On February 27, 2025 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the fourth quarter and full year ended 2024 and provided a corporate update (Press release, Aclaris Therapeutics, FEB 27, 2025, View Source [SID1234650685]).

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"2024 was a transformative year that has positioned Aclaris with multiple clinical catalysts expected in 2025 across our expanded pipeline," stated Dr. Neal Walker, Chief Executive Officer and Chair of the Board of Directors of Aclaris. "We are particularly excited about the upcoming Phase 2 data for bosakitug in both severe asthma and chronic rhinosinusitis with nasal polyps anticipated from our partner CTTQ, which we expect will provide important insights into our future development of bosakitug in respiratory diseases. We also anticipate top-line data from our Phase 2a trial of ATI-2138 in atopic dermatitis in the first half of 2025. With multiple clinical catalysts expected throughout 2025 across our pipeline of differentiated assets with mechanisms shown to have proven activity in the diseases we are addressing, we look to drive continued innovation for the patients we seek to treat."

Fourth Quarter 2024 Highlights and Recent Updates

Pipeline:

Announced Exclusive, Global License Agreement with Biosion, Inc., Adding Potential Best-in-Class Biologic Assets to Pipeline: Aclaris acquired worldwide rights (excluding Greater China) to bosakitug (ATI-045), a potential best-in-class, clinical-stage, novel anti-TSLP monoclonal antibody, and ATI-052, a potential best-in-class, pre-clinical stage, novel bispecific antibody that is directed against both TSLP and IL4R. As a result of this license agreement, the Company recorded a one-time $86.9 million in-process research and development charge. (press release here)
Confirmed Expectation of Phase 2 Data in the First Half of 2025 for Bosakitug in Chinese Patients with Certain Pulmonary Disorders: Aclaris’ regional partner, Chia Tai Tianqing Pharmaceutical Group, Co., Ltd. (CTTQ), is conducting concurrent Phase 2 studies in China for patients with severe asthma, CRSwNP, and chronic obstructive pulmonary disease. Data from trials in severe asthma and CRSwNP expected in first half of 2025 to inform internal development programs.
Initiated Clinical Trial Activities for a Phase 2b Trial of Bosakitug in Atopic Dermatitis (AD); Enrollment Expected to Begin in the First Half of 2025: This trial will investigate the safety, tolerability, pharmacokinetics, efficacy, and pharmacodynamics of bosakitug in patients with moderate to severe AD.
Confirmed Expectation of Top Line Results in the First Half of 2025 for Phase 2a Trial in AD of ATI-2138, an Investigational Oral Covalent ITK/JAK3 Inhibitor: This ongoing Phase 2a open-label trial is being conducted to investigate the safety, tolerability, pharmacokinetics, efficacy, and pharmacodynamics of ATI-2138 in patients with moderate to severe AD.
Announced Plan to File an Investigational New Drug (IND) Application for ATI-052 in the First Quarter of 2025: Following allowance of the IND, Aclaris expects to initiate a Phase 1 clinical trial evaluating single ascending doses and multiple ascending doses of ATI-052.
Announced New Publication Highlighting the Unique Properties of ATI-2138: New publication provides important clinical and non-clinical evidence of the potential for ATI-2138 to be a best-in-class inhibitor of key signal transduction kinases due to its unique mechanism of action. (press release here)
Corporate:

Completed $80 Million Private Placement in November 2024 to Bolster Cash Runway: Aclaris’ cash runway expected into 2028. (press release here)
Provided Update on Senior Leadership:
Dr. Neal Walker, formerly interim Chief Executive Officer, has been named Chief Executive Officer. Dr. Walker is a co-founder of Aclaris and has served as a member of the Board of Directors since its inception. He previously served as Aclaris’ Chief Executive Officer until 2022 before being appointed as interim Chief Executive Officer in January 2024. Dr. Walker serves as Chair of the Board of Directors of Aclaris.
Hugh Davis, Ph.D. joined Aclaris as President and Chief Operating Officer. Dr. Davis brings over 35 years of experience in biologics development, clinical pharmacology, and business development to Aclaris. He most recently served as Biosion’s Chief Business & Development Officer and President.
William Roberts has been appointed as Senior Vice President, Corporate Communications and Investor Relations. Mr. Roberts brings 30 years of corporate communications, investor relations, and scientific experience in the biotech/biopharma industry to the Company. He most recently served as the Communications Officer of G1 Therapeutics, which was recently acquired by Pharmacosmos Group.
Fourth Quarter and Full Year 2024 Financial Results

As of December 31, 2024, Aclaris had aggregate cash, cash equivalents and marketable securities of $203.9 million compared to $181.9 million as of December 31, 2023. The Company believes that its cash, cash equivalents and marketable securities as of December 31, 2024 will be sufficient to fund its operations into 2028, without giving effect to any potential business development transactions or financing activities.

Net loss was $96.6 million for the fourth quarter of 2024 compared to $1.5 million for the fourth quarter of 2023. Net loss was $132.1 million for the year ended December 31, 2024 compared to $88.5 million for the year ended December 31, 2023.

Total revenue was $9.2 million for the fourth quarter of 2024 compared to $17.6 million for the fourth quarter of 2023. The decrease was primarily driven by a one-time upfront payment under the license agreement with Sun Pharmaceutical Industries, Inc. received in the fourth quarter of 2023, offset by the achievement of a commercial milestone under the license agreement with Eli Lilly and Company in the fourth quarter of 2024. Total revenue was $18.7 million for the year ended December 31, 2024 compared to $31.2 million for the year ended December 31, 2023.

Research and development (R&D) expenses were $9.0 million for the quarter ended December 31, 2024 compared to $26.6 million for the prior year period. The $17.6 million decrease was primarily the result of lower zunsemetinib development expenses, lepzacitinib preclinical and clinical development activities, and compensation-related expenses. For the year ended December 31, 2024, R&D expenses were $33.6 million compared to $98.4 million for the year ended December 31, 2023.

General and administrative (G&A) expenses were $5.0 million for the quarter ended December 31, 2024 compared to $8.2 million for the corresponding prior year period. The decrease was primarily due to a reduction in personnel and stock-based compensation expenses. For the year ended December 31, 2024, G&A expenses were $22.2 compared to $32.4 million for the year ended December 31, 2023, primarily due to lower compensation-related costs.

Licensing expenses were $8.6 million for the quarter ended December 31, 2024 compared to $5.7 million for the prior year period. The increase was primarily attributable to a milestone achieved during the fourth quarter of 2024, the entirety of which was payable to a third party. For the year ended December 31, 2024, licensing expenses were $12.7 million compared to $14.7 million for the year ended December 31, 2023.

Revaluation of contingent consideration resulted in a $1.3 million gain for the quarter ended December 31, 2024 compared to a $26.3 million gain for the prior year period. For the year ended December 31, 2024, revaluation of contingent consideration resulted in a charge of $2.5 million compared to a $26.9 million gain for the year ended December 31, 2023.

During the quarter and year ended December 31, 2024, the Company recorded $86.9 million of in-process research and development expenses, representing the fair value of consideration expensed in connection with the in-license of bosakitug (ATI-045) and ATI-052, as well as transaction costs incurred. During the quarter ended December 31, 2023, the Company recorded an intangible asset impairment charge of $6.6 million representing the full balance of its in-process research and development intangible asset.

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 [SID1234650765]).

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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 longterm 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.

On February 26, 2025 Renovaro Biosciences Inc. (NASDAQ: RENB), a TechBio leader focused on next-generation diagnostics, drug discovery, and genetically enhanced cancer therapies, reported a definitive agreement to merge with BioSymetrics, an artificial intelligence (AI)-driven drug discovery and biomarker identification company (Press release, Renovaro Biosciences, FEB 26, 2025, View Source [SID1234650756]). This transformative partnership is designed to enhance Renovaro’s data repository, biomarker discovery capabilities, accelerate translational research, and bring precision medicine solutions to cancer and other critical disease areas.

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At the core of this collaboration is BioSymetrics’ proprietary Elion platform, a cutting-edge AI and machine learning engine that uncovers complex biological relationships to accelerate the discovery of diagnostics and therapeutics. BioSymetrics’ Phenograph provides a translational engine that maps human clinical signals to prioritized therapeutic targets and is designed to expedite and improve target and biomarker identification and enable patient stratification and drug repurposing. BioSymetrics’ advanced AI in vivo modeling and machine vision systems enable high throughput phenotypic screening, leveraging AI-powered analysis to detect subtle biological responses with unprecedented accuracy. Through in vivo modeling BioSymetrics has amassed an incredible database of proprietary in vivo experimentation, with associated behavioural and morphological analysis. This integrated approach hastens the discovery and validation of transformative therapeutics by bridging computational insights with real-world biological validation, and has enabled platform and analytic partnerships with Janssen, Pfizer, Merck, Supernus Pharma, and Deerfield Cures. By integrating Elion into Renovaro’s workflow, the combined entity aims to streamline the translation of biomarker insights into accelerated discovery timelines, enhancing precision in target identification and improving overall research efficiency, ultimately enabling faster and more effective drug discovery and therapeutic development.

"This merger represents a pivotal step in our mission to diagnose cancer and advance precision medicine," said David Weinstein, CEO of Renovaro. "By combining our expertise in oncology with BioSymetrics’ AI-driven biomarker discovery, we are creating a powerful synergy that will enhance our ability to identify new therapeutic targets, validate diagnostics and accelerate drug development."

BioSymetrics’ AI technology has been instrumental in uncovering novel disease signatures and optimizing precision medicine strategies. Through this merger, Renovaro will gain access to cutting-edge computational tools that enhance the ability to stratify patients, predict treatment responses, and drive more effective therapeutic interventions.

"We are excited to join forces with Renovaro to translate cutting-edge biomarker discoveries into tangible advancements in drug development," said Anthony Iacovone, CEO of BioSymetrics. "Our AI-driven Elion platform is significantly impacting precision medicine and by aligning with Renovaro’s deep expertise in immunotherapy and oncology, we can accelerate the journey from discovery to clinical application."

On February 26, 2025 Argobio and the Institut Pasteur reported the launch of Enodia Therapeutics, a groundbreaking French biotech company dedicated to block and degrade disease-causing proteins for treating cancer, inflammatory diseases and viral infections (Press release, Argobio, FEB 26, 2025, View Source [SID1234650658]). "With the convergence of an unprecedented diverse set of small molecules inhibitors of the Sec61/translocon molecular complex and advancements in generative artificial intelligence (AI), our vision is to develop novel small molecules drugs with good pharmaceuticals properties that could overcome the most critical limitations of today’s medicines," said Yves Ribeill, CEO of Enodia Therapeutics.

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Enodia Therapeutics stems from pioneering research at the Institut Pasteur, with the discovery of the mechanism of action of Mycolactone, a natural Sec61 inhibitor responsible for Buruli Ulcers. "Our infectious diseases research revealed a new way to target disease-causing proteins. This breakthrough highlights how fundamental research can help uncover entirely new therapeutic opportunities," said Pr. Caroline Demangel, co-founder of Enodia Therapeutics and Head of the Immunobiology and Therapy Unit at the Institut Pasteur.

Rooted in groundbreaking research at the Institut Pasteur and supported by Argobio, Enodia Therapeutics received Pfizer x BioLabs’ prestigious Golden Ticket Award. "This recognition supports our newest small molecule Targeted Protein Degrader (TPD) developed by the combined teams of Argobio and the Institut Pasteur. We are grateful for Pfizer’s support and proud to be part of the vibrant BioLabs life sciences ecosystem in Paris Hotel Dieu, where collaboration and innovation are driving significant advancements in healthcare," Yves Ribeill remarked.

"Pfizer is committed to supporting innovative biotech startups that have the potential to redefine patient care. We are excited to see how Enodia Therapeutics will harness its technology for novel treatment approaches," said Dr. Luca Mollo, Vice-President and Medical Director at Pfizer in France.

Enodia Therapeutics was incubated by Argobio, a premier French biotech studio company dedicated to fostering innovative science and accelerating its transition into a fully-fledged biotech company. The scientific project behind Enodia Therapeutics was supported by the Institut Pasteur’s Innovation Accelerator. The aim of this program is to accelerate the market launch of innovative products resulting from laboratory discoveries. With the creation of its first spin-off company in 1997, the Institut Pasteur has been a pioneer in the creation of spin-off companies based on its research. To date the Institut Pasteur has been involved in the development of 38 spin-off companies.

On February 26, 2025 MAIA Biotechnology, Inc., (NYSE American: MAIA) ("MAIA," the "Company"), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, reported the trial design for the expansion of its THIO-101 pivotal Phase 2 trial in non-small cell lung cancer (NSCLC) (Press release, MAIA Biotechnology, FEB 26, 2025, View Source [SID1234650657]). Following successful outcomes to date in THIO-101, the expansion of the study will assess overall response rates (ORR) in advanced NSCLC patients receiving third line (3L) therapy who were resistant to previous checkpoint inhibitor treatments (CPI) and chemotherapy.

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The THIO-101 study in 3L will enroll up to 48 patients with two arms: Arm 1, continuing the evaluation of THIO sequenced with Libtayo (cemiplimab); and Arm 2, evaluating THIO as a monotherapy, to further gain experience of THIO in the contribution of components. Treatment cycles for patients in both arms will administer THIO on 3 consecutive days, followed by immune activation on day 4. Arm 1 will administer Libtayo on day 5. The Company plans to enroll an additional 100 patients for the registration phase of the trial. MAIA expects to conduct the trials in the U.S. and select countries in Europe and Asia.

MAIA recently announced an amended clinical supply agreement with Regeneron to include the expansion portion of THIO-101. Under terms of the amended agreement, MAIA continues to sponsor THIO-101 and Regeneron will provide Libtayo for the treatment of all patients including the additional patients in the expansion and potentially, the registration studies.

"We are excited to start the expansion arm of our THIO-101 trial which is designed to determine overall response rates in third line NSCLC. We expect to have new patients enrolled in the coming weeks," said Vlad Vitoc, M.D., CEO of MAIA. "Through THIO-101 to date, THIO has delivered unprecedented disease control, response, and survival results. Continued efficacy and safety data generated by our study could support an FDA NDA submission directly, particularly as we plan to seek an accelerated approval of THIO in the U.S.

"We have multiple milestones that we believe are attainable for 2025 and we look forward to keeping our shareholders and investors well informed of our progress on value creation," Dr. Vitoc added.

As of January 15, 2025, data indicated that Median Overall Survival (OS) in third-line treatment was reached at 16.9 months, with a 95% confidence interval (CI) lower bound of 12.5 months and a 99% CI lower bound of 10.8 months). The treatment has been generally well-tolerated to date in this heavily pre-treated population1.

Details on safety can be found on the previously announced SITC (Free SITC Whitepaper) 2024 presentation available on MAIA’s website.

About THIO
THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

About THIO-101, a Phase 2 Clinical Trial
THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. It is the first trial designed to evaluate THIO’s anti-tumor activity when followed by PD-(L)1 inhibition. The trial is testing the hypothesis that low doses of THIO administered prior to cemiplimab (Libtayo) will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of THIO administered as an anticancer compound and a priming immune activator (2) to assess the clinical efficacy of THIO using Overall Response Rate (ORR) as the primary clinical endpoint. Treatment with THIO followed by cemiplimab (Libtayo) has been generally well-tolerated to date in a heavily pre-treated population. For more information on this Phase II trial, please visit ClinicalTrials.gov using the identifier NCT05208944.