On March 29, 2023 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported financial results for the full year of 2022 and provided an update on progress toward achieving key corporate objectives (Press release, Abeona Therapeutics, MAR 29, 2023, View Source [SID1234629488]). The Company will host a conference call and webcast today, March 29, 2023, at 8:30 a.m. ET, to discuss its financial results and business update.

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"Since announcing positive topline results for the Phase 3 VIITAL study late last year, we have had multiple recent collaborative interactions with the FDA, and are continuing to work diligently toward completing and submitting our Biologics License Application for EB-101 in recessive dystrophic epidermolysis bullosa (RDEB)," said Vish Seshadri, Chief Executive Officer of Abeona. "We believe the VIITAL study results highlight the value that EB-101 could bring to RDEB patients as does the encouraging feedback we have received from our initial interactions with payors. In addition, we continue to make progress in our earlier stage programs that have the potential for treating serious eye diseases with new adeno-associated virus-based gene therapies, and we expect to keep advancing these toward an Investigational New Drug filing and clinical trials."

Fourth Quarter and Recent Operating Highlights, and Upcoming Events

EB-101 for recessive dystrophic epidermolysis bullosa (RDEB)

● In November 2022, Abeona announced positive topline results with both co-primary endpoints and all other endpoints met in the pivotal Phase 3 VIITAL study of investigational EB-101 in RDEB. Additional data from the VIITAL study has been accepted for an oral presentation at the International Societies for Investigative Dermatology (ISID) Meeting in May 2023. Furthermore, durability of EB-101 clinical outcomes was published in the Orphanet Journal of Rare Diseases, demonstrating sustained wound healing with a mean follow up of 5.9 years and symptomatic relief based on long term follow up of the Phase 1/2a study.
● Abeona continues to make progress in the submission process for a Biologics License Application (BLA) for EB-101 to the U.S. Food and Drug Administration (FDA). The Company currently plans to submit a BLA for EB-101 in late second quarter to early third quarter of 2023. If the BLA is approved, Abeona may be eligible for a Priority Review Voucher (PRV), which can be used to receive expedited review by the FDA of a subsequent marketing application for a different product or sold to another company.
● In the first quarter of 2023, as part of its commercial planning, the Company initiated discussions with stakeholders across the healthcare system, including public and private payors, and healthcare providers to better understand market access and potential pricing for EB-101

Preclinical programs

● Abeona’s preclinical programs are investigating the use of novel adeno-associated virus (AAV) capsids in AAV-based therapies for serious eye diseases, including ABO-504 for Stargardt disease, ABO-503 for X-linked retinoschisis (XLRS) and ABO-505 for autosomal dominant optic atrophy (ADOA).
● In 2022, Abeona evaluated the ability of its gene constructs and capsids to deliver and express the recombinant protein in target eye tissues and rescue mutant phenotypes in mouse disease models.
● The Company has started to submit pre-Investigational New Drug (IND) meeting requests to the FDA for these candidates and anticipates meetings to take place in the second quarter of 2023.
● The Company expects to present new preclinical data from these programs at a scientific congress in the second quarter of 2023.

Corporate highlights

● The Company enhanced its senior management team with three experienced biotechnology industry leaders. Abeona appointed Dmitriy Grachev, M.D., Ph.D., as Chief Medical Officer and Madhav Vasanthavada, Ph.D., M.B.A. as Vice President, Business Development. In addition, Amanda Moore, MSHS, was promoted to the role of Vice President, Program Leadership and Clinical Operations. Dr. Grachev has over 20 years of industry experience across multiple therapeutic areas including dermatology, ophthalmology, and oncology, and has multiple global drug approvals while leading clinical development programs at pharmaceutical and biotechnology organizations. Dr. Vasanthavada was most recently at Bristol Myers Squibb, where he led Global Marketing for the Global Car T Cell Therapy Franchise. Ms. Moore most recently served as Senior Director, Head of Program Leadership.
● In November 2022, the Company successfully completed a $35.0 million private placement financing with participation from new and existing institutional investors.

Full Year 2022 Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $52.5 million as of December 31, 2022, compared to $50.9 million as of December 31, 2021. Net cash used in operating activities was $43.5 million for the full year of 2022, compared to $65.7 million in the full year of 2021. Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments are sufficient resources to fund operations into the third quarter of 2024.

"With our existing cash resources, we believe we are funded through the anticipated timing for EB-101 BLA potential approval," said Joe Vazzano, Chief Financial Officer of Abeona.

License and other revenues for the year ended December 31, 2022 were $1.4 million, as compared to $3.0 million for the same period of 2021. The revenues in 2022 and in 2021 primarily represent clinical milestone payments under two licensing agreements with Taysha Gene Therapies for investigational AAV-based gene therapies for Rett syndrome and CLN1 disease (also known as infantile Batten disease), respectively.

Research and development expenses for the full year ended December 31, 2022 were $29.0 million, compared to $38.7 million for the full year ended December 31, 2021. General and administrative expenses were $17.3 million for the full year ended December 31, 2022, compared to $21.6 million for the year ended December 31, 2021. Net loss attributable to common shareholders for the full year ended December 31, 2022 was $43.5 million, or $5.53 loss per common share as compared to $84.9 million, or $21.57 loss per common share, for the full year of 2021

Conference Call Details

Abeona Therapeutics will host a conference call and webcast today, March 29, 2023, at 8:30 a.m. ET. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 195981 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 29, 2023 Avalo Therapeutics, Inc. (Nasdaq: AVTX), reported business updates and year-end financial results for 2022 (Press release, Avalo Therapeutics, MAR 29, 2023, View Source [SID1234629483]).

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"We made significant strategic and operational progress over the past year, by executing our goals of streamlining the pipeline to the most promising assets/trials, implementing corresponding infrastructure cost reductions, executing operationally and extending the cash runway through business development transactions," said Dr. Garry Neil, Chief Executive Officer and Chairman of the Board. "2023 will be a transformational year for Avalo as we look forward to our upcoming data readout of the Phase 2 PEAK trial of AVTX-002 in patients with NEA in the second quarter."

Program Updates and Milestones:

•AVTX-002: Anti-LIGHT monoclonal antibody (mAb) targeting immune-inflammatory diseases.
◦NEA: Avalo completed enrollment of the Phase 2 PEAK trial evaluating the safety and efficacy of AVTX-002 in 91 patients with NEA. Topline data expected in the second quarter of 2023.

•AVTX-008: B and T Lymphocyte Attenuator (BTLA) agonist fusion protein targeting immune dysregulation disorders.
◦Avalo identified a lead molecule and is currently evaluating several immune dysregulation disorders, with a target IND submission planned in 2024.

•AVTX-803: Fucose replacement for leukocyte adhesion deficiency type II (LAD II, also known as SLC35C1-CDG), a congenital disorder of glycosylation (CDG).
◦Timing of pivotal data from the pivotal LADDER trial evaluating the safety and efficacy of AVTX-803 in approximately 2 patients with LAD II is under evaluation.

2022 Financial Update:

Avalo had $13.2 million in cash and cash equivalents as of December 31, 2022, representing a $41.4 million decrease compared to December 31, 2021. The decrease was primarily driven by operating expenditures and a $15.0 million partial prepayment under its loan and security agreement, partially offset by $19.5 million of upfront payments received from out-license and business development activity. In February 2023, we closed an equity financing in which we raised approximately $13.7 million in net proceeds.

Total net revenues increased $12.7 million for the year ended December 31, 2022. The increase was driven by $14.5 million of upfront consideration received pursuant to the out-license of AVTX-007. Total operating expenses decreased $32.0 million for the year ended December 31, 2022, mainly driven by significantly reduced research and development expenses. Research and development expenses decreased $28.5 million as a result of our narrowed focus in 2022 on primarily progressing our lead development asset, AVTX-002, as opposed to the progression of multiple programs in 2021. We also recognized a $10.0 million upfront license

fee in 2021 for the AVTX-002 expanded indication license agreement that did not repeat in 2022. As a result of the focused pipeline, Avalo executed a cost reduction plan, including reducing its headcount and supporting infrastructure. Selling, general and administrative expenses decreased $3.9 million as a result, however, these decreases were partially offset by $6.7 million of severance and stock-based compensation expense from employee separations.

The net loss and net loss per share for the year ended December 31, 2022 was largely driven by operating expenses and partially offset by revenue from business development transactions.

Consolidated Balance Sheets
(In thousands, except share and per share data)

December 31,
2022 2021
Assets
Current assets:
Cash and cash equivalents $ 13,172 $ 54,585
Accounts receivable, net — 1,060
Other receivables 1,919 3,739
Inventory, net 20 38
Prepaid expenses and other current assets 1,290 2,372
Restricted cash, current portion 15 51
Total current assets 16,416 61,845
Property and equipment, net 2,411 2,695
Other long-term asset — 1,000
Intangible assets, net — 38
Goodwill 14,409 14,409
Restricted cash, net of current portion 131 227
Total assets $ 33,367 $ 80,214
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable $ 2,882 $ 3,369
Deferred revenue 88 —
Accrued expenses and other current liabilities 13,214 16,519
Notes payable, current 5,930 —
Total current liabilities 22,114 19,888
Notes payable, non-current 13,486 32,833
Royalty obligation 2,000 2,000
Deferred tax liability, net 141 113
Derivative liability 4,830 —
Other long-term liabilities 1,711 2,298
Total liabilities 44,282 57,132
Stockholders’ (deficit) equity:
Common stock—$0.001 par value; 200,000,000 shares authorized at December 31, 2022 and 2021; 9,430,535 and 9,399,517 shares issued and outstanding at December 31, 2022 and 2021, respectively1
9 9
Additional paid-in capital1
292,900 285,239
Accumulated deficit (303,824) (262,166)
Total stockholders’ (deficit) equity (10,915) 23,082
Total liabilities and stockholders’ (deficit) equity $ 33,367 $ 80,214

1 Amounts for prior periods presented have been retroactively adjusted to reflect the 1-for-12 reverse stock split effected on July 7, 2022. See Note 1 for details.

The consolidated balance sheets as of December 31, 2022 and 2021 have been derived from the audited financial statements, but do not include all of the information and footnotes required by accounting principles accepted in the United States for complete financial statements.

Consolidated Statements of Operations
(In thousands, except per share data)

Year Ended December 31,
2022 2021
Revenues:
Product revenue, net $ 3,364 $ 4,773
License and other revenue 14,687 625
Total revenues, net 18,051 5,398
Operating expenses:
Cost of product sales 3,434 1,491
Research and development 31,308 59,835
Selling, general and administrative 20,711 24,658
Amortization expense 38 1,548
Total operating expenses 55,491 87,532
(37,440) (82,134)
Other expense:
Interest expense, net (4,170) (2,391)
Other expense, net (20) (20)
Total other expense, net from continuing operations (4,190) (2,411)
Loss from continuing operations before income taxes (41,630) (84,545)
Income tax expense (benefit) 28 (196)
Loss from continuing operations $ (41,658) $ (84,349)
Loss from discontinued operations — (27)
Net loss $ (41,658) $ (84,376)
Net (loss) income per share of common stock, basic and diluted1:
Continuing operations $ (4.43) $ (9.95)
Discontinued operations — 0.00
Net loss per share of common stock, basic and diluted $ (4.43) $ (9.95)
Net (loss) income per share of preferred stock, basic and diluted1:
Continuing operations $ (4.15)
Discontinued operations —
Net loss per share of preferred stock, basic and diluted $ (4.15)

1 Amounts for prior periods presented have been retroactively adjusted to reflect the 1-for-12 reverse stock split effected on July 7, 2022. See Note 1 for details.

The consolidated statements of operations for the year ended December 31, 2022 and 2021 have been derived from the audited financial statements, but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements.

About AVTX-002

AVTX-002, Avalo’s lead development asset, is a fully human monoclonal antibody (mAb), directed against human LIGHT (Lymphotoxin-like, exhibits Inducible expression, and competes with Herpes Virus Glycoprotein D for Herpesvirus Entry Mediator (HVEM), a receptor expressed by T lymphocytes). There is increasing evidence that the dysregulation of the LIGHT-signaling network which includes LIGHT, its receptors HVEM and LTβR and the downstream checkpoint BTLA, is a disease-driving mechanism in autoimmune and inflammatory reactions in barrier organs. Therefore, we believe reducing LIGHT levels can moderate immune dysregulation in many acute and chronic inflammatory disorders, including NEA. AVTX-002 previously demonstrated proof of concept in COVID-19 induced acute respiratory distress syndrome including reduction in mortality and respiratory failure.

About AVTX-002 PEAK Trial

The Phase 2 PEAK Trial is a randomized, double-blind, placebo-controlled, parallel group trial designed to evaluate the safety and efficacy of AVTX-002 for the treatment of poorly controlled NEA (NCT05288504). Following 12 weeks of treatment, the efficacy and safety of AVTX-002 will be evaluated compared with placebo. The primary endpoint is the proportion of patients who experience any of the following asthma-related events: (i) ≥6 additional reliever puffs of a short-acting beta-agonist (compared to baseline) in a 24-hour period on 2 consecutive days, or (ii) increase in inhaled corticosteroid dose ≥4 times than the dose at baseline, or (iii) a decrease in peak flow of 30% or more (compared to baseline) on 2 consecutive days of treatment, or (iv) an asthma exacerbation requiring the use of systemic corticosteroids (tablets, suspension, or injection) for at least 3 days, or (v) a hospitalization or emergency room visit because of an asthma exacerbation.

On March 29, 2023 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported the first patient has been dosed with CB-011 at dose level 1 (50×106 CAR-T cells) in the CaMMouflage Phase 1 trial for adults with relapsed or refractory multiple myeloma (r/r MM). CB-011 is an allogeneic anti-BCMA CAR-T cell therapy designed to improve antitumor activity by reducing T and natural killer (NK) cell-mediated rejection (Press release, Caribou Biosciences, MAR 29, 2023, View Source [SID1234629482]).

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"We are excited to announce the first patient has been treated with CB-011 in the CaMMouflage Phase 1 trial for relapsed or refractory multiple myeloma," said Rachel Haurwitz, PhD, Caribou’s president and chief executive officer. "Initiation of the CaMMouflage trial is the first step in evaluating the safety of CB-011 and assessing how our immune cloaking approach for CB-011 may improve the antitumor activity. We continue to be humbled by the patients, their families, and the physicians who are partnering with Caribou as we develop off-the-shelf CAR-T cell therapies that have the potential to address the needs of broad patient populations."

CB-011 is the first allogeneic CAR-T cell therapy, to Caribou’s knowledge, with an immune cloaking approach that includes both removal of the endogenous beta-2 microglobulin (B2M) protein and insertion of a B2M–human-leukocyte-antigen-E–peptide (B2M–HLA-E) transgene. This strategy has the potential to improve the antitumor activity by blunting CAR-T cell rejection mediated by both the patient’s T cells and NK cells.

"Approved therapies have demonstrated efficacy in patients with relapsed or refractory multiple myeloma, but challenges remain with patient access, tolerability, and treatment burden," said Sundar Jagannath, MD, professor of medicine and Mount Sinai endowed chair for multiple myeloma at Mount Sinai School of Medicine and director of the Multiple Myeloma Center of Excellence at Tisch Cancer Institute, Mount Sinai Hospital, New York. "There is a significant unmet need for an off-the-shelf CAR-T cell therapy as a readily available treatment option that does not require multiple rounds of treatment."

Caribou plans to continue to enroll additional patients at dose level 1 in the CaMMouflage trial and provide an update on the clearance of dose levels as appropriate.

About the CaMMouflage Trial
The CaMMouflage Phase 1 trial (NCT05722418) is an open-label, multicenter clinical trial designed to evaluate CB-011 in adults with relapsed or refractory multiple myeloma (r/r MM). Part A, a 3+3 dose escalation design, will evaluate the safety and tolerability of CB-011 at multiple dose levels and will

be utilized to determine the maximum tolerated dose and/or the recommended Phase 2 dose. Part B is the dose expansion portion with the primary objective of determining tumor response after a single dose of CB-011. CaMMouflage will include patients who have had 3 or more prior lines of therapy and will exclude patients who have received a BCMA-targeted therapy within the last 3 months and/or any prior CAR-T cell therapy. For more information about the CaMMouflage trial (NCT05722418), please visit clinicaltrials.gov.

About Multiple Myeloma
As of 2022, multiple myeloma (MM) made up 18% of hematologic malignancies in the United States and 1.8% of all cancers. In 2022, there were an estimated 34,470 new cases in the United States and an estimated 12,640 deaths. Median age of diagnosis for MM is 69 years and the five-year survival in these patients is approximately 58%.

About Caribou’s Novel Next-Generation CRISPR Platform
CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of Class 2 CRISPR systems: the nuclease protein that cuts DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to an edit at the targeted genomic site. CRISPR systems are capable of editing unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function and phenotype. In response to this challenge, Caribou has developed CRISPR hybrid RNA-DNA guides (chRDNAs; pronounced "chardonnays") that direct substantially more precise genome editing compared to all-RNA guides. Caribou is deploying the power of its Cas12a chRDNA technology to carry out high efficiency multiple edits, including multiplex gene insertions, to develop CRISPR-edited therapies.

On March 29, 2023 Abbott reported that it will announce its first-quarter 2023 financial results on Wednesday, April 19, 2023, before the market opens (Press release, Abbott, MAR 29, 2023, View Source [SID1234629481]).

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The announcement will be followed by a live webcast of the earnings conference call at 8 a.m. Central time (9 a.m. Eastern), and will be accessible through Abbott’s Investor Relations website at www.abbottinvestor.com. An archived edition of the call will be available later that day.

On March 28, 2023 Oncopeptides, a biotech company focused on research, development and commercialization of therapies for difficult-to-treat hematological diseases, reported that the company has received a research grant of 3 MSEK from Sweden´s Innovation Agency (Vinnova), to explore the development of new treatment options for glioblastoma, an aggressive and incurable form of brain cancer (Press release, Oncopeptides, MAR 28, 2023, View Source [SID1234646784]). The grant enables exploratory research to better understand the potential of the PDC platform in solid tumors such as glioblastoma.

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Preclinical data demonstrate that the proprietary Peptide Drug Conjugate (PDC) platform has a significant potential to address the treatment challenges in glioblastoma. The PDC compounds are designed around two components, a peptide carrier and a cytotoxic payload. The peptide carrier utilizes the increased metabolic activity of glioblastoma cells to rapidly hydrolyze the PDC compounds into multiple hydrophilic metabolites leading to intracellular accumulation in cancer cells. This can increase the therapeutic index compared to conventional chemotherapy.

"We are very excited to advance our pre-clinical portfolio and explore the potential of our innovative PDC platform in other indications outside hematological diseases," says Monica Shaw, CEO of Oncopeptides. "Glioblastoma is a common and very aggressive brain tumor type with only very few treatment options and no cure. As such there is an imminent need for more effective therapies with different mode of actions".

The project has received a financial grant from the Eurostars 3-program, it is co-financed by the EU´s research and innovation program "Horizon Europe" and is driven by an international research consortium. Oncopeptides coordinates the project and is responsible for lead optimization to design and synthesize new compounds profiled as potential glioblastoma drugs. Our partners, who represent both academia and biotech, will contribute with their expertise in glioblastoma as well as in the development of advanced preclinical models utilizing cancer cells derived from patients.

The consortium is expected to generate a preclinical data set that may support the selection of a candidate drug for glioblastoma in 2026. The data package may enable Oncopeptides to finalize preclinical and IND enabling studies and subsequently start clinical development.