On December 13, 2022 ESSA Pharma Inc. ("ESSA", or the "Company") (NASDAQ: EPIX), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, reported a corporate update and reported financial results for the fiscal year ended September 30, 2022 (Press release, ESSA, DEC 13, 2022, View Source [SID1234625234]). All references to "$" in this release refer to United States dollars, unless otherwise indicated.

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"ESSA achieved major milestones in 2022 with the release of Phase 1 monotherapy and combination data for our lead candidate EPI-7386 from three clinical trials in patients with metastatic castration-resistant prostate cancer ("mCRPC"), which demonstrated initial anti-tumor activity in certain patients, and EPI-7386’s favorable safety profile as a single agent and in combination with second-generation antiandrogens," stated David Parkinson, MD, President and CEO of ESSA. "Early data from these clinical studies demonstrated notable PSA reductions in the Phase 1 study of EPI-7386 and Astellas’ Xtandi (enzalutamide) and in the Phase 1 study of EPI-7386 and Janssen’s antiandrogens Erleada (apalutamide) and Zytiga (abiraterone acetate). In addition, the Phase 1a dose escalation monotherapy results showed tumor volume decreases and PSA declines in a subset of heavily pretreated mCRPC patients who had progressed on standard-of-care therapies."

Dr. Parkinson continued: "Looking ahead to 2023, we expect a busy year as we advance clinical studies of EPI-7386 as a monotherapy and in combination with approved antiandrogens in a number of prostate cancer patient populations. Our cash runway is strong and expected to fund our operations and clinical programs through 2025, including the Phase 1b monotherapy expansion and Window of Opportunity studies, a Phase 2 combination study with enzalutamide, additional cohorts in a Phase 1 study evaluating EPI-7386 with Janssen’s antiandrogens, and an investigator-sponsored study of EPI-7386 and darolutamide."

Clinical and Corporate Highlights for 2022 Fiscal Year

EPI-7386 Combination Studies

Updated clinical data from the first two cohorts of the Phase 1/2 study of EPI-7386 in combination with enzalutamide were presented at the 2022 Prostate Cancer Foundation ("PCF") Scientific Retreat. The data showed preliminary evidence of anti-tumor activity, with five of six patients achieving PSA90 and four of six patients achieving PSA90 within 90 days.
In October 2022, the Company announced that Janssen Research and Development is suspending enrollment into the Phase 1 clinical study of EPI-7386 with apalutamide or EPI-7386 with abiraterone acetate plus prednisone in mCRPC patients as a result of operational recruitment challenges. Before suspending enrollment, Janssen treated three mCRPC patients (chemotherapy naive) for up to four months of therapy, with two of the three patients achieving PSA90 within 90 days. ESSA anticipates enrolling additional cohorts in the Phase 1 study in 2023 to further assess the safety and tolerability of abiraterone acetate plus prednisone or apalutamide (administered at the dose recommended in their prescribing information) when administered in combination with EPI-7386 and to establish recommended Phase 2 dosing for these combinations.
Preclinical data for the Company’s lead first generation androgen receptor ANITen bAsed Chimera ("ANITAC") N-terminal domain degrader were presented in a poster session at the 34th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Annual Symposium on Molecular Targets and Cancer Therapeutics.
EPI-7386 Monotherapy

In June 2022, the Company presented a clinical update on EPI-7386 monotherapy and combination therapy clinical development. Initial data from 33 heavily pretreated mCRPC patients enrolled in the Phase 1a dose escalation monotherapy study demonstrated that EPI-7386 was well-tolerated and reached clinically relevant exposures at all dose levels tested. Clinically important signals of anti-tumor activity were observed in a subset of these patients (less than 3 lines of treatment for mCRPC, lack of visceral disease, no prior chemotherapy and lack or few non-AR mutations).
Corporate Updates

In September 2022, the Company announced the appointment of Philip Kantoff, M.D., to its Board of Directors. Dr. Kantoff is a renowned medical oncologist and leader in the clinical development of new prostate cancer treatments.
Summary Financial Results

Net Loss. ESSA recorded a net loss of $35.1 million for the year ended September 30, 2022, compared to a net loss of $36.8 million for the year ended September 30, 2021. For the year ended September 30, 2022, this included non-cash share-based payments of $7.9 million compared to $9.5 million for the prior year, recognized for stock options granted and vesting. The net loss for the fourth quarter ended September 30, 2022 was $6.3 million compared to a net loss of $8.5 million for the fourth quarter ended September 30, 2021. The decrease in the fourth quarter was primarily attributed to a decrease in research and development expenditures.
Research and Development ("R&D") expenditures. R&D expenditures for the year ended September 30, 2022 were $24.4 million compared to $24.3 million for the year ended September 30, 2021 and include non-cash costs related to share-based payments ($4.3 million for the year ended 2022 compared to $3.6 million for the year ended 2021). The R&D expenditures for the year ended September 30, 2022 largely remained consistent when compared to the year ended September 30, 2021, as the increased expense in preclinical and data analysis was offset by the decreased expense in manufacturing costs related to the Phase 1 clinical trial of EPI-7386. For the fourth quarter ended September 30, 2022, R&D expenditures were $4.4 million (net and gross), compared to $6.3 million (net and gross) for the fourth quarter ended September 30, 2021. The decrease in the fourth quarter was primarily attributed to a decrease in non-cash share-based payments, manufacturing costs and travel costs.
General and administration ("G&A") expenditures. G&A expenditures for the year ended September 30, 2022 were $12.5 million compared to $12.9 million for the year ended September 30, 2021 and include non-cash costs related to share-based payments of $3.6 million for the year ended 2022 compared to $5.8 million for the year ended 2021. For the fourth quarter ended September 30, 2022, G&A expenditures were $2.8 million, compared to $2.9 million for the fourth quarter ended September 30, 2021. The decrease in the full year and fourth quarter is the result of decreased professional fees as well as the decrease in non-cash share-based payments.
Liquidity and Outstanding Share Capital

At September 30, 2022, the Company had available cash reserves and short-term investments of $167.2 million reflecting the gross proceeds of the February 2021 financing of approximately $150.0 million and July 2020 financing of $48.9 million, less operating expenses in the intervening period. The Company’s cash position is expected to be sufficient to fund current and planned operations through 2025.

As of September 30, 2022, the Company had 44,073,076 common shares issued and outstanding.

In addition, as of September 30, 2022 there were 3,234,750 common shares issuable upon the exercise of warrants and broker warrants. This includes 2,920,000 prefunded warrants at an exercise price of $0.0001, and 314,750 warrants at a weighted average exercise price of $49.69. There were 7,902,061 common shares issuable upon the exercise of outstanding stock options at a weighted-average exercise price of $5.13 per common share.

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 castration-resistant prostate cancer ("CRPC") whose tumors have progressed on standard-of-care therapies and a Window of Opportunity study in patients with non-metastatic CRPC. 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 is also conducting a Phase 1/2 clinical trial (NCT05075577) of EPI-7386 in combination with enzalutamide in metastatic CRPC patients who have not yet been treated with second-generation antiandrogen therapies. ESSA retains all rights to EPI-7386 worldwide.

On December 13, 2022 Treadwell Therapeutics, a clinical-stage biotechnology company developing novel, small molecule therapeutics for highly aggressive cancers, reported a presentation for the Company’s CFI-400945 program, a first in class inhibitor of Polo-like Kinase 4 (PLK4) a critical regulator of centriole duplication, at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held from December 10-13, 2022 (Press release, Treadwell Therapeutics, DEC 13, 2022, View Source [SID1234625233]). The poster presentation described the preliminary results from the monotherapy dose optimization portion of the TWT-202 in advanced leukemias.

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"CFI-400945 had previously shown single agent complete remissions in refractory high risk AML. As we optimize dose for the agent in unselected leukemia patients in study TWT-202, we are encouraged by the continued signs of safety and tolerability with this oral dosing regimen," said Principal Investigator, Dr. Gautam Borthukar, MD, Professor, Department of Leukemia, Division of Cancer Medicine, MD Anderson Cancer Center.

"We look forward to dose selection for CFI-400945, and expansion into populations of interest, including patients with TP53 mutations, where there is substantial unmet need," said Dr. Michael Tusche, Co-CEO at Treadwell Therapeutics.

2022 ASH (Free ASH Whitepaper) Poster Presentations and Details:

Preliminary Results from a Phase 2 Open-Label, Multicenter, Dose Optimization Clinical Study of the Safety, Tolerability, and Pharmacokinetic (PK) and Pharmacodynamic (PD) Profiles of CFI-400945 As a Single Agent or in Combination with Azacitidine or Decitabine in Patients with Acute Myeloid Leukemia, Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia (TWT-202)

Publication Number: 4087
Session: 616; Poster III
Date and Time: December 12th, 2022, 6:00 PM-8:00 PM

Data presented on CFI-400945, an oral, first-in-class PLK4 inhbitor, showed a tolerable safety profile at the 32, 48 and 64 mg cohorts (N=12), with exposures being approximately dose linear. No dose limiting toxicities have been observed to date, suggesting further dose optimization is required. Five cases of stable disease have been observed – 3 per ELN with 1 at 48 mg, and 2 at 64 mg, as well as 2 at 48 mg per IWG. Adverse events (AEs) for CFI-400945 in this study were in line with those observed in previous studies in similar patient populations. Main AEs (any grade) were hematologic, gastrointestinal and metabolism/nutritional disorders. Most predominant severe AE was febrile neutropenia. No treatment emergent adverse events led to study drug discontinuation.

On December 13, 2022 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported the publication of a manuscript in Cancer Discovery, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), which describes the design and characterization of NVL-520 and details Nuvalent’s approach to rationally targeting ROS1 (Press release, Nuvalent, DEC 13, 2022, View Source [SID1234625232]). NVL-520 is currently being studied in the ongoing ARROS-1 Phase 1/2 clinical trial for patients with advanced ROS1-positive non-small cell lung cancer (NSCLC) and other solid tumors.

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The paper, entitled "NVL-520 is a selective, TRK-sparing, and brain-penetrant inhibitor of ROS1 fusions and secondary resistance mutations," is published online and can be accessed here: View Source

"The ROS1 kinase is a clinically validated target for the treatment of NSCLC, and ROS1 tyrosine kinase inhibitors (TKIs) are established as an important treatment option for ROS1-driven lung cancers. However, limitations do exist with available ROS1 TKIs, including treatment-emergent drug resistance, off-target neurological adverse events, and inadequate control or prevention of brain metastases," said senior author Jessica J. Lin, M.D., Thoracic Oncologist at Mass General Cancer Center, Assistant Professor of Medicine at Harvard Medical School, and investigator in the ARROS-1 trial. "As described in this paper and earlier this year at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium, preclinical characterization and preliminary clinical data support the opportunity for NVL-520 to overcome these limitations as a potential best-in-class ROS1 kinase inhibitor and provide compelling rationale for its ongoing evaluation in patients with TKI-experienced ROS1 fusion-positive cancers and planned evaluation in treatment-naïve patients."

The paper details the design principles underlying the activity of NVL-520 against ROS1 and its most commonly occurring resistance mutation, ROS1 G2032R, and a molecular rationale for the selectivity of NVL-520 for ROS1 over the structurally-related TRK family. TRK inhibition in the CNS by approved or investigational ROS1 TKIs has been associated with neurological adverse events that can be dose limiting. Extensive preclinical characterization of the activity and selectivity of NVL-520 is presented, spanning biochemical and cellular assays, in vivo xenograft studies, and preclinical assessments of brain penetrance and intracranial activity.

In addition, the paper includes three case studies of patients with ROS1 fusion-positive lung cancers that had relapsed on or were refractory to a range of ROS1 TKIs, and includes patients with tumors that harbored ROS1 G2032R or had intracranial metastases. NVL-520 elicited tumor responses in the patients with no observed neurological toxicities. These findings support the potential for NVL-520 to treat these patient populations while also enhancing tolerability through improved selectivity for ROS1.

"At Nuvalent, we aim to solve for multiple, and at times competing, challenges in structure-based drug design with the goal to advance novel therapeutics with the potential for best-in-class activity against recalcitrant targets. This publication in Cancer Discovery provides insight into our focused approach to the discovery of NVL-520 and additional support for the potential achievement of our design goals through comprehensive biochemical and cellular profiling, evaluation across diverse ROS1-fusion driven preclinical models, and patient case studies, said Joshua Horan, Ph.D., Vice President, Chemistry at Nuvalent. "We are grateful to our collaborators for their contributions to this paper and to the continued advancement of NVL-520."

Initial data from the Phase 1 dose-escalation portion of the trial were presented during the "New Drugs on the Horizon" plenary session at the 2022 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium. The ARROS-1 trial is continuing to enroll patients in the Phase 1 portion of the study and is focused on further characterizing the safety profile of NVL-520, its pharmacokinetic profile, and determining the recommended Phase 2 dose.

About NVL-520

NVL-520 is a novel brain-penetrant ROS1-selective inhibitor designed to remain active in tumors that have developed resistance to currently available ROS1 inhibitors, including tumors with the prevalent G2032R resistance mutation and those with the S1986Y/F, L2026M, or D2033N resistance mutations. NVL-520 has been optimized for brain penetrance to potentially improve treatment options for patients with brain metastases. NVL-520 has been observed in preclinical studies to selectively inhibit wild-type ROS1 and its resistance variants over the structurally related tropomyosin receptor kinase (TRK) family to potentially avoid TRK-related CNS adverse events seen with dual TRK/ROS1 inhibitors and drive more durable responses for patients. NVL-520 is currently being investigated in the ARROS-1 study (NCT05118789), a first-in-human Phase 1/2 clinical trial for patients with advanced non-small cell lung cancer (NSCLC) and other solid tumors.

On December 13, 2022 Doctors and scientists with City of Hope, one of the largest cancer research and treatment organizations in the United States, reported data on potential new treatment options for hematological cancers and a way to predict disease outcomes in patients with diffuse large B cell lymphoma, as well as novel therapies targeting complications like post-transplant infections at this year’s ASH (Free ASH Whitepaper) conference in New Orleans, Dec. 10 to 13 (Press release, City of Hope, DEC 13, 2022, View Source [SID1234625231]).

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"The robust data presented at the ASH (Free ASH Whitepaper) conference this year demonstrates the innovative science and research happening across a broad range of blood cancers, and the tremendous promise and hope this can mean for patients now and in the future," said Eileen Smith, M.D., City of Hope’s Francis & Kathleen McNamara Distinguished Chair in Hematology and Hematopoietic Cell Transplantation. "From promising investigational immunotherapies and stem cell transplantation to potentially practice changing prognostic tests, City of Hope, and our colleagues at Cancer Treatment Centers of America and Translational Genomics Research Institute, continue to accelerate the development and delivery of transformative treatments, with the ultimate goal of improving the outcomes for people with blood cancers."

Robust data presented at the ASH (Free ASH Whitepaper) conference this year demonstrates City of Hope’s innovative science

Highlights of City of Hope research presented at the ASH (Free ASH Whitepaper) conference include:

Posoleucel Helps Prevent Post-Transplant Viral Infections in High-Risk Allo-HCT Recipients in Phase 2 Trial
For people receiving infusions of stem cells from a donor — known as an allogeneic hematopoietic cell transplantation (allo-HCT) — to help treat blood cancers, viral infections are a constant threat due to limited immune function. Keeping viruses like Epstein-Barr, cytomegalovirus, human herpesvirus 6, adenovirus and polyoma viruses at bay is a challenge.

New data in a study led by Sanjeet Dadwal, M.D., City of Hope professor and chief of the Division of Infectious Diseases in the Department of Medicine, who specializes in treating infections in cancer patients, shows promising results for posoleucel, off-the-shelf multivirus-specific T cells, in preventing clinically significant viral infections after high risk allo-HCT.

Twenty-six participants received infusions of posoleucel after high-risk allo-HCT. Results showed a low incidence of clinically significant viral infections with six targeted viruses: adenovirus, BK virus, cytomegalovirus, Epstein-Barr virus, human herpesvirus 6 and JC virus.

According to Dadwal, the therapy was well-tolerated, even after multiple infusions. And benefits from posoleucel persisted even in the face of expected low-level viral reactivations throughout the study.

Pending results from an ongoing placebo-controlled Phase 3 study of posoleucel, the therapy could be approved in future for clinical use in preventing significant infections from these six DNA viruses among allo-HCT recipients.

"Based on our findings, in addition to preventing infections, other potential applications could include treatment of active viral infections using off-the-shelf, virus-specific T cells in patients who are unable to mount an adequate immune response, particularly allo-HCT patients," Dadwal said.

New Way to Profile Risk for Patients With Previously Untreated Diffuse Large B Cell Lymphoma
For people with diffuse large B cell lymphoma (DLBCL), induction chemotherapy — a short and intense course of drugs to try and clear the blood of cancer cells — is the first line of defense against the disease. Now, results from a clinical trial suggest that testing levels of circulating tumor DNA (ctDNA) in previously untreated DLBCL patients after one to two cycles of induction therapy could help predict disease outcomes.

Alex Herrera, M.D., associate professor in City of Hope’s Department of Hematology & Hematopoietic Cell Transplantation, reported on an analysis of data from the POLARIX study, a Phase 3 clinical trial in patients with DLBCL comparing two lines of chemotherapy: polatuzumab vedotin with rituximab-cyclophosphamide, doxorubicin and prednisone (Pola-R-CHP) versus rituximab-cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP). Herrera and a team of researchers investigated ctDNA levels at baseline and on day one of participants’ second cycle of chemotherapy.

"What we saw in this study is that patients who had lower levels of circulating tumor DNA in their blood after one treatment cycle had better rates of progression-free survival and longer overall survival than patients who did not see a notable reduction of circulating tumor DNA after one cycle of chemotherapy," Herrera said.

Furthermore, the researchers saw that ctDNA clearance was predictive of better outcomes independent of other key baseline risk factors.

"Our findings show that early changes in circulating tumor DNA levels may be a helpful tool to design clinical trials that improve on our standard treatment by adapting therapy based on ctDNA results," Herrera said. "We could imagine developing a study where we add a novel therapy in patients with an insufficient ctDNA response, or we de-escalate therapy in patients with an excellent early ctDNA response."

New Cell Therapy Found to Reduce GVHD for Haploidentical Allo-HCT Recipients
Allogeneic hematopoietic cell transplantation (allo-HCT) can be curative for many blood disorders, including cancer, but the process works best when the patient and donor have the same human leukocyte antigens type. While progress has been made in making half-matched, or haploidentical, transplants more successful using post-transplant cyclophosphamide, a potent chemotherapy drug, high rates of relapse and chronic graft-versus-host disease (GVHD) following myeloablative conditioning, which destroys a patient’s bone marrow cells before they are replaced, pose significant risks for recipients.

Amandeep Salhotra, M.D., associate professor in the Department of Hematology & Hematopoietic Cell Transplantation, reported on a potential new alternative to cyclophosphamide called Orca-Q, which is an investigational precision engineered cell therapy.

Orca-Q is made up of enriched stem cells combined with specific T cell subsets to help build up the blood and immune systems.

In a Phase 1 study of 21 patients with haploidentical donors and eligible for myeloablative regimens, a research team led by Salhotra found Orca-Q to be beneficial. It resulted in low toxicity and superior control of acute and chronic GVHD and, therefore, better long-term survival.

"This type of graft is better tolerated by patients as it avoids the use of high-dose chemotherapy after the infusion of cells, which is currently the only way to do haploidentical transplants," Salhotra said. "As such, older patients and those with co-morbid conditions will likely be able to benefit from myeloablative conditioning using Orca-Q graft therapy."

This Phase 1 study is ongoing, and Salhotra hopes to be able to extend the benefits of Orca-Q to patients undergoing reduced-intensity conditioning regimens.

Novel Salvage Therapy Is Successful in High-Risk Refractory and Relapsed Hodgkin Lymphoma Patients
For high-risk people with classic Hodgkin lymphoma whose disease has relapsed or is resistant to treatment, a new line of therapy has demonstrated promising results in clinical trials.

Matthew Mei, M.D., City of Hope associate professor in the Department of Hematology & Hematopoietic Cell Transplantation, presented findings from a study that investigated the anti-tumor effects of nivolumab, an anti-PD1 monoclonal antibody, in combination with ifosfamide, carboplatin and etoposidechemotherapy in 32 high-risk patients with relapsed/refractory Hodgkin lymphoma.

Mei and his team found the combination is well-tolerated and a highly effective, second-line treatment, which facilitated subsequent autologous stem cell transplant. All patients had a response to the treatment and 88% achieved a complete remission with toxicity comparable to that of chemotherapy alone.

"The combination of checkpoint inhibition with cytotoxic chemotherapy has now been validated in multiple studies with much higher complete remission rates than historical controls," Mei said. "The next step will be to compare this approach to chemotherapy salvage alone, and a large cooperative group study is planned to address this question."

Clinical Trial for New Drug Combination to Treat Multiple Myeloma Announced
Multiple myeloma remains incurable for most patients, but the development of new treatment regimens is helping to improve outcomes.

Amrita Krishnan, M.D., director of the Judy and Bernard Briskin Center for Multiple Myeloma Research at City of Hope, discussed a new clinical trial that is expected to open at City of Hope next year. The goal is to further test combination frontline therapies for multiple myeloma that do not include autologous stem cell transplants.

"The trial uses the newest Food and Drug Administration-approved drug for multiple myeloma called teclistamab, which is currently approved for patients with relapsed disease," Krishnan said. "We are trying to find out if using it earlier in the course of multiple myeloma will lead to deeper and longer remissions than the current approaches."

Called MajesTEC-7, the trial is a Phase 3, randomized study comparing the efficacy of teclistamab plus daratumumab plus lenalidomide (Tec-DR) versus daratumumab + lenalidomide + dexamethasone (DRd) in newly diagnosed multiple myeloma patients who cannot receive or do not wish to undergo an autologous stem cell transplant as an initial treatment.

Krishnan and her team hope to enroll 1,000 participants with newly diagnosed and measurable disease, ages 18 or above, who will be randomized to receive either Tec-DR or DRd.

Targeting Energy Supplies in Acute Myeloid Leukemia
Because acute myeloid leukemia (AML) is a blood cancer that progresses rapidly, it is currently associated with poor outcomes, particularly in adults. But City of Hope researchers believe they may have found a new way to stop AML in its tracks.

Flavia Pichiorri, Ph.D., M.S., professor in the Department of Hematologic Malignancies Translational Science and the Judy and Bernard Briskin Center for Multiple Myeloma Research, presented novel findings associated with a molecule called CD84 that her team found expressed at high levels on the surface of AML cells.

This study was led by Yinghui Zhu, Ph.D., a former postdoctoral researcher in Pichorri’s laboratory and currently an assistant professor at Tongji University in Shanghai, China.

To test whether this increased expression promotes AML growth, the researchers ran experiments to disrupt CD84. They found that these disruptions inhibited AML cell growth and deregulated mitochondria — which play a large role in metabolism — in these cancer cells.

"These findings open the door for potential novel treatments for AML that target CD84, including the development of immune engagers or directing potent toxins to selectively kill AML cells," said Pichiorri, the study’s senior investigator.

Based on their discoveries, Pichiorri and her team, in collaboration with the laboratory of John Williams, Ph.D., City of Hope professor in the Department of Cancer Biology and Molecular Medicine, tested antibodies that target CD84 and showed that their administration in vivo prevents AML progression and increases survival in preclinical mouse models.

"We will now work on translating these laboratory experiments into CD84-targeting agents with the intention of bringing therapies to patients in short order," Pichiorri said.

Additional City of Hope Participation at ASH (Free ASH Whitepaper)
Michael Caligiuri, M.D., president of City of Hope National Medical Center and Deana and Steve Campbell Physician-in-Chief Distinguished Chair, received an ASH (Free ASH Whitepaper) Mentor Award, which recognizes outstanding mentors in the hematology community. Caligiuri has dedicated much of his career to mentoring the next generation of physicians, scientists and physician-scientists: Over the last three decades, he has mentored more than 100 individuals and has had a profound impact on their careers.

Monzr Al Malki, M.D., an associate professor in City of Hope’s Department of Hematology & Hematopoietic Cell Transplantation and director of the Unrelated Donor BMT Program and Haploidentical Transplant Program, was one of the authors of "LBA-4 Post-Transplant Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil As the New Standard for Graft-Versus-Host Disease (GVHD) Prophylaxis in Reduced Intensity Conditioning: Results from Phase III BMT CTN 1703," which featured the results of a Phase 3 clinical trial comparing outcomes of allogeneic hematopoietic cell transplantation in patients randomized to receive a novel therapy against graft-versus-host disease versus a standard treatment. Al Malki was also the chair of an ASH (Free ASH Whitepaper) education session titled "Are Alternative Donors Now Mainstream in Allogeneic Transplant?"

Andrew Artz, M.D., M.S., professor in City of Hope’s Department of Hematology & Hematopoietic Cell Transplantation, moderated an ASH (Free ASH Whitepaper) symposium on hematopoietic stem cell mechanisms of aging evaluating cellular changes that impact how a cancer develops.

Joseph Mikhael, M.D., a professor in the Applied Cancer Research and Drug Discovery Division at the Translational Genomics Research Institute, an affiliate of City of Hope, is moderating an ASH (Free ASH Whitepaper) symposium titled "Decentralization of Clinical Trials – Moving Forward" and also presented research on identifying and overcoming racial disparities in multiple myeloma care.

Tycel Phillips, M.D., an associate clinical professor in City of Hope’s Division of Lymphoma in the Department of Hematology & Hematopoietic Cell Transplantation — who is leading clinical trials into new targeted therapies, has published over 40 peer-reviewed studies and attracted dozens of research grants — chaired an education session on new developments and treatment for low-stage lymphoma at ASH (Free ASH Whitepaper).

On December 13, 2022 Envisagenics, an Artificial Intelligence ("AI")-driven biotechnology company that delivers therapies for RNA splicing diseases, reproted a research collaboration agreement with Cancer Research Horizons, Cancer Research UK’s innovation engine, and Queen Mary University of London (Press release, Envisagenics, DEC 13, 2022, View Source [SID1234625230]). Envisagenics will leverage its SpliceCore AI platform and use Queen Mary’s de-identified data to further explore the role of alternative splicing in hematopoietic cancers for research and development.

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"As an AI-driven biotechnology company, data plays a critical role in facilitating the discovery of quality candidates. With the support of Cancer Research Horizons’ team and its principal investigators, we are thrilled to partner with organizations that have comprehensive data packages which are vital for reaching the right patients sooner," said Martin Akerman, Ph.D., Envisagenics’ CTO and Co-founder. "Through our SpliceCore platform, we will continue to enhance our data-driven R&D strategy and acquire insights with Cancer Research Horizons’ rich datasets to ultimately deepen our understanding of complex tumor biology and accelerate the development of therapeutics for patients with hematopoietic cancers."

Tony Hickson, Chief Business Officer at Cancer Research Horizons, said: "We are at an exciting crossroads where researchers and industry are realizing the great potential in combining the wealth of healthcare data within our network, with advanced computational approaches to drive innovation towards new therapies and diagnostics for patients. We are delighted to be collaborating with Envisagenics to explore the role of alternative splicing in a subset of particularly poorly served hematopoietic cancers and hope to uncover new avenues to bring improved treatment options to patients faster."

Ana Rio-Machin, Ph.D., lead researcher on the project from Queen Mary’s Barts Cancer Institute said: "We have completed multi-omics profiling that combines genomics, proteomics and drug screening, in samples from more than 50 patients with an aggressive type of blood cancer called acute myeloid leukemia ("AML"). This collaboration will allow us to explore the RNA splicing landscape in our cohort of primary AML samples by applying Envisagenics’ expertise and bioinformatics tools to our multi-omics data. We hope this new analysis and the integration with our previous findings will shed light on the pathogenesis of this disease and provide significant preclinical data to support precision medicine approaches for difficult-to-treat hematopoietic cancers."