On April 20, 2021 Transcenta Holding Limited (Transcenta), a clinical stage global biotherapeutics company with fully-integrated capabilities in discovery, development and manufacturing of antibody-based therapeutics, reported that it has received clearance of its IND for TST005 from US FDA for initiating Phase I clinical trial of its bi-functional anti-PD-L1/TGF-β antibody (Press release, Transcenta, APR 20, 2021, View Source;antibody-tst005-301272664.html [SID1234578271]).

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TST005, is a bi-functional anti-PD-L1 and TGF-β trap fusion protein designed to simultaneously target two immuno-suppressive pathways, transforming growth factor -β (TGF-β) and programmed cell death ligand-1 (PD-L1), that are commonly used by cancer cells to evade the immune system. TST005 consists of a high affinity PD-L1 antibody fused with an engineered TGF-β Receptor Type II protein in its C-terminal. TST005 lacks FcR binding activity and thus has reduced FcR mediated killing of PD-L1 expressing effector T cells. TST005’s high PD-L1 binding activity and enhanced TGF-β trap stability enables the targeted delivery of TGF-β trap into PD-L1 expressing tumors, thereby minimizing off-target toxicities of systemic inhibition of TGF-β signaling. TST005 displayed potent activity in vitro in reversing TGF-β induced T-cell suppression. In multiple syngeneic tumor models, TST005 induced significant increase of CD8 T-cell infiltration into PD-L1 expressing tumors and displayed dose-dependent tumor growth inhibition in tumor model with high level TGF-β. TST005 is well tolerated in non-human primates and displayed a linear PK profile. TST005 is a potential novel bi-functional immunotherapy candidate with improved therapeutic window.

"TST005 is one of the few leading PD-L1/TGF-β bi-functional antibody drug candidates currently in clinical development globally," said Dr. Michael Shi, Transcenta’s EVP, Head of Global R&D and CMO, "We plan to simultaneously develop TST005 both in China and the United States under the same Phase I protocol with an innovative basket trial design. With the IND Clearance for TST005 in the US, we will accelerate the clinical development globally by allowing Chinese patients dosed at the current dose level when joining the study upon Chinese IND clearance. Once safety and tolerability are established, we plan to further evaluate TST005 in multiple other pretreated tumor types globally, benefiting patients worldwide at an early date."

On April 20, 2021 A trans-Atlantic collaborative group of researchers led by Children’s Hospital of Philadelphia (CHOP) reported that it has received approximately $680,000 from a group of research charities led by Solving Kids’ Cancer UK to study slowly progressive, or "indolent," neuroblastoma, which does not respond to chemotherapy and lacks other treatment options (Press release, CHOP, APR 20, 2021, View Source [SID1234578270]). The grant will fund research that could ultimately lead to better diagnosis of this form of the disease, as well as targeted treatment options.

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The interdisciplinary group involves researchers from CHOP; the Icahn School of Medicine at Mount Sinai; The Institute of Cancer Research, London; Texas Tech University Health Sciences Center; and Seattle Children’s.

"This grant will allow us to make progress in diagnosing and treating so-called indolent neuroblastoma, which is currently fatal in most patients," said principal investigator John M. Maris, MD, pediatric oncologist at the Cancer Center at CHOP and Giulio D’Angio Chair in Neuroblastoma Research at CHOP. "If we can identify signatures that allow us to diagnose this form of neuroblastoma in patients early, we can avoid unnecessary chemotherapy, which is not effective in these patients, and instead work toward targeted treatments that could ultimately improve patient outcomes."

Neuroblastoma is a type of cancer that forms in developing nerve cells and can present differently depending on the form of the disease. Some types can spontaneously regress and become benign, whereas others progress steadily and are fatal. The rate of progression varies. While certain forms of the disease progress rapidly but can be treated effectively with chemotherapy, other forms, like indolent neuroblastoma, progress slowly and are resistant to chemotherapy. The latter form predominately affects older children, teenagers and adults, who have very little chance of surviving their disease.

Currently, researchers lack tools to identify patients with indolent neuroblastoma and therapies with which to cure them. The joint award from Zoé4Life, Joining Against Cancer in Kids (JACK), Merryn Lacy Trust, Oscar Knox Fund, Solving Kids’ Cancer (US) and Solving Kids’ Cancer UK will allow the trans-Atlantic group of researchers to validate a specific and sensitive molecular test in tumors, which will be further developed for use with patient blood, in order to reliably identify patients with slowly progressing disease, rather than waiting for chemotherapy to fail them. The research team will also create and validate robust laboratory models of indolent neuroblastoma, which they will use to test combinations of immunotherapy with targeted small molecular therapies. Ultimately, the researchers hope to rapidly move a completely new therapy to an international clinical trial.

"Putting your child through multiple rounds of chemotherapy and watching them suffer from horrible side-effects only to find it’s made no difference to their disease is a truly devastating blow for parents," said Nick Bird, Research Trustee at Solving Kids’ Cancer UK. "We have to identify these children at diagnosis so we can spare them from chemotherapy that doesn’t work, and then we have to find better ways to treat their disease instead."

On April 20, 2021 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported it will release results for the first quarter 2021 on Wednesday, May 5, 2021, after the market closes (Press release, Vanda Pharmaceuticals, APR 20, 2021, View Source [SID1234578269]).

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Vanda will host a conference call at 4:30 PM ET on Wednesday, May 5, 2021, during which management will discuss the first quarter 2021 financial results and other corporate activities. To participate in the conference call, please dial 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode 5709209.

The conference call will be broadcast simultaneously and archived on Vanda’s website, www.vandapharma.com. Investors should go to the website at least 15 minutes early to register, download, and install any necessary audio software.

A replay of the call will be available on Wednesday, May 5, 2021, beginning at 7:30 PM ET and will be accessible until Wednesday, May 12, 2021, at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 5709209.

On April 20, 2021 Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that results from four clinical studies of the company’s three apoptosis-targeted drug candidates have been selected for presentations at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Ascentage Pharma, APR 20, 2021, View Source [SID1234578268]). These include two oral presentations: one based on a global Phase I clinical study of the Bcl-2 inhibitor lisaftoclax (APG-2575); and the other based on a global Phase II clinical trial of the MDM2-p53 inhibitor alrizomadlin (APG-115) in combination with the PD-1 checkpoint inhibitor pembrolizumab.

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Convening on June 4 to 8, 2021, the ASCO (Free ASCO Whitepaper) Annual Meeting showcases the most cutting-edge research in clinical oncology and the most advanced cancer therapies, and is the world’s most influential and prominent scientific gathering of the clinical oncology community.

"This year marks the fourth consecutive year in which our clinical study results were selected for presentations at the ASCO (Free ASCO Whitepaper) Annual Meeting, and we are pleased to have an opportunity to share multiple progress from our clinical development programs," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "We are thrilled that the results from two of our clinical studies have been selected for oral presentations at the meeting this year. Through these oral presentations, we will release the data from the Phase I study of lisaftoclax at a prominent scientific event for the first time. This signifies the drug candidate’s therapeutic potential and the global research community’s strong interest in this novel therapeutic, and shows Ascentage Pharma’s capability in the global research and development of apoptosis-targeted cancer therapies. We look forward to sharing detailed results during the meeting. Moving forward, we will strive to accelerate our clinical development programs to hopefully soon provide cancer patients with more treatment options."

At this year’s ASCO (Free ASCO Whitepaper) Annual Meeting, Ascentage Pharma will present results from four clinical studies of the company’s three apoptosis-targeted drug candidates as follows:

The novel Bcl-2 inhibitor lisaftoclax (APG-2575)

Lisaftoclax is a novel, orally administered small-molecule Bcl-2‒selective inhibitor being developed by Ascentage Pharma. Lisaftoclax is designed to treat hematologic malignancies and solid tumors by selectively blocking antiapoptotic protein Bcl-2 to restore the normal apoptosis process in cancer cells. Lisaftoclax is the first China-developed Bcl-2 inhibitor entering clinical development in China.

Selected study: A global Phase I clinical study of lisaftoclax, a novel Bcl-2 inhibitor, in patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL) and other hematologic malignancies (HMs)
Format: Oral Presentation
This global multi-center, single-agent, open-label Phase I clinical study is designed to assess the safety, pharmacokinetics (PK), pharmacodynamic (PD), and efficacy of lisaftoclax, and to determine its maximum tolerated dose (MTD)/recommended Phase II dose (RP2D) in patients with R/R CLL and other HMs.

In the preliminary data released by Ascentage Pharma in December 2020, lisaftoclax has demonstrated an objective response rate (ORR) of 70%, with favorable tolerability and manageable safety profiles in patients with R/R CLL. Detailed updated results will be released in an oral presentation at the ASCO (Free ASCO Whitepaper) Annual Meeting during June 4 – 8, 2021.

The novel MDM2 inhibitor alrizomadlin (APG-115)

Being developed by Ascentage Pharma, alrizomadlin is an orally administered, selective, small-molecule inhibitor of the MDM2 protein. Alrizomadlin has strong binding affinity to MDM2 and is designed to activate tumor suppression activity of p53 by blocking the MDM2-p53 protein-protein interaction. Alrizomadlin is the first MDM2-p53 inhibitor entering clinical development in China, and is currently being investigated in multiple Phase Ib/II clinical studies in solid tumors and HMs in China, Australia and the US.

Selected study: A Phase II study of alrizomadlin in combination with pembrolizumab in patients with unresectable or metastatic melanoma or advanced solid tumors that have failed immuno-oncologic drugs
Format: Oral Presentation
This study is designed to evaluate the efficacy and safety of alrizomadlin in combination with pembrolizumab in patients with unresectable or metastatic melanoma or advanced solid tumors that have failed immuno-oncologic drugs.

The results of this Phase Ib study released at the 2020 ASCO (Free ASCO Whitepaper) Annual Meeting demonstrated that alrizomadlin in combination with pembrolizumab is well-tolerated, with preliminary antitumor activity in advanced solid tumors.

Selected study: A Phase I/II trial of alrizomadlin, with or without platinum chemotherapy, in patients with p53 wild-type salivary gland carcinoma
Format: Poster Presentation
This multi-center, open-label Phase I/II study in the US is designed to evaluate the efficacy of alrizomadlin, with or without platinum chemotherapy, in patients with p53 wild-type salivary gland carcinoma.

The Bcl-2/Bcl-xL inhibitor pelcitoclax (APG-1252)

Pelcitoclax is a novel, highly potent, small molecule drug designed to restore apoptosis through selective inhibition of Bcl-2 and Bcl-xL proteins.

Selected study: A multi-center Phase Ib/II study of pelcitoclax plus paclitaxel in patients with relapsed/refractory small-cell lung cancer (R/R SCLC)
Format: Poster Presentation
This multi-center, open-label Phase Ib/II study is designed to evaluate the safety and preliminary efficacy of combination therapy with pelcitoclax plus paclitaxel in patients with R/R SCLC.

On April 20, 2021 Moderna, Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported a new supply agreement with Israel for 2022 (Press release, Moderna Therapeutics, APR 20, 2021, View Source [SID1234578267]). Under the terms of this agreement, Israel also retains an option to purchase doses of one of Moderna’s variant-specific vaccine candidates subject to regulatory approval. Today’s announcement follows two earlier agreements between Israel and Moderna to supply a total of 10 million doses of the COVID-19 Vaccine Moderna. The Israeli Ministry of Health authorized COVID-19 Vaccine Moderna for use on January 4, 2021.

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"We appreciate the continued confidence and collaboration with the Israel Ministry of Health with this new agreement," said Stéphane Bancel, Chief Executive Officer of Moderna. "This is an important moment for our company as the first firm order for 2022 supply and for the supply of our variant-specific booster vaccine candidates against COVID-19, currently being studied in human clinical trials. Recent preclinical results have shown that our variant-specific booster candidates were effective against COVID-19 variants of concerns, and we hope to continue to see positive results from the clinical studies."

About the COVID-19 Vaccine Moderna

The COVID-19 Vaccine Moderna (referred to in the U.S. as the Moderna COVID-19 Vaccine) is an mRNA vaccine against COVID-19 encoding for a prefusion stabilized form of the Spike (S) protein, which was co-developed by Moderna and investigators from the National Institute of Allergy and Infectious Diseases’ (NIAID) Vaccine Research Center. The first clinical batch, which was funded by the Coalition for Epidemic Preparedness Innovations, was completed on February 7, 2020 and underwent analytical testing; it was shipped to the National Institutes of Health (NIH) on February 24, 42 days from sequence selection. The first participant in the NIAID-led Phase 1 study of the Moderna COVID-19 Vaccine was dosed on March 16, 63 days from sequence selection to Phase 1 study dosing. On May 12, the U.S Food and Drug Administration granted the Moderna COVID-19 Vaccine Fast Track designation. On May 29, the first participants in each age cohort: adults ages 18-55 years (n=300) and older adults ages 55 years and above (n=300) were dosed in the Phase 2 study of the vaccine. On July 8, the Phase 2 study completed enrolment.

Results from the second interim analysis of the NIH-led Phase 1 study of the Moderna COVID-19 Vaccine in the 56-70 and 71+ age groups were published on September 29 in The New England Journal of Medicine. On November 30, 2020, Moderna announced the primary efficacy analysis of the Phase 3 study of the vaccine conducted on 196 cases. On November 30, 2020, the Company also announced that it filed for Emergency Use Authorization with the U.S. FDA and a Conditional Marketing Authorization (CMA) application with the European Medicines Agency. On December 18, 2020, the U.S. FDA authorized the emergency use of the Moderna COVID-19 Vaccine in individuals 18 years of age or older. Moderna has also received authorization for its COVID-19 vaccine from health agencies in Canada, Israel, the European Union, the United Kingdom, Switzerland, Singapore, Qatar and Taiwan. Additional authorizations are currently under review in other countries and by the World Health Organization.

Preclinical data on the Company’s variant-specific booster vaccine candidates have been submitted as a preprint to bioRxiv and will be submitted for peer-reviewed publication. These variant-specific vaccine candidates include mRNA-1273.351, which is more specifically targeted against the SARS-CoV-2 variant known as B.1.351 first identified in the Republic of South Africa, and a multivalent booster candidate, mRNA-1273.211, which combines mRNA-1273 (Moderna’s authorized vaccine against ancestral strains) and mRNA-1273.351 in a single vaccine. The Company’s Phase 2 study to evaluate three approaches to boosting is ongoing.

Authorized Use

Moderna has received approval to import and market COVID-19 Vaccine Moderna in Israel under Regulation 29 (A)(9). COVID-19 Vaccine Moderna is approved as a two-dose series for patients 18 years of age and older.