On April 20, 2021 Case Western Reserve University and Atelerix Life Sciences Inc., a Charlottesville, Virginia-based biotechnology company, reported that they have signed a two-year option to license a novel family of small molecules called Active Thiol-Based Compounds (ATBC’s) to prevent or reverse life-threatening opioid-related side effects (Press release, Case Western Reserve University, APR 20, 2021, View Source [SID1234578254]).

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The lead compound, ATLX–0199 (also called sudaxine), will be first targeted at opioid-induced respiratory depression (OIRD) in the hospital setting.

A recent study by the medical technology company Medtronic found that 46% of surgical patients receiving opioids for post-operative pain-relief suffer one or more episodes of OIRD, presenting perhaps the most significant obstacle to discharge from intensive care units and the hospital generally.

"No compound now on the market is able to reverse OIRD without also eliminating pain relief—an unacceptable trade-off in the hospital setting," said David Kalergis, Atelerix CEO, co-founder and biotechnology entrepreneur. "This unmet need is well-recognized by medical practitioners, speeding the prospect of rapid adoption and reimbursement in this potential $1 billion market."

The ATBC family and ATLLX-0199 are the result of several decades of collaborative scientific discovery and research among the company’s three co-founders: Stephen Lewis, a professor in the Department of Pediatrics at the Case Western Reserve School of Medicine, Benjamin Gaston, formerly of Case Western Reserve and now Billie Lou Wood Professor of Pediatrics at Indiana University’s School of Medicine, and James Bates, associate professor of Anesthesia at the University of Iowa’s Carver College of Medicine. Bates will become chief medical officer of Atelerix.

Development of the technology has been supported by funding and technical assistance from the National Institutes of Health, Harrington Discovery Institute at University Hospitals and the National Heart, Lung, and Blood Institute’s Catalyze Program.

The ATBC technology harnesses new, cutting-edge understanding of the molecular pathways involved in the effects of opioids, both as a pain reliever and a source of life-threatening side-effects like suppressed breathing. Rather than affecting the opioid receptors directly, they target the inhibition of pathways responsible for the side effects, allowing a more selective therapeutic intervention.

Studies on animal models have shown that ATLX–0199 can be injected intravenously to immediately reverse the negative effects of opioids on breathing without causing withdrawal or reversing the effects of pain control. Long-term, the company expects to expand the technology for use in trauma and overdoses, as well as administering the drug nasally, through patches or other routes.

"As a practicing anesthesiologist, I deal with the problem of opioid-induced depression of breathing in my patients every day," Bates said. "In my new role as chief medical officer of Atelerix Life Sciences, I will be directly involved in developing a solution to this serious unmet medical need by helping speed sudaxine through the U.S. Food and Drug Administration-approval process and into widespread use in the hospital setting."

The two-year option to license agreement, managed through Case Western Reserve’s Technology Transfer Office (TTO), will allow pre-clinical testing of the technology, including safety profiling and feasibility of scaled-up manufacturing.

"After several years of supporting this project within the university, we believe our timely partnership with Atelerix will provide a product that will address the real needs of patients receiving opioids," said Stephanie Weidenbecher, TTO’s senior licensing manager. "Follow-on programs in trauma, military and overdose settings for sudaxine and other molecules in the ATBC family will present comparable opportunities."

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 Tubulis reported the appointment of Günter Fingerle-Rowson, MD, PhD, as Chief Medical Officer to complement the leadership team and to further build the company’s clinical expertise (Press release, Tubulis, APR 20, 2021, View Source [SID1234578239]). Dr. Fingerle-Rowson is an experienced hematologist and medical oncologist who brings to Tubulis more than two decades of academic, biotechnology and pharmaceutical industry experience. Moreover, he has a proven track record of advancing product candidates from early clinical phase through regulatory approval and into clinical practice. In this newly established role, he will use his extensive knowledge in the development of cancer therapeutics to oversee Tubulis’ clinical activities and advance the company’s Antibody Drug Conjugates (ADCs) towards clinical evaluation.

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"Günter is an expert in developing successful clinical oncology programs that resulted in the regulatory approval of several monoclonal antibodies. His wealth of experience and his track record of execution will be a great asset to Tubulis as we transition towards becoming an established clinical-stage drug developer," said Dr. Dominik Schumacher, CEO and co-founder of Tubulis. "In addition, his background in translational research in immuno-oncology will further strengthen our team and our capabilities to rapidly deliver the benefits of our ADC approach to cancer patients."

Dr. Fingerle-Rowson commented: "I am thrilled to join Tubulis to be part of the team that brings ADC technology to a new level. The technological advancements that the company has made in a short period of time and the team’s clear vision to become a leader in the field of ADCs instantly drew my interest. I look forward to help shape the clinical development plan for Tubulis’ exciting technology platform and to bring its first candidate TUB-010 to patients in the near term. Tubulis´ proprietary ADC technology enables us to develop novel ADCs that have a better versability and an improved benefit/risk profile than current ADC options."

Prior to joining Tubulis, Dr. Fingerle-Rowson worked in Global Clinical Development at MorphoSys and F. Hoffmann-La Roche as well as in Medical Affairs at Janssen-Cilag, a J&J company. He has contributed to bring three drugs to patients with malignancies. In his latest role as VP, Global Product Head at MorphoSys, he steered the clinical development as well as the regulatory approval for Tafasitamab (MOR208, Monjuvi). From 2011-2018, he served as Associate Medical Group Director, Global Development Team Leader and Global Clinical Leader at F. Hoffmann-La Roche where he was responsible for the clinical development and regulatory approvals of Obinutuzumab (GA101, Gazyva). Dr. Fingerle-Rowson also worked as Medical Manager at Janssen-Cilag, and as academic physician in internal medicine, hemato-oncology at the University Hospitals in Cologne and Munich where he also led own research in the field of immuno-oncology. Dr. Fingerle-Rowson was a longstanding active member of the German CLL Study Group where he contributed to the approval of Rituximab. He is a board-certified hematologist/medical oncologist and holds an MD in Internal Medicine from Munich University, and a MD-PhD in Molecular Medicine from New York University.

On April 20, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that the Company will host a webcast conference call to report its first quarter 2021 financial results and provide an update on the company’s business and outlook on Tuesday, May 4, 2021 at 4:30 p.m. (ET) after the closing of the market (Press release, PTC Therapeutics, APR 20, 2021, https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-host-conference-call-discuss-first-quarter-2021 [SID1234578255]).

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The call can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 4292410. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at View Source A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

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."