On November 15, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported a business update and an update on its cash position at the end of September 2021 (Press release, ERYtech Pharma, NOV 15, 2021, View Source [SID1234595692]).

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!

"While the outcome of our TRYbeCA-1 trial in second-line pancreatic cancer did not achieve its primary endpoint of overall survival, we remain encouraged by the observed improvement of overall survival in a subset of patients treated with FOLFIRI and will continue analyzing the sizeable TRYbeCA-1 data set in order to distill the reasons for this disappointing outcome," said Gil Beyen, CEO of ERYTECH. "We are also encouraged by the progress we are making towards seeking an approval for eryaspase for the treatment of ALL patients who experienced hypersensitivities to pegylated asparaginase. The dialogue with the FDA is continuing and we are hopeful we can submit our first BLA around year end. We were pleased with the granting of the Fast Track designation for this high unmet need indication in July."

Business Highlights

Path to BLA in hypersensitive ALL, based on results of NOPHO-sponsored Phase 2 trial

The NOPHO trial evaluated the safety and pharmacological profile of eryaspase in acute lymphoblastic leukemia (ALL) patients who had previously experienced hypersensitivity reactions to pegylated asparaginase therapy. In December 2020, positive trial results were presented at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting.

Eryaspase in combination with chemotherapy, administered every two weeks, provided a sustained asparaginase enzyme activity level, and was generally well tolerated with few hypersensitivity reactions.

The Company continued its interactions with the U.S. Food and Drug Administration (FDA) regarding a potential regulatory approval in this indication based on the NOPHO-sponsored trial. A pre-BLA meeting to discuss the submission of a Biologics License Application (BLA) took place in June after which the Company confirmed its intention to submit a BLA subject to successful completion of remaining activities.
In July, the Company announced that the FDA had granted eryaspase Fast Track designation for the treatment of ALL patients who have developed hypersensitivity reactions to E. coli-derived pegylated asparaginase.
Subject to review of remaining information requests, the Company intends to submit a BLA around year-end.

TRYbeCA-1, pivotal Phase 3 clinical trial in second-line advanced pancreatic cancer

As reported in late October, the Phase 3 TRYbeCA-1 trial did not meet the primary efficacy endpoint of overall survival (OS). The median OS for patients treated with eryaspase plus chemotherapy was 7.5 months, compared to 6.7 months for chemotherapy alone, with an OS hazard ratio (HR) of 0.92 in the intent-to-treat (ITT) population (p-value 0.375).

The prespecified subgroup of patients treated with eryaspase and FOLFIRI, an irinotecan-based chemotherapy, demonstrated a nominal increase in median OS of 2.3 months, from 5.7 to 8 months (HR = 0.77; per protocol population), which the Company believes merits further investigation.
Patients treated with eryaspase demonstrated improved disease control compared to patients treated with chemotherapy only. Other secondary endpoints showed nominal improvement.
The safety profile of eryaspase was consistent with earlier clinical trials results and safety reviews.

Final data are being analyzed and will be presented at an upcoming medical conference.

rESPECT, Phase 1 investigator-sponsored trial (IST) in first-line pancreatic cancer

rESPECT is a Phase 1 trial, sponsored by the Georgetown Lombardi Comprehensive Cancer Center, evaluating the safety of eryaspase in combination with mFOLFIRINOX as a first-line treatment for locally advanced and metastatic pancreatic cancer in approximately 18 patients.

Patient enrollment started in January 2021, and the first dose cohort (75 U/kg) of three patients was enrolled by the end of February. No dose-limiting toxicity (DLT) was observed, and the trial was escalated to the next dosing cohort (100 U/kg).
After review of the safety data in the first two dose cohorts, the dose escalation committee concluded that the novel combination of mFOLFIRINOX plus eryaspase was well tolerated with no DLT. Consequently, the maximum tolerated dose (MTD) was determined at a dose of 100 U/kg eryaspase.
Additionally, all six patients evaluated for response achieved disease control; three patients with objective response and three with stable disease.

The trial will continue enrolling up to approximately 18 patients. Reporting of final data is expected in the first half of 2022.

TRYbeCA-2, randomized Phase 2 clinical trial in triple-negative breast cancer (TNBC)

The TRYbeCA-2 trial is evaluating eryaspase in combination with gemcitabine and carboplatin chemotherapy, compared to chemotherapy alone, in metastatic TNBC. Target enrollment is approximately 64 patients. The primary end point of the trial is objective response rate.

Following the disappointing results of eryaspase in combination with a gemcitabine-based chemotherapy in the TRYbeCA-1 trial in second-line pancreatic cancer, the Company has, in consultation with the trial’s Steering Committee, decided to stop further enrollment in the TRYbeCA-2 trial.

The results of the patients enrolled in the TRYbeCA-2 trial to date are expected to be reported in the first half of 2022.

Process to review strategic options and partnering alternatives launched

The Company launched a process and appointed a specialized advisor to evaluate its strategic and partnering alternatives, including for the further development and commercialization of eryaspase. Gil Beyen, the CEO of ERYTECH, will lead these partnering efforts and take on the role of acting Chief Business Officer (CBO), as Jean-Sebastien Cleiftie, current CBO of ERYTECH will leave the Company at the end of this month.

Update on Q3 2021 Cash Position

As of September 30, 2021, ERYTECH had cash and cash equivalents totaling €38.0 million (approximately $43.9 million), compared with €44.4 million as of December 31, 2020 and €46.3 million on June 30, 2021. The €6.5 million decrease in cash position during the first nine months of 2021 was the result of a €7.8 million net cash utilization, which was mostly comprised of a €46.2 million net cash utilization in operating activities, €0.3 million used for investing activities and €38.8 million generated in financing activities, while the variation of the U.S. dollar against the euro led to a €1.3 million positive currency exchange impact.

Financing activities in the first nine months of 2021 included a $8 million placement in the United States through the Company’s at-the-market (ATM) equity financing program for net proceeds of €6.4 million, a $30 million Registered Direct offering for net proceeds of €22.4 million, and the drawdown of four tranches under the convertible notes (OCABSA) financing agreement signed with Alpha Blue Ocean, for net proceeds of €11.4 million.

At the date of this press release, nine OCABSA tranches have been called since the initiation of the program in June 2020. During the last 12 months, the OCABSA converted notes, together with the shares issued under the ATM program, have resulted in the issuance of 4,690,904 new shares and 235,690 warrants, representing 17.6% of the Company’s outstanding share capital.

The Company believes that its current cash position can fund its planned operating expenses and current programs into the second quarter of 2022. Further, the Company has engaged in cash preservation measures and believes that these measures, together with further utilization of the OCABSA agreement, subject to the regulatory limit of 20% dilution, could extend its cash horizon into the third quarter of 2022. The Company is currently exploring potential financing and partnering options to further extend its cash horizon beyond key 2022 development milestones.

The release on October 25, 2021 of the TRYbeCA-1 Phase 3 trial in pancreatic cancer, which did not meet its primary endpoint, is considered a triggering event for impairment analysis, which will require the Company to test tangible and intangible assets for possible impairment. The Company is therefore not in a position to announce full financial results for the third quarter of 2021 until current uncertainties on business assumptions are clarified. The Company will conduct an impairment analysis in light of its new business situation, which may potentially lead to an impairment of some of its assets.

Key News Flow and Milestones Expected Over the Next 12 Months

Planned BLA submission of eryaspase in hypersensitive ALL (around year end 2021)
Results from the Phase 1 rESPECT Trial of eryaspase in combination with mFOLFIRINOX in first-line pancreatic cancer (1H 2022)
Presentation of full dataset of TRYbeCA-1 trial at a medical meeting (1H 2022)
Data from the randomized Phase 2 TRYbeCA-2 trial of eryaspase in TNBC (1H 2022)
Third Quarter 2021 Conference Call Details

ERYTECH management will hold a conference call and webcast on Tuesday, November 16, 2021 at 8:30am EST / 2:30 pm CET to discuss the recent business and financial updates. Gil Beyen, CEO, Eric Soyer, CFO/COO, and Iman El-Hariry, CMO, will deliver a brief presentation, followed by a Q&A session.

The audio call is accessible via the below registering link:

View Source (Conference ID : 6425429)

Once registered, participants will receive a unique access code and the call number details to join the teleconference.

The webcast can be followed live online via the link: View Source

An archived replay of the call will be available for 7 days by dialing + 1 855 859 2056, Conference ID: 6425429#.

An archive of the webcast will be available on ERYTECH’s website, under the "Investors" section at investors.erytech.com

Financial Calendar 2021

Business Update and Financial Highlights for the Fourth Quarter and Full Year 2021: March 11, 2022 (after U.S. market close), followed by a conference call & webcast on March 14, 2022 (2:30pm CET/8:30am ET)

ERYTECH plans on attending the following upcoming investor conferences:

Jefferies 2021 Global Healthcare Conference, November 16-19, London
LifeSci Partners 11th Annual Corporate Access Event, January 5-7, 2022
H.C. Wainwright, BioConnect Conference, January 10-13, 2022
JPMorgan HealthCare Conference, January 10-13, 2022, San Francisco

On November 15, 2021 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics to treat chronic diseases and extend healthy lifespan, reported its financial results for the third quarter ended September 30, 2021 (Press release, CohBar, NOV 15, 2021, View Source [SID1234595708]).

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 were pleased to report positive topline data from our first clinical study this past quarter and believe that this data not only demonstrates the potential of our CB4211 program but also validates our unique approach of creating novel therapeutics sourced from the mitochondrial genome," stated Dr. Joseph Sarret, Chief Executive Officer. "With a strengthened balance sheet from our recent financing, we are now well-positioned to continue to advance our pipeline."

Third Quarter 2021 and Recent Highlights

Positive Topline Data Announced from the Phase 1a/1b Study of CB4211 Being Developed for the Treatment of Nonalcoholic Steatohepatitis (NASH) and Obesity: In August, the company announced topline results from the multi-center, randomized, double-blind, placebo-controlled Phase 1a/1b clinical study of CB4211, under development for NASH and obesity. The study met its primary endpoints as CB4211 was well-tolerated and appeared safe with no serious adverse events. The evaluation of the exploratory endpoints showed robust and statistically significant improvements in key biomarkers of liver damage, ALT and AST, as well as in glucose levels in the CB4211 group compared to placebo. There was a trend towards lower body weight in the CB4211 group after four weeks of treatment.
CB4211 Phase 1b Data Selected for Late Breaker Presentation at The Liver Meeting 2021: In October, the company announced that data from its CB4211 Phase 1a/1b clinical study had been selected for presentation during the late-breaking poster session at The American Association for the Study of Liver Diseases (AASLD) Annual Meeting (The Liver Meeting 2021). The lead author on the poster was Dr. Rohit Loomba, MD, MHSc, Professor of Medicine, Director, NAFLD Research Center, and Director of Hepatology, University of California at San Diego. The poster can be viewed by visiting: CohBar’s Publication Page.
U.S. Patent Granted Covering CB4211 Compositions and Use for Treating NASH: In September, the company announced that the United States Patent and Trademark Office had granted a patent covering CohBar’s lead candidate CB4211 and related compositions, as well as methods of treatment, including methods of treating NASH. This foundational patent will be eligible for listing in the FDA Orange Book upon approval of CB4211 as a therapeutic in the United States.
Completed $15M Financing: On November 1st, the company completed an underwritten public offering of common stock and warrants, with aggregate gross proceeds of approximately $15 million. The company intends to use the proceeds from this offering to fund research and development and other general corporate purposes.
Gained additional bank research coverage: Recently, Wall Street bank Cantor Fitzgerald initiated coverage on CohBar and issued a research report on the company.
Carol Nast and Joanne Yun, Ph.D. Appointed to the Board of Directors: The company announced the appointment Carol Nast and Joanne Yun, Ph.D. as independent directors. Ms. Nast has spent her career in executive level positions with both large multinational companies and early-stage companies in the medical industry. Dr. Yun brings extensive research and development, commercial, and governance experience from the pharmaceutical industry.
Third Quarter 2021 Financial Highlights

Cash and Investments: The company had cash and investments of $15 million as of September 30, 2021, compared to $21 million as of December 31, 2020. The cash burn for the quarter ended September 30, 2021, was approximately $3.3 million.

R&D Expenses: Research and development expenses were $1.6 million for the three months ended September 30, 2021, compared to $1.2 million in the prior year quarter. The increase in research and development expenses was primarily due to the investment in the company’s research programs focused on the continued development of its peptides partially offset by a decrease in stock based compensations costs.

G&A Expenses: General and administrative expenses were $1.8 million for the three months ended September 30, 2021, compared to $1.4 million in the prior year quarter. The increase in general and administrative expenses was primarily due to higher stock-based compensation costs.

Net Loss: For the three months ended September 30, 2021, net loss, which included $0.7 million of non-cash expenses, was $3.4 million, or $0.05 per basic and diluted share. For the three months ended September 30, 2020, net loss, which included $0.9 million of non-cash expenses, was $3.2 million, or $0.06 per basic and diluted share.
Third Quarter Investor Call:

For individuals participating in the Investor Call, please call into the conference audio approximately 10 minutes prior to its start.

An audio replay of the call will be available beginning at 8:00 p.m. Eastern Time on November 15, 2021, through 11:59 p.m. Eastern Time on December 6, 2021. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID# 10161656. The audio recording will also be available at www.cohbar.com during the same period.

On November 15, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported the acceptance of an abstract with the results of its expanded access program (EAP) evaluating eryaspase in acute lymphoblastic leukemia (ALL) for poster presentation at the upcoming 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held from December 11-14, 2021, both in Atlanta, Georgia and virtually (Press release, ERYtech Pharma, NOV 15, 2021, View Source [SID1234595525]).

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!

Abstract #1214: Expanded Access Program: Evaluating Safety of Erythrocytes Encapsulating L-Asparaginase in Combination with Polychemotherapy in Patients Under 55 Years Old with Acute Lymphoblastic Leukaemia (ALL) at Risk to Receive Other Formulations of Asparaginase

The eryaspase Expanded Access Program (EAP) was conducted at ten clinical sites in France and enrolled 18 patients. The EAP evaluated tolerability and biological efficacy in patients under 55 years of age with ALL, unable or at risk to receive any other available asparaginase formulation. Patients in this study had developed hypersensitivities to prior E-Coli- and Erwinia-derived asparaginase therapies.

Hypersensitivity is the most common cause of truncated asparaginase therapy which has been associated with decreased event free survival. In the EAP and consistently across eryaspase ALL studies, including the NOPHO1 study, eryaspase provides a sustained asparaginase enzyme activity level with few hypersentivity reactions and is generally well tolerated in combination with chemotherapy.

Eryaspase provides a promising additional option for patients for whom further asparaginase treatment is contraindicated. The company intends to move forward towards the submission of a BLA to the US Food and Drug Administration (FDA) for eryaspase in hypersensitive patients.

The study findings will be presented as a poster presentation by Prof Dr. Yves Bertrand, Institute of Pediatric Hematology and Oncology, Civil Hospital of Lyon, Lyon, France.

On November 15, 2021 Redx Pharma (AIM: REDX), the clinical-stage biotechnology company focused on discovering and developing novel, small molecule, highly targeted therapeutics for the treatment of cancer and fibrotic disease, reported that the first patient has been dosed in the monotherapy arm of the Phase 2 clinical trial of its investigational drug RXC004 in patients with advanced microsatellite stable (MSS) metastatic colorectal cancer (mCRC) who have progressed following treatment with standard of care (Press release, Redx Pharma, NOV 15, 2021, View Source [SID1234595549]). RXC004 is Redx’s wholly-owned, highly potent and selective, orally active once-daily Porcupine inhibitor being developed as a targeted therapy for Wnt-ligand driven cancer.

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 multi-centre Phase 2 clinical trial (clinicaltrials.gov NCT04907539) will evaluate preliminary efficacy and safety of RXC004 in genetically-selected patients with Ring finger protein 43 (RNF43) or R-spondin (RSPO) aberrated, advanced MSS mCRC. Topline data is expected to report in the first half of 2023.

A second arm of the trial, evaluating RXC004 in combination with the anti-PD-1 antibody nivolumab in patients with MSS mCRC, is expected to commence in the first half of 2022 once a recommended dose has been established in the ongoing Phase 1 dose escalation combination trial.

D r Natalie Cook, University of Manchester and Christie NHS Foundation Trust, UK, and International Coordinating Investigator of the study in the UK, commented: "Microsatellite stable metastatic colorectal cancer is a devastating disease, with limited treatment options. A subgroup of these colorectal cancers possess RNF43 mutations or RSPO fusions leading to activation of the Wnt pathway as a driver of the cancer. This study will assess whether RXC004, a novel Porcupine inhibitor, has a clinically meaningful anti-cancer effect in this well-defined patient cohort.”

Lisa Anson, Chief Executive Officer of Redx Pharma, added: "We are excited to be dosing patients in Redx’s first ever Phase 2 clinical trial of a wholly-owned drug candidate, an important corporate milestone. Our encouraging Phase 1 results, recently reported at the ESMO (Free ESMO Whitepaper) Congress, combined with our preclinical data, strongly support the hypothesis that patients with Wnt-ligand driven tumours could benefit from RXC004."

A second Phase 2 clinical trial evaluating RXC004 as a monotherapy in advanced genetically selected pancreatic cancer and unselected biliary cancer is also expected to start in 2021.

About microsatellite stable metastatic colorectal cancer (MSS mCRC)
Metastatic colorectal cancers have a poor prognosis with a 5-year survival rate of approximately 15% (1). Standard first line and second line treatments are combinations of chemotherapy and a VEGF inhibitor or EGFR inhibitor. MSS cancers account for 95% of metastatic CRC and tend to be unresponsive to treatment with immune checkpoint inhibitors. In the third line treatment setting the response rate to standard agents is <5%, median progression free survival is approximately 2 months and overall survival approximately 6 months (2,3). Approximately 8% of MSS mCRC patients have Wnt-ligand driven tumours (3% RNF43 mutations and 5% RSPO fusions) (4)

About RXC004
RXC004 is a wholly owned, potent, selective, oral, small-molecule inhibitor of the Porcupine enzyme, a key activator of Wnt ligands in the Wnt-signalling pathway. The Wnt pathway is well established as a driver of both tumour growth and immune evasion. Aberrant Wnt signalling contributes directly to tumour growth and plays an important role in immune evasion, which has also been linked to resistance to immune-checkpoint inhibitors (ICIs) such as nivolumab. By selecting patients with tumours that have high Wnt-ligand dependency, such as those with loss of function mutations in the RNF43 gene and fusions in the RSPO gene family, RXC004 has an opportunity to both directly inhibit the tumour growth and have an immune-enhancing effect to allow the patient’s immune system to better recognise and attack the tumour.

ICIs such as anti-PD-1 antibodies have revolutionised the treatment of cancer, but do not work in all patients. Wnt-pathway activation can enhance the ability of the tumour to evade destruction by the immune system and has been linked to lack of response to ICIs in these tumours. Redx scientists have observed in preclinical studies that RXC004 can block activation of the Wnt pathway and restore the ability of the immune system to fight the tumour. Thus, RXC004 offers potential to address some of the shortcomings of ICI therapies through increasing both response rates and duration of response, particularly in patient populations unresponsive to ICI therapy.

On November 15, 2021 T-knife Therapeutics, Inc., a biopharmaceutical company dedicated to developing novel therapeutics to fight cancer, reported preclinical data demonstrating that its novel MAGE-A1-specific T cell receptor (TCR) induced meaningful anti-tumor activity and enhanced engraftment as compared to human donor derived TCRs (Press release, T-Knife, NOV 15, 2021, View Source [SID1234595592]). In vivo data presented at the SITC (Free SITC Whitepaper) annual meeting highlight the potential of T-knife’s lead product candidate TK-8001, a TCR engineered T cell therapy (TCR-T) that is being developed to treat MAGE-A1 positive solid tumors. The company plans to initiate a first-in-human, Phase 1/2 trial in early 2022.

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!

T-knife has used its proprietary HuTCR platform to discover multiple naturally optimized high-affinity TCRs specific for the cancer-testis antigen MAGE-A1. These MAGE-A1-specific TCRs were isolated from the HuTCR platform and in vitro and in vivo activity were compared to different human-derived MAGE-A1-specific TCRs. T-knife’s TCRs, including the TCR incorporated in TK-8001, were found to be of higher affinity than human derived TCRs and resulted in superior anti-tumor activity, as shown in a syngeneic preclinical model. In addition, TK-8001 exhibited enhanced engraftment rates, as measured by TCR+ cells in peripheral blood. The data generated in these preclinical evaluations highlight the potential therapeutic benefit of TK-8001.

In a separate trial-in-progress poster at the conference, the company provided detail on its planned IMAG1NE Phase 1/2 trial of TK-8001 to evaluate the safety, tolerability, and clinical activity of TK-8001 in advanced-stage, metastatic, MAGE-A1 positive solid tumors. The trial will be conducted in two phases, including a dose escalation phase designed to select the optimal dose to be utilized in a subsequent open-label expansion cohort. A supporting translational poster validating MAGE-A1 as an attractive solid tumor target was also presented.

"It is gratifying for our R&D team to present this exciting preclinical efficacy data in solid tumors expressing MAGE-A1," said Elisa Kieback, Ph.D., founder and Chief Technical Officer of T-knife Therapeutics. "Advancing this program toward the clinic and clinical proof of concept for our TCR platform is an important corporate milestone and highlights the substantial progress we have been making over the past year. We look forward to generating clinical data with TK-8001 with the initiation of our IMAG1NE Phase 1/2 study in select patients with MAGE-A1 expressing solid tumors.

About TK-8001 TCR-T

TK-8001 is a CD8 TCR-T specific for the Melanoma-associated Antigen Gene-A1, or MAGE-A1. MAGE-A1 is associated with hallmarks of aggressive cancers and poor clinical prognosis, and there is an emerging body of evidence indicating its involvement as a potential driver of tumorigenesis. MAGE-A1 represents an attractive therapeutic target given the high unmet need in MAGE-A1 expressing cancers, no reported protein expression in healthy tissues other than testis and significant consistency of expression between the primary tumor and metastases. As high affinity TCRs specific for MAGE-A1 peptides in humans are eliminated through central tolerance, we believe our HuTCR platform is a differentiated means to discover and select MAGE-A1 specific TCRs with an optimal affinity and high specificity profile.

About the IMAG1NE Phase 1/2 Trial

The IMAG1NE Phase 1/2 trial is an open-label, multi-center Phase 1/2 trial designed to evaluate the safety and efficacy of TK-8001 in patients with MAGE-A1 positive solid tumors. The first phase of the IMAG1NE trial is planned to enroll approximately 6 to 18 patients to assess the initial safety and tolerability of ascending dose levels of TK-8001. A key outcome of the first phase of the trial is to select a dose to be evaluated in an open label expansion phase of the study. The open label expansion phase of the IMAG1NE trial is designed to enroll approximately 30 additional participants to assess the efficacy of TK-8001 across a range of tumor indications