On April 13, 2021 Iterion Therapeutics, Inc., a venture-backed, clinical stage biotechnology company developing novel cancer therapeutics, reported that it has confirmed the safety of Tegavivint, a novel, potent and selective nuclear beta-catenin inhibitor, after completing enrollment and dosing the final patient in a multicenter Phase 1/2a dose expansion clinical study of Tegavivint in patients with desmoid tumors (Press release, Iterion Therapeutics, APR 13, 2021, View Source [SID1234577997]).

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Nuclear beta-catenin is a highly-studied oncology target associated with numerous cancer types. Tegavivint is unique among nuclear beta-catenin inhibitors in that it binds to TBL1 (Transducin Beta-like Protein One), a novel downstream target in the Wnt-signaling pathway. As such, Tegavivint enables silencing of Wnt-pathway gene expression without affecting other Wnt/beta-catenin functions in the cell membrane, thus avoiding toxicity issues common to other drugs in this pathway.

The Phase 1/2a clinical trial of Tegavivint in patients with progressive desmoid tumors was designed as an open-label, non-randomized dose-finding study. The primary objectives of the study were to evaluate the safety and tolerability of Tegavivint. Secondary objectives were to determine the durability of response (DOR) to Tegavivint after the achievement of best response. The total study enrolled 24 patients. During the dose expansion portion of the trial 16 of these patients were treated with a recommended Phase 2 dose (RP2D) that was established based on pharmacokinetic exposure levels and clinical responses in a recently completed Phase 1 study.

Data from patients treated in the dose expansion portion of the trial reaffirmed Tegavivint’s safety at the RP2D level. No dose-limiting toxicities or significant adverse events were observed. This data will enable Iterion to accelerate clinical activity in additional cancer indications where nuclear beta-catenin signaling has been identified as a potential therapeutic target, including AML, NSCLC, and certain pediatric cancers. Iterion expects to initiate clinical programs investigating Tegavivint for these indications in 2021.

"We have seen very good tolerability with no dose-limiting toxicities and no significant adverse events in escalating clinical doses," said Casey Cunningham, Chief Medical Officer of Iterion. "We are seeing a very strong safety signal in patients who have been on Tegavivint for over a year and are also observing tumor activity in patients. We continue to follow the patients that are still receiving treatment and look forward to sharing efficacy results at an upcoming medical conference."

Rahul Aras, CEO of Iterion, stated: "The completion of enrollment in the dose expansion phase of our desmoid tumor clinical trial and demonstration of safety and clinical activity at the RP2D represent important milestones in our clinical development of Tegavivint. We look forward to advancing the clinical development of Tegavivint in desmoid tumors as this disease target is greatly underserved. The results of this study also provide a ‘green light’ to initiate clinical development of Tegavivint in additional, high-value cancer settings, including AML, NSCLC, and certain pediatric cancers, that are characterized by nuclear beta-catenin overexpression."

About Desmoid Tumors

Desmoid tumors are rare, non- metastasizing sarcomas that overexpress nuclear beta-catenin, a historically "undruggable" oncology target implicated in cell proliferation, differentiation and immune evasion. An estimated 1,500 patients in the US are newly diagnosed with desmoid tumors each year. Desmoids are most commonly diagnosed in young adults between 30-40 years of age and are associated with significant morbidities, including severe pain, disfigurement, internal bleeding and organ damage, range of motion loss and, in rare cases, death. Iterion has received Orphan Drug Designation for Tegavivint to treat desmoid tumors, a disease for which there are no FDA approved therapies.

On April 13, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday, April 29, at 4:30 p.m. ET to discuss first quarter 2021 financial results and provide a general business update (Press release, BioMarin, APR 13, 2021, prnewswire.com/news-releases/biomarin-to-host-first-quarter-2021-financial-results-conference-call-and-webcast-on-thursday-april-29-at-430pm-et-301266955.html [SID1234577996]).

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Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.biomarin.com. A replay of the call will be archived on the site for one week following the call.

On April 13, 2021 Qilu Puget Sound Biotherapeutic Corp. (Sound Biologics), an emerging biotechnology company focused on developing a novel MabPair platform for antibody-based combination therapies for the treatment of cancer, inflammation and autoimmune disease, reported a clinical update on its first MabPair product PSB205 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, Sound Biologics, APR 13, 2021, View Source [SID1234577995]). Dr Li Zhang of Sun Yat-Sen University Cancer Centre (SYSUCC), Guangzhou, China presented a poster on Phase 1 Clinical Trial results of PSB205, a combination of anti-PD1 and anti-CTLA-4 antibodies that are manufactured together as a single product, in patients with advanced malignant tumors. The study is sponsored by Qilu Pharma in China.

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Details of the presentation:

Presentation Title: Development and Preliminary Clinical Activity of QL1706 (PSB205), a Combination of anti-PD1 and anti-CTLA-4 Antibodies Manufactured Together as a Single Product

Session Title/Category: Phase I Clinical Trials

Session Type: E-Poster Session, 8:30 AM – 11:59 PM, Saturday, April 10

Poster Number: CT119

Forty-seven patients, 95% of whom had refractory Naso-Pharyngeal (NPC) or lung (NSCLC) cancers, were enrolled at a single clinic with over 5 ascending dose levels in a standard 3+3 design. A dose of 5 mg/kg was selected for continued clinical development. The most common side effects were Grade 1 pruritus and rash. Dose-limiting immune-related reactions occurred only at doses higher than 5 mg/kg. The Overall Rate of Response (ORR) was 29% among 35 patients evaluable for tumor response; among 20 patients without prior exposure to checkpoint-targeted immunotherapy, 40 % achieved Partial Response (PR), whereas 2 of 10 patients (20%) previously treated and progressed on earlier immunotherapy realized PR. For 5 patients on study, pre-study checkpoint inhibitor exposure status was unknown due to earlier participation in a still-blinded randomized immunotherapy trial.

"The Phase 1 data presented at AACR (Free AACR Whitepaper) provides encouraging proof-of-concept and early clinical validation of the MabPair platform," commented Sound Biologics’ Chief Executive Officer Wei Yan, PhD. "The early data offer multiple opportunities for further development of this dual immune checkpoint blockade product."

About PSB205

PSB205 is a first-in-class bifunctional product that contains a mixture of unique anti-PD-1 and anti-CTLA-4 monoclonal antibodies produced by a single cell line via the company’s proprietary MabPairTM technology. MabPair products offer many advantages over bispecific antibodies. The relative ratio of the two antibodies in the MabPair can be well controlled, and each antibody is individually engineered for optimal target coverage, effector function, pharmacokinetics and exposure. The anti-CTLA-4 component of the MabPair is designed with a shorter half-life than currently available anti-CTLA-4 antibodies in an effort to reduce known side effects associated with CTLA-4 blockade. Preclinical studies with PSB205 demonstrated superior efficacy in tumor models compared to either checkpoint inhibitor alone. PSB205 represents a potentially best-in-class immuno-oncology product that promises to exhibit robust combination activity while being significantly more tolerable to patients than currently approved anti-PD-1/anti-CTLA-4 combinations. Parallel Phase 1 studies in a broad range of refractory solid tumors are currently ongoing in China and the United States (www.clinicaltrials.gov; NCT03986606), and the AACR (Free AACR Whitepaper) poster represents the first release of clinical data from one of these studies.

On April 13, 2021 HTL , world leader in biotechnology solutions based on hyaluronic acid (HA) and other biopolymers, and Echelon Biosciences Inc. , global supplier of biochemical reagents, tests and Services for US-based Research and Development Laboratories reported a new partnership (Press release, Echelon Biosciences, APR 13, 2021, View Source [SID1234577993]). This partnership concerns the distribution of HA and high quality glycosaminoglycans (GAGs) as well as the development of new GAGs.

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This collaboration allows university research and pharmaceutical development laboratories to have access to a wide range of products based on hyaluronic acid and glycosaminoglycans (GAG), compliant with good manufacturing practices (GMP). By providing researchers with superior quality, clinical grade products at the early stages of research, the partnership aims to bridge the gap between laboratory research, development and commercialization, and accelerate innovation around the world.

"Innovation is at the heart of HTL’s DNA," said HTL CEO Yvon Bastard . "This line of development is fully in line with our mission and our vision: to provide science today with the means to develop products that will improve health in the world in the future".

"This new partnership provides scientists and researchers with key technologies to develop innovative biopolymer solutions for new medical applications in the fields of aesthetics, ophthalmology, rheumatology, tissue engineering and drug administration, "said Charles Ruban , deputy CEO of HTL. "This is the true meaning of the signature of HTL: ‘Beyond, together’. HTL’s ambition is not only to develop its leading position in the production and distribution of hyaluronic acid around the world, but also to build the solutions of tomorrow based on glycosaminoglycans ".

"With hyaluronic acid and HTL’s other GAG-based products, Echelon is giving itself the opportunity to expand its line of industry-leading HA-based extracellular kits, tests and matrices. HTL is internationally recognized for its production of premium quality biopolymers. Says Bert Israelsen , President of Echelon Biosciences . "Echelon offers its customers access to innovative tools and unparalleled technical support, thereby maximizing creativity in basic and applied research. With its fermentation and organic synthesis platforms, HTL creates functionalities and added value by developing chemically modified biopolymers, targeted for specific needs ".

"This new partnership is an important step in the deployment of HTL in the North American market and to the American medical community," said Glenn Prestwich, Ph.D. , One of the founders of Echelon and today hui expert in science and innovation at HTL. By entrusting Echelon with the distribution of its hyaluronic acid and research glycosaminoglycans products, HTL aims to increase its support and participation in biopolymer research in the United States and globally, as well as its ability to research and development.

"This new initiative is a testament to HTL’s determination to invest in healthcare innovation," said Humberto C. Antunes , HTL strategic advisor and former CEO of Galderma. He added: "In the areas of health and wellness, the biotechnology ecosystem for innovation, incubators and established and emerging companies is incredibly vibrant in the United States, Mexico and Canada. . This means enormous growth potential for HTL’s products ".

On April 13, 2021 RubrYc Therapeutics, Inc., a pre-clinical biotherapeutics company developing epitope selective therapies, reported that it has entered into a research collaboration and license option agreement with Zai Labs, Inc. for the use of RubrYc’s Meso-scale Engineered Molecules (MEMs) platform, to identify monoclonal antibodies with enhanced biological function for an undisclosed oncology target (Press release, RubrYc Therapeutics, APR 13, 2021, View Source [SID1234577992]).

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Under the terms of the agreement RubrYc and Zai Labs will collaborate to identify product candidates using RubrYc’s Discovery Engine, which leverages machine learning and structural data to identify antibodies that bind to subdominant epitopes and exhibit differentiated activity. Upon exercise of the license option Zai Labs would have exclusive global rights to develop and commercialize product(s) of the Research Collaboration.

RubrYc Therapeutics, Inc. will receive an upfront payment and is eligible to receive future research, development and commercialization milestones on a per candidate basis, along with royalties on world-wide net sales of each product. Financial terms of the agreement were not disclosed.

Dr. Isaac Bright, CEO, said, "We are excited that Zai Labs has selected the RubrYc Discovery Engine as part of its efforts to identify biotherapeutics against challenging oncology targets. We look forward to collaborating with Zai Labs to accelerate their research by identifying epitope-specific antibodies with differentiated modes of action. Zai Labs’ oncology expertise complements our interest in bringing novel therapeutics with breakthrough potential to patients in need."