On September 30, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported that David Baltimore, Ph.D., a director of Regulus, is the recipient of the 2021 Lasker-Koshland Special Achievement Award in the category of Medical Science (Press release, Regulus, SEP 30, 2021, View Source [SID1234590618]).

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Dr. Baltimore’s recognition is based on the breadth and substance of his discoveries in virology, immunology, and cancer as well as his leadership within the academic and scientific communities and his extensive work as a public advocate for science.

The Lasker Awards program honors discoveries and clinical advances that improve human health and highlights the importance of public support for science. The awards program is widely considered one of the most prestigious awards programs in medical science and numerous recipients, including Dr. Baltimore, also hold Nobel Prizes.

"On behalf of the Regulus team, we congratulate David for this recognition of his extensive and impressive body of work," said Jay Hagan, President and Chief Executive Officer of Regulus. "As a 1975 Nobel Prize winner in Physiology or Medicine, David has made multiple foundational scientific discoveries over the course of his career, including the discovery of reverse transcriptase, as well as his additional research, which has contributed broadly to our understanding of cancer, AIDS, and the underlying biology of the immune response. He has been a powerful advocate for science over the years, advancing work that helped America respond to the AIDS crisis, and has played a significant role in the development of the biotechnology industry in America. We are proud that he is a part of Regulus and continually benefit from his guidance as we advance our pipeline of microRNA therapeutics."

More information about the foundation and the Special Achievement Award can be found here.

About David Baltimore, Ph.D.

Dr. David Baltimore is a Director of Regulus and President Emeritus and Distinguished Professor of Biology at Caltech. Awarded the Nobel Prize in 1975, Dr. Baltimore has profoundly influenced national science policy on such issues as recombinant DNA research and the AIDS epidemic. Dr. Baltimore graduated from Swarthmore College with a degree in chemistry. He took graduate courses at Massachusetts Institute of Technology and received his Ph.D. from Rockefeller University. He was a postdoctoral fellow at MIT and Albert Einstein College of Medicine. He was a Research Associate at The Salk Institute in 1965 and joined the faculty at MIT in 1968. He has served as Director of the Whitehead Institute for Biomedical Research, President of Rockefeller University, and President of Caltech. He was awarded the 1999 National Medal of Science and 2000 Warren Alpert Foundation Prize. He is a member of the National Academy of Sciences, fellow of the American Academy of Arts and Sciences, and a foreign member of the Royal Society of London and the French Academy of Sciences. He has also been President and Chair of the American Association of the Advancement of Science.

On September 30, 2021 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported three poster presentations at the AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) to be held October 7-10, 2021 (Press release, ORIC Pharmaceuticals, SEP 30, 2021, View Source [SID1234590617]).

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Details of the poster presentations are as follows:

Title: Initial results from a Phase 1b study of ORIC-101, a glucocorticoid receptor antagonist, in combination with enzalutamide in patients with metastatic prostate cancer
Poster #: P041

Title: Biomarker results supporting selection of RP2D from a Phase 1b study of ORIC-101, a glucocorticoid receptor antagonist, in combination with enzalutamide in patients with metastatic prostate cancer progressing on enzalutamide
Poster #: P015

Title: ORIC-114, an orally bioavailable, irreversible kinase inhibitor, has superior brain penetrant properties and enhanced potency in preclinical studies of HER2-positive breast cancer
Poster #: P234
Full abstracts and poster presentations will be available for on-demand viewing via the online platform for AACR (Free AACR Whitepaper)-NCI-EORTC on October 7, 2021, at 9 a.m. ET.

Company Webcast and Conference Call

ORIC will host a webcast and conference call to review data presented at AACR (Free AACR Whitepaper)-NCI-EORTC on Thursday, October 7, 2021, at 9:00 a.m. ET. To participate in the conference call, please dial (833) 651-0991 (domestic) or (918) 922-6080 (international) and refer to conference ID 3575856. A live webcast and audio archive of the conference call will be available through the investor section of the company’s website at www.oricpharma.com. The webcast will be available for replay for 90 days following the presentation.

On September 30, 2021 Sutro Biopharma, Inc. (NASDAQ: STRO), a clinical-stage drug discovery, development and manufacturing company focused on the application of precise protein engineering and rational design to create next-generation cancer and autoimmune therapeutics, reported that Merck, known as MSD outside the United States and Canada, has extended the research term for the first cytokine derivative program under the 2018 Merck Agreement, for an additional two years (Press release, Sutro Biopharma, SEP 30, 2021, View Source [SID1234590607]). The research extension is intended to facilitate completion of preclinical research and development activities for a second candidate, which has a novel design and approach. As part of this extension, Sutro is eligible to receive up to $10 million.

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"We are encouraged by the strength of the continued research efforts in collaboration with Merck on the cytokine derivative programs," said Trevor Hallam, President of Research and Chief Scientific Officer of Sutro. "This research extension has the potential to produce an additional candidate using a different approach towards the same target. We believe that this productive research program underscores the potential of Sutro’s platform to engineer therapeutics with novel design and approach."

About the Merck Collaboration
Under the terms of the 2018 Merck collaboration agreement, Sutro and Merck are conducting preclinical research and development of cytokine derivatives utilizing Sutro’s proprietary cell-free protein synthesis and site-specific conjugation platforms, XpressCF and Xpress CF+. Merck has exclusive worldwide rights to therapeutic candidates derived from the collaboration. In March 2020, Merck exercised its option to extend the research term of the first collaboration program by one year, which generated a payment of $5 million to Sutro. In April 2021, Merck initiated IND-enabling toxicology studies for the first candidate under the first collaboration program for which Sutro earned a $15 million milestone payment. Additionally, research on a second cytokine derivative program on a separate target is ongoing.

On September 30, 2021 I-Mab (the "Company") (Nasdaq: IMAB), a clinical-stage biopharmaceutical company committed to the discovery, development and commercialization of novel biologics, reported multiple clinical advancements of its anti-CD47 monoclonal antibody lemzoparlimab (also known as TJC4) (Press release, I-Mab Biopharma, SEP 30, 2021, View Source [SID1234590606]).

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I-Mab has initiated a phase 2 expansion trial (NCT03934814) of lemzoparlimab in combination with rituximab (Rituxan) in non-Hodgkin’s lymphoma (NHL) patients in China. The expanded trial is part of the ongoing international multi-center trial (IMCT) that is being conducted in the U.S. and now also in China. The study is designed to evaluate the safety, pharmacokinetics (PK), pharmacodynamic (PD) and determine the recommended phase 2 dose (RP2D) of lemzoparlimab in combination with rituximab in patients with lymphomas. On September 28, 2021, the first patient in the expanded trial was dosed. Patient enrollment for the trial is expected to be completed in a few months.

Progress in clinical trials of lemzoparlimab in NHL and AML/MDS:

In the U.S. clinical trial of lemzoparlimab in combination with rituximab for NHL patients, the preliminary efficacy and safety data have been summarized and submitted for presentation at the 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The expanded clinical trial is on track and expected, pending approval by the NMPA, to lead to a registrational trial in patients with NHL in 2022 in China.
In the China clinical trial of lemzoparlimab in combination with azacitidine (AZA) in patients with myelodysplastic syndrome (MDS), preliminary clinical response data is being evaluated, and patient enrollment is on track for completion in Q4 2021, the complete data set will be analyzed and reported at a later time. The Company plans to initiate another registrational trial in patients with MDS in 2022 in China based on the efficacy and safety data from this study, pending approval by the NMPA.
Progress in clinical trials of lemzoparlimab in patients with solid tumors:

I-Mab is currently investigating lemzoparlimab in combination with pembrolizumab (Keytruda) in advanced solid tumors in U.S. Data readout is expected in early 2022. Further, on September 16, the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) approved the Company’s IND application to advance to a phase 2 clinical trial of lemzoparlimab in combination with toripalimab (TUOYI) in patients with advanced solid tumors. The combined clinical results will potentially support a registrational trial later in China.

In all clinical trials conducted so far by the Company, including NHL, AML/MDS and solid tumors, lemzoparlimab have been evaluated without the need of a priming dose.

"Accumulative data from the ongoing lemzoparlimab clinical trials further increase our understanding of its safety, PK and efficacy profile," said Dr. Joan Shen, CEO of I-Mab. "We are encouraged by the clinical data of lemzoparlimab obtained so far and are rapidly advancing the clinical development of lemzoparlimab towards multiple registrational trials, with the goal of becoming the first CD47 antibody drug in China."

About CD47 and Lemzoparlimab

CD47 is a cell surface protein over-expressed in a wide variety of cancers and can act to protect tumors by delivering a "don’t eat me" signal to otherwise tumor-engulfing macrophages. CD47 antibody blocks this signal and enables macrophages to attack tumor cells. However, development of CD47 antibody as a cancer therapy is hampered by its hematologic side effects, such as severe anemia, caused by natural binding of CD47 antibody to red blood cells. Scientists at I-Mab have discovered a novel CD47 antibody, lemzoparlimab, that is designed to target tumor cells while exerting a minimal untoward effect on red blood cells.

I-Mab continues to advance a combination study of lemzoparlimab with Keytruda for solid tumors in the U.S. and with Rituxan for lymphoma in the U.S. and China, in addition to an on-going clinical trial in patients with AML in China.

In September 2020, I-Mab and AbbVie entered into a global strategic collaboration to develop and commercialize lemzoparlimab, including to design and conduct further clinical trials to evaluate lemzoparlimab in multiple cancers globally and in China. AbbVie has assumed sponsorship of the U.S. study as of April 2021.

On September 30, 2021 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, reported the dosing of the first patients in its Berubicin clinical development program for the treatment of recurrent glioblastoma multiforme (GBM), one of the most aggressive types of brain cancer (Press release, CNS Pharmaceuticals, SEP 30, 2021, View Source [SID1234590605]). Further patient enrollment, randomization and dosing is currently underway as well as a robust lineup of clinical sites located globally which are advancing toward activation and enrollment.

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"As one of the most aggressive, deadly and treatment-resistant cancers that forms in the brain where current standard of care remains ineffective in ~60% of patients, the treatment landscape for GBM is in desperate need of a better treatment option. We continue to be encouraged by the data seen from Berubicin to date and are excited to now have patient dosing underway in this potentially pivotal trial. Our team is committed to expeditiously advancing the study and look forward to further understanding the full potential of Berubicin to offer improvement and overall survival in this devastating disease," stated Erin Dunbar, M.D., founding physician of the Brain Tumor Center and Director of Neuro-Oncology at Piedmont Atlanta Hospital, and Principal Investigator for the study.

Berubicin is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier currently being evaluated in a global potentially pivotal study evaluating its efficacy and safety. The potentially pivotal trial is an adaptive, multicenter, open-label, randomized and controlled study in adult patients with recurrent glioblastoma multiforme (WHO Grade IV) after failure of standard first-line therapy. Approximately 243 patients with GBM after failure of standard first line therapy will be randomized in a 2:1 ratio to receive Berubicin or lomustine for the evaluation of Overall Survival, the primary endpoint of the study. Overall Survival is a rigorous endpoint that the U.S. Food and Drug Administration (FDA) has recognized as a basis for approval of oncology drugs when a statistically significant improvement can be shown relative to a randomized control arm.

In the Phase 1 clinical trial previously conducted evaluating Berubicin in patients with previously treated disease, 44% of the patients showed clinical benefit (49% of the Avastin-naïve patient population), with the demonstration that it was effective based on this patient population with a dismal median survival rate of only 14.6 months from diagnosis. One patient in the Phase 1 study had a durable Complete Response (CR, a demonstrated lack of detectable cancer cells) that has continued for 14 years, and another patient had a durable partial response, with others showing substantial stabilization of disease. Additionally, the novel anthracycline agent Berubicin appears to have a toxicity profile consistent with that of other anthracyclines and demonstrates activity as monotherapy for recurrent glioblastoma multiforme. Berubicin has side effects that are able to be effectively treated and managed.

"I am extremely pleased with the progress made to-date in this potentially pivotal trial. Our team has been working intensely to open sites in the U.S. and in Europe, understanding where we can best advance this important study. With hundreds of potentially competing GBM trials currently enrolling patients, the fact that we’ve been able to bring these initial sites on-line and get patients enrolled and dosed not only supports our strategic evaluation and selection, but also allows our data demonstrating Berubicin’s potential effectiveness to continue to convince the medical community that we have a new drug with impressive potential. With the de-risked profile of Berubicin, its mechanism of action, history of development, encouraging Phase 1 data, and safety in study design, I am personally more optimistic about our work than at any time since joining the Company," commented John Climaco, CEO of CNS Pharmaceuticals.

"The investigator dedication to advance the science and development of Berubicin we’re seeing here is meaningful to these patients that are out of options. Berubicin provides an innovative option for treatment in GBM as a safe and potentially effective therapy. We look forward to providing data at the interim analysis of the study," added Sandra L. Silberman, M.D., Ph.D. Chief Medical Officer of CNS Pharmaceuticals.

A pre-planned, non-binding futility analysis will be performed after approximately 30 to 50% of all planned patients have completed the primary endpoint at 6 months. This review will include additional evaluation of safety as well as secondary efficacy endpoints. Enrollment will not be paused during this interim analysis.

The FDA recently granted CNS Pharmaceuticals Fast Track Designation for Berubicin which enables more frequent interactions with the FDA to expedite the development and review process. As previously announced, the Company also received Orphan Drug Designation from the FDA which may provide seven years of marketing exclusivity upon approval of an NDA. Taken together the Company believes these important designations can be seen as a recognition of the significance of not only the unmet clinical need in GBM, but of our Berubicin program.

For more information about the potentially pivotal Berubicin trial, visit clinicaltrials.gov and reference identifier NCT04762069.

About Berubicin

Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin treatment of brain cancer patients appeared to demonstrate positive responses that include one durable complete response in a Phase 1 human clinical trial conducted by Reata Pharmaceuticals, Inc. Berubicin, was developed by Dr. Waldemar Priebe, Professor of Medicinal Chemistry at The University of Texas MD Anderson Cancer Center.