On March 31, 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 full year 2021 and provided a business update (Press release, AIM ImmunoTech, MAR 31, 2022, View Source [SID1234611287]).

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"We believe 2021 was an important, foundational year for AIM. Our oncology pipeline continued to develop and generate important data across our clinical programs. This progress has substantially and positively impacted our growing body of data – data which continues to be consistent with key findings across multiple indications. Importantly, this data provides valuable guidance as we are identifying the clear pathways for next steps with Ampligen, as a monotherapy as well as its utility as a combination therapy, in high-value indications with the potential to fill significant gaps in various treatment paradigms," commented Thomas Equels, Chief Executive Officer of AIM. "So far, in 2022 Ampligen data has been published in peer-reviewed journals and multiple abstracts and posters have been accepted at prestigious scientific congresses, representing noteworthy progress across our pipeline. Over the course of 2022, we expect to achieve a number of additional potentially value-driving clinical, regulatory and operational catalysts. Our team is laser focused on executing on our strategy and propelling AIM toward its next phase of growth."

Recent Highlights

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.
Announced the strategic sale of its facility located in New Brunswick, New Jersey for a purchase price of $3.9 million.
Appointed 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 the potential for standalone efficacy in a number of solid tumors. Additionally, Ampligen has shown success in increasing survival rates and efficacy in the treatment of animal tumors when used in combination with checkpoint blockade therapies. 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 around the country. 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

Advanced Recurrent Ovarian Cancer – Phase 1 portion was completed. 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 has been completed; 15 patients were enrolled and treated. Data was accepted for a late-breaking presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 being held April 8-13, 2022 and are under embargo until then. ClinicalTrials.gov: NCT03403634
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. Data was accepted for a late-breaking presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 being held April 8-13, 2022 and are under embargo until then. 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 conducted 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 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. Early data from the ongoing AMP-511 EAP and data from an earlier study, AMP-502, has indicated that patients with cognitive function deficiency have reported improvements in cognitive function after Ampligen treatment.

Summary of Recent Ampligen Data Publications

Abstracts accepted for presentation at American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, being held April 8-13, 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
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.
Rintatolimod Induces Antiviral Activities in Human Pancreatic Cancer Cells: Opening for an Anti-COVID-19 Opportunity in Cancer Patients?
Phase II Trial of Adjuvant Dendritic Cell Vaccine in Combination with Celecoxib, Interferon-α, and Rintatolimod in Patients Undergoing Cytoreductive Surgery and Hyperthermic Intraperitoneal Chemotherapy for Peritoneal Metastases
Summary of Financial Highlights for Fiscal Year 2021

As of December 31, 2021, AIM reported cash and cash equivalents of $48.3 million, compared to $54.4 million as of December 31, 2020.
Research and development expenses for the year ended December 31, 2021 were $7.6 million, compared to $5.7 million for the year ended December 30, 2020.
General and administrative expenses for the year ended December 31, 2021 were $8.7 million, compared to $8.7 million for the year ended December 31, 2020.
The net loss from operations for the year December 31, 2021 was $19.1 million, or $0.40 per share, compared to $14.4 million, or $0.45 per share, for the year ended December 31, 2020.

On March 31, 2022 Silverback Therapeutics, Inc. (Nasdaq: SBTX) ("Silverback"), a biopharmaceutical company leveraging its proprietary ImmunoTAC technology platform to develop systemically delivered, tissue targeted therapeutics for the treatment of chronic viral infections, cancer, and other serious diseases, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Silverback Therapeutics, MAR 31, 2022, View Source [SID1234611286]).

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"Upon comprehensive review of our clinical and preclinical data for our TLR8 oncology programs, we have made the decision to discontinue the development of SBT6050 and SBT6290, and focus our resources on SBT8230 for chronic HBV as well as our ImmunoTAC discovery programs," said Laura Shawver, Ph.D., chief executive officer of Silverback. "We would like to thank the investigators and the staff at each of our sites, and most importantly, the patients who participated in our trial and their families."

Business Update and Strategy

SBT6050 and SBT6290 (HER2-TLR8 and Nectin4-TLR8 ImmunoTAC conjugates for oncology)

Silverback has discontinued the SBT6050 development program. In the Phase 1/1b trial, a total of 58 patients were enrolled and received SBT6050 as monotherapy and in combination with a checkpoint inhibitor at dose levels ranging from 0.15 mg/kg through 1.2 mg/kg with the length of patient experience ranging from 2 weeks through 41 weeks. A dose response was observed in serum and intratumoral exposure, and in pharmacodynamic markers, inclusive of data that demonstrates immune activation in biopsies collected from patients after treatment. Further development was discontinued based on limited monotherapy anti-tumor activity and cytokine-related adverse events that limited the dose in combination with pembrolizumab.

SBT6290, comprised of the same linker payload conjugated to a Nectin4 antibody, was expected to show a similar clinical profile and, therefore, this development program was also discontinued.

SBT8230 (ASGR1-TLR8 ImmunoTAC conjugate for chronic HBV)

"Our understanding of TLR8 conjugates in preclinical species and in the clinic provides a lens for interpretation of the preclinical characteristics of SBT8230," said Valerie Odegard, Ph.D., president and chief scientific officer. "The comparative preclinical data between SBT6050 and SBT8230 suggest that the clinical safety, pharmacokinetic and pharmacodynamic profiles for SBT8230 will likely be different than those for SBT6050, given the significant differences in preclinical serum exposures and expected overall conjugate disposition for SBT8230 in patients due to its efficient liver targeting. We continue to advance SBT8230 and are on track to complete a Phase 1 regulatory submission in the fourth quarter of 2022."

SBT8230 is comprised of an ASGR1 monoclonal antibody conjugated to a TLR8 linker-payload and is designed to elicit an anti-viral immune response by targeting TLR8 activation to the liver. ASGR1 is highly expressed in liver and is restricted in its expression to this organ. An anti-viral immune response is achieved through activation of myeloid cells and subsequent indirect activation of B cells and T cells. In non-human-primate studies, SBT8230 demonstrated lower serum exposures compared to SBT6050 due to its efficient localization to liver. Liver-localized TLR8 agonism has the potential to lead to durable responses and possibly seroconversion, an important determinant of functional cure. At the AASLD Liver Meeting 2021, Silverback presented preclinical studies demonstrating that SBT8230 was efficiently delivered to the liver, resulting in myeloid cell activation in the liver but not in the blood. Silverback initiated Phase 1-enabling toxicology studies for SBT8230 in the first quarter of 2022.

ImmunoTAC Discovery Program

Silverback will continue advancement of early-stage discovery research that is focused on exploring different antigen targets, novel linker technologies, and small molecule payloads that expand the reach of the ImmunoTAC platform.

Key Strategic Priorities and Cash Runway Extension

Complete the Phase 1 regulatory submission for SBT8230 in the fourth quarter of 2022

Open enrollment for a Phase 1 single ascending dose study of SBT8230 in healthy volunteers in the first half of 2023

Provide an update on Silverback’s discovery pipeline in the fourth quarter of 2022

Restructure workforce to focus resources on SBT8230 program and discovery pipeline, reducing headcount by 27%

Estimated cash runway extended into the second half of 2026 following strategic prioritization

Dr. Shawver added, "Over the course of the next few days and weeks, we are restructuring our workforce and allocating resources around our new strategic priorities. It will be difficult to part with valued team members who have been so committed to the organization, and I’d like to thank each one of them for their valuable contributions towards our mission to develop the next generation of tissue targeted therapeutics."

Financial Results

For the fourth quarter ended December 31, 2021, Silverback reported a net loss of $23.5 million, compared to a net loss of $13.1 million for the comparable period in 2020. For the year ended December 31, 2021, Silverback reported a net loss of $89.5 million, compared to a net loss of $32.9 million for 2020. Net loss for the fourth quarter and full year of 2021 included non-cash stock-based compensation expense of $5.2 million and $19.2 million, respectively, compared to $2.3 million and $2.6 million for the same periods in 2020, respectively.

Research and development expenses for the fourth quarter ended December 31, 2021 were $15.9 million, compared to $8.8 million for the same period in 2020. For the year ended December 31, 2021, research and development expenses were $61.5 million, compared to $24.6 million for 2020. The increases in Silverback’s research and development expenses for the 2021 periods, as compared to the same periods in 2020, were primarily attributable to an increase in direct costs related to the development of SBT6050 and SBT6290 and direct costs related to SBT8230 and other preclinical research efforts. Silverback also incurred additional personnel-related expenses in 2021 as compared to 2020 as operations grew in support of program advances.

General and administrative expenses for the fourth quarter ended December 31, 2021 were $7.6 million, compared to $4.3 million for the same period in 2020. For the year ended December 31, 2021, general and administrative expenses were $28.1 million, compared to $8.3 million for 2020. The increases in general and administrative expenses for the 2021 periods, as compared to the same periods in 2020, were primarily attributable to an increase in personnel-related expenses due to increased headcount in 2021, including new executives that were new in 2020 being present for a full year in 2021, as well as increases in salaries, bonuses, and stock-based compensation. To a lesser extent, the increase in general and administrative expenses was due to an increase in professional fees primarily attributable to legal, insurance, and outside consultant costs.

As of December 31, 2021, Silverback reported cash, cash equivalents, restricted cash, and investments of $319.1 million, compared to cash and cash equivalents of $386.6 million at December 31, 2020, which is expected to fund operating expenses and capital expenditure requirements into the second half of 2026 following strategic prioritization. As of December 31, 2021, Silverback had 35,133,934 shares of common stock outstanding.

Conference Call and Webcast on Thursday, March 31, 2022 at 5:00 PM ET

Silverback’s management team will host a conference call on Thursday, March 31, 2022 at 5:00 PM ET to discuss the strategic prioritization and corporate update. A live webcast, including slides, can be accessed through the Events section of the Company’s website at View Source An archived replay will be available shortly after the conclusion of the event.

On March 31, 2022 Celularity Inc. (Nasdaq: CELU) ("Celularity"), a clinical-stage biotechnology company developing placental-derived off-the-shelf allogeneic cell therapies, reported a corporate update and financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Celularity, MAR 31, 2022, View Source [SID1234611285]).

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"The past year has been exciting for Celularity, during which we achieved 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, we transitioned to a public company through a business combination with GX Acquisition Corp., which along with a companion PIPE, contributed to advancing our preclinical and clinical programs targeting significant unmet medical needs. We also were granted five regulatory designations by the FDA, which we believe provides validation for the potential of our approach to change the cell therapy landscape and improve the lives of patients. Beyond our active clinical development programs, we forged new strategic and commercial partnerships with companies at the forefront of their respective fields, again consistent with our mission to pioneer new, innovative approaches to cellular medicine."

Dr. Hariri continued, "Our achievements over the past year establish a solid foundation for growth through 2022. We expect Phase 1 data readouts in all three ongoing clinical programs and the submission of an investigational new drug application, or IND, for our CYCART-19 program to enable initiation of a Phase 1 trial in B cell malignancies. We believe we are well positioned to lead the next evolution of cellular therapeutics for the treatment of cancer, infectious and degenerative diseases using our proprietary placental-based cell therapy platform."

Clinical and Regulatory Updates

CYNK-001 for the Treatment of AML and GBM:

CYNK-001 is Celularity’s unmodified cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy candidate that is enriched with CD56+/CD3- NK cells and expanded from human placental CD34+ cells. CYNK-001 is currently being investigated in two Phase 1 clinical trials, in AML and in GBM.
In March 2021, the FDA granted Fast Track designation to CYNK-001 for the treatment of adults with recurrent GBM.
In April 2021, the FDA granted Orphan Drug Designation to CYNK-001 for the treatment of patients with malignant gliomas, including GBM.
In June 2021, Celularity announced the expansion of its ongoing Phase 1 clinical trial of CYNK-001 in AML (NCT04310592) to include patients with relapsed/refractory AML (r/r AML) in addition to an ongoing trial in patients with minimal residual disease (MRD).
In December 2021, the FDA granted Fast Track designation to CYNK-001 for the treatment of AML.
Also in December 2021, Celularity hosted an AML update with analysts in which the Company highlighted data findings in both r/r AML and MRD (NCT04310592) that provided evidence of a dose effect and biologic activity, and outlined plans to expand with new cohorts in both arms, add interleukin-2 (IL-2) to the treatment regimen, add a fourth dose on day 21 and potentially increase the dose of NK cells. The presentation from the event is available on Celularity’s investor relations website under Events & Presentations.
CYNK-101 for the Treatment of HER2+ Gastric Cancer:

CYNK-101 is a novel allogeneic off-the-shelf human placental CD34+-derived NK cell product candidate that is genetically modified to express high-affinity and cleavage-resistant CD16 (FCGRIIIA) variant to drive antibody-dependent cell-mediated cytotoxicity. CYNK-101 is currently being investigated in the Phase 1 portion of a Phase 1/2a clinical trial in HER2+ gastric cancer.
In November 2021, Celularity received clearance of its IND by the FDA for the use of CYNK-101 in combination with standard chemotherapy, trastuzumab and pembrolizumab, in patients with first-line locally advanced unresectable or metastatic HER2/neu positive gastric or gastroesophageal junction (G/GEJ) adenocarcinoma.
Also in November 2021, Celularity presented preclinical data in a poster presentation at the Society of Immunotherapy Cancer (SITC) (Free SITC Whitepaper) annual meeting. The data demonstrate the synergistic effect of combining CYNK-101 with Cetuximab to drive antibody-dependent cellular cytotoxicity activity against EGFR+ tumors.
In December 2021, Celularity presented preclinical data for CYNK-101 at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting.
In January 2022, the FDA granted Fast Track designation to CYNK-101, which is being developed in combination with standard chemotherapy, trastuzumab and pembrolizumab in first-line locally advanced unresectable or metastatic HER2/neu positive G/GEJ adenocarcinoma.
In February 2022, the FDA granted Orphan Drug Designation to CYNK-101 for the treatment of gastric/gastroesophageal junction cancer.
CYCART-19 for the Treatment of B-Cell Malignancies:

CYCART-19 is an allogeneic Chimeric Antigen Receptor (CAR) engineered human placental -derived T cell that is a potential drug candidate in B-cell malignancies.
In December 2021, preclinical data demonstrating the feasibility and functionality of expressing a CAR directed to CD19 on placental CD34+derived, cryopreserved, off-the-shelf, allogeneic CYNK cells were presented at the ASH (Free ASH Whitepaper) Annual Meeting.
Corporate Developments

In May 2021, Celularity formed a multi-year strategic partnership with Palantir Technologies to leverage the unique combined strengths of Celularity’s deep dataset and Palantir’s Foundry platform to accelerate and advance cellular therapies.
In July 2021, Celularity completed a business combination with GX Acquisition Corp. and commenced trading on the Nasdaq exchange under a new name and new ticker symbols. Gross proceeds from the transaction totaled approximately $138 million, which included funds held in the trust account and a concurrent private placement investment in public equity (PIPE) financing led by existing Celularity stockholders.
Also in July 2021, Celularity entered into an exclusive supply and distribution agreement for multiple commercial products with Arthrex to distribute and commercialize Celularity’s biomaterial products for orthopedic surgery and sports medicine.
In August 2021, Celularity entered into an exclusive strategic partnership to develop the combination of Imugene’s CD19 oncolytic virus technology and Celularity’s CD19 targeting allogeneic CAR T cellular therapy, CYCART-19, for the treatment of solid tumors.
In September 2021, Celularity appointed Andrew L. Pecora, M.D., as President. Dr. Pecora, a hematologist and oncologist, was a Celularity director prior to the business combination with GX Acquisition Corp. and chaired its Scientific Advisory Board.
Also in September 2021, Celularity entered into a research collaboration with Oncternal Therapeutics under which the companies will evaluate placental derived-cellular therapies targeting receptor-tyrosine kinase-like Orphan Receptor 1 (ROR1).
Celularity’s clinical and commercial GMP production facility has been fully operational for over a year and has been developing and supplying all placental-derived cell therapy product candidates, including CYNK-001, CYNK-101, and CYCART-19 as well as Celularity’s tissue-derived therapies including BIOVANCE, BIOVANCE•3L Ocular, and Interfyl.
Fourth Quarter and Full Year 2021 Financial Results

Cash and Cash Equivalents: Cash, cash equivalents and marketable securities were $37.2 million as of December 31, 2021, compared to $54.3 million as of December 31, 2020. Gross proceeds of the business combination and related PIPE transactions, which totaled approximately $138 million, were offset by cash used in operations, capital expenditures and professional fees related to becoming an operating public company.
Total Revenues: Total revenues were $4.9 million for the fourth quarter of 2021 and $21.3 million for the full year ended December 31, 2021, compared to $3.2 million for the fourth quarter of 2020 and $14.3 million for the full year ended December 31, 2020. The increase in revenues was primarily due to recognition of deferred revenue in connection with termination of a license agreement, as well as new distribution agreements.
Research & Development (R&D) Expenses: R&D expenses were $24.7 million for the fourth quarter of 2021 and $88.4 million for the full year ended December 31, 2021, compared to $13.9 million for the fourth quarter of 2020 and $52.7 million for the full year ended December 31, 2020. The increase was primarily driven by increased stock-based compensation as well as costs associated with increases in both preclinical and clinical activities, and headcount.
Selling, General & Administrative (SG&A) Expenses: SG&A expenses were $13.2 million for the fourth quarter of 2021 and $71.3 million for the full year ended December 31, 2021, compared to $6.0 million for the fourth quarter of 2020 and $31.3 million for the full year ended December 31, 2020. The increase in SG&A expenses was primarily caused by increased stock-based compensation expense, a legal settlement charge as well as an increase in headcount and costs associated with being a public company.
Net (loss) income: Net loss attributable to common stockholders was $4.0 million, or $0.03 per share, for the fourth quarter of 2021 and $100.1 million, or $1.49 per share for the year ended December 31, 2021, compared to income of $3.9 million, or $0.21 per share, for the fourth quarter of 2020 and a loss of $208.2 million, or $11.31 per share, for the full year ended December 31, 2020.

On March 31, 2022 Aravive, Inc. (Nasdaq: ARAV, "the Company"), a late clinical-stage oncology company developing targeted therapeutics to treat metastatic disease, reported fourth quarter and full year ended December 31, 2021 financial results and provided corporate updates (Press release, Aravive, MAR 31, 2022, View Source [SID1234611284]).

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"In 2021, Aravive made significant progress in advancing the clinical development of batiraxcept for the potential treatment of ovarian, kidney and pancreatic cancer," said Gail McIntyre, Ph.D., DABT, Chief Executive Officer of Aravive. "We initiated the registrational Phase 3 trial of batiraxcept in ovarian cancer and recently reported positive safety and efficacy data from the ongoing Phase 1b trials of batiraxcept in clear cell renal cell carcinoma and pancreatic cancer. We are encouraged by batiraxcept’s early profile in patients across each of these life-threatening cancers with unmet medical needs and are laser-focused on building value for patients and shareholders."

Recent Corporate Highlights

Batiraxcept in Platinum Resistant Ovarian Cancer (PROC): Aravive dosed the first patient in the registration directed Phase 3 trial in April 2021 and is on track to complete enrollment in 2H’22. Topline data from the trial is anticipated to be available in 2Q’23 and the Company expects to be ready to potentially file a BLA during 4Q’23. The global, randomized, double-blind, placebo-controlled Phase 3 trial is evaluating efficacy and tolerability of batiraxcept at a dose of 15 mg/kg in combination with paclitaxel versus placebo in combination with paclitaxel. The trial aims to enroll 350 platinum resistant, high-grade serous ovarian cancer patients who have received 1-4 prior lines of therapy.

Batiraxcept in Clear Cell Renal Cell Carcinoma (ccRCC) and Serum-Based Biomarker: Aravive dosed the first patient in the Phase 1b portion of the Phase 1b/2 trial of batiraxcept in patients with ccRCC in March 2021 and completed enrollment and reported positive preliminary data in November 2021. In March 2022, the Company announced positive updated data and new biomarker data from the Phase 1b portion of the trial in patients with ccRCC. Aravive had previously reported an observable correlation of baseline levels of serum soluble AXL (sAXL)/GAS6 to clinical activity in its Phase 1b PROC trial. As such, one of the objectives of the ongoing Phase 1b/2 ccRCC trial is to evaluate the correlation of baseline sAXL/GAS6 with radiographic response in patients with ccRCC treated with batiraxcept plus cabozantinib. The company anticipates to continue to provide updated biomarker data throughout 2022.

As of February 4, 2022, 26 patients with ccRCC were treated with batiraxcept in the Phase 1b portion of the trial at doses of 15 mg/kg (n=16) and 20 mg/kg (n=10), plus cabozantinib 60 mg daily in previously treated (2L+) patients with ccRCC. There were no dose limiting toxicities observed at either dose and at a median follow up of 4.9 months, 92% of patients remained on study. The best overall response rate (ORR, confirmed + unconfirmed) in the ITT population was 46% and 56% in patients dosed with 15 mg/kg (the recommended Phase 2 dose). The best ORR in the biomarker high population was 63%, and 75% in the biomarker high population dosed at 15 mg/kg. The 6-month progression-free survival (PFS) rate was 79% in the ITT population, 77% in the biomarker high population, and 91% in the 15 mg/kg biomarker high group. The company is on track to report additional updated results from the Phase 1b portion of the trial in the second quarter of 2022.

In January 2022, Aravive dosed the first patient in the Phase 2 portion of the Phase 1b/2 clinical trial of batiraxcept in combination with cabozantinib for the treatment of ccRCC. The company is on track to report preliminary results from the Phase 2 portion of the trial in the second quarter of 2022. The Phase 2 portion of the Phase 1b/2 trial of batiraxcept in ccRCC is an open-label study and aims to enroll 55 patients across three parts.
Batiraxcept in Pancreatic Adenocarcinoma: In August 2021, Aravive dosed the first patient in the Phase 1b portion of its Phase 1b/2 trial of batiraxcept in combination with gemcitabine and nab-paclitaxel as a first-line treatment in patients with advanced or metastatic pancreatic adenocarcinoma who are eligible to receive gemcitabine and nab-paclitaxel combination therapy. Enrollment was completed in January 2022. As of February 4, 2022, 13 patients have been treated with 15 mg/kg batiraxcept in combination with gemcitabine and nab-paclitaxel as a first-line treatment. Analysis of all safety data to date demonstrates that batiraxcept has been generally well-tolerated with no unexpected safety signals. The best ORR was 31% and all patients have had at least eight weeks of follow-up (one radiological image). The company is on track to report additional updated data from the Phase 1b portion of the trial in the second quarter of 2022.

Strengthened Balance Sheet: In January 2022, Aravive raised approximately $10.0 million from the sale of a pre-funded warrant to purchase 4,545,455 shares of the company’s common stock to Eshelman Ventures, LLC at a price of $2.20 per share, which was the consolidated closing bid price of the company’s common stock on The Nasdaq Global Select Market on December 31, 2021. Additionally, Fred Eshelman, Pharm.D., was appointed the Executive Chairman of Aravive, having served as the Non-Executive Chairman of the board since April 2020. In March 2022, the Company raised an additional approximately $10.0 million from the sale of common stock and a pre-funded warrant for an aggregate of a combination of 4,850,241 shares of the company’s common stock and pre-funded warrants to a single healthcare-focused institutional investor and Eshelman Ventures, LLC and issued warrants to purchase an additional aggregate of 4,850,241 shares of common stock in a registered direct offering priced at-the-market under Nasdaq rules. Combined, the additional capital infusions strengthen the Company’s financial position and fund operations as currently planned into 1Q’23.

Expanded Board of Directors: In May 2021, the Company appointed John A. Hohneker, M.D., Sigurd C. Kirk, and Peter T.C. Ho, M.D., Ph.D. to its Board. These independent directors strengthen oversight and broaden the Company’s technical capabilities in clinical and business development.
Fourth Quarter and Full Year 2021 Financial Results
Revenue for the three and twelve months ended December 31, 2021 was $1.0 million and $7.4 million, respectively, compared with $5.7 million for the same periods in 2020. Revenues for 2021 and 2020 were derived solely from the Company’s collaboration and license agreement with 3D Medicines, executed in November 2020 to develop and commercialize batiraxcept in oncology indications in Greater China. Revenues represent a portion of initial signing and milestone payments received from 3D Medicines that is recognized at the time of the receipt and a portion of the payments that is deferred and recognized over the PROC trial period.

Total operating expenses for the three and twelve months ended December 31, 2021 were $14.6 million and $48.1 million, respectively compared with $9.7 million and $36.5 million, respectively, for the same periods in 2020. Non-cash stock-based compensation for the three and twelve months ended December 31, 2021 was $0.6 million and $2.3 million, respectively compared with $0.4 million and $2.0 million, respectively, for the same periods in 2020. In addition, during the twelve months ended December 31, 2020, there were non-recurring non-cash charges for impairment of the Company’s right-of-use asset and leasehold improvements of $5.8 million.

For the three and twelve months ended December 31, 2021, Aravive reported a net loss of $13.0 million and $39.2 million, or $0.62 per share and $1.95 per share, respectively compared to a net loss of $4.0 million and $30.5 million, or $0.25 per share and $1.93 per share, respectively, for the same periods in 2020.

Cash Position
As of December 31, 2021, cash and cash equivalents were $59.4 million, compared to $60.5 million as of December 31, 2020. In January 2022, Aravive announced an approximately $10.0 million investment by Eshelman Ventures and in March 2022, Aravive announced an additional approximately $10 million investment by a healthcare-focused institutional investor and Eshelman Ventures, LLC. The combined approximately $20 million in new capital strengthened the Company’s financial position ahead of multiple anticipated clinical milestones throughout 2022 for each of the Company’s three ongoing clinical programs. The Company anticipates that its current cash and cash equivalents will fund operating plans into 1Q’23.

On Armata 31, 2022 Pharmaceuticals, Inc. (NYSE American: ARMP) ("Armata" or the "Company"), a biotechnology company focused on pathogen-specific bacteriophage therapeutics for antibiotic-resistant and difficult-to-treat bacterial infections, reported that, following a vote in favor of the transaction by the Armata shareholders, the Company has completed the closing of the second and final tranche of the Company’s $45 million private placement of its common stock with Innoviva Strategic Opportunities LLC, a wholly-owned subsidiary of Innoviva, Inc. (NASDAQ: INVA) (together, "Innoviva") (Press release, AmpliPhi Biosciences, MAR 31, 2022, View Source [SID1234611283]). In connection with the second closing, Armata issued 5,385,208 common shares and 2,692,604 warrants with an exercise price of $5.00 per share, at a per unit price of $5.00 per unit, in exchange for gross proceeds of approximately $26.9 million. Approximately 99% of the Armata shares represented and voting at the special meeting of shareholders voted in favor of the transaction.

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The Company and Innoviva closed an initial tranche of the investment on February 9, 2022, which raised gross proceeds of approximately $18.1 million through the issuance of 3,614,792 common shares and warrants to purchase an additional 1,807,396 common shares at a strike price of $5.00 per share.

As of March 31, 2022, and following the second closing, Armata has 36,112,299 shares outstanding and warrants exercisable for 21,147,229 shares of common stock.

Armata was represented in the transaction by Thompson Hine LLP, and Ladenburg Thalmann & Co. Inc. provided a fairness opinion.

Willkie Farr & Gallagher LLP represented Innoviva in the transaction.

In addition, Armata Pharmaceuticals, Inc. filed its Annual Report for the year ended December 31, 2021 on Form 10-K with the SEC on March 17, 2022. The audit opinion included in the Company’s Form 10-K contains a going concern explanatory paragraph. This announcement is made pursuant to the disclosure requirements of NYSE American Company Guide Sections 401(h) and 610(b) and does not represent any change or amendment to the Company’s financial statements or to its Annual report on Form 10-K for the year ended December 31, 2021.

This release does not constitute an offer to sell or the solicitation of an offer to buy any security. The shares offered have not been registered under the Securities Act of 1933, as amended, or applicable state securities laws and may not be offered or sold in the United States or any state thereof absent registration under the securities act and applicable state securities laws or an applicable exemption from registration requirements.