On March 27, 2025 CStone Pharmaceuticals ("CStone", HKEX: 2616), a biopharmaceutical company dedicated to developing innovative cancer therapies, reported 2024 annual results and recent business updates (Press release, CStone Pharmaceauticals, MAR 27, 2025, View Source [SID1234651546]).

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Business Highlights

For the year ended December 31, 2024 and up until the date of this results announcement, key milestones have been achieved across regulatory approvals, clinical advancements, and strategic collaborations, reinforcing our position as a leader in innovative therapeutics. A shortlist of our achievements over this period includes:

Commercial Products

• CEJEMLY (sugemalimab), anti-PD-L1 antibody

– Global expansion and regulatory approvals

Sugemalimab secured three new drug application approvals in 2024, including its fifth indication in mainland China for first-line treatment of gastric/gastroesophageal junction adenocarcinoma (GC/GEJC). Sugemalimab also gained approvals in the EU and UK for first-line treatment of stage IV NSCLC, marking its entry into major international markets. We have recently completed the regulatory submission to the European Medicines Agency (EMA) for the new indication application of sugemalimab in the treatment of Stage III NSCLC.

– Strategic alliances drive global commercialization

Partnered with Ewopharma AG in May 2024 for commercialization in Central and Eastern Europe (CEE) and Switzerland.
Collaborated with Pharmalink Store – L.L.C – O.P.C in November 2024 to expand access across the Middle East, North Africa (MENA) Region and South Africa.
Entered an agreement with SteinCares in January 2025 for Latin America (LATAM).
Additional partnerships in Western Europe, Southeast Asia, and Canada are anticipated in 2025.
– Robust clinical data reinforce efficacy

GEMSTONE-304 Study: Final progression-free survival (PFS) and interim overall survival (OS) data for the first-line esophageal squamous cell carcinoma (ESCC) published in Nature Medicine (February 2024).
GEMSTONE-302 Study: Four-year OS data for stage IV NSCLC presented at the 2024 Congress of European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper), confirming sustained survival benefits.
GEMSTONE-303 Study: Final PFS and OS analysis for GC/GEJC with combined positive score (CPS) ≥5 published in the top-tier medical journal-Journal of the American Medical Association (JAMA) (February 2025).
ESMO Guideline Recognition: CEJEMLY recommended as first-line NSCLC therapy in ESMO (Free ESMO Whitepaper)’s 2025 Non-Oncogene-Addicted Metastatic NSCLC Living Guidelines, covering both squamous and non-squamous NSCLC (February 2025).
• AYVAKIT (avapritinib), KIT/PDGFRA inhibitor

– Localized manufacturing in China

The National Medical Products Administration of China (NMPA) approved domestic production of AYVAKIT tablets (300 mg and 100 mg) in June and August 2024 respectively. Full transition to localized supply is expected in 2025, improving cost efficiency.

– NRDL inclusion enhances accessibility

AYVAKIT (avapritinib) has been included to China’s National Reimbursement Drug List (NRDL), effective January 1, 2024 for unresectable or metastatic gastrointestinal stromal tumor (GIST), harboring the PDGFRA exon 18-mutated, including PDGFRA D842V mutations. This significantly improves AYVAKIT’s affordability for eligible patients.

– Commercial partnership with Hengrui

In July 2024, we forged a new partnership with Jiangsu Hengrui Pharmaceuticals Co., Ltd., granting them the exclusive promotion rights in mainland China to amplify commercial reach and profitability.

• GAVRETO (pralsetinib), RET inhibitor

– Progress towards localized manufacturing

The NMPA’s Center for Drug Evaluation (CDE) accepted the manufacturing localization and bioequivalence (BE) study application in April 2024, and regulatory review is ongoing.

– Sustained collaboration with Allist

Commercial operations has been transferred to Shanghai Allist Pharmaceuticals Co., Ltd in the first half of 2024 under an exclusive agreement signed in November 2023. Collaboration remains strong to maximize market penetration.

Clinical Stage Core Assets

• CS5001 (ROR1 ADC)

– Phase Ib clinical trial with registration potential

A global multicenter clinical trial of CS5001 is actively enrolling patients across the United States of America (U.S.), Australia and China. Recruitment is ongoing for monotherapy cohorts targeting aggressive and indolent advanced lymphomas with potential to be expanded into a Phase II single-arm registrational study. Additional cohorts, including CS5001 in combination with R-CHOP (Rituximab + Cyclophosphamide + Hydroxydaunorubicin + Vincristine + Prednisone) for the first-line diffuse large B-cell lymphoma (DLBCL), and CS5001 in combination with standard of cares (SOC) for front-line DLBCL will be initiated to expand the therapeutic potential of CS5001 across all DLBCL stages. CS5001 is also being studied as both a monotherapy and in combination with a PD-L1 inhibitor for advanced solid tumors, underscoring its versatility across oncology indications.

– Compelling clinical data at ASCO (Free ASCO Whitepaper) & ASH (Free ASH Whitepaper) 2024

Phase Ia data for CS5001 presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated that CS5001 is so far the first receptor tyrosine kinase-like orphan receptor 1 (ROR1) antibody-drug conjugate (ADC) known to demonstrate clinical anti-tumor activity in both solid tumors and lymphomas. Clinical data of CS5001 revealed superior efficacy and favorable safety profile as a monotherapy for both aggressive and indolent advanced lymphomas. At the tentative recommended Phase II dose (RP2D) (125 μg/kg), CS5001 achieved an objective response rate (ORR) of 70% in non-Hodgkin lymphoma (NHL) and 100% in Hodgkin lymphoma (HL). These results support its potential as a faster-to-market candidate and a frontline combination therapy backbone.

• CS2009 (PD-1/VEGF/CTLA-4 trispecific antibody)

– Global Phase I clinical trial initiated

A global multicenter first-in-human (FIH) trial among solid tumors has been launched in Australia in December 2024, with subsequent expansion to China and the U.S.. The first patient was dosed in March 2025.

– Potential FIC/BIC next-generation I/O backbone

Preclinical data presented at the 39th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) highlighted its first-in-class (FIC) or best-in-class (BIC) potential with superior antitumor activity compared to potential competitors benchmarks, including PD-1/CTLA-4 or PD-1/VEGF bispecific antibody and PD-1/ CTLA-4 combination therapies. Unique trispecific mechanism positions CS2009 as a next-generation immuno-oncology backbone with broad applicability.

Preclinical/IND-enabling Stage Programs and ADC Platform

CStone’s preclinical pipeline includes more than nine promising candidates, each with global rights and focusing on FIC/BIC profiles with broad indications. These candidates include multispecific antibodies, ADCs and radionuclide drug conjugates (RDC) covering various therapeutic fields such as oncology, autoimmune diseases, and metabolic disorders. The Company is dedicated to delivering clinical value through the development of these Pipeline 2.0 candidates, which will undergo international, multi-center clinical trials to maximize their global potential.

CStone has also developed an in-house ADC technology platform featuring proprietary linkers, optimized for diverse targets and payloads. This platform supports multiple upcoming ADC projects in Pipeline 2.0, including CS5007 (dual targeting epidermal growth factor receptor (EGFR) & human epidermal growth factor receptor 3 (HER3)), CS5005 (targeting somatostatin receptor 2 (SSTR2)), CS5008 (dual targeting delta-like ligand 3 (DLL3) & SSTR2) and CS5006 (targeting ITGB4).

Future and Outlook

As we look to the future, we reaffirm our commitment to advancing a robust and differentiated pipeline by prioritizing internal discovery capabilities and sustained R&D investments. Concurrently, we aim to maximize the global commercial potential of our approved therapies through strategic collaborations and localized manufacturing enhancements. Key growth drivers in 2025 include:

• Clinical milestones

– Progress CS5001 (ROR1 ADC) and CS2009 (PD-1/VEGF/CTLA-4 trispecific) towards pivotal trials and in parallel pursue global partnerships to expedite development.
– Advance early-stage candidates (e.g., CS2011, CS5007, CS5005, CS5008, CS5006) into clinical stages within the next two years.

• Commercial excellence

– Maximize the value of approved products through strategic collaborations and manufacturing localization (e.g., AYVAKIT, GAVRETO) to enhance profitability and market reach.
– Accelerate ex-China commercialization of CEJEMLY (sugemalimab) via regional partnerships.

• Innovation and technology

– Strengthen proprietary platforms (e.g., ADC technology) to sustain pipeline growth.
– Present key clinical data at major conferences (e.g., AACR (Free AACR Whitepaper), ESMO (Free ESMO Whitepaper), ASH (Free ASH Whitepaper))

Financial Highlights

International Financial Reporting Standards (IFRS) Measures:

• Revenue was RMB407.2 million for the year ended December 31, 2024, composed of RMB175.1 million in sales of pharmaceutical products (avapritinib and pralsetinib), RMB204.0 million in license fee income and RMB28.1 million in royalty income of sugemalimab, representing a year-on-year increase of RMB108.3 million, or 113.1%, in license fee which was largely offset by a decrease in revenue from sales of pharmaceutical products, such that total revenue decreased by RMB56.6 million, or 12.2%, year on year.

• Research and development expenses were RMB134.7 million for the year ended December 31, 2024, representing a decrease of RMB393.1 million from RMB527.8 million for the year ended December 31, 2023, primarily due to the decrease in milestone fee and third-party contracting costs.

• Administrative expenses were RMB77.8 million for the year ended December 31, 2024, representing a decrease of RMB104.9 million from RMB182.7 million for the year ended December 31, 2023, primarily due to the decrease in employee costs.

• Selling and marketing expenses were RMB133.8 million for the year ended December 31, 2024, representing a decrease of RMB65.5 million from RMB199.3 million for the year ended December 31, 2023, primarily attributable to the decrease in employee costs and others which was partially offset by an increase in channel service fee.

• Loss for the year was RMB91.2 million for the year ended December 31, 2024, representing a decrease of RMB276.0 million, or 75.2%, from RMB367.2 million for the year ended December 31, 2023, primarily attributable to a substantial decrease in operating expenses.

• Cash and cash equivalents and time deposits were RMB672.9 million as of December 31, 2024.

Non-International Financial Reporting Standards (Non-IFRS) Measures:

• Research and development expenses excluding the share-based payment expenses were RMB124.7 million for the year ended December 31, 2024, representing a decrease of RMB410.0 million from RMB534.7 million for the year ended December 31, 2023, primarily due to the decrease in milestone fee and third party contracting costs.

• Administrative and selling and marketing expenses excluding the share-based payment expenses were RMB224.4 million for the year ended December 31, 2024, representing a decrease of RMB113.8 million from RMB338.2 million for the year ended December 31, 2023, primarily attributable to the decrease in employee costs.

• Loss for the year excluding the share-based payment expenses was RMB94.0 million for the year ended December 31, 2024, representing a decrease of RMB236.2 million, or 71.5%, from RMB330.2 million for the year ended December 31, 2023, primarily attributable to a substantial decrease in operating expenses.

2024 Annual Results Conference Call

The Company will host a 2024 annual results conference call at 10:00 a.m. (Beijing Time) on Friday March 28, 2025. Please attend the conference call through the link: View Source (Password:067784).

On March 27, 2025 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported that the first patient in Cohort 4 of its Phase I clinical trial has received their initial dose (Press release, TransCode Therapeutics, MAR 27, 2025, View Source [SID1234651545]). TransCode also reported that two additional patients in Cohort 4 are scheduled to receive TTX-MC138. The therapeutic candidate being evaluated, TTX-MC138, is TransCode’s lead candidate designed to inhibit microRNA-10b, a microRNA critical to the emergence and progression of metastatic cancer. No significant safety or dose limiting toxicities have been reported. The dose administered to the fourth cohort, as originally planned in the clinical protocol, is approximately fifty percent higher than the dose administered in the third cohort.

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Under the clinical protocol, patients may remain on study absent safety events or disease progression. Of the ten patients treated with TTX-MC138 in the first four cohorts, seven remain on study for continued treatment as there have been no dose limiting toxicities or disease progression in these patients. In addition to approving opening the fourth cohort, the Safety Review Committee approved enrollment of additional patients in Cohort 3 to build upon the safety profile of TTX-MC138. Further, data analysis of both pharmacokinetic (PK) and pharmacodynamic (PD) activity from Cohorts 1, 2 and 3 is ongoing and suggests that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode’s Phase 0 clinical trial.

"Commencement of treatment in Cohort 4 has been our vision for evaluating the potential of TTX-MC138 at multiple dose levels. Cohort 4 builds upon our safety, PK/PD and exploratory data and is an important element of our clinical development strategy. We believe this milestone will inform the dose expansion stage of the clinical trial and may allow us to obtain initial evidence of clinical activity as the program continues to advance," commented Sue Duggan, TransCode’s Senior Vice President of Operations. Duggan added, "Enrollment into the study continues based on the cumulative safety data review. Additional Cohort 4 patients are scheduled for treatment in Cohort 4 for treatment with TTX-MC138 while preliminary data analysis continues."

About TTX-MC138

TTX-MC138 is a first-in-class therapeutic candidate that targets microRNA-10b, a micro-RNA widely believed to be a driver of metastatic disease. TransCode’s 2023 Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.

About the Trial

TransCode’s Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study, designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers. While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138. The trial comprises an initial dose-escalation phase followed by a dose-expansion phase. The primary objective of the dose-escalation phase is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion phase, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types selected based on preliminary results from the dose-escalation phase.

Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).

On March 27, 2025 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, reported ATNM-400, a novel, non-PSMA targeting, first in class radiotherapy for prostate cancer utilizing the Actinium-225 (Ac-225) radioisotope (Press release, Actinium Pharmaceuticals, MAR 27, 2025, View Source [SID1234651544]). Initial preclinical data from ATNM-400 will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting being held on April 25 – 30, 2025, in Chicago, IL. The ATNM-400 AACR (Free AACR Whitepaper) abstract will include the results from in vitro and in vivo studies including biodistribution imaging and efficacy analyses with various dose levels of ATNM-400. Actinium continues to advance ATNM-400 with additional data expected from Pluvicto-resistant prostate cancer models at AACR (Free AACR Whitepaper). Pluvicto (Lu-177-PSMA-617) is a prostate-specific membrane antigen (PSMA) directed targeted radiotherapy that uses the beta-particle emitting radioisotope Lutetitium-177 (Lu-177) that is approved for patients with metastatic prostate cancer. Pluvicto is marketed and sold by Novartis and generated sales of $1.39 billion in 2024. ATNM-400 is differentiated from Pluvicto as it targets a different marker than PSMA that has been shown to be overexpressed in patients with prostate cancer and uses the alpha-particle emitter Ac-225, which is more potent than Lu-177 but has a shorter path length, which could result in fewer off-target effects.

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Sandesh Seth, Actinium’s Chairman and CEO, said, "The current era of radiotherapy is built on the clinical and commercial success of Pluvicto in prostate cancer. The field is now looking to address patients that do not respond or progress after Pluvicto therapy. We believe ATNM-400 can address this high unmet need and we are incredibly excited by our data to date. As anticipated, we have seen robust tumor control and ATNM-400 has shown to be well tolerated in preclinical studies, which we believe is due to the precise and potent cell-killing of Ac-225. We are also highly excited by the results of our biodistribution studies that showed selective tumor uptake with minimal uptake in normal tissues. By focusing on a non-PSMA target, we also believe ATNM-400 has the potential to address some of the toxicities reported with Pluvicto and other PSMA targeting radiotherapies such as xerostomia. We are eager to present our ATNM-400 data at AACR (Free AACR Whitepaper) and to continue to advance this highly novel prostate cancer candidate."

Highlights from the abstract include:

ATNM-400 selectively binds to prostate cancer cells, undergoes rapid internalization, and induces dose-dependent cytotoxicity
In prostate cancer xenograft mouse models, ATNM-400 accumulated in tumors for up to 144 hours, while showing minimal uptake in normal tissues
Small animal SPECT/CT imaging with Indium-111-labeled antibody confirmed selective tumor accumulation and clearance from healthy tissues
A single dose of ATNM-400 achieved 68.5% tumor growth inhibition at 20 µCi/kg and 99.8% at 40 µCi/kg, with all doses being well tolerated
ATNM-400 AACR (Free AACR Whitepaper) Presentation Details

Title: ATNM-400 is a novel Actinium-225 antibody radioconjugate with strong efficacy in preclinical models of prostate cancer

Abstract Number: 578

Session: PO.ET08.01 – Theranostics and Radiotheranostics

Date & Time: April 27, 2025 – 2:00 pm – 5:00 pm

On March 27, 2025 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported its financial results for 2024, and provides an update on its product pipeline and outlook for 2025 (Press release, Transgene, MAR 27, 2025, View Source [SID1234651543]).

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"By demonstrating clinical proof of principle for TG4050, Transgene’s first individualized therapeutic cancer vaccine derived from the myvac platform, we achieved a significant milestone in 2024. The highly encouraging early results from the Phase I part of our Phase I/II trial of TG4050 presented at SITC (Free SITC Whitepaper) 2024 have enabled the initiation of the ongoing Phase II part of the study. Enrollment is progressing well, reflecting strong support from both patients and clinicians for this novel candidate. We are confident that this Phase II trial will allow us to further confirm our promising initial data. We are monitoring innovation in the adjuvant setting of operable head and neck cancer and assessing the potential next steps to further accelerate our program."

"In 2025, we will continue to expand the potential of myvac. Our goal is to launch at least one new clinical trial in a second indication by year-end and continue optimizing our design and manufacturing processes. This innovative platform leverages the power of the MVA vector, the latest innovations in AI-powered neoantigen prediction and our strong bioengineering environment to design to design a tailored therapy for each patient and allows us to take advantage of the current momentum in individualized cancer vaccines."

"Driven by these technology advancements and supported by an innovation-driven team, we remain committed to executing our growth strategy. We are confident that our focus on viral vector-based immunotherapies will enable us to deliver transformative benefits to a broad spectrum of cancer patients," commented Dr. Alessandro Riva, MD, Chairman and CEO of Transgene.

Key events and upcoming milestones

Individualized neoantigen therapeutic cancer vaccine (TG4050)

Significant progress has been made with Transgene’s myvac individualized cancer vaccine program in 2024:

ü Proof of principle data from Phase I part of the Phase I/II trial of TG4050 in the adjuvant setting of head and neck cancer. All TG4050-treated patients remained disease-free after median follow-up of 24.1 months (compared to three relapses in the control arm);
ü Phase II part of study launched in June 2024 based on these promising early data.

Positive data from Phase I part:

Transgene and NEC presented promising data from the ongoing randomized Phase I part of the Phase I/II trial (NCT04183166) of the neoantigen individualized therapeutic cancer vaccine, TG4050, at AACR (Free AACR Whitepaper) 2024 and at SITC (Free SITC Whitepaper) 2024 (see poster here).

In the Phase I part of the trial, all patients who received TG4050 after successful completion of adjuvant standard of care, remained disease-free and had not relapsed after a median follow-up of 24.1 months, comparing favorably to the observational arm which showed three out of 16 patients had relapsed (data cut-off: end of September 2024).

Transgene and Institut Curie also presented compelling immunogenicity data in patients, showing the induction of specific immune responses against selected personalized antigen targets. Additionally, immune responses were shown to be sustained over a 7-month period.

In this trial, primary objectives were safety and tolerability. Feasibility and disease-free survival (DFS) were secondary objectives. Exploratory objectives included immunogenicity and assessment of tumor biomarkers (TMB, PD-L1).

These data provide robust clinical proof of principle for Transgene’s lead asset in the adjuvant head and neck cancer setting, a patient population at risk of relapse.

Progress into Phase II part:

Based on the promising Phase I data, the randomized trial has progressed, with the Phase II part having started patient enrollment in June 2024, in collaboration with NEC.

Patient enrollment is progressing at a good pace and completion of randomization is expected in Q4 2025. In this trial, the primary objective is 24-month DFS (disease-free survival).

Upcoming news flow for TG4050 and the myvac platform:

Transgene’s objective for TG4050 is to extend DFS and reduce the risk of relapse. The Company will present 24-month DFS for all patients in the Phase I part of the Phase I/II trial in Q2 2025. In locally advanced, resectable head and neck cancers, 25% of patients are expected to relapse within 24 months after successful completion of surgery and adjuvant chemoradiotherapy (Cooper JS et al. NEJM, 2004; DY Lee et al. Head Neck, 2020).

These updated clinical data combined with innovation in the adjuvant treatment of operable head and neck cancer will be instrumental in determining TG4050’s optimal development path towards registration in this indication.

The myvac individualized cancer vaccine platform is applicable across a range of solid tumors where a significant unmet medical need remains, despite current and future treatment options, including immunotherapies.

Consequently, Transgene is starting initial preparations for a new Phase I trial in a second undisclosed indication, with the aim to initiate the trial in Q4 2025.

Other viral vector-based assets

Shared antigens cancer vaccine (TG4001)

In October 2024, Transgene announced that its randomized Phase II study evaluating TG4001 in combination with avelumab versus avelumab alone in patients with recurrent or metastatic HPV16-positive cervical and anogenital tumors did not meet its primary objective (improvement in progression-free survival).

However, analysis of a pre-planned subgroup of patients showed a positive efficacy trend in favor of the TG4001 containing regimen in cervical cancer patients.

Transgene is currently evaluating the full clinical and translational study results to determine the best way forward for this program. Clinical data from this trial will be presented at a scientific congress in Q2 2025.

BT-001 (oncolytic virus — intratumoral administration)

The Phase I/IIa trial (NCT04725331) is ongoing and the last patient in the Phase I part was enrolled in August 2024. In Part A of the trial, patients are given BT-001 as monotherapy, while in Part B, patients are given BT-001 in combination with pembrolizumab. In this part, KEYTRUDA (pembrolizumab) is provided by MSD (Merck & Co). KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

Preliminary data were presented at ESMO (Free ESMO Whitepaper) 2024 (see press release here). The data indicated that BT-001 replicated in the tumor without being detectable in blood. As monotherapy and in combination with pembrolizumab, BT-001 was shown to be well tolerated. BT-001 also showed first signs of efficacy with clinical response in two out of six refractory patients, when given in combination with pembrolizumab, with shrinkage of injected and non-injected lesions. Treatment with BT-001 converted "cold" tumors into "hot" ones, and induced T-cell infiltration, as well as PD(L)-1 expression in the tumor microenvironment. Transgene and partner BioInvent are currently analyzing the second cohort of Part B of the Phase I to define the strategy for further development. Updated data is expected to be presented in H2 2025.

TG6050 (oncolytic virus — intravenous administration)

The Phase I Delivir trial, evaluating TG6050 in patients with advanced non-small cell lung cancer who have failed standard therapeutic options, has completed enrollment.

Initial data from the Phase I trial are expected to be reported in Q2 2025. Transgene will complete the analysis of these data to determine the best way forward for this candidate.

In July 2024, Transgene published preclinical data in the Journal for ImmunoTherapy of Cancer (JITC) (see article here), where the paper on TG6050 won the JITC Best Oncolytic and Local Immunotherapy Paper Award. The study demonstrated that TG6050 induces tumor regression in several "hot" and "cold" mouse tumor models. This antitumor activity was amplified when TG6050 was combined with an immune checkpoint inhibitor.

Transgene’s new leadership structure focused on accelerating the development of its innovative immunotherapy portfolio

To drive its ambitious strategic plan centered on the individualized cancer vaccine platform myvac, Transgene has gathered an expert leadership team.

Transgene’s Management Committee comprises the following members:

– Alessandro Riva, Chairman & Chief Executive Officer (CEO);

– Christophe Ancel, Chief Quality Officer & Qualified Pharmacist;

– Maurizio Ceppi, Chief Scientific Officer (CSO) (as of September 2024);

– Emmanuelle Dochy, Chief Medical Officer (CMO) (as of September 2024);

– John Felitti, General Counsel & Corporate Secretary;

– Lucie Larguier, Chief Financial Officer (CFO) (as of March 2024);

– Christelle Schwoerer, Chief Human Resources Officer (as of April 2024);

– Simone Steiner, Chief Technical Officer (CTO) (as of April 2025);

– James Wentworth, Chief Business Officer (CBO) (as of January 2024).

Key elements of the 2024 income statement

Operating revenue was €6.4 million in 2024 compared to €7.9 million in 2023. Operating revenue was mostly comprised of the Research Tax Credit (€6.0 million in 2024 and €6.4 million in 2023). The reduction in total operating revenue reflects the discontinuation of the AstraZeneca collaboration in 2023.
Net operating expenses stood at €42.0 million in 2024, compared to €37.9 million in 2023, reflecting intense activity on all clinical-stage candidates, including the preparation and launch of the Phase II part of the trial evaluating TG4050 in head and neck cancer. This activity is reflected in R&D expenses, at €34.3 million in 2024 versus €29.6 million in 2023. General and administrative expenses amounted to €7.8 million in 2024 (€7.0 million in 2023).
In 2024, the operating loss was €35.7 million, compared to a loss of €30.0 million in 2023.
Net loss was €34.0 million in 2024, compared to a net loss of €22.3 million in 2023.
The net cash burn was €27.7 million in 2024, compared to €24.0 million in 2023.
Cash available at year-end 2024 stood at €16.7 million, compared to €15.7 million at the end of 2023.

Business funded until the end of April 2026

The Company has signed a new amendment to the current account advance agreement with TSGH (Institut Mérieux), which increases the total amount of the facility by €15 million to €48 million. The Company has drawn down €22.5 million from this facility as of today.

With this credit facility and the support of TSGH (Institut Mérieux), the Company is now able to fund its business until the end of April 2026.

In July 2024, TSGH subscribed to Transgene a €33 million capital increase and requested the reimbursement of an equivalent amount from the current account advance. This reimbursement was carried out by way of set-off against the payment of the subscription price of the capital increase by TSGH.

The financial statements for 2024 as well as management’s discussion and analysis are attached to this press release (appendices A and B).

The Board of Directors of Transgene met on March 27, 2025, under the chairmanship of Dr. Alessandro Riva and closed the 2024 financial statements. Audit procedures have been performed by the statutory auditors and the auditor’s reports are in the process of being issued.

The Company’s universal registration document (URD), which includes the annual financial report, will be available early April 2025 on Transgene’s website, www.transgene.fr.

A conference call in English is scheduled today on March 27, 2025, at 6:00 p.m. CET (1:00 p.m. ET).

Webcast link to English language conference call:

View Source

On March 27, 2025 Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, reported financial results for the fourth quarter and full year ended December 31, 2024, and highlighted recent corporate progress (Press release, Tyra Biosciences, MAR 27, 2025, View Source [SID1234651541]).

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"2024 was a momentous year for TYRA and the patient communities we serve, highlighted by the positive interim results from our SURF301 study, which demonstrated a combination of high anti-tumor activity with favorable tolerability results in very sick, heavily pre-treated cancer patients. Importantly, the oncology doses tested in SURF301 are significantly higher than those to be tested in BEACH301, giving us confidence as we advance TYRA-300 in ACH," said Todd Harris, CEO of TYRA. "Our conviction in TYRA-300 has never been stronger and we are working diligently to advance this potential best-in-class agent for multiple high-value indications in oncology and skeletal dysplasia into three Phase 2 studies in NMIBC, ACH and mUC."

Fourth Quarter and Full Year 2024 and Recent Corporate Highlights

TYRA-300

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Advanced Clinical Evaluation of TYRA-300 into Three Phase 2 Studies. During 2024, TYRA progressed TYRA-300, an oral, investigational FGFR3-selective inhibitor, for the treatment of IR NMIBC, mUC and ACH, and achieved the following milestones:
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Cleared Phase 2 NMIBC IND with US FDA – SURF302. TYRA expanded the clinical development of TYRA-300 into NMIBC to address the unmet needs in this cancer population for an efficacious, orally available therapy. SURF302 is an open-label Phase 2 clinical study evaluating the efficacy and safety of TYRA-300 in participants with FGFR3-altered low-grade, IR NMIBC. The study will enroll up to 90 participants at multiple sites primarily in the United States. Participants will be randomized initially to treatment with TYRA-300 at 50 mg once-daily (QD) (Cohort 1) or treatment with TYRA-300 at 60 mg QD (Cohort 2). Following a review of efficacy and safety, an additional dosing cohort may be evaluated. The primary endpoint is complete response (CR) rate at three months. Secondary endpoints include time to recurrence, the median duration of response, recurrence free survival (RFS), progression free survival (PFS), safety and tolerability.
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Cleared Phase 2 ACH IND with US FDA – BEACH301. The study is a Phase 2, multicenter, open-label, dose-escalation/dose-expansion study evaluating TYRA-300 in children ages 3 to 10 with achondroplasia with open growth plates. The study will enroll children who are treatment-naïve (Cohort 1) and those who have received prior growth-accelerating therapy (Cohort 2) at multiple sites across the globe. Each of these cohorts is expected to enroll up to 10 participants per dose level (0.125, 0.25, 0.375, 0.50 mg/kg) for up to 12 months. The study will initially enroll a safety sentinel cohort of up to 3 treatment-naïve participants per dose level in children ages 5 to 10.

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Reported Interim Clinical Proof-of-Concept Results in mUC Patients – SURF301. TYRA-300 demonstrated encouraging preliminary anti-tumor activity in a heavily pre-treated population: at ≥ 90 mg QD, 6 out of 11 (54.5%) patients with FGFR3+ mUC achieved a confirmed partial response (PR), with 100% disease control rate and sustained duration of activity; positive safety results were reported across all QD doses, with infrequent FGFR2/FGFR1-associated toxicities (data cutoff of August 15, 2024). TYRA-300 is being evaluated in Part B of SURF301 (NCT05544552) at potentially therapeutic QD doses in preparation for potential future Phase 2 studies.

TYRA-200

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Advanced Phase 1 SURF201 Study. TYRA-200 is an FGFR1/2/3 inhibitor with potency against activating FGFR2 gene alterations and resistance mutations. SURF201 (Study in PrevioUsly treated and Resistant FGFR2+ Cholangiocarcinoma and Other Advanced Solid Tumors) (NCT06160752) is a multi-center, open label study designed to evaluate the safety, tolerability, and pharmacokinetics of TYRA-200 and determine the optimal and maximum tolerated dose and recommended Phase 2 dose, as well as evaluate the preliminary antitumor activity of TYRA-200. The SURF201 study is currently enrolling and dosing adults with unresectable locally advanced/metastatic intrahepatic cholangiocarcinoma and other advanced solid tumors with activating FGFR2 gene alterations.

TYRA-430

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Cleared Phase 1 IND with US FDA – SURF431. TYRA-430 is an oral, investigational FGFR4/3-biased inhibitor for FGF19+/FGFR4-driven cancers. The Phase 1 study will be a multicenter, open-label, first-in-human study of TYRA-430 in advanced hepatocellular carcinoma (HCC) and other solid tumors with activating FGF/FGFR pathway aberrations (SURF431). We believe TYRA-430 has the potential to address a significant unmet need in HCC, where there are no approved biomarker-driven, targeted therapies.

Corporate

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Strengthened Leadership Team and Board of Directors. In 2024, TYRA appointed Doug Warner, MD, as Chief Medical Officer, and Erik Goluboff, MD, as SVP, Clinical Development to lead the Company’s oncology strategy and clinical development plans. In 2025, TYRA appointed accomplished drug developer Adele Gulfo to its Board of Directors, Sinette Heys as SVP, Clinical Operations to lead the Company’s clinical operations team, and Will Charlton, MD, as SVP, Clinical Development to lead the Company’s skeletal dysplasia clinical development group.

SNÅP Platform and Pipeline

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TYRA continued to advance its in-house precision medicine discovery engine, SNÅP, to develop therapies in targeted oncology and genetically defined conditions.

Fourth Quarter and Full-Year 2024 Financial Results

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Cash, Cash Equivalents and Short-Term Investments. As of December 31, 2024, TYRA had cash, cash equivalents, and marketable securities of $341.4 million, compared to $203.5 million at the end of 2023. The increase was primarily due to the completion of a private placement financing for net proceeds of $199.6 million in the first quarter of 2024. The Company’s current cash, cash equivalents and marketable securities are expected to allow TYRA to execute on its plans through at least 2027.
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Research and Development (R&D) Expenses. Research and development expenses for the three months ended December 31, 2024 were $22.2 million compared to $20.7 million for the same period in 2023, and $80.1 million for the full year 2024 compared to $62.5 million for the same period in 2023. The increases were primarily driven by increased expenses incurred in connection with our ongoing and planned clinical trials and personnel-related costs, including stock-based compensation, partially offset by decreased drug manufacturing and preclinical costs.
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General and Administrative (G&A) Expenses. General and administrative expenses for the three months ended December 31, 2024 were $7.6 million compared to $5.0 million for the same period in 2023, and $24.1 million

for the full year 2024 compared to $17.4 million for the same period in 2023. The increases were primarily driven by increased personnel-related costs, including stock-based compensation.
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Net Loss. Fourth quarter 2024 net loss was $25.6 million compared to $22.8 million for the same period in 2023, and $86.5 million for the full year 2024 compared to $69.1 million for the same period in 2023.

Upcoming Anticipated Milestones and Events

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BEACH301: dose first child with achondroplasia with TYRA-300 – Q2 2025
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SURF302: dose first NMIBC patient with TYRA-300 – Q2 2025
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SURF431: dose first HCC patient with TYRA-430 – Q2 2025

About TYRA-300

TYRA-300 is the Company’s lead precision medicine program stemming from its in-house SNÅP platform. TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasia, including achondroplasia and hypochondroplasia. In oncology, TYRA-300 is being evaluated in mUC and IR NMIBC. In mUC, TYRA-300 is being evaluated in a multi-center, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors) (NCT05544552). The study is designed to determine the optimal and the recommended Phase 2 dose of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. In October 2024, TYRA reported interim clinical proof-of-concept data in mUC from SURF301. TYRA has received IND clearance from the US FDA to proceed with its SURF302 clinical trial in patients with IR NMIBC. In skeletal dysplasia, TYRA-300 has demonstrated positive preclinical results in achondroplasia and hypochondroplasia, and its BEACH301 clinical trial in children with achondroplasia is now recruiting.

About TYRA-200

TYRA-200 is an oral, investigational, FGFR1/2/3 inhibitor with potency against activating FGFR2 gene alterations and resistance mutations. The Phase 1 clinical study of TYRA-200, SURF201 (Study in PrevioUsly treated and Resistant FGFR2+ Cholangiocarcinoma and Other Advanced Solid Tumors) (NCT06160752), is a multi-center, open label study designed to evaluate the maximum tolerated dose (MTD) and the recommended Phase 2 dose of TYRA-200, as well as to evaluate the preliminary antitumor activity of TYRA-200. SURF201 is currently enrolling and dosing adults with advanced/metastatic intrahepatic cholangiocarcinoma and other advanced solid tumors with activating alterations in FGFR2.

About TYRA-430

TYRA-430 is an oral, investigational FGFR4/3-biased inhibitor for FGF19+/FGFR4-driven cancers. The US FDA has cleared Tyra’s IND to proceed with a Phase 1 clinical study of TYRA-430. The Phase 1 study will be a multicenter, open-label, first-in-human study of TYRA-430 in advanced HCC and other solid tumors with activating FGF/FGFR pathway aberrations (SURF431).