Marker Therapeutics Receives FDA Orphan Drug Designation for its Multi-Antigen Targeted T Cell Therapy for Pancreatic Cancer

On January 19, 2022 Marker Therapeutics, Inc. (NASDAQ:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported that the United States Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug designation to MT-601, a multi-tumor-associated antigen (MultiTAA)-specific T cell product optimized for the treatment of patients with pancreatic cancer.

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"The FDA’s orphan drug designation underscores MT-601’s potential as a treatment for pancreatic cancer, a cancer typically diagnosed at an incurable advanced stage with a total overall 5-year survival rate of 10%," said Peter L. Hoang, President & CEO of Marker Therapeutics. "Our novel therapy has shown encouraging results in an ongoing Phase 1 trial sponsored by Marker’s partners at the Baylor College of Medicine. In results reported at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Annual Meeting, our therapy has demonstrated the potential to safely produce durable responses in combination with chemotherapy as a first-line treatment option for patients with advanced or metastatic pancreatic adenocarcinoma. The results also revealed that epitope spreading was consistent in responders to Multi-TAA-specific T cells. Following MT-401 for the treatment of post-transplant acute myeloid leukemia (AML), MT-601 is Marker’s second novel MultiTAA-specific T cell product to receive orphan drug designation and the first in a solid tumor indication, underscoring the potential of Marker’s multi-antigen targeting T cell therapy approach in both solid tumors and blood cancers."

Marker developed MT-601, a new product targeting six tumor-associated antigens (PRAME, NY-ESO-1, Survivin, MAGE-A4, SSX2, WT1) highly expressed in pancreatic cancer. The Company intends to initiate a Phase 1 multicenter study of MT-601 administered in combination with front-line chemotherapy to patients with locally advanced unresectable or metastatic pancreatic cancer. Marker designed the study to include an initial antigen escalation period followed by a dose escalation period and will enroll 20 – 25 patients for the study.

The Company plans to file an Investigational New Drug Application (IND) for MT-601 for the treatment of pancreatic cancer in 2022.

Orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax credits, FDA user-fee benefits, and seven years of market exclusivity in the United States following marketing approval by the FDA. The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. For more information about orphan designation, please visit the FDA website at www.fda.gov.

Novartis-backed ImmPACT Bio snags $111M and new CEO as cancer CAR-T yields phase 1 results

On January 20, 2022 ImmPACT Bio USA, Inc. ("ImmPACT Bio"), a clinical-stage company developing transformative logic-gate based chimeric antigen receptor (CAR) T-cell therapies for treating cancer, reported that it has closed $111 million in Series B financing, led by venBio Partners along with co-leads Foresite Capital and Decheng Capital (Press release, ImmPACT-Bio, JAN 19, 2022, View Source [SID1234605608]). Surveyor Capital (a Citadel company) also joined this financing round. Additional investors include the company’s existing investors OrbiMed, Novartis Venture Fund, RM Global Partners (RMGP), and Bukwang Pharmaceutical. The company also announced the appointment of Sheila Gujrathi, M.D., as chair of its board of directors, and Sumant Ramachandra, M.D., Ph.D., M.B.A., as president and chief executive officer (CEO), who will also join ImmPACT Bio’s board.

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In addition, ImmPACT Bio provided an update to the Phase 1 clinical study evaluating its CD19-CD20 bi-specific CAR T in patients with relapsed or refractory B-cell non-Hodgkin’s lymphoma (R/R B-cell NHL) at University of California, Los Angeles (UCLA). In a total of eight patients treated to date with 12 months median followup, seven patients achieved and remain in complete remission (CR). A favorable tolerability profile was noted with no treatment-related neurotoxicity and no cytokine release syndrome above grade 1. Data will be presented in a future scientific forum.

The company’s logic-gate based CAR T platforms address key biological challenges in treating cancer. ImmPACT Bio’s technologies are specifically designed to address antigen escape, ‘on-target – off-tumor’ toxicities, and the immunosuppressive tumor microenvironment. The company’s technologies are based on the work of pioneering scientists Yvonne Chen, Ph.D., and Antoni Ribas, M.D., Ph.D., both from UCLA, and Gideon Gross, Ph.D., from the MIGAL-Galilee Research Institute. Proceeds from the financing will be used to further the company’s development of logic-gate-based CAR T-cell platforms.

"ImmPACT Bio is thrilled to receive such strong support from this outstanding group of investors," said newly appointed ImmPACT Bio CEO Dr. Ramachandra. "We are encouraged by these initial results of our Phase 1 clinical study and look forward to advancing our pipeline of transformative next-generation CAR T-cell therapies that harness the immune system and address key challenges for current cell therapies in cancer."

"We believe ImmPACT Bio’s technology platforms will help to expand the curative potential of cell therapies in certain cancers and to address challenges that have limited the efficacy of cell therapies in hard-to-treat tumor types," said Richard Gaster, M.D., Ph.D., managing partner at venBio Partners. "We are excited to work with the scientists and clinicians at ImmPACT Bio to translate their engineering innovations into novel cell therapy products for multiple malignancies."

"We’re honored to work with ImmPACT Bio as they develop novel strategies to address unmet oncology needs," said Jim Tananbaum, founder and CEO of Foresite Capital. "With Drs. Gujrathi and Ramachandra at the helm, this is an incredibly exciting time for ImmPACT Bio and the patients who are waiting for next-generation cancer therapies."

"ImmPACT Bio’s logic-gate-based CAR T platform holds tremendous potential to usher in a step-change in the treatment of solid tumors and blood cancers. We are pleased to join this distinguished group of healthcare investors supporting this world-class team to advance what we believe could become a new paradigm in cancer treatment," said Min Cui, Ph.D., founder and managing director of Decheng Capital.

As part of the financing, Yvonne Yamanaka, Ph.D., of venBio Partners, Cindy Xiong, Ph.D., of Foresite Capital, and Min Cui, Ph.D., of Decheng Capital will join the company’s board.

Sheila Gujrathi Named New Chair of Board of Directors

ImmPACT Bio also announced the appointment of Dr. Gujrathi as board chair. Erez Chimovits, partner at OrbiMed and ImmPACT Bio’s chairman, will remain on the company’s board.

"It is my pleasure to welcome Sheila to the board of ImmPACT Bio," said Mr. Chimovits. "This is a transformational period in ImmPACT Bio’s evolution, and Sheila is an exceptional fit for the company’s next stage of growth, given her deep experience leading biotech companies."

"ImmPACT Bio possesses an innovative and proprietary CAR T-cell platform, compelling clinical and early-stage assets, and a dynamic leadership team. Today’s announcement of the company’s Series B financing bolsters the potential to transform the oncology therapeutic landscape," said Dr. Gujrathi. "I’m thrilled to join as board chair and help ImmPACT Bio achieve its vision of developing transformative oncology medicines for cancer patients who have exhausted their treatment options."

Dr. Gujrathi is currently a venture advisor at OrbiMed and serves as executive chair of Ventyx Biosciences, chair of ADARx Pharmaceuticals, and director of Janux Therapeutics. She previously served as chair of Turning Point Therapeutics and is the co-founder and former CEO of Gossamer Bio. Prior to Gossamer, Dr. Gujrathi served as chief medical officer of Receptos, which was acquired by Celgene. Previously, she held development leadership positions at Bristol-Myers Squibb and Genentech where she led development and numerous global regulatory filings and approvals for immunology and oncology programs. Earlier in her career, she was a management consultant in McKinsey & Company’s healthcare practice. Dr. Gujrathi received both her M.D. in the accelerated Honors Program in Medical Education and her B.S. in biomedical engineering with highest distinction from Northwestern University. She completed her internal medicine internship and residency at Brigham and Women’s Hospital and Harvard Medical School and additional fellowship training in allergy/immunology at University of California, San Francisco (UCSF) and Stanford University Medical Center.

Industry Veteran Sumant Ramachandra Named President and CEO

Dr. Ramachandra brings more than 30 years’ experience in healthcare, including more than two decades in the pharmaceutical and medical device industry across multiple roles. CEO Rick Kendall, Ph.D., will continue as chief scientific officer (CSO) of ImmPACT Bio.

"I am confident ImmPACT has selected in Sumant the right leader to build on our success to date and to drive value for the company as he has done for several companies in the healthcare industry," said Dr. Gujrathi. "I want to thank Rick for his leadership in bringing ImmPACT Bio forward to a clinical-stage company and I look forward to working closely with this ‘dream team’ and contributing to our continued growth."

"I am excited to join ImmPACT Bio at such a crucial time in our development. I am impressed with the strong scientific foundation that the co-founders, management team, and board have built across its programs and platforms that aim to bring curative therapies to patients living with cancer," said Dr. Ramachandra.

Prior to joining ImmPACT Bio, Dr. Ramachandra was chief science, technology, and medical officer at Baxter International, which he joined in June 2017. In addition to these responsibilities, he was appointed president of Baxter Global Pharmaceuticals in mid-2019 and was appointed as chair of Baxter’s Global Inclusion Council focused on inclusion and diversity. Previously, he was at Pfizer, most recently as senior vice president, head of research and development, Pfizer Essential Health. He served as CSO at Hospira from 2008 to 2015 prior to Pfizer’s acquisition of Hospira in 2015. Earlier in his career, Dr. Ramachandra worked with Pfizer and Merck & Co. in various senior-level oncology global product development, medical affairs, and business development and licensing roles, and as a clinical pharmacologist. Before entering the industry in 2000, he was an intern and resident physician at Massachusetts General Hospital and Harvard Medical School.

Dr. Ramachandra earned a Ph.D. in experimental pathology in the study of chronic lymphocytic leukemia (CLL), an M.D., and a B.A. in biochemistry at Rutgers University. In addition, he holds an M.B.A. from the Wharton School of the University of Pennsylvania.

Leading the Next Generation of CAR T

For hematological malignancies, the company’s lead program is a CD19-CD20 bispecific ‘OR-gate’ CAR T designed to address antigen escape, which is a key challenge for current approved CD19 therapies. Early results indicate the potentially best-in-class bispecific CAR T-cell candidate is safe and effective in patients with R/R B-cell NHL.

Treating solid tumors is even more challenging, partially due to off-target safety considerations and the immunosuppressive tumor microenvironment. ImmPACT Bio is developing novel strategies for addressing this unmet need, designing next-generation CAR T to assure tumor specificity and efficacy in the hostile tumor environment, while protecting the normal tissue.

ImmPACT Bio’s second platform features engineered T cells with an activator/inhibitor ‘NOT-gate’ CAR combination that uniquely differentiates between tumor and normal cells. The activator CAR kills the tumor cells while the inhibitory CAR protects normal cells. The activator/inhibitor platform technology has the potential to deliver multiple products across solid tumors by pairing different activator/inhibitor combinations, providing diverse opportunities in an emerging and promising area of cancer therapy.

The company’s third platform, a TGF-β CAR that can be modularly equipped with tumor-targeting binding domains to generate bispecific ‘OR-gate’ CARs, rewires the T-cell response to TGF-β to convert this potent immunosuppressive cytokine into a T-cell stimulant. Single-chain bispecific CARs that simultaneously convert TGF-β signaling while directly targeting surface-bound tumor antigens enable T cells to robustly eliminate tumor cells in otherwise highly immunosuppressive microenvironments.

ESSA Pharma Announces First Patient Dosed in a Phase I/II Clinical Trial of EPI-7386 in Combination with Enzalutamide in Patients with Metastatic Castration-Resistant Prostate Cancer

On January 19, 2022 ESSA Pharma reported the first patient dosed in the Company-sponsored Phase I/II study to evaluate the safety, tolerability and preliminary efficacy of ESSA’s lead product candidate, EPI-7386, a first-in-class N-terminal domain androgen receptor inhibitor, in combination with Astellas Pharma and Pfizer’s ligand-binding domain androgen receptor inhibitor, enzalutamide, in patients with metastatic castration-resistant prostate cancer ("mCRPC") (Press release, ESSA, JAN 19, 2022, View Source [SID1234605589]).

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"The initiation of this combination trial with Astellas is a watershed moment for ESSA as we investigate the potential clinical benefit of inhibiting the androgen receptor through two independent pathways in the treatment of patients with mCRPC who have not yet received treatment with a second-generation antiandrogen drug," said Dr. David. R. Parkinson, Chief Executive Officer, ESSA Pharma Inc. "Combining our two therapies will simultaneously target both ends of the androgen receptor. In preclinical models, we have seen that combining EPI-7386 with current antiandrogens can lead to deeper and broader inhibition of androgen biology. This Phase 1/2 trial marks the first of a series of clinical studies to evaluate EPI-7386 in combination with current antiandrogen therapies in patients with mCRPC, with additional Phase 1/2 combination trials anticipated to begin in 2022."

The Phase 1/2 clinical trial (NCT05075577) begins with an initial phase 1 portion wherein the doses of each drug are adjusted followed by a phase 2 portion wherein the single agent enzalutamide is compared to the combination of enzalutamide and EPI-7386. The phase 1 study is expected to enroll up to 30 mCRPC patients who have not yet been treated with second-generation antiandrogen therapies. The goal of the phase 1 portion of the study is to evaluate the safety and tolerability of the drug combination and establish the recommended phase 2 doses for EPI-7386 and enzalutamide when dosed in combination. The phase 2 study is expected to enroll 120 mCRPC patients who have not yet been treated with second-generation antiandrogen therapies. The goal of the phase 2 part of the study is to evaluate the safety, tolerability and antitumor activity of EPI-7386 in combination with a fixed dose of enzalutamide compared with enzalutamide as a single agent.

About EPI-7386
EPI-7386 is an investigational, highly-selective, oral, small molecule inhibitor of the N-terminal domain of the androgen receptor. EPI-7386 is currently being studied in a Phase 1 clinical trial (NCT04421222) in men with metastatic castration-resistant prostate cancer whose tumors have progressed on current standard-of-care therapies. The Phase I clinical trial of EPI-7386 began in calendar Q3 of 2020 following FDA allowance of ESSA’s Investigational New Drug application and Health Canada acceptance. EPI-7386 is also currently being studied in a Phase 1/2 clinical trial (NCT05075577) in mCRPC patients who have not yet been treated with second-generation antiandrogen therapies. The U.S. FDA has granted Fast Track designation to EPI-7386 for the treatment of adult male patients with mCRPC resistant to standard-of-care treatment. ESSA retains all rights to EPI-7386 worldwide.

Natera to Present Latest Signatera™ MRD Data in Colorectal and Pancreatic Cancers at the ASCO GI Symposium 2022, and Host Special Investor Call

On January 19, 2022 Natera reported the company, along with its collaborators, will present new data on Natera’s personalized, tumor-informed molecular residual disease (MRD) test, Signatera, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s 2022 Gastrointestinal Cancers Symposium (ASCO GI), taking place January 20 – 22, 2022 (Press release, Natera, JAN 19, 2022, View Source [SID1234605588]).

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Natera has two oral presentations and four collaborative poster presentations highlighting clinical findings on the performance of Signatera across colorectal (CRC), pancreatic and anal cancers. The oral presentations include an interim analysis from the GALAXY cohort of the CIRCULATE-Japan trial, based on 12-month outcomes from more than 1,000 patients, demonstrating how MRD testing can help predict treatment benefit for CRC patients post-surgery, regardless of stage. A second oral presentation addresses results from the first major study on the utility of MRD testing for patients with pancreatic cancer, compared to the standard of care biomarker CA19-9.

"The predictive data coming out of CIRCULATE-Japan is exceptional, and we think it has the potential to transform clinical practice in CRC," said Solomon Moshkevich, general manager of oncology at Natera. "We also look forward to presenting high-impact data from our pancreatic and other GI studies, further expanding the pan-cancer utility of Signatera’s personalized and tumor-informed method."

Natera and its collaborators will present the following studies:

Oral Abstract Session C: Cancers of the Colon, Rectum and Anus |
Presenter: Masahito Kotaka, M.D., Ph.D. | Jan. 22, 11:00 AM – 12:30 PM PST
Association of circulating tumor DNA dynamics with clinical outcomes in the adjuvant setting for patients with colorectal cancer from an observational GALAXY study in CIRCULATE-Japan

Oral Abstract Presentation: Emerging Roles of ctDNA on the Horizon of GI Cancers |
Presenter: Gregory Botta, M.D., Ph.D. | Jan. 20, 3:30 PM – 5:00 PM PST
Association of personalized and tumor-informed ctDNA with patient survival outcomes in pancreatic adenocarcinoma

Poster Session C: Cancers of the Colon, Rectum, and Anus |
Presenter: Georges Azzi, M.D. | Jan. 22, 6:30 AM – 7:55 AM PST
Circulating tumor DNA-based molecular residual disease detection and recurrence monitoring in patients with advanced or metastatic anal squamous cell carcinoma

Poster Session C: Cancers of the Colon, Rectum, and Anus |
Presenter: RuoBing Xue, M.D. | Jan. 22, 6:30 AM – 7:55 AM PST
Prospective study of the correlation of ctDNA with pathologic complete remission (pCR) and other efficacy outcomes in rectal cancer patients undergoing neoadjuvant chemotherapy and radiation

Poster Session C: Cancers of the Colon, Rectum, and Anus |
Presenter: Sherise Rogers, M.D., M.P.H. | Jan. 22, 6:30 AM – 7:55 AM PST
A phase II randomized therapeutic optimization trial for subjects with refractory metastatic colorectal cancer using circulating tumor DNA (ctDNA): Rapid 1 trial

Poster Session C: Cancers of the Colon, Rectum, and Anus |
Presenter: Marwan Fakih, M.D. | Jan. 22, 6:30 AM – 7:55 AM PST
Exploratory biomarker analyses of the single-arm, phase 2 study of regorafenib plus nivolumab in patients (pts) with mismatch repair-proficient (pMMR)/microsatellite stable (MSS) colorectal cancer (CRC)

Additionally, Natera will host an investor call to discuss ASCO (Free ASCO Whitepaper) GI results:

Event:

ASCO GI Conference Data Review

Date:

Monday, Jan. 24, 2022

Time:

1:30 p.m. PT (4:30 p.m. ET)

Live Dial-In:

(877) 823-0171, Domestic

(617) 500-6932, International

Password:

7697903

Webcast:

View Source

A webcast replay will be available at investor.natera.com.

About Signatera

Signatera is a custom-built circulating tumor DNA (ctDNA) test for treatment monitoring and molecular residual disease (MRD) assessment in patients previously diagnosed with cancer. The test is available for both clinical and research use, and has been granted three Breakthrough Device Designations by the FDA for multiple cancer types and indications. The Signatera test is personalized and tumor-informed, providing each individual with a customized blood test tailored to fit the unique signature of clonal mutations found in that individual’s tumor. This maximizes Signatera’s accuracy for detecting the presence or absence of residual disease in a blood sample, even at levels down to a single tumor molecule in a tube of blood. Signatera is intended to detect and assess how much cancer is left in the body, to identify recurrence earlier and to help optimize treatment decisions.

Qualigen Therapeutics Secures Worldwide Rights to G4-Selective Transcription Inhibitors from UCL to Develop as Cancer Therapeutics

On January 19, 2022 Qualigen Therapeutics, Inc. (Nasdaq: QLGN), a biotechnology company focused on developing treatments for adult and pediatric cancers with potential for Orphan Drug Designation, has reported the exclusive worldwide in-license of a genomic quadruplex (G4)-selective transcription inhibitor drug development program, which was developed by Professor Stephen Neidle and his team from the UCL School of Pharmacy (Press release, UCLB, JAN 19, 2022, View Source [SID1234605585]). The license agreement was carried out by UCL Business.

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Qualigen intends to develop the lead compound, now called QN-302, as a treatment for pancreatic ductal adenocarcinoma (PDAC), which represents the vast majority of pancreatic cancers. QN-302 was also supported by the UCL Technology Fund, and in the earlier years, in part by Cancer Research UK funding.