On March 31, 2021 ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF) ("ProMIS or the Company"), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, reported its operational and financial results for the year ended December 31, 2020 (Press release, ProMIS Neurosciences, MAR 31, 2021, View Source [SID1234577439]).

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"Despite the many challenges resulting from the COVID-19 pandemic and over the course of the past year, the value of our unique discovery and development platform was further evidenced as ProMIS made considerable progress in expanding its portfolio of opportunities across multiple neurodegenerative diseases", stated Eugene Williams, ProMIS’ Executive Chairman.

Corporate Highlights

During 2020, we received proceeds from the exercise of warrants in the amount of $2,197,245. In March 2021, the Company completed a US$7.0 million (CDN$8.75 million) private placement of debentures. The debentures are convertible into common shares at the option of the holder at a conversion price of US$0.10 per share and accrue interest at 1% per annum, which is payable annually.

In May 2020, ProMIS announced it had identified novel antagonists against the receptor for activated protein kinase C1 (RACK1) that prevent the formation of dysfunctional protein aggregates and act to restore normal function. Evidence indicates that targeting RACK1 is a promising new strategy to address the complex mechanisms involved in the pathogenesis of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS).

In July 2020, the Company entered into two joint venture business arrangements (JV) with BC Neuroimmunology Lab, Inc. (BCNI). The first JV (JV1) will develop and market highly accurate, objective tests for the detection, diagnosis and monitoring of Alzheimer’s disease (AD). JV1 will offer existing blood-based assays for NfL (neurofilament light chain) and P-tau181 (phosphorylated tau181). Further assays will be developed, potentially incorporating our proprietary peptide antigens and tests for additional neurodegenerative diseases. The second JV (JV2) is a collaboration to develop a high-throughput, highly specific serological assay to accurately detect the presence of antibodies against SARS-CoV-2, the virus responsible for the COVID-19 pandemic. The Company and BCNI each own 50% of JV1 and JV2.

In July 2020, the Company announced the voting results of the Corporation’s annual meeting of shareholders held in Vancouver, BC. All of the resolutions announced in the Management Proxy Circular and placed before the Meeting were approved by the shareholders. All Directors were elected, with each nominee receiving more than 75% of the votes cast.

In September 2020, ProMIS announced initiation of a program to construct and test a multivalent peptide vaccine for AD. The critical first steps in vaccine development will be carried out at the University of Saskatchewan’s Vaccine and Infectious Disease Organization-International Vaccine Centre (VIDO-InterVac), a global leader in vaccine research and development.

In November 2020, the Company closed on a special warrant financing. The Company issued 16,219,581 special warrant certificates for gross proceeds of $1,946,350 ($1,636,590 net of issuance costs). Each special warrant will be exercisable, without payment of any additional consideration by the holder, into one common share and one transferrable common share purchase warrant (Warrant).

In March 2021, the special warrant financing converted into 16,219,581 common shares and 16,219,581 warrants. Each warrant entitles the holder to acquire one common share at an exercise price at $0.20 per warrant share for 60 months until November 2025.

People

In October 2020, Dr. David Wishart, Distinguished University Professor in the Departments of Biological Sciences and Computing Science at the University of Alberta, was appointed to the Company’s Scientific Advisory Board.

Financial Results

Annual Results of Operations

The Company’s net loss for the year ended December 31, 2020 was $5,662,392 compared to a net loss of $7,396,259 year ended December 31, 2019. Included in the net loss amount for the year ended December 31, 2020 were non-cash expenses of $430,242 representing share-based compensation, warrant modification and valuation and amortization of an intangible asset, compared to $655,954 for the year ended December 31, 2019. The decrease in the net loss for the year ended December 31, 2020 reflects decreased costs associated with external contract research organizations for internal programs, patent costs, share-based compensation, consultant salaries and associated costs and general corporate expenditures.

Research and development expenses for the year ended December 31, 2020 were $3,183,149, as compared to $4,735,317 in the year ended December 31, 2019. The decrease in research and development expense for the year ended December 31, 2020, compared to the same period ended December 31, 2019 reflects the conservation of existing cash resources and decreased spending on external contract research organizations for internal programs, reduced patent expense, share-based compensation, contracted research salaries and associated costs and external consulting expense.

General and administrative expenses for the year ended December 31, 2020 were $2,481,030, as compared to $2,662,144 in the year ended December 31, 2019. The decrease for the years ended December 31,2020, compared to the same period in 2019, is primarily attributable to a reduction in consulting and professional fees and a decrease in foreign exchange losses offset by warrant modification and valuation expense.

Outlook

Following on from the completion of a US$7M (CDN$8.75M) financing in March 2021, we plan to advance progress toward our priority programs:

Advance the PMN310 monoclonal antibody, our potential "best in class" next generation Alzheimer’s treatment, into clinical testing;
Enhance our partnering prospects by allowing us to invest in additional validation data for key R&D programs;
Expand our portfolio of products and intellectual property into new target areas, using our proprietary discovery platform;
Advance our diagnostic programs in partnership with BCNI;
Achieve NASDAQ listing;
Expand our Board of Directors and our management team, to support a growing and ambitious scope of activity.

On March 31, 2021 NexImmune, Inc. (Nasdaq: NEXI), a clinical-stage biotechnology company developing a novel approach to immunotherapy designed to orchestrate a targeted immune response by directing the function of antigen-specific T cells, reported its financial results for 2020 and highlighted recent corporate accomplishments (Press release, NexImmune, MAR 31, 2021, View Source [SID1234577438]).

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"2020 was a transformational year for NexImmune," said Scott Carmer, Chief Executive Officer. "Our first two programs, NEXI-001 and NEXI-002, entered Phase 1/2 clinical trials and successfully enrolled patients throughout the year despite the COVID-19 pandemic. We were very pleased to have initial data from our first cohort of patients in the NEXI-001 trial accepted for oral presentation at the ASH (Free ASH Whitepaper) annual meeting in December. In addition, we strengthened our management team, scientific advisory board and Board of Directors with the addition of several industry-leading scientists and executives. All of these advancements contributed to our successful IPO in February, 2021."

Mr. Carmer added, "With a strong cash position from the completion of our IPO, we are focused on driving our current clinical trials toward completion, and to accelerating ongoing development of our AIM ACT product pipeline and platform technology. This work will concentrate on clinical programs targeting solid tumors and IND-enabling pre-clinical experiments to support the initial application of our ‘AIM Injectable’ formulation, respectively. In developing our proprietary AIM nanoparticle technology, our mission is to transform cell-mediated immunotherapy for the benefit of patients facing a number of difficult-to-treat diseases."

Select corporate highlights

On February 17, 2021, NexImmune completed a successful IPO and raised gross proceeds of $126M. The IPO was oversubscribed and priced at the top of the range.

During January 2021, NexImmune announced the appointments of Robert Knight, MD as Chief Medical Officer; Jerome Zeldis, MD, PhD as Executive Vice President, Research and Devopment; Jeffery Weber, MD, PhD as Chief Scientific Advisor and Scientific Advisory Board Chair; and Grant Verstandig as a member of the Board of Directors.

On December 7, 2020, lead investigators for the Company’s ongoing Phase 1/2 clinical trial evaluating NEXI-001 provided an oral presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition that highlighted initial results from the first five patients treated. Initial data demonstrated early signs of safety, tolerability and robust immune responses in acute myeloid leukemia (AML) patients with relapsed disease after allogeneic hematopoietic stem cell transplantation (allo-HSCT).

On October 6, 2020, NexImmmune announced dosing of the first patient in its Phase 1/2 trial of NEXI-002 for the treatment of patients with relapsed and/or refractory multiple myeloma that had failed at least three lines of prior therapy.

NEXI-001 and NEXI-002 are both in Phase 1/2 clincal trials. The company expects to share preliminary data from the initial safety cohorts of each trial at a conference in Q2 2021, with more complete results for each trial at the end of Q4 2021.

Select financial highlights

Cash and cash equivalents for the company as of December 31, 2020 were $5.0M, compared to $9.1M at December 31, 2019. Based upon its current operating plans and cash and cash equivalents, including the net proceeds from the IPO, the Company expects to have sufficient capital to fund its operating expenses and capital expenditure requirements through the second quarter of 2022.

Research and development expenses were $17.8M in 2020, compared to $15.2M in 2019. The increase in R&D expenses were mainly attributable to costs for the two clinical trials as well as personnel-related expenses driven by increased headcount, offset partially by reduced preclinical and regulatory-related spending.

General and administrative expenses were $10.0M and $5.7M in 2020 and 2019, respectively. The increase was due primarily to increases in headcount and fees related to professional and consulting services.

Net loss in 2020 was $29.9M, compared to $20.5M in 2019.

On March 31, 2021 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on acquiring, developing and commercializing or monetizing promising biopharmaceutical products and product candidates cost-effectively, reported financial results and recent corporate highlights for the full-year ended December 31, 2020 (Press release, Fortress Biotech, MAR 31, 2021, View Source [SID1234577437]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We are delighted with our continued progress, and achievement of significant milestones throughout 2020 and in recent months. Notably, our partner company, Cyprium Therapeutics ("Cyprium"), and Sentynl Therapeutics, a wholly-owned subsidiary of the Zydus Group, signed a Development and Asset Purchase Agreement for CUTX-101 for the treatment of Menkes disease, in February 2021. Also, our full-year 28% revenue growth during the pandemic shows the resilience of the Fortress business model. Since the company’s management took over in 2014, Fortress and our partner companies have built a growing portfolio of six marketed dermatologic pharmaceutical products and more than 25 product candidates in our pipeline across multiple therapeutic categories, including oncology, gene therapy, dermatology, and rare diseases. We are on track to submit our second New Drug Application ("NDA") to the U.S. Food and Drug Administration ("FDA") later this year."

Dr. Rosenwald continued, "As we look ahead to 2021, Fortress expects to deliver further top-line growth through a diversified, long-term revenue stream, and we and our partner companies anticipate multiple key value-creating milestones throughout the year. We have four product candidates in five ongoing pivotal clinical trials, with multiple other earlier-stage clinical trials progressing as well. Additionally, we intend to continue to in-license promising drugs and drug candidates, and to seek partners for late-stage programs to maximize each opportunity to its full potential. We continue to demonstrate the ability of our business model to generate meaningful value creation opportunities with the potential to produce life-changing therapies for people in need."

2020 and Recent Corporate Highlights1:

Marketed Dermatology Products

·Our six dermatology products are marketed by our partner company, Journey Medical Corporation ("Journey").

1 Includes product candidates in development at Fortress, majority-owned and controlled partners and partners in which Fortress holds significant minority ownership positions. As used herein, the words "we", "us" and "our" may refer to Fortress individually or together with our affiliates and partners, as dictated by context.

·Our products generated net revenues of $44.5 million for full-year 2020, compared to full-year 2019 net revenues of $34.9 million, representing growth of 28%. Our products generated $13.7 million in revenues in the fourth quarter of 2020, compared to $11.1 million in the fourth quarter of 2019, representing growth of 23%. While we generated revenue growth year-over-year in 2020, sales of our products were impacted by the COVID-19 pandemic, which caused a temporary supply shortage and impacted the ability of our sales force to make in person calls, due to states slowly re-opening.
· We in-licensed and recently launched our sixth prescription dermatology product.
·We intend to launch up to two additional new prescription products this year and expect to generate further sales growth in 2021.
·We currently have 42 sales representatives dedicated to the dermatology product portfolio and we expect that number to continue to grow this year.

CUTX-101 (Copper Histidinate for Menkes disease)

·In January 2020, the FDA granted Rare Pediatric Disease Designation to CUTX-101 for the treatment of Menkes disease.
·In June 2020, we announced the publication of a study, "Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome Aggregation Database (gnomAD)," in Molecular Genetics and Metabolism Reports. Assuming Hardy-Weinberg genetic equilibrium, the allelic frequency of loss-of-function variants suggests a minimum birth prevalence for Menkes disease of 1 in 34,810 males, higher than previously recognized. If likely pathogenic missense variants are included, the estimated birth prevalence could potentially be as high as 1 in 8,664 live male births. The study can be accessed here.
·In July 2020, we announced the publication of a study, "Targeted Next Generation Sequencing for Newborn Screening of Menkes Disease" in Molecular Genetics and Metabolism Reports. The study assessed the analytic validity of an ATP7A targeted next generation DNA sequencing assay as a potential newborn screen for Menkes disease. The study can be accessed here.
·In July 2020, we announced that the European Medicines Agency ("EMA") Committee for Orphan Medicinal Products issued a positive opinion on our application for Orphan Drug Designation for CUTX-101 for the treatment of Menkes disease. In August 2020, the European Commission granted Orphan Drug Designation to CUTX-101. EMA Orphan Drug Designation provides companies with certain benefits and incentives, including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees and 10 years of market exclusivity.
·In August 2020, we reported positive top-line clinical efficacy results for CUTX-101. The study demonstrated statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (ET) with CUTX-101, compared to an untreated historical control (HC) cohort, with a nearly 80% reduction in the risk of death (Hazard Ratio = 0.21, p<0.0001). Median survival for the ET cohort was 14.8 years (177.1 months), compared to 1.3 years (15.9 months) for the untreated HC cohort.
·In September 2020, we raised net proceeds of approximately $7.1 million in a private offering of Cyprium’s 9.375% Series A Cumulative Redeemable Perpetual Preferred Stock.
·In December 2020, the FDA granted Breakthrough Therapy Designation to CUTX-101 for the treatment of Menkes disease.
·In February 2021, our partner company, Cyprium, and Sentynl Therapeutics, a wholly-owned subsidiary of the Zydus Group, signed a Development and Asset Purchase Agreement for CUTX-101 for the treatment of Menkes disease. Under the terms of the agreement, Cyprium received $8 million upfront to fund the development of CUTX-101 and could receive up to $12 million in regulatory milestone payments through NDA approval, and is eligible to receive sales milestones plus royalties. Royalties start from mid-single digits, scaling up to 25% on sales exceeding $100 million annually. Cyprium will retain 100% ownership over any FDA priority review voucher that may be issued at NDA approval for CUTX-101.
·We intend to begin the rolling submission of the NDA for CUTX-101 to the FDA in the second half of 2021.
·CUTX-101 is currently in development at our partner company, Cyprium Therapeutics, Inc.

CAEL-101 (Light Chain Fibril-reactive Monoclonal Antibody for AL Amyloidosis)

·In 2020, Caelum Biosciences, Inc.’s ("Caelum") Phase 2 dose selection clinical trial of CAEL-101, a light chain fibril-reactive monoclonal antibody for the treatment of AL amyloidosis, met its primary objective, supporting the initiation of two parallel Phase 3 studies that will enroll approximately 370 AL amyloidosis patients.
·In September 2020, Caelum announced the initiation of two Phase 3 studies of CAEL-101 for AL amyloidosis.
·Also in September 2020, positive long-term Phase 1a/1b data were presented at the International Symposium on Amyloidosis (ISA) 2020. The data demonstrated prolonged overall survival (78% at 37 months) and durable organ response.
·In December 2020, CAEL-101 Phase 2 data were selected for oral and poster presentations at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which was held virtually in December 2020. Links to the abstracts can be found here: oral presentation and poster presentation.
·Caelum formed a collaboration with Alexion Pharmaceuticals, Inc. in 2019, which includes an option to acquire Caelum. AstraZeneca announced the execution of a definitive agreement to purchase Alexion Pharmaceuticals, Inc.; in event of the closing of such transaction, the timeline for a potential exercise of the option to purchase Caelum will be accelerated to six months following the date of acquisition closing.
·CAEL-101 is currently in development at Caelum Biosciences, Inc., a company founded by Fortress in 2017 and in which Fortress maintains a minority position.

Cosibelimab (formerly CK-301, an anti-PD-L1 antibody)

·In January 2020, we announced confirmation of the registration path for cosibelimab in metastatic cutaneous squamous cell carcinoma ("mCSCC"). FDA feedback supports the plan to submit a Biologics License Application ("BLA") based on data from the ongoing Phase 1 clinical trial.
·In April 2020, we announced that the U.S. Patent and Trademark Office issued a composition of matter patent for cosibelimab. U.S. Patent No. 10,590,199 specifically covers the antibody, cosibelimab, or a fragment thereof, providing protection through at least May 2038, exclusive of any additional patent-term extensions that might become available.
·In September 2020, at the European Society for Medical Oncology Virtual Congress 2020, we announced updated interim results from the ongoing global, open-label, multicohort, Phase 1 clinical trial of cosibelimab in patients with advanced cancers, including the registration-enabling cohort of patients with mCSCC. Cosibelimab demonstrated a 51.4% objective response rate ("ORR") and 13.5% complete response rate, which is nearly double the complete response rate observed at the time of previous analysis.
·The registration-enabling study in mCSCC is approximately 90% enrolled and we are on track to report full top-line results in the second half of 2021. With a potentially favorable safety profile and a plan to commercialize at a substantially lower price, we believe cosibelimab has the potential to be a market disruptive product in the $25 billion and growing PD-(L)1 class.
·In November 2020, we announced the expansion of a long-term manufacturing partnership for cosibelimab with Samsung Biologics. Building upon an existing contract manufacturing agreement entered into in 2017, Samsung Biologics will provide additional commercial-scale drug substance manufacturing for cosibelimab.
·Also in November 2020, we announced updated interim results from the ongoing global, open-label, multicohort Phase 1 clinical trial of cosibelimab in patients with advanced cancers, including a cohort of patients with previously untreated high PD-L1 expressing advanced non-small cell lung cancer ("NSCLC"). The updated interim results were presented in a poster presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting. Cosibelimab demonstrated a 44.0% objective response rate and 10.3-month median progression-free survival in the NSCLC cohort. A Phase 3 registration-enabling trial is planned to begin in first-line metastatic NSCLC in mid-2021.
·Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc. ("Checkpoint").

CK-101 (Third-generation Epidermal Growth Factor Receptor ("EGFR") Inhibitor)

·In November 2020, Checkpoint’s collaboration partner in Asia for CK-101, Neupharma Inc., initiated a Phase 3, registration-enabling study of CK-101 in first-line, EGFR mutation-positive locally advanced or metastatic NSCLC. We plan to meet with the FDA to discuss the ongoing Phase 3 study design and its potential use, upon a successful study, to support a new drug application submission in the United States.
·CK-101 is currently in development at our partner company, Checkpoint.

MB-107 and MB-207 (Lentiviral Gene Therapies for XSCIDIn April 2020, we announced that the EMA granted Advanced Therapy Medicinal Product classification to MB-107, a lentiviral gene therapy for the treatment of newly diagnosed infants with X-linked severe combined immunodeficiency ("XSCID"), also known as bubble boy disease.
·In the second quarter of 2020, we submitted an IND application with the FDA to initiate a multi-center pivotal Phase 2 trial of MB-107 in newly diagnosed infants with XSCID who are under the age of two. The trial is expected to enroll 10 patients who, together with patients enrolled in the current multicenter trial led by St. Jude Children’s Research Hospital, will be compared with 25 matched historical control patients who have undergone hematopoietic stem cell transplant ("HSCT"). The primary efficacy endpoint will be event-free survival. On January 28, 2021, the FDA removed a CMC hold on the MB-107 Phase 2 clinical trial IND application after reviewing a comprehensive CMC package that was submitted in December 2020. We expect to enroll the first patient in this pivotal multicenter trial in the second quarter of 2021 and are targeting top-line data from the trial in the second half of 2022.
·We further expect to file an IND in the second quarter of 2021 for a pivotal multi-center Phase 2 clinical trial of MB-207, our lentiviral gene therapy in previously transplanted XSCID patients who received prior treatment with HSCT and for whom re-treatment is indicated. We anticipate enrolling 20 patients and we are targeting topline data for this trial in the first half of 2023.
·In August 2020, we announced that the FDA granted Rare Pediatric Disease Designations to MB-107 for the treatment of XSCID in newly diagnosed infants and to MB-207 for the treatment of XSCID in patients who were previously treated with HSCT and for whom re-treatment is indicated.
·In September 2020, we announced that the FDA granted Orphan Drug Designations to MB-107 for the treatment of XSCID in newly diagnosed infants and to MB-207 for the treatment of XSCID in patients who were previously treated with HSCT and for whom re-treatment is indicated.
·In October 2020, we in-licensed LentiBOOST technology from SIRION Biotech GmbH for the development of MB-207.
·In November 2020, we signed an agreement with Minaris Regenerative Medicine GmbH to enable technology transfer and GMP clinical manufacturing of the MB-107 lentiviral gene therapy program for the treatment of XSCID in Europe.
·Also in November 2020, we announced that the European Commission issued a positive opinion on our application for Orphan Drug Designation for the MB-107 lentiviral gene therapy for the treatment of XSCID.
·MB-107 and MB-207 are currently in development at our partner company, Mustang Bio, Inc. ("Mustang Bio").

MB-106 (CD20-targeted CAR T Cell Therapy)

·In February 2020, we announced that the first subject treated with the modified MB-106 (CD20-targeted, autologous CAR T cell therapy) manufacturing process, developed in a collaboration between Mustang Bio and the Fred Hutchinson Cancer Research Center ("Fred Hutch"), achieved a complete response at the lowest starting dose in an ongoing Phase 1/2 clinical trial. The trial is evaluating the safety and efficacy of MB-106 in subjects with relapsed or refractory B-cell non-Hodgkin lymphomas ("NHL") and chronic lymphocytic leukemia ("CLL").
·In December 2020, we announced positive interim Phase 1/2 data on MB-106 for patients with relapsed or refractory B-cell non-Hodgkin lymphomas, which were presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Data presented showed a favorable safety profile and clinical activity with an 89% ORR and 44% complete response rate over 4 dose levels in 9 patients treated with the modified cell manufacturing process.
·In the second quarter of 2021, we expect to announce follow up data from the Fred Hutch trial in NHL and initial data in CLL.
·MB-106 is currently in development at our partner company, Mustang Bio.

MB-102 (CD123-targeted CAR T Cell Therapy)

·In October 2020, we announced that the first patient was dosed in a Mustang Bio-sponsored, open-label, multicenter Phase 1/2 clinical trial to evaluate the safety and efficacy of MB-102 (CD123-targeted CAR T cell therapy) in patients with relapsed or refractory blastic plasmacytoid dendritic cell neoplasm.
·MB-102 is currently in development at our partner company, Mustang Bio.

MB-101 (IL13Rα2-targeted CAR T Cell Therapy)

·In December 2020, we announced that a Phase 1 single-center, two-arm clinical trial was initiated to establish the safety and feasibility of administering MB-101 to patients with leptomeningeal brain tumors (e.g., glioblastoma, ependymoma or medulloblastoma).
·MB-101 is currently in development at our partner company, Mustang Bio.

MB-105 (PSCA-targeted CAR T Cell Therapy)

·In October 2020, we announced that initial Phase 1 data on MB-105, a PSCA-targeted CAR T administered systemically to patients with PSCA-positive metastatic castration-resistant prostate cancer (mCRPC), were presented by City of Hope at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat. A 73-year-old male patient with PSCA-positive mCRPC was treated with MB-105 and lymphodepletion (a standard CAR T pre-conditioning regimen) after failing eight prior therapies. On day 28 of the patient’s treatment, MB-105 demonstrated a 94% reduction in prostate-specific antigen, near complete reduction of measurable soft tissue metastasis by computerized tomography, and improvement in bone metastases by magnetic resonance imaging.
·MB-105 is currently in development at our partner company, Mustang Bio.

MB-104 (CS1-targeted CAR T Cell Therapy In May 2020, City of Hope presented two posters pertaining to MB-104, an innovative CS1 CAR T cell therapy, at the virtual 23rd Annual Meeting of the American Society of Gene & Cell Therapy.
·MB-104 is currently in development at our partner company, Mustang Bio.

IV Tramadol

·In October 2020, Avenue Therapeutics ("Avenue") announced that it had received a Complete Response Letter ("CRL") from the FDA regarding Avenue’s NDA for IV tramadol. The FDA held a Type A meeting with Avenue in November 2020 to discuss the issues outlined in the CRL. On February 12, 2021, Avenue resubmitted its NDA to the FDA for IV tramadol. The NDA resubmission followed the receipt of the official minutes from Avenue’s Type A meeting with the FDA. The NDA resubmission included revised language relating to the proposed product label and a report relating to terminal sterilization validation. On February 26, 2021, Avenue received an acknowledgement letter from the FDA that Avenue’s resubmission of its NDA is a complete, class 1 response to the CRL, and a PDUFA goal date was set for April 12, 2021.
·Also in October 2020, Invagen Pharmaceuticals Inc. ("InvaGen") communicated to Avenue that it believes a Material Adverse Event (as defined in the Stock Purchase and Merger Agreement entered into on November 12, 2018 between Avenue, InvaGen and Madison Pharmaceuticals Inc., a newly formed, wholly-owned subsidiary of InvaGen) has occurred due to the impact of the COVID-19 pandemic on potential commercialization and projected sales of IV tramadol. Additionally, in connection with the resubmission of Avenue’s NDA in February 2021, InvaGen communicated to Avenue that it believes the proposed label for IV tramadol would also constitute a Material Adverse Event on the purported basis that the proposed label under certain circumstances would make the product commercially unviable, and in addition that the indication that the FDA approves may fail to satisfy a condition precedent to InvaGen’s obligation to consummate the second stage closing of the Avenue SPMA. While Avenue disagrees with InvaGen’s assertions, it is possible InvaGen could attempt to avoid its obligation to consummate the merger, terminate the Stock Purchase and Merger Agreement, and/or pursue monetary claims against Avenue and/or Fortress.
·IV tramadol is currently in development at our partner company, Avenue.

ONCOlogues (proprietary platform technology using PNA oligonucleotides)

·In May 2020, we entered into an exclusive worldwide licensing agreement with Columbia University to develop novel oligonucleotides for the treatment of genetically driven cancers. The proprietary platform produces oligomers, known as "ONCOlogues," which are capable of binding gene sequences 1,000 times more effectively than complementary native DNA.
·In addition, we are exploring the potential of the platform to treat novel coronaviruses, such as COVID-19. We are also evaluating the platform to treat Huntington’s disease and myotonic dystrophy.
·The ONCOlogues platform is currently in development at our partner company, Oncogenuity, Inc.

General Corporate

·From February to August 2020, we closed on a gross total of approximately $39 million in underwritten public offerings of our 9.375% Series A Cumulative Redeemable Perpetual Preferred Stock (FBIOP).
·In June 2020, Fortress and Avenue were added to the Russell 3000 index.
·Also in August 2020, we announced a $60 million long-term debt refinancing agreement with Oaktree Capital Management, replacing $60 million of existing debt that was due over the subsequent seven quarters, with the debt now due in August 2025.
· November 2020, Fortress ranked in Deloitte’s Technology Fast 500 for the second year in a row. Deloitte’s Technology Fast 500 is an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors. Fortress’ 874 percent revenue growth based on the increase in net product sales from 2016 to 2019 secured its spot in the rankings.

Financial Results:

To assist our stockholders in understanding our company, we have prepared non-GAAP financial results for the three months and twelve months ended December 31, 2020 and 2019. These results exclude the operations of our three public partner companies: Avenue Therapeutics, Inc. ("Avenue"), Checkpoint Therapeutics, Inc. ("Checkpoint") and Mustang Bio, Inc. ("Mustang"), as well as any one-time, non-recurring, non-cash transactions, such as the gain of $18.4 million we recorded in the first quarter of 2019 resulting from the deconsolidation of Caelum Biosciences, Inc. ("Caelum"). The goal in providing these non-GAAP financial metrics is to highlight the financial results of Fortress’ core operations, which are comprised of our commercial-stage business, our privately held development-stage entities, as well as our business development and finance functions.

·As of December 31, 2020, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit) and restricted cash totaled $235.0 million, compared to $220.0 million as of September 30, 2020, and $153.4 million as of December 31, 2019, an increase of $15.0 million for the fourth quarter and an increase of $81.6 million for the full year.
·On a GAAP basis, Fortress’ net revenue totaled $45.6 million for the full year ended December 31, 2020, which included $44.5 million in net revenue generated from our marketed dermatology products. This compares to net revenue totaling $36.6 million for the full year ended 2019, which included $34.9 million in net revenue generated from our marketed dermatology products. Fortress expects to achieve year-over-year revenue growth in 2021; however, it continues to monitor the spread of COVID-19 and assess the impact it may have on 2021 revenues.

·On a GAAP basis, consolidated research and development expenses including license acquisitions totaled $64.1 million for the full year ended December 31, 2020, compared to $81.3 million for the full year ended December 31, 2019. On a non-GAAP basis, research and development expenses including research and development license acquisitions totaled $10.0 million for the full year ended December 31, 2020, compared to $11.2 million for the full year ended December 31, 2019.
·On a GAAP basis, consolidated selling, general and administrative expenses were $61.2 million for the full year ended December 31, 2020, compared to $55.6 million for the full year ended December 31, 2019. On a non-GAAP basis, selling, general and administrative expenses were $45.5 million, of which $22.1 million is attributed to Journey, for the full year ended December 31, 2020, compared to $38.9 million, of which $19.7 million is attributed to Journey, for the full year ended December 31, 2019.
·On a GAAP basis, consolidated net loss attributable to common stockholders was $46.5 million, or $0.65 per share, for the full year ended December 31, 2020, compared to net loss attributable to common stockholders of $40.0 million, or $0.73 per share for the full year ended December 31, 2019.
·Fortress’ non-GAAP loss attributable to common stockholders was $13.3 million, or $0.18 per share, for the full year ended December 31, 2020, compared to Fortress’ non-GAAP loss attributable to common stockholders of $14.7 million, or $0.27 per share, for the full year ended December 31, 2019.

Use of Non-GAAP Measures:

In addition to the GAAP financial measures as presented in our Form 10-K filed with the Securities and Exchange Commission ("SEC") on March 31, 2021, the Company, in this press release, has included certain non-GAAP measurements. The non-GAAP net loss attributable to common stockholders is defined by the Company as GAAP net loss attributable to common stockholders, less net losses attributable to common stockholders from our public partner companies Avenue, Checkpoint, and Mustang, as well as Caelum. In addition, the Company has also provided a Fortress non-GAAP loss attributable to common stockholders which is a modified EBITDA calculation that starts with the non-GAAP loss attributable to common stockholders and removes stock-based compensation expense, non-cash interest expense, amortization of licenses and debt discount, changes in fair values of investment, changes in fair value of derivative liability, and depreciation expense.

Management believes these non-GAAP measures provide meaningful supplemental information regarding the Company’s performance because (i) it allows for greater transparency with respect to key measures used by management in its financial and operational decision-making; (ii) it excludes the impact of non-cash or, when specified, non-recurring items that are not directly attributable to the Company’s core operating performance and that may obscure trends in the Company’s core operating performance; and (iii) it is used by institutional investors and the analyst community to help analyze the Company’s results. However, non-GAAP loss attributable to common stockholders and any other non-GAAP financial measures should be considered as a supplement to, and not as a substitute for, or superior to, the corresponding measures calculated in accordance with GAAP. Further, non-GAAP financial measures used by the Company and the manner in which they are calculated may differ from the non-GAAP financial measures or the calculations of the same non-GAAP financial measures used by other companies, including the Company’s competitors.
1.Avenue net loss from their external SEC report for the years ended December 31, 2020 and 2019 of $5.2 million and $25.9 million, respectively, net of non-controlling interest of $4.0 million and $19.0 million, respectively.

2.Checkpoint net loss from their external SEC report of $23.1 million net of non-controlling interest of $13.3 million, PIK dividend of $4.6 million, MSA fee to Fortress of $0.5 million and financing fee to Fortress of $0.9 million for the year ended December 31, 2020; and net loss of $24.7 million net of non-controlling interest of $14.7 million, PIK dividend of $2.5 million, MSA fee to Fortress of $0.5 million and financing fee to Fortress of $0.7 million for the year ended December 31, 2019.

3.Mustang net loss from their external SEC report of $60.0 million net of non-controlling interest of $36.4 million, PIK dividend of $7.6 million, MSA fee to Fortress of $0.5 million and financing fee to Fortress of $2.4 million for the year ended December 31, 2020; and net loss of $46.4 million net of non-controlling interest of $25.7 million, PIK dividend of $4.9 million, MSA fee to Fortress of $0.5 million and financing fee to Fortress of $1.7 million for the year ended December 31, 2019.

4.Increase in fair value of investment in Caelum Biosciences for the year ended December 31, 2020.

5.Related to issuance of Cyprium warrant in connection with 2018 Venture Debt for the year ended December 31, 2020.

1.Includes Research and development expense and Research and development – licenses acquired expense for the years ended December 31, 2020 and December 31, 2019, respectively.

2.Excludes $4.6 million and $2.5 million PIK Dividend for the years ended December 31, 2020 and 2019, respectively.

3.Excludes $0.3 million for Fortress MSA and $7.6 million PIK Dividend for the year ended December 31, 2020; excludes $0.3 million for Fortress MSA and $4.9 million PIK dividend for the year ended December 31, 2019.

4.Excludes $0.5 million of Fortress MSA expense and $0.9 million Fortress financing fee for the year ended December 31, 2020; and $0.5 million of Fortress MSA expense and $0.7 million Fortress financing fee for the year ended December 31, 2019.

5.Excludes $0.3 million of Fortress MSA expense and $2.4 million Fortress financing fee for the year ended December 31, 2020; and $0.3 million of Fortress MSA expense and $1.7 million Fortress financing fee for the year ended December 31, 2019.

On March 31, 2021 Bolt Biotherapeutics, Inc. (NASDAQ: BOLT) a clinical-stage biotechnology company pioneering a new class of immuno-oncology agents that combine the targeting precision of antibodies with the power of both the innate and adaptive immune systems, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided an update on recent business highlights (Press release, Bolt Biotherapeutics, MAR 31, 2021, View Source [SID1234577436]).

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"Our upsized Initial Public Offering, which we completed in February 2021, leaves us in a strong financial position to execute on our vision of developing this new class of immuno-oncology products to help patients. We continue to enroll patients in the dose escalation part of our Phase 1/2 trial for our lead candidate, BDC-1001, for the treatment of patients with HER2-expressing solid tumors. We reported preliminary clinical results from an initial 20 patients at a data cutoff of January 29, 2021, which demonstrated 4 patients with stable disease and one patient with a confirmed partial response. We’re looking forward to completing the dose escalation and initiating both the monotherapy Phase 2 dose expansions part and the combination studies with an anti-PD-1 antibody part later in 2021," said Randall C. Schatzman, Ph.D., Chief Executive Officer of Bolt. "We continue to progress our broader pipeline of targeted immunotherapies derived from our Boltbody ISAC platform, a novel technology that can be applied across a diverse range of tumor targets and has the potential to enable cancer patients to generate immunological memory against their own tumors. We plan to advance our second Boltbody ISAC BDC-2034, which targets the cancer antigen CEA, into the clinic in 2022."

Recent Business Highlights and Anticipated Milestones

Completed upsized Initial Public Offering in February 2021 – In February 2021, Bolt completed its Initial Public Offering (IPO) of 13,225,000 shares of common stock, inclusive of the full exercise by the underwriters of their option to purchase 1,725,000 shares, at a public offering price of $20.00 per share. Gross proceeds from the IPO were $264.5 million and net proceeds from the offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $241.7 million.

Reported preliminary clinical results from the first 20 patients from the ongoing Phase 1/2 trial of lead candidate BDC-1001 for the treatment of patients with HER2-expressing solid tumors –BDC-1001 is a human epidermal growth factor receptor 2, or HER2, Boltbody Immune-Stimulating Antibody Conjugate (ISAC) comprised of a HER2-targeting biosimilar of trastuzumab conjugated to one of Bolt’s proprietary TLR7/8 agonists, for the treatment of patients with HER2-expressing solid tumors, including HER2-low tumors. A Phase 1/2 study evaluating BDC-1001 in patients with HER2-expressing cancers, which includes patients with breast and gastric cancers that are refractory to Herceptin and Kadcyla, as well as cancers for which no HER2-targeting therapies have yet been approved, is ongoing. Bolt is currently enrolling patients in the dose escalation portion of the trial. As of January 29, 2021, Bolt had treated 20 patients and BDC-1001 appeared to be well tolerated with mild to moderate adverse events and no dose-limiting toxicities or drug-related serious adverse events were observed. Clinical activity was seen in the form of stable disease, reductions in tumor volume including a confirmed partial response and increases in pharmacodynamic markers that Bolt believes are consistent with its proposed mechanism of action. Later this year, Bolt plans to advance to part 3 of the trial, monotherapy Phase 2 dose expansion cohorts, and part 2, BDC-1001 dose escalation in combination with an anti-PD-1 antibody.

Strengthened Board of Directors with appointment of Kathleen LaPorte – In January 2021, Kathleen LaPorte joined the Board of Directors as Chair of Bolt’s Audit Committee. Ms. LaPorte has more than 30 years of experience in building and operating private and public biotech companies as former chief executive officer of Nodality Inc. and a founding partner of New Leaf Venture Partners. The addition increased Bolt’s board to eight members.

Published data in Nature Cancer highlighting proof of concept for Boltbody ISAC platform to eliminate tumors following systemic administration – In December 2020, Bolt announced the publication of a manuscript in Nature Cancer highlighting the development and use of Boltbody ISACs for the treatment of HER2-expressing tumors in preclinical models. The data indicate that Boltbody ISACs activate the innate and adaptive immune systems, ultimately resulting in complete tumor regressions in multiple tumor models and durable anti-tumor immunity. ISAC-mediated immunological memory extended beyond the target antigen as ISAC-treated mice were protected from re-challenge with the parental tumor lacking HER2 expression.

Completed an oversubscribed $93.5 million Series C financing with notable crossover investors – In July 2020 and January 2021, the Company raised funding to support BDC-1001 and continued development of the Boltbody ISAC platform and Bolt’s pipeline of immuno-oncology programs. Sofinnova Investments led the investment, which included notable crossover investors RA Capital Management, Surveyor Capital (a Citadel Company), Rock Springs Capital and Samsara BioCapital.
Upcoming Events

Bolt’s Chief Scientific Officer David Dornan, Ph.D. will present a talk on BDC-1001 as part of "New Drugs on the Horizon: Part 2" at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting on Saturday, April 10, 2021, from 4:00 PM – 5:45 PM ET.

Manish R. Sharma, M.D. of START Midwest, a principal investigator in Bolt’s ongoing BDC-1001 Phase 1/2 trial, will present a trial-in-progress e-poster entitled "CT218 – Phase 1/2 study of a novel HER2 targeting TLR7/8 immune-stimulating antibody conjugate (ISAC), BDC-1001" in the session "PO.CT08.01 – Phase I Clinical Trials in Progress" at AACR (Free AACR Whitepaper) on Saturday, April 10, 2021, from 8:30 AM – 11:59 PM ET.

Bolt’s CEO, Randall Schatzman, Ph.D., will present a corporate overview at the virtual 20th Annual Needham Virtual Healthcare Conference on Thursday, April 15, 2021 at 10:15 AM ET.
Fourth Quarter and Full Year 2020 Financial Results

Cash Position – Cash, cash equivalents, and marketable securities were $22.8 million as of December 31, 2020, as compared to $34.8 million as of December 31, 2019. Total cash, cash equivalents, and marketable securities at December 31, 2020 does not include total net proceeds of approximately $293.6 million from Bolt’s C-2 convertible preferred stock offering in January 2021 and its IPO in February 2021. Bolt expects its cash balance to fund operations into 2023, through achievement of key milestone for the BDC-1001 and BDC-2034 programs.

Research and Development (R&D) Expenses – R&D expenses were $14.9 million for the quarter and $40.4 million for the full year ended December 31, 2020, compared to $7.4 million and $26.0 million for the same quarter and year in 2019. The increase in R&D spending from 2019 to 2020 is due primarily to the 2020 start of Bolt’s Phase 1/2 clinical trial for BDC-1001, increased manufacturing of BDC-1001 to support the clinical trial and additional hiring.

General and Administrative (G&A) Expenses – G&A expenses were $2.1 million for the quarter and $9.1 million for the full year ended December 31, 2020, compared to $2.1 million and $5.2 million for the same quarter and year in 2019. The increase in G&A spending from 2019 to 2020 is due primarily to an increase in accounting and legal fees associated with IPO preparation and additional hiring to support operations as a public company.

Loss from Operations – Loss from operations was $16.9 million for the quarter and $49.2 million for the full year ended December 31, 2020 compared to $9.5 million and $31.0 million for the same quarter and year in 2019.

On March 31, 2021 Notable Labs Inc., a leader in technology-powered life science with a proprietary platform for predicting patient outcomes and accelerating precision drug development, reported the appointment of Thomas A. Bock, MD, MBA, as Chief Executive Officer (Press release, Notable Labs, MAR 31, 2021, View Source [SID1234577435]). Dr. Bock joined Notable’s Board of Directors in August 2020 and now succeeds Laurie Heilmann as the company’s executive leader, with Notable Founder Matt De Silva remaining as Chairman of the Board.

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"Thomas has built four successful life science organizations with tremendous impact for patients and investors, achieving leadership in novel and fast-growing markets including cancer precision medicine and ultra-rare disorders. His dual experience in life science and technology makes him the right leader to champion Notable in its technology-powered advancement of cancer medicine and precision oncology therapies," said De Silva. "Notable’s integration of cutting-edge predictive technology and life science therapeutic programs not only opens strategic commercial opportunities, but also creates a unique workplace blending top talent across tech and life science."

Dr. Bock founded and served as Chief Executive Officer of HeritX Inc., a pioneer in cancer prevention through pre-cancer vaccines, immunoprevention, and gene repair, with the largest cancer prevention pipeline in the industry. Previously, he served as Senior Vice President Medical Affairs on the executive management team of Alexion Pharmaceuticals, building a start-up into a global frontrunner in ultra-rare disorders, a growth leader on NASDAQ, and the world’s second most innovative company, according to Forbes. Before joining Alexion, Thomas built and led the worldwide Medical Departments of Novartis Oncology and Celgene as their Vice President and Global Head of Medical Affairs. Prior to Celgene, Thomas served as the medical head of Amgen Europe for hematology and oncology. He developed and commercialized six lifesaving paradigm-changing blockbuster medicines in cancer, ultra-rare disorders, and inflammation. He is recognized for achieving five of the most successful product launches in life science.

Thomas earned his MD degree at RWTH Aachen University and his MBA at Columbia Business School. He is the Chair of the Healthcare Advisory Board at Columbia Business School, former Chair of the Board of Directors of HeritX Inc., and former Board Chair and President of FORCE, the US patient organization for inherited breast and ovarian cancer.

"I am thrilled to work together with Notable’s outstanding team and seize the tremendous opportunities that our predictive technology platform offers for accelerating the development of personalized cancer treatments," said Bock. "Building on our robust platform, collaborations and insights, we can now focus on the most promising projects and advance them with the sense of urgency that patients deserve."