On November 14, 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 it has amended the clinical trial protocol with the U.S. Food and Drug Administration ("FDA") for its Phase 3 pivotal trial protocol evaluating 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") for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (MB-108) (Press release, Moleculin, NOV 14, 2024, View Source [SID1234648407]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US. Additionally, the Company released a Virtual Investor "What This Means" segment to discuss the amended protocol. Access the segment here.

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"Our team has been thoughtful and strategic with the design of the MIRACLE trial, which may allow for possible accelerated approval of Annamycin in combination with cytarabine for the treatment of relapsed or refractory AML. This amended protocol enables us to share definitive data earlier, which helps to partially de-risk financing the trial and potentially accelerates the timeline for strategic partnering. We believe that the unblinding of data at 45 subjects will enable us to begin assessing all three arms of the study and provide us with a clear path forward in understanding the potential of Annamycin for AML patients. This change now puts us potentially less than 12 months away from definitive unblinded data that could be a strong indicator of our likelihood of approval, and the kind of data that is likely to drive advanced partnering discussions," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

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 the first 75 to 90 subjects will be randomized in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, such doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting. The amended protocol will allow for the unblinding of preliminary primary efficacy data (CR) and safety/tolerability of the three arms at 45 subjects. This early unblinding will yield 30 subjects with Annamycin (190mg/m2 and 230/m2) and HiDAC and 15 subjects with just HiDAC. The Company expects to reach 45 subjects in the second half of 2025, in addition to the planned unblinding expected in 2026 of the next 30-45 subjects.

For Part B of the trial, approximately 244 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 on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative. This increase from 240 to 244 subjects represents the statistical "cost" of the additional unblinding.

The amended protocol is currently being reviewed by the Institutional Review Board (IRB). Once approved, the amended protocol will be filed with the amendment for the Company’s Initial New Drug (IND) application in the US with the FDA.

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).

On November 14, 2024 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that Joseph Romanelli, president, Human Health International, and Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories, are scheduled to participate in a fireside chat at the Jefferies London Healthcare Conference on Thursday, Nov. 21, 2024, at 6:30 a.m. EST / 11:30 a.m. GMT (Press release, Merck & Co, NOV 14, 2024, View Source [SID1234648406]).

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Investors, analysts, members of the media and the general public are invited to listen to a live audio webcast of the presentation at this weblink.

On November 14, 2024 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, and LaNova Medicines Ltd. (LaNova), a privately held clinical-stage biotechnology company, reported that Merck has entered into an exclusive global license to develop, manufacture and commercialize LM-299, a novel investigational PD-1/VEGF bispecific antibody from LaNova (Press release, Merck & Co, NOV 14, 2024, View Source [SID1234648405]).

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"At Merck, we continue to assemble a strong and diversified oncology pipeline spanning differentiated mechanisms and multiple modalities," said Dr. Dean Y. Li, president, Merck Research Laboratories. "This agreement adds to Merck’s growing oncology pipeline and we look forward to advancing LM-299 with speed and rigor for patients in need."

Under the agreement, LaNova has granted Merck an exclusive global license to develop, manufacture and commercialize LM-299. LaNova will receive an upfront payment of $588 million. LaNova is also eligible to receive up to $2.7 billion in milestone payments associated with the technology transfer, development, regulatory approval and commercialization of LM-299 across multiple indications.

"This agreement with Merck is a strong testament to the hard work of LaNova’s talented team of scientists who created LM-299," said Dr. Crystal Qin, founder, chairwoman and chief executive officer, LaNova. "Through internal R&D innovation and strategic external partnerships, LaNova is committed to advancing its pipeline to benefit patients worldwide."

Closing of the proposed transaction is subject to approval under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions. The transaction is expected to close in the fourth quarter of 2024. Merck expects to record a pre-tax charge relating to the $588 million payment due upon closing to be included in GAAP and non-GAAP results in the quarter that the transaction closes, and the EPS impact of such charge will be disclosed at that time.

About LM-299

LM-299 is an investigational bispecific antibody targeting both programmed cell death protein-1 (PD-1) and vascular endothelial growth factor (VEGF). This innovative therapeutic approach is designed to inhibit both PD-1/PD-L1 and VEGF/VEGFR receptor signaling pathways releasing a key immune checkpoint while also inhibiting the production of new blood vessels (angiogenesis). LM-299 has a differentiated molecular design, comprising an anti-VEGF antibody linked to two C-terminal single domain anti-PD-1 antibodies. A Phase 1 clinical trial for LM-299 is currently enrolling patients in China.

On November 14, 2024 Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumors, reported corporate updates and financial results for the third quarter ended September 30, 2024 (Press release, Marker Therapeutics, NOV 14, 2024, View Source [SID1234648404]).

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"As we approach the end of 2024, we continue to build momentum across our clinical and corporate programs," said Juan Vera, M.D., President and Chief Executive Officer of Marker Therapeutics. "During the third quarter, we made significant progress in our ongoing Phase 1 APOLLO study investigating MT-601 in patients with lymphoma who have relapsed after anti-CD19 chimeric antigen receptor (CAR)-T cell therapy or where CAR-T cells are not an option. Enrollment is ongoing and we expect to be able to report preliminary safety and efficacy data by the end of this year. We, along with our study investigators, have been encouraged by the promising activity shown in this platform and look forward to continuing assessments from the trial."

"We also recently strengthened our financial position with the receipt of two, $2 million Small Business Innovation Research (SBIR) grants from the National Institutes of Health (NIH). The non-dilutive funding will be used to support further development of MT-601 in patients with non-Hodgkin’s lymphoma who have relapsed following anti-CD19 CAR-T cell therapy, as well as assist in funding the clinical investigation of MT-601 in pancreatic cancer. We continue to make meaningful progress and remain focused on execution as we move our programs through the clinical trials," added Dr. Vera.

PROGRAM UPDATES & EXPECTED MILESTONES

MT-601 (Lymphoma)

- Marker’s lead program is investigating MT-601 in the nationwide multicenter Phase 1 APOLLO study (clinicaltrials.gov identifier: NCT05798897) in patients with lymphoma who have relapsed after anti-CD19 CAR-T cell therapy or where CAR-T cells are not an option.

- The Company previously reported that one of the Principal Investigators presented preliminary safety and efficacy with sustained objective responses observed in three study participants treated at City of Hope National Medical Center (Press Release, April 8, 2024). Treatment was well tolerated among all study participants with no observation of severe adverse effects such as immune effector cell associated neurotoxicity syndrome (ICANS).

- All study participants continue to be observed for long-term treatment effects and durability of response.

- The Company is enrolling additional study participants in the Phase 1 APOLLO trial and expects to provide an update on safety and durability during the fourth quarter of 2024.

- Marker Therapeutics was awarded a $2 million grant from NIH Small Business Innovation Research Program (SBIR) to support the clinical investigation of MT-601 in patients with lymphoma who have relapsed following anti-CD19 CAR-T cell therapy (Press Release, August 12, 2024).

MT-601 (Pancreatic)

- The Company was awarded a $2 million grant from NIH Small Business Innovation Research (SBIR) Program to support the clinical investigation of MT-601 in patients with metastatic pancreatic cancer.

- The Company expects to start the clinical program of MT-601 in patients with metastatic pancreatic cancer in 2025.

MT-401-OTS (Acute Myeloid Leukemia or Myelodysplastic Syndrome)

- The Company previously secured non-dilutive funding to support the clinical investigation of MT-401-OTS in patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) and anticipates clinical program initiation during the first half of 2025.

THIRD QUARTER 2024 FINANCIAL HIGHLIGHTS

Cash Position and Guidance: At September 30, 2024, Marker had cash and cash equivalents of $9 million. The Company believes that its existing cash and cash equivalents will fund its operating expenses into October 2025. This estimate is subject to the Company’s ability to effectively manage its costs and utilize drawdowns of available grant funds.

R&D Expenses: Research and development expenses were $3.5 million for the quarter ended September 30, 2024, compared to $2.0 million for the quarter ended September 30, 2023 as a result of our increased clinical trial activity in the quarter.

G&A Expenses: General and administrative expenses were $0.9 million for the quarter ended September 30, 2024, compared to $1.4 million for the quarter ended September 30, 2023 reflecting the savings from the reorganization that was completed in late 2023.

Net Loss: Marker reported a net loss from continuing operations of $2.3 million for the quarter ended September 30, 2024, compared to $3.0 million for the quarter ended September 30, 2023.

About multiTAA-specific T cells

The multi-tumor associated antigen (multiTAA)-specific T cell platform is a novel, non-genetically modified cell therapy approach that selectively expands tumor-specific T cells from a patient’s/donor’s blood capable of recognizing a broad range of tumor antigens. Unlike other T cell therapies, multiTAA-specific T cells allow the recognition of hundreds of different epitopes within up to six tumor-specific antigens, thereby reducing the possibility of tumor escape. Since multiTAA-specific T cells are not genetically engineered, Marker believes that its product candidates will be easier and less expensive to manufacture, with an improved safety profile, compared to current engineered T cell approaches, and may provide patients with meaningful clinical benefits.

On November 14, 2024 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported its third quarter 2024 financial and operating results (Press release, Lineage Cell Therapeutics, NOV 14, 2024, View Source [SID1234648403]). The Company will host a conference call today at 4:30 p.m. Eastern Time to discuss these results and to provide a business update.

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"We were delighted to see our partners’ continued commitment to the OpRegen program, in this instance by seeking and successfully obtaining RMAT designation," stated Brian M. Culley, Lineage CEO. "We believe OpRegen continues to showcase itself as an asset with the potential to be ‘a transformational medicine’ and view the recent RMAT designation as additional positive progress for this pioneering cell transplant program. As we worked to return our second cell transplant program, OPC1 for spinal cord injury, back into the clinic, we also presented promising preclinical results from our third program, ReSonance, for sensorineural hearing loss. We look forward to continuing to create value through the advancement of our clinical and preclinical pipelines, applying both our technology and extensive manufacturing expertise to validate our cell transplant approach."

Recent Operational Highlights
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RG6501 (OpRegen)
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Roche and Genentech, a member of the Roche Group, announced receipt of RMAT designation from the U.S. FDA for OpRegen, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
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Continued execution under our collaboration with Roche and Genentech across multiple functional areas, including support for the ongoing Phase 2a clinical study (the "GAlette Study") in patients with GA secondary to AMD.
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Continued activities under the separate services agreement with Genentech to support ongoing development of OpRegen. Lineage has been providing additional clinical, technical, training and manufacturing services funded by Genentech, that further support the ongoing advancement and optimization of the OpRegen program and include: (i) activities to support the ongoing Phase 1/2a study and currently-enrolling Phase 2a study; and (ii) additional technical training and materials related to Lineage’s cell therapy technology platform to support commercial manufacturing strategies.

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OPC1
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DOSED (Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device) clinical study for the treatment of subacute and chronic spinal cord patient start-up activities and FDA interactions continue.

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ReSonance (ANP1)
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Preclinical results presented at 59th Annual Inner Ear Biology Workshop
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ReSonance manufactured by a proprietary process, developed in-house, at clinical scale, with relevant in-vitro functional activity

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Immediate-use, thaw-and-inject formulation durably engrafted in multiple preclinical hearing loss models
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ReSonance is currently being evaluated in a functional model of hearing loss through a collaboration with the University of Michigan Kresge Hearing Research Institute.

Balance Sheet Highlights

Cash, cash equivalents, and marketable securities of $32.7 million as of September 30, 2024 is expected to support planned operations into Q1 2026.

Third Quarter Operating Results

Revenues: Revenue is generated primarily from collaboration revenues, royalties, and other revenues. Total revenues for the three months ended September 30, 2024 were $3.8 million, a net increase of approximately $2.5 million as compared to approximately $1.2 million for the same period in 2023. The increase was primarily driven by more collaboration revenue recognized from deferred revenues under the collaboration and license agreement with Roche.

Operating Expenses: Operating expenses are comprised of research and development (R&D) expenses and general and administrative (G&A) expenses. Total operating expenses for the three months ended September 30, 2024 were $7.6 million, a decrease of $0.3 million as compared to $7.9 million for the same period in 2023.

R&D Expenses: R&D expenses for the three months ended September 30, 2024 were $3.2 million, a net decrease of approximately $0.6 million as compared to $3.7 million for the same period in 2023. The net decrease was primarily driven by $0.6 million for our OPC1 program, $0.4 million for our preclinical programs, and partially offset by $0.5 million for our OpRegen program.

G&A Expenses: G&A expenses for the three months ended September 30, 2024 were $4.4 million, a net increase of $0.4 million as compared to $4.0 million for the same period in 2023. The net increase was primarily driven by $0.3 million for personnel costs and $0.1 million for stock-based compensation expense.

Loss from Operations: Loss from operations for the three months ended September 30, 2024 were $3.8 million, a decrease of $2.9 million as compared to $6.7 million for the same period in 2023.

Other Income/(Expenses): Other income (expenses) for the three months ended September 30, 2024 reflected other income of $0.8 million, compared to other expenses of approximately ($0.4) million for the same period in 2023. The change was primarily driven by exchange rate fluctuations related to our international subsidiaries.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended September 30, 2024 was $3.0 million, or $0.02 per share (basic and diluted), compared to a net loss attributable to Lineage of $7.1 million, or $0.04 per share (basic and diluted), for the same period in 2023.

Conference Call and Webcast

Interested parties may access the conference call on November 14th, 2024, by dialing (800) 715-9871 from the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through November 21st, 2024, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 2238934.