On August 12, 2020 Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported financial results for the quarter and six months ended June 30, 2020 and provided an update on corporate progress (Press release, Exicure, AUG 12, 2020, View Source [SID1234563485]).

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!

"The second quarter was marked by important new expansion," said Dr. David Giljohann Exicure’s Chief Executive Officer. "We grew our organization with the addition of neurology expert, Dr. Douglas Feltner, as Chief Medical Officer, expanded our clinical team and opened additional clinical sites in support of our Phase 2 clinical trial in cancer patients using our drug candidate, cavrotolimod. In July, we moved into our new 30,000 sq. ft. laboratory and corporate headquarters where our R&D team continues to advance our growing neurology therapeutic area," concluded Dr. Giljohann.

XCUR-FXN, Exicure’s Friedreich’s ataxia therapeutic candidate

Despite the ongoing COVID-19 pandemic, our laboratories have continued operations with limited impact on our research and development activities.
We remain on track to initiate IND-enabling studies for Friedreich’s ataxia in the fourth quarter of this year.
Cavrotolimod (AST-008) Phase 1b/2 clinical trial is open and actively enrolling patients

In the second quarter of 2020, we began enrolling patients in the Phase 2 dose expansion phase of the clinical trial of intra-tumoral cavrotolimod in combination with approved checkpoint inhibitors pembrolizumab or cemiplimab, to treat patients with advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma.
Currently, 14 clinical trial sites are open, and we expect to open up to 11 additional sites for a potential total of 25 sites.
We are continuing to monitor the impact that COVID-19 may have on the trial’s patient enrollment and safety, site initiation, and study integrity. We have put in place and continue to maintain a variety of measures to mitigate the effects of COVID-19 and our top priority is to maintain patient safety and trial continuity.
Second Quarter Financial Results, Financial Guidance and Recent Developments

Cash Position: Cash, cash equivalents, and short-term investments were $85.8 million as of June 30, 2020 as compared to $98.8 million as of March 31, 2020.

Research and Development (R&D) Expenses: Research and development expenses were $7.0 million for the quarter ended June 30, 2020, as compared to $3.4 million for the quarter ended June 30, 2019. We have increased full-time staffing in R&D from 23 at June 30, 2019 to 41 at June 30, 2020 and the associated increase in activity, in addition to growth in cavrotolimod (AST-008) clinical trial activities, has driven our increase in R&D costs. The increase in staffing and associated increases in platform and discovery related costs reflects increased preclinical R&D activities associated with our collaboration with Allergan plc, increased costs related to XCUR-FXN, our Friedreich’s ataxia program, as well as other preclinical discovery work in neurology and ophthalmology.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.2 million for the quarter ended June 30, 2020 as compared to $2.0 million for the quarter ended June 30, 2019. This increase is primarily due to higher legal costs associated with operating as a public company, higher franchise tax costs, and higher D&O insurance expense, partially offset by lower travel and other costs.

Net Loss: We had a net loss of $4.3 million for the quarter ended June 30, 2020 as compared to a net loss of $5.2 million for the quarter ended June 30, 2019. This decrease in net loss of $0.9 million was driven principally by the recognition of $4.8 million of revenue associated with our collaboration with Allergan plc, offset by the increases in R&D expenses and G&A expenses discussed above.

Capital Resources Guidance: We believe that, based on our current operating plans and estimates of future expenses, as of the date of this press release, our existing cash, cash equivalents and short-term investments will be sufficient to fund our operations into early 2022.

Response to COVID-19: With the global spread of the ongoing COVID-19 pandemic in the first half of 2020, we have been closely monitoring developments and have taken active measures to protect the health of our employees and their families, our communities, as well as our clinical trial investigators, patients and caregivers. We continue to carefully manage laboratory staffing and take other appropriate managerial actions to maintain progress on our preclinical and collaboration programs. We also continue to work closely with our third-party manufacturers and other partners to manage our supply chain activities and will take such action as we believe appropriate with our clinical operations to maintain patient safety and trial continuity.

About our Pipeline

Neurology

In December 2019, Exicure announced the development of XCUR-FXN, an SNA–based therapeutic candidate for the treatment of Friedreich’s ataxia (FA). FA is driven by triplet repeats in the frataxin gene which compromises the patient’s ability to generate adequate levels of frataxin protein. Exicure believes its SNA technology has the potential to address this genetic challenge and that its therapeutic strategy may lead to increases in the frataxin protein. Exicure plans to design and develop XCUR-FXN with guidance from, and in collaboration with, the Friedreich’s Ataxia Research Alliance (FARA). Preclinical research is ongoing and IND-enabling studies for XCUR-FXN are expected to commence in late 2020.
Exicure is continuing preclinical research on the application of its SNA technology in neurological conditions, building on its early proof-of-concept work with nusinersen and its new therapeutic candidate, XCUR-FXN. Exicure is currently exploring additional neurological conditions, including spinocerebellar ataxia, Batten disease, amyotrophic lateral sclerosis (ALS) and Huntington’s disease.
Immuno-oncology; Cavrotolimod (AST-008)

Cavrotolimod (AST-008) is an investigational SNA consisting of toll-like receptor 9 (TLR9) agonists designed for immuno-oncology applications. Exicure has now dosed 20 patients and completed enrollment in the Phase 1b stage of the Phase 1b/2 clinical trial. To date, we have not observed any treatment related serious adverse events or any dose-limiting toxicity.
Biomarker data from the Phase 1b stage of the clinical trial showed dose-related systemic immune activation and a trend towards increased tumor immune cell infiltration.
In the second quarter of 2020, we dosed the first patient in a Phase 2 dose expansion phase of our Phase 1b/2 clinical trial for intra-tumoral cavrotolimod in combination with pembrolizumab or cemiplimab to treat two cohorts of patients with advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma. Each cohort is expected to enroll up to 29 patients.
Collaborations

In late 2019, Exicure entered into a collaboration, option and license agreement with Allergan plc, which was acquired by AbbVie Inc. in May 2020, and is now actively engaged in preclinical research and discovery in two programs related to the treatment of hair loss disorders. In early 2019, Exicure also entered into a collaboration agreement with Dermelix Biotherapeutics under which Dermelix has the option to develop a targeted therapy for the treatment of Netherton Syndrome (NS).

About FARA

The Friedreich’s Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich’s ataxia. www.CureFA.org.

On August 12, 2020 VBL Therapeutics (Nasdaq: VBLT) reported that the independent Data Safety Monitoring Committee (DSMC) has completed its second, pre-planned interim analysis in the ongoing OVAL Phase 3 study investigating ofranergene obadenovec (VB-111) in patients with platinum-resistant ovarian cancer, and unanimously recommended that the study continue as planned (Press release, VBL Therapeutics, AUG 12, 2020, View Source [SID1234563484]).

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 this second interim analysis, the DSMC reviewed unblinded Overall Survival (OS) data of the first 100 randomized patients with a followup of at least 3 months. OS is the primary endpoint of the OVAL study. The committee also looked at response rate and safety information.

"We are pleased by the DSMC recommendation to continue the OVAL trial as planned," said Dror Harats, MD, Chief Executive Officer of VBL Therapeutics. "This is the second successful analysis in the OVAL study, which reviewed unblinded overall survival data comparing VB-111 to placebo. The OVAL study continues to show strong recruitment despite the COVID-19 pandemic, and we are very encouraged by the high response rate of over 50% of the trial participants, which has been maintained. This latest DSMC recommendation, together with the remarkable response rate observed in our first interim efficacy analysis and the survival benefit seen in the Phase 2 trial of VB-111 in patients with platinum-resistant ovarian cancer, support the confidence we have in OVAL. We are excited to advance VB-111 for the potential benefit of ovarian cancer patients."

In March 2020, the Company announced results of the first interim analysis in the OVAL study, which reviewed unblinded data and assessed CA-125 response, measured according to the GCIG criteria, in the first 60 enrolled subjects evaluable for CA-125 analysis. The overall response rate in the first 60 randomized evaluable patients across both arms was 53%. Assuming a balanced randomization, the response rate in the treatment arm (VB-111 in addition to weekly paclitaxel) was 58% or higher. In patients who had post-dosing fever, which is a marker for VB-111 treatment, the response rate was 69%.

The next DSMC review in the OVAL study is expected in the first quarter of 2021.

About the OVAL study (NCT03398655)
OVAL is an international Phase 3 randomized pivotal potential registration clinical trial that compares a combination of VB-111 and paclitaxel to placebo plus paclitaxel, in patients with platinum-resistant ovarian cancer. The study is planned to enroll approximately 400 patients. OVAL is conducted in collaboration with the GOG Foundation, Inc., an independent international non-profit organization with the purpose of promoting excellence in the quality and integrity of clinical and basic scientific research in the field of gynecologic malignancies.

About VB-111 (ofranergene obadenovec)
VB-111 is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. VB-111 is a unique biologic agent that uses a dual mechanism to target solid tumors. Its mechanism combines blockade of tumor vasculature with an anti-tumor immune response. VB-111 is administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in an "all comers" Phase 1 trial as well as in three tumor-specific Phase 2 studies.VB-111 has received an Orphan Designation for the treatment of ovarian cancer from the European Commission. VB-111 has also received orphan drug designation in both the US and Europe, and fast track designation in the US for prolongation of survival in patients with rGBM. VB-111 successfully demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970).

On August 12, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported its financial results for the quarter ended June 30, 2020 and provided a business update (Press release, Moleculin, AUG 12, 2020, View Source [SID1234563483]).

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!

Management Discussion

Walter Klemp, Chairman and CEO of Moleculin, stated, "Despite the difficult backdrop resulting from the global pandemic, we made tremendous progress across all three of our core technologies, particularly in our infectious disease platform of antimetabolites, bolstered our financial position, and added to our experienced leadership team. Importantly, we were pleased to progress our efforts to combat COVID-19, as WP1122, which is often referred to as a "prodrug" of 2-DG and one of our antimetabolites, demonstrated its potential in a number of pre-clinical studies and independent research publications. Independent research found 2-deoxy-D-glucose ("2-DG") reduced replication of SARS-CoV-2, the virus that causes COVID-19, by 100% in in vitro testing. A second independent publication at the University of Campinas in São Paulo further demonstrated the potential of WP1122’s mechanism of action as it showed SARS-CoV-2 infection is supported by elevated glucose levels and that inhibition of glycolysis with 2-DG effectively eliminated viral load in vitro. Additionally, two rounds of preclinical testing at one independent lab confirmed by a round of in-vitro testing at a second independent lab in a separate virus host cell line continued to demonstrate WP1122’s antiviral activity in SARS-CoV-2. We are very encouraged by this early demonstration of efficacy and are now even more motivated to continue to drive its development. Based on guidance from the FDA, we are pursuing additional studies in animal models, which we believe is the critical path to our expected timing, to further assess WP1122’s antiviral capability, with the goal of a possible IND filing in 2020, in preparation for beginning a human clinical trial thereafter."

Mr. Klemp continued, "Although we have expanded the scope and focus of our WP1122 program, our lead candidate, Annamycin, a ‘next generation anthracycline’ demonstrating little to no cardiotoxicity, still remains one of our key priories. The results from the Phase 1 portion of our US Phase 1/2 clinical trial in acute myeloid leukemia (AML) have been highly encouraging, as Annamycin met its primary endpoint and demonstrated a clean safety profile with no evidence of cardio-toxicity when delivered to patients at or below the lifetime maximum anthracycline dose established by the FDA. Following these strong results and an independent review of Annamycin, in which the independent expert concluded that he ‘does not see evidence of cardio-toxicity’, we received authorization from the Polish Department of Registration of Medicinal Products to increase the Phase 1 dose escalation portion of our clinical trial for the treatment of AML. We believe this to be a substantial development in the acceleration of our trial as it allows for the increase in dose escalation increments between cohorts from 30 mg/m2 to 60 mg/m2. This will enable our next cohort to increase to 300 mg/m2, assuming all requirements for safety are met with the 240 mg/m2 cohort, for which we are currently recruiting. With these dosing expectations, the Company believes that European dosing will increase in 2020, providing for a recommended Phase 2 Dose to be established in 2021. In addition to driving the development of Annamycin, during the second quarter we saw the advancement of WP1066, the lead molecule in Moleculin’s portfolio of immune stimulators and modulators of transcription. Importantly, Emory University began recruiting and treating patients in its Phase 1 clinical trial of WP1066 for the treatment of brain tumors in children. We are pleased by the progress of the trial, which has now enrolled and treated three patients. The Emory study, which is being at conducted at the Aflac Cancer & Blood Disorders Center at Children’s Healthcare of Atlanta, represents a new approach for treating pediatric brain cancer and benefits from safety data generated in the ongoing clinical trial of WP1066 in adult brain tumors being conducted by MD Anderson Cancer Center."

Mr. Klemp concluded, "With our focus on the continued progression of our candidates and our expansion into infectious disease, we found it prudent to bolster our expertise. As a result, we made several strategic additions to our team at Moleculin. In March, we added Dr. Hongbo Zhai, who has two decades of research and development experience in pharmaceuticals and biotechnology, to our Science Advisory Board. To complement our expanded focus on COVID-19, we then added to our Science Advisory Board Dr. Dominique Schols, a leading virologist at the Rega Institute in Leuven, Belgium, and Dr. Richard Whitley, head of the NIAID Antiviral Drug Discovery and Development Center. With the additions to our team and the progress we made in all three of our programs, we believe we are well positioned to continue to build on the momentum we have achieved thus far in 2020."

Recent Milestones and Accomplishments:

Next Generation Anthracycline – Annamycin

Announced preclinical data corroborating the efficacy of Annamycin in lung metastases at AACR (Free AACR Whitepaper)
In process with the Polish regulatory authorities’ approval to open two additional clinical sites for the Phase 1/2 clinical study
Approved to accelerate European clinical trial in AML, URPL doubled dose escalation
Received additional positive safety data in EU AML trial, none of the 19 patients evaluated thus far have shown signs of cardiotoxicity
Received positive independent report confirming absence of cardiotoxicity (unlike currently approved anthracyclines)
Announced positive results and successful completion of the Phase 1 portion of the AML Phase 1/2 trial in the US
Found to be active against tumor metastases to the lung in pre-clinical testing
Confirmed anti-tumor efficacy of Annamycin in AML through new animal data
Expanded drug production to support positive clinical activity
Received FDA Fast Track designation
Immune/Transcription Modulators – WP1066 Portfolio

Reported findings that WP1066 used in combination with traditional whole brain radiation therapy (WBRT) resulted in long-term survivors and enhanced median survival time relative to monotherapy in mice with implanted human brain tumors
Emory University has treated three patients in a Phase 1 clinical trial of WP1066 for the treatment of brain tumors in children after receiving FDA Approval of IND and Emory University Clinical Trial Review Committee approval for STAT3 inhibitor in Investigator Initiated Clinical Trial
Patent protection filed by MD Anderson covering combination of immune stimulating/transcriptional modulator, including combination with radiation therapy
Presented preclinical pancreatic cancer data at American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting
Received Orphan Drug Designation from FDA
Infectious Disease and Metabolism/Glycosylation Inhibitors – WP1122 Portfolio

Independent research team at the University of Campinas in São Paulo, Brazil demonstrated that SARS-CoV-2 infection is supported by elevated glucose levels and that inhibition of glycolysis with 2-DG effectively eliminated viral load in vitro
Corroborated antiviral activity of WP1122 against coronavirus in pre-clinical testing at IIT Research Institute in another virus host cell line
Agreement with Sterling Pharma USA LLC for U.S. production of WP1122 to support expanded development efforts
Two rounds of preclinical assessment of the potential for WP1122 to address COVID-19 at ImQuest BioSciences demonstrated that WP1122 has an antiviral effect on HCoV-229E. The virus yield reduction assay demonstrated a 5 to 10-fold inhibition of coronavirus production by WP1122 when compared to untreated virus control.
University of Frankfurt found 2-DG to reduce replication of SARS-CoV-2, the virus that causes COVID-19, by 100% in in vitro testing
Patent filed by MD Anderson covering WP1122 as anti-viral drug candidate
Final data from Phase 1 proof-of-concept clinical trial for the treatment of cutaneous T-cell lymphoma
Began preclinical testing of new approach to Pancreatic cancer, opportunity to attack cancer by inhibiting tumor metabolism
Corporate Strategy

Appointed Dr. Whitley, who leads the Drug Discovery and Development Center for the National Institute of Allergy and Infectious Diseases, to Science Advisory Board
Added Dr. Dominique Schols, a leading virologist from the Rega Institute to the Moleculin development team as a consultant and a member of our Science Advisory Board
Appointed Dr. Hongbo Zhai, former Senior Faculty and Supervisor of Postdoctoral Fellow at University of California San Francisco, to Science Advisory Board
Anticipated 2020 Milestones

Achieving an MTD or a dose level at or above 300 mg/m2 in EU AML Phase 1/2 trial for Annamycin
Expanding our infectious disease portfolio via testing of WP1122 against CoV-2 in animal models and in vitro testing on CoV-2 and other hard to treat viruses with other antimetabolites – resulting in a cancer or virus related IND or its equivalent possibly being filed in 2020
IND submission for Annamycin for the treatment of tumor metastases to the lung
Moving WP1220 for the treatment of CTCL forward via a new Phase 2 clinical trial by filing an IND or its equivalent or attempt to join efforts with a strategic partner
Continued clinical testing in adult and pediatric brain tumors with WP1066 via physician sponsored trials
Financial Results for the Quarter Ended June 30, 2020

Research and development (R&D) expense was $3.3 million and $2.1 million for the three months ended June 30, 2020 and 2019, respectively. The increase of $1.2 million is mainly related to increased clinical trial activity, increased license fees and costs related to sponsored research agreements, costs related to manufacturing of additional drug product and two additional employees in R&D headcount.

General and administrative expense was $1.7 million and $1.5 million for the three months ended June 30, 2020 and 2019, respectively. The increase of $0.2 million was mainly attributable to increased stock-based compensation expense for annual employee stock options and increased costs for directors and officer’s liability insurance.

Loss from operations for the second quarter was $5.0 million compared to a net loss of $3.6 million for the second quarter of 2019. This increase was largely due to the above-mentioned increase in R&D.

Net loss for the second quarter of 2020 was $10.1 million, compared to a net loss of $1.2 million in the second quarter of 2019, and was attributed to the above-mentioned increase in R&D and the change in fair value on revaluation of warrant liability associated with warrants issued in conjunction with stock offerings. Changes in our stock price can result in a material gain or loss during the quarter related to the revaluation of our warrant liability. The loss from the change in the fair value of the warrant liability for the second quarter of 2020 was $5.1 million compared to a gain of $2.4 million in the same quarter in 2019. This is a non-cash item.

Liquidity and Capital Resources

As of June 30, 2020, we had cash and cash equivalents of $16.7 million and prepaid expenses and other of $3.0 million. We also had $1.9 million of accounts payable and $1.8 million of accrued expenses. A significant portion of the accounts payable and accrued expenses are due to work performed in relation to our clinical trials. For the six months ended June 30, 2020 and 2019, we used approximately $9.3 million and $9.2 million of cash in operating activities, respectively, which represents cash outlays for research and development and general and administrative expenses in such periods. For the six months ended June 30, 2020 and 2019, net proceeds from financing activities were $15.3 million and $20.8 million, respectively, predominately from the sale of our common stock and the exercise of warrants. Cash used in investing activities for the six months ended June 30, 2020 and 2019 was approximately $0.02 million and $0.03 million, respectively.

We believe that our existing cash and cash equivalents as of June 30, 2020 plus the $1.9 million cash raised subsequent to the quarter will be sufficient to fund our planned operations into the first quarter of 2021, without the issuance of additional equity for cash. Any such issuances should extend the funding of our planned operations beyond the first quarter of 2021. Such plans are subject to our stock price, market conditions, changes in planned expenses depending on clinical enrollment progress, the use of drug product or a combination thereof.

On August 12, 2020 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing reported pipeline updates, recent business highlights and second quarter 2020 financial results (Press release, Beam Therapeutics, AUG 12, 2020, View Source [SID1234563482]).

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!

"Throughout the first half of 2020, we made significant progress across all aspects of our business, culminating into naming the first two base editing development candidates from our portfolio. As we move closer to the clinic, we have also made the important strategic decision to establish a build-to-suit manufacturing facility, which will significantly enhance our capability to manufacture a wide range of base editing medicines," said John Evans, chief executive officer of Beam. "In addition, we continue to execute our strategy of establishing innovative partnerships to access new capabilities and to accelerate the development of base editors as a new class of precision genetic medicines for patients. Amidst the evolving COVID-19 situation, our team is performing well, and we remain on track to initiate IND-enabling studies in 2020 and file at least one Investigational New Drug application in 2021."

Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam added, "Achieving our first development candidates with base editing is one of the most important milestones for our company yet. BEAM-101 and BEAM-102 are highly differentiated editing programs that may enable a one-time treatment option for patients with sickle cell disease. Both candidates are supported by promising preclinical data, and we are working to advance them to the next stage of development to assess the impact they could have in treating this devastating disease."

Base Editing Progress

First Base Editing Development Candidates Named for Treatment of Sickle Cell Disease: Beam is pursuing two differentiated base editing approaches to treat hemoglobinopathies and recently named the first two development candidates from its portfolio. Promising preclinical data from these two complementary editing programs were presented at the 23rd American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Virtual Annual Meeting.
BEAM-101 is an adenine base editor (ABE) that reproduces single base changes observed in individuals with hereditary persistence of fetal hemoglobin, or HPFH, in which elevated levels of fetal hemoglobin protect these individuals from the effects of sickle cell disease or beta-thalassemia.

BEAM-102 is an ABE that directly corrects the causative mutation in sickle cell disease, converting it into a naturally-occurring human hemoglobin variant, Hb-G Makassar. Individuals with the Makassar variant have normal hematologic parameters and no evidence of hemoglobin polymerization or sickling of red blood cells.

Preclinical Non-Human Primate Data Validating Beam’s ABE Technology Presented by Verve Therapeutics: Verve Therapeutics presented preclinical proof-of-concept data in non-human primates, utilizing ABE technology licensed from Beam, that demonstrate the successful use of base editing to turn off a gene in the liver and thereby lower blood levels of either LDL cholesterol or triglyceride-rich lipoproteins. Beam and Verve previously announced a strategic collaboration, under which Verve has exclusive access to Beam’s base editing, gene editing and delivery technologies for human therapeutic applications against certain cardiovascular targets. After the completion of Phase 1 studies, Beam has the ability to participate in future development and commercialization, and share 50 percent of U.S. profits and losses, for any product directed against these targets.
Recent Business Highlights

Lease Agreement for Beam’s In-House Manufacturing Facility to Support Future Product Development: On August 11, 2020, Beam entered into a lease agreement with Alexandria Real Estate Equities, Inc. to build a 100,000 square foot current Good Manufacturing Practice (cGMP) compliant manufacturing facility in Research Triangle Park, North Carolina that will support a broad range of clinical programs. Beam will invest up to $83 million over a five-year period and anticipates that the facility will be operational by the first quarter of 2023. The project will be facilitated, in part, by a Job Development Investment Grant (JDIG) approved by the North Carolina Economic Investment Committee, which authorizes potential reimbursements to Beam based on new tax revenues generated through the project. The facility will be designed to support manufacturing for the company’s ex vivo cell therapy programs in hematology and oncology and in vivo non-viral delivery programs for liver diseases, with flexibility to support manufacturing of its viral delivery programs, and ultimately, scale-up to support potential commercial supply.

Collaboration Established with Magenta Therapeutics to Evaluate MGTA-117 as a Conditioning Regimen for Base Editing Therapies: In June 2020, Beam and Magenta Therapeutics announced a non-exclusive research and clinical collaboration agreement to evaluate the potential utility of MGTA-117, Magenta’s targeted antibody-drug conjugate, for conditioning of patients with sickle cell disease and beta-thalassemia receiving Beam’s base editing therapies.

Strategic Alliance with Boston Children’s Hospital to Accelerate Translational and Clinical Research in Gene Editing for Complex Conditions: Beam has entered into a strategic alliance agreement with Boston Children’s Hospital designed to facilitate the development of disease-specific therapies using Beam’s base editing technology. Under the terms of the agreement, Beam will sponsor multiple research programs at Boston Children’s Hospital to advance translation of Beam’s pipeline across certain therapeutic areas of interest, including programs in sickle cell disease and pediatric leukemias and exploration of new disease areas. Boston Children’s Hospital will also serve as a clinical site in the future to advance bench-to-bedside translation of Beam’s pipeline.

Research Collaboration Established with Institute of Molecular and Clinical Ophthalmology Basel (IOB) to Develop Gene Editing Programs for Ocular Diseases: Beam has established a research collaboration agreement with the IOB, under which the partners will leverage IOB’s expertise in the field of ophthalmology and Beam’s base editing technology to advance programs directed to the treatment of certain ocular diseases, including Stargardt disease. Through this collaboration, IOB will leverage insights from ProgStar, an international collaboration studying the natural history study of Stargardt disease progression and helping to determine clinical outcome measures that could be used in clinical trials of future therapies. Additionally, IOB has developed technology that images the retina and choroid with better quality and dimension, as well as living models of the retina, which can be used to study the therapeutic impact base editing could have on ocular diseases.
Second Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $228.0 million as of June 30, 2020.

Research & Development (R&D) Expenses: R&D expenses were $19.4 million for the quarter ended June 30, 2020, compared to $12.7 million for the quarter ended June 30, 2019. This increase was primarily due to the growth in the number of research and development employees and their related activities, as well as the expense allocated to R&D related to Beam’s leased facilities.

General &Administrative (G&A) Expenses: G&A expenses were $6.9 million for the quarter ended June 30, 2020, compared to $5.0 million for the quarter ended June 30, 2019. This increase was primarily a result of increased personnel related costs due to an increase in general and administrative employees and the cost of being a public company.

Net Loss: Net loss attributable to common stockholders was $34.2 million, or $0.69 per share, for the quarter ended June 30, 2020, compared to $21.1 million for the quarter ended June 30, 2019.

On August 12, 2020 TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage immunotherapy company with a pipeline of novel T cell therapies for patients suffering from cancer, reported financial results for the second quarter ended June 30, 2020 and provided a corporate update (Press release, TCR2 Therapeutics, AUG 12, 2020, View Source [SID1234563481]).

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!

"The second quarter was incredibly strong for the Company as we continued to deliver significant progress in our clinical trials of TC-210 and TC-110 despite the pandemic. We are particularly pleased to update that, following the earlier announcement of five of our first five cancer patients showing tumor regression with TC-210, one patient is now a confirmed partial response based on independent central review and experienced further tumor regression from 42% to 75%," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "The initial data from TC-210 provide meaningful readthrough for our TRuC-T cell platform and we believe our robust preclinical pipeline supports our next phase of growth as we build a leading cell therapy company treating solid tumors. The strengthening of our balance sheet through our recent financing puts us in an excellent position to execute on our objectives and provide further clinical and scientific updates in 2020."

Recent Developments

TCR2 announced positive interim data from the first five patients treated in the Phase 1 portion of the TC-210 Phase 1/2 clinical trial for mesothelin-expressing solid tumors. All five patients showed tumor regression including two with RECIST partial response, one of which is now confirmed and two patients with stable disease through six months. Translational data further demonstrated TRuC-T cell expansion and activation. A manageable toxicity profile was observed with only one patient exhibiting TC-210-related non-hematologic grade >2 toxicity and no evidence of neurotoxicity or on-target, off-tumor toxicity.
TCR2 highlighted preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II of the Company’s proprietary T Cell Receptor Fusion Construct (TRuC) T cells that co-express a PD-1:CD28 switch receptor, which acts as a cell-intrinsic mechanism to overcome PD-L1/PD-L2 mediated immunosuppression. Upon repeated antigen stimulation, co-expression of the switch receptor in mesothelin-targeting TC-210 T cells enhanced TCR downstream signaling, prevented PD-L1-mediatied functional T-cell inhibition, significantly increased proliferation and augmented the production of growth and effector cytokines.
TCR2 announced the expansion of its leadership team with key business development and regulatory affairs cell therapy experts with the appointments of Gregg McConnell as Head of Business Development and Viera Muzithras as Vice President of Regulatory Affairs.
Anticipated Milestones

TCR2 anticipates an interim update from the Phase 1 portion of the TC-210 Phase 1/2 clinical trial for patients with mesothelin-expressing solid tumors in 2H20.
TCR2 anticipates an interim update from the Phase 1 portion of the TC-110 Phase 1/2 clinical trial for patients with CD19+ non-Hodgkin lymphoma or adult acute lymphoblastic leukemia in 2H20.
TCR2 anticipates certification of its manufacturing facility in Stevenage, UK, in 2H20.
TCR2 anticipates an IND filing for a third TRuC-T cell program in 2021.
Financial Highlights

Cash Position: TCR2 ended the second quarter of 2020 with $124.8 million in cash, cash equivalents, and investments compared to $158.1 million as of December 31, 2019. As a result of the recent equity offering, TCR2 raised an additional $134.6 million. Net cash used in operations was $16.0 million for the second quarter of 2020 compared to $10.2 million for second quarter of 2019. TCR2 continues to project net cash use of $60-70 million for 2020.

R&D Expenses: Research and development expenses were $12.9 million for the second quarter of 2020 compared to $8.8 million for the second quarter of 2019. The increase in R&D expenses is primarily related to increase in headcount, activities related to the Phase 1/2 clinical trial of TC-210 and activities related to the Phase 1/2 clinical trial of TC-110.

G&A Expenses: General and administrative expenses were $3.8 million for the second quarter of 2020 compared to $3.3 million for the second quarter of 2019. The increase in general and administrative expenses was primarily due to an increase in personnel costs and costs associated with operations as a public company.

Net Loss: Net loss was $16.2 million for the second quarter of 2020 compared to $11.1 million for the second quarter of 2019, driven predominantly by increased R&D expenses.
Upcoming Events

TCR2 Therapeutics management is scheduled to participate at the following upcoming conferences.

2020 Wedbush PacGrow Healthcare Virtual Conference: Ian Somaiya, Chief Financial Officer of TCR2 Therapeutics, will provide a company update using a virtual platform on Wednesday, August 12, 2020 at 10:55am ET
Cantor Fitzgerald Virtual Global Healthcare Conference: Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics, and Ian Somaiya, Chief Financial Officer of TCR2 Therapeutics, will provide a company update using a virtual platform on Wednesday, September 16, 2020 at 8:40am ET