On March 31, 2022 Affimed N.V. (Nasdaq: AFMD) ("Affimed", or the "Company"), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, reported financial results for the year ended December 31, 2021, and provided an update on clinical and corporate progress (Press release, Affimed, MAR 31, 2022, View Source [SID1234611236]).

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"Over the course of 2021 we continued to make strong progress with each of our ICE candidates by executing on our clinical development objectives," said Dr. Adi Hoess, CEO of Affimed. "In particular, the clinical data from AFM13 pre-complexed with cord blood-derived natural killer (NK) cells in relapsed/refractory CD30+ lymphomas demonstrated the broad potential of our ICE platform. We are very encouraged by this data as this could significantly broaden the AFM13 market opportunity targeting CD30-positive Hodgkin, T cell, and potentially B cell lymphoma. Furthermore, we believe these results validate that our ICE candidates can meaningfully enhance NK cell-driven efficacy in underserved cancer patients, and we look forward to the presentation of further data at AACR (Free AACR Whitepaper).

"With the establishment of the recommended phase 2 dose for AFM24, we have embarked on a broad development strategy investigating AFM24 as monotherapy and in two combination studies – one with atezolizumab and the second with SNK01 NK cells," Dr. Hoess continued.

"We spent a good portion of 2021 transforming our organization by growing our team of dedicated scientists and industry experts. This investment in acquiring the right resources and talent, as well as our strong cash position, will ensure that we continue to deliver and advance our programs in 2022 and beyond."

Clinical Stage Program Updates

AFM13 (CD30/CD16A)

Affimed completed enrollment of its REDIRECT study (AFM13-202). The Company expects to report top-line data in the second half of 2022.

REDIRECT is a phase 2, registration-directed study of AFM13 monotherapy in patients with relapsed or refractory CD30-positive peripheral T-cell lymphoma (PTCL).
In December 2021, Affimed reported updated data from AFM13-104, the investigator sponsored trial (IST) led by The University of Texas MD Anderson Cancer Center investigating the combination of AFM13 pre-complexed with cord blood-derived natural killer cells followed by AFM13 monotherapy. The data reported findings for the first 19 patients enrolled in the study, including six patients treated at lower doses and 13 patients treated at the recommended phase 2 dose. There was impressive anti-tumor activity with a 100% objective response rate (ORR) and 38% complete response rate (CRR) at the RP2D after a single cycle of therapy.
Data of patients receiving two treatment cycles will be presented at the AACR (Free AACR Whitepaper) Clinical Trials Plenary Session on April 10, 2022, by Yago Nieto, M.D., Ph.D., Professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson. The presentation will also be included in the AACR (Free AACR Whitepaper) Press Conference on April 10, 2022.

MD Anderson has initiated enrollment of patients into the phase 2 portion of the trial and the Food and Drug Administration (FDA) has approved an amendment to the AFM13-104 trial protocol to increase the patient population treated at the RP2D to 40 CD30-positive lymphoma patients including HL, TCL and BCL and allow for the treatment of patients with more than the two cycles of therapy, at the investigator’s discretion.

AFM24 (EGFR/CD16A)

For AFM24, an EGFR/CD16A ICE, Affimed achieved a key milestone through the identification of the RP2D of 480 mg weekly dosing for the treatment of patients with EGFR-expressing solid tumors. The Company has initiated a broad development strategy intended to deliver the highest probability of success which includes three studies investigating various solid tumor indications.
In the monotherapy phase 1/2a clinical trial (AFM24-101), Affimed has initiated enrollment in the expansion phase at the RP2D. The expansion cohorts include patients with renal cell carcinoma, non-small cell lung cancer and colorectal cancer. Data from the dose escalation phase of the trial will be presented in a poster at the AACR (Free AACR Whitepaper) 2022 meeting in April.
Enrollment was initiated in AFM24-102, the phase 1/2a combination study of AFM24 with the anti-PD-L1 checkpoint inhibitor atezolizumab (Tecentriq) to treat patients with non-small cell lung cancer, gastric and gastroesophageal junction adenocarcinoma and pancreatic/hepatocellular/biliary tract cancer patients.
Enrollment was also initiated in AFM24-103, the phase 1/2a combination study of AFM24 with the SNK01 NK cells to treat patients with non-small cell lung cancer, squamous cell carcinoma of the head and neck, and colorectal cancer.
Affimed expects to report initial data from the AFM24 studies during 2022.
Preclinical Programs

AFM28 (CD123/CD16A)

Preclinical candidate AFM28, developed on the Company’s proprietary ROCK platform, is a bispecific, tetravalent ICE that targets CD16A on NK cells and macrophages, as well as CD123 on leukemic cells and leukemic stem cells that are prevalent in acute myeloid leukemia (AML).
Preclinical data demonstrates that AFM28 induces tumor cell lysis more potently than conventional anti-CD123 antibodies, in particular at low CD123 expression. Further, AFM28 shows a 100-fold more potent NK cell activation in an ex vivo analysis, compared to Fc-enhanced IgG1 antibodies. In a preclinical toxicology study in cynomolgus monkeys, AFM28 was safe and well-tolerated and exhibited the expected pharmacodynamic activity suggesting a good safety profile and the potential to eliminate CD123+ cells in vivo.
An IND is planned to be submitted in the first half of 2022 and clinical investigation of AFM28 is planned to start in second half of 2022.
Pre-clinical pipeline

Affimed is continuing the generation of several novel ICE molecules derived from its proprietary ROCK platform.
AFM32 and other partnered pre-clinical programs

Genentech and Roivant partnered programs continue to progress and future updates will be provided at their discretion.
Full Year 2021 Financial Highlights

Affimed’s consolidated financial statements are prepared in accordance with IFRS as issued by the International Accounting Standard Board (IASB). The consolidated financial statements are presented in euros, which is the Company’s functional and presentation currency.

As of December 31, 2021, cash and cash equivalents totaled €197.6 million compared to €146.9 million as of December 31, 2020. Based on its current operating plan and assumptions, Affimed anticipates that its cash and cash equivalents will support operations into the second half of 2023.

Net cash used in operating activities for the year ended December 31, 2021, was €86.6 million compared to €19.4 million for the year ended December 31, 2020. The increase is due to higher cash expenditure for research and development as well as general and administrative activities. In addition, net cash used in operating activities in 2020 included cash received from an initial upfront payment and committed funding of €33.3 million (USD 40 million) from the Roivant collaboration, as well as a milestone payment received pursuant to the Genentech collaboration.

Total revenue for the year ended December 31, 2021, was €40.4 million compared with €28.4 million for the year ended December 31, 2020.

Revenue for the years ended December 31, 2021, and December 31, 2020, predominantly relate to the Genentech and Roivant collaboration. Collaboration revenue for the year ended December 31, 2021, was €39.3 million, with €21.6 million coming from the Genentech collaboration and €17.7 million from the Roivant collaboration. Collaboration revenue for year ended December 31, 2020, was €27.8 million, with €26.2 million from the Genentech collaboration and €1.4 million from the Roivant collaboration.

Research and development expenses for the year ended December 31, 2021, increased 63 percent from €50.0 million for the year ended December 31, 2020, to €81.5 million for the year ended December 31, 2021. The increase was primarily due to increased expenses for AFM24 and AFM28 including costs to produce clinical trial material, an increase in costs associated with other early-stage programs and infrastructure, and an increase in share-based payment expenses.

General and administrative expenses for the year ended December 31, 2021, increased 77 percent, from €13.7 million for the year ended December 31, 2020, to €24.2 million in the year ended December 31, 2021. The increase predominately relates to higher share-based payment expenses in 2021, higher premiums for D&O liability insurance, and higher consulting expenses.

Net finance income for the year ended December 31, 2021, was €6.5 million compared to net finance costs of €6.6 million for the year ended December 31, 2020. Net finance income/costs is largely due to foreign exchange gains/losses related to assets denominated in U.S. dollars as a result of currency fluctuations between the U.S. dollar and Euro during the year.

Net loss for the year ended December 31, 2021, was €57.5 million, or €0.48 per common share compared with a net loss of €41.4 million, or €0.50 per common share, for the year ended December 31, 2020.

The weighted number of common shares outstanding for the year ended December 31, 2021, was 119.5 million.

Additional information regarding these results will be included in the notes to the consolidated financial statements as of December 31, 2021, included in Affimed’s filings with the U.S. Securities and Exchange Commission (SEC).

Note on International Financial Reporting Standards (IFRS)
Affimed prepares and reports consolidated financial statements and financial information in accordance with IFRS as issued by the International Accounting Standards Board. None of the financial statements were prepared in accordance with Generally Accepted Accounting Principles in the United States. Affimed maintains its books and records in Euro.

Conference Call and Webcast Information

Affimed will host a conference call and webcast March 31, 2022, at 8:30 a.m. EDT to discuss full year 2021 financial results and recent corporate developments. The conference call will be available via phone and webcast.

To access the call, please dial +1 (409) 220-9054 for U.S. callers, or +44 (0) 8000 323836 for international callers, and reference passcode 6590614 approximately 15 minutes prior to the call.

A live audio webcast of the conference call will be available in the "Webcasts" section on the "Investors" page of the Affimed website at View Source

A replay of the webcast will be accessible at the same link for 30 days following the call.

On March 31, 2022 Adagene Inc. ("Adagene") (Nasdaq: ADAG), a company transforming the discovery and development of novel antibody-based therapies, reported financial results for the full-year ended December 31, 2021, and provided corporate updates (Press release, Adagene, MAR 31, 2022, View Source [SID1234611235]).

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"We are committed to delivering on our promise to transform cancer immunotherapy, concentrating on overcoming the known safety issues linked to promising yet challenging targets," said Peter Luo, Ph.D., Co-founder, Chief Executive Officer and Chairman of Adagene. "On the clinical front, we are focused on revitalizing anti-CTLA-4 as a safe and efficacious backbone therapy, which remains a huge market opportunity and the only checkpoint inhibitor approved as both monotherapy and combination therapy with anti-PD-1. We are developing potential best-in-class molecules to unleash the full potential of this target for strong Treg depletion in the tumor microenvironment (TME) and superior safety profiles."

Dr. Luo continued, "We are also leveraging our SAFEbody technology to overcome challenges of bispecific T-cell engagers (TCEs), particularly for solid tumors, and to address safety issues of widely expressed targets like CD47. We have made wonderful progress to de-risk our clinical pipeline, grow our transformative preclinical assets, and validate our AI-powered, scalable antibody technology platform with global partnerships. With a solid cash position, we are well-positioned to achieve our expected milestones while continuing to enhance value of our pipeline."

PIPELINE GROWTH & HIGHLIGHTS

During 2021, Adagene advanced its wholly-owned, differentiated pipeline of antibody-based therapeutics, including three clinical programs in single and combination phase 1b/2 trials, five programs in IND-enabling studies and over 50 more across stages of discovery.

Clinical candidates include multiple modalities of antibody therapeutics against established targets such as CTLA-4 with ADG116 (NEObody) and ADG126 (SAFEbody), challenging targets such as CD137 with ADG106 (NEObody) and ADG206 (the masked anti-CD137 POWERbody), and targets with known safety issues such as CD47 with ADG153 (SAFEbody). The company’s expanding preclinical portfolio further applies the company’s AI-powered technology platform to create transformative antibody-based therapeutics across targets with different MOAs.

A summary of pipeline progress and recent corporate highlights is below:

ADG116: This NEObody program, targeting a unique epitope of CTLA-4, is being evaluated in patients with advanced/metastatic solid tumors. ADG116 is designed to provide an enhanced efficacy profile by potent Treg depletion in the TME and to maintain its physiological function by soft ligand blocking to address safety concerns associated with existing CTLA-4 therapeutics.

·Advanced the global phase 1b/2 trial evaluating ADG116 as monotherapy (ADG116-1003) in dose escalation and dose expansion in targeted tumors.

·Presented clinical data at ESMO (Free ESMO Whitepaper)-IO 2021 in December from the dose-escalation part of the ADG116 monotherapy trial. Results showed strong safety profile and early signals of efficacy profile in a heavily pre-treated patient population with advanced metastatic diseases, including dose-dependent T-cell activation and tumor suppression in treatment-resistant "cold" and "warm" tumors such as pancreatic, ovarian and renal cell cancers.

oADG116 monotherapy was well-tolerated up to 10 mg/kg with primarily Grade 1 and limited Grade 2 treatment-related adverse events (TRAEs) observed in 25 patients (data cut off was October 15, 2021); a single rash (Grade 3) and dose limiting toxicity event (Grade 4 hyperglycemia) occurred in a patient with renal cell carcinoma who relapsed on nivolumab. This safety profile compared favorably to the benchmark antibody in a comparable dose range.

oDose escalation at 10 mg/kg is now complete and dose expansion is ongoing at 10 mg/kg in this monotherapy trial (ADG116-1003). No DLTs have occurred for additional patients at the ongoing 10 mg/kg dose level.

·Initiated dose escalation in phase 1 trial in China evaluating ADG116 as monotherapy (ADG116-1002) in patients with advanced/metastatic solid tumors.

·Initiated dose escalation of ADG116 in combination with either anti-PD-1 (toripalimab) or anti-CD137 (ADG106) therapy in patients with advanced/metastatic solid tumors.

·Initiated the global phase 1b/2 trial (ADG116-P001 / KEYNOTE-C97) following FDA clearance to evaluate ADG116 in combination with anti-PD-1 antibody, pembrolizumab in patients with advanced/metastatic solid tumors at multiple sites in the U.S. and Asia Pacific.

ADG126: This SAFEbody program applies precision masking technology to ADG116 for conditional activation in the TME to expand the therapeutic index and to further address safety concerns with existing CTLA-4 therapies. ADG126 is designed to provide enhanced safety and efficacy profiles due to the combination of the potent Treg depletion in the TME and soft ligand blocking.

·Following completion of dose escalation up to 10 mg/kg, initiated monotherapy dose expansion of an ongoing global phase 1b/2 clinical trial evaluating the safety profile and tolerability of ADG126 in patients with advanced/metastatic solid tumors (ADG126-1001).

oIn dose escalation cohorts, ADG126 was well tolerated with no dose-limiting toxicities up to 10 mg/kg even in patients who received more than four cycles. Following evaluation of data by a Safety Review Committee (SRC), dose expansion was approved at 10 mg/kg and initiated in both "warm" and "cold" tumor types.

oPatients have received multiple cycles with continuous dosing, and favorable pharmacokinetic and pharmacodynamic activity compared to ADG116 has been observed. ADG126 has consistently shown a potential best-in-class profile, which is supported by preclinical evaluation, including GLP toxicology data, and enabled by the broad species cross-reactivity of ADG126.

oThe ADG126 clinical results have been submitted for presentation at the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting.

·Initiated a global phase 1b/2 trial (ADG126-P001 / KEYNOTE-C98) following FDA clearance to evaluate ADG126 in combination with pembrolizumab in patients with advanced/metastatic solid tumors at multiple sites in the U.S. and Asia Pacific.

·ADG106: This NEObody program is a fully human ligand-blocking, agonistic anti-CD137 IgG4 monoclonal antibody (mAb) that is being evaluated in patients with advanced solid tumors and/or non-Hodgkin’s lymphoma.

·Initiated dose escalation for the phase 1b/2 clinical trial in Singapore (ADG106-T6001) evaluating ADG106 in combination with the anti-PD-1 antibody, nivolumab, for patients with advanced non-small cell lung cancer (NSCLC) who have progressed after prior treatment. The investigator-initiated trial is being conducted at the National University Cancer Institute, Singapore and the National Cancer Centre Singapore, in collaboration with the Singapore Translational Cancer Consortium.

·As noted above, initiated a dose escalation cohort in the global phase 1b/2 trial (ADG116-1003) evaluating ADG106 in combination with ADG116.

·Advanced the phase 1b/2 trial (ADG106-1008) in China evaluating safety and preliminary efficacy profiles of ADG106 in combination with toripalimab, an approved anti-PD-1.

·In December 2021, presented data at ESMO (Free ESMO Whitepaper)-IO 2021 on the biomarker kinetics for ADG106 as a monotherapy or combined with toripalimab. The combination of ADG106 with toripalimab resulted in a 2-fold synergistic effect for immune activation compared to ADG106 monotherapy, even amongst patients who failed prior anti-PD-1 and CTLA-4 therapies.

Preclinical Discovery Programs: The company continues to expand its preclinical pipeline by applying its three-body technology platforms – NEObody, SAFEbody and POWERbody – across modalities. New POWERbody candidates are designed to unleash the efficacy of a therapeutic through Fc-engineering, drug conjugation, or T-cell engagement, while securing safety by precision masking with SAFEbody technology. Thus, POWERbody candidates incorporate SAFEbody precision masking technology.

·In November 2021, unveiled preclinical data at the 63rd ASH (Free ASH Whitepaper) Annual Meeting & Exposition on two highly differentiated programs for transformative therapies for novel or validated targets: anti-CD47 (ADG153) and CD20xCD3 (ADG152).

oADG153, an anti-CD47 SAFEbody in IgG1 format, introduces IgG1-mediated effects for potent tumor killing with a compelling safety profile, minimal red blood cell (RBC)-related and antigen sink liabilities, and 8-fold prolonged half-life in comparison with benchmark clinical antibodies in the IgG4 subclass. By integrating efficacy and safety profiles, as well as prolonged pharmacokinetics into one single modality, anti-CD47 ADG153 IgG1 SAFEbody or ADG153 has a potential best-in-class profile.

oADG152, is a CD20xCD3 POWERbody integrating the company’s proprietary bispecific TCE platform with its precision masking technology. In preclinical mouse xenograft tumor models, ADG152 has demonstrated strong and sustained anti-tumor activity; in comparison with a benchmarked antibody in clinical development, improved safety with cytokine release control even at a 100-fold higher dose than the benchmarked antibody analog and 2- to 3-fold prolonged half-life than the benchmarked antibody analog in exploratory preclinical monkey studies.

·Announced presentation of four posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022. At AACR (Free AACR Whitepaper), presentations will show the potential best-in-class profiles for three differentiated preclinical product candidates in IND-enabling studies: ADG138, ADG206 and ADG153, which all three apply the SAFEbody precision masking technology. The fourth presentation introduces a new capability for the company’s proprietary bispecific TCEs with CD28.

oADG138 is a novel HER2×CD3 POWERbody integrating a bispecific TCE with precision masking technology to control CRS and on-target off-tumor toxicity for single agent and combination therapies in HER2-expressing solid tumors.

oADG206, is an Fc-engineered anti-CD137 agonistic POWERbody with tailor-made efficacy and safety profiles by strong crosslinking and tumor selective activation for single agent and combinational cancer immunotherapy.

oThe proprietary tumor-targeted CD28 bispecific POWERbody platform is designed for safe and synergistic T cell-mediated immunotherapy candidate.

oAdditional data will be presented supporting the advancement of the anti-CD47 antibody ADG153 into clinical development for both hematologic and solid tumor indications.

·During 2022, Adagene plans to submit an IND or equivalent for two POWERbody product candidates: ADG206 and ADG153.

Collaborations:

·Established an exclusive technology licensing agreement with Sanofi in March 2022 to generate novel masked monoclonal and bispecific candidate antibodies, with a potential transaction value of US$2.5 billion. Under the terms of the agreement, Adagene will be responsible for early-stage research activities to develop masked versions of Sanofi provided candidate antibodies, using Adagene’s SAFEbody technology. Sanofi will be solely responsible for later stage research and all clinical, product development and commercialization activities. The collaboration includes an upfront payment of US$17.5 million for the initial two programs (US$8.75 million per program), an option fee for two additional programs, potential milestone payments of up to US$2.5 billion (US$625 million per program), and tiered royalties.

·Achieved a US$3 million milestone in December 2021 for the successful nomination of lead SAFEbody candidates in a technology licensing agreement with Exelixis, established in February 2021 to develop SAFEbody novel masked antibody-drug conjugate candidates. Terms of the agreement included an upfront payment of US$11 million for 2 programs (US$5.5 million per program), potential milestone payments of up to US$780 million (US$390 million per program), and tiered royalties.

·Finalized clinical trial collaboration and supply agreements with Merck to evaluate pembrolizumab in combination therapy with all three wholly-owned clinical candidates.

·Advanced global partnerships and collaboration with Sanjin and Dragon Boat Biopharmaceutical for two antibodies out-licensed in Greater China, including an anti-PD-L1 (ADG104) in phase 2 development, and a novel anti-CSF-1R (ADG125/BC006) in phase 1.

Corporate Updates:

·In light of the Holding Foreign Companies Accountable Act (HFCAA), Adagene is proactively evaluating additional business processes and control changes to meet the requirements of the HFCAA and intends to leverage its flexible, global infrastructure and its focus on developing highly differentiated therapeutics for patients worldwide.

·Liu Yuwen was appointed as an independent board member and member of the Audit Committee, to support the company’s corporate development. Liu Yuwen is a leading advocate for the biotechnology, biopharmaceutical and medical technology industries, with over 20 years as an entrepreneur, advisor and investor.

·The company has expanded and strengthened its leadership team with recent hires across strategy, clinical, regulatory, communications and CMC functions.

EXPECTED 2022 MILESTONES & OUTLOOK

Adagene has previously provided its outlook for 2022, including planned advancement of both its clinical product candidates and preclinical portfolio. The company recently achieved its goal to complete a major collaboration following the Sanofi licensing agreement, and it continues to work towards strategic development collaborations for its pipeline. Additional milestones and expected progress during 2022 include:

·Demonstrate single-agent activity for anti-CTLA-4 programs (ADG116, ADG126) in heavily pretreated patients with "warm" and "cold" tumors.

·Demonstrate potential best-in-class safety and preliminary efficacy profiles for anti-CTLA-4 programs with anti-PD-1 therapy.

·Evaluate the profile for novel combinations of wholly owned anti-CD137 (ADG106) with either anti-CTLA-4 or anti-PD-1 therapy.

·Submit filings to advance at least two candidates to clinic, and expand programs into IND-enabling phase.

·Continue efficient discovery operations, with more than 50 projects underway.

FULL-YEAR 2021 FINANCIAL HIGHLIGHTS

Cash and Cash Equivalents:

Cash and cash equivalents were US$174.4 million as of December 31, 2021, compared to US$75.2 million as of December 31, 2020. The increase was mainly due to net proceeds of US$145.9 million from the company’s Initial Public Offering in February 2021. Further, the year-end 2021 cash balance does not include the US$3 million milestone payment from Exelixis received in January 2022, or the expected US$17.5 million upfront payment from Sanofi for the recently announced technology licensing collaboration.

Net Revenue:

Net revenue in 2021 was US$10.2 million compared to US$0.7 million in 2020. The increase was due to recognition of US$8.5 million from the collaboration and technology license agreement with Exelixis and a payment of US$1.2 million from Dragon Boat Biopharmaceuticals, a subsidiary of Sanjin, related to the companies’ collaboration to develop antibody-based therapies. Due to the Exelixis collaboration, contract liabilities were US$5.5 million as of December 31, 2021, compared to US$0.7 million as of December 31, 2020.

Research and Development (R&D) Expenses:

R&D expenses were US$68.1 million for the year ended December 31, 2021, compared to US$33.5 million for the same period in 2020. The increase in R&D was primarily due to an increase in personnel, including non-cash share-based compensation of US$13.6 million, and greater preclinical testing, clinical activities and CMC activities (provided by related parties and third parties) associated with the company’s three clinical candidates and five preclinical programs in the IND-enabling phase.

General and Administrative (G&A) Expenses:

G&A expenses were US$14.4 million for the year ended December 31, 2021, compared to US$10.3 million for the same period in 2020. The increase was primarily due to an increase in personnel, professional fees and office-related expenses.

Net Loss:

The Company reported a net loss of US$73.2 million and US$42.4 million for the full year ended December 31, 2021, and 2020, or (US$1.46) and (US$2.67) per ordinary share on diluted basis, respectively. The 2021 net loss was higher largely due to increases in clinical, operating and CMC activities.

Non-GAAP Net Loss:

Non-GAAP net loss, which is defined as net loss attributable to ordinary shareholders for the period after excluding (i) share-based compensation expenses and (ii) accretion of convertible redeemable preferred shares to redemption value. The Non-GAAP net loss was US$54.5 million for the year ended December 31, 2021, compared to US$32.3 million for the same period in 2020. Please refer to the section in this press release titled "Reconciliation of GAAP and Non-GAAP Results" for details.

Non-GAAP Financial Measures

The Company uses non-GAAP net loss and non-GAAP net loss per ordinary shares for the year, which are non-GAAP financial measures, in evaluating its operating results and for financial and operational decision-making purposes. The Company believes that non-GAAP net loss and non-GAAP net loss per ordinary shares for the year help identify underlying trends in the Company’s business that could otherwise be distorted by the effect of certain expenses that the Company includes in its loss for the year. The Company believes that non-GAAP net loss and non-GAAP net loss per ordinary shares for the year provide useful information about its results of operations, enhances the overall understanding of its past performance and future prospects and allows for greater visibility with respect to key metrics used by its management in its financial and operational decision-making.

Non-GAAP net loss and non-GAAP net loss per ordinary shares for the year should not be considered in isolation or construed as an alternative to operating profit, loss for the year or any other measure of performance or as an indicator of its operating performance. Investors are encouraged to review non-GAAP net loss and non-GAAP net loss per ordinary shares for the year and the reconciliation to their most directly comparable GAAP measures. Non-GAAP net loss and non-GAAP net loss per ordinary shares for the year here may not be comparable to similarly titled measures presented by other companies. Other companies may calculate similarly titled measures differently, limiting their usefulness as comparative measures to the Company’s data. The Company encourages investors and others to review its financial information in its entirety and not rely on a single financial measure.

Non-GAAP net loss and non-GAAP net loss per ordinary shares for the year represent net loss attributable to ordinary shareholders for the year excluding (i) share-based compensation expenses, and (ii) accretion of convertible redeemable preferred shares to redemption value. Share-based compensation expense is a non-cash expense arising from the grant of stock-based awards to employees. The Company believes that the exclusion of share-based compensation expenses from the net loss in the Reconciliation of GAAP and Non-GAAP Results assists management and investors in making meaningful period-to-period comparisons in the Company’s operating performance or peer group comparisons because (i) the amount of share-based compensation expenses in any specific period may not directly correlate to the Company’s underlying performance, (ii) such expenses can vary significantly between periods as a result of the timing of grants of new stock-based awards, and (iii) other companies may use different forms of employee compensation or different valuation methodologies for their share-based compensation.

Please see the "Reconciliation of GAAP and Non-GAAP Results" included in this press release for a full reconciliation of non-GAAP net loss and non-GAAP net loss per ordinary shares for the year for the year to net loss attributable to ordinary shareholders for the year/period.

On March 31, 2022 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in cell and gene therapy, reported a business and strategy update and reported 2021 financial results (Press release, Abeona Therapeutics, MAR 31, 2022, View Source [SID1234611234]).

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"We are committed to developing novel cell and gene therapies for patients with rare diseases with no approved treatment options," said Vish Seshadri, Ph.D., Chief Executive Officer of Abeona. "We are focused on EB-101 and, having recently achieved target enrollment in our pivotal Phase 3 VIITAL study, have increased confidence that we will share topline results in the third quarter of 2022. We also expect animal proof-of-concept data from our preclinical eye programs beginning in the second half of 2022 that could support pre-IND meetings with the FDA. We believe the strategic steps announced today reflect the operating discipline needed to extend our cash runway beyond these near-term catalysts."

Strategy and Business Update

●Following a comprehensive portfolio review, Abeona announced today that it is focusing research and development (R&D) resources on the topline data readout for its EB-101 pivotal Phase 3 VIITAL study while the Company actively pursues a potential commercialization partner for EB-101.
●Target enrollment was achieved in Abeona’s EB-101 pivotal Phase 3 VIITAL study for recessive dystrophic epidermolysis bullosa (RDEB). The Company anticipates topline results for the co-primary endpoints related to wound healing and pain reduction measured at 24 weeks post treatment in the third quarter of 2022. Abeona received positive feedback from the U.S. Food and Drug Administration (FDA) related to a Type B meeting on the proposed Chemistry, Manufacturing and Controls (CMC) requirements of the EB-101 development program, gaining alignment on the characterization and validation plans that could support a potential Biologics License Application (BLA) for EB-101 in RDEB.

●In connection with its shift in priorities, the Company has intensified its pursuit of a strategic partnership to take over development activities for ABO-102, and has ceased build-out of additional AAV manufacturing space. As part of the FDA’s feedback on the Transpher A Statistical Analysis Plan (SAP) in January 2022, the agency recommended that all participants be followed to an age of at least 60 months, which would shift timing of the neurocognitive outcomes data readout to late-2024/early-2025, as compared to the Company’s prior projection of the second quarter of 2023.
●As part of the Company’s portfolio prioritization, Abeona will discontinue development of ABO-101 for MPS IIIB.
●Abeona plans to continue development of AAV-based gene therapies designed to treat ophthalmic and other diseases and next-generation AAV-based gene therapies using the novel AIM capsid platform and internal AAV vector research programs. Abeona will present results from testing of novel AAV capsids in non-human primates at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting being held on May 1-4, 2022 in Denver, CO. The preclinical data could support pre-IND meetings with the FDA for Abeona’s undisclosed eye gene therapy indications. The Company previously reported preclinical data showing the potential for AIM AAV vectors to efficiently target the photoreceptor and retinal epithelium cell layers after intravitreal injection, creating the potential for new pipeline candidates that can address multiple ophthalmic disorders.
●The strategic changes will reduce the Company’s operating expenses and extend the estimated runway of current cash resources to mid-2023.
●In December 2021, Abeona raised approximately $17.5 million in aggregate gross proceeds, before underwriting discounts and commissions and other offering expenses, from an underwritten public offering of common stock.
●Joseph Vazzano was appointed as Chief Financial Officer (CFO) at Abeona, and will serve as the Company’s principal financial officer and principal accounting officer effective March 31, 2022. Mr. Vazzano previously served as CFO of Avenue Therapeutics, Inc. (Nasdaq: ATXI), where he secured multiple equity financings for Avenue and served in a leadership role for signing a complex, two-stage acquisition of the company with future contingent value rights.

Full Year 2021 Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $50.9 million as of December 31, 2021, compared to $96.0 million as of December 31, 2020. Net cash used in operating activities was $65.7 million for the full year of 2021, including a $20 million payment in November 2021 in accordance with a settlement agreement with REGENXBIO. Net cash used in operating activities was $35.0 million for the full year of 2020.

License and other revenues for the full year of 2021 were $3.0 million, compared to $10.0 million in 2020. The revenue in 2021 resulted from a clinical milestone achieved in December 2021 under a sublicense agreement with Taysha Gene Therapies for ABO-202 for CLN1 disease.

R&D expenses were $34.3 million for the full year of 2021, compared to $30.1 million in 2020. General and administrative (G&A) expenses were $22.8 million for the full year of 2021, compared to $23.8 million in 2020.

Net loss was $84.9 million for the full year of 2021, or a $0.86 basic and diluted loss per common share as compared to a net loss of $84.2 million, or a $0.91 basic and diluted loss per common share, in 2020. The net loss in 2021 included a non-cash goodwill impairment charge of $32.5 million. The impairment charge has no impact on the Company’s cash position, cash flow from operating activities, and does not have any impact on future operations.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast today, Thursday, March 31, 2022 at 8:30 a.m. ET, to discuss its full year 2021 financial results and business update. To access the call, dial 877-545-0320 (U.S. toll-free) or 973-528-0002 (international) and Entry Code: 851784 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

On March 31, 2022 Fresenius Kabi reported that it has agreed to acquire a stake of 55% of mAbxience Holding S.L. ("mAbxience") (Press release, Fresenius, MAR 31, 2022, View Source [SID1234611221]). The purchase price will be a combination of €495 million upfront payment and milestone payments, strictly tied to the achievement of commercial and development targets. The contractual provisions also include a put / call option scheme regarding the current owners’ remaining shares in mAbxience (45%).

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mAbxience is a leading international biopharmaceutical company, focused on the rapidly developing biosimilars market. The company was founded in 2010 by Dr. Hugo Sigman and Dr. Silvia Gold as the biotechnology division of Insud Pharma S.L. mAbxience has established itself as a leader in the development and manufacturing of biological drugs, with two commercialized biosimilar products (Rituximab and Bevacizumab) and a mid-single-digit number of molecules across immunology and oncology expected to be launched globally in the years 2024 to 2029. This is supported by internal R&D laboratories and state-of-the-art manufacturing facilities in Spain and Argentina. In addition to highly competitive production costs for the internal programs, the manufacturing platform allows mAbxience to offer third party biological CDMO services, including a recent contract with AstraZeneca to produce the drug substance for its COVID-19 vaccine in Latin America. The company currently employs approximately 600 staff and generated sales of approx. €255 million in 2021.

The acquisition of a majority stake in mAbxience follows Fresenius Kabi’s recently unveiled Vision 2026 strategy, delivering on one of the core growth vectors – to "Broaden Biopharma" – by expanding along the value chain and further enhancing the existing Fresenius Kabi biosimilars pipeline.

Fresenius Kabi expects, through its in-house biosimilars programs and through its investment in mAbxience, to capture an overproportionate share of the underlying rapid growth in the biopharmaceutical market. Fresenius Kabi’s footprint in biopharmaceuticals will be significantly strengthened by broadening its biosimilars portfolio and by gaining access to the distinctive manufacturing capabilities of mAbxience. It will also allow Fresenius Kabi to provide end-to-end integrated biopharmaceutical solutions for customers from its state-of-the-art facilities.

mAbxience operates three state-of-the-art facilities for the production of biologic drug substance. This addresses a critical gap in Fresenius Kabi’s value chain, adding flexible, single-use biologic drug substance capacity that can be leveraged to provide competitive cost of production for the enlarged biosimilars portfolio. This manufacturing capability also offers end-to-end integrated biopharmaceutical solutions for customers and thus establishes a strategic foothold for Fresenius Kabi in the fast-growing biologic CDMO sector, complementing the existing small molecule API and fill & finish operations.

Once completed, the transaction is expected to deliver material operating and cost synergies for Fresenius Kabi, primarily driven by leveraging mAbxience’s manufacturing capabilities for Fresenius Kabi’s existing biosimilars business.

The transaction remains subject to regulatory approvals and other customary closing conditions and is expected to close by mid-2022.

Ivenix strengthens Fresenius Kabi’s MedTech business and accelerates growth

Delivers on core growth vector "Expand MedTech" of Vision 2026
Provides next-generation infusion therapy platform for U.S. market
Complements Fresenius Kabi’s global infusion therapy offering
Provides Fresenius Kabi with key capabilities in hospital connectivity and creates new options for growth of MedTech business
Significant scale and growth synergies expected
Fresenius Kabi announced today that it has agreed to acquire Ivenix, Inc. („Ivenix"), a specialized infusion therapy company. The purchase price will be a combination of US$240 million upfront payment and milestone payments, strictly linked to the achievement of commercial and operating targets.

Ivenix is a privately held company based in North Andover, Massachusetts, USA. The company has developed the technologically most advanced infusion system including a large volume pump ("LVP") with administration sets, infusion management software tools, applications and analytics to inform care and advance efficiency. The Ivenix Infusion System’s innovative design and architecture sets a new standard in infusion safety, simplicity and interoperability. The system is centred around the patient and clinician and is designed to reduce infusion-related errors and drive down the total cost of ownership. After having received the U.S. Food and Drug Administration’s (FDA) approval, the Ivenix Infusion System was successfully launched in late 2021.

Ivenix’ Infusion System provides access to attractive growth potential for Fresenius Kabi in the large and growing infusion therapy market. The combination of Ivenix’ leading hardware and software products with Fresenius Kabi’s offerings in intravenous fluids and infusion devices will create a comprehensive and leading portfolio of premium products, forming a strong basis to enable sustainable growth in the high-value MedTech space.

The transaction is subject to regulatory approvals and other customary closing conditions and is expected to close by mid-2022.

Financing and implications on Group financials

mAbxience is expected to be accretive to Group cash earnings per share (earnings before amortization and integration costs) right after closing. Ivenix is expected to be neutral to Group cash earnings per share in 2025 and accretive from 2026 onwards.

Combined, these acquisitions are expected to be broadly neutral to Group cash earnings per share in 2022 and accretive as of 2023.

The transactions are currently expected to be financed by cash flow and available liquidity.

Conference Call

A telephone conference on the acquisition of a majority stake in mAbxience Holding S.L. and the acquisition of Ivenix, Inc. will be held on March 31, 2022 at 1:30 p.m. CEST (7:30 a.m. EDT). All investors are cordially invited to follow the conference call in a live broadcast over the Internet at www.fresenius.com/investors. Following the call, a replay will be available on our website.

On March 31, 2022 TILT Biotherapeutics, a clinical-stage biotechnology company developing oncolytic immunotherapy for enabling therapies based on T cells, such as immune checkpoint inhibitors and adoptive cell therapies, reported that positive interim progress and safety data from its phase 1 clinical trials in metastatic melanoma (T215 – the ‘TUNINTIL’ trial) and solid tumors (T115 – the ‘TUNIMO’ trial) (Press release, TILT Biotherapeutics, MAR 31, 2022, View Source [SID1234611203]). These two trials, with patients in Denmark, France, and Finland, are of the company’s oncolytic immunotherapy asset TILT-123, designed to stimulate T-cells to better fight cancer.

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TILT Biotherapeutics’ CEO, Akseli Hemminki, a biotech entrepreneur and cancer clinician who has personally treated almost 300 patients with ten different oncolytic viruses, said, "We are delighted that our phase 1 trial in solid tumors (T115), which consists of a total of 11 patients being treated to date, is progressing at pace. Our first-in-human melanoma (T215) trial is showing equally positive results, with 10 patients being treated and some now being treated under an extension protocol. I am thankful for the excellent work by the clinical teams. We are showing that the treatment is well tolerated, has prominent biological effects on tumors, and has the potential to increase the efficacy of immunotherapies, and deliver the anti-tumor benefits of armed oncolytic viruses."

The ‘TUNINTIL’ clinical trial (1) is a phase 1, open-label, dose-escalation study of the company’s oncolytic adenovirus coding for Tumor Necrosis Factor Alpha (TNF alpha) and Interleukin 2 (IL-2), known as TILT-123. A total of about 15 metastatic melanoma patients receive TILT-123 as an initial monotherapy over one month, followed by up to two administrations of tumor infiltrating lymphocytes (TILs) in the second month as well as ongoing consecutive doses of TILT-123, for up to 24 months.

The ‘TUNIMO’ clinical trial (2) is also a phase 1 trial of TILT-123. A total of 15-20 patients with solid tumors receive TILT-123 as a monotherapy over a three-month period, and, as with the TUNINTIL protocol, followed up by a 24- month extension period.

The primary objective of both trials is to evaluate the safety of TILT-123 and to deliver insights about the behavior of TILT-123 in humans, such as systemic tumor transduction following intravenous delivery and virus replication in the tumor, as well as immunological responses.

To date, most frequent adverse events across both trials have been fever, chills, and fatigue, consistent with the administration of an oncolytic adenoviral immunotherapy, with no treatment related serious adverse events.

The heart of TILT’s innovative approach revolves around the use of armed oncolytic adenoviruses, using cytokines and other molecules to boost the patient’s immune response to better enable it to find and destroy cancer cells. The company is advancing its pipeline of other assets and programs with TILT-123 towards further clinical trials, including in combination with the immune checkpoint inhibitors (3) (4).