Interim Results for the Six Months to June 30, 2026, and Business Update

On September 29, 2026 Avacta Therapeutics (AIM: AVCT, "the Company", "Avacta"), a life sciences company developing innovative, targeted oncology drugs, reported its unaudited interim results for the six months ended June 30, 2026 ("H1 26").

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H1 highlights and post period

Research & development

Next Generation pre|CISION pipeline

AVA6103 (FAP-Exd)
Preliminary preclinical and clinical data with AVA6103 have been presented recently. Data reported in the Phase 1a trial in patients with select solid tumors demonstrate proof of mechanism with two key findings:
AVA6103 pre|CISION controlled-release exatecan demonstrates a clean safety profile through the first three dose levels, including a payload dose level 50% higher than the maximum tolerated dose (MTD) of conventional exatecan at dose level 3, and
The comparison of the preclinical modeled pharmacokinetic (PK) data and clinical trial PK data demonstrates an exceptional alignment through the first 3 dose levels with controlled release of exatecan evident in patients for days after dosing
The first head-to-head preclinical comparison of AVA6103 and Enhertu, a marketed antibody drug conjugate (ADC) that targets HER2, demonstrates that AVA6103 shows better antitumor activity vs. Enhertu, with deep and durable responses delivered by our dose dense regimen that has been applied in the FOCUS-01 trial.
Updated preclinical pharmacology and exposure data analyses, highlighting the favorable delivery profile, presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2026.
Comparative analyses of pre|CISION payload delivery preclinical pharmacokinetics (PK) via AVA6103, compared with now two approved ADCs (Enhertu and Datroway), presented at the Company’s Science Day, showing the clear advantages of pre|CISION over traditional ADCs.
U.S. Food and Drug Administration (FDA) granted clearance of the Investigational New Drug (IND) application for AVA6103 in January, 2026 and the first patient was treated in the trial in March, 2026.
AVA6207 (Dual Payload)
Presented first in vivo efficacy and exposure pharmacology for the pre|CISION dual payload technology program at AACR (Free AACR Whitepaper) 2026.
Presented updated in vivo studies of the dual payload delivery system AVA6207 at Science Day.
pre|CISION platform and First Gen (AVA6000)

Reported data validating the pre|CISION ​PDC platform, with AVA6000 in patients with salivary gland cancer, where robust tumor responses are observed with low expression of fibroblast activation protein (FAP) and the persistence of FAP expression despite tumor response.
Published new data demonstrating the favorable delivery profile and advantages of pre|CISION compared to a marketed antibody drug conjugate (ADC)
AVA6000 (Faridoxorubicin, pending partnering)
Presented updated Phase 1a/1b data showing encouraging early efficacy signals for AVA6000 in salivary gland cancers at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
At the end of Phase 1 meeting, certain pivotal trial elements and the regulatory path forward with Phase 1b data were agreed with the U.S. Food and Drug Administration (FDA) for potential full regulatory approval.
Agreed updates with the FDA to the ongoing Phase 1 trial protocol including the removal of the maximum dosing limit and to allow flexibility in dosing levels to identify the dose for further development.
Financial

Strengthened financial position to support R&D programs:

Completed oversubscribed placing and subscription, raising £10 million (March 2026), and raised gross proceeds of approximately £9 million in an equity fundraise (June 2026), from institutional investors and existing shareholders.
Today, separately announcing a proposed capital raise to further strengthen the balance sheet
Cash and short-term deposit balances at June 30, 2026, of £20.27 million (31 December 2025: £16.9 million million). As of August 31, 2026, cash held was £10.59 million.
Corporate

Strengthened leadership and corporate governance via Board appointments:

Richard Hughes as non-executive Chairman (June 2026)
Patrick Vink as Deputy Chairman (in July 2026)
Mats Blom as Chair of the Audit Committee (in September 2026)

Outlook 2026 and beyond

The design of the FOCUS-1 trial of AVA6013 and preclinical updates were presented in Trials in Progress presentations at both the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Conference on Pancreatic Cancer, being held on September 25-28, 2026, and the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress, being held on October 23-27, 2026.
First efficacy data from the FOCUS-01 trial with AVA6103 is anticipated to be presented in H1 2027 – including data from clinical tumor biopsies which are anticipated to confirm AVA6103 is being retained in a ‘drug reservoir’ in the tumor, based on the preliminary Phase 1 data.
The selection of the payloads and data to support clinical candidate selection for the Dual Payload Next Gen Program (AVA6207) will be presented in Q4 2026.
Clinical data from the First Gen faridoxorubicin (AVA6000) program will also be presented in Q4 2026 at the ESMO (Free ESMO Whitepaper) Congress, Oct 23-27, 2026.

Christina Coughlin, CEO of Avacta, commented:

"This has been a transformative first half for Avacta, with compelling clinical validation of our pre|CISION platform delivering robust and durable tumor responses and demonstrating the clear advantages of our approach over conventional ADCs. The controlled-release mechanism of our Next Gen pipeline with AVA6103 as the lead asset is now working in patients, exactly as modelled, and we are moving towards initial efficacy data in 2027.

"This progress is underpinned by a strong and complementary leadership team. We have further reinforced the expertise and governance at Avacta with several senior appointments in 2026 to management and our Board, who bring the benefit of their scientific, industry, markets and financial acumen to the Company as we advance our proprietary pipeline and explore partnering opportunities to maximize the potential of our pre|CISION platform.

"Our financing activities have raised approximately £19.0 million in 2026 to date, which together with the anticipated proceeds of our planned capital raise being launched today, will ensure funding is in place to execute on multiple value inflection milestones, including the efficacy data on AVA6103 in H1 2027, as well as payload selection for our dual-payload program AVA6207 later this year."

Enhertu (trastuzumab deruxtecan; T-DXd) is a protease cleavable-linker ADC, approved for both breast cancer and gastric cancer indications (an AstraZeneca/Daiichi Sankyo product). Datroway (datopotamab deruxtecan-dlnk; Dato-DXd) is a protease cleavable-linker ADC, approved for certain types of breast and lung cancer (a Daiichi Sankyo product).

Interim report

Strategic overview

We are making excellent progress in the development of our unique pre|CISION technology platform, pioneering a novel, differentiated class of pre|CISION-based medicines to revolutionize drug delivery.

Our platform technology demonstrates multiple advantages over conventional therapeutics by addressing one of the primary challenges of effective treatment of diseases, specifically the balance between efficacy and safety. pre|CISION-based medicines are specifically designed to be silent (inert) in the bloodstream and in the tissues, and to only activate once in the tumor.

We are confident in Avacta’s ability to partner preCISION across a number of modalities and in the management team’s ability to secure the requisite funding to progress the pre|CISION platform through multiple value driving events.

pre|CISION – our proprietary technology

The challenge in oncology is that the most effective therapies cause the most toxicity in normal tissues. The ability to deliver the active drug directly to the tumor is the promise of our proprietary pre|CISION platform. Avacta’s innovative pre|CISION peptide drug conjugates (PDC) are a novel entry to the XDC drug class, leveraging the success of antibody drug conjugates with alternative methods of delivery beyond antibodies.

The key aspect of pre|CISION peptide drug conjugate (PDC) technology is that the conjugated drug (the combination of the oncology drug and our peptide) is inert. It is incapable of entering cells and killing until the peptide is specifically released when it comes into contact with common tumor-associated protein, known as fibroblast activation protein or FAP, in the tumor.

When a pre|CISION PDC encounters FAP in the tumor, the peptide is cleaved and the active payload is released. The release of the payload from the pre|CISION compound in the tumor results in higher concentration of the drug at the tumor and lower blood and healthy tissue levels than would be achievable with standard systemic administration. Importantly, the increased toxicities (payload) at the tumor are directly associated with the pre|CISION medicines.

Two factors that dictate the antitumor potential of pre|CISION medicines are (1) the expression of FAP in the tumor to cleave the peptide (the amount of the FAP protein that exists in the tumor) and (2) the inherent susceptibility of the associated tumors to the chemotherapy (chemicals in the drug) that is released.

We believe that pre|CISION can deliver higher drug levels within tumors which will lead to improved antitumor activity while reducing systemic toxicities. This will dramatically impact the therapeutic index and efficacy of a given anticancer drug. We have observed this with our first clinical program, faridoxorubicin. This program has demonstrated a dramatic reduction in the toxicities associated with conventional doxorubicin and is delivering exciting preliminary efficacy data.

To further extend the pipeline, Avacta has invented the Next Generation Controlled Release pre|CISION technology that adds a chemical capping group and linker with two key advantages: (1) greater control over the release of the payload in the tumor, allowing optimized pharmacokinetic delivery even with payloads that have challenging PK properties, and (2) implementing the linker technology opens a large number of payloads that can be delivered with Next Gen pre|CISION, permitting a full exploration of the landscape opportunity of pre|CISION with ~90% FAP expression among solid tumors.

Programs

Our pre|CISION PDCs leverage this tumor-specific release mechanism in a small molecule format to provide unique benefits over traditional antibody drug conjugates (ADC), releasing active payload in the tumor and reducing systemic exposure and toxicity which enables dosing to be optimized to deliver the best outcomes for patients. The lead clinical program is AVA6103, a Next Generation FAP-enabled controlled release pre|CISION version of exatecan that delivers the payload directly in the tumor with limited peripheral blood exposure and is currently in clinical development as a treatment for tumor types sensitive to exatecan including cervical cancer, HR+ breast cancer, small cell lung cancer, gastric cancer, colorectal cancer and pancreatic cancer.

Our first generation asset, Faridoxorubicin (AVA6000) continues in Phase 1b. We anticipate presenting data in October, 2026, at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) meeting from the cohort of patients with salivary gland cancer. This program is intended to go forward within a partnership and discussions are ongoing regarding such an arrangement.

AVA6207 is our Next Gen Dual Payload program which is on track for the goals, in 2H 2026, of naming of the two payloads that will be included in the molecule and clinical candidate selection. Clinical candidate selection is based on the preclinical pharmacology, chemical properties of the molecule with initial assessment of manufacturing for use in the clinic. These data will be presented in the fourth quarter of 2026.

Outlook

Avacta continues to build our value proposition and our unique world-class scientific and clinical capabilities. Our data are robust and building and industry interest in our innovative platform continues to increase. We are very excited about the next stages of Avacta’s journey with the recent clinical data demonstrating proof of mechanism for AVA6103 in the clinic demonstrating the potential in the Next Gen pre|CISION platform. Our upcoming data catalysts demonstrate the progress made in the programs, including the presentation of the FOCUS-01 trial and updated preclinical work with AVA6103, our updated Farodoxorubicin data being presented in October at ESMO (Free ESMO Whitepaper), and the payloads and clinical candidate selection in the Next Gen pre|CISION Dual Payload program in the fourth quarter of 2026.

Financial Review

Revenue

Revenues from continuing operations for the six months ended 30 June 2026 were £0.06 million (H1 2025: £0.06 million; FY 2025: £0.113 million).

Revenues from discontinued operations for the six months ended 30 June 2026 was £nil million (H1 2025: £6.10 million; FY 2025: £6.20 million).

Research costs and selling, general and administrative costs

Research costs relate predominantly to the clinical and pre-clinical development work of the pre|CISION therapeutics programs as planned increased to £9.69 million (H1 2025: £7.20 million; FY 2025: £18.76 million).

Other costs and charges

Depreciation from continuing operations decreased to £0.56 million (H1 2025: £0.73 million; FY 2025: £1.27 million). Amortization expense remained at £0.01 million (H1 2025: £0.01 million; FY 2025: £0.01 million).

The share of the costs from the AffyXell joint venture was £0.26 million (H1 2025: £0.19 million; FY 2025: £0.45 million). The share of losses reflects the Group’s 21% ownership share of the losses accumulated in the year. The Group investment remained at 21%.

Share-based payment charges were £0.54 million (H1 2025: £0.74 million; FY 2025: £2.13 million).

Operating loss

The Group’s operating loss from continuing operations decreased to £13.91 million (H1 2025: £14.18 million; FY 2025: £29.89 million).

Convertible bond costs

During the period, the Group made interest-only cash repayments of £0.66 million in accordance with the amended bond terms and settled a further £1.20 million of the debt component through an early equity conversion.

The Board continues to consider each settlement event as it arises, taking into account a range of factors including the Company’s cash runway, shareholder dilution and broader business prospects.

The bond agreement contains embedded derivatives in conjunction with an ordinary host debt liability. Accordingly, the convertible bonds are presented in the Consolidated Statement of Financial Position as two separate components: ‘Convertible bond – debt’ and ‘Convertible bond – derivative’. The derivative element is measured at fair value using a Monte Carlo option pricing model.

The derivative element was revalued as at 30 June 2026 to £3.29 million (30 June 2025: £0.27 million; 31 December 2025: £2.79 million). After taking account of the £0.37 million reduction arising from the early conversion, this resulted in a £0.87 million charge recognised in profit or loss in the period (H1 2025: £1.01 million credit; FY 2025: £1.51 million debit).

The debt element of the bond increased to £15.77 million (H1 2025: £18.17 million; 31 December 2025: £13.36 million). The movement primarily reflects a non-cash interest expense of £3.46 million and a £0.82 million loss arising from revisions to expected future cash flows, partially offset by £0.66 million of interest-only cash repayments and an early conversion of £1.20 million. As of 30 September, the outstanding loan balance is £12.00 million.

Loss for the period

The reported loss from continuing operations after taxation was £19.02 million (H1 2025: £16.13 million; FY 2025: £36.26 million).

The basic loss per share from continuing operations was 4.23p (H1 2025: 4.23p; FY 2025: 9.07p).

The basic loss per share from discontinued operations was nil (H1 2025: 3.82p; FY 2025: 0.59p).

Cash flow

The Group reported cash and cash-equivalent balances of £20.27 million (30 June 2025: £12.65 million; 31 December 2025: £16.86 million). Cash and cash-equivalents were £10.59 million as of 31 August 2026.

There was a cash outflow from operations and working capital movements of £15.69 million (H1 2025: £12.33 million; FY 2025: £24.93 million) and cash outflow from investing activities was £0.02 million (H1 2025: inflow of £8.77 million; FY 2025: inflow of £9.90 million, reflecting the proceeds of the sale of Launch and Coris).

Cash inflow from financing activities, being net proceeds from the issue of share capital and share options, net of the principal elements of lease payments amounted to £17.76 million (H1 2025: inflow of £0.67 million; FY 2025: inflow of £15.64 million). The cash inflows for H1 2026 and FY 2025 related to equity fundraising.

Financial position

Net assets as at 30 June 2026 were £3.77 million (30 June 2025: £0.14 million; 31 December 2025: £2.48 million) of which cash and cash equivalents amounted to £20.27 million (30 June 2025: £12.65 million; 31 December 2025: £16.86 million).

Right-of-use assets amounting to £0.63 million (30 June 2025: £1.77 million; 31 December 2025: £1.32 million) are recognized in relation to the Group’s leasehold properties, together with a corresponding lease liability of £0.80 million (30 June 2025: £1.97 million; 31 December 2025: £1.55 million).

Intangible assets decreased to £1.55 million (30 June 2025: £1.83 million; 31 December 2025: £1.55 million) due to amortization of intangible assets.

Liabilities in relation to the unsecured senior convertible bonds issued in October 2022 result in a fair value of the derivative element of £3.29 million (30 June 2025: £0.27 million; 31 December 2025: £2.79 million). The convertible bond debt element at 30 June 2026 was £15.77 million (30 June 2025: £18.17 million; 31 December 2025: £13.36 million).

With current committed expenses, current cash runway takes the Group into early first quarter of 2027.

Events after the reporting period

In July 2026 the Company received an accelerated payment notice from the convertible bond holder in respect of the January 2026 quarterly deferred convertible bond repayment, as referenced most recently in the financing announced on 5 June 2026, which the Company has elected to settle in cash. Accordingly, the Company has settled the accelerated quarterly amortization payment of £3.67 million representing principal of £2.4 million together with the associated interest and fees payable in the deferment.

In September 2026 the Company received an accelerated payment notice from the convertible bond holder in respect of the April 2026 quarterly deferred convertible bond repayment, which the Company elected to settle in cash. Accordingly, the Company has settled the accelerated quarterly amortization payment of £2.613 million, representing principal of £2.4 million together with the associated interest and fees payable in the deferment. Following the cash settlement of the April 2026 quarterly deferred convertible bond repayment, both deferred payments have now been satisfied.

As announced on 1 September 2026, the Board of Directors granted share options under the Company’s Equity Share Option Scheme incentive plan ("ESOS") to all members of the Management Team (the "Options"). The Options are exercisable at an exercise price of 69 pence per share, being the mid-market closing price of the Company’s Ordinary Shares on 27 August 2026, the last trading date before the Options were granted.

As announced on 14 September 2026, Mats Blom, an experienced biotechnology finance executive and Board member with a strong track record as Chief Financial Officer in several life sciences companies, was appointed as a member of its Board of Directors and Chair of the Audit Committee.

(Press release, Avacta Life Sciences, SEP 29, 2026, View Source [SID1234671157])

Vividion Announces Publication of Clinical Data for VVD-214 in Nature Medicine, Highlighting Potential in Colorectal Cancer

On September 29, 2026 Vividion Therapeutics, Inc. (Vividion), a clinical-stage biopharmaceutical company and a wholly owned and independently operated subsidiary of Bayer AG, reported the publication of the first peer-reviewed clinical data from the Phase 1 study of VVD-214, its investigational oral inhibitor of Werner helicase (WRN), in Nature Medicine. The findings demonstrated encouraging antitumor activity, including sustained disease control in patients with high microsatellite instability (MSI-high) colorectal cancer, and a manageable safety profile, supporting the ongoing Phase 1b and Phase 2 combination studies with VVD-214.

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VVD-214, referred to as RO7589831 (VVD-133214) in the publication, is an investigational oral therapy and the first covalent WRN inhibitor in clinical development. It is designed to exploit MSI-high cancer cells’ dependence on WRN-mediated DNA repair, causing lethal DNA damage in cancer cells while minimizing harm to normal cells.

The article, "Werner helicase inhibitor for advanced microsatellite instability solid tumors: a Phase 1 trial" (Yap et al., Nature Medicine,September 2026), reports results from 88 previously treated patients with advanced MSI-high and/or deficient mismatch repair (dMMR) solid tumors. The paper’s lead author is Timothy Yap, M.B.B.S., Ph.D., clinical investigator and professor of Investigational Cancer Therapeutics at The University of Texas MD Anderson Cancer Center. The publication expands on initial findings Dr. Yap presented at the 2025 AACR (Free AACR Whitepaper) Annual Meeting.

A total of 66 patients who had a median of three prior lines of therapy, including 95.5% who received prior immune checkpoint therapy and had MSI-high tumors were evaluable for efficacy. VVD-214 demonstrated a disease control rate of 74.2%, including seven confirmed partial responses (10.6%). Median progression-free survival was 6.7 months, and median overall survival was 17.6 months. Among the subset of those patients with colorectal cancer (CRC), the disease control rate was 80.5%, including three confirmed partial responses (7.3%), and median progression-free survival was 7.3 months. Estimated overall survival at 12 months in this CRC population was 78.3%, with median overall survival not yet estimable. All three CRC responders remained in response at the data cutoff.

A total of 88 patients were evaluable for safety. Common treatment-emergent adverse events were mostly Grade 1 or 2, and treatment-emergent adverse events led to discontinuation in 3.4% of patients.

"These findings reinforce our conviction that synthetic lethal approaches can open new treatment possibilities for patients with MSI-high cancers," said Aleksandra Rizo, M.D., Ph.D., President and Chief Executive Officer of Vividion. "The encouraging colorectal cancer results strengthen the rationale for our ongoing combination studies as we work to extend the potential of VVD-214 across treatment settings."

Vividion is evaluating VVD-214 with bevacizumab in an ongoing Phase 1b study in patients with previously treated advanced MSI-high or dMMR colorectal cancer (NCT06004245). An ongoing Phase 2 study is evaluating VVD-214 with pembrolizumab in patients with previously untreated advanced MSI-high or dMMR colorectal cancer (NCT07811193). These studies are assessing whether complementary therapeutic approaches can build on the monotherapy activity observed in Phase 1.

(Press release, Vividion Therapeutics, SEP 29, 2026, View Source [SID1234671154])

Nouveau Biosciences and KYAN Technologies Enter Collaboration to Apply AI-Powered Precision Medicine to Oncology Drug Discovery

On September 29, 2026 Nouveau Biosciences, a biotechnology company developing precision-targeted nanomedicines for cancer, reported that it has entered into an agreement with KYAN Technologies, a functional precision medicine company leveraging small data artificial intelligence and biological experiments, to identify novel synthetically lethal drug combinations for the treatment of cancer.

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The collaboration will combine KYAN’s Optim.AI platform with Nouveau Biosciences’ proprietary drug discovery and polymer nanomedicine platform. The companies aim to identify promising drug combinations using patient-derived tumor data and translate those insights into novel nanomedicines designed to improve drug targeting, pharmacologic performance, tolerability and therapeutic efficacy.

KYAN’s Optim.AI platform applies artificial intelligence and advanced analytics to evaluate drug combinations directly in patient samples or patient-derived tumor organoids (PDO). Using proprietary small data AI technology and biological experiments, KYAN can assess how combinations of therapeutic agents perform against an individual patient’s tumor and statistically rank potential treatment combinations based on their observed activity. KYAN’s CAP/CLIA-accredited laboratory provides results to treating oncologists within approximately seven to 10 days and evaluate therapeutic combinations that include immuno-oncology agents. Their streamlined assays can provide near-real-time actionable information that can be implemented to help patients at that time of the patients encounter.

Nouveau Biosciences is developing an integrated approach to identifying and engineering precision drug combinations based on synthetic lethality, the concept of targeting two or more biological vulnerabilities that, when inhibited together, result in cancer cell death. Under the collaboration, Nouveau plans to use insights generated by KYAN’s platform to inform the design of polymer-based nanomedicines incorporating selected drugs or drug combinations. Importantly, the integration of Nouveaus pipeline tonto their assay models has the potential to identify other drugs with which the Nouveau pipeline will by complementary, and to markedly expand the potential disease indications for their assets.

Nouveau’s polymer nanomedicine technology pioneered by Dr. Langer and his colleagues at MIT, is designed to optimize the delivery and pharmacologic properties of therapeutic agents, with the goal of increasing drug exposure to the intended target while reducing off-target toxicity. By connecting KYAN’s patient-derived functional data and small data AI-driven analysis with Nouveau’s drug engineering capabilities, the companies intend to establish a more direct path from identification of promising combinations to the creation of rapidly deployable and highly differentiated therapeutic candidates.

"This collaboration has the potential to establish an entirely new approach to precision oncology drug discovery," said Charles Karan, Ph.D., Chief Scientific Officer of Nouveau Biosciences. "KYAN has built a powerful platform for extracting actionable insights from patient-derived tumor models. Our objective is to take those insights and translate them into precision-targeted nanomedicines engineered for improved pharmacologic properties, safety and efficacy. We are excited to work with the KYAN team and believe this collaboration can create a highly productive engine for discovering new cancer therapeutics."

"Nouveau addresses an important challenge in precision medicine: how to translate increasingly sophisticated biological and AI-generated insights into differentiated therapeutic products," said Hugo Saveda, Chief Executive Officer of KYAN Technologies. "Nouveau has established compelling proof of principle with its lead asset, Kromastat, and we believe the combination of our technologies creates an opportunity to systematically identify actionable novel therapeutic combinations with the potential to meaningfully improve cancer treatment with unprecedent speed and efficiency."

Nouveau Biosciences has developed a proprietary drug discovery platform coupled with polymer-based nanochemistry designed to enhance drug targeting, tolerability and efficacy. The platform is intended to enable the creation of differentiated anticancer therapeutics supported by strong intellectual property. With a clinic-ready lead program based on validated chemistry and biology, Nouveau is building a pipeline of precision oncology candidates designed to address areas of significant unmet medical need.

"There is no company in the field with the ability to identify and deploy actionable drugs or drug combinations in such a personalized precision targeted fashion", notes Owen A. O’Connor, M.D., Ph.D., Founder and CEO of Nouveau Biosciences. "One of our goals is to close that gap that exists in translating the latest and most sophisticated advances in cancer biology and AI and the identification of actionable treatments to help our patients today, the collaboration with KYAN will be unprecedented and will be a quantum leap in that direction" Dr. O’Connor goes on to say.

(Press release, Nouveau Biosciences, SEP 29, 2026, View Source [SID1234671153])

Moleculin Biotech Completes Enrollment in Part A of Phase 2/3 R/R AML MIRACLE Trial, Keeping Program on Track for Q1 2027 Data Readout

On September 29, 2026 Moleculin Biotech, Inc. (Nasdaq: MBRX) ("Moleculin" or the "Company") reported that it has completed and closed enrollment in Part A of its pivotal Phase 2/3 MIRACLE trial evaluating Annamycin in combination with cytarabine (AnnAraC) for the treatment of adults with relapsed or refractory acute myeloid leukemia (R/R AML), with 89 subjects enrolled and an additional subject currently in screening. With Part A enrollment closed, the Company remains on track for its planned comprehensive data readout of Part A in the first quarter of 2027.

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The rapid pace of enrollment underscores the significant unmet need in R/R AML as well as strong engagement from investigators and clinical sites participating in MIRACLE. The Company expects the expanded Part A dataset to provide important insight into the potential efficacy of AnnAraC across subjects who have relapsed from or are refractory to different first-line (1L) AML treatment approaches, including subjects previously treated with intensive 7+3 chemotherapy and those previously treated with venetoclax-based regimens. Such treatment approaches associated with 1L patient populations are commonly, while not universally, deemed as "fit" and "unfit", respectively.

"Completing enrollment in Part A of MIRACLE is an important milestone for Moleculin and a strong demonstration of the operational execution behind this pivotal program," said Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "We have maintained a rapid pace of enrollment in a global, randomized AML trial and, importantly, remain on track in the first quarter of 2027 for our planned comprehensive data readout – one much deeper in data than our first interim n45 readout. Our focus turns to the maturation and analysis of this substantially larger dataset and what it may tell us about the potential of AnnAraC across distinct and clinically important R/R AML patient populations such as "fit" and "unfit" subjects."

Mr. Klemp continued, "We are particularly excited about the composition of the additional subjects enrolled in Part A and the opportunity that the full Part A population may provide to better understand efficacy based on prior 1L treatment. We estimate that Part A will end with approximately a 50/50 split between "fit" and "unfit" subjects, compared to a roughly 70/30 split, respectively, in the first 45 subjects in Part A. While we are mindful that this shift in composition to more "unfit" subjects represents a more challenging environment for achieving treatment responses, it should provide valuable insight into Annamycin’s ability to fill critical unmet needs. Lastly, we continue to see a lack of cardiotoxicity in MIRACLE’s blinded data."

"Completing Part A enrollment reflects the execution of our team and investigators and moves us directly toward what we believe will be one of the most important milestones in Annamycin’s development," Mr. Klemp added. "We are grateful to the patients and families participating in MIRACLE and to the investigators and clinical teams whose commitment has enabled us to reach this milestone at such a rapid pace. With enrollment complete and the trial on track for a comprehensive Q1 2027 readout, we believe Moleculin is entering a potentially defining period for the MIRACLE program."

About the MIRACLE Trial

MIRACLE is a pivotal Phase 2/3 study of Annamycin in combination with cytarabine for the treatment of adult subjects with AML who are refractory to, or have relapsed (R/R) after, induction therapy. Part A of the trial is a randomized, double-blind portion of the study that compares two different doses of Annamycin plus cytarabine to a control arm of cytarabine plus placebo, all with just one cycle of therapy.

Importantly, as specified by the MIRACLE protocol, remission rates for all three arms, including the control arm, reflect outcomes measured after only a single cycle of therapy. The most commonly cited historical benchmarks in this setting, including the MIRROS and CLASSIC I studies, as well as Moleculin’s own MB-106 study, permitted multiple cycles of treatment. The Company therefore expects absolute remission rates for both the control and Annamycin arms to be lower than those reported in such multi-cycle datasets and believes the most meaningful comparison is the performance of the Annamycin arms relative to the concurrent, randomized control arm evaluated on the same single-cycle basis.

The comprehensive Part A analysis is expected to evaluate efficacy and safety across the complete randomized population and support selection of the optimal Annamycin dose for advancement into Part B of MIRACLE.

For additional information on the MIRACLE trial, please visit ClinicalTrials.gov and reference Identifier NCT06788756.

(Press release, Moleculin, SEP 29, 2026, View Source [SID1234671152])

Medicus Pharma Submits Optimized Teverelix® Registrational Protocol to FDA to Create Partner Ready Program in Advanced Prostate Cancer

On September 29, 2026 Medicus Pharma Ltd. (NASDAQ: MDCX) ("Medicus" or the "Company"), a precision guided, biotech/life sciences company focused on advancing novel and potentially disruptive therapeutic assets, reported that it has submitted an optimized seamless Phase 2b/3 registration-intent protocol to the U.S. Food and Drug Administration ("FDA") for Teverelix, its investigational, next generation, long-acting GnRH antagonist, in advanced prostate cancer ("APC") patients at increased cardiovascular ("CV") risk.

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The submission is designed to establish a defined, capital-efficient and partner-ready registrational pathway for Teverelix without Medicus independently funding an additional clinical development study in the APC program at this time. Importantly, the proposed design is stage-gated: it begins with approximately 80 randomized patients, incorporating and replacing the previously FDA-cleared stand-alone 40-patient open-label Phase 2b dose-optimization study. Once predefined continuation criteria have been satisfied, the program would then proceed to add approximately 528 patients, for approximately 608 patients in total, with eligible patients from the initial stage contributing to the final registration analyses.

If the FDA agrees with the proposed approach, Medicus believes the optimized design could reduce the overall planned clinical development program from approximately 1,500 patients to approximately 600 patients while providing a single pathway encompassing dose optimization, androgen suppression and prospective evaluation of cardiovascular differentiation in APC patients at increased cardiovascular risk, without an upfront commitment to enroll the full study population.

Following FDA feedback, and subject to the nature of that feedback, Medicus’ intent is to seek to out-license or otherwise strategically partner the Teverelix APC program rather than independently fund execution of the proposed registrational study.

There can be no assurance that the FDA will agree with the proposed protocol, that the optimized development pathway will ultimately support registration, or that Medicus will successfully enter into a licensing or other strategic transaction involving Teverelix.

Management Commentary

"Our objective with this submission is not to commit Medicus capital to another APC clinical study-it is to define the most efficient registrational pathway possible and make Teverelix ready for a strategic partner," stated Dr. Raza Bokhari, Executive Chairman and CEO of Medicus. "The FDA previously cleared a 40-patient open-label dose-optimization study. Rather than independently funding that study, we have now proposed an approximately 600-patient seamless registration-intent program that incorporates a more rigorous 80-patient randomized dose-optimization stage and could reduce the overall development population from ~ 1,500 patients to ~ 600 and is more likely to be partner-ready."

Designed to Reach Partner Readiness Without Additional Medicus R&D Spend

The submission represents a further implementation of the Company’s capital-efficient portfolio strategy announced on September 8, 2026, under which Medicus is concentrating capital and organizational resources on programs where focused clinical development may offer the most compelling risk-adjusted opportunity.

Medicus’ objective with the current FDA submission is to complete the regulatory optimization of the Teverelix APC development pathway before committing additional internal capital to clinical execution.

If the FDA provides constructive feedback on the proposed approach, Medicus intends to use the resulting regulatory-defined development pathway as the basis for strategic partnering discussions and seek a pharmaceutical partner to fund and execute subsequent APC clinical development and commercialization

From FDA-Cleared Dose Optimization to a Seamless Registration-Intent Program

On February 10, 2026, Medicus announced that FDA had provided "Study May Proceed" clearance for a 40-patient open-label Phase 2b dose-optimization study of Teverelix in men with advanced prostate cancer appropriate for androgen deprivation therapy. That study was designed to confirm the Teverelix dosing regimen and sustained testosterone suppression.

In addition to focusing its efforts towards GMP grade manufacturing, quality-release, and other clinical study readiness steps, which are anticipated to be completed before the end of this year, the Company continued to optimize the APC development strategy. The newly submitted protocol proposes an approximately 80-patient randomized dose-optimization stage as the first part of an approximately 608-patient seamless Phase 2b/3 registration-intent study. Subject to predefined criteria, the study would then continue with approximately 528 additional patients, with eligible patients from the initial stage contributing to the final registration analyses.

The proposed design is intended to avoid conducting a stand-alone dose-optimization study followed by a separate, substantially larger Phase 3 program. Compared with a previously discussed development approach involving approximately 1,500 patients, the seamless strategy could reduce total planned enrollment by approximately 60% while reducing duplication, time and capital requirements associated with sequential studies.

Cardiovascular-Focused Registrational Strategy

The optimized strategy incorporates emerging randomized clinical evidence published in JAMA Cardiology in 2026 showing that differences in coronary plaque progression between GnRH treatment pathways can be objectively measured over 12 months using non-invasive coronary CT angiography ("CCTA"). CCTA can be incorporated into a multicenter study using standardized scans at baseline and Month 12 with blinded central quantitative analysis.

The proposed protocol combines established testosterone-suppression requirements with a 12-month coronary non-calcified plaque endpoint, while allowing longer-term cardiovascular event follow-up to continue in the same patients. The development strategy is designed to evaluate whether Teverelix can achieve effective androgen deprivation while demonstrating cardiovascular differentiation in advanced prostate cancer patients already at increased CV risk.

If the FDA agrees that this approach could support registration, Medicus believes the proposed design could provide a substantially shorter and more capital-efficient development pathway than the previously contemplated sequential program. Any development timeline remains subject to FDA feedback, successful study execution, enrollment, clinical results and regulatory review.

About Teverelix

Teverelix is an investigational next-generation, long-acting GnRH antagonist formulated as a microcrystalline suspension. Unlike GnRH agonists, which induce an initial testosterone surge, Teverelix provides immediate receptor antagonism, enabling rapid suppression of luteinizing hormone, follicle-stimulating hormone and downstream sex hormones without flare. Medicus is evaluating Teverelix across hormone-dependent indications and is pursuing a capital-efficient development strategy intended to advance programs through regulatory and clinical value-creating milestones while evaluating strategic partnership opportunities.

(Press release, Medicus Pharma, SEP 29, 2026, https://medicuspharma.com/medicus-pharma-submits-optimized-teverelix-registrational-protocol-to-fda-to-create-partner-ready-program-in-advanced-prostate-cancer/ [SID1234671151])