Allogene Therapeutics Announces Participation in March Investor Conference

On February 26, 2025 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer and autoimmune disease, reported that it will participate in one upcoming investor conference in March (Press release, Allogene, FEB 26, 2025, View Source [SID1234650621]).

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TD Cowen 45th Annual Health Care Conference
Wednesday, March 5, 2025
8:20AM PT/11:50AM ET

Any available webcasts will be posted to the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following a live webcast, a replay will be available on the Company’s website for approximately 30 days.

Alligator Bioscience Announces Continued Encouraging Overall Survival Benefit for Mitazalimab

On February 26, 2025 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported updated results from the Phase 2 OPTIMIZE-1 study evaluating mitazalimab in combination with standard chemotherapy (mFOLFIRINOX) as a first-line treatment for metastatic pancreatic cancer (Press release, Alligator Bioscience, FEB 26, 2025, View Source [SID1234650617]). The 24-month analysis of the 900 µg/kg dose reconfirms and extends the significant survival benefit and reinforces the potential of mitazalimab as a breakthrough immunotherapy candidate in this challenging disease.

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The data demonstrated a 24-month survival rate of 29.4% in patients treated with mitazalimab in combination with mFOLFIRINOX. These numbers compare well to estimates of 24-month survival rates of 8% for FOLFIRINOX alone, and 20% for NALIRIFOX alone¹,².

Median follow-up duration for the 24-month analysis was 25.4 months, indicating the maturity of these outcomes. At the time of analysis, a total of 16 (28%) patients were still alive, and of these, 5 (9%) were still on treatment. The longest ongoing treatment duration was 32 months.

Median Overall Survival (mOS) in the study was 14.9 months, comparing favourably to the 11.1 months demonstrated by FOLFIRINOX¹ and more recently by NALIRIFOX². The duration of response was confirmed at 12.6 months, as compared to 5.9 and 7.3 months reported for standard of care¹,².

Hence, the results show that patients responding to mitazalimab in combination with mFOLFIRINOX continue to show stable and encouraging survival outcomes over time. This highlights the consistency of mitazalimab’s therapeutic effect and underscores the durable efficacy of mitazalimab when paired with standard treatment.

In addition, the analysis provided top-line 6-month follow-up data from the 450 µg/kg dose cohort conducted to meet FDA’s request for further dose characterization prior to advancing mitazalimab into Phase 3. This data showed an objective response rate of 22.7% (unconfirmed), compared to 54.4% for the 900 µg/kg dose. These findings indicate a dose-response relationship for mitazalimab and support the selection of 900 µg/kg as recommended Phase 3 dose.

"These 24-month data further validate mitazalimab’s potential to meaningfully impact treatment outcomes for pancreatic cancer patients, and the continued survival and response duration observed in the study reinforce our confidence in mitazalimab’s clinical promise and its potential to reshape the treatment landscape for this aggressive disease" said Søren Bregenholt, CEO of Alligator Bioscience. "Moreover the 450 µg/kg topline data suggest a positive dose-response correlation, further strengthening the rationale for developing mitazalimab at the 900 µg/kg dose."
Based on these results and a confirmed regulatory path, Alligator Bioscience remains on track in advancing mitazalimab toward confirmatory clinical trials. With mitazalimab demonstrating strong clinical efficacy, durability of response, and a clear regulatory pathway forward, Alligator Bioscience continues to actively explore strategic collaborations to accelerate late-stage development and maximize the potential of mitazalimab as a transformative treatment option for pancreatic cancer.

Alector Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update

On February 26, 2025 Alector, Inc. (Nasdaq: ALEC), a late-stage clinical biotechnology company focused on developing therapies to counteract the devastating progression of neurodegeneration, reported fourth quarter and full year 2024 financial results and recent portfolio and business updates (Press release, Alector, FEB 26, 2025, View Source [SID1234650618]). As of December 31, 2024, Alector’s cash, cash equivalents and investments totaled $413.4 million.

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"Alector is entering a potentially transformative period for the company, as we continue to advance our clinical programs toward key milestones while also driving the development of our wholly owned, early-stage pipeline," said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. "We look forward to reporting topline data from the pivotal INFRONT-3 Phase 3 trial of latozinemab targeting frontotemporal dementia with a granulin gene mutation by the fourth quarter. Additionally, we expect to complete enrollment in the PROGRESS-AD Phase 2 trial of AL101/GSK4527226 in participants with early Alzheimer’s disease by mid-2025. With our broad and diverse portfolio of genetically validated drug candidates for the treatment of neurodegenerative diseases, backed by a strong financial position that will fund operations through 2026, Alector is well-positioned to advance our mission to deliver transformative therapies for patients with neurodegenerative brain disorders."

Sara Kenkare-Mitra, Ph.D., President and Head of Research and Development at Alector, added, "We are making significant progress in advancing Alector’s preclinical and research pipeline. By leveraging our expertise in neuroscience and drug discovery, and selectively applying our proprietary Alector Brain Carrier technology platform, we are able to enhance brain delivery and biodistribution of therapeutic cargo to potentially improve safety and efficacy. We are currently selecting lead candidates for two programs, ADP037-ABC, targeting amyloid beta, and ADP050-ABC, replacing GCase. We aim to advance these programs toward IND-enabling studies later this year, with plans to enter the clinic in 2026."

Recent Clinical Updates

Progranulin Programs (latozinemab (AL001) and AL101/GSK4527226) Being Developed in Collaboration with GSK

Latozinemab

The pivotal, randomized, double-blind, placebo-controlled INFRONT-3 Phase 3 clinical trial of latozinemab targeting frontotemporal dementia with a granulin gene mutation (FTD-GRN) is ongoing. Topline data are anticipated by the fourth quarter of 2025.
Latozinemab is a novel investigational human monoclonal antibody (mAb) designed to block and downregulate the sortilin receptor to elevate the level of progranulin (PGRN) in the brain. It has been granted Orphan Drug, Breakthrough Therapy and Fast Track designations, and the company believes it is the most advanced PGRN-elevating candidate in development for this condition.
AL101/GSK4527226

PROGRESS-AD, a global, randomized, double-blind, placebo-controlled Phase 2 clinical trial evaluating AL101/GSK4527226 in early Alzheimer’s disease (AD) continues to enroll well, with substantial progress toward its target enrollment of 282 participants. Alector and GSK plan to complete trial enrollment by mid-2025.
AL101/GSK4527226 is an investigational human mAb designed to block and downregulate the sortilin receptor to elevate the level of PGRN in the brain in a manner that is similar to investigational latozinemab but with different pharmacokinetic and pharmacodynamic properties, making it suitable for the potential treatment of more prevalent neurodegenerative diseases.
Preclinical and Research Pipeline

Alector is advancing a preclinical and early research pipeline focused on removing toxic proteins, replacing deficient proteins, and restoring immune and nerve cell function. Leveraging its deep expertise in drug development and proprietary technologies, including protein engineering, antibody discovery, and its Alector Brain Carrier (ABC) platform for blood-brain barrier transport, the company is progressing a curated portfolio of programs targeting genetically validated disease mechanisms. This approach has led to a suite of innovative programs with the potential to make a significant impact on neurodegeneration.

ADP037-ABC is a proprietary anti-amyloid beta (Aβ) antibody paired with the company’s ABC for the treatment of AD. It is designed to remove brain Aβ plaques, with the potential to reduce the incidence and/or severity of amyloid-related imaging abnormalities (ARIA) and enable subcutaneous delivery. It targets a validated epitope, specific to brain Aβ plaques, combined with an optimized antibody constant region to enhance phagocytosis of Aβ plaques. By leveraging ABC technology, ADP037-ABC aims to clear Aβ efficiently, thereby reducing plaque accumulation and potentially slowing disease progression while minimizing ARIA.
ADP050-ABC is a GCase replacement therapy paired with the company’s proprietary ABC for GBA gene mutation carriers with Parkinson’s disease (PD) and Lewy body dementia. In these patients, mutations in the GBA gene lead to deficient GCase activity. ADP050-ABC uses Alector-engineered GCase, which is proprietary and has been designed to have a longer half-life and to break down glucocerebroside, a lipid that accumulates in neurons and contributes to neurodegeneration. This mechanism aims to reduce cellular dysfunction and slow disease progression.
ADP056 is a Reelin modulator designed to block tau pathology and promote synaptic function in AD. Reelin, a large, secreted protein, regulates neuronal function and tau accumulation. Gain-of-function Reelin variants protect against familial AD through a mechanism that appears to uncouple amyloid and tau pathology. ADP056 is designed to mimic these protective effects of the Reelin mutation.
ADP063-ABC and ADP064-ABC are therapeutic candidates paired with the company’s proprietary ABC that target tau pathology in AD through distinct approaches. First, ADP063-ABC will focus on combining a proprietary anti-tau antibody directed to a validated tau epitope with ABC and an optimized antibody constant region. It is designed to block the spread of tau aggregates and has the potential for subcutaneous delivery. Second, ADP064-ABC will focus on using an anti-tau siRNA combined with ABC, which aims to prevent the synthesis of the tau mRNA and protein. Both approaches seek to leverage a highly brain-penetrant approach to remove toxic tau and potentially slow cognitive decline in AD.
In December 2024, Alector and co-recipient University of Luxembourg were awarded a $1.7 million grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) for collaborative research on glycoprotein nonmetastatic melanoma protein B (GPNMB), a PD target.
Alector plans to hold an educational webinar in the second quarter of 2025 to present additional preclinical data on the company’s anti-amyloid beta and GCase programs, as well as advancements in ABC.
TREM2 Program (AL002)

On April 5, 2025, Alector plans to present the results from the INVOKE-2 Phase 2 clinical trial, which evaluated the safety and efficacy of AL002, a TREM2 agonist, in individuals with early AD, during an oral presentation at the AD/PD 2025 International Conference on Alzheimer’s and Parkinson’s Diseases, taking place in Vienna, Austria. While the trial did not meet its primary endpoint, Alector remains committed to advancing the understanding of AD pathophysiology and the development of effective therapeutics for the disease.
Fourth Quarter 2024 Financial Results

Revenue. Collaboration revenue for the quarter ended December 31, 2024, was $54.2 million, compared to $15.2 million for the same period in 2023. Collaboration revenue for the year ended December 31, 2024, was $100.6 million, compared to $97.1 million for the same period in 2023. The increase in year-over-year collaborative revenue was primarily due to an increase in revenue recognized for the AL002 and latozinemab programs. Collaboration revenue under the AbbVie Agreement was fully recognized as of December 31, 2024.

R&D Expenses. Total research and development expenses for the quarter ended December 31, 2024, were $46.5 million, compared to $47.7 million for the quarter ended December 31, 2023. Total research and development expenses for the year ended December 31, 2024, were $185.9 million compared to $192.1 million for the same period in 2023. The decrease in year-over-year R&D expenses was mainly due to the Company’s strategy to prioritize selected programs.

G&A Expenses. Total general and administrative expenses for the quarter ended December 31, 2024, were $15.0 million compared to $14.9 million for the quarter ended December 31, 2023. Total general and administrative expenses for the year ended December 31, 2024, were $59.6 million compared to $56.7 million for the year ended December 31, 2023. The increase in year-over-year G&A expenses is primarily due to the impairment of the right-of-use asset and the leasehold improvements as the Company approved a plan to transition operations from our laboratory and offices in Newark, California to our South San Francisco Headquarters.

Net Loss. For the quarter ended December 31, 2024, Alector reported a net loss of $2.1 million, or $0.02 net loss per share, compared to a net loss of $41.4 million, or $0.49 net loss per share, for the same period in 2023. For the year ended December 31, 2024, Alector reported a net loss of $119.0 million or $1.23 net loss per share, compared to a net loss of $130.4 million or $1.56 net loss per share, for the same period in 2023.

Cash Position. Cash, cash equivalents, and investments were $413.4 million as of December 31, 2024. Management expects that this will be sufficient to fund current operations through 2026.

2025 Guidance. Management anticipates, for the year ending 2025, collaboration revenue to be between $5 million and $15 million, total research and development expenses to be between $175 million and $185 million, and total general and administrative expenses to be between $55 million and $65 million.

Fourth Quarter and Full Year 2024 Conference Call

Alector’s management team will host a conference call to discuss Alector’s results for the fourth quarter and full year 2024, as well as provide a business update. The conference call will be webcast and accessible via the investor relations section of Alector’s website at www.alector.com.

To access the call, please use the following information:

Date: Wednesday, February 26, 2025
Time: 4:30 p.m. ET, 1:30 p.m. PT

The event will be webcast live under the investor relations section of Alector’s website at View Source and following the event a replay will be archived there for 30 days. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.

Adcendo ApS Announces U.S. FDA Clearance of IND Application for Phase I Tiffany-01 Trial of ADCE-T02

On February 26, 2025 Adcendo, a biotech company pioneering the development of first and best-in-class ADCs for cancers with a high unmet medical need, reported that the U.S. Food & Drug Administration (FDA) has provided clearance of the IND application for a Phase I study evaluating ADCE-T02 in patients with advanced solid tumors (Press release, ADCendo, FEB 26, 2025, View Source [SID1234650619]).

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Tiffany-01 is an ongoing first-in-human Phase I multicenter, open-label, dose escalation study of ADCET02 as a monotherapy in patients with advanced solid tumors. The primary objectives of the study are to determine the maximum tolerated dose and recommended Phase II dose and schedule of ADCET02 monotherapy in addition to assessing ADCE-T02 safety and tolerability. The secondary objectives are to characterize the pharmacokinetics and to evaluate the preliminary efficacy of ADCE-T02. The study is currently recruiting in Australia and will start recruiting in the U.S. in the next few months.

"Tissue factor (TF) is a validated ADC target with overexpression in many high unmet need solid tumor indications, however, the currently approved TF targeting ADC has severe limitations due to a suboptimal side effect profile and a limited therapeutic window. The highly differentiated profile of ADCE-T02, based on the use of an improved monoclonal antibody and a next generation Topoisomerase I inhibitor linker/payload technology, could overcome those limitations and offer an enhanced therapeutic window and improved side effect profile, which may lead to better clinical outcomes for patients," said Dr. Lone Ottesen, Chief Medical Officer of Adcendo. "The U.S. IND clearance of ADCE-T02 is an important milestone for our program and our company, and we look forward to initiating patient enrollment in the U.S. and working closely with all of our investigators to evaluate the therapeutic utility of this drug in multiple advanced solid tumor indications."

Prof. Vinod Ganju. Managing Director of Peninsula and Southeast Oncology (PASO), Melbourne, Australia and Principal Investigator of Tiffany-01, commented: "ADCs have in the past years shown highly encouraging results and have already become Standard of Care in quite a number of solid tumor indications. ADCE-T02 represents an attractive new option to explore in advanced solid tumors with high unmet need. I am pleased to be working with Adcendo to develop ADCE-T02, potentially offering a broader therapeutic window and a better safety profile for our patients."

About ADCE-T02

Tissue Factor is a clinically validated ADC target with strong overexpression in multiple tumor indications with high unmet medical need including Cervical Cancer, Head and Neck Cancer, Colorectal Cancer, Non-Small Cell Lung Cancer, and Pancreatic Ductal Adenocarcinoma. ADCE-T02 is a highly differentiated anti-TF ADC, and the first ADC with a Topoisomerase I inhibitor-based linker/payload, clinically developed in Australia and the U.S. Its unique antibody design minimizes the impact on the coagulation pathway, while the T1000-exatecan linker-payload technology platform has been shown to amplify the bystander effect, improve linker stability, and has the potential to overcome emerging resistance mechanisms. These differentiated features are expected to translate into a superior therapeutic window, a better safety profile, enhanced response rates and longer duration of response.

Interim Report and Half-Year Financial Results

On February 26, 2025 Starpharma (ASX: SPL, US OTC: SPHRY), an innovative biotechnology company with two decades of experience in advancing dendrimer technology from the lab to the patient, reported its Interim Report and Half-Year Financial Results for the period ended 31 December 2024 (H1 FY25) (Press release, Starpharma, FEB 26, 2025, View Source;mc_eid=bf52dd3418 [SID1234650550]).

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Starpharma’s Chief Executive Officer, Cheryl Maley, commented:

"In the first half of FY25, Starpharma has made good progress in executing our strategy, aimed at maximising the value of our DEP assets, accelerating early asset development, and building long-term sustainability. I know our internal progress may not always be evident externally, but I can vouch for the dedication and hard work of everyone at Starpharma as we work towards achieving our strategic objectives.

"We have made important progress in advancing our DEP clinical assets, particularly DEP SN38, which has shown promising clinical outcomes in patients with high unmet need. A key regulatory milestone during the period was the recent meeting with the US Food and Drug Administration (FDA) regarding DEP SN38. The FDA provided feedback on the path to market for DEP SN38, confirming that the 505(b)(2) regulatory approval pathway is appropriate for DEP SN38 and the potential for Fast Track designation and accelerated approval. The positive response from the US regulator increases our confidence in the potential of DEP SN38 for treating platinum-resistant ovarian cancer. During the six months to December, our partner engagement through ongoing meetings and conference participation highlighted the importance of FDA feedback to potential partners for the commercialisation of DEP SN38.

"We are advancing our early-stage DEP programs, particularly in the area of radiopharmaceuticals, with the aim of initiating a first-in-patient clinical trial this calendar year. We are also expanding our research pipeline, positioning us well for future collaborations and long-term growth. Commercially, we anticipate the launch of VivaGel BV in key markets in the Middle East and continue to support sales of Viraleze online through digital marketing initiatives, which have resulted in a ~30% increase in revenue from the e-commerce channels compared to the prior corresponding period.

"We have focused on building a sustainable organisation and have sufficient capital to support our medium-term objectives, with a cash balance of $20.3 million as at 31 December 2024. The completion of multiple clinical programs in FY24 has led to a reduction in our research and development expenses, extending our cash runway. Over the past 12 to 18 months, we have implemented a number of cost-saving initiatives that are positively impacting our cash balance. Notably, our Corporate and Administration costs in this half-year period have decreased by $0.5 million compared to the prior corresponding period.

"Starpharma is committed to driving revenue growth, advancing our pipeline, and managing costs effectively to deliver improved and long-term value for our shareholders. We are focused on prioritising resources for our high-impact DEP programs, pursuing partnerships to advance our assets toward commercialisation, and increasing revenue through collaborations, licensing and product sales."