On February 22, 2024 DermBiont, a clinical-stage biotechnology company that is advancing targeted topical therapeutics to address patient needs in three of the most frequently diagnosed dermatological indications, reported updates on the company’s development pipeline, including the completion of enrollment in a Phase 2b clinical trial of SM-020 gel 1.0% for the treatment of SKs, and anticipated clinical milestones for 2024 (Press release, DermBiont, FEB 22, 2024, View Source [SID1234640407]).

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"We are pleased to have completed enrollment in our CT-213 clinical trial evaluating SM-020 gel 1.0% for the treatment of SK lesions, the most common benign tumors of the skin," commented Karl Beutner, M.D., Ph.D., CEO, and Co-Founder of DermBiont, adding "We believe that positive results from this trial will keep us on track for an End of Phase 2 Meeting with the FDA and ultimate regulatory alignment for designing DermBiont’s Phase 3 pivotal trial."

CT-213 is a Phase 2b randomized, double-blind, vehicle-controlled clinical trial in 60 subjects with five to ten SK target lesions treated with SM-020 gel 1.0% or vehicle twice daily for 28 days. The primary endpoints of the study are the proportion of SK lesions with a Physician Lesion Assessment (PLA) score of clear (PLA=0) at last visit and the safety of SM-020 gel 1.0% based on application site reactions. Key secondary endpoints include the proportion of SKs with clear (PLA=0) or nearly clear (PLA=1), proportion of subjects with 100% of lesions clear (PLA=0), proportion of subjects with 60% of lesions clear (PLA=0), and the diagnostic accuracy of clinical versus dermoscopy assessments.

DermBiont currently anticipates that data will be available in Q3 2024. The data generated in CT-213 as well as clinical and non-clinical work, including various formulation and development studies, will support an End of Phase 2 Meeting request package to the FDA in Q4 2024.

"The data we have generated to date treating SKs with SM-020 gel 1.0% is highly encouraging, providing a safe and effective treatment option for patients whose only other option would be painful ablative surgical procedures that require multiple days of post-operative wound care and carry meaningful risk of scarring," stated Nichola Eliovits, Co-Founder and Chief Business Officer of DermBiont.

Additionally, DermBiont is initiating a Phase 2a (CT-217) open-label trial in up to 40 subjects with one to five basal cell carcinomas (BCC) or squamous cell carcinoma in situ (SCCIS). This open label trial’s primary endpoints are the reduction in greatest tumor diameter at week 6 compared to baseline, and the safety of drug product for BCCs or SCCISs based on application site reactions. Secondary endpoints include the percentage of BCCs or SCCISs that achieve histologic cure at week 6.

"We are excited to initiate the CT-217 trial evaluating efficacy and safety in subjects with nodular, superficial, and sclerosing BCC tumors, following an initial finding where a BCC lesion was highly responsive to treatment with this drug product. A detailed analyses of the drug’s mechanism of action demonstrated activity on the pathways known to be the cause of BCCs," said Emma Taylor, M.D. and Chief Medical Officer at DermBiont. Adding, "If our initial finding is confirmed, a patient applied targeted topical product with excellent safety and tolerability would represent a major breakthrough for patients suffering from BCCs (the most frequently diagnosed skin cancer), especially for patients with Gorlin Syndrome."

Rounding out this year’s clinical activities, DermBiont is commencing a randomized, observer-blind, vehicle-controlled Phase 2b trial (CT-214) of SM-030 in 138 subjects with melasma in Q2 2024. The CT-214 trial follows positive results in an earlier completed Phase 2a study treating solar lentigos and photoinduced hyperpigmentation, demonstrating comparable efficacy to hydroquinone with superior tolerability and safety given SM-030’s targeted mechanism of action and increasing concerns over hydroquinone’s long-term safety and toxicity.

Melasma and other hyperpigmentation disorders of the skin are typically recurring and chronic skin conditions. While lasers can provide some benefit, they are expensive, and only provide temporary improvement, predominantly to dermal melanin. Patients frequently experience recurrence following laser or cessation of hydroquinone therapy, leaving patients with little to no long-term maintenance or treatment solutions. However, SM-030 works as a targeted topical to safely downregulate excess production of melanin by melanocytes in the epidermis, addressing the root cause of excess melanin production, and offering patients a viable long-term treatment option to obtain and maintain monochromatic skin.

Upcoming Milestones:

SKs: DermBiont expects to report data from its CT-213 Phase 2b clinical trial of SM-020 gel 1.0% in Q3 2024.
BCCs and SCCISs: DermBiont expects to initiate its CT-217 Phase 2a clinical trial in Q1 2024 and report data in Q3 2024.
Melasma: DermBiont anticipates enrolling the first patient in its randomized, observer-blinded, vehicle-controlled CT-214 Phase 2b trial of SM-030 in 138 subjects with melasma in Q2 2024

On February 22, 2024 Kling Biotherapeutics ("Kling" or "the Company"), a clinical stage biotech company developing antibody-based drugs against targets identified from its primary B-cell screening platform, reported that it will be presenting findings demonstrating the power of its clinically validated, best-in-class, B cell screening platforms, Kling-Select and Kling-Evolve, at the ESMO (Free ESMO Whitepaper) Targeted Anticancer Therapies Congress, in Paris, France on 26 February (Press release, Kling Biotherapeutics, FEB 22, 2024, View Source [SID1234640406]).

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Michael Koslowski, Chief Executive Officer at Kling Bio, said: "Our findings consistently highlight and validate our world class, proprietary primary B cell screening platform and its ability to efficiently identify novel therapeutic targets and antibodies. Our aim is to maximise disease fighting antibodies and find a cure for all patients battling illnesses which include cancer and infectious diseases. Our lead clinical candidate KBA1412, a unique and previously undiscovered epitope of CD9, is advancing in a Phase 1 study, demonstrating a clean and safe profile in patients with advanced solid tumors. We look forward to sharing data in the foreseeable future and exploring the therapeutic potential of snRNP200 and our uniquely identified CD43 glycoform."

Poster one, entitled "KBA1413, an antibody derived from a cured AML patient, recognizes a unique CD43 glycoform shared by AML, MDS and solid cancer cells," details the identification of KBA1413, a fully human antibody screened from a single acute myeloid leukemia (AML) patient who remains in long term remission following allogeneic hematopoietic stem cell transplantation. The antibody identified using Kling’s proprietary B cell screening platform, Kling-Select, recognised a unique, previously undescribed, sialylated epitope on CD43 that is reactive to freshly isolated blasts of over 60 randomly selected AML and myelodysplastic syndrome (MDS) patients. It also recognised CD43 on several solid tumor indications, including melanoma and breast cancer. KBA1413 triggered TR NK-mediated antibody dependent cell-mediated cytotoxicity (ADCC) against AML cells both in vitro and in vivo, suggesting that the antibody played a role in the graft versus leukemia response of the original donor patient. KBA1413 was made into a bispecific T-cell engaging antibody (bTCE), to increase its therapeutic potential and induced potent cytotoxicity in vitro and in vivo on cell lines in primary AML. Data demonstrates that KBA1413 warrants further development as a potent and promising treatment candidate for AML, MDS and solid tumors.

Poster two, entitled "snRNP200 reactive antibodies isolated from multiple AML patients show promise for targeted therapy," details how Kling’s proprietary Kling-Select platform has the ability to identify new therapeutic target-antibody pairs to improve patient outcomes by interrogating the B cell repertoire of patients that have achieved remission via a strong tumor-directed immune response.

The Kling-Select platform was used to discover and characterise anti-snRNP200 antibodies from three AML patients that have achieved complete response after allogeneic stem cell transplantation, establishing that anti-snRNP200 specifically and selectively bind to AML cell lines that display snRNP200 on the outer cell surface. These findings have been independently confirmed[2] and show that snRNP200 surface expression is limited to malignant cells and not expressed on normal hematopoietic stem cell progenitors. Kling will further explore the therapeutic potential of anti-snRNP200 with affinity matured variants produced with Kling-Evolve.

A first-in-human, Phase 1 clinical trial on Kling’s first-in-class anti-CD9 antibody in patients with advanced solid malignant tumors is ongoing and can be found on www.clinicaltrials.gov under the identifier NCT05501821.

All accepted abstracts will be published online only in the ESMO (Free ESMO Whitepaper) TAT 2024 Abstract Book, a supplement to the ESMO (Free ESMO Whitepaper) journal, ESMO (Free ESMO Whitepaper) open.

Details of the conference and poster presentations are as follow:

Poster Title: KBA1413, an antibody derived from a cured AML patient, recognizes a unique CD43 glycoform shared by AML, MDS and solid cancer cells
Authors: V. Clerico Mosina, M. Kedde, B. Pieters, B. Monica, K. Maijoor, R. Schotte, A. Bakker, M. Koslowski, S. Gullà[1]
Date and Time: 26 February, 17:15-18:15 CET
Session: Cocktail and Poster Display Session
Location: Hall Bordeaux
Presentation Number: 11P

Poster Title: snRNP200 reactive antibodies isolated from multiple AML patients show promise for targeted therapy
Authors: S. Baumann, M. Kedde, B. Monica, K. Maijoor, R. Schotte, A. Bakker, J. M. Koslowski, S. Gullà[1]
Date and Time: 26 February, 17:15-18:15 CET
Session: Cocktail and Poster Display Session
Location: Hall Bordeaux
Presentation Number: 11P

On February Medivir AB (Nasdaq Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, reported that clinical pharmacokinetic (PK) data on dose linearity and systemic exposure from the first study with fostroxacitabine bralpamide (fostrox) (NCT03781934) will be presented at the European Association for the Study of the Liver (EASL) Liver Cancer Summit on February 23, 2024, in Rotterdam (Press release, Medivir, FEB 22, 2024, View Source [SID1234640405]).

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The abstract, titled "Population pharmacokinetic modeling of orally administered fostroxacitabine bralpamide (fostrox, MIV-818) and its metabolite troxacitabine in a phase I/IIa liver cancer study" will be presented at the conference by Karin Tunblad PhD, Project Director for fostrox at Medivir. The presentation includes pharmacokinetic results from 42 patients in the phase I/IIa clinical study with fostrox monotherapy and the fostrox + Lenvima combination in advanced liver cancer.

The results show dose linearity and that the systemic exposure, at the recommended phase 2 dose for fostrox, aligns with the encouraging safety and tolerability profile experienced in the ongoing phase 1b/2a study with fostrox in combination with Lenvima in second line treatment of advanced hepatocellular carcinoma (HCC). In addition, the study shows that the exposure of fostrox is not impacted by administration of Lenvima, providing additional support for this combination.

– "Ensuring predictable exposure is crucial when combining two pharmaceutical drugs. The data presented at EASL strengthen the clinical results previously reported where patients treated with fostrox + Lenvima have shown good tolerability and ability to stay on treatment long-term. The data further supports our upcoming regulatory interactions with FDA and continues to build our confidence in the clinical development of fostrox," says Pia Baumann, Chief Medical Officer at Medivir.

The abstract and the poster will be available on Medivir’s website after the presentation.

On February 22, 2024 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported its results for the financial year ended 31 December 2023. All figures are in AUD$ unless otherwise stated (Press release, Telix Pharmaceuticals, FEB 22, 2024, View Source [SID1234640404]).

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2023 highlights

Total Group revenue of $502.5M, an increase of 214% from $160.1M in 2022 primarily driven by continued strong growth in sales of Illuccix in the second year since commercial launch (April 2022)
Delivered positive adjusted earnings before interest, tax, depreciation, and amortisation (adjusted EBITDA) of $58.4M an increase of $126.2M, compared to a loss of $67.8M in 2022
Inaugural full year profit of $5.2M after tax. A substantial improvement on the net loss after tax of $104.1M in 2022
Investment in research and development (R&D) and selling, general and administration (SG&A) reflects progress across the late-stage pipeline and scale-up of the commercial organisation
Overall operating costs as a percentage of revenue have reduced to 52% from 105% in 2022
Gross margin has improved to 63% (vs. 59% in 2022) reflecting distribution and manufacturing costs optimisation
Positive operating cash inflow in line with commercial sales growth, demonstrated through customer receipts of $463.7M (vs. $124.1M in 2022), and
Closing cash balance was $123.2M as at 31 December 2023.
Progress across the core pipeline includes:

First patients dosed in the ProstACT GLOBAL Phase III clinical trial of TLX591, a first-in-class investigational rADC[2] for prostate cancer therapy
Positive interim readout from the ProstACT SELECT Phase I clinical trial reinforcing the differentiation of TLX591, including favourable safety profile and clinical utility of the patient-friendly short dosing regimen
Submission of the Biologics License Application (BLA) for TLX250-CDx (Zircaix[3]) to the United States Food and Drug Administration (U.S. FDA) for kidney cancer imaging on a rolling review basis
Patients dosed in multiple clinical trials of Telix’s therapeutic candidate TLX250 for clear cell renal cell carcinoma (ccRCC) and other solid tumours expressing carbonic anhydrase IX (CAIX)
First cohort of patients dosed in the IPAX-2 trial investigating Telix’s glioblastoma therapy candidate, TLX101, in newly diagnosed patients
Commercialisation plans underway for glioma imaging agent, TLX101-CDx (Pixclara[3]), ahead of the planned submission of a New Drug Application (NDA) to the U.S. FDA in Q1 2024, and
Ethics approval granted to commence biodistribution and safety study of TLX300-CDx, first human study of radiolabelled olaratumab being developed as a therapeutic candidate for soft-tissue sarcoma.
Dr Christian Behrenbruch, Managing Director and Group CEO commented:

"This is an excellent result which demonstrates the strength of the Telix business model. We have achieved profitability while intensively investing in the development of our late-stage assets and the scale-up of our commercial infrastructure and marketing activity. This has resulted in Telix capturing a meaningful market share in the growing urology imaging market whilst laying the foundation for our next commercial products.

"We are highly focused on the development of our theranostic pipeline and vertical integration of supply and manufacturing. This activity is key to diversifying our revenue streams, creates additional value for our therapeutic assets and further differentiates Telix as a fully integrated global radiopharmaceutical company."

Further details on the Company’s results can be found in the Appendix 4E, the accompanying investor presentation, and 2023 Annual Report lodged with the ASX and also available on the Company’s website.

Guidance

Full year revenue for 2024 expected range of US$445M to US$465M ($675M to $705M at current exchange rates), representing an approximate 35-40% increase on 2023.

Revenue guidance is based on worldwide sales of Illuccix, with potential upside from Zircaix[3] (kidney cancer imaging) and Pixclara[3] (glioma imaging), subject to product regulatory approvals. Guidance will be updated throughout the year, as appropriate, to reflect product approvals.

Expected additional investment of 40-50% in R&D (compared with 2023), including both external and internal costs funded by operating cash flow and broadly in line with revenue growth.

2024 R&D investment activity is expected to include validation of commercial manufacturing and market launch activities in preparation for approval of Zircaix[3] and Pixclara[3] a fully operationalised ProstACT GLOBAL therapy trial in prostate cancer, and initiation of additional therapeutic clinical trials, including manufacturing activity, across the broader pipeline. 2024 R&D investment also includes indication expansion and life-cycle management of Illuccix.

Investor call

An investor webcast will be held at 9.00am AEDT on Friday 23 February 2024 (Thursday 22 February 2024, 5.00pm EST)

Participants can register for the webcast and find audio call details at the following link: View Source

On February 22, 2024 STORM Therapeutics Ltd. (STORM), the clinical stage biotechnology company discovering and developing novel small molecule therapies targeting RNA modifying enzymes (RMEs) for oncology and other diseases, reported that Alexandra Sapetschnig, Group Leader at STORM, will present results and conclusions on its METTL1 tRNA methyltransferase program at the ESMO (Free ESMO Whitepaper) Targeted Anticancer Therapies Congress 2024 in Paris, France on 26 February (Press release, STORM Therapeutics, FEB 22, 2024, View Source [SID1234640403]).

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The presentation entitled "Targeting the tRNA methyltransferase METTL1 with small molecule inhibitors in cancer" illustrates STORM’s new data showing that pharmacological inhibition of a tRNA methyltransferase affects tumour growth in animal models.

Data demonstrated that:

Two distinct chemical series exhibit METTL1 inhibition in vitro at low nanomolar concentrations while displaying high selectivity over other RNA and protein methyltransferases.
Mechanistically, METTL1 inhibition leads to reduced tRNA methylation and stability of a subgroup of tRNAs.
In several cancer cell lines, METTL1 inhibition impairs cell proliferation and cell cycle progression accompanied by reduced expression of cell cycle regulators.
In vivo, METTL1 inhibitors induce tumour growth inhibition in both immune-deficient and immune-competent mouse strains.
Oliver Rausch, Chief Scientific Officer at STORM Therapeutics, said: "We are delighted to present this exciting new work which demonstrates that targeting specific tRNA pathways via inhibition of the novel RNA methyltransferase METTL1 results in cancer cell reprogramming and profound cancer growth inhibition in vivo. This follows hot on the heels of our groundbreaking work on METTL3 leading to the discovery of STC-15, currently in clinical testing for advanced malignancies, and highlights the immense potential of targeting RNA modifications for the development of new cancer treatments. As we continue to advance and develop our novel proprietary drug discovery pipeline, these new findings illustrate the advancements that STORM are making to transform the treatment landscape for cancer.

All accepted abstracts will be published online only in the ESMO (Free ESMO Whitepaper) TAT 2024 Abstract Book, a supplement to the ESMO (Free ESMO Whitepaper) journal, ESMO (Free ESMO Whitepaper) open.

Details of the conference and poster presentation are as follow:
Poster Title: Targeting the tRNA methyltransferase METTL1 with small molecule inhibitors in cancer
Presenter: Alexandra Sapetschnig1, Beth Thomas1, Eliza Yankova2, Harry Fischl1, Aleksandra Azevedo1, Sarah Bucknell1, Richard Fosbeary1, Sapphire Sawyer1, Sian Evans2, Carmen Livi1, Byron Andrews1, Jack Rogan1, Natalie Webster1, Matthew Fyfe1, Konstantinos Tzelepis2, Oliver Rausch1
Date and Time: 26 February 2024 at 17:15 – 18:15 CET
Session: Cocktail and Poster Display Session
Location: Hall Bordeaux
Presentation Number: 105P