On November 2, 2023 Kura Oncology, Inc. (NASDAQ: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, and Mirati Therapeutics, Inc. (NASDAQ: MRTX), a commercial-stage targeted oncology company, reported a clinical collaboration and supply agreement to evaluate the combination of KO-2806, a next-generation farnesyl transferase inhibitor (FTI), and adagrasib, a highly selective KRASG12C inhibitor, in patients with KRASG12C-mutated non-small cell lung cancer (NSCLC) (Press release, Kura Oncology, NOV 2, 2023, View Source [SID1234636815]).

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"Recent findings suggest that combining KO-2806 with adagrasib can drive tumor regressions and enhance both duration and depth of antitumor response in preclinical models of KRASG12C-mutated NSCLC," said Stephen Dale, M.D., Chief Medical Officer of Kura Oncology. "This collaboration highlights the potential to address the urgent need for more durable and effective treatment options for patients with advanced solid tumors, and we look forward to collaborating with Mirati, an established leader in targeted oncology."

"We are pleased to collaborate with Kura Oncology on this clinical study of KO-2806 with adagrasib. Preclinical work demonstrates the ability of adagrasib, in combination with a FTI, to improve patient outcomes," said Alan Sandler, M.D., Chief Medical Officer, Mirati Therapeutics. "This collaboration exemplifies the potential combinability of adagrasib as a key differentiation from other KRASG12C inhibitors."

Under the terms of the agreement, Kura will sponsor the Phase 1 study of KO-2806 and adagrasib in patients with KRASG12C-mutated NSCLC. Mirati will supply Kura with adagrasib for the study.

About KO-2806

KO-2806 is a next-generation inhibitor of farnesyl transferase designed to improve upon potency, pharmacokinetic and physicochemical properties of earlier FTI drug candidates. Earlier this year, Kura received FDA clearance of its Investigational New Drug application for KO-2806. In addition to KRASG12C NSCLC, KO-2806 has demonstrated encouraging preclinical activity in clear cell renal cell carcinoma (ccRCC). The Company recently dosed the first patients in a Phase 1 dose-escalation trial of KO-2806 (FIT-001). Concurrent with dose escalation as a monotherapy, Kura also plans to evaluate KO-2806 in dose-escalation combination cohorts with other targeted therapies in advanced solid tumors, including adagrasib in KRASG12C-mutated NSCLC and a tyrosine kinase inhibitor in ccRCC.

About Adagrasib

Adagrasib is being evaluated as monotherapy and in combination with other anti-cancer therapies in patients with advanced KRASG12C-mutated solid tumors, including NSCLC, colorectal cancer, and pancreatic cancer. For more information, visit Mirati.com/science.

KRAZATI (adagrasib) U.S. Indication

KRAZATI is indicated for the treatment of adult patients with KRASG12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.

This indication is approved under accelerated approval based on objective response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of a clinical benefit in a confirmatory trial(s). For Prescribing Information, visit Mirati.com/KRAZATI_USPI.

KRAZATI (adagrasib) Important Safety Information

WARNINGS AND PRECAUTIONS

Gastrointestinal Adverse Reactions

In the pooled safety population, serious gastrointestinal adverse reactions observed were gastrointestinal obstruction in 1.6%, including 1.4% grade 3 or 4, gastrointestinal bleeding in 0.5% of patients, including 0.5% grade 3, and colitis in 0.3%, including 0.3% grade 3. In addition, nausea, diarrhea, or vomiting occurred in 89% of 366 patients, including 9% grade 3. Nausea, diarrhea, or vomiting led to dosage interruption or dose reduction in 29% of patients and permanent discontinuation of KRAZATI in 0.3%
Monitor and manage patients using supportive care, including antidiarrheals, antiemetics, or fluid replacement, as indicated. Withhold, reduce the dose, or permanently discontinue KRAZATI based on severity
QTc Interval Prolongation

KRAZATI can cause QTc interval prolongation, which can increase the risk for ventricular tachyarrhythmias (eg, torsades de pointes) or sudden death
In the pooled safety population, 6% of 366 patients with at least one post-baseline electrocardiogram (ECG) assessment had an average QTc ≥501 ms, and 11% of patients had an increase from baseline of QTc >60 msec. KRAZATI causes concentration-dependent increases in the QTc interval
Avoid concomitant use of KRAZATI with other products with a known potential to prolong the QTc interval. Avoid use of KRAZATI in patients with congenital long QT syndrome and in patients with concurrent QTc prolongation
Monitor ECGs and electrolytes prior to starting KRAZATI, during concomitant use, and as clinically indicated in patients with congestive heart failure, bradyarrhythmias, electrolyte abnormalities, and in patients who are taking medications that are known to prolong the QT interval. Withhold, reduce the dose, or permanently discontinue KRAZATI, depending on severity
Hepatotoxicity

KRAZATI can cause hepatotoxicity
In the pooled safety population, hepatotoxicity occurred in 37%, and 7% were grade 3 or 4. A total of 32% of patients who received KRAZATI had increased alanine aminotransferase (ALT)/increased aspartate aminotransferase (AST); 5% were grade 3 and 0.5% were grade 4. Increased ALT/AST leading to dose interruption or reduction occurred in 11% of patients. KRAZATI was discontinued due to increased ALT/AST in 0.5% of patients
Monitor liver laboratory tests (AST, ALT, alkaline phosphatase, and total bilirubin) prior to the start of KRAZATI, and monthly for 3 months or as clinically indicated, with more frequent testing in patients who develop transaminase elevations. Reduce the dose, withhold, or permanently discontinue KRAZATI based on severity
Interstitial Lung Disease /Pneumonitis

KRAZATI can cause interstitial lung disease (ILD)/pneumonitis, which can be fatal. In the pooled safety population, ILD/pneumonitis occurred in 4.1% of patients, 1.4% were grade 3 or 4, and 1 case was fatal. The median time to first onset for ILD/pneumonitis was 12 weeks (range: 5 to 31 weeks). KRAZATI was discontinued due to ILD/pneumonitis in 0.8% of patients
Monitor patients for new or worsening respiratory symptoms indicative of ILD/pneumonitis (eg, dyspnea, cough, fever). Withhold KRAZATI in patients with suspected ILD/pneumonitis and permanently discontinue KRAZATI if no other potential causes of ILD/pneumonitis are identified
Adverse Reactions

The most common adverse reactions (≥25%) are nausea, diarrhea, vomiting, fatigue, musculoskeletal pain, hepatotoxicity, renal impairment, edema, dyspnea, decreased appetite
Females and Males of Reproductive Potential

Infertility: Based on findings from animal studies, KRAZATI may impair fertility in females and males of reproductive potential
Please see Full Prescribing Information.

On November 2, 2023 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported third quarter 2023 financial results and provided a corporate update (Press release, Kura Oncology, NOV 2, 2023, View Source [SID1234636814]).

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"I am proud of our team’s considerable progress, as we continue to execute across all three of our wholly owned, clinical-stage programs," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We believe our lead drug candidate, ziftomenib, is well positioned for market leadership, with multibillion dollar global revenue potential in acute leukemias and beyond. Our conviction is supported by a growing body of clinical data as a monotherapy and, increasingly, in combination with standards of care. We continue to be encouraged by the rapid pace of enrollment in our KOMET-001 registration-directed trial in NPM1-mutant acute myeloid leukemia (AML), as well as in our KOMET-007 combination trial. We look forward to sharing preliminary combination data in NPM1-mutant and KMT2A-rearranged AML early next quarter."

"Meanwhile," continued Dr. Wilson, "we continue to unlock the substantial therapeutic and commercial value of farnesyl transferase inhibition. We believe the positive results from our AIM-HN registration-directed trial of tipifarnib and the favorable safety and tolerability profile of tipifarnib in combination with alpelisib in our ongoing KURRENT-HN trial significantly de-risk development of our next-generation farnesyl transferase inhibitor (FTI), KO-2806. We are pleased to be in the clinic with KO-2806 and look forward to evaluating it in combination with other targeted therapies, including adagrasib in KRASG12C-mutated non-small cell lung cancer (NSCLC) and cabozantinib in clear cell renal cell carcinoma (ccRCC). If successful, we believe KO-2806 could become an ideal combination partner for multiple targeted therapies in large solid tumor indications."

Recent Highlights

Rapid pace of enrollment continues in registration-directed trial of ziftomenib in NPM1-mutant AML – The KOMET-001 registration-directed trial of ziftomenib in NPM1-mutant relapsed or refractory AML is expected to enroll a total of 85 patients in the U.S. and Europe, with a primary endpoint of complete remission (CR) or CR with partial hematologic recovery (CRh). In the Phase 1 trial, ziftomenib demonstrated a 35% CR rate and 45% overall response rate in 20 patients with NPM1-mutant AML treated at the recommended Phase 2 dose. NPM1-mutant AML accounts for approximately 30% of new AML cases annually and represents a disease of significant unmet need for which no approved targeted therapy exists. Kura expects to complete enrollment of all 85 patients in the Phase 2 registration-directed trial no later than mid-2024.
Preliminary data from KOMET-007 combination trial of ziftomenib upcoming – KOMET-007 is a Phase 1 dose-escalation study designed to assess safety, tolerability and preliminary activity of ziftomenib in combination with either: 1) venetoclax and azacitidine in patients with relapsed/refractory NPM1-mutant and KMT2A-rearranged AML or 2) standard induction cytarabine/daunorubicin chemotherapy (7+3) in NPM1-mutant and KMT2A-rearranged patients in the frontline setting. The Company expects to share preliminary data from 20 patients in KOMET-007, including NPM1-mutant and KMT2A-rearranged patients treated with ziftomenib in the newly diagnosed and relapsed/refractory AML settings, early in the first quarter of 2024.
Positive results from AIM-HN registration-directed trial of tipifarnib in HRAS mutant HNSCC – Kura recently presented positive results from the AIM-HN registration-directed trial of tipifarnib as a monotherapy in patients with HRAS mutant head and neck squamous cell carcinoma (HNSCC). The results were featured during a late-breaking mini-oral session at the European Society for Medical Oncology Congress in Madrid. The Company continues to evaluate whether the combination of tipifarnib and alpelisib has potential to extend the clinical benefit observed in the AIM-HN trial to a broader set of HNSCC patients in its ongoing KURRENT-HN study.
First patient dosed in FIT-001 dose-escalation study of KO-2806 – Last month, Kura announced that the first patient was dosed in its FIT-001 Phase 1 dose-escalation trial of KO-2806. KO-2806 is a next-generation FTI designed to improve upon potency, pharmacokinetic and physicochemical properties of earlier FTI drug candidates. Concurrent with dose escalation as a monotherapy in the FIT-001 trial, the Company also plans to evaluate KO-2806 in dose-escalation combination cohorts with adagrasib in KRASG12C-mutated NSCLC and with cabozantinib in ccRCC.
Preclinical data supports clinical combinations of KO-2806 with adagrasib and cabozantinib – Kura presented preclinical data at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) supporting its rationale to combine KO-2806 with adagrasib in KRASG12C-mutated NSCLC and with cabozantinib in ccRCC. The new findings illustrate the potential for FTIs to drive enhanced antitumor activity and address mechanisms of innate and adaptive resistance to targeted therapies such as KRAS inhibitors and tyrosine kinase inhibitors.
Clinical collaboration with Mirati to evaluate KO-2806 and adagrasib in KRASG12C-mutated NSCLC – Earlier today, Kura announced a clinical collaboration and supply agreement with Mirati Therapeutics to evaluate the combination of KO-2806 and adagrasib in patients with KRASG12C-mutated NSCLC. Under the terms of the agreement, Kura will sponsor the Phase 1 study and Mirati will supply adagrasib for the study. The collaboration highlights the potential to address the urgent need for more durable and effective treatment options for patients with cancers driven by the KRASG12C-mutant oncogene.
Brian Powl appointed as Chief Commercial Officer – Mr. Powl joined Kura in August 2023 with more than two decades of experience in building commercial brands in hematology and oncology, with expertise in developing and executing patient-focused strategies across sales, marketing and market access for global biotech and pharmaceutical products, including extensive global experience in hematologic malignancies.
Financial Results

Research and development expenses for the third quarter of 2023 were $29.3 million, compared to $25.0 million for the third quarter of 2022.
General and administrative expenses for the third quarter of 2023 were $13.1 million, compared to $11.6 million for the third quarter of 2022.
Net loss for the third quarter of 2023 was $38.6 million, compared to a net loss of $35.5 million for the third quarter of 2022. This includes non-cash share-based compensation expense of $7.1 million, compared to $6.4 million for the same period in 2022.
As of September 30, 2023, Kura had cash, cash equivalents and short-term investments of $452.6 million, compared to $438.0 million as of December 31, 2022.
Based on its operating plan, management expects that cash, cash equivalents and short-term investments will fund current operations to mid-2026.
Forecasted Milestones

Report preliminary data from 20 patients in the KOMET-007 trial of ziftomenib in combination with venetoclax and azacitidine or 7+3 early in the first quarter of 2024.
Dose the first patients in the KOMET-008 trial of ziftomenib in combination with additional standards of care, including the FLT3 inhibitor gilteritinib, in the first quarter of 2024.
Initiate the ziftomenib post-transplant maintenance program in the first quarter of 2024.
Complete enrollment of 85 patients in the KOMET-001 registration-directed trial of ziftomenib in NPM1-mutant AML by mid-2024.
Determine the optimum biologically active dose for tipifarnib in combination with alpelisib and determine next steps for the program by mid-2024.
Dose the first patients in the FIT-001 dose-escalation trial of KO-2806 in combination with adagrasib in KRASG12C-mutated NSCLC by mid-2024.
Dose the first patients in the FIT-001 dose-escalation trial of KO-2806 in combination with cabozantinib in ccRCC by mid-2024.
Conference Call and Webcast

Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, November 2, 2023, to discuss the financial results for the third quarter 2023 and to provide a corporate update. The live call may be accessed by dialing (888) 886-7786 for domestic callers and (416) 764-8658 for international callers and entering the conference ID: 34983466. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com.

On November 2, 2023 Lisata Therapeutics, Inc. (Nasdaq: LSTA) ("Lisata" or the "Company"), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, reported its financial results for the third quarter ended September 30, 2023 (Press release, Caladrius Biosciences, NOV 2, 2023, View Source [SID1234636812]).

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"Momentum we established during the first half of the year continued during the third quarter with the achievement of several milestones related to ongoing and planned clinical studies of our lead investigational product, LSTA1," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Lisata. "For example, LSTA1 is now the recipient of multiple orphan drug designations including pancreatic cancer in both the U.S. and Europe, as well as malignant glioma in the U.S. Additionally, as we recently announced, we have successfully treated the first patient in each of the head and neck squamous cell carcinoma and cholangiocarcinoma cohorts of the BOLSTER trial ("BOLSTER"), and we expect a steady uptake in enrollment over the coming quarters. In September, we announced that the ASCEND study ("ASCEND") in Australia received a positive outcome from the planned interim futility analysis by the study’s Independent Data Safety Monitoring Committee ("IDSMC"), which recommended continuation of the study without modification. We are also happy to report that full enrollment in Cohort A of ASCEND has been achieved and that overall enrollment in the study is now approximately 95% complete. With that, we now expect that we could have topline data from Cohort A as early as the fourth quarter of 2024, a full year earlier than originally anticipated. We intend to use the results of the ASCEND trial to explore possible conditional approvals in several jurisdictions and to design an optimized Phase 3 program in Pancreatic Ductal Adenocarcinoma ("PDAC"). Finally, the iLSTA study, done in collaboration with WARPNINE in Australia, a foundation dedicated to accelerating the development of treatments for gastrointestinal cancers, is enrolling rapidly."
Dr. Mazzo continued, "With continued careful management of capital, we reiterate that our expected cash runway projects into early 2026, funding each of our trials through to data. We believe we remain well-positioned to focus on the execution of our development plans and achieve our goal of getting to meaningful clinical data readouts as soon as possible."

Development Portfolio Highlights

LSTA1 as a treatment for solid tumor cancers in combination with other anti-cancer agents
LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered (i.e., molecularly bound) anti-cancer drugs to target and penetrate solid tumors more effectively. LSTA1 actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor, while normal tissues are not expected to be affected. In preclinical models, LSTA1 has also shown the ability to modify the tumor microenvironment, thereby making tumors more susceptible to immunotherapies and inhibiting the metastatic cascade (i.e., the spread of cancer to other parts of the body). Lisata and its development collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies, and RNA-based therapeutics. To date, LSTA1 has also demonstrated favorable safety, tolerability and activity in completed

and ongoing clinical trials designed to test its ability to enhance delivery of standard-of-care chemotherapy for pancreatic cancer. Currently, LSTA1 is the subject of multiple ongoing or planned Phase 1b/2a and 2b clinical studies being conducted globally in a variety of solid tumor types in combination with a variety of anti-cancer regimens. These studies include:

•ASCEND: Phase 2b double-blind, randomized, placebo-controlled clinical trial evaluating LSTA1 in patients with metastatic Pancreatic Ductal Adenocarcinoma ("mPDAC"). The trial is being conducted at up to 40 sites in Australia and New Zealand led by the Australasian Gastro-Intestinal Trials Group in collaboration with the University of Sydney and with the National Health and Medical Research Council Clinical Trial Centre at the University of Sydney as the Coordinating Centre. Planned interim futility analysis by the IDMC received a positive outcome. Cohort A has been fully enrolled. Total enrollment completion is projected for the second quarter of 2024; however, current total enrollment is at approximately 95% of target, so earlier total enrollment completion may be achieved.
•BOLSTER: Phase 2a double-blind, placebo-controlled, multi-center, randomized basket trial in the U.S., Europe, Canada, and Asia evaluating LSTA1 in combination with standards of care in advanced solid tumors including head and neck, esophageal and cholangiocarcinoma. Enrollment is open and proceeding as planned. First patients have been treated in head and neck cancer as well as cholangiocarcinoma cohorts.
•CENDIFOX: Phase 1b/2a open-label trial in the U.S. of LSTA1 in combination with neoadjuvant FOLFIRINOX based therapies in pancreatic, colon and appendiceal cancers. The trial continues to make steady progress with enrollment completion expected by the fourth quarter of 2023 and data readouts expected in 2024.
•LSTA1 is currently being evaluated in combination with gemcitabine and nab-paclitaxel in a Phase 1b/2a open-label trial in China led by Qilu Pharmaceutical. During the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting, Qilu Pharmaceutical presented an abstract sharing preliminary data from the study which, thus far, has corroborated previously reported findings from the phase 1b/2a trial of LSTA1 plus gemcitabine and nab-paclitaxel conducted in Australia in patients with mPDAC. Final data is expected by the end of the second quarter of 2024.
•iLSTA: Phase 1b/2a randomized, single-blind, single-center, safety and pharmacodynamic trial in Australia evaluating LSTA1 in combination with the checkpoint inhibitor, durvalumab, plus standard-of-care chemotherapy, nab-paclitaxel, and gemcitabine, versus standard-of-care alone in patients with locally advanced non-resectable PDAC. Enrollment completion is expected by the end of the second quarter of 2024.
•The Company will soon initiate the study of LSTA1 in combination with temozolomide in Glioblastoma Multiforme ("GBM"). This study is designed as a Phase 2a double-blind, placebo-controlled, randomized, proof-of-concept study evaluating LSTA1 when added to standard of care ("SoC") temozolomide versus temozolomide and matching LSTA1 placebo in subjects with newly diagnosed GBM. It will be conducted across multiple sites in Estonia and Latvia and is targeted to enroll 30 patients with a randomization of 2:1 LSTA1 + SoC versus Placebo + SoC. Target for the first patient treated is in the fourth quarter of 2023. Importantly and as the Company recently announced, LSTA1 has been granted orphan drug designation by the U.S. FDA for malignant glioma. This action by the FDA not only highlights the unmet medical need but also recognizes the potential of LSTA1 to benefit patients in this indication.
•Lisata will also soon initiate a study of LSTA1 in combination with HIPEC interoperative intraperitoneal lavage in peritoneal carcinomatosis, a condition which develops as a result of the contiguous spread of primary cancers such as ovarian, colorectal and appendiceal along the peritoneum. The study is a Phase I single-center, unblinded, randomized controlled trial to determine the safety and tolerability of LSTA1 administered intraperitoneally in patients with peritoneal metastases from colorectal, appendiceal, or ovarian cancer undergoing Cytoreductive Surgery ("CRS") and HIPEC. Twenty-one total participants will be randomized 2:1 to receive LSTA1 with HIPEC versus HIPEC alone after CRS. We anticipate the first patient treated to be in the fourth quarter of 2023.

Third Quarter 2023 Financial Highlights

Research and development expenses were approximately $3.4 million for the three months ended September 30, 2023, compared to $3.3 million for the three months ended September 30, 2022, representing an increase of $45,000 or 1.3%. Expenses this quarter were primarily due to study activities associated with the BOLSTER trial, enrollment activities for the ASCEND study, startup activities for the LSTA1 GBM study and chemistry, manufacturing and control activities for LSTA1 to support all development activities.
General and administrative expenses were approximately $2.6 million for the three months ended September 30, 2023, compared to $4.0 million for the three months ended September 30, 2022, representing a decrease of $1.4 million or 35.3%. This was primarily due to non-recurring merger related costs in the prior year, a decrease in equity expense due to prior year performance stock unit vesting, merger option assumption expense, departing board member restricted stock unit vesting and timing of our annual stockholder meeting versus the prior year.
Overall, net losses were $5.3 million for the three months ended September 30, 2023, compared to $37.4 million for the three months ended September 30, 2022. Excluding the in-process research and development expense of $30.4 million relating to our merger with Cend Therapeutics in September 2022, net losses for the three months ended September 30, 2023 decreased by $1.7 million or 24.7% compared to the three months ended September 30, 2022.

Balance Sheet Highlights

As of September 30, 2023, the Company had cash, cash equivalents and marketable securities of approximately $54.4 million. Based on its current expected capital needs, the Company believes that its projected capital will fund its current proposed operations into early 2026, encompassing anticipated data milestones from all its ongoing and planned clinical trials.

Conference Call Information

Lisata will hold a live conference call on Thursday, November 2, 2023, at 4:30 p.m. Eastern time to discuss financial results, provide a business update and answer questions.
Those wishing to participate must register for the conference call by way of the following link: CLICK HERE TO REGISTER. Registered participants will receive an email containing conference call details with dial-in options. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time.
A live webcast of the call will also be accessible under the Investors & News section of Lisata’s website and will be available for replay beginning two hours after the conclusion of the call for 12 months.

On November 2, 2023 Wugen, Inc., a clinical-stage biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, reported that it will be presenting four abstracts, now available online, at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition Dec. 9-12 (Press release, Wugen, NOV 2, 2023, View Source [SID1234636804]).

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In an oral presentation, Armin Ghobadi, M.D., from Washington University in St. Louis, will share data from study WU-CART-007 1001, "A Phase 1/2 dose-escalation and dose-expansion study of the safety and efficacy of anti-CD7 allogeneic CAR-T cells (WU-CART-007) in patients with relapsed or refractory T-cell Acute Lymphoblastic Leukemia (T ALL)/Lymphoblastic Lymphoma (LBL)."

In a second oral presentation, Sergio Rutella, M.D., Ph.D., from Nottingham University in the U.K. will present pre-clinical findings for WU-NK-101, a cytokine-reprogrammed, expanded, cryopreserved, off-the-shelf, un-engineered memory-like NK cell product derived from peripheral blood mononuclear cells (PBMCs).

The Wugen oral and poster presentation schedule at ASH (Free ASH Whitepaper) is below. The meeting will take place at the Marriott Marquis in San Diego and virtually. Access the full schedule here.

Oral Presentations

Title: Phase 1/2 Dose-Escalation/Dose-Expansion Study of Anti-CD7 Allogeneic CAR-T Cells (WU-CART-007) in Relapsed or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma (T-ALL/LBL)

Presenter: Armin Ghobadi, M.D., Washington University, St. Louis, MO

Presentation time/location: Monday, Dec. 11, 10:45 a.m. PT; Room 6CF

Session: #704, Cellular Immunotherapies: Early Phase and Investigational Therapies: Novel Approaches to Enhance Cellular Therapies and Immune Responses in Leukemias and Lymphomas, 10:30 a.m.- 12 p.m. PT

Title: WU-NK-101 (W-NK), a Memory-like (ML) NK Cell, Intrinsically Overcomes Factors Restricting Adoptive Cell Therapy (ACT) in Acute Myeloid Leukemia (AML)

Presenter: Sergio Rutella, M.D., Ph.D., Nottingham Trent University, Nottingham, UK

Presentation time/location: Sunday, Dec. 10; 10:15 a.m. PT; Room 6A

Session: #703, Cellular Immunotherapies: Basic and Translational: Novel Approaches for Next Generation Cellular Immunotherapies, 9:30-11 a.m. PT

Poster Presentations

Title: Adoptively Infused Memory-like Natural Killer Cells Impact Adaptive Immune Responses in Patients with Acute Myeloid Leukemia

Presenter: Sergio Rutella, M.D., Ph.D., Nottingham Trent University, Nottingham, UK

Presentation time/location: Monday, Dec. 11; 6-8 p.m. PT; Halls G-H

Title: WU-NK-101 (W-NK), a Memory-like (ML) NK cell, Naturally Overcomes Tumor Microenvironment (TME) Metabolic Challenges, Retaining Anti-tumor Potency

Presenter: Nupur Bhatnagar, Ph.D., Metafora Biosystems, Paris, France

Presentation time/location: Monday, Dec. 11; 6-8 p.m. PT; Halls G-H

About WU-CART-007

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). Additional information is available on clinicaltrials.gov, identifier NCT# 04984356. WU-CART-007 has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration for the treatment of R/R T-ALL/LBL.

About WU-NK-101

WU-NK-101 is a novel immunotherapy harnessing the power of memory natural killer (NK) cells to treat liquid and solid tumors. Memory NK cells are hyper-functional, long-lasting immune cells that exhibit enhanced anti-tumor activity and a cytokine-induced memory-like (CIML) phenotype. This rare cell population has a superior phenotype, proliferation capacity, and metabolic fitness, making it better suited for cancer therapy than other NK cell therapies. Wugen is applying its proprietary MonetaTM platform to advance WU-NK-101 as a commercially scalable, off-the-shelf cell therapy for cancer. WU-NK-101 is currently in development for acute myelogenous leukemia (AML). Wugen is planning to initiate solid tumor studies of WU-NK-101 in combination with cetuximab. Studies of WU-NK-101 to date have shown promising robust in vivo activity in various tumor indications, retention of anti-cancer activity in TME, resistance to immune suppression, and enhanced activity with checkpoint inhibitors. WU-NK-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of AML.

About the MonetaTM Platform

Wugen’s proprietary MonetaTM manufacturing platform is a robust, efficient, scalable process to generate off-the-shelf memory natural killer (NK) cell therapies with enhanced anti-tumor functionality. The MonetaTM platform uses cytokine fusion complexes for streamlined and consistent manufacturing, is free of feeder cells for enhanced safety, and integrates cryopreservation to allow convenient dosing options for cancer patients.

On November 2, 2023 Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, reported that preclinical and clinical data supporting the Company’s novel platform and approach for treating acute myeloid leukemia (AML), will be presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, being held from December 9-12, 2023, in San Diego, CA (Press release, Vor BioPharma, NOV 2, 2023, View Source [SID1234636802]).

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"These three oral and two poster presentations represent further preclinical and clinical validation of our platform and reflect the significant progress we continue to make as we develop truly novel next generation transplants that have the potential to improve the lives of patients with blood cancers," said Dr. Robert Ang, Vor Bio’s President and Chief Executive Officer.

VBP101 Clinical Data Update

An abstract providing a clinical update from the VBP101 clinical trial (NCT048499910), a Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (VOR33) in patients with AML, was accepted by ASH (Free ASH Whitepaper) for oral presentation. This data supports robust neutrophil engraftment of trem-cel and provides evidence of hematologic protection from MylotargTM, a CD33-targeted antibody drug conjugate.

An updated data release from VBP101 is expected by the Relapse After Transplant and Cellular Therapy (HSCT²) conference taking place November 10-11, 2023.

CD33CART Study Clinical Data Update

The Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) released data providing a clinical update on a Phase 1/2 study of CD33CART (NCT03971799)1, an autologous CAR-T therapy targeting CD33 (also referred to as VCAR33AUTO) which uses the same CAR-T construct as VCAR33ALLO. Nineteen pediatric and young adult patients with relapsed/refractory AML with a median age of 16 years were infused in the Phase 1 portion of the study. This data shows that as of the cutoff date of June 1, 2023, 2 of 5 (40%) evaluable patients treated at the highest dose level (DL4, 1 x 107 CAR+ cells/kg) achieved complete remission. Transient CD33CART expansion was detected in 11 (58%) subjects across all doses tested and in all 6 (100%) subjects evaluated at DL4, as of the cutoff date. Four out of 19 evauable patients treated had cytokine release syndrome (CRS) ≥ Grade 3. The study is being led by Nirali Shah, MD, MHSc, Head, Hematologic Malignancies Section, Pediatric Oncology Branch, National Cancer Institute and Richard Aplenc, MD, PhD, MSCE, Professor of Pediatrics, Children’s Hospital of Philadelphia (CHOP). This abstract was accepted by ASH (Free ASH Whitepaper) for oral presentation.

Sarah K. Tasian, MD, is a co-investigator on the PTCTC-supported clinical trial and led the preclinical testing and translation of CD33CART with Terry Fry, MD, at the University of Colorado.

Dr. Tasian commented: "The interim results from our CD33 CAR T cell immunotherapy clinical trial are very encouraging. CD33CART is clearly an active therapy based upon our data to date, and the expansion phase of the study will provide additional critical safety and efficacy data. Our results provide a compelling foundation for Vor Bio’s approach using the same construct for their VBP301 study."

Vor Bio’s VCAR33ALLO uses the same CAR-T construct used in CD33CART. However, VCAR33ALLO uses a potentially superior T cell source from healthy transplant donors, which the Company believes are likely to have a more stem-like phenotype and greater potential for expansion, persistence, and anti-leukemic activity compared to a product derived from autologous sources.

Additional ASH (Free ASH Whitepaper) Presentations

The Company also released data from a single cell analysis studying molecular signatures from 28 AML patients in various stages of AML progression. This data is the most comprehensive analysis to date on AML profiling. This abstract was accepted by ASH (Free ASH Whitepaper) for oral presentation.

The preclinical collaboration between Vor Bio and Janssen yielded in vitro and in vivo xenotransplant data demonstrating that CD33-deleted allografts were synergistic with Janssen’s CD33 directed immunotherapy candidate (JNJ-67571244), and maintained robust on-target cytotoxicity while reducing production of inflammatory cytokines associated with CRS. This abstract was accepted by ASH (Free ASH Whitepaper) for poster presentation.

Lastly, a trial-in-progress poster will be presented on the Company’s VBP301 clinical trial, a Phase 1/2 multicenter, open-label, first-in-human study of VCAR33ALLO in patients with relapsed or refractory AML after allogeneic stem cell transplantation. This abstract was accepted by ASH (Free ASH Whitepaper) for poster presentation.

Full details of the ASH (Free ASH Whitepaper) 2023 presentations are as follows:

Vor Bio Clinical Abstracts

Abstract Title: Trem-cel, a CRISPR/Cas9 Gene-Edited Allograft Lacking CD33, Shows Rapid Primary Engraftment with CD33-Negative Hematopoiesis in Patients with High-Risk Acute Myeloid Leukemia (AML) and Avoids Hematopoietic Toxicity During Gemtuzumab Ozogamicin (GO) Maintenance Post-Hematopoietic Cell Transplant (HCT)

Format: Oral presentation

Session Name: Gene Therapies: New Approaches from Bench to Bedside

Session date and time: Sunday, December 10, 2023, 10:00 AM PST

Abstract Title: Phase 1/2 Study of Donor-Derived Anti-CD33 Chimeric Antigen Receptor Expressing T Cells (VCAR33) in Patients with Relapsed or Refractory Acute Myeloid Leukemia after Allogeneic Hematopoietic Cell Transplantation

Format: Trial in Progress Poster, #483

Session Name: Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster III

Session date and time: Monday, December 11, 2023, 6:00 PM – 8:00 PM PST

PTCTC Clinical Abstract

Abstract Title: CD33 CAR T-cells (CD33CART) for Children and Young Adults with Relapsed/Refractory AML: Dose-Escalation Results from a Phase I Multicenter Trial

Format: Oral presentation

Session Name: Cellular Immunotherapies: Early Phase and Investigational Therapies: Novel Approaches to Enhance Cellular Therapies and Immune Responses in Leukemias and Lymphomas

Session date and time: Monday, December 11, 2023, 11:00 AM PST

Vor Bio Preclinical Abstracts

Abstract Title: Multimodal Atlas of Paired Diagnosis and Relapse AML Samples Enables Novel Therapeutic Targeting of Surface Antigens

Format: Oral presentation

Session Name: Acute Myeloid Leukemias: Biomarkers, Molecular Markers and Minimal Residual Disease in Diagnosis and Prognosis: Biomarkers and Therapeutics

Session date and time: Saturday, December 9, 2023, 2:15 PM PST

Abstract Summary: The most comprehensive single cell AML atlas known to date (More than 450,000 cells from 28 AML patients) was generated to identify potential differences in molecular signatures in AML tumor types at multiple stages of AML progression. This extensive AML profiling offers deep insight into cell surface changes during disease progression and reveals the potential for multi-targeted treatment strategies.

Abstract Title: CD33-Deleted Hematopoietic Cells (trem-cel) are Protected from CD33xCD3 Bispecific Antibody Treatment and Produce Significantly Reduced Levels of Inflammatory Cytokines in Preclinical Studies

Format: Poster, #3425

Session Name: Experimental Transplantation: Basic and Translational: Poster II

Session date and time: Sunday, December 10, 2023, 6:00 PM – 8:00 PM PST

Abstract Summary: Preclinical proof-of-concept data resulting from the Company’s strategic collaboration with Janssen, demonstrated that the CD33 deleted hematopoietic compartment was protected from Janssen’s CD33 directed immunotherapy (JNJ-67571244) both in cytotoxicity assays and xenotransplantation studies, with reduction of inflammatory cytokines associated with CRS. These findings may enable the development of a next-generation AML treatment strategy by pairing a trem-cel transplant with a subsequent CD33-directed bispecific compound that could enhance the safety and effectiveness of the treatment while decreasing the harmful effect on the bone marrow.

Conference Call & Webcast Information

Members of the Vor Bio management team, joined by Sarah K. Tasian, MD, will conduct a live conference call and webcast to discuss the abstracts, today at 9:30 AM ET.