in the Netherlands.

Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) / COVID-19

On January 6, AIM announced that the post-COVID-19 "Long Hauler" portion of the active AMP-511 Expanded Access Program (EAP) in the U.S. dosed its first "Long Hauler" patient with the drug Ampligen, marking a significant milestone in AIM’s efforts to develop an effective therapeutic for people suffering from post-COVID-19 infection chronic fatigue-like symptoms. Additional patients are now in treatment and AIM intends to provide further updates as the trial progresses.

"We continued to make positive clinical and operational progress during the second quarter," commented, Thomas K. Equels, CEO of AIM ImmunoTech. "With our strong balance sheet, we are well positioned to execute on our corporate strategy and advance our clinical trials to meaningful milestones that we believe will drive significant shareholder value. We anticipate reporting material developments throughout the remainder of this year and through 2022. We remain extremely encouraged by the outlook for Ampligen."

On August 17, 2021 MorphoSys AG (FSE: MOR; NASDAQ: MOR) reported that Incyte, its development and commercialization partner for tafasitamab, entered into a collaboration and license agreement with a subsidiary of InnoCare for tafasitamab in Greater China (Press release, MorphoSys, AUG 17, 2021, View Source [SID1234586675]). Under the terms of the agreement, InnoCare will receive the rights to develop and exclusively commercialize tafasitamab, a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody, in hematology-oncology in mainland China, Hong Kong, Macau and Taiwan.

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In January 2020, MorphoSys and Incyte entered into a collaboration and license agreement to develop and commercialize MorphoSys’ proprietary anti-CD19 antibody tafasitamab globally. MorphoSys and Incyte co-commercialize tafasitamab in the U.S., and Incyte holds the development and commercialization rights for tafasitamab outside the U.S. MorphoSys will receive tiered royalties on ex-U.S. net sales.

About Tafasitamab
Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Monjuvi(R) (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi(R) is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.

In June 2021, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending the conditional marketing authorization of tafasitamab in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplantation (ASCT). The CHMP opinion is currently being reviewed by the European Commission, which has the authority to grant marketing authorization for medicinal products in the European Union (EU).

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

Monjuvi(R) is a registered trademark of MorphoSys AG.
XmAb(R) is a registered trademark of Xencor, Inc.

On August 16, 2021 Celularity Inc. ("Celularity") (Nasdaq:CELU), a clinical-stage biotechnology company developing off-the-shelf placental-derived allogeneic therapies, reported financial results for the quarter ended June 30, 2021, and provided a summary of recent corporate highlights (Press release, Celularity, AUG 16, 2021, View Source [SID1234591816]).

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"This has been an exciting time for Celularity, with the achievement of multiple transformational milestones and significant progress in our unique approach to cellular medicine," said Robert J. Hariri, M.D., Ph.D., founder, Chairperson and Chief Executive Officer of Celularity. "Most notably, this quarter marked our transition to a public company through a merger with GX Acquisition Corp., which along with a companion PIPE provided significant funds to support our work. Additionally, we made noteworthy advances in our clinical programs, including the expansion of our Phase 1 trial in patients with acute myeloid leukemia, to include difficult to treat patient populations. Beyond our program development, we forged new strategic and commercial partnerships with companies at the forefront of their respective fields that continue our legacy of pioneering new and innovative approaches to cellular medicine. We look forward to continuing to advance the field of cellular medicine and developing treatments capable of addressing significant unmet needs in cancer, autoimmune and infectious disease."

Corporate Highlights

Celularity closed the merger with GX Acquisition Corp. ("GXGX"). Proceeds from the transaction totaled approximately $138 million, which included funds held in GXGX’s trust account and a concurrent private placement investment in public equity (PIPE) financing led by existing Celularity shareholders.
Celularity expanded its ongoing Phase 1 clinical trial of CYNK-001 in patients with acute myeloid leukemia (AML) (NCT04310592) to include patients with relapsed/refractory AML (r/r AML) in addition to its ongoing trial in patients positive for minimal residual disease (MRD).
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to Celularity’s CYNK-001, a non-genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy, for the treatment of patients with malignant gliomas.
Celularity entered an exclusive strategic partnership with Imugene Ltd to develop a novel oncolytic virus – allogeneic chimeric antigen receptor (CAR) T-cell immunotherapy combination for the treatment of solid tumors. The collaboration will initially explore the therapeutic potential of a combination of Imugene’s CF33-CD19 oncolytic virus (onCARlytics) and Celularity’s placental-derived CD19 targeting CAR T-cell therapy, CYCART-19.
Celularity established a partnership to leverage Palantir’s next generation software and computational capabilities to analyze Celularity’s cellular data and accelerate research and development activities.
On July 1, 2021, Celularity announced its agreement with Arthrex whereby Arthrex would receive exclusive rights to distribute and commercialize Celularity’s placental-derived biomaterial products for orthopedics and sports medicine in the U.S. Under the terms of the agreement, Celularity will provide Arthrex with exclusive commercial distribution rights for orthopedic surgery and sports medicine and will continue to be responsible for product manufacturing and supply.
Second Quarter 2021 Financial Results

Revenues for the three months ended June 30, 2021, experienced a decrease of $0.3 million compared to the prior year. This was due to a decrease in product sales and rentals revenue resulting from the $24.5 million sale of the MIST/UltraMIST assets in August 2020, partially offset by (i) an increase of $0.6 million in license, royalty and other revenues related to the license arrangement with Sanuwave and (ii) an increase of $0.2 million in services revenues primarily due to higher biobanking storage revenues.
Research and development expenses for the three months ended June 30, 2021, increased $7.1 million compared to the prior year. The increase in research and development expenses was primarily due to a non-cash stock compensation charge related to the grant of fully vested senior management awards.
Selling, general and administrative expenses for the three months ended June 30, 2021, increased $21.3 million compared to the prior year, primarily due to a non-cash stock-based compensation charge related to the grant of fully vested non-employee director and senior management awards.
Net loss for the second quarter of 2021 was $64.5 million, or $2.69 per share.

On August 16, 2021 Sino Biological, a Beijing company that offers biological research reagents and related contract research services to global markets, reported that it has completed a $772 million IPO on Shenzhen’s ChiNext Exchange (Press release, Sino Biopharmaceutical, AUG 16, 2021, View Source [SID1234586738]). On the first day of trading, the stock closed 68% higher at a market capitalization of $5.2 billion. The company, which was founded in 2007, said the IPO funds would represent a new starting point, allowing Sino Biological to expand its existing capabilities.

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On August 16, 2021 Biosight Ltd., a pharmaceutical development company developing innovative therapeutics for hematological malignancies and disorders, reported the initiation of a Phase 2 trial to evaluate aspacytarabine (BST-236), Biosight’s proprietary antimetabolite, as a second line treatment for patients with relapsed or refractory myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (Press release, Advaxis, AUG 16, 2021, View Source [SID1234586688]). The investigator sponsored trial will be led by Dr. Pierre Fenaux of the Groupe Francophone des Myélodysplasies (GFM), the French Study Group of the European Myelodysplastic Syndromes (MDS) Cooperative Group (EMSCO). GFM is a non-profit organization comprised of most French hematology centers that conducts and sponsors clinical trials and translational research and coordinates diagnostic and therapeutic guidelines for MDS.

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"This new trial is an important step forward in expanding the reach of aspacytarabine to a broader patient population, addressing the unmet needs in the treatment of relapsed/refractory AML and MDS," said Dr. Ruth Ben Yakar, Chief Executive Officer of Biosight. "Initiating an additional trial furthers our clinical momentum, building on updated, encouraging data from our ongoing first-line Phase 2b study presented at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the receipt of Orphan Medicinal Product Designation for the European Medicines Agency. GFM is an ideal partner to expand the clinical evaluation into MDS with their extensive network of hematology centers, including registered centers of excellence, and we look forward to applying their expertise in clinical trials, treatments and diagnostics as we advance this Phase 2 trial."

Professor Pierre Fenaux, M.D., Ph.D., Head of Hematology at Hospital Saint Louis in Paris, and founding member and Chairman of GFM, said, "The first-line Phase 2b data that were presented at ASCO (Free ASCO Whitepaper), with demonstrated efficacy across key measures including encouraging complete remission and negative minimal residual disease rates, duration of response and overall survival, further increase my conviction that aspacytarabine may serve as a more tolerable, end effective standard of care treatment for patients with both AML and MDS. We are thrilled to be collaborating with Biosight to advance this potentially transformative treatment for relapsed or refractory MDS and AML patients who currently are faced with poor prognoses and no effective standard of care treatment."

About Aspacytarabine (BST-236)

Aspacytarabine is a novel proprietary anti-metabolite. It is composed of cytarabine covalently bound to asparagine, acting as a pro-drug of cytarabine. Cytarabine serves as the backbone of AML therapy for over 45 years due to its superior efficacy, however, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities, which significantly limit its use, especially in older and medically compromised patients. Due to its unique pharmacokinetics and metabolism, aspacytarabine enables high-dose therapy with lower systemic exposure to free cytarabine and relative sparing of normal tissues. As such, aspacytarabine may serve as a new therapy for AML and other hematological malignancies and disorders, including for older adults who are unfit for intensive therapy.

Aspacytarabine was granted FDA Fast Track Designation for treatment of AML patients unfit for standard chemotherapy, and FDA and EMA Orphan Drug Designations, which entitle Biosight to seven and ten years of market exclusivity in the U.S. and Europe, respectively, upon aspacytarabine marketing approval for the treatment of AML in each territory.

Interim results from an ongoing Phase 2b study evaluating aspacytarabine as a single-agent first-line AML therapy demonstrate safety and single-agent activity, and additional studies are launched to evaluate aspacytarabine as a second line treatment for patients with relapsed or refractory MDS or AML. For more information regarding the Phase 2b clinical study of BST-236, please visit www.clinicaltrials.gov.