On November 9, 2020 Forte Biosciences, Inc. (www.fortebiorx.com) (NASDAQ: FBRX), a clinical-stage biopharmaceutical company, reported third quarter 2020 financial results and provided a general business update (Press release, Tocagen, NOV 9, 2020, View Source [SID1234570456]).

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

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

                  Schedule Your 30 min Free Demo!

"This was an eventful quarter for Forte with significant progress in advancing FB-401 for the treatment of atopic dermatitis." said Paul Wagner, Ph.D., CEO of Forte Biosciences "The initiation of our randomized trial in atopic dermatitis patients, including adults and children 2 years of age and older, was a pivotal event and the achievement of this important milestone was the result of a fantastic effort by the Forte team. There is a significant need for safe and effective atopic dermatitis therapies, particularly for children, and we were pleased by the FDA’s recognition of this unmet need with the granting of Fast Track designation to FB-401 for the treatment of atopic dermatitis."

Third Quarter 2020 Results

Business Highlights

In September 2020, Forte initiated a multi-center, placebo controlled clinical trial of our lead product candidate, FB-401 which is expected to enroll pediatric, adolescent and adult atopic dermatitis ("AD") subjects aged 2 years of age and older. For additional information about the trial, see ClinicalTrials.gov using the identifier NCT04504279.

In October 2020, the U.S. Food and Drug Administration ("FDA") granted Fast Track Designation to FB-401 for the treatment of atopic dermatitis.

Forte Biosciences ended the quarter with approximately $20.2 million in cash which the company believes is sufficient to fund operations for at least the next 12 months. Approximately $2.4 million of the cash utilization in the quarter was due to merger related costs. Forte had 11.2 million shares of common stock outstanding as of September 30, 2020. Following the close of the quarter, Forte raised $46.0 million in gross proceeds from a public offering on November 2nd.

Operating Results

Research and development expenses were $3.7 million and $7.0 million for the three and nine months ended September 30, 2020, respectively, compared to $0.4 million and $1.5 million for the comparable periods in 2019. The increases in 2020 were primarily due to manufacturing costs and clinical development costs as we continue to advance FB-401.

General and administrative expenses were $1.3 million and $2.8 million for the three and nine months ended September 30, 2020, respectively, compared to $0.3 million and $1.0 million for the comparable periods in 2019. The increases in 2020 were primarily due to professional fees for legal, auditing, tax and business consulting services, and personnel expenses as we transitioned to being a public company.

In 2Q 2020, we recognized a charge of $32.1 million for acquired in-process research and development related to the reverse merger with Tocagen which closed on June 15th.

Conference Call and Webcast Information

The Forte management will host a conference call and webcast today at 4:30 PM Eastern Time. Participants may access the call by dialing 877-705-6003 (Domestic) or 201-493-6725 (International), the conference ID number is: 13712591. The call will also be webcast and can be accessed from the investor relations section of Forte’s website at View Source or View Source A replay of the call will also be available through November 16th.

On November 9, 2020 Bolt Biotherapeutics, a clinical-stage immuno-oncology company developing tumor-targeted therapies that leverage the power of the innate and adaptive immune systems, reported the trial design of its ongoing Phase 1/2 clinical trial and preclinical data demonstrating proofof-concept of its lead clinical drug candidate, BDC-1001 (Press release, Bolt Biotherapeutics, NOV 9, 2020, View Source [SID1234570446]). The clinical observations and preclinical data are now available in three posters at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, being held virtually November 9-14.

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

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

                  Schedule Your 30 min Free Demo!

"We are proud to present our recent scientific advances at the SITC (Free SITC Whitepaper) Annual Meeting highlighting that systemically-delivered trastuzumab ISACs induce a robust, target-dependent activation of the immune system in preclinical models as well as further mechanistic insight for the observed robust anti-tumor activity, said David Dornan, senior vice president of research and manufacturing at Bolt. "We hope to confirm the promising results from our preclinical work in our clinical trial that is currently ongoing."

The in-progress clinical trial poster titled, "Phase 1/2 study of novel HER2-targeting, TLR7/8 immune-stimulating antibody conjugate (ISAC) BDC-1001 with or without immune checkpoint inhibitor in patients with advanced HER2-expressing solid tumors," highlighted the framework of Bolt’s Phase 1/2 clinical trial, an actively enrolling global study in patients with refractory HER2-expressing solid tumors. The primary objectives of the study are to evaluate safety and tolerability and determine the recommended Phase 2 dose for both monotherapy and combination with a checkpoint inhibitor. BDC-1001 is a novel treatment which combines the targeting and antitumor effect of trastuzumab with localized stimulation of the immune system via dual TLR7 and TLR8 agonism.

The preclinical results were shown in two posters. The first titled, "Covalent attachment of a TLR7/8 agonist to tumor-targeting antibodies drives potent anti-tumor efficacy by synergistically activating FcgR-and TLRsignaling and enables safe systemic administration," demonstrated that Bolt’s immune stimulating antibody conjugates (ISACs) are safe and well-tolerated in mice and non-human primates. The ISACs enable potent tolllike receptor (TLR) agonists to be safely administered systemically in preclinical models. ISACs provided distinct and unexpected biological advantage over unconjugated TLR agonists leading to more robust and effective antitumor efficacy.

The final poster titled, "Systemically administered HER2-targeted ISACs provoke a rapid, local response that engages the innate and adaptive arms of the immune system to eradicate tumors in preclinical models," demonstrated that within 24 hours of administration, HER2-directed ISACs induced robust, target-dependent activation of the immune system. There was robust activation of immune cells following anti-HER2 ISAC treatment. In contrast to other immune therapies, such as anti-PD1/PD-L1 and anti-CD40, systemically administered ISACs locally engage both the innate and adaptive immune system to eradicate tumors.

"These preclinical data further validate our tumor-targeting BDC-1001 Boltbody ISAC as a promising candidate to treat HER2-expressing solid tumors in our ongoing Phase 1/2 trial. Recruitment is going well thanks to our dedicated investigators and patients," said Edith Perez, M.D., Chief Medical Officer at Bolt. "We look forward to reporting the initial data readout of our clinical trial soon."

About Bolt Biotherapeutics’ Immune Stimulating Antibody Conjugate (ISAC) Platform Technology The BoltbodyTM ISAC platform technology harnesses the ability of innate immune agonists to convert cold tumors into immunologically hot tumors, thereby illuminating tumors to the immune system and allowing them to be invaded by tumor killing cells. Boltbody ISACs have demonstrated the ability to eliminate tumors following systemic administration as monotherapy in preclinical models and have also led to the development of immunological memory, which is predicted to translate into more durable clinical responses for patients.

About the Ongoing BDC-1001 Phase 1/2 Study in Patients with HER2-Expressing Solid Tumors The Phase 1/2, multi-center, open-label study is evaluating the safety, pharmacokinetics, pharmacodynamics and proof of mechanism of BDC-1001 in patients with HER2-expressing solid tumors. The first portion of the study includes a monotherapy dose-escalation phase in which cohorts of patients will receive ascending intravenous doses of BDC-1001 to determine the maximum tolerated dose and/or the recommended dose to advance into expansion cohorts and Phase 2 based on safety and tolerability. The second portion of the study is a dose expansion phase in which patients will receive BDC-1001 monotherapy to further evaluate the safety, tolerability and clinical antitumor activity of the recommended Phase 2 dose. Please refer to www.clinicaltrials.gov NCT04278144 for additional clinical trial information.

On November 9, 2020 AVEO Oncology (Nasdaq: AVEO) reported financial results for the third quarter ended September 30, 2020 and provided a business update (Press release, AVEO, NOV 9, 2020, View Source [SID1234570441]).

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

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

                  Schedule Your 30 min Free Demo!

"In advance of the Food and Drug Administration’s (FDA) March 31, 2021 Prescription Drug User Fee Act (PDUFA) target action date for our tivozanib New Drug Application, we are actively building out AVEO’s U.S. commercial infrastructure to support the FDA-pending U.S. launch of tivozanib in relapsed or refractory renal cell carcinoma (RCC)," said Michael Bailey, president and chief executive officer of AVEO. "Market data suggests that a significant and growing demand exists for effective and better tolerated treatment options for these patients. These data also suggest that roughly half of patients currently forego treatment in the third- and fourth-line of treatment.1 We believe tivozanib has the potential to help address some of these challenges and provide patients with a new option for continuing their fight against cancer."

Mr. Bailey added, "Beyond our ongoing development of tivozanib as a potential monotherapy treatment, we are continuing our work in the immunotherapy combination setting as a key area of focus, given tivozanib’s tolerability profile and effects on reducing regulatory T-cells2 to potentially enhance activity of the immune system. In addition, we further enhanced our pipeline by regaining full global rights to our second late-stage asset, ficlatuzumab, and have secured additional manufacturing capacity to enable a potential registrational Phase 3 trial in head and neck squamous cell cancer (HNSCC) in 2022. Finally, we remain on track to file an Investigational New Drug (IND) application by the end of the year for AV-380 for the treatment of cancer cachexia, with a potential Phase 1 study in the first quarter of 2021, following the IND safe to proceed letter."

Tivozanib Updates

Announced Publication of Results from Phase 1b/2 TiNivo Study of Tivozanib in Combination with OPDIVO (nivolumab) in RCC in Annals of Oncology. In November 2020, AVEO announced that previously reported results from the Phase 1b/2 TiNivo study of oral (PO) tivozanib (FOTIVDA), AVEO’s next-generation vascular endothelial growth factor (VEGF) receptor tyrosine kinase inhibitor (TKI) drug candidate, in combination with intravenous (IV) nivolumab (OPDIVO, Bristol-Myers Squibb), an immune checkpoint, or PD-1, inhibitor, for the treatment of advanced RCC, were published in Annals of Oncology. The article, titled "TiNivo: Safety and Efficacy of Tivozanib-Nivolumab Combination Therapy in Patients with Metastatic Renal Cell Carcinoma", is available online first via this link.

AVEO is also studying tivozanib in combination with IMFINZI (durvalumab), AstraZeneca’s human monoclonal antibody directed against programmed death-ligand 1 (PD-L1), in patients with first-line metastatic hepatocellular carcinoma (HCC) in the Phase 1b/2 DEDUCTIVE clinical trial, which is currently in Phase 2.
Published Final Overall Survival Results from Phase 3 TIVO-3 Study of Tivozanib in RCC in European Urology. In September 2020, AVEO announced that final overall survival (OS) results from its pivotal Phase 3 TIVO-3 study comparing tivozanib to sorafenib in third- and fourth-line RCC were published in the journal European Urology. The article, titled "Final Overall Survival Results from a Phase 3 Study to Compare Tivozanib to Sorafenib as Third- or Fourth-line Therapy in Subjects with Metastatic Renal Cell Carcinoma," is available online via this link.
Preparations Ongoing to Support Commercial Launch of Tivozanib in the U.S. Following the PDUFA Date (March 31, 2021). In preparation for the potential commercial launch of tivozanib in the U.S., the Company continues to expand its sales, marketing, market access and medical affairs teams, and is working to put in place its U.S. distribution capabilities by the end of the year. In connection with these preparations, in October 2020, AVEO announced the appointment of David W. Crist as vice president of sales. Mr. Crist, who brings to AVEO over twenty years of oncology sales experience in both launch-stage and late-stage companies, will be responsible for building out AVEO’s sales force in anticipation of the potential marketing approval and launch of tivozanib in the U.S.
Tivozanib Non-Oncology Partnership Update

$2.8 Million Development Milestone from Kyowa Kirin Co., Ltd. In August 2020, the Company announced that it earned a $2.8 million development milestone payment from partner Kyowa Kirin. The milestone relates to acceptance by the Japanese Pharmaceuticals and Medical Devices Agency of an IND application for a new formulation of tivozanib in a non-oncology indication being developed by Kyowa Kirin.

Under the terms of AVEO’s agreement with Kyowa Kirin, in addition to the 2019 upfront payment of $25 million to AVEO, waiver of AVEO’s obligation to make an $18 million milestone payment upon AVEO gaining U.S. marketing approval of tivozanib for RCC, and the $2.8 million IND development milestone, Kyowa Kirin has also agreed to pay AVEO up to an additional $388 million in potential milestone payments upon the successful achievement of certain development, regulatory, and commercial objectives in non-oncology indications of tivozanib. Kyowa Kirin will also be obligated to make tiered royalty payments on the net sales of a product for these indications, ranging from a high single-digit to low double-digit percent.
Ficlatuzumab Update

Regained Full Global Rights to Ficlatuzumab. In September 2020, AVEO regained full global rights to ficlatuzumab, the Company’s potent hepatocyte growth factor (HGF) inhibitor antibody which binds to the HGF ligand with high affinity and specificity to inhibit HGF/c-Met biological activities. The Company announced plans to fund the clinical manufacture of ficlatuzumab to enable a potential registrational Phase 3 clinical trial in HNSCC, as well as additional potential development in Phase 2 studies in pancreatic cancer and acute myeloid leukemia. Ficlatuzumab is being studied in an ongoing randomized confirmatory Phase 2 study in combination with cetuximab, an EGFR-targeted antibody, in metastatic HNSCC, which is expected to conclude enrollment in the fourth quarter of 2020, with results from the study expected in the middle of 2021. In that timeframe, the Company plans to provide an update on its pivotal program for ficlatuzumab.
AV-380 Update

Company on Track for IND Application Submission by Year-end, with Phase 1 Clinical Study Planned for the First Quarter of 2021. AVEO plans to submit an IND application to the FDA for AV-380, its first-in-class, potent, humanized inhibitory antibody targeting GDF15, for the treatment of cancer cachexia by year-end. Cachexia, a common complication in patients with advanced cancer and other chronic diseases, is a complex metabolic syndrome characterized by malnutrition and severe involuntary weight loss due to the loss of muscle and fat tissue, as well as the clinical manifestation of anemia, inflammation and suppression of immune functions. Preparations for a Phase 1 trial are currently underway.
Corporate Updates

Announced Restructuring of Existing Term Loan with Closing of New Tranched, $35 Million Debt Facility, Potentially Providing for Working Capital into 2022. In August 2020, The Company announced the closing of a tranched, $35 million debt facility with Hercules Capital, Inc. and its affiliates. Under the terms of the agreement, the initial tranche of $15 million fully refinanced AVEO’s existing Hercules term loan facility, which had an outstanding principal amount of approximately $9.7 million, providing net new proceeds of $5.3 million. A second $10 million tranche is contingent upon the approval of the tivozanib New Drug Application by the FDA as a treatment for RCC, and certain other terms and conditions. An additional two $5 million tranches would potentially become available after that time – one if net product revenues of tivozanib reach $20.0 million within a specified time frame, and the other upon the lender’s consent.
A current summary of the Company’s activities and corporate updates is available in AVEO’s corporate presentation available on the investor relations portion of the Company’s website at investor.aveooncology.com.

Third Quarter 2020 Financial Results

AVEO ended Q3 2020 with $68.8 million in cash, cash equivalents and marketable securities as compared with $47.7 million at December 31, 2019.
Total revenue was approximately $3.6 million for Q3 2020 compared with $25.7 million for Q3 2019. The third quarter of 2019 included the $25.0 million upfront payment pursuant to AVEO’s agreement with Kyowa Kirin for non-oncology indications of tivozanib.
Research and development expense for Q3 2020 was $5.9 million compared with $4.0 million for Q3 2019.
General and administrative expense for Q3 2020 was $5.8 million compared with $2.9 million for Q3 2019.
Net loss for Q3 2020 was $8.4 million, or net loss of $0.33 per basic and diluted share, compared with net income of $16.4 million for Q3 2019, or net income of $1.02 per basic and diluted share, respectively.
Financial Guidance

AVEO believes that its $68.8 million in cash, cash equivalents and marketable securities as of September 30, 2020, along with anticipated partnership cost sharing reimbursements, royalties from EUSA’s FOTIVDA sales and, if the pending marketing application for FOTIVDA is approved by the FDA, resulting product revenues upon commercial launch and the potential additional $20 million in credit under the Hercules loan, would allow the Company to fund planned operations into 2022.

In accordance with Accounting Standards Update No. 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern (Accounting Standards Codification Subtopic 205-40), cash flows that are contingent on FDA approval, such as product revenues, cannot be reflected in the going concern assessment. As a result, Hercules loan funding contingent on such approval and revenue is also excluded from the Company’s going concern assessment. Accordingly, the Company continues to have a going concern opinion.

About Tivozanib (FOTIVDA)

Tivozanib is an oral, once-daily, next-generation vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI) discovered by Kyowa Kirin and approved as FOTIVDA for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union and other countries in the EUSA territory. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.3,4 Tivozanib is being studied in the TIVO-3 trial, which is supporting a regulatory submission of tivozanib in the U.S. seeking marketing approval as a treatment for adult patients with relapsed or refractory advanced RCC. Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models2 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC.5 Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal, ovarian and breast cancers. Tivozanib is also being studied by partner Kyowa Kirin in non-oncology indications.

On November 9, 2020 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a leader in cell therapy to treat cancer, reported data from the dose escalation cohorts of its Phase 1 SURPASS trial using ADP-A2M4CD8 in a poster at the Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) ("SITC") Conference (Press release, Adaptimmune, NOV 9, 2020, View Source [SID1234570434]).

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

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

                  Schedule Your 30 min Free Demo!

In these cohorts of heavily pre-treated patients with advanced cancers (n=6), three were treated with target doses of 1 billion SPEAR T-cells, and three with target doses of 5 billion. Most adverse events were consistent with those typically experienced by cancer patients undergoing cytotoxic chemotherapy or cancer immunotherapy.

"We have seen responses in two out of six patients treated in the safety cohorts of the SURPASS trial as well as antitumor activity in five of them. The responses and antitumor activity we have seen with our next-generation ADP-A2M4CD8 SPEAR T-cells, across a range of solid tumors, support our belief that this is a highly active product," said Ad Rawcliffe, Adaptimmune’s Chief Executive Officer. "Based on these data, we will initiate the Phase 2 trial in gastroesophageal cancers in the first half of 2021 and look forward to identifying additional indications to take into late-stage development."

There were two confirmed partial responses (PRs): one in a patient with esophagogastric junction (EGJ) cancer, previously reported, and one in a patient with head and neck cancer, reported as unconfirmed in May. The four other patients had best overall responses of stable disease (SD). Overall, five out of six patients treated had initial tumor shrinkage.

In vitro translational data using the manufactured products from patients in the SURPASS trial indicate that co-expression of the CD8α co-receptor on CD4+ ADP-A2M4 SPEAR T-cells enables them to kill MAGE-A4 expressing target cells with equal potency as CD8+ SPEAR T-cells targeting MAGE-A4. These data, combined with the responses and antitumor activity observed at low doses, indicate that ADP-A2M4D8 may be a more potent product than the first-generation ADP-A2M4 SPEAR T-cells.

Best Overall Response (BOR) and maximum changes from baseline in target lesions in Cohorts 1 and 2

Indication Dose x 109 BOR Tumor reduction
Head and neck 4.6 PR -63.16%
EGJ 1.2 PR -51.52%
EGJ 1.0 SD -34.07%
Ovarian 1.1 SD -16.13%
Esophageal 6.0 SD -13.37%
MRCLS 5.7 SD +1.35%

As of data cut-off: October 1, 2020

At SITC (Free SITC Whitepaper), Adaptimmune also presented a poster entitled "Inhibition of AKT signaling during expansion of TCR-engineered T-cells from patient leukocyte material generates SPEAR T-cells with enhanced functional potential in vitro." These preclinical data indicate that AKT inhibition during the manufacture of SPEAR T-cells results in a more consistent expansion and phenotype of the final product. This process is currently being used for manufacture of ADP-A2M4CD8 for the SURPASS trial.

The Company also presented two posters summarizing data for the two completed Phase 1 trials with ADP-A2M10 (a previously terminated program)

On November 9, 2020 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported financial results for the third quarter 2020 and recent business progress (Press release, Abeona Therapeutics, NOV 9, 2020, View Source [SID1234570433]).

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

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

                  Schedule Your 30 min Free Demo!

"Abeona remains committed to pursuing the development of our portfolio of advanced and early stage programs toward providing our novel gene and cell therapies to patients who currently have no approved treatment options," said Michael Amoroso, Chief Operating Officer of Abeona. "In particular, we are continuing to propel our clinical programs in recessive dystrophic epidermolysis bullosa (RDEB) and Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB) to drive Abeona’s long-term growth and unlock shareholder value."

Third Quarter and Recent Highlights

Corporate and Business Development Updates

In August 2020, Abeona entered into definitive agreements with Taysha Gene Therapies to license its rights to ABO-202, an AAV-based gene therapy for CLN1 disease (also known as infantile Batten disease). As part of the transaction, Abeona received initial cash proceeds of $7.0 million in October 2020. Abeona is also eligible to receive up to $56.0 million upon the achievement of certain clinical, regulatory and sales milestones, plus royalty-based payments based on net sales.
In October 2020, Abeona entered into a license agreement with Taysha Gene Therapies with respect to certain intellectual property rights, materials, and know-how relating to a potential AAV-based gene therapy for Rett syndrome. In connection with the agreement, Abeona received a one-time upfront payment of $3.0 million and is eligible to receive up to $56.5 million upon the achievement of certain clinical, regulatory and sales milestones, plus royalty-based payments based on net sales.
In October 2020, Michael Amoroso, Senior Vice President and Chief Commercial Officer at Abeona, was promoted to Chief Operating Officer. In this newly created role, Mr. Amoroso’s responsibilities are broadened to include oversight and leadership of all operations.
Abeona continues to work with Jefferies LLC as its financial advisor in the review of strategic options focused on advancing the Company’s mission and maximizing stakeholder value.
EB-101 (Autologous, Gene-Corrected Cell Therapy)

Patient enrollment is ongoing for Abeona’s EB-101 pivotal Phase 3 VIITAL study for RDEB. The Company currently anticipates completing enrollment in the VIITAL study in the first half of 2021, depending upon the impact from the COVID-19 pandemic, including travel restrictions and safety concerns.
ABO-102 and ABO-101 (AAV-based Gene Therapies)

Target enrollment in the ABO-102 Transpher A study for MPS IIIA (15 to 22 patients) has been achieved. To date, 18 patients have been dosed in the Transpher A study, including 12 patients dosed in cohort 3. Abeona intends to continue enrolling patients into the study through the first quarter of 2021 given the lack of treatment options for MPS IIIA and encouraging efficacy and safety data from cohort 3.
In the ABO-101 Transpher B study for MPS IIIB, 11 patients have been dosed to date, including 4 patients dosed in cohort 3. The Company anticipates completing target enrollment in the Transpher B study (15 to 20 patients) in the first quarter of 2021.
In the third quarter, Abeona presented its plan toward registration of ABO-102 for MPS IIIA (Sanfilippo syndrome type A) during a kick-off meeting under the European Medicines Agency’s (EMA) PRIority MEdicines (PRIME) program that offers a path for accelerated assessment of promising therapies targeting unmet medical needs. Based on the meeting, along with previous input from the Committee for Medicinal Products for Human Use and the Pediatric Committee of the EMA, Abeona anticipates submitting a marketing authorization application for EU conditional approval of ABO-102 for MPS IIIA after completion of two-year follow-up of the last patient treated in the Transpher A study. Regarding the U.S. regulatory path for ABO-102 in MPS IIIA, Abeona plans to request a meeting with the FDA to take place in the first quarter of 2021, depending on FDA’s availability.
Manufacturing Activities

Process development at the Company’s GMP manufacturing facility in Cleveland, Ohio is ongoing that will enable production of the retrovirus used for EB-101 manufactured at the facility, allowing for increased control of the supply chain and product quality, as well as reduced costs. In addition, Abeona continues process development activities to enable in-house manufacturing of commercial supply of ABO-102 and ABO-101.
Third Quarter Financial Results

Cash, cash equivalents, receivables and short-term investments totaled $103.9 million as of September 30, 2020, compared to $129.3 million as of December 31, 2019. Accounts receivable were $7.0 million at September 30, 2020, which was paid by the customer in October 2020. Net cash used in operating activities was $10.7 million for the third quarter of 2020.

License and other revenues for the third quarter of 2020 were $7.0 million, comprised of initial proceeds from the ABO-202 transaction, compared to zero revenues in the year-ago quarter.

Research and development (R&D) expenses were $8.0 million for the third quarter of 2020 and $20.9 million for the nine months ended September 30, 2020, compared to $10.9 million and $39.0 million in the comparable periods in 2019. The decrease in R&D expenses was primarily due to decreased manufacturing, clinical and non-clinical development activities arising from the effects of the COVID-19 pandemic, and cost savings associated with the decision to internally manufacture retrovirus for the EB-101 program.

General and administrative (G&A) expenses were $4.4 million for the third quarter of 2020 and $16.4 million for the nine months ended September 30, 2020, compared to $4.7 million and $16.0 million in the comparable periods in 2019. The decrease in G&A expenses in the third quarter of 2020 was primarily due to decreased salary and related costs. The increase in G&A expenses in the nine months ended September 30, 2020 was primarily from severance costs associated with management changes, partially offset by decreased professional fees.

Net loss was $7.2 million for the third quarter of 2020 and $68.4 million for the nine months ended September 30, 2020, compared to net loss of $17.4 million and $59.9 million for the comparable periods in 2019. The increase in net loss for the nine months ended September 30, 2020 includes the non-cash impairment charge of $32.9 million related to the termination of the license agreement with REGENXBIO.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast tomorrow, Tuesday, November 10, 2020 at 8:30 a.m. ET, to discuss its third quarter 2020 financial results and business update. To access the call, dial 844-369-8770 (U.S. toll-free) or 862-298-0840 (international) 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.