On March 31, 2022 Celsion Corporation (NASDAQ: CLSN), a clinical-stage drug-development company focused on DNA-based immunotherapy and next-generation vaccines, reported financial results for the year ended December 31, 2021, and provided an update on clinical development programs with GEN-1, a DNA-based interleukin-12 (IL-12) immunotherapy in Phase II clinical development for the treatment of advanced-stage ovarian cancer and preclinical studies with PLACCINE, a proprietary, multivalent DNA plasmid technology utilizing synthetic, non-viral vaccine delivery vectors, being evaluated for superiority over the current generation of nucleic acid vaccines (Press release, Celsion, MAR 31, 2022, View Source [SID1234611238]).

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"GEN-1 continues to show momentum as patient enrollment in OVATION 2, our randomized Phase II study of advanced ovarian cancer patients, is now over 80%. Full enrollment is expected by the third quarter of 2022. We remain encouraged by surgical resection scores among patients being treated at the 100 mg/m² dose cohort in the Phase II OVATION 2 Study. Early reports from the first 39 patients who have undergone interval debulking surgery showed a 27% improvement in the surgical resection (R0) rate in the GEN-1 treatment arm over the control arm. A complete R0 is a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer

"At various vaccine conferences, we announced results from preclinical in vivo studies showing production of antibodies and cytotoxic T-cell response specific to the spike antigen of SARS-CoV-2 when immunizing BALB/c mice with our next-generation PLACCINE DNA vaccine," added Mr. Tardugno. "Our goal is to demonstrate the superiority of Celsion’s DNA vaccine over current mRNA vaccines with respect to the quality of immune response against SARS-CoV-2 variants, including a longer duration of immune response and a stable product at commercially favorable temperatures. To that end, we have engaged BIOQUAL, Inc., a preclinical testing contract research organization, to conduct a non-human primate (NHP) challenge study of our first of many vaccine candidates as protection against SARS-CoV-2. We expect first inoculation of NHP subjects to occur during the last week of March with the goal of generating important data to inform human clinical studies."

Recent Developments

GEN-1 Immunotherapy

Poster Presented at Cytokine-Based Cancer Immunotherapies Summit. In November 2021, the Company announced that Khursheed Anwer, Ph.D., executive vice president and chief science officer, made a presentation on the Company’s GEN-1 interleukin 12 (IL-12) immunotherapy program at the Cytokine-Based Cancer Immunotherapies Summit held in Boston on November 30 to December 2, 2021. Dr. Anwer’s presentation, was titled "A Non-Viral Gene Therapy Approach to IL-12 Delivery for The Treatment of Cancer." The Company was invited to submit a poster presentation which aligned with Dr. Anwer’s oral presentation on the GEN-1 IL-12 program.

In his presentation, Dr. Anwer discussed how local delivery of IL-12 without significant systemic toxicity is feasible with a non-viral gene therapy approach that involves administration of an IL-12 plasmid with a synthetic DNA delivery system. Dr. Anwer also discussed how weekly intraperitoneal administration of GEN-1 yields durable increases in IL-12 and IFN-y, and why repeated weekly administration of GEN-1 in combination with standard chemotherapy remodels the tumor immune environment to favor immune stimulation over immune suppression.

Dr. Anwer also participated in two panel discussions titled "What Do We Know & Where Do We Want to Go?" and "Side Effects – Mitigating Against Hypotension + Fever with Immune-Stimulating Agents (NK Cell Engagers, PD-1s, Cytokines, T-Cell Engagers) = Cytokine Release Syndrome (CRS)?"

Data Safety Monitoring Board’s Unanimous Recommendation to Continue Dosing Patients in the Phase II Portion of the OVATION 2 Study with GEN-1 in Advanced Ovarian Cancer. In February 2022, the Company announced that following a pre-planned interim safety review of 81 as treated patients randomized in the Phase I/II OVATION 2 Study with GEN-1 in advanced (Stage III/IV) ovarian cancer, the Data Safety Monitoring Board (DSMB) unanimously recommended that the OVATION 2 Study continue treating patients with the dose of 100 mg/m2. The DSMB also determined that safety is satisfactory with an acceptable risk/benefit, and that patients tolerate GEN-1 during a course of treatment that lasts up to six months. No dose-limiting toxicities were reported.

The Company also announced that over 75% of the projected 110 patients have been enrolled in the OVATION 2 Study. Interim clinical data from the first 39 patients who have undergone interval debulking surgery showed that the GEN-1 treatment arm is showing a 27% improvement in R0 surgical resection rate over the control arm. A complete tumor resection (R0) is a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed.

Vaccine Initiative

Oral Presentation on Celsion’s Ongoing Work with DNA-based Vaccines at International Vaccines Congress. In October 2021, the Company announced that Dr. Anwer presented at the International Vaccines Congress. His presentation was titled "Immunogenicity of DNA Vaccines based on Multicistronic Vectors and Synthetic DNA Delivery Systems" and can be viewed here.

Dr. Anwer discussed ongoing proof-of-concept studies in SARS-CoV-2 with the Company’s DNA-based vaccine approach utilizing its PLACCINE platform. PLACCINE, Celsion’s proprietary design for DNA vectors, encompasses molecular elements designed to improve the immune response by targeting multiple antigens of a pathogen or multiple mutants of the same antigen. Dr. Anwer also reviewed the PLACCINE technology and the production of a family of DNA vaccine vectors expressing one or more SARS-CoV-2 surface antigens as a proof-of-concept target, verified vector composition and demonstrated expression of the encoded genes.

Added Key Resources to the Company’s Vaccine Development Initiative. In October 2021, the Company announced the strengthening of its management team with a new hire and a promotion in its vaccine development program as follows:

●Carlo Iavarone, Ph.D. joined as Senior Director, Non-Clinical Research
●Subeena Sood, Ph.D. promoted to Senior Manager, Biology and Preclinical Studies

Dr. Iavarone is project leader for the PLACCINE vaccine initiative. He brings to Celsion more than 15 years of experience investigating and leading the development of vaccines, including molecular target identification and characterization of RNA vaccines. Most recently, from 2019 until 2021 he was a science advisor for both Guidepoint and Clora, providing input for a viral target and RNA vaccine delivery system. Dr. Iavarone joined GlaxoSmithKline in 2015 as a senior scientist studying small molecules and RNA vaccines in animal and human cell lines. From 2007 until 2015 he held positions of increasing responsibility at Novartis, including as a principal scientist for a melanoma vaccine project.

Dr. Sood is responsible for assay development and in vivo experiments for the PLACCINE DNA vaccine and gene therapy program. She has experience with several pharmaceutical companies in experiment design, pharmacological and biochemical assays, manufacturing process design and development, and optimization and implementation of Quality by Design. Dr. Sood joined Celsion as manager of animal research in 2019, where she has designed and conducted all preclinical research. Prior to Celsion, since 2017 she was a Formulation Scientist at Novocol Healthcare. From 2016 to 2017, Dr. Sood was a Research Associate at Nektar Therapeutics, and from 2015 to 2016 she was a Quality Control Chemist at Par Pharmaceuticals. She also worked in regenerative medicine as a Research Fellow at Medstar Heart Institute, Washington Hospital Center in Washington, D.C. from 2010 to 2013.

Proof of Concept Vaccine Candidate Advanced to Non-Human Primate Challenge Study Against SARS-CoV-2. In January 2022, the Company announced it had engaged BIOQUAL, Inc., a preclinical testing contract research organization, to conduct a non-human primate (NHP) challenge study with Celsion’s DNA-based approach for a SARS-CoV-2 vaccine. The NHP pilot study follows the generation of encouraging mouse data and will evaluate the Company’s lead vaccine formulations for safety, immunogenicity and protection against SARS-CoV-2.

In completed preclinical studies, Celsion demonstrated safe and efficient immune responses including IgG response, neutralizing antibodies and T-cell responses that parallel the activity of commercial vaccines following intramuscular (IM) administration of novel vaccine compositions expressing a single viral antigen. In addition, vector development has shown promise of neutralizing activity against a range of SARS-CoV-2 variants. Celsion’s novel DNA-based vaccines are based on a simple intramuscular injection that does not require viral encapsulation or special equipment for administration. Ongoing directional and technical guidance from our Vaccine Advisory Board, which is comprised of leaders in commercial vaccine development, virology, vector engineering and drug development, has been invaluable as we approach this critical advancement in our platform development program. We expect NHP studies to begin during the second quarter of 2022 with the goal of generating important data to inform human clinical studies.

Corporate Developments

Priced a $30 Million Registered Direct Offering of Convertible Redeemable Preferred Stock. In January 2022, the Company announced that it has entered into a securities purchase agreement with certain institutional investors to purchase 50,000 shares of Series A convertible redeemable preferred stock and 50,000 shares of Series B convertible redeemable preferred stock. Each share of Series A and Series B preferred stock has a purchase price of $285, representing an original issue discount of 5% of the $300 stated value of each share. Each share of Series A preferred stock is convertible into shares of Celsion’s common stock at an initial conversion price of $0.91 per share. Each share of Series B preferred stock is convertible into shares of Celsion’s common stock at an initial conversion price of $1.00 per share. Shares of the Series A and Series B preferred stock are convertible at the option of the holder at any time following the Company’s receipt of stockholder approval for a reverse stock split of the Company’s common stock. Celsion will be permitted to compel conversion of the Series A and Series B preferred stock after the fulfillment of certain conditions and subject to certain limitations. Total net proceeds from the offerings, before deducting the placement agent’s fees and other estimated offering expenses, is approximately $28.5 million.

The Series A and Series B preferred stock permit the holders thereof to vote together with the holders of the Company’s common stock on a proposal to effectuate a reverse stock split of the Company’s common stock at a special meeting of Company stockholders to be held on February 24, 2022. The Series A preferred stock permits the holder to vote on such proposal on an as converted to common stock basis. The Series B preferred stock permits the holder to cast 45,000 votes per share of Series B preferred stock on such proposal. The Series A and Series B preferred stock will not be permitted to vote on any other matter. The holders of the Series A and B preferred stock agreed not to transfer their shares of preferred stock until after the special meeting of Company stockholders. The holders of the Series A preferred stock agreed to vote their shares in favor of that proposal and the holders of the Series B preferred stock agreed to vote their shares in the same proportions as the shares of common stock and Series A preferred stock are voted on that proposal. The holders of the Series A and Series B preferred stock have the right to require the Company to redeem their shares of preferred stock for cash at 105% of the stated value of such shares commencing after the earlier of the Company’s stockholders’ approval of the reverse stock split and 90 days after the closing of the issuances of the Series A and Series B preferred stock and until 120 days after such closing.

On March 3, 2022, the Company redeemed for cash at a price equal to 105% of the $300 stated value per share all of its 50,000 outstanding shares of Series A Preferred Stock and its 50,000 Series B Preferred Stock. As a result, all shares of the Preferred Stock have been retired and are no longer outstanding and Celsion’s only class of outstanding stock is its common stock, par value $0.01 per share. Each share of common stock entitles the holder to one vote.

Announced Stock Consolidation. In February 2022, the Company announced that, as previously authorized by its shareholders, the Company implemented a consolidation (reverse stock split) of its outstanding Common Shares on the basis of one (1) new Common Share for every fifteen (15) currently outstanding Common Shares.

The new Common Shares was effective for trading purposes as of the commencement of trading on Tuesday, March 1, 2022, and will trade under a new CUSIP number 15117N 602. The Company’s ticker symbol, CLSN, remained unchanged. The Company has filed a Certificate of Amendment to its Certificate of Incorporation to effect the stock consolidation.

The new number of outstanding common shares is approximately 5.8 million shares. The number of authorized shares (112.5 million) and the par value per share ($0.01) were unchanged. The number of outstanding options and warrants were adjusted accordingly, with outstanding options being approximately 437,500 and outstanding warrants being approximately 168,500.

Received $1.4 Million in Non-Dilutive Funding from the Sale of its New Jersey State Net Operating Losses, with an Additional $3.5 Million Expected in 2022 – 2023. In February 2022, the Company announced it has received $1.4 million in net cash proceeds from the sale of approximately $1.5 million of its unused New Jersey net operating losses (NOLs). The NOL sales cover the tax year 2020 and are administered through the New Jersey Economic Development Authority’s (NJEDA) Technology Business Tax Certificate Transfer Program. The Company plans to sell an additional $3.5 million of unused New Jersey NOLs available to the Company under the program over the next 2 years.

The Technology Business Tax Certificate Transfer Program administered by the NJEDA enables qualified companies to sell up to $20 million of their unused New Jersey net operating losses and R&D tax credits to unaffiliated, profit-generating corporate taxpayers in the state of New Jersey. The economic development program is designed to allow technology and biotechnology companies with NOLs to turn their tax losses and credits into cash proceeds to fund more R&D, expand its workforce, and cover other allowable expenditures.

Financial Results for the Year Ended December 31, 2021

Celsion reported a net loss of $20.8 million ($3.83 per share) in 2021, compared with a net loss of $21.5 million ($10.08 per share) in 2020. Operating expenses were $21.5 million in 2021, which represented a $2.5 million (13%) increase from $19.0 million in 2020.

Net cash used for operating activities was $16.2 million in 2021, compared with $15.6 million in 2020. This was in line with the Company’s projected cash utilization for 2021 of approximately $17 million, or an average of approximately $4.25 million per quarter. The Company raised approximately $58.4 million in new financings during 2021 – (i) $6.9 million in gross proceeds from the use of its JonesTrading Capital on DemandTM financing facility, (ii) $35 million from a registered direct financing completed in January 2021, (iii) $15 million from a registered direct financing completed on April 5, 2021, and (iv) $1.5 million from warrant exercises. With $56.9 million in cash and cash equivalents, restricted cash, short-term investments and interest receivable at December 31, 2021, the Company has sufficient capital resources to fund its operations through the end of 2024.

Research and development expenses decreased $0.7 million from $11.3 million in 2020 to $10.6 million in 2021. Costs associated the OVATION 2 Study were consistent at $1.3 million in 2021 and 2020. Costs associated with the OPTIMA Study decreased to $1.0 million in 2021 compared to $2.2 million in 2020. In July 2020, the Company unblinded the OPTIMA Study at the recommendation of the DMC to halt the study due to futility. Other clinical and regulatory costs were $2.5 million in 2021 and 2020. R&D costs associated with the development of GEN-1 to support the OVATION 2 Study as well as development of the PLACCINE DNA vaccine technology platform increased to $4.3 million in 2021 compared to $3.1 million in the same period of 2020. CMC costs decreased to $1.5 million in 2021 compared to $2.1 million in 2020 due to the discontinuation of the ThermoDox clinical development program in primary liver cancer.

General and administrative expenses increased to $10.9 million in 2021 compared to $7.6 million in 2020. This increase is primarily attributable to higher non-cash stock compensation expense of approximately $1.3 million, an increase in professional fees of $1.5 million (largely legal fees to defend various suits filed after the announcement in July 2020 of the OPTIMA Phase III clinical results) and an increase in premiums for directors’ and officers’ insurance of $0.3 million in 2021 when compared to 2020.

Other expenses included the following:

●A non-cash credit of $1.6 million for the change in valuation of the earn-out milestone liability for the GEN-1 ovarian product candidate during 2021, compared with a non-cash charge of $1.3 million during 2020.
●A non-cash charge of $2.0 million related to the impairment of goodwill recorded in the fourth quarter of 2021 compared with a $2.4 million non-cash charge related to the impairment of certain in-process research and development assets related to the development of the Company’s GBM cancer product candidate in the third quarter of 2020.
●In connection with the Company’s venture debt facilities, the Company incurred interest expense of $0.6 million during 2021, compared with $1.3 million during 2020. In June 2021, the Company entered into a new $10.0 million loan facility with SVB, with a portion of the proceeds used to retire all outstanding indebtedness under the Company’s venture debt facility with Horizon Technology Finance Corporation. In connection with the termination of the Horizon Technology Financing Facility in the second quarter of 2021, the Company paid early termination and end of term charges to Horizon and recognized $0.2 million as a loss on debt extinguishment.

During the fourth quarter of 2021, the Company entered into an agreement to sell the approved portion of its New Jersey Net Operating Losses (NOLs) applied for in 2021 for $1.4 million. At December 31, 2021, the Company evaluated the valuation reserve for its tax net operating losses associated with its New Jersey NOLs, reduced the valuation reserve, and recognized $1.4 million as a deferred income tax asset from the sale of its New Jersey net operating losses and an income tax benefit. The Company completed the sale of these net operating losses in February of 2022. In 2020, the Company entered into an agreement to sell the approved portion of its New Jersey NOLs applied totaling $1.9 million. At December 31, 2020, the Company evaluated the valuation reserve for its tax net operating losses associated with its New Jersey NOLs, reduced the valuation reserve and recognized $1.9 million as a deferred income tax asset and an income tax benefit. The Company completed the sale of these net operating losses in May of 2021.

Conference Call

The Company is hosting a conference call to provide a business update, discuss 2021 financial results and answer questions at 11:00 a.m. EDT today. To participate in the call, interested parties may dial 1-888-394-8218 (Toll-Free/North America) or +1-323-794-2588 (International/Toll) and ask for the Celsion Corporation 2021 Earnings Call (Conference Code: 7900710) to register ten minutes before the call is scheduled to begin. The call will also be broadcast live on the internet at www.celsion.com. The call will be archived for replay on Thursday, March 31, 2022, and will remain available until April 14, 2022. The replay can be accessed at +1-719-457-0820 or 1-888-203-1112 using Conference ID: 7900710. An audio replay of the call will also be available on the Company’s website, www.celsion.com, for 90 days after 2:00 p.m. ET Thursday, March 31, 2022.

On March 31, 2022 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, reported that William G. Rice, Ph.D., Chairman, President and Chief Executive Officer, will participate in a panel to discuss new approaches of treating acute myeloid leukemias (AML) at Canaccord Genuity’s 2022 Horizons in Oncology Virtual Conference (Press release, Aptose Biosciences, MAR 31, 2022, View Source [SID1234611237]):

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Canaccord Genuity’s 2021 Horizons in Oncology Virtual Conference

•Panel: Attacking AML: New Approaches

Date: Thursday, April 14, 2022
Time: 1:30 – 2:30 PM ET
Format: Panel Moderated by John Newman, Ph.D., Managing Director, Biotechnology Analyst
Webcast: Link
The audio webcast also will be accessible through the Aptose website at www.aptose.com and will be archived shortly after the live events.

The Aptose management team will be hosting 1×1 investor meetings during the event.

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.