On May 26, 2020 Imago BioSciences, Inc. ("Imago"), a clinical stage biopharmaceutical company developing innovative treatments for myeloid diseases, reported that positive Phase 2 data from its lead pipeline program bomedemstat (IMG-7289), will be presented at the Virtual Edition of the 25th EHA (Free EHA Whitepaper) Annual Congress beginning June 12, 2020 (Press release, Imago BioSciences, MAY 26, 2020, View Source [SID1234558488]).

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Title: A PHASE 2 STUDY OF BOMEDEMSTAT (IMG-7289), A LYSINE-SPECIFIC DEMETHYLASE-1 (LSD1) INHIBITOR, FOR THE TREATMENT OF LATER-STAGE MYELOFIBROSIS (MF)

Session Topic: 16. Myeloproliferative Neoplasms

Final Abstract Code: EP1080

The data demonstrates the potential of bomedemstat as a monotherapy in intermediate-2 and high-risk patients with myelofibrosis who have become intolerant of, or resistant to, or are ineligible for a Janus Kinase (JAK) inhibitor.

Imago is currently conducting a Phase 2 study of bomedemstat in five countries. Clinical endpoints include spleen volume reduction, reduction in total symptom scores, and improvement in circulating inflammatory cytokines, anemia, bone marrow fibrosis and blast count. For additional information, visit cliniciatrials.gov (NCT03136185).

About Bomedemstat (IMG-7289)

Bomedemstat is being evaluated in an open-label Phase 2 clinical trial for the treatment of advanced myelofibrosis (MF), a bone marrow cancer that interferes with the production of blood cells. The endpoints include spleen volume reduction and symptom improvement at 12 and 24 weeks of treatment. Bomedemstat is used as monotherapy in patients who are resistant to, intolerant of, or ineligible for a Janus Kinase (JAK) inhibitor.

Bomedemstat is a small molecule developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme shown to be vital in cancer stem/progenitor cells, particularly neoplastic bone marrow cells. In non-clinical studies, IMG-7289 demonstrated robust in vivo anti-tumor efficacy across a range of myeloid malignancies as a single agent and in combination with other chemotherapeutic agents. Bomedemstat (IMG-7289) is an investigational agent currently being evaluated in ongoing clinical trials (ClinicalTrials.gov Identifier: NCT03136185 and NCT02842827). Bomedemstat has FDA Orphan Drug and Fast Track Designation for the treatment of myelofibrosis and essential thrombocythemia, and Orphan Drug Designation for treatment of acute myeloid leukemia.

On May 26, 2020 Qurient Co. Ltd. (KRX: 115180), a clinical stage biotech company in Korea, reported that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for Q702, orally available immuno-oncology therapeutic small molecule targeting Axl, Mer and CSF1 receptor tyrosine kinases (Press release, Qurient Therapeutics, MAY 26, 2020, View Source [SID1234558487]).

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Under this IND, Qurient plans to initiate a Phase 1 clinical study in patients with advanced solid tumors for whom standard of care therapies are currently ineffective. The Phase 1 study is expected to begin in 3Q2020 and is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of Q702. The study will be conducted at multiple clinical centers in the United States.

"IND clearance for Q702 is an important milestone presenting a novel drug candidate that not only boosts immune cells in the tumor microenvironment but also makes tumor cells more visible to the immune system," said Kiyean Nam, Ph.D., CEO of Qurient. "We believe Q702 may have an important role in the cancer immunotherapy, improving clinical responses in patients who are unresponsive and/or refractory to currently available immunotherapy."

Q702 is an orally available, selective Axl/Mer/CSF1R triple kinase inhibitor showing significant in vivo activity as monotherapy as well as in combination with anti-PD-1 antibody. Q702 not only modulates innate immune components such as myeloid derived suppressor cell (MDSC), tumor associated macrophage (TAM) in tumor micro-environment (TME), but also increases MHC I expression in tumor cell.

The Axl inhibitor program was licensed from Lead Discovery Center (LDC) and the Max-Planck Society at lead stage and further optimized by Qurient. The research program initially originated from Professor Axel Ullrich’s laboratory from the Max Planck Institute of Biochemistry, Martinsried/Germany.

"We are excited to see the progress in this project and are looking forward to the application in humans in the near future. With Qurient, we have identified an ideal partner for this project and we are more than happy about the results of our strategic partnership with them," said Matthias Stein-Gerlach, Senior Patent and Licensing Manager at Max-Planck Innovation GmbH.

"Reaching a clinical candidate for development is one of the most important milestones in our partnerships," adds Bert Klebl, CEO and CSO of the LDC. "Starting an early-stage collaboration with Ullrich’s lab from Max Planck, leading to a licensing agreement with Qurient, we jointly mastered the pharmaceutical research phase and are now very eager to receive the results from this drug candidate in patients. Starting with this program, we have since built a sustainable and strong partnership with our partner Qurient, focusing on the translation of innovative biology and drug discovery programs from LDC’s academic network."

On May 26, 2020 Monte Rosa Therapeutics, a biotechnology company developing small molecules to degrade disease-related proteins, reported stealth mode revealing a $32.5 million Series A commitment from founding investor Versant Ventures and New Enterprise Associates. Monte Rosa was launched from Ridgeline, Versant’s Discovery Engine based in the Basel Technology Park (Press release, Monte Rosa Therapeutics, MAY 26, 2020, View Source [SID1234558486]). The company is now headquartered in Boston, MA, with research operations in both Boston and Basel, Switzerland.

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Monte Rosa has developed a platform to rationally design small molecules that reprogram ubiquitin ligases to eliminate disease drivers previously deemed undruggable. The platform includes computational capabilities to predict and model ligase-neosubstrate interactions and quantitative proteomics to obtain protein degradation profiles.

Expanding the horizon for cereblon reprogramming

In 2018, Monte Rosa was established in Versant’s Ridgeline laboratories in collaboration with The Institute of Cancer Research (ICR) and Cancer Research UK. Academic co-founders Rajesh Chopra, MD, and Ian Collins, PhD, are prominent leaders in the field of protein degradation.

Nicolas Thomä, PhD, a world-leading chemical and structural biologist at the Friedrich Miescher Institute in Basel, also joined Monte Rosa as scientific advisor. Dr. Thomä recently published a series of seminal papers revealing mechanistic details of cereblon-mediated protein degradation. His work demonstrated the pivotal role of a glycine loop degron on target proteins, and opened up the potential for drug discovery on many more disease-relevant targets. This work underpins Monte Rosa’s platform and approach.

"With our improved understanding of the broad potential of cereblon and other ubiquitin ligases, there is now an opportunity to eliminate major – and currently undruggable – drivers of solid tumors such as transcription factors and adaptor proteins," said Markus Warmuth, MD, CEO of Monte Rosa and a venture partner at Versant.

Developing drug candidates with novel degradation profiles

Monte Rosa has built an integrated drug discovery platform that combines one of the most diverse chemical libraries of protein degraders with in-house proteomics and structural biology capabilities. By the end of 2020, the chemical library is expected to grow to more than 10,000 structures designed for ubiquitin ligase reprogramming.

To date, several validated small molecule leads have been identified through conventional and phenotypic screens along with rational drug design. A number of these leads possess novel degradation profiles and have demonstrated in vivo efficacy across several different tumor models.

"Using this approach we can potentially design drugs for the hundreds to thousands of proteins with glycine loop degrons," said Alex Mayweg, PhD, managing director at Versant and Monte Rosa board member. "Unlike protacs, these small molecules can degrade proteins that lack classical drug-binding pockets, which include known drivers of certain forms of cancer and other serious diseases."

Operating and financing plans

Monte Rosa plans to build out its platform and concurrently develop a portfolio of drug candidates for multiple indications. One of its portfolio leads, MRT-048, has demonstrated a differentiated degradation profile and promising in vivo activity in several models of resistant breast cancer. The molecule is undergoing further preclinical development and safety testing. The company expects to file one or more IND submissions during 2021.

"Monte Rosa has built a formidable drug discovery capability and portfolio based on access to unique insights into ubiquitin ligase reprogramming," said Ali Behbahani, MD, general partner at NEA and Monte Rosa board member. "This approach is now demonstrating the potential to impact patients with intractable forms of cancer."

The company will soon launch a Series B financing to back the development of multiple clinical-stage programs. To capture the full potential of its technology platform, Monte Rosa also will explore discovery-stage pharma collaborations.

On May 26, 2020 SQZ Biotechnologies (SQZ), a cell therapy company developing novel treatments for multiple therapeutic areas, reported that Armon Sharei, PhD, chief executive officer, will be presenting at the following upcoming virtual investor conferences (Press release, SQZ Biotech, MAY 26, 2020, View Source [SID1234558485]):

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Jefferies Virtual Healthcare Conference
Tuesday, June 2, 2020
Presentation Time: 3:30pm-3:55pm ET

Raymond James Human Health Innovations Conference
Wednesday, June 17, 2020
Cell Therapy Panel Time: 12:00pm ET

Dr. Sharei and Teri Loxam, chief financial officer will also be holding one on one meetings during the conferences.

On May 26, 2020 Ribon Therapeutics, a clinical stage oncology company developing first-in-class therapeutics targeting stress response pathways, reported that Victoria Richon, Ph.D., President and CEO, will provide a corporate overview and an update on the company’s lead clinical program, PARP7 inhibitor RBN-2397, at the Jefferies 2020 Virtual Healthcare Conference (Press release, Ribon Therapeutics, MAY 26, 2020, View Source [SID1234558484]).

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Jefferies Virtual Healthcare Conference – June 2-4, 2020
Corporate Presentation
Presenter: Victoria Richon, Ph.D. – President and CEO
Date: June 2, 2020
Time: 3:00 p.m. EDT