March 28, 2022 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on efficiently acquiring, developing and commercializing or monetizing promising therapeutic products and product candidates, reported financial results and recent corporate highlights for the full-year ended December 31, 2021 (Press release, Fortress Biotech, MAR 28, 2022, View Source [SID1234611033]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress and its partner companies had an exceptional year in 2021, generating a record-setting annual net revenue of $68.8 million, representing more than 50% growth over 2020. We also began to realize the value of our monetization strategy when AstraZeneca acquired Caelum Biosciences ("Caelum"), a company founded by Fortress, during the fourth quarter of 2021, when we received a $56.9 million payment for our investment in Caelum. Fortress has the potential to receive up to an additional approximately $155 million in future milestone payments from the transaction, which includes proceeds from the release of escrow funds. Our subsidiary Cyprium Therapeutics ("Cyprium") executed an agreement with Sentynl Therapeutics to commit development funding for and contingently acquire Cyprium’s proprietary rights to CUTX-101, its Copper Histidinate product candidate for the treatment of Menkes disease, upon FDA approval. Also of note, Journey Medical Corporation ("Journey Medical") completed its $30.6 million initial public offering ("IPO"), net of discounts and other offering costs, launched Accutane (isotretinoin), acquired Qbrexza (glycopyrronium) from Dermira, Inc. and entered into a collaboration agreement with Dr. Reddy’s Laboratories Ltd. to develop and commercialize DFD-29 (minocycline modified release capsules 40 mg) for the treatment of rosacea."

1 Figure is net of miscellaneous transaction expenses and a 10% holdback for an indemnification escrow.

Dr. Rosenwald continued, "Looking ahead in 2022, we anticipate continued progress and growth from our nine marketed prescription pharmaceutical products and over 30 product candidates in development. We have 30 ongoing clinical trials, including four product candidates in seven2 ongoing pivotal clinical trials. We expect the rolling submission of the New Drug Application ("NDA") for CUTX-101 to be complete in mid-2022. After announcing positive top-line results from the registration-enabling study of cosibelimab in metastatic cutaneous squamous cell carcinoma ("cSCC") in January, our partner company Checkpoint Therapeutics, Inc. ("Checkpoint") intends to submit a Biologics License Application ("BLA") for cosibelimab in 2022, followed thereafter by a Marketing Authorization Application submission in Europe. Mustang Bio, Inc. ("Mustang Bio"), another one of our partner companies, plans to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted, autologous CAR T cell therapy for relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL") in the first half of 2022. Mustang Bio also plans to enroll the first patient in a pivotal multicenter Phase 2 clinical trial to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with X-linked severe combined immunodeficiency ("XSCID") in the second half of 2022. This ongoing advancement showcases the ability of Fortress’ business model to generate value for our shareholders and develop innovative therapies to help patients with unmet needs across multiple disease areas."

2021 and Recent Corporate Highlights3:

Marketed Dermatology Products and Product Candidates

·Journey Medical currently has nine prescription dermatology products. In 2021 and early 2022, Journey Medical acquired and launched four prescription dermatology products including Accutane, Qbrexza, Amzeeq and Zilxi, and two product candidates, DFD-29 and FCD105.
·Our products generated net revenues of $63.1 million for full-year 2021, compared to full-year 2020 net revenues of $44.5 million, representing growth of 42%.
·In March 2022, Journey Medical dosed the first patient in the Phase 3 clinical program of DFD-29 for the treatment of papulopustular rosacea. Topline data is anticipated in the first quarter of 2023 with an NDA filing expected in the second half of 2023.
·
In February 2022, Journey Medical received notice from its exclusive licensing partner in Japan, Maruho Co., Ltd. ("Maruho"), that Japan’s Ministry of Health, Labor and Welfare ("MHLW") approved Rapifort Wipes 2.5% (glycopyrronium tosylate hydrate) for the treatment of primary axillary hyperhidrosis. This approval triggered a milestone payment of $10 million to Journey Medical, of which $7.5 million will be paid to Dermira, Inc. ("Dermira"), a wholly owned subsidiary of Eli Lilly and Company, pursuant to the terms of the Asset Purchase Agreement between Journey Medical and Dermira, with net proceeds of $2.5 million to Journey Medical.

·In November 2021, Journey Medical completed its IPO of common stock of 3,520,000 shares at a public offering price of $10.00 per share, for net proceeds of $30.6 million, after deducting underwriting discounts and offering expenses. All of the shares of common stock were offered by Journey Medical.
·In July 2021, Journey Medical completed its final private placements under the 8% Cumulative Convertible Class A Preferred Stock Offering (the "Preferred Offering"), issuing an aggregate of 758,680 preferred shares at a price of $25.00 per share, raising approximately $19.0 million in gross proceeds and, after deducting commissions, fees and expenses, receiving approximately $17.0 million in net proceeds. These shares converted into Journey Medical common stock upon the IPO.
·On April 1, 2021, Journey Medical entered into an agreement with East West Bank ("EWB") in which EWB provided a $7.5 million working capital line of credit. In January 2022, Journey Medical expanded the borrowing capacity of the EWB credit agreement to $30.0 million, which includes an increase to the working capital line of credit to $10.0 million and a $20.0 million term loan.
·We intend to launch an additional prescription product this year in the first half of 2022.

2 Includes two trials at partner company Caelum Biosciences, which was sold to AstraZeneca in October 2021 and with respect to which Fortress remains eligible to receive up to approximately $155 million in future milestone payments from the transaction.

3 Includes product candidates in development at Fortress, majority-owned and controlled partners and/or subsidiaries, and partners and/or subsidiaries 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, subsidiaries, and partners, as dictated by context.

CUTX-101 (Copper Histidinate for Menkes disease)

·In February 2021, our subsidiary Cyprium signed a Development and Asset Purchase Agreement with Sentynl Therapeutics, a wholly owned subsidiary of Zydus Lifesciences Ltd., 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 related to the NDA submission and approval process 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. Cyprium is responsible for the development of CUTX-101 through approval of the NDA by the FDA, and Sentynl will be responsible for commercialization of CUTX-101, as well as progressing newborn screening activities.
·In October 2021, we announced positive results from an efficacy and safety analysis of data integrated from two completed pivotal studies in patients with Menkes disease treated with CUTX-101, copper histidinate (CuHis). These data were presented as a virtual poster at the 2021 American Academy of Pediatrics National Conference & Exhibition. Overall, a 79% reduction in risk of death was observed in patients treated within four weeks of birth compared with an untreated historical control cohort of patients and median overall survival (OS) was 177.1 and 16.1 months, respectively, with a hazard ratio (HR) of (95% CI) = 0.208 (0.094, 0.463) p<0.0001. A 75% reduction in the risk of death was also observed in patients treated after four weeks of birth compared with untreated historical control subjects and median OS was 62.4 and 17.6 months, respectively; HR (95% CI) = 0.253 (0.119, 0.537); p<0.0001.
·In December 2021, we initiated the rolling submission of an NDA to the U.S. Food and Drug Administration ("FDA") for CUTX-101. We intend to complete the rolling submission of the NDA for CUTX-101 in mid-2022.
·In March 2022, Cyprium announced positive data on CUTX-101 were presented as a "Top-Rated Abstract" and Poster at the 2022 American College of Medical Genetics and Genomics Clinical Genetics Meeting. The abstract can be viewed here.
·CUTX-101 was sourced by Fortress and is currently in development at Cyprium.

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

On October 5, 2021, AstraZeneca plc as successor-in-interest to Alexion Pharmaceuticals, Inc. ("Alexion") acquired Caelum for an upfront payment of approximately $150 million paid to Caelum shareholders, of which approximately $56.9 million was paid to Fortress, net of the $6.4 million, 24-month escrow holdback amount and other miscellaneous transaction expenses. The agreement also provides for additional potential payments to Caelum shareholders totaling up to $350 million, payable upon the achievement of regulatory and commercial milestones. Fortress is eligible to receive 42.4% of all potential milestone payments, totaling up to approximately $212 million.

·There are two ongoing Phase 3 studies of CAEL-101 for AL amyloidosis. (ClinicalTrials.gov Identifiers: NCT04512235 and NCT04504825)
·In December 2021, CAEL-101 data were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting ("ASH2021"). Abstracts can be viewed online through the ASH (Free ASH Whitepaper)2021 website here.
·In June 2021, Caelum announced that CAEL-101 clinical data were presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress ("EHA2021"). The data, presented in two e-posters, demonstrates the safety and tolerability profile of CAEL-101 to further support the dose selection for the ongoing Phase 3 study, and suggest possible cardiac and renal response.
·Also in June 2021, the FDA granted Fast Track designation to CAEL-101 for the treatment of light chain AL amyloidosis.
·CAEL-101 was sourced by Fortress and was developed by Caelum until Caelum was acquired by AstraZeneca.

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

·In December 2021, we announced the initiation of the CONTERNO study, a global, randomized Phase 3 trial of cosibelimab in combination with pemetrexed and platinum chemotherapy for the first-line treatment of patients with non-squamous non-small cell lung cancer.
·In January 2022, we announced positive topline results from our registration-enabling clinical trial evaluating the safety and efficacy of our anti-PD-L1 antibody, cosibelimab, administered as a fixed dose of 800 mg every two weeks in patients with metastatic cSCC. The study met its primary endpoint, with cosibelimab demonstrating a confirmed objective response rate of 47.4% (95% CI: 36.0, 59.1) based on independent central review of 78 patients enrolled in the metastatic cSCC cohort using Response Evaluation Criteria in Solid Tumors version 1.1 criteria. Checkpoint intends to submit a BLA for cosibelimab in 2022, followed by a Marketing Authorization Application submission in Europe and other territories worldwide. With a potentially favorable safety profile versus anti-PD-1 therapy and a plan to commercialize at a substantially lower price, we believe cosibelimab has the potential to be a market disruptive product in the $30 billion and growing PD-(L)1 class.
·Cosibelimab was sourced by Fortress and is currently in development at our partner company, Checkpoint.

Olafertinib (formerly CK-101, a third-generation epidermal growth factor receptor ("EGFR") inhibitor)

·During the second quarter of 2021, we had productive interactions with the FDA regarding Checkpoint’s ongoing development program for olafertinib (formerly CK-101), our third-generation EGFR inhibitor being evaluated by our partner in an ongoing double-blind, randomized Phase 3 study in China.
·Olafertinib was sourced by Fortress and is currently in development at our partner company, Checkpoint.

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

·In May 2021, we announced that the FDA approved Mustang Bio‘s Investigational New Drug ("IND") application to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted, autologous CAR T cell therapy for relapsed or refractory B-NHL and CLL. We intend to dose the first patient in that trial in the first half of 2022.
·In June 2021, we announced that MB-106 CD20-targeted CAR T data were presented at EHA (Free EHA Whitepaper)2021. Dr. Mazyar Shadman of Fred Hutchinson Cancer Research Center presented updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL, which showed a favorable safety profile and compelling clinical activity, with a 93% overall response rate and 67% complete response rate in patients treated with the modified cell manufacturing process.
·Also in June 2021, we hosted a key opinion leader webinar featuring a presentation from Dr. Shadman, who discussed interim results from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for B-NHL and CLL.
·In November 2021, we announced that Mustang Bio was awarded a grant of approximately $2 million from the National Cancer Institute of the National Institutes of Health. This two-year award will partially fund the Mustang Bio-sponsored multicenter trial to assess the safety, tolerability and efficacy of MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B- NHL or CLL.
·In December 2021, we announced that MB-106 data were presented at ASH (Free ASH Whitepaper)2021. Dr. Mazyar Shadman of Fred Hutchinson Cancer Research Center presented updated interim data showing a 95% overall response rate, 65% complete response rate and favorable safety profile from the ongoing Phase 1/2 clinical trial for B-NHL and CLL. A copy of the poster can be viewed online here.
·Also in December 2021, we hosted a key opinion leader webinar featuring a presentation from Dr. Shadman, who discussed interim results from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for B-NHL and CLL. A replay of the webinar can be found here.
·In January 2022, we announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL and CLL, were selected for a poster presentation at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research, rescheduled to take place April 23-26, 2022, in Salt Lake City, Utah. A copy of the abstract can be viewed on the meeting website here.
·MB-106 was sourced by Fortress and is currently in development at our partner company, Mustang Bio.

Dotinurad (Urate Transporter (URAT1) Inhibitor)

·In May 2021, we announced an exclusive license agreement with Fuji Yakuhin Co. Ltd. to develop Dotinurad in North America and Europe. Dotinurad is a potential best-in-class urate transporter (URAT1) inhibitor for gout and possibly other hyperuricemic indications including chronic kidney disease (CKD) and heart failure. Dotinurad (URECE tablet) was approved in Japan in 2020 as a once-daily oral therapy for gout and hyperuricemia. Dotinurad was efficacious and well-tolerated in more than 500 Japanese patients treated for up to 58 weeks in Phase 3 clinical trials. Over 1,000 Japanese patients have been treated safely with this drug.
·In December 2021, we filed an IND with the FDA. We expect to initiate a Phase 1 clinical trial to evaluate Dotinurad for the treatment of gout in the first half of 2022.
·Dotinurad was sourced by Fortress and is currently in development at our subsidiary company, UR1 Therapeutics.

MB-107 and MB-207 (Lentiviral Gene Therapies for XSCID)

·In February 2021, we announced encouraging MB-107 and MB-207 clinical updates from our XSCID investigator-IND trials, as well as additional consistent safety and efficacy data.
·In August 2021, we announced that the European Medicines Agency ("EMA") granted Priority Medicines ("PRIME") designation to MB-107, a lentiviral gene therapy for the treatment of XSCID in newly diagnosed infants, also known as bubble boy disease.
·In the second half of 2022, we expect to enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang Bio’s IND to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with XSCID.
·Mustang Bio filed an IND application in December 2021 for its pivotal multicenter Phase 2 clinical trial of MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with a hematopoietic stem cell transplantation ("HSCT") and for whom re-treatment is indicated. The trial is currently on hold pending CMC clearance from the FDA, and based on feedback from the Agency, Mustang Bio expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial in the first quarter of 2023.
·MB-107 and MB-207 were sourced by Fortress and are currently in development at our partner company, Mustang Bio.

Triplex (Cytomegalovirus ("CMV") vaccine)

·In December 2021, we announced that a Phase 2 double-blind, randomized, placebo-controlled clinical trial was initiated to evaluate the safety and efficacy of Triplex, a cytomegalovirus ("CMV") vaccine, in eliciting a CMV-specific immune response and reducing CMV replication in people living with HIV. The trial is being conducted by the AIDS Clinical Trials Group and is funded by the National Institute of Allergy and Infectious Disease, part of the National Institutes of Health.
·Triplex was sourced by Fortress and is currently in development at our subsidiary company, Helocyte, Inc.

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

·In May 2021, we announced that the first patient was dosed at City of Hope in a clinical trial to establish the safety and feasibility of administering MB-101 (autologous IL13Rα2-directed CAR T cells) to patients with leptomeningeal brain tumors (e.g., glioblastoma, ependymoma or medulloblastoma).
·In October 2021, Christine Brown, Ph.D., Deputy Director, T Cell Therapeutics Research Laboratory Professor, Departments of Hematology & Hematopoietic Cell Transplantation and Immuno-Oncology and The Heritage Provider Network Professor in Immunotherapy at City of Hope, presented updated Phase 1 clinical data regarding MB-101 (IL13Rα2-targeted CAR T cells) for the treatment of glioblastoma at two scientific conferences, the First Annual Conference on CNS Clinical Trials, co-sponsored by the Society for Neuro-Oncology and American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Special Conference: Brain Cancer.
·MB-101 was sourced by Fortress and is currently in development at our partner company, Mustang Bio.

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

·In February 2022, Phase 1 data on MB-105, a PSCA-targeted CAR T cell therapy administered systemically to patients with PSCA-positive metastatic castration-resistant prostate cancer ("mCRPC"), were presented by City of Hope at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium. The data results indicated that PSCA-CAR T-cell therapy is feasible in patients with mCRPC with dose limiting toxicity of cystitis and show preliminary anti-tumor effect at a dose of 100 million cells plus lymphodepletion. It was concluded that escalation up to the next dose level of 300 million cells can proceed in the trial.
·MB-105 was sourced by Fortress and is currently in development at our partner company, Mustang Bio.

MB-109 (MB-101 (IL13Rα2-targeted CAR T Cell Therapy) + MB-108 oncolytic virus)

·In March 2022, we announced that an abstract reporting on Phase 1 trials being conducted at the University of Alabama at Birmingham (UAB) and City of Hope of Mustang Bio’s exclusively licensed oncolytic viral and CAR T-cell therapies for the treatment of patients with glioblastoma (GBM) was selected as a late-breaking poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, taking place April 8 – 13, 2022, in New Orleans, Louisiana. The abstract will also be published in the online Proceedings of the AACR (Free AACR Whitepaper).

Novel CAR T Technology

·In August 2021, we announced an exclusive license agreement with Mayo Clinic for a novel technology to create in vivo CAR T cells that may be able to transform the administration of CAR T therapies and has the potential to be used as an off-the-shelf therapy.
·The novel CAR T technology was sourced by Fortress and is currently in development at our partner company, Mustang Bio.

Ex Vivo Lentiviral Gene Therapy for RAG1 Severe Combined Immunodeficiency ("RAG1-SCID")

·In November 2021, we announced the execution of an exclusive license agreement with Leiden University Medical Centre for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.
·The ex vivo lentiviral gene therapy was sourced by Fortress and is currently in development at Mustang Bio.

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, 2021 and 2020. These results exclude the operations of our four public partner companies: Avenue Therapeutics, Inc. ("Avenue"), Checkpoint, Journey Medical and Mustang Bio, as well as any one-time, non-recurring, non-cash transactions. The goal in providing these non-GAAP financial metrics is to highlight the financial results of Fortress’ core operations, which are comprised of our privately held development-stage entities, as well as our business development and finance functions.

·As of December 31, 2021, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $308.0 million, compared to $254.4 million as of September 30, 2021, and $235.0 million as of December 31, 2020, an increase of $53.6 million for the fourth quarter and an increase of $73.0 million for the full year.

·On a GAAP basis, Fortress’ net revenue totaled $68.8 million for the full year ended December 31, 2021, which included $63.1 million in net revenue generated from our marketed dermatology products. This compares to net revenue totaling $45.6 million for the full year ended 2020, which included $44.5 million in net revenue generated from our marketed dermatology products.
·On a GAAP basis, consolidated research and development expenses including license acquisitions totaled $128.9 million for the full year ended December 31, 2021, compared to $64.1 million for the full year ended December 31, 2020. On a non-GAAP basis, research and development costs including research and development license acquisitions totaled $18.0 million for the full year ended December 31, 2021, compared to $10.0 million for the full year ended December 31, 2020.
·On a GAAP basis, consolidated selling, general and administrative costs were $86.8 million for the full year ended December 31, 2021, compared to $61.2 million for the full year ended December 31, 2020. On a non-GAAP basis, selling, general and administrative expenses were $28.6 million for the full year ended December 31, 2021, compared to $23.4 for the full year ended December 31, 2020.
·On a GAAP basis, consolidated net loss attributable to common stockholders was $(64.7) million, or $(0.79) per share, for the full year ended December 31, 2021, compared to net loss attributable to common stockholders of $(46.5) million, or $(0.65) per share for the full year ended December 31, 2020.
·
Fortress’ non-GAAP income attributable to common stockholders was $25.5 million, or $0.31 per share basic and $0.25 per share diluted, for the full year ended December 31, 2021, compared to Fortress’ non-GAAP loss attributable to common stockholders of $(30.8) million, or $(0.43) per share basic and diluted, for the full year ended December 31, 2020.

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 28, 2022, 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, Journey Medical and Mustang Bio ("public partner companies"), as well as our former subsidiary, 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. The Company also provides non-GAAP research and development costs, defined as GAAP research and development costs, less research and development costs of our public partner companies and non-GAAP selling, general and administrative costs, defined as GAAP selling, general and administrative costs, less selling, general and administrative costs of our public partner companies.

Management believes each of 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 standalone results separate from the results of its public partner companies. 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.Results for the year ended December 31, 2020 have been adjusted to remove Journey Medical as a public entity due to its IPO in November, 2021.

2.Avenue net loss for the years ended December 31, 2021 and 2020 of $3.7 million and $5.2 million, respectively, net of non-controlling interest of $2.9 million and $4.0 million, respectively.

3.Checkpoint net loss for the year ended December 31, 2021 of $56.7 million net of non-controlling interest of $39.2 million, Master Services Agreement ("MSA") fee to Fortress of $0.5 million, financing fee and payment-in-kind ("PIK") dividend to Fortress of $1.0 million and $6.6 million, respectively; and net loss for the year ended December 31, 2020 of $23.1 million net of non-controlling interest of $13.3 million, MSA fee to Fortress of $0.5 million, financing fee and PIK dividend to Fortress of $0.9 million and $4.6 million, respectively.

4.Journey Medical net loss for the year ended December 31, 2021 of $44.0 million net of non-controlling interest of $5.7 million and tax expense recognized on a stand-alone basis of $1.6 million; and net income for the year ended December 31, 2020 of $5.3 million, net non-controlling interest of $0.5 million and stand-alone tax expense of $1.9 million.

5.Mustang net loss of $66.4 million net of non-controlling interest of $48.5 million, MSA fee to Fortress of $0.5 million and financing fee and PIK dividend to Fortress of $1.9 million and $4.2 million, respectively, for the year ended December 31, 2021; and net loss of $60.0 million net of non-controlling interest of $36.4 million, MSA fee to Fortress of $0.5 million and financing fee and PIK dividend to Fortress of $2.4 million and $7.6 million, respectively, 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, 2021 and 2020, respectively.

2.Excludes $6.6 million and $4.6 million of PIK dividend payable to Fortress for the year ended December 31, 2021 and 2020, respectively.

3.Excludes $0.3 million of Fortress MSA expense and $4.2 million PIK dividend payable to Fortress for the year ended December 31, 2021; and excludes $0.3 million of Fortress MSA expense and $7.6 million PIK dividend payable to Fortress for the year ended December 31, 2020.

4.Includes Selling, general and administrative expenses and wire transfer fraud loss for the year ended December 31, 2021.

5.Excludes $0.5 million of Fortress MSA expense and $1.0 million Fortress financing fee for the year ended December 31, 2021; and $0.5 million of Fortress MSA expense and $0.9 million Fortress financing fee for the year ended December 31, 2020.

6.Excludes $0.3 million of Fortress MSA expense and $1.9 million Fortress financing fee for the year ended December 31, 2021; and $0.3 million of Fortress MSA expense and $2.4 million Fortress financing fee for the year ended December 31, 2020.

On March 28, 2022 Cannabics Pharmaceuticals Inc. (OTCQB: CNBX), a global leader in the development of cancer related cannabinoid-based medicine, reported that it has changed its corporate name to "CNBX Pharmaceuticals Inc (Press release, Cannabics Pharmaceuticals, MAR 28, 2022, View Source [SID1234611055])."

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The Company’s new brand identity reflects the significant transformation that has already taken place across the Company’s platform, as well as the team’s forward-looking approach to molecule-based drug development for the treatment of colorectal cancer, and in preparation of a Phase I/II (a) clinical validation study for its proprietary RCC-33 drug candidate.

The Company is in the process of developing a new corporate website and updated social media accounts reflecting the new corporate company name. The new website, www.cnbxpharma.com, and the updated social media accounts, are expected to go live by April 1st, 2022.

The Company’s stock will continue trading under the ticker OTCQB: CNBX. The corporate name change to CNBX Pharmaceuticals Inc. does not affect the rights of the company’s stockholders and no action is required by stockholders with respect to the name change. Outstanding stock certificates are not affected by the name change and will not need to be exchanged.

On March 28, 2022 Everest Medicines (HKEX 1952.HK, "Everest", or the "Company"), a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products to address critical unmet needs in Asia Pacific markets, reported its financial results for full year ended December 31, 2021, along with a corporate progress update (Press release, Everest Medicines, MAR 28, 2022, View Source [SID1234611070]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Despite a challenging backdrop for the biopharmaceutical industry in 2021, we propelled the Company forward by achieving an impressive number of accomplishments across our business, including fostering key strategic partnerships, expanding its global presence by building teams in South Korea, Singapore, and the Taiwan region, receiving the first of several expected drug approvals for Trodelvy in Singapore and investing in COVID-19 vaccines and therapeutic candidates to address the world’s ongoing public health crisis," said Kerry Blanchard, MD, PhD, Chief Executive Officer of Everest Medicines.

"As 2022 progresses, we look forward to building on the momentum established last year to position our company for sustained future growth. We will persist in growing our clinical capabilities, pursuing our global expansion strategy and building a commercial-ready biopharmaceutical company in order to provide innovative medicines to patients in need. We will strive to accomplish these goals, committed as always to the highest standards of quality and integrity," said Blanchard.

Recent Product Highlights and Anticipated Milestones

Sacituzumab govitecan-hziy (Trodelvy), our anchor drug candidate in the oncology therapeutic area, is a first-in-class TROP-2 directed antibody-drug conjugate (ADC).

Clinical and regulatory development achievements during the Reporting Period:

– In January 2021, we submitted a New Drug Application ("NDA") to the Health Sciences Authority ("HSA") of Singapore for sacituzumab govitecan for the treatment of patients with unresectable locally advanced or metastatic triple-negative breast cancer (mTNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease, and the indication was subsequently amended to second-line and later lines metastatic TNBC.
– In January 2021, the Center of Drug Evaluation ("CDE") of the China National Medical Products Administration ("NMPA") approved a China clinical trial application ("CTA") for sacituzumab govitecan for the treatment of patients with metastatic urothelial cancer ("mUC").

– In March 2021, the CDE of the China NMPA approved a CTA for a phase 2 basket trial for a variety of cancers with high TROP-2 expression. The trial is designed to evaluate sacituzumab govitecan monotherapy in 180 patients with relapse/refractory esophageal squamous cell carcinoma, gastric cancer, and cervical cancer at select sites in China.

– In April 2021, the Company’s partner, Gilead Sciences, Inc. (Gilead), received full approval from the US FDA for Trodelvy for the treatment of adult patients with second-line metastatic triple-negative breast cancer (mTNBC).

– In April 2021, Gilead received accelerated approval from the US FDA for Trodelvy for the treatment of adult patients with locally advanced or metastatic urothelial cancer (mUC) who have previously received a platinum-containing chemotherapy and either a programmed death receptor-1 (PD-1) or a programmed death-ligand 1 (PD-L1) inhibitor.

– In May 2021, NMPA accepted the Biologics License Application ("BLA") for sacituzumab govitecan for the treatment of second-line and later lines metastatic TNBC in adult patients. Following the BLA acceptance, sacituzumab govitecan was granted priority review by the CDE of China NMPA in May 2021.

– In May 2021, the Ministry of Food and Drug Safety ("MFDS") of South Korea had granted Fast Track Designation and Orphan Drug Designation to sacituzumab govitecan for the treatment of later line metastatic TNBC.

– In July 2021, Taiwan Food and Drug Administration ("TDFA") granted Pediatric and Rare Severe Disease Priority Review Designation for adult patients with second-line metastatic TNBC.

– In November 2021, the Company announced the topline results of its bridging study, EVER-132-001, a single-arm, multi-center Phase 2b study of sacituzumab govitecan conducted in China on patients with unresectable locally advanced or metastatic TNBC who have received two or more prior systemic therapies, at least one for metastatic disease.

– In December 2021, MFDS of South Korea accepted the Company’s NDA for sacituzumab govitecan for the treatment of second-line and later lines metastatic TNBC in adult patients.

– In December 2021, TFDA accepted the NDA submission of sacituzumab govitecan for the treatment of second-line and later lines metastatic TNBC in adult patients.

– The phase 3 Asia study was ongoing, which is designed to assess and compare the efficacy and safety of sacituzumab govitecan versus treatment of physician’s choice in Asian patients with hormone receptor positive, HER2 negative metastatic breast cancer ("HR+/HER2- mBC") who have failed at least two prior chemotherapy regimens. The trial will enroll approximately 330 HR+/HER2- mBC patients in Greater China and South Korea. The enrollment of this study is expected to complete enrollment by the first half of 2022.

Post-Reporting Period milestones and expected achievements:
– In January 2022, the Company announced it will participate in a study pursuant to a clinical trial collaboration between Gilead and Merck & Co., Inc. (MSD) to evaluate the combination of sacituzumab govitecan and MSD’s anti-PD-1 therapy Keytruda (pembrolizumab) in first-line metastatic non-small cell lung cancer (NSCLC).

– In January 2022, the HSA of Singapore approved the Company’s NDA for sacituzumab govitecan for the treatment of second-line and later lines metastatic TNBC.

– In March 2022, our partner Gilead announced results from the Phase 3 TROPiCS-02 study evaluating Trodelvy in patients with HR+/HER2- mBC who received prior endocrine therapy, CDK4/6 inhibitors and two to four lines of chemotherapy. The study met its primary endpoint with a statistically significant improvement in progression-free survival versus physician’s choice of chemotherapy.

– We anticipate receiving the BLA decision from China NMPA and NDA decision from the TDFA for sacituzumab govitecan for the treatment of second-line metastatic TNBC in 2022.

Nefecon (TARPEYO), our anchor drug candidate in cardio-renal therapeutic area, is a novel oral formulation of budesonide (budesonide delayed release capsules) in the development for the treatment of primary immunoglobulin A nephropathy ("IgAN").

Clinical and regulatory development achievements during the Reporting Period:
– The Company completed the Chinese patient enrollment into the NefIgArd Phase 3 global registrational study evaluating NEFECON as a treatment for primary IgAN.

– Our partner Calliditas Therapeutics AB, ("Calliditas") in April, 2021 granted an accelerated assessment procedure on its marketing authorization application ("MAA") for Nefecon in IgAN and submitted the MAA in May of 2021. In September 2021, Calliditas announced that the European Medicine Agency’s ("EMA") Committee for Human Medicinal Products ("CHMP") has decided to continue the assessment of the MAA for Nefecon under standard procedure assessment timelines, with potential conditional approval in second quarter of 2022.

– In December 2021, our partner Calliditas, announced that the US FDA approved TARPEYO (developed under Project name NEFECON) delayed release capsules the first and only treatment indicated to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression.

Post-Reporting Period milestones and expected achievements:
– In March 2022, the Company entered into a license agreement with Calliditas to develop and commercialize NEFECON for the treatment of primary IgAN in South Korea, expanding its license in addition to rights held in Greater China and Singapore.

– We expect to conduct an interim analysis of the Chinese patients in the global phase 3 NefIgArd study and this is expected to lead to a regulatory submission in China in second half of 2022.

PTX-COVID19-B, a potentially best-in-class lipid nanoparticle-formulated mRNA COVID-19 vaccine with a strong immunogenicity and tolerability profile.

Clinical and regulatory development achievements during the Reporting Period:
– In December 2021, we announced jointly with Providence Therapeutics ("Providence") that scientists from both companies had analyzed the sequence of the SARS-CoV-2 Omicron variant, selected viral sequences and designed plasmid clones to develop a new version of the COVID-19 vaccine specifically targeting the new Omicron variant.

– In December 2021, PTX-COVID19-B was selected to be part of a World Health Organization (WHO) Solidarity Trial Vaccines (STV) clinical trial, an international, randomized clinical trial designed to rapidly evaluate the efficacy and safety of promising new candidate vaccines.

Post-Reporting Period milestones and expected achievements:
– Providence will readout the data for the phase 2 trial of PTX-COVID19-B around mid-2022 and initiate a phase 3 trial for booster indication in mid-2022 as well.

Eravacycline (Xerava), is a novel, fully synthetic fluorocycline intravenous antibiotic developed for use as first-line empiric monotherapy for the treatment of multidrug resistant (MDR) infections, including MDR Gram-negative infections.

Clinical and regulatory development achievements during the Reporting Period:
– In March 2021 and September 2021 respectively, the China NMPA and the Department of Health of Hong Kong accepted an NDA for eravacycline for the treatment of complicated intra-abdominal infections (cIAI).

– In August 2021, the CDE of the China NMPA approved the CTA for eravacycline for the treatment of community-acquired bacterial pneumonia (CABP).

Post-Reporting Period milestones and expected achievements:
– We expect NDA approval for eravacycline for the treatment in cIAI in China within 2022.

Other clinical-stage assets

Clinical and regulatory development achievements during the Reporting Period:
– In August 2021, the Phase 1b/2 study evaluating FGF401 in combination with PD-1 inhibitor, pembrolizumab, in patients with advanced solid tumors, such as hepatocellular carcinoma (HCC) reached recommended Phase 2 dose. The trial is ongoing.

– In September 2021, we received the approval from the CDE of the China NMPA for the Investigational New Drug application under the Class One category for SPR206 (also known as EVER206), a novel, intravenous next-generation polymyxin product candidate in development for the treatment of MDR Gram-negative bacterial infections.

– Ralinepag is a potentially best-in-class oral, selective potent, once-daily IP receptor agonist intended for the treatment of pulmonary arterial hypertension (PAH). We continue to progress our Phase 3 registrational trial for PAH in China as part of a global Phase 3 study conducted together with our partner United Therapeutics.

Post-Reporting Period milestones and expected achievements:
– In March 2022, our licensing partner, Venatorx Pharmaceuticals, reported positive results from its pivotal Phase 3 study, CERTAIN-1, evaluating cefepime-taniborbactam, an investigational new drug, versus meropenem as a potential treatment for hospitalized adult patients with complicated urinary tract infections (cUTI), including acute pyelonephritis.

– In March, our partner, Pfizer Inc. announced positive topline results from a phase 3 study of etrasimod in development for the treatment of moderately to severly active ulcerative colitis ("UC") patients. These data along with results from ELEVATE 52 are expected to form the basis for planned future regulatory filings. Results from the ELEVATE 52 study will be available by the end of first quarter.

– We are conducting a Phase 3 study for etrasimod for the treatment of moderately to severely active UC, which is expected to complete enrollment in 2023.

– We anticipate initiating the Phase 1b/2 trial of the EVER-001 (also known as XNW1011), a next-generation covalent reversible Bruton’s tyrosine kinase (BTK) inhibitor for the treatment of renal diseases in 2022.

– We expect Phase 1 clinical trials of EDDC-2214, as oral antiviral treatment against SAR-CoV-2 and its variants, to commence by the end of 2022.

Key Corporate Developments

On 18 February 2021, we appointed Kevin Guo as our chief commercial officer. Mr. Guo has more than 22 years of commercial leadership and business management experience across a number of multinational pharmaceutical companies. Under Mr. Guo’s leadership, we continue to remain focused on advancing our work across four strategic pillars to launch strategy formulation, develop commercial capabilities, embrace and deploy innovative solutions, and expand our international footprint.
Effective 15 March 2021, the Company was selected as a constituent stock of the Hang Seng Composite Index, the Hang Seng Healthcare Index and the Hang Seng Hong Kong-Listed Biotech Index in accordance with the latest index series release by Hang Seng Indexed Company Limited. At the same time, the Company became eligible for Southbound Trading under the Stock Connect Scheme, which is a channel that facilitates stock trading and investment between Hong Kong and a broader base of Chinese investors.
On 15 April 2021, we appointed Dr. Jennifer Yang as our chief scientific officer, whose deep expertise in drug discovery and translational medicine will help the Company establish a robust discovery organization that contributes to the strategic expansion of our clinical development pipeline.
Effective 18 June 2021, the Company’s stock was included as a constituent stock of the Small Cap Index, FTSE All-Cap Index and FTSE Total-Cap Index in the FTSE Global Equity Index Series.
Effective 30 November 2021, the Company’s stock was added to the MSCI Global Small Cap Indexes — MSCI China Index.
Business Development Updates

In July 2021, the Company established key strategic partnerships with Tencent Holdings Limited (Tencent), Medbanks Health Technology Co., Ltd (Medbanks) and MediTrust Health Co., Ltd. (MediTrust) to explore innovative tools in digital marketing, patients’ access to novel medicines and payment solutions. In January 2022, the Company announced a strategic commercialization cooperation with Yuanxin Group and a strategic cooperation with the Sinopharm Group ("Sinopharm").
In September 2021, the Company entered into two separate definitive agreements with Providence, a clinical stage biotechnology company developing mRNA therapeutics and vaccines, to (i) license rights to Providence’s mRNA COVID-19 vaccine candidates in Asia’s emerging markets, including Greater China, Southeast Asia and Pakistan, and (ii) establish a broad, strategic partnership to develop mRNA products globally leveraging Providence’s cutting-edge mRNA technology platform.
In September 2021, we entered into an exclusive licensing agreement with Sinovent and SinoMab to develop, produce and commercialize EVER-001 (also known as XNW1011), a covalent reversible BTK inhibitor, globally for the treatment of renal diseases.
In September 2021, we announced a multi-year collaboration and license agreement with AbCellera Biologics Inc. (AbCellera) to discover therapeutic antibodies for up to 10 targets selected by the Company. The partnership will help to expand Company’s portfolio of novel medicines across multiple indications, with the initial programs focusing on targets in oncology and the renal space.
In January 2022, we entered into a global licensing agreement with Singapore’s national platform for drug discovery and development, the EDDC for the exclusive worldwide rights to develop, manufacture and commercialize EDDC’s series of viral 3C-like (3CL) protease inhibitors as a potentially best-in-class oral antiviral treatment against SARS-CoV-2 and its variants. The Company has full rights to sub-license the drug further and will receive a full technology transfer.
Financial Highlights

IFRS Numbers:

Research and development ("R&D") expenses increased by RMB236.0 million to RMB613.4 million for the year ended 31 December 2021, from RMB377.4 million for the year ended 31 December 2020, primarily due to: (i) increased number of clinical trials for our drug candidates; (ii) expansion of internal discovery team to build up in-house R&D capabilities; and (iii) increased costs occurred in the process of technical transfer for our drug candidates.
General and administrative expenses decreased by RMB35.1 million to RMB242.7 million for the year ended 31 December 2021, from RMB277.8 million for the year ended 31 December 2020, primarily due to decreased expenses in relation to the public listing of the Company in 2020.
Distribution and selling expenses increased by RMB165.0 million to RMB198.2 million for the year ended 31 December 2021, from RMB33.2 million for the year ended 31 December 2020, primarily due to expansion of commercial organization and pre-launch and launch activities carried out for product commercialization.
Net loss for the year ended 31 December 2021 was RMB1,008.7 million, from RMB5,658.2 million for the year ended 31 December 2020, primarily attributable to the decrease in loss from fair value change in financial instruments issued to investors.
Cash and cash equivalents amounted to RMB2,640.1 million as of 31 December 2021
Non-IFRS Measure

Adjusted loss for the year[1] was RMB777.3 million for the year ended 31 December 2021, representing an increase of RMB174.4 million from RMB602.9 million for the year ended 31 December 2020, primarily due to increase in R&D expense and distribution and selling expenses.
Conference Call Information

A live conference call will be hosted on March 29, 2022 at 9:00 AM Beijing Time (March 28, 2022 at 9:00 PM U.S. Eastern Time).

The live webcast of the conference call will be available at View Source

Alternatively, participants may dial in to the conference call using below dial-in information:

A replay will be available shortly after the call and can be accessed by visiting the Company’s website at View Source

On March 28, 2022 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company focused on the discovery and development of innovative gamma-delta T cell therapies utilizing its DeltEx platform, reported a clinical update from the ongoing Phase 1 trial of INB-100 (Press release, In8bio, MAR 28, 2022, View Source [SID1234611034]). This program is an allogeneic, or donor-derived, gamma-delta T cell therapeutic candidate in development for patients with leukemia undergoing haploidentical hematopoietic stem cell transplant (HSCT). Two of the INB-100 patients treated have been in remission for nearly two years, and the third patient is in continuing remission at nine months post-treatment.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The exploratory clinical correlative data highlights the robust reconstitution of the immune system of treated patients. The data show positive trends in levels of immune cells, including alpha-beta T cells, B cells and gamma-delta T cells. This suggests that the systemic immune system may show long-term positive trends in treated patients. The toxicity profile continues to be manageable with no treatment-related Grade 3 or greater adverse events. The clinical and correlative update was presented at the virtual European Society for Blood and Marrow Transplantation (EBMT) 48th Annual Meeting.

"We are encouraged by the patients’ responses to INB-100 treatment given this population’s high risk for recurrence," said Lawrence S. Lamb, Ph.D., Chief Scientific Officer and co-founder of IN8bio. "As we near the two-year mark in remission for our first and longest enrolled patient, we continue to monitor data from our ongoing clinical correlative studies, which are indicating a positive systemic immune response following the infusion of INB-100. Patient recruitment in this trial is continuing, and we look forward to releasing further clinical and correlative data with additional patients later this year."

About the INB-100 Phase 1 Trial

The Phase 1 clinical trial (NCT03533816) is a dose-escalation trial of allogeneic derived, gamma-delta T cells that have been expanded and activated ex vivo and administered systemically to patients with leukemia following haploidentical HSCT. Three high-risk AML patients with complex cytogenetics have been treated to-date. The single-institution clinical trial is currently being conducted at The University of Kansas Cancer Center (KUCC). The primary endpoints of this trial are safety and tolerability, and secondary endpoints include rates of GvHD, relapse rate and overall survival.

On March 28, 2022 MannKind Corporation (Nasdaq: MNKD), a company focused on the development and commercialization of inhaled therapeutic products for patients with endocrine and orphan lung diseases, reported that its Chief Executive Officer, Michael Castagna, PharmD, will participate in the Lytham Partners Spring 2022 Investor Conference taking place virtually on April 4-7, 2022 (Press release, Mannkind, MAR 28, 2022, View Source [SID1234611056]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The Company’s webcast presentation will be available for viewing at 11:00am ET on Monday, April 4, 2022, on the Company’s website at or View Source The webcast will also be archived and available for replay.

Management will be participating in virtual one-on-one meetings throughout the event. To arrange a meeting with management, please contact Lytham Partners at 1×[email protected] or register at www.lythampartners.com/spring2022invreg.