On September 17, 2025 ProteinQure, a Toronto-based biotech company pioneering computational peptide drug discovery, reported the successful dosing of the first patient in its Phase I clinical trial evaluating PQ203 – a novel, rationally designed peptide therapeutic for advanced metastatic solid tumors (Press release, ProteinQure, SEP 17, 2025, View Source [SID1234656043]).

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"Dosing our first patient with PQ203 represents a defining moment not just for ProteinQure, but for the vision from the founding of the company — that computational tools can unlock new medicines," said Lucas Siow, CEO and Co-Founder of ProteinQure. "Having created PQ203 in Toronto and starting the trial with the exceptional clinical team at Princess Margaret underscores the ability to advance world-class science in Canada."

The trial is being started at Princess Margaret Cancer Centre in Toronto, one of the world’s top five cancer research centers. There, the trial is led by investigator Dr. Philippe Bedard,

"It’s exciting to be part of a groundbreaking program that was discovered using the latest in computational science," said Dr. Bedard. "Peptides open up a new and promising path for targeted cancer treatments, and we’re looking forward to seeing how PQ203 might help patients who currently have limited treatment options."

About the Trial

The Phase 1 trial is a first-in-human trial with three parts: dose escalation, dose expansion (in multiple tumor types), and dose optimization. The study will evaluate the safety, tolerability, pharmacokinetics, preliminary activity, and pharmacodynamics of PQ203. It will take place across Canada and the US at clinical sites including Princess Margaret Cancer Centre, McGill, Yale, MD Anderson, and Next Oncology.

For more information about the PQ203 trial, visit www.clinicaltrials.gov (NCT# pending) or contact [email protected].

About PQ203

PQ203 is the company’s first internally owned AI designed peptide therapeutic entering the clinic 3 years after the program was started. PQ203 is composed of a peptide targeting the Sortilin receptor conjugated to the cytotoxic agent MMAE. The Sortilin receptor is expressed in a high percentage of diseased tissue from Triple Negative Breast Cancer (TNBC) patients. ProteinQure has generated data that PQ203 exhibits potent efficacy in a patient-derived xenograft (PDX) model resistant to Sacituzumab Govitecan (Trodelvy), an antibody drug conjugate that is the standard of care for metastatic TNBC.

On September 17, 2025 Purdue Pharma L.P. ("Purdue") reported that it has entered into an agreement to have its pipeline medication tinostamustine included in the Phase 2/3 GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) adaptive clinical trial for glioblastoma patients led by the Global Coalition for Adaptive Research (GCAR) (Press release, Purdue Pharma, SEP 17, 2025, View Source [SID1234656046]).

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Tinostamustine is a first-in-class, new chemical entity that combines two potentially synergistic mechanisms of action, bifunctional alkylating activity and pan histone deacetylase inhibition (or HDAC inhibition). Tinostamustine is under investigation in patients with glioblastoma, an aggressive brain cancer that is very challenging to treat and for which there is no cure.1 Most patients do not survive more than 15 months with current treatment approaches.1 Tinostamustine has the potential to be a first-line treatment. Nearly 14,000 people in the U.S. are diagnosed with glioblastoma each year. Tinostamustine will be investigated in patients with newly diagnosed glioblastoma as an adjuvant therapy following standard treatment with surgery, chemotherapy and radiation, as well as in patients with recurrent disease.

GBM AGILE is a pioneering, international adaptive platform trial designed to accelerate the identification of effective treatments for glioblastoma. It is led by GCAR, a non-profit corporation, and is supported by a global network of oncologists, neuro-oncologists, clinicians, researchers, biostatisticians, and patient advocates. Unlike traditional trials, GBM AGILE offers a seamless phase 2/3 design that supports a well-defined path for FDA registration. It allows for the simultaneous assessment of multiple investigational therapies against a common control arm, significantly reducing development time by adapting to emerging data and prioritizing promising new treatments.

"We are pleased to partner with GCAR in GBM AGILE to further study tinostamustine, which has shown promise in early trials," said Dr. Julie Ducharme, Vice President and Chief Scientific Officer, Purdue. "GBM AGILE accelerates the clinical trial timeline to speed medicines to patients, which aligns with our mission to address serious, unmet medical needs."

"We are pleased to be evaluating tinostamustine in GBM AGILE," said Dr. Meredith Buxton, Chief Executive Officer/President, GCAR. "Glioblastoma is the deadliest brain cancer, and our mission is to find promising new treatments to improve overall survival as compared to standard treatments. We are now hard at work collaborating on the Investigational New Drug Application (IND) and Clinical Trial Application (CTA) submissions necessary to include tinostamustine in our innovative and patient-centric adaptive trial."

The study is anticipated to begin following IND submission and FDA approval of the protocol with activation in Canada, Europe, and Australia to follow.

On September 17, 2025 TATUM bioscience, a preclinical biotechnology company developing a new immunotherapy to fight cancer, reported the publication of new research showing strong antitumor activity from its proprietary bioengineered multimodal nanofilaments (Press release, TATUM bioscience, SEP 17, 2025, View Source [SID1234656047]). The study, published in the new issue of the Journal for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (JITC), details how this groundbreaking therapy exposes ("uncloaks") cancer cells and orchestrates a comprehensive immune response that eradicates tumors.

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"Our innovative nanofilament-directed immunotherapy overcomes cancer’s ability to hide from the immune system and unleashes a coordinated immune response against tumor cells," said Jean-François Millau, Ph.D., co-founder and Chief Executive Officer of TATUM Bioscience.

"The immune system can be compared to an orchestra: for a powerful antitumor response, each instrument — or cellular component — must play its part at the right moment. That’s what our drug candidate, TAT003, achieves." Dr. Millau is a co-author on the article, "Nanofilament immunotherapy induces potent antitumor vaccine responses."

How TAT003 Works

TAT003 is administered directly into the tumor ("intratumorally") and combines three biological functions to drive the immune system’s antitumor response:

Checkpoint Blockade: Anti–PD-L1 single-chain antibody fragments at one end of the nanofilament bind to tumor cells, decorating the tumor and blocking the PD-L1 immune checkpoint. This allows the immune system to recognize and target the cancer.
Innate Activation: Encapsulated within the nanofilament is a TLR9 agonist, which activates macrophages to engulf tumor cells. Activated macrophages present tumor antigens to the broader immune system.
T Cell Stimulation: IL-2 molecules on the nanofilaments stimulate T cells, triggering them to seek out and destroy other cells bearing tumor antigens — mimicking a vaccine-like response.
"Strikingly, the antitumor response triggered by TAT003 nanofilament immunotherapy was both systemic and durable in animal models," said Kevin Neil, Ph.D., co-founder and Chief Scientific Officer of TATUM. "Replicating these results in patients is our goal as we move toward clinical development."

Unique Advantages Over Personalized Vaccines

TATUM’s engineered nanofilaments offer distinct advantages over other therapeutic strategies. According to Dr. Neil, "TAT003 elicits a vaccine-like effect without the complexity and cost of individualized neoantigen vaccines, which require tumor sequencing and custom manufacturing for each patient. Our approach is designed to be more practical and scalable."

Manufacturing complex, multi-specific biologics remains a major challenge in biopharma. TAT003 addresses this with a proprietary synthetic biology platform: "We have engineered bacteria to function as miniature drug factories, producing fully assembled therapeutic nanofilaments in a single step," explained Dr. Millau. "We simply culture the bacteria, and they secrete the complete, functional drug."

Dr. Gerald Batist, director of the Segal Cancer Centre at Jewish General Hospital in Montréal, commented, "These results are highly promising and suggest that nanofilament-directed immunotherapy could overcome some of the limitations seen with today’s personalized neoantigen approaches. Engaging both innate and adaptive immunity, as shown here, is likely key to improving outcomes for patients who do not benefit from current immunotherapies. This represents a major step forward in the evolving landscape of immuno-oncology." Dr. Batist is a medical advisor to TATUM.

Looking Ahead

These findings represent a major milestone for TATUM Bioscience and the advancement of nanofilament-directed immunotherapy, paving the way for a powerful and innovative approach to treating solid tumors. TATUM is currently raising funds to accelerate development and bring TAT003 into clinical trials. The company welcomes interest from investors, partners, and clinicians seeking to be part of this next era in cancer immunotherapy.

On September 16, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported its half year financial results for the six-month period ended June 30, 2025, and provided a business overview (Press release, MaaT Pharma, SEP 17, 2025, View Source [SID1234656011]).

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"In the first half of the year, MaaT Pharma achieved key clinical and regulatory milestones, bringing us closer to providing a much-needed therapeutic option for patients with aGvHD and to becoming the first Company to bring a microbiome medicine to market in Europe. With the EMA submission of Xervyteg (MaaT013), our Clinigen partnership, and the EIB financing, we have strengthened both our operations and financial position. These achievements illustrate our strategy to advance development while preserving shareholder value through a balanced mix of dilutive and non-dilutive funding. As we move into the second half of 2025, we remain focused on execution, preparing with Clinigen for potential market entry, and strengthening our leadership in microbiome-based therapies," stated Eric Soyer, Chief Financial Officer of MaaT Pharma.

Pipeline Highlights

In Hemato-Oncology

Acute Graft-versus-Host Disease (aGvHD) – Xervyteg (MaaT013)

In January 2025, the Company announced positive topline results from the pivotal Phase 3 ARES Study evaluating Xervyteg(MaaT013) in aGvHD. The study met its primary endpoint with a significant gastrointestinal overall response rate at Day 28 of 62% and demonstrates the unprecedented efficacy of Xervyteg as third-line treatment of aGvHD with gastrointestinal involvement (GI-aGvHD) consistent with communicated EAP results.
In March 2025, the Company received positive opinion from European Medicines Agency (EMA).Pediatric Committee on the Pediatric Investigation Plan for Xervyteg(MaaT013), a key milestone achieved towards the Marketing Authorization Application (MAA) submission to the EMA.
In March 2025, the Company announced a positive outcome from the final DSMB meeting on ARES Phase 3 trial, confirming the remarkable efficacy results and positive risk/benefit profile of MaaT013 in third-line GI-aGvHD.
In June 2025, the Company announced the submission of a Marketing Authorization Application (MAA) to the EMA for its lead drug candidate MaaT013, under the registered brand name of Xervyteg. If approved, the Marketing Authorization would establish Xervyteg as the first microbiota therapeutic approved by the EMA, the first one in hemato-oncology worldwide and the first approved therapy in third-line GI-aGvHD.
In June 2025, the Company presented positive updated data in Early Access Program for 173 patients at the 2025 annual EHA (Free EHA Whitepaper) Congress supporting the high efficacy and good safety profile of Xervyteg. This dataset confirms the breakthrough potential of Xervyteg for aGvHD patients with limited treatment options.
In July 2025, the Company announced the signature of a license and commercial agreement with Clinigen, a global specialty pharmaceutical services group and a leading European player in hospital distribution and market access, to streamline the pathway for ensuring access to this medicine across Europe. With this partnership, MaaT Pharma demonstrates its capability to supply products to pharmaceutical companies, including those specializing in rare diseases while ensuring scale-ups for commercial. The Company received an upfront payment of €10.5 million and could receive additional payments of up to €18 million depending on the achievement of pre-specified regulatory and sales milestones. The Company will also be eligible to receive royalty payments on net sales of a percentage in the mid-thirties and regular cash flow as per the supply agreement.
Final results from the pivotal ARES study, including 12-month overall survival data, are expected before the end of 2025 and will be incorporated into the filing dossier. Data are also being submitted in a peer reviewed journal and upcoming scientific congress.
The potential marketing authorization could be delivered around mid-2026 (if approved), then enabling the start of the commercialization of Xervyteg in Europe in the second half of 2026.
In parallel, the Company continues discussions with the FDA for a dedicated pivotal study in the U.S., with the objective of enabling the earliest possible access to Xervyteg for U.S. patients. Such a study could be initiated in 2026, subject to regulatory confirmation as MaaT Pharma continues watching the evolving regulatory policies and process in the United States, and subject to appropriate financing.
The Company continues to expand its U.S. footprint through its Early Access Program, with recurring patient requests now coming from three leading hospitals: City of Hope (Duarte- Los Angeles, CA), Massachusetts General Hospital (Boston, MA), and the University of Alabama Hospital (Birmingham, AL).
Allogenic Hematopoietic Stem Cell Transplant (allo-HSCT) – MaaT033

Over the past 12 months, four DSMB safety assessments were conducted for MaaT033 in the Phase 2b PHOEBUS randomized trial designed to be pivotal: three routine evaluations and one interim analysis focused on excess mortality. All confirmed a favorable safety profile and recommended continuation of the trial without modifications.
The last patient enrollment in the trial is anticipated for mid-2026, with 1-year Overall Survival results expected in H2 2027.
In Immuno-Oncology

Xervyteg and MaaT033 – Exploratory trials using the MET-N platform (donor derived conducted as Investigator-Sponsored Trials (ISTs) to inform further developments

In March 2024, the Company completed patient recruitment for the Phase 2a randomized clinical trial (NCT04988841) (PICASSO) sponsored by AP-HP in Paris and in collaboration with INRAE and Institut Gustave Roussy, evaluating Xervyteg in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy) and nivolumab (Opdivo), in metastatic melanoma patients. The primary endpoint is to assess whether the safety of Xervyteg combined with ipilimumab and nivolumab differs from that of ipilimumab and nivolumab plus placebo. The Company provided its Xervyteg drug candidate and placebo and contributes to the microbiome profiling of patients using its proprietary gutPrint AI research engine, while the trial investigator-sponsor handled recruitment, treatment and is overseeing data collection and analysis. Data readout is expected in H2 2025 as previously announced.
In May 2024, the Company announced its participation in the IMMUNOLIFE ‘RHU’ (university hospital trial) program, a consortium including academic partners, such as Institut Gustave Roussy (IGR), a world-renowned center in the field of cancer treatment, and biotech companies. MaaT033 will be tested as a concomitant treatment to cemiplimab (Regeneron), an anti-PD1 therapy, to assess the potential increase in response rate in patients who have received antibiotics. The primary endpoint will be the disease control rate (DCR), defined as the proportion of patients who do not exhibit disease progression (CR, PR, or SD) according to RECIST 1.1 criteria. This investigator-sponsored, randomized, multicenter Phase 2 trial will evaluate MaaT033 in patients with advanced non-small cell lung cancer (NSCLC), with MaaT Pharma supplying the investigational product. The trial is expected by the sponsor to start in H2 2025.
MaaT034 – Next-generation drug candidates with co-cultured technology (MET-C platform) to expand in solid tumors

In April 2025, the Company presented new preclinical data for MaaT034, its next generation product, showing compelling anti-tumor efficacy results in germ-free mice at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025.
Key results included:

Metagenomic analysis shows that MaaT034 reproduces the microbial functions of Xervyteg, improves DC-mediated T cell activation and potentiates anti-tumor effects mediated by anti-PD-1 checkpoint blockade in vitro.
MaaT034 optimizes anti-PD1 mediated activity in tumor-bearing, germ-free mice. While anti-PD1 alone reduced tumor growth by 10%, the combination of anti-PD1 and MaaT034 resulted in a 83.7% tumor growth reduction (compared to a 24.2% reduction when using a single strain of Akkermansia muciniphilabacteria).
In Neurodegenerative Diseases

Amyotrophic Lateral Sclerosis (ALS) – MaaT033

In May 2025, MaaT Pharma announced positive final Phase 1b results for MaaT033 in ALS, showing a favorable safety and tolerability profile supported by biomarker and microbiome analyses. Rapid and sustained microbial engraftment was observed, along with a slower rate of disease progression (ALSFRS-R slope to be interpreted with caution). The Company is seeking a partner to further advance clinical evaluation in ALS.
Corporate updates

In March 2025, the Company announced the completion of a capital increase of €13 million with historical shareholders.
In July 2025, the Company announced that it has secured a €37.5 million, 4-tranche financing from the European Investment Bank (EIB). The financing will support the advancement of its late-stage hemato-oncology clinical programs including the lead-asset Xervyteg, recently partnered with Clinigen in Europe, and currently under regulatory review by the European Medicines Agency (EMA) for the treatment of aGvHD and the second drug candidate, MaaT033, currently being evaluated in a Phase 2b randomized controlled trial in improving survival for patients receiving allo-HSCT.
Following the annual review of the Euronext Paris indices on September 11, 2025, the Scientific Index Committee has decided to include MaaT Pharma in the CAC Small, CAC Mid & Small, and CAC All-Tradable indices. In addition, during the first part of 2025, the Company’s free float increased from 12.57% to 24.94%.
MaaT Pharma announces evolution of its leadership team, with the appointment of:
Frédéric Fasano, Pharm.D, MBA, ICD.D, joining MaaT Pharma as Chief Strategy & Corporate Development Officer, following a long-standing collaboration as a strategic consultant to the Company. With a strong track record in corporate leadership across the pharmaceutical and biotech sectors — including nearly a decade as CEO of Servier Canada — Frédéric will play a pivotal role in shaping and executing MaaT Pharma’s corporate strategy. He will also lead efforts to forge strategic partnerships and drive the Company’s business growth.
Behzad Kharabi Masouleh, M.D. as Acting Chief Medical Officer, together with Emilie Plantamura, Pharm.D., Deputy Chief Medical Officer, will jointly oversee medical, clinical (including the Marketing Authorization Application process for Xervyteg (MaaT013)), and pharmacovigilance activities. Dr. Kharabi brings extensive industry experience in hematology and cancer research across all stages of clinical development and has a proven track record of successfully advancing novel therapies to approval, having previously held senior positions at Johnson & Johnson, Kite Pharma/ Gilead Sciences, and T-Knife Therapeutics. The Company extends its gratitude to Gianfranco Pittari, M.D., Ph.D., for his commitment and contributions in advancing innovative therapies for patients in need and wishes him success as he pursues new opportunities.
Sheri Simmons, Ph.D., as Acting Chief Scientific Officer. Sheri brings extensive experience in biotechnology, particularly in the microbiome field, having held scientific leadership positions at Seres Therapeutics, Johnson & Johnson’s Microbiome Solutions team, and most recently at Seed Health, a leading probiotics company. In her new role, she will strengthen the Company’s scientific leadership, overseeing preclinical research, AI/data initiatives, and supporting efforts toward the Marketing Authorization of Xervyteg in aGvHD.
Key Financial Results

The key unaudited financial results for the first half of 2025 are as follows:

Income Statement

In thousands of euros

2025.06

(6 months)

2024.06

(6 months)

Revenue

2 427

1 721

Cost of Goods Sold

(790)

(537)

Gross Margin

1 637

1 184

Other Income

2 494

1 935

Sales and distribution costs

(491)

(308)

General and administrative costs

(3 611)

(2 872)

Research and development costs

(14 778)

(12 695)

Operating Income (loss)

(14 749)

(12 756)

Financial Income

87

161

Financial Expense

(422)

(262)

Net financial income (expense)

(336)

(101)

Income (loss) before income tax

(15 085)

(12 856)

Income tax expense

–

–
Net Income (loss) for the period

(15 085)

(12 856)

Prepared in accordance with international accounting standards IFRS

Revenues totaled €2.4 million as of June 30, 2025, compared with €1.7 million on June 30, 2024, mostly driven by the 37.5% increase in sales from the Early Access Program at €2.3 million vs €1.7 million in the previous year.

Operating loss was €14.7million in the first half of 2025 compared with €12.8 million in the first half of 2024. The €2.0 million increase was mostly attributable to research and development costs, which progressed from €12.7 million in the first half of 2024 to €14.8 million in the first half of 2025, consistent with the advancement of the Company’s late stage clinical programs, in particular with the data analysis and regulatory activities for MaaT013 (Xervyteg) and with the ongoing patient recruitment in the PHOEBUS Phase 2b trial in allogeneic-HSCT with MaaT033.

Cash Position

As of June 30, 2025, total cash and cash equivalents were €15.0 million, compared with €20.2 million as of December 31, 2024. The cash position as of June 30, 2025 does not include the upfront payment received from Clinigen’s partnership in July 2025 and the upcoming Tranche A from the EIB expected in September 2025.

Over the first half of 2025, net cash utilization was €5.1 million. Net cash used in Operating and Investment activities was €15.4 million, in line with the operating loss for the period, while cash generated in the Financing activities was €10.3 million, with net proceeds from the €13 million capital raise in March 2025, while loan repayments were €1.6 million.

With the upfront payment from its commercialization agreement with Clinigen and the already available and upcoming first tranche from the loan agreement with the EIB, and with continued strong cash discipline, the Company believes it has sufficient cash to cover its current needs and planned development programs to the end of February 2026. The Company is pursuing several dilutive and non-dilutive financing options, leveraging on the financing initiatives announced over the summer, to further extend its cash horizon.

The Company has updated its corporate presentation available on its website: www.maatpharma.com/investors and has filed its half-year Financial Report to the AMF (Autorité des Marchés Financiers).

Upcoming financial communication and conferences*

September 23, 2025: Lyon Pole Bourse Forum
September 25, 2025: KBC Securities Life Sciences Conference, Brussels
October 1, 2025: BNP-Portzamparc Conference
October 7-8, 2025: Investor Access Event, Paris
November 4, 2025: Publication of revenues and cash position for Q3 2025

On September 17, 2025 Akari Therapeutics, Plc (Nasdaq: AKTX), an oncology biotechnology company developing novel payload antibody drug conjugates (ADCs), reported that it has filed a provisional patent application with the United States Patent and Trademark Office (USPTO) covering the Company’s antibody drug conjugate (ADC) platform using Akari’s spliceosome payload PH1 for treating cancer by modulating alternative splicing within cancer cells (Press release, Akari Therapeutics, SEP 17, 2025, View Source [SID1234656030]). This patent application represents a new patent family to further extend Akari’s proprietary position with respect to the Company’s novel payload, PH1 (a novel Thailanstatin analog).

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Abizer Gaslightwala, President and Chief Executive Officer of Akari Therapeutics commented, "These novel data included in our provisional patent application continue to demonstrate the advances in our understanding of spliceosome modulation and the growing potential of our PH1 payload to build first-in-class ADCs that work in unique ways from current options, and could potentially drive differentiated clinical outcomes and remissions for cancer patients in the future. We will continue to progress our exciting research on our spliceosome modulating payload PH1 and present evolving data and insights. This specific provisional patent application increases the scope of our intellectual property estate and enables long term value creation for Akari and potential partners we elect to work with on our ADC portfolio."

The new application relates to how Akari’s novel PH1 payload demonstrates its ability to modulate the spliceosome to disrupt alternative splicing drivers and the subsequent synthesis of proteins needed for cancer tumors to survive and grow.

Alternative splicing (AS) leads to the production of functionally distinct protein isoforms. Cancer cells hijack this process to produce isoforms that support the "hallmarks of cancer," a set of capabilities that often tumors acquire, which contribute to the growth, proliferation, and survival of the cancer in the tumor microenvironment, and a host of other deleterious effects. Changes in AS have been linked to almost all aspects of tumor formation and cancer growth and metastasis, including changes affecting cancer cell metabolism, inhibiting apoptosis (delaying natural cell death), cell cycle control, evading the immune system, invasion of cancer cells to other tissues/organs (metastasis and spreading), and resistance of cancer cells to current therapies. Disrupting AS is a central, generalized approach to target all cancers at a fundamental level of their survival and growth, including those difficult cancers driven by specific spliced variants (i.e. AR-v7 in prostate cancer) or cancers driven by other known factors.(i.e. ,VEGF, HER2, Caspase-2). This opens up the possibilities and potential for Akari to become a leader in developing therapies against a wide range of cancers utilizing spliceosome modulation using Akari’s ADC platform.

This provisional patent application is a culmination of the exciting and groundbreaking work in alternative splicing Akari is progressing with its novel ADC payload PH1. This patent also adds to the body of knowledge already established around the PH1 payload including its potent cytotoxicity and robust immune cell activation demonstrated in multiple preclinical cancer models. The Company is committed to applying this expanded knowledge around disrupting alternative splicing to advance its current ADC portfolio, consisting of AKTX-101 (Trop2 ADC with PH1 payload), as well as future programs (AKTX-102, undisclosed target with PH1 payload).