On March 10, 2022 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported a business update and reported fourth quarter and full year 2021 financial results (Press release, Oncternal Therapeutics, MAR 10, 2022, View Source [SID1234609879]).

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"This past year was a decisive one for Oncternal, as we reached consensus with the FDA on our Phase 3 clinical trial ZILO-301 of zilovertamab in patients with MCL, advanced our ROR1-targeting CAR-T cell therapy candidate ONCT-808 towards IND submission, and initiated IND-enabling studies for ONCT-534, our DAARI product candidate that may address key resistance mechanisms in metastatic prostate cancer," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We are focusing our resources on hematological malignancies and prostate cancer, areas of high unmet patient need where we believe our potentially first-in-class or best-in-class product candidates can make the greatest difference. We believe our strong balance sheet will enable us to advance these programs into mid-2023, as we navigate a historically challenging macro environment."

Recent Highlights

In January 2022, we announced that we reached consensus with the FDA on the design and major details of the Phase 3 Study ZILO-301 to treat patients with relapsed or refractory mantle cell lymphoma (MCL) with zilovertamab, an investigational anti-ROR1 monoclonal antibody, in combination with ibrutinib. The agency also provided positive feedback on the proposed key clinical and regulatory requirements of our development program for zilovertamab in patients with MCL.
In December 2021, we announced an interim clinical data update from the ongoing Phase 1/2 clinical trial of zilovertamab in combination with ibrutinib for patients with MCL and chronic lymphocytic leukemia (CLL) [NCT03088878] at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2021 Virtual Annual Meeting.
Objective response rate (ORR) of 81% (21 of 26 evaluable patients) observed for patients with MCL treated with zilovertamab plus ibrutinib, which compares favorably to the historical ORR of 66% for ibrutinib monotherapy
Complete response (CR) rate of 35% for MCL patients treated with zilovertamab plus ibrutinib (9 of 26 evaluable patients), which compares favorably to the historical ORR of 20% for ibrutinib monotherapy, and with CRs remaining durable for up to 32 months
Median progression-free survival (PFS) of 35.9 months for MCL patients with median follow-up of 14.4 months, which compares favorably to the historical ibrutinib monotherapy median PFS of 12.8 months
Landmark PFS of 100% at 36 months for CLL patients who had previously received one or two prior lines of therapy, which compares favorably to historical ibrutinib monotherapy PFS of ~ 75%
Median PFS had not been reached for CLL patients with one or two prior lines of therapy, and median PFS was 36.1 months for patients receiving > 2 prior lines of therapy, with a median follow-up of 29.0 months
The combination of zilovertamab and ibrutinib continued to be well tolerated, with a safety profile consistent with or improved compared with historical data for ibrutinib monotherapy
In November 2021, we announced joining the Karolinska Institutet’s NextGenNK Competence Center to support our next generation ROR1-targeted cell therapy initiatives, and the establishment of a Cell Therapy Scientific Advisory Board, comprised of industry and academic leaders in the cell therapy field.
In October 2021, we presented encouraging preclinical data with ONCT-534, the lead candidate in our preclinical dual-action androgen receptor inhibitor (DAARI) program, during a virtual poster presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC Virtual International Conference on Molecular Targets showing anti-tumor activity in preclinical studies relevant to important tumor resistance mechanisms, including those involving expression of the androgen receptor splice variant (AR-V7).
In November 2021, we announced an interim clinical data update from the ongoing Phase 1/2 clinical trial evaluating ONCT-216, an investigational, potentially first-in-class, targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins, in patients with relapsed or refractory Ewing sarcoma [CT02657005] at the Connective Tissue Oncology Society 2021 Virtual Annual Meeting. Two patients continue to demonstrate durable complete responses, including one patient with a durable CR for 24 months on treatment, and no evidence of disease off treatment after several months.
Expected Upcoming Milestones

Zilovertamab (ROR1 antibody) program
Initiation of global registrational Phase 3 Study ZILO-301 in the second quarter of 2022
Interim clinical data update for patients with MCL and CLL treated with zilovertamab plus ibrutinib in ongoing Phase 1/2 clinical study in the second quarter of 2022
Have a Phase 1b investigator sponsored trial of zilovertamab plus docetaxel initiated for patients with metastatic castration-resistant prostate cancer (mCRPC) in mid-2022
ONCT-808, lead candidate in autologous ROR1-targeted CAR-T cell therapy program
Investigational New Drug (IND) application submission in mid-2022
ONCT-534, lead candidate in our DAARI program
IND-enabling GLP toxicology studies and GMP manufacturing initiated in the second quarter of 2022
ONCT-216 (ETS inhibitor) program
Updated interim clinical data for patients with Ewing sarcoma treated in the dose intensified expansion cohort in the fourth quarter of 2022
Fourth Quarter and Full Year 2021 Financial Results
Our grant revenue was $0.6 million for the fourth quarter ended December 31, 2021. Our grant revenue is derived from a subaward under a grant from the California Institute for Regenerative Medicine (CIRM) to the University of California, San Diego and two research and development grant awards from the National Institutes of Health (NIH). For the full year 2021, grant revenue was $4.3 million.

Our total operating expenses for the fourth quarter ended December 31, 2021 were $8.6 million, including $1.7 million in non-cash stock-based compensation expense. Research and development expenses for the quarter totaled $6.0 million, and general and administrative expenses for the quarter totaled $2.6 million. Net loss for the fourth quarter was $8.1 million, or a loss of $0.16 per share, basic and diluted. For the full year 2021, total operating expenses were $35.7 million, including $5.9 million in non-cash stock-based compensation expense, and our net loss was $31.3 million, or a loss of $0.64 per share, basic and diluted.

As of December 31, 2021, we had approximately 49.4 million shares of common stock outstanding. $90.8 million in cash and cash equivalents and no debt. We believe these funds will be sufficient to fund our operations into mid-2023. Our cash guidance is subject to a number of assumptions, including those related to the severity and duration of the COVID-19 pandemic, and the pace of our research and clinical development programs, among other aspects of our business and the geopolitical environment.

On March 10, 2022 xCures reported their collaboration with Tyra Biosciences for a real-world biomarker study involving patients with FGFR-mutant bladder cancer receiving Balversa (erdafitinib) as their standard of care medical therapy (Press release, xCures, MAR 10, 2022, View Source [SID1234609878]). The study is supported by Tyra Biosciences, Inc., a precision oncology company focused on developing purpose-built therapies to overcome tumor resistance and improve outcomes for patients with cancer. This study will investigate a patient’s tumor DNA through their blood and urine samples to provide information on their treatment and what options might be available if their disease worsens.

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Bladder cancer is the sixth most common cancer in the United States and is associated with genetic mutations in the patient’s bladder or urothelium (the lining of the lower urinary tract). Approximately one in five patients with recurrent and refractory bladder cancer has alterations in fibroblast growth factor (FGFR) genes. These are typically detected with a tumor biopsy, a procedure that may be invasive and anxiety provoking.

US FDA has recently approved Balversa to treat adult patients with locally advanced or metastatic urothelial carcinoma with certain FGFR2 or FGFR3 gene mutations. Overall, about one third of patients treated with Balversa have a confirmed tumor response. However, acquired resistance can be a consequence of treatment and is a devastating reality for patients initially responding to targeted cancer therapies. As new treatments have become more selective, there is a strong demand to address this significant limitation.

In this study, samples collected from bladder cancer patients receiving standard of care Balversa will be tested using a non-invasive diagnostic method that uses the blood and urine samples, instead of tissue, to detect gene alterations that may occur at the time of tumor progression. These less intrusive samples may allow for the assessment of how treatment with Balversa affects clinical response, disease progression, and/or genetic alterations detected in cell free tumor DNA.

"xCures is excited to partner with Tyra Biosciences to deliver on the promise of precision oncology and translational medicine for bladder cancer," said Mika Newton, CEO of xCures. "This study is a great example of how patients and their doctors have the opportunity to both better understand their options and contribute to research in real time."

"We’re excited to support xCures on this biomarker study, which utilizes a non-invasive approach to identify FGFR mutations in patients with bladder cancer," said Todd Harris, CEO of Tyra Biosciences. "This study could yield valuable insights into FGFR-mutant biology and overall tumor progression in bladder cancer, and we hope to leverage these findings as we advance our TYRA-300 program toward the clinic."

The study is being conducted under a decentralized model, leveraging xCures’ direct-to-patient precision oncology platform to reach patients anywhere in the United States. Patients interested in participating in the study can get more information at:

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On March 10, 2022 Amgen (NASDAQ:AMGN) reported that it will virtually present at the 32nd Annual Oppenheimer Healthcare Conference at 11:20 a.m. ET on Wednesday, March 16, 2022 (Press release, Amgen, MAR 10, 2022, View Source [SID1234609877]). Rob Lenz, M.D., Ph.D., senior vice president of Global Development and Peter H. Griffith, executive vice president and chief financial officer at Amgen will present at the conference . The webcast will be broadcast over the internet simultaneously and will be available to members of the news media, investors and the general public.

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The webcast, as with other selected presentations regarding developments in Amgen’s business given at certain investor and medical conferences, can be accessed on Amgen’s website, www.amgen.com, under Investors. Information regarding presentation times, webcast availability and webcast links are noted on Amgen’s Investor Relations Events Calendar. The webcast will be archived and available for replay for at least 90 days after the event.

On March 10, 2022 Phosplatin Therapeutics Inc., a clinical stage pharmaceutical company focused on oncology therapeutics, reported an in-person poster presentation will be given at the American Academy of Cancer Research (AACR) (Free AACR Whitepaper) on its lead candidate, PT-112, and how it selectively induces potent mitochondrial stress and immunogenic cell death in human prostate cancer cell lines (Press release, Phosplatin, MAR 10, 2022, View Source [SID1234609876]). The presentation will be held at the 2022 AACR (Free AACR Whitepaper) Annual Meeting taking place on April 8-13, 2022 in New Orleans.

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The work to be presented was generated by the company under a research collaboration with the Anel Lab at the University of Zaragoza, Spain. The characterization of intracellular pathways leading to ICD and the adaptive immune response is an important research aim of the company, and a related peer-reviewed manuscript is under preparation.

Presentation Title: "PT-112 induces potent mitochondrial stress and immunogenic cell death in human prostate cancer cell lines"
Lead Author: R. Soler, University of Zaragoza/Aragón Health Research Institute, Biochemistry and Molecular and Cell Biology, Zaragoza, Spain
Session Category: Experimental and Molecular Therapeutics
Session: Mechanisms of Drug Action 3
Session Date / Time: Monday, April 11, 2022 from 9:00am – 12:30pm CST
Location: New Orleans Convention Center, Exhibit Halls D-H, Poster Section 24
Poster Number: 5

The presentation will be available both in person and online in Proceedings of the AACR (Free AACR Whitepaper) through the conference website.

For more information about Phosplatin’s clinical trials, visit the website at www.Phosplatin.com.

About PT-112

PT-112 is the first small-molecule conjugate of pyrophosphate in oncology, and possesses a unique pleiotropic mechanism of action that promotes immunogenic cell death (ICD), through the release of damage associated molecular patterns (DAMPs) that bind to dendritic cells and lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 may represent the best-in-class inducer of this immunological form of cancer cell death. Further, PT-112 harbors a property known as osteotropism, or the propensity of the drug to reach its highest concentrations in certain areas of the bone, making it a candidate for treatment of patients with cancers that originate in, or metastasize to, the bone. The first in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses among heavily pre-treated patients and won "Best Poster" within the Developmental Therapeutics category at the ESMO (Free ESMO Whitepaper) 2018 Annual Congress. The combination Phase 1b dose escalation study of PT-112 with PD-L1 checkpoint inhibitor avelumab in solid tumors was reported in an oral presentation at the ESMO (Free ESMO Whitepaper) 2020 Virtual Congress. The Phase 1 study in patients with relapsed or refractory multiple myeloma presented at ASH (Free ASH Whitepaper) is the third completed Phase 1 study of PT-112. Monotherapy Phase 2 development is ongoing in mCRPC, and the PD-L1 combination study is ongoing in a dose confirmation cohort of non-small cell lung cancer (NSCLC) patients, and will soon include the Phase 2 proof of concept study in thymic epithelial tumors under the company’s collaboration with the NCI.

On March 10, 2022 Manhattan BioSolutions, an emerging biotechnology company focused on the development of innovative biologic therapies for the treatment of advanced and metastatic cancers, reported that it has been awarded grant funding as part of Round 3 of the New Jersey Commission on Science, Innovation, and Technology Direct Financial Assistance component (Phase I&II) of the Small Business Innovation Research (SBIR) and Small Business Technology Transfer Program (STTR) (Press release, Manhattan BioSolutions, MAR 10, 2022, View Source [SID1234609874]).

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This award will enable early-stage preclinical validation of therapeutic leads previously supported by the SBIR and STTR Phase I grants. Novel therapeutic platforms developed by Manhattan BioSolutions leverage pathogen-specific immune responses to fight disease, particularly cancers. Dr. Borys Shor, CEO of Manhattan BioSolutions, commented: "This award expands our capacity to discover novel therapies at the intersection of innate immunity, age-related diseases, and cancer. We expect to have a deep pipeline of discovery stage products by year-end and bring those into human trials in 2023."