On Februray 1, 2023 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a synthetic lethality focused precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported its participation in the 2023 Guggenheim Oncology Conference (Press release, Ideaya Biosciences, FEB 1, 2023, View Source [SID1234626742]).

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2023 Guggenheim Oncology Conference
Thursday, February 9, 2023 at 7:45 AM PT (10:45 AM ET)

Fireside chat with Yujiro S. Hata, President and Chief Executive Officer and Michael White, Senior Vice President and Chief Scientific Officer, hosted by Charles Zhu, Ph.D. Vice President and Equity Research Analyst

A live audio webcast of the event will be available, as permitted by conference host, at the "Investors/ Events" section of the IDEAYA website at View Source A replay of the webcast will be accessible for 30 days following the live event.

On February 1, 2023 Beactica Therapeutics AB, the Swedish precision oncology company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to BEA-17 for the treatment of glioblastoma (GBM) (Press release, Beactica, FEB 1, 2023, View Source [SID1234626741]).

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BEA-17 is a first-in-class small molecule targeted degrader of the epigenetic enzyme LSD1 and its co-factor CoREST. Overexpression of LSD1 has been implicated in the pathogenesis of a variety of cancers, including GBM. There are currently no approved therapies for GBM that target this enzyme.

"The FDA’s decision to grant orphan drug status to BEA-17 marks an important milestone for the programme and highlights the significant need for novel therapies to treat these devastating brain tumours." said Dr Per Källblad, Co-Founder and CEO of Beactica Therapeutics. "The designation will facilitate our development of this agent which we believe has the potential to serve as a much-needed therapeutic option for patients affected by glioblastoma."

The FDA’s Orphan Drug Designation program supports the development of drugs that address rare diseases which affect fewer than 200,000 people in the United States. Incentives that come with the designation include eligibility for federal grants, tax credits for qualified clinical trials, prescription drug user fee exemptions, and a seven-year marketing exclusivity period upon FDA approval.

About BEA-17

BEA-17 is a first-in-class small molecule targeted degrader (non-PROTAC) of the epigenetic enzyme LSD1 and its co-factor CoREST. The compound has shown promising preclinical in vivo potentiation of immune-modulating treatments in several cancer forms, including standard of care (temozolomide and radiation) in state-of-the-art models of GBM. Pharmacokinetic studies of BEA-17 show good blood-brain-barrier penetration and oral availability. BEA-17 is investigational and not approved anywhere globally. Its efficacy and safety in humans have not been established.

About Glioblastoma (GBM)

GBM is the most common and most aggressive brain tumour. Approximately 35,000 people in the U.S. and Europe are diagnosed with GBM each year. The median overall survival is 15 months, and the five-year overall survival is only 5%.

On February 1, 2023 Sirnaomics Ltd. (the "Company" or "Sirnaomics", stock code: 2257.HK), a leading biopharmaceutical company in discovery and development of RNAi therapeutics, reported that it is advancing the clinical development of STP705 for the treatment of two non-melanoma skin cancers: Squamous Cell Carcinoma in situ (isSCC) and Basal Cell Carcinoma (BCC) (Press release, Sirnaomics, FEB 1, 2023, View Source [SID1234626740]). Based on positive clinical readouts from a Phase IIb study for isSCC and a Phase II study for BCC, Sirnaomics is formulating a communication package for the U.S. Food and Drug Administration (FDA) to seek guidance for conducting a late-stage clinical development for the treatment of isSCC and BCC. Additionally, the Company has developed a commercialization plan to position STP705 when upcoming clinical studies reach primary endpoints.

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An interim report in December 2022 in respect of the Phase IIb study of STP705 for the treatment of isSCC showed that the majority (78%) of the 32 patients treated with STP705 achieved histological clearance, the primary endpoint of the study. One out of the three treatment cohorts achieved 89% histological clearance. The positive results further validated STP705 as a novel siRNA (small interfering RNA) therapeutic, taking advantage of a dual-targeted inhibitory property and a polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expressions.

In August 2022, the Company reported that the Phase II clinical study of STP705 in five patients with BCC showed an optimal clinical dosage of 180µg and achieved a 100% histological clearance and completed response (CR), indicating the potential viability of the treatment. Apart from achieving a 100% CR, the data in this cohort showed improved or stable cosmetic results with an excellent safety profile and no significant cutaneous skin reactions. The success of BCC treatment with STP705 further validated the potential for the treatment of non-melanoma conditions.

"In our clinical studies for STP705 to date, we have achieved significant efficacy and a strong safety profile," said Dr. Michael Molyneaux, M.D., Executive Director and Chief Medical Officer of Sirnaomics. "As we continue to study this promising therapeutic candidate, we will further confirm the most effective dose and the therapeutic benefits for the treatment of isSCC, SCC and BCC. Those non-melanoma skin cancers are affecting millions of people in the United States and globally. Currently, there are only a small number of approved non-surgical treatments. We have formulated a marketing and launch strategy for using our injectable treatment approach to compensate the shortcoming of the surgical treatment."

"The latest results from the Phase IIb study of isSCC and Phase II study of BCC has shown efficacy of STP705 without any drug related Adverse Events (AEs) and Serious Adverse Events (SAEs), validating the broad potential of STP705 for treatment of non-melanoma skin cancers," said, Dr. Patrick Lu, Founder, Chairman of the Board, Executive Director, President and CEO of Sirnaomics. "With guidance from the FDA, Sirnaomics is confident to move our lead drug candidate into later-stage clinical development to further explore and evaluate the great potential of STP705 for the treatment of non-melanoma skin cancers."

About Non-Melanoma Skin Cancers

Non-melanoma skin cancers (NMSC) are the most common forms of human neoplasia. NMSC constitute a large group of skin cancers that are not melanoma, including squamous cell carcinoma (SCC), basal cell carcinoma (BCC), Extramammary Paget’s Disease (EMPD), Merkel cell carcinoma (MCC), and skin adnexal carcinomas. Among these, BCC and SCC account for the majority of NMSC with an estimate of more than 5 million newly diagnosed cases in the United States every year. Along with BCC, SCC is one of two major subtypes of NMSC. The market size of the NMSC treatment market in the United States is expected to increase from US$6.5 billion in 2020 to US$22 billion in 2030. In China, the market size of NMSC treatment was US$38 million in 2020 and is also expected to grow rapidly in the coming years, reaching US$149 million in 2030.

A World Health Organization authorized report from the "International Agency for Research on Cancer" (2019) revealed that the number of deaths in 2018 globally for both men and women from NMSC is 65,155, of which 41.9% were within the populations of Asians, which is significantly more than other populations.

Surgery is currently the most common treatment option for the treatment of NMSC. The various forms of surgical modalities carry significant cutaneous adverse events, risk of scar, infection and bleeding. As a result, there is a high unmet need for an FDA-approved local injection therapy that is safe and effective.

On February 1, 2023 OncoHost, a precision diagnostics company centered on predictive biomarker development for improved patient care, reported the official launch of its PROphet non-small lung cancer (NSCLC) Test in the United States (Press release, OncoHost, FEB 1, 2023, View Source [SID1234626739]).

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OncoHost’s first test, PROphet NSCLC, guides first line treatment decisions for advanced unresectable non-small lung cancer patients.

The PROphet NSCLC Test provides clinicians with actionable clinical insights into optimal first line therapeutic choices, and a better understanding of their patients’ personalized cancer dynamics. Requiring just one pre-treatment blood test, PROphet scans approximately 7,000 proteins in a patient’s blood plasma and delivers a report that predicts their clinical benefit from anti-PD-1/PD-L1 immunotherapy-based treatment plans.

"After years of research, development and determination, we are proud to be launching our PROphet NSCLC test in the United States," said Dr. Ofer Sharon, CEO of OncoHost. "While driver mutation detection is an essential factor in determining effective treatment, approximately 85% of all patients diagnosed with NSCLC do not have any detectable driver mutations. In these cases, the PROphet NSCLC Test, when combined with PD-L1 results, will serve an invaluable purpose in predicting the best treatment plan for each individual patient. We hope to create a shift in the industry and improve the lives of those fighting this disease."

The PROphet algorithm is trained on OncoHost’s large-scale clinical trial, PROPHETIC. To date, the trial has over 1,500 patients recruited across 40 sites worldwide, making it one of the largest prospective cohorts in the precision oncology field. The PROphet NSCLC test is supported by a blinded validation demonstrating that it accurately predicts a patient’s clinical benefit and associated overall survival differences with single agent versus combination treatment plans.

"We are thrilled to be launching the PROphet NSCLC test, offering a predictive biomarker to guide treatment planning for this large subset of patients," said Chris Dingman, OncoHost CCO. "Access to our test will have a significant impact on patient care and treatment protocols. The PROphet Report will guide physicians’ treatment decisions by offering personalized insights on the predicted clinical benefit of immunotherapy. Combining these findings with the patients PD-L1 level will allow them to offer the most effective plan and avoid unnecessary treatments."

To order the PROphet NSCLC test, any physician can visit the OncoHost website at www.oncohost.com and complete a Test Requisition Form.

On February 1, 2023 Ratio Therapeutics Inc., a pharmaceutical company specializing in the development of targeted radiotherapeutics for the treatment of cancer, reported the close of its Series A financing extension today, bringing the total raised to date to over $40 million (Press release, Ratio Therapeutics, FEB 1, 2023, View Source [SID1234626738]). The latest round was led by Duquesne Capital and Schusterman Family Investments, with Kenan Turnacioglu, Ph.D., General Partner at Catalio Capital Management representing them and joining as an executive advisor, along with continued significant participation from existing investor base.

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The proceeds will be used to optimize and expand the breadth of the company’s proprietary Trillium and Macropa technology platforms, accelerate the discovery and development of targeted radiotherapies and diagnostics leveraging the platforms’ tunable nature to advance programs through clinical-stage development as well as expand the team through hiring and forming new strategic partnerships.

Founded by entrepreneurial scientists Jack Hoppin, Ph.D., and John Babich, Ph.D., Ratio emerged in June of 2022 with over $20 million in seed financing while engaged in fully funded development alliances with Bayer and Lantheus Holdings Inc. Since then, the company has progressed its collaboration programs, established additional internal discovery and preclinical programs, implemented a comprehensive intellectual property strategy in targeted radiotherapies and diagnostics and filed numerous patent applications.

"We are grateful for the support and honored by the confidence of our investors, who have witnessed our tremendous progress in the past year and recognize the potential of our technology and the strength of our team," commented Dr. Hoppin, Ratio’s Chairman and Chief Executive Officer. "This financing extends our cash runway into 2024 and will allow us to continue making progress toward our milestones. We look forward to working closely with our shareholders as we continue to advance our R&D platforms and bring innovative treatments to address the unmet needs of patients as quickly as possible."

Ratio also welcomes Susan K. Whoriskey, Ph.D., to its board of directors as a strategic advisor who will work closely with the executive team at Ratio to implement strategies for translating scientific advancements into approved therapies. Dr. Whoriskey is a seasoned executive with 30 years of experience in the biotechnology and pharmaceuticals sectors and has sequentially been part of the Founding Executive Team for four successful biotechnology companies: Vera Therapeutics, Moderna, Momenta Pharmaceuticals and Cubist Pharmaceuticals.

Dr. Whoriskey’s expertise lies at the interface of integrating research, intellectual property and corporate development. Throughout her career, she has focused on pioneering new technologies and clinical candidates to address unmet needs in the healthcare industry. She has been involved in 5 FDA drug approvals.

"Both the financing round and Susan’s addition to our board of directors are a testament to the value of Ratio’s pipeline and the science behind our technology. We warmly welcome her!" said Dr. Babich, Ratio’s President and Chief Scientific Officer. "Susan’s expertise in working with teams to advance scientific discoveries from the lab bench to the market with several top biotech companies has already been invaluable and will continue to be as we advance our R&D platforms, pipeline and grow as a company in the emerging field of radiopharmaceuticals to address the unmet needs in solid tumors."

About Trillium and Macropa

Ratio Therapeutics’ fully integrated proprietary R&D platforms, Trillium and Macropa, harness the tumor-killing power of alpha particles. The tunable nature of the platforms enables the efficient and timely development of numerous novel radiopharmaceuticals for a broad range of high unmet need in solid tumors, while addressing the trifecta of typical challenges seen with most radiopharmaceuticals: delivery, safety and efficacy. Trillium is a pharmacokinetic modulation platform that can be altered to bind to any antigen-specific target, while Macropa is a best-in-class Actinium-225 chelator. The combination of these platforms enables the tumor-killing power of alpha particles with potential for first- and best-in-class radiopharmaceuticals.