On November 11, 2024 Mural Oncology plc (Nasdaq: MURA), a clinical-stage immuno-oncology company developing novel, investigational engineered cytokine therapies designed to address areas of unmet need for patients with a variety of cancers, reported that members of the management team will participate in two upcoming investor conferences (Press release, Mural Oncology, NOV 11, 2024, View Source [SID1234648074]).

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Jefferies London Healthcare Conference

1×1 meetings with Caroline Loew, Ph.D., CEO, Vicki Goodman, MD, Chief Medical Officer, Adam Cutler, Chief Financial Officer

Date: Tuesday, November 19-Wednesday, November 20, 2024

Piper Sandler 36th Annual Healthcare Conference

Fireside chat with Caroline Loew, Ph.D., CEO, Vicki Goodman, MD, Chief Medical Officer, and Adam Cutler, Chief Financial Officer

Date: Wednesday, December 4, 2024

Time: 9:30 a.m. ET

A webcast of the presentation will be available at View Source

On November 11, 2024 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported its financial results for the quarter ended September 30, 2024 (Press release, Moleculin, NOV 11, 2024, View Source [SID1234648073]). As previously announced, the Company will host a conference call and live audio webcast to discuss the operational and financial results at 8:30 AM ET on Monday, November 11, 2024 (details below).

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"We are thrilled to have emerged as a late stage company. Our focus remains on the ramp up for and execution of our upcoming MIRACLE trial. We have been extremely active and our recent interactions with potential clinical trial sites globally have been overwhelmingly positive as we prepare for the start of enrollment and dosing early next year. We believe with our recent clinical and regulatory ‘wins,’ we have foundationally set the stage for a transformational year ahead and the opportunity to drive significant value for all stakeholders. With that said, we continue to follow our CRc (complete response composite) preliminary data from our MB-106 Phase 1B/2 AML clinical trial. We are also pleased with the median durability continuing to climb – and is now in excess of 8 months," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

Recent Highlights

Appointed Daniel D. Von Hoff, M.D., F.A.C.P., FASCO, FAACR, leading expert in Pancreatic Cancer to its Scientific Advisory Board to Support Development of Annamycin;
Hosted a Virtual Acute Myeloid Leukemia KOL event with internationally renowned Acute Myeloid Leukemia (AML) Key Opinion Leaders to discuss Annamycin, the use of anthracyclines, how Annamycin could significantly change the AML treatment landscape, and the Company’s recently announced global Phase 3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (the "MIRACLE" trial);
Presented positive in vivo efficacy data of Annamycin in orthotopic and experimental lung metastatic models of Sarcoma in a poster titled "Annamycin: Opening New Doors for Organotropic Targeting of Primary and Metastatic Lung Cancer," at the IASLC 2024 World Conference on Lung Cancer;
Commenced enrollment and treatment of patients in the Investigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for the treatment of adults with glioblastoma; and
Closed a $5.5 million financing upfront with up to an additional $11.0 million of potential aggregate gross proceeds upon the exercise in full of milestone-linked warrants.
Clinical Development Update

Annamycin is currently being evaluated in ongoing clinical trials for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).

AML

The Company recently announced the positive discussion and outcome of its End of Phase 1B/2 (EOP1B/2) meeting with the US Food and Drug Administration (FDA) supporting the advancement of Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") to a Phase 3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US.

The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, is expected to initially utilize an adaptive design whereby approximately the first 75 to 90 subjects will be randomized to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin. At that point, the trial will be unblinded to select the optimum dose for Annamycin. For the second part of the trial, approximately 240 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin. The selection of the optimum dose will be based not only on the overall balance of safety, tolerability, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

Expected Milestones for Annamycin AML Development Program

4Q 2024 – Contracting with MIRACLE trial sites and IRB approval
1Q 2025 – First subject treated in MIRACLE trial
4Q 2025 – Recruitment and overall efficacy rate update (n=45)
2H 2026 – Interim efficacy and safety data (n=90) unblinded and Optimum Dose set for MIRACLE trial
2027 – Begin enrollment of 3rd line subjects in MIRACLE2
2027 – Enrollment ends in 2nd line subjects
2028 – Primary efficacy data for 2nd line subjects in MIRACLE
2028 2H – Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE
STS Lung Metastases

As previously announced, the Company completed enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases. Subjects who had stable disease at the time of study discontinuation were followed for progression free response and overall survival. The study database is locked, and the clinical study report is being written and should be completed in early 2025 and will be released in detail at that time

Expected Milestones for Annamycin STS Lung Mets Development Program

2025 – Final MB-107 data readout
2025 – Identify next phase of development / pivotal program
Summary of Financial Results for the Third Quarter 2024

Research and development (R&D) expense was $4.9 million and $3.3 million for the three months ended September 30, 2024 and 2023, respectively. The increase over the prior year period is mainly related to costs incurred for clinical trials (clinical research organization and drug production) and sponsored research costs.

General and administrative expense was $2.2 million and $2.6 million for the three months ended September 30, 2024 and 2023, respectively. The decrease of $0.4 million is mainly related to a decrease in regulatory and legal fees.

As of September 30, 2024, the Company had cash and cash equivalents of $9.4 million and believes that the existing cash and cash equivalents as of September 30, 2024, will be sufficient to fund planned operations into the first quarter of 2025.

Conference Call and Webcast

Moleculin management will host its quarterly conference call and webcast for investors, analysts, and other interested parties on Monday, November 11, 2024 at 8:30 AM ET.

Interested participants and investors may access the conference call by dialing (877) 407-0832 (domestic) or (201) 689-8433 (international) and referencing the Moleculin Biotech Conference Call. The live audio webcast will be accessible on the Events page of the Investors section of the Moleculin website, moleculin.com, and will be archived for 90 days.

On November 11, 2024 Imvax, Inc., a clinical-stage biotechnology company developing personalized, whole-tumor derived immunotherapies, reported an oral presentation and two poster presentations at the 2024 Society for NeuroOncology (SNO) 29th Annual Meeting in Houston, Texas, from November 21-24, 2024 (Press release, Imvax, NOV 11, 2024, View Source;utm_medium=rss&utm_campaign=imvax-to-present-safety-data-on-lead-program-igv-001-at-2024-sno-annual-meeting [SID1234648072]). Separately, the Company will also host a symposium focused on the development of IGV-001 for the treatment of newly diagnosed glioblastoma.

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Imvax will present blinded safety data from the Company’s ongoing randomized, multicenter, double-blind, placebo-controlled Phase 2b clinical trial of IGV-001 in patients with newly diagnosed glioblastoma (ndGBM). The Phase 2b study to assess the safety and efficacy of IGV-001 in patients with ndGBM (NCT04485949) has completed enrollment and top-line results are expected to be presented in mid-2025. Imvax will also present follow-on data from its Phase 1b study of IGV-001 in patients with ndGBM, as well as preclinical data of IGV-001 in combination with checkpoint inhibitors.

The details of the oral presentation and two poster presentations are below:

Title: Early safety data from a randomized, multicenter, double-blind, phase 2b study of IGV-001, an autologous cell immunotherapy, versus placebo, in newly diagnosed glioblastoma (ndGBM)
Number: CTIM-14
Timing: November 22, 7:25 p.m. – 7:35 p.m. CST
Presenter: Brad Elder, M.D.

Title: Long-term survivors from a Phase 1b study of IGV-001 in patients with newly diagnosed glioblastoma
Number: CTIM-11
Timing: November 21-22, 7:30 p.m. – 9:30 p.m. CST
Presenter: Raul-Perez Olle, M.D., Ph.D.

Title: IGV-001: A biologic-device combination product elicits potent anti-GBM responses as a monotherapy and in combination with check point inhibition
Number: IMMU-30
Timing: November 21-22, 7:30 p.m. – 9:30 p.m. CST
Presenters: Jenny Zilberberg, Ph.D. & Mark A. Exley, Ph.D.

On Friday, November 22, 2024, from 12:45 p.m. – 1:45 p.m. CST, Raul Perez-Olle, M.D., Ph.D., SVP, Head of Clinical Development and Medical Affairs at Imvax, Brad Zacharia, M.D., M.S., FAANS, Associate Professor of Neurosurgery and Otolaryngology at Penn State Health, and Brad Elder, M.D., Director, Neurosurgical Oncology, and Associate Professor, Department of Neurological Surgery at The Ohio State University Wexner Medical Center, will host a symposium, entitled "Development of IGV-001 for the treatment of newly diagnosed glioblastoma," focused on Imvax’s proprietary Goldspire platform and the development of IGV-001 for the treatment of ndGBM. Register for the session here.

About IGV-001
IGV-001 is an autologous biologic-device combination product derived from Imvax’s proprietary Goldspire immuno-oncology platform for solid tumors, which involves a unique approach to inducing a broad and durable immune response against tumors. Phase 1 studies showed that IGV-001 was safe and well tolerated, and a Phase 1b ndGBM study also yielded several efficacy signals, including significant improvements in PFS, OS, radiographic evidence of tumor response, and multiple biomarker changes that supported the presence of an immune response (Andrews DW, et al., Clin Cancer Res. 2021;27(7):1912-1922). In ten Stupp-eligible ndGBM patients in the highest dose cohort treated with IGV-001, median PFS was 17.1 months, compared with 6.5 months in historical standard-of-care (SOC) treatment, and median OS was 38.2 months, compared with 16.2 months in historical SOC.

On November 11, 2024 Pharma Two B Ltd. ("Pharma Two B"), a late-clinical stage company that is developing P2B001, an innovative combination product candidate for the treatment of Parkinson’s Disease ("PD") and Hepion Pharmaceuticals, Inc. (Nasdaq: HEPA) ("Hepion"), a clinical stage biopharmaceutical company that had been developing a treatment for non-alcoholic steatohepatitis ("NASH"), hepatocellular carcinoma ("HCC"), and other chronic liver diseases, reported that the U.S. Securities and Exchange Commission ("SEC") has declared effective the registration statement on Form F-4 (as amended, the "Registration Statement") filed with the SEC related to Pharma Two B’s merger transaction with Hepion as previously announced on July 22, 2024 (the "Proposed Transaction") (Press release, Hepion Pharmaceuticals, NOV 11, 2024, View Source [SID1234648071]).

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The Proposed Transaction, which has been approved by the respective boards of directors of Pharma Two B and Hepion, is expected to close in the fourth quarter of 2024 and remains subject to approval by both Pharma Two B and Hepion’s respective stockholders, regulatory approval, listing of Pharma Two B’s ordinary shares on Nasdaq under the ticker symbol "PHTB" and other customary closing conditions. Upon the anticipated closing of the Proposed Transaction, the combined company will operate under the "Pharma Two B" name.

Hepion also announced that a special meeting (the "Special Meeting") of its stockholders will be held on December 12, 2024 to approve the Proposed Transaction. The Special Meeting will be held at 9:00 a.m. Eastern Time via live webcast at www.virtualshareholdermeeting.com/HEPA2024SM. Hepion stockholders of record at the close of business on the record date of November 6, 2024 are entitled to vote at the Special Meeting. Hepion filed its definitive proxy statement/prospectus relating to the Proposed Transaction with the SEC and will mail it to stockholders on or about November 8, 2024. More details about the Proposed Transaction and the resolutions to be voted upon at the Special Meeting can be found in the definitive proxy statement/prospectus, available at View Source Hepion stockholders who need assistance in completing the proxy card, need additional copies of the proxy statement/prospectus, or have questions regarding the Special meeting may contact Hepion’s proxy solicitor, Campaign Management, by calling 1-855-422-1042 or emailing [email protected].

A.G.P./Alliance Global Partners is serving as financial advisor to Hepion and Sheppard, Mullin, Richter & Hampton LLP is acting as U.S. legal advisor to Hepion and Lipa Meir & Co.is acting as Israeli legal advisor to Hepion. Sullivan & Worcester LLP is serving as legal advisor to A.G.P.

Laidlaw & Company (UK) Ltd. is acting as financial advisor to Pharma Two B. and Meitar Law Offices and Goodwin Procter LLP are acting as legal advisors to Pharma Two B.

On November 11, 2024 Gritstone bio, Inc. (OTC: GRTSQ), a clinical-stage biotechnology company working to develop the world’s most potent vaccines, reported encouraging updated interim Phase 2 data from the ongoing Phase 2 study evaluating GRANITE, its individualized neoantigen targeting immunotherapy, in first-line microsatellite stable colorectal cancer (MSS-CRC) (Press release, Gritstone Bio, NOV 11, 2024, View Source [SID1234648070]). The ongoing randomized, controlled study is evaluating the clinical benefit of maintenance therapy with GRANITE (GRT-C901/GRT-R902) in combination with immune checkpoint inhibitors (ICI) in addition to fluoropyrimidine/bevacizumab versus fluoropyrimidine/bevacizumab alone.

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"Our encouraging Phase 2 data for GRANITE in MSS-CRC continue to mature and demonstrate durable benefit over time. With two additional months of follow-up, relative progression-free survival has further improved in the analysis of all patients treated with GRANITE, and most notably, in those with a lower tumor burden at study baseline," said Andrew Allen, MD, PhD, Co-founder, President & CEO of Gritstone bio. "The powerful anti-tumor immunity induced by GRANITE in an immunologically cold tumor setting like MSS-CRC underscores a differentiated approach with a multitude of expansion opportunities across solid tumor indications. Moreover, these data also further de-risk SLATE, our off-the-shelf cancer immunotherapy program, and more importantly, reinforce the transformative potential of Gritstone’s overall immunotherapy platform."

Dr. Allen continued, "We are focused on our mission to deliver potentially life-saving treatments like GRANITE to millions of patients worldwide and continue to explore several strategic and funding alternatives during the financial restructuring process. The compelling progression-free survival data in a challenging disease context, coupled with a well-tolerated safety profile, supports further continuation of the GRANITE program by a party who shares our vision to deliver novel treatments leveraging the immune response to fight tough to treat cancers."

Key Findings from an Updated Interim Phase 2 Analysis in Front-Line Metastatic MSS-CRC
Data cut as of October 17, 2024 vs. August 19, 2024

An updated October analysis of progression-free survival (PFS) per RECIST v1.1 included an additional two months of follow-up.

28% (11/39) GRANITE and 13% (4/30) of control patients remain on study and free of progression vs. 33% (13/39) GRANITE and 23% (7/30) of control patients from August analysis; the majority of GRANITE patients still on study have undetectable ctDNA using Gritstone’s high-sensitivity, tumor-informed assay
Clinical benefit improved compared to previous analysis in patients with low and high disease burden (based on ctDNA levels)
Clinical benefit was most notable in patients with low disease burden at study entry
Low baseline ctDNA levels (eg at study entry) is a likely prognostic and predictive factor
Overall survival data remain immature, with mature data expected in 2H 2025
GRANITE continues to demonstrate a favorable safety and tolerability profile
Summary of Progression-Free Survival Results
Relative Risk Reduction;
PFS Hazard Ratio*
October Analysis 2024 August Analysis 2024
All patients
n = 69 27%;
0.73, [90% CI, 0.44-1.21] 21%;
0.79, [90% CI, 0.47-1.35]
ctDNA-lo^
n = 31 50%;
0.5, [90% CI, 0.20-1.28] 43%;
0.57, [90% CI, 0.20-1.58]
ctDNA-hi^
n = 30 22%;
0.78, [90% CI, 0.39-1.58] 13%;
0.87, [90% CI, 0.41- 1.86]
*The protocol-defined PFS analysis (per RECIST v1.1) is reported herein for all groups. An exploratory analysis using both radiographic and clinical progression data was reported for the ctDNA subgroups on September 30, 2024.
^Not all patients had baseline samples for analysis

Restructuring Process
On October 10, 2024, Gritstone filed a voluntary petition under chapter 11 of the United States Bankruptcy Code in the United States Bankruptcy Court for the District of Delaware (the "Court".) During its financial restructuring process, Gritstone intends to operate in the ordinary course and remains committed to advancing its clinical programs, including its ongoing neoantigen immunotherapy and infectious disease programs, and driving innovation in immunotherapy and vaccine development.

Gritstone is continuing its sale process and actively pursuing bidders for all or any portion of the Company’s assets to continue research and development of its next-generation vaccines and immunotherapies for oncology and infectious diseases. The bid deadline for the sale process is December 4, 2024. For more information, please reach out to [email protected].

About the GO-010 Study
GO-010 (NCT05141721) is a Phase 2, randomized, open-label study designed to evaluate the clinical benefit of maintenance therapy with GRANITE (GRT-C901/GRT-R902), a neoantigen targeting immunotherapy, in combination with immune checkpoint inhibitors (ICI) in addition to fluoropyrimidine/bevacizumab versus fluoropyrimidine/bevacizumab alone in patients with first-line microsatellite stable colorectal cancer (MSS-CRC). 104 patients were randomized 1:1 in the study: 69 patients (39 GRANITE arm, 30 control arm) are included in the treated analysis above. Demographics and clinical characteristics were balanced between arms (e.g., stage, sidedness, presence of liver metastases), with the vast majority (80%) of patients having liver metastases in the treated analysis. Thirty-five patients did not advance to study treatment after oxaliplatin most commonly due to withdrawing consent (n=15), disease progression (n=7), and other reasons (n=13) (12 in GRANITE arm; 23 in control arm).