On january 8, 2023 Ascendis Pharma A/S (Nasdaq: ASND) reported an update on its Vision 3×3 strategic roadmap and planned 2023 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 9, at the 41st Annual J.P. Morgan Healthcare Conference (Press release, Ascendis Pharma, JAN 8, 2023, View Source;3-strategic-roadmap-0 [SID1234626004]).

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!

"As we approach anticipated launch of our second endocrinology rare disease product, TransCon PTH for hypoparathyroidism, we remain on track to achieve Vision 3×3 to become a sustainable, profitable leading biopharma company," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "We are deeply committed to advancing our pipeline of endocrinology rare disease, oncology, and now ophthalmology product candidates designed to make a meaningful difference in the lives of patients."

Key Updates

TransCon hGH:

During the fourth quarter, 2022, we completed recruitment into the Phase 3 foresiGHt Trial in adult growth hormone deficiency (GHD). Topline results from foresiGHt are expected in the fourth quarter of 2023.
During the third quarter of 2023, we anticipate completing enrollment in New InsiGHTS, a Phase 2 trial evaluating the safety and efficacy of TransCon hGH in patients with Turner Syndrome.
First European SKYTROFA (lonapegsomatropin) commercial launch planned in Germany for the third quarter of 2023.
SKYTROFA (lonapegsomatropin-tcgd) U.S. preliminary, unaudited fourth quarter 2022 revenue is expected to be approximately €17.1 million. This includes an estimated negative foreign currency translation impact of €0.4 million, compared to a benefit of €0.5 million in the third quarter of 2022.
TransCon PTH:

Phase 3 PaTHway Japan trial achieved its primary objectives; topline results consistent with North American and EU trials.
FDA Priority Review continues for use in adult patients with hypoparathyroidism, with an April 30, 2023 PDUFA date; if approved, U.S. commercial launch planned in the second quarter of 2023.
Enrollment opened in January 2023 for U.S. Expanded Access Program (EAP).
European Commission decision anticipated during the fourth quarter of 2023; if approved, EU commercial launch planned shortly thereafter.
Once-weekly TransCon PTH in preclinical development for patients on stable daily TransCon PTH dose.

TransCon CNP:

First-ever randomized, double-blind, placebo-controlled Phase 2 trial (ACcomplisH) suggests a potential for safety, efficacy, tolerability, and convenience in children with achondroplasia as young as two years of age; all 57 patients currently remain in the trial with treatment duration up to 3 years.
During the second quarter of 2023, we expect to complete enrollment in ApproaCH, a global randomized, double-blind, placebo-controlled Phase 2b trial in children ages 2–11 years with achondroplasia. The trial targets enrollment of ~80 patients.
During the third quarter of 2023, we expect to submit an IND or similar in children under the age of two years with achondroplasia.

TransCon TLR7/8 Agonist:

Reported topline data from the dose escalation portion of the Phase 1/2 transcendIT-101 Trial at SITC (Free SITC Whitepaper) 2022. Early signs of clinical activity were observed in patients receiving TransCon TLR7/8 Agonist as monotherapy or in combination with pembrolizumab.
Enrollment in transcendIT-101 continues with dose expansion focused on investigating TransCon TLR7/8 Agonist in combination with pembrolizumab in four cancer types.

TransCon IL-2 β/γ

The Phase 1/2 IL-βelieγe Trial evaluating TransCon IL-2 β/γ monotherapy in patients with locally advanced or metastatic solid tumors continues to enroll patients. Results from monotherapy dose escalation are expected during the first quarter of 2023.

Ophthalmology selected as the third therapeutic area:

In vivo data demonstrates, TransCon Hydrogel Platform supports continuous local drug release over at least 6 months supporting twice yearly administration.
TransCon RBZ (ranibizumab) selected as the first product candidate.

Presentation at J.P. Morgan Healthcare Conference on Monday, January 9th

A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at View Source The presentation will begin at 12:00 p.m. Eastern Time / 9:00 am Pacific Time. A webcast replay will be available for 30 days.

The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at View Source

On January 8, 2023 Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, reported that the company expects to report revenue between $550.7 million and $552.7 million for the fourth quarter ended Dec. 31, 2022 (Press release, Exact Sciences, JAN 8, 2023, View Source [SID1234625998]).

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!

"Exact Sciences’ fourth quarter results show the strength of our business and the momentum building behind the best brands in cancer diagnostics, Cologuard and Oncotype DX," said Kevin Conroy, chairman and CEO. "The foundation supporting Cologuard and Oncotype DX will fuel consistent revenue growth and profitability, putting Exact Sciences in a leading position to deliver the next wave of innovation in cancer diagnostics to patients, health systems, and health care providers globally."

Preliminary, Unaudited Fourth Quarter 2022 Financial Results

For the three-month period ended Dec. 31, 2022, as compared to the same period of 2021:

Total revenue between $550.7 million and $552.7 million, an increase of 16 percent, or 28 percent excluding COVID-19 testing
Screening revenue between $401.8 million and $402.8 million, an increase of 45 percent, or 41 percent excluding the PreventionGenetics acquisition
Precision Oncology revenue between $142.9 million and $143.9 million, a decrease of 4 percent, or an increase of 1 percent excluding the divested Oncotype DX Genomic Prostate Score test and the impact of foreign currency exchange rates
COVID-19 testing revenue of approximately $5.9 million, a decrease of 87 percent
Preliminary, Unaudited 2022 Financial Results

For the twelve-month period ended Dec. 31, 2022, as compared to the same period of 2021:

Total revenue between $2,082.0 million and $2,084.0 million, an increase of 18 percent, or 25 percent excluding COVID-19 testing
Screening revenue between $1,423.0 million and $1,424.0 million, an increase of 34 percent, or 30 percent excluding the PreventionGenetics acquisition
Precision Oncology revenue between $601.0 million and $602.0 million, an increase of 7 percent, or 11 percent excluding the divested Oncotype DX Genomic Prostate Score test and the impact of foreign currency exchange rates
COVID-19 testing revenue of approximately $58.0 million, a decrease of 59 percent
Screening includes laboratory service revenue from Cologuard tests, PreventionGenetics, and immaterial revenue from Biomatrica and Oncoguard Liver products. Precision Oncology includes laboratory service revenue from global Oncotype DX products and therapy selection products.

Exact Sciences has not completed preparation of its financial statements for the fourth quarter or full year of 2022. The revenue ranges presented in this news release for the fourth quarter of 2022 and for the year ended Dec. 31, 2022 are preliminary and unaudited and are thus inherently uncertain and subject to change as we complete our financial results for the fourth quarter of 2022. Exact Sciences is in the process of completing its customary year-end close and review procedures as of and for the year ended Dec. 31, 2022, and there can be no assurance that final results for this period will not differ from these estimates. During the course of the preparation of Exact Sciences’ consolidated financial statements and related notes as of and for the year ended Dec. 31, 2022, the company’s independent registered public accountants may identify items that could cause final reported results to be materially different from the preliminary financial estimates presented herein.

Exact Sciences plans to report 2022 financial results during its February 2023 earnings call.

About Cologuard

The Cologuard test was approved by the FDA in August 2014, and results from Exact Sciences’ prospective 90-site, point-in-time, 10,000-patient pivotal trial were published in the New England Journal of Medicine in March 2014. The Cologuard test is included in the American Cancer Society’s (2018) colorectal cancer screening guidelines and the recommendations of the U.S. Preventive Services Task Force (2021) and National Comprehensive Cancer Network (2016). The Cologuard test is indicated to screen adults 45 years of age and older who are at average risk for colorectal cancer by detecting certain DNA markers and blood in the stool. Do not use the Cologuard test if you have had precancer, have inflammatory bowel disease and certain hereditary syndromes, or have a personal or family history of colorectal cancer. The Cologuard test is not a replacement for colonoscopy in high risk patients. The Cologuard test performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older. The Cologuard test performance in repeat testing has not been evaluated.

The Cologuard test result should be interpreted with caution. A positive test result does not confirm the presence of cancer. Patients with a positive test result should be referred for diagnostic colonoscopy. A negative test result does not confirm the absence of cancer. Patients with a negative test result should discuss with their doctor when they need to be tested again. Medicare and most major insurers cover the Cologuard test. For more information about the Cologuard test, visit cologuard.com. Rx only.

On January 8, 2023 CymaBay Therapeutics, Inc. (NASDAQ: CBAY) ("CymaBay" or the "Company") reported that it has entered into a collaboration and license agreement with Kaken Pharmaceutical Co., Ltd. ("Kaken") for the development and commercialization in Japan of CymaBay’s investigational drug seladelpar for the treatment of primary biliary cholangitis ("PBC") (Press release, CymaBay Therapeutics, JAN 8, 2023, View Source [SID1234625997]).

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!

Under the terms of the agreement, Kaken receives an exclusive license to develop, commercialize and market seladelpar in Japan for PBC. Kaken will make an upfront payment to CymaBay of ¥4.5 billion (approximately $34.0 million at current exchange rates) with additional potential milestone payments to CymaBay totaling up to ¥17.0 billion (approximately $128.4 million at current exchange rates) for the achievement of certain regulatory and sales milestones in addition to 20+% royalties. Kaken will be responsible for development, regulatory approval and commercialization of seladelpar in Japan.

There are currently no approved second line treatments for PBC in Japan, representing a significant unmet medical need for PBC patients in Japan.

"We are honored to collaborate with Kaken, a company that shares our passion and dedication to working together to bring to market novel therapies targeting serious rare diseases," said Sujal Shah, Chief Executive Officer of CymaBay. "Kaken is a well-regarded leader in development and commercialization in Japan with a proud history of scientific and medical innovation. By working together, we believe that if approved, we can potentially make seladelpar broadly available to Japanese patients to complement CymaBay’s efforts to provide access in the U.S. and other parts of the world. This collaboration is an important next step in achieving our vision of becoming the global leader in treating cholestatic liver diseases."

"We are pleased to begin a collaboration with CymaBay, a company with a strong passion toward developing novel therapeutics for cholestatic liver diseases," said Hiroyuki Horiuchi, President and Representative Director of Kaken. "With this collaboration, we hope we can help improve the lives of patients with cholestatic liver disease in Japan."

On January 7, 2023 Fosun Pharma (600196.SH, 02196.HK) reported that it has recently entered into an exclusive license agreement with Shanghai Henlius Biotech, Inc. (2696.HK) for the commercialisation of Henlius independently developed anti-PD-1 monoclonal antibody (mAb) serplulimab in the United States (US) (Press release, Fosun Pharma, JAN 7, 2023, View Source [SID1234626007]. This marks an important milestone for Fosun Pharma’s development in the US.

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!

Fosun Pharma will have the right to commercialise serplulimab upon approval in the US, and Henlius will retain responsibility for development, manufacturing, and supply. For many years, the two companies have worked closely on product commercialisation and other areas, developing consensus in terms of corporate vision and values, global development strategy, product operations, and management communication while focusing on patients’ and clinical needs. Through this collaboration, the two parties aim to intensively develop serplulimab, integrate superior resources, cooperate in securing approval, and promote its rapid launch in the United States to benefit more patients.

Mr. Wu Yifang, Chairman of Fosun Pharma, said, "We are very happy to make full use of Fosun Pharma’s accumulated innovation capabilities, internationalization advantages and commercialization capabilities over the years to promote the commercialization of an approved anti-PD-1 mAb serplulimab in the US market and help benefit more patients around the world. Fosun Pharma has always been patient-centered and clinical needs-oriented. We have been practicing internationalization strategies in the dimensions of innovative R&D, license introduction, production operation and commercialization, and continuously strengthening the construction of full capacity in global operations."

Mr. Wenjie Zhang, Chairman and CEO of Henlius, said, "We are excited to partner with Fosun Pharma in the United States on serplulimab. The impressive clinical data of serplulimab was published in the top international medical journal and has gained international attention. As we move forward, we’ll seek approvals for additional indications in China, the US, and the European Union. In the US, Fosun Pharma has a fast-growing commercialisation team and a dedicated sales network. Together, we will, upon approval, accelerate the launch of this differentiated anti-PD-1 mAb in the US market and benefit more overseas patients with high-quality biological drugs."

Joining Forces for a Global Blueprint

Founded in 1994, Fosun Pharma is a global innovation-driven pharmaceutical and healthcare industry group. Fosun Pharma strengthens its independent R&D and external cooperation and enriches its product pipelines, as well as promotes global networks. Thanks to its industry-leading two-way licensing capabilities and international advantages, Fosun Pharma helps maximize the value of innovative products of partners. In the international layout, Fosun Pharma focuses on building the U.S. operation center and conducts all-around capacity building in R&D, production, and sales in the local market. On the important partnership in the U.S. for serplulimab, and the potential impact on patients with lung cancer, Mr. Rong Yang, Senior Vice President of Fosun Pharma and CEO of Fosun Pharma USA Inc. said, "It is a very important step for us, an opportunity to be a part of the community, and serving patients with critical unmet needs, and making a difference every day."

Fully leveraging the experience of biologics development and harnessing the power of innovation, Henlius has built a vertically integrated biopharmaceutical platform with core capabilities of high-efficiency and innovation embedded throughout the whole product life cycle, including R&D, manufacturing and commercialisation, laying the solid foundation for a diversified and robust innovation pipeline. To date, 5 products have been successfully marketed in China, and 1 approved for marketing in overseas markets. The pipeline of products under development covers targets with great market potential, such as PD-1, HER2, EGFR, BRAF, etc. Synergising the innovation centres in China and the US and global product development teams, Henlius continues the momentum for a diversified innovation product pipeline and has conducted more than 20 clinical trials in China, the EU, the US, Australia, etc. with more than 70 clinical trial approvals worldwide. In terms of manufacturing capabilities, Henlius strictly follows the International Good Manufacturing Practice (GMP) standards for production and quality control. The products in the pipeline are produced by Henlius’s self-built manufacturing facilities, with rich production experience from clinical stages to commercialised products. Also, the manufacturing facilities have successfully passed the on-site inspection conducted by NMPA, the European Medicines Agency, the EU qualified person, and multiple international business partners, and are certified by China and EU GMP. Henlius’ strong R&D capabilities, international-standard quality management system, and production capacity enable Henlius to boost the global commercialisation of its products.

About HANSIZHUANG

HANSIZHUANG, as the product is known in China, (recombinant humanised anti-PD-1 monoclonal antibody injection, generic name: serplulimab injection) is the first innovative monoclonal antibody developed by Henlius. Up to date, 2 indications are approved for marketing in China, 2 NDAs have been accepted by the NMPA, and more than 10 clinical trials are ongoing across the world.

HANSIZHUANG was launched in China in March 2022 and has been approved by the NMPA for the treatment of MSI-H solid tumours and squamous non-small cell lung cancer (sqNSCLC). Its synergy with in-house products of the company and innovative therapies are being actively promoted. It has successively obtained clinical trial licenses in China, the United States, the European Union and other countries and regions to initiate 12 clinical trials on immuno-oncology combination therapies in a wide variety of indications, such as lung cancer, esophageal carcinoma, head and neck squamous cell carcinoma and gastric cancer, etc., and covering the full range of first-line treatments of lung cancers. As of now, the company has enrolled more than 3,100 subjects in China, Turkey, Poland, Georgia and other countries and regions, and the proportion of White is over 30% in two MRCTs, making HANSIZHUANG an anti-PD-1 mAb with one of the largest global clinical data pools. The NDAs of the first-line treatment for squamous non-small cell lung cancer (sqNSCLC), extensive-stage small cell lung cancer (ES-SCLC), and esophageal squamous cell carcinoma (ESCC) have been accepted by the NMPA, which makes HANSIZHUANG potentially the world’s first anti-PD-1 mAb for the first-line treatment of SCLC. Furthermore, HANSIZHUANG was recommended for the treatment of ES-SCLC in the 2022 CSCO Guidelines for Diagnosis and Treatment of Small Cell Lung Cancer (SCLC), and the associated clinical trial became the first study published in JAMA on SCLC immunotherapy. Serplulimab was also granted orphan drug designations by the FDA and EC for the treatment of SCLC, and the first patient has been dosed in a bridging head-to-head trial in the United States to comparing HANSIZHUANG to standard of care Atezolizumab (anti-PD-L1 mAb) for the first-line treatment of ES-SCLC.

On January 6, 2023 Biotechnology company Fraser Therapeutics has completed attracting Series A investment from domestic venture capital (Press release, Prazer Therapeutics, JAN 6, 2023, View Source;idx=226&page=1&code=news [SID1234635351]). A total of KRW 17 billion was invested, and the corporate value after investment was recognized as approximately KRW 67 billion.

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!

Founded in October 2019, Fraser Therapeutics is challenging new drug development based on ‘SPiDEMTM’, a new platform technology that degrades target proteins. This is not only a new platform that overcomes the shortcomings of bio-based technology, but also an innovative method of targeting untargetable proteins (undruggable targets) in cells that cannot be accessed by any existing method.

Fraser Therapeutics, the first domestic company to build its own TPD Platform (Target Protein Degradation Platform), is pursuing the commercialization of Speedem.

Fraser Therapeutics said, "Based on our research and development know-how and expertise in Cell Signaling Pathway and Epigenetics, we will continue to develop treatments step by step, focusing on the field of immuno-anticancer drugs.