On October 21, 2021 PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported plans to present new updates on its pipeline during the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, December 11-14, 2021 (Press release, PharmaEssentia, OCT 21, 2021, View Source [SID1234591710]).

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"Since we initiated the study in the U.S. earlier this year, we have heard from clinicians outside the U.S. who are eager to consider a new investigational option to support their ET patients. We are pleased to partner with these renowned MPN specialists to participate in this important study."

During the meeting, the company will provide an update on SURPASS ET, a Phase 3 pivotal clinical trial of its investigational ropeginterferon alfa-2b (P1101), a novel mono-pegylated proline interferon under evaluation for the treatment of essential thrombocythemia (ET). PharmaEssentia continues to enroll participants in the trial at sites throughout the United States and Asia, and has now expanded with new sites in Canada.

"People diagnosed with a myeloproliferative neoplasm (MPN), and particularly those with ET, have limited therapeutic options that can help them manage the progressive effects of the disease, so through this study, we will evaluate whether our mono-pegylated interferon could represent a useful alternative to currently available options," said Raymond Urbanski, M.D., Ph.D., U.S. Head of Clinical Development and Medical Affairs. "Since we initiated the study in the U.S. earlier this year, we have heard from clinicians outside the U.S. who are eager to consider a new investigational option to support their ET patients. We are pleased to partner with these renowned MPN specialists to participate in this important study."

The SURPASS ET open-label, randomized, active-controlled study is comparing the efficacy, safety, and tolerability and pharmacokinetics of P1101 as second line therapy for ET as compared with anagrelide, a commonly used oral platelet-reduction therapy. The study aims to enroll approximately 160 patients, who will be randomized to receive either P1101 subcutaneously every two weeks or anagrelide at a standard dose. Eligible patients include high-risk ET patients, those who are resistant to or intolerant of hydroxyurea and who have not received prior therapy with interferon.

The primary efficacy endpoint for the study employs the modified European Leukemia Net (ELN) criteria. Secondary efficacy endpoints include time to and durability of response, response rates based on hematologic parameters and symptomatic improvements. Changes in CALR, MPL, and JAK2 allelic burden over time is also being investigated. Topline data are expected by late 2022. More details on the study design and sites are available at www.clinicaltrials.gov (NCT04285086) or at www.SURPASSET.com.

PharmaEssentia continues to build momentum with its pipeline to deliver new solutions in MPNs. The company is seeking approval for its first indication in polycythemia vera (PV) in the U.S. and expects a decision by the U.S. Food and Drug Administration (FDA) in November 2021.

About Ropeginterferon alfa-2b

Ropeginterferon alfa-2b is a long-acting, mono-pegylated proline interferon aimed to be administered once every two weeks or longer. Ropeginterferon alfa-2b has Orphan Drug designation for treatment of polycythemia vera (PV) in the United States. Marketed as Besremi in Europe, the product was approved by the European Medicines Agency (EMA) in 2019. Ropeginterferon alfa-2b was discovered and is manufactured by PharmaEssentia in its Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018.

About Essential Thrombocythemia

Essential thrombocythemia (ET) is a myeloproliferative neoplasm (MPN) characterized by an overproduction of platelets in the blood that results from a genetic mutation; data indicates a JAK2 gene mutation is present in approximately half of diagnosed patients. ET is estimated to affect up to 57 per 100,000 people in the U.S. The disease is most commonly diagnosed through routine blood work, and is most common in people over the age of 50, with women 1.5 more times more likely to be diagnosed than men. As a chronic, progressive disease, ET requires regular monitoring and appropriate treatment. Over time, the disease may progress into more deadly conditions such as myelofibrosis or acute leukemia.1,2

On October 21, 2021 Cedilla Therapeutics, a biotechnology company bringing a new dimension to precision oncology, reported its two lead conditional inhibitor programs: an inhibitor of TEAD for the treatment of solid tumors, such as mesothelioma and certain squamous cell carcinomas; and a highly selective inhibitor of CDK2 for the treatment of multiple tumor types, including CDK4/6-resistant breast cancer, ovarian, uterine, stomach, and esophageal cancers (Press release, Cedilla Therapeutics, OCT 21, 2021, View Source [SID1234591709]). Both programs are wholly owned by Cedilla. In addition, Cedilla is pursuing discovery research efforts against a portfolio of high value cancer targets.

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"Over the past three years, we have built an integrated suite of capabilities that enable us to understand the relevant functional states of important yet elusive cancer targets, and identify previously unrecognized small molecule binding sites," said Alexandra Glucksmann, Ph.D., President and Chief Executive Officer of Cedilla Therapeutics. "Today, we are excited to announce our two lead programs from our internal efforts to discover conditional inhibitors: an inhibitor of TEAD and a highly selective inhibitor of CDK2. We look forward to advancing these programs closer to the clinic and creating novel medicines with the potential to deliver profound benefit to patients."

The discovery of both programs was enabled by Cedilla’s novel approach to developing small molecule conditional inhibitors. Cedilla recognizes proteins as dynamic entities, whose function is orchestrated by inter-molecular associations and post-translational tailoring. By accounting for the native full-length protein with relevant post-translational modifications, protein-protein interactions and sub-cellular localization, Cedilla is able to understand proteins in their functional state to discover new ways to access key cancer drivers that have been considered undruggable.

"Since our founding, we have worked to develop a deep understanding of high-value, historically inaccessible targets, and to identify new vulnerabilities that may enable us to drug them more effectively, delivering superior clinical benefit," said Brian Jones, Ph.D., Chief Scientific Officer. "Based on preclinical data, I believe our TEAD and CDK2 programs have clear advantages relative to historical approaches, offering the opportunity for preferential clinical utility, in terms of targeted efficacy or combinability with other therapeutic mechanisms. We look forward to advancing both programs into IND-enabling studies next year."

About Cedilla’s TEAD Program
TEAD (transcriptional enhanced associate domain) is a key component of the Hippo signaling pathway that is aberrantly regulated in solid tumors such as mesothelioma and certain squamous cell carcinomas. TEAD is also increasingly implicated in resistance to targeted therapies, including those for the treatment of EGFR-mutated and KRAS-mutated lung cancer.

Cedilla’s program is designed to inhibit the function of TEAD by preventing a post-translational modification required for full function. The company’s portfolio of TEAD inhibitors encompasses multiple chemotypes with different effects on TEAD isoforms and cofactors, providing Cedilla a range of starting points for selecting a candidate with an optimal profile for effective and combinable TEAD inhibition. Cedilla plans to be conducting investigational new drug (IND) application-enabling studies in the first half of 2022.

About Cedilla’s CDK2 Program
CDK2 (cyclin dependent kinase 2) has been a major target of interest for cancer indications driven by amplification or high levels of Cyclin E, including in roughly half of patients with CDK4/6-resistant breast cancer. In addition, Cyclin E amplification drives genetically defined subsets of ovarian, uterine, stomach and esophageal cancers. The CDK2-Cyclin E cancer node has remained inaccessible due to challenges achieving selectivity over other CDKs (cyclin dependent kinases), particularly CDK1, and Cyclin E isoforms.

Cedilla has developed a unique series of inhibitors that bind to a previously unreported site on the CDK2-Cyclin E complex with unprecedented selectivity, potentially offering a substantial advance over two decades of industry efforts. Preclinical characterization suggests that the exquisite selectivity of Cedilla’s inhibitor could result in a better safety profile compared to traditional kinase inhibitors, particularly with respect to dose-limiting hematological toxicities. Cedilla plans to be conducting IND application-enabling studies in the second half of 2022. In addition, based on its unique insights into Cyclin biology, Cedilla has the potential to pursue additional drug discovery programs against related targets.

On October 21, 2021 Imvax, Inc., a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies, reported a poster presentation at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in Washington, D.C., from November 10-14, 2021 (Press release, Imvax, OCT 21, 2021, View Source [SID1234591708]).

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The poster highlights recent preclinical data for the company’s most advanced product candidate, IGV-001. The data elucidate potential mechanisms for the observed immune-stimulating and tumor-suppressing activity of IGV-001 seen in the Phase 1 clinical study for glioblastoma.

"We’re pleased to share new insights into the mechanism of action of IGV-001, which are supportive of the clinical efficacy data we’ve previously reported," said John Furey, Chief Executive Officer. "These insights are valuable both as we prepare for a Phase 2b trial in glioblastoma and as we expand our platform to address other solid tumor types."

Details of the poster presentation are as follows:

Title: Autologous glioblastoma tumor cells and an antisense oligonucleotide against insulin-like growth factor type 1 receptor protect against tumor challenge and generate T cell anti-tumor responses
Number: P218
Timing: November 13, 2021, 7:00 a.m-8:30 p.m. EST
Presenter: Mark Exley, Ph.D., Chief Scientific Officer

On October 21, 2021 Oncodesign (ALONC – FR0011766229), a French biopharmaceutical company specialized in kinase inhibitor research and precision medicine, and TiumBio (KOSDAQ:321550), a company specializing in R&D for rare diseases, reported the signature of a research collaboration agreement on R&D of potential drug candidates for fibrosis (Press release, Oncodesign, OCT 21, 2021, View Source [SID1234591707]).

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Under this agreement, Oncodesign will be responsible for identification, chemical synthesis and optimization of Nanocyclix drug candidates and their early-stage analysis, while TiumBio will be responsible for the advanced evaluation of fibrotic efficacy of the drug candidates. This initial phase of the collaboration will be funded by TiumBio.

Also, TiumBio has secured an exclusive option to in-license global development and commercialization rights of the discovered drug candidates after their evaluation upon reaching predefined success criteria. Financial conditions for this option, which might be lifted over the next year, cannot be disclosed at this stage.

Hun-Taek Kim, CEO of TiumBio, stated: "We are delighted to work with Oncodesign to achieve our common goal of developing treatments for rare and incurable diseases. If we combine Oncodesign’s kinase inhibitor-based platform technology and our fibrosis-centered R&D experience and expertise in rare diseases, I am confident that we can develop new and innovative drugs in the fibrosis area."

Philippe Genne, CEO and founder of Oncodesign, said: "This new collaboration with TiumBio, an expert company in fibrosis in South-Korea, further expands the global reach of our unique technologies. Following earlier collaborations with international pharmaceutical companies including BMS (Bristol-Myers-Squibb), Ipsen, Sanofi, Servier and UCB, our platform technology Nanocyclix again is poised to bring solutions in a difficult area. I am thrilled to team up with TiumBio to find solutions for patients suffering from Fibrosis."

Jan Hoflack, CSO of Oncodesign, added: "This agreement is another example where an expert pharma company looking for compounds with unique and hard-to-meet criteria comes to benefit from our Nanocyclix technology platform, looking for high potency and exquisite selectivity. Upfront testing of the Nanocyclix diversity set has allowed to identify starting points satisfying TiumBio’s initial criteria and has led to this exciting collaboration."

About Fibrosis

Fibrosis occurs in most tissues of the human body, which is a phenomenon of fibrous connective tissue formation. As the disease progresses, the function of the corresponding organ is defected, which can lead to various chronic fatal disease conditions such as pulmonary and liver fibrosis. In addition, fibrosis accounts for about 45% of all cause disease mortality1, and since there are few effective treatments currently available, it is a disease area with a very high unmet medical demand from patients.

On October 21, 2021 Blueprint Medicines Corporation (NASDAQ: BPMC), reported that it will host a live conference call and webcast at 8:30 a.m. ET on Thursday, October 28, 2021 to report its third quarter 2021 financial results and provide a corporate update (Press release, Blueprint Medicines, OCT 21, 2021, View Source [SID1234591705]).

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To access the live conference call, please dial 844-200-6205 (domestic) or 929-526-1599 (international), and refer to conference ID 963004. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.