On April 17, 2024 Evaxion Biotech A/S (NASDAQ: EVAX) ("Evaxion" or the "Company"), a clinical-stage TechBio company specializing in developing AI-Immunology powered vaccines, reported that the first patient in its EVX-01 Phase 2 trial in metastatic melanoma received the last vaccine dose in combination with KEYTRUDA (NCT05309421) (Press release, Evaxion Biotech, APR 17, 2024, View Source [SID1234642138]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Company initiated its Phase 2 clinical study in September 2022 to assess the efficacy, safety and ability to induce a tumor-specific immune response of the EVX-01 cancer vaccine in metastatic melanoma patients. The EVX-01 vaccine was designed using Evaxion’s proprietary AI-Immunology platform and is an individualized therapy matching the unique tumor profile and characteristics of the patient’s immune system. Each patient enrolled in the trial receives a unique vaccine designed and manufactured based on their individual biology. Patients are administered ten EVX-01 doses over a period of 78 weeks in combination with the anti-PD-1 therapy, KEYTRUDA (pembrolizumab).

Birgitte Rønø, CSO of Evaxion, commented, "With the progress made in the Phase 2 study, we are one step closer to fulfilling our mission of saving and improving lives with AI-Immunology. We eagerly anticipate sharing the one-year clinical readout in Q3 this year and look forward to being one step closer to market with a novel personalized cancer vaccine."

Professor Adnan Khattak at One Clinical Research, Hollywood Private Hospital, Western Australia, expresses enthusiasm, stating, "We are now entering into the era of personalized cancer therapies, where we adopt a tailored approach against an individual patient’s tumor. In other words, we are treating each patient with the right drug. As a physician, I firmly believe this is the future."

At the end of 2023, Evaxion reported initial EVX-01 Phase 2 data confirming the favorable safety profile and promising immunological data as observed in the previously successful Phase 1 clinical trial. To learn more, please read the related press release.

About EVX-01 Phase 2 Clinical Trial

EVX-01 is Evaxion’s lead clinical asset and constitutes a peptide-based personalized cancer vaccine. The Phase 2 clinical study is a self-sponsored open-label, single-arm, multi-center trial carried out in collaboration with Merck Sharp & Dohme LLC that, together with leading principal investigators and research centers from Italy and Australia, aims to evaluate the efficacy and safety of EVX-01 vaccination in combination with anti-PD1 therapy KEYTRUDA (pembrolizumab) in treatment-naive patients with metastatic or unresectable malignant stage III or IV melanoma. More information can be accessed under clinical trial ID NCT05309421.

On April 17, 2024 Enterome, a clinical-stage company developing first-in-class immunomodulatory drugs for solid and liquid malignancies and inflammatory diseases based on its unique Mimicry platform, reported database lock of its Phase 2 study of EO2401, in combination with an immune checkpoint inhibitor (nivolumab) +/- an anti-VEGF therapy (bevacizumab), for the treatment of patients with recurrent glioblastoma (EOGBM1-18/ROSALIE trial) (Press release, Enterome, APR 17, 2024, View Source [SID1234642137]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A total of 100 patients have been enrolled in ROSALIE, an international, open-label Phase 1/2 trial of EO2401, an innovative, off-the-shelf immunotherapy derived from Enterome’s oncomimicry platform. Interim results have been presented at multiple congresses, most recently at the Society for Neuro-Oncology (SNO) 2023 annual meeting demonstrating a 43.1% survival rate at 18 months in a 26-patient cohort dosed with EO2401 in combination with nivolumab+bevacizumab.

Jan Fagerberg, Chief Medical Officer of Enterome, said: "The achievement of the first trial evaluating EO2401 represents a major milestone for Enterome. With a total of 100 patients enrolled, of which 41 received the combination of EO2401 with nivolumab and bevacizumab, alongside extensive follow-up, we are confident that the ROSALIE study provides a compelling foundation for evaluating this novel immunotherapy as a potential treatment for patients with recurrent glioblastoma. With an encouraging clinical efficacy, we are now looking forward to sharing the final data with the scientific community in the coming months."

OncoMimics immunotherapies utilize bacteria-derived peptide antigens that closely mimic those expressed by tumor cells. In contrast to Tumor-Associated Antigens (TAAs), OncoMimics peptides are recognized as non-self by the immune system, inducing a strong and durable cytotoxic CD8+ immune response stemming from circulating effector memory T cells cross-reacting against the tumor, therefore offering enormous potential to create a new class of immunotherapies.

To date, Enterome has generated a repertoire of OncoMimics peptides targeting TAAs across a wide range of solid and liquid tumors. In addition to EO2401, the other candidates include EO2463, in Phase 2 clinical trial for indolent Non-Hodgkin Lymphomas, and EO4010, in Phase 2 clinical trial for metastatic colorectal cancer.

Pierre Bélichard, Chief Executive Officer of Enterome, said: "T cells are Nature’s most effective weapons against cancer cells, yet their potential is restrained by immunological self-tolerance. The ROSALIE study represents the first demonstration of OncoMimics immunotherapies’ ability to overcome immune tolerance, promising new avenues for targeting cancer cells. The trial provides a strong basis for pursuing a registrational path for EO2401 and expanding our pipeline to other indications. I am proud of the immense work accomplished by the Enterome team since the recruitment of the first patient in 2020. I also would like to thank patients, their families, and investigators whose dedication made this groundbreaking study possible."

About ROSALIE

ROSALIE (EOGBM1-18, ClinicalTrials.gov Identifier: NCT04116658) is a multicenter, open-label, first-in-human, Phase 1/2 study of EO2401 in combination with an immune checkpoint inhibitor (nivolumab, Opdivo) +/- bevacizumab for the treatment of patients with first progression/recurrence of glioblastoma. The study aims to assess the safety, tolerability, immunogenicity, and preliminary efficacy of the combination in 100 patients enrolled at 10 clinical sites in Europe and the US.

Clinical publications on ROSALIE to date include:

Reardon et al., EO2401 Peptide Immunotherapy + Nivolumab +/- Bevacizumad in First Recurrent Glioblastoma: The Phase 1/2 EOGBM1-18/ROSALIE Study, Neuro-Oncology, Volume 25, Issue Supplement_5, November 2023 DOI: 10.1093/neuonc/noad179.0265
Wick et al., EO2401 peptide immunotherapy + nivolumab +/- bevacizumab in recurrent glioblastoma: EOGBM1-18/ROSALIE. Journal of Clinical Oncology 41 2023 DOI:10.1200/JCO.2023.41.16_suppl.2020
Reardon et al., EO2401 Therapeutic Vaccine for Patients with Recurrent Glioblastoma: Phase 1/2 ROSALIE Study, Neuro-Oncology, Volume 24, Issue Supplement_7, November 2022 DOI:10.1093/neuonc/noac209.249
Maia et al., Strong immune response to therapeutic vaccination with EO2401 therapeutic vaccine + nivolumab: interim report of the EOGBM1–18/ROSALIE study, Journal for ImmunoTherapy of Cancer 2022 DOI:10.1136/jitc-2022-SITC2022.0641
Reardon et al., EO2401 therapeutic vaccine for patients with recurrent glioblastoma: Phase I/II ROSALIE study, Annals of Oncology, Volume 33, Supplement 7, September 2022 DOI:10.1016/j.annonc.2022.07.437
Reardon et al., EO2401 microbiome derived therapeutic vaccine + nivolumab, with/without standard continuous, or low-dose symptom directed, bevacizumab, in recurrent glioblastoma: phase 1–2 EOGBM1–18/ROSALIE study, Journal for ImmunoTherapy of Cancer 2022 DOI:10.1136/jitc-2022-SITC2022.0642
Wick et al., EO2401, a novel microbiome-derived therapeutic vaccine for patients with recurrent glioblastoma: ROSALIE study, Journal of Clinical Oncology Volume 40, Number 16_suppl DOI:10.1200/JCO.2022.40.16_suppl.2034
Idbaih et al., EO2401, a novel microbiome-derived therapeutic vaccine for patients with recurrent glioblastoma: ROSALIE study, Neuro-Oncology, Volume 24, Issue Supplement_2, September 2022 DOI:10.1093/neuonc/noac174.004

On April 17, 2024 Derm-Biome reported that the rates of precancerous skin conditions and skin cancers are soaring in many parts of the world. Actinic keratosis (AK) is the most common form of precancer, with over 40 million Americans a year developing this condition (Press release, Derm-Biome Pharmaceuticals, APR 17, 2024, View Source;utm_medium=rss&utm_campaign=derm-biome-pharmaceuticals-topical-therapy-shows-positive-results-in-preclinical-skin-cancer-trial-drug-prevents-the-development-of-precancerous-skin-conditions-and [SID1234642136]). Treating AKs before the cells become cancerous and spread to other parts of the body is crucial. For those patients with multiple AKs, common treatment options are chemotherapy creams and photodynamic therapy. Although effective, these treatments come with significant side effects, such as redness, blistering, and peeling, with recovery times that can be lengthy and uncomfortable. The global actinic keratosis treatment market size is projected to reach as high as $10 billion USD by 2031, fuelled by increasing cases of AK and rising healthcare spending.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Derm-Biome Pharmaceuticals is a Vancouver-based biopharmaceutical company that has developed a topical anticancer treatment for patients with or at high risk of developing multiple AKs. In a recent UV-induced skin cancer trial, mice were exposed to UVB radiation over a period of 25 weeks. The prolonged exposure mimics chronic sun exposure over time and replicates the gradual progression from precancerous skin conditions to cutaneous squamous cell carcinoma (cSCC). Topical application of Derm-Biome’s compound prior to UVB exposure significantly reduced the number and size of precancer lesions, while treatment blocked the progression of squamous cell carcinoma tumors.

Frédéric Couture, Researcher and Head of Pharmaceutical Sciences at TransBIOtech: "My research group conducted this study using Derm-Biome’s compound. The compound exhibited a significant protective effect without toxicity or side effects. Moreover, existing tumor progression was blocked with drug treatment. We actually noticed an improvement to the look of skin treated with the compound."

Dr. Poul Sorensen, University of British Columbia Professor and Distinguished Scientist at BC Cancer Research Centre, and Derm-Biome CSO: "The results of these studies are very promising. We tested our compound using a very aggressive UV-induced skin cancer model. We observed highly significant decreases in the number of tumors in treated mice and strong preventative effects when mice were pretreated with the compound. These findings suggest that our compound has great potential to be a highly effective and well-tolerated agent for both the treatment and prevention of squamous cell carcinoma."

Derm-Biome CEO Gordon Eberwein: "Currently available topical treatments cause debilitating side effects that make them unattractive to patients. There is a real need for safer and more targeted topical therapies."

Derm-Biome expects to start topical formulation development this summer, with IND-enabling studies slated to begin in Q4.

On April 17, 2024 Circio Holding ASA (OSE: CRNA), a biotechnology company developing next generation circular RNA vector technology for gene therapy, reported that it has established technical in vivo proof-of-concept for its proprietary circVec circular RNA platform by demonstrating statistically significant improvement in durability over mRNA-based expression (Press release, Circio, APR 17, 2024, View Source [SID1234642135]). The circVec technology has broad potential, particularly to enhance the potency and reduce cost of current gold-standard gene therapy, and the R&D strategy is centered on this rapidly expanding therapeutic area.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The circVec 2.1 design is performing very well in vitro, and this is now being translated in vivo with demonstration of enhanced expression level and durability for circVec 2.1 DNA vectors in mouse models. This provides an important technical proof-of-concept for Circio´s technology platform in a real biological system, which we expect will translate into improved gene therapies for patients in the future," said Dr. Thomas B Hansen, CTO at Circio. "This new data indicate that circVec has the potential to outperform current gold-standard gene therapy approaches, and we are rapidly advancing to design and test circVec in several AAV and DNA-based therapeutic vector formats."

In parallel to the in vivo characterization, Circio has tested and incorporated further features into the circVec platform. A dual-function ‘remove-&-replace’ concept has been designed and validated in vitro for Alpha-1-antitrypsin deficiency (AATD), with the ability to both replace functional AAT protein and remove the disease variant. This genetic disease causes severe symptoms in the lung and liver, and there are currently no satisfactory therapeutic options available. AATD represents a major unmet medical need and there are over 200,000 patients affected in the USA and EU.

"Establishing a robust technical in vivo proof-of-concept is a major milestone for the development of the circVec platform. Based on this validation, Circio will now explore which targets and diseases represent the best therapeutic and commercial opportunities," said Dr. Erik Digman Wiklund, CEO at Circio. "Initially, we have selected AATD as the lead program where our unique ‘remove-&-replace’ circVec design has an opportunity to solve two pathological issues in a single differentiated product. Our aim is to establish in vivo proof-of-concept in AATD within the next twelve months and select a lead therapeutic candidate by the middle of next year. We are confident that circVec can be highly effective in AATD and produce novel gene therapies that outperform current approaches."

To Circio´s knowledge, circVec 2.1 far exceeds other known intra-cellular circRNA-based expression systems, both in terms of circRNA biogenesis efficiency and protein yield. The platform still has further potential, and Circio is continuously improving the technology towards circVec 3.0 and beyond. The platform is protected by deep internal expertise and know-how, with three patents protecting the core technological features filed to date, and additional applications in progress.

"Although AATD is Circio´s lead internal focus, we are continuously exploring new applications and disease targets to build and broaden our technology platform and have several ongoing external dialogues to identify opportunities for future collaborations. Circio is currently working to address specific questions and requests from these prospective partners and aim to complete our first business development transaction within the next twelve months," said Dr. Lubor Gaal, CFO and Head of Business Development at Circio.

The recent circVec data, as well as a financial update and information about the intended fundraising during Q2 2024, are presented and discussed in a webcast available via Circio´s webpage and the Redeye platform link:

Link to webcast – access via Redeye

Circio company update 17 April PDF.pdf

On April 17, 2024 Circio Holding ASA (OSE: CRNA, "Circio"), a biotechnology company developing next generation circular RNA vector technology for gene therapy, reported that it is initiating a fundraising process with the intent to raise around NOK 50-60 million or more in gross proceeds from existing shareholders and new investors in a partially underwritten rights issue to be completed during Q2 2024 (Press release, Circio, APR 17, 2024, View Source [SID1234642134]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The board strongly believes in the potential of Circio´s unique circVec platform, and this intended fundraising will provide the company with the required capital to validate the significant advantage of the technology in gene therapy," said Damian Marron, Chairman of Circio. "With twelve months runway, Circio will have time to reach important R&D milestones and will aim to achieve its first business development deal, thereby delivering value to our shareholders."

Existing convertible bond investor Atlas Capital Markets is supportive of, and intends to participate in, the intended fundraising. Members of Circio´s board and management have pre-committed to participate with around NOK 2 million, including NOK 500.000 by CEO Dr. Erik D Wiklund.

"Circio has now demonstrated technical validation for its circVec circular RNA platform both in vitro and in vivo and is pushing ahead to establish in vivo proof-of-concept for its novel circVec gene therapy formats," said Dr. Erik Digman Wiklund, CEO at Circio. "This financing will enable Circio to develop the lead program in AATD towards selection of a lead therapeutic candidate by the middle of next year. In parallel, Circio will continue to improve and expand the circVec platform, which will open opportunities for multiple future revenue-generating partnerships both in the short- and long-term."

Circio will update the market in due time when the structure and timing of the intended transaction have been determined.

Redeye AB will be acting as financial advisor to Circio and sole bookrunner in the transaction and Advokatfirmaet Thommessen AS is acting as legal advisor.